Biogen, Ionis ALS drug flops in trials

March 29, 2022

Biogen and Ionis Pharmaceuticals were hoping to be the first to find a cure for ALS. That dream has now fizzled out with the companies calling it quits after receiving disappointing results from a phase 1 clinical trial.

Study results from a phase 1 study of the drug found that it did not have any real benefits for patients involved in the study. The drug, called BIIB078, did not produce results any different from the drug in the placebo group. In fact, participants who received a 90 mg dose in the study trended toward a greater decline than those in the placebo group, Ionis said. The program will be discontinued as it failed to meet its endpoints.

“While these are not the results we were hoping for, they are clear and will inform future research across our broad pipeline of investigational ALS therapies,” said Toby Ferguson, vice president and head of the neuromuscular development unit at Biogen.

Biogen and Ionis designed BIIB078 based on the current hypothesis that disease mechanisms of ALS are associated with toxicity and repeats within RNA and corresponding dipeptides, short proteins that maintain the pH of cells or act as antioxidants. 

The phase 1 study did not support this hypothesis, but Ionis said it will use this information to develop a “deeper understanding” of the disease moving forward.

During the study, adverse events were mostly mild and moderate, with the most common ones being falling, procedural pains, and headaches, according to Ionis. Data from the phase 1 study of BIIB078 will be presented at a future scientific forum, the company said.