Alterity Therapeutics Limited (ASX:ATH) CEO & Chairman, Geoffrey Kempler, discusses the company’s development pipeline and lead drug candidate PTB434 for use treating Multiple System Atrophy (MSA).
Katrina Bullock: Hello. I'm Katrina Bullock for the Finance News Network. Joining me today at FNN's Investor Event is the CEO and chairman of Alterity Therapeutics (ASX:ATH), Mr. Kempler. Thank you for joining us.
Geoffrey Kempler: Good morning. Thank you for having me.
Katrina Bullock: Now, first up could we start with a bit of an introduction to the company?
Geoffrey Kempler: So, Alterity Therapeutics has spent the last several decades understanding some diseases that affect people while they're ageing, as well as generally neurodegenerative diseases that can affect people's brains, unfortunately, with loss of cognitive function, loss of motor function. There's a wide variety of diseases but they all have one thing in common, and that is the presence of proteins in the brain that serve us very well when we're young and healthy. But these proteins can misfold, and form clumps in the brain. Different proteins, depending which disease we're looking at. But today, I'm going to speak specifically about Parkinsonian diseases. And, what our approach is to try to bring a profound difference to people's lives with those diseases.
Katrina Bullock: I understand it's quite difficult to get approvals, particularly for brain diseases through the USFDA. Does your team have any experience doing that previously?
Geoffrey Kempler: Well, they've had multiple drugs approved for brain diseases. Huntington disease, Tardive dyskinesia and other type of disease of the brain. So they've had a lot of experience. But the other thing that's worthy of note is that the often designation that was given to us by the FDA says several things. First of all, it gives us patent protection for a specific period of time, and we expect to get the designation approved in Europe as well. It also means that they've actually understood that what we're doing could work, even if it's at this theoretical level at the moment, and that they think it's worth supporting it. So, the orphan designation gives us regulatory assistance, will also affect with the timing, and help with our interactions with the FDA as well.
The market size that we're looking at beyond the number of patients that we're trying to treat is very substantial, because these orphan diseases, or rare diseases track very high pricing. We've had independent assessments that says for our primary indication of this particular, atypical Parkinsonian disease that we're targeting, called Multiple System Atrophy, we anticipate peak sales of $750 million, just in the US, it's possible that even people with some more general Parkinson's disease, their doctors might choose to prescribe it to them as well.
Katrina Bullock: Geoffrey, can you tell us a little bit about the size of the market for your lead drug candidate?
Geoffrey Kempler: In order to justify the investment in this drug, we've had to do a thorough commercial assessment. Once we secured the orphan disease designation from the FDA, we got a stronger view on what the potential sales are.
Katrina Bullock: Thanks, Geoffrey. Now focusing in on your lead drug candidate, PTB434, could you tell us a little bit more about that, starting with the clinical studies?
Geoffrey Kempler: So, we've just finished our phase one clinical trial, it was in about a little under 80 patients in multiple ascending doses, single ascending doses. The drug's proven to be very safe, so far. Our chief medical officer, Dr Stamler presented the data at the American Academy of Neurology just a few weeks ago in Philadelphia. We're also pleased that our drug showed that it could get through the blood-brain barrier, that is no mean feat. The blood-brain barrier is actually designed to keep things out, so you have to design drugs particularly so they can get through and we're able to get it into the brain and we measured it in the CSF, the spinal fluid and we saw it in concentrations that were high enough to create efficacy in the pre-clinical models that we saw.
Geoffrey Kempler: So, so far, the drug's looking very promising and we're excited to start our phase 2 trial
Katrina Bullock: Has Alterity looked at other applications for the drug?
Geoffrey Kempler: Absolutely. Our drug targets the alpha-synuclein protein in the brain, it also targets the tau protein in the brain. There's a lot of complex science between those two proteins, but it does give us a multiplicity of diseases that we could target over time.
Katrina Bullock: And now turning to financials, can you give us a comment on your funding, and your share price over the past 12 months?
Geoffrey Kempler: Recently, we secured funding from a group called Life Biosciences, based in Boston. They're not a bank, they're not a venture capital firm, they're actually specialists in healthcare. They're particularly interested in anti-ageing and at our recent AGM, they were asked why did they invest. And it turns out that the principles that we're using to treat our disease targets, may also help with ageing in some parts of the population. So that's not what we're about, but that was one of the things that attracted them.
But, it's very clever money, it's a real imprimatur for what we're trying to do.
Katrina Bullock: So, last question Geoffrey before I let you go, why should investors consider investing in Alterity Therapeutics?
Geoffrey Kempler: So, first the caveats, obviously healthcare is difficult and brain diseases can be very, very difficult. However, I personally have continued to invest, because I'm very confident about our science, I'm very confident about our development team. I'm very confident that the population with these diseases desperately needs better treatments. For me, it's an opportunity to do good personally, by doing good for patient populations that aren't being treated at the moment.
Katrina Bullock: Geoffrey Kempler, thanks for the update.
Geoffrey Kempler: Thank you.