Myrtelle Announces Positive Preliminary Clinical Data for Its Proprietary Gene Therapy in Canavan Disease

Myrtelle’s Phase 1/2 clinical trial introduces first-of-its-kind proprietary recombinant adeno-associated virus (rAAV) vector designed to enable targeting of oligodendrocytes

To date, 3 patients received gene therapy with the rAAV vector at a dose of 3.7 x 10¹³ vg delivered via intracerebroventricular administration

Available follow-up data in treated patients demonstrate favorable safety and tolerability with encouraging initial efficacy results

Excerpt from the Press Release:

WAKEFIELD, Mass.–(BUSINESS WIRE)–Myrtelle Inc. (“Myrtelle” or the “Company”), a gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that its recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy administered to 3 patients thus far in the Company’s open-label Phase 1/2 First-in-Human (FIH) clinical trial at Dayton Children’s Hospital (Dayton, Ohio) has shown favorable safety and tolerability with no drug-related adverse events. Myrtelle’s FIH trial utilizes the Company’s proprietary rAAV vector to directly target oligodendrocytes, the cells in the brain responsible for producing myelin – the insulating material that enables proper neuronal function. In Canavan disease (CD), the production of myelin is affected due to a mutation in the Aspartoacylase gene (ASPA) responsible for coding the enzyme Aspartoacylase (ASPA). The oligodendrocyte-targeting rAAV vector-based gene therapy is intended to restore ASPA function, thus enabling metabolism of N-Acetylaspartate (NAA), a neurochemical that is abundant in the brain, and thereby supporting myelination. Myrtelle entered into an exclusive worldwide licensing agreement with Pfizer Inc. in 2021 to develop and commercialize this novel gene therapy for the treatment of CD.

Per the FIH trial protocol, the gene therapy is administered as a single total dose of 3.7 x 10¹³ vg delivered by intracerebroventricular (ICV) injection to target the oligodendrocytes of the brain where ASPA activity needs to be restored for successful treatment of CD. “We are encouraged by the initial findings in the 3 patients treated thus far in this landmark gene therapy trial in Canavan disease. These initial patients are between 36 to 60 months old, representing the oldest study cohort, and have now been followed for at least 6 months post-treatment. The efficacy measures include Gross Motor Function Measurement (GMFM) and the Mullen Scales of Early Learning (MSEL) as well as relevant biomarkers such as NAA levels and white matter and myelin content. We are looking forward to treating additional patients and moving forward with the clinical development of this novel gene therapy,” said Christopher Janson, MD, Principal Investigator and Assistant Professor of Neuroscience at Wright State University Boonshoft School of Medicine and Director of Human Gene Therapy Center at Wright State Neuroscience Institute in Dayton, OH.

“All treated patients have exhibited favorable safety and tolerability to date. The gene therapy is administered via an intracerebroventricular route, and the post-procedure course has been in line with expected recovery in patients undergoing similar neurosurgical procedures. Based on the available data, the Data Monitoring Committee recommended opening the study to the younger cohorts: 15 to <36 months and 3 to <15 months old, as previously announced in January of this year,” said Robert Lober, MD, PhD, FAANS, Co-Principal Investigator and Associate Professor of Pediatrics at Wright State University Boonshoft School of Medicine and Attending Neurosurgeon at Dayton Children’s Hospital in Dayton, OH.

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