REDWOOD CITY, Calif.--(BUSINESS WIRE)--Tranquis Therapeutics, Inc., a clinical stage immuno-neurology company developing innovative medicines with the potential to revolutionize the management of neurodegenerative and aging-related diseases, today announced the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for TQS-168, a small molecule modulator of PGC-1a, for the treatment of amyotrophic lateral sclerosis (ALS). ALS is a devastating disease with a high unmet need. Tranquis expects to begin a Phase 2 study of TQS-168 in people living with ALS by the end of 2022.
The FDA’s Office of Orphan Drug Products grants orphan status to drugs and biologics that demonstrate promise for the treatment of diseases or conditions affecting fewer than 200,000 people in the United States. Orphan drug designation provides Tranquis with certain development incentives, including tax credits for qualified clinical trials, exemptions from FDA user fees, and the potential for seven years of market exclusivity, if TQS-168 is approved.
“The orphan drug designation is an important regulatory milestone for Tranquis, further validating our efforts to develop TQS-168, an orally administered compound that targets PGC-1a in myeloid cells, as a novel therapeutic option for people living with ALS,” said Jonas Hannestad, MD, PhD, Chief Medical Officer at Tranquis. “Earlier this month, we reported Phase 1 data on TQS-168 in 78 healthy volunteers demonstrating excellent pharmacokinetic properties and blood plasma exposures associated with clinical benefit in preclinical models, along with a favorable safety and tolerability profile. We look forward to rapidly advancing our Phase 2 program in ALS.”
About TQS-168 and PGC-1a
TQS-168 is a PGC-1a-targeting small molecule in development for the treatment of ALS and other neurodegenerative diseases in which dysfunction of myeloid cells plays a critical role. PGC-1a is a transcriptional coactivator that regulates genes involved in cell energy metabolism, acting as a key regulator of mitochondrial biogenesis. When upregulated in dysfunctional myeloid cells, PGC-1a normalizes cell energy metabolism and immune function, giving it potential as a target in a variety of neurodegenerative diseases. TQS-168 showed survival and functional benefits in various animal models of neurodegenerative disease, including ALS and Parkinson’s disease. TQS-168 also demonstrated compelling biomarker data, including increased PGC-1a gene expression, reduction of inflammatory monocytes and cytokines, and reduced levels of neurofilament light in animal models. In vitro, TQS-168 also shows specific immunomodulatory effects in myeloid cell lines and in white blood cells from people living with ALS.
Tranquis is a clinical-stage biopharmaceutical company developing a portfolio of small molecule therapeutics based on its unique approach of reprogramming dysfunctional myeloid immune cells to restore normal cell homeostasis and function. Tranquis’ therapeutic candidates can “switch” dysfunctional microglia and monocytes back to a homeostatic functional state by targeting master regulators of cell energy metabolism, such as PGC-1a. Tranquis’ platform has the potential to revolutionize the management of an entire range of indications in the fields of neurodegeneration, mitochondrial diseases, immuno-inflammation, and immunosenescence. In addition, the Company’s approach may promote healthy aging and longevity.