Ocugen Shares Q1 2024 Financial Results; Conference Call/Webcast at 8:30 a.m. ET

Ocugen, Inc., a biotech firm specializing in novel gene and cell therapies as well as vaccines, has shared its financial results for the first quarter of 2024, along with updates on its business activities. The company, headquartered in Malvern, Pennsylvania, has been making significant strides in the clinical and regulatory arenas, especially in the field of treatments for blindness.

Dr. Shankar Musunuri, Ocugen’s Chairman, CEO, and Co-Founder, highlighted the major milestones achieved in early 2024, which are expected to transform the treatment landscape for blindness diseases. The FDA has approved the commencement of a Phase 3 clinical trial for Ocugen’s lead gene therapy candidate, OCU400, which targets retinitis pigmentosa (RP)—a significant step forward since existing treatments address only a fraction of the gene mutations causing RP.

The Phase 3 liMeliGhT trial for OCU400 will begin patient dosing in the second quarter of 2024, with 150 participants divided into two groups: one with the RHO gene mutation and another gene-agnostic group. The primary endpoint will assess the proportion of responders showing at least 2 Lux levels of improvement in vision.

Ocugen also plans to expand the Phase 3 trial to include patients with Leber congenital amaurosis (LCA) in the latter half of 2024, contingent on positive results from the ongoing Phase 1/2 study.

OCU400’s gene-agnostic mechanism provides hope for a broader RP patient population, which affects nearly 300,000 people in the U.S. and Europe combined. Additionally, Ocugen is advancing other gene therapy candidates, such as OCU410 for dry age-related macular degeneration (dAMD) and OCU410ST for Stargardt disease. Both candidates are in Phase 1/2 trials, with progress being made in dosing cohorts.

In April, Ocugen completed dosing the second cohort in the Phase 1/2 ArMaDa trial for OCU410 and moved to the second cohort in the Phase 1/2 GARDian trial for OCU410ST. These trials are crucial as they address diseases like geographic atrophy (GA) and Stargardt disease, which affect millions of patients.

Ocugen is also working on a cell therapy, NeoCart®, and has completed the renovation of a facility to meet Good Manufacturing Practice (GMP) standards. The company aims to initiate a Phase 3 trial for NeoCart® pending sufficient funding.

In the vaccine domain, Ocugen is collaborating with the National Institute of Allergy and Infectious Diseases (NIAID) on the clinical development of OCU500, a mucosal vaccine platform. An Investigational New Drug (IND) application is planned for mid-2024 to begin a Phase 1 clinical trial.

On the financial front, Ocugen reported $26.4 million in cash and cash equivalents as of March 31, 2024, down from $39.5 million at the end of 2023. The company’s operating expenses for the first quarter totaled $13.2 million, a decrease from $18.5 million for the same period in 2023. Research and development expenses were $6.8 million, and general and administrative expenses were $6.4 million. Ocugen posted a net loss of $0.05 per common share for the quarter, compared to $0.08 in the first quarter of 2023.

Despite the financial loss, Dr. Musunuri remains optimistic about Ocugen’s potential, emphasizing the importance of the company’s gene therapy programs and the broader promise of its cell therapy and vaccine platforms. Ocugen continues to seek partnerships to support its pipeline and maximize its therapeutic and financial potential.