John Carroll
@JohnCendpts
47 yrs before the mast. Invented FierceBiotech, founded Endpoints News w/ 243,000 subs. Devoting time to the big showdown with Merkel cell carcinoma . We'll see
An Endpoints 11 team has been hard at it working on this year's selection of up-and-coming biotechs. Harvard's George Church and I will be doing the fireside chat. Really looking forward to gathering at The State Room on the evening of September 18. You can get tickets here:…
You get the sense that there's a lot more ahead....
Recapping $SRPT chaos: - undisclosed patient death in gene therapy trial - CEO says immediate disclosure wasnt material, stock craters - FDA requests $SRPT pull a diff. DMD therapy, revokes platform desig., and adds clinical hold on gene therapy - Sarepta PR says no to pull
We debate how much of this stuff is worth covering, but when the MAGA political world starts getting really into one of the FDA's most important leaders, it's newsworthy. I will be happy to go back to MFN, trials and earnings, however
Quite the uproar over Prasad's past, especially since the CBER director role is not a political one (or is it?)
🚨 ALERT 🚨 VINAY PRASAD, THE LEFTIST SABOTEUR UNDERMINING TRUMP’S @US_FDA, MUST BE FIRED NOW! We have a crisis at the FDA! A wolf in sheep’s clothing is sabotaging President Trump’s bold “Make America Healthy Again” (MAHA) agenda! Dr. Vinay Prasad @VPrasadMDMPH, a…
New rare disease treatments not getting the same reception as they did under Marks
$REPL rejection follows the rejection of $CAPR and $RARE drugs, and the FDA's actions against $SRPT. CBER Director Vinay Prasad is setting a different tone than his predecessor.
$SRPT crumbles.
Sarepta halts US shipments of Duchenne therapy, bowing to FDA endpoints.news/sarepta-halts-…
AstraZeneca is putting up $50B to fund a new "state of the art" R&D center in Kendall Square along with a major manufacturing commitment to help get Trump off of Big Pharma's back. Best I can see Trump has been playing pat-a-cake with the industry, talking big but really not…
Tidmarsh also founded Horizon Pharma in 2005 and served as CEO until 2008, staying on as a director and consultant until 2011. During that time, Horizon won regulatory approval for Duexis, an arthritis drug that combines the active ingredients of Motrin and Pepcid. Tidmarsh’s…
Yes, the new head of CDER is not a big fan of @endpts -- playing at semantics, attacking the media, playing the troll. Should go far with the Trump gang at #FDA.
On FDA, some of you saw the news about Makary & KalVista last month. The drug was shortly approved after the @endpts story broke which helped prevent a potential investigation or congressional hearing re: FDA. Here's what the new head of CDER posted on Linked In at the time:
PR firms sometimes like to complain about media headlines referring to complete response letters as rejections, too. The argument always struck me as a bit silly. If a CRL isn't a rejection, then can the FDA ever be said to have rejected a drug approval application?
On FDA, some of you saw the news about Makary & KalVista last month. The drug was shortly approved after the @endpts story broke which helped prevent a potential investigation or congressional hearing re: FDA. Here's what the new head of CDER posted on Linked In at the time:
Just put two and two together on something I'm not planning to get into the weeds about. But this is the funniest #biotech news I've read in weeks. And that is saying something. endpoints.news/fda-names-form…
It’s a great example of how much damage you can do by being “kind” Sarepta should have been put out if it’s misery a decade ago. They played on the desperation of families to continue forward with poorly effective approaches. And they are calling on those same desperate parents…
Speaking of #biotech disasters, $SRPT down 6% this a.m., extending losses after Friday's calamitous, way late disclosure of another patient death and refusal to obey the FDA's request that it pull Elevidys from the market. This from a company that has only survived and thrived…
This is the kind of deal you do when you set up a #biotech junkyard and specialize in acquiring wrecked companies so you can extract any remaining value. It's kind of intriguing. If you're into that sort of thing. Target rich environment, as they say. $ITOS…
Really good dialogue and news re $SRPT today on BiotechX. Fast forward. Like old times.
Sarepta Therapeutics has refused the FDA’s request that it pull gene therapy from the market following patient deaths statnews.com/2025/07/18/sar…
Will Sarepta comment? Gonna be a real test today of what they consider material, given the last few hours...
Pretty trenchant commentary from @BrianSkorney on $SRPT and FDA: "The trash-talking pharma-critic Vinay Prasad that we have followed on Twitter the last decade would have pulled Elevidys due to liver tox weeks ago. The benign personality characterized by his public appearances…
Like I said, CEO has to exit. Skorney just now on $SRPT: The reports of this safety event follow the conference call earlier this week, when we asked directly about whether safety considerations drove the discontinuation of the LGMD program, and management replied that the…
🚨FDA is asking Sarepta to pull DMD drug Elevidys endpoints.news/fda-asks-sarep…