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REGENXBIO Announces Dose Escalation in AFFINITY DUCHENNE® Trial
First patient received dose level 2 of RGX-202, a potential one-time AAV Therapeutic for the treatment of Duchenne that includes an optimized transgene for a
About this update from Regenxbio Inc.
[{"type":"text","content":"First patient received dose level 2 of RGX-202, a potential one-time AAV Therapeutic for the treatment of Duchenne that includes an optimized transgene for a novel microdystrophin On track for pivotal dose determination and initiation of pivotal program in 2024 ROCKVILLE, Md., Nov. 29, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that the first patient received RGX-202 at dose level 2 in the Phase I/II AFFINITY DUCHENNE® trial. 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.\n\"Progressing to dose level 2 is an important milestone in our updated strategic plans and for accelerating the development of RGX-202,\" said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. \"There is a large unmet need for new therapies for boys with Duchenne, and the market is capable of supporting multiple gene therapies. We believe RGX-202 has unique, differentiating features that support its potential to be a best-in-class product.\"\nInitial biomarker data in two patients who received RGX-202 at dose level 1 who completed three-month assessment demonstrated robust RGX-202 microdystrophin expression with localization to the muscle cell membrane. In recently completed preclinical efficacy studies, RGX-202 at dose level 2 showed improvement in functional performance, compared to dose level 1, as determined by forelimb muscle strength and treadmill exhaustion in mdx mice.\nREGENXBIO expects to share initial strength and functional assessment data for both dose levels and anticipates pivotal dose determination and the initiation of a pivotal program in 2024. The Company plans to use RGX-202 microdystrophin expression as a surrogate endpoint to support a Biologics License Application (BLA) filing using the accelerated approval pathway.\nAFFINITY DUCHENNE Trial DesignThe Phase I/II AFFINITY DUCHENNE trial is a multicenter, open-label dose escalation and dose expansion clinical study to evaluate the safety, tolerability and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 i...