OPGx-BEST1 / Opus Genetics - LARVOL DELTA

Opus Genetics, Inc…announced…new clinical data from its ongoing Phase 1/2 study of OPGx-BEST1 gene therapy, presented at the 49th Annual Meeting of the Macula Society, in San Diego, California. (Opus Genetics Press Release) - "The sentinel participant is a 63-year-old female with Autosomal-Recessive Bestrophinopathy (ARB) disease with severe functional impairment. The data demonstrated that OPGx-BEST1 was well tolerated with no ocular inflammation, no ocular or treatment-related adverse events, and no dose limiting toxicities. Early signals of functional vision improvement were observed, including an equivalent 12-letter gain in Best Corrected Visual Acuity (BCVA) in the treated study eye...Recruitment in the Phase 1/2 study is ongoing at two clinical sites in the U.S., with additional sites expected to open in Florida, Cincinnati and New York. Two participants have been enrolled to date, with 3-month results from the full Cohort 1 expected in mid-year 2026."

Enrollment status • P1/2 data • Macular Degeneration

OPGx-BEST1 – Gene Therapy for BEST1-Related IRD (Opus Medicus) - "First participant dosed in Phase 1/2 trial (BIRD-1) with enrollment ongoing; Initial data expected this quarter at Macula Society with 3-month results from the entire Cohort 1 expected in mid-2026; OPGx-BEST1 is potentially eligible for multiple regulatory designations which the Company expects to file for in 2026."

Enrollment status • Filing • P1/2 data • Macular Degeneration

BIRD-1: Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB) (clinicaltrials.gov) - P1/2 | N=10 | Recruiting | Sponsor: Opus Genetics, Inc

New P1/2 trial • Pan tumor • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

OPGx-BEST1 - Gene Therapy for BEST1-Related IRD (The Manila Times) - "Investigational New Drug (IND) submission and Phase 1/2 trial initiation remain on track for the second half of 2025."

IND • New P1/2 trial • Inherited Retinal Dystrophy

Opus Genetics…announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for OPGx-BEST1, a gene therapy for the treatment of bestrophin-1 (BEST1)-related IRD. - "With this IND clearance, Opus Genetics plans to initiate a Phase 1/2 clinical trial in the second half of 2025."

IND • New P1/2 trial • Macular Degeneration

Expected Growth Drivers in 2025 and Beyond (GlobeNewswire) - "Initial data from three pediatric patients treated with OPGx-LCA5 anticipated in Q3 2025; IND filing and initiation of a Phase 1/2 clinical trial for OPGx-BEST1 is planned for 2025, with preliminary data expected in Q1 2026; Topline data from the LYNX-2 pivotal Phase 3 trial evaluating Phentolamine Ophthalmic Solution 0.75% for visual loss in low light conditions associated with keratorefractive surgery are expected mid-year 2025; Topline data from the VEGA-3 pivotal Phase 3 clinical trial evaluating Phentolamine Ophthalmic Solution 0.75% for the treatment of presbyopia are expected in the first half of 2025."

Clinical data • IND • New P1/2 trial • Ophthalmology • Retinal Disorders

Opus Genetics Announces Presentations on Inherited Retinal Disease Programs at Medical Conferences in May (GlobeNewswire) - "In this rat model of retinal degeneration, subretinal administration of OPGx-MERTK (1E10 vg/eye) demonstrated effective preservation of both photoreceptors and retinal function....This preclinical study evaluated the efficacy of OPGx-MERTK in a Royal College of Surgeons (RCS) rat model, which exhibit a RPE phagocytosis defect due to a natural mutation in MERTK, following a single bilateral subretinal injection....Results from a preclinical study evaluating the safety and efficacy of OPGx-BEST1 in a canine model of BEST1 related IRD will be presented....Opus plans to commence a Phase 1/2 trial of OPGx-BEST1 in 2025 and aims to obtain preliminary data by Q1 2026."

New P1/2 trial • Preclinical • Macular Degeneration • Retinitis Pigmentosa