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BioWorld - Sunday, July 12, 2026
Home » Topics » Drugs » Small molecule

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Cardiovascular

AZD-0780 lowers LDL-C plasma levels by stabilizing the PCSK9 C-terminal domain

Sep. 5, 2024
Researchers from Astrazeneca plc presented the structure and preclinical characterization of a novel PCSK9 inhibitor, AZD-0780, being developed for the treatment of cardiovascular disease. A new potentially druggable functional binding pocket was identified on the PCSK9 C-terminal domain (CTD), and multiple fragment screens generated a single CTD-binding hit.
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Fosgo death blow? Athira theory intact post-phase II/III AD bust

Sep. 4, 2024
By Randy Osborne
Athira CEO Mark Litton said the firm is “evaluating all the options” after disclosing top-line results from its phase II/III Lift-AD clinical trial of fosgonimeton (fosgo), a hepatocyte growth factor-positive modulator, in patients with mild to moderate Alzheimer’s disease (AD). Javier San Martin, chief medical officer, said the company will schedule “a conversation at the right time” with the U.S. FDA.
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Jacobio chairman, CEO Yinxiang Wang (right) and Allist chairman and general manager Jinhao Du at a signing ceremony Aug. 30

Jacobio, Allist shake on ¥900M China deal for lung cancer combo

Sep. 3, 2024
By Marian (YoonJee) Chu
Jacobio Pharmaceuticals Group Co. Ltd. out-licensed rights for two lung cancer assets in China to Shanghai Allist Pharmaceuticals Co. Ltd. through a potential ¥900 million (US$126.4 million) deal. Beijing-headquartered Jacobio said Aug. 30 that it signed off development, regulatory and commercial milestone rights to both glecirasib, a KRAS G12C inhibitor first targeting non-small-cell lung cancer, and a SHP2 inhibitor called JAB-3312.
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Blood pressure gauge, ECG and medication

George’s triple-drug hypertension combo succeeds in phase III

Sep. 3, 2024
By Nuala Moran
George Medicines Ltd. has published positive data from two phase III studies showing GMRx2, a low-dose triple-drug combination treatment for hypertension, is superior to dual combinations of its components.
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Sanofi BTK inhibitor tolebrutinib hits phase III endpoints in MS

Sep. 3, 2024
By Tamra Sami
Sanofi SA’s brain-penetrant Bruton's tyrosine kinase (BTK) inhibitor, tolebrutinib, met the primary endpoint in the phase III Hercules trial in non-relapsing secondary progressive multiple sclerosis (nrSPMS). The first compound to show reduction in disability accumulation in MS, tolebrutinib delayed the time to onset of confirmed disability progression in people with nrSPMS, a population for which there are currently no approved therapies.
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Brain activity concept illustration

Despite clinical failure, new analysis supports Actinogen's Xanamem

Sep. 3, 2024
By Tamra Sami
After initially reporting that Xanamem failed to meet the primary endpoint in cognitive attention in the phase IIa Xanacidd cognition and depression trial, Actinogen Medical Ltd. CEO Steven Gourlay said a new analysis shows clinically and statistically significant benefits in depression.
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Yunovia cleared for Korea phase I trial of small-molecule GLP-1 asset

Sep. 3, 2024
By Marian (YoonJee) Chu
Yunovia Co. Ltd. gained clearance in South Korea to start a multiple ascending dose phase I study for ID-110521156 – a novel, orally available, small-molecule, glucagon-like peptide-1 (GLP-1) agonist.
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Cancer

Cumulus raising $50M, spinning GPR68 small molecules into GIO

Sep. 2, 2024
By Nuala Moran
Cumulus Oncology Ltd. is in the thick of raising a $50 million series A round as its model of sourcing novel drug targets emerging from academia, shaping them up for clinical development, and spinning them into startups, gathers pace. At the same time, Nodus Oncology Ltd., the first spinout created around an acquired asset, has just reached in vivo proof of concept with its lead DNA damage response inhibitor, and it, too, is looking to raise a series A to take the program through to the end of phase I.
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Brain activity concept illustration

Despite clinical failure, new analysis supports Actinogen's Xanamem

Aug. 30, 2024
By Tamra Sami
After initially reporting that Xanamem failed to meet the primary endpoint in cognitive attention in the phase IIa Xanacidd cognition and depression trial, Actinogen Medical Ltd. CEO Steven Gourlay said a new analysis shows clinically and statistically significant benefits in depression.
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3D dollar sign

Cumulus raising $50M, spinning GPR68 small molecules into GIO

Aug. 29, 2024
By Nuala Moran
Cumulus Oncology Ltd. is in the thick of raising a $50 million series A round as its model of sourcing novel drug targets emerging from academia, shaping them up for clinical development, and spinning them into startups, gathers pace. At the same time, Nodus Oncology Ltd., the first spinout created around an acquired asset, has just reached in vivo proof of concept with its lead DNA damage response inhibitor, and it, too, is looking to raise a series A to take the program through to the end of phase I.
Read More
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