After 25 years of research. Allschwil, Switzerland’s Idorsia Ltd. has had its first drug approved by the FDA – Quviviq (daridorexant) for adults with insomnia. The okay for Quviviq is the result of painstaking research led by the firm’s chief scientific officer, Martine Clozel, whose husband Jean-Paul Clozel is CEO.
Harmony Biosciences Holdings Inc.’s plan disclosed at the start of last December to launch a phase III study with Wakix (pitolisant) for idiopathic hypersomnia (IH) during the first half of this year brought renewed attention to the sleep space, where a handful of players are jostling for position.
Astrazeneca plc’s recently acquired Alexion Pharmaceuticals Inc. has signed a deal worth up to $760 million with Neurimmune AG, the Swiss biotech that discovered Alzheimer’s drug Aduhelm (aducanumab), buying rights to amyloidosis drug NI-006. While Biogen Inc.’s Aduhelm targets amyloid plaques thought to cause Alzheimer’s in the brain, the phase Ib drug in Alexion’s deal is intended to tackle the build-up of the rogue protein that causes heart disease caused by transthyretin amyloid cardiomyopathy (ATTR-CM).
The hinge of the new development deal between Entos Pharmaceuticals Inc. and Eli Lilly and Co. is also the core of Entos’ business: delivering a drug without significant toxicity. Development of the cargo is only part of the story in creating safe and effective medicine, Entos CEO John Lewis told BioWorld. “You have to have a safe and effective delivery system.”
Amneal Pharmaceuticals Inc. has bought a small portfolio of drugs from fellow U.S. biotech Saol Therapeutics Inc., adding the skeletal muscle relaxant baclofen to its portfolio and boosting its pipeline with a potential therapy to treat spasticity in a deal worth $83.5 million plus royalties.
Annexon Inc. deemed promising – as did analysts – the interim phase II data with C1q protein complex inhibitor ANX-005 in Huntington’s disease (HD), but the safety profile took Wall Street aback, and shares (NASDAQ:ANNX) sank $3.75, or 34%, to close at $7.26.
Schizophrenia remains one of the toughest mental health issues to treat, as well as an indication that has created substantial challenges for drug makers. The pipeline of potential new schizophrenia medicines is busy, with Cortellis showing that 10 new phase III schizophrenia trials that began recruiting patients in 2021 and 59 phase III trials ongoing at the end of the year.
Researchers at PTC Biotherapeutics Inc. have identified orally available small-molecule compounds that broadly lowered the levels of mutant huntingtin protein in both the brain and the periphery by affecting its splicing. One of those compounds, PTC-518, is currently in phase I trials as a therapy for Huntington's disease.
Attempts to modernize the conceptual framework of brain function and dysfunction are one prerequisite for brain disorders to benefit from precision medicine. For the circuit-based insights that are slowly emerging to benefit patients, though, better targeting methods are needed.
Brain disorders have not yet profited from advances in precision medicine to the same extent that other disorders have. With the advent of magnetic resonance imaging and other technologies, watching the brain at work has made great strides in recent decades. But those data have often been shoehorned into the categories of the Diagnostic and Statistical Manual of Mental Disorders. Researchers are working to bring diagnostic categories in line with a modern understanding the brain.
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