The FDA has cleared Ocugen, Inc. to begin enrollment of pediatric patients in its ongoing phase 1/2 trial of OCU400 for the treatment of inherited retinal diseases (IRDs) including retinitis pigmentosa (RP) and Leber Congenital Amaurosis (LCA).
Tell me about OCU400.
As a novel gene therapy product candidate, OCU400 is based on NR2E3—a nuclear hormone receptor (NHR) gene—that regulates physiological functions within the retina by delivering an adeno-associated viral (AAV) vector by subretinal injection.
OCU400 may help to reset the affected/altered gene cell networks along with reestablishing retinal homeostasis, potentially stabilizing cells and rescuing compromised photoreceptors from degenerative processes.
To note, the FDA granted OCU400 Orphan Drug designation in December 2022 for both RP and LCA.
Now this trial.
Initiated in January 2022, the phase 1/2 trial (NCT05203939) is a non-randomized, open-label study designed to assess the safety of a unilateral subretinal administration of OCU400 in patients with RP associated with NR2E3 and rhodopsin (RHO) gene mutation as well as in patients with LCA due to mutations in the CEP290 gene (LCA10).
Three cohorts of patients are receiving three level of doses:
- Cohort 1: up to 1.66 x 10^10 vg/mL (low dose)
- Cohort 2: up to 3.33 × 10^10 vg/mL (mid dose)
- Cohort 3: up to 1.66 × 10^11 vg/mL (high dose)
How many patients have been enrolled so far?
Ocugen has completed enrollment of 21 adult patients with RP; of those, 10 patients were dosed who had the NR2E3 and rhodopsin (RHO) gene mutations associated with RP; and an additional 8 patients with the mutations—as well as 3 patients with the CEP290 gene mutations associated with LCA—were also dosed.
Enrollment for additional LCA patients is ongoing.
Why include pediatric patients?
According to Arun Upadhyay, PhD, Ocugen’s CSO, “This approval further demonstrates the consistent, positive, and timely progress we are making in the phase 1/2 trial in adult patients.
Further, keeping in mind the significant number of pediatric patients diagnosed with RP and LCA, “it is very important for us to cover this age group in our clinical trials,” Upadhyay stated.
How many pediatric patients will be enrolled?
As of now, that number has yet to be defined. Check back here on the Clinical Trials site for updates.
What’s next?
Per Clinical Trials, the study is expected to conclude in December 2023. Ocugen also plans on initiating a phase 3 near the end of 2023.