PVLA

Image source: The Motley Fool. Tuesday, Aug. 4, 2026 at 8:30 a.m. ET Founder and Chief Executive Officer - Wesley Kaupinen Chief Scientific Officer - Jeffrey Martini Chief Financial Officer - Matthew Korenberg Vice President of Investor Relations and Corporate Affairs - Marcy Nanus Operator: Good day, and thank you for standing by. Welcome to the Palvella Therapeutics' Second Quarter 2026 Financial Results 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, Marcy Nanus, Vice President of Investor Relations and Corporate Affairs. Marcy Nanus: Thank you, operator. Good morning, and thank you for joining the Palvella Therapeutics' second quarter 2026 Financial Results And Corporate Update Call. As a reminder, our press release detailing today's announcements can be found in the Investors section of our website at www.palvellatx.com. On today's call, I am joined by Wes Kaupinen, our Founder and Chief Executive Officer; Dr. Jeff Martini, our Chief Scientific Officer; and Matt Korenberg, our Chief Financial Officer. Before we begin, please note that today's remarks may include forward-looking statements regarding our development programs, regulatory strategy, commercial planning and financial outlook. These statements are based on current assumptions and are subject to risks and uncertainties that could cause actual results to differ materially. Please refer to our SEC filings for a full discussion of these risk factors. And now I'll turn the call over to Wes. Wesley Kaupinen: Thanks, Marcy. Good morning, everyone, and thank you for joining us. The second quarter marked the culmination of many years of work to pioneer and accelerate the development of QTORIN rapamycin through 2 successful clinical studies in microcystic lymphatic malformations, a serious, rare, chronically debilitating lifelong genetic disease for which there are no FDA-approved therapies. During the quarter, we achieved 3 important milestones. First, the compelling safety and efficacy results from our Phase III SELVA study, supported an in-person pre-NDA meeting with the FDA. Second, following that meeting, FDA granted Palvella rolling review, a feature available under Fast Track and Breakthrough Therapy Designation that is intended to expedite review and help bring important new therapies to patients earlier by allowing FDA to begin reviewing completed sections of the NDA before the full application is submitted. And third, thanks to the exceptional execution of the Palvella team, we completed the submission of the first module of our NDA. These milestones have brought us meaningfully closer to achieving our most important near-term corporate objective, securing FDA approval for QTORIN rapamycin. I am pleased to report today that, #1, we remain on track to complete our NDA submission in the second half of this year. And #2, we also remain on track for potential FDA approval in the first half of 2027. In terms of launch readiness, we have continued assembling the leadership required to support a successful U.S. launch. We've recruited commercial and medical affairs leaders with deep experience in rare disease and dermatology launches, and I'm pleased to report that team has rapidly advanced key prelaunch activities. In parallel, under the leadership of our Chief Scientific Officer, Dr. Jeff Martini, significant progress continues to be made across our late-stage pipeline and QTORIN platform. This includes our QTORIN rapamycin programs in cutaneous venous malformations and clinically significant angiokeratomas, both of which have been granted Fast Track Designation by the FDA as well as our QTORIN pitavastatin program in disseminated superficial actinic porokeratosis. Palvella stands today with both a late-stage rare disease pipeline and an internal product development engine powered by the QTORIN platform, designed to repeatably bring first-in-disease therapies to rare disease communities with significant unmet need and no approved treatment options today. We are developing therapies for 4 serious rare skin diseases and vascular malformations that have been overlooked despite significant unmet need. These diseases have historically been underappreciated, not because their clinical burden is misunderstood, but because their true prevalence and incidence have been poorly characterized. On the top row, our data-driven epidemiologic work indicates that these indications each may represent multi-billion-dollar total addressable markets in the U.S. based on estimated diagnosed U.S. prevalence and the expectation for orphan pricing at launch, an estimated greater than 30,000 patients with microcystic LMs, greater than 75,000 patients with cutaneous venous malformations, greater than 50,000 with clinically significant angiokeratomas and greater than 50,000 patients estimated with disseminated superficial actinic porokeratosis. In the middle row, at Palvella, we focus exclusively on diseases with no FDA-approved treatments and the potential for Palvella to pioneer first-in-disease therapies. We believe such a strategy is advantageous when compared to a more conventional biotech approach of pursuing incremental differentiation in competitive markets with well-resourced entrenched incumbents. For each of the diseases you see listed here, we believe, assuming continued clinical and regulatory execution, that we're on a trajectory to potentially introduce the first FDA-approved therapies for each of these indications. Finally, physician market research further reinforces the potential for an attractive uptake curve at launch across all 4 indications. More than 80% of physicians surveyed indicated they would consider the QTORIN product candidate targeted for that indication as a first-line therapy, if approved. Moving to QTORIN rapamycin. QTORIN rapamycin was designed as a pipeline-in-a-product, one product candidate with the potential to address multiple rare diseases in which hyperactivated mTOR signaling is a central pathogenic driver. We are now executing on that strategy across several indications. In the last couple of years, we've expanded the program from microcystic lymphatic malformations into cutaneous venous malformations and clinically significant angiokeratomas. We anticipate potential FDA approval for QTORIN rapamycin in cutaneous venous malformations in 2029 and clinically significant angiokeratomas in 2031, creating the potential for 2 significant indication expansions while the microcystic LM launch is still in its early years. Later this year, we expect to announce a fourth indication with additional indications beyond the fourth indication already in planning by our R&D team. Overall, our pipeline-in-a-product strategy provides a highly efficient path to expand QTORIN rapamycin across multiple mTOR-driven skin diseases. Under our current development plan, potential approvals in multiple indications could expand QTORIN rapamycin's addressable U.S. patient population from more than 30,000 patients with microcystic lymphatic malformations to more than 300,000 patients across multiple mTOR-driven indications. Our approach to launch readiness is informed by learnings from successful first-in-disease orphan drug launches, including Oxervate, VYJUVEK and TEPEZZA, which demonstrate how focused early execution across a small number of critical areas can meaningfully shape adoption. First, we have recruited experienced rare disease and dermatology leaders across commercial and medical affairs functions who are already executing prelaunch activities in the field. Second, the strength and consistency of our clinical data, together with physician market research that indicates strong interest in first-line use, support the potential for QTORIN rapamycin to become the first approved therapy for microcystic LMs and, if approved, a potential first-line treatment and future standard of care. Third, our teams are actively engaging physicians, including specialists at vascular anomaly centers, to deepen disease education and prepare treatment centers for a potential launch. Fourth, we are building the patient services infrastructure required to support patient access, coverage and treatment initiation following a potential approval. Finally, our balance sheet, significantly strengthened in the first quarter through a $230 million capital raise, allows us to invest ahead of approval and build commercial readiness with urgency and strength. We are deeply grateful to the leading biotechnology investors who participated in that financing and whose support is enabling us to advance our mission of bringing QTORIN rapamycin and other QTORIN programs to patients. Taken together, these initiatives are designed to ensure that, if approved, QTORIN rapamycin reaches pediatric and adult patients living with microcystic lymphatic malformations as quickly and effectively as possible. The core of Palvella's commercial and medical affairs leadership team is now assembled. We have recruited an exceptional team of leaders with deep experience across rare disease, dermatology, medical affairs, market access, sales, marketing and successful orphan drug launches while continuing to add talented professionals at all levels of the organization. They understand the critical requirements of a first-in-disease launch: building disease awareness, educating physicians, supporting patient identification, preparing treatment centers, establishing access pathways and enabling seamless treatment initiation following a potential approval. Our senior leaders and I remain deeply involved in recruiting and selecting these teams, and we've augmented our own efforts by engaging world-class executive search firms to help us attract the very best talent. We have also increased our planned sales force at launch to approximately 40 sales reps, at the upper end of our prior guidance. We believe this additional investment will strengthen field coverage, physician education, patient identification and access support from day 1, ultimately helping pediatric and adult patients who may potentially benefit from QTORIN rapamycin, if approved, to access treatment as efficiently as possible. Overall, I am grateful to work alongside Ashley, Jen, Kent, Vimal and Peter and the exceptional team they are continuing to build to advance the Palvella mission. Together, they bring passion, thoughtfulness and deep collective commercial and medical experience to a shared ambition, making the potential launch of QTORIN rapamycin the best launch any of us have been a part of, for patients, physicians and for the broader microcystic LM community. We believe QTORIN rapamycin has the potential to become the first approved therapy, a first-line treatment and ultimately, a future standard of care for microcystic LMs based on 3 important attributes. First, QTORIN rapamycin is designed to address the causal mTOR pathway directly within the pathogenic tissue of interest. This targeted localized approach could be particularly compelling in a lifelong disease that may require chronic treatment. Second, the Phase III SELVA study delivered highly compelling results. The study met its primary endpoint, key secondary endpoint and all 4 prespecified secondary endpoints with high statistical significance, with 95% of patients demonstrating improvement on the primary endpoint at week 24. Third, QTORIN rapamycin demonstrated a favorable safety profile. That profile is especially meaningful when contrasted with invasive procedures and off-label systemic approaches that carry substantial treatment burden, monitoring requirements and tolerability limitations. Taken together, the therapeutic approach, the consistency and strength of the SELVA results and the favorable safety profile provide what we believe is a foundation for QTORIN rapamycin to become the first approved therapy for microcystic LMs and, if approved, to establish a new first-line standard of care for pediatric and adult patients living with this serious lifelong disease. Additional prelaunch activities are accelerating in terms of physician engagement. We have already engaged more than 200 of our initial 400 target clinics, while our broader reach extends well beyond that group through a meaningful presence at major medical congresses, vascular anomaly meetings and other scientific forums. Together, these efforts are deepening physician understanding of microcystic lymphatic malformations, including the underlying genetics, the causal role of mTOR signaling and the importance of timely diagnosis and treatment, while strengthening engagement within the vascular anomaly and dermatology communities. We are also building what we believe can become a best-in-class patient services organization. We made the strategic decision to internalize our core patient support services, giving Palvella greater ownership of the patient experience and tighter coordination across patient access, reimbursement and treatment initiation. Our leadership team and initial hires bring deep recent experience launching a first-in-disease therapy for a serious rare skin disease, and we are actively expanding the team with additional top talent. Our recent payer research confirms our earlier payer findings. Payers consistently recognize microcystic LMs as a serious rare vascular malformation with substantial unmet need and no FDA-approved therapies available today. Against that backdrop, our research indicates orphan drug pricing ranges are likely to be well supported with a favorable outlook for patient access and reimbursement. Finally, we're executing from a position of financial strength with approximately $250 million in cash at the end of the quarter, we are well capitalized through a potential FDA approval and a successful stand-alone commercial launch. As I mentioned earlier, our NDA submission remains on track for the second half of 2026. Our application is supported by Breakthrough Therapy Designation, Fast Track Designation, Orphan Drug Designation and an FDA Orphan Product Grant. We're pursuing approval through the 505(b)(2) regulatory pathway, which allows us to leverage FDA's prior findings for rapamycin while supporting our application with the robust clinical data generated through our own development program. Our evidence package includes the positive Phase III and Phase II studies as well as real-world clinical evidence, and we intend to seek a broad label and traditional full approval. Before moving on, I'd like to recognize and thank our NDA team, including our Head of Regulatory Affairs, Shama Munim, for their unwavering commitment to delivering a high-quality NDA submission and for executing with urgency, discipline and meticulous attention to detail. Their work reflects what makes Palvella special, a shared commitment to the patients and families we serve, a deep sense of purpose and an unwavering determination to achieve a potential near-term FDA approval and bring QTORIN rapamycin to patients as quickly as possible. With that, I'll turn the call over to Jeff to discuss our rare disease pipeline programs. Jeffrey Martini: Thank you, Wes. As you've heard this morning, Palvella has built tremendous momentum across the business. I'm very excited about the pipeline, including the data we have presented over the last quarter from both SELVA and TOIVA, the progress we are making in our clinically significant angiokeratoma and DSAP programs and the additional new program announcements later this year. During the past quarter, we continued to strengthen our scientific presence at the major congresses, helping us expand disease awareness, deepen relationships with the treating community and support launch readiness. I want to highlight our participation at the ISSVA World Congress in May, where Palvella served as a platinum sponsor. Dr. Jim Treat delivered a late-breaking presentation that included results from both our Phase III SELVA study in microcystic lymphatic malformations and our Phase II TOIVA study in cutaneous venous malformations. I'll begin with our lead program in microcystic lymphatic malformations. Before reviewing the data, I want to put these results in context. Microcystic lymphatic malformations is a serious condition that often presents in childhood and persists throughout a patient's life. These lesions cause leaking, bleeding, recurrent infections and substantial physical and emotional burden during some of the most formative years of a child's life. As a reminder, our previously reported Phase III SELVA study results demonstrated that 95% of patients improved on the mLM-IGA, our primary endpoint. At ISSVA, Dr. Jim Treat presented the new analysis shown here, focused on children aged 6 to 11. What we observed was a rapid, large-magnitude treatment effect that was consistent across all 13 children studied. By week 24, the mean mLM-IGA improvement was 2.46 points, and every child in this cohort was rated as either much improved or very much improved. The photographs shown here help bring those numbers to life. They illustrate not only the magnitude of improvement that can be achieved with continued treatment, but also what that improvement may mean for a child living every day with a visible, symptomatic, lifelong disease. Importantly, every patient in this cohort elected to continue treatment in the treatment extension, further supporting the potential role of QTORIN rapamycin in the chronic management of this disease. One key objective of SELVA was to better understand the natural variability of the disease and place the observed treatment response in that context. SELVA incorporated an innovative trial design with input from clinicians, patients and regulatory experts. Before treatment began, patients completed an 8-week run-in period, allowing us to prospectively assess changes in the disease without treatment in the same patients. Photographs from both the run-in and treatment periods were then evaluated through a prespecified blinded independent review. During the untreated run-in period, disease severity remained essentially unchanged with a mean change in the mLM-MCSS of negative 0.1. This demonstrates that the disease did not spontaneously improve during the observation period. The mLM-MCSS was a key secondary endpoint in SELVA, and the improvement observed during treatment was highly statistically significant. Following 24 weeks of treatment with QTORIN rapamycin, blinded mean mLM-MCSS improved by 3.4 points, representing 48% of the maximum potential improvement from baseline. Importantly, this design allowed us to contrast disease stability without treatment with the marked improvement observed after treatment, all based on blinded independent assessment. We believe these findings provide objective confirmation that the improvements observed in SELVA resulted from QTORIN rapamycin and further strengthen the overall body of evidence supporting the program. Cutaneous venous malformations represent a significant unmet need with more than 75,000 diagnosed patients in the United States and, importantly, no FDA-approved therapies. Recent publications continue to identify sirolimus or rapamycin as the most established medical therapy for internal venous malformations, reinforcing the rationale for QTORIN rapamycin. As a reminder, our Phase II TOIVA study demonstrated that 73% of patients improved on the cVM-IGA, more than twice our predefined success threshold. Based on the positive data from TOIVA, our immediate priority is initiating the Phase III study. The planned next steps are clear. First, we expect to meet with FDA at our End of Phase II meeting to review the TOIVA data and finalize the pivotal study design. We will then initiate the Phase III study and remain on track to do so in the fourth quarter. Before moving to the ISSVA data, I want to take a moment to address our Breakthrough Therapy Designation request. At the time of our submission, we included 12-week data, and FDA did not grant the designation based on our initial package. This does not impact the path forward in cutaneous venous malformations that I just laid out. We remain on track and enthusiastically committed to pursuing an approval in the cVM indication as quickly as possible. That said, we now have the complete 24-week efficacy data set and the final Phase II qualitative report, both of which will be reviewed at our planned End of Phase II meeting. Following our upcoming FDA interaction, we believe these additional data can support a substantially stronger Breakthrough Therapy Designation resubmission package following the initiation of the Phase III cVM study. Turning to the ISSVA presentation, I want to review the additional data that Dr. Treat highlighted during his late-breaking presentation. He presented results for 2 key clinical signs of disease: lesion height or engorgement and overall appearance. Both are important manifestations of disease burden and arise directly from abnormal dilated venous channels within the skin. These visible manifestations can cause physical discomfort, interfere with daily activities and create a meaningful burden for patients. Improvements in both measures were evident at week 4, were statistically significant at every assessed time point and continued to improve through week 24. The continued improvement through week 24 suggests that patients derive increasing benefit with exposure to drug over time. That is an important profile for a chronic disease in which long-term treatment is likely required. After reviewing the clinical data from TOIVA, I also spent time reading through the qualitative interviews from the 24-week study. For me, those interviews brought the data to life. I was genuinely moved by the impact QTORIN rapamycin had, and I want to share one of those quotes that particularly stood out to me, and there are many others like it. "It's definitely had a big impact. It's a lot easier to focus on school and have fun, hang out with friends and be in the moment when I'm not in as much pain." We look forward to submitting these 24-week data and qualitative findings to FDA and reviewing them at our planned End of Phase II meeting to inform and support finalization of the Phase III study design. We're also very excited about our clinically significant angiokeratoma program. This represents another natural extension of the QTORIN rapamycin pipeline-in-a-product strategy, addressing a serious rare lymphatic malformation affecting more than 50,000 diagnosed patients in the United States with no FDA-approved therapies. The first patients were dosed in April, and our Phase II LOTU study is evaluating QTORIN rapamycin in up to 15 patients. We look forward to presenting data from the study in the second half of 2027. We have also seen strong enthusiasm from investigators at leading vascular anomaly and dermatology centers. Last week, an independent KOL call featuring Dr. [ Macario ] and Graig Suvannavejh further highlighted the significant unmet need and limitations of current treatment options. This is consistent with our physician research in which 96% of surveyed physicians indicated that they would incorporate QTORIN rapamycin into their practice. Importantly, we expect this program to follow a supplemental NDA pathway, providing another potential opportunity to efficiently expand QTORIN rapamycin following initial approval. When we consider the scientific rationale, investigator enthusiasm, physician interest and potential supplemental NDA pathway, we believe this represents another important opportunity to address a serious rare disease with substantial unmet need. One of the capabilities we take great pride in is how closely our scientific team tracks advances across rare diseases. Through our network of medical and scientific experts, we are often among the first to hear about important scientific breakthroughs and emerging changes in clinical practice. We have also incorporated AI-enabled tools to continuously monitor developments in the scientific literature, intellectual property landscape as well as patterns of off-label systemic drug use. Together, these efforts deepen our understanding of disease biology and unmet need and help us to identify new opportunities for the QTORIN platform. New literature published this quarter adds to the growing evidence around both the substantial unmet need in clinically significant angiokeratomas and the potential role of QTORIN rapamycin. These reports highlight that angiokeratomas can develop and proliferate during childhood and adolescence and may cause persistent bleeding, pain, pruritus, hyperkeratosis and substantial disease burden. The literature also underscores the limitations of current treatment, which remains largely dependent on destructive procedures, while identifying rapamycin as a potential therapeutic option. Together, these independent publications strengthen the scientific foundation for our QTORIN rapamycin program as we continue advancing this important indication. Turning to DSAP. This remains a highly compelling program targeting a chronic, progressive, precancerous skin disease with no FDA-approved therapies. QTORIN pitavastatin is designed to be the first pathogenesis-directed therapy for DSAP by targeting the causal mevalonate pathway, and we remain on track for Phase II initiation in the fourth quarter of 2026. We also continue to see strong patient interest in this program, which underscores both the unmet need and the potential opportunity. We've already received a high level of inbound interest from patients seeking to participate in the study. The quotes on this slide are particularly powerful. One patient shared, "Thank you for doing the work you're doing. Our lives go dark after having this. It mentally and physically takes a toll. Life cannot be enjoyed the way it once was." Another said, "Looking for a breakthrough. This has been devastating." These statements highlight both the significant burden of disease and the strong desire for an effective FDA-approved treatment option. With that, I'll turn the call over to Matt to review our financial results. Matthew Korenberg: Thanks, Jeff. As of June 30, 2026, Palvella had approximately $251 million in cash, providing significant financial flexibility to invest in maximizing the potential launch of the company's first commercial product, if approved. In addition, our balance sheet provides sufficient capital to advance our entire pipeline during what we believe will be one of the most catalyst-rich periods in Palvella's history. Our strong cash position is a result of the successful financing completed in February. While our original objective was to raise $150 million, we ultimately raised $230 million, allowing us to invest in multiple high-return initiatives designed to derisk and strengthen our commercial launch. Commercially, we have expanded our launch plans, including increasing our expected field force to approximately 40 sales reps and investing in several high-impact marketing and disease awareness initiatives. Within medical affairs, we've begun hiring medical science liaisons earlier than originally anticipated and now plan to build a larger team than initially envisioned. I've been personally involved in the recruiting process and have met every candidate that we've hired. I'm incredibly impressed with the quality of the candidates we've been able to hire, including individuals with rare disease experience at Horizon, Disc Medicine and other rare disease companies. Collectively, these commercial and medical affairs investments are intended to improve the initial launch performance and to deliver drug to patients sooner. As a result of these incremental investments, we expect our targeted 2026 cash spend to increase modestly. We're now expecting approximately $85 million to $95 million in cash expenses this year. As we reflect back on our plans from earlier this year and the subsequent changes following our positive Phase III SELVA data and the subsequent successful financing, we now have more resources on the commercial and medical fronts. Our plans for pipeline expansion are accelerating, and we plan to increase our resources during our commercial marketing of QTORIN rapamycin, if approved. Factoring in all of these changes, we still expect to go into our launch with more capital on the balance sheet than originally expected to support the business. With that, I can turn the call back over to Wes for some additional comments prior to opening the line for questions. Wesley Kaupinen: Thanks, Matt. In closing, what sets Palvella apart is both our exceptional team and our repeatable model for identifying, developing and commercializing first-in-disease therapies for serious rare diseases previously thought to be untreatable. Our model is unique. We focus on high unmet need, commercially attractive rare diseases with well-understood biology, emerging human proof-of-concept data that signals the potential for clinical benefit and meaningful unmet need. We then apply the QTORIN platform to develop targeted localized therapies designed to optimize the risk-benefit profile while generating new and durable intellectual property. What gives me the greatest confidence in Palvella's future is the team executing this strategy. I have the privilege of working alongside a highly dedicated team of colleagues every day who bring deep scientific, clinical, regulatory, commercial and operational expertise, together with an extraordinary work ethic, a strong sense of urgency and an unwavering commitment to patients. Together, those capabilities enable us to advance innovative therapies toward FDA approval with greater speed, discipline and capital efficiency than traditional drug development approaches. Our goal remains clear, to serve patients with serious rare skin diseases and vascular malformations for which there are no FDA-approved therapies while building Palvella into the leading rare disease biopharmaceutical company in this field. I'd like to thank our employees, patients, advocacy partners, external collaborators and our shareholders for their continued trust and support. With that, operator, we will now open the line for questions. Operator: [Operator Instructions] Our first question comes from Alexa Deemer at Cantor Fitzgerald. Alexa Deemer: This is Alexa Deemer on for Josh, and congrats on a great quarter. So perhaps you could elaborate a bit more about your ongoing efforts to identify MLM patients? And then are your recent findings in line with the initial estimates of around 30,000 diagnosed patients? Wesley Kaupinen: Great. Alexa, thanks for being on, and thanks for the questions. I can confirm that our recent findings are in line with previous estimates. Last year, we published a claims analysis at a medical congress that indicated somewhere between 45,000 and 95,000 diagnosed MLM patients in the U.S. Importantly, in that analysis that's published, there was also an estimated annual incidence of 1,500 or more newly diagnosed patients that will come into that pool. We like to be conservative in our approach on epi, so we can confirm that we believe there is greater than 30,000 diagnosed patients in the United States with microcystic lymphatic malformations. And I appreciate you asking the question in terms of patient identification. The key there is to have your team in the field. We know where a lot of these patients are concentrated. This is a market that has experienced organic market development as a function of vascular anomaly centers emerging over the last 20 years that have high patient volumes. We know where those centers are. We know who the physicians are that take care of these patients. And so we're making efforts to be in front of those physicians at their sites, but also with a strong presence at medical congresses as well. Operator: Our next question comes from Whitney Ijem at Canaccord Genuity. Whitney Ijem: Just first one on DSAP Phase II. Can you remind us of the target enrollment for that study? Sorry if I missed it, I was juggling calls. And I guess just given your comments on demand there so far, is there a scenario where that program could proceed more quickly than the angiokeratoma program just as we think about cadence of data readouts next year? Wesley Kaupinen: Yes. Thanks for those questions. Whitney, I'll start off with some comments and then ask Jeff to also add additional color. So on the DSAP Phase II study, we expect that to be about a 15-patient Phase II study. Will it proceed more quickly than angio? To speak about angio for 1 minute, we did start that trial ahead of original expectations. That trial started in the first half of this year, originally expectations for that were second half of this year. Our clinical operations team has done a great job engaging sites, screening patients, making sure we're getting the right patients into the study, and that study is anticipated to read out in the second half of next year. We'll firm up timelines in terms of the DSAP readout around the time of initiation of the Phase II study, which we expect to be sometime in the second half of this year. Whitney Ijem: And then just a quick follow-up. We conducted a KOL or a physician survey recently. And I guess from the feedback of that survey, there was about 60% of patients on average of the MLM patients managed by DSAPs who are actively seeking treatment for their MLM, with the main reasons why patients were not seeking treatment being just kind of like comments around not bothered, some are asymptomatic, et cetera. So I'm just curious, as you think about the greater than 30,000 number, is that focused specifically on those patients who would be kind of thought to be symptomatic enough to be seeking treatment? Or how should we kind of think about that headed into launch? Wesley Kaupinen: Yes. So our claims data show that there was 45,000 to 95,000 patients in clinical medicine, Whitney. We've said greater than 30,000 to be conservative. We know now as of about 10 years ago that there's been discoveries around the genetics and the causal biology of this disease. So microcystic lymphatic malformations are a proliferative disease that is progressive in nature. So patients who may have less burden from their daily disease, we believe are also very good candidates for QTORIN rapamycin if approved. I think some of the data that Jeff showed earlier around pediatric patients who may be earlier in their disease cycle and those patients had very good responses. And we think that approach applies to patients who may be less burdensome from a symptom perspective because if the disease is left -- goes untreated, it will predictably proliferate and progress and become more problematic. So that will be the approach that our medical affairs team takes, our commercial team. This is an approach that we've derived from our interactions with the thought leaders such as Jim Treat at Children's Hospital of Philadelphia, Mike Kelly at the Cleveland Clinic. Operator: Our next question comes from Ritu Baral at TD Cowen. Ritu Baral: Wes, I wanted to ask about the deployment strategy of the 40 reps that you mentioned across the vascular anomaly clinics. Do you guys currently have an estimate of how many identified vascular anomaly clinics there are, either now or at the time of the commercial launch since you mentioned more opening up? What percentage of the 35,000 conservatively diagnosed and documented patients are at the centers versus your strategy in the community setting? And how much that drove the sort of expansion of the rep number that you mentioned? And then I've got a follow-up about your hub. Wesley Kaupinen: Great. Ritu, thanks for the questions. To address your question on where the reps will be focused, we think of our market as 3 tiers. That first tier is about 400 centers. Those 400 centers, we estimate, based on claims data, have about 15,000 or more MLM patients under their management. Of those 400 centers, we'd say about half of those are going to be vascular anomaly centers. There's an excellent publication from Dr. Sally Cohen-Cutler that talks about the emergence of vascular anomaly centers from a few years ago, and we've been able to leverage that publication. Reps will also, in addition to that, what we'll call the Tier 1, which is the high-volume centers, we will also have personal promotion with the reps into our Tier 2 and our Tier 3. So all segments of the market will receive personal promotion. I mentioned Peter Finlayson earlier on this call. Peter has a lot of experience in digital marketing. He's brought on 2 new hires who have just started, who are both very impressive. And so we are going to be deploying digital marketing approaches across not only that Tier 1 of 400 centers, but also the Tier 2 and the Tier 3. In addition to personal promotion through the reps, we expect to be building an inside sales team. This is an approach and strategy that Ashley had at Dompe through the launch of Oxervate that she has described as a high return on investment activity in an orphan launch. And so under Kent Taylor's leadership, we're starting to assemble that team as well. I think Matt covered it with his comments, which is just to say, we're very well resourced for the launch. We continue to be disciplined in our capital allocation. But by deploying more reps at launch, a slightly larger medical team and a very strong marketing team, we think that sets us up for early launch success. Ritu Baral: Great. And then on the hub and specifically reimbursement support plans that you have, what's the size of the force in the hub that you currently plan on being available to patients and your practices to help? And as you think about your pricing and your first insurance conversations, do you have a sort of list of likely suspects for either prior authorizations or potentially even, obviously, unapproved step-throughs that you think insurance may utilize? Wesley Kaupinen: Yes. Thanks for the question. So we've just recently brought on our leader for the patient services team. His name is Matt Giordano. Matt was previously at Krystal Biotech. He's working closely with Jennifer McDonough, who was also previously at Krystal. We're in the process of ensuring that team is appropriately sized, similar to our guidance of 20 to 40 reps and how we landed on 40. Our internal thinking is to make sure that team is resourced at the high end of the range. So we look forward to coming back with specifics on the size of that team. On your second question, our payer research, thanks for flagging that question. We mentioned our payer research. We tested for that, Ritu. We would not expect at this point in time to have step-throughs of unapproved therapies. When there is the presence, if we're approved, of a drug that has 95% efficacy in Phase III and is taking this on-target, addressing the causal mTOR pathway and in-tissue, doing it in the skin approach. So we don't anticipate that based on our recent payer research. Ritu Baral: Would prior auths really just be diagnosis? Wesley Kaupinen: Yes, we'll have some of that research that we're continuing to do. Oftentimes, payers in rare diseases can request prior auths. The key is to have that mapped out and have your patient access team and payer team be able to seamlessly navigate those prior auths. And I think there's a lot of precedent from the 3 precedents we mentioned, TEPEZZA, VYJUVEK and Oxervate, that we can model. Operator: Our next question comes from Annabel Samimy at Stifel. Annabel Samimy: Congratulations on the progress. So you talked a lot about the MLM population size. Have you done the same for cVM? And what are the prospects for orphan designation for that indication? Is cVM a lot larger than the 75,000 that you've cited? And then separately, for MLM, I know that you have an OLE study ongoing. Is any of that data needed for completion of the filing? What can we expect as far as data trickling out from that study and just additional data releases through the year? Wesley Kaupinen: Annabel, thanks for the questions. I really appreciate you asking about the size of the cVM market. What I've found from my time at Insmed and Palvella is that what's in the literature is generally unreliable in terms of estimating epi. So we take data-driven approaches through real-world occurrence studies, through claims analysis to really appropriately size these markets. There's a recent publication in Orphanet Journal of Rare Diseases with Jack Gallagher as the first author that estimates that there's 135,000 cutaneous venous malformation patients in the United States. Again, applying some conservatism in our corporate deck, we talk about greater than 75,000. What we do know about venous malformations is that it is the most common type of vascular malformation, more common than microcystic lymphatic malformations, for example, or more common than other forms of vascular malformations. Your question around orphan designation, we do intend to pursue orphan designation for that indication. And then I'll pass it over to Jeff to talk about the OLE data and what will be incorporated in the filing as well as some of the additional opportunities to share data from the SELVA study. Jeffrey Martini: Yes. Thank you, Annabel, for the question. Yes, we do have the ongoing open-label extension study as part of SELVA. So the patients that completed efficacy had the opportunity to stay on drug, and they remain on drug at this time. And we are going to be planning to submit a data cut from that as part of our safety update to the FDA after the original NDA goes in. So we're actively planning that now. We are having a large medical affairs and medical congress presence this summer and next year, we're planning all those activities now. So we continue to do new data cuts, continue to have different ways we're analyzing data, including some of the long-term safety data and PK and other data will be coming out at future medical congresses. Annabel Samimy: Got it. And if I could just ask for a follow-up on cVM. What are your expectations at this point for what a Phase III trial design will look like? And if you have to have a placebo-controlled arm, what are your prospects for enrollment now that the data is out and they see that this is a very effective drug? Wesley Kaupinen: Yes. Thanks for that question. We're meeting with the FDA. We plan to meet with them in the coming months here to have an End of Phase II meeting and to align on a Phase III study design. Annabel, I think whether that ends up being a placebo-controlled study or a non-placebo-controlled study, based on all the analysis we've done of the Phase II data, including some of these patient qualitative interviews that Jeff referenced, we think that we will demonstrate a robust and strong treatment effect of QTORIN rapamycin based, again, on the Phase II results, but also the acceptance of rapamycin/sirolimus as a targeted therapy addressing the underlying mTOR driver for these venous malformations. We expect to have FDA approval, based on the internal modeling that we've done of various study designs, Annabel, in the 2029 time frame for cutaneous venous malformations. Operator: Our next question comes from Graig Suvannavejh at Mizuho. Ryan Ries: This is Ryan on for Graig today. Maybe just the first question, focusing on angiokeratomas, maybe for Jeff. Can you talk a little bit how LOTU is coming along and maybe talk a little bit about some of the overlap in both the etiology and the symptomology in angiokeratomas relative to the more advanced programs in MLM and cVMs? And then maybe just as a second question for Wes. What's your sense of investor interest in the angiokeratoma program so far and the level of awareness that investors have regarding overlap with these other conditions? Jeffrey Martini: Thanks for the question, Ryan. This is Jeff. So the status is we've started the study. We started earlier than originally planned. It's gone really well. I've had the opportunity to train all the clinicians on the study design and the endpoints. And I could say, anecdotally, there's a lot of enthusiasm for this trial. There's a lot of unmet need, and they are seeing these patients in their clinic, and they're not wanting to do some of the destructive procedures that I've talked about. They're destructive, they're painful and the disease often comes back. And that kind of goes into the second part of your question about the symptoms and the overlap. We started the program in angiokeratomas because of the unmet need, but also because of the fact that there's a lot of biological and clinical overlap with the microcystic lymphatic malformations program. Angiokeratomas are a type of isolated lymphatic malformation. They have some of the same molecular markers. There are some differences with microcystic lymphatic malformations. There's -- bleeding is much more common in angiokeratomas. But overall, very, very symptomatic disease, significant unmet need, and we're seeing a lot of investigator interest as well as patient interest in the trial. Wesley Kaupinen: Yes, Ryan, thanks for both your questions. I'd say the level of awareness of these rare diseases that have no approved therapies is generally low. That's been my experience both at Palvella and at Insmed. I think where you start to see the level of awareness rise is when you run these studies, particularly Phase II studies. And if you're fortunate enough, like we've been fortunate in microcystic LMs and cVMs, to demonstrate a strong treatment effect, I think investor awareness rises over time. We do like to use the opportunity on these earnings calls to educate. Jeff did a great job, I thought, walking through the 3 papers in angiokeratomas and also the quotes that he had for patients who are interested in the porokeratosis program. So it's incumbent upon us to drive this disease state awareness with all of our stakeholders, but also execute these studies on a timely basis with urgency, advance our therapies, get them to patients who currently have nothing. Ryan Ries: And then maybe just as a last question for me. Can you talk about the pursuit of the platform designation for QTORIN? Like what sort of data package you're going to put together for that? And how is that going to benefit both the ongoing programs and the programs that you plan to announce here? Wesley Kaupinen: Yes. Thanks, Ryan, for the question. We've followed others who have secured this FDA's Platform Designation. And similar to some of these other designations that we've secured, our interpretation is that the Platform Designation can serve to expedite therapies to patients. So Platform Designation should be available to Palvella in terms of submitting an application after our first approval for QTORIN rapamycin in microcystic lymphatic malformations. We believe the beneficiary of the Platform Designation would be future QTORIN product candidates such as QTORIN pitavastatin as well as the third product candidate that we're going to announce later this year. So we'll exit this year with QTORIN rapamycin, QTORIN pitavastatin and a third product candidate. Each of those formulations have similar characteristics in terms of the anhydrous gel base, in terms of some of the excipients, release characteristics, penetration characteristics. So we're excited to secure that first FDA approval in the first half of next year and then have a collaborative dialogue with the FDA about our eligibility for a Platform Designation. Operator: Our next question comes from Ryan Deschner at Raymond James. Ryan Deschner: Two quick questions for me. One, have your expectations for what a potential label might look like in MLM evolved since your pre-NDA meeting with FDA in terms of age cutoff or otherwise? And did regulators cite specific areas from your initial cVM data package that need to be addressed or strengthened with more mature data in order to be granted or reconsidered for Breakthrough Designation? Wesley Kaupinen: Yes. Thanks for the questions, Ryan. No changes in the label conversations as a result of the pre-NDA meeting. We're going to pursue a broad label for microcystic lymphatic malformations, and we believe that should include patients at pediatric ages. We think that's best for patients, and we think we have strong data to support that as part of our NDA data package. In terms of your question on the data package for cutaneous venous malformations, I think Jeff highlighted it nicely earlier, which is we'd like to submit more patient experience data. We'd like to submit patient interview transcripts. We think those will be additive to the cVM data package. They help regulatory agencies interpret the effect sizes and what those effect sizes really meant to patients. So this was a smart approach that Jeff implemented to do these qualitative interviews to understand disease burden at baseline, but also understand whether there was a change in disease burden following 12 weeks of therapy. So those will be core to a future breakthrough resubmission package as well as that 24-week data, which Jim Treat presented at ISSVA and Jeff highlighted on this call. Operator: Our next question comes from Sam Slutsky at LifeSci Capital. Samuel Slutsky: Just real quick, any updates on how you're thinking about pricing analogs for MLMs? And then can you just remind us on kind of the extended body surface area in MLM patients and kind of expectations for tube size and what it could cover, et cetera? Wesley Kaupinen: Yes. Sam, thanks for the question. On pricing analogs, we have 3 of those listed in our corporate deck: TEPEZZA, Oxervate and ARIKAYCE. We guided to a pricing range of $100,000 to $200,000 per patient per year in microcystic lymphatic malformations. I mentioned that we've done recent payer research. We can confirm that we would expect to have strong payer coverage in those pricing ranges of $100,000 to $200,000 per patient per year, and we'll come back to the market closer to the time of FDA approval with our launch price. On your second question, will pass it over to Jeff. Jeffrey Martini: Thanks, Sam, for the question on BSA and tube size. So microcystic lymphatic malformations are caused by somatic mutations in PIK3CA, which lead to mTOR over-activating and driving the disease. And because they're somatic in nature, they tend to be very localized in nature, usually in areas of high lymphatic density, often in the trunk or the groin area. As a result, the size of them is usually between 9 cm2 and 200 cm2 are the majority of patients with lymphatic malformations. They can be larger, but that's less common. So we've typically dosed the patients according to lesion size and not BSA, although we do have that data. But for lesion size, one actuation of our pump is enough to cover up to 200 cm2. So the product will be provided in a pump, which is enough to cover one actuation of the pump for a 30-day supply. Operator: Our next question comes from Danielle Brill at Truist. Unknown Analyst: This is Alex on for Danielle. Question on the upcoming End of Phase II in cVM. Based on your experience with MLM, how does the presence of Breakthrough Designation impact the content and the tone of the End of Phase II meeting? Specifically how the FDA approaches whether or not a placebo arm is necessary? Just curious if the lack of Breakthrough Designation changes your calculus for how you approach the upcoming End of Phase II meeting. Wesley Kaupinen: Yes, Alex, thanks for the question. The absence of Breakthrough does not impact how we think about the End of Phase II meeting. We have a drug that in Phase II had a large effect size in a serious rare progressive disease where there's no FDA-approved therapies. I think one of the keys for the End of Phase II meeting in addition to stepping through that data and some of the newer data that Jeff has aggregated that the FDA hasn't seen is for the FDA to have an exchange with our key opinion leaders who treat these patients today and be able to hear their input on what they think is the most appropriate study design for a Phase III study. We do know, thanks to the [ Fujino ] publication out of Japan, that there is no documented spontaneous progression in this disease. And so that will be a key point of discussion for our regulatory interactions. And as you and others have gathered, we have a very collaborative relationship with the agency. We're grateful in MLM for Breakthrough, Fast Track, Orphan Designation, Orphan Product Grant. We have Fast Track in cVM and angiokeratomas. So we're looking forward to working collaboratively to align on the right study design that efficiently brings this drug to patients. Operator: This concludes the question-and-answer session. I would now like to turn it back to Wes Kaupinen for closing remarks. Wesley Kaupinen: Great. Thank you, operator, and thank you to everyone for your participation on today's call and for your continued strong interest in what we're building at Palvella. We look forward to updating you on our continued progress as we work to bring first-in-disease therapies to patients living with serious rare skin diseases and vascular malformations. Operator, you may now conclude the call. Operator: Thank you for your participation in today's conference. This does conclude the program. You may now disconnect. 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First module of the rolling NDA for QTORIN™ rapamycin for microcystic lymphatic malformations submitted to FDA, with completion of the NDA submission on track for the second half of 2026 Preparing for a planned standalone U.S. commercial launch of QTORIN™ rapamycin for microcystic lymphatic malformations in the first half of 2027, if approved Initiation of Phase 3 trial of QTORIN™ rapamycin for the treatment of cutaneous venous malformations planned for the fourth quarter of 2026 Initiation of Phase 2 trial of QTORIN™ pitavastatin for the treatment of disseminated superficial actinic porokeratosis planned for the second half of 2026 Topline results from the Phase 2 LOTU trial of QTORIN™ rapamycin for clinically significant angiokeratomas expected in the second half of 2027 Cash, cash equivalents and short-term investments of $250.6 million as of June 30, 2026 Company to host conference call at 8:30 a.m. ET today WAYNE, Pa., Aug. 04, 2026 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc. (Palvella or “the Company”) (Nasdaq: PVLA), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies for serious, rare skin diseases and vascular malformations for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today reported financial results for the second quarter ending June 30, 2026 and provided a corporate update. “We made significant progress during the second quarter, completing our pre-NDA meeting with FDA and initiating the rolling NDA submission for QTORIN™ rapamycin in microcystic lymphatic malformations,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “We are working closely with FDA under the program’s Breakthrough Therapy and Fast Track designations to expedite development and review, with the objective of potentially introducing the first approved therapy to pediatric and adult patients living with this serious, lifelong disease. Completion of the NDA submission remains on track for the second half of 2026, and we are preparing for a planned standalone commercial launch in the first half of 2027, if approved. We have recruited commercial and medical affairs leaders with deep experience in rare disease and dermatology who are now in the field executing key pre-launch activities, while we continue to advance our other rare disease programs and pursue additional opportunities across the QTORIN™ platform.” Recent Research and Development Highlights QTORIN™ rapamycin for microcystic lymphatic malformations (microcystic LMs) James Treat, M.D., of Children’s Hospital of Philadelphia presented additional Phase 3 SELVA data during a late-breaking session at the International Society for the Study of Vascular Anomalies World Congress (ISSVA), including a statistically significant improvement in the 6–11-year-old cohort and other supportive findings showing improvements in clinical signs and patient-reported outcomes with QTORIN™ rapamycin. Completed an in-person pre-New Drug Application (NDA) meeting with FDA which addressed nonclinical, clinical pharmacology, clinical information, and the planned evidence package for the NDA. Following the pre-NDA meeting, FDA granted Palvella’s request for Rolling Review of the QTORIN™ rapamycin NDA for microcystic LMs, allowing the Agency to begin reviewing completed portions of the application before submission of the full NDA. Submitted the first module of its rolling NDA to the FDA seeking approval of QTORIN™ rapamycin for the treatment of microcystic LMs. The Company remains on track to complete the NDA submission in the second half of 2026. QTORIN™ rapamycin for cutaneous venous malformations (cutaneous VMs) In May 2026, at the 83rd Annual Meeting of the Society for Investigative Dermatology (“SID”), Palvella presented new data from our Phase 2 TOIVA trial of QTORIN™ rapamycin for the treatment of cutaneous VMs highlighting that 100% of patients with bleeding at baseline demonstrated improvement on the Cutaneous Venous Malformations Investigator Global Assessment Bleeding scale at Week 12. Dr. Treat presented additional Phase 2 TOIVA data, including 24-week results, at ISSVA demonstrating statistically significant improvements in both cVM-MCSS Height/Engorgement and cVM-MCSS Appearance at all measured time points, with increasing clinical response observed with longer duration of QTORIN™ rapamycin therapy. Phase 3 trial initiation remains on track for the fourth quarter of 2026 following completion of the planned End-of-Phase 2 meeting. QTORIN™ rapamycin for clinically significant angiokeratomas Dosed the first patients in LOTU, a multicenter Phase 2 trial evaluating Fast Track-designated QTORIN™ rapamycin for clinically significant angiokeratomas, a rare, chronic and debilitating isolated lymphatic malformation affecting an estimated more than 50,000 diagnosed patients in the U.S. and for which there are no FDA-approved therapies. Topline results from LOTU are expected in the second half of 2027. QTORIN™ pitavastatin for disseminated superficial actinic porokeratosis (DSAP) Palvella's second product candidate, QTORIN™ pitavastatin, is for the treatment of disseminated superficial actinic porokeratosis, a premalignant genetic skin disease that presents as persistent, often extensive lesions that enlarge and increase in size, number, and extent over time, causing chronic loss of skin integrity which can severely impact quality-of-life; no FDA-approved therapies currently exist for the estimated more than 50,000 diagnosed patients in the U.S. Strengthened the intellectual property position supporting QTORIN™ pitavastatin through the issuance of U.S. Patent No. 12,636,273, exclusively licensed from Yale University and building on pioneering work by Keith Choate, M.D., Ph.D. The issued claims cover the topical administration of HMG-CoA reductase inhibitors, including pitavastatin, for the treatment of porokeratosis, including DSAP, and provide protection into 2043. Phase 2 trial initiation expected in the second half of 2026. QTORIN™ rapamycin and QTORIN™ platform expansion Palvella plans to announce the fourth target clinical indication for QTORIN™ rapamycin in the second half of 2026. The expansion of QTORIN™ rapamycin into additional indications is supported by a growing body of published literature highlighting the broad potential of rapamycin in several difficult-to-treat, mTOR-driven skin diseases while advocating for targeted, topical approaches suited to improve tolerability and safety. Palvella plans to announce the third product candidate from the QTORIN™ platform in a serious, rare disease with no FDA-approved therapies in the second half of 2026. Recent Corporate Highlights Appointed accomplished rare disease biotech executive and commercial leader Matt Pauls, J.D., M.B.A., to the Board of Directors, further strengthening the Board with extensive experience in rare disease drug development, commercialization, and corporate strategy from executive and Board roles at Savara Inc., Soleno Therapeutics, Strongbridge Biopharma, and Insmed Incorporated. Awarded “Healthcare & Life Sciences Company of the Year” at the 2026 Philadelphia Alliance for Capital and Technology Ecosystem Awards, recognizing Palvella's leadership in advancing innovative therapies for rare diseases and its contributions to the region's life sciences ecosystem. Completed the uplisting to the Nasdaq Global Market, providing increased visibility within the investment community and reflecting the Company’s continued growth and achievement of key corporate milestones. Second Quarter 2026 Financial Results Cash, cash equivalents, and short-term investments as of June 30, 2026 were $250.6 million. Research and development expenses for the three months ended June 30, 2026 were $12.5 million, as compared to $5.1 million for the three months ended June 30, 2025. The increase was primarily due to increased spending for manufacturing activities, clinical development of QTORIN rapamycin for the treatment of angiokeratomas, costs associated with the submission of the first module of the rolling NDA, and costs resulting from increased headcount and consulting services in 2026. General and administrative expenses for the three months ended June 30, 2026 were $8.9 million, as compared to $4.1 million for the three months ended June 30, 2025. The increase was primarily due to increased headcount in 2026, as well as increased professional services related to operating as a publicly-traded company. Net loss was $21.9 million, or $1.52 per basic and diluted share, for the three months ended June 30, 2026, as compared to net loss of $9.5 million, or $0.86 per basic and diluted share, for the three months ended June 30, 2025. Weighted average shares outstanding for calculation of EPS in Q2 2026 and YTD 2026 were 14,356,219 and 13,724,256 respectively. Shares outstanding were 15,802,768 as of July 31, 2026, including 14,408,007 shares of common stock and 1,394,761 common share equivalents assuming conversion of outstanding pre-funded warrants. Conference Call Details Palvella will host a conference call and live audiovisual webcast to discuss the Company's second quarter 2026 financial results and provide a corporate update at 8:30 a.m. ET today. To access the live webcast, including presentation slides, please click here or visit the “Events & Presentations” section of Palvella’s website. To access the conference call by phone, register using this link, and you will be provided with dial-in details. A replay of the webcast will be available approximately two hours after the conclusion of the call and will remain archived for 90 days under the “Events & Presentations” section of the Company's website at www.palvellatx.com. About Palvella Therapeutics Founded and led by rare disease biotech veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients living with serious, rare skin diseases and vascular malformations for which there are no FDA-approved therapies. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare skin diseases and vascular malformations, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being developed for the treatment of microcystic lymphatic malformations, cutaneous venous malformations, and clinically significant angiokeratomas. Palvella’s second product candidate, QTORIN™ pitavastatin, is currently being developed for the treatment of disseminated superficial actinic porokeratosis. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter). QTORIN™ rapamycin and QTORIN™ pitavastatin are for investigational use only and neither has been approved by the FDA or by any other regulatory agency for any indication. Forward-Looking Statements This press release contains forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended (Securities Act)). These statements may discuss goals, intentions, and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the management of Palvella, as well as assumptions made by, and information currently available to, the management of Palvella. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Statements that are not historical facts are forward-looking statements. Forward-looking statements include, but are not limited to, statements regarding the expected timing of the presentation of data from clinical trials, Palvella’s clinical development plans and related anticipated development milestones and anticipated timing of regulatory submissions, Palvella’s plans with respect to the timing of, and anticipated FDA review process for, the NDA for QTORIN™ rapamycin, Palvella’s plans to pursue Breakthrough Therapy Designation, Palvella’s plans to meet with regulatory authorities, Palvella’s expectations regarding the benefits of orphan drug designation and potential benefit of orphan drug exclusivity for QTORIN™ rapamycin for the treatment of microcystic lymphatic malformations, Palvella’s cash, financial resources and expected runway, Palvella’s expectations regarding its programs, including QTORIN™ rapamycin and QTORIN™ pitavastatin, and its research-stage opportunities, including its expected therapeutic potential and market opportunity. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the ability to raise additional capital to finance operations; the ability to advance product candidates through preclinical and clinical development; the ability to make regulatory submissions on anticipated timelines; the ability to obtain regulatory approval for, and ultimately commercialize, Palvella’s product candidates, including QTORIN™ rapamycin and QTORIN™ pitavastatin; the outcome of early clinical trials for Palvella’s product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; the fact that data and results from clinical studies may not necessarily be indicative of future results; Palvella’s limited experience in designing clinical trials and lack of experience in conducting clinical trials; Palvella’s limited experience in commercial manufacturing; the ability to identify and pivot to other programs, product candidates, or indications that may be more profitable or successful than Palvella’s current product candidates; the substantial competition Palvella faces in discovering, developing, or commercializing products; the negative impacts of global events on operations, including ongoing and planned clinical trials and ongoing and planned preclinical studies; the ability to attract, hire, and retain skilled executive officers and employees; the ability of Palvella to protect its intellectual property and proprietary technologies; reliance on third parties, contract manufacturers, and contract research organizations; and the risks and uncertainties described in the filings made by Palvella with the Securities and Exchange Commission (SEC), including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties that Palvella may face. Except as required by applicable law, Palvella does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release. Contact Information InvestorsWesley H. KaupinenFounder and CEO, Palvella [email protected] NanusVice President of Investor Relations and Corporate AffairsPalvella [email protected]

Interested in Palvella Therapeutics, Inc.? Here are five stocks we like better. Palvella remains on track to submit its NDA for QTORIN rapamycin in microcystic lymphatic malformations in the second half of 2026, with potential FDA approval targeted for the first half of 2027. The application is being filed under the 505(b)(2) pathway and has received rolling review. The Phase III SELVA study met its primary and key secondary endpoints, with 95% of patients showing improvement at week 24; all 13 children in a reported subgroup were considered much or very much improved. Palvella is preparing for a potential launch by expanding its sales force to about 40 representatives and engaging its target clinics. Palvella ended Q2 with approximately $251 million in cash, which it expects to support operations through a potential launch and pipeline development. The company increased projected 2026 cash expenses to $85 million-$95 million and is advancing QTORIN rapamycin programs in venous malformations and angiokeratomas, alongside a planned QTORIN pitavastatin study in DSAP. Palvella Therapeutics (NASDAQ:PVLA) said it remains on track to complete its new drug application submission for QTORIN rapamycin in microcystic lymphatic malformations, or mLM, during the second half of 2026, with potential FDA approval targeted for the first half of 2027. The company has submitted the first module of its NDA after the FDA granted rolling review, which allows the agency to review completed sections before the full application is filed. Palvella is seeking approval through the 505(b)(2) pathway and said its application will include data from its Phase III SELVA study, Phase II study and real-world clinical evidence. → SpaceX’s First Earnings Report Could Decide Whether Shorts or Bulls Have Control Founder and Chief Executive Officer Wes Kaupinen said QTORIN rapamycin has received Breakthrough Therapy, Fast Track and Orphan Drug designations, as well as an FDA Orphan Product Grant, for the mLM program. The company intends to pursue a broad label that includes pediatric patients. Palvella highlighted previously reported Phase III SELVA results in mLM, a rare vascular malformation that the company said has no FDA-approved treatments. Kaupinen said the study met its primary endpoint, key secondary endpoint and four prespecified secondary endpoints, with 95% of patients demonstrating improvement on the primary endpoint at week 24. → Financials Hit Record Highs as the AI Trade Unravels—Can They Keep Leading? Chief Scientific Officer Jeff Martini discussed a subgroup analysis of 13 children ages 6 to 11 presented at the ISPOR World Congress. At week 24, the mean improvement on the mLM Investigator Global Assessment was 2.46 points, and all children were assessed as either much improved or very much improved, according to Martini. SELVA included an eight-week untreated run-in period. During that period, disease severity was essentially unchanged, with a mean mLM-MCSS change of negative 0.1 points. After 24 weeks of treatment, the blinded mean mLM-MCSS improved by 3.4 points, representing 48% of the maximum potential improvement from baseline, Martini said. → Why Rare Earth Processing Could Be the Real 2027 Opportunity Palvella is building its commercial infrastructure ahead of a potential launch. The company has recruited leaders across commercial and medical affairs and increased its expected launch sales force to about 40 representatives, at the upper end of its prior guidance. It has already engaged more than 200 of its initial 400 targeted clinics, Kaupinen said. The company is also internalizing core patient-support services, including functions related to access, reimbursement and treatment initiation. In response to analyst questions, Kaupinen said Palvella’s payer research did not suggest insurers would require patients to try unapproved therapies before accessing QTORIN rapamycin, if it is approved. He said prior authorization requirements are possible in rare diseases. Beyond mLM, Palvella is advancing QTORIN rapamycin for cutaneous venous malformations, or cVM, and clinically significant angiokeratomas. Both programs have Fast Track Designation from the FDA. For cVM, the company plans to meet with the FDA in coming months for an end-of-Phase-II meeting to review Phase II TOIVA results and finalize a pivotal-study design. Palvella expects to initiate its Phase III cVM trial in the fourth quarter of 2026 and continues to target potential approval in 2029. TOIVA showed that 73% of patients improved on the cVM Investigator Global Assessment, exceeding the company’s predefined success threshold, according to Martini. The FDA did not grant Palvella’s initial Breakthrough Therapy Designation request, which had included 12-week data. The company plans to discuss its complete 24-week efficacy data and qualitative patient-report findings with the agency, and believes those materials could support a later resubmission for the designation after Phase III initiation. The angiokeratoma Phase II LOTU study began dosing patients in April and is designed to enroll up to 15 patients. Palvella expects to report data in the second half of 2027. The company said it anticipates the program could follow a supplemental NDA pathway after an initial QTORIN rapamycin approval. Palvella also expects to start a Phase II study of QTORIN pitavastatin in disseminated superficial actinic porokeratosis, or DSAP, during the fourth quarter of 2026. Kaupinen said the planned study is expected to enroll about 15 patients. The company said it would provide additional guidance on the DSAP data-readout timeline closer to trial initiation. Kaupinen said Palvella expects to announce a fourth QTORIN rapamycin indication later in 2026. The company estimates that QTORIN rapamycin could potentially address more than 300,000 U.S. patients across multiple mTOR-driven indications under its current development plans, compared with more than 30,000 estimated diagnosed mLM patients. Chief Financial Officer Matt Korenberg said Palvella ended the second quarter with approximately $251 million in cash. The balance sheet was bolstered by a $230 million financing completed in February, exceeding the company’s original $150 million fundraising objective. Palvella said the cash balance is expected to support operations through a potential FDA approval and standalone commercial launch of QTORIN rapamycin, if approved, while also funding the company’s pipeline programs. As it expands spending on commercial launch readiness, medical affairs, marketing and disease-awareness initiatives, the company now expects 2026 cash expenses of approximately $85 million to $95 million. Korenberg said the increased spending reflects a larger planned field force, earlier hiring of medical science liaisons and greater pipeline investment. Regarding pricing, Kaupinen reiterated Palvella’s prior guidance for a potential annual QTORIN rapamycin price of $100,000 to $200,000 per patient in mLM. He said company payer research indicated support for coverage within that range, though Palvella plans to disclose a launch price closer to a potential approval date. Palvella Therapeutics, Inc (NASDAQ: PVLA) is a clinical‐stage biopharmaceutical company devoted to the discovery and development of innovative therapies for immunological and inflammatory diseases. The company employs a proprietary small‐molecule and biologics platform to identify and modulate key molecular pathways that drive neutrophil‐ and complement‐mediated inflammation, aiming to deliver targeted treatment options for patients with significant unmet medical needs. Palvella's pipeline comprises several preclinical assets designed to address both prevalent chronic inflammatory conditions and rare autoinflammatory syndromes. 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 "Palvella Therapeutics Q2 Earnings Call Highlights" was originally published by MarketBeat. 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This article first appeared on GuruFocus. Cash Position: Approximately $251 million in cash as of June 30, 2026. 2026 Cash Expenses Guidance: Expected to be approximately $85 to $95 million. Capital Raise: Raised $230 million in a February financing, exceeding the original $150 million target. Planned Sales Force: Increased to approximately 40 sales representatives at launch. Warning! GuruFocus has detected 2 Warning Sign with PVLA. Is PVLA fairly valued? Test your thesis with our free DCF calculator. Release Date: August 04, 2026 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Palvella Therapeutics Inc (NASDAQ:PVLA) reported highly compelling Phase 3 SELVA study results for QTORIN rapamycin in microcystic lymphatic malformations (LMs), meeting its primary endpoint, key secondary endpoint, and all four pre-specified secondary endpoints with high statistical significance, including 95% of patients showing improvement at week 24. The company has achieved significant regulatory milestones, including a successful pre-NDA meeting with the FDA, the granting of rolling review for its NDA, and the submission of the first module of the application, with the full submission on track for the second half of 2026 and potential FDA approval in the first half of 2027. Palvella Therapeutics Inc (NASDAQ:PVLA) has a strong financial position with approximately $251 million in cash, following a $230 million capital raise, which provides sufficient capital to fund operations through a potential FDA approval and a standalone commercial launch. The company is executing a 'pipeline-in-a-product' strategy for QTORIN rapamycin, expanding into additional indications like cutaneous venous malformations (CVMs) and clinically significant angiokeratomas, which could expand the addressable US patient population from over 30,000 to more than 300,000 patients. Palvella Therapeutics Inc (NASDAQ:PVLA) has made significant progress in launch readiness, including recruiting an experienced commercial and medical affairs leadership team, engaging over 200 of its initial 400 target clinics, and building an internal patient services organization to support patient access and treatment initiation. The Phase 2 TOIVA study in cutaneous venous malformations demonstrated that 73% of patients improved on the CVM-IGA, more than twice the predefined success threshold, supporting the advancement of the program into a Phase 3 study, which is on track to initiate in the fourth quarter of 2026. Physician market research indicates strong interest in QTORIN rapamycin, with more than 80% of surveyed physicians considering it as a first-line therapy across all four indications if approved, and 96% of physicians indicating they would incorporate it into their practice for angiokeratomas. Palvella Therapeutics Inc (NASDAQ:PVLA) has not yet received FDA approval for QTORIN rapamycin, and the company remains subject to regulatory risks and uncertainties associated with the NDA review process, with approval only anticipated in the first half of 2027. The FDA did not grant breakthrough therapy designation for the cutaneous venous malformations (CVM) program based on the initial 12-week data package, although the company plans to resubmit with the complete 24-week data set. The company's pipeline is heavily reliant on the success of QTORIN rapamycin, and any delays or failures in the ongoing or planned clinical trials for other indications, such as CVM, angiokeratomas, or DSAP, could negatively impact the company's growth prospects. Palvella Therapeutics Inc (NASDAQ:PVLA) expects its 2026 cash expenses to increase to approximately $85-95 million due to expanded commercial and medical affairs investments, which could increase the company's cash burn rate and potentially require additional financing in the future. The company faces significant commercial execution risks, including the need to successfully launch a first-in-disease therapy in a rare disease market with limited awareness, and the potential for challenges in patient identification, market access, and reimbursement despite favorable payer research. The company's estimates of patient populations and market sizes are based on its own data-driven epidemiological work, which may be subject to inaccuracies or revisions, and the actual addressable market could be smaller than projected. Palvella Therapeutics Inc (NASDAQ:PVLA) is developing therapies for diseases with no FDA-approved treatments, which means there is no established market precedent, and the company may face challenges in setting pricing and securing favorable reimbursement for its products. Q: Can you elaborate on the ongoing efforts to identify MLM patients, and are recent findings in line with the initial estimates of around 30,000 diagnosed patients? A: Wesley Kaupinen (CEO): We can confirm that our recent findings are in line with previous estimates. Last year, we published a claims analysis indicating between 45,000 and 95,000 diagnosed MLM patients in the US, with an estimated annual incidence of 1,500 or more newly diagnosed patients. We like to be conservative, so we confirm there are greater than 30,000 diagnosed patients in the US. In terms of patient identification, we know where these patients are concentrated, particularly at vascular anomaly centers that have emerged over the last 20 years. We are making efforts to be in front of those physicians at their sites and through a strong presence at medical congresses. Q: What are your expectations for a potential label for MLM following the pre-NDA meeting, and what specific areas from the initial CVM data package need to be strengthened for a breakthrough therapy designation resubmission? A: Wesley Kaupinen (CEO): There were no changes in label conversations as a result of the pre-NDA meeting. We are pursuing a broad label for microcystic lymphatic malformations that should include pediatric patients, as we have strong data to support that. For the CVM data package, we plan to submit more patient experience data and patient interview transcripts, which will be additive. These qualitative interviews help regulatory agencies interpret the effect sizes and what they meant to patients. The 24-week data presented at ISFA will also be core to a future breakthrough therapy designation resubmission package. Q: Can you discuss the deployment strategy of the 40 sales reps across vascular anomaly clinics, and what percentage of the diagnosed patients are at these centers versus the community setting? A: Wesley Kaupinen (CEO): We think of our market as 3 tiers. The first tier is about 400 centers, which we estimate have about 15,000 or more MLM patients under their management based on claims data. About half of those are vascular anomaly centers. Reps will also provide personal promotion into our tier 2 and tier 3 segments, so all segments of the market will receive personal promotion. We are also deploying general marketing approaches and building an inside sales team, an approach that has been described as a high return on investment activity in an orphan launch. Q: What are the prospects for orphan designation for the CVM indication, and is CVM a lot larger than the 75,000 cited? Also, is any of the OLE data needed for completion of the MLM filing? A: Wesley Kaupinen (CEO) & Jeff Martini (CSO): There is a recent publication in the Orphanet Journal of Rare Diseases estimating 135,000 cutaneous venous malformation patients in the US. Applying conservatism, we talk about greater than 75,000. Venous malformations are the most common type of vascular malformation. We do intend to pursue orphan designation for that indication. Regarding the OLE study, we are planning to submit a data cut from it as part of our safety update to the FDA after the original NDA goes in. We are actively planning new data cuts and analyses for future medical congresses. Q: What are your expectations for the phase 3 trial design for CVM, and what are the prospects for enrollment given the positive phase 2 data? A: Wesley Kaupinen (CEO): We are meeting with the FDA in the coming months for an end of phase 2 meeting to finalize the phase 3 study design. Whether it ends up being placebo-controlled or not, we believe we will demonstrate a robust and strong treatment effect based on the phase 2 results and the acceptance of rapamycin as a targeted therapy addressing the underlying mTOR driver for venous malformations. We expect FDA approval in the 2029 timeframe for cutaneous venous malformations. Q: Can you talk about how the LOW-2 study for angiokeratomas is coming along, and what is the level of investor interest in the program? A: Jeff Martini (CSO) & Wesley Kaupinen (CEO): The study has started earlier than originally planned and is going really well. There is a lot of enthusiasm from clinicians who are seeing these patients and don't want to do destructive procedures. Angiokeratomas are a type of isolated lymphatic malformation with biological and clinical overlap with microcystic lymphatic malformations, though bleeding is much more common. The level of awareness of these rare diseases is generally low, but it rises when you run studies and demonstrate a strong treatment effect. It's incumbent upon us to drive disease state awareness with all stakeholders and execute these studies on a timely basis. Q: Can you talk about the pursuit of the platform designation for QTorn, and what data package will be put together for it? A: Jeff Martini (CSO): We have followed others who have secured the FDA's platform designation. Our interpretation is that it can serve to expedite therapies to patients. The designation should be available to Palvella after our first approval for ketor and rapamycin in microcystic lymphatic malformations. The beneficiaries would be future ketorin product candidates, such as ketorin pitavastatin and the third product candidate we will announce later this year. Each of these formulations has similar characteristics in terms of the anhydrous gel base, excipients, release characteristics, and penetration characteristics. Q: Have your expectations for a potential label in MLM evolved since the pre-NDA meeting, and what specific areas from the initial CVM data package need to be strengthened for breakthrough designation? A: Wesley Kaupinen (CEO): No changes in label conversations resulted from the pre-NDA meeting. We are pursuing a broad label for microcystic lymphatic malformations that should include pediatric patients. For the CVM data package, we plan to submit more patient experience data and patient interview transcripts, which will be additive. These qualitative interviews help regulatory agencies interpret the effect sizes and what they meant to patients. The 24-week data presented at ISFA will also be core to a future breakthrough therapy designation resubmission package. Q: Any updates on how you're thinking about pricing analogs for MLMs, and what is the extent of body surface area in MLM patients and expectations for tube size? A: Wesley Kaupinen (CEO) & Jeff Martini (CSO): We have 3 pricing analogs listed in our corporate deck: Tepezza, Oxervate, and Vyjavec. We For the complete transcript of the earnings call, please refer to the full earnings call transcript.

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. Achieved critical regulatory milestones for QTORIN rapamycin in microcystic lymphatic malformations (MLM), including a successful pre-NDA meeting and the initiation of a rolling NDA submission. Management attributes the strong Phase III SELVA results to the QTORIN platform's ability to deliver targeted, localized treatment directly to pathogenic tissue, addressing the causal mTOR pathway while minimizing systemic burden. Strategic focus remains exclusively on 'first-in-disease' therapies for rare skin conditions with no approved treatments, avoiding competitive markets with entrenched incumbents to maximize market share potential. Epidemiologic data suggests a multi-billion-dollar addressable market across four lead indications, with management emphasizing that these diseases are underappreciated rather than rare in clinical burden. Internalized patient support services to ensure tighter coordination across reimbursement and treatment initiation, leveraging leadership experience from previous successful orphan drug launches. Strengthened the balance sheet with a $230 million capital raise, providing the financial flexibility to invest in commercial readiness and accelerate the broader QTORIN platform pipeline. On track to complete the MLM NDA submission in the second half of 2026, targeting potential FDA approval in the first half of 2027. Planning to initiate the Phase III study for cutaneous venous malformations (cVM) in Q4 2026, with a target approval window in 2029. Expanded the planned launch sales force to 40 representatives to ensure comprehensive field coverage and physician education from day one of a potential MLM launch. Anticipate announcing a fourth rare disease indication for the QTORIN platform later this year, further diversifying the late-stage pipeline. Expect to seek FDA Platform Designation following the first approval, which management believes will expedite the regulatory path for future QTORIN product candidates. FDA did not grant Breakthrough Therapy Designation for the cVM indication based on initial 12-week data; management plans a resubmission using more robust 24-week efficacy and qualitative data. Increased 2026 cash spend guidance to $85 million - $95 million due to accelerated hiring of medical science liaisons and expanded commercial infrastructure. The 505(b)(2) regulatory pathway for QTORIN rapamycin allows the company to leverage existing FDA findings for rapamycin while maintaining a robust clinical evidence package for a broad label. Phase II LOTU study for clinically significant angiokeratomas is currently ahead of schedule, with data readout expected in the second half of 2027. One stock. Nvidia-level potential. 30M+ investors trust Moby to find it first. Get the pick. Tap here. Management confirmed their estimate of over 30,000 diagnosed U.S. patients is conservative, noting claims data suggests a range of 45,000 to 95,000. The strategy focuses on 400 high-volume centers, including specialized vascular anomaly clinics, where approximately 15,000 patients are already managed. Management stated the lack of designation does not change their clinical strategy or the End of Phase II meeting approach. They intend to present new qualitative patient interview data to the FDA to demonstrate the clinical significance of treatment effects beyond just physical measurements. Management guided to an orphan pricing range of $100,000 to $200,000 per patient per year, citing analogs like TEPEZZA and Oxervate. Payer research indicates strong support for this range given the lack of approved alternatives and the high efficacy demonstrated in Phase III trials. High patient inbound interest for the DSAP program suggests rapid enrollment potential for the Phase II study starting in Q4 2026. The angiokeratoma program is expected to follow a supplemental NDA pathway, providing a faster route to market expansion following the initial MLM approval.

WAYNE, Pa., July 28, 2026 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc. (Palvella or the “Company”) (Nasdaq: PVLA), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies for serious, rare skin diseases and vascular malformations for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, announced today that it will report its second quarter 2026 financial results before market open on Tuesday, August 4, 2026. Palvella management will host a conference call for investors at 8:30 a.m. ET on that same day to discuss the results and provide a corporate update. To access the live webcast, including presentation slides, please click here or visit the “Events & Presentations” section of Palvella’s website. To access the conference call by phone, register using this link, and you will be provided with dial-in details. A replay of the webcast will be available approximately two hours after the conclusion of the call and will remain archived for 90 days under the “Events & Presentations” section of the Company's website at www.palvellatx.com. About Palvella Therapeutics Founded and led by rare disease biotech veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients living with serious, rare skin diseases and vascular malformations for which there are no FDA-approved therapies. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare skin diseases and vascular malformations, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being developed for the treatment of microcystic lymphatic malformations, cutaneous venous malformations, and clinically significant angiokeratomas. Palvella’s second product candidate, QTORIN™ pitavastatin, is currently being developed for the treatment of disseminated superficial actinic porokeratosis. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter). QTORIN™ rapamycin and QTORIN™ pitavastatin are for investigational use only and neither has been approved by the FDA or by any other regulatory agency for any indication. Contact Information Investors Wesley H. Kaupinen Founder and Chief Executive Officer Palvella Therapeutics [email protected] Media Marcy Nanus Vice President of Investor Relations and Corporate Affairs Palvella Therapeutics [email protected]

By John Vandermosten, CFA NASDAQ: LGND READ THE FULL LGND RESEARCH REPORT Ligand Pharmaceuticals, Inc. (NASDAQ: LGND) reported first quarter 2026 results with revenues of $51.7 million and adjusted core earnings per share (EPS) of $1.63. Revenue growth of 14% generated a 23% EPS increase. By line item, royalties rose 56% while Captisol and Contract revenue fell. The big news since the prior financial update was the XOMA Royalty transaction, which adds over 100 new assets to the portfolio, including seven key royalty-generating assets. Other activity since the start of the year includes the approval of Filspari for focal segmental glomerulosclerosis (FSGS) and advancement of Palvella’s QTORIN rapamycin on several fronts, including an upcoming regulatory submission and start of two clinical trials. Additionally, Ligand gave notice to Viking Therapeutics regarding the TR-Beta program and remitted an additional $15 million to Orchestra Biomed, along with expanded clinical work in other portfolio assets. Along with the XOMA announcement on April 27th, Ligand raised its revenue and earnings guidance for 2026 and earnings guidance for 2027. 2026 revenue guidance was increased by $25 million to a range of $270 million to $310 million, and earnings per share were increased by $0.50 to a range of $8.50 to $9.50. For 2027, Ligand anticipates that XOMA revenues will add $1.50 to EPS. 1Q:26 Financial and Operational Results Ligand reported first quarter financial and operational results disclosed in a press release and Form 10-Q filing with the SEC on May 7th and 8th, respectively. A conference call was held with an accompanying presentation to discuss results with investors following the release. For the quarter ending March 31st, 2026, Ligand recognized revenues of $51.7 million. GAAP loss per share for 1Q:26 totaled $0.67, and adjusted core EPS was $1.63, with the primary difference related to a change in fair value of the Pelthos holdings. For 1Q:26 versus the same prior year period: Revenues of $51.7 million rose 14% from $45.3 million, driven by strong growth in royalties. Intangible royalties grew 53% to $32.9 million, and financial royalties grew 70% to $10.0 million. Captisol revenues were $8.7 million, falling 36%. Despite the decline, management has visibility into sales over the next year and maintains its 2026 guidance of $35 to $40 million. Contract revenue and other income fell 97% to $110,000 as a regulatory milestone from Xi’an Xintong recognized in the prior year was not repeated; Cost of revenue, which is related to Captisol cost of goods sold, totaled $3.3 million, falling 33% over prior year levels. The decrease is due to lower Captisol sales. Captisol gross margin fell to 62.2% from 64.0%; Amortization of intangibles was $8.1 million vs. $8.3 million, with the change due to deconsolidation of LNHC, the holding vehicle for the spin-out of Pelthos, on July 1st, 2025; Research and development expense fell 96% to $2.1 million versus $50.1 million. The decline was due to the absence of a funding payment for D-Fi royalty rights and expenses related to Pelthos; General & Administrative expenses were $20.8 million, up 11% from $18.8 million, with the increase primarily due to increases in headcount, higher employee-related costs, and share-based compensation; There were no fair value adjustments to partner program derivatives compared to a $443,000 expense; Total non-operating expense was $41.6 million vs. $14.0 million. Material items include a $49.2 million loss related to Pelthos holdings, a $3.9 million gain from short-term investments related to increases in Palvella stock, partially offset by declines in Viking stock, and $1.5 million in gains from other equity securities and financial instruments. This line item also includes net interest income, which totaled $4.9 million; Income tax benefit of $10.9 million represents a tax rate of 45.0%; Net loss was $13.3 million ($0.67 per share) versus a net loss of $42.5 million ($2.21 per share). Adjustments to 2025 GAAP earnings added $2.30 per share to generate core earnings of $1.63 per share.[1] Material adjustments include $2.32 for Pelthos offset by ($0.77) for income tax effect, among other items. As of March 31st, 2026, cash, equivalents, and short-term investments totaled $779 million. This amount compares to the $734 million balance held at the end of 2025. Free cash flow for the quarter totaled $48.5 million, while cash used in financing was $14.1 million, entirely related to taxes paid for equity awards. The company maintains access to a revolving line of credit and an at-the-market (ATM) facility with Stifel, Nicolaus, that can expand its access to capital as needed. Following the end of the quarter, Ligand announced that it intends to acquire XOMA for $739 million to be funded with cash on the balance sheet and accessing an existing revolving credit facility.[2] Access to the two sources of capital, along with future anticipated cash flows, is expected to continue to allow Ligand to deploy from $150 to $250 million on new royalty assets. Viking Therapeutics Program Termination Viking Therapeutics’ (NASDAQ: VKTX) TR-Beta program is a licensed thyroid hormone receptor beta agonist platform. It is developing VK2809 for MASH and VK0214 for X-linked adrenoleukodystrophy. The core license behind the TR-Beta program was part of a Master License Agreement signed on May 21st, 2014, with Ligand’s subsidiary Metabasis Therapeutics. On April 24th, 2026, Ligand delivered written notice to Viking, notifying them of termination of the TR-Beta Program. Details of the notification were included in a Form 8-K filed on April 30th. The termination was based on Ligand’s assertion that Viking materially breached its obligation to develop and commercialize the TR-Beta program. Upon successful termination, Viking must grant Ligand a non-exclusive, worldwide, royalty-bearing sublicense under any patent rights controlled by Viking. Viking disputes Ligand’s right to terminate the program, and we anticipate that the parties are reviewing the matter. Ligand’s goal is to get the programs based on TR-Beta developed due to the substantial unmet need, particularly for Metabolic Dysfunction-Associated Steatohepatitis (MASH) and replicate the success of other commercially available therapies. SUBSCRIBE TO ZACKS SMALL CAP RESEARCH to receive our articles and reports emailed directly to you each morning. Please visit our website for additional information on Zacks SCR. DISCLOSURE: Zacks SCR has received compensation from the issuer directly, from an investment manager, or from an investor relations consulting firm, engaged by the issuer, for providing research coverage for a period of no less than one year. Research articles, as seen here, are part of the service Zacks SCR provides and Zacks SCR receives payments totaling a maximum fee of up to $50,000 annually for these services provided to or regarding the issuer. Full Disclaimer HERE. ________________________ [1] Details of the GAAP to core earnings reconciliation are in Ligand’s earnings press release. Material adjustments include Share-based compensation expense, Amortization, change in fair value for Pelthos securities and gain on sale of Pelthos. [2] Ligand has access to a $125 million credit facility with Citibank, of which $124.4 million is available as of March 31st, 2026.

Interested in Palvella Therapeutics, Inc.? Here are five stocks we like better. Palvella Therapeutics said it is still on track to file an NDA in the second half of 2026 for QTORIN rapamycin in Microcystic Lymphatic Malformations, after reporting positive Phase 3 SELVA results that met the primary endpoint and all key secondary endpoints. The company said the FDA has granted an in-person pre-NDA meeting later this quarter, and management is planning for a potential FDA approval and U.S. launch in the first half of 2027. Backed by a recent $230 million financing, Palvella boosted launch preparations and ended the quarter with $261.9 million in cash, while estimating peak U.S. sales potential for QTORIN rapamycin at more than $1 billion. Palvella Therapeutics (NASDAQ:PVLA) said it remains on track to submit a New Drug Application in the second half of 2026 for QTORIN rapamycin in Microcystic Lymphatic Malformations, after reporting what executives described as positive Phase 3 results and outlining an expanded commercial plan supported by a recent $230 million financing. On the company’s first-quarter 2026 financial results and corporate update call, Founder and Chief Executive Officer Wes Kaupinen said the quarter represented “a major inflection point” for Palvella, citing Phase 3 SELVA study results, a strengthened balance sheet, new commercial and operational hires and increased U.S. launch preparation. → Insider Sales: Top AST SpaceMobile Insider Cuts Postion Over 30% Kaupinen said the Phase 3 SELVA study of QTORIN rapamycin in Microcystic Lymphatic Malformations met its primary endpoint and all pre-specified key secondary and secondary endpoints. He said the results, together with earlier Phase 2 data that supported the FDA’s Breakthrough Therapy Designation, provide what the company believes is a “robust evidence package” ahead of an NDA filing. The company also said the FDA has granted an in-person pre-NDA meeting for later this quarter. Kaupinen said Palvella’s objective is to align with the agency on an expedited submission plan, including whether a rolling submission could be appropriate under Fast Track and Breakthrough Therapy features. → Light Speed Returns: Corning Cashes In on NVIDIA Growth Palvella is developing QTORIN rapamycin as a localized topical therapy for rare skin diseases and vascular malformations with no FDA-approved treatments. Chief Scientific Officer Dr. Jeff Martini said the company is pursuing the 505(b)(2) regulatory pathway because oral rapamycin is already FDA-approved and has a substantial base of scientific and clinical knowledge. Martini said the program is intended to support traditional approval, rather than accelerated approval based on surrogate biomarkers. He said SELVA evaluated clinical endpoints, including physician-assessed disease improvement and patient-reported outcomes. → Years in the Making, AMD’s Upside Movement Has Just Begun Martini also discussed patient interview data from SELVA, saying patients at baseline described “constant bleeding and leaking” and the need for bandages or clothing changes. At week 24, he said, patient descriptions aligned with trial measurements, including themes of bleeding stopping, reduced leakage and lesions resembling normal skin. Palvella plans to include the qualitative interview work in its planned NDA. The company said its current goal is potential FDA approval and launch in the first half of 2027, assuming the NDA is submitted in the second half of 2026. Kaupinen said Palvella is increasing launch investments following its upsized $230 million equity financing in February. He said the financing “does not change our commitment to disciplined capital allocation,” but gives the company confidence that a potential QTORIN rapamycin launch will not be under-resourced. The company now plans to target a field sales force of 30 to 40 representatives, compared with its prior range of 20 to 40 representatives, and aims to have the team in place before the PDUFA date. Kaupinen said Palvella has also hired Medical Science Liaisons and is expanding that team nationally. Palvella said it is engaging high-volume treatment centers, including vascular anomaly centers, to support disease education and prepare for a focused launch model. Kaupinen said the company estimates more than 30,000 diagnosed U.S. patients with Microcystic Lymphatic Malformations, with about half concentrated in roughly 400 centers. Kaupinen said Palvella estimates peak U.S. sales potential of more than $1 billion for QTORIN rapamycin in Microcystic Lymphatic Malformations. He said that estimate is based on prevalence work, field checks with high-volume centers and the company’s view that the therapy could support orphan pricing within its previously guided range of $100,000 to $200,000 per patient per year. Beyond Microcystic Lymphatic Malformations, Palvella outlined progress across several programs tied to its QTORIN platform. Cutaneous venous malformations: Palvella said it remains on track to initiate a pivotal Phase 3 study in the second half of 2026. Martini said Phase 2 data showed 73% of patients improved, exceeding the company’s target of 30%. The company has submitted a Breakthrough Therapy Designation application and expects a decision in the middle of the year. Clinically significant angiokeratomas: Palvella said the Phase 2 LOTU trial has begun and multiple patients have already been dosed. The company expects data in the second half of 2027. The program has received Fast Track Designation. Disseminated superficial actinic porokeratosis, or DSAP: Palvella said it plans to initiate a Phase 2 study of QTORIN pitavastatin in the second half of 2026. Martini said the company has received more than 40 inbound unsolicited patient inquiries about the planned study. Chief Innovation Officer Dr. David Osborne said the QTORIN platform is designed to deliver therapeutic levels of drug into diseased skin, including the dermis, while maintaining tolerability and minimizing systemic absorption. He said QTORIN rapamycin has six issued patents, additional applications pending, proprietary formulation and manufacturing know-how, and potential orphan drug exclusivity. In response to an analyst question, Osborne said he had personally tested 15 compounds in QTORIN, all of which dissolved to high degrees, and that the platform delivered each of the roughly nine compounds taken into in vitro skin permeation testing. “I haven’t found the one yet that it doesn’t work for,” Osborne said, while cautioning that it would be “foolhardy” to claim the platform could work for every molecule. Chief Financial Officer Matt Korenberg said Palvella ended the first quarter with $261.9 million in cash, cash equivalents and short-term investments in U.S. Treasuries. Following the February financing, he said the company believes it has sufficient cash to last “well into a potential commercial launch.” Based on current assumptions for a first-half 2027 approval and launch, and using current consensus analyst revenue estimates, Korenberg said Palvella would expect its cash to last through cash flow break-even. Korenberg said Palvella’s model is focused on first-in-disease therapies for serious rare diseases with no approved products, using the QTORIN platform with existing molecules. He said the company believes that approach can allow Palvella to move from concept to Phase 2 human data on less than $10 million of capital. Looking ahead, Palvella said expected catalysts include the in-person pre-NDA meeting for QTORIN rapamycin in Microcystic Lymphatic Malformations, NDA submission in the second half of 2026, initiation of a Phase 3 study in cutaneous venous malformations, Phase 2 initiation in DSAP, and announcements of two additional disease programs in 2026. The company said one will be a new QTORIN product candidate and the other will be a fourth indication for QTORIN rapamycin. During the question-and-answer session, Kaupinen said Palvella is also evaluating opportunities outside the United States after receiving inbound interest from potential licensing partners in Japan and Europe. He said the company’s current preference is likely to remain focused on the U.S. market and intensify partnering discussions after a potential FDA approval. “Our goal remains clear,” Kaupinen said, “to serve patients with serious rare skin diseases and vascular malformations for which there are no FDA-approved therapies.” Palvella Therapeutics, Inc (NASDAQ: PVLA) is a clinical‐stage biopharmaceutical company devoted to the discovery and development of innovative therapies for immunological and inflammatory diseases. The company employs a proprietary small‐molecule and biologics platform to identify and modulate key molecular pathways that drive neutrophil‐ and complement‐mediated inflammation, aiming to deliver targeted treatment options for patients with significant unmet medical needs. Palvella's pipeline comprises several preclinical assets designed to address both prevalent chronic inflammatory conditions and rare autoinflammatory syndromes. The article "Palvella Therapeutics Q1 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for May 2026.

Interested in Ligand Pharmaceuticals Incorporated? Here are five stocks we like better. Ligand's Q1 was driven by royalties: total revenue was $52 million (+14% YoY) with royalty revenue $43 million (+56%) and adjusted diluted EPS $1.63 (+23%), and the company finished the quarter with about $780 million in cash/investments and nearly $1 billion of available capital ahead of the XOMA close. The announced acquisition of XOMA Royalty Corporation—adding more than 120 assets—is expected to be immediately accretive, contributing $0.50 to adjusted EPS in 2026 and $1.50 in 2027, and management updated 2026 guidance to $270–$310M total revenue, $225–$250M royalty revenue, and $8.50–$9.50 adjusted EPS (assuming a Q3 close). Key portfolio catalysts include the April FDA full approval of FILSPARI for FSGS (broad label with meaningful upside beyond 2026) and Palvella’s positive Phase III SELVA results for QTORIN™ rapamycin in mLM, with an NDA planned in H2 and multiple expedited FDA designations. Ligand Pharmaceuticals (NASDAQ:LGND) reported first-quarter 2026 results that management said reflect the operating leverage of its royalty aggregation strategy, highlighted by growing royalties from key commercial products and the pending acquisition of XOMA Royalty Corporation. CEO Todd Davis told investors the year is “off to an exciting start” with “transformative milestones within our existing portfolio” and the announced XOMA transaction. Davis said Ligand delivered 56% royalty revenue growth and 23% adjusted EPS growth versus the first quarter of 2025, which he framed as the outcome of a strategy shift implemented in 2022 toward a “pure royalty aggregation model” and away from “infrastructure-heavy technology platforms.” → Insider Sales: Top AST SpaceMobile Insider Cuts Postion Over 30% CFO Tavo Espinoza said Ligand’s first-quarter total revenue was $52 million, up 14% year-over-year, with royalty revenue of $43 million, up 56%. Adjusted diluted EPS was $1.63, up 23% year-over-year. Espinoza attributed the quarter’s performance primarily to “continued growth from FILSPARI, Ohtuvayre, and Qarziba.” On a GAAP basis, Ligand reported a first-quarter diluted EPS loss of $0.67, compared to a loss of $2.21 in the year-ago period. Espinoza said the 2026 GAAP loss was “primarily driven by fair value adjustments on our equity holdings,” while the prior-year loss “largely reflects a one-time $44 million accounting charge” tied to Castle Creek’s funding of a Phase III study. → Light Speed Returns: Corning Cashes In on NVIDIA Growth Ligand ended the quarter with approximately $780 million in cash and investments and $200 million of undrawn revolving credit capacity, which Espinoza said provided “nearly $1 billion of available capital” as the company works toward closing the XOMA transaction. Management emphasized that, while Travere Therapeutics’ FILSPARI has become a major driver, it is part of a broader portfolio. Espinoza said FILSPARI is performing “very well,” with “strong demand trends and growing physician adoption,” and noted Travere had built a field force of more than 100 professionals with overlap between IgA nephropathy (IgAN) and focal segmental glomerulosclerosis (FSGS) prescribers. → Years in the Making, AMD’s Upside Movement Has Just Begun Lauren Hay, vice president of portfolio strategy and investments, highlighted the April FDA full approval of FILSPARI for FSGS in patients without nephrotic syndrome. She called the label “highly positive” and said it was broad, encompassing “patients with primary, secondary, and genetic FSGS.” Hay cited Travere’s estimate of more than 30,000 eligible U.S. FSGS patients and said early launch indicators were encouraging, noting that “the first FSGS patients were treated within just one week” after approval. In Q&A, Espinoza said FILSPARI’s FSGS contribution had been included in Ligand’s model on a risk-adjusted basis and that the timing of approval—April rather than early January—“offsets the de-risking,” resulting in limited incremental impact in 2026. He added, “Where we’ll see a significantly more impactful impact from FSGS sales is as we get out into, you know, 2028 and beyond,” and clarified that the company’s “$0.50 increase in guidance is purely related to the XOMA acquisition.” Espinoza also discussed Ohtuvayre, describing “strong year-over-year growth” but noting sequential sales were “modestly impacted” by seasonality and reimbursement timing, with trends improving as the quarter progressed. Hay added that Ligand is watching for potential geographic catalysts, including regulatory activity related to Ohtuvayre in China. On Tzield, Hay said Ligand was “encouraged” by the product’s label expansion and characterized the impact as incremental, emphasizing that the market requires identifying patients prior to symptoms, which involves significant screening investment by Sanofi. Hay also pointed to Palvella’s Phase III SELVA trial results for QTORIN™ rapamycin in microcystic lymphatic malformations (mLM), calling the outcome “clinically transformative.” She said the drug demonstrated “a highly statistically significant outcome on the primary endpoint and all secondary endpoints,” including a “positive 2.13 point improvement” on the mLM Investigator Global Assessment scale. Hay noted Palvella had previously guided that a positive one-point change would be a “decisive win,” with an upside case of 1.5 points. According to Hay, Palvella plans to submit an NDA in the second half of the year and is accelerating U.S. launch readiness. She added that QTORIN™ rapamycin has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA for mLM, and that Palvella is also developing it for cutaneous venous malformations (CVM), with a breakthrough designation application planned following a recent FDA meeting. Ligand’s largest recent corporate development is its announced acquisition of XOMA Royalty Corporation, which Davis said will add “more than 120 commercial, clinical, and pre-clinical stage assets” and is expected to be “immediately accretive.” Davis also said the XOMA deal is expected to add $0.50 per share of adjusted EPS in 2026 and $1.50 in 2027. Asked how the transaction came together, Vice President of Investments and Business Development Michael Vigilante said Ligand had a “longstanding relationship” with XOMA and believed its business had reached “an inflection point.” He said the parties engaged “full swing in December,” with diligence continuing into 2026 before reaching alignment in April. Vigilante said the deal provides XOMA shareholders liquidity and, for Ligand, offers “meaningful synergies” by broadening the portfolio across stages of development while delivering “immediate EPS accretion” and “long-term durable accretion.” Davis said the XOMA portfolio is relatively unique in breadth, and that most opportunities in Ligand’s pipeline are “single or double assets.” On integration and synergies, Davis said synergies are “extremely high, approaching 100%,” adding that Ligand has “set up the business to absorb these types of partnerships.” Espinoza reaffirmed updated 2026 guidance tied to the XOMA announcement, assuming the deal closes in the third quarter: Total revenue: $270 million to $310 million Royalty revenue: $225 million to $250 million Adjusted EPS: $8.50 to $9.50 Looking ahead, Espinoza said Ligand expects about $1.50 per share of incremental adjusted EPS in 2027 from a full year of XOMA portfolio contribution, and “combined operating cash flow of approximately $300 million,” reflecting tax attributes acquired in the transaction. He added that Ligand’s capital deployment strategy remains investing $150 million to $250 million annually in new royalty opportunities. Espinoza also addressed the contingent value right (CVR) associated with the deal, explaining it relates to proceeds from XOMA’s litigation with Janssen. He said the litigation assets will remain in a post-reorganization XOMA LLC, which will distribute 75% of any net proceeds to former XOMA shareholders via the CVR, while Ligand retains 25% of rights. “Importantly, Ligand has no obligation to fund the litigation,” he said. In Q&A, Espinoza said XOMA’s portfolio includes milestone opportunities that could be realized in the second half of 2026, assuming a third-quarter close. On tax attributes, he said the company had not disclosed the “quantum,” adding that NOL usage will be limited, while Section 174 R&D tax credits “will come over 100%” and be usable immediately. Separately, Davis addressed the termination of Viking’s TR-Beta program and Ligand’s goals for its 2809 asset, saying the company’s objective is to move it forward in development. Espinoza added Ligand carries nothing for those assets on its balance sheet, as related intangible assets are “fully amortized,” and any impact would likely be limited to “minor incremental legal expenses.” As Ligand prepares to integrate XOMA, Davis said the company believes it has embedded growth in its current portfolio but plans to continue doing new deals to compound growth further. He said Ligand will provide an updated five-year plan at an Investor Day expected in December. Ligand Pharmaceuticals, Inc is a biopharmaceutical company that acquires, develops and out-licenses proprietary technologies designed to help pharmaceutical and biotechnology companies discover and develop novel medicines. Operating primarily through its research services and royalty-generating businesses, Ligand focuses on building a diversified portfolio of technology platforms and partnering with industry leaders to advance therapeutic candidates across multiple disease areas. The company's product offerings center around several core platforms. The article "Ligand Pharmaceuticals Q1 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for May 2026.