Work at Praxis Precision Medicines Inc. has led to the identification of new compounds comprising an azaspiroheptane and acting as T-type calcium channel blockers, particularly voltage-dependent T-type calcium channel subunit α-1G (Cav3.1) blockers. As such, they are reported to be useful for the treatment of essential tremor.
Praxis Precision Medicines Inc. CEO Marcio Souza said it would be “disingenuous not to move forward” – U.S. FDA willing – into a phase III effort with an alternate design targeted for the second half of this year, given top-line results from the phase IIb Essential-1 study with ulixacaltamide (PRAX-944) for essential tremor.
Researchers from Praxis Precision Medicines Inc. presented the discovery and preclinical evaluation of a novel voltage-gated sodium channel (Nav) blocker, PRAX-628, being developed as a potential antiepileptic drug candidate.
Following a negative phase II/III study of an investigational treatment for major depressive disorder, Praxis Precision Medicines Inc. is dropping staff from the payroll and refocusing the company’s resources on tremor and epilepsy. The Aria study of PRAX-114, a positive allosteric modulator extrasynaptic GABA receptor, missed its primary endpoint, statistical significance as measured on the Hamilton Depression Rating Scale. It also missed the study’s secondary endpoints.
A phase III study of Sage Therapeutics Inc.’s zuranolone in major depressive disorder hit not only its primary endpoint but also its key secondary endpoint. Despite that, the Cambridge, Mass.-based company’s share value (NASDAQ:SAGE) closed 17.4% lower Feb. 16, at $35.91 each.
Sage Therapeutics Inc. on March 17 disclosed another batch of positive phase III data with zuranolone, its oral, once-daily, two-week therapy for the treatment of major depressive disorder (MDD) and postpartum depression. News regarding the GABAA receptor-positive allosteric modulator came the same day that Praxis Precision Medicines Inc. reported fourth-quarter and full-year financial results, updating investors on PRAX-114, its drug in the same class for MDD.
With the prevalence of central nervous system disorders such as Alzheimer’s disease, multiple sclerosis, epilepsy, Parkinson’s disease and stroke increasing annually, the need for novel therapeutics to treat neurologic and psychiatric disorders has never been greater. Unfortunately, even though there is a significant unmet medical need, because of the high risk and low approval rates of drugs targeting those devastating diseases, in the past decade big pharma companies have been dramatically reducing their R&D spending on CNS disorders, noted Naheed Kurji, president and CEO of Toronto-based Cyclica Inc.
Praxis Precision Medicines Inc. CEO Marcio Souza told BioWorld that the line figuratively walked by PRAX-114, a GABAA positive allosteric modulator in major depressive disorder (MDD), means upside relief without the downside of adverse effects.
