Latest / BioSpace / Amylyx’s redemption story, Summit’s survival woes, Definium’s anxiety win, more
Transcript
- Jef: Hello, and welcome to Biospaces the Weekly. I'm your host, Jeff XT. Today we've got a range of industry updates for you from some much needed good news for Amyx and Dhinium's positive readout for anxiety. That's for its LSD-based psychedelic therapy. And then we've got some not so great news, some concerns about Ivaneskimab survival benefits and a spate of deaths among patients treated with neurocrines approved Prater Willie drug. Plus, we'll end the show with a little bit of a discussion about Eli Lilly and how it is cracking down on the black market for its next generation weight loss asset retatrotype. Let's get started. Hi guys, how you doing? Heather: Hey Jef: We finally have all four of us. gabby: I d Jef: It's good to see you all together again. Annalee Armstrong: I know, we're all back, the whole team. Heather: Hmm. Good to be here. Yes, birthday. Hmm. Hmm. Alright. Hmm. And then Gabby's out as well for Jef: Briefly, before Annalie leaves on her PCO, it is still the summer. gabby: Feels good. Annalee Armstrong: Yes, but I will be here for the podcast next week, so I'm just temporarily leaving. Jef: Excellent. Yeah, it's hard to gabby: I know you're coming back for the podcast. Yes. Heather: a week, I think, September. Hmm? Jef: It is just the tail end of all the summer gabby: Yes. Jef: vacations. I hope you had a great break last week, Heather. Heather: Hmm. I did. Yeah. It was it it was awesome to see them. it was my my best friend and her seven year old daughter came to visit and seven year olds are kind of hurricanes a little bit. you guys, Jef: Ha ha ha Heather: Jeff and Annalie obviously know this. but yeah, it was like yeah, they just the hurricane blew in and it and it blew out on Friday, and then I spent kind of the rest of the weekend sleeping. Yeah. Jef: Recovery. Annalee Armstrong: I I can confirm that a six year old is also a hurricane and a three year old. Yeah. Heather: Okay. Yeah, Jef: Yes, Heather: I I thought Jef: no. Heather: it was either six or seven, so Jef: I'm not sure at what age they stop becoming like that. Stop just terrorizing everyone in the house, but for the most part, it can be a fun hurricane. Heather: I mean I'm kind of a hurricane, so yeah. Eighteen, gabby: Eighteen, maybe? Jef: Eighteen. Still s right. There's still hurricanes just Heather: maybe thirty. Annalee Armstrong: As soon as they're out of your house. Jef: somewhere else bothering other people. gabby: Right, exactly. Heather: Do you guys read that study gabby: Off site. Heather: that that said that actually we don't gain full maturity until 33 or something like that? so maybe it's a little further. gabby: Promising for me. Jef: argue that we never stop maturing. I think we should always continue self-reflecting and growing Heather: Yeah, I like that too. Yes. Jef: and I don't think I was very mature at thirty three, so Heather: I don't think I was either to be honest. Annalee Armstrong: I'm I'm older than that and I still don't know if I've reached that. Jef: I will say those hurricanes grow you up real fast though. I didn't start having kids till my mid Heather: Mm-hmm. Jef: 30s and then I started finally growing up. They kind of push you to figure out all your own weaknesses and stop reacting to their I don't know. It stop you know, when you're the adult in the room, you start have to start acting like one. So that's what caused me to really make some strides in that direction, I think. Heather: Yeah. Well I'll keep working on it. Jef: All right, so let's get into our show. starting of course with intros. Heather, why don't you get us started this week? Heather: Sure. I am Heather Mackenzie, senior editor, and I am in charge of clinical space, which you guys all get on Monday mornings. Jef: Thank you. And Annalie? Annalee Armstrong: And I am Annalie Armstrong. I am senior editor and I do the Biofarm Executive Newsletter, which hits your inboxes every Wednesday morning. Jef: And last but not least, Gabby. gabby: And I'm Gabrielle Mason, senior editor, and I curate our daily Gene Pool newsletter and our manufacturing brief, which goes out every Tuesday. Jef: Fantastic. And once again, I'm Jeff Axt, managing editor. let's talk about Amyx and specifically its GLP one blocker that is showing some promise in treating low blood sugar in patients that have undergone weight loss surgery. So note that this mecha mechanism is the opposite of the traditional GLP ones that we talk about all the time. Those from Novo and from Lily, those activate rather than block the GLP receptor. And the point there is to bring down blood sugar. This is why we help use it to help treat diabetes and also to help patients lose weight. AmyLix's acid, on the other hand, is meant to prevent an exaggerated insulin response that can crop up after bariatric surgery and cause dangerous drops in blood sugar. So this week the company released some phase three data suggesting that the drug is effective. Heather: yes, they did. I, you know, I I really like this one. It is kind of a real redemption story for for Anux. for those who may not remember, a little over two years ago, Anux actually pulled its ALS drug, which had been approved the previous year, from the market after it failed to show efficacy in a phase three trial. so the company's co-CEOs, Josh Cohen and Justin Klee. Had actually made a promise to an advisory committee, which was actually a really rare second advisory committee in the first place, but that if this r unfortunate result happened, that that they would go ahead and and pull the drug from the market. So it appears that good things come to those who keep their promises and those who invest in GLP1s. So shortly after that setback in July 2024, Amyx acquired a vexatide from IGER Biopharmaceuticals. And today, Tuesday, it aced a phase three trial, as Jeff said. So in patients with PBH, avexatide led to a 55% reduction in the composite rate of level two and level three hypoglycemic events. so this actually beat the expectations of analysts like Mizuho, who said in a Monday note that the trial was powered to detect a 35% treatment effect. you know, I I attended a call this morning, Amylex's call to explain the results and and they, you know, that they really emphasized as well the p-value was like 0.00003. so it was very highly statistically significant. but yeah, so Anux, you know, Amyx has definitely got a a good drug to bring hopefully to the market next. but they've also insisted in previous interviews that they are still fully committed to ALS and other rare diseases. in fact, they're working on an earlier stage program in ALS as well as one for a rare neurodegenerative disorder called Wolfram syndrome. so they announced positive long-term data. from a phase two study this spring for that purpose. Jef: Yeah, this has been a really, really interesting one to follow. You've done a great job covering AMLIX over the years. And obvious obviously that ALS withdrawal was pretty devastating to the community and to the company. But yeah, you'd love to see a comeback story and we'll keep watching for those other readouts. But for now, some good news in that postbariatic hypoglycemia. All right, well, moving on now to Ivan Eskimab. This is Summit's a Kiso partnered VEGF PD1 bispecific antibody that's kind of been the star of this new modality since it beat Key Truda in a late-stage trial trial in China. This drug has since been approved in China for a few indications, but questions have continued to linger over a potential US approval as we kind of await more and more data from these global trials. Annalie, what's the latest on this one? Annalee Armstrong: Yeah, so Summit's path to the FDA has been getting more complicated with every data readout that happens. Last week, Ivan Eskimab was approved by Chinese regulators for squamous non-small cell lung cancer. This is its third indication in China. It has yet to be approved by the FDA and the US for anything. but this third approval in China was a really big win for Summit and their Chinese partner Akizo. However, Summit's role in this partnership is to replicate the benefits that they have been seeing in China in these trials that were initiated by Aquizo. when the Chinese regulators approved the drug, they published in the label some updated data that actually showed a degrading progression-free survival benefit in this population. So this has really raised a lot of concerns among investors that an upcoming phase three trial, which is called Harmony Three, it's potentially one of the most anticipated readouts of this year, analysts and investors are now worried that it may not show substantial benefit. Someone is trying to go for an accelerated approval for this asset. And without a positive or a strong positive readout from Harmony 3, they may not be able to get that early market access. Analysts think that this could push the launch back to 2029 instead of 2027. Jef: Oof, that that is quite a long delay. A couple of years there. Especially for a company. I you know, I know that Summit just reported in its second quarter earnings they had just six hundred and ninety million in cash available. That's enough, I think, to keep the lights on for less than a year, according to the company report. So they need a win here. Annalee Armstrong: Absolutely. And you know, it is important to note that they do have an FDA decision pending for Ivan Eskimab in a different indication in EGFR mut mutated non-small cell lung cancer. That's due in November. So if all goes well there, they could have at least one marketed indication in the US, which is of course the you know the largest market in the world, but I think every hit to the clinical program really cuts into the overall prospects for this drug. Jeff, you mentioned key truda. Analysts once called Ivan Eskimab a potential Keytruda killer. Every time they have a hit to these clinical programs, it really cuts down the overall prospects. Jef: Do we know when we're expecting that phase three readout? You said it's upcoming. The trial's ongoing, but the readout is upcoming. Do we know when we're expecting that? Annalee Armstrong: Yeah, it's supposed to be by the end of the year. And then there's more Jef: Okay. Annalee Armstrong: data to come in 2027, which will hopefully add to the this weight of evidence, but that's kind of the key one coming up. Jef: Okay, very good. Well, yep, that's one we'll keep covering for you. Definitely all eyes on Summit and Akiso with this new VEGF PD1 space taking off as the next generation of I.O. Again, with Key Truda going off patent. there's a lot in that space of next generation immuno oncology assets. So we'll keep an eye on that one. And now for my favorite topic of late, psychedelics. We've had the pleasure of talking about this one almost every week for the past month or two. So last week when we talked about it, we mentioned that we were awaiting a phase three readout from Definian, Definium, excuse me, for its LSD candidate in anxiety. Well, we didn't have to wait long. It was just hours after the podcast aired that the announcement came through and it was a good one. So that momentum in the psychedelic space continues. Gabby, fill us in on the latest details. gabby: Yeah, you're right, Jeff. It's been so busy in psychedelics lately. Definian's program is a single-dose oral LSD candidate that had already read out in a study for major depressive disorder. So most recently, though, the biotech notched another late-stage win for the asset, this time in generalized anxiety disorder. In a 12-week trial, Daphinium's candidate significantly lowered anxiety symptoms based on an industry scale, with a reduction rate that even came in slightly higher than what analysts had expected. So the efficacy was pretty rapid with changes starting as early as the second day. safety also looks pretty clean so the biotech is now moving into a 40 week extension for generalized anxiety and also is waiting for anxiety findings from a second late stage study designed a little differently than the one that just read out. and if that trial is successful, analysts believe Definium would likely file a new drug application with the FDA sometime in the first half of next year. Heather: Yeah, so that's that's really terrific news. we we actually put out a special edition of Clinica's phase yesterday, Monday, including a mini upcoming catalyst calendar. So first up later this quarter is that second a generalized anxiety readout from Definium. and then coming up in Q4, Eli Lilly has a phase two A readout for a tie Beckley, who they just acquired. For their BPL003 in treatment resistant depression, which is one of the hottest targets for psychedelics. And of course, there is also the possible FDA approval of COMPASS' own TRD drug or treatment resistant depression drug, which is under a rolling MDA right now. so lots of action coming up in psychedelics. Jef: Including that potential first to market, would love to see that. Heather: That would be huge. That would be huge for a long time. gabby: And it's going to have implications like all over the space. I just want to flag that or plug our Nick, our contract writer, Nick Paul Taylor, he wrote an excellent story kind of exploring the rise of psychedelics internationally and how it's prompting manufacturers to look to position themselves as like the go-to sources for the US once a drug hits the market here. So definitely, you know, check that out on our site under manufacturing. It led our manufacturing newsletter this week. So be sure to subscribe to that as well if you haven't already. Jef: Yeah, and speaking of subscriptions, if you did not get Clinica Space on Monday, the only way to see this special edition on Psychedelics is to subscribe. If you subscribe now, you will automatically get that special edition. And then, of course, all future Clinica Space emails. All right, moving on again now. Annalee Armstrong: Moving on again now. Jef: Unfortunately, too, we have some concerning news regarding Neurocrines approved Prater Willie drug, Vicat. There have been seven deaths among patients who received the treatment. While a causal link has not been determined, the news definitely cast a shadow on the therapy, which was just approved by the FDA last year. Annalee Armstrong: It was a really tough day for the Prater Willie community. you know, there were kind of two news events here. this disease causes the insatiable desire to eat, which can lead to extreme weight gain, cognitive disabilities, and behavioral changes. So it's just a really difficult disease. this patient community finally got a treatment last year when Vicat was approved. that asset. was later bought by Neurocreen. and that deal, I believe, closed earlier this year. So they are just kind of taking this one under their wing at this point. But now, unfortunately, there has been the report of 100 adverse events associated with VICAT, including seven deaths. As Jeff already mentioned, there hasn't been a causal link here, but the community was kind of warned by a group of doctors in the space. so the Prater Willie Syndrome Association was really quick to point out that this is a really difficult disease and patients tend to be at very high risk already. They urged the patient community to wait for a full investigation here and practice what they called informed prescribing. So being aware of those risks, having really thorough conversations with your doctor and so on. So it's not a time to really like freak out and and stop prescribing the drug, but we just need to know what the potential risks are. Neurocrean pretty similarly pointed out the difficult population here and the risks that they face. They also said in a statement that they are in close contact with the FDA about post-marketing surveillance of VICAT. So hopefully, we will have some more information coming out about what happened here and whether those deaths are definitively linked to this treatment or not, and potentially some risk mitigation strategies that could be put into place to help patients. but like I said, there were two news events here in Prater Willie last week. elsewhere, ARDVAR therapeutics terminated a phase three trial of a therapy called ARD 101 in this disease after they discovered some concerning cardiac issues earlier this year. So just kind of a really rough one-two punch for patients who really need some more options. Jef: Yeah, with so few options in the space, the this approved therapy. I hope we get some good news on the investigation there. Certainly good to advise caution, but you know, we don't like to see that pipeline thinning at the same time. All right. Well, last on our agenda today is a legal update from Eli Lilly on their next generation obes obesity drug, retatrotide. So we've talked a lot about this drug in terms of its you know, clinical readouts. It's one of the most anticipated up-and-coming Options in the weight loss space, so highly anticipated, in fact, that it has there have been reports of a small number number of patients being granted early access to the drug. But many, many more people are eager to try it. And this has created a bit of a black market around the drug. There's compounding pharmacies, medical spas, online sellers all advertising retatride for research purposes, but people are buying it and and taking it themselves. So now Lily is taking steps to stop that illegal sale. They have sued six US entities, but their attempt to stem the this black market is much broader than that. Lilly has referred more than 200 other players to U.S. authorities. They've reported 14,000 websites, social media posts, advertisements, online listings, things of that nature. These span you know, a hundred country countries for the illegal sale of Rachatride. The pharma has called the situation an urgent public health crisis and says it's aided by criminal networks in pretty strong language here. And beyond just those particular entities that that Lily has either sued or reported to US authorities, the pharma is also calling on, you know, all regulatory regulatory authorities, law enforcement, social media and commerce platforms, consumers themselves, doctors, credit card companies and other payment services, logistic companies. So really just casting the net as wide as possible. For people to do their part to stop enabling this black market and actively block the sale of Ratatride before it officially hits the market. So definitely interesting to see Lily trying to clean that up in advance of a potential approval. and that is a great segue to our BE corner this week, of Biofarm Executive. Annalee Armstrong: bioformat. Jef: We do have a bit of a a deep dive into the obesity space that touches on retatride, but I'll let you give the details, Annalie. Annalee Armstrong: Yeah, for sure. So our writer Tristan Manilak took a really great deep dive into the ongoing battle between Novo Nordisk and Eli Lilly in the oral GLP1 market. He really broke down this issue. essentially, Novo Nordisk is selling more of their oral Wigovi, whereas Eli Lilly is kind of playing catch up because they are entering this market with a molecule that consumers weren't as familiar with. So he really breaks down the market dynamics there. And of course, Retatro Tide makes a cameo because we can't talk about what's going on now without talking about the future. So make sure you check that out. we also have features on how Hong Kong is positioning itself amid China's rise in the biotechnology world. And finally, we have a look at the impact of Takeda's new narcolepsy approval. and how that is going to shape the sleep market to come. So a really jam-packed BE this week. So please subscribe at the top right corner of our website to BioFarm Executive to get all of that in your inbox. Jef: All right, well thank you all for the updates. It was a busy week. We will be back with you next week as always here on the weekly. And don't forget to subscribe to the podcast and all of your favorite podcast channels.