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Happy Saturday and welcome back to Endpoints Weekly! We had a flurry of news this week, so let’s dive in.
Our team has the latest news and analysis around the Trump administration’s executive order intended to lower drug pricing via a “most favored nation” model. Keep reading for details on Bayer’s latest staff cuts, Galapagos’ reconsideration of its spinout plans, and GSK’s $1.2 billion deal in MASH, a field that has been reinvigorated on the heels of a landmark approval last year.
Set aside some extra time to read Ryan Cross’ inside story on the first bespoke CRISPR therapy used to treat an infant with a rare metabolic disease. The patient, at nine and a half months old, is now eating an age-appropriate amount of protein, has gained weight, and biomarkers in his urine suggest the treatment worked. The story is part of an Endpoints series on the future of CRISPR. Be sure to also check out the first two installments if you haven’t already. — Nicole DeFeudis
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Nicole DeFeudis |
Editor, Endpoints News |
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What we know about ‘most favored nation’ |
🖋️ President Donald Trump signed an executive order on Monday aimed at lowering the cost of drugs in America. The order “instructs the Administration to communicate price targets to pharmaceutical manufacturers to establish that America, the largest purchaser and funder of prescription drugs in the world, gets the best deal,” according to a fact sheet.
A similar model was attempted during Trump's first term, but it was halted in courts following procedural challenges. According to the Monday order, HHS is expected to communicate “most favored nation price targets” to drugmakers within 30 days, with the goal of bringing “prices for American patients in line with comparably developed nations.”
If companies do not make “significant progress” toward that pricing, the executive order reads, HHS Secretary Robert F. Kennedy Jr. will be tasked with proposing a rulemaking to “impose” a most favored nation pricing plan.
While industry trade groups have pushed back on MFN pricing in the past, Jefferies analysts said on Monday that the order was “better than feared.” Several large pharma company stocks were trading up on Monday, including Merck, Regeneron and AbbVie, Max Gelman reported. Benjamin Jackson, a Jefferies analyst, wrote in a note to investors that questions remain about how the plan would be implemented.
Endpoints’ Drew Armstrong dug into some of those questions on Tuesday in a live conversation with Nick Shipley, a former top lobbyist for the biotech industry. Watch the replay here. And see what Bristol Myers Squibb CEO Chris Boerner had to say about the policy and America’s broader position as a global biopharma leader during the Financial Times’ US Pharma and Biotech Summit here. |
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GSK’s $1.2B MASH deal |
💰 The UK pharma company will serve up a huge wad of cash to acquire a program from Boston Pharmaceuticals called efimosfermin alfa. Previously known as BOS-580, the drug is being tested in two Phase 2 trials, one of which reported data last November. For accounting purposes, GSK is treating the deal as an M&A transaction by acquiring a Boston subsidiary called BP Asset IX. Boston can also get up to another $800 million in potential milestones.
GSK isn’t typically known as a metabolic disease drug developer. The company sat out the obesity craze, ceding ground to European rival Novo Nordisk. But execs were apparently impressed enough — and considered it to be a safe bet, given the front-loaded nature of the deal — to shell out the big bucks for efimosfermin alfa. The drug, in fact, was originally developed by Novartis before Boston licensed it in 2020, and Novartis is also eligible for some milestone payments.
Phase 3 trials are the likely next step, though GSK didn’t say this week when such studies might start. The company did say, however, that if everything goes according to plan, efimosfermin alfa could launch in MASH in 2029. Boston had previously said late-stage studies would begin by the end of this year. GSK also plans to test the drug in alcohol-related liver disease. |
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Bayer details staff cuts |
Bayer said it cut 2,000 positions in Q1, bringing total reductions to 11,000 since the company started a multiyear restructuring. Bayer noted that the recent cuts mostly affected those in management and coordination positions.
Bayer has been working toward a new operating model for more than a year. Last year, CEO Bill Anderson cited the company’s “hierarchical bureaucracy” as one of four “badly broken” areas at the company. He said Bayer is focused on “two big enablers” during a media call this week: “First, freeing up resources so our teams can flow them to the highest impact work. Second, installing people enablement tools that are fit for our new system.” |
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Galapagos reconsiders spinoff plans |
🔄 Back in January, Galapagos outlined a €2.45 billion plan to spin out its dealmaking and pipeline hunting efforts into a new company, while Galapagos focused on its cell therapy business. But the biotech surprisingly said it would reconsider the move this week, throwing the plans into question. Galapagos cited “regulatory and market developments” as the reason for its decision.
With the update, CEO Paul Stoffels stepped down immediately (though he had announced plans to retire last month). Former AbbVie and Neumora executive Henry Gosebruch, who was expected to run the spinoff, took Stoffels’ place instead. There is no timeline for when Galapagos might make its strategic shift, with Gosebruch telling senior reporter Kyle LaHucik that it’s “hard to commit to a specific timeline or predict exactly how this would play out.” |
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CRISPR therapy corrects infant’s DNA for first time |
🧬 In a landmark study, researchers said they’ve corrected an infant’s genetic mutation for the first time using CRISPR. A team at the Children’s Hospital of Philadelphia and the University of Pennsylvania developed the treatment for nine-month-old KJ, who had an ultra-rare disease that caused excessive buildup of ammonia in his liver. While it’s too soon to declare this a cure, KJ is eating protein like a healthy infant his age and biomarkers in his urine suggest the gene editing worked.
Senior science correspondent Ryan Cross has the inside story of this study, whose results were published in the New England Journal of Medicine this week. Grab a cup of coffee and be sure to read all about the six-month sprint that led to the creation of a custom CRISPR gene editing drug to directly fix one of KJ’s mutations. It’s likely the fastest-ever creation of a new drug after nabbing a one-week FDA review in February. |
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