Latest / BioSpace / FDA hears input on psychedelics, PDUFA, lung cancer’s next-gen therapies boost survival
Transcript
- Jef: Hello and welcome to Biospaces the Weekly. The FDA has been busy this week. They held public meetings on psychedelic therapies as well as PADUFA 8. The agency has also formally announced the launch of the application process for the IND pilot program that it's running as part of the HHS-wide initiative known as Operation Trial Blazer, aimed at reshoring early clinical trials. So we'll discuss all of that with you. Meanwhile, the World Conference on Lung Cancer just wrapped up in Seoul, South Korea, with several companies reporting positive survival data for their next generation therapies. And we'll also check in on Novartis after its terrible, no good, very bad week last week. and we will discuss Novo's rebrand as the Danish Pharma continues working to right the ship after plummeting from its high in mid-2024. Let's get started. Hi everyone, how y'all doing today? Heather: Good. It's a beautiful September day. Annalee Armstrong: I am so happy that it is fall. I I don't for those of you in the United States, I'm sure it's still summer there, but in Canada it is Jef: It is Heather: Mm. Nicht. gabby: Same. Annalee Armstrong: fall and it is glorious. Jef: We Heather: It was Jef: actually have our first fall, good fall weather this morning too. It broke overnight, woke up to a crisp morning. I've actually I closed my window here so you guys can have that great sound quality audio for studio quality audio for this podcast. But I've been working here all morning with the window open, which has just been it's made me so happy. Annalee Armstrong: Yeah, I'm like I'm bundled up in a hoodie, like my cold. I'm gonna make some Heather: Just beautiful. Jef: Ha ha ha. Annalee Armstrong: soup later. I'm so excited. Heather: I was in my my furry robe. I have this furry blue robe. It's it's so furry and g not not actual fur 'cause I'm an animal lover and that would not be Annalee Armstrong: Mm-hmm. Heather: okay. but anyway, I was I was bundled up in that last night after I got home from the gym and which might have been 'cause I was cold or my muscles were just really tired. Jef: Good workout. gabby: This a hundred percent is my absolute favorite weather, except for today here it is raining. So a little Heather: Mm, mm. gabby: less, but in general fall weather is my favorite. Jef: I completely agree. I think I've told you all summer is my least favorite season by a mile and fall is my most favorite season by a mile. So like this is this transition is just it it doesn't it doesn't get any better than this. I'm always ready for the change of seasons, but summer to fall is by far the very best. Heather: In a nutshell, biospace loves fall. Jef: Yes. That's our intro for today. Annalee Armstrong: Yeah. Pumpkin spice everything. Jef: All right, well let's get into our show for the meat of the episode that you all wanna hear. Let's start off though with some introductions. Annalie, why don't you get us started today? Annalee Armstrong: Yeah, I'm Annalie Armstrong. I am senior editor here at Biospace, and I do the weekly Biofarm Executive newsletter, which goes to your inboxes on Wednesdays. Jef: And Heather? Heather: And I am Heather Mackenzie. I am senior editor here at Biospace and I curate Clinica Space, which hits your inboxes on Monday morning. Jef: And last but not least, Gabby. gabby: And I'm Gabrielle Mason, senior editor with Biospace, and I curate our weekly manufacturing brief, which hits your inboxes on Tuesdays, and then our daily Gene Pool newsletter. Jef: And I am Jeff Ax, managing editor. I think I forgot to say that at the top of the show, so definitely need to introduce myself today. Thank you all for joining us. All right, so as I said at the top, we're gonna start with the FDA this week. In addition to some of the news I teased in the intro, last week, shortly after recording this podcast, in fact, we learned from the FDA that it was making the acting directors of Seber and Cedar. Those are its two main drug and biologics review units. Along with the acting director of the Center for Tobacco Products, made all three of those men permanent in their positions. I can't really speak directly to Bet Koplau's work at the tobacco unit, but I have heard Kareem McKeel and Michael Davis speak at Bio at the International Convention in June, they spoke, and I I honestly was impressed. So just to recap a little bit, Mikhail and Davis have led Seber and Cedar since mid-May. They stepped into those roles on an acting basis. After former division heads Vinay Prasad and Tracy Beth Hogue left the agency. And as we know, we've discussed a lot here on this podcast. Prasad and Hogue, along with former Commissioner Marty McCary, were closely tied to several controversial regulatory decisions. The FDA staff is also just still reeling from the major cuts that happened in the spring of 2025. So at Bio, Mikhail and Davis both emphasized that their priority was on boosting morale, retaining the staff that they had. hiring more and also just generally returning to a sense of normalcy at the agency. And by all accounts, they were really succeeding in these efforts. So to me, it seems like a real net positive for the men to have been transitioned from acting to permanent directors of their divisions. and just for there to be more permanent leadership at the FDA, where this is has largely been lacking. Analysts at RBC Capital Markets agreed. They said that this move of creating these permanent leaders is likely to be seen as a sign of continuity and stability. That could help, should help ease concerns that the FDA will see major shifts in policy. Of course, we are still lacking a permanent commissioner. Kyle DiMontes still sits at top in an acting capacity. we are still awaiting the confirmation of Heidi Overton, who President Donald Trump nominated last month. So still missing a commissioner, pretty important role there, but definitely good to see a little bit more stable leadership at FDA. And with that, it was good timing. They launched right into a very busy week this week. So first up on Monday, the agency held a public hearing on my favorite up-and-coming modality, psychedelics. we've talked about the two front runners in this space, Compass Pathways and Athenium Therapeutics. Compass is expecting an approval as early as this year for its psilocybin-based therapy for treatment resistant depression. So we're closely watching that progress. so I can see why the FDA thought it was a good time to gather stakeholders to discuss how these drugs should be regulated. Heather, what can you tell us about what went down at the meeting? Heather: Yeah, so experts including physicians, psychiatrists, obviously type of physician, nurses and pharmaceutical leaders, they all called for the FDA to really define data and treatment standards ahead of the first psychedelic approval. there is there's a lot of excitement going into the meeting about the new therapeutic class and its potential to really help patients with depression and PTSD in a way that Current therapies really haven't been able to for a lot of patients. And, you know, some experts were called for the FDA to make further risk assessment and adverse event monitoring a part of any approval. You know, and this they noted was for for both for patients, but also for the for the sake of the class, so that it doesn't get off to a bad start because it has been so controversial. and there's th this enthusiasm also really matches recent White House and FDA support. Jef: Yeah, that's part of what we've been covering a lot for sure. So that that support really kicked off with Trump's executive order in April. That order instructed the FDA to leverage its new priority review program to accelerate psychedelics development. And so that then led to Commissioner National Priority vouchers for Compass as well as for Transcend Therapeutics and USona Institute. Interestingly, Uson Institute is where Davis, now newly permanent director of CEDAR, used to serve as CMO. So not surprisingly, Davis was at Monday's meeting. Definitely good to see continued support for psychedelics. I totally agree with some of the amazing efficacy results we've seen, as well as the need for a lot of caution in launching these therapies onto the market, given the, you know, very unique nature of these drugs. All right, well before we move on to the next part of the FDA's busy week, let's stay on psychedelics for a minute because one of those front runners I mentioned Compass, I also mentioned Daphneum Therapeutics, they had some more results for us this week. Heather: Yes, they did. also on Monday, the same day as the public meeting. Daphinium probably not, no. Jef: Probably not a coincidence, right? Heather: but Daphinium reported yeah, they reported more positive data for their LSD drug. And this was the second positive readout for the drug called DT one twenty in anxiety disorder, and the third overall this year, after it showed success in treating major depressive disorder in a June readout. So analysts said this latest data in anxiety now shift the narrative toward commercialization. So good news for Difinium. Jef: Yep, and they're we're you know, they don't have a filing in yet what the FDA like Compass does, but we know we're expecting something as early as next year for sure. Heather: Yeah, I think I read one analyst said that it they're expecting an approval by late twenty twenty seven. Jef: what will be really interesting and exciting to watch is how they roll out, how these launches happen if and when these drugs are approved, because that's gonna be the real sticking point here. I think one of the we say it in our story, one of the people in the meeting said, you know, the medicine's just the beginning. You that that starts the ball rolling, but there's all these other pieces that need to fall into place to actually help patients. So we'll be watching that one closely. But for now, moving on from psychedelics, but sticking with the FDA. Like I said, it was a busy week for the agency. They are having another public meeting today, Wednesday, as you're listening to this. This one will hear what industry stakeholders want to see included in the latest iteration of PADUFA. That's the Prescription Drug User Fee Act. This is a legislative agreement that's been in place for many years at this point, but it does need to be renewed every five years. And so this iteration, PADUFA eight, will set the FDA's priorities for twenty twenty eight through twenty thirty two. Heather: Yes, so based on some of the early comments and other rumblings, the discussion today is expected to focus on rare disease reform, CMC risk and the reshoring of US trials, particularly early trials. Jef: maybe not coincidental timing, it may explain why the FDA on Tuesday, the day before this hearing, chose to formally open up the application process for the IND pilot program that's a part of that HHS-wide initiative initiative known as Operation Trial Blazer. So this initiative, like you just said, Heather, it's designed to expedite first in human trials in the US in hopes of bringing more early stage clinical research back home. In the past several years, as we know, China has taken over as the lead country for our all trials, especially early stage. Heather: Right. And another issue that that could be discussed at at today's PADUFA meeting is an FDA proposal that would offer a 50% reduction in the new drug application fee for investigational products with early stage trials conducted in the US. That could mean a savings for companies of roughly $2.3 million. so that's according to Jeremy Levin. he's the CEO of Ovid Therapeutics, who recently shared his thoughts in an opinion piece published in Biospace last week. Jef: Yeah, I spoke to Jeremy after this was first announced, this potential discount for if if a company ran early stage trials in the US. But Levin really questioned whether this financial incentive would tip the scales because it wouldn't benefit companies until they file that NDA, which is obviously late in the drug development process when the asset is is basically at the FDA's doorstep. So is a couple million dollars at that point enough to convince companies to spend more money and take the additional time it does take to launch those early stage trials in the US? Years prior. Levin argued that the more valuable commodity is probably time. And he suggested that Padufa should address how efficiently investigators can kick off first in human study. And that's exactly what Operation Trial Blazor is aimed at with this new IND program we just discussed. But he's advocating for some levers to be pulled in the PADUFA language as well to help improve efficiency at this early stage. All right. Well that was a lot, but that's what the FDA's got going on this week. Moving on now, we're gonna pivot to talk about the World Conference on Lung Cancer. So it started over the weekend and it ran through Tuesday and it yielded a number of interesting readouts that we thought were worth highlighting. So Gabby, take it away. gabby: Yeah, so several pharmaceutical biotechs shared data that showed overall survival benefits across, you know, various types of lung cancer. one of the most interesting readouts was from Beyond Tech and their partner on Co C4. The partner shared the first overall survival data from their phase three of GOTIS BART. not sure if I'm saying that right, but close enough for patients with metastatic squamous non small cell lung cancer. it one. Jef: in cancer both the drugs and the indications are just a complete mouthful. It's hard to get it all out. gabby: I know, I know. Annalie, can you tell me what specifically some of those findings were? Annalee Armstrong: Yeah, I'm gonna give it a shot here. I think it's gotistobart, is how I think I'm gonna say it. So we'll start gabby: That's great. Annalee Armstrong: there. so this is an anti-CTLA for monoclonal antibody. This drug was tied to a median over survival of 18.5 months, which is compared to the 10 month median for patients who just had chemotherapy. So that's a pretty significant improvement of eight months in terms of overall survival. gabby: Right. And and that kind of improvement there is confirming the drug's advantage over chemotherapy, is what analysts said, which is good since it's now being tested in the second part of the same phase three trial. So these results are from the first portion of the trial, which was designed to help find the proper dose to move forward with for the second part. and now there's an interim readout for that, expected sometime later this year. So we'll definitely keep our eye out for that. Summit and Akiso were also back with more data on Ivaneska Mab, which of course the industry has been watching very closely over the last couple of years. Yes, Jef: So often that we know how to say that one. gabby: yes, with no stumbling. Jef: Ha ha ha. gabby: What did we learn from this new readout, Annalie? Annalee Armstrong: Yeah, this is definitely one of those drugs where like every little teeny tiny scrap of data is just being like eaten up by investors and analysts. So the latest findings come from a phase three trial in PDL1 positive advanced non-small cell lung cancer. Summit Nikizo had already announced the primary results of this trial last year. But this time around, they were sharing details behind a secondary endpoint win. The new findings show a twenty-seven percent death risk reduction for patients on this drug compared to those who just received ketruda. gabby: And so analysts with BMO said that this, new data shows some additional class benefit for PDL1 VEGF bi-specifics. But they're still waiting on a global assessment of the data to get a fuller picture, a better understanding of the efficacy in non-Chinese populations. because the data from this trial was conducted solely in China. so we're still waiting on that as well. Other big news came from GSK and partner Hanso Pharma. Their phase three trial included patients in China with relapse small cell lung cancer that had progressed after first line therapy. Heather, what did those two partners reveal at WCLC? Heather: Yeah, so GSK and Hanzo are developing RISRES, which is actually really easy to say. It's an antibody drug conjugate targeting B7H3. Now RISRES elicited a median overall survival of 18.5 months, reducing death risk by 54% when compared to standard of care. The partners previously said that the ADC had met the main goal of the study. but hadn't provided any details. so now they're able to truly understand the benefit, which is quite significant. gabby: And Rizrez is the the nickname for the drug, which I won't bother pronouncing the whole name, but we do love and appreciate easy to pronounce nicknames from drug makers. Jef: Riz Riz is Heather: That's why has a nickname. Jef: great. I love it. Annalee Armstrong: Use it as the marketing name, honestly. gabby: It's fun to say too. Yeah, yeah. Jef: Yeah. Heather: Yeah, if if they can choose, that's a great name. Jef: Save us the headache of that transition when the drug gets approved and branded, then we gotta learn a whole new name, which might be just as hard to pronounce sometimes. gabby: Biospaces campaign for Riz Res. Jef: Ha ha ha. gabby: But anyways, there were a few, other really interesting readouts too that I kind of broke down in my article. So definitely check that out if you're interested in learning more. A lot going on in the lung cancer space. Jef: All right, yes, seconded, definitely check out that article. but for now we will move on to the topic that was kind of the big the big headliner last week, which was Novartis's string of trial fails and other speed bumps. So just to recap briefly, last week a main asset from Novartis's $12 billion acquisition of Avidity failed a phase three trial in myotonic dystrophy type one. And then also an investigational anti-sense therapy did not show significant benefit benefit on cardiovascular risk and its own late stage trial. And both of those trial fails followed the pause of phase two trials for a CAR T cell asset after three patient deaths. So it was, like I said, a terrible, no good, very bad week for Novartis. Analy, what can you tell us about the broader impacts of all this? Annalee Armstrong: So this flurry of pretty bad news did have a very big, meaningful impact on Novartis. Their shares fell about 13%, but this really spread across the pharma industry. The top six pharma companies by market cap also saw stock declines on this news. this was to a lesser extent, but it was still very jarring to see these declines across the board. Even Eli Lilly, which is worth roughly $8 trillion, depending on the moment you look at their shares. they were down a little bit as the rest of them declined. So Amgen saw the steepest fall because they have an asset that rivals one of the ones that failed from Novartis. So this was just like a pretty tough week for all pharmas and it really stemmed from what was going on at Novartis, which is pretty interesting. Jef: Why do you think it was so impactful? Annalee Armstrong: Yeah, so phase three failures do happen, of course. I wouldn't call them rare, but bigger companies really tend to have these things pretty locked in by the time they get to phase three. We like it it's it's unusual to see so many things fail at once at that late of a stage. So we had two trial fails, you know, a program that was kind of in trouble with the cell therapies and that kind of thing happening at Novartis. So I think it was just all of these things stacked on top of each other that. Kind of made everybody think, whoa, like what is going on in phase three across pharma is not always set in stone. Jef: Right. And then of course Novartis hit the hardest with all the news coming directly from from their end. So the company's board is actually facing scrutiny over their oversight of these deals that Novartis has conducted. Of course, I said one of those main phase three trial fails came from an asset that they spent twelve billion on as part of that Obidity Buy. Annalee Armstrong: Yeah, and we did an a a really good deep dive into what exactly is going on at Novartis, what might happen next, and kind of the overall leadership from CEO Vasenera Simhan. So you can check that out on Biospace. Jef: All right. And finally, one last thing to touch on before we sign off today, and that is Novo. And I do mean Novo, actually. I'm not just using that for shorthanded anymore, because this week the Danish Pharma has officially dropped its last name of Nordisk. So we will we only really use that on first mention anyway, and we call them Novo all the time, but now it's official. Annalee Armstrong: Yeah, this is like big small news, it almost feels like. Jef: Yeah. Annalee Armstrong: It's like a rebranding. you know, they're just dropping a name that very few people were using, anyways. but it does kind of signal a change in direction. so they they dropped Nordisk. They also switched their apus bull around. he's always been facing to the left and now he's facing to the right, which Novo said is facing the future. they also adopted a new culture, the Novo Way. and laid out some new pillars for their future. So investors welcomed it. Analysts were, you know, complimentary of the change, even if it seems small. and they kind of appreciate the shift towards like more simple branding and and a more forward looking culture. Jef: Yeah, I love the flip of the bowl. Like we're not looking to the past, not looking to the left, we're looking to the right. That says it all. I mean, I l a little bit of sarcasm there. It seems like such a subtle thing, but I do I do applaud the a attempt to, you know, kind of rebrand with a more positive forward looking culture. Annalee Armstrong: Yeah, I know. And like the other thing is like the bull is pretty iconic. Like I'm happy that that they didn't like, you know, go like very boring with their label. There's been some marketing changes recently that have kind of stripped all the flair out of some of these major pharma's brandings. so happy to see the bull stayed. but yeah, like you know, branding is one thing, but analysts were kind of hoping for a bigger change. and by that they mean across the business. They may get that. But later this month, Novo is holding a Capital Markets Day, and analysts hope that this new branding is also going to come with a completely new business strategy from the new CEO, Mazir, Mike Doostar, who will of course be presenting at that meeting. Jef: All right. Yeah, that meeting going to be held on September twenty first. So we will definitely be keeping an eye on it and see if Novo can write that ship. Like I said, they really peaked in mid twenty twenty four and it's kind of been a steady downhill slide since then. So investors and analysts are are looking for more. All right. And to end our show, as we like to do, what's coming up in Biofarm Executive this week, Gannelly? Annalee Armstrong: Yeah, we've had a really busy week with, of course, Novo, no Novartis, all of this stuff. But we also have a really great look at a scrappy group of biotechs that are shunning big pharma partnerships to commercialize their own drugs. I really love small companies that embark on marketing their own drugs alone. So this this is a fun feature. We take a closer look at Denali Bridge Bio and some more who are doing this right now. Jef: Awesome. Well definitely check that out. That should hit your inboxes this morning. If it didn't, don't forget to subscribe, top right corner of Biospace. And as always, don't forget to subscribe to this podcast on all of your favorite podcast channels. Thanks for listening. We'll see you next week.