Biotech Hangout

FDA issues final guidance for designing clinical trials for psychedelic drugs & schedules public hearing on 14-Sep. Guidance document addresses the issue of functional unblinding, including appropriate placebos and proposals for evaluating expectancy

OK, $AGEN is "discontinuing financial support" for a study that's actively enrolling 834 patients. I'm sure that's ethically fine. Good luck to all involved!

The FDA was told in April '25 to suspend a program that checks whether its own labs can correctly identify contaminants in food—incl detecting the parasite Cyclospora in spinach. FDA was asked this week if the program was restarted in '26 & other Q's- didn't answer those Q's:

Say you have £100M net worth. The proposed wealth tax is 2% on assets over £10M… so you’d owe £1.8M every year in additional taxes. To pay the additional £1.8M each year (2% on £90M) you would need to pay a £4M dividend. Of that £4M dividend, you get zero ALL of it is tax (see calculation below). Given that most scalable assets pay less than 4% yield as it is, there is no point in owning UK assets. Especially because you’d also be paying tax to buy the assets, to employ people to run the assets and you’d be losing CGT or IHT if you sell them or die. All serious investment would dry up and the economy becomes a backwater, stagnant economic wasteland rapidly.

FDA returns to releasing pharma rejection letters with 14 new CRLs endpoints.news/fda-returns-to…

@MatteisPaul on what this means for other companies in the ATTR space, points out: “The $ALNY drug Amvuttra — and nucresiran to even more of an extent — get better lowering of the TTR protein with less variance, and attain their peak faster, those are definitely true…I think siRNA for almost every target seems to be more potent outside of some of the stuff that ASOs have accomplished in the CNS.” #BiotechHangout

On today's #BiotechHangout, @SamFazeli8. @biotech1 (Josh Schimmer), @EricSchmidt151, @MatteisPaul, & @statnews @matthewherper will cover regulatory & policy: Pazdur's potential return, CRL walk backs, $AGIO's priority review, Medicare GLP-1 Bridge begins, Anthropic launches Claude Science, US lawmakers review China-based clinical trial activities, deals: Ipsen/Kartos ($1.7B), $NVS/Myricx ($1.5B), Ipsen/Memo ($800M), $VRTX/$CRNX ($10B), $AZN/Abbisko collaboration in NSCLC, data: $AZN/$IONS ATTR-CM results & readthroughs to $ALNY, $BBIO & $PFE, $CMPS data, $ABVX UC data, $BBIO in achondroplasia results, $GMAB/$ABBV's DLBCL success, Skyhawk’s Huntington's data, $RVMD ESMO-GI G12D PDAC data, #AAIC26 preview ($BIIB & Roche data), & much more: x.com/i/spaces/1RKjp…

The hosts dissect the surprising results of the $AZN and $IONS trial of eplontersen in ATTR-CM. @biotech1 (Josh Schimmer) says there are still lots of questions. "We'll have to see what that actually means. Does that mean it was not stat sig? Does that mean there was a trend or literally nothing going on? And if that's the case, now we're going to have to figure out what that likely reflects. Could it be that tafamidis is a better drug than many of us thought it was? Very unlikely, just given the totality of data. Could something else have been going wrong with eplontersen? We've known ASOs can have some cardiotox element to them. So, is it possible that that manifests in this trial for some reason? Lots and lots of questions. But for the time being, it looks like it's going to remain a three-way market battle between $BBIO, $ALNY, and $PFE.” #BiotechHangout

$NVS is expanding its ADC portfolio with the acquisition of Myricx; @SamFazeli8 observes: “$NVS is paying this $1.1B cash upfront for a private company not in the clinic yet, and there's a total another of another $400M they can pay. It felt a little bit rich for a for a private company, so there must be something really exciting in there for $NVS to have done this. And this is the second ADC deal out of Europe in recent months of course, following $GILD buying Tubulis.” #BiotechHangout

@MatteisPaul says that $VRTX making its largest-ever deal to acquire $CRNX for $10B is evidence that “this specialty rare endo space has quietly become pretty hot. It feels like it’s maybe the next niche space to become a little sub vertical, like the renal space has in the past three to four years…It's one of those drugs that works decently well, but really the big selling point is that it's super safe, easy to use, and this disease is a big unmet need. Patients have been using steroids for 50 years to treat it, and it's on newborn screening — so there's a lot of patients out there right away that you don't have to find. I think the only real debate I've heard from the $VRTX side of this deal is just, did they overpay?” #BiotechHangout

On Anthropic’s new AI tool directed at researchers in drug discovery, @matthewherper says: “I think there are two things we have to separate, for the whole AI field and Anthropic in particular, which are: is there useful stuff you can do now? Which I think the answer is yes... And then there's this big pie in the sky stuff, is this going to change biology…There I think the jury is still out. It's certainly plausible that this helps industry pick better targets, that it helps lower the failure rate.” #BiotechHangout Read the article: statnews.com/2026/07/06/ant…

@biotech1 (Josh Schimmer) weighs in on FDA transparency for rare disease drug development. “Rare disease datasets are often in the gray area because they’re not able to run rigorous controlled Phase 3 trials...Anyone who's tasked with that job is always going to be in a nearly impossible position, and as a result, the more transparency that the FDA can bring to their decision-making process, the more they can at least try to align everyone around where that line in the sand is being drawn and why.” #BiotechHangout

FDA grants $AGIO priority review for mitapivat in sickle cell disease despite missing one of its primary endpoints. @EricSchmidt151 gives his perspective: “Maybe we're swinging a little too far in the other direction. The direction of lowering the bar and approving anything that looks active in rare diseases, despite maybe not having met the statutory requirements for proving efficacy…Everyone pretty much gave up on the program after the data last year. But the company, after talking to the FDA, moved forward with the filing. And this week, we learned that that filing was accepted for priority review.” #BiotechHangout

@EricSchmidt151 adds: “What's interesting to me -- and I hadn't appreciated this, -- we haven’t seen CRLs released since April of this year… So there may be a meaningful challenge underway right now that prevents investors, at least, from getting some of the transparency that I think many of us have relished.” #BiotechHangout

On FDA’s halting the release of CRLs, @matthewherper says, “I wrote a lot about Dr. Makary, and one of the big issues throughout his tenure was not really worrying about legality or regulation before making a decision, and that can result in things being walked back.” #BiotechHangout

Kicking off the show this week, the hosts talk about recent movements in the biotech markets. @SamFazeli8 observes, “The index is down, there are some massive share price moves, and I can't get my head around it. I don't know why.” But @biotech1 (Josh Schimmer) says, “XBI is still up 30% year to date, nearly 80% year over year. So, a little pullback from time to time is not any cause for concern. We'll see what the future brings.” #BiotechHangout

IRR for angels and seed investors that don’t have large funds to follow/protect investment is mostly negative. That ecosystem has more or less been wiped out in biotech. Tech related thread below but pls respond w biotech stories

$ALNY up 15% $BBIO up 12% cc $IONS $AZN