Syndax Pharmaceuticals, Inc. H.C. Wainwright 28th Annual Global Investment Conference

Yeah. First, I want to thank Andres and H.C. Wainwright for having us here. It's been a great conference yesterday and today. I feel like this is a really great time for Syndax, to use a motor analogy, really hitting on all cylinders. We are a small company, but we have two commercial assets, both in their second year of launch. The first is Revuforj. We own global rights, long IP out to 2040-ish. Right now, we have two indications that we sell the drug for, KMT2A-r acute leukemia, that includes AML, ALL, pediatrics and adults, as well as NPM1 mutated AML, also adults and pediatrics. Promote that with our own field force and annualizing well over $200 million, just six full quarters into our launch. Second drug is axatilimab, known commercially as Niktimvo. We co-promote that with our partners at Incyte.

They report net revenue, we report collaboration revenue. That drug is indicated for third-line plus chronic cGVHD. Also annualizing well over $200 million, actually closer to $240 million. Both these drugs, which I'm sure we'll get into, both these drugs have extensive life cycle management programs in place, with actually a couple near-term readouts coming out here in the fourth quarter for axatilimab, two phase III trials, which are really important to us. Beyond that, we have a pipeline of two preclinical assets that we rolled out at an R&D day earlier this summer, which I'm sure we'll talk about as well, SNDX-4321, an allosteric EGFR for non-small cell lung cancer, as well as SNDX-62122, which we are developing for MF. Exciting times at Syndax, for sure.

You want to comment on the treatment journey?

Yeah. Firstly, thank you for having us. It's a pleasure to be here and appreciate the opportunity to talk about our programs at Syndax. It's an exciting time in the treatment of AML. This has always been a difficult area of drug development. We're making real progress now, and I would say really transforming the paradigm and the treatment of AML. And we're proud to be leading the field of menin inhibition, driving the scientific leadership and to be the first menin inhibitor approved. And as we're seeing that evolution in the treatment of AML, it's exciting to see treatment practice evolving alongside it. Obviously, our approval is in relapsed/refractory AML covering NPM1, KMT2A. Also very proud of our indication that includes pediatrics, also ALL.

What we're learning now in clinical practice, and we've been the first to publish a series of real-world evidence, is actually sort of augmenting what we actually see in the label, just as a function of clinical practice and what clinicians are seeing. The first observation is that increasingly, patients get treated earlier in their treatment journey. I think that's important because once they've progressed on their first-line therapy, you really want to come with the next best available therapy to them, and people see that as Revuforj. That's number one. Number two is increasingly we see patients getting treated in combination. Again, our current indication doesn't cover that, but we have published compelling data now in combination with readily available standards of care, which significantly improves the response rates for these patients.

In the real-world series that we have presented from Moffitt Cancer Center and other leading cancer centers across the U.S., we do see up to 80% of patients potentially being treated in combination. One of the impacts of that is we see increasing numbers of patients actually going on to get stem cell transplant. That's really important for patients because that gives them the best possible chance of a long-term remission and a favorable outcome. Potentially even some patients, maybe we're even talking about cure, and that would be very exciting. The last piece in the treatment journey that is very important for patients and a big driver for uptake of Revuforj is the use of Revuforj after patients have had a transplant.

We're seeing now nearly 50% of patients going on to get a transplant, which is very different from what we experienced in our original pivotal study AUGMENT-101. Increasingly, we're seeing patients going back onto therapy after they've had a transplant. Up to 70% of patients are offered and will then receive Revuforj as a post-transplant maintenance, which gives them, we believe, the best possible chance of a durable remission. I think those are the big drivers of clinical practice and clinical uptake.

It's a good question. I think clearly The biggest driver of revenue longer term is going to be that stacking effect you see from patients coming back on revumenib.

Let's just take KMT2A-rearranged acute leukemia, for an example. The incident population is about 2,000 patients per year in the U.S. So new patient starts are always going to be important to add to the top of the funnel. But over time, as Nick mentioned, we're getting about 50% of those patients to transplant, and we're seeing already 50% of those patients who go to transplant coming back on Revuforj maintenance therapy, and they could be back on for six months. They could be back on for two years.

That recurring revenue piece from that stacking effect, it's already making up the majority of the revenue in any given period, whether it's a week, a month, or a quarter today, and it's only going to continue to build.