Additional promising first-in-class autophagy drug candidate set to enter clinical trials this year bringing hope for Parkinson’s and MND sufferers.

Samsara Therapeutics, a pioneering biotechnology company which was created by Apollo Health Ventures, with founders experienced in the fields of geroscience and cellular homeostasis, has received a grant from The Michael J Fox Foundation for Parkinson’s Research (MJFF), further propelling its research efforts in developing a promising drug for Parkinson’s disease.

This funding, coupled with its groundbreaking candidate SAM001, which is set to enter clinical trials soon, places Samsara at the forefront of autophagy-inducing drug development. By targeting Transient Receptor Potential Mucolipin 1 (TRPML1), SAM001 has exhibited the ability to enhance autophagy and mitigate damage to brain cells affected by Parkinson’s Disease and Amyotrophic Lateral Sclerosis (ALS). This dual achievement positions Samsara as a leading force in addressing the pressing clinical needs of neurodegenerative disorders while extending the potential for longer and healthier lives.

Longevity.Technology: Parkinson’s disease, a neurodegenerative disorder characterized by motor and non-motor symptoms, poses significant challenges to longevity and healthspan. The progressive nature of the disease, with its impact on motor functions and overall quality of life, can significantly reduce both lifespan and the ability to enjoy a healthy and active lifestyle. However, the advancements made by Samsara Therapeutics in autophagy-inducing drug development offer a glimmer of hope – by targeting the underlying cause of Parkinson’s disease through enhancing autophagy (the natural degradation and recycling of cells and their components), these therapeutics could not only slow down the progression of the disease, but also improve overall healthspan.

An oral, daily, disease-modifying treatment like SAM001 or SAM0021 could have a profound impact by reducing treatment complexity, increasing adherence,and ultimately extending the ability of individuals with Parkinson’s disease to lead happier, healthier lives while also reducing the burden of care. These innovative approaches offer the possibility of enhancing longevity and healthspan for those affected by Parkinson’s disease, addressing a significant unmet clinical need in the field of neurodegenerative disorders.

SAM001 has exciting potential for in boosting autophagy and reducing neuronal damage in Parkinson’s Disease and ALS. The drug specifically targets TRPML1, a protein critically involved in autophagy regulation, and has demonstrated a remarkable affinity for this target – even showcasing the ability to reverse disease symptoms in mouse models.

One of the significant challenges in designing SAM001 as a viable treatment was ensuring its bioavailability as an orally administered drug. Samsara Therapeutics has successfully overcome this obstacle by engineering the molecule to pass through the blood-brain barrier efficiently, while minimizing the risk of significant side effects. This achievement paves the way for a once-daily oral regimen, a protocol that would keep things simple for patients, encouraging adherence.

Peter Hamley, PhD, MBA, Chief Scientific Officer at Samsara Therapeutics 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.”

In addition to the progress made with SAM001, Samsara has also landed a grant from The Michael J Fox Foundation. This grant will further accelerate its investigation into SAM0021, another autophagy-inducing agent developed by the company.

SAM0021 has shown promise in removing the build-up of alpha-synuclein, a protein associated with Parkinson’s disease, and restoring motor functions affected by the disease. By enhancing autophagy, a vital cellular process responsible for clearing waste and toxins, SAM0021 addresses the root cause of the disease, and the MJFF grant will allow Samsara Therapeutics to gather more concrete evidence regarding SAM0021’s capabilities, bringing them closer to initiating clinical trials.

Hamley emphasized the importance of the research, stating: “Our research indicates that alpha-synuclein build-up in Parkinson’s disease results from dysfunctional autophagy. We have very encouraging data, including in cells from people with Parkinson’s disease, that SAM0021 can boost autophagy and strongly believe this will allow us to treat the underlying cause of the disease.”

He added that with the support of the MJFF grant, Samsara Therapeutics aims to accelerate its research, achieve proof of concept and advance SAM0021 into clinical trials by the end of 2024.