Latest / BioSpace / FDA’s absent leaders, the millions pharma CEOs make, and pancreatic cancer’s momentum
Transcript
- Jef Akst: Hello, you all may have noticed right off the top that I am not your usual host. We are making a little bit of a change here at Biospace. Heather has run The Weekly to great heights for the last two years, but she has officially passed the baton onto me. And the other big change we're making is we are welcoming our newest editor to the group, ⁓ Gabrielle Mason. Thank you so much for joining The Weekly. The Weekly 5.12: Yes, thanks so much, Jeff. I'm so happy to be here. Jef Akst: So Gabby joined our team about a month ago at this point and she's hit the ground running. You'll see her byline. She's in all of our newsletters. She's putting together Jean Poole now as well as manufacturing brief. Gabby, just as a way of introduction to our audience, why don't you tell us just a little bit about your background? The Weekly 5.12: Yeah, so ⁓ from Fierce Biotech where I was a senior editor. So I'm not new to biopharma I love this space. ⁓ Before then, worked at Becker's Healthcare where I reported on the pandemic and I have degree in criminal justice ⁓ and creative writing, is interesting. ⁓ So very passionate social reform, patient safety. And in my free I love to Annalee: You The Weekly 5.12: paint, be outside, anything with the arts really, except for movies. My movie education I am working on, ⁓ Annalee knows and you guys are starting to learn, ⁓ lot of movie references go over my head. But yeah, that's a little bit about me. Jef Akst: criminal justice is an interesting background. I hope I don't ever need that personally, but I bet it'll come in handy on occasion with our coverage. Heather: Thank Gabby, I didn't know that you paint. That's cool. What kind, like or landscapes or what do you paint? Jef Akst: we launch the video. ⁓ The Weekly 5.12: I started out with paint by numbers and it has evolved into, yeah, like just kind of abstract. I'm not good at very like intricate, you know, like details. People can't do that. Heather: I know, I've just recently gotten back into adult coloring books. So maybe I can evolve from that. The Weekly 5.12: Yes. Jef Akst: I like both of those things. The adult coloring books are so fun. When my daughter wants me to color with her, I'm like, sure, get me my coloring book, I'll do that. And then I actually did a Paint My Numbers one time, a very intricate, very detailed lion, and it hangs in my closet to this day. I love looking at that and remembering that at one point I could do painting, but no, it's generally not for me. The Weekly 5.12: it's. Heather: Bye. Hmm. The Weekly 5.12: It's very cathartic and I like to listen to audiobooks when I do it, so that's how I get my reading in. Jef Akst: I love that. All right, well that's Gabby to everyone. while we're on this, let's get to our other introductions. Annaleigh. Annalee: Yeah, I'm Anneli Armstrong. I'm senior editor for Biospace and I do our weekly Biofarm executive newsletter that goes out every Wednesday. Heather: And I am Heather McKenzie, your former host, now subject matter expert. And am senior editor here at Biospace and ⁓ I together Clinica Space, which arrives in your inboxes every Monday. Jef Akst: And once again, I'm Jeff Axt, Managing Editor. And with that, let's get into it. So the first topic of conversations today has got to be, once again, the FDA. So as Annali and Heather were recording last week and discussing the rumors of McCary's ouster, it became official. Marty McCary did resign as FDA commissioner last Tuesday. And dominoes have fallen since. So it's not just him. ⁓ also Tracy Beth Hogue. I'm Jeff Axt, Managing Editor. who was the acting director of CDER for a good six months or so. So acting director turned into a ⁓ somewhat temporary or somewhat permanent position there, she has now also left. And we also have Catherine Zarama who was acting director at CBER following the exit of Benign Prasad. She is also out as the CBER director, although we got confirmation from HHS just today that she will be staying at FDA in a different capacity. ⁓ We have the chief of staff, as well as the Chief AI Officer, all out of their positions at in the short week since McCarrie left. So, like I said, feel like the dominoes are falling. Heather: Yeah, it seems kind of like a bit of a Mia culpa, know, from HHS. They're saying like, we were wrong about the whole McCarrie-Bressard tandem. So these people are going, although we're going to tuck a couple of them in over here. ⁓ Jef Akst: You know, it's interesting that you say that, because I had that thought when McCary left so shortly after Prasad left. I feel like McCary maybe had some ownership over Prasad, who he recruited twice, because he also, know, or sorry, Prasad only came back after that 10 day vacation last summer at McCary's behest. And of course, Prasad was the center of all this controversy. And so I wondered if McCary had some ownership over that and if there's any relation there. I'm not sure. I'm speculating at this point. Heather: thought there's probably a pretty close relationship between those two because they've written a lot of articles together even before their time at the FDA. as we all know, everything back on the boss, right? So, you McCary the ⁓ credit or the non-credit for anything Prasad did, which was a lot as we know. Jef Akst: Thanks for following. Absolutely. No, I think there's something there. And we all know that Prasad was a center of much controversy and controversy. And after the Moderna gate back in February, I feel like that's when that was kind of the beginning to the end of his reign. Just a month later, they announced that he would be out at end of April, ostensibly at the end of a one year leave of absence. And then, yeah, all within weeks of that departure, the rest of this has has fallen into place. Heather: Yeah. Jef Akst: That domino effect aside though, let's talk a little bit about the reaction from the industry to McCarrie's exit. I do feel like it's been mostly positive, not Heather? Heather: Yeah, it's mostly positive. It also kind of seems like the analysts are like, okay, here we go again. But last Capital Alpha, who has some really interesting insights, ⁓ they and this is, quote, McCary oversaw what must be the most damaging period in FDA history. So that was pretty definitive right there. And BMO Capital said, quote, this is likely a broad positive for biopharma. given the regulatory inconsistency that defined his tenure. Jef Akst: Yeah, that's the feeling I was getting too. McCurry maybe wasn't the greatest FDA commissioner, so his exit by default is positive. I did think it was very interesting though that truest securities analysts put out there that this isn't the worst in part only because like you said, this is the norm. It's just more of the same. And so for that reason, the industry has gotten a little used to dealing with it. flip side of that same coin though, the industry is not happy with the instability at FDA leadership. so... they're still clamoring for that stability and they want a permanent leader and Capital Alpha again, back to their note, talked about how it's gonna be a real struggle to find somebody who actually wants this job. And I heard on last week's podcast, you mentioned Brett Griot as ⁓ of the names that was thrown out there. He actually took to LinkedIn to say, please no, please leave me out of this. I don't want this job. Heather: I was joking around earlier with, I think it was with Gabby, that we should just, that everyone at FDA should just send a blanket email saying that they don't want the job if they don't want the job, or maybe they should just send around a little forum. I'm willing to be FDA commissioner, not willing to be FDA commissioner. I mean, at this point, it kind of just, yeah, like am this much willing to be FDA commissioner. I am not willing. Yeah, but yeah, it's definitely, Jef Akst: Right. Annalee: Like a- do you mean like a scurvy monkey? one of those? Jef Akst: You Heather: It's definitely a tough job. Jef Akst: I mean, if the industry has it say, they would like Richard Pazder to come back. So hundred biotech leaders wrote a letter directly to President Trump suggesting that Pazder do come back as the acting FDA commissioner. Heather: Yeah, this is really interesting and not really surprising. He's very well respected. He's an FDA veteran of 26 years who actually stepped in to lead CDER very briefly last fall. The industry really celebrated this appointment in, I think it was late October or November. But just a few weeks later, Pester suddenly left. he later pointed the finger at McCary saying, quote, wall between the commissioner's office ⁓ and the review staff has been breached McCary's leadership. So with McCary out, maybe he would consider back. Jef Akst: Yeah, we'll have to see. Whoever takes the role though, it may be a while. I've definitely heard a lot of commentary. Obviously the new commissioner would need to be Senate confirmed. You know, we've talked a lot about the CDC challenges and getting a permanent leadership there. That's still just an acting director. So I don't know how long it will take we get a permanent FDA commissioner. I have heard that maybe it would be after the midterms at the earliest. For now though, Diamantis is in charge and we'll see how that goes. ⁓ you know, doesn't have the normal qualifications of an FDA commissioner. Heather: Yeah, I think I read somewhere the first FDA commissioner acting or non-acting in about 50 years to not have a scientific background. I saw this LinkedIn post yesterday from Steve, ⁓ Oustin. said that ⁓ Kyle DeMantis getting really dinged as the acting commissioner pick. But he's the person in years to leave the FDA without scientific or medical credentials. Ustin argued that his limitations may paradoxically as a stabilizing function ⁓ without scientific standing to challenge or try to override career reviewers, he is unlikely to try. Jef Akst: I took note of that too, because obviously one of the complaints about how the FDA is being run right now is the intervention of leadership and oftentimes into departments where they don't have authority. And so, yeah, maybe Diamantis will just kind of be a steady hand staying in the background until the position can be filled. We'll see. In terms of what's at stake though, McCary started a lot of policy initiatives that I think the future of them is very unclear with him now being out. So just to throw out a couple, we've got the plausible mechanisms pathway. We've covered that a lot. This is for personalized therapies for rare diseases, know, jumping off of this baby success story last year. McCary also the number of pivotal trials required for approval from two to one. RBC analysts called these pro-innovation initiatives and I think they were broadly ⁓ received well by the industry. But with McCarrie out, we just don't know what the future of these programs is going to be. I the other that Truist brought up is that we're also in a pretty critical time for PDUFA eight reauthorization negotiations. So that's another question mark. RBC put it bluntly when they said, whoever ultimately leaves this agency will have a major impact on the FDA's overall direction. We are in a time of flux and suddenly we don't have a leader at this agency. So whoever comes in to replace them will pick up that time of flux, step in at this time of flux and determine which direction it goes, either continuing in the direction McCary had pushed or in a completely different direction. Heather: getting back to McCary, he you know, he might not have been a good manager, but he did have some good ideas. And the rare disease community has spoken about this. Although I read an article ⁓ written three rare disease advocates yesterday who appear to pretty much believe that he just created more red tape. So I guess it really does come down to the implementation. Annalee: thing I have to wonder is like this did seem like it was a long time coming and there was a lot of discussion that Trump was unhappy with McCarrie's leadership. So when he was from his position, why didn't they have somebody ready to nominate? It just, you you think about how important this agency is and the time of flux that we're in, like would have really been a stabilizing force they had said he's resigning, but this is ⁓ this is who we're to nominate. ⁓ That seems like a missed opportunity. Jef Akst: That's a really good point, because rumors first circulated that he might be out last November. so we, that's when I think that was when we started doing our pre-writes for, know, McCarrie potentially getting out. And he seems to have survived that for another six months. But you're right, if that was even in the conversations that were being had among his bosses, they could have planned a little better. Heather: Maybe they were just waiting for somebody to step up and say, I'll do it, and nobody did. Jef Akst: Instead, people are stepping up and saying, no, I will not do it. Heather: Yes. The Weekly 5.12: Yeah, I think it speaks to how hard it is to recruit for that job and how no one wants it, honestly. Jef Akst: I mean, even at the Cedar Director role, they had to convince Pazder to step up. He was, you know, the longtime oncology regulator. He seemed happy in that department and McCarrie really had to push to get him to step up to the Cedar role. FDA commissioners, a whole nother level. don't, yeah, I don't know who would want it, especially at this time of a lot of controversy and a lot of negative feedback from the industry. So a really, high spotlight would be on the position at this time. All right, well that's the FDA. We will keep you updated on those many developments. We will have more leaders coming in and out, I'm sure, all of at FDA Commissioner and at CDER and at CBER. These are all acting positions. So we are actively awaiting news of permanent leadership at the and stay tuned to for updates on that. In the meantime, let's move on to some other topics that our listeners are very excited to hear about. Lee, you have been doing Biospaces annual analyses on top. big pharma executive pay packages. So let's hear what you've got coming up next. Annalee: Yeah, for sure. So this is our kind of compensation month. do this every year where we analyze the compensation of the pharma CEOs. ⁓ Last week, we profiled the highest paid CEOs in 2025. Eli Lilly's David Ricks, of course, came out on top. But this week, we're looking at how those salaries compare to everyday workers at these companies. So J &J's Jaqueen Duado made the most relative to rank and file employees. he had a median pay ratio of 358 to one. So that means 358 employees stacked up equals his salary, which was $32.6 million total for 2025. So pretty, yeah, pretty big gap. Jef Akst: Not too bad. Well, how about Lilly though? Lilly is obviously the top earning farmer, or least the top I shouldn't say top earning. I know J &J took that spot, but with Lilly, with their market cap pushing a trillion, what's going on with Rix? Annalee: Yeah, for sure. So Ricks made $36.7 million total. That's, you know, in stock and salary and et cetera, et cetera. Like they don't exactly take home that exact value, but that's how much his compensation package was worth. So Lilly, their employees were slightly higher paid overall. ⁓ So ratio was only 293 one. So again, 293 at Lilly stacked up to equal their CEO, David Ricks. So those numbers are a little astonishing. It can be a little hard to swallow that. But then on the other side of the list, we had Vertex's Reshma Kualramani. She had a ratio of just 80 to one. She still had a, you her salary was $21.1 million. But the actual median employee at Vertex was the highest paid with $264,000. So. We got the low end and the high end. It's still a little crazy to think about those numbers. yeah, you read more about that in Biofarm Executive this week. Jef Akst: I feel like that surprises me more than the spread of the CEO pay packages is the spread of the median salary. Like that's a huge range from like, that's a pretty good average median salary at Vertex and then some quite low ones at the other companies. Annalee: Right, and J &J's median was $91,000, if I didn't say that off the top, which was the lowest. think that makes sense for J &J because it's a very big company. Like, it's not just pharma, they have medtech, they have healthcare. I it's just like, I actually maybe take a look at the employee counts because I bet it's ⁓ that number really going to skew those values. But yeah, Vertex, high paying company, good place to work. Jef Akst: but jumping back into the science now, we do wanna talk a little bit about pancreatic cancer this week. We obviously talked about it a month or so ago when Revolution Medicines announced its pretty awesome phase three trial results that doubled survival. As a reminder, pancreatic cancer is an extremely deadly malignancy. It's usually about a 13 % five-year survival rate. So to be able to double that survival was huge news. And I feel like the news cycle has continued to say a bit on pancreatic cancer. We got a note just this week from Truist analysts who have nominated Revolution as the quote, the next oncology titan. Of course, that title currently is held by Merck and it's blockbuster cancer drug, Keytruda. So Heather, I know you've taken a couple of deep dives into what's going on with Revolution and the pancreatic cancer space more broadly. What can you tell us? Heather: Yeah, Revolution or RevMed as they're called is a really cool company. yeah, they had those awesome results about a month ago. And know, we are in the midst of a revolution in ⁓ the cancer space, seems. I read, a dive into other companies in this space and there's really some great data coming out and some great data that we're going to be hearing I think at the conferences coming up. Merck, interestingly current Titan, reportedly was in talks to buy RevMed for up to 32 billion late last year. So ⁓ maybe they their own worth and they stayed as a standalone company. But the other thing that I really kind of pressed people on during that deep dive article was that Diraxon or Diraxon, which Truis it, has some pretty rough side effects. So I spoke with CEOs from a couple of companies on actuate therapeutics and immune nearing who believe that their candidates can one negate the side effect and two possibly provide even more time to patients either on their own or in combination ⁓ with like, ⁓ with like revolution. Jef Akst: Yeah, yeah, the Diractin, that's RevMed's product and that's the one that doubled survival. Yeah, no, I did hear about those side effects as well, but doubling survival is nothing, you know, that's huge. And obviously cancer patients care more about survival than the side effects. And so I could see that one moving forward, but obviously plenty of room for growth and differentiation if you can improve upon those side effects. Heather: Yeah, no, you know, you say it doubled survival, it went from 6.7 months in advanced pancreatic cancer, it's another good thing to point out is that this was metastatic disease to months. And that's awesome. know, there's a lot incremental gains that are ⁓ common to the space. And ⁓ I kind of to be a person where I want it all. I want, you know, like I want five, 10 years, you know, like I want, or just cure. But that's also a that oncologists are very careful about using. But yeah, it's cancer conference season and Revolution is planning on presenting full data from their phase three trial at ASCO. And Truist in their notes said that these data, are supposed to include progression-free survival, overall response rate, and duration of response could really help to fine tune treatment duration assumptions for them. Jef Akst: yeah, we'll have to look forward to those data. And yeah, I'm just excited. If anyone knows someone had pancreatic cancer, it's just the most devastating diagnosis. So yeah, it does seem kind of incremental, just an extra six and a half months on survival, but when the average was only six months to start out with, it's a ray of hope the space. Pivoting now though to bigger safety concerns, not just the relatively compared to what we're to talk about side effects of RevMed's drug. The gene therapy space continues to be riddled with safety concerns. And most recently, this came from Regenexx Bio. last week, they announced mixed results from a phase three program for a gene therapy for Duchenne muscular dystrophy. And it did lead to improvements, there were two serious adverse events caused the company's stock to drop by 37 % to just $6. So, ⁓ Annali, covered news. What can you tell us? Annalee: Yeah, this was kind an interesting result. Regenexx Bio in general is having a bit of a tough year. Last they reported what they tried to sell as a major win for their DMD gene therapy. they reported that their trial did its efficacy endpoint. But there were two cases of serious adverse events. which understandably spooked investors sending the shares down. One of those events was liver related, which is always big when it comes to gene therapies. The other was a case of swelling in the heart, both resolved, but still this was not a great signal for that trial. Regenexx's shares fell 37 % after the announcement. Jef Akst: Yeah, liver concerns have definitely been a recurring issue with gene therapies. I mean, we all remember Sarepta last summer, there were three patient-related, or treatment-related deaths, two with Elevitis, the Duchenne muscular dystrophy gene therapy that's on the market, and another one with an investigational gene therapy for limb girdle muscular dystrophy. they both use the same viral ⁓ platform, and they were both associated with liver complications. So that is certainly a concern in this space. I'm hoping that these gene therapy makers can improve upon that. That's something that we've continued to watch and looked into. You know, we've got a couple of stories on organ specific delivery. If you can avoid the systemic delivery, that might be one way to go. More targeted vectors. There's certainly a lot of different approaches that we could take to improve safety, but, it's something that people are working on. So we'll continue to cover that. But in terms of the muscular dystrophy space, the positive result is that Regenexx did get some good efficacy data and they're not alone in terms of these late stage positive results. Heather: Yeah, no, they're definitely not. And I wrote another article that's going to be out in the Clinica space next week, and it's on the DMG space more generally. And there are a lot of companies working on solving these problems with the gene therapies, these safety issues, but also just tons of different other products that are, you know, there's exon skippers, there's lots of different approaches. There's no Vardis, which bought Avidity, of course, last year for $12 billion. And they have a highly anticipated readout for actually a different type of muscular dystrophy called DM1 drug that one analyst told me could make or break the acquisition. So putting a lot of pressure on one readout. Back to Duchenne, there's who ⁓ recently reported a positive meeting for its Exxon skipper. And we of course talked a lot about in the past year or so, has August date with the FDA for its DMD cardiomyopathy cell therapy that was rejected last July. And so then, Darumysl has aced a phase three trial. So they had a pretty good comeback with that. So yeah, there's lot of muscular dystrophy drugs that are either currently in review or approaching review now. So exciting year. Jef Akst: Yeah, that'll be really fun to watch. Hopefully we get some drug options and therapy options, I should say, available to that patient population. Well, finally, we do have one more safety concern to touch on. This one pertains to Amgen's rare disease drug, Tavneos. So going back to January, the FDA had asked Amgen to pull the drug off the market due to an issue with the data that had led to its approval. Amgen denied that request. And then last month, the FDA alleged that the data had been doctored. This was all swirling around in the background and then this week we have now reports that this drug has been linked to 20 deaths in Japan. Gabby? The Weekly 5.12: Yeah, so the drug has been tied to those 20 patient deaths since market launch in 2022 in Japan. So the findings come from Angem's partner, Kisai Pharmaceutical, and most of the deaths are tied to a complication of drug-induced liver injury. So kind of despite these new findings, which did prompt a safety alert to providers in Japan, that instructed them not to start new patients on the drug. Amgen is standing besides their drug. They put out a Friday statement providing further context behind the deaths. Interestingly though, Amgen, while they issued a release about the drug, which it acquired back in 2022 with a 4 billion cash buyout of a company called chemocentrics, the pharma did reach out to us asking for their name to be removed from the headline of our story since Kasai, who markets the drug in Japan, was the one who shared the news about the deaths. Obviously, we did not remove Amgen's name from our story since they are the owner of the drug. Jef Akst: Yeah, that does feel like splitting hairs to me. So yes, but yes, that is good to clarify Amdun does not own rights to the drug in Japan where the deaths happen. All right, well finally, last but not least, we always like to check in on Biofarm executive. Annalia already kind of gave us the update on the exact pay packages, but what else you got going on in BE this week? Annalee: Yeah, I didn't want to leave out my, one of my favorite stories I've written so far this year, I got to tour a radiopharmaceutical facility while I was in Salt Lake City last year, last month. Feels like it's been a year apparently. But yeah, I toured a new Sano's facility. It is brand new, state of the art. They took through the entire radiopharma manufacturing hall. ⁓ It was super I got to see the particle accelerator. ⁓ their ion source, is how the are created. And I got to go into what they call the Linac Hall, which where the actual radiopharmaceuticals will be created. The facility isn't completely up and running yet. So I was actually one of the very few people who's ever going to get to tour like the entire facility and see this in real life. I had so much fun. over at New Sauna was so great. One I really appreciated is how good they all were at like describing this in layman's for someone like me, which helped me ⁓ describe it in way for our readers that anybody ⁓ can drop in check that out. So please check out my story. It was super fun. This took me a long time to put this one together. So you can check out in BioFarm Executive this week. and in manufacturing grief, I should say. Jef Akst: You don't want to miss that because you'll see Annali in a hard hat. So check that out. Annalee: Yes. Yes. And I got to play with their little simulator that is used to move the targets around. was super cool. Heather: you Jef Akst: Awesome. Well, thank you all for your time and your input and thank you all for listening. That's all for the weekly this week. We'll see you back next week. Hello, and welcome to Biospaces, Weekly. Today, we're going to dive the fallout from the resignation of FDA Commissioner Marty McCary. That news broke as my colleagues recorded last week's episode, and then other senior regulators were removed from their posts in the immediate aftermath. We'll also circle back to Revolutions pancreatic cancer breakthrough and give you an update on Regenexx Bio and its gene therapy for Duchenne muscular dystrophy. a spoiler alert there, safety issues have again marred otherwise positive results. Speaking of safety issues, there's also continued drama around Amgen's rare disease drug, Tavneos. and finally we'll get another sneak peek at Biospace's analysis of top paid CEOs in pharma. Let's get into it.