Prescient Therapeutics Limited (PTX) Earnings Call Transcript & Summary

Good afternoon, everyone. Just ticked over midday, Eastern Standard Time. So we'll get the session going. While people are settling in, I'll just go through disclaimers and some housekeeping. Presentations that we put on are suitable for self-directed investors only, people that have got the experience and capabilities to do their own analysis and make their own informed decisions. Any information contained in today's presentation is general in nature. It does not consider your personal circumstances. You need to decide for yourself whether it's appropriate for you. The information we're giving is for educational purposes. Past performance is not a reliable indicator of future performance. Now with all that said and done, welcome to Prescient's investor and shareholder update. My name is Patrick Nelson. I'm the MD at Reach. I'll host the session today, but we're joined by James McDonnell, the CEO of Prescient, to run the session. We scheduled today's session off the back of the major development this week, which was the FDA granting Fast Track designation for PTX-100. Now PTX-100 is one of the most advanced cancer therapies on the ASX. And this is a crucial step towards being in a position to turn the IIb study into a registrational study. This is a huge development. Today, James is going to step through some of the slides to give you a run across PTX-100, the cancer it's treating, the results of the trial to date and the market opportunity. And then we'll get to the Q&A and ask a few questions about the FDA Fast Track designation. Now we expect the session to run for about 20, 25 minutes. [Operator Instructions] Just a quick introduction to the company before I hand over to James. PTX are making significant strides in addressing critically unmet medical needs for cancers that are presenting -- or for a cancer that is presenting effectively a death sentence for those who contract it. 64% of PTX patients on the trial are now experiencing a halt or a reversal of tumor growth. And as such, they have strong backing from the FDA, including Orphan Drug designation, IND, and now receiving FDA Fast Track designation. This unlocks increased access to the FDA rolling submissions on new drug applications. And this is why they had the opportunity to make their Phase IIb, their registrational study, which they are progressing rapidly along the pathway to commercialization, representing one of the fastest commercial pathways in oncology drug development on the ASX. The initial market for T-cell lymphoma treatment is approximately 27,000 patients with a $2.9 billion market. It's a really exciting time for PTX. So James, you should now have control of the slides. I'll hand over to you.

Thanks, Patrick, and it's a pleasure to be here to discuss the FDA approval of Fast Track for PTX. It's a very, very, very good time and sits well with our strategy. So here is our disclaimer and safe harbor statement. And so just a quick summary. In fact, Patrick has already given you pretty much of the summary. So we are an advanced cancer therapy and we have a high mortality -- focusing on high-mortality cancers. So FDA Orphan Drug designation and an IND already, and now we have Fast Track designation. So the market is reasonably sized at $1.8 billion in terms of the top 8 markets. So we're looking in a good space. The results from the Ib is what really is exciting with a strong response, with 64% of patients either experiencing stable or improved disease, extended period of time for that response and a favorable safety profile compared to the others. So what we have is really good Phase Ib results, and now we're looking at the Phase II. Why are we in this space? Well, how does PTX work actually? It actually disrupts geranylgeranyl transferase, which is an enzyme which is important to the RAS family pathway. And RAS is involved in around 22% of human cancers. So by targeting GGTase, we end up blocking this pathway. And what that does is it increases the cancer cell death and reduces the growth survival and migration of cancer cells. So an interesting pathway, and we are first-in-class, which I'll come back to later. So we have a study starting in cutaneous T-cell lymphoma. And this is a rare cancer of the white cells, the T cells, where the cancers divide uncontrollably in the skin environment. There's a number of subtypes, but 2 common subtypes are mycosis fungoides and Sézary syndrome, and they are graded between 1 to 4. And in our study, we're looking at refractory/relapsed patients of IB and above. So these patients have very, very few options in terms of the space. So we see in the U.S. alone, there are 3,000 new patients each year. So the market is significant there. You can see the pictures here of a patient with T-cell lymphoma or cutaneous T-cell lymphoma, and it's not pleasant. And so their quality of life is also very poor. Not only are they battling their cancer, but they're also battling a lot of social isolation and things like that, so a challenging disease. I had the opportunity to spend some time with Prof. Miles Prince, who was our lead investigator in the Phase Ib and is, in fact, our lead investigator in Phase II as well. And he's very excited with the Phase Ib results. And if you haven't had a chance to look at his Q&A on our website, I encourage you to do that. We do have a snapshot of that Q&A coming up. And I will hand over to [ Corey ] to actually take you through that. [Presentation]

Thanks, [ Corey ]. So you can see Miles is very keen to find an appropriate treatment for his patients who are refractory/relapsed. And he has already seen some significant results in the Phase Ib and he's looking forward to seeing more in the Phase II. So why the excitement about the Phase Ib? Well, if we look at the results, you can see from a benchmark, which is what we -- the minimum of what we'd expect before we progress the study, Lymphir, which is a drug which was approved on the FDA recently, and PTX-100 Ib. You can see response rates across there, Lymphir approved at 36%, Phase Ib had a 45% response compared to benchmark. In terms of our clinical benefit where you have CR, PR, plus stable disease, 64% of patients achieved benefit. And these are patients that have been in a progressive disease environment, have either stable or improved disease, so that's promising. Duration is also important. It's no use having a response that doesn't last. And you can see the response rates for PTX-100, compared to benchmark and others, are super good. And then severe adverse event profiles, typically, we expect more than 30%, and Lymphir was approved and had a 36% severe adverse event rate, but PTX is 4%, which is an unusual thing to talk about. And when we compare -- why am I talking about that? Well, because it is favorable compared to our other competitors in the market space. And you can see in this graph that there are significant adverse events with other products in the space versus PTX-100. And so this favorable safety profile actually has advantages because in the T-cell lymphoma forum recently, I heard -- or there's another discussion about combination therapy. The CTCL and TCL tumor microenvironments are very active. And so clinicians feel like they're treating one thing and something else pops up. And so they're afraid of trying to do different things. And so their focus is now looking at combination therapies. So if you've got a treatment that is not further affecting the patient who is already fragile that offers an opportunity, it becomes a very nice product to look at and move forward with. So what's our rationale for choosing CTCL for our Ib study, which is a TCL one study? So firstly, we've got a high confidence in this from our responders in the Ib. There's a greater need for new therapies. The sponsors are not in the space. And so therefore, there's a very big need for it. And we've seen, having our approval in terms of fast approval, fast -- yes, anyway, it's very helpful. So they're likely to recruit faster a larger patient pool and the cost of the study is less. So we do not ignore the PTCL patients. So we see that they're more taken up in the large Phase I studies in lymphoma. And if we did run a study there, we would find that would be too large a study and that, with the comparator, will be quite messy. But we're not forgetting these patients. So we're looking at an investigator-led program. And the reason we're doing that is for the U.S. market where you can actually -- if you gain a compendium listing, you then become able to be reimbursed through the payer and the insurance schemes based on that compendium listing. So compendiums have approved and unapproved products in there. And so they become eligible for reimbursement in that space. So it's a unique thing with the U.S., but it's very, very important. So progressing to our Phase II with PTX-100, here's the schema. It's actually divided into 2. And that's a very, very good thing, particularly with our Fast Track designation now. So firstly, the first cohort of patients will be enrolled, randomized into 2 doses. And the reason for this is the FDA have a program called Optimus, and they prefer an optimized dose and reasons for that. And so we have a dose optimization committee, which will assess those patients. We say 40 patients, but it may actually be less than that depending on the results coming through. It's an open-label study. So we'll be able to see the results based on what the dose optimization committee inform us. We then get to that Phase IIb, a larger study, but we get there at a point where we're likely to have efficacy. We already have Fast Track designation. So we're talking to the FDA. And so this becomes the opportunity for a registrational approach moving forward. And the FDA have obviously recognized this is an unmet need with our Fast Track designation. So just how we go about the registrational pathway? This is a typical pathway for a large study. And so you go right through the Phases I to III, NDA, marketing authorization, on the market. But for smaller studies, for smaller patient populations with unmet needs, there's a potential other pathway, which we now are able to engage with in terms of our Fast Track designation and speak to the FDA about the potential of using a different approach. And so using these approaches means we save time and money. And so when I'm talking about why the IIb is such an important inflection point in that study environment is that's where we'll be talking to the FDA about what are our likely changes we can make to the protocol, protocol amendments that we can submit that could make this a registrational component and move us through this fast approach to being on the market and commercialization. So we're able to do that because of these results. So great response rates, clinical benefit, duration and also that adverse event profile. So that gives us the opportunity. And this is the data that the FDA were looking at when they were considering the Fast Track designation, and they have since given us that designation. The other component we have from the FDA support -- the support we have is the Orphan Drug designation. And so this is a 7-year guaranteed market exclusivity in the U.S., and it's actually 10 in Europe, and so we'll be looking at that as well. So you get great prices, a consistent market. And uniquely to the U.S., you also get as well as the growth of the market, you get an opportunity to change the price. And so you can see the CAGR for Orphan is also higher. In our T-cell lymphoma market, where we're looking and heading, you can see the addressable market in 8 major markets is big. These patients are often all are relapsing, most likely. And you can see $1.8 billion estimate for the year 2030. And the component of that is 67% of that is in the U.S. So the U.S. is a very important market. In the CTCL U.S. alone situation, we see an incidence of 3,000 patients. And again, they are relapsing. The combination therapy is becoming very keen in that space and that estimated potential is relatively -- is pretty high as well, so a very important large opportunity. And now we're down to our milestones, what we've achieved. And you can see that this is a slide I presented 2 weeks ago. And already, we actually have achieved the FDA Fast Track designation, which was slated for Q2, earlier than we anticipated, which was a great surprise. So we still have first patient in U.S. sites, European sites and the continuous disclosure of the Phase IIa data because it's an open label, and we have a dose optimization committee reviewing that process. And we have some cell therapy milestones. So I mentioned at the beginning that we are first-in-class. And the reason I mentioned that is because we need to look at that first-in-class approach and how that can impact other cancer states because we obviously have proof of principle in T-cell lymphoma. We know that RAS is involved in 22% of all cancers. And so here's an example of where RAS and the cancers that are involved are there. So we will need to do some -- a lot of work to really look at how we embrace this opportunity. We have an experienced team to do all this. And so you can see the team there, our management team and the Board of Directors. And you can see the companies, which we've all been through, and have gained the experience to enable us to be able to take this PTX-100 through to commercialization and what the deals are that we need to make. So quickly summing up, you can see it is an inflection point that we're driving. We see the Phase Ib study, 64% of clinical benefit, good response duration and really, really strong response that moved us into the Phase IIa. It's a global site. International experts are involved. And you can see we have FDA support to date of the Orphan Drug designation, the IND acceptance and Fast Track designation. We also know that the FDA are really interested in sponsors that are looking at unmet needs. And so we feel there's potential for that registration. And with our study, our Phase II study, set up between IIa and IIb, that inflection point within the study, the ability to communicate, it's really, really positive. The market sizes are significant. So it's a great environment to be in and a great strategy that we're invoking. So I'm happy to take questions. As we move forward, as you can tell, I'm quite excited about this process, and I'm looking forward to taking your questions.

Thank you. And the question box, if you type in there, they will come through to me here. So James, just on Fast Track designation, maybe we'll just go back to that for a moment, what are some of the key things that happened by virtue of getting Fast Track designation that are helpful for you moving towards that registration study?

Yes. Well, Fast Track designation really provides us an ability to communicate with the FDA. And that communication, which you typically don't have, means that you get to discuss study results of the Phase IIa and looking at progression, the dose and things like that. We satisfy Optimus with the dosing, and we have good equivalent sort of response rates from the Ib. It will be a very, very good conversation with the Phase IIa. The other component with the Fast Track designation is that you can also do rolling NDA. So what that means is that they -- the FDA can look at part of your dossier ahead of the clinical response that you get. And that speeds up -- so it's all about expediting the drug to approval so the patients can actually access it. So there's a lot of advantage with the Fast Track designation.

Thank you. FDA also, you've got IND and Orphan Drug designation. How does this come together with those programs?

Well, if you put it in order, I guess, you get the IND acceptance. So they were really happy with that study, and so they have agreed to that. And then you have Fast Track designation. So we get continuous conversations with the FDA to really see how we flow through that Phase II study from a to b. And then at commercialization phase, Orphan designation provides us that 7 years of market exclusivity in the U.S., and that's really important. The important factor there with the Orphan Drug information is that we need to be ready for that commercialization step. So we will have a lot of BD or activity as we go through the Phase IIb, if it indeed is a registration study, preparing ourselves for that Orphan approach.

Yes. And further to that, what sort of needs to happen in this IIa stage so that IIb becomes a registration study?

Yes, it's a good question. Firstly, we're able to communicate, which we weren't able to before, and so that's a positive. The second is the data that we get from that study, resembling the Phase Ib would be fantastic. And thirdly is that we define a dose. And so with those 3 aspects, we have that communication and ability to talk about accelerated pathway, which would be a registrational approach. And so it really is fitting in with our strategy. We described the strategy a couple of weeks ago and with potential of Fast Track designation. Now we can say we have Fast Track designation, and that's a big plus.

Yes. All right. So I'm laboring this point a little bit, but just digging into this registrational study, I mean, what does it mean in terms of getting to the point where you're marketing your therapy? What does this IIb -- if you receive IIb registration, what does that mean for Prescient?

Yes. A good question, actually. What that means is that the registrational study is providing the clinical input to the new drug application that will be submitted to the FDA. We know that with the Fast Track designation, they can do a rolling submission, so they might look at the CMC components and other components of the dossier as the study is progressing. And then with the completion of the registration study, we can add that clinical component to the new drug application and therefore, move towards an accelerated approval.

Okay. So what would you compare -- like, who could you compare yourself to? Because obviously, registrational study is a major sort of inflection point for Prescient. Can you compare it to any other similar-type therapies or businesses?

Yes. I mean there is a company called Mirati, which recently, in 2023, I guess, was sold to BMS. And they had a RAS inhibitor, which went through the Phase I with Orphan designation. They had a Breakthrough Therapy designation, which is an expedited designation, in similar terms to Fast Track designation. And so they were following that process. And then they went to an accelerated approval and then reached the market within a fairly short period of time. And then BMS acquired them 10 months after that accelerated approval. So they were in -- so $4.8 billion, but what I'm -- they're in non-small cell lung cancer. But it's the process that we could compare here, that they -- you go from the Orphan designation, you go through to an expedited designation and then accelerated approval, and then to market. So you get to the patients much quicker and commercialization opportunities and therefore, BD opportunities.

So relative to where we are today, the next step would be registrational study. And then, I guess, at what point are we in this process relative to Mirati?

Well, we're really at that -- the Fast Track designation, which they received, was about between their Phase I and Phase II trials. And so we're in a very similar spot. And so like them, we will be able to communicate with the FDA and really look at the data coming from the Phase IIa to engineer, if possible, the type of study we do in Phase IIb. So it will require a protocol amendment, but the FDA will be heavily involved in the process now that we have a Fast Track designation. So that's where you can talk about accelerated pathways.

Okay. And then with a USD 4.5 billion acquisition, a similar-sized market, and yes, so that's interesting. So [ Stephen ] had asked the question, what point do pharmaceuticals and large funds managers take interest in what we're doing? Well, I think that some of the bigger insto investors probably about now, [ Stephen ] in terms of the pharma, probably at around the point of a registration study. James, do you have an opinion?

Yes. I mean I think when we're in this phase, it's a defined pathway. We're ticking the milestones along the pathway. So we become more open to that. We're more into the institutional base type attractive phases, I guess.

[ David ] asked, what were the frequency of feedback to shareholders regarding the IIa trial results of patients. Have you got an answer to how frequently or at what points will those updates be provided?

I mean, we're expecting something around the 20-patient mark, which was half, but we will be relying on the dose optimization committee to provide that data and see. But like you, I'd be very excited to see as much data as possible. But there will be certain points at a time when we look at that. We need to look at it as a whole, not individual patients and those sorts of things. So we do need some patients in there to create reasonable data.

Beautiful. Now has anyone got any further questions for James while we've got him here? If not, I'll call the session to a halt in a couple of moments. Well, I'm not seeing anything come through, James, thank you very much for the update today. And congratulations, fantastic news. You must be very excited. And look, there's quite a few milestones coming up. So as news is released, we'll run another session like this and might do another shareholder update in the coming weeks. So James, thank you very much. Thanks to everyone for taking the time and joining us today. It's greatly appreciated. I'll leave the last word to you, James.

Thanks, Patrick. Well, I just want to say thanks for joining today for the webinar. It's a privilege to have you here. In the words of our Chairman, we have intellectual capital in terms of PTX-100. We have people capital as we move our people into Australia and create a really high-performing team. And we have financial capital. So we have a great strategy, we're really focused, and we're moving forward with our program, which is very exciting. So I think there's never been a better time to be a part of Prescient, and I look forward to your continuing support. Thanks very much.

Thanks, James.