Palvella Therapeutics (NASDAQ:PVLA) held its second-quarter earnings conference call on Tuesday. Below is the complete transcript from the call.
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Summary
Palvella Therapeutics reported significant progress in the development of QTORIN rapamycin, achieving key milestones such as compelling Phase 3 SELVA study results, a pre-NDA meeting with the FDA, and receiving Rolling Review designation from the FDA.
The company is on track to complete the NDA submission for QTORIN rapamycin in 2026 and anticipates FDA approval in the first half of 2027. They have recruited experienced leaders to ensure successful U.S. launch readiness.
Palvella is developing therapies for multiple rare skin diseases and vascular malformations, aiming to introduce first-in-disease therapies. They estimate a significant addressable market for each condition, with potential orphan drug pricing.
QTORIN rapamycin showed strong results in clinical studies with high efficacy and a favorable safety profile, positioning it as a potential first-line treatment and future standard of care for microcystic lymphatic malformations.
The company raised $230 million in capital, strengthening its financial position to support pipeline advancement and commercial readiness. Palvella remains well-capitalized through potential FDA approval and commercial launch.
Palvella’s strategic focus includes expanding QTORIN rapamycin to other indications, leveraging Fast Track and Breakthrough Therapy designations to expedite review processes.
Management highlighted the importance of ongoing physician engagement and patient services infrastructure to support treatment initiation and access post-approval.
Full Transcript
OPERATOR
Good day and thank you for standing by. Welcome to the Palvella Therapeutics Second Quarter 2026 Financial Results Conference Call. At this time, all participants are in a listen-only mode. After the speaker’s presentation, there will be a question-and-answer session. To ask a question during the session, you will need to press star 1-1 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 1-1 again.
Please be advised that today’s conference is being recorded. I would now like to hand the conference over to your first speaker today, Marcie Nanis, Vice President of Investor Relations and Corporate Affairs.
Marcie Nanis, Vice President of Investor Relations and Corporate Affairs
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 Koppanen, 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.
Wes Koppanen, Founder and Chief Executive Officer
Thanks, Marcie. 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 two 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 three important milestones. First, the compelling safety and efficacy results from our Phase 3 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, number one, we remain on track to complete our NDA submission in the second half of this year and, number two, 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.
We are developing therapies for four 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 multibillion-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 LM, 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 Therapeutics, 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. Finally, physician market research further reinforces the potential for an attractive uptake curve at launch across all four 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 two 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 pre-launch 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 LM 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 salesforce 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 one, 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 LM based on three 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 3 SELVA study delivered highly compelling results. The study met its primary endpoint, key secondary endpoint, and all four 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 LM 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 LM 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 standalone 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 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 3 and Phase 2 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.
Matthew Korenberg, Chief Financial Officer
Thanks, Jeff. As of June 30, 2026, Palvella Therapeutics 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 de-risk and strengthen our commercial launch. 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 3 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.
Wes Koppanen, Founder and Chief Executive Officer
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
Thank you. At this time we will conduct the question-and-answer session. As a reminder, to ask a question, you will need to press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. Please stand by while we compile the Q&A roster. Our first question comes from Alexa Deamer at Cantor Fitzgerald.
Alexa Deamer, Analyst at Cantor Fitzgerald
Hi, guys. This is Alexa Deamer 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? Thanks so much.
Wes Koppanen, Founder and Chief Executive Officer
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’s greater than 30,000 diagnosed patients in the United States with microcystic lymphatic malformations. And 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
Thanks so much. Our next question comes from Whitney Ajum at Canaccord Genuity.
Whitney Ajum, Analyst at Canaccord Genuity
Hey, good morning, guys. Thanks for taking the questions. Just first one, on DSAP phase two, 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?
Wes Koppanen, Founder and Chief Executive Officer
Yeah. 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 two study, we expect that to be about a 15-patient phase two study. Will it proceed more quickly than angio? To speak about angio for one minute, we did start that trial ahead of original expectations. That trial started in the first half of this year. Original 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 2 study, which we expect to be sometime in the second half of this year.
Whitney Ajum, Analyst at Canaccord Genuity
Got it. That’s helpful. And then just a quick follow-up. We conducted 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 ZSTOCs who were actively seeking treatment for their MLM, with the main reasons why patients were not seeking treatment being just kind of like comments around not bothersome or 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? Thanks.
Wes Koppanen, Founder and Chief Executive Officer
Yeah. So our claims data show that there were 45,000 to 95,000 patients in claims data. 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 that approach applies to patients who may be less burdensome from a symptom perspective, because if the disease, unless it 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
Great, thanks. Our next question comes from Ritu Baral at TD Cowen.
Ritu Baral, Analyst at TD Cowen
Good morning, guys. Thanks for taking the question. 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.
Wes Koppanen, Founder and Chief Executive Officer
Hey, Ritu, thanks for the questions. To address your question on where the reps will be focused, we think of our market as three 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 anomalies 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 one, which is the high-volume centers, we will also have personal promotion with the reps into our tier two and our tier three. 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 two 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 one of 400 centers, but also the tier two and the tier three.
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 Dompé 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, Analyst at TD Cowen
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 practices to help? And as you think about your pricing and your first insurance conversation, 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?
Wes Koppanen, Founder and Chief Executive Officer
Yeah, 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 Crystal Biotech. He’s working closely with Jennifer McDonough who was also previously at Crystal. We’re in the process of ensuring that 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 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 three 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, Analyst at TD Cowen
Would prior auths really just be diagnosis?
Wes Koppanen, Founder and Chief Executive Officer
Yeah, 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 three precedents we mentioned to Pezov, Ijavec and Oxurvate that we can model.
OPERATOR
Our next question comes from Annabel Samimy at Stifel.
Annabel Samimy, Analyst at Stifel
Hi all, thanks for taking my question. 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 75k 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?
Thank you.
Wes Koppanen, Founder and Chief Executive Officer
Yeah. Hey Annabel, thanks for the questions. Really appreciate you asking about the size of the CVM market. What I 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 analyses 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 into the filing as well as some of the additional opportunities to share data from the SELVA study.
Jeff, UNKNOWN
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 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. MPK and other data will be coming out at future medical congresses.
Annabel Samimy, Analyst at Stifel
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 three trial design will look like and if you have to have a placebo control arm, what are your prospects for enrollment now that the data is out and they see that it’s a very effective drug?
Wes Koppanen, Founder and Chief Executive Officer
Yeah, 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-two meeting and to align on a phase three 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 two 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 two 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 Greg Souvenue at Mizuho.
Ryan, Analyst at Mizuho
Hello, this is Ryan on for Greg today. Thanks for taking the question, maybe just the first question focusing on angiokeratomas, maybe for Jeff, can you talk a little bit how LO2 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 angiokeratoma program so far and the level of awareness that investors have regarding overlap with these other conditions?
Thank you.
Jeff, UNKNOWN
Thanks for the question, Ryan, this is Jeff. So the status is, you know, 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’re seeing these patients in their clinic and they’re not wanting to do some of the destructive procedures that I 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 malformations. They have some of the same molecular markers.
There’s 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.
Wes Koppanen, Founder and Chief Executive Officer
Yeah, 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 clarity, awareness rise is when you run these studies, particularly phase two studies, and if you’re fortunate enough, like we’ve been fortunate in microcystic LM 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. You know, Jeff did a great job, I thought, walking through the three 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, Analyst at Mizuho
Great. 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?
Wes Koppanen, Founder and Chief Executive Officer
Yeah, thanks, Ryan, for the question. We followed others who have secured this FDA’s platform designation and similar to some of these other designations that we’ve secured. You know, 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 and 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, Analyst at Raymond James
Hi, good morning. 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? Thanks.
Wes Koppanen, Founder and Chief Executive Officer
Yeah, 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 Tree presented at ISVA and Jeff highlighted on this call.
Ryan Deschner, Analyst at Raymond James
Thanks Wes.
OPERATOR
Our next question comes from Sam Slutsky at Lifeside Capital.
Sam Slutsky, Analyst at Lifeside Capital
Hey, thanks for taking the questions. 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 extended body surface area in MLM patients and kind of expectations for tube size and what it could cover, etc.
Matthew Korenberg, Chief Financial Officer
Hey Sam, thanks for the question on pricing analogs. We have three of those listed in our corporate deck: Topeza, Oxyrvate, and Erricase. We’ve 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, we’ll pass it over to Jeff.
Jeff, UNKNOWN
Thanks again for the question on BSA and tube size. So microcystic lymphatic malformations are caused by somatic mutations in PIK3CA, which lead to mTOR overactivating 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 cm^2 and 200 cm^2 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 cm squared. 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.
Alex, Analyst at Truist
Hey guys, this is Alex on for Danielle. Thanks for the question on the upcoming end of Phase 2 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 2 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 2 meeting.
Thanks so much.
Wes Koppanen, Founder and Chief Executive Officer
Yeah, Alex, thanks for the question. The absence of breakthrough does not impact how we think about the end of Phase 2 meeting. We have a drug that in Phase 2 had a large effect size and a serious, rare, progressive disease where there’s no FDA-approved therapies. I think one of the keys for the Phase 2 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 3 study.
We do know, thanks to the Fujino publication out of Japan, that there is no documented spontaneous regression 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 Designations, 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.
Thanks so much.
OPERATOR
This concludes the question and answer session. I would now like to turn it back to Wes Koppanen for closing remarks.
Wes Koppanen, Founder and Chief Executive Officer
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 Therapeutics. 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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