Cancer treatment has been transformed, at its root, by a transformational change in how it is classified. These days, which organ a tumor arises in is often less important than its molecular drivers, which can be sensitive either to specific targeted treatments, or increase the chance that a tumor will respond to immunotherapy. Those successes have not escaped the notice of researchers in other areas of biomedicine, and diseases including heart failure, asthma and polycystic ovarian syndrome are being looked at with an eye to subdividing them in ways that brings diagnostics into the molecular era. Nowhere do those changes have greater potential than in disorders of the brain – in part because there is nowhere much to go but up as far as classifying neurological diseases goes.
Testing Nuplazid (pimavanserin) against major depressive disorder (MDD), Acadia Pharmaceuticals Inc. became the latest to fail in the indication as the company unveiled top-line results from the 298-patient phase III effort called Clarity.
Chief Business Officer David McNinch told BioWorld that South San Francisco-based Encoded Therapeutics Inc.’s $135 million from an oversubscribed series D financing follows by about a year the hefty series C round that “helped us get the [lead gene therapy] program poised to its current stage, which is IND-enabling studies.” The asset, ETX-101 for SCN1A-positive Dravet syndrome (DS), is expected to enter human trials next year.
The FDA has approved Xywav (calcium, magnesium, potassium and sodium oxybates; JZP-258), an oxybate product for treating both cataplexy and excessive daytime sleepiness in narcolepsy patients ages 7 and older, from Jazz Pharmaceuticals plc.
With an increasing number of neurological manifestations being reported in patients suffering from COVID-19 infection, the need for therapies to treat those conditions as well as other major CNS disorders has, once again, been highlighted. That may be one of the reasons why investors have turned their attention to the space once again, with several members of the BioWorld Neurological Diseases index enjoying an uptick in their share valuations.
DUBLIN – Roche Holding AG has joined Jnana Therapeutics Inc. in its quest to drug the human repertoire of solute carrier (SLC) metabolite transporters as a means of tackling a swath of immune-mediated and neurological diseases. The Basel, Switzerland-based pharma is paying Boston-based Jnana $40 million up front and could pay over $1 billion more in research funding, preclinical, development and commercial milestones, as well as sales royalties, in a multitarget deal.
LONDON – New human brain organoids that precisely model the three hallmarks of Alzheimer’s disease – amyloid plaque-like lesions, progressive neuronal death and abnormal accumulations of tau – are now ready to be developed for use in high-throughput drug screening.
Inmune Bio Inc. CEO Raymond Tesi told BioWorld his firm is taking an “oncology-style” approach to Alzheimer’s disease (AD) as the firm tests next-generation tumor necrosis factor (TNF) inhibitor XPro-1595 against neuroinflammation.
HONG KONG – Tokyo-headquartered Eisai Co. Ltd. is targeting new markets for its orexin receptor antagonist, Dayvigo, to treat insomnia. The company launched the drug in Japan on July 6, alongside a new fine granule formulation for its antiepileptic drug, Fycompa.
Armed with intellectual property generated in the lab of Edgar Engleman at Stanford University, Tranquis Therapeutics Inc. has emerged from stealth mode through a $30 million series A round.
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