| | | |   | Hello, everyone. Damian here with some long-awaited mRNA news, analysis of Amgen's big deal, and an update on monkey smuggling. | | | Mirati wins FDA approval, but expectations have dimmed Mirati Therapeutics’ drug for hard-to-treat lung cancer won FDA approval last night, but long-held hopes that it might reach a large swath of patients have dimmed in the wake of disappointing clinical trial results.
As STAT’s Adam Feuerstein reports, the FDA approved Mirati’s drug, a pill called Krazati, for patients with advanced non-small cell lung cancer containing a specific mutation to the gene KRAS, one found in about 13% of lung tumors. Krazati is the second approved treatment aimed at KRAS, a target once deemed undruggable, after Amgen’s Lumakras, which won approval in 2021.
Mirati’s approval clears the drug for the relatively small number of people whose lung cancer has stopped responding to previous treatments. The ideal future for Krazati — and for Mirati’s business — relies on proving that the drug can benefit the larger number of patients with earlier-stage disease, but a key clinical trial combining the medicine with Keytruda, a blockbuster immunotherapy, suggested the regimen was relatively safe but with an overall response rate was underwhelming.
Read more. | Amgen’s $30 billion bet is a strategic pivot Amgen is paying nearly $30 billion for Horizon Pharmaceuticals, the companies said yesterday, embracing the lucrative business of selling expensive drugs for rare diseases after years on the sidelines.
The deal, which is the largest pharma merger announced this year, is Amgen’s biggest acquisition in two decades. At the center is Tepezza, Horizon’s treatment for moderate to severe thyroid eye disease, which affects about 10,000 patients in the U.S. The drug, which costs upward of $200,000 per year, is expected to generate annual revenues in excess of $2 billion.
Amgen’s purchase marks the company’s first major entrance into the field of rare disease drugs, years after many of its peers bought into the burgeoning business. The acquisition is expected to strengthen Amgen’s revenues as its top-selling medicines, including the arthritis treatment Enbrel and psoriasis drug Otezla, face mounting competition.
Read more. | The power of observation Microscopy can reveal how immune cells kill tumor cells and how cancer evades our normal defenses. Unraveling these complex relationships has revolutionized cancer care through the development of treatments like immunotherapies, which boost the immune system’s ability to fight cancer. Take a journey with us and see what Genentech researchers see through sophisticated microscopes as they carefully observe cellular activity to reveal how the immune system interacts with cancer. | Vertex inches closer to a Holy Grail There’s a compelling slide in Vertex Pharmaceuticals’ investor presentations, updated over the years to show how each of its successive medicines came to help more and more people with cystic fibrosis. But there’s always a gap: About 10% of patients have genetic mutations that make Vertex’s treatments unusable. For them, the only option would be a drug that can fully replace the key proteins their bodies don’t make, something that has eluded scientists for decades.
Yesterday, Vertex took a step toward changing that. After about six years of preclinical work, Vertex and partner Moderna are moving into human trials with an mRNA therapy that might finally work for the 10% of patients without approved treatments. Like Moderna’s Covid-19 vaccine, it relies on encasing strands of mRNA in lipid nanoparticles. But instead of getting injected into muscle tissue, Vertex and Moderna have formulated the drug to be nebulized directly into the lungs, where the mRNA can make its way into cells and compel the body to produce the missing CFTR protein.
Vertex said it will start a Phase 1 trial in the coming weeks and eventually test multiple doses to inform further development. Analysts expect preliminary data some time next year. | And now some monkey-smuggling news A poaching scandal in Cambodia threatens to dramatically limit the supply of research primates that are integral to preclinical drug development, a situation that could imperil progress at a wide array of companies and institutions.
At issue, STAT’s Ed Silverman reports, are long-tail macaques, which are the non-human primates of choice for corporate, government, and academic research laboratories around the U.S. In Cambodia, from which roughly 60% of research primates are imported, local authorities have halted exports following a string of indictments accusing suppliers of poaching wild macaques, which are protected by international law, and passing them off as research animals bred in captivity.
That breeds “tremendous uncertainty” for researchers and the many companies that sell animal models to industry and academia, Baird analyst Eric Coldwell wrote in a note to clients. “We do not know how long current inventories may last or what will happen to pricing on non-human primate studies,” Coldwell wrote, “given the dramatic shortage expected in an already tight market.”
Read more. | More reads - ‘Out of control’: Dozens of telehealth startups send sensitive health information to big tech companies, STAT
- Billionaire John Paulson stands to make $500 million from Horizon’s sale to Amgen, Forbes
- Roche pharmaceuticals CEO Bill Anderson to step down after four-year tenure, STAT
- Clovis finally throws in the towel, files for bankruptcy, FiercePharma
| Thanks for reading! Until tomorrow,  | | | |
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