Syndax Pharmaceuticals, Inc. Q2 FY2025 earnings call

• Revuforj has seen strong commercial and pipeline execution, with over 500 patients treated since launch and expected expansion into frontline settings. The sNDA for relapsed/refractory mutant NPM1 AML has a PDUFA date of October 25, 2025.
• Niktimvo had a successful first full quarter with sales ramping up, and is positioned for growth in the chronic GVHD market with ongoing development for other indications like IPF.
• Ongoing R&D includes trials for Revuforj in frontline settings and Niktimvo in earlier GVHD lines and IPF. The company has a strong leadership team and excellent market access for Revuforj.

Revuforj: Second quarter 2025 net revenue was $28.6 million, a 43% quarter-over-quarter increase. It's the first and only FDA-approved treatment for relapsed or refractory acute leukemia with a KMT2A translocation, with strong uptake and growing contributions. Niktimvo: Incyte reported $36.2 million in net revenue for the second quarter, and Syndax's collaboration revenue from Niktimvo was $9.4 million. Niktimvo is already a positive cash flow contributor and positioned for growth in the chronic GVHD market.

• Operating expenses are expected to remain stable over the next few years.
• Anticipated approval of Revuforj's sNDA in October 2025, which would expand the addressable market.
• Niktimvo is expected to continue growing with sales ramping and long-term patient use.

• Uncertainties related to FDA approval timelines and regulatory feedback.
• Market competition and potential challenges in achieving full market penetration.

Q: Just wanted to ask a question about this path to profitability. Keith, I know you mentioned operating expenses staying stable over the next couple of years. But what are you assuming in terms of the top line, in terms of treatment settings for Revuforj and Niktimvo? Can you get to profitability on sort of the refractory settings alone? Is there some sort of assumptions on frontline expansion baked into getting to profitability? How should we be thinking about that?

A: Anupam, thanks for the question. We have been pretty consistent since November that we expect to get to profitability with the existing resources that we have today. And I would say the only thing that's changed since then is that we have 2 launches that are both outperforming our expectations. So the new disclosure that we provided today, stable operating expenses for the foreseeable future, say, the next 2 to 3 years, we're doing that because we want to give the buy-side and sell-side, we want to give the street the appropriate data for them to model our business better, so you guys can get to the same answer that we're getting to. We're not giving revenue guidance per se, Anupam, but I will say that given the time lines to get to approval in the frontline setting, you can definitely assume that we are getting to profitability on the relapsed/refractory indications alone. And I think, I just want to add, the guidance that we are giving, stable operating expenses, is not to be taken as we are taking our foot off the gas pedal, because we're not. The modeling that we've done allow us to fully invest in the continuing successful launches of 2 products, executing commercially, but additionally, executing our integrated clinical development plan, as Nick talked about, both for Revuforj and Niktimvo. So, I think we're in a pretty unique position to control our own destiny. We have 2 launches that are both outperforming and a stable expense base. And the team has worked extremely hard to put ourselves in this position to specifically reward our shareholders for their investment. So, thanks for the question.

Q: This is Kevin Strang on for Corinne. I had a quick question on the patients moving on to transplant. After how many cycles is that typically occurring? And for patients that are going back on drug, you said that it was about 1/3 of patients so far. What are your expectations for the ultimate proportion of patients that will move on to maintenance therapy? And is this something you'll report quarterly?

A: Kevin, thanks for your questions. So, look, I think we're very encouraged by what we're seeing with patients going to transplant. As we had noted early on when we first started developing this drug, patients do respond very quickly to revumenib, and that's usually within the first few cycles. That generally is preceded by a transplant thereafter. And that transplant can happen very quickly. It can happen within a couple of weeks. That's usually for KMT2A patients, a goal to get them to transplant as quickly as possible. So, a few cycles, certainly 2 to 3, getting into remission, moving to transplant. That's how it generally works. We do see in our trial about 1/3 -- in our commercial experience, rather, about 1/3 of patients getting to transplant. We expect that number to accelerate. And the reason for that is we are treating patients earlier and earlier in the treatment paradigm. Physicians had told us they would put patients on Revuforj in sort of second line or first relapse. We see the vast majority of our patients being treated now in second and third line, which is a great outcome for them, generally means patients do stay on drug longer, do better and have a better chance of going to transplant. So we do think that, that 1/3 number could go up from here, and we expect it too. And ultimately, we will continue to track this over time. It's early days in the launch, and we do expect to report on that metric at some point going forward. And then in terms of maintenance, I think your last question, patients are coming back on maintenance. We know that and we reported that based on the earliest patients -- earliest cohort of patients that we've seen. About 1/3 of the patients have already come back. Again, early days of launch, that's a very good indicator. And physicians have told us repeatedly that they expect to put the vast majority of their patients back on maintenance, and that could range anywhere -- 70%, 80%, 90% of the patients, assuming that they're eligible for maintenance. And so that's, I think, a goal for us, and we'll see that play out over time.