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16-05-2025
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Palvella Therapeutics Inc (PVLA) Q1 2025 Earnings Call Highlights: Strong Clinical Progress ...
Cash and Cash Equivalents: $75.6 million as of March 31, 2025. Research and Development Expenses: $4.1 million for Q1 2025, up from just under $1 million in Q1 2024. General and Administrative Expenses: $3.8 million for Q1 2025, compared to $800,000 in Q1 2024. Net Loss: $8.2 million or $0.74 per diluted share for Q1 2025, compared to $2.7 million or $1.54 per diluted share in Q1 2024. Cash Spend Forecast: Approximately $30 million total cash spend for 2025, with an expected year-end cash balance of at least $55 million. Clinical Trials: Exceeded enrollment target of 40 patients in Phase 3 study for QTORIN and rapamycin; Phase 2 study for cutaneous venous malformations has six sites open and enrolling. Funding and Cash Runway: Financing provides cash runway into the second half of 2027. Warning! GuruFocus has detected 6 Warning Signs with TSX: Release Date: May 15, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Palvella Therapeutics Inc (NASDAQ:PVLA) has exceeded its enrollment target of 40 patients in the Phase 3 Selva study for QTORIN rapamycin, indicating strong interest and progress in their clinical trials. The company has received FDA's breakthrough therapy, fast track, and orphan drug designations for QTORIN rapamycin, which could expedite the regulatory approval process. Palvella Therapeutics Inc (NASDAQ:PVLA) is financially strong with $75.6 million in cash and cash equivalents, providing a clear cash runway into the second half of 2027. The company is planning to unveil two new QTORIN programs in the second half of the year, indicating a robust pipeline and potential for future growth. Palvella Therapeutics Inc (NASDAQ:PVLA) has expanded its intellectual property portfolio with the issuance of a fifth US patent, extending anticipated claims into 2038. Palvella Therapeutics Inc (NASDAQ:PVLA) reported a net loss of $8.2 million for Q1 2025, which is a significant increase from the $2.7 million loss in the same period in 2024. The company faces the challenge of commercializing its products in a market with no existing FDA-approved therapies, which requires careful planning and execution. There is uncertainty regarding the pricing strategy for QTORIN rapamycin, as it will depend on the results of the Phase 3 trial and further market analysis. Palvella Therapeutics Inc (NASDAQ:PVLA) is still in the process of hiring a Chief Commercial Officer, which is crucial for guiding the company's commercial strategy. The company needs to manage the complexities of a 505B2 submission pathway for NDA approval, which requires careful coordination with regulatory advisors. Q: What is the target enrollment for the Phase 3 MLM study, and are there any plans to explore additional subpopulations? A: The target enrollment has always been 40 patients, and we are pleased with the demand from all clinical study sites. The study is designed based on Phase 2 efficacy results, and with 40 patients, we are more than 99% powered for the Phase 3 study. (Wesley Kaupinen, CEO; Jeffrey Martini, CSO) Q: How does the recent epidemiology study impact the company's view on the total addressable market for MLM? A: The study aligns with previous estimates of over 30,000 diagnosed US patients. It also introduces new insights, suggesting over 1,500 new cases annually. This information will help refine our market model and commercial strategies, particularly targeting the 150 vascular anomaly centers. (Wesley Kaupinen, CEO) Q: What are the plans for commercializing the pipeline, and would you consider a global partner? A: We plan to prioritize a stand-alone US launch for our products. For markets outside the US, such as Europe and Japan, we will consider both stand-alone launches and partnerships, with a bias towards leveraging partners' footprints. (Wesley Kaupinen, CEO) Q: Can you provide details on the NDA submission timeline and any additional work required for the 505B2 submission? A: We aim to submit the NDA in the second half of 2026, following top-line data in Q1 2026. We expect a six-month priority review due to breakthrough and fast track designations. The 505B2 pathway will streamline the review process by referencing existing data on oral rapamycin. (Wesley Kaupinen, CEO) Q: How is the company addressing the heterogeneity in MLM and CBM patient recruitment for the Phase 3 study? A: The study is enriched for moderate to severe patients to ensure a clear treatment effect. We have strict inclusion criteria and controls to ensure the right patients are enrolled, which is crucial given the small sample size. (Wesley Kaupinen, CEO; Jeffrey Martini, CSO) For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
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16-05-2025
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Q1 2025 Palvella Therapeutics Inc Earnings Call
Wesley Kaupinen; President, Chief Executive Officer; Palvella Therapeutics Inc Jeffrey Martini; Chief Scientific Officer; Palvella Therapeutics Inc Operator Good day and thank you for standing by. Welcome to Palvella's first quarter 2025 Financial results and corporate Update conference call. (Operator Instructions) I would like to turn the conference over to your speaker today. Bohan way, please go ahead. Thank you, operator. Good morning and thank you for joining the Palvella Therapeutics Q1 2025 Financial Results and corporate update call. As a reminder, our press release summarizing today's updates is available under the investor section of our website at On today's call, we are joined by Wes Kaupinen, our Chief Executive Officer, Jeff Martini, our Chief Scientific Officer, and Matt Kornberg, our Chief Financial we begin, please note that today's remarks may include forward-looking statements regarding our development programs, regulatory plans, commercial outlook, and financial performance. These statements are based on current assumptions and subject to risks and uncertainties that may cause actual results to differ. Please refer to our filings with the SEC for more now I'll turn the call over to West. Wesley Kaupinen Thank you, Bohan. Good morning, everyone, and thank you all for joining us today. This past Monday morning, the Palvella team, including our Scientific and Clinical Collaborators, Investors, Senior Advisors, Board Members, and family members, converged on New York City for the ringing of NASDAQ's opening becoming a publicly traded company approximately five months ago is an important milestone for the company, and it is a testament to Palvella's vision of building the leading rare disease biotech company focused on serious genetic skin diseases with no FDA approved therapies. It is also a testament to our mission, which is to relentlessly serve those rare disease patient populations who have historically been to our strategy, which is to focus our therapeutic development efforts and eventually our commercial efforts exclusively on those diseases with no FDA approved therapies. We want to be first for patients. Becoming public is furthermore a testament to the Palvella management team, who have turned mere concepts into breakthrough designated medicines while turning the challenges of drug development and rare complex diseases into collective sentiment at NASDAQ that day was that our substantial progress to date, including both developing a late-stage pipeline with QTORIN and rapamycin, and a compelling platform with QTORIN is truly only the beginning for hope is to return to NASDAQ one day in the near future to celebrate the first regulatory approval of QTORIN 3.9% Rapamycin hydrous gel, a milestone we believe we're even closer to achieving. Based on today's exciting update that we have exceeded our enrollment target of 40 patients in the landmark Selva phase 3 study of QTORIN rapamycin for the treatment of microcystic lymphatic quarters, four anticipated high-impact milestones, moving left to right, microcystic lymphatic malformations are a serious, rare, chronically debilitating, and lifelong monogenic disease. The genetics, disease biology, target skin tissue, and natural history of microcystic lymphatic malformations have all been well they're caused by PI3K mutations. Biologically, the PI3K mutation results in the hyperactivation of the [M4] pathway, which ultimately produces malformed vessels that protrude through the skin, as you can see from the picture here on the target skin tissue is the dermis, which is the site of disease origin cystic lymphatic malformations. From a natural history perspective, the disease is present at birth, has no spontaneous regression, and is proliferative and major clinical burden to these patients is referred to as lymphorhea, which is the leaking or discharge of internal lymphatic fluid onto the skin or into the soft tissues. The result of this is that these patients are at persistent risk of serious infections, including acute cellulitis, which can cause repeated are unfortunately no FDA approved therapies for these patients. We'll provide an update today on our phase 3 Selva study of QTORIN and rapamycin, which has received FDA's breakthrough therapy, fast track, and orphan drug designations. We anticipate top line results from the study in Q1 to cutaneous venous malformations, the disease pathology of cutaneous venous malformations is similarly driven by the [MTOR] pathway caused by mutations in either PI3K or TI 2, which leads to overactivated MTOR signaling. Cutaneous venous malformations are a serious disease, and they're characterized by dysregulated growth of malformed veins in the skin, which can cause functional impairment and of the genetic driver of the disease, cutaneous venous malformations continue to grow over time and do not spontaneously regress. There are unfortunately no FDA approved therapies for patients afflicted with cutaneous venous malformations. We'll update today on our phase 2 to study of QTORIN and rapamycin, which has received FDA's fast track designation, and we anticipate the study will report top line results in Q4 of if approved, we believe based on market research that QTORIN and rapamycin has the potential to be first line therapy and standard of care for patients with both microcystic lymphatic malformations and cutaneous venous pipeline beyond our two lead programs and microcystic LMs and cutaneous venous malformations continues to make tangible progress under the strong leadership of our Chief Scientific Officer, Dr. Jeff Martini. In the third panel on this slide, we truly believe QTORIN rapamycin can represent a pipeline and a product with clinical and commercial potential that extends well beyond microcystic lymphatic malformations and cutaneous venous anticipate unveiling our third target clinical indication for QTORIN rapamycin in the second half of this year. Moving to the fourth panel, we also anticipate unveiling our next QTORIN program in the second half of this year, which would be our second product candidate from the QTORIN platform after QTORIN and see both our next QTORIN rapamycin indication and our next QTORIN platform program as key near-term value drivers for Paul Bell. Dr. Martini will provide updates on our progress on both programs on this strong momentum at alvella is highlighted first by what's presented at the top of the slides. We have now exceeded our enrollment goal of 40 patients in our landmark phase 3 study evaluating QTORIN and rapamycin for the treatment of microcystic our phase 2 (toyva) study of QTORIN and rapamycin for the treatment of cutaneous VMs, we now have six sites open and enrolling, and top line readout is anticipated in Q4 recently participated in the Society of Investigative Dermatology. And the International Society for the Study of Vascular Anomalies meetings where we gathered new insights and deepened our relationships with scientific and clinical leaders in the an organizational standpoint, We've added Jason Burdett to our executive team as SVP of CMC and technical operations. Jason brings 30 years of experience in global pharmaceutical development, technical operations, and supply chain to Pulvella, and we're thrilled to have him on our senior team as we approach planned NDA submission next year and if approved, stand-alone US commercialization in also continue to make progress on our search for an exceptional Chief Commercial Officer with that higher plan for the second half of this year. For this position, we're seeking a proven leader with experience launching novel therapies that were first to market in serious rare addition to the proven experience commercializing in rare diseases consistent with Palvella's historical and current model of operating with capital efficiency, we are seeking to recruit a leader who can lead a launch in a thoughtful and capital efficient look forward to keeping everyone updated on our progress, and we've also expanded our intellectual property portfolio with the issuance of our fifth US patent with anticipated claims from that patent into to our lead program, QTORIN and rapamycin for the treatment of microcystic lymphatic malformations, I'm pleased to share thanks to the dedicated efforts of our phase 3 investigators and the research coordinators at clinical trial sites, our patient advocacy collaborators, the highly committed Paulvella clinical operations team, and most of all the patients living with microcystic lymphatic we have exceeded enrollment of 40 patients and our pivotal phase 3 trial evaluating QTORIN and rapamycin in patients with microcystic LMs. The study has generated strong interest with all 13 sites, including institutions such as Stanford University, the Cleveland Clinic, Children's Hospital of Philadelphia, the Mayo Clinic, to name a few, all enrolling patients into the terms of concluding enrollment, we're anticipating closing study enrollment in June after all pending enrolled patients have completed screening. As a result of the diligent efforts and commitment from investigators and sites beyond these pending enrolled patients, there is additional potential for more patients to enter the believe that it is in Palvella's best interests to honor the site's work and their relationships with patients and accommodate additional patients into the study in a timely the same time, now that we've exceeded our enrollment target of 40 patients, our goal is to bring the phase 3 trial to an orderly close while communicating closely and clearly with our study investigators and sites on defined study closure you can see from the graphic at the bottom of the slide, if patients meet study eligibility criteria, they are then enrolled into the study. Patients first move through an eight-week baseline period before moving to the efficacy evaluation period of 24 weeks, followed by the treatment extension period of 24 look forward to providing more detail on final patient enrollment numbers at the time of achieving full enrollment in the near future. Overall, we are pleased that we've been able to accomplish our phase 3 study enrollment goals while remaining on track to report top line data in the first quarter of addition, as you'll hear from our CFO Matt Kornberg later in this call, we are also staying on track for our 2025 cash expenses and long-term cash runway forecast. Moving to our regulatory overview, we've all been carefully monitoring the recent and ongoing changes at FDA, and the Palvella management team will continue to remain vigilant in doing so along with our regulatory advisors. For Palvella's QTORIN and rapamycin, we wanted to provide additional clarity that our program is regulated by the division of Dermatology and is within the Center for Drug Evaluation Research, or CEDA, which you can see listed at the top of this slide. Importantly, the division of dermatology and dentistry continues to be led by Dr. Jill Lindstrom, a dermatologist and the division director who began as director of the division in late 2023 and continues in that role to our plan 505B2 pathway for our NDA submission, we anticipate division level leadership. In this case, Dr. Lindstrom, would be responsible for the NDA review and approval decision. So, while there have been many changes across the FDA that we've all read about and that we continue to track, there has by contrast been consistency for Palvella in terms of number one, the division regulating Palvella 's program. Dermatology and dentistry and number two, the leadership of that division in this case, Dr. a reminder, assuming positive phase 3 data, we anticipate that our program could potentially be expedited as a result of some of the designations we've achieved over the years. In Q4 of 2023, we were awarded FDA's breakthrough therapy designation based on the results from our phase 2 baseline controlled single arm study of 12 therapy designation was added to our previously granted fast track and orphan drug designations. Additionally, out of 51 applications received by FDA's Orphan Products grants program in fiscal year 2024, Paulvella's Phase 3 Selva trial was one of just seven clinical programs and the only phase 3 study to be named an awardee of a non-dilutive FDA our case, we anticipate up to $2.6 million from the FDA over multiple years in non-dilutive funding to support the Phase 3 to the bottom of the slide in terms of the regulatory environment for our other planned pipeline programs, the Paulvella team is closely monitoring emerging regulatory frameworks, including a newly proposed plausible mechanism accelerated approval pathway for certain rare diseases that has been publicly highlighted at a conceptual level by Commissioner Dr. Marty were encouraged by the comments about the need to expedite therapies to patients living with rare, ultra rare, and oftentimes serious diseases, and we will continue to follow updates from FDA on these new pathways and their potential relevance to our earlier stage programs.I'd like to now turn to the commercial opportunity we see ahead for QTORIN and rapamycin. As a reminder, at a high level, Palvella firmly believes that the opportunity to develop and commercialize in serious rare diseases where Palvella can introduce the first FDA approved therapy can represent very attractive commercial opportunities, especially considering the lower competitive intensity dynamics in these markets compared to more conventional and competitive rare disease markets which are already well a function of having existing approved therapies in most cases, as this slide shows for microcystic lymphatic malformations, a recent epidemiology study conducted by a multidisciplinary team that leveraged an extensive medical claims database resulted in the following terms of estimated diagnosed US prevalence of microcystic LMs, the study indicated. That there could be greater than 44,000 diagnosed US patients, figures that are largely in line with Paul Della's previous estimates. In terms of estimated annual incidence of microcystic LMs, the study indicated that there could be 1,500 or more new microcystic LM cases per taking these prevalence and incidence estimates and overlaying them with anticipated orphan pricing levels, the data supports that microcystic lymphatic malformations could be a multi-billion-dollar total addressable market in the United the posts are also indicated that about one third of diagnosed patients are currently managed at vascular anomaly centers, which are already established centers of excellence for diagnosing and treating microcystic lymphatic malformations and other vascular look forward to providing more information related to the commercial opportunity and Palvella's commercial planning as we on board a Chief Commercial Officer in the coming months. With that, I'll turn it over to our Chief Scientific Officer, Dr. Jeff Martini. Jeffrey Martini Thank you, Wes. In April, I had the opportunity to represent Palvella at the International Society for the Study of Vascular Anomalies, or ISSFA conference in Paris. The leading global scientific and medical forum focused on vascular malformations, including microcystic lymphatic malformations and cutaneous venous there, I met with many of the field's foremost clinicians to discuss our two lead indications, MLMs and CBMs, as well as other serious rare skin diseases that may be strong candidates for QTORIN rapamycin and future QTORIN-based themes emerged that reinforce the direction we're headed. First, the field is clearly moving towards targeted therapeutic approaches based on the underlying genetic drivers of each vascular there's a real need for therapies that can improve efficacy while reducing the systemic side effects associated with current treatments, many of which were originally developed for internal infiltrative diseases rather than localized cutaneous third, there's a significant opportunity to expand treatment options across a broader spectrum of cutaneous vascular malformations both within and outside the MTOR pathway, which we are actively pursuing as part of our pipeline expansion activities. These insights continue to support the potential of the QTORIN platform and help guide how we prioritize future indications within our development on to our clinical trial of cutaneous venous malformations, or our toy study in CVMs is actively enrolling. This single arm baseline control trial includes patients aged six and over and evaluates both clinician and patient reported outcomes. The purpose of the study is to one, evaluate safety and tolerability, and two, determine which endpoints are most sensitive to change with QTORIN currently have six sites open, five of which came online in the past two months. Topline data from Tuiba are expected in the fourth quarter of this we continue to build momentum behind our lead programs, we're very focused on expanding our QTORIN platform through two distinct development tracks. First, we are advancing additional indications for QTORIN rapamycin on what we've seen in the clinic and through ongoing dialogue with experts in the field, we view QTORIN rapamycin as a pipeline in a product. We've identified several diseases that fit the stringent Pavella criteria of serious rare skin diseases that lack FDA approved therapies and where MTR dysregulation is a key driver of the disease opportunities fit directly within Pavella's development strategy and offer the potential to further extend the impact of our lead we're progressing a novel QTORIN-based program that delivers a different therapeutic agent or API. This effort is being guided by our close collaboration with many of the world's leading rare skin disease experts, clinicians who frequently see the limitations of current therapies and who help us identify promising targets both based on both biology and real-world treatment experiencing experience, including off-label use of systemic each candidate is rigorously evaluated through our disease selection process, which links biological rationale with commercial viability and alignment with Palvella's mission. Both programs, our next QTORIN and rapamycin indication and our novel QTORIN asset have identified lead diseases, and the latter has progressed to defined target both are tracking towards potential fast track and breakthrough therapy designation eligibility. And remain on schedule to be announced in the second half of this year. With that, I'll turn it over to Matt to provide a financial update. Thanks, Jeff. Before I turn to my financial comments, I first wanted to touch on one last point on Jeff's slide. In addition to all the key criteria that Jeff mentioned for choosing new products to develop, we also focus on diseases and programs that align with our capital efficient our next QTORIN molecule, we're targeting generating phase 2 proof of concept data within 2.5 years and for less than $10 million of clinical spend. We believe this approach allows Palvella to deliver maximum benefit for both patients and value for now to the financials, Palvella remains in a strong financial position, allowing us to continue confidently on our path to successfully developing and commercializing drugs to treat rare diseases. Cash and cash equivalents as of March 31, 2025 or $75.6 fortunate to have two years of cash runway remaining following the oversubscribed pipe financing we completed in connection with our reverse merger at the end of by BVF Partners and Frasier Life Sciences and with participation from our major existing investors plus a roster of new investors, the financing combined with existing cash, provided Palvella with a clear cash runway into the second half of current funding covers our major upcoming milestones including the completion of our phase 3 microcystic LM trial, completion of our phase 2 cutaneous venous malformations trial, submission of the microcystic LM NDA filing, and pre-commercialization efforts, as well as the addition of two new programs to the cash spend and financial results for Q1 2025 were in line with our expectations, and I'll briefly review the results reported in this morning's press and development expenses were $4.1 million for Q1 2025 as compared to just under $1 million for the comparable period in 2024, with the increase driven primarily by the expenses associated with our microcystic LM and cutaneous VM clinical and administrative expenses were $3.8 million for Q1 2025 as compared to $800,000 for the comparable period in 2024. The increase here was driven by public company costs and the cost associated with our increased net loss was $8.2 million or $0.74 per diluted share for Q1 2025 compared to a net loss of $2.7 million or $1.54 per diluted share for the comparable period in 2024. We remain on track to end the year with at least $55 million in cash and cash equivalents based on our current strategic operating cash spend for 2025 remains on track for approximately $30 million in total cash spend. With that, I'll turn the call back over to West for some closing remarks before we open up the call for questions. Wesley Kaupinen Great, thank you, Matt. Looking ahead, Palvella has a clear set of four high impact milestones. We expect top line data from our phase 3 SELVA study in Q1 2026. We expect top line data from our phase 2 toIVA study in Q4 2025. And we have planned to announce two new QTORIN programs in the second half of this year, an additional QTORIN rapamycin indication and a novel product from our QTORIN preparation is underway, and we're actively building towards commercialization if approved, including the planned addition of a Chief Commercial Officer later this year. In closing, Paul Vella is positioned for a transformative year ahead, and we want to thank everyone for attending today's call. We'll now open the line for questions. Operator Thank you, ladies and gentlemen. (Operator Instructions) Our first question comes from Ritu Barra with TDC and your line is open. Good morning guys. Thanks for taking the question. Wes, I wanted to ask you about target enrollment for Phase 3 MLM. Is there a soft target enrollment you have in mind beyond the 40? I know you mentioned keeping the relationship. With the KOLs and patients, which is very important, but I'm wondering if there may be some additional powering targets or important subpopulations worth exploring with a bigger then I have a manufacturing related follow up. Wesley Kaupinen Great. Hey, thanks for being on today's call. To answer your first question, the target enrollment has always been 40 patients enrolled into this study, so we were really pleased with the demand that we saw from all of our clinical study sites, all13 have enrolled patients.I'll ask Jeff to briefly comment as it relates to your question around powering and subpopulations. Jeffrey Martini Yeah, so, thanks for the question. This is Jeff. So we designed the study based on the efficacy results that we observed in phase that study, all12 patients were in the top two categories of that 7 point change scale, much improved or very much improved, and based on the results, with 40 patients or more, we're greater than 99% powered into the phase 3 study, and those assumptions remain on track. Got it. And you know what, I changed my mind. My follow-up question is going to be on something different. How does your poster impact the way that the company is looking at the TAM for MLM? Like if the incidence of MLM is higher than expected, how will this is this, I mean, I know you don't have a Chief Commercial Officer yet, but Wes, given your background, how is this changing your plan in your head on how to target these patients and those, I believe 142 or 150 ish vascular anomaly centers? Thanks. Wesley Kaupinen Well, thanks for you well know, and others on this call on these rare diseases where there's no approved therapies. There's really an obligation by the innovator in the space, in this case, Paul Bella, to do prospective methodical work to try to really understand what the prevalence and incidents are of these patient our perspective or just a little bit of history. We had done a real-world occurrence study that's published in an orphaned Journal of Rare Diseases that estimated that there could be upwards of 80,000 microcystic or mixed patients. We followed that with a claims analysis last year which suggested that there's around 40,000 diagnosed patients in the to answer your question on the prevalent side. We think the numbers from the poster are largely in line with what we've been guiding on US diagnosed prevalence. We've been guiding greater than 30,000 diagnosed US patients. What is new, and I'm glad you asked the question, is there hadn't been any rigorous work done around estimated annual incidence of microcystic lymphatic Matt and the team are working with our commercial consultants to really closely evaluate our market model to reflect that there could be 1,500 or more new patients coming into that eventual total addressable pool of patients that can be addressed with QTORIN and the on the targeting question, we've certainly wanted to understand, given that there are established centers of excellence, these vascular anomaly centers, we think that that's favorable as we think about commercial dynamics is that these centers of excellence in we want to understand from this analysis approximately how many patients with diagnosed microcystic LMs are currently within those about 150 centers. We estimate, as you see from the poster, that about a third of those, so greater than 10,000, somewhere perhaps between 10,000 and 15,000 are in these vascular anomaly do think, and we've got a lot more work to do from a commercial planning perspective, but we do think that that concentration of patients should provide efficiencies for how we think about building out commercial and medical infrastructure in the United States. Thank you. Operator One moment for our next question. Our next question comes from Annabelle Sammy with Stefo. Your line is open. Hi, thanks for taking my question. Actually just following on Rita's questions about the commercial side. And the infrastructure you might need. So clearly you've identified that 1/3 of the patients are sitting in these centers, but that, follows that 2/3 are not sitting at those so does that require a larger infrastructure than you may have anticipated or are you going to stick with just targeting the 142 centers and doing Separate outreach to bring people into those. And then if I can follow on the on the higher side, I guess you've said in the past that pricing is really going to depend on the results of the trial, and it's rare that you see 100% efficacy, no less carrying that into phase when you think about pricing, have you established thresholds of efficacy for various price ranges, like, say for example 70% reduction will get you X and 90% reduction will get you Y, or is it still kind of nebulous in that range? Like what do we need to see to see like really clear rare orphan pricing? Thanks. Wesley Kaupinen Yeah, thanks for the question, Annabelle. So to answer your first question, we think that it's a very attractive commercial dynamic to have to have greater than an estimated 10,000 patients concentrated in about 150 centers. So right now, as we stand today, and this is all pending bringing in a Chief Commercial Officer who's going to refine our plan. We think that that's second area, and we're doing more digging on this with some of our consultants is that beyond those 150 vascular anomaly centers, there's a second wave of academic medical centers that do have a fairly significant patient we think that we can also efficiently go after not just the 150 established vascular anomaly centers, but that second wave of academic medical centers that has a higher concentration of patients, so that's going to be the second wave and then obviously from a targeting perspective those physicians and sites that maybe only have call it one patient that's diagnosed those, we will pursue those in a capital efficient and thoughtful manner, and there are some strategies that have been articulated by our commercial advisers in terms of how to go after that smaller percentage of the the payer front, we can share that we've engaged very experienced pricing and reimbursement advisors with deep experience in the rare disease space, having worked closely with companies like Crystal Biotech. There's a lot of things we believe that go into pricing, but most importantly, let's just talk about the fundamentals of microcystic one, this is a serious rare genetic disease where when these patients have incidence of lymphorhea, they can be hospitalized. They can have acute cellulitis, sepsis. Talking about a very serious disease and one that we think payers should be motivated to see these patients do two, there are no FDA approved therapies. I think precedents will show that companies that launch into diseases where there are no FDA approved therapies are able to command orphan pricing when you have those two dynamics. At this point in time, we have not established thresholds of efficacy and what the resulting price pricing range would my experience, typically what we would do is we would take the totality of the phase 3 data and then we would work closely with our pricing advisers and do some third-party sort of blinded payer work to help then establish what we price point would be for QTORIN and rapamycin in the US. So, as we sit here today in May of 2025, we're about two years away from commercialization. I think the most important factor on making sure that we have an exceptional commercial launch, if we're approved, is securing the right leader that's done this before, done it successfully, and can guide these really important decisions, Annabelle, that you're asking about on this call. Great. Thank you so much. Operator One moment for our next question. Our next question comes from Louise Chan with Scotiabank, your eyes open. Hi, thanks for taking my questions here and congratulations on all the progress this quarter. So I wanted to ask you how you think about the market size of the two assets that you will disclose later this year versus the MLM and CBM then do you plan to commercialize your pipeline on your own? I know there's only a few centers to target and the second wave of centers that you mentioned, but would you consider a global partner to help you in the US and outside the US? Thank you. Wesley Kaupinen Yeah, great. So, on your second question, Louise, and thanks for being on the call, on your second question, we are going to prioritize stand-alone US launch for all of our products in the rare genetic skin disease space. I think we've all seen there's been a number of successful rare disease launches do everything we can to learn from those launches. What are the best practices and incorporate them into our launch in terms of outside the United States, those are valuable markets we believe for QTORIN and Rapamycin as look, as any thorough company would do at both stand-alone launches in Europe and Japan. We'll also look at partnering, I think, where we stand today. We want to stick with our core competencies, which is really focusing on resources on the US, so it's most likely that we'll prioritize partnering those those again, I think if we're doing this in a thorough way, we'll look at both launching alone and partnering, albeit with the bias, to leverage others' footprint in places like Europe and Japan to terms of how we think about our next indications for QTORIN and Rapamycin, as well as our next QTORIN platform, maybe we'll just start with the criteria of serious, rare. And nothing approved. We really want to be is part of the fiber of this company and the entire management team is being first for these patients. We also think that has the benefit of by definition, being commercially attractive just to give a little bit of guidance, so we're answering your question. Typically we prioritize those diseases that are rare, not ultra rare. So it's unlikely that you'll see us in, these ultra rare indications with less than 2000 tend to gravitate towards those diseases that have, 10,000 or more patients. We think that those are commercially attractive in nature and look forward to providing you and everyone else with more detail when Jeff makes his big reveal later this year. Thank you. Operator One moment for our next question. Our next question comes from Angela rang with Canaccord Genuity. Your line is open. Congrats on the quarter. This is actually Juan Kim. I'm for Whitney. Thanks for taking our question. Maybe just a quick one from us on phase 3. It was very encouraging to see that enrollment had completed so quickly. And so any additional color on how many of those patients are between the 3 year to 5 year old range and as a follow up, can you remind us how you intend to use that data, whether you'll be submitting a cut of that data concurrently with the initial filing, or do you expect that they will be submitted at a later date for potential label expansion? Thanks so much. Wesley Kaupinen Juan, thanks for your comments about the efforts of our clinical operations team and sites and investigators. We're similarly encouraged to have exceeded our enrollment target. We look forward to providing much more information at the point of full enrollment and beyond in terms of the breakout of the patients who are greater than six, as well as this additional cohort of 3- to answer your question from a regulatory perspective. We are going to concurrently include data from 3- to 5-year-olds in our NDA submission. We think that that's the best strategy, assuming that we're seeing a consistent safety and efficacy profile between those two cohorts, the 3- to 5-year-olds and then the six and so the strategy there would be to concurrently submit that data, keep our NDA timelines on track, and obviously the goal here is to one, be able to serve this disease down to the age of 3, but 2, getting that broader label should enrich the commercial opportunity because we're able to do patients from 3 to 5, and as we've seen from the poster at SID, there's about 1,500 new patients coming into the pool per year. Great, thanks so much. Operator One moment for our next question. Our next question comes from Ananda Gos with AC Wainwright. Your line is open. Hey, hi, good morning. Maybe switching gears. I just wanted to get, both MLMs and CBMs have been notoriously defined as, like the patient with, a lot of heterogeneity in terms of, the disease pathogenesis as well as in terms of the how you know if you can if you can shed light on the recruitment, the patient recruitment aspect in terms of, what were the screening techniques you have used to maximize the utility of the phase 3, that would be very helpful as well as for the. Wesley Kaupinen Yeah, thanks for the question. In terms of patient screening for the phase 3 study, we've enriched that study for patients that are that are determined to be moderate to severe at baseline. That's patients in the trial that are that are less severe, it can be hard to show a delta or separation, show a treatment effect. So that's one point of enrichment. We also have a number of techniques or controls in our protocol to make sure that we're getting the right patients into the study, and these rare disease studies where you have, sample size like our Selva study of 40 or more patients, every patient we go to great lengths in all of our rare disease studies to have strict criteria, as Jeff mentioned as it relates to patient inclusion exclusion. On the phase 2 study, I'll pass it over to Jeff to talk about how we're optimizing for the right patient population for that trial. Jeffrey Martini Yeah. Thank you. So as a reminder, the phase 2 TIVA study is our first study in this disease. We'll be looking at safety and efficacy and then looking at a number of different end points to see how the patients potentially respond to repeating a lot of what we did in MLM, which was successful, which was number one, training all the clinicians as they as they start the study. I'm involved in training all those patients that are of moderate or worse severity, so we've been rich for disease severity there, and then they go through as well an external third-party check just to confirm eligibility and make sure that these are the right patients for the trial. Great, thanks. Operator One moment for our next question. Our next question comes from Dev Prasad with Lucid Capital Markets. Your line is open. Hi, thank you for taking my question. Could you talk about steps and timeline to NDA submission?Once top line data is released in first quarter of next year, additionally, do you need to do some additional work around 505B2 submission package? Thank you. Wesley Kaupinen Yeah, hey, thanks, Deb, for those questions. So our goal after topline data in Q1 of 2026, that data will be presented on a top line basis to the FDA, and then we expect to submit the NDA in the second half of 2026 that puts us on an approval trajectory assuming those timelines are met or exceeded, which is our goal of having the drug approved in Q2 of top line dev Q1 of next year and then. In half of next year to have that NDA submitted, we will, as we're modeling this out and looking at timelines, we will be eligible for priority review. That's as a function of both breakthrough designation and fast track we expect potentially a six-month priority review based on having those designations in hand in terms of the 505B2 pathway. That's really a pathway that's designed to streamline the review process. We're able to reference existing data in our case, the reference listed oral rapamycin or oral (serolimus), so we've got to have the right advisors assembled who are familiar with 505b2 submissions. I've done those before many times and done them successfully, and we expect to work closely with those advisors so that we have a detailed list of every item that needs to be submitted in the 505B2 pathway. But overall we think that that having 5055B2 augment breakthrough, fast track, and orphan is favorable for the program. Good, thank you. Operator One moment for our next question. Next question comes from Catherine Novak with Jones trading. Your line is open. Hi, morning. Thanks for taking my question. I just want to ask a little bit about the difference between the scales, phase 2, phase 3. The physicians not have to reference photos in phase 2. And as well, do you have confirmation from the FDA that this new scale satisfies the need for static measures of LMs as well as the improvement relative to baseline. Thanks. Wesley Kaupinen Great. So, to answer your first question, in phase two, physicians were able to access the photos that were baseline photos, but they were not required to in the protocol. Given that we extended Catherine treatment duration from 12 weeks to 24 weeks, we felt that the addition to the protocol of requiring the physicians at that primary endpoint visit, which is a live clinician 24 weeks to reference back to that baseline photo, we think that that added an important layer of objectivity to that assessment. So that was the change that we supported, and we think strengthened the data. It also helps to mitigate recall bias when a physician is trying to recall what a patient's status was or lesion's severity was 24 weeks I'm going to pass it over to you to answer the second part of Katherine's question. Jeffrey Martini Yeah, thank you, Katherine Jeff. So we've had extensive, conversations with the FDA on the primary endpoints, starting with the breakthrough therapy designation, we presented the clinician interviews from the trial as well as clinician testimonies, and this was the most sensitive end points. Subsequent to that meeting, we did apply for the FDA breakthrough therapy designation. And that was or not, the FDA orphan drug grant which we were awarded, and overall, we are aligned on the end points with the FDA. Got it. And then, I think you just you had disclosed your power and assumptions that it could detect a 0.5 point change on the scale, is that correct? Is that is that meaningful? Or, what, to what extent would an improvement be considered meaningful to these patients? Wesley Kaupinen Yeah, so, Katherine, on that question, in rare diseases, typically you're not defining minimal clinically important differences on a pre-specified basis. I think what you're referencing on the 0.5 what we would need to see to achieve the lower threshold of statistical significance. So, zero on our scale, which goes from negative three very much worse to plus three very much improved. Zero is no change with 40 patients in the study, if the mean change is 0.5, which would be significantly below what we saw in phase two, which was about a 2.42 average change. With a standard deviation that's higher than what we assumed in phase two, we'd still be at a threshold of achieving statistical significance according to our biostatistical assumptions. Okay. Got it. Thanks, that's helpful. Operator And I'm not showing any further questions at this time, and as such, this does conclude today's presentation. You may now disconnect and have a wonderful day. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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