Ultragenyx Pharmaceutical's apazunersen did not meet the goals of its Phase 3 test in Angelman syndrome, a rare genetic neurological disorder with no FDA-approved therapies. The disappointing result has some readthrough to Ionis Pharmaceuticals and Oak Hill Bio, each in clinical development with Angelman drugs designed to work in a similar way as Ultragenyx’s failed molecule.
Wearable technology seems to offer great potential for early detection and management of agitation experienced by people with communication-impaired neurological disorders, allowing caregivers to provide personalized interventions that could significantly improve patients’ lives.
We have the opportunity to design systems that give people greater capacity for the work where human presence matters most: listening, understanding, reassuring, deciding, and caring.
With pimavanserin's Phase 3 failure in schizophrenia, Acadia Pharmaceuticals said it will no longer run clinical trials for the drug, whose lone approval is for treating psychosis from Parkinson's disease. The FDA previously rebuffed regulatory submissions for the drug in dementia and Alzheimer’s disease.
Marinus Pharmaceuticals drug Ztalmy is now FDA approved for treating the seizures in patients who have CDKL5 deficiency, an inherited form of epilepsy. The drug is the first approved treatment for the rare disorder and it’s also Marinus’s first approved product.
Eli Lilly is paying $125 million up front to team up with Rigel Pharmaceuticals on drugs that block a signaling protein involved in inflammation. With the alliance, Lilly joins Sanofi and Denali Therapeutics in the pursuit of drugs that block this protein.
New, wearable technology not only helps monitor symptoms automatically, it may be able to predict seizures and disease progression in patients with neurological disorders.
Healthcare payments are no longer about transactions. It’s about controlling the revenue cycle. And increasingly, that control is not sitting with ISOs.