# Episode 193 - August 14, 2026

An episode of [Biotech Hangout](https://invisiblenetwork.ai/podcast/1568911820), 14 Aug 2026, 59 min.

Audio: https://anchor.fm/s/55bdff38/podcast/play/124232532/https%3A%2F%2Fd3ctxlq1ktw2nl.cloudfront.net%2Fstaging%2F2026-7-14%2F429855624-44100-2-bdcd4b0906ae8.m4a

On this week's episode, Chris Garabedian, Graig Suvannavejh, Paul Matteis, and Tess Cameron open with the biotech market. They describe 2026 as strong but discerning, with 19 Nasdaq IPOs this year and generalist investors still not fully back. The co-hosts also cover recent launches and raises, including Boulevard Bio's launch with Deerfield backing and Epicrispr's $90 million Series C raise for FSHD. In data news, Tenax's HFpEF candidate missed its primary and secondary endpoints, while Sionno's cystic fibrosis drug missed its biomarker target, sending shares down 90%. On the dealmaking front, Jazz acquired epilepsy biotech Actio for $820 million upfront to deepen its rare epilepsy pipeline, and Revolution Medicines struck a RAS-inhibitor partnership covering China development alongside a PRMT5 clinical collaboration. Elsewhere, AbCellera raised $200 million on positive Phase 2 vasomotor data, and Silence Therapeutics raised $175 million on positive polycythemia vera data, while Definium posted positive Phase 3 data for its psychedelic GAD candidate, and Cullinan/Taiho did the same for their lung cancer drug zipalertinib. In regulatory news, Neurocrine's Prader-Willi drug faced new safety scrutiny after reports of seven deaths, and Amgen's Tavneos was pulled by the EMA over misleading trial data. Belite Bio's tinlarebant earned FDA priority review for Stargardt disease, while rival Tarsus acquired Alkeus for about $800 million. The episode closed with a debate on expanding the COINS Act into biosecurity. This episode aired on August 14, 2026.

## Transcript

[0:00] You're listening to Biotech Hangout, a live and unedited
[0:03] weekly discussion of all the latest news in our industry with
[0:06] a group of biotech leaders and experts.
[0:08] I'm Chris Garabedian and my Co host today are Greg Suwanoway,
[0:12] Paul Matisse and Tess Cameron. For more information about our
[0:16] hosts and guest speakers or to listen to the most recent
[0:18] episode, please go to biotechhangout.com.
[0:23] So, so Tess, we, we often like to just start with a pulse on
[0:26] the markets. Would you like to share how are
[0:29] you thinking about the public markets as we sit here today?
[0:33] You know, public markets have been really positive.
[0:36] You know, the XBI, you know, has been, you know, performing,
[0:40] performing really well. And you know, I think what's
[0:43] important though is that we are seeing strong performance, but
[0:47] we are also seeing kind of selective performance, right?
[0:50] So it's not, it's not that like everything is going up, right?
[0:54] It's like IPOs are performing well.
[0:56] But like also the companies that are going public are like really
[0:59] strong companies with like really strong data, you know,
[1:03] companies that have, you know, negative updates, which we'll
[1:08] talk about a couple of those. You know, those stocks are going
[1:11] down pretty dramatically. So it's, you know, the market is
[1:13] the market is strong, but it is also discerning, you know, which
[1:18] is, which is important. So I think like healthy, you
[1:21] know, healthy, but discerning is like a really good way to
[1:24] describe. This well, I kind of look at it
[1:26] when the markets are on fire, right?
[1:28] Everybody can look good as a biotech investor, but it, it's
[1:31] the specialty investors, right, of which are a perceptive
[1:35] others. You know, this is where I think
[1:38] specialty investors thrive, right?
[1:40] That discernment and understanding, right, where
[1:43] value goes and when the markets are behaving, whether it's on
[1:46] the upside or the downside, I think that favors the, the long
[1:50] standing, right? Decades long investors have been
[1:53] doing this for a long time and I think that's a good thing for
[1:56] our industry. Yep, absolutely, absolutely.
[1:59] I think we're seeing, you know, at a general level, right, we're
[2:03] seeing rational responses to data and that's that's really
[2:05] important. And you've talked a little bit
[2:07] about which we've noticed this past year the kind of a
[2:13] bifurcation of tech and biotech. I mean we've seen, you know,
[2:17] Anthropic going out in an IPO worth trillions or Overtron,
[2:22] SpaceX, etcetera. And so these tech investors are
[2:25] very different and the general investors that invest in tech.
[2:28] And so we've seen this separation.
[2:31] And I know you, you had some thoughts on that.
[2:34] Would love to hear your perspective on that.
[2:37] Yeah, absolutely. So there was a great article
[2:39] this week from the Wall Street Journal that I loved that was
[2:42] called healthcare investing is now an AI short in disguise, you
[2:47] know, about how, you know, healthcare companies are really,
[2:51] you know, kind of treating like opposite to tech.
[2:54] And, you know, I think, I think there's, you know, for the past
[2:57] year, you know, year and a half, there's been this kind of like
[3:00] flow of funds question, right? Which is like, Oh my gosh, it's
[3:03] just like all the money is like going into AI and like, you
[3:06] know, where is everyone getting their money from?
[3:08] Are they like, you know, getting money out of Lilly?
[3:10] Are they getting money out of like, you know, other companies,
[3:13] you know, to, to other healthcare companies to, to kind
[3:15] of invest that in AI. And, you know, the Wall Street
[3:19] Journal article was kind of, you know, taking a bit of a, you
[3:22] know, different view on that. Where like, you know, when we
[3:24] look at some of the, you know, AI related sectors that have
[3:28] done super well, it's a lot of the, you know, picks and shovels
[3:31] companies that are like going into like the AI infrastructure,
[3:34] like, you know, CHIP and other companies.
[3:37] And like those are really cyclical.
[3:39] Healthcare is like not cyclical. So they were kind of pointing to
[3:43] this like, you know, hey, actually like healthcare stocks
[3:47] are kind of like doing a bit better when like AI is doing
[3:50] poorly. And it's that people, you know,
[3:52] looking for, you know, looking for, you know, something of a
[3:55] hedge on their AI exposure, which I thought was really
[3:59] interesting. It's like we still don't know,
[4:01] like it's actually how much money our AI firms like going to
[4:06] make. What's that revenue curve gonna
[4:08] look like? And people are going to need
[4:10] medicines, right? So that is a good it was a good
[4:18] reminder with a nice punchy title.
[4:21] Yeah, who who knew that we might be considered maybe a safer,
[4:24] more predictable type of investment strategy.
[4:27] I think 2025 years ago, right, you know, we saw the.com bubble
[4:31] combined with the genomics bubble.
[4:33] But I think this is a very different mature market for the
[4:37] IP OS today. And so I think there there are
[4:40] some good distinctions to draw from when you know we would be
[4:44] lumped into these tech. We can't predict it.
[4:46] I think frontier there is a lot of uncertainty.
[4:48] So I think that that's an interesting one to watch to see
[4:50] if we end up urging at any point with tech in terms of how we we
[4:54] trade as a sector. Yeah.
[4:56] Yeah. I think it's also interesting
[4:57] because like just, you know, again, going back like a while,
[5:00] and this is obviously after like the tech, you know, tech bubble
[5:03] and everything likeyouknow.com bubble.
[5:06] But you did see like just more consistent multiples, right, and
[5:10] kind of forward growth rates. And now there's like forward
[5:13] growth rates have obviously just diverged significantly.
[5:17] And hey, I guess like healthcare looks cheap on like a relative
[5:20] basis. Yep.
[5:23] So before I talk about the private markets, Paul, Greg, do
[5:26] you guys have any, anything to add to the to the public market
[5:29] sentiment? I mean, I agree that like it's
[5:33] sentiment continues to be pretty good.
[5:36] The fundraising environment looks pretty good, But you know,
[5:39] unlike past periods like this, right?
[5:41] Like I still think most investors and and myself like
[5:44] don't feel like things are, are super overheated, right?
[5:48] And you know, like Tess said, like there's, there's variance
[5:51] in market in this market that is going that's going in both in
[5:55] both directions. I will say that like, despite
[5:58] this being like a good year for sort of biotech sentiment and
[6:03] vibes, like when I talk to different investors, it hasn't
[6:06] been a great year for for everybody.
[6:09] You know, depending on your investment strategy, depending
[6:12] on if you owned some of the takeouts that happened this year
[6:15] were really big takeouts for companies that didn't have like
[6:18] an imminent catalyst. Not everyone was there.
[6:20] And you know, maybe it goes back to what you were saying, Chris,
[6:22] right? Like this is a market that feels
[6:25] healthy, but it's also kind of like an inefficient stock
[6:28] pickers market with an IPO window that's open.
[6:32] And it feels like a bunch of these factors are just like
[6:35] usually all these factors aren't in play at the same time, right?
[6:38] Usually when we've got an open IPO window, a big fundraising
[6:40] window, it's like everyone's just making a ton of money.
[6:44] And I don't, I don't feel like that's the case.
[6:46] Anything to? Add.
[6:49] Yeah, I think I'll add that generally, yes, I agree with
[6:52] what Tess and what Paul had to say.
[6:54] BXBIS is year to date still outperforming the broader
[6:58] market. You know, biotech is has been
[7:01] the darling within healthcare. So I think fundamentals are
[7:04] still really good. But biotech is always a nervous
[7:08] type of a market. There's always skittishness,
[7:10] especially around individual names.
[7:13] You know, you still see some, you know, disappointing data
[7:16] readouts and I think we're going to talk about two very high
[7:19] profile ones, not not too far away.
[7:21] But you know, it is as Paul said, it's still very much a
[7:24] stock pickers market. I know I've been asked this on
[7:27] prior biotech Hangouts whether you know, the generalist
[7:30] investor is back in yet. And I don't really know that the
[7:34] generalist investor is fully back in.
[7:36] I mean, I think they're seeing, you know, the IPO performances
[7:40] year to date have been pretty, pretty solid as as Tess
[7:44] mentioned, you know, we've had 19, I think by my count, 19
[7:47] issues on NASDAQ this year so far, which is a, a really
[7:52] healthy number. So again, I'm, I'm, I'm very
[7:54] positive what we're seeing in the space.
[7:56] But the same time like you know, any day is a is a different day
[8:00] and you know, our stocks are, are very volatile well.
[8:02] Well, some are predicting as the tech gets overheated and frothy
[8:07] or the perception of that, that they might as test was described
[8:10] might look to biotech to as a, as a safer shelter for that.
[8:15] So we'll we'll have to see. But I, I agree it hasn't begun
[8:17] in earnest. And I, I think that's something
[8:19] to watch maybe, maybe just. One, maybe just one other point
[8:24] and I'd be interested, you know, Paul or Greg, you may have more
[8:27] insights into this. But like as you're talking about
[8:29] fun performance, Paul, like I think that short exposure has
[8:32] also played like a pretty significant role in that where I
[8:35] think, you know, some of the neutral stuff has been harder
[8:38] to, you know, harder, harder to kind of manage this year than
[8:43] you know then then maybe in the past.
[8:46] Yeah, I mean, I the subtext of what I was saying is I don't
[8:50] have the exact numbers, but my perception was that Chronetics
[8:54] Apogee, some of these companies were pretty heavily shorted
[8:57] stocks, not necessarily because they were seen as bad companies
[9:01] or anything like that, but you know, they were kind of seen as
[9:03] companies where like clinical pieces of the story were already
[9:06] de rest. There wasn't a major binary
[9:09] event coming up. And I think what people listen
[9:11] this call have to understand is that there's a whole, there's a
[9:14] whole bunch of biotech funds out there that have some mandate on
[9:17] the percent short they have to be some of which it has to be
[9:20] completely market neutral. And the reality is those funds
[9:23] often look for certain companies that lack catalysts, but are
[9:27] liquid stocks to kind of be sure to fund their lungs, right.
[9:31] And so, you know, I, without talking to anyone specific, I
[9:35] mean that that type of model, I think has been more has been
[9:38] more challenging this year. You know, probably the long only
[9:41] model hasn't been as challenging.
[9:43] And then, you know, sort of in between, right?
[9:45] It depends on have you been in the right, in the right places,
[9:48] with the right events or the right takeouts.
[9:50] Great, great, great comments. So on the venture side, you
[9:53] know, we often track, you know, and lag the public markets and
[9:58] you know, overall I think it's been a safe place for venture
[10:00] investing this this week. We've seen a range of venture
[10:03] investors, I'll just name a, a, a few without going into the
[10:06] details, but we've seen three deals that were announced this
[10:11] week. We had Blvd.
[10:14] Bio, which was a spin out of a Deerfield company, so company
[10:19] creation with a good amount of capital of behind it.
[10:23] We've also seen what was it EPI, what is it EPI CRISPR, which is
[10:29] a series C $90,000,000 going after FSHD.
[10:33] It's been a challenging clinical indication, an epigenetic target
[10:36] silencing Dux 4. And then we also saw another on
[10:42] my, my notes now are escaping me here, but there was a Series B
[10:46] for, to support a phase three pivotal study for allosteric
[10:51] inhibitor of AKT 1/2 and being developed for the treatment of a
[10:56] rare disease HHT. And again, so that the venture
[11:01] investments are thriving. We're seeing a lot of a range of
[11:04] deals, but there's still a lot of opportunities that are later
[11:07] stage, clinical stage and we haven't seen the real big tick
[11:10] up in seed and Series A. So aside from some specific
[11:14] company creations like Blvd. I think you know, Q2 we saw the
[11:18] numbers go down in terms of the number of seed and Series A
[11:22] rounds. So I'm looking for the next year
[11:24] to see that start to pick up and a little bit more risk tolerance
[11:27] in the venture community. But you know, the other piece
[11:29] which was alluded to is the pipe markets and reverse merger
[11:33] candidates are also buttressing the health of the market in my
[11:37] opinion. And it's almost like every time
[11:40] there's a failure of a, you know, largely single product
[11:43] binary biotech that has a failure, there's almost a frenzy
[11:47] of like, OK, are they going to be the next reverse merger
[11:50] candidate? You know, let's call the banks,
[11:52] let's, you know, figure out if this can be a pathway to go
[11:55] public. And I think this is partly
[11:57] because there's a lot of the public funds, you know, many of
[12:00] them might have some PTSD for all of the private crossover
[12:02] rounds when the market turned against them that they were
[12:05] sitting on that actually prefer either a direct IPO or a pipe
[12:11] reverse merger that will, you know, fast track the public
[12:15] trading as opposed to the traditional crossover mezzanine
[12:18] round, you know, which are still happening.
[12:20] And so I just wanted to highlight that dynamic, which I
[12:23] think is healthy. And so even when there are
[12:25] failure, they could end up working well for those companies
[12:33] ending up, you know, with a reverse merger.
[12:35] And two of those companies were announced Monday of this week.
[12:38] Tess, you want to cover 10X and then I'll have Paul cover Siona.
[12:43] Yeah, absolutely. So 10X was developing a drug for
[12:47] hep, which is a notoriously very, very challenging
[12:51] indication. But you know, also enormous,
[12:55] also enormous unmet needs, also enormous unmet needs.
[13:00] So very, you know, very highly anticipated readout, you know,
[13:04] so the company reported earlier this week that they did not meet
[13:08] the primary endpoint of 6 minute walk versus placebo.
[13:11] They didn't meet the secondary endpoint of KCCQ, which is kind
[13:15] of a, a patient, you know, patient reported outcome.
[13:19] Actually let me take that back. I can't remember if it's a
[13:22] patient reported outcome, but it's it's really measuring, you
[13:25] know, measuring symptoms. They did see a reduction in, you
[13:31] know, NTNT proven P And you know, what was kind of, you
[13:37] know, interesting here is that, you know, there was a there was
[13:42] some kind of treatment effect by, you know, disease severity
[13:46] where it seemed like, you know, in healthier patients.
[13:51] You know, it there there was kind of a question around, you
[13:56] know, did this did this actually become like kind of harmful and
[13:59] like, you know, healthy patients just based on the, you know,
[14:02] decline in six minute walk that they observed throughout the
[14:05] trial? So I think, you know, a lot of a
[14:08] lot of open questions, but you know, clear kind of myths on
[14:11] the, on the primary and secondary and and maybe some,
[14:15] you know, questions about, you know, the the observations on NT
[14:20] probe NP, which they pointed to was like a, you know, a
[14:26] mechanistic kind of rationale to, you know, believe, believe
[14:30] this is having a, you know, the right mechanistic impact.
[14:34] And you know a question of of how that would would translate.
[14:39] Test. Do you think there's like a path
[14:40] forward for this given that context?
[14:44] I mean, I think it's open question, right?
[14:47] But like, I think it's I think it's tough, right?
[14:49] It's like you've what is what does that suggest?
[14:51] Does it have to be a much longer trial?
[14:52] Does it have to be like a different patient population?
[14:55] Does it have to be like a, you know, a much like a larger trial
[14:59] to to kind of see the effects, right?
[15:01] Like it, it certainly doesn't seem like a straightforward path
[15:05] forward, right? I would expect that they're, you
[15:08] know, really, you know, kind of getting into the data and, and
[15:11] trying to think about like, you know, what, you know, is there
[15:15] is there a path forward? And you know, what they could do
[15:17] next. So they're going to meet with
[15:19] the FDA, right? They have a, they said they're
[15:21] going to look at a type C meeting.
[15:23] They'll be with the EMA, you know, see what can they do to
[15:26] like enrich patient population or their subgroups, right?
[15:29] Maybe they focus on this like more severe patient population
[15:32] where like there was a benefit in, in, you know, 6 minute walk.
[15:34] But I think, you know, you'd want to understand like
[15:37] mechanistically, like, does that make sense?
[15:39] Right. And you know, really have
[15:42] confidence that this would work in like a larger trial.
[15:44] So I think I think that's really going to depend on some of those
[15:48] additional analysis. Very curious in your thoughts.
[15:53] No, I mean, I'm not as close to it.
[15:54] I just know this point's like super controversial and it's
[15:59] always tricky, right? When you like don't meet the
[16:01] expectation, like can you kind of like can you pivot right and
[16:05] still like keep, keep the show going with the program?
[16:08] So yeah, then thank you. That's super interesting.
[16:10] Paul, you want to cover Fiona? Yeah, this one was such a bummer
[16:16] for a few reasons. One, I mean, it's a bummer that,
[16:18] you know, from a cystic fibrosis disease perspective, even though
[16:21] Vertex has made crazy, crazy strides in making this a livable
[16:25] disease, it's still a bummer that there's not, at least for
[16:28] now, seemingly going to be another option with a new
[16:30] mechanism to. I mean, the Siona team is an
[16:33] awesome team of, of good people who I think really, you know,
[16:37] did their best to try to make this work and also be
[16:39] transparent. And, and 3rd, I really thought
[16:43] this was going to work. So, you know, you never this,
[16:46] this industry always like really humbles you.
[16:47] But basically Siona was developing a, or is I guess
[16:50] still developing a novel mechanism to try to stabilize a
[16:53] domain of the CFTR protein called NBD, one that the gene
[16:59] CFTR gene is mutated in cystic fibrosis patients don't make
[17:02] enough functional protein. You know, vertex for 90% of
[17:05] patients that have at least one FOF 5 O 8D mutation has made
[17:09] this a very livable disease. But not everybody responds to
[17:13] the same magnitude. There's some patients that have
[17:15] side effect issues. And so Siona was kind of trying
[17:17] to come into the game to offer a novel option.
[17:20] And the study that they were running, you know, was a very
[17:22] clever study. They were essentially because
[17:24] it's hard to, you know, enroll the placebo-controlled trial in
[17:29] this disease. And also you kind of try to
[17:32] think about what's the right patient to select, what's the
[17:34] right duration. They had this clever idea of
[17:36] adding one of their NBD one correctors on top of patients
[17:39] who inadequately responded to Trikafta and to look at sweat
[17:43] chloride reduction as a pharmacodynamic biomarker since
[17:47] this has been super historically predicted for the vertex drugs
[17:50] and for this disease, it's used as like a diagnostic, right?
[17:53] It's used in newborn screening. So it's a pretty uncontroversial
[17:56] biomarker. They had guided to hoping to see
[17:59] at least a 10 millimole change. And you know, this is our work,
[18:03] not Siona's, but we had tried to do we tried to kind of recreate
[18:06] what their PKPD modeling could look like ahead of that, right?
[18:09] You know, try to figure out why did they guide to 10?
[18:11] And you know, I think the answer was that the the confidence
[18:14] interval suggested that 10 could potentially be conservative,
[18:18] right, depending again on the study population and things like
[18:20] that. So they didn't meet that bar and
[18:22] they didn't come close. They saw only a 1 mill mole
[18:25] reduction far from stat sig stock was down 90%.
[18:29] You know, the investor expectations were pretty high.
[18:31] I think people were even debating if 10 would have even
[18:34] been enough to move the stock given how much higher
[18:36] expectations had been. And so, you know, I mean, the
[18:38] question kind of like was test was just talking about like the
[18:41] question with this one is sort of is there a path forward?
[18:45] You know, they think there could have been this potential drug
[18:48] drug interaction with Trikafta. And so if patients were, you
[18:51] know, getting the Siona drug and then the Trikafta exposures were
[18:54] going down, I mean, that could easily erase a pharmacodynamic
[18:57] effect. But, you know, it's unclear if
[18:59] they're going to be able to look at this data set, which is only
[19:02] 14 patients and find an an unequivocal explanation.
[19:06] The other nuance is that the end game of this company was really
[19:09] never to develop a combo drug on top of Trikafta.
[19:12] The end game was to develop their own combo therapy, but use
[19:15] this data from this Trikafta study to be able to kind of
[19:19] better understand and calibrate their PKPD modeling, which all
[19:22] stems back from this human bronchial epithelial cell assay.
[19:27] The CFBE assay that Vertex had used for years, that Siona had
[19:31] thought that they had kind of cracked the way Vertex does it
[19:34] from a predictiveness perspective.
[19:36] And you know what, we'll never, I don't know if we'll ever
[19:38] really know 100% if it was the assay or something else.
[19:41] But you know, that ultimately did not translate.
[19:43] And so tough situation for a really smart and good group of
[19:47] people. You know, I think they're going
[19:49] to try to come back at some point in the not so distant
[19:51] future with a better explanation of what happened here and, you
[19:54] know, maybe or maybe not a plan on trying to run another study.
[19:58] They have this other MBD one corrector.
[20:00] They have these other molecules that could combine it with.
[20:02] But yeah, total bummer. And for this one, like, you
[20:06] know, this company as a stock was somewhat controversial. 1,
[20:09] because it's biotech, and two, because I think there's a debate
[20:12] on the street of just, hey, Vertex has set such a high bar,
[20:15] right? Like what, what, how much more
[20:17] do we really need? But the perception was the study
[20:20] was pretty likely to work. And so, you know, big
[20:22] unfortunate surprise on Monday of this week.
[20:25] All right, let's move. There was a deal this week and
[20:28] again what I've been encouraged by in the marketplace is these
[20:32] bolt on acquisitions and these I'd call it you know half a
[20:35] billion to you know 3 billion acquisitions and then you've got
[20:40] the kind of bigger acquisitions from the 5 to 15 billion, but we
[20:43] had one this week. Greg, you want to start and
[20:46] maybe Paul can comment on, you know the the acquisition by
[20:49] Jazz? Yeah, thanks so much.
[20:51] So Jazz Pharmaceuticals, which is a interesting company in that
[20:56] it's a diversified company focused on both neuro and
[21:01] oncology now, but they beefed up their epilepsy franchise efforts
[21:06] by buying a company called Actio.
[21:09] It's a deal that's about in total dollars about 1.3 billion.
[21:12] It was 820 million upfront with an additional 500 million in
[21:18] regulatory and commercial related milestones.
[21:22] I like this deal for Jazz, which I don't cover.
[21:24] I used to cover it in the past, but I don't cover it now.
[21:27] But they are really beefing up their epilepsy, epilepsy
[21:30] franchise efforts. If you remember, they got big
[21:32] into the space by buying GW Pharma.
[21:35] That was I think back in 2021 that might have been like a $7
[21:39] billion deal for their drug Epidiolex, which was a very
[21:44] controversial drug at the time. But that being said, I think
[21:48] Epidiolex is like over a billion dollars in annual sale.
[21:52] So an incredibly very successful drug for jazz.
[21:56] They've done some other deals in the epilepsy space that have
[22:01] given them a whole pipeline of epilepsy type drugs.
[22:05] And I think a lot of companies are are now focused on rare
[22:08] epilepsies and obviously you can get better pricing, but it's
[22:13] it's a deal that again, as Chris, you mentioned, like you
[22:17] like seeing the big deals, you know, that garner the five plus
[22:21] billion kind of price tag, but you also like to see these
[22:23] smaller deals. Not that a billion 3 is
[22:26] necessarily a small deal, but that being said about a very
[22:29] interesting deal for Jazz it it really helps them with these
[22:34] rare epilepsy efforts. Paula, do you want to add some
[22:37] more comments? Yeah, sure.
[22:41] I'll try man. I, I mean, I thought this was a
[22:42] super interesting deal. I had met with Actio a number of
[22:45] times. I thought what they were doing
[22:46] was, was super cool. They also have a, a Charcot Mary
[22:50] tooth program as well. I, I wanted to add maybe like a
[22:53] positive side and sort of AI guess maybe a question just on
[22:57] this space and that's really on the on the positive side, right?
[22:59] It's, it's really cool to see that we're getting this rise and
[23:02] targeted therapies in epilepsy. I mean, I think the concept of
[23:06] targeted genetically oriented therapy and neuroscience is like
[23:09] of theoretically very high interest.
[23:11] But then when you think about like neuro, psych or
[23:13] Alzheimer's, I mean, talk about something that's super, super
[23:17] hard to execute in practice, you know, with epilepsy, right?
[23:20] Because the biology can potentially be a little bit
[23:23] simpler, right? Some of these targeted therapies
[23:25] like, you know, Stoke or things like that, like we're seeing
[23:28] like pretty, pretty great data. There's another private company
[23:31] too that I'm, I'm drawing a blank on that has one that works
[23:33] for the NMDA pathway. I do think though, the
[23:36] challenging thing as an analyst with some of these companies has
[23:39] really to kind of been able to garner conviction on how
[23:42] prevalent some of these indications are, right?
[23:44] Like I remember with GW, there were a lot of questions on how
[23:49] prevalent drive a actually is. And from covering GW, my
[23:53] perception is that Epidiolexes probably less than $100 million
[23:58] product than Dravet, right? It's really everywhere else that
[24:01] that drug is used. A lot of it's kind of used just
[24:03] maybe more broadly in refractory epilepsy.
[24:05] And so for Actio with the KCNT one program, I mean, it's a
[24:09] super interesting, super interesting concept like, but
[24:13] you know, that is the question, right?
[24:14] There's just usually not a lot of good literature in these.
[24:16] And so I love like the therapeutic thesis, but the
[24:19] market thesis is often a little bit trickier to diligence.
[24:23] Yeah. And I'll just highlight it
[24:24] wasn't on our our list, but because Ovid had to announce
[24:28] there was a newly formed company backed by perceptive advisors to
[24:35] developed a statistical stat the overdrug.
[24:40] So they gave new life to that. But the other thing we see a
[24:44] common trend is raising financings off of positive data.
[24:48] Greg, there were a couple this week where large financings on
[24:51] the backs of a phase two data. Yeah, I think there were a
[24:55] number of financings. I'm just going to talk about two
[24:57] smaller ones. I think they're in spaces that
[24:59] probably don't get as much as attention as they probably
[25:05] should. The first I want to talk about
[25:07] is a Canadian company, but it is listed on the NASDAQ, it's
[25:10] called Absalera. They're much to my surprise a
[25:15] three and a 3 1/2 billion dollar market cap company.
[25:18] But they announced positive phase two data for menopausal
[25:22] symptoms or what's known as vasovasomator motor symptoms and
[25:28] they were able to, you know, raise announce $175,000,000,
[25:34] follow an offering which ultimately was upsized to 200
[25:38] million. Again, you don't see a lot of
[25:39] biotechs playing in kind of the Women's Health space.
[25:45] I think the last drug that was approved is called Vioza, but we
[25:51] hadn't really seen innovation on that side.
[25:54] And I've looked at this space in, in the 2000s when I was
[25:57] covering large cap pharma, there's a lot of antidepressants
[25:59] being used. And so it's, it's great to see
[26:03] that a biotech is, is leading the way with hopefully getting
[26:06] another therapy on the market. And then the other deal that I
[26:09] wanted to quickly mention was a company called Silence
[26:12] Therapeutics, which is focused on siRNA and they announced a
[26:17] positive phase two data in a condition called polycythemia
[26:20] Vera or PV. There aren't too many drugs
[26:24] approved here. I think I don't know this space
[26:26] particularly well, but I think there's only two approved drugs.
[26:29] There is a drug that's being developed by Takeda and a
[26:32] collaboration with Protagonist that I think has a third quarter
[26:35] pedufa. I think Takeda is a bit, I don't
[26:38] want to say cagey about exact pedufas, but I think the
[26:41] guidance is a third quarter pedufa that should be hopefully
[26:45] a a newer entrant for this polycythemia Vera market.
[26:50] But that being said, the data from Silence seemed at least on
[26:54] the face of it pretty competitive at least with the
[26:58] Takeda data. And again, being able to
[27:00] announce it off of that, that they wanted to raise 150
[27:03] million, then we're able to upsize that to 175,000,000, I
[27:07] thought was just another a good positive sign for the health and
[27:11] in the biotech sector. Absolutely.
[27:14] Test Revolution Medicine has been one of the big stories of
[27:16] the year. They announced the deal.
[27:18] Do you want to talk about that? Yes.
[27:19] So we have, you know, talked a lot about drugs from China
[27:22] coming into the US And, you know, this was, you know, a
[27:27] great example of, you know, a drug developed by an American
[27:31] company, Revolution Medicines, you know, establishing A
[27:34] collaboration with B1 to, you know, bring their medicines to,
[27:39] you know, China market. So, you know, that was, I think,
[27:44] I think pretty interesting. And what was, you know, it was
[27:46] quite interesting is that it wasn't just a deal about B1
[27:50] being able to commercialize and develop.
[27:55] You know it was a mix of Derek's on RASIB, you know there was a
[28:02] sold in RASIB, you know Eleron, Eleron RASIB as well as like the
[28:09] G12V that they're developing. So pretty broad portfolio and
[28:13] what's interesting is that in addition to the, you know,
[28:18] commercialization and development in Asia, they also
[28:23] established A clinical collaboration.
[28:25] And the clinical collaboration includes, you know, B ones, PRMT
[28:29] 5, you know, also an EGFR met met Tri specific that B1 has.
[28:36] So you know, that's, that's pretty interesting.
[28:38] I think we're just seeing with, you know, revolution medicines a
[28:42] really smart strategy of like, you know, just trying to create
[28:44] like a, you know, a lot of really differentiated
[28:47] combinations with their portfolio.
[28:50] And this is another great example of that combined with,
[28:54] you know, market access to market access to to certain
[28:57] markets in Asia. I didn't see any economics
[29:01] announced with this. So that kind of made me wonder
[29:05] like, did you know, presumably like be one had to pay something
[29:08] for, you know, getting access to these medicines.
[29:11] But you know, I maybe maybe that's kind of offset through
[29:14] the, you know, through the clinical development
[29:17] collaboration. So maybe we'll, we'll find out
[29:19] some more, you know, find out some more details later.
[29:23] Yeah, Tess, I'm just curious when you have the value that Rev
[29:26] Med has created in the stock currency and the cash, you know,
[29:31] often times you expect companies just to be able to go alone and
[29:34] build out those operations. Do you think this was just a
[29:36] choice of, you know, it's going to be easier to partner on this
[29:41] than trying to, you know, build out that territory ourselves?
[29:45] Yeah. I think like China
[29:46] commercialization is like really hard, right, Really hard.
[29:50] You need a lot of people. And so doing something that is,
[29:54] you know, focused there just to get that regional access
[29:56] definitely makes sense. It's like a very different
[29:59] market dynamic, both in terms of like reimbursement and like how
[30:01] do you get the right price on, you know, NRDL in order to get
[30:05] the right access? You know, you need, you know,
[30:08] you do typically need like a pretty large, you know, field
[30:11] force as well. So I think it's an area where
[30:15] like, yeah, could Rev Med like raise money to do that?
[30:18] Like no problem. Absolutely.
[30:20] Right, Because they hire like really smart people to do it
[30:22] also no problem, right. But do they necessarily want to
[30:25] be focusing management time and attention there like, you know,
[30:28] maybe not. Yeah.
[30:29] So that's that's obviously me, you know, hypothesizing and I
[30:32] haven't talked with them about that.
[30:34] But I do think it's a, you know, I do think it's a rational
[30:37] approach. And then it also makes me wonder
[30:39] like, oh, OK, maybe like B ones like PRMT 5 is like awesome,
[30:45] right? They just like really want to
[30:47] work with that. And they're like, oh, like, OK,
[30:50] cool. So like maybe you can have these
[30:52] China, right? So we can, you know, access your
[30:54] drugs. So again, like these are, you
[30:57] know, me speculating. Good, good trade off.
[30:59] So yeah. Yeah, it's actually.
[31:01] It's actually. But it's really interesting
[31:02] that, like those two things, you know, those two things kind of
[31:06] came together and it wasn't like, you know, there was
[31:08] separate economics announced for each of them or something, you
[31:10] know, something like that. So it does make me wonder, like,
[31:13] which one of those came first, the PRMG 5 or like the China
[31:15] commercialization? Yeah, yeah.
[31:18] Yeah. Well, we talked about some
[31:20] negative data readouts, but we've got some positive data
[31:22] readouts that we had this week. Paul, you want to start with
[31:26] Definium first. Yeah, sure.
[31:30] Thanks. So Definium is a psychedelics
[31:32] company developing their their proprietary formulation of LSD
[31:36] for depression and anxiety. They announced positive phase
[31:41] three data from their GAD study, generalized anxiety disorder
[31:44] study that succeeded with a very large effect size.
[31:49] And I think there's just a couple quick things that were
[31:51] were notable about this study versus all the, you know,
[31:54] supportive data we've seen for this company and also just
[31:57] across the psychedelic space, you know, the first one that
[31:59] continues to just amaze me. And, you know, and I wish we
[32:03] even had a better idea beyond the high level explanation of
[32:06] what's happening biologically here.
[32:07] I mean, Definium ran a study for 12 weeks that was
[32:10] placebo-controlled and at 12 weeks, right, dosing is day one.
[32:14] The effect size is still really big, right?
[32:18] Which is just kind of unbelievable.
[32:19] You know, I mean, I think we've talked about how psychedelics
[32:22] seem to have this plastic, you know, at least temporary, but
[32:27] temporary for quite a bit of time rewiring effect in the
[32:29] brain. And just to see that in a
[32:32] placebo-controlled study, 12 weeks, right where I think the
[32:35] definium data is supporting once quarterly, if not less frequent
[32:39] dosing. I think that's I think that's
[32:42] really compelling. And you know, even as people are
[32:43] kind of jaded to it, like still sort of blows me away.
[32:46] I thought the other interesting thing about this trial and it's
[32:49] sort of a gets at maybe a nuance that FDA I think is going to be
[32:53] thinking about in this space as the indications brought in, is
[32:57] that definium went a long way in this study to try to find
[33:01] patients with significant anxiety symptoms, but without
[33:05] comorbid depression. And that's not easy, right?
[33:08] Because you know, when you think about a psychedelics trial, you,
[33:11] you generally think about patients with any sort of
[33:14] psychiatric element that are at the more severe end of the
[33:16] spectrum, right? To come into a study for
[33:18] something like this. And if you have severe anxiety,
[33:21] significant depression is very common.
[33:24] And so to find patients with a much lower baseline depressive
[33:27] burden and still show such a big effect size, right, you've kind
[33:32] of D enriched your population a little bit to have a significant
[33:34] response. I just thought that was really
[33:37] cool, you know, and I, and I think what's interesting here is
[33:39] it, it, it feels to me like reading the subtext, like the
[33:42] FDA now has seen enough data across psychedelics trials that,
[33:46] you know, they, they sort of know many of these drugs work
[33:48] for depression. But if you're going to be
[33:50] pursuing indications beyond depression, they want to get at
[33:54] the question of pseudospecificity, essentially
[33:56] making sure you're not, you know, by improving depressive
[33:59] symptoms, improving all of these other things as an indirect
[34:02] byproduct. The psych division has been
[34:03] pretty sensitive to, to things like that and schizophrenia.
[34:06] They, you know, you can't get a benefit on your label for
[34:09] cognition if it's an acutely psychotic population.
[34:12] You have to study it independently.
[34:14] And so, you know, I mean, definium now has, they have 3
[34:18] positive large placebo-controlled studies, 2IN
[34:21] anxiety, one in depression. They've kind of proved an
[34:24] independent benefit on both and now they've got one more anxiety
[34:28] phase three, but they could be looking at an approved product
[34:30] in the not so distant future. Well, the psychedelic space come
[34:32] a long way. I mean, you know, Greg mentioned
[34:36] jazz acquiring GW back then. And then when you look at, you
[34:39] know, the recent acquisition of Ty Beckley, this data set, I, I
[34:42] think, you know, across different analogs in the
[34:45] psychedelic space really, really shows that this, this
[34:47] marketplace is here to here to stay.
[34:50] Well, Greg, you know Greg, were you Greg?
[34:52] Were you an AbbVie when GW was acquired or were you back on the
[34:55] street? I was back on the street.
[34:59] I was back on the street when that transaction happened
[35:01] because. I was just curious, like you
[35:03] probably have a perspective from like your industry role, but you
[35:07] know, you referenced the GW drug as controversial.
[35:10] And I do think like on the M&A side, right, there had been
[35:14] reporting around that company that there wasn't like a very
[35:18] long list of buyers, right? And I feel like a part of the
[35:21] reason was just the perception around CBDI.
[35:24] Mean you think about that now with Lily, right, the big dog
[35:27] buying 5 Mio DMT. It's kind of hilarious to think
[35:31] about reticence around CBD. Five years ago, I think I
[35:33] remember the time I think I was covering Jazz when that deal
[35:37] happened. And I think the feedback from
[35:41] investors was, yeah, the the data were very good for
[35:47] Epidiolex. And I think they thought that it
[35:49] could be a very successful drug, but there weren't many acquirers
[35:54] where this would fit in the mold.
[35:55] And it it would it could only be someone like Jazz who for those
[36:00] who who know Jazz is, is has been a very successful company,
[36:05] you know, took the orphan drug pricing model, I think to new
[36:10] heights with others. Xyrem drug for narcolepsy.
[36:15] But with that said, you know, Jazz was looking for, you know,
[36:18] they've been looking for CNS assets for a while, and this was
[36:22] the largest deal that they did. But it was going to require a
[36:25] combination of, I guess 2, two players that were a bit off the
[36:30] beaten path and Fast forward to where we are today.
[36:35] Now we're talking about psychedelics.
[36:36] But again, with Gilgamesh getting acquired by AB V or now
[36:41] you're seeing Lily buying a Ty Beckley.
[36:43] It does take some time, but I I I think the time is now.
[36:47] Yeah, we've come a long way in a few short years.
[36:50] I just wanted to highlight real quick we had a a another phase
[36:54] three lung cancer readout. Lung cancer has been pretty
[36:56] elusive and a lot of failures in the space.
[36:59] But Cullinan, which has a collaboration with Ty Ho, which
[37:02] I think was an original Ty Ho drug that they required code
[37:06] development and commercialization rights for,
[37:09] they already had a second line readout that they have filed.
[37:12] The pedufa date is in February. They've now got a first line
[37:16] study that just read out this week that was also positive.
[37:21] And this is in a very interesting space.
[37:22] J&J has their combo products in this space that are on track to
[37:26] do over a billion this year. The Arizona has some competitive
[37:31] a product coming. So just something to watch as we
[37:35] expected a PDUFA date approval potentially in February of next
[37:39] year and test Moon Lake had phase three data read out as
[37:43] well. You want to talk about that?
[37:44] Moonlight announced data for their, they announced data for
[37:49] their Illinois 17 that I think was, you know, an important part
[37:55] of the, the thesis, you know, for, for Moon, for the company,
[37:59] for many people. And they were studying this in,
[38:04] you know, psoriatic arthritis and the bar there, you know, it
[38:08] was really bimicizumab, right? And I think what everyone was
[38:11] looking for was, you know, hey, is this going to be potentially
[38:14] better than bimicizumab? And, you know, I think the, you
[38:19] know, the PR, you know, highlighted, you know,
[38:23] highlighted positive results in, you know, the bio naive, you
[38:27] know, bio naive patients. And you know, the, the ACR 50,
[38:32] you know, 20 Puzzi 90, that all looked good.
[38:36] And I think the stock reaction, you know, the stock was, you
[38:39] know, the stock was, was down, you know, after, after those
[38:43] data was, you know, pretty indicative of hey, interesting.
[38:48] But like, how is this going to compete, right?
[38:51] How is this going to compete against Ben McKenzie Mab And you
[38:55] know, I think it I think it's you know, it kind of shows just
[38:58] some of the challenges commercially where, you know,
[39:03] that differentiation for any, you know, follow on drug really
[39:06] becomes, you know, is really, you know, a critical a critical
[39:11] emphasis. And, you know, sometimes we've
[39:13] seen things where, you know, there's disappointment kind of
[39:16] after clinical data because, you know, everyone was excited about
[39:20] the potential for differentiation.
[39:22] And then the company gets closer to launch and everyone's like,
[39:24] Oh yeah. But like, they can still sell,
[39:26] right? Like they'll, they'll still be a
[39:27] market for this. And, you know, then, you know,
[39:30] people can often kind of identify some, you know, patient
[39:33] population or, you know, commercial strategy that can be
[39:37] a fit and, you know, can can make, you know, can still make
[39:41] the the commercialization, you know, really worthwhile.
[39:45] So it'll be interesting to see, you know, how how Moon Lake ends
[39:50] up driving the the positioning, the positioning for this asset.
[39:55] Great. We also had another kind of
[39:59] readout, safety readout on from Neurocrin, which was the product
[40:04] that they acquired with the Celino acquisition where 7
[40:09] deaths were reported. In this indication, Prater Willy
[40:13] Syndrome, Greg and Paul, I think both have followed this.
[40:17] Greg, you want to start on the Neurocrine, What was the data
[40:20] released there? Yeah.
[40:22] So I'll just introduce the news and since I don't cover
[40:25] neurocrine, but Paul does, he'll he'll go into much bigger
[40:28] detail. But this was an interesting
[40:30] development because the the news of of new safety issues or or
[40:34] safety issues for Vicat XR, which is a proof of Prater Willy
[40:38] syndrome actually did not come directly 1st.
[40:42] I believe it didn't come from neurocrine actually came from
[40:44] the foundation for Prater Trader Willy research.
[40:48] So, you know, it was basically a communication by, you know,
[40:53] let's call it, you know, a patient based organization or a
[40:57] research based organization where they just put out
[41:01] basically what they call clinical clinician
[41:04] recommendations for real world use and monitoring of side
[41:09] effects. And I think importantly, the
[41:11] message is really about, you know, this is an important new
[41:14] medication to treat the specific symptom of hyperphagia, which
[41:20] for those who don't know what Prater Willy is, it's just a
[41:23] genetic mutation which unfortunately creates this
[41:27] insatiable hunger and it impacts particularly children.
[41:33] And with that said, this was really kind of a clinical
[41:37] perspective around kind of how to use this drug and it but it
[41:41] did identify 7 deaths, which I don't think previously had been
[41:46] appreciated. And of course, this is
[41:47] heartbreaking for for patients who, you know, hope for a new
[41:51] treatment that can hopefully, you know, change their lives.
[41:55] But I don't think you know what the foundation for Peter Willey
[41:59] research was trying to do necessarily was to say, you
[42:02] know, this is a problem. We need to get this drug pulled
[42:06] off the market. I think it was really just more
[42:08] of a, let's call it APSA, But obviously it it raised alarms
[42:13] both, you know, in the community, but also, I guess
[42:15] amongst investors. With that, I'll I'll turn it all
[42:17] over to to Paul for his further comments.
[42:21] No thanks, Greg. I mean, I think from the
[42:23] neurocrine like stock perspective, right?
[42:25] They bought Soleno can't say it was at a distressed price,
[42:30] right, but not close to Soleno's all time stock high.
[42:33] And that is, you know, in large part because there had been
[42:37] reports of stuff like this dating back to a year ago.
[42:41] I'll be it. You know, they weren't from as
[42:43] sort of credible as a source as the Prater Willie Foundation.
[42:46] There was also stat news article from, from Adam Forstein about,
[42:49] about all of this. You know, I think just for
[42:52] again, like I'm not trying to be sent to be insensitive to the
[42:56] greater issue here, but from like a neurocrine stock
[42:58] perspective, you know, they bought Seleno for not like a
[43:02] huge value sort of relative to what the drug was already
[43:05] selling, right? I think the deal was a little
[43:06] bit less than 3 billion and Selena was already a profitable
[43:10] company on a standalone basis. So I kind of think like the
[43:14] investor expectations here for this drug aren't, aren't huge,
[43:17] right. I mean, it's, it's going to do
[43:19] close to 400 million this year. You know, I think the hope is
[43:23] like, can it get to 5 to 600? You know, it's, it's profitable
[43:28] for neurocrine still feels like this disease is severe enough
[43:32] that there's going to be demand to try it, even if it sounds
[43:34] like people clearly have to be careful.
[43:36] And look from my perspective, like the clinical data on
[43:39] efficacy for this drug is not overwhelmingly convincing,
[43:42] right? I mean, the most positive study
[43:43] is a randomized withdrawal trial.
[43:45] And those trials tend to, I think be better enriched for for
[43:49] a larger effect size. So I I think for Neurocrine like
[43:52] as a stock and as a company, like certainly if this is just
[43:56] the beginning of a lot more noise around this, you know, it
[43:59] becomes I think a bigger problem.
[44:00] And you know, maybe a greater question around, you know, was
[44:04] this the right deal? But you know, given that we
[44:06] already had some of this noise before, given the unmet need,
[44:10] you know, my perspective, is it still going to be like a viable
[44:13] commercial product that can probably grow at least some from
[44:16] here for them? Yeah, and presumably right that
[44:18] that rumor was out there from the short thesis and that that
[44:21] would have been part of their, you know, due diligence.
[44:24] Right, exactly. Deeper on on that and it's
[44:27] always interesting to watch I mean, just even thinking about
[44:29] like Sereptus gene therapy when the deaths came out that there's
[44:32] always a trade off or risk benefit that every patient
[44:34] family has to go through. It's always interesting to see,
[44:37] you know, there may be those who are aware of these risks and
[44:41] still feel the risk benefit is justified.
[44:43] So it's just interesting to see how these death reports
[44:47] ultimately play out in the in the market.
[44:50] Similarly. So Amgen had drug Tavnios where
[44:55] it was pulled by the EMA this past week.
[44:59] And this followed the FDA really urging Amgen to pull the drug
[45:07] from the US market of which Amgen initially, you know,
[45:09] really, you know, pushed against and said, you know, and defended
[45:13] the drugs, you know, risk benefit profile.
[45:16] But now with EMA pulling that same drug, it does call into
[45:20] question if that puts pressure on Amgen to pull the drug from
[45:24] the US. Now, this was interesting.
[45:26] I didn't really follow this story too closely, but
[45:28] apparently Chemo Centrix, which Amgen acquired in 2022, it was
[45:34] discovered that they really provided, you know, quote,
[45:38] incorrect and misleading pivotal data in their filing, right?
[45:42] And So what this was uncovered to be was there was a unblinding
[45:48] by selected members of the Chemo Centrix team and they realized
[45:53] that it was going to miss the P value.
[45:55] And then they re adjudicated, I think it was 9 patients.
[45:59] And by classifying them essentially as you know,
[46:02] responders, the P value was positive and that enabled the
[46:06] drug to be filed and approved under that statistical
[46:10] significance. So I, I don't know all the
[46:12] details on that, but I'm just reading what has been reported
[46:15] in the press. So it just doesn't look good for
[46:18] our industry in general that this kind of stuff goes on.
[46:21] And you know, Amgen pushing back against the FDA initially and
[46:25] now Emma acts so, but it really it's kind of raising the the
[46:29] bigger question. You know, this happens a lot.
[46:31] I just think of like when GBT sold the Pfizer and Pfizer ends
[46:34] up pulling the market, You know, whether that was right by Pfizer
[46:38] or not, that's that was their decision to voluntarily pull the
[46:41] GPT drug. But you know, we see this a lot
[46:43] where there's shoes that drop post acquisition often with
[46:48] multi billion dollar right acquisition prices.
[46:51] And just curious if anybody has any comments on that.
[46:53] And like, you know, I tell you, the biotech that's selling looks
[46:57] brilliant and this is putting aside the fraud or, you know,
[47:00] misleading, you know, unethical behavior.
[47:04] But you know, just, I don't know, how do you guys look at
[47:07] that when you see these billion dollar, you know, we celebrate
[47:10] them when they're announced and then you see some huge drops
[47:13] after the fact. And, and, and maybe Paul, you,
[47:16] you mentioned that, you know, Neurocon should have been aware
[47:18] of this and it probably was part of their due diligence, but any
[47:22] comments about this because it, it's, it's not an infrequent
[47:24] occurrence anyone. I think I'll just say it's some,
[47:38] I mean, it's, it's a, it's a, it's a capitalist system that we
[47:41] work in. You obviously want to have
[47:45] honesty and transparency in transactions, whether it's in
[47:49] biotech or or not within biotech.
[47:51] It could be real estate, but you know, I have not been following
[47:55] this story closely. I, I don't have kind of skin in
[47:59] the game as to who did what. And with that said, I mean it's,
[48:04] it, it does I think create a little bit of a black eye for
[48:08] the industry when these things happen.
[48:10] But I think by and large, these tend to happen in, in a small
[48:14] minority of cases versus more generally speaking.
[48:17] I I guess that's all I'll add on this.
[48:22] Yeah, I, I think that's fair. I look, I think it for any
[48:25] company we meet with companies raising, you know, trying to
[48:27] raise money all the time. And you know, you have to dig
[48:30] deep in due diligence to make sure that, you know, there we
[48:33] know that they always put their best foot forward, but we do
[48:36] expect them not to hide or you know, have any kind of, you
[48:39] know, misleading data in data rooms, et cetera.
[48:43] But you know, I think it does fall, it is incumbent on the
[48:47] acquirer to make sure that they're digging into the due
[48:49] diligence. And most pharma companies, large
[48:51] biotech, you know, it can be a laborious task to get through
[48:56] there, you know, 50 people who might be involved in a multi
[48:59] billion dollar acquisition. So at the end of the day, right,
[49:02] you have to put the onus on them to make sure that they've
[49:06] checked every box and really assess the risks short of, you
[49:10] know, anybody being fraudulent, you know, in what they present.
[49:15] Were you going to? Mention anything?
[49:16] No, sorry. I think you guys, I think you
[49:18] guys got it makes sense. There were two company news
[49:21] events that you followed this week, Believe Bio and Axel.
[49:25] Yeah. Thanks so much.
[49:27] Yeah, I just wanted to mention news flow out of two companies
[49:30] that I follow closely. One came from Beelight Bio
[49:35] publicly traded company, remarkably has been a darling of
[49:42] a success story over the past several years.
[49:45] For those who don't, for those of you who don't know be like
[49:48] Bio, they are a company that is based on the West Coast.
[49:53] They have been working on developing therapies for
[49:57] ophthalmology diseases. And with that in mind, they have
[50:01] a drug that in news that happened this week.
[50:04] The drug is called Tenlarabant. It is being developed for a rare
[50:08] orphan retinal disease called Stargardt diseases.
[50:12] And with that said, there's about 40,000 patients in the US,
[50:15] So the EPI data would suggest, but they got their new drug
[50:18] application accepted by the FDA. This is following very positive
[50:23] phase three data. I'll remind everyone, there are
[50:26] no currently approved drugs for Stargardt disease, which is
[50:31] ultimately blinding and does affect patients as young as, you
[50:37] know, when they're in their teenage years or even younger.
[50:42] But with that said, they did get a priority review.
[50:45] They had breakthrough designation, orphan drug
[50:47] designation. They had all of the bells and
[50:49] whistles that you would get from hopefully get from the FDA.
[50:52] So we now have a, an official Padufa assigned.
[50:57] That date is February 12th of 2027.
[51:01] So hopefully we could have a new drug approved for the very first
[51:05] time for patients with Stargard disease.
[51:08] It is caused by a genetic mutation.
[51:10] Interestingly, this drug does not work on correcting that
[51:14] mutation, but there are companies working from a gene
[51:18] therapy perspective. But with that said, it's almost
[51:21] like ADMD story as I understand it.
[51:23] We're like the gene that you would want to kind of fix and
[51:26] put into a gene therapy is a bit too large.
[51:29] So you're going to need some kind of second or third
[51:34] generation attempts at gene therapy to be able to try to
[51:38] tackle it from a genetic perspective.
[51:42] But with that said, there's nothing else out there.
[51:44] This is an oral drug and we do look forward to hopefully
[51:48] getting that approval. But I do think the timing is
[51:51] interesting because last week another company that I follow
[51:54] called Tarsus Pharmaceuticals, this is a company that
[51:57] historically has been just focused on what we call front of
[52:00] the eye diseases. They have a drug on the market
[52:03] called Xtemvi. It is one that is an eye drop
[52:08] that treats a condition called Demodex blepharitis, which is
[52:14] caused by Imites, microscopic Imites that I guess live on your
[52:19] eyelashes, but you may have seen commercials on TV or on social
[52:24] media. The DTC campaign is quite robust
[52:28] and they've been able to grow this is Tarsus.
[52:31] They've been able to grow Xtemvi to be the guidance was raised on
[52:35] their earnings recall last week from 670 million to 700 million
[52:42] this year to now 685,000,000 to 705,000,000.
[52:46] So the drug is doing well, but they did announce an acquisition
[52:50] of B Lite Bio's biggest rival, which is a company called Alkias
[52:54] Pharmaceuticals. It's a private company.
[52:58] And with that said, they are working on a very similar drug
[53:01] called Gilda Retinol, which works in a somewhat similar
[53:05] mechanism of action. And they are in phase three,
[53:08] they're ongoing phase three study, I think it's called North
[53:12] Star. They won't get data until the
[53:16] second-half of 2029. So it does create, I think some
[53:19] interesting questions for Tarsus as I think about enrolling this
[53:23] phase three North Star study, because I think the study is
[53:27] going to be somewhat in the neighborhood, about 250
[53:29] patients. It did recently open up for
[53:32] enrollment, but you could have a situation where patients with
[53:36] Stargart disease are faced with the option of either going into
[53:40] a trial or just getting the drug which is already approved.
[53:44] So I think it'll be an interesting dynamic going
[53:47] forward between B Lite Bio and now Tarsus Pharmaceuticals,
[53:51] which just bought Alcheus. That was about $800 million for
[53:54] that acquisition. And then just very quickly on
[53:56] Axon Therapeutics, which I cover, they reported results,
[54:00] second quarter results Monday. The investor focus is on an
[54:03] antidepressant, which is done very well.
[54:05] That drug is called Aveldi. That drug was approved several
[54:11] years ago for depression, but it just got approved more recently
[54:15] to treat Alzheimer's disease related agitation.
[54:18] And all eyes are focused on the launch and how that drug is
[54:22] going. There's a lot of sampling.
[54:24] A lot of these patients are Medicare patients because
[54:27] they're older patients, you know, they're being treated in,
[54:30] you know, Alzheimer's care facilities or, you know, long
[54:33] term care facilities. So people are trying to look at
[54:36] the prescription data. It's very hard to to basically,
[54:40] in my opinion, try to, you know, directly get a read from what's
[54:45] available through IQV or Bloomberg.
[54:47] But that being said, management comments were very positive.
[54:50] They did say like you know new to brand prescriptions, which
[54:54] is, you know a patient getting a drug for the first time in the
[54:58] 65 year old and older segment are up 126%.
[55:05] So we'll continue to monitor this launch very closely.
[55:08] But so far so good for Axon with its launch for ability and
[55:12] Alzheimer's agitation. I'll turn it back to.
[55:14] You yeah, good stuff, yeah. And also it's nice to see again
[55:17] a diversity and an expansion of the potential acquirer.
[55:20] So even though you know, Tarsus, I think is a few billion dollars
[55:23] acquiring a you know, Alkis again, it's nice to see these
[55:28] acquisitions happening with not just the large pharma companies
[55:31] out there for, you know, all the private and public companies out
[55:35] there. I wanted to end with Tess, your
[55:38] your boss, Peter Kulchinsky, who's been very vocal about the
[55:43] non protectionist stance and you and he has written white papers
[55:46] about this. There was an interesting debate
[55:49] that endpoints hosted that I would encourage everybody.
[55:52] It's free online to watch, but it was Peter Kulchinsky, head of
[55:56] RA. They had a Fiona Marshall from
[56:00] a, you know, multinational Europe based pharma company and
[56:03] then Jason Kelly, who's the CEO of Ginkgo Byerworks.
[56:07] So we had kind of a diverse panel had very different
[56:10] opinions about this, but Bensa was re established.
[56:13] So, you know, adding biotech to bio security from the coins Act
[56:17] is reigniting this China debate and what level of you know,
[56:21] quote protectionism that we're seeing out there, but would love
[56:24] to hear your thoughts test. We know, you know, Ari's
[56:27] position on this. I believe that is the right kind
[56:30] of stance, but you want to share what's transpired in that in
[56:33] that debate? Absolutely.
[56:35] I mean, I thought it was wonderful that Endpoints did
[56:37] this. So this was Drew, who's, you
[56:40] know, one of their editors. And, you know, I love that.
[56:44] He just was like, can we just talk about this?
[56:47] And like, let's actually have a real conversation that's like,
[56:50] you know, multiple sides of the debate.
[56:52] So we set it up really nicely. You know, Fiona Marshall was
[56:55] kind of there. She's like a professor at MIT
[56:57] that does a lot of national security, you know, national
[56:59] security work like really across sectors.
[57:02] Like, you know, biotech is actually more new for her.
[57:04] She said she's like more focused on drones and like other
[57:07] technologies these days. But you know, she was there to
[57:11] really, you know, Fact Check and kind of provide like the, you
[57:15] know, the the national security kind of like cross sector lens.
[57:19] You know, obviously, you know, Peter and Jason being people who
[57:23] are within biotech have very different perspectives on how
[57:28] to, you know, how to compete with China and how much that,
[57:33] you know, competition should include various protectionist
[57:36] measures. And you know, it was a good, it
[57:39] was a good, like good discussion, right?
[57:42] Like lots of just disagreements and like, you know, important
[57:46] disagreements to kind of get out.
[57:48] And I thought Fiona did like a really nice job, you know, kind
[57:52] of highlighting how, you know, the current legislation that's
[57:55] being proposed. You know, I think, I think the,
[57:57] the bottom line, that kind of the debate, the debate came to
[58:01] that. I think both Fiona and Drew did
[58:02] a really nice job summarizing was like, look like, you know,
[58:06] there's, there's, there's, there's reasonable and valid,
[58:09] like concerns around US competitiveness in biotech,
[58:12] right? So like, let's like take that
[58:15] really seriously, but you know, is, you know, this a broad based
[58:20] ban, like that's probably not the right legislative approach,
[58:23] right? And like that would also take a
[58:25] lot of coordination, you know, for that ban to kind of like be
[58:27] effective. Like one of the big points of
[58:29] debate, you know, where where Peter was was really pushing
[58:32] Jason was on this, like, how do you enact this ban?
[58:34] Because you know, Jason was saying, like, look, well, the
[58:36] FDA can ban like, you know, this, we've talked about this
[58:39] euro washing saying we're like, well, can Europeans just like
[58:43] get China drugs and like bring them over to the US?
[58:45] And you know, Jason was like, wow, like the FDA could ban like
[58:47] even that, right? It could just be like something
[58:50] that like you discover in China, but like.
[58:52] Basically, Peter tells him on the details, right?
[58:54] Yeah, you're not providing the details.
[58:56] I, I have to applaud Peter. I, I think a lot of people would
[58:59] have expected him. He doesn't suffer fools gladly.
[59:02] So people were expecting him to lose his coal more quickly and
[59:05] he was very restrained in my opinion.
[59:08] And he, you know, and I kind of just like Jason talk and it kind
[59:11] of helped the the argument that he was trying to.
[59:14] Make Yeah, totally did. Yeah, yeah, yeah.
[59:16] So anyway, well, we're at the top of the hour.
[59:18] Hey, thanks for all of our Co hosts.
[59:20] We appreciate hope you enjoyed this session of biotech.

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