May 20, 2025

The need-to-know this morning

Pfizer has acquired an experimental PD-1 -VEGF antibody for cancer from China's 3SBio, paying $1.25 billion upfront.

biotech

Tourmaline drug cuts inflammation, but investors aren't impressed

Tourmaline Bio said this morning that its experimental antibody treatment demonstrated deep reductions in a measure of inflammation often tied to heart disease, although a high placebo response in the mid-stage study may muddy interpretation of the outcome.

In the Phase 2 study of patients with chronic kidney disease, the highest dose of the drug lowered by 86% the levels of high-sensitivity C-reactive protein (hs-CRP), a measure of inflammation, through 90 days. Study participants treated with a placebo showed hs-CRP reductions of 15%.

When adjusted for placebo, Tourmaline's drug cut hs-CRP levels by less than a similar antibody being developed by Novo Nordisk.

Tourmaline's stock fell in pre-market trading today.

gene therapy

Prime Medicine to undergo major overhaul

As the gene editing field suffers a prolonged winter, Prime Medicine announced yesterday that it's replacing its CEO, laying off a quarter of its staff, and scrapping its lead program.

Prime was formed in 2019 around a CRISPR-based technology from biochemist David Liu that can, in theory, be used to correct about 90% of known disease-causing mutations. But former employees said the company stretched itself too thin trying to go after too many diseases.

The company is now scrapping its lead program in one subtype of chronic granulomatous disease, a rare immune deficiency, and is instead focusing on two more common liver conditions: Wilson's disease and alpha-1 antitrypsin deficiency.

gene therapy

CRISPR Therapeutics looks beyond gene editing

In another sign of the challenges gene-editing companies are facing — CRISPR Therapeutics, the company that co-developed the first approved treatment made with CRISPR gene editing, is now looking at other mechanisms.

It announced yesterday an agreement with Sirius Therapeutics to develop and commercialize small interfering RNA (siRNA) therapies. Under the deal, CRISPR will pay $25 million in cash and $70 million in equity to Sirius upfront.

The lead program is a therapy that targets Factor XI, a protein associated with thromboembolic disorders. CRISPR also has rights to license up to two additional siRNA programs.

CRISPR's decision to move beyond gene editing comes as its approved therapy for sickle cell disease, called Casgevy, has proven to be a tough sell, reaching relatively few patients since its approval in December 2023.

Covid

When the FDA requires a large vaccine trial, who pays?

From STAT's Matthew Herper: Novavax finally received full FDA approval for its Covid-19 vaccine over the weekend, but it was limited to people 65 and older or those who have a risk factor for bad outcomes if they get severe Covid. (Some 75% of the overall population have such risk factors, but the limitation will still hurt sales.) The FDA is also requiring a new clinical trial.

That study, the agency said in its approval letter to Novavax, will be in adults aged 50 to 65 without high-risk conditions for severe Covid, to evaluate the clinical efficacy and safety of the vaccine. While details are scant, the required study, as proposed, might require tens of thousands of participants. Today, people have more immunity to Covid, either from having caught the virus before or from previous vaccination. This makes it tougher to show a vaccine is effective, because people in the placebo group will be less likely to get sick.

So who pays for this? Novavax agreed to co-commercialize its vaccine with Sanofi last May. In a filing with the Securities and Exchange Commission, Novavax indicated that it is not settled yet which company will take the lead and which will reimburse the other. The differences don't appear financially material, but show how quickly the plans for the new trial were put in place.

politics

Trump's science adviser laments 'scientific slowdown'

Michael Kratsios, the new head of the Office of Science and Technology Policy, called for a return to reproducible and transparent research to kickstart what he characterized as a "scientific slowdown."

In his first detailed public remarks since taking on his role, Kratsios said there's been an increase in federal funding for biomedical research, but it hasn't led to an increase in new drug approvals.

"Spending more money on the wrong things is far worse than spending less money on the right things," he said during a speech at the National Academy of Sciences.

His remarks come as the federal government freezes funding to some universities, terminates grants that it deems in conflict with its political agenda, and lays off thousands of employees across scientific agencies.