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Good morning, my colleagues were diligently roaming the halls of Congress yesterday, talking to senators about their thoughts on RFK Jr. Read on to hear what the lawmakers said.
The need-to-know this morning
- Merck acquired a preclinical, oral GLP-1 obesity drug candidate from China’s Hansoh Pharma. Shares of Viking Therapeutics fell on the Merck announcement. Viking is developing its own obesity drugs and has been viewed as a potential acquisition target by pharma companies, including Merck.
RFK Jr. builds Senate support, as FDA deputy commissioner prepares to leave
Robert F. Kennedy Jr. is meeting with nearly two dozen Republican senators this week in a bid to clinch their votes for his confirmation as HHS secretary. STAT approached more than a dozen of those senators yesterday, and none said they’d oppose his nomination outright, though a few said they have concerns about his views on vaccines.
Some described productive conversations and signaled they support Kennedy’s nomination. He appears to have appeased more conservative lawmakers on his views around abortion. Read more from my D.C. colleagues.
Meanwhile, Namandjé Bumpus, principal deputy commissioner of the FDA, will leave the agency at the end of the year, Commissioner Robert Califf announced yesterday. Bumpus replaced the retiring Janet Woodcock late last year and oversaw the FDA’s recent reorganization, including the creation of a more unified human foods center.
Her departure comes as the Trump administration prepares to take office. The incoming FDA leader is expected to bring new staff and confidantes into the commissioner’s office; additionally, RFK Jr., a top ally to President-elect Trump, was hostile to FDA career civil servants during the election cycle. He said that if staff are part of a “corrupt system,” that they should “pack their bags.” Read more from STAT’s Lizzy Lawrence.
Tessera’s in vivo sickle cell program gets Gates funding
From my colleague Jason Mast: Tessera Therapeutics said this morning it received an investment from the Bill & Melinda Gates Foundation to support the development of an in vivo gene-editing treatment for sickle cell disease. Tessera and the Gates Foundation will co-fund the program, with Gates potentially putting in up to $50 million.
A gene-editing sickle cell treatment approved last year, developed by Vertex Pharmaceuticals, is ex vivo, meaning a patient’s cells have to be removed from the body, edited in the lab, and then returned. In the meantime, patients need to receive toxic chemotherapy to clear out old diseased cells. Because of the toll that chemo can take, only a tiny handful of patients have so far received a commercial sickle cell gene therapy.
An in vivo treatment would mean editing patients’ cells with a simple IV infusion. Such an approach could be safer and more scalable, both to patients in the U.S. and Europe and around the world — which is why it’s been a focus for a growing number of drugmakers and the Gates Foundation. Gates has funded in vivo sickle cell efforts at Novartis and Intellia, though both remain at an early stage.
Tessera has emerged as a potential leader in the space, showing data at the American Society of Hematology meeting suggesting it can edit 24% of blood stem cells in monkeys. But the company is likely still years away from the trials.
An ‘obvious’ choice for 2024’s worst biopharma CEO
Yesterday, we brought you my colleague Adam Feuerstein’s pick for 2024’s best-ish industry CEO. (He actually didn’t name a single best CEO, since he thought there wasn’t one individual that stood out among the others.)
Today, we bring you his pick for worst CEO of the year, and for this, he had no difficulty choosing one: Remi Barbier, the former CEO of Cassava Sciences.
Despite overwhelming evidence of the futility of Cassava’s Alzheimer’s drug candidate, Barbier continued to spin data, mislead investors, and exploit the desperation of Alzheimer’s, Adam writes. This year, Barbier was penalized by the SEC, and he remains a defendant in ongoing shareholder and class-action lawsuits.
A key decision on compounding is expected this week
There’s been a lot of uncertainty in the GLP-1 compounding space over the past few months. To refresh: In early October, the FDA pulled Eli Lilly’s tirzepatide off its drug shortage list after nearly two years. That should have prohibited compounding pharmacies from continuing to making copies of the drug, as they’re allowed to do so only when a treatment is on the shortage list. But after a compounding trade group took the FDA to court, the agency made a sudden about-face, saying it would reconsider its decision and allow compounders to continue for the meantime.
The FDA and the compounding trade group are meant to provide updates in court on or before this Thursday. The agency may decide that tirzepatide should stay off the shortage list, which would be a significant blow to compounders and the telehealth companies they work with. Or, the FDA may decide that tirzepatide should stay on the shortage list and that compounders can continue making it — a move that would rile Eli Lilly, whose CEO has raised concerns that the growth of compounding could create a “backdoor generic world.”
Whatever the FDA does in the situation would set a precedent for how it handles Novo Nordisk’s semaglutide, which may be close to coming off the shortage list soon.
In the meantime, telehealth companies that prescribe compounded tirzepatide appear to be using the deadline to urge patients to stock up. One of them, Pomegranate Health, sent an email to users yesterday, saying, “The decision to uphold the removal of tirzepatide from the shortage list may be overturned or upheld on December 19, 2024. Either way, this could lead to a surge in demand for the medication as providers and patients scramble to stock up on supply. Secure your three month supply to avoid disruption to your care.”
Correction: Monday’s newsletter incorrectly stated the revenue of Amgen’s drug Tepezza. It’s on track to do $2 billion in sales, not $1 billion.