After 2-Year Hiatus, Novartis Resumes Branaplam Clinical Trial in SMA Type 1 Infants

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by Magdalena Kegel |

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Mestinon trial

Novartis will resume clinical development of LMI070 (branaplam) to treat spinal muscular atrophy (SMA) after a two-year pause brought on by safety concerns, the Swiss company announced in a letter published by Cure SMA.

The ongoing Phase 1/2 clinical trial (NCT02268552) will start enrolling patients as soon as the research team obtains approvals by health authorities and the ethics committee.

The study recruits infants younger than six months with type 1 SMA. All will receive branaplam, the name Novartis chose for its compound when reopening clinical investigations.

While the study originally only included four European study centers — in Belgium, Germany, Denmark and Italy — Novartis said it plans to add more sites including some in the United States, following a green light from the U.S. Food and Drug Administration (FDA).

The branaplam clinical trial halted new enrollments in May 2016 after simultaneous animal studies linked branaplam to unexpected nerve damage and other injuries.

Infants already enrolled continued treatment and have been closely monitored. Novartis has worked with outside experts to understand the meaning of the animal findings. The fact that enrollment has resumed signals that Novartis — which has not commented on its relevance — no longer perceives the animal data as an immediate patient hazard.

Besides resuming recruitment, the company has modified its trial design after considering feedback from the babies’ parents and study investigators. For instance, infants can now receive their weekly dose of branaplam orally, instead of via a feeding tube, which was the only option when the trial started.

The company also added nerve tests to the trial as an additional safety procedure.

“We believe that the best way to bring a product that is most beneficial to SMA patients and their families is by working in close partnership with you. So, moving forward, we will make sure that the SMA community is informed regularly as development for branaplam advances,” said the company’s letter to CureSMA.

SMA affects roughly one in 11,000 babies, and about one in every 50 Americans is a genetic carrier, said the Chicago-based nonprofit group. The disease, which can affect any race or gender, is the top genetic cause of death for infants.

As clinical development of branaplam continues, Novartis is also working with regulators to expand drug testing to include patients with other types of SMA.

“We would like to thank all of you who are or who will be participating in the study for branaplam, and those of you who support them,” the letter said. “Your contribution to bringing new and innovative treatments for SMA patients is invaluable.”