A biotech scorecard for the first quarter

Each quarter, STAT's Adam Feuerstein outlines some of the most noteworthy biotech events likely to move stocks. This quarter, there's plenty to watch out for, including an interim Phase 3 analysis from Ultragenyx on its osteogenesis imperfecta antibody drug setrusumab, and another from Praxis Precision Medicines on its experimental drug ulixacaltamide, which it's testing for essential tremor.

Comapnies such as Incyte, Protagonist Therapeutics, and Solid Biosciences are also nearing critical trial results and Kura Oncology may soon be ready for a marketing application. Several PDUFA dates are coming up as well. The FDA will make a decision about drugs including suzetrigine, made by Vertex Pharmaceuticals, which is a non-opioid approach to acute post-surgical pain.

REGULATION

Trump's FDA transition plan takes shape

The Trump administration is forming its FDA transition plan, STAT's Rachel Cohrs Zhang reports. Figures including Lowell Zeta, a former senior counselor to ex-FDA Commissioner Stephen Zahn, and Jim Traficant, CEO of communications firm Pinkston Strategy, are assisting with outreach and recruitment efforts.

The team is working with Grace Graham, a prominent Capitol Hill health policy expert, about potentially taking on a significant role at the FDA. Trump has said he will nominate Marty Makary, a surgeon at Johns Hopkins, to be his FDA commissioner.

glp-1 drugs

Eli Lilly seeks to join lawsuit on compounded drugs

From STAT's Elaine Chen: For several months now, a compounding trade group has been engaged in litigation against the FDA over the agency's declaration that Eli Lilly's tirzepatide (aka Mounjaro/Zepbound) is no longer in shortage. Lilly now wants to join the lawsuit as a defendant, to "protect its interests and help bring this suit to a swift end," according to a legal filing last week.

For background, the FDA in October pulled tirzepatide of its shortage list after almost two years. Compounders are allowed to make copies of branded drugs only when the treatments are on the list. The trade group, called the Outsourcing Facilities Association, sued the FDA, arguing that the shortage wasn't actually over. The FDA took some time to reconsider its decision and last month re-affirmed that tirzepatide should indeed be off the shortage list.

Lilly, in its legal filing, argues that the position of the compounding group "directly affects Lilly's interests in preserving its exclusive right to sell its FDA-approved medicines." The FDA, as a government agency "will not 'adequately represent [Lilly's] interest[s],'" the filing said.

This is the latest move in Lilly's long-standing campaign against compounders. Its executives have said that compounded products aren't safe and that the growth of compounding could create a "backdoor generic world."

honors

Biden grants highest U.S. award in science, tech

President Biden has recognized a number of individuals for their contributions to science and technology with the country's highest award. CRISPR scientists including Jennifer Doudna and Feng Zhang won the National Medal of Technology and Innovation, as did the companies Moderna and Pfizer, each of which developed mRNA vaccines of critical importance during the Covid-19 pandemic.

Among the National Medal of Science recipients were MIT materials scientist Angela Belcher, who has used genetically modified viruses to build batteries and cancer therapies, and stem cell biologist Helen Blau of Stanford, who works on tissue regeneration technology.

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