A recent paper in the Journal of Clinical Medicine quantified some of the potential bodily ravages ahead for patients with hypoparathyroidism (hypoPT), specifically the loss of kidney function, with the risk of chronic kidney disease going up every year by 11%. A handful of drug developers continue to advance prospects in hypoPT, where Ascendis Pharma A/S’ hormone replacement therapy Yorvipath (palopegteriparatide) – the first and only treatment for adults with the rare endocrine disease – has set the bar for efficacy.
Acute pancreatitis took center stage as Wall Street took heed of phase III data from Ionis Pharmaceuticals Inc. with olezarsen for severe hypertriglyceridemia (sHTG), while another player in the space, Arrowhead Pharmaceuticals Inc., signed a sizeable deal in a separate therapeutic area with Novartis AG.
To strengthen its development efforts, Novo Nordisk A/S will collaborate in a deal that could bring Replicate Bioscience Inc. about $550 million. In return, Novo is getting an exclusive, worldwide license to use Replicate’s self-replicating RNA (srRNA) platform to develop the candidates. The two are aiming at targets in cardiometabolic diseases that include treatments for obesity and type 2 diabetes.
Leal Therapeutics Inc. is taking advantage of new genetic data that suggest products from metabolic pathways are involved in psychiatric and neurodegenerative diseases.
A few years after it was founded with the aim of taking RNA therapies to the next level, Arnatar Therapeutics Inc. emerged from stealth, disclosing a $52 million series A round raised in 2024 as well as U.S. FDA orphan and rare pediatric disease designations for ART-4, an antisense oligonucleotide candidate targeting the root cause of Alagille syndrome.
Eli Lilly and Co.’s regulatory ducks are lined up nicely with the latest positive top-line results from the phase III Attain-2 trial testing orforglipron, an oral glucagon-like peptide 1 (GLP-1) receptor agonist, in adults with obesity or overweight and type 2 diabetes.
Multiple datasets are due to roll out soon in the Prader-Willi syndrome (PWS) space, where contenders are trying varied mechanisms of action to tackle the rare genetic disorder characterized by insatiable hunger, or hyperphagia. In the mix are Aardvark Therapeutics Inc., Acadia Pharmaceuticals Inc., Rhythm Pharmaceuticals Inc. and Soleno Therapeutics Inc.
Rates of adverse events (AEs) gave pause to some on Wall Street, and shares of Viking Therapeutics Inc. (NASDAQ:VKTX) fell 42%, or $17.73, to close Aug. 19 at $24.36 after the firm reported 13-week data from the phase II trial with the oral tablet form of VK-2735, the company's dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors.
With the U.S. FDA clearance of the sNDA for Wegovy (semaglutide) from Novo Nordisk A/S to treat noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH), Wall Street speculation deepened as to the consequences for Madrigal Pharmaceuticals Inc.’s Rezdiffra (resmetirom), a thyroid hormone receptor-beta (THR-beta) agonist that won the MASH go-ahead last year.
Superluminal Medicines Inc. and Eli Lilly and Co. are collaborating in a deal to develop cardiometabolic disease and obesity therapies by aiming at undisclosed G protein-coupled receptor targets. The deal could bring Superluminal as much as $1.3 billion, including up-front and near-term investments, an equity investment, development and commercial milestones, plus tiered royalties on net sales.
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