Myrtelle Announces Presentation of Encouraging Interim Results from its Phase 1/2 Gene Therapy Trial for Canavan Disease at the 2025 Cell & Gene Meeting on the Mesa

NEW YORK, Oct. 6, 2025 /PRNewswire/ -- Myrtelle Inc. ("Myrtelle" or the "Company"), a gene therapy company pioneering transformative treatments for neurodegenerative diseases, today announced that Dr. Michael Muhonen, M.D., Co-Chief Medical Officer, will present an overview of the Company and encouraging interim results from its Phase 1/2 clinical trial of the investigational gene therapy rAAV-Olig001-ASPA (MYR-101) for Canavan disease at the 2025 Cell & Gene Meeting on the Mesa, organized by the Alliance for Regenerative Medicine (ARM) in Phoenix, Arizona.

This presentation marks an important milestone in Myrtelle's mission to bring the first disease-modifying therapy to children living with Canavan disease—a devastating, fatal leukodystrophy caused by a single-gene defect. The results highlight the successful clinical translation of Myrtelle's oligodendrocyte-targeted AAV platform, designed to address the underlying cause of the disease by restoring myelin-forming cell function in the brain.

Key findings from the study, recently published in Nature Medicine, include:

• Well-tolerated therapy: MYR-101 demonstrated a favorable safety profile with no serious adverse events related to treatment.
• Evidence of biological activity: Significant reductions in N-acetylaspartate (NAA) levels in cerebrospinal fluid (CSF), consistent with restoration of ASPA enzyme function.
• New myelination: Increases in brain myelin volume measured by Synthetic MRI (SyMRI) confirm remyelination consistent with therapeutic benefit.
• Functional improvement: Participants demonstrated measurable developmental progress compared with historical controls, reflecting broad functional gains.
• Durable potential: Ongoing follow-up continues to show promising long-term outcomes and durability of effect.

"These data represent a major step forward for children and families affected by Canavan disease," said Dr. Michael Muhonen, Co-Chief Medical Officer of Myrtelle. "For the first time, we're seeing clear biological and functional evidence that gene therapy targeting oligodendrocytes can directly address the core pathology of this disease."

Regulatory Highlights

rAAV-Olig001-ASPA (MYR-101) was selected by the U.S. Food and Drug Administration (FDA) for inclusion in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program—one of only four gene therapies regulated by CBER to receive this distinction. The START program provides enhanced FDA engagement to accelerate the development of promising therapies for rare diseases.

In addition, MYR-101 has been granted the following designations:

• Regenerative Medicine Advanced Therapy (RMAT) designation
• Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the FDA
• Orphan Drug Designation and Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA)
• Innovative Licensing and Access Pathway (ILAP) designation from the UK Medicines and Healthcare products Regulatory Agency (MHRA)

About Myrtelle

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.

About Canavan Disease

Canavan disease (CD) is a fatal childhood genetic brain disease caused by mutations in the ASPA gene (ASPA) which prevent the normal expression of aspartoacylase, a critical enzyme produced in oligodendrocytes. The lack of normal aspartoacylase expression negatively impacts brain bioenergetics and development, including myelin production. 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 [email protected].

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 Myrtelle developing transformative treatments for neurodegenerative diseases. While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based in 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 ability to deliver the first approved therapy for Canavan disease; the program demonstrating safety and efficacy, as well as 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 Canavan disease, and the ability to continue its 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, our ability to pursue our regulatory strategy, our ability to advance ongoing partnering discussions, our ability to obtain regulatory approvals for commercialization of product candidates or to comply with ongoing regulatory requirements, our ability to develop strategic partnership opportunities and maintain collaborations, our ability to obtain or maintain the capital or grants necessary to fund our research and development activities, our ability to complete clinical trials on time and achieve desired results and benefits as expected, regulatory limitations relating to our ability to promote or commercialize our product candidates for specific indications, acceptance of our product candidates in the marketplace and the successful development.

SOURCE Myrtelle, Inc