A Phase 3 clinical trial that’s testing FNP122, Ferrer’s oral formulation of edaravone, in people with amyotrophic lateral sclerosis (ALS) is now rolling participants into its open-label extension study. The ADORE trial (NCT05178810) is testing FNP122 against a placebo in about 300 patients across Europe whose…
News
VRG50635, an experimental oral therapy being developed by Verge Genomics for amyotrophic lateral sclerosis (ALS), was found to be safe and well-tolerated in healthy volunteers, new Phase 1 trial data show. Top-line data from the trial (ISRCTN14792372) also showed a promising tolerability and pharmacological profile supporting once-daily…
The Committee for Medicinal Products for Human Use (CHMP) has issued an opinion opposing Amylyx Pharmaceuticals’ bid for conditional European Union approval of AMX0035, its oral treatment to slow the progression of amyotrophic lateral sclerosis (ALS). The negative recommendation from the advisory committee, an arm of the…
The first healthy volunteer has been dosed in a Phase 1 clinical trial evaluating VTX3232, Ventyx Biosciences’ investigational oral therapy for amyotrophic lateral sclerosis (ALS) and other neurodegenerative conditions. Top-line data from the trial are expected in the first half of 2024. “VTX3232 is our fourth internally discovered…
Magnetic stimulation restored the health of lab-grown motor neurons — the muscle-controlling nerve cells that die in amyotrophic lateral sclerosis (ALS) — derived from people with familial ALS, according to a proof-of-concept study. The approach improved several biological processes in motor neurons, including the movement of cellular components along…
Albrioza, the approved treatment for amyotrophic lateral sclerosis (ALS) formerly known as AMX0035, is one giant step closer to being eligible for reimbursement under public drug plans in Canada, according to its developer, Amylyx Pharmaceuticals. Amylyx has reached an agreement with the pan-Canadian Pharmaceutical Alliance, or pcPA —…
PEG10, an ancient, virus-like protein that supports the development of the placenta, appears to also participate in the nerve cell loss associated with amyotrophic lateral sclerosis (ALS), researchers discovered. ALS-causing mutations in the UBQLN2 gene were found to prevent the proper degradation of PEG10. This led to a…
BenevolentAI is advancing BEN-34712, its experimental oral therapy for amyotrophic lateral sclerosis (ALS), to late-stage preclinical studies. The studies are expected to support a future investigational new drug (IND) application, a formal request to the U.S. Food and Drug Administration (FDA) to test a potential new treatment in clinical…
QurAlis soon expects to open its ongoing Phase 1 clinical trial testing QRL-201 in people with amyotrophic lateral sclerosis (ALS) at sites in various European Union countries, including Germany, Belgium, the Netherlands, and Ireland. The company’s announcement comes after these countries reviewed and accepted an application for…
To better understand amyotrophic lateral sclerosis (ALS) and improve patient care, Mitsubishi Tanabe Pharma America (MTPA) is partnering with the ALS/MND Natural History Consortium in collecting real-world data on people with the progressive neurodegenerative disorder. The collaboration will give MTPA access to the consortium’s data repository,…
Recent Posts
- Developers expand collaboration to test 2 novel targets for ALS treatments
- Trial testing new ALS gene therapy starts dosing patients in Australia
- In life with ALS, we find moments of ‘genius’ to keep our minds active
- Brain imaging agent shows promise for detecting toxic ALS protein
- I reframed my life with ALS by becoming a calmer duck