Gene Therapy to be Assessed in Pediatric Patients With LCA, Retinitis Pigmentosa

OCU400 is also being evaluated in other retinal degeneration indications.

The FDA has approved the enrollment of pediatric patients with retinitis pigmentosa (RP) associated with NR2E3 and RHO mutations and Leber congenital amaurosis (LCA) associated with CEP290 gene mutations in Ocugen'songoing OCU400 phase 1/2 trial (NCT05203939).1

“This approval moves us 1 step closer in our efforts to bring OCU400, a novel gene-agnostic modifier gene therapy, to market as a potential life-changing treatment for children afflicted with inherited retinal diseases, such as RP and LCA,” Arun Upadhyay, PhD, chief scientific officer, Ocugen, said in a statement.1 “This approval further demonstrates the consistent, positive, and timely progress we are making with the Phase 1/2 trial in adult patients. Since a significant number of individuals in the pediatric age group are diagnosed with RP and LCA, it is very important for us to cover this age group in our clinical trials.”

Ocugen’s announcement also included an update that enrollment of adult patients with RP had completed while enrollment of adult patients with LCA continues. The study is currently evaluating 3 doses: 1.66 × 1010 vg/mL, 3.33 × 1010 vg/mL, and 1.66 × 1011 vg/mL, and is primarily assessing incidence of treatment-related adverse events (AEs), treatment-emergent AEs, and serious AEs. Secondary outcome measures include changes in Best-corrected visual acuity, low-luminance visual acuity, Electronic Visual Acuity Tester, Slit-lampbiomicroscopy, Intraocular pressure, Indirect ophthalmoscopy, anti-AAV5 and anti-hNR2E3 antibodies, and T-cell response. Other outcome measures include multi-luminance mobility testing, changes in ellipsoid zone width/length on wide-field 45° SD-OCT, contrast sensitivity, Full Field Light Stimulation Threshold, Static Visual Fields, Vision on Quality of Life, Full Field Electroretinogram, and wide-field fundus autofluorescence. The company plans to initiate the phase 3 trial near the end of 2023.

READ MORE: Gene Therapy Continues to be Well-Tolerated in Retinitis Pigmentosa

OCU400 uses Nuclear Hormone Receptors to be able to address multiple retinal diseases in a gene-agnostic mechanism. The therapy is based on the NR2E3 gene that regulates diverse physiological functions within the retina.

OCU400 also recently received orphan drug designation (ODD) from the FDA for both RP and LCA indications.2 The therapy has also received ODDs from the FDA for PDE6B gene mutation-associated retinal diseases, RHO mutation-associated retinal degeneration, NR2E3 mutation-associated retinal degeneration, and CEP290 mutation-associated retinal degeneration.3

“Receiving orphan drug designation is incredibly encouraging at this stage in the development of OCU400,” Upadhyay said in a previous statement.“We are excited by the potential of OCU400, a nuclear hormone-based modifier gene therapy product, to treat RP and LCA in a gene agnostic manner. We look forward to working collaboratively with the FDA and other agencies to progress OCU400 through clinical development to commercialization.”

1. Ocugen announces FDA approval for enrollment of pediatric patients in ongoing OCU400 phase 1/2 clinical trial for the treatment of retinitis pigmentosa (RP) and Leber Congenital Amaurosis (LCA). March 27, 2023.
2. Ocugen announces OCU400 receives orphan drug designations for retinitis pigmentosa and leber congenital amaurosis. News release. Ocugen, Inc. December 15, 2022.
3. Ocugen receives fourth FDA orphan drug designation for the same product, OCU400 (AAV-NR2E3) gene therapy, for the treatment of another key inherited retinal disease (IRD), PDE6B gene mutation-associated retinal diseases. News release. Ocugen, Inc. August 10, 2022.
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