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Image source: The Motley Fool. Tuesday, Aug. 4, 2026 at 8:00 a.m. ET Chairman, Chief Executive Officer and President - David Meeker Executive Vice President, Head of North America - Jennifer Chien Chief Financial Officer - Hunter Smith Executive Vice President, Head of International - Yann Mazabraud Investor Relations - Dave Connolly Operator: Good day, and thank you for standing by. Welcome to the Rhythm Pharmaceuticals Second Quarter 2026 Earnings Conference Call. [Operator Instructions] Please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Dave Connolly, Investor Relations at Rhythm Pharmaceuticals. Please go ahead. David Connolly: Thank you, Didi. I'm Dave Connolly here at Rhythm Pharmaceuticals. For those of you participating on the conference call, our slides can be accessed and controlled by going to the Investors section of our website, ir.rhythmtx.com. This morning, we issued our press release that provides our Q2 2026 financial results and a business update, and that press release is available on our website. We also released data for RM-718's Phase II trial in acquired hypothalamic obesity. Our agenda today is listed on Slide 2. On the call are David Meeker, our Chairman, Chief Executive Officer and President; Jennifer Lee, Executive Vice President, Head of North America; Hunter Smith, Chief Financial Officer; and Yann Mazabraud, Executive Vice President, Head of International is on the line joining us from Europe. On Slide 3, I'll remind you this call contains remarks concerning future expectations, plans and prospects, which constitute forward-looking statements. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in our most recent annual or quarterly reports on file with the SEC. In addition, any forward-looking statements represent our views as of today and should not be relied upon as representing our views as of any subsequent dates. We specifically disclaim any obligation to update such statements. With that, I'll turn the call over to David Meeker, who will begin on Slide 5. David Meeker: Good morning, and thank you for joining us. We have a lot to share today, headlined by a strong start to the U.S. launch of IMCIVREE for acquired hypothalamic obesity, reinforcing our conviction that HO represents a meaningful long-term opportunity for Rhythm. Progress during the quarter wasn't limited to our commercial business. In June, we presented positive 6-month data in Prader-Willi at ENDO showing setmelanotide achieved clinically meaningful BMI and BMI-Z score reductions, reductions in fat mass and preservation of lean mass and improvements in hyperphagia and anxiety measures. These data confirm the mechanistic rationale that MC4R agonism plays a key role in the pathology of PWS and demonstrated the positive impact IMCIVREE can have in this difficult-to-treat patient population. We look forward to updating you on the path forward for PWS in the next few months. Which brings us to our pipeline. We believe our next-generation MC4R agonists have the potential to bring meaningful new treatment options to patients living with rare neuroendocrine diseases. And today, we announced encouraging results to demonstrate RM-718, our weekly MC4R agonist has the potential to deliver clinically meaningful BMI reductions in patients with acquired hypothalamic obesity with efficacy comparable to what we've demonstrated with setmelanotide and bivamelagon and importantly, a favorable tolerability profile with no reports of generalized hyperpigmentation. But first, let me comment on the IMCIVREE launch in HO. This is a unique severe rare disease marked by accelerated and sustained weight gain caused by a brain tumor and/or its treatment or other brain injury to the hypothalamus that impairs the MC4R pathway. Acquired HO is clearly distinct from general obesity and remains underdiagnosed and underrecognized. With an estimated 10,000 patients in the U.S., a similar number in Europe and between 5,000 and 8,000 patients in Japan, this represents a significant global opportunity. In July, results from the Phase III HO TRANSCEND trial were published in the New England Journal of Medicine by lead author, Dr. Jennifer Miller; and senior author, Dr. Christian Roth, 2 of the world's leading experts in HO. In addition, as shown on Slide 6, the New England Journal of Medicine published an accompanying editorial with its science behind the study feature authored by Professor Sadaf Farooqi, one of the world's leading experts in the MC4R pathway diseases. Articles like this in the New England Journal of Medicine 10 years after the POMC deficiency New England Journal of Medicine article that put Rhythm on the map, raised visibility of a historically underrecognized disease among clinicians, researchers and payers and further establishes setmelanotide as a clinically meaningful advancement for patients. The awareness that there is an effective therapy for a rare disease increases the urgency on all parts of the health care system. Getting to a diagnosis matters when there's a precision medicine available. The New England Journal of Medicine article follows on the heels of the FDA approval on March 19 of this year. Throughout 2025 and 2026, our teams have been in the field focused on disease awareness and patient identification, engaging endocrinologists to deepen understanding of acquired HO and the connection between hypothalamic injury and the disruption of the MC4R pathway. Our HO launch builds on this engagement and a successful commercial effort in BBS, an opportunity that continues to grow. Rhythm now has a mature rare disease commercial platform built on 4 years of commercial success and established relationships across physicians, payers and patient communities. We're off to a promising start with this next phase of growth with strong demand from patients and families, a broad and growing prescriber base and a positive reception from payers, we are encouraged with the first 14 weeks of the U.S. launch. Since approval on March 19, we have received more than 400 start forms from approximately 300 prescribers. Jennifer will provide additional color on the U.S. launch. Yann will detail the next steps toward an anticipated approval and launch in Japan this year and country-level launches in Europe next year. We are mindful that it is early days. However, the first launch quarter performance reinforces our conviction that HO represents a long-term durable and sustainable opportunity for Rhythm. Now I want to briefly review preliminary data from the open-label Phase II Part C trial of RM-718, our weekly MC4R agonist in acquired HO. RM-718 is 1 of the 2 next-generation MC4R agonists that have the potential to improve on the hyperpigmentation and convenience of setmelanotide. The weight loss efficacy for each of these 3 assets has been remarkably consistent. We believe these preliminary results are encouraging and meaningfully derisk 718 as a development candidate going forward. Beginning on Slide 8, we show the patient disposition and patient demographics. Eleven patients were enrolled and 8 patients remain on active therapy. Seven patients have reached 16 weeks, and that is the data we are sharing here. Two patients discontinued because of AEs during the first 4 weeks of treatment. One patient stopped because of injection site reactions and a second patient discontinued secondary to ongoing nausea, which cannot be controlled even with dose reductions. A third patient completed the 16-week trial period and later withdrew long-term extension for personal reasons. Two additional patients have yet to reach the 16-week time point. Patients in the trial were 12 years of age or older with a mean BMI of 40.1. Per protocol, doses were escalated to 40 mgs as tolerated. The mean BMI change of 11.6% for the 7 patients who have reached week 16 is shown on Slide 9. As you can see on Slide 10, these results compare favorably with setmelanotide and bivamelagon results at a similar time point in patients with acquired HO. In a pooled analysis of patients with HO in the Phase II and Phase III setmelanotide trials, the week 16 BMI reduction was 10.1%. For bivamelagon at 600-milligram dose, mean BMI reduction for 7 patients at week 14 was 10.1% and Slide 11 shows a continued -- 10.1%, sorry. And Slide 11 shows a continued deepening of the effect in 4 patients with 28 weeks or more of treatment. As shown on Slide 12, RM-718 was generally well tolerated with the most common adverse events being injection site reactions and nausea. Importantly, we have observed no generalized hyperpigmentation with either bivamelagon or 718, which you would expect to see with MC1R agonism. Overall, these results validate our conviction that 718 has the potential to be a viable treatment option for rare MC4R pathway diseases. Both 718 and bivamelagon have been shown to affect BMI reductions comparable to setmelanotide in patients with acquired HO and both have greater specificity for the MC4 receptor without generalized hyperpigmentation. Importantly, the patent protection for both 718 and biva goes past 2040. We believe today's data further derisks our ability to build a durable franchise of MC4R agonists. Enrollment of PWS patients in Part D of this open-label trial will complete by the end of the year with a goal of enrolling 10 to 15 patients. As I mentioned, we are moving closer to a decision on a potential path forward for PWS with setmelanotide, bivamelagon or 718, all viable options. As a reminder, we are aiming to initiate the Phase III trial of bivamelagon in RH over the end of this year, which is listed among the upcoming milestones on Slide 13. The first patients enrolled will be patients 12 and older as we finalize CMC work for the oral dissolvable tablets in the first quarter of next year. With that, I'll turn the call over to Jennifer and then Yann to provide more color on our strong commercial progress during the quarter. Jennifer Chien: Thank you, David. I'll begin on Slide 15. I'm pleased to provide an update on the U.S. launch of IMCIVREE for acquired hypothalamic obesity. This was the first full quarter of launch plus 1 additional week following FDA approval on March 19. While we remain very early in the launch, we have seen strong demand, broad and expanding prescriber engagement and activation and positive early payer experience supporting access. Overall, the early indicators are encouraging and reinforce our conviction in the long-term opportunity for acquired hypothalamic obesity. On Slide 16, I'll start with the patient level metrics. In the 14 weeks between FDA approval on March 19 and June 30, we received more than 400 start forms for acquired hypothalamic obesity. This includes 66 start forms for converted trial patients accounting for almost all the U.S. trial patients. The age distribution for these patients is evenly split between patients 21 and younger and those 22 and older with 21% of prescriptions for patients ages 4 to 11, 18% for patients 12 to 17 and 12% for patients 18 to 21. Care for acquired HO patients tends to be more coordinated in the pediatric setting, and this contributes towards higher rates of recognition and diagnosis. As a result, pediatric patients make up a larger share of our early IMCIVREE patients. We expect this to evolve over time and to have a higher contribution for adults in the future. Among these early prescriptions, we see a broad mix of both incident and prevalent patients. Based off an internal analysis of a portion of prescriptions received, approximately 15% of patients suffer the injury resulting in their acquired HO within the last 2 years. The majority of start forms are from prevalent patients living with HO for more than 2 years, with approximately 50% of patients having their injury occur more than 10 years ago. These early dynamics are encouraging and demonstrate meaningful demand across a broad range of patients. On Slide 17, our provider level metrics for the acquired HO launch. We are seeing broad activation and engagement of health care providers. Between approval and the end of the second quarter, we have approximately 300 unique prescribers for acquired HO. Approximately 20% of these physicians have prescribed IMCIVREE for more than 1 patient with acquired HO. In line with our view that this is primarily a specialty opportunity, the majority of prescribers are endocrinologists with adult and pediatric endocrinologists accounting for 43% and 37% of prescribers, respectively. We are encouraged by the early activation levels of our priority accounts and top prescriber targets. We've made solid progress in penetrating our priority accounts with 65% activated by the end of June and almost 25% of prescriptions coming from these accounts. On to Slide 18 and our progress with payers. The early payer experience has been positive and supportive of access, a reflection that payers recognize the severity of acquired HO and similar to BBS, understand the distinction from general obesity and the benefit of IMCIVREE. While we still work to gain access for prescriptions received since approval, we are encouraged by the number of initial approvals that came in at the prior authorization stage during this first quarter of launch, many through payers without a specific acquired HO policy for IMCIVREE yet in place. By the end of Q2, we secured positive policies for HO covering approximately 25% of Medicaid covered lives and 35% of commercial covered lives. In line with our expectations, we expect additional policies to become established within the 3 to 9 months post approval or by the end of this year. The strength and consistency across these early launch metrics give us confidence this opportunity will continue to build over time, reinforcing our long-term conviction in the acquired HO opportunity and our belief that IMCIVREE can become the standard of care for these patients. Moving to Slide 19. While we are encouraged by their progress in the HO launch, we also feel there remains opportunity for growth in BBS. We recently supported the publication of a new evidence-based consensus-driven diagnostic algorithm designed to help physicians identify and diagnose patients with BBS earlier in their disease journey. In a population that has historically been very difficult to diagnose, we believe this is an important step towards enabling more BBS patients to get to a timely diagnosis and appropriate care. Lastly, on Slide 20, we are also evolving our North America commercial organization to ensure acquired HO and BBS each receive dedicated focus to maximize our ability to help patients. With the acquired HO launch now underway, we have made a transition to focus our 42 territory managers exclusively on HO. We modified our field team structure, so we now have a new territory manager group dedicated to BBS with plans to grow this to 10 TMs. This reflects our long-term commitment to patients living with BBS. These changes allow us to pursue both opportunities with dedicated teams, ensuring focused execution in acquired HO while continuing to build upon the strong foundation we have established in BBS. Now let me hand it over to Yann. Yann Mazabraud: Thank you, Jennifer. Our strong international commercial performance continued during the second quarter as the number of patients on IMCIVREE continues to grow, either through national reimbursements or named patient sales. And we have also made significant progress in Europe and Japan towards our goal of bringing IMCIVREE to patients with acquired hypothalamic obesity. Slide 22. We are entering the final stages in the regulatory process towards approval and launch in Japan. We anticipate IMCIVREE approval for acquired HO and launch in Japan by year-end 2026. Following the PMDA review, we anticipate marketing authorization from the Japan Ministry of Health, Labor and Welfare or MHLW. Once we receive marketing authorization, we would begin pricing discussion with Japan National Health Insurance Authority, and this process can take 2 to 3 months. With this time line, we would anticipate commercial launch in Japan in the fourth quarter. With approximately 5,000 to 8,000 patients living with acquired hypothalamic obesity in Japan, which is a higher per capita prevalence than the United States or Europe, Japan represents a meaningful opportunity. Our team in Japan is in place, actively engaging with the community of experts, patients and caregivers. Similar to the approach Jennifer's team took in the U.S., we are engaging with the treatment community to identify potential patients ahead of launch. We will share more details about Japan on our next quarterly conference call. Next slide, turning to commercialization of IMCIVREE for acquired HO in Europe. In May, the European Commission granted marketing authorization for IMCIVREE for the treatment of obesity and control of hunger in patients 4 years of age and older with acquired hypothalamic obesity due to hypothalamic injury or impairment. We have a very experienced team in place working through country-level processes to secure market access and reimbursement, which we have successfully done in the past years for [ OCVPAR ], BBS and the pediatric label expansion. In Germany, we anticipate launching in the first half of 2027. We are encouraged by the initial engagement in the process to secure an exemption from the German Federal Committee or G-BA Annex II exclusion list. The G-BA Annex II exclusion list prohibits reimbursement for lifestyle drugs such as drugs indicated for smoking cessation and general obesity. We are confident in our ability to secure an exemption, enabling a reimbursement for acquired HO as we have done it before for all our previous indications. We are making progress towards market access elsewhere in Europe. We have submitted the dossiers to begin pricing discussions in France, in the U.K., in Italy, in Spain and in the Netherlands with launches in each country anticipated in 2027 following Germany. With an estimated prevalence of approximately 10,000 patients in Europe, it is a meaningful opportunity. And our early access programs in France and in Italy have enabled many of the leading physicians to gain experience with setmelanotide and see the benefit in patients living with HO. In May, we presented approximately a dozen posters at the European Congress of Endocrinology and at the European Congress on Obesity. Next month, at the 2026 European Society for Pediatric Endocrinology Meeting, we have had 3 abstracts accepted for oral presentations and 3 accepted for poster presentations. And there are 2 additional non-Rhythm-sponsored presentations that will highlight findings in patients living with BBS from Germany and the U.K. We have approximately 75 abstracts, both originals and encores submitted and presented and are slated for presentation this year in European and Japanese medical congresses, giving us an unparalleled level of engagement with the international experts and health care providers. With that, I will turn it over to Hunter. Hunter Smith: Thank you, Yann. I will begin on Slide 25. We exited the second quarter in a solid financial position and are encouraged by our strong initial launch execution in acquired HO in the U.S. At the same time, we continue to make meaningful progress with BBS in the U.S. and internationally, underscoring the strength of our business and the opportunity we see for continued growth. We had a strong second quarter of 2026. Global net product sales of IMCIVREE totaled $71.3 million, which represents 19% sequential growth over Q1. During the second quarter, $51 million or 72% of product revenue was generated in the United States. Globally, we saw continued growth in patients on reimbursed therapy with an increase of greater than 20% over the prior quarter, primarily driven by the acquired HO launch and continued growth in BBS in the U.S. as well as ex-U.S. growth in BBS and our HO early access programs. On Slide 26, I'll walk you through the quarter-over-quarter revenue change with revenue increasing from $60.1 million in Q1 '26 to $71.3 million in Q2. We saw an $11.3 million in revenue growth attributable to increased product demand in the U.S. A bit more than half of this increase came from the strong start in the U.S. launch for acquired HO, where a percentage of our early scripts converted to starts at the prior auth stage, as Jennifer had mentioned previously. BBS had a strong quarter as well with higher demand growth and strong compliance compared to what we have experienced in other recent quarters. GTN improved somewhat in the quarter to 86%, providing an additional support to Q2 revenue. With the anticipated increase in demand for IMCIVREE following FDA approval for acquired HO, product shipments to IMCIVREE specialty -- I'm sorry, to Rhythm Specialty Pharmacy exceeded patient dispenses by approximately 2.9 million, providing a modest benefit to revenue in the quarter. Inventory on [ Hampton Specialty Pharmacy ] increased slightly to 20 days as of June 30. International revenue decreased from $23.2 million to $20.3 million in Q2. Even though we saw a steady increase in the number of patients on reimbursed therapy, the decrease in revenue was mainly attributable to a $3.8 million retrospective charge associated with the French contribution M mechanism, which levies a charge on pharmaceutical companies when reimbursed drug sales industry-wide exceeds specified statutory thresholds. $2.3 million of this $3.8 million charge was related to 2025 revenue. Excluding this impact, our international business remains strong as evidenced by the continued growth in patients on therapy during the quarter. On Slide 27 is a financial snapshot of the second quarter of '26 results compared to the second quarter of 2025. Cost of goods sold this quarter was 12.5% of product revenue, within our normal range, and primarily driven by cost of materials and royalty payments on setmelanotide in conjunction -- I'm sorry, in connection with higher net product revenue during the quarter. As a percentage of product revenue, COGS varies quarter-to-quarter based on changes in inventory balances and manufacturing activity, and this quarter increased slightly because of that. R&D expenses were $43.4 million for the second quarter of 2026 compared to $42.3 million in the same period last year. Sequentially, R&D expenses increased $1.7 million compared to the first quarter of 2026. This sequential quarter-over-quarter increase was due to increased spending on bivamelagon clinical trials and preclinical work associated with our congenital hyperinsulinism program. These were partially offset by lower headcount-related costs. The year-over-year increase is primarily attributable to an increase in headcount-related costs, increased costs related to genetic testing and preclinical work and the increase partially offset by reduced costs associated with RM-718 development and clinical supply. SG&A expenses were $67.4 million for the second quarter of 2026 compared to $45.9 million in the prior period. Sequentially, SG&A expenses increased by $3.8 million or approximately 6% compared to the first quarter of 2026. The increase was primarily driven by higher personnel-related costs to support our expanding commercial operations. Weighted average common shares outstanding were 68.6 million for Q2 2026. GAAP EPS for the second quarter of 2026 was a net loss per basic and diluted share of $0.73, including $0.02 per share from accrued dividends on convertible preferred stock of $1.1 million. Cash used in operations was approximately $9 million during the quarter. We ended the second quarter with approximately $331 million in cash, cash equivalents and short-term investments, which we continue to expect will be sufficient to fund planned operations for at least 24 months. Lastly for me, on Slide 28, there is further detail on our operating expenses for the second quarter and our full year operating expense guidance. For the second quarter, operating expenses of approximately $110.9 million included $26.1 million of stock-based compensation. Looking ahead to the second half of this year, we are updating our annual OpEx guidance. We now anticipate approximately $363 million to $397 million in non-GAAP operating expenses for 2026 comprised of non-GAAP R&D expenses of $175 million to $195 million and non-GAAP SG&A expenses of $188 million to $202 million. SG&A guidance remains unchanged, while the midpoint of our R&D expense guidance has been reduced by $20 million as the timing of certain CMC activities related to RM-718 and bivamelagon has shifted from late 2026 to early 2027. We do not expect these changes to affect overall time lines for ongoing or planned clinical development work with either asset. And with that, I'll turn the call back over to David. David Meeker: Thank you, Hunter, and we'll open it up now for Q&A. Operator: [Operator Instructions] And our first question comes from Tazeen Ahmad of Bank of America. Tazeen Ahmad: Congrats on a good quarter. It's early, but can I ask what type of expectations you have around discontinuation rates? Presumably, you're not seeing any, but if you are, can you just give us any color on why those might be happening on a go-forward basis? What do you think over time, discontinuation rate from setmelanotide could be for the HO indication? Jennifer Chien: Thanks, Tazeen. So it is really early in the launch to articulate color really in terms of the discount rate expected. I would say one thing, though, in the past, we have outlined that the global discount rate for the BBS patients was around 30%. And holistically, there are differences just in terms of what our data has shown in the BBS patient population versus the HO population in terms of the strength of efficacy that may make that discount rate lower in this particular population. There is also different factors of the HO population that we're experiencing in terms of the ease of injections and such that may also overall make the ongoing discount rate for this patient population a bit less than the BBS patient population. And that was also seen in the clinical studies, like when you compare the BBS clinical studies versus the HO clinical study and compare the discount rates there. So it's one thing that we're definitely going to be monitoring. There's a huge focus on as an internal organization, and we'll continue to monitor that moving forward. Operator: And our next question comes from Phil Nadeau of TD Cowen. Philip Nadeau: Congratulations on the strong progress. A question on the HO launch. It seems to be going really well. Based on our math, if you back out the clinical trial patients, you're doing about 110 patient start forms per month. We're curious to get your thoughts on whether you think that's sustainable for the next couple of quarters. Is there any sign of an early launch bolus? Or do you expect a more even trajectory to patient starts? Jennifer Chien: So overall, in terms of the start forms experienced in the first quarter plus the 1 week since approval, we're really happy in terms of what we're seeing with the Rxs coming in. As you mentioned, there's onetime event in terms of the clinical trial patients. And if you take that out, I would say that in any launch, there may be some patients who were really anxiously waiting in terms of getting that on to therapy upon approval. With that said, I think all of the launch metrics that we have been really monitor -- strengthen our belief holistically in terms of this long-term opportunity for sustained ongoing growth in the HO opportunity. As I outlined, the Rxs are really coming from a good breadth of prescribers versus being localized in terms of just a few writing the vast majority of these Rxs. We're also seeing a lot of different things around the patients and the background from an age perspective, disease severity, incident versus prevalent patients, which really point to the breadth in terms of patients within the HO indication that are really interested in getting on to this therapy. And even with the Rxs that we have received, there's still physicians who have additional patients under their care that they have not yet Rx. So overall, a lot of opportunity still remains. I would say that with that said, there are some considerations in terms of the speed of the uptake, which I've said in the past as well. These are really very, very busy endo offices. They have long wait times for their patients. That can also impact our ability to get in, in terms of having the ongoing discussion. But our teams are very persistent from that perspective, but that can take some time just in terms of being able to continue the dialogue with these HCPs. The other piece is that all these patients are not yet diagnosed. So it takes some time after we're in there to educate the physician for that patient to come in and be educated and further evaluated by the HCP before they can have that IMCIVREE discussion. And it's a new drug on the market. So some of the physicians, even with more than one patient, they want to experience IMCIVREE before prescribing to all of their patients at once. So once again, there's so much opportunity that remains. We feel very confident in terms of the ongoing trajectory, but there are some considerations as well. David Meeker: And Phil, as you know, we don't guide for the future here, but everything that Jennifer just outlined, I think, speaks strongly to the fact that although there may have been a little bit of pent-up urgency, there was no bolus in this thing. We feel really good about all these metrics and how they speak to the future, so. Operator: And our next question comes from Derek Archila of Wells Fargo. Derek Archila: Congrats on the updates and the progress here. I know you highlighted greater than 400 start forms against, I think, a 2,000 number for identified HO patients. So implying about 20% penetration into that pool in 14 weeks. So I guess, should we be thinking that number of identified patients is materially larger now? Jennifer Chien: Yes. Thanks, Derek. We provided that number back in September, and we have not updated that number. We have been ongoing in terms of since that time, educating additional physicians within our target list, and we still have a ways to go in terms of penetrating the full list. So that number in terms of both that suspected or diagnosed AHO patient population is continuing to grow. Operator: And our next question comes from Paul Matteis of Stifel. Paul Matteis: Congrats on the quarter. I was wondering if you could comment on what you're seeing as it relates to either concomitant use with GLP-1s or whether there's physicians or many physicians are kind of looking to try a GLP-1 before IMCIVREE. And then I know it's really early on the payer side, but are you seeing any payers put in a GLP-1 step edit? For accessing HO? Jennifer Chien: Sure. Let me start with the last one. We are pleased just in terms of the progress that are being made around the HO-specific policies. Of the ones that we have in place, there is no step edit requirement in terms of needing to try a GLP before IMCIVREE which is also something that we're pleased overall with. It's pretty much aligned with our label in terms of background patients. Relating to GLP use in the AHO patients, of the ones that have been prescribed IMCIVREE, we are experiencing that this patient population was one that was seeking treatment. So about 50% of our IMCIVREE-prescribed AHO patients had prior or current experience on GLP. But currently, there's about 25% of the patients that are currently still on a GLP. And when I outlined that, it's not known in terms of if it's for diabetes versus for the obesity indication. But about 25% of the patient population is on GLP. I would say that, that is also in a time where IMCIVREE wasn't available. There wasn't a specific treatment to treat their underlying condition. So as we move forward, it will be available for these patients. And that means through the metrics that I just outlined, 50% of these patients were never on a GLP, but still interested in getting on to IMCIVREE after understanding that there was a treatment available for their specific conditions, so. David Meeker: Yes. And Paul, just to add a little bit. As you remember from the clinical trial, we had also about 25% of the patients, 30 out of the 120 in the original cohort who either had a prior experience or were actively on GLPs in the trial. And they came in, met all the criteria for entry and then had a virtually identical response to IMCIVREE as the others. So I think, as Jennifer said, it's really, I think, quite instructive, the extent to which a high percent of these patients have been on GLPs or continue on GLPs, she said, speaking to the unmet need here, but they're not -- it's not addressing the unmet need here. They're coming over to IMCIVREE. So I think that overall experience has been really reinforcing that a precision medicine here is the right way to go for this patient population. Operator: And our next question comes from Dennis Ding of Jefferies. Yuchen Ding: I have a broader question for David. So there's been some investor questions around biotech M&A that came out of the AstraZeneca and Bristol merger speculation. So I'm curious, given Rhythm is obviously a leader in the rare disease space and David, you're on the board of several biotech companies. I'm curious, how would you characterize pharma M&A appetite recently? Obviously, without going to specific discussions, but, like, do you have any general comments on what pharma is excited about, but also at the same time, if they think premiums may be getting harder to justify given the broader move in the [ XCI ]? And then as a quick follow-up, the territory manager split 42 for HO and 10 for BBS, did that get implemented already? Or when will that happen? And do you actually expect an acceleration in HO adds in the second half? David Meeker: Yes. So thanks, Den. I think let me take that briefly on the biotech M&A, and this is completely unrelated to Rhythm. As you've heard here, we feel really good about where we are and Rhythm's ability to grow the company and platform and the product, expanding pipeline here. So we're watching like everybody else, I am. I think that was a bit of a surprise coming over the weekend. And we'll see how if anything happens there and how it might shake up. But I think the larger landscape here, pharma with many, many acquisitions over the first half of the year just speaks to the way our health care ecosystem works. We've got a strong and robust early biotech community that's creating value, and we have a pharmaceutical community and some midsized caps who are looking everywhere to expand their pipeline. So I expect it to continue. I don't see this having a big dampening effect if it were to go forward, but it's clearly a noteworthy event here. So with that, I'll turn it over to [indiscernible]. Jennifer Chien: Relating to the question around the TM split. So this was already implemented. So we already have split the teams into 2. I would say that, like, one of the guiding factors around this decision-making point was we really feel strongly that there's an opportunity in both BBS and HO. I would say the vast majority of the time spent of the 42 territory managers when it was combined under their care was already focused on HO. So we just wanted to make sure that we had the right amount of focus also in terms of unlocking the BBS opportunity and being able to get those patients to a diagnosis and treatment as well, so. Operator: And our next question comes from Mike Ulz of Morgan Stanley. Michael Ulz: Congratulations on the strong quarter, 718 data as well. Maybe just a question on 718 now that you have your early HO data there, and it seems to be trending in line with your other assets. Maybe you can talk about early thoughts on read-through to the PWS data and remind us when that data is coming. David Meeker: Yes. Thanks, Mike. So yes, we're really pleased with the 718 data. I think to remind everybody, all of you have asked me multiple times between these different assets, which one do we think was most likely to be successful. This was before we had the biva data, and was handicapped this as 718 probably had the higher chance of being successful. And we had the very good data on bivamelagon. So that was great. But I think this data validates the original hypothesis, which is 718 was built off of setmelanotide and lots of similarities there with a major difference being one, the weekly formulation, but second and importantly, the greater specificity for MC4R. So we really don't have the hyperpigmentation. And I'll just -- when you run these clinical trials and you ask them to report, there's been this -- was the case with bivamelagon. There was a few patients in both of these trials where localized "increased" pigmentation under intense scrutiny. We tried to get closer on the bivamelagon side. I don't have as quite as much detail on the 718. But to me, I don't hear anything in these reports that suggest to be honest, that this is necessarily drug-related or if it is, it just highlights the fact that in maybe around a nevus or something in melanocytic nevus you might get a little more darkening. But again, it's all been data is super supportive of the fact that these 2 drugs don't have anywhere near the same level of pigmentation change. Readthrough to PWS, yes, it's very early. Again, as we said, we'll update -- sorry, we'll complete enrollment by the end of the year. We'll look for an opportunity to update. We've been asked before, will we wait for the results of that -- those [indiscernible] data to make a final decision on which asset will go forward? The answer is no, meaning that, that decision-making is being driven much more around, to be honest, some of the CMC issues and timing of availability of these different formulations and drug supply, right? If we do everything with one asset, that will put a little more pressure on its supply than if we spread it around. So -- and there's a number of things we'll take into consideration over the next few months, which will get us in a position to make that decision. But we're not waiting in terms of our planning and protocols are being drafted, and we're working to be in a position once we have that information to make that decision, we'll be ready to go. Operator: And our next question comes from Seamus Fernandez of Guggenheim Securities. Seamus Fernandez: So just 2 on my end. Hoping you guys could provide us a little bit more color on developments in Japan and how we're likely to see a rollout of AHO emerge in Japan and the pace at which that could play through just a very different market and would be interesting to get a better understanding of how you see that market evolving as it comes forward. And then just a second question, David, I think there's still a little bit of confusion on this daily versus weekly discussion as it relates to PWS. Just trying to get a better understanding of where you're likely to come out on that? And if there are critical decisions that need to be made along those lines? Or do you feel like there's an opportunity that's robust enough in PWS to actually warrant advancing both bivamelagon and 718 to offer choice to that patient population? David Meeker: Yes. Let me just quickly on Japan, and then I'll let Yann make a comment. So we -- as Yann indicated, we'll update -- we'll do a deeper dive on the Japan opportunity on our next earnings call. He laid out the time line in terms of what the process we're going through in terms of regulatory review and then we get into pricing negotiations, all of which we expect to put us in a position to launch by the end of the year. But Yann, I don't know if there's any additional color you want to add to your comments? Yann Mazabraud: No. So maybe 2, 3 things. Thank you for the question. So first, as you heard, we plan to launch before the end of the year. Two, the prevalence is meaningful. Three, in terms of efficiency in Japan, we can leverage claims data or hospital data like in the U.S. We have a strong team in place for many months and the field medical affair teams in place for now 6 months. And we also have, which is very important in Japan, the support of the scientific committee, the support of leading experts. And as you may remember, we had a few sites for the HO Phase III in Japan, which did help a lot, both in terms of drug understanding, adoption, and also interaction with the regulators. And maybe last thing with the regulators and the payers, I've been myself in Japan a few weeks ago. I've met with some senior executive of the MHLW and what was really clear is that they value a lot the speed with which Rhythm has tackled the problem, which is an important one for them and the drug lag, drug loss issue, which is, as you know, a meaningful one for Japan. So we are very optimistic in terms of what we can achieve there. David Meeker: Great. And then with regard to the daily, weekly question, I realize this all got triggered, I think, by Dr. Miller's comments on our June conference call, where she was strongly in favor of a daily for PWS patients who like their routine. So obviously, we'll take that into consideration as we make final decisions. I'm not sure in the Prader-Willi community given how complicated this disease is that we'll get one single opinion, and we'll obviously get some opinions from some other experts as well. So yes, that will be considered. It's not the overwhelming factor that will cause us to pick one over the other. And then the last part of your question is, could we envision developing both in Prader-Willi? Absolutely. I think for both HO and Prader-Willi, very significant opportunities, maybe even a comparable magnitude depending on how you cut them. Yes, we want patients to have that choice. So you can very much imagine that we would get it done, but maybe not in parallel. So I leave it there. Operator: And our next question comes from Jon Wolleben of Citizens. Jonathan Wolleben: A couple for me. I might have missed this, Jen, but I think last update you said time to paid drug was about 60 to 90 days. And I was wondering if there's an update there. And then can you remind us how many priority accounts you guys have? And with 25% of prescriptions coming from those as of now, do you expect that to accelerate in the near term? Or will that take a little bit more time? David Meeker: Sorry, Jen. So the first question was just around the time to approval, 60 to 90 days. What's your sense about where it is now? Yann Mazabraud: Yes, we are expecting the feedback from the... David Meeker: Sorry, Yann, actually, apologies. That was for Jennifer. Yann Mazabraud: Okay. Sorry, 60, 90 days. I thought it was surprising... David Meeker: No, no, it's good. I was looking to be updated on the Japan schedule here, but no, that was good. Jennifer Chien: So from the time to approval standpoint, I would say that holistically, on average, we expect that time to approval to continue to get better. I would say that overall, when we take a look at BBS, where the payers had very little understanding of diseases impacting the MC4 pathway, a little understanding in terms of the differentiation from general obesity, there was a lot of education that we've had to do on the payer side to really educate them on this piece and on IMCIVREE. And that helped a lot even pre-approval as we move forward with the pre-approval discussions with the payers. And that is leading to approvals that are quicker than what we experienced, at least in those early approvals to date than what we experienced with BBS. With that said, we have a lot more pending that we're still working towards. And we're also working to make sure that we get HO-specific policies in place with the payers as we move forward into the year as well. So that was the question in terms of time approval. The priority accounts, so these are the accounts that, for the most part, are where patients with brain tumors go for holistic management of their tumors pre-surgery and management as well as post-surgery and management in terms of any type of hypothalamic dysfunction. So there's around 43 across the nation that our teams have identified and working towards. And it's where likely the incident, but also the near-term, like, prevalent type of patient population remains for a couple of years after the surgery or procedure to maintain appropriate care. From there, they go on to other sites outside of these centers for treatment. So overall, these are very centralized just in terms of patient populations. And the 25% is aligned with what we have seen in claims as to identified patients that may be HO because within these accounts, we have identified or estimated about 33% of the HO patients are within these centers. So we are going after both physicians within these centers as well as outside, and we're going to continue to make sure that the patients are diagnosed and put on to therapy moving forward. Operator: [Operator Instructions] Our next question comes from Whitney Ijem of Canaccord. Whitney Ijem: Congrats on the quarter. Sorry if I missed this, but you talked about 80% of prescribers being endocrinologists. What are the other 20%? And are those doctors that you're proactively targeting? Or are they just kind of coming to you because they have the patients? Jennifer Chien: So the backgrounds of the other 20% are primarily primary care as well as pediatricians. In terms of our target list, we provide the teams also physicians who may have a patient that have backgrounds in terms of potential obesity management holistically, which may include like primary care physicians that are ABOM-certified, for example. So the background in terms of the physicians are endocrinologists primarily, but also supplemented with these physicians that have interest in obesity medicine overall. Operator: And our next question comes from Samantha Semenkow of Citi. Samantha Semenkow: I just have a follow-up on one that was just asked about the about 75 prescripts that are outside of your priority targets. Can you speak to the type of physicians that are writing scripts and how we should think about the growth among that segment going forward? Jennifer Chien: So outside the priority accounts, the backgrounds are very similar just in terms of the focus in terms of endocrinologists as well as physicians with backgrounds in terms of obesity medicine that have -- we've been tagged as potentially having HO patients for follow-up for our teams. We go where the patients lead us to in terms of the targets that we have. So there's patients within the priority accounts, there's patients outside the priority accounts. There's the access issue. So when our teams have a list and they can have an end in terms of being able to engage with the physician, that is where they will go in terms of having the discussion. But we've outlined just in terms of the priority accounts being a key focus because it would enable patients to not leave without an appropriate diagnosis of HO and would prevent sort of the broader bleed into the community centers moving forward. So we have an ability to really change the paradigm just in terms of how these patient populations are treated, but we go where we can have a discussion with physicians who have the patient. David Meeker: And if you think about how our health care system works for any complex medical problem, you're perhaps more likely to be seen at an academic center of excellence. So you're there, you get your problem managed. And as Jennifer said, these are the chronic management of these patients. They have a lifelong need for pituitary hormone replacement and they will have a lifelong need for management of their hypothalamic obesity, then they go back to a local endocrinologist, obviously, closer to home, somebody they may know better. So [indiscernible], we'll try to reach all of those patients. But yes, there's a natural flow here. Operator: And our next question comes from Lisa Walter of RBC Capital Markets. Lisa Walter: Congrats on the quarter. Maybe just another one on RM-718. Given the early but positive results shared today, just curious what are the next steps? Could you perhaps run a basket study across HO and Bardet-Biedl, maybe some of the other indications to accelerate the path to approval and perhaps gain a label eventually similar to IMCIVREE? Any color here would be helpful. David Meeker: Yes. Thanks, Lisa. Yes, so one, the development path will be at this point, indication by indication. We've had feedback earlier on, and we'll perhaps go back at some point. I don't think we're there yet that the regulators, FDA specifically, was not ready to entertain something like an MC4R pathway label, which might argue you have the ability to treat anybody where you can document there's a pathway label. So given that, we'll be going indication by indication, one. Two, our plan will be for all of our currently approved indications, so the POMC leptin receptor biallelic, the BBS population, we will get 1 of our 2 next-generation molecules into those populations and get that and get the indication. We may not develop both molecules in all of those indications. They're smaller. But as I said in my earlier response, for the large indications, HO and Prader-Willi specifically, we very much may entertain developing both of the molecules. Operator: Our next question comes from Priyanka Grover of JPMorgan. Priyanka Grover: Congrats on the quarter. So we just have a question about Europe. Aside from the onetime revenue recognition charge in France, were there any other factors to think about for Europe? In the past quarters, Europe has been a strong growth driver for Rhythm. So curious how you're thinking about Europe going forward, especially with acquired HO launch in 2027. David Meeker: Yann? Yann Mazabraud: Yes. Thank you for the question. So maybe a few words about the HO launch in 2027. First, we are encouraged by what we have seen in France and Italy in terms of diagnosis and willingness to treat. As you know, we have named patient sales in place for more than 1 year in those 2 countries. So we have observed the conviction, the growing conviction among the treating community. Another very positive aspect is that we will leverage most, if not all, the same centers of excellence and prescribers that we already have for PPL and BBS, but we also have new medical specialties to explore, as Jennifer also mentioned, in the U.S. So we feel good. We also know very well the payers because we have spoken with them for many years, and this is the same teams across Europe, and they know the drug very well, and they know the drug benefits very well. So with all that and other aspects, we feel good. Operator: And our next question comes from Thomas Smith of Leerink Partners. Thomas Smith: Let me add my congrats on the strong quarter and the nice 718 data here. Just on 718, wondering if you could expand a little bit on the comments regarding the dose escalation in the study. How many of these initial HO patients are getting up to the target dose? And then just on the tolerability profile, could you provide a little bit more color on the injection site reactions and how those compare to the IMCIVREE experience? David Meeker: Sure. So the vast majority got to the 40 milligrams. As I indicated, we had one patient who actually didn't get much above 10 or 20 mgs. They might have reached 20 mgs weekly. But were having nausea. And we saw this in our earlier development program with setmelanotide, where there was a small percentage of patients who just were extraordinarily sensitive in terms of their GI side effects. And I think that's part of the background of this disease. So -- but aside from that, everybody basically got to the 40 mgs. The injection site reactions, it's reactions/the way the drug works. This is a weekly formulation, the formulation itself is somewhat viscous and the mechanism essentially you inject and you get a nodule in the subcutaneous tissue under the skin and then the drug disperses from there. So that's how it's delivered. The nodule is present. And I think in the case of the patient who wasn't thrilled with the injections, part of it was didn't like the nodules per se. And the other thing is we also -- we're delivering this drug without the auto-injector. And so it has to be pushed in by hand. And the auto-injector, we know for a fact will make a significant difference, I think, in terms of the ease and also probably the patient experience. So -- but that's what -- for the most part, is being referenced when they reported the injection site reactions. It's the formation of this nodule, which is just part of how the drug is delivered. Operator: And our next question comes from Joseph Stringer of Needham & Company. Joseph Stringer: A question on the early-stage pipeline. You have a CHI program in preclinical development. Can you provide any updates there? And more broadly, any updated thoughts on pipeline expansion beyond the MC4R assets? David Meeker: Yes. Thanks. So CHI, we've been working on for a while here, as you know, and we look forward to updating you. As I've said before, and I'll say it again, we're actually making good progress there. We're -- we look forward to an update. We're not prepared to give one today, but we are making good progress. And as Hunter highlighted, we're beginning to increase our spend there. So we're moving deeper into the developmental program. So we'll talk more about that. We're excited about that program. I think we have a unique approach to addressing that disease, but more to come there. I think in terms of other approaches here, we'll continue to think about the biology around the MC4R pathway signaling through the receptor and the like. Like every company, we entertain other approaches to these diseases, and we're doing some of that. We're not at a point now where we're in a position to talk about that, but we'll look at that. And then externally, we got this question, would you consider going outside? And I think our answer continues to be, we feel really good about the opportunities in front of us, the pipeline and the product strategy we've been pursuing, and there's many indications, including our whole genetic pillar, if you will, which we will absolutely come back to and work our way through there. And as we look at some things, biomarkers, for example, we're continuing to try to understand how we can better understand and refine the patient population most likely to respond in terms of that genetic population. So that's going to be our focus. That said, we'll remain opportunistic. We're obviously in a stronger position in terms of our balance sheet and our position as a company. So we'd be not adverse to doing something that made sense, but we're not specifically looking to do something, so. Operator: And our last question comes from Ram Selvaraju of H.C. Wainwright. Raghuram Selvaraju: Just with respect to Prader-Willi syndrome, given the extended history so far with at least one approved drug in that condition and greater familiarity overall with that target indication, can you share with us any additional information at this time regarding market segmentation analysis and what you anticipate to be the key patient population within the Prader-Willi syndrome community that you might focus on for your products? David Meeker: Yes. Thanks. I think -- so one, as we've indicated on our prior calls, our initial developmental indication as other companies in the space have done following the Soleno approval would be for hyperphagia, makes total sense. There's a very clear, I think, template for how that trial can, should be done. Our mechanism, as we said, we started with a very clear focus on both hunger/hyperphagia and BMI weight change because our drug signaling through this receptor gives you a satiety signal, increases energy expenditure. And we always believe that we've got a weight change, BMI decrease, we would, of course, get -- achieve that end by, in some way, decreasing caloric intake, decreasing the hunger/hyperphagia. So long story short is we'll continue to pursue that paradigm. I think in terms of market segmentation, no, I think it's way too early. I think this is an extremely complex disease. You all know it as well as we do or maybe better some of you. It's remarkably heterogeneous, but they share a lot of common features. Currently, approved therapies have some limitations. We know that. I don't think there's likely to be one drug solution to Prader-Willi. So obviously, we've included patients who are on VYKAT in our current trial that Dr. Miller is running. So combination therapy is another strategy that I'm sure the community looking at and we'll be thinking about as we get -- go through our developmental plans and the like. But no, I think our goal is to serve the Prader-Willi population writ large, recognizing that the clinical trials might need to be more targeted to run that successful trial. Operator: This concludes our question-and-answer session. I'd like to turn it back to David Meeker for closing remarks. David Meeker: Great. Well, thanks, everybody, for tuning in August. As you've heard, incredibly excited, I'll say, about our start here. I mean, we've been working on HO for a number of years and now to be at a point where we're actually getting the drug to patients, and we're seeing a community that's embracing it and responding well to their initial experience is, like I said, it's very exciting for us. Lots more to come. And hopefully, we've outlined some of that. We'll have more discussions going forward, but we look forward to our next update at the Q3 call. Thanks all. Operator: This concludes today's conference call. Thank you for participating, and you may now disconnect. 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Our analysts just identified a stock with the potential to be the next Nvidia. Tell us how you invest and we'll show you why it's our #1 pick. Tap here. The U.S. launch of IMCIVREE for acquired hypothalamic obesity (HO) is off to a promising start with strong demand from patients and a growing prescriber base., driven by high unmet need and established rare disease commercial infrastructure. Management attributes early success to a broad prescriber base of approximately 300 unique providers, indicating the disease is being recognized beyond a few specialized centers. Strategic focus on 'priority accounts'—academic centers managing brain tumors—captured 25% of early prescriptions, validating the coordinated care model for incident patients. The international business remains robust despite a one-time retrospective charge in France, with reimbursed patient growth exceeding 20% sequentially. Positive Phase II data for RM-718 in HO demonstrates efficacy comparable to setmelanotide while eliminating generalized hyperpigmentation, a key strategic differentiator for the next-generation portfolio. The company restructured its field force to provide dedicated focus, with 42 territory managers now exclusive to HO and a new 10-person team dedicated to Bardet-Biedl Syndrome (BBS). Management anticipates a commercial launch in Japan by the fourth quarter of 2026, following expected marketing authorization and a 2-3 month pricing negotiation period. European expansion for HO is slated for 2027, beginning with Germany, where management is confident in securing a 'lifestyle drug' reimbursement exemption based on prior precedents. A pivotal decision on the development path for Prader-Willi Syndrome (PWS) is expected in the coming months, weighing daily versus weekly dosing based on patient preference and manufacturing capacity. Full-year R&D guidance was reduced by $20 million due to the strategic shifting of certain CMC activities for RM-718 and bivamelagon into early 2027 without impacting clinical timelines. The company expects additional U.S. payer policies for HO to be established by the end of 2026, typically occurring within 3 to 9 months post-approval. A $3.8 million retrospective charge associated with the French contribution mechanism impacted international revenue, reflecting industry-wide sales exceeding statutory thresholds. Inventory at the specialty pharmacy increased slightly to 20 days to accommodate the anticipated demand surge from the HO launch. Management noted that while early demand is strong, the speed of uptake is naturally constrained by long wait times at endocrinology offices and the need for physician experience with a new therapy. Two patients discontinued the RM-718 trial due to adverse events, including injection site reactions and nausea, though the asset was generally well-tolerated. One stock. Nvidia-level potential. 30M+ investors trust Moby to find it first. Get the pick. Tap here. Approximately 50% of early IMCIVREE patients had prior or current GLP-1 experience, yet still sought IMCIVREE, suggesting GLP-1s do not adequately address the underlying MC4R pathway injury. Management confirmed that current payer policies for HO do not include step-edit requirements for GLP-1s, treating IMCIVREE as a precision medicine. Management believes the 400 start forms represent sustainable demand rather than a one-time bolus, citing the mix of incident and long-term prevalent patients. They emphasized that many physicians are starting with one patient to gain experience before prescribing to their broader patient pool, suggesting a 'steady build' ahead. While some experts suggest PWS patients prefer the routine of a daily injection, management will not let this be the sole deciding factor for the PWS asset choice. The final decision between setmelanotide, bivamelagon, or RM-718 will also depend on manufacturing timelines and the strategic goal of offering patient choice over the long term.

-- U.S. launch of IMCIVREE® (setmelanotide) for acquired hypothalamic obesity (HO) starts strong with more than 400 patient start forms since FDA approval as of June 30, 2026 ---- Second quarter 2026 net product revenue from global sales of IMCIVREE of $71.3 million ---- Weekly injectable MC4R agonist RM-718 achieved mean BMI reduction of 11.6% in patients with acquired HO (n=7) after 16 weeks of treatment -- -- Phase 3 TRANSCEND Trial Results in Acquired HO published in the New England Journal of Medicine -- -- Management to host conference call today at 8:00 a.m. ET -- BOSTON, Aug. 04, 2026 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today reported financial results and provided a business update for the second quarter ended June 30, 2026. “Rhythm continues to demonstrate strong commercial and clinical development progress, highlighted by a strong start in the U.S. launch of IMCIVREE for acquired hypothalamic obesity (HO),” said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm Pharmaceuticals. “The demand we are seeing from both patients and physicians reinforces the significant unmet need in acquired HO and the opportunity for IMCIVREE to transform the treatment paradigm for this devastating disease.” Dr. Meeker added, “During the quarter, we strengthened Rhythm’s foundation for long-term growth with positive Phase 2 results for setmelanotide in Prader-Willi syndrome (PWS), as well as positive RM-718 results in acquired HO patients. With strong commercial execution in the United States and meaningful launches in HO upcoming in Japan and Europe, we remain focused on expanding treatment options for patients and realizing the potential of our pipeline of MC4R agonists.” Recent Business and Development Highlights Today, the Company announced that more than 400 patient start forms had been received for IMCIVREE for acquired HO from approximately 300 prescribers as of June 30, 2026, since the approval by the U.S. Food and Drug Administration (FDA) on March 19, 2026; Revenue from global sales of IMCIVREE was $71.3 million for the second quarter of 2026, an increase of 19% on a sequential basis from the first quarter of 2026. The number of patients on reimbursed therapy globally increased by greater than 20% as compared to the first quarter of 2026. Today, the Company announced preliminary data from Part C of the Phase 1/2 study of RM-718 in acquired HO patients. Today, the Company announced that six abstracts, including three oral presentations and three poster presentations have been accepted for presentation at the European Society for Pediatric Endocrinology’s Annual Meeting (ESPE 2026) September 8-10, 2026. The presentations include: On July 8, 2026, results from Rhythm’s pivotal Phase 3 TRANSCEND trial evaluating setmelanotide in patients with acquired hypothalamic obesity were published in The New England Journal of Medicine by Dr. Jennifer Miller, et al.; The publication was accompanied by a 'Science Behind the Study' editorial authored by Professor Sadaf Farooqi, M.B., Ch.B., Ph.D., titled Treating Acquired Hypothalamic Obesity; On June 13, 2026, at Endocrine Society's Annual Meeting (ENDO 2026), the Company presented positive six-month data from its Phase 2 trial of setmelanotide that demonstrated patients with Prader-Willi Syndrome (N=17) achieved clinically meaningful BMI or BMI z-score reductions, reductions in fat mass with preservation of lean mass, and improvements in hyperphagia and anxiety measures; Also during ENDO 2026, held June 13–16, 2026, the Company presented new data in multiple presentations, highlights included durable BMI reduction for patients with acquired HO after 2.5 years of setmelanotide; durable weight reduction after 1 year of bivamelagon treatment in patients in acquired HO; post-hoc analysis from the TRANSCEND trial in patients with acquired HO with a history of bariatric surgery demonstrated BMI reductions and weight category improvement after 1 year of setmelanotide treatment; and positive real-world efficacy analyses of U.S. patients with BBS; On May 28, 2026, the Company announced the publication of a new evidence-based, consensus-driven diagnostic algorithm for (BBS) in the peer-reviewed American Journal of Medical Genetics; On May 12-15, 2026, at the European Congress on Obesity (ECO 2026), the Company presented data on weight outcomes at five years of setmelanotide treatment in patients with BBS and obesity, weight outcomes at six years in patients with POMC or LEPR deficiency obesity, and a 52-week indirect comparison of patients aged 2-5 years with BBS treated with setmelanotide versus patients with BBS from a natural history cohort in the International Clinical Registry Investigating Bardet-Biedl Syndrome. On May 12, 2026, the Company announced six data presentations at The European Congress of Endocrinology (ECE); highlights included real-world efficacy results in patients with acquired HO enrolled in France’s early-access program; study results on hyperphagia severity in patients with BBS; post-hoc TRANSCEND trial results of patients with acquired HO treated with setmelanotide that demonstrated significant improvements in multiple metabolic index scores; a study that highlighted the persistent and under-recognized health burden of acquired HO across life stages in patients with childhood-onset craniopharyngioma from the Netherlands, Germany, and the UK. Anticipated Upcoming Milestones Rhythm expects to achieve the following near-term milestones: Complete enrollment in the setmelanotide substudy in congenital HO in the second half of 2026; Complete enrollment in the Phase 1/2, Part D trial evaluating RM-718 in PWS in the second half of 2026; Anticipate launch of IMCIVREE for acquired HO in Japan pending a decision by Japan's Ministry of Health, Labour and Welfare (MHLW) by year-end 2026; Initiate a pivotal Phase 3 trial evaluating bivamelagon in acquired HO by year-end 2026; Anticipate country-level launches of IMCIVREE for acquired HO in Europe beginning in 2027. Second Quarter 2026 Financial Results Cash Position: As of June 30, 2026, cash, cash equivalents and short-term investments were approximately $330.9 million, as compared to $388.9 million as of December 31, 2025. Revenue: Net product revenues from global sales of IMCIVREE were $71.3 million for the second quarter of 2026, as compared to $48.5 million for the second quarter of 2025. R&D Expenses: R&D expenses were $43.4 million in the second quarter of 2026, as compared to $42.3 million in the second quarter of 2025. The year-over-year increase was primarily due to personnel costs, data analytics related to the Company's genetic testing programs, pre-clinical expense and bivamelagon trials which were offset partially by a decrease in chemistry, manufacturing, and controls (CMC) activity and clinical trial costs due to the winding down of the EMANATE and TRANSCEND phase 3 trials. SG&A Expenses: SG&A expenses were $67.4 million for the second quarter of 2026, as compared to $45.9 million for the second quarter of 2025. The year-over-year increase was primarily due to increased personnel costs, including stock-based compensation, related to expanded business operations. Other income (expense), net: Other expense, net was $(0.3) million for the second quarter of 2026, as compared to other expense, net of $(1.0) million for the second quarter of 2025. The year-over-year decrease was primarily due to a decrease in non-cash interest expense associated with the LG Chem liability. Net Loss attributable to common stockholders: Net loss attributable to common stockholders was $(50.4) million for the second quarter of 2026, or a net loss per basic and diluted share of $(0.73), as compared to a net loss attributable to common stockholders of $(48.0) million for the second quarter of 2025, or a net loss per basic and diluted share of $(0.75). Year to Date 2026 Financial Results Revenue: Net product revenues relating to sales of IMCIVREE were $131.4 million for the six months ended June 30, 2026, as compared to $86.2 million for the six months ended June 30, 2025. R&D Expenses: R&D expenses were $85.2 million for the six months ended June 30, 2026, as compared to $79.3 million for the six months ended June 30, 2025. The increase was primarily driven by higher personnel-related costs to support the growth of the Company's research and development programs, partially offset by lower CMC and clinical trial expenses. SG&A Expenses: SG&A expenses were $131.0 million for the six months ended June 30, 2026, as compared to $85.0 million for the six months ended June 30, 2025. The increase was primarily driven by increased personnel-related costs related to expanded business operations and marketing costs. Other income (expense), net: Other income (expense), net was $(3.0) million for the six months ended June 30, 2026, as compared to $(3.4) million for the six months ended June 30, 2025. The decrease was primarily due to a decrease in non-cash interest expense associated with the LG Chem liability and an increase in the fair value of the embedded derivative for our deferred royalty obligation. Net Loss attributable to common stockholders: Net loss attributable to common stockholders was $(107.1) million for the six months ended June 30, 2026, or a net loss attributable to common stockholders per basic and diluted share of $(1.57), as compared to a net loss attributable to common stockholders of $(98.8) million for the six months ended June 30, 2025, or a net loss per basic and diluted share of $(1.56). Financial Guidance: For the year ending December 31, 2026, Rhythm anticipates approximately $363 million to $397 million in Non-GAAP Operating Expenses. Non-GAAP Operating Expenses are derived from: GAAP total operating expenses, inclusive of: Non-GAAP Operating Expenses is defined as GAAP operating expenses excluding stock-based compensation and fixed consideration related to in-licensing (see below under "Non-GAAP Financial Measures" for more details). Based on its current operating plans, Rhythm expects that its cash, cash equivalents and short-term investments as of June 30, 2026 will be sufficient to fund the Company’s planned operations for at least 24 months. Conference Call InformationRhythm Pharmaceuticals will host a live conference call and webcast at 8:00 a.m. ET today to review its second quarter 2026 financial results and recent business activities. Participants may register for the conference call here. It is recommended that participants join the call ten minutes prior to the scheduled start. A webcast of the call will also be available under "Events and Presentations" in the Investor Relations section of the Rhythm Pharmaceuticals website at https://ir.rhythmtx.com/. The archived webcast will be available on Rhythm Pharmaceuticals’ website approximately two hours after the conference call and will be available for 30 days following the call. About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 4 years and older with acquired hypothalamic obesity, adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. The European Commission (EC) has authorized setmelanotide for the treatment of obesity and control of hunger in patients 4 years of age and above with acquired hypothalamic obesity. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA. Setmelanotide IndicationIn the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 4 years and older with acquired HO, and in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign Other types of obesity not related to BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity Contraindication Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression, suicidal ideation and depressed mood have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation, darkening of pre-existing nevi, development of new melanocytic nevi and increase in size of existing melanocytic nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including “gasping syndrome” can occur in neonates and low birth weight infants treated with benzyl alcohol preserved drugs. ADVERSE REACTIONS Most common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the safety, efficacy, potential benefits of, and clinical design or progress of any of our products or product candidates at any dosage or in any indication, including, setmelanotide, bivamelagon, and RM-718; the use of setmelanotide in patients with acquired HO and the success of our commercial launch; our expectations surrounding potential regulatory submissions, progress, or approvals and timing thereof for any of our product candidates, including potential marketing approval and launch in Japan and launches in the European Union and the timing thereof; the commercial growth of IMCIVREE; the estimated market size and addressable population for our drug products, including setmelanotide for the treatment of acquired HO; the future announcement of data from our ongoing clinical trials, including the substudy evaluating setmelanotide for patients with congenital hypothalamic obesity, Part C and Part D of the Phase 1 trial evaluating RM-718, and the open-label Phase 2 trial evaluating setmelanotide in patients with PWS, and ongoing enrollment in our clinical trials; existing or future collaboration agreements; the Company’s business strategy and plans; our anticipated financial performance and financial position for any period of time, including our estimated Non-GAAP Operating Expenses for the year ending December 31, 2026; and the sufficiency of our cash, cash equivalents and short-term investments to fund our operations for at least 24 months; and the timing of any of the foregoing. Statements using words such as “expect”, “anticipate”, “believe”, “may”, “will” and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, risks associated with the laws and regulations governing our international operations and the costs of any related compliance programs, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic conditions, and the other important factors, including those discussed under the caption “Risk Factors” in Rhythm’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2026 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this press release or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise. Non-GAAP Financial MeasuresThis press release includes Non-GAAP Operating Expenses, a supplemental measure of our performance that is not required by, or presented in accordance with, U.S. GAAP and should not be considered as an alternative to operating expenses or any other performance measure derived in accordance with GAAP. We define Non-GAAP Operating Expenses as GAAP operating expenses excluding stock-based compensation and fixed consideration related to in-licensing. We caution investors that amounts presented in accordance with our definition of Non-GAAP Operating Expenses may not be comparable to similar measures disclosed by our competitors because not all companies and analysts calculate this non-GAAP financial measure in the same manner. We present this non-GAAP financial measure because we consider it to be an important supplemental measure of our performance and believe it is frequently used by securities analysts, investors, and other interested parties in the evaluation of companies in our industry. Management believes that investors’ understanding of our performance is enhanced by including this non-GAAP financial measure as a reasonable basis for comparing our ongoing results of operations. Management uses this non-GAAP financial measure for planning purposes, including the preparation of our internal annual operating budget and financial projections; to evaluate the performance and effectiveness of our operational strategies; and to evaluate our capacity to expand our business. This non-GAAP financial measure has limitations as an analytical tool, and should not be considered in isolation, or as an alternative to, or a substitute for operating expenses or other financial statement data presented in accordance with GAAP in our consolidated financial statements. Rhythm has not provided a quantitative reconciliation of forecasted Non-GAAP Operating Expenses to forecasted GAAP operating expenses because the Company is unable, without making unreasonable efforts, to calculate the reconciling item, stock-based compensation expenses, with confidence. This item, which could materially affect the computation of forward-looking GAAP operating expenses, is inherently uncertain and depends on various factors, some of which are outside of Rhythm's control. Corporate Contact:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, [email protected] Media Contact:Layne CosgroveReal [email protected]

Interested in Rhythm Pharmaceuticals, Inc.? Here are five stocks we like better. IMCIVREE sales rose 19% sequentially to $71.3 million in Q2 2026, supported by early demand following its U.S. launch for acquired hypothalamic obesity. Rhythm received more than 400 start forms from roughly 300 prescribers during the first 14 weeks. Preliminary RM-718 Phase II data showed a mean 11.6% BMI reduction at 16 weeks among seven evaluable acquired-HO patients, although injection-site reactions and nausea led to two early discontinuations. Rhythm ended June with approximately $331 million in cash and short-term investments, which it expects to fund operations for at least 24 months. The company is planning a bivamelagon Phase III trial, additional RM-718 development in Prader-Willi syndrome, and international IMCIVREE launches beginning in Japan by year-end 2026. 7 Stocks That Will Drive the Weight Loss Drugs Market Rhythm Pharmaceuticals (NASDAQ:RYTM) reported second-quarter 2026 global net product sales of $71.3 million for IMCIVREE, up 19% sequentially from $60.1 million in the first quarter, as the company cited an early positive reception to its U.S. launch in acquired hypothalamic obesity, or HO. Chief Executive Officer David Meeker said the launch of IMCIVREE for acquired HO, approved by the FDA on March 19, represented the company’s next phase of growth. He also highlighted preliminary Phase II data for RM-718, a weekly MC4R agonist, as well as ongoing preparations for further international launches and potential development in Prader-Willi syndrome. → SpaceX’s First Earnings Report Could Decide Whether Shorts or Bulls Have Control 2 biotechs with promising weight-loss drugs coming During the first 14 weeks following approval through June 30, Rhythm received more than 400 IMCIVREE start forms for acquired HO from approximately 300 unique prescribers. The total included 66 forms for converted clinical-trial participants, representing nearly all U.S. trial patients, according to Jennifer Lee, executive vice president and head of North America. Lee said early prescriptions reflected a mix of incident and prevalent patients. Based on the company’s internal review of a portion of the prescriptions, about 15% of patients had sustained the injury associated with acquired HO within the past two years, while approximately half had experienced the injury more than 10 years ago. → Financials Hit Record Highs as the AI Trade Unravels—Can They Keep Leading? Prescriber engagement was led by endocrinologists. Adult endocrinologists accounted for 43% of prescribers and pediatric endocrinologists represented 37%. About 20% of physicians had prescribed IMCIVREE for more than one acquired HO patient. The company said 65% of its priority accounts had been activated by the end of June, and those accounts generated nearly 25% of prescriptions. Rhythm has identified approximately 43 priority accounts, which Lee described as centers where patients with brain tumors receive care before and after surgery and management for hypothalamic dysfunction. → Why Rare Earth Processing Could Be the Real 2027 Opportunity On access, Rhythm said it had secured positive acquired HO coverage policies representing approximately 25% of Medicaid-covered lives and 35% of commercial covered lives by quarter-end. Lee said the company expects more policies to be established within three to nine months following approval. Rhythm also noted that approximately half of prescribed acquired HO patients had prior or current experience with GLP-1 therapies, while about 25% remained on a GLP-1. The company said none of the acquired HO-specific payer policies in place required patients to try a GLP-1 before IMCIVREE. U.S. product revenue totaled $51 million, or 72% of second-quarter product sales. Chief Financial Officer Hunter Smith said U.S. revenue growth was driven by increased demand in both acquired HO and Bardet-Biedl syndrome, or BBS. More than half of the $11.3 million sequential U.S. revenue increase came from the acquired HO launch. International revenue declined to $20.3 million from $23.2 million in the first quarter. Smith attributed the decline primarily to a $3.8 million retrospective charge associated with France’s Contribution M mechanism, including $2.3 million related to 2025 revenue. Excluding that charge, he said the international business continued to add patients on reimbursed therapy. Research and development expense was $43.4 million, compared with $42.3 million a year earlier. Selling, general and administrative expense was $67.4 million, compared with $45.9 million in the prior-year period. GAAP net loss was $0.73 per basic and diluted share, including $0.02 per share from accrued dividends on convertible preferred stock. Cash used in operations was approximately $9 million. Cash equivalents and short-term investments totaled approximately $331 million at June 30. The company said its cash resources are expected to fund planned operations for at least 24 months. Rhythm lowered the midpoint of its 2026 non-GAAP R&D expense outlook by $20 million because certain chemistry, manufacturing and controls activities for RM-718 and bivamelagon shifted from late 2026 to early 2027. It now expects total non-GAAP operating expenses of $363 million to $397 million for the year, including R&D expense of $175 million to $195 million and SG&A expense of $188 million to $202 million. Rhythm released preliminary results from Part C of an open-label Phase II trial of RM-718 in acquired HO. Of 11 enrolled patients, seven had reached the 16-week time point and showed a mean BMI reduction of 11.6%. The company compared that result with a 10.1% BMI reduction at week 16 in a pooled setmelanotide analysis and a 10.1% reduction at week 14 for bivamelagon at a 600-milligram dose. Eight patients remained on active RM-718 therapy. Two patients discontinued during the first four weeks because of adverse events: one due to injection-site reactions and one due to nausea that continued despite dose reductions. A third patient completed the 16-week trial period but later withdrew from the long-term extension for personal reasons. Meeker said RM-718 was generally well tolerated, with injection-site reactions and nausea as the most common adverse events. The company reported no generalized hyperpigmentation with either RM-718 or bivamelagon, which it said supports their greater specificity for the MC4R receptor relative to setmelanotide. Rhythm plans to complete enrollment of 10 to 15 Prader-Willi syndrome patients in Part D of the open-label RM-718 study by the end of 2026. The company is also targeting initiation of a Phase III trial of bivamelagon in acquired HO by year-end. Initial enrollment will include patients age 12 and older while the company finalizes manufacturing work for orally dissolvable tablets during the first quarter of 2027. In Japan, Rhythm expects marketing authorization for IMCIVREE in acquired HO and a commercial launch by the end of 2026. Yann Mazabraud, executive vice president and head of international, said the company expects to begin pricing discussions with Japan’s National Health Insurance Authority after receiving authorization from the Ministry of Health, Labour and Welfare. Rhythm estimates there are approximately 5,000 to 8,000 people with acquired HO in Japan. In Europe, the European Commission granted marketing authorization for IMCIVREE in acquired HO in May. Rhythm expects to launch in Germany in the first half of 2027, subject to securing an exemption from the German Federal Joint Committee’s Annex II exclusion list. The company has also submitted dossiers to begin pricing discussions in France, the U.K., Italy, Spain and the Netherlands, with launches in those markets anticipated in 2027 following Germany. Rhythm said it continues to see growth opportunities in BBS and has reorganized its North American field organization. Its 42 territory managers are now dedicated to acquired HO, while a separate BBS-focused territory manager group is expected to grow to 10 managers. Rhythm Pharmaceuticals, Inc is a clinical‐stage biotechnology company dedicated to developing targeted therapies for rare genetic diseases of obesity and metabolic dysfunction. The company's research focuses on the melanocortin‐4 receptor (MC4R) pathway, which plays a central role in regulating appetite, energy expenditure and body weight. Using proprietary peptide technology, Rhythm aims to provide precision treatments to patients with specific genetic variants that disrupt normal weight regulation. The company's lead investigational product, setmelanotide, is a selective MC4R agonist designed to restore signaling in patients with deficiencies in genes such as POMC, LEPR and PCSK1. 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 "Rhythm Pharmaceuticals Q2 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for August 2026.

Rhythm Pharmaceuticals, Inc. (RYTM) is expected to deliver a year-over-year decline in earnings on higher revenues when it reports results for the quarter ended June 2026. This widely-known consensus outlook gives a good sense of the company's earnings picture, but how the actual results compare to these estimates is a powerful factor that could impact its near-term stock price. The earnings report, which is expected to be released on August 4, might help the stock move higher if these key numbers are better than expectations. On the other hand, if they miss, the stock may move lower. While the sustainability of the immediate price change and future earnings expectations will mostly depend on management's discussion of business conditions on the earnings call, it's worth handicapping the probability of a positive EPS surprise. This company is expected to post quarterly loss of $0.82 per share in its upcoming report, which represents a year-over-year change of -9.3%. Revenues are expected to be $65.87 million, up 35.8% from the year-ago quarter. The consensus EPS estimate for the quarter has remained unchanged over the last 30 days. This is essentially a reflection of how the covering analysts have collectively reassessed their initial estimates over this period. Investors should keep in mind that the direction of estimate revisions by each of the covering analysts may not always get reflected in the aggregate change. Price, Consensus and EPS Surprise Estimate revisions ahead of a company's earnings release offer clues to the business conditions for the period whose results are coming out. Our proprietary surprise prediction model -- the Zacks Earnings ESP (Expected Surprise Prediction) -- has this insight at its core. The Zacks Earnings ESP compares the Most Accurate Estimate to the Zacks Consensus Estimate for the quarter; the Most Accurate Estimate is a more recent version of the Zacks Consensus EPS estimate. The idea here is that analysts revising their estimates right before an earnings release have the latest information, which could potentially be more accurate than what they and others contributing to the consensus had predicted earlier. Thus, a positive or negative Earnings ESP reading theoretically indicates the likely deviation of the actual earnings from the consensus estimate. However, the model's predictive power is significant for positive ESP readings only. A positive Earnings ESP is a strong predictor of an earnings beat, particularly when combined with a Zacks Rank #1 (Strong Buy), 2 (Buy) or 3 (Hold). Our research shows that stocks with this combination produce a positive surprise nearly 70% of the time, and a solid Zacks Rank actually increases the predictive power of Earnings ESP. Please note that a negative Earnings ESP reading is not indicative of an earnings miss. Our research shows that it is difficult to predict an earnings beat with any degree of confidence for stocks with negative Earnings ESP readings and/or Zacks Rank of 4 (Sell) or 5 (Strong Sell). For Rhythm Pharmaceuticals, the Most Accurate Estimate is higher than the Zacks Consensus Estimate, suggesting that analysts have recently become bullish on the company's earnings prospects. This has resulted in an Earnings ESP of +1.46%. On the other hand, the stock currently carries a Zacks Rank of #3. So, this combination indicates that Rhythm Pharmaceuticals will most likely beat the consensus EPS estimate. Analysts often consider to what extent a company has been able to match consensus estimates in the past while calculating their estimates for its future earnings. So, it's worth taking a look at the surprise history for gauging its influence on the upcoming number. For the last reported quarter, it was expected that Rhythm Pharmaceuticals would post a loss of$0.86 per share when it actually produced a loss of -$0.83, delivering a surprise of +3.49%. Over the last four quarters, the company has beaten consensus EPS estimates two times. An earnings beat or miss may not be the sole basis for a stock moving higher or lower. Many stocks end up losing ground despite an earnings beat due to other factors that disappoint investors. Similarly, unforeseen catalysts help a number of stocks gain despite an earnings miss. That said, betting on stocks that are expected to beat earnings expectations does increase the odds of success. This is why it's worth checking a company's Earnings ESP and Zacks Rank ahead of its quarterly release. Make sure to utilize our Earnings ESP Filter to uncover the best stocks to buy or sell before they've reported. Rhythm Pharmaceuticals appears a compelling earnings-beat candidate. However, investors should pay attention to other factors too for betting on this stock or staying away from it ahead of its earnings release. Among the stocks in the Zacks Medical - Biomedical and Genetics industry, Jazz Pharmaceuticals (JAZZ), is soon expected to post earnings of $6.04 per share for the quarter ended June 2026. This estimate indicates a year-over-year change of +173.2%. This quarter's revenue is expected to be $1.11 billion, up 5.7% from the year-ago quarter. The consensus EPS estimate for Jazz has remained unchanged over the last 30 days. However, a higher Most Accurate Estimate has resulted in an Earnings ESP of +0.41%. When combined with a Zacks Rank of #3 (Hold), this Earnings ESP indicates that Jazz will most likely beat the consensus EPS estimate. Over the last four quarters, the company surpassed consensus EPS estimates three times. Stay on top of upcoming earnings announcements with the Zacks Earnings Calendar. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Rhythm Pharmaceuticals, Inc. (RYTM) : Free Stock Analysis Report Jazz Pharmaceuticals PLC (JAZZ) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

In early August 2026, Rhythm Pharmaceuticals is scheduled to report quarterly results that analysts expect will show higher revenue but a year-over-year earnings decline, with many anticipating a potential earnings beat versus consensus estimates. This combination of rising sales and the prospect of outperforming forecasts, despite weaker earnings, has drawn fresh attention to how Rhythm’s growth profile and cost base are evolving. We’ll now examine how anticipation of an earnings beat, despite expected weaker profitability, could influence Rhythm Pharmaceuticals’ broader investment narrative. Uncover the next big thing with 21 elite penny stocks that balance risk and reward. To own Rhythm Pharmaceuticals, you need to believe that setmelanotide can support a sustainable rare disease franchise while the company moves closer to profitability. The coming August earnings, where analysts expect higher revenue but weaker earnings, appear more like a checkpoint on how Rhythm is managing its cost base than a change to its core catalyst, which remains converting recent HO label expansions and clinical data into durable, reimbursed demand. The biggest near term risk is continued operating losses requiring fresh capital. Among recent developments, the March 2026 FDA approval of IMCIVREE for acquired hypothalamic obesity in adults and children is most relevant here, because it underpins the revenue growth that analysts expect to see in the upcoming quarter. This expanded indication, supported by TRANSCEND Phase 3 results and followed by a positive EMA opinion, sits at the heart of Rhythm’s growth story, but it also raises the stakes on securing consistent reimbursement and building international commercial scale efficiently. Yet investors should be aware that persistent losses and potential future dilution could... Read the full narrative on Rhythm Pharmaceuticals (it's free!) Rhythm Pharmaceuticals' narrative projects $971.3 million revenue and $263.4 million earnings by 2029. This requires 64.8% yearly revenue growth and a $471.2 million earnings increase from -$207.8 million today. Uncover how Rhythm Pharmaceuticals' forecasts yield a $139.47 fair value, a 31% upside to its current price. Two fair value estimates from the Simply Wall St Community span roughly US$139 to over US$540, underscoring how far apart views on Rhythm’s potential sit. Against that backdrop, the reliance on setmelanotide and ongoing operating losses give you important context for thinking about how those views might play out in practice. Explore 2 other fair value estimates on Rhythm Pharmaceuticals - why the stock might be worth just $139.47! Disagree with existing narratives? Extraordinary investment returns rarely come from following the herd, so go with your instincts. A great starting point for your Rhythm Pharmaceuticals research is our analysis highlighting 3 key rewards that could impact your investment decision. Our free Rhythm Pharmaceuticals research report provides a comprehensive fundamental analysis summarized in a single visual - the Snowflake - making it easy to evaluate Rhythm Pharmaceuticals' overall financial health at a glance. Our daily scans reveal stocks with breakout potential. Don't miss this chance: This technology could replace computers: discover 26 stocks that are working to make quantum computing a reality. The future of work is here. Discover the 34 top robotics and automation stocks leading the charge in AI-driven automation and industrial transformation. Outshine the giants: these 16 early-stage AI stocks could fund your retirement. 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. Companies discussed in this article include RYTM. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email [email protected]

BOSTON, July 21, 2026 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that it will host a live conference call and webcast at 8:00 a.m. ET on Tuesday, August 4, 2026 to report its second quarter 2026 financial results and provide a corporate update. To access the live conference call, participants may register here. While not required, it is recommended that participants join the call ten minutes prior to the scheduled start. A recording of the call will also be available under "Events and Presentations" in the Investor Relations section of the Rhythm Pharmaceuticals website at https://ir.rhythmtx.com/. The archived webcast of the financial results conference call will be available on Rhythm’s website approximately two hours after it concludes and will be available for 30 days following the call. About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 4 years and older with acquired hypothalamic obesity, adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. The European Commission (EC) has authorized setmelanotide for the treatment of obesity and control of hunger in patients 4 years of age and above with acquired hypothalamic obesity; and both the EC and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA. Setmelanotide IndicationIn the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity, in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign Other types of obesity not related to acquired HO, BBS, or POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity. Important Safety Information CONTRAINDICATIONS Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections and increased frequency of penile erections in males have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression and suicidal ideation have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation occurred in the majority of IMCIVREE-treated patients. IMCIVREE may also cause development of new melanocytic nevi or darkening of pre-existing nevi. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Acute Adrenal Insufficiency with Acquired HO: Patients with acquired HO and secondary adrenal insufficiency reported serious adverse reactions related to acute adrenal insufficiency in 5% of IMCIVREE-treated patients and no placebo-treated patients. In patients with secondary adrenal insufficiency, monitor for clinical signs of acute adrenal insufficiency. Sodium Imbalance in Patients with Acquired HO and Central Diabetes Insipidus: Patients with acquired HO and concomitant central diabetes insipidus (DI)/arginine vasopressin (AVP) deficiency reported hyponatremia in 6% of IMCIVREE-treated patients and 2% of placebo-treated patients and hypernatremia in 5% of IMCIVREE-treated patients and 4% of placebo-treated patients. Monitor serum sodium levels with changes in fluid intake and hydration status. Adjust the doses of concomitant therapies for DI/AVP deficiency as needed. ADVERSE REACTIONS Most common adverse reactions (incidence ≥20% in at least 1 indication) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the release of our financial results and our participation in upcoming events and presentations, and the date, time and content thereof. Statements using words such as “expect”, “anticipate”, “believe”, “may”, “will” and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, risks associated with the laws and regulations governing our international operations and the costs of any related compliance programs, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic conditions, and the other important factors, including those discussed under the caption “Risk Factors” in Rhythm’s Quarterly Report on Form 10-Q for the three months ended March 31, 2026, and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise. Corporate Contacts:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, [email protected] Kate WalshDirector, Corporate CommunicationsRhythm Pharmaceuticals, Inc.(857) [email protected]

BOSTON, July 08, 2026 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that results from its pivotal Phase 3 TRANSCEND trial evaluating setmelanotide, a melanocortin-4 receptor (MC4R) agonist, in patients with acquired hypothalamic obesity have been published in The New England Journal of Medicine (NEJM). The TRANSCEND study is the largest and longest placebo-controlled clinical trial ever conducted in patients with acquired hypothalamic obesity. The publication highlights robust improvements in weight and hunger achieved with setmelanotide therapy in adult and pediatric patients aged four years and older. “Patients with acquired hypothalamic obesity and their families face an urgent need for effective treatment options,” said Dr. Christian Roth, a pediatric endocrinologist and principal investigator of the Norcliffe Foundation Center for Integrative Brain Research at Seattle Children’s Research Institute, who served as the senior author and was instrumental in the trial’s planning. “The results of the TRANSCEND trial demonstrate meaningful and consistent reductions in body mass index as well as improvements in hunger. For patients and families who experience the accelerated and sustained weight gain associated with hypothalamic injury, these findings represent a potentially transformative therapeutic advancement.” In this 52-week, randomized, double blind, placebo-controlled Phase 3 study, the first 120 patients who reached 52 weeks on therapeutic regimen were evaluated as the primary analysis cohort. Patients treated with setmelanotide achieved: -19.8% placebo-adjusted difference in body mass index (BMI) reduction (n=120); Primary endpoint of mean BMI reduction of -16.5% from baseline for all patients on setmelanotide therapy (n=81) compared with +3.3% BMI change for patients on placebo (n=39) at 52 weeks (p<0.0001); 80% of patients on setmelanotide achieved BMI reduction of 5% or greater at 52 weeks; and Clinically meaningful improvements in hunger. Setmelanotide was generally well tolerated. No new safety signals were observed, and adverse events leading to treatment discontinuation were comparable between treatment and placebo groups. In March 2026, the U.S. Food and Drug Administration (FDA) approved setmelanotide (IMCIVREE®) as the first and only therapy for acquired hypothalamic obesity in adults and children aged 4 years and older. Also in March 2026, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended marketing authorization for IMCIVREE to include the treatment of obesity and the control of hunger in adults and children 4 years of age and above with acquired hypothalamic obesity due to hypothalamic injury or impairment. “Publication of the TRANSCEND data in NEJM underscores both the strength of the clinical evidence and the potential positive impact of setmelanotide for people living with acquired hypothalamic obesity,” said David Meeker, M.D., Chairman, President and Chief Executive Officer of Rhythm Pharmaceuticals. “We are deeply grateful to the study authors, investigators, patients and families whose commitment made this landmark trial possible. With FDA and EU approvals now in place and the regulatory submission under review in Japan, our goal is to bring this first‑in‑class therapy to patients worldwide who urgently need a targeted treatment option.” About the Phase 3 TRANSCEND TrialThe global, randomized, double blind, placebo-controlled Phase 3 TRANSCEND trial evaluated the efficacy and safety of setmelanotide in patients aged 4 years and older with acquired hypothalamic obesity. A total of 120 participants were randomized 2:1 to once daily subcutaneous setmelanotide or placebo for 52 weeks. The primary endpoint was mean percent change in BMI after 52 weeks of treatment. Full topline results were previously announced in April 2025. About Acquired Hypothalamic ObesityAcquired hypothalamic obesity is a rare disease characterized by accelerated and sustained weight gain caused by an injury to the hypothalamus. Hypothalamic injury may lead to decreased alpha-melanocyte-stimulating hormone (α-MSH) production and impairment of MC4R pathway signaling. The MC4R pathway is responsible for regulating energy balance and body weight. Acquired hypothalamic obesity most frequently follows the growth or treatment of craniopharyngioma, astrocytoma or other hypothalamic-pituitary tumors. Additional causes of injury may include traumatic brain injury, stroke, or inflammation. Due to impairment of the MC4R pathway, patients experience accelerated and sustained weight gain, often accompanied by hyperphagia and/or decreased energy expenditure. Acquired hypothalamic obesity can occur as early as six months following hypothalamic injury. About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 4 years and older with acquired hypothalamic obesity, adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. The European Commission (EC) has authorized setmelanotide for the treatment of obesity and control of hunger in patients 4 years of age and above with acquired hypothalamic obesity; and both the EC and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA. Setmelanotide IndicationIn the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity, in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign Other types of obesity not related to acquired HO, BBS, or POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity. Important Safety Information CONTRAINDICATIONS Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections and increased frequency of penile erections in males have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression and suicidal ideation have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation occurred in the majority of IMCIVREE-treated patients. IMCIVREE may also cause development of new melanocytic nevi or darkening of pre-existing nevi. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Acute Adrenal Insufficiency with Acquired HO: Patients with acquired HO and secondary adrenal insufficiency reported serious adverse reactions related to acute adrenal insufficiency in 5% of IMCIVREE-treated patients and no placebo-treated patients. In patients with secondary adrenal insufficiency, monitor for clinical signs of acute adrenal insufficiency. Sodium Imbalance in Patients with Acquired HO and Central Diabetes Insipidus: Patients with acquired HO and concomitant central diabetes insipidus (DI)/arginine vasopressin (AVP) deficiency reported hyponatremia in 6% of IMCIVREE-treated patients and 2% of placebo-treated patients and hypernatremia in 5% of IMCIVREE-treated patients and 4% of placebo-treated patients. Monitor serum sodium levels with changes in fluid intake and hydration status. Adjust the doses of concomitant therapies for DI/AVP deficiency as needed. ADVERSE REACTIONS Most common adverse reactions (incidence ≥20% in at least 1 indication) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the safety, efficacy, potential benefits of, and clinical design or progress, potential regulatory submissions, approvals and timing thereof for any of our products or product candidates at any dosage or in any indication; the presentation of clinical data and results from our trials, including the ongoing Phase 2 trial of setmelanotide in patients with PWS, clinical and real-world efficacy and safety data related to the use of setmelanotide and any of our other product candidates in patients with acquired hypothalamic obesity and our participation in upcoming events and presentations and publications, including the publication of the results from our pivotal Phase 3 TRANSCEND trial evaluating setmelanotide in patients with acquired hypothalamic obesity in The New England Journal of Medicine, and the content, date and timing of any of the foregoing. Statements using words such as “expect”, “anticipate”, “believe”, “may”, “will” and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, risks associated with the laws and regulations governing our international operations and the costs of any related compliance programs, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic conditions, and other important factors, including those discussed under the caption “Risk Factors” in Rhythm’s Quarterly Report on Form 10-Q for the three months ended March 31, 2026, and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise. Corporate Contacts:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, [email protected] Kate WalshDirector, Corporate CommunicationsRhythm Pharmaceuticals, Inc.(857) [email protected]

BOSTON, June 12, 2026 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that it will host a live conference call and webcast on Saturday, June 13 at 9:00 a.m. ET/8:00 a.m. CT to discuss interim six-month results from the Company’s Phase 2 trial evaluating setmelanotide in patients with Prader-Willi syndrome (PWS). David Meeker, M.D., Chairman, President and Chief Executive Officer will be joined by Jennifer Miller, M.D., Pediatric Endocrinology, University of Florida College of Medicine to discuss the data. Conference Call Information Rhythm Pharmaceuticals will host a live conference call and webcast at 9:00 a.m. ET/8:00 a.m. CT on Saturday, June 13 to discuss this update. Participants may register for the conference call here. It is recommended that participants join the call ten minutes prior to the scheduled start. A webcast of the call will also be available under "Events and Presentations" in the Investor Relations section of the Rhythm Pharmaceuticals website at https://ir.rhythmtx.com/. The archived webcast will be available on Rhythm Pharmaceuticals’ website approximately two hours after the conference call and will be available for 30 days following the call. About Rhythm Pharmaceuticals Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 4 years and older with acquired hypothalamic obesity, adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. The European Commission (EC) has authorized setmelanotide for the treatment of obesity and control of hunger in patients 4 years of age and above with acquired hypothalamic obesity; and both the EC and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA. Setmelanotide Indication In the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity, in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign Other types of obesity not related to acquired HO, BBS, or POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity. Important Safety Information CONTRAINDICATIONS Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections and increased frequency of penile erections in males have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression and suicidal ideation have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation occurred in the majority of IMCIVREE-treated patients. IMCIVREE may also cause development of new melanocytic nevi or darkening of pre-existing nevi. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Acute Adrenal Insufficiency with Acquired HO: Patients with acquired HO and secondary adrenal insufficiency reported serious adverse reactions related to acute adrenal insufficiency in 5% of IMCIVREE-treated patients and no placebo-treated patients. In patients with secondary adrenal insufficiency, monitor for clinical signs of acute adrenal insufficiency. Sodium Imbalance in Patients with Acquired HO and Central Diabetes Insipidus: Patients with acquired HO and concomitant central diabetes insipidus (DI)/arginine vasopressin (AVP) deficiency reported hyponatremia in 6% of IMCIVREE-treated patients and 2% of placebo-treated patients and hypernatremia in 5% of IMCIVREE-treated patients and 4% of placebo-treated patients. Monitor serum sodium levels with changes in fluid intake and hydration status. Adjust the doses of concomitant therapies for DI/AVP deficiency as needed. ADVERSE REACTIONS Most common adverse reactions (incidence ≥20% in at least 1 indication) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the safety, efficacy, potential benefits of, and clinical design or progress, potential regulatory submissions, approvals and timing thereof for any of our products or product candidates at any dosage or in any indication; the presentation of clinical data and results from our trials, including the ongoing Phase 2 trial of setmelanotide in patients with PWS, including at The Endocrine Society’s Annual Meeting taking place June 13-16, 2026 in Chicago, and the content, date and timing of any of the foregoing. Statements using words such as “expect”, “anticipate”, “believe”, “may”, “will” and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, risks associated with the laws and regulations governing our international operations and the costs of any related compliance programs, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic conditions, and other important factors, including those discussed under the caption “Risk Factors” in Rhythm’s Quarterly Report on Form 10-Q for the three months ended March 31, 2026, and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise. Corporate Contacts: David Connolly Head of Investor Relations and Corporate Communications Rhythm Pharmaceuticals, Inc. 857-264-4280 [email protected] Kate WalshDirector, Corporate CommunicationsRhythm Pharmaceuticals, Inc. [email protected]

Shareholders of Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM) will be pleased this week, given that the stock price is up 18% to US$96.23 following its latest quarterly results. The business exceeded expectations with revenue of US$60m coming in 7.2% ahead of forecasts. Statutory losses were US$0.83 a share, in line with what the analysts predicted. This is an important time for investors, as they can track a company's performance in its report, look at what experts are forecasting for next year, and see if there has been any change to expectations for the business. So we gathered the latest post-earnings forecasts to see what estimates suggest is in store for next year. We've found 21 US stocks that are forecast to pay a dividend yield of over 6% next year. See the full list for free. Taking into account the latest results, the most recent consensus for Rhythm Pharmaceuticals from 16 analysts is for revenues of US$295.8m in 2026. If met, it would imply a major 36% increase on its revenue over the past 12 months. Losses are expected to increase slightly, to US$3.32 per share. Before this latest report, the consensus had been expecting revenues of US$287.4m and US$3.14 per share in losses. So it's pretty clear consensus is mixed on Rhythm Pharmaceuticals after the new consensus numbers; while the analysts lifted revenue numbers, they also administered a pronounced increase to per-share loss expectations. Check out our latest analysis for Rhythm Pharmaceuticals There was no major change to the consensus price target of US$138, with growing revenues seemingly enough to offset the concern of growing losses. That's not the only conclusion we can draw from this data however, as some investors also like to consider the spread in estimates when evaluating analyst price targets. The most optimistic Rhythm Pharmaceuticals analyst has a price target of US$159 per share, while the most pessimistic values it at US$105. These price targets show that analysts do have some differing views on the business, but the estimates do not vary enough to suggest to us that some are betting on wild success or utter failure. These estimates are interesting, but it can be useful to paint some more broad strokes when seeing how forecasts compare, both to the Rhythm Pharmaceuticals' past performance and to peers in the same industry. We can infer from the latest estimates that forecasts expect a continuation of Rhythm Pharmaceuticals'historical trends, as the 51% annualised revenue growth to the end of 2026 is roughly in line with the 58% annual growth over the past five years. Compare this with the broader industry, which analyst estimates (in aggregate) suggest will see revenues grow 21% annually. So although Rhythm Pharmaceuticals is expected to maintain its revenue growth rate, it's definitely expected to grow faster than the wider industry. The most important thing to take away is that the analysts increased their loss per share estimates for next year. Pleasantly, they also upgraded their revenue estimates, and their forecasts suggest the business is expected to grow faster than the wider industry. There was no real change to the consensus price target, suggesting that the intrinsic value of the business has not undergone any major changes with the latest estimates. Following on from that line of thought, we think that the long-term prospects of the business are much more relevant than next year's earnings. We have estimates - from multiple Rhythm Pharmaceuticals analysts - going out to 2028, and you can see them free on our platform here. Another thing to consider is whether management and directors have been buying or selling stock recently. We provide an overview of all open market stock trades for the last twelve months on our platform, here. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team (at) simplywallst.com. 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.