Precigen, Inc.

Good evening, and welcome to the President'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 Harrison, from Investor Relations. Please go ahead.

Thank you, Alan, and thank you for everyone joining us this afternoon. With me today are Dr. Helen Sabzavary, President and CEO of Precision, Harry Tomasian, our CFO, Phil Tennant, our Chief Commercial Officer, and Ritul 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-hour statement contained in our most recent SEC filings, as well as risk factors contained in precedent filings. With that, I would like to now turn the call over to Dr. Sabzabari. 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 the Precision, and as we are on the verge of commercializing our lead asset, PRGM 2012 in RRP, potentially bringing a treatment to this patient population with high unmet needs. From discovery in 2020, phase one initiation in 2021, phase two with breakthrough designation and accelerated approval pathway in 2023, followed by publication in science translation and presentation of the groundbreaking data at ASCO in 2024. This program has advanced with a remarkable efficiency and agility. We finished 2024 with submissions 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 some time, a few minutes, to recap of our data which actually recently has come out in Lancet publication. We are extremely excited about PRG in 2012, potential in RRP to not only be the first, but the best in the class treatment due to significant effect in terms of efficacy, safety, ease of route of administrations. 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 most importantly, a prospectively defined statistical primary efficacy endpoint of complete responses in RRP patients. Our pivotal study met primary efficacy endpoint with 51 percent complete response rate. That was a statistically significant and handling beat pre-specified success criteria established in alignment with FDA. Complete responses have been durable with median durability of response at 24 months. And all of our phase one complete responders remain surgery-free and in complete response three years. This is highly significant, and I'm going to stress that the data that was presented at ASCO showing collectively phase one and phase two, it has such a close repeat that In our phase one, we had 50% complete responders and phase two, 52% for the average of 51% complete response in a prospective manner. Furthermore, 86% of all of our patients had reduction in the number of surgeries. In multiple publications, we have also shown significant enhancement of HPV6 and or 11 T cell responses, which is directly corresponds to the mechanism of action of PRGN 2012 and our clinical responses. And the first set of data was published actually in 2023 in Science Translation in regard to this. We have shown that PRGN 2012, it's again because of the differentiation that the gorilla adenovirus has shown. It can be repeatedly dosed, not only in our current program of PRGN 2012, which there has been four doses, but also in our other programs such as PRGN 2009, which has been dosed more than 20 times. This platform is different than other AAVs or other viral platforms that they have a limited amount of time that you can dose them, for instance, once and twice. Gorilla adenovirus, it can be repeat dosed and continues to lead to the enhancement of the T-cells in the absence of neutralizing antibodies, significant neutralizing antibodies. And finally, I would like to say that the PRGN 2012, based on all the data that has been shown and the follow-ups, is extremely well-tolerated with no dose-limiting toxicity, no treatment-related adverse events greater than grade 2. As we have already shown, the route of the administration is just a simple sub-Q administration that can be given at any office. Well, we also have continued with our readiness to hit the ground running post of PDUFA Day in August of 2025. As we shift from R&D to commercialization, And Phil, our chief of commercial, would be speaking to that. Finally, I would say a few words about our confirmatory trial, which already has been initiated and has started enrolling patients. Our confirmatory trial, in alignment with the FDA, is a single arm, no placebo control required, with 35 patients to be enrolled. And we are well on our way. Not only we have started an enrolling patient, but to move very rapidly in finishing this trial. So with that, I will now turn the call to Ritul Shah, our Chief Operation Officer, which will be speaking to our CMC and manufacturing readiness.