For a company that was running out of money, a missed phase III endpoint for its only development product knelled a death blow for Marinus Pharmaceuticals Inc., tanking its stock by 82%. The Radnor, Pa.-based company will no longer develop oral ganaxolone for seizures associated with tuberous sclerosis complex, or for any other indication, as it reduces its workforce and explores strategic alternatives.
Privately held Dyno Therapeutics Inc. has added another notch to its adeno-associated virus (AAV) vectors development portfolio in a deal with the Roche Group that includes $50 million up front and ultimately could top $1 billion. Dyno will help in developing next-generation AAV vectors, optimized by artificial intelligence, to target neurological diseases.
Scientists from different laboratories around the world have presented the latest advances in research into malignant brain tumors at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), which is being held Oct. 22 to 25 in Rome.
Despite positive findings from an earlier trial, Alto Neuroscience Inc.’s BDNF-targeting candidate, ALTO-100, failed to best placebo in a phase IIb study in major depressive disorder, sending shares of the company to their lowest price since going public in a February 2024 IPO, as investors worried about readthrough to Alto’s biomarker-based approach for treating psychiatric disorders.
The U.K. Medicines and Healthcare products Agency has become the third to approve Eli Lilly and Co.’s Kisunla (donanemab), but the drug’s spending watchdog has simultaneously ruled the Alzheimer’s disease treatment is not cost effective.
Targeting NMDA in mental health has chalked wins but not universally, as shown by Sage Therapeutics Inc.’s failure of the placebo-controlled phase II Lightwave study testing dalzanemdor in Alzheimer's disease, which missed the primary outcome measure, another bit of bad luck from the company that was disclosed Oct. 8.
Cureverse Inc. and Angelini Pharma SpA signed a potential $360 million deal for CV-01, an oral small-molecule candidate for Alzheimer’s disease and neurological disorders like epilepsy. As a novel candidate, CV-01 suppresses neuroinflammatory reactions through the Kelch-like ECH-associated protein 1 and nuclear factor erythroid 2-related factor signaling pathway.
Seaport Therapeutics Inc. has followed up its recent fundraiser with an oversubscribed $225 million series B financing that will help set it on the path to a phase IIb study in major depressive disorder. The company’s lead candidate is allopregnanolone, an endogenous neurosteroid that is taken orally and bypasses the liver. Once it is absorbed through the lymphatic system, allopregnanolone enters through a pathway that avoids the liver and the possibility of hepatoxicity and elevated liver enzyme counts, Michael Chen, Seaport’s chief scientific officer, told BioWorld.
With two complete response letters in the rearview mirror, Abbvie Inc.’s Vyalev (foscarbidopa/foslevodopa) has been approved by the U.S. FDA for treating Parkinson’s disease. The drug is the first subcutaneous 24-hour infusion of levodopa-based therapy for treating motor fluctuations in adults with advanced disease.
Gene therapy specialist Meiragtx Holdings plc got a market bump courtesy of newly released top-line data from its phase II bridging study in Parkinson’s disease. The six-month, three-arm randomized, double-blind, sham controlled trial of AAV-GAD, a one-time infusion, demonstrated significant and clinically meaningful improvements in key efficacy endpoints. The primary objective was evaluating the therapy’s safety and tolerability. The study of participants with idiopathic disease showed the therapy was safe and well-tolerated with no serious adverse events. Meiragtx is pursuing approvals in the U.S., Europe and Japan.
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