Latest / BioSpace / Q1 earnings take off, Lilly strikes deals, Regeneron notches historic approval, FDA raises questions
Transcript
- Jef Akst: Hello friends, you guys all geared up for earnings hell week? Heather: Hey, friends. Yes. Oh, it's tomorrow and Thursday. Thursday is even more hellish because we have later morning calls, which kind of gets into our newsletters, Jef Akst: You Annalee: You know I am! I'm ready! Yeah. Jef Akst: Which is exactly why I'm jetting off to Chicago. I wish you guys good luck. Annalee: Yeah, thanks. Thanks. honestly cannot think of a time when this many were concentrated over two days. Like, there's always one week that's particularly bad, but it's never I don't know, it's never been back to back like this. ⁓ Heather: Yeah, there's a lot. Jef Akst: nine in two days. at least nine that we flagged. i'm sure there are even others, but nine that we intend to try to cover in two days. yeah. Annalee: live, cover live. Heather: think we've split them up pretty well though. I'm looking and I'm like, nobody has more than one tomorrow or Thursday, right? So that's, or today or Thursday if you're listening to us on Wednesday. yeah. Yeah, you can't actually move that. ⁓ Annalee: Yeah. Yeah, I don't even- Jef Akst: I do feel bad peacing out on Thursday. That was not planned. If I had looked at the earnings schedule, I might have adjusted mine. Although I can't really. This is a trip for my parents 50th anniversary and Thursday is the day. So we will be celebrating. Yeah. Annalee: you Heather: ⁓ Chicago, I'm jealous. You're gonna have a lot of fun. going to Wrigley and you're gonna go to the Bean, right? ⁓ ⁓ that happens. That happens in Chicago. I to live there for those of you who don't know. And yeah, just when you think it's summer, nope. It goes Annalee: you Jef Akst: Yes we will. It is going to be like 45 and windy so it will be freezing but we will see a Cubs game. Heather: Well, get to our show. Let's get to intros. ⁓ Lee, do want to go first this time? Annalee: Yeah, I'm Annaleigh Armstrong, I'm senior editor and I do the weekly Biofarm executive newsletter which goes out on Wednesdays. Heather: am Heather McKenzie. I am senior editor and I co-alate Clinica Space, which hits your inboxes on Mondays. Jef Akst: And I'm Jeff Axt, Managing Editor, and as always, plugging the rest of our newsletters, we have our daily gene pool, as well as manufacturing brief on Tuesdays and Career Insider on Thursdays. Heather: All right, thank you. Well, let's get to it. As you're listening to this, the Biospace team is rapidly preparing for two busy days of Q1 earnings calls. We have Lilly and Regeneron and Amgen and BMS and Biogen and several more on deck. But Sanathy and Novartis have already kicked off this week. Annalee: Yeah, you know, it's funny is the only one that isn't reporting this week is like Pfizer. They're like randomly in May. I don't know how they worked out that schedule, but we're happy to dedicate some time for them on the first week of May. But yeah, so Sanofi was reporting last week. It was of a weird earnings call because one CEO Paul Hudson is gone. He left in February and they are waiting for their incoming ⁓ CEO, Belen who is starting on the 29th today, Wednesday, as you're listening to this. So during their earnings call, was kind of like, all of a sudden they started talking about the patent issues for Dupixent, which is their selling drug. It sold $4.9 billion for the quarter. None of their other drugs even hit a billion dollars. So this is literally their biggest product. Analysts were kind of taken aback. They were like, why are you all of a sudden talking about the patent issues with this drug? I know that one of our analysts faves, Steve Scala of TD Cowan wondered if maybe it was because of the departure of Paul Hudson. So Sanofi's interim leadership immediately insisted that was not the case. In fact, that had promised to start updating on the patent situation last year and he had said that he would start doing that this year. they said, nothing to see We're just talking about it. They did say that they are. going to rigorously defend all the patents on Dupixent, and at the same time, they're working with their partner Regeneron to find new medicines that will start to replace Dupixent as time goes on. Heather: Well, the new ⁓ and welcome, Belen, Sanofi, but she has lot of firepower available to her, doesn't she, Annalee? ⁓ Annalee: Yeah, no kidding. ⁓ There's definitely, know, there could be some deals. I think that's one of the big challenges besides this dupixin issue that she's going to have to solve. You know, Hudson, I think that he ushered in quite a few programs. There's been some real mixed clinical success. I think of the things that kind led to his downfall was ⁓ that Tala-Brutinib, which believe was for MS, right Heather? Yeah, that drug was... Heather: Yeah, sorry, I messed up. Annalee: it failed to get FDA approval. It's now been approved in the EU, but you know, the FDA, that's the market. The US market is really where companies want their drugs approved. So that was kind of the final challenge for him is that that drug did not get approved here. So Baylin will have to, you know, come up with a solution ⁓ and, know, point Sanofi in a new direction. So we a story in Biofarm executive this week that kind of goes over what she can expect as she comes in, what the transition is going to mean for Sanofi. ⁓ and what to expect based on her previous time at Germany's Merck. Jef Akst: Yeah, so speaking of patent defense, this is actually a great segue to the other company that just reported already Novartis. So Novartis actually suffered a 5 % drop in revenue in the first quarter, and that included a 46 % no dive for their blockbuster heart failure drug Entresto. executives attributed this to generic competition and another drug is at threat of generic competition is actually their radiopharmatherapy Lutathera. So the FDA granted a tentative approval for a radio equivalent from a company called Lanthias, but Novartis has really in and defended its patents for Lutathera. And why the FDA only gave quote unquote tentative approval. So basically the company makes this radio equivalent again is called Lanthias. They had to wait 30 months, so two and a half years. that's ⁓ stay, that 30 stay will expire in June. point this radio equivalent will hit the market and then Novartis might expect to see further decline in revenue. Although, like I said, executives were pretty confident CEO Vass Narisimhan was confident that this would affect the Lutatharist very minimally and that the company would continue to grow or rather I should say return to growth in the second half of this year since they did slide in the first quarter. Heather: We've got patent issues for Novartis and Sanofi and we will see what we have tomorrow for Lilly. Given that they're reporting tomorrow, we wanted to highlight that Lilly is on a real deal-making spree. Annalee: Yeah, no kidding. think we like basically this morning there was another deal from Lilly and we were like, ⁓ goodness, this is like every single day there's been a deal. I think there's been what like four in the past week and a half. It's just crazy. Heather: Yeah, why don't they announce the deals and, you know, or give us like embargoes for them and then we can set them up for every day. That would be really, really great, Annalee: I know, I know, we could plan ahead. this latest one was a 2.3, sorry, I mixed them up. See, there's been so many. They have signed a licensing collaboration with AI Biotech Pro Fluent, and they're gonna work on finding new DNA tools for new genetic medicines. Then the other one this week was the $2.3 billion acquisition of Ajax, which is developing treatments for rare blood cancers. Jef Akst: you Annalee: And then there was the ADC specialist Crossbridge then the deal in, woo, see, I'm getting this all mixed up. And then was also ⁓ Colonial Therapeutics, which is working on in vivo CAR-Ts. That kind of goes back to a deal they did earlier this year, which was also in the CAR-T space. So this is all complimentary, lots of cancer stuff here. So yeah, it's just been crazy keeping up with this. Jef Akst: Yeah, we've been talking. We've been talking so much about all the money they have from their GLP one franchise and it's keeping their BD team busy clearly. Heather: So, Annalee: Yeah, no kidding. And I wonder, like, if you think about it, we're a couple of months on from JP Morgan, maybe we're starting to get some of those deals that were, that they worked on there. So, you know, perhaps there's more to come. It seems like Lilly maybe I wouldn't say that Lilly's BD Firepower is like limitless because no company is but they do seem to have probably the biggest capacity for deals in this in the entire pharma industry. So I wrap all of this, I'm going to try, a story coming out today in a Biofarm executive, so you can check that out. Heather: Well, of Regeneron, Sanity's vaunted partner, they got a historic approval last week. So it's the first treatment. were waiting for this for a while. But ⁓ it's called OdorMENi, and is the first treatment target an underlying cause of hearing loss. Lots of firsts for this one. ⁓ Jef Akst: Yeah, no, we have been watching. I remember at ASGCT a couple of years ago, the first time that Anna Lee and I actually crossed paths, there was a big readout for this drug, for this gene therapy. And it was all the talk at ASGCT that year. I was ⁓ trying to track down the pronunciation for this new drug because we always struggle. I think it's our tarmini. A tarmini. I think that's what it's called. A tarmini. At least that's according to one video I found. I always struggle with new drug names. Heather: you Artarmini? Artarmini? Autarmini. Okay, Atarmini. Atarmini. Sorry. ⁓ Autarmini is also the first gene therapy, approved gene therapy for Regeneron. So that's another first. And it is the first to not cause any serious adverse events in a clinical trial. And that is according to one of the phase one, two studies investigators whom I spoke with. Jef Akst: Yeah, it really does seem all around like great news. I mean, the anecdotes of stories of these patients and in particular their parents, right? Because these are young children, but there was one, you know, of a mom who just clapped her hands behind her child and her child turned to see her. And then that just brought her to tears because that child, ⁓ I'm going to get all emotional now. It's really incredible to see these drugs really paying off. So I was really excited to see this. like you said, Heather, we've been waiting for it. Heather: Yeah. Jef Akst: was very interesting timing though, and that's because Regeneron also struck a deal with the White House on the very same day. And this is with regard to Trump's push for most favored nation drug pricing. if you recall last summer, Trump sent 17 letters to major biopharma companies requesting them to make these commitments to reduce drug prices. Regeneron was actually the last of those 17 companies to reach a deal. Heather: Hmm. Jef Akst: And again, they reached on the very same deal as this approval. There were reports of the MFN deal in the morning before the approval came down, but Regeneron didn't confirm with an official statement until after the approval came down. So I'm curious about why that timing worked out. But the point is that Regeneron now has an MFN deal with the White House. They have agreed to lower the prices of several of their drugs, including their cholesterol-lowering drug, Prowluit. I didn't practice that one. these will be offered on the government's direct-to-consumer platform, TrumpRx. And then when it comes to Otarmany, this was very interesting. We did find this with the release of the approval. Otarmany, this new gene therapy for a certain type of deafness, is going to be free in the United States. So that's pretty exciting. Heather: I asked them that question. I did a free interview for this and I asked them, have you determined the price for this drug yet? And they had said, no, we're still working on it. So it's funny. I wonder if they knew even back then, but it makes some sort of sense because there are only about 50 US patients for this drug. And it's always seemed to be more of a proof of concept or a platform prover to me. Annalee: Right, and this is less like about Regeneron having a major product. This is more about saying, listen, these gene therapies work, we can come up, we can make them for conditions like this that are maybe a little more common. I mean, this particular form of deafness has a small population, but this is really big for gene therapy, the modality in general, rather than it is like a big market play for Regeneron. Jef Akst: I hear what you all are saying, but I'll just remind our listeners that most gene therapies on the market cost millions. And this is a real problem with making gene therapy a sustainable modality. Yes, they can save lives. Yes, they can, you know, restore hearing these amazing things, but to this point, they've not been widely available in part because of that price tag. So it is really exciting to see them offering this one for free. Heather: Yeah. Annalee: Right. And I'd love to know more about how Regeneron is doing that. how, you know, is this something that the government is covering? That's not what I understood from this announcement. It's just, they are just offering this particular therapy. So it'll be interesting to see, like, is Regeneron going to lose money on this? it, you so we have to kind of follow up on that. Heather: You Thank you. Yeah? Well, Regeneron, I think we're going to find out soon because Regeneron is reporting tomorrow their Q1 earnings. And if we don't find out, at least a lot of analysts are going to be asking the question. Well, as always, the FDA has been busy this week. They have been busy, for one, dealing with a drug that was acquired by Amgen. Annalee: Yeah, so this has been kind of an ongoing battle. The FDA on Tuesday stepped up its call for Amgen to remove the autoimmune therapy, Tavneos, from the market. agency has questioned the integrity of the evidence that supported the drug's approval five years ago. This drug was originally developed by Chemocentrics, which was acquired by Amgen in 2022 for $3.7 billion. So this, of course, all of this evidence generation would have occurred before that deal, but it's Amgen's problem to deal with now. So the FDA is saying that unblinded study personnel have manipulated results of the pivotal clinical trial that was actually used to support the approval of this drug to make it look more effective. The FDA has not provided any new information besides what had been made public previously in their request this week asking Amgen withdraw the There previously some concerns of liver toxicity raised with eight deaths reported in patients who had taken Tevneos. Amgen, however, remains confident in the safety of this drug and they said they're going to reevaluate their next steps. Jef Akst: Yeah, I mean, they straight up refused to pull the drug from the market the first time the FDA asked. So what can the agency do if they continue to refuse to pull that drug? Annalee: Yeah, this is a really rare situation actually. FDA can absolutely force a company to withdraw a drug. They just don't typically have to do that. Usually the case of a safety issue, the company and the FDA will just kind of come together and say, all right, let's withdraw this or the company will just withdraw it ahead of time. The most recent example I can think of is Pfizer withdrawing a blood disorder drug. I ⁓ think it last year. They just did on their own accord. So the FDA can formally request a recall or a withdrawal. If the company declines, they can take the matter to court. ⁓ Jef Akst: or they can change their minds as is the case of Alevitis last year. We went through this with Surepta and there was a lot of back and forth. And we had these same discussions about what could the FDA do if, that wasn't totally pulling the drug from the market, but halting shipments and Surepta refused to do that. ⁓ we were looking at what can the FDA do in that point? But then the FDA just changed its mind. So I'm very curious to see how this plays out, not only with the drugstates on the market, but how FDA handles it. Annalee: Yeah, and Amgen could just submit some more information. They seem to be very confident that they have the data to support this drug. So maybe they just need to repackage that and submit that to the FDA. Heather: Well, psychedelics also to be in the news. there last week there were three commissioners national priority vouchers granted to three unnamed companies. So they weren't named by the FDA. How neither were the products. However, ⁓ one company, Compass ⁓ come up with a press a couple of hours later announcing that they were one of the recipients ⁓ for Comp360, that's their lead compound. It's a psilocybin-based candidate for depression. That's what they got the CNPV for. And it later reported by Psychedelic Alpha, that's a publication that ⁓ focuses on therapies, that the other two given to Otsuka ⁓ ⁓ Transcend Therapeutics. So owns now for PTSD. ⁓ and USONA Institute for major depressive disorder. Jef Akst: And actually that just confirms what you had already figured out, Heather. So like you said, the FDA didn't name these companies, but they gave us enough information in that release that you just did a little bit of and found they named the specific drugs that were receiving the and the indications. And psychedelics is in a huge space. So it does raise the question of ⁓ why the FDA not just name them? And not only did they not name them in the release, but of course we immediately reached out and asking for more information and they declined to comment on the record. Heather: Yeah. Yeah, well, let me, that's nice of you, Jeff, but let me give you some credit. While I was busy typing away on this article, you were helping me to figure that out, the companies and the indications. So, ⁓ yeah, yeah. But I mean, we all wondered and we have sought answers to this question, why the would choose to keep these company names private? Jef Akst: I love a little sleuthing. Annalee: It's a team effort here. Heather: I mean, they announced them most of the companies, think at least 17 of the 18 first CMPB awardees. So why not name these companies like they did for the first two rounds, especially as the agency continues to preach radical transparency. Jef Akst: Well, mean, as your reporting showed, Heather, there's a lot of concern about psychedelics are being pushed forward in this way. And given CNPBs, I think there may be some concerns about that politicization of the FDA and maybe somehow not naming the companies as trying to avoid of that chatter. But obviously you got plenty of comments in your article about how this is kind of concerning the way this is playing out. The administration should not be the ones choosing which drugs were accelerating. That should squarely come from the FDA. Heather: Hmm. Jef Akst: Nevertheless, the FDA was basically given this directive. And there's some weirdness with Joe Rogan. Read the whole article, how that goes, it dies into an ibogaine. There's a lot of weirdness with this one, a lot of kind of nuances. So yeah, I don't have an answer for you, but a lot of questions. Heather: I'm really happy. Yeah, it's a fun article and we will continue to seek answers to these questions we go along here. But Compass has no concerns. They were happy to say that they were one of the companies and yeah, so we'll see. ⁓ Jef Akst: Yeah, they were the only one I think that we kind of predicted ahead of time. were a few, you know, we knew these CNPVs were coming based on announcement earlier in the week. And there was of course a lot of discussion of who might they be. And Compass was one of those companies. There were a couple others though that were not ultimately those recipients. So Compass was a shoe in, I guess. Heather: Yeah, there was to Phenium, is, which is working on LSD for generalized anxiety and a couple of other disorders. a lot of people thought they were a shoe in, but they didn't get one, at least not yet. ⁓ Because, know, we don't think this is the end. was reading ⁓ one, ⁓ one note this week that said that this isn't the end. are going to be more. So, you know, to Phenium, Lycos could still have a chance there. Well, So it is the end of April. ends tomorrow ⁓ we were promised a new Sea Bird Chief by the end of the month. So we're all keeping a close eye on that. So Banai is going to be leaving after his one year leave absence from his professor position at the end of this month. And yeah, we're all, everyone's conjecturing and wondering ⁓ who is it going to But we published an interesting opinion in Clinica Space this week where Joshua Bansback, and he's a longtime healthcare communications strategist, he argues that Prasad's departure this week is only the beginning of what needs to happen for the rare disease space to trust the FDA again. So the new head, whomever they are, they need to repair a trust that's been broken by recent disruptions and ⁓ the late reversals that we've talked about quite a bit on this show. That's what Vanspec hard use. Jef Akst: Yeah, he makes a strong argument there. To me, I keep thinking of these parallels with the CDC as well, right? Like we've got a new CDC lead and then there's the same question. Yes, she seems like a reasonable voice to be entering the agency, but there has been so much trust lost. Is one person, one new person, even the right person enough to restore that trust and move the industry forward? Heather: Mm. ⁓ Yeah, I think that's going to take some time. Well, moving on, Anna Lee, what do you have for us this week in BioPharma Executive? Annalee: I like I've talked a lot about my stories far, but there's more, don't worry. So we have a couple of stories coming ⁓ from tour in Salt Lake City. In particular, got to stop by Altitude Labs, which is a biotech accelerator that was started by Recursion, which is a tech bio AI focused drug discovery company. So I got meet some of the scientists who are creating the next generation of biotechs. So you can read about their stories. And then I crossed the street, literally, this is all happening right in the downtown core of Salt Lake City. I visited Recursion's All Hands meeting where they heard from a patient with a disease that they are targeting with one of their programs. So you can check out both those stories in BioPharm Executive this week and on at Biospace. Heather: Thanks, Annali. Well, that is it for us this week. We will be back with you next week, next month, when maybe we'll be talking about a new biologics head. hello. Welcome to the by biospace. I'm your host, Heather McKenzie. Q1 earnings are here in full force with Novartis and Sanofi reporting already and Eli Lilly, as well as many others still to come. Regeneron's Otarmany was approved last week. marking the first targeted treatment for hearing loss just ahead of the company signing an MFN deal with the White House. Elsewhere, psychedelic therapies continue to draw headlines, with the FDA granting national priority review vouchers to three developers who it declined to name. And as April comes to a close, everyone's asking the same question. Who will replace Vinay Prasad as head of CBER? Let's get to it.