News

A large Phase 3 trial designed to confirm the safety and efficacy of the approved therapy Relyvrio (sodium phenylbutyrate and taurursodiol) for amyotrophic lateral sclerosis (ALS) is now fully enrolled. Amylyx Pharmaceuticals announced the completion of patient recruiting for the trial, called PHOENIX (NCT05021536), which will…

Note: This story were updated Feb. 14, 2023, to correct that ICER analyses found both Radicava ORS and Relyvrio are not cost-effective for the treatment of ALS. The recently approved therapies Relyvrio (sodium phenylbutyrate and taurursodiol) and …

uniQure has entered an agreement to acquire the full rights to Apic Bio‘s investigational gene therapy APB-102, which is being developed for amyotrophic lateral sclerosis (ALS) associated with SOD1 mutations. uniQure now is planning to start a Phase 1/2 clinical trial of APB-102 later this year. Under the…

Cytokinetics has awarded $20,000 grants to five patient advocacy organizations, including two serving the amyotrophic lateral sclerosis (ALS) community, under its fifth annual Communications Grant Program. Each grant is intended to support the nonprofit’s outreach, engagement, and awareness efforts, allowing the organizations to broaden their work in underserved…

A new pig model of amyotrophic lateral sclerosis (ALS) more accurately reflects the gradual motor neuron loss and disease progression seen in patients, and it may speed the development of ALS treatments, included gene and cell-based therapies, researchers report. The model carries a mutation in the SOD1 gene, an…

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)…

Elevated levels of high-density lipoprotein (HDL) — commonly called the “good” cholesterol — are significantly associated with a poorer survival rate among people with amyotrophic lateral sclerosis (ALS), according to a population-based study in the Netherlands. In contrast, levels of total cholesterol and low-density lipoprotein…

The U.S. Food and Drug Administration (FDA) has announced an advisory committee meeting seeking advice on whether to approve Biogen‘s investigational therapy tofersen for the treatment of amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The public meeting, to be held online March 22, by…

Neopharm has acquired exclusive rights to commercialize AMX0035 (sodium phenylbutyrate and taurursodiol) in Israel, Gaza, the West Bank, and to the Palestinian Authority as a treatment of amyotrophic lateral sclerosis (ALS). The company’s agreement with Amylyx Pharmaceuticals makes it responsible for the distribution and marketing of AMX0035…

An oral suspension form of Radicava (edaravone), an approved treatment of amyotrophic lateral sclerosis (ALS), was favored for patient reimbursement in Canada by a branch of the Canadian Agency for Drugs and Technologies in Health (CADTH). The opinion issued by the CADTH Canadian Drug Expert Committee recommended that Radicava Oral…