Pfizer and REGENXBIO Announce Licensing Agreement to Co-Develop Gene Therapy for Friedreich’s Ataxia

On July 31, 2019, VCBeat (WeChat ID: vcbeat) learned from foreign media reports that Pfizer announced a licensing agreement with REGENXBIO for gene therapy targeting Friedreich’s ataxia (FA). Under the licensing agreement, REGENXBIO granted Pfizer a non-exclusive global license to use the NAV AAV9 vector to develop and promote gene therapies for patients with ataxia.

REGENXBIO Inc. will receive an upfront payment, with the potential to receive subsequent payments based on development and commercialization milestones, as well as royalties on sales of products incorporating the company’s intellectual property.

Friedreich’s Ataxia (FA) is a common hereditary ataxia caused by mutations in the FXN gene, which restrict the production of frataxin. This leads to various debilitating symptoms and complications, including loss of coordination and balance, muscle weakness, visual impairment, hearing and speech disorders, scoliosis, diabetes, and cardiomyopathy. It affects approximately one in five people in the United States, and there is currently no effective treatment.

Founded in 2009 and headquartered in Maryland, USA, REGENXBIO Inc. is a clinical-stage biotechnology company dedicated to the development and commercialization of AAV (adeno-associated virus) gene therapies. REGENXBIO’s NAV Technology Platform is a proprietary AAV gene delivery platform comprising more than 100 novel AAV vectors, including AAV7, AAV8, AAV9, and AAVrh10. Zolgensma, Novartis’ FDA-approved therapy for spinal muscular atrophy (SMA) launched this year, utilizes REGENXBIO’s AAV vector technology.

REGENXBIO’s lead candidate is RGX-314, which primarily utilizes an AAV vector to deliver genes into cells for correcting genetic defects or producing therapeutic proteins within in vivo cells. All of REGENXBIO’s candidates are developed using its NAV Technology Platform. Currently, REGENXBIO is developing gene therapy candidates for the treatment of retinal, metabolic, and neurodegenerative diseases.

Earlier this month, RegenxBio expanded its product pipeline by leveraging its NAV vector platform to develop treatments for hereditary angioedema (HAE) and neurodegenerative disorders, including psoriasis. The HAE candidate utilizes the NAV vector to deliver a gene encoding a therapeutic antibody designed to target and bind plasma kallikrein, a dysregulated protein in patients with HAE.

Pfizer, founded in 1849 with a history spanning over 160 years, is headquartered in New York, USA. It is currently the world’s largest research-based biopharmaceutical company and a Fortune Global 500 enterprise. In its early days, Pfizer was primarily a chemical company focused on the production of chemical products, with pharmaceuticals, as a category of chemicals, also falling within its business scope.

Pfizer’s portfolio spans a broad and highly promising range of therapeutic and health areas, including small-molecule drugs, biologics, vaccines, and consumer healthcare products, with its R&D and manufacturing capabilities ranking among the world’s leading. Based on 2018 revenue, Pfizer topped the global pharmaceutical industry with $53.43 billion in sales.

“This license agreement further validates the strength of our intellectual property portfolio and the potential of NAV AAV9 in treating movement disorders and central nervous system conditions,” said Kenneth T. Mills, President and Chief Executive Officer of RegenxBio. “We are pleased to partner with Pfizer as they advance this program and develop a potential gene therapy for Friedreich’s ataxia.”

(Compiled by Cheng Tao)