Ocuphire Pharma Acquires Opus Genetics, Expanding Gene Therapy Pipeline for Inherited Retinal Diseases
核心洞察
Ocuphire Pharma has acquired Opus Genetics in an all-stock transaction, with the combined company renamed Opus Genetics, Inc., focusing on inherited retinal diseases (搜索).
The acquisition expands the pipeline to include AAV-based gene therapies for IRDs and phentolamine ophthalmic solution for presbyopia (搜索), while seeking a partner for diabetic retinopathy (搜索) program.
OPGx-LCA5, a gene therapy for LCA5 (搜索), shows promising early Phase 1/2 data with visual improvements in patients, and pediatric enrollment is expected in early 2025.
Ocuphire Pharma, Inc. has finalized the acquisition of Opus Genetics, Inc. in an all-stock transaction. Effective October 23, 2024, the combined entity will operate under the name Opus Genetics, Inc., trading on Nasdaq under the ticker symbol “IRD” starting October 24, 2024. This strategic move aims to bolster the development of transformative therapies for inherited retinal diseases (搜索) (IRDs).
Acquisition Terms and Leadership
Under the terms of the acquisition, Ocuphire issued 5.2 million shares of its common stock and 14.1 thousand shares of its convertible preferred stock to Opus Genetics' existing stockholders. Post-issuance, Ocuphire's pre-acquisition stockholders will hold approximately 58% of the combined company's fully diluted capitalization, while Opus Genetics' pre-acquisition stockholders will own approximately 42%. George Magrath, MD, will continue as CEO, emphasizing the potential of Opus Genetics' pipeline for IRDs, citing promising early data and anticipating four major clinical milestones in 2025. Ben Yerxa, PhD, former President and CEO of Opus Genetics, will assume the role of President of the combined company.
Expanded Pipeline and Strategic Focus
The acquisition significantly broadens the combined company's therapeutic pipeline. Key assets include adeno-associated virus (AAV)-based gene therapies targeting various IRDs and phentolamine ophthalmic solution 0.75%, currently under evaluation for presbyopia (搜索) and dim light vision disturbances following keratorefractive surgery. Given the resource demands and timelines associated with APX3330, an oral small-molecule inhibitor of Ref-1 (搜索) for non-proliferative diabetic retinopathy (搜索) (NPDR), the company intends to seek a strategic partner to further its clinical development, reallocating resources to the newly acquired gene therapy programs.
OPGx-LCA5: Advancing Gene Therapy for LCA5
The most advanced gene therapy candidate, OPGx-LCA5, is designed to treat LCA5 (搜索), an early-onset retinal degeneration. An ongoing open-label, dose-escalation Phase 1/2 clinical trial has demonstrated early clinical proof-of-concept. Recent six-month data revealed visual improvements in all three adult patients with late-stage disease participating in the trial. Jean Bennett, MD, PhD, scientific co-founder of Opus Genetics, highlighted the potential transformative impact of OPGx-LCA5, particularly for individuals with significant vision loss and limited treatment options.
Enrollment of pediatric patients in the Phase 1/2 trial is slated for the first quarter of 2025, with initial data expected in the third quarter of 2025. OPGx-LCA5 has been granted Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA, potentially qualifying it for a priority review voucher upon BLA approval.
Anticipated Milestones and Board Appointments
The combined company's extended cash runway into 2026 is expected to support the achievement of several clinical milestones. These include data readouts from pediatric patients in the OPGx-LCA5 Phase 1/2 trial, the first patients in the OPGx-BEST1 Phase 1/2 trial, and from the ongoing LYNX-2 and VEGA-3 Phase 3 trials. The LYNX-2 trial targets patients with decreased visual acuity in low-light conditions post-keratorefractive surgery, while the VEGA-3 trial addresses presbyopia (搜索). Top-line results from these trials are anticipated in early and mid-2025, respectively.
Jean Bennett, Ben Yerxa, and Adrienne Graves, former members of the Opus Genetics Board of Directors, will join the Board of the newly combined company, bringing extensive expertise in gene therapy and ophthalmic drug development.