Recursion Pharmaceuticals Inc. (Nasdaq: RXRX) recently announced promising preliminary results from its ongoing Phase 1b/2 TUPELO trial of REC-4881, an investigational MEK1/2 inhibitor being developed for Familial Adenomatous Polyposis (FAP). This rare genetic disorder, caused by mutations in the APC gene, leads to the growth of numerous gastrointestinal polyps and poses a near 100% risk of colorectal cancer if untreated. The study revealed a median 43% reduction in polyp burden among six patients after 13 weeks, with 83% experiencing reductions between 31% and 82%. Additionally, half of the patients showed an improvement in Spigelman stage, indicating reduced upper GI disease severity. The safety profile of REC-4881 was generally consistent with previous MEK1/2 inhibitors, mostly resulting in mild to moderate adverse events. These encouraging results provide hope for the 50,000 individuals affected by FAP in the US and EU5, who currently have no FDA-approved treatments.
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