PureTech has announced positive results from a Phase II trial of KarXT for the treatment of acute psychosis in patients with schizophrenia.

The company announced that in the trial, KarXT demonstrated a statistically significant and clinically meaningful mean reduction in total Positive and Negative Syndrome Scale (PANSS) score, compared to the placebo.

The drug also demonstrated good overall tolerability, and statistically significant reduction in the secondary endpoints of PANSS-Positive and PANSS-Negative scores were also observed.

Further to the findings, KarXT was well tolerated in the Phase II trial, with similar discontinuation rates between KarXT (20%) and placebo (21%), with the number of discontinuations due to treatment emergent adverse events (AEs) equal in the KarXT and placebo arms.

The drug in question is an oral coformulation of xanomeline - a novel muscarinic receptor agonist - and is designed to treat psychosis and related symptoms through preferential stimulation of muscarinic receptors in the central nervous system (CNS).

The trial results are “impressive” and “encouraging”, said Jeffrey Lieberman, professor and chairman of the department of psychiatry, Columbia University, as they “indicate that KarXT, if approved, could represent a game-changing therapeutic advance in the treatment of patients with schizophrenia.”

He continued, “The effectiveness of antipsychotics has been limited by the frequent and serious side effects of first- and second-generation drugs which are difficult for many patients to tolerate, are potentially harmful, and lead to high rates of discontinuation and relapse. In addition to its novel mechanism of action, KarXT could be a new therapeutic option that has the potential to offer robust efficacy devoid of weight gain, metabolic effects and extrapyramidal side effects.”

At least one-third of patients with schizophrenia fail to respond to current treatments, with 74% of patients discontinuing within 18 months of initiation. The World Health Organisation ranks psychosis as the third-most disabling medical condition in the world.