Syndax Pharmaceuticals, Inc. Citigroup’s Biopharma Back to School Summit 2026

Well, thanks, Ygal. Good to be with you. Good to be back at school. Look, I think it is a very exciting time for the company and a lot going on. Really 2 important launches. I think what we have done, we have really reset the category in AML in terms of what a launch could look like, which is really exciting. I think people maybe missed that through the trees a little bit. But we are annualizing at about well north of $200 million for its second year of launch, which is fantastic. As you pointed out, we have 2 indications, KMT2A and NPM1, very broad label, best-in-class efficacy. I think the efficacy story is really resonating with physicians. Using the drug in monotherapy, in combination, and really building the Menin story and the leadership that we have exhibited is really coming through.

We are very excited about the forward, excited about what we are building with that franchise. I think NPM1 has become an increasingly important part of our story, both new patient starts and time on therapy. So duration of therapy is an important driving force behind the whole franchise. Recent Medicare data, as well as script data, indicates that we are firmly in the lead in terms of new patient starts for NPM1 as well as KMT2A. So we are really moving well, dominating, we are at 85% market share plus in the category. Thinking about relapse refractory, I do not think you can get more dominant than that, really building the business, and it is growing, right? We have shown 6 quarters, quarter after quarter of growth, substantial double-digit growth, and we expect that to continue.

So that is where we are today, and we are certainly going to continue to build as we get to frontline, which is hopefully right around the corner. We are really moving quickly, enrolling trials, and we will talk about how we are doing. Certainly the data that we have and the upcoming milestones, catalysts we will talk about, but really quite exciting year for us.

Well, I think the numbers speak for themselves.

We posted $55 million in the last quarter, competitor did about 9. You can see that. I do also see or are encouraged by what physicians tell us about the profile and how they are using the drug, using it as monotherapy, using it in combination with standard of care, being supported by the real-world data that we're putting out, as well as what we're producing in trials and so forth. There's a lot of support, and when we speak to physicians, they tell us we're the go-to Menin inhibitor, and we expect that to continue to build as we produce the data that we have set out to bring forward. That's really the sentiment is quite positive.

Right. It's a very important dynamic, as you all know if you've been following our story. Revuforj is an opportunity to bring people to transplant in numbers that we've never seen before in AML with patients who have KMT2A and NPM1. We're seeing transplants across both indications. More transplants, of course, on the KMT2A population, and right now we're at about 50% as of last quarter, 50% of the patients going to transplant, which is remarkable. It's remarkable because it drives the ability to get into a response first, then you get to transplant, then you have the opportunity to go back on maintenance, and maintenance can extend the life of a patient and keep them in remission. That's what we're setting out to do. The target is, we tend to call it the target for maintenance.

It's really what do we think we could achieve? How many patients will go back on maintenance after transplant? We expect the transplant numbers to move up, probably not meaningfully beyond 50% of the patients, but could be higher than 50%. But really the opportunity to bring patients back 70%-80% is roughly the target. We say that because unfortunately, some patients don't make it through transplant, having nothing to do with Revuforj, but just the transplant procedure themselves. Then you have patients who have choice in the matter, and obviously there's some things that intervene and so forth. A high percentage of patients going to maintenance will continue to drive that franchise in terms of TRx, and we see that steadily increasing each quarter, and that's a very positive forward indicator for the growth.