Ocugen (OCGN) – OCU400 Gene Therapy Trial Moving Forward With Highest Dose


Thursday, December 08, 2022

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product, Covaxin, is a killed-virus vaccine for COVID-19 in-licensed from Bharat Biotech (India). The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

DSMB Recommends MTD and Further Treatment.  Ocugen has announced that patient data from the Phase 1/2 trial testing OCU400 in retinitis pigmentosa (RP) has been reviewed by its Data Safety and Monitoring Board (DSMB).  The highest dose of the three tested was determined to be the maximum tolerated dose (MTD).  The DSMB found no serious adverse events (SAEs) in the treated cohorts, and recommended treating patients in the expansion phase at the highest dose. 

Additional Patients To Be Treated. The OCU400 Phase 1/2 trial has treated patient cohorts at three dose levels. These patients have gene mutations associated with RP (NR2E3 and RHO) that control and regulate expression of genes that act in later pathways. A total of 10 patients have been treated, with an additional 8 patients to be added. A cohort of 3 patients with a different mutation, CEP290, associated with Leber congenital amaurosis (LCA) are also being added. Enrollment is expected to be complete by 1Q23.


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