Overview
The FDA granted a second approval to Syndax’s menin inhibitor Revumenib, expanding its use to a larger genetic subset of acute myeloid leukemia and marking a rare back-to-back win nearly 20 years after the science first emerged.
Two decades of painstaking leukemia research have suddenly yielded one of the fastest regulatory streaks in recent memory. In less than a year, the same targeted pill for acute myeloid leukemia (AML) earned two FDA approvals—an outcome made possible by an unusually tight partnership between academic researchers and a biotech developer, and by a clear genetic roadmap that allowed regulators to move quickly.
The FDA’s second clearance for the rare cancer therapy came in October, capping an unusually fast stretch for the new medicine. Within a year, the same oral drug had won back-to-back approvals for two genetically defined groups of patients with acute myeloid leukemia who have few good options once their disease comes back.
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The Collaboration Behind the Success
The speed on the regulatory side sits on top of a long, methodical arc in the lab and a well-functioning partnership between academic researchers and an industry sponsor, according to Scott Armstrong, MD, PhD, senior vice president for Drug Discovery and Chief Research Strategy Officer at Dana-Farber Cancer Institute. His organization and Boston Children’s Hospital helped lay the groundwork for the drug, Revumenib, that was then developed by Syndax Pharmaceuticals.
“There was very close collaboration between the academic lab and the biotech, with the lab doing a lot of the early discovery right alongside the company doing most of the chemistry,” Armstrong told Cure. “We were both doing what we were best at, and it was a terrific collaboration.”
A Targeted Approach to AML
Revumenib, or Revuforj as it is commercially known, is a menin inhibitor taken as a pill. The FDA first cleared it in late 2024 for a small group of adults and children whose acute leukemia carries a specific genetic signature and has come back or failed to respond to other treatments. Regulators expanded its use to a second genetic group that makes up a much larger share of patients with AML.
AML is diagnosed in more than 20,000 people in the United States each year, and only about 30 percent are alive five years after diagnosis. The mutation targeted in the new approval is the most common genetic change seen in adults with the disease, present in up to a third of patients. Combined with the earlier indication, the medicine can now be an option for as many as 40 percent of people with the condition, according to Dana-Farber.
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A Decades-Long Scientific Arc
That long arc began almost 20 years ago in Armstrong’s lab, where he and colleagues had been trying to understand how certain genetic changes drive the spread of leukemia. In 2006, his group published work mapping a specific pattern of misfiring genes in a rare form of the disease, then later found the same pattern in a more common subtype—suggesting the same machinery might be driving both. That insight turned what looked like a niche problem into a target that might matter for close to half of patients with this cancer.
Once that biology was clear, Syndax entered the picture. In the mid-2010s, Armstrong’s team began working with the company to hunt for small molecules that could block menin, a protein that helps keep the leukemia program switched on. His lab tested candidate compounds in leukemia cells and animal models, looking for signs that the abnormal gene activity was shutting down, while Syndax focused on refining the chemistry into a drug that could be given to people.
Why the Academic–Biotech Model Succeeded
Armstrong said the partnership worked well because each team understood its strengths. The academic group stayed focused on the same target for decades but could hand off a mature concept to a biotech partner with the capital and infrastructure to move quickly into multi-center trials.
“Given the right set of circumstances, if you can arrange it such that academic labs are doing what they're really good at, and biopharma is doing what it's really good at, it can be quite productive,” Armstrong said. “And allowing the groups to get out of the relationship what they need—the academics get to publish the papers, and biopharma gets to move their drugs into clinical development.”
A clear biological pathway and a well-defined genetic marker made it possible to start with a very small, high-risk population, demonstrate benefit, and then expand into a larger group.
“This is great news for patients,” said Lore Gruenbaum, PhD, Chief Scientific Officer of advocacy group Blood Cancer United, in a news release. “We need to keep working to find treatments for all types of AML.”
