Karuna has two novel programs in development, Harrigan said, and has been incubating at the Boston firm PureTech Ventures, whose partner, Eric Elenko, was in charge of day-to-day operations before Harrigan took over. Karuna’s founding team also includes PureTech associate Andrew Miller.
Elenko and Harrigan declined to reveal specifics but said one of the firm’s molecules with a novel mechanism for treating schizophrenia is being tested in humans.
Earlier in his career at Pfizer, Harrigan played a key role in the development of the antipsychotic pill ziprasidone (Geodon).
Karuna’s lead drug is making progress, Harrigan said: “We have clinical evidence in the target population that, using the standard measurement for symptoms of psychosis in patients with schizophrenia, there is significant improvement.’’
In true PureTech fashion, Karuna is building a team with top names in pharma and academia. Steven Paul, former president of research at Lilly, is the start-up’s chairman and a scientific adviser. The scientific advisory board includes Joseph Coyle, a professor of psychiatry at Harvard Medical School; Jeffrey Conn, a professor of pharmacology and director of the drug discovery program at Vanderbilt; Anthony Grace, a professor of neuroscience, psychiatry, and psychology at the University of Pittsburgh; and Carol Tamminga, a psychiatric researcher at the University of Texas Southwestern.
Karuna has not raised money from outside investors, Elenko said. It is largely a virtual operation. Harrigan still lives in the new London, Conn., area, and the company is using contract research facilities and talent.
Sometimes it takes a parent’s perspective to understand what is really at stake for patients.
And two parents of children with cystic fibrosis, a genetic disease, say the regulators got this one wrong.
“I appreciate that they are trying very hard not to do anything bad, but the measures that they were asking us to use for this particular product were meaningless,’’ said Francine Healey, a Needham mother of two children with cystic fibrosis.
Cystic fibrosis, which causes a buildup of mucus in the lungs, also affects digestion and the ability to absorb nutrients. about 30,000 Americans have the disease, and they have a median life expectancy of 37 years.
Liprotamase — the Lilly drug that it picked up through its buyout of Alnara Pharmaceuticals of Cambridge — is intended to provide certain pancreatic enzymes that patients with the disease and other conditions need to get nutrition from food.
Yet the FDA panel voted 9 to 3 that there was not enough evidence the drug is effective.
The FDA is expected to say by mid-April whether it will take the panel’s advice.
Healey’s 19-year-old son and 16-year-old daughter take digestion supplements made with enzymes from pigs. Liprotamase is made in a microbial process and would be the first approved treatment of its kind not to use the pig enzymes.
FDA staff noted that liprotamase scored lower in a measure of fat absorption than the existing supplements made with pig enzymes.
Patrick Marshall, whose daughter has cystic fibrosis, said the fat absorption measure has never been an important one for his daughter.
He would leave it to her doctor to decide whether she should take liprotamase instead of the pig-derived supplement.
The nonprofit Cystic Fibrosis Foundation has funded development of liprotamase, in part because of concern that sick pigs could contaminate or limit the supply of supplements.
Both Healey and Marshall have been fund-raisers for the foundation.
Lilly might have to make a tough decision about whether to invest in another clinical trial of liprotamase, depending on the FDA’s ruling. The company said it is confident in the clinical trial data that has been submitted.
Cambridge-based Ironwood Pharmaceuticals inc. is in a new drug-discovery collaboration with Redwood City, Calif.-based Protagonist Therapeutics inc., which will use its proprietary technology for developing peptide drugs against biological targets picked by Ironwood.
© Copyright 2011 Globe Newspaper Company.