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

Wakefield, Mass., October 4th, 2022 – 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.


Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program. For more information, please visit the Company’s website at: www.myrtellegtx.com.


Canavan disease (CD) is a fatal childhood genetic brain disease in which mutations in the Aspartoacylase gene (ASPA) prevent the normal expression of Aspartoacylase (ASPA), a critical enzyme produced in oligodendrocytes that breaks down the neurochemical N-Acetylaspartate (NAA). When not properly metabolized by oligodendrocytes, NAA accumulates in the brain and negatively affects bioenergetics, myelin production, and brain health. Patients with CD are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD, and only palliative treatments are available.

More information on Myrtelle’s clinical trial in Canavan disease can be found on https://clinicaltrials.gov/ under the identifier NCT04833907 or by emailing PatientAdvocacy@MyrtelleGTX.com.

Forward-Looking Statements

This press release contains forward-looking statements. Words such as “may,” “believe,” “will,” “expect,” “plan,” “anticipate,” “estimate,” “intend” and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. Forward-looking statements are based upon current estimates and assumptions and include statements regarding meeting with regulatory agencies to discuss the data and create a potential roadmap for registration and the rAAV-Olig001-ASPA as a potential treatment for patients with Canavan disease (CD). While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on information available to us on the date of this release. These forward-looking statements are subject to various risks and uncertainties, many of which are difficult to predict, that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, Myrtelle’s program demonstrating safety and efficacy, as well as future results that are consistent with prior results, the ability to generate the data needed for further development of this novel gene therapy in the patients with CD, and the ability to continue its clinical trials and to complete them on time and achieve the desired results. All forward-looking statements are based on Myrtelle’s expectations and assumptions as of the date of this press release. Actual results may differ materially from these forward-looking statements. Except as required by law, Myrtelle expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events, or otherwise.

Media Contact:
Jordana Holovach
Head of Communications and Community
Myrtelle Inc.
781-621-2797 Ext. 102