MapLight Therapeutics says it still sees a way forward for its experimental autism drug, even after the treatment fell short in a key clinical trial.
According to Endpoints News, the biotech's drug, known as ML-004, failed to meet the primary goal of its Phase 2 study. In drug development, a Phase 2 trial is a mid-stage test designed to show whether a treatment actually works as intended. Missing the "primary endpoint" — the main result the study was built to measure — is typically a serious setback for a drug program.
Rather than shelve the program, MapLight plans to take its case to the U.S. Food and Drug Administration. Endpoints News reports that the company intends to discuss the drug's future with the FDA, signaling it believes there may still be a viable path despite the disappointing data.
The source material does not detail what ML-004 is meant to treat within autism, the size or design of the trial, or what specific results the company is pointing to as grounds for optimism. Those details, along with the FDA's response, will determine whether the program moves ahead.
Why it matters: There are very few approved medicines aimed at the core features of autism, so each clinical setback — and each attempt to salvage a program through talks with regulators — shapes how much hope families and investors can place in the next generation of potential treatments.