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Amicus Therapeutics, Inc. (NASDAQ:FOLD)

Phase 3 Fabry Monotherapy Study (Study 011) Results Conference Call

April 29, 2014 8:00 AM ET

Executives

Sara Pellegrino – Head-Investor & Media Relations

John F. Crowley – Chairman & Chief Executive Officer

Jay A. Barth – Chief Medical Officer

Bradley L. Campbell – Chief Operating Officer

William D. Baird – Chief Financial Officer

Analysts

Ritu Baral – Canaccord Genuity, Inc.

Edward H. Nash - Cowen & Co. LLC

Anupam Rama – JPMorgan Securities LLC

Kim Lee – Janney Montgomery Scott LLC

Joseph P. Schwartz – Leerink Partners LLC

Operator

Good day, ladies and gentlemen, and welcome to the Phase 3 Fabry Monotherapy Study Results Conference Call. At this time, all participants are in a listen-only mode. Later, we’ll conduct a question-and-answer session, and instructions will follow at that time. (Operator instructions) As a reminder, today’s conference call is being recorded.

I would now like to introduce your host for today’s conference, Ms. Sara Pellegrino, Director of Investor Relations. Ms. Pellegrino, please go ahead.

Sara Pellegrino

Thank you. Good morning, everyone, and welcome to our conference to discuss our positive Phase 3 Fabry Monotherapy Study Results from our Study 001. I’ll remind everyone today that this presentation 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, ongoing collaboration and the timing and reporting of results from clinical trials evaluating Amicus’ candidate drug products.

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 this cautionary statement and Amicus undertakes no obligation to revise or update this presentation to reflect events or circumstances after the date hereof.

And now it is my pleasure to turn the call over to Amicus Chairman and Chief Executive Officer, John Crowley.

John F. Crowley

Great. Thank you, Sara. Good evening, everybody, and we are certainly very excited to be able to share these important and very positive data set with you here this morning. We think it sets a very solid foundation for a pass forward for the migalastat monotherapy program in Fabry disease.

Let me begin on Slide #3 with just an overview before I hand it over to our medical team to review the data itself. So, I’m just going to summarize the 12 months and 24 months data set, the finding hoc from the banner migalastat demonstrated a statistically significant and a durable substrate reduction on the 12-month pre-specified primary analysis in Fabry patients with amenable mutations.

If you see the first cohort that subjects to originally for the six months of the study Stage 1 on placebo when they switched to migalastat after six months on migalastat treatment, they demonstrated a statistically significant reduction in kidney interstitial capillary GL-3 at month 12. Again, this is a pre-specified primary analysis of the study you see the p-value there at 0.013.

We also continued to follow and biopsy the patients who are on migalastat for the first six months of the study, who continued for further six months, so 12 months total at the end of that Stage 2. Those patients and you will see the data here showing graphically shortly in the slide is very clear. Those patients continued to show a persistent and durable reduction in the kidney interstitial capillary GL-3. As you know, we measured a number of other endpoints in the study beyond the primary, one, that we think is important and have increasing important to key opinion leaders and investigators in the field is plasma lyso-Gb3.

In those patients who switched from placebo to migalastat, we showed a statistically significant reduction in that important biomarker of disease. And you can see very statistically significant at p-value of 0.001. Also, significant as we continued to follow the kidney function on these patients to the end of the study, the full 24-month period in this Phase 3 study, we show that kidney function both on the measures of ease estimate eGFR, estimated glomerular filtration rate, as well as iohexol, measured GFR. Those patients remain stable in the 18-month to 24-month period.

Our safety continues to look very good migalastat was generally safe and well-tolerated we had a separate slide to go through a safety summary. And then just as we look at patient compliance and patient experience on the drug of the 41 patient in the study with the HEK amenable mutations who completed the entire 24 month study period, 35 of those 41 patient or 85% remain in a voluntary extension study.

We have had a chance over the last couple of days as the data came in, and was thoroughly checked to that it with the number of the experts in the field, confidentially and Fabry disease. We could have put many quotes in this press release. We chose of a quote first from Dr. Raphael Schiffmann at Baylor Research Institute, Dr. Schiffmann was one of the very first investigators to begin working with us to study migalastat as a monotherapy in patient, and we very much appreciate as work and perspective and certainly his comment has reflected in the press release.

And then also you see a quote here importantly from Dr. Robert Desnick, Dean of Genetics and Genomic Medicine, Dr. Desnick was not an investigator in that study, but we have reviewed these data with Dr. Desnick and Dr. Desnick is all for the inventor of Fabrazyme, so tremendous experience over 40 years of working experience. And I think he spoke these volumes to that the data that we see today.

With that as an introduction, let me go through who is in the room with me here today. I had Dr. Jay Barth our Chief Medical officer, Chip Baird, our Chief Financial Officer Bradley Campbell, Chief Operating Officer; and we are also joined by Dr. Jeffrey Castelli, Vice President Program Management. So with that Jay I’ll turn it to you to run thought the study results.

Jay A. Barth

Thanks, John. I’ll begin on slide four, looking at the reviewing the study design of the Phase 3 study those divided into Stage 1 initially. A six months placebo-controlled days and this was the original period of the study that has been previously reported I’ll review that shortly. 67 patients were enrolled with their one-to-one randomization, the migalastat 150 milligrams every other day and Placebo.

And the primary end point in that six month period was kidney interstitial capillary GL-3. Then patients who into Stage 2, which was an open-label period for six months and which all patients received migalastat. And the primary end point at the end of this Phase was the 12 months looking at kidney interstitial capillary GL-3. Subsequent to that patients continued in an optional open-label extension for an additional 12 months. And we will be reporting to you clinical data up to 24 months of treatment duration in the upcoming slide.

The next slide 5 shows the results that they were originally reported from the Stage 1 and this was from December 2012, in which the pre-specified primary endpoint was a responder analysis for a kidney ICGL-3. Response was defines as the restate 50% reduction from baseline in ICGL-3. And we saw a 41% of patients on migalastat had achieved the response and that was inline with expectation somewhat surprising at the time was the very high placebo response rate, which confounded the results.

And we understand that now due to the fact that the quite a number of the patients had very low baselines and with a very small absolute decrease actually reached the response definition. And with a lot of discussion both internally with external experts with regulators as well is become apparent that the best way to look at kidney interstitial capillary GL-3 in our patient population it is not through this type of responder analysis, but through a continuous variable looking at main change from baseline.

And the slide six that looks adds this analysis these data represented at the recent world meeting. As a continues stable on main change from baseline in Kidney ICGL-3 and those apparent in the blue line as that the patients who received migalastat during that first six months had a significant decline in Kidney ICGL-3. As you can see that in the blue line versus the placebo treated patients in the dashed red line, the p-value for this difference is P.008. We predefined success in page two as the 12 months data for patients who switched from placebo to migalastat, and that’s reflected in this slide. The patients who are on placebo in the first six months in the red dash line now on migalastat for the subsequent six months, that’s the blue dash line experienced a significant reduction in kidney IC GL-3.

John F. Crowley

Just to highlight Jay, you are on Slide 7.

Jay A. Barth

Yes, thanks. Yes. And this reduction has been in these patients who switched from placebo to migalastat is almost exactly the same as the reduction seen in patients who received migalastat over the first six months, replicating the results from Stage 1 and the p-value associated with the patients switching from placebo to migalastat is 0.013 statistically significant.

At the same time the patients who are on migalastat for the first six months, so looking at Slide 7, who are on migalastat for the first six months and remained on migalastat for the remainder of the 12 months had a persistent reduction, the effect that was seen in the first six months was durable. So we feel these results are very important in showing the effect of migalastat on its substrate in the kidney, and it’s been replicated potentially twice in the regional migalastat-treated patients, as well as those who switched from placebo to migalastat.

Next slide, John had mentioned, what has become recognized as a very important biomarker in this disease and responsive treatment, which is the plasma lyso-Gb3. And these are new results for Stage 1 and for Stage 2. We’re able to see in Stage 1 the patients between baseline and month six, in the solid blue line, the patients who are on migalastat for the first six months had a significant reduction in plasma lyso-Gb3 p-value 0.0033.

And as seen for the kidney IC GL-3, same pattern for plasma lyso-Gb3, the patients who are on placebo for the first six months in the red dash line and then switch to migalastat for the next six months, the blue dash, those patients experienced a significant decline in plasma lyso-Gb3, p-value less than 0.001.

And as with kidney IC GL-3, the patients who are on migalastat for the full 12 months had a durable effect, a reduction that was shown in the first six months persisted and continued for the entire 12-month period of time. So it’s very important to have been able to show the reduction in substrate both in the kidney and in plasma lyso-Gb3 demonstrating the effect of the drug.

Slide 9, is a representation of the kidney function that was looked at in this study. And we have the ability to analyze these data over 18 months for patients who are on placebo for the first six months and 24 months for the patients who are on migalastat for that entire duration. And these results shown as with the previous results for IC GL-3 and for plasma lyso-Gb3 in the patients with amenable mutation showed stabilization of GFR over this period of time up to 24 months.

Renal dysfunction is a major cause of morbidity and mortality in Fabry disease. And looking at the GFR, the renal function in three ways, the eGFR through CKD‐EPI and MDRD, as well as measured through iohexol, GFR showed stabilization of the renal function over this period of time up to 24 months on treatment.

This is very much in contrast with what is known about Fabry patients who are not treated where there is a significant decline over time. And even in healthy individuals depending on age, healthy people may experience declines of 0.5 to 1 per year declines, of course, much greater in Fabry patients, and it’s very encouraging therefore to see the stabilization in renal function over a period of up to 24 months.

The next slide is a summary of the safety data, demonstrating on migalastat has been previously was generally safe and well tolerated.

You can see for the common adverse events that are represented here, those experienced by at least 10% of subjects. The adverse events were similar in frequency for migalastat and placebo in the first six months. And then subsequently in the Stage 2 and in the open-label extension up to 24 months, there are really no new safety signals identified in the patients who received migalastat up to 24 months, confirming the favorable safety profile of the drug.

John F. Crowley

Great. So just a comment on the global regulatory strategy, Jay, if you want to take us through that as well in Slide 11.

Jay A. Barth

Sure. There are two, of course, main regions in which we’re focusing in terms of the regulatory strategy in the U.S. and in Europe. In the U.S. in speaking with the FDA, the approach that we are taking is one, of course, as often the case in rare diseases.

Looking at the totality of the clinical data, we now have over eight years of data and extension studies on patients who’ve been receiving migalastat. And with these data from 011 is first Phase 3 and the data from Study 012 that are expected later this year. We will have a data package of these two Phase 3 studies that will be the basis of discussions with the FDA. And we’re very much looking forward to continuing that discussion based on those data.

In Europe, we have a clear regulatory path that is based on the data that we have accumulated to-date and the upcoming Phase 3 Study, Study 012, in which the endpoint is non-inferiority to ERT. And this is an approach that is expected to satisfy the EMA and with the data from Study 012 will be able assuming success in that study to proceed with a submission in Europe for approval of migalastat in Fabry disease.

John F. Crowley

Thanks, Jay, that was an excellent summary. Thank you. And, again, for many of you on the call, this is the first time you’ve heard Dr. Barth go through any clinical results here at Amicus.

So this is a wonderful way for you to start here, 10 year as Chief Medical Officer. As a reminder, Jay joined us in March as Chief Medical Officer having most recently come from PTC Therapeutics, where he served as Senior Vice President of Clinical Operations and Research. So, Jay, excellent summary, again, and thank you.

Maybe just on Slide 12, Brad, if you would comment a bit more on patient experience as you’ve involved in this program for many years now.

Bradley L. Campbell

Sure, I think, this is a slide we’ve shown before, but taking into context of the data from 011 Study that Jay just presented, the fact that almost 100 patients today take migalastat as their only therapy for Fabry disease. Many of these patients for more than two years now, some for up to eight years, we think speaks volumes to help physicians and patients are experiencing their – the effects of migalastat.

And we’re really excited to see the outcome of the 012 Study. And we think this is a great day and a great step forward for what we hope to be further advancements for the drug from migalastat and set us up well for conversations with the regulators later this year. And Chip now will walk us through some of the milestones we have coming up.

William D. Baird

Sure, thanks, Bradley. So as you can see on Slide 13 today’s results, our important milestone in a presentation directed development program that came back in late 2009. Today’s results are testament to the hard work and persistence of our development teams, as well as our investigators, study coordinators, and of course, with Fabry patients as stated in this study.

Looking forward the next milestone program is top line data from Study 012, the second Phase 3 study. And as Jay mentioned, the primary measure, there is GFR. We are on track with Fabry’s results every quarter of this year. It’s probably very exciting day for Amicus and for the broader Fabry community.

And with that, I’ll turn it back to John for any closing comments or questions.

John F. Crowley

Great. Thank you, Chip. So in need we still have some important events ahead of us that puts us a significant milestone for Amicus and to this program. Again, the chaperone monotherapy program was the foundation for what Amicus together a number of years ago. It has been a long journey with this molecule. It is a very unique mechanism of action distinct them away, again it’s an oral therapeutic option for Fabry disease. We think it’s an important advancement in the field also due to the extent that we believe we can well identified patients with response mutation would be suitable to this therapy. We also think it’s a very important used to pharmacogenomic information in personalized medicine in the field of rare diseases.

So to highlight as well, this is also the largest Phase 3 ever conducted in Fabry disease and we think very significant as well. There is a lot to be said for persistent in this field, we have always believe that this medicine is safe and effective for patients of this data set, I think very convincingly shows that. So, we’re very pleased that we’re able to share this data with you today we think it’s very important for Amicus for our shareholders and most importantly for people living with Fabry disease.

With that operator we’re happy to take any questions.

Question-and-Answer Session

Operator

Thank you very much. (Operator Instructions) And the first question is from Ritu Baral of Canaccord. Please go ahead.

Ritu Baral – Canaccord Genuity, Inc.

Good morning guys. Thanks for hosting the call on this. One question and then follow-up, what does the – what terms is the control for the new P value that you presented on 12 month data. So basically, the people who switched over to the divisions are switched over of their own controls on that P value or with their another control form?

Dr. Jay Barth

No. That’s right, this is Jay the – it was the patients switching from placebo to make and that’s the basis of P value for the point 013.

Ritu Baral – Canaccord Genuity, Inc

As a follow-up did you down the responder analysis on the new data set?

Jay A. Barth

We have not.

John F. Crowley

No, we didn’t for two as we indicated based on the data that we saw in interim Stage 1 analysis, the meetings we had after that with investigators. And importantly the meeting with FDA we all agree that given this patient population, but that wasn’t an appropriate measure to look for surrogate biomarker response.

Ritu Baral – Canaccord Genuity, Inc

Got it. Just one more in, can you take us through the meaning of the new placebo marker on some of the slides, it’s one that I’m not familiar with?

John F. Crowley

Yes. It is, its one where there is number of publications and we’re happy to put those out there. It is a very important disease biomarker increasingly recognized and utilized by physicians. And Jay, I let you comment on specifically what that is and then maybe Dr. Jeff Castelli can add some through the color to it is Jeff is work closely with that so Jay?

Jay A. Barth

I’ll start, it has become increasingly recognized these four biomarker in Fabry diseases, and that Plasma Lyso-Gb3 meet the characteristics for that. There is an increasing amount of data that has come out showing that Plasma Lyso-Gb3, which is in the sense of variance of GL-3 that those responsive to treatments, but there is reduction experienced over a relatively short period of time. And that this effect can persist overtime as well. There is also data that is starting to the accumulated as well showing its importance overtime in terms of predicting outcomes in patients with Fabry diseases and Jeff, if you could expand on it?

Jeff Castelli

Not much expand on it, I think Jay as pointed out, there is the new biomarker and Fabry diseases when you speak to the KLOs and researchers and ask about biomarkers, it’s the one now that they really reference as best correlated with disease severity and the changes in treatment with ERT and now we’re seeing that with migalastat. And I think it was important in this study to see they’re correlated very tightly with the interstitial capillary GL-3. So measuring GL-3 by biopies and kidney correlated very well with this measure in blood as well.

Ritu Baral – Canaccord Genuity, Inc

Great, thanks for taking the question.

Jeff Castelli

Of course, thank you.

Operator

The next questioner is Edward Nash of Cowen and Company. Please proceed.

Edward H. Nash - Cowen & Co. LLC

Great, congratulations guys. Thanks for taking my call. I want to ask just how many order any patients between the six month and the 12 month time period that you found to be non-amenable that were pulled out that were that have gone through the first six months of treatment?

John F. Crowley

I mean, in terms of patients discontinuation.

Unidentified Company Representative

Correct. Those that you would not have considered to been responders, because they are not responder, but would have not been considered valuable, because they did not meet the requirements of being the inclusion criteria, they were potentially not HEK amenable. They went from 6 to 12, but they we’re, there you considered them to be HEK amenable from the zero to six-month timeframe. Does that make sense?

Edward H. Nash - Cowen & Co. LLC

Yes. They were – you mentioned in the press release there were 17 patients totaled who once we moved from the non GLP assay to GLP assay who redeem to be non-amenable and that was prior to the looking at the data of course.

John F. Crowley

And I would clarify that fully analysis that we presented today from Stage 1 and from Stage 2 are based on that same population of amenable patients. So the – it’s the same patients carry through from Stage 1 and Stage 2 in the results that we’re presenting.

Edward H. Nash - Cowen & Co. LLC

Okay, great. That’s well going forward, great. Thank you. And I’m just – I wanted to find out just kind of, have you had based on this data, have you had any interaction with the agency on this FDA?

John F. Crowley

We have not yet. We have not shared this with the FDA. We, of course, have a continuing dialogue with them, at the Type C Meeting last year, they were very hopeful in our – putting together the revisions to the statistical analysis plan that included this primary specified analysis Edward. So as we think about working with FDA in the future, we will, of course, have – I think a very robust dialogue.

The prior message to us was that they would like to see the totality of the data from Stage 1 and Stage 2 of the study, which we now have as well as the 24 months data, as well as the full data set from the 012 Study. So right now we are going on the assumption that they would still like to see the totality of the data from both Phase 3s, but were engaged in dialogue and if that changes, we’ll certainly provide an update.

Edward H. Nash - Cowen & Co. LLC

Great. Thanks a lot and congratulations.

John F. Crowley

Very welcome.

Operator

The next question is from Anupam Rama of JPMorgan. Please proceed.

Anupam Rama – JPMorgan Securities LLC

Hey, guys, congrats on the data and thanks for taking my question. I was hoping to begin to Slide 9 just a little bit. Can you remind us of Study 012, what the primary GFR measure would be? And then in terms of looking at this data set, what extrapolations or re-throughs can you make to that Study 012 Study? Thanks.

Jay A. Barth

Yes, very good question Anupam, it’s Jay, that you feel that. In 012, as in 011, we are looking at all methods of GFR that are showing here, it’s actually GFR, I had so on the two EFR methods, the specified primary analysis that we are – have at this point for the 012 data and GFR, but I would say that all of the GFR measurements are important in looking at renal function.

And the – seeing these results, which are very encouraging over 24 months, 18 months to 24 months, showing that renal function is stable and these patients would be expected to decline because of the Fabry disease. Does make it seem that in 012 we would be looking for similar results in the 012 study, of course, we’ll have the ability to compare patients, and as patients who are continuing on the ERT to those who are switched to migalastat over 18 months. And we will be able to see directly in comparison in 012, how the renal function on migalastat compares to the ERT-treated patient. But these data from 011 all breaking to 24 months are certainly very encouraging in terms of showing stabilization of renal function.

Anupam Rama – JPMorgan Securities LLC

Great. Thanks for taking our questions.

John F. Crowley

Of course, thank you, Anupam.

Operator

And the next question is from Kim Lee of Janney Capital. Please go ahead.

Kim Lee – Janney Montgomery Scott LLC

Good morning, and congrats also on this positive data. Just a quick question on the plasma Lyso-Gb3, is this a surrogate that were agreed upon by the FDA in your conversions for Gb3, can you remind us that. Thanks.

John F. Crowley

Given that it wasn’t the primary specified analysis, we didn’t have any discussions with FDA, this was the secondary or exploratory endpoint. And I think it’s important Kim, this really when we began this study, plasma Gb3 wasn’t used, they are accepted as a endpoint. Between 2009 and today, there have been a number of publications using this all in the ERT field actually so using this endpoint. And I think it reflects an evolving understanding of the disease and the need for non-invasive biomarker of the disease.

So, again, if this was the only thing that looked encouraging plasma lyso-Gb3, we might ask a lot more questions. But it seems entirely consistent with the GL-3 that we measured by the kidney biopsies, which is very important correlation, as well as, as Jay just indicated the very encouraging stabilization of kidney function out to two years.

Bradley L. Campbell

Kim, this is Brad. One of the thing that I will add is that, there is a recent article that Barrons has released looking at the outcomes of switching back and forth between Fabrazyme and Replagal during the shortage. And as you might expect, kidney biopsies were not really an appropriate way for them to measure those outcomes. But in the absence of clear kidney function data, the data that Barron’s used to show the impact between that switch was Lyso‐Gb3. And, in fact, it show that when patients went to a lower dose of Fabrazyme or Replagal there, their Lyso‐Gb3 performed worse than when they are on the full dose of Fabrazyme. And so, as John said, this is an important correlation then between the IC GL-3 and the Lyso‐Gb3.

Kim Lee – Janney Montgomery Scott LLC

All right. Thanks for that clarification.

Operator

The next question is from Joseph Schwartz of Leerink Partners. Please go ahead.

Joseph P. Schwartz – Leerink Partners LLC

Hi, thanks, and congratulation as well. I was wondering if you could put the absolute magnitude of kidney interstitial capillary GL-3 reduction in clinical context either in terms of the baseline levels or anything else that might be helpful to understand the clinical relevance?

And then as my second question, I was wondering we didn’t see a continued reduction from the six-month to the 12-month period, but it was shown to be durable. I was wondering if you had any rationale for why that might have been the case?

Bradley L. Campbell

Yes, Joe, we’ve added all of that with the experts over the last week or so and a lot of the investigators in the field. On your last point it seems that there is a fuller effect if you can drive down to GL-3 to a point where you either just can’t measure it further or it sets the floor of what the treatment period would be. So that’s where we think it’s very significant and positive that the people, the patients who showed that reductions in six months continued to maintain that baseline or floor effect.

To the first part of your question, we had a wide range of patients with Fabry disease that we think it’s very representative of the actual troop of Fabry population. They were many patients, many males in particular, who were quite symptomatic with Fabry disease. Some of these patients had previously been on ERT and then removed for a period of time. So, Jay, I let you comment further on the clinical relevance to Joe’s question about at both baseline, what we saw this population and of course, the relevance and reduction in GL-3.

Jay A. Barth

We already know from Stage 1 that this patient population had relatively low levels of kidney IC GL-3. And, in fact, that is fairly confirmed by fact that over the second six months of treatment, we did hit the floor effect that patients really had gone to such a low level that would not possible to measure for the reduction or it was not being possible to even reduce it further, because their levels so low.

Nevertheless, we are able to show actually an increase in the rotations who had no inclusions in their capillaries. The – and the clinical relevance of it, I think is reflected by the fact that we saw stabilization in the GFR. So along with the reductions in kidney IC GL-3 along with the reductions in plasma lyso‐Gb3, we saw that over the 24-month period that GFR was stable. And I think that’s an indication that you we have a clinically relevant effect to the substrate reduction. That is something that, I think we find to be very encouraging going forward as we move into the next phase and looking at the future looking for Phase 3 results from the Study 012 as well.

Joseph P. Schwartz – Leerink Partners LLC

Okay great. Thank you.

John F. Crowley

Thank you, Joe.

Operator

I’m not showing any further questions in the queue. And we would like to turn the conference back to Mr. John Crowley for any further remarks.

John F. Crowley

Great. Thank you, Operator. So, again, we think this is a very important and positive data set and sets the strong foundation for a pass-forward for migalastat as a monotherapy for patients with amenable mutations in Fabry disease. We also think it’s one very important part of the two-part strategy of addressing the entire Fabry population.

We are filtrations with amenable mutations, we believe that migalastat monotherapy would offer an important new treatment option for patients with these mutations may take this as a lifelong therapy could treat their Fabry disease without the need for any enzyme replacement therapy. For the more than half of the population though that do not have amenable mutations, we continue to advance our next-generation proprietary enzyme replacement product that incorporates in its formulation the migalastat chaperone therapy to improve uptick in target patient itself.

So part of a Fabry franchise with a monotherapy in the next generation ERT, and of course, we continue to advance the CHART platform and Callidus technology in combination as we move forward into the clinic next year with our next generation ERT. But obviously the focus of this call was on this very important data set with Fabry monotherapy. And we think hopefully, there is a very bright future for this medicine as an option for patients.

So thank you all for listening. Have a great day.

Operator

Ladies and gentlemen, thank you for joining today’s presentation. This does concludes the program and you may all disconnect. Everyone have a wonderful day.

John F. Crowley

You too. Thank you.

Operator

Okay.

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