Oncopeptides AB (publ) (ONCO) Earnings Call Transcript & Summary

Welcome to Oncopeptides Press Conference 2022. [Operator Instructions] Today, I'm pleased to present CEO, Jakob Lindberg; and CMO, Klaas Bakker. Please begin your meeting.

Thank you very much. Good afternoon, good morning. This is Jakob Lindberg, CEO of Oncopeptides, speaking. And as told, Klaas Bakker is also here today. Before we start to flip the pages in the presentation, the reason for us having this webcast is, of course, that we had an Oncology Division Advisory Committee meeting last Thursday night, Central European Time, and we want to be available for any questions today. There will be topics that we can't cover, but we'll do our best to answer the questions you have. With that, I think we should go to Slide #2. As always, we have the standard disclaimer regarding forward-looking statements, and it's also available on our web page if anyone wants or needs to read up on that. But there's nothing new, obviously there. Slide #3, thank you. So today, I am here, and then also Dr. Klaas Bakker, Chief Medical Officer, and we'll try to cover as much of your questions as possible. If we then go to Slide #4. So in summary, we received a negative vote from the FDA Oncology Drug Advisory Committee regarding whether OCEAN as a study confirmed the favorable benefit/risk for Pepaxto. We will, of course, collaborate with the FDA regarding next steps with Pepaxto in the U.S.A. And I must highlight, we -- at this current point, we do not know exactly how the FDA will come back to us, nor do we know exactly when, even though it's most common that this happens within a 14-day or a 2-week period post-ODAC. But there is no rule. There is no statutory rule regarding this. Before we go into the actual Q&A session, I would like us to go to Slide #5, which, of course, just contains 2 words, The Patient. And I want to go back to January when we rescinded our voluntary withdrawal in the U.S. Why did we do that? As you know, the OCEAN study has extraordinary large differences in clinical outcome across prespecified subgroups. To make it very simplistic, and it's an oversimplified but still fairly accurate representation, an elderly non-transplanted patient survives for roughly twice as long on melflufen compared to pomalidomide, while a young transplanted patient survives roughly twice as long on pomalidomide compared to melflufen. And the whole heart of this discussion is whether this -- these results are real or not. We thought in January that we could prove that these differences are real in the study. And as a consequence, then rescinded our voluntary withdrawal because if you are of the opinion that we are and feel that we scientifically can prove that, this product, of course, is very important for patients in the elderly non-transplanted population to get the access to. Since pomalidomide plus dexamethasone even as a treatment combination is one of the most commonly used treatments for exactly these patients. In June, EMA agreed with us. And as a result of that agreement, we received full approval, no post-marketing requirements, no post-marketing commitments. Of course, we have yet to achieve the same result with the FDA, and we don't know if we will. But the reason why we are continuing to fight for this is, of course, that we are of a very firm and honest scientific opinion that these results are real. And if you sit on a treatment, which you believe have those very large outcomes, positive outcome differences compared to the current standard of care in a patient population, you have a moral obligation to stand up for that and see if you can convince the authorities that this will happen. There are a lot of things at work here, including different regulatory environments. And regulatory environments have a lot of important things on their plate, apart from the products that they're actually looking at, normally precedent. That is if we make a certain ruling in one case, we also have to apply it to others. So a lot of considerations are taken into account when doing these things. And we have the uttermost respect for that. We'll see what happens next with the FDA and hence, the future or the potential future for Pepaxto in the U.S. And at this current date, we don't know. And as soon as we get clarity, we will, of course, return back to the markets with such information. That was actually just the very short beginning of, a, what happened Thursday; b, what the heart of the scientific topic is. And now I would like to go to Slide #6 and just say that we are absolutely open for questions and answers from anyone that has them. So please go ahead.

[Operator Instructions] And our first question comes from Patrik Ling, DNB.

Jakob and Klaas, a couple of questions. I mean maybe you can answer a question that we get quite a lot and that is, can the FDA based on what the committee came -- sort of the conclusions from the committee, can they sort of force the product off the market? Is that one option for them?

Okay. And I can answer that. Yes. So in -- the committee, drugs advisory, to force a product off the market, that's a legal process. And FDA or EMA, for that matter, can always initiate the legal process to take a product off the market. That legal process, of course, requires you to then bring solid scientific arguments for why the product, why that -- because that's a right that the market authorization holder has, why that right should be taken away. And then you need to make the scientific case that there is sort of a public health risk for the product to remain, right? And whether they will choose that path or not, I don't know. And we don't know that yet.

Could I also ask, I mean, at ODAC meeting, you presented ways of making the indication for Pepaxto a little bit more narrow, and they didn't really seem to like that. That -- it came through the ODAC meeting. Have you had any other discussions with the FDA about narrowing down the indications?

Yes, we have had. And I must say that the meeting was a bit ambiguous on this point because they welcome further studies, and we welcome that as well to confirm benefit in these subgroups, right? For that to happen, we then need to be in agreement about a -- most likely a positive benefit/risk for those patients. So we need to wait for the FDA to return to us to actually comment on exactly what their position is post the ODAC. They have a lot to think about there. So -- and I respect that.

And if I may chime in there, Jakob. I think it's critically important to understand that none of the advisers has actively said that this product should no longer be on the market, but also that they were also encouraging further studies. And this comes exactly to Jakob's point, for further studies, you need to accept that there is a group that has a positive benefit/risk. So in that way, everything is tied together, the future chemical development program and the question whether the product can stay on the market or not.

Okay. I mean the FDA stated several times that this was a meeting where they only considered the U.S. regulatory environment. And you also mentioned that sort of the third party in the room there about the European authorities. Have you had any reactions from the European authorities after this meeting because I suppose they looked at it as well?

Well, no, no reactions, Patrik. I mean they were extremely aware of this when -- I mean the European authorities knew the FDA's stance from last summer when the European authorities looked at all the data this spring. So this didn't come as a surprise to them in itself. So they -- it's not like the FDA position brings any news to the Europeans. So I've seen some ideas that this meeting would influence EMA somehow to change or anything. No. The FDA position was made clear last summer and the Europeans disagreed with that position, and hence, invited the FDA throughout the spring to participate in the process because they, of course, want to collaborate to the largest extent possible as 2 competent regulatory authorities. So no, no, nothing there.

Critical there, Patrik, to also understand is that an agency, in this case the EMA, would only be able to act if we would have submitted new data or if this ODAC would comprise completely new data about the product. The reality is that both regulatory authorities have looked at exactly the same data sets. So there was no new data available for Pepaxto, hence, there should not be any discussion about whether to keep this product with a full approval in Europe. They have all the data and no new data has been presented. So we can rule that out basically.

Okay. Great. I have one more question, and it's not really maybe directly related to the ODAC meeting. So maybe I can jump back in the queue and see if there are any more questions, then otherwise, I'll come back.

And the next question comes from Adam Karlsson from ABG.

First one on how -- it may be premature for you guys to respond to it, but I'll ask it anyway. How do you anticipate responding if the FDA went down the route of trying to legally remove the accelerated approval as opposed to using other weapons in its armory, clinical holds and safety warnings and so on, to keep Pepaxto off the market? Do you envisage fighting that in the courts? Or would that be a too tall an order?

As you said, I do not want to really speculate in what we will do. What I can say is that we will, of course, stay true to science and what we believe the scientific truth is, and hence, how that translates into what patients should be treated with. Then, of course, you don't want to enter into any kind of [ Sisyphean ] exercise. So depending on what comes, we need to respond at that time with that -- with what I said as guidance. But I don't want to state exactly what we're going to do based on scenarios right now. And I first -- I'm very curious exactly what the FDA will come back with to be frank first.

Okay. No that's fully understandable. Second question then on the relevance of other upcoming readouts, as you alluded to in the -- in your part of the presentation at the ODAC readouts that could have a bearing on the Pepaxto situation. And my question is, I guess, against the backdrop of FDA's apparent position on the relevance of other studies that directly or indirectly isolate the potential age effect of IMiDs, speaking specifically on the DREAMM-3 readout that's coming up, and the FDA's position or how they treat that argument of looking at other studies. How, I guess, likely is it that the FDA would use another line of argument other than what they view so far, that these are the studies while potentially of interest and so on, that they would not be enough to change their view of the OCEAN study? How do you see the likelihood of that DREAMM study if it came out in a way that would be favorable to you, being enough to sort of sway the FDA to reconsider? It might be another tough question to answer but...

Yes. We are speculating again. But I think, Adam, that you are mixing 2 topics because any regulatory agency needs to stand by the labels that are fully approved until they don't. So this means that any speculation about what DREAMM-3 could or could not influence in terms of us, since that would involve other labels, they can't comment on, and they have to keep that line, and I fully respect that until they don't have that line anymore. So you shouldn't read too much into the fact that the regulatory authorities stands by the current labels, until they don't. So I think that study might have an impact. I have -- I don't know how much. But if it ends up the way that I personally anticipated to, it will at least be part of the discussion. Will it be sufficient to move in one direction or the other? I don't know.

[Operator Instructions] We just received a question here from Patrik Ling, DNB.

Yes. It's not directly related to ODAC, but maybe you can give us a little bit of an update on the processes in Europe for Pepaxto? And the upcoming launch in Germany?

Absolutely. So the launch planning is going full ahead, and we are actually on schedule with everything from inspections to various certifications to have product in the channel, et cetera. And we will, within short, in conjunction with the launch in Q4 that we have promised, also give much more of details regarding price, estimated -- addressable patient population and things of that sort. We have also, of course, initiated the processes in other geographies in Europe. We are slightly behind the curve, obviously, from where another company would be at this stage, given everything that happened over the last year, because as you know, Germany is the only country where you can launch at the same time as you initiate negotiations about price. While in all other geographies, almost without exception, you need to first negotiate the price and land that negotiation before you can launch, which means that what will happen now is that we initiate price negotiations in multiple geographies beginning now and through the rest of the year and beginning of next year. And they take between 16 -- sorry, between 6 and 24 months to land these pricing negotiations depending on country. And then, of course, in the meantime, as we file the so-called value dossier in Germany, we can initiate the launch there. So all of this is going according to schedule. And in probably somewhere in October, we will call for a webcast where we will shed a lot more light on that. The reason for not doing it today was that it felt a little bit like you were mixing messages where everyone was focused on the ODAC, and to start to talk about the European launch and give all the details at the same time felt like we were sort of making a sandwich with different layers instead of keeping on message. But we will come back within short, Patrik, and everything is going according to plan right now.

And the next question comes from Nicholas Lorusso.

This is Nick on for Boris Peaker at Cowen. Just a quick question, and you obviously may not be able to answer this too well until you're meeting with the FDA, but I was just wondering if they were to allow for additional studies for Pepaxto, what your initial thoughts on this would be? And what type of studies this would need? Like how big they would be, how many additional studies? Just any initial thoughts that you have would be great.

Yes. Klaas, you can comment as well on this, but I think if you think about it what boundary conditions you would like to meet given the discussions? First of all, you would like a study that is recruitable in the U.S., which is really rare for Phase III studies in myeloma, unfortunately, and that more or less requires certain designs. One such design would be Dara-Vd versus melflufen Dara-Vd for example, that would be such a design. . And the second boundary condition, I think, is that alkylators are normally combined less great with IMiDs but are very good in combination with proteasome inhibition. So some sort of combination treatment with a proteasome inhibitor is probably also the second boundary condition you would like to meet. Now how that plays out? I just gave you 1 potential such design that would actually satisfy both of these criteria. And I'm not saying this is what we're going to do, but it's something along those lines. And of course, it would be in the recommended population that is that we would still exclude patients with a less successful stem-cell transplant. At least that would be our basic assumption, but we need to agree with the regulator here as well. I don't know, Klaas, if you would like to add something to that.

No, I have nothing to add there.

Just one additional question. FDA mentioned during the ODAC meeting, I think their third point, about the potential changes in the dose for Pepaxto. Would this be possible in that trial? Or would this need an additional trial for like a different dose expansion or dose escalation or something like that?

Klaas, would you like to comment on that?

Yes. So like we pointed out also during the ODAC meeting in our presentation, we firmly believe in a flat dose, which is also the reason why we, in the European situation, have a flat dose supported by almost 500 patients of clinical data. We have made some dosing adjustments that we would like to prospectively test and that could very well be in the new trial in the recommended patient population. So I would see these issues going hand-in-hand if possible. But as said during the ODAC, it was not the most important topic for the committee, and we feel pretty strong about the position of having a flat dose.

Thank you. No further questions at this time. I hand over the [ word ] back to you speakers for any closing remarks.

So thank you, everyone. We are, of course, available for further questions if you have them to e-mail or call us directly. I'm sorry that we can't give a lot more guidance. And as I stated in an interview this morning, I don't envy you being shareholders here, to make sort of what is north and south in all this. I think even the ODAC expressed that it didn't know what to think about the topics, right? Because it's such different views on the same substance matter from various parties, not just from us and the FDA, but also from EMA. So exactly what the outcome will be here is a bit unknown to us. And as soon as we know better, we will, of course, come back with as much information as possible. Thank you very much for joining us here today, and do not hesitate to reach out to us should you have any further questions. Thank you.