September 2, 2025

The need-to-know this morning

United Therapeutics reported positive results from a Phase 3 study of its inhaled medicine Tyvaso in patients with idiopathic pulmonary fibrosis.
Replimune Group said a meeting with FDA officials has been scheduled to discuss the recent rejection of its treatment for advanced melanoma.

startups

Veteran drugmakers launch Corsera to fight heart disease

Biotech veterans John Maraganore, previously the CEO of Alnylam, and Clive Meanwell, previously the CEO of The Medicines Company, have teamed up before to develop drugs. Now they're doing so again to launch Corsera Health, a startup aiming to prevent cardiovascular disease, STAT's Allison DeAngelis writes. The company is developing an AI risk-prediction tool and a dual-target RNAi therapy against PCSK9 and angiotensinogen

Backed with $50 million from their own funds, the pair plan to start clinical trials this year while tackling the enormous challenge of scaling oligonucleotide manufacturing to reach hundreds of millions of patients. Both men, who previously collaborated on the drugs Angiomax and Leqvio, say Corsera is the most important project of their careers — a bold third act they hope can transform how the world combats its leading killer.

vaccines

Trump pressures drugmakers amid CDC turmoil

In a social media post, President Trump yesterday demanded that pharmaceutical companies publicly prove the effectiveness of their Covid-19 products. Putting aside the overwhelming evidence of their effectiveness, the striking post, in which Trump also said he'd been shown "extraordinary" data by Pfizer "and others," appeared to underscore tensions within his administration after the ouster of CDC Director Susan Monarez and the resignation of several top agency officials, STAT's Daniel Payne and Matthew Herper write.

The post comes as health secretary Robert F. Kennedy Jr. pushes an aggressive overhaul of the CDC as part of an effort that former directors and public health experts warn is crippling the agency. Reactions ran the gamut.

"If you look at the reactions to that weird post… maybe one begins to understand it," said one former government official, speaking on condition of anonymity to address the issue candidly. "The anti-vaxxers love it, they think he's saying Pfizer lied to him. The public health people like it too, they think he's trying to create daylight with RFK."

The former official said he "would not want to be Al Bourla today," referring to the Pfizer CEO.

drug development

A clinical success for Ionis with olezarsen

Olezarsen, an antisense drug made by Ionis Pharmaceuticals, hit the mark in two Phase 3 studies, cutting triglycerides by up to 72% and reducing pancreas inflammation by 85% in patients with severe hypertriglyceridemia. The therapy, already approved as Tryngolza for the ultra-rare FCS at nearly $600,000 a year, was safe and well tolerated, with most patients continuing into an extension trial, STAT's Jonathan Wosen writes.

"These are really groundbreaking outcomes in a highly prevalent disease where the unmet need is very, very big," CEO Brett Monia told STAT. "It's a big deal for the medical community. It's a big deal for patients. And for Ionis, it's also incredibly strategic."

The company has been shifting into a fully independent commercial operation, bringing all sales in house. An expanded approval could buoy that effort: Analysts say peak sales of olezarsen could hit $2 billion.

heart disease

Cytokinetics drug outperforms beta blocker in HCM

Cytokinetics' experimental drug aficamten beat the standard-of-care treatment in a head-to-head Phase 3 trial of newly diagnosed patients with obstructive hypertrophic cardiomyopathy. Aficamten improved exercise capacity in patients while metoprolol, a beta blocker, worsened it.

In a 175-patient study, aficamten also delivered stronger symptom relief and biomarker improvements, with fewer dose reductions than metoprolol, STAT's Adam Feuerstein writes.

CEO Robert Blum called the results "provocative," saying that "these data will call into question, what should be a first-line therapy, eventually."

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