September 4, 2025

The need-to-know this morning

Agios Pharmaceuticals said the FDA extended the review of its drug Pyrukynd by three months in order to fully vet a drug-safety plan to mitigate the risk of liver toxicity. The company is seeking to expand Pyrukynd's approval to cover the treatment of the blood disorder thalassemia. The new approval decision date is Dec. 7.

CHRONIC DISEASE

Sanofi shares drop after disappointing trial results

From my colleague Andrew Joseph: Sanofi this morning reported underwhelming results from a closely watched trial in the skin condition atopic dermatitis, part of the French firm’s major push into immunology.

The drug being tested, called amlitelimab, met all primary and key secondary endpoints in the Phase 3 COAST 1 trial, including lowering disease severity and clearing patients’ skin versus placebo, Sanofi said. But the results may not stack up against those from other medicines, including Dupixent, the mega-blockbuster approved for a number of inflammatory diseases that Sanofi sells with Regeneron Pharmaceuticals.

Sanofi shares slid in early trading.

covid-19

Pfizer CEO: Trump should get the Nobel Peace Prize

Pfizer CEO Albert Bourla is defending his company's Covid vaccines and Operation Warp Speed, arguing the effort saved more than 14 million lives. He also suggests President Trump may deserve that Nobel Peace Prize he has openly coveted.

Bourla pushed back on Trump’s claim of the other day that drugmakers are withholding data, STAT’s Matthew Herper writes, noting Pfizer’s shot has been studied in more than 600 peer-reviewed papers, scrutinized by regulators in 130 countries, and has been validated abroad for both effectiveness and rare risks like myocarditis.

Moderna and Novovax issued their own proud statements about Operation Warm Speed without hours later. The statements follow Trump’s demand on Truth Social that vaccine makers “justify” their products amid turmoil at the CDC, now overseen by HHS Secretary Robert F. Kennedy Jr., who recently forced out the CDC director and is reshaping federal vaccine policy.

Exclusive

Atlas raises $400 million to double down on biotech

Atlas Venture has raised a $400 million opportunity fund — its third — to deepen bets on companies already in its portfolio, STAT’s Allison DeAngelis writes.

So-called opportunity funds, once ubiquitous, have dwindled industry-wide as VCs face skittish investors, but Atlas joins OrbiMed and Lightspeed in reviving the model.

Atlas partner Kevin Bitterman said the approach balances small, high-return seed funds with the realities of pricey biotech development, adding that recent market “green shoots” suggest recovery — and that he’s “optimistic that the IPO market will come back and hopefully come back strong.”

startups

Treeline expands Series A another $200 million

Treeline Biosciences has extended its Series A by $200 million, bringing total funding to $1.1 billion. The company is announcing its first clinical trials debuting its pipeline: a BCL6 degrader, EZH2 inhibitor, and pan-KRAS inhibitor, all now in Phase 1.

Founded in 2021 by Loxo Oncology veteran Josh Bilenker and former Novartis oncology chief Jeff Engelman, the stealthy biotech is targeting 2026 for early readouts while lining up additional INDs. Treeline is betting on hard-to-drug cancer targets and a flexible strategy. Bilenker said in a release that executives “aspire to create the next great enduring biopharma company.”

government

Report urges streamlined research rules amid skepticism

A new National Academies report offers 53 proposals to cut red tape in U.S. research, spotlighting rare alignment between Trump administration rhetoric and academia’s push to reduce scientists’ administrative burdens. The ideas include creating a Federal Research Policy Board or a White House role to streamline cross-agency rules, easing requirements for lower-risk studies, and leaning heavily on technology, STAT's Jonathan Wosen and Anil Oza report.

While some find the report an important diagnostic tool for research redundances, other experts said the solutions are recycled and light on implementation detail — and past attempts to enact similar changes have fizzled.

Regarding centralizing and standardizing regulations, one science policy expert said this: “At some parts of my career, I would think that’s a pretty good idea. Right now, I think it’s a really awful idea....I have no confidence that that would be carried out in a way that would benefit research in the current administration.”

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