Positive efficacy results led to Praxis Precision Medicines Inc.’s phase II Embold study in developmental and epileptic encephalopathies (DEEs) being halted early, propelling the company’s shares dramatically upward.
Dravet syndrome (DS) is a rare and severe form of epilepsy that causes intellectual disability and motor deficits and can lead to premature death. A loss-of-function mutation in one copy of SCN1A, encoding the voltage-gated sodium channel (NaV)1.1 α subunit, is the most frequent alteration found in DS patients and has been linked with inhibitory neuron dysfunction. Despite the potential of gene therapies, AAV-mediated SCN1A gene replacement for DS has not been possible yet due to AAV genome size constraints.
Selene Therapeutics Ltd. has identified brain permeable dihydroorotate dehydrogenase (DHODH) inhibitors reported to be useful for the treatment of epilepsy, cancer, Alzheimer’s disease, rheumatoid arthritis and multiple sclerosis.
With a move into Lilly Gateway Labs in Boston’s Seaport District, privately held Tevard Biosciences Inc. is ramping up development of its transfer RNA (tRNA)-based therapies to cure everything from Dravet syndrome and other neurological conditions to cardiology indications and muscular dystrophies.
With a move into Lilly Gateway Labs in Boston’s Seaport District, privately held Tevard Biosciences Inc. is ramping up development of its transfer RNA (tRNA)-based therapies to cure everything from Dravet syndrome and other neurological conditions to cardiology indications and muscular dystrophies.
Disappointing top-line data from two failed phase III studies of soticlestat for Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) have crumpled the stock of co-developer Ovid Therapeutics Inc., which lost three-quarters of its value June 17.
Disappointing top-line data from two failed phase III studies of soticlestat for Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) have crumpled the stock of co-developer Ovid Therapeutics Inc., which lost three-quarters of its value June 17.
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.
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.
