Good afternoon, everyone, and thank you for joining the 2024 H.C. Wainwright 26th Annual Global Investment Conference. I'm Dr. Jade Montgomery, an Associate Research Analyst at H.C. Wainwright, and I'm happy today to introduce our presenter for this session, Russell Skibsted, CFO of Processa Pharmaceuticals, a biopharmaceutical firm developing next-gen chemotherapies to improve the safety and efficacy of cancer treatment. Russell?
Thank you, Jade. Appreciate it. And thank you, all of you, for joining us today to hear about Processa. First of all, I'll be making some forward-looking statements, so please refer to all our publicly available information before making an investment decision. So in a world where so many drug developers are conducting cutting-edge, exciting science projects, they're looking for the next great generation, great innovation products like KEYTRUDA, on our way to curing cancer, where we're never gonna have to worry about cancer again. Things like immuno-oncology, that works on more than 20% of the patients, like CAR T, the checkpoint inhibitors, cell therapies. As you know, most of those programs fail, and that's fine. That is the nature of science. But Processa is pursuing a different strategy. It's pursuing a de-risked strategy for new cancer therapies.
We're finding existing drugs, ones that we know work and are currently being heavily prescribed, but they're limited in their effectiveness due to toxicity. So we have a regulatory science approach that we're using in order to, like I said, we're taking these existing drugs, these drugs that we know work but are limited. If you think about things like Capecitabine, Gemcitabine, Irinotecan, they work, but they're limited in their effectiveness because so many patients are unable to continue the full treatment regimen because of the side effects.
And so what we're doing is we're working to either impact the metabolism of the drugs, or maybe the delivery methodology in which they're using in order to in essence try to make them less toxic, try to make them more effective, and maybe even more have a stronger have more potency. So currently Processa has three anti-cancer drugs that are in clinical development. We have two that are in clinic in phase II, and we have one that's near clinic-ready that we recently announced. We also have an experienced team.
We have an experienced team that's been responsible for over 30 regulatory approvals, and we've got it and had developed a proprietary regulatory science approach that we found and that our team has found works very well, and I'll talk about that in a minute, so this regulatory science approach is something that you would think that every drug developer would pursue. You know, what we want to be able to do is we want to be able to communicate early and often with the FDA. We want them to understand what we're thinking. We want to understand what they're thinking, and you know, but the reality is they don't.
A lot of companies don't do that, and whether it's because of hubris, ego, or fear, maybe, or I would say many of the companies, and maybe even most, do not have the level of communication that really creates a two-way level of communication with between FDA and the development of the drugs. We're all familiar with examples of companies or trials that have failed, and after the game film is looked at, you realize that it was failed due to poor communication or miscommunication with FDA. I, you know, one in my past that I'm familiar with was GPC Biotech, right?
This was a company that was a partner of ours, and 17 years ago, they had a phase III that, phase III succeeded, but the, drug never got approved. So, it's something to keep in mind. Dr. David Young, who's our Co-founder and has been involved in these over 30 regulatory approvals with FDA, he's developed and pursued and fine-tuned our regulatory science approach over his multi-decade career. Like, and then with what we're doing at Processa, we're combining this regulatory science approach with Project Optimus guidelines that the FDA issued last year for oncology drug development.
We believe that by focusing Processa on oncology, where we can combine our regulatory science approach with the Project Optimus guidelines, with the programs that we're looking at, where we've got drugs that we know work and that we're making them better, we think this could be a very successful strategy for us. I don't need to tell you that oncology remains a significant unmet need, as it remains the second leading cause of death in our country, with an expected more than 2 million new cases annually. But the thing that many people maybe don't fully appreciate, unless they're going through a cancer therapy, is that, you know, chemotherapy, it doesn't get the headlines. We don't talk about chemotherapies, right?
This is something that started many years ago, I think what, World War I, they with mustard gas, they discovered it. It's not exciting, it's not a new thing. It's something we hope one of these new science projects are going to come and remove the need for chemotherapy. But the reality is, it remains a foundation of cancer therapy, and it's projected to grow, not shrink in the future. This is an area that we're focusing on, because the reason that a lot of these chemotherapies are less than optimal is because of the toxicity associated with them.
So our approach, unlike the industry approach, what we talked about, let's go find those new, you know, cool new technologies, and I'm glad there's a lot of money going into that, because eventually, somewhere down the line, likely won't be in my lifetime, but we're gonna see great, great improvements in therapy and in survival, like we've seen with KEYTRUDA. But the science programs have a high failure rate in the clinic and in the market. So us focusing on these drugs where we have, you know, great benefit, but it's limited because of the toxicity, and we mentioned Capecitabine, Gemcitabine, Irinotecan. What we're working to do is improve how the body metabolizes the drugs, and yet, in many cases, even increase the ability of the body to metabolize the cancer-killing aspect of it, but to reduce the toxicity and but maintaining the efficacy.
So the NGC or the next generation chemo compounds that we're developing, you know, they have the potential to really improve the efficacy and tolerability of these cancer-killing molecules. And our goal is that if we can demonstrate that these drugs still work, but it's an improvement over the standard of care, then we think that through FDA's Project Optimus guidelines, that we should have a fairly clean pathway toward approval, with clinical success. And with that improvement, we think the markets could be substantial. So our pipeline includes the two programs in the clinic, both in phase II. We've got one that we just started a phase II in NGC- Capecitabine.
We have another program that has completed a phase II-A, and we're preparing for a meeting with FDA later this year, where we're going to finalize the more appropriate next pathway for trial for our phase II-B study, and we'll be able to announce that later on. And then, with the NGC- Irinotecan, it's preclinical, but we announced some very promising data recently, and we're moving toward completing the IND-enabling, you know, toxicity studies manufacturing so that we can get that program moving as well. So Capecitabine. What is Capecitabine? Probably don't need to tell this audience, but, you know, Capecitabine is a commonly used chemotherapy with significant side effects. Oral pro-D rug 5-FU, it's used in breast cancer, colon cancer, a lot of solid tumors.
What we do know is only 20%-40% of patients respond to Capecitabine, and largely this has to do with the significant side effect profile. If you're familiar with Capecitabine, Xeloda, you get about 50% of the patients get this very horrible side effect called hand-foot- syndrome, and it is a dose-limiting toxicity that these patients have. And because of that, many patients have to either go off therapy or reduce therapy. And yet, with that, in 2021, Medicare patients received over 9.2 million doses, and as we know, Medicare represents a percent of the total market. This is a heavily used foundational chemotherapy in two very critical cancers, and yet it's, it's highly toxic. So if we can improve that, we think that could be very important. What are we doing?
So what we're able to do by manipulating some of the chemistry that we're able to improve the metabolism in the body. So what we're seeing is actually that we're able to increase the percent of the anabolites, the cancer-killing molecules, in the tumors, while at the same time reducing the formation of the catabolites to less than 10% of what they were in the healthy patients. So, you know, the data to date is pretty impressive.
Where we look at, we're able to get essentially 5x- 10x greater exposure to the 5-FU in the tumors, and yet, what we've seen is with the toxicity, that we've only, we only had one patient in our study, albeit a small study, one patient with a mild case of hand-foot- syndrome, which represented 6% of the patients in the study. But, you know, given what you would expect with, with Capecitabine, you would have expected 50% or, you know, what would have been about six patients, give or take, to have, experienced that side effect. So we're very excited to move this program forward in the phase II study that we just started. But even though, you know, we were doing a phase I study in very late stage, cancer patients, we still had positive results.
You know, we saw some partial responses, we saw some progression-free survival, so again, very optimistic, very positive data so far to date that gives us a lot of excitement as we move forward in the phase II and in the future clinical developments of the program, but we do obviously know this was a small study, and it was an early study, so we've started our phase II study that I mentioned. This is gonna be a bit larger study. We are focusing in patients that are, excuse me, patients with advanced progressive GI cancers, and what we're looking at here is really, you know, is there a maximum tolerated dose that we get to?
And then, but more importantly, can we find an optimum dose for patients where we're gonna get the best concentration, the best improvement of the cancer-killing molecules in the tumor, but yet the best side effect profile and the best tolerability for the patients in the non tumor tissues. So the other piece of the study, which I want to mention, is it is an open label study, which is we expect the initial interim data to be published mid-next year. So this is one that, you know, although the study is scheduled to be a couple of years long, we do expect to be announcing data on that on a regular basis.
So NGC- Capecitabine, so far what we've seen is it increases the efficacy, it increases the distribution of 5-FU into the cancer cells while reducing the metabolites and side effects in the healthy tissue. We have patent protection on it. We filed some additional filings, which we expect to give us protection until 2024, 2044 or later. And the clinical development program, as I mentioned, is ongoing, and we're recruiting patients. Gemcitabine, it's our other phase II program. What is Gemcitabine? You know, it's a commonly used chemotherapeutic that's used in multiple solid tumor types. 20%-40%, depending on the tumor types, will respond to Gemcitabine. The big issue with Gemcitabine, you should probably be familiar with, is resistance.
It becomes a big problem with a majority of the patients, and as a result, the patients don't have quite the response that they might otherwise get, but what we found is that actually there's an additional target that if we're able to bring the Gemcitabine to this other target, we're able to overcome that resistance and bypass the receptor that built up the resistance. The other advantage to Gemcitabine is the fact that it's oral therapy, right? The original Gemcitabine, current Gemcitabine is IV, and the reason oral is important is obviously it's more convenient, but also, you know, Gemcitabine is many times given in combination with Capecitabine, among other things, which is an oral medication.
So it would be much more convenient for the patients and for the physicians to be able to match the dosing methodology for those drugs. So even with resistance, all this, we still had Medicare patients receiving over 840,000 doses of Gemcitabine in 2021. Again, represents 8% of the of those of the total patient. Studies to date have demonstrated that NGC- Gemcitabine is able to increase the concentration of the cancer cell and more cancer-killing molecules in the tumors, even after the cells have become resistant to the IV Gemcitabine. And this was due to the pathway that we're talking about, whereas the dCK pathway becomes resistant, the UCK2 pathway is able to still absorb the drug.
So data to date, you know, so far we've seen, again, in very late-stage patients that have been refractory to treatment, that we saw 31% of the patients with progression-free survival, eight weeks or more. We had five patients with stable disease for more than four months, and we even had a tumor reduction for 28 days. So, and the side effects that were experienced were mild to moderate, which would provide, you know, had no problem with the drug being further developed. So, what we're doing now is meeting with FDA to identify the right tumor type, the right cancer to go after, and then get a phase II-B study going, shortly thereafter.
So efficacy, again, the results to date, we've shown we can increase the efficacy of the into the tumor cells. Side effects are similar to that of Gemcitabine. We have IP that protects it, and I've already finished phase II and continuing into the second half of the phase II pro development after meeting with FDA. Irinotecan. I'm not going to spend a lot of time on this because, you know, Irinotecan is a very powerful drug that is, you know, is used quite a bit, over 1.8 million doses to Medicare patients in 2021. Again, a small percentage of the totals that received it. But, you know, Irinotecan, commonly used in multiple solid tumors. About 15%-35% of patients respond to it across different tumor types.
But the big issue with Irinotecan is that the SN-38, you know, metabolite, the side effects that are associated with that have generated a BlackB ox warning, and that's for diarrhea that can be fatal, and for myelosuppression, it can result in fatality. This is a very toxic drug, but it's one that works, and yet, like I said, even with the BlackBox warning and the serious side effects, it's still a foundational therapy that doctors are using in treatment solid tumors.
We have preclinical data right now, but what we found with our methodology is that we're able to get about 200 x more of the SN-38 in the tumor than in the muscle, and that compares to 15 x with Irinotecan or Onivyde, based on published information. We get significantly more of the drug into the cancer cells versus going into the healthy tissue. We then looked at it and said, "Well, if we're that much more potent, then by reducing the dose, are we able to maintain that efficacy with that lower dose, which would potentially lead to the removal of a BlackBox warning with the drug?" What we found is that, yes, in fact, we did.
We were able to maintain efficacy with a much lower dose. So, we're pretty excited about that. And, and then the other information we were showing was, you know, the tumor-to-muscle ratio of approximately two hundred for NGC- Irinotecan versus the 15%. Combining that with the tumor-to-plasma ratio of approximately 10 for NGC- Irinotecan versus less than seven for Irinotecan or Onivyde, in the... And then the, what I should say, in the non-cancer tissue, we demonstrated that we had significantly less of the drug in the non-tumor tissue rather than in the healthy tissue than either Irinotecan or Onivyde. So with this data that we have, we want to move quickly to try to get an IND approved.
We're going to be finishing some additional tox studies, and then we're, you know, working on the CMC filing and manufacturing to be able to get that submitted as soon as possible. We would expect, hopefully, to get most of that done, you know, by the end of next year and be able to get this program moving. It's an exciting one because it is so toxic, but so effective, that this is something that if we can get this to work, we think this would be a significant one. The physicians would happily use this in lieu of the other alternatives. We have a lot of milestones going over the rest of the year.
We're, you know, we're enrolling patients in our phase II study of NGC- Capecitabine program, meeting with FDA on the Gemcitabine program. We're also, we have some non-oncology assets that we're looking at, and we're actively seeking partners for those. And, you know, hopefully, we'll be able to announce something along those lines, if we're able to find a good partner. And, in 2025 , I think, you know, one of the biggest news items that we'll have, hopefully, is gonna be reporting the data, you know, the initial interim efficacy and safety data of the NGC- Capecitabine program.
Because that data should then give us a real good signal of what that, the rest of that development program is gonna look like and its likelihood of success with FDA. I mentioned we had a couple of non-oncology programs that we're looking at. If you're familiar with Processa, you looked at those positive press release last fall, about data we had to gastroparesis. It's really good. It's really good data. It's a disease state that needs to be addressed, but it's one that the clinical trials are a little more challenging to get to and a little more expensive to get to.
And as we wanted to, we thought that with the resources that Processa had, better to focus on oncology with the Project Optimus guidelines that we had, and be able to find a partner that is much better able to, to accrue and acquire the patients needed to get this program through the clinic, in a fast timeframe. Finally, we've got an experienced management team. And, you know, many of you, experienced investors have been around, familiar with Questcor.
You know, David Young and his team were instrumental at Questcor in being able to get Acthar approved, and you know, I think at the time that David joined, I think the Questcor stock price was about $0.50, and you know, the company ended up getting sold for $5.6 billion and over a $100 stock price. It's a team that's got a lot of experience, a lot of credibility, not only on the street, but also with FDA and with the, you know, the various investigational sites and the various KOLs that we're talking to. I feel pretty, you know, pretty lucky to be joining a team of this caliber.
So in summary, Processa has a de-risked strategy to develop better chemotherapies, next generation chemotherapies, where we can take advantage of a path, Project Optimus path that the FDA has laid out that is ideally suited to these type of development programs. We've got a great track record of our team of getting programs through the clinic and approved by FDA. These development programs, you know, they may not be the next immuno-oncology or the next cell therapy, but they're pretty exciting, and they really are cutting-edge science by making these toxic chemicals a little less toxic and a little more effective. And then, likewise, we've got some other programs that we're out licensing. So, hopefully, we'll even keep more of those programs for us.
So with that, I thank you, and feel free to reach out to me anytime you want if you have any further questions.
Thank you so much, Russell. I'd also like to extend a thank you to all of our presenters this year, as well as everyone who took the time to watch this presentation, and I hope you have a great rest of your conference.
Thank you.