Ocugen Announces Q1 2024 Financial Results and Business Update

Ocugen, Inc., a biotechnology company specialized in gene and cell therapies and vaccines, has released its financial results for the first quarter of 2024, along with a business update. The company has achieved significant clinical and regulatory milestones, which are paving the way for new treatments for blindness diseases.

Dr. Shankar Musunuri, Chairman and CEO of Ocugen, emphasized that the company has received FDA clearance and EMA support for the Phase 3 clinical trial of its lead modifier gene therapy candidate, OCU400. This therapy offers a potential one-time treatment for life and is expected to revolutionize the treatment paradigm for patients with retinitis pigmentosa (RP).

The FDA clearance allows Ocugen to begin the Phase 3 liMeliGhT clinical trial for OCU400, marking the first gene therapy to enter late-stage trials with a broad RP indication. RP, a genetic disorder affecting nearly 300,000 people in the U.S. and Europe, currently has only one marketed treatment targeting just one of its 100 gene mutations. OCU400's gene-agnostic mechanism of action offers hope for a larger patient population.

The Phase 3 trial is set to start dosing patients in the second quarter of 2024, involving 150 participants, with 75 having the RHO gene mutation and 75 being gene-agnostic. The primary endpoint for the study, Luminance Dependent Navigation Assessment (LDNA), will evaluate the proportion of responders achieving at least 2 Lux levels of improvement in vision from baseline.

Ocugen plans to expand the Phase 3 trial in the latter half of 2024 to include patients with Leber congenital amaurosis (LCA), contingent upon favorable Phase 1/2 results. Modifier gene therapy holds promise not only for inherited retinal diseases but also for multifactorial blindness diseases affecting millions.

OCU410, another of Ocugen’s gene therapy candidates, focuses on regulating pathways involved in dry age-related macular degeneration (dAMD). Designed as a one-time gene therapy, it targets geographic atrophy (GA), an advanced dAMD stage affecting 2-3 million people in the U.S. and Europe. OCU410ST is being developed for Stargardt disease, impacting approximately 100,000 individuals in these regions.

Dosing for the second cohort in the Phase 1/2 ArMaDa clinical trial for OCU410 was recently completed, and the first cohort dosing for the Phase 1/2 GARDian trial for OCU410ST was also finished earlier this year. Following approval from the Data Safety and Monitoring Board, the trial will progress to the second cohort.

Ocugen's business operations underscore the importance of their gene therapy programs. The company is open to partnerships to support its pipeline, including cell therapies and vaccine platforms, which hold significant therapeutic and financial potential.

In the realm of ophthalmic gene therapies, the company has made notable progress:
- OCU400 has received FDA clearance for the Phase 3 liMeliGhT clinical trial and EMA's acceptability for the same trial, currently underway.
- OCU410 is in Phase 1/2 with patient enrollment ongoing and dosing completed in the second cohort.
- OCU410ST has completed dosing for the first cohort and has initiated enrollment for the second.

For regenerative cell therapies, Ocugen has renovated a facility to meet FDA's Good Manufacturing Practice standards and plans to initiate a Phase 3 trial of NeoCart®, subject to funding.

Ocugen's vaccine portfolio also sees collaboration with NIAID on clinical development for OCU500, with plans to submit an IND by mid-2024 to start Phase 1 trials.

Financially, Ocugen reported $26.4 million in cash and equivalents as of March 31, 2024, down from $39.5 million at the end of 2023. Operating expenses for Q1 2024 totaled $13.2 million, down from $18.5 million during the same period in 2023. The company reported a net loss of $0.05 per share for Q1 2024 compared to a $0.08 net loss per share in Q1 2023.

Ocugen has scheduled a conference call and webcast for 8:30 a.m. ET to discuss these results and business highlights, making the session accessible to all interested parties.