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Samsara Therapeutics Unveils Promising Autophagy Drug Candidate

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Samsara Therapeutics, a biopharmaceutical company that aims to discover therapeutics to treat rare genetic and neurodegenerative disease, has announced the company’s autophagy inducing candidate SAM001, the first agent of its class to enter clinical trials, with a phase 1 study planned to begin before the end of the year. 


Autophagy is how our cells self-clean to remove waste and prevent the build-up of toxic material. The process slows with age, and if damaged, is a contributing factor in many serious diseases. Since the discovery that boosting autophagy could extend lifespan and counteract chronic, age-related diseases, the field has attracted significant research and investment but has, until now, failed to generate any new therapies.


The research presented shows that SAM001 boosts autophagy and improves brain activity in mice and in cells taken from people with Parkinson’s Disease and Amyotrophic Lateral Sclerosis (ALS), the most common form of motor neurone disease. SAM001 targets Transient Receptor Potential Mucolipin 1 (TRPML1), a protein important in regulating autophagy.

In designing SAM001 as a once daily, oral treatment, Samsara has also overcome substantial bioavailability challenges, engineering the molecule to ensure that the required dose is able to pass through the blood brain barrier without causing significant side-effects.

Speaking about the announcement Peter Hamley said, ‘We chose to prioritize investigating autophagy targets in neurodegenerative disorders as there is such a huge unmet clinical need in this area, and very few therapies that delay or slow the progression of these diseases by targeting their underlying cause. A once daily, oral, disease-modifying treatment, could have a tremendous impact’, he continued. ‘Reducing treatment complexity, increasing adherence, improving quality of life for people with Parkinson’s and ALS, extending their ability to remain in the workforce and reducing the cost of care.’

SAM001 was discovered using Samsara Therapeutics’ Lysoseeker™ Platform. Lysoseeker™ is a unique suite of technologies capable of screening and testing the autophagy boosting potential of thousands of molecules, to identify drug candidates, their mechanisms, and the cell targets they interact with. The platform is a break away from traditional processes for drug discovery which typically prioritize identifying targets involved in a disease process, and then finding the molecules they interact with.