One Firm, Two Conflicting Takes on Alnylam's HELIOS-B
Good morning. STAT has just released its annual list of top books and podcasts for this summer with recommendations from notable figures like Robert Califf and Rochelle Walensky, as well as our staff and readers.
Let's dive into today's key stories, including the intriguing HELIOS-B data announced yesterday.
Early Data on Huntington’s Treatment
Wave announced early data for an experimental Huntington’s treatment which, unlike previous approaches, targets only the mutant protein.
Artificial Intelligence in Protein Drug Design
Former Meta scientists have raised $142 million to create a new company that designs protein drugs using AI.
AstraZeneca's Imfinzi Shows Promise
AstraZeneca reported that its drug Imfinzi has improved overall survival for certain bladder cancer patients.
Will Zepbound Hamper CPAP Sales? Experts Are Divided
Last Friday, we received the full results of the Phase 3 trial testing Lilly’s obesity drug Zepbound in sleep apnea patients. Detailed findings support the benefits of the treatment in this common disease. Following the news, shares of CPAP maker ResMed and nerve stimulator Inspire dropped.
Expert Opinion Divided:
- Some believe Zepbound is more convenient than a CPAP device, offering a range of health benefits.
- Others argue that CPAP machines provide immediate relief to patients.
From Skeptic to CEO of a Gene Editing Company
Tal Zaks, former chief medical officer at Moderna, who was once a critic of gene editing, is now the CEO of a new gene editing company called Exsilio Therapeutics. The startup has raised $82 million in Series A funding, with Zaks citing their ability to insert whole genes with minimal disruption to human DNA as a key innovation.
Grail Debuts on Nasdaq With Cancer Screening Test
Grail, known for its liquid biopsy technology Galleri, debuted on the Nasdaq today. The company faces several challenges in obtaining approval and achieving widespread adoption of its cancer screening tests.
My colleague, Matt Herper, sat down with Grail executives to discuss the data that doctors and regulators seek and the hurdles in getting reimbursements for liquid biopsies.
Ketamine Tablet Shows Potential in Treating Depression
In a Phase 2 trial for treatment-resistant depression, participants taking the highest dose of a ketamine tablet showed significant improvement in depressive symptoms compared to a placebo. This trial, sponsored by New Zealand-based Douglas Pharmaceuticals, is part of ongoing research into the use of psychedelic drugs in treating mental health disorders.
One Firm, Two Conflicting Takes on HELIOS-B
Alnylam recently announced results for its ATTR-CM drug vutrisiran from the pivotal HELIOS-B study. Industry watchers are comparing its efficacy against Pfizer and BridgeBio's treatments. Analysts within the same firm provided conflicting views based on the limited data:
- Ritu Baral from TD Cowen called the results "best case scenario," emphasizing statistically significant primary outcomes and larger-than-expected effect sizes. Baral believes vutrisiran will emerge as a first-line therapy for severe patients.
- Meanwhile, Tyler Van Buren from TD Cowen sees vutrisiran as a second-line treatment. He pointed out that BridgeBio's drug showed a greater effect in its trial, with a 42% reduction in the risk of cumulative death and cardiovascular hospitalizations.
As the industry dissects Alnylam's data, eyes are set on the full presentation later this year for more detailed insights.
More Reads:
- Ginkgo job cuts to impact at least 35% of the workforce, about 400 jobs.
- Amylyx licenses a GLP-1 drug in search of new opportunities post-ALS.
- The WHO warns that the global health toll of alcohol remains 'unacceptably high.'
- The Surgeon General declares gun violence a public health crisis.
