Opus Genetics Announces Positive Low Dose Cohort 1 Data from Phase 1/2 Clinical Trial of OPGx-BEST1 and Successful FDA Type C Meeting with Potential Phase 3 Dosing in 2027
Opus Genetics reported positive 3- and 6-month Cohort 1 data from its Phase 1/2 BIRD-1 trial of OPGx-BEST1 in BEST1-related retinal diseases, with all five participants showing clinically meaningful visual function improvement and no serious adverse events or dose-limiting toxicities. A successful August 2026 FDA Type C meeting produced alignment on a potential pivotal endpoint of at least 3 decibels of microperimetry improvement in five or more prespecified loci, paired with a patient-reported outcome, with Phase 3 participant dosing expected to begin in 2027.
Key figures
- Drug Name
- OPGx-BEST1
- Phase Of Trial
- Phase 1/2
- Trial name
- BIRD-1
- Cash runway
- into 2029
- Cohort1 dose
- 1.5 x 10⁹ vg/eye
- Cohort2 dose
- 4.5 x 10⁹ vg/eye
- Bcva improvement
- 60% (3/5)
- Bvmd 6month data
- Q2 2027
- Llva improvement
- 40% (2/5)
- Phase3 dosing start
- 2027
- Cohort1 participants
- 5
- Cohort2 participants
- 8
- Cohort2 topline data
- Q2 2027
- Fda pivotal endpoint
- ≥3 dB microperimetry improvement in ≥5 prespecified loci plus patient-reported outcome
- Us diagnosed patients
- 13000
- Serious adverse events
- 0
- Structural improvement
- 4/5 participants
- Us symptomatic patients
- 23600
- Us undiagnosed patients
- 10600
- Dose limiting toxicities
- 0
- Cohort2 dosing completion
- Q4 2026
- Microperimetry improvement
- 75% (3/4 evaluable)
- Global symptomatic patients
- 45400
- Contrast sensitivity improvement
- 40% (2/5)
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