The FDA has approved a pivotal Phase 2/3 trial for OCU410ST, a gene therapy targeting Stargardt disease, which could potentially expedite clinical development by up to three years.

Ocugen, Inc. announced FDA alignment for this trial on February 27, 2025, marking a significant step forward in their efforts to address this condition.

Dr. Shankar Musunuri, CEO of Ocugen, expressed optimism about the therapy's potential, highlighting the urgency of the trial as Stargardt disease currently affects around 100,000 people in the U.S. and Europe with no approved treatment options.

The upcoming trial will involve 51 subjects, with 34 receiving the OCU410ST injection and 17 serving as controls, focusing on changes in atrophic lesion size as the primary endpoint.

Ocugen's fourth-quarter and full-year 2024 financial results showcased advancements in gene therapy and regulatory initiatives, with the company ending the year with $58.8 million in cash.

In 2024, the company reported a decrease in research and development expenses to $32.1 million from $39.6 million the previous year, reflecting a strategic focus on cost management.

OCU410ST utilizes an AAV delivery platform to target the RORA gene, which is involved in the pathology of Stargardt disease.

Earlier trial results indicated a 52% reduction in lesion growth and significant vision improvement in patients, with the GARDian trial demonstrating a favorable safety profile.

Patients receiving OCU410ST showed a statistically significant improvement in visual function, with a two-line increase in best corrected visual acuity at the six-month follow-up.

The trial's secondary endpoints will evaluate visual acuity against untreated controls, using one-year data for the Biologics License Application filing.

Looking ahead, Ocugen plans to submit three potential Biologics License Applications between 2026 and 2028, positioning itself for long-term growth.

Dr. Huma Qamar, Chief Medical Officer at Ocugen, noted that the FDA's approval accelerates the clinical development timeline, potentially reducing costs and addressing the disease burden more swiftly.