After receiving favorable data regarding the efficacy of its clinical research, REGENXBIO Inc. (NASDAQ: RGNX) witnessed a significant gain in value, with its shares soaring by 15.17% to $24.60. RGNX today reported early findings from the Phase I/II AFFINITY DUCHENNE research of RGX-202, a potential treatment for Duchenne muscular dystrophy (Duchenne), in children ages 4 to 11.
The latest data revealed notable microdystrophin expression from dose level 2 of RGX-202, particularly showcasing a substantial increase in a 12-year-old patient. This marks a positive step forward, especially given the scarcity of data for boys older than 7 years, emphasizing the company’s commitment to transparency and addressing the urgent need for effective treatments in the Duchenne community.
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Moreover, the safety profile of RGX-202 appears encouraging, with no drug-related serious adverse events reported among the patients across both dose levels. Initial observations from caregivers indicate improvements in strength and motor function among boys treated with RGX-202, further bolstering its potential as a viable therapeutic option.
Noteworthy reductions in serum creatinine kinase (CK) levels, an indicator of muscle injury, were observed in patients who completed the trial assessments, providing supporting evidence of clinical enhancement. These findings were corroborated through meticulous measurement methods, ensuring reliability and accuracy in assessing RGX-202’s efficacy.
Additionally, clinic assessments and home videos shared by caregivers illustrate promising signs of functional improvement among patients treated with RGX-202, underscoring its impact on enhancing everyday activities for individuals with Duchenne. REGENXBIO expects to start a pivotal trial in the second half of 2024 and hopes to determine a key dose by the middle of the year.
Through the accelerated approval pathway, RGNX hopes to expedite the regulatory process for prospective market entry by using RGX-202 microdystrophin expression as a surrogate endpoint to support a Biologics License Application (BLA) submission. A major step forward in the development of RGX-202 as a possible treatment for Duchenne muscular dystrophy has been made with the encouraging interim results from the AFFINITY DUCHENNE study.
