Prescient Therapeutics Limited (PTX.AX) Earnings Call Transcript & Summary

Good morning, everyone, and welcome to Prescient Therapeutics Investor Briefing. While everyone settles in, I'll do the usual disclaimer housekeeping. So the presentations that Reach put on are suitable for self-directed investors who have the experience and capabilities to make their own informed decisions. All the information contained in today's presentation is general in nature. It does not consider your personal circumstances, and you need to decide for yourself whether it's appropriate for you. And you should treat the information as educational and to give you the ability to make your own decisions. Past performance are not a reliable indicator of future performance. Now my name is Patrick Nelson, I'm Managing Director at Reach. I'll host the session today, but we're joined by James McDonnell, the CEO of Prescient for this, which is a Q&A session. And we aim to get an update from the IIa -- sorry, this is off the back of the recent 4C announcement. And from this session, we'll get an update on the IIa CTCL trial, including recruitment, the path forward and decisions being made around where they head from here and then broader discussion around their platform, their technology and so forth. So feel free to ask any questions by putting them into the question box. I've got a range of questions to run through, and I'll keep an eye out for questions as they come through. We'll get to all questions throughout the session. So I think this session will run for approximately 30 minutes. And look, what I might do, first of all, is just set the scene for anyone that's new to Prescient. It trades under the stock code PTX on the Australian Stock Exchange. And they've developed a unique technology that can stop cancer from reproducing. It does this by attacking biochemical processes referred to as the Ras pathway that are behind 22% of all cancers. The first cancer that they've sought to treat is Cutaneous T-Cell Lymphoma, CTCL, which is a cancer that is considered by many clinicians to have no viable treatment, therefore, effectively a death sentence. In the Ib trials for CTCL, an astonishing 100% of patients had their tumor or the cancer stop growth or reduce. And very importantly, 0 patients experienced any adverse events, which particularly important as a lot of cures for cancer can be worse than the cancer itself. Now the CTCL is now in their IIa clinical trials. They've received FDA Fast Track designation, Orphan Drug designation. And in the last quarter, the European Medical Agency, EMA, also gave them Fast Track. So this is one of the most advanced cancer therapies on the ASX with Fast Track designation and the opportunity, which sort of gives the opportunity for the IIb clinical trials to turn into a registration study. What they've got is a revolutionary technology that stops this Ras pathway, which controls the cell growth. And PTX-100 can therefore halt cancerous cell growth and can be applicable to cancers that have that Ras pathway involvement. So they believe this can be applied to numerous different types of cancers beyond CTCL. And therefore, this technology platform has the potential to fight many cancers and help millions of people. It's very exciting. We're delighted to be involved with Prescient.

So James, thank you for joining me today. Maybe we start off with how would you explain to investors what Prescient do and what they provide?

Well, we are an oncology company. So we look to find and develop technologies or therapeutics to treat cancer. PTX-100 is our first technology or therapeutic, and we're leading the way in CTCL at this stage.

Yes. So maybe how does this technology actually work?

Well, it actually -- it disrupts the Ras pathway and Ras is involved in 22% of all cancers. So by disrupting it, it means we actually turn the switch off. So the Ras proteins are unable to turn on the oncogenic pathways. So that's how it works.

Yes. So in order, how have you to date applied this technology? And what have you -- where have you focused your energy?

Well, currently, we're in a Phase IIa for CTCL. So what we saw in our Phase I study was a really good signal in PTCL and lymphomas, those sorts of lymphomas. So we're focused on CTCL now. We feel that, that's an appropriate way forward for PTX-100. We've received Fast Track designation from the FDA, orphan designation as well. And also, as you mentioned, orphan designation from the European Medicines Agency. So all very helpful in the development of PTX-100 and CTCL.

Yes. And what were the results? I mean, you touched on, but the results from this Ib with, I guess, the last readout. Can you talk us through those results?

Those results were very encouraging. We saw 70 valuable patients, 43% response rate, but all of them received a clinical benefit. So in CTCL, clinical benefit is considered very helpful because these are progressing patients. And so the other component there was 0 adverse events or serious adverse events attributed to PTX-100. So not only are we finding good results, we're also not adding to the complications of these patients. And that will be very helpful downstream when we look at combination therapies.

And for the -- why did you choose CTCL? Like what was it -- why go after that particular cancer?

Well, CTCL, there's 2 main subtypes, whereas with PTCL, there were many subtypes. So it means that we can have a more directed study and have a result more clear as we progress forward. Our Phase IIa has 2 different doses. So once we define and improve the dosing based on responses and activity, we'll move forward with IIb. And that depending on those results could be quite interesting in terms of maybe pivotal, maybe not.

Okay. And so the FDA gave you that Fast Track and the Orphan Drug designation. What are they -- like -- I mean, what's the benefit of going after a therapy that was likely to get that if you could prove you were getting good results.

We talk about Fast Track, Fast Track is actually an expedited pathway. And that means that the FDA recognized that CTCL is a clear unmet need and that PTX-100 seems to have -- based on the Phase I data, had good activity. And so that means the FDA want to get these active therapies to patients sooner. So it's potential to have an accelerated pathway based on data, based on our results in the Phase IIa, and that's what we're really focused on.

Yes. All right. Sorry, someone mentioned there was some echoing, and I hope we've just done something that fixes that for you. All right. Now let's move on to the IIa trial and the recruitment of that. So I guess, why is recruitment so important and such a challenge for cancer trials?

Well, recruitments obviously you need the data and patients provide that data. But this is a rare disease, and patients have failed multiple other therapies. And so there's a process in finding them. We know that some patients go through screening and because of serious medical episodes, they are unable to participate in the study. Last quarter, we didn't have any new patients, but we had 3 new patients in January. So this is the variability of recruitment, but it's essential, and that's where the data comes from.

Yes. How many patients have you recruited to date?

Recruited to date, we're at 9. And the other thing is really you need data -- you need the sites to recruit these patients. And so we're really focused on implementation and looking at -- we now have all the U.S. sites engaged. And we're now looking at -- we've just recently engaged another the Italian site, so Professor Zinzani, and we'll look at engaging the next 2 and then France as well. So we need those sites engaged to get the patients and the patients give us the data.

Yes. Sorry. I might just try to put your headphone on fully. So if we could just check the echoing again, David, I think you're giving us some feedback. Has that improved at all? We're not getting any echoing on our testing as well. So hopefully -- thank you, David. All right. So in terms of how many people you need to recruit for IIa, what's that goal?

Well, the IIa is designed to be evaluated by the dose optimization committee. So they will assess 20 patients. So once we receive 20 patients, they'll look at the data and make a call. And that is a very important component to the Phase IIa. The dose optimization committee may decide that they have the right dose and we'll go to the FDA and start talking about what IIb looks like or they may decide that we need a few more patients in a particular dose. So there's -- that's where the interesting begins, and so it's an exciting time.

And so your strategy in terms of getting clinics set up that you can run trials through, I mean, how is -- what -- maybe just talk us through how that works and what you've done to date?

Well, so far, we've had the 3 Australian sites set up, and we've had 6 American sites set up. And so that does take a while because you have to engage the clinicians and then the pharmacy and all the ethics committees. So we have U.S. and Australia set up, and now we're heavily focused on setting up the European sites in Italy and France. It takes some time, but we need that to happen before we can enroll patients.

And then how comfortable are you with all these different clinics being set up that you can achieve that milestone? Or how quickly do you think you can achieve that milestone getting to 20 patients?

Yes. No, we said midyear, and I think that's still on track. The more sites we have, the more likelihood we are getting patients. And so that's why I have the team really focused on implementation because implementation will drive the performance of the study.

Very good. The -- so maybe just back to those next milestones, we might touch on that again. So maybe talk us through where do you think the key milestones are for you the next steps?

Well, the key milestones is 20 patients or when there's data significant enough to allow presentation. And so that's really what we're focused on because the variables after that, there are quite a lot, and we will deal with those when they come. So I would say the 20 patients is a big driver for us.

Okay. And when do you start getting data from a patient for how long after you've recruited them and they start going on the trial, do you start to get meaningful data from [ them ]?

So the patients go through a screening process, which is 2 or 3 weeks or up to a month, and then they can be initiated with the dose, and that's the first from dose 1 to cycle 4. So cycle 1 to cycle 4 is when they are initiated, and we get the first readout in cycle 4, which is about the 2-month mark. And then we -- they continue on the study, and we'll look at their best response after that.

Paul has asked, is Japan on board?

Yes. We don't have a study site in Japan, but it's a good pickup, Paul. Japan do have an Orphan Drug designation philosophy or a protocol. And we are actively looking at Japanese companies at the moment and seeing how we may engage with them.

Okay. I think 4C said you had circa $9 million in the bank. Is that enough to get through Phase IIa?

Well, we actually got another $4.3 million from the R&D tax credit. So we're fairly comfortable that we can -- we have enough cash to get through these milestones.

Very good. Sarah, I can see -- we don't have any questions from Sarah. I can see you had some -- noted you have some questions below they haven't come through, maybe recut and paste or type them in, please for Sarah. All right. Thank you. So you mentioned CTCL 1b trial saw a 100% halt reduction in patient tumor growth. How does this compare to other therapies available in the market?

It compares favorably. It is 70 evaluable patients. So it is a Ib study. So we have to be cautious about that data, but it's really encouraging. Clinicians consider this a win if they can stabilize the disease. This disease is progressing. And from Stage IIb through to Stage IV, the prognosis is very poor. So if you can halt progression, that's really good. And so yes, it's very positive.

So the safety profile and so 0, I think, adverse effects or I think probably you've got a more technical way of defining that than I do. But like maybe talk to that. And then -- but why was that so important in that Ib, those results?

It is important because there were 0 adverse events attributed to PTX-100, serious adverse events. And what does that mean? It means we are providing a new mechanism of action or a new way of addressing CTCL patients and then cancer without adding to the burden. And so I've recently come back from the TCLF (sic) [ TCLLF ] in San Diego, and it continues to be combination therapies where the clinicians are going. And so if we can have a new mode of action, which is not adding to the burden of disease, we're really well positioned for combination therapy. So we get single-agent activity, we then roll into combination therapy is really important.

So how is the technology working that allows you to get these results?

Well, PTX-100 inhibits a particular enzyme called GGTase-1. And so what that enzyme does is it combines 2 proteins, the Ras protein and prenylation protein. And when that happens, it allows the Ras protein to trigger downstream oncogenic pathways. So basically, we inhibit that process, we turn the switch off and we inhibit the switching on of the downstream oncology pathways.

The orphan drug and fast track give you access to the FDA. What sort of engagement do you -- have you had to date? And how does that continue going forward?

Well, at this stage, we're in the sort of the funny point where we need some more data. And so we're waiting on data. We're preparing from our CMC perspective and things like that. And we will engage the FDA once we have more data, that will be particularly around when the dose optimization make a call. And we're preparing for that in terms of what we would like to say, what the future study might look like and with a goal to potential to have that a pivotal study, but we'll have to wait and see on the data.

Okay. So obviously, we're all focusing on CTCL at the moment. Going back to your comments around how your technology works. I mean the application beyond CTCL, there's 22% of cancers who have got the involvement of that Ras pathway. What -- how does this play out going forward?

Well, yes, you hit on a good point there. With 22% of cancers having Ras involvement and the fact that we are able to disrupt the prenylation of Ras proteins, it's important for us to understand more of that detail of the mechanism and therefore, look at other tumor types that have a dominant Ras component to their mechanism and see whether we can make an impact there. So I have the team working really hard on further understanding our mechanism for us to be more directed in our approach when we look at other tumor types.

Very good. All right. Just -- you mentioned the -- well, maybe I mentioned the IIb has the potential to be a registration study. I mean if you enter -- obviously, you've got to -- you make some decisions about whether that's the pathway that you go down. But if you do go down that pathway, how do things change as you move into a registration study from where they sit today in IIa?

Well, it changes because the patients will have access to a potential therapeutic sooner because we're in a pivotal and hopefully, we're going through to registration. It changes in terms of our commercial orientation. Means we can have very serious discussions with partners. And we know that the partnering environment is now companies are willing to partner later as opposed to very early, and that's what we'll be looking at. So 2 really important things, one for patients, one for our commercial benefit.

How big is the CTCL market?

It's another good question. The global data information we have is the CTCL market in 8 major markets around USD 600 million. But we know that this data is relatively old now, and we're looking for new data sources to see that. I would expect it to be higher, but we will look at that but it's still substantial.

U.S. dollars.

U.S. dollars. Yes.

So you've got a technology that can inhibit the cancer growth. You've applied it to CTCL. It's applicable to 22% of all cancers, which opens up obviously a much, much, much larger market. I mean what's your strategy going forward in taking this potentially to solve and look after more people with a wider range of different cancers?

Well, our strategy is to actually get the study done, but also in the process, understand our mechanism a little more and then be more directed about where we can go with other tumor types. And this, in turn, will really make it more attractive, and we can really progress in different areas. So on top of that, we'll also be looking at how we can dose PTX-100 in terms of formats and things like that. So lots to do. It's actually really exciting that there's a lot to do. But essentially, we need the CTCL study to get to at least the dose optimization committee stage, and we also need to really fine-tune our mechanism.

Now Sarah, we've never got your questions. So apologies, we haven't got to those. I've got one final question. If anyone has any other questions. Paul missed the question about Japan. Maybe we'll send you the replay, and I'll get someone to notate the minute, but it was discussed, Paul, for you. All right. Here we go, Sarah. So I think we've got some of these questions. All right. So we talked about how many patients have been dosed already. Out of those patients -- out of all the patients that applied to the study, how many are accepted?

That's a good question. Well, we go through a screening process, and that screening process is quite arduous. We do know in the last quarter, we had 2 patients that actually got approved through the screening process, but because of medical episodes, they are unable to participate in the study. So I guess it's a handy reminder that we're dealing with patients who have a refractory and relapsed disease so they had multiple other therapies. They have a serious condition and it's quite challenging. So we look -- I don't know the actual numbers in terms of the failure rates, but it is quite solid. I can further investigate that if needed.

Okay. Sarah, I'm going to shorten your question a little bit and roll it into one. OmniCAR and CellPryme, could you make a comment on the strategy for both of these going forward?

Yes. I mean we're looking at CellPryme in terms of manufacturing. We continue to do that. The cell therapies market is a little bit flat at the moment. For OmniCAR, we would like to progress that, but we will be progressing that through a partnering approach. And so we've initiated looking out for partners for OmniCAR. So those are the 2 areas that we're looking at for the cell therapy platforms that we have.

Very good. Okay. Paul has asked, would there not be an easier cancer to recruit for other than CTCL?

With our Ib, we saw that PTCL and therefore, CTCL was a very clear pathway. And because we have Fast Track designation and Orphan Drug designation, it is actually a really good approach to take. So for where we are with PTX-100 at the moment, it's an ideal approach. And then we will look at expanding our IP, et cetera, for other tumor types. So it's a complex situation, but CTCL is a very good first step.

And is that the motivation -- is that why your strategy has been to go out into Australia, U.S. and Europe? Would you -- otherwise, maybe with some of the other cancer types not need to have a broader strategy in getting into different clinics?

Yes. That is one of the situations because the Australian community doesn't have enough CTCL patients to meet our clinical needs. But we need experience in the U.S., we need experience in Europe. And Paul, you also mentioned Japan. I mean, ideally, it would be nice to have a site in Japan, and that's something that would be good looking at, although Japan regulatory environments are changing a little bit now. So we should be fine.

Very good. All right. Well, you've been with PTX for a year now. Is it a year?

Yes.

There you go. So how do you feel about your decision to join and the opportunity that Prescient presents?

Yes, it was an opportune time to join. It's very rare that you can have a first-in-class therapy that's going into Phase II, which also has a whole lot of other potential. We've got a great team and the team are working very hard. We're focused on the implementation and the clinicians can see the benefits here. So I'm excited about it. I continue to be excited about it, and I look forward to moving forward.

Beautiful. One last question for you from Kerry. So current patients are required to have failed prior treatments before moving on to the PTX-100 trial. What stage will PTX-100 be available as a frontline treatment?

That will be a clinician choice. It's very rare that new therapies end up -- start frontline. But we know that with good clinical data, with adverse events 0 has been attributed to PTX-100. We know that it's going to be really suitable for combination. So first line, it may actually be used in combination with other first-line therapies. So it's a complex work there, but I would say once in the market, the clinicians do what they do.

So I mean -- but to that point, is that post registration study?

Yes, definitely post registration study. Once it's in the market, the clinicians have a little more flexibility. And if they like what they see and they see that it's -- we're not adding to the burden of the patient and it's a unique mode of action, they're likely to start adding it in the combination approaches. I suspect it will be -- that will be more first-line whereas monotherapy or single-agent approach will be a bit further down the path.

Very good. All right, Sarah. Good question, but I'm not going to ask that one. The -- all right. Look, I think that's everything that's come through. Thank you to -- we've got such great engagement from Prescient shareholders coming on through all the sessions and getting updated and asking good questions and also supporting the company. So thank you for everyone taking the time for joining us today. We look forward to some more updates from the company in the near term. We'll send a copy of the session around, so keep an eye out for that. Anyone that's not getting the updates from Prescient PTX into the chat box, we'll make sure we add you on to the list or at least check into that. And James, I'll leave the last word with you.

Well, I think you said it, there's some really good questions. So I think we have some really engaged investors and supporters. And I thank you for that, and I hope you can continue the journey with us. So thank you very much.

Thanks, guys. Have a wonderful day. Cheers.