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Novartis to pursue SMA drug branaplam in Huntington's disease

FILE PHOTO: The company's logo is seen at the new cell and gene therapy factory of Swiss drugmaker Novartis in Stein, Switzerland, November 28, 2019. REUTERS/Arnd Wiegmann

ZURICH (Reuters) - Novartis is seeking to repurpose its investigational oral spinal muscular atrophy (SMA) drug branaplam to treat Huntington’s disease, the Swiss drugmaker said on Wednesday, as it plans a clinical trial after winning U.S. orphan drug designation.

Novartis has been studying branaplam in SMA, but last year hinted that it would pivot to new neurological diseases after its head of research Jay Bradner said he did not see a “big opportunity” in its original disease indication.

The drug remains under study for SMA, but now Novartis said it wants to see if trials in humans can duplicate preclinical models in which branaplam was shown to reduce levels of a mutant protein that plays a role in Huntington’s disease, for which there are no approved disease modifying therapies that delay disease onset or slow progression.

“Novartis intends to start a development program for branaplam to determine if it has the potential to be a transformative treatment for people living with this devastating condition,” the company said in a statement.

Roche beat Novartis’s branaplam to the market with an oral SMA therapy, Evrysdi, which was approved earlier this year.

Reporting by John Miller, editing by John Revill

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