What is the drug thought to do in the body?
Utreloxastat (PTC857) is thought to block the action of a protein in the body called Lipoxygenase. This enzyme regulates stress, the formation of protein clumps and inflammation in cells. Cell stress and the formation of protein clumps have been shown to cause damage to neurons in MND. Inflammation in the brain and spinal cord is also thought to contribute to MND progression. Reducing the activity of this enzyme in cells may reduce the damage to and death of motor neurons.
Trial outcome
The Phase 2 (cardinALS) trial was investigating Utreloxastat (PTC857) over 24 weeks. PTC Therapeutics reported that the trial did not meet it's primary or secondary endpoints. There was no significant change in ALSFRS-R (from baseline to 24-weeks) for those who received the treatment compared to those who received placebo (dummy drug). The data also showed no significant reduction in a marker of nerve damage called neurofilament light chain (NfL) when comparing Utreloxastat to placebo over 24-weeks. This drug was found to be safe and well-tolerated. Further details on the trial outcome are expected in the coming months.
Previous Clinical Trials
Phase 1
The Phase 1 trial was looking at the safety and tolerability of Utreloxastat in healthy people. It also assessed how the drug moves into, through, and out of the body. Results were announced in December 2022 and showed that the treatment was safe and well-tolerated by healthy participants. You can read more here.
Phase 2
The phase 2 trial was a randomised, double blind, placebo controlled trial. It was looking to investigate the efficacy and safety of Utreloxastat, measured by monitoring the effects of the drug and if there are changes in the ALSFRS-R for people on the treatment. Participants will either given Utreloxastat or placebo twice a day for 24 weeks. Once participants completed the 24 week trial, there was an open label extension where everyone on the trial could choose to receive Utreloxastat for 108 weeks. This trial aimed to recruit 307 participants across the US, Argentina, Europe, Australia and Japan. You can read more here.
Latest News
2024
November 2024- PTC Therapeutics reported that the Phase 2 trial did not meet it's primary or secondary endpoints over the 24 weeks of the trial. You can read more in the press release.
2022
December 2022- Results of the Phase 1 study were announced. The study found that Utreloxastat was safe, well tolerated, and seemed to work as expected in healthy people. You can read more here.
Last updated: 27/11/2024