Rates of age-related neurodegenerative disease are rising. Diseases such as Parkinson’s and Amyotrophic Lateral Sclerosis (ALS) have no effective treatments to halt or prevent their progression. For patients and their families, a diagnosis is devastating. And for health systems worldwide, there are serious challenges in meeting long-term care needs.
A unique suite of technologies, our LysoSeeker® drug discovery platform is capable of screening hundreds of thousands of molecules to identify autophagy-activating compounds in cellular and disease models. Our drug screening campaigns have identified multiple therapeutic candidates that modulate distinct autophagy events through novel and first-in-class mechanisms.
SAM001 is a small molecule in development for treatment of ALS and Parkinson’s disease. SAM001 activates TRPML1, an ion channel expressed on lysosomes and an important regulator of autophagy.
In cultures of neurons derived from patients, SAM001, an oral daily therapy for ALS patients regardless of genotype, demonstrated greater clearance of pathological protein aggregates such as TDP-43 and α– synuclein which are hallmarks of ALS and Parkinson’s. In animal disease models, SAM001 has elicited improvements across various measurements including motor function and survival.
SAM001 clinical strategy will be supported by use of biomarkers that have emerged from our understanding of molecular mode of action. In the early stages of clinical development, this approach enables detection of target engagement and changes in disease-relevant biomarkers in addition to traditional endpoints such as safety and pharmacokinetics.
SAM0021 is a promising treatment for Parkinson’s Disease. Supported by funding from the Michael J. Fox Foundation.
The molecules in the SAM0022 series are related to SAM0021 series however, instead of directly affecting the brain, this series acts on peripheral axons innervating muscles. Molecules in this series have demonstrated efficacy in animal models of CMT disease Type 1A (CMT1A) and the program is in lead optimization stage. Samsara expects to nominate SAM0022 for IND-enabling studies in 2024.
Molecules in the SAM005 series activate autophagy via a completely novel mechanism, the activation of an undisclosed enzyme known to be involved in diseases caused by protein aggregation. This program is in lead optimization stage with proof-of-concept evidence in animal models of the CMT1a disease.
Samsara Therapeutics, the autophagy biotech company, has appointed eminent neurologist and world-leading researcher in Amyotrophic Lateral Sclerosis (ALS)/Motor Neuron Disease (MND) Professor Dame Pamela Shaw to its scientific advisory board.
Samsara Therapeutics announced today that it has received a grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to further its research into a promising Parkinson’s disease drug.
Samsara Therapeutics is first in the race to find a viable, potent autophagy inducing drug, with the potential to give us longer and healthier lives.
