SAN DIEGO — Crinetics Pharmaceuticals announced on Sunday that its experimental drug for acromegaly, a rare and serious hormonal disorder, succeeded in a late-stage trial, bringing the San Diego biotech a step closer to applying for Food and Drug Administration approval — and entry into a crowded market with blockbuster therapies sold by pharma giants.
In the Phase 3 study, dubbed PATHFNDR-1, nearly 60 patients were randomly assigned to receive a placebo or a daily dose of paltusotine, an oral medicine designed to lower levels of growth hormone, which is produced in excess by people with acromegaly. After 36 weeks of treatment, 25 of the 30 patients given the drug had growth-factor levels that were within the normal range compared to just 1 of 28 participants who took the placebo, a difference that was statistically significant. Researchers also found that the treatment’s side effects were mild overall.
Crinetics plans to file for FDA approval if it gets positive results from a second, ongoing Phase 3 trial of the drug in acromegaly patients. The biotech expects results from that study, known as PATHFNDR-2, by the first quarter of next year.