Ep. 347 - Speeding China's Innovation. Plus: Neuro Catalysts and Newcos

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<v Jeff Cranmer>New rules in China are adding rigor while fueling the country's gene and cell therapy engine.

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<v Jeff Cranmer>We'll have a look at the new rules which take effect in May.

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<v Jeff Cranmer>On this week's BioCentury this Week podcast plus 2026 catalysts for the biotech industry.

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<v Jeff Cranmer>This week, BioCentury kicks off its annual look at the milestones that will drive biotech in the year ahead, we'll be starting with neuro.

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<v Jeff Cranmer>Plus a couple of gems from our emerging company profile series.

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<v Jeff Cranmer>We'll take a look at Elkedonia, an ArgoBio founded French biotech, which is investigating a potentially first in class intracellular target to treat neuropsychiatric disorders, starting with treatment resistant depression and Bexorg, a Yale spin out that is developing a platform to test drugs in donated whole human brains.

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<v Jeff Cranmer>That remain molecularly active.

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<v Jeff Cranmer>It's an approach that the company believes could transform CNS drug discovery.

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<v Jeff Cranmer>I'm Jeff Cranmer, host of the BioCentury This Week podcast, and joining me today are my colleagues.

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<v Steve Usdin>Steve Usdin, Washington Editor.

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<v Selina Koch>Selina Koch, Executive Editor.

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<v Danielle Golovin>And Danielle Golovin, Senior Biopharma Analyst.

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<v Jeff Cranmer>Okay, well, we've been telling you recently about our East-West Summit, which will be in March in Seoul, Korea.

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<v Jeff Cranmer>returning for its 26th year in May is Bio€quity Europe.

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<v Jeff Cranmer>This is BioCentury's flagship conference sells out every year, so now is your chance to take advantage of early bird rates, which end on Friday.

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<v Jeff Cranmer>Bio€quity Europe is where CEOs and investors gather to network partner and debate critical issues facing the biotech industry.

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<v Jeff Cranmer>We also have a podcast fun Run, which uh.

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<v Jeff Cranmer>Our colleague Josh Berlin is always plugging.

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<v Jeff Cranmer>I don't know if Josh has ever, done the fun run and nor do I think running is very fun, but nonetheless,

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<v Steve Usdin>Is the podcast interviewing people while they're running?

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<v Jeff Cranmer>you know, we'd have to get probably Stephen Hansen to host that episode because I do not think I can keep up with some of, uh, the folks in the industry who are always out, cycling and, and, running, uh, my, my speed's more uh, elbow on the bar and asking people about the cool things they're doing with their technologies.

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<v Jeff Cranmer>I dunno.

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<v Jeff Cranmer>Steve, would you be up for hosting that one?

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<v Steve Usdin>If I can ride a bike, yeah.

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<v Steve Usdin>I'll ride a bike alongside them jogging and, and running.

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<v Steve Usdin>Yeah.

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<v Jeff Cranmer>All joking aside, take BioCentury with you on the go.

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<v Jeff Cranmer>listen to the podcast while you're out for a run, uh, a ride or, uh, a walk in the hills, which is my preferred, type of exercise.

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<v Jeff Cranmer>And look out for signups for the BioCentury's Fun runs at Bio€quity Europe.

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<v Jeff Cranmer>Alright, Steve, you've been talking to, a few folks about these new rules that China is around gene and cell therapy investigator initiated trials, I believe.

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<v Jeff Cranmer>What did you learn?

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<v Steve Usdin>here's the most important part of it.

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<v Steve Usdin>For the first time, Chinese medical centers are gonna be allowed to charge for investigational therapies, cell and gene therapies, that have been tested in an IIT, an investigator initiated trial.

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<v Steve Usdin>There are limits around it, but in general, I think that this is going to fuel an acceleration in the rate of biomedical innovation, it's going to lead to, the development of more gene and cell therapies.

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<v Steve Usdin>it's going to generate data that proof of concept data, that will be useful for the traditional, approvals in China, and also in de-risking acquisitions of assets from China, around the world.

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<v Jeff Cranmer>what are some of the around these new rules?

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<v Steve Usdin>so, uh, one, they've limited the number of hospitals or the type of hospitals that will be able to do.

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<v Steve Usdin>Investigator initiated trials are gonna be Class A tertiary hospitals.

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<v Steve Usdin>That's a nomenclature in China, that's about 1700 hospitals.

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<v Steve Usdin>They're, uh, mostly large academic medical centers.

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<v Steve Usdin>After an IIT has been completed.

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<v Steve Usdin>The medical center will be able to apply to national authorities for permission to translate that, therapy, into routine use in their medical center and to charge for it.

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<v Steve Usdin>They won't be able to use it, in other medical centers.

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<v Steve Usdin>They'll be limited to that one medical center, but they'll be able to use it in routine care.

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<v Steve Usdin>and as, and I said to be able to, um, charge for it.

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<v Selina Koch>So they apply for that.

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<v Selina Koch>So it's not the same thing as NMPA approval.

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<v Selina Koch>But there is an application process, so how, how are they deeming the ITT positive enough?

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<v Steve Usdin>You know, that is something we don't know the answer to.

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<v Steve Usdin>Okay.

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<v Steve Usdin>I, I spoke with, with a half a dozen.

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<v Steve Usdin>Experts, CROs and companies that are, developing therapies in China and none of them know what the criteria, will be for deeming that a therapy, um, can be transferred from the status of an investigational therapy that can't be charged for because in China, can't charge patients, you can't charge anyone for a therapy that's given in a trial.

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<v Steve Usdin>Nobody knows what the criteria is going to be to go from that to something that's considered, ready for routine use.

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<v Steve Usdin>And they can be charged for the assumption of the people that I spoke with is that, you know, there'll be an expectation that the medical centers will demonstrate, statistically, significant safety and efficacy.

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<v Steve Usdin>But, we really don't know.

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<v Steve Usdin>Obviously the, the, the trials are gonna be, um, smaller in scale than the ones that are traditionally used, for approval by the NMPA.

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<v Steve Usdin>That's the, Chinese version of the FDA.

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<v Selina Koch>I'm also curious about throughput.

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<v Selina Koch>So they've narrowed the number of hospitals that can do these.

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<v Selina Koch>Right.

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<v Selina Koch>So now that's, it's focused on kind of specialized centers, which is good for the robustness, the quality and all of that.

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<v Selina Koch>but is it, are IIT is gonna be less common.

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<v Selina Koch>So like the resources available to do your de-risk if you wanna have your thing de-risked in an IIT, is there gonna be a longer line for that?

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<v Selina Koch>Do you have any sense?

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<v Steve Usdin>I, I don't think so.

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<v Steve Usdin>I think the, the people that I spoke with said that there's gonna be a tremendous amount of enthusiasm among these, medical centers, and it's a lot of 'em still.

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<v Steve Usdin>It's 1700 medical centers.

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<v Steve Usdin>There's gonna be tremendous enthusiasm and incentive for them to get these IIT trials launched and to do them.

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<v Steve Usdin>At the level of quality and rigor that's gonna be required to get approval, for, um, routine therapeutic use.

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<v Steve Usdin>So the, the people I spoke with say that they think that there's gonna be an acceleration both in the speed and in the throughput, as a result of this.

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<v Steve Usdin>And, you know, and this is gonna add speed to a process that's already, you know, faster than what happens in, Any, anywhere else in the, in the world.

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<v Jeff Cranmer>Which is, which is kind of insane.

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<v Jeff Cranmer>that's really what stood out to me when I read your, piece, you know, talking about, a government committee turning around an application in like 20 days is, uh, is pretty fast.

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<v Steve Usdin>Yeah.

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<v Steve Usdin>So, yeah, so that's, I I should have, should have mentioned that.

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<v Steve Usdin>So there's, there's very, strict and very ambitious timelines for the whole process.

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<v Steve Usdin>And at the end of it, after a medical center has completed an IIT, and believes that it's ready to be transitioned to routine therapeutic use they submit it, to a National Authority, which has a total of 20 working days, to provide an answer, a yes or no answer.

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<v Steve Usdin>And then there's gonna be the same kind of, um.

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<v Steve Usdin>Post-market or post-approval follow-up that you would expect for any, gene and cell therapy.

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<v Steve Usdin>and there will be the possibility, that approvals could be, modified or, or withdrawn, based on data that's collected after, after they go into routine use.

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<v Jeff Cranmer>One up shot that you mentioned, is this ability to get therapies into routine clinical use this fast is really gonna reduce the cycle time for iterative improvements in therapies and in the technology for manufacturing.

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<v Steve Usdin>I, I think so, and I think especially if you look at something like CAR T therapies that are already having big impacts around the world.

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<v Steve Usdin>They're not optimized by any stretch of the imagination.

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<v Steve Usdin>And I think that the ability of many, many medical centers in China to launch, CAR T therapies and similar kinds of therapies, to start charging for them to learn from, their experience, to do new trials and to modify the therapies and to modify the way that they're manufactured, quickly.

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<v Steve Usdin>Is, is gonna have a big impact on, moving the, whole field forward.

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<v Jeff Cranmer>What do you think the likely impact on Western, uh, drug developers and investors might be?

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<v Steve Usdin>Well, I, I think you're going to see, Western companies, it, it wouldn't surprise me if you see Western companies looking to partner, with Chinese institutions, Chinese biotech companies, Chinese medical centers, in an effort to get proof of concept data on therapies I think you're gonna start seeing, more western companies acquiring, assets from China, that have been de-risked, at least to some extent, because there's high quality human data that's available that makes them more comfortable, launching, investing in and launching, um, a more traditional clinical development program outside of China.

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<v Jeff Cranmer>All right.

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<v Jeff Cranmer>Well, Steve's story is out now on BioCentury.com.

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<v Jeff Cranmer>It is, uh, going to be very interesting to watch how this unfolds.

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<v Jeff Cranmer>Okay, catalysts first up will be Selina's story.

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<v Jeff Cranmer>the catalyst package.

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<v Jeff Cranmer>We kick off every year with it, with the editorial staff, led by Selina's and Lauren Martz, coming up with the milestones.

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<v Jeff Cranmer>In various indications that they believe will move the needle, for biotech and pharma.

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<v Jeff Cranmer>Selina, you have, uh, you're a, a little bit cross-eyed, I can tell you.

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<v Jeff Cranmer>You've just sent me this massive, uh, deck to edit.

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<v Jeff Cranmer>What stood out to you, uh, among the various, catalysts in neurology?

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<v Jeff Cranmer>What milestones are you looking out for this year?

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<v Selina Koch>Well, the good news, Jeff, there's a lot of them.

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<v Selina Koch>It is a really big slide deck

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<v Jeff Cranmer>that is great for neuro.

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<v Selina Koch>Yeah.

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<v Selina Koch>So people should, if they're interested, go in there, spend some time.

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<v Selina Koch>maybe we'll take it in part, so in psychiatry, where disease biology is still poorly understood, where the animal models are terrible, everybody acknowledge there's still a lot of really interesting things going on.

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<v Selina Koch>Okay, let's start with psychedelics.

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<v Selina Koch>there was obviously the first program from Lykos, formerly MAPS, did not make it.

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<v Selina Koch>Um, but this year we'll have Phase III data coming from three programs and depression and one in, in anxiety.

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<v Selina Koch>And, These programs are producing, been producing really big effect sizes, in their earlier trials.

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<v Selina Koch>And, you know, as I go about and talk to neuroscience companies at JP Morgan and other places, I, I ask about this field and, there's just a lot of people out there who think, yeah, one of these companies is definitely gonna make it like we're gonna have a psychedelic on the market the next few years.

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<v Selina Koch>So this is an area to watch.

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<v Selina Koch>I think big questions have always been, can you really know what the effect size is, given that there's functional and blinding, right?

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<v Selina Koch>People understand which group they're in, for the most part, whether they've gotten placebo or not, because these are powerfully mind altering drugs.

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<v Selina Koch>and the answer there seems to be, well, if the really large effect sizes that we've seen in Phase II carry through into Phase III.

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<v Selina Koch>Then even if some expectation bias is contributing, some, you know, placebo effect is contributing, um, it probably won't explain all of the effect, right?

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<v Selina Koch>So people are hoping for really big effect size is that's gonna be important.

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<v Selina Koch>The other thing that's gonna be important is durability of these, treatment effects.

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<v Steve Usdin>I, I'll just jump in there and say this I think is one area that's gonna benefit from, the new leadership at FDA and the new leadership at HHS.

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<v Steve Usdin>There's a great deal of, enthusiasm for psychedelics and, and I would say even impatience for FDA to approve psychedelics, coming from Secretary Kennedy, coming from Commissioner Makary.

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<v Steve Usdin>So I think that the things that kind of doubts or the things in the past might have been impediments or speed bumps to getting things, approved are likely to be easier to overcome now, than they were in the past.

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<v Selina Koch>That's right.

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<v Selina Koch>And, and one of the debates in this field is whether you need the psychoactive, the trip part of the psychedelics for the efficacy.

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<v Selina Koch>We're not gonna get an answer on that this year.

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<v Selina Koch>All these Phase III companies, are kind of, even if they have synthetic versions, analogs, you know, it's basically psilocybin, LSD, the classic psychedelics, which have the trip.

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<v Selina Koch>So that, that's a future question to be answered.

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<v Selina Koch>Not, not of this year one.

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<v Selina Koch>Um, but if things go well and they make it on the market, I think one of the big questions just for the industry is what is the care pathway gonna look like?

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<v Selina Koch>What are the payment models gonna look like?

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<v Selina Koch>Because these require.

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<v Selina Koch>In clinic administration, it's six plus hours of monitoring afterward.

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<v Selina Koch>It's a full day.

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<v Selina Koch>It uses a lot of hospital resources.

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<v Selina Koch>Um, and so, so these companies, Compass, Cybin, and the likes are, are, working on solutions problems.

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<v Selina Koch>And then behind them and Phase II, there's Abby AbbVie, the Gilgamesh deal, which was kind of a big deal, um, in neuropsych last year.

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<v Selina Koch>they have a synthetic version that's meant to be just a lot more, consistent from patient to patient in terms of how long it lasts, and it's much shorter.

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<v Selina Koch>So they're hoping to make some progress there and then further behind our companies who are trying to like, get rid of the, the trip aspect altogether.

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<v Selina Koch>Um, but that's gonna be a little while.

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<v Jeff Cranmer>how about outside of the psychedelic space?

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<v Selina Koch>So we have, um, so a really interesting area to watch over the long term will be precision psychiatry, precision medicine in the sense of selecting patients.

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<v Selina Koch>this is still very nascent, but, um.

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<v Selina Koch>Some of the leaders there, like Alto Neuroscience, have proof of concept, you know, Phase II and Phase IB readouts coming this year.

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<v Selina Koch>where they're using say EEG or cognitive testing.

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<v Selina Koch>Some objective measurement that makes sense with the mechanism, mechanism of action of the drug that can help find responsive patients because everybody knows, depression, schizophrenia these big psychiatric indications probably are not one indication and there's not gonna be one thing that really a needle for patients.

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<v Selina Koch>So I'm excited to see, um, you know, the progress that gets made there

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<v Steve Usdin>And, and you think there's gonna be catalyst this year we're gonna have

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<v Selina Koch>Yeah, they're not like the, they're not the late stage, like Phase III but there's proof of concept catalyst coming.

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<v Selina Koch>So, three of them are coming from Alto Neuroscience, two in depression, one is schizophrenia.

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<v Selina Koch>Um, but it's not the only company, there is one that's looking at, um, like a thyroid pathway mechanism, and they have a genetic test for that.

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<v Selina Koch>It's this company called HMNC.

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<v Selina Koch>It's partnered with Spruce, so companion diagnostic for that one.

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<v Selina Koch>Also Phase II, first half this year.

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<v Jeff Cranmer>Are there any, uh, particular companies that you're excited about for this year that have a lot of, uh, things going on?

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<v Selina Koch>Yeah.

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<v Selina Koch>I mean, I think one company, with breakout story potential is Praxis Precision Medicines.

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<v Selina Koch>It had promising, data last year, both an essential tremor, which is an area of definitely big unmet need.

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<v Selina Koch>And in a rare developmental epilepsy condition and it will have more data this year in fact if all goes well for that company, for its lead program essential tremor it could submit to FDA and even given approval by year end so that will, that company might be on might be on the cusp of of transitioning to commercial stage and it may even have more than one drug on its hands in the field of neuroscience Um, so, so that's one to watch.

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<v Steve Usdin>You know, I'll, You know, I'll, I'll just throw one other thing in here, which is when we're looking at catalyst and thinking about the year ahead.

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<v Steve Usdin>it's gonna be scrambled up compared to, previous years by the commissioner National Priority Voucher Program, which is likely to cause some things to jump ahead of the queue to jump ahead of where you ordinarily, you know, the timelines that you would ordinarily think about.

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<v Steve Usdin>And I think, you know, like we talked about last week also, that's also probably going to, have a knock on effect, which is that other things are likely to be slowed because, they're limited resources at, at FDA and if they're, prioritizing on one thing, they're likely to, um, deprioritize other things.

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<v Selina Koch>If I could just make, I don't wanna ignore neurodegeneration altogether before we move on.

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<v Selina Koch>Um, I don't wanna suggest that nothing's going on there.

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<v Selina Koch>There's a lot of interesting stuff going on.

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<v Selina Koch>'cause that's an area where human genetics have been very productive at pointing to, to.

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<v Selina Koch>Targets where you can, you know, have some high conviction around them.

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<v Selina Koch>And we're gonna see rare readouts that I think, are gonna be really interesting.

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<v Selina Koch>There's proof of concept coming for a, a group of therapies for a rare form of frontotemporal dementia, which is driven by granulin mutations, across a couple of different modalities.

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<v Selina Koch>There's more Huntington's disease data coming.

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<v Selina Koch>And then, you know, I think in, in Alzheimer's, well, everybody wants to know if these amyloid targeting molecules are gonna be more effective if you give them to people before they have symptoms.

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<v Selina Koch>That's not a 2026 story.

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<v Selina Koch>That's, that's a little further down, down the road.

00:18:59.938 --> 00:19:05.777
<v Selina Koch>but there is one, at least one important readout from like the classic mechanisms, um, tau and amyloid this year.

00:19:05.877 --> 00:19:09.413
<v Selina Koch>I, I wouldn't say it's make or break because these things never break.

00:19:09.748 --> 00:19:22.894
<v Selina Koch>Um, but in the tau field, Biogen has, data coming Phase II data coming from, from BIIB080, which is important because it's an antisense molecule that just knocks down tau at the mRNA level.

00:19:22.894 --> 00:19:24.462
<v Selina Koch>So all forms of tau.

00:19:24.863 --> 00:19:33.271
<v Selina Koch>Tau is a very complicated protein with lots of, um, different splice variants Combinations of phosphorylation and we have whatnot.

00:19:33.271 --> 00:19:34.306
<v Selina Koch>Lots of proteoforms.

00:19:34.506 --> 00:19:40.278
<v Selina Koch>It's also an intracellular protein, but it has some extracellular pathology as well, right?

00:19:40.278 --> 00:19:45.150
<v Selina Koch>And so like exactly how to target it is just not an easy question to answer.

00:19:45.150 --> 00:19:50.721
<v Selina Koch>And so this mechanism just turns off the faucet that spits out new tau.

00:19:51.088 --> 00:19:55.394
<v Selina Koch>And so it can in theory, kind of take down all of these various forms and test.

00:19:55.826 --> 00:20:09.840
<v Selina Koch>The hypothesis very broadly, and so people will be watching for that to see if, if this target or if this protein tau that we know is involved in Alzheimer's can in fact be a target for a drug, right?

00:20:10.142 --> 00:20:13.178
<v Selina Koch>Because that's very much still not not understood.

00:20:14.813 --> 00:20:16.114
<v Jeff Cranmer>Well, there's a lot here.

00:20:16.347 --> 00:20:17.583
<v Jeff Cranmer>Uh, to digest.

00:20:17.583 --> 00:20:25.390
<v Jeff Cranmer>Uh, the slide format I think really, uh, helps some cool pictures, uh, to go along with it to help you understand the mechanisms.

00:20:25.856 --> 00:20:29.661
<v Jeff Cranmer>yeah, Selina didn't leave, much on the table.

00:20:29.661 --> 00:20:33.131
<v Jeff Cranmer>There's, uh, MS readouts, she's tracking.

00:20:33.198 --> 00:20:39.871
<v Jeff Cranmer>Um, we're looking for a third win for Roche's BTK, which could lead to regulatory submissions.

00:20:40.271 --> 00:20:42.007
<v Jeff Cranmer>And just a lot more.

00:20:42.007 --> 00:20:46.278
<v Jeff Cranmer>So, uh, looking forward to our readers being able to get their hands on this.

00:20:46.510 --> 00:20:58.757
<v Jeff Cranmer>it will be out this week along with, stories from Lauren, and of course Stephen Hansen writing about, obesity milestones, tracking the, uh, the new orals.

00:20:58.957 --> 00:21:04.829
<v Jeff Cranmer>And, I believe he says there's, a lot of other data that will keep investors, interested.

00:21:05.096 --> 00:21:13.571
<v Jeff Cranmer>Okay, let's take a quick break and then we'll come back to Danielle who leads our emerging company profile series

00:21:14.071 --> 00:21:18.743
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00:22:02.086 --> 00:22:02.653
<v Jeff Cranmer>Okay.

00:22:02.653 --> 00:22:05.156
<v Jeff Cranmer>Danielle, thanks for joining us again this week.

00:22:05.656 --> 00:22:05.990
<v Danielle Golovin>Happy to be here.

00:22:06.424 --> 00:22:06.825
<v Jeff Cranmer>Awesome.

00:22:07.125 --> 00:22:26.510
<v Jeff Cranmer>Danielle heads up our emerging company profile series, which, has gotten reenergized lately with, uh, the addition of a couple of new staff members joining, Danielle and writing, about hot new companies, uh, Tierney Baum, and Lindsay Martin, have both been, uh, writing up a storm.

00:22:26.711 --> 00:22:28.579
<v Jeff Cranmer>and Danielle, where do you wanna start?

00:22:28.846 --> 00:22:30.082
<v Jeff Cranmer>we've got one.

00:22:30.481 --> 00:22:33.652
<v Jeff Cranmer>That you wanna talk about, that you wrote and one that Tierney wrote?

00:22:34.019 --> 00:22:34.486
<v Danielle Golovin>That's right.

00:22:34.486 --> 00:22:34.685
<v Danielle Golovin>Yeah.

00:22:34.685 --> 00:22:37.689
<v Danielle Golovin>These actually fit in really well with what Selina was just talking about.

00:22:37.689 --> 00:22:46.198
<v Danielle Golovin>So non hallucinogenic neuropsych medicines, and then better models for CNS drug discovery.

00:22:46.263 --> 00:22:47.365
<v Danielle Golovin>So we can start with.

00:22:47.699 --> 00:22:57.976
<v Danielle Golovin>former, so a French startup called Elkedonia, and the name actually gives you a big clue into their target of interest, Elk1 and what they're going after anhedonia.

00:22:58.242 --> 00:23:02.279
<v Danielle Golovin>So that's the inability to feel pleasure in a, a core symptom of depression.

00:23:03.147 --> 00:23:04.415
<v Jeff Cranmer>tell us about Elk1.

00:23:04.915 --> 00:23:05.784
<v Jeff Cranmer>There's a new one on me.

00:23:06.384 --> 00:23:08.586
<v Danielle Golovin>Yeah, it, it hasn't slipped.

00:23:08.854 --> 00:23:09.554
<v Danielle Golovin>You know, your mind.

00:23:09.554 --> 00:23:10.721
<v Danielle Golovin>It's actually a new target.

00:23:10.721 --> 00:23:13.625
<v Danielle Golovin>It's, we don't have any other products for it in BCIQ.

00:23:13.625 --> 00:23:27.071
<v Danielle Golovin>So this would be a, a potential first in class. Elk1 is a transcription factor and it's activated by ERK. Elkedonia compound inhibits this activation by interrupting the, the PPI, the protein protein interaction between those two.

00:23:27.605 --> 00:23:33.278
<v Danielle Golovin>And then that downstream that leads to modulating gene expression involved in neuroplasticity.

00:23:33.644 --> 00:23:43.320
<v Danielle Golovin>And the CEO Delphine Charvin actually said that it turns off genes involved in inflammation, which is sort of a new axis being discovered in as important in depression.

00:23:43.922 --> 00:23:44.088
<v Jeff Cranmer>Yeah.

00:23:44.088 --> 00:23:44.588
<v Jeff Cranmer>Excellent.

00:23:44.588 --> 00:23:48.692
<v Jeff Cranmer>Um, the, the investor, I mean, this is a pretty early stage company.

00:23:48.692 --> 00:23:49.059
<v Jeff Cranmer>They're,

00:23:49.260 --> 00:23:49.560
<v Danielle Golovin>Yeah,

00:23:50.127 --> 00:24:12.049
<v Jeff Cranmer>using a seed round that they raised, uh, last year, I believe, to identify a small molecule drug candidate, Kurma along with WE Life Sciences and the French Tech Seed fund, uh, which is a government fund, part of Bpifrance, so certainly, uh, some savvy investors backing this.

00:24:12.349 --> 00:24:13.951
<v Jeff Cranmer>anything else to say about the tech Danielle?

00:24:14.719 --> 00:24:29.201
<v Danielle Golovin>Yeah, I think this, to highlight some of the unique points is that Elk1 is intracellular, so this is in neurons, highly expressed in the hippocampus and nucleus accumbens, which are important regions for memory, context and reward.

00:24:29.567 --> 00:24:32.103
<v Danielle Golovin>So that's the context for like the depression connection.

00:24:32.703 --> 00:24:46.317
<v Danielle Golovin>But this in contrast to most classical antidepressants, which are acting extracellular on, you know, transporters and monoamine neurotransmitters at the synapse, like just keeping the transmitter, in the synapse for a longer amount of time.

00:24:46.317 --> 00:24:49.653
<v Danielle Golovin>But it's not really modulating gene expression downstream.

00:24:49.688 --> 00:25:02.601
<v Danielle Golovin>So this could be a more durable, potentially treatment for depression and as the CEO Delphine said to us that the ultimate goal for the company is to make depression an acute condition and not a chronic one anymore.

00:25:03.300 --> 00:25:04.869
<v Jeff Cranmer>How does it compare with ketamine?

00:25:05.470 --> 00:25:05.871
<v Danielle Golovin>Right?

00:25:05.871 --> 00:25:10.141
<v Danielle Golovin>So ketamine, they said it's similar as in it's fast acting.

00:25:10.407 --> 00:25:11.809
<v Danielle Golovin>that's like a great part about ketamine.

00:25:11.809 --> 00:25:14.278
<v Danielle Golovin>You go in the clinic, you get it, and you feel better almost instantly.

00:25:14.980 --> 00:25:20.585
<v Danielle Golovin>But it does have some of those, as Selina was mentioning, like mind altering, you know, aspects.

00:25:20.585 --> 00:25:25.089
<v Danielle Golovin>And so you do have to stay in the clinic and be monitored for several, several hours.

00:25:25.523 --> 00:25:28.192
<v Danielle Golovin>And this molecule would not have that aspect.

00:25:28.960 --> 00:25:29.528
<v Jeff Cranmer>Excellent.

00:25:29.528 --> 00:25:35.599
<v Jeff Cranmer>Well, the company plans to raise a series A after it identifies that lead compound.

00:25:35.933 --> 00:25:41.740
<v Jeff Cranmer>and the company is headquartered in Strasbourg, probably saying that wildly wrong.

00:25:42.073 --> 00:25:49.480
<v Jeff Cranmer>We also had Bexorg, this is a Yale spin out, that Tierney wrote about in December.

00:25:49.748 --> 00:25:53.384
<v Jeff Cranmer>it's a company that's rethinking CNS drug discovery.

00:25:53.585 --> 00:25:54.486
<v Jeff Cranmer>how's it doing that, Danielle?

00:25:55.287 --> 00:25:58.722
<v Danielle Golovin>Yeah, they're using whole human brains, which is kind of wild.

00:25:58.856 --> 00:26:05.430
<v Danielle Golovin>they're really trying to solve the problem that CNS therapies fail at far higher rates than drugs for other organ systems.

00:26:05.730 --> 00:26:10.535
<v Danielle Golovin>This comes back to Selina’s point about how we know the CNS are just not great.

00:26:10.669 --> 00:26:16.473
<v Danielle Golovin>they're, the company's thesis is that cultured tissues, you know, lack the complexity of a living human brain.

00:26:16.508 --> 00:26:20.444
<v Danielle Golovin>And then the animal models just don't predict clinical outcomes.

00:26:20.811 --> 00:26:23.213
<v Danielle Golovin>So what better model than just use the human brain itself?

00:26:23.714 --> 00:26:24.449
<v Danielle Golovin>No big deal.

00:26:24.982 --> 00:26:25.250
<v Steve Usdin>Well,

00:26:25.482 --> 00:26:26.284
<v Selina Koch>No big

00:26:26.451 --> 00:26:26.483
<v Steve Usdin>it,

00:26:26.550 --> 00:26:26.785
<v Selina Koch>deal.

00:26:27.285 --> 00:26:33.959
<v Steve Usdin>it does sound like a big, so one thing you said, you said, uh, you know, living brains, but we're not really talking about living brains that are in living people.

00:26:33.959 --> 00:26:34.326
<v Danielle Golovin>Right.

00:26:34.625 --> 00:26:36.193
<v Selina Koch>Might have some issues ethical issues there.

00:26:36.627 --> 00:26:38.262
<v Danielle Golovin>yeah, they're right.

00:26:38.329 --> 00:26:47.005
<v Danielle Golovin>So these are donated post mortem brains, and the company is keeping them molecularly active, but they're not functionally active still.

00:26:47.005 --> 00:26:52.344
<v Danielle Golovin>They don't have, they're not firing, you know, the neurons are not firing, but the cells are alive.

00:26:52.344 --> 00:27:07.525
<v Danielle Golovin>And so this whole idea started almost a decade ago when Bexorg’s Co-founder and CEO, Zvonimir Vrselja was working in a lab at Yale as a postdoc, and you might have remembered this headline, the group revived brain activity in pigs hours after they died.

00:27:07.625 --> 00:27:14.932
<v Danielle Golovin>I remember this, I was in grad school and what a, what a fun lunch conversation with my colleagues, in grad school, the pig is, you know, revived.

00:27:15.599 --> 00:27:16.201
<v Danielle Golovin>So.

00:27:16.334 --> 00:27:27.244
<v Danielle Golovin>it was based on a, it's it's hemoglobin based and it's a cytoprotective perfusion platform, so they're maintaining brain circulation at normal body temperature, and that's really the key aspects.

00:27:27.578 --> 00:27:34.051
<v Danielle Golovin>It's preventing cellular death, so it's keeping it molecularly active for a few hours, but again, there's no functional activity,

00:27:34.653 --> 00:27:38.556
<v Steve Usdin>so so you, so you have a brain that's that's molecularly active for a few hours.

00:27:39.356 --> 00:27:43.228
<v Steve Usdin>And you can do something to perturb it and then you can get some kind of a readout.

00:27:43.228 --> 00:27:44.596
<v Steve Usdin>How do you, how do you do that?

00:27:44.628 --> 00:27:47.598
<v Steve Usdin>What's the input to it, and then what's the readout from it?

00:27:48.465 --> 00:27:52.069
<v Danielle Golovin>Yeah, so the input could be drug perturbations, right?

00:27:52.369 --> 00:27:53.805
<v Danielle Golovin>You put a drug on and see what changes.

00:27:53.805 --> 00:28:04.782
<v Danielle Golovin>And that's, they say they have multi-scale data from tissue plasma CSF, so that's cerebral spinal fluid, and this is spatial and temporal responses, and they're using, they've even had some experience with.

00:28:05.583 --> 00:28:14.159
<v Danielle Golovin>Postmortem brains with neurodegenerative diseases, Alzheimer's, ALS, FTD, and when you put the drug on, you can get PK biodistribution.

00:28:14.159 --> 00:28:16.728
<v Danielle Golovin>You can see if it penetrates the blood-brain barrier.

00:28:17.061 --> 00:28:27.505
<v Danielle Golovin>So just a lot about de-risking, you know, what the drug's gonna act like in the clinic is basically their, their big thesis, they have 42 and a half million in financing.

00:28:28.239 --> 00:28:32.977
<v Selina Koch>But can't tell you if it modulates synaptic transmission or plasticity.

00:28:33.010 --> 00:28:34.445
<v Selina Koch>'cause those things are not active.

00:28:35.145 --> 00:28:35.480
<v Danielle Golovin>True.

00:28:35.547 --> 00:28:36.681
<v Danielle Golovin>There are limitations here,

00:28:36.948 --> 00:28:37.248
<v Selina Koch>Yeah.

00:28:37.781 --> 00:28:39.250
<v Steve Usdin>and then what's it, how's it gonna work?

00:28:39.483 --> 00:28:42.820
<v Steve Usdin>they're, they're providing this as a service for other companies.

00:28:42.820 --> 00:28:45.690
<v Steve Usdin>Are they gonna use this as a platform for developing their own drugs?

00:28:46.924 --> 00:28:47.291
<v Danielle Golovin>so they.

00:28:47.759 --> 00:28:51.395
<v Danielle Golovin>Told us, you know, for the, for the ECP, that they have a three track strategy.

00:28:51.496 --> 00:28:54.065
<v Danielle Golovin>Near is collaborations with pharma and biotech companies.

00:28:54.065 --> 00:28:58.569
<v Danielle Golovin>So they had a June partnership with Biohaven to advance to their preclinical programs.

00:28:58.970 --> 00:29:04.776
<v Danielle Golovin>Then they're gonna complete their, what they're calling a brain native AI model, in the medium term.

00:29:04.843 --> 00:29:08.145
<v Danielle Golovin>And then eventually plan to develop an internal pipeline.

00:29:08.880 --> 00:29:09.146
<v Jeff Cranmer>yep.

00:29:09.247 --> 00:29:10.848
<v Jeff Cranmer>they have a presence in New Haven.

00:29:10.914 --> 00:29:12.182
<v Jeff Cranmer>Uh, not surprising, but also.

00:29:12.916 --> 00:29:18.123
<v Jeff Cranmer>out  my neck of the woods and Selina's, neck of the woods, San Francisco.

00:29:18.355 --> 00:29:24.461
<v Jeff Cranmer>another good company to watch and, keep a lookout for the next companies in our emerging profile series.

00:29:24.461 --> 00:29:29.968
<v Jeff Cranmer>Thanks for that, Danielle, and thanks to Selina and Steve for joining as well.

00:29:30.134 --> 00:29:37.541
<v Jeff Cranmer>And thank you to you for tuning in if you like what you're hearing, subscribe, follow, like all those terms that you hear.

00:29:38.009 --> 00:29:45.115
<v Jeff Cranmer>shout out to Kendall Square Orchestra, which creates the music for BioCentury's podcasts.

00:29:45.415 --> 00:29:46.317
<v Jeff Cranmer>We'll catch you next week.