Blog Exposure – FDA Granted Fast Track Designation for REGENXBIO’s RGX-121 Gene Therapy for Mucopolysaccharidosis Type II

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LONDON, UK / ACCESSWIRE / May 4, 2018 / If you want access to our free research report on REGENXBIO Inc. (NASDAQ: RGNX) all you need to do is sign up now by clicking the following link www.active-investors.com/registration-sg/?symbol=RGNX as the Company’s latest news hit the wire. On May 2, 2018, the Company announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for RGX-121, a novel, one-time investigational treatment for Mucopolysaccharidosis Type II (MPS II). Register today and get access to over 1,000 Free Research Reports by joining our site below:

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REGENXBIO will Begin Phase-I/II Trial in the Coming Months

Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO, stated that Children living with MPS II have limited treatment options, making this Fast Track designation tremendously important.

He added that the Company is honored to support the MPS community and committed to offering innovative solutions to people with MPS II and their families.

Kenneth further said that REGENXBIO looks forward to working closely with the FDA to facilitate the development of RGX-121 and will begin the Phase-I/II trial in the coming months.

Primary Endpoint of Phase-I/II Trial is to Assess Safety & Tolerability of RGX-121 at 24 Weeks

RGX-121 will be evaluated in a Phase-I/II, multi-center, open-label, multiple-cohort, dose-escalation study in pediatric subjects with MPS II. Eligible patients with documented evidence of early-stage neurocognitive deficit due to MPS will be enrolled. Around six male subjects with MPS II greater than or equal to four months old and less than five years old will be treated in two dose cohorts and will receive a single dose of RGX-121 administered by an injection directly in the cerebrospinal fluid (CSF). Patients will receive immunosuppression for the first year after RGX-121 is administered. The primary endpoint of the clinical study is to assess the safety and tolerability of RGX-121 at 24 weeks. The secondary and exploratory endpoints include the effect of RGX-121 on biomarkers of I2S activity in the CSF, serum and urine and effect of RGX-121 on neurocognitive deficits as well as other outcome measures. Following completion of the primary study period, subjects will continue to be assessed for a total of 104 weeks following treatment with RGX-121 and then be asked to participate in a long-term follow-up.

The Company is likely to initiate patient recruitment and dosing in mid-2018. Leading international gene therapy and lysosomal storage disease centers will participate in the Phase-I/II clinical trial for RGX-121 for the treatment of MPS II.

FDA Granted Rare Pediatric Disease Designation to RGX-121 for Treatment of MPS II

In August 2016, the FDA granted Rare Pediatric Disease Designation to RGX-121 for the treatment of MPS II. Rare Pediatric Disease Designation was built upon the Orphan Drug Designation granted to RGX-121 at the end of 2015 by the FDA. The FDA defines a “rare pediatric disease” as a disease that affects fewer than 200,000 individuals in the US, primarily aged from birth to 18 years.

About Mucopolysaccharidosis Type II (MPS II)

MPS II, also known as Hunter syndrome, is a rare genetic disorder caused by a mutation in the gene that is responsible for making the enzyme iduronate-2-sulfatase (IDS). IDS is needed to break down long chains of sugar molecules known as mucopolysaccharides. These sugar molecules are cellular waste products. As they build up in tissues, like the central nervous system (CNS), they cause progressive damage to these tissues and to many organs in the body, including the brain. MPS II is characterized by distinctive facial features, a large head, hydrocephalus, enlargement of the liver and spleen, umbilical or inguinal hernia, and hearing loss. Individuals with this condition may additionally have joint deformities and heart abnormalities involving the valves.

About RGX-121

RGX-121 is our product candidate for the treatment of MPS II, which is designed to use the AAV9 vector to deliver the human IDS gene to the CNS. Delivery of the enzyme that is deficient 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. This strategy could also provide rapid I2S delivery to the brain, potentially preventing the progression of cognitive deficits that otherwise occurs in MPS II patients.

About REGENXBIO Inc.

Founded in 2008 and headquartered in Rockville, Maryland, REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno‐associated virus (AAV) gene therapy. REGENXBIO’s NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors.

Stock Performance Snapshot

May 3, 2018 – At Thursday’s closing bell, REGENXBIO’s stock dropped 2.25%, ending the trading session at $39.10.

Volume traded for the day: 261.16 thousand shares.

Stock performance in the last month – up 30.99%; previous three-month period – up 50.97%; past twelve-month period – up 103.65%; and year-to-date – up 17.59%

After yesterday’s close, REGENXBIO’s market cap was at $1.21 billion.

The stock is part of the Healthcare sector, categorized under the Biotechnology industry.

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