Becoming the first treatment for rare genetic disorder Prader-Willi syndrome, breakthrough therapy drug DCCR (diazoxide choline) gained U.S. FDA approval as Vykat XR the evening before its March 27 PDUFA date, sending shares of developer Soleno Therapeutics Inc. up 38%.
Losing nearly 60% of its value, Mural Oncology plc backed away from its lead interleukin-2 (IL-2) drug, nemvaleukin alfa, in platinum-resistant ovarian cancer based on interim results showing it failed to reach significance in the overall survival primary endpoint.
Elevation Oncology Inc. has nixed its lead pipeline product, a claudin 18.2 antibody-drug conjugate (ADC) called EO-3021, on disappointing phase I data, sending shares tumbling by 42% and placing its preclinical HER3 ADC to the forefront of development.
Cardiothoracic surgeon and television personality Mehmet Oz, President Donald Trump’s nominee for administrator of the Centers for Medicare & Medicaid Services (CMS), faced the U.S. Senate’s finance committee on March 14, suggesting the implementation of artificial intelligence (AI) technologies and the reduction of drug prices would be among his top priorities, if confirmed.
Bristol Myers Squibb Co.’s decision this week to snag Bluebird Bio Inc. spinout 2seventy Bio Inc. for $102 million net – just weeks after investors bid $30 million for Bluebird itself – seemed to place a final blow on what was once a promising gene therapy company. The space in general has struggled to make business sense out of the one-time therapies that often involve complicated manufacturing and exorbitant prices, despite the life-changing value that gene therapies bring to patients. But despite some recent setbacks, biopharmas continue to plow forward with promising research in the field.
Trimtech Therapeutics closed a £25 million (US$31 million) oversubscribed seed funding round to advance its targeted protein degradation treatments for neurodegenerative and inflammatory diseases.
Trimtech Therapeutics closed a £25 million (US$31 million) oversubscribed seed funding round to advance its targeted protein degradation treatments for neurodegenerative and inflammatory diseases.
About 17 months after emerging from stealth with its Trueglue discovery platform, Magnet Biomedicine Inc. has joined with Eli Lilly and Co. to discover, develop and commercialize molecular glue therapeutics for oncology. “It’s a really important deal, especially given the discovery of Trueglues is something Magnet is pioneering,” CEO Brian Safina told BioWorld. The deal includes $40 million in up-front and near-term payments, including an equity investment, as well as potential milestone payments, bringing the deal total to more than $1.25 billion, plus tiered royalties.
The prospects of an oral obesity candidate used as a monotherapy or combination therapy is in question after Kallyope Inc. posted disappointing phase II data on a government website, indicating that its lead candidate, K-757, failed to meet all endpoints.