Pulmonary arterial hypertension (PAH), characterized by vasoconstriction and pulmonary vascular remodeling, has a 10% annual mortality rate among patients due to right heart failure. There are genetic variants known to impact the risk of PAH, but susceptibility from epigenetic changes is poorly understood.
BMPR2 mutations are the most common genetic cause of pulmonary arterial hypertension (PAH). Pulmonary artery endothelial cells (PAECs) with reduced BMPR2 expression are linked to a persistent DNA damage after reoxygenation. Forkhead box F1 (FOXF1) is a transcription factor with affinity for endothelial cells in the lung, and its reduced expression has also been associated with DNA damage in those cells and PAH.
Zymedi Co. Ltd. has signed a clinical cooperative research and development agreement (CRADA) with the National Heart, Lung and Blood Institute (NHLBI), part of the National Institutes of Health (NIH), to develop ZMA-001 for the treatment of pulmonary arterial hypertension (PAH).
A few weeks after Gossamer Bio Inc. said it was pausing enrollment in a phase Ib/II study of CNS-penetrant BTK inhibitor GB-5121 in relapsed/refractory CNS lymphoma, citing the drug’s risk/benefit profile observed to date and a prioritization of resources, the company is dropping the drug’s development entirely.
Actelion Pharmaceuticals Ltd. has disclosed platelet-derived growth factor receptor beta (PDGFR-β) inhibitors reported to be useful for the treatment of pulmonary arterial hypertension.
Gossamer Bio Inc. shares (NASDAQ:GOSS) fell 74.6% to $2.36 Dec. 6 after the disclosure of phase II results that, while meeting the study's primary endpoint of reducing pulmonary vascular resistance vs. placebo, failed to show the company's pulmonary arterial hypertension (PAH) candidate, seralutinib, holding its own against the performance of Merck & Co. Inc.'s PAH candidate, sotatercept, in separate PAH studies.
The closely watched pivotal phase III trial called Stellar by Merck & Co. Inc. with sotatercept delivered in a big way for the company, helping to validate the firm’s $11.5 billion buyout about a year ago of Acceleron Pharma Inc.
Aqualung Therapeutics Corp. has been awarded two 3-year National Institutes of Health (NIH) Fast Track awards to support development of ALT-100 (enamptcumab), a humanized monoclonal antibody therapy for the chronic indications of pulmonary arterial hypertension (PAH) and inflammatory bowel disease (IBD).
