Servier and Neurochlore have recently announced that, following a lack of efficacy in two Phase III trials of bumetanide for autism spectrum disorder (ASD) in adolescents, they would be terminating these trials early. These results are particularly disappointing given the promise bumetanide had shown in an earlier Phase IIb trial, and because there is a large unmet need for treatments for the core symptoms of ASD, says GlobalData. The leadng data and analytics company notes that currently, the only FDA-approved drugs for autism are the atypical antipsychotics, which aim to treat the associated symptom of irritability.

The large unmet need for a treatment for ASD is reflected in a diverse pipeline, with numerous companies recognizing the market potential for such a product. GlobalData’s proprietary database shows that the autism pipeline consists of 47 drugs spanning all stages of development, including two drugs in Phase III, five drugs in Phase II, and five drugs in Phase I development.

Philippa Salter, Neurology Analyst at GlobalData, comments: “There are a variety of drug types in the autism pipeline, such as small molecules, antibodies, and cell therapies, as well as a wide variety of mechanisms of action reflecting the heterogenous nature of the disease. Some of the common targets in the autism pipeline include the glutamate NMDA receptor and the gamma-aminobutyric acid (GABA) receptor.”

The most advanced pipeline drug is Curemark’s CM-AT, an enzyme replacement therapy that addresses the digestive problems suffered by a large portion of autistic children.

Salter adds: “CM-AT received fast track designation from the FDA in 2010, and in a Phase III trial demonstrated statistically significant improvements in core and non-core symptoms of autism in the preschool subpopulation when compared to placebo, giving it the potential to become the first treatment approved for the core symptoms of ASD.”