August 4, 2023

The need-to-know this morning

The FDA rejected a stem cell therapy made by Mesoblast intended to treat children with an advanced form of graft-versus-host disease following bone marrow transplants.
Amgen said the combination of its KRAS-targeted cancer drug Lumakras with another antibody drug called Vectibix delayed the growth of tumors in patients with advanced colon cancer, achieving the goal of a Phase 3 study. Detailed results were withheld for presentation at a future medical meeting.
An FDA approval decision is expected later today for zuranolone, a treatment for major depressive disorder and postpartum depression developed by Sage Therapeutics.

diagnostics

Micronoma's microbiome-based cancer test scrutinized

The San Diego startup Micronoma has built its platform on the idea that microbes colonize tumors — and the molecular signatures they create could accurately identify cancer types. However, the Nature paper upon which the company's whole premise is based was just challenged by researchers, who published a manuscript on the preprint site bioRxiv.com that shows two "fatal errors" in the study.

It claims that the Nature paper didn't correctly filter out human DNA from a database of tumor samples, classifying millions of human sequences as bacterial. And another reported error in the team's algorithm drew inaccurate connections between some microbial signatures and cancer types.

"From that point on, nothing is valid," said the Johns Hopkins computational biologist who coauthored the preprint. "Garbage in, garbage out, as they said."

podcast

Can biotech startups survive a downturn?

Is Covid-19 a good business? And when did ophthalmology get so dramatic?

We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast. First, we discuss how some recent layoffs and a reverse merger mean for biotech in 2023. We'll also discuss the latest news in the life sciences, including a dramatic saga in ocular medicine, the volatile business of Covid-19, and some major milestones in gene therapy.

Listen here.

supply chain

White House task force on drug shortage meets

A new White House task force met last Friday to discuss ongoing drug shortages — gathering experts from hospitals, biopharma companies, pharmacies, and other parts of the drug supply chain. The focus was on how to quickly get the drug supply back on track, potentially by importing medicines from other countries in the near term. The task force ultimately intends to recommend drug shortage reforms, but hasn't laid out a timeline for this yet.

Part of the meeting was focused on cisplatin, a widely prescribed chemotherapy drug that's in short supply. The FDA has already allowed temporary imports of cisplatin from China — but it's still unclear whether the new stocks are indeed meeting an unmet need, or if facilities are buying it in bulk to shield against future shortages. These uncertainties have made it difficult to broadly address the drug shortage crisis.

cystic fibrosis

Vertex criticized for high cost in low- to middle-income countries

Vertex Pharmaceuticals is being pressured by health equity advocates to improve global access for its cystic fibrosis medicines. Clinicians and advocacy groups are arguing in public letters that the company's pricing is excessive for low- and middle-income countries: "Vertex's pricing strategy represents a formidable barrier to universal access to treatment," one letter directed at Vertex CEO Reshma Kewalramani says.

Latin advocacy groups point out that patients in Colombia have a "considerably lower life expectancy" compared to those in the U.S. — saying "this stark disparity in life expectancy underscores the urgent need" for the company's powerful treatments.

These new missives are in addition to many more from cystic fibrosis patients and their families, who have long argued that the cost of Vertex's medicines are excessive.