Recursion Pharmaceuticals announced preliminary results from its Phase 1b/2 TUPELO trial of REC-4881, a MEK1/2 inhibitor for Familial Adenomatous Polyposis (FAP). The study showed a median 43% polyp burden reduction after 13 weeks of treatment in six patients. This is notable as FAP, a rare genetic disorder causing numerous gastrointestinal polyps and a high risk of colorectal cancer, currently lacks FDA-approved treatments.
These preliminary findings offer potential hope for FAP patients, who often face a lifetime of invasive procedures and the constant threat of cancer. A 43% reduction in polyp burden at just 13 weeks suggests REC-4881 could significantly alter disease management and improve patient outcomes. The results compare favorably to other investigational agents, which have reported 20-30% burden reduction over six months in separate studies. This speedier response could translate to a faster improvement in quality of life and potentially a reduced need for surgery.
In the Phase 2 portion, five of six patients saw polyp burden decrease between 31% and 82%, while one experienced a substantial increase. Half of the patients also experienced a clinically meaningful improvement in Spigelman stage, a measure of upper GI disease severity. Safety data from 19 patients across both Phase 1b and 2 revealed that most treatment-related adverse events were mild (Grade 1 or 2). Grade 3 events occurred in 16% of patients, and no Grade 4 or higher events were observed. The most common side effects were acneiform rash, diarrhea, and decreased left ventricular ejection fraction (LVEF), which aligns with known profiles of MEK1/2 inhibitors.
The positive efficacy signal from the TUPELO trial warrants continued investigation. Further data analysis, including a larger patient population and longer-term follow-up, is anticipated in the latter half of 2025. This data will be crucial in confirming the observed efficacy and safety trends, further defining the role of REC-4881 in FAP treatment, and potentially paving the way for regulatory submission. The development of REC-4881 represents a significant step towards addressing the unmet needs of FAP patients and highlights the potential of targeted therapies in this area.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
