Latest / BioSpace / Genmab and AbbVie’s Epkinly win, a rush of deals, Sarepta’s turnaround hopes, more
Transcript
- Jef: Hello and welcome to Biospaces the Weekly. I'm your host, Jeff Axt. Today we'll talk about some new clinical data for GenMAB and Abby's biospecific antibody, Epkinley. These results impressed analysts and could set up the partners for an expanded approval into frontline treatment of diffuse large B cell lymphoma. We'll also recap the deals of the past week, which have been plentiful and included Novartis's nearly $8 billion bet on mRNA drugs with China's Abigin Biosciences. Then we'll have an update for you on Sereptotherapeutics. That is the embattled biotech we talked a lot about over the course of last year. It is not out of the woods just yet, but things do seem to be trending in the right direction for the biotech. And finally, we'll look back at where the money flowed in the third quarter of this year as we enter the home stretch of 2026. Let's get started. we are in the fourth quarter. How you guys doing? Heather: I feel very fourth quarterish. I gabby: that's crazy. And how does that feel, Heather? Jef: Yeah. Heather: feel tired, although that could have gabby: Mm-hmm. Heather: been partly due to my Spain class last night. We have we have Jef: Nice. Heather: the instructor, but gabby: Bed tire. Heather: she's like you know, yeah, she's she's legit. gabby: Doesn't mess around. Jef: Spin classes are legit. They're hard. Well hopefully that cold snap will wake us up a little bit. It was nice and brisk this morning. I'm very excited for that part of the fourth quarter. Heather: We had frost on the rooftops. Jef: Okay, it's here. you Canadians get that long snow on the ground for months at a time winter. We don't get that. How about you, Gabby? Do you have snow on the ground for months at a time? gabby: yes. I I would say Jef: Okay. gabby: my weather is probably much more akin to Heather's weather and Annalie's than yours. Jef: I yeah, you're you're pretty far up north there. I guess the other thing to touch on we forgot already is baseball. Heather, I know you're a huge baseball fan. I'm a postseason fan and a Braves fan, so I'm watching at the moment, and man, is it stressful. Yes. Heather: Go Braves, although they didn't do too well a couple of days ago, but they're playing again tonight. my main focus is that the Braves need to beat the evil empire, which is the LA Dodgers. Yes, I'm still bitter. Jef: I mean, I think everyone is. I mean managers are wanting salary caps, salar players are not wanting salary caps. Will we even have baseball next year? I don't know. Heather: Yeah, you look at the you look at the salary caps and it's like the Dodgers, four hundred and fifty million or something. Jef: Mm-hmm. Heather: And then the Jays are like in fifth place and and they're like nearly three hundred three hundred billion, million, million. so it's yeah, it's it's just a big big jump from the Dodgers to anybody else, pretty much. Jef: It really is. But I can understand also why the players don't want those salary caps. I don't know. I don't know what the right decision is here. I'm not an expert on sports management. Heather: The right decision is anything that gets baseball back to us in March. gabby: recently learned that baseball was six months long, like a season, that seems incredibly too long. Heather: Very long. Yeah. Jef: It's a long season and they play so much. It's part of why regular season baseball kind of bores me. Cause you know, I grew up watching football and every single football game matters so much. You know, if you lose a game, it could end your season. Baseball doesn't really matter until you get to the postseason. And then it's really exciting. Heather: Kind of like that long game though. gabby: So are we in the posts? Heather: It's like a team like the Jays can have no chance at the beginning of the year last year and then end up winning the most games in the American League. You know, it's like yeah, it's a long game. gabby: a season is six months and then there's a postseason. Heather: Yes. April, May, June, July, August, September. Yes. Yeah, Jef: Yeah, it goes I World Series is usually November, so yeah, we're Heather: November. November first, twenty twenty five is a very sad day. Jef: I'm sorry. gabby: All I know is Wisconsin sports have been hitting it out of the park. Apparently, the Brewers had a crazy comeback game this weekend. And then I was at the Badgers game and we did win that one. And then the Packers also won, apparently. So great, Jef: You guys are on a roll. gabby: weekend to be at the bandwagon Wisconsin. Just kidding. I'll always be a Wisconsin Heather: come on. Jef: Ha ha ha. gabby: fan, Heather: I'm also going for the Brewers because they're a smaller market team and I I like the smaller market teams. Like Cleveland has the lowest market. I could give a monologue on all of this if you guys Jef: Okay. We should have to have a special edition, a special episode all about baseball. gabby: That will be Yeah. Jef: Well, I guess we should focus back on biopharma for our listeners. so before we dive into our first topic, let's get started with introductions. Heather? Heather: I am Heather Mackenzie, senior editor here at Biospace. I curate Clinica Space, which hits your inboxes every Monday. Jef: Thank you. And Gabby? gabby: And I'm Gabrielle Mason and I put together our daily gene pool newsletter and then our weekly manufacturing news brief, which goes out every Tuesday. Jef: And I'm Jeff Axt, managing editor. And I'll also put in a plug for our weekly biofarm executive email, should say. That's the one that senior editor Annalee Armstrong usually puts out. She is actually on her way to Virginia right now. So I'm hoping to actually meet up with her tomorrow. It'd be great to see her in person. She's heading down for a little fun trip with her family. But for now, we will carry on the show without her. First up, let's talk about some exciting data put out by Jen Mab and Abby this week. So this is for their bi-specific. Antibody Epkinley, this drug was first approved back in May 2023 for patients with diffuse large B cell lymphoma, but who had already undergone at least two prior rounds of systemic treatment. So the new data speak to the drug's potential use in frontline treatment, where it would go up against Roche's poly. Analysts are pretty bullish on the data that just was put out and on that showdown becoming a reality. Heather: Yeah, definitely. analysts are are saying that Ep Kinley set a new standard of care in this indication. so the the biospecific plus RCHOP, which is a five drug regimen based on Roche's checksumab, led to a 51% reduction in the risk of disease progression or death. analysts actually used a lot of superlatives to describe these data. William Blair said the readout. quote, knocks it out of the park to get back to baseball. And truist said that they see the results as highly differentiated. So not only did the result hit the study's primary endpoint, it greatly exceeded analyst expectations of about a 30 to 35%. Jef: Yeah, that was a lot. Heather: Yeah, and in in contrast, Poly V was approved in first line lymphoma in April twenty twenty three and it hit twenty-seven percent reduction of risk of disease, progression, relapse, or death in its phase three trial. Jef: Yep. And then there's also insides Monjuvi. I guess that one hit twenty five percent progression free survival in a phase three earlier this year. Heather: Yeah, and and these trials hit their primary endpoints with those numbers, which is just an indication of how stunning Upkin Lee's PFS data are. Truist did kind of couch their excitement by saying that the full data, including durability, overall survival, and safety are important to determine absolute benefit. that phase three study is expected to wrap up in June next year. Jef: Okay, perfect. We'll definitely be looking for that one. Definitely good news for this space. Shifting our attention now to the business side of biopharma, this past week saw a slew of deals that we thought were worth covering. First, there was a noteworthy acquisition to add to 2026's growing total, and that was for intrabio. So just weeks after securing expanded approval for their rare disease drug, Agnursa, Intrabio exited with a $2 billion buyout from Japan Shinogi. So the drug was first cleared by the FDA back in September 24 for Neiman Pick disease type C. But like I said, just a couple of weeks ago, it got an expanded approval and is now the first ever treatment For ataxia to langiaxia. I'm probably saying that wrong, but this is a fatal disease that involves the progressive degeneration of the cerebellum. So pretty devastating brain disease there. So Shinogi obviously saw the value in this drug. Jeffries analyst predicted global peak sales of about 1 billion. So that was an exciting acquisition. But like I said at the top of the show, the biggest deal of the week was Novartis's new partnership with China-based Abigin Biosciences. Heather: Yeah, this is a pretty big deal, especially in terms of biobucks, at a potential 7.8 billion. So the deal centers around an mRNA drug candidate that is designed to deplete disease-causing B cells. According to Abigin, this this in vivo approach could lower the risk of cytokine release syndrome, which is a common and often very severe safety issue with T cell engagers. Now the companies didn't list any targets in their press release, but they did say that the candidate has potential to transform the treatment landscape for patients with autoimmune diseases. Autoimmune diseases was mentioned a few times in in the press release. Jef: Yeah, definitely really interesting to see. And to see an in vivo approach to T cell engagers is really exciting. We've we've heard more about car Ts going in this direction, but to be able to just give that mRNA code to the patient and not have to extract the cells, manipulate them in the lab, reinfuse them, it definitely simplifies the administration and supply process. So this is a big one, like you said, potential $7.8 billion total there. The upfront payment is also noteworthy. It's seven five hundred and seventy-five million. So That's a that's a chunk of change just for the upfront payment, obviously still way less than the total in bio bucks. The buyer is also noteworthy. So Novartis has made RNA therapies a strategic focus, and that's really a valid this decision of theirs to buy into Abigen is really a validation of the biotech's platform. Heather: Yeah, and an OER spokesperson actually told us on Monday that the company was attracted to Abigin specifically because of its differentiated proprietary RNA platform and and then the work they've done with mRNA coded encoded T cell engagers. Jef: Yeah, really interesting stuff. Definitely one to watch still early, but this is the kind of stuff that I love to watch. The the new modalities just right from the start. So in another five years on this podcast, we'll be talking about all the approvals in this space and all the momentum, I'm sure. Okay, so a couple other deals to touch on before we move on. We did see Eli Lilly expand its partnership with Gate Bioscience. This is an alliance that the big farmer first struck last year. Committing up to 856 million at that point. They're now basically doubling down with another potential 870 million. These two companies are collaborating on a new small molecule drug class that they're calling Molecular Gates. This is discovered with Gates Molecular Gate discovery engine. So not surprising, the name is not too surprising there. But basically, the goal is to eliminate certain extracellular proteins, ones that have been historically hard to target and to treat diseases with high unmet need. And separately, we got word this week that CSL is fronting 355 million and putting up to 1.2 billion in bio bucks on the line to develop a drug with a lentis therapeutics for progressive diseases that affect the kidney and liver. And then there was one more that we're gonna dive into in a bit more detail, and that's Sonofi and Regeneron. So obviously longtime partners, but this is another partnership continuation, and they are set on coming up with the next dupixant, which is their blockbuster INI drug that. Top the list, well, not top the list, but was among the top of the list last year. It was actually the fourth best-selling drug of twenty twenty five, bringing in nearly eighteen billion. gabby: Yeah, so they've they've been partners for decades and Sanofi has renewed that alliance with a one billion dollar upfront payment in efforts to kind of develop a successor to their blockbuster Dupixon, you just mentioned like a 18 billion. I can't even fathom that amount of money. but so Regeneron will have the chance to collect up to seven billion more in BioBucks in this new deal. as the two work together on four next gen antibodies from Regeneron's pipeline for immune-mediated conditions. So beyond that large upfront billion-dollar payment, the deal is a little surprising given the pair's past relationship, which BMO capital market analysts characterize as sometimes contentious. just last year, so extremely recently, Regeneron actually sued Sonofi. claiming that the partner didn't provide full access to material information about DuPixent sales. So in the same announcement about the New Deal that just came out, the pair shared that they had settled this issue. I guess this dispute is no longer. a key US patent for DuPixent is set to expire in 2031. So this new pipeline collaboration really positions both companies for continued leadership in the development of next gen therapeutics for type two inflammation driven disease, and that's according to BMO. Jef: Yeah, I love how they had a whole legal dispute of like, we're all good now, we'll just throw another several billion at them. Water under the bridge. Yeah. Heather: I can make up a makeup. gabby: That's biopharma for ya. Jef: Yeah. They have they have a chance to make a lot more money if they stay together, I guess. They they follow the money. gabby: The patent cliff proved larger. Jef: Yes, exactly. Heather: What? Jef: All right, so next up is a story we've sort of been waiting for, and it could have gone one way or the other. fortunately for Serepta, things are looking up, at least according to Jeffries analysts. So rewind to the summer of last year. we talked a lot about the major and very public saga that Serepta faced over its Deshayne muscular dystrophy gene therapy, Elevitus. Multiple deaths were associated with the viral vector that underlies the treatment, led to two deaths in patients who had taken elevitus and one who had taken an investigational gene therapy and a limb girdle muscular dystrophy trial. And there were it also the the vector technology also underlay some of Sarepta's other pipeline candidates. So a lot of fallout from that whole saga. The FDA had requested that. The company paused shipments of Elevitus, which it did agree to for about a week. all shipments were paused and then they resumed for only the ambulatory population. The FDA did revoke the therapy's approval in the non-ambulatory population In addition, the the company has pivoted away from its gene therapy focus. So obviously Alevitus is still there and there are some ongoing trials, but The company at that time said that they were hoping to right the ship by putting more of their focus in other modalities. But like I said, it could have gone one way or the other. We've been watching this company for a long time and the stock has not rebounded. It's really been hovering around $20 or less per share, which is a fraction of where the company sat at its heyday, which it topped in 2024, it topped $150 per share. So it's been a big question mark: can Surreptors survive this? What will be the outcome? Recently, Jeffries analysts did say that Serepta could be a potential turnaround story. They cited specifically recovering elevator sales and a promising pipeline. Heather: That's right. Yeah, I really like this note. I I like their optimism. But Jef: Mm. Heather: but yeah, so in terms of Elevatus, last week at the World Muscle Society's annual meeting in Japan, which is a really cool location, by the way, we should we should go to more conferences there. Jef: Yeah, maybe next year Heather. Heather: the the gene therapy it showed durable and clinically meaningful benefits in older patients with DMD. And the com and Serepta noted that between eight and twelve years old, that's when boys with DMD generally start to really decline. So it's terrific news. but but as you said, Jeff, the new data aren't the only thing behind Jeffrey's prediction. so the analyst also said that Serepta is expecting phase one, two readouts in myotonic dystrophy type one and FSHD, which is another type of muscular dystrophy, I'm not even gonna try to pronounce. Jef: Yeah, well done not trying now. I always try and then I butcher them. Those Heather: Yeah, Jef: assets you were just talking about, Heather, those were from Serepta's Airhead partnership with the S I R and A platform? Heather: Yes, and and Jeffrey said that both of them could generate more than one billion in sales if they're successful. so that could be a a pretty big part of this turnaround story. and then coming up early next year, the FDA will decide whether to grant traditional approval for Serepta's Exxon skippers for DMD. Amanda's 45 and Beyond is 53. I always think of them as the Sarepta twins. but both unfortunately failed to improve motor function in a confirmatory trial last year. and Serepta is banking on real world and safety data to get these over the traditional approval line. So if they do secure that traditional approval, Jeffrey said it could help to stabilize the DMD franchise. Jef: really curious to see what's gonna happen with these. when do you know when we expect to hear? Heather: Yeah, so the FDA has set a target action date of February twenty eighth, for both therapies. ironically that's gonna be a rare disease day. Yeah. Jef: interesting. Maybe not so coincidental, I don't know. All right. Well, with that's the wrapped update, we will now move on to Annalise sort of bailiwick here in BioFarm Executive. I'll do my best to fill in for her and kind of tell you what she's got lined up for this week. obviously, as I said at the top of the show, we are now in the fourth quarter, which means third quarter is in the rear view, and we'd like to take a look back and see what was happening in the quarter in terms of where the money was flowing. We did notice that the pace of MA's slowed in Q3. There were a total of 22 buys in the quarter as compared to 31 in Q2. But Q3 did see one of the largest transactions when Vertex bought Krymetics in July for about 10 billion. Vertex is not a big buyer, so that was actually a really interesting one. It pushes the statistic fibrocious drug maker into an entirely new territory, the specialty endocrinology market. So I'm eager to see what Vertex does with this. Annalise's full MA breakdown is on the site now, so check that out. Meanwhile, our staff writer Tristan Manilak took a deep dive into the biggest VC raises of the quarter. And these incl included some really sizable rounds. So experts told Biospace the money is definitely flowing, but still the story remains that capital is constrained and investors continue to look for those later stage and more de-risked assets. So good news, bad news situation there, I guess, in terms of how the capital is flowing, but check out that full analysis on Biospace as well. And then while we're talking about VC raises, I will add that we did already kick off the fourth quarter with a very interesting raise. We saw Varda Space Industries raise two hundred and fifty one million to manufacture medicines in space for patients on Earth. So I've been hearing about manufactured in space for a long time. I think we're still years out, but really exciting to see a good chunk of money going toward making that a reality. Okay, outside of the quarterly trends, we also have a new feature to share in Biofarm Executive this week. Gabby kicked off a new column for Biospace taking a look at who's hot and who's not. Tell us about it, Gabby. gabby: Yeah, so I wanted to really provide timely, frequent kind of insight into both the biotech and pharma worlds of kind of big catalysts and you know, sentiment from analysts and investors about certain companies. Just everything moves so quickly in the space. I wanted to, you know, try to ground us a little bit. so I took a look at some of the most significant stock changes in the industry this last month, so for September. and Viking Therapeutics came out as one of the top stock gainers with significant catalysts in the month, while Langeberon experienced some of the largest stock loss in September. so Viking stock popped 30% on its injectable GLP1 and GIP dual agonist maintenance data, which truest analysts deemed the best case scenario. so a day after that data drop. Viking did propose a common stock and convertible notes offering, which ultimately did bring down their stock a little bit at the end of the month. And then there was Elon Gebron whose stock plummeted more than 60% after a phase 2B trial. And that tested its cell stem cell therapy out in a rare heart disorder. So the asset failed to improve blood flow for patients' hearts, and the company is now kind of reviewing all options. and their stock loss was sustained and by the end of the month their shares were worth two dollars and sixty-nine cents apiece. Jef: yeah, that was a huge drop and definitely a rough time for that company. We'll see how how their review pans out and what comes next from them. gabby: Yeah. And then, you know, shifting from biotech to pharma, I spoke with Evan Siegerman at BMO, and he shared that they're most bullish on Merck's patent cliff pipeline strategy and most bearish on BMSs right now, of the large pharma companies that they cover. So almost all big pharmas, you know, face patent cliffs. We were just talking about Sonofi Regenerons, Dupixant. Except Eli Lilly, they actually are not facing any major patent clips right now. But Merck and BMS both face some of the greatest exposure. so it's interesting that these were the two that they were bullish and most bearish on. both of them for their PD one blockbusters, Keytruda for Merck and Opdevo for BMS. so Siegermann thinks that Merck has, you know, done a better job overall putting large D-risk growth products in place before some of that patent erosion really begins, as compared to BMS. So he cited Merck's mRNA cancer vaccine, which it shares with Moderna. and that's seen, you know, recent phase three success. That was pretty lauded throughout the industry. And then they also have the already approved WinRevare, that's for high blood pressure in the lungs. And then they have a TROP2 antibody drug conjugate called SAC TMT that Merc licensed from Kalen Biotech. meanwhile, BMS kind of has a little less of a gap to fill with up Devo than Keytruda, but they actually also face another patent loss of a top-selling drug, which is the Pfizer partnered. blood thinner, Eloquis. So the products that BMS has kind of set up as potential revenue replacements for those two are much riskier than Merck has lined up. and that's what Siegerman told me. So one of those products that BMS has lined up is Cobemphi, that's a schizophrenia drug picked up from Coruna, which hasn't been selling as well as anticipated. And then we're currently waiting on data for the drug in Alzheimer's psychosis, which is also pretty risky. They also have the J and J partnered Milvexian for atrial fibrillation and a separate large or and a separate late stage pulmonary fibrosis candidate. but Siegerman, you know, believes that there's just so much writing on these assets. there's truly so much at stake, which then is just, you know, leads BMO to be more bearish on the plan in general. So that's kind of the the look at what's up, what's down in the space this month. Jef: Thank you. I find these features incredibly helpful. Obviously, we know bits and pieces, but to put it all together and take the holistic look at it, it is really valuable. So check those out. And finally, just a final note here, Biospace did on Tuesday launch its annual 40 under 40 list. So we like to highlight young biopharma leaders who demonstrate industry's values in terms of innovation, impact, and commitment to improving outcomes for patients. So we had A ton of names to look through and we came up with 40 really powerful folks who seem to be doing some cool stuff. So check that out. And as always, don't forget to subscribe to all the newsletters we spoke about at the top and like and subscribe to this podcast as well. Thank you so much for listening and we'll see you next week.