Shares of Massachusetts-based biotechnology company Minerva Neurosciences (NASDAQ:NERV) cratered late last week following the company’s announcement of disappointing results from its phase 3 trial of schizophrenia treatment roluperidone (MIN-101). The 32mg and 64mg doses of the company’s key drug were found to be not statistically significant in treating negative schizophrenia symptoms as compared to placebo in the 12-week study.
Both the primary endpoint, the PANSS Marder Negative Symptoms Factor Score, and the secondary endpoint, the Personal and Social Performance Scale Total Score, failed to meet the trial’s objectives. While roluperidone exhibited a positive safety profile this was the perhaps the lone bright spot in an otherwise devastating result for the late stage program.
Momentum from phase 2b trial abruptly halted
A total of 513 patients were included in the phase 3 trial which took place at various locations in the U.S., Europe, and Israel. Roughly one-third of the patient population each received 32mg of roluperidone, 64mg of roluperidone, and placebo, respectively. The statistically insignificant results contrasted with the favorable outcome from the phase 2b trial that showed improvement in both the primary and multiple secondary endpoints. Past data for the experimental drug has demonstrated an ability to block brain receptors that regulate a patient’s cognition, mood, sleep, and anxiety level.
Study representatives asserted that while the phase 3 endpoints were not met, the 64mg dose group showed functional improvement which makes the drug worthy of continued investigation.
Minerva develops therapies to treat central nervous system disorders. Aside from its primary roluperidone compound, it has a treatment called seltorexant (MIN-202) for insomnia and major depressive disorder (MDD) that is in clinical development. Its MIN-301 product candidate is in pre-clinical development as a treatment for Parkinson’s disease.
Executive Chairman and CEO Dr. Remy Luthringer stated, “We are encouraged by the results obtained in this study which expand upon the outcome of the phase 2b study that showed improvements in the primary endpoint and in multiple secondary endpoints. Even though this study didn’t achieve its primary and key secondary endpoints, primarily due to a larger than expected placebo effect at Week 12, results obtained with the 64mg dose including the early onset of effect and functional improvement as measured by PSP suggest roluperidone merits continued investigation for the treatment of primary negative symptoms.”
Despite management’s attempt at putting a positive spin on the phase 3 performance, the market punished the company for the weaker than expected results. Minerva’s stock plummeted 72% on the day of the announcement to its lowest level since 2016 as nearly 81 million shares traded hands.
Minerva originally scheduled a webcast for June 1st to discuss additional data and offer further insight from the roluperidone trial, but later postponed the event until June 5th at 8:30am.
After completing an analysis of the trial’s data, the company plans to consult with the U.S. Food and Drug Administration (FDA) regarding potential next steps for roluperidone’s development. It remains unclear, however just how much additional information can be gleamed from the disappointing study as the company noted “limited inference can be drawn from this data”.