| | | | | Meghana here. We're seeing competition ramp up at EQRx, delve into why therapeutic technique matters more than brain chemistry with psychedelics, and more. Have a nice weekend! | | | Two children died after being treated with Zolgensma Two children have died from acute liver failure after receiving a one-time infusion of the Novartis gene therapy Zolgensma. The children, who lived in Russia and Kazakhstan, were being treated for spinal muscular atrophy, a genetic disorder that causes muscle weakness and wasting. They passed away about a month and a half after receiving the drug, and between one and 10 days after corticosteroid tapering happened, which is done to maintain liver function. Although acute liver failure is listed as a possible toxicity in the therapy’s labeling, these are the first fatal cases for Zolgensma that can be attributed to that side effect. There were no deaths tied to acute liver failure in clinical trials. The drug, which costs $2.1 million, was approved three years ago. At the time, the FDA called it a “milestone in the transformational power of gene and cell therapies to treat a wide range of diseases.” The company will mention the potential for fatal acute liver failure in the labeling moving forward, STAT reports. Read more. | Are biotech’s fortunes finally changing? Is Big Science impeding actual science? And what will Medicare negotiation mean for drug prices? We cover all that and more this week on “The Readout LOUD,” STAT’s biotech podcast. STAT’s Jason Mast joins us to discuss Jim Wilson, a pioneering scientist synonymous with the rise of gene therapy who staffers say presided over a toxic, abusive workplace. We also explain the latest news in the life sciences, including a multibillion-dollar deal, a novel approach to treating schizophrenia, and what the Senate drug pricing legislation means for the pharmaceutical industry. Listen here. | EQRx pitting its cancer drug against Tagrisso EQRx, a Cambridge-based pharma company with the mission statement of developing drugs at “radically lower prices,” is launching a Phase 3 trial to test its experimental lung cancer drug aumolertinib against AstraZeneca’s blockbuster, Tagrisso. Both drugs target the protein EGFR to stop tumor cell growth, BioPharma Dive writes. For oncologists, Tagrisso has become a go-to for lung cancer — with sales in just the first half this year a stunning $2.7 billion. So EQRx is planning, with this new study, to see how aumolertinib might perform compared to standard of care for non-small cell lung cancer. Despite pushing forward with its aims to compete head-to-head with costly top-selling drugs, EQRx’s shares fell 17% Thursday — thanks to increased expenses and a wider net loss in its second quarter, per MarketWatch. | Preparing for the future of mRNA in the need-for-speed era mRNA got its spot in the limelight in the midst of a global pandemic, but it also holds great potential in moving us closer to the reality of personalized medicine. That opportunity, of course, will come with considerations for how the biopharma industry plans for the future. Hear from Linda Matthiason, Strategic Customer Lead, Nucleic Acid Therapeutics at Cytiva, who will highlight some of the unique hurdles in advancing future mRNA therapeutics. Read the article here. | It’s the therapy part of psychedelics that matters Lysergic acid. Psilocybin. Peyote. DMT. There are many psychedelic substances being explored in a therapeutic context, and their chemical composition and mechanism of action are important to probe. But even more critical is how they are used: “What matters for therapy and healing is how the experience is processed when the drug wears off,” opines Michael Pollack, who works as a venture capitalist and philanthropist in the therapeutic psychedelics space. The difference between a euphoric moment at a rave and a profound psychological shift comes down to the way a therapist guides a person through a psychedelic experience, he writes. As compelling as the history of these drugs may be, or the shifts in brain chemistry, these time-tested substances need to be supported by “a robust research agenda to create a therapeutic framework that embraces the truth,” says Pollack. Read more. | Lip service from Eli Lilly Indianapolis-based Eli Lilly recently expressed concern that the state’s new abortion ban would “hinder” its ability to attract “diverse scientific, engineering, and business talent.” The company responded to the legislation by saying that it might have to expand outside of Indiana. But that message sounds just a touch disingenuous, STAT’s Ed Silverman writes. Lilly has supported Indiana’s Republican lawmakers who supported the law — giving more than $3 million to committees that supported the state’s top proponents of the abortion bill. “If they really believed in protecting abortion rights and the welfare of women, they had other options,” an Indiana University management researcher said. “They could have lobbied or indicated they would cut off contributions to lawmakers, but they weren’t that aggressive.” Read more. | More reads - Band of biotechs cut pipeline prospects to focus cash on priority programs, FierceBiotech
- More than half a billion Covid vaccine doses wasted after Emergent’s Baltimore fiasco, Endpoints
- FDA needs to fully review ACAM2000 before allowing its widespread use as a monkeypox vaccine, STAT
| Thanks for reading! Until next week, | | | |
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