Great. First, welcome to the 47th Annual Goldman Sachs Healthcare Conference. Very pleased to be joined by Helen and Phil from Precigen. First, welcome. Maybe to start things, for those who might be a little bit less familiar with Precigen and overall story as it stands today, maybe Helen, if you could give a brief overview of the company. Sure. First of all, thank you for having us here. Precigen is a cell and gene therapy company. It works on a really innovative, differentiated platform. With combining it really for fit our technology to the right indication with the right regulatory pathway. This is very important from the very beginning because it allows the focus and allows us to move very rapidly, which a good example of this, we have a portfolio of two technology platform. One is our AdenoVerse platform, which is a very unique and differentiated, basically, delivery platform that allows that you can repeat those patients, with very specific adenovirus vectors that are not similar to what you have seen in the field. The reason for that, it has a large capacity, and it allows for continuous repeat dosing and without very little or no neutralizing antibodies. The good part of that is that you can repeat those as many times, which we have shown in our clinical trial. The frontrunner of that, is using this platform, is our papillomavirus drug. That has been now approved in the rare disease space for recurrent respiratory papillomatosis, RRP, which for the past 100 years have not had any kind of approved treatment. These patients basically, as a result of HPV infection with either HPV 6 or HPV 11, they develop these tumors, benign tumors in the throat or on the vocal cord. As a result of that, they continuously, the only thing that physicians could offer them up to the last year was continuous surgery. Some of our patients have had over 300 surgery. Some of them have to receive a surgery every practically month. This disease can be basically established either as a child, by a child passing through the birth canal and getting infected from the mother, or in adult transmission. It's a devastating disease. For the first in a very exciting data that we are able now to treat. 51% of these patients go to a complete response, not requiring any basically surgery or any other treatment, which is unheard of in this field. On the back of the same platform, we have our PRGN-2009, which we are very excited, and again, it applies to the cancers, HPV-related cancers such as head and neck and cervical cancer, which we will be actually looking forward to sharing a data in early Q4 of this year on head and neck cancer. We also have our cellular therapy, which is our UltraCAR-T cells, which is, this is truly an overnight with the manufacturing directly in patient. With that, we have finished our end of, basically phase Ib, and we are moving now to setting up the pivotal for phase II. As a result of having this platform, it allows Precigen to come up with very innovative treatments for the patient, which not only has a very good safety profile, but also have a very unique efficacy and a durability of response that we will be touching base on that, for instance, in regards to papillomavirus, which is now well over into the 40-month post-treatment. Great. You mentioned upcoming data, maybe near and medium-term catalysts that investors should keep in mind in the coming quarters. Absolutely. First of all, our PAPZIMEOS, as we mentioned, we received under Accelerated Approval pathway from FDA last year in August, in full approval, actually, it became a full approval. Currently, as Phil will be speaking, we are very excited about the commercial revenues and the path that is coming in. At the same token, we have started the redosing the trials for this, which becomes very important. As we mentioned, not only we had a 51% complete response in this patient population that they never had any treatment before, but we have an 86% overall response. Based on that, actually, FDA was very interested, and safety that we showed, that those partial responders, that should also be re-dosed, and they perhaps will benefit from this. We have started those studies of redosing. We also start the studies in a pediatric population. Again, because of the safety and efficacy and durability of response that we see with PAPZIMEOS. This was highly encouraged by the FDA, we are extremely excited because this is a unique platform that will lend itself to the pediatric trial. At the same token, we have already submitted for the approval for the European in EMA. We will be waiting in the upcoming quarters to communicate in regard to that. Our application is under review from the PAPZIMEOS side. Also, one of the other aspects is that currently our PRGN-2009, which I mentioned, is the drug that is on the same backbone of the viral vector with PAPZIMEOS. It is in the clinic on phase II for head and neck and cervical cancer, we are very excited about reporting on a trial in fall, probably by early Q4, the result of the head and neck. As you know, this is actually a two-arm trial in patients that have head and neck. The response rate of the patients in these settings are between 15%-18%, even with all the checkpoint inhibitor. In phase I, what we show was the objective responses of 30%. Currently, obviously, this is an open trial that we have a view to the data, and we are very excited to be sharing that in Q4 of this year, and I think this will be very relevant for the investors. Great. You recently got orphan drug exclusivity. Can you talk about the importance of that to the company and overall? Absolutely. We had originally received the orphan drug designation when we submitted our application. As part of that, if you receive approval, then you become qualified for the orphan drug market exclusivity. It doesn't mean that every company will receive that. It's just that they will be qualified, especially in rare diseases. This is a special program that FDA had basically put forward for the innovative treatments. What they have done is to encourage the companies to work on rare diseases. With receiving the orphan drug market exclusivity, we have seven years of market exclusivity that basically similar type of drugs are not going to be approved by FDA. You can imagine, and Phil will speak to this more, that this now allows PAPZIMEOS and Precigen have the full seven years of basically having the marketing of the PAPZIMEOS and the drug for RRP, and it's very important because similar drugs cannot be receiving approval from FDA, and also, they have to meet the same efficacy as well as durability, as well as basically the safety. It puts the bar very high for seven years and allows us to move very rapidly. That's quite exciting for us. That's great. Maybe turning to the commercial side and maybe Phil. Obviously, early innings still, and I'm not going to try to pin you down on Q2. Yeah How's the launch going so far, and any update you can give investors there? Sure. Well, we've covered a lot of the dynamics that are leading to our excitement about the launch. You're right, we won't go into Q2 in any great detail, but we look forward to sharing the Q2 results in August. A lot of the building blocks of launch have exceeded expectations. We had a rapid increase in payer coverage to well over 215 million lives within six, eight months of launch. When you include Medicare, Medicaid, and the others, we've got over 90% of insured lives covered. As you can imagine, for any rare disease launch, without that, your launch is going to be in trouble. To get the payer coverage has been phenomenal. Our pre-launch work told us that there was a concentration of patients in the academic centers, so we estimated there were 27,000 adults in the U.S. with this condition. Most of those, because of the cyclical nature of surgery, they were concentrated in the academic centers, the IDNs. That is indeed the case, but one of the things that we're also seeing through launch is the community interest in the drug and the community uptake. That makes sense for a number of reasons. It's closer to home for the patients. We've put in place simple low-cost solutions because our drug is ultra-cold chain, but freeze and release and just-in-time shipping mean that basically we can ship the drug, and a patient can get treated anywhere. That community uptake has been very important. We had a paper published under the auspices of the foundation in January, where 16 thought leaders basically put their names to the fact that PAPZIMEOS is the standard of care for all adult patients with RRP, irrespective of surgical burden. Then in April, we got the permanent J-code, which is an important piece of the jigsaw puzzle as you're launching a drug. That just takes a lot of the drama out of the whole patient journey to access. We were aware of certain institutions that were waiting for the permanent J-code, and now we've got the permanent J-code. As in other rare disease launches that we've looked at, we expect that to help with our acceleration and our- Maybe I can just add to what Phil mentioned. In regard to our label from FDA, we have a very broad label that covers all adult patients with RRP. That has become very important because now we see that patients across the spectrum are being treated. One of the reason for the community centers is because they are the first line, and when patient actually start the journey with this disease, they go first to their community doctors. We are seeing the uptake not only just in severe patient population, but as early as just being diagnosed. The patients now have started receiving this, which is excellent for the patient because this is a kind of a disease that by fifth surgery, these patients have irreversible damage. The sooner that you get to them, the better. Great. In terms of real-world usage, what are you seeing from a compliance standpoint in the initial innings? Yeah. Pretty much what we saw in our registrational studies, where all 35 patients took all four doses of the treatment. We are seeing very good observance of that regimen in clinical practice, which reflects the safety and the promise of durability that we've seen in the data and we continue to see in the extended follow-up. Yeah, we're very pleased. We're not seeing anything untoward in the real-world use of the drug. Great. Phil, you mentioned a little bit ago academic versus community centers, especially on the other side of J-code. Maybe a little bit more detail in terms of how you're seeing usage patterns and how that may inform things going forward. We've talked a lot about hub, our manufacturer's hub. This is a site where basically patients can register, and physicians can register patients with the intent of getting them onto drug. That's one source of the top of the funnel, as it were. Some institutions are very well-versed in their own patient support services, and they don't use our hub, so it's only part of the story. 25% of the patients registered in our hub are from the community, which gives you an idea of what I talked about earlier in terms of the community uptake of PAPZIMEOS. Both sides of that coin, the academic and the community centers, are embracing the drug and basically treating patients. To Helen's point, early on in launch, you would expect at the academic institutions, the severe patients, those who have multiple surgeries, those who have highest unmet need to be treated. Now with community and even at the IDNs, we're seeing that now expand into less severe patients, which is important for the long-term routine growth of our label. Great. Helen, you mentioned earlier your recent ASCO data. Can you give a little bit more detail in terms of the updated durability data you gave, and especially how that's going to inform the profile of the product? No, absolutely. I think this is one of the most important indicator for this drug, is not only the safety and efficacy that we saw in the pivotal trial, which was looking at a very robust endpoint of not having any surgery or requiring any treatment for the first year. Now we have gone well over that. The median of the patients have passed 36 months, and at ASCO, we showed there are a number of patients that they have passed 40 months, and these were the most severe patient population. Imagining a patient that they require the surgery, practically some of them every month, and to go now more than 40 months, not requiring, not only any surgery, no treatment at all. This is quite, with what some of our investigators and the physicians say out there, is really outstanding. They have never seen anything like this, with the safety that it has. As I mentioned, the total response rate is 86% in this patient population, and this has put a standard now for the treatment that is really outstanding as the standard of care for this. Rob, I mentioned that we're in a great position with payers, but as you can imagine, this increasing durability- Yeah ...just adds to that confidence that a lot of patients are going to be treated with this drug and have a great durable response, payers like that sort of thing. Certainly helps with the health economics, that is for sure. Back to kind of the practicalities of commercial launch. Can you walk us through the steps involved from getting scripts to treatment, maybe along similar lines, any characteristics of treated patients in terms of- We operate in a complex talk about site activation internally, there are a number of things that have to happen. Basically, when a doctor registers the patient, that's an intention to prescribe. That's a prescription. They may or may not be within our hub, as I mentioned earlier. That triggers, if they are in our manufacturer's hub, a benefits verification process to make sure the patient has the insurance and coverage and everything else. Once that is done, it's really up to the institution then to help get the prior authorization through dialogue with the payer. Then the patient is ready. When those three things come together, you get the treatment, that should take a matter of weeks, that entire process, once those things are in place. We have put dedicated resources in field to help the institutions get through that prior authorization step, because a lot of these ENT sites, they've never had to do this before. We have the expertise now, we've dedicated that resource to make sure that those patients are accelerating through to treatment. In terms of the characteristics of the patients, we talked earlier, initially, as you would expect, the more severe patients, we have a broad label, it's important now to stress that we are seeing the use beyond those severities that needs to continue, our marketing programs are designed to make sure that every single patient with this condition has a conversation about PAPZIMEOS with their treating physician. That has to be our goal. Makes sense. On the label, I guess, how are you seeing things regarding the label and potential for an opportunity for redosing? From the label, as we mentioned, when we received the full approval from FDA, we received a broad label, which meant that regardless of severity of the disease, the patients are qualified to receive this treatment. Based on the original discussions with FDA, it was very clear, FDA, based on the safety and efficacy that we were seeing and the immunological data that we showed, that the patients that also had reduced their number of surgeries but had not gone to zero, they perhaps can benefit from redosing. As part of that, we have already started our redosing, and actually patients are enrolling in that program currently, we very rapidly move with that concept. At the same token, our label is not really restricted already in this redosing, so it's at the discretion of the physicians. Obviously, we are generating also further data on a redosing. This will be very important because it adds the 36% or 35% of the patients that they have been partial responders. They have benefit, and we showed that data as well, that they've reduced their number of surgery. As the patient, they say, even reduction with one surgery for them is very relevant. We would be moving very rapidly with that. That's great. Especially early in a launch, things can be lumpy and not always the easiest to track. I guess, from an investor standpoint, what's the best way to think about tracking the launch on the forward? Well, revenue is quite a nice indicator of how we're doing. Important KPI. Yeah, all roads lead to revenue, right? We shared our Q1 revenue, which I think got favorable responses, which is an indicator of all the work that we've done, the platform that we've set in Q4 into Q1. As I said, we're very excited about sharing. We've still got a best part of a month to go of the second quarter, but we're already looking forward to sharing that information. I'd say revenue is a pretty big clue. We'll continue to share information on patient hub numbers, because that represents your intention to prescribe, both in our hub and from elsewhere. We talked about payer numbers, which are very healthy. There's not going to be too much more to say on that because we're in such a great position, but we'll continue to reinforce that. We'll continue to talk about the community and the IDN sort of split. I think that the community side of the business, we would expect that to be a more prominent part as we go forward of our overall business opportunity, we'll continue to share that. Makes sense. We've talked about the patient hub a few times. Can you give us a sense of the conversion you're seeing and also a sense of how you think that might evolve? Yeah. Well, first and foremost, our ambition is to make sure that all of those patients in our manufacturer's hub get onto treatment. We have the resources in place to help with that conversion. The revenue that we shared in Q1 obviously gives a big clue as to how we're doing in terms of that conversion. Now we have the permanent J-code as of April. I think that will help provide a little bit more momentum. We expect that conversion to continue to even accelerate and to be represented in the revenues that we'll share in Q2. Yeah. Maybe I can just add to what Phil mentioned. In regard to our hub, as we have seen from last year to this year, continuous increase in the number of hundreds in the hub, just our hub. This, as Phil mentioned, is not really talking about the patients that are in the other hubs of the institute. Also, I think one of the things that I'm going to address more is really how fast this treatment has been picked up by the payers. Both Phil and I, we have quite a long experience in the drug development, and this is one of the fastest pace that we have seen, especially for rare diseases, which are not necessarily very low pay for the payers and insurers. To be able to approve it and approve it so rapidly and get to more than 90% of the payers agreeing with the label and also agreeing with the payment. This has helped tremendously, it would get reflected, as has been reflected in the revenue. Great. Switching gears to the Europe side. You filed late last year. Can you give an update on regulatory for Europe? We have already submitted the application. Our application is under review by EMA. We are looking forward, obviously, to the results from there, and this is a similar application that we had submitted to the FDA. I think we have also communicated, we are looking in the path for the commercialization, which a major part of that is with the partnerships in Europe, and to perhaps as soon as we hopefully get approval, then within the right strategy, we also will start the treatments in Europe. One of the things that our team has done over the years, again, looking in the numbers of the patients in Europe, and very interestingly, Europe and Japan probably have similar or more of the patients than United States. It's quite a large market there as well. Of course, China is even a much bigger market, which we will be paying our attention. Great. Makes sense. We talked a little bit about the expansion opportunities for redosing. Can you talk a little bit about pediatric opportunities? Absolutely. I think pediatric opportunity, as I mentioned, some of the patients, actually a lot of adult patients, they are infected as a child and as early as age of one, we have had a patient at one year old was infected. Can you imagine basically taking a one-year-old to OR every month or every other month? There is a continuous population, somewhere estimated by FDA, that between 1,000 to 1,500 cases per year. Unfortunately, I think, without taking the preventive vaccine, that this might become even larger population. For children, the safety of the drug, it becomes, and the side effects, is extremely important, as you can imagine, because the threshold of the tolerance of adult is different than a child, and it becomes very important. FDA, based on the results that we have shown, they have been extremely excited as we take this to the pediatric population end of this year in Q4. We will be opening our trial for the pediatric, and we look forward to having a similar, at least, or even hopefully, more in pediatric because we are now intervening at a much earlier stage even of the disease. We look forward to that, and I think PAPZIMEOS is the only drug that has the ability to go very rapidly in the pediatric. Great. We talked a little bit about it earlier, you have a same platform in the clinic for HPV-associated head and neck as well as cervical. Can you talk in a little bit more detail in terms of when we would expect a readout and expectations going into it in terms of what you might see? Absolutely. PRGN-2009 is our second molecule that is exactly on a backbone of the vector that PAPZIMEOS is built on. First of all, as you can imagine, it's very important under also the new guidances from FDA, we are going towards applying for the Platform Technology Designation. This Platform Technology Designation allows now all of the next drugs that comes with the same platform, with the same vector, and they will have the same safety and manufacturing, which from a regulatory perspective, it makes it much easier and faster to move. PRGN-2009 of cervical cancer and head and neck. These are in patients that they have failed practically on the cervical cancer, everything. On the head and neck, we have gone to various stages of head and neck, not only stage 4 in our phase I, but also now in phase II, we have gone to even earlier stages. Just to give you a perspective, checkpoint inhibitor in these settings, they have had between 15%-20%. In a head and neck, when they combined the checkpoint inhibitors with the standard of care in the earlier stages of head and neck cancer, there was no benefit whatsoever. What we have done now is in conjunction with pembrolizumab, we have gone in to both cervical cancer and head and neck, we showed that actually quite a substantial increase to 30% objective responses, complete responder, partial responder, that they went well over, for instance, complete responders over two years, and some of them continued. Now in head and neck, we will be reporting on the data in early Q1, which is going to be very exciting in the different stages in conjunction with pembro. In that space, really the standard of care has been really harsh. You have surgeries followed by extensive chemo and radiation, which really the patients become devastated as a result of that. We are looking forward to show that data. I think, in my opinion, it will change the paradigm. That's great. Such high unmet need. Yeah. Absolutely. In both patient populations. Maybe in closing, in our last couple minutes, what do you think is the most underappreciated aspect of Precigen that you want investors to take away? Absolutely. I think I have heard from a lot of investors that they have said to me, said, "Helen, we missed this," "We missed Precigen. It was under the radar." First of all, to say that still there is a huge potential for Precigen as it's going up, this is just the tip of an iceberg for us. We are looking forward to this portfolio and the next molecules. As we have shown, Precigen, really, we started the company in 2020. If you can imagine in the middle of a pandemic then the global markets crashing. What we have done, our PAPZIMEOS drug entered to a phase I in 2021. By end of 2021, we had finished the phase I, started the phase II in 2022. We receive a full approval in 2025. This is unprecedented for drug development, even by a biggest pharma, let alone by a small biotech. We have been very focused from basically financially and strategically. We position our assets with our technology regulatory path, not just basically hoping and wishing for a miracle. This is not our style. We are very focused on that. I think people, they really didn't understand differentiate the technology at the beginning. Also, I think the discipline that we have shown over the years on our portfolio advancement having a management team that is well-versed, not only in discovery and development, but also in commercial. At various stages, there has been always question, especially going in a rare disease, that there was, well, what is the market value of this? Basically, how fast can you get approval? Would the payers pay for this? All those questions were all along, I think that has led to underappreciation. I think one by one, we have taken this obstacle, we have answered them, we have moved the assets forward the portfolio. I think, hopefully, the investors see the evolution of our company. Also, as I mentioned, this is just the tip of the iceberg for us. We are looking forward in the upcoming quarters, not only from our PAPZIMEOS commercialization, but also the next generation of the molecules in the oncology indication. Wonderful. Well, that is a great place to close. Thank you, Helen and Phil, for joining me today. Thank you. Thank Thank you very much for having me