Abeona Therapeutics Inc (NASDAQ:ABEO) has announced that it has signed up the first two patients that will take part in its ABO-102 Phase 1/2 clinical trial.
The clinical trials are being carried out in sites located in Australia and the U.S. The two patients are the first voluntary patients participating in the clinical trials and they will receive Cohort 3 doses of the ABO-102. The new trial is a pivotal expansion of the treatment which is in development for addressing the MPS IIIA disease. ABO-102 will be delivered to the patients in the clinical trial through one intravenous injection that will introduce the AAV viral vector into the body. The viral vector will then act as a corrective copy of the SGSH gene which is the cause of the disease.
“The recently announced one-year data on our Cohort 1 patients showed durable, time-dependent responses as measured in reductions of heparan sulfate storage pathology in the CSF and urine, reduction in liver volume, stabilization of deep brain architecture and signals of stabilization of neurocognitive decline one-year post-administration,” stated Timothy J. Miller, the president and Chief Executive Officer of Abeona Therapeutics.
Miller also pointed out that the biopharma decided to pursue the new clinical trial after it identified positive improvements in patients in cohort 2. The CEO also revealed that the decision to pursue the new trial was also supported by regulatory bodies in three countries.
Miller also revealed that the treatment was well-tolerated across all the doses at all timeframes. This has played a key part in enabling a rapid enrollment schedule that will take place over the next few months. Juan Ruiz, the Chief Medical Officer at Abeona stated that there will most likely be improvement outcomes. He believes this due to the results of the many natural and clinical history studies that involve neurodegenerative conditions such as MPS IIIA.
Ruiz also stated that he was encouraged by the early outcome data from all of the patients that have participated in the ABO-102 clinical trials. He also added that it has demonstrated an appealing safety profile so far.
Abeona Therapeutics stock closed the latest trading session on Wednesday at $19.35.