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Hello, everyone. Damian here with the highlights from the STAT Summit, a question on CRISPR, and career advice from a biotech elder statesman.
J&J is gunning for AstraZeneca’s spot
The jewel of AstraZeneca’s recent renaissance is Tagrisso, a cancer treatment whose potency won a standing ovation from treating oncologists and made the company the envy of its contemporaries in cancer. But Johnson & Johnson, maker of a competing combination, is working to unseat its rival with promising new data.
That’s just one subplot of the annual meeting of the European Society for Medical Oncology, which kicks off Friday. Last month, J&J said a pair of its medicines outperformed Tagrisso in a study enrolling patients with a form of lung cancer. As STAT’s Andrew Joseph reports, experts are eager to pick apart the details of that study when it’s presented at ESMO, data that will determine whether J&J has a viable competitor to AstraZeneca’s top-selling medicine.
Elsewhere at ESMO are potentially practice-changing results in breast, prostate, and lung cancers, with anticipated presentations from Novartis, Amgen, and others.
What if there’s no market for a CRISPR cure?
Intellia Therapeutics is on pace for a medical milestone, preparing the first pivotal study of a CRISPR medicine that does its genome editing inside a patient’s body. But the company’s chosen target, an increasingly prevalent heart disease, will likely have a bevy of powerful drugs by the time Intella’s treatment might hit the market, suggesting the pathbreaking therapy might amount to a middling product.
The news is that Intellia won the FDA’s blessing to start a Phase 3 trial for NTLA-2001, a CRISPR-based treatment for the progressive cardiovascular disorder ATTR-CM, a disease that results from misfolded proteins that damage healthy tissue. Existing drugs for the disease work by either stabilizing those proteins or silencing the gene responsible for making them. Intellia’s would be a one-time treatment designed to correct the genetic defect for good.
The scientific case for NTLA-2001 is strong, but the commercial one is a little murky. The only approved ATTR-CM drug is a pill from Pfizer that brings in about $2.5 billion a year. BridgeBio is expected to win approval for a competing and arguably superior drug next year, while Alnylam Pharmaceuticals is awaiting pivotal data that would allow it to enter the ATTR-CM market in 2025.
None of those treatments is a cure, as NTLA-2001 could turn out to be, but by the time Intellia can compete, its forebears will have years of data and physician experience that might significantly cut into demand for a newfangled medicine with irreversible effects.
We can’t all be wunderkinds
John Maraganore, the former long-time CEO of Alnylam Pharmaceuticals, has settled into the role of an elder statesman, advising new companies, sitting on boards, and mentoring biotech’s next generation. One common piece of advice: There’s value in working your way up.
“It seems like every Ph.D. graduate student wants to be the CEO of their graduate thesis company,” Maraganore said at the STAT Summit yesterday. “… If my child asked me what to do, I’d say, ‘Spend 10 years learning drug discovery, learning drug development before you do anything on your own. Because it’s hard. It’s very hard.’”
Maraganore made clear that he balances those words of caution with encouragement for any young entrepreneurs with enough passion to pursue scientific ideas on their own. But the notion that every scientist with a doctorate and dream needs to be a CEO? “I think that’s a little bit crazy,” Maraganore said.
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When science is tough, it helps to have a few billion dollars
The biggest stories in drug development — obesity and Alzheimer’s disease — are driven not by plucky biotech companies and risk-taking venture capitalists, but rather by a nearly 150-year-old pharma company based in a city better known for speed racing than science.
That’s no coincidence, Eli Lilly chief scientist Daniel Skovronsky said at the STAT Summit yesterday. Mounjaro took years and years of work in a space once perceived as a graveyard for drug research. Donanemab, Lilly’s Alzheimer’s treatment, came only after a series of painful and costly failures. Committing that kind of time and money is much more practical when you’re a profitable, multinational company with an army of researchers.
“It’s duration of interest, perseverance in a field for decades, which small companies just can’t do,” Skovronsky said. “But big companies can do it, and we can do it in areas that no one else wants to work in.”
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More reads
• Zuckerberg and Chan announce a New York biohub to build disease-fighting cellular machines, STAT
• Biotech investors push to extend time before pills are subject to price negotiations, Boston Globe
• Lotte Knudsen, who pushed Novo Nordisk into the Ozempic era, wins STAT Innovation award, STAT
• Hyloris Pharma wins U.S. FDA approval for pain treatment, Reuters
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