January 30, 2025

The need-to-know this morning

Read all about Roche Q4 and 2024 earnings here, and Sanofi earnings here.
Tectonic Therapeutics shares rose sharply in pre-market trading after the company announced positive results from an early stage study of a drug to treat a form of hypertension caused by heart failure. Tectonic was co-founded by billionaire Harvard scientist and veteran biotech entrepreneur Tim Springer.
Takeda CEO Christophe Weber will step down next year, and will be replaced by Julie Kim, currently president of Takeda’s U.S. business unit.

politics

RFK Jr. takes tough stance on pharma in first hearing

Let's take a look at the major moments from Robert F. Kennedy Jr.'s confirmation hearing before the Senate Finance Committee yesterday. (Note that the committee will vote soon on whether to advance his nomination to the full Senate, but the vote has not yet been scheduled.)

RFK Jr. came out tough on the drug industry, despite recently dining (along with President Trump) with pharma executives. He indicated that the Trump administration would issue an executive order in support of the Medicare drug price negotiation program authorized by the Inflation Reduction Act. That order has not been made public.

“We’ve already negotiated the first 10 drugs, and we want to expand it to the next 10 to 15 that the Biden administration has put forward,” he said.

He also said “we should end that disparity” between drug prices in Europe and in the U.S.

Read more on the other big topics covered in yesterday's hearing.

Today, RFK Jr. will sit through another hearing held by the Senate health committee. My D.C. colleagues will continue to live blog — follow along here.

politics

Trump rescinds memo on federal funding freeze

Earlier this week, the White House budget issued a memo ordering agencies to freeze financial assistance programs and assess which ones run afoul of executive orders issued by President Trump (such as ones that roll back DEI and climate change initiatives). This created widespread confusion and uncertainty about funding for programs like Medicaid, though the White House later clarified that Medicaid payments should not be affected.

The budget office has now rescinded that memo, but said that Trump’s underlying executive orders remained in place.

biotech

Cargo to lay off staff after CAR-T data show safety concerns

Cargo Therapeutics said yesterday it will stop developing a closely watched experimental CAR-T treatment and lay off 50% of its staff after early data showed little long-term efficacy and significant safety concerns, including at least one patient death.

Founded by two leading cell therapy researchers and a pediatric cancer advocate, Cargo raised over $500 million to develop a new fleet of cell therapies, particularly for patients who have relapsed after receiving one of the CAR-T treatments already on the market. These treatments can be curative for certain blood cancer patients, but those whose cancer progresses or returns have few remaining options. Early academic data had been promising.

Yet those did not hold up in a large Phase 2 trial. The study, Cargo said, was stopped early after 18% of patients experienced a severe inflammatory syndrome called IEC-HS. Cargo indicated that included at least one patient death but did not say how many. Meanwhile, early effectiveness data was lackluster: Only 18% of patients remained in complete remission after 3 months. CEO Gina Chapman said Cargo will keep advancing another CAR-T treatment while the company "evaluate[s] our strategic options"

biotech

A big decision for Vertex is expected today

Today is the deadline for the FDA to issue an approval decision on Vertex's non-opioid pain drug, called suzetrigine, for the treatment of acute pain.

While the agency is widely expected to approve the medicine, it's unclear how widely prescribed the drug may end up being. Market analysts generally think the treatment has blockbuster potential in acute pain, but some experts see the drug as only modestly effective. (It didn’t outperform a weak opioid in late-stage trials.)

Still, an approval would be a key milestone in Vertex's ambitions to transform the treatment of pain, similar to the way in which it has revolutionized cystic fibrosis care.

Vertex has hit some key hurdles when studying this drug for chronic pain, a bigger and broader market than acute pain. In a mid-stage trial, sciatica patients on suzetrigine didn’t fare better than those on placebo, though patients on the medicine had less pain by the end of the study than they had at the beginning. Vertex is still planning to run a Phase 3 trial in this indication, hoping it can design its study to reduce the placebo effect.

artificial intelligence

Paris biotech begins trial of 'AI-optimized' candidate

Owkin, a Paris-based biotech, said today that the first patient has been dosed in a Phase 1 study of its “AI-optimized” drug candidate for solid tumors.

What does “AI-optimization” mean? In this case, Owkin is using AI to synthesize information from the literature plus its own proprietary data to suggest potential tumor types that might be good fits for the drug. The company is also looking at ways to create synthetic control arms during Phase 1 studies to better estimate the drug's effect and correctly size Phase 2 studies.

My colleague Brittany Trang sat down with Andrew Pierce, Owkin’s senior vice president of discovery and development, to discuss the company’s use of AI and how it may fill in blind spots in clinical trial planning.