Latest / BioSpace / Moderna’s FDA adcomm, IPO mania, biopharma layoffs, more
Transcript
- BioSpace Marketing: Mm-hmm. Welcome to Biospaces the Weekly. I'm your host, Jeff Axt, and today we've got a packed lineup for you. First, we'll preview Moderna's upcoming FDA adcom for its mRNA-based flu vaccine, which was initially turned away from the regulator in February. We'll also cover the uptick in biotech IPOs, including two new records in as many weeks in terms of the money raised. And unfortunately, the uptick in biopharma layoffs at the same time. We'll check in on the hottest deal maker in industry right now, Eli Lilly. And discuss the latest discussions surrounding the commissioner's national priority review vouchers from the FDA. Finally, what do we know about the notorious psychedelic ibogaine and the FDA's green light of clinical trials for an Ibogaine metabolite? Stick around to find out. Hello, hello. You ready for bio, Annalie? I am. You know what just happened this morning is like another person asked me for an interview. So I now have like in in per like very typical Annalie fashion, I now have like back to back to back to back interviews. on my Tuesday. Like I can't I can't do it any other way when I go to a conference. Well at least it's all in one place compared with like JPM where you're running around the city. But San Diego does have a pretty big convention center. So give yourself a little bit of breathing room there. I know. I think I can get away with just my flats like my this year. Or or for this conference too. JPM it's like running shoes or nothing else. Yeah, no, I would recommend that pretty much for all conferences. But yes, JPM, it's an absolute must. You had that fun story where you had to stop by a shoe store this past year. Yes, and they did not have any shoes in my size, so I had to s trudge on in my horrible shoes. Wait. What? They didn't have any shoes in your size? Okay, for those for those of us who are just learning about this, I am six feet tall, which, you know, like co you know, coordinates with very large or correlates with very large feet, so It's it's a journey. I I don't get to pick shoes. Yeah, I've always wor I've worn flats at conferences for the past probably ten years. You know, before that I I was still in my phase where I was like, I want to wear pumps and heels. And yeah, no, no more. Nope. Comfort is king. And I have like two pairs of shoes that I've been wearing probably for like five years worth of conferences, and they're at the end of their life. So like I have to find new conference shoes now. I'm gonna do have to do that before JPM next year. ⁓ I'm gonna miss bio this year. I I've been ⁓ every other year for the past few years, but that's that's all right. You guys will represent well. We will miss you though, Heather. We will. So it'll be yeah, it'll be me and Annalie out there this year and then we'll be joined by a couple of folks outside the editorial team at Biospace. So it should be a good time. Mm-hmm. Nice. We'll live by curiously through you. Absolutely. And we'll of course be checking in from San Diego next week. We'll send you guys over a clip to include in next week's podcast, but For today, ⁓ let's dive in. Heather, you want to start us off with introductions? Sure. I am Heather McKenzie, Senior Editor at Biospace, and I am in charge of Clinica Space, which hits your inboxes every Monday morning. Thank you. And Annalie? Hi, I'm Annalie Armstrong. I am senior editor for Biospace, and I do the weekly Biofarm Executive newsletter, which comes out on Wednesdays. And last but not least, Gabby. I'm Gabrielle Mason, senior editor with Biospace, and I'm in charge of putting together our daily gene pool newsletter and our weekly manufacturing brief, which comes out on Tuesdays. And once again, I'm Jeff Axt, managing editor. So let's get started. First up on our agenda for today is Moderna, ever a popular biotech since the pandemic days when it got to be known for its mRNA-based COVID vaccine. The news today revolves around Moderna's mRNA flu vaccine. So this first made headlines back in February. At that time, the FDA's Vinay Prasad personally issued a refuse to file letter. So basically that means he did the FDA declined to even review Moderna's application. And so this quickly rose up the ranks. So FDA Commissioner Marty McCary at the time was called up to Trump's office. And then just days later, the FDA reversed course and did allow the application to proceed. They ended up setting a decision date for August. But before that decision comes down, the FDA has decided to hold an adcom. And that's going to be happening tomorrow, Thursday, June 18th. So we now have those initial documents from the FDA out about that meeting. Annalie, what can you tell us? Yeah, this marks a bit of a return to adcoms. ⁓ we've had a couple being scheduled after kind of a pretty long absent absence. So there was a lot of drama as as you kind of Talked about Jeff between the FDA and Moderna earlier this year. And it seems that the ⁓ two parties have finally reached an agreement. The FDA still kind of has some concerns about Moderna's use of a standard dose comparator vaccine in its trials. So typically in older populations, they receive a high dose flu shot ⁓ every season just because they are more ⁓ immunocompromised, have different health concerns. But Moderna actually used a standard dose in their trials. So that was kind of At the heart of the FDA's concerns. So to kind of get the regulatory filing back on track, Moderna asked for a two-pronged approval process. So one approval would be for people aged 50 to 64 years. And then they're seeking an accelerated approval for approval for people who are over 65. So the FDA seems to be okay with that now, according to these documents. Moderna has promised to do a confirmatory phase four trial to generate more data in that older population. So yeah, it seems like they've kind of come together. The FDA is ⁓ in agreement. They are asking for some clarifications from this adcom, particularly around some gaps in efficacy data, but seems like Moderna's maybe on the path to approval now. Yeah, seems like potentially good news for a b biotech that's been pretty battered recently. Yeah, definitely. We all know Moderna from the COVID-19 pandemic, ⁓ but they've been really struggling to successfully, you know, validate their second act, I guess you could say. ⁓ and this regulatory blockade from the FDA was was ⁓ a pretty tough one for them to swallow. They have a pretty ambitious goal of launching like or of having like seven medicines approved by 2028. And they also had been hoping to break even financially ⁓ by 2028. So this flu vaccine represented about a one billion dollar revenue opportunity for them. So when the FDA stepped in and and said, no, we're not even gonna look at the application, that was a big deal for Moderna. You know, I f I feel like this is just a sense I'm getting, but I feel like the there's more alignment during the past month ⁓ with the with the FDA, like companies coming out and saying, We're now aligned. ⁓ you know, DIS medicine was one of those ones last week. We'll talk more about that in a few minutes. But are you guys getting the same kind of sense? Yeah, absolutely. And it like it I don't know. It seems crazy that that would be happening because we've done a lot of reporting, obviously, that there is a real leadership void at the FDA right now. There are no permanent leaders in these really key agencies at the FDA. And yet we're starting to hear a little bit of a softening of the FDA's stance kind of across the board. So it's pretty crazy ⁓ that that's happening and and Moderna seems to be benefiting. It actually kind of makes sense to me. I remember one of the comments from some of the analysts when McCary first left and all those leaders along with him and Kyle Diamantes, the head of food, stepped up as acting commissioner. One of the comments at the time was that maybe things will go more smoothly. Kyle Diamantes isn't doesn't have those scientific and medical credentials to maybe feel prepared or qualified to step in and make overriding decisions over the reviewers, which I think that was a lot of the big concern under McCary was that there was a lot of leadership interference with the normal FDA review team. So maybe a lack of leadership. is better than bad leadership, ⁓ if if you want to put it that way. Like if the if ⁓ the the former leadership couldn't agree and couldn't get on the same page with their staffers, that caused a lot of the turmoil and their absence maybe does kind of smooth the path for some of these companies. Yeah, he's staying in his lane, which is, you know, and letting some of these career staffers make some of these decisions. It's also reaching out to stakeholders too, like they had that rare disease meeting a couple of weeks ago that a lot of the patient group seemed seemed happy about. And we'll have to see The proof is in the pudding, right? We'll have to see what happens with some of these potential approvals. But but yeah, he's getting kind of from what I've read on on LinkedIn, he's getting kind of rave reviews from a lot of pretty senior people. Yeah. No, that's really good to hear. Obviously we still want stable, steady leadership at the FDA, but for now, I'm glad to hear that things are going, you know, as smoothly as can be possible, lacking that leadership. So we've got a lot more to talk about with the FDA. We'll get back to that a little bit later in the show. But moving on to the markets right now, we have to talk about IPOs. It's just been a really exciting time. There was a pretty long dry spell in the middle of last year, a good six months where we didn't see any IPOs. Then we slowly started to see them, you know, start to tick up. And this past month has seen not one but two record-setting public debuts. So that's pretty exciting. First, there was chilera therapeutics. It hit the market in April with 625 million. And that outpaced Moderna's 2018 IPO, which had been the record holder with the its 600 million, speaking of Moderna. But now another one, Kylero, was quickly dethroned by Parabolas Medicines, which ⁓ came out last week with 670 million. So these obviously still pair in comparison. The other big IPO we saw last week was SpaceX with 85 billion in its IPO. So like a whole different ⁓ ballgame, a few extra zeros there, but still pretty exciting times for biotech. Annaly, what does all this activity stay about the say to you about the state of the public markets right now? Right. Yeah. The the SpaceX or what's happening in the tech space space? SpaceX, OpenAI, ⁓ what's the other one? Anthropic. Like it's just absolutely crazy. The markets are going crazy for these IPOs. So I talked to a bunch of experts about these kind of record breakers in biotech. They absolute they say this is that this is absolutely being a positive thing for the industry. It's great to see more of these. And Critically, we're not seeing too many IPOs because if you look back in 2021 and kind of right after, we had so many biotechs that were going public that really didn't have a lot of data to show for them. We did an an analysis a few months back just looking at what happened to some of these companies. And a lot of them still don't have pipelines, which kind of suggests that it maybe wasn't the right thing to push some of these companies public with little to show. But that's really crucial to Parabolis and Kylira. This is what multiple people pointed out to me is that these are companies that have Pretty late stage pipelines and a lot of force behind them. Kylira is a kind of this new co-China partnership ⁓ company. They took a bunch of assets that had already been trialed in China, which allowed them to bring them over and move them pretty quickly into the clinic. And Parabolis was a little bit slower. They've been around for a few years, previously known as Fog Pharma. But again, they have some really some assets that are pretty far along in the clinic. So the experts that I spoke to said that. We're kind of unlu like we shouldn't really expect every new biotech IPO to top what these two companies have achieved. It would really take a pretty similar company that has some de-risked assets, a lot of validation behind them to really pull another record breaker. Same kind of story we're seeing in the MA space right now, too. It's really I think we have exact quote saying this, but a story about the have and the have nots, right? We are seeing an uptick in deal making. We are seeing more IPOs, but it's like you said, there's not a ton of companies, it's just a small handful of de-risk companies with, you know, clinical assets that are getting big bucks and kind of drawing the news. Yeah. And what's really critical with these IPOs and MA deals is that that money's going to get recycled. So when we talk about the haves and the have nots, we're seeing the haves maybe pushing some money back to help some of the have nots get there. So this is really great to see. We always need a a boost to early stage research in this industry. So We can't really talk about IPOs without talking about that that earlier ⁓ I guess link. So it's great to see. I hope that the IPOs continue at a good pace. Not too many. We're kind of at the like the ⁓ what is it? Sorry, I'm trying to think of a phrase. Sweet spot? Yeah, sweet spot. Yeah. So we're kind of at like the sweet spot for IPOs, it seems. That's what the the experts told me, and hopefully that will continue. Yeah, absolutely. Well, unfortunately, now we have to pivot to a little bit less positive news. So while IPOs and MAs may be ticking up, so are biopharma layoffs, unfortunately, still. So there are reasons for this. Big pharma is facing some pretty serious patent cliffs right now. The numbers I've seen are something like up to a 300 billion in revenue that could be lost by 2030, with something like 70 blockbuster drugs that are slated to lose market protections in the next few years. Layer on top of that, the macro headwinds that we're talking about ad nauseum on this show. High interest rates, constrained capital, plus the regulatory uncertainty we were just discussing. It's still pretty tough times for biopharma, and that's definitely reflected in biospace numbers, showing that layoffs are really still battering the industry. This is in large part because pharma trying to protect their bottom lines from these patent cliffs by restructuring major sweeping restructuring initiatives by several big pharma that are wiping out thousands of staff salaries. Yeah, so two of those big pharma's ⁓ in May were Takeda and Beyond Tech. So May layoffs as a total hit nearly 7,000. The number of employees laid off spiked by almost 50% year over year compared to last May, but that jump is mostly due to significant cuts at those two companies, Takeda and Beyond Tech. So those companies alone announced layoff plans for a combined six thousand three hundred and sixty staffers. ⁓ while the total number of layoffs announced in May was six thousand nine hundred and fifty six. So making up the vast majority of those numbers. And then when you look at just the year as a whole, May kind of took the cake for monthly layoff count, with the previous high being back in February at three thousand seven hundred and thirteen jobs. That adds up fast though. That's over ten thousand jobs this just this year. Yeah, and those are just just those two months. More recently, that has been really making headlines was Genentech. They made a big change, kind of laying off an undisclosed number of workers, but including several longtime vets, including Vishva Dixit, who served as VP and senior fellow of physiological chemistry and research. So he was with Genentech since nineteen ninety seven. One of the originals at one of the original biotechs. That's gotta be a hard way to end your career or at least end your stint at Gigentech. Well, thanks for that summary. ⁓ a little bit of a downer that that was. Let's pivot back to some p more positive news. ⁓ we can't get through a whole podcast without checking in on our favorite deal maker, Eli Lilly. The pharma, of course, continued its deal making spree last week with the another deal. This one was with Al Zacure. They struck a licensing agreement worth up to a billion dollars centered on a small molecule asset for Alzheimer's disease. Lily, of course, already has their anti amyloid antibody, Kisuna, but they're obviously trying to stack up and narrow a little bit more with all that GLP one money they keep throwing back into the industry. But interestingly, I would say the bigger Lily news this in the past week was actually some clinical results showing a promise for a previous acquisition that the pharma had made, that of Ajax Therapeutics back in April. Yeah, no, and this is obviously keeping with the trend of everything coming up. Lily, the the company got a quick return on investment ⁓ from this deal. It's new JAC inhibitor, especially. And that was at the the European Hematology Association or EHA meeting this weekend. Lily pr reported positive early data from the drug in myelfibrosis, which is a a chronic blood cancer. BMO Capital Markets actually said the data, while early, appear highly competitive. We actually just did an analysis last week that showed that Lily's MA deals so far this year account for pretty much more than half of all capacity spent by the top ten big pharmas, which is just absolutely crazy. I don't think it's gonna slow down anytime soon. They still seem to be hungry for more deals. Yeah, it's pretty astonishing. So yeah, definitely check out that analysis and the Ajax news was was very interesting. We'll continue to watch out for more results from all of Lily's deal making and of course more deals coming our way. But pivoting now, I did promise that we'd get back to the FDA. There's one other issue or program that we want to talk about with the FDA, and that's the Commissioner's National Priority Vouchers Program. So early this month, the agency held a public listening session to discuss the program. It's been pretty controversial from the jump. It was first announced last year by Fanai Prasad and Marty McCary. And they've handed out a number of vouchers, but more of what the discussion has been around are the questions than the lack of transparency that surrounds this program. And interestingly, at that public listening meeting last week, or I'm sorry, earlier this month, it was actually at the beginning of June that that meeting happened, several patient industry and trade groups called for a pause to the program, again, citing those concerns about transparency and also political involvement. One of the companies that is have has been particularly worried about the political interference was Sonofi. They had a drug that received a a voucher called TZIALD. And the reviewers had seemingly been in support of ⁓ an extended approval for T Sealed, but Cedar Director or former acting Cedar Director Tracy Beth Hogue reportedly disagreed with the FDA reviewers. And when all that news became public, Sonofi actually requested to withdraw the drug. It's a diabetes drug. from the program entirely. So we covered all that news. It's not clear to us whether that withdrawal from the program happened. But what did happen was a T sealed approval. Yeah. So this is one of the more one of the more interesting cases that that's happened so far in CMPV. But yeah, so T Sealed, the application for older kids and teenagers was one of the first national priority vouchers awarded. But in their announcement of the approval last week, Santa Fe said nothing about it being a ⁓ CMPB approval. And the FDA didn't say anything about a voucher in its own announcement, which was posted on Monday. And they usually really talk these up. It usually comes with a ⁓ you know, celebratory quote from from Marty McCary, or I guess now ⁓ maybe from Tyle Diamantis or I I've seen the other ⁓ division division leaders, temporary division leaders be quoted in press releases. But yeah, nothing here. Yeah, I'm I'm really curious what happened there. I should have said that Tracy Beth Hogue, the one who reportedly interfered with the initial FDA review, was out last month as part of the big shakeup following Marty McCarry's departure. So very unclear how that happened, but good news for Sanofi getting that approval. Other than Sanofi, the agency has distributed more than 20 other vouchers. Several of these have been approved. That includes Eli Lilly's GLP One Pill, Foundado. As well as the hearing loss gene therapy from Regeneron that made huge news a couple months ago or so. But there was one notable rejection from a voucher holder, and that was a rare disease drug from Disc Medicine. Analysts at the time attributed the rejection to interference from Vinay Prasad. And he, as again, one of the many who has left in the last couple of months. And so with him out, just like with Hogue out and Sanofi having good news, Disc had some good news for us this week. Yeah, they did. And and this is just what I was talking about earlier about companies seeming to be aligning now with the FDA. But the DISC announced that the FDA will allow its current phase three trial to support another regulatory filing for the drug is called Bidapertin. So Bitapurten was rejected in February and experts have have made it clear that this that a CNFV does not, of course, guarantee approval. It it only guarantees a much faster review. But anyway, ⁓ BM BMO said that they while they had expected this news ⁓ about the the phase three trial being enough to support approval, it nevertheless provides, quote, more confidence in the regulatory path for Bid of Purtain. Definitely good to see. Hoping, as we talked about earlier at the top of the show, that, you know, with some of the leadership shake up, the regulatory path will be clear for some of these companies that have seemingly gotten the runaround. So another one that we'll be watching closely. And then finally to wrap up our show, we have one more actually FDA item here. I said that last one was was the other program, but there's one more. And I don't know if you all remember, but just a month or so ago, President Donald Trump signed an executive order. Actually, it was back in late April. Man, time flies. President Trump signed an executive order supporting the psychedelic space. Specifically, this relates to the CMPB program, the voucher program, because three psychedelics makers received the priority vouchers following the president's executive order. Those went to Compass Pathways, the USona Institute, and Transcend Therapeutics. They all have psychedelic drugs in late stage development. But there was a little piece of this executive order that we didn't give as much focus to initially. The president also encouraged access to Ibogaine or Ibogaine derivatives. This is a pretty controversial psychedelic. That to this point has not been tested thoroughly, and there's not a ton of of of research on it, especially in the US. But the president's executive order did result in an FDA green light to begin human testing of a metabolite of ibogaine called nor ibogaine. So again, ibogaine is a plant-derived compound that's pretty notorious for its safety risk. It's been associated with some deaths. It's a pretty severe trip. And the most interesting part to me is that this whole push for ibogaine or ibogaine derivatives seemed to stem from a text conversation between the president and podcaster and UFC commentator Joe Rogan. So Rogan was turned on to the potential benefits of ibogaine in treating addiction and other mental health issues from a friend involved in the Texas Ibogaine initiative. He had a few folks on his podcast, and he Pretty much got excited about Ibogaine and texted the president. And the president said, You want FDA approval? Let's do it. And not long after that, we have the IND for a noribogaine clinical trial. And just to note that Joe Rogan was at the White House for the announcement of this executive order. So there's definitely ⁓ you know, a relationship there that I think is playing in. All of that's just to say I got very curious of what do we know about Ibogaine and its derivatives and what can we expect out of these clinical trials. So Gabby, you took a deep dive into this for us. Yeah, so there's definitely so much to unpack as we all kind of quickly learned. There was so much information that I actually ended up writing two different stories, one breaking down how the drug might potentially work, and the other kind of exploring the that more regulatory side that you mentioned and the path ahead. Something that really presents a huge challenge for just drug development is that there isn't a definitive understanding yet of how IBogaine works. It's a psychedelic, it's a hallucinogen, and it's touted for its, you know, kind of ability to completely eradicate opioid or alcohol dependence, but it also carries really significant cardiotoxicity risks that have have led to death, as you mentioned. So to kind of just understand the latest research behind how classic Ibagame works, I did speak with Dollybor Summit, he's a researcher and a professor at Columbia University. And he's been studying Ibogaine for decades. And so most recently, he and a team of colleagues, you know, they hypothesized that the drug works using what he calls matrix pharmacology. So the way that he explains it, you know, Ipogaine acts across different molecules and cells impacting serotonin signaling via A broader transporter network rather than acting on a single target. So this makes the drug a lot less potent, which makes it harder to kind of separate the safety risks from the therapeutic effects. So that's why, you know, drug developers are likely moving forward with analog analogs of the drug to hopefully maintain Ibigaine's therapeutic effects, but really bring down those dangerous side effects. So Same's himself, he's the co-founder of Gilgamesh, and they have a preclinical derivative of the drug that holds potential in substance use disorders, PTSD, and traumatic brain injury. So still very early on in the field for, you know, Ibogaine and its derivatives, but we'll definitely be following the space. ⁓ and it's super interesting. For sure. Yeah. Thanks for that recap. Obviously, psychedelics have been a lot of there's been a lot of discussion around them. You know, going back a couple of years, we had the Lycos rejection, but then we've seen a lot of movement back in that space. I think there's real promise for these drugs in treating mental health, but there are the understandable concerns both with safety risks and how do you manage with, you know, additional therapy needed and monitoring for these psychedelics. So again, one more watch. ⁓ thank you so much for listening and we'll be back with you next week.