Karuna Pharmaceuticals targeting schizophrenia with new discoveries licensed from Vanderbilt University

By Scott Kirsner, Globe Columnist

Boston-based Karuna Pharmaceuticals is set to announce this week that it has licensed a set of scientific discoveries from Vanderbilt University's Center for Neuroscience Discovery. The start-up hopes to develop them into effective new drugs for schizophrenia, a disabling mental illness that affects more than 24 million people worldwide.

A problem with existing drugs for schizophrenia, explains Karuna chief executive Ed Harrigan, is that they only treat some of the symptoms of the disease. "Current drugs do a reasonable job with hallucinations and delusions. But they don't address some of the impairments of attention and memory, or the asociality and anhedonia that can accompany the disease. And those are all symptoms that can really block people from being reintegrated into society."

The family of chemical compounds that Karuna has licensed from Vanderbilt are called glycine transport inhibitors. Harrigan believes that they may be useful in addressing neurological signaling problems in the brains of schizophrenics. The hypothesis, he explains, is that there may be insufficient levels of the neurotransmitter glutamate in the synapses, or spaces between neurons, of people suffering from schizophrenia. Increasing the amount of glycine — another neurotransmitter — may dial up the effectiveness of the glutamate that is present.

Karuna is also developing another treatment for schizophrenia, based on a drug that is no longer patented. (The company has made modifications and applied for a new patent.) Harrigan, formerly a senior licensing executive at Pfizer, says both programs "have the potential to be a breakthrough treatment." The off-patent drug has already been through clinical trials, and Karuna may soon begin trials of its own. The compounds licensed from Vanderbilt have not yet been tested in humans.

Karuna is set up as a "virtual" pharma company, with a handful of employees working with a network of outside service providers to shepherd along promising chemical compounds, perhaps to the point where they'll be acquired or licensed by larger entities. (Virtual pharmas typically don't have their own labs, or plans to set up manufacturing facilities.) Harrigan works in Waterford, Connecticut, and the company has office space that it shares with Puretech Ventures, the Boston venture firm that helped form Karuna. The company, formed in January, has received about $1.5 million in seed funding so far, and Harrigan says it may soon raise more: "We're talking to a number of potential funding sources."