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RESEARCH TRIANGLE PARK, N.C. - Opus Genetics (NASDAQ:IRD) announced Monday that the U.S. Food and Drug Administration has accepted its Investigational New Drug application for OPGx-BEST1, a gene therapy targeting bestrophin-1 (BEST1)-related inherited retinal disease. The micro-cap company, valued at approximately $72 million, has seen its shares surge over 13% in the past week on positive developments. According to InvestingPro data, analysts see significant upside potential for the stock, with price targets ranging from $5 to $8.
The company plans to initiate a Phase 1/2 clinical trial in the second half of 2025. The multi-center, open-label study will evaluate the safety, tolerability, and preliminary efficacy of a single subretinal injection of OPGx-BEST1 in patients with genetically confirmed BEST1-related inherited retinal disease. While the company maintains a healthy current ratio of 1.9, InvestingPro analysis indicates challenges ahead with negative EBITDA of -$36.8 million in the last twelve months.
Best disease, or vitelliform macular dystrophy, is a rare inherited retinal condition caused by mutations in the BEST1 gene, which can lead to progressive vision loss and in some cases blindness. Currently, there are no approved treatments for BEST1-related inherited retinal diseases.
"The FDA’s clearance of our BEST1 IND is a significant step forward for the IRD community," said George Magrath, Chief Executive Officer of Opus Genetics, according to the press release.
The therapy is designed to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium cells where the defective gene resides. The company stated that preclinical work has demonstrated restoration of BEST1 protein expression and improved retinal function in relevant disease models.
OPGx-BEST1 will be Opus Genetics’ third ongoing clinical program. The company’s pipeline also includes OPGx-LCA5, which is in an ongoing Phase 1/2 trial for Leber congenital amaurosis, and Phentolamine Ophthalmic Solution 0.75%, which is being studied in two Phase 3 programs. Discover more insights and 6 additional key tips about Opus Genetics with an InvestingPro subscription, including detailed financial health scores and valuation metrics.
In other recent news, Opus Genetics reported positive outcomes from its VEGA-3 Phase 3 trial for Phentolamine Ophthalmic Solution 0.75%, which successfully met its primary endpoint in treating presbyopia. The trial demonstrated that 27.2% of patients experienced significant improvement in near vision without affecting distance vision. Additionally, the company announced favorable results from its Phase 3 LYNX-2 clinical trial, targeting night vision impairment, with 17.3% of patients showing notable gains in visual acuity. Meanwhile, Opus Genetics has seen changes in its executive team, with CFO Nirav Jhaveri departing and Amy Rabourn stepping in as the interim principal financial officer. The company emphasized that the departure was not due to any disagreements over accounting practices. On the analyst front, H.C. Wainwright reiterated a Buy rating for Opus Genetics, driven by the company’s strategic partnership with the Global RDH12 Alliance. Lucid Capital Markets also initiated coverage with a Buy rating, citing promising developments in Opus’s gene therapy pipeline. These recent developments highlight the company’s ongoing efforts in advancing its therapeutic programs.
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