December 18, 2023

The need-to-know this morning

Illumina, the leading maker of DNA sequencing machines, said it would divest Grail, the developer of a multi-cancer screening test, which it bought over the objections of regulators in the U.S. and Europe in August 2021.
A GLP-1 pill made by Structure Therapeutics cut blood sugar and weight in an early diabetes study, although the results don't quite match a competing pill from Eli Lilly.
Point Biopharma, an acquisition target of Eli Lilly, reported results from a Phase 3 study of its radiopharmaceutical in metastatic prostate cancer.

rare disease

Small biotech may abandon ultra-rare disease drug amid FDA frustrations

The FDA approval pathway is particularly challenging for small biotechs chasing ultra-rare disease treatments. Take the case of Stealth BioTherapeutics, which is developing a therapy for Barth Syndrome.

Stealth has already spent $75 million in attempts to win approval for its drug, called elamipretide, but has all but given up hope. The FDA rejected the drug two years ago, and if that happens again, Stealth will shutter the program in the U.S. — cutting off access to what one family calls a "lifesaving drug."

Their son nearly died of a heart attack before receiving the experimental medicine through the compassionate use program.

"This drug helped his heart revert back to normal," the child's mother told STAT. "The FDA literally let us save his life when they approved compassionate use, but now they won't look at the data to save more than 120 other lives."

biopharma

The best of the biopharma CEOs this year

There's a clear king of the biopharma jungle this year, according to STAT's Adam Feuerstein: David Ricks of Eli Lilly, who delivered a flabbergasting 60% return, thanks largely to explosive demand for Mounjaro and Zepbound, its GLP-1 drugs for diabetes and obesity. There's also anticipation of the likely approval of its Alzheimer's drug donanemab, which slows disease progression by 35%. Lilly's market cap has now surpassed $500 billion, and there's talk that it will ultimately become the first trillion-dollar pharma company.

Check out the runners-up on Adam's list here.

talent

NIH panel recommends paying post-docs more

An NIH committee is urging that post-doc salaries increase in a bid to help stem the unprecedented exodus of young life scientists away from academia and into industry.

The group said a post-doctoral salary should be no less than $70,000 beginning next year, and that the work should be limited to no more than five years. It also considered the benefits of further supporting international post-docs.

In 1995, about 64% of newly minted Ph.D.s sought out post-doctoral positions. Last year, that figure was down to 53% — and there are already signs that this is impacting science. There are more untested hypotheses, unused grant dollars, and incomplete projects because faculty can't find researchers to do the work. A STAT analysis showed that Ph.D. graduates who have children, student debt, or are Black or female, are less likely to pursue a postdoc than their peers.

patient advocacy

Reforming the current approach to patient advocacy

Patient advocacy is potent. When groups champion their cause loudly enough, the FDA listens — and change is made. Patients are often willing to accept some uncertainty about a drug's efficacy if it can move the needle, even a little, in an otherwise devastating disease. This mindset — and the significant resources that came along with it — likely helped push along the controversial aducanumab approval for Alzheimer's disease.

"But the current state of patient advocacy needs reform so that the best, safest drugs come to market," opines Jon Hu, co-founder of Pepper Bio. Emotional appeals, however compelling, aren't always based in scientific rigor.

Hu suggests that advocacy groups stop sharing anecdotal evidence with the FDA about therapies they think ought to advance; instead, he says, they should testify to Congress about why there's a need for increased research funding. And they should engage more directly with researchers and startups to help brainstorm how better to develop treatments for the disease, he says.