Amicus' CEO Discusses Q1 2013 Results - Earnings Call Transcript

| About: Amicus Therapeutics, (FOLD)

Amicus Therapeutics, Inc. (NASDAQ:FOLD)

Q1 2013 Results Earnings Call

May 9, 2013 5:00 PM ET


Sara Pellegrino - Director, Investor Relations

John Crowley - Chairman and CEO

Chip Baird - Chief Financial Officer

Bradley Campbell - Chief Business Officer

David Lockhart - Chief Scientific Officer


Anupam Rama - J.P. Morgan

Joseph Schwartz - Leerink, Swann

Greg Wade - Wedbush

Kim Lee - Janney Capital


Good day, ladies and gentlemen. And welcome to the Amicus Therapeutics First Quarter 2013 Results Conference Call. 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 call maybe recorded. I would now like to introduce your host for today’s conference, Sara Pellegrino, Director of Investor Relations. Ma'am, you may begin.

Sara Pellegrino

Good evening. And thank you for joining our conference call to discuss Amicus Therapeutics first quarter 2013 financial results. Speaking on today's call we have John Crowley, our Chairman and Chief Executive Officer; and Chip Baird, our Chief Financial Officer. Bradley Campbell, our Chief Business Officer; and David Lockhart, our Chief Scientific Officer are also on today's call and available to participate in the Q&A.

Today’s prepared remarks coincide with the slide presentation that is now available on our corporate website at The slides are located in the Investors section under Events and Presentations right below the webcast link to today’s call.

Turning to the slide two, you will find a reference to our Safe Harbor statement. 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, 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 this expectation 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, 2012.

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.

With that, it is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer of Amicus.

John Crowley

Great. Thank you, Sara, and good evening, everybody. So, as you see on slide three, I’ll go to the first two sections here. The update on the migalastat monotherapy program in Fabry, as well as the chaperone-ERT combination program, and turn it over to Chip to go through the details of Q1 financial results, and then I’ll come and handle the milestones, and certainly, as we always do turn it over to the Q&A.

Let me just begin by commenting that as you see the first quarter really was one where we focused on execution, primarily the clinical operations execution for migalastat monotherapy, as well as the regulatory strategy and interactions around migalastat monotherapy as well.

You saw from the press release that we issued that we have a Type C meeting now scheduled and in person meeting with FDA this quarter, that meeting is not yet occurred, although the briefing documents have all been submitted to the agency.

And again, with the CHART programs the company continues to evolve more and more in addition to our monotherapy program toward developing these programs, multiple program using the CHART or Chaperone Advance Replacement Therapy Platform Technology, the goal here of course to develop next-generation proprietary enzyme replacement products for the lysosomal diseases and potentially beyond.

So that’s the overview of what we are going to cover. Let’s go ahead and please turn to slide four, and again this is here for reference I’ll be providing some more statistics around these studies as well.

As we know that we have two Phase 3 studies for monotherapy in Fabry disease, Study 011 and Study 012. Study 011 is an ongoing Phase 3 study looking into the use of migalastat, given 150 milligram at the other day compared to placebo in patients with Fabry disease. It is a 12 month study.

As you know, we’ve taken earlier or late last year a look at the interim six month data or the Stage 1 data, that’s been announced, it’s been presented at the World Meeting back in February and we are reconfirming again on this call that the 12-month data is anticipated in Q3 of this year and this is a study intended to support the U.S. registration.

The original statistical analysis planned for this study of six months with yes/no responder analysis, and again that was looking at the number of patients who had a 50% or greater reduction in the interstitial capillary GL-3. The-six month interim data set favored migalastat versus placebo but it did not achieve statistical significance.

We presented back in February at the World Meeting some further detail through a platform presentation at that meeting. It showed a wider separation from placebo in the patients who had higher level of the interstitial capillary GL-3, notably those with 0.3 or above inclusion bodies.

This was an important analysis we think for explaining what happened and why we get above the noise of the BLISS methodology on this histological measure. It was however a post hoc analysis. It wasn’t predefined and it was in certain subgroups of patients.

We do think that was very important in understanding the mechanism of action, how the drug is working and we think very clearly showing in patients for whom there is measurable levels of the GL-3 substrate in the interstitial capillaries at the kidney, what actually truly going on and we think speaks to the effect of the drug is having.

As we’ve moved forward with the further analysis we had also presented originally and continue to evaluate what was a prespecified secondary analysis of this primary and that was looking at the overall burden of GL-3 in the kidney as a continuous variable in that same subtype.

Again, we didn’t separate out any patients, it all patients, 30 placebo, 30 active group and there we came very close to achieving statistical significance even on this interim analysis where we showed a median 41% reduction of GL-3 in the migalastat treated group versus 6% in placebo, the P of 0.093 and again this captured all patients with any level of GL-3, so no subgrouping what so ever.

Again to remind you we did see and this was presented at the World Meeting, a trend encouraging in terms of kidney function is measured by eGFR favoring migalastat over placebo. And again we continue to see a very strong safety profile for this drug with no drug-related SAEs, no withdrawal related to NAEs.

So that’s where we’ve been, we’ve spent quite a bit of time, it’s been an enormous effort at Amicus to get with our partners at GSK to look at this data in great detail, to back to review all of our Phase 2 data, we brought in quite few consultants to help us with some expertise in areas like biostastics for instant.

And now that we have the FDA guidance that we, they will consider the risk and benefit analysis of this drug and the totality of the data, we’ve been thinking quite a bit in preparing for and asked and what to asked the FDA’s guidance, in terms of what would define the totality of this data and what are the key areas to look at.

So as we get ready for the 12-month data in Q3 we stay, we are here today with still being blinded to that data, both Amicus and GSK. The six-month data is what it is and we think its every encouraging and we think it gives us based on our Phase 2 experience good reason to believe that for the group treated with placebo at six months that crossed to migalastat those patient with response limitations we would expect to see a drug effect and reductions, further reductions in their GL-3.

For those people who are in Stage 1 the interim analysis who were treated with migalastat for the first six months at 12 months we’d hope to see base again in our Phase 2 experience that the effect of the GL-3 or the effect of the drug AND the GL-3 would be to see a persistent and durable effect in a greater level of GL-3 reduction. Those are the key things that we’re looking for.

So we set about to ask a couple of months ago the FDA for further guidance, we requested a Type C meeting. The FDA granted that request. They didn’t have to. The FDA also granted that as an in-person meeting again to take place this quarter. It’s not yet taken place. It is one where we expect to be able to add to our statistical analysis plan not take away from and further define what is going to be success at 12 months, so we can communicate that to all of our interested outside constituencies.

So the key purpose of this meeting really is to allow us to get feedback for the agency on how we plan to analyze the totality of the data at six months and if the data looks consistent with what we hope for and again based on Phase 2 to allow us to move forward very quickly towards pre-MDA meeting in the second half of this year and again we continue to expect this top-line data in the third quarter of this year. So that’s where we are with the Type C meeting.

As we look at the migalastat monotherapy program, it’s very clear that we have a couple of important gates to get through this Type C meeting this quarter, the 12-month data release in the third quarter. Assuming we’re good on those two gates, we then move in the second half of this year towards the pre-MDA meeting. So very important point for the company; very important point moving forward with this 011 study as the study for the basis of regulatory approval, again seeking conditional approval on a surrogate endpoint likely to predict clinical benefit. So that’s where we are with the monotherapy.

I remind you we do have a second Phase 3 study, the Study 012 that is an ongoing study. If like Study 011 also over enrolled, we’re targeting 50 patients, 60 patients came in. And in terms of what we know today, we know that of those patients 36 randomized coming off of ERT completely and switching to migalastat monotherapy, 24 patients with randomization schedule remained on the ERT product either Fabrazyme or Replagal. That is a study with clinical endpoints looking at kidney function measured by iohexol GFR.

Patient compliance to date in that study has been excellent and we expect that data in the second half of 2014. So, lots of progress with the analysis of the data in the 011 study in the first quarter of this year, the development of a very strong regulatory strategy, the early interactions with the FDA, the preparation of briefing document, the submission of the briefing document, and now this Type C meeting here in the second quarter. So that’s where we are with monotherapy program.

If we can move to Slide 5 and will talk just a little bit more about the co-administration programs, again an increasingly important part of our pipeline, focus of our activity is that we think a very important part of value creation for our shareholders going forward. So the Fabry Co‐Formulation study, again this is using the enzyme that’s been developed by JCR Pharmaceuticals and partnered with GSK; this was the basis of our revised and extended agreement last summer with GSK. It’s great for us as we sit here looking to this as an IND planned for the end of this year and we’re still on track for that and entry into the clinic in early 2014.

This was an idea first just over two years ago that we proposed to JCR and GSK that led to a series of preclinical studies and now that drug is in very advance development, again scale to 2000 liters and one intended a product intended for patients that are non-amenable to migalastat as a monotherapy. So for that part of the population that still would need to take enzyme replacement therapy as their primary treatment for Fabry disease, we think that this could be a very, very good option for those patients, again a drug that’s in preclinical studies has been shown to be better targeted to the key tissues and the key organs in Fabry disease with a potential for reduced levels of immunogenicity.

So we again intend to file the IND this year. We continue though to move forward with a very important program for us as well in Pompe disease. Two applications of the CHART technology we’re utilizing in Pompe disease. One is our Chaperone AT2220 now formulated in an IV formulation preparing for entry into its repeat dose study combined with Myozyme or Lumizyme in Q3 of this year that remains on target. A repeat-dose clinical study we’re finalizing the protocol with key investigators but again a novel intravenous delivery of a co-administered Chaperone building on the results of the co-administration PK and safety study that we did last year Study 010.

And with that a good focus of our R&D efforts under David’s leadership at Amicus has been the development of a proprietary next-generation ERT. Basically, what we did in Fabry disease with our partners at JCR and GSK, we intend now to do independently at Amicus. We’re moving forward with the cell line has been developed where again we have a contract for further developments and manufacturing of that with our contract partner at Laureate Pharmaceuticals here in New Jersey, we’re looking at multiple ways in which we can produce an enzyme that we think has the potential to have improvements on the current standard of care for enzyme replacement therapy in Pompe disease, looking at the targeting of the enzyme, its immune profile, as well as the addition of a small molecule in the manufacturing process that could further stabilize it.

Again we don’t have it on this slide but for those of you -- many of you whom we’ve met with over the last couple of months, we’ve shown our pipeline slide and we also indicate other next-generation ERT programs in development. We haven’t yet disclosed that. We’re not yet prepared to disclose that but again a good part of our R&D efforts are to build this very robust pipeline utilizing the CHART platform technology to develop these next-generation ERTs. The first of which is poised to enter the clinic, again in early 2014. So the pipeline continues to advance both the early stage and the late stage and I feel very good about where we’re positioned in the company.

Let me turn it over down to Chip to go to the financial guidance and then Q1 results.

Chip Baird

Great. Thanks John and good evening everyone. I’ll start today’s financial discussion with a few comments about our current cash position and guidance starting on Slide 6. As indicated in this afternoon’s press release, Amicus continues to maintain a very strong balance sheet and we’re on track to achieve our full-year operating expense guidance.

At March 31st, we had $84.8 million in cash and cash equivalents compared to $99.1 million at the end of last year. We continue to estimate full year 2013 net cash spend between $52 million to $58 million and that’s net of anticipated cost sharing under the GSK collaboration.

As a reminder, GSK is responsible for 60% of the global development cost from the migalastat for Fabry disease, 2013 and beyond. Our current cash position along with the anticipated GSK reimbursements, are projected to fund our current operating plan into the second half of 2014.

Turning over slide seven, you will find a snapshot of our first quarter financial results, which you also appear in tables one and two of this press release we issued early today. Additional details can be found in our quarterly report on Form 10-Q, which will be filed later this evening.

For the three points ending March 31, 2013, no revenue was reported whereas we recorded total revenue of $7.8 million in the first quarter of 2012. The year-over-year decrease is entirely due to a change in revenue recognition accounting under the expanded GSK collaboration. This accounting change does not impact cash and we will explain this in further detail on the next slide.

Moving down to P&L, total operating expenses for the first quarter of 2013 totaled $17.3 million compared to $18.5 million in the first quarter of last year. The year-over-year decrease was primarily attributed to lower research and development expenses, as well as a decrease in personnel-related costs.

We had non-operating expense of $207,000 in the first quarter of 2013, compared to non-operating expense of $2.4 million in the first quarter of last year. The change in non-operating expense, which is a non-cash item, is primarily due to the change in evaluation of our warrant liability.

Net loss attributable to common stockholders in the first quarter of this year was $17.5 million, compared to a net loss of $13.1 million in the first quarter of last year. The wider net loss versus the year ago period is attributed to the change in revenue recognition under the expanded GSK collaboration.

Net loss per share of $0.35 in the first quarter of 2013 was unchanged from the year ago period and reflects an increase in shares outstanding, as a result of our March follow-on public offering as well as shares issued in conjunction with the GSK collaboration expansion in July 2013. As of March 31, 2013, we had 39.6 million shares outstanding.

Turning over the page to slide eight, I will provide a brief reminder on the change in revenue recognition accounting under the expanded GSK collaboration. Beginning in the third quarter of 2012, payments received from GSK under the agreement are being recorded in the deferred reimbursements account on the liability side of the balance sheet.

The deferred reimbursement balance on March 31, 2013, totaled $31.7 million and this includes $9 million in Fabry cost sharing since the expansion of the GSK agreement in the summer of 2012, as well as $22.7 million for the unrecognized balance of the upfront payment and premium on equity, originally received back in 2010. Previously, research reimbursements in GSK were recognized as research revenue and a straight-line amortization of the total upfront consideration was recognized each quarter as collaboration revenue.

While the recognitions of payments under the collaboration have changed, there is no cash impacted and mechanics of cost sharing remain the same. GSK continues to reimburse Amicus, for the portion of our actual spend that exceeds our 40% obligation.

Each quarter, the deferred reimbursement balance will increase by the amount of reimbursement of GSK paid to Amicus for share development costs. Any future milestones payable by Amicus to GSK will then be applied against the balance of the deferred reimbursements.

So, this summarizes our key financials for the first quarter as well as our full-year 2013 guidance. More information on our financials will be available in the 10-Q, which will be available online later this evening. I’m happy to address any financial questions during the Q&A session. But for now, we will turn the call back to John.

John Crowley

Great. Thank you, Chip. So on slide 9, I will go ahead and just summarize. Again, Q1 was a really important quarter for us to buckle down to work on the clinical executions, work on the regulatory strategy for monotherapy, to make sure all the rest of the pipeline advances. And I’m very pleased with the company’s performance in the first quarter and heading now into midyear.

If you look on this slide, you can see the migalastat monotherapy. Milestones going forward are very importantly now after this Type C meeting. The first important piece of communication of course is going to be the topline study 011 12-months data in Q3 and then providing, if that looks positive we would move forward with an FDA meeting, Pre-NDA meeting to discuss the U.S. approval pathway in the second half of this year.

I can tell you, getting even deeper into the data with all the work that we did to prepare these documents to the FDA, the briefing documents and preparing for the meeting, I feel even more confident that the data shows that the drug is having a positive benefit in the patients for whom it’s intended to benefit. So consistent with what we saw in Phase 2, we think that this could be potentially a very, very strong option for people with minimal mutations, living with Fabry disease.

Pompe is again an increasingly important part of Amicus. The Pompe ERT co-administration program, the first data was released at that WORLD Conference back in -- the complete data I should say was released at the WORLD Conference back in February.

We continue to move forward with that on target from a timeline perspective, working on finalizing protocols with lead investigators in the world in Pompe disease. And we expect to initiate that repeat-dose clinical study in the third quarter of 2013. Again, that’s an open-label study.

And very exciting, the second application of our CHART technology, seeing that move forward in its final IND-enabling studies and still on track for that IND submission for fourth quarter of 2013 and entry into the clinic in 1Q of 2014. So a lot of milestones moving forward and still a lot in R&D behind the curtain.

I can also comment that we continue to be very active on the business development front, lots of interests. Again, we are partnered in a strong partnership with GSK in the Fabry program but beyond that Pumpe in the rest of the pipeline and our platform technology.

Amicus continues to retain 100% rights to that technology in that platform and our standard continues to be that if there is a partner who can help us move those programs better and faster together than we can do independently that we will certainly entertained that and certainly as a potential tool for further financing of the company, while retaining significant residual financial rights in the program and control of the program development given our expertise in the field. We will certainly entertain that as well. So we have many sets of robust discussions in Pompe, Parkinson’s and other programs continuing and we will continue to provide updates as appropriate.

With that, those are my closing comments and operator, I think we are happy to take questions.

Question-and-Answer Session


Thank you. (Operator Instructions) Our first question comes from the line of Anupam Rama [J.P. Morgan]. Your line is open.

Anupam Rama - J.P. Morgan

Hey, guys. Thanks for taking the question. Just a quick question on Pompe program. Have you finished all the formulation runs for AT2220 and how many doses are you going to be studying in the repeat-dose study? Thanks.

John Crowley

Yeah. So we finished all that formulation work and check back in early to mid Q1. We did the aseptic fill/finish with the contractor that was completed successfully. So the formulation is in very good shape. It’s in its final top studies and so far so good. In terms of the protocol, we are just finalizing that now on the firm I would imagine. In the next month or two, we will be able to provide more clear guidance in terms of what that study is going to look like from a repeat-dose perspective. But I could tell you and we’ve shared this publically before that we are considering this to be a 12 to 24-week open label study. We are just finalizing a lot of details right now.

Anupam Rama - J.P. Morgan

Okay. Thanks for taking my question.

John Crowley

You bet.


Our next question comes from the line of Joseph Schwartz with Leerink, Swann. Your line is open.

Joseph Schwartz - Leerink, Swann

Great. Thanks very much. I was wondering if you could talk some more about the interactions with the FDA that you’ve had and they expect to have. And in particular, if you could give us some insight into what the main argument, do you think that might resonate with the FDA based on the existing data, first of all and then -- what would be positive data that you think would enhance your case from a 12-months end points?

John Crowley

Sure, Joe. I want to be somewhat sensitive since these discussions are going on in real time. But when we first got the data, we -- obviously we reviewed with the Board and we immediately released it and our partners at GSK. We didn’t have plans for Type C meeting prior to unveiling the stage to 12 months data.

However, given the FDA’s guidance that they would consider the entirety of the data such that there is not one primary endpoint that would be just positive, our Board and I felt very strongly that it is better to interact with the agency as frequently and as in depth as possible to try to understand what that means.

And I think a good part of that reflects the very changing regulatory environment especially in the rare and orphan diseases where there is still such unmet need. And the FDA, I think much to their credit working very hard to try and get figure out the right and best solutions to bring these medicines forward especially under PDUFA 5 and fiduciary.

So with that we went ahead in early Q1, requested a Type C meeting. Again, the FDA didn’t have to grant it in the middle of an ongoing phase 3 study. They very quickly came back and said yeah, that’s fine and they agreed to make it an in-person meeting. That led to quite a bit of work and activity here for probably better part of six to eight weeks to draft that briefing document.

In terms of -- I can’t release it, of course, within there, it’s very important that we maintain those confidences going into this FDA meeting. What I can tell you, though, is its not cherry picking or cutting the data. Even that point three cut-off that we’ve talked about before in the stratification of patients by baseline GL-3, we discuss that certainly in a briefing document and we will in person with the FDA. But more it’s an explanation in terms of why we didn’t the hit statistical significance at six months on the interim analysis and why we saw that large placebo effect. I think that data speaks very strongly to it.

We think the most powerful argument with a key part of what should constitute the entirety of the data are going to be based on what we’ve already discussed and that’s the overall level of GL-3. And again, the FDA has indicated that GL-3 is an appropriate surrogate endpoint, the reduction of which it correlates with the likely prediction of clinical benefit.

So we’ve shown very good results, even at six months, again almost reaching statistical significance on that continuous variable migalastat versus placebo. We’d want to look for instance at what we think very important to look at 12 months for that migalastat treated group. Do they continue to have a consistent and durable effect and further reduction at 12 versus six months.

For the placebo group crossing over, we’d like to see did they begin to move in the direction of that first six months of treatment for the migalastat group. So lot of different ways and again, at every point, what we’ve done in this briefing document is to add to this sophistical analysis plan, not to remove elements of it and to highlight what we think will be the important factors to the FDA to consider as they view the entirety of the data.

So I hope that’s clear and I’m confident that doesn’t go into too much of the confidences of the preparations for that meeting and what will be, I think a very good exchange with the FDA.

Joseph Schwartz - Leerink Swann

Okay. It’s very helpful. Thank you. Can I just as a quick follow-up as far as your plans to share with investors, the insights that you’re able to gain. Can you give us some more granularity on when and what form that might take?

John Crowley

Yeah. I think we need to go to that meeting. First, we need to digest the results with GSK. Our bias at Amicus is always to be as transparent and real time as possible. Of course, they need to be respectful of the GSK needs. So I think it’s unclear right now, Joe, whether we would be able to release those, the feedback from FDA and to provide further guidance on with any changes or addition to this statistical analysis plan could be in advance of the 12 months data but certainly it would come with at lease come with the top line’s 12 months data in Q3, so kind of stay tuned.

Joseph Schwartz - Leerink Swann

Thanks again.

John Crowley



Our next question comes from the line of Greg Wade with Wedbush. Your line is open.

Greg Wade - Wedbush

Good afternoon and thanks for taking my questions as well. John, with respect to the -- are you closing with the FDA a new statistical analysis planned for the 12 month’s data?

John Crowley

We’re proposing a revised statistical analysis plan. So our original plan Greg had that primary endpoint being the six month responder yes/no the percent of patients, you show that 50% had greater reduction. That’s would have been in place for several years. If you recall we had a Type C meeting last summer, where we went through a number of different aspects of the program.

And the FDA discussed with us potentially expanding that to look at the entirety of the data. We actually put in place additions to the statistical analysis planned back in the fall of last year. And now we’re looking for further additions to the statistical analysis plan.

So this is somewhat unchartered territory. The advantage here for us is that the FDA has already characterized that six months as interim Stage 1 data. The FDA is viewing this as a 12-month study for safety and for efficacy. And again it’s somewhat unusual because they are looking at the entirety of the data on this risk benefit analysis.

And what we’re trying to do again is to define as best we can what success is going to be when that 12-month data comes. So we can communicate certainly to investors but also to a number of key interested parties in putting physicians and patients and very importantly be able to set up the guidelines and the grounds and the framework for that pre-NDA meeting in the fall. Does this answer your question?

Greg Wade - Wedbush

It does. I think we’re trying to get from this phone call is the parameters around which you’re looking to change things. So for example, if you are successful in convincing FDA to allow you to look at their continuous variable as the primary endpoint in the 12-month study. Well that would more likely because of the success of the six months study lead to potentially statistically significant result.

There is also the flipside too which is the risk right. So on the continuous variable side, if those responders show less of the response at 12 months then that would suggest not a durable impact of therapy and obviously with this being a chronic disease, that’s not going to be good for business perspective. So I think it will be helpful to us to understand what your preference is with respect to this negotiation of the statistical analysis plan. So that we at this end can make an assessment of….

John Crowley


Greg Wade - Wedbush

What the risks are is that -- is what you end up getting?

John Crowley

Yeah. I’ll answer and then I’ll ask David to add just a little more color to it to make sure I capture it all accurately. So our preference and we think the better way to look at this is rather than a responder pure yes or no analysis to look at the overall burden of GL-3 in the cell and the reduction of that overtime and as such we think the continuous variable is going to be a more accurate and more telling way to see what the impact is actually going to be.

So let me just comment that we -- there is not going to be one primary endpoint at 12 months. The FDA has already been very clear that given the nature of this disease, it will be the entirety of the data including the safety components. What we do though want to do is to emphasize the importance and elevate the importance of that already pre-specified secondary analysis. So it becomes a very important part of the FDA analysis at 12 months. Whether it’s just positive or not, it’s ultimately going to be seen at, I think, at pre-NDA meeting.

But I think it certainly helped give you guys because I understand there is some confusion, Greg, in terms of how do we view success. I do agree with you that if we were to see the migalastat treated arm regress at 12 months that would give us some pause for concern as to whether or not this is having the effect that we saw in phase 2 and is persistent and durable. But I think we need to look at the data to make that judgment. David, anything to add please?

David Lockhart

Hi, Greg, this is David.

Greg Wade - Wedbush

Hey, David.

David Lockhart

So just to follow up with what John said, so we’re past, there is now primary at this point. I mean, we’ve past it that was a six months. And so at this point our job is to put together the best view of the data and what it tells us about the treatment effect of the drug and about the safety of the drug. And so it is by its very nature a multi-component and so it will -- there are multiple part of the analyses. And there would be multiple analysis to from different ways of looking at the data in order to determine whether there really is a clear treatment effect in the patients with the amenable mutations and -- versus patients who were either on placebo for the first six months or who do not have amenable mutations.

So it really -- there is no simple answer to saying what do you hope to see on the primary because there is no primary. We’ve have past the primary. But when it comes to the durability of the fact that the key is that for the first six months, half the patient were on drug and half the patients were on placebo. And then at six months to 12 months, everybody is on drug. So we have the chance to see whether the initial placebo group when they’re on drug for six months, whether that looks like what happened in the original group that was on drug for six months.

And then we also get to see the patients who were on drug for the first six months, if they were on drug for subsequent six months whether they have maintained what they -- what we saw in the first six months and whether that got even larger. So I think exactly you said as in any study, there is a chance that the data won’t go in the direction you hope but we have reason to believe that with longer treatment things get better because that’s exactly what we saw in phase 2.

So you’re exactly right there is always a chance that it won’t go as hoped, as with any study. But we think we have the right analysis in place in order to make that assessment. And we have reasonable confidence that there will be both a durable effect and potentially even a greater effect when the patients are on longer based on what we saw in phase 2.

Greg Wade - Wedbush

Okay. And just lastly based upon the things you’re thinking about, do you think you’ll have to have sequence level efficacy results by genotype in order to support the use of the drug across the board in patients or will we use biochemistry or chemical analysis of the amenable mutation? Thanks.

David Lockhart

John, do you want me to answer that.

John Crowley

Yeah. I’m sorry. David, go ahead, please.

David Lockhart

So, Greg, so the selection of the patients that we -- that should go on drug will be -- we expected to be the same as what we’ve done in phase 3, where that decision is based on their mutation. So it is based on sequencing. It is based on the sequencing of the entire coding region of the GLA gene and the patient that have a mutation that we have determined in our cell-based assay to be responsive to the drug. Those are the patients that we believe should go on the drug.

And those that do not have such mutation are ones that we think should not be on the drug and that’s exactly what we did in phase 3 as part of the inclusion and exclusion criteria. So we expect it will be consistent with what we did in phase 3 based on the mutation, based on DNA sequencing.

Greg Wade - Wedbush

And in the phase 3 results that you have presently, you believe they’re supportive of identifying the right patients because the response rate looks like it doesn't quite match up with the number of patients that were included?

David Lockhart

So the answer to that is yes. We do believe that and we have data from phase 2 as well that’s supportive of being able to identify the patients who are likely to respond based on their personal mutation.

So, yes, everything is pointing in that direction, of course, that we will be getting more data as part of the phase 3 study and we will be getting more data for patients who for example on the first six months, they were on placebo and with -- what we now know to be non-amenable mutations who for the second six months will then go on drug and will see if that, whether the amenable group showed an effect where the non-amenable group didn’t.

Greg Wade - Wedbush

Okay. Great. Thank you.

John Crowley

Operator. Yeah, go ahead.


Our next question comes from the line of Kim Lee with Janney Capital. Your line is open.

Kim Lee - Janney Capital

Good afternoon. Apologies if you already addressed this but I got out paunch, what exactly do you expect from this Type C meeting besides going over there the statistical plan? Thanks.

John Crowley

I think we actually did cover that in pretty good detail, Kim. So, I think that’s in the transcript but I will just kind of highlight that what we did was to prepare a very thorough briefing document based on the six months Stage 1 analysis, the interim data for the 011 Phase 3, and a more detailed analysis of all of our Phase 2 studies to add to this statistical analysis plan, focusing on a number of different element including this notion of the continues variable that was a pretty specified secondary endpoint. But to elevate the importance of that in the assessment of the drug from a risk benefit prospective in terms of declining the entirety of the data.

So, I think it will be very important to have this good exchange with the regulators to be able to explain and lay out the data as we see it. We think again it shows very encouraging results that the drug is having a positive benefit in those patients with amenable mutation. And as best we can to frame, how we should look at the entirety of the data once this 12-month data is unveiled.

Again, we and GSK are blinded to the data still. It’s a very unique and important aspect of this study to remember that the primary reason, maybe the only reason that we can add to the statistical analysis plan in an ongoing Phase 3 is that we have not lock the data base and we still remain blinded to that 12-month data, although remember of course all those patients samples were collected by the end of 2012.

Kim Lee - Janney Capital

Okay. And just as a follow-up and I appreciate you reviewing this.

John Crowley

That’s okay.

Kim Lee - Janney Capital

But also what time show will this be -- with the FDA after the Type C meeting or could thing still be flexible and move around and change as far as agreements go after you on blinded data?

John Crowley

It certainly could be subject to further analysis and interpretation after the 12-months data. We are trying before we get to the 12-months data to as best we can define what the key elements of analysis should be. So stay tuned we will see I think I’m very, very pleased with the FDA accepting this request for Type C meeting. They are making personal meeting down in Maryland and I think hopefully speaks to there interest in seeing therapies for Fabry advance.

Kim Lee - Janney

Great. Thank you.

John Crowley

You’re welcome.


At this time, I’m not showing any further question in queue, I would now like to turn the call over to John Crowley for any further remarks.

John Crowley

Great. Operator, that’s all I have. Thank you, everybody for listening to the great questions and a lot more to come in the months ahead. So, thank you.


Ladies and gentlemen, thank you for participation in today’s conference. This does conclude today’s program. You may all disconnect and everyone have a great day.

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