Precigen, Inc.

and welcome to the President's second quarter and first half 2024 financial results call. At this time, all lines are in a listen-only mode. Following the presentation, there will be a question and answer session. Instructions will be provided at that time for you to get up for a question. If anyone has any difficulties hearing the conference, please press star zero for operator assistance at any time. Please note, this event is being recorded. I would now like to turn the conference over to Steve Harrison, Vice President of Investor Relations. Please go ahead.

Thank you, Jenny, and thank you for everyone joining us this afternoon. With me today are Dr. Helen Sabzavari, President and CEO of Prestigen, Harry Tomasi, our CFO, Bill Tennant, our Chief Commercial Officer, and our Chief Operating Officer, Ritul Shah. 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 materially from those indicated by forward-looking statements. Please read the safe harbor statement contained in our most recent SEC filings, as well as risk factors contained in Presagen's filing. With that, I would like to now turn the call over to Dr. Sabzabari. Helen?

Thank you, Steve, and thank you to all joining us today. It is a pivotal time at Precision as we work hard to get what could be the first FDA-approved treatment for the devastating recurrent respiratory papillomatosis disease, or as we know it, RRP, to the market. What I would like to do at the beginning is take you through some of the key program and business highlights, and then go through some of the strategic prioritization that has been communicated recently. Let me start by talking about PRGN 2012, which is the focus now of our portfolio. As you know, PRGN 2012 is our ad universe, gene therapy that is designed to elicit immune responses directed to HPV6 and 11 infected tumors and to raise the immune system to both of these epithelium, both HPV6 and 11, for the treatment of recurrent respiratory papillomatosis. Let's establish some of the facts up front in regard to our PRGN 2012. As I mentioned, it's delivered by our gorilla adenovectors, and it's a gene therapy. It's delivered subcutaneously. It does not require any kind of a device. So it's an ease of administration that can be done at any office setting, for instance. The second important part of this gene therapy treatment is that our gorilla adenovirus platform in general and the viral vector that is used for delivery of PRGN 2012 is differentiated from other viral vectors. Even though there is this perception that all viral vectors are similar and therefore they can be given a very limited number of times, perhaps only once or twice, and they cannot be used in redosing because humans have preimmunity to them. This is not applying to the gorilla adenoviruses. And why do I say that? Simply, humans either have zero preimmunity or very little preimmunity to these vectors. And by the way, these vectors are designed in such a way that elicit a high affinity C cell and they push toward the CD8 responses. And therefore, in a number of clinical trials that we have done across various indications, We have shown over a period of time you can keep redosing with these vectors and keep enhancing the immune responses and especially high affinity CD8 cell responses. So, I just wanted to clarify that fact. This is one of the differentiating factors of our Gorilla Adenovirus platform in general compared to other AAV and viral delivery factors. And also, these guerrilla adenoviruses allows us to have a high payload that we can put many epitopes and many genes. And therefore, when I say that we are targeting HPV 6 and 11 and raise immune responses to both, we have shown that and we have shown that in the clinical trial. Now, with that in mind, What have we done with the PRGN 2012? As you are well aware, in the PRGN 2012 is the first drug ever received a breakthrough designation and an accelerated path from FDA in June of 2023. And with that designation, we also had received the orphan drug designation from FDA as well as EMA. The interesting part of this was that because we had designed our clinical trial in such a way that, first of all, addressed the most advanced patient and most patients with the highest number of surgeries, and also already put a very robust endpoint, not just reduction in the number of surgeries, but actually having safety and efficacy that leads to a complete response to the severe patient population that at least maintain for a whole year, that they do not require any surgery and beyond. As a result of that, the FDA agreed that our phase one single-arm trial as well as our phase two single-arm trial can serve as a pivotal trial And we did not have, and we have not been required to do any kind of randomization or placebo control trial in that setting. And that is very important. And one other aspect that I want to stress here, it is, and FDA agrees, that these patient populations, they are their own best control. And therefore, by having such a high standard of not only in the number of being in a severe patient population, but also a robust endpoint that negated the need for having a randomization or placebo control. So let's just take a look at what happened as a result of our Phase I and Phase II. We had enrolled 12 patients in our Phase I and 23 patients in our Phase II single-arms And we recently in June actually presented the full set of data, both from phase one as well as phase two, the breakthrough session at ASCO. And it was received extremely well by investigators as well as our patients and patients in RRP for a number of reasons. First of all, if you look at the primary safety of this, it's extremely favorable, and it's also the efficacy when you look at the endpoints for this trial, which was a complete response, meaning that the patient to go from number of surgery, which the average was four and a half surgery per prior year before the treatment, completely zero surgery requirement. 12 months follow-up, and we have been, by the way, following these patients for a durability of response, which we have seen quite a durable responses in this patient population. 18 out of 35 of these patients went to a complete response. That's 51% of the population. If you look at Our secondary endpoint, which was the reduction in the number of surgeries, now we have 30 out of 35 patients. They reduced their number of surgeries. That's 86% of our population that was treated. This has been an unprecedented result by any treatment for this patient population. There has never been a data that was presented with this robustness of the endpoint, as well as the safety, and also being in the most severe patient population that clearly it really is the much tougher treatment to be treated, not only in this disease, in any disease in general, as we all agree. So I think this was quite exciting. And as I mentioned, in 2023, in June 2023, we received the accelerated approval path and the rolling BLA. And our submission, we are on track to submit our BLA by the end of this year, in 2024. We also, what I'm very excited about is that we have initiated our enrollment in the confirmatory trial already. And this, we had reached an already agreement with the FDA that, by the way, our confirmatory trials are also single-arm, no placebo requirement, no randomization, which, as you can imagine, it's going to make things extremely challenging for these patients if you have to randomize because Each patient is their own best control, and the randomization is quite challenging, and this is what all the investigators believe as well. We already not only have initiated the confirmatory trial, which is part of submission of the BLA, but we have started the enrollment, and I have to say that we are quite taken by the number of the patients. that they already have shown excitement to participate in our confirmatory trial. On the side of our commercial scale for drug substance manufacturing, also our facility is operational, and we are quite confident that we can adequately provide the doses for both U.S. and ex-U.S. on a potential launch. And as part of our patient advocacy effort this year, we had the joint recurrent respiratory papillomatosis inaugural RRP awareness day on June 11, because this disease is actually as a result of the infection by HPV 6 and 11, therefore June 11. And we are really excited that we have been working very, very closely with RRP Foundation to advance this innovative treatment for the patients in this setting. So, with that in mind, just to recap what we have done and why we are so excited and so focused on PRGN 2012 is we have established a well-differentiated innovative therapy That doesn't require any device. You can give it subcutaneously. It elicits immune responses to the root cause of these benign tumors which take place on the trachea or the vocal cords of this patient and cause a devastating scenario for this patient. And as a result of the mechanism of the action and the safety, advanced safety and safety that we have shown, these patients, we get 51% of these patients going to a complete response, 86% of them reducing their number of surgeries. The durability of the response, the median has not been reached at this point in 20 months. Our phase one patients that they have been obviously have a much longer follow-up have surpassed 32 months of the durable responses and have not required any surgery. So with that, we believe that this, and we are extremely excited that this drug has the potential to be quite effective for RRP patients. And the first drug has the potential to receive the first drug ever approved by the FDA. So in that process, we also, have been preparing for the potential commercialization of the drug in 2025. And in that setting, I am thrilled to welcome Phil Tennant as the Chief Commercial Officer for our company. And we have been very excited that Phil has joined the leadership and now currently leading all activities around the commercialization for PRGN 2012. And I would like to hand over actually to Phil to hear from him and his perspective on the commercial path of PRGN 2012. Phil?