Opus Genetics Receives FDA Clearance for OPGx-BEST1 Gene Therapy Candidate, Initiating Phase 1/2 Trial in 2H 2025 for Treatment of BEST1-Related Inherited Retinal Disease.

Opus Genetics has received FDA clearance for its Investigational New Drug application for OPGx-BEST1, a gene therapy for bestrophin-1 (BEST1)-related inherited retinal disease. The Phase 1/2 trial is expected to initiate in 2H 2025 and will evaluate the safety, tolerability, and preliminary efficacy of a single subretinal injection of OPGx-BEST1 in patients with genetically confirmed BEST1-related IRD. This is Opus Genetics' third ongoing clinical program and reflects its commitment to advancing multiple therapies for patients with urgent, unmet needs.

Opus Genetics (Nasdaq: IRD) has received FDA clearance for its Investigational New Drug (IND) application for OPGx-BEST1, a gene therapy targeting BEST1-related inherited retinal disease (IRD). This regulatory milestone marks a significant step forward for the company and the IRD community. The Phase 1/2 trial is expected to commence in the second half of 2025 and will evaluate the safety, tolerability, and preliminary efficacy of a single subretinal injection of OPGx-BEST1 in patients with genetically confirmed BEST1-related IRD.

Opus Genetics' proprietary AAV-based gene therapy platform will deliver a functional copy of the BEST1 gene directly to retinal pigment epithelium (RPE) cells. The trial aims to assess the gene therapy's impact on visual function and retinal structure, critical measures for establishing therapeutic value. This is the company's third ongoing clinical program, reflecting its commitment to advancing multiple therapies for patients with urgent, unmet needs.

Best disease, also known as vitelliform macular dystrophy, is a rare, inherited retinal condition causing macular degeneration due to mutations in the BEST1 gene. This condition leads to progressive vision loss and, in some cases, blindness. Currently, there are no approved treatments for BEST1-related IRDs, making Opus Genetics' OPGx-BEST1 trial a significant advancement in the field.

The FDA's clearance of Opus Genetics' IND application for OPGx-BEST1 represents a significant regulatory milestone in the company's clinical development program. This approval enables the company to initiate its planned Phase 1/2 trial, which will be a multi-center, open-label study. The trial will focus on safety, tolerability, and preliminary efficacy, with a particular emphasis on functional and anatomical endpoints.

Opus Genetics' pipeline features several gene therapies targeting various IRDs, including Leber congenital amaurosis (LCA) and retinitis pigmentosa. The company's lead gene therapy candidate, OPGx-LCA5, is currently in an ongoing Phase 1/2 trial for LCA5-related mutations. Additionally, Opus Genetics is advancing Phentolamine Ophthalmic Solution 0.75%, a partnered therapy being studied in two Phase 3 programs for presbyopia and reduced low light vision and nighttime visual disturbances.

The FDA's clearance of Opus Genetics' IND application for OPGx-BEST1 is a testament to the company's scientific rigor and commitment to developing innovative therapies for rare diseases. The trial's results will provide valuable insights into the potential of gene therapy for BEST1-related IRDs, potentially paving the way for future treatments.

References:
[1] https://www.stocktitan.net/news/IRD/opus-genetics-announces-fda-clearance-of-ind-application-for-gene-4sin3lhpftx1.html