Key points from article :
Tranquis Therapeutics' experimental therapy TQS-168 has received orphan drug designation from the U.S. Food and Drug Administration for treating amyotrophic lateral sclerosis (ALS), a rare disease with no effective therapies. This designation provides several incentives to accelerate development, including tax credits, fee exemptions, and potential market exclusivity. The therapy aims to address inflammation, which plays a critical role in ALS's progression.
TQS-168 is designed to modulate a protein called PGC-1a on myeloid cells, which regulate energy metabolism. By normalizing energy metabolism, the drug helps reprogram overactive immune cells, reducing inflammation that damages nerve cells. In mouse models of ALS, the therapy led to reduced inflammatory monocytes and pro-inflammatory molecules, and also extended the mice's lifespan by six days.
When blood samples from ALS patients were treated with TQS-168, the levels of inflammatory monocytes, which are typically high in ALS patients, were reduced. Based on these results, Tranquis initiated a Phase 1 trial in 78 healthy volunteers in the U.K. to evaluate the safety, tolerability, and pharmacokinetics of TQS-168.
The trial, which included both single and multiple ascending doses, showed that TQS-168 was well tolerated with mild, temporary side effects. The therapy also demonstrated a promising pharmacokinetic profile, reaching blood levels linked to therapeutic benefits seen in preclinical models.
Tranquis plans to begin a Phase 2 study of TQS-168 in ALS patients by the end of the year, following positive Phase 1 results. The company is also exploring the therapy’s potential for treating other conditions like frontotemporal dementia, Huntington’s disease, and muscular dystrophy.
