Lowering levels of tau protein improved multiple symptoms of autism spectrum disorders (ASD) in two different mouse models of the disease, both of which are driven by hyperactivity of the mTOR PI3 kinase pathway.
Launching a broad new front in its long-running battle against dementia and other neurological diseases, Biogen Inc. has moved to license multiple Sangamo Therapeutics Inc. programs for $350 million up front plus up to $2.37 billion in development, regulatory and commercial milestone payments.
With the FDA’s blessing, Biohaven Pharmaceutical Holding Co. Ltd.’s Nurtec ODT (rimegepant) enters a massive market for treating migraine in adults. In the U.S. alone, more than 25 million people have been diagnosed and seek treatment.
Everything’s good for something. Including, it turns out, 5’ untranslated trinucleotide repeats. In the Feb. 17, 2020, issue of Nature Neuroscience, researchers have demonstrated a role for such repeats in controlling protein levels of fragile X mental retardation protein (FMRP).
H. Lundbeck A/S’ antibody-based migraine therapy, Vyepti (eptinezumab-jjmr), which just received FDA approval as the first and only intravenous preventive treatment for adults, enters a market forecast to grow to $7 billion by 2027. While some analysts placed Vyepti’s earnings potential at about $800 million annually, the drug itself is positioned to become a possible blockbuster, earning $1 billion annually.
Investors continue to keep a close eye on the progress of companies involved in developing medicines targeting the central nervous system and, in the main, their reaction has been generally positive. The BioWorld Neurological Diseases index, a price-weighted index of public biopharmaceutical companies that are focused on developing therapies to treat neurological diseases, closed the year up over 16% and after, a dip in January, is now tracking up more than 12% by market close on Friday Feb. 21, well ahead of the general markets for the same period.
During a conference call with investors, Baudax Bio Inc. CEO Gerri Henwood let out an exuberant “woohoo!” to celebrate the FDA’s approval – after two turndowns and much haggling over data – of Anjeso (meloxicam) for moderate to severe pain. Echoing her sentiment was Piper Sandler analyst David Amsellem. “It’s nice to see the pain division finally get this one right,” he said.
Teva Pharmaceutical Industries Ltd.’s Austedo (deutetrabenazine) lived up to the lack of expectations in pediatric Tourette syndrome (TS), missing identical primary endpoints in a pair of late-stage trials.
PERTH, Australia – Medicinal cannabis company Zelira Therapeutics Ltd. reported that its ZLT-101 therapy met the primary endpoint of a statistically significant improvement in insomnia in a phase Ib/IIa study in patients diagnosed with chronic insomnia.
What with the lawsuits, declining prescriptions and approval hurdles that seem to be the U.S. answer to a nationwide opioid epidemic, drug companies have to think long and hard before spending the resources to develop yet another opioid drug. And patients are paying the price.
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