Amylyx Pharmaceuticals has raised $135 million to advance the clinical development and the potential launch of its lead candidate AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). The Series C financing round, led by Viking Global Investors, also will be used to support further research and…

Dosing has begun in a clinical trial of WVE-004, Wave Life Sciences‘ investigational therapy for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) associated with C9orf72 gene mutations. “ALS and FTD are devastating illnesses where therapeutic progress has been extremely limited. Advancing discovery and development of new treatments…

A subsidiary of Sperogenix Therapeutics has acquired exclusive rights to develop and commercialize ABSK021, an investigational oral therapy for amyotrophic lateral sclerosis (ALS) and other rare neurological diseases in mainland China, Hong Kong, and Macao. Under the terms of the agreement between Sperogenix MedTech and Abbisko Therapeutics, the therapy’s…

Treatment with the investigational oral therapy masitinib can extend lifespan when given early in the course of amyotrophic lateral sclerosis (ALS), according to a new study. “These long-term survival data, with an average follow-up of 75 months since diagnosis, suggest that masitinib can offer a substantial survival benefit…

AB Science’s proposed measures to ensure patient safety in clinical trials of its experimental oral therapy masitinib were deemed sufficient by the French National Agency for Medicines and Health Products Safety (ANSM), and patient enrollment in the country may resume. The announcement comes less than two months after…

In keeping with its commitment to turn amyotrophic lateral sclerosis (ALS) into a “livable disease” by 2030, and to ramp up discovery and funding of new therapy candidates, the ALS Association is moving to a unified structure from a federated one. “Our best opportunity to fulfill this promise and…

Verge Genomics and Eli Lilly have entered into a three-year collaboration to find and develop therapies for amyotrophic lateral sclerosis (ALS). Verge will focus on discovering and validating potential new therapeutic targets, while Lilly will select up to four candidates to advance through clinical testing with a goal of…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Prilenia’s pridopidine for the treatment of amyotrophic lateral sclerosis (ALS). The decision follows a recent positive opinion from a branch of the European Medicines Agency recommending the treatment be given orphan drug status in…

Ubiquitination — the cell’s process of tagging proteins, often to degrade unwanted ones — is essential for the disassembly of stress granules that, by not being cleared, damage cells in amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, a study showed. The study, “Ubiquitination is essential for…

Coya Therapeutics’ ALS001, a potential regulatory T-cell (Treg) therapy designed to halt amyotrophic lateral sclerosis (ALS) progression, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA). Furthermore, a Phase 2a trial (NCT04055623) testing ALS001 versus a placebo in 12 ALS patients…