Boehringer Ingelheim's Schizophrenia Drug iclepertin Fails Phase 3 Trial

Boehringer Ingelheim announced that its schizophrenia drug, iclepertin, did not achieve statistically significant improvements in cognition or functioning in its Phase 3 trial. As a result, the trial failed to meet both its primary and secondary endpoints.

Overall, no statistically significant effects on cognition or functioning were observed in patients treated with iclepertin versus placebo at six months. All three trials demonstrated that iclepertin, a glycine transporter 1 (GlyT1) inhibitor, was generally well tolerated, with a safety profile that remains consistent with previous studies.

“While these findings are disappointing, we remain dedicated to finding effective solutions for those living with serious mental illnesses. Our innovative pipeline includes over 20 additional investigative therapies in all stages of development and in different disease areas including schizophrenia and major depressive disorder,” said Shashank Deshpande, Member of the Board of Managing Directors and Head of Human Pharma at Boehringer Ingelheim. “In the near future more can be expected. Our gratitude goes to the patients and clinical investigators whose time and commitment made this research possible.”

CONNEX was the largest program for cognitive impairment in schizophrenia to date. Boehringer believes that the results will aid scientific understanding and inform future research for patients living with cognitive impairment in schizophrenia, an area of high unmet need with no approved targeted pharmaceutical treatment options available. Full efficacy and safety data will be submitted for presentation at an upcoming medical meeting.

About cognitive impairment in schizophrenia

Schizophrenia affects approximately 24 million people worldwide. Cognitive impairment is one of the three core symptom domains of schizophrenia, along with positive and negative symptoms. Cognitive impairment affects more than 80% of people with this condition, manifesting early in patients’ lives, and representing a leading cause of disability and impairment in daily functioning. People with schizophrenia often face cognitive challenges that impact their problem-solving abilities, attention and memory. These problems can make it difficult to live an independent and productive life. They can also cause a person to struggle with simple tasks such as attending appointments, which can make treatment difficult.

There are no approved targeted pharmaceutical treatments for cognitive impairment in schizophrenia.

About iclepertin and the CONNEX clinical program

Iclepertin (BI 425809), an investigational oral inhibitor of glycine transporter 1 (GlyT1), was studied as a potential treatment for cognitive impairment in adults with schizophrenia. Iclepertin has not been approved by any regulatory authorities.

CONNEX was a Phase III clinical program designed to assess the safety and efficacy of iclepertin for improving cognitive impairment in adults with schizophrenia. The program comprised three clinical trials, all of which were randomized, double-blind, placebo-controlled parallel group trials investigating treatment with oral iclepertin 10 mg once daily over a 26-week period in adults with schizophrenia who received stable antipsychotic treatment. The CONNEX clinical program enrolled 1,840 patients in 41 countries.

Boehringer Ingelheim decided to discontinue the long-term extension trial, CONNEX-X, effective immediately.