Alterity Therapeutics Limited (ASX:ATH) Chairman and CEO Geoffrey Kempler presents an overview of the company, discussing Parkinsonian disorders, progress with the company's lead drug candidate ATH434, the bioMUSE natural history study, and Phase 2 trial design.
Good afternoon to everybody, my name is Geoffrey Kempler, I'm the CEO and chairman and founder of Alterity Therapeutics. We're listed on both the Australian stock Exchange, as well as on the NASDAQ. And as just mentioned, we're developing treatments for neurodegenerative diseases. Our lead compound, that I'll talk about, ATH434, has successfully completed a Phase 1 clinical trial, already has orphan drug designation in both the US and in Europe, and is advancing toward a Phase 2 trial. I've worked in the biopharmaceutical industry for several decades now, and I've been involved with several different companies, but I've never been more encouraged or confident about Alterity than I am today, which is why I'm very happy to have the opportunity to tell you our story.
So next slide, please.
We'll move past the safe harbour program, which you'll be used to. Our purpose had been mentioned, but we really are there to try to create alternative futures for people living with these diseases that we target. These diseases have a tremendous debilitating impact on people's lives. You'll probably be familiar with the big ones like Alzheimer's and Parkinson's disease. I'll be talking about a special type of Parkinsonian disease today, but these are shocking outcomes for patients. We really want to have to disrupt that, and we have every reason to be confident that we can.
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So it's been a terrific year for us, and as terrible as COVID has been, it's actually given us an opportunity to really focus on what we planned for this year anyway, which was largely a planning period. So the impact of the disease overall, of COVID overall, has not been too big. It hasn't been nil of course. But we've had some good news recently, the US patent office gave us approval for a big new portfolio of molecules. It's a really big deal. It's why the marked responded so quickly to it and so strongly to it. And it's because basically it gives us the opportunity to really blow open our commercial opportunities across multiple disease targets over time. These patents give you several decades of protection, so they're very valuable.
We've also had the benefit in the last few weeks only of the support of our sophisticated investors in Australia and in the US and in Europe. And they've supported us with an oversubscribed fundraising, in fact, of $35 million that just recently closed. We've started our multiple system atrophy natural history study at the Vanderbilt University Medical Center in America, in Tennessee, it's called the bioMUSE study, I'll comment on that in this presentation. Our lead compound 434, ATH434 targets a protein in the brain, that protein is called α-synuclein. This is not a science presentation, but suffices to say that there are proteins in the brain that serve us very well when we're young and when we're healthy, and like many things, things start to go wrong either with disease or you can get age related diseases, and that includes neurodegenerative diseases. So what happens is that these proteins can start to, rather than be our best friends, can start to misbehave and become toxic to our bodies instead of useful to our bodies. I'll explain a bit about how that happens.
We've been to the FDA on more than one occasion and we've been very encouraged by this year's meeting that we had with them during COVID. It was meant to be a face-to-face meeting, but it became just Zoom face to face, but we're able to go through our development pathway to get their support for what we're trying to do, and that was very important for us as well. We've also had a chance to represent our Phase 1 data at some conferences, which has been very useful. And we had some very strong reactions to Phase 1 data, which perhaps even stronger than we would have anticipated. And I think it was for two reasons. One is that we will demonstrate that we could get our drugs across the blood brain barrier.
That is not a tiny achievement. The brain is a very privileged and protected part of our bodies. We have a big skull on top to protect it from falling rocks, but we also have membranes inside to protect it from any of the wrong chemicals getting into it. So it's a specialty to get drugs across that barrier. And we're also able to demonstrate that we could get it to the target in concentrations that we're optimistic about. Other agencies besides the FDA have spoken to us, including the regulatory authorities that issue orphaned disease status. We've had two shots at this, we've been successful both times. Once with the European commission, as well as with the US. US is not on this slide because we announced that last year. And so we've been very pleased about all of those achievements this year, despite COVID.
So next slide please.
So as I mentioned, we're dealing with Parkinsonian disorders. The word Parkinsonian of course puts us into the camp of Parkinson's diseases, which I'm sure our listeners would mainly associate with movement disorders. And there are some specialty subsets of this. If I was to explain it in non-scientific terms, that would be a problem with this protein, but it's not just limited to one small part of the brain, but you can get it in multiple parts of the brain, hence multiple system atrophy, because it can affect different parts of the brain all negatively, all with shocking impacts on the patients. And so our goal is to be able to really address these major disabilities.
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So this slide may look a little bit busy, so I apologise for that. But drug development desk doesn't happen in one hit. It is actually a process and we've made excellent process this year and last year. In fact, since I last presented to this forum. I think very excitingly, we've got some very strong, robust efficacy in animals. As you'd know that in science, you begin in the lab, you go to animal models, and then eventually you go to humans. But there's animal models and there's animal models. And these are good models. We've done it in more than one type of animal model. And we've got a lot of confidence that we should be optimistic, that this will translate into humans.
And I think particularly when we look at that second box that we've completed Phase 1, which as we all know is really about safety, but we're able to escalate that so beyond safety, we could actually see that we crossed the blood brain barrier and that we got to the two concentrations that gave us some enthusiasm. And these are the sorts of things that drug developers look for along the way so they know that they've got increasing confidence of success.
We've also taken our drug out to speak to experts in the field so they can understand the mechanism of action. We've spoken to movement disorders specialists, including specialists in MSA, multiple system atrophy, as well as speaking with general neurologists. I'll speak about it a bit more later, but it gives us confidence in the usage of the drug and the breadth of usage of the drug as well, which will give us confidence behind the numbers that we're projecting as revenue. As I said, we've got a variety of things we could have gone for, but we've selected multiple system atrophy as the one that we think we're at the best shot of getting approved. I've mentioned that we've had the orphan designation approved and we're well underway to get our Phase 2 trial started next year. And I'll explain some of the steps that we've done to get there.
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So this is, in fact, the trial that's already started. It's at the Vanderbilt University Medical Center in Tennessee, in America. Its objective is to actually look at a group of multiple system atrophy patients. At this stage they're not getting the drug, we're just observing them. And you can see from the second bullet point that we'll be identifying biomarkers that will become the main thing that we'll be looking for in a treatment Phase 2 trial, which will be a full cover blind study. The FDA is interested in the way we're approaching this, which is why we've had meetings with them so that we can really study a bit of the natural history of this to pick the end points that we think will give us the greatest chance of getting an approval.
And you'll notice under biomarkers, I'm not going to go through all of this clearly, but you'll notice under biomarkers, it refers to iron content. This is iron in the brain. Iron is a metal. You're born with it. Once that protective blood-brain barrier closes after a few months when you're a child, a baby, in fact, that's your compliment of iron for awhile. And it's when iron starts to misbehave later in life that you can get these proteins that are the good guys, usually, becoming toxic. So that's one of the things that we'll be measuring as well as these clinical end points as well. As I said, this is not a deep scientific presentation, but we're here for any further follow up questions for any scientists on the phone or anyone who wants more detail.
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So this is the Phase 2 trial that will come on the heels of the natural study that I just mentioned. It is a randomised, double blind placebo controlled trial. It's really a trial, once again, to build on the efficacy, but primarily its objective is to see if the drug works. And we're going to maximise our chance to success by making sure we picked the best end points to actually test that. Currently we're planning for 60 patients for six months. It'll be in Europe and America. You can see that we're going to have two doses as well as placebo. And once again, all the end points that we'll be picking from to go into that trial.
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So I was insinuating this, but now I'm very pleased to say that after some very high level research in the market with actual prescribers, doctors who will be writing scripts, you can see the projected sales there of up to $725 million. I'll emphasise this is in US dollars, and it's only in America, and it's only for multiple system atrophy. Clearly we will be marketing it globally, and because of the way that the drug works, if it treats multiple system atrophy and gets approved for that, it's highly likely based on the feedback from the physicians that they're going to have a look at its usefulness in other Parkinsonian disorders, including Parkinson's disease itself. So we're very comfortable putting this up as a highly conservative estimate of peak sales for our drug just in the US and just for this indication.
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So this is a slide that I'm the proudest of in the sense that it took a long time to achieve, to really get a crack team together. And the beauty about introducing my Chief Medical Officer, David Stamler, is I'm introducing a man who's actually had three FDA approvals for brain diseases. The last was in Huntington disease. That company where he was doing this was Auspex, it was sold for US$3.5 billion as you can see there. We didn't just succeed in bringing David to our company, he actually brought the key players along with him. So we have a team that has done this before several times. It's not an easy area, neuroscience, but it's very doable in the right hands. So I'm very pleased with being able to share this information with you.
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So in fact, my last slide, just to recap the things that I've said, that it's an unmet medical need for a major category of diseases, even though the first one that we picked is multiple system atrophy. We've received the imprimatur of orphan disease status both from Europe and America, and we have peak sales have explained that they are more than enough to justify an investment, but certainly quite conservative in the way we've presented that. A very successful development team that's being welcomed into discussion with the FDA, which is important. The drug is advancing well. It's in the trial at moment in the natural history study at Vanderbilt in America. It's had its Phase 1. I've mentioned the animal model there.
That novel mechanism, we can speak for an hour adapt that term novel mechanism targeting, that would be the science talk. But I think what matters when you heard things like oxidative stress talked about in the world, we're actually doing things in a very unique way. We're really addressing the root cause of these diseases. We've built up a very large level of expertise over many years through our contacts at Harvard University, where one of our founding labs was, and at the University of Melbourne. And now Vanderbilt University is another group that are helping us along the way as well. We'll be able to report data about the efficacy of the drug, fingers crossed for that, in the second half of 2022.
Because of these new patents that have just been approved, we have a strong pipeline of potential to really blow open commercial opportunities over the longer term. And because of the fund raising that we've just completed, I'm very excited to be able to say that we've got a strong balance sheet, which is important for any CEO to be able to say. Unfortunately because of the virtual nature of this presentation I can't take Q and A now, but my contact details will be available and I'm very, very happy to... in fact, I hope you can ask me lots of questions and we look forward to answering them. Clive, thank you to you and thank you to all your listeners.