Category: ALS

  • New blow for ALS pipeline as Biogen and Ionis candidate fails Phase 1 trial

    Less than six months after the late-stage failure of tofersen, Biogen and Ionis have suffered further disappointment in the amyotrophic lateral sclerosis (ALS) pipeline after BIIB078 (IONIS-C9Rx) failed to demonstrate clinical benefit in a Phase 1 trial. In the randomised, placebo-controlled, dose-escalating trial, researchers evaluated the efficacy of the investigational antisense oligonucleotide BIIB078 as a […]

  • Amylyx Submits NDA to the US FDA for AMX0035 to Treat Amyotrophic Lateral Sclerosis

    Shots: The NDA is based on CENTAUR trial evaluates AMX0035 vs PBO in 137 patients with ALS The results showed that patients receiving AMX0035 had a significant functional decline at the end of the 6mos. as measured by the ALSFRS-R. In an interim survival analysis @3yrs., 44% lower risk of death, median survival duration (25.0mos. […]

  • Blow to Biogen as ALS hope tofersen flunks phase 3 trial

    Already struggling to make headway with its recently launched Alzheimer’s drug, Biogen suffered a major late-stage pipeline setback after its tofersen drug for amyotrophic lateral sclerosis (ALS) missed the mark in a late-stage study. Tofersen missed its primary objective in the VALOR study, with patients on the drug failing to seen a statistically significant improvement […]

  • Abbisko Signs an Exclusive License Agreement with Sperogenix to Develop ABSK021 for ALS and Other Rare Neurological Diseases in Greater China

    Shots: Abbisko to receive upfront, milestones and royalties on net sales of the therapy & hold the rights for all other countries & indications. Sperogenix gets an exclusive right to develop, manufacture & commercialize ABSK021 in mainland China, Hong Kong SAR and Macao SAR for non-oncology rare neurological diseases Sperogenix will be responsible for the […]

  • Health Innovators: Teresa Arroyo-Gallego, nQ Medical

    In this episode of our Health Innovators series, Paul Tunnah is joined by Teresa Arroyo-Gallego, chief data scientist at artificial intelligence technology company nQ Medical. As a researcher in the biomedical engineering lab of the Massachusetts Institute of Technology (MIT), Teresa leads a team developing the neuroQWERTY technology to detect early signs of motor decline in […]

  • Tackling diabetes & ALS with cell therapy

    Israeli company Kadimastem is developing regenerative medicine therapies based on differentiated cells derived from human embryonic stem cells to treat diseases such as Amyotrophic lateral sclerosis (ALS) and diabetes. Catherine Longworth spoke with Kadimastem’s head of diabetes research, Kfir Molakandov to find out more. In the 1990s, when the first transplant of islets took place, […]

  • BMS joins forces with insitro to develop neurodegenerative treatments

    insitro has landed another big biopharma partnership, signing a five-year collaboration with Bristol Myers Sqibb to develop therapies for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).  Neurodegenerative disorders such as ALS and FTD are considered a challenging therapeutic area, with no disease modifying treatments available today. insitro uses machine-learning technology to discover novel drug […]