Myrtelle Completes Dosing of 8 Patients with Canavan Disease in Its Phase 1/2 Clinical Trial of the Investigational Gene Therapy rAAV-Olig001-ASPA

Eight patients have been treated in the ongoing Phase 1/2 First-in-Human clinical trial with favorable safety and tolerability observed to date

Assessments of the initial 3 patients reaching their six-month follow-up showed improvements on validated functional scales and increases in brain white matter and myelin content

Encouraging efficacy and safety data support further development of rAAV-Olig001-ASPA and discussions with regulatory authorities for a potential road map to registration

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 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.

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