Argo Biopharmaceutical Co. Ltd. and its RNAi work drew Novartis AG back to the table for a third time, as the companies entered a potential $5.2 billion deal involving cardiovascular-focused assets, including a right to first negotiation for BW-00112, an angiopoietin-like protein 3 (ANGPTL3)-targeting siRNA in phase II testing in mixed dyslipidemia. Under the terms, Argo receives $160 million up front, with the remaining value tied to milestone and option payments. All are on top of the two deals announced in early 2024, also focused on cardiovascular disease, bringing the companies’ potential collaborations to more than $9 billion.

OBBB changes the odds for next round of US Rx price negotiations

Oddsmakers placing their bets on which drugs would be in play for round 3 of the U.S. Inflation Reduction Act (IRA) price negotiations are doing some reshuffling, thanks to an orphan drug provision tucked into the Trump administration’s One Big Beautiful Bill (OBBB) that was signed into law on the Fourth of July. The 2022 IRA exempted drugs with only one orphan indication from the Medicare price negotiations. The OBBB changed that, exempting drugs with multiple orphan indications – provided they had no nonorphan indication. It also changed the reckoning of when a drug with both orphan and nonorphan indications would be eligible for selection, making it clear that the clock started based on when the first nonorphan indication was approved. Consequently, some of the drugs, which were considered sure bets for round 3, including Merck & Co. Inc.’s mega-blockbuster Keytruda, are either now off the table or delayed.

Wave Life says data are positive but stock wavers

As Wave Life Sciences Ltd. released more results from its ongoing phase Ib/IIa study of small interfering RNA editing oligonucleotide WVE-006 for treating alpha-1 antitrypsin deficiency (AATD), the company’s stock (NASDAQ:WVE) dropped by about 20%. Shares were going for $7.68 each at midday. The company called the data positive and said it showed a durable production of serum alpha-1 antitrypsin protein at levels associated with a lower risk of AATD liver and lung diseases after patients received multiple 200-mg doses of WVE-006. AATD is a rare and genetic condition that can lead to lung and liver disease.

Kennedy critic axed from FDA vaccine adcom

The vaccine dominoes continue to fall in the U.S. This time one fell on the FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC), as one of Health and Human Services Secretary Robert Kennedy’s most outspoken critics was removed from the panel nearly a year and a half before his term was to expire. While word broke yesterday that Paul Offit was dismissed from the committee, the VRBPAC roster, which was last updated Thursday, doesn’t include Offit, an infectious disease physician and co-inventor of the rotavirus vaccine. At the same time, the roster indicates there are no vacancies on the committee, but it does include the standard adcom language: “Note, one or more vacancies may be in the nomination process or a final appointment may have been made.”

Argenica’s stock drops 56% on mixed phase II results

Argenica Therapeutics Ltd. saw its stock drop 56% following mixed results for its ARG-007 phase II Seancon trial in acute ischemic stroke patients undergoing endovascular revascularization, or thrombectomy. Although ARG-007 met the primary endpoint of safety and was well-tolerated, efficacy was lacking, and the compound did not show a treatment effect compared to placebo. ARG-007 is a glutamate receptor modulator that is believed to reduce brain tissue death after stroke.

ESC 2025: From heart failure to hypertension, research advances

There have been numerous improvements in the treatment of cardiovascular disease since the European Society of Cardiology (ESC) first met in 1950, but unmet medical need remains, and the science continues to advance, as delegates heard at the 75^th annual meeting in Madrid, Spain, Aug. 29-Sept. 1. More than 40 clinical trials were presented at the conference, covering new therapies, repurposing, label extensions and real-world studies, for conditions ranging from heart failure to hypertension. BioWorld presents some of the highlights.

South Korea IND roundup: Pimedbio, Sillajen, Genosco, SK Bio, Ami

Several South Korean biotech and biopharmaceutical companies completed IND submissions or won nods to start clinical trials in either the U.S. or South Korea, including SK Bioscience Co. Ltd., Genosco Inc., Pimedbio Inc., Sillajen Inc. and Ami Pharm Co. Ltd.

Also in the news

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