Myrtelle's rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Regenerative Medicine Advanced Therapy (RMAT) Designation from the U.S. Food and Drug Administration

NEW YORK, April 2, 2024 /PRNewswire/ -- 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) granted Regenerative Medicine Advanced Therapy (RMAT) Designation to the Company's lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of Canavan disease (CD). The RMAT designation program was created to expedite drug development and the review process of promising pipeline drugs, including gene therapies.  Designation is granted based on preliminary clinical evidence indicating that a drug or therapy has the potential to address unmet medical needs and is intended to treat, modify, reverse, or cure serious or life-threatening conditions. 

"The RMAT designation by FDA for rAAV-Olig001-ASPA is significant in that it recognizes the potential of this treatment for children with Canavan disease who are without approved treatment options.  The RMAT program provides FDA guidance on drug development and interactions that support accelerated approvals for treatments that serve communities like Canavan who face considerable unmet medical need," said Nancy Barone Kribbs, PhD, Senior Vice President of Global Regulatory Affairs at Myrtelle.

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. 

In addition to RMAT designation, rAAV-Olig001-ASPA has been granted Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the FDA, Orphan Drug Designation & Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency, as well as Innovative Licensing and Access Pathway (ILAP) from the United Kingdom Medicines & Healthcare products Regulatory Agency.

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 benefits to be derived from the RMAT designation. 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 CD, 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.

SOURCE Myrtelle, Inc