Encouraging results from the Abeona trial were presented today (8/2/17) at the WORLDSymposium for Lysosomal Diseases in San Diego. Ten children are now enrolled in the Sanfilippo type A study in the US, Spain and Australia and have been treated at one of three different doses. The first results from the Abeona Sanfilippo type B trial were also released this week.
This is the first time data has been shared from the four patients that have been given the highest dose of the AAV gene therapy for Sanfilippo type A (ABO-102). The gene therapy continues to be safe and tolerable and reductions in heparan sulfate, the toxic substance that builds up in children with Sanfilippo, have been seen in both the urine and the cerebral spinal fluid (CSF). After 30 days the heparan sulfate was reduced by 64% and 92% in the CSF and urine respectively in those children receiving the highest dose. The liver, which is enlarged in children with Sanfilippo, was also reduced at all three doses.
Information on the effect of the high dose on cognitive function is not yet available but the trial continues to show encouraging signs of stabilisation in some of the cognitive tests in some of the children treated at the lower doses.
Abeona plans to recruit another four to five patients in this phase 1/2 trial and are planning a further trial to treat more patients. It was also announced today that the FDA has given clearance for the minimum enrolment age for the phase 1/2 trial to be lowered from two years to six months.
In the Sanfilippo type B trial, one patient has been treated so far and, similar to the type A trial, there are no safety concerns so far and 30 days after treatment significant heparan sulfate reductions were observed in cerebral spinal fluid (50%), urine (69%), plasma (60%).
Sanfilippo type B trial press release
Sanfilippo type A trial press release