Therapy Type: Small Molecule (timeline)
Target Type: Other Neurotransmitters (timeline)
Condition(s): Parkinson's Disease
U.S. FDA Status: Parkinson's Disease (Phase 2)
Cerevance performs single-nuclei RNA sequencing of defined cell types isolated from postmortem brain tissue to identify therapeutic targets for neurologic diseases (Xu et al., 2018).
According to information on the company website, CVN424 is an orally available, brain-penetrant small molecule. It modulates an undisclosed, non-dopaminergic target present in striatal neurons that express dopamine receptor D2. The company claims that CVN424 does not affect D1-dependent pathways, and that this selective targeting will help avoid dyskinesia, a side effect of long-term therapy with current Parkinson’s therapies associated with D1 activation. CVN424 is being developed as an add-on to levodopa therapy for Parkinson’s disease.
No preclinical results have been published.
In May 2019, Cerevance completed a Phase 1 single- and multiple-dose safety study in healthy adults. Sixty-six volunteers received single CVN424 doses from 1 to 225 mg, or seven daily doses of 25, 75, and 150 mg or matching placebo. According to an April 2019 company press release, the drug caused no serious adverse events or changes in common safety measures including vital signs, cardiac function, and laboratory analyses.
In December 2019, a Phase 2, randomized, place-controlled study began enrolling 66 people with Parkinson's who experience motor fluctuations on a stable dose of levodopa. To be eligible, patients must average more than 2 hours per day of “off time,” i.e., periods when symptoms reappear between levodopa doses. Participants will be randomized to high or low dose drug or placebo, taken as an oral suspension once daily for one month. The primary outcomes are adverse events and safety measures; secondary outcomes include an unspecified efficacy measure, and pharmacokinetics. This trial takes place at 16 sites across the U.S., and is expected to end in September 2020.
For details on CVN424 trials, see clinicaltrials.gov.
Last Updated: 12 May 2020
- Xu X, Stoyanova EI, Lemiesz AE, Xing J, Mash DC, Heintz N. Species and cell-type properties of classically defined human and rodent neurons and glia. Elife. 2018 Oct 15;7 PubMed.
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