A prespecified interim analysis revealed that Astrazeneca plc’s oral factor D inhibitor danicopan met the primary endpoint of a phase III trial as an add-on therapy for patients with paroxysmal nocturnal hemoglobinuria (PNH) who were also taking a C5 inhibitor, but who still experienced extravascular hemolysis.
Bearish investors dwelling on a single grade 4 liver enzyme elevation seemed to be the cause for Intellia Therapeutics Inc.’s sinking stock Sept. 16, despite the company reporting impressive, though early stage, data for its leading systemically administered CRISPR candidates targeting hereditary angioedema (HAE) and amyloid transthyretin (ATTR) amyloidosis.
After a midstage trial showed no benefit from adding ELX-02 (exaluren) to Kalydeco (ivacaftor, Vertex Pharmaceuticals Inc.) for certain cystic fibrosis (CF) patients, Eloxx Pharmaceuticals Inc. said it will discuss next steps for the program, its lead candidate, with the trial's sponsor, the CF Foundation. A proof-of-concept trial for EXL-02 in the rare kidney disease Alport syndrome will start later this year, the company said.
Altimmune Inc. has come under scrutiny from investors after weight loss data from a phase Ib trial of its nonalcoholic fatty liver disease drug, pemvidutide, sent its shares on a roller coaster ride.
Top-line phase III results showing Hutchmed Ltd.’s targeted therapy fruquintinib reduced the risks of death and disease progression by 34% and 68%, respectively, for patients with metastatic colorectal cancer could “lay a solid foundation for Hutchmed to initiate discussions with regulatory authorities worldwide,” a spokesperson of Hutchmed, told BioWorld.
Rubius Therapeutics Inc. is taking a giant step back so that it can attempt taking a few steps forward. The company said it’s restructuring to focus on taking its Rubius Erythrocyte Design (RED) cell therapeutics platform from what it considers a good thing and turn it into a better one. Rubius engineers red blood cells to become cancer fighters but a lack of progress and the lure of similar but new technology could lead to bigger and better results.
Shares in Akero Therapeutics Inc. more than doubled in value after the company announced results from a phase IIb nonalcoholic steatohepatitis trial that it believes show lead candidate efruxifermin could challenge other candidates in development for the fatty liver disease targeted by so many biotechs and big pharmas.
An investigational drug developed in Cuba for the potential treatment of Alzheimer´s and Parkinson's diseases has moved forward to phase III trials there, where scientists are optimistic about results of previous studies. The candidate, named Neuroepo and branded Neuralcim, was developed by the Center of Molecular Immunology (also known by its Spanish acronym CIM), a state-owned lab in the Caribbean nation.
The gamma secretase inhibitor nirogacestat (Springworks Therapeutics Inc.) reduced the risk of disease progression by roughly 70% in a randomized placebo-controlled phase III trial in patients with desmoid tumors.
Data presented Sept. 9 at the European Society of Medical Oncology 2022 Congress showed impressive effects for KRAS inhibitors. But they also illustrated their limitations. Earlier-stage trials and researcher presentations, meanwhile, suggested ways those limitations might be addressed. Results from the Codebreak 200 study, presented in the day’s Presidential Symposium, were typical of the best that targeted therapies have to offer: large effects for brief time periods.
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