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Gilead sours on the ‘don’t eat me’ business

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Good morning, everyone. Damian here with the latest Senate harangue for the drug industry, green shoots in biotech sentiment, and the potential of a gene therapy eye drop.


The need-to-know this morning

  • AstraZeneca reported fourth quarter and 2023 earnings.
  • BioNTech and Autolus announced a CAR-T cell therapy partnership.
  • Kyverna Therapeutics, a developer of cell therapies for autoimmune diseases, priced its IPO at $22 per share, raising $319 million.
  • The Phase 3 study of resmetirom, a treatment for MASH made by Madrigal Pharmaceuticals, was published in the New England Journal of Medicine.

Pharma returns to Washington

Sen. Bernie Sanders, who all but séanced Frederick Banting to scold the CEO of Eli Lilly and tried to sell Moderna’s Stéphane Bancel on nationalizing pharmaceutical research, will again play haranguing host to drug industry executives.

This time, the CEOs of Merck, Johnson & Johnson, and Bristol Myers Squibb will appear before the Senate health committee, led by Sanders, in a hearing titled “Why Does the United States Pay, by Far, the Highest Prices in the World for Prescription Drugs?” that kicks off at 10 a.m. ET today.

These things rarely lead to much in the way of substantive debate, but it’s worth noting that each CEO is fairly new to the job, and each is following in the footsteps of long-tenured executives who had some experience with congressional grilling. How they hold up in front of Sanders could be instructive.


You can watch the hearing here, and be sure to check STAT later today for more coverage.

Gilead sours on the ‘don’t eat me’ business

Gilead Sciences will no longer develop magrolimab, a treatment targeting the “don’t eat me signal” exploited by tumors in blood cancer after another clinical setback that makes its $5 billion investment in the space look like money entirely wasted.

The news is that Gilead terminated a Phase 3 study in acute myeloid leukemia after an interim analysis found the trial was futile and patients had an increased risk of death when given the treatment. The FDA placed a clinical hold on all studies of magrolimab, effectively halting its development.

Treatments like magrolimab, which target a protein called CD47, were always a high-risk, high-reward proposition. Back in 2018, years before Gilead bought into the space by acquiring a firm called Forty Seven, researchers warned that blocking CD47 could have disqualifying safety issues.

‘Spring is coming early to biotech’

At least according to TD Cowen’s latest “sentimometer,” a quarterly survey of investors that has typically pointed to pessimism, doubt, and even despair in recent years.

This time, everything’s much rosier. About 44% of respondents said mid-sized biotech companies were now under-valued, reversing a long-standing trend, and roughly half expect that same basket of companies to outperform the broader market. And for the first time since the early days of Covid-19, a majority of respondents expect small-cap biotech companies to beat the market.

All told, sentiment has rebounded from record lows last year, TD Cowen analysts wrote in a note to investors, and with investors bullish about some of the major catalysts ahead in 2024, “spring is coming early to biotech.”

How a wound-healing gene therapy restored vision

A topical gene therapy from Krystal Biotech is FDA approved to treat epidermolysis bullosa, a rare disorder that leaves skin fragile and prone to blisters and tears. But could an ocular dosing of the treatment help patients whose disease affects the tissues of the eye?

The answer, at least for one patient, is yes. As STAT’s Andrew Joseph reports, physicians administered the therapy to a 13-year-old boy whose epidermolysis bullosa, or EB, had severely impaired vision in his right eye. After the dosing, conducted under the FDA’s compassionate use program, his vision is now 20/25, not far off normal.

The case, detailed in a New England Journal of Medicine paper published yesterday, could help expand the utility of Krystal’s therapy, marketed as Vyjuvek. A company spokesperson said Krystal is discussing with the FDA what sort of evidence would be required to win authorization for an eye-drop version of its product.

Read more.

More reads

  • Government warns Medicare Advantage insurers not to deny care based on AI, STAT
  • China panel plans biotech field hearing, Axios
  • E.U. watchdog looking into impact on drug availability from Catalent-Novo deal, Reuters
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