Prescient Therapeutics Limited (PTX) Earnings Call Transcript & Summary

September 18, 2026

ASX AU Health Care Biotechnology special 51 min

Earnings Call Speaker Segments

Patrick Nelson

attendee
#1

All right. Good morning, everyone, and welcome to Prescient's investor briefing, while everyone settles in, I'll go through the disclaimer and housekeeping. Any of the presentations that Reach Markets put on are suitable for self-directed investors and those that have the experience and capability to make their own informed decisions. Any information contained in today's presentation is general in nature. It doesn't take into consideration your personal circumstances, and you need to decide for yourself whether it's appropriate for you. Past performance is not accurate or reliable indicator of future performance, and we're providing you detailed information and the ability to ask the company questions for your own educational purposes. All right. My name is Patrick Nelson, MD at Reach. I'll host the session today, but we're joined by James McDonald, the CEO of Prescient Therapeutics that trades under the code PTX on the ASX. In today's presentation, James will run through his investor deck but we'll start off the session with a couple of questions around commercial opportunities and the upcoming dose committee optimization meeting, and then James will go through the deck, and then we'll go into more detailed Q&A. [Operator Instructions] Now where -- this session is put on for investors in general. We know there's a couple of shareholders that have joined the session today. But just to get everyone on the same age, I'll do a quick intro. Prescient a company that Reach have been involved in now for 5 years. And look, it's become 1 of Australia's most advanced cancer therapies on the ASX. It targets CTCL, which is cutaneous T-cell lymphoma, which at its advanced stages, as seen by many clinicians as a death sentence. It's a truly terrible disease. Patients become -- constantly suffer from secondary infections, struggled to sleep, and so forth. And yes, again, the options for treatment are very slim and the results have been proven to be very good. So the previous, the Phase Ib trial of Prescient for PTX-100 had remarkable results with 100% of patients recording either a halt or a reduction in their cancer. And even more remarkable is there was 0 serious adverse effects. And for those patients, this is really meaningful, even if it's not a reduction of tumor, the halt of the tumor can assist greatly including the itchiness stopping and the ability to get to your sleep back and remove -- move back to a more normalized life. But since CTCL is seen as a cancer of unmet need, it is getting -- received fast track designation, orphan drug designation from the FDA, particularly important, moving this forward. PTX is a maturing biotech. It's a major inflection point. It's got multiple near-term catalysts at moving them towards commercialization. But into the interest of pharma, big pharma, mid-tier generics, and other groups, getting a dose set. I mean, the next major milestone is this dose committee meeting, which is now scheduled for December. Getting this dose set is a significant derisking moment and it's exactly where deals get done and significant ones. There is also the potential for the Phase IIb, so we're IIa at the moment, for IIb to be turned into a registration study, which is this major accelerated pathway to market, which will be another incredible -- incredibly valuable development and highly attractive for big pharma. There is more deals getting done in this sector than ever, big pharma are competing for assets that are derisked, have high margins, and have market exclusivity. And PTX is positioned as a potential to offer all 3 of these. We saw recently that in CTCL, a deal done with Sobi acquiring lacutamab, which saw them pay USD 75 million upfront and a deal, which had other milestones, which would see up to USD 580 million in total, that would be a AUD 815 million deal, and it had -- additionally, it had a double-digit royalty, which is incredibly valuable. That drug addresses just 5% of the CTCL market. PTX is applicable to 64%. So it's roughly 12% larger addressable market. The Phase II trials for PTX-100 are essentially confirmatory, replicating Ib in a larger cohort using the same smaller doses, not chasing the higher 200-milligram dose that could raise safety concerns. 28% of the required patients were already recruited and everything here is happening. I mean this is part -- this is coming to that end of a 12-year journey. So PTX currently trades around circa that $70 million market cap, I mean you just think back to that lacutamab deal, can't get my word straight there, which is AUD 815 million plus double-digit royalties, and that's just a fraction of addressable market. That deal activity out there at the moment in biotech land is really heating up. So I think we're in the right place at the right time. But our journey with PTX has been over 5 years, and it's always been a pleasure to be involved. The focus on PTX-100 and what it's actually achieving for patients is just something that's good to be involved in. Being part of it can be generally rewarding, seeing the results for people. What that actually means is something we've really enjoyed being part of, but the numbers here are exciting and the stage of this business is with the dose committee meeting in December is a really interesting point, and this is the point where deals get done. So James, thanks for joining us today. You've been here I guess from -- for the last 18 months, and I mean, driving the company forward as it moves into commercialization. I mean, to that end, I mean, what sort of engagement are you now seeing from? And who are you seeing that engagement come from?

James McDonnell

executive
#2

Yes. No, we're seeing quite a bit of engagement over the -- for the -- particularly this year, we're well over 20 partners and discussions are ongoing. They are interested in the fact that PTX-100 is moving towards this dose optimization committee meeting and then, eventually, to a dose, and that's a derisked profile. And so pharma companies are really looking for deals that have some derisked element to it and a reasonable market size. And the Sobi-Innate deal with lacutamab really demonstrates that there is substance in the space. And so validates our commercial presence and also with the Phase Ib results, replicating those will be pretty impressive.

Patrick Nelson

attendee
#3

Yes. And then -- and so who -- I mean, where are the discussions coming from? Is it mid-tier, generics, big pharma? What sort of...

James McDonnell

executive
#4

Yes. I mean it's come from a selection of companies actually. So typically, the big, big pharma want a broader appetite, but we talk to big-ish and mid-tier pharma companies. They are experts in the series. Some are already in the space, and so they know the market. And so these are significantly large companies compared to Australian companies. And so we're encouraged by that engagement. They have a global reach, which is something that's really important to -- for me, that's the idea is to have 1 partner doing a lot, and all these companies that ability. And that's the key element that we also need to take care of. There are companies that are -- have been set up globally, which are owned by parent companies, which are massive who are in the generics business, and they realize that we'll -- we can't go further in generics. We need branded environment. So they've set up the separate large pharma companies to take on assets similar to PTX-100 in terms of rare disease, absolute market exclusivity. Those are the sort of conversations we're having. They're gearing up now because they all recognize that the dose optimization committee meeting, which is happening in December, is the first real look at the progression of the Phase IIa study. And so that's important to them. It's important to us in those discussions, and that really will motivate further discussion. The Phase Ib results are the reason why we're here. So Phase Ib results are sort of what we're at least aiming for, what that looks like when we get to something in the dose optimization...

Patrick Nelson

attendee
#5

What does that mean? Like if you get -- if you're able to replicate Phase Ib results?

James McDonnell

executive
#6

Well, that -- I would be impressed with that. I mean, that's from a single center going into a multi-center study. That would be ideal. If we look at the Innate deal, they have a 42.9% response rate in Sezary syndrome, and that allowed them to do that deal. So we're talking that objective response rate on our Ibs. We get there we're talking deals. And that's -- in this space, we target a 30% objective response rate as being quite significant...

Patrick Nelson

attendee
#7

With the Innate deal, what's transpired there?

James McDonnell

executive
#8

The Innate deal?

Patrick Nelson

attendee
#9

Yes.

James McDonnell

executive
#10

Yes. So the Innate deal was actually a deal which, for me, as a commercial person, if I was their CEO, I'll be pretty happy with that deal. They have a deal which allows them to fund their pivotal program. So they've just completed their Phase II. They have an approved pivotal program which has got an accelerated path to it for the Sezary syndrome. So only 5% of mycosis fungoides and then a more confirmatory part for the rest CTCL. And with that, they've been able to negotiate this year, which is fully funding their Phase III or the pivotal program with that USD 75 million upfront. It's also got USD 40 million in milestones as they progress. and then a $465 million number they get at approval plus double-digit royalties. And so that, to me, is a good deal. And, yes. Every deal is...

Patrick Nelson

attendee
#11

So all U.S. dollar numbers you're quoting there. And that is for 5% of CTCL market and PTX-100 is addressing 64%. Is that -- we can expect a 12x the size of that, James?

James McDonnell

executive
#12

I think that would be wishful thinking, but the -- but yes, you're right. That's -- when you go to these discussions, you would like to have options. And so that's a benchmark deal in the CTCL space, but we can say, "Well, that's for Sezary. Mycosis fungoides is a bigger patient pool and so that's a subtype that we're -- we seem to be clear, and we've got a fast-track designation in that. So, yes. I mean, I couldn't say what the impact on the total number would be. But it's certainly when you're preparing your options, that's definitely an option we'll be discussing.

Patrick Nelson

attendee
#13

And look, I mean, 1 of the things we think about, there are good times to be in biotech. They are really, really good times to be in biotech, and we're seeing deal activity really heat up at the moment. What is it about where you're approaching that, the specific things that pharma are looking for, if you name it like the top 3 things that like big pharma would be looking for right now that PTX will be able to offer?

James McDonnell

executive
#14

Well, yes, I mean, firstly, we've got a first-in-class asset. So we're leading the way in this asset pool we're heading towards a de-risked environment. So it's -- as we progress through our Phase II program, we also -- we've got fast-track designation. So the program itself has got all the ticks -- the bells and whistles in terms of what we need. And then the market size is significant. We've seen our $1.2 billion estimate by 2024. But it's also validated by the recent Innate deal. So yes, we have some really nice pieces of the pie, if you like, that big pharma are looking for because they are looking, because their asset pools are starting to drop in terms of patent cliffs. And so they're looking at supplementing their pipelines. They want to supplement with the -- at least some derisked assets, and that's pretty much why you see these deals done between Phase II and pivotal and those sorts of areas. And so that -- yes, so we're heading into that sweet spot, if you like.

Patrick Nelson

attendee
#15

Very good. All right. Look, I think I'll pause the questions there. We'll come back for Q&A, but I'll let you run through the slide presentation. Cheers.

James McDonnell

executive
#16

So thanks, Patrick. And here's our disclaimer and safe harbor statement for forward-looking statements, et cetera. So -- but here we are, a company snapshot in terms of -- at the end of the financial year, we had $9 million in the bank and a reasonable market cap. And since this time, we've noted a capital raise. The highlights from this presentation are really, really quite impressive in terms of we have a lead drug, which is PTX-100, it's from Yale. So very good pedigree. It's pioneering a platform where we inhibit a process which is involved in up to 22% of cancers. So 1 of 5 cancers could be in the target profile there and first-in-class, as I mentioned just a moment ago. First-in-class cancer candidates are really important, but we lead the way as we're the most clinically advanced in the space. And so that's -- we're setting the ground. We're working in a rare blood cancer interestingly. It's called cutaneous T-cell lymphoma. So it's an unusual lymphoma where it exhibits a lot in the skin, because that's where the lymphoma is really attacking. And there's some near-term milestones that we've talked about, the dose optimization committee. This is a committee that is designed to look at the Phase IIa program at the halfway mark, firstly, so 10 evaluable patients in each arm. And we know that this is going to take place in December. So this is a very near-term milestone. And we're creating value. We've just heard Patrick and I discussed the deals that we made -- have been made, of the transaction recently with Innate Pharma, but there's real value here, value for the patient and value for the investors. So it's a really impressive highlights at the moment. So just to take you through where we set with PTX-100, this is a cancer foundation slide. which talks about normal growth and abnormal cell growth. I've added a little component there about the Ras family mediating the cancer cell division and therefore, moving to a malignant tumor. And that's -- the reason I've done that is because PTX-100 has an activity in this space. And so if you look at on the left, you can see that Ras family proteins use an enzyme called GGT-1 to actually prenylated. So they add a small group of molecules that allows them to hold on to the cell membrane, switch on Ras -- Ras and Rho and therefore, triggered pro-tumor effects. Typically, this process is -- when it's working normally, it turns on and off. And again, in the cancer environment, the switch doesn't turn off. It's just stuck on. And so our role is to inhibit -- is to disrupt that on switch. So when you look at PTX-100,we actually work on the GGTase enzyme, where PTX-100 fits very nicely in the prenylation pocket, preventing the protein, Ras family proteins, attaching in that pocket and the geranylgeranyl groups attaching in there as well. So they're unable to prenylate and therefore, we disrupt the switching of those Ras family proteins. So that's the theory. And we've since worked with CSIRO, doing some modeling. So we've done some AI modeling and physics. And we know where PTX-100 fits. And so you can see that there on the left. And in the middle, we have done some in vitro work. And you can see that it's a sensor. So if you move the graph to the right, so PTX-100 is in the green, it has definitely moved to the right. This means that there's strong binding to the area that we're looking at. So that's a confirmation. And then on the right side, the -- looking at the percentage of prenylated Ras protein. So we inhibit the ability for proteins to prenylate. And in this circumstance, these are 4 different tumor cell lines, and we can inhibit between 40% and 60% of that prenylation. So we're 100% would be fully prenylated. We can see here that we're not getting anywhere near that amount with the action of PTX-100. So it seems the dairy makes the practice. So we mentioned first-in-class. First-in-class is very helpful because it allows -- your first time you can go to the FDA or other regulatory bodies and gain designations, and that gives you a head start. We are further developed than any other GGTase's inhibitor and the only 1 in the clinic at this stage. And we're not really competing with any other. So therefore, our clinical programs can progress without that sort of competition, adding to a KOL engagement. And then we -- as we gain more data, we'll have first advantage and [ navigate ]. So first-in-class advantage is actually a real advantage. We have talked earlier about CTCL. And why are we talking about CTCL? With PTX-100, we inhibit the prenylation of Ras proteins and so disrupting the Ras pathway. So we're looking for something in that space when we start looking for an indication. You can see some other characteristics here that we looked at. And with CTCL, when we look at data, we see that, yes, there's some Ras family involvement. It's an orphan disease. It's really clearly unmet need because it's quite dramatic. And the market size is substantial. And so we're in this space because at the moment in our Phase Ib program, we saw that 43% objective response rate versus other products is impressive and there were no drug-related adverse events, serious adverse events, which is important in the space also. We've gained fast-track designation and mycosis fungoides from the FDA as well as orphan and drug status in both U.S. and EU. So we're really set up. The strategy so far with -- as we're progressing from our Ib is absolutely on target. So CTCL, Patrick did mention that it is a cumbersome disease. And it's actually a rare blood type -- blood cell which are lymphoma. So the T-cell actually migrates to the skin where it replicates and actually disrupts the skin and eventually impacting the [ node, the stream ] and blood. So as you progress through this disease area, it becomes quite challenging. It's really significant because you're talking about the skin, you're talking about quite a significant service area. You're itching, your secondary infections, your appearance is all socially isolating and really disrupting, and fatigue is that real challenge for this disease. We know that this -- it's rare, but there's 3,000 new cases around them in the U.S., and that's increasing. And then CTCL, there's 2 main subtypes, we've talked about this a little bit earlier. Mycosis fungoides is between 50% and 70%, depending on which data points you look at. And Sezary syndrome is about 5% of CTCL. So that's important as we progress. So just on that, mycosis fungoides is, really, the CTCL space where you have the main skin involvement and there's a less dramatic area and then as you advance, it become quite a challenging disease. And Sezary syndrome is much more aggressive to have this. There's no early phase. It's all quite an aggressive phase. Typically is the skin involved, but there's also blood involvement as well. And it's slightly more leukemic in nature. And so when I talk about outcomes in advanced stages, you can see the early-stage mycosis fungoides on the top there that if you're graded as 1A to 2A, your overall survival for 5 years is -- well, it's diminishing, but it's not the main issue. The main issue here is quality of life. It's the itching. It's the secondary infections, that really social isolation and fatigue, et cetera. But as you progress and if you have progress from IIb down to IVb, you can see that it's quite a significant change in your 5 year overall survival percentage and right down at the bottom there, only 18% 5-year overall survival. So it is clearly a challenging disease. Not only are you challenged with quality of life, you're also challenged on your prognosis. And so we know this -- that this is an issue. We know that current drugs have the modest efficacy and then some challenging safety profiles. Lots of patients had really run out of options. So there's we call them refractory relaxed patients have a clear unmet need, and hence, the fast-track designation that we gained from the FDA, recognizing that there's a clear unmet need in mycosis fungoides patients and FDA would like to have active therapies to these patients as soon as possible. So if we look at the drugs that we see in the CTCL space, firstly, you have a lot of -- in the mild areas, you have a lot of topical things. You might get a little bit of chemotherapy. But then you get these second-line, more targeted therapies. And you can see on the left there, you sort of biologics that carry payloads. So LymfCare is carrying an IL-2 payload. And that's a way of targeting the CD25 and CD30. So you can see there, those are antigens on the cell membrane. And on the right side there, you can see POTELIGEO and CAMPATH. These are also targeting antigens. There are antibodies to antigens on the cell membrane. And the lacutamab drug there, which we talk about is actually the IPH4502. And this is the product that has just signed that transaction with Sobi Pharma. So there's a number there. But as I mentioned, there's a clear unmet need in these patients. So they're really going through these therapies a lot. Down the bottom, we see HDAC inhibitors. And in the middle, you see where the Ras family proteins pit. So they get their geranylgeranyl group with the GGTase, they get together, they're attached to the cell membrane, and they're switching and they stay switched on when they should be switched off. So when you put these -- you can group these drugs into different areas, but you can see there's a very clear distinction between PTX-100 and where it works. And so with solid results, that we've seen in the 1b with PTX-100 with those sorts of solid results. And with this unique mode of action, it can create a situation where you have a backbone product that works in these treatment combinations. So we talk about the data. We've talked about the Phase Ib. And if you go to the right there, on the PTX-100 CTCL-only patients, these were the vatable patients from our Ib program, which was in T-cell lymphoma, the 70 [ valid ] patients. And you can see the response rates there and were 43, 100% had a benefit. So stable disease PR or CR, in a quality of life circumstance, that's actually encouraging. Duration and no serious adverse events attributed to PTX-100. So if we go right over to the left now, we see a benchmark. So you have to know what type, what you're going for. We've listed here as benchmark. Technically, it's called a target product profile, and we're talking about 30% objective response rate various elements there, but also less than 30% serious adverse events. So that's quite a high number. So to be less than that, there must be some issues with products in this space. And you can see the 2 middle products there, [indiscernible] and POTELIGEO have objective response rates at around the 30% mark, 1 higher, 1 lower. But you can see that serious adverse events rates are way high. And so this is quite challenging for patients who are already challenged with their disease. Now we talk about lacutamab, having done the deal with Sobi Pharma, where they have fully funded their pivotal program with USD 75 million with $40 million in terms of milestones. And then as they get through, they'll get $465 million, it's pay plus double-digit royalties. And so they did this with this data here. And they did it with the data essentially for Sezary syndrome at 42.9%, and encouraging adverse event profile attributed to the drug as well. So that's what they were able to do the deal with, as we mentioned before, mycosis fungoides is what we had fast-track designation for and the much larger cohort of patients. So we talk about these favorable safety profiles. The reason we do that for PTX-100 is when you compare across -- in our early research, we've seen that our safety profile is very favorable and not challenging as you can see other products, which have obviously had more research done, but you can see the challenging profile. So we're looking at a new mode of action, really without impacting the patient's fortuity, and therefore indicating that it will be a great combination process and understanding how that will work, will be, whether it's with HDAC inhibitors or whether it's using the antibodies in the [indiscernible] approach or different things. There's lots to do in that space. So we're running a Phase II program and our Phase II program was actually broken into 2 at this stage. And so we have a Phase IIa program, which is looking at refractory labs, CTCL patients of mycosis fungoides or Sezary syndrome who've had at least 2 prior lines of systemic therapy failure. And we're enrolling up to 40 evaluable patients. When we get to the halfway mark, which is 10 evaluable patients in each arm, the dose optimization committee charter indicates that they'll start looking at data. Now we said that will happen in December for the first time. So this is a really near-term milestone looking at the halfway mark of this Phase II program, the dose optimization committee will be looking for a dose that we're comparing the 2 arms for the safety, efficacy, and ultimately, trying to find a dose. Will they find it at the half way mark? Maybe not we'll probably suggest that we continue further to perhaps 30 evaluable patients, and then they can get a look-see. So this is -- it's a little bit fluid at that point, but we do know absolutely that December will be the first dose optimization committee, and that's the best reasonable look at data from the Phase II program. And you can see the endpoints here or objective response rate, lots of secondary endpoints. The first 1 there is the mSWAT is a particular assessment of the skin specifically for CTCL, which will be very clinically valid and the clinicians tend to use that in their day-to-day assessments. We're looking at safety and also quality of life. We've talked about quality of life here. We are measuring all the quality of life aspects that we can in the study because that will also be substantial in terms of going forward. Our program looks like this at this stage. So we've completed the Ib. We're ongoing in the IIa. And the typical pathway is that as you see there, the expected clinical. Now we do have fast-track designation in CTCL for mycosis fungoides, orphan designation. With that fast-track destination, where we have an ability to speak to the FDA on a more frequent basis. They also acknowledge that there's a clear unmet need. And so our goal in those conversations will be to gain as much data from the Phase IIa program, have a dose data, safety admonishments and then look at what we can do in terms of accelerating more to a pivotal program and an accelerating approval and particularly in mycosis fungoides. So we've seen this happen with lacutamab where the Phase II program, went to a pivotal accelerated [ transitionary ] and confirmatory for CTCL generally. So we know that the FDA are creative in what they do here. And so the ability to speak to them more often as -- will be important for us as we gain data from the study. So we -- the market itself has been validated by a near-term transaction, but this is the market that's estimated by a third-party researcher, DelveInsight. You can see it's about 1.2 billion in 2024 is the estimated number for the U.S. alone. And so this is a big market. and there's 3,000 new patients per annum in the U.S. So there's something there. And when we look at our products already on the market, so mogamulizumab is POTELIGEO, which had the 28% particular response rate. I put this graph here because the yellow line looks at the sales from 2020. It was approved in late 2018. So just after the launch, you can see it's already gained momentum here. And it's used in early and late-stage mycosis fungoides, which is encouraging because even though you may have early stage, if you run out of options, clinicians are looking for something to use. And so this is what we see. And the other line at the top there is the current total market, but you can see it's growing at the same rate as mogamulizumab. And so that shows that there's a clear unmet need in the space. And if you've got something unique mode of action approved, clinicians will use it. And the other good thing about this for PTX-100 is, yes, we have a unique mode of action, but it also gives you a comparison from a market access perspective, and that's important as well. So we're moving along. We're very CTCL-centric at the moment. We're demonstrating efficacy and safety through our Phase II program following a very successful Phase Ib program. We -- our goal is to complete registration and look at these partnering and commercial pathways. The expansion component is we inhibit -- we disrupt the Ras pathway in terms of preventing relation of Ras family proteins. And so there's other indications that makes sense. And in fact, that graph which has 4 bars on it were actually 4 different tumor types or cell lines. And so we know that there's some benefit out there. And so we will do some further work on that and create more opportunity, particularly -- most likely an orphaned area first. And then as we progress with more IP, it will be a much broader tumor type. So we're looking at commercial pathways and leveraging all that. So it's an exciting time. So we have some near-term milestones. And when we look at this milestone, really, we've talked about the main one, which is the dose optimization committee review with 20 evaluable patients. This is key. And then as we progress, we'll have the franchises up and running. So even increasing the recruitment more rapidly and other dose optimization. But the real key milestone is Q4 this year, which is -- we know it's December now. And then the whole aim is to get to the Type B meeting with the FDA, where we can really look at what that next program is going to be. So that's where we are. This what our focus is, and that's what we have to do. So we've got a team to do it, and we've got a very supportive board. And so this has been pretty consistent, and we've been working hard as we progressed. And so we have a really solid value proposition in terms of our unique first-in-class asset, PTX-100, appealing to 22% of all cancers because we disrupt Ras family proteins, which affect that. So we've got some great data. We're progressing with FDA designations, and we know that we're building a nice foundation for future partnering discussions. So it's all on track. Thank you.

Patrick Nelson

attendee
#17

Thanks, James. All right. We've had a few questions come through. [Operator Instructions] I'll start off with [ Paul ]. PTX-100 could potentially sold 22% of all cancers. Where do we stand right now in terms of timing if all goes well in December meeting?

James McDonnell

executive
#18

Yes. So at the moment, we are looking at CTCL in terms of -- it's almost a proof of concept that disrupting the Ras family protein using an inhibiting prenylation goes well. Our dose optimization committee meeting in December will give us the first look at how that's progressing and depending on what that is, we'll go to the next stages of the program. I would like to also then start looking at more pre-clinical work and other work in other tumor types. So as I mentioned, with the orphan and things like that, we'll progressively work through tumor types that have Ras involved. There are 170 Ras proteins, and so it's not a simple picture, but it's a picture that we're certainly very focused on.

Patrick Nelson

attendee
#19

Okay. A couple of questions regarding big pharma take-out comps, et cetera. And I know James has got referenced the Sobi deal before. Look, it's 1 that we did a bit of work on at the start of a couple of months ago, and I think it -- what comps -- I mean, there are different elements to each one. So the Sobi example, which is what it is, but that's 5% of CTCL compared to PTX-100, which is 64%. Mirati was another 1 that we looked at. I mean, that was -- they sold their Ras inhibitor, which was adagrasib, I think, was the pronunciation of that. James can probably correct me on that later. But that's focused on a different cancer but otherwise, quite similar. And they went through a similar process to Prescient as -- and also receive drug orphan designation in '21 and '22, the FDA gave them accelerated approval. And they took the drug to market in '23 and got bought out by Bristol-Myers Squibb in October that year, 10 months after getting that accelerated approval for [ $7 billion ]. Now we're not saying that's kind of the same thing is going to transpire for PTX. But it's an example of how fast things move. And also an example of the kind of numbers that things trade for. From Mirati's case, orphan drug designation during Phase 1 to buy out around 2 years. So we're seeing this sort of deal activity. These are the deals that are getting done. I guess, like what other things, James, that you think are important that you need to achieve to put yourself in the best position to -- for a transaction to occur.

James McDonnell

executive
#20

Well, I mean, you mentioned there the timing. So our goal is to make sure that we're running parallel paths. We need to implement the study. We can do everything we can to make sure that we -- that study is progressing as well as it can and quickly as we can. We can't control data, but we can make sure we can control what we can control. The parallel path is actually engagement with partners, and we've been really ramping that up this year. And as I mentioned earlier, we've had over 20 companies that we're now engaging with at different levels and they are all now very focused on what's going to happen in December and what's going to happen after that. So our goal is to make sure, again, like the study that we are engaging and implementing that process.

Patrick Nelson

attendee
#21

So orphan drug designation, I mean, how -- maybe talk through the elements of that, that are important for the potential acquirers.

James McDonnell

executive
#22

Yes. So orphan drugs actually are really helpful. It's 7 years in the U.S. and there's 10 years in Europe. Other jurisdictions such as Japan, where we'll look at it as we 7 as well...

Patrick Nelson

attendee
#23

When you say 7 and 10, what does that mean?

James McDonnell

executive
#24

So that means market exclusivity. So therefore, when you have an approval, you have that exclusivity in that market for that period of time. Now the good thing about that is it starts when you get approval. And so you're not using it while you're building up your data and getting through the approval process. Your only start using it there. So you've got clear 7 years commercial benefit in the U.S., 10 in Europe. So it's beholden to the commercialization team partnership or whatever, to actually be ready to go on day 1. And so it takes a team to be there ready to go because every time -- every month that you are delaying the process, you're using up your [ year ]. So -- that's what's important. And that's why pharma companies who are established are looking for assets like this because they already have the ability to go from day 1. So that's important for us. It's important for them. And so that's -- yes, so it's key. We've got companies that have -- are looking for derisk assets that have had their patent clerks drop-off, so they've run out of patients and other things, and they don't have that protection that we -- that orphan drugs designation gives. And so those are the reasons why there's those dynamics in the space.

Patrick Nelson

attendee
#25

Yes. The royalties, I know that there are businesses that just trade in royalties and the -- what I mean, for Sobi, a double-digit royalty or a deal like that, what does that -- what -- can you put that in some context around what that might mean for Prescient, if it was to achieve something like that?

James McDonnell

executive
#26

Yes. I mean, we should not be talking about another company in this, but I saw a Neuron now looking at dividends from all their royalties that they're gaining from the sales of their asset, which they have royalty numbers from their partner. So it can be a significant non-dilutive approach where your funding further development of the company and really adding significant value. And to the point now where Neuron is actually having to issue dividend. So the royalties are a critical part. Yes, the upfront number is good, the USD 580 million from Sobi to Innate will look good on their balance sheet. But the ongoing royalties will also be quite a significant player in that space. And I mean they have other assets that they'll be progressing, so they'll find that helpful. We have a slightly different environment. So every deal are a bit different, but the royalty numbers are significant.

Patrick Nelson

attendee
#27

Yes. And so I mean, thinking beyond CTCL for a moment, and there was back to sort of Paul's question, the -- I mean, obviously, CTCL is just an absolute game changer if you're able to get a deal done here, and we're at that point where things are converging and the markets behaving itself at exactly the right time, which is great as well. But if you were to look at 22% of all cancers have got Ras involvement and clearly, this is doing something that gives us a great safety profile, but also good efficacy. What is the approach to thinking about a program to explore what the next indications could be? Is that what you were doing with the CSIRO? Maybe give us some thoughts.

James McDonnell

executive
#28

Yes. I mean we will work at the next stage. I mean, we -- there are -- looking at cancers that are really impacted with a high percentage of Ras involvement than others. And even the type of Ras involvement, it's KRAS, NRAS, HRAS but we'll do some pre-clinical work and then move into a clinical environment as quickly as we can. Most likely in another orphan disease area because PTX-100, we need that to make further work. But as we gain momentum with our CTCL program and another program, we'll be looking at what we can do to really strengthen our IP to go in a bigger cancer types such as guts, lung, and those other larger tumor types that have Ras involvement that would appeal if you've got a more solid IP behind you. So it's a 2-stage approach for the next step, if you like. But we have to get through the CTCL step, and that's we are progressing quite well through that phase at the moment. And we've got a milestone point coming.

Patrick Nelson

attendee
#29

Paul's asked about Keytruda, which was in the news recently. How does PTX compare?

James McDonnell

executive
#30

Well, Keytruda is collecting indications as it goes along. It's 1 of those benchmark drugs. But if you look at their strategy, they achieved clear response in their first cancer type. And now you can see that they're aggressively adding other indications as they go along. And so that's because they inhibit an area which is attracted to all different types of tumors. And so they have that ability to stretch another tumor type. In inhibiting Ras is also impacting different tumor types. And so if you look at progression in terms of what other tumor indications you'd go for, similar principle, you validate your inhibition and then you look at other tumor types. So, yes. Keytruda is probably at a really big level, but all the similar principles apply.

Patrick Nelson

attendee
#31

Yes. There was a -- [ John's ] asked a question, which is sort of a -- we asked at the start of the session, but I'll ask it maybe in a slightly different way, which is the -- coming back to the size of PTX-100, part of the market for CTCL. So how do you -- why a big pharma paying such big numbers for something as small as Sobi was in was the question. And are we going to be a much big multiple of that.

James McDonnell

executive
#32

Yes. I mean if it was as simple as maths like that, that would be great because Sezary syndrome is 5% and mycosis fungoides is between 50% or 70%, or 60%. Yes, we impact a larger-- the largest subtype of CTCL, which is significantly more than Sezary syndrome. And so that's something we will be highlighting in our discussions commercially-wise, how that rolls out into what the deal looks like or it will be dependent on the deal. But the -- if you're appealing to a larger cohort of patients, you've got a stronger argument to make. And it's all about providing options at the table for a negotiation, and that's certainly 1 that we won't miss out on. So that was the discussion about CTCL, the mycosis fungoides, is both early stage and advanced stage. And we've seen from POTELIGEO, or mogamulizumab. It's a unique mode of action, which is used in both of those cohorts, even though 1 is -- the prognosis is not so bad and then the progress is get much worse. It's used right across the board. That's because patients are refractory laps to available therapies, and there are a lot of them out there.

Patrick Nelson

attendee
#33

All right. I don't have a name on this one, [ RB ]. Could you go through the actual dosing levels? Is this a dose escalation? How are the doses being evaluated compared to earlier study? So maybe I think we're referring to IIa and I think we're saying, what's the difference between Ib and IIa? Are we increasing the doses...

James McDonnell

executive
#34

Yes. So the reason why we have the IIa is because the FDA has a program called Project Optimus. And you'll notice in our Phase IIa program, there's 2 dosing arms, 500 milligrams per meter squared and 1,000 milligrams per meter squared. Now in our Phase Ib program, we actually also have a 2,000 milligrams per meter squared arm, so a higher dose. The FDA on reviewing PK or pharmacokinetic data, et cetera, decided that it's better to have an optimal dose rather than the highest effective dose. And so this is why this dose optimization component of the Phase II program is here. And so they're looking at 500 milligrams per meter squared and 1,000 milligrams per meter squared of the 2 dosing comparisons. And that's based on pharmacokinetics. It's based on the FDA wanting the optimal dose. And so this is where it goes. The good thing is that it means that we have a dose optimization committee charter, which is we'll take a look-see at the halfway mark, and that look-see is coming in December. That's important.

Patrick Nelson

attendee
#35

Very good. All right. [Operator Instructions] otherwise, I think we've gone through everything that we wanted to go through. I think from our side, we look at this in biotech is something that trying to understand what you're investing in, the technical expertise that's required, especially in the early stages, quite a tricky space to play in. However, we are now at a point where we've got 1b results, the safety and the efficacy. We're not looking to see whether or not we have to up the dose or do anything funky to try and we need to see. We want to see this thing progress with similar results, even some way of what there could be materially value creating for the business and the investment and it opens up the whole world at the right time when deals are getting done out there, and we're seeing an escalation of deal activity and interest in this space. They're in the right place at the right time. It's particularly exciting window of time. And as an investor in Prescient, we've been on 5 years of the journey, the next couple of months are really -- we think there are significant inflection points and we're very, very excited to be involved. So I think it's an interesting time for anyone now getting new to the story coming in. I think it's something worth really paying attention to. And if you're thinking about a trade, yes, look, the share price can drift up from here, we definitely think, but there are some inflection points, which could be absolutely game changing. There are plenty of things to reference if you want to have a look, Mirati of the world, the Sobi deals and these other types of things where you can see that. It's not a little bit of a lift that we're talking about. Therefore, we think it's 1 worth having a bit of a think about. James, thank you very much for your time today and annual presentation. You've now 18 months into the role here. I mean, how are you feeling about PTX and where you sit?

James McDonnell

executive
#36

Yes. No, I'm pretty comfortable with joined as the CEO of Prescient knowing that we were advancing a Phase II program, which you don't often get a chance to do in Australia, and we're progressing it well. It's in a substantial market. We're also looking at that commercialization discussions with multiple partners. And so that's kind of my sweet spot as well, the commercialization and moving through the market and understanding those conversations. So I'm very happy to be here and working pretty solidly on this. So we -- as I mentioned before, we've got a very strong implementation of the study arm, and we're working very hard on the commercialization partnering discussion arm. And those are key critical steps for us as we progress. So it's actually a pretty good time to be here.

Patrick Nelson

attendee
#37

Excellent. Thank you so much. Thanks for everyone taking the time to attend. If you can any follow-up questions, please just let us know. All the very best. We'll see you all soon. Cheers.

James McDonnell

executive
#38

Thanks, Patrick.

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