Opus Genetics advances gene therapy in pediatric trial

Published 18/02/2025, 13:34
Opus Genetics advances gene therapy in pediatric trial

DURHAM, N.C. - Opus Genetics, Inc. (NASDAQ:IRD), a biotechnology firm focusing on gene therapies for inherited retinal diseases, has dosed the first pediatric patient in a Phase 1/2 clinical trial of OPGx-LCA5, an investigational treatment for Leber congenital amaurosis (LCA). According to InvestingPro data, the company maintains a strong financial position with a current ratio of 6.63, indicating robust liquidity to support its clinical programs. The company expects to release initial data from the pediatric cohort by the third quarter of 2025.

The trial, which includes adult participants, has shown early signs of success, with the first three adult patients experiencing meaningful visual improvement after treatment. These results, initially observed at six months and to be further detailed with 12-month follow-up data, will be presented at a major medical conference in the second quarter of 2025. InvestingPro analysis shows that while the company is currently pre-profit, analysts have set price targets ranging from $8 to $15, suggesting significant upside potential if the trials succeed.

An FDA Type D meeting is scheduled for March 2025 to discuss the design and endpoints for a potential Phase 3 trial. CEO George Magrath emphasized the importance of early intervention in pediatric patients to preserve or restore vision and expressed optimism about the upcoming discussions with the FDA.

The Phase 1/2 trial is designed to assess the safety and preliminary efficacy of OPGx-LCA5, which uses an adeno-associated virus 8 (AAV8) vector to deliver a functional LCA5 gene to the retina in patients with biallelic mutations in the LCA5 gene.

Opus Genetics continues to develop its pipeline, including a BEST1 gene therapy expected to enter Phase 1/2 trials in 2025 and treatments for other ophthalmic disorders, such as Phentolamine Ophthalmic Solution 0.75% and APX3330, which are in various stages of clinical evaluation. With a market capitalization of just $35.67 million and InvestingPro’s Fair Value analysis indicating the stock is currently undervalued, investors seeking detailed financial insights can access additional analysis and 8 more ProTips through InvestingPro.

This news is based on a press release statement from Opus Genetics, Inc. The company’s forward-looking statements are subject to risks and uncertainties that could affect the actual results of the trials and the development of their product candidates. Additional information about the trial can be found on clinicaltrials.gov under identifier NCT05616793.

In other recent news, Opus Genetics, Inc. has made significant moves in its operations and corporate governance. The company has launched a $40 million at-the-market equity issuance program in collaboration with Leerink Partners LLC, aiming to raise capital for general corporate purposes including the advancement of product candidates and clinical trials. The pharmaceutical company has also updated its executive employment agreements, enhancing severance terms for its top executives, Dr. George Magrath, CEO, and Nirav Jhaveri, CFO.

Furthermore, Opus Genetics has aligned with the U.S. Food and Drug Administration for a Phase 3 clinical trial of APX3330, an oral medication being tested as a treatment for moderate to severe non-proliferative diabetic retinopathy. The company has also announced the date for its 2025 Annual Meeting of Stockholders, scheduled for April 30, 2025.

These developments have been announced in line with Opus Genetics’ efforts to standardize its executive employment agreements, advance its product candidates, and adhere to the guidelines outlined in the Company’s Amended and Restated Bylaws. These are recent developments and crucial for investors and stakeholders in understanding the company’s governance practices and its future plans.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

Latest comments

Risk Disclosure: Trading in financial instruments and/or cryptocurrencies involves high risks including the risk of losing some, or all, of your investment amount, and may not be suitable for all investors. Prices of cryptocurrencies are extremely volatile and may be affected by external factors such as financial, regulatory or political events. Trading on margin increases the financial risks.
Before deciding to trade in financial instrument or cryptocurrencies you should be fully informed of the risks and costs associated with trading the financial markets, carefully consider your investment objectives, level of experience, and risk appetite, and seek professional advice where needed.
Fusion Media would like to remind you that the data contained in this website is not necessarily real-time nor accurate. The data and prices on the website are not necessarily provided by any market or exchange, but may be provided by market makers, and so prices may not be accurate and may differ from the actual price at any given market, meaning prices are indicative and not appropriate for trading purposes. Fusion Media and any provider of the data contained in this website will not accept liability for any loss or damage as a result of your trading, or your reliance on the information contained within this website.
It is prohibited to use, store, reproduce, display, modify, transmit or distribute the data contained in this website without the explicit prior written permission of Fusion Media and/or the data provider. All intellectual property rights are reserved by the providers and/or the exchange providing the data contained in this website.
Fusion Media may be compensated by the advertisers that appear on the website, based on your interaction with the advertisements or advertisers
© 2007-2025 - Fusion Media Limited. All Rights Reserved.