September 12, 2024

The need-to-know this morning

Summit Therapeutics raised $235 million in a private placement, or PIPE, priced at $22.70 per share, the stock's closing price on Wednesday. Company insiders, including co-CEO Robert Duggan, bought $79 million of the offering, with the rest purchased by biopharma institutional investors.
Fulcrum Therapeutics said its experimental treatment for a degenerative muscle disease called FSHD failed to improve outcomes compared to a placebo in a Phase 3 study.

biotech

Moderna cuts R&D spending by $1.1 billion

Moderna said it would cut its annual R&D spending by 23%, or $1.1 billion, between 2024 and 2027 in a concession to investors who have viewed the company's outlays following its pandemic-era windfall as excessive.

The company also said that it plans to break even by 2028, when it expects it will reach annual sales of $6 billion. These announcements were made ahead of an R&D update the company is holding today.

Moderna also outlined a series of incremental wins in vaccines and some setbacks, as well.

Read more from STAT's Matt Herper and Jason Mast.

policy

A controversial rare disease program is ending

In 2012, the U.S. government set up an obscure voucher program aimed at spurring drug development for rare childhood diseases. But at the end of this month, that program is due to expire.

The vouchers allow holders to ask the FDA to review a drug in six months, as opposed to 10, and they can sold for many millions of dollars. Patient advocates say they're critical to incentivizing industry interest in rare diseases. "If the voucher goes away — I don't want to say it'll doom rare disease, but it'll be a significant hit," one advocate said.

But the program has also been criticized as as ineffective, convoluted, and a handout to pharma.

Read more from STAT's Jason Mast and John Wilkerson.

regulation

FDA commissioner: 'Sarepta's like a curse on me'

Robert Califf dismissed criticism that the FDA is lowering standards for new drug approvals at an event with journalists earlier this week. In particular, he was asked about the recent decision by a top official, Peter Marks, to expand Sarepta's accelerated approval for the Duchenne gene therapy Elevidys, despite the drug failing its Phase 3 trial.

This came after Califf dealt with a similar controversy over another Sarepta therapy when he was commissioner under in 2016. "Sarepta's like a curse on me," he said, arguing that it's not his place as a political appointee to interfere with decisions made by civil servants, unless there's "malfeasance or corruption or mental health difficulties by the center director."

regulation

FDA scolds AbbVie over ad featuring Serena Williams

Meanwhile, the FDA isn't afraid to scold drug companies after their products are already on the market.

The agency recently sent a warning letter to AbbVie about its TV ad for the migraine pill Ubrelvy. The ad suggests the drug providers a greater benefit than what's been actually demonstrated, and that its inclusion of celebrity athlete Serena Williams amplifies the misleading representations, the FDA letter said.

This incident should tell drugmakers that "representations of efficacy have to adhere really closely to the clinical trial experience and dramatizations have to be looked at critically for the potential be perceived as mischaracterizing efficacy," a consultant said.

oncology

Blood cell counts lead to unfair trial exclusions

To enroll in clinical trials, patients are often assessed on the amount of neutrophils, a type of white blood cell, in their blood. Certain cancer therapies like chemotherapy can be harsh on these cells, so patients need their neutrophil count to be in a given range when entering studies.

That can be a problem for people who have a natural blood variation called the Duffy-null phenotype, which leads them to have a naturally lower level of neutrophils in their blood, but doesn't affect their health or ability to fight infections.

This phenotype is most frequently seen in people with African and Middle Eastern ancestry, leading them to be unfairly excluded from cancer trials, thereby negatively affecting their treatment, a new study found.