Trial of ABO-102 Gene Therapy Ends Citing No Neurocognitive Benefits

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A clinical trial that had been testing the experimental gene therapy ABO-102 in people with advanced syndrome type A has ended after an analysis indicated the therapy was not significantly improving neurological or cognitive functioning.

“All of us here at the Society and in the MPS community are deeply concerned regarding this news,” N. Matthew Ellinwood, chief scientific officer of the National MPS Society, wrote in an announcement.

The MPS society is planning to host an interactive webinar for the Sanfilippo A community in the coming weeks, once more is learned about the trial and its closure, according to Ellinwood. Until that time, the Society is encouraging those in need of support to contact Terri Klein at [email protected].

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Those who are affected by the news can also contact Abeona Therapeutics, the company developing ABO-102, for more information and guidance by emailing [email protected].

Sanfilippo syndrome type A, also called mucopolysaccharidosis IIIA (MPS IIIA), is caused by mutations in the gene SGSH, which is needed to break down a complex sugar molecule called heparan sulfate. ABO-102 is designed to deliver a healthy version of this gene using a specifically engineered viral vector to ultimately allow for this sugar molecule to break down normally.

Abeona sponsored a clinical trial (NCT04088734) to test the safety and effectiveness of ABO-102 in children ages 2 to 17 with relatively advanced Sanfilippo A, which is defined as a developmental quotient of less than 60. Developmental quotient, or DQ, is a measure of developmental ability compared to what’s normal, similar to intelligence quotient (IQ) for cognitive ability.

Participants were to receive a single ABO-102 injection into the bloodstream. The main goals of the trial were to assess the treatment’s safety, as well as its effect on heparan sulfate levels and spleen and liver volume. A number of developmental, cognitive, and life quality outcomes also were monitored.

According to Ellinwood, the trial ended as of March 10. Abeona made its decision “based on a recent review of available neurocognitive and biomarker data, which they concluded did not demonstrate improvements in neurocognitive function.”

“Until we have effective treatments for all in our community, this news is reminder of both the need to support our Sanfilippo A and allied communities and to redouble our efforts in research, family support, and advocacy, to ensure the discoveries, reforms, and supports are available to all those who are in need,” Ellinwood wrote.

Abeona is running a separate Phase 1/2 clinical trial called Transpher A (NCT02716246), which is testing ABO-102 in children with Sanfilippo A ages 6 months to 2 years who have a DQ of 60 or higher. This study is still ongoing and recruiting participants in the U.S., Australia, and Spain.