Myrtelle Receives FDA Fast Track, Rare Pediatric Disease, and Orphan Drug Designations for its Proprietary Gene Therapy for the Treatment of Canavan Disease

WAKEFIELD, Mass.–(BUSINESS WIRE)–Myrtelle Inc. (“Myrtelle” or the “Company”), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track, Rare Pediatric Disease (RPD), and Orphan Drug designations for its lead clinical-stage gene therapy, rAAV-Olig001-ASPA, for the treatment of patients with Canavan Disease (CD).

“FDA’s decision to grant these designations for our investigational gene therapy utilizing rAAV-Olig001-ASPA aligns with our mission to provide treatments for patients where few if any options exist and highlights the urgency of developing a treatment for patients with Canavan disease, a devastating disease of young children which results in short life expectancy,” said Nancy Barone Kribbs, Ph.D., Senior Vice President of Regulatory Affairs at Myrtelle Inc.

Myrtelle’s ongoing Phase 1/2 First-in-Human clinical trial utilizes a novel proprietary recombinant adeno-associated virus (rAAV) vector, AAV-Olig, that for the first time directly targets oligodendrocytes in the brain. Oligodendrocytes are the cells in the brain responsible for producing myelin, the insulating material that enables proper function of neurons. The production of myelin is affected in CD due to mutations in the Aspartoacylase gene (ASPA) coding for the Aspartoacylase enzyme (ASPA). The oligodendrocyte-targeted gene therapy is intended to restore ASPA function, enable metabolism of the abundant brain chemical N-Acetylaspartate (NAA), and support myelination. Myrtelle entered into an exclusive worldwide licensing agreement with Pfizer Inc. in 2021 to develop and commercialize this novel gene therapy for the treatment of CD.

About Fast Track Designation
FDA’s Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Fast Track designation allows for frequent communication with the FDA throughout the drug development and regulatory review process, which assures questions and issues, are resolved quickly often leading to earlier drug approval and access by patients. The designation also enables eligibility for Accelerated Approval, Priority Review, and rolling review of a company’s New Drug Application, if relevant criteria are met.

About Rare Pediatric Disease Designation
Under FDA’s Rare Pediatric Disease (RPD) designation, a sponsor who receives marketing approval for a product with such a designation may be eligible for a voucher that can be redeemed to obtain priority review for any subsequent marketing application. For the purpose of RPD designation, the FDA defines a “rare pediatric disease” as a disease that affects fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals from birth to 18 years of age.

About Orphan Drug Designation
FDA grants Orphan Drug designation to drugs or biological products intended for the treatment of patients with rare diseases that affect fewer than 200,000 people in the US. The designation gives companies who are developing therapies for rare diseases an extended term of 7 years marketing exclusivity upon regulatory approval, reductions in certain application fees, and tax incentives.

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 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. CD patients 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 10 years of age. Currently, there are no cures for CD and only palliative treatments are available.

More information on Myrtelle’s clinical study 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 the company having the ability to benefit from Fast Track, Rare Pediatric Disease (RPD), and Orphan Drug designations. 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 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.