News

OrphAI’s AIT-101 granted orphan drug status in Europe for ALS

OrphAI Therapeutics’ experimental therapy AIT-101 has been awarded orphan drug status in the European Union for amyotrophic lateral sclerosis (ALS), following a similar designation granted in the U.S. last year. In Europe, orphan drug status is given to medicines designed to treat life-threatening or chronically debilitating conditions affecting…

Committee favors EU approval of Qalsody for adults with SOD1-ALS

An advisory committee of the European Medicines Agency (EMA) has recommended that Biogen’s Qalsody (tofersen) be approved in the European Union under exceptional circumstances to treat adults with amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations. The positive opinion from the Committee for Medicinal Products for Human Use,…

Phase 1 trial testing prosetin for ALS expands to EU countries

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…

Denali therapy candidate fails to slow ALS progression in trial

Denali Therapeutics’ SAR443820 (DNL788) treatment candidate — now in late-stage clinical testing for amyotrophic lateral sclerosis (ALS), per the company’s website — has failed to significantly slow disease progression in adults in a Phase 2 trial. The experimental therapy “did not meet the primary endpoint of change” in outcomes…

Experimental gene therapy CTx1000 extends survival in mice

An experimental gene therapy called CTx1000, which is designed to reduce toxic buildups of the protein TDP-43 in nerve cells, was found to significantly extend survival and improve motor function in mouse models of amyotrophic lateral sclerosis (ALS) and the related disorder frontotemporal dementia, known as FTD. According to…

TPN-101 improves ALS/FTD caused by C9orf72 gene mutations

TPN-101, an investigational oral molecule developed by Transposon Therapeutics, reduced multiple biomarkers of neuroinflammation and neurodegeneration, and slowed respiratory decline in some people with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD). That’s according to an interim analysis of a Phase 2a clinical trial (NCT04993755) that’s testing…

ALS Association granted $58M in largest single donation ever

An unprecedented $58 million donation to the ALS Association is a “beacon of hope” for the amyotrophic lateral sclerosis (ALS) community and will finance treatment centers, aid to financially strapped patients, and clinical trials of promising treatments, according to the U.S.-based nonprofit. The donation from the estate…