FDA approves Crinetics Pharmaceuticals' first drug

Crinetics Pharmaceuticals just won its first Food and Drug Administration approval with Palsonify, a once-daily pill for adults with acromegaly, a rare and debilitating hormonal disorder, who cannot undergo surgery or did not respond to it.

Backed by two late-stage trials showing significant drops in growth hormone and improved symptoms, the small-molecule drug offers patients an oral alternative to injectable or restrictive oral therapies from Novartis, Ipsen, and the Italian firm Chiesi, STAT's Jonathan Wosen writes.

Analysts see blockbuster potential if the drug is later approved to treat related disorders, though competition will be fierce.

gene therapy

Elpida lands funding, widens rare disease focus

Elpida Therapeutics, founded by CEO Terry Pirovolakis to develop a gene therapy for his son's rare hereditary disease, is broadening its reach.

Backed by $1.5 million from the CMT Research Foundation and CureCMT4J, the company will manufacture doses of its trial-ready gene therapy for Charcot-Marie-Tooth disease type 4J, an ultra-rare nerve disorder, with plans to launch a Phase 1/2 study in the U.S. and Spain. While the funding will cover most of the high costs of gene therapy manufacturing, it won't close the gap entirely.

At the same time, Pirovolakis has launched a consultancy to guide other rare disease efforts — from family foundations to emerging biotechs — through the current funding drought.

"We tried to give our programs away — to the NIH, to companies. We said, 'Take it for free, just save these kids.' No one would take it on," Pirovolakis told STAT. "So we decided to do the hard thing and start the consulting division."

podcast

Gene therapy success for Huntington's, Trump on autism

Why were doctors and investors so excited about trial results of Uniqure's gene therapy for Huntington's disease? Are autism rates actually rising? And what does the research tell us about Tylenol and autism?

We discuss all that on this week's episode of the "The Readout LOUD," STAT's biotech podcast. We bring on Uniqure CEO Matt Kapusta to discuss the company's announcement this week that its one-time treatment for Huntington's disease significantly slowed down the neurological condition in a key study.

We also chat about the Trump's administration initiatives on autism, and bring on by Ari Ne'eman, an assistant professor of health policy and management at Harvard's school of public health who researches how policies affect people with disabilities.

Listen here.

BIOTECH

In the U.K., mRNA is gaining ground

The U.K. is racing to stake its claim as a global hub for mRNA innovation, including by cutting the ribbon this week on a new $1 billion Moderna manufacturing site, STAT's Andrew Joseph writes.

As the U.S. pulls back funding under health secretary Robert F. Kennedy Jr., British leaders are framing the facility as both a symbol of industrial cooperation and a bet on mRNA's future beyond Covid. The site, which is meant to be able to churn out 100 million doses within six months of a crisis, could be used to develop everything ranging from cancer vaccines to heart disease treatments.

The "sector is one of our greatest national assets, and our partnership with Moderna is one of the jewels in the crown," U.K. Health Secretary Wes Streeting said.

Politics

Trump threatens 100% tariffs on prescription drugs — unless companies build in the U.S.

President Trump late yesterday threatened pharmaceutical companies with a 100% tariff — unless they build manufacturing plants in the U.S. — though precisely what he meant, or whether it was simply a negotiating tactic with the industry, was unclear.

The tariff would begin on Oct. 1 and could affect all "branded or patented" drugs, the president said in a social media post. Companies could avoid it by building manufacturing facilities in the U.S., Trump said, defining "building" as "'breaking ground' and/or 'under construction.'"

Read more from STAT's Daniel Payne.

breast cancer

FDA clears imlunestrant for ESR1-mutant breast cancer

The FDA has approved Eli Lilly's imlunestrant, sold as Inluriyo, for adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer after prior endocrine therapy. This is the company's second oral selective estrogen receptor degrader to reach the market, FierceBiotech points out.

Notably, regulators also cleared the Guardant360 CDx assay to help identify eligible patients. The approval is based on studies showing that imlunestrant cut the risk of disease progression or death by 38% over standard endocrine therapy, extending median progression-free survival to 5.5 months vs. 3.8 months.

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