Canavan Disease

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: . 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 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

BOSTON, Feb. 1, 2024 /PRNewswire/ -- National Tay-Sachs & Allied Diseases Association (NTSAD), leader in the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases, is hosting the first-ever, Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) on February 15, 2024, 9:30am-3pm Eastern. EL-PFDD meetings give the U.S. Food and Drug Administration (FDA) and other key stakeholders, including medical product developers, health care providers, regulatory agencies, an important opportunity to hear from patients and their families about the symptoms that matter most to them, the impact the disease has on patients' daily lives and their experiences with currently available treatments. This input can inform FDA's decisions and oversight during drug development and their review of marketing applications. Nearly 30 people will share their stories and experiences with infantile, juvenile, or late onset GM2, including adult patients, parents, caregivers, and bereaved family members. Other presenters include Jacqueline Karp, MD, U.S. FDA Center for Drug Evaluation and Research; Florian Eichler, MD, Massachusetts General Hospital; Cynthia Tifft, MD, PhD, National Institutes of Health; and Kathleen Flynn, NTSAD CEO. "NTSAD was founded by families. They are the experts on living with these devastating rare, genetic diseases, and their voices should be heard," said NTSAD CEO Kathleen Flynn. This virtual meeting will be livestreamed via YouTube, open to the public, free to attend, but advance registration is required. There will be opportunities to call in, submit comments, or participate in live polling. To learn more about the GM2 EL-PFDD meeting, register to attend, or submit comments, visit . Meeting sponsors include Sanofi, Cure Tay-Sachs Foundation, New York Area Fund, Mathew Forbes Romer Foundation, Azafaros, and JCR Pharmaceuticals. About NTSAD One of the oldest patient advocacy organizations, NTSAD supports families and individuals globally through one-on-one support, mentoring and peer groups, information and resources, and its Annual Family Conference. NTSAD also advocates for patients and advances research. Since 2002, the organization has awarded more than $4.7 million in grants that attracted more than $30 million in research investments, leading to the development of potential therapies for Tay-Sachs, Canavan, GM1, and Sandhoff diseases. For more information, visit NTSAD.org. SOURCE National Tay Sachs and Allied Diseases Association Inc

WAKEFIELD, Mass., Nov. 7, 2023 /PRNewswire/ -- Myrtelle Inc. ("Myrtelle" or the "Company"), a clinical-stage 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 entitled "Translation of Gene Therapy Brain Biomarkers from Nonclinical Science to Clinical Trials: A Case Study in Canavan Disease" on Tuesday, November 7, 2023, at the 5th Annual Gene Therapy for Neurological Disorders Summit in Boston, Massachusetts. Dr. Janson's presentation will highlight encouraging data to date from Myrtelle's Phase 1/2 gene therapy clinical study of the Company's proprietary rAAV vector in patients with Canavan disease (CD). The rAAV vector directly targets 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 myelin production and brain development. This unique oligodendrocyte-targeting rAAV vector is a novel gene therapy approach directed at restoring ASPA function and brain development specifically in these target cells in patients with CD. The presentation will review encouraging post-treatment improvements through the primary analysis timepoint at one year in the Phase 1/2 clinical study, including emerging data on biomarkers identified by magnetic resonance imaging (MRI) that precede clinical score improvements in motor and cognitive function using validated scales. Specifically, statistically significant early gains in target white matter and myelin were observed following gene therapy which predict later improvements in specific functional scores on the validated Gross Motor Function Measure and Mullen Scales of Early Learning. Improvements generally continued to progress from the 6-month timepoint to the 12-month readout. As presented previously, treated patients were also observed to outperform an untreated age-matched natural history cohort on multiple measures. Dr. Janson's presentation will also cover foundational nonclinical data illustrating the gene therapy efficacy in model systems. "Biomarker improvements observed on MRI imaging in patients are supportive of underlying recovery of brain anatomy following gene therapy treatment. These results reinforce the hypothesis that addressing the gene deficit directly in the brain, and specifically oligodendrocytes, of patients with Canavan disease can potentially restore myelination and support functional improvements," said Dr. Janson. "We look forward to sharing details at the upcoming summit." Date/Time: Tuesday, November 7, 2023, 2:30 pm Location: Hilton Back Bay Hotel, Boston, MA 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: . 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 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 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 results obtained to date continuing to reinforce the hypothesis that addressing the gene deficit directly in the brain, and specifically in oligodendrocytes, of patients with Canavan disease can potentially restore myelination and support functional improvements. 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.