BrainCells Inc. has made its name with a novel approach for treating depression, by stimulating the growth of new neurons. Now the San Diego-based company will have some hard choices to make, since a clinical trial showed its drug doesn’t work any better than a placebo for most people with depression. The results also suggest, though, that the drug might help those with a combination of depression and anxiety.
The findings were from a mid-stage study of 90 patients who were randomly assigned to get a once-daily dose of the BrainCells drug, a three-times daily dose, or a placebo. The patients were severely depressed, and weren’t helped by anti-depressant drugs. The study found after six weeks that the BrainCells drug was no different than a placebo in the overall treatment group, although it did appear to offer a benefit when given three times a day to a subpopulation of patients who had a combination of depression and anxiety. The drug was well-tolerated, BrainCells said. Results are being submitted for presentation at an upcoming medical meeting.
Any savvy biotech watcher knows that the word “subpopulation” sounds like a company digging around for something positive after the fact, which the study wasn’t designed or statistically powered to prove. BrainCells CEO Jim Schoeneck and chief scientist Carrolee Barlow acknowledge that the result isn’t statistically significant, and really is more of a hypothesis-generator for future trials than anything else. The company hasn’t made any firm decision yet on what its next move will be, although it will consider pursuing post-traumatic stress disorder and the combination of major depressive disorder and generalized anxiety.
An estimated 40 percent of depressed patients have these sorts of overlapping diagnoses, Schoeneck says. Only about 30 to 40 percent of depressed patients today are thought to benefit from existing anti-depressants. That’s still a very big number, and big potential market, when an estimated 20 million people in the U.S. are thought to suffer from depression.
“What we are excited about is that we enrolled patients with increased anxiety, and were able to treat a more difficult population,” Barlow says.
This experimental drug, BCI-540, is one of the two primary assets at BrainCells Inc. The drug was previously tested by Japan-based Mitsubishi Pharma as an Alzheimer’s treatment. BrainCells Inc. negotiated for a license to the compound after its in-house platform, which uses neural stem cells to create neurons in a lab dish, showed the drug could spark the kind of effect it likes to see, with an impact on the differentiation and survival of new neurons.
The other potentially valuable treatment at BrainCells is called BCI-952, a combination of a generic drug and a dietary supplement. Back in July, that drug showed “comparable” effectiveness to existing anti-depressants in a mid-stage study of 142 patients, according to that study’s lead investigator, Massachusetts General Hospital’s vice chair of psychiatry, Mauricio Fava. That unorthodox drug candidate is currently being formulated into a single pill that would be more convenient and commercially viable, Schoeneck says. The combination intrigued researchers because it offers fewer side effects than the existing meds.
It will be interesting to see if BrainCells presents the full results from the most recent BCI-540 study at a medical meeting, and what might be learned from it. Some of the investigators on the trial are excited about the result, Barlow says, because they saw a clinically meaningful benefit for some very hard to treat patients. But the company emphasized it is still combing through the data itself, and can’t really say which patients responded better than others or what it is about their genetics or environment that makes them respond better than the rest.
I asked Schoeneck if BrainCells has the capital it needs to take the next step in development on its own, and he says the company does. BrainCells Inc. raised $50 million back in April 2008, right before the worst of the recession became apparent. But back when I met with Schoeneck and Barlow at the JP Morgan Healthcare Conference in January, Schoeneck said the company will either need to raise more capital, or secure a Big Pharma partnership at some point. If and when that point comes, the company will have to have some pretty compelling data that BrainCells has discovered a truly different and powerful way for treating depression.
