Ep. 348 - Biotech's 2026 Catalysts. Plus: China's New Orphan Rules

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<v Jeff Cranmer>RNAi and exon skipping therapies headline the list of regulatory and pivotal data Catalysts in 2026.

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<v Jeff Cranmer>Meanwhile, new lipid lowering mechanisms, modalities and precision medicines will define the year in cardiovascular disease.

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<v Jeff Cranmer>Part two of our discussion of the catalyst that will drive biotech this year is on the plate for this week's BioCentury This week podcast.

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<v Jeff Cranmer>And we'll also discuss a new regulation in China, introducing orphan drug exclusivity, A move that could bolster the market for rare disease drugs, and incentivize their development.

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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 Simone Fishburn>Simone Fishburn, Editor in Chief.

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<v Selina Koch>Selina Koch, Executive Editor.

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<v Lauren Martz>Lauren Martz, Executive Director of Biopharma Intelligence.

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<v Steve Usdin>And Steve Usdin, Washington Editor.

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<v Jeff Cranmer>All right.

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<v Jeff Cranmer>We'll be jumping into the second part of our discussion of the key catalysts in the biotech sector in just a moment.

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<v Jeff Cranmer>Of course, you'll remember.

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<v Jeff Cranmer>Uh, or if you didn't remember, and you are interested in neuro catalyst checkout last week's podcast,  Selina's gave us a little, preview of, uh, her, her deck and, story that she, uh, cooked up for us.

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<v Jeff Cranmer>But first, Bio€quity Europe it's coming up in May.

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<v Jeff Cranmer>This year's theme, the Need for Speed, how Europe can compete in the New Era.

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<v Jeff Cranmer>It's the first time we're taking our longstanding conference.

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<v Jeff Cranmer>This will be the 26th year to Prague.

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<v Jeff Cranmer>Advanced pricing is in effect through April two, and that's no joke.

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<v Jeff Cranmer>Limited presenting company presentation presentation slots and sponsorship opportunities remain.

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<v Jeff Cranmer>Don't wait.

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<v Jeff Cranmer>This meeting always sells out.

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<v Jeff Cranmer>if you're interested in being a presenting company, reach out to me directly.

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<v Jeff Cranmer>And, our sponsorship team, can be reached at conferences @BioCentury.com, and you can go to Bio€quityEurope.com.

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<v Jeff Cranmer>Alright.

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<v Jeff Cranmer>RNAi, Lauren, great deep dive here.

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<v Jeff Cranmer>Where do you wanna start?

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<v Lauren Martz>Thanks Jeff.

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<v Lauren Martz>when we're talking about the RNAi catalyst, RNAi and Antisense catalyst for 2026.

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<v Lauren Martz>I think the big one that we're hearing a lot about sort of covers that new modality theme and it also covers the cardiovascular disease theme that, is the highlight of another one of the stories that, that we ran last week.

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<v Lauren Martz>And that is the first Phase III data for an Lp(a) program.

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<v Lauren Martz>this half.

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<v Lauren Martz>We expect to see Phase III data from the Horizon study of Pelacarsen.

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<v Lauren Martz>And this is  from Ionis and Novartis.

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<v Lauren Martz>And this is a cardiovascular outcomes trial.

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<v Lauren Martz>So this will be the first time that the hypothesis is tested, the hypothesis of whether reducing Lp(a) actually translates to cardiovascular benefits for the patients who have high levels of this target.

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<v Lauren Martz>So that's about 30%, up to 30% of cardiovascular disease patients.

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<v Lauren Martz>So it could possibly be a, a pretty important contributor to, you know, residual risk in patients who are treated with other mechanisms.

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<v Lauren Martz>I think this is a big deal for the RNA field and the cardiovascular disease field just because, well first of all, it's a big deal for the target because it's a very popular development area right now.

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<v Lauren Martz>This is one of four programs in Phase III testing across different modalities against this target.

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<v Lauren Martz>It's the only one to read out this year.

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<v Lauren Martz>And it's from Phase II data the levels of Lp(a) reduction achieved for this program are slightly lower than the other three programs.

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<v Lauren Martz>So if this is successful efficacy-wise, that's a good indicator for the other programs.

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<v Lauren Martz>We've also heard from companies in the earlier stages of development that that how this study turns out might determine whether they go forward with a compound that they already have more advanced in preclinical development versus, trying to come up with something that can reduce Lp(a) levels even further.

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<v Lauren Martz>Yeah, I think it's  potentially another example, of an RNA or antisense therapy in this cardiometabolic space that could become a a clinical success is kind of making this modality more established.

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<v Lauren Martz>We've recently seen the PCSK9.

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<v Lauren Martz>field move forward too.

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<v Lauren Martz>We'll see what happens.

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<v Selina Koch>So on the Lp(a), I think it's also, I see it as, it's gonna be an important benchmark.

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<v Selina Koch>The extent of Lp(a) reduction they see relative to how much clinical benefit they see.

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<v Selina Koch>Everybody will learn from that.

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<v Selina Koch>But I think the other interesting parameter there is just durability, right?

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<v Selina Koch>I think these next ones coming down the line are longer acting perhaps.

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<v Simone Fishburn>And would you say that that's really gonna be.

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<v Simone Fishburn>what makes 'em take off versus small molecules or something like that?

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<v Simone Fishburn>The ability of RNAi to just produce, what, what are they shooting for Lauren?

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<v Simone Fishburn>Once a year, like twice a year at the moment moment.

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<v Simone Fishburn>I think ultimately, you know, Vas Narasimhan from Novartis has talked about trying to get very long acting products out there.

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<v Simone Fishburn>So How much of this is dependent on their ability to be really long lasting as a modality?

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<v Lauren Martz>I think that's incredibly important for all of these RNA targeting RNA based therapies.

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<v Lauren Martz>That's the benefit that we're hoping to see.

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<v Lauren Martz>That's the difference from taking, you know, your statin.

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<v Lauren Martz>Every day.

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<v Lauren Martz>But within the Lp(a) space, this is a precision medicine opportunity.

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<v Lauren Martz>These are, you know, this will be for a, potentially a targeted group of patients.

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<v Lauren Martz>And, as I mentioned at the beginning, there's, there are multiple different modalities in the advanced stages of development for reducing Lp(a) so.

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<v Lauren Martz>I think how successful each one is will depend on the durability, will depend on, you know,  as Selina mentioned,  how much the risk comes down with it, with its ability to reduce levels of, of Lp(a), so

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<v Selina Koch>But it's

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<v Lauren Martz>be

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<v Selina Koch>Great to see a genetic, a genetic target advancing.

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<v Selina Koch>And even though it's precision and it's great that it's precision and that's gonna increase its chances of success, it's also large.

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<v Selina Koch>Right?

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<v Selina Koch>So it's one of those windows of opportunity.

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<v Selina Koch>Um, yeah.

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<v Simone Fishburn>about outside of cardiovascular or cardiometabolic?

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<v Simone Fishburn>Because you've also been looking at the neuromuscular space, right.

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<v Simone Fishburn>Talk a little bit about that and the importance of RAI there and the catalysts.

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<v Lauren Martz>Sure, so with the catalyst that we found and that we chose to focus on, we found 10 Phase III trials across different indications that are expected to read out for antisense or siRNA therapies this year, which is a lot.

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<v Lauren Martz>And I think what was interesting for me was the fact that this was, it spans so many different indications.

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<v Lauren Martz>You've got a Phase III trial from Roche for IgA nephropathy, Angelman syndrome is in there.

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<v Lauren Martz>And then as you mentioned, um, a bunch of the siRNA, Phase III readouts are for musculo skeletal diseases.

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<v Lauren Martz>There are also a bunch of exon skipping antisense in there too.

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<v Lauren Martz>I think that those, there's been a lot of activity in DMD, for example.

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<v Lauren Martz>Um, we, you know, ev everyone's following the gene therapy approval.

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<v Lauren Martz>there's, the potential for a second gene therapy, to maybe reach the market this year, from Regenxbio.

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<v Lauren Martz>And, just given anyone who's been following the story of the gene therapies, in this indication, I think having a second option is a really big deal for patients.

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<v Lauren Martz>There are also next generation versions of the exon skipping therapies that will, have late stage data this year.

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<v Lauren Martz>You know, the Exon 53 and Exon 51, I think there's an Exon 54, which doesn't have any approved therapies.

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<v Lauren Martz>With a big step change in the level of exon skipping and the level of dystrophin expression that's, achieved with those.

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<v Lauren Martz>So that could be really important for patients.

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<v Lauren Martz>And then there are multiple late stage milestones in musculoskeletal diseases that don't have approved therapies, Myotonic Dystrophy Type 1 could see Phase III data this year.

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<v Lauren Martz>And FSHD could also have Phase III data.

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<v Lauren Martz>Which I, I think is very exciting.

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<v Selina Koch>Let's take it from the company perspective.

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<v Selina Koch>Um, maybe, some of the leaders in this particular technology, I mean, obviously Ionis has been in antisense forever as a pioneer, but it seems like it's gonna be a big year and especially big year for them.

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<v Selina Koch>They have four, I think, readouts in pivotal ones in your, in your analysis deck.

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<v Selina Koch>They have some regulatory things happening, but, who else should we keep an eye on?

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<v Lauren Martz>Sure.

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<v Lauren Martz>So, uh, from.

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<v Lauren Martz>When we're talking about Big Pharmas, there are quite a few from Novartis.

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<v Lauren Martz>So Novartis has some programs that are collaborations with Ionis.

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<v Lauren Martz>We mentioned pelacarsen, the Myotonic dystrophy and FSHD programs that will have Phase III readouts are coming from Novartis.

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<v Lauren Martz>Um, Novartis, uh, is also acquiring Avidity, which should have some Phase III data this year.

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<v Lauren Martz>We have companies like Arrowhead and Wave, uh, that are very active in the late stage catalyst as well.

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<v Lauren Martz>Biogen has, some spinal muscular atrophy, data expected, and some data for an anti tau.

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<v Lauren Martz>So there are  a lot of players that are listed in our story about this topic.

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<v Steve Usdin>Well, Lauren, I guess there's another company you gotta mention too, right?

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<v Steve Usdin>Alnylam.

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<v Lauren Martz>Of course.

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<v Lauren Martz>Yeah.

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<v Lauren Martz>So Alnylam, um, features a lot in our proof of concepts catalysts analysis.

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<v Lauren Martz>they have a bleeding disorder program that should have Phase I and Phase II data this year.

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<v Lauren Martz>There's an RNAi therapy for Huntington's Disease.

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<v Lauren Martz>That's, um, a partnership between Alnylam and Regeneron.

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<v Lauren Martz>So the, um, definitely another player to watch in this space.

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<v Jeff Cranmer>Alright.

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<v Jeff Cranmer>Yeah, you can't, you can't, uh, not mention Alnylam when it comes to talking about, key catalysts the way that company has been rolling over the past couple of decades.

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<v Jeff Cranmer>Lauren Renal also factored into the three stories you wrote for this five part package of catalysts.

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<v Jeff Cranmer>what was the most compelling thing you found in that space?

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<v Lauren Martz>So there were two take homes from the analysis of the renal disease catalyst, and this was a smaller set, but I think it's just a really important space right now.

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<v Lauren Martz>So the first is that the progress that we've seen in IgA nephropathy is going to continue.

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<v Lauren Martz>There's a ton of activity that's still coming up through the pipeline for this indication that's seen five drugs approved in the past five years.

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<v Lauren Martz>And the other take home message is that other indications within this renal disease space.

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<v Lauren Martz>I think are starting to kind of benefit from the, the clinical development and regulatory path that's been created for IgA nephropathy.

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<v Lauren Martz>So there are at least three renal diseases that don't have therapy specifically approved to treat them, that will either have late stage data or, regulatory submissions this year.

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<v Lauren Martz>What's been happening in IgA nephropathy is the first approval was in 2021, based on a surrogate

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<v Jeff Cranmer>First ever.

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<v Jeff Cranmer>I mean, that's, that's one of the things you really highlighted, right?

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<v Jeff Cranmer>It was like five years ago there was nothing for that.

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<v Lauren Martz>Absolutely.

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<v Lauren Martz>And this was based on reduction in proteinuria as a surrogate endpoint.

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<v Lauren Martz>And you know, that data, the fact that it was able to be used then, it's been used for these subsequent approvals and you know, all the whole body of evidence supporting that surrogate endpoint.

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<v Lauren Martz>Is what's made that an attractive development, path for a lot of companies.

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<v Lauren Martz>So we continue to see additional programs and additional mechanisms move through the pipeline for this indication, We have six, potential catalysts based on our list.

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<v Lauren Martz>Two of them are BLA submissions, three of them are Phase III data.

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<v Lauren Martz>So.

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<v Selina Koch>Beyond that, what really struck me, Lauren, is you have the full pipeline in there.

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<v Selina Koch>I think there's like 15 development programs that you highlighted, even if some of them don't have catalysts this year.

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<v Selina Koch>Just goes to show you what happens when you have a well worked out regulatory path like that.

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<v Selina Koch>Yeah.

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<v Lauren Martz>and in some cases these are, um, therapies that are approved for other indications so we can, you know, take it forward in this, what used to be an untreatable disease.

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<v Lauren Martz>Based on that pathway.

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<v Lauren Martz>And then my second point was these other renal diseases that are having potentially important readouts this year.

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<v Lauren Martz>So there's APOL1-mediated kidney disease.

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<v Lauren Martz>We are highlighting two catalysts.

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<v Lauren Martz>One of them is an interim Phase II/III readout.

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<v Lauren Martz>We've also got proof of concept data that's, um, from Vertex and Maze Therapeutics.

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<v Lauren Martz>FSGS, there is a PDUFA date for FILSPARI from Travere, which is already approved for IgA nephropathy, And that's one where, you know, it missed the,

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<v Jeff Cranmer>April 13th, I think.

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<v Lauren Martz>Yeah, so it, it missed the traditional endpoint that you would wanna see to get approval on that indication, but it did have a benefit on, um, the proteinuria levels.

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<v Lauren Martz>So, that'll be really interesting to see what happens there.

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<v Lauren Martz>And then the other indication was membranous nephropathy, which is, an inflammatory autoimmune type disease where, Roche has its anti with a potential sNDA submission this year and some Phase III data.

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<v Jeff Cranmer>It's a, it's a really great table you put together for this story as, uh, you can quickly see what the mechanism is, product, catalyst, timing.

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<v Jeff Cranmer>Great piece here, Lauren.

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<v Jeff Cranmer>we also had our colleague, Stephen Hansen, added again with obesity, a space that he watches quite closely.

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<v Jeff Cranmer>Uh, heading into 2026, Steven says The obesity race is widening.

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<v Jeff Cranmer>There, I said it.

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<v Jeff Cranmer>Some recent readouts, have been teeing up new mechanisms, new players that could challenge the standard, that's GLP-1 of course, on efficacy or convenience.

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<v Jeff Cranmer>the biggest obesity catalyst, he is watching, it's already underway at this point.

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<v Jeff Cranmer>The launch of the oral GLP-1 receptor agonist.

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<v Jeff Cranmer>Novo is already out there Lily is gonna follow.

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<v Jeff Cranmer>So if you're interested in the obesity space, definitely check out Stephen's story, it like most of these other stories, comes with a very handy slide deck that you can kind of cruise right through and, and zero zero in on what's most interesting for you.

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<v Jeff Cranmer>You can download it and the like,

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<v Selina Koch>And when you go there, you'll see we have catalyst, at different phases of the development spectrum, right?

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<v Selina Koch>We have, um, regulatory catalyst, we have Phase III catalyst, but we also are really keen to look at proof of concept for new targets, new technologies, lots of proof of concept in there, and you'll find in the obesity one, there are some that follow the same theme of RNA therapies.

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<v Selina Koch>So some of the newer targets like INHBE a genetic target, ALK7 a genetic target.

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<v Selina Koch>There you have Alnylam, you have Wave Life sciences, um, with knockdown approaches, and others.

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<v Jeff Cranmer>a lot of interesting stuff happening.

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<v Jeff Cranmer>And, uh, well, Stephen does talk quite a bit about, the titans in the space.

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<v Jeff Cranmer>I, I, I'm trying to remind Stephen not to use titan so often, but you know, it's rarely appropriate.

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<v Jeff Cranmer>But in this case, Novo and Lily, uh, are some serious market leaders, but AstraZeneca had a very busy week last week.

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<v Jeff Cranmer>One thing it did was it, it kind of signaled that it is gonna go all in on obesity  via yet another China deal for the company.

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<v Jeff Cranmer>$1.2 billion upfront, partnering with CSPC, one of the bigger players in China.

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<v Jeff Cranmer>The biobucks are, uh, they're bio Bucky, 18.5 billion.

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<v Jeff Cranmer>And what Stephen wrote was that, this is really showing that AstraZeneca is planning on joining Pfizer and Roche, in making an all in bet to compete with Lily and Novo.

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<v Jeff Cranmer>Big week for AstraZeneca.

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<v Jeff Cranmer>A big week for the U.K. and China.

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<v Jeff Cranmer>Simone.

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<v Jeff Cranmer>Uh, tear in the eye for your motherland?

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<v Simone Fishburn>Tear in the eye?

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<v Simone Fishburn>I don't think so.

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<v Simone Fishburn>Um, wouldn't, yeah, gotta think about that one, Jeff.

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<v Simone Fishburn>Um, I, I think that it's, just, to be honest, I think this is part of a broader geopolitical picture where the U.K. is cozying up to China.

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<v Simone Fishburn>Hedging, given that it's very unsure of where it is with the U.S. You know, we know from AstraZeneca's point of view, AstraZeneca has been, I think for a while now, the most heavily invested pharma in, or western pharma, I should say, in China.

00:17:22.875 --> 00:17:26.577
<v Simone Fishburn>They've had a very big footprint, notwithstanding some of the setbacks they've had.

00:17:27.078 --> 00:17:35.253
<v Simone Fishburn>I, I just see this as, as part of a, picture that is definitely led by AstraZeneca, not exclusive to them.

00:17:35.421 --> 00:17:36.221
<v Simone Fishburn>A lot of pharmas.

00:17:36.221 --> 00:17:45.963
<v Simone Fishburn>I know, Steve, you've got some thoughts on this that we'll tap into, but I think that, You know, China is a very real alternative, to the US and to Europe at the moment.

00:17:46.164 --> 00:17:48.700
<v Simone Fishburn>Cause these are R&D moves that they're building there.

00:17:49.701 --> 00:18:07.586
<v Steve Usdin>one, one thing that was really remarkable though is that the press release that AstraZeneca put out about their $15 billion investment in China included a paragraph of quotes from Keir Starmer, the U.K. Prime Minister, hailing AstraZeneca's investments in China.

00:18:08.019 --> 00:18:15.560
<v Steve Usdin>And if you stop and think about it a moment, it's really difficult to imagine that scenario in the United States, for example, or, or anywhere else.

00:18:15.560 --> 00:18:22.768
<v Steve Usdin>Can you imagine a US company investing$15 billion in China and US president coming out and saying, well, that's great.

00:18:22.768 --> 00:18:25.436
<v Steve Usdin>It's showing America America's leadership.

00:18:25.537 --> 00:18:26.270
<v Steve Usdin>so it's, uh,

00:18:26.438 --> 00:18:26.672
<v Jeff Cranmer>uh,

00:18:26.704 --> 00:18:27.873
<v Steve Usdin>you know, it was quite remarkable.

00:18:27.940 --> 00:18:36.848
<v Simone Fishburn>one bit of context that I wanna add, which is, um, so the U.K. has made biotech and biopharma biopharma one of its pillars of growth.

00:18:36.848 --> 00:18:37.082
<v Simone Fishburn>Okay?

00:18:37.082 --> 00:18:38.817
<v Simone Fishburn>It's one of its major pillars of growth.

00:18:39.617 --> 00:18:56.501
<v Simone Fishburn>We know that China did a similar thing, what is it, 15 years ago, whenever they basically set out a strategy to grow their biopharma ecosystem, and this is something that was never strategically done in the U.S. It's sort of, is that fair to say that Steve?

00:18:56.501 --> 00:19:01.573
<v Simone Fishburn>I know that there were, you know, tax incentives in, say, Massachusetts and places like that.

00:19:01.807 --> 00:19:04.643
<v Simone Fishburn>But I don't know that there was ever a top down, strategy.

00:19:04.643 --> 00:19:17.288
<v Simone Fishburn>But what I'm trying to say is that I think that this move in the statement by Starmer is consistent with both the bigger geopolitical thing and the idea that the country wants to, reinforce its own life sciences, footprint.

00:19:18.056 --> 00:19:24.896
<v Steve Usdin>Well, you know, I, I think actually if you look at what's happened in what happened in the United States, what really created the biotech industry in the United States.

00:19:25.596 --> 00:19:32.203
<v Steve Usdin>Was a very happy confluence of public policy and science and economic opportunity.

00:19:32.371 --> 00:19:55.359
<v Steve Usdin>But it was, it was based, the foundation of it was good public policy in the United States, starting from massive investment in basic science support for, for IP, a world-class independent regulatory environment, and  an immigration system that welcomed the best and the brightest from around the world.

00:19:55.359 --> 00:20:00.932
<v Steve Usdin>All of those things came together and made the biotech industry possible.

00:20:01.133 --> 00:20:11.643
<v Steve Usdin>And made the United States for a time, and maybe, um, maybe that time has passed, but for a time for and made the United States the absolute best place in the world to discover and develop drugs.

00:20:12.944 --> 00:20:26.023
<v Jeff Cranmer>Speaking of Starmer and, uh, immigration, Simone, uh, is there any truth to the rumor that, uh, Starmer is in negotiations with Spain for Carlos Alcaraz?

00:20:29.493 --> 00:20:32.497
<v Simone Fishburn>Now that that would be a, a rumor worth reporting.

00:20:32.497 --> 00:20:34.732
<v Simone Fishburn>I, I definitely think, but you know what, Jeff?

00:20:34.732 --> 00:20:42.708
<v Simone Fishburn>Um, there's really only one way to do this, and you just start a rumor and, uh, maybe it comes true, which is what I think you just did there actually.

00:20:42.840 --> 00:20:43.642
<v Jeff Cranmer>I think I did.

00:20:43.642 --> 00:20:51.148
<v Jeff Cranmer>And, uh, you know, it's no secret that, Djokovich is the, uh, the villain of the BioCentury This Week podcast.

00:20:51.148 --> 00:20:54.286
<v Jeff Cranmer>So, extra kudos, to Alcaraz.

00:20:54.752 --> 00:20:57.855
<v Simone Fishburn>I just wanna say Stephen Hansen, very lucky that he's not here.

00:20:57.855 --> 00:21:07.798
<v Simone Fishburn>Very lucky he's still got his job because he texted me at the wrong moment causing Sinner to not convert two break points and end up losing.

00:21:07.798 --> 00:21:10.701
<v Simone Fishburn>And so that was a very dangerous part of the weekend

00:21:10.736 --> 00:21:12.203
<v Jeff Cranmer>It, it was, um,

00:21:12.336 --> 00:21:13.105
<v Simone Fishburn>But we'll move on.

00:21:13.305 --> 00:21:14.439
<v Simone Fishburn>Stephen Hansen has many other

00:21:14.640 --> 00:21:17.643
<v Jeff Cranmer>I'm bracing to give him his performance review.

00:21:17.709 --> 00:21:19.911
<v Jeff Cranmer>Um, after, after that episode.

00:21:20.178 --> 00:21:25.549
<v Jeff Cranmer>Alright, we're gonna take a quick break, and we will be back to talk China.

00:21:25.549 --> 00:21:26.250
<v Jeff Cranmer>What else?

00:21:26.518 --> 00:21:31.155
<v Alanna Farro>BioCentury This Week is brought to you by The 5th East-West Biopharma Summit in South Korea.

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00:22:14.633 --> 00:22:15.166
<v Jeff Cranmer>Okay.

00:22:15.166 --> 00:22:19.203
<v Jeff Cranmer>We are back on the BioCentury This Week podcast.

00:22:19.337 --> 00:22:23.508
<v Jeff Cranmer>Check out our catalyst package, it's awesome What more could I say about it?

00:22:23.508 --> 00:22:25.309
<v Jeff Cranmer>Go read all five stories.

00:22:25.609 --> 00:22:26.545
<v Jeff Cranmer>Four decks.

00:22:26.845 --> 00:22:41.559
<v Jeff Cranmer>And let's turn now to China where Steve is extending biotech's long history of digging into the regulations that have unlocked biotech innovation in China, which shows no signs of letting up.

00:22:41.559 --> 00:22:48.467
<v Jeff Cranmer>Last week we talked about the new rules, that will accelerate the country's cell gene therapy sector.

00:22:48.733 --> 00:22:53.171
<v Jeff Cranmer>And today, uh, Steve, let's talk about your, the second story you wrote last week.

00:22:53.337 --> 00:23:09.121
<v Jeff Cranmer>New law taking effect May 15th, which for the first time is going to grant orphan drugs, seven years of market exclusivity, plus two more years of exclusivity for pediatric indications.

00:23:09.121 --> 00:23:09.820
<v Jeff Cranmer>What did you learn, Steve?

00:23:10.721 --> 00:23:15.594
<v Steve Usdin>I think it's, it, it is really interesting to see, you know, this is another example I think.

00:23:15.861 --> 00:23:16.361
<v Steve Usdin>of China.

00:23:16.361 --> 00:23:27.405
<v Steve Usdin>Looking at what were the, the pillars I just mentioned before, the kind of the pillars that of public policy that made, the biotech industry flourish in United States and and in other countries.

00:23:27.772 --> 00:23:30.776
<v Steve Usdin>And orphan drug exclusivity was certainly one of them.

00:23:31.009 --> 00:23:42.253
<v Steve Usdin>I have to say though, there's a great deal of uncertainty still, even though these laws are gonna go in, this law's gonna go into effect May 15th, about how it's gonna work, and one of the elements of uncertainty is something that you just mentioned, okay.

00:23:42.253 --> 00:23:45.190
<v Steve Usdin>So there's seven years of orphan exclusivity.

00:23:45.457 --> 00:23:52.063
<v Steve Usdin>There's two years of pediatric exclusivity, what isn't known yet, but will be I think before May 15 known.

00:23:52.696 --> 00:24:06.678
<v Steve Usdin>Is whether that two years is gonna be tacked onto the end of the seven years for an orphan, drug that is relevant for a pediatric indication or whether that two years will be included in the seven years.

00:24:06.678 --> 00:24:19.156
<v Steve Usdin>So we don't know if it's seven plus two, if it's nine for an orphan drug, for a pediatric indication, or if it's just seven for an orphan indication, period regardless of whether it's for, pediatric or not.

00:24:19.324 --> 00:24:24.462
<v Steve Usdin>So I spoke with, CANbridge, CEO, James Xue, a former Genzyme executive.

00:24:24.695 --> 00:24:25.730
<v Steve Usdin>And he told me that there are other.

00:24:26.131 --> 00:24:29.733
<v Steve Usdin>Important unanswered questions about, um, the orphan exclusivity.

00:24:30.335 --> 00:24:33.204
<v Steve Usdin>Starting with the definition of rare disease.

00:24:33.605 --> 00:24:34.705
<v Steve Usdin>China doesn't have one.

00:24:35.039 --> 00:24:41.813
<v Steve Usdin>It has two catalogs, listing 207 rare diseases, but obviously that's only a small portion, right?

00:24:41.813 --> 00:24:43.515
<v Steve Usdin>There's over 10,000 rare diseases.

00:24:43.815 --> 00:24:51.288
<v Steve Usdin>It also, as I said, there's also this uncertainty about how the, um, two year pediatric exclusivity, will work.

00:24:51.589 --> 00:25:06.403
<v Steve Usdin>But overall, you know, what he said is that up until now for Orphan drugs, both for, um, orphan drug development and especially as in terms of sales of orphan drugs, China has really been an afterthought.

00:25:06.637 --> 00:25:19.483
<v Steve Usdin>His hope is that orphan drug exclusivity will make China a viable market for orphan drugs, both for developing them and, um, as a market for selling orphan drugs into China.

00:25:19.917 --> 00:25:25.022
<v Steve Usdin>I think there's still a lot of things, a lot of uncertainty that has to happen in order to make that happen.

00:25:25.323 --> 00:25:31.028
<v Steve Usdin>My personal view is that the orphan drug exclusivity, the impact of that.

00:25:31.429 --> 00:25:33.431
<v Steve Usdin>Has been exaggerated in the United States.

00:25:33.464 --> 00:25:34.499
<v Steve Usdin>It was important.

00:25:35.165 --> 00:25:48.046
<v Steve Usdin>But the thing that really created the orphan drug industry in the United States was Henry Tamir, the CEO of Genzyme, figuring out that you could charge a great deal for orphan drugs.

00:25:48.113 --> 00:25:57.355
<v Steve Usdin>And if you had orphan drugs that were really solving unmet medical needs, governments and other payers would pay very, very high prices for orphan drugs.

00:25:57.355 --> 00:26:00.025
<v Steve Usdin>And that created tremendous market incentive.

00:26:00.025 --> 00:26:10.000
<v Steve Usdin>It was bolstered by the seven years of exclusivity because, um, some of the orphan drugs had ambiguous or, or lacking, uh, were lacking in patent protection.

00:26:10.201 --> 00:26:18.309
<v Steve Usdin>But the key to it, is the reimbursement, the reimbursement market in China, reimbursement environment in China, I should say, is quite complex.

00:26:18.542 --> 00:26:22.113
<v Steve Usdin>But there is reimbursement for orphan drugs, especially for orphan drugs.

00:26:22.580 --> 00:26:24.583
<v Steve Usdin>Uh, and it makes it, you know, viable.

00:26:24.583 --> 00:26:39.564
<v Steve Usdin>What, um, what the CEO of CANbridge said to me is that it really makes it viable to develop and to sell orphan drugs in China for markets that are in what, you know, what he called the, kind of the medium range of rare, medium, rare, 10,000 and above.

00:26:39.998 --> 00:26:41.700
<v Steve Usdin>I think it'll be very interesting to watch.

00:26:42.467 --> 00:26:45.470
<v Steve Usdin>One, how this law actually is implemented in practice.

00:26:45.836 --> 00:26:57.648
<v Steve Usdin>And two, what its impact is both in terms of creating a market for in-licensing drugs, um, to China and also for China becoming a springboard for developing orphan drugs for the world market.

00:26:58.215 --> 00:27:00.618
<v Selina Koch>I think that's really interesting, the implications.

00:27:00.618 --> 00:27:09.594
<v Selina Koch>So if it takes off and it supports development of orphan disease therapies in China, 'cause there's now, um, you know, more exclusivity, a better market.

00:27:10.060 --> 00:27:27.545
<v Selina Koch>Presumably the clinical trial infrastructure would mature there for rare diseases, cause when I, you know, like we just, some of us went to JP Morgan recently and we were running around talking dialogues of companies and one of the things I asked everybody I met was, well, who was heading into Phase I, I'm like, where are you doing your Phase I trials?

00:27:27.545 --> 00:27:33.284
<v Selina Koch>And when it came to rare diseases and a lot of rare, rare neurological ones, nobody had China on their map.

00:27:33.785 --> 00:27:38.789
<v Selina Koch>Even though China's becoming a destination for clinical trials for all kinds of things, including early stage trials.

00:27:39.190 --> 00:27:41.859
<v Selina Koch>And so I wonder if it will trickle down to

00:27:41.992 --> 00:27:44.095
<v Steve Usdin>Yeah, that's, that that's gonna be really interesting to watch.

00:27:44.729 --> 00:27:47.097
<v Steve Usdin>I, I don't, I don't think anybody knows the answer to that yet.

00:27:47.798 --> 00:27:49.034
<v Jeff Cranmer>Where are they doing their trial?

00:27:49.834 --> 00:27:51.269
<v Selina Koch>Early stage trials where people doing them?

00:27:51.736 --> 00:27:52.037
<v Jeff Cranmer>Yeah.

00:27:52.436 --> 00:27:53.904
<v Selina Koch>Well, it depends on your modality.

00:27:53.904 --> 00:28:05.717
<v Selina Koch>Like for some, some companies with genetic medicines, they don't find the US is, um, especially accommodating, some of the requirements, here are a little burdensome sometimes what FDA requires for safety margins.

00:28:05.717 --> 00:28:12.891
<v Selina Koch>It seems like it's still focused on what you would do for small molecules, say, so people are going to Europe, they're going to Australia.

00:28:12.891 --> 00:28:14.792
<v Selina Koch>Of course, Australia's been a destination.

00:28:14.792 --> 00:28:15.826
<v Selina Koch>They're going to Canada.

00:28:16.694 --> 00:28:28.306
<v Selina Koch>And increasingly what I thought was really interesting is how many people said that they were at least considering a U.K, earlier U.K. first, 'cause MHRI has been very accommodating of late.

00:28:29.273 --> 00:28:29.907
<v Jeff Cranmer>Interesting.

00:28:29.907 --> 00:28:36.580
<v Jeff Cranmer>And, and I'm hearing more and more that the U.K. is interested in becoming a, a magnet for that.

00:28:36.580 --> 00:28:39.584
<v Jeff Cranmer>Simone, didn't you, uh, look into something on that front?

00:28:40.050 --> 00:28:41.318
<v Jeff Cranmer>U.K. speeding up the dials.

00:28:41.419 --> 00:28:51.296
<v Simone Fishburn>So, uh, Lawrence Tallon, who is the CEO MHRA, he, oh, I forget what month it was, but he joined last year.

00:28:51.296 --> 00:28:56.667
<v Simone Fishburn>He took the role last year and I mean, the first thing they really wanted to do is stop the decline.

00:28:56.901 --> 00:29:02.574
<v Simone Fishburn>Their time from, like the clinical startup time for first inhuman was very, very long.

00:29:02.574 --> 00:29:05.477
<v Simone Fishburn>It had slipped from like 150 days to 250.

00:29:05.710 --> 00:29:16.354
<v Simone Fishburn>So I think the first thing that they wanted to do was, return to like par with the U.S. or something like 150 days, but now they've gotten more ambitious and like Selina.

00:29:16.354 --> 00:29:30.101
<v Simone Fishburn>I'm also hearing, more and more things coming out of the U.K. in terms of incentives and pathways for speeding entry to the clinic and reducing the burden, and we will be talking to them soon.

00:29:31.068 --> 00:29:40.778
<v Steve Usdin>and I've also with Lawrence Tallon about that, and they've got a, you know, kind of an integrated plan for taking advantage of some of the competitive advantages that the U.K. has.

00:29:41.112 --> 00:29:47.852
<v Steve Usdin>Excellent genomic data, genomic data that,  you know, coverage in, uh, that, doesn't exist in in other countries.

00:29:48.353 --> 00:29:59.329
<v Steve Usdin>An ability, at least in theory to link incentives for development of orphan drugs, especially for ultra orphan diseases, to an HDA and to, to payment and reimbursement.

00:29:59.564 --> 00:30:08.807
<v Steve Usdin>And I think that they do, they definitely have the ambition to become a, a kind of, a launch pad for orphan drugs and especially for ultra orphan drugs.

00:30:09.440 --> 00:30:19.750
<v Steve Usdin>With the idea that it'll be easier and quicker to develop them in the U.K. And then, to use the kind of imprimatur of the MHRA to gain,  rapid approvals globally.

00:30:20.818 --> 00:30:23.354
<v Jeff Cranmer>Alright, thanks for that, Steve.

00:30:23.587 --> 00:30:27.525
<v Jeff Cranmer>Thank you for tuning into the BioCentury This Week podcast.

00:30:27.791 --> 00:30:30.028
<v Jeff Cranmer>Like us, subscribe to us.

00:30:30.028 --> 00:30:31.695
<v Jeff Cranmer>Uh, let us know what you think.

00:30:32.029 --> 00:30:33.431
<v Jeff Cranmer>We'd love to hear from you.

00:30:33.463 --> 00:30:43.040
<v Jeff Cranmer>Uh, we appreciate our listeners and Lauren, Selina glad to see you both still on your feet after that massive catalyst package.

00:30:43.307 --> 00:30:51.915
<v Jeff Cranmer>Steve Simone, hope you continue to, navigate the glorious weather that you're having in the Washington area.

00:30:52.250 --> 00:30:57.654
<v Jeff Cranmer>I know Lauren's really enjoying making snowmen, in her yard over and over.

00:30:58.189 --> 00:31:00.325
<v Jeff Cranmer>Alright, we'll catch you next week.

00:31:00.592 --> 00:31:06.597
<v Jeff Cranmer>Special thanks to Kendall Square Orchestra for providing the music for BioCentury's podcasts.