- Cerevel Therapeutics, a young biotechnology company focused on the brain, announced Tuesday positive clinical trial results showing that one of its experimental drugs outperformed placebo at treating adults with schizophrenia.
- The early-stage trial tested two doses of the drug, known as CVL-231, and found both did significantly better than placebo as measured by a scale that evaluates schizophrenia symptoms. Between the trial's start and six weeks in, participants given 30 mg of CVL-231 once a day had an average reduction of 19.5 points on the scale. The group that got 20 mg of Cerevel's drug twice a day had a average reduction of 17.9 points, while the placebo group had an average reduction of 6.8 points.
- Cerevel said its drug was well-tolerated, with the rates of headache, nausea and gastrointestinal side effects similar for patients on placebo and those who got CVL-231. The trial results support a mid-stage schizophrenia program, according to Cerevel. The company also plans to explore the use of its drug in related diseases, including dementia-related psychosis.
Cerevel was created in late 2018 by Pfizer and Bain Capital. Pfizer, which earlier that year decided to back away from neuroscience, provided a handful of drug compounds to the new company, while Bain outfitted it with $350 million in initial funding. In exchange, Bain took a 75% stake, with Pfizer holding onto the remaining quarter.
Cerevel then went public last summer, raising roughly $450 million by merging with a SPAC, or special purpose acquisition company. As of Monday, the company had a market value of $1.6 billion.
Now, Cerevel's first data as a public company appear to be igniting investor interest in the CVL-231 program and in the company more broadly. Cerevel's share value doubled Tuesday morning, ticking just above $25 and pumping its valuation to more than $3 billion.
Though early, the data suggest CVL-231 could be effective and safe. Research indicates that one of the causes of schizophrenia is an abnormal amount of dopamine activity in the brain. Cerevel is trying to fix this problem with CVL-231, as the drug is designed to target the muscarinic M4 receptor, a protein that regulates dopamine.
While there are several other muscarinic receptors that help control dopamine activity, Cerevel sees value in a drug that only works on the M4 type. By being selective, Cerevel hopes its drug can regulate dopamine enough to have an antipsychotic effect, but not so much that it results in the motor and gastrointestinal side effects that have been an issue for other, less-specific compounds.
"Ultimately, the question for CVL-231 is whether this selective approach can thread the needle: achieving comparable efficacy to a more promiscuous drug, but with potentially improved side-effects," wrote Paul Matteis, an analyst at Stifel, in a note to clients ahead of Tuesday's results.
To that end, Cerevel's drug appears to hold up against a rival treatment in development at Karuna Therapeutics, a Boston-based drugmaker led by the renowned researcher Steve Paul.
Karuna's treatment, called KarXT, targets both the M1 and the M4 muscarinic receptors. In late 2019, results from a Phase 2 study that used the same scale as Cerevel's showed schizophrenia patients had, on average, an 11.6-point greater reduction in their scores when given KarXT versus placebo. In Cerevel's trial, the reductions compared to placebo were 12.7 points for the 30 mg group and 11.1 points for the 20 mg group.
Brian Abrahams, an analyst at RBC Capital Markets, wrote in a Tuesday note to clients that while there are limitations when comparing trials, Cerevel's efficacy data does look as strong as Karuna's.
The analyst added that CVL-231 "may also have a few potential advantages over KarXT, though it is still early days to compare." Those advantages include that Cerevel's drug appears to have a clean safety profile, and that it's effective at a once-daily regimen with no need to gradually increase the dose.
Still, Abrahams noted, however, that "even if ultimately successful, we see room for both drugs on the market."
While difficult to pinpoint, estimates hold that schizophrenia and related psychotic disorders effect somewhere in the range of 1 in 150 people to 1 in 400 people in the U.S.