-TQS-168 modulates myeloid immune cells through upregulation of PGC1a, representing a potential new classof treatment for ALS and other neurodegenerative diseases-
REDWOOD CITY, CA—Dec. 8, 2021, Tranquis Therapeutics, a clinical stage private immuno-neurology company developing innovative medicines with the potential to revolutionize the management of neurodegenerative and aging-related diseases today announced the initiation of dosing in a Phase 1 clinical study of TQS-168 in healthy volunteers.
"This is an important milestone for Tranquis as we initiate clinical development of TQS-168, a novel PGC1a modulator that offers great potential as a new class of treatment for ALS patients and a potential advance over approved therapies such as riluzole and edaravone," said Sanjay Kakkar, MD, MSc, MPH, President and CEO of Tranquis. ”It is also a strong proof point for both our team and our approach to developing novel small molecule therapies which reprogram dysfunctional myeloid immune cells to treat some of the most difficult diseases patients and their caregivers face. I want to thank the team as well as our external partners for all their hard work in making tremendous progress. We look forward to moving TQS-168 into a Phase 2 study in the near-term.”
“Our preclinical data show that our differentiated approach to modulating myeloid cell biology has the potential to enable the development of disease-modifying therapies that would benefit patients with a variety of neurodegenerative and other diseases. TQS-168 has shown benefits in various animal models of neurodegenerative disease, including ALS. In vitro, TQS-168 also shows specific immunomodulatory effects in cell lines and white blood cells from patients with ALS,” said Jonas Hannestad, MD, PhD, Chief Medical Officer and Head of R&D at Tranquis. “We believe we are the first company to develop PGC1 a -targeting small molecules for neurodegenerative diseases where dysfunctional myeloid cells play a key role. PGC1a is a transcriptional coactivator that regulates genes involved in cell energy metabolism, acting as a key regulator of mitochondrial biogenesis. We believe PGC1a has enormous potential as a target because when you upregulate it in dysfunctional myeloid cells, it normalizes cell energy metabolism and immune function.”
This Phase 1 clinical trial is being conducted in the United Kingdom and is expected to enroll 68 healthy volunteers. It will test the safety and tolerability of single and multiple doses of TQS-168, characterize the pharmacokinetics of several oral formulations of TQS-168, and evaluate key biomarkers to investigate the relationship between the pharmacokinetics and pharmacodynamics of TQS-168.
Tranquis Therapeutics is a breakthrough biopharmaceutical company focused on developing a portfolio of promising small molecule drugs with a unique mechanism of action, capable of reprogramming dysfunctional myeloid immune cells to revolutionize the management of a broad range of mitochondrial and immune mediated CNS and non-CNS indications and to significantly improve the lives of millions of patients. Founded on groundbreaking neuro-immunology research from the laboratory of Professor Edgar Engleman, MD, at Stanford University, Tranquis’ novel therapies work by restoring normal mitochondrial biogenesis, cell homeostasis and function, effectively “switching” microglia and monocytes from a dysfunctional to a functional state by targeting master regulators of cell energy metabolism. For more information, visit www.tranquis.com.