Recent Phase 3 data show oral small molecules succeeding in complex indications. Roche's phenobrutinib met its endpoint in progressive multiple sclerosis, and Bridge Biopharma's Imfegratinib improved height velocity in achondroplasia. This signals a potential shift toward more convenient, patient-friendly oral therapies in areas historically reliant on injectables.
The FDA issued Complete Response Letters for both REGENXBIO's gene therapy and DISC Medicine's oral drug, signaling high scrutiny for accelerated approvals. The agency specifically cited concerns over the relevance of surrogate endpoints and required more robust clinical trial data, highlighting the risks of relying on non-traditional approval pathways.
Within one week, Eli Lilly executed two massive deals: an $8.5B potential collaboration with Innovent for antibody therapeutics and a $2.4B acquisition of Orna Therapeutics for its circular RNA CAR-T platform. This signals an aggressive, multi-pronged strategy to dominate both established and next-generation therapeutic modalities.