In Duchenne muscular dystrophy (DMD), deficiency of dystrophin leads to cardiomyocyte membrane instability, abnormal calcium influx, and progressive fibrotic remodeling of cardiac tissue. Histone deacetylase 6 (HDAC6) contributes to disease progression by regulating cytoskeletal dynamics and proteostasis in dystrophic muscle cells. Consequently, inhibition of HDAC6 represents a potential therapeutic strategy for addressing both the skeletal and cardiac manifestations of DMD.
Taiwan plans to invest NT$24 billion (US$752 million) over four years to bolster national biosecurity, expand domestic pharmaceutical production and safeguard against global supply chain volatility.
Although Aisa Pharma Inc’s cilnidipine (AISA-021) failed to meet the primary endpoint, multiple secondary endpoints showed statistically significant improvement in treating sclerosis-associated Raynaud's phenomenon, potentially positioning the drug for a phase III program in a disease with no approved oral therapies globally.
Entering its first major cardiovascular disease collaboration with a biopharma company, while it advances two internal gene therapies, Tenaya Therapeutics Inc. signed on with Alnylam Pharmaceuticals Inc. to deliver up to 15 novel genetic targets that could lead to new heart disease medicines. The deal comes with $10 million up front, and up to $1.13 billion is available to South San Francisco-based Tenaya if all targets meet certain milestones, leading to approved therapeutics that Alnylam develops and commercializes.
Jiangsu Hengrui Pharmaceuticals Co. Ltd. and Shanghai Hengrui Pharmaceutical Co. Ltd. have disclosed amide-substituted cycloalkyl compounds acting as relaxin receptor 1 (RXFP1; LGR7) agonists potentially useful for the treatment of heart failure and hypertension.
Entering its first major cardiovascular disease collaboration with a biopharma company, while it advances two internal gene therapies, Tenaya Therapeutics Inc. signed on with Alnylam Pharmaceuticals Inc. to deliver up to 15 novel genetic targets that could lead to new heart disease medicines. The deal comes with $10 million up front, and up to $1.13 billion is available to South San Francisco-based Tenaya if all targets meet certain milestones, leading to approved therapeutics that Alnylam develops and commercializes.
Polares Medical SA raised $50 million in a series C financing round for Mrace, its posterior leaflet replacement system designed to treat mitral regurgitation.
GSK plc is paying $950 million cash to acquire 100% of 35Pharma Inc.’s equity and pipeline, including phase II-ready candidate HS-235 for pulmonary hypertension. The acquisition agreement comes a day after GSK added two small interfering RNA assets from Nanjing, China-based Frontier Biotechnologies Inc. through a license deal reaching up to $1 billion.
China’s National Medical Products Administration has accepted for review Jiuyuan Genetic Biopharmaceutical Co. Ltd.’s drug application for Jikeqin, a biosimilar product to Novo Nordisk A/S’s Wegovy (semaglutide) for obesity.
Another phase III failure of Theravance Biopharma Inc.’s norepinephrine reuptake inhibitor ampreloxetine to treat symptomatic neurogenic orthostatic hypotension, this time in patients with the rare disease multiple system atrophy, means the end of the company’s R&D efforts. Cutting its workforce in half and terminating all development of ampreloxetine, its only pipeline product, the Dublin-based company’s shares (NASDAQ:TBPH) sank by 26%, or $4.99, to close March 3 at $13.96.
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