Ocugen completes dosing in Stargardt disease trial

MALVERN, Pa. - Ocugen, Inc. (NASDAQ: NASDAQ:OCGN), a biotechnology company specializing in gene and cell therapies and vaccines, has announced the completion of dosing for the third cohort in the Phase 1/2 GARDian clinical trial of their drug candidate OCU410ST for Stargardt disease. This genetic disorder, affecting around 100,000 individuals in the U.S. and Europe, leads to progressive vision loss due to retinal degeneration.

The latest development marks the end of Phase 1 in the dose-escalation part of the trial, with three subjects receiving the highest dose tested. The trial is conducted at six leading retinal surgery centers across the U.S., aiming to evaluate the safety and efficacy of a one-time subretinal administration of OCU410ST.

Dr. Huma Qamar, Chief Medical Officer of Ocugen, expressed the company's commitment to advancing the trial efficiently to address the unmet medical needs of patients with Stargardt disease. No FDA-approved treatments are currently available for this condition.

OCU410ST is a novel gene therapy that utilizes an AAV delivery platform to transport the RORA gene to the retina. The RORA gene is involved in regulating pathways linked to Stargardt disease, including lipofuscin formation and oxidative stress.

According to Dr. Charles Wykoff, MD, PhD, Director of Research at Retina Consultants of Texas and lead investigator in the study, the safety and tolerability profile of OCU410ST is encouraging as the clinical trial progresses.

Ocugen will continue to provide periodic updates on the clinical progress of OCU410ST. The company's broader mission includes developing treatments for various retinal diseases through innovative gene therapy platforms and conducting research in infectious and orthopedic diseases to address unmet medical needs.

This news is based on a press release statement from Ocugen, Inc.

In other recent news, Ocugen Inc., a biopharmaceutical entity, has made significant strides in its clinical programs and financials. The company successfully raised $32.6 million, extending its financial runway into the third quarter of 2025, and reported a cash balance of $16 million as of June 30, 2024. However, the total operating expenses for the same quarter amounted to $16.6 million, with a noted decrease in cash from $39.5 million on December 31, 2023, to $16 million on June 30, 2024.

One of the major developments from Ocugen includes the progress of its OCU400 gene therapy platform. The therapy, which is currently in Phase 3 trials for retinitis pigmentosa, is on track for a Biologics License Application submission in 2026. The company is also working on its OCU410 and OCU410ST programs for geographic atrophy and Stargardt disease, respectively, and is actively addressing the FDA's clinical hold on the OCU200 program.

In addition, Ocugen has received a No Objection Letter from Health Canada for the Phase 3 clinical trial of OCU400, enabling the company to extend its liMeliGhT study to Canadian patients. This move could potentially accelerate the treatment's development for a gene-agnostic approach to retinitis pigmentosa, a rare, inherited retinal disease. These are among the recent developments in the company's pursuit to solidify its position as a biotech leader.

InvestingPro Insights

As Ocugen, Inc. (NASDAQ: OCGN) makes strides in its GARDian clinical trial for Stargardt disease, investors and industry observers are closely monitoring the company's financial health and stock performance. According to InvestingPro data, Ocugen has a market capitalization of $382.85 million, reflecting the biotech market's interest in its potential to address unmet medical needs. Despite the optimism surrounding their clinical developments, analysts have highlighted concerns, as evidenced by an anticipated sales decline in the current year and weak gross profit margins.

An InvestingPro Tip notes that Ocugen's stock price has been quite volatile, which could be indicative of the market's response to both the company's clinical trial advancements and its financial metrics. The company's Price / Book ratio stands at a high 22.62, suggesting a premium valuation compared to its book value as of the last twelve months ending Q2 2024. Additionally, while Ocugen has experienced a significant year-to-date price total return of 131.3%, it's important to note that the stock has fared poorly over the last month, with a -18.9% return.

Investors considering Ocugen as part of their portfolio should be aware that the company is not expected to be profitable this year, and it has not been profitable over the last twelve months. For those looking for more detailed analysis and additional insights, there are 11 more InvestingPro Tips available, which can provide further guidance on the stock's prospects.

For more information, visit the InvestingPro platform for Ocugen at https://www.investing.com/pro/OCGN.

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