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Good morning. Today, STAT published our annual list of the top books and podcasts to check out this summer. Take a look — we’ve sourced recommendations from Robert Califf, Rochelle Walensky, and also our staff and readers.
Now, let’s get into the news of the day, and then some discussion on the HELIOS-B data that was announced yesterday.
The need-to-know this morning
- Wave announced early data for an experimental Huntington’s treatment, designed — unlike previous approaches — to target only mutant protein.
- Ex-Meta scientists raised $142 million for a new company to design new protein drugs with AI.
- AstraZeneca said Imfinzi improved overall survival in certain bladder cancer patients.
Will Zepbound hamper CPAP sales? Experts are divided
Late last Friday, we got full results of the Phase 3 trial testing Lilly’s obesity drug Zepbound in sleep apnea patients. The detailed findings further support the benefits of the treatment in this increasingly common disease. As a result, shares of CPAP maker ResMed and nerve stimulator Inspire dropped yesterday.
But when colleague Lizzy Lawrence and I asked some experts how much Zepbound would actually affect sleep device sales, we got mixed responses. On one hand, Zepbound may be seen as more convenient than a device, and patients may also opt for it since it offers a range of health benefits. On the other hand, CPAP machines can provide immediate relief to patients.
From skeptic to CEO of a new gene editing company
Tal Zaks, former chief medical officer at Moderna, used to routinely bash gene editing. But today, he announced that he’s now the chief executive of a new company called Exsilio Therapeutics that’s focused on, of all things, gene editing.
The startup emerged from stealth with $82 million in Series A funding and a seed round led by Orbimed, where Zaks has been a partner. Zaks has not disclosed much about what the company is doing, but said it has technology that could allow researchers to insert whole genes with minimal disruption to a patient’s DNA.
Read more from STAT’s Jason Mast.
A catchup with Grail as it debuts on the Nasdaq
As liquid biopsy company Grail debuts on the Nasdaq today, it faces a slew of challenges in getting approval and ultimately wide adoption of its cancer screening test called Galleri.
My colleague Matt Herper sat down with Grail executives to chat about the data that doctors and regulators will want to see on its test, as well as hurdles with getting reimbursement for liquid biopsies.
Ketamine tablet show potential in treating depression
In a Phase 2 trial of people with treatment-resistant depression, those taking the highest dose of a ketamine tablet experienced a statistically significant and clinically meaningful improvement in depressive symptoms compared to placebo.
The trial, sponsored by New Zealand-based Douglas Pharmaceuticals, is the latest attempt by researchers to study psychedelic drugs’ role in treating mental health disorders. This comes after an FDA advisory panel earlier this month voted against an MDMA-based treatment for PTSD.
Read more from STAT’s Mohana Ravindranath.
One firm, two conflicting takes on HELIOS-B
After Alnylam yesterday announced results of its ATTR-CM drug in the pivotal HELIOS-B study, industry watchers immediately tried to determine how the company’s drug, called vutrisiran, stacks up against an approved treatment from Pfizer and a late-stage candidate from BridgeBio. But if it’s any indication of how difficult that is to do right now with the limited data, see the conflicting takes written by two analysts from the same firm:
TD Cowen analyst Ritu Baral, who covers Alnylam, wrote that these were the “best case scenario” results, noting that the primary outcomes were statistically significant and the effect sizes exceeded average investor expectations.
In HELIOS-B, some participants were already taking Pfizer’s treatment, and a big question is how much added benefit vutrisiran adds on top of that background therapy. While Alnylam didn’t report that data, Baral said that based on commentary from executives, she believes the full data will show that vutrisiran has an added benefit. Ultimately, the data support use of vutrisiran as a first-line therapy in severe patients, she said.
Meanwhile, TD Cowen analyst Tyler Van Buren, who covers BridgeBio, wrote that he sees vutrisiran, a “silencer” drug, as a second-line treatment behind “stabilizer” drugs, such as Pfizer’s and BridgeBio’s treatments.
On the primary outcome in HELIOS-B, vutrisiran cut the risk of death and recurrent cardiovascular events by 28% in the overall trial population and by 33% in the group of patients who weren’t on background therapy. Van Buren noted that BridgeBio’s drug appeared to have a greater effect in its trial, with a 42% risk reduction on cumulative death and cardiovascular hospitalizations over a shorter time period studied. (Note, though, that BridgeBio’s trial was designed differently, and the 42% risk reduction was not the primary outcome. This number was not part of the journal publication of the results; it’s part of a subsequent analysis recently communicated by BridgeBio.)
As analysts continue to pick apart the few pieces of data in Alnylam’s topline announcement, all eyes will be on the full presentation later this year. Observers want more details on when how quickly benefits started to emerge in HELIOS-B and what the efficacy looked like across subgroups.
More reads
- Ginkgo job cuts to hit at least 35% of workforce; as many as 400 jobs to be cut, Boston Business Journal
- Amylyx, searching for post-ALS future, licenses a GLP-1 drug, Endpoints
- WHO warns global health toll of alcohol remains ‘unacceptably high’, STAT
- Surgeon general declares gun violence a public health crisis, STAT
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- Source: https://www.statnews.com/2024/06/25/biotech-news-alnylam-wave-astrazeneca-el-lilly-zepbound-moderna-grail-nasdaq/?utm_campaign=rss