All, and a warm welcome to Nanoform's webcast presentation and Q&A session. Today is January 26th, and we have today press released promising clinical results, and we will present the conclusions on this call. My name is Henri von Haartman, and I'm your Director of Investor Relations. Today, our CEO, Edward Hæggström, CFO, Albert Hæggström, General Counsel, Peter Hänninen, and Chief Commercial Officer, Christian Jones, will present to you. This presentation is webcasted through Financial Hearings, and after the presentation, we will hold a Q&A session, and it's possible to ask questions by calling in. Today, our CEO will start with controlled crystallization by CESS, the clinical trial conclusions, and the strategy going forward for this drug candidate. Our CFO will present the business opportunity within amorphous solid dispersions. Our General Counsel, Peter Hänninen, will present the IP position.
Our Chief Commercial Officer will conclude with his views on the overall commercial impact from today's announced results. With these words, our CEO, founder, Professor Edward Hæggström, please go ahead.
This is Edward, and welcome also on my behalf. So I would like to walk you through a few bullet points related to what we press released. First of all, this is the biggest news for Nanoform in its history. It's a clinical demonstration that Nanoforming is a credible alternative to improve a fantastic product that currently sells for more than EUR 5 billion a year. This is a leading opportunity to get the Nanoform product onto the market. This study also validates the use of Nanoforming to improve current ASD formulations on the market and in the development. These amorphous spray-dried assets are often manufactured either by spray-drying or hot melt extrusion, which are two leading technologies. There are approximately 50 ASDs products on the market, and by our own estimate, hundreds in the development.
Therefore, we see this presented data as a further validation of our belief that Nanoforming can be a new important technology in the global pharma industry. So bottom line, we are really, really happy that we have gotten this far, and we think this is a very important milestone for us as a company. Over to you, Henri.
Thank you. Albert, are you there?
Okay, I think I will continue. It's Albert here. With the business opportunity behind this, and when I think back, since the IPO and since before the IPO, our ambition has always been that, and our vision has always been that we might, on our hands, have a technology that is superior, it's greener, it's potentially superior to the best technologies out there, like Spray Drying, Hot Melt Extrusion, and so forth. During the last few years, we have been building a facility, a fantastic factory, and we have also been gathering data, both animal data, and as you remember, our first clinical data in 2020 with the piroxicam study. During these years, we have still kept our vision and dream that Nanoforming could become really big.
For me, this news and this study is another one of these really great moments in Nanoform's history, where we can with pride say that we have again done something quite amazing. So we are really pleased with the results. They're really promising, and what we can clearly say is that this, again, is one step further on our vision and dream of making Nanoforming big in the global pharma industry. Enzalutamide is a very big product. It's a fantastic product that saves many people's lives. It's been on the market some 10 years, and we have now been able to make what we see data showing the potential for an even better version.
You would take one pill instead of four pills, and you would still have the same, sort of impact on your life when you are a patient. Enzalutamide is only the first case as we see it, because we know that, the ASDs have been a very successful, way of bringing many drugs to the market. So as Edward said, there are 50 on the market, and there's probably 100 or more than 100 in the pipeline. We see, this as the sort of data that indicates that Nanoforming could be a alternative to many of the ASDs on the market.
Some of you analysts will certainly ask, "So what's the financial impact now?" It's too early to talk about the details around that, but as we all know, the pharma industry is an industry that when you have success, the financial reward is quite big... in addition to the fact that you help lots of people. With this, I'll stop and let Peter, our General Counsel, who is also very knowledgeable in intellectual property rights, continue a little bit to talk about the IP situation we are looking into. Peter, please go ahead.
Thank you, Albert. Yes, seeing this data come in is, of course, very encouraging and it really sets Nanoform as a credible development strategy to both improve current amorphous solid dispersion products and as the choice for APIs under development that would currently need to choose the amorphous route. Then from an IP perspective, already from the start, a key objective for this specific development program has been to leverage Nanoform's technology to create a unique IP position that enables both an improved product, which is, of course, important, but also one that is not in the scope of the originator ASD formulation patents.
And we expect that this may provide the opportunity to launch the product already after the expiry of the API patents on the product, but before the ASD formulation patents expire in 2033. And this sets also our product apart from generics based on the ASD formulation that the originator has developed. It is, of course, important that the product developed is also IP protected. We have a proprietary patent-protected process to manufacture the nanoparticles with our CESS technology, and we have filed additional patent applications for the nanoenzalutamide formulation itself. And all in all, I would say that this is a great milestone for Nanoform and all the people who have contributed with dedicated and innovative work. Christian, do you want to continue with a more commercial perspective?
Of course. Thank you, Peter. I think when we reflect on the clinical trial and what we have achieved, we set out to measure the comparative bioavailability of our nanoenzalutamide, which was in a crystalline form, versus the marketed product, which is Xtandi, which is an amorphous solid dispersion. We used a 160 mg single tablet to be equivalent to four by 40 mg tablets. Really, the drive was to improve the product profile, so it was more patient-centric. Patients that are suffering with diseases such as this often have to take many tablets in their daily regimen. So being able to develop a more patient-centric version for them clearly has value.
Not only is there a patient benefit, but there is also, as Peter quite rightly mentioned, an IP angle here. But the product itself, and this is an area where we see a lot of interest from some of our pharma partners, would be a lot greener. So we have a technology, the CESS technology, which is proprietary. It is a green alternative to the likes of spray drying and other amorphous solid dispersion technologies. We use recycled carbon dioxide. We don't use thousands of liters of organic hydrocarbon solvents to produce the material that we produce. So not only do we have the major patient benefit, but we also have a greener product, and this will help partners to reduce their carbon footprint.
I can speak broadly for all of the customers that we engage with. We now work with 10 of the top 20 pharma companies worldwide, and all of them are interested in reducing their carbon footprint and having more sustainable technologies for their pharmaceutical manufacturing. They are all focused on improving patient centricity. They want the most competitive products available to differentiate themselves in the marketplace. So when you combine these two together, I think what Nanoform has is really the next generation of formulation technology for the pharmaceutical industry. And as you can see here, this clinical results have clearly demonstrated our value and demonstrated that we can achieve better products for patients. And I'll leave with that note.
Thank you, Christian. Operator, we are ready for questions.
If you wish to ask a question, please dial pound key five on your telephone keypad to enter the queue. If you wish to withdraw your question, please dial pound key six on your telephone keypad. The next question comes from Christian Glennie from Stifel. Please go ahead.
Yeah. Good morning, good afternoon, guys, and congrats, and congrats on reaching this milestone. I guess a few questions, maybe start off with the trial and the data themselves. Obviously, at the moment, saying, you know, obviously promising results. Is there any... I mean, just trying to tease out a little bit more detail, if we can here, around the nature of the data, and if we can't, you know, when we should expect to get some data. And then just to confirm on the pill, the size of the pill, you've got 4x the amount of active. But is it comparable size of pill? 'Cause I understand these pills are generally relatively large, in the first place, even the 40 mg.
Is it comparable or potentially even smaller?
I'm happy to take that question, Christian. So, Christian speaking. Talking about the size of the pill, I think this is a great question. You know, it's very easy to make a larger pill, but it has to be a pill that's easy to swallow. And there will be companies trying to make a single tablet, but they're likely to be horse-sized pills that patients won't swallow and possibly won't even from the regulatory authorities. The size of our pill is comparable to the existing products that are on the market within the Xtandi tablets.
So we have achieved something quite remarkable in the fact that we can fit so much drug load into a single tablet, that is not possible with existing technologies in the marketplace. Could you remind me your first question, Christian?
Christian Glennie, you asked about the trial data.
Yeah.
As you can imagine, this data is just very recently into us, and therefore, we are still in the process of sifting through and combing through for details. I think what we guided previously, that we will have the readout completed during Q1 still holds. Important is that on the headline level, the numbers are already stamped, which means that we feel confident saying that we were very positively encouraged by what we saw. Details to follow, but many, maybe fewer than you would like to see for commercial reasons.
Okay, thank you. And then just coming to the, you know, the addressing of the underlying issue. I mean, do you have any data as it relates to, you know, dysphagia as a problem, the lack of, you know, adherence to Xtandi, in particular here, on the basis of this pill burden, that you know, obviously your technology addresses and therefore would be of interest to both a commercial partner and, or, you know, the regulators and the medical community?
So the short answer is yes, there are studies on it and of course, since this is a blockbuster product, there will be a lot of information that we are not even privy to. I think your question is really, is the pill burden a relevant thing? As far as we understand, yes, and within our own consortium, where people have been working with these kind of assets for a long time, the thinking is the same. Then when you come to the size, it's also well documented in the literature that the dysphagia is a real problem, both with the elderly and prostate cancer is a disease, prevalently in the elderly, but also as a result of many other comorbidities.
I think that the answer to both your questions is yes, we are addressing relevant problems, and of course, in an extension, we hope that being able to address them is not only commercially important, but also clinically so.
Okay, thank you. And then one final one, I'll get back in the queue. Obviously, turning... You know, you, you've still got to do the, you know, the further studies, but, you know, the key focus, I guess, starts to move to, to potential partnerships. You flagged some prospect there. Can you give a bit more of a sense of the nature of those discussions? You know, as it, as you think about the next... Obviously, the, the, there's a, there's a, yeah, everybody's agreed to pay 25% of future costs here in terms of development, but is there a scenario in which that partner may also take on the cost of, you know, the registrational trials?
And then just finally on that, I mean, it sounds like Astellas is the originator here, is not one of those potential partners, and if not, why not? Thank you.
Albert, would you like to take this both with the costs and then the partnering?
Yes. So, the cost is nothing has changed there, so we are continuing as we have said before. We are our four partners in this project, and everybody takes 25% of the cost, and therefore we will get 25% of the net income as well. Naturally, as we have said before, during this year we target to do one or several deals. The deals can be done either with the value-added medicine companies or with the originator.
What we have seen so far is that this is a very interesting novel technology that has now clinical data on a very interesting alternative, both on the sort of the green angle, but also on the patient angle, one pill instead of four, crystalline version of the pill instead of amorphous. So, based on what we have learned during the last half year or so, it's very clear that the interest in our technology and the interest in this project is clear. When it then comes to with who we will do and when we will do it, the thing is that, as we have said, we target to do deals during this year.
Peter, is there something else you would like to add, potentially, around?
Yeah. Maybe just to say that of course these potential discussions with different partnership opportunities are handled under strict confidentiality, and therefore we can't really go into the details of who we are talking with or what those discussions in detail look like. But as Albert very well put it, there is sort of interest there to the approach that has been taken.
Okay, thank you.
Christian, I also, of course, can confirm that enzalutamide and Astellas are, of course, very closely linked. And, as you're well aware, prostate cancer has a certain progression, and, both when it comes to disease and when it comes to, lines of treatment and medications. And it is, thrilling to us to see that we have now good data on, at least one of them.
Thank you.
I could actually add one-
The next question comes from Christopher Uhde from SEB. Please go ahead.
I apologize for any background noise. Wasn't able to plan this in advance. My question is related to some things that have been asked earlier. But I guess the salient issue here most immediately is, do you have anything you can say about whether or not, or how confident you are that you will not need to optimize the nanoforming process further for the nanoenzalutamide before going to a pivotal study? That's my first question. Thank you.
Okay. So, here, as we recall, there are two parts. One is the Nanoforming part, which is to make the nanoparticles. And, I'm fairly confident that we will not have to optimize the Nanoforming part. That's the first part of your question. The second part of your question is, we are very proficient in nanoformulations, and that is the reason why we are where we are now. And, when it comes to the nanoformulation part, I would like to first have this sort of detailed go through of the study, before we sort of make bold statements related to that.
Okay, thanks. And then the second, I guess, question I have is, I hadn't gotten this impression before, but... Well, let's put it like this, is it still possible that the originators of enzalutamide could do a deal with you for this project? Or is it, should we assume that the potential partners are more super generics-type players? Thank you.
So here you ask two questions in one question. From a purely logical point of view, the answer to your first part of the question, of course, it is possible to make a deal with both originators and not originators. Then the probabilities for how this will play out, we don't have a crystal ball, so we are unclear about those probabilities. Nanoform's approach is very simple. Our next steps now is to make sure that we can guarantee the security of supply in the upscaling of this program, and I'm very confident that we can do that. Albert, do you want to add something to this?
Well, I would just add what Peter said earlier, that in this industry, usually, you know, there might be NDAs and other reasons why we cannot talk too much about details. And of course, the fact is that the deal is, the deal or the deals are done when the deal or the deals are done, and before that, it's just speculation. What we have today is we are very, very happy with the promising results we have received. And I would like to add one thing related to the earlier question related to the ASDs and so forth. So this is one product within the prostate cancer area, and this is one where we have used StarMap, our AI engine.
As you remember in our Q3 report, we showed that there are many ASDs, both within prostate cancer, but also other areas, other disease areas, where we have utilized StarMap and where we have great opportunities to utilize the Nanoforming technology. Have a look at that also as a reminder about the link between StarMapping first, having the project, planning it, and then, of course, executing it, doing the Nanoforming, doing the formulation, and then doing the clinical study before you go and have serious negotiations with partners.
Maybe one more point, also, Christopher, this is a very interesting readout because as we have said before, the reason we entered into this study is that we had good animal data, and now we have, on a certain level, maybe proven is a too strong word, but still shown a link between the in vitro-in vivo correlation, by which I mean the correlation between the animal data and human data. And I think this is very interesting because, since this was done with the nanoformulation approach, this is also very unique data, globally now.
If there are no more questions, I would just end by saying that we are very-
Albert, I think there are more questions, actually.
Okay, okay.
I think, operator, are you there?
The next question comes from Sami Sarkamies from Danske Bank. Please go ahead.
Okay, thanks. Yeah, I still have a couple of questions. Firstly, starting from the actual study that's been in the making or conducted during Q1. Can you share how many patients have been involved? And how extensive measurement data has been gathered?
We cannot yet share the specifics of the study. There are many reasons for that. This year was carried out by a reputable CRO in Canada. It was carried out the way it's supposed to be carried out, and it has been analyzed the way it's supposed to be analyzed. The size is normal, the approach is normal, the dosings are normal, and the readouts are normal according to industry standards.
Okay, thanks. And then the second question would be on sort of the bioequivalence. This was asked earlier, but just curious, do you think you have already shown required bioequivalence and that there will not be a need to do any iterations? And I'm not thinking about now iterations on the kind of nano, Nanoform side, but iterations related to the formulation of the tablets, which could sort of be needed. So, do you think you're already there, or will there be a need to tweak the product, so to say?
So we are part of this own consortium, and as you can appreciate, this data is hot in from the press, and I think it would be too early to start to hear or talk about these details before we have a consensus within the own consortium, exactly how we're gonna play the game. What I can say is that, with the consortium members we have spoken, we have all been very pleased with the headline readouts and the sort of hard numbers that we got from that.
Okay. And then next question is, when, when do you think you will be able to share any data, or will that even be the case?
So as I said a little bit earlier, there are strong commercial reasons for being a little bit restrictive in how we publicly share data. So we will do a weighing here, where we want to serve the financial community on the aspects that are relevant, but we also want to maximize the commercial play that we can play, both on this asset and then also on all the other assets that are linked, since this is an ASD play now. Albert, do you wanna say something else?
No, I think you said it very well that this is an industry where in some cases, all data is published, and in some cases it's not, and we will do what is smart from a business point of view.
But of course, then at some point, all the data will be, will be available, but whether it's this study or, or then later studies, that's another discussion.
Okay, thanks. My final question would be on those next steps. So what will happen now? What will be the next phases of this program?
Yes, it's very clear. Step number one, look into the details of the readout, to understand exactly what we have now. What we have been talking about today is the sort of headline numbers... then make sure that we can guarantee the security of supply for going forward. That means that we will provide much more material than we've started to, think, you know, maybe up to hundreds of kilos, depending on the requirements from the regulatory. And then, make sure that we, design our commercial approach as well as we can. This is where the OnConcept come in. They have tens and tens of years of experience, doing this kind of, of place, and we will, of course, very, very keenly, listen and discuss with them how to most effectively play that game.
Having said that, one of the reasons why we can have those discussions is the fact that the technology has opened up this opportunity. Albert, anything you want to add?
No, I think that is a very good summary of the situation. And the good thing is, of course, that we have been preparing already for this positive outcome, and these promising results, so we are ready to step up to the plate in a sense. We have built the facility, we have built the factory, we have gathered lots of knowledge on nanoforming, and we are ready to do bigger amounts, and we are ready for upcoming pivotal studies, both in the U.S. and Europe, if so is decided, and in the coming years. And, as you all know, you know, when the patents, the API patents go off, even though it feels like it's several years ahead, it does not mean that there's lots of time.
There is lots of work to be done, in the coming years, and the good thing is we have started early.
Okay, maybe just one clarification. What will be the next clinical steps, I think, about the future studies that you alluded? What kind, and when would those start, and how will kind of the funding be tackled?
So, as we said before, there will be the idea is that you do the pivotal studies, and those you use then towards the regulatory bodies and to get the approvals. And we have the consortium where 25% of the costs are carried by each member. And then, of course, if and when we get partners, everything sort of the details of the deals will then be announced. And the industry usually works like this, as you know, when you make a deal, there can be upfront payments, there can be milestones, and then when the product is on the market, there would be royalties. And we see no reason why we would not target to make similar deals as is common in the industry.
The technology is new, but we, as you know, we have always used the industry's way of doing deals.
Okay, and can you still elaborate on those pivotal studies that, what would be kind of the objective in those? And, I mean, what's roughly the magnitude that you know, how many patients, you know, will they be long studies or short ones? Just to get a feel on, on, what's ahead.
If you think about it, a pivotal study is a study required for a marketing authorization application. In practice, it means that FDA and EMA will have the final say of exactly what they are gonna require and what they are gonna accept. To give you a flavor, if you go into Wikipedia, you can see that there will be requirements, of course, on the cohorts, on the sizes. You can also see what kind of numbers that are there. I think from the important part here is that since we are being material providers, these studies have two requirements on us. We must have the material needed, and we must have it when needed.
Earlier is always better than later because, you know, it means that you have a little bit time to correct if something needs to be corrected. My own guesstimate, I have not run a pivotal study before in this space, is that they are gonna be probably 10 x bigger than the one what we have here. Albert can give you more flavor.
Yeah, I mean, if you think about the one we have done, just we didn't give the exact number. So they are not patients, they were volunteers, and the size were not 10 and not 100, somewhere between. But the pivotal studies will probably be bigger, but they are not like they should not be enormous in that sense, because this is different from having an NCE that you bring to the market. But we will get we are not the experts, and Nanoform is not the experts in how to design all these trials and how it exactly goes. That's why we are very happy to be part of the ONConcept, and they have done tens of these tens and tens of these kind of studies.
They have a long history.
... lots of experience, so we will of course discuss with them and also the CROs that we use then.
Yeah. Just to make sure that I understood it correctly, we will be talking about bioequivalence when you sort of referred to these biological studies, that we're not talking about phase II, III type of kind of proof of concept study?
Yes, you are right. The costs are very different.
Right.
Much lower.
Yeah. Okay. Thank you. Thank you very much. I don't have any further questions.
The next question comes from Christian Glennie from Stifel. Please go ahead.
Hi, guys. Just a quick couple of follow-ups, if I can. I guess just the consortium, just to touch on very briefly, just get a bit of a flavor in terms of who these other players are, Bluepharma, Helm, and Welding. Bluepharma previously discussed, but Helm and Welding, new disclosure here. Just a bit more around, you know, what each of those guys bring to this consortium. Then I've got one more.
Sure. So I think that generally speaking, they have been in the space for a long time. They have been working, crafting, and selling dossiers. And Bluepharma, of course, have the ability to do the GMP processing all the way to something that can actually be dosed. So I think that these companies they are really, really experienced in value-added medicine games. I also think they have an angle to 505(b)(2) games. My understanding is that they have not been playing NCE games.
Maybe I can add to that, Edward. So, Bluepharma are a well-recognized drug product manufacturing company based in Portugal. They have clinical all the way through to commercial manufacturing capability, and the ability to handle highly potent molecules, of which in the oncology space, that's a necessary requirement, as indeed do Nanoform with our high potency capabilities as well. Together, Bluepharma, Welding, and Helm have a partnership called the ONConcept Consortium. They bring different strengths to that partnership. Bluepharma, obviously on the drug product development aspects and commercial manufacturing. Helm and Welding bring a lot of regulatory IP, CMC, and dossier experience.
They understand how to launch generic products, and they have significant experience in developing products, complex and differentiated products, in the oncology space. So when we got together and realized that, you know, enzalutamide is really a complex product, and that the challenge is quite great, combining technology into that partnership made a lot of sense. And, you know, we certainly don't know everything, and that's the point of the partnership. You know, we bring to this partnership the strength of the technology to really create something unique. We have Bluepharma that bring together the strength and knowledge around drug product development and commercialization of drug products.
And we have Welding and Helm that bring together a lot of API sourcing, regulatory, IP, and other commercial knowledge for making successful products hit the market and identifying the right partners. So by bringing all of this together between four companies, we really believe we've got something very powerful.
Thanks. That
And as he said in the press release, also, in the description about ONConcept, so they have more than 15 molecules that they are developing. And so you can read more there, and you can, of course, read more about the partners on the web.
Thank you. Then, I mean, just to confirm then, you know, the regulatory pathway, at least in the U.S., for this asset is most likely gonna be 505(b)(2), and it would be a, you know, product that somebody will have to promote. It's obviously not gonna be a generic substitutable product.
That's not something which we are disclosing at this point, in terms of the regulatory approval pathway. That's something that we can disclose more in due course.
Okay. Thank you.
There are no more questions at this time, so I hand the conference back to the speakers for any closing comments.
Thank you, operator. Thank you, Financial Hearings. On behalf of Nanoform, I would like to thank all participants for today. Thank you for the questions as well. If somebody has more questions, you know where to reach us, and we hope to see you all soon again. We wish everybody a great Friday afternoon and evening. Thank you and goodbye.