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Right this moment, we see a small biotech on the verge of ending a rare-disease program due to FDA difficulties — and the way that impacts sufferers. We fete one of the best of this 12 months’s biopharma CEOs (trace: GLP-1s), and speak about post-doc and affected person advocacy reform.
The necessity-to-know this morning
- Illumina, the main maker of DNA sequencing machines, stated it might divest Grail, the developer of a multi-cancer screening take a look at, which it purchased over the objections of regulators within the U.S. and Europe in August 2021.
- A GLP-1 tablet made by Construction Therapeutics minimize blood sugar and weight in an early diabetes research, though the outcomes don’t fairly match a competing tablet from Eli Lilly.
- Level Biopharma, an acquisition goal of Eli Lilly, reported outcomes from a Part 3 research of its radiopharmaceutical in metastatic prostate most cancers.
Small biotech could abandon ultra-rare illness drug amid FDA frustrations
The FDA approval pathway is especially difficult for small biotechs chasing ultra-rare illness therapies. Take the case of Stealth BioTherapeutics, which is growing a remedy for Barth Syndrome.
Stealth has already spent $75 million in makes an attempt to win approval for its drug, referred to as elamipretide, however has all however given up hope. The FDA rejected the drug two years in the past, and if that occurs once more, Stealth will shutter this system within the U.S. — chopping off entry to what one household calls a “lifesaving drug.”
Their son practically died of a coronary heart assault earlier than receiving the experimental medication via the compassionate use program.
“This drug helped his coronary heart revert again to regular,” the kid’s mom instructed STAT. “The FDA actually allow us to save his life after they accredited compassionate use, however now they gained’t have a look at the info to save lots of greater than 120 different lives.”
Learn extra.
One of the best of the biopharma CEOs this 12 months
There’s a transparent king of the biopharma jungle this 12 months, in accordance with 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 medicine for diabetes and weight problems. There’s additionally anticipation of the doubtless approval of its Alzheimer’s drug donanemab, which slows illness development by 35%. Lilly’s market cap has now surpassed $500 billion, and there’s discuss that it’s going to in the end develop into the primary trillion-dollar pharma firm.
Take a look at the runners-up on Adam’s record right here.
NIH panel recommends paying post-docs extra
An NIH committee is urging that post-doc salaries enhance in a bid to assist stem the unprecedented exodus of younger life scientists away from academia and into business.
The group stated a post-doctoral wage ought to be a minimum of $70,000 starting subsequent 12 months, and that the work ought to be restricted to not more than 5 years. It additionally thought of the advantages of additional supporting worldwide post-docs.
In 1995, about 64% of newly minted Ph.D.s sought out post-doctoral positions. Final 12 months, that determine was right down to 53% — and there are already indicators that that is impacting science. There are extra untested hypotheses, unused grant {dollars}, and incomplete initiatives as a result of school can’t discover researchers to do the work. A STAT evaluation confirmed that Ph.D. graduates who’ve youngsters, scholar debt, or are Black or feminine, are much less more likely to pursue a postdoc than their friends.
Learn extra.
Reforming the present strategy to affected person advocacy
Affected person advocacy is potent. When teams champion their trigger loudly sufficient, the FDA listens — and alter is made. Sufferers are sometimes keen to just accept some uncertainty a couple of drug’s efficacy if it may possibly transfer the needle, even slightly, in an in any other case devastating illness. This mindset — and the numerous sources that got here together with it — doubtless helped push alongside the controversial aducanumab approval for Alzheimer’s illness.
“However the present state of affected person advocacy wants reform in order that one of the best, most secure medicine come to market,” opines Jon Hu, co-founder of Pepper Bio. Emotional appeals, nonetheless compelling, aren’t at all times based mostly in scientific rigor.
Hu means that advocacy teams cease sharing anecdotal proof with the FDA about therapies they suppose should advance; as a substitute, he says, they need to testify to Congress about why there’s a necessity for elevated analysis funding. And they need to have interaction extra straight with researchers and startups to assist brainstorm how higher to develop therapies for the illness, he says.
Learn extra.
Extra reads
- The Biden administration’s plan to make use of march-in rights to handle drug costs would kill future world-changing improvements, STAT
- FDA finds management lapses at Moderna manufacturing plant, Reuters
- Arcutis’ Zoryve luggage 2nd FDA nod, this time as a foam to deal with ‘the good uncared for illness in dermatology’, FiercePharma
- FDA converts accelerated to full approval for Padcev-Keytruda combo in bladder most cancers, Endpoints