Myrtelle Announces Presentation at Hydrocephalus Association & Rudi Schulte Research Workshop on Developing Non-Invasive Hydrocephalus Therapies: Molecular and Cellular Targets

WAKEFIELD, Mass.–(BUSINESS WIRE)–#aav—Myrtelle Inc. (“Myrtelle” or the “Company”), a gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that Christopher Janson, M.D., Principal Investigator on Myrtelle’s Canavan Disease Gene Therapy Trial and Associate Professor of Neuroscience & Founding Director of Human Gene Therapy Center at Premier Health Wright State Neuroscience Institute in Dayton, Ohio, will deliver a presentation on the Company’s open-label Phase 1/2 First-in-Human (FIH) clinical trial for Canavan disease using the Company’s recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy entitled “Canavan Disease as a Prototype for Gene Therapy of Hydrocephalus Advancing Translational Sciences” on Friday, September 29, 2023, at the upcoming Hydrocephalus Association & Rudi Schulte Research Workshop on Developing Non-Invasive Hydrocephalus Therapies: Molecular and Cellular Targets in Dallas, Texas.

The presentation will review encouraging post-treatment improvements in representative cases from the Phase 1/2 clinical study in multiple anatomic and biomarker measurements by magnetic resonance imaging (MRI), including myelin, white matter and extra-parenchymal water. These improvements in treated patients contrast the deterioration in untreated age-matched CD patients based on natural history. MRI-based improvements are correlated with improvements in motor and cognitive function in treated patients, which will be presented at upcoming conferences.

Myrtelle’s FIH trial utilizes the Company’s proprietary rAAV vector to directly target oligodendrocytes, the brain cells affected in CD that 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 ASPA gene that encodes the enzyme aspartoacylase. The lack of normal aspartoacylase activity negatively impacts brain bioenergetics and development, including myelin production. The oligodendrocyte-targeting rAAV vector-based gene therapy is intended to restore ASPA function and brain development in patients with CD.

“The improvements observed on MRI imaging in patients are encouraging evidence of underlying recovery of brain anatomy following gene therapy treatment which we believe is at the root of the functional advances being made by treated patients,” said Dr. Janson. “We look forward to sharing details at the upcoming workshop.”

Session: Modes of Treatment Delivery

Date/Time: Friday September 29, 2023, 1:05 – 1:25pm

Location: Le Meridien Dallas, Stoneleigh Hotel – Grand Salon

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 the oligodendrocyte-targeting rAAV vector-based gene therapy being intended to restore ASPA function and brain development in patients with CD and the underlying recovery of brain anatomy following gene therapy treatment being at the root of the functional advances being made by treated patients. 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.

Contacts

Media:

Jordana Holovach

Head of Communications and Community

Myrtelle Inc.

781-621-2797 Ext. 102

[email protected]