All right. Hello, everyone, and welcome to the Jaguar Health presentation, and we're going to do a fireside chat combo here with this next one. Again, my name is Robert Bloom, Managing Partner at Lithium Partners, and up next here is Lisa Conte, the Chief Executive Officer of Jaguar Health. As a reminder, Jaguar trades under the ticker symbol JAGX on the Nasdaq. Lisa, thanks so much for joining us today.
Oh, thanks, Robert. I'm happy to be here. This is my favorite conference, and it's an important week, so I'm happy to give an update.
Fantastic. Well, why don't you go ahead and work through a couple of slides that you've prepared, and then we'll jump into Q&A.
Okay, thank you. We are a public company, so there's our forward-looking statements. As a reminder of who we are and what we do, we do all our drug development from plants that are used traditionally in tropical areas. The name is Jaguar Health. We have a wholly owned subsidiary called Napo Pharmaceuticals. So every once in a while, I might use those words interchangeably. It's the same company.
We took a product all the way from a tree growing in the rainforest to a first-in-class FDA-approved anti-secretory agent with really a paradigm-shifting mechanism of action to normalize gut function across multiple diseases, multiple patient populations. So the generic name of the product is called crofelemer. The brand name is Mytesi, and that's the first approval for a specialty indication of chronic diarrhea in people living with HIV/AIDS.
It's also FDA-approved by the Center for Veterinary Medicine for a conditional approval for chemotherapy-induced diarrhea in dogs under the brand name Canalevia-CA1. Crofelemer, Mytesi, Canalevia is plant-based. It's natural. It's organic. It's fair trade, sustainably harvested, and it is an FDA-approved drug. And it's the only FDA-approved drug, only oral drug under botanical guidance. And under botanical guidance at the FDA, there is no clear pathway to bring a generic to market.
So even though we have over 150 patents issued and always looking for new IP opportunities, we essentially have exclusivity to infinity and beyond because of the approval under botanical guidance. This was a very exciting week for the company because we have been commercializing Mytesi. It was approved on December 31st, 2012.
Earlier this week, we entered a license agreement, an exclusive license agreement with a company called FuturePak, which is a private company that recently acquired Theratechnologies. Under this license agreement, this can be worth up to $38 million to us with $16 million, $18 million as an upfront payment, $16 million, which already came into our bank account this week, and about $20 million in milestones based on commercial progress. To give you a feel for why this is important and why this happened, we have about eight sales reps. There are five to six times as many at Theratechnologies. Theratechnologies has products that focus on supportive care.
So their targets are physicians who are looking at, find the importance of the dignity and life of treating supportive care, and also salvage therapy for HIV patients, which is important in the long-term survivor community, which is another important target for the diarrhea indication and therapy, inflammation, leaky gut syndrome. So it really makes sense for these products to be together.
And the products are now in the commercial tutelage, the commercial leadership of Theratechnologies, and Jaguar is able to focus on its pipeline, and particularly with these funds which have come in, which are non-dilutive. And so where we're focusing on our pipeline, first and foremost, is our rare disease program, which is a global program.
And it gives us the opportunity to potentially bring a product to the market next year to FDA filing by the end of this year, 2026, for the rare diseases that we are focused on. Jaguar also, Jaguar Napo, continues to be the manufacturer of crofelemer, so we're manufacturing for Mytesi, which is being commercialized by Theratechnologies, owned by FuturePak, so manufacturing has actually become a profit center for the company at this point.
I'm going to stop at this point, and this is the most exciting deal of the moment, but the pipeline, of course, is where our future blockbuster opportunities are. Mytesi really put us on the map and gave us this opportunity to bring in these really important funds into the company this week.
All right. Perfect. Well, thanks for the update there. Let's start right there. First off, congratulations on the agreement here with FuturePak. What was really the genesis of this deal?
It really came from, as I was talking about, the match of the targets for their two products and our product. Supportive care sometimes just doesn't get the attention in any field, in HIV, in cancer. Our rare disease program, where you have disease modifying, so there's a little bit more importance associated with the normalization of gut function, and because Theratechnologies is focused on supportive care, and as I mentioned, also the population that we're dealing with,
it was just a natural match for the products and has been for some time, and then we had to figure out what is the deal to make that happen, so it's really a bonanza win for the HIV community, which is so meaningful to us to get that word out, even with greater depth and greater breadth.
Yeah. So I guess, as you move forward here now, does it make sense for investors to view Jaguar as more of a research and development-focused biotech, sort of in this rare disease space now that the transfer of the marketing and sales responsibilities for Mytesi and Canalevia have moved to FuturePak?
Absolutely, so our commercial organization is gone at this moment with now a strong, strong focus, and in particular on our rare disease programs because our two rare diseases are MVID, microvillus inclusion disease, which is an ultra-rare disease, which is in the midst of a clinical trial right now. We've met with the FDA about the opportunity to file for this ultra-rare indication with a single trial and potentially even a single-digit number of patients because it's so ultra-rare,
which would mean filing this year in 2026. That is crofelemer, but it's not Mytesi. It's a completely different formulation that's appropriate for the physiology and the age and the medical situation of these patients. It's a highly concentrated, lyophilized, ultimately liquid formulation. That is the same formulation for short bowel syndrome patients as well.
We're also in a phase two program for short bowel syndrome, though the lever to get the product on the market to these intestinal failure patients comes from the ultra-rare indication of MVID. Now, this is still crofelemer. So the manufacturing, the CMC of the active ingredient, the safety that all carries over from this long legacy that we have had with crofelemer/Mytesi in the HIV patient population.
And these indications are blockbuster. Third-party market research puts the short bowel syndrome market at over $5 billion. We've seen some as high as $7 billion or $12 billion. And there's nothing new out there. The only thing out there is basically a growth hormone approach, which has a lot of limitations, in particular in cancer patients, patients that have had surgery where they need to have bowel adaptation, where you can't have a growth hormone out there.
Maybe taking a step back here, what sort of led you to focus on primarily these orphan and rare diseases?
There was an initial inquiry years ago that came from a physician and came from some of the patient advocacy groups who are constantly searching for answers for these, in this case, for children who are affected, so let me give you some idea of what the disease is. In short bowel syndrome, as the words say, short bowel, you don't have enough real estate. You don't have enough surface area to absorb the nutrients of life, so patients often end up on parenteral nutrition, parenteral support, 20 hours a day, seven days a week, and what you're trying to do is decrease those secretions a bit so they have a bit more opportunity to absorb their own nutrients of life.
And if you can get them off parenteral support even by 10%-15%, it's a huge, huge impact, not only in quality of life, not to be hooked up to IVs all the time, but the intense toxicity associated with parenteral nutrition, the liver toxicity, the kidney toxicity, the cognitive toxicity. So now if we move to MVID, same thing, intestinal failure, but they don't have a short bowel syndrome.
Their gut is fully intact, but it's not functioning. So a growth hormone approach doesn't work for them. There's nothing to grow. It's already there. It's just not functioning. So these kids either die when they're born, if they're not diagnosed, or if they're diagnosed from the moment they're born, IV nutrition for the rest of their life. They normally die when they're about 12 or 13 years old.
As I mentioned, patient support groups, families are constantly searching for ways to modify the disease progression. It actually came to us from the physician community and the patient community, an anti-secretory agent approach. We presented data this past year, 2025, that was groundbreaking. Proof of concept, MVID patient, we were able to reduce parenteral support by 39%. Unheard of in how that changes the patient's life and is life-extending.
They're basically on life-shortening therapy in order to live or toxic therapy in order to live. On the SBS side, the preliminary proof of concept data, I believe, was up to 12%-15% reduction in parenteral support. If that data continues, and we now have two patients who have completed, two MVID patients who have completed the phase 2 clinical trial, we'll have six to eight patients totally in that trial. That type of data could be sufficient for breakthrough designation in the United States, prime designation in EMA, and regulatory filings globally this year, 2026.
Yeah. You mentioned $5 billion on SBS here a moment ago. Talk about sort of the opportunity for intestinal failure.
Yeah. And so in SBS, there is a product that's approved right now. It's called, very hard to say, teduglutide. And as I mentioned, it's a growth hormone. It's a GLP-2, not a GLP-1, a GLP-2. And so what it attempts to do is to grow the gut a bit so that the patient can reduce their parenteral nutrition by 10% or 15%. It is approved. The only thing that's in the pipeline are other,
I would say, perhaps analogs of GLP-2, those that perhaps can be administered with a different frequency or a different side effect profile. Two have been unceremoniously refused by the FDA. So there's nothing really happening in that particular pathway, that particular mechanism of action. Growth hormone treatment is limited in short bowel syndrome and is not standard of care.
It can't be used, as I mentioned, in a cancer patient where any sort of hyperproliferative abnormal situation, you wouldn't want to give a growth hormone. So that's a lot of surgical patients who have had their bowel resected because of bowel cancer, GI cancers. Also, about a third of the patients have had bowels resected anyway. They could be Crohn's disease, inflammatory disease, and they usually need about 12-18 months of bowel adaptation before you could give a growth hormone.
And then there's a lot of side effects of the growth hormone approach as well, in addition to the side effects of the parenteral nutrition. Crofelemer, with its beautiful safety profile that's been out there for well over a decade in the sickest of the sick HIV patients, not systemically absorbed, disease progression modifying, it's a totally new way of thinking about treating and potentially curing diseases. That's the real opportunity for it to come in and be a standard of care.
All right. Very good. Coming back to, on November 8th, I think you were at the, it was the annual meeting of the North American Society for Pediatric, what was it, Gastroenterology?
Gastroenterology, yeah, Pediatric.
There you go. Yeah. And talk about the results that were presented there and what it really means for your development plans for crofelemer.
Yeah. So as I mentioned, we have two double-blind placebo-controlled trials going on, one with MVID, one with SBS. We've met with the FDA. So the one for MVID is the one that we have filed for an amendment and also to continue to treat the patients who finish that trial, which, of course, the physicians and the families want based on the results that they're seeing.
We also have investigator-initiated trials going on, which are not placebo-controlled. And three patients, two short bowel syndrome and one MVID, I hope I have that right, were treated in Abu Dhabi or still being treated in Abu Dhabi at the Sheikh Khalifa Medical City, Dr. Mohamad Miqdady. He did a first presentation. I think they had been treated for about six-to-eight weeks at their international conference, which was in May of this year.
Groundbreaking, standing ovation from the physician community, the treating community. That was the first demonstration of reduction of parenteral nutrition between 8 and 10% in short bowel syndrome and 20-something% in MVID, and then those patients continued to be treated. Actually, there was one step first. They were taken off crofelemer.
They relapsed, and then they were put back on crofelemer, and the reduction in parenteral nutrition continued such that the MVID patient is now down to 29% reduction, and that was just absolutely groundbreaking. Nobody really recognized that an anti-secretory would be able to have that much of an impact on this very, very toxic life-shortening treatment, TPN, that is necessary to keep these patients alive, and that is so toxic to the patients.
All right. Very good. I want to come back. You talked briefly about breakthrough therapy designation, both from the FDA as well as the EMA. Talk about why these programs are beneficial.
Yeah. So it's Breakthrough Designation with the FDA. So that can give us the opportunity, as I mentioned, with a single trial, single-digit number of patients, again, supported by the strong, strong safety package of crofelemer that's already demonstrated. So when you think of the FDA approving something based on benefit-risk, if your risk is zero, your benefit-risk ratio goes to infinity.
And so with that designation, we could get approval in a short period of time, less than six months, even after we file the new drug application. This would be a different new drug application than Mytesi, as I mentioned, a different formulation, yet benefiting from the CMC that is already part of the API of crofelemer that's already approved.
In Europe, prime designation gives you an opportunity for a lot of communication and sort of speeds along the whole regulatory process for the entire EU, which I think is 27 different countries. So with prime designation, you would have an accelerated pathway to get a full approval for the entire EU universe.
And these two, well, distinct parallel pathways are somewhat influencing each other because particularly when you have an ultra-rare disease like MVID, there's a few centers of excellence around the world where these patients go. And by the way, most of them are involved in our clinical trial with great enthusiasm and great benefit that they're seeing for the patients. And then this is the link to Short Bowel Syndrome. You'd be getting the same product that is utilized for Short Bowel Syndrome approved, while Short Bowel Syndrome will have to have a pivotal trial after our phase 2 completes.
All right. Very good. Let's transition to Canalevia here for a moment. Congratulations are in order. They award, I think it was last month, a grant for Canalevia-CA1, development from the FDA, and then approval earlier in December by the FDA of the renewal of the conditional approval for Canalevia-CA1. Talk about maybe what the grant funds will be used for.
Yeah. So Canalevia CA1, to put that in perspective, that is our second FDA-approved product for chemotherapy-induced diarrhea in dogs that is also part of the license agreement. So FuturePak will now be commercializing that approval in the United States. That approval ends in December of 2026. So the renewal that we got in December happens every year for five years. This is the fifth year. In order to keep Canalevia available in the United States for chemotherapy-induced diarrhea, we need to do a study to show expectation or to show efficacy to full efficacy.
It was conditionally approved because the need is so dramatic in chemotherapy-induced diarrhea. So it's called a MUMS approval, which is like orphan drug on the human side. So this then would be a full approval for chemotherapy-induced diarrhea. And that will become a discussion through FuturePak and Jaguar.
However, that approval, and we have funds now, too. We're in the midst of that clinical trial. We're about two-thirds of the way through with those funds get to be applied to that clinical trial. So in addition to the non-dilutive funds from the FuturePak license agreement, we have these funds that are focused on that clinical trial.
We do have the rights to crofelemer, of course, outside the United States, and there's a great deal of interest in not only chemotherapy-induced diarrhea, but general diarrhea, general acute diarrhea in doggies as well as in cats and all sorts of other animals because GI dysfunction, as we know, is a problem in all mammals, and if you look at the mechanism of action of crofelemer from the perspective of a gut, it's a highly conserved target between humans, dogs, cows, horses, so it applies to all mammals.
All right. Very good. In the couple of minutes we have left here, a question I know I've asked you before, but maybe to remind folks that haven't had a chance to listen to some of our past discussions, talk about sort of the link, if you will, between cancer indication for crofelemer in dogs and then in people, right, in humans. How are they sort of related?
So the point that I just mentioned, the target mechanism of action, which is these chloride-mediated channels that are in the guts, are highly conserved in people and dogs. And then in particular, cancer is rampant in dogs now. It's like 50% of the dogs over the age of 10. There's a whole bunch of diagnosis that doesn't happen, of course, for dogs because it just doesn't happen.
They get targeted agents. We have completed a first phase 3 study in cancer therapy-related diarrhea in humans, those who are on targeted therapies. Dogs get targeted therapies as well. Dogs often get human therapies. So here they are as a model and a patient population at the same time. With the deal that we've done, the license deal, we've done the license deal for our approved indications.
It leaves our business development opportunities for these blockbuster indications of cancer therapy-related diarrhea for Mytesi, of course, the rare diseases for MVID and short bowel syndrome, and even outside the United States for other animal indications intact, and those are the business activities that we will continue as our commercial operations have shut down. We focused on R&D, manufacturing, and then the business relationships for these blockbuster indications.
All right. Very good. In a couple of minutes left here, talk about the key milestones investors should be looking for here in 2026 and maybe any final takeaways.
Yeah. The key milestones are on the clinical side, MVID trial and the SBS trial. We'll finish this year. MVID should lead to breakthrough designation, prime designation, a global regulatory filing. Business development deals, we will not be commercializing outside the United States. So business development deals on the rare disease side, independent from other indications for Mytesi, independent from Canalevia.
So three different shots on goal, and hopefully we hit them all for business development deals. And then watching Mytesi having, as I said, greater depth and breadth of commercial resources devoted to it from an organization that is devoted to the commercial aspects and the serving of the HIV community, which leads to milestone payments to Jaguar and Napo.
All right. Very good. Well, we will leave it there. Lisa, thank you as always for joining us here. Greatly appreciate it. Thank you to everybody here for watching. If there are any additional questions or would like to schedule a meeting with Jaguar, please reach out to the company's investor relations team, or I'd be happy to coordinate as well. Again, my email is bloom@lithiumpartners.com. We do have additional presentations, fireside chats coming up. So please continue to stick around. Lisa, again, thank you so much.
Thank you. Great questions. Enjoy the discussion.