Branaplam on FDA’s Fast Track; Phase 2 Trial Now Enrolling

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The experimental oral medication branaplam (LMI070) has been granted fast track designation by the U.S. Food and Drug Administration (FDA) as a potential treatment for Huntington’s disease, according to an announcement from the therapy’s developer, Novartis.

This designation is designed to speed the development and review of investigational medicines that have the potential to substantially improve care for serious diseases and fill unmet medical needs. Branaplam received orphan drug designation from the FDA in 2020.

Novartis recently launched a Phase 2 clinical trial called VIBRANT-HD (NCT05111249) that is testing branaplam in people with early manifest Huntington’s. The study is currently enrolling, according to Novartis, and plans to recruit about 75 participants, ages 25 to 75. Study sites have not been announced yet.

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The first part of the study intends to determine optimal dosage of the investigational treatment. The first group of patients enrolled will be given 56 mg of branaplam or a placebo, taken as an oral solution once weekly.

Based on data from the first group, the next group will test a higher dosage of the investigational medicine, 112 mg per week. Informed by results from the first two groups, the third and final group will test a dosage of either 154, 84, or 28 mg per week.

The study’s main goals are to assess the treatment’s safety and tolerability, as well as its effect on mutant huntingtin protein — the underlying cause of Huntington’s — in the fluid around patients’ brains after 17 weeks.

After the initial dose-finding portion of the trial, participants can enter into an open-label extension, where all will receive treatment with branaplam and be regularly monitored for about a year.

Branaplam, also called LMI070, was developed originally to treat spinal muscular atrophy (SMA), a genetic disease characterized by the progressive death of motor neurons (the nerve cells that control movement).

In the course of clinical testing in SMA, it was observed that the investigational therapy lowered levels of huntingtin messenger RNA, the “template” that cells use to make the huntingtin protein when its gene is “read.” Branaplam also has been shown to decrease levels of mutant huntingtin protein in animal models. Since mutant huntingtin protein causes Huntington’s, reducing levels of the protein may ease symptoms or delay disease progression.