Becoming the second drug approved by the U.S. FDA for tenosynovial giant cell tumors, Deciphera Pharmaceuticals Inc.’s oral CSF1R inhibitor vimseltinib, newly branded Romvimza, could take significant market share from a competitor in the same class. A phase III trial showed a higher objective response rate with Romvimza than a separate phase III trial showed with Daiichi Sankyo Co. Ltd.’s Turalio (pexidartinib), and the latter’s label contains a boxed warning on rare but serious and sometimes life-threatening liver side effects. The label for Romvimza contains a warning of those effects, while also mentioning that they have not been observed with Romvimza.
AAAS 2025: CAR T approach shows early promise in MS trial
The first clinical data from an FDA-approved trial of a CAR T-cell therapy in the treatment of multiple sclerosis (MS) show the cells cause complete B-cell depletion, leading on to a reset of the immune system that is accompanied by an improvement in symptoms. Only four patients have been treated so far but, said Jeffrey Dunn, professor of neurology and neurological science at Stanford University, who is leading the open-label trial, “This is potentially a paradigm changer.”
Thousands of US HHS staff terminated
As part of a U.S. government-wide reduction in force, more than 5,000 Health and Human Services (HHS) employees received their pink slips over the weekend, with more than a fifth of those at the NIH. Most of the terminated employees were probationary staff who had been hired or promoted within the past year or so. When asked about the cuts, Andrew Nixon, director of communications at HHS, told BioWorld that the action follows the administration’s guidance and supports President Donald Trump’s “broader efforts to restructure and streamline the federal government.” The majority of NIH terminations were program officers, science review officers, researchers and directors, Leerink Partners LLC’s Puneet Souda said. Noting that the NIH cuts were fewer than had been expected, Souda added that “it is unclear if these are just phase 1 layoffs with more to come.”
AAAS 2025: Former NIH head Bertagnolli sounds warning on funding cuts
The former head of the U.S. NIH has sounded a warning that the uncertainty caused by the Trump Administration’s funding cuts and layoffs is blocking “critical work” and “paralyzing” biomedical research. “Every time we launch a new program, every time we continue to commit resources to ongoing work, those are important decisions that we make every single day, and in times like this, that decision-making is paralyzed,” Monica Bertagnolli, who stood down as director of the NIH on Jan. 17, told attendees of the American Association for the Advancement of Science Annual Meeting in Boston on Feb. 15.
Septerna pulls lead hypoparathyroidism drug on bilirubin levels
Septerna Inc.’s stock (NASDAQ:SEPN) plunged 64% in early trading Feb. 18 on news that the company was stopping a phase I trial of SEP-786 in healthy volunteers following two severe events of elevated unconjugated bilirubin in the multiple ascending-dose portion of the study. In both cases, there were no elevations in alanine transaminase (ALT), aspartate aminotransferase (AST) and gamma-glutamyl transpeptidase (GGT) liver enzyme levels, and the elevated bilirubin was reversible. The two events were grade 3, and, among all study participants, there was no liver injury, cholestasis, hemolysis or serious adverse events. Investors didn’t like the news, however, as the oral small-molecule parathyroid hormone 1 receptor (PTH1R) agonist was Septerna’s lead pipeline asset in development for the rare endocrine disease hypoparathyroidism. Shares of the company were trading at $4.64, down $8.31.
Arcus prices $150M stock offering to drive its cancer therapy
With Gilead Sciences Inc. stepping away from its exclusive option rights to casdatifan in treating cancer, Arcus Biosciences Inc. has decided to move on by pricing a $150 million common stock offering. Arcus said Gilead’s rights to the HIF-2a inhibitor had expired. Arcus said the financing, which has Gilead as an investor, is expected to fund the company through the PEAK-1 phase III study of casdatifan combined with cabozantinib vs. cabozantinib monotherapy as a first- or second-line treatment in those with metastatic clear cell renal cell carcinoma who have previously received anti-PD-1 therapy. The study’s primary endpoint will be progression free survival and overall survival as a key secondary endpoint. Arcus stock (NYSE:RCUS) was sagging at midday, with shares down 10% at $11.70 each.
Positive DMD phase I/II data push Solid stock higher
Shares of Solid Biosciences Inc. (NASDAQ:SLDB) were trading at $5.98, up $1.95, or 48%, on positive initial data from the phase I/II trial with next-generation gene therapy SGT-003 in Duchenne muscular dystrophy (DMD). Called Inspire Duchenne, the study generated interim 90-day biopsy data from the first three participants showed an average microdystrophin expression of 110%, as measured by Western blot. The new DMD data helped Solid to a public offering that raised about $200 million.
New chikungunya vaccine from Bavarian Nordic wins FDA nod
With the U.S. FDA’s approval of Vimkunya, Bavarian Nordic A/S is now in the chikungunya virus infection mix along with Valneva SE’s Ixchiq. A single-dose, adjuvanted virus-like particle-based vaccine to protect against the chikungunya virus infection, Vimkunya is the first of its kind available in the U.S. for those 12 and older. Bavarian Nordic said the vaccine, which has virus-like particles to mimic the virus without infecting cells, replicating or prompting the virus, should be available in the U.S. sometime in the first half of 2025.
GSK’s meningitis vaccine second pentavalent approved by the FDA
A second pentavalent vaccine for preventing meningococcal disease has been approved by the U.S. FDA. GSL plc’s Penmenvy will now go up against Pfizer Inc.’s Penbraya, which had a two-year head start into the market. GSK’s recombinant protein vaccine targets five groups of the bacteria Neisseria meningitidis (A, B, C, W and Y) to prevent meningococcal disease in those ages 10 through 25. The new vaccine combines two separate GSK vaccines, Bexsero (meningococcal group B) and Menveo (meningococcal group ACWY), into a single dose. The aim is to lower the number of injections and deliver the same level of protection, improving coverage and compliance with a vaccination schedule, according to Clarivate’s Drugs to Watch 2025.
Holiday notice
BioWorld's offices were closed in observance of Presidents Day in the U.S. No issue was published Monday, Feb. 17.
Also in the news
Allogene, Aptose, Arcturus, Bayer, Bristol Myers Squibb, Corbus, CSL, Entero, Evolus, Galderma, Hyloris, Immunitybio, Inflarx, Inhibikase, Loqus23, Matinas, Moderna, Moleculin, Nexus, Nuclera, Nura, Oculis, Olema, Oryzon, Otsuka, Pfizer, Sarepta, Theratechnologies, Zymeworks