OPGx-BEST1 / Opus Genetics - LARVOL DELTA

Opus Genetics…provided updates on its ongoing OPGx-BEST1 Phase 1/2 clinical trial (BIRD-1) targeting BEST-1 associated IRDs (GlobeNewswire) - "Opus expects to announce three-month topline data from Cohort 1 of the Phase 1/2 trial during the second week of September 2026, assuming all participants complete their assessments as scheduled. In addition, the Company plans to present the data at the annual EURETINA Congress taking place in Vienna, Austria from October 1 - 4, 2026. BIRD-1 is an adaptive, open-label, Phase 1/2 study evaluating the safety and efficacy of single-eye subretinal administration of OPGx-BEST1 in adult participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)."

P1/2 data • Inherited Retinal Dystrophy

Evaluation of the Toxicity and Efficacy of an Adeno-Associated Viral Vector Expressing BEST1 Delivered by Subretinal Injection in a Canine Model of Human Bestrophinopathy. (PubMed, Hum Gene Ther) - "Noninvasive retinal imaging by optical coherence tomography showed improved structural integrity with a reduction or prevention of appearance of vitelliform lesions and reversal of microdetachments in the retinal areas treated with OPGx-BEST1. These results support the use of OPGx-BEST1 in clinical studies with patients affected with bestrophinopathies and define the no-observed-adverse-effect level at 4.5 × 1010 vg/eye (0.15 mL, 3.0 × 1011 vg/mL)."

Journal • Preclinical • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Retinal Disorders

OPGx-BEST1 (The Manila Times) - "In the ongoing Phase 1/2 clinical trial, three BVMD and two ARB participants were enrolled in the first cohort, with dosing completed in May 2026....Three-month topline data from Cohort 1 is expected in September 2026."

P1/2 data • Trial status • Retinal Disorders

Therapeutic Platform for BEST1-Associated Blindness: Anticodon-Engineered tRNA and Gene Augmentation (ASGCT 2026) - "To evaluate the broader applicability of this approach across mutations, we prioritized ⁓25 most prevalent autosomal dominant BEST1 mutations across the USA, Europe, China and the UK for clinical development of OPGx-BEST1, an AAV-based gene therapy expressing full length human BEST1 cDNA...In parallel, ACE-tRNA-mediated nonsense suppression offers a precise, personalized strategy by enabling insertion of WT amino acids at premature termination codons. Although further development is needed before clinical evaluation, ACE-tRNA offers a scalable, versatile, and clinically relevant alternative to gene-specific therapies for nonsense-mutation-driven diseases."

Age-related Macular Degeneration • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology

Preliminary results from adult participants in a Phase 1b/2a clinical study of OPGx-BEST1 gene therapy for ARB and BVMD due to BEST1 mutations (ARVO 2026) - P1/2 | "Conclusions OPGx-BEST1 is a targeted gene augmentation approach to restore RPE function and address the genetic cause of BEST1-related disease. Preliminary results will offer early insights into safety, tolerability, and potential biological activity in ARB or BVMD and help guide dose selection and future clinical development for this otherwise untreatable IRD."

Clinical • Gene therapy • P1/2 data • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

Development of cell-based expression and functional potency assays for OPGx-BEST1 gene therapy (ARVO 2026) - "The results showed that the therapy works within the expected range and meets important safety and quality standards. This is an important step toward bringing a safe and effective treatment to people living with Bestrophinopathies."

Gene therapy • Inherited Retinal Dystrophy • Ophthalmology

Opus Genetics Completes Enrollment in Cohort 1 of Phase 1/2 OPGx-BEST1 Gene Therapy Study (Opus Genetics Press Release) - "In a session today at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, the study’s principal investigator, Mark Pennesi, M.D., Ph.D., Chief Medical Officer and Director, Inherited Retinal Degeneration Clinic, Retina Foundation of the Southwest, presented baseline demographics of Cohort 1 (summarized in Table 1) and 3-month results from the first (sentinel) participant treated in the study, highlighting positive tolerability and biological activity following subretinal administration of OPGx-BEST1...Opus expects to announce 3-month topline data from Cohort 1 in September 2026, followed by the presentation of data at an ophthalmology medical conference later this year. "

P1/2 data • Macular Degeneration

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