... Good afternoon, everyone, and thank you for joining the twenty-seventh Annual H.C. Wainwright Global Investment Conference 2025. My name is Daniel Smith, and I'm an H.C. Wainwright Equity Research Associate in Biotechnology. With that said, let me introduce our presenter for the session. I'd like to welcome Lisa Conte, Founder, President, and CEO of Jaguar Health, a commercial-stage pharmaceutical company committed to identifying and developing first-in-class, plant-based prescription medicines for global unmet medical needs. Jaguar trades on the Nasdaq under the ticker JAGX. Lisa, the floor is yours.
Thank you, Daniel. Thank you, everybody. I'd like to start by saying this is the year of catalysts and events which we feel at the company will be transformative to value recognition in Jaguar. So I'm gonna go through those and just give a brief introduction to remind anybody or remind some people or people who are new what the company is all about. We are a public company, so I will dispense with the forward-looking statements. Also, Jaguar Health is the head of a family of companies, including Napo Pharmaceuticals, which is a wholly owned subsidiary of Jaguar. We trade under the name Jaguar, so I might use the names Jaguar and Napo interchangeably. What we do is all drug discovery and development, prescription drug discovery and development from plants that have been used traditionally in tropical areas.
That is our basic enabling technology to more efficiently find agents that are likely to be safe and active in man, because they've been in man for thousands of years, and that has been an effective drug discovery approach. We did take a product all the way from a tree growing in the rainforest, utilized by Elias there with our ethnobotanist and physician, to a first-in-class FDA-approved drug. The active agent is called crofelemer. The brand name is Mytesi. Mytesi is plant-based, it's organic, it's sustainably harvested, it's fair trade, and it's an FDA-approved drug, and it's the only oral drug approved by the FDA under something called botanical guidance, and under botanical guidance, there is no practical way to bring a generic to market.
So even though we have a full patent strategy like any pharmaceutical company, a hundred and sixty-something patents issued, new ones being filed all the time, we essentially have exclusivity to infinity and beyond, which is really interesting when we do terminal value calculations, for example, when we're negotiating license deals and business development deals. Now, crofelemer, as I mentioned, is already approved, and it's approved for the specialty market indication of HIV-related diarrhea, chronic diarrhea. Another word for specialty is relatively small. So why did we do the small market first? It was fast-track priority reviewed by the FDA, and there is a value in getting your first indication approved on the market.
The great value of crofelemer, beyond its paradigm-shifting mechanism of action, which we'll talk about in a moment, is that it is a pipeline within a product with multiple follow-on indications, almost all of which we have proof of concept phase 2 data. We can't do everything, so the ones that we have focused on in late-stage clinical development to bring to regulatory approval, with a target of the end of 2026, is cancer therapy-related diarrhea and orphan indications of intestinal failure for short bowel syndrome and MVID, microvillus inclusion disease. So I'm gonna tell you a little bit about that. The most recent catalyst that just occurred, that we just announced, is associated with MVID. MVID is an ultra-rare disease. There's about two hundred patients or so around the world.
We do have orphan drug designation in the U.S. and Europe, and we recently announced a reduction in parenteral nutrition in these patients of about 27%, which is groundbreaking, a remarkable achievement, and I'm gonna talk about that and put that in context in a moment. But what that has led to is a meeting that we now have scheduled, a face-to-face Type C meeting with the FDA, to talk about how, with our scientific advisory board, how to bring this product to this ultra-rare orphan population in an expedited manner. We now have four patients in clinical trials. That's about 2% of the entire patient population, and with this remarkable result for the first patient, that has been accepted for a presentation at NASPGHAN, the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition. That's occurring in Chicago in November of this year.
So what is MVID, and what is this groundbreaking result that we have? MVID is a situation where children are born with a fully intact intestine, a fully intact gut, but it's not functioning, so they're not absorbing the essentials of life, protein, carbohydrates, vitamins, et cetera. If they're not diagnosed immediately, they die, and if they are diagnosed, they immediately go on parenteral nutrition, IV nutrition, 20 hours a day, 7 days a week. Obviously, a major catastrophic healthcare event, and also, obviously, quality of life impact not only on the child, the patient, the family, the entire care-providing environment, physicians, nurses, nutritionists, everybody who's involved in the care of this child. TPN is one of the most toxic things that you can do to a patient, and so it leads to toxicity and risk of liver function, appropriate kidney function, cognitive function.
The children never get on an appropriate growth curve. The key thing that you can do to benefit these patients is provide some reduction in the amount of time that they're on TPN, time and quantity of TPN. So again, nobody ever thought that this was possible in an MVID patient. There's nothing in clinical development that we are aware of for these patients. There's nothing that has shown any sort of benefit like that. Crofelemer was able to show a reduction, as I mentioned, of 27% and increased urine production, which is an indication that the patient is better absorbing the nutrients of life, and reduction of stool volume. These patients typically have cholera levels of diarrhea. It's like a sieve, whatever goes in, comes out.
The data on the first patient was presented in May of this year at the PD League conference, a very prestigious international conference. It was in Abu Dhabi this year, and that patient's result, which were first presented there, have now continued, where the patient, after 12 weeks, according to the investigator-initiated protocol, was taken off crofelemer, and within 10 days, the relapse was so terrible, the patient was put right back on crofelemer, and we will be supplying crofelemer for the rest of that patient's life. There was also a first patient in this investigator-initiated trial who had intestinal failure due to short bowel syndrome.
The same implications due to a short bowel and not having enough surface area, the patient was unable to absorb the nutrients of life and went on parenteral nutrition, as opposed to MVID, where there's full surface area, but the intestine is just not functioning. Same implications, manifestations, the patient goes on parenteral nutrition. In the case of the intestinal failure with short bowel syndrome, the patient had a reduction of TPN of 12.5%. Same situation, when the patient was taken off crofelemer, they relapsed, and again, that patient has been put on, and we will be providing product for the rest of that patient's life.
These are both situations where crofelemer is modifying the disease progression, and in particular, in MVID, because it is ultra-rare, we are looking to qualify for PRIME designation in Europe and potential breakthrough designation in the United States. As I mentioned, we do have a meeting scheduled with the FDA, Type C face-to-face meeting, to address how can we get crofelemer to these patients, MVID, in as expedited a process as possible? Now, the product is not the current approved formulation of crofelemer. Mytesi is a tablet, an enteric-coated tablet. A tablet would go, you know, right through these patients because of the volume of diarrhea that they have. This is a highly concentrated liquid formulation that's appropriate for the medical situation.
It is therefore a different product, and it's gonna have a different business model that is more typical of what you see with rare diseases, where there's high cost, high mortality, high morbidity, high patient, family involvement, and, as I said, a whole different business model associated with the product. Third-party market research puts the short bowel syndrome market in anywhere from $5 billion to, we have seen, as high as $12 billion a year. The MVID product, which is ultra-rare, would be a smaller market opportunity. However, it is the same formulation, the same product that is in clinical trials in short bowel syndrome as well. So right now, we have two major programs associated with crofelemer, a third minor program. We have multiple catalysts that have occurred and that are occurring real time, including four clinical trials that...
or I should say, two phase 2 clinical trials on multiple continents, the U.S., Europe, and the Mideast, for short bowel syndrome and MVID. The manifestation of these clinical and regulatory catalysts associated with crofelemer is to move to where we are right now in business development discussions and negotiations to license our products and bring in non-dilutive dollars, to allow us to continue to support these clinical programs, to continue to support the regulatory and the development activities, and bring these products in an expedited way to patients, hopefully by the end of 2026, with expedited planning. Additional catalysts in our other major program. I should say that the two major programs are intestinal failure, which we've talked about, cancer therapy-related diarrhea, which I'm about to talk about, and also an animal health program.
We do have crofelemer approved under the brand name Canilevia, Canilevia CA1 for chemotherapy-induced diarrhea in dogs. Each one of those programs is a separate, encapsulated business development opportunity, again, looking for non-dilutive dollars, negotiating for non-dilutive dollars to help offset the development costs in the company. Cancer therapy-related diarrhea. That has been a passion and an ongoing program for about eight years now that we are bringing to conclusion based on an FDA meeting that we had earlier this year. This is Mytesi, so it will be a supplemental NDA for a drug that is already approved, a formulation that's already approved, a chronic safety package that's already been approved, and what we're looking for is based on the additional efficacy in cancer therapy-related diarrhea, a supplemental NDA to expand our opportunity to educate, promote, and get reimbursed for Mytesi for this indication.
We did a prophylactic trial for all solid tumors, patients on targeted therapy. There are about 24 different targeted therapies, with or without cytotoxic chemotherapy. It concluded last year. It was a big, bold study to embrace the entire solid tumor cancer patient population, and we missed the primary endpoint, and that had a lot to do with the variability of different types of cancer patients. However, and everybody forgets the however, however, in breast cancer patients, which were over 60%. I think it was 63 or 64% of the patients in the clinical trial, we achieved statistical significance in a responder analysis, and that was presented at the very prestigious San Antonio Breast Cancer Symposium.
Based on that data, we met with the FDA earlier this year, again, a face-to-face meeting, and the outcome of that meeting has provided a pathway where we're looking to expedite the approval in breast cancer patients. So one of the responses that we got from that meeting is the data from that trial is invaluable, including learning more about the natural history of diarrhea with these new targeted therapies, a first-of-its-kind study. The meeting kicked off with patient advocate participation. Our patient advocates are formal members of our scientific advisory board, and they gave very real and raw descriptions of their experience, including one of our patient advocates, who had received an off-label prescription for Mytesi. She's metastatic. She's on salvage therapy at this point and was not able to stay on her therapy. She told her husband she was done.
She was putting out 10 to 15 watery stools a day. Once she received the prescription, she had a remarkable response. That was eight months ago. She continues to respond today and described that situation to the FDA. We had another patient advocate who talked about really what makes life worth living and not just existing, and where we came out in agreement with the FDA is that the results of the prophylactic trial are supportive of an efficient pathway to move forward, and where we're going to move forward is in metastatic breast cancer patients, and that also qualifies as an orphan indication, so it continues our focus and our strategy on orphan indications and some of the regulatory flexibility that gives a company in that situation. The trial that we will move forward with is a treatment trial.
It's a low-risk trial relative to the prophylactic trial in all the different patients that we talked about because of a minimization of variability, and then we are immediately going to start, and immediately are starting, an expanded access program for patients who would not qualify for that trial, so that patients right now who are experienced the type of diarrhea that is preventing them from staying on their life-saving cancer therapy, as well as having some form of a quality of life, would be able to get access to Mytesi. This is a graph of the rates of diarrhea with some of the targeted therapies that are out there.
So, to clarify, targeted therapies. This is not the chemotherapy that we see in the movies, the toxic chemotherapy, you lose your hair, the fatigue, et cetera, which also have a rate of diarrhea, but are typically for, you know, six months, 12 months, depending on the cancer and the relapse of the cancer. Targeted therapies are these agents that are taken every single day for the rest of their life, typically in a metastatic patient, and even in a curative situation, nine months, sometimes as high as several years, to keep the cancer in check, to make it a chronic situation that the patient is able to live with.
All the targeted agents, TKIs, epidermal growth factor receptor antibodies, CDK4/6s, have a diarrhea that is induced by chloride ion secretion, which is the type of mechanism that crofelemer uniquely is able to normalize, as opposed to Imodium, loperamide. These are opioids. They work by the mechanism of constipation. This is a very targeted agent to stop specifically what's causing the secretory diarrhea, and these are some of the rates of diarrhea that are out there. Imagine, even... There's four grades of diarrhea. Even Grade Two is four to seven loose, watery stools a day with uncertainty, the surprise. How is that possibly a tolerable situation for the rest of your life?
And that's where the strong patient advocacy to bring this relevance to the regulatory agency, because it's not just quality of life and patient comfort, which is extremely important, but about 40% of the time, the patients are going off their therapeutic dose or going off the treatment completely, very specifically because of the side effect of diarrhea. Now, interestingly, crofelemer, as I mentioned, is approved under the name Canilevia CA1 for chemotherapy-induced diarrhea in doggies. Dogs often also get targeted therapy. Interestingly, they often get human-approved therapies. An amazing rate of cancer in dogs, about one in four, about one in two over the age of 10, and cancer is often underdiagnosed for any number of reasons.
Remarkably similar to the human market, about 40% of the time, dogs who are being treated with chemotherapy end up having to go off their therapy specifically because of diarrhea, and the quality-of-life issues are expanded and even more highlighted and poignant in the animal population because it's the dog's comfort. You can't talk to the dog, and they just suck it up and deal with it for a bit. Then also, there's the quality of life for the family and the home, the carpets, the bed, for a dog that has lost control and is remarkably uncomfortable. And this is, you know, as just underscoring what we know from one of the vets who is using Canilevia, that this innovative therapy has allowed the dog to stay on his or her cancer therapy.
So it's a remarkable situation here in the United States right now, that if you're a cancer patient experiencing diarrhea, to be educated, promoted by the company, you have to be a dog, not a human, not yet, and that's what we're working towards happening in a very communicative and collaborative process with the FDA. We do have revenue, about $13 million a year. This is primarily from Mytesi for the approved HIV indication. We did increase about 35% second quarter over the first quarter. We released those results a little bit ago, and continue to see growth in this market, about, you know, 5% or so a year.
It'll never be bigger than maybe a $25-$30 million market, but has been really, really important as the first product that we've commercialized, to learn how to differentiate a novel mechanism of action, a paradigm-shifting way to treat and potentially cure various gastrointestinal disorders. The team is remarkable. There are 15 of us who've been... or 10 of us who've been together for over 15 years, five of us who've been together for over 30 years, committed to bring crofelemer to all the different populations around the world in need. We will continue to have a direct market presence in the United States. At the very least, we might do a co-promote. But around the United States, and we have global unencumbered rights to crofelemer, we are looking for partnering and licensing deals.
And I shouldn't say we are looking; we're in the midst of negotiation based on the catalysts that we've had this year, again, to bring in non-dilutive dollars and recognize the difficult situation of financing small market cap companies right now. And I'll leave you with the investment highlights, all of which have been discussed, and conclude at this time, and thanks very much for the attention.