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Hey hey, this is Meghana. Today, we talk about major restructuring at Editas Medicine, see a new commercialization strategy from Ionis, and learn what people in Trump’s orbit think of GLP-1 drugs.
The need-to-know this morning
- Advisers to the European Medicines Agency recommended the approval of BridgeBio’s heart drug acoramidis. The drug will be marketed there by Bayer under the brand name Beyonttra.
- EMA advisers also recommended the approval of a drug for myelodysplastic syndrome from Geron and a kidney cancer drug from Merck.
Editas Medicine lays off two-thirds of its staff
Editas Medicine is laying off 65% of its workforce, and is shelving its lead ex vivo gene-editing treatment for sickle cell disease, Reni-Cel — even though early data suggested it might be able to outperform Vertex’s approved therapy. STAT’s Jason Mast reports that the company is pivoting now to in vivo gene editing, which avoids the intensive chemotherapy needed for ex vivo treatments. The plan, the company says, is to develop safer and more scalable therapies.
This restructuring reflects broader challenges in the gene-editing field — such as financial pressures, competition, and the high costs of existing treatments.
Ionis will start commercializing its drugs in-house
Ionis Pharmaceuticals typically has partnered with other companies when commercializing its antisense oligonucleotide therapies. These days, however, the company is looking to maximize profit by transitioning to a more independent sales approach, as STAT’s Jonathan Wosen and Matt Herper report. That’ll start with its drug olezarsen, which it’s developing for familial chylomicronemia syndrome, a rare disease.
Although Ionis has had its share of setbacks, successes like Spinraza for spinal muscular atrophy have cemented its credibility. But it’s a gamble to commercialize drugs fully in-house.
“There’s risk, and the risk is real, that we can damage the great science that we’ve done here for 30 years,” CEO Brett Monia said. “We have to take that risk and manage that in favor of getting our treatments to patients, to the health care community, because they’re not getting there.”
GLP-1 drugs spark disagreements in Trump’s circle
From STAT’s Elaine Chen: GLP-1 drugs seem to be a source of contention among the people in President-elect Trump’s circle.
Earlier this week, Elon Musk tweeted: “Nothing would do more to improve the health, lifespan and quality of life for Americans than making GLP inhibitors super low cost to the public.” It’s a sentiment he’s expressed several times before. (The drugs are not actually inhibitors; they’re agonists.)
Then, Calley Means, an entrepreneur who was critical in birthing the MAHA movement, responded, saying that Musk’s post “misses the spiritual crisis we are facing.” Means said the drugs should only be a small part of the effort to address chronic disease rates, and he claimed that Novo Nordisk “has been able to pay US regulators, media, and lawmakers to force this drug down our throats as the only option.”
It’s yet to be seen how the Trump administration will actually approach potential policies around GLP-1 drugs. Axios recently reported that the CEO of Eli Lilly, maker of Mounjaro and Zepbound, went to Mar-a-Lago to meet with Trump.
And what does RFK Jr., Trump’s pick for HHS secretary and nominal leader of the MAHA movement, think of GLP-1s? Before the election, he issued a post on X that was quite critical of the drugs, noting that they have potential side effects and suggesting that there should be a greater priority on addressing the root causes of obesity. “We should consider replanting the kitchen garden before sending more money to Denmark,” he said, referring to Novo Nordisk’s headquarters.
Lately, though, it seems his tone on GLP-1s has softened. When asked very briefly by CNBC’s Jim Cramer about his thoughts on the treatments, he said that making lifestyle changes is most important, but GLP-1 drugs “have a place.”
In general, cracks have started to form in the MAHA coalition as the Trump administration announces nominations. My colleague Isa Cueto dug into that here.
What do drugmakers think of RFK Jr.?
Why has it been so hard to bring gene therapies to market? And what tacos did Adam have this week while attending the American Society of Hematology conference?
We discuss all that and more on this week’s episode of “The Readout LOUD,” STAT’s biotech podcast. Washington correspondent John Wilkerson joins us to discuss why drugmakers have not lobbied against Robert F. Kennedy Jr.’s nomination to lead the Department of Health and Human Services, despite his repeated criticisms of the pharmaceutical industry.
Padlock investors suing Bristol Myers Squibb
Investors in Padlock Therapeutics are suing Bristol Myers Squibb, alleging that the pharma giant manipulated patents and withheld information to avoid some $450 million in milestone payments after it acquired the startup in 2016. The investors include Atlas Venture and its founding chief executive Michael Gilman, and claim BMS used a “deceptive scheme involving manipulation of the patent process,” according to the lawsuit.
The case shows the dark side of “biobucks” deals, as STAT’s Adam Feuerstein writes. Although BMS advanced one of the antibody drugs developed by Padlock to the clinic, the promised milestone dollars have yet to be shared with investors.
More reads
- HHMI’s move to diversify funding sparks debate about ‘biomedical elite,’ STAT
- Tenpoint, Visus merge in effort to push presbyopia-correcting eye drop over FDA finish line, FierceBiotech