Drug candidate provides hope for treating Parkinson's and MND

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

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 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 reduces damage to brain cells taken from people with Parkinson’s Disease and Amyotrophic Lateral Sclerosis (ALS), the most common form of motor neurone disease. It does this by targeting Transient Receptor Potential Mucolipin 1 (TRPML1), a protein important in regulating autophagy. Demonstrating a strong affinity for this target, SAM001 has even been shown to reverse the symptoms of disease in mice.

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.

Chief scientific officer 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. 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 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 prioritise identifying targets involved in a disease process, and then find 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.