Good afternoon. Thanks for the introduction. So, good afternoon, everyone. I'm delighted to be here and tell you a little bit about Quince Therapeutics. We are a publicly traded company, so there will be forward-looking statements during this presentation, and this is our disclosure. So, let me tell you a little bit about some of the highlights of our company, and I'll go into all of this material in more detail through the presentation. The first thing you should know is that we have currently an active phase III clinical pivotal program for our lead drug, EryDex, which is underway in a pediatric rare disease. We are targeting a disease, ataxia-telangiectasia, or A-T for short. And unfortunately for this patient population, there are no currently approved treatments on the market. We have done a market assessment and believe that this is a $1 billion commercial opportunity.
I'm going to share with you some data from a previous phase III study, which shows some very encouraging trial results, and it's those results that really led us to the acquisition of this company. We also have tried to mitigate the risk associated with our phase III study. We have a Special Protocol Assessment, or a SPA agreement, in place with the FDA. We also have Fast Track designation, and we also have Orphan Drug Designation. So, those allow us to move our program through the FDA more quickly and actually save us some money as well. The technology that I'm going to tell you about, although we are focused currently on A-T, it has applications in a number of other therapeutic areas.
We've done a pretty extensive review of various therapeutic areas and have decided that DMD, or Duchenne's muscular dystrophy, is an ideal next indication for EryDex. The other good news for our company is that we do have enough cash currently to get to our phase III top-line data, which we're expecting Q4 of next year. And so, we have cash that takes us into early 2026. So, let me tell you a little bit about A-T. I really did not know anything about this disease population until I joined Quince a year ago. This is an inherited rare neurodegenerative and immunodeficiency disorder. Kids are usually diagnosed at around age two, so they present very healthy. They start to have difficulty in motor functions.
Typically, they see a pediatric neurologist, and at that time, they'll have a genetic test that will confirm that, in fact, they have the mutation of the ATM gene. Unfortunately, these kids are often wheelchair-bound by the time they're early teens, and their life expectancy currently is between 25 and 30 years of age. It has a very similar epidemiology to Friedreich's ataxia, and that's relevant, which I'll share with you in a moment, for what we're trying to accomplish at Quince. So, Quince acquired a company called EryDel in Italy. As it turns out, Mirandola, which is in northern Italy, is one region in the world where there's a lot of device technology that's been developed over the years. And so, this company, EryDel, spent 15 years and over $100 million developing the machine that you see here. And we call this the AIDE technology, Autologous Intracellular Drug Encapsulation.
Essentially, what they have figured out how to do is we take a patient's red blood cells, about 50 ml, which is like a double espresso amount of blood. We put that blood into this fully automated machine, and through a series of washings, both hypertonic and hypotonic solutions, we open up the red blood cell, then we infuse the drug, and in this case, it's dexamethasone sodium phosphate, or DSP. It's a steroid, into the patient's own red blood cell. Then we close that red blood cell, and then that blood, with the drug encapsulated, is then infused back into the patient. This entire process takes about an hour and a half, and it's a once-a-month treatment.
And so, what we're able to do is deliver a steroid, and I'm going to talk a little bit about steroid therapy and why it's so important for this patient population, but we're able to do it in an efficacious and safe way for the patient. We have very good IP protection running out to 2034 and 2035, and also the device itself is CE marked in Europe. So, as I said, our lead asset is a phase III encapsulation technology, and we call this AIDE. I think the first thing you should know is that what we've really done is found a way to deliver steroids in a safe and effective manner to patient populations. And because it's delivered through the red blood cell, it has biodistribution that is unique and different from other conventional therapies. So, it has unique biodistribution.
And if you look at the PK curve, one of the things that you need to do is you have to have saturation of the receptors early on, but then you need to have a steady state of drug release. And so, through our technology, what we have found is that when we give the initial infusion, give the initial blood back to the patient, they reach the Cmax that they need for saturation of the receptors, and then they see a very gradual release of drug over the 30-day period. And so, it is a once-a-month treatment, but it really has fundamentally altered the way that steroids can be administered in a chronic manner. For those of you that know very much about steroids, they are widely used, but unfortunately, they have a lot of really negative side effects.
Those include growth suppression, delayed puberty, immunosuppression, hyperglycemia, excessive weight gain, hysterium, and acne. We have actually been using this technology for many years, and in fact, over 270 patients have been treated at least once with our technology, and we have not seen these side effects. So, one of the things that we've discovered is a way to effectively administer steroids to a patient population without all of the negative side effects that are typically seen in conventional therapy. In terms of the commercial opportunity, this is a rare disease. The prevalence of A-T is estimated at around 10,000 patients in the U.S. and in the U.K. and the four major countries within Europe, and we use IQVIA data for this.
We would be the very first to market this therapy, and because we've been granted Orphan Designation and Fast Track designation, we believe that we'll be able to really move very quickly. In terms of pricing, so I talked a little bit about Friedreich's ataxia. So, there's a drug, Skyclarys, which a company, Reata, developed and Biogen acquired a year ago, and they're priced at around $370,000 per year for treatment. It's a very good comparator for us in terms of the patient population, the size of the market, and so it allows us to kind of benchmark our own commercial opportunity. We do have a very scalable manufacturing structure, and our cost of goods is less than 1%.
Obviously, physicians who have been working with this patient population for many, many years have always been challenged by the fact that they don't really have a therapy to offer the patient in terms of the specific disease. What they do is they essentially treat the various conditions that the patient is presented with, and so patients go throughout a series of stages of progression, obviously downward throughout their lifespan, but they don't really have something to really treat this disease. And so, when we talk to pediatric neurologists, one quote we received is, "I would use this in as many ambulatory patients as possible. The disease has a devastating course. I would look forward to using this to try to slow the progression." So, that's really what we're striving to do, is to give back quality of life and slow the progression of this disease in patients.
This is one of the most important slides, and I think I shared with you that we acquired this company, EryDel, in Italy, and they ran the largest clinical study, phase III clinical study, both in Europe and the U.S. on A-T patients. And unfortunately, they missed statistical significance by 0.07, and for those of you that are familiar with the FDA, you need to have a 0.05 or lower typically for approval. But in any clinical design, you look at subpopulations of data, and they pre-specified some of this analysis to try to understand exactly what occurred throughout the trial. And this was over 170 patients. They had three arms of the study, so they had a high dose, a low dose, and placebo. And what they discovered was that the high dose was more efficacious compared to the low dose.
The other thing that they discovered is that the kids in the younger age population, 6 to 9, responded to therapy quite well. And the tool that the FDA uses, it's an ICARS scale, so you have a rater that actually examines various functionality of the patient throughout the clinical trial. The FDA actually is looking for something called a rescored Modified ICARS, which is that box that you see on the right. And what they want to really focus in on is motor function within this patient population and functionality. And when you look at the data from the previous study that EryDel ran on EryDex, which is the technology that we're working on, you will see that they reached statistical significance by 0.009, and this was with 33 patients which were in the 6 to 9-year-old range.
This data for us is very encouraging because in our clinical study that we are undertaking now, we have the exact same technology, the same device, the same dosage, and the same patient population being the six- to nine-year-olds. So, that is our current clinical study that is underway, but it's not often that you have the benefit of a previous clinical study to look at that data and give you some guidance on clinical design and also predictions of outcome. So, this is our current phase III study. So, as I said, it's underway, both sites in the U.S. and Europe. It's a pivotal study conducted under a SPA with the FDA. It's a very important milestone for us to have an agreement with the FDA around our protocol and clarity about what would be required for success. So, that is in place.
It is a randomized, double-blind, placebo-controlled study. The study runs for six months in duration. So, a patient comes in at the beginning of the study, they'll be assessed, they get an initial dose, and then they'll have treatment for 6x , so six months of observation and analysis, and then at the end of that treatment, every patient will be given the opportunity to receive treatment because, as you can appreciate, 50% are on placebo and 50% are on treatment. In our particular study, we have 86 patients, so 43 in treatment, 43 in placebo, and then we have an additional 20 patients who are over the age of 10.
They're not part of the clinical assessment, so in terms of determining what the P value is, the additional 20 patients over the age of 10 are not in that subgroup, but it is important for label expansion to understand and safety to understand how these older patients fare with it. We will be putting out guidance. Our 10-Q is coming out in mid-November, so currently, I can only give you, tell you about how many patients were enrolled. So, in August, we had seven patients enrolled in the study. Obviously, there are many more now, and you'll get those specifics in the coming weeks. So, as I shared with you, this platform technology we believe has broad applications in many, many other areas. And so, we began that process of really looking at some of the other therapeutic areas and determining what else we might consider.
When we looked at DMD, it's a patient population where steroids are widely used despite their side effects, and so, one of the challenges that physicians who are treating these patients have is trying to figure out how to get the benefit of the drug but also managing the side effects, so sometimes they'll go on a drug holiday or they'll dose them and then stop dosing them, and we know from our research to date that we are able to give a very steady state administration of a steroid to a patient over a period of time, so we have selected DMD as our next indication. We will look to do a proof of concept study. We do not have the cash for that today, so we can begin to design that study. We are looking for grant opportunities, and unfortunately, there are some out and available.
If we were to do any additional raises in the coming months or in the next year, certainly one of the things and goals would be to get a DMD program underway and get to a proof of concept study. We really see this as an important next step for Quince in terms of developing the broader applications of this exciting technology that we acquired from EryDel. When you look at the rare disease category and look at the interest in biotech and strategics, certainly there's a great deal of interest in rare disease. I think I shared with you that Friedreich's ataxia, the drug that Reata developed, is very similar in epidemiology to what we're trying to do here at Quince, and they had a very attractive exit with Biogen in July of 2023 at over $7 billion.
So, the other thing as we think about this opportunity, we really want to understand what is the ability to partner or find a corporate partner that would be interested in what we're doing. And I think based on our assessment to date, we think that rare disease category is still very attractive within the pharma community. We have a great team. So, I serve as president of the company. Dirk Thye, who's an MD, Stanford residency. Dirk is West Coast based. He and I met when we were together at Forest Laboratories here in New York. So, I was with Forest Laboratories until its acquisition in 2014. And so, we've worked on a number of opportunities together, and we really have an extensive team of seasoned professionals who have worked in a number of therapeutic categories with very successful exits throughout their career. So, it's a great team.
I will say we are East Coast and West Coast based primarily, and then we have the team in Italy. So, when we acquired EryDel a year ago, Quince had $100 million in cash, and they had a public shell, but the lead asset they were pursuing at the time had failed, so they were looking for something new. And so, they did an exhaustive search, and Brendan and Dirk really led that search with a number of other people. And after a very extensive review of more than 100 opportunities, they found this company in Italy, EryDel, who had this amazing technology, EryDex, where they had had the failed phase III study, and they were looking for an opportunity to continue to the next phase III study. So, we came in and acquired that company just over a year ago. In fact, it was a year last week.
That's a little bit of the story of Quince and how it came to pass. To tell you a little bit about some of our corporate milestones, what we've accomplished in 2024 and what we're looking to accomplish in 2025, we obviously enrolled our first patient this year. We selected DMD as our second indication for EryDex, and we'll begin doing some study designs around that. We have looked at other potential indications for this, and there are other R&D activities underway. We did receive Fast Track designation for A-T from the FDA. We have initiated a phase III open label extension study. The data, what's interesting, the data from the ATTEST study, that's the previous phase III study that EryDel conducted, it had never been published. We were delighted to be able to publish this just in September in Lancet Neurology.
So, now we have all the phase III data published and available. In 2025, our big milestones will be completing the enrollment of this phase III study, so last patient, last visit. We'll be announcing that in 2025. And then we look to have top-line results of this phase III clinical program in Q4 of 2025. And then, obviously, the assumption that we have positive data, we'll look to prepare the NDA and MAA submissions into 2026 and then begin initiating the DMD clinical study using the EryDex system. And then, in conjunction with all of this, we continue to talk to various corporate partners and explore potential out-licensing opportunities along the way. So, this is actually my last slide, and these are what I think are some of the key investment takeaways. The first is that we have a very compelling clinical proposition.
We've got a pivotal phase III study underway with top-line results expected in Q4 of 2025. I see this phase III study as risk mitigated in several ways. First, through the various agreements we have, the SPA agreement with the FDA, the Fast Track Designation, as well as the Orphan Designation, and on top of that, we have these encouraging phase III results from a previous clinical study that we were able to learn from in designing our clinical study, so the previous clinical study had both young kids and older kids, whereas in our study, we are looking to focus it on the 6- to 9-year-old kids where we saw efficacy in that previous study. In terms of our commercial opportunity, we've done an assessment determining that it is a $1 billion opportunity, and we have selected DMD as a second indication.
There are many more that could also be viable for this technology, but we're being mindful of the fact that we need to find a second opportunity. Then we're positioned for success. We have the cash to complete this study, and we really have a leadership team that has the experience necessary, I think, to achieve the results that we're hoping to achieve. So, with that, I'll take questions. Yes, please.
Can we talk a little bit about sort of the the patent coverage that you're going to write the patent coverage?
The patent coverage is what you're asking about?
Yeah.
Is it about device? Yeah. So, we have it. Yeah. So, actually, I'm a patent attorney by training, so I was the chief intellectual property counsel at Forest Laboratories. I have a PhD and a law degree, so I'm happy to talk about this topic. So, we have IP on the device itself, and then we also have IP on the method of using the device and then method of treating patients using this device. So, we've really looked at all the different ways, and we are still, one of the things we're working on now is this convenience kit. It's some of the materials that are necessary. And so, we're actually talking to some IP attorneys about figuring out a way to even capture some IP around some of the ancillary things as well. So, but always a great question, IP. Thanks. Any more questions? Please.
Yeah. I was curious, if I understood you correctly, the idea is to put your device in centers of excellence.
Yes.
And that way, patients would come to there.
Yeah.
As you look to expand across different indications and things like that, do you believe that you'll be able to put the device anywhere you need? It has to be manufactured.
Yeah. Yeah. So, it's a great question. Kind of like, what is the commercial plan around the device itself, and how are we going to implement that? So, I also work with the chief commercial officer, and so we're really developing this strategy. I think it's multi-pronged. So, certainly, centers of excellence is where you start. The other is infusion centers, and especially if you can contract with companies that have large infusion centers within regions, because one of the things we're trying to do is simplify the strategy by identifying partners that have broad outreach.
And then the third thing that we're looking at long-term is whether or not you would ever use something like a van or some kind of mobile in more of rural areas and actually have some methodology of getting the device closer to patients, etc. So, but I think that's a further opportunity down the road. But yeah, great question. Any other questions? Thank you very much for your time. I appreciate it.