Watch for OPGx-LCA5 Phase 3 dosing initiation in Q4 2026 and further OPGx-BEST1 Cohort 2 data readouts, which will be key catalysts. The small sample sizes (n=4-6 per program) limit current statistical power, but the consistency of functional improvement across both programs is encouraging for a clinical-stage biotech.
Price Chart
Executive Summary
Opus Genetics presented positive interim clinical data from its OPGx-BEST1 and OPGx-LCA5 gene therapy programs at Retina in Rio 2026. OPGx-BEST1 showed improved retinal sensitivity in 75% of evaluable low-dose Cohort 1 participants, with new structural imaging insights guiding future trial design. OPGx-LCA5 demonstrated sustained visual acuity and light sensitivity improvements through 24 months in adults and 6 months in pediatrics, with Phase 3 dosing expected Q4 2026. The data are early-stage and from small cohorts, but support continued pipeline advancement.
Key Facts
- OPGx-BEST1: 3 of 4 evaluable participants (75%) in low-dose Cohort 1 showed improved retinal sensitivity in the treated transitional zone.
- OPGx-BEST1: High-dose Cohort 2 fully enrolled with 11 participants.
- OPGx-LCA5: 3 adult participants showed sustained visual acuity improvement through 24 months; 3 pediatric participants showed improvement through 6 months.
- OPGx-LCA5: Phase 3 natural history and treatment cohorts fully enrolled; dosing initiation expected Q4 2026.
- OPGx-LCA5 primary efficacy endpoint: mean retinal sensitivity improvement of >7 dB across central 16 test loci.
- OPGx-BEST1 natural history data: untreated BVMD transition zone sensitivity declines at 0.36-0.56 dB/year (statistically significant), while whole-macula averages appear stable.
Financial Impact
No financial figures reported. Filing covers clinical data updates only.
Risk Factors
- Very small sample sizes (n=4-6 per program) limit reliability of efficacy signals.
- OPGx-BEST1 data are early (3-6 month follow-up); durability and dose-response from Cohort 2 unknown.
- OPGx-LCA5 Phase 3 endpoint (>7 dB improvement across 16 loci) is ambitious and unvalidated in LCA5.
- Both programs target ultra-rare diseases with small addressable patient populations (~45,400 BEST1 global, ~3,240 LCA5 global).
Investment Themes
Documents Analyzed
This report is based on 7 SEC documents filed with EDGAR.
| Document | Accession Number |
|---|---|
| 8-K Filing (Primary) | 0001140361-26-039781 |
| Document: ef20083857_8k.htm | 0001140361-26-039781 |
| Document: ef20083857_ex99-3.htm | 0001140361-26-039781 |
| Document: ef20083857_ex99-1.htm | 0001140361-26-039781 |
| Document: 0001140361-26-039781-index-headers.html | 0001140361-26-039781 |
| Document: 0001140361-26-039781-index.html | 0001140361-26-039781 |
| Document: 0001140361-26-039781.txt | 0001140361-26-039781 |
Filters
| Type | Now | ||||
|---|---|---|---|---|---|
Oct 23, 2026 3d ago | 144 | $3.52 | awaiting T+60 | — | $3.47 (−1.42%) |
Oct 21, 2026 5d ago | 8-K | $3.82 | awaiting T+60 | — | $3.47 (−9.16%) |
Oct 15, 2026 11d ago | 8-K | $3.84 | awaiting T+60 | — | $3.47 (−9.64%) |
Oct 15, 2026 11d ago | Press Release | $3.84 | awaiting T+60 | — | $3.47 (−9.64%) |
Sep 22, 2026 4w ago | Institutional Cluster | $5.16 | awaiting T+60 | — | $3.47 (−32.75%) |
Sep 11, 2026 6w ago | 8-K | $6.04 | awaiting T+60 | — | $3.47 (−42.55%) |
Sep 9, 2026 6w ago | 8-K | $5.73 | awaiting T+60 | — | $3.47 (−39.44%) |
Sep 2, 2026 7w ago | Press Release | $4.45 | awaiting T+60 | — | $3.47 (−22.02%) |
Aug 6, 2026 11w ago | 8-K | $3.72 | awaiting T+60 | — | $3.47 (−6.72%) |
Jul 23, 2026 13w ago | 144 | $2.92 $4.05 | ▲ +38.70% | ▲ +34.15% | $3.47 (+18.84%) |
US Market Status
Subscribe to SecBot
Get Real-Time SEC Filing Intelligence
Comprehensive SEC filing analysis delivered the moment filings hit EDGAR. Sentiment scoring, impact analysis, and actionable insights for every material event.
Create a free account Live analysis, Telegram alerts, watchlists and the MCP server for subscribers