Hi, everyone. I'm Brendan Hannah, the COO and CBO of Quince Therapeutics. And I'll provide a short introduction of the company today. So we just started a pivotal phase III study with our lead drug, EryDex, in a rare pediatric neurodegenerative disease called ataxia telangiectasia. There's no currently approved drugs for this disease, and it's a $1 billion+ commercial opportunity. There's prior phase III data that had good clinical signals and were encouraged by this data, and we've designed a new study around this. So our ongoing study is risk-mitigated. It's under a special protocol assessment with the FDA, and we also have Fast Track and orphan drug designations for it. We selected Duchenne muscular dystrophy. It's our second indication for EryDex, and we'll have top-line data in Q4 of next year, and then we'll have cash all the way through that into 2026.
The technology that Quince is focused around has had more than $100 million invested into it, and we call it the AIDE technology, the autologous intracellular drug encapsulation technology. This is a machine about the size of a desktop computer that will sit in an infusion center or in a cart and is used at the point of care. This is one-touch, fully automated, and it allows us to encapsulate different drugs inside the red blood cells of a patient. We have the ability to encapsulate a wide range of drugs from small molecules all the way up to large molecules, and we have IP exclusivity until 2034 in the U.S., or 2034 globally and 2035 in the U.S., and it's also CE marked in Europe. The lead product from this technology is called EryDex.
This is dexamethasone, the potent anti-inflammatory steroid encapsulated inside a patient's own red blood cells. And the main issue with dexamethasone is that there are significant toxicities associated with it. And by encapsulating dexamethasone inside patients' own red blood cells, we're able to alter the biodistribution and the pharmacokinetics of the drug so that way we can provide constant concentrations that are efficacious but without triggering these toxicity thresholds and causing adverse events for patients. So we're able to provide chronic steroid therapy but without the safety issues. And there are significant safety issues associated with conventional daily steroid therapy, especially in adolescent populations. So you have growth suppression, delayed puberty, immunosuppression, diabetes, weight gain, osteoporosis, hirsutism, acne. Whereas with EryDex, we have several hundred patient years of data and over 6,000 doses administered at this point once monthly.
We haven't seen these steroid-related adverse events, so we have a very clean body of data to present to the FDA when we file for approval, and the disease we're going into is called ataxia telangiectasia. It's a rare neurodegenerative and immunodeficiency disease caused by mutations in the ATM gene, so patients are usually diagnosed somewhere between two to five years old, then they progress rapidly until they're in a wheelchair by 10- 12, and then usually die from either pulmonary infections or cancer by 25- 30. There's nothing currently approved for A-T and no other late-stage drugs in development, and then the epidemiology is very similar to Friedreich's ataxia, so the commercial opportunity is very interesting to us for A-T for EryDex. We believe it's over a $1 billion market in this indication alone.
There's about 5,000 patients diagnosed in the U.S. and 5,000 in the Big Five EU. We have the first-to-market potential here without any other late-stage competition. And then we have orphan drug designation from the FDA and EMA, and then Fast Track designation from the FDA. A drug was approved last year by Reata that's called Skyclarys for Friedreich's ataxia, and they priced it about $400,000 per year. So this provides a good correlate of what you can get for a rare disease without any other approved products. And then the cost of goods for this is very minimal. We'll provide a procedure pack for each once-monthly infusion for everything that they need, including the drug, the tubing, the process solutions, and that is 1% of what we expect to be charging for this drug. And we've completed both payer and physician research on EryDex in A-T.
I would say the key takeaway with pediatric neurologists, which are usually the treating physicians, is that there is a huge unmet need here. Patients have nothing, and really anything that provides a benefit for efficacy that is non-toxic will be used in the vast majority of patients. We expect pretty significant patient share regardless of ambulation status once this drug is approved. Then there was a prior phase III study completed in A-T with this drug. It narrowly missed statistical significance. The P-value is 0.07, and that's likely because they included all ages in this study, whereas there's rapid neurological progression between six to nine years old, and then usually it plateaus after that. In the pre-specified subgroup of just six to nine-year-olds, they had statistical significance across really all the key different endpoints here.
That's what we're going to be focusing our current study on that's already enrolling patients. So this is under a special protocol assessment with the FDA that allows us to submit an NDA on a single pivotal study and apply for approval based upon this NEAT study that we're running right now. It's standard, randomized, double-blind, placebo-controlled study, six infusions once a month. And we've enrolled the first patient in June, and we'll be reading out top-line results in Q4 of next year. So it's 86 patients aged six to nine. The FDA asked us to include another 20 patients that are 10 or older so that way our label is broader than just six to nine-year-olds. And then all patients have the ability to roll into the open-label extension study.
The primary efficacy endpoint that's at six months of dosing is called the responder score to Modified ICARS scale. This is a portion of the ataxia scale, and this is primarily focused on posture and gait in these patients. Given the profile of EryDex of being a steroid without the steroid-related safety issues, there's a very broad range of indications we can go into here. We've narrowed the list down to about 12 different indications that are severe rare diseases in which we can utilize the same pricing as we would for A-T, but also not trigger IRA price negotiations. The second indication that we've selected is DMD, Duchenne muscular dystrophy.
Really all the physicians we've talked to have said that patients are on gene therapies, exon-skipping therapies, but they're still all going to be on steroids, and the vast majority have steroid-related toxicity issues. We plan to start a trial next year, likely in steroid-intolerant patients, which is the majority of the patients. We have the ability to go into a broad range of other diseases after that, including neuromuscular diseases, autoimmune diseases, and rheumatology, but all focused on severe rare diseases. Then there's been quite a few billion-dollar-plus acquisitions in the rare disease space over the past couple of years, usually around phase III data or all the way through approval. We think the closest correlate is the acquisition of Reata by Biogen last year for $7.3 billion. Reata got a drug approved for Friedreich's ataxia, which has very similar epidemiology to A-T.
Pretty shortly after approval, Biogen acquired them strictly for that drug. So it just goes to show what values can be attained here with a similar patient population to A-T and a similar unmet need. We have a very experienced leadership team. Dirk Thye is our CEO and our CMO. He's been CEO 10 different times with seven successful exits, gotten several drugs approved, taken them all the way from animal studies through approval. And then we also have several other experts in each of their own domains that have gotten dozens of drugs across the line, primarily in rare disease. So we have the leadership team to take us all the way to approval and launch right now. And we've checked off quite a few different milestones for 2024. We've enrolled the first patient in our phase III study. We selected DMD as our second indication.
We've provided a numerical ranking of other indications after A-T and DMD that we can go into. We've received Fast Track from the FDA for A-T. We've initiated the open-label extension study, and then we got the prior study published in Lancet Neurology last month. So for 2025, we'll be completing the NEAT study. We'll read out top-line results in Q4, and then assuming those are positive, we'll start preparing for NDA and MAA submissions. We plan to also initiate a clinical study in DMD, and then we can look into outlicensing ex-U.S. or territorial rights for EryDex that could provide funding all the way through approval. So really to wrap everything up, there's a very compelling clinical proposition here. We have a phase III study underway right now in a severe rare disease, and we expect top-line results in Q4 next year.
This is a risk-mitigated clinical trial that's supported by an SPA with the FDA, Fast Track from the FDA, and also optimized based upon previous clinical trial results. It's a very compelling commercial opportunity. There's no approved treatments in A-T. We believe it's a $1 billion+ commercial opportunity and very similar to Friedreich's ataxia. In addition to A-T, we've also selected DMD as our second indication. And given the high unmet need and clinical benefits of steroids in these patients, we believe that we can move quickly through trials there and transition patients from conventional steroids to EryDex. And then we're well-positioned to execute. We have cash runway through phase III results in Q4 of next year all the way into 2026. And then we have a very experienced leadership team that can take us all the way through approval and launch. So that finishes up the corporate presentation.
Are there any questions? All right. Well, thank you for your time today. Oh, one question.
Sir, so going back on the history of the company, you guys acquired EryDex, if I recall correctly, as a medium of.
Correct. Yeah. We acquired EryDel. That was a small private Italian company last October.
Understood. So I guess my question would be, as far as that's concerned, as this kind of continues, where do you guys see yourselves basically after EryDex gets to FDA approval and along those lines? Is this going to be another case of you'll get to that point and then you'll look for another acquisition, do you think? Or what do you think? Where do you see yourself?
I think our plan would be to expand out into other indications, kind of like argenx, where you're in a dozen different rare disease indications, where we already own the product. We have worldwide rights. We have no royalties on it. So I think we would prefer to stick with that since we can move directly into human trials with it. All right. Thank you for your time today.