Abeona Therapeutics (ABEO) Announces DSMB Approval of ABO-102 Dose Escalation in Phase 1/2 Second Cohort for Sanfilippo Syndrome Type A

October 5, 2016 7:05 AM EDT
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Abeona Therapeutics Inc. (Nasdaq: ABEO) announced that the Data Safety Monitoring Board (DSMB), an independent group of medical experts closely monitoring the clinical trial, has reviewed the initial safety data from the low dose cohort (n=3) in the Phase 1/2 clinical trial of ABO-102 (AAV-SGSH) enrolling at Nationwide Children's Hospital in Columbus, Ohio. Following review of the safety data, the DSMB authorized that the clinical trial proceed with enrollment and dose escalation for the second cohort. The high-dose cohort will enroll up to six additional patients dosed at 1.0 X 1013 vg/kg, which is twice the amount of ABO-102 received by patients in the low-dose cohort.

"These early results support Abeona's unique approach to treating patients with Sanfilippo syndrome, where there are both profound CNS and whole body manifestations of the disease," stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. "We look forward to reporting on future progress and potential for ABO-102 as we begin to enroll patients at the high dose and open additional clinical sites internationally."

Abeona's ABO-102 program has been granted both Orphan Product Designation and Rare Pediatric Disease Designation in the USA and plans to open two additional clinical sites, one in Spain and one in Australia, to test ABO-102. A Phase 1/2 clinical study of ABO-102 in Spain was recently approved by the Agencia Espanola de Medicamentos y Productos Sanitarios, and the Company is preparing to conduct this clinical study at Cruces University Hospital in Bilbao, Spain.

Sanfilippo Syndrome Type A, or MPS IIIA, is a rare lysosomal storage disease caused by genetic mutations that result in a deficiency of SGSH enzyme activity, leading to abnormal accumulation of certain sugars (specifically, the glycosaminoglycan (GAG) heparan sulfate) in the central nervous system (CNS) and systemic tissues and organs. The accumulation of heparan sulfate results in neurocognitive decline, speech loss, loss of mobility, and premature death.

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