Phase 1 Clinical Trial

ProJenX has been given the green light to expand its ongoing Phase 1 clinical trial of prosetin for amyotrophic lateral sclerosis (ALS) across the Atlantic Ocean to testing sites in Europe. The PRO-101 trial (NCT05279755), launched in 2022, is a three-part study evaluating prosetin’s safety, tolerability, and pharmacological…

Verge Genomics has launched a proof-of-concept trial to test whether VRG50635, its oral PIKfyve inhibitor therapy, can be used as a potential treatment for people with amyotrophic lateral sclerosis (ALS). The Phase 1b study will assess the safety and tolerability of escalating doses of VRG50635 — which works…

PathMaker Neurosystems has launched a Phase 1 clinical trial that will test its experimental noninvasive nerve-modulating device in people with amyotrophic lateral sclerosis (ALS). This is the first time that the device — called the MyoRegulator and designed to slow disease progression in ALS — will…

An ongoing Phase 1 clinical trial of QurAlis’ experimental therapy QRL-201 in people with amyotrophic lateral sclerosis (ALS) is expected to open in the U.K. by the end of the year. The announcement follows the trial’s clearance by the country’s Medicines and Healthcare Products Regulatory Agency. “We look…

Treatment with the investigational therapy DNL343 was generally well-tolerated among people with amyotrophic lateral sclerosis (ALS) in an early clinical trial, and biomarker data from the trial suggest that the therapy is working as intended. Results were presented at the annual meeting of the American Academy of Neurology (AAN),…

Utreloxastat (PTC857), an investigational therapy for people with amyotrophic lateral sclerosis (ALS), was safe, well tolerated, and displayed promising pharmacological properties in healthy people, according to a first-in-human Phase 1 study. Based on these findings, the therapy’s developer, PTC Therapeutics, has launched a Phase 2 trial (NCT05349721)…

A Phase 1 clinical trial has begun dosing adult healthy volunteers to investigate QurAlis Corporation‘s QRL-101, an oral treatment candidate for amyotrophic lateral sclerosis (ALS), the company announced. The first-in-human Phase 1 trial (NCT05667779) aims to assess the safety, tolerability, and pharmacokinetics — the movement of a…

Treatment with Coya Therapeutics‘ ALS001, a regulatory T-cell (Treg) therapy being developed for amyotrophic lateral sclerosis (ALS), was found to reduce markers of oxidative stress — a type of cell damage — and inflammation in a small early trial. The experimental Treg therapy was tested in a Phase 1…

Based on data from a Phase 1 clinical trial, Biogen and Ionis Pharmaceuticals are discontinuing development of BIIB078, their experimental treatment candidate for amyotrophic lateral sclerosis (ALS) caused by mutations in the C9orf72 gene. While the therapy was generally safe and well-tolerated in people with C9orf72-associated ALS, it…

NeuroSense Therapeutics will soon begin a Phase 1 clinical trial to investigate the impact of food on PrimeC, a combo medication being developed as a treatment for amyotrophic lateral sclerosis (ALS), in a group of healthy volunteers. The open-label Phase 1 trial (NCT05232461) is expected to enroll…