Basilea Pharmaceutica AG (BSLN) Earnings Call Transcript & Summary

So good afternoon, everybody. Welcome to the Basilea presentation at our health care -- virtual health care conference this year. It's my pleasure to introduce the CEO of Basilea, David. And without further ado, I will hand it over to David and his presentation.

Thank you, [ Michael ], and hello. I'd like to thank UBS for inviting Basilea to present at this UBS Virtual Global Health Care Conference. I understand you have access to our presentation. So I will refer to slide numbers from that presentation from time to time. I'm going to provide a general update on Basilea, our programs, our revenues and also make mention of any COVID-19 impacts on our company's business. If you go to Slide 2, you'll see in summary, for those of you less familiar with the company, we are a commercial-stage biotech company with significantly growing cash flows from our commercialized products. We are focused in the areas of oncology and infectious diseases. And we believe we have the potential for sustainable growth and value creation based on both the growth of our commercialized brands and also innovative pipeline. We've got experience of bringing products all the way from research through development to the marketplace. We've done this in fact 3 times. One compound we sold after it was commercialized. But we have 2 remaining compounds that we're commercializing now, Cresemba and Zevtera, and I'll come back to those compounds shortly. And then we have 3 oncology drug candidates in development, which reflect our strategic additional pillar we added about 10 years ago to the company from the anti-infective beginnings to an oncology anti-infective company. If you move to Slide 3 in the presentation, you'll see our pipeline in its entirety as of today. At the top of this slide, you'll see the commercialized assets, Cresemba and Zevtera. And then below that, the oncology clinical assets. What's important to bear in mind about our strategy is that we build our pipeline through both internal research and development, but also through in-licensing. And proof of that is in the clinical oncology arena, where, for example, our lead oncology asset, derazantinib, the FGFR inhibitor, was in-licensed from ArQule. Now obviously, it's part of Merck. Behind the clinical stage assets, we have a number of preclinical assets in both anti-infectives and oncology. What I should probably also add is that whilst I say Cresemba and Zevtera are commercialized, they are in many markets. So Cresemba is available now marketed in more than 40 markets. Zevtera is marketed in about 18 markets. But in addition, there are many important markets where we're currently going through the clinical trial or regulatory pathway in order to gain approval of those compounds. Going to Slide 5 in our presentation, you'll see an example of another part of our strategy, which is our commercial stage partnership strategy. What this enables us to do -- we don't have a Basilea presence around the world. We operate through competent partners and the agreements we have enable us to potentially maximize the global revenues of our commercial stage assets. This example here is for Cresemba. We have a similar picture for Zevtera. On Slide 6, you'll see that our partnerships, we divide them into 2 types really. There's our license partnerships and distribution partnerships. The license partnerships are essentially ones whereby the partner is responsible for the manufacturing. The distribution partnerships is Basilea retains that responsibility for the manufacturing. It doesn't mean that we have our own manufacturing facilities, it means that we have the contracts with CMOs, for instance. And then in both these situations, the license and distribution partnerships, we obviously participate in the revenues. In terms of the license partnerships, it's through royalty and then with milestone payments. And then with the distribution partnerships, it's with a transfer of price, which is a percent of net sales and also milestone payments as well. What I wanted to do is just briefly cover a few points on each of the 2 commercialized assets and then the 2 lead oncology clinical-stage assets, derazantinib and lisavanbulin. So beginning with the commercialized assets. If you go to Slide 9 in the deck, you'll see that our lead commercial asset Cresemba, which it's fair to say we don't break out separately the Cresemba and the Zevtera revenues. But what we do say is the majority of the revenues are coming from Cresemba currently. And Cresemba is more specifically an invasive mold infection compound. So it works against the indications of the diseases of aspergillosis, mucormycosis. So it's the mold infections, which are really in immunocompromised patients. So patients, for example, that are undergoing intensive cancer treatment or have had solid organ transplants. So the immune system has weakened, and they're prone to invasive mold infections. In terms of how's Cresemba doing since it was launched in terms of its revenues, if you go to Slide 10 in the deck, you'll see the Cresemba revenue uptake. And this is particularly strong growth, a linear picture. Majority of the sales at the moment from the U.S., but that's purely as a result of the fact the U.S. launched effectively a year earlier than Europe and also market access was a lot quicker. Europe is increasingly taking a proportion of the sales. You can see that in the graph. And what's encouraging for us is when you look at the best analogue for Cresemba, which is voriconazole. The sales in the EU major 5 markets were very similar to the sales in the U.S. So we believe, and you can see that on this graph, there's a lot more growth to come from the U.S., but there's even more growth available to come from Europe. And then as I alluded to earlier, there are some really important markets like China and Japan where we're not even licensed at the moment. We're going through the clinical or regulatory pathway in order to get license and then start commercializing. So Cresemba has a long runway and a lot of future growth potential ahead of it. If you move to Slide 11 in the deck, you'll see a question we often get asked is what's the potential of Cresemba. I think there's a point here, which points to, as I said, the main analogue for us for this compound is voriconazole. And that peaked at about $900 million of global sales. We've sold, as I said, $200 million of in-market IQVIA sales. And basically, the important thing for us is that we've got a runway ahead of us, which our exclusivity runway, that last to 2027 in the U.S. and 2027 in the EU, as long as we complete the pediatric program, which would extend the exclusivity in the EU from '25 to '27. And that program is on track. The important other element, which is quite unusual across different therapy areas is that the competition is really not very fierce in terms of the future competition. In terms of the -- there's nothing that we can see that really would replace Cresemba as a treatment of choice in invasive mold infections that's in Phase III development, for example. So we've got many years where we believe there's no real competition to the current status quo and no other compounds already promoted in this market. So Cresemba has got hopefully a healthy runway of growth -- sales growth in front of it. In terms of the other compound that we're commercializing now, that's our antibiotic Zevtera, ceftobiprole. If you turn to 13, Page 13 in the deck, you'll see that this is a broad spectrum gram-positive gram-negative antibiotic, but particularly interesting activity against MRSA. And then if you go to Slide 14 in the deck, you'll see that basically, the MRSA analogues that we've highlighted here, had a very different picture of sales distribution versus what I showed you, what I told you with regard to Cresemba. So for MRSA antibiotics, we believe the lion share of the potential is in the U.S. and you can see this by looking at other antibiotics, as I say, like daptomycin or ceftaroline with activity against MRSA. And about 90% of their sales were in the U.S.A., and that's largely because of factors such as the MRSA rate in the U.S. and also the volume of antibiotics used in the U.S. as compared with many other countries. So with that said, obviously, the next question is, how do we access the U.S.? Because currently, we're not licensed for the U.S. marketplace. In terms of the strategy as shown on Page 15 in the presentation. And basically, we've agreed with the FDA on 2 cross-supportive studies. One on acute bacterial skin and skin structure infection study, which actually has completed already, which completed in all of the top line results were released in August of last year. And then the second study is staphylococcus aureus bacteremia or bloodstream infection study. That's on track to report top line results in the second half of 2021. Now importantly, for us, is that this Phase III program is largely funded by BARDA, so by effectively the U.S. government through a non-dilutive grant of up to $128 million, which pays for about 70% of the total program costs, for the Phase III program. And then the second other relevant point, I think, is the point that with the QIDP designation, ceftobiprole would have 10 years of market exclusivity following the approval. So if you follow my time line's point that the second of the Phase III studies would report top line results at the end of 2021. If this was all on track, this would lead to an approval, say, at the end of 2022, which then if we got the product approved then, we would have 10 years of exclusivity from 2020 -- in 2022 to 10 years after that date, independent of the patent situation, which ends before that exclusivity period. One other point on the U.S. strategy is that it's also worth stressing is that like we've done for ceftobiprole or isavuconazole or Cresemba around the world, we would look for a partner to commercialize ceftobiprole, Zevtera, in the U.S. That's worth stressing that point. In terms of the -- moving to the oncology clinical stage assets, as I said at the beginning, our focus on the lead to assets, derazantinib and lisavanbulin. In terms of derazantinib, if you go to Slide 17 in the presentation, you will see that this summarizes really our strategy with regard to derazantinib. As I said at the very beginning, we in-licensed this from ArQule back in April 2018, and when we did, there was no approved FGFR inhibitor. Now move the clock forward. There were actually 2 approved FGFR inhibitors, which gives us great confidence in terms of the value of the cost and the target that this involves, the FGFR target. In terms of our particular development strategy for our compound, we're really focused on achieving differentiation by leveraging the unique properties of derazantinib. And what I mean by -- we mean the innate properties of the compound that make it different than other compounds. And a couple of examples, a key couple of differentiation factors we've identified. One is the specific kinase inhibition profile, and the other is the evolving safety profile differences between the different FGFR inhibitors. I'll come back to these 2 points in just a second. It's worth stressing. We have 2 studies ongoing, one in intrahepatic cholangiocarcinoma or bile duct cancer and one in urothelial cancer. And then we also kind of start in a third study in the Q3 of this year, which will be in gastric cancer. And these studies, when we took over the compound in April 2018, there was an ongoing study in ICCA. So what we've been spent a lot of time doing is profiling the compound, as I said, to look at the differentiated profile of the compound and move into tumor types where we believe we can show a difference. In terms of the 2 points of differentiation I highlighted, first of all, the kinase inhibition profile. If you look at Slide 18, you'll see this depicted here. And what this just points out is that many of the marketed or late-stage FGFR inhibitors have activity against FGFR 1, 2 and 3. Derazantinib seems to be unique in terms of its CSF1R activity. And the VEGFR2, again seems to be unique, along with Atezolizumab that also has activity against VEGFR2. But in particular, the CSF1R activity looks to be a completely differentiated feature for our compound versus the other late-stage development compounds. In terms of the relevance of that is depicted on Slide 19, which is -- you may be aware of the hypothesis of the CSF1R inhibition. They reprogram the immunosuppressive tumor-infiltrating macrophages storing T cell activity and thereby improving susceptibility to PD-1s or PD-L1 inhibitors. So the concept or the hypothesis that we are keen to explore that is whether or not having these sort of dual modes of action, actually can confer a clinical benefit to patients versus a compound that doesn't have the CSF1 activity in addition to the FGFR activity. This is also why Roche partnered with us in terms of a clinical supply agreement in terms of the use of Tecentriq in combination with derazantinib in the urothelial cancer setting. Actually, this has since been expanded, and Roche is also partnering with us in terms of the clinical supply agreement in the gastric cancer study as well, looking at the combination in that setting as well. In terms of the second key differentiating factor that seems -- and this is an evolving situation, but on Slide 20 in the deck, we just profile what we can glean from either the [ SFPCs ], the posters and abstracts at congresses, and looking at the various -- the information that's available on the safety profile of the different late-stage FGFR inhibitors in cholangiocarcinoma and urothelial cancer. And what this -- and it's fair to say this is in a noncomparative trial setting. But what we see is the fact that derazantinib and all the compounds seem to have what we believe are sort of class, FGFR class effects. And then there seem to be some specific safety signals with some compounds, which you don't get with derazantinib. For example, in the areas of retinopathy, hand-foot syndrome, nail toxicity, stomatitis, a number of safety features where derazantinib seems to be at a lower level. Obviously, this needs to play out as we continue to evolve in the clinical trials we have ongoing. But this is just something that we've seen to date from the data that's available today. In terms of where we are then in the clinical studies, to bring this together on Page 21 in the presentation, you will see that we have the ongoing Phase II intrahepatic cholangiocarcinoma study. As I mentioned a moment ago, we have the interim data from the FGFR gene infusion cohort that we at the beginning of 2019, and then we expect the top line data from this cohort in the second half of 2020. And then we have a second cohort, looking at FGFR gene mutations and amplifications. So these are the non-gene FGFR gene fusions. And here, again, we're expecting interim data rather than the top line results, the interim data from this cohort, which was started after the first cohort, again in the second half of this year. And again, if we could show data, positive data in this cohort, this we believe could potentially differentiate us versus other FGFR inhibitors. And then on Page 22 in the presentation, you will see the -- both the urothelial cancer and the gastric cancer studies in terms of the outline of the studies with, again, the first interim data expected in the second half of this year in the ongoing urothelial cancer study. And then as I mentioned a moment ago, the gastric cancer study is expected to start enrollment in Q3 of this year. Just switching to the last of the compounds I wanted to profile today, and that's lisavanbulin. So on Slide 24 in the deck, it is profiles of the compound. This is a novel compound. It's a tumor checkpoint controller that crosses the blood vein barrier. So probably not surprisingly, we've studied it most extensively in glioblastoma. It binds to a unique site for an oncology compound on the microtubule. And interestingly, what we have now observed is shown on, first of all, on Page 25 in the deck where we just talk about, first of all, the preclinical evidence behind this biomarker, EB1, the plus-end binding protein 1. This is -- look, it's a protein located on the microtubules, and it's involved in microtubule dynamics. And what we found in mice -- in mouse models, was that the EB1 was predictive of response to lisavanbulin. There was a benefit in terms of down regulated EB1 to mice. So there was a bigger difference, a survival advantage that more than doubled in EB1 expressing GBM mouse models. Now that in itself is interesting, but what became even more interesting for us was when -- if you go to Slide 26, you'll see in Slide 26, we identified in the study, the Phase I/II study we carried out in GBM patients with the oral lisavanbulin that we completed at the end of last year, and the full report will come out later this year. But what we noticed was there was a particularly strong response in a patient whose tumor reduced by -- a glioblastoma tumor reduced by more than 80%. And when we looked at the tumor material, we actually found that the patient was a very strongly expressing EB1 patient. Then we looked at some of the tissue material from patients who had not such a strong response, and we found these were all not express in EB1. So armed with the information from both the clinical study and the preclinical data in the mouse models, we felt compelled to begin in Q3, mid this year, stroke Q3 this year, we anticipated to start EB1 biomarker-driven Phase II study of lisavanbulin in glioblastoma. And we think this is very exciting because -- not least because we'll get to result quickly. This will be an open-label study. And the first cohort of patients, of 10-or-so patients, we would pretty quickly know whether our hypothesis here is playing out in terms of the data, in terms of the response rate or whether it doesn't. And so we'll know pretty quickly during the course of 2021 if this data plays out in terms of the results. So that's the hypothesis behind the lisavanbulin program. In terms of bringing this together and then just mentioning about the COVID-19. First of all, if you go to Slide 28 in the presentation, you'll see our financial summary. What I should say is, first of all, if you look at the revenues, it looks as if, from a revenue, but also from a P&L point of view for that matter, that 2019, 2020 look similar. What we have to do is go a little bit under the surface to understand really what's going on, particularly on the revenue perspective. And what's really going on is that the Cresemba or Zevtera, what we call non-deferred revenues. So basically, those most actively reflect what I showed or talked about earlier. In terms of the net sales uptake, particularly of Cresemba, they are driving the non-deferred revenue growth from CHF 68.7 million in 2019 to CHF 77 million to CHF 87 million in 2020. That's the range we give in our guidance. In terms of -- we have the -- another element of the revenues, which is our deferred revenues, which are Cresemba and Zevtera deferred revenues. And these are actually as a result of upfront or milestone payments, which we recognize over a number of years. And actually, after 2020, they'll largely be recognized, fully recognized. But in 2019, '20, that recognition goes down. So we cover that by the ongoing royalty and milestone payments in the Cresemba and Zevtera era. And then the final element of our revenues is the -- what we call other revenue. This is effectively -- the majority of this, almost all of it is the BARDA reimbursement. So as the U.S. government give us reimbursement for the cost of ceftobiprole Phase III, we booked the gross costs in our P&L that we booked the reimbursed cost as revenues in the P&L. And that's decreased slightly as well. So with these sort of deferred revenues going down, the other revenue going down a bit, you get -- the total picture looks flat from a sort of total revenue point of view, but the underlying health of the Cresemba and Zevtera business is significantly increasing. The positive aspect of this also, obviously, is that the cash-generated revenue component is going up. So our cash burn is going down. So you can see on Slide 28 that our cash position at the end of 2019 was CHF 161 million, which at the end of this year, we're guiding at CHF 100 million to CHF 110 million, which is a decrease in our cash burn versus '19 to '18 and prior to that. So our cash burn is going down, obviously. Then I should make a comment about COVID-19 at this point, which we said a few weeks ago that we see no negative impact on our revenues to date. So I know it's early days, but we see no impact on our revenues and no reason to change our guidance. We'll obviously update our guidance -- our half year results in August. But in terms of now, there's no reason to update the market because we see no reason why our guidance, we would come off our guidance at all. So our revenues and our different elements of our guidance stay as they are. In terms of the clinical trials and probably that's worth going to be my final slide, on Slide 29 in the presentation. Slide 29 talks about the next data readouts across our compounds and across time until the end of 2021. And what's worth saying is at the moment, obviously, we're in the area of advanced cancer anti-infectives. And certainly, in the cancer area, we've seen no drop-off in terms of -- due to COVID-19 in terms of patient accrual in our studies. Our studies were actually the new studies, which we started in gastric cancer for derazantinib and lisavanbulin, the biomarker-driven study. We're not planning to be started now anyway. So we still will keep to the concept outlined in this slide here that these studies will start in the second half of 2020. And then the ceftobiprole Phase III study, it doesn't report its top line results, as I said earlier, until the second half of 2021. So it's too difficult to tell now whether or not there might be a blip for a few months, but we've got time to catch up. So again, we've got no reason to come off our current guidelines for all of our compounds in terms of their next value inflection points or data readouts at the moment. In terms of summarizing then what I've said so far, you -- on this slide, Slide 29, it quite nicely, in a way, summarizes our focus, which is the arrow at the top of this slide depicts. We've got the focus of increasing our cash flows through the revenue growth of Cresemba and Zevtera. As I said, that will be done by both growing the commercial success in the markets we're in on the one hand and then launching in additional new markets, and that's the one focus of the company. And then the second focus of the company is on striving to develop the pipeline to these, as shown on this slide here, the next data readouts of each of the compounds. And obviously, derazantinib and lisavanbulin are really critical for us from the clinical stage assets. And then, as I said earlier, ceftobiprole, the U.S. program is critical. And then for isavuconazole, finishing the study, as shown here, to access Japan. And then we're also for completeness, give other markets like China, where we're going through the regulatory process at the moment. So that's very much the focus of the company's efforts at the moment. Hopefully, you've managed to follow my presentation. And I thank you for your interest in Basilea. And I wish you enjoy the rest of the conference. Thank you very much.