Ocuphire Pharma, Inc. (Nasdaq: OCUP), based in Farmington Hills, Michigan, USA announced the all-stock acquisition of Opus Genetics, Inc., a private ocular gene therapy company based in Raleigh, North Carolina. According to Ocuphire Pharma Inc., the merger creates “a transformative biotech company committed to being a leader in the development of gene therapies for the treatment of IRDs”. The combined company will be renamed Opus Genetics, Inc., effective October 23, 2024, and will trade on Nasdaq under the ticker symbol “IRD”, effective October 24, 2024.
Opus Genetics Inc., is supported by the Retinal Degeneration Fund (RD Fund), the venture arm of the Foundation Fighting Blindness aimed at rapidly driving research toward preventions, treatments and cures for the entire spectrum of retinal degenerative diseases. The company’s lead programs are licensed from the University of Pennsylvania and will focus on treatments to address mutations in genes that cause different forms of Leber congenital amaurosis (LCA). According to the company, “AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel manufacturing scale and efficiencies. The company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients”.
Following the announcement, George Magrath, M.D., who will serve as CEO of the combined company, stated that, “Opus Genetics has created a compelling pipeline of transformative therapies for patients with inherited retinal diseases, with promising early data. This is an opportunity to advance these treatments quickly, with four major clinical milestones on the horizon in 2025 for the combined company. We are encouraged by the new LCA5 six-month proof-of-concept data showing visual improvement in three out of three patients with advanced disease and are excited to bring together a leadership team with deep expertise in the development of potentially ground breaking gene therapies. We look forward to continuing our progress, creating value, and improving patient outcomes together.”
Figure 1. In a corporate presentation, issued from the company’s website, the combined company presented an expanded pipeline that includes multiple compelling assets from its adeno-associated virus (AAV)-based gene therapy portfolio, which is currently being developed for IRDs, as well as phentolamine ophthalmic solution 0.75%, currently being evaluated in presbyopia and dim (mesopic) light vision disturbances (sometimes referred to as DLD) after keratorefractive surgery.
In connection with the acquisition, Ocuphire issued 5.2 million shares of its common stock and 14,100 shares of its convertible preferred stock to existing stockholders of Opus Genetics. The shares of convertible preferred stock will be convertible into shares of common stock, subject to stockholder approval at the company’s annual meeting of stockholders, to be held in April 2025. Following the issuances, pre-acquisition stockholders of Ocuphire will own approximately 58% of the combined company’s fully diluted capitalization, and pre-acquisition stockholders of Opus Genetics will own approximately 42% of the combined company’s fully diluted capitalization.
The most advanced gene therapy candidate in Opus Genetics Inc., OPGx-LCA5, for the treatment of LCA5, is conducting an open-label, dose-escalation Phase 1/2 clinical trial. The trial has shown early clinical proof-of-concept, with new six-month data demonstrating visual improvement in three out of three adult patients participating in the trial, each of whom has late-stage disease. According to Jean Bennett, M.D., Ph.D., scientific co-founder of Opus Genetics, commented, “this level of efficacy in patients with late-stage disease is exciting and supportive of the potential for a one-time treatment with OPGx-LCA5, which could have a transformative impact on individuals who have experienced devastating vision loss and for whom no alternative treatment options exist”.
Figure 2. Three members of the leadership team of Opus Genetics Inc., provides commercial, investment and scientific expertise aimed to increase significant value through to 2026.