Ocugen (OCGN) investor relations material

Platform and pipeline

• Modifier-gene approach uses NR2E3 for OCU400 and RORA for OCU410 and OCU410ST, targeting broad disease pathways and mutation populations.

• OCU400 targets retinitis pigmentosa (RP); OCU410ST targets Stargardt disease in a Phase II/III trial; OCU410 targets geographic atrophy (GA) in Phase III.

• OCU410ST topline results expected in Q2 2027, followed by BLA and market authorization applications; OCU410 is recruiting for a 240-patient global trial, with recruitment targeted for completion in 2027.

• OCU410 received RMAT designation after Phase II data; the trial spans the U.S., Canada, and EU, with a one-year treatment period.

OCU400 clinical evidence and milestones

• Phase I/II included three mutations; 89% of patients showed stabilization or improvement on a key functional measure, with no gene-therapy-related SAEs reported.

• Phase III enrolls 140 patients across ~30 mutations; the primary endpoint compares the share of treated and control patients improving by two levels on a mobility test.

• Management cited 99% statistical power for the Phase III design and said a 20% responder rate could meet the endpoint if untreated responders remain near the 0%-1% Phase I/II level.

• Topline results expected by end-Q1 2027; planned pre-BLA meeting immediately afterward, BLA filing targeted for Q2 2027, and potential PDUFA timing in Q4 2027.

• Europe MAA filing planned after the U.S. BLA; discussions with Japan’s PMDA are underway, with international filings to follow.

Commercial strategy

• All programs use subretinal administration; management positions treatment as one-time and plans to expand its 30-35 trial-center network to 50-100 centers of excellence.

• Management cited U.S. and EU pricing assumptions of $1.5 million per patient for RP and Stargardt and $450,000 for GA, describing large potential markets and one-time treatment demand.

• Commercial planning includes payer engagement, CMS work, centers of excellence, and ex-U.S. partnerships.