Ocugen, Inc. Common StockOCGN
Recorded
Ocugen, Inc. Common Stock Canaccord Genuity's 46th Annual Growth Conference
Review the key takeaways and the transcript of this earnings call.
PeriodFY 0Duration26 min
Key takeaways
- Ocugen currently leads in ophthalmology gene therapies with three late-stage programs targeting inherited retinal diseases (IRDs) including retinitis pigmentosa, Stargardt disease, and dry age-related macular degeneration (AMD).
- The company aims to file Biologics License Applications (BLAs) and obtain market authorizations in the US, EU, and Japan for retinitis pigmentosa and Stargardt disease by 2026, and for AMD by 2028.
- Ocugen's gene modifier technology uses nuclear hormone receptors, specifically NR2E3 and RORA, to regulate gene networks and cellular homeostasis in retinal cells, potentially stopping or reversing vision loss.
- The Stargardt disease program uses a subretinal gene therapy vector delivering the modifier gene to reset cellular homeostasis and create a healthy environment for retinal cells, aiming to stop or reverse disease progression with a one-time injection.
- The ongoing phase 2/3 pivotal trial for Stargardt disease expects top-line data in Q2 2024, with plans to file BLAs shortly thereafter and potential approvals in 2028.
- For geographic atrophy (GA) in dry AMD, Ocugen uses a similar gene construct with a different dose; the phase 3 trial is planned to start in Q2 2024 as a single global trial with FDA and EMA alignment.
- The retinitis pigmentosa (RP) program targets NR2E3 to regulate phototransduction pathways affecting peripheral and night vision, with a phase 3 trial ongoing and top-line data expected in Q1 2024.
- Phase 1/2 RP data showed promising safety and efficacy with no serious adverse events related to the gene therapy across over 325 patients treated in clinical trials and expanded access programs.
- Ocugen has completed commercial-scale manufacturing and pharmacokinetic validation runs for RP and Stargardt gene therapies, enabling readiness for rolling BLA submissions.
- The company converted a $130 million convertible note financing, extending its cash runway into 2028 to support ongoing phase 3 trials and future commercialization efforts.
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