Regenxbio (RGNX) Issues Update on NAV Gene Therapy for MPS I, MPS II

September 13, 2016 7:07 AM EDT
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REGENXBIO Inc. (Nasdaq: RGNX) provided an update on the recent publication of data from ongoing preclinical studies of NAV Gene Therapy for the treatment of Mucopolysaccharidosis Type I (MPS I) and Mucopolysaccharidosis Type II (MPS II).

“We are pleased to share additional positive preclinical results, which were generated by our development partners at the University of Pennsylvania, on the potential of our NAV Technology Platform in MPS I and MPS II,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “These results support our long-term clinical development plans and our mission of utilizing NAV gene therapies to improve the lives of patients suffering from severe neurodegenerative diseases.”

RGX-111 for the treatment of MPS I

Data from a preclinical study of RGX-111 for the treatment of MPS I in a canine model were published online in July 2016 in the journal Molecular Genetics and Metabolism. RGX-111 uses the NAV AAV9 vector to deliver the human α-l-iduronidase (IDUA) gene to the central nervous system (CNS).

The results demonstrate dose-dependent expression of IDUA and correction of disease pathology in the brain, as well as reduction in spinal cord compression after a single administration of NAV AAV9 vectors expressing IDUA. These data are expected to help establish the minimum effective dose for REGENXBIO’s planned first-in-human studies.

The study, titled “Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model,” is available online at: http://www.mgmjournal.com/article/S1096-7192(16)30105-6/abstract.

REGENXBIO expects to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) and a Clinical Trial Application (CTA) with Health Canada for a Phase I/II clinical trial of RGX-111 in the first half of 2017. RGX-111 has been granted both orphan drug designation and rare pediatric disease designation by the FDA.

RGX-121 for the treatment of MPS II

Data from a preclinical study of RGX-121 for the treatment of MPS II in a mouse model were published online in August 2016 in the journal Human Gene Therapy. RGX-121 uses the NAV AAV9 vector to deliver the human iduronate-2-sulfatase (IDS) gene to the CNS.

The results demonstrate dose-dependent expression of IDS and correction of disease pathology in the brain after a single administration of NAV AAV9 vectors expressing IDS. Levels of IDS in the brain tissue, cerebrospinal fluid (CSF), and serum all approached or exceeded normal levels. The treated mice also demonstrated improvement in long-term memory in a novel object recognition test, as well as evidence of correction of disease in the liver and heart.

The study, titled “Delivery of an adeno-associated virus vector into CSF attenuates central nervous system disease in mucopolysaccharidosis type II mice,” is available online at: http://online.liebertpub.com/doi/abs/10.1089/hum.2016.101.

REGENXBIO expects to submit an IND to the FDA for a Phase I/II clinical trial of RGX-121 in the first half of 2017. RGX-121 has been granted both orphan drug designation and rare pediatric disease designation by the FDA.



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