Sangamo Therapeutics Inc. continued its collaboration spree, signing up its sixth big pharma/biotech partner: Novartis AG. The three-target deal will use Sangamo's zinc finger protein transcription factors to up-regulate undisclosed genes to treat autism spectrum disorder and other neurodevelopmental disorders.
In a move that Lexicon Pharmaceuticals Inc. chief Lonnel Coats said will focus the company on its phase II neuropathic pain program, The Woodlands, Texas-based venture has agreed to sell one of its two approved products, the carcinoid syndrome diarrhea therapy Xermelo (telotristat ethyl), to Tersera Therapeutics LLC for $159 million in cash.
UCB SA, a Belgian company developing an antibody targeting a toxic protein tied to Alzheimer’s disease (AD), said Roche Holding AG has negotiated an exclusive global license to the potential therapy for $120 million up front, plus almost $2 billion in milestone payments following positive proof of concept for the anti-tau candidate, UCB-0107, in AD.
Nura Bio Inc., a company working to discover and develop new neuroprotective medicines, has closed a $73 million series A financing that President and CEO Alpna Seth said would help her team advance a multitarget pipeline, initially led by an inhibitor of the sterile alpha TIR motif protein 1 inhibitor (SARM1).
CYBERSPACE – Data presented at the virtual 2020 Alzheimer's Association International Conference (AAIC) and reported in the July 28, 2020, online issue of the Journal of the American Medical Association (JAMA) demonstrated that blood levels of phosphorylated tau-217 (Ptau-217) did as well as cerebrospinal (CSF)- and PET-based biomarkers, and significantly better than other blood-based biomarkers, at discriminating individuals with Alzheimer’s disease (AD) from those with other neurodegenerative disorders.
HONG KONG – Daejeon, South Korea-based biotechnology company G2GBIO Inc. has raised ₩11.4 billion (US$9.53 million) from a series B financing round, with the funds to be used on clinical trials for a sustained-release Alzheimer’s treatment as well as nonclinical trials for diabetes and sustained-release postoperative pain treatments.
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.
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