January 29, 2024

The need-to-know this morning

Sarepta Therapeutics announced results from a mid-stage clinical trial of its next-generation treatment for patients with Duchenne muscular dystrophy caused by mutations in a gene called exon 51.
Pfizer kicks off a busy week of pharma earnings on Tuesday. Novo Nordisk, Novartis, and GSK report on Wednesday; Merck, Sanofi, and Roche on Thursday; and Abbvie, Bristol Myers Squibb, and Regeneron Pharma on Friday.

gene therapy

How will Medicaid pay for sickle cell gene therapies?

The FDA recently approved two curative gene therapies for sickle cell disease, but the costs of these drugs — $2.2 million and $3.1 million — are creating a strain for government health coverage. About 100,000 Americans have the disease, and between 30% and 40% of these people are believed to rely on Medicaid for care. While not all of them would be eligible for the new wave of sickle cell gene therapies or even want to take them, the cost would still be staggering.

"The magnitude of the cost is really dwarfing anything we've seen before," the executive director of the National Association of Medicaid Directors told STAT's Ed Silverman. "The key argument is whether the investment in curative therapies would offset future costs. But it will be extraordinarily difficult for the state programs to solve this on their own."

The Centers for Medicare & Medicaid Services is developing a pilot program to help create outcomes-based agreements between state Medicaid programs and cell and gene therapy manufacturers.

data privacy

23andMe breach targeted Chinese, Jewish populations

A new class action lawsuit accuses 23andMe of failing to protect the privacy of customers whose data were exposed during a breach last year that affected nearly 7 million profiles. It notes that customers with Chinese and Ashkenazi Jewish profiles seem to have been targeted — and their personal genetic information seems to have been siphoned into "specially curated lists" that were shared and sold on the dark web, the New York Times writes.

"Now when we look at data breaches, our first concern will be whether the information will be used to physically harass or harm people on a systematic, mass scale," an attorney for the plaintiffs said. "The standard for when a company acts reasonably to protect data is now a higher one, at least for the type of data that can be used in this manner."

On Oct. 1, a hacker who called himself "Golem" leaked the personal data of more than 1 million 23andMe users with Jewish ancestry on an online forum used by cybercriminals. The data included the users' full names, home addresses, and birth dates. "Golem" also linked to profiles of 100,000 Chinese customers.

drug pricing

Merck and J&J chiefs agree to testify to Congress

The CEOs of Merck and Johnson & Johnson have decided to testify before the Senate health committee after all — avoiding a subpoena threatened recently by Bernie Sanders. They will testify before Congress about drug pricing on Feb. 8. The companies recently chose to sue the Biden administration over the recent Medicare drug price negotiation program, and had balked on attending a congressional hearing because of this conflict. They offered up executives other than the CEOs to testify — but Sanders was unmoved.

"The use of a subpoena was clearly a last resort and I'm delighted that these CEOs will be coming into our committee voluntarily," Sanders wrote in a statement. The CEO of Starbucks also agreed to testify last year only when treated by Sanders with a subpoena.

Pd-1 inhibitors

Keytruda has a kidney cancer win; Opdivo does not

New data show that Keytruda can extend patient lives when used as a post-surgical treatment for resectable kidney cancer: It cut the risk of death by 38% compared with placebo, FiercePharma writes. After four years of treatment, 91.2% of patients in the Keytruda arm were still alive, versus 86% for the control group. The Merck PD-1 inhibitor was approved in 2021 as an adjuvant therapy kidney cancer patients at risk of relapse.

Interestingly, this survival data comes right on the heels of Bristol Myers Squibb sharing that its own PD-1 inhibitor, Opdivo, failed for a second time as an adjuvant treatment for kidney cancer. Opdivo reduced the risk of recurrence or death by 13%, which wasn't considered statistically significant. And at 18 months, 78.4% of people taking Opdivo were estimated to be alive without their disease worsening, compared with 75.4% of those taking placebo.

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