T-type calcium channel blocker(Nippon Chemiphar)

Praxis Precision Medicines has stopped a trial of its sodium channel modulator in a rare brain disease early for efficacy. An interim analysis of the Phase 2/3 EMBOLD study found the once-daily pill, called relutrigine, to be effective in the second cohort of patients, the Boston biotech said Thursday. This cohort is registrational, and Praxis will meet with the FDA “in the coming weeks” to discuss the next steps. After that, it will decide when to file for approval. The company’s shares $PRAX were up 25% before trading opened Friday. The Thursday press release did not reveal data, but Cowen analysts wrote in a Dec. 4 note that EMBOLD was designed to detect a reduction in monthly seizure frequency of about 30% compared with placebo. The interim analysis was based on roughly 70% of the patients — around 60 individuals — enrolled in the four-month study, they wrote. The topline data from EMBOLD will be presented on Saturday at the American Epilepsy Society’s annual confab. The trial tested relutrigine in a disorder known as SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). DEEs are a group of severe forms of epilepsy, in which patients have seizures, which are often drug-resistant, as well as significant developmental delay or loss of developmental skills. The SCN2A and SCN8A genes encode sodium channels in the brain, which are vital for brain cell communication. Mutations in these genes lead to disrupted sodium ion flow, impairing brain function. Relutrigine inhibits persistent sodium current, which Praxis said is a key driver of seizure symptoms in severe DEEs. There are no approved treatments for SCN2A and SCN8A DEEs. Praxis reported last year that in the first cohort of EMBOLD, patients taking relutrigine had a 46% reduction in countable motor seizures versus placebo. The biotech said Thursday that the drug was well-tolerated in cohort 1 of the trial, but did not share safety findings from the second cohort. Jefferies analysts project peak sales of $250 million or more in SCN2A and SCN8A DEEs, based on a US patient population of around 5,000, according to a Thursday note. They forecast another $250 million-plus in other DEEs such as Dravet syndrome. They add that the findings in EMBOLD could increase the chances of success in the ongoing Phase 3 EMERALD study of the pill in other forms of DEE. EMERALD is set to conclude in the second half of 2026, potentially allowing an additional approval filing the following year. Separately, Praxis confirmed Thursday that it plans to seek approval of ulixacaltamide in essential tremor early next year, following “collaborative discussions” with the FDA. The submission will be the biotech’s first NDA. Phase 3 trials of the T-type calcium channel blocker were unexpectedly disclosed as successes in October, tripling Praxis’ market cap. Jefferies analysts forecast peak sales in excess of $2.5 billion for ulixacaltamide.

Plus, news about Astellas, Clover, ElevateBio, Monopar, Idorsia and Neurocrine Biosciences: Vera Therapeutics plans $300M offering: The California biotech is selling shares after presenting Phase 2b data for an experimental drug called atacicept over the weekend. The company shared 96-week data for patients with immunoglobulin A nephropathy. — Kyle LaHucik Abeona Therapeutics resubmits rare skin disease therapy: Back in April, the FDA rejected Abeona’s cell therapy, called pz-cel, requesting more information about manufacturing. Abeona is developing pz-cel for recessive dystrophic epidermolysis bullosa, a rare genetic skin disease. — Lei Lei Wu Astellas yanks Izervay’s submission in Europe: The company said it withdrew its marketing authorization application for Izervay, a C5 protein inhibitor for geographic atrophy secondary to age-related macular degeneration. Astellas did not detail feedback from the Committee for Medicinal Products for Human Use, but said it is still “confident” in Izervay’s risk-benefit profile and will continue to engage with regulators in Europe and elsewhere. There are currently no treatments approved for GA secondary to AMD outside of the US, where Izervay was greenlit in August of last year. — Ayisha Sharma Clover releases early RSV vaccine data: The Shanghai-based biotech said its bivalent vaccine candidate, SCB-1019, produced “comparable” levels of RSV neutralizing antibodies to GSK’s Arexvy at 28 days in a Phase 1 test. Based on these data, Clover plans to advance the asset into studies focused on re-vaccination next year. — Ayisha Sharma ElevateBio lays off 4%: The genetic medicines company, which employs about 500 people, is ending its preclinical cellular engineering work, a spokesperson told Endpoints News on Monday. The Waltham, MA-based biotech laid off about 13% of its staff in October 2023. It’s one of the most well-funded private biopharma startups after having raised about $1.25 billion, including its $401 million Series D last year. Partnerships with companies like Novo Nordisk, Moderna, Kyverna and Affini-T are unaffected, the spokesperson confirmed. — Kyle LaHucik Monopar Therapeutics prices $19.2M offering: The Chicago-area biotech sold shares to Janus Henderson, RA Capital and others. A few days ago, Monopar licensed a Wilson disease drug candidate from AstraZeneca’s Alexion. — Kyle LaHucik Idorsia ends work with Neurocrine Biosciences: The companies were developing Idorsia’s T-type calcium channel blocker for patients with a rare form of pediatric epilepsy. The treatment failed a Phase 2 study in 2022, and further analysis led to the decision to stop work. — Jaimy Lee