Precigen, Inc. (PGEN) Earnings Call Transcript & Summary

Good morning, and welcome to the Precigen PAPZIMEOS approval call. [Operator Instructions] Please note this event is being recorded. I would now like to turn the conference over to Steve Harasym, Head of Investor Relations. Please go ahead.

Thank you for joining us to discuss the U.S. FDA approval of PAPZIMEOS, the first and only FDA-approved therapy for the treatment of adults with recurrent respiratory papillomatosis, or RRP. With me are Helen Sabzevari, the President and CEO of Precigen; Phil Tennant, our Chief Commercial Officer; and Rutul Shah, our Chief Operations Officer. Before we get into this call, I would like to remind you that we will be making certain forward-looking statements. These statements are based on our current expectations. We encourage you to review the slide in this presentation and our most recent SEC filings, which include information that could cause actual results to differ from those in the forward-looking statements. After the prepared remarks, we will open the call to Q&A. With that, I will now turn the call over to Helen. Helen?

Thank you, Steve, and a warm welcome to all participating in call today. Yesterday was a day of great significance for all those in RRP community, treating physicians and the RRP foundation. For the first time since RRP was recognized as a distinct disease, there is now an FDA-approved therapy for adults. We are thrilled to announce that PAPZIMEOS has been approved for the treatment of adults with RRP. It is the first and the only approved therapy for RRP and the first and the only approved therapy that treats the underlying cause of the disease. As you all recall, Precigen completed submission of a rolling BLA in December 2024 under an accelerated approval pathway. I'm excited to announce that the FDA has granted PAPZIMEOS a full approval. With this full approval, the confirmatory study is no longer required. The approval of PAPZIMEOS also signifies the transformation of Precigen to a commercial stage company. Bringing this medicine to market with utmost efficiency and agility is an incredible accomplishment. I would like to send my profound gratitude to all those involved in this achievement. I will now take a few minutes to briefly give an overview of this rare disease. RRP is a rare, difficult-to-treat and potentially life-threatening chronic disease of the respiratory tract caused by either the HPV 6 or HPV 11 infection. Until now, RRP was mainly managed by repeat surgical interventions. This disease affects both adults and children and can cause severe voice disturbances, compromised airway, fatal pulmonary lesions and invasive cancers. Based on the extensive analysis of claims data, we anticipate a patient population of 27,000 adult patients in the U.S. alone. Given this patient data and the broad label, we believe we have a significant opportunity to maximize our reach and impact adults with RRP. PAPZIMEOS has potential to define a new treatment paradigm for this disease. Let's look at the prescribing information. We are excited to announce that we have received full approval for PAPZIMEOS, which eliminates the need for the confirmatory trial. This is a testament to the clinical data, including a strong efficacy and durability and a favorable safety. In addition, we received a broad label, which makes all adult RRP patients eligible for PAPZIMEOS and positions PAPZIMEOS to become the standard of care treatment in this patient population. This broad label that covers all adult RRP patients will allow treating physicians to intervene at an earlier stage of the disease and potentially reduce the risk of irreversible damage that occur from repeated surgeries. PAPZIMEOS is a non-replicating adenoviral vector-based immunotherapy. It is administered via subcutaneous injection of 4 doses over a 12-week interval. Importantly, the safety profile is extremely favorable. PAPZIMEOS was well tolerated with no dose-limiting toxicities and no treatment-related adverse events greater than Grade 2. Here, I will take you through the pivotal data that led to the approval of PAPZIMEOS. PAPZIMEOS' mechanism of action targets the root cause of RRP. Upon subcutaneous administration, PAPZIMEOS acts by generating an immune response directed against HPV 6 and HPV 11 infected papillomas in the patients with RRP. 51% of those treated met the primary endpoint of a complete response. This is defined as no need for surgical intervention for a minimum of 12 months following the completion of PAPZIMEOS dosing. In addition, complete responses have been durable and a vast majority of complete responses are ongoing. Specifically, 15 of 18 complete responders remained in an ongoing response at 2 years. We plan to present additional data at an upcoming medical conference showing ongoing durability of responses up to 36 months. PAPZIMEOS induced HPV 6, HPV 11 specific T cell responses in RRP study patients with a significantly greater expansion of peripheral HPV-specific T cells in responders compared with nonresponders. I would now like to turn the call over to Phil to walk us through our commercial strategy and launch plans. Phil?

Thank you, Helen, and thank you to everyone listening in on this historic day. Let's turn to what makes PAPZIMEOS a compelling opportunity for this rare disease population. This approval positions PAPZIMEOS to become the new standard of care. Importantly, the product has a broad label, covering adult patients with RRP and it demonstrates durable complete responses. In fact, as mentioned, the vast majority of complete responders remain in complete response for 2 years, which speaks to the long-term efficacy and potential for sustained treatment impact. From a commercial standpoint, the value proposition for PAPZIMEOS is extremely compelling. We are servicing a concentrated patient population. Integrated delivery networks, or IDNs, and community hospitals account for over 90% of patient potential. And this allows us to focus our commercial efforts efficiently and support early adoption. In summary, PAPZIMEOS combines its status as the first and only approved therapy for RRP with clinical durability and a targeted commercial footprint, making it a compelling breakthrough for RRP patients and the broader health care system. Let's talk about launch execution, where we stand today and why we are confident in our ability to scale quickly. We know that there is demand for a therapeutic treatment option that addresses the underlying cause of the disease, and we are ready to meet the demand with the launch of PAPZIMEOS. We've built a robust commercial infrastructure to support PAPZIMEOS. Our 18 dedicated sales territories cover over 90% of the ENT patient potential. This footprint ensures our ability to reach the highest volume of prescribers at launch. Our field teams, including medical science liaisons, payer engagement specialists and sales leadership have been in place since April and making substantial progress in building key relationships. We've completed targeting across IDNs, community hospitals and other health care providers, covering over 90% of the account, patient and procedural potential. Pre-approval information exchange or PI presentations with population health decision-makers have also laid the groundwork for market entry. We've also built a strong base of national and regional thought leaders to champion early access. And our commercial distribution channels, patient hub services and support platform, papzimeossupport.com, are fully operational to support launch. In addition to the online portal, we have established a dedicated support line for providers and patients to be able to call. To put this in perspective, 93 IDNs and community hospitals account for 80% of our target potential and our total IDN-community hospital universe includes approximately 500 accounts. We're also supporting formulary and P&T processes across payers and IDNs. As we look ahead, we've outlined a clear framework for tracking the commercial success of PAPZIMEOS in several key areas, and some of those critical areas are shown on this slide. We have begun and will continue to monitor national and regional KOL advocacy and support for PAPZIMEOS. We'll be tracking detailed prescription metrics at the individual and institutional levels to understand the breadth and depth of utilization. In addition, we'll be paying close attention to market access metrics to understand the proportion of lives who are increasingly covered with favorable access. And finally, we will utilize our hub to provide the data we need to ensure all eligible patients are securing the support they need to access the treatment. Together, these metrics form the backbone of our commercial dashboard and will guide investor expectations as we build towards sustained revenue growth. On the subject of patient support, we're not just launching a product, we are delivering a full service experience designed to accelerate adoption and ensure continuity of care. From a pricing perspective, we are priced competitively and the price point is representative of the innovation and value PAPZIMEOS brings to the market as the first and only approved treatment. Our support program is built to remove barriers for both health care providers and patients. It includes treatment education, insurance navigation and order coordination through specialty pharmacy services. These resources streamline the onboarding process and reduce administrative friction for prescribers. We've also prioritized financial assistance and coverage support. Patients receive help understanding their insurance benefits and accessing affordability programs, which is critical for ensuring adherence. For providers, we offer dedicated support through the treatment journey from education to logistics, making PAPZIMEOS easy to prescribe and manage. This infrastructure is not just about access, it's about scalability. By enabling smooth engagement across IDNs, community practices and payers, we're positioning PAPZIMEOS for sustained growth and long-term market leadership. With that, I will now turn back to the operator.

[Operator Instructions] With that, our first question comes from the line of Jason Butler with Citizens.

Congrats on the approval. Two for me. First, sorry if I missed this, but what is the list price of the drug? And then secondly, the RRP Foundation has a database of a large number of -- sorry, RRP patients. I guess to what extent have you been able to leverage this already? Or can you leverage this in the future to help refine patient and physician targeting?

Jason, thank you for the questions. I think in regard to the pricing, the first question, I'm going to ask Phil to answer and I take the second question.

Jason, thank you for the question. So -- as we all know, it's 4 subcutaneous injections over 12 weeks. And as I mentioned, we've priced it appropriate to the degree of innovation that it's bringing to this market as a rare disease treatment and the first and only approved treatment. So the Wholesale Acquisition Cost or WAC price for each vial of PAPZIMEOS is $115,000. And we believe this will support broad utilization of PAPZIMEOS in adult RRP patients. I'd like to stress, of course, that we have a comprehensive patient support program in place, which does include financial assistance for those that require it, ultimately helping RRP patients access therapy. And we have a clear goal which is to ensure every eligible patient will have access to PAPZIMEOS.

And in regard to the RRP Foundation and the question, as you know we have been extremely sort of active in working very, very closely with the RRP foundation from the beginning of this journey. And -- especially in the past 2 years, as you have seen on RRP day that we have cosponsored with the patient advocacy group and Kim has been a tremendous inspiration and help in advancing this treatment for the patients in an indication that there has not been any approved therapy for -- let's think about this, 100 years, for the past 100 years, people have known about this disease and the therapy, believe it or not, from a 19th century, the 20th century to 21st century has been the same, repeated surgeries which cause the damage. So we are working very closely with the patient advocacy group and of course, in establishing the not only making sure all patients have access, but also from perspective of the data bases that can be very, very useful across the United States to identify the patient and to ensure that all patients have access to this treatment. And we look forward to a continuous work and excellent work that patient advocacy group has done in this manner.

Great. And just to clarify, on the price, it's 4 vials per course of treatment, correct?

Correct.

And your next question comes from the line of Jennifer Kim with Cantor Fitzgerald.

Congrats again on the approval. Maybe to start on the broad label, and this might be a question for Phil. What are the expectations in terms of market access based on your conversations with payers in terms of treatment to trial criteria versus treatment to label? And then my second question is, just looking at the label, are there any limitations around redosing? I know that was a consideration for the confirmatory trial, but that's not required any longer.

Thank you, Jennifer. Thanks for the question. Yes, in terms of discussions with payers, as you would imagine, we've had extensive discussions with them. And there's a high degree of anticipation. I mean, fundamentally, they understand the unmet need. They understand the inappropriateness of surgery as a long-term solution for these patients. They're impressed by the clinical efficacy and the value that this drug will bring as the first and only treatment. So we expect in line with our label that every patient is in a position to benefit from PAPZIMEOS, and we'll be working with payers to secure broad access accordingly.

And Jennifer, in regard to the redosing, as we have been communicating, the FDA has been extremely interested in these aspects. Our pivotal study included obviously a single course of 4 subcutaneous injections of PAPZIMEOS, which could make a lot of scientific sense here to redose the patients to further elevate the immune responses against papillomas to potentially improve the responses in partial or nonresponders and eventually to extend the duration in the complete responders. As published in Lancet Respiratory Medicine, 86% of our patients had a clinical benefit in reduction or elimination of their surgical burden in 12 months after treatment. So based on that, we have a very -- and based on the safety also and the efficacy that has been seen from our pivotal study, redosing makes a lot of sense. And at this point, it will be for physicians to evaluate patients and decide on redosing.

And your next question comes from the line of Swayampakula Ramakanth with H.C Wainwright.

Congratulations. I know this is a great -- it's a big win for you folks and also for the patients. A couple of quick questions. So Phil, are these vials a single-use vial or can it be multiple use? And then also in terms of the launch itself, is it going to be in some sort of waves in the sense, initially you reach out to the IDNs and then to the community hospitals and so forth? Or is it going to be a complete launch across the different segments of the patient population?

Yes. Thanks, RK. So firstly, to the first question, yes, single-use vial. And regarding the launch, no, I wouldn't say it's going to be in waves. We've clearly identified over 90% of the patients and the procedural concentration in the IDNs and the community centers. So our initial footprint includes both. But we do know that the patients are concentrated in those centers. They've been on a tremendous journey for many years and ultimately end up having surgeries at these hospitals. And as I mentioned earlier, of the 500 or so IDNs and community practices that are in the total target list, there are just under 100 of those that represent the vast majority of the potential there, and that's where we'll be focusing our initial work.

Thank you very much, RK. So with that, I'd like to thank you for taking the time to join us today. We are profoundly grateful to the NIH clinicians, the FDA and most importantly, the patients and families whose trust, commitment and perseverance made this breakthrough possible. The FDA approval of PAPZIMEOS marks a truly historic milestone for the RRP community, delivering the first and the only approved therapy for adults with this rare, debilitating and potentially life-threatening disease. With an estimated 27,000 adult patients in the U.S., this approval represents a fundamentally new era of treatment. This is a proud day for Precigen, especially our team who have worked so hard to bring this to reality. We look forward to swiftly delivering PAPZIMEOS to physicians and patients and to continuing our mission to advance meaningful innovations for those with high unmet needs. Thank you again for participating.

Thank you, presenters. And ladies and gentlemen, this concludes today's conference call. Thank you all for joining. You may now disconnect.