Welcome to the Life Sciences Investor Forum. On behalf of OTC Markets and our co-host, Zacks Small Cap Research, we are very pleased you have joined us. The next presentation of the day is from Jaguar Health. Please note you may submit questions for the presenter in the box to the left of the slides. You can also view a company's availability for one-on-one meetings by clicking Book a Meeting. At this point, I am very pleased to welcome Lisa Conte, Founder, President, and Chief Executive Officer of Jaguar Health, which trades on Nasdaq under the symbol JAGX. Welcome, Lisa.
Oh, thanks so much, Lily. I really appreciate it, and always pleased to talk about my favorite topics, Jaguar Health, in front of a new audience. We are a public company. Let me dispense with those forward-looking statements. Now to go into the introduction. Jaguar Health, as you mentioned, trades on JAGX on Nasdaq. We have a wholly owned subsidiary, Napo Pharmaceuticals, so I may use the names Jaguar and Napo interchangeably throughout the presentation. What we do is all drug discovery from plants that are used traditionally in tropical areas, and we did take a product all the way from a tree growing in the rainforest to a first-in-class, FDA-approved antisecretory agent. The active ingredient is named crofelemer, and the brand name that is approved by the FDA is Mytesi for humans.
There is a product approved under the Center for Veterinary Medicine of the FDA, Canalevia, for dogs. crofelemer is a natural product. It is plant-based, it is organic, it's sustainably harvested, it's fair trade, and it's an FDA-approved drug, and it's the only oral drug approved under botanical guidance by the FDA. Under botanical guidance, there is no practical pathway to bring a generic to market. Even though we have a very robust IP patent strategy like any other pharmaceutical company, we essentially have exclusivity to infinity and beyond, which is very valuable as we talk about terminal value calculation, for example, in business development and licensing deals. What's really wonderful about crofelemer is based on this normalizing, as I mentioned, first-in-class mechanism of action in the gastrointestinal area, it has multiple indications.
It is currently approved in the human area for chronic diarrhea in people living with HIV/AIDS. In the animal area, it is conditionally approved for chemotherapy-induced diarrhea in doggies. However, the pipeline opportunities, pipeline end product include multiple follow-on indications. At this point, we have licensed Mytesi to a company called Future Pak. The rest of this presentation, let me give you the preview, is going to focus on our sharp strategic focus on pipeline opportunities, late-stage clinical opportunities in rare disease indication of intestinal failure. That is in fact being fueled by the license deal that we just did with Future Pak, which brought in substantial non-dilutive resources. We did the deal in January of this year, so just two months ago. $18 million upfront payment, license fee payment to Jaguar. In March, there was an additional $3 million payment.
There are up to $20 million in milestones and other future payments. Again, these are all non-dilutive dollars, which has been a main business development strategic focus of the company, and we did come through with that. Jaguar continues to be the manufacturer of crofelemer. There's important economies of scale in manufacturing the product, and we do supply it to Future Pak at a profit, so it is essentially a profit center, the manufacturing of the product. Why did we choose Future Pak? Why did Future Pak choose us? About six months ago or so, Future Pak acquired a company called Theratechnologies and Theratechnologies . has products that focus in the HIV marketplace, in particular for salvage therapy and supportive care, the exact overlap of the patient demographic that can benefit from crofelemer.
These are patients who are typically long-term survivors, a great deal of inflammation in their gut enteropathy, which leads to a leaky gut and chronic diarrhea. Again, a beautiful match for the mechanism, the target of Mytesi, and so much more efficient for one sales force to have three products in the bag. That's financial fuel that is out licensed, and now I'm going to spend the rest of this time talking about what's very exciting for us and our late-stage clinical programs in the rare disease area. These are crofelemer, same active ingredient, but a completely different formulation that is physiologically and clinically necessary and relevant for the populations that we're going after, so it will be a new drug application, a new product, but the same active ingredient.
The indications are pediatric congenital disorder, pediatric microvillus inclusion disease, and short bowel syndrome and intestinal failure. These are both intestinal failure indications. We have clinical proof of concept that you're about to hear about and a very near-term regulatory and clinical catalyst program in front of us. Rare disease, just to remind anybody who's in the pharmaceutical industry, it's very much in vogue now. The reasons for that are these are populations that are defined as less than 200,000 patients in the U.S. It's defined differently in Europe, but it comes out to be about the same number. Very rare for a particular indication, but interesting that about 10% of the population as a whole is dealing with a rare disease and orphan indication.
It is something that's important to the population as a whole, and of course, to the particular patient advocacy groups. Small populations, but high morbidity, high mortality, typically lifetime treatments, very expensive, a lot of patient advocacy for accelerating treatments and reimbursements. They're typically quite valuable markets. Third party market research puts, for example, the short bowel syndrome market, we're going after short bowel syndrome, intestinal failure, at more than $8 billion in 2033. There's regulatory advantages, greater communication with the FDA, smaller clinical trials, so much less expensive, particularly for a small company to go after. In intestinal failure, what's happening? What does that mean? It means a patient who doesn't have the capability to absorb the nutrients of life, proteins, carbs, vitamins, et cetera.
Typically, these patients exist on total parenteral nutrition, parenteral support, IV nutritional support, 20 hours a day, seven days a week. Obviously, a huge impact on quality of life, catastrophic for the patient, and very serious physiological impacts. There are toxicity to the liver, toxicity to the kidney, cognitive functions for children. They go off their growth curve. What you're looking to do is reduce the toxicity associated with the parenteral support by reducing the time and the necessity to be on parenteral support. If you could reduce the time the patient is on parenteral support, even by 5% or 10%, a huge impact on quality of life, health benefits, the toxicities associated with this, and extension of life. The endpoints that you're looking for are the reductions of parenteral support, enhanced urine output.
That means the patient is absorbing more of the nutrients of life themselves. Reduction in stool volume and stool formation because it's like a sieve, and as you can imagine, these patients are just having chronic watery diarrhea. Let's focus on Microvillus inclusion disease first. Intestinal failure is the umbrella rare disease indication. We do have orphan designation in the U.S. and Europe, both for microvillus inclusion disease and short bowel syndrome. Two separate intestinal failure indications that share the same treatment paradigm. Microvillus inclusion disease, these are patients who are born, it's congenital, and if you think about the intestine, it has like little fingers, villi, that absorb the nutrients of life, they're inverted. These babies, these infants, these newborns can't absorb the nutrients of life, and they're just born with massive diarrhea.
If they're not diagnosed immediately, they die immediately. If they are diagnosed, they immediately go on life-saving parenteral nutrition, parenteral support for the rest of their lives. They're on IV support for the rest of their lives, the rest of their short lives, because of the toxicity that I mentioned that's associated with the parenteral nutrition, the parenteral support, the kidney, the liver, cognitive, going off their growth curve. Everything that I mentioned happens to these kids in addition to infections from being on IV nutrition and the lines that are into them. They typically don't last, you know, past 11, 12, 13 years old. It's a lethal natural history of the disease. Again, what would make a difference? There's no treatment whatsoever out there for these children. Their intestines are fully intact, but not functioning.
Is there some way to reduce their need on parenteral support? We're very excited. We're very, very excited. Investigators were very excited. At NASPGHAN last year, which is the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition meeting, we presented the first proof of concept data with 1 MVID patient and two short bowel syndrome patients who also had intestinal failure and were on parenteral support, reductions of up to 37% in MVID and about 15.5%, 15.6% in the short bowel syndrome patients. It was groundbreaking. It was unheard of. It was a standing ovation from the clinical investigators. Very, very exciting. In addition, this was an investigator-initiated trial, so they were just given crofelemer treatment.
When they were taken off crofelemer, and it was supposed to be for 30 days, the families immediately demanded to get back on the product because they relapsed very quickly, and then they were put back on crofelemer and continued to show improvement. It's now been over a year, no safety issues whatsoever, and those children have maintained those levels of reduction in parenteral support. As I said, hugely important, not only to their quality of life, the entire family, the entire set of caregivers, but to the potential extension of their life because of the reduction of toxicity risk. What's happening now? MVID is an ultra-rare disease. There's about 200 patients around the world. This gives the opportunity for approval with literally a single-digit number of patients. We are now in the midst of a pivotal trial.
It is completely enrolled for what we are seeking to get FDA-fileable data for a new drug application by the end of this year, with a filing in the beginning of 2027. We'll also be seeking with continued performance of the product as we've seen, and of course, the safety profile, seeking Breakthrough designation towards the end of this year, which can give even a further accelerated approval, and having that FDA-approved product available to children by the end of 2027. We've already met with the FDA and had multiple communications back and forth, and one of the key aspects of those communications is with each patient having a bit of a unique situation, how do you write a label that is appropriate for this particular population?
Which is a very, very positive sign from the regulators that we are talking about how to write a comprehensive label. What this chart shows is just the progression. As I mentioned, all the patients that we intend to seek a filing for are enrolled in the trial. It's a placebo-controlled trial. The patient is their own placebo or their own control, so they either go on crofelemer or placebo, and there's a crossover effect. Then at the end of that, should there be any benefit that is seen, then the patient can request to go into a treatment-only extension, which gives us even longer-term opportunity to collect the safety and the benefit of the product. At this point, we are expecting all patients to go into the treatment-only extension. All those who have finished the placebo control have already requested to go in the treatment-only extension.
What you're looking for with the FDA is benefit/risk. With zero risk, this product has had no safety issues. Remember, the active ingredient is already on the market in very sick, compromised HIV patients. We've never had a serious adverse event associated with the active ingredient. Benefit/risk, your risk is close to zero, your benefit is close to infinity and beyond. Very exciting for these patients that have no alternative treatment. Now I'm gonna move to intestinal failure with short bowel syndrome. This is the same formulation of the product, the same situation, intestinal failure patients who are on parenteral nutrition for 20 hours a day. It's sort of a different situation for the cause. These are patients. Our intestine, a normal person, is typically 25 ft or so. These are patients whose intestine are 5 ft or less.
It could be due to an accident, cancer, surgical resection, inflammation, IBD, Crohn's disease. It could be congenital as well. For whatever reason, they don't have enough surface area to absorb the nutrients of life. Same treatment, parenteral nutrition, but a different cause than MVID, where the intestine is fully intact. They have the same risk of toxicity and the same endpoints that we're looking for. There is a convergence of activities here. MVID, because there's no alternatives whatsoever in the ultra-rare nature of the disease, we're seeking to file a new drug application by the beginning of next year, 2027, based on the pivotal trial that's ongoing right now. We also have a placebo-controlled trial going on in intestinal failure with short bowel syndrome, and there'll be the convergence.
We'll have those results out at about the same time that we're filing for MVID. The same formulation, MVID approval is a stepping stone to then get to the pivotal program for short bowel syndrome, which is a larger market. Still an orphan indication, but a larger market. Instead of 200 patients around the world, we're talking about 40,000 patients around the world. Again, third party put that market opportunity at about $8 billion. How does crofelemer do what it's doing? It's really a paradigm-shifting treatment. Short bowel syndrome does have a product that's out there now. It's called Gattex in the United States, Revestive in Europe. It's a GLP-2, not GLP-1. It's a GLP-2, so it's essentially a growth hormone. What it does and attempts to do is grow.
This is in short bowel syndrome, grow the gut a bit so that there's a bit more surface area to absorb the nutrients of life and therefore get to that important toxicity-reducing endpoint, which is the reduction of need on parenteral support, Total Parenteral Nutrition. It did establish a regulatory endpoint, a primary regulatory endpoint of the reduction of parenteral support, and it has in clinical trials achieved that reduction of about 10%-15%. It's not standard of care. It's utilized in less than 10% of the patients. It cannot be used. It's a growth hormone, so it can't be used in a patient that has cancer or any abnormal hyperproliferative disease. Of course, you don't wanna give a growth hormone in that situation. Can't be used in patients who have had surgical bowel resection for about 18-24 months.
There's some other toxicities. As I said, it did establish a primary endpoint. It establishes a need and a reimbursement. It's reimbursed at the rate of about $500,000 a year in the United States. Again, as I talked about the mortality, the morbidity, and the expense of taking care of IF patients. We are looking to become the standard of care in IF. Some of the advantages that we have with an agent, an API, an active ingredient that's already been approved. Two most common reasons why new drug applications get pushed back or get failed are safety and manufacturing.
We already have those aspects of the product approved, and what we're looking for is continuation of the type of the response that we are seeing already in our published clinical proof of concept studies continued as we finish up the placebo-controlled trials that we have. I'm gonna move on from our rare disease program right now and just talk a bit about the continuity of what we were doing with Mytesi. Again, Mytesi at this point for the HIV indication has been licensed out to Future Pak. That formulation, different than the IF, intestinal failure formulation. That formulation of Mytesi was in a first pivotal trial, phase III clinical trial for cancer therapy-related diarrhea.
It was a big, bold study that looked at patients who are on targeted therapies, those therapies that you stay on for the rest of your life to deal with cancer as a chronic situation. Tyrosine kinase inhibitors, epidermal growth factor receptor antibodies. These are agents that all work by a mechanism that causes secretory diarrhea. We did not achieve the primary endpoint with all comers, all tumor types, 24 different targeted agents. However, we did in breast cancer patients. This is an important endpoint because we're not talking about garden variety diarrhea here. About 40% of patients on cancer therapy go off their therapy or go to a subtherapeutic dose specifically because of the reason of diarrhea. Much more expensive. They end up in the hospital having to be hydrated. Strong patient advocacy for supportive care.
There's about 20 different unmet medical needs in supportive care in cancer. We will not be pursuing this because it's going to require another clinical trial unless we have a partner supporting it. Our sharp strategic focus, our non-diluted dollars from the Future Pak deal are going towards our rare disease program, but I did wanna mention that this is another asset that we have for future business development. You never know. There's all sorts of news that comes out of the FDA, and Makary, the new head of the FDA, is talking about maybe there should only be one pivotal trial necessary approval for new indications. That could change the game for cancer, but not at the moment. Interestingly, crofelemer is approved for chemotherapy-induced diarrhea. In doggies, conditionally approved.
If you are a cancer patient in the United States experiencing diarrhea and you're a dog, we can promote and educate you and get a prescription for crofelemer, but a human not yet. That's an interesting point. That product is also licensed under license to Future Pak. We do have a lot of assets in this company from firsthand field investigation where crofelemer came from. Working with healers and looking at symptoms, signs and symptoms, and what are some of the novel agents that come from plants that have been in man for thousands of years.
We have about 2,300 plants in our collection, 20% of which have psychoactive or mental health utilizations in the field, and those are the subject of a joint venture with another company that has natural product chemistry capabilities, to look at new ways to potentially cure and treat mental health disorders. There have been so little innovation in that field. It cost us nothing, it mobilized an asset that we had, and it came with outside funding from a venture capitalist. It sort of leverages some of the enthusiasm that's going on right now with psychoactives and psychedelic agents. They're all chasing the same thing, and as psilocybin, MDMA, ibogaine, what's the next generation of paradigm-shifting mechanisms of action as we have done with crofelemer? We have a lot of catalysts, and these are meaty.
We already have clinical proof of concept, and that will continue in our rare disease program. As you can imagine, getting ready for an NDA filing in early 2027, there's a lot of regulatory communication, regulatory catalysts, much greater access because of the rare disease and the orphan indications, both in U.S. and Europe. These are global filings. In the U.S., there's breakthrough designation, which accelerates the development even further, or the regulatory approval even further. For a rare disease in Europe, there's something called PRIME, which we will be filing for. Our trials are occurring on a global basis. It includes U.S., Europe, and the Mideast, particularly congenital disorders, where based on consanguineous marriages, you have a higher incidence and prevalence of these diseases. Our team has.
There's about 10 of us who have been together for over 20 years. We are absolutely committed to bringing crofelemer to all populations in need in all different parts of the world. As I said, there's a great economy of scale of manufacturing this agent and then getting further down in our pipeline. These are investment highlights that I'll leave up at the end and try to leave a few moments for questions. Thank you very much for allowing me to introduce you to Jaguar. Okay, let's see. Question. FDA has already responded in February 2026 allowing continuation of your MVID study. How confident are you that the 68-patient design will be sufficient for a breakthrough therapy designation and a strong NDA?
With the type of clinical performance that we have seen in the proof of concept in MVID patient and patients, I am as confident as you could possibly be that the six patients is going to be sufficient. Remember, six patients is like 3% of the global population for a lethal natural history disease that has absolutely nothing in development. I did wanna make one other comment. When I talked about intestinal failure in short bowel syndrome, I did mention that there is a growth hormone approach that is out there. Can't use that for MVID. The intestine is fully intact. It's just not functioning, so there's nothing to grow further. Let's see if I can go down further. Theratechnologies acquisition, how do you see the strategic alignment accelerating Mytesi growth versus what Jaguar could have done alone? Thank you for that question.
I love the collaboration and the license to Theratechnologies. You know, these products should have been together a long time ago. Theratechnologies has one product for salvage therapy, so antiretroviral therapy for salvage therapy. Who ends up on salvage therapy? It's the patients that typically have had HIV in their gut for over 10 years, which by the way, over 50% of the HIV population now has had the virus in their gut for over 10 years, is over the age of 50. They're sort of affectionately referred to as long-term survivors. These are also patients who went early on the first really toxic therapies like AZT and really, you know, shredded their gut.
The inflammation they are facing overall in their body from the long-term infection from HIV is what triggers the enteropathy in the gut, the leaky disease, the chronic diarrhea. As I said, complete overlap with the targets of patients for salvage therapy. One of the interesting things about diarrhea is patients, HIV patients who are dealing with whole body inflammation have acceleration of aging disease. They have earlier onsets or accelerated onsets of cardiovascular disease, diabetes, cancers. Often you don't see those, the silent killers, but you do see the symptom of diarrhea. Really important in that overlap population. Their other product is for lipodystrophy, which means physicians who are prescribing that are attuned to supportive care. Sometimes physicians are not fully attuned to supportive care, and Mytesi falls into that category as well.
Very, very excited that these products should have been together long ago. Now they are, and confident that for those reasons it'll accelerate the growth of the revenues of Mytesi. Okay. Another question. I think I have time. Do you see an opportunity to expand from pediatric into adolescent and young adult MVID and SBS-IF populations over time, and how meaningful could that be commercially? Yes, I see the opportunity for more MVID patients to become adolescents and young adults. As I said, with the reduction of the toxicity by reducing the amount of parenteral support and TPN, those very, very toxic agents, we would look for extension of life. We do know of one patient with our clinical investigator who is in. I'm not sure if it's a he or her. I think it's her.
In her late twenties, has been exquisitely monitored over her life. We would be really proud to expand the number of patients who are living with MVID. With your validated commercial scale supply chain for the lyophilized crofelemer powder, how quickly can you scale if demand in MVID and SBS-IF exceed initial expectations? Scaling of manufacturing is our pride and joy. It's something that I learned years ago from one of my important mentors in the pharmaceutical industry, G. Kirk Raab, who's now dead, who's the CEO of Genentech. He was chairman of our board for a long time, and he impressed on me very, very early on that supply, manufacturing supply, GMP manufactured supply, you gotta think in advance. You need stability. You know, you can't do two-year stability in less than two years.
That is an at-risk expense when you're in clinical trials to make sure that you have supply and are never out of supply. There's nothing stupider than getting a drug approved and not having sufficient supply for it. We are completely confident of being able to supply any need for crofelemer for any indication in any population that we're pursuing right now. I think I better stop 'cause I'm out of time. Thank you so much for the time and attention and the really great questions.