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Our science

Our pipeline

We’re working at pace to bring life-changing therapies into the clinic. Our focus is on novel targets and mechanisms with a strong genetic link to dysfunctional autophagy, a hallmark of age-related neurodegenerative and rare monogenic diseases.

Series & Indication
IND Enabling
SAM001 Indication: Parkinson’s Disease, ALS
Target: TRPML1 activator
Progress: IND Enabling

SAM001 is a novel activator of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) also known as Mucolipin-1.  TRPML1 is an ion channel which resides on the lysosome and is vitally important for healthy lysosomal function.  Loss of function mutations of TRPML1 in humans give rise to a serious neurodegenerative disorder known as Mucolipidosis Type IV, which is a lysosomal disease, thus demonstrating in humans how proper functioning of this channel impacts lysosomal and neuronal health.  Our therapeutic, an orally active small molecule with high brain levels, shows great promise in models of Parkinson’s disease and Motor Neuron disease (ALS).

SAM0021 Indication: Parkinson’s Disease
Target: Beclin modulator CNS activator
Progress: Optimisation
Partner: The Michael J.Fox Foundation For Parkinson’s Research

SAM0021 is a series of compounds which activate Beclin1, a key autophagy protein, via a completely new mechanism.  The compounds have high brain levels and show great promise in Parkinson’s and Alzheimer’s disease and are in late stage lead optimization.

SAM0022 Indication: CMT1A
Target: Beclin modulator peripheral
Progress: Optimisation
Partner: CMT Research Foundation

SAM0022 is a series of compounds which activate Beclin1, a key autophagy protein, via a completely new mechanism.  The compounds are related to SAM0021 but are peripherally restricted and have demonstrated efficacy in models of Charcot-Marie Tooth disease Type 1A (CMT1A).  CMT1A is the most common inherited peripheral neuropathy, involving the duplication of the PMP22 gene and subsequent buildup of the PMP22 protein in muscle fibres.

Target: Enzyme activator
Progress: Optimisation

The SAM005 series is a new emerging story for Samsara – inducing autophagy via a completely novel mechanism, the activation of an enzyme known to be involved in diseases caused by protein aggregation.

New LysoSeeker® biology
Progress: Discovery

Samsara’s LysoSeeker® platform allows us to screen thousands of molecules for their autophagy inducing potential and to define their mechanism and therapeutic potential.  When we have identified a promising starting point we use our technologies and expertise to identify and validate its biological target.