Ocugen Gets Dsmb Nod for Phase 1/2 Trial with Medium Dose of OCU410

Ocugen Gets Dsmb Nod for Phase 1/2 Trial with Medium Dose of OCU410

By, Admin 9 April 2024

OOcugen gets DSMB nod to proceed dosing in phase 1/2 ArMaDa trial with medium dose of OCU410 to treat geographic atrophy

Overview

Ocugen, Inc., a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, announced that the Data and Safety Monitoring Board (DSMB) for the phase 1/2 ArMaDa clinical trial for OCU410 recently convened and approved to proceed dosing with the medium dose of OCU410 in the dose-escalation phase of the study.

Trial Candidates

  • Three subjects with geographic atrophy (GA) were dosed in the phase 1/2 clinical trial to date. 
  • An additional three subjects will be dosed with the medium dose (Cohort 2) and three patients with the high dose (Cohort 3) of OCU410 in the dose-escalation phase.

Words from Trial Chair

  • “The DSMB has recommended moving forward to medium dose for dosing subjects with GA,” said Dr. Peter Chang, MD, FACS, DSMB chair for the OCU410 clinical trial. 
  • “No serious adverse events (SAEs) related to OCU410 have been reported to date. I believe that this marks a critical next step towards determining the optimal dosing regimen and an important milestone for the clinical development of OCU410.”

Words from Ocugen

  • “The positive DSMB review for the first modifier gene therapy for GA significantly builds on the favorable safety and tolerability profile exhibited by OCU410,” said Huma Qamar, M.D., MPH, chief medical officer of Ocugen. 
  • “We are very enthusiastic about the potential of OCU410 as a one-time treatment for life with a single sub-retinal injection.”

One Remedy for Various Pathways

  • Currently approved products to treat GA have significant limitations, as they require multiple injections per year and only target one pathway contributing to GA. 
  • OCU410 regulates multiple pathways involved with the disease including: lipid metabolism, inflammation, oxidative stress, and membrane attack complex (complement).

ArMaDa Clinical Trial

  • The ArMaDa clinical trial will assess the safety of unilateral subretinal administration of OCU410 in subjects with GA and will be conducted in two phases. 
  • Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels [low dose (2.5×1010 vg/mL), medium dose (5×1010 vg/mL), and high dose (1.5 ×1011 vg/mL)]. 
  • Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which subjects will be randomized in a 1:1:1 ratio to either one of two OCU410 treatment groups or to an untreated control group.

Ocugen’s Committed for Ophthalmic Diseases

  • Ocugen is committed to finding solutions for inherited retinal diseases as well as blindness diseases affecting millions. 
  • GA is an advanced form of dry age-related macular degeneration (dAMD) and affects approximately 1 million people in the United States.

About dAMD

  • dAMD affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula. 
  • The macula is the part of the retina responsible for clear vision in one’s direct line of sight. dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function and central vision impairment. 
  • dAMD accounts for 85-90% of the total AMD population.

OCU410

  • OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (ROR Related Orphan Receptor A) gene. 
  • The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role as well as inhibiting the complement system in in-vitro and in-vivo (animal model) studies. 
  • These results demonstrate the ability for OCU410 to target multiple pathways linked with dAMD pathophysiology. 
  • Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of GA.

Ocugen

Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe.

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