By way of comparison, Ocugen earlier this year presented results from the Phase 1/2 trial of the gene therapy, OCU 400 in Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA).
Safety and efficacy data was provided for 18 patients. Improvement in LLVA was one of the exploratory endpoints used in the trial.
After 12 months, on the LLVA endpoint, 39% of patients had gained⦥ 5 letters and ~ 22% had gained ⦥10 letters.
In comparison, in patients dosed with 30mcg VP-001, after just 6 months, the treated eyes of all 3 patients had gained ⦥10 letters and one had gained ⦥ 15 letters. (S10)
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