Latest / BioSpace / Trump’s psychedelics push, Lilly’s up to $7B CAR T deal, Replimune’s saga and Denali’s big win
Transcript
- Heather: Hi guys! Annalee: Hello. Jef Akst: Welcome back. Hi, welcome back, Annali. How was Biohive? Annalee: It was really fun. I had a great time in Salt Lake City. Heather: Hm. Hm. Jef Akst: What did you do? Just talk to lot of folks. Heather: That's not like... Annalee: I talked to a lot of people. of the best things was that I got to sit down with a bunch of early stage company founders, young people who were starting companies, and they were just so passionate about everything and so excited. I've been a lot about how we're starting to see some good news out of biotech, and it was just really cool to see how excited people still are in this industry. ⁓ see that happening in real time. Jef Akst: I do find that really refreshing. I mean, I'm thinking back to last summer when we went to bio and we weren't in such an upswing in terms of where the industry was. And it was almost a little bit depressing to be covering this industry with all the uncertainty and the challenges, but going to bio and being on the ground with these scientists and executives, young biotech leaders, I mean, their excitement is just so infectious. Even in the worst of times, they are committed to their science, they're committed to their patients, and it's really a lot of fun. Annalee: Yeah, and like I, you know, I'm not ⁓ to give away what I'm going to be writing about in the coming weeks, but got to tour a radio pharmaceuticals ⁓ manufacturing that is going to be coming online later this year. And these guys were so excited to show me everything. They were like, this is so cool. And like, it was just so much fun, this tour. like, you know, stay tuned for that. It's going to be coming out in the next couple of weeks because I need to wrap my head around it. But yeah, just a great trip. Heather: You Jef Akst: you That's so cool that you got to go in there because usually those are like pretty protected, right? Because it's radioactive. Okay. Heather: I love it. Annalee: Yeah, and it's not yet. I'm one of the very few people who will ever get to tour this place outside of like ⁓ in like nuclear hazmat suits. So was a pretty special thing that I got to do. Jef Akst: Very cool. Heather: your outfit, Annali. I think you should use that picture with the hard hat as your new LinkedIn profile. look good in hard hats. Well, speaking of biotech and all good things, why don't we get to our show? So, intros. ⁓ Annali, why don't you go first? ⁓ Annalee: Yeah, I know. I really rocked hard hat that day. ⁓ Jef Akst: You Annalee: Yeah, I am Annaleigh Armstrong. am senior editor and I do the Biofarm executive newsletter, which comes out to your inboxes every Wednesday. And as I said, lots to come from my Salt Lake City trip in the next couple of weeks. Heather: And I am Heather McKenzie, editor. ⁓ responsible for ⁓ Space, which put out a special edition this week on the oral obesity race between Eli Lilly and Noquin Ordisk. Jef Akst: Definitely check that out. If you're not already subscribed to Clinica Space, if you sign up now, you will get that special edition, which is only available through the newsletter subscription right now. I'm Jeff Axt. I am managing editor. And as always, putting in plugs for some of our other newsletters, we have our daily gene pool, as well as manufacturing brief on Tuesdays and Career Insider on Thursdays. Heather: All right. Well, let's get to our show. So this weekend I was, I was again at the mail salon and I read news alert about a press conference or an executive order signing ⁓ that President was holding in the Oval Office. So. The president signed an executive order instructing the FDA to grant commissioners priority vouchers CNPVs as we tend to call them around here to appropriate psychedelic drugs that fit the program's criteria of aligning with certain national priorities. Thanks Jef Akst: That aligns with RFKs. He's been, I should say his full name upon first mission. If you don't know Robert ⁓ Kennedy Jr. He's been talking about psychedelics since he became health secretary, right? So he's been kind of a proponent of this modality as well. Heather: Yeah, definitely. He's been critical of past by the FDA of not approving ⁓ certain or taking this long. And they are planning to not take very long anymore. So the reaction the psychedelics companies, I've been talking to a few of them in the past couple of days, I talked to a very frazzled but excited Compass executive yesterday who they'd had the news for all of about four hours. for business day hours when I talk to them. But they're excited. It's been overwhelmingly positive, as you would imagine. But some people do have concerns. ⁓ Yeah, so lawyer, sorry, lawyer, she's an ethicist, a professor of ethics that I spoke with. She mentioned kind of concerns around the perception of a new class of drugs being ⁓ Jef Akst: yeah, one of those. Heather: granted these priority vouchers, can shorten the time of review from 10 to 12 months down to one to two months. So there just is some concern around whether it's true or not. These programs, ⁓ the that we're assuming probably will get the CNPVs, ⁓ have robust data. But there's the idea that can you really make a decision on these or really review everything in one to two months? Jef Akst: Yeah, and a whole new class of drugs. It's interesting though, once one of these drugs does hit the market, we've reported a couple of times now, you have Heather, on how there'll be an additional delay because we've got to now delist these drugs that are schedule 1A drugs. So Trump support behind this drug class could potentially expedite that next part of the process, which would be positive. Heather: Yeah. Thank Yeah, the executive order also addressed the DEA and the process there as well. So these are definitely coming. They're coming and we're to have to work out a lot of logistical issues. I think I've seen some of this data and I've heard some of the stories and it just sounds fantastic for psychiatric field, psychiatric diseases that really have so much need. Annalee: Okay. If you think about the last time these drugs came before the FDA too, it was Lycos, right? They've changed their names like six times. ⁓ But they, you know, their therapy was ultimately rejected because of some problems with their study. it it's going be interesting to see if the FDA is able to move these along you think about that last experience because Heather: Mmm. Mmm. Yeah. Annalee: Just seems like, again, new class of medicine, they really should go through a very robust review. There's already a history. I know all of these drugs are different and have gone through different pathways, but it just seems to me that this is not a drug class that should go through without an meeting, frankly. Heather: Yeah, the ethicist that I was talking to mentioned that too. She mentioned that they're putting all of these psychedelics kind of in the same bucket. Like they're just talking about them as psychedelics instead of psilocybin and LSD and Ibogaine, which the executive order doubled down on and which doesn't even have any clinical trials currently going. Although apparently there was ⁓ an IND, investigative new drug application approved for but I can't find it. So I have to do some digging there. Jef Akst: agree that too often these drugs are all lumped into just psychedelics. And yeah, it's exciting. And yeah, there's a lot of promising data from different types of psychedelics, but it is very confusing. So we do have, if you know, technically a psychedelic on the market and J &J Spravato, excuse me, and it's it's esketamine. So J &J will tell you it's not a true psychedelic, but so many of our analysts have come through and talk about Spravato as sort of the comparator for all these new psychedelics coming to market. So there's yet another example of a very different drug. Heather: Mmm. Mmm. Right. Jef Akst: N-antium of ketamine, S-ketamine. It's not LSD, it's not MDMA, it's not psilocybin, but it's discussed in the same breath. So ⁓ I agree with that. We need to take these a case by case basis. There's a lot of promise here, but each one is going to need a thorough review. Heather: You Annalee: I remember I did the edit on our story about this and like it's, know, an analyst said, this will be the first psychedelic or, you know, ⁓ on the market. was like, wait a minute, Spravato. And then I like went way back into it like, I was like, okay, this is technically true, but you know, so like, there's a lot of debate happening there. ⁓ Heather: Yeah. Yeah, and J &J is very insistent that it is not a true psychedelics. We've gotten emails about that. But yeah, it'll be really, really interesting to see what happens here. There are three priority vouchers that are supposed to be given this week, and ⁓ we're that two of them will go to Compass Pathways and Definiium, formerly MindMed. But it's of up in the air with a lot of analysts who the third is going to be. Jef Akst: Yeah, a lot of nuance. Annalee: They sure are, yeah. Heather: It'll be interesting to see. well, this isn't the only kind of big news and kind of controversial news that has happened over the past past few weeks. So that that controversy revolves around replamune. And whether the FDA is using appropriate flexibility when approval or rejection decisions. So replamune has kind of become the poster child for this. Jef Akst: Yeah, getting rejected twice in a year is not a good look, not fun for the company. Heather: Yeah, and they've laid off, I think it is about 60 % of their workforce at this point. So ⁓ was a real hit for them and they're not sure that they're going to be able to take this product forward. But it was interesting. I read a few weeks ago a Politico poll that ⁓ said that majority of Americans are actually in favor of slowing down the approval of new drugs to ensure they properly tested. even if it means delaying access to those therapies for patients who really need them. So we put our own poll out in the field because I was going to write an editorial and was a little bit more mixed actually. So 34 % said that the FDA was too inflexible, they wanted flexibility. 23 % said that the agency is too flexible. 19 % thought there was an appropriate balance. And 24 % were not sure. Jef Akst: surprised that we got different numbers than Politico. Politico has definitely a broader, more general audience. We are very focused in biopharma, so it's interesting to see what industry professionals are thinking about all this. I'm not surprised that 24 % were not sure. I kind of expected that number to be higher just given the uncertainty and lack of clarity on some of these decisions. But yeah, some interesting numbers there. Heather: Mmm. Yeah, so interesting too, because even at the highest levels, people seem to be really conflicted. So the rep-l-immune had two different FDA review groups and they did that to prevent bias. But both of them came down to the same decision that it wasn't efficacious. However, 22 melanoma experts who wrote a of a protest letter after the first rejection, believe that RP1 should be approved. a lot of really high level people there disagree. Jef Akst: Yeah, I don't quite know what to make of that and it's gotta be frustrating for patients in this area. Heather: . Yeah, and definitely for Rep Immune as well. their CEO has expressed that frustration. you know, I totally get that. This is your baby or your, you know, your project for years. And, yeah. Jef Akst: Yeah, and he did say that another trial would probably not be viable. So that might be the end of the road for RP-1. Heather: Yeah, just too bad. meanwhile, week, RFK Jr. appeared before ⁓ and he defended FDA Commissioner Marty McCary's decisions such as Reflum Mew. And he said, and this is a quote, the pharma industry own Congress, they own the which I have a bone to pick with that. I do not think that they own us. We have very ⁓ coverage. ⁓ yeah, to make that clear. But, you know, any time that RFK appears in front of Congress and says the Ways and Means Committee, and I think there was there's one more, it ends up being controversial. Annalee: I do not. Yeah, just to put it out there, Biospace, no ownership by Pharma Company. Jef Akst: Well, the FDA is not the only agency that has some controversy right now. Of course, we've covered the CDC, not as extensively, it's not as directly relevant to our core coverage area, but we do want to know what's going on at the CDC. And as you well know, we've had many leaders over the past year and only one Senate confirmed leader, Susan Meneris, last year for like a month. So we have been eagerly awaiting a nomination for a new CDC director after the Heather: Thank Jef Akst: fourth, I believe it was, temporary interim director left. Until this point, NIH director Jay Bhattacharya has been running the CDC. But now, as of last week, we do have a nomination in Erica Schwartz. This was announced Thursday afternoon by President Trump on Truth Social. Schwartz was a deputy surgeon general ⁓ under Trump's first She has a medical degree, she has a law degree, she served for ⁓ 24 years in the US Public Health Service Commission Corps, and she also served in the Coast Guard. So interesting history. And like I said, she follows both Susan Monteros and then we had a temporary leader in the HHS Deputy Secretary, Jim O'Neill, and then most recently, Jay Bhattacharya. So everybody is eagerly waiting a more permanent post at the head of CDC. And then at the same time as announcing this nomination, we got word of some other nominations for the CDC. So a couple of other folks to throw out there, Jennifer Shuford, she's current commissioner of the Texas Health Department, and she will serve as, if Shorts is confirmed as CDC leader, will serve as her deputy director and the agency's chief medical officer. And then finally, Brenner, who is currently FDA principal deputy commissioner, will serve as public health advisor to Kennedy. have Sean Slovinsky, a former executive at Walmart and Humana. He's going to become the Deputy Director and Chief Operating Officer at CDC. Heather: So lots of appointments and or nominations. And yeah, this has been almost as much of a revolving door as the FDA's ⁓ CEDAW director. hopefully, is confirmed and will stick because she's been pretty well received throughout Pharma. She's got a lot of credentials. Yeah. ⁓ Yeah. Jef Akst: And just that stability, you know, we've talked about it. We've covered the FDA more extensively and we have had a consistent FDA commissioner in Marty McCary, but a lot of the positions under him have turned over a lot. And that, like you say, revolving door, that turnover of leadership has just created and continued this uncertainty for the industry. Heather: Yeah. Well, that's the regulatory space for this week. Moving on to the business side, ⁓ &A continues to roll. Eli Lilly spent some more of its money this week, yeah, this week on CAR ⁓ Annalee: It's really hard to keep track, isn't it? So many deals. So yeah, ⁓ you mentioned, Lilly definitely made another acquisition. They are buying Colonia Therapeutics for up to $7 billion. It was something like 3.25 billion upfront, the rest in milestones. So ⁓ Lilly was of the big farmers that was predicted, I believe by Jeffries, that they could buy again, and they sure did. So... Heather: very hard to keep track, but like one week. Annalee: Colonial Therapeutics will bring some more CAR-T technology to Lilly. initially entered the space in February with the $2.4 billion acquisition of Orna Therapeutics, which was very exciting at the time. Lilly has never been in CAR-T and they it earlier this year. So now they're buying another company just to double down on that technology. Analysts said that the Colonial Deal is very complimentary. is bringing a gene delivery system while Orna offered up an anti-BCMA CAR T cell therapy approach. So combining those two things, have differentiation. They have kind of like a broad platform now. So lots of cool stuff happening at but Lilly is not even the only company that did something great in cell therapy this week. Jef Akst: Yeah, it's really interesting to see the commitment that we're seeing from Lily. And now I'm going to talk with you about UCB, which scooped up Neurona, another cell therapy commitment. And this is on the heels of some big dropouts from the space. So we heard last year that Takeda was no longer going to invest in cell therapies and Novo Nordisk similarly pulled back. So it's exciting to see this investment. With the UCB deal, they committed a $650 million upfront and up to $1.15 billion total to get Neurona. The lead candidate that is kind of the centerpiece of the deal is an investigational cell therapy that's currently in phase one, two development for a type of So it'd really exciting to see cell therapy kind of get the second wind and see what comes of these deals. Annalee: I don't know why I'm just hearing about cell therapy and epilepsy right now, but that's very cool. Heather: Thanks. It's also really cool to see Lilly spending its mountains of cash on really promising areas like this. cool to see them differentiating and it's a great business model for them. Annalee: Right. This is what you want to see. You want to see companies that have innovation, that are making a lot of money, take that and bring it back to the start and figure out new ways to make medicines. again, cell therapy isn't a new way to make medicine, but Lilly is doing something new to it, at least here, and out a modality that has, you know, kind of been on a challenging path the past couple of years. Heather: Mmm. Yeah, it really just shows that biospace, so, biospace as well, that biopharma is working. That was a great flip, huh? Annaleigh, you wrote in a great editorial this week ⁓ talking about the many ways in which biopharma is working right now. Annalee: Love that. Love that slip there, Heather. Yeah, so there's just been so much great news in biotech. It's really kind of, I wouldn't say inspiring because it's like, I don't know. But I was... ⁓ Heather: You Annalee: On my layover in Chicago on my way home from Salt Lake City, and I just kind of kept thinking about all of the really great stories we've been writing about lately. RevMed, doubling survival in pancreatic cancer. We talked about that a couple of weeks ago on the podcast. On Friday, Kylera Therapeutics had a massive $625 million IPO, which officially now holds the record as biotech's biggest IPO. I don't... I don't cheer that because, you know, a bunch of investors make money. I think about it again as money getting recycled back into this industry to hopefully fund more biotechs, fund more innovation. biotech is just really shining right now. ⁓ can read kind of about my thoughts in my editorial that appeared in Biofarm executive this week. Heather: Yeah, it's a terrific piece. It has a really nice sunshine on it too. So if you're having a, it's a picture. So if you're having a rainy spring day, make sure you read Annalise editorial. You'll both feel happy and be happy. So moving to our weekly weight loss segment, Elia and Lily, so Elia and Lily got their oral weight loss drug, Vandeo, approved on April 1st. So not very long ago. And In Fentana's approval letter, the FDA had flagged safety risks affect the heart and liver. And signals, they found them serious enough to require that do a post-marketing trial. Just a couple of days later, however, because everything comes up Lilly, the company posted data from a diabetes trial where no liver signals were observed ⁓ and there was a reduction in cardiovascular events compared to long-acting insulin. So that may have answered some questions. Annalee: love how Lily's clinical program for this drug is just like insane that they're like, okay, here's some data. We can figure this out really quickly. it just, no kidding. It really speaks the breadth of these clinical programs, which I mean, I don't know if there's ever been anything like it, what Novo and Lily are doing with their GLP ones. Heather: Great PR strategy. Jef Akst: Yeah, I mean, the launches kind of speak to that as well, right? So we were eagerly awaiting numbers from the first week of Foundeo's market potential and we got 1390, 1390 prescriptions. That did kind of look not nearly as impressive as Novo's or Orgovi, which got more than 3000 prescriptions in that first week, but it wasn't really apples to apples. Foundeo was launched on a Thursday, so it's really just two days worth of prescriptions. nearly half of the 3,000 or so that Novo got in a full week on the market at the beginning of January. So very impressive numbers for a little bit of context to ZepBound. Lilly's Injectable hit 1,300 new prescriptions in the first week. We thought that was impressive back in December, 2023. So every new obesity drug just keeps outpacing, especially these oral options are definitely kind of flying off the shelf, so to speak. I would say, you know, I wrote an editorial on this a couple of weeks ago. I am very eager to continue to watch these numbers and see how they play out. Obviously, Lilly has very clearly won the injectable race and that was, I think, for a very clear reason of it had this great efficacy advantage, this head-to-head trial, % more weight loss on ZetBound. If you're choosing between two shots, you might as well go with the one that you're going to lose more weight. For the pills, it's less of a clear upper hand for Lilly or Novo, to be honest. NOVO will argue that they have higher efficacy. If you look at the numbers at 72 weeks, they do have a higher weight loss. There has not been a head-to-head comparison here. And there are some other differentiating factors such as manufacturing and diet restrictions for when you actually take these pills. So I'm very unclear on who will succeed in this race in the long run. I feel like obviously there's a huge market here. Probably both companies will do just fine. But I wonder if Lilly will kind of have the slam dunk victory as they seem to have taken in the injectable race. Annalee: Right, and you know, we still are having plenty of back and forth on the injectable side too, in terms of data. There was a pre-print article that came out last week suggesting that Novo-sema-glutide may produce better body composition than Lillie's terzapatide. This was an observational study of 8,000 people who had ⁓ taken GLP-1. There's a of caveats to go through here. Like, you know, this isn't a great apples to apples comparison, but they did it. And basically the study suggests that patients taking Novo's drug lost less which has been kind of a big issue with these GLP-1 drugs. As you lose weight, you're losing muscle too. So without kind of exercise and being very careful about that, people on these drugs can experience loss of strength those kinds of things. So again, Novo appeared to take the edge. However, the patients and the trisapatite arm actually lost more weight overall. So it would make sense that they would lose more muscle as well. So it's a bit messy, just another data point in this really heated battle between these two companies. Heather: I think we're gonna be watching this battle play out for years, but hopefully the biggest winners are the patients. I know I read something earlier this year where the numbers in terms of in the US are already dropping for the first time in maybe a decade or a couple of decades or something. Annalee: Yeah, wasn't Lily's CEO, David Ricks, quoted as saying that he's disappointed that everybody in America is done with one of these drugs? Heather: Thank Jef Akst: I saw that. was like, you're disappointed you're only making so many billions. need more. But yes, there is a huge market. So there you go. Heather: It's like... You know, well, give them a break. Maybe they want to invest in something really expensive. I mean, it's okay. just to also note, AACR is ongoing. So a lot of cancer experts convening in San Diego. We've covered results from Merck, Moderna, RevMed, and B1 among others. So out Biospace that. Annalee: the app. Jef Akst: You Heather: Well, Annalee, I you spoke at the top of the show about your trip to Salt Lake City. What can expect to see in biopharma exec this week? Annalee: Yeah, so in contrast to, you you were talking about how RepliMune CEO was saying that this is a really difficult time for him. I got the chance to actually sit down with Denali Therapeutics, their CEO, Ryan Watts, to about kind of the flip side of that journey. Denali was able to approval for the Hunter syndrome drug Avlea a few weeks ago. So this has obviously been a deeply personal journey for ⁓ Ryan Watts. So I to sit down with him. on the sidelines of the Biohive Live conference last week. And he told me all about where this started, ⁓ it felt to get this approved, and really the kind of personal stories that have come up along the way. So really enjoyed getting to tell this story. You can read that ⁓ in Executive this week. And then other story that we have this week in light of Kyleria securing the largest ever biotech IPO. Tristan Manilak put together a rundown of the top five companies on that list and where they are now. Spoiler alert, Moderna has now moved down to number two. They had a IPO that was worth about 600 million in 2018, right before the pandemic. So that's a really cool story, just recapping some ⁓ history. Heather: Yeah, I edited that Denali piece and it's really, ⁓ that one out. Ryan seems like a really good dude. Tears. ⁓ Yeah, of stories are really just great. Jef Akst: I was tearing up this morning during the QA on that. Like, the opening is just so good. Annalee: see, this is what I strive for as a journalist, is to make people cry. I don't get to do it very often. Jef Akst: You Heather: Well, that is it for us this week. I hope you all have a beautiful spring week and we'll see you next week. Hello. Welcome to the weekly by Biospace. I'm your host, Heather McKenzie. President Trump energized the psychedelic therapy space this weekend, signing an executive order instructing the FDA to grant commissioners priority review vouchers. to drugs that fit that program's criteria. ⁓ &A mania continues with Eli Lilly acquiring Colonial Therapeutics in a deal that could hit 7 billion. Lilly is in the limelight as usual with newly approved weight loss pill, Fondeo, generating headlines and sales. And everything is coming up biopharma. pancreatic cancer candidate doubled survival in this brutal disease. the biggest IPO ever and billions being pumped into and around the space. Let's get to it.