The appointment May 6 of Vinay Prasad as the head of the U.S. FDA’s Center for Biologics Evaluations and Research sent ripples across the industry. Shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) ended the day down 26.6% vs. an XBI drop of 6.6% at the close – this ahead of the after-hours earnings disclosure that pushed Cambridge, Mass.-based Sarepta’s stock down even further by more than 20%, with the XBI unchanged. Prasad has been especially disapproving of the FDA’s approval of Elevidys (delandistrogene moxeparvovec), an AAV-based gene therapy for Duchenne muscular dystrophy. Other biopharmas had cause for worry as well.

South Korea urges US to exempt pharma, allies from tariffs

South Korean government and biopharmaceutical industry representatives urged American policymakers May 7 to refrain from imposing tariffs on pharmaceutical imports, and to spare allies if pharma tariffs are deemed necessary. Both Korea’s Ministry of Health and Welfare (MOHW) on May 4 and the Korea Biotechnology Industry Organization on May 6 submitted comments to the U.S. Department of Commerce in response to its ongoing investigation of pharmaceutical imports. “Despite the difference in the size of the pharmaceutical industry and medical market between Korea and the U.S.,” the MOHW wrote May 6, “the U.S. is a major importer of [Korean] pharmaceutical goods, demonstrating the close economic and health cooperation between the two countries.”

Theriva’s VCN-01 improves survival in phase IIb pancreatic cancer trial

Theriva Biologics Inc. plans to engage with regulators on the design of a phase III trial for oncolytic adenovirus candidate VCN-01 (zabilugene almadenorepvec) on the back of positive top-line results from a phase IIb trial in metastatic pancreatic ductal adenocarcinoma. The Virage trial, which enrolled newly diagnosed patients for whom standard-of-care (SOC) gemcitabine/nab-paclitaxel is the recommended first-line option, showed VCN-01 plus SOC bested SOC alone on endpoints of overall survival, progression-free survival and duration of response. Shares of Theriva (NYSE:TOVX) were trading down 42% at midday, but the stock reaction was likely in response to the firm’s concurrent pricing of a $7.5 million public offering.

US gain-of-function EO broader, yet narrower, than feared

Rather than banning federal funding of all gain-of-function research as some scientists feared, U.S. President Donald Trump signed an executive order (EO) Monday limiting the prohibition to gain-of-function research in countries of concern, including China and Iran, and in foreign nations deemed to have insufficient research oversight. However, while the EO allows funding of such research in the U.S. and in several other countries, it creates more oversight – even of domestic gain-of-function research that’s not funded by the government and of what it calls “other life-science research that could cause significant societal consequences” that’s privately supported.

Marea looks to a phase IIb study in cardiovascular disease

With positive phase IIa cardiovascular disease results in hand, Marea Therapeutics Inc. said it is ready to move on to a phase IIb study sometime in the second quarter of 2025. MAR-001, a monoclonal antibody targeting the protein coding gene ANGPTL4, produced an up to 52.5% placebo-adjusted mean reduction in remnant cholesterol and up to a 52.7% placebo-adjusted mean reduction in triglycerides at 12 weeks. Results showed a roughly 65% placebo-adjusted mean reduction in remnant cholesterol and triglycerides in those patients who had greater triglyceride elevation at baseline. Marea presented the data at the European Atherosclerosis Society Congress in Glasgow, U.K.

Congress poised to revisit two legislative proposals for patents

The U.S. Senate and House of Representatives have another legislative cycle to consider two bills that would impose significant changes on patent prosecution, including the Patent Eligibility Restoration Act (PERA) of 2025. PERA is largely identical to the version that was previously considered, but while the latest version waters down the previous edition’s language regarding patent eligibility for DNA, it would nonetheless overwrite a couple of instances of Supreme Court jurisprudence on subject matter eligibility, which by itself would be a big win for companies in the life sciences.

Also in the news

Actuate, Akamis, Alchemab, Aldeyra, Armatus, Ascidian, Astrazeneca, Bavarian Nordic, Biomea Fusion, Cantargia, Celldex, Eli Lilly, Essential, Ferring, Firstgene, Grin, Hovione, Ideaya, Italfarmaco, Kane, Khondrion, Lipocine, Maia, Mersana, Opus, OSE, Ousia, Redx, Silence, Sitryx, Solve FSHD, Tharimmune, Thetis, Tiziana, Topas, Viatris, Xlife