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Abeona Therapeutics Treats First Patient in Second Cohort of Phase 1/2 Clinical Trial for ABO-101 in Sanfilippo Syndrome Type B (MPS IIIB)NEW YORK and CLEVELAND, May 14, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the Company has completed dosing cohort 1 and treated the first patient in cohort 2 in an ongoing Phase 1/2 clinical trial evaluating ABO-101, its novel one-time gene therapy for Sanfilippo syndrome type B (MPS IIIB). The Company expects to report interim data from the trial in the second half of 2019. “We believe that ABO-101 can help address the underlying lysosomal enzyme deficiency that ultimately results in progressive cell damage and neurodevelopmental and physical decline in children with MPS IIIB,” said João Siffert, M.D., Chief Executive Officer. “The product safety profile to date and clear evidence of biological activity provide support for advancing this clinical trial to the second cohort. This multicenter study is made possible by our close collaborations with expert academic investigators and the participants who enrolled to date. We look forward to presenting interim data in the second half of this year.” The Phase 1/2 trial is a two-year, open-label, dose-escalation, global clinical trial in patients diagnosed with MPS IIIB who are older than six months of age and have a minimum cognitive Development Quotient of 60 or above. The study is expected to include up to nine patients and is designed to evaluate two doses of ABO-101 with cohort 1 receiving 2 X 1013 vg/kg and cohort 2 receiving 5 X 1013 vg/kg. ABO-101 is delivered using NAV® AAV9 technology via a one-time intravenous infusion. The primary endpoint of the study is to assess neurodevelopment and safety, with multiple secondary and exploratory endpoints including neurocognitive and behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, biomarkers in CSF, plasma and urine, and brain and liver volume. Abeona is enrolling eligible patients with MPS IIIB at sites in the U.S. and Spain and expects to activate additional sites globally this year to accelerate enrollment. Additional information about the study is available at AbeonaTrials.com and ClinicalTrials.gov (NCT03315182). About Sanfilippo syndrome type B (MPS IIIB) About ABO-101 About Abeona Therapeutics Forward Looking Statement Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to: continued interest in our rare disease portfolio, our ability to initiate and enroll patients in clinical trials, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company’s annual reports on Form 10-K and quarterly reports on Form 10-Q and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws. Investor Contact: Media Contact: |