Good morning. Before I turn the call over to management, I'd like to remind you that management may make forward-looking statements relating to such matters as continued growth prospects for the company, uncertainties regarding market acceptance of products, the impact of competitive products and pricing, industry trends, and product initiatives, including products in the development stage which may not achieve scientific objectives or meet stringent regulatory requirements. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those contemplated in such forward-looking statements. These statements are based on currently available information and management's current assumptions, expectations, and projections about future events. While management believes its assumptions, expectations, and projections are reasonable in view of currently available information, you are cautioned not to place undue reliance on these forward-looking statements.
The company's actual results may differ materially from those discussed during this webcast for a variety of reasons, including those described in the forward-looking statements and risk factors sections of the company's Form 10-K for the year 2024, which was filed March 31, 2025, and its other filings with the SEC, which are available on the investor relations section of Jaguar's website. Except as required by law, Jaguar undertakes no obligation to update or revise any forward-looking statements contained in this presentation to reflect new information, future events, or otherwise. Today's conference is being recorded. At this time, it's my pleasure to turn the call over to Lisa Conte, Jaguar Health's founder, president, and chief executive officer. Lisa, the floor is yours.
Thank you very much. Happy to speak with everybody and greet everybody this morning. Thank you, all of you, for joining. As you heard, my name is Lisa Conte, and I'm the founder, president, and CEO of Jaguar Health and our wholly-owned subsidiary, Napo Pharmaceuticals, and I am the chairman of our Italian subsidiary, Napo Therapeutics. As usual, I may use the words Jaguar and Napo interchangeably when referring to our company. I am here with Dr. Pravin Chaturvedi, my longtime colleague, chief scientific officer, and chair of our scientific advisory board. He also will quite likely use the words Napo and Jaguar interchangeably. Pravin is the successful developer of seven new drug applications, including his favorite and mine, crofelemer.
A few moments ago, we released initial results from a proof-of-concept study for intestinal failure associated with MVID, microvillus inclusion disease, an ultra-rare disease, and intestinal failure associated with SBS, short bowel syndrome, another rare disease in pediatric patients. In both situations, intestinal failure requires total parenteral nutrition, which is going to be referred to throughout this webcast as TPN, at least 50% of the time, IV nutrition. For MVID patients, TPN is necessary from day one of birth to survive, and that need does not change during their short life, a short life due to complications of liver, renal failure, IV line complications, among others. The biggest impact one can have on a patient with intestinal failure is reduction in the quantity and time of TPN.
These results that we have out today show that crofelemer reduced TPN in the MVID patient by up to 27% and 12.5% in the SBS patient. These results, in the words of frontline healthcare professionals, HCPs, healthcare professionals, are groundbreaking medicine that have the opportunity to modify disease progression for a catastrophic patient condition, intestinal failure. Why that is important is best expressed by the principal investigator in this proof-of-concept trial and the real-time reaction of his colleague, a key opinion leader, a leading investigator and practicing expert healthcare professional of pediatric patients with intestinal failure. We have an impactful session here today.
You will hear directly from them in a fireside chat. I'm going to ask my colleague, Pravin, to introduce the principal investigator, Dr. Mohamad Miqdady, and Dr. Tzivinikos, and describe the congress both Pravin and I attended this past weekend in Abu Dhabi with these two experts, building it right to letting the professionals speak to why these results with crofelemer are transformative for patients and all Jaguar stakeholders involved. Pravin and I will then come back and reflect on what this means for Jaguar's business development and drug development strategies and ongoing activities, and we'll take Q&A. Questions can be submitted via the webcast link for today's event that appears on the events and presentations page of the investor relations section of Jaguar's website. The URL for Jaguar's website is jaguar.health. I'll now hand the discussion over to Pravin. Good morning, Pravin.
Thank you, Lisa. Good morning, and good morning, everybody, to all of you who woke up early to attend this. I am very pleased to discuss the results from the recent presentation at the Elite Pediatric Gastroenterology Congress that we attended, Lisa and I, between April 24th and April 26th, which is the brainchild of Dr. Mohamad Miqdady, who is the Chief of Pediatric Gastroenterology, Hepatology, and Nutrition at Sheikh Khalifa Medical City, SKMC for short, in Abu Dhabi in the United Arab Emirates. This is his annual conference. Now it's in its 11th year, which brings over 300 leading pediatric gastroenterologists from around the world into one room.
Because many pediatric gastroenterology diseases are complications that have a congenital aspect to them, the Middle East-North Africa region, which is called the MENA region, is kind of central to this research because of the prevalence and the incidence of these rare congenital disorders. The innovation in diagnosing, preventing, treating, and potentially sometimes even curing some of these disorders, the leaders of that world are in the Middle East-North Africa region. As such, SKMC, Sheikh Khalifa Medical City, and other institutions in the United Arab Emirates, such as in Abu Dhabi and Dubai, have significant research collaborations also and exchange programs with training centers like Cleveland Clinic, Johns Hopkins, Cincinnati Children's, and Boston Children's, and many more.
It was a three-day event, had more than 36 sessions, was run flawlessly, and the last presentation on the final day was a highlight as it was a clinical presentation by the Chair of the conference, Dr. Miqdady, on the initial results that Jaguar reported today on the first two pediatric patients with intestinal failure proof-of-concept results. One had microvillus inclusion disease, short as MVID, and the other had short bowel syndrome with intestinal failure, short as SBS. Dr. Miqdady actually spoke as to why even a single patient result that had a TPN reduction was important and relevant to basically changing the trajectory of disease progression and also very relevant for drug development for new therapies for these intestinal failure patient populations. Right after his presentation, we recorded a fireside chat with Dr. Miqdady and his colleague, Dr. Christos Tzivinikos.
This presentation in the past weekend, which was presented to a full room, and many of the attendees subsequently wanted to know how they would have access to our drug, crofelemer, because the value of this product, as well as the value of the Elite Pediatric GI Congress in Abu Dhabi, was not lost on any of them. What I will do is, before I say much more, I will have Peter play the recording of the video of the fireside chat between me, Dr. Tzivinikos, and Dr. Miqdady. Just to give you background on Dr. Tzivinikos, Dr. Christos Tzivinikos is the founder of the pediatric gastrointestinal department at Al Jalila Children's Specialty Hospital in Dubai, and he's an adjunct clinical assistant professor at the Mohamad bin Rashid University of Medicine and Health in Dubai. He's also an investigator and key opinion leader in another trial of ours.
Those are the two people you will see: Dr. Mohamad Miqdady and Dr. Christos Tzivinikos. Peter, would you mind playing the video for us? Thank you.
Thank you. When I had first met you, Dr. Tzivinikos, you told me none of my patients will ever get an intestinal transplant. It's just these patients, many times, are candidates for intestinal transplant. Neither you nor Dr. Miqdady like that option. Can you just tell the audience?
I mean, there are certain criteria for multidisciplinary transplantation. Some of them see the publication was took up in 2020. If we go 20 years back, microvillus inclusion disease actually per se was the indication for intestinal transplant. This is not anymore the case. Of course, some.
Wonderful. Wonderful. I hope everyone can hear me again, and I hope you all enjoyed watching the fireside chat. I was there, and every time I watch it, I learn something new. I love the passion, the commitment, the promise for changing medical outcomes in such a meaningful way for intestinal failure patients and their families. As you heard, any reduction in TPN would have been important. 27% and 12.5% reduction in TPN is huge. I met Dr. Miqdady eight years ago when I was dropping my daughter at NYU Abu Dhabi and walked into this conference that was going on just around the corner. It's been nothing less than inspiring as we have spent the past eight years developing close working relationships with key opinion leaders and principal investigators around the world, regulatory interactions and protocols, endpoint definition and formulation development.
These patients are the true personification of a phrase which sometimes is overused, but the true personification of the phrase unmet medical need. Their HCPs and caregivers, the personification of nurture, of being there every day, every hour. These patients are quite fragile, and it's a family disease. Jaguar, through Napo Pharmaceuticals and Napo Therapeutics in Italy, is currently supporting two ongoing proof of concept investigator-initiated trials and conducting two placebo-controlled phase two trials for profoundness for MVID and adult short bowel syndrome intestinal failure patients in the United States, European Union, and the MENA regions. In addition to Dr. Miqdady's ongoing study, an investigator-initiated trial in adult patients with short bowel syndrome intestinal failure has been initiated at Cleveland Clinic.
We expect to have proof of concept results from these investigator-initiated trials throughout 2025, and the two placebo-controlled phase two trials are expected to conclude and have results in the first half of 2026. Pravin, as you can see, has established a fabulous collaborative and productive relationship with our KOLs and clinical investigators, as I think you can glean from the general genuine rapport in the fireside chat. Dr. Miqdady even got Pravin to put on a suit. Genuinely, there is a lot of patient passion there. Pravin, I'm going to ask you, please mention the names here of a few heroes in the community of intestinal failure, and then speak a bit more to Prime, which you mentioned at the end of the fireside chat.
Thank you, Lisa. Indeed, it's a pleasure to watch the video because we did it really live and extempore, so none of that was a cookie-cutter question. Everything was extempore. In addition to Dr. Miqdady and Dr. Tzivinikos, who are both pediatric gastroenterologists and are participants in our microvillus inclusion disease campaign, we have in Italy Dr. Antonella Diamanti, and she is a leading pediatric gastroenterologist at Bambino Gesù, which is a children's hospital in Vatican City. Of course, a national and international principal investigator for the MVID campaign that we have is here at Boston Children's. Her name is Dr. Lissette Jimenez, and she'll be joining me next week at an investigators' meeting. These are for microvillus inclusion disease campaigns that we're doing.
In addition, for adults, including SBS, Lisa mentioned Cleveland Clinic, which is the largest adult gastroenterology clinic for short bowel syndrome with intestinal failure in the world. The person we are working there is Dr. Lindsay Russell, and she is going to do a small proof of concept study in addition to a phase two study that we're going to initiate in European Union first in Italy and Germany. The Italy and Germany adult gastroenterologists who treat intestinal failure patients who are on home parenteral nutrition are the who's who list of intestinal failure. In Italy, we have sites in Naples, and we have Dr. Pacanisi there. We have Dr. Morabito who's still thinking about doing a pediatric study. We have Dr. Barberio in Padova, and we have Dr. Lamprecht in Rostock in Germany, Dr. von Websky in Essen.
We have three others in Germany, including Dr. Ulrich Pape, who's a world leader in this section. We have visited all of them, met them in person, and they are truly interested in the use of crofelemer because crofelemer, as Dr. Tzivinikos mentioned in the video, is ambivalent and agnostic to what other treatments the patients are on because it is actually an anti-secretory, and it works on the other side of reducing secretory diarrhea rather than changing any of the bowel adaptation, which is what all of the GLP-2 analogs need, as Dr. Miqdady and Dr. Tzivinikos were talking about. What Lisa had asked me also just now is to say, "Hey, let's talk about why PRIME, the priority medicines initiative, has been of interest to us." In fact, we did not know that because through our Italian subsidiary, Napo Therapeutics, we had preliminary communications with European Medicines Agency regulatory members that focus on PRIME.
PRIME stands for priority medicines, which is a program that provides enhanced interactions and early dialogue for the development of novel medicines targeting unmet medical needs. With these initial results in pediatric patients with MVID and SPS with intestinal failure, and given the ultra-rare nature of MVID, there are only 50-100 cases in the US that are known because incidence and prevalence are just about equal. These patients do not live very long. If they live longer, we want to help them have good quality of life, as Dr. Miqdady was talking about. Even with a small number of patients, we can actually get an expedited review and approval. The discussion we want to have with them is to explore pathways for expedited regulatory approval in Europe.
If crofelemer, it might qualify for PRIME in the European Union, and the PRIME designation in the European Union may actually help us to get Breakthrough Designation with the US FDA, which is also an expedited regulatory pathway. Now, just for those of you who may not know this, crofelemer is being evaluated in a novel liquid formulation for pediatric patients and adult patients with intestinal failure because their volumes of nutrition and electrolyte supplementing is very tightly controlled. We have made a highly concentrated lyophilized product, and it is called a type 3 NDA in the US, which means it's a new product. It is not the same tablets. It's different from Mytesi. That's why it doesn't have a name yet. This is very important for us to understand that this liquid formulation is literally given at 0.1 milliliters per kilogram body weight.
There is no big change in the volume for these patients who are receiving more than 50% of their nutrient and electrolyte requirements through parenteral nutrition. If we get the PRIME designation, which is a pediatric MVID indication, SBS obviously has a longer duration for the phase two to be finished, which has about 40,000 patients worldwide, to our estimate, at least between the EU and the United States. The market size for just SBS has been estimated in the US to be exceeding $5 billion based on what we know with tetra-glutide and other biosimilars for that. This is an opportunity to make a very meaningful impact for intestinal failure patients, as well as actually do something which is completely novel, has never been done before, which is to reduce the need for parenteral support.
One of my KOLs for adult SBS is the Chair Director of the Inflammatory Bowel Disease Center at Dartmouth. Dr. Siegel has taught me that the two most toxic treatments in medicine are total parenteral nutrition and chemotherapy. Anything we can do to reduce that is extremely meaningful in good outcomes. I will actually turn it back to Lisa now so that we can continue our discussion. Lisa, I hope that was helpful.
That was terrific. Thank you. That is how drug development is done. It is quite a process for those who are not in this industry to understand the long-term nature and the relationships and all the thought leaders and thought process that goes into it. Thank you, Pravin, for your leadership here on the scientific side. What we have discussed today is the first catalyst in one of our major two programs, our rare disease intestinal failure program. As you heard, we are currently supporting four clinical efforts in this rare disease intestinal failure program. The first proof of concept results are also catalysts to enhance potential business development plans for partnering and receiving non-dilutive dollars. We own the global rights to crofelemer.
There has been a strategic shift in the pharmaceutical industry towards rare and orphan indications, smaller populations, therefore potentially less expensive clinical development programs, opportunities for expedited regulatory pathways, as you've heard, such as PRIME and breakthrough designation, business models that support the relevant pricing necessary for these small populations and reimbursement for these diseases, which typically have high morbidity, high mortality, and high expense. For example, a typical patient on lifelong TPN costs the system approximately $500,000 a year to treat without complications, and they all end up with complications or double or more with the complications, as Dr. Tzivinikos mentioned. These initial results that you heard today catalyze the value proposition of crofelemer in treating and managing intestinal failure patients.
There are many precedents for major, highly valued rare disease collaborations in the pharmaceutical industry, tens to hundreds of millions of dollars, based on initial proof of concept data in rare diseases. We're actively pursuing in discussions for collaborations to cover our development costs and more until crofelemer reaches these patients through commercial success. Finally, while I have Pravin here, I will mention another near-term catalyst in our second major drug development program that Pravin is also leading. Crofelemer, in the same delayed-release pill formulation of Mytesi, which is our commercialized formulation, distinct from the rare disease program, as Pravin mentioned, a distinct formulation, a distinct product. Mytesi is approved for HIV-related diarrhea, and crofelemer in this formulation will be the subject of an FDA type C meeting in the second quarter of 2025.
Now, right now, regarding the potential pathway to bring the product to a supplemental new drug application for crofelemer, for prophylaxis of cancer therapy-related diarrhea in breast cancer patients on targeted therapy with or without cytotoxic chemotherapy. Results, which have been presented with statistical significance in a responder analysis based on a prospectively described subgroup from our phase three clinical trial called OnTarget. These results were just presented this past December 2024 at the acclaimed San Antonio Breast Cancer Symposium. I am sure Pravin would be pleased if there are any questions that should refer to that program as well. Now back to the intestinal failure results that we presented today. They are groundbreaking. They do humble us, and we are quite proud of what we've put together to allow crofelemer to show its benefit preliminarily in these patients.
I'm going to go look to questions now, which have come in written. Some, Pravin, are going to be for you. Here's an easy one. What is the current number of patients in the proof of concept trial? How many further will be enrolled? I think there is a related second question similarly about the phase two trials, number of patients in the phase two trials, and the timing for that. Basically, Pravin, the four trials, the two investigator-initiated, and the two placebo-controlled trials. If you could speak to the number of patients and timing, please.
Okay. Can you hear me okay?
Yeah, we can.
Okay. Good. All right. In this proof-of-concept study that Dr. Miqdady was discussing, which is an open-label study, he plans to enroll, as he mentioned, up to six patients with intestinal failure. He's got, to my knowledge, the third patient enrolled out of the six just recently, and that's also a short bowel syndrome patient. He's got a plethora of patients in the region, and he's expecting maybe one or two more MVID patients and maybe two to four SBS patients in this. They're all pediatric. His study is only for the age groups of 1 to 18.
The total number of patients in the other investigator study that Lisa would have mentioned, which is for the adult SBS intestinal failure patients in Cleveland Clinic, also intends to have six adult patients, and that's about to start this quarter, and that'll go on for a while. For the phase two studies that are Napo-sponsored, remember, these studies are investigator-sponsored, so Napo is just providing support through formulation and drug, doesn't have any oversight or accountability for data management. For the two phase two studies that are under our sponsorship, the first one is pediatric microvillus inclusion disease study. This study was initiated first in the United States under the IND with the FDA, with Boston Children's having two patients. Subsequently, we got interest from the European Union, from Italy, in particular, from Dr. Diamanti, which is a center of excellence for intestinal failure patients in Europe.
We also got interest from Dr. Tzivinikos in the UAE. We had to basically make it into a global trial from a US trial, keep the same protocol, and then basically get regulatory approvals or clearances, sorry, from all three geographies. Now the MVID trial has become a global trial. We expect up to eight patients with MVID, pediatric, obviously, to be enrolled and randomized and treated in our Napo-sponsored study, which may seem like a small number to some of you, but with the prevalence of being 50-100 cases, eight patients represents almost 5% of the living population of these pediatric children. The second phase two study that Lisa mentioned is an adult SBS study, which we are only doing under the auspices of EMA, European Medicines Agency, and we are doing it in two countries, Italy and Germany.
We have eight centers there, and we intend to enroll up to 18 patients. It is randomized, double-blind, placebo-controlled, and six months of treatment. Again, that phase two study, we expect some results in the first half of 2026, and MVID, we will have some of that data in the first half of 2026. I hope I answered the question, Lisa.
Well done, Pravin. I'm going to continue with the questions for you before I move on to the questions that I should answer. Another question has come in is, how confident is the team that Prime designation will be granted, leading to early access and potentially expedited review for the patients in the 27 EU countries?
We are cautiously optimistic that we will have a good dialogue with the European Medicines Agency for the Prime designation. Obviously, this is not something that we can predict because it's a review issue. We intend to have a submission to them later in about four weeks from now, and we'll hear from them in mid-July if they want to invoke a meeting. The fact that they invited us to speak with us speaks to the burden of the unmet need in this pediatric patient population and the rarity and the desire to give some treatments other than TPN, which is all that these MVID patients get. I am cautiously optimistic, but I cannot put a % on that.
Terrific. Thank you. Here's a question. It's probably more sort of understanding how clinical trials work on a global basis. Are the treatments that are happening in Germany and Italy during the clinical trials, are they reimbursed by these countries? How does that happen? How do these patients in European countries participate in our clinical trial?
Are you asking if the TPN is considered a standard of care? Is that the question?
No. I think the question is asking about, does crofelemer get reimbursed during the clinical trial? It is sort of understanding how the clinical trial process works in the pharmaceutical industry sort of gets to why it is so expensive to develop new drugs.
I see. I see. Okay. Sorry. In the Napo-sponsored studies, we are paying the investigators and the sites a per-patient cost because TPN is standard of care, so that is reimbursed by their government, but we pay for all of the testing and everything else. It is not something that is normally reimbursed by insurance in their country. If we get good results and we are the subject of approval by EMA, we still have to negotiate with the HTAs, the Health Technology Assessment Groups, which are the payers in Europe, one by one. We have a market access lead person for our Italian subsidiary, Napo Therapeutics. She's already engaging with them along with our medical affairs head over there with some of these HTAs.
Our anticipation is that upon approval in Europe, the first country that is likely to allow us within six months after approval to launch and get reimbursed will be Germany because the Germans are the fastest. After that, we expect Italy to come second and France to come third. I think that's the way it's going to work. At this point in time, under clinical trials, TPN is standard of care, so we don't have to pay for that. Other than that, any other testing and the use of any of our materials and home nurse or anything else is all covered by Napo in our Napo-sponsored trial.
Terrific. Don't go away, Pravin. There is another question coming up for you, but there is a question that I think I'll answer, which is talking about, where are our business here? What is the market size? What are we talking about? Rare diseases are really remarkable, the incentives that are in place, the reimbursement opportunities for small populations, but as you can hear, the activities, the time, the expense involved in coming up with breakthrough medicines and breakthrough interventions. Let's talk about MVID first. Actually, let's talk about SBS. There is a product that's approved and approach that you heard Dr. Suzenago talk about, and Pravin mentioned, a GLP-2, which is essentially a growth hormone.
It does have some utility in a small number of patients, literally single-digit number of patients with SBS, not standard of care, lots of limitations in how a growth hormone can be used. For example, you can't use it in an abnormal hyperproliferative disease situation. You can't use it in the cancer patient. Nevertheless, the product is reimbursed in the United States at about $500,000 a year and a couple of hundred thousand dollars a year in Europe. If you use that as a benchmark for annual reimbursement with MVID, which is ultra-rare, you're talking about the market opportunity of a couple of hundred million dollars, about 200 patients, $500,000 a year total market opportunity. That same formulation, that same product, then in the short bowel syndrome market, which is about 40,000 patients around the world, that's where you're getting into blockbuster territory.
As I mentioned, third-party analysts have put that market. We've seen estimates from about $5 billion up to about $12 billion a year, which was not even taking into account the opportunity of something like crofelemer with the safety, the broad use opportunity without limitations, for example, in cancer patients or patients after surgery having to have bowel adaptation. This is quite impactful for the financial stakeholders in the company. Of course, we've talked what's driving force here is the need for patients as well. The strategy here, the clinical results are playing into a very important business strategy because MVID has nothing. MVID patients, their gut is fully intact. It's not short. It's just not functioning. There's no growth hormone approach. Again, as you heard from Dr. Tzivinikos, there's nothing. There's nothing in development. There's nothing for these patients.
Nothing was ever expected to have an opportunity to reduce TPN in these patients. That's the driving force behind PRIME, which is potential accelerated approval, potentially gets us breakthrough designation, gets that product out there as quickly as possible, gets the reimbursement challenges and all that done, and it's the same product, the same formulation that will go into the short bowel syndrome market as well, which is, I think, a good lead-in to what I believe is the last question here. Pravin, I'm going to send this one to you. The question is, why do you think in these two patients, there was one patient with 25% reduction in TPN and another with 12.5%?
Yeah. It's a good question. I see the question in the chat box here. I think what this is guesswork on my part. There are two possibilities that I can speculate upon. In MVID patients, nobody had quite understood the possibility that if we were able to reduce the amount of secretion, which we had done in an enteroid organoid model at Boston Children's and published that, that that would have an indirect benefit in oral absorption, oral intake. An indirect benefit of that is what Dr. Miqdady spoke about in the fireside chat with us, which is that while the mom was complaining that the child was peeing more, that is actually a direct effect of more oral absorption.
I think in the MVID person, over time, we see sort of a gradual reduction in stool volume output and a little bit of a bounce, but more urine output over time, and we could see that. In the SBS child, we do not know the gastro surgeons when they cut the bowel. They don't record the remnant bowel's length. These patients, it is likely, let's say this child is a type 1 patient, that's my guess, which means that the patient does not have a colon in continuity. The reason for saying that is that the volume of TPN that this child needed every day, and I saw that number from Dr. Miqdady's presentation, on a weekly basis, the child was getting more than 18, 20 liters, 21 liters in TPN.
I think because they don't have much capacity to absorb, that might take a little bit longer. It is not that the reduction is different, as it may be more a temporal effect. Maybe it will take a little bit longer. This was only Dr. Miqdady was doing only a 12-week study in which he dose escalated. I think in MVID, we saw it more because the child had a full-length cut, and maybe that helped with the absorption, and then the benefit of crofelemer in its anti-secretory mechanism actually played a good role in that. That's my guess. It's not precise, but I think over time, all our other trials are 24 weeks or longer, and we are continuing Dr.
Miqdady's exploratory open-label study, and if the child continues to benefit, we'll get long-term data both in SBS as well as in MVID. We will be able to answer that question more accurately in the coming weeks. We just have the first 12 weeks of data right now. I hope that answers the question, Lisa, on that one.
Yeah. I think that's well put. In summary, one word, yes, right? That's how much so far. You know what, Pravin? Another thing that I think would be important to actually highlight here is the MVID patient, according to the protocol, after 12 weeks, which is you just described, is limited. The protocol called for the patient to go off crofelemer for two months. Why don't you talk about what happened in that situation?
Okay. Okay. I see that another question has come in on MVID on the patient's appetite. Sorry, I'll try to take that as well later. Yeah. One of the things that we had done is these are called N-of-1 trials regulatorily because the FDA had put out guidances about 10, 12 years ago on what is called substantial evidence of effectiveness. In that, they had, for rare diseases and orphan diseases, created a paradigm wherein you show N-of-1 trials where you basically treat a patient and then withhold treatment, and then if the condition worsens, you restart and you show if there is benefit again. Pretty much is exactly what we did in the open-label study that Dr. Miqdady described, where the patients were supposed to get the drug for 12 weeks and to the maximum dose that Dr. Miqdady determined based on safety.
At the end of 12 weeks, TPN continues, but crofelemer as an investigational product is stopped. In the protocol that Dr. Miqdady had submitted to the authorities, he had asked for a 30-day withholding period, but he amended that with his IRB to say if the symptoms start to worsen, he would like to reinitiate crofelemer treatment in addition to the TPN and other therapies. Good thing he did that because after stopping in the MVID patient, eight days later, the mother called Dr. Miqdady and said, "My child's stool volume output is going up. Urine output is going down. Please, please, can we reinitiate the treatment with crofelemer?" He restarted eight days later itself.
This was something that another regulatory clinician had warned me about and said that don't have and don't wait for the entire protocol specified 30 days because sometimes the worsening does more harm. Reinitiate at the first signs that in the clinical judgment are necessary, which is what Dr. Miqdady said. Now that's also evidence of the N-of-1 trial that the patient was truly benefiting from the crofelemer therapy because that was the only other additional variable that was taken away. I think Dr. Miqdady was quite pleased to be able to reinitiate after eight days rather than having to wait for 30 days.
Terrific.
Yeah. There's another question related to the oral intake, Lisa, in there.
Go ahead. Take that question.
Yeah. The question is, in the proof of concept study, did you observe any changes in patients' appetite or oral intake in the 12-month oral treatment period? I can't answer that question because that's Dr. Miqdady's patient. It's an investigator-sponsored. I can indirectly answer that question that the urine output going up and the TPN duration going down. You heard from the video from Dr. Tzivinikos and Dr. Miqdady that this child was getting 16 hours of parenteral support every day and was down to 11 hours by the end of the study, and his urine output was going up. That's indirect evidence that the oral intake did go up.
This has been sort of postulated, at least in the U.S., with my colleagues at Boston Children's, that MVID patients may benefit, and they might be able to improve their oral intake if we are able to basically reduce some of these needs for parenteral support. I think it's our first evidence. It's early days, but I'm cautiously optimistic that the oral intake is improving. I hope that answers that question for your MVID person.
Yeah. I love this dialogue. We could go on forever, but just a comment about that. One of the things you learn as you start to interact in this community is that it's very important early on, even though the patients can't absorb anything from what they're taking, any nutrition orally, if you don't get them in the habit of taking things orally and eating, they will never eat. They just have no interest, no desire. It's almost garbagey in their mouth to have the meat. That's very, it's something that they've learned, and they try to start as soon as a child can put something in their mouth, even though it doesn't get absorbed. There is a question here about potential cognitive benefits. This was very interesting.
When I was sitting in the conference in Abu Dhabi, unrelated to this presentation, which was the last presentation, there were many presentations on cognitive disorders and parenteral nutrition and a whole bunch of disorders that have intestinal failure well beyond the MVID and the SBS that we're talking about, where we expect and hope crofelemer will eventually be able to have impacts as well. The number one thing that the investigator I actually happened to be sitting next to him, his name was Dr. Cole from Johns Hopkins, a new facility that they opened in Gainesville, again, another collaboration with the renowned centers here in the Mideast. The number one issue for him was the cognitive impact and deficits in these patients because they're young. They're pediatric patients. Often, it's from day one when they're born, and they're not getting adequate nutrition on a regular basis.
These patients are fragile. TPN is not an easy thing to monitor. These patients are monitored, for example, by Dr. Miqdady every single week. There were some patients in the Children's Hospital in Rome who've never even left the hospital. With that variability, there's variability in their nutrition and obviously in their cognitive development. That's what he finds as the number one impact to these patients. Okay. There are many. Are there some more elsewhere? Oh, here's a question, a business question. With these rare patient populations, do you plan on marketing the product your own or partner regionally? What is your strategy here? Okay. Right now, commercially, we do have a commercial product for Jaguar, and that is Mytesi for HIV-related diarrhea. We do commercialize ourselves in the United States. The strategy, we have global unencumbered rights to our technology, and we have some partnerships.
For example, we have a partnership in place in Turkey and some Eastern European countries. Our intention is to bring on partners and collaborations, as I mentioned, with this proof of concept data. By the way, separately in our cancer program, as we get clarity from our FDA meeting, to bring in non-dilutive dollars from these transformative catalysts that will help fund the company and these programs till these products come through for commercial access. In the United States, we would like to continue to commercialize ourself. Perhaps in some of the bigger areas like cancer, we would have co-promotion, so we have an opportunity to bring additional feet on the ground. For rare diseases, these are really very focused centers of excellence, the leaders that we've already identified that are working in our clinical trials, patient advocacy organizations. Patient advocacy is near and dear to our hearts.
The patient voice is so important. For example, we've done a very strong role on the cancer side with Make Cancer Less Shitty, and the patient advocacy and opportunities for the entire community in rare diseases is very strong. That, in general, is the plan. Rights outside the United States, bring in non-dilutive dollars based on these catalysts and these very near-term happening right now transformative events and hang on to the U.S. rights or, at the very least, co-promotion in the United States.
Okay. Lisa, there are two more clinical questions. Let me just handle them so that we can answer. I know we are running out of time here. The first one is whether we are going to do every MVID patient mutation one at a time. The short answer is no because genetic mutations do not dictate the phenotype. The diagnosis of MVID is basically done with clinical symptoms and genetic mutations. All four of them are included. We already have the enteroid study we had done with two different mutations. They present with the same phenotype regardless of the underlying genetic mutation, and genetic testing results come four to six months afterwards. Our KOLs have told us not to worry about what the underlying mutation is. That's the answer. The other question is about cognitive benefits that we expect to augment your answer, Lisa, from Dr. Cole.
This is something that every pediatric gastroenterologist always wonders, that as tightly as they regulate the TPN, growth is stunted and neurodevelopment is slow. If we can improve oral absorption, the question is basically reversed. Yeah, I'll basically say yeah. Our hypothesis is that the more the patients can absorb orally, these MVID patients, their neuro and cognitive development will be better. That is the working hypothesis. We will see that. We are not including that as a trial endpoint, but we are definitely going to assess that over time. Those are the two questions which are clinical. Lisa, back to you. Sorry.
Okay. I think we are done here with the questions, and we've run a little over. I want to thank everybody for participating. I'll leave you. I've so enjoyed this session. I enjoyed the dialogue. Let me just leave you with a few words here, which is catalyst, transformative, groundbreaking, hope for these patients and for all the stakeholders involved in this endeavor of drug development. Thank you, Pravin, for your leadership and your participation. Thank you all for listening this morning, and more to come. Here we go.
Thank you all. Good.