Huntington's Program Bites the Dust as Novartis Cleans House

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Novartis is cleaning house, cutting its Huntington’s disease program along with several others. The company announced multiple program stops and delays in its full-year 2022 report Wednesday.

August brought a temporary halt to the Phase IIb trial of branaplam for Huntington’s after the discovery of possible side effects. At the time, the pharma was “gathering more data” to decide the next steps.

It looks like Novartis got that data.

In the year-end report, the company noted that the program was discontinued after a benefit-risk assessment from the VIBRANT-HD study. Early indications of side effects included peripheral neuropathy or nerve pain. The drug was initially developed for spinal muscle atrophy but was dropped for that indication.

The branaplam program is just one of many disappointments for Huntington’s of late, as the fatal disease continues to frustrate biopharma with trial holds and failures.

The decision follows an analysis that confirmed a signal of neurotoxicity in the majority of VIBRANT-HD participants, a company spokesperson said in an email to BioSpace.

Novartis’ blockbuster drug Cosentyx was also affected by the decluttering. A Phase III drug trial for active peripheral spondyloarthritis was “terminated due to strategic decision of senior management,” according to

Cosentyx is already approved in five inflammatory conditions - plaque psoriasis, ankylosing spondylitis, psoriatic arthritis, non-radiographic axial spondyloarthritis and enthesitis-related arthritis, which garnered a total $4.8B in sales last year for the company.

“We disclosed at Q3 that we have stopped the Cosentyx pSpA trial. We continue to invest in new modes of administration to benefit as many axSpA patients as possible, and we hope to bring an IV formulation to U.S. patients with PsA, AS and nr-axSpA in 2023,” the same spokesperson said. 

Other pipeline updates include:

  • Crizanlizumab “does not show superiority compared to placebo” for pediatric sickle cell disease, and “health authorities have been informed”
  • IgA nephropathy and C3 glomerulopathy program submissions were delayed due to slow recruitment, but readout is confirmed for both in 2023
  • Program for membranous nephropathy dropped due to a non-compelling competitive profile
  • Breast cancer drug Piqray: canceled plans to add two more indications due to reprioritization

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