Boehringer Ingelheim’s investigational schizophrenia candidate has failed to improve cognitive impairment or functioning in a trio of late-stage trials.
The phase 3 program missed both primary and key secondary endpoints, with no statistically significant effects on relevant measures observed for patients receiving investigational treatment compared to placebo at six months, according to a Jan. 16 release.
Given the results, Boehringer is discontinuing a related long-term extension study effective immediately.
Boehringer’s phase 3 program, called CONNEX, was made up of three double-blinded and placebo-controlled studies evaluating oral iclepertin, an investigational GlyT1 inhibitor. The program enrolled 1,840 adults with schizophrenia who received stable antipsychotic treatment. It was the largest trial program of its kind in patients with cognitive impairment tied to schizophrenia, according to Boehringer.
The FDA had granted iclepertin a breakthrough therapy tag for the indication, citing the lack of targeted pharmaceuticals currently available for cognitive impairment in schizophrenia.
“While these findings are disappointing, we remain dedicated to finding effective solutions for those living with serious mental illnesses,” Shashank Deshpande, Boehringer’s head of human pharma, said in the release. “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. In the near future, more can be expected.”
Boehringer plans to present full data from the late-stage program at an upcoming medical conference.
The clinical flop comes just a few days after Johnson & Johnson’s $14.6 billion buyout of Intra-Cellular Therapies. In that deal, the pharma giant picked up the CNS biotech’s schizophrenia and bipolar treatment Caplyta.
On the heels of the deal—biopharma’s biggest acquisition since Novo Nordisk’s $16.5 billion Catalent buy in February 2024—Alto Neuroscience founder and CEO Amit Etkin, M.D., Ph.D., argued that there's a need for new approaches in the neuro sector. Caplyta, first approved in 2019, represents "more of the same" in the traditional antipsychotic space, he said.
“With all the recent failures across companies, how do we actually go forward in this high-need place?” Etkin told Fierce Biotech at the J.P. Morgan Healthcare conference yesterday. Going into 2025, the biotech’s mantra is to “rethink everything,” the CEO said. That includes reimagining how drug development happens, reconsidering challenges in trial execution and conduct that has plagued the field, and rethinking how tech is leveraged.