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Germany’s Ifo index has reached a one year high, which hints at a more confident backdrop for companies planning to grow earnings rather than just defend margins. That creates an interesting setting for investors looking for earnings momentum combined with balance sheet discipline. This article examines three stocks from the Healthy high growth potential screener and explains why this blend of growth and financial strength may be appealing at this point. The stocks covered below are only a first sample from this idea. The full screen surfaced 283 more companies with similarly strong growth expectations and balance sheet profiles that are not covered here. To go straight to the source, use the Healthy high growth potential screener to identify, analyze, and prioritize the highest conviction opportunities that fit your own criteria. Overview: Amylyx Pharmaceuticals is a clinical stage drug developer focused on treatments for rare endocrine and neurodegenerative diseases, with its Healthy high growth potential link coming from late stage programs like AMX0035 and avexitide that could move it from research focused spending to a commercial footing. The company is advancing a pipeline that includes AMX0035 for conditions such as Wolfram syndrome and progressive supranuclear palsy, as well as avexitide and other GLP 1 receptor antagonists for post bariatric hypoglycemia and related rare disorders. Market Cap: US$4.3b Investors looking at Amylyx Pharmaceuticals are getting exposure to a company whose growth story hinges on late stage trial assets that sit squarely in the Healthy high growth potential theme. The recent Phase 3 LUCIDITY success for avexitide in post bariatric hypoglycemia, with a reported 55% reduction in serious hypoglycemic events and a planned NDA filing by the end of 2026, provides a clearer line of sight to potential first commercial revenues. At the same time, Amylyx is still loss making, relies on external funding and has issued new equity to support commercialization and research, so dilution and execution risk are important considerations. The broader pipeline around AMX0035 and AMX0114 adds additional programs that could matter for earnings quality and durability depending on how future developments unfold. Amylyx Pharmaceuticals looks like a rare mix of high potential trial assets and real balance sheet questions that many investors may be glossing over. Before you lean into the story, review the 2 key rewards and 4 important warning signs (2 are major!) Overview: Oracle is a global enterprise software company best known for its cloud based applications and databases, with Oracle Fusion Cloud ERP, HCM, SCM and industry suites like NetSuite and Oracle Health giving it a strong link to the Healthy high growth potential theme as customers move from on premise software to subscription cloud services. Operations: Oracle generates the bulk of its revenue from Cloud and software at about US$58.5b, with smaller contributions from Services at about US$5.7b and Hardware at about US$3.1b. Market Cap: US$410.3b Oracle gives you exposure to two connected growth stories. Its cloud SaaS businesses in ERP, HCM and healthcare applications are central to analysts’ expectations for strong earnings and revenue growth over the next few years, and its AI ready Oracle Cloud Infrastructure and huge contracted backlog add another layer of potential. At the same time, this is a heavily leveraged balance sheet that leans on debt and planned capital raises to fund AI data centers, while free cash flow coverage of the dividend looks thin. For investors willing to accept execution and financing risk, the mix of high margin software, large AI contracts and a valuation that screens as attractive versus peers can be a compelling combination. Oracle’s accelerating cloud story can be easy to focus on, while the real swing factor may be hiding in how future cash flows match up with its debt load and dividend promises. Get the Oracle financial health report Overview: Iovance Biotherapeutics is a commercial stage biotech company focused on autologous tumor infiltrating lymphocyte cell therapies, led by Amtagvi for advanced melanoma and a late stage TIL pipeline that targets multiple solid tumors such as lung, cervical and endometrial cancers. This TIL platform is the clearest link to the Healthy high growth potential screener, as success in these indications is central to the company’s path to stronger earnings over the coming years. Operations: Iovance Biotherapeutics currently generates about US$325 million in revenue, all from its autologous TIL therapy business, with roughly US$321 million from the United States and about US$4 million from the rest of the world. Market Cap: US$3.7b Iovance Biotherapeutics gives you direct exposure to a commercial TIL therapy platform that already includes Amtagvi for advanced melanoma and a growing late stage pipeline. Analysts link this to rapid earnings growth and an expected move into profitability within 3 years. Recent quarters have highlighted strong Amtagvi driven revenue, improving margins and a fast expanding Authorized Treatment Center network, while analysts continue to raise targets as management reaffirms 2026 revenue guidance of US$350 to US$370 million. At the same time, the company still reports losses, relies on higher risk external funding and has diluted shareholders, so execution and financing risk remain central. For investors comfortable with biotech volatility, that mix of commercial traction, pipeline breadth and valuation sensitivity can be a compelling but high stakes proposition. Accelerating Amtagvi sales and a widening TIL pipeline make Iovance Biotherapeutics appear to be more than a high risk biotech swing. Get the full growth picture in the analyst forecasts for Iovance Biotherapeutics Fresh stock ideas can move from quiet to flying in a hurry. Use these curated screens before momentum gets fully caught by the crowd. Act now. Spot companies with steady fundamentals and low risk scores by running the 74 resilient stocks with low risk scores while they are still under the radar for now. Capture income opportunities that aim for durability by scanning the market with the 12 dividend fortresses before yields drop and attention surges. Explore potential moves in digital finance by checking the curated 20 cryptocurrency and blockchain stocks while the theme is still developing. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Have feedback on this article? Concerned about the content? Get in touch with us directly. 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Image source: The Motley Fool. Thursday, Aug. 6, 2026 at 8:00 a.m. ET Vice President, Investor Relations and Communications - Lindsey Allen Co-Chief Executive Officer - Justin Klee Co-Chief Executive Officer - Josh Cohen Chief Medical Officer - Camille Bedrosian Chief Commercial Officer - Dan Monahan Chief Financial Officer - James Frates Operator: Good morning. My name is Carrie, and I will be your conference operator today. At this time, I would like to welcome everyone to the Amylyx Pharmaceuticals Second Quarter 2026 Earnings Conference Call. [Operator Instructions] Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Lindsey Allen, Vice President, Investor Relations and Communications. Please proceed. Lindsey Allen: Good morning, and thank you all for joining us today to discuss our second quarter 2026 financial results and business updates. With me on the call today are Josh Cohen and Justin Klee, our Co-CEOs; Dr. Camille Bedrosian, our Chief Medical Officer; Dan Monahan, our Chief Commercial Officer; and Jim Frates, our Chief Financial Officer. Before we begin, I would like to remind everyone that any statements we make or information presented on this call that are not historical facts are forward-looking statements that are based on our current beliefs, plans, and expectations and are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, our expectations with respect to avexitide, AMX0035, AMX0114, and AMX0318, statements regarding regulatory and clinical development, the impact thereof and the expected timing thereof, and statements regarding our cash runway. Actual events and results could differ materially from those expressed or implied by any forward-looking statements. You are cautioned not to place any undue reliance on these forward-looking statements, and Amylyx disclaims any obligation to update such statements unless required by law. Now, I will turn the call over to Justin. Justin Klee: Good morning, everyone, and thank you for joining us. This is an exciting time for Amylyx and the post-bariatric hypoglycemia community as we approach the pivotal Phase III LUCIDITY top-line data readout. At the beginning of the year, we outlined 3 strategic priorities for avexitide, our investigational, first-in-class GLP-1 receptor antagonist with FDA breakthrough therapy designation in post-bariatric hypoglycemia or PBH. First, advancing the pivotal Phase III LUCIDITY trial toward top-line data. The last participant has recently completed their last visit in the trial. We are on track and eagerly anticipating the top-line data readout in late August or early September. The database has yet to be locked at this time, and we remain blinded to the data. Second, in parallel, advancing NDA readiness and regulatory preparations. We are actively preparing all applicable sections of the NDA to support a potential submission. And third, strengthening our launch readiness to prepare for the potential commercialization of avexitide in 2027, if approved. We've deployed our field medical affairs team to facilitate on-the-ground HCP scientific exchange, and we've continued to build out our teams across marketing, market access, and commercial operations. At ENDO 2026 in June, we activated our disease state education campaign, 'Uncover the Mystery of Post-Bariatric Hypoglycemia,' designed to increase awareness, understanding, and action around PBH. Dan will provide more details on our launch readiness efforts later in the call. We're fully focused on execution as we work toward the potential of delivering the first FDA-approved therapy for people living with PBH. With that, Camille, I'll turn the call over to you. Camille Bedrosian: Thank you, Justin. PBH is characterized by recurrent and often debilitating hypoglycemia thought to be caused by an exaggerated GLP-1 response, primarily after food intake. The American Diabetes Association recognizes hypoglycemia as a potential medical emergency because low blood glucose levels can compromise the body's ability to maintain essential physiologic processes and can result in cognitive dysfunction, seizures, or loss of consciousness. PBH is a chronic condition with no FDA-approved therapies. Many people with PBH live with the ongoing fear of hypoglycemia. Our pivotal Phase III LUCIDITY trial is a randomized, double-blind, placebo-controlled trial evaluating avexitide 90 mg once daily in adults with PBH following Roux-en-Y gastric bypass surgery. The LUCIDITY trial is anchored in the robust data generated to date from the 5 prior avexitide clinical trials in PBH that demonstrated statistically significant reductions in hypoglycemic events. The primary endpoint is the FDA-agreed-upon outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through week 16. At the ENDO 2026 Annual Meeting in June, several case reports describing many aspects of PBH and hypoglycemia were presented, highlighting a growing recognition of PBH and the seriousness of hypoglycemia across the medical community. Dr. Colleen Craig presented a poster characterizing the clinical, economic, and humanistic burden of PBH in the U.S. and evaluating how Level 2 and Level 3 hypoglycemic events in PBH impact healthcare utilization, productivity, and overall cost. At the patient level, patients face direct medical costs, productivity loss, such as missed work, diminished quality of life, caregiver burden, and out-of-pocket non-medical costs. At the systemic level, societies face resource utilization and downstream clinical consequences in addition to direct healthcare costs, non-medical system costs, and societal productivity loss. Also at ENDO 2026, we had meaningful scientific dialogue about PBH with endocrinologists that underscored the tremendous unmet need for people living with this condition. We met many endocrinologists with whom we had not previously engaged, and many of them already were aware of PBH. They describe the challenges in managing these individuals, given the limited options for treatment and had strong interest in learning more about this condition. This interest is inspiring as our field medical affairs team furthers PBH scientific exchange and engagement with endocrinologists. In addition, in June, CMS and CDC published their 2027 ICD-10 code files, which represent the official code set to be used for patient encounters beginning October 1, 2026. This code set includes an ICD-10 code specific to PBH, also demonstrating the growing recognition of this condition by the medical community. In closing, we are encouraged by the increasing awareness and understanding of PBH, along with our informative engagements with healthcare professionals. We continue to be focused on strong execution as we approach our anticipated LUCIDITY top-line data readout. With that, Dan, I'll now turn over the call to you. Dan Monahan: Thank you, Camille, and good morning, everyone. We continue to make significant progress in our preparations for the potential commercialization of avexitide next year. We remain encouraged by the growing recognition of PBH among healthcare professionals. Importantly, our understanding of the PBH population continues to strengthen. We estimate that approximately 160,000 people in the U.S. are living with PBH following the 2 most common bariatric procedures, Roux-en-Y gastric bypass and sleeve gastrectomy. This estimate remains supported by our independent claims analysis, ongoing field insights, and a growing body of published prospective and retrospective literature. Together, these data continue to reinforce both the significant unmet need and the opportunity to identify and reach appropriate patients. Our market research also continues to demonstrate a high intent among endocrinologists to treat PBH with an approved therapy, providing further confidence in the commercial opportunity should avexitide be approved. We are excited to share that we recently activated our disease state education campaign, 'Uncover the Mystery of Post-Bariatric Hypoglycemia,' to address the educational need among HCPs and the PBH community. As part of this initiative, we launched UncoverPBH.com. This platform provides educational resources for both healthcare professionals and the PBH community. The HCP website is intended to help clinicians better recognize and understand PBH. The community website empowers people with PBH with information to support discussions with their healthcare teams. As Camille mentioned, engagement at ENDO 2026 was high, where we also introduced an interactive disease state education experience that generated strong engagement and positive feedback. One of our most encouraging early observations was the level of familiarity many healthcare professionals already had with PBH, reinforcing our view that awareness of the condition continues to grow within the endocrinology community. In parallel, we continue to add key talent across the organization and advance our commercial readiness efforts as we prepare for the potential launch of avexitide in 2027, should it be approved. And with that, I will now turn the call over to Jim to review our financials. James Frates: Thanks, Dan. Good morning, everyone. Our financial results for the second quarter reflect our continued disciplined execution of the Phase III LUCIDITY trial and targeted investment in advancing our broader pipeline. We ended the second quarter with $250.8 million in cash and marketable securities, compared to $279.8 million at the end of the first quarter. This capital provides us with an anticipated cash runway into 2028 to fund our operations through expected milestones, including our key focus, LUCIDITY top-line readout, potential FDA approval, and potential commercial launch of avexitide in 2027. Turning now to our results for the quarter, total operating expenses for the quarter were $45.7 million, up 7% for the same period in 2025. Research and development expenses for the quarter were $23.8 million compared to $27.2 million in Q2 2025. The decrease was primarily due to a decrease in spending related to AMX0035 for the treatment of progressive supranuclear palsy, offset primarily by an increase in spending related to the clinical development of avexitide in PBH and other costs related to avexitide. Selling, general and administrative expenses for the quarter were $21.9 million, compared to $15.6 million in Q2 2025. This increase was primarily due to increased legal expenses, including a one-time legal charge and investment in commercial strategic initiatives. We recognized $8.3 million of non-cash stock-based compensation expense for the quarter compared to $7.4 million of non-cash stock-based compensation expense in Q2 2025. With the LUCIDITY top-line data readout later this month or early September, we're entering into an exciting phase. We're investing in a disciplined manner as we prepare for the potential launch of avexitide, and we believe we're well-funded to execute. With that, I'll turn the call over to Josh. Joshua Cohen: Thanks, Jim. In addition to avexitide, we continue to advance our broader pipeline and our commitment to the communities we serve with high unmet needs. For AMX0035 and Wolfram syndrome, we presented week 96 data from the Phase II open-label HELIOS clinical trial in the spring, which continued to show stabilization or improvement in measures of glycemic control and vision, consistent with week 24 and week 48 data. Under AMX0114 and ALS, we continue to advance the Phase I LUMINA multiple ascending dose clinical trial in people living with ALS. We are progressing through the dose cohorts given the favorable safety profile AMX0114 has exhibited in LUMINA so far. We are currently enrolling cohort 3. And for AMX0318, our long-acting GLP-1 receptor antagonist, IND-enabling studies are underway, and we are targeting a 2027 IND filing. Additionally, we are pleased to share we entered into a second research collaboration with Gubra in July, following the collaboration that identified AMX0318. This new collaboration will screen and develop peptide candidates for another rare endocrine disease of high unmet need. We're looking forward to sharing additional updates on this program as it advances. In closing, we are executing against our 3 strategic priorities for avexitide, including delivering top-line data from LUCIDITY, advancing our NDA readiness, and strengthening our commercial launch preparations. Guided by the profound unmet need of the PBH community, we are working with urgency to bring this potential treatment to people living with PBH. Before we turn it over to the operator for Q&A, I want to reiterate that this is an exciting time for Amylyx as we are eagerly awaiting Phase III top-line data. Because we are getting close to locking the LUCIDITY trial database, we will be entering a quiet period after this call. Operator: [Operator Instructions] We'll pause momentarily to assemble our roster. Your first question will come from Seamus Fernandez of Guggenheim. Seamus Fernandez: So I just have a couple here. You know, I believe maybe you guys could just update us on when the last patient visit was. And what are the, kind of, key factors that, kind of, need to be completed to lock the database? Where are the areas of, kind of, core focus, or is it the, sort of, need to get all of the sites, kind of, fully aligned to be able to lock the database? And then the second question is, I'm guessing you won't be able to update us on specific numbers, but maybe you can provide us a little bit of color on how the Early Access Program is progressing at this point. Are patients actively being recruited into the EAP? And, how would you kind of characterize the demand for that program at this point? It's obviously an opportunity to really put the company commercially on a very strong footing going forward. Camille Bedrosian: Great. Seamus, Hi. Thanks for the questions. So your first question revolved around last patient visit and what efforts need to continue to be ready for the database lock and then top-line data. If we start with when our last patient's first visit was, we announced we completed enrollment late March or mid-March, I would say. And so it's a 16-week double-blind period. So advance toward that. We get to about mid-late July. And there are, as you point out, rightly so, there are a number of activities that the team is working on, and they're continuing to execute beautifully and the enthusiasm continues very strong by the trial participants as well. There is data cleaning, making sure that everything matches up, dotting all the i's, crossing all the t's, because we only lock a database once in this instance for the core study, double-blind period. So the team is being very diligent in that regard. And as we said, we expect, you know, top-line data late August, early September. With regard to the Early Access Program, yes, we're so excited to have an Early Access Program for people living with PBH. The initial phase, if you will, of the EAP as we abbreviate it, is for those individuals in LUCIDITY who have completed the study, double-blind period, of course, and then the open-label extension. And then they had the opportunity to roll over into the EAP. And also, at this period of time, it's open for those individuals who have been in prior avexitide trials. Many of those individuals are at other centers, so they have to be activated as well in order to participate in the EAP. It's a separate study, separate protocol. So we're early days yet. The enthusiasm is great about being able to participate in the EAP, and we'll continue to give you updates along the way. Operator: Your next question will come from Joseph Thome, TD Cowen. Joseph Thome: I'm not sure if you can answer this, but I guess in terms of the patient population that you did enroll for the Phase III, I guess, does it overall look similar to the prior Phase II experience or any sort of comments you can make on that? And then second, can you just remind us what sort of mechanisms were in place during the study to monitor compliance with the injections, just to make sure that patients were getting all the recommended doses over the study period? Camille Bedrosian: Sure, sure. Thank you. So just to reiterate, we designed LUCIDITY with the Phase II studies in mind, the highly successful statistically significant outcomes for those Phase II studies. And we're enrolling participants who are very consistent with the populations that were enrolled in the Phase II and Phase IIb. Furthermore, while I can't give you specifics. Furthermore, our Amylyx team conducting the study were those who decided at the end of the day whether an individual was eligible or not. We went through a very rigorous assessment of eligibility before patients enrolled. So we're very confident and very pleased with how the study has been executed from day one, actually. Your second question is about study drug compliance. Yes, that is monitored throughout as well. And, you know, we're also pleased with those metrics and parameters also. Justin Klee: And I'll just add in terms of the trial conduct and sort of compliance, I'd say first it starts with the right clinical trial sites. So we were very rigorous in finding clinical trial sites who not only could recruit the right participants, but also were good clinical trial sites with good experience. Second, we paid particularly close attention during the run-in period to see that not only were participants meeting inclusion and exclusion criteria, in particular the 3 events in 3 weeks, including one Level 3 event, but also that they were good study participants, that they were consistent, and we thought would be good trial participants for the study. And then all throughout the study, our team had very close oversight and monitoring in partnership with the clinical trial sites to make sure that people stayed compliant with all study protocol needs as well as drug, as you were mentioning. Operator: Your next question will come from Michael DiFiore with Evercore ISI. Michael DiFiore: What evidence package would be required for an initial broad label encompassing all PBH? Like does LUCIDITY have enough sleeve patients to make for a compelling subgroup analysis here? And if the FDA restricts the label, potentially, what study design, enrollment size, and timeline would be needed to add sleeve patients? That's my first question. And the second's on a commercial question. Hoping you could bridge the 160,000 patient prevalence estimate into the commercial funnel better. What percent of these 160K patients represent a realistic 5-year treated population? Justin Klee: Hi, Mike. Thank you for the question. So, the first in terms of label, I'll start with that, you know, given that we have yet to see the Phase III trial results, we have yet to submit the NDA, it's, you know, it's early to know too much about what the label will be. I'll give you the context as we see it today. So first and foremost, we believe PBH is PBH, regardless of the surgery that leads to PBH, we believe the pathophysiology is the same and that the primary driver of hypoglycemic events is this exaggerated GLP-1 response. Now, looking through the clinical studies of avexitide, the majority of people with PBH studied with avexitide have had Roux-en-Y gastric bypass surgery leading to PBH, and that includes the Phase III LUCIDITY trial. One of the inclusion criteria was Roux-en-Y gastric bypass surgery that led to PBH only. Now, in the Phase IIb, different surgery that led to PBH were allowed in for inclusion criteria, and avexitide appeared to work just as well regardless of the surgery that led to PBH. So as we submit our NDA and have the discussions with FDA, we think there's sort of two paths that could emerge. What we will strongly make the case for is that we believe avexitide should be for people with PBH, regardless of the surgery led to it due to the same pathophysiology and due to the evidence that we've generated to date. FDA could of course take the stance that the Phase III trial was studied only in Roux-en-Y gastric bypass PBH patients. And so could say, you know, that would be the indication in the label. If that's the case, then I think you're right. The question would be what additional evidence would we need in order to show that avexitide should be applicable regardless of the surgery that led to PBH. And so we're working through those, various scenarios right now. I'd say the way we view it, it should be pretty efficient. You're just trying to show that, you know, that these are not different and that avexitide works regardless. We're able to see the effects of avexitide in a single dose. So we think that should not be an overly burdensome study. And maybe last piece I'll say on the sort of commercial side of things, if we go back to the Stanford prevalence work, their estimate is that of the current 160,000 people with PBH in the United States, about 120,000 had Roux-en-Y gastric bypass leading to their PBH. So I'd say at launch, we're already talking, if we were in that narrower label scenario, we're already talking about a very substantial population. And so our goal would be then to run an efficient study to show that avexitide should work regardless of the surgery that led to PBH. And for your second commercial question, I'll pass to Dan. Dan Monahan: Thanks, Justin. Mike, thanks for the question on the commercial front. And just to build on what Justin had shared on the 160,000 patient population in the prevalent market. So we are aware through market research and through our claims-based analyses of centers and academic centers or different settings of care that have 50 to 60 patients. Some settings even have informed us that they have potentially 100 patients in their care. So now that we know where those patients are, we'll certainly focus there at our launch, and then as our educational efforts take shape and we have more and more education with our commercial colleagues and alignment with our medical colleagues, we look to see that launch trajectory continue to grow. But again, the prevalent population is 160,000 and we remain confident in that number. Operator: Your next question will come from Geoff Meacham with Citi. Geoffrey Meacham: Just had a couple of quick ones. The first is, when you look at pricing reimbursement sort of guidelines, do you think that, I get it that it's a completely unmet medical need, PBH is, but do you think there'll be differentiation between Level 2 and 3 events that you'll see in the study? So that's the first question. The second one is just beyond the Phase III. Are you totally done with other elements of the filing on preclinical, CMC, et cetera, et cetera? Joshua Cohen: Sure. Yes, I'm happy to maybe take part of that and maybe pass to my colleagues as well. So for Level 2 and Level 3, I think it, kind of, bears noting that usually these travel pretty closely together. The reason the ADA selected less than 54 mgs per deciliter as a level for a Level 2 event is because once you're less than 54 mgs per deciliter, that's when your brain is really starved of glucose. So people often begin experiencing, below 54 mgs, serious glycemic events, whether that's loss of consciousness, seizures, severe confusion, et cetera. So it's not often the case that you have a patient that has Level 2s but not Level 3s or vice versa, these things really do tend to travel together. I guess in terms of the NDA preparation as well, we have been working throughout the year to be prepared as possible, and we do believe that the 16-week double-blind outcome from the LUCIDITY Phase III trial, which we're very excited for, is, kind of, the last piece of the NDA that we need to, kind of, put together. Regarding pricing guidelines, I guess I would pass over to Dan. Dan Monahan: Sure. Thanks, Josh. And Geoff, thank you for the question. I would just add, PBH, as we all know, is a very dangerous condition without any approved medications for these patients living with post-bariatric hypoglycemia. So, when we go down the pricing journey, we will certainly take a look at and consider various analogs across both rare as well as rare endocrinology. And again, following top-line data and the LUCIDITY results, we'll then initiate our pricing research. Operator: Your next question will come from Marc Goodman with Leerink Partners. Marc Goodman: Yes, as you have done this review of where the patients are, can you help us understand just the concentration of patients? You just started to mention there were some sites over 100, some 50 to 60. I mean, if you think about the 160,000 patients, are 50% of them at concentrated sites or is it only 20%? Just give us a sense of that. And then one question that we just keep getting, I just want to make sure we hear it from you guys now, and that is, what should we expect the placebo response to be in this study? Dan Monahan: Sure, I'll start with the first question just on where patients are, and we continue to do more and more research using really the claims-based analyses from multiple databases that continue to point us in the direction as to where these patients are. So yes, I've mentioned earlier that there's some settings of care where there's 50 to 60. Some have 70, some have 100, and the more research we do, the more confident we get in those conversations. This is something our medical affairs colleagues have also begun to validate as our MSL team has engaged with various customers to understand and validate some of our claims-based analysis. And again, as we get closer to a potential launch, we will share what our go-to-market plans are in terms of how we'll engage these various centers. Camille Bedrosian: Great, and thanks for the question regarding placebo. I'll start by reiterating that we've had 5 highly successful trials of avexitide in PBH to date, with the Phase II trials showing statistically significant and clinically meaningful reductions in both Level 2 and Level 3. And at ENDO 2025, we shared an ad hoc analysis of the composite as well, which was also highly statistically significant. LUCIDITY is designed to replicate those Phase II studies and we have powered the study very conservatively even in the face of those very highly significant studies. So even with, we're powering at least at 90% with a very modest effect size and with a placebo rate as well. We don't know specifically what the placebo effect is going to be. This is the first Phase III pivotal study of people living with PBH. You know, so that's an important consideration. And finally, as we've been discussing this morning, PBH is a devastating condition. And people already are doing whatever they can to control or mitigate their events. Clearly those who are enrolling in our study continue to have events, but they -- and they continue to do whatever they can to minimize it, given that there is, you know, much fear of hypoglycemia that these patients experience. Justin Klee: And I'll say...Sorry, go ahead, Marc. Marc Goodman: No, I was just going to say, should we be thinking, you know, placebo has a 20% response and the drug does 55%, you know, and we have that 35% delta, like, is that a realistic, you know, and a good scenario? Justin Klee: Well, I think there are 2 different ways, I think, to look at it. I think the first is, what does the prior data tell us? And so the best placebo-based data we have comes from the first Phase II PREVENT study. If you look at the crossover design, so if you look at the run-in to placebo to active on the means, there's a difference of about 30%. If you look at the median, though, there's no difference. So I would say then looking at the Phase III study, our goal was to be conservative in our assumptions. We believe avexitide should work for PBH. That's what the prior studies tell us. And so conservatively powering the study, we thought was the right thing to do. So, we powered the study to be 90% powered to detect a 35% treatment effect with even an up to 50% placebo effect. We have not seen any evidence of such a placebo effect in prior trials, but again, we thought that the right thing to do is to power conservatively. And going back to the Phase II trial, if you look at those very statistically significant results, including for example, 55% reduction in Level 2, Level 3 composite hypoglycemic events, that's against the placebo. So again, if we're powering to 35% difference versus placebo, you can see why we're saying we're using conservative assumptions. Operator: Your next question will come from Rami Katkhuda with LifeSci Capital. Rami Katkhuda: I guess for LUCIDITY, what secondary endpoints will be included in the top-line release and which do KOLs view as the most important? And maybe more broadly, can you provide any additional color on the second research collaboration with Gubra and the type of endocrine indication or target that you're ultimately going after? Camille Bedrosian: Sure. So, we're very excited and eager to see the top-line data, as I expect all of you are as well. And we plan to share the data that is consistent with other rare diseases that present Phase III top-line data. So stay tuned. We're very eager as well. Joshua Cohen: Absolutely. And maybe I just add on the second collaboration with Gubra as well. So yes, we really quite enjoyed working with Gubra. I think they have a particular peptide expertise that we certainly saw with AMX0318, where we tried to develop as optimized a GLP-1 receptor antagonist as we possibly could. We are looking at another rare endocrine disease here of significant unmet need. I think it's, you know, it, kind of, fits with, kind of, our focus on focusing in rare diseases of really serious unmet need, where there are either no or really inadequate treatments as well. But we'll share more details there as maybe it gets further along as well. And I think just to Camille's earlier point as well, we are quite excited for the top line. I think you can expect us to share, kind of, typical data that you'd expect for a top-line data release. And I'd say in talking with KOLs as well, our primary endpoint really is quite a meaningful endpoint here. Level 2 and Level 3 hypoglycemia are viewed as very significant clinically meaningful events. Physicians describe that they have nothing for these patients and any impact on what they basically describe as medical emergencies, these Level 2 and Level 3 events would be quite significant. So I really think that's where people are mostly focused. But we are evaluating in our secondaries, things like the individual Level 2 and Level 3 rates, as well as, you know, kind of, other patient-reported outcomes and otherwise, you know, in the study as well. Operator: Our next question will come from James Condulis with Stifel. James Condulis: On the commercial front, as you've done more work here, curious if you have any more thoughts on, sort of, what the right analogs are here, specifically as it relates to what a launch trajectory may look like and, essentially do you worry at all about a bolus in the sense of getting a lot of traction at those centers that have 50, 60, or 100 patients, but it's taking longer to kind of get to the next layer or next rung of patients? Or, you know, do you think it'll be more steady because there's so many patients here? Just like curious how you're starting to think about that as you've done more work. Dan Monahan: Sure. Thanks for the question, James. And on the launch trajectory, the first thing I'd say and just reiterate is, again, that there's -- for these patients with post-bariatric hypoglycemia, there are no approved therapies. And we continue to stand behind the 160,000 prevalent population. We're very confident in the market size there. This is a market we continue to see building over time. A point that we haven't emphasized yet is just the research that we've done with our endocrinologists. They have a very high intent to treat these PBH patients. So, on the trajectory, again, we will be focused on these centers at launch because that's where we know there are existing patients from the KOLs and endocrinologists who have already communicated to us that they've raised their hands and identified patients. So we'll focus there. But then again, as that educational effort takes shape, as we continue to engage with the marketplace, we do see this building over time. So we'll start with those key centers, educate the centers, and then expand broader into the endocrinology community. And we're certainly looking forward to this educational opportunity with the endos. Operator: Your next question will come from Graig Suvannavejh with Mizuho. Graig Suvannavejh: Just trying to anticipate that in the case that you do get positive data and then you do end up getting the drug approved, could you just remind us how you're thinking about the competitive landscape? There aren't all that many companies that are focused on PBH, but I think it would be good as we think about our uptake curves over whether it's a 5-, 10-year period, how we should be thinking about the competitive landscape? Joshua Cohen: Yes, great question Graig. I'd say, maybe first our view of the kind of pathophysiology of the disease is that this is really driven by an exaggerated GLP-1 response and that's why we believe that avexitide is sort of on target and, a really appropriate way to, try to, ameliorate or significantly impact the disease. So I think, based on the, I mean, obviously we got to get the top-line data and we're quite excited for that. But, we don't really see, much else that has shown, close to the profile of avexitide to date. So we really do believe this will sort of be the first and only, if pending an approval would be the first and only therapy, and we don't really see other things that, have shown the same profile. Justin Klee: And I would just add and underscore the point that, because we really believe in this mechanism of, exaggerated GLP-1 driving hypoglycemic events in PBH, that's why we started development on our long-acting AMX0318 early as well. That's still in IND-enabling studies. Our focus right now is on avexitide, but we really do intend to continue to innovate. And we think AMX0318 may be a very nice way to do so. Graig Suvannavejh: Can I just quickly follow up just on 318? I know it's still early days, but what are the different types of formulations that are being considered for 318? Joshua Cohen: Yes, great question. So maybe first just to say on 318, kind of came out of the collaboration with Gubra where our goal was to -- and what we did was screened a large number of peptides to try to find as optimal a GLP-1 antagonist as we possibly could. And this is really sort of Gubra's bread and butter. It's their kind of main focus is on peptide drug development. And we do believe based on our preclinical data that we were able to arrive at a very strong GLP-1 receptor antagonist. I'd say from a formulation perspective, I think all the options for injectables are certainly available to us, whether that's a vial and syringe or more advanced formulation such as autoinjectors or pens or otherwise. Luckily, I think both with avexitide and with how we've been designing AMX0318, at this time, we don't really see technical hurdles to being able to do that. Operator: Your next question will come from Jason Gerberry with Bank of America. Dina Ramadane: Hi, good morning. This is Dina on for Jason. In your filings, I believe you disclosed a total of $35 million in CMO payment commitments throughout 2028 for avexitide and in the event of a positive Phase III readout is your current manufacturing scale sufficient for a commercial launch? Or would a positive readout trigger additional CMO capacity obligations? And then just a quick question on AMX0035 for Wolfram. Any guidance on when we can maybe expect the update on regulatory interactions regarding the Phase III trial design and just maybe openness to that accelerated approval pathway? Joshua Cohen: Yes, I'd say great question. So I'd say we're certainly kind of proceeding with our manufacturing, in line with, prepping for commercial launch as well. And so we do from time to time make commitments to secure supply and capacity. And I think we expect to continue doing so, as we get towards our launch. And then I guess on your question on Wolfram as well. Yes, so I mean, we are quite excited about our week 96 data, which continued to show stabilization or improvement across glycemic measures as well as visual measures. This would be the first Phase III trial conducted in Wolfram syndrome, and it is a multifactorial disease. People have not only diabetic symptoms, but also visual symptoms, sensorimotor symptoms, kind of, vestibular symptoms of, kind of, balance and otherwise. And patients ultimately usually pass away from respiratory or swallowing difficulties in their early 30s. So there is thought about what is kind of the best design that kind of most efficiently can show, efficacy in the Wolfram population. So that's what we're continuing to work on, but we remain quite excited, including from our week 96 results. Operator: Your next question will come from Ananda Ghosh with H.C. Wainwright. Ananda Ghosh: A couple of questions from me. Can you tell me, like from our KOL discussions, use of avexitide has been always highlighted in certain surgeries, especially the gastric cancer or upper GI surgeries. So I just wanted to get your thoughts on what you are hearing from the KOLs on that. Now, the second question is, what is the development plan for the long-acting avexitide vis-a-vis the avexitide when you are thinking from a commercial point of view? The third is, does a dedicated ICD-10 code change the payer conversation and given the diagnosis is a barrier to penetration of avexitide, what are the efforts from the company on that end? Justin Klee: Excellent. So, I'm happy to start with the first. So, yes, I appreciate your mentioning the potential use of avexitide in other surgery-induced hypoglycemias. That's certainly something we hear a lot about from key opinion leaders. There are many gastric surgeries, as you mentioned, for example, gastrectomy for gastric cancer, that can lead to this same hypoglycemic condition. And in fact, in the Phase IIb trial, there were people who had a gastrectomy or esophagectomy due to cancer-induced hypoglycemia who responded very well to avexitide as well. So we do believe that the mechanism there is very similar, that it's this exaggerated GLP-1 response that is primarily driving the hypoglycemic events so that is certainly something we're interested in studying in the future. And I'll add that while this is certainly an unmet need in the United States, it's a very substantial unmet need in most countries in Asia due to their very high rates of gastric cancer and esophageal cancer, and one of the risks is that people develop this hypoglycemic condition. As far as the 318 development plan, I'd say, please stay tuned. We're in IND-enabling studies now. I'd say, as Josh was mentioning, we really screened for molecules that would meet all of our criteria, including good drug-like properties and good sort of manufacturing qualities. So we, as we go through the IND-enabling studies, we'll outline our development plan. But we really do believe that the primary driver of events in PBH, in surgery-induced hypoglycemia, is this exaggerated GLP-1 response. And so that's why we're investing in 318 as well as investing in avexitide because we really believe that there's a substantial unmet need and opportunity here. For the ICD-10 code, maybe I'll pass to Dan to share more on that. Dan Monahan: Sure. Thanks, Justin. And as Camille mentioned earlier, in June, CMS and the CDC, they did publish their 2027 ICD-10 code files. So beginning October 1st, this is when we will have a specific code for post-bariatric hypoglycemia. First thing I would say is this new code really recognizes PBH within the broader medical community. ICD-10 codes, they are certainly helpful for tracking and diagnosing patients. And oftentimes the coding, the ICD-10 codes are used for epidemiology. From a payer perspective, an ICD-10 code is not necessary for reimbursement. And this is again, just back to the claims analysis. Patients can still be identified today through the claims analysis. You don't need an ICD-10 code to identify them. But again, this is really a recognition of PBH in the broader community, and we're excited that a code is going to be effective October 1st. Justin Klee: And on your last question, in terms of diagnosis, so I think the first thing I'll say is I think PBH awareness and diagnosis is high. We've certainly seen that in all of our market research. But as Dan was saying as well, this is also a condition where there had not been FDA-approved treatments. There are many education gaps. And so I think starting on the education front is why we were so excited to launch our disease state education campaign. We've been very pleased with the feedback on that so far. So those efforts will continue. Operator: Your final question will come from Christopher Chen with Baird. Christopher Chen: Two for me. Can you characterize just the nature of recent interactions, if any, with FDA surrounding the NDA? And do you plan on having any additional interactions before submission? And then just one for Dan. I know it's still somewhat early days, but assuming positive data and approval, how soon do you think you can launch following approval? And what would you say are the primary factors gating a launch? Camille Bedrosian: Great. So, thank you, Chris. With regard to our FDA interactions, as we don't really discuss the details of the interactions. We have had, as part of avexitide's breakthrough therapy designation, we have had consistent interactions with the agency throughout this period of time. Not starting with but one of course was, reviewing the LUCIDITY protocol and throughout this time. So we're very excited about the potential to submit an NDA. We have a tremendously experienced team working on the NDA elements and they're making great progress. Obviously, when we have the core 16-week data set, those will be included in the NDA and we'll go from there. And we continue to plan for a launch in 2027. Dan Monahan: Chris, I'll just jump in on the launch date and the launch timing. We're certainly excited about the Phase III LUCIDITY results. At this point in time, we've guided towards a launch in 2027. From a launch preparation perspective, as we've mentioned, we've made key hires across the organization within medical affairs and across the commercial side in market access, marketing, as commercial operations. So our launch plans are on track and certainly underway as we prepare for a successful launch. Operator: There are no further questions at this time. I'll turn the call back over to Mr. Klee for any closing remarks. Justin Klee: Thank you, operator, and thank you all for your time. We're looking forward to connecting when we report the top-line data after final database cleaning and lock. We're excited about what these results could mean for people with PBH. We hope you have a great rest of your day. Operator: Thank you for your participation. Before you buy stock in Amylyx Pharmaceuticals, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the 10 best stocks for investors to buy now… and Amylyx Pharmaceuticals wasn’t one of them. The 10 stocks that made the cut could produce monster returns in the coming years. 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This article first appeared on GuruFocus. Release Date: August 06, 2026 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Amylyx Pharmaceuticals Inc (NASDAQ:AMLX) is on track for the pivotal Phase 3 LUCIDITY trial top-line data readout in late August or early September 2026, with the last participant having completed their final visit. The company is actively preparing all sections of the NDA for a potential submission, with the 16-week double-blind data being the last major piece needed. Commercial launch readiness for avexitide is progressing well, including the deployment of a field medical affairs team and the activation of a disease state education campaign ('Uncover the Mystery of Post Bariatric Hypoglycemia'). The company estimates a substantial US market opportunity of approximately 160,000 people living with post-bariatric hypoglycemia (PBH), supported by claims analysis and field insights. A new specific ICD-10 code for PBH will be effective October 1, 2026, which is expected to increase disease recognition and aid in patient identification and tracking. The broader pipeline is advancing, including positive week 96 data for AMX-0035 in Wolfram syndrome and progress in the Phase 1 trial for AMX-114 in ALS. The company maintains a strong cash position of $250.8 million, providing a runway into 2028 to fund operations through key milestones, including a potential 2027 launch. High intent to prescribe among endocrinologists for an approved PBH therapy has been demonstrated in market research, providing confidence in the commercial opportunity. The company remains blinded to the LUCIDITY data and the database has not yet been locked, leaving uncertainty around the final results. There is potential for the FDA to restrict the initial label to only Roux-en-Y gastric bypass patients, which could require an additional study to expand the indication to all PBH patients. The company faces uncertainty regarding the placebo response rate in the Phase 3 trial, which is the first pivotal study in PBH and could impact the observed treatment effect. SG&A expenses increased significantly (up 40% year-over-year) due to higher legal expenses, including a one-time legal charge, and investments in commercial initiatives. The company is entering a quiet period after the call, which will limit communication and updates until the top-line data is released. The early access program (EAP) is still in its early days, and the full demand and patient recruitment into the program have not yet been quantified. The competitive landscape, while currently sparse, could evolve, and the company's long-term market position depends on the success of its first-in-class therapy and future pipeline candidates like AMX-318. Warning! GuruFocus has detected 3 Warning Sign with AMLX. Is AMLX fairly valued? Test your thesis with our free DCF calculator. Q: Can you update us on when the last patient visit was and what key factors need to be completed to lock the database? Also, can you provide color on how the early access program (EAP) is progressing? A: Josh Cohen (Co-CEO): The last patient visit occurred in mid-to-late July, following the completion of enrollment in mid-March. The team is diligently working on data cleaning and ensuring all details are aligned before locking the database for the core 16-week double-blind period. Top-line data is expected in late August or early September. Regarding the EAP, the initial phase is open to individuals who completed the Lucidity trial's double-blind period and rolled over into the open-label extension, as well as those from prior vexitide trials. Enthusiasm for participation is high, though it is still early days. Q: Regarding the potential label, what evidence package would be required for an initial broad label encompassing all PBH? Does Lucidity have enough sleeve patients for a compelling subgroup analysis? Also, can you bridge the 160,000 patient prevalence estimate into a realistic 5-year treated population? A: Justin Klee (Co-CEO): We believe PBH is PBH regardless of the surgery that leads to it, as the pathophysiology is the samean exaggerated GLP-1 response. The phase 3 Lucidity trial enrolled only Roux-en-Y gastric bypass patients, but phase 2b data showed vexitide worked equally well regardless of the surgery. We will strongly advocate for a broad label, but if the FDA restricts it, we believe an efficient study could demonstrate efficacy across surgeries. Dan Monahan (Chief Commercial Officer): Of the 160,000 prevalent PBH patients, approximately 120,000 had Roux-en-Y gastric bypass. We are confident in this estimate and have identified centers with 50-100 patients, which will be our initial launch focus. Q: What should we expect the placebo response to be in the Lucidity study, and is a scenario of a 20% placebo response versus 55% drug response realistic? A: Camille Bedrosian (Chief Medical Officer): We have had 5 highly successful trials of vexitide in PBH with statistically significant reductions in level 2 and level 3 events. Lucidity was conservatively powered with 90% power to detect a 35% treatment effect even with an up to 50% placebo effect. In the phase 2 trial, we saw a 55% reduction in the composite endpoint versus placebo. We don't know the specific placebo rate as this is the first phase 3 pivotal study, but we have not seen evidence of a high placebo effect in prior trials. Q: What secondary endpoints will be included in the top-line release, and can you provide additional color on the second research collaboration with Gubra? A: Camille Bedrosian (Chief Medical Officer): We plan to share data consistent with other rare disease phase 3 top-line releases. The primary endpointlevel 2 and level 3 hypoglycemic eventsis viewed as highly clinically meaningful by KOLs, who describe these as medical emergencies. Secondary endpoints include individual level 2 and level 3 rates and patient-reported outcomes. Josh Cohen (Co-CEO): The second collaboration with Gubra focuses on another rare endocrine disease of high unmet need, leveraging their peptide expertise. We will share more details as the program advances. Q: As you've done more commercial work, what are the right analogs for a launch trajectory? Do you worry about a bolus of traction at centers with 50-100 patients but slower uptake in the next layer? A: Dan Monahan (Chief Commercial Officer): We are confident in the 160,000 prevalent population and the high intent among endocrinologists to treat PBH. At launch, we will focus on key centers where we know existing patients are located, then expand through educational efforts into the broader endocrinology community. We see this as a market that builds over time, starting with those centers that have already identified patients. Q: How are you thinking about the competitive landscape for PBH over a 5-10 year period? A: Josh Cohen (Co-CEO): We believe the disease is driven by an exaggerated GLP-1 response, and vexitide is on target to address this. We don't see other therapies with a similar profile, and we believe vexitide would be the first and only approved therapy. We are also developing AMX 318, a long-acting GLP-1 receptor antagonist, to continue innovating in this space. Q: In your filings, you disclosed $35 million in CMO payment commitments through 2028 for vexitide. Is the current manufacturing scale sufficient for a commercial launch, and what is the guidance for AMX 0035 in Wolfram syndrome? A: Josh Cohen (Co-CEO): We are proceeding with manufacturing preparations in line with commercial launch readiness and will continue to make commitments to secure supply and capacity. For AMX 0035 in Wolfram syndrome, we are excited about the week 96 data showing stabilization or improvement in glycemic and visual measures. We are working on the most efficient phase 3 trial design for this multifactorial disease, which affects patients in their early 30s. Q: From KOL discussions, vexitide has been highlighted for use in other surgeries like gastrectomy or upper GI surgeries. What are your thoughts on this, and what is the development plan for AMX 318? Also, does the dedicated ICD-10 code change the payer conversation? A: Justin Klee (Co-CEO): We hear from KOLs about the potential use of vexitide in other surgery-induced hypoglycemia, such as gastrectomy for gastric cancer. In phase 2b, patients with cancer-induced hypoglycemia responded well to vexitide. This is a substantial unmet need, particularly in Asia where gastric cancer rates are high. For AMX 318, we are in IND-enabling studies and will outline the development plan as we progress. Dan Monahan (Chief Commercial Officer): The new ICD-10 code for PBH, effective October 1, 2026, recognizes the condition within the broader medical community. However, an ICD-10 code is not necessary for reimbursement, as patients can already be identified through claims analysis. Q: Can you characterize recent interactions with the FDA surrounding the NDA, and how do you plan to launch following approval? A: Josh Cohen (Co-CEO): As part of the breakthrough therapy designation, we have had consistent For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Last participant completed final study visit in the 16-week double-blind period of the pivotal Phase 3 LUCIDITY trial of avexitide in post-bariatric hypoglycemia; topline data on track and anticipated in late August or early September 2026 Cash runway expected to fund operations into 2028 Management to host conference call and webcast today at 8:00 a.m. Eastern Time CAMBRIDGE, Mass., August 06, 2026--(BUSINESS WIRE)--Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) ("Amylyx" or the "Company") today reported financial and business results for the second quarter ended June 30, 2026. "With the last participant’s final study visit recently completed, we are eagerly anticipating the expected topline data readout of the pivotal Phase 3 LUCIDITY trial in post-bariatric hypoglycemia in late August or early September," said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. "In parallel, we are continuing to advance our NDA readiness and our pre-commercial work to support a potential submission and commercial launch of avexitide in 2027, if approved. At ENDO in June 2026, we activated our disease state education campaign, ‘Uncover the Mystery of Post-Bariatric Hypoglycemia,’ designed to increase awareness and understanding action around PBH. We look forward to sharing updates as we work toward potentially delivering the first FDA-approved therapy for the PBH community." Second Quarter and Recent Updates: The last participant has recently completed the final study visit in the 16-week double-blind period of the pivotal Phase 3 LUCIDITY clinical trial of avexitide, an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist that has received U.S. Food and Drug Administration (FDA) Breakthrough Therapy Designation in post-bariatric hypoglycemia (PBH). LUCIDITY enrolled 78 participants and is a multicenter, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of avexitide in adults with PBH following Roux-en-Y gastric bypass (RYGB) surgery. Participants who complete the 16-week double-blind period are eligible to enter a 32-week open-label extension period. Amylyx presented two posters at the Endocrine Society’s Annual Meeting (ENDO 2026) in June 2026. The first poster characterized the clinical, economic, and humanistic burden of PBH in the U.S. on both a patient and systemic level, evaluating how Level 2 and 3 hypoglycemic events in PBH impact healthcare utilization, productivity, and overall cost. The second poster described participant-centric trial design considerations for the LUCIDITY trial of avexitide in PBH. Amylyx announced the initiation of a U.S. Expanded Access Program for the use of avexitide to treat adults with PBH following RYGB surgery in May 2026. Initial eligible patients include individuals who have completed the LUCIDITY trial or participated in a prior trial of avexitide in PBH following RYGB surgery. Amylyx entered into a second research collaboration with Gubra A/S in July 2026 to identify potential development candidates for a rare endocrine disease of high unmet need. Amylyx presented data from its first-in-human, dose-ranging Phase 1 LUMINA clinical trial of AMX0114, an investigational antisense oligonucleotide (ASO) targeting calpain-2 for the potential treatment of amyotrophic lateral sclerosis (ALS), at the 2026 European Network to Cure ALS (ENCALS) Annual Meeting in June 2026. AMX0114 showed no drug-related serious adverse events (AEs) and no serious neurological AEs in Cohort 1, supporting continued evaluation at higher dose levels. Cohort 1 evaluated the lowest of four planned dose levels. Cohorts 1 (12.5 mg) and 2 (25 mg) are fully enrolled, and Cohort 3 (50 mg) is currently enrolling. Amylyx announced the peer-reviewed publication of Week 24 and Week 48 results from the Phase 2 open-label HELIOS clinical trial of AMX0035 in adults living with Wolfram syndrome in The Journal of Clinical Investigation in May 2026. The results reinforced consistency of observed stabilization or improvement across multiple outcomes related to disease progression, including pancreatic beta cell function, glycemic control, vision, and overall symptom burden. AMX0035 was generally well-tolerated, consistent with previously presented safety data. Amylyx also presented longer-term Week 96 data from HELIOS in June 2026. At Week 96, measures of pancreatic function and other measures of glycemic control were stable or improved relative to baseline in most participants. Visual acuity and patient- and clinician-reported outcomes showed patterns consistent with disease stabilization with interpretation limited by the open-label, single-arm design and small sample size (n=9 participants with data at Week 96). The safety profile of AMX0035 in HELIOS at Week 96, Week 48, and Week 24 were generally consistent with prior safety data from the studies of AMX0035. Nearly all AEs were mild or moderate, and there were no serious AEs related to AMX0035 treatment. The Company continues to work with the FDA on a Phase 3 trial in Wolfram syndrome. Upcoming Expected Milestones: Topline data readout for the Phase 3 LUCIDITY clinical trial of avexitide in PBH is on track and anticipated in late August or early September 2026. LUCIDITY is evaluating the FDA-agreed-upon primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. The design of the LUCIDITY trial was informed by data from five prior clinical trials of avexitide in PBH, which showed consistent effects, most notably statistically significant reductions in Level 2 and Level 3 hypoglycemic events. Across previous clinical trials, avexitide was generally well-tolerated with a favorable safety profile. If approved, commercial launch of avexitide is anticipated in 2027. Investigational New Drug (IND)-enabling studies for AMX0318, a novel GLP-1 receptor antagonist for long-acting administration to treat PBH and other rare diseases, are underway with an IND filing targeted for 2027. AMX0318 was selected as a development candidate after demonstrating robust preclinical and chemical properties, including a favorable pharmacokinetic profile that may support long-acting administration, a robust chemical stability profile, strong in vitro potency, evidence of in vivo activity and tolerability, and high solubility. AMX0318 was identified through a research collaboration with Gubra A/S, a company specializing in peptide-based drug discovery and preclinical contract research services. Financial Results for the Second Quarter Ended June 30, 2026 R&D Expenses: Research and development expenses for the second quarter of 2026 were $23.8 million, compared to $27.2 million for the same period in 2025. The decrease was primarily due to a decrease in spending related to AMX0035 for the treatment of progressive supranuclear palsy. The decrease was offset primarily by an increase in spending related to the clinical development of avexitide in PBH and other costs related to avexitide. Research and development expenses include $2.5 million of stock-based compensation expense for the quarter ended June 30, 2026, compared to $2.0 million of stock-based compensation expense for the quarter ended June 30, 2025. SG&A Expenses: Selling, general, and administrative expenses for the second quarter of 2026 were $21.9 million, compared to $15.6 million for the same period in 2025. This increase was primarily due to higher legal expenses and increased investment in commercial strategic initiatives. Selling, general, and administrative expenses include $5.8 million of stock-based compensation expense for the quarter ended June 30, 2026, compared to $5.4 million of stock-based compensation expense for the quarter ended June 30, 2025. Net Loss: Net loss for the three months ended June 30, 2026 was $43.4 million, or $0.39 per share, compared to net loss of $41.4 million, or $0.46 per share, for the same period in 2025. Cash Position: Cash, cash equivalents, and short-term investments were $250.8 million at June 30, 2026, compared to $279.8 million at March 31, 2026. Based on its current operating plans, Amylyx expects a cash runway into 2028. Investor Conference Call Information Amylyx’s management team will host a conference call today, August 6, 2026, at 8:00 a.m. ET. To access the conference call, please dial +1 (888) 880-3330 (U.S. & Canada) or +1 (646) 357-8766 (international) at least 10 minutes prior to the start time and ask to be joined into the Amylyx Pharmaceuticals call. A live audio webcast of the call will be available under "Events and Presentations" in the Investor section of the Company’s website, https://investors.amylyx.com/events-presentations. The webcast will be archived and available for replay for 90 days following the event. Available Information Amylyx periodically provides other information for investors on the Company’s corporate website, https://amylyx.com, and the Company’s investor relations website, https://investors.amylyx.com. This includes press releases and other information about financial performance, information on corporate governance, and details related to our annual meeting of stockholders. Amylyx intends to use its website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor Amylyx’s website, in addition to following the Company’s press releases, SEC filings, and public conference calls and webcasts. About Avexitide Avexitide is an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist that has been evaluated in five Phase 1 and Phase 2 clinical trials for post-bariatric hypoglycemia (PBH) and has also been studied in congenital hyperinsulinism (HI). The U.S. Food and Drug Administration (FDA) has granted avexitide Breakthrough Therapy Designation for both indications, Rare Pediatric Disease Designation in congenital HI, and Orphan Drug Designation for the treatment of hyperinsulinemic hypoglycemia (which includes PBH and congenital HI). In PBH, an exaggerated GLP-1 response leads to excessive insulin secretion, resulting in recurrent hypoglycemic events. Avexitide is a GLP-1 receptor antagonist designed to competitively bind to the GLP-1 receptor on pancreatic islet beta cells and inhibit the exaggerated GLP-1-driven insulin response characteristic of PBH, reducing inappropriate insulin secretion and stabilizing blood glucose levels. In two Phase 2 PBH clinical trials, avexitide demonstrated highly statistically significant reductions in hypoglycemic events. About Post-Bariatric Hypoglycemia (PBH) PBH is a chronic metabolic condition that is estimated to affect approximately 8% of people in the U.S. who have undergone the two most common types of bariatric surgery, sleeve gastrectomy and Roux-en-Y gastric bypass (approximately 160,000 people in the U.S.). PBH is thought to be driven by an exaggerated glucagon-like peptide-1 (GLP-1) response, primarily in response to food intake, leading to persistent, recurrent, and often debilitating rapid drops in blood glucose, known as hypoglycemia. The American Diabetes Association (ADA) recognizes hypoglycemia as a potential medical emergency because low blood glucose levels can compromise the body’s ability to maintain essential physiologic processes. In addition, hypoglycemia in the context of PBH may manifest as neuroglycopenia – an inadequate supply of glucose to the brain – which can cause confusion, cognitive dysfunction, loss of consciousness, and seizures. PBH can be associated with substantial disability, compromising safety, disrupting independent living, and affecting nutritional status and overall quality of life. Despite the substantial burden, there are currently no FDA-approved therapies for PBH. About the LUCIDITY Trial LUCIDITY (NCT06747468) is a 78-participant, multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the efficacy and safety of avexitide in participants with PBH following RYGB surgery. The Phase 3 trial is being conducted at 21 sites in the U.S. Participants were randomized 3:2 to receive either 90 mg of avexitide subcutaneously once daily or placebo. The trial includes an up to six-week screening period, including a three-week run-in period, a 16-week double-blind treatment period, and an open-label extension (OLE) period with a duration of 32 weeks. The primary efficacy objective of LUCIDITY is to evaluate the FDA-agreed-upon primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. Safety and tolerability will also be evaluated. About AMX0114 AMX0114 is an investigational antisense oligonucleotide (ASO) with U.S. Food and Drug Administration (FDA) Fast Track designation for the potential treatment of ALS. AMX0114 targets calpain-2 (CAPN2), a calcium-activated protease that is one of the fundamental drivers of axonal degeneration and consequent disease progression in ALS. In preclinical studies, treatment with AMX0114 resulted in potent, dose-dependent, and durable reduction in CAPN2 mRNA and calpain-2 protein levels in disease-relevant cell models of axonal degeneration. This translated to improved neuronal survival, including in a model of TDP-43 ALS, and reductions in extracellular neurofilament light (NfL) levels across multiple disease models and paradigms of neuronal injury. AMX0114 was generally well-tolerated in LUMINA trial participants enrolled in Cohort 1 (n=12), with no treatment-related serious adverse events (SAEs). About Amylyx Pharmaceuticals At Amylyx, our mission is to usher in a new era of treating diseases with high unmet needs. Where others see challenges, we see opportunities that we pursue with urgency, rigorous science, and unwavering commitment to the communities we serve. We are currently focused on four investigational therapies across several endocrine conditions and neurodegenerative diseases in which we believe can make the greatest impact. For more information, visit amylyx.com and follow us on LinkedIn and X. For investors, please visit investors.amylyx.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, Amylyx’s expectations regarding: the therapeutic potential of avexitide as a treatment for PBH; the timing for the topline data readout and completion of the Phase 3 LUCIDITY clinical trial of avexitide; the timing for potential commercialization of avexitide, if approved; the expected enrollment and progress of the Expanded Access Program for avexitide; the therapeutic potential for AMX0114 as a treatment for ALS; the expected enrollment and progress of the LUMINA trial; the therapeutic potential of AMX0318 and the expected timeline for a potential IND submission; the therapeutic potential of AMX0035 as a treatment for Wolfram syndrome and plans for a Phase 3 trial; the potential benefits of expedited program and orphan designations held by Amylyx; the potential benefits of the research collaborations with Gubra A/S; and financial performance, cash runway and longer-term strategy. Any forward-looking statements in this press release and related comments in the Company’s earnings conference call are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx’s program development activities; Amylyx’s ability to execute on its regulatory development plans and expectations regarding the timing of results from its planned data announcements and initiation of clinical studies; Amylyx’s ability to fund operations, and the impact that global macroeconomic uncertainty, geopolitical instability, and public health events will have on Amylyx’s operations, as well as the risks and uncertainties set forth in Amylyx’s United States Securities and Exchange Commission (SEC) filings, including Amylyx’s Annual Report on Form 10-K for the year ended December 31, 2025, and subsequent filings with the SEC. All forward-looking statements contained in this press release and related comments in our earnings conference call speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. View source version on businesswire.com: https://www.businesswire.com/news/home/20260806299550/en/ Contacts Media Amylyx Media Team+1 (857) [email protected] Investors Lindsey AllenAmylyx Pharmaceuticals, Inc.+1 (857) [email protected]

Interested in Amylyx Pharmaceuticals, Inc.? Here are five stocks we like better. LUCIDITY trial results are expected in late August or early September. Amylyx has completed final patient visits in the Phase III avexitide study for post-bariatric hypoglycemia, with database cleaning and locking still underway. Amylyx is preparing for a potential U.S. avexitide launch in 2027, contingent on positive data and FDA approval. The company is building commercial capabilities and estimates roughly 160,000 U.S. patients have PBH after common bariatric procedures. The company ended Q2 with $250.8 million in cash and marketable securities, which management expects to fund operations into 2028. Amylyx also reported $45.7 million in quarterly operating expenses and continued advancing programs for Wolfram syndrome, ALS and other rare diseases. Amylyx Stock: Why the Full Pipeline Story Matters Amylyx Pharmaceuticals (NASDAQ:AMLX) said its pivotal Phase III LUCIDITY trial of avexitide in post-bariatric hypoglycemia, or PBH, has completed final patient visits, with top-line data expected in late August or early September. The company remains blinded to the data and said the trial database has not yet been locked. LUCIDITY is a randomized, double-blind, placebo-controlled study evaluating 90 mg of avexitide once daily in adults with PBH following Roux-en-Y gastric bypass surgery. → 3 Drone Stocks That Should Soar After the Summer Slump 3 small-cap biotechs with potential breakthroughs in 2024 Avexitide is an investigational GLP-1 receptor antagonist that has received FDA breakthrough therapy designation for PBH. The primary endpoint in LUCIDITY is the reduction in the composite of Level 2 and Level 3 hypoglycemic events through week 16, an outcome Amylyx said was agreed upon with the FDA. Chief Medical Officer Camille Bedrosian said the company is completing data-cleaning work and ensuring trial records are complete before locking the database. She said Amylyx has been “very diligent” because the database will be locked once for the 16-week double-blind core study. → Meta’s Earnings Drop Shows Wall Street Wants More Than Ad Growth These biotechs targeting multiple neurodegenerative diseases Bedrosian said the Phase III population was designed to be consistent with patients enrolled in prior Phase II studies of avexitide. The company also said it monitored treatment compliance throughout the study, supported by site selection, a run-in period and ongoing oversight of participating clinical sites. Co-CEO Josh Cohen said Amylyx believes the LUCIDITY 16-week double-blind results represent the final major component needed for its new drug application, or NDA. The company is preparing applicable NDA sections ahead of a potential submission, though management did not provide a filing timeline. → Jersey Mike's Serves Fresh Gains After IPO Stumble Management said the FDA could potentially limit an initial indication to PBH following Roux-en-Y gastric bypass because LUCIDITY enrolled only those patients. However, Co-CEO Justin Klee said the company intends to argue that PBH should be treated similarly regardless of the surgery that led to the condition, citing what it described as a shared underlying mechanism involving an exaggerated GLP-1 response. Klee noted that the prior Phase IIb study allowed patients with different surgeries leading to PBH and that avexitide appeared to work across those groups. If the initial label is narrower, Amylyx believes additional evidence to support broader use could potentially be generated efficiently, although it did not provide a specific study design or timeline. Amylyx is preparing for a potential U.S. launch of avexitide in 2027, contingent on positive data and regulatory approval. The company has deployed a field medical affairs team and continued adding personnel in marketing, market access and commercial operations. Chief Commercial Officer Dan Monahan estimated that about 160,000 people in the U.S. are living with PBH following the two most common bariatric procedures, Roux-en-Y gastric bypass and sleeve gastrectomy. He said the estimate is supported by claims analyses, field insights and published literature. Of that total, Klee said approximately 120,000 patients had Roux-en-Y gastric bypass associated with their PBH, based on Stanford prevalence work cited by the company. Monahan said Amylyx has identified care settings with 50 to 60 PBH patients and, in some instances, up to 100 patients. The company expects to focus initial launch efforts on those centers before expanding outreach more broadly within the endocrinology community. The company also launched a disease-state education campaign, “Uncover the Mystery of Post-Bariatric Hypoglycemia,” including the uncoverpbh.com website for healthcare professionals and patients. Amylyx said it received strong engagement at the ENDO 2026 meeting and observed growing familiarity with PBH among endocrinologists. In June, CMS and the CDC published 2027 ICD-10 code files that include a PBH-specific code, which will take effect for patient encounters beginning Oct. 1, 2026. Monahan said the code recognizes PBH within the broader medical community and may assist with tracking and diagnosis, although he said an ICD-10 code is not required for reimbursement or for identifying patients through claims data. Amylyx ended the second quarter with $250.8 million in cash and marketable securities, down from $279.8 million at the end of the first quarter. Chief Financial Officer Jim Frates said the company expects its cash position to fund operations into 2028, including the LUCIDITY readout, a potential FDA approval and a possible commercial launch in 2027. Total operating expenses were $45.7 million, up 7% from the same quarter of 2025. Research and development expense was $23.8 million, compared with $27.2 million a year earlier. Selling, general and administrative expense was $21.9 million, compared with $15.6 million a year earlier. Non-cash stock-based compensation expense totaled $8.3 million, compared with $7.4 million in the prior-year quarter. Frates attributed the decline in research and development expense primarily to lower spending on AMX0035 for progressive supranuclear palsy, partly offset by higher avexitide development costs. The increase in selling, general and administrative expense reflected increased legal expenses, including a one-time legal charge, and investments in commercial initiatives. Beyond avexitide, Amylyx said 96-week data from the Phase II open-label HELIOS trial of AMX0035 in Wolfram syndrome continued to show stabilization or improvement in measures of glycemic control and vision. The company said it is continuing to assess the most efficient design for a potential Phase III trial in the multifactorial rare disease. For AMX0114 in amyotrophic lateral sclerosis, Amylyx said it is enrolling Cohort 3 in the Phase I multiple-ascending-dose LUMINA trial after what it characterized as a favorable safety profile to date. The company also said IND-enabling studies are underway for AMX0318, a long-acting GLP-1 receptor antagonist, with an investigational new drug filing targeted for 2027. Amylyx entered a second research collaboration with peptide-specialist Gubra in July to identify candidates for another rare endocrine disease, but did not disclose the target indication. Amylyx Pharmaceuticals, Inc is a biopharmaceutical company dedicated to developing treatments for rare and debilitating neurological diseases. Founded in 2013 and headquartered in Cambridge, Massachusetts, the company focuses on leveraging novel approaches to target cellular pathways implicated in neurodegeneration. Amylyx's research platform centers on small-molecule therapies designed to protect neurons and support cellular health in patients with conditions that currently have limited or no disease-modifying treatment options. The company's lead product, AMX0035, is marketed under the trade name Relyvrio following U.S. This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. The article "Amylyx Pharmaceuticals Q2 Earnings Call Highlights" was originally published by MarketBeat. 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We recently compiled a list of the 8 Best Small Cap Pharma Stocks to Buy Right Now. Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) is among the best pharmaceutical stocks. TheFly reported on May 7 that AMLX reported first-quarter 2026 results for the period ended March 31, 2026, alongside pipeline and clinical updates. R&D expenses rose to $27.6 million from $22.1 million a year earlier, driven mainly by increased spending on avexitide development for post-bariatric hypoglycemia and milestone payments tied to the AMX0318 program. SG&A expenses increased slightly to $16.2 million from $15.7 million due to higher professional and consulting costs. Net loss widened to $41.3 million, or $0.37 per share, compared with $35.9 million, or $0.42 per share, in the prior-year period. The company ended the quarter with $279.8 million in cash, cash equivalents, and short-term investments, supporting operations into 2028. Moreover, earlier on May 5, Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) announced the initiation of a U.S. Expanded Access Program allowing treatment access to avexitide for up to 250 adults with post-bariatric hypoglycemia. The program provides physicians the ability to request the investigational glucagon-like peptide-1 receptor antagonist for patients with significant unmet medical needs who cannot join ongoing clinical trials and have limited remaining treatment options. Eligibility includes individuals with PBH following Roux-en-Y gastric bypass surgery, including those who have completed the Phase 3 LUCIDITY trial or previously participated in related studies. The program is designed to expand controlled access to avexitide under defined clinical criteria while broader development continues for the therapy in PBH. Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) is a clinical-stage biopharmaceutical company developing therapies for neurodegenerative and endocrine diseases with high unmet medical needs. While we acknowledge the potential of AMLX as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 33 Stocks That Should Double in 3 Years and 15 Stocks That Will Make You Rich in 10 Years Disclosure: None. Follow Insider Monkey on Google News.

Image source: The Motley Fool. Thursday, May 7, 2026 at 8 a.m. ET Co-Chief Executive Officer — Justin Klee Co-Chief Executive Officer — Joshua Cohen Chief Commercial Officer — Daniel Monahan Chief Medical Officer — Camille Bedrosian Chief Financial Officer — James Frates Need a quote from a Motley Fool analyst? Email [email protected] Justin Klee: Good morning, everyone, and thank you for joining us. The first quarter of 2026 was marked by execution across our pipeline. Most notably, we continue to progress the pivotal Phase III LUCIDITY trial of avexitide, our investigational first-in-class GLP-1 receptor antagonist with FDA breakthrough therapy designation in post-bariatric hypoglycemia or PBH. We are executing on the 3 strategic imperatives for avexitide that we outlined earlier this year. First, we are advancing the pivotal Phase III LUCIDITY trial toward top line data. Randomizing and dosing the last participant in late March was a significant milestone. We have a clear line of sight toward the completion of the 16-week trial, and we remain on track for a top line readout next quarter. Second, we are advancing NDA readiness and regulatory preparations. We are already drafting NDA sections to support a potential submission. And third, we continue to strengthen our launch readiness. We are executing against a comprehensive commercial readiness road map to help ensure we are fully prepared for commercialization of avexitide, if approved, in 2027. In addition to avexitide, we continue to make progress across our broader pipeline. For AMX0318, our long-acting GLP-1 receptor antagonist, IND-enabling studies are underway. We are targeting a 2027 IND filing. For AMX0035 in Wolfram syndrome, we anticipate presenting longer-term week 96 data from the Phase II open-label HELIOS clinical trial at an upcoming scientific meeting. And for AMX0114 in ALS, we fully enrolled Cohort 2 of the Phase I LUMINA trial in March. At the ENCALS Annual Meeting this June, we expect to present early biomarker data from Cohort 1, the first and lowest of 4 doses being evaluated in the trial. We expect these data will provide initial information about the levels of the ALS biomarkers being assessed in the LUMINA trial from the first cohort. As we continue to advance our pipeline, we are simultaneously preparing for the potential commercial launch of avexitide. To discuss our launch readiness efforts, Dan Monahan, our Chief Commercial Officer, is with us on the call today. Dan joined Amylyx in January 2024, bringing more than 2 decades of experience. He was instrumental in the commercialization of Otsuka's Rexulti, Novartis' Cosentyx and Sanofi's Lantus and Actonel, among others. With that, Dan, I'll turn the call over to you. Dan Monahan: Thank you, Justin, and good morning, everyone. I'm pleased to be on the call today to discuss how we have been refining our launch strategies as we prepare for the potential commercialization of avexitide next year. The more we engage with the PBH community, the more we understand the profound unmet need that exists. We are operating with a deep sense of urgency. To date, our commercial efforts have been focused on gaining key insights into the PBH market. This includes gathering direct insights from people living with PBH and the health care professionals who are managing their condition. In addition, we are developing a deep understanding of the patient journey and continuing ongoing claims work to help us determine where patients are being treated. To enable our commercial preparations, we've made key hires across marketing, market access and commercial operations. Ahead of the potential approval and commercial launch of avexitide, our immediate focus is on disease state education. This includes raising stakeholder awareness of PBH with an emphasis on the pathophysiology, the importance of accurate and timely diagnosis and the profound unmet need and burden of the condition. We plan to launch this disease state education campaign this summer. Looking at the market opportunity, our independent claims analysis and ongoing field engagements continue to support our estimate of approximately 160,000 people living with PBH in the U.S. who have undergone the 2 most common types of bariatric surgery: sleeve gastrectomy and Roux-en-Y gastric bypass. Our estimates are firmly rooted in the growing body of prospective and retrospective published literature, including large long-term cohort studies evaluating hypoglycemia in people who have undergone bariatric surgery. Importantly, our ongoing market research indicates that endocrinologists have a high intent to treat PBH if there were to be an approved medicine. To reach appropriate patients, we have initiated our marketplace sizing efforts and continue to identify key centers and endocrinologists that manage this condition. Building up to the potential launch, we will refine these efforts as more insights are generated. Our commercial preparations are advancing in lockstep with our clinical progress, and we look forward to sharing additional details as we move closer to the commercialization of avexitide, if approved. With that, I'll turn the call over to Camille to provide an update on our clinical and medical affairs progress this quarter. Camille Bedrosian: Thank you, Dan. To start, PBH is a chronic metabolic condition driven by an exaggerated GLP-1 response, primarily after food intake, resulting in persistent recurrent and often debilitating hypoglycemia. These events cause an inadequate supply of glucose to the brain known as neuroglycopenia with potential clinical consequences such as cognitive dysfunction, seizures and loss of consciousness. For people living with PBH, this can create a life of perpetual vigilance where a meal with friends or a drive to work carries the risk of debilitating hypoglycemia and its ramification. This fear can disrupt independence and compromise safety, nutrition and overall quality of life. Currently, there are no FDA-approved therapies. Our pivotal Phase III LUCIDITY trial is evaluating avexitide, 90 milligrams once daily, in individuals with PBH following Roux-en-Y gastric bypass surgery using the FDA agreed-upon primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through week 16. LUCIDITY was designed with the goal of replication. Five prior avexitide trials in PBH, which demonstrated statistically significant results, including reductions in hypoglycemic events directly informed the dose, the primary endpoint, inclusion criteria and surgical subtype for LUCIDITY. Echoing Justin's earlier remarks, our clinical team remains deeply focused on the execution of the LUCIDITY trial, and we continue to work closely with our investigators as we approach our anticipated data readout next quarter. In parallel with our clinical trial execution, we are actively ramping up our field medical affairs team to facilitate on-the-ground engagement with KOLs. I also am pleased to share that we recently launched a U.S. expanded access program to provide avexitide for up to 250 adults with PBH following Roux-en-Y gastric bypass. This program is a direct response to the urgent need we are hearing from individuals who are struggling with the devastating daily realities of PBH and the physicians who treat them. Initial eligible patients include individuals who have either completed LUCIDITY or participated in previous clinical trials of avexitide in PBH. As a reminder, avexitide is an investigational drug and has not been approved by the FDA for any indication. Working directly with the PBH community and seeing the everyday impact of this devastating condition drives our continued commitment to our clinical and medical efforts. And with that, I will now turn over the call to Jim to review our financials. Jim? James Frates: Thanks, Camille. Our financial results for the first quarter were in line with our plans and reflect our focus on the Phase III LUCIDITY trial and targeted investments in advancing our broader pipeline. We ended the fourth quarter with $279.8 million in cash and marketable securities compared to $317 million at the end of the fourth quarter of last year. This capital funds our anticipated cash runway into 2028, including our key expected milestones: the LUCIDITY top line readout expected in Q3 2026, potential FDA approval and potential commercial launch of avexitide in 2027. Turning now to our results for the quarter. Total operating expenses for the quarter were $43.8 million, up 16% from the same period in 2025. Research and development expenses were $27.6 million compared to $22.1 million in Q1 2025. The increase was primarily due to an increase in spending related to the clinical development of avexitide in PBH. This quarter, we also recognized a milestone payment of $4 million to Gubra following the identification of AMX0318 as a development candidate for PBH and other rare diseases. The increase was offset by decreased spending related to the clinical development of AMX0035 for progressive supranuclear palsy. Selling, general and administrative expenses were $16.2 million compared to $15.7 million in Q1 2025. This increase was primarily due to an increase in consulting and professional services as we prepare for the potential commercial launch of avexitide. We recognized $6.1 million of noncash stock-based compensation expense for the quarter compared to $6.8 million of noncash stock-based compensation expense in Q1 2025. Turning to our balance sheet. Our cash usage was slightly higher in Q1 compared to Q4 because of our Gubra milestone payments and the payment of our annual corporate bonus during the quarter. We're in the midst of a pivotal year for Amylyx with the top line data readout for LUCIDITY expected in Q3. The team will continue to focus on scaling our business with discipline, and we're actively laying the groundwork for a potential commercial launch. This focus positions us well, particularly for our work with avexitide. We continue to believe Avexitide has the potential to be a breakthrough treatment for PBH. With that, I'll turn the call over to Josh. Joshua Cohen: Thanks, Jim. To close, we are focused on the execution of the LUCIDITY trial as we track toward our anticipated top line readout next quarter. PBH is a chronic lifelong condition with symptoms that often emerge 1 to 3 years following bariatric surgery. Many people who receive bariatric surgery are in their 40s, suggesting that if they develop PBH, they may have decades of life impacted by this condition. The broader medical community continues to recognize this critical need in PBH. In March, Dr. Colleen Craig and her colleagues at Stanford published the first U.S. prevalence model for PBH in surgery for obesity and related diseases, the official peer-reviewed journal of the ASMBS. And in April, CMS published their annual list of ICD-10 codes to be potentially effective October 1, 2026, which includes an ICD-10 code specific to PBH. The planned adoption of an ICD-10 code shows the growing recognition of this condition by the medical community. We believe that avexitide, if approved, could play a meaningful role in addressing this highly underserved patient population. In parallel with LUCIDITY, we are actively preparing for a regulatory submission following top line results while simultaneously scaling our commercial and medical teams to support a strong commercial launch of avexitide in 2027, if approved. With that, I would like to now open the call up for questions. Operator: [Operator Instructions] Your first question comes from the line of Seamus Fernandez with Guggenheim. Seamus Fernandez: I hope you'll bear 2 for me quickly. The first question is really on the initiation of the EAP. Typically, we see companies sort of waiting for the completion of their Phase III and then the announcement of an EAP in the wake of a positive Phase III. Just wanted to get a better sense of, obviously, how you were able to execute this and get it approved, and then also how you are really responding to the community with the implementation and announcement of the EAP. And then just a quick follow-up question. I wanted to get a sense of just sort of that relative impact that working on the NDA now could actually have from a filing perspective. Typically, when we see biotech companies with positive Phase III data, it will take as much as 6 to 9 months to see that file. So just wanted to get a sense of how working on the NDA now might advance that ahead of those types of time lines. Camille Bedrosian: Thank you very much, Seamus. This is Camille. So for the EAP, indeed, we are responding to the community where there is currently no approved therapies for PBH, and we recognize and have received several requests and demands. Importantly, we're starting the EAP now because we want to be sure there's continuity of treatment for people in the LUCIDITY study who are completing the OLED portion of the LUCIDITY trial. So that drives the timing for the EAP. Now with regard to your question about NDA preparation, Yes, we're sort of not typical for sure. And we are working, as noted, on NDA preparations. And we do hope that, that will allow us to be most efficient as we reach top line data next quarter. And because there is a great sense of urgency, there are no treatments for people with PBH. And if positive, we want to be sure that we're providing the opportunity for access as promptly as possible. Joshua Cohen: Yes. And maybe just -- maybe underscore from Camille as well. I think both of these activities both underscore too, the unmet need in PBH. We know that patients urgently need a new therapy and also our excitement about avexitide. We've had 5 prior trials of avexitide, all which showed very strong results. We have breakthrough therapy from FDA. So we want both to get patients access as quickly as possible, which, of course, is reflected with the EAP, but also, pending positive results, to be able to submit as soon as we possibly can, which is reflected by our ongoing work on the NDA. Justin Klee: And just one more thing to add, thank you, Seamus, is we also have a very experienced team here. Our team has experience with global regulatory approval, certainly a lot of experience with FDA as well. And so that allows us to start working on the NDA documents now. Again, everything is driven by the urgent unmet need for people with PBH, but I think coupled with the strong experience we have here, both for regulatory submissions and process as well as commercialization. Operator: Your next question comes from the line of Joseph Thome with TD Cowen. Unknown Analyst: This is Jacob on for Joe. We were wondering how the baseline for the enrolled Phase III population compare to the patients in the Phase II studies, and then what the expected placebo response in Phase III might be versus what we know about the patients in Phase II and the differences in the run-in periods. Camille Bedrosian: Sure. So the study is ongoing and blinded. And so we will not really comment on the details of this ongoing study. Having said that, we are very much looking forward to top line data next quarter where we'll share the top line data with you and hope you're sharing our excitement as well for this possibility. With regard to the placebo, remind as well, we've had 5 prior highly successful trials. The Phase II trial showed statistical significance and clinically meaningful reductions in the composite as we presented at ENDO. In the PREVENT study, 55% reduction with a highly statistically significant value, and with -- in the Phase IIb with a 90-milligram dose, the dose in LUCIDITY, 64% reduction with a p-value of 0.0031. Those p-values do take into account all aspects of the trial, including the possibility of any placebo effect. We designed LUCIDITY with the goal of replicating these prior successful trials. So -- and we powered -- we're highly powered, 90%, to detect a clinically meaningful reduction in events even under the most conservative circumstances. Operator: Your next question comes from the line of Corinne Johnson with Goldman Sachs. Kevin Strang: This is Kevin on for Corinne. Could you just talk about the steps that you've taken beyond the event rate quota to ensure patient quality in the study for LUCIDITY and then also to ensure, sort of as much as possible, adherence to study protocols for the full 16-week treatment period? Camille Bedrosian: Sure. So we -- again, we're replicating as much as possible the way LUCIDITY is being conducted, mirroring what was done in the successful Phase II and Phase IIb studies. We have training -- extensive training at the clinical sites at the onset of the clinical trial. That training is reinforced throughout the conduct. And we also have materials for the participants to guide them on the study procedures throughout the study as well. And we also do have quality checks on the data overall to be sure that things are moving along well. Joshua Cohen: Yes. And I'd just add too, we have a very experienced team at Amylyx and quality starts from selecting great sites, having strong oversight. And I think throughout the whole course of the study, I've been quite proud of the team's efforts, just kind of continually keeping close and making sure that quality is kind of built in from the start and continues through the whole study. Operator: Your next question comes from the line of Michael DiFiore with Evercore ISI. Michael DiFiore: Congrats on all the continued progress. First one for me is, now that LUCIDITY is fully enrolled, can you help us think through what the top line disclosure will actually look like, what it will contain, beyond whether the primary endpoint is met? What do you think will be the most important aspect of the data for people to understand, again, beyond the primary endpoint in that initial release? And secondly, since you're already preparing for NDA, since the last update, can you share more light on what work remains to be done between top line and potential submission, maybe in terms of QC, any additional studies, et cetera? Justin Klee: Yes. Thank you, Mike. So I think first, in terms of top line disclosure, you know us well. We're a transparent company. So our goal is always to present things as they are. I think what's important in this study is probably 2 things to remind. The first is that the primary outcome, which is Level 2, Level 3 hypoglycemic events, not only is it the primary outcome, it's in FDA guidance, but it's also very well known by endocrinologists and it's inherently clinically meaningful. Level 2 events being less than 54 milligrams per deciliter blood glucose, which is the blood value at which neuroglycopenia occurs. Level 3, of course, means that the clinical manifestations of hypoglycemia have already occurred. So those are inherently clinically meaningful. And then I think a second important point is that there are currently no treatments for PBH. And so what we have heard consistently, not just from people with PBH, but from physicians as well is that any reduction in hypoglycemic events is meaningful. Each one of these events is a medical emergency. And so what we hear from physicians often is that they are worried for their patients, and they have really very few tools to help prevent these medical emergencies. So any reduction in these hypoglycemic events is meaningful. In terms of the NDA, we're working hard on everything we can now. I think the goal would be that the last really substantial piece of work would be everything associated with the Phase III trial. So we're trying to write everything in advance that we can. As I said, we have a very experienced team who's been through many regulatory submissions before. So they're hard at work as we speak. Operator: Your next question comes from the line of Marc Goodman with Leerink Partners. Marc Goodman: This process of adjudicating the claims data, can you give us an example or 2 of just some of these efforts and just so we understand what you have done so far and how confident you are that you're finding these patients in the places that you think they are based on the claims data? And are you only counting, like, the moderate to severe ones, you're not counting the benign ones, right? Dan Monahan: Thanks, Marc. Appreciate the question. Just to talk a little bit about the claims analysis, so within the claims databases, we start with identifying patients that have a presence of bariatric surgery. And then, we look at patients who also have documented hypoglycemia, so nondiabetic hypoglycemia. And then, after that, we'll then apply and we have applied additional signs and symptoms associated with PBH such as fatigue, dizziness, seizures, even blood glucose tests or even ER visits. And then, to add to that, we can look at it from how many of those type of events they've also had within the claims databases. So that's really how we've continued to look at the databases. I'll say we've done it several times. And each time we do it, we are confident in the 160,000 patient population that we've mentioned a few times. Joshua Cohen: Yes. And I might just add, too, some added work the team has done, too, is both speaking to many of the sites and doing kind of market research with many of the sites to validate what we're finding in claims. For example, if the claims are saying a site has 50 patients, actually checking with the site and seeing if they do have 50 patients. And so far, those have been quite confirmatory as well. And I'll also just add from the call today, we also received notification kind of through the CMS manual list that there's likely to be an ICD-10 code for PBH going live in October, which will provide an additional tool kind of to track the claims data as well. Justin Klee: Yes. And directly to your point, too, on excluding benign, based on the coding, both on hypoglycemia, as well as the signs and symptoms, these are people who have severe hypoglycemia. Operator: Your next question comes from the line of Geoff Meacham with Citibank. Geoffrey Meacham: I have 2 quick ones. So ahead of LUCIDITY top line, I know the primary outcome measure is Level 2 or 3 events as a composite. But is there a thought of looking at each one of those separately, in particular, Level 3, just to have a cleaner look at the profile from maybe a more commercial context? And then, the second question. As you guys begin to focus on commercial and further evaluate the PBH prescriber base, how has your thinking evolved in terms of size and scope of sales force and MSL teams? Camille Bedrosian: Geoff, I'll take your first question and then pass to Dan for your second. So, as you say, our primary endpoint, which is FDA agreed upon, we were looking at the reduction in the composite of Level 2 and Level 3 events, and that is well established in the ADA, Diabetes Association, literature and community as well. We do intend as well -- our secondary endpoints are looking separately at Level 2, which is by fingerstick blood glucose level of less than 54 grams per deciliter, and separately Level 3, which is independent of glucose level, signs and symptoms that require individuals to have another individual help or signs and symptoms that would have required someone else to help them if no one else is around. And that is adjudicated by an independent group of experienced endocrinologists who are blinded to the data as well, and they do that adjudication on an ongoing basis. Justin Klee: And I'll just add, too, from the sort of commercial point of view, so you're right, absolutely, Level 3 means the person has had the manifestation of hypoglycemia. And so, of course, that's important. But Level 2 being less than 54 is very well established, too. So I think endocrinologists will be interested in both. And if you think about the outcome, it's really a nice mix. You have a blood value which indicates severe hypoglycemia, so you kind of know what's happening in the body. And then, you have a clinical outcome in Level 3, where you know the person has had the impact of severe hypoglycemia. And then, for your question on prescriber base, I'll turn it to Dan. Dan Monahan: Geoff, thanks for the commercial question. So, on the sales force sizing, we are initiating the go-to-market efforts at this moment. I would say, this is a rare endocrine launch. So from an expectation -- you can expect that the sales force would reflect this particular size of the sales force. I'd also add that on Camille's team and the medical affairs function, we have initiated hiring our regional Scientific Director team, also known as an MSL team, but those hires are in place. Operator: Your next question comes from the line of Rami Katkhuda with LifeSci Capital. Rami Katkhuda: I guess, can you touch on the degree of natural variability there is in Level 2 and Level 3 hypoglycemic events for PBH patients? Do you expect a massive difference from one week to another? And then, secondly, from a commercial perspective, are these PBH patients generally managed at centers of excellence? Or would you need to target endocrinologists more broadly? Joshua Cohen: Sure. Maybe starting with the variability. So all of that's taken into account in our powering analysis, and I think we were quite conservative in our powering, both on the effect size and on the placebo effect, whereas we saw a 50% and 64% effect size at the 60 and 90 mg doses, we powered to a 35% effect, and then, of course, retaining power up to a 50% placebo effect, even though I don't think we expect that in this condition. You can certainly look at the variability kind of from previous studies. But again, that's all kind of accounted for in our powering analysis. And generally, it's a chronic condition. Generally, people are not able to prevent these events from occurring. So often, if people are having events, that will be a continuous thing. They don't kind of come in fits and starts, so to speak, all that often. In terms of the question of how we'll target centers of excellence versus the broader endo community, I'll pass it over to Dan. Dan Monahan: Sure. Thanks, Josh. And I appreciate the question on the potential [indiscernible]. On the -- where the patients are potentially treated, so we have initiated that work, and we are aware, and Josh mentioned this earlier, but there are centers of excellence. There are also key opinion leaders, and that's likely where we'll start from a launch perspective. We know that there's potentially 50 to 60 patients at certain centers, and some centers have even mentioned even more. But we'll start there. We know there's a concentration. And then, as our disease state education efforts take a foothold, we'll expand into the broader endocrinology community. Operator: Your next question comes from the line of James Condulis with Stifel. Unknown Analyst: This is Mark on for James. One for me on Recordati. I believe we should be getting some data this quarter. And just curious your thoughts here as it relates to potential placebo effect and what the implications are for LUCIDITY and whether really this is something that can actually be sort of read through on your trial. Justin Klee: Yes. Thank you, Mark. So I would say, no, I wouldn't think there should be any read-through. They're very different studies. And of course, we're conducting our study and Recordati is conducting their study. As I understand it, I think their study is a Phase II, looking at mixed meal tolerance test. And ours is based on the prior Phase IIs, which is a much more real-world type approach, looking at Level 2, Level 3 hypoglycemic events, which, of course, is within FDA guidance, to support a potential registration. So, no, I don't think there should be any read-through between the studies. Camille Bedrosian: Plus the mechanisms of the drugs are very different as well. Operator: Your next question comes from the line of Graig Suvannavejh with Mizuho. Samuel Lee: This is Sam on for Graig. Maybe switching over to 0114 with the ALS data coming up shortly, can you just remind us of the specific biomarkers potential [indiscernible]? I know there was some prior analysis done by you guys highlighting certain biomarkers. But maybe just a reminder, and then also some of the expectations you guys have or we should be thinking about going ahead into the data. Camille Bedrosian: Sure. Thanks very much. So just to remind, our ALS study with AMX0114, which is an ASO against calpain-2, is ongoing. We announced that we completed enrollment of Cohort 1 in March and that we are recruiting Cohort 2 at the moment. And earlier this year, we reported on the safety data for Cohort 1. And we do anticipate, as you point out, reporting on the biomarker data. Actually, it will be this June at ENCALS in Madrid, Spain. So the biomarkers that we're studying are related to the mechanism of the calpain-2 ASO, blocking this protease, as well as biomarkers that are related also to the ALS disease process. And we look forward very much to sharing those data with you. Joshua Cohen: And just to add as well, as Camille said, the study is proceeding incredibly well. So I think as Camille was mentioning, we've completed enrollment in Cohort 2, and we're now recruiting for Cohort 3 as well. Operator: Your next question comes from the line of Jason Gerberry with Bank of America. Unknown Analyst: This is [indiscernible] on for Jason. Maybe just a couple of commercial questions on avexitide. I think you've mentioned before that the ICD-10 code was not necessary for successful commercialization. But just curious how ultimately having an ICD-10 code kind of alters your confidence in identifying and capturing patients at scale. I know you've outlined the centers you're targeting and what your commercial strategy is, but just curious if it impacts how you're approaching your commercial plan. And then, just a second quick follow-up. I believe you plan to position avexitide as a chronic therapy. I'm just curious what your -- what assumptions you're making around expectations for persistence and adherence in the real world. Dan Monahan: Great. Thanks, [ Tina ]. So, on the ICD-10 question, so in April, CMS, they published a list of ICD-10 codes to be effective October 1. These codes demonstrate a recognition of PBH in the broader medical community. The ICD-10 code, yes, it is helpful for diagnosis and tracking of patients. However, it's not necessarily -- it's not a necessity. ICD-10 codes are often used for epidemiology. So, in implementation of the code, this will enable patients to be tracked across the various electronic medical record systems. It's also important to note that patients, today, they still can be identified via the claims analysis, and that's how we validated the 160,000 patient population. Joshua Cohen: And then, to your other question, too, about kind of chronic therapy and persistence, it's probably early to comment there. We're quite excited about avexitide. And PBH is a chronic condition where the needs do not go away over time. So certainly, we do think that patients will have an ongoing need for therapy. Operator: Your next question comes from the line of Chris Chen with Baird. Christopher Chen: Congrats on the progress. Just a quick one on the OLE. Can you just remind us what the setup is specifically for that? And are you able to kind of share high level how enrollment in that is going? Camille Bedrosian: Sure. So, are you speaking of the OLE or the EAP, just so I'm clear, please? Christopher Chen: The OLE for LUCIDITY, the... Camille Bedrosian: Open-label extension? Christopher Chen: Open-label extension, yes. Camille Bedrosian: Yes. So while I will not comment on details of our ongoing blinded trial, I am pleased to share that LUCIDITY is proceeding well, including participants transitioning from the double-blind period to the OLE portion of the study. We're confident that we're running the right study, and we're very pleased on how the team has been executing on the trial. We have such an experienced team, and they're overseeing the trial with great focus and care. Joshua Cohen: Yes. And you asked the OLE setup. So just to add there as well, so we expect the randomized double-blind study is the study that will -- we expect to use in our NDA to support potential commercialization. The OLE itself, though, also has a Part A and a Part B. During the Part A, we keep the study very similar to how it's conducted during the double blind. That allows to look at kind of data in a similar way to we look at as the double blind. And then later, they enroll in the [ OLE B ] after 8 weeks in the OLE, at which point, it's a little more -- there's less -- it's a less burdensome kind of trial participation at that point. But overall, just to kind of reiterate what Camille said as well, we're very pleased with the conduct of the study. There's a lot of excitement from sites and otherwise as well, and we'll look forward to reporting our data in Q3. Justin Klee: And I think it's obvious, but just to say also, for the open-label extension, I think it's very important when you work in rare debilitating conditions like post-bariatric hypoglycemia that you always try to think about the people we are trying to help. And so, for an open-label extension, if you're on treatment and you believe that you're benefiting in open-label extension, allows you to continue. And if you are randomized to placebo, then allows you to take active medication. So that's something that we always really try to think about in our programs. Operator: Your next question comes from the line of Ananda Ghosh with H.C. Wainwright. Ananda Ghosh: Maybe one question. People have been focusing on U.S. opportunities. So one question would be, have you done work with respect to avexitide on ex-U.S. opportunities? What are you hearing from the KOLs or stakeholders? And then, I have one follow-up question on LUMINA. Justin Klee: Absolutely. Thank you, Ananda. So there's a tremendous unmet need globally for post-bariatric hypoglycemia. Now, our focus is very much on the U.S. right now with 160,000 people in the U.S. with PBH today. That's a substantial unmet need and people to help and address. But our -- first of all, bariatric surgeries occur globally. And also, as we mentioned before, virtually any gastric surgery has the potential to cause the same debilitating hypoglycemia. So, for example, in major Asian countries, gastric cancer rates, esophageal cancer rates are very high. And so, gastrectomy or esophagectomy is often indicated. And so, for people with those surgeries, they also have the potential of developing the same debilitating hypoglycemia, and we have data from the Phase IIb study of avexitide that avexitide may be beneficial for people who had those surgeries leading to this debilitating hypoglycemia as well. The pathophysiology is the same regardless of the surgical intervention. So, our focus is really on the U.S., really on the U.S. population of post-bariatric hypoglycemia. But absolutely, there's a huge unmet need internationally. We get compassionate use requests from people around the world constantly. Ananda Ghosh: Got it. One question on LUMINA. I know there was a question on biomarkers. So given that it's the lowest dose, do we -- are we -- can we expect the preliminary NfL data or target engagement data with respect to calpain-2 levels or other downstream markers like SBDP-145 in the data readout, or that is for later... Joshua Cohen: Yes. I mean, it's hard to know -- sorry to interrupt. It's hard to know until we have the data. I'd say, we do preclinically believe we have a potent ASO. As you look at past ASOs that have been in clinic, usually, they've been studied between the range of generally about 10 mgs to 100 mgs for CSF injection. And we're at the very low end of that range. So we may see signals, but it also may require us to go to a higher dose before we start significantly moving the biomarkers. Justin Klee: But I'll add, too, Ananda, to your point, we really are -- our goal is to have a picture of, first, are we replicating the biology that we saw in the preclinic to the clinic? Are we seeing the implications of calpain-2 knockdown? And then, are we seeing effects on biomarkers that we believe to be prognostic for ALS as well? So that is indeed the goal of the biomarkers in all of these cohorts is to try to get a picture of are we seeing the impacts of calpain-2 and are we seeing potential impacts on ALS as well? Operator: Thank you. There are no further questions at this time. I'll turn the call back to Mr. Klee. Justin Klee: Thank you, operator, and thank you all for your time. If you have any follow-up questions, please reach out to Lindsey. And we hope you have a great rest of your day. Operator: Thank you, everyone. Before you buy stock in Amylyx Pharmaceuticals, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the 10 best stocks for investors to buy now… and Amylyx Pharmaceuticals wasn’t one of them. The 10 stocks that made the cut could produce monster returns in the coming years. 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As with all our articles, The Motley Fool does not assume any responsibility for your use of this content, and we strongly encourage you to do your own research, including listening to the call yourself and reading the company's SEC filings. Please see our Terms and Conditions for additional details, including our Obligatory Capitalized Disclaimers of Liability. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy. Amylyx (AMLX) Q1 2026 Earnings Transcript was originally published by The Motley Fool

Interested in Amylyx Pharmaceuticals, Inc.? Here are five stocks we like better. Avexitide Phase 3 LUCIDITY completed dosing and the company says it’s on track for a near-term top-line readout, with management and the CFO pointing to potential readout timing around Q3 2026. Expanded access program (EAP) launched in the U.S. to provide avexitide to up to 250 adults with post‑bariatric hypoglycemia after Roux‑en‑Y gastric bypass, intended to ensure continuity of treatment for trial participants; the drug remains investigational. Commercial and financial readiness: Amylyx is drafting NDA sections, building a launch organization and disease‑awareness efforts targeting an estimated ~160,000 U.S. PBH patients, and ended Q1 with $279.8 million in cash to fund its runway into 2028 while preparing for a potential 2027 launch if approved. 3 small-cap biotechs with potential breakthroughs in 2024 Amylyx Pharmaceuticals (NASDAQ:AMLX) executives highlighted continued progress across the company’s pipeline during its first-quarter 2026 earnings call, with a heavy focus on the pivotal Phase 3 LUCIDITY trial evaluating avexitide in post-bariatric hypoglycemia (PBH) and preparations for a potential 2027 commercial launch if the drug is approved. Co-CEO Justin Klee said the quarter was “marked by execution across our pipeline,” pointing first to the LUCIDITY study of avexitide, which the company describes as an investigational first-in-class GLP-1 receptor antagonist with FDA Breakthrough Therapy designation in PBH. Klee said the company randomized and dosed the last participant in late March, calling it “a significant milestone,” and said the company remains “on track for a top-line readout next quarter.” → Berkshire Hathaway’s Record Cash Hoard: Why and What's Next? These biotechs targeting multiple neurodegenerative diseases Chief Medical Officer Dr. Camille L. Bedrosian described PBH as a chronic metabolic condition driven by an exaggerated GLP-1 response after food intake, leading to recurrent hypoglycemia and neuroglycopenia. She emphasized there are currently no FDA-approved therapies. Bedrosian said LUCIDITY is evaluating avexitide 90 mg once daily in PBH following Roux-en-Y gastric bypass surgery, using an FDA-agreed primary endpoint: reduction in the composite of level 2 and level 3 hypoglycemic events through week 16. Bedrosian said LUCIDITY was designed with “the goal of replication,” noting that five prior avexitide trials in PBH informed the dose, endpoint, and inclusion criteria. In Q&A, management said they intend to report additional information beyond the primary endpoint, including secondary endpoints that look separately at level 2 and level 3 events. Bedrosian explained that level 3 events are adjudicated by an independent group of endocrinologists blinded to study data. → A Prada Payday: Is AMC Back in Style? Amylyx Pharmaceuticals Turns Profitable On Successful Drug Launch When asked about comparing baseline characteristics in Phase 3 versus earlier studies and the potential placebo response, Bedrosian said the trial remains ongoing and blinded and the company would not comment on details of the study population. She added that prior Phase 2 trials showed statistically significant and clinically meaningful reductions in hypoglycemic events and that the Phase 3 study was powered to detect a clinically meaningful reduction “even under the most conservative circumstances.” Bedrosian also disclosed that Amylyx recently launched a U.S. expanded access program (EAP) to provide avexitide for up to 250 adults with PBH following Roux-en-Y gastric bypass surgery. She said the program was launched in response to requests from patients and physicians and is intended to provide continuity of treatment for patients completing portions of the LUCIDITY program. Initial eligible patients include adults who have completed LUCIDITY or participated in previous avexitide PBH trials. → Insider Sales: Top AST SpaceMobile Insider Cuts Postion Over 30% “We’re starting the EAP now because we wanna be sure there’s continuity of treatment for people in the LUCIDITY study,” Bedrosian said in response to a question about the timing of the program relative to typical Phase 3 timelines. Management repeatedly emphasized that avexitide remains investigational and is not approved by the FDA for any indication. Klee said Amylyx is advancing three “strategic imperatives” for avexitide: moving toward top-line data, strengthening NDA readiness, and building launch readiness. He said the company is already drafting NDA sections to support a potential submission. In Q&A, Bedrosian said the company’s early NDA work is intended to improve efficiency after top-line results. She cited a “great sense of urgency” given the lack of approved therapies in PBH, adding that the company has an experienced team with prior regulatory submission experience. Chief Commercial Officer Dan Monahan outlined efforts to prepare for a potential U.S. launch, including hiring across marketing, market access, and commercial operations, and a near-term focus on PBH disease state education. Monahan said the company plans to launch a PBH awareness campaign this summer, emphasizing pathophysiology, timely diagnosis, and disease burden. Monahan said Amylyx’s independent claims analysis and field engagement support its estimate of approximately 160,000 people living with PBH in the U.S. following sleeve gastrectomy and Roux-en-Y gastric bypass. He added that market research indicates endocrinologists have a “high intent to treat PBH if there were to be an approved medicine.” When asked how the company identifies patients using claims data, Monahan said the analysis begins with bariatric surgery patients and then filters for documented non-diabetic hypoglycemia, followed by additional signs and symptoms associated with PBH, such as fatigue, dizziness, seizures, blood glucose tests, and emergency room visits. He said repeated analyses have reinforced the company’s confidence in its prevalence estimate. Management also said it is validating claims findings through outreach to clinical sites. Co-CEO Josh Cohen also pointed to signals of growing recognition of PBH, citing a March publication from Stanford researchers modeling U.S. prevalence and noting that CMS published a list of ICD-10 codes potentially effective Oct. 1, 2026, that includes a PBH-specific code. Management said an ICD-10 code would be helpful for tracking and diagnosis but is not required for commercialization given other identification methods such as claims analysis. On commercial deployment, Monahan said the company is initiating go-to-market efforts and suggested salesforce expectations consistent with a “rare endocrine launch.” He also said medical affairs has begun hiring regional scientific directors (MSLs), with hires in place. He added that the company expects to begin with centers of excellence and key opinion leaders and later expand outreach into broader endocrinology as disease state education progresses. Klee provided updates across other programs: AMX0318: The company’s long-acting GLP-1 receptor antagonist has IND-enabling studies underway, with a 2027 IND filing targeted. AMX0035 (Wolfram syndrome): Amylyx expects to present longer-term week 96 data from the Phase 2 open-label HELIOS trial at an upcoming scientific meeting. AMX0114 (ALS): The company fully enrolled cohort 2 of the Phase 1 LUMINA trial in March. Management said it expects to present early biomarker data from cohort 1 in June at ECTRIMS in Madrid, Spain. Bedrosian said the biomarkers include measures related to the calpain-2 ASO mechanism as well as biomarkers related to the ALS disease process. During Q&A, management said it is difficult to predict biomarker movement at the lowest dose cohort for AMX0114 until data are in hand, noting the dose is at the low end of ranges typically studied for intrathecal ASOs. The company said its biomarker goal is to assess whether the clinical data replicate preclinical biology and whether there may be impacts on prognostic ALS biomarkers. Chief Financial Officer James Frates said first-quarter results were “in line with our plans” and reflected focus on the Phase 3 LUCIDITY trial and “targeted investments” across the pipeline. The company ended the quarter with $279.8 million in cash and marketable securities, compared with $317 million at the end of the fourth quarter of the prior year. Frates said the company expects that capital to fund its anticipated cash runway into 2028, including expected milestones such as the LUCIDITY top-line readout in Q3 2026 and potential FDA approval and commercial launch of avexitide in 2027. Total operating expenses were $43.8 million, up 16% from the same period in 2025. R&D expenses rose to $27.6 million from $22.1 million, driven primarily by increased spending related to avexitide clinical development. Frates also noted a $4 million milestone payment to Gubra following identification of AMX0318 as a development candidate for PBH and other rare diseases. SG&A expenses were $16.2 million, compared with $15.7 million, primarily due to higher consulting and professional services costs tied to launch preparation for avexitide. Frates said cash usage was slightly higher in Q1 than Q4 due to the Gubra milestone payments and payment of the annual corporate bonus. Closing the call, Cohen reiterated the company’s focus on LUCIDITY execution and said Amylyx is preparing for a regulatory submission after top-line results while scaling commercial and medical teams for a potential 2027 launch “if approved.” Amylyx Pharmaceuticals, Inc is a biopharmaceutical company dedicated to developing treatments for rare and debilitating neurological diseases. Founded in 2013 and headquartered in Cambridge, Massachusetts, the company focuses on leveraging novel approaches to target cellular pathways implicated in neurodegeneration. Amylyx's research platform centers on small-molecule therapies designed to protect neurons and support cellular health in patients with conditions that currently have limited or no disease-modifying treatment options. The company's lead product, AMX0035, is marketed under the trade name Relyvrio following U.S. The article "Amylyx Pharmaceuticals Q1 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for May 2026.

Topline data readout from Phase 3 LUCIDITY clinical trial of avexitide in post-bariatric hypoglycemia on track; anticipated in Q3 2026 Cash runway expected to fund operations through potential avexitide commercialization and into 2028 Management to host conference call and webcast today at 8:00 a.m. Eastern Time CAMBRIDGE, Mass., May 07, 2026--(BUSINESS WIRE)--Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) ("Amylyx" or the "Company") today reported financial and business results for the first quarter ended March 31, 2026. "With enrollment complete in the pivotal Phase 3 LUCIDITY clinical trial, we have a clear line of sight to the anticipated Q3 topline data readout, bringing us one step closer to the potential of delivering the first approved therapy for the post-bariatric hypoglycemia community," said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. "We understand the devastating daily burden of this condition and are operating with a sense of urgency to advance our program. We have initiated regulatory and commercial readiness activities to help ensure we are positioned to move swiftly following LUCIDITY topline data. Supported by an expected cash runway extending into 2028, we are executing with focus and discipline as we work to bring this treatment to the PBH community in 2027, if approved." First Quarter and Recent Updates: Amylyx completed enrollment for the pivotal Phase 3 LUCIDITY clinical trial of avexitide, an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist with U.S. Food and Drug Administration (FDA) Breakthrough Therapy Designation in post-bariatric hypoglycemia (PBH), in March 2026. LUCIDITY enrolled 78 participants and is a 16-week, multicenter, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of avexitide in adults with PBH following Roux-en-Y gastric bypass (RYGB) surgery. Participants who complete the 16-week double-blind period are eligible to enter a 32-week open-label extension (OLE) period. Amylyx announced the initiation of an Expanded Access Program (EAP) for the use of avexitide to treat U.S. adults with PBH following RYGB surgery in May 2026. Initial eligible patients include individuals who have completed the pivotal Phase 3 LUCIDITY clinical trial and participants in a prior trial of avexitide in PBH following RYGB surgery. Amylyx completed enrollment of Cohort 2 (n=12) of the Phase 1 LUMINA clinical trial of AMX0114, an investigational antisense oligonucleotide (ASO) targeting calpain-2 with FDA Fast Track Designation for the potential treatment of amyotrophic lateral sclerosis (ALS), in March 2026. The LUMINA trial is a randomized, double-blind, placebo-controlled, multiple ascending dose clinical trial of AMX0114 in people living with ALS. LUMINA is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114 in people living with ALS and assessing both novel and broadly researched ALS biomarkers, including change from baseline in neurofilament light chain (NfL) levels. Upcoming Expected Milestones: Topline data readout for Phase 3 LUCIDITY clinical trial of avexitide in PBH is on track and anticipated in the third quarter of 2026. If approved, commercial launch of avexitide is anticipated in 2027. LUCIDITY is evaluating the FDA-agreed-upon primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. LUCIDITY was informed by data from five prior PBH clinical trials of avexitide showing consistent effects, most notably statistically significant reductions in Level 2 and Level 3 hypoglycemic events. Avexitide was generally well-tolerated, with a favorable safety profile replicated across previous clinical trials. Presentation of early biomarker data from Cohort 1 (n=12) of the Phase 1 LUMINA clinical trial of AMX0114 in ALS is expected at the 2026 European Network to Cure ALS (ENCALS) Annual Meeting in June 2026. Cohort 1 of LUMINA is investigating the first and lowest of four doses being evaluated in the trial. The data are expected to provide initial information about the levels of the ALS biomarkers being assessed from the first dose in the LUMINA trial. The Company previously presented early safety and tolerability data from Cohort 1 of LUMINA showing AMX0114 was generally well-tolerated, with no treatment-related serious adverse events (SAEs). Investigational New Drug (IND)-enabling studies for AMX0318, a novel GLP-1 receptor antagonist for long-acting administration to treat PBH and other rare diseases, are underway with an IND filing targeted for 2027. AMX0318 was selected as a development candidate after demonstrating robust preclinical and chemical properties, including a favorable pharmacokinetic profile that may support long-acting administration, a robust chemical stability profile, strong in vitro potency, evidence of in vivo activity and tolerability, and high solubility. AMX0318 was identified through a research collaboration with Gubra A/S (Gubra), a company specializing in peptide-based drug discovery and preclinical contract research services. Financial Results for the First Quarter Ended March 31, 2026 R&D Expenses: Research and development expenses for the first quarter of 2026 were $27.6 million, compared to $22.1 million for the same period in 2025. The increase was primarily due to an increase in spending related to the clinical development of avexitide in PBH. Milestone payments totaling $4.0 million to Gubra were also recognized following the selection and handover of AMX0318 as a development candidate for PBH and other rare diseases. The increase was offset primarily by decreased spending related to AMX0035 for the treatment of progressive supranuclear palsy (PSP). Research and development expenses include $1.8 million of stock-based compensation expense for the quarter ended March 31, 2026, compared to $1.8 million of stock-based compensation expense for the quarter ended March 31, 2025. SG&A Expenses: Selling, general, and administrative expenses for the first quarter of 2026 were $16.2 million, compared to $15.7 million for the same period in 2025. This increase was primarily due to an increase in consulting and professional services. Selling, general, and administrative expenses include $4.4 million of stock-based compensation expense for the quarter ended March 31, 2026, compared to $5.0 million of stock-based compensation expense for the quarter ended March 31, 2025. Net Loss: Net loss for the three months ended March 31, 2026 was $41.3 million, or $0.37 per share, compared to net loss of $35.9 million, or $0.42 per share, for the same period in 2025. Cash Position: Cash, cash equivalents, and short-term investments were $279.8 million at March 31, 2026, compared to $317.0 million at December 31, 2025. Based on its current operating plans, Amylyx expects a cash runway into 2028. Investor Conference Call Information Amylyx’s management team will host a conference call today, May 7, 2026, at 8:00 a.m. ET to discuss financial results and provide an update on the business. To access the conference call, please dial +1 (888)-880-3330 (U.S. & Canada) or +1 (646)-357-8766 (international) at least 10 minutes prior to the start time and ask to be joined into the Amylyx Pharmaceuticals call. A live audio webcast of the call will be available under "Events and Presentations" in the Investor section of the Company’s website, https://investors.amylyx.com/events-presentations. The webcast will be archived and available for replay for 90 days following the event. Available Information Amylyx periodically provides other information for investors on the Company’s corporate website, https://amylyx.com, and the Company’s investor relations website, https://investors.amylyx.com. This includes press releases and other information about financial performance, information on corporate governance, and details related to our annual meeting of stockholders. Amylyx intends to use its website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor Amylyx’s website, in addition to following the Company’s press releases, SEC filings, and public conference calls and webcasts. About Avexitide Avexitide is an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist that has been evaluated in five Phase 1 and Phase 2 clinical trials for post-bariatric hypoglycemia (PBH) and has also been studied in congenital hyperinsulinism (HI). The U.S. Food and Drug Administration (FDA) has granted avexitide Breakthrough Therapy Designation for both indications, Rare Pediatric Disease Designation in congenital HI, and Orphan Drug Designation for the treatment of hyperinsulinemic hypoglycemia (which includes PBH and congenital HI). In PBH, an exaggerated GLP-1 response leads to excessive insulin secretion, resulting in recurrent hypoglycemic events. Avexitide is a GLP-1 receptor antagonist designed to competitively bind to the GLP-1 receptor on pancreatic islet beta cells and inhibit the exaggerated GLP-1-driven insulin response characteristic of PBH, reducing inappropriate insulin secretion and stabilizing blood glucose levels. In two Phase 2 PBH clinical trials, avexitide demonstrated highly statistically significant reductions in hypoglycemic events. About Post-Bariatric Hypoglycemia (PBH) PBH is a chronic metabolic condition that is estimated to affect approximately 8% of people in the U.S. who have undergone the two most common types of bariatric surgery, sleeve gastrectomy and Roux-en-Y gastric bypass (approximately 160,000 people in the U.S.). PBH is thought to be driven by an exaggerated glucagon-like peptide-1 (GLP-1) response, primarily in response to food intake, leading to persistent, recurrent, and often debilitating rapid drops in blood glucose, known as hypoglycemia. The American Diabetes Association (ADA) recognizes hypoglycemia as a potential medical emergency because low blood glucose levels can compromise the body’s ability to maintain essential physiologic processes. In addition, hypoglycemia in the context of PBH may manifest as neuroglycopenia – an inadequate supply of glucose to the brain – which can cause confusion, cognitive dysfunction, loss of consciousness, and seizures. PBH can be associated with substantial disability, compromising safety, disrupting independent living, and affecting nutritional status and overall quality of life. Despite the substantial burden, there are currently no FDA-approved therapies for PBH. About the LUCIDITY Trial LUCIDITY (NCT06747468) is a 78-participant, multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the efficacy and safety of avexitide in participants with PBH following RYGB surgery. The Phase 3 trial is being conducted at 21 sites in the U.S. Participants were randomized 3:2 to receive either 90 mg of avexitide subcutaneously once daily or placebo. The trial includes an up to six-week screening period, including a three-week run-in period, a 16-week double-blind treatment period, and an open-label extension (OLE) period with a duration of 32 weeks. The primary efficacy objective of LUCIDITY is to evaluate the FDA-agreed upon primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. Safety and tolerability will also be evaluated. About Amylyx Pharmaceuticals At Amylyx, our mission is to usher in a new era of treating diseases with high unmet needs. Where others see challenges, we see opportunities that we pursue with urgency, rigorous science, and unwavering commitment to the communities we serve. We are currently focused on four investigational therapies across several endocrine conditions and neurodegenerative diseases in which we believe can make the greatest impact. For more information, visit amylyx.com and follow us on LinkedIn and X. For investors, please visit investors.amylyx.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, Amylyx’s expectations regarding: the potential of avexitide as a treatment for PBH; the timing for the topline data readout and completion of the Phase 3 LUCIDITY clinical trial of avexitide; the timing for potential commercialization of avexitide; the expected enrollment of the Expanded Access Program for avexitide; the potential for AMX0114 as a treatment for ALS; the expected timeline and announcement for Cohort 1 biomarker data from the Phase 1 LUMINA clinical trial, and enrollment and progress of the LUMINA trial; the therapeutic potential of AMX0318 and the expected timeline for a potential IND submission; and the potential benefits of expedited program and orphan designations held by Amylyx; and financial performance, cash runway and longer-term strategy. Any forward-looking statements in this press release and related comments in the Company's earnings conference call are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx’s program development activities; Amylyx’s ability to execute on its regulatory development plans and expectations regarding the timing of results from its planned data announcements and initiation of clinical studies; Amylyx’s ability to fund operations, and the impact that global macroeconomic uncertainty, geopolitical instability, and public health events will have on Amylyx’s operations, as well as the risks and uncertainties set forth in Amylyx’s United States Securities and Exchange Commission (SEC) filings, including Amylyx’s Annual Report on Form 10-K for the year ended December 31, 2025, and subsequent filings with the SEC. All forward-looking statements contained in this press release and related comments in our earnings conference call speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. View source version on businesswire.com: https://www.businesswire.com/news/home/20260507409392/en/ Contacts Media Amylyx Media Team +1 (857) 320-6191 [email protected] Investors Lindsey Allen Amylyx Pharmaceuticals, Inc. +1 (857) 320-6244 [email protected]