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BioWorld - Thursday, January 1, 2026
Home » Topics » Cardiovascular, BioWorld

Cardiovascular, BioWorld
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Heart scientific overlay

‘Enhancement’ or delay to Helios-B? Alnylam falls, Bridgebio rises

Feb. 16, 2024
By Karen Carey
Alnylam Pharmaceuticals Inc.’s decision to change its analysis plan for the phase III Helios-B trial of RNAi therapeutic Amvuttra (vutrisiran) to treat transthyretin amyloidosis with cardiomyopathy, pushing top-line results back by three months, pressured its share price (NASDAQ:ALNY) down by 10% on Feb. 15, while also boosting shares of competitor Bridgebio Pharma Inc. (NASDAQ:BBIO) by 14%.
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Woman in pain holding hand over heart

CSL’s phase III misses primary endpoint in reducing cardiac events

Feb. 12, 2024
By Tamra Sami
CSL Ltd. will review the data further to see if there is a path forward for CSL-112 (human apolipoprotein A-I) after the phase III AEGIS-II trial failed to meet the primary efficacy endpoint in reducing the risk of major adverse cardiovascular events in patients following an acute heart attack.
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3D illustration of heart cross section

Bridgebio seeks Japan approval of acoramidis for rare heart disease

Feb. 5, 2024
By Marian (YoonJee) Chu
Armed with strong phase III safety data in Japanese patients, Palo Alto, Calif.-based Bridgebio Pharma Inc. is planning to file for Japan approval of its investigational drug acoramidis for a rare heart disorder.
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Doctor with stethoscope listening to heart of girl

FDA offers life support to Mesoblast’s Revascor via rare pediatric disease designation

Jan. 22, 2024
By Tamra Sami
The U.S. FDA has granted Mesoblast Ltd.’s allogeneic cell therapy Revascor (rexlemestrocel-L) rare pediatric disease designation following submission of results from a clinical trial in children with hypoplastic left heart syndrome, a potentially life-threatening congenital heart condition.
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Gene therapy next in HCM? Tenaya making strides

Jan. 18, 2024
By Randy Osborne
With one approved myosin inhibitor on the market and another coming up fast, researchers such as those at Tenaya Therapeutics Inc. are casting for new strategies to treat hypertrophic cardiomyopathy (HCM).
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Blue heart and data grid

Windtree out-licenses China rights for heart failure candidate istaroxime to Lee’s Pharma for $138M

Jan. 18, 2024
By Tamra Sami
Windtree Therapeutics Inc. is out-licensing China rights for phase III-ready heart failure candidate istaroxime to Lee’s Pharmaceutical Ltd. for $138 million, plus royalties. Lee’s, of Hong Kong, plans to begin a phase III study for istaroxime in acute heart failure in greater China, while Windtree, of Warrington, Pa., will conduct a global trial in cardiogenic shock, a form of sudden heart failure. Windtree reported positive phase II data in April 2022 that tested istaroxime in cardiogenic shock, which happens when the heart cannot pump enough blood and oxygen to vital organs.
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China’s Ji Xing signs flurry of Asian deals to advance cardiovascular pipeline and global ambitions

Jan. 12, 2024
By Tamra Sami
Shanghai-based Ji Xing Pharmaceuticals has signed a number of licensing deals over the last week for China rights to cardiovascular drugs to advance its pipeline and its global ambitions, partnering most recently with Tokyo-based TMS Co. Ltd. after the Chinese company acquired global rights for TMS-007 (also known as BIIB-131) from Biogen Inc.
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3D illustration of liver and photomicrograph showing triglyceride fat accumulated in liver cells.

Boehringer backs £30M study to find new liver disease treatments

Jan. 9, 2024
By Shani Alexander
Boehringer Ingelheim GmbH partnered with Newcastle University and the University of Edinburgh to fund a £30 million (US$38 million) study into liver cirrhosis in a bid to provide scientists with new insights into liver health. The partners hope that the study will not only enhance the understanding of nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH) cirrhosis, but also help identify translational biomarkers using a cutting-edge approach called single-cell RNA sequencing to accelerate the development of future therapies.
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RNA strand

Argo, Novartis sign two deals for CV assets for potentially $4B+

Jan. 8, 2024
By Marian (YoonJee) Chu
Announcing two licensing deals with Swiss pharma giant Novartis AG, Shanghai-based Argo Biopharmaceutical Co. Ltd. said on Jan. 7 that it stands to gain up to $4.165 billion for two of its cardiovascular assets combined. Marking the “first significant overseas out-licensing transaction in the RNAi field from a Chinese biotech company,” the deal includes an up-front payment of $185 million from Novartis to Argo.
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Applied Therapeutics falls on phase III miss in diabetic cardiomyopathy

Jan. 5, 2024
Shares of Applied Therapeutics Inc. (NASDAQ:APLT) dropped 36% Jan. 5 on news that its oral aldose reductase inhibitor, AT-001 (caficrestat), failed to reach statistical significance in a phase III study in patients with diabetic cardiomyopathy. The New York-based firm, however, pointed to positive trends in the Arise-HF study that could offer a path forward, albeit via a partner.
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