September 16, 2025

The need-to-know this morning

Areteia Therapeutics said its experimental drug, a twice-daily pill called dexpramipexole, improved lung function in patients with a type of asthma driven by an increase of white blood cells. The outcome achieved the primary goal of a placebo-controlled Phase 3 study. Privately held Areteia is conducting two more Phase 3 studies of dexpramipexole in asthma.

exclusive

Hill staffers mull Trump's drug pricing plan

Congressional staffers and health policy experts from major think tanks recently held a closed-door meeting to discuss policy options related to President Trump's plan to peg U.S. drug prices to what other countries pay, or "most-favored nation" pricing.

STAT previously reported that Sen. Bill Cassidy (R-La.) has circulated proposed legislation for an MFN policy. The latest meeting is another example of growing interest among Republicans to pursue aggressive measures to lower drug prices.

However, congressional staffers were “pretty tight-lipped about what might be going on on the hill,” one of the think tank members told STAT. They noted that “pharma holds a lot of sway in Congress over these issues.”

politics

RFK Jr. adds ACIP members ahead of key meeting

Health secretary Robert F. Kennedy Jr. has added five new members to the CDC's vaccine panel, the Advisory Committee on Immunization Practices, just days before the group is set to meet and vote on recommendations related to vaccines for Covid-19 and hepatitis B.

STAT previously reported that Kennedy was considering these new members. This is the second batch of members he's appointed to the panel. He chose the first batch in June, after he suddenly fired all previous members of the committee.

The new members include a pediatric cardiologist who has called for mRNA shots to be pulled off the market and an infectious disease expert who opposed Covid-19 mandates.

Read more from STAT's Chelsea Cirruzzo and my other colleagues.

Stay tuned for the meeting of the advisory committee this Thursday and Friday. Also, on Wednesday, Susan Monarez, who was recently ousted from her role as CDC director, will be testifying in front of the Senate health committee about her firing.

politics

Scientists question how much gene Sarepta’s therapy actually delivers

Some scientists are questioning a calculation that was a key part of Sarepta Therapeutics’ ability to win FDA approval for its gene therapy for Duchenne muscular dystrophy.

In its studies, Sarepta took muscle biopsies from patients and measured how much microdystrophin — a mini version of the gene broken in Duchenne — is present. Sarepta has said that treated patients produce roughly one-third of the amount of microdystrophin as healthy humans have of the full-length protein.

But that number may be misleading, a group of researchers wrote last month in a letter to the editor in Neurology. If so, then families of patients may have a warped view of how much benefit the therapy can confer.

financing

New startup wants to make novel bispecific antibodies for immune diseases

From my colleague Jonathan Wosen: Bispecific antibodies have generated intense interest among cancer researchers and investors, with observers hopeful these Y-shaped immune proteins, which latch onto two different targets at once, could be potent and precise treatments. A new biotech believes this same therapeutic class could transform the treatment of immune-mediated diseases.

Dualitas Therapeutics, based in South San Francisco, launched today and announced that it raised $65 million in a Series A round. While some companies are repurposing bispecifics originally developed for cancer, Dualitas’ core technology revolves around screening for promising candidates that target novel target pairs. “There are no ‘me too’ combinations in what we're finding. These are truly different, novel biologies you wouldn't find in other portfolios out there,” co-founder Forbes Huang, COO and CBO, said in an interview.

The firm has two lead preclinical assets, one for rheumatoid arthritis and another for allergic asthma. (It has yet to disclose the drugs’ targets). It plans to advance one of these therapies on its own and identify a development partner for the other.

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