Compugen at Stifel Forum: Strategic Insights on Immunotherapy Pipeline

On Wednesday, 09 April 2025, Compugen Ltd (NASDAQ: CGEN) participated in the Stifel 2025 Virtual Targeted Oncology Forum. The conference call, led by CEO Anat Kowandiyag and CSO Haran Ophir, provided a strategic overview of Compugen's clinical-stage cancer immunotherapy pipeline. The discussion highlighted the company's strong financial position and partnerships, particularly the COM701 program in collaboration with AstraZeneca. While the outlook is largely positive, challenges in the competitive landscape were also acknowledged.

Key Takeaways

• Compugen's COM701 program focuses on an anti-PVRIG antibody for ovarian cancer, with ongoing trials.
• The company benefits from a robust financial position, with cash runway extending into 2027.
• AstraZeneca's rilvegostomig program, a PD-1/TIGIT bispecific antibody, is a key partnership focus.
• Compugen is leveraging its computational platform to discover novel drug targets and expand its pipeline.
• The company is open to strategic partnerships to advance its programs.

Financial Results

• Cash runway extends into 2027, indicating strong financial health.
• Compugen is eligible for over $11 billion in milestone payments and royalties from existing partnerships.

Operational Updates

• COM701 (Anti-PVRIG Antibody):

- Ongoing clinical trial in recurrent platinum-sensitive ovarian cancer.

- Adaptive trial design allows for potential combination therapies based on interim data.

- Interim data expected in the second half of 2026, with the potential for earlier insights.

• AstraZeneca Partnership (Rilvegostomig - PD-1/TIGIT Bispecific):

- Eight pivotal studies are ongoing, targeting lung and gastrointestinal cancers.

- AstraZeneca projects peak year revenue of over $5 billion for this asset.

- Upcoming presentation at the AACR conference in May for troponin lung twelve trial.

Future Outlook

• COM701 Development:

- Plans to discuss a path to registration with regulatory authorities if trial endpoints are met.

- Considering COM701 as a backbone for combination therapies in earlier treatment lines.

• Partnership Strategy:

- Open to partnerships to advance programs, including COM701.

• Pipeline Expansion:

- Aims to introduce more first-in-class assets through its target discovery platform.

Q&A Highlights

• COM701 Trial Design:

- Randomized, blinded study with 40 patients on COM701 monotherapy and 20 on placebo.

- Stratification by prior treatment with SPARC for population homogenization.

• Competitive Landscape:

- Monitoring GSK's PVRIG targeting antibody and other clinical programs.

- Awaiting Phase 3 data from AstraZeneca and Gilead's TIGIT programs for COM902 evaluation.

• AstraZeneca's Rilvegostomig Strategy:

- Focuses on tumor types responsive to PD-1, using a bispecific antibody in combination therapies.

For further details, readers are encouraged to refer to the full transcript below.

Full transcript - Stifel 2025 Virtual Targeted Oncology Forum:

Steven Willey, Senior Biotech Analyst, Stifel: Alright. Hello, everyone. I'm Steven Willey, one of the senior biotech analysts here at Stifel. And glad to have with us here for the next presentation Compugen. Representing Compugen, we have Anat Kowandiyag, who is the chief executive officer, and Haran Ophir, who is the chief scientific officer.

We're gonna kinda have an informal discussion. I think most of you know that there's a q and a function that you can populate should you have any questions. We'll try to get those asked and answered as appropriate. It's good to see you both. Always appreciate the time.

Always good to chat. And now I'm not sure if you just wanna kinda make any opening statements here before we get into q and a. Maybe just give us a quick overview of the Compugen story.

Anat Kowandiyag, Chief Executive Officer, Compugen: Sure. And thank you, Steve and Stifel, for for inviting us. Yeah. I'll give an overview. Compugen is a clinical stage cancer immunotherapy company.

We discover new drug targets by a computational discovery platform that we developed over the years. That's basically a mix of artificial intelligence and machine learning with human expertise, and we developed first in class drugs to address these drug targets. We have a rich pipeline, validating partnerships. We're eligible for more than $11,000,000,000 in milestone payments plus plus royalties, and we have a solid balance sheet with cash runway into 2027. So we're well positioned for growth, and we're looking forward for this discussion with you.

Steven Willey, Senior Biotech Analyst, Stifel: Alright. Maybe we can start in on COM701. You know? So this is an anti PVRIG antibody. You're one of, I think, two companies that are currently in the clinic with a version of this.

You know, you've talked about looking at this as single agent maintenance therapy in recurrent platinum sensitive ovarian cancer. I wanted to maybe just kinda get into the design specifics of the trial a little bit, but maybe first, we can just kinda talk about the rationale for this. Right? So I know you first generated a lot of COM701 based triplet data in the platinum resistant setting, both in combination with PD one and with TIGIT. You know, you're now pursuing this single agent COM701 trial as as maintenance therapy in in in platinum sensitive patients.

You know, I guess we know the history of IO based therapy in ovarian isn't a great one. So I guess my questions are, you know, what's the data that you have to support monotherapy activity within this tumor type specifically? And what's kind of the underlying biological rationale to support this pivot that you're now making into the into the platinum sensitive setting?

Haran Ophir, Chief Scientific Officer, Compugen: Sure. So first of all, I would say, yes. Checkpoints did poorly and miserably in ovarian cancer. And the reason is because ovarian cancer has a very strong suppressive microenvironment, and it's it's difficult for checkpoints to induce sufficient anticancer immunity. Right?

So why PIVRAG will do what others have failed? I think, like, everything we do, first of all, is the biological rationale. PVRIG has a very, very different biology from all the other checkpoints. And we published last year a very elaborated paper in a high profile peer reviewed journal, how the biology is different. And that biology enables PVRIG to drive additional waves of T cells in indication which are typically not responsive to other checkpoints.

And this is the biology, and this is the signals we have seen. In few trials that we did in difficult to treat patient population, in patients which are PD L1 negative, in patients in indication which are not responsive, we are seeing that PVRIG blocker COM701 is active. So this is in general about the biology, why should it be active or other checkpoints have failed. Then we had our own data in ovarian. In ovarian cancer was a target indication for us from the beginning.

The PVRIG pathway is very highly expressed in that indication. And we started actually by seeing a monotherapy signal. We didn't treat many patients in in monotherapy because in this platinum resistant ovarian cancer patients, which failed everything, we thought that the best chance for the patient would be eventually to to combine more drugs. TIGIT and PD-one, we have the hypothesis why we thought it will be more active. But we started by having a small cohort in monotherapy in which we have seen clinical response in monotherapy.

And we also seen that in the most of the patients we treated, we saw translationally that we induced T cells exactly with the MOA of COM701. Then we moved to the triplet mostly. And what we wanted to see there, do we have a signal? And the circle data was p d one or p d one even plus digit giving you, like, something like eight percent of response. And we had across 40 patients in triplet, a twenty percent response or close to that.

And and that's what we we in addition to the monotherapy data, we said, okay. We have a signal that COM701 is active in ovarian cancer. Now looking at the data of the trial, we saw that many patients relapsed so fast. They probably never even had the chance to respond to chemotherapy free regime like the triplet IO that we used. But the patient who did respond had very long term responses, excellent safety profile.

So the rationale is we take this drug that showed activity in the last line patients, which is safe and durable in responses into the maintenance settings. So we're going into patients who are second or third line platinum sensitive. They're still platinum sensitive. They responded to the platinum, so they have low tumor burden, more active immune system. And then we're starting this adaptive trial design.

We're gonna discuss the trial in a sec. But we're starting with monotherapy maintenance settings to see if these signals in the last line and the biology is translating indeed to an efficacy in PFS in the maintenance settings in patient who responded to the second or third line of platinum.

Steven Willey, Senior Biotech Analyst, Stifel: Okay. So I guess I guess that's kind of an overview of the of the of the biological rationale and, the monotherapy approach here. So so as you think about the trial now, I think it's been posted to clintrials.gov. I don't think it's actively recruiting yet. But maybe you can just kinda talk about some of the key design features, you know, in terms of in terms of patient numbers, eligibility criteria, endpoint assessments.

And then maybe you can also just kinda talk about, you know, how quickly you think you can complete patient enrollment into this study and how big of a of a trial site footprint do you need in order to in order to accomplish that?

Haran Ophir, Chief Scientific Officer, Compugen: So the trial is in adaptive trial design, which gives us a lot of flexibility. And we start, as mentioned, by COM701 monotherapy. But then we have the flexibility to add more combination. I will discuss it. So we are talking about first, second, or third line platinum sensitive patients.

They are still platinum sensitive, meaning they respond to the platinum. They already received bevel PARP bevel and or PARP, or they're not eligible to receive bevor PARP. So these patients have no standard of care. They receive the platinum, six cycles, and normally, they sit and wait or they they receive receive some off label. And eventually, they will relapse after a median of five point five months and eventually will become last line or platinum resistant.

So we're talking about this kind of patients. We're excluding patients with liver metastasis. We are stratifying by prior treatment with SPARC because we thought that this is an awesome discussion with KOL, like a key thing that could homogenize properly the population, the previous SPARC treatment. And then we're randomizing. It's a double it's a it's a randomized blinded study in which you're gonna enroll in the first part.

40 patients COM701 monotherapy and 20 patients, so it's a two to one, that we're gonna see a placebo. And then this is gonna read in half two twenty six. And then we have the flexibility to move forward either to continue in the monotherapy, maybe even some faster designation. We can then add combination. So as mentioned, it's an adaptive trial design that after the first part of the monotherapy of the flexibility to make different choices.

Anat Kowandiyag, Chief Executive Officer, Compugen: Yeah. And maybe to get to See, doctor Maybe

Steven Willey, Senior Biotech Analyst, Stifel: to the

Anat Kowandiyag, Chief Executive Officer, Compugen: enrollment yeah. Maybe to get to the enrollment part, you were asking about, about, this and the sites. Look. This specific indication, the platinum sensitive is less competitive than the platinum resistant, specifically the patient population that we're targeting is a patient population that is not candidates for for maintenance treatment. But since most of the patients are going to be a third line third line platinum treated patients, it's about thirty percent of the second line, which is second line is about thirty four, thirty five thousand patients.

So on one hand, it's less competitive. On the other hand, it may still be challenging due to the size of the population. But we feel that, you know, we we know the investigators from prior studies. These are ovarian cancer investigators. We know them.

They are engaged, and we have the relationship. And we will open enough sites in order to address it, and we'll keep flexibility in how many sites we need to open in order to keep the pace of the enrollment.

Steven Willey, Senior Biotech Analyst, Stifel: Okay. You talked about progression free survival as a primary endpoint. You know, can you maybe just kinda speak to the data that you have that informs that assumption around progression free survival both in the control arm? And, I guess, what is the magnitude of difference that you would deem to be clinically meaningful that you would want to see COM701 do in terms of in terms of magnitude of benefit above and beyond a control arm?

Haran Ophir, Chief Scientific Officer, Compugen: So in this case, in this patient population, actually, there is quite solid historical control. There are quite few phase three trials in a similar population that showed quite consistently that the PFS is around 5.5 months. So this is what we expect to see in the placebo. And then we we think, and after the the discussion with the three months improvement. So 5.5 for the placebo and then three months improvement in PFS in the COM701 monotherapy is will be a win.

And then, again, we can have the flexibility to choose how we continue depending on how big is the effect. It could be more than three months of effect, and then you can think also on an accelerated approval and other options we can think about after seeing first that you have these three months of improvement.

Steven Willey, Senior Biotech Analyst, Stifel: Okay. So it sounds like you do have some confidence around the control arm in terms of the assumption that you're making here.

Haran Ophir, Chief Scientific Officer, Compugen: Yeah. Very Which obviously

Steven Willey, Senior Biotech Analyst, Stifel: important in in in winning in any randomized controlled trial. But I know the randomization scheme that you're using, obviously, you wanna get as many COM701 data points as possible, but your control arm consists of 20 patients. Right? And so there's obviously some, potential for inherent variability to pop up. So how are you controlling for variability in this trial such that, you know, it's it's it's not heterogeneity that then, you know, overrides this control arm assumption that you have on the progression side?

Haran Ophir, Chief Scientific Officer, Compugen: Yeah. It's a very good point. So so, yes, we had the decision to have 40 to 20 to have, as you mentioned, more constituent treated data points. And the reason we did that because because of that solid historical control. The the I mean, we know when we have clinical development in different trials, but in this case, we really have a solid data in few phase three studies to show what we should expect in the placebo, so we think that's why we could allow ourselves to go a bit lower in the in that to 20 patients.

While in addition, we also have a very strict inclusion criteria to make sure we have an monogenized the population going to the trial. As mentioned before, these are patient who respond to the platinum. These are patient who were excluded patient with liver metastasis. We are we are stratifying by previous exposure to POPs. So we think that being very strict on the population, in addition to the historical data, should make this trial a trial that we could read into the conclusion if COM701 is active in these settings.

Steven Willey, Senior Biotech Analyst, Stifel: Okay. You mentioned data would be available in the second half of twenty six. Is is that a final analysis? Have you structured in any kind of interim or futility analysis into this trial? And I guess if so, you know, what triggers, any kind of interim or futility analysis?

Haran Ophir, Chief Scientific Officer, Compugen: So so the planned interim analysis that will happen anyway is half to '26. In addition, there was a data monitoring committee that review regularly the data. And if they will think either that the trial is futile or that the reserve is actually to unblind the data and move to the next step because we see activity, it could also happen earlier.

Steven Willey, Senior Biotech Analyst, Stifel: Okay. Okay. So second half twenty six for the interim, but a DMC decision could potentially occur before that. Alright. So let's assume that COM701 achieves the benefit that you're hoping in this trial.

What do you think are the next steps? I mean, do you just run this study back as a single agent registrational study in a randomized control manner? You know, do you start to look at combination based approaches here maybe in earlier lines of maintenance therapy? Obviously, it's all data dependent, but what are the options here that you think you may potentially have?

Anat Kowandiyag, Chief Executive Officer, Compugen: Yeah. I think as you said, first, it will be data dependent. And as always, we're data driven. That's the way we'll make the decisions. But I think that positive data opens the door for us for multiple paths forward.

One would be to to go and discuss with the FDA, with the regulatory authorities, and to understand what is it that we should do in order to take this COM701 as a single agent for an approval in this specific patient population? What would it require us to do? And build a path to registration. That's one. The second would be to to expand the test study, the platform study, and test COM701 as a backbone as a as a backbone for combination for different combinations.

Either go to earlier settings as you as you indicated in combination with Bev and Pop, other combinations. Obviously, we're still believing the triplet combination, and we we we may see how this would fit, but but it will open a door for us to use it as a backbone for for drug combinations. I think that with this, you know, we're not looking forward also to take we're not looking to take this program forward alone. So maybe part of it will be do internally and some of it with partners. We'll see.

But we'll be data driven.

Steven Willey, Senior Biotech Analyst, Stifel: Okay. So we know Glasso also has PVRIG targeting antibody in the clinic. I think this was procured through the surface transaction, if I remember correctly. Right. What kind of competitive intelligence do you have on this asset at all?

And, you know, can you maybe talk about what GSK is currently doing within the clinic?

Anat Kowandiyag, Chief Executive Officer, Compugen: So GSK, as far as we know, they did not present any data, anything in the in the public. So, you know, we don't have specifically any competitive intelligence, but they progressed the program to phase two, and they tested as a triplet combination. So that one. So they're following basically the DNAM hypothesis that we, you know, that we came up with, and we'll wait to see their data. Obviously, any data from GSK and by by the way, also from others that are testing it in phase one studies.

There are others as well. So for example, Bioceus is testing it as a as a bispecific. So any data from any other party, any clinical data, may serve as a validation. And I want to I want to remind that, you know, we have solid IP. And I think that maybe, you know, if you're following the public domain, you'll see that the IP that we have

There are there is there are attempts to challenge our IP. And as of now, these were not successful, but we see it also as a validation to what we're doing.

Steven Willey, Senior Biotech Analyst, Stifel: Mhmm. Mhmm. Okay. Maybe we can talk a little bit about the partner programs. Right?

So you have the partnership with AstraZeneca. This is a PD one TIGIT bispecific rilvagostomyg, which I usually have a very difficult time saying. Astra's put a ton of resources behind this. Right? So Astra's putting a lot of a lot of muscle behind this program.

I think, what, seven phase three trials now, another five to 10 phase one, two trials. I guess when you look at the suite of phase three studies that are now enrolling, you know, which are you the most excited about, and where do you think the biology makes the most sense?

Anat Kowandiyag, Chief Executive Officer, Compugen: So I think before going more to the specific studies, I think that I'll say first, it's by now, it's eight pivotal studies. So, you know, we're still counting. Okay. Yes. It's eight, and they are spread across lung and GI, gastrointestinal.

They use very broad and com comprehensive strategy, which is really to replace the p d one the p d one, PD L1 inhibitors. They are testing it in multiple combinations, chemo, and ADC. They're addressing really different patient populations. So really in lung, they have so many studies where they are testing different subpopulations of non small cell lung cancer in a very methodical way comparing to standard of care. They they are really allocating a value of a peak year revenue target of more than $5,000,000,000 for this.

I think that, you know, they already presented compelling promising data in gastric and in lung in 2024. It seems comparable to to to what's been what what have been there with the standard of care or with the PD ones, and they are pursuing indications where PD one works. So when you're talking about the biology, I think that AstraZeneca is taking the strategy to address the tumor types that are the more inflamed ones, those that where PD one works, and they're investing their resources there. And they plan to present data in 2025 for the combination of rilvagostomy with ADCs. And, and, by the way, they're also going to present, trial in progress for troponin lung twelve, as as an oral poster in the a AST conference in May.

So that's very soon. And I think the fact that they are targeting the biology with the with the with the specific tumor types and really focusing on on the various patient populations in these indications is a is a smart way to move. But, you know, Iran can give maybe examples maybe better examples and diving into the details of the studies. Yeah.

Steven Willey, Senior Biotech Analyst, Stifel: And maybe, Ron, maybe in in in in speaking to this, you can maybe offer up your opinion on their strategy to pursue ADC based combos. Right? That appears to be kind of a core element of what they're doing. Would just be curious on your thoughts around that as well.

Haran Ophir, Chief Scientific Officer, Compugen: So they have Rilve as a potential new IO backbone, which is safe because it's not binding Fc receptors, and it's potentially more active than p d one because it's a p d one TIGIT bispecific, and they have shown it, at least in single arm studies, to be superior to historical data of p d ones. And then you have the smart chemos, right, the ADCs, which are maybe a bit like chemotherapies, inducing immunogenic cell death, reducing tumor burden, and makes the environment favor more favorable to the TIGIT p d one relevant to be active. So I think this is really exciting. And what is interesting also in some of their studies, even though we think and it was shown that TIGIT is active, because they have a bispecific molecule, they don't really have to prove it. So if they are doing a combination of RILEVE and ENERTO and compare it to pembro plus chemo, they are combining the forces of two strong drugs versus pembro plus chemo, they could get approval potentially even without showing directly that Rilve is beating pembro even though I think it will.

So this is, I think, what's interesting about their strategy. Okay.

Steven Willey, Senior Biotech Analyst, Stifel: But we had a discussion this morning with Tom Maren from Sinai. He gave a shout out to this drug. He he's he finds it interesting. So he's obviously very well schooled in all things lung cancer. I mean, it doesn't seem like Astra gets a ton of credit for this asset.

Not that I understand the intricacies of Astra valuation all that well, but I'm guessing it's probably a byproduct of the TIGIT component here. And, you know, look. It's been the space littered with attrition. I think Beijing dropped out of this space last week. You know, you have com nine zero two.

You've kinda paused development there. Is there anything that you're kinda monitoring now on the competitive landscape? Anything that you're kind of anxiously awaiting here to maybe give you some kind of confidence that $9.00 2 might be, an asset either worth dedicating resources to internally or maybe trying to pursue opportunities externally.

Anat Kowandiyag, Chief Executive Officer, Compugen: Yeah. Sure. Obviously, we're monitoring the field, and I'll explain our approach to it and what what are we looking for. I think in fairness, there were many failures, and there is a lot of skepticism. We were always saying that not all digits are the same.

And that and we're focusing not only on the for not only on the format, which is Fc silent or inactive, but but in general, we believe that our antibody, the COM n o two, is is a very good antibody. We reached to the conclusion that there is no study that we can do that is a phase one or two study with our TGT antibody that will make a difference. We really believe that the that the field needs to see a very good phase three study, positive phase three study in order in order to make a difference. AstraZeneca and Gilead are having the right format, and they're pushing forward. They have the studies.

We wait to see how their studies are panning out, and we believe that this will also reflect on us not only by what we, you know, what we're eligible to from AstraZeneca in terms of financial reward, but also in terms of how it reflects on our community too. At the end of the day, we're not familiar with that many companies that have an FC silane that is ready to be tested if we're correct with what we're saying in this, in this TIGIT space and the antibody format.

Steven Willey, Senior Biotech Analyst, Stifel: Okay. Maybe last question because I know we're pressed up against time here. So, I mean, look, the company has a very good history, very validated history of of of leveraging, you know, this computational platform that you have to discover novel targets. You guys were first with Digit, first with PVRIG, first with IL 18 binding protein, which is now in phase one partnered with Gilead. We need a chance to talk about that.

But how big of an effort do you still have ongoing there with respect to kind of target discovery and validation? And should we expect those efforts to yield any kind of pipeline expansion over the course of the next one, two, three years?

Haran Ophir, Chief Scientific Officer, Compugen: So so definitely. I mean, we use this IA AI based platform, which is focusing on target discovery. Not many companies are taking the challenge of identifying new drug targets to develop first in class. So we are doing this when combining the very strong computational capabilities in AI, which something that people some tend to forget, and non AI. I mean, we also use human intelligence, and we have the know how to take this kind of novel drug assets all the way from clinical prediction to validation and clinical testing, and the platform is proven, as you mentioned yourself.

So definitely, we use all the resources we got from our collaborations to continue and build our internal computational platform and continue to build our internal pipeline, which is derived from this competition platform. So, definitely, we do expect to bring more assets with our first in class potential into the field.

Steven Willey, Senior Biotech Analyst, Stifel: Alright. I guess we'll stop there. Anat, Aran, always a pleasure to speak with you guys. Best of luck, and I'm sure we'll be catching up here over the near term. Thank you, and thanks, everyone.

Haran Ophir, Chief Scientific Officer, Compugen: Thank you.

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