Tranquis Therapeutics Presents Positive Preclinical Data on TQS-168 for the Treatment of ALS at Neuroscience 2021 Annual Meeting

Tranquis Therapeutics, a private immuno-neurology company developing innovative medicines with the potential to revolutionize the management of neurodegenerative and aging-related diseases by reprogramming dysfunctional myeloid immune cells, today announced it presented positive preclinical data on its lead program TQS-168 at the Neuroscience 2021 meeting, which is the annual meeting of the Society for Neuroscience (SfN). This meeting is being held virtually November 8-11, 2021, with Preview Days taking place November 3-7. The data illustrate the potential of TQS-168 for the treatment of amyotrophic lateral sclerosis (ALS). TQS-168 showed increased median survival and decreased pro-inflammatory cytokines and inflammatory monocytes in a widely used SOD1-G93A transgenic mouse model and demonstrated a similar decrease in inflammatory monocytes in blood from ALS patients.

"These data provide convincing evidence that validates our unique approach of developing first-in-class therapeutics that target immune mediated CNS and non-CNS indications by reprogramming dysfunctional myeloid cells, and that our lead program has the potential to become a disease-modifying therapy that would benefit ALS patients," said Jonas Hannestad, MD, PhD, Chief Medical Officer and Head of R&D at Tranquis. "We believe TQS-168 represents a new class of treatment for ALS patients and these data paint a compelling picture which shows, in this preclinical model, a potential advance over approved treatments such as riluzole and edaravone. Based on these findings, we are rapidly advancing TQS-168 into Phase1 clinical studies to assess safety, pharmacokinetics, and pharmacodynamics."

While the pathogenesis of ALS is not fully understood, it's known that inflammation, including activation of myeloid cells, plays a key role and that pro-inflammatory cytokines and monocytes are hallmarks of disease progression.

Poster Presentation Details
Title: The small molecule myeloid cell modulator TQS-168 normalizes the inflammatory phenotype of immune cells from ALS patients and extends survival in the SOD1-G93A ALS mouse model
Presenter: Hope Lancero, Ph.D., Scientist, Tranquis Therapeutics
Date/Time: Tuesday Nov. 9, 2021 at 9:45 AM CT
Poster number: 2021-S-7606-SfN
Session: P230

Key Conclusions:
The effects of TQS-168 were evaluated in vivo in a SOD1-G93A murine model of ALS and ex vivo in blood samples from ALS patients. A 50-mg/kg dose of TQS-168 was administered three times per week in the SOD1-G93A murine model. Blood samples from patients with ALS were incubated for 4 hours at various concentrations of TQS-168.

• A 50 mg/kg dose of TQS -168 administered three times per week in a SOD1-G93A mouse model resulted in:
  • Increased survival when compared to untreated mice (135 vs. 129 days)
  • Decreased plasma levels of pro-inflammatory cytokines and inflammatory monocytes
• In blood samples of patients with ALS, TQS-168 reduced the percentage of CD14^hi CD16^hi pro-inflammatory monocytes, which are increased in blood from ALS patients compared to healthy controls

About Tranquis

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.