ALS

DDW’s Diana Spencer speaks to Peter Hamley, Chief Scientific Officer of Samsara Therapeutics, to understand the role autophagy plays in neurodegenerative diseases and how boosting this process could increase healthspan. DS: Samsara’s autophagy inducing candidate SAM001 will be the first agent of its class to enter clinical trials. What is autophagy? How does SAM001 work? […]

Bloom Science has announced positive topline data from a Phase I clinical trial of BL-001, a potential first-in-class therapeutic the company is developing for Dravet syndrome and amyotrophic lateral sclerosis (ALS). In the trial, BL-001 demonstrated a favourable safety profile and was well tolerated with no serious adverse events (SAEs) across all four dose cohorts. […]

Biogen has revealed an agreement to acquire Reata Pharmaceuticals for approximately $7.3 billion. The news follows an announcement by the company last week that it would cut around 1,000 jobs, or 11% of its workforce, to allow investment into its product pipeline. In its second quarter results, the company said its new ‘Fit for Growth’ […]

A new project by researchers in Edinburgh aims to identify combinations of existing drugs that could be used together to treat motor neurone disease (MND). Led by Prof Siddharthan Chandran, the £3.3 million project is funded by the medical research charity LifeArc, as part of an ongoing partnership between the charity and the UK DRI. […]

BenevolentAI, developers of AI that accelerates biopharma discovery, has delivered BEN-34712, its pre-clinical candidate for the potential treatment of amyotrophic lateral sclerosis (ALS).  BEN-34712 is an oral, potent and selective brain penetrant RARɑ𝛃 (retinoic acid receptor alpha beta) biased agonist and will now enter investigational new drug (IND)-enabling studies.  Impaired retinoic acid signalling has been […]

Not-for-profit medical research organisation LifeArc and techbio company PrecisionLife have agreed a strategic R&D collaboration to accelerate the discovery of targeted treatments for motor neurone disease (MND). MND is also known as amyotrophic lateral sclerosis (ALS) and Lou Gehrig’s disease. The collaboration will accelerate findings by PrecisionLife working with the Motor Neurone Disease Association and […]

Cerevance has expanded its Series B funding round with an additional close of $51 million, bringing the total to $116 million.  The financing will support upcoming clinical trials focused on Parkinson’s disease, Amyotrophic Lateral Sclerosis (ALS), and schizophrenia.   “We are thrilled to secure additional funding from world-class investors who strongly support the need for novel […]

Verge Genomics, a clinical-stage, tech-enabled biotechnology company using artificial intelligence (AI) and human data to transform drug discovery, has announced today that the first subject has been dosed in a Phase I clinical trial of VRG50635 for amyotrophic lateral sclerosis (ALS).  VRG50635 is a small molecule inhibitor of PIKfyve, a novel therapeutic target for ALS […]

News round-up for 3-7 October by DDW Digital Content Editor Diana Spencer This week has brought new trial results and regulatory approvals shedding light and bringing hope into therapy areas of unmet need. The University of Birmingham and King’s College London have furthered our understanding of difficult to treat cancers, while Janssen has revealed encouraging results […]

The US Food and Drug Administration (FDA) has granted approval to AMX0035, a new treatment for people living with amyotrophic lateral sclerosis (ALS). Development of the drug was funded by $2.2 million from the ALS Association, raised by the 2014 social media craze the ‘Ice Bucket Challenge’. Another 40 potential new treatments are being investigated.  […]