- French pharma Servier and biotech partner Neurochlore have agreed to end early a pair of late-stage studies of an experimental medicine for autism after finding no benefit to treatment, the companies said in a statement Wednesday.
- The drugmakers joined forces in 2017 after a smaller study conducted by Neurochlore suggested bumetanide could improve symptoms in children with autism spectrum disorders. But initial results from Servier's two Phase 3 studies showed no significant difference between the drug and a placebo.
- Neurochlore President Yehezkel Ben-Ari said his team will continue to explore whether bumetanide can help specific subgroups of people on the spectrum, noting in the Tuesday statement that it's likely no one treatment will work for all patients because autism spectrum disorders are so different.
Autism spectrum disorders affects about 1 out of every 54 children in the U.S. But while there are drugs available for some of the symptoms patients experience, there are no approved treatments for the underlying condition.
Neurochlore had hoped that bumenatide, a diuretic commonly used to treat fluid retention, would offer a way forward because it affects a chemical messenger in the brain that some researchers think may play a role in autism. Parents said children involved in earlier research appeared "more present," Ben-Ari said in 2017, according to the autism research-focused publication Spectrum.
But even as researchers expressed early optimism, experts suggested the 2017 trial results may have reflected a placebo effect, Spectrum reported at the time. As bumenatide is a strong diuretic that causes a need to urinate frequently, some parents probably figured out their child was getting the active medicine instead of a placebo.
In the companies' statement, Servier's head of R&D Claude Bertrand emphasized the "extremely rigorous" standards of its two Phase 3 trials and expressed disappointment in the study results. The two trials enrolled 422 children and adolescents between the ages of two and 17.
According to the companies, none of the effectiveness criteria of the studies were reached after six months of treatment. That was true for both younger children as well as adolescents, they said.
Servier has been expanding its research reach, especially in the U.S., where it opened a Boston headquarters in 2019. Late last year, the company agreed to pay Agios Pharmaceuticals $1.8 billion for a portfolio of cancer medicines and extended job offers to about a third of the Massachusetts-based biotech's staff.