May 5, 2026

The need-to-know this morning

Vertex Pharma reported first-quarter earnings that generally matched consensus, although the company said it was shelving an early stage, mRNA-based therapy for cystic fibrosis due to poor tolerability. The drug, called VX-522, was being developed as part of a collaboration with Moderna.
Pfizer and Alkermes also reported earnings.
Viridian Therapeutics announced positive top-line results from a Phase 3 study of its drug, called elegrobart, in patients with chronic thyroid eye disease.

biotech

Cytokinetics drug Myqorzo meets twin efficacy goals in study of genetic heart disease

Cytokinetics said its drug Myqorzo significantly improved heart failure symptoms and cardiovascular fitness in patients with non-obstructive hypertrophic cardiomyopathy, an inherited heart disorder.

The results, reported in a company press release, achieved the dual efficacy goals of a Phase 3 clinical trial, called ACACIA, with statistical significance.

Cytokinetics is in the early days of Myqorzo’s commercial launch as a treatment for the more severe “obstructive” form of hypertrophic cardiomyopathy, or HCM. The successful outcome of the ACACIA study, if also cleared by regulators, could greatly expand the number of HCM patients eligible for treatment — and boost the drug’s peak sales.

Cytokinetics' shares were trading higher in the pre-market session.

immunology

J&J will advance IBD therapy despite trial miss

In closely watched trials of Johnson & Johnson's combination therapy for inflammatory bowel disease, the combination performed better than the individual drugs, but did not meet statistical significance.

It’s a disappointing outcome after a 2022 study showed that the combination of Tremfya and Simponi led to a significant benefit for patients, spurring several companies to develop their own combination approaches for IBD.

But the company said it still plans to advance the combination into late-stage testing, focusing on one subgroup of patients.

Read more from STAT's Allison DeAngelis.

policy

French regulator fines Novo, Lilly for weight loss ads

France’s medicines agency has fined the two big GLP-1 drugmakers for their advertisements, reflecting increasing concern among regulators that weight loss drugs may be misused.

French laws prohibit companies from advertising medicines directly to the general public. The ads that the agency cited were not explicitly about Novo and Lilly's specific products, but touched on the risks of obesity and the benefits of losing weight, so the regulator saw them as indirect promotions of the GLP-1 drugs.

obesity

Are analysts too quick to gloss over Lilly's liver case?

Investors yesterday noticed that in the FDA's adverse event reporting system, there was a case of hepatic failure reported in a patient taking Lilly's newly launched GLP-1 pill Foundayo.

Even though Lilly said it hasn't seen any liver issues in its clinical trials, investors have wondered whether there's a risk of liver toxicity since Foundayo is a small molecule, and some investigational GLP-1 small molecules had shown liver side effects. (Novo's rival Wegovy pill doesn't carry the same concerns since it's an oral peptide.)

Analysts, however, were quick to dismiss concerns about the case. Wolfe Research analyst Alexandria Hammond stressed that the FDA database collects self-reported cases and it can't be used to assess a causal relationship with the drug. Lilly told her that it has assessed the case and “determined it was not reasonably related to Foundayo,” so she thinks the pre-market drop was “overdone.”

Evercore analyst Umer Raffat noted that liver failure has also been reported in patients taking other, older GLP-1 drugs, so “we cannot look at this single liver case in a silo… and such cases do tend to occur on other GLPs as well because of various confounding factors.”

By the end of the day yesterday, Lilly's stock was up slightly.

It's hard to read much into a single self-reported case, and cases might be expected given the large number of people already taking the drug. But it's also reasonable for investors and doctors to be on edge and look out for any further cases. Foundayo doesn't have as large of a safety database as other GLP-1 drugs on the market. Lilly is running a cardiovascular outcomes trial that aims to enroll over 7,000 participants, but it's not expected to end until 2031.

Also, Foundayo was approved through Commissioner Marty Makary's voucher program that expedites review for drugs deemed to meet national health priorities. There hasn't been transparency into how the drugs that received vouchers are assessed, and staffers have expressed concerns that this program has been a vehicle for political interference.

As former FDA Commissioner Scott Gottlieb noted at a STAT event earlier this year, when drugs that received vouchers are launched to a broad population, new safety issues may emerge that weren’t previously seen in trials, and the public will ask questions around how the approval decision was made and whether FDA staff made a rigorous enough review.

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