December 20, 2024

The need-to-know this morning

Applied Therapeutics said its embattled CEO Shoshana Shendelman resigned and was replaced on an interim basis by current CFO Les Funtleyder. The company also withdrew its European marketing application for the rare-disease drug govorestat. The FDA rejected the drug earlier this month.

obesity

Novo's CagriSema disappoints in pivotal trial

The long-awaited CagriSema results are here.

The next-gen obesity drug candidate from Novo Nordisk led patients to lose 20% of their weight at 68 weeks in a late-stage study when looking at all participants, including those who dropped out. It led to 23% weight loss when considering only the participants who stuck with treatment. The company had earlier projected weight loss of about 25%.

Shares of Novo sank over 20% in pre-market trading.

The results cast into doubt the future competitiveness of Novo in the booming weight loss market. Investors had been hoping for CagriSema to be a blockbuster obesity treatment that could succeed Wegovy, especially as Novo faces growing competition from Eli Lilly and other companies.

Drug approvals

FDA clears Ionis rare disease therapy

The Food and Drug Administration yesterday approved olezarsen, an antisense oligonucleotide drug developed by Ionis Pharmaceuticals to treat a rare genetic disorder called familial chylomicronemia syndrome. This is the first time Ionis will launch a drug entirely on its own — a strategic shift whereby no partnerships will help with commercialization.

In late-stage trials, olezarsen, which will be marketed as Tryngolza, significantly lessened triglyceride levels and reduced cases of pancreatitis, which is linked to the disease. Ionis is currently testing Tryngolza in three other Phase 3 studies in severe hypertriglyceridemia, which could expand the market for the treatment dramatically.

podcast

Is a contrarian leader good for the FDA?

Which ATTR-CM drug will have the best launch? And what are the hosts making for their holiday dinners?

We discuss all that and more on this week’s episode of “The Readout LOUD,” STAT’s biotech podcast. FDA reporter Lizzy Lawrence joins us to discuss her profile of Marty Makary, Trump’s nominee for FDA commissioner. Then, we discuss the results of Vertex’s latest pain drug trial, the medication launches to watch in 2025, and present our burning questions for the biopharma industry.

Listen here.

CLINICAL TRIALS

Need for more transparency in gene therapy studies

Despite the promise of gene therapy, many patients have died in trials testing the technology. There’s an urgent need for more transparency and better safety protocols in the space, opine ethics consultant Rafael Escandon and NYU medical ethics professor Arthur Caplan. The secrecy in the pharmaceutical industry is particularly problematic, they write, since it limits the information shared about adverse events — leaving regulators overburdened with analyzing and addressing safety issues across more than 1,000 gene therapy trials.

The authors say a more collaborative framework, where sponsors voluntarily let regulators share safety data across trials, could reduce risk.

glp-1 drugs

FDA declares tirzepatide shortage over

The FDA has confirmed that the ongoing tirzepatide shortage has come to an end. Now, regulators will allow compounding pharmacies a 60- to 90-day grace period to continue producing their copies of the wildly popular Eli Lilly diabetes and weight loss drug.

The move has sparked debate over the true availability of the drug, as patients and pharmacists still report ongoing difficulties in accessing it. Critics argue that the FDA’s shortage list often fails to reflect real-world access issues because it relies mainly on data from manufacturers.

Correction: Yesterday's newsletter misstated the terms of a deal Astellas signed with Sangamo Therapeutics. Astellas is paying $20 million upfront for the use of a gene therapy virus for a single neurological target, with options to pay for up to four more. The amount is in line with some deals for new viruses.

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