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

Good afternoon, ladies and gentlemen, and welcome to the Precigen Third Quarter 2025 Financial Results and Business Update Conference Call. [Operator Instructions] This call is being recorded on Thursday, November 13, 2025. I would now like to turn the conference over to Steve Harasym.

Thank you, operator, and thank you to all those joining us for our third quarter 2025 update call. Joining me today are Helen Sabzevari, our President and CEO; Phil Tennant, our Chief Commercial Officer; Rutul Shah, our Chief Operating Officer; and Harry Thomasian, our CFO. Before we begin our prepared remarks, I remind everyone that we will be making certain forward-looking statements. These statements are based on our current expectations and beliefs. We encourage you to review the slide in this presentation and in our SEC filings, which include risks and uncertainties that could cause actual results to differ materially from today's forward-looking statements. With that, I will now turn the call over to Helen. Helen?

Thank you, Steve, and thank you to all those joining us for our third quarterly update call. The approval of PAPZIMEOS in August marked a monumental turning point for all those impacted by recurrent respiratory papillomatosis or RRP, patients, families, physicians and the RRP Foundation line. We would like to welcome you to the new era of RRP treatment with PAPZIMEOS poised to become the standard of care. PAPZIMEOS is the first and only available treatment for adults with RRP, and it represents the best data, and that's by a wide margin ever generated in adults with RRP. Why is PAPZIMEOS a groundbreaking therapy? Let's look at the facts. First, PAPZIMEOS addresses underlying root cause of RRP by generating an immune response against HPV-6 and 11 infected papilloma cells. Secondly, PAPZIMEOS has demonstrated transformative clinical benefit. What do I mean by that? 51% of patients achieved complete response, requiring no surgery for 12 months post treatment with the durability shown in 15 of 18 complete responders remaining surgery-free at median duration of 3 years without any additional treatment. Also, overall, 86% of our patients had reduction in their surgical burden after PAPZIMEOS treatment. PAPZIMEOS has a very favorable safety profile with nothing greater than grade 2 TRAs, which are similar to those of all receiving a flu vaccine, for instance. Also, the ease of administration of PAPZIMEOS, it's given as a subcutaneous administration that can be administered at any clinic or any of the physician offices. Furthermore, PAPZIMEOS is not associated with a painful device necessary for administration. Let me be very clear here. We have treated the most severe RRP patients and demonstrated unmatched complete response rate, which has been durable with excellent safety profile. Based on the RRP pathology, it is easy to extrapolate PAPZIMEOS results to a less severe patient population, which has been reflected in FDA's review and subsequent grant of a broad label for all adult RRP patients irrespective of severity of their disease. In contrast, it is very difficult to extrapolate the results achieved in a less severe patient population to a more severe RRP population as is the case with a competitor. I would like to emphasize that PAPZIMEOS's pivotal study is the first and to date, the only clinical trial in RRP conducted with this robust, prospectively defined statistical primary endpoint. PAPZIMEOS clinical data not only beat the high statistical bar set for the pivotal study using the most robust clinical efficacy endpoints ever evaluated in RRP, it furthermore demonstrated the strongest data shown to date for RRP. In summary, PAPZIMEOS was granted full approval by the FDA with a broad label of adult RRP that does not include restriction on a number of prior surgeries. This is a testament to the transformative clinical data that include unmatched efficacy and a strong ongoing durable responses from a pivotal study with a prospectively defined statistical primary efficacy endpoints of complete response rate. In addition, due to the mechanism of action of PAPZIMEOS, there is an opportunity for redosing of PAPZIMEOS. And with full approval, we have significantly raised the bar for clinical data for any competitor to enter the adult RRP space in the future. This approval also marks a pivotal transition for Precigen, propelling the company into a commercial state. Delivering this transformative therapy to market with exceptional speed and agility is a remarkable achievement. In the short time since approval, we have made great strides towards recognizing the robust commercial opportunity and building the strong foundation for PAPZIMEOS to be the new standard of care treatment. As always, our dialogue with the FDA continues to be very productive, including the completion of a successful post-approval meeting. We are currently working towards initiation of PAPZIMEOS clinical trial for the pediatric RRP population. In addition, we have initiated our efforts for geographic expansion of PAPZIMEOS. With that in mind, I'm pleased to announce that we have submitted marketing authorization application with the EMA. I will now turn the call over to our Chief Commercial Officer, Phil, to walk us through our commercial development. Phil?

Thank you, Helen, and I am delighted to share with you all today the exciting progress we are making with the launch of PAPZIMEOS. We've achieved a lot in a relatively short space of time. As a reminder, the approval in mid-August was the trigger to bring the full sales team of 18 key account managers on who were hired, onboarded and deployed in September. In the few weeks since full team deployment, we have made great progress towards our goal of quickly establishing PAPZIMEOS as the new standard of care for adults with RRP, so let me highlight the key achievements to date. Firstly, the drug is available and has started shipping to prescribers in the U.S. for the treatment of all adults with RRP. Our field team has now engaged with 90% of our target institutions, which cover a significant portion of the 27,000 adult patients with RRP. These engagements are focused on supporting and expediting the formulary inclusion process, and we have been very impressed by the enthusiasm of the HCPs in accelerating that process. We have already seen multiple formulary approvals nationally. We're also working with HCPs in those institutions to enroll patients who are waiting for treatment. To that end, we have over 100 patients registered in our Precigen patient services hub, and a significantly larger number also being processed through institutions own patient services teams. In line with our expectations, there is clearly pent-up demand at these hospital systems that is now being processed for treatment with in PAPZIMEOS. Pleasingly, it's not just the large academic sites or IDNs that are expressing an interest PAPZIMEOS. We're also making good progress with a number of community practices to expedite product uptake, including some of the super groups affiliated to ENT and oncology networks around the country. This clearly reinforces what we heard in our market research ahead of launch, and we're now seeing in practice. There is a strong preference for PAPZIMEOS due to its efficacy, durability, safety, and the mode of administration. The drug can be shipped anywhere as well as stored and forward easily. No device needed, no training on device needed, just a simple subcutaneous injection. And as Helen said, no need for painful electroporation. Payer coverage is advancing rapidly. As of last week, over 80 million lives are covered. and a number of other policy updates are expected to be announced in the near future. Importantly, PAPZIMEOS is also covered through Medicare and Medicaid. Suffice it to say, we're extremely pleased with this momentum, which is in line with our expectations. Finally, we're seeing strong support from physicians, whether from a large institution or a community practice, again, reflective of the speed at which our teams have engaged with our target customers. We continue to see strong support and efficacy from the RRP Foundation. We have published important new data regarding the significant burden of RRP, both at the individual level and to the health care system, including the data released this week at the ISR meeting in Europe. These data, coupled with the impressive and evolving durability profile of PAPZIMEOS, is helping to propel us forward as we look to establish a new treatment paradigm. In summary, we are extremely pleased with the progress being made. The market is embracing PAPZIMEOS as expected, and we also expect to further build on this momentum throughout the rest of Q4 and into Q1 2026. I look forward to continuing to share further progress across key indicators of success as we complete Q4 and move into the new year. I will now turn the call over to our Chief Operating Officer, Rutul Shah, to give a brief update on manufacturing. Rutul?

Thank you, Phil, and good afternoon, everyone. I'm excited to share PAPZIMEOS manufacturing operations updates today. As part of our strategic commitment to long-term value creation, we have made significant investments to have control over our cGMP manufacturing operations. We operate a dedicated in-house cGMP facility for commercial PAPZIMEOS drug substance manufacturing. Our facility has been fully operational, had a successful pre-approval inspection by the FDA, and has been manufacturing PAPZIMEOS drug substance since prior to approval. With significant in-house expertise in the production of adenoviral vectors, we are executing on our operational plan to supply PAPZIMEOS to both current and anticipated future demand. I'd like to take the opportunity today to briefly address the cold chain requirement of PAPZIMEOS. We have done our homework in detail regarding this topic, and our interactions indicate no impact on the adoption of PAPZIMEOS. In fact, post-COVID, the significant majority of our target IDNs and community centers are equipped to handle frozen drug products like PAPZIMEOS. We have end-to-end validated logistics in place to distribute PAPZIMEOS efficiently. And as Phil mentioned, PAPZIMEOS drug product is available on the shelf is being ordered and shipped to our customers. With that, I would like to turn the call over to Harry, our Chief Financial Officer, for a financial update. Harry?

Thank you, Rutul, and good afternoon to those participating in this call. Before touching on the quarter, I want to thank our long-term shareholders for providing us the support necessary through the development and ultimate approval of PAPZIMEOS. It has been less than 5 years for this drug to go from the lab to approval, and we could not have achieved this momentous feat without your support. In addition, I'd like to say that I am proud to be part of the team that has provided patients with the first and only therapy targeting the root cause of RRP. Turning to our quarterly financial statements, specifically starting with our balance sheet. At September 30, 2025, we had $123.6 million in cash, cash equivalents, and investments following our recent drawdown of the first tranche of our credit facility which was entered into during the quarter. We expect this balance plus our projected revenues from PAPZIMEOS to fund our operations to cash breakeven, which includes continuing PAPZIMEOS launch costs and further development of our pipeline. I want to pause and repeat this point. We expect that our cash and investment balance plus expected projected revenues from PAPZIMEOS to fund our operations to cash breakeven. We remain confident in Precigen's financial future as we continue to execute on our upcoming milestones. Additionally, on the balance sheet, we ended the quarter with approximately $3 million in inventory, which represents the manufacturing costs that we have incurred subsequent to the approval of PAPZIMEOS. Costs incurred in manufacturing the product prior to the approval have been expensed as part of our R&D expenses. Lastly, during the quarter, all of our preferred shares were converted into common shares, providing a simplified capital structure going forward. In regard to our statement of operations, the one item to note within our operating expenses is the increase to our SG&A costs of approximately $14 million in the quarter ended September 30, 2025, versus the same quarter in the prior period. The majority of this increase was driven by increased commercialization spending related to the PAPZIMEOS launch and to a lesser extent, additional employee-related costs, most of which is attributable to the accounting for share-based awards. Additionally, our net loss attributable to common shareholders for the quarter ended September 30, 2025, includes 2 large accounting-related non-cash items, a change in the warrant liability, and a deemed dividend related to the conversion of our preferred shares. Those 2 items combined represent $0.95 per share of the $1.06 per share loss attributable to common shareholders. We do not expect these 2 items to recur in future periods. For more information on our financial statements, I refer you to today's press release and our 10-Q, which was filed with the SEC after market close this afternoon. I do want to provide certain guidance relating to our gross-to-net revenue adjustment. We anticipate that this adjustment will be in the high teens to low 20%, which is consistent with peers in our industry. Lastly, it has been quite a year at Precigen. As we prepare for the approval of PAPZIMEOS, we made a number of infrastructure investments, including the implementation of a new ERP system this past year. With these investments, we have positioned Precigen with appropriate systems, personnel, and controls to manage the various processes of a commercial company. With that, I'd like to turn it over to the operator for Q&A. Operator?

[Operator Instructions] Your first question comes from the line of Jason Butler from Citizens.

Congrats on the launch. Wondering if you can give us any color on whether any patients have received reimbursement approvals yet, or whether any patients have been dosed with the first dose of PAPZIMEOS? And then a follow-up is, how should we think about the cadence of the pull-through from patients that are now registered in the hub to getting a reimbursed drug?

Thank you, Jason. And I think for the first question, I would refer to our Chief Commercial Officer, Phil. Phil?

Sure. Jason. Yes, Phil here. So, as we mentioned, we started shipping PAPZIMEOS to institutions basically for patients who are being scheduled for treatment as we speak. And as I mentioned, we've got payer coverage coming through thick and fast. So those two things are coming together. And we're not going to go into details about specific patients being dosed at the moment. But I think in Q4, we'll see that come through and we'll be -- when we report our Q4 earnings, we'll be able to talk specifically to numbers of patients dosed and, of course, the earnings and the revenues that go along with that. The second question regarding patients in the hub. Again, those -- they're sitting there now ready for benefit verification and prior authorization. So -- and we're also seeing a whole load of those patients who are not necessarily in our hub but are going through the institution's own patient services systems. So that pull-through will be institution by institution, but we expect that to be starting to pick up the pace as we go through Q4 as these processes come together, both on the institution side and the payer side. But we're very pleased with the number of patients that we're seeing coming into the top of the funnel ready to be activated and treated.

Sorry, can I just squeeze in a quick clarification point there? So, Phil, do you expect the majority of patients that go into one of the hubs to actually become -- to pull through into receiving drug?

That would be our expectation, yes.

And Jason, maybe I can add on these patients, both the ones that are being registered at the PAPZIMEOS hub or at the centers that they have their own registration hub, basically, they are various adult RRP patients that through their physicians have been identified for the treatment and will be joining to receive. So that's very exciting. And I think prior to approval of PAPZIMEOS, our analysis have shown that there will be a large patient population. And as you have seen it, the estimate is 27,000 in the United States. And it's really important to say that we are seeing that kind of a demand coming through from the various centers.

And just to reinforce, Jason, a lot of these centers, they prefer to use their own expertise and systems and patient services initially. Now obviously, if they want to explore co-pay support or free drug support where for appropriate patients, they would need to ultimately come into our hub. So, we've, as you said, sort of got these 2 hub components that are co-existing at the moment, both of which suggest that the pent-up demand that we identified is absolutely there.

Your next question comes from the line of Swayampakula Ramakant from H.C. Wainwright.

Congratulations, everybody, everyone on the team there. It's a great moment, and it's a transitory quarter for you guys. Excellent. So, for my one question, I want to check with Harry regarding the statement saying that you are funded to cash flow breakeven, which is obviously a significant statement you're making. What sort of assumptions are you taking into this in terms of either revenue or patient penetration, how should we think through to get there?

Yes. RK, good to talk to you and appreciate the question. I would say at this point, we're not guiding on revenue. So, it's kind of difficult to say when or how we get to cash flow breakeven. But I think we're willing to state that by the end of 2026, we'll be cash flow breakeven.

Your next question comes from the line of Michael DiFiore from Evercore ISI.

Huge congrats on all the progress here. Number one, just given the obvious pent-up demand and bolus of patients that are expected to go on therapy soon, could you give us any color as to how long that bolus may last, just considering the reimbursement hurdles that any new therapy encounters during the first year? And I have a follow-up.

Well, we do -- we look at analogs of rare diseases and the uptake where you do have pent-up demand. So, we think that's going to last for quite a while. The 27,000 adult patients that we've identified are already there in the system. Obviously, some more severe than others, some see the health care practitioner more often than others. But those are the patients that are actually there in the treatment, and then you would have the incident population on top of that. So, I think this bolus of patients is going to be there for quite a while to come.

And Michael, maybe I can also further add is the importance of our broad label, which covers basically all adult RRP, which means anyone who is also going to be diagnosed immediately, or they had, for instance, even 1 surgery, and it will be continuously added to these hubs for the treatment. And it's very clear, at least from what we are seeing from the patients' enthusiasm as well as the physicians, that based on the data that has been published from Hopkins prior, that basically patients by fifth surgery, they have irreversible damages to their either trachea or vocal cords. Clearly, now the patients as early as their diagnosis, they will be joining. So, the pent-up demand, obviously, all of the severe patients, but also now all of the patients that have been diagnosed or going -- undergoing diagnosis, they are basically eligible to receive the PAPZIMEOS and the physician, actually, this is the importance. And as for instance, if you look at our press release today, Dr. Best, which is one of the renowned physicians for the treatment of RRP in the world, he refers to PAPZIMEOS as nothing short of remarkable data for these patients' treatment of adult RRP and then also positioning it will -- it's poised to become a standard of care, which then covers all that population. And I think that's very important.

Excellent. And just my quick follow-up is just for modeling purposes, how should we think about subsequent cycles of therapy? And would payers even allow subsequent cycles beyond the first 4 doses?

Yes. I think this is an excellent question. First of all, it's very important, and this was one of the interesting concept that FDA has encouraged us very much for redosing of the PAPZIMEOS and for the expansion of that. And the reason has been based on the safety, obviously, the efficacy and the durability of the response results that we have seen. And at the moment, of course, in the label, it's at the discretion of the physicians in order to re-dose. So, if they feel that the patient needs to be re-dosed, they can prescribe to that. And of course, as we are moving forward, we are further generating further data on redosing of the patients. So, I think there is a huge expansion from that side. And one of the other things that we have mentioned is that our partial responders, as I mentioned, 86% of our patients, they reduced their number of surgeries. And it's very, very important, and that was part of the discussions along for our BLA that clearly, they will benefit, it seems, that from the redosing because their immune system is being enhanced to address the root cause of this disease, which is HPV 6 and 11. And I think this is one of the areas that we are also expanding besides our pediatric clinical trials. It's further generation of the data on the dosing of the patients, redosing of the patients, I should say, which is initiated in the next year.

And I would just add from a payer perspective, the one characteristic of the drug that really stands out for them and will support us in any redosing conversations is the durability. So, we started off with the 1 year from our clinical registrational studies. We then get the 2-year follow-up in our label. We've just published our 3-year data. All of this is very important data for the payers. And obviously, we share that with them, build that into our value proposition so that we can support the concept of redosing.

Your last question is from the line of Brian Cheng from JPMorgan.

Congrats on your progress here. I just want to clarify how you record PAPZIMEOS revenue on your financial statements. Do you recognize revenue following each injection or at the end of the 4 injections? And I have a follow-up.

Brian, this is Harry. Thanks for the question. Yes, we recognize revenue when title transfers to -- we're either shipping through a specialty pharmacy or directly to the IDN or the community hospital, and we recognize revenue upon receipt by those entities. So, we don't wait until the injection occurs, which generally is going to be within a day of it being received.

And as we think about the registered patient population within your patient hub and also the institutional patient hub. I'm curious if you can help us think about the size and the trajectory of the registered patients. And then is it safe to assume that all these registered patients will get PAPZIMEOS within a defined period of time?

Yes. I mean, look, our hub and the hubs that we are understanding are being set up and implemented at the institutions, they're recruiting patients rapidly. There's a lot of momentum there, and we would expect that to continue, obviously, with the bolus of patients that we know is out there. They'll be worked through, as we said earlier, those patients are there for a reason because they've been identified for treatment with PAPZIMEOS. And so, we would expect the vast majority of those to ultimately then make it on to PAPZIMEOS. The period of time is difficult to pin down, but there's a high sense of urgency that we have picked up in our interactions with the physician community, whether it's at the IDNs, but actually also at the community level with physicians approaching us and wanting to expedite access at the community level. So, I do think there will be an expedited uptake from the initial pool of patients that we're seeing, but that will continue for quite a while.

Yes. And maybe, Brian, I can add really to what Phil said. I think what we are seeing, especially with the broad label that now we are going to be treating the patients that as early as having 1 surgery or actually just being diagnosed and they have to go through the surgery plus all the other severe patient population that exists there, and they are being scheduled by their physicians. And as we have already knew prior to the approval, but clearly have been seeing it post approval as well from the physicians, physicians do not want to do these surgeries, and they are trying to prevent having these surgeries as soon as possible for these patients because they know that with every surgery, there is basically closer to that damage line of 5 surgeries that causes irreversible damage for these patients. So, I think collectively, when you look at all of that, there is a continuous patient addition and with the understanding that, of course, these patients not only are identified and they will be getting a treatment and they are enrolling as we speak and actually, the prescription is ongoing.

There are no further questions at this time. So, I'll turn the call over back to Helen Sabzevari for closing comments.

Thank you to all those participating in the call today. As you can see, times are very exciting for us at Precigen and for the RRP community as a whole. We look forward to providing you with further updates as our launch progresses. In closing, I would like to leave you with words from a PAPZIMEOS patient who is one of the complete responders from the clinical study. The sound of hope living with RRP in the last 24 years has been the sound of my own voice when I have it. It's being heard even when this virus is trying to destroy my vocal cords. RRP not only took away my voice physically, but also emotionally. So, hope for me has been the moment I've been able to speak, and others have been able to hear me. Power of voices is camaraderie and advocacy, being part of a community that refuses to be silenced. When my voice is weak, others are standing beside me and if needed, speaking up for me. That's power and that's hope. Today, hope sounds different. It's a physician telling their long-time RRP patient, you don't need another surgery. There's something new. Today, the sound of hope is PAPZIMEOS.

Ladies and gentlemen, this concludes today's conference call. Thank you very much for your participation. You may now disconnect.