[intro music]
Intro clip:
Renée Aguiar Lucander: “We actually were the first company globally that was allowed to run this pivotal trial with this design, this two-step design, with using a surrogate marker, which in this case was reduction of proteinuria, which actually could lead to then an accelerated approval, and then follow that up with longer-term data to then achieve full approval based on more kind of accepted clinical endpoints, such as kidney function, which is what we achieved in March of 2023. … So, really, what we’ve seen is that many, many companies are now pursuing development programs in the renal area, which is fantastic, right? I mean, this is really… for patients, this is great.”
Intro v/o: Ideas to Innovation. From Clarivate.
Neville Hobson
In 2024, the pharma industry stands at a pivotal juncture, brimming with the promise of groundbreaking drugs that have the potential to revolutionize treatments for ailments like kidney disease, breast cancer, Crohn’s disease and more. With the possibility for standout commercial as well as clinical success, these potential blockbuster drugs are not just scientific achievements. They represent beacons of hope for patients worldwide.
However, as we explore these therapeutic advancements, we also confront the complex tapestry of challenges that the life sciences sector faces. External factors such as government initiatives aimed at curtailing healthcare costs, the persistently high cost of capital, and global geopolitical tensions are reshaping the landscape. These dynamics have a profound impact on investor sentiment towards the pharma sector, leading to a cautious approach. Nevertheless, innovation is rife in this sector.
Clear evidence of this is the 13 new to market therapeutics and drugs poised to launch this year. In its Drugs to Watch 2024 report, Clarivate predicts that each of these could achieve blockbuster status in the next five years, deliver game-changing benefits to patients, and fuel the next generation of medical breakthroughs.
Welcome to Ideas to Innovation, a podcast from Clarivate with conversations that explore how innovation spurs people and organizations to think forward and achieve their full potential in areas such as science, business, academia, technology, sport, and more. I’m Neville Hobson.
In this episode, we’ll take a close look at one of these drugs to watch, which promises to revolutionize the treatment of a specific kidney disease and significantly reduce the loss of kidney function. We will share perspectives on its journey to market in the United States.
The drug is called Tarpeyo, an advanced version of budesonide, a common anti-inflammatory medicine. It was developed by Calliditas Therapeutics, a specialty pharmaceutical company headquartered in Stockholm, Sweden, developing high-value products for patients with significant unmet medical needs. I’m delighted to welcome our special guest, Renée Aguiar-Lucanda, Chief Executive Officer of Calliditas Therapeutics. Welcome, Renée. Thanks for joining us.
Renée Aguiar Lucander
Thank you so much. It’s a pleasure to be here.
Neville Hobson
I’d also like to welcome Mike Ward, the Global Head of Life Sciences and Healthcare Thought Leadership at Clarivate, and a lead author for the Drugs to Watch program. Hi Mike, and welcome.
Mike Ward
Happy to be here.
Neville Hobson
So, Renée, great possibilities for Tarpeyo in the American market, high expectations. Before we delve into our close look, please tell our listeners about your role at Calliditas. What drew you into this field? What’s your passion about pharma?
Renée Aguiar Lucander
So I would say, I mean, I’ve always been attracted to innovation and the ability to improve things. And I actually have a fairly long career in the healthcare field, mainly from a financial and management perspective. I’ve spent the last seven years as chief executive officer of Calliditas Therapeutics. But prior to that, I spent about 12 years investing in life sciences.
And so, actually, I had an opportunity to really kind of look across a variety of investment cases and development organizations in Europe, which was truly fascinating. And prior to that, I spent another 12 years actually on the capital market side, raising capital for this very capital-intensive industry. So both health care and technology. And so, really, apart from my kind of operational experience, I’ve also spent a fair amount of time on different boards.
Again, really kind of being able to look into and learn from the mistakes, the opportunities, the ways to kind of build businesses. So I find this to be a challenging but truly fascinating industry to be in.
Neville Hobson
Well, you set the scene there. You’ve got the credentials, certainly. And I think you mentioned you’ve been CEO since 2017, and you’re leading an organization whose share price is up 40 percent in the last year by many reports.
There’s also the milestone, because this is the core of what we’re going to be talking about today. The milestone you’ve reached with securing approval by the US Food and Drug Administration, the FDA, for Tarpeyo.
We’ll come back to this point in a few minutes. I think it would benefit our listeners, though, if you would tell us about why this is so significant. How did Tarpeyo emerge?
Renée Aguiar Lucander
So with regards to the rare disease, this is a rare disease called IgA Nephropathy, which is an autoimmune disease of the kidney. And very little actually has happened with regards to development for this disease since it was originally discovered in the 60s. And one of the issues in terms of just kind of renal diseases, if you look at them in general, is that there’s been quite little kind of innovation over the last couple of decades, really kind of fundamentally driven by the fact that the bar is fairly high in terms of reaching clinical hard clinical endpoints with regards to kidney disease because it means you really have to investigate this in quite a lot of patients and it takes quite a long time for kidney function to decline generally.
So with regards to Tarpeyo, this really was a drug that was designed specifically to target the very origin of this disease called IgA Nephropathy. The company ran a very successful Phase 2B clinical trial, published in The Lancet. But the issue then was, how were we going to take this forward in a Phase 3 and potential approval in a rare disease where we really couldn’t run trials including a very huge number of patients or run it for very, very long time because it’s very difficult to raise capital for those type of trials.
So we spent a lot of time in parallel with the Phase 2B and after the Phase 2B really working with interest organizations with universities, etc. Using the data clinical data that we had, but also looking at other kinds of interventions in IgA Nephropathy, and really kind of came up with a meta-analysis that was ultimately, again, published in a peer-reviewed journal.
And on the basis of all of that work and effort, together with the clinical data, we were actually able to interact with the FDA and actually present kind of compelling scientific data and reasons for why we should be able to run a pivotal trial in this disease based on what’s called a surrogate marker. So not necessarily a clinical hard outcome, but in a two-step design, being able to show that there is an effect on this surrogate marker that has a reasonable likelihood to actually lead to an impact on a clinical hard end outcome.
So with that, we actually were the first company globally that was allowed to run this pivotal trial with this design, this two-step design, with using a surrogate marker, which in this case was reduction of proteinuria, which actually could lead to then an accelerated approval, and then follow that up with longer-term data to then achieve full approval based on more kind of accepted clinical endpoints, such as kidney function, which is what we achieved in March of 2023. And we started this trial obviously in 2018. So even under these types of kind of more accelerated forms, it’s still obviously a huge undertaking for the company, but obviously a successful one, which was fantastic.
Neville Hobson
Yeah, absolutely. Thanks very much, Renée.
Mike, let me turn to you. You said that the 2024 edition of Drugs to Watch highlights trends that will likely be consequential to the discovery, development and delivery of new medicines. It spotlights drugs and drug candidates that are likely to achieve important milestones in the coming years.
So tell us how the Drugs to Watch program actually works. Tarpeyo is one of the 13 drugs included in the report, as I mentioned earlier. What is it about Tarpeyo that got it on the list?
Mike Ward
Let me start about talking about Drugs to Watch. It’s an annual report. In fact, we’re in our 13th edition this year, and it takes about six months to prepare. And that process starts with us asking our 160 analysts that we have here at Clarivate to nominate candidates for inclusion in the report. And in that process, they have to explain why they believe that their nomination is worthy of inclusion.
We then prioritize the selections by tapping into the vast array of data sets and analytical solutions that we have here at Clarivate, including Cortellis Clinical Trials Intelligence, Cortellis Competitive Intelligence, our drug landscape and forecasting, and Cortellis Regulatory Intelligence. And then to get deeper insights into the potential competitive landscape, we also track the latest scientific breakthroughs using Clarivate’s Web of Science, Derwent Innovation’s ability to identify key patents; and conduct, actually also conduct interviews with leading physicians.
So using all that methodology, Calliditas Tarpeyo was chosen principally because it was sort of seen as a low locally acting drug with very limited bioavailability, it sort of reduces potential systemic side effects. And moreover, its delayed release design leads to, we believe, better results for patients. And it has a much better safety profile compared to conventional treatments.
Neville Hobson
Thanks Mike, that’s a very helpful explainer.
Let’s consider the significant step we mentioned earlier, FDA approval for Tarpeyo. Renée, I believe Tarpeyo triggered a unique shift in the FDA’s approach to regulation and approval of a new drug, especially from a European pharma company. You’ve also said that the lack of a regulatory pathway in the US was a challenge. What can you tell us about these points?
Renée Aguiar Lucander
Yes, so with regards to the regulatory pathway, I think that this really was a very significant event that the division actually kind of agreed to use a different kind of endpoint, this surrogate marker of reduction of proteinuria. And that really has led to almost an explosion of development efforts, not just in this rare disease but kind of really in rare diseases in renal in general, because it does allow for companies now to hopefully on the basis of this more kind of symptomatically related reduction in proteinuria, achieve acceptance, accelerated approval to kind of be on the market, and then actually kind of on a longer-term basis show that kind of clinical outcome.
So, really, what we’ve seen is that many, many companies are now pursuing development programs in the renal area, which is fantastic, right? I mean, this is really… for patients, this is great. There’s been so little studied in renal disease, and now I actually think that there’s a lot of real activity going on with real hope for many different indications related to renal. I do think it’s actually very significant. I think it is something that I think actually everyone that you would talk to who is developing will point to the fact that this was quite a very important trigger in some of these decisions. Probably not the only one, but certainly a very important one.
And of course, being a European company, I think obviously, people always say that, you know, it takes a village to do most things. And certainly in this kind of industry, it certainly does. So I think that having been a fairly small European company, taking on this kind of pioneering, really mantle of really driving this first-time-ever approach on a global stage was really extremely challenging, but also obviously very, very exciting. So, you know, the fact that we ran a global Phase 3 study and then really kind of was the first company ever to get accelerated approval on this basis in 2021 on this surrogate marker and being able to actually for the first time ever in the US offer this drug. So we actually were commercialized it ourselves in the US.
We really came out to the market for the first time ever; nephrologists had something that they could use in this rare disease, which again was super exciting.
Then, obviously, taking it all the way, continuing with the Phase 3 trial, having a successful readout, and really being able to show that we can significantly reduce loss of kidney function with this treatment is obviously fantastic. I think that we’re extremely proud of what we’ve achieved. And I think we’ve really worked very closely with patient organizations, with KOLs and international bases.
And I think it’s a great situation when we are kind of, we’ve always been considered a bit of an underdog in terms of, you know, clearly we weren’t going to be able to do this or that, or achieve these kinds of outcomes that we have. So when these things happen, and you actually can provide a disease-modifying drug to patients, it’s obviously an extremely rewarding feeling.
And I think it really goes to show that the incredible kind of focus and expertise, agility, commitment really of the whole Calliditis team has really been to some extent, you know, some extent really driven by this kind of very strong sense of the fact that we are truly pioneering something for the benefit of patients with this kind of rare disease in that we’ve been operating in for many years.
Neville Hobson
It’s quite a story. It actually makes me think, in my mind, listening to how you described all that, Renée, size doesn’t really matter whether you’re small, its attitude, maybe its approach. I mean, do you see yourselves as trailblazers for others to emulate your success? Are you a kind of a living use case in how to get approval from the FDA and the US for a new drug? Is that one way of looking at it, I suppose?
Renée Aguiar Lucander
Well, I do think that you’re right, that actually, obviously in this industry, you need so many different components in order to make everything work. We do know that there’s quite a lot of risks and biology is complicated. But I do think actually that having this attitude and the team spirit and really kind of working very closely with patient organizations, KOLs and all the other actors, and I think truly being committed to trying to make this work, I think is an important component, right? If you don’t have, you know, you have to kind of use the capital that’s available. You have to be a little bit smarter. You have to be a little bit trying to really kind of try and think about how can we possibly make this work?
And I think having that kind of commitment and background and focus, as I said, I think really it gets the best out of the team. Sometimes it can be extremely hard work and you don’t, you know, things don’t always go according to plan.
But I think it is a little bit of a leading star in terms of saying we can get this done. It is possible to get it through. And I hope that other companies, small, medium-sized, again, are going to try to do this because certainly, you know, we do need more medicines for rare disease. And we do need more kind of companies who are willing to take on some of this kind of hard work. I’m trying to pave the way for how can we actually kind of get through the regulatory process with a high level of quality and all of these things. We don’t wanna cut corners, but obviously sometimes in order to get medications out to patients, we have to be a little flexible and try a little bit maybe different pathways or methods to get there. Obviously it still has to show at the end obviously that this is the risk reward is appropriate and this is a safe and efficacious medication.
But sometimes if the barriers are so high that you can’t even start the process, then ultimately you’re leaving patients with very, very poor choices of off-label, ineffective, often toxic alternatives that clearly is also not a good alternative to trying to bring some of these innovative medications to the market.
Neville Hobson
It’s a good example. Mike, any thoughts you have on what Renée’s been saying? This is quite an innovation example, it seems to me.
Mike Ward
Absolutely. And I think that sort of Renée kind of ducked part of the question when you said, you know, are they almost a sort of a poster child for how a biotech can take innovation all the way through to patients? I think she was being sort of modest. I think that what they’ve done is they’ve actually shown that is possible. She was talking about some of the key issues about, you know, being agile. But I think it was also about listening to what the regulators are saying and actually having robust evidence to support their actions.
So I actually sort of think that other biotech companies who are looking to make that transition from R&D outfits into fully-fledged commercial businesses would do themselves a favor if they looked at the Calliditas story.
Neville Hobson
That’s a good one. So 2024 is proving to be quite an exciting year already, it seems to me. But what about 2034, 10 years from now? Can we expect similar excitement with blockbuster drugs and breakthrough medicines? Who will be the pharma kings then, I wonder? Where will they spring from? China, perhaps?
I’d like to hear your views, your perspectives on what’s next in the coming decade. Renée, would you care to share your thoughts?
Renée Aguiar Lucander
Sure, it always seems to me as if we think that we’ve kind of made all of the big discoveries to some extent. People are always wondering where is the next big innovation going to come from? All of these blockbuster drugs are kind of coming to the end of life and how are we possibly going to replace them and what possible things are there to do? But I do think that there is, the human mind is incredibly inventive and curious and I think there’s always kind of great innovation out there.
And I think that from what we’ve seen, I would expect actually that in two areas, obviously some of this is gonna come from our lifestyle. I mean, now we’re trying to almost have medications that goes against things that we to some extent, maybe create ourselves. I think that is going to be a big area of continuous development and work around that. I also think that in terms of how our brain works is still I think much shrouded in mystery.
I think there’s still a lot to be discovered there and a lot of kind of terrible diseases which really have no answer today. And so I think there’s going to be ability to do something there. And obviously we’re using different methods and different ways of developing these drugs today. It’s not all small molecules. I mean, it’s all kind of moving on and we’re using different tools and different kind of ways of actually kind of addressing some of these diseases, including rare diseases, where again, I think there’s a still a very significant unmet medical need.
Neville Hobson
Okay, Mike, what do you think?
Mike Ward
Yeah, that’s totally echoing what Renée was saying there. I mean, with Drugs to Watch, in addition to highlighting some of the molecules that we believe are gonna be consequential to patient wellbeing, we also look at new technologies that are going to be the cornerstones of the next generation of medicines.
So, especially exciting, for example, is the potential of gene editing. So one of the Drugs to Watch in this year’s report is Exocell, which was created using the Nobel Prize-winning CRISPR-Cas9 genetic technology. And just in the past month or so, it’s won approvals to be a treatment for sickle cell disease and also for beta-thalassemia. So these are some of the rare diseases that Renée was talking about.
Also, one of the other things that Renée again sort of alluded to is around neuroscience. So for the past decade and a half, we’ve seen huge advances in the oncology space. And it’s now sort of, if one listens to the sort of the chatter in the corridors at some of the big partnering conferences, you can sort of see that big pharma looking to get back into the neuroscience space, and there is a sort of a belief that our understanding of how the brain works is going to spawn a new generation of medicines, whether it’s treating major depressive disorder or schizophrenia, or of course, you know, Alzheimer’s.
So I think we’re going to sort of see what we saw in oncology may be being repeated in the neuroscience, in science space in the next 10 years. And it’s also, again, Renée sort of you mentioned it, a lot of these revolutionary treatments that we’ve had and have made a dramatic impact on patient outcomes in recent years are about to go off patent. And so actually less expensive generic or biosimilar versions of them are going to be available.
So that’s going to also help more patients. So we’ve got a whole raft of products that we know work that are really successful, really helpful to patients, are going to be less expensive and therefore more patients will benefit. And I do believe also with all the new technology platforms that are being developed, the future is bright.
Neville Hobson
That’s a good end point, I think, for our conversation. Thank you both, Renée and Mike, for your time today and for sharing your insights on what’s happening in the industry and transformation, full of candidates to breakthrough that are poised to have extraordinary impacts on patient outcomes in the coming years. Thank you, both of you.
Renée Aguiar Lucander
Thank you.
Mike Ward
Thank you.
Neville Hobson
You’ve been listening to a conversation with Renée Aguilar-Lucanda, CEO of Calliditas Therapeutics, and Mike Ward, Global Head of Life Sciences and Healthcare Thought Leadership at Clarivate and the lead author for the Drugs to Watch program, about a journey to market for an innovative new drug and considered perspectives on the market itself.
For information about Calliditas Therapeutics innovations, visit Calliditas dot se. For information about the Clarivate Drugs to Watch Report 2024, visit clarivate dot com slash drugs dash two dash watch.
In a few weeks, we’ll release our next episode. Visit clarivate dot com slash podcasts for information about Ideas to Innovation. And for this episode, please consider sharing it with your friends and colleagues, rating us on your favorite podcast app or leaving a review.
Until next time, thanks for listening.
Outro v/o: Ideas to Innovation. From Clarivate.
