Good evening, everyone. Thank you very much for joining our Global Healthcare Conference. I'm Miyabi Yamakita, a Japan biotech analyst at Jefferies in Tokyo. This is a fireside chat session with PeptiDream, represented by CEO Patrick Reid. Patrick, thank you very much for your time today. Would you like to start with the overview about the business model and platform technology of your company?
Yes. Thank you, Miyabi-san, for the introduction. Yep. Patrick Reid, one of the founders and Representative Director and CEO of PeptiDream. This is our 20th year of operations. We were founded as a spin-out company from the University of Tokyo in 2006. We initially spent the first 10 or so years of the company as a platform company, largely focused on developing a novel and highly diverse macrocyclic peptide discovery engine, which we then leveraged to enter into a number of discovery partnerships with most of the global pharmaceutical companies in the world, and really played, I think, a large contributing role to the creation of the macrocyclic peptide environment and where it is today. Over the years, we have focused on five core modalities or uses of macrocyclic peptides. Using them as oral peptide therapeutics or injectable peptide therapeutics.
Using peptides as RI-PDCs or using the peptide to deliver targeted RI. Using peptides to deliver oligonucleotide drugs. Utilizing peptides to deliver cytotoxic drugs is the fourth area, and then as the fifth area is utilizing peptides as bispecific, trispecific, what we call MPCs formats to replace some of the complex protein engineering molecules that are out there in the space. We've focused these five core modalities across a number of different global partnerships, of course. We now are at a transition phase where we are seeing the fruits of our efforts, of our preclinical and discovery efforts, seeing a lot of these programs now very quickly move into clinical testing. With that was a part of the success was we actually had six programs move into clinical development in 2025.
We ended the year with 13 clinical stage programs, largely across collaborations or partnerships. As we head into 2026, we expect to see anywhere from 6-12 programs entering clinical development. We're very much at a transition or natural evolution of the company where we see a lot of driving our clinical expansion, pipeline expansion. We're complementing those efforts, of course, our collaborations, of course, with our wholly owned internal pipeline, to which we're seeing the first four compounds that we will bring into the clinic here in 2026 ourselves. It's a very exciting time for PeptiDream. I'm looking forward to today's discussion.
As you mentioned, in recent years, the number of clinical programs at PeptiDream has increased rapidly, which is impressive progress. My first question is, what is driving this rapid increase in clinical stage programs, particularly in radiopharmaceutical area? Which pipeline are you most excited about, if any?
Certainly, we've probably made the most progress around our utilizing macrocyclic peptides for delivering RI. Those are quite impactful and move quickly. We had programs with Novartis move into the clinic. We've had programs with RayzeBio or now BMS move into the clinic, and we have, as I mentioned, our first wholly owned programs for CA9 and claudin 18.2 moving into the clinic this year. I think that's driven largely because macrocyclic peptides are really the ideal ligand to deliver these radioisotope payloads very effectively to tumors. They have excellent PK that very quickly arrives and accumulates in the tumor, and what doesn't accumulate in the tumor is very rapidly renally cleared, I think making them, again, optimal for this type of use.
There's been a lot of hard work behind that over the years with our partners and ourselves to optimize the peptides to do this type of function. Beyond RI, we're also seeing, again, the non-RI applications of oral peptide therapeutics. We have a number of oral peptide therapeutic programs that are really driving or in the clinic or in driving toward the clinic, which again, is only being further supported by the success of Merck in PCSK9 and J&J and Protagonist, their IL-23 receptor. It's a wonderful time to be in oral macrocyclic peptides. Again, beyond that, we're seeing some real success, I think, in our other modalities also. It's the contribution of years of preclinical discovery and collaborative efforts that are now finally being harvested to see us progress a lot of programs forward. Yeah.
Great. In terms of the modality, as you mentioned, you have now three new modalities, which are Oligo-PDC, cytotoxic PDC, and multifunctional peptide.
Yeah.
Could you explain the current progress in these areas and when we can expect further updates in these areas?
Yeah. Thus far, our clinical programs are largely RI or oral or injectable peptide therapeutic programs. As you mentioned earlier, we've had some exciting news that we've announced recently from one of our partnerships. We have some very strong partners in the peptide oligo delivery space, which I think we're moving toward the first clinical development program announcements here. It's a very exciting time utilizing macrocyclic peptides to deliver oligos ex liver, specifically to other tissues in organ types. Quite convincingly, I think the peptides are proving to be extremely effective in taking ASOs or siRNAs specifically to these cells and/or organs. Really excited about the application there. We have some really compelling partnerships in that space covering a variety of different types of targets and organs. We're expecting those to yield the first kind of clinical phase I starts probably in 2027.
Again, these are being led by our collaboration partners. I can't speak to any specific timelines. I think that's really exciting to see that modality proving its worth and proving the investment that we've made. Also in the peptide cytotox, this is not necessarily a novel space with the ADCs and other peptide-drug conjugates out there. We have a very strong partnership with Merck in this space. That's been very productive. I think we're nearing a stage, again, not to speak for Merck. We're nearing a stage where we're really seeing the fruits of those efforts leading toward, I think, clinical programs in the not so distant future. I think that modality or that effort for us is also going to be rewarded. We're looking forward for the outcomes.
I think macrocyclic peptides, similar to RI, are an ideal way to deliver cytotoxic drugs to tumor cells and offer some potential compelling differentiation compared to an antibody-based approach. This isn't trying to simply just replace the ADC approach. This is offering some differentiation and in some cases, a potential better effect that we could see. Really exciting to see both of those two, our third and fourth modalities, then contributing clinical programs to the pipeline, which is further going to allow us to expand our overall clinical pipeline faster. Lastly is around MPCs, which is we're largely focused on a number of different kind of areas in that space, which we'll have more to come in the future and we'll disclose more as we see those near clinical stage.
Okay. Thank you so much. In terms of the oral peptide, I understand that the myostatin inhibitor is no longer your only key product.
Yeah.
Because you now have multiple important programs such as IL-17 and in your pharmaceutical area, there are multiple candidates.
Yeah.
Some people are still worrying about the progress of the myostatin program. Could you explain your current development or partnering strategy on myostatin?
A number of years back, we've been a discovery company that's partnered with large pharma largely, of course, some mid-size and small-size companies too, where we discover the peptides, we optimize the peptides in collaboration and allow the partner to develop those forward. As you can imagine, as the world has shifted, and I think this is a natural evolution, we started to do more of our own in-house programs. Initially that focused, I think, a little bit more on RI, but is also, of course, consists of some oral, macrocyclic peptide programs that we have, including myostatin, which started a number of years ago, and IL-17, which we've also made oral. These are just two examples that we talk about. We don't normally like to talk too much about our preclinical programs until we have clear partnership strategies, I think, in place.
Around specifically on the non-RI side of the business, myostatin has been one of very attractive assets that we have. We think it's a very compelling program. I believe still we're the only company with an oral myostatin inhibitor. It's extremely potent. Because it's a macrocyclic peptide, we see excellent distribution from the blood into the skin and muscle, really the site of action, where we really want to inhibit myostatin, which we think is a key differentiator from the biologic-based approaches. As far as partnering, there remains a lot of excitement, of course, as inhibiting myostatin or the myostatin activin pathway in the context of muscle preservation, combined with these weight loss agents, which I'm sure everyone is very aware of.
For us, the partnering strategy on muscle preservation would be finding the right partner, which means finding the right partner is not just about the financial value of any deal. It's finding the company that has a good oral weight loss agent that we would be able to combine with or formulate with to make an end oral product. That is the end goal, of course, for use in that aspect. Those are discussions that continue to ongoing. We had those all through 2025. We still have a number of discussions ongoing, but when you're trying to make a combination product and one of the pieces comes from somebody else, it's a little bit more like a marriage than us just selling something to somebody and then hoping.
I think the oral obesity space is still a fluid environment for many of the companies, maybe besides the top two players. I think there's more to be seen as far as that kind of indication use. The other use that is very attractive for us, we originally started the program for DMD. I know, of course, the myostatin antibodies did not do so well in DMD in the past, and potentially a big part of that was the different distribution of an antibody versus a macrocyclic peptide. When we ran the animal studies in the DMD, where we couldn't get any real efficacy with the antibody approach, but our macrocyclic peptide yielded very exceptional efficacy in returning grip strength, one of the key aspects that you want to see in these animal models.
We could actually see that with a peptide approach versus the antibody approach, which was quite striking. Really gave us confidence that a peptide potentially diffusing into the muscle, and localizing to the muscle at a different rate than a biologic does, could give you a higher exposure into really the sites of action that you need to be hit. We, in addition to considering myostatin for muscle preservation indications, of course, is also looking at the variety of muscular dystrophies, DMD for one. Of course, with the exciting news around SMA, and the breakthroughs in that space and the potential upcoming approval of a myostatin inhibitor for SMA from Scholar Rock. Even potential other related muscular dystrophies. We think there's a path forward there also, and there's interest there in that space.
For us, the myostatin program, again, I think it's an exceptional program that we have. There's some complex biology and there's some complex business to be considered on the best way to find a partner that's going to help us bring this to patients, which is the end goal at the end of the day, not just getting a short term upfront. I think as far as guidance, I don't know when any specific deal will happen, and my goal isn't the date of a deal. My goal is to find the best deal to maximize the value of this exciting program, both for patients and potentially shareholders. I would say again, in addition to myostatin is our very exciting oral IL-17 program, where we have AA, AF, and FF cross. It has a very, very nice profile. Again, orally bioavailable.
We think this is a very exciting program where we've put the knowhow of oral peptides to this type of program. It's something also that we're considering all of our development options for, whether that's developing it ourselves, whether that's partnering in that, and even partnering is from large pharma all the way to mid-size to small. There's a variety of options that are being considered for this. I think this is really a part of our strategy is to make best-in-class assets, take advantage of the power of these macrocyclic peptides, and then, from a partnering approach, finding the best strategy to take those forward. Yeah.
Okay, I'd like to ask about the in-license thing as well. In the RI-PDC area, you in-licensed the Curium program, which is progressing well. Do you expect any further in-licensing? If so, what kind of program are you targeting?
Yeah. PeptiDream is a 20-year-old macrocyclic peptide discovery company. In 2022, we acquired what was one of two radiopharmaceutical businesses in Japan, which we renamed PDRadiopharma. That company was actually started in 1968, has radioisotope handling licenses, has manufacturing, sales, regulatory, has the full spectrum, and really complemented our own discovery efforts that we had been doing. That company already sells 32 products in the Japan market, largely on the diagnostic side of the equation, but is a very strong business that again will allow us to take some of our targeted peptide RI compounds through the Japan market. The synergy is that our deals with, for example, RayzeBio BMS, our Glypican-3 project with them, we secure the Japan rights to that program.
They have ex-Japan rights, we can bring that to market in Japan now that we have this radio pharm business. It's additive in that way for us to attain more of the value or retain more of the value of the peptide RI programs we're developing ourselves or even collaborating with. In addition to that, as you mentioned, we're supplementing that by in-licensing. Japan really just has three players at current in the radiopharmaceutical space, and one of those is what was called Nihon Medi-Physics, which has been fully acquired. It was partially owned by GE HealthCare. Now it's fully owned by GE HealthCare. Novartis, who has entered the Japan market in the recent years here. We're the kind of really maybe only company to be able or amenable to partnerships to bring in assets from outside Japan into the Japan market.
We're of course taking advantage of that to supplement our own efforts. That resulted in the licensing of or the deal with Curium to bring their potential Pluvitco competitor into the Japan market, both the copper diagnostic and also the lutetium therapeutic. Which through the partnership, which is a co-development, co-sales partnership, we just put those into registrational studies very recently this year. Exciting to see how those go. We also have a glioblastoma drug that we in-licensed from LinkMed in Japan that also is now in a phase III registrational study in Japan for glioblastoma, or a number of type of gliomas. Yes, that's a supplement to the business. We do expect other type of in-licensing deals. As you know, we sell Omemvia in Japan and Talvey in Japan for Lilly.
We have a long history of kind of being the partner of choice to allow these companies to bring their radiodiagnostics or radiotherapeutics to the Japan market. This is something we will continue to do, and there's a robust number of assets now globally available with the rapid expansion of radiopharmaceuticals globally. We have a number of discussions ongoing, but we'll of course be selective in what we can do and what we can bring into the Japan market. I do think that's going to be a growing part of the business also. Yeah.
Okay. Thank you so much. Apart from the R&D, you introduced a new organizational structure from April of this year. Could you explain the purpose of this transformation?
Yeah. Thank you. With this transition, we're kind of evolving from just largely a discovery company now into a discovery and development, right? Instead of simply just finding these drugs and, again, giving these to partners to develop and crossing your fingers that they develop them forward, we've decided to take on more, utilize the profits from that type of business and invest this into expanding our own internal pipeline, which means taking programs into clinical development. We think that's going to be a continued expansion of the business. It's a natural evolution of the company to begin with, but that requires a different skill set, right, and maybe a deeper management team that can handle those type of aspects and those type of efforts. We are looking forward to it.
Here in July, we will be announcing a number of new hires joining the company. We started an executive search in late 2025, which has resulted in, I think, finding some really promising, some very highly experienced individuals that will be coming on board to the company. We also evolved the management into an EVP, SVP, VP kind of situation, where we've promoted a number of internal people into VP roles. I think, again, this management structure, while maybe very common in the U.S., was kind of something that was much needed at the PeptiDream side to really put us in the position for the next 10 years of company growth.
Very excited again to start to announce some of these and talk more about this management structure, which I think will just simply deepen our expertise and allow us to deliver value to both patients and shareholders faster. Very excited about this, yeah.
Great. Okay, I would like to ask a little bit about the capital allocation.
Yeah.
In the case you successfully close some deals in the next couple of years.
Yeah
Of course it will generate a decent size of cash flow.
Yeah.
What are you going to use the cash flow for?
I think at this phase of the company, of course the radiopharm business has very stable quarter-to-quarter revenue that's slightly growing, of course, as sales increase. The PeptiDream side of the business is largely driven by R&D funding and milestones or new deals, which can be very quarter-to-quarter volatile, as everyone is very much aware. The goal, of course, is to continue to expand the clinical pipeline to transition into royalty-bearing programs, right, that brings long-term stability to revenue. That's the, again, I think the natural evolution of any biotech company. That's really what we're focused on. PeptiDream is, I think, a rare exception. We went public in 2013, so 13 years as a public company, and besides the IPO, we've never sold shares. We've never capital raised. We've actually been profitable for 10 of the last 11 years.
We've continued to reinvest those profits into our own pipeline, right, and further expanding our shots on goal, I guess, if you will. I think over at least certainly the next five years, that is what we will continue to do, right? Our goal has always been to simply operate in the black and then utilize those profits to take more shots on goal, right? Depending on the profitability of any given year, allow that to kind of dictate, do we take one program in the clinic this year, or do we take two programs in the clinic this year? I think that's the model over the next couple of years that we will continue, right, to operate under. Also, again, certain programs we'll simply out-license to bring in revenue to fund other programs and continued expansion.
I think we're really at that stage of growth as a company. We'll see over the next couple of years, the end goal is to get as many products to market as possible, both across our collaboration partners, but also driving as much value from our in-house programs to deliver those end goals.
Okay. AI remains a hot topic in every industry. Any benefit from AI for you?
Yes, certainly. Our libraries consist of on-demand made 1 trillion macrocyclic peptides in a library that we basically select for through an iterative round over round kind of process. That type of process is really amenable to large language models and prediction algorithms, right? I think over the 20 years, we've generated so much empirical data that we've been able to feed that data into better and better prediction algorithms to allow us to know the kind of peptide hits that come out of this discovery process to tell us winners and losers in some respect, at least on the drug discovery side. I think the main benefit of AI, if you will, for us is maybe less about picking winners, but it's really good at telling us who a loser is.
I think that over the last couple of years has probably been part of the largest value for us instead of having to make maybe 1,000 peptides to get to the one that we really want, that we can make 100 peptides and get to the one we really want because our algorithms allowed us to weed out 90% that were never going to be the favorite peptide. I see that as if we can continue that really adds a significant layer of efficiency, operational efficiency, and get the same outcome at a far lower cost, which is cost control is, again, super beneficial for a company like us at this stage.
That's, of course, not getting into the attributes of AI on all of the back office related aspects, of course, from writing press releases, to back-end kind of legal, other accounting aspects, just some of the operational efficiencies on the back office side that AI and the various tools are bringing to the equation. Also, I don't think it should be lost on anybody what AI is bringing to the table as far as searching the research literature. One of the biggest challenges for any discovery company is staying abreast of the most recent findings and the recent methods, the scientific literature. When you're a company like us that has a lot of programs spanning a lot of therapeutic space, it's impossible for you to stay on top of the scientific literature, and AI is in fact making that possible.
We are big fans, of course, of AI, of everything back office. On the drug discovery side, I think is, again, our large language models and our prediction softwares are bringing cost efficiencies that are going to be super beneficial for us downstream. Yeah.
Okay. Thank you so much. I think we covered most of the topics. I have one more question about your developmental strategy. CA9 program in RI.
Yeah.
Could you help us understand the rationale for conducting the global phase I trial in-house on your own? Could you also remind us of the timeline?
For our CA9 program, we had run a phase 0 or an imaging study in Japan. That data was presented at ASCO GU last year. It was very compelling data, which gave us a lot of confidence to decide to take this forward ourselves into a phase I. Again, with the RadioPharm business, we think we had the skill set. We had been growing a development team at PeptiDream also to support the clinical development. As you know, we have a number of programs already partnered with Novartis and RayzeBio, BMS, and Genentech Roche, we really felt it was the right time to take some of these exciting programs, our in-house programs, into the clinic ourselves. CA9 was the first one. We're taking that forward in a U.S. phase I.
We filed INDs at the end of last year, which were accepted both for a copper-based diagnostic or an imaging agent and an actinium-225 therapeutic agent, both targeting CA9, of course. Those will start a phase I actually momentarily, in the very near future. Should be first patient, first dosing here this summer. Again, we followed our CA9 with our 18.2, which was our second program, which we'll be taking in the later half of the year into a phase I in Australia, which has some interesting operational differences compared to going forward in the U.S. We clearly have a global development footprint now. I think PDRadiopharma just gives you the ability really to see and treat these tumors because the imaging agent lights up the tumors, and then you can really see the therapeutic benefit of your agent.
Because both have the exact same peptide attached and the only difference is the radioisotope, I think you know what the imaging agent is going to yield. It's going to yield therapeutic benefit when you switch from the copper to the actinium-225. We're very excited to see how those programs do. I think we have high confidence that they're going to yield very strong data. Normally, the phase I in RI is kind of an escalation into an expansion phase type of design. I'm not sure again when we'll start releasing data, but usually that can take two years to 2.5 years to complete the entirety of the design study. We think as soon as we can release data, we will release data. I don't have any specific timelines for when yet.
That's going to give us some really strong programs to take forward here. Very excited to see where those go.
Okay, great. We are running out of time, so let's conclude this meeting here. Thank you very much for joining, everyone.
Thank you.