Rockwell Medical's CEO Hosts Phase III CRUISE-1 Clinical Trial Results Conference (Transcript)

| About: Rockwell Medical, (RMTI)

Rockwell Medical, Inc. (NASDAQ:RMTI)

Phase III CRUISE-1 Clinical Trial Results Conference

July 11, 2013 8:00 am ET

Executives

Paul Arndt - Former Senior Manager of Investor Relations

Robert L. Chioini - Founder, Chairman, Chief Executive Officer, President and Member of Stock Option Committee

Raymond Dennis Pratt - Chief Medical Officer

Ajay Gupta - Chief Scientific Officer and Member of Scientific Advisory Board

Analysts

Annabel Samimy - Stifel, Nicolaus & Co., Inc., Research Division

Carol Werther - Summer Street Research Partners

David Moskowitz

Operator

Good day, ladies and gentlemen, and welcome to the Rockwell Medical Technologies Phase III CRUISE-1 Clinical Trial Results Conference Call. [Operator Instructions] As a reminder, today's conference call is being recorded.

I'd now like to turn the conference over to your host, Mr. Paul Arndt. Please go ahead.

Paul Arndt

Thank you, Allie. Good morning, and thank you for attending the Rockwell Medical conference call. I am Paul Arndt from LifeSci Advisors. On the call this morning are Rob Chioini, CEO and Chairman of Rockwell; Dr. Ray Pratt, Chief Medical Officer of Rockwell; and Dr. Ajay Gupta, Chief Scientific Officer of Rockwell. We will be sharing the results of the CRUISE-1 Phase III clinical study with you. We will also do a Q&A following the presentation.

Before we begin, I'd like to remind everyone that various remarks that we may make about our future expectations, plans and prospects constitute forward-looking statements for purposes of Safe Harbor provisions under the Private Securities Litigation Reform Act of 1995. Rockwell cautions that these forward-looking statements are subject to risks and uncertainties that may cause our actual results to differ materially from those indicated.

Among the factors that could cause our actual results to differ materially include risks related to the timing for a submission of the NDA and whether the FDA will accept such submissions for review following submission and ultimately approve them; whether the FDA will concur with our interpretation of our Phase III study results or the conduct of the study; our ability to successfully and cost effectively complete clinical trials, submit new drug application and obtain marketing approvals for SFP. Top line results are based on a preliminary analysis of then available data, both safety and efficacy, and there is the risk that such findings and conclusions could change following a more comprehensive review of the data; our ability to meet anticipated development timelines for SFP due to clinical trial results, manufacturing capabilities or other factors; uncertainties related to the regulatory process and other risk factors identified from time to time in our reports filed with the Securities and Exchange Commission.

Any forward-looking statements made on this conference call speak only as of today's date, Thursday, July 11, 2013, and we do not intend to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof.

Rockwell would also like to remind everyone that the results that will be discussed on this call are top line results and that the full safety and efficacy data set will be presented at a future medical conference. This conference call is being recorded for audio rebroadcast on Rockwell's website, www.rockwell.com (sic) [www.rockwellmed.com], where it will be available for the next 14 days. [Operator Instructions]

I'll now turn the call over to Rob Chioini, Founder, Chairman and CEO of Rockwell Medical.

Robert L. Chioini

Thank you, Paul. Good morning, everybody. Thank you for joining us. This is obviously a very exciting day for the company, our employees, our investors and the physicians who have worked diligently to advance SFP to this point. I want to thank the clinical investigators, the Rockwell clinical team and certainly, the patients who volunteered to be part of this clinical program.

By now, many of you have seen our press release regarding the results from our pivotal Phase III CRUISE-1 study of SFP, the company's late-stage investigational iron delivery drug for iron replacement in chronic kidney disease patients receiving hemodialysis. On this call, we will discuss these highly positive top line clinical results that SFP delivered, focusing on the primary and secondary endpoints, as well as the safety profile.

There is no question that SFP delivered strong data today and that this Phase III data is a resounding success. We believe the data presented today clearly suggests that SFP has the potential to become the market leader in the iron treatment space. The CRUISE-1 study met its primary endpoint and achieved statistical significance with a p-value of 0.011. This is exceptional Phase III efficacy data. For reference, 2 clinical trials with a p-value less than 0.05, along with strong safety data, is generally required by the FDA to approve new drugs.

These successful Phase III results, coupled with the recent positive PRIME study data, demonstrating SFP's ability to significantly reduce ESA use by 35%, support our belief that SFP will set a new paradigm in iron therapy treatment for hemodialysis patients as it will deliver both significant clinical and pharmacoeconomic benefits. We believe SFP is positioned to become the new standard of care on iron therapy, and we expect it to severely disrupt the current IV iron market in dialysis.

CRUISE-1 is the first of 2 identical efficacy studies, and we anticipate that upcoming CRUISE-2 results confirm the CRUISE-1 data. It appears CRUISE-2 data will be released prior to our original guidance of October of this year. We are working toward submitting our new drug application to the FDA.

Joining me on today's call to discuss the significance of the Phase III efficacy data and the potential positive impact that SFP could have on dialysis patients suffering from functional iron deficiency is Dr. Ray Pratt, our Chief Medical Officer; and Dr. Ajay Gupta, our Chief Scientific Officer, both of whom were obviously very involved in this study. Ray will take us through the data, and then we will field your questions.

I will now turn the call over to Dr. Ray Pratt, our Chief Medical Officer, who will take you through the Phase III CRUISE-1 efficacy results.

Raymond Dennis Pratt

Thank you, Rob. For those of you on the phone who don't know me, let me introduce myself. I'm a board-certified nephrologist with extensive experience in basic science research, academic and clinical nephrology practice and 20 years in pharmaceutical clinical development. I have been involved in multiple NDAs for both renal and CNS drugs. I was responsible for FOSRENOL and epoetin delta for renal patients, as well as instrumental in the approvals of Aricept for Alzheimer's disease and Daytrana for patients with ADHD. I joined Rockwell 1 year ago to lead the team efforts for the NDA for SFP after evaluating the opportunity and understanding the potential that SFP and the company has.

I would like to point out that the results I will discuss today are top line data. We do not have granularity into patient level data at this time, and we will be happy to provide additional information once we have completed our full analysis of the study. First, I want to say we're thrilled with these outstanding clinical results from the Phase III efficacy study. The study was well designed and well run. Rockwell would like to thank all the sites and patients who participated in this study. We would also like to extend congratulations to our investigators and the entire clinical team for the work they put in to making the trial a success.

As presented in the press release, the CRUISE-1 pivotal study met its predefined primary endpoint, demonstrating a statistically significant change from baseline in hemoglobin values between SFP and placebo groups. The mean difference between SFP and placebo at the end of treatment was 3.6 grams per liter in favor of SFP with a p-value of 0.011. This is a fantastic result, and we expect to see a similar outcome from our soon-to-be completed CRUISE-2 study.

Now let me take you through the study design before I talk more about the results. The CRUISE study was designed to show that SFP administered via dialysate can maintain hemoglobin levels by providing iron to the bone marrow, replacing the iron loss patients experience during their dialysis treatment. The primary efficacy endpoint in the study was a statistically significant change from the baseline in hemoglobin values between SFP and placebo groups.

Hemoglobin value is the FDA-defined, clinically meaningful endpoint for this trial. Because SFP is a maintenance therapy, we designed this trial to control the factors which influence hemoglobin level in dialysis patients, primarily erythropoiesis-stimulating agents or ESAs and IV iron. As a result, the study did not permit any adjustment to ESA dose once the patient entered an initial run-in period nor does it allow IV or oral iron.

Only 1 group of patients received iron, and that was the iron from SFP delivered via the dialysate during regular hemodialysis in the active treatment arm of the study. Therefore, we had 1 group of patients getting SFP-iron and the other receiving no iron. Neither group was able to have their ESA dose altered. This control was designed to show that SFP can consistently deliver iron and maintain hemoglobin, and that's exactly what these successful results confirmed.

This was a single-blind, placebo-controlled study. Patients could not distinguish SFP dialysate from placebo. Patients who were iron replete by TSAT and ferritin parameters with a hemoglobin within a range of 95 to 115 grams per liter and who had stable ESA doses during the run-in period could be randomized. Once randomized, patients' hemoglobins were followed weekly and iron parameters every other week.

Over time, we expected that some patients' hemoglobins would drop, some would rise and at certain levels, there would be safety issues to take into consideration. To address that, the study incorporated predefined criteria based on the CKD HD guide -- anemia guidelines to remove patients during the study when certain criteria were met. Those criteria were: one, a need to change ESA dose for hemoglobin values that became too low, less than 90 grams per liter, or too high, greater than 120 grams per liter; two, a rapidly rising hemoglobin of greater than 115 grams per liter, with an increase of 10 grams per liter over a 4-week period; or three, a serum ferritin less than 100 microgram per liter.

Patients remained in the study for as long as their hemoglobin and ferritin values remain within prespecified limits. When patients met the predefined criteria I just mentioned, they were removed from the study and eligible to enroll in the open-label extension study. Patients could also be removed for any of the other reasons common in clinical studies, including transfusions, withdrawal consent, kidney transplant or adverse events.

The study population was well balanced at baseline, with 149 patients receiving SFP and 151 placebo. The population for the primary endpoint consisted of all patients who received at least 1 dose of study drug and had at least 1 post-baseline hemoglobin value. The primary endpoint was defined as the change from baseline of the average of the hemoglobin values during the last 1/6 of the time patients were in the randomized days of the study. Patients met criteria for anemia management changes and other criteria for removal at the same rate during the study in both treatment groups. Importantly, withdrawals due to adverse events were the same in both groups. All patients contributed hemoglobin values for the endpoint calculation.

The CRUISE-1 study met its predefined primary statistical endpoint, the difference in the change from baseline in hemoglobin values between SFP and placebo groups. At the end of treatment, in the SFP group, the mean change from baseline in hemoglobin was positive 0.6 grams per liter. In the placebo group, the change from baseline was a significant minus 3.0 grams per liter drop in hemoglobin. Thus, the mean difference between SFP and placebo at the end of treatment, the primary endpoint, was 3.6 grams per liter in favor of SFP, with a p-value of 0.011. The average time that patients remained in the randomized treatment phase of the study was 23 weeks or almost 6 months.

Secondary endpoints included the reticulocyte hemoglobin concentration and serum ferritin. The reticulocyte hemoglobin concentration is the most important index of iron delivery to erythrocyte precursors and is the earliest indicator of the development of iron deficiency. In the SFP group, the reticulocyte hemoglobin remained stable near baseline levels, and this is an important clinical result. In the placebo group, the reticulocyte hemoglobin declined by a statistically significant 2.1% from baseline, with a p-value less than 0.001. This indicates that in patients receiving SFP, iron is delivered via the dialysate, binds to apo-transferrin and is rapidly taken up by the bone marrow for erythropoiesis. In the placebo group, over time, the declining reticulocyte hemoglobin would ultimately lead to iron deficiency anemia, if unchecked.

Ferritin, an index of body iron stores declined in both groups. The decline from baseline in the SFP group was 14.8%, while the decline in the placebo group was 28.5%. The difference between the groups was also statistically significant, with a p-value less than 0.001. This is also a very important result. It indicates that patients receiving SFP-iron use the dialysate-delivered iron for RBC production rather than storing it. That SFP can deliver iron to the bone marrow, maintain hemoglobin and not increase iron stores demonstrates that iron is being utilized for hemoglobin production and not deposited into reticuloendothelial system. This is the opposite of what occurs with IV iron preparations, where iron must be processed by macrophages in the RE systems before it is available for utilization in erythropoiesis.

Let us now turn to the safety. The safety profile of SFP was excellent. The overall profile for SFP is similar to placebo with respect to the incidence and severity of adverse events, serious adverse events and abnormal laboratory values. There were 8 deaths in the study, 5 in the SFP group and 3 in the placebo group. No death was considered related to study drug. Intradialytic hypotension was reported at a similar frequency in the SFP and placebo groups. Treatment-emergent cardiovascular disorders were also similar between SFP and placebo. There were no differences in vascular access complications between treatment groups.

With regard to infections, there were also no differences between SFP and placebo in the total number of infection or in any specific infection type. Very importantly, SFP did not cause anaphylaxis or hypersensitivity reactions during the study, which encompassed over 10,000 individual administrations of SFP during dialysis.

So our conclusions from the Phase III CRUISE-1 efficacy study. CRUISE-1 met its prospectively defined primary endpoint for regulatory purposes. SFP maintains hemoglobin in the absence of IV iron administration. SFP also maintained reticulocyte hemoglobin. SFP maintained iron stores relative to placebo as reflected by a difference in ferritin values between SFP and placebo groups at the end of treatment. SFP did not induce iron overload as evidenced by no increase in ferritin values above baseline. SFP did not need to be discontinued because of evidence of iron overloads. There were fewer patients transfused in the SFP group, 1 compared to 7 patients transfused in placebo group.

Overall, the safety profile was excellent. Adverse events and serious adverse events in the SFP group were similar to patients receiving placebo. There was no increase in intradialytic hypotension, infections, anaphylaxis or hypersensitivity reactions compared to placebo-treated patients. The benefit-risk profiles for SFP to maintain hemoglobin levels was very favorable. The population receiving SFP had a statistically significant difference from placebo in hemoglobin levels, with an absence of adverse events commonly attributable to iron administration.

In closing, the CRUISE-1 study was the first of 2 identical Phase III pivotal studies. Based upon CRUISE-1 results, we anticipate that the CRUISE-2 study, scheduled to complete shortly, will show confirmatory results. With a positive CRUISE-2 study, Rockwell will be on track to submit an NDA as soon as possible. The CRUISE-1 study supports and strengthens the results of the PRIME study reported at the ERA EDTA meeting earlier this year. Together, the 2 studies demonstrate that SFP can effectively deliver iron to patients, maintain hemoglobin, significantly reduce ESA use and not increase iron stores.

We are very excited about SFP. As a clinical nephrologist, I believe this is a real paradigm-shifting drug not only in the field of renal anemia, but in nephrology as well. I agree with the opinion of our study KOLs that SFP may be one of the most important therapeutic advances in the treatment of renal anemia since the introduction of recombinant erythropoietin some 25 years ago. We expect to present the results of this study as a late-breaking clinical trial at the upcoming American Society of Nephrology meeting in November 2013.

Thank you, all, for your attention, and I'll turn the call back over to Rob.

Robert L. Chioini

Thank you, Ray, for that comprehensive overview. Before we go to questions, I want to provide some perspective related to the company's strategic vision. As you know, SFP is the company's lead drug candidate and was the focal point in making our decision to rebrand Rockwell in 2008 and embark on a new strategic path to become a dominant biopharmaceutical company in the renal space.

Since the company's inception, we have built a world-class commercial business selling our dialysate products to dialysis centers in the U.S. and x U.S. When we made the decision to leverage this existing infrastructure to become a dominant biopharmaceutical company in the renal space, we hired and developed a top-notch clinical team and raised additional capital to fund our development programs, among many other initiatives, while still managing and growing our dialysate concentrate business.

Our view is that our core business is the ideal sales channel for our pipeline products as they reach the market. Our initiative has required significant effort and commitment but was required and needed to build the type of company that we have today and whose goal is to be a leader in the renal drug space. We believe that today's results move us closer to achieving that objective.

With that, I will now ask the operator to open up Q&A.

Question-and-Answer Session

Operator

[Operator Instructions] Our first question comes from Annabel Samimy of Stifel, Nicolaus.

Annabel Samimy - Stifel, Nicolaus & Co., Inc., Research Division

Congratulations on the data. I wanted to discuss the predefined dropout that was incorporated into the trial. And I guess the first thing is, we understand that there's a big dropout. I just wanted to understand what is the dropout rate difference between the SFP arm and the placebo arm. Realistically, we think that the placebo arm will have a greater dropout rate, but can you just give us a little bit of color around that and exactly what the incidence of the various adverse events -- the difference between the adverse events in those 2 groups, the dropout between the placebo and the dropout between SFP?

Raymond Dennis Pratt

Okay, very well. I can tell you that for the protocol-mandated changes in anemia management, the dropout rate in the SFP group was approximately 45% of the patients, whereas the dropout rate in the placebo group was approximately 53% -- 54% of the patients. For the rest of -- the dropouts for other reasons were relatively well balanced across the SFP and placebo groups. With regard to the adverse events, specifically in those that dropped out of -- for the protocol-mandated changes, we don't have that information available just yet. The study design really did call for the patient removal, though because of the safety concern of hemoglobins going below 9 or above 12 grams per liter, again, according to the latest guidelines, and dialysis units can get very touchy about having patients staying in studies with hemoglobins either dropping or going up above these levels, given the safety concerns that have been brought up in the community.

Annabel Samimy - Stifel, Nicolaus & Co., Inc., Research Division

Okay. And is this how other iron studies have been designed with this kind of dropout -- predefined dropout?

Raymond Dennis Pratt

Well, the other iron studies have all been conducted in patients with iron deficiency anemia and were very, very short-term studies. They really have been no long-term controlled studies such as this in patients with iron deficiency with the other iron products. Again, they're designed for treatment of iron deficiency anemia. We are working on being a maintenance therapy for replacing iron losses during -- in patients during dialysis, so therefore, this is why we did a very, very long study.

Annabel Samimy - Stifel, Nicolaus & Co., Inc., Research Division

Okay. Can you also help us understand how the open label was designed, once these patients dropped out, how they were sort of managed through the open-label study?

Raymond Dennis Pratt

Patients were transitioned into the open-label study where IV iron and ESA doses could be titrated at will, and these patients were able -- are able to stay in the study for up to a total of -- if you look at the CRUISE -- at the randomized treatment period plus the open-label study, for a total of 72 weeks. We have approximately 206 patients enrolled, moved over from the randomized treatment phase into the open-label study. And we've had approximately 84 of patients from the CRUISE-1 study already complete 72 weeks of therapy in the open-label study. The open-label study is still ongoing, so we don't have any data that we can share with you at this point.

Annabel Samimy - Stifel, Nicolaus & Co., Inc., Research Division

Okay. And if I can ask 1 more question. Obviously, this specific study is not necessarily real-world practice, and so between the PRIME study and the CRUISE study, what can you glean from this in how SFP is going to be used in the real world?

Raymond Dennis Pratt

We believe that SFP will be used in dialysis patients to maintain their hemoglobin therapy and replace those -- replace the iron losses that actually occurs during each dialysis treatment from residual blood left in the dialyzer circuit and the multiple phlebotomies that these patients undergo all the time. It will -- it should decrease the use of ESA, and it should also reduce the use -- the need for repetitive megadoses of intravenous iron in this patient population.

Operator

Our next question comes from Carol Werther of Summer Street.

Carol Werther - Summer Street Research Partners

I'm also trying to understand better the patients that dropped out. So with the 43% -- 45% that dropped out in SFP, in the real world -- I mean, I guess you don't have details whether or not they dropped out due to higher hemoglobin or lower hemoglobin. But in the real world, would they potentially stay on SFP and get iron replacement if their hemoglobin is low? Or would they get an increase in ESA? How does that work?

Raymond Dennis Pratt

Okay. Typically, in the real world, and again, going back to the PRIME study, which could actually -- which was very similar to what would happen in the real world, you would see that patients whose hemoglobin would be rising, then the algorithms that the dialysis centers will kick in and ESA doses would be decreased, if that was the case. For patients whose hemoglobins were dropping, again, the ESA dose could be increased to increase the utilization of iron delivered via SFP. On the other hand, if hemoglobins are dropping, you need to realistically look at what are sources -- what are the other sources of blood loss that these patients actually have. Many of them are having GI bleeds. They have access problems, where they come in and they need to have -- they lose blood because they need access surgery. There's a myriad of reasons that are there. It won't eliminate the need completely for intravenous iron when patients become iron deficient, and that's what we showed in the PRIME study, is that when patients reach a very low serum ferritin value, they require the use of some IV iron. But the SFP group reduced that usage of IV iron by about 50%. Ajay, our Chief Scientific Officer, would also like to make a comment about that.

Ajay Gupta

Thank you, Ray. If patients actually drop their hemoglobin, these patients have their iron parameters monitored monthly. And then, if the iron parameters are consistent with iron deficiency, then the patients may need some supplemental intravenous iron. But we believe the majority of patients would maintain their iron balance on SFP without the need for intravenous iron.

Carol Werther - Summer Street Research Partners

Okay. So from this study, we would be able to tell, like, what percentage of patients might need supplemental IV iron? Or is that correct? Or will we have that information later on?

Raymond Dennis Pratt

Yes. I mean, we will be able to have a basic index of patients who actually reach criteria for requiring intravenous iron. Again, one of the criteria was if the ferritin level dropped below 100 micrograms per liter, which is indicative of iron deficiency. So we do have -- we will have that data later.

Carol Werther - Summer Street Research Partners

Okay. And then your thoughts on commercialization, are you thinking about doing partnerships with the big dialysis centers?

Robert L. Chioini

So this is Rob, Carol. I'll answer that question. As far as commercialization goes, we expect to get this drug out to all the dialysis providers. And as you know, the market is very segmented or fragmented. You've got the 2 big providers that control maybe 2/3 of the market and then a handful of change and other smaller groups that make up the rest of the market. Our plan is to get the drug to all of them. Obviously, with, like, a group like DaVita, who we have an excellent relationship with and we just recently signed a 5-year contract with, they were involved intimately in the study, as well as Fresinius. And we will expect that with these groups, we would negotiate with them, they would do some evaluation, and then, if they decide to go with the drug, they would roll it out to all their clinics.

Carol Werther - Summer Street Research Partners

And so from your CRUISE trials and the PRIME study, do you think that's enough of a definition of how much ESA and IV iron they're going to save so that you can price it correctly?

Robert L. Chioini

I think both studies represent what we think will happen in the marketplace. We look -- I look at the CRUISE study and what we see is that the patients are able to be given SFP in place of IV iron and maintain their hemoglobins. We look at the PRIME study and we see that patients are able to be given SFP, again maintain their hemoglobins in place of IV iron and also reduce ESA use. And so the drug -- what makes this drug very special, in our view, is that it clinically -- it has clinical benefits for the patients, great clinical benefits; it has strong safety, which falls under that category; and then it also has great economic benefits for the providers in the marketplace.

Carol Werther - Summer Street Research Partners

Okay. And I guess -- so we would imagine that once it's commercialized, it would -- companies would change over to this dialysate. And would we imagine that the dropout rate would be lower than the 45% then because you could make adjustments in the real world?

Robert L. Chioini

I'll -- let me first say, just to clarify, it's a drug that will be packaged and shipped separately to the clinics. It's not going to be packaged into dialysate. It will be added to the dialysate once at the clinic site. And then I'll let Ajay and Ray answer the second part of that.

Raymond Dennis Pratt

Yes. I mean, first thing is this is -- this was a clinical trial in which patients' ESA doses were clamped. They could not be changed during the course of participation in the trial. So in the real world, as we saw in the PRIME study, ESA doses were changed over time to match the hemoglobin values according to the site's -- to the algorithms that are out there for use of ESA. So this was a study that was designed to maintain patients within the safety bands that have been established by the anemia guidelines for hemodialysis patients. And when patients exceeded or dropped out of that, rather than keep them in the study -- who dropped below those limits, rather than keep them in the study, they were removed to the open-label study, where normal practice could then be retained. So these patients were not dropped out, they were removed prospectively because of the design of the study.

Carol Werther - Summer Street Research Partners

Okay. I just have 1 last question. So would there be any dialysis patients that would be contraindicated for SFP?

Ajay Gupta

This is Ajay. So it is -- there's no incidents of any anaphylactic reactions to SFP. And also, patients do not get iron overloaded on SFP, and the ferritins do not increase. So at this point in time, we cannot identify any patient population or any patients who would not be eligible for SFP administration.

Operator

[Operator Instructions] Our next question comes from Dan Bailey [ph] of Source Capital Group.

Unknown Analyst

Congratulations, guys. I think you've got a very sick population of patients who are going to be very happy now with this new therapy. Two quick things. One, because there seems to be a lot of focus on it here, you are dealing with very sick patients, so you had to have some strict controls in place to make sure these clinical needs are met. And the big takeaway here is that you're not giving them something to help replace their iron that's going to give them other medical issues to worry about such as the anaphylaxis shock and the hypersensitivity, correct?

Ajay Gupta

That is correct, yes. We have now delivered approximately 60,000 doses of SFP in our clinical program, and we haven't had a single instance of hypersensitivity or anaphylactic reactions.

Unknown Analyst

And I think people are losing sight of the fact how sick these patients actually are and the dropout rate, it is what it is, these are very sick patients. My question is on the CRUISE-2 that you mentioned at the start of the call, you then announced in May that the last patient was dosed on the CRUISE-1. You'll have results in July. I believe we had guidance that we'd see probably data for the CRUISE-2 in October. Sounds like you're a little bit ahead of schedule. Can you give us any better guidance than that at this point in time?

Robert L. Chioini

Yes. All we can tell you is the study looks like it will -- the last patient was dosed sooner than what we anticipated, and so we know it will be sooner than October. But we still don't have -- it would be too difficult to give you a hard gauge. So right now, we're just saying we know it's going to be sooner than later.

Operator

Our next question comes from David Moskowitz of Empire Asset Management.

David Moskowitz

Just one more time back on the dropout rate, did you guys offer the proportion of dropouts in the SFP group due to hemoglobin changes?

Raymond Dennis Pratt

We know that -- actually, we don't have that number for hemoglobin changes. We do know that for proposed ESA dose changes, it's about -- the majority of patients are actually -- of those patients who moved out from the protocol-mandated change to be removed from the study was about 43% of SFP patients and about 45% of the placebo patients.

David Moskowitz

So you're saying that's out of the 40-and-change percent dropout rate for SFP, 43% of those?

Raymond Dennis Pratt

Yes. 40 [ph] -- almost 80%. I'm giving you the exact numbers based on the total of the patients there. But it was the majority of them were due to ESA dose changes.

David Moskowitz

So just again clarifying, so out of the dropout rates you're giving, most of those patients are the full dropouts that you're going to drop out to...

Raymond Dennis Pratt

They're not dropped out. It's the patients prospectively removed from the study. There's a big difference between the dropout for other -- for adverse events and the normal way people look at dropouts and for patients who are removed from the study for a prospective protocol-mandated change.

David Moskowitz

Okay, I appreciate that. And one more on the -- since ESA use with SFP is lower by -- you've proven that it's lower by about 50%, I mean, shouldn't -- isn't it intuitive that these dropouts are due to the higher levels of hemoglobin in these patients?

Ajay Gupta

You would expect that in the SFP group, the patients' hemoglobin would rise. And once the hemoglobin rises above 12, we cannot keep the patient in the study because the ESA dose is clamped. And for safety reasons, the ESA has to be reduced, and therefore, these patients are not dropped out. They are actually removed from the study because of high hemoglobin so that the ESA dose can be titrated. And now these patients have transitioned into an open-label phase, where they get SFP, the ESA dose can be titrated down, and the patients can receive, if needed, intravenous iron. So these are removals from the study according to protocol-specified criteria in compliance with the FDA guidance around ESA usage and hemoglobin targets. Just to give you some specific numbers, these protocol-mandated dose changes in anemia management led to the withdrawal of 69 patients in the SFP group and 82 patients in the placebo group.

Operator

Our next question comes from Enith Bilal [ph] of KVP [ph] .

Unknown Analyst

Congratulations on the data. It looks very impressive. I had a couple of questions. Let me just first start with this. Can you -- first of all, I didn't see in the press release or in the comments what the baseline ferritins and ESA dose were . Can you comment?

Raymond Dennis Pratt

Sure. We can -- I can tell you about the baseline ferritins, which were about 550 in each of the 2 groups. And the baseline hemoglobins going into the study were, again, very well balanced at about 109 grams per liter values. I don't have the data yet for the baseline ESAs.

Unknown Analyst

Okay. Sorry, one last thing, baseline TSAT?

Raymond Dennis Pratt

Baseline TSATs were about 27% to 29%.

Unknown Analyst

Okay, great. My second question was there's been a lot of discussion about the patients that were removed from the study. And I guess one other attack on this question is, have you guys run a completer analysis?

Raymond Dennis Pratt

No, we have not done that yet. We're waiting for those results to come.

Unknown Analyst

Okay, great. And then my last question is, I guess, in some ways the -- as we -- the changes in hemoglobin were -- are very impressive and are larger than, I think, anyone expected. At some level, the changes in ferritin are smaller than we expected -- as I expected in the control arm and in the SFP arm. And we are also seeing sort of a much higher variability in terms of variance in change from baseline. Just I'm trying to wrap my head around -- obviously, the study -- the results are very significant, but it's interesting how you got there. Wondering just how to connect the dots here. Were you surprised at that sort of constellation of findings?

Raymond Dennis Pratt

Go ahead, Ajay.

Ajay Gupta

So the decrease in the ferritin that we have observed in our clinical program, when you look at the Phase IIb study, when we look at the PRIME study and now looking at the CRUISE-1 study, these are actually very similar across the placebo group and the SFP group. Just to give you an example, in the Phase IIb study, which was a 6-month study, we saw about 7%, 8% decrease in ferritin over a 6-month study. And now in this 12-month CRUISE-1 study, we are seeing about a 14% decrease in the ferritin levels, which we believe is very desirable. So the goal of iron therapy is to maintain the iron delivery to the bone marrow for hemoglobin generation and erythropoiesis at the cost of or concomitantly with low body iron stores. Because iron stored in the body, in terms of high ferritin levels, is actually harmful and correlates with oxidative stress, inflammation, cardiovascular disease. And therefore, we feel that given the fact that we maintain iron delivery to the bone marrow, as demonstrated by maintaining the reticulocyte hemoglobin while concomitantly the reduction in the ferritin level is a very desirable effect of SFP therapy.

Operator

Our next question is a follow-up from Annabel Samimy of Stifel.

Annabel Samimy - Stifel, Nicolaus & Co., Inc., Research Division

I just want to go back to the removal of these patients. We've been referring to them as dropout. But you're, I guess, correcting us by saying they're not really dropouts, they're removals into this open-label study. If I understood correctly, most of the patients who were removed on the SFP arm, they were removed quite possibly because their hemoglobin was increasing too much and then you had to reduce the ESAs. I mean, I guess the first thing people assume is when the patient is removed, it's going to be because of a negative condition or a negative event. But are we to assume that it's because they are seeing very good effects that they need adjustment and reduction of ESAs?

Raymond Dennis Pratt

Yes. I mean, I think there are going to be some patients who are going to have very, very good responses, some patients maybe not so good. And you're also trying to keep patients within this target hemoglobin range by not changing your ESA doses, so it goes both ways. There are some patients that are also prospectively removed because their hemoglobin actually is going below the specified value there and would require either an increase in their ESA dosage or perhaps, if they actually have become iron-deficient, they might perhaps need a dose of IV iron. We don't have the granularity on that data just yet, so we can't really go into any further detail on it.

Annabel Samimy - Stifel, Nicolaus & Co., Inc., Research Division

Okay. I guess I was going to go to my next question, which was, is it because they're responding well or not responding well enough. But I guess, we'll get that granularity at ASN or with follow-up data releases. Can you just give us an idea when we might see that?

Raymond Dennis Pratt

Probably ASN is when we're planning on actually releasing the full results of this study.

Operator

Our next question is a follow-up from Carol Werther of Summer Street.

Carol Werther - Summer Street Research Partners

So with the open label extension data, will you have some of that at ASN?

Raymond Dennis Pratt

No, we won't really have that. That study is still ongoing, and we still have a substantial number of patients both from CRUISE-1 and CRUISE-2 in the open-label studies. And we don't anticipate having that until those studies actually close out.

Carol Werther - Summer Street Research Partners

And when might they close out?

Raymond Dennis Pratt

Yes, they will be closed out around the end of the year.

Carol Werther - Summer Street Research Partners

Okay. And then can you just update us, now that the CRUISE-2 trial is going to have a result earlier, does that change the timing of the NDA filing?

Raymond Dennis Pratt

At this time, no, we don't have any -- we don't really have an update on the NDA filing. We don't anticipate it will change anything.

Operator

That's all the time we have for today's conference. I would now like to turn the conference back over to Rob Chioini for any closing remarks.

Robert L. Chioini

I want to thank everyone on the call today. I want to thank our investors for their commitment and support. Needless to say, this is a very exciting moment for all of us at Rockwell, as well as all the investigators that are involved in the study.

Please feel free to call us with any follow-up questions you might have. We are available. Again, thank you for your participation and commitment. This is a very, very exciting day for the company, and we look forward to great things with this drug.

Operator

Ladies and gentlemen, this does conclude today's conference. You may all disconnect, and have a wonderful day.

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