CMT Research Foundation funding boosts Samsara Therapeutics Charcot-Marie-Tooth treatment

October 13, 2022 - 4 minutes
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The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, has invested in Samsara Therapeutics Inc.

Samsara Therapeutics is a biotech company focusing on discovering and developing therapeutics to restore autophagy – the process by which cells recycle damaged or dysfunctional components.   Samsara is developing several novel, orally active autophagy enhancers and demonstrated they can rescue CMT1A neuropathy in mice in a dose-dependent manner.

The partnership with Samsara Therapeutics represents CMTRF’s largest investment to date and is dedicated toward a project to further optimize pipeline autophagy compounds that may eventually become a treatment option for CMT patients.  

About Charcot-Marie-Tooth

Charcot-Marie-Tooth encompasses a group of inherited, chronic peripheral neuropathies that result in nerve degradation. CMT patients suffer from progressive muscle atrophy of legs and arms causing walking, running and balance problems and abnormal hand and foot functioning. CMT affects one in 2,500 people (about the same prevalence as multiple sclerosis), including 150,000 Americans and nearly 3 million people worldwide. At the moment, there is no treatment or cure for CMT.

Currently, CMT1A is an underserved type with no therapeutic treatment options.  CMTRF seeks to bridge the divide between novel, compelling science and the development of novel therapeutic approach for CMT1A patients. CMT1A is caused by duplication of a stretch of DNA that includes the PMP22 gene, and people with CMT1A produce approximately 33% more PMP22 protein than people who do not have CMT1A. 

While the mechanism of disease is not known precisely, this overproduction of PMP22 impedes the normal inclusion of PMP22 in the myelin sheath produced by Schwann cells in the peripheral nerves, leading eventually to demyelination, axon loss, and disability. One hypothesis about the disease is that the extra PMP22 protein clumps together to form aggregates that are toxic.  

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Decreasing PMP22 overexpression

Samsara Therapeutics has shown its pipeline asset significantly decreases PMP22 overexpression by stimulating autophagy in axons and myelinating Schwann cells, thus restoring neuromuscular impairment and sciatic nerve electrophysiology in preclinical testing.   

“Samsara has discovered a number of autophagy enhancers and they have studied one series in CMT1A model mice with very promising results,” says Keith Fargo, chief scientific officer of the CMTRF. 

“This project is designed to further optimize these compounds, then scale up manufacturing of the candidate, and subject it to profiling studies to determine if the potential therapeutic has favorable drug properties for first-in-human dosing in clinical trials.” 

Game-changing potential 

“To our knowledge, Samsara Therapeutics is the only company so far pursuing an approach that targets autophagy-induced restoration of neuromuscular function in CMT1A,” says Cleary Simpson, CEO of the CMTRF. 

“This pipeline asset has the potential to be a game-changing therapeutic for CMT patients.” 

“This investment by The CMT Research Foundation and our close working relationship with their network of leaders in the field shows great promise,” says Peter Hamley, chief scientific officer of Samsara Therapeutics. 

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“We are gratified by the trust placed in our team and are confident that we will make significant progress towards developing a first disease-modifying therapeutic for patients living with CMT1A.There is compelling evidence from human genetics that many diseases are driven by autophagy dysfunction. Samsara’s mission is to discover new mechanisms which can restore autophagy and deploy new drugs targeting these mechanisms for the treatment of genetically defined diseases like CMT1A.”

Samsara Therapeutics Inc. is an Oxford, U.K. and Boston, U.S.-based biotechnology company with a screening platform for autophagy induction and a growing pipeline of small molecule therapeutics targeting neurodegeneration, rare diseases, cardiology, and ophthalmology.  

The CMT Research Foundation (CMTRF) is focused solely on delivering treatments and cures for CMT. Founded by two patients who are driven to expedite drug delivery to people who live with CMT globally, the organization funds research for drug development. The 501(c)(3) federal tax-exempt organization is supported by personal and corporate financial gifts.

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