China’s National Medical Products Administration (NMPA) granted new approvals to several Chinese biopharmaceuticals this week, including expanding indications of four different cancer drugs and clearing one sublingual tablet for stroke.
Shares of Relmada Therapeutics Inc. (NASDAQ:RLMD) plummeted 77% to close at 63 cents Dec. 4 after a pre-planned interim analysis prompted the independent data monitoring committee to conclude the phase III Reliance II study testing REL-1017 in major depressive disorder is likely to fail, leaving the future of the NMDA receptor channel blocker in doubt while the company looks ahead to an earlier-stage psilocybin-based program targeting metabolic disease.
Researchers at the University of Rochester have described a neuroimaging-based biomarker that could identify individuals with early psychosis, and improved their identification when it was added to a standard neurocognitive diagnostic test. In a group of roughly 160 participants in the Human Connectome Early Psychosis Project, individuals who were in the early stages of psychosis had stronger connections from the thalamus (a midbrain sensory processing area) to the cortex, but weaker connections between different cortical areas, than controls.
PTC Therapeutics Inc. said a “highly competitive process with several parties involved” led to the deal centered on the firm’s Huntington’s disease program with Novartis AG, an arrangement that brings $1 billion up front along with as much as $1.9 billion in development, regulatory, and sales milestone rewards.
Our immune cells are not just “defenders” against deadly viruses and pathogens but also a great balancer for tissue homeostasis. For neurological disorders, understanding the neuro-immune axis could be key to treating previously untreatable conditions such as autism spectrum disorder, according to Jun R. Huh, professor of immunology at Harvard Medical School.
At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA level were among the approaches that were presented to fight muscular dystrophies.
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.
The Alzheimer’s disease (AD) space took another blow along with the shares of Cassava Sciences Inc. (NASDAQ:SAVA), which plummeted by 83.8%, or $22.19, to end Nov. 25 at $4.30 after the firm unveiled top-line data from the phase III Rethink-Alz study with simufilam. The study tested simufilam in mild to moderate AD, where results fell short of each of the prespecified co-primary, secondary and exploratory biomarker endpoints.
China’s drug regulator approved Alpha Biopharma Ltd.’s EGFR tyrosine kinase inhibitor (TKI), Zorifer (zorifertinib hydrochloride tablets; AZD-3759), as a first-line treatment for EGFR-mutated non-small-cell lung cancer (NSCLC) with brain metastases.
Rising from a $51 million series A round a year ago to a $1.1 billion acquisition, Kate Therapeutics Inc. has stepped under the umbrella of Novartis AG, which gains preclinical adeno-associated virus-based gene therapies for neuromuscular diseases.
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