BioCentury This Week

Ep. 320 - U.K. Biotech, U.S.-China, Insmed

• BioCentury • Season 6 • Episode 320

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Merck's announcement that it is moving its R&D out of the U.K. highlights concerns about the country’s life sciences policies. On the latest BioCentury This Week podcast, BioCentury’s analysts discuss the life sciences ecosystem in the U.K., including MHRA CEO Lawrence Tallon’s plans to create a world-class regulatory environment. They also discuss a bill passed by the U.S. House of Representatives that would end most U.S.-China academic research collaborations, and the recent success of 40-year-old biotech Insmed. This episode of BioCentury This Week is sponsored by IQVIA Biotech.

View full story: https://www.biocentury.com/article/656997

#biotech #biopharma #pharma #lifescience #MHRA #China #RandD #DrugDevelopment

00:01 - Sponsor Message: IQVIA Biotech
01:25 - U.K. Biopharma
04:40 - Grand Rounds Cambridge
09:45 - SAFE Research Act
18:01 - Spotlight on Insmed
26:32 - Sen. Cassidy's Vaccine Callout

To submit a question to BioCentury’s editors, email the BioCentury This Week team at podcasts@biocentury.com.

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[AI-generated transcript.]

Eric Pierce:

BioCentury This Week is brought to you by IQVIA Biotech. For biotech companies striving to bring innovative therapies to market and maximize patient impact, IQVIA Biotech is the trusted CRO of choice. Backed by 25 years of unparalleled experience and deep therapeutic expertise, our full-service clinical development solutions are purpose-built to accelerate success. IQVIA Biotech helps early-stage biotechs de-risk by developing strategic clinical development plans, guiding drug candidates along the most promising pathways. Leverage data-driven models and dynamic tools to craft a compelling value story and maintain momentum through every phase of drug development.

Jeff Cranmer:

Amid an exodus of pharmaceutical companies, the new head of the U.K.'s regulatory body, MHRA is focusing on the country's strengths. We will discuss what is going on at MHRA and more on the latest BioCentury this week podcast. I'm Jeff Cranmer, executive editor here and host of the BioCentury this week podcast. And joining me are my colleagues.

Simone Fishburn:

Simone Fishburn, Editor in Chief.

Steve Usdin:

Steve Usdin, Washington Editor.

Stephen Hansen:

Stephen Hansen, Director of Biopharma Intelligence.

Jeff Cranmer:

Steve, welcome to the show. I wanted to find out about your conversation with the new head of the MHRA.

Steve Usdin:

So, yeah, so I, I had an interview with, Lawrence Tallon. He's been on the job about five months. basically what we talked about is. How does he plan to position MHRA, in the U.K. and, and, globally? I think the summary would be he wants to focus on strengths, which for the U.K. are, or science, cell and gene therapies, rare diseases, vaccines, mRNA therapeutics. and he's wants to also pursue and to continue to pursue a policy which, MHRA has, has been very active in, which is, in collaborating with regulators in other countries, for, uh, mutual recognition and for reliance. the idea be basically being that, regulators in middle and smaller sized countries can, focus on their strengths and kind of reinforce each other, both in terms of technology and in terms of, um, regulatory capacity for things like, inspections and reviews.

Simone Fishburn:

Steve, so, Obviously we can't ignore the news that's come out this week. We're also on the cusp of Grand Rounds, and we're gonna have more to say at the end of the week about the feeling on the ground here regarding Merck pulling out and the pressure being put on by pharmas on the U.K. ecosystem. Let's say specifically on the government. One thing I wanted to ask you, you know, confining the conversation to this, is that one of the complaints, I mean obviously a lot of it is drug pricing, but one of the complaints is that the regulatory environment is too unfriendly as well. That was sort of in Merck's list. whether that actually is to what degree that's actually responsible for their decision, I don't know, To what degree do you think that what Lawrence Tallon is envisioning if successful, would actually address, the barriers that people see to regulation?

Steve Usdin:

Well, I, I think that one of the biggest problems in the U.K. one of the biggest concerns, is the same as, an enormous concern that companies have in the United States, which is the time that it takes to get a, a clinical trial started. and, um, to iterate and to get new, um, first in human trials on, um, iterated, drugs started and, he is, he, he announced goals there that, you know, basically would put the U.K. in line with the United States. It, it's not going to be as fast as China, it's not gonna be fast as, um, as some other countries probably, but his goal is to have it, you know, similar to the United States. So I think that would address. Some of the major issues. sense

Simone Fishburn:

do you think that's, under ambitious, Steve, or do you think that's realistic? How, how would you rate that?

Steve Usdin:

I think it's realistic and I think that, The talk about the, the regulatory, concerns in, in the U.K. is probably just adding something to the laundry list. But the truth is. That reimbursement and coverage are the real issues that the pharmas are concerned about.

Simone Fishburn:

Yeah, I think concerned is an understatement. Furious might be, um, closer to what I think they are. and, and, and you know, I think a lot of people have said, and I think that there's a lot of truth in this, that. Merck per se, is tremendously under pressure and restructuring generally. So a lot of things can be true at the same time. as I said, we'll, we'll come back to this issue when we talk to people on the ground. Stephen and I are gonna be at Grand Rounds in Cambridge this week. We have Patrick Vallance, Minister for Science in conversation with Kate Bingham. I am sure there will be questions for him, so it's gonna be an interesting, week to be here.

Steve Usdin:

and there's another dynamic at play, of course, also, which is that companies are under tremendous pressure from the Trump administration to be seen as investing heavily, in the United States, in manufacturing and in R&D. And there have been a whole stream of announcements over the last few months of very large, investments in both manufacturing and R&D in the United States. Know, it is a zero sum game. There's a certain, there's only a certain amount of money that companies have to invest globally, and if they're under, strong pressure to invest more in the United States, then that makes it more difficult for them to justify investments elsewhere.

Simone Fishburn:

Yeah. And even though I keep saying we're not gonna talk about it this week, just one more point on that front, which is, you know, it's, there's no question that having pharmas. Involved and on the ground is, a huge boost to the ecosystem. I think the question for the U.K. is how much is it a necessity? So in the U.S. obviously having them in Boston made a huge difference. The China ecosystem has actually managed to create a lot of momentum. Without necessarily having R&D as a pillar of it from the pharma companies. And not all ecosystems are the same. There's some people who are saying AI will make the difference, and the U.K. is strong in that. So we'll see where it goes. But, just coming back to where you started, which is what Lawrence Tallon is doing at MHRA. I think that that is essential. I think that, you know, that's got to be built regardless of whether the government can, bring the pharmas back to the table, because if they don't have a efficient regulatory environment, it's gonna be virtually impossible to make it a place where clinical trials are attractive to run.

Steve Usdin:

Well, and, and there's more to it also that, you know, people should read, should read the interview and the, the story that I did.

Simone Fishburn:

They should

Steve Usdin:

one one, of the things that he brought up, which I thought was, was really interesting is that if you have an environment in which there's a great deal more mutual reliance and mutual recognition, then you can envision a world in which getting an approval, for example, a product approval in the U.K. would unlock much bigger markets. If there's a, kind of a default assumption, that, MHRA approval is gonna unlock a, uh, approvals in, developing markets, for example, um, and those markets are gonna become increasingly important. then that again makes, makes it more important and more attractive,

Simone Fishburn:

'cause what you're saying is you're, then not talking about a population of 60 million. You're talking about a population of 60 million plus whatever other regions opens up. Right. And that's very different equation. Absolutely. Alright, so having said we wouldn't talk about it, Jeff, let's go back to you.

Jeff Cranmer:

Simone, you mentioned Grand Rounds. Well, it's the last call to join our conference in Cambridge U.K., starting this Wednesday. Stephen reluctantly, Oxford man coming Grand Rounds is gonna focus on how to make early stage R&D investible. And how to overcome key bottlenecks in translation. It's our third Grand Rounds. our Chicago session was a big hit in June, debut meeting last year in Nashville, Tennessee, now we're headed to the U.K. There's been a jump in registration since everyone returned from summer. and it's gonna be a packed house. There's a sold out presenting company track, sold out academic poster session, and a great lineup of speakers including Kate Bingham. And James Sabry, the former, head of deal making at Roche and Genentech, who is now at BioMarin here in California. there'll also be two days of partnering and networking. There's still time to register at BioCenturyGrandRoundsEurope.com. You can also register on site in Cambridge on the afternoon of September 17th. We'll be at the Cambridge Union Society and hope to see you there.

Simone Fishburn:

Jeff, just just one note. We're at the Cambridge Union on Wednesday night, but I believe we will also take walk-in. On Thursday, Friday, which is at Churchill College.

Jeff Cranmer:

Churchill. I've heard that name before.

Simone Fishburn:

and yes, and we will

Jeff Cranmer:

that fellow that would, uh, wake up and drink champagne?

Simone Fishburn:

they, they they like, they do like this. Churchill hit good dude here. But, um, it, importantly, Jeff, I will be sending you pictures of the buttery.

Jeff Cranmer:

I, I, you know, America, uh, say what you will about it. we don't have butteries here and, uh, I, I think that might be the cause of, uh, many of the problems in this country. Where should we go from here? Hey, why don't we head to Washington to find out about, a new piece of legislation that Steve, uncovered last week. you loved the Bio Secure Act. but wait, there's more Steve.

Steve Usdin:

So, the House of Representatives when they passed the, National Defense Authorization Act, included an amendment on the amendment, um, is called the Securing American Funding and Expertise from Adversarial Research Exploitation Act, otherwise known as the Safe Research Act, sponsored by representative John Moolenaar. The idea is basically to make it impossible for American academics to collaborate with academics in China. what the legislation would do if it's enacted and there's a lot of steps that have to happen between now and then. would basically say that, um, it would be impossible to have NIH funding for any researchers who collaborate with researchers. At institutions in China that meet certain criteria, and the criteria are very broad. Most universities that scientists in the United States, would have collaborators at, would meet, this criteria. and basically you have, you'd be erecting a wall between the United States and China at an academic level. and I think that it's something that's of, of, of real concern. To me, it's a manifestation of this idea that, biotechnology and the bio economy have been defined as national security issues. That concept is attractive in part because thinking about biotechnology and bioeconomy as national security. a way to get enthusiasm for government support. It's a way to shield, um, the industry from cuts and from some of the negative impacts of anti-science, antip, pharma, and anti-government movements in the United States. But there's a flip side to it. There's a difference between saying that an industry is a strategic asset, which I think anybody would acknowledge that biotech and, pharmaceuticals is for the United States. and that's different from saying that it's part of the defense or the military sphere, which most of biotech and most of of pharmaceuticals are not. And the, the problem is, is that when you muddle these definitions, then you get to a space like where Representative Moolenaar is. Where he says, you know, that it's a national security threat to the United States for American researchers who are conducting research on basic science, who are advancing the frontiers of biology to collaborate with researchers in China. And it's not just a, a kind of a, feel good issue or something that, um, that you think, oh, well, science should be, should be open. And, um, which of course it, it should, but they're very concrete. negative consequences from cutting off, collaborations between the United States and and China. There are papers that have been published based on these collaborations, and both parties bring really important insights and resources to the table and these collaborations.

Simone Fishburn:

so Steve, as you know, we are big fans of, collaborations, global collaborations, cross border ones, I think many people can acknowledge that China is almost past a tipping point now in terms of, uh, a little bit, like we said before, it's certainly better and healthier for everybody to have these collaborations, but maybe China is less dependent than it was several years ago. one of my questions to you is if you wanna handicap the likelihood of this passing, and the reason I ask is that I know that there are reports that sort of fairly big figures have been put in the appropriations, desired appropriations for NIH. So I wanted to ask you to comment on, on that whether it's that there are members of Congress who sort of see the light and will what we would consider see the light and will push back on this, or whether you think that, That we're really in a completely new era, and those voices just aren't there.

Steve Usdin:

You know, first just the kind of mechanics of what has to happen next. So this has passed the house. In order for it to be enacted into law, it, it has to get into the final bill, which is, uh, probably going to be, passed in December. you know, first the, the Senate is gonna pass a, a version of the bill. It's likely to be quite different from the house version, and then there's going to be. you know, behind the scenes negotiations between the House and the Senate to come up with a final version of the bill. We don't know how, how much people, in the house, how much they, they're going to push for this. whether there's gonna be any pushback in the Senate. I don't know. I think that shine the light, uh, shine some light on it, is a good thing because it will, uh, hopefully it will. Encourage people to think about it and to think about the consequences. My guess is that the vast majority of, uh, members of the house who voted for this have no idea, what it is. And they probably don't understand what the consequences would be of cutting off, scientific collaboration between United States and China. Either if all of that comes out, maybe won't happen. I think that there's enthusiasm in in Congress. In general, for disentangling, um, the United States and China in any respects where that is possible. and I think that they'll see science and, um, technology as a particularly ripe area for it. And I think there's also kind of a conflation between different areas of science and technology. The bill that the house passed doesn't specifically call out biotech or the life sciences, and I think that's probably why it hasn't gotten any attention because people don't realize it. but it would have those consequences.

Simone Fishburn:

I mean, I think certainly those of us who think that American primacy isn't a given can envisage this. In the long term, becoming a huge own goal, right? Basically the U.S. disentangling itself, while Chinese researchers can go making discoveries and partnering with other people all over the world who aren't doing this. So it, it's not necessarily a strategic benefit to the U.S. to do this.

Jeff Cranmer:

Put it gently. yeah. I've noticed like Canada is advertising on, TV here in the U.S. saying, Hey, why don't you move north? We got a place for you. So I'm sure a lot of scientists are listening to that. we're gonna go to a break shortly, but first, BioCentury and Advarra are running a survey together to better understand cancer clinical trials. want to help us identify bottlenecks and best practices to improve trial execution? Go to BioCentury survey.com. The survey only takes five minutes, and as a bonus, respondents will get a complimentary copy Of the findings. And hey, don't forget, BioCentury's Back to School. signature issue. It's our signature issue. It is out now. And for immediate access, you can go to BioCenturyBacktoSchool.com. Steve did a, Kind of an epic seven part, plus, podcast, re-imagining of, what FDA, should, could be. and uh, if you go to that, you can get access to BioCentury as well as, Steve's piece, edited by my colleagues, Selina Koch and Simone Fishburn, our editor in chief. we will be back in a moment.

Alanna Farro:

BioCentury This Week is brought to you by BioCentury Grand Rounds Europe. Grand Rounds, BioCentury's R&D conference, will make its European debut in 2025. Join us to debate key translational bottlenecks and unlock scientific breakthroughs with commercial potential. Connect with VCs, academic innovators, and biopharma R&D and BD leaders to explore cutting edge advancements in early stage R&D as we dive into disease biology, therapeutic modalities, and enabling technologies. Join us for the inaugural BioCentury Grand Grounds Europe in Cambridge. U.K. the September 17th to 19th. Register and learn more at BioCenturyGrandRoundsEurope.com.

Jeff Cranmer:

We're back. Stephen, you have been thinking about Insmed. tell us what you're finding.

Stephen Hansen:

Yeah. Thanks Jeff. Insmed is a, uh, what I would say is a maybe classic biotech story in that it's a, nearly 40 year going overnight success. maybe just a very, very quick history. You know, maybe this will sound familiar from a lot of other success stories that take a long time to, come. Um. So Insmed was founded as a diabetes company. and when that didn't work, it had, several reinventions of itself from being a growth factor company, to biosimilars. And, you know, a lot of this was, like in lots of biotech companies not successful. And so, you know, at the end of 2008, Insmed found itself, with a market cap of. 57 million and not a really clear path forward. And so this is where, I think that the story started to get interesting or, or not, depending on, on your perspective. So they did a very, very small deal where they brought in a, uh, drug inhalation platform and started, working on the lead program, which was basically an inhaled formulation of a 50 60-year-old antibiotic. And that's partly why I think a lot of people may be. have overlooked inmed for a while. I mean, if you look 10 years back, that was the lead program. They had a inhaled formulation of this antibiotic for an orphan setting that they were running through late stage trials. And this was at a time when, you know, everyone was focused on PD ones or Car Ts or Crisper. And so. Wasn't exactly the, uh, maybe the sexiest pipeline, around. And so I think that's maybe partly explains why they were, they were largely overlooked. but then, like lots of these companies, they came across an asset that they were able to find, you know, new opportunity for. So in 2016, they did a deal with AstraZeneca that brought in a, DPP1 inhibitor. Which had been developed for sort of pulmonary indications and they found a new one for it. A bronchiectasis ran a Phase II trial, was successful in the Phase II trial, and then moved into Phase III, and that's really where things started to take off for them. And that just read out last year. So they had positive Phase III data in bronchiectasis for two different doses. And basically it's been one of the hottest stocks of the past several years. almost every big specialist fund has an ownership stake in them, and they are now trading just above $30 billion. So it's, it's quite a remarkable sort of turnaround here from being a largely, unknown or maybe not often talked about company to now being. Basically one of the hottest biotechs, uh, in the sector.

Simone Fishburn:

So Stephen, I mean, as you say, classical biotech story. Of course, every company that's not doing well wants to have that end. It doesn't always work out that way. I think you earlier sort of referred to it as. Similar to the Horizon, story, and that that ended up being a takeout by Angen, of course, for 24 billion.

Stephen Hansen:

28 billion, I think was the takeout for Horizon and

Simone Fishburn:

Obviously, the question now is, does this make Insmed a prime takeout target? And maybe you can expand on the analogy with Horizon for the audience.

Stephen Hansen:

Sure. No, I think it's a good analogy and I think there's a good parallel here, potential because, so the reason I, I, I see similarities between Insmed and Horizon Pharma was because, Horizon Pharma, for all intents and purposes, was a, you know, fairly small specialty pharma, that. Also had unlicensed a largely sort of, you know, discarded drug that was targeting IGF, target that had long been sort of gone after and had of lost its luster per se. But they found a new place in, thyroid eye disease where you could use it, and it not only worked in the clinic, but proved to be a much larger and more lucrative commercial opportunity. Than anybody had, really anticipated and that sort of shot Horizon up to, to being this sort of large cap company that, as you say was eventually taken up by Amgen. Insmed is is similar in that they also acquired an asset, as I said, and has now, you know what, they are projecting to be potentially a $5 billion peak sales in bronchiectasis alone. an indication that, doesn't have any approved drugs. They're also looking at expansion opportunities into chronic rhinosinusitis without nasal polyps. And Simone, one of your favorite indications indications hidradenitis

Simone Fishburn:

It is so fluent in that.

Stephen Hansen:

And, um, you know, looking at a sort of pipeline in, in a product just there. But then on top of that, they are getting some other assets out of their inhaled, platform that they had acquired in 2010. They also have an inhaled formulation of treprostinil, that just had positive Phase II B data in, uh, pulmonary arterial hypertension. So you kind of lined up, they have one blockbuster now coming that just got approved, for bronchiectasis They have another one that's going into Phase III that they think has 2 billion potential. So, at a $30 billion valuation, they kind of look like they might be maybe two big be acquired at this with, with multiple drivers.

Simone Fishburn:

I mean, they'd be very expensive to buy What you are saying is that the value isn't exclusively in the lead asset. I mean, maybe predominantly, and of course, and Jeff can give you the the URL of course, but this really does relate to our back to school essay of two years ago where we talked about the tier jumpers. And this baby jumped from the under $5 billion tier to the 20 plus billion dollars tier. And you know, who knows? Maybe marching to that 50 plus, who knows?

Stephen Hansen:

It's moving quick, but it's, I, I, I, I, I, want to go back to the Horizon parallel as well, because I think that's also a potential road that this goes. You can foresee a scenario in which, and something maybe that that Insmed obviously needs to maybe, protect against, because the reason Horizon ended up getting acquired is because they essentially got a little bit too far ahead of themselves in terms of their guidance for the growth that they were gonna see from Tepezza there, that lead assets and effectively. guided for too quick a growth. That growth didn't show up and so they had to pull back the stock then pulled back and it reached a point to where it basically became, an acquisition target. And Amgen was able to take it out, as you said, for 28 billion. And you could foresee a scenario in which something similar could happen if in Insmed is too aggressive with their guidance on the growth for Brinsupri. And so I think that's something they need to, um, you know. Be wary of, you know, take the long, you know, good advice of, you know, under, under promise and over deliver rather than being too aggressive here. But, 5 billion peak sales for one indication here

Simone Fishburn:

I mean,

Stephen Hansen:

feels pretty aggressive to me. we'll see where this goes.

Simone Fishburn:

but we do know, I mean, with all the conversation. About pharmas needing late stage assets and the fact that there aren't that many of them, what's 10 billion here? Are they,

Stephen Hansen:

billion thing, 30 is another. This

Simone Fishburn:

they, yeah. Well, I meant in of DI meant in terms of differential, but I mean, let's just acknowledge one more thing. It's also a different regulatory environment, so, in that takeout there was a sort of. FTC that made it take forever, and I think that there was some kind of oversight of that, whereas I think now it's considered a more friendly environment for takeouts.

Stephen Hansen:

True. Yeah. No, I think you're right. But it's, again, I, I, I just think it's been really interesting diving into the, diving into the past of Insmed and, and sort of. Looking at this, success story and, you know, something that, is one of these success stories that we should celebrate from our sector. This is one of the areas where, you know, it's, it's fun to see a, see a company, uh, get a win like this.

Simone Fishburn:

And a disease that's had nothing get the breakthrough.

Stephen Hansen:

That's right.

Jeff Cranmer:

All right. Thank you for that. Stephen. Stephen, We'll have a story out, early this week, on Insmed. And, as Simone mentioned, our Back to School, uh, two years ago focused on tear jumpers, building the road to sustainable biotechs. if you'd like to check that out, drop me a line. and, uh, we can set you up with a free trial and it's a great story. We look at 17 growth companies, and Simone, Stephen and Lauren Martz, wrote that piece. it's quite interesting looking at companies like Insmed as they, jump up the ranks. When Stephen told me he was working on the Insmed story, I was, shocked that they were all of a sudden, uh, you know, like, your kids, like all of a sudden they've. They grow up so fast. Steve, uh, quickly before we go, signs of life from Senator Cassidy. what's going on?

Steve Usdin:

Senator Cassidy wrote a letter to. Robert F. Kennedy Jr. The HHS Secretary last week, calling on him to publicly endorse the pertussis vaccine. Actually a combination vaccine that includes, pertussis antigen. Cassidy noted that there's, an outbreak of pertussis in his state, Louisiana, that's resulted in deaths and hospitalizations and, that it's growing in other states in the United States. It will be interesting to see what, Kennedy's response to this is for years, in his life. Before he was at HHS, he cast doubts about, the pertussis vaccine, about its use in the United States and internationally. he canceled or postponed, funding for Gavi, the organization that's it, is the largest funder of, vaccination in developing countries around the world. Since becoming HHS secretary, and he used concerns about a different pertussis vaccine as a justification for pausing funding, for Gavi internationally. it will be very, very interesting and I think, important. both from a public health standpoint and politically to see, how, Kennedy responds responds to this call from Cassidy. and since talking about vaccines and Cassidy, I think, you know, the other thing that, to to point out that's really important of course, is that, um, CDC's ACIP which has been reconfigured with appointees from. Secretary Kennedy will be meeting this week, Thursday and Friday. There's gonna be a great deal of attention obviously on, what they do. Senator Cassidy actually called for, the meeting to be postponed. Um, that doesn't seem to be in the cards, so, it's quite possible we're gonna see. A bigger showdown between, Kennedy and Cassidy over vaccines and public health issues going forward.

Jeff Cranmer:

All right. Thanks for that Steve, Steve story, along with the others that we spoke about, available at BioCentury. Dot com. thanks for tuning in. If you've liked what you've heard, subscribe, give us a thumbs up. drop us a note. we'd love to hear from you. And a special thanks to Kendall Square Orchestra. Which provides the music for BioCentury this week. the group is now, in its eighth season, gearing up for its kickoff concert tickets on sale. The group connects science and technology professionals and other members of the Greater Boston community to collaborate, innovate, and inspire through music, while supporting causes related to healthcare and education.

Eric Pierce:

BioCentury would like to thank IQVIA Biotech for supporting the BioCentury This Week podcast. Learn why IQVIA Biotech is proud to be the go-to CRO supporting biotechs from innovation to patient impact. Visit IQVIABiotech.com.

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