For three years, a group of investors and experienced neuroscience executives have been stealthily advancing one of the more ambitious new startups in neurology, called BlackThorn Therapeutics. Today they’re finally shedding a little light on what they’ve been up to.
As Xconomy reported last year, South San Francisco, CA-based BlackThorn was spun out of Scripps Research Institute, which on its website lists the company as one of its 2013 spinouts. It was seeded by Arch Venture Partners, which has a track record launching ambitious biotech firms with massive rounds of funding, like Juno Therapeutics (NASDAQ: [[ticker:JUNO]]) in 2013 and another neuroscience firm, Denali Therapeutics, last year.
Today, BlackThorn is making its first formal announcement. It’s closed a $40 million Series A round led by Arch, with participation from the venture arm of Johnson & Johnson, Alexandria Real Estate Equities, and several other investors. It’s also disclosed plans to develop drugs for neurobehavioral diseases—such as obsessive compulsive disorder, depression, and schizophrenia—and test them in a way that its executives believe can help overcome some of the field’s notorious roadblocks.
Developing drugs for neurobehavioral diseases is difficult for a number of reasons. Much remains unknown about the underlying biology of diseases like autism, for instance, and tests of drugs for schizophrenia and depression often give different results from trial to trial, often because of a strong placebo effect. This year alone, antipsychotics and depression drugs developed by Alkermes (NASDAQ: [[ticker:ALKS]]), Intra-Cellular Therapies (NASDAQ: [[ticker:ITCI]]), and the now-defunct Forum Pharmaceuticals have failed Phase 3 trials, and all three cited a high level of placebo effect in the control group as a significant problem.
Compounding the difficulty is the lack of reliable indicators, what are known as “biomarkers,” that a patient has say, schizophrenia or depression, or that a drug is actually affecting his or her disease. A drug’s effects in a clinical trial must be measured by observations of mood or behavior, documented by clinicians or via standardized questionnaires—a process that is less precise than running a blood test or a scan, as a company might do to test the effects of a drug for, say, heart disease.
“There is no such toolkit for [neurobehavioral diseases] yet,” says Scott Forrest (pictured), BlackThorn’s VP of corporate development. “It’s emerging from clinical research.”
BlackThorn’s contribution to the toolkit is called Inform, a method of, as it says in a press release, “linking brain physiology to behavior.” Forrest says Inform is a combination of imaging tools and behavioral evaluation techniques—some of which are proprietary, others aren’t—meant to help it both find drug targets important in neurobehavioral diseases, and figure out which subsets of patients might best respond to its drugs.
The hope is that by better identifying and understanding these patient groups, BlackThorn will be able to develop targeted drugs to alter specific behaviors, and then run smaller, more efficient clinical trials with a higher chance of success than is typical in the neurobehavioral field.
“You wouldn’t take all comers who have cancer” in a cancer drug trial, Forrest says. “You would segment [them] by the biology in some way. It’s similar here. Unless you learn about the patients and do so in a way that allows you to target the right treatments to those patients, you’re basically consigning yourself to the track record of the past in this field, which we all know has had significant challenges.”
BlackThorn is on the verge of the first key test of its approach. The company in-licensed a drug that blocks the activity of a protein called nociceptin receptor which is involved in a variety of emotional and behavioral changes. Known as BTRX-246040, the drug has already been tested in early studies and is set to begin a Phase 2 program next year. Forrest says the company has a total of four drug programs currently underway, and the second of them should begin human trials next year as well. The programs aside from BTRX-246040 come from the research of its scientific founders at Scripps, Edward Roberts and Hugh Rosen, who both also co-founded the recently-acquired drugmaker Receptos.
Typically mid-stage trials test a variety of doses of a drug, and measure its safety and effectiveness against a specific disease. In the Phase 2 studies of BTRX-246040, Forrest says
