Verastem, Inc. (VSTM) Q3 2025 Earnings Report, Transcript and Summary

Good morning, and welcome to Verastem Oncology's Third Quarter 2025 Earnings Conference Call. My name is Liz, and I'll be your call operator for today. Please note, this event is being recorded. [Operator Instructions] I will now turn the call over to Julissa Viana, Vice President of Corporate Communications, Investor Relations and Patient Advocacy at Verastem Oncology. Please go ahead.

Thank you, operator. Welcome, everyone, and thank you for joining us today to discuss Verastem's Third Quarter 2025 Financial Results and recent business updates. This morning, we issued a press release detailing our financial results for the quarter and year-to-date. This release, along with the slide presentation that we will reference during our call today, are available on our website. Before we begin, I would like to remind you that any statements made during this call are not historical and are considered to be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these statements as a result of various important factors, including those discussed in the Risk Factors section in the company's most recent annual report on Form 10-K filed with the SEC on March 20, 2025, and the current report on Form 10-Q that will be filed later today as well as other reports filed with the SEC. Any forward-looking statements we make represent Verastem's views as of today, and we disclaim any obligations or responsibility to update. Joining me on today's call are Dan Paterson, President and Chief Executive Officer of Verastem, who will provide opening remarks and recap key highlights from the quarter. Matt Ros, Chief Operating Officer; and Mike Crowther, Chief Commercial Officer, who will walk through the continued progress of the AVMAPKI FAKZYNJA CO-PACK commercial launch; and Dan Calkins, Chief Financial Officer, who will provide an overview of our financial results. I will now turn the call over to Dan.

Thank you, Julissa. Good morning, and thank you for joining us today to discuss our third quarter financial results and business update. In Q2, we shared our excitement about achieving FDA approval for AVMAPKI FAKZYNJA CO-PACK in KRAS-mutated recurrent low-grade serous ovarian cancer, or LGSOC, and reported our first 6 weeks of commercial performance. Today, I'm pleased to share that the strength we saw in those initial weeks has accelerated. With a full quarter of commercial operations now complete, the fundamentals we put in place to guide our commercial launch are translating into meaningful results. Our third quarter net product revenue of $11.2 million surpassed our expectations and was driven by consistent adoption among both academic centers and community oncologists. We set 3 key objectives to support our commercial execution and drive sustainable growth. Physician engagement, patient initiation and retention and streamlined reimbursement, all 3 are trending positively. We're simultaneously advancing our broader strategic priorities, specifically expanding the opportunity set for AVMAPKI FAKZYNJA CO-PACK and accelerating the clinical path of VS-7375, our KRAS G12D (ON/OFF) inhibitor program. Let me highlight a few achievements. We completed the enrollment of our expansion cohort in RAMP-205, our first-line pancreatic cancer trial evaluating avutometinib plus defactinib and standard of care chemotherapy. We completed the planned enrollment of our confirmatory Phase III clinical trial, RAMP-301 in recurrent LGSOC, and we'll be making a modest increase in enrollment per the IDMC's recommendation. This does not have a meaningful impact on our expected time lines. We shared initial safety and tolerability data from our G12D program with VS-7375. We cleared 2 monotherapy doses with no dose-limiting toxicities. We reported that we did not observe nausea, vomiting or diarrhea above Grade 1. Importantly, these dose levels included the Phase II go-forward dose that was chosen by our partner in China. We're now moving ahead of opening the combination cohort with cetuximab. These are exciting developments that add to our continued success. In the fourth quarter, we remain focused on our 3 key launch objectives and maintaining our execution discipline across all commercial, clinical and operational functions. Let me now turn the call over to Matt to review some specific highlights from the third quarter.

Thank you, Dan. The strong AVMAPKI FAKZYNJA CO-PACK growth reflects the high unmet medical need and physician enthusiasm for this first-ever treatment option. The team has achieved significant results since our May approval, and we continue to execute well against all 3 strategic launch imperatives. Dan touched upon these, and they are: first, to effectively reach health care providers, remembering that the top 100 commercial health care organizations comprise about 50% of the sales opportunity. Second, to engage and support patients throughout their journey as we know that as patients progress through other therapies, many will be ready for a new treatment option. And third, to ensure seamless access so we can support patients and ensure any barriers to reimbursement are removed. Our approach is highly targeted, and we're utilizing a deliberate mix of one-on-one meetings, group discussions and conference engagements to maximize the impact of every interaction in this rare disease market. Thus far, each element of this approach has proven to be successful. As Dan shared, we generated $11.2 million in net product revenue in the third quarter, which was our first full quarter of launch. We've leveraged the momentum from the first 6 weeks of launch and uptake has been strong. With 133 prescribers of that AVMAPKI FAKZYNJA CO-PACK, physician excitement is palpable and our field teams continue to do an excellent job in engaging with health care providers to ensure they understand the unique benefits of the CO-PACK and how to administer. Consistent with Q2, we continue to see prescriptions generated by gynecological oncologists and medical oncologists. This well-rounded base of prescribers reinforces the touch points our teams are making across our top 100 organizations and Tier 1 and Tier 2 targets. We are experiencing high levels of engagement within community practices that are either large affiliated practices or are associated with group purchasing organizations. We are directly contracting with the GPOs and conducting educational programming. We are also having meaningful success in accounts that are typically closed to sales representatives. We continue to engage and support patients with outreach efforts to help educate them about the treatment and support their conversations with their doctors. And while we won't be speaking to future trends or prescriptions at this time, we are encouraged by specific insights following our first full quarter of launch. Approximately 65% of prescriptions written have been generated by our top 100 organizations. What's great about this is that we are making strong headway in our Tier 1 and Tier 2 accounts, but we are also seeing prescriptions coming from other accounts as well. We believe that speaks to the strength of the data and brand awareness. More than half of total prescriptions are coming from the academic setting, and we expect the split to be consistent between the community and academic setting providers over time. 60% of the prescriptions written are coming from GYN oncologists and 40% written from medical oncology. Our specialty distributors are now fully on board, and we see a good mix between the 2 specialty pharmacies onboarded in Q2 and the 4 specialty distributors we added this quarter. The initial orders across our specialty distributors were managed closely and have been consistent with the initial orders from our 2 specialty pharmacies at launch. For these reasons, we believe inventory stocking has been minimized, and we plan to continue to manage this closely through year-end. Lastly, reimbursement has not been a barrier to any access, and Mike will provide more specifics in that regard shortly. Looking at the fourth quarter, we aim to continue to build on our momentum while staying laser-focused on our strategic imperatives to ensure every appropriate patient benefits from this novel treatment. The key opinion leader community continues to reinforce our thesis that every KRAS-mutated LGSOC patient should not only receive this treatment, but should do so at their first recurrence. Given our early achievements, our team's effective execution and the high unmet need in this rare form of ovarian cancer, we believe we are well positioned for continued growth. Now I will turn the call over to Mike to speak further about the launch dynamics. Mike?

Thanks, Matt. Let's get right into the specifics of our AVMAPKI FAKZYNJA CO-PACK launch. I'm extremely pleased with how well the launch is going as net product revenue growth accelerated in the third quarter. While we consider ourselves still in the early days of launch, the underpinning of success is built upon the breadth and reach of our field engagement to raise awareness of the availability of the first-ever treatment, specifically for people living with KRAS-mutated recurrent LGSOC. These impressive results are driven by a few key factors: high unmet need, increased engagement with both academic and community oncology practices, expanding reach and removing barriers to access through specialty distributors and their GPO partners and continued efforts to ensure seamless access. From an engagement standpoint in the third quarter, we have had high engagement among our top 100 organizations and top 100 offices, which includes a mix of academics and community providers. These efforts have resulted in approximately 65% of prescriptions coming from them and specifically within our Tier 1 and Tier 2 accounts. We continue to see both repeat prescriptions from physicians prescribing to multiple patients and refill for patients given the CO-PACK's favorable safety profile. An important insight we have gained is that HCPs treating LGSOC have a good understanding of where their patients are in the treatment journey and are keeping CO-PACK top of mind for when the patient's current therapy fails due to either intolerability or clinical progression. Doctors continue to share that they are actively assessing and identifying patients that may become appropriate candidates for this targeted combination therapy, demonstrating that our efforts with HCPs are creating visibility into new patients becoming available for treatment. Additionally, the awareness about AVMAPKI FAKZYNJA CO-PACK is high. Our medical science liaisons and oncology nurse educators have engaged in 800 scientific exchanges and well over 100 educational forums with health care providers within this quarter alone. We believe payers are acknowledging the unmet need that can now be addressed by the CO-PACK as well as the clinical value of the combination therapy. The payer coverage continues to be broad and the time to fill prescriptions has been fast within approximately 12 to 14 days. We can also confidently share that covered lives has now exceeded 80% and that the payer mix for our combination therapy is about half commercial and half Medicare. From a patient perspective, we continue to see high engagement in our branded website. Our digital campaign is effectively driving traffic to this resource and patients are downloading our patient brochure and opting in to receive more details associated with how the CO-PACK can be appropriate for them. To close, we strongly believe that the AVMAPKI FAKZYNJA CO-PACK combination therapy has the potential to make a significant impact on the lives of patients who previously had no treatment options specific to their disease. With several months now under our belt, the team is executing well against all our launch objectives. We continue to believe a steady adoption will occur over time, and our early observations post approval support this perspective. I look forward to sharing more in the coming quarters as we progress through the launch and gain more experience and insights. With that, I'll turn the call over to Dan Calkins to provide an update on our financials.

Thank you, Mike. We issued a press release before the call today with the full financial results so I'll focus on the highlights for the third quarter. In our first full quarter of launch, I'm also pleased to report $11.2 million of net product revenue for the third quarter. Cost of sales were $1.7 million for the third quarter of 2025 and did not include a significant amount of product costs as inventory produced prior to FDA approval was fully expensed at the time of production. Currently, we're not providing guidance on gross to net other than to say that expectations should be consistent with other oncology small molecule therapeutics. Turning to research and development expenses. They were $29.0 million for the third quarter of 2025. R&D expenses were driven by both the ongoing global confirmatory Phase III RAMP-301 clinical trial and the ongoing BS-7375 Phase I/IIa clinical trial as well as higher costs associated with drug substance production activities related to BS-7375. SG&A expenses were $21.0 million for the third quarter. The expenses were driven by commercial activities and operations, which included personnel-related costs to support the ongoing CO-PACK launch. We continue to be prudent in our expense management, making the right investments at the right time to support the ongoing commercial launch efforts while simultaneously advancing our pipeline. For the third quarter of 2025, non-GAAP adjusted net loss was $39.4 million or $0.54 per share diluted compared to non-GAAP adjusted net loss of $35.3 million or $0.88 per share diluted for the 2024 quarter. Please refer to our press release for a reconciliation of GAAP to non-GAAP measures. Moving to the balance sheet. We ended the third quarter of 2025 with cash, cash equivalents and investments of $137.7 million. We believe our current cash, combined with future revenues from AVMAPKI FAKZYNJA CO-PACK sales and the exercise of the outstanding cash warrants provides runway into the second half of 2026. We had a solid first full quarter as a commercial company. We have sufficient capital to fund our ongoing commercial launch in the U.S. and continue advancing our current clinical development plans. With that, I'll turn the call back over to Dan.

Thanks, Dan. Before we open the call to Q&A, I'll share a few final remarks to close out today's presentation. We've seen another strong quarter of execution at Verastem as we continue to deliver on all our strategic priorities, meeting our key milestones and delivering a strong commercial launch. As we're in the final quarter of 2025 and look to 2026, I want to reaffirm our strong confidence in our growth trajectory and the significant value creation opportunities ahead for our company and shareholders. Commercial execution remains a top priority. The fundamentals are driving AVMAPKI FAKZYNJA CO-PACK adoption and the launch is progressing as planned. Our clinical pipeline continues to advance on multiple fronts. We expect several important data readouts in the first half of 2026 that will further demonstrate the breadth of our RAS/MAPK pathway-driven approach. We expect to share safety and efficacy results from our RAMP-205 expansion cohort in first-line advanced pancreatic cancer in the first half of 2026. We also plan to share initial results from our Phase I/IIa trial evaluating VS-7375 and advanced G12D mutant solid tumors in the first half of 2026. We'll continue to advance our trial of VS-7375 in both monotherapy and combination expansion cohorts in pancreatic, lung and colorectal cancers. Importantly, we believe VS-7375 has demonstrated significant and best-in-class potential among KRAS G12D inhibitors to date in both advanced pancreatic cancer and lung cancer. And we're committed to moving quickly to registration-enabling studies in these and other high potential priority indications. This is an active area of focus for the company, and we plan to engage with the FDA in the first half of 2026 to discuss our path forward. This would include seeking their input on how to harmonize the abundance of existing data generated by our partner in China to advance the program efficiently on behalf of patients who currently have no FDA-approved treatments for their KRAS G12D mutated cancers. We now have a commercial product generating growing revenue and a robust clinical pipeline with multiple near-term catalysts that will determine the future development plans. We are building a sustainable multi-asset oncology company to address important unmet needs in RAS/MAPK Pathway-Driven cancers. With that, we'll open up the call for questions. Operator?

[Operator Instructions] Your first question comes from the line of Michael Schmidt with Guggenheim Securities.

On the LGSOC launch, yes, just wondering if you could provide a few more comments on how the product is being used in the market in terms of patients having had prior lines of therapy. I'm just thinking about some of the market dynamics around incidence of new patients that relapse versus that sort of existing prevalence pool. How is the product being utilized in that context? And what are you seeing in terms of KRAS mutant versus wild-type use?

Yes. I mean we're early in the launch. So you do end up seeing some patients with later lines of therapy, but we're also seeing patients that are first recurrence, and it is a mix of wild-type and mutant as well as some just off-label totally. We don't have exact numbers on that. Again, we don't see total visibility of that through the distribution channel that goes through the distributors as opposed to specialty pharmacy. And we don't always have a good view in the total number of lines of therapy. I don't know, Mike, if you wanted to give a little more color.

Sure, Dan. I mean, consistent with what you've said, we've seen a variety of patients across a range of lines of therapy. We're not always giving the information about what prior therapies they've been on, but obviously, they've seen most of the classical mix of chemotherapy, AI, bevacizumab plus or minus a MEK inhibitor. Since we're promoting just on label, the vast majority of our patients that we've seen so far are KRAS mutant LGSOC.

Great. And then a question on the RAMP-301 study update. Just curious if you could comment on what type of analysis the IDMC did? Was this just looking at event rates and adjusting for event rates? Or did they perhaps look at additional information in terms of effect size? Any comments there would be helpful.

Yes, great question. I mean, to be clear, we're blinded by what the IDMC did. And we had put this interim analysis in place because -- and we've mentioned this before, there wasn't perfect information on the comparators. There weren't prior studies with prospectively broken out KRAS mutant and wild-type. And we tried to keep the sample size as low as possible, but also have the ability to be able to upsize that if needed. I'm optimistic because the number of recommended additional patients was relatively small, about 30. It was across both wild-type and mutant, which, again, I think speaks to them being within the range. And what I was told is because the study accrued faster than we had projected, there were less events than one normally would have had. And I think part of the reason for adding a couple more patients is there just aren't enough events yet really to draw any definitive conclusion, and we want to make sure we're -- we have enough patients to be in the range.

And congrats again on a great quarter.

Your next question comes from the line of Justin Zelin with BTIG.

Congrats on the strong quarter. I wanted to ask about the NCCN committee review in October, if you had heard back on a recommendation for the labels to be expanded to include KRAS wild-type patients? And I have some follow-ups.

Yes, that's a great question. And to be clear, had we heard, we would have told people, we don't know. We know the committee met. We don't know the outcome of that yet.

Got it. Do you have an expectation on any time lines on when you might expect to hear back?

We actually don't. We've heard it can be as long as early next year, could be earlier. I think different committees operate differently. We've not been given a lot of guidance. It's a relatively opaque and what I would say, secret process, and they've all signed NDAs and things. And so as much as I would love to know the outcome of the meeting, we just don't know yet.

Understood. And maybe just one additional question just on the commercial launch. Do you have any color on new patient starts versus patients who are refilling prescriptions as far as contribution to your strong quarter?

Matt, do you want to take that one?

Yes, sure. Great question. We aren't providing that level of detail or specificity on new to Rx refills. However, we are continuing to see significant new prescriptions come in for patients and patients that have started on therapy, particularly in the beginning of the third quarter have continued to receive refills. So we are seeing the dynamic in the marketplace, but providing that level of granularity at this point is a bit too premature for us. We wanted to see another full quarter or 2 underneath our belts before we provide that level of detail.

[Operator Instructions] your next question comes from the line of Sean Lee with H.C. Wainwright.

Congrats on a good quarter. I just have 2 quick ones. First, on the LGSOC market. I was wondering whether you could provide some details on what are you seeing in terms of patient retention. Correct me if I'm wrong, I think on the clinical study, the average treatment duration was about 10 months. So it's still a little bit early for that. Maybe if you can provide some color on the patient dropout rates, has that been in line with what you expect?

Yes. I would say it's really early to tell. And actually, average duration was about 18 months in the clinical trial. I don't know, Matt or Mike, if you want to provide any more color. It is really too early to tell.

Yes. I mean it's a great question. Dan is right. The performance of the CO-PACK in the clinical program, the DOR was around 18 months. We're seeing patients that are coming in at first recurrence. And so we would expect if they're coming in, in an earlier line of treatment that the benefit would be prolonged, but it is still fairly early to provide specific commentary.

I see. My second question is on the VS-7375 study. I was wondering whether there are any significant differences between how you're treating the patients compared to the study that your partners running in China? Because I think I recall that you were discussing some prophylactic antiemetics and such. Are there any notable differences?

Yes. Thanks, Sean. That was a great question. Yes, one of the things that we've said we were doing differently is -- and this was based on experience with the G12C inhibitors being developed and a number of our investigators participated in those studies is really the differences where the patients in China were fasted. This first couple of cohorts we treated in the U.S. were fed. They were also mandated to have prophylactic antiemetics, which is not a part of the protocol in China. And part of the reason we released the information on the first 2 cohorts is, a, we thought it was important that we cleared those first 2 cohorts, which included the recommended Phase II dose in China without any DLTs. But also the early data that we're seeing is that those interventions are making a difference. And as we said earlier, we didn't see any GI toxicities, nausea, vomiting, diarrhea that were greater than grade 1, which we were very happy to see, and we hope that carries forward.

Your next question comes from the line of Yuan Zhi with B. Riley Securities.

Congratulations on the commercial launch. And maybe my first question is for your confirmatory trial, can you remind us what was the enrollment plan for the KRAS mutant patient population and the KRAS wild-type patient population separately?

So the total enrollment was planned for 270, and there were guardrails to set up to keep the amount somewhere between 1/2 and 1/3 KRAS mutant to mirror the population. And so this accrual has come out that way. And as I mentioned, the data monitoring committee recommended that we put a couple more patients on both of those groups. And so we were glad to see, a, that it was a small number of patients that actually could have gone up quite a bit and that it was both arms, which tells us that we're in play with both of them.

Got it. My second question is, what is your next step or priority in the commercial launch? Do you plan to target more prescribers or just make sure a higher number of prescription per doctor?

I would say both. We're not going to change what we're doing. We feel that between our direct calling on individual doctors, the programmatic work we're doing with the organizations and our digital work as well as reaching out to patients that we're covering the waterfront. So we're not planning on changing what we're doing, but it's really a matter of making sure existing prescribers continue to prescribe. New prescribers come on because in any launch, you've got early adopters, mid-adopters, late adopters, and we're working through that chain. And then importantly, that when patients go on, they stay on.

Got it. Maybe before I jump back to the queue, my last question is on the patient's journey. So let's say a patient got their prescription, how long do they have to refill? And how often do they have to visit the doctors to check either symptoms or any side effects? Additional color will be very helpful.

Yes, Mike, do you want to take that one?

Sure. So prescription is for a month supply, 3 weeks out of 4. And in terms of doctors' visits, there is a small amount of visits to begin with just to make sure they're being monitored closely for early toxicities, but that rapidly goes down to every 3 to 6 months.

Your next question comes from the line of Eric Schmidt with Cantor Fitzgerald.

Apologies, I hopped on a little bit late. But with regard to the RAMP-301 IDMC recommendation to moderately upsize the study, can you talk about what the potential outcomes could have been through that look and what data the committee had access to in order to make the decision?

Yes. So the committee had the full data set and the outcomes could have ranged from everything from futility adding, I believe, up to 100 patients to they could have added none. Again, we're blinded to the actual results, but our understanding was there were less events than one would have anticipated given the rapid accrual and that may have led to the small number of patients being added on, but they are being added on to both KRAS wild-type and mutant and it's about 30 across the 2 groups.

So that's helpful. There wasn't any prespecified criteria for adding the 30-ish, 27 patients -- sorry, 29 patients. It was just what the IDMC chose to do, that number?

My understanding is it was within their purview and they made a recommendation to us and we followed it. And again, we don't have full transparency into exactly what they were doing.

And then maybe switching to the 7675, the G12D in your ongoing study. We're very clear that GI tolerability was good in the first dose with no more than grade 1 cases of GI issues. Were there any other side effects to report in that initial cohort? Anything at all of grade 2 or 3?

Well, I believe there were some grade 2 or 3 in very, very small numbers, but nothing -- no signal that we had not expected based on the Chinese data. I think the only thing that was really different was the level of GI tox. And we'll give a more full release of the full efficacy once we've got a few more patients on. I think we've guided early next year, we'll give an update on both efficacy and safety.

Your next question comes from the line of James Molloy with Alliance Global Partners.

I was wondering, could you share any sort of anecdotal updates from the launch, talking to the usage and potential off-label usage on the wild-type versus mutant and sort of any feedback you're getting early stages of the launch? And then I have a couple of other questions as well.

Sure. Mike, Matt, you guys want to give a little more color?

Sure. I mean I think as we shared in our scripted remarks and an earlier question, we're promoting obviously, our labeled indication. So the vast majority of use we've seen thus far has been within the KRAS mutant LGSOC population. That doesn't mean there haven't been wild-type patients because they have, and those have also been seeing coverage through the payers as well thus far.

Okay. Great. Then maybe a follow-up, looks like there's been some M&A in the oncology space recently. You guys are obviously off to an excellent launch here. Any thoughts -- care to discuss any inbound interest you may or may not have from other partners?

I mean obviously, we wouldn't talk about any specifics. But given the launch trajectory to date, and I'd say even more so the excitement around G12D and how the molecules perform both preclinically and clinically. We do get a fair amount of inbound interest and entertain those discussions all the time. We've got some very exciting plans to take these forward, but we're always evaluating could we do more with more resources.

Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.