Shares of Xenon Pharmaceuticals Inc. (NASDAQ:XENE) were trading at $30.25, down $5.60, or 15%, on word of the delay of data from the phase III X-tole2 trial with azetukalner in focal onset seizures (FOS). Xenon, with offices in Vancouver, British Columbia, and Boston, has moved the readout from the second half of this year to early 2026. Azetukalner is believed to work by targeting KCNQ2/3 (Kv7.2/Kv7.3) voltage-gated potassium channels. FOS, once known as partial seizures, are those that begin in one area of the brain and cause a variety of symptoms. They are estimated to afflict about 60% of people with epilepsy.

More TIGIT trouble: GSK and Iteos part ways

TIGITs took another tumble with GSK plc’s decision to end a development program and a collaboration with Iteos Therapeutics Inc. New top-line results from a phase II study of non-small-cell lung cancer using belrestotug, an anti-TIGIT monoclonal antibody, were disappointing so the companies are calling it a day. GSK and Iteos had planned to split the U.S. profits in a June 2021 deal that brought Iteos $625 million up front from GSK and promised as much as $1.45 billion in potential milestone payments. The breakup didn’t hurt Iteos’ stock (NASDAQ:ITOS) as shares were up 20% at midday to $8.32 each.

Azafaros raises €132M in series B for parallel phase III trials

Lysosomal storage disorder specialist Azafaros NV has raised €132 million (US$146.7 million) in a series B to fund two phase III studies of its lead product, a small-molecule glycosidase inhibitor that the company says has shown disease-modifying effects. The two trials will test nizubaglustat in GM1/GM2 gangliosidoses and in Niemann-Pick type C, and also lay the ground for extending the use of the brain-penetrant azasugar to the treatment other lysosomal storage disorders. “We are enrolling about 70 patients in each study and running global studies in both,” said Stefano Portolano, Azafaros CEO. “We plan to run them both in parallel.” Each will be an 18-month, placebo-controlled trial with an open label expansion phase.

Trump tariffs trigger U.S. capital commitments

A number of biopharma and med-tech companies are committing to expand manufacturing and other facilities in the U.S. Some shifts could be driven by the tariffs imposed by President Trump in 2025, signaling a strategic move to mitigate costs and ensure continued growth.

China’s NMPA gives thumbs up to Remegen’s ADC targeting HER2

China’s National Medical Products Administration has approved Remegen Co. Ltd.’s antibody-drug conjugate (ADC), disitamab vedotin (RC-48), for treatment of HER2-positive advanced breast cancer in patients with liver metastasis. Branded Aidexi, disitamab vedotin (DV) is the first original ADC developed by Remegen targeting the HER2 protein. It is the first ADC in China to receive dual recognition as a breakthrough therapy by the FDA in the U.S. and the NMPA in China. The company’s NDAs for the treatment of gastric cancer and uroepithelial carcinoma were reviewed under the priority review and approval process and were conditionally approved for marketing in China in June 2021 and December 2021, respectively.

Korea pharma stocks rally; Celltrion welcomes US drug pricing

Korean pharmaceutical stocks rose across the board May 13, a day after U.S. President Donald Trump signed off on the most favored nation (MFN) executive order, a drug pricing policy expected to benefit biosimilar makers in the U.S., according to Celltrion Inc. The Trump administration also announced that it secured an initial trade deal with China to pause the trade war for 90 days and to bring down tariffs on both sides by 115%. “Under the current Trump administration, the U.S. pharmaceutical market is undergoing rapid change under two major frameworks: ‘lowering drug prices’ to make Americans healthier and ‘raising import tariffs’ to strengthen U.S. pharmaceutical manufacturing,” according to a note from the Korea Biotechnology Industry Organization’s center of bioeconomy research. 

US lawmakers call for action in addressing Rx supply chain

In a continuing déjà vu, the Senate Judiciary Committee held yet another hearing today on pharmacy benefit managers, focusing on a lack of transparency. Tired of hearing after hearing filled with finger pointing and blame games that leave lawmakers thinking “What a mess,” Sen. Thom Tillis, R-N.C., asked Chair Chuck Grassley, R-Iowa, to convene a working group that could sit down with stakeholders throughout the biopharma supply chain in an environment outside of a congressional hearing to work out solutions to the problems that keep U.S. drug prices the highest in the world.

Tilt’s $25.6M series B advances TILT-123 for ovarian cancer

Tilt Biotherapeutics Ltd. has raised $25.6 million in a series B round that will fund phase II development of the lead oncolytic virus in the treatment of platinum resistant ovarian cancer. TILT-123 (igrelimogene litadenorepvec), designed for intravenous administration, is the only engineered oncolytic virus armed with two immunostimulatory cytokines: tumor necrosis factor and interleukin-2. The round closed soon after Helsinki, Finland-based Tilt published results of a phase I study of TILT-123 in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) in ovarian cancer. The data, also presented at the American Association of Cancer Research meeting in May, showed treatment was well-tolerated with an excellent safety profile and promising efficacy in some patients.

HHS, FDA ask for input on regs to kill

As they begin the work of weeding out old regulations, the U.S. Department of Health and Human Services (HHS) and the FDA are requesting comments to help them identify and eliminate outdated or unnecessary regulations. The request for information is the first step in implementing President Donald Trump’s executive order requiring agencies to kill 10 old regulations for every new one they implement.

FDA shift from animal testing fuels organoid, organ-on-chip demand

The U.S. FDA’s decision to phase out animal testing for INDs is driving a new market of alternative, nonanimal testing technologies like organoids and organs-on-a-chip, speakers at Bio Korea 2025 said. However, the products may not fully replace traditional mice studies, especially for rare or severe diseases like cancer. “Animal testing was utilized for 85 years until the FDA Modernization Act 2.0 (2021-2022), which moved to eliminate the requirements of animal testing for clinical trial clearance,” Humanase Co. Ltd. CEO Koh Sung-ho told attendees of the alternative toxicity testing session in Seoul, South Korea, May 9. “The legislation [opened the door] for alternative technologies like organoids starting January 2023.”

Also in the news

Abeona, Actinium, Aera, Agenus, Astrazeneca, Biodexa, CASI, Cytomx, Elevatebio, Entera, Fortrea, Genentech, Iaso, Innocare, Kaixin, Lexeo, Meiragtx, Roche, Thermosome, UTR, Valneva, Verastem