Exelixis at Barclays Healthcare Conference: Strategic Pipeline Progress

On Tuesday, 11 March 2025, Exelixis (NASDAQ: EXEL) presented at the Barclays 27th Annual Global Healthcare Conference, offering insights into its strategic pipeline progress. The company highlighted ongoing developments in its Cabozantinib (CABO) and Zanza programs, while also addressing potential challenges. The conference showcased both optimism and cautious planning, reflecting Exelixis’ commitment to advancing treatment options.

Key Takeaways

• Exelixis is confident about the PDUFA date for CABO’s NET indication, leveraging prescriber familiarity.
• The company anticipates a $1 billion market opportunity for CABO in NET.
• Zanza’s development is progressing, with promising data from the STELLAR-1 trial.
• Strategic collaborations, particularly with Merck, are crucial for resource leveraging.
• Exelixis maintains a robust supply chain to mitigate potential disruptions.

Pipeline Progress

Cabozantinib (CABO):

• Exelixis is focusing on the NET indication for CABO, with a PDUFA date set for April 3. The company estimates the NET market opportunity at $1 billion.
• The prostate NDA submission has been temporarily deprioritized but will be revisited post-NET PDUFA resolution.
• A small increase in the sales force was made in Q4 to prepare for the NET launch.

Zanza Development:

• The STELLAR-1 trial results indicate the Zanza + Atezolizumab combination outperforms monotherapy, with favorable comparisons to Regorafenib in non-LiverMet CRC.
• Pivotal data from CRC trials and updates from STELLAR-2 are expected later this year.
• Collaborations with Merck are underway for RCC studies, evaluating Zanza and Belzutifan.

Regulatory Environment and Planning

• Exelixis maintains a redundant supply chain, sourcing from multiple countries to ensure product availability.
• The company reports a positive relationship with the FDA and is engaged in ongoing discussions regarding regulatory processes.
• While NIH budgetary cuts are a concern, Exelixis does not foresee immediate impacts on its operations.

Future Outlook

• Exelixis is optimistic about changing the standard of care through compelling data that extends patient survival.
• The USP1 inhibitor program is positioned as a promising next step, with potential to enhance responses in combination with PARP inhibitors.
• Scenario planning remains a priority to navigate potential challenges, including regulatory shifts and supply chain issues.

Conclusion

For a more detailed analysis, readers are encouraged to refer to the full transcript provided below.

Full transcript - Barclays 27th Annual Global Healthcare Conference:

Unidentified speaker: With me, I’ve got Andrew Peters, SVP of Strategy and also Chris Senna, EVP and CFO. And maybe the first question I’ve been asking our company is just because of the at least part of the disruption we’ve seen recently. The supply chain disruptions, if there’s any kind of you’re worried about thinking about how you’re planning and if there’s any parallels there with COVID?

Mike, Exelixis: Yes. So Peter, thanks for having us. It’s been a great set of meetings today. And so but before we start, we just want to say that the things that Andrew and I will be part of today, we’ll have make forward looking statements and for a full understanding of the risks associated in our business refer you to our SEC filings. From your question perspective, we have a redundant supply chain we source for many companies.

And so we feel comfortable where we are from a supply chain perspective. We source from India, we source from China, but we have a very redundant supply chain that allows us to survive things like COVID. We had a lot of inventory onshore and allowed us to operate and make sure patients had product on time. Good.

Unidentified speaker: And then as we think about the FDA and potential disruptions there, is the dialogue dropped off, slowed down, any noticeable changes, are you anticipating any changes?

Mike, Exelixis: Yes. I mean, I guess, we’re in the process of discussions with the FDA. Obviously, we have a PDUFA that’s on April 3 for the net indication. And we’ve always had a positive relationship with the FDA and it continues. And I can’t say that anything’s really changed from our perspective.

So it seems to be normal.

Unidentified speaker: And any impact do you think or worry about from like NIH budgetary cuts? I know you’ve had fantastic ISTs in the past. So just curious if there’s any near to mid term impact that you kind of worry about?

Mike, Exelixis: Yes. We always worry when there’s change. But we don’t really see anything directly impacting us right now from an NIH perspective. Obviously, there’s a lot of great research that happens at that level and we as a country benefit from that. And so it’s an important part of some basic research that companies like us utilize, but there’s no direct impact that I would say.

If Andrew, anything else?

Andrew Peters, SVP of Strategy, Exelixis: No, I think that’s exactly the point is that it’s certainly an area that we as an industry are trying to kind of wrestle with and understand what those dynamics are. But at the core of it, our industry is dedicated to helping patients, kind of full stop. And how the NIH and kind of our collaborative relationship over the years kind of plays into that dynamic is something that we want

Mike, Exelixis: to see continue because ultimately we’re here to help patients. Yes.

Unidentified speaker: Are there other peripheral effects that we should be thinking about with the change in the administration? I kind of in my head kind of run through tariffs and supply chain, FDA, NIH, but are there other kind of secondary effects we should be how do you kind of plan for it?

Mike, Exelixis: Yes, we try to think through everything. Obviously, we don’t know everything is going to happen in the future. Otherwise, we’d be doing different jobs. But we try to plan for that. We try to risk mitigate.

I can’t tell you that we’ve come up with anything that you haven’t asked already. So, we’ll continue to manage the business and plan for the business in the best way possible based on the available information and we’ll try the scenario plan. That’s what we get paid for is to run the business and manage the risks that are associated with the business. Yes.

Andrew Peters, SVP of Strategy, Exelixis: I mean, one of the themes that we always kind of joke about is that Exelixis is really good at scenario planning, just because I think we want to prepare for and have all of these opportunities or optionality kind of at our fingertips. And so we had asked that question a lot in the last twelve months around the ANDA. So certainly, it’s an exercise that we’re familiar with. Okay.

Unidentified speaker: Fortune prepares benefits to prepared, right? Yes. So on the covers Antenib franchise, I mean, you mentioned kind of the looming PDUFA and kind of if we should think about that net opportunity, what’s the overlap of prescribers, patients or prescribers and your sales force?

Mike, Exelixis: Yes. So we talked about some of this on the third quarter call, but we see there’s a universe around the universe of around 3,500 prescribers or so and prescribing for net of which we cover probably 80% of those right now. And so we have good overlap and doctors have good knowledge of cabo. So we’re really excited about that. And then the rest of the universe is a good proportion of those that it’s been a call point before and they have some familiarity with cabo, either as part of another group organization.

So we’ve been planning for net for the last several months. We added sales force, small amount of sales force in the fourth quarter and we’ve been planning, we have scenario planning for net for many months and we’re ready to go. We’re looking forward to a launch and we’re excited for the opportunity to be able to communicate the data. Obviously, we don’t promote off label or anything like that, but when it’s approved, we will start promotion right away.

Andrew Peters, SVP of Strategy, Exelixis: And yes, I mean, just to kind of add to that, one of the dynamics that I think comes through when PJ talks about this or when Mike talks about kind of our enthusiasm for the launch, it’s that inherent familiarity with cabo that I think is one of the real kind of initial drivers for us. Because as you think about how patients are managed and kind of these AE protocols, physicians get to when a new drug is launched, physicians have to understand how the drug is used, how to work through adverse events, how to down titrate patients, how to do all of those things. Given the overlap and prescriber base with existing cabo prescribers for other indications, there’s just not really as much of that that we need to do. And I think the other thing candidly is when you have a data set like ours in the cabinet that was published in the New England Journal, It’s one that I think has been embraced enthusiastically by the community.

Mike, Exelixis: And we’ve done market research and we show the profile of the drug without name and with physicians and they’re like, yeah, we’ll prescribe it. And then when we cabo, we know cabo. And so it’s, we think it’s going to be very well received as another option for patients and doctors.

Andrew Peters, SVP of Strategy, Exelixis: Okay,

Unidentified speaker: perfect. Thank you. And then the commercial launch, how far is that commercial launch? Will it take before you kind of include that into the guidance? Or how do you think through that process?

Mike, Exelixis: Yes. We haven’t given a specific time or date. We’re going to look at it. We’re going to see where we are, where we projected to be on the launch timeline and see where that is. And then we feel comfortable we can project better the future better, then we’ll provide guidance at that point in time.

But we’re not giving guidance on guidance yet. So when we get to that point in time, we’ll let you guys know. But it’s something that we’re very we understand very well that the street generally wants us to provide guidance, but we’ll do that one like we have in the past when we’re comfortable being able to predict the future with some obviously error bars around it. But yes.

Unidentified speaker: So it’s kind of a matter of taking your internal projections, see how they play out over the quarter? Yes.

Mike, Exelixis: And just seeing the dynamics in the market, like how is it being utilized? Where is it being utilized? Where do we see opportunity? And then trying to translate those data into a projection that we feel comfortable we can meet.

Unidentified speaker: The comment you made about adding to the sales force ahead of that launch, It’s that the classic pattern and do you kind of add is it 50% before, 50% after? How do you kind

Andrew Peters, SVP of Strategy, Exelixis: of think about that? It was

Mike, Exelixis: a small number. It was literally like a handful of sales reps. So it wasn’t that many. So we felt confident one, in the data in our filing, but two, in our ability to utilize those sales reps in other indications in the meantime. And then we’ve added them and so we’re ready to go literally today.

Andrew Peters, SVP of Strategy, Exelixis: Got you.

Unidentified speaker: Okay. Perfect. How do you kind of square away the market opportunity for peanut?

Mike, Exelixis: I think the way we talk about it, and we talked about it on the third quarter call, contemporary pricing, we think it’s like a $1,000,000,000 opportunity from a market perspective. We haven’t given any guidance on what we think that our proportion of that is, but obviously we think CABO is a very good alternative for physicians and we’re going to do everything appropriately to get it in patient’s hands so that they benefit from that.

Unidentified speaker: And then just as we think about prostate, where are you for the NDA submission?

Andrew Peters, SVP of Strategy, Exelixis: Yes. So kind of as Mike has talked about, so when we were getting ready to file or going through the regulatory conversations with NET, obviously, we heard about the ADCOM and kind of that became an all hands on deck opportunity given the prioritization and the opportunity that we see for NET. And so we essentially put the prostate filing on the back burner and said, let’s devote all of our time, focus, energy, etcetera, and getting kind of nets across the finish line. And then now the adcom has been canceled. We’re waiting on the PDUFA and kind of once that is over the finish line, we’re going to refocus again on prostates.

We’ll give an update there later this year.

Unidentified speaker: Okay. So no adding of sales force for prostate yet. And then, so as we think about your next generation molecule, ZENZA, I guess STELLAR-one hundred and one is forefront of my mind at least at the moment. And CRC, what are we going to learn from the additional data that we see in the first half of twenty twenty five?

Andrew Peters, SVP of Strategy, Exelixis: Yes. So that was the data set that was presented at the ASCO GI conference earlier this year. And that was really a study that we embarked on to really flush out kind of the contribution of components from a regulatory perspective, given this is the first pivotal study of Zanza. We really wanted to understand contribution of components. So that trial, that randomized study that we presented there was looking at the combination of ZANZZA ATEZZA versus ZANZZA alone to really understand what those dynamics there.

And the data showed really across all of the core efficacy endpoints, response rate PFS and overall survival that the doublet performs better than Zansa monotherapy. And so we looked at that data and kind of were enthusiastic about kind of that contribution of components angle. And then one of the obvious questions that we got in response to it was really to understand, well, how does this make you feel about three zero three? And I’m sure we’ll get to later, but understanding how that, XANZA ATEZO compares to what would be expected with rego and with OS in the eighteen to twenty month range at ASCO GI in that non Liberumet segment, we think that compares all the caveats with cross track comparisons, etcetera, compares favorably with that kind of twelve, twelve point five month expectation for rego that we would expect to see based on some of the RCAD data. Okay.

Unidentified speaker: And do we get an update of STELLAR-one in the first half of that?

Andrew Peters, SVP of Strategy, Exelixis: Yes. So beyond kind of what was published at ASCO GI, don’t want to say when that data is going to be presented next. I think kind of really in that CRC segment, the next big step is going to be pivotal data later this year from 03/2003. Got you.

Unidentified speaker: Okay. And then you kind of talk through the rego kind of what you want to see for OS and kind of what’s the feedback you’re getting from physician of just like physicians what they want to see for an improvement?

Andrew Peters, SVP of Strategy, Exelixis: Yes. I mean kind of a somewhat of a statement of the obvious, but one of the things that we talk about a lot internally at Exelixis is we’re in the business of changing the standard of care. And so what we want to see, what we think physicians want to see is a data set that is compelling enough that has an opportunity to show that patients hopefully live longer on the combination of Xantaytazone versus rigrafinibenolone. Obviously, a large study, we haven’t talked about kind of the specifics around the SAP, but we think the study is both designed to show statistically significant and clinically meaningful advantage again to hopefully establish a new standard of care, so we can go out and help patients. Just as a reminder, the amendment that we made to the study a couple of years ago now, really looks at first that delineation between the LiberMet and the non LiberMet population, which is kind of this emerging continuing to emerge dynamic in colorectal cancer where you really see this big distinction in terms of how those two patient groups perform, not only on kind of sensitivity around checkpoint inhibitors, for example, but even kind of the ARCAD data for rego show kind of clear differences there.

So that’s something that we’re keeping in mind and why it was a kind of key feature of the data that we presented at GI.

Unidentified speaker: Okay. What’s the path for success you think in non livermit kind of just that for OS?

Andrew Peters, SVP of Strategy, Exelixis: Yes. So again, coming back to the RCAD data. So RCAD is a French group that has a robust database of virtually every clinical trial that’s been run for rego and LONSRF and those active agents that are used in colorectal cancer. And their data would show that in that kind of non liver met population, it’s probably around 12, 12 point five months. And then ITT probably closer to nine, ten ish months, maybe nine, ten, eleven.

And then in kind of that liver met segment, it’s a little bit lower kind of that six, seven, eight months or so. Okay.

Unidentified speaker: How should we think about dividing that group into like the non liver mets versus the broader intent to treat what’s the cut? Like the market opportunity?

Andrew Peters, SVP of Strategy, Exelixis: Yes. So I think that’s kind of one of the interesting dynamics is from a TAM perspective, market opportunity perspective. We really think that they’re about the same, even though the non liver met patient group is about thirty percent of patients overall, given that those patients tend to live a little bit longer, have longer duration, as you think about kind of the smaller number of patients, but potentially longer duration, they’re about equal in size actually. And so obviously, we’re hoping to show a strong signal in the ITT group, but non liver mets we think is a sizable opportunity on itself.

Unidentified speaker: Got you. Thank you. And then STELLAR-two and kind of what we should expect from it because I believe there’s additional data they’re rolling out in the first half, kind of what should we expect to see?

Andrew Peters, SVP of Strategy, Exelixis: Yes. So we haven’t kind of given the what, when, where of it all, kind of per our practice where we wait for abstract titles to be presented to kind of make sure that everything is everything’s all locked in place. We’ve guided, we’ve commented that there’s going to be updates from SELR-two this year and kind of stay tuned on the specifics, but it’s going to give another more mature look at how the emerging Zanza profile is looking, kind of all the caveats with small and kind of early studies aside. What we always joke about internally is the cosmic truth of all of this is ultimately going to come out in these large randomized pivotal studies. And the good news there is what we said is we’re expecting data from both 03/2004 to potentially happen this year.

And then the go, no go on the Phase two, three, four, three zero five also this year. So it’s going to be a very kind of data rich year for XANZZA, not only kind of with these more early updates on kind of the Phase onetwo programs, but seeing real pivotal data is exciting.

Unidentified speaker: Yes. It’s a I know they’re different indications in a sense, but in RCC, is there a nice read through from ’2 over to 03/2004?

Andrew Peters, SVP of Strategy, Exelixis: I think understanding kind of the similarities and differences between clear cell and non clear cell, RCC can be challenging sometimes, but obviously, it’s a sensitive tumor type. CABO is used there. All RCC approved drugs are used in that non clear cell space. So don’t want to get ahead of, again, of specifics and kind of when we’ll see what. But certainly, we’re excited to share not only that data, but importantly, that pivotal three zero four readout as well.

Unidentified speaker: Got you. And as we kind of do those cross trial comparisons, what’s the buzz for success as we think about the three zero four readout? What’s the best way of thinking about the patient types, etcetera?

Andrew Peters, SVP of Strategy, Exelixis: Yes. I mean, ultimately it’s as I was saying before, we’re in the business of P values and we’re in the business of shifting standards of care. So the one kind of interesting thing or important thing to consider about three zero four is that while all of the existing RCC drugs have kind of on label approval for the non clear cell indication, there’s actually been no pivotal studies run-in that space. They tend to be based single arm unrandomized data. And so what three zero four does is it provides that opportunity to really plant a flag in the ground with level one evidence and say, this is the only treatment option out there, the only combination out there that has been proven hopefully in a large randomized study against the standard of care to show activity.

So that’s kind of the initial goal around three zero four was to actually provide some real level one evidence around activity and let that kind of inform utilization decisions and all of the kind of the downstream effects from there.

Unidentified speaker: Good. All these are in collaboration with Merck, is there any kind of restriction about how much data you can release or you’ve kind of got a clear strategy around?

Andrew Peters, SVP of Strategy, Exelixis: Yes. So three zero four is actually our study. The two Merck studies, we’ll announce details on later this year. But just as a reminder for the audience, we announced in October a collaboration with Merck who one of the things I like about it is Merck’s our biggest competitor in RCC. So anytime you can work with your biggest competitor, it tends to be a good thing.

But we’re running or Merck is running two studies in RCC evaluating Zanza and belzutifan, their HIF2 inhibitor. And kind of the important thing in one of the dynamics that we’ve certainly done in the past with the 9ER study that we’re continuing is this clinical collaboration model where Merck’s running the studies, Merck’s paying for one of the studies and we’re paying for half of the other study. And that gives us kind of a pretty nice attractive bang for the buck, so to speak, on the expense side, as Chris can talk about, where coming back to the 9ER study, Bristol paid for half, our partners Ipsen, Takeda paid for half of our half. And so we took a $0.25 on the dollar investment to really more than double top line revenue. So that’s a model that we liked and learned a lot from 9ER and we’re hoping to continue that with Merck and that was kind of opening the gate, so to speak, for other clinical collaborations with potential partners to come up as well.

Yes.

Mike, Exelixis: I mean, it’s a great way to leverage both of our P and Ls and de risk it to some degree. They’re also providing free pembro for three zero five. So that’s another benefit to the P and L that allows us to continue to think about how we manage R and D expense. Mike has said, we’ll keep R and D expense in that $1,000,000,000 range. And so we’re below that this year and we’ll continue to strive towards that around $1,000,000,000 number over the next several years.

Unidentified speaker: And then I guess three or five head neck, how should we think about that for just like the data disclosure within the second half? Is that

Andrew Peters, SVP of Strategy, Exelixis: Yes. So that one’s pretty straightforward. It’s a Phase II, III trial design and so that includes a go, no go decision, Phase II, III gate, so to speak. Given the design of the study, it’s really going to be a yes or no, will the study continue? Because we actually won’t see that data.

Because if we did and we were unblinded to it, we wouldn’t be able to use that for the Phase three portion. And so the decision we’ll get later this year, the data we’ll get later this year is really, will the study continue to the Phase III portion or not. We haven’t talked about what exactly the parameters that are behind kind of that gate, but from a just disclosure perspective, it’s will continue or not. Got you. Okay, perfect.

Unidentified speaker: Thank you. And then the USP1 inhibitor, kind of where are you for the monotherapy and combinations that is in data?

Andrew Peters, SVP of Strategy, Exelixis: Yes. So that’s a program that I kind of think of as like next on deck, so to speak, after XANZZA. It’s a target, it’s a mechanism that we’re particularly excited about just given the complementary nature of the USP1 pathway and kind of DNA damage repair and to the PARP space, really the opportunity there is, can adding a USP1 either as a monotherapy or in combination with the PARP deeper in responses, can it broaden responses? And can we grow upon that 3,500,000,000 or so PARP inhibitor market right now. And so we’re continuing to escalate not only in the monotherapy, but in combination.

And so so if we want to generate data as quickly as possible to make a decision on that program, because we’re in the business of making smart, prudent investment, as Chris talked about kind of P and L management. So we want to be able to decide as soon as we can, is this something we’re going to invest in and really run or is it not going to hit the bar for differentiation and we’re going to shut it down. So those cohorts are continuing to enroll.

Unidentified speaker: Yes. Because there’s at least been one company that’s pulled out the USP1 space. What do you want to solve for? What’s the differentiation? Yes.

Andrew Peters, SVP of Strategy, Exelixis: I mean, from a pure structure perspective and Dana, our CSO talked about that a little bit at our R and D Day in 2023. It’s an instance where we went from kind of third in class, but with what we thought was a best in class asset to not only first in class, but likely best in class as well as some of the other molecules either fell off from tox or due to kind of drug like properties, solubility issues, dosing issues. And so, it’s one where we kind of found ourselves all of a sudden in the lead with what we think of is a differentiated molecule. So we’re excited to generate that data.

Unidentified speaker: Perfect. That’s a problem there. So thank you so much.

Mike, Exelixis: Thank you. Thank you. Thanks again for having us.

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