New Positive Phase 1/2 Interim Data Presented at WORLDSymposium™ Shows Neurocognitive Development of Young MPS IIIA Patients Preserved up to Three Years Following Treatment with Abeona's ABO-102 Gene Therapy
In addition to preservation of neurocognitive development with ABO-102 in MPS IIIA, new clinical results of ABO-102 in MPS IIIA and ABO-101 in MPS IIIB continue to show dose-dependent and sustained reductions in disease-specific biomarkers, denoting clear biologic effects
In addition, ABO-102 and ABO-101 continue to show favorable safety profile in ongoing studies
Abeona to host investor webinar on Tuesday, February 16, 2021 at 1:00 p.m. EST
NEW YORK and CLEVELAND, Feb. 12, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced new positive data from two ongoing Phase 1/2 clinical trials of the company’s investigational AAV-based gene therapies ABO-102 and ABO-101 in MPS IIIA and MPS IIIB, respectively. The interim data was presented in late-breaking platform oral presentations at the 17th Annual WORLDSymposium™. The presentation slides are available on the company’s website at www.abeonatherapeutics.com.
Michael Amoroso, Principal Executive and Chief Operating Officer of Abeona, stated, “We are excited to share updated positive efficacy and safety results that continue to suggest ABO-102 has the potential to be a life-altering treatment option for children with MPS IIIA, a rare, debilitating condition with no approved treatment that leads to progressive neurodevelopmental and physical decline, and often results in death early in life. We have requested a meeting with the FDA later this quarter to discuss the ABO-102 data and the potential path towards a Biologics License Application filing for ABO-102. In addition, the new results from the Transpher B study continue to support ABO-101’s biologic activity in patients with MPS IIIB.”
The updated results from the Transpher A study evaluating ABO-102 in Sanfilippo syndrome type A (MPS IIIA) demonstrated that neurocognitive development was preserved within normal range of a non-afflicted child for 2.5 years to 3 years (the latest time point measured) after treatment with ABO-102 (3x1013 vg/kg) in three young patients in the high-dose cohort 3. The three young patients were treated with ABO-102 at ages 27 months, 19 months, and 12 months and are now at ages ranging from 3.5 years to 5+ years, the timepoint at which patients with MPS IIIA have already started to experience neurocognitive decline based on the natural history of disease progression. Dose-dependent and statistically significant reductions in cerebrospinal fluid heparan sulfate, denoting enzyme activity in the central nervous system (CNS), and liver volume were sustained for two years after treatment. ABO-102 has been well-tolerated with long-term safety remaining favorable 24-55 months following treatment. There have been no treatment-related severe adverse events and no clinically significant adverse events reported.
Kevin Flanigan, M.D., Director, Center for Gene Therapy at AWRI at Nationwide Children's and Transpher A study principal investigator, said, “The results presented today show a single intravenous dose of ABO-102 can help preserve neurocognitive development for up to 3 years following treatment in young MPS IIIA patients during early stages of their disease. The data shows ABO-102’s ability to deliver a functional copy of the disease-causing SGSH gene to cells of the CNS and peripheral organs, as evidenced by the clinical benefits in neurocognition and biophysical measures and improvements in disease-specific biomarkers.”
Results from the Transpher B study evaluating ABO-101 in Sanfilippo syndrome type B (MPS IIIB) showed that treatment with ABO-101 is associated with a dose-dependent and sustained improvement in central nervous system and systemic biomarkers, indicating the potent biologic effect of ABO-101 in patients with MPS IIIB. ABO-101 has been well-tolerated with no infusion related or early acute reactions and no clinically significant adverse events or laboratory abnormalities.
Maria Jose de Castro, M.D., Hospital Clínico Universitario Santiago de Compostela and Transpher B study investigator, said, “The results from the Transpher B study provide evidence of ABO-101’s impact on disease biomarkers and potential to break down the accumulation of glycosaminoglycans that underlie MPS IIIB pathology. We look forward to continued follow-up to assess ABO-101’s potential to preserve neurocognitive development in patients with MPS IIIB.”
Investor Webinar on MPS III Gene Therapy Programs
Abeona management along with Dr. Flanigan and Dr. de Castro will host an investor webinar on February 16, 2021 at 1:00 p.m. EST. The live webinar, including audio and presentation slides, will be accessible at https://investors.abeonatherapeutics.com/events at the time of the meeting. To register in advance for the live webinar, click here.
An archived replay of the webinar will be available after the conclusion of the live event at https://investors.abeonatherapeutics.com/events.
About the Annual WORLDSymposium™
About the Transpher A Study
About the Transpher B Study
About Sanfilippo Syndrome Type A (MPS IIIA)
About Sanfilippo syndrome type B (MPS IIIB)
About Abeona Therapeutics
Investor and Media Contact: Greg Gin VP, Investor Relations and Corporate Communications Abeona Therapeutics +1 (646) 813-4709 email@example.com