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Operator
Good day, ladies and gentlemen, and welcome to the Amicus 3Q results conference call and webcast. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. (Operator Instructions). As a reminder, this conference is being recorded.
I would like to introduce your host for today's conference, Director of Investor Relations, Ms. Sara Pellegrino. Ma'am, you may begin.
Sara Pellegrino - Associate Director of IR
Thank you, Vince. Good evening, and thank you for joining our conference call to discuss our third-quarter 2014 financial results. Speaking on today's call we have John Crowley, our Chairman and Chief Executive Officer; Chip Baird, our Chief Financial Officer; Jay Barth, our Chief Medical Officer; and Bradley Campbell, our Chief Operating Officer.
Today's prepared remarks coincide with the slide presentation that is now available on our corporate website at www.AmicusRx.com. The slides are located in the Investor section under Events and Presentations, right below the webcast link to today's call.
On slide 2, you will find reference to our Safe Harbor. This conference call contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to business operations and financial conditions of Amicus, including but not limited to, preclinical and clinical development of Amicus candidate drug products, cash runway, and the timing and reporting of results from clinical trials evaluating Amicus candidate drug product. Words such as, but not limited to, look forward to, believe, expect, anticipate, estimate, intend, plan, would, should and could, and similar expressions or words, identify forward-looking statements.
Although Amicus believes the expectations reflected in such forward-looking statements are based upon reasonable assumptions, there can be no assurance that its expectations will be realized. Actual results could differ materially from those projected in Amicus forward-looking statements due to numerous known and unknown risks and uncertainties, including the risk factors described in our Annual Report on Form 10-K for the year ended December 31, 2013. All forward-looking statements are qualified in their entirety by the cautionary statements, and Amicus undertakes no obligation to revise or update this presentation to reflect events or circumstances after the date hereof.
At this time, it is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer of Amicus.
John Crowley - Chairman and CEO
Great. Thank you, Sara, and good evening, everybody. And it's certainly good to be back full-time here at Amicus after my several-month leave for a military reserve duty. And I find Amicus in an even better position than when I left the Company at the end of the summer. So, I'm very excited for the future here at Amicus.
I will only make some very brief opening comments, as we've got a lot of substantive materials to work our way through with the senior team that I have here. And I'll draw your attention to slide number 3 and the third-quarter investment highlights.
There are a lot of great rare disease companies that are out there today doing many great things in the field. And I think Amicus in that field is very uniquely positioned. And if you look on slide 3, we state that we have strength of our clinical programs and the breadth of technology programs, with the potential to create significant value for shareholders and patients living with lysosomal storage disorders. And I think that's never been more true.
Obviously, we're very pleased and excited with migalastat monotherapy -- again, an unpartnered post-Phase III program preparing for its regulatory filings outside the United States as well as here in the United States; and again, the first oral therapy with a unique mechanism of action very distinct from enzyme replacement therapy for Fabry patients with amenable mutations, now again with two positive Phase III studies. And, as Jay will highlight here, we are on track for an EMA pre-submission meeting this quarter.
So, very, very valuable program for us, and I think will offer the potential for great benefit for people living with Fabry. But beyond a precommercial global asset and a rare disease, we also have a terrific pipeline. And we'll talk more about that, because we've had some terrific advances in the pipeline that we will go through -- with the three-and-three strategy, the notion of three new next-generation enzyme replacement therapies into the clinic over the next three years in Fabry, Pompe and NPS.
And we'll spend a bit of time today on our Pompe program, as that's met some great milestones over the past quarter. And again, the notion that we not only have a precommercial asset, we have an expanding pipeline and a series of great proprietary technologies.
So that's where we find ourselves at Amicus today. We have more data -- and we highlight this in the press release that just came out -- additional long-term Phase III data, very important data, that will be presented at the American Society of Nephrology coming up here in a couple of weeks.
We, of course, had some additional long-term Phase III data presented in October at ASHG, the American Society of Human Genetics, that give us further confidence in migalastat as a monotherapy. And Jay will highlight some of that.
I've got Dr. Hung Do and Brad Campbell, who will talk more about our next-generation Pompe ERT. It's been almost a year since our acquisition of Callidus, and Hung rejoining the Company in a senior role in the science organization. And that program could not have gone better over the last year. We'll talk specifically about some good success we've had in the scale-up of that, as well as the very recent first regulatory interaction with the European authorities that Jay will provide an update on, and Bradley, as well.
So, across the board, I think we are in a very, very good position.
With that, let me turn the call now over to Jay, and he will take us through the next couple of slides focused on Fabry monotherapy.
Jay Barth - Chief Medical Officer
Thanks, John. I want to remind everybody on slide 4 of the two studies that have been conducted, the Phase III studies that are part of our global registration plan. And having a robust data set at this point as we do, really puts us in very good position for global approvals of migalastat monotherapy for the patients with amenable mutations.
Just to remind everyone, first Phase III study shown here is the FACETS study. And this is a study in which 67 patients who are naive to ERT were enrolled. The first six months were placebo-controlled. We have those data. And the primary endpoint was the reduction in substrate histology in the kidneys, looking at kidney GL-3 at six and 12 months.
And the important secondary endpoint was the kidney function, which was measured both by estimated eGFR and measured GFR, mGFR, at 12 and 24 months. And these are data that we had, of course, presented before, and I'll be discussing a little more of that shortly.
On the bottom of the slide, the ATTRACT study, our second Phase III study. And this was a study that enrolled 60 ERT experienced patients. And 36 were switched to migalastat, 24 remained on ERT. And the primary endpoint that we looked at here was comparability of migalastat to ERT based on the eGFR and the mGFR over 18 months. And, of course, as we had mentioned previously, that that study did meet the primary endpoint, showing comparability to ERT on both measures of renal function.
On slide 5, we have some specific data that further validates the assay, the GLP HEK assay that we developed to identify patients who have amenable mutations. Very briefly, what you're looking at are individual patient data for their interstitial capillary GL-3. And the amenable patients are in blue; non-amenable patients are in red. One can easily see looking at this slide that the amenable patients had decreases from day-five in their GL-3, and the non-amenable patients had no change or increases in their GL-3.
So, clearly, the assay is effective in distinguishing patients who have amenable mutations. And this is a key part of our regulatory strategy as well. So having these data are able to support that point in a very strong way.
In terms of the functional outcome -- and these are to slide 6, the data we presented at ASHG in October for long-term kidney function. So we're able now, since patients had continued from Study 011, the FACETS study, into an extension study, an open label extension -- and patients are continuing as we speak -- we can measure their kidney function over longer and longer periods of time. And at the time of the ASHG meeting, we could report on data for 32 months, almost 3 years of renal function data. And the longer-term data is even more meaningful.
Of course, kidney function decline is a major cause of morbidity and mortality in these patients. And one can see from the results for EGFR by two methods, CKD-EPI and MDRD, that the change in GFR was negligible over a 32-month period. The stability of the kidney function compares very well to what is in the literature for Fabry patients.
And as Dr. Bichet, who is a nephrologist expert in the field of Fabry disease, says in the quote that you can see, that if you are able to look at patients with similar levels of proteinuria, you can see that the migalastat-treated patients are more stable than the untreated comparative patients from the literature. So, these data are very strong for the long-term kidney function. And so that study, open label extension is continuing, as we gather more and more data to support that.
The next slide, 7, shows highlights of what is upcoming. And these are the Study 012 results; the very high level as presented -- mentioned previously. But we are able to present the data in some detail. And it includes, of course, the kidney function data as well as new data on cardiac endpoints, as well as other important secondary endpoints.
It's going to be a poster presentation; you can see the information there at the American Society of Nephrology meeting in Philadelphia. And we are very -- and it will be presented by Dr. Kathleen Nicholls, who is an investigator and expert who has been working with us for years. And we are very excited to be able to share that data with you in just a little over a week.
The next slide, slide 8, really puts together the patient experience that we have at this point with migalastat, which is very extensive in the rare disease setting. There are 97 patients today who are on migalastat as their only treatment for Fabry disease. And the total patient years of therapy is 377, very substantial. And the maximum years on therapy right now is over 8.5 years and continuing. So patients have been on this treatment after only specific therapy for Fabry disease, for a very long time. And the fact that the retention rate into next-study is 96%, very high; shows the patient's satisfaction with the treatment and their response to the treatment.
So all of these data support the clinical trial data from our Phase III data in our regulatory strategy. And that's what's highlighted on the next slide, slide 9, which shows, in both US and Europe, how we are pursuing the fastest paths to have approval for migalastat in these regions.
Starting on the right side in Europe, the centralized procedure is underway. And as John had mentioned, a pre-submission meeting is occurring this quarter. The regulatory pathway is very clear, and based primarily on non-inferiority or comparability to ERT in Study 012, which has been demonstrated, as I said, using both of the co-primary endpoints. And this is all leading towards the MAA submission in 2015. So it's on track.
In the US, we now have, as I said, the robust data set from both of the Phase III studies as well as the long-term clinical trials data; eight-plus years in the extension studies. And all of these data, across all of the endpoints, are going to be discussed in a meeting with the FDA in early 2015, where we will be discussing the regulatory path in the US. So that is very much underway as well.
With that, I'll turn it --
John Crowley - Chairman and CEO
Great. Yes, thank you, Jay. And again, just to emphasize the importance of the presentation next week at the ASN meeting, with cardiac disease being increasingly known to be a cause of mortality in Fabry and complications, something I think the data will be very, very relevant. So we will be eager to share that.
Let me turn the conversation now over to Dr. Hung Do. We're going to talk about our next-generation enzyme replacement therapy for Pompe disease. That's a program that's shown great progress. Hung will review for us some of the unique characteristics of ATB200, which is our next-generation ERT and Pompe, distinguished from some of the work we have done comparing that to Lumizyme, both in the analytical and in vitro setting, as well as the in vivo setting.
So I'll turn it to Hung. And then Brad will highlight some of the advancements this quarter in that program that keep us very much on track.
Hung Do - SVP of Discovery Biology
Great. Thank you, John. As a reminder to everyone, Amicus developed our own proprietary replacement enzyme therapy for Pompe disease designated as ATB200, as alluded to by John.
ATB200 was developed with optimal glycosylation for improved drug targeting. It has distinct glycosylation patterns from any existing GAA ERT to date. It contains significantly higher amounts of mannose-6-phosphate for binding the intended mannose-6-phosphate receptor. This receptor is known to be utilized for uptake in delivery of this enzyme to target cells.
As shown on slide 11, this difference in glycosylation and significantly better receptor binding is shown in these particular graphs. We perform multiple animal studies to evaluate ATB200 relative to Lumizyme. Based on a number of different studies, our data indicate that ATB200 is significantly better for improved uptake in target muscle tissues as well as the corresponding glycogen clearance. These data are highlighted on slide 12.
When in combination with the pharmacological chaperone, we see that the reduction clearance is further enhanced. We believe that our next-generation Pompe ERT has significant advantages over the current ERT. And we believe these data, up to this point, suggest that it should be a superior treatment for Pompe disease.
John Crowley - Chairman and CEO
Great. Thank you, Hung. It was an excellent summary. And I'm sure we will have some follow-up questions.
Let me turn the call over to Brad. And, on the next slide, slide 13, Brad, if you'd highlight the three-and-three strategy and the pathway to the clinic, and begin with a discussion building on Hung's presentation of where we are with the Pompe program.
Bradley Campbell - COO
Great. Thanks, John. So, as John mentioned on slide 13, we go -- do go through some of the key milestones that we have achieved and expect to achieve over the course of the year. I would like to expand on a couple of them here, because I think it shows the great progress we've made on this exciting program.
So, in the third quarter, we did, in fact, complete our manufacturing scale-up from the pilot clinical -- preclinical scale that we had at the beginning of the year, all the way to the scale that will be used to support both our IND-enabling tox studies as well as our clinical studies. And during the process, we're able to maintain those key quality attributes, in particular the mannose-6-phosphate content that Hung alluded to, which is so key to the superior attributes of this program.
We also got to seek scientific advice and, in fact, very recently, had a very positive meeting with the EMA, where we gained initial feedback on the program going forward. I'll share a couple of those details with you here now.
In particular, they did acknowledge that there remains a significant unmet need in Pompe disease, and they were supportive of our proposed clinical trial enabling tox program. So that means we are on track to start those IND-enabling studies at the end of this year. Importantly also- - and this is a question we've gotten quite a bit -- they did acknowledge that, given the rarity of Pompe disease and the proof-of-concept that we have already demonstrated, that our clinical study will evaluate the effect of the combination therapy, rather than needing to show the individual molecules, the chaperone and the enzyme replacement therapy and their impact.
So, very positive and very important feedback from the European agencies. We will seek US regulatory feedback as well, most likely sometime in 2015. We will give further guidance on that as we get closer. But I think very strong progress in the Pompe program, and very exciting for patients and for shareholders as well.
On the Fabry next-generation enzyme replacement therapy program, again, there, too, we've made great progress. We did complete our manufacturing activities. We also reported earlier that we had completed our IV migalastat study in healthy volunteers, where we look to learn more about the dose and dose selection to use in the next study in Fabry patients. However, we would reiterate at this time that we are holding start of that study in Fabry patients pending the outcome of several business development discussions.
We do plan on providing an update here in the fourth quarter on our next-generation ERT development strategy. And we will provide guidance there later in the quarter.
So, again, great progress in both of our key programs; continue to make good progress on our earlier enzyme replacement therapy programs as well, and look forward to providing further updates as they come. John?
John Crowley - Chairman and CEO
Great. Thanks, Brad. Excellent. So, Chip, I'll turn it to you for the financials.
Chip Baird - CFO
Great. Thanks, John. Good evening, everyone, I'll start today's financial discussion with a few comments about our current cash position and guidance. This is shown on slide 14.
As indicated in this afternoon's press release, Amicus continues to maintain a very strong balance sheet. At September 30, 2014, we had $85.2 million in cash and cash equivalents. This compares to $82 million at the end of last year. As a reminder, we significantly strengthened the balance sheet in second quarter, due to the successful execution of a $40 million at-the-market equity financing.
We began sales under the ATM in May, and sold the final block of shares in early July. In terms of additional sources of funding, we still have $10 million remaining available under the debt facility we entered into in the fourth quarter of 2013.
As previously guided, we expect full-year 2014 net cash spend to total between $54 million and $59 million. We believe that our current cash position is sufficient to fund our current operating plan into 2016.
Turning to our third-quarter 2014 financial results on slide 15, I'll be referencing tables one and two in the press release we issued earlier this afternoon. Additional details will be found in our Quarterly Report on Form 10-Q, which will be filed later this evening.
For the quarter, we recorded modest revenues of $293,000 compared to $39,000 in the year-ago period. Revenues were recorded in conjunction with the Biogen collaboration, which, as we disclosed last quarter, has since concluded. Total operating expenses for the third quarter of 2014 increased to a little over $17 million compared to $15.2 million for the third quarter of 2013. The year-over-year increase here was primarily due to increases in our Research and Development costs.
Net loss attributable to common stockholders in the third quarter of 2014 was $17.1 million, compared to a net loss of $14.6 million in the third quarter of last year. Net loss per share of $0.22 in the third quarter was narrower than the net loss per share of $0.29 in the year-ago period. The narrower net loss, net loss per share, versus the year-ago period is attributed to an increase in the share count compared to last year period. And as of September 30, 2014, we had approximately 79.3 million shares outstanding, and this compares to 49.6 million shares outstanding on September 30, 2013.
So this summarizes our key financials for the third quarter of 2014 as well as our 2014 full-year guidance. I'm happy to address any questions on the financials during the Q&A session, but for now, we'll turn it back to John.
John Crowley - Chairman and CEO
Great. Thanks, Chip. So, I have, again, never seen Amicus in a better position; we've never been busier. And there is a real palpable sense of excitement around the Company as these programs advance.
So, with that, operator, I'll turn it back. And we are happy to take questions.
Operator
(Operator Instructions). Ritu Baral, Cowen.
Ritu Baral - Analyst
Thanks for taking the question. And I apologize if you addressed it; I scrambled on when Jay was on slide 4 or 5. But how do you look at the data package that you have right now, in terms of how FDA might interpret this in the path forward in the US? What do you have right now that you think the Agency would find most compelling? And what may come out on the 15th that has a high level of meaningfulness for the Agency?
John Crowley - Chairman and CEO
Yes, I'll just comment briefly and then ask Jay to comment. You know, Ritu, when we last met with the FDA in the second quarter of 2013, we had a much more limited data set than we have today. So, in that meeting, the FDA said that they would need to see the 012 study results in addition to 011. We've now done that, and done that successfully. And they also said they would consider the totality of the data.
So considering where we were, we see that as great progress. And we think it will form the foundation of a very strong filing with the US FDA. We have -- we are more advanced, and I think there is a more clear path with the Europeans. And we've gone down that path immediately after getting the 012 data in the third quarter. And as Jay indicated, we will have that meeting this quarter, the pre-submission meeting with the European authorities. But we would expect it to follow very quickly in the first quarter with discussions with the US FDA on the path there.
So, we have a lot more data; it's much stronger data than the interim data set that we presented in the second quarter of 2013. So when it comes to showing the 012 data set, we feel very comfortable that that will support filing. And this whole notion of the totality of the data -- certainly, 011, 012, the long-term Phase II study data, as well as now this important data on the secondary endpoints, including the cardiac outcomes, that would be part of the filing.
So, Jay, if you have any color to fill in?
Jay Barth - Chief Medical Officer
I'd love to go into more detail about that, but some of it will have to wait until ASN. But as John captured, the totality of the data has only grown over time from what we had discussed initially. Of course, the focus had been and still is on the substrate reduction and stability of renal function. Those are very critical.
But with the additional data that has even supplemented what we had mentioned before, in terms of other endpoints, cardiac included, really makes the benefit/risk assessment clearly weighted towards the benefit of patients. And I think that really puts us in a good place when we talk to FDA. Agreed?
Ritu Baral - Analyst
Got it.
Jay Barth - Chief Medical Officer
Is that clear, Ritu?
Ritu Baral - Analyst
Yes. I guess we'll -- I'll ask this question again after the 15th when we've got the full data set. As far as your business development discussions, what's important for us to keep in mind in the deals that you may be pursuing? What do you want the most? And where do you want assets that you get access to, to take you?
John Crowley - Chairman and CEO
Sure. Again, here, I'll just comment briefly and then ask Brad to add more color. Right?
You know, you characterize it as deals we are pursuing; I would actually characterize it as deals and partners that are pursuing us. And it's a nice place to be in. After 011, and certainly 012, a number of multiple parties, all very capable organizations, approached us about partnering and commercializing migalastat either globally or ex-US.
Our intention right now is that Amicus has the capabilities to build a global infrastructure to commercialize migalastat. And until somebody can convince us that we can do it better partnered with them than we can do alone, in terms of our mission to deliver the drug to patients, and to create and maximize shareholder value, that's where we are.
We are entertaining those discussions. Some of them are more intense, of course. But right now, we have a go-it-alone strategy that we think we have the capabilities and the experience globally to sell that drug. With that, we've also had people interested in a broader Fabry franchise that would include the combination therapy and a next-generation enzyme therapy in Fabry.
We've had people approach us with broader interest, including Pompe and the platform. And Pompe is one we really feel strongly that we need to take that into the clinic. So, we've been less open to those overtures. Obviously an extraordinary deal is something we would consider; but again, with the standard that somebody would have to convince us that it fills enormous shareholder value, and gets the drug to patients sooner and better than we can do alone.
So, Brad, I'll let you -- if there's anything to add.
Bradley Campbell - COO
No, I think that was a very complete response, John. I think that's exactly what I was thinking about. The good news is, as John said, we have a lot of options at the table; the bar is high for doing any of those deals. But the key will be whether we can increase the value for shareholders and for patients. And that's the yardstick we'll use to evaluate what we have in front of us.
John Crowley - Chairman and CEO
I find, Ritu, you're either popular in this business or you are not. There's not a lot of middle ground. And it's nice to be in the popular category.
Ritu Baral - Analyst
Got it. Thanks for taking the questions, guys.
John Crowley - Chairman and CEO
Thank you.
Operator
Anupam Rama, JPMorgan.
Anupam Rama - Analyst
Hey, guys, thanks for taking the question.
John Crowley - Chairman and CEO
Welcome back to JPMorgan, Anupam.
Anupam Rama - Analyst
Oh, thanks. Yes, thanks a lot. I had a couple -- a question on the three-and-three strategy. It sounds like we're going to get an update on Pompe disease and Fabry disease by the end of the year. I was wondering, by the end of the year, if we might get an update on MPS I as well, just in terms of what you guys have been doing on that program? Maybe some preclinical data sometime in 2015 on what an enzyme kind of looks like. Just wondering what's going on behind the scenes on that program. Thanks.
John Crowley - Chairman and CEO
Yes, I would probably look to your conference in January as when we would have a more robust discussion around that program. There's some important work going on right now in the Company. But we've got so much news between now and the end of the year in our monotherapy migalastat program, and in the Fabry and Pompe next-generation ERT programs, I think we will hold the MPF data to the early part of the year.
Anupam Rama - Analyst
Okay, great. And also can you give us an impression of what you hear from physicians about enzyme replacement therapy in Fabry disease in terms of cardiac improvement? The literature we've looked at seems a little bit mixed. And I just wanted to hear some perspective from you guys.
John Crowley - Chairman and CEO
Yes, Jay, please.
Jay Barth - Chief Medical Officer
I think you characterized it well for the literature on ERT and cardiac. There isn't a lot of data to support it; there may be some effect of the ERT on the heart, but it's not a good, really ideal treatment for the cardiac aspects of the disease. And that's something we feel is a need that needs to be met, that there's room for improvement on what ERT can offer in the realm of the cardiac involvement with Fabry disease.
Anupam Rama - Analyst
Thanks for taking our questions.
Operator
Joseph Schwartz, Leerink Partners.
John Crowley - Chairman and CEO
Hello, Joe? If you're talking, we can't hear, I'm sorry.
Joseph Schwartz - Analyst
Muted. But anyway, congrats on the progress. I was wondering, first of all, if we could talk a little bit more about the Pompe ERT program for ATB200? And, in particular, I know it's been a challenge for other companies, so just curious about how you are feeling about the scale-up and expression levels of that enzyme? And then what are you thinking about at this point for the design and endpoints in the Phase I/II study to start next year?
John Crowley - Chairman and CEO
Yes. But just on that latter point, Joe, we are not yet prepared to talk about the clinical aspects of that program. The EU meeting that Jay just got back from a few days ago was focused on preclinical CMC toxicology, and we got great feedback. And I was particularly struck by just how explicit the European regulatory authorities were in noting the unmet need on multiple levels with the existing standard of care.
So we are not yet ready to talk about our clinical strategy. We are still working with some experts on that.
But, Brad, if you'd talk about how well the scale-up has gone and the characteristics of that.
Bradley Campbell - COO
Sure. So, Joe, it's a great question. And, as you might recall, John and myself and Hung were all at Genzyme, and so we lived some of the earlier days. And Dr. Do and (multiple speakers) were there at Novazyme, previously working on enzyme replacement therapy for Pompe. So a lot of experience here in that process and understand full well the challenges there.
So I think part of it is just backing it with the benefit of experience. I could tell you that while it's taken a lot of hard work, and a lot of long nights, and a lot of trips back and forth to China, we have been able to successfully scale up to the scale necessary to support our clinical development in the early years here. So that went very well.
As I mentioned during the call, we were able to maintain the carbohydrate structure that is so critical to the uptake of Pompe enzyme replacement therapy. And that's both in terms of carbohydrate structure and also the M-6-P content specifically. So those are things that we knew to look out for, and design the processes specifically to be able to control that. And we were able to successfully do it.
So, not easy for sure. But I think built on years of experience, and also the technology and know-how that Dr. Do and some of his colleagues brought along with Callidus, was certainly a good starting point. So, we feel like we are in a good position, and we are on track to start those studies this year.
John Crowley - Chairman and CEO
Yes, I mean, Joe, that was the greatest risk this year, was choosing the right cell line. We knew by the second quarter that we had an excellent cell line, but we also knew from the experience that we had to scale it. So we just focused like a laser and put the best people around it. And I think we're very, very pleased with these manufacturing reports we've gotten in the last couple of weeks, that we are now at scale and we've done it successfully.
And you're right. I think it's distinct from other experiences over time with other people who have sought to make this enzyme at larger scale. So I think we've got a very, very special product here, and we are eager to get that into patients.
Joseph Schwartz - Analyst
Okay. Sounds encouraging. Could I also ask, I know you've got the data coming in a couple of weeks, but wanted to -- and don't want to steal your thunder, but can you talk a little bit about the cardiac disease measures that you've captured, just broadly, what they are? And if there's natural history data there that you can point to, to further differentiate migalastat monotherapy?
John Crowley - Chairman and CEO
Yes. No, of course. Jay, I'll let you comment on what the (multiple speakers) --.
Jay Barth - Chief Medical Officer
Yes, I could talk about the methodology that was used in assessing the patients. It's very rigorous eco-cardiography that was performed and done in a centralized procedure in a blinded fashion. So we know that the quality of the data was high.
We looked at a number of parameters. The most important one in this disease is left ventricular mass, because cardiac hypertrophy is a characteristic finding in these patients. So the left ventricular mass, or LVM, LVMI, Left Ventricular Mass Index, are really the important parameters to focus on.
And I think that's the kind of data that we'll be able to discuss at the time. The natural history is quite well-characterized in patients in that they have gradual progressive increase in their cardiac size in their left ventricular mass. So that's a well-recognized phenomenon in Fabry patients. And really can interpret our data in light of what's known in this natural history for the cardiac disease in Fabry.
Joseph Schwartz - Analyst
Okay. Thanks very much.
Jay Barth - Chief Medical Officer
You're very welcome.
Operator
Thank you. At this time, I'm showing no further questions. I'd like to turn it back to you, Mr. Crowley, for any closing remarks.
John Crowley - Chairman and CEO
Great. Thank you, operator. Thank you all for joining. We've got a lot more news to share yet here before the end of the year. So, stay tuned. Thank you very much.
Operator
Ladies and gentlemen, thank you for your participation in today's conference. This concludes the program. You may now disconnect. Everyone have a great day.