September 16, 2024

The need-to-know this morning

Ascendis Pharma reported positive results from a Phase 3 study of Transcon CNP in children with achondroplasia, the most common form of dwarfism. Children treated with the Ascendis drug grew an average of 1.49 centimeters in a year relative to a placebo, achieving the primary goal of the study.
BioMarin Pharmaceutical was trading lower in the pre-market session on investors' concerns that its currently marketed treatment for achondroplasia, called Voxzogo, could lose market share to Ascendis' more convenient treatment, if approved.

restructuring

Is Genentech losing its zest for innovation?

Genentech has long been known for innovation, but that reputation has been questioned in the wake of multiple rounds of layoffs and the company's closure of its cancer immunology group. On top of that, the departure of renowned scientist Ira Mellman has sparked fears that Genentech has a new strategy: moving away from in-house innovation and instead licensing external therapies, which some worry will lessen its scientific rigor.

"Does the world need yet another mega pharmaceutical company that just buys innovation?" a former employee told STAT's Jonathan Wosen. "I love Genentech, what it stands for, what its values are, and I would like it to be the Genentech that I've always known, because there isn't another one like it."

cancer

An unusual wager on Exact Sciences data

Screenshot 2024-09-15 at 3.55.03 PM

A liquid biopsy test for colon cancer made by Exact Sciences detected 88.3% of cancers and 31.2% of advanced precancers in a study of some 3,000 samples, STAT's Jonathan Wosen writes. But the sample size, which was presented at ESMO, is small — and a larger, 20,000-participant study is underway. Experts warn that data from smaller trials often yield better results than larger, definitive studies.

On that note, in a highly unorthodox LinkedIn post, Guardant Health CEO AmirAli Talasaz bet $1 million that the ESMO data from Exact, his company's competitor, won't hold up in the follow-on trial.

"And if I was a cancer patient reading that line?!?" Michael Pellini, former CEO of Foundation Medicine, commented in response. "Friend or foe, I've always wanted everyone to win for the betterment of cancer care. Try donating money instead."

animal testing

Opinion: Be kinder to these sad mice

The forced swim test was developed in 1977 to trigger depression in mice: The animals are placed in small tanks filled with water, and escape is impossible. The mouse at first tries to escape, but as it tires, it floats immobile, which is then considered a mouse model of a depressive state. Another test, the tail suspension test, was developed in 1985 and has a similar premise. Scientists have tested antidepressants on these mice for decades — but results have been unreliable and the practices here are unjust and inhumane, opines Harvard psychiatrist Karen S. Greenberg.

"It is high time for researchers to retire the forced swim and tail suspension tests," she writes. "Stop spending taxpayer dollars on obsolete tests that needlessly harm animals and leave patients in the lurch by diverting research funds to bad science."

esmo

Imfinzi increases survival rates in bladder cancer

Imfinzi, an immunotherapy made by AstraZeneca, was used before and after surgery in a clinical trial of patients with muscle-invasive bladder cancer — and it dramatically improved survival rates, according to new data. A Phase 3 study presented at ESMO showed that it reduced the risk of death by 25% and disease recurrence by a third.

Two years after treatment, 82.2% of patients given Imfinzi were alive, versus 75.2% in the control arm.

This could potentially reshape bladder cancer care — though there are still some regulatory concerns around the company's trial design. At issue is AstraZeneca combination of pre- and post-surgery data instead of parsing individual efficacy.

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