Schizophrenia affects millions of people around the world, which is why any prospective treatment that advances garners close attention. Minerva Neurosciences jumped into the spotlight this morning when an experimental schizophrenia drug it’s developing showed some promise in a mid-stage trial, sending the company’s shares soaring. Now the Waltham, MA-based biotech has to prove that the drug’s benefits hold up when it’s put through larger trials.
Minerva (NASDAQ: NERV) said that a drug known as MIN-101 hit its main goal in a phase 2 trial in 244 patients with “negative symptoms” of schizophrenia—things like a lack of motivation or initiative, which represent a loss of normal function. “Positive” symptoms, by comparison, are the delusions or hallucinations that patients can experience. There are no treatments currently available specifically for negative symptoms.
After 12 weeks of treatment, both low and high doses of MIN-101 led to a statistically significant improvement of negative symptoms on the Positive and Negative Syndrome Scale (PANSS)—a common ratings scale used to judge the effectiveness of treatments for schizophrenia and other mental health disorders—compared to a placebo. The drug also succeeded on various secondary measures, Minerva said.
Minerva said that the incidence and types of side effects didn’t differ much between those on MIN-101 and placebo. Patients on the drug didn’t gain weight either, a common problem associated with other schizophrenia drugs.
Shares of Minerva more than doubled in early trading Thursday, soaring from a $3.54 close on Wednesday to over $9 apiece as of 10:40 a.m. ET.
In the study, 244 patients at sites in Russia and five other European countries were randomized and spilt into three groups, getting either a 32 mg or 64 mg dose of MIN-101 or a placebo, for 12 weeks. Minerva didn’t reveal specific numbers for the change in patients’ PANSS scores from the beginning to end of the study—but said that the “effect size,” a statistical measure of the change in the scores, met the bar of significance.
These results, of course, are just a small step in a long journey. MIN-101 is meant to be a chronic treatment, so its benefits would have to last over time, without complications, to be meaningful. And Minerva has to reproduce these results in larger, longer trials, where a number of drugs for antipsychotic disorders—like depression and schizophrenia—have tripped up. A prospective drug from Alkermes (NASDAQ: ALKS) failed the first two of three late-stage studies for depression in January. And a schizophrenia drug from Forum Pharmaceuticals failed two Phase 3 studies in March. Both Alkermes and Forum cited a high level of placebo effect in the control group as a significant problem in the studies.
Minerva may eventually confront that issue as well. For now, it’s following patients to get a better read on MIN-101’s staying power. All patients in the Phase 2 study were given the chance to advance on to a six-month open-label extension study; those who had been on placebo have been randomized to get one of the two drug doses. Minerva will complete that study in the third quarter.
“We believe the results from this trial constitute a key step forward in the development of a novel treatment for schizophrenia and specifically the negative symptoms of the disease, which represent a significant unmet medical need,” said Minerva CEO Remy Luthringer, in a statement. “Negative symptoms contribute substantially to poor quality of life and functional outcomes of schizophrenic patients.”
Minerva, which went public at $6 per share in June 2014, was formed from the 2013 merger of Cyrenaic Pharmaceuticals and Sonkei Pharmaceuticals. Cyrenaic had licensed what’s now known as MIN-101 from Mitsubishi Tanabe Pharma in 2007. The drug is a small molecule that blocks two types of receptors in the brain—5-HT2A and Sigma 2. Intra-Cellular Therapies (NASDAQ: ITCI), based in New York, is also developing a schizophrenia drug currently in Phase 3 testing, with results expected later this year. Intra-Cellular’s drug also blocks 5-HT2A.