SNDX - Syndax: Biotech To Watch With Regulatory Filings Before End Of 2023

2023-10-13 14:52:22 ET

Summary

• Syndax Pharmaceuticals, Inc. reported positive results from its phase 1/2 trial using revumenib to treat relapsed/refractory leukemia patients.
• The trial met its primary endpoint with a complete remission rate of 23%, significantly higher than the current standard of care.
• It is expected that an NDA of revumenib for AML is expected before end of 2023; it is also being evaluated in other patient populations which could expand its potential.
• A Biologics Licensing Application of axatilimab for the treatment of patients with cGvHD is expected to be filed before the end of 2023.

Syndax Pharmaceuticals, Inc. ( SNDX ) has been making great progress in being able to advance its pipeline. That's because it was able to report positive results from its phase 1/2 AUGMENT-101 trial, which used its drug revumenib for the treatment of relapsed/refractory KMT2A-rearranged [KMT2Ar] acute myeloid leukemia [AML] and acute lymphoid leukemia [ALL] patients. With this positive data on hand, the biotech will be able to file its New Drug Application [NDA] to the FDA of revumenib to treat this patient population. As I will explain below, the trial was stopped early due to the efficacy that was achieved. Not only that, but another item to note is that there is potential for an expansion opportunity as well.

That's because it is also evaluating the use of revumenib for the treatment of patients with relapsed/refractory NPM1 mutant AML as well. The reason why I state that is because it is nearing enrollment completion of the cohort which is targeting this other AML patient population. Matter of fact, it expects to complete enrollment of this specific cohort by the end of 2023. Thus, eventually Syndax will be able to release results from this cohort and possibly expand upon the possible patient population that it could treat.

There is another major catalyst which is also expected to be released by the end of 2023, which is that Syndax and its partner Incyte intend to release a Biologics Licensing Application [BLA] of axatilimab for the treatment of chronic graft-versus-host disease [cGvHD] patients.

Positive Results From Phase 1/2 Study Using Revumenib Sets Up Major Year-End 2023 Catalyst

As I noted above in the beginning, Syndax Pharmaceuticals was able to report positive results from its phase 1/2 AUGMENT-101 study , which used revumenib for the treatment of patients with relapsed/refractory KMT2A-rearranged [KMT2Ar] acute myeloid leukemia [AML] and acute lymphoid leukemia [ALL]. A total of 94 acute leukemia patients were recruited into this pivotal trial with a cutoff date of July 2023. However, it was note that a total of 57 had been confirmed to have KMT2Ar status and were evaluable for efficacy. These were very sick patients, because many of them had already gone through several other types of treatments. The following percentages were noted for patients prior to entering this phase 1/2 study:

• 56% of patients relapsed following treatment with one salvage regimen
• 46% had undergone stem cell transplantation
• 72% were previously treated with venetoclax.

As you can see, these were very sick patients. It was noted that the trial met its primary endpoint at this interim analysis with a complete remission [CR] or a CR with a partial hematological recovery [CRh] rate of 23%. This difference that was observed was statistically significant with a p-value of p=0.0036. Besides the fact that positive results were announced, there is something else very important to consider here. That is, there are no approved treatments for relapsed/refractory KMT2Ar acute leukemias. Even worse than that, the response rate to current standard of care [SOC] treatment is less than 10% as well. Thus, even a most improvement of response to 23% is still far better than what current SOC offers.

The achievement of positive results from this phase 1/2 study sets up a huge catalyst opportunity for this particular program. It is expected that Syndax Pharmaceuticals will be able to file a New Drug Application [NDA] of revumenib for the treatment of relapsed/refractory KMT2Ar acute leukemia patients before the end of 2023. There is even an opportunity to expand upon the possible patient population that can be targeted upon. What do I mean by that? Well, this biotech is nearing completion of enrolling relapsed/refractory NPM1 mutant AML patients. It expects to complete enrollment of this specific patient population before the end of 2023. Thus, it's quite possible that it could release results from this cohort at some point in 2024.

Financials

According to the 10-Q SEC Filing , Syndax Pharmaceuticals had cash, cash equivalents, and short- and long-term investments of $418.3 million as of June 30, 2023. It believes that it has enough cash on hand to fund its operations for at least the next 12 months. Thus, it is likely going to have to start looking for ways to raise cash.

The good news is that it has an ATM in place, just in case it needs to raise additional funds. Specifically, in May of 2023 it entered into a new sales agreement with TD Cowen under which time it could sell shares of its common stock with an aggregate sales proceeds of up to $200 million. For the 3 months ending June 30th 2023, it did not sell any shares under this agreement. With that said, it has $200 million available under the 2023 ATM program as of July 27th 2023 that it can tap into if it needs to.

Risks To Business

There are several risks that investors should be aware of before investing in this biotech. The first risk to consider would be with respect to the possible NDA filing of revumenib for the treatment of patients with relapsed/refractory KMT2Ar AML. That's because this company expects to file an NDA of this drug for the treatment of this patient population before the end of 2023. Even though the submission is expected to happen, there is no assurance that the FDA will accept the application. On top of that, there is no way of knowing whether or not this drug will be approved for this KMT2Ar AML patient population.

A second risk to consider would be the expected Biologics Licensing Application [BLA] filing of axatilimab for the treatment of patients with chronic graft-versus-host disease [cGvHD]. The filing is being done together with Syndax's partner Incyte, so that both can obtain regulatory approval for this specific indication. The risk here is that the company notes that it won't be able to file this BLA unless it aligns with agreement from regulatory authorities. Thus, if it doesn't accept a green light from the FDA to file the BLA, then it won't be able to do so.

The third and final risk to consider would be the financial position that Syndax Pharmaceuticals, Inc. is in. That's because it believes that it only has 12 months of cash to fund its operations. This means that there is a risk that it might have to either use its ATM agreement or find another way to raise cash.

Conclusion

The final conclusion is that Syndax Pharmaceuticals is a good speculative biotech play to look into. That's because it expects to file two regulatory applications before the end of 2023. One regulatory application will be with revumenib for the treatment of relapsed/refractory KMT2Ar AML and then the other regulatory application will be with axatilimab for the treatment of patients with cGvHD. It remains to be seen if the FDA ultimately approves either of these drugs for the treatment of either of these patient populations, but at least Syndax Pharmaceuticals, Inc. has two shots on goal when it comes to possibly obtaining FDA approval.

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