information: Interim results
Announcement: interim results
Company: Abeona Therapeutics (USA - NY)
Product: ABO-101 (AAV NAGLU)
- gene therapy. ABO-101 (AAV NAGLU) is a next generation adeno-associated viral (AAV)-based gene therapy for MPS IIIB. This gene therapy involves a one-time delivery of a genetically modified virus to deliver a normal copy of the defective gene to cells of the central nervous system and peripheral organs with the aim of reversing the effects of the genetic errors that cause the disease. After a single dose in preclinical animal models of Sanfilippo syndrome, ABO-101 induced cells in the CNS and peripheral organs to produce the missing enzymes and help repair damage caused to the cells. Preclinical in-vivo efficacy studies in animals with Sanfilippo syndrome have demonstrated functional benefits that remain for months after treatment. A single dose significantly restored normal cell and organ function, corrected cognitive defects that remained months after drug administration, increased neuromuscular control and increased the lifespan of animals with MPS III over 100% one year after treatment compared to untreated control animals.
Disease: mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome Type B)
area: Rare diseases
Country: Spain, USA
- • On February 7, 2018, Abeona Therapeutics reported preliminary 30-Day safety and biopotency signals from the first patient dosed in the company’s ongoing Phase 1/2 trial for ABO-101, a gene therapy treatment for patients with MPS IIIB (Sanfilippo syndrome Type B), enrolling at Nationwide Children’s Hospital in Columbus, Ohio. The ABO-101 therapy involves a single intravenous injection of AAV gene therapy for subjects with MPS IIIB, a rare autosomal recessive disease causing neurocognitive decline, speech and mobility loss, and premature death. Abeona plans to enroll a total of three patients in Cohort 1 (2E13 vg/kg) before dose-escalating to the Cohort 2 dose (5E13 vg/kg). The Phase 1/2 study is designed to evaluate safety and preliminary indications of efficacy of ABO-101 in subjects suffering from MPS IIIB. In the first patient treated in Cohort 1:
- • ABO-101, at a systemic dose of 2E13 vg/kg, is well-tolerated, with no treatment related adverse events or serious adverse events (SAEs) through 30 days of follow up.
- • Early biopotency signals include significant heparan sulfate (HS) reductions observed in cerebral spinal fluid (50%), urine (69%), plasma (60%) and urinary total glycosaminoglycan (GAG) (67%).
- • 50% decline in CSF heparan sulfate from baseline supports previous AAV9 clinical observations that ABO-101 crossed the blood brain barrier after intravenous administration.
• Normalized NAGLU enzyme activity observed represented by a greater than 300-fold increase over baseline at 30 days post administration.
- Subjects in the Phase 1/2 trial receive a single, intravenous injection of ABO-101, which uses an AAV vector to introduce a corrective copy of the NAGLU gene associated with MPS IIIB disease. Subjects will be evaluated at multiple time points over the initial 30 days post-injection for safety assessments and initial signals of biopotency. Results in the first patient dosed with ABO-101 suggest strong CNS and broader systemic distribution, with the potential to reduce levels of glycosaminoglycans (GAGs) that represent the lysosomal storage pathology central to MPS IIIB disease progression.
- • On May 24, 2016, Abeona Therapeutics announced the FDA has allowed an Investigational New Drug (IND) Application for its phase 1/2 clinical study with gene therapy candidate ABO-101 (AAV-NAGLU) for patients with Sanfilippo syndrome type B (MPS IIIB) to be conducted at Nationwide Children's Hospital (Columbus, OH). This is the second FDA allowance for a gene therapy trial from Abeona this year, following allowance of an IND in February for ABO-102, for patients with MPS IIIA which commenced with dosing of the first cohort of patients in May.
- • On January 11, 2016, Abeona Therapeutics announced the Interministerial Council of Genetically Modified Organisms has approved the Genetically Modified Organism (GMO) Voluntary Release regulatory filings for Phase 1/2 Gene Therapy Clinical Study to treat patients with AB0-101 (AAV NAGLU) for patients with Sanfilippo syndrome type B (MPS IIIB). Additionally, the Comite Etico De Investigacion Clinica de Euskadi (CEIC-E) has approved the ethical committee regulatory filings for both ABO-101. The Company plans to file CTAs for the programs shortly for the upcoming clinical studies to be conducted at Cruces University Hospital (Bilbao, Spain).