Years after a major trial setback, Novartis switches gears with SMA drug. This time they’re trying it for Huntington’s – Endpoints News

Posted: October 22, 2020 at 2:54 am

Four years after a Phase I/II setback in spinal muscular atrophy (SMA), Novartis is hoping its drug branaplam will find more success in a new neurological indication: Huntingtons disease.

The decision was announced a year after the head of research, Jay Bradner, said he did not see a big opportunity in SMA, according to Reuters. Novartis says it has preclinical data showing that branaplam reduces levels of mutant huntingtin protein, and SMA data showing patients on the drug had reductions in huntingtin mRNA. The FDA gave branaplam their orphan drug designation, and Novartis plans to move forth with a Phase IIb trial next year.

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.

Novartis isnt dropping the SMA indication altogether according to a company statement, branaplam is still under investigation for SMA. But a 2-year testing hiatus set Novartis behind its rival Roche, which won approval for its SMA therapy Evrysdi last year.

In 2016, Novartis halted enrollment in a Phase I/II study in SMA after signs of nerve injury appeared in some animal tests, according to a letter it released. The trial was resumed about 2 years later in Belgium, Germany, Denmark and Italy, with additional nerve tests added to the protocol.

It is still a long road, but we are pleased to be back on track, the letter read.

Evrysidi is priced by weight, beginning at less than $100,000 for infants under 15 pounds and capping at $340,000 per year. Analysts predicted that the drug would threaten Biogens SMA drug Spinraza, which comes in at $382,500 on the low-end. But Mani Foroohar of SVB Leerink predicted Novartis gene therapy Zolgensma would take the lead in young SMA patients. Given strong data, one-time therapy, and simple IV administration, we see Zolgensma as remaining the treatment of choice for very young SMA patients, he wrote, according to aFierce Pharma report.

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Years after a major trial setback, Novartis switches gears with SMA drug. This time they're trying it for Huntington's - Endpoints News

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