Ensoma Inc.’s lead program, EN-374, has been granted orphan drug and rare pediatric disease designations by the FDA for the treatment of X-linked chronic granulomatous disease. Ensoma anticipates filing an IND application for EN-374 in the first half of this year.
A 15% cap on indirect cost reimbursement that was announced by the U.S. NIH has been stalled by a court order for the time being. But researchers remain deeply concerned about the attempt, and about the new administration’s adversarial approach to research and universities.
The market took a strong liking to the latest U.S. FDA guidance for Moleculin Biotech Inc.’s upcoming Miracle phase III trial testing annamycin in cancer. So much so that the company’s stock (NASDAQ:MBRX) soared 202% on Feb. 13 to close at $1.27 a share.
Coming as no surprise, the U.S. Senate’s Feb. 13 confirmation of Robert Kennedy as the next secretary of the Department of Health and Human Services (HHS) did nothing to ease the uncertainty hanging over the FDA and other HHS agencies.
The U.K. Medicines and Health Care Products Regulatory Agency reported a pilot program for the use of RWE in regulatory decision making, which is largely directed toward drug products.
The European Commission’s proposal for an AI-specific liability law seemed destined to pile onto existing EU liability law, but the commission reported it will pull the legislative proposal dubbed the AI Liability Directive.
Snagging its second U.S. FDA approval since being spun out of Pfizer Inc. in 2017, Springworks Therapeutics Inc. is aiming to position Gomekli (mirdametinib), cleared for neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) not amenable to complete resection, as a first-in-class drug for adults and potentially best-in-class option for children. The priority approval, which came after market close Feb. 11, more than two weeks ahead of the Feb. 28 PDUFA date, marks the second MEK1/2 inhibitor to hit the market for NF1-PN, a rare, genetic condition.
The Biosecure Act may have died with the 118th U.S. Congress, but efforts to stop U.S. government funding of R&D in China are alive and well. Rep. Claudia Tenney, R-N.Y., introduced the Stop Funding our Adversaries Act in the House Feb. 7 to prohibit direct and indirect federal funding of research in China or entities owned by China.
The European Commission on Feb. 5 cleared Shanghai Henlius Biotech Inc.’s serplulimab (HLX-02) under the brand name of Hetronifly as a first-line combination therapy with carboplatin and etoposide to treat extensive-stage small-cell lung cancer.
“This current administration is like nothing that we've seen before,” said a managing partner of a global venture capital firm who spoke to BioWorld on the condition of anonymity. “President Trump’s first term was bad,” he said, “but nobody knows what’s coming.” “This is truly nationalism at its worst, because he won on the campaign [largely] to protect American jobs, claiming that Americans have been unfairly treated.” And it's not just China, he said, but India and other countries will also likely be affected.
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