June 12, 2025

The need-to-know this morning

BioNTech, the German maker of mRNA-based vaccines and drugs, is acquiring fellow German drug developer CureVac in an all-stock deal valued at $1.25 billion.

vaccines

RFK Jr. installs vaccine critics on CDC panel

Health secretary Robert F. Kennedy Jr. yesterday appointed a new, smaller vaccine advisory committee at the CDC — just two days after firing every member of the panel, the Advisory Committee on Immunization Practices, and calling for a "clean slate."

The new, eight-member panel includes several high-profile vaccine critics, including Robert Malone, who has both claimed he was one of the inventors of messenger RNA and denounced the technology. Also on the roster is former Harvard epidemiologist Martin Kulldorff, who co-authored the Great Barrington Declaration, which opposed vaccine mandates.

"The slate includes highly credentialed scientists, leading public-health experts, and some of America's most accomplished physicians," Kennedy wrote in a post on X.

Read more on the announcement here. And for a deeper dive on each new member of ACIP, read this.

drug pricing

Cassidy explores 'most favored nation' legislation

Sen. Bill Cassidy is quietly exploring legislation that would link U.S. Medicare drug prices to lower prices in other wealthy countries, STAT's John Wilkerson reports. The effort signals the ongoing shift in Republican attitudes toward the most-favored national policy to lower drug prices, now that President Trump backs the approach.

STAT viewed a draft of legislation that Cassidy has floated to industry lobbyists, which includes language saying Medicare should take back what it is owed when drugmakers charge prices higher stateside than they do in other wealthy countries.

Read more.

Meanwhile, yesterday was the day drugmakers were expecting to hear more details about the Trump administration's own policy on this front. It didn't happen, as STAT's Daniel Payne and Elaine Chen note, possibly complicating the industry's approach to further talks with the White House.

Wall STREET

Are Regeneron's troubles in the rearview mirror?

Regeneron had a rough several months — reeling from a series of commercial, clinical, and regulatory setbacks. Its financial performance to suffer and its stock price to plummet by nearly half.

Now, is that all a thing of the past?

In the next few months, Regeneron is expecting FDA decisions on the approval of a pre-filled Eylea syringe, monthly dosing, and an expanded label that should stabilize sales. If the company can stop the financial hemorrhaging from Eylea, its eye drug, 2025 could be the Regeneron's trough for revenue and earnings, with growth returning, driven by the Dupixent franchise (the company's other blockbuster product) plus oncology drug sales, at least one analyst says.

cancer

Nuvation drug approved in non-small cell lung cancer

The FDA has approved taletrectinib, a Ros1-targeting tyrosine kinase inhibitor developed by Nuvation Bio to treat advanced or metastastic non-small cell lung cancer in patients who carry that oncogene. That said, there are several drugs on the market already that target Ros1 — including Pfizer's Xalkori, which was approved for non-small cell lung cancer nearly a decade ago.

The approval is based on data from two small Phase 2 studies, and can be used even in patients whose cancer has spread to the brain — which is perhaps what sets this approval apart from the many other approved Ros1 drugs.

"I think the unique characteristics of taletrectinib is [the] lower rate of neurological toxicity that might made it make it attractive for patients with newly diagnosed Ros1-mutant non–small cell lung cancer," University of California, San Diego, oncologist Lyudmila Bazhenova told Targeted Oncology. "The reported incidence [of brain metastases] is about 20 to 30%, so it's relatively common to see brain mets in the [Ros1-mutant] population."

gene therapy

FDA clears Capsida gene therapy for clinical trials

The FDA has cleared Capsida Biotherapeutics to test its gene therapy for Parkinson's disease linked to GBA mutations — a genetic subtype affecting up to 15% of patients with the disease. The therapy was able to cross the blood-brain barrier in primate studies, and is designed to boost Gcase enzyme activity in key brain regions of people with this form of Parkinson's. Gcase is the protein encoded by the GBA gene, and is deficient in patients with the disease.

Capsida launched in 2021 with a $50 million Series A and $90 million from AbbVie to develop gene therapies for CNS indications. The company has forged further deals with AbbVie and also Eli Lilly, though in 2023 it did lay off about 25% of its staff.

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