Abeona Therapeutics Inc (NASDAQ:ABEO) has a new update from the gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA) that is currently ongoing. The leading clinical-stage biopharmaceutical company made this declaration at the 13th Annual WORLDSymposium™ 2017 conference in San Diego.
Results from the ongoing Phase 1/2 trial for ABO-102 have been very encouraging. Principal investigator and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine, Kevin M. Flanigan says that they are confident about the unfolding of events.
Some of the notable results include stabilization of various cognitive functions just after six months of post-dosing. Flanigan points out that there has also been a continued sign of tolerance in the low-dose cohort.
The updated data exhibits an early and robust systemic delivery of ABO-102
Abeona has been putting so much focus into the development of therapies for critical rare genetic diseases. Nonetheless, there are various observations that are likely to declare the readiness of ABO-102. For example, there is a conclusion that it is safe for use in subjects given with the small doses of 5E13 vg/kg ABO-102.
President and CEO of Abeona Therapeutics, Timothy J. Miller also notes that there were enhanced reductions in CNS HS GAG support. The trial has had continued biomarker signals and first-hand positive indications in the neurocognitive assessments. Nonetheless, Miller says they are now looking at further expansion and enrollment of the clinical trial.
Abeona’s MPS IIIA program, ABO-102, obtains Orphan Product Designation
To some extent, ABO-102 seems to be moving from one level of success to another. As for the safety assessment, results indicate that it was able to reach the target tissues throughout the body, the central nervous system included.
Of interest to note is that ABO-102 has been endorsed for Orphan Product Designation both in the SA and in the European Union. As if this is not enough, it has also obtained the Rare Pediatric Disease Designation in the US. Being one of the lead programs for Abeona, it is clear that it is designated for greater heights.