August 19, 2025

Viking oral obesity pill study results disappoint

From my colleague Allison DeAngelis: A new oral weight loss drug developed by Viking Therapeutics hit the mark in a mid-stage clinical trial, but the side effect and discontinuation rates have disappointed investors.

Viking reported this morning that its pill, dubbed VK2735, succeeded in a Phase 2 study. The medication led to statistically significant reductions in weight, as much as 12.2% after 13 weeks, on the highest dose of the medication. The company also reported the rate of weight loss didn’t plateau, setting the stage for further weight loss on a longer-term study.

The rate of gastrointestinal distress was substantial, though. Approximately 38% of the subjects taking the highest dose of VK2735 ultimately discontinued the medication early. Discontinuation rates across all other dose levels were also high.

Viking’s stock price dropped 30% in premarket trading, following the news.

drug pricing

Will direct-to-consumer offerings actually lower drug costs?

As President Trump demands pharma companies implement direct-to-consumer models as part of his plan to lower U.S. drug prices, we've heard executives from numerous pharma companies (Pfizer, Takeda, Roche, AstraZeneca, etc.) voice their support for the effort on earnings calls.

The reality, though, is that selling drugs directly to consumers is unlikely to make them much more affordable, if at all, drug pricing experts said. The cash prices that pharma companies are willing to offer for their drugs (several hundred dollars per month) will never be as low as the prices patients can get through insurance. And if patients buy their drugs directly from pharma companies, none of their spending contributes to their deductibles or out-of-pocket maximums meant to limit costs over time.

That’s not to say the model wouldn’t benefit the drug industry, particularly in the case of medicines not widely covered by insurance.

“I think the drug companies are trying to do things out of the optics of looking good, like they're trying to make progress on drug prices without actually being willing to tackle some of the fundamental challenges of pricing in the system,” one researcher said.

politics

Trump is expected to push for more aggressive Medicare negotiations

Some Wall Street analysts and Washington insiders say they think that the Centers for Medicare & Medicaid Services under Trump will press for bigger price cuts from pharma companies than the agency did under President Biden, as part of Medicare's authority to negotiate prices, which was granted under the Inflation Reduction Act.

Leerink analysts wrote that they think Trump could negotiate discounts in the 30% to 35% range, steeper than the 22% average discount seen in the first round of Medicare negotiations under Biden.

Their reasoning is that Trump will want to show it can deliver bigger savings than Biden.

regulation

FDA delays decision on Regenxbio gene therapy

Regenxbio said yesterday that the Food and Drug Administration would extend its review of its gene therapy for Hunter syndrome, delaying the deadline from Nov. 9 to Feb. 8.

This comes after the biotech submitted longer-term clinical data from its study of the therapy, called RGX-121, following a request from the FDA. Regenxbio said that these 12-month results are consistent with the biomarker and neurodevelopmental data data that it had previously submitted.

Hunter syndrome is a rare genetic disorder that primarily affects boys. Patients can't break down certain sugar molecules, which leads the molecules to build up in the body and cause physical and neruological complications.

This is the latest regulatory setback for gene therapy makers in recent months. Last month, the FDA declined to approve Ultragenyx's gene therapy for Sanfilippo syndrome type A due to manufacturing qualms.

politics

Study questions RFK Jr.'s view of federal vaccine panels

Health secretary Robert F. Kennedy Jr. has long criticized vaccine advisory committees for including members who work with the pharma industry. But it turns out that conflicts of interest on these panels have declined to “historically low levels” in recent years, according to a new study.

The rate of conflicts among members of the Centers for Disease Control and Prevention's vaccine advisory panel dropped from nearly 43% in 2000 to 5% last year, according to the study, published yesterday in JAMA. Conflicts on the FDA's Vaccines and Related Biological Products Advisory Committee fell from 11% to 0 during the same period.

The findings come after Kennedy recently fired all members of the CDC's panel and selected new members, vowing for a “clean sweep.”