April 26, 2024

The need-to-know this morning

The last pharma earnings report for the week! Abbvie posted better-than-expected results for the first quarter, driven by strong sales from its immunology drug franchise. The company raised its outlook for the remainder of the year. Humira sales fell 35% to $2.27 billion, slightly above consensus, according to Factset.
Apellis Pharmaceuticals said European regulators have agreed to "reset" the review of its eye drug Syfovre, following a prior negative decision. The company expects European regulatory advisers to render a new recommendation on the drug by July.

gene therapy

Pfizer's gene therapy wins approval

From STAT's Jason Mast: The FDA approved Pfizer's hemophilia B gene therapy, the company announced this morning. It's the second one-time treatment authorized for the rare bleeding disorder, joining a therapy developed by Uniqure and CSL Behring. And it's the first gene therapy developed by Pfizer, which began investing heavily in the field in the mid-2010s.

Pfizer will charge $3.5 million for the drug, known as Beqvez, but it's not clear if it'll be a moneymaker. CSL has so far struggled to sell its treatment, which carries the same price, although some hematologists think uptake will increase over time, given the treatments' ability to dramatically reduce the risk of severe bleeds.

Concerns about market potential played a role in Pfizer's decision last year to wind down much of its gene therapy work and sell it to AstraZeneca. Alongside early stage gene editing work, the company is still developing a gene therapy for Hemophilia A and could announce data on a muscular dystrophy gene therapy soon.

artificial intelligence

David Baker has his eye on Goldilocks drug candidates

Computational biologist David Baker rose to acclaim after his University of Washington lab unveiled an AI-driven protein-folding prediction system that rivaled Google's DeepMind AlphaFold. Now, he's looking beyond proteins, developing a trove of macrocycle drug candidates — that is, medicines that are larger than small molecules but smaller than biologics.

"Look, there's this huge class of compounds we can make that chemically are just very, very different from proteins," Baker told STAT's Brittany Trang. They're made up of "building blocks that really don't look anything like the L-amino acids that existing proteins [have], and have all kinds of unnatural backbones and chemistries."

Macrocycle drugs can be taken orally and are svelte enough to slip through cell membranes like small molecule drugs. But they can also target surface receptors that are typically only compatible with antibody drugs.

markets

And we're back in a bear market again

The XBI has sunk down to $82.22 — marking its official reentry into bear market territory. Behold, a view of the biotech index's drop over the past two months:

pJNH0-xbi-enters-bear-market(1)

This is sadly reflected in the performance of biotech companies that IPO'd this year: They simply "haven't traded so well," Baird analyst Mike Perrone told CNBC a couple days back. "We kind of started Q1 with a roar and ended with a whimper."

artificial intelligence

Using AI to diagnose Mendelian disorders

An intriguing new artificial intelligence approach to diagnosing inherited diseases was just outlined in NEJM-AI. The tool, called AI-MARRVEL, trained on 3.5 million genetic variants from thousands of Mendelian disorders. With a precision rate of 98%, it correctly identified 57% of diagnosable cases out of 871 cases.

"The diagnostic rate for genetic disorders is only about 30%, and on average, it is six years from the time of symptom onset to diagnosis," Pengfei Liu, one of the study's authors and a researcher at Baylor University, said in a statement. "There is an urgent need for new approaches to enhance the speed and accuracy of diagnosis."

The tool does have several limitations, however: While it can process single gene mutations and small insertions or deletions, it isn't yet able to analyze structural variations or copy-number variations. But the authors say that it has the potential to discover novel disease genes — and think AI-MARRVEL could ultimately be used by clinicians and researchers to identify and interpret many genetic variations.

return on investment

Deloitte report: What's the value of an approval?

There's been a boost in the return on investment for pharma R&D, a new Deloitte report shows. In 2023, ROI rose by 4.1% — a substantial leap from the sector's low of 1.2% in 2022, and a pandemic peak of 6.8% in 2021. That said, it's still stymied by patent cliffs, a shapeshifting regulatory system, and how complex and quickly evolving science is becoming.

Screenshot 2024-04-25 at 1.35.14 PM

The average R&D cost to develop an asset from discovery to launch seems fairly level. But with the exception of that Covid-19 sales boost in 2021, there hasn't been improvement in terms of peak sales per asset, the Deloitte numbers show. Which does stir up an ever-looming question: What's the true financial value of an approval today?

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