CMTRF funding for CMT treatment from Samsara Therapeutics

The CMT Research Foundation (CMTRF), a non-profit organization focused solely on providing treatments and cures for Charcot-Marie-Tooth disease, has invested in Samsara Therapeutics Inc.

Samsara Therapeutics is a biotechnology company focused on the discovery and development of therapies to restore autophagy – the process by which cells recycle damaged or dysfunctional components. Samsara is developing several novel orally active autophagy activators and has demonstrated that 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 to a project to further optimize pipeline autophagy compounds that could eventually become a treatment option for patients with CMT.

About Charcot-Marie-Tooth

Charcot-Marie-Tooth encompasses a group of hereditary chronic peripheral neuropathies that lead to nerve damage. Patients with CMT suffer from progressive muscular atrophy of the legs and arms leading to problems with walking, running and balance and abnormal functioning of the hands and feet. CMT affects one in 2,500 people (about the same prevalence as multiple sclerosis), including 150,000 Americans and nearly 3 million people worldwide. Currently, there is no treatment or cure for CMT.

Currently, CMT1A is an underserved type with no therapeutic treatment options. The CMTRF seeks to bridge the gap between compelling new science and the development of a new therapeutic approach for patients with CMT1A. CMT1A is caused by the duplication of a segment of DNA that includes the PMP22 gene, and people with CMT1A produce about 33% more PMP22 protein than people without CMT1A.

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

Decreased PMP22 overexpression

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

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

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

Game changing potential

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

“This pipeline asset has the potential to be a breakthrough treatment for CMT patients.”

“This investment from the CMT Research Foundation and our close working relationship with their network of leaders in the field is very promising,” said Peter Hamley, Chief Scientific Officer of Samsara Therapeutics.

“We are pleased with the trust placed in our team and are convinced that we will make significant progress towards the development of a first disease-modifying treatment for patients with CMT1A. There is compelling evidence from human genetics that many diseases are caused by dysfunctional autophagy. Samsara’s mission is to discover new mechanisms capable of restoring autophagy and to deploy new drugs targeting these mechanisms for the treatment of genetically defined diseases like CMT1A.

Samsara Therapeutics Inc. is a biotechnology company based in Oxford, UK and Boston, USA, with a screening platform for induction of autophagy and a growing portfolio of small molecule therapeutics targeting neurodegeneration , rare diseases, cardiology and ophthalmology.

The CMT Research Foundation (CMTRF) is solely focused on providing treatments and cures for CMT. Founded by two patients committed to accelerating drug delivery to people with CMT around the world, the organization funds research for drug development. The federal 501(c)(3) tax-exempt organization is supported by personal and corporate financial donations.