June 10, 2025

The need-to-know this morning

AI drug developer Recursion Pharmaceuticals said it was laying off 20% of its employees to reduce spending and extend its existing cash runway into the fourth quarter of 2027. Last month, Recursion shelved three drug prospects from its research pipeline.
Insmed said its once-daily, inhaled formulation of trepostinil achieved the primary goal of a Phase 2 study in patients with pulmonary arterial hypertension. The company expects to begin Phase 3 studies before the end of the year. Currently approved inhaled trepostinil treatments require more frequent dosing.

politics

RFK Jr. abruptly fires vaccine advisory panel

Health secretary RFK Jr. has taken the extraordinary step of firing the CDC's vaccine advisory panel, saying that "a clean sweep" is needed to restore faith in vaccines.

Proponents of vaccines have feared that Kennedy, a longtime immunization critic, might take such a step, but were still shocked. "We have just demonstrated that politics will overrun science in this administration. It scares me to think of what's ahead," one epidemiologist said.

The Advisory Committee on Immunization Practices, or ACIP, studies vaccines in the regulatory pipeline and ones that have recently been licensed by the FDA.

New members haven't yet been named, but it appears they may have been selected, because a statement from Kennedy's department said that a meeting scheduled for late June will take place. In normal times, ACIP candidates go through a vetting process that can take upwards of a year, my colleagues report.

infectious disease

Merck's RSV antibody gets FDA clearance

The FDA yesterday approved Merck's monoclonal antibody to protect against RSV, the third such drug to go onto the market.

The antibody, called Enflonsia is approved only for babies in the first year they face the virus. Merck is running a study to try to also get it approved for high-risk children in their second RSV season.

ACIP was expected to vote at its meeting in late June on whether to recommend use of Enflonsia, but the news that RFK Jr. fired the entire panel (as discussed above) raises questions about how the June meeting will function.

biotech

Bob Nelsen's manufacturing brainchild to wind down facilities

National Resilience, a startup that promised to transform drug manufacturing, said yesterday that it would "wind down" many of its facilities.

Through a bit of financial engineering, an affiliate of the company that holds leases for some of the company's manufacturing facilities is filing for bankruptcy, but what remains of the firm will seek to raise an additional $250 million from existing investors and continue to build its business.

National Resilience was the brainchild of ARCH Venture Partners co-founder Bob Nelson. The company has raised $2 billion, with the goal of developing new manufacturing technologies that could bring down the cost of scaling gene therapy viruses and other new medicines.

But the company has been on a turbulent journey, seeing frequent turnover at its highest levels. The development reflects the prolonged downturn in the biotech industry since 2020, when companies were awash in cash and a wave of mRNA vaccines and gene therapies seemed to be on the horizon.

regulation

Avidity's agreement with FDA bodes well for rare disease drugs

Avidity Biosciences reached an agreement with the FDA yesterday to seek accelerated approval of its treatment for an inherited muscle-weakening disease, a development that should further assuage concerns from the biotech industry about the agency's stance on rare disease therapies.

The company is developing its drug, called del-brax, to treat facioscapulohumeral muscular dystrophy, or FSHD, a genetic disease that causes muscles in the upper and lower body to weaken and lose function over time.

Avidity sought accelerated approval based on a new biomarker called cDUX that's thought to be associated with improved muscle-function outcomes. The fact that it's a new biomarker, coupled with the recent instability at the FDA with staffing cuts and new leadership, led to concerns that Avidity would not be able to go the accelerated approval route.