Blog Coverage Abeona Provides Update on ABO-102 Trial Closely After EMA Committee Approval to Grant Orphan Drug Designation to the Program

1 year ago

LONDON, UK / ACCESSWIRE / October 21, 2016 / Active Wall St. blog coverage looks at the headline from Abeona Therapeutics Inc. (NASDAQ: ABEO) as the company provided update on the clinical results through 30 days post-injection for the completed low-dose cohort in the on-going Phase 1/2 trial for ABO-102 (AAV-SGSH) at the Orphan Drugs & Rare Disease Conference in London, UK. Register with us now for your free membership and blog access at:

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The Trial Details

Abeona noted that the first-in-man clinical trial utilizes a single intravenous injection of AAV gene therapy for subjects with MPS IIIA, a rare autosomal recessive disease affecting every cell and organ in the body causing neurocognitive decline, speech loss, loss of mobility, and premature death in children. The on-going Phase 1/2 study is designed to evaluate safety and preliminary indications of efficacy of ABO-102 in subjects suffering from MPS IIIA.

The company stated that the observations 30 days post-injection for the low dose cohort demonstrated, that ABO-102 was well-tolerated in subjects injected with the low dose of 5E13 vp/kg ABO-102 with no treatment related adverse events or serious adverse events (SAEs). Following favorable review of the safety data by the independent Data Safety Monitoring Board (DSMB), enrolment in the high dose cohort has commenced. All subjects in the low-dose cohort experienced reductions from baseline in both urinary HS and CSF. At 30 days post-injection, urinary HS reduction was 57.6% +/- 8.2%. Reduction in CSF HS was 25.6% +/- 0.8%, suggesting that ABO-102 crossed the blood brain barrier after intravenous administration. All three subjects demonstrated significant reductions in liver volume (17.1% +/- 1.9%), and spleen volume (17.6% +/- 7.1%) from baseline, as measured by MRI at 30 days post-injection.

As per the design of the clinical trial, subjects in the low-dose cohort received a single, intravenous injection of AB0-102 to deliver the AAV viral vector systematically throughout the body to introduce a corrective copy of the gene that underlies the cause of the MPS IIIA disease. Subjects were evaluated at multiple time points over the initial 30 days post-injection for safety assessments and initial signals of biopotency, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system, to reduce GAG content that underlies the lysosomal storage pathology central to Sanfilippo syndrome type A (MPSIIIA).

“We remain encouraged by continued signs of tolerability and by early signals demonstrating reduced urinary and CSF GAG,” stated Kevin M. Flanigan, MD, principal investigator with the Center for Gene Therapy at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine.

A more complete analysis of these data will be presented from the low-dose cohort and initial high dose cohort at a scientific conference in Q1 FY17. The Data Safety Monitoring Board has approved dose escalation of the high dose cohort this quarter.

European Approval Achieved

Abeona’s MPS IIIA program, ABO-102, has been granted Orphan Product Designation in the USA and received the Rare Pediatric Disease Designation, and on October 18th, 2016, Abeona announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products has granted Orphan Drug Designation to the ABO-102 program.

Stock Performance

After the release of the results, on Thursday, the stock closed the trading session at $8.70, surging 12.11% from its previous closing price of $7.76. A total volume of 3.55 million shares have exchanged hands, which was higher than the 3-month average volume of 269.94 thousand shares. Abeona Therapeutics’ stock price rallied 32.42% in the last month, 248% in the past three months, and 214.08% in the previous six months. Furthermore, on a year to date basis, the stock surged 158.93%.

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