Core Insights - Opus Genetics reported positive 12-month results from the Phase 1/2 trial of OPGx-LCA5, showing sustained treatment benefits for adult patients with Leber congenital amaurosis 5 (LCA5) [2][10] - The company is enrolling pediatric patients in the LCA5 study, with initial data expected in Q3 2025 [1][10] - Opus is on track to file an IND for OPGx-BEST1 and initiate a Phase 1/2 trial by Q4 2025, with preliminary data anticipated in Q1 2026 [1][6] Gene Therapy Programs - OPGx-LCA5 is the most advanced investigational gene therapy candidate targeting inherited retinal degeneration due to mutations in the LCA5 gene [5][6] - The pipeline includes seven AAV-based gene therapy assets for various inherited retinal diseases (IRDs) and the OPGx-BEST1 program for BEST1-related IRDs [6][16] - The FDA granted RMAT designation to OPGx-LCA5, allowing for expedited development and review [10] Financial Highlights - Opus reported a net loss of $8.2 million for Q1 2025, compared to a net loss of $7.1 million in Q1 2024, with a loss per share of $(0.24) [15][28] - License and collaborations revenue increased to $4.4 million in Q1 2025 from $1.7 million in Q1 2024, primarily from the Viatris License Agreement [12][27] - As of March 31, 2025, the company had cash and cash equivalents of $41.8 million, expected to fund operations into Q2 2026 [11][24] Clinical Trials and Development - The LYNX-2 pivotal Phase 3 trial for Phentolamine Ophthalmic Solution 0.75% completed enrollment in Q1 2025, with topline data expected mid-2025 [10][14] - The VEGA-3 pivotal Phase 3 trial for the same solution in presbyopia also completed enrollment in Q1 2025, with topline data expected in the first half of 2025 [10][14] - Initial data from three pediatric patients treated with OPGx-LCA5 is anticipated in Q3 2025 [10][14]
Opus Genetics Announces Financial Results for First Quarter 2025 and Provides Corporate Update