The synergy – or lack of it – between knockdown drugs and stabilizers in treating transthyretin (TTR)-mediated amyloid cardiomyopathy (ATTR-CM) became one of the topics for debate in the aftermath of the phase III fizzle with gene silencer Wainua (eplontersen), advanced by Astrazeneca plc and Ionis Pharmaceuticals Inc.
While biomedical resources in the form of specialized tools, hundreds of thousands of published papers and huge repositories of ‘omics, health records and other data, are growing exponentially, discovery is getting slower and more expensive. That is the perspective from which scientists at Stanford University approached the development of their artificial intelligence (AI) research assistant Biomni.
News of the unexpected phase III failure of the gene silencing drug Wainua (eplontersen) in the treatment of transthyretin-mediated amyloid cardiomyopathy sent Astrazeneca plc’s shares (NYSE:AZN) down $10.88 to close July 9 at $178.40.
What appears to be a routine item on a routine list of the U.S. Department of Health and Human Services’ (HHS) proposed rulemakings for the next fiscal year is being seen as anything but routine, given HHS Secretary Robert Kennedy’s stance on vaccines and several commonly used drugs.
Encouraging phase I data this week of new treatments for vitiligo, a chronic autoimmune disorder that destroys melanocytes, drove investor excitement, with Forte Biosciences Inc. the latest company to disclose a statistically significant benefit.
While the emergence of immune checkpoint inhibitor (ICI) therapy in recent years has significantly improved cancer outcomes, some patients have been unable to experience the full therapeutic benefits of ICIs due to significant gastrointestinal inflammation linked to treatment. Seres Therapeutics Inc. is looking to change that with its live biotherapeutic candidate, SER-155, offering impressive findings from phase I data in ICI-related enterocolitis, or irEC.
Of all the U.S. SEC’s recent proposed rules to make going, and staying, public more attractive, perhaps the most beneficial for biopharma and med-tech startups is the amendment that would give public companies the flexibility to file semi-annual rather than quarterly reports, Ben Bradford, head of external affairs at Massbio, told BioWorld.
Astrazeneca plc has added a dual PDE3/4 inhibitor candidate to its chronic obstructive pulmonary disease (COPD) portfolio via a deal worth up to $2.1 billion with Chia Tai Tianqing Pharmaceutical Group Co. Ltd., a Sino Biopharmaceutical Ltd. subsidiary.
After years of hearings, finger-pointing and litigation, the U.S. Congress finally seems to be getting serious about fixing the 340B prescription drug discount program, which today is the second largest federal drug program.
Tvardi Therapeutics Inc. rebounded from a phase II blowup last fall in idiopathic pulmonary fibrosis (IPF) with oral STAT3 inhibitor TTI-101 by showing that a next-gen phosphate prodrug dubbed TTI-109 kept its potency in phase I without gastrointestinal (GI) troubles.