WAKEFIELD, Mass.– Myrtelle Inc. (“Myrtelle” or the “Company”), a gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced the completion of dosing of 8 patients with the Company’s recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy for Canavan disease (CD). The Company’s open-label Phase 1/2 First-in-Human (FIH) clinical trial conducted at Dayton Children’s Hospital (Dayton, Ohio) has shown encouraging early efficacy data and favorable safety and tolerability to date with no drug related adverse events in the treated patients.
Myrtelle’s FIH trial utilizes the Company’s proprietary rAAV vector to directly target oligodendrocytes, the brain cells affected in CD which are responsible for producing myelin – the insulating material that enables proper neuronal function. In CD, the production of myelin is impaired due to a mutation in the Aspartoacylase gene (ASPA) that encodes the enzyme Aspartoacylase (ASPA). The deficiency of ASPA enzyme results in multiple biochemical and anatomic changes, including inability to metabolize N-Acetylaspartate (NAA), a neurochemical that is abundant in the brain and plays an important role in myelin synthesis and brain bioenergetics. The oligodendrocyte targeting rAAV vector-based gene therapy is intended to restore ASPA function and hence the metabolism of NAA and myelination in patients with CD.
Results from the initial 3 patients treated in the Phase 1/2 trial and for whom data are available for at least six months have shown improvements on validated functional scales, including the Gross Motor Function Measure (GMFM) and the Mullen Scales of Early Learning (MSEL). Additionally, observations of these patients at 6 months post-treatment using Magnetic Resonance Imaging (MRI) demonstrated increases in brain white matter and myelin content. Observed improvements in these treated patients are in contrast to the continuous clinical deterioration expected with the natural progression of CD. An update with additional safety and efficacy data on the Phase 1/2 clinical trial will be included in a presentation by Armen Asatryan, MD, MPH, Chief Medical Officer of Myrtelle, at the October 2022 Meeting on the Mesa conference being held by the Association for Regenerative Medicine in Carlsbad, CA.
When additional data from all 8 of these treated patients are available, the Company intends to meet with regulatory agencies to discuss the data and create a potential roadmap for registration. “Encouraged by the efficacy and safety results to date, we plan to meet with regulatory authorities to discuss advancing this therapy into the final phase of clinical development with the ultimate goal of seeking approval. Given the progressive nature of Canavan disease and the data from the ongoing trial, we are driven to continue our work to bring this potentially important therapy to the patients who currently do not have treatment options,” commented Armen Asatryan, MD, MPH, Chief Medical Officer of Myrtelle.
Myrtelle recently announced that rAAV-Olig001-ASPA received Advanced Therapeutic Medicinal Product (ATMP) classification, specifically, a Gene Therapy Medicinal Product (GTMP), from the European Medicines Agency (EMA). This classification follows the US Orphan Drug, Rare Pediatric Disease, and Fast Track designations by the FDA which support the Company’s mission to provide treatments for patients with CD.
