Good morning, everyone. Thanks for joining us for another session at the 44th JP Morgan Healthcare Conference. I'm Brian Cheng, and I'm one of the senior biotech analysts at the firm. On stage, we have Precigen. I'll now pass the mic to them for a short presentation followed by a live audience Q&A. Helen, the stage is yours.
Thank you very much, Brian, and thank you for the invitation to the JP Morgan meetings, and I would say good morning, or after noon, to some of the audience that are on a webcast. Today, what I would like to do is take you through the advancements of our portfolio at Precigen. Obviously, I would be making some forward-looking statements. Please read that, and first of all, I just want to introduce, really, for those of you who might not be familiar with Precigen and Precigen story, who we are. We are a biotech company that has focused on cell and gene therapy for various indications, including rare diseases and in oncology. We are located in Maryland.
Our approach has been, from the very beginning, a start of a company is to really have differentiated technology and platforms that can be applied to a specific indication with a very specific regulatory strategy. As I take you through the, basically, presentation, you will see how we have done that with our first drug that has been approved last year, Pepcimeus, in a very, very rapid manner to go from a really discovery development to approval in four years. With that in mind, today, I'm going to have the focus on our main platform, which is an adenovirus platform. This is a viral vector platform, but it's unlike any other adenoviruses. These are actually gorilla adenoviral vectors that are non-replicating. But the major difference, as you see on the right side of the slide, is, first of all, their large payload capacity.
So for instance, if a regular adenovirus or other viruses, they have somewhere between three to four or five kb. You're looking at somewhere between 12- 15 kb of capacity, which means you can put more epitopes, much more genes. And this is really unique and differentiated from that perspective. Also, what is very different in our platform for adenovirus platform is the ability to repeat dose. And in a regular adenoviral vector, as you all have seen, you can give one dose, and perhaps by the second time that you go in, you have zero seropositivity because all of the people, healthy or some of the patients, they already are seropositive. They have neutralizing antibodies to these viruses because they have seen it before. The immune system has seen it, has generated neutralizing antibody.
By the second, third time that you start injecting these viral vectors, basically, you are getting very high titers of neutralizing antibody, which inhibits their capability of activating the immune system. As a result, it's like really shooting, basically, empty bullets. That's what happens. Not in the case of adenovirus platform. Here, because we have done extensive studies, both in healthy volunteers in the U.S. and also in Africa, the majority of the people are either completely seronegative, and the few percentage that they have a seropositivity is very, very low. The way we have designed these vectors, which is very interesting, it pushes toward a T cell immunity and not humoral B cell.
Even upon injections of these multiple times, and by that, I don't mean two or three or four times, we have had clinical studies in our cervical cancer and head and neck studies that we have patients have received over 16 injections over a period of two years. You do not see those high titers, and you keep seeing the expansion of a specific and high affinity T cell. This is very, very important for this. We have the full IP around this platform, and we are the only company that has established this and has the IP.
Obviously, as I spoke, it pushes more toward T cell immunity than a B cell immunity, at least the designs of the vectors that are currently I'm speaking about, and a very high productive manufacturing, basically, process, which has been really one of the bases for us having our own manufacturing in-house and has allowed us to move very rapidly. On the left-hand side, you see the portfolio that is associated with our adenovirus platform. Of course, at the top is our Pepcimeus, is a drug for a rare disease, as I will take you through the journey of it today, and which we received the full approval from FDA last year, and followed by exactly the same platform and the same vectors, but now addressing HPV 16 and 18, which is the cause of head and neck in the indications for cervical cancers, head and neck, anal cancers.
It's the reason that these tumors are developed. And we are in two phase II at this point, and actually quite interesting data. Some of it we have reported, and some we will be reporting in the upcoming quarters and year. And it's very, very interesting. Obviously, I will talk about the platform further. So as has been our tradition, we always go to what was our goals and what did we accomplish in a prior year to coming to JP Morgan. And as you see on the top of the list is the full approval for Pepcimeus for the treatment of all adults with recurrent respiratory papillomatosis. And I will, again, take you through what this disease is for those of you who might not be familiar with it. We also have commenced our commercial U.S. launch of Pepcimeus, and we have launched the manufacturing.
Currently, our patients across the United States are being dosed with Pepcimeus, and this is quite exciting. Finally, we have secured up to $125 million in non-dilutive funding to fortify our balance sheet. Quite an exciting year for us, definitely a paradigm shifting for a company, as we have moved now from an R&D company to a commercial company with a first FDA-approved drug in the history of this drug, which is over 100 years for RRP. What is RRP? Very shortly, explain recurrent respiratory papillomatosis. It's caused by the infection of HPV 6 and 11, and this can happen in children or adults. That's the root cause of formation of these benign tumors, which takes place on either the vocal cords or in trachea.
It's quite a devastating disease because the patients, they can't speak, or the picture that you see in the center, this is through the trachea they're looking at, or they can't breathe. The only treatment that was available, and this is not because other treatments have not been tried in this indication, but the only thing that works, it's continuous surgery until before the approval of Pepcimeus. You can imagine how devastating this is if you have it from childhood or in cases of adults. Just to give you a little bit of perspective, because at the beginning, I said, what is the vision of our Precigen and how we are advancing ourselves? This is really a snapshot of what we did. Precigen was basically initiated in 2020, so five years.
What we did in 2021, we cleared basically IND phase one, and we finished the phase one in the middle of the pandemic, and it started our single-arm phase two, and of course, then discussions with the FDA at that time. By 2023, you see that we basically FDA granted accelerated path 2024. At ASCO, we presented the data, pivotal data from our phase one and phase two. By actually in August of 2025, despite the fact that we were on an accelerated path for a conditional approval based on the safety, efficacy, and durability of the response, which I will take you through in a very short slide, FDA decided that there is no need for further trials, and we were granted actually a full approval in advance of our PDUFA date, which we were very pleasantly happy with this news.
As you can imagine, our commercial team started hitting the ground running in September of 2025. So first and only FDA-approved therapy, this is what Pepcimeus is. And that means a lot for the patients and also for the physicians that, by the way, they do not like using surgery because they know that surgery is not a treatment for these patients. This has been the only way that they could have kept the patients from actually tremendous difficulties and even death, because if they can't breathe, obviously, we know what the outcome is. So first and only FDA-approved for a disease that I mentioned, it's chronically challenging and debilitating rare disease. And we have shown really transformative clinical data, which I will take you through. And this has been the basis for FDA decision. And finally, we have been able to use this platform.
This is really, I think, it showcased the ability of the adenovirus platform. And for those of you who have heard Commissioner yesterday, Dr. Makary, actually one of the strategic ways of FDA moving forward is really the platform approvals now when a platform works across a certain indication and how it can be rapidly moving for other indications. So we are very excited about this for our portfolio. So let's look at a little bit about the pivotal data again for those of you who might have not seen it and the mechanism of action. Pepcimeus is really a drug that is given just subcutaneously, very simple injection. And it doesn't require any device. It doesn't require any kind of special challenges. Simply like the way you receive a flu vaccine, you get it in either arm or in the leg, very easy administration.
However, quite a sophisticated mechanism of action. What it basically does, it contains a number of epitopes, and it will basically sort of educate your immune system to find the infected cells or the cells that are the cause of these benign tumors and destroy them simply. That's the mechanism through the high affinity T cells. So with that, this is the pivotal data from our clinical trials. On the left-hand side, you see the response of our patients. We went to the most severe patient population, actually, and this was designed by purpose, because if you can make the drug work in that setting, obviously coming to the less severe patient population, it's well accepted. And that's what you see. We have patients in here that they had 10, for instance, surgery in a prior year.
Then by receiving a four-injection of Pepcimeus in a span of three months subcutaneously, they went to zero. They are looking at the complete responders. And first, when we started, was the minimum of 12 months. But what you see on the right-hand side, these patients now have been followed for years. And in the last actually news that we had out was we have followed these patients. Now they have passed median of three years, and they have not required any surgery or any other treatment for that matter. So this is quite exciting. And by the way, they are continuing to be in response.
And another group of what we refer to as partial responders. They also responded by reducing their number of surgeries, but they clearly didn't go to zero, which gives the ability for us to redose this patient, especially in view of the safety of this drug that basically when you receive it, it's a grade one or maybe a grade two, which is equivalent to when you get a flu shot, a little bit of fever, a little bit of rash on the arm, and then it goes away in a few days. So this is quite exciting. And this is the data that FDA has seen and the safety data and has allowed to move from a single-arm pivotal to full approval. So Pepcimeus, the commercial opportunity, and we will take questions afterwards.
The 27,000 patients in the U.S., in excess of 150, and we have done these studies now also in Europe. The four countries in Europe, plus the U.K. and Japan, we will be in excess of 35,000 patients, 50,000 patients in other territories, and clearly in China, a very high population, in excess of 85,000. So quite a large patient populations that, and this is adult in these cases. One of the things that there is a lot of discussions about the biotechs and how ready they are to commercialize, and the indication for the commercialization to support a successful launch is, first of all, you have to know your market size before you obviously launch your commercial teams out, and we had done that prior to approval, 27,000 patients in the U.S.
You have to optimize your footprint, knowing where these patients are located, so you don't have to deploy 300, 400 salesperson all across in the territories that there might not be that many patients. We had done those studies prior to the approval and had a very, very good understanding of the territories and also discussions with the payers to be able to establish a price that is going to be accepted by payers. Obviously, build awareness, both patient education as well as physician education besides just KOLs that they are out there. And we did that prior to our launch and approval. And finally, set up a distribution. Important. You can do all of that, but if you don't have your commercial material and you don't have your supply chain and distribution, obviously that is a recipe for failure, and we were not going to do that.
So with that in mind, after we received our approval in August of 2025, we exactly executed against all of that, and we went to a full mode of commercialization, full promotional campaign. And Phil will talk about this with me. We have now more than 96% of our target centers has been covered in the past three months. This is an incredible speed by anybody's count. As far as formulary and IDN and community support, we have more than 50 accounts here and that they have been prepared and ready, and they have been also prescribing Pepcimeus. The payer pull through, this is very important because this is a rare drug disease with obviously a different pricing. And we have already got coverage through Medicare, Medicaid. We have covered more than 170 million lives through big and small, basically private health insurances. And we will discuss this.
This is almost close to 80% of the coverage for these patients. And finally, the patient support hub. And I just want to stress, this is the hub just for our company that we have set up. In November, there were already more than 100 patients there, and we just reported in a matter of a month and a half, it's more than 200 patients in this hub. This hub does not just represent all the patients because the centers of excellence, they have their own hubs that they enter the patient. So as you can see, this is just a fraction of the patients that are coming in, and this is directly to the Precigen hub. And just to give you a view, that is a significant increase in a Pepcimeus brand awareness since approval. We went from 7%- 66% in a matter of three months.
And in the community centers, again, from 6%- 58%. This is a huge increase, and it shows the interest of the patients and the physicians in this drug because this is the one and only FDA-approved drug for this rare disease. And looking ahead, what is in front for us is obviously we are laser-focused on the commercialization of Pepcimeus in the U.S. This is what our commercial team is out, and continuously we are focusing to expand that. And we are very, very excited with the reception that this drug has had among the patients and the physicians. And by the way, this drug, the FDA has given a very broad label for Pepcimeus that all RRP patients, regardless of severity, can receive this. So if an RRP patient is just recently diagnosed, they can receive this drug.
And if the RRP patient has been dealing with this disease for 10, 20, 30 years, and we had patients that they had this disease from age of one, if you can imagine, excess of 300 surgeries, they will receive it as well. And this is extremely important because this drug is not just for severe patient population; it's for any adult RRP patient with diagnosis. And I would like to add one other point. Why is this important? The studies that have come out have very clearly shown these patients, by the time they are into their fifth surgery, they have irreversible damage to their vocal cords and to the trachea. And actually, over the years, a good third of these patients actually might have to go through tracheotomy. So that's why the patients want to receive this.
The physicians want to prescribe it because they know what the end result is. One more surgery is one more damage to these patients and irreversible damage. The regulatory expansion of Pepcimeus is very important for us. And as we have communicated already, last year, we submitted our application to EMA. We received the actually acceptance validation of that, and it currently is under review for EU and in EMA. We are planning to submit to Japan very rapidly, and we have had very, very constructive discussions with Japan FDA and very exciting ones. And we are initiating our Pepcimeus pediatric because this is a drug that, based on the safety, efficacy, and durability of response, it fits perfectly for children, and we do not want to be leaving children behind.
Again, we heard from the FDA commissioner how important it is to make sure that there are more drugs for the pediatric population. Finally, as I mentioned at the beginning, this is the proof of the platform for adenovirus. We are very excited about what we are seeing in our phase II data that will be reported in the future, both in cervical cancer and in head and neck, because we are applying the exact same actually vector with the same safety, with the same profile, and in HPV-related cancers. We are very excited with that and also from a perspective of regulatory strategy that we are moving with that. Finally, I'm going to finish by simply addressing Precigen has a very strong foundation for a long-term value creation.
Pepcimeus for us is our first, and we are very excited about a commercial drug, but it will not be the only one, and on our adenovirus platform, as I mentioned, we have two others that are in phase two and rapidly moving with this, so clearly, we have a technology that is differentiated. We have a commercial asset that is quite exciting and is out there. We are also expanding our territory to ex-U.S., and we have de-risked the technology platform, and clearly, a very, very solid leadership team that, as I showed you from our start or initiation of Precigen in 2020, despite all the challenges with the pandemic, we have gone from a discovery drug development in four years to approval, so this speaks to the expertise on every level of the company: research, development, manufacturing, and we have inside manufacturing as well as commercial.
So we are very excited about that. So on that, obviously, we also have established a very strong balance sheet that clearly allows us a runway that with the revenues of Pepcimeus to get us through the cash flow positiveness. So with that, I would like to thank all the audience and Brian, and we'll be happy to take questions. And I invite our Chief Commercial Officer to come with me also so we can answer any questions that the audience might have and Brian.
Thank you.
Well, thank you so much for joining us. Let's start the Q&A. For those who are in the audience, if you have any questions, feel free to raise your hands. For those joining us virtually, you can also submit questions on the portal. How has the Pepcimeus launch been going for, you know, going so far?
And, you know, are there any surprises from your vantage point today?
Okay, so I think I'm going to ask Phil answer that.
I mean, we're very pleased with the launch. As you saw, some of the leading indicators in terms of patient identification, site activation. I think the surprises that we've had have been positive ones, been nice surprises. A couple I would call out is one is the speed of payer uptake. You know, that number of 170, approximately 170 million covered lives plus Medicare plus Medicaid. You know, that's a significant portion of the target market already covered within a quarter and a half of the full launch. So we're thrilled with that. The second pleasant surprise has been community interest. A lot of those patients that Helen talked about in the hub are actually from the community.
So not only the academic institutions, the large hospital systems or IDNs, but the community. And that has been very pleasing from a market perspective in terms of embracing this as the new standard of care. On one of your slides, you said that more than 50 accounts have prescribed Pepcimeus. Can you tell us if these 50 accounts are, you know, are these numbers reflective of the commercial products, meaning that, you know, this is revenue-generating and they should reflect in your fourth quarter number? So the prescription means that those patients have been selected by the institution to go into the process by which they have benefit verification and ultimate payer adjudication. So, you know, there's a transmission mechanism from prescription to revenue realization. We'll talk more about some of that at the next earnings call.
We won't be talking about specific numbers of patients or revenue projections, but that is the first step in getting these patients onto treatment.
Yeah, and maybe I can add what is very important and I think should be recognized that if you can imagine that our commercial teams basically will receive this approval in August of 2025, and then there is a process, especially for bringing in the centers of excellence, because all of them, they have their committees, approvals that especially for a drug that has, and there is no precedent for it, and then formulary committees that has to go.
So obviously, the team has done an outstanding job in the Q3, Q4, covering all of that and very rapidly bringing this, which will be leading to, sorry, Q1, Q2, and really the increase in the prescription and as well as the, we already have seen enrollment of the patients and the patients have been dosed across the nation from East Coast to West Coast. And we are very happy that also that speaks to our supply chain and manufacturing capabilities.
Brian, the thing I would add is, you know, just to remind everyone that we are fundamentally transitioning, transforming this therapy, this condition from what was a surgically managed condition to a medically managed condition. I mean, that's a deep systemic change that has to impact all facets of the healthcare system.
So we don't take that lightly, but as you can see, the early signs and the momentum that we've created is very pleasing and I think consistent with the establishment of a new standard of care in the therapy area.
With the unrestricted label, how should we think about just the receptivity of usage among the milder versus the more severe subset of the population?
Yeah, I mean, I'll say a couple of things and then maybe Helen, you know, we've got clear signals. Helen showed the increase in awareness, which comes from our quarterly ATU research. We do that every quarter to understand how sentiment, trial utilization is happening in our target audience. There's a clear indication from those doctors in the community and in the academic institutions to use the drug across all severities.
I don't think we should be surprised that some of the earlier patients may be more severe. That's where the highest unmet need is, and we see that in many drug launches across many therapy areas, but we fully expect as we go forward that we will see utilization across all severity,
and maybe I can add this is what we are hearing from our patients. As I mentioned, the study that came out, I believe it was either a year or a year and a half ago maybe from Johns Hopkins, it pointed out to the patients that by their fifth surgery, as I mentioned, they have irreversible damages, and therefore, patients are very educated about their disease, and what we are hearing is really the need to receive these as early as possible, so they do not even need to have one extra surgery at this point.
I think this is a very, very important. The physicians and the surgeons, we have to say, they agree with that because the surgeons in general, this is not the type of the surgery that they like to do because they know the outcome what it is, which means another surgery and further damage to these patients.
So. Any questions from the audience? Just going back to your comment earlier related to committee approval and formulary, you know, can you talk about just the process, you know, the steps involved in terms of getting patients on drugs? So, you know, if a patient gets a prescription for Pepcimeus, what happens after? And I guess what are the steps involved and also how long does it take for patients to get the first dose of Pepcimeus?
Yeah, there's no single answer in terms of how long it takes.
It's very specific to each patient actually and each institution. But certain things have to come together. Obviously, the patient has to be identified and we've seen that already from the numbers that Helen mentioned. That then starts the process of benefit verification to make sure that the patient is supported from a benefits perspective. You have to interact with their health plan. The payer has to then sign off on the coverage. And of course, the institution has to be ready from a formulary perspective to be able to administer the drug. Although actually in advance of some of these formularies being set, and we are seeing a lot of formularies already come on from these big institutions, but even in those that haven't had their formularies approved yet, we're seeing use through a medical exception process.
So there's a number of steps that have to come together. I wouldn't put a timeline on that. In some cases, it's relatively quick. In others, it can take a bit longer. But the most important thing from our perspective is that signal of intent, which is represented by the patients in our hub, but also the patients that we know are going through the IDNs and not necessarily in our hub. And some of the early patients that have been treated have actually not come from our hub. So there's sort of two parallel paths of patient demand that we're working through at the moment.
And maybe if I can just add this, the most important thing was getting the coverage by the payers.
Imagining that in three, four months to cover almost 80% and 170 million lives and even Medicare and Medicaid through the government shutdown, this speaks again to payers having been educated about what is the need and the power of this drug. And then also having these approvals from all large and small payers and insurance companies, it really has helped the patient uptake.
And in fact, in a few cases, we've had a payer that hasn't issued its formal policy yet approve the drug through a medical exception process when they've had peer-to-peer discussions with the treating physician, which again, I think speaks to the high unmet need and the value that we're delivering to the treatment of the condition.
Great. Can you talk about just, you know, your current financial outlook towards cash flow breakeven?
What are the assumptions that go into the projection that, you know, you will get there? How do you think about the projection of also revenue accumulation over time from Pepcimeus?
Okay, so as we have mentioned, the guidance that we have given with the cash that we have at hand and we raised actually last summer, which with a dilution to our shareholders, this will put us in place with the revenues that will be coming. Currently, we are not giving any guidance from a perspective, but we have said and continue saying that this will bring us to the cash flow positive by the end of this year, and we will be obviously communicating further as we go along.
But, Brian, to your point about 2026, 2027 outlook, I mean, we've obviously looked at analogs, we've looked at rare disease uptake, we've looked at the situation that we're the first and only treatment for this condition. So we expect our trajectory to be consistent with that, the philosophy of establishing a new standard of care in this therapy area.
Okay. What would be a fair comp, just, you know, kind of latching on your last comment, you know, what would be a fair comp just, you know, just to understand how the trajectory will look like? So I think from a trajectory, we obviously are expecting Q1, Q2, and we are increasing in our patients' uptake. And we will be, that is what we have accounted for.
I think we are very pleased with what we are seeing again through the patients coming in not only to our hubs, but to the others and also from our physicians that across the United States have been reaching out and going back to this concept of community, which was a very pleasant surprise because we originally thought all or majority will be through the actual centers of excellence. We are finding that that is not the case. Actually, this is a huge boost. We are very excited about that, that the community, basically physicians, are very, very advanced in knowing what this drug is all about and wanting to prescribe it to their local patients.
Great. Maybe just, you know, in the minute we have left, how do you think about just the ex-U.S. opportunities?
You know, how do we think about partnership potential as well?
Absolutely. As we mentioned, we have already submitted our application and it's validated for EU and we are planning for Japan submission as well soon. This is obviously very important, ex-U.S., but definitely we are also very much interested in looking at partnership for ex-U.S. as our focus is currently and laser focus is on U.S., and we are evaluating various options from that perspective as hopefully we receive the approvals around the world,
and in the meantime, we do a lot of the work, the groundwork obviously on pricing market research, understanding sequence of countries and all the work you need to do. We're part of the new JCA process in Europe at the moment, so we're doing all the groundwork, but as Helen said, partnership is one of the key options.
Great.
Thank you so much for your time. Thanks for joining us.
Thank you for having us.