Opus Genetics' OPGx-BEST1 Shows Positive Phase 1/2 Data, FDA Aligns on Pivotal Endpoint
IRD has more than doubled off its 52-week low of $1.28 on elevated volume (2.0× avg).
Summary
Opus Genetics reported positive low-dose Cohort 1 data from its Phase 1/2 trial of OPGx-BEST1 in BEST1-related retinal diseases. All five participants showed clinically meaningful visual function improvement, with structural improvements in four. No serious adverse events or dose-limiting toxicities were observed. The company also announced a successful FDA Type C meeting, aligning on a potential pivotal endpoint of ≥3 dB microperimetry improvement with patient-reported outcomes, and expects Phase 3 dosing to begin in 2027. Cohort 2 has been over-enrolled with eight participants, with topline 3-month data expected in Q2 2027. New epidemiology research suggests a larger addressable patient population than previously estimated. This is a major clinical and regulatory milestone for a lead gene therapy program, with cash runway into 2029 supporting multiple inflection points.
At the time of this announcement, IRD was trading at $4.64 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $361.1M. The 52-week trading range was $1.28 to $5.81. This news item was assessed with positive market sentiment and an importance score of 9 out of 10. Source: GlobeNewswire.