Ocugen Announces FDA Agreement on Phase 2/3 Trial for OCU410ST in Stargardt Disease

Ocugen, Inc., headquartered in Malvern, Pa., has announced progress in its development of a gene therapy for diseases that cause blindness. The company, recognized for its pioneering work in biotechnology, has reached an understanding with the U.S. Food and Drug Administration (FDA) to proceed with a Phase 2/3 clinical trial for its treatment, OCU410ST. This trial, if successful, could lead to a biologics license application (BLA) submission.

The GARDian trial for OCU410ST has shown promising results. Notably, the therapy has a favorable safety profile, with no significant adverse reactions linked to the drug. The trial reported no instances of ischemic optic neuropathy, vasculitis, or other severe eye conditions. Additionally, there were no adverse events of special interest recorded. The treatment also demonstrated a significant reduction in lesion growth, showing a 52% slower progression in the treated eyes compared to untreated ones over six months. Furthermore, patients experienced a clinically meaningful improvement in visual function, with a statistically significant enhancement in best corrected visual acuity (BCVA) after six months.

Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen, expressed his satisfaction with the FDA’s support. He emphasized that this regulatory advancement will enable the company to begin a pivotal trial for what he describes as a groundbreaking treatment for Stargardt disease. Musunuri is hopeful that Ocugen will be able to file for a BLA by 2027, offering new hope to patients who previously had no treatment options for this condition.

Stargardt disease, a genetic disorder leading to vision loss, affects around 100,000 individuals in the United States and Europe. Currently, there are no available treatments for this condition. OCU410ST has been granted orphan drug status by both the FDA and the European Medicines Agency in recent years.

Dr. Lejla Vajzovic from Duke University highlighted the significance of receiving approval for a Phase 2/3 trial, noting that this strategy has not been previously attempted for Stargardt disease. The FDA’s approval is seen as a recognition of OCU410ST's potential to address a significant unmet medical need for approximately 44,000 patients in the U.S.

The upcoming clinical trial will involve 51 participants, with 34 receiving a single subretinal injection of OCU410ST in the eye with poorer vision, while the remaining 17 will act as control subjects. The primary goal of the trial is to assess changes in lesion size, with secondary objectives including visual acuity measurements. The data gathered over one year will be crucial for the BLA filing process.

Dr. Huma Qamar, Chief Medical Officer at Ocugen, pointed out that the collaboration with the FDA has accelerated the clinical development timeline by several years. Recent trial results have shown significant improvements in both structural and functional parameters, reinforcing the therapy's safety and tolerability.

The expedited clinical pathway for OCU410ST is expected to lower costs and provide relief for patients sooner than anticipated. The treatment utilizes an AAV delivery system to introduce the RORA gene to the retina, part of Ocugen's modifier gene therapy approach. This approach leverages Nuclear Hormone Receptor RORA to regulate pathways associated with Stargardt disease.

Ocugen, Inc. is a biotech company committed to developing innovative gene and cell therapies, biologics, and vaccines aimed at improving global health. Their work focuses on creating treatments for various retinal and infectious diseases, as well as addressing unmet needs in orthopedic conditions.