Samus Therapeutics Launches PU-AD Clinical Program in Alzheimer's Disease

Samus Therapeutics announced the initiation of its clinical program for PU-AD, an oral, brain permeable inhibitor of epichaperomes in Alzheimer's Disease (AD) following clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA). Samus is a privately held, Boston-based, biopharmaceutical company developing multiple novel therapeutics targeting epichaperomes to induce degradation of aberrant proteins driving the pathology of neurodegenerative disease and cancer.

PU-AD is designed to inhibit epichaperomes, complexes of regulatory networks that nucleate on Heat Shock Protein 90 (Hsp90) in diseased cells and maintain and drive the pathologic cellular phenotype.  Epichaperomes protect against the degradation of mutated and aberrant proteins, such as tau, enabling them to stabilize and aggregate. The presence of epichaperomes has been found to contribute to, or possibly initiate, many neurodegenerative diseases, including Alzheimer's Disease. PU-AD specifically inhibits epichaperomes, eliminating aggregation and hyperphosphorylation of tau, affects downstream events associated with the disease, and initiates degradation of mutant tau by a mechanism distinct from other protein degradation platforms. PU-AD has negligible effect, if any, on housekeeping Hsp90 in normal cells.

"Epichaperomes play a critical role in the pathogenesis and characteristics of neurodegenerative diseases and cancers," said Gabriela Chiosis, PhD, a co-founder of Samus Therapeutics and Tri-Institutional Professor at Memorial Sloan Kettering Cancer Center. "Targeting epichaperomes represents one of the most novel and exciting new pathways toward finding new treatments for these diseases."

Samus' clinical program will begin with a single ascending dose (SAD) Phase 1 study to evaluate the safety and tolerability of PU-AD in healthy subjects, with the first subject now dosed, and is expected to be followed by a multiple ascending dose (MAD) cohort. Assuming the expeditious completion of this study, the Company would begin PU-AD clinical testing of Alzheimer's patients in Phase1b/2a in the first half of 2020.

"In AD and tauopathy mouse models, inhibiting the epichaperome with PU-AD effected degradation of mutated hyperphosphorylated and aggregated tau protein," said Barbara Wallner, PhD, Chief Scientific Officer of Samus Therapeutics. "This is supported by a series of memory tests in treated mice, which indicated improved or restored cognitive functions. Acceptance of our IND application and initiation of our clinical program are important steps in validating our approach."

-----------------------------------------------------------------------
Editor's Note:

If you have any suggestion to the content, 

please email: Julia.Zhang@ubmsinoexpo.com