The HEALEY ALS platform trial, which is evaluating the safety and efficacy of several potential treatments for amyotrophic lateral sclerosis (ALS), has passed the 50% participant enrollment target for its first three regimens. The investigational treatments are UCB’s zilucoplan, Biohaven Pharmaceuticals’ verdiperstat, and Clene Nanomedicine’s CNM-Au8. A fourth candidate…

“Have you ever participated in clinical trials?” That’s a common question among ALS patients. What’s my standard answer? “It’s complicated. I’m participating in several studies, but even though I’d like to, I haven’t been in any clinical trials. I’m excluded because I’ve had ALS for too many years.” ‘What? Tell…

Mitsubishi Tanabe Pharma America (MTPA) is taking proactive measures to address patient safety in its REFINE-ALS biomarker study during the COVID-19 pandemic. The company announced revisions to the study’s protocol that give participants the option to participate virtually, through the use of telemedicine (medical care delivered virtually) and…

Cytokinetics is preparing a global Phase 3 trial that will enroll more than 500 people with early amyotrophic lateral sclerosis (ALS) to test its investigational therapy reldesemtiv in slowing progressive muscle weakness, the company announced. Details from the trial design were shared at the 31st International Symposium…

The first patient has been dosed in a Phase 2a clinical trial evaluating the safety and effectiveness of ALZT-OP1a, a dry-powder inhaled formulation of cromolyn, in people with mild-to-moderate amyotrophic lateral sclerosis (ALS). The trial (NCT04428775) is recruiting up to 80 patients, ages 18–75, who have been…

Patient enrollment is now complete for the Phase 2 clinical trial of Clene Nanomedicine‘s CNM-Au8 as a potentially disease-modifying therapy for amyotrophic lateral sclerosis (ALS). The trial, named RESCUE-ALS (NCT04098406) will test the safety, efficacy, pharmacokinetics, and pharmacodynamics —…

The greater incidence of amyotrophic lateral sclerosis (ALS) among professional athletes and people with head trauma, and the faster functional decline seen in animal models after localized nerve injury, strongly suggests that nerve injury plays a role in initiating disease processes, a review study reported. Needed avenues of future…

Kadimastem’s cell therapy candidate, AstroRx, significantly lowers the rate of disease progression in people with amyotrophic lateral sclerosis (ALS) for at least three months after treatment, updated findings from the company’s Phase 1/2 clinical trial show. The company already had reported promising…

Tofersen, Biogen’s investigational therapy for people with familial amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations, was safe and generally well-tolerated over three months, and appeared to lower SOD1 protein levels in the central nervous system, a Phase 1/2 clinical trial has found. There…