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Our science


Our first-in-class autophagy-inducing agent SAM001 targets the dysfunctional cell processes seen in Parkinson’s and ALS patients. It’s designed as a once-daily, oral treatment, with Phase 1 trials due to start in 2025*.

*deck indicates that IND submission & CTA filing will occur in 2024, with study initiating in 2025

How SAM001 works in ALS patients

One of the hallmarks of Amyotrophic Lateral Sclerosis is the mislocalization of TDP43 protein. TDP43 should be present only in the nucleus of cells, but in ALS patients it is detected in the cell body in a patient’s nervous system. This correlates with the classic symptoms of the disease. It is believed that this mislocalization of TDP43 is caused by lysosomal misprocessing, in turn thought to be caused by dysfunctional autophagy. There is strong genetic evidence for this hypothesis.

Our research shows that SAM001 boosts autophagy and reduces damage to neurons derived from people with ALS and Parkinson’s disease. In vivo models show that SAM001 also has profound effects on ALS survival rates, as well as restoring motor function.

SAM001’s effects

Restoring autophagy

Before After

Brain motor neurons derived from the stem cells of ALS patients show reduced autophagy and lysosome levels versus healthy controls

Treated with SAM001, ALS motor neurons show increased autophagy and lysosome levels

Blue = DAPI (nuclei)
Red = LAMP1 positive lysosomes
SAM001’s effects

Removing mislocalized TDP43

Before After

ALS patient motor neurons display mislocalized (cytosolic) TDP43

Treated with SAM001, ALS patient motor neurons show a significant reduction in levels of mislocalized (cytosolic) TDP43

Red = SMI-32 (motor neurons)
Blue = DAPI (nucleus)
Green = TDP43 (pTDP43 antibody)