Researchers publish results of previously-terminated Sanfilippo Type A enzyme replacement study

July 29, 2022

Study results from Sobi’s previously-terminated Sanfilippo Type A enzyme replacement study have been released. The study results were published in the August 2022 edition of Molecular Genetics and Metabolism and provide some important insights.

For context, this program was wound down based on Sobi’s business strategy prior to dose escalation results being available. Hence treatment efficacy, would not have been able to be considered at that time.

Despite program closure, the authors have provided data to help the community continue to build on learnings from this trial.

Some key take-aways from the paper, “Chemically modified recombinant human sulfamidase (SOBI003) in mucopolysaccharidosis IIIA patients: Results from an open, non-controlled, multicenter study“:

  • It is possible for systemically administered, modified SGSH enzyme to cross the BBB.
  • HS reduction in the CSF correlated with increased drug doses and with anti-drug antibody levels.
  • Data show that in response to intravenous modified SGSH enzyme, an average of 70-80% reduction in CSF HS levels correlated with stabilization of cognitive scores on the Bayley test over the two year study. (Stabilization in Sanfilippo is a massive win when it comes to treatment effect)

“These are very important findings that could inform future trials and therapeutic development programs targeted at reduction of heparan sulfate. They also provide important information in considering the use of HS as a surrogate biomarker in Sanfilippo syndrome,” said Dr. Cara O’Neill, Chief Science Officer for Cure Sanfilippo Foundation. “And to the families that participated in this trial, these results must be very difficult to hear, given the closure of the trial.

“We THANK the families that participated in the Sobi trial and share with them the heartbreak that likely continues since the discontinuation of this clinical trial program and potential therapy,” said O’Neill. “We continue to work hard to make more opportunities to participate in clinical trials available for children.”

Read the publication.

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