RARE Daily

Novartis Discontinues Development of Experimental Therapy for Huntington Disease

February 2, 2023

Rare Daily Staff

Novartis, as part of a company update with its 2022 earnings, said it has discontinued the development of branaplam, its experimental therapy for the rare neurodegenerative condition Huntington’s disease.

In August, the company had sent a letter to the Huntington’s disease community to say it had temporarily halted dosing in the VIBRANT-HD phase 2b study of branaplam due to early signs of side effects in some participants that suggest it may cause peripheral neuropathy.

Peripheral neuropathy results from injury to the nerves located outside of the brain and spinal cord. The study steering committee endorsed the recommendation made following a regularly planned review by an independent Data Monitoring Committee, and temporarily suspended dosing. The committee did not recommend terminating the study at that time.

Huntington’s disease (HD) is a progressive disorder that causes brain cell (neuron) degeneration in certain areas of the brain. It is caused by mutations in the HTT gene and is inherited in an autosomal dominant manner. The defective gene codes for a protein called huntingtin, which in its defective form results in uncontrolled movements, loss of intellectual abilities, and emotional disturbances. Symptoms typically appear between the ages of 35 and 44 years. People with Huntington’s disease most often live for 15 to 18 years after the condition appears.

Branaplam is a small molecule that was originally developed by Novartis to treat spinal muscular atrophy. In late 2020 Novartis reported that branaplam lowers the level of huntingtin protein, and held promise as an attractive way to treat HD.

The Huntington’s community has seen several setbacks to the development of a treatment, which has been a stumbling block for many companies. In 2021, Roche said it was stopping dosing of its antisense candidate tominersen in a late-stage trial. This was followed by Wave Life Sciences dropping two earlier stage trials.

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