Ep. 341 - BioCentury's '25-'26 Picks. Plus: BioMarin & Biotech ICYMI

This past year was an inflection point for biotech: The markets woke up, M&A and China had strong showings, drug development offered key trends, and new leadership at FDA shuffled the regulatory deck. On the latest BioCentury This Week podcast, BioCentury’s analysts make their picks for the biggest biopharma readouts, deals and regulatory and policy moves of 2025 and what they are forecasting for the year ahead.
The analysts also recap biotech highlights of the past two weeks, including the $4.8 billion takeout of Amicus Therapeutics by BioMarin Pharmaceutical.

View full story: https://www.biocentury.com/article/657933

#BiotechOutlook2026 #BiopharmaTrends #BiotechMA #FDALeadership #ChinaBiotech

00:00 - Introduction 
02:31 - BioMarin's $4.8B Amicus Deal
06:07 - Year-end Biotech Highlights
16:37 - Analyst Picks and Predictions
30:01 - Policy and Regulatory Landscape

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

Transcript

0:00

[AI-generated transcript.]

Jeff Cranmer: 0:02

Seeking a continuous run of deals BioMarin has added rare disease drug maker Amicus via a $4.8 billion takeout, we'll have BioCentury news editor Paul Bonanos' takeaways from his conversation with BioMarin dealmaker in chief James Sabry on the latest BioCentury This Week podcast. And we'll catch you up on other highlights from the biopharma world from the past two weeks in case you were off on a beach or wandering around Argentina or simply throwing a lovely Hanukkah or Christmas party. We've seen MFN deals galore, we've had Sanofi by Dynavax, approvals, and a Phase III miss for Mereo and Ultragenyx among them. Plus was 2025 and inflection point for biotech BioCentury's analysts took a look back at a year when the markets picked up, translation delivered some turning points, and FDA delivered some challenges we set out to predict what we think will make news in 2026. I'm Jeff Cranmer and joining me today on the BioCentury This Week podcast are my colleagues.

Simone Fishburn: 1:31

Simon Fishburn, Editor in Chief.

Steve Usdin: 1:34

Steve Usdin, Washington Editor.

Paul Bonanos: 1:36

Paul Bonanos, Director of Biopharma Intelligence.

Jeff Cranmer: 1:39

Okay. JPM 2026 is right around the corner, and I can tell you this last week it rained every day out here in the Bay Area winds up to 80 miles per hour, and the past two days have been beautifully sunny, so we'll roll the dice in a couple weeks and see what weather we can provide, but what. I do know we will have and it's out now is BioCentury's JPM Guide. You can head to jpmguide.com to browse the lineup of sideline events and receptions planned across San Francisco. And if you're hosting something, you can use the online form to make sure your event appears in our guide, again, that's jpmguide.com to make the most out of your JPM week. Well, Paul, it, seems like a little while ago, but this was one of the bigger deals of the year, uh, BioMarin buying Amicus, uh, a long time rare disease company. BioMarin obviously has been, uh, revamping the way it's doing things. Adding, James and, and a new CEO, uh, in Alex Hardy out of Genentech. And, uh, they're starting to do some deals. What did you learn?

Paul Bonanos: 2:57

Yeah, two older companies coming together, BioMarin around since the 90's. Amicus established in 2002. Obviously with a long history of, um, John Crowley and, Pompe disease in his family. And now they have two drugs, uh, it's actually three drugs technically, but uh, one for, uh, Fabry and one for Pompe disease and BioMarin gets the whole thing. They're buying them for$4.8 billion as you said. And I did speak with James Sabry at BioMarin. For background, James joined BioMarin last year after a 14 year run leading partnering for Roche and Genentech. and he joined with the intention of making deals that will spur growth at BioMarin. We spoke with him about that, uh, more than once. They're kind of an interesting company as far as BD goes, you know, they market eight drugs already. It's about an $11 or $12 billion company in terms of market cap, uh, right now. And none of the drugs are true blockbusters, but two of them are fairly close, VOXZOGO for, uh, MPS IVA. That figures to approach $900 million in sales this year. VIMIZIM for achondroplasia isn't too far behind, they're rare disease drugs. Most of what BioMarin has are enzyme replacement therapies, right. And the way they're positioned, they have a bunch of revenue generating products and consistent cash flow. It's not as big as a big pharma, but it's still positioned to be a buyer, and the way James said it, and you've said it in your intro, the Dealmaking process there, he does not expect it to be episodic. It will be continual. So they will keep doing deals, whether for marketed products or for pipeline programs, uh, stretching back to preclinical ones even. He framed it, uh, in our conversation as BD being a way of life for that company. They will need to, uh, be strategy first around BD, with implications for R&D and for commercial. And that's how the company will sustain itself. So, um, marketed drugs and pipeline, they get both with this deal. The Amicus, acquisition gives it, uh, I said two drugs in one. That's the Pompe program, Pombiliti-Opfolda. And then Galafold for Fabry disease is the bigger one, or at least right now, bigger selling one. So those have been on the market and will be for some time, so more cash flow from that. There's also a kidney disease program, DMX-200 for FSGS very long name that I'll use the abbreviation for our convenience, Amicus had in licensed it from an Australian company this year called, uh, it's the company is Dimerix. Um, and they expect Phase III results next year. So, like I say, it's a continual process. It's the second deal since James joined last year, they acquired Inozyme in May. Got an enzyme replacement therapy for ENPP1 Deficiency. Another abbreviation I'll stick with. That one's in Phase III data are due next year, price tag for that deal was much lower, $270 million. we can say that Amicus is on the bigger side. I believe James referred to it as an anchor for what's to come. But we can expect a run of more deals, um, maybe closer in size to Inozyme compared with Amicus going forward.

Simone Fishburn: 6:03

And just a quick note there, Paul, when you say a run of more deals. And we'll talk a little bit about trends, but we are gonna see more deals where the acquirer is a sort of medium, large size biotech as opposed to a pharma. We're gonna talk a little bit about M&A. We tend to think about, about M&A being really all the pharma story and their loss of exclusivity, but really it's a much richer picture than that. And this is one example of that.

Paul Bonanos: 6:31

yeah, some other companies in that tier, maybe like Insight, Ionis, companies like that could be buyers as well. But yes, without the massive patent cliffs that we'll see from drugs like Keytruda, in BioMarin's case, they're more of a mid-size. Their products aren't huge blockbusters, but they have their lifespan and they will need to replenish.

Steve Usdin: 6:53

I, I would look at it from another point of view also, which is what it means for patients. And I think the companies the size of BioMarin, are more likely to pursue products that are for smaller populations and that may be less profitable, um, than some of the products that big pharmas need to move the needle. that could be a really good thing for patients, especially patients with rare diseases who need products. And they not necessarily gonna be products that are gonna be blockbusters or have blockbuster potential, but they're blockbusters in terms of their potential for changing people's lives. And Amicus is a really good example of that. You know, John Crowley was the founder of the company. obviously his, his, he had his personal stake in it because of the rare disease that his, kids had. And he stuck with it with an amazing tenacity. And the company, I've, I've spoken with him about it several times. The company had near death experiences, many times, and the products that it, it had brought to market have been transformative for the lives of patients. So I think, it's a trend that's gonna be really important and really interesting to follow. Um,

Simone Fishburn: 7:57

Steve, I wanna.

Steve Usdin: 7:58

because it opens up this window. Yeah.

Simone Fishburn: 8:01

Yeah. Sorry, Steve. Yeah. I wanted to add to this because you know, John Crowley's story and that journey is an exceptional one, but actually what you're saying speaks even broader than that because it's not just whether the large pharmas will follow rare diseases that they will in some cases. But all the evidence suggests that smaller companies are better at communicating with those patient populations, with those patient communities, they create ties. In this case, as you said, John Crowley sort of had a direct tie, personal tie to the patient community. But we do hear a lot that smaller companies are nimbler and able to, to create the ties that you really need for these successful programs. And so I agree with you, I think we'll see more and more success in that kind of arena from there.

Steve Usdin: 8:47

And the other thing though that's also interesting in Amicus, is an example of this it's still, I think up in the air for the rare disease space, how much companies are gonna be willing to invest in, in gene therapies, in particularly in the United States now, the regulatory environment has turned really sour, for gene therapies. Amicus tried to get into gene therapies and invested quite a bit in some gene therapy programs and then abandoned it in, uh, 2022 or thereabouts. So I think that's another thing that's kind of telling about the Amicus journey and BioMarin.

Jeff Cranmer: 9:21

Okay, well, BioCentury was off last week and, uh, today I got to my desk, uh, dusted it off a bit and, uh, opened our morning news budget. And I could tell that, uh, Paul had gotten up quite early because, uh, biotech certainly did not go to sleep over the past week and change. Uh, Paul, what are some other highlights that, you caught up with this morning?

Paul Bonanos: 9:47

Yeah, a couple of deals before Christmas. Sanofi buying, uh, Dynavax, for$2.2 billion, they gain, um, an HBV vaccine that is on the market, as well as a candidate, uh, that would compete with Shingrix in the shingles. Let's see, also, um, Shionogi buying an ALS drug from Tanabe for $2.5 billion, that's RADICAVA approved for about a decade. Ipsen reaching into, uh, China to do a deal with Simcere. For an ADC targeting LRRC15$45 million upfront, more than a billion dollars in total deal value. So yeah, a few deals there. There's also a pretty substantial market moving, um, clinical failure setrusumab targeting sclerostin from Ultragenyx and Mereo. The two partners both lost value, after it missed the primary in two Phase III studies to treat osteogenesis imperfecta. When I looked, Ultragenyx had lost about 42% of its value, and Mereo had lost about 90. So unfortunate there for patients. Also Biohaven had a potassium channel program that missed its primary endpoint in a Phase II proof of concept study, in major depressive disorder. They won't do any more psychiatric trials of it, although I think it's still alive, in epilepsy,

Jeff Cranmer: 11:12

And, Uh, of course we've had a few approvals, uh, in the past couple of weeks. Uh, I'm sure most folks heard that, uh, Novo Nordisk got its oral version of Wegovy approved via the Commissioner's National Priority Voucher Program. We saw Abbisko and Merck, German Merck get an approval in China of one of their therapies. And, a hats off to, uh, Cytokinetics who got a, recommendation out of EMA's CHMP approval from China's. NMPA and approval, from FDA for its first ever drug in the U.S., that's aficamten. I got to speak with Robert Blum, couple weeks ago, so I'll have a story on that in the coming days. And that's a company with ties to James Savory as well, and, uh, he was the og, CEO of that company. Any other highlights that you wanted to touch on Paul?

Paul Bonanos: 12:16

Well, there was, uh, I think two Fridays ago, late, there was an IPO filing Aktis Oncology radiopharma company,

Simone Fishburn: 12:23

What is this word? Paul, what does this word mean that

Paul Bonanos: 12:26

never heard of it. It's, it's, uh, for, for our younger listeners, um, there used to be a time when, um, companies typically

Simone Fishburn: 12:35

old enough to remember.

Paul Bonanos: 12:37

were able to go public on nasdaq. Uh, all, all they had to do was file some paperwork. Um. I'm kidding, of course. But, um, yeah, it's been a very slow season and, uh, it's possible that IPOs will make a comeback in 2026 among biotechs. Uh, there's some cautious optimism around that, where, um, some companies will be able to get liquidity that way and trade on the open market, um, instead of hewing more closely to the M&A exit pattern, which has picked up so much lately. Maybe that's, uh, maybe these things are connected.

Jeff Cranmer: 13:10

And Steve, in the, uh, policy world, uh, there were quite a few Most Favored Nation deals. Any quick thoughts there?

Steve Usdin: 13:19

so, the Administration sent out letters to 17 companies, requesting that they come up with voluntary agreements on what the Administration is calling, uh, Most Favored Nation Drug Pricing. With the deals that were recently announced, all but three companies, have announced deals. The companies that haven't announced them yet are AbbVie, Johnson & Johnson and Regeneron. I know that, that a deal has been done with Regeneron and with Johnson Johnson. I suspect one has also been done with AbbVie. So, and I think that the administration is pushing, they want to have all the deals announced. By the end of the year. So I wouldn't be surprised if we get another few announcements this week. If not, I think we'll see them early next year.

Jeff Cranmer: 14:06

That's a tasty wrap up there, obviously a lot more happening. And we will have our finance report this week, a deals report. So, you'll be able to go on to BioCentury.com to, check out the tables that we put out along with Paul's, words and, catch up quickly on what has happened. Also, you might have missed on our sister podcast, The BioCentury Show. Simone sat down with one of those, you know, sort of one name people in biotech, Stelios, Simone. Uh, how was the conversation with Stelios?

Simone Fishburn: 14:43

You know, to say it, it was charming, would really undersell it, Stelios is charming. And he came fully togged in a tie and a suit just raising the whole level of BioCentury's, uh, podcast, I gotta tell you, looking at these t-shirts around the screen here. Um, but no, I mean, Stelios really, it's very interesting because he has a long view. He's been in biotech a long time. But he always brings the long view to look at what is happening right now and to look at where he thinks is going. So this is not sort of wallowing in the past, but there is a sort of reality check of, you know what, this is not really a bear market in historical terms this is not a bear market, you know. Anyway, I encourage people to listen. Stelios really is an interesting thinker. I love the conversation. Really free flowing and, and a lot of thoughts from him. So, yeah. Check it out on Jeff

Jeff Cranmer: 15:39

The BioCentury.com.

Simone Fishburn: 15:41

Dot.

Jeff Cranmer: 15:43

Yeah. Oh, right. Dot com. Sorry. Yeah. Um, of course, yeah, a little, uh, groggy from the, the turkey and prime rib and, uh, oodles and oodles of red wine over the past few days. But yes, if you're newer to biotech, and by newer I mean people like me, uh, who have been in it only 20 years or so, Stelios is a name to know. He's been there since the very beginning. He was the former chair of Biogen. He is the current chair of Exelixis, nearly two decades as an investment banker. And he really, has been one of the long-term leading voices in biopharma it's just, just a person to check out and, uh, listen to what he's got to say. He also has some interesting, points for, budding entrepreneurs or current entrepreneurs on how to build a company, which, he likens to climbing Everest. And so check that out. All right. Our analyst picks and predictions are in. And so I'm just gonna turn it over to Simone here. I know she's been, uh, fine tuning the story, which we, are releasing on Monday, that's Monday, December 29th, as we're all a little unstuck from time right now. Simone, what stood out most to you?

Simone Fishburn: 16:58

Thank you Jeff. So a quick word about the process. the process. is fairly unbiased. Effectively, we ask everybody on the team to send in a couple of paragraphs of what stood out to them this year, good, bad, other, and what they think about next year. It's fairly uncoordinated. We then go through it and, and organize it into themes and so on. And really, as I looked through this, the theme that just kept popping up to me. Was this question of like whether 2025 is going to turn out to have been an inflection point. And in fact, I don't even mean this specifically from a policy and regulation point of view, which we're going to come to last because, it's probably the biggest thing this year. But otherwise, we'll spend hours and hours and Steve will just bring us back to earth with a thud. So I'm gonna start, in fact, um, and you know, ask Paul, because Paul wrote a little bit about M&A being the safe haven. I want to preface this by saying. 2025 was really not been a very good year. Not a good year for biotech. A lot of overhangs little money, as said, regulatory risk. Policy upheaval and just generally, people have not had a good year in terms of their mood, but it is ending in a slightly better place. And as Stelios talked about, the numbers actually pretty good with follow-ons. The stock market is pretty good. And Paul, I want you to talk us a little bit about what you, you know, what stood out to you for M&A.

Paul Bonanos: 18:29

Well, yeah. You mentioned follow ons there, there was a historically strong day, uh, just a couple of weeks ago with, was it eight companies raising something like $4 billion in a day?

Simone Fishburn: 18:38

Something like that. who's gonna quibble but a billion dollars these days. Yeah.

Paul Bonanos: 18:43

M&A was pretty strong, mostly in the back half of the year. I know there was a big deal at the beginning where J&J bought, uh, Intra-Cellular the neuro company. But, um, there were a lot of, a lot of very substantial deals in the 10 billion range this year. Avidity acquired by Novartis, Merck acquiring Verona, Pfizer buying Metsera, the hot obesity space, Sanofi and Blueprint, Merck paying top dollar for an antiviral program from Cidara. So, yeah, there was one that caught my eye not too long ago. Also J&J buying that company Halda, that was, uh, in Phase I/II very early, $3 billion plus. And the biggest deal of its kind for a company that Phase. There had been a couple of deals for in vivo CAR T therapies as well that were also very early stage. And it got me wondering, they say three is a trend, right? So, whether buyers were gonna go earlier, or at least, be willing to spend big on earlier stage programs. We'd been in this pattern for such a long time, of, buyers focusing on late stage in marketed products that had been de-risked. But there was a time again for you younger listeners, uh, there was a time when biopharmas would take a gamble on things that were earlier or didn't have proof of concept data yet, or did platform deals. And if the market has bottomed out, we've obviously seen some acceleration in the XBI in the past six months. If the market has bottomed out and there's a perception that sale prices are over, then maybe you will start to see some, uh, some higher risk deals or risk some appetite for risk, uh, going up.

Simone Fishburn: 20:22

Yeah, and you know I'm not gonna steal the thunder from our colleague Stephen Hansen, who will be coming out with a financial preview package for 2026 very soon. Although I can tell you, you know what the bear market is over is what he's saying. So you heard it here first or not. I think you know what you're talking about, Paul, listening to podcasts and reading commentary on the broader markets. There's certainly some optimism. I think what we've seen in biotech is a certain amount of shrugging off of the tariffs and so on. We haven't yet seen the IPO market warm up, but we have, Jeff and you are gonna talk a little bit about this. I think that one of the trends that we're going to see next year is really moving to, to other markets. So we started that this year, Jeff, you know, going back to the theme of turning points or inflection, so talk a little bit about China and what you were seeing.

Jeff Cranmer: 21:21

This year, I mean, we're talking about JP Morgan. Uh, I, I feel like JP Morgan 2025 was it was sort of this moment when western biotechs, western investors really woke up to the reality of competition from China. We saw a flurry of so-called NewCo deals, at the beginning of the year timed with JPM, and near daily announcements since, have shown how deal sizes, types of deals, deal terms are, are just rapidly evolving. In part that reflects China biotech's ability to move really quickly into hot areas of innovation. and that's really grounded in prowess, in navigating new modalities, which is something, Simone, you've talked a lot about this year. Some of the deals, that really reflect this, uh, earlier deals, strategic collaborations, Harbour BioMed's partnership with AstraZeneca, on multi specific antibodies, for immunology and oncology. A bigger deal that that followed quickly after that was GSK's tie up with Jiangsu Hengrui to co-develop up to 12 drugs for $500 million up front. So really deals going beyond a single asset or building a company around a single asset. So activity hasn't been limited to China. Starting to see a lot of things happening in South Korea. We're seeing VCs such as Angelini Ventures, opening their first Asia offices, a Flagship named a special advisor in South Korea, BG Rhee. And we're starting to see a lot of Western VCs, take a fresh look at Japan and figure out ways to build companies that sort of solve for some of the hurdles, uh, that have held back Japanese biotech. Blending Western management teams with Western money, incentives from the Japanese government and talent and innovation out of Japan's academic powerhouses.

Simone Fishburn: 23:34

I think that's a, interesting point there because I think a lot of people will be like, Japan really? I've been hearing this for so long. But the reality also, you know, you talk about China, many times this year have I seen western biotechs, sort of, this is why we use the word waking up. Sort of thinking, you know, China is the same as it was five years ago or 10 years ago or whatever. Not really understanding. And just this year starting to realize that there's a very real talent pool and opportunities from China that are a very significant competitive. You could call 'em a competitive threat. They're also a collaborative opportunity. And so we are, as you pointed out, Jeff, looking at what are the other places in the world and starting to see that in other places in Asia as well. Sort of the idea like don't be late to that story. Right. So I wanna just move a little bit onto some of the science standouts.'Cause this was also one of the reasons why there was this sort of inflection point theme coming through it. My colleague Lauren Martz talked about turning points in cancer. You know, um, cell therapy, it's not been a good year for cell therapy. It's been a bad year for cell and gene therapies, but you know, the rise of in vivo CAR T, could that solve the access problems that have held it back? Um, you know, you mentioned EsoBiotec biotech, a couple of deals there, in multiple myeloma. So seeing, seeing evidence of in vivo CAR Ts. But there are also academic investigators showing the evidence of efficacy in patients with autoimmune diseases, and that just shows how fast things are moving now. And so we'll find out actually within a year or two, I expect. as to whether, you know, whether the, the in vivo CAR T dream also, fades away or, or, or whether that's, that's a real opportunity. But Lauren also talked about a, a few other turning points in cancer immunotherapy, for example, where she talked about PD1 blockers moving from late, late line use to earliest treatment settings, even organ sparing applications. So some of these from GSK for jump early, um, being alternatives to surgery in certain specific cancers. And she sort of lists a few others. You know just going back to China, I think one of the other things we've seen is this idea of rapid path to the clinic. Again, something that happened through the in vivo CAR T story and we're seeing other countries now really want to improve, including the U.S. in fact, to expedite that path from idea or discovery to human testing. And, you know, maybe, maybe we're starting to solve that translational bottleneck. Jeff, gimme a couple more minutes to call out some other, um, on, on the science front because I think these are really interesting.

Jeff Cranmer: 26:26

please do, I, one thing, just, uh, I know Danielle Golovin, touched on some highlights in women's health, which was great to see.

Simone Fishburn: 26:34

I think that's really important. I wanted to, she, she sort of talked about some wins in research, but also an approval in fibromyalgia and that is a pain condition that predominantly affects women. And that was the first new fibromyalgia drug in over 15 years from Tonix Pharmaceutical.

Jeff Cranmer: 26:54

Well, our great colleague Selina is, uh, off on walkabout, but I know she has been looking into the neuro space, was there anything that caught your eye there?

Simone Fishburn: 27:03

Absolutely. So there's some data from, uh, multiple sclerosis, but I think one of the important things about this is that the data's really bolstering confidence of neuroinflammation being attractable target in neurological diseases. We had orexin 2 programs also. With like a whole new class of treatments for narcolepsy. And an important one, I don't know if Steve is gonna weigh in on this in a minute, is a breakthrough in Huntington's disease gene therapy data from uniQure. And so that was a really huge thing in terms of data. The enthusiasm was tempered because FDA reversed an earlier decision. Where they'd accepted an actual history comparator. So now we are kind of entering the, um, regulatory risk domain. But before we go there, gimme a couple more minutes. Not a good year for Alzheimer's disease. Um, set back with Semaglutide. But Tierney Baum, one of our, our new analysts sort of really talked about drugging the undruggable and turning a corner on KRAS G12C mutations. Over 20 new programs entering the clinic in last year alone. So 20 in the last year, right. And so that sort of largely by targeted protein degradation. And our other newest, member of the group, Lindsay Martin, talked about an important thing with Ai. We talk a lot about Ai, we talk a lot about the molecules. But actually what she, really shone a light on was solving one of the biggest roadblocks to AI in discovery, which is the lack of high quality large training data sets. And so she highlighted the launches of various drug discovery, federated learning platforms. And these protect users raw data and they collectively train the models for all the participants. And that's a really important, and I think probably somewhat undersung, development. So a lot of these things really feel like either the seeds of turning points or actual turning points.

Jeff Cranmer: 29:03

Just quickly before we jump to Steve, You mentioned Alzheimer's and it not being a great year. Selina is looking ahead to, a few things in 2026, she says, uh, Biogen may have the most consequential tau data yet, that is coming from its antisense therapy, BIIB080. She's also looking ahead to two clinical readouts and symptomatic Alzheimer's treatments. And another thing that she's been looking at quite closely in neuro, and these are some cool pieces to go back and dig out of our archives, blood brain barrier shuttles. she says, 2026 could bring the first FDA decision. On a protein therapy delivered to the CNS via one of these BBB shuttles. So that's, uh, coming out of Denali. Well, I think the time has come. Simone, what do you think? Should we, uh,

Simone Fishburn: 30:01

we let Steve loose on this podcast to tell us about what 2025 was for him as he sat in his corner of Washington DC not taking me for falafel. Go ahead, Steve.

Steve Usdin: 30:13

Okay, well, maybe we can do the falafel over the holidays. Um, yeah, so look,

Jeff Cranmer: 30:18

Don't, don't sleep on Ben's Chili Bowl, Simone. He, he, he knows his way around that beloved institution.

Steve Usdin: 30:25

so the, there's no other, there's no nicer way to put it. Look, the pillars of US biomedical innovation, were seriously eroded in 2025. I think in 2026 we could see some of the impacts of that. NIH research has been severely disrupted. the SBIR program, was allowed to lapse. immigration policies have constricted a critical source of talent across the life sciences. You know, overarching all of it, FDA staff has been demoralized and politicized. Staff with thousands of years of experience have walked out the door, that isn't going to be easy, to replace. And it, even if there was a will to do it, it wouldn't happen quickly. Vaccine regulation has been handed to ideologues who oppose science based public health. One of the other things we just talked about, the MFN deals. Seemed to have taken some of the risk off the table. The impacts for the individual companies in the short term are less than was feared in 2026. We'll get a sense of the larger impacts and how real the so-called MFN pricing, turns out to be as well as the impacts of some of the, models that CMS has created, for MFN pricing for Medicare Part B and Part D. But I think that the most important thing that we're gonna see in 2026 in public policy are gonna be the consequences of FDA's, leadership and ideologically driven decision making. Regulatory uncertainty it's turning the US into a no-fly zone for investment in gene therapies and vaccines. I think that the issue at FDA is deeper than inconsistency. It's a, it's basically, it's a repudiation of science-based regulation. The Commissioner's National Priority Voucher Program epitomizes this. They start with the answer, and then they search for data to support it. And they're winners and losers clearly. The program rewards specific companies, it punishes others. And investors and patients are gonna be thrilled by some of the results when they get the results that they want. The question is, what impact this has on the integrity of the system as a whole. A couple of things, really, quickly, because I could go on all day about this and I will go on all day about it, in the new year when I've got big stories a, about all these things. What are some of the things that were put into, motion in, 2025 that will learn in 2026 how they may be implemented. One is the plausible mechanism pathway. So far, it's an article in The New England Journal of Medicine and nothing more. Presumably there'll be some guidance and there'll be some policy making around it in the new year. The idea that a single pivotal trial will be the default standard, for demonstrating efficacy going forward, it's something that Commissioner Makary has prioritized. He drafted a press release about this. HHS has not approved it. It hasn't been put out yet. But he did choose to leak it to one publication, which has described it, and now it's kind of out there dangling. We don't know what's gonna happen. Expert panels have more or less replaced advisory committees as the, source of independent outside expertise and advice for FDA. Clinical trials diversity mandates. Remember that at the beginning of the Trump Administration, there was a lot of talk and a lot of action to eliminate DEI across government. It still isn't completely clear how that's gonna affect FDA's, uh, mandates for diversity in clinical trials. America first policies, the Commissioner's Priority voucher Program has explicit America First policies in it. The PDUFA reauthorization negotiations include America First Policies. And then there's specific drugs that, um, are high on the make America healthy again, agenda, either for positive or negative, impacts. Commissioner Makary is an enormous, advocate for hormone replacement therapy. We've seen changes in the labeling already. I think we'll see more, action to promote hormone replacement therapy in the new year. There's autism drug labeling, um, and indication changes as a result of Make America Healthy Again policymaking from the HHS secretary.

Simone Fishburn: 34:40

thank you Steve for bringing us back to Earth on that. Um, but give us just a, a big picture overview because I feel what we're really looking at for 2026 with regulation, but also a lot of these other things I've talked about 2025 being an inflection point. I think we're gonna find out in 2026 really the consequences of many of these things. So, so give us your big picture.

Steve Usdin: 35:06

Big picture. I'm gonna go out on a limb and make some very specific predictions. This time next year I'll either, um, look really good or look, uh, an idiot. I don't think that HHS Secretary Kennedy will be HHS Secretary a year from now. I think that he'll be out of there shortly after the midterm elections will be my prediction. I would be surprised if Marty Makary is still the FDA Commissioner a year from now. And also I'll be surprised if the se if CBER Director, Vinay Prasad, and CDER Director, Tracy Beth Høeg are in their jobs a year from now. My guess is that they will all be gone, by this time next year or, or shortly thereafter.

Simone Fishburn: 35:49

Well, certainly if that's the case, um, it'll be interesting to see what does unfold after that.

Jeff Cranmer: 35:55

Steve, some bold predictions, but, uh, that's, that's what we love to get from you. You can check out all of our teams, 2025 picks and 2026 predictions, uh, at bioCentury.com BioCenturypodcast.com. We'd love for you to, like and subscribe to our podcasts. And we're really grateful to all of our listeners, who have made this podcast the success that it is as we wrap up our sixth season really helping us do well on the charts, that keeps us, uh, wanting to come back for more. And giving you these, uh, insights and, little tidbits from the world of, uh, falafel in Washington, and elsewhere. As we wrap up the year here at BioCentury, uh, we will also have the best of BioCentury, Simone's picks for the top stories we ran this year. As always, looking ahead in the new year, we'll have Steve's take on the policy environment, how it's shifted, where it's going. We'll have Stephen Hansen with his 2026 Public Markets Preview. We'll have Lauren Martz and Selina Koch leading us on what they believe are the key catalysts of the year. We'll have Danielle Golovin, give, her, 2026, addition of the prior year's Series A companies and the technologies they brought forth. And finally, we'll also have Lauren's, uh, look at the year in regulatory approval. We wish everyone a very happy New Year, and we look forward to seeing, uh, many of you at, JP Morgan this year or at other places around the globe where BioCentury is hosting its conferences. Uh, so from Simone, Steve, Paul, and the rest of the BioCentury team, we wish you all a very happy New Year and looking forward to many great biotech stories in the year ahead. Kendall Square Orchestra. we are also very grateful to that group for providing all of the music for our podcast