May 2, 2023

The need-to-know this morning

Another week of earnings: Even with declining sales for its Covid-19 products, Pfizer beat expectations in the first quarter thanks in part to pneumococcal vaccines.
J&J is paying $245 million upfront to license two CAR-T therapies from Cellular Biomedicine Group, also known as CBMG. The two medicines, which target B-cell malignancies, are in early-stage trials.

middlemen

An expected delay to the big PBM reform

The Senate is pushing back its plan to reform PBMs by at least 10 months, STAT's Rachel Cohrs and John Wilkerson report. The legislation is meant to increase transparency for these drug middlemen, and mandate they pass 100% of the rebates they collect from drugmakers to the plans that employ them.

The reforms were originally supposed to take effect on Jan. 1, 2025. Now, however, it looks as though they won't take effect any sooner than November 2025. And, realistically, the date that this legislation would affect PBMs would likely be even later than that, since the bill would have to make it through the Senate, a Republican-led House, and be signed by the president to become law.

legal

Battle between U.S. and Gilead heads to trial

It's been four years since the federal government filed a lawsuit accusing the drugmaker Gilead of infringing patents on a pair of HIV prevention pills and unfairly reaping hundreds of millions of dollars from research funded by taxpayers. This week, the case is finally headed to trial, STAT's Ed Silverman reports.

At issue is the extent to which taxpayer-funded research may be used by a pharmaceutical company to reap enormous profits, and whether the prices charged for any resulting medicine are out of reach for many Americans.

The U.S. Department of Health and Human Services argues that Gilead refused to reach a licensing agreement despite "multiple attempts" at reaching a deal. The company, in turn, argues that the patents are not valid and disputed there was any infringement.

regulation

FDA vetoes hormone therapy from Ascendis

The FDA has declined to approve a hormone replacement therapy made by Ascendis Pharma, citing issues with the company's manufacturing control related to the dosing of the drug-device combo. The FDA didn't take issue with the clinical data for the hypoparathyroidism treatment, which is called TransCon PTH, and the company had expected an approval. But last month, regulators found "deficiencies" in Ascendis' filing that stopped discussions on labeling and post-marketing requirements, FiercePharma writes.

"We are committed to working collaboratively with the FDA and, because the agency did not suggest that additional Phase 3 studies may be needed to demonstrate the product's safety and efficacy, we believe we are well prepared to address their concerns," CEO Jan Mickelsen said in a statement.

rare disease

Travere's kidney drug fails in Phase 3

A Phase 3 trial testing an experimental treatment made by Travere Therapeutics for a rare and deadly kidney disease, has failed. The 371-person study among patients with focal segmental glomerulosclerosis found that while patients receiving the drug, called sparsentan, had higher kidney function than those receiving placebo, the difference was not statistically significant. Secondary endpoints, like urine protein levels, were lower among those taking the drug, which suggests that there was some activity, the company said.

Travere, lest we forget, used to be called Retrophin, and was run by Martin Shkreli. After Shkreli was imprisoned for defrauding investors and fired from Retrophin's board, the company rebranded.