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.
Parkinson’s disease (PD) is a neurodegenerative disorder. But not just. And it may not start that way.
There is increasing evidence that a-synuclein, the protein whose aggregates eventually destroy midbrain dopaminergic neurons in PD (and that are the cause of other diseases collectively known as the synucleinopathies), first aggregates “in enteric neurons, the neurons that control gastrointestinal function,” Collin Challis told BioWorld.
SEATTLE – Applying single cell transcriptomics to brain tissue from living donors is laying bare the vast differences between the human brain and the mouse brain, calling into question the use of animal models in psychiatric drug discovery.
Privately held Zhittya Genesis Medicine Inc. received approval from Mexico’s Comisión Federal para la Protección contra Riesgos Sanitarios (Cofepris) to begin a phase I trial at the Zambrano Hospital in Monterrey for patients with Parkinson’s disease.