-Interim results from Phase II AAVIATE and ALTITUDE trials of dose level 3 cohorts with short-course prophylactic steroid eye drops resulted in zero cases of intraocular inflammation
-Initial safety results from Cohort 1 of the AFFINITY DUCHENNE study support well-tolerated profile to date; initial efficacy data to be presented at World Muscle Society meeting in October
-A new program in Duchenne with preclinical data using innovative science to produce near full-length wild-type dystrophin; expects to file IND to the FDA in the first half of 2025
-Detailed updates will be presented at its Virtual Investor Day, today at 8:30 a.m. ET
ROCKVILLE, Md., July 11, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) will announce today new interim data from the Phase II AAVIATE® and ALTITUDE® trials of ABBV-RGX-314 suprachoroidal delivery in wet AMD and diabetic retinopathy (DR). The company will also provide a comprehensive Duchenne program overview including initial safety data for RGX-202 and a new exon 53 program.
REGENXBIO will host a virtual Investor Day today at 8:30 a.m. ET to discuss these updates. The webcast and slides of the presentation can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com.
"We continue to perform at a high level as we execute on our mission of improving lives through the curative potential of gene therapy," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "Today, we are pleased to share an update on REGENXBIO's pipeline of AAV-based gene therapies with a focus on our retinal and Duchenne programs. The safety and tolerability data presented today for the high-dose cohorts of our in-office wet AMD and DR programs is encouraging and further validate the potential of the suprachoroidal space for AAV delivery to treat chronic retinal diseases."
Mr. Mills continued, "I am also thrilled to share an update on our Duchenne program, from our neuromuscular franchise, which includes progress on RGX-202 and our new exon-skipping program that we have initiated. We continue to develop highly differentiated, transformational treatments with an unwavering commitment to advance therapies designed to prevent ambulation loss and improve the quality of life in boys worldwide, regardless of age, who suffer from Duchenne."
"I believe we have a clear and definable path to achieve our '5 by '25' vision," said Mr. Mills. "We are advancing a strong pipeline of AAV-based therapeutic candidates in diseases representing large current market opportunities and that address significant unmet needs for millions of patients."
Key Investor Day Highlights
Retinal Program Update
ABBV-RGX-314 is an investigational one-time AAV therapeutic being developed in collaboration with AbbVie for the treatment of wet AMD, DR and other chronic retinal conditions. ABBV-RGX-314 uses the NAV® AAV8 vector to deliver a gene encoding therapeutic antibody fragment to inhibit vascular endothelial growth factor (VEGF).
Interim data to be presented today from the Phase II AAVIATE & ALTITUDE trials demonstrated that ABBV-RGX-314 suprachoroidal delivery administered to patients in cohorts at dose level 3 (1.0x1012 genome copies per eye) with short-course (seven-week) prophylactic topical steroid eye drops (N=39) resulted in zero cases of intraocular inflammation. Time of post-administration follow up ranged from six weeks to six months.
Duchenne Program Update
RGX-202 is an investigational one-time AAV therapeutic for Duchenne Muscular Dystrophy (Duchenne), using the NAV AAV8 vector to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain as well as a muscle-specific promoter to support a targeted therapy for improved resistance to muscle damage associated with Duchenne.
REGENXBIO will present interim safety data from Cohort 1 of the ongoing Phase I/II AFFINITY DUCHENNE™ trial, which continues to recruit patients (aged 4 to 11 years) and is using commercial-scale cGMP material from the REGENXBIO Manufacturing Innovation Center.
New Exon Skipping Program: REGENXBIO will also announce today the development of a potential one-time gene therapy for Duchenne, which is based on a novel exon-skipping construct. REGENXBIO scientists have demonstrated that AAV-mediated exon skipping by the expression of antisense ribonucleic acid (AS RNA) sequences has the potential to restore high and sustained levels of near full-length dystrophin in Duchenne patients with relevant mutations in the DMD gene.
Virtual Investor Day Webcast Information
The virtual event will take place on Tuesday, July 11, 2023, at 8:30 a.m. Eastern Time. The live webcast and presentation slides can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com. An archived replay of the webcast will be available in the Investors section of the website for approximately 30 days following the presentation.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8 and AAV9. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a "5x'25" strategy to progress five AAV Therapeutics from its internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.
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SOURCE REGENXBIO Inc.