WAKEFIELD, Mass.– Myrtelle Inc. (“Myrtelle” or the “Company”), a gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced updated initial findings of the 8 patients treated with the Company’s recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy for open-label Phase 1/2 First-in-Human (FIH) clinical trial for Canavan disease (CD), a fatal genetic disorder in children. Early data from the study, being conducted at Dayton Children’s Hospital (Dayton, Ohio), at three months following gene therapy treatment have shown encouraging results to date.
Assessments for all 8 patients through their three-month follow-up visits have shown increases in white matter, grey matter, and total brain volumes within three months and reduction in the volume of cerebrospinal fluid (CSF) in most patients by magnetic resonance imaging (MRI). Additionally, clinical measurements of motor and cognitive function using the Gross Motor Function Measure (GMFM) and Mullen Scales of Early Learning (MSEL) tools have demonstrated improvements across several domains. The observed improvements following gene therapy are in contrast to the continuous clinical decline expected for untreated CD patients. The first three patients treated with the gene therapy in cohort one are now at least 18 months post therapy. As announced previously, assessments of these initial three patients at later time points also showed improvements on imaging and validated functional scale measurements. No serious drug-related adverse events have been observed in treated patients to date.
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, normal brain development is impaired due to a mutation in the gene that encodes the enzyme Aspartoacylase (ASPA). The deficiency of ASPA enzyme results in multiple biochemical and anatomic changes, including the inability to metabolize N-Acetylaspartate (NAA) which in turn causes abnormal accumulation of NAA and deficiency of downstream metabolites, leading to impaired bioenergetics and abnormal brain development. The oligodendrocyte-targeting rAAV vector-based gene therapy is intended to restore ASPA function and hence the metabolism of NAA and brain development in patients with CD.
“The early results to date in patients treated in Myrtelle’s Phase 1/2 clinical trial are encouraging. The available evidence suggests the delivered gene therapy is, within three months, rapidly eliciting its intended effect. Given these data, we are encouraged to continue tracking these treated patients and to advance development of the candidate gene therapy to bring this potential treatment options to CD patients,” commented Rob Lober, MD, Principal Investigator on the Phase 1/2 study and Attending Neurosurgeon at Dayton Children’s Hospital and Associate Professor of Pediatrics at Wright State University Boonshoft School of Medicine.
Myrtelle has previously announced that rAAV-Olig001-ASPA has received Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the US Food and Drug Administration as well as Orphan Drug Designation and Advanced Therapeutic Medicinal Product classification from the European Medicines Agency.
