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Welcome back to another edition of Endpoints Weekly! We’re doing a little bit of celebrating here at Endpoints, as we took home three Neal Awards, which recognize excellence in B2B journalism. The winning entries came from Max Bayer’s government, legislative and regulatory coverage, Ryan Cross’ series on CRISPR, and our JP Morgan healthcare conference event, which was led by Nicole Wetsman and our events team. We also had two other finalists — Max’s pharma "frequent fliers" story and our coverage of China’s biotech industry. Congrats to all, and have a great
weekend! — Max Gelman |
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Max Gelman |
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Senior Editor, Endpoints News |
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Sanofi’s immunology reckoning |
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📊Sanofi reported its first-quarter earnings this week, and new CEO Belén Garijo starts next week. But the company will likely face a reckoning on its immunology strategy before she even begins, given the mixed track record in the clinic under predecessor Paul Hudson. It probably doesn’t make sense for Garijo to continue down the same path, Jefferies managing director Michael Leuchten said in an interview. “I think
that’s off the table,” he said.
The Hudson strategy homed in on a dozen potential blockbuster immunology drugs, three of which were projected in late 2023 to reach at least $5 billion in annual peak sales. One succeeded in a Phase 2 study for multiple sclerosis and now has pivotal trials underway, but the second brought new safety concerns and the third flunked multiple mid-stage tests.
Sanofi also held a call with reporters before Thursday’s earnings event, where executives defended the Garijo appointment and said the company is “fully committed” to immunology. The call was an exercise in redirecting the narrative, Ayisha Sharma writes.
Elsewhere in earnings news, Roche also recapped its first quarter, with CEO Thomas Schinecker making bold predictions about its experimental breast cancer pill giredestrant. Schinecker said it could reach three times as many patients as blockbuster Herceptin, which at its peak in 2018 had sales of about $7 billion. Read more on Roche here from Elizabeth Cairns. |
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Lilly acquires in vivo CAR-T biotech |
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💰Eli Lilly is going big on in vivo CAR-T, Elizabeth Cairns wrote this week. The company struck a deal worth up to $7 billion to buy Kelonia Therapeutics, which has a Phase 1 program for multiple myeloma and a couple of preclinical candidates. The deal includes $3.25 billion upfront.
The deal is Lilly’s second for an in vivo CAR-T biotech, following its February bid for Orna Therapeutics. Competition is heating up, as AbbVie, AstraZeneca, Bristol Myers Squibb and Gilead all acquired in vivo cell therapy startups last year.
It’s still early days for the approach. BMO Capital Markets analysts cautioned that in vivo CAR-T remains “an unproven and risky area of development.” Traditional CAR-Ts involve removing T cells from a patient, altering their genetic makeup and reinfusing them back into the patient. Kelonia’s lead program KLN-1010 works by generating anti-BCMA CAR-Ts within the patient. Early data from a first-in-human study presented at last year’s ASH meeting showed that a single infusion eliminated detectable cancer cells in four heavily refractory multiple myeloma patients.
Kelonia’s investors are probably happy. Unlike many startups that have raised multiple $100 million-plus rounds, Kelonia only raised a total of $60 million in private capital. One lead investor, Venrock, put in $20 million and will make $900 million back on the upfront payment. |
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RevMed’s "Herceptin moment" |
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🥼Revolution Medicines’ pancreatic cancer data were all the talk at the annual American Association for Cancer Research conference this week. The stunning results, which showed its drug daraxonrasib doubled the survival time of second-line patients, could be a “Herceptin moment” for pancreatic cancer, Stifel managing director Tim Opler told Endpoints.
Before Herceptin’s approval, chemotherapy was often the only treatment for breast cancer. The first targeted cancer therapy, Herceptin extended survival by three months. Now, almost three decades later, many patients respond to targeted breast cancer therapies for multiple years. The hope is that something similar happens in pancreatic cancer. “It reinvigorates the whole field,” said Daniel Von Hoff of the Translational Genomics Research Institute and City of Hope.
Also at the conference, KRAS drugmakers are taking another, better swing at lung cancer, reporting early-stage data for the next generation of drugs. Read all about it from Lei Lei Wu, who was on the ground in San Diego, here. |
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Flagship’s new gene therapy startup |
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🧬 Serif Biomedicines unveiled this week with $50 million and a bold mission: delivering DNA into the body without the aid of an engineered virus. Swapping viruses for other delivery vessels like lipid nanoparticles could lead to safer and cheaper treatments, Ryan Cross describes in his story. It’s no small challenge: Several other startups have tried and failed to get the idea to work. But Serif
has been working on its technology since 2021, and it thinks it could be close.
Serif is calling the technology “modified DNA,” which CEO Jacob Rubens said is “meant to draw a direct analogy to modified RNA.” Viruses are a natural choice for sneaking genes into cells, in part because the viral shell helps shield the DNA itself from the immune sensors that reside within cells. Serif’s solution was to tweak DNA’s shape and chemical structure until it found versions of the molecule that didn’t trigger those alarm bells.
Rubens didn’t give much detail on how long the company’s current funding will last or where it will take Serif. Ryan spells out all the information we have for now here. |
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Regeneron signs MFN deal |
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🤝Regeneron agreed to a “most favored nation” deal with the White House, marking the last of the 17 drugmakers that President Donald Trump singled out in letters last summer to make a deal. HHS chief counselor and head of Medicare Chris Klomp said there’s more work for the administration to do, adding that it’s negotiating “with the many hundreds of biotechnology and
pharmaceutical companies” that were not among those initially singled out.
Regeneron also announced that it won approval for its first commercial gene therapy. The drug, called Otarmeni, was approved under the Commissioner’s National Priority Voucher program to treat a type of inherited hearing loss caused by mutations to a gene called otoferlin. The company said it plans to offer the treatment for free in the US, though that does not include the cost of administration. |
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