Chiesi Farmaceutici SpA scored U.S. FDA clearance of the enzyme replacement therapy (ERT) Lamzede (velmanase alfa-tycv) for non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. An ultra-rare, progressive lysosomal storage disorder, AM is caused by deficiency in the enzyme alpha-mannosidase. Lamzede is the first ERT to win approval in the indication, characterized by an inability to properly break down certain groups of complex sugars in the body’s cells.
Metabolic health is at an odd juncture. With the advent of glucagon-like peptide (GLP-1) agonists, pharmacologically induced weight loss has matured into a viable therapeutic option at long last. GLP-1R agonists, which are also called incretin mimetics and GLP-1 analogs, are likely to continue their success across multiple areas of medical care. Already, the class has transformed diabetes care, making a splash in weight management, and it may yet do the same for other indications.
Researchers at Stanford University School of Medicine have developed a method to measure several thousand metabolites, including proteins, metabolites, inflammatory markers such as cytokines and, to a degree, lipids. “It’s like Theranos, except it works,” corresponding author Michael Snyder, director of the Center for Genomics and Personalized Medicine at Stanford Medicine, told BioWorld.
The U.S. FDA has lifted the clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating adeno-associated virus (AAV) gene replacement therapy AT-845 in adults with late-onset Pompe disease.
Metabolic health is at an odd juncture. With the advent of glucagon-like peptide (GLP-1) agonists, pharmacologically induced weight loss has matured into a viable therapeutic option at long last. And research into the drug class is continuing apace.
The positively charged nanoparticle polyamidoamine generation 3 (P-G3) can be specifically targeted to either visceral or subcutaneous fat, and affects both types of fat in different ways, researchers from Columbia University reported in two papers recently published. The studies, published online in Nature Nanotechnology on Dec. 1, 2022, and in Biomaterials on Nov. 28, 2022, are both “a conceptual advance” and “quite amenable to translation,” co-corresponding author Kam Leong told BioWorld.
Neurons are specialized cells with a high metabolic demand to fulfill their function, survive or keep a healthy half-life. In this sense, the anabolism and catabolism of proteins and lipids could be associated to different neurodegenerative diseases. At the 2022 annual meeting of the Society for Neuroscience, scientists reported the latest discoveries on neuron metabolic needs at a session on 'Powering Thoughts: The Regulation of Neuronal Energy Metabolism and Mitochondria.'
Ardelyx Inc. could have a rocky row to hoe when it makes its case for tenapanor, as a hyperphosphatemia therapy in adults with chronic kidney disease, before the U.S. FDA’s Cardiovascular and Renal Drugs Advisory Committee Nov. 16. The big question facing the adcom is whether the change in baseline serum phosphorous levels achieved by the drug is clinically meaningful. Clearly, FDA reviewers don’t think so, as that question already has resulted in delayed PDUFA dates, a complete response letter and two formal dispute resolution requests.
Minoryx Therapeutics SL is banking what it described as “a significant double-digit up-front payment” and could earn up to €258 million (US$262.1 million) more in milestone payments and development funding from a license agreement with Neuraxpharm GmbH, which covers European rights to its lead drug candidate, leriglitazone, in central nervous system indications. It will also receive tiered double-digit royalties on product sales.
Innovent Biologics Inc. said that, compared to a placebo, both doses of its anti-PCSK-9 monoclonal antibody tafolecimab tested in a phase III trial “yielded significant and durable reductions” in low-density lipoprotein cholesterol levels and showed a favorable safety profile in Chinese patients with non-familial hypercholesterolemia.
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