Regenxbio and Clearside Biomedical announced an option and license agreement for exclusive worldwide rights to Clearside’s proprietary, in-office SCS Microinjector for the delivery of RGX-314 to the suprachoroidal space to treat wet age-related macular degeneration (AMD), diabetic retinopathy (DR), and other conditions for which anti-vascular endothelial growth factor (anti-VEGF) treatment is currently the standard of care.
Regenxbio plans to evaluate RGX-314 using Clearside’s SCS Microinjector for in-office, nonsurgical delivery into the suprachoroidal space, while continuing to advance its RGX-314 subretinal delivery program currently in development for wet AMD and DR.
Delivery of NAV AAV8-based gene therapy through the suprachoroidal space can potentially provide a targeted, in-office, nonsurgical approach to obtaining widespread transgene expression in the retina without exposing the vitreous and the anterior segment of the eye to the injected drug, according to a Regenxbio news release. Clearside’s patented SCS Microinjector is specifically designed to allow for consistent injection into the suprachoroidal space and has been tested in over 1,000 injections in clinical trials to date.
Under the terms of the agreement, Clearside has granted Regenxbio an option to receive an exclusive, worldwide commercial license, with rights to sublicense, to Clearside’s SCS Microinjector for the delivery of AAV gene therapies for the treatment of wet AMD, DR, and other conditions for which chronic anti-VEGF treatment is currently the standard of care. In return for these rights, Clearside will receive a fee upon Regenxbio’s exercise of its option, as well as up to $34 million in total development milestones across multiple indications, up to $102 million in sales milestones, and mid-single digit royalties on net sales of products using the SCS Microinjector. Regenxbio will be responsible for all development and commercialization activities for gene therapy product candidates. Clearside will be responsible for supplying the SCS Microinjector and supporting Regenxbio’s preclinical studies, clinical studies and commercial use.
“We are pleased to partner with Clearside in evaluating the use of the SCS Microinjector to deliver RGX-314 to the suprachoroidal space in the eye. We believe this approach can potentially allow for the treatment of an expanded population of patients with wet AMD and DR by providing access to gene therapy treatment in all settings of patient care,” Kenneth T. Mills, President and Chief Executive Officer of Regenxbio, said in the news release. “Delivery into the suprachoroidal space, which in clinical trials has demonstrated minimal associated procedural risks, may allow physicians to treat patients with diseases like DR earlier in the disease course with RGX-314. We will continue to advance the RGX-314 subretinal delivery clinical program, with a phase 2b clinical trial in wet AMD and a phase 2 clinical trial in DR expected to begin by the end of 2019, and we look forward to moving both delivery approaches through development as part of our deep commitment to patients.”
“Regenxbio is the ideal partner for us given its leadership, expertise and pioneering work in the development and manufacturing of AAV vectors for the delivery of anti-VEGF therapeutic antibodies in the ophthalmology space,” George Lasezkay, Pharm.D., JD, Chief Executive Officer of Clearside, said in the news release. “We are excited by the recent positive interim data reported by Regenxbio from the current RGX-314 phase 1/2a trial in wet AMD. This is an exciting time for us to collaborate with Regenxbio to evaluate the potential application of our proprietary in-office SCS Microinjector for AAV gene therapy.”