Investors anxious to see data from Marinus Pharmaceuticals Inc.’s phase III Raise study testing intravenous ganaxolone in refractory status epilepticus (RSE) will have to wait a little longer, as the trial failed to meet the predefined criteria for stopping at the interim analysis, sending shares (NASDAQ:MRNS) down nearly 83%.
Updated results from Stoke Therapeutics Inc.’s studies of antisense oligonucleotide STK-001 showed a reduced convulsion seizure frequency in those ages 2 to 18 with Dravet syndrome. The new data, which also showed improved cognition and behavior in participants, substantially boosted the company’s stock value and caused analysts to applaud.
Despite numerous marketed anti-seizure medications in the U.S., patients with epilepsy continue to experience breakthrough seizures and tolerability issues compounded with drug interactions and side effects, leaving room for companies like Praxis Precision Medicines Inc. to fill treatment gaps. The Boston-based company presented phase IIa proof-of-concept data March 26 showing that in an evaluation of photo paroxysmal response, its once-daily oral small-molecule treatment, PRAX-628, resulted in a complete response in three epilepsy patients receiving 45 mg of the drug and four patients receiving 15 mg for a combined complete response rate of 88%.
Longboard Pharmaceuticals Inc.’s positive – and then some – phase Ib/IIa top-line data with 5-HT2C receptor superagonist bexicaserin (LP-352) in developmental and epileptic encephalopathies (DEEs) sparked Wall Street speculation about competitive odds as well as the shape of the firm’s upcoming phase III effort.
Serotonin 5-HT2 receptor agonists have shown efficacy in treating seizure disorders, such as epilepsy. At the American Epilepsy Society meeting this week, Longboard Pharmaceuticals Inc. presented data on their 5-HT2C receptor superagonist LP-352 for the treatment of epilepsy.
PGI Drug Discovery LLC, the drug discovery arm of Psychogenics Inc., has entered into an exclusive license agreement with F. Hoffmann-La Roche Ltd. to obtain global development, manufacturing and commercialization rights to RO-7117997. Psychogenics intends to evaluate RO-7117997 as a potential treatment for several psychiatric and neurological conditions.
The U.S. FDA put out a safety warning Nov. 28 that antiseizure drugs levetiracetam and clobazam can cause a rare but serious hypersensitivity drug reaction that may start as a rash but can progress to injure internal organs. In addition, U.K. health care providers are being told to get a plan in place now to implement the first phase of the Medicines and Healthcare products Regulatory Agency’s new regulatory measures to reduce the risks of valproate, a treatment for epilepsy and bipolar disorder.
Stoke Therapeutics Inc.’s stock tumbled 32% after the company reported phase I/IIa data of its antisense oligonucleotide, STK-001, in Dravet syndrome, showing it reduced convulsive seizures in children most consistently at the highest dose, with benefits increasing as time progressed.
Researchers from Kyungpook National University presented data from a study that aimed to investigate the endogenous mechanisms involved in granule cell dispersion (GCD) and its role in epileptic seizures in temporal lobe epilepsy (TLE).