REGENXBIO Inc. (Nasdaq: RGNX) celebrated a major milestone as the first patient received RGX-202 at dose level 2 in the Phase I/II AFFINITY DUCHENNE® trial. This trial is investigating RGX-202, a novel one-time AAV Therapeutic designed for Duchenne muscular dystrophy (Duchenne). The NAV® AAV8 vector is employed to transport a transgene for a unique microdystrophin, which includes the functional elements of the C-Terminal (CT) domain and a muscle-specific promoter. The objective is to provide a targeted therapy that enhances resistance to muscle damage associated with Duchenne.

REGENXBIO, highlighted the significance of advancing to dose level 2 as a crucial milestone in their strategic plans for expediting RGX-202 development. He emphasized the substantial unmet need for new Duchenne therapies and expressed confidence in RGX-202's potential to be a best-in-class product due to its unique features.

Preliminary biomarker data from two patients who received RGX-202 at dose level 1 revealed robust microdystrophin expression localized to the muscle cell membrane. Preclinical efficacy studies at dose level 2 demonstrated improved functional performance compared to dose level 1 in mdx mice, as indicated by enhanced forelimb muscle strength and treadmill exhaustion.

REGENXBIO plans to share initial strength and functional assessment data for both dose levels and anticipates pivotal dose determination, initiating a pivotal program in 2024. The company aims to leverage RGX-202 microdystrophin expression as a surrogate endpoint for a Biologics License Application (BLA) filing through the accelerated approval pathway.

The AFFINITY DUCHENNE trial, designed in consultation with the Duchenne community and key opinion leaders, is a multicenter, open-label study assessing the safety, tolerability, and clinical efficacy of a one-time intravenous dose of RGX-202 in Duchenne patients. The trial incorporates a short-term immunosuppression regimen to proactively address potential immunologic responses. Trial endpoints include safety assessments, pharmacodynamic and pharmacokinetic measures of RGX-202, and strength and functional assessments.

Duchenne muscular dystrophy is a severe, progressive muscle disease affecting a significant number of boys globally. It is caused by mutations in the Duchenne gene, leading to the absence of dystrophin, a critical protein for muscle structure and signaling pathways. RGX-202, with its innovative design and delivery system, aims to address this debilitating condition and potentially offer a breakthrough therapeutic option.

 

 

Source: prnewswire.com

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