Watch Alliance to Cure Cavernous Malformation CEO Connie Lee’s video announcement here.
Exactly a year ago, many of you watched with heartbreak as Recursion Pharmaceuticals discontinued its REC-994 Phase 2 clinical trial for cavernous malformation. Our community was devastated, and we shared that pain. At the time, it felt like the end of the road for the first drug ever developed specifically for cavernous malformations.
Today, we have important and cautiously optimistic news.
On May 1st, we signed a license agreement with Recursion to transfer development of REC-994 to us. We’re grateful to Recursion for their partnership over many years—and for reaching out to us after discontinuing the trial to ask whether we’d like to carry the work forward.
We’ve been granted a license to the clinical trial results as well as ownership of the remaining drug supply, the biosamples collected during the trial, and the regulatory permissions to use REC-994 in its current form for symptomatic CCM.
This is a daunting and exciting development that moves our organization into an entirely new sphere – direct drug development.
Our goal is to use this gift to better understand who responded to REC-994 and why — and to explore whether there may be a path back to patients.
From 2022 through 2025, 62 Alliance members across 16 sites participated in the REC-994 trial. We’re forever grateful to every one of you. Your impact will now be felt well beyond the formal end of your participation.
Why are we hopeful? In the Phase 2 trial, REC-994 was well-tolerated and safe. Exploratory efficacy trends were observed in Part 1 of the trial over 12 months of treatment, including decreases in lesion volume and improvement in overall function and CCM-associated symptoms. While the trends didn’t continue through the Part 2 long-term extension, that is not nothing. That is a reason to keep going.
Our next step is to dig into the participants’ precious data and biosamples to continue the research to understand REC-994’s effect. We’re also testing REC-994 in combination with other medicines in our CureDriver lab. The information we gain may guide the path back to patients and increase the likelihood of a successful next trial.
We want to be honest with you: the project is challenging, and the timeline isn’t short. But we don’t shy away from hard things, and we are not giving up. No other drug has shown this level of promise in our patients with so few side effects, and we believe it’s worth pursuing.
If we’re successful, our goal is to share the evidence and insights we develop with companies that have the capacity to bring REC-994 back into trials and, ultimately, back to patients.
One year ago, we felt the end of a trial, and it was hard. Now, we are poised to potentially resurrect the promise of a CCM treatment. This opportunity brings us new hope and new possibilities.
Last updated: 5/22/26
