Funding Received for Phase 2 Trial of Xanomeline-Tropsium Chloride

Wednesday, August 08, 2018


A $42-million financing round has been completed and will be used to finance a phase 2 trial of xanomeline-tropsium chloride (KarXT; Karuna, Boston, MA) for treatment of persons with schizophrenia.

Xanomeline-tropsium chloride has the antipsychotic and procognitive effects of xanomeline without the previously seen side effects related to muscarinic antagonist activity in the periphery (eg, syncope, gastrointestinal tract, sweat glands, and salivary glands). In this formulation, tropsium chloride, a muscarinic agonist that does not cross the blood-brain barrier counteracts the muscarinic effects of xanomeline in the periphery without diminishing its positive effects in the central nervous system. If found safe and effective, this would be the first antipsychotic with a positive rather than neutral or negative effect on cognition.

Using a large body of already existing preclinical data on xanomeline, for which they have a worldwide license, Karuna Pharmaceuticals has taken a relatively unique physiologic approach to drug development. Instead of seeking to chemically modify an experimental drug that demonstrated efficacy in clinical trials and narrow its target to avoid side effects, they are using the normal physiology of the blood-brain barrier and creating combinations that have neutral effects in the periphery (which was the source of previous side effects) and positive effects in the central nervous system.

In addition to the phase 2 clinical trial for treating persons with schizophrenia, Karuna also plans to use this funding to explore potential other uses including other neurologic illnesses in which cognition is affected. Another exciting potential use of xanomeline-tropsium chloride is as a nonopiate treatment for pain. 

“Karuna has all of the characteristics we look for in a game-changing CNS company. Its lead drug candidate has a unique mechanism of action for treating both psychosis and cognition, as well as an exciting nonopiate application for pain,” said Robert Nelsen, Cofounder and Managing Director of ARCH Venture Partners, one of the key investors in this funding round.

Andrew Miller, PhD, Cofounder and CEO of Karuna expressed appreciation for the investors and noted, “. . . KarXT could be a promising new treatment that safely and effectively addresses the debilitating positive, negative, and cognitive symptoms in patients living with schizophrenia and other central nervous system disorders.”

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