Closer Look
How 30 years of research brought the first drug for frostbite
Earlier this week, the FDA granted a pioneering approval to the first medicine that can prevent the need for amputation for people who develop frostbite. It only took three decades, some circuitous development, and a pivot from an obscure biotech company.
As STAT's Jason Mast reports, the drug is iloprost, an intravenous medicine that affects blood flow. Back in the 1990s, after decades of misdirected and sometimes misinformed research, a French scientist found a case report suggesting iloprost might stave off the worst effects of frostbite. Over the course of nearly 15 years, he conducted a first-of-its-kind study enrolling mountain climbers in the Alps.
The results — none of the 16 patients who took iloprost needed amputations — set in motion a transcontinental effort to treat frostbite, a particularly vexing public health problem in cold-weather cities dealing with homelessness. And they informed an FDA approval with a winding backstory.
Read more.
Financials
Business is good for biotech's payday lender
The recent downturn for small drug companies has been a drag on biotech and the many firms that make money off of it. But the industry's misfortune has been kind to Royalty Pharma, a company that trades short-term cash for a cut of biotech's future revenue.
Royalty Pharma's latest quarterly earnings beat Wall Street's expectations, and the company forecast annual growth in 2024 that outpaced analysts' projections.
The company's business model is essentially counter-cyclical. The harder it is for biotech companies to raise money, the better Royalty Pharma's position to negotiate deals. And the company expects its work during the downturn to keep paying off: Royalty Pharma projected that its transactions since 2020 will add about $1.2 billion to its revenue in 2025.
Biotech
Alnylam thickened the plot
The results of Alnylam Pharmaceuticals' Phase 3 cardiovascular study was already biotech's biggest binary event in the first quarter of 2024. Now, with a delay and change in study design, it's the biggest one of the year.
The news is that Alnylam's study, testing whether vutrisiran can improve the lives of patients with the progressive heart disease ATTR-CM, will now read out in the summer. Previously, the primary endpoint was comparing vutrisiran to placebo on the measures of mortality and cardiovascular problems. Now, Alnylam will analyze data both from the overall study population and the roughly 60% of patients who did not receive Pfizer's tafamidis, the only approved treatment for ATTR-CM.
To Wall Street, that doesn't bode well. Shares of the company fell more than 10% on fears that Alnylam made this decision after looking at blinded data and noticing that the rates of heart failure and death looked consistent among all patients, suggesting little difference between vutrisiran and placebo. But it's also possible the company is simply being prudent, maximizing its odds of success and tweaking the trial to minimize future pushback from payers. We'll find out in a few months.
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