Shares of REGENXBIO surged on Wednesday following the announcement that one of its trials had successfully reached its primary endpoint, while another trial showed promising early results.

Promising Biomarker Data for Duchenne Muscular Dystrophy Treatment

The Rockville, Md.-based biotechnology company revealed that recent biomarker data from its trial for a potential treatment of Duchenne muscular dystrophy displayed a significant clinical improvement. Specifically, all three patients who received dose level one of RGX-202 demonstrated increased levels of RGX-202 microdystrophin and lower levels of serum creatinine kinase. These biomarkers indicate protection against muscle degradation and reduced muscle injury, respectively, both of which are associated with Duchenne muscular dystrophy.

The early results align with previous evidence of clinical improvement and show that the drug RGX-202 has been well tolerated as of February 6, 2024. The company expects to determine the optimal dosage for RGX-202 by mid-2024.

Positive Outcome for Hunter Syndrome Treatment Trial

In addition to the favorable results in the Duchenne muscular dystrophy trial, REGENXBIO announced at midday on Wednesday that its trial for a Hunter syndrome treatment had also achieved its primary endpoint. As a result, the company plans to submit a Biologics License Application to the U.S. Food and Drug Administration later this year.

These recent developments have contributed to a 10.4% increase in REGENXBIO's stock, reaching a value of $14.41 in afternoon trading. Despite this positive news, it is important to note that shares are still down by 20% since the beginning of the year.

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