On Aug. 21, the Supreme Court handed the Trump administration another partial victory in its attempts to defund the NIH. In a 5-4 decision, the justices paused a June 16 District Court order to restore $783 million in funding for NIH research grants focusing on gender and diversity, equity and inclusion. The funding had first been cut through a series of executive orders shortly after President Donald Trump resumed power in January. As NIH funding has been cut and politicized, companies, foundations and private individuals have stepped in to replace some fraction of the lost funds. The latest example is Immunai Inc., which announced late this week that it was launching an opportunity for academic researchers to gain access to its single-cell sequencing and analysis platform, which includes single-cell transcriptomic, surface proteomic and T-cell receptor profiling.
UK drug pricing talks called off without a deal
Negotiations between the U.K. government and the pharmaceutical industry have broken down with no agreement over the level of rebates companies must pay back to the government under the current voluntary pricing scheme. The two sides have been locked in discussions since April, when the government agreed to bring forward a midterm review of the Voluntary Scheme for Branded Medicines Pricing, Access and Growth. That was in response to soaring rebate rates, which in 2025 require pharma companies to pay back between 23.5% to 35.6% of their sales of branded drugs to the National Health Service.
Prader-Willi syndrome research to yield more results soon
Heating up as an indication is Prader-Willi syndrome (PWS), the complex disease characterized by insatiable hunger, or hyperphagia. Developers in the space include Aardvark Therapeutics Inc., Acadia Pharmaceuticals Inc., Rhythm Pharmaceuticals Inc., and Soleno Therapeutics Inc. Next to report data is Boston-based Rhythm, with phase II findings due in the second half of this year from testing in PWS of Imcivree (setmelanotide), the melanocortin-4 receptor agonist first approved by the U.S. FDA in 2020 for weight management.
US FDA approves six NMEs in July, sending up shares of Kalvista, PTC, Regeneron
The U.S. FDA approved 17 drugs in July, down from 23 in June, bringing the year-to-date total to 125. Six new molecular entities (NMEs) were approved in the month: Regeneron Pharmaceuticals Inc.'s Lynozyfic (linvoseltamab), Dizal (Jiangsu) Pharmaceutical Co. Ltd.’s Zegfrovy (sunvozertinib), Kalvista Pharmaceuticals Inc.’s Ekterly (sebetralstat), Leo Pharma A/S' Anzupgo (delgocitinib), PTC Therapeutics Inc. 's Sephience (sepiapterin) and Lenz Therapeutics Inc.’s Vizz (aceclidine ophthalmic solution).
Rare immune disease inspires broad-spectrum antiviral mRNA
Deficiencies in interferon-stimulated gene 15 (ISG15), a protein that normally regulates the immune response, causes mild but persistent inflammation. However, its absence also provides an unexpected advantage by increasing resistance to viral infections. Inspired by this condition and using mRNA technology, scientists at Columbia University and the Icahn School of Medicine at Mount Sinai have developed a broad-spectrum antiviral platform. The discovery proved effective in small animals against COVID-19, influenza and Zika.
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Abbvie, Advanz, Allos, Alvotech, Aptose, Beone, Bioaegix, Biodlink, Circle Pharma, Crinetics, Denali Capital, Dynavax, Elix, Immuneering, Interius, Invivyd, IRLAB, Keros, Kite, Kyorin, Larkspur, Lilly Neuroscience, Medicus, Molecular Partners, Nervgen Pharma, Nicox, Novavax, Nucleus Radiopharma, Nurexone, Realta Life Sciences, Regeneron, Rhythm, Sana, Scilex Holding Co., Semnur, Stealth, Strand, Tivic, Trinity Biotech, Veongen