Sanofi has reported positive results from the ASCEND and ASCEND-Peds trials of olipudase alfa in patients with acid sphingomyelinase deficiency (ASMD)/Niemann-Pick disease, a rare and progressive disorder.

Olipudase alfa is an enzyme replacement therapy that is said to be the first of its kind in late-stage development to treat ASMD, which currently lacks approved therapies.

The randomised Phase II/III ASCEND trial was conducted in 36 adult patients at 24 sites across 16 countries. Participants were given intravenous olipudase alfa every two weeks at a dose of up to 3mg/kg over 52 weeks.

Data showed that the trial met both its independent primary endpoints, which measured lung function improvement and the drug’s effect on spleen size, respectively.

Lung function improvement was 22% from baseline to week 52 in the olipudase alfa group versus 3% in the placebo arm, while the spleen volume reduction was 39.5% versus 0.5%.

Patients in both groups had a minimum of one adverse event over the 52-week treatment period. The number of events in the olipudase alfa group was 242, compared to 267 in the placebo group.

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ASCEND-Peds is a single-arm, open-label Phase II study conducted in 20 paediatric patients in six countries. The trial’s primary objective was safety and tolerability up to a 3mg/kg intravenous dose of olipudase alfa administered every two weeks over 64 weeks.

The majority of events experienced by participants were mild and moderate. One subject had a severe and serious anaphylactic reaction linked to Sanofi’s drug.

ASCEND-Peds also monitored progressive lung disease and enlarged spleen as secondary endpoints.

Following one year of treatment, the predicted diffusing capacity of carbon monoxide (DLco) showed an average of a 33% increase in nine patients who performed the test at baseline. A 49% decrease was also observed in the spleen volumes.

Sanofi Research and Development global head John Reed said: “These significant results for olipudase alfa mark a major scientific advancement for ASMD and an important step toward providing a potential therapy for adult and paediatric patients who currently have no approved treatment options for this devastating disease.”

The company plans to start filing regulatory submissions for the drug in the second half of next year.