Jacob Bell

In case you missed it, our latest feature takes a deep dive into a very interesting area of drug research: ion channels. These proteins made $VRTX one of the world's wealthiest biotechs. Now others are getting in on the action. #biotech #science #biopharma biopharmadive.com/news/ion-chann…

Read this Deep Dive for @realJacobBell 's excellent scientific and business reporting, and for one of the best outro quotes I've read in awhile.

Biotech M&A on track to have 2nd most active year in history w/ more early stage deals. Nice summary of which funds have benefited from recent M&A from Stifel @TimOpler $XBI $IBB

RBC: Revolution, Xenon, Arrowhead among top #biotech takeover targets biopharmadive.com/news/biotech-t… by @realJacobBell $ARWR $XENE $RVMD $DYNE

NEW FDA vaccines chief Vinay Prasad will leave the agency at the end of April Gift link below

This is an incredible ending for a therapy I've followed for years. If you're interested in the backstory, here's a feature from 2020, when the FDA issued an astonishing rejection to Roctavian's approval application. biopharmadive.com/news/hemophili…

BioMarin has chosen to pull its once highly anticipated gene therapy for hemophilia from the market. The therapy struggled commercially, which led $BMRN to search for a potential buyer. In the company's latest earnings, it reveals it couldn't find one.

$NBIX "has the scarcity value of being one of only several biotech companies whose ~$3B in existing commercial revenue is expected to grow by more than 50% by 2030 and be durable well into the next decade," he wrote.

So, Abrahams writes, while $NBIX hasn't often been seen as a takeout target, if this gap continues between where investors vs. analysts value the stock, Neurocrine could certainly be attractive to a large pharma acquirer.

In a new note, RBC Capital Markets analyst Brian Abrahams poses a question: Could Neurocrine Biosciences be a takeout target? $NBIX

"This is why clear, pragmatic, consistent regulatory guidance is essential. The types of individualized genetic therapies that we're trying to develop simply do not fit in the traditional model of drug development."

"The biggest hurdle, though, is moving beyond a first in human trial to regulatory approval. Getting past that gap is what allows treatments to become commercially available, reimbursed and scaled so all patients who need them can access them ... "

Rebecca Ahrens-Nicklas, an attending physician at CHOP and part of Baby KJ's treatment team: "Disease communities face real uphill battles when it comes to developing new therapies." #FDA #HHS

"Families heard the same thing. There are not enough patients. The approval will take too long. You just have to wait [for] the science to catch up with your child. That ends today."

"Under the framework that we're announcing today, one well controlled clinical investigation, supported by confirmatory evidence, can support approval for decades."

Just released: FDA draft guidance for developing individualized therapies for ultra-rare diseases fda.gov/news-events/pr…

The rejection of Moderna's flu shot application is the latest example of Vinay Prasad overruling career FDA scientists to place vaccines under harsher scrutiny. trib.al/6UGBYwa