failure

Relyvrio (sodium phenylbutyrate and taurursodiol), an approved treatment for people with amyotrophic lateral sclerosis (ALS), has failed to meet its primary goal of slowing disease progression in an ongoing Phase 3 clinical trial designed to confirm its effectiveness. That’s according to new data from the trial, dubbed PHOENIX,…

Treatment with Ultomiris (ravulizumab-cwvz) — a therapy approved for several rare diseases that works by blocking activation of part of the immune system called the complement cascade — failed to slow the progression of amyotrophic lateral sclerosis (ALS) in a Phase 3 clinical trial that had been launched…

Biohaven‘s therapy candidate verdiperstat did not significantly slow functional decline among people with amyotrophic lateral sclerosis (ALS) in the HEALEY ALS platform trial, failing to meet its primary goal, data show. Key secondary efficacy goals, such as survival and changes in lung function and muscle strength, also were…

Dimethyl fumarate or DMF — an oral therapy approved for multiple sclerosis (MS) — was well-tolerated among adults with amyotrophic lateral sclerosis (ALS) but failed to significantly slow disease progression or to improve survival and quality of life in these patients, according to data from a Phase 2…

A Phase 3 clinical trial evaluating Orphazyme’s investigational oral therapy arimoclomol for amyotrophic lateral sclerosis (ALS) has failed to meet its primary and key secondary goals. While no safety issues were reported in the trial (NCT03491462), arimoclomol did not significantly extend patients’ lives nor…

BrainStorm Cell Therapeutics’ cell-based therapy NurOwn failed to significantly slow disease progression in people with rapidly progressing amyotrophic lateral sclerosis (ALS), top-line data from a Phase 3 trial show. The therapy, however, did result in clinically meaningful responses in a pre-specified group of patients with early ALS…

Investigational therapy NP001 failed to improve disease severity and pulmonary function in a Phase 2 confirmatory trial for amyotrophic lateral sclerosis (ALS) patients with elevated levels of systemic inflammation, Neuraltus Pharmaceuticals announced. The findings, showing the trial failed to meet its primary and secondary goals, were recently shared during the…

A Phase 2 clinical trial, exploring ozanezumab as a treatment of amyotrophic lateral sclerosis, failed to show any benefits. The data at hand means the search for treatments that may outperform the only approved ALS medication, riluzole, continues. The study, “Safety and efficacy of ozanezumab in patients with…