News

The HEALEY ALS platform trial, the first study to test several therapy candidates for amyotrophic lateral sclerosis (ALS) simultaneously, has completed enrollment for at least two of its first three regimens. These include Clene Nanomedicine’s CNM-Au8 (NCT04414345) and Biohaven Pharmaceuticals’ verdiperstat (NCT04436510). The recruitment status…

A first patient has been enrolled in a Phase 3 clinical trial investigating FNP122, an oral formulation of edaravone being developed by Ferrer to treat amyotrophic lateral sclerosis (ALS). The ADORE trial aims to enroll 300 patients across Europe to assess the safety and efficacy of FNP122, as well as its…

TQS-168, an investigational therapy being developed by Tranquis Therapeutics, reduced inflammation in mouse and human models of amyotrophic lateral sclerosis (ALS), and prolonged survival in the mice, according to a preclinical study. Based on these findings, Tranquis is moving the therapy into Phase 1 clinical studies. “We believe…

The burden of caregiving is heavier for people who are anxious and reasonably knowledgeable about amyotrophic lateral sclerosis (ALS), a study of patients and their caregivers in China suggests. The study, “Caregiver burden and associated factors among primary caregivers of patients with ALS in home care:…

Amylyx Pharmaceuticals‘ global Phase 3 clinical trial investigating AMX0035 as an oral treatment for amyotrophic lateral sclerosis (ALS) has begun dosing participants. The PHOENIX trial (NCT05021536) is expected to include 600 participants whose symptoms began in the past two years, which is a less-stringent criteria than was required for…

A small open-label study evaluating the epilepsy medication perampanel in adults with amyotrophic lateral sclerosis (ALS) was halted due to adverse events that affected behavior. Despite the findings, larger clinical trials investigating perampanel in ALS patients are ongoing, which might determine if the medication contributed to behavioral side effects. The…

AC Immune has extended its research collaboration with scientists at the University of Pennsylvania (UPenn) working to uncover how TDP-43 protein clumps contribute to different disease manifestations in amyotrophic lateral sclerosis (ALS). In the partnership’s first two years, researchers with the Perelman School of Medicine at UPenn received…

CNM-Au8, an investigational disease-modifying therapy for people with early amyotrophic lateral sclerosis (ALS), failed to prevent motor neurons loss and lung function decline relative to a placebo, according to top-line data from the RESCUE-ALS Phase 2 clinical trial. The trial, in other words, did not meet its primary…

The United to End MND campaign in the United Kingdom is “disappointed” the nation’s government did not accept its plea to invest £50 million (about $68 million) in targeted motor neuron disease (MND) research. MNDs are a group of progressive neurological disorders that includes amyotrophic lateral sclerosis, commonly…

Amylyx Pharmaceuticals is seeking approval in the U.S. for AMX0035, its investigational therapy for slowing functional decline in people with amyotrophic lateral sclerosis (ALS). The company submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for regulatory review. The decision had been…