RegenxBio and Pfizer Partner on Gene Therapy for Friedreich's Ataxia

Partnership handshake

REGENXBIO, based in Rockville, Maryland, announced it entered a license deal with Pfizer for a gene therapy for Friedreich’s ataxia, the most common hereditary ataxia.

Under the terms of the deal, RegenxBio granted Pfizer a non-exclusive global license, with rights to sublicense, RegenxBio’s NAV AAV9 vector. In return, Pfizer is paying RegenxBio an upfront payment, various milestones and royalties on net sales. No financial details were disclosed.

Patients with Friedreich’s ataxia (FA) have a genetic mutation in the FXN gene. This limits the creation of the frataxin protein, which leads to various debilitating symptoms and complications including coordination and balance problems, muscle weakness, impaired vision, hearing and speech, scoliosis, diabetes, and cardiomyopathy. It affects about one in 50,000 people in the U.S. There are currently no available treatments.

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“This license agreement further validates the strength of our intellectual property portfolio and the potential of NAV AAV9 for the treatment of systemic and CNS manifestations of movement disorders,” stated Kenneth T. Mills, president and chief executive officer of RegenxBio. “We are pleased to establish our relationship with Pfizer as they advance this program to develop a potential gene therapy treatment for Freidreich’s ataxia.”

Earlier this month, RegenxBio expanded its pipeline to include treatments for hereditary angioedema (HAE) and neurodegenerative diseases, including tauopathies, using NAV Vectors. The HAE product candidate uses NAV Vectors designed to deliver a gene for a therapeutic antibody targeting and binding to plasma kallikrein, a protein that is unregulated in HAE patients.

Also, at the same time, RegenxBio and Neurimmune AG announced an exclusive license, development and commercialization deal to discover and develop novel AAV gene therapies using NAV Vectors for chronic neurodegenerative diseases.

RegenxBio’s lead product candidate is RGX-314, made up of the NAV AAV8 vector encoding a gene for an antibody fragment that binds vascular endothelial growth factor (VEGF). It is being developed as a one-time subretinal therapy for wet age-related macular degeneration (wet AMD) and diabetic retinopathy.

On May 30, RegenxBio completed dosing for its Phase I/II clinical trial of RGX-314 in wet AMD. The trial is being conducted in eight retinal surgery centers in the U.S. and is designed to evaluate the safety and tolerability of RGX-314 as a one-time therapy for patients with wet AMD who already received anti-VEGF injections.

“Using NAV Vectors to deliver therapeutic antibodies has enormous potential for patients who lack treatment or who are currently underserved by existing therapies, and provides a significant opportunity to expand our pipeline through the application of our AAV-mediated antibody delivery capabilities and expertise to a number of validated and new targets in multiple therapeutic areas and tissues,” Mills stated at the time.

Of today’s deal, Seng Cheng, senior vice president and chief scientific officer of Pfizer’s Rare Disease Research Unit stated, “We are excited to partner with RegenxBio on the use of NAV AAV9 vector for the treatment of Friedreich’s ataxia, a condition with significant unmet medical need. We believe the AAV9 vector has the potential to have a profound impact on patients with severe and debilitating diseases where treatment options are limited today.”

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