Good morning, everyone, and welcome to our next session. I'll be your moderator, Sarah Nick, and I'd like to introduce our next presenter, Jill Broadfoot, CFO of aTyr, a biotherapeutics company focused on researching the extracellular functionality and signaling pathways of tRNA synthetases. With that, Jill, the floor is yours.
Thank you, and thank you, to H.C. Wainwright for inviting us to present at the conference again. Before I start, I need to remind you that any forward-looking statements I may make are subject to certain risks and uncertainties, so please refer to our SEC documents. So who are we? What makes us different than any other biotech company out there? I think the most exciting thing that we have right now is that we have a phase III readout next year and an indication that has a $2-3 billion market opportunity with little competition. So we are positioned very well from a cash perspective, from a data perspective, and particularly from a platform perspective. This drug is based upon our platform, tRNA synthetases, that was discovered by Dr. Paul Schimmel. Dr.
Schimmel has been able to translate new science into multi-billion dollar companies, so he has a proven track record with Alnylam, Cubist, and a few other companies, where he's been able to take science that he's developed and move it into medicines that have been helping patients. So this tRNA synthetase biology is something that he discovered a little while ago. We've been working on it. We own all the IP around it. There's 20 tRNA synthetase genes but there's over 200 fragments and splice variants that we own patents over. Our first focus has been on interstitial lung diseases, and that's the drug that I said that really differentiates us among other biotechs here at this conference. It's in an area of interstitial lung disease called pulmonary sarcoidosis. Very few people know what pulmonary sarcoidosis is.
Today is 9/11 . Many of the nine eleven workers developed pulmonary sarcoidosis. It's a very severe disease. We enrolled 268 patients in this trial. It's the first phase III trial in pulmonary sarcoidosis, and it's the largest trial in pulmonary sarcoidosis. We're very excited about our opportunities in interstitial lung diseases. To just take a look at the platform and tRNA synthetases and what that is, how it's worked for efzofitimod, is basically we've known that tRNA synthetases make proteins within cells. What Dr. Schimmel discovered is that these splice variants and fragments actually migrate out of the cells and create homeostasis of everyone's immune system. These fragments or splice variants have evolved over billions of years to acquire novel domains, and that's how basically our immune system is being regulated.
We can see some of these fragments and splice variants migrate to certain tissues within the body, which is how we developed efzofitimod from the one of the HARS fragments that we saw was enriched in lung tissue. So let's look at the pipeline before we dive into efzofitimod. That's where we're really gonna focus this presentation on, but we are looking at the other nineteen tRNA synthetases out there and the fragments associated with those in the background. So with ATYR0101 and ATYR0750, what we've done is we've identified the receptor, which is the same thing we did for efzofitimod, and for example, in ATYR0101, we identified LTBP1 as the receptor, and we're looking at types of fibroses, like kidney fibrosis.
We found a way to basically target each of these different synthetases by finding that receptor. But going to efzofitimod, we found that the receptor was NRP2 and went into interstitial lung diseases. You can see our phase 3 trial there in pulmonary sarcoidosis, but we also have a phase II trial in scleroderma ILD in up to 25 patients. Both of those have orphan drug designation in the U.S. and in Europe, and they both have Fast Track designation in the U.S. We've partnered efzofitimod in Japan with Kyorin Pharmaceuticals. It's a $175 million partnership, where we've realized $20 million to date through upfront and milestone payments. So going into efzofitimod, efzofitimod is an Fc fusion protein.
We fused it with the human IgG1 Fc to enhance its life, so that we can dose once a month through a sixty-minute IV infusion. We believe the mechanism of action down-regulates myeloid cells via that NRP2 receptor, so we bind selectively to NRP2 and that allows us to actually change those myeloid cells and stop the inflammation that's occurring in the lungs. We saw this through over half a dozen different types of animal models that we did prior to going into pulmonary sarcoidosis patients. We saw both an anti-inflammatory effect and an anti-fibrotic effect. And then we believe our phase two trial in pulmonary sarcoidosis actually has given us clinical proof of concept.
So looking at our therapeutic hypothesis with regards to the mechanism of action, in these interstitial lung diseases, there's different immune triggers that happen within the lung. Something happens, your immune system starts fighting it, and it's fighting it, it's fighting it, it can't resolve it, and the lung just becomes inflamed, and eventually, that leads to fibrosis. What efzofitimod does is, during this inflammation process, NRP2 is up-regulated. So efzofitimod is basically binding to NRP2 and stopping that. I call it a circular reference. It's stopping that inflammation process through changing the macrophages and how they're working to basically restore immune balance to the lungs. That's a very simplified way of explaining it, but we have done a lot of work over the last few years to really understand this mechanism of action.
Looking at interstitial lung diseases, I didn't even know about interstitial lung diseases when I first started at aTyr, but there's over 200 types of interstitial lung diseases. IPF is the most well-known, but sarcoidosis, like I said earlier, is still very, very. Most people don't understand what it is. It's an orphan indication, like I said, at about 200,000 patients in the U.S. Scleroderma ILD is about 60,000 patients in the U.S., but those are both the highly inflammatory types of interstitial lung diseases. You can see at the bottom, some have more inflammation, and some have more fibrosis. Because of our understanding of the mechanism of action, that's why we've decided to start with sarcoidosis and scleroderma ILD, and in those two is where we see the $2 billion-$3 billion market opportunity.
Anything outside of that is potential upside that, if we're successful with sarcoidosis, we will most likely move into. So specifically for pulmonary sarcoidosis, like I said, there's about 200,000 patients in the U.S. We just did a claims analysis, where that number looks very solid. For sarcoidosis patients, approximately 90% of the patients have sarcoidosis in the lung. About 20% develop fibrosis. The standard of care here, so there's really no approved drugs out there. The standard of care is steroids, and then there are some patients that go on to immunosuppressants and anti-TNF antibodies. But all of that treatment is very highly toxic. We haven't seen any safety signals in efzofitimod. Efzofitimod has had a phase I-A, I-B ,II-A trial.
We also did a phase II trial in COVID, and then our partner, Kyorin, also did a phase I trial in Japan. No signals, no issues with safety, and in our phase III trial, we've already had two DSMBs. Our PIs believe this could be a first-line treatment to help these patients that really have nothing right now. In our phase II trial, we enrolled 37 patients with pulmonary sarcoidosis, and we had three arms, a one mg, three mg, and five mg arms, including placebo, and we saw a dose response across all of the different endpoints that we were looking at. We saw a reduction in the average daily steroid use versus placebo, we saw improvement in lung function, and we saw improvement in their symptoms.
What's important is the patients that were on drug, as they were tapering down with their steroids, they were actually feeling better, so their symptoms were improving. We did see that some patients on placebo were able to taper down some, but their symptoms worsened. So they were able to get maybe not all the way down to where we wanted them to be with on placebo, but they just couldn't stand it, which is one of the reasons why we designed our phase III trial to be twelve months. This was a six-month study where we took the patients down to five milligrams, and about halfway through the study, the PIs came to us and said, "Hey, I've got some patients. They're feeling really good.
Can I take it down, them down to zero?" So we modified the protocol and allowed the PIs to take their patients down to zero and take them completely off of steroids, and the three patients that were able to do that were in the five mg arm. So we saw dose-response in all of these endpoints, as well as dose-dependent improvement in inflammatory biomarkers. So we're very happy with this data. We feel it's proof of concept. It was published in a peer-reviewed journal, in The Chest Journal. So our phase III trial, we took everything we learned from the phase II trial to design the phase III trial. We increased it from six months to a year to have those patients that are on placebo hopefully have to be rescued during that year-long period.
We're also tapering the patients down to zero from the beginning, versus the phase II, we tapered them down to five mgs and then waited to see if they could handle it, and then allowed them to go down to zero. So between week 2 and week 12, we're tapering the patients, and then from week 12 - 48, we're measuring how many steroids they've taken and just dividing by the number of days. And we're hoping to see about a 25%-35% decrease in the amount of steroids that they're taking. Inclusion/exclusion criteria as well, we tried to modify that a bit based upon what we saw from the phase II trial to really enrich this population. The key secondary endpoints that we're looking at are also lung function and symptom control.
We expect the top-line data in Q3 of next year. And one important thing as well, again, we had the PIs come back to us and say, "Hey, our patients are feeling really good. They've finished the trial, and they want to stay on drug." Well, we don't know if it's drug or placebo, but they want to stay on treatment. So we initiated an individual patient expanded access program. It's done through the center, so it's outside of our clinical trial, but at least it's allowing these patients that are feeling good to stay on drug. Anybody that gets into the EAP is automatically put on 5 mg, regardless of what they were on, and we don't know what they were on. So going over to our phase two trial for scleroderma ILD, again, this is a little bit smaller population.
There is a little bit more competition here. There are some approved drugs here, versus in sarcoidosis, pulmonary sarcoidosis, there are no approved drugs and little competition. We have a little bit more competition here, but there are no disease-modifying therapies available, and everything that is available has significant toxicity, so our positioning here is second line, and hopefully first line to replace MMF and CYC. Our design trial here, since they're not on steroids, there's no reduction, where the primary endpoint is lung function, and we're also looking at the skin, so in our animal studies, we did see improvement in fibrosis, so if we can show something in skin, we think that could be obviously very significant, since nobody has ever seen any improvement for scleroderma patients with skin. We expect interim data in the second quarter of 2025.
So from a value proposition, again, I can't stress enough that we're in an indication that has very little competition. We understand the mechanism of action here. We've seen robust efficacy, we've seen dose response, we've improved lung function, we've resolved symptoms, we've been able to get a significant reduction in the daily steroids that these patients are taking. No known safety issues, so we own all of the IP around not only efzofitimod, but the whole platform that this is built upon. So we really have a great value proposition in efzofitimod and a strong partner in Japan with Kyorin Pharmaceuticals. So just reviewing all of that, again, our drug discovery platform is based upon this tRNA synthetase biology. We own all the IP around that.
We'll have two significant readouts next year, and we are in the background growing that pipeline of tRNA synthetase-derived candidates in ATYR0101 and ATYR0750, and looking at the other tRNA synthetases as well. We have a robust financial position. We have no debt. We have a very clean balance sheet with over $80 million in cash as of the last quarter. So I will go ahead and end that here. And again, thank you very much for inviting us to your conference.