| |   | Good morning, all. Damian here with the surprising success of a long-maligned heart drug, a new use for a blockbuster rare disease medicine, and a "landmark" victory for cell therapy. | | | Esperion’s umpteenth chance Esperion Therapeutics is a small heart drug developer that, in its history, has been eaten and spit out by Pfizer and has known the triumph of getting a new drug approved and the failure of it just not selling. Now, it might be getting yet another another chance.
Yesterday, the company said that its cholesterol medicine, Nexletol, reduced the rate of heart problems — a composite measure of stent procedures, heart attacks, and strokes among patients who attested that they could not take a statin, the first-line drugs for people at risk of heart problems, because of side effects. No specific data were provided in the press release.
The question now is whether insurers, after seeing the full data released, will want to start covering the medicine for such patients in the real world. There’s also a question as to whether Esperion, which has a fairly distressed balance sheet, can survive. Despite the apparently good news, the firm’s stock fell almost 15%. | How Vertex’s CF drugs could treat deadly pneumonia Vertex Pharmaceuticals’ succession of treatments for cystic fibrosis have transformed how the rare disease is treated. Now, there’s emerging evidence that those same drugs could help with a much more common lung disease: severe pneumonia.
As STAT’s Jason Mast reports, researchers in Germany found that Kalydeco, one of Vertex’s cystic fibrosis treatments, led to dramatic improvements when given mice in the early stages of pneumonia-induced acute respiratory distress syndrome, or ARDS. The condition, which killed around 200,000 people each year in the U.S. before the pandemic, became frighteningly prevalent after Covid-19 induced it in millions worldwide.
The study, published yesterday in Science Translational Medicine, suggests Kalydeco’s biological target, a mutation to the CFTR gene, could play a role in the lung damage caused by ARDS. And while promise in mice is hardly a guarantee of clinical success, the researchers are hopeful they can replicate those results in a small human study.
Read more. | STAT Digital Event: The ASH Recap, Live! Register for our virtual recap of the biggest event in hematology research. On Dec. 15, STAT’s Angus Chen and Damian Garde will host a discussion with hematology experts about the most impactful data and breakthroughs related to blood diseases, and what to expect from the field in the years ahead. | A long-in-the-making cancer therapy succeeds in ‘landmark’ study A cell therapy whose development dates back to the 1980s worked better than approved immunotherapy in a study enrolling patients with advanced skin cancer, a milestone built on decades of research.
As STAT’s Angus Chen reports, scientists enrolled patients whose melanoma had spread despite available treatments and randomized them to receive either the immunotherapy Yervoy or a treatment with tumor infiltrating lymphocytes, or TILs. Patients who received TIL therapy had an average progression-free survival more than double that of the Yervoy group, and the rates of response and complete remission were both significantly greater.
“This is really a landmark study because it’s the first randomized Phase 3 study for cell therapy in solid tumors,” said George Coukos, an immunotherapy physician-scientist who was not involved in the trial. TILs are created by surgically removing patients’ tumors, isolating the immune cells that have managed to evade the cancer’s defenses, and then turning them into an infusable medicine.
Read more. | Prometheus’ inflammatory bowel drug changes the narrative Prometheus Biosciences said its investigational medicine for inflammatory bowel disease met its goals in two mid-stage studies, roughly doubling the company’s stock price and lending weight to what had been a controversial scientific idea.
The drug, PRA023, significantly reduced symptoms of ulcerative colitis in a placebo-controlled study and met its goals in a separate, open-label trial enrolling patients with Crohn’s disease. The results will inform Phase 3 trials slated to begin next year, the company said.
Beyond Prometheus’ ambitions, PRA023’s incremental success is a vote of confidence that targeting TL1A, a bodily protein that drives inflammation, could be a viable way to treat ulcerative colitis, Crohn’s, and other inflammatory diseases. Earlier this month, Pfizer out-licensed its competing TL1A antibody to Roivant, seeding speculation that the company had lost faith in its potential.
Read more. | More reads - Congress has a chance to close the FDA’s Theranos loophole, STAT
- How AI that powers chatbots and search queries could discover new drugs, Wall Street Journal
- ‘I question what I know’: Top cancer specialist Otis Brawley on valuing orthodoxy in medicine, STAT
| Thanks for reading! Until tomorrow,  | | | |
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