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Neurogene Initiates Natural History Study of Charcot-Marie Tooth Disease (CMT4J)

Key data now being collected to support endpoint assessment for future gene therapy clinical trial

NEW YORK, July 18, 2019 – Neurogene Inc., a company founded with a mission to bring life-changing medicines to patients and families affected by rare neurological diseases, today announced that it has enrolled the first patient in a natural history study of Charcot-Marie Tooth disease, type 4 (CMT4J), a rare inherited peripheral neuropathy. The goal of the study is to provide important data to support a future gene therapy clinical trial. This natural history study is being conducted under a collaboration between Neurogene and UT Southwestern Medical Center as one of multiple sponsored research agreements.

Charcot-Marie Tooth disease is a group of the most common inherited peripheral and sensory neuropathies caused by pathogenic changes in genes that affect peripheral nerve axons or the myelin sheath. CMT4J is a rare form of CMT caused by defects in the FIG4 gene that lead to uneven loss of myelin in sensory and motor nerve axons. Individuals with CMT4J typically develop symptoms of weakness and muscle atrophy in the upper and lower extremities in childhood. Early onset of the disease can be associated with a rapidly progressive course, ultimately leading to loss of ambulation, quadriplegia, respiratory compromise and premature death. Later disease onset has a more variable course that can lead to transient muscle weakness for some patients and rapid onset of symptoms for others.

“This is the world’s first prospectively designed natural history study specifically for CMT4J,” said Diana Castro, M.D., Assistant Professor at UT Southwestern Medical Center and lead investigator of the study. “With the help of the patients participating, this work will generate invaluable data to help us define optimal endpoints for a future planned gene therapy trial.”

“Patients with CMT4J and their families have been waiting for a therapy, and we view this study as a critical component of the clinical development plan to address that unmet need,” said Effie Albanis, M.D., Neurogene’s Chief Medical Officer. “We are taking a streamlined approach to clinical development to facilitate the fastest possible approval timeline for a future gene therapy medicine, consistent with our belief that patients should not be waiting longer than necessary for life-altering medicines.”

About the natural history study

Neurogene is sponsoring a longitudinal, prospective observational natural history and outcome measure discovery study into which approximately 20 patients with genetically confirmed CMT4J will be enrolled. Patients, who are currently being enrolled at UT Southwestern Medical Center, will undergo multiple assessments, including a series of Charcot-Marie Tooth disease measurements, magnetic resonance imaging (MRI), nerve conduction study and pulmonary function testing. Diana Castro, M.D., Assistant Professor at UT Southwestern Medical Center, is the lead investigator. For additional information about the trial, please visit www.clinicaltrials.gov, NCT03810508.

About Neurogene Inc.

Neurogene was founded to bring life-changing medicines to patients and families affected by rare neurological disorders. We partner with leading academic researchers, patient advocacy organizations and caregivers to bring to patients therapies that address the underlying genetic cause of a broad spectrum of neurological diseases where no effective treatment options exist today. Our lead programs use AAV-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Neurogene is also investing in novel technology to develop treatments for diseases not well served by gene therapy. For more information, visit www.neurogene.com

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