| |   | Hope y’all had a wondrous elongated weekend, celebrating hot dogs and flags and whatnot. On tap today: CRISPR stuff, VC stuff, and a scorecard.
- Meghana | | | CRISPR is working. But do we know enough? After years of anticipation, we now have seen encouraging results for CRISPR-based treatments. But some scientists believe we still don't know as much as we should about long-term risks. They note that Vertex, which is developing a CRISPR-based treatment for sickle cell disease and beta thalassemia, has released data on more than 70 clinical trial participants, showing that its therapy appears to be a functional cure. But the company hasn't released certain information on how the therapy is impacting the DNA of patients in the trials.
Such data, they argue, could ultimately be critical in understanding long-term adverse effects that might only show up years down the road, after the therapy has been widely used. “I don’t think they’ve been irresponsible,” one gene editing expert told STAT, speaking of Vertex. “But I think if they continued to push forward and ignore this, that would be irresponsible.” Read more. | The biotech downturn is spooking VCs The ongoing market downturn in biopharma is now impacting privately held startups, drumming up anxiety among executives and investors alike.
In some cases, private companies that had been planning IPOs are now desperately seeking out new pathways to financing. In others, they're being asked to raise funding based on prior valuations, or even lower ones — something that was basically unthinkable just last year. “Most of the conversations I’m having are with folks who are really scared and frightened,” one investor and CEO said. Read more. | Nominate a talented young scientist to be named a STAT Wunderkind STAT is on the lookout for the most impressive young minds in science to give them the recognition they deserve. Nominate someone by July 29. | A biotech score card for the third quarter Time for STAT’s quarterly biotech scorecard! In Q3’s installment of stock-moving events, there’ll be a critical readout from Alnylam. Its APOLLO-B Phase 3 study will demonstrate whether the RNA-interference medicine Onpattro can be used for a broader swath of patients with a rare form of cardiomyopathy. Data from a Phase 3 study of a new schizophrenia treatment from Karuna Therapeutics is also forthcoming. Should it be approved, that could ultimately generate billions of dollars in sales. Other companies with impactful news on the horizon include Arrowhead Pharmaceuticals, Takeda Pharmaceuticals, and Iveric Bio. Read more. | Biogen’s next Alzheimer’s drug is in a curious situation Among the many lessons of Aduhelm is that physicians, insurance companies, and Medicare have little interest in an Alzheimer’s disease therapy that has proven only to reduce the amount of amyloid plaques in patients’ brains. And with that in mind, last night’s news that lecanemab, the latest Alzheimer’s therapy from partners Biogen and Eisai, could win early FDA approval in January is unlikely to change the narrative in amyloid.
According to the companies, the FDA accepted their application for accelerated approval, which would clear lecanemab based on its ability to reduce amyloid, under the assumption that doing so slows the cognitive decline associated with Alzheimer’s. The FDA will make a decision by Jan. 6, Biogen and Eisai said.
To analysts, the prospect of accelerated approval is essentially meaningless if lecanemab doesn’t succeed in an ongoing Phase 3 study testing whether the treatment actually improves patients’ lives compared to placebo. What’s notable is that the companies expect data from that trial this fall. That means the FDA and its advisers will almost certainly know at least the top-line results of that study while considering whether to grant approval based on amyloid reduction, setting the stage for a potentially fraught process. | Amylyx’s ALS drug up for a second adcomm The FDA is convening a second advisory committee to determine whether an ALS treatment made by Amylyx Pharmaceuticals is worthy of approval. It's an unusual move — suggesting that regulators are torn over whether to grant accelerated approval for the experimental therapy, called AMX0035. The meeting has been scheduled for Sept. 7. The first expert panel was in March, during which the FDA raised concerns about the treatment’s efficacy — saying Amylyx’s data wasn’t statistically persuasive enough. The panelists were split over whether the drug should be conditionally approved, even with inconclusive data.
Canada, meanwhile, has already approved the drug, which will be sold under the brand name Albrioza. Read more. | More reads - Anebulo Pharma says study supports development of its pill as an antidote for cannabis intoxication. STAT
- TeneoTwo, put in play by Amgen, lands $100M AstraZeneca buyout on back of blood cancer bispecific. FierceBiotech
| Thanks for reading! Until tomorrow,  | | | |
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