Samsara Therapeutics unveils promising autophagy drug candidate, offering new hope for people with Parkinson's and motor neurone disease

The announcement from Chief Scientific Officer, Peter Hamley, puts Samsara Therapeutics first in the race to find a viable, potent autophagy inducing drug, with the potential to give us longer and healthier lives.   

The company’s autophagy inducing candidate SAM001 will be the first agent of its class to enter clinical trials, with a phase 1 study planned to begin before the end of this year.   

The research shows that SAM001 is able to remove the build-up of cellular proteins that can lead to Parkinson’s disease and amyotrophic lateral sclerosis (ALS) the most common form of motor neuron disease (MND) and restore some of the motor functions affected by the conditions. It does this by targeting Transient Receptor Potential Mucolipin 1 (TRPML1) and boosting autophagy, the vital self-cleaning process present in all cells of the body which eliminates disease causing waste and harmful toxins. Demonstrating a strong affinity for this target, SAM001 has even been shown to reverse disease symptoms in mice^[i].   

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 ^[ii].  

Speaking about the announcement Peter Hamley said, ‘We chose to prioritise 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, like SAM001, 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’s Lysoseeker™ Platform. Lysoseeker™ is a unique suite of technologies capable of screening and evaluating 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 prioritise identifying targets involved in a disease process, and then finding the molecules they interact with.  

Combining Lysoseeker™ with the team’s passion and unparalleled knowledge of new autophagy biology, Samsara aims to bring multiple drug candidates rapidly to the clinic, offering new therapies for rare genetic diseases and neurodegenerative disorders, where there are few or no known treatment options.