RegenxBio Inc. (NASDAQ:RGNX), Rockville, Maryland, USA, has announced the successful raising of $75.9 million through the sale of 3.7 million shares at $20.50/share in a follow-on funding underwritten by Morgan Stanley, Bank of America, Merrill Lynch, Piper Jaffray and Chardan Capital Markets. The clinical stage company claims to control a broad stable of international intellectual property filings focused on viral vector gene delivery, including associated manufacturing proprietary technologies. One of the company’s lead products, RGX-134, represents a “novel, one-time subretinal treatment for wet AMD”, which aims to overcome the current treatment regimes of regular anti-VEGF injections of approved products such as Lucentis and Eylea.
Earlier in the year the company announced the launch of a phase I clinical trial entitled, “Open-label, Multiple-cohort, Dose-escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With RGX-314 in Subjects With Neovascular AMD (nAMD).” The study will be run in six US retinal surgery clinics with an interim trial update expected before the end of 2017. The company will use its “novel adeno-associated virus”, (NAV), delivery technology which it claims provides higher gene expression and longer lasting sustained expression, thereby facilitating the treatment of wet AMD with a “one-time sub-retinal treatment”. The initiative is under development in collaboration with a number of gene therapy research pioneers including James M. Wilson, M.D., Ph.D., Jean Bennett, M.D., Ph.D. and Albert Maguire, M.D., all from the University of Pennsylvania’s Gene Therapy Program and Center for Advanced Retinal and Ocular Therapeutics (Penn), and Peter Campochiaro, M.D. from the Johns Hopkins Wilmer Eye Institute. The clinical study is understood to include an estimated eighteen (18) previously treated wet AMD patients, over the age of fifty (50) years and responsive to anti-VEGF therapy. Three different doses of RGX-314 will be evaluated: 3 × 10^9 genome copies (GC)/eye, 1 × 10^10 GC/eye, and 6 × 10^10 GC/eye. The primary endpoints for the study include BCVA, SD-OCT, immunological parameters, ocular examinations and the recording of adverse events. The timeline for the evaluation of safety and tolerability is set at 24 weeks following a single dose of the experimental therapeutic administered by subretinal delivery. Long term follow up is scheduled to continue until week 106 in order to capture any long term issues regarding safety and durability of effect.
Commenting on the milestone, Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO stated, “The goal of the RGX-314 program is to develop a single-dose treatment for wet AMD that prevents future disease recurrence while reducing or eliminating the need for regular injections that are the current standard of care in wet AMD. We are on track to meet our next program objectives for RGX-314, beginning with trial enrollment by mid-2017 and an interim trial update by the end of the year, and we look forward to working with leading U.S. researchers and retina surgeons on this novel clinical program.”