Opus Genetics Granted FDA Regenerative Medicine Advanced Therapy Designation for Gene Therapy Candidate

Opus Genetics, Inc. recently announced the US 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. The FDA’s decision indicates recognition of the strength of this initial data and the unmet need in this patient population.

“The FDA’s decision to grant RMAT designation to OPGx-LCA5 is a major milestone for the LCA5 patient community and a strong validation of our early clinical data,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “We’re encouraged by the potential of OPGx-LCA5 to meaningfully impact patients living with this ultra-rare and debilitating form of inherited blindness, and we look forward to continued collaboration with the FDA to accelerate its development.”

In addition to the RMAT designation, Opus has been invited to participate in the FDA’s Initial Comprehensive Multidisciplinary RMAT Meeting to support Opus’ development and manufacturing strategy. Lastly, Opus has been invited to join the FDA’s Chemistry, Manufacturing and Controls (CMC) Development and Readiness Pilot (CDRP) program, which provides additional guidance for accelerating CMC development of products under an investigational new drug application.

The RMAT designation program offers the potential for expedited development and review of regenerative medicine therapies that demonstrate the potential to address serious or life-threatening diseases based on preliminary clinical evidence. The designation provides sponsors with early interactions with the FDA, guidance on efficient development and manufacturing, and the opportunity to discuss surrogate endpoints to support accelerated approval.

OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial with Dr. Tomas Aleman at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.

Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company developing therapies to treat patients with inherited retinal diseases (IRDs) and other treatments for ophthalmic disorders. Our pipeline includes adeno-associated virus (AAV)-based investigational gene therapies that address mutations in genes that cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa. Our most advanced investigational gene therapy program, which has been granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA, is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein. OPGx-LCA5 is currently being evaluated in a Phase 1/2 open-label, dose-escalation trial, with encouraging early data. Our pipeline also includes BEST1 investigational gene therapy, designed to address mutations in the BEST1 gene, which is associated with retinal degeneration. The pipeline also includes Phentolamine Ophthalmic Solution 0.75%, a non-selective alpha-1 and alpha-2 adrenergic antagonist being investigated to reduce pupil size, and APX3330, a novel small-molecule inhibitor of Ref-1 being investigated to slow the progression of non-proliferative diabetic retinopathy. Phentolamine Ophthalmic Solution 0.75% is currently being evaluated in Phase 3 trials for presbyopia and dim (mesopic) light vision disturbances. We have reached agreement with the FDA under SPA for a Phase 3 trial to evaluate oral APX3330 for the treatment of DR. For  more information, visit www.opusgtx.com.