# Episode 196 - September 18, 2026

An episode of [Biotech Hangout](https://invisiblenetwork.ai/podcast/1568911820), 25 Sep 2026, 55 min.

Audio: https://anchor.fm/s/55bdff38/podcast/play/126308894/https%3A%2F%2Fd3ctxlq1ktw2nl.cloudfront.net%2Fstaging%2F2026-8-25%2F432668244-44100-2-5b5f92b6d1063.m4a

On this week's episode, Josh Schimmer, Eric Schmidt, Paul Matteis, and guest Adam Feuerstein open with the OpenAI/Hugging Face AI agent controversy, debating whether calls for AI regulation reflect genuine safety concern or capital rotating out of biotech into AI. Turning to markets, the group describes a choppy September for biotech, driven more by rate jitters and a quiet M&A backdrop than any real shift in FDA or drug-pricing sentiment. In data news, Xenon Pharmaceuticals voluntarily paused enrollment in its Phase 3 depression trial after adverse events, sending shares down as investors worry about the company’s epilepsy franchise. The hosts then cover North Immunology's reverse merger, framing the ongoing wave of reverse mergers as a viable, capital-efficient alternative to IPOs. The conversation turns to Sionna Therapeutics failed cystic fibrosis study, leading to a broader discussion of Vertex's durable moat in CF—its proprietary cell-based assay, willingness to cannibalize its own pipeline, and multimodal drug combinations. The episode closed on rare disease, citing recent approvals as signs of a resurgence, along with the measles outbreak and RFK Jr.'s renewed ties to anti-vaccine groups. *This episode aired on September 18, 2026.

## Transcript

[0:00] You're listening to Biotech Hangout, a live and unedited
[0:02] weekly discussion of all the latest news in our industry with
[0:06] a group of biotech leaders and experts.
[0:08] I'm Josh Schumer, hopefully to be joined very soon by my
[0:12] colleague Eric Schmidt, who's going to be leading us.
[0:15] I'm very, very pleased to have Adam Feuerstein, who probably
[0:18] needs no introduction whatsoever to this group, and same for Paul
[0:21] Matthias. So hopefully Eric will be
[0:25] joining in in a second or or two.
[0:27] And if not, Paul, if you want to kick things off, go for it.
[0:32] Well, I mean, there's a lot to talk about.
[0:34] I know we were saying there's not as much news and we want to
[0:37] be more thematic. I think we could probably wait
[0:41] for Eric, Josh to have the conversation and debate around
[0:46] AI that you guys wanted to do, especially because I just want
[0:51] to watch you guys Duke it out. I I feel like I am just such an
[0:55] amateur when it comes to sort of predicting what might happen
[0:58] with AII mean. Maybe like Josh, is there any
[1:01] news you thought was super interesting this week that you
[1:03] want to start with? Like maybe we can hit some of
[1:05] the news items at the top. Like you could go and I could
[1:08] always talk about Xenon or Siona and then, you know, maybe we
[1:11] we'll get Eric by then and get more thematic.
[1:13] What do you think? Well, on the AI news news topic,
[1:20] for those of you who've been been following the open AI
[1:23] hugging face incident, basically they, they had set up a sandbox,
[1:29] which is like kind of a cornered off experiment that doesn't have
[1:33] access to the Internet. And they had a whole bunch of AI
[1:36] agents working on a problem. And the, and the agents weren't
[1:39] supposed to be communicating. And yet somehow they on their
[1:43] own found a way not only to communicate with each other, but
[1:48] break out of the sandbox and find a way to the broader
[1:51] Internet hack, hack their way in to a hugging face, which is
[1:57] itself a kind of an, an open community for AI innovators.
[2:02] Sorry, Hugging face, Hugging Face had its own AI agents
[2:08] defending against the incoming attacking agents and like
[2:14] completely out of control from the open AI team that's caused
[2:18] this this broad debate around, you know, whether we need
[2:21] stronger guardrails around AI now.
[2:25] Now, fortunately, when it comes to innovation in AI in biotech,
[2:30] I don't think anyone's particularly worried that these
[2:33] efforts are going to destroy the world if anything really help
[2:36] the world. But but very relevant because we
[2:38] have started to see a shift to capital maybe away from biotech
[2:44] for a number of reasons, but one of which is positioning around,
[2:48] you know this this ever expanding AI trade.
[2:52] And so, you know, we're, we're hoping investors will perhaps
[2:56] come back to biotech and come back to even AI in biotech,
[3:00] because for the most part, I think we can all agree that the
[3:03] companies that we work with, engage with and those who are
[3:07] advancing medical therapeutics for AI aren't, aren't at risk
[3:13] of, you know, whatever happened to with the, the Hugging Face
[3:16] experience and open AI in terms of hacking into other networks,
[3:22] etcetera, and potentially causing significant damage.
[3:25] Not, not to say that there can never be some bad actors in, in
[3:28] our world that, you know, they, they can be found anywhere.
[3:31] And so guardrails perhaps even in even in biotech may be
[3:35] appropriate because we don't want AI agents that are kind of
[3:38] cracking into competitor companies and, and stealing
[3:42] proprietary information. So be interesting to see how
[3:45] this all evolves. And you know, governments, I
[3:47] think the US and China have resisted calls to put more
[3:51] guardrails because they do see AI as the way the future, but on
[3:55] the other hand, appropriate for everyone to have a little bit of
[3:57] their their guard up, whether it's trade secrets or otherwise.
[4:00] Josh, what do you think about this like debate on whether or
[4:03] not like the calls for regulation around AI from some
[4:08] of the leading companies are like true earnest calls for
[4:13] actual like, you know, altruistic protectionism.
[4:16] Or actually some of the leading companies thinking that
[4:20] regulation could actually help them and help build the remote
[4:24] when you have all these other labs and groups that are kind of
[4:27] figuring out very rapidly how to do everything that they do, but
[4:29] for like cheaper. I don't know.
[4:32] I mean, my, my impression is that, you know, a lot, a lot of
[4:35] the innovators are, are innovating for the betterment of
[4:38] the world. And when they see a risk like
[4:39] this and recognize that, you know, there are dynamics to this
[4:43] that that can get out of control and need to be protected that,
[4:46] that it is genuine, right. I mean, you, you think even
[4:48] broadly like nuclear proliferation treaties and
[4:52] agreements amongst countries to make sure that they, they stay
[4:56] good actors for the benefit of society.
[4:59] Now, you know, just like rogue actors can try to get their
[5:02] hands on nuclear weapons, so can rogue actors try to get their,
[5:06] their hands on powerful AI. And so, you know, it's just the
[5:09] start of a dialogue around, you know, national security, global
[5:13] defense considerations when it when it comes to AI.
[5:16] And, you know, hopefully it's something that countries can
[5:18] actually unite around, in the way that they united around the
[5:21] nuclear anti proliferation treaties and figure out a way
[5:26] not just for for, you know, established companies and
[5:29] governments to to stay inbounds, but to ensure that any rogue
[5:34] actors are also contained. So I think we got Eric on.
[5:37] Hopefully he's able to to join in and and be heard here, yeah.
[5:42] Thanks. Thanks Josh for always having my
[5:44] my back and hi Adam and Paul and apologies to to the listeners
[5:48] for my technical difficulties. Did you guys just start on the
[5:52] AI hugging face topic, Josh? Yeah, we just covered that one
[5:58] as we were waiting for you. So maybe maybe worth moving on
[6:01] to some of the other topics that you wanted to to cover?
[6:04] Well, let's let's talk. Thank you for that.
[6:06] And maybe we'll we'll wind the clock back a little bit.
[6:09] And and just start with the biotech markets more broadly.
[6:12] It's kind of been a choppy start to the month.
[6:15] I think we're down about 4% month to date in September and
[6:19] maybe off almost 10% from the highs. 910% down from the highs.
[6:22] I think you know, the XBI is is still up about 30% on the year.
[6:27] So nobody is complaining too loudly.
[6:29] But we keep hearing that the wrong stocks are working from
[6:33] from buy side investors. The the gene editing stocks, the
[6:36] modernas the world, the heavily shorted names summit and others
[6:40] come to mind while quote the the good names are are languishing.
[6:45] So just want to take temperature of the markets here from from
[6:49] both Adam and Paul and and see if you guys think there's a
[6:53] problem or if there's something to be blamed or maybe this is
[6:56] just a little bit of a healthy consolidation in the space.
[7:00] Who wants to start? I would just say it feels just
[7:04] as much psychological as anything.
[7:06] You know, I think we've talked over and over on this podcast of
[7:11] to what degree do we need MA for stocks to work?
[7:14] To what degree is that a referendum on like the business
[7:17] model? And I, Josh, you always have
[7:19] some interesting thoughts about how the sector is less reliant
[7:21] on MA. But you know, when things kind
[7:23] of get a little sloppy on the macro side, right?
[7:26] Like some of the macro headlines have been a little scary.
[7:28] We've had rates going up, you know, we've had you know a
[7:31] string of just certain high profile trial failures, many of
[7:34] which are not even in SMID cap. I think some of that like
[7:37] emotional drift can kind of trickle down into some of the
[7:40] better performing SMID caps, especially when we have gone
[7:44] through a period that's quieter for for M&A.
[7:47] So I mean when I talk to people, it's not like I don't actually
[7:50] hear anything like, oh man, like things have flipped now where
[7:52] I'm really worried about the FDA or I'm really worried about drug
[7:55] pricing. Like if anything, I think we're
[7:56] in a better spot on those topics than we've been in the past
[7:59] couple years. But it's sort of just like a lot
[8:02] of some negative data readouts, some rate stuff.
[8:06] It's quiet on the catalyst front, you know, so I'm certain
[8:11] stocks are up a lot like I it feels a little bit more like
[8:14] kind of a negative kind of like on we sort of kind of market.
[8:18] I don't know what. What do you think, Adam?
[8:21] I hear the same, Paul. I, I, I don't have much to add
[8:24] other than I think you covered kind of everything that I hear.
[8:26] You know, I think there are people, you know, people were
[8:30] complaining. I mean, you know, not
[8:31] complaining is maybe the wrong word, but you know, we're seeing
[8:34] fewer opportunities maybe to buy stocks were getting copy.
[8:39] So, you know, there is more maybe more opportunity now to
[8:42] get into some stocks. But yeah, I think all those, I
[8:46] think all the all the things that you said, Paul, I think are
[8:49] are pretty true ring true. I'd I'd maybe add just two two
[8:53] other kind of data points to this one.
[8:56] You know, the setbacks of Delbracs for Novartis after the
[9:01] the very sizable avidity acquisition.
[9:05] You know, we, we, we've heard you know, somewhat putting a
[9:09] chill in terms of appetite for large deals for assets that
[9:14] aren't fully de risk. So you know, that may may be
[9:16] playing a role as well if you look a little bit deeper into
[9:19] the rising interest rates and what's going on, on the on the
[9:23] macroeconomic scale. And you juxtapose that to
[9:30] continuing to see very, very aggressive rising drug prices at
[9:35] launch, right. The industry responding to all
[9:39] the criticism of about drug spend and drug prices
[9:42] essentially by doubling and tripling down on even higher and
[9:45] higher drug prices, especially as you kind of see the political
[9:49] landscape shift a little bit as we get closer to the midterms.
[9:54] Not unreasonable to have a little bit of concern that we we
[9:59] may see a partisan or bipartisan push to bring drug pricing back
[10:04] to the table, which, you know, as we've learned in the past, it
[10:08] just takes one tweet to roll the sector over.
[10:11] And so, you know, we'll kind of see how it all evolves.
[10:14] I think hopefully, you know, that that's not going to
[10:17] necessarily be a key election driver given how many other
[10:22] drivers there are this time around.
[10:25] At our healthcare conference last week, John Crowley seemed
[10:28] to suggest that, you know, politicians are not necessarily
[10:31] going after drug prices, certainly not for smaller
[10:35] biotech companies, but something we'll have to keep an eye on and
[10:38] something that may be naturally adding to some jitters around
[10:42] the sector. And I would say like, you know,
[10:43] you mentioned one tweet, Josh, but also one deal, right?
[10:47] I mean it all it will, all it will take is 1, you know, sort
[10:50] of big deal M and a deal to kind of get people excited again.
[10:54] I think people are somewhat nervous about the M and a pause.
[10:58] You know, I hate to, I hate to, I hate to put it up, put it all,
[11:01] put it on one single stock. But I feel like Avivax kind of
[11:05] causes everybody to have major agita these days.
[11:10] I see it's down again today. But, you know, it's seems like a
[11:14] lot of attention is focused on whether or not Avivax is going
[11:17] to get acquired. OK, well, well, good stuff.
[11:22] We do have a couple of important news events we want to talk
[11:25] about. And maybe Paul and I know you're
[11:29] keen to talk about the Xenon news that's been also maybe
[11:32] causing some folks enjoying it today.
[11:34] Yeah, sure. I mean, this kind of thing
[11:36] doesn't help sentiment because, you know, some of the investors
[11:39] I've talked to in the past 12 hours in Xenon have lamented
[11:43] that they thought this was a, you know, a quieter, safer name
[11:47] to own, right? Where you sort of have this core
[11:50] de risk aspect of the value and then a bunch of, you know, risk
[11:53] reward upside bias drivers. And I think the perception is
[11:57] that this stock, you know, wouldn't go down this much if
[12:00] one of those things failed. So essentially this company
[12:04] Xenon has a drug as a 2 calendar with very good data and
[12:07] epilepsy, they've been developing it for depression,
[12:10] bipolar depression. They also have some earlier pain
[12:12] programs, which are pretty cool. But their depression data was
[12:15] supposed to come out next year. It still is.
[12:17] But last night they announced they were pausing enrollment due
[12:22] to adverse events that we're seeing in the depression study.
[12:25] And I think part of the issue here was just that the
[12:27] disclosure, it's ultimately not that confusing when you when you
[12:32] sort of hear the call and talk to them.
[12:34] But at face, I think the disclosure made it sound like
[12:36] there was this new major safety issue in depression and it
[12:40] spooked people that the value of this drug and epilepsy could be
[12:43] in question. And I think the stock trading
[12:45] off as much as it is almost 30% tells you that some people are
[12:48] still, still nervous about that. You know, what the company says
[12:52] is that the adverse events they're seeing in MDD for this
[12:55] drug, certain cognitive adverse events like, you know, confusion
[12:59] or aphasia, they reported a couple cases of psychosis that
[13:04] these have been seen in epilepsy.
[13:06] We know they've been seen at the high dose in epilepsy before,
[13:09] but they hadn't been seen in depression.
[13:12] And I think the depression phase two data were actually kind of
[13:14] surprisingly clean with this drug, which led to the pause.
[13:17] And so, you know, I think again, in a, in a nervous market, stuff
[13:20] like this is digested and, and people sort of right off
[13:23] depression worry about increased risk, worry if this, you know,
[13:27] means Xenon's not an MNA target anymore in the next three to
[13:30] four months, right? Like you know, I, I think our
[13:33] view is that if you look at epilepsy drugs, I think a really
[13:37] good analog is look at the label of Keppra.
[13:40] Keppra is kind of the aspirational drug for most of
[13:43] these companies going after mass market epilepsy because it was a
[13:47] couple billion dollar drug at much lower pricing and it kind
[13:50] of got escape velocity or it was written not just by specialists
[13:52] but by general neurologists. And the Kepler label says
[13:56] everything that I just kind of mentioned that Xenon was talking
[13:59] about seeing in their MDD study. So I think we pretty strongly
[14:01] feel that in epilepsy like nothing here has changed, like
[14:05] there's no clinical hold, like you know, like like the epilepsy
[14:08] profile is the epilepsy profile. But you know, I think again,
[14:12] it's a testament to what we said on the macro side that this is a
[14:15] much more nervous market than it was three or four months ago.
[14:20] Josh, I know you've been following the space closely and
[14:23] you know on a particular anything you want to.
[14:25] Add I could point about Keppra it it is a little confusing.
[14:29] I think we're all all all still digesting as well the extent to
[14:32] which, you know, where we need to read through because
[14:35] depression and epilepsy do overlap.
[14:37] And that was actually one of the things that Xenon highlighted as
[14:40] they were advancing the depression study as, as you
[14:42] know, that kind of a beneficial signal odd mood could read
[14:46] through favorably to to epilepsy.
[14:48] But, you know, on the other hand, whatever led to this
[14:51] heightened concern around around the depression indication, you
[14:55] know, hard to hard to square that with with a seeming lack of
[14:58] concern with with epilepsy. I I think Paul overall though
[15:03] phrased it, framed it, framed it very well and.
[15:09] Paul, I I wasn't on the call, but what's the, what's the
[15:12] rationale for the pause? I mean, what are they going to
[15:14] learn and how they're going to go forward?
[15:16] That's an interesting question because I think some of the
[15:18] people who are close to this company are kind of wondering
[15:20] why. Why pause it?
[15:23] And you know, would some other CEOs who are maybe that's risk
[15:29] averse have have paused this at all, right.
[15:31] I'm not going to name names, but you know, there's again, there's
[15:34] no FDA issue here. Their DSMB recommended when they
[15:39] talked to them to institute a dose escalation or dose
[15:42] titration. I think a lot of these adverse
[15:44] events are seen early in dosing. We've seen with a lot of CNS
[15:48] drugs that, you know, the right kind of dose titration can cut
[15:52] AE rates in half even. And because this study was
[15:56] already at 360 patients, so it's already at a point where, in my
[16:01] opinion, like it's powered to sort of show a clinically
[16:05] significant difference or even a very small difference.
[16:08] I think the idea was why don't we just not enroll any more
[16:11] patients in this study, pause the next trial, the other
[16:14] trials, figure out what the dose modification might be and then
[16:18] go from there. But yeah, I mean, I also think
[16:22] Josh, back to your point about people trying to figure out the
[16:23] read through to epilepsy, I think the other big difference
[16:26] is that the therapeutic index of this drug and anti epileptic
[16:30] drugs in general are usually a lot wider in epilepsy patients
[16:35] than on epilepsy patients, right?
[16:36] You look at some of these ASMS in bipolar and the side effect
[16:39] profile is very different, right?
[16:40] It's a much more sensitive population.
[16:42] Like what's unique with xenon and epilepsy is they have this
[16:45] low dose that has a Keppra like effect size had a very low rate
[16:49] of drug related adverse events, right?
[16:51] It's really their high dose that has some of the stuff they're
[16:53] seeing in MDD. And so it's actually like a
[16:56] whole fascinating side note that with some of these CNS drugs,
[17:01] the tolerability profiles often worst and healthy volunteers or
[17:04] people without the disease, and then people with the disease
[17:06] might actually be better at tolerating some of these side
[17:09] effects. And so I that's also, I think
[17:11] part of my kind of epilepsy is insulated narrative.
[17:14] But yeah, Eric, I mean, I think, you know, it's, it's unclear if
[17:18] they actually had to pause it. And I think when people hear a
[17:22] pause, that their mind goes to a place that, you know, implies
[17:26] greater risk. Well, thanks for covering that.
[17:30] Very interesting indeed. Adam, do you have anything to
[17:32] add on this or should we? Keep going.
[17:35] All right, let's come back to you that Adam then and and I
[17:38] know you had a piece on the North Immunology reverse merger,
[17:41] maybe it Tees up a broader discussion about reverse mergers
[17:45] in our space, but why don't you first fill us in on, on what
[17:48] North Immunology? Yeah, since we are on X,
[17:51] probably a lot of you follow sheep of Wall Street.
[17:55] Dan Schneeberger, portfolio Manager at Adair One Capital,
[17:59] and he and an analyst of his have been, they've been
[18:02] incubating an immunology company for the last two years called
[18:05] North Immunology that is now developing an Illinois 13,
[18:10] Illinois 18 bispecific antibody for, for atopic dermatitis.
[18:16] And yeah, they went, they went public via a reverse, reverse
[18:20] merger yesterday. What was the name of that shell?
[18:24] Athon Medical, I guess is the one that they, they reverse
[18:27] merge into and then obviously did a, you know, like a lot of
[18:30] these, they did a, a subsequent pipe, you know, concurrent pipe
[18:33] financing. You know, to your point, Eric,
[18:37] yeah, a lot, lots of reverse mergers this year, right?
[18:41] I, I, I don't know, I think I saw someone said like maybe like
[18:44] 20 or 21 companies have gone public through reverse mergers.
[18:50] I kind of like reverse mergers mostly because it just gets rid
[18:53] of shit goes right. You know, like we always
[18:57] complain about too many, too many biotech companies, too many
[19:01] bad biotech companies out there. So I guess, you know, we replace
[19:06] one with, with another. We don't add any more to the
[19:10] pile, which conceptually makes me happy because there's already
[19:14] too many of them. I don't know how you guys feel
[19:16] about that. I mean, this phenomenon is kind
[19:21] of relatively recent. I, I, I think you know, up until
[19:25] the last year or so, if you were a higher quality biotech
[19:28] company, you would not go public via the reverse merger pathway.
[19:32] You'd almost certainly choose an IPO.
[19:35] But now today new day and era, we have kind of a, a choice for
[19:39] companies. I, I'll put some numbers around
[19:41] it. By my count, we have 21 IP OS
[19:43] this year, including one that's just trading this morning that
[19:47] that we're part of. So I, I can't mention and we've
[19:50] got I, I have a few less than 20 atom on the reverse mergers, but
[19:55] but each have kind of performed well.
[19:59] Well, one each have raised a lot of capital.
[20:01] I think the average raise in IPO is about $340 million.
[20:05] The average raised in a reverse merger plus a pipe is about $200
[20:09] million. So these companies are?
[20:11] Are certainly able to drive a a lot of of capital to these
[20:16] transactions and I think one of the different oh, they've also
[20:20] both traded quite well in the aftermarkets.
[20:22] I think the IP OS are about 3540% and the reverse mergers
[20:27] that have happened year to date are are up about 2025%.
[20:30] So, so both are viable pathways. What's different of course with
[20:34] the reverse merger is at the front end of the transaction you
[20:38] choose or you, you, you get a few shareholders and you bake in
[20:42] the deal and they tend to be smaller select group of
[20:46] syndicates. So you don't have the big coming
[20:47] out party that's an IPO. You don't get the necessarily
[20:51] the, the visibility or or the big hurrah out of it, but but
[20:54] you do get your capital and you really lock in your capital well
[20:58] in advance kind of independent of the markets, which is, which
[21:01] is I think why that pathway is of interest to some.
[21:05] I mean, those with a historical perspective, we'll remember, I
[21:08] guess back in the day we had we had Cougar kind of go public as
[21:11] the first legit company. Oh, I remember that.
[21:14] Yeah, yeah. Right, Alan Arbach and he kind
[21:18] of yeah. And then his second company,
[21:21] Puma went, went public the same way.
[21:22] But but again, up until recently, it was a shun pathway
[21:26] and and maybe it had a taint around it.
[21:28] I don't know, Josh and Paul, whether you guys think that you
[21:32] know this day and era that that the reverse merger pathway is
[21:36] anything other than a quick path to an IPO where you lock in some
[21:39] funding. Yeah, it's it's I kind of now
[21:42] group the the reverse mergers with IPO's because that the
[21:46] dichotomy that used to exist of, you know, the reverse merger
[21:50] path primarily being used by lower quality companies is
[21:53] obviously no longer the case. And you know, they're raising
[21:56] about as much as they would have as as an IPO.
[21:58] So the end of the day it's it's not a huge difference.
[22:01] You know, it's private companies that should be public, find a
[22:05] way to go public, reverse merger, IPO and a SPAC.
[22:08] You know, we may even see SPAC's make a comeback.
[22:10] We'll we'll see. But also a testament just to the
[22:13] incredibly high caliber of private companies, the number of
[22:17] high caliber private companies that that we're seeing it's, it
[22:20] is a remarkable time in the industry.
[22:22] Yeah. Now, when I was talking to Dan
[22:24] about the deal earlier in the week, you know, he mentioned, I
[22:28] didn't put it in my story, but he mentioned to me that, and
[22:30] I've heard this, I've heard this elsewhere too, is that, you
[22:32] know, the, there's there's a lot of demand for these, for these
[22:36] shells now. And it's almost like the, it's
[22:40] almost like the public shells can kind of start driving.
[22:43] You have a little sort of look driving, having a little bit
[22:46] more leverage and higher demands in terms of how these seals can
[22:49] play now a. Shell a shell cost.
[22:53] I'm sorry, what'd you say? Did he tell you how much a shell
[22:56] cost? He, he did not, we didn't get
[22:58] all so deeply into the civic specifics, but he just said, you
[23:02] know, that there's a lot of competition, that there are a
[23:04] lot of private companies out there looking to do reverse
[23:08] mergers. And they're only a limited
[23:10] number of shells out there. And so, you know, you're, you're
[23:14] kind of competing for those for those shells and it puts the
[23:19] shells in a little bit of the driver's seat.
[23:22] I have no idea how accurate this is, but I heard a shell can cost
[23:24] $20 million. I just thought it was like kind
[23:27] of wild, you know? You can back into this based on
[23:33] the residual value or ownership that the shell has in the new
[23:37] code, right? So I think you're right.
[23:39] I've heard that the price has gone up and, and you're correct,
[23:42] I've heard, you know, anywhere between 10 to $20 million.
[23:45] So there aren't as many of these as there used to be because
[23:48] they're all getting gobbled up. And I think there are some
[23:50] market dynamics at play. But Paul, how do you think as an
[23:53] analyst about a company that goes publicly to the Shell
[23:56] versus the IPO route? Do you care?
[23:58] No. Not really.
[23:58] I mean I think you know, I mean I think you it's interesting,
[24:03] right? Like I feel like the reverse
[24:04] deals just tend to be IP OS in the sector are often clubby, but
[24:08] like they tend to even be clubbier, right.
[24:12] And sometimes, you know, from covering a company that went
[24:14] publicly reverse, you'll be marketing and really
[24:18] sophisticated biotech investor loyalists will have never heard
[24:21] of the company. And I almost wonder if sometimes
[24:24] that's not just a downside of reputation, but sometimes
[24:28] there's some hurt feelings when people are like, Oh, I didn't I
[24:30] didn't get the call on that. You know, does this have like a
[24:33] long term negative impact? Like I'm not really sure, right.
[24:35] But you know, I think maybe I think when it maybe when a
[24:39] company goes publicly a reverse merger, they can kind of remain
[24:42] anonymous to a lot of the market for longer, even a lot of the
[24:45] specialists. But I mean, in the grand scheme
[24:47] of things, if you believe that like the market is efficient
[24:50] over time, like it doesn't seem to matter because it does not
[24:53] seem to really sacrifice over the quality of investor
[24:55] syndicates at all, right? Yeah, I, I agree with you on
[25:00] that. You know, that, that broader
[25:03] point of not having the visibility to go public via the
[25:06] reverse merger, it's I think really incumbent upon the team
[25:09] after the deal to, to get out there and to, to make its, its
[25:14] name known more broadly. I think we've, we've been
[25:16] involved with a bunch of both IP OS and reverse mergers over the
[25:20] course of the past 12 months here.
[25:22] And it does seem like when you go through the IPO route, you
[25:26] have a much more established brand.
[25:30] You know, you get all the media and articles written about your
[25:34] IPO and you've had the roadshow to meet with 100 or so clients,
[25:39] including test the waters meetings.
[25:40] So companies that do go the route of an IPO are, are almost
[25:44] again, kind of made men in, in our industry.
[25:47] And then of course, those that gone through the, the reverse
[25:50] merger route, you still have a lot of that publicity and, and
[25:52] work to be done for you on the other side of things.
[25:54] So just kind of urge the management teams to to stick
[25:58] with it after those deals are done and make sure they get
[26:00] their due. And you're right, Paul, over
[26:02] time, it'll be a more efficient market, but that's not always
[26:04] the case immediately, which is an opportunity.
[26:08] Let's continue with a little bit more of the news of the week,
[26:12] maybe less important, Paul, but you, you took note of the Siona
[26:15] update and of course they had a riff as as well as an update on
[26:19] their strategy. I know probably not too many of
[26:23] our listeners are, are that familiar or maybe even care that
[26:26] much with Siona. But but I think this is a, a
[26:29] great discussion that it Tees up around monopolies in our
[26:34] business and how Vertex has been so dominant in the CF space.
[26:37] So why don't you tell us what happened?
[26:38] Yeah, Siona company focused on a novel mechanism, strategy and
[26:43] cystic fibrosis. It's the market that has built
[26:46] Vertex into a behemoth. They've, you know, they've
[26:49] turned CF for the majority of patients into a livable disease
[26:53] with a long lifespan and health span.
[26:55] It's pretty tremendous. And interestingly, a lot of the
[26:58] Vertex work goes back to this cell based assay, which over
[27:04] time Vertex was essentially able to use the cell based assay and
[27:07] PK data for a drug and basically predict which drugs would work
[27:11] and which populations and how well they would work.
[27:13] And we've seen a number of companies come at Vertex and say
[27:16] they've cracked the assay and and they've come up short.
[27:20] It felt like Siona in, in my opinion, and I think the eyes of
[27:24] many in the market probably had the best chance of any of these
[27:27] companies at challenging Vertex, at least since Abby tried, you
[27:31] know, because they had really interesting assay data.
[27:34] They have a chief medical officer who was at Vertex,
[27:37] right? So they have that kind of
[27:39] pedigree and you know, I would say before I really thought this
[27:43] was going to work and I thought the debate was going to be more
[27:46] about the implications of the study because it was this 1B
[27:49] biomarker study. But the study failed on the ITT
[27:52] analysis. They showed very minimal drug
[27:54] effect for their drug on top of the Vertex regimen.
[27:57] And this week they did this post hoc analysis trying to explain
[28:01] why it failed. They put up a number of reasons,
[28:05] you know, a couple which I thought were a little bit harder
[28:08] to kind of, I guess really get that much conviction in.
[28:11] One was sort of related to PK outliers.
[28:13] The other one was related to this sort of complicated
[28:16] pharmacodynamic interaction between their drug and a Vertex
[28:19] potentiator. I mean, the one that I think
[28:21] people are most focused on is really that the exposures of the
[28:25] Vertex drugs seem to go down in patients who got the Siona drug
[28:29] and perhaps that massive Siona drug effect.
[28:31] But it's very hard to research. You know, I mean, it's just like
[28:35] it's, it's a hypothetical and, you know, I think we know the
[28:37] Vertex drugs are really powerful and reducing the exposure of
[28:40] those drugs could limit their efficacy.
[28:43] But how quickly you would see something like that in what was
[28:46] only a couple weeks study, I think we kind of have no idea.
[28:49] So I mean, ultimately, right, Siona's going to give it another
[28:51] go and they're going to try to do a switch study.
[28:54] It's a much simpler trial, but there's a much broader
[28:56] conversation here around how unique this vertex mode is.
[29:00] And I know others want to talk about that.
[29:02] And Eric, you know, when you kind of brought up that thematic
[29:04] discussion, I was trying to think about other companies I've
[29:07] covered, right, who had these special drug franchises that
[29:10] went from these mega high multiple stocks to ones where
[29:14] competition became this existential bear case.
[29:16] Alexion kind of came up to my mind first.
[29:19] But yeah, maybe I'll leave it to you to leave the conversation on
[29:21] that and others can chime in and I can maybe come back at some
[29:24] point. We'll just first maybe a
[29:27] specific question on Seattle versus Vertex is, is Siona a set
[29:30] back you think more of a of a Siona specific or idiosyncratic
[29:36] issue or does it really speak to the big mode that Vertex has and
[29:41] that mode is impenetrable? I think it speaks to Vertex's
[29:44] mode. I mean, I think the reality is
[29:46] like this is probably the 6th or 7th company we've seen come
[29:51] saying they could do something different that either Vertex
[29:55] couldn't. Like, I don't know how you don't
[29:58] give a tremendous amount of credit to like the Vertex San
[30:01] Diego group. And you know, Vertex has been
[30:03] telling people that this assay, this human bronchial epithelial
[30:08] assay, it's not simple. They've been able to kind of
[30:12] recreate the physiologic conditions of this disease in a
[30:15] dish. You don't know their
[30:17] methodology. You know, the Siona data on this
[30:20] assay, if you believed it looked tremendous and would have
[30:24] predicted a really significant drug effect.
[30:26] And I know people can be different than what happens in a
[30:29] dish. And this could be more nuanced
[30:30] and maybe the Siona drug does work.
[30:32] But I I mean I think after a number of these failures I I
[30:36] feel like the null hypothesis has got to be sort of like
[30:39] believe what Vertex is saying about what they built until
[30:42] someone proves the otherwise, right?
[30:44] I mean, I think we're all, you know, we're all somewhat
[30:45] historians of biotech and you'd have to say that Vertex's
[30:50] acquisition of Aurora, you know, which brought them all of this
[30:53] stuff back in 2001 is probably one of the most value creating
[30:59] transactions in the industry ever.
[31:02] I mean, it's like, if you think about what that brought them,
[31:06] it's crazy. What's it is?
[31:10] What's on this crazy deal, man? It's like 600 million.
[31:13] Dollar deal, it was like A5 hundred $600 million deal.
[31:16] They bought Aurora four, yeah. And think about that, think of
[31:19] that, think about, think about what's what's come out of that.
[31:22] It's just incredible. Right.
[31:24] What's been interesting about Vertex's approach is how they'll
[31:27] often advance kind of two competing molecules within their
[31:30] own pipeline and let them vie for, you know, the the pole
[31:34] position. We don't see that often in in
[31:36] biotype. But if you really want to own a
[31:38] therapeutic category, you have to be willing to cannibalize
[31:41] yourself. And I think a lot of companies
[31:43] make the investment instead of playing, you know, defense as a
[31:47] form of offense, they go out and play offense in other disease
[31:52] categories, sometimes completely different therapeutic
[31:55] categories. And how many times have we seen
[31:57] companies, you know, venture off into an area that's no longer
[32:01] their area, their domain of expertise only to struggle,
[32:05] right. You've seen Insight, Regeneron
[32:07] both kind of make try to try to move into solid tumor type
[32:11] oncology. I don't think, I mean
[32:13] Regeneron's had more success relatively than Insight, but
[32:16] compared to the expectations that I think each of the
[32:19] companies had and their investors had.
[32:21] And you see this time and time again that companies, you know,
[32:24] just decide to give up innovating in their own space,
[32:27] sometimes to their own, because that leaves the door open for
[32:30] another company. But you know, they're, they're
[32:33] making their own calculation. They're, they're calculating
[32:35] that an offensive maneuver is going to be more value creative
[32:39] to shareholders than defense. And it's it's always very
[32:41] nuanced. Well, interesting point that you
[32:45] raised Josh, that sticking to ones knitting might be part of
[32:49] building this mode. Interestingly, Insight's
[32:52] actually kind of coming back to MPNS and probably is now double
[32:55] down, double down on that area of medicine that created
[32:59] Jackify. Then Paul brought up Alexion on
[33:02] earlier. I mean we all want modes.
[33:05] We all want these everlasting businesses where we have to use
[33:09] an old term from one of our friends, domain domination, and
[33:11] and where the competition is almost futile other than
[33:16] sticking to our knitting. How how do we get there?
[33:18] What? What made Vertex truly special?
[33:20] Yes, you're right, they continue to focus on CF and more CF and
[33:26] iterated before anyone else was able to innovate.
[33:29] But is it? Is that the only lesson here or
[33:31] there are others? I.
[33:39] Feel like that people are stumped or trying to figure out
[33:42] like how like, you know, if we all knew what about Vertex
[33:47] allowed them to do this, then there probably would be a
[33:49] competitor, right? This is what's so hard to figure
[33:52] out. I mean, I think Vertex does show
[33:53] you that like our small molecule franchise is, you know, that is
[34:00] like much, much harder to emulate, right?
[34:02] I feel like we see more commoditized petition quickly
[34:06] and like antibody areas or they're like like, you know,
[34:08] it's like with Vertex like developing these small molecules
[34:11] that are so potent and dose so low and safe in combination,
[34:15] you're giving patients three drugs like that's I think that's
[34:18] I think that also just like intrinsically like very, very
[34:20] challenging. I don't know.
[34:21] I don't. I don't.
[34:21] Know if it'll ever be repeated? This wet vertex is done.
[34:25] It's just it's so you know, what's the combination of the
[34:29] disease and the, and the work they've done and it's just so
[34:32] hard. I mean, I always think about the
[34:34] other, the other super value creating transaction that I
[34:37] always think about is when Gilead bought triangle, you
[34:40] know, which brought them, you know, you know, brought up the
[34:43] antiretroviral combination therapies, right?
[34:46] We we started seeing that and that sort of that sort of set
[34:50] them off on that HIV dominance. But even there, right?
[34:55] I mean, they, you know, they, there was no, there wasn't a
[34:57] Moat there, right? I mean, there were other
[34:59] companies that were competing and have continued to compete in
[35:02] HIV And obviously Gilead has done very, has been very
[35:06] successful, probably the the most successfully HIV company,
[35:10] but it's not the only one, you know, So it's just it's God.
[35:14] I can't, I mean, I, you know, again, I can't think of another
[35:18] disease or an indication where you have this vertex situation.
[35:21] I don't know if you'll ever have that.
[35:24] Well, it's not. It's.
[35:24] Also not a huge mystery, right? Like it's, it's a single
[35:27] indication, right? It's genetic mutation.
[35:33] So they're only a limited number of pathways one can even
[35:36] contemplate to, to innovate around it.
[35:39] They found a very, you know, to the to the point that Paul made
[35:42] very complex. So it's hard to hard to
[35:45] replicate, but they also set the bar incredibly high.
[35:48] So it's hard to to beat that. So, yeah, whether this can be
[35:53] seen again, you know, I think you're, you're you're kind of
[35:56] looking for this type of a, of a dynamic or, or situations with
[36:03] similar enough dynamics. I think the other point here is
[36:07] that, you know, oftentimes a company like this would have
[36:10] just been acquired by pharma and we wouldn't, we wouldn't be so
[36:13] impressed about, you know, this, this big franchise that they've
[36:16] built because it would just be buried in, you know, many other
[36:19] lines of pharma type revenues. So Vertex also stayed
[36:23] independent, you know, as as part of the, the criteria for,
[36:27] for what they've accomplished, not to diminish it.
[36:30] It's it's amazing on the HIV front, you know, you pointed out
[36:34] Gilead and yes, they've had competition, but for, for the
[36:37] longest period of time, it's primarily been Gilead and GSK,
[36:40] you know, decades, decades, just like a duopoly.
[36:44] How come no one else? I mean, in, in HIV, you would
[36:47] have thought we'd have far more competition, but it gets to, you
[36:51] know, just the way you approach innovation, the way you approach
[36:53] the patient community, the way, you know, you, you attack a
[36:56] therapeutic category to really establish that dominance.
[36:59] You just, you just don't see it that often for for, I think in
[37:03] part because it is a unique set of circumstances that come
[37:07] together to create this. You know, I, I think it is.
[37:13] Interesting to note that both in HIV and and CF we're talking
[37:17] about multimodal therapy, we're talking about drug combinations,
[37:21] right? And I, I think if you have two
[37:23] or three components of the three component regimen that does give
[37:28] you a little bit of a competitive advantage.
[37:30] We've, we've seen multiple companies in the CF field fail
[37:33] because they didn't quite have all the necessary parts and and
[37:37] it's hard to get those necessary parts.
[37:39] So the mode is deeper when you have two or or three drugs at
[37:43] once. And certainly I think that's one
[37:44] lesson learned from HIV, maybe HCV 2 as well as CF that domain
[37:50] domination can happen a little bit more facile there.
[37:53] The other point to make is that both Gilead and Vertex just had
[37:56] a lot of time when nobody was looking at these indications, or
[38:00] maybe not less so in in HIV, but certainly in Vertex in this
[38:04] case, nobody was thinking about CF for probably 5 or 10 years
[38:07] while they were behind the scenes working through all these
[38:10] cellular assays and coming up with some good modulators.
[38:14] And that wouldn't happen today, right?
[38:15] Today's environment with China and other fast followers almost
[38:20] immediately upon the first signs of success, you'd have 234
[38:23] competitors. So I don't know to Adam's point
[38:25] that we will ever see this again or not it.
[38:27] It may be that we won't because the world is just much more
[38:31] competitive than ever spent. Any other thoughts on this
[38:35] topic? OK, well, let's move on to a
[38:41] couple of other updates from this week.
[38:42] Some, some news in the rare disease space, we had a drug
[38:46] approved scholar Oxapotegra mab. We had another drug approved
[38:51] Ultragenix's MPs 3B product that was developed in partnership
[38:56] with Abiona. And then Josh, you had some news
[39:00] on the development front from, from Rocket and Dannon disease
[39:03] as well. I don't know if we need to recap
[39:06] any of that. I'm I'm happy to spend a, a few
[39:09] minutes or, or you can Josh, but I think you know these updates
[39:13] in the rare disease space. So by the way, this is also the
[39:15] 10th anniversary of adipleursin's approval at the
[39:20] FDA. So, you know, 10 years ago,
[39:23] really the regulatory environment shifted in a major
[39:26] way with that approval of adipleursin, the first ever drug
[39:30] for Duchenne. And in some ways, you know,
[39:32] we're still dealing with the repercussions from that size and
[39:35] the confirmatory study is still not.
[39:37] Done. Yeah.
[39:42] Unbelievable. Right, so let's talk about rare
[39:48] diseases and, and what's going on in the space and, and Josh,
[39:52] I'll turn it over to you to to give us your thoughts and how
[39:55] you spent some time here a lot of.
[39:57] Time, you know, it was super exciting at the beginning with
[40:01] some of the breakthroughs in the innovators.
[40:03] Then the space got so crowded and so complex and so arguably
[40:09] lost. With the innovation and so you
[40:11] know, with a number of setbacks including efficacy setbacks,
[40:16] safety setbacks, manufacturing setbacks, but what I what I find
[40:21] so interesting right now and regulatory setbacks too.
[40:24] But, but we're kind of coming out of all of that all all
[40:27] together. Essentially, we have new
[40:30] programs in the cell and gene therapy in the rare disease
[40:34] space that are really generating phenomenal results.
[40:40] You know, I just just met with a company developing an
[40:45] optogenetic AV gene therapy essentially to treat blindness
[40:48] and, and, and patients are starting to see again, thanks to
[40:54] the persistence of those in the field, the power of gene
[40:57] therapy, the unique power of gene therapy.
[41:00] Because for some reason we're still stuck with AV vectors,
[41:03] which, you know, if you'd asked me 10 years ago if we'd still be
[41:07] talking about AAV vectors today, I would have said not a chance
[41:10] that it's such a limited technology.
[41:12] But on the other hand, it's a powerful and differentiated
[41:15] technology. There's, you know, emerging
[41:17] drugs to treat pain and overactive bladder type
[41:21] conditions, the rare diseases, the ones that you just
[41:25] mentioned, we're starting to see a return to regulatory
[41:28] flexibility, which is essential, critical.
[41:32] And, you know, kudos to those in the field who persevered
[41:36] because, you know, a lot of investors just just exited this
[41:39] whole space out of frustration. And a lot of companies exited
[41:42] the space out of the same frustration.
[41:45] But there's been this kind of die hard group of just wonderful
[41:48] people continuing to grind away because they realized that, you
[41:53] know, just just because there's some obstacles in the way
[41:56] doesn't mean they should stop trying to deliver these
[41:58] therapies for for patients. So we're kind of now in the
[42:01] phoenix rising from the ashes moment for this space.
[42:04] It is incredibly exciting. It's much smaller than it than
[42:08] it was. I think there are tons and tons
[42:10] of lessons that have been learned around CMC, around
[42:13] regulatory, around data, around Natural History comparator arms.
[42:18] So it's really exciting to see the these updates is really
[42:21] marking the progress that the field has been making somewhat
[42:24] quietly, but I think it's just going to get louder, louder and
[42:27] louder. I I love that there's a.
[42:31] Community around this space, as you mentioned, I, I really do
[42:34] think that people in the rare disease field probably
[42:37] collaborate and cooperate with one another sometimes even in
[42:40] the same indication, which is is bizarre to the rest of us.
[42:44] But but people do really put the, the best interests of the
[42:47] patient ahead of sometimes even commercial benefit.
[42:51] You saw that with Abiona, for example, which did not have the
[42:54] resources to put forward it's MPs 3 program and, and ended up
[42:58] giving that or essentially selling it to, to Ultragenix
[43:01] because they knew that, that they could probably take it
[43:03] forward economically and from a resource standpoint when, when
[43:06] Aviona couldn't. So, so that's wonderful.
[43:09] But you know, from a return on investment standpoint, Josh, you
[43:12] bring up an interesting point, which is that we, we haven't
[43:15] seen rare disease kind of take off in terms of an opportunity.
[43:20] Many of the companies in the space have have struggled.
[43:23] Pharma has kind of gone from in to out to in to out in terms of
[43:27] its interests in in the rare disease space.
[43:31] I mean in theory at least you would think this is a ready made
[43:35] opportunity for a great return on your investment, right?
[43:38] You can go after a defined genetic population.
[43:41] You can get your drug approved in a very short period of time
[43:46] on just a few patients treated in phase three studies.
[43:49] You've got all kinds of biomarker opportunities.
[43:52] You got to, as you mentioned today, at least a seemingly very
[43:56] friendly FDA and hopefully it stays that way.
[43:59] Yet again, we just haven't seen the, you know, kind of
[44:01] commercial successes. We haven't seen the Rockets and
[44:05] and rares kind of grow up and and become major players.
[44:09] Just wondering on the group's thoughts on why that might be.
[44:16] Or or any other comments? You guys have about right I I
[44:19] can jump in on. That on that point, I mean it,
[44:21] it was kind of it was like watching a train wreck in in
[44:24] action, you know, with a front row seat to how sloppy and
[44:31] excessive the innovation was. Because recall, this was at a
[44:34] time, you know, in part through the through the bubble and
[44:37] excess funding and limited discipline and limited
[44:40] appreciation of all the challenges.
[44:43] You know, some, some just unforeseeable because it was all
[44:49] such new territory, right? But there are myriad challenges
[44:53] looking back that I don't think most most of us realized and
[44:56] appreciated ranging from all the things we talked about CMC
[45:00] regulatory. And then at the end of the day,
[45:02] you know, the one big contrast and, and point of frustration
[45:05] for me, right, is, you know, if you take a drug that with the
[45:09] the recent price creep of new product launches, you know, a
[45:14] new rare disease drug can easily cost 300 to 600, even more $1000
[45:19] per year. And we had patients on on that
[45:22] drug for five years, 10 years, 20 years.
[45:25] You know, that's a per patient value that can be well above 10
[45:28] million, you know, potentially substantially higher than that.
[45:32] And you look at the prices of cell and gene therapies, they've
[45:35] been in the hundreds of thousands of dollars, low single
[45:38] digit millions. You're not capturing the same
[45:40] value. I never really heard any
[45:43] discussion about that and realization that a market that
[45:47] can be very lucrative for, you know, a chronic therapy, you
[45:54] know, 500 patients on a chronic therapy, if it's priced high
[45:57] enough, is actually the nucleus for a real business model
[46:00] because they're going to be on that drug for a very long period
[46:03] of time. Whereas in gene therapy, when
[46:05] you're only capturing 1 to 2 million per patient times, you
[46:09] know, 100 or 200 patients, you no longer have a viable,
[46:13] sustainable business model. And I think that was one of the
[46:15] big prices and lessons that the industry had to learn.
[46:19] And now as we're seeing it regain and recover, we're not
[46:23] seeing as many. Here's a gene therapy, you know,
[46:26] for an important unmet medical need that affects 100 patients.
[46:29] Now we need to figure out how to bring those forward, how to fit
[46:33] them into the capitalist drug development model that we have.
[46:38] One of the ways to get there is to raise the price even higher
[46:42] to make it more commensurate with what the what the value of
[46:45] an annuity stream would be. But everyone is so scared to do
[46:48] that. No one wants to be the one that
[46:50] comes out with a $10 million product all at once, even though
[46:54] the lifetime value of a, of a patient on a chronic therapy
[46:57] could be way above that. So we're still trapped in that
[47:00] kind of dynamic of reimbursement.
[47:02] That's also been a factor. Well, thanks for that I I
[47:07] realize. I've been saying MPs 3B when the
[47:09] approval was for San Felipe, which is MPs 3A.
[47:12] So apologies for that misstatement.
[47:15] But Adam, I know we're not going to discuss rare disease is
[47:18] without getting your views on what's going on here.
[47:21] Oh, you want to end? It with me that is that you want
[47:23] you want me to say something mean.
[47:25] No, no, I mean like you, you know, obviously you brought up
[47:29] the you know, the the end of person and while it's great and
[47:33] all but you know, I think it does raise issues and with the
[47:37] both with the rare disease community and these drugs in
[47:39] that, you know, whether they're you know how we how we confirm
[47:45] or prove benefit of these drugs. And I know the difficulties of
[47:50] of testing them, small patient populations and you know, the
[47:55] need for regulatory flexibility, but there has to be some kind of
[47:59] balance there too, right? And at the end of the day, it's
[48:03] these are, you know, you want all these feel good stories,
[48:06] sure, but you know, you also want drugs that are safe and
[48:09] effective. And I think we have, we have
[48:12] instances, we have examples where, you know, drugs are out
[48:15] there where you're not really sure if they really if they do
[48:18] work or not. And we have to figure out a way
[48:21] to make sure that patients are getting drugs that work for
[48:24] them. Don't you think what the FDA is?
[48:27] Doing now Adam with like the confirmatory study expectations
[48:31] is like a big step forward. I think so.
[48:34] Yeah, I know. I, I do, I think that that's
[48:36] helpful, right? I think, you know, you, you
[48:38] know, trying to, you know, adding, adding a stick to the
[48:42] carrot, so to speak, you know, and making sure that companies
[48:46] follow through on their commitments.
[48:49] You know, you know, if you're going to approve a drug for
[48:52] accelerated approval and you know, you, you have to have that
[48:55] confirmatory study, you know, well underway and whatever,
[48:59] however you define that and make sure that those studies get done
[49:02] in a reasonable amount of time. And then, you know, just as
[49:06] importantly as if if those studies don't.
[49:09] Confirm the benefit. Then then you know, then what do
[49:12] you do? You know, I think, you know, I
[49:14] think you remove those drugs from the market and we, and I
[49:17] think that's part of the problem here is that we've, we haven't
[49:20] seen the FDA willing to sort of be maybe be the bad guy in some
[49:24] way, in some ways there as as often as they should.
[49:29] And you know, we, I think there just seems to be a lot of excuse
[49:32] making when these confirmatory studies fail that, you know,
[49:36] it's, oh, it's the study, it's not the drug.
[49:39] Well, you know, at some point it's the drug.
[49:47] Well, this has been a tough. Conundrum for many, many years,
[49:50] at least 10 years exact. So I'm not sure we're going to
[49:53] solve it here, but a good discussion nonetheless.
[49:56] Thanks for those comments, Adam. And yeah, I don't, I don't know
[49:59] what you would do, whether you pull it, of course or not.
[50:01] Thankfully for DMD patients, I think there's a much better
[50:05] yeah, that is that. Is good news, exactly so.
[50:09] We can let the market dynamics play out and and hopefully
[50:12] patients with Exxon 51 skippable mutations.
[50:15] We'll we'll see a better therapy soon enough.
[50:17] OK, we just got a couple of more minutes.
[50:21] I don't know what we want to talk about.
[50:22] Josh, you want to talk about some of the measles stuff that's
[50:25] going around? Goodness.
[50:26] Oh, Eric. Well, well said there, Eric.
[50:30] Yeah, measles is going. Around there's a lot of
[50:33] controversy and you know, folks who have there, there's even
[50:36] debate as to whether people who died, quote, with measles died
[50:40] of measles or or not, which is just beyond misguided,
[50:45] frustrating, disappointing. You know, you saw RFK junior
[50:48] back in front of his old group of the, the, the, I think
[50:52] not-for-profit group that was a very anti vax and they, they
[50:57] obviously welcomed, welcomed him back.
[50:59] I mean, it's, it's so disappointing to see all the
[51:01] damage, you know, that's been caused to the FDA, to the CDC,
[51:06] all in pursuit of like pseudoscience type paranoid
[51:11] thinking around, around science and innovation.
[51:15] On the other hand, you know, you have to acknowledge like, like
[51:18] vaccines have some limitations. Net, net they do far more good
[51:23] than harm. But you know, there it's
[51:26] actually a very interesting time now maybe coming back coming to
[51:29] it to in Vivid that we hadn't been necessarily planning on on
[51:33] talking about but but, but it's relevant as part of this
[51:35] discussion and Vivid is developing monoclonal antibodies
[51:39] as prophylaxis against respiratory pathogens.
[51:43] And very recently they signaled that HHS and FDA are going to
[51:47] give them an accelerated approval path to bring a their
[51:51] COVID prevention antibody to to market.
[51:55] You know, important because there is a lot of vaccine
[51:58] hesitation around COVID and, you know, some of it's for good
[52:01] reason that, you know, there can be side effects.
[52:03] The vaccine efficacy seems to have waned.
[52:06] The durability seems to have waned.
[52:07] The reactogenicity is an issue. And, you know, in this setting,
[52:12] a prophylactic monoclonal antibody could be a superior
[52:15] option, right, in terms of safety, efficacy, durability,
[52:19] tolerability. And so why shouldn't we embrace
[52:22] change when it comes to vaccines?
[52:25] So all the pro vaccine people that are so dug into this
[52:28] technology perhaps would benefit from expanding their own
[52:31] apertures. And considering that maybe this
[52:33] is a time to think about, you know, moving to an alternate
[52:37] technology, one that may not have negative sentiment, one
[52:41] that will need the the support and protection of the scientific
[52:44] community to keep it immune to, to, you know, whatever anti vax
[52:50] rhetoric may emerge around a protective antibody.
[52:53] And so a lot of change, all happening at the same the same
[52:57] point in time. But the end that the ultimate
[53:01] message here is that it's all about innovation still, even in
[53:04] the vaccine and related spaces. Well, I I hope we don't have.
[53:11] To move or get forced to move for new technologies just
[53:13] because of the Maha movement. Look all for bringing in new
[53:18] stuff all the time if it's better.
[53:20] But but we do have vaccines, right?
[53:22] And and they're working extremely well and in this day
[53:24] and age, nobody should be dying of measles.
[53:26] So let's just first call a spade a spade.
[53:29] Josh, I think you were trying to recall the very inappropriately
[53:32] named Children's Health defense organization, which which RFK
[53:38] junior is now back in touch with after trying to distance himself
[53:42] himself himself from the anti vax movement.
[53:45] I, I don't quite understand it. I, I thought that we got through
[53:49] this earlier in the year where it was very clear that
[53:52] politically at least, it was a liability for RFK junior to be
[53:56] associated with, with the anti vax movement to the degree he
[53:59] was. Obviously we know, we know who
[54:01] he is and, and we know what his stripes are.
[54:03] But for much of this year, he's kind of been put into a bit of a
[54:09] closet with regard to his anti vax statements.
[54:12] And, and yet in the last couple weeks, it seems like he's come
[54:16] out of that closet. I think stat news Adam did a
[54:18] nice piece on how he appointed some, some new folks to some
[54:21] kind of a vaccine advisory panel.
[54:23] And we're seeing him go back to the Children's Health defense
[54:26] organization and, and tout the anti vax sentiment, all while
[54:31] people are dying in Pennsylvania.
[54:33] So I don't know, maybe this will come out in the wash and and
[54:37] it'll be seen as political liability heading into the mid
[54:40] cycle elections and and we'll be done with it again.
[54:42] But but all a bit of a worry and well, you know it'll be.
[54:45] Interesting to see if all, if any of this comes up next
[54:48] Thursday. You know, the Senate's holding
[54:51] their first confirmation hearing for Heidi Overton, the FDA
[54:56] commissioner. So we'll see if this comes up.
[55:00] Well, thanks for flagging. That that'll definitely be worth
[55:02] the price of admission as well. OK, I think we're at the top of
[55:06] the hour and out of time, but let me just close by thanking
[55:10] our listeners, Adam Forrestein, Paul Matisse and sorry our our
[55:14] Co host Adam Forrestein, Paul Matisse and and Josh Schemer and
[55:18] and thank all of you guys for for listening in to another
[55:20] edition of the Biotech Hangout.

Shows are listed from Apple Podcasts' charts and directory, and Podcast Index where it is used. Every show and episode belongs to its publisher and plays from the publisher's own feed; this site hosts none of them.