Key points from article :
Tranquis Therapeutics has concluded its Phase I clinical trial of TQS-168, a drug being developed to treat amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The trial involved 78 healthy participants, testing both single and multiple doses of TQS-168. The drug, a small molecule modulator of PGC-1a, has previously shown benefits in animal models of ALS and Parkinson's disease, including enhanced gene expression and a reduction in inflammatory markers.
The results from the trial showed that TQS-168 was well tolerated by the participants, with no serious adverse events observed. While some mild and transient side effects were reported, they did not lead to discontinuation of the treatment. The drug also demonstrated favorable pharmacokinetic properties, with satisfactory plasma exposures.
In laboratory tests, TQS-168 showed specific immunomodulatory effects, particularly in the white blood cells of ALS patients. These findings support the drug's potential to target dysfunctional myeloid cells, which play a critical role in the progression of neurodegenerative diseases.
Based on these results, Tranquis plans to begin a Phase II trial of TQS-168 for ALS by the end of the year. The company's chief medical officer, Jonas Hannestad, expressed confidence in the drug's potential, noting that the Phase I trial data align with preclinical findings.
The company is also developing a second drug, TQS-621, and aims to use the insights from the ALS program to further develop these treatments for other diseases involving myeloid cell dysfunction. This trial was published in June 2022, but the specific journal was not mentioned in the source.
