Shares of Insmed Inc. (NASDAQ:INSM) were trading midday at $26.50, up $3.86, or 17%, on positive top-line results from the phase III study called Arise with Arikayce (amikacin) in patients with newly diagnosed or recurrent nontuberculous mycobacterial lung infection caused by Mycobacterium avium complex (MAC) who had not started antibiotics. The study met its primary objective of demonstrating that the Quality of Life-Bronchiectasis (QOL-B) respiratory domain works effectively as a patient-reported outcome (PRO) instrument in patients with MAC lung disease. Bridgewater, N.J.-based Insmed will propose to the U.S. FDA that the QOL-B respiratory domain PRO be the primary endpoint for the ongoing, registrational phase III Encore study without any modifications.

Star adds $90M series C for VWD drug, additional company formations

Continuing its goal of creating a constellation of portfolio companies, Star Therapeutics Inc. closed a $90 million series C round. That brings the firm’s total funding to more than $190 million. Since emerging from stealth mode in early 2022, Star has launched two portfolio firms, Electra Therapeutics Inc. and Vega Therapeutics Inc. The latest financing will support Vega’s clinical-stage drug for von Willebrand disease (VWD) as well as support the launch of additional portfolio firms that continue Star’s in-house discovery efforts in immunology and hematology.

Insilico inches closer to claiming title of first generative AI drug developer

As a pioneer of harnessing artificial intelligence (AI) to drug discovery, Insilico Medicine Inc. recently made breakaway progress to bring a generative AI-designed drug to life, announcing the progression of its INS018-055 candidate to phase II trials for idiopathic pulmonary fibrosis. What sets the Hong Kong and New York-based Insilico apart from the multitudes of AI-based biotech firms, however, is that the company built its own end-to-end generative AI platform and, importantly, licenses its technology and therapeutic assets to the wider industry to accelerate drug discovery. “Many so-called AI biotechs are popping up, but we have to ask what makes companies a true AI biotech,” Insilico Medicine founder and CEO Alex Zhavoronkov told BioWorld. The answer, he said, comes down to whether the firm has proprietary AI software with a large user base that can validate the program. “Anyone can use AI and generative AI software,” he said. “Not many people have their own programs.”

Court challenges to IRA Rx price negotiations piling up

The queue of lawsuits challenging the constitutionality of the U.S. Inflation Reduction Act’s (IRA) prescription drug price negotiations continues to grow. Novartis AG is the latest drug company, but probably not the last, to join the line. It filed its challenge in federal court in New Jersey Friday, a few days after the Centers for Medicare & Medicaid Services included the company’s heart failure drug, Entresto (sacubitril and valsartan), on its list of the 10 drugs that will be subject to the first round of IRA negotiations.

Otsuka to acquire Mindset for $50M in move to bolster psychiatric pipeline 

Otsuka Pharmaceutical Co. Ltd. announced plans to acquire Mindset Pharma Inc. for C$80 million (US$50.76 million) in an all-cash deal that will see Otsuka strengthen its neurology and psychiatric pipeline. Mindset, of Toronto, is developing next-generation psychedelic therapeutics to treat psychiatric and neurological disorders with high unmet needs. It was one of the first biotechs in the psychedelic space to apply medicinal chemistry and behavioral pharmacology techniques to novel scaffolds of psychedelic drugs with the goal of discovering new medicines. Otsuka has invested in the psychedelic space since 2020, in part through its U.S.-based, early stage investment arm, The McQuade Center for Strategic Research and Development (MSRD), an Otsuka spokesperson told BioWorld.

Biopharmas receive $1.43B in BARDA grants in 2023 

Biopharma grants are up nearly 150% compared to the same time period last year, largely due to a number of $100 million-plus grants from the Biomedical Advanced Research and Development Authority (BARDA). Meanwhile, nonprofit deal value has declined year-over-year, with the number of transactions down 42.21%.

Holiday notice

BioWorld's offices were closed in observance of Labor Day in the U.S. No issue was published Monday, Sept. 4.

Also in the news

Acticor, AI Therapeutics, Alvotech, Bavarian Nordic, Betterlife, Biontech, Bitt, Bristol Myers Squibb, Cadrenal, Carisma, Centogene, Daré, Engitix, Eterna, Foghorn, Fuji, Genentech, Glyscend, Goodlife, Grifols, GSK, Invios, Iomx, Ipsen, Macrogenics, Merck, Mirum, Nestlé, Novozymes, Nuvox, Olema, Pfizer, Pleco, Portage, Roche, Sernova, Sinovac, Solid, Stallergenes Greer, TC Biopharm, Tetris, Theragenix, Travere, Tvardi, Vistagen, Xilio, Zenas