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    Abeona Therapeutics, Inc. (ABEO) Stock Relatively Stable Following Positive Data for Sanfilippo Syndrome Treatment

    By Shimrez Hyder

    Jul 27,2021

    6:36 AM UTC

    Abeona Therapeutics, Inc. (ABEO) stock prices were up 0.76% as of the market close on July 26th, 2021, bringing the price per share up to USD$1.32 at the end of the trading day. Subsequent current market fluctuations saw the stock fall by 5.30%, bringing it down to USD$1.25.

    Promising Topline Data

    July 26th, 2021 saw ABEO stock announce magnetic resonance imaging (MRI) data from the Phase 1/2 Transpher A clinical study. The study indicated that ABO-102 increases grey matter, corpus callosum, and amygdala volumes in the brain. The study consisted of three young patients with Sanfilippo Syndrome Type A (MPS IIIA) at 24 months, compared to afflicted patients who did not receive treatment. The findings were presented by ABEO stock during an oral presentation at the 16th International Symposium on MPS and Related Diseases.

    Scope of Findings

    Brain volume loss is typical in children with MPS IIIA and is linked to long-term cognitive and physical disability. Grey matter has been found to be important for cognitive development, whereas the corpus callosum is crucial for motor function. The amygdala plays a critical role in fear learning as well as social/emotional development. The findings highlight the potential of ABO-102 to increase brain grey matter, corpus callosum, and amygdala volumes. This potential is in line with ABEO stocks’ previously reported results of preservation of neurocognitive development in the three young patients in the Transpher A study.

    ABEO Stock’s Study Endpoints

    The Transpher A study primary endpoints targeted neurodevelopment and the treatment’s safety. Secondary endpoints include, but are not limited to, brain volume, behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma, and urine, and liver volume.

    About the Study

    The Transpher A Study is a two-year process that is currently ongoing as an open-label, dose-escalation, Phase 1/2 global clinical trial. It is designed to assess the treatment of patients with Sanfilippo syndrome type A (MPS IIIA) with the use of ABEO stock’s ABO-102. The study is known as ABT-001 and is intended for patients ranging from birth to 2 years of age, or for patients older than 2 years with a cognitive developmental quotient of 60% or above.

    Future Outlook for ABEO

    Armed with the latest data from its highly promising study, ABEO stock is poised to capitalize on the opportunities ahead of it. The company is keen to push for the accelerated development and proliferation of ABO-102, which investors hope will translate into long-term gains in shareholder value.

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