OPGx-LCA5 / Opus Genetics - LARVOL DELTA

Opus Genetics, Inc…announced the successful completion of a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the U.S. Food and Drug Administration (FDA) regarding OPGx-LCA5, its gene therapy candidate for Leber congenital amaurosis (LCA) caused by mutations in the LCA5 gene. (The Manila Times) - "Opus will incorporate the FDA’s feedback into its updated clinical development and CMC plans for the Phase 3 portion of the study to include enrolling as few as 8 participants in a single arm, 12-month study utilizing an adaptive design, which provides flexibility on endpoints and number of participants, reflective of LCA5 as a rare condition with an urgent medical need....Following availability of validated clinical drug supply manufactured with the intended commercial processes, dosing with OPGx-LCA5 is anticipated in the second half of 2026, with topline clinical data expected approximately one year later."

FDA event • New P3 trial • P3 data: top line • Ophthalmology

OPGx-LCA5 - Gene Therapy for Leber Congenital Amaurosis (LCA) (The Manila Times) - "Initial pediatric data at one-month post-treatment showed vision improvement with no drug-related adverse events; three-month pediatric data is expected to be reported in Q3 2025."

P1/2 data • Inherited Retinal Dystrophy

Recovery of Cone-Mediated Vision in Lebercilin-Associated Severe Retinal Ciliopathy (LCA5) after Gene Therapy: One Year Results of a Phase Ib/IIa Trial. (PubMed, Mol Ther) - P1/2 | "We assessed the preliminary safety of a recombinant adeno-associated virus serotype 8 vector carrying the native human LCA5 cDNA (OPGx-001) in LCA5-associated Leber congenital amaurosis (LCA5-LCA), a congenital blindness...Visual acuity returned to baseline or improved in the treated eyes of all participants. The favorable safety profile and efficacy outcomes pave the path for enrolling milder phenotypes with careful dose escalation."

Journal • P1/2 data • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) (clinicaltrials.gov) - P1/2 | N=15 | Recruiting | Sponsor: Opus Genetics, Inc | Trial completion date: Jan 2028 ➔ Jun 2028 | Trial primary completion date: Dec 2024 ➔ Jun 2028

Trial completion date • Trial primary completion date • Ophthalmology

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 Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for OPGx-LCA5 Gene Therapy Candidate (GlobeNewswire) - "Opus Genetics, Inc...announced today that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OPGx-LCA5, its investigational gene therapy for the treatment of Leber Congenital Amaurosis (LCA) due to genetic variations in the LCA5 gene....The RMAT designation for OPGx-LCA5 is based on early clinical evidence from Opus’s ongoing Phase 1/2 open-label, dose-escalation trial, which is evaluating the safety and potential efficacy of OPGx-LCA5 in patients with severe vision loss due to confirmed mutations in the LCA5 gene."

FDA event • Ophthalmology

Recovery of Cone-Mediated Vision in a Severe Ciliopathy after Gene Augmentation: One Year Results of a Phase I/II Trial for LCA5-LCA (ARVO 2025) - "Purpose To assess the safety and efficacy of an investigational subretinal delivery of a recombinant adeno-associated virus serotype 8 (AAV8) vector carrying the native human LCA5 cDNA (OPGx-001) in LCA5-LCA...Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

P1/2 data • Ophthalmology

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) (clinicaltrials.gov) - P1/2 | N=15 | Recruiting | Sponsor: Opus Genetics, Inc | Trial completion date: Sep 2027 ➔ Jan 2028 | Trial primary completion date: Aug 2024 ➔ Dec 2024

Trial completion date • Trial primary completion date • Ophthalmology

AAV8-hLCA5 Subretinal Delivery to the Macula in LCA5-associated Leber Congenital Amaurosis (LCA5-LCA): Preliminary Safety and Efficacy Results of an Ongoing Phase I/II Gene Therapy Trial (ARVO 2024) - "Administration of OPGx-001 by SR injection appears to be safe and well tolerated. There were subjective and objective signs of biologic efficacy even in these severely affected patients. Efficacy at lowest dose warrants careful dose escalation."

Clinical • Gene therapy • P1/2 data • Inherited Retinal Dystrophy • Ophthalmology

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) (clinicaltrials.gov) - P1/2 | N=9 | Recruiting | Sponsor: Opus Genetics, Inc | Not yet recruiting ➔ Recruiting | Initiation date: Jan 2023 ➔ Jun 2023

Enrollment open • Trial initiation date • Ophthalmology

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) (clinicaltrials.gov) - P1/2 | N=9 | Not yet recruiting | Sponsor: Opus Genetics, Inc | Trial completion date: Sep 2026 ➔ Sep 2027

Trial completion date • Ophthalmology

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) (clinicaltrials.gov) - P1/2 | N=6 | Not yet recruiting | Sponsor: Opus Genetics, Inc

New P1/2 trial • Ophthalmology