Abeona Therapeutics Inc (NASDAQ:ABEO) reported new interim positive data from its Phase 1/2 ongoing clinical studies of ABO-101 and ABO-102 in MPS IIIB and MPS IIIA.
A life-altering treatment for children
COO and Principal Executive of Abeona, Michael Amoroso, said the company is excited to share the interim positive safety and efficacy data. These positive results show the potential of ABO-102 to be a life-changing treatment option for children suffering from a rare and debilitating condition – MPS IIIA, which often causes early death.
Seeks discussion with FDA
Abeona seeks a meeting with the US FDA to discuss the interim data of ABO-102 and the subsequent path to submit BLA (Biologics License Application). It also supports the biologic activity of ABO-101 in patients suffering from MPS IIIB.
The Transpher A clinical study, which evaluates the drug ABO-102 in treating SSA (Sanfilippo syndrome type A) (MPS IIIA), shows that neurocognitive development is preserved in children who are not afflicted for up to 3 years. It is on the backdrop of treating children in cohort 3 with a high dose of ABO-102.
The three patients aged 12 months, 19 months, and 27 months are treated with ABO-102; they are aged between 3.5 to over five years. When the treatment commenced, the children with MPS IIIA are already experiencing a decline in neurocognitive abilities. The children have well tolerated the treatment using ABO-102, ensuring safety for up to 55 months. They have not reported any serious side effects.
The principal investigator of Transpher A trial, Kevin Flanigan, said the results obtained from a single dose of ABO-102 demonstrate preservation of neurocognitive development after the treatment for up to three years.
Also, the result of the Transpher B study that evaluates ABO-101 in MPS IIIB demonstrates dose-dependent treatment using ABO-101 sustains improvement in systemic biomarkers and the central nervous system. It is well tolerated in patients with no serious effects or abnormalities.
Another Transpher B investigator, Maria Jose de Castro, said the results of this clinical trial show the potential of ABO-101 to breakdown glycosaminoglycans accumulation. The team will continue the follow-up to assess the potential of ABO-101 to sustain neurocognitive abilities in patients suffering from MPS IIIB.