Phase 3 clinical trial

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,…

On the heels of promising HEALEY-ALS Phase 2 trial data, Prilenia Therapeutics plans to launch a pivotal Phase 3 study of pridopidine, its candidate amyotrophic lateral sclerosis (ALS) treatment, in the second half of this year. Relative to a placebo, pridopidine was associated with slower disease progression,…

Note: This story was updated Jan 24, 2024, to correct that Radicava and Radicava Oral Suspension are available in Switzerland but not in other European countries. FAB122, an oral formulation of edaravone developed by Ferrer, failed to slow disease progression or extend survival in adults with amyotrophic lateral…

The U.S. Food and Drug Administration (FDA) has signed off on the chemistry, manufacturing, and controls (CMC) clinical development plan for NeuroSense Therapeutics‘ lead candidate for amyotrophic lateral sclerosis (ALS) ahead of a planned Phase 3 study. The Phase 3 trial of PrimeC is due to start…

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…

An ongoing Phase 3 clinical trial in Europe is testing whether tauroursodeoxycholic acid, added to standard therapy, can slow disease progression and extend survival among people with amyotrophic lateral sclerosis (ALS). The oral compound, also known as TUDCA, is one of the two ingredients in Relyvrio (sodium phenylbutyrate…

NurOwn, Brainstorm Cell Therapeutics’ investigational cell-based therapy, leads to meaningful disease-related biomarker changes regardless of amyotrophic lateral sclerosis (ALS) severity at the start of treatment, according to data from a Phase 3 trial. Specifically, the therapy increased neuroprotection biomarker levels and anti-inflammatory molecules, and reduced those of neurodegeneration…

Corestem is still enrolling participants in its Phase 3 clinical trial of NeuroNata-R (lenzumestrocel), a stem cell therapy that is conditionally approved to treat amyotrophic lateral sclerosis (ALS) in South Korea. The trial, ALSummit (NCT04745299), is evaluating the safety of repeat NeuroNata-R injections and how well…

The first patient has been dosed in a Phase 3 clinical trial (NCT03491462) evaluating the effectiveness of Orphazyme’s investigational therapy arimoclomol in the treatment of amyotrophic lateral sclerosis (ALS). One of the disease mechanisms known to be involved in the development of ALS is protein misfolding and aggregation…

BrainStorm Cell Therapeutics is adding Canadians to a Phase 3 U.S. clinical trial of its ALS stem cell therapy NurOwn. It decided to add the Canadians when a number of them expressed interest in participating, BrainStorm said. The trial (NCT03280056) is investigating the safety and effectiveness of repeated doses…