Regulators in Spain Give Abeona Okay to Begin MPS IIIB Gene Therapy Trial

September 12, 2018

Rare Daily Staff

Abeona Therapeutics said regulators in Spain have given it authorization to begin a phase 1/2 clinical trial there for the company’s gene therapy product ABO-101 for patients with MPS IIIB, or Sanfilippo syndrome type B, a rare, progressive metabolic disease.

MPS IIIB is a lysosomal storage disorder caused by genetic mutations that result in a deficiency of NAGLU enzyme activity, which leads to accumulation of heparan sulfate in the brain and other organs as well as progressive brain atrophy with cortical gray matter volume loss. The accumulation of abnormal HS results in neurocognitive decline, behavioral disturbances, speech loss, increasing loss of mobility, and premature death. MPS IIIB typically presents in children during the first few years of life, and 70 percent of patients do not reach 18 years of age. There are no approved treatments for MPS IIIB.

ABO-101 is an adeno-associated virus (AAV)-based gene therapy designed to deliver a corrective copy of the underlying gene that causes MPS IIIB. Prior data has shown 50 percent reductions in heparin sulfate in cerebral spinal fluid compared to baseline.

ABO-101 is Abeona’s first-in-human, adeno-associated viral-based gene therapy for MPS IIIB. Treatment involves a one-time intravenous delivery of a functioning copy of the NAGLU gene to cells of the central nervous system and peripheral organs with the aim of correcting the effects that result from the genetic aberrations that are the root cause of the disease.

Following administration of a single dose in Sanfilippo preclinical animal models, ABO-101 induced cells in the central nervous system and peripheral organs to produce the missing NAGLU enzyme. In preclinical in vivo efficacy studies in Sanfilippo syndrome animal model, ABO-101 demonstrated functional benefits that continue for months after treatment. A single dose of ABO-101 significantly restored normal cell and organ function, corrected cognitive defects, increased neuromuscular function and normalized the lifespan of animals with MPS IIIB after treatment compared to untreated control animals.

ABO-101 has been granted rare pediatric disease designation in the United States and Orphan Product designation in both the United States and European Union.

Patients in the Phase 1/2 trial will receive a single, intravenous infusion of ABO-101. They will be evaluated at multiple time points post-injection for safety assessments and efficacy parameters.  The clinical program is supported by a Natural History Study, which included potential efficacy assessments consisting of neurocognitive evaluations, biochemical assays and MRI data generated over one year of follow-up assessments.

“The authorization of our ABO-101 trial in Spain is a significant milestone for children suffering from MPS IIIB, a devastating and deadly disease with no approved treatment options,” said Carsten Thiel, CEO of Abeona. “We are encouraged by the preliminary results observed in our U.S. trial to date, both in clinically relevant biomarkers and in the ongoing safety profile and are excited to bring this therapy to patients in Europe.”

September 12, 2018
Photo: Carsten Thiel, CEO of Abeona


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