REGENXBIO Announces Dosing of First Patient in Cohort 3 of Phase I/II Trial of RGX-121 for the Treatment of MPS II (Hunter Syndrome)
ROCKVILLE, Md., April 14, 2021 /PRNewswire/ --
REGENXBIO Inc. (Nasdaq: RGNX) today announced that it dosed the first patient in Cohort 3 of the ongoing Phase I/II trial of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome, in patients up to five years old. RGX-121 is an investigational one-time gene therapy designed to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme using the AAV9 vector. RGX-121 is administered directly to the central nervous system (CNS).
"We are pleased with our continued progress in the Phase I/II trial of RGX-121 as we increase the dose level to further our understanding of the potential treatment effects, including potential systemic benefit for patients," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. "We are encouraged by the positive interim data we've reported previously from the first two cohorts, including signals of I2S enzyme activity in the CNS, continued neurocognitive development, and evidence of I2S enzyme activity in plasma and urine following administration of RGX-121. We look forward to further program updates later this year."
The Phase I/II trial of RGX-121 in patients with MPS II up to five years old is an open-label, dose escalation trial. RGX-121 is administered directly to the cerebrospinal fluid (CSF) and the study is designed to evaluate three dose levels, ranging from 1.3x1010 GC/g of brain mass to 2.0x1011 GC/g of brain mass. The primary endpoint of the study is safety and tolerability of RGX-121. Additional endpoints include the effect of RGX-121 on biomarkers of I2S enzyme activity in the CSF, serum and urine; neurocognitive development; and other outcome measures. The trial is being conducted at four leading clinical centers in the United States and Brazil.
RGX-121 is a product candidate for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome. RGX-121 is designed to use the AAV9 vector to deliver the human iduronate-2-sulfatase gene (IDS) which encodes the iduronate-2-sulfatase (I2S) enzyme to the central nervous system (CNS). Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted I2S beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS. RGX-121 has received orphan drug product, rare pediatric disease and Fast Track designations from the U.S. Food and Drug Administration.
About Mucopolysaccharidosis Type II (MPS II)
MPS II, or Hunter Syndrome, is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme iduronate-2-sulfatase (I2S) leading to an accumulation of glycosaminoglycans (GAG), including heparan sulfate (HS) in tissues which ultimately results in cell, tissue, and organ dysfunction. In severe forms of the disease, early developmental milestones may be met, but developmental delay is readily apparent by18 to 24 months. Specific treatment to address the neurological manifestations of MPS II and prevent or stabilize cognitive decline remains a significant unmet medical need. Key biomarkers of I2S enzymatic activity in MPS II patients include its substrate HS, which has been shown to correlate with neurocognitive manifestations of the disorder.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
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Company Codes: NASDAQ-NMS:RGNX