Good evening, and welcome to the Precigen's Full Year 2024 Financial Results and Business Update Call. At this time, all lines are in listen-only mode. Following the prepared remarks, there will be a question-and-answer session. Please note that this event is being recorded. I would now like to turn the conference over to Steve Harasym from Investor Relations. Please go ahead.

Thank you, Alan, and thank you for everyone joining us this afternoon. With me today are Dr. Helen Sabzevari, President and CEO of Precigen; Harry Thomasian, our CFO; Phil Tennant, our Chief Commercial Officer; and Rutul Shah, our Chief Operating Officer. Before we begin, let me briefly review our forward-looking statements. During today's call, we will make various forward-looking statements. Investors are cautioned that our forward-looking statements are based on current expectations and are subject to risks and uncertainties that could cause actual results or outcomes to differ from those indicated by our forward-looking statements. Please read the safe harbor statement contained in our most recent SEC filings as well as the risk factors contained in Precigen's filings. With that, I would like to now turn the call over to Dr. Sabzevari. Helen?

Thank you, Steve, and thank you to all for taking the time to join us for our year end 2024 update. It is indeed a transformative time at Precigen as we are on the verge of commercializing our lead asset, PRGN-2012 in RRP, potentially bringing a treatment to this patient population with high unmet needs. From discovery in 2020, Phase 1 initiation in 2021, Phase 2 with breakthrough designation, an accelerated approval pathway in 2023, followed by publication in Science Translational Medicine and presentation of the groundbreaking data at ASCO in 2024, this program has advanced with remarkable efficiency and agility. We finished 2024 with submission of our BLA and announced FDA's acceptance with priority review with an upcoming PDUFA date of August 27, 2025. The FDA has indicated that they are not currently planning to hold an advisory committee meeting to discuss the BLA. I would like to now take a few minutes to recap our data, which has recently been published in The Lancet. We are extremely excited about PRGN-2012's potential in RRP to not only be the first but the best-in-class treatment due to significant effects in terms of efficacy, safety, and the ease of route of administration. In our pivotal clinical data, PRGN-2012 demonstrated a statistically significant efficacy. I want to emphasize that our clinical study was designed with a robust, clinically meaningful, and prospectively defined statistical primary efficacy endpoint of complete responses in RRP patients. Our pivotal study met the primary efficacy endpoint with a 51% complete response rate. That was statistically significant and handled pre-specified success criteria established in alignment with the FDA. Complete responses have been durable with median durability of response at 24 months, and all of our Phase 1 complete responders remain surgery-free and in complete response three years later. This is highly significant. The data that was presented at ASCO combining Phase 1 and Phase 2 shows closely overlapping results: in Phase 1 we had 50% complete responders and in Phase 2 52%, for an average of 51% complete response in a prospective manner. Furthermore, 86% of all of our patients had a reduction in the number of surgeries. In multiple publications, we have also shown significant enhancement of HPV-6 and/or HPV-11 T-cell responses, which directly corresponds to the mechanism of action of PRGN-2012 and our clinical responses. The first set of immunologic data was published in 2023 in Science Translational Medicine. We have shown that PRGN-2012, due to the differentiation provided by the gorilla adenovirus, can be repeatedly dosed — not only in PRGN-2012, which has been dosed four times, but also in our other programs such as PRGN-2009, which has been dosed more than 20 times. This platform differs from other viral platforms that allow only a limited number of doses. The gorilla adenovirus can be repeat dosed and continues to lead to enhancement of T cells in the absence of significant neutralizing antibodies. Finally, PRGN-2012, based on all the data and follow-ups, is extremely well tolerated with no dose-limiting toxicity and no treatment-related adverse event greater than Grade 2. The route of administration is a simple subcutaneous injection that can be given in an office setting. We have continued our readiness to hit the ground running post our PDUFA date in August 2025 as we shift from R&D to commercialization, and Phil, our Chief Commercial Officer, will be speaking to that. Finally, I would say a few words about our confirmatory trial, which has already been initiated and has started enrolling patients. Our confirmatory trial, in alignment with the FDA, is single-arm with no placebo control required, with 35 patients to be enrolled, and we are well on our way. Not only have we started enrolling patients but we are moving rapidly to finish this trial. So with that, I will now turn the call to Rutul Shah, our Chief Operating Officer, who will be speaking to our CMC and manufacturing readiness. Rutul?

Thank you, Helen. I would like to take a few moments to discuss our manufacturing strategy and provide an update. As you are aware, we have made significant commitment and investment to be in control of our GMP manufacturing activities. With that strategic goal in mind, we built an in-house dedicated GMP facility for our adenovirus drug substance manufacturing back in 2019. We then manufactured all PRGN-2012 drug substance lots utilized in our clinical trials at this facility. Now, we have upgraded the facility, and with a strong leadership and operations team, we are ready to support the commercial launch of PRGN-2012. In addition, we utilize an established commercial CDMO for fill/finish operations for the PRGN-2012 drug product. We have also built significant GMP quality control capabilities in-house to support release testing of PRGN-2012. Together, our GMP manufacturing and testing strategy is designed to give us better control over more specialized operations, overall timelines and provide independence from external vendors as much as practical while containing costs. As part of the PRGN-2012 BLA, we have completed the process validations as previously aligned with the FDA. We are confident in our ability to supply PRGN-2012 product to meet anticipated demand at launch and beyond. With that, I'd like to now hand over to Phil Tennant, our Chief Commercial Officer, to provide an update on PRGN-2012 market opportunity and our commercialization strategy. Phil?

Great. Thank you, Rutul. Good afternoon, everyone. The past few months have been incredibly dynamic for our commercialization team as we advanced preparations for the commercial launch around the August 27 PDUFA. As Helen said, this is a pivotal milestone for us as an organization and I'm excited to share the progress that we've made since our last update. We've made significant strides in our launch readiness, including several major accomplishments. Firstly, we've completed the build-out of our Precigen commercial leadership team. We now have a very strong and experienced team across sales, marketing, medical affairs, market access and distribution who have the experience and capabilities to guide the launch with precision and impact. Secondly, we've established a comprehensive commercialization strategy for the U.S. launch spanning all facets of our operations to ensure we hit the ground running at launch. In terms of our broader commercial infrastructure, we're delighted to announce our partnership with EVERSANA to implement the U.S. launch. As a proven leader in supporting rare disease launches, EVERSANA brings a wealth of experience that complements our vision for PRGN-2012. Together, we are executing on critical launch activities across the spectrum, including the training and deployment of dedicated field teams to ensure a seamless and impactful launch. Our updated analytics underscore the importance of this work. Our advanced analysis, as shared at J.P. Morgan, suggests that up to 27,000 adult patients in the U.S. are living with RRP, which is higher than previous estimates of up to 20,000 and indicative of an even greater unmet need. These data strengthen our resolve to launch PRGN-2012 with a patient-centric focus ensuring timely access to this transformative treatment. In summary, we're moving decisively towards launch driven by an experienced leadership team, a highly capable commercialization partner, and a robust strategy. These elements position us well to realize the full potential of PRGN-2012 with commercial revenues expected to begin in the second half of 2025. We are primed to hit the ground running given our PDUFA date and that would clearly position us as the first and only medical treatment available for these patients. Back to you, Helen.

Thank you, Phil. As you can see, the level of excitement at Precigen is high; PRGN-2012 has been and remains our highest priority as we transform the organization from R&D to a commercial company. With that in mind, I would also like to give you an update on two other programs that are very exciting and important. The first is PRGN-2009, our gorilla adenovirus drug product that targets HPV-16 and HPV-18, which are leading causes of HPV-related cancers. Approximately 5% of all cancers are HPV-related; that includes cervical cancer, head and neck, and anal cancers. PRGN-2009 targets HPV-16 and HPV-18 and we presented Phase 1 data at ASCO in 2023. We showed not only a very favorable safety and tolerability profile but, importantly, in relapsed-refractory patients who had failed checkpoint inhibitors, we were able to show for the first time in this setting a 30% objective response rate, including complete and partial responses. Our complete responders have had responses spanning close to two years. In some of these same patients, we have delivered in excess of 20 doses of PRGN-2009, which has the same backbone as PRGN-2012, and we have shown that neutralizing antibodies do not increase and there is clear enhancement of T-cell responses on redosing. This clinical and immunologic data points to the differentiation of our gorilla adenovirus platform versus other platforms. PRGN-2009 is advancing in Phase 2 for both cervical cancer and head and neck cancer at the NCI, and we will provide updates as we have them. At the same time, I'd like to update you on our UltraCAR-T platform. To our knowledge, UltraCAR-T is the only CAR-T platform that can deliver autologous CAR-T to patients overnight at the hospital without centralized manufacturing; it supports near-patient, decentralized manufacturing, and it is autologous. It contains the CAR of interest plus a safety switch, which is very important because if anything goes wrong, you can eliminate these cells immediately. At the same time, the membrane-bound IL-15 mechanism allows persistence and expansion of these cells in vivo without requiring in vitro expansion. Last year, we communicated that we had finished our Phase 1b in AML patients and that we were preparing for an end-of-Phase 1b meeting with the FDA. At J.P. Morgan, we presented additional exciting data that, in our view, is the first time any company has shown specific biomarkers that distinguish AML responders from non-responders following CAR-T treatment. We presented part of that data at J.P. Morgan and I encourage people to review that presentation if they have not. We are preparing regulatory discussions with the FDA to discuss the platform, our AML data, and strategy for a pivotal Phase 2 and a path for approval starting in AML. Please stay tuned; we will provide updates. Finally, our UltraCAR-T platform is generating exciting data in autoimmune settings. Autoimmune indications require a safe, repeatable, and scalable approach, and our CAR-T platform meets those requirements, including safety switches and next-generation adjustments that avoid the need for checkpoint inhibitors. With these data, we believe we have the potential to be first-in-class and best-in-class in autoimmunity. This work is ongoing and we are very excited. With that update, I would like to hand over to our CFO, Harry. Harry?

Thanks very much, Helen, and welcome to those participating in the call today. This really is an exciting time at Precigen as we prepare for the launch of our first commercial drug product. You've already heard from other members of the Precigen team as to our preparedness toward this goal, and I'd like to spend some time discussing our financial position as we move toward commercialization. I'll begin by highlighting our financial results for 2024. Overall, we finished 2024 with a net loss of $126.2 million or $0.47 per basic and diluted share compared to a net loss of $95.9 million or $0.39 per basic and diluted share in the year ended December 31, 2023. I'd like to point out that our current year net loss included over $55 million of non-cash charges, net. Our cash burn for 2024, consisting of cash used in operations plus capital expenditures, totaled $76.8 million. Our press release and management's discussion and analysis included in our 10-K, both of which were just filed with the SEC, provide further detail as to fluctuations in our statement of operations between 2024 and 2023. With that summary of our operating results, I'd like to spend a little time focused on our financial position. As many of you are aware, we raised $79 million at the end of 2024 via a preferred stock issuance, which included the issuance of warrants to purchase common stock. We believe that the terms of this preferred share instrument are friendly to the company. The preferred stock carries an 8% dividend, of which the first two years are to be paid in kind; beginning in the third year, while dividends will be payable in cash, the payment of such are due when and if declared by Precigen's Board of Directors. While the preferred shares are convertible into common stock, the conversion feature resets each quarter, thus reducing the dilutive effect of a potential conversion as our stock price increases. In addition, the company has the ability to redeem the preferred shares for the stated value plus accumulated and unpaid dividends, while the holders do not have the ability to put the shares to the company. You can find a more detailed summary of the terms of the preferred stock issuance in the financial statements in our Form 10-K or within our 8-K announcing the transaction that was filed with the SEC on December 30, 2024. In addition to funds from the preferred stock issuance, we monetized, in a non-dilutive manner, certain intellectual property rights and royalty rights related to non-core assets of the company in December 2024, which resulted in proceeds of $8.5 million. The preferred issuance plus the sale of intellectual property rights and royalty rights helped shore up our balance sheet and we finished the year with cash, cash equivalents, and investments of $97.9 million. We're confident that this balance will support us well beyond our anticipated launch date and well into 2026. This runway is based on current projections, which include anticipated revenue from the commercialization of PRGN-2012. As Helen mentioned earlier, our BLA was accepted by the FDA in February under a priority review with a PDUFA target action date set for August 27, 2025. This target action date plus the preparedness of our commercial team, as you heard from Phil earlier, provides me with a level of comfort to include revenue in my projections. Although from an accounting perspective, this anticipated revenue is considered outside of our direct control because it depends upon the successful FDA approval of the PRGN-2012 BLA. In closing, I want to reiterate that Precigen has always shown strong financial discipline and we will continue to do so in the future. Our focus has and always will be to utilize our resources to garner the highest value for our shareholders. With that, I'd like to open up the call for Q&A and turn it over to Alan, the operator.

Your first question comes from Jason Butler of Citizens. Your line is already open.

Hi, thanks for taking the questions, and congrats on the progress. Two for me. One, just at a high level, can you give us an update on the interactions with FDA and how you would characterize the ongoing review for PRGN-2012? And then on the commercial side, can you speak to your current thoughts on the size of the field force and the number of centers that you'll be targeting during the initial launch? Thank you.

Hi, Jason. Thank you for the questions. Regarding our BLA submission and receiving a priority review, we have had very close interactions and alignments with the FDA throughout. We are grateful to the FDA for the close interaction and guidance and we continue to engage with them throughout the review cycle. We believe we are in a very good position with our BLA submission and will remain closely aligned with the FDA as the review proceeds.

Yeah, sure. Regarding the size of the field force, we've been fairly consistent in our thinking that it will be somewhere in the range of 15 to 20 representatives in the field. So a fairly modest sales team. We believe there are around 500 fellowship-trained otolaryngologists who will be responsible for treating the bulk of the patients, and those physicians will largely be concentrated in urban academic centers and large IDNs. That will be an important part of our targeting.

Your next question comes from Ben Burnett of Stifel. Your line is already open.

Hello, good afternoon. This is Carolina Ibanez Ventoso on for Ben Burnett. Thank you for taking our questions and congratulations on all the progress. Do you have any line of sight at this point on the timing of any additional meetings with the FDA, pre-licensing inspections and labeling discussions during the review process? I have a follow-up as well.

Hi, Carolina, and thank you for the questions. As part of the regulatory process, there are inspections for facilities and because of that, we will not be making specific comments on timing, but inspections are typical, especially for GMP facilities, as I mentioned. We have always been in close alignment with the FDA and continue to be, and we look forward to further communication as we get closer to our PDUFA date.

Okay. Thank you. And Tom, if PRGN-2012 gets approved, do you anticipate there will be a bolus of patients? And how do you plan to position yourself to address it?

Yeah, great question. We absolutely do think there's pent-up demand here. These patients have had no on-label treatment options; it's primarily surgery and some off-label treatments that do not address the underlying infection. So we believe there will be a concentration of patients in desperate need of this medication when we get to market. We will deploy our team and efforts to make sure that as many of those patients as possible get treated as soon as possible.

Thank you, Carolina.

Your next question comes from Swayampakula Ramakanth of H.C. Wainwright. Your line is already open.

Thank you. This is RK from H.C. Wainwright. Good afternoon, Helen, Phil, Rutul, and Steven. So in terms of the indication itself, Phil, you are saying based on your looking at the claims the number seems to be higher and in the order of 27,000 in the United States and probably around 125,000 outside the U.S. But that is with no approved therapy and only patients who are brave enough to go under the knife. So, with an approved drug, how easy is it for patients to get diagnosed that they do have RRP? And second, how are you thinking of trying to reach patients who have not yet been diagnosed or is there a mechanism by which you can actually get these patients diagnosed correctly?

Great. Let me take the first part, RK. The patients are very visible because they are having surgeries; many of them have frequent surgeries annually. We believe many of those patients will be readily identifiable at launch, and that's a key area of focus. Like all good launches, we will have personal and non-personal promotion and remote promotion. Where we can't reach everyone face-to-face, we will provide information and direct people to appropriate resources for their patients. Another phenomenon we've seen in rare diseases is that when a new treatment becomes available, the number of diagnosed or presenting patients increases because of enhanced awareness and education. We don't think this will be different for RRP.

RK, this is Helen. I can add to what Phil mentioned. Adult patients living with RRP are highly engaged in seeking innovative treatments because for almost 50 years there has been no medical treatment other than repeated surgery. We have been conducting educational efforts with patient groups; last year we supported "RRP Day" for patient and disease awareness. These campaigns will continue through our PDUFA and beyond to ensure patients, investigators, laryngologists and KOLs are familiar with the aspects of this new standard of care upon FDA approval. We are committed to broad education and outreach.

Perfect. Thanks for all that color. Let me try another two-part question. First, what's the current status of the confirmatory trial for PRGN-2012? Second, do you think you will be close to completion by the PDUFA date? And a follow-up: on PRGN-3006, should we expect any Phase 1 data to be published either in the first half or later in the year?

Great questions. Regarding the confirmatory trial for PRGN-2012, as I mentioned earlier, we initiated the trial last year ahead of the BLA submission. The trial design is in full alignment with the FDA: a single-arm trial of 35 patients with no placebo control and the primary endpoint is complete responses measured over a minimum of 12 months. Because the primary endpoint requires at least 12 months of follow-up, we will not have completed confirmatory data close to the PDUFA date. We anticipate reporting on those data in 2026 and 2027. We have high enthusiasm from patients and investigators given the safety, efficacy, ease of administration (a subcutaneous injection similar to a flu shot), and the durability of response, which supports rapid enrollment and engagement. Regarding PRGN-3006 and the Phase 1 data, we are preparing our materials and regulatory discussions with the FDA and will provide updates on timing and venues for data presentation in the near future.

Your next question comes from Jennifer Kim of Cantor. Your line is already open.

Hi, thanks for taking my questions. Maybe to start off to follow-up on one of the previous questions. I just want to clarify, will the FDA's manufacturing facility inspection come ahead of the mid-cycle review? And I assume that's happening in late May, but do you think you'll have a sense by the mid-cycle review how the FDA is thinking?

Thank you, Jennifer. We do anticipate a pre-approval inspection (PAI) before approval, but as Helen mentioned earlier, the timing is part of the regulatory process and we are not able to comment specifically on timing at this point. We look forward to hosting the inspection and anticipate that it will occur prior to approval.

Okay. And maybe a question for Phil. The 500 doctors that you're initially targeting, what percent of the market does that capture? And could you give some color on how the internal sales force is coordinating with the EVERSANA partnership?

Right. The 500 ENTs I mentioned will be responsible for the vast majority of patients we believe are out there. There is a community presence as well, and we're not ignoring that, but the concentration is in urban centers and that will guide our field force deployment. All field teams are through EVERSANA; we are not sending Precigen employees into the field as such. The EVERSANA teams will act like Precigen employees through our partnership, and importantly they are dedicated to PRGN-2012.

Okay, that's helpful. If I could squeeze one more question in for Phil. In your discussions with payers, is the expectation that access to the treatment if approved would depend mostly on prior authorization to label versus medical exceptions, or have those discussions evolved?

We continue to speak to many payers and have now engaged payers representing over 300 million lives in the U.S. We're getting a consistent response about the value proposition. We expect a degree of utilization management such as prior authorizations. Our goal is PA to label, which is what we're trying to achieve, but some payers may include inclusion and exclusion criteria in their policies. We're building a strong economic and clinical value proposition to support access. Medical exception would be the last resort for some payers based on current discussions, and we are preparing accordingly.

I would add that this is a rare disease with no standard of care for many years and payers recognize the significant burden to patients from repeated surgeries, including irreversible damage to vocal cords and trachea. Payers appreciate the safety, efficacy, durability of response, ease of administration, and the potential to prevent irreversible harm. Those factors have been important in our payer discussions.

Your next question comes from Brian Cheng of J.P. Morgan. Your line is already open.

Thanks for taking our questions this afternoon. Maybe first, can you give us an update on the latest on preparing the sites to administer PRGN-2012? You talk about the 500 initial doctors. How do those 500 doctors overlap with the initial sites that are ready to administer PRGN-2012? I have a follow-up as well. Thank you.

Thanks, Brian. There is nothing overly complex about administering the drug at the site or any special preparation required other than standard cold chain considerations and subcutaneous injection. Academic institutions and large IDNs are well-equipped. We're working to understand the last '100 yards' of the drug journey so we can deliver a seamless distribution service from ordering to administration. There is nothing particularly complex in terms of preparation that needs to be called out.

Okay. And then maybe just one on your thoughts on pricing and payer access. What's your latest take on pricing for PRGN-2012? And should we expect value-based agreements here based on ongoing engagement with payers?

We continue to speak to payers and receive a fairly consistent response around the economic and clinical value proposition we're presenting. Value-based pricing is being considered; payers find the concept attractive in theory but sometimes difficult in practice, so we're exploring it. We believe the price point and the way we deliver value will be straightforward and within the wheelhouse of these institutions, not something unfamiliar to them.

Brian, I would add that payers recognize the disease burden, the potential to avoid irreversible damage, and the safety and efficacy profile we have shown, including 51% complete responders and durable responses. These factors are a significant part of our value discussions.

There are no further questions at this time. I would hand over the call to Dr. Helen Sabzevari for closing remarks. Please go ahead.

Thank you. As we move one step closer to commercialization, I want to take this opportunity to thank our team at Precigen for their tireless work and steadfast dedication to bringing PRGN-2012 to market. Our team understands that patients with RRP are burdened significantly by this disease and that repeated surgery carries innumerable risks and potential long-term irreversible injury. We are acutely focused on delivering the first and only FDA-approved therapy to the RRP community as quickly as possible. With that, I wish you all a very good evening and thank you for participating in our call.

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