Regenxbio Inc. (RGNX), Tuesday announced positive results from the Phase I/II/III CAMPSIITE trial of RGX-121 for the treatment of patients with Mucopolysaccharidosis Type II, also known as Hunter syndrome, a rare, X-linked recessive disease.
The study found that RGX-121 led to a long-term, sustained reductions in CSF levels of HS D2S6, a key biomarker of brain disease in Mucopolysaccharidosis Type II, positioning RGX-121 as a potential first gene therapy and one-time treatment for the condition.
Further, the company noted that the long-term data from the trial showed that patients receiving RGX-121 at the pivotal dose level experienced an 85 percent median reduction of cerebrospinal fluid levels of heparan sulfate D2S6, with approaching normal levels and sustained for up to two years.
The biotechnology company is planning to initiate a rolling Biologics License Application submission using the accelerated approval pathway in the third quarter of 2024.
Currently, Regenxbio's stock is trading at $12.15, down 0.16 percent on the Nasdaq.
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