Alkermes is chasing Takeda Pharmaceutical, whose orexin receptor-targeting pill is on track to become first in a new class of narcolepsy medicines. Blair Jackson, chief operating officer of the neuroscience biotech, says Alkermes’s drug offers advantages and also paves the way to bring orexin agonism to other indications.
Avidity Biosciences brings to Novartis three RNA therapies in pivotal testing for rare neuromuscular diseases along with the technology that produced them. This platform develops drugs in a new class of targeted medicines offering potential to expand the delivery of RNA treatments beyond the liver.
Across the nation, state-based health insurance exchanges are searching for ways to better serve their communities while making enrollment easier, more personal, and more accessible for everyone.
Praxis Precision Medicines’ essential tremor drug ulixacaltamide met the goals of two Phase 3 clinical trials, paving the way for discussions with the FDA about a new drug application. The once-daily pill could become the first approved treatment for this movement disorder affecting an estimated 7 million Americans.
With the new capital infusion, Sanofi Ventures’ total assets under management now tops $1.4 billion. Our recap of other financings includes startups raising money for novel therapies in development for autoimmune disease, neurological disorders, cancer, and more.
UniQure plans a first quarter 2026 FDA submission for AMT-130 as a treatment for Huntington’s disease. This gene therapy delivers microRNA designed to silence the mutated gene that produces a protein driving this disorder, which so far has no FDA-approved therapies.
Ionis Pharmaceuticals plans a 2026 FDA submission for zilganersen as a treatment for Alexander disease, an ultra-rare inherited neurological disorder. The antisense oligonucleotide developer has been moving toward commercializing drugs on its own rather than seeking partners.
In a recent interview, Esperion president and CEO, Sheldon Koenig talks about the reality of statin intolerance and his company’s expansion into new diseases
Takeda Pharmaceutical’s oveporexton is designed to treat the root cause of narcolepsy. Statistically significant Phase 3 results for the twice daily pill were presented Monday during the World Sleep 2025 Congress in Singapore.
Saniona’s drug candidate could expand Jazz Pharmaceuticals portfolio in epilepsy. But this preclinical small molecule will need to show advantages in safety and efficacy to compete with Biohaven Pharmaceuticals and Xenon Pharmaceuticals, each with clinical-stage drugs that hit the same epilepsy target.
Draig Therapeutics’ lead drug is designed to safely target the AMPA receptor as a way of mediating the interplay between two neurotransmitters whose imbalance is associated with mood disorders. The startup is one of several that have recently raised mega rounds of financing to support programs in the clinic or entering clinical testing.
The Alzheimer’s disease drugs that have reached the market in the past two years are antibodies designed to break up amyloid plaques. Vigil Neuroscience brings to Sanofi a different approach: an oral small molecule designed to activate microglia, immune cells that play a key role in brain health.
In a landscape where complexity has long been the norm, the power of one lies not just in unification, but in intelligence and automation.
A planned interim clinical trial analysis found Praxis Precision Medicines drug ulixacaltamide was unlikely to succeed as a treatment for essential tremor. But Praxis is continuing two late-stage tests of the drug; data are expected in the third quarter of 2025.
Johnson & Johnson’s agreement to acquire Intra-Cellular Therapies brings Caplyta, which is projected to become a blockbuster seller across several neurological indications. In other M&A news, GSK and Eli Lilly each struck deals to fill their cancer drug pipelines.
Lundbeck says Longboard Pharmaceuticals’ drug, bexicaserin, has potential applications in a range of rare epilepsies. Last month, the biotech began a Phase 3 clinical trial testing the drug in Dravet syndrome.
Bristol Myers Squibb’s Cobenfy treats schizophrenia by going after a different target than currently available antipsychotic drugs, which is intended to offer better efficacy and safety. The drug came from the pharma giant’s $14 billion acquisition of Karuna Therapeutics.
By prioritizing user-centric design, responsible data practices, and evidence-based solutions neurotech companies can not only drive user adoption but also foster long-term brand loyalty.