Sara Pellegrino - Director, IR John Crowley - Chairman and CEO Chip Baird - CFO Bradley Campbell - President and COO Jay Barth - CMO Hung Do - CSO Dipal Doshi - Chief Business Officer Ken Peist - VP, Intellectual Property.

Ritu Baral - Cowen & Company Anupam Rama - JPMorgan.

Good day, ladies and gentlemen and welcome to the Amicus 3Q Results Conference Call and Webcast. At this time, all participants on the phone lines have been placed on mute. Later, we will conduct a question-and-answer session, and the instructions will be given at that time [Operator Instructions]. Please do note, today's program is being recorded.

I would now like to introduce, Sara Pellegrino, Director of Investor Relations for opening remarks..

Good evening and thank you for joining our conference call to discuss Amicus Therapeutics’ third quarter 2015 corporate highlights program updates and financial results. Present on today’s call, we have John Crowley, Chairman and Chief Executive Officer; Chip Baird, Chief Financial Officer; Bradley Campbell, President and Chief Operating Officer, Dr.

Jay Barth, Chief Medical Officer; Dr. Hung Do, our Chief Science Officer and Dipal Doshi our Chief Business Officer. The slide deck to accompany this call is also available on our corporate Web site at www.amicusrx.com in the Investors section.

This presentation will contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of Amicus’ candidate drug products.

The timing and reporting of results from preclinical studies and clinical trials evaluating Amicus’ candidate drug products, financing plans and the projected cash position for the company.

Words such as but not limited to look forward to, believe, expect, anticipate, estimate, intend, potential, plan, targets, likely, may, will, would, should and could and similar expressions or words identify forward-looking statements.

Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. The inclusion of forward-looking statements should not be regarded as a representation by Amicus that any of its plans will be achieved.

Any or all of the forward-looking statements in this conference call may turn out to be wrong. They can be affected by inaccurate assumptions Amicus might make or by known or unknown risks and uncertainties.

For example, with respect to statements the regarding goals, progress, timing and outcomes of discussions with regulatory authorities and in particular the timing of an NDA submission for Migalastat Monotherapy and the potential goals, progress, timing and results of preclinical studies and clinical trials.

Actual results could differ materially from those set forth in this presentation due to the risks and uncertainties inherent in the business of Amicus, including without limitation the potential that results of clinical or preclinical studies indicate that product candidates are unsafe or ineffective, the potential that it may be difficult to enroll patients in our clinical trials.

The potential that regulatory authorities may not grant or may delay approval for our product candidates, the potential that preclinical and clinical studies could be delayed because we identified serious side effects or other safety issues, the potential that we will need additional funding to complete all of our studies and our dependence on third-parties and the conduct of our clinical studies.

Further the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. With respect to statements regarding projections of the company's cash position, actual results may differ based on market factors and the company's ability to execute its operational and budget plans.

In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2014 and Form 10-Q for the quarter ended June 30, 2015. You're cautioned not to place undue reliance on these forward-looking statements which speak only as the date hereof.

All forward-looking statements are qualified in their entirety by this cautionary statement and Amicus undertakes no obligation to revise or update this conference call to reflect events or circumstances after the date hereof. This caution is made under the Safe Harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.

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

Great. Thank you, Sara and good evening everybody. I am very pleased to be joined here tonight by our executive leadership team, and we’re going to go ahead and highlight our recent progress with our development programs, as well as an overview of our third quarter 2015 financial results.

We do have a document online for those of you who can access it so I’ll begin on Page 4. And at the outfit, I really want to emphasize the enormous effort and commitment by this team and everybody at Amicus together with all the folks that we work with to advance these programs.

And in addition, I’ll begin on the Slide 4 by highlighting the very short focus we have here at Amicus. We have four key business priorities, and let me begin by outlining those; so first is the approval and then the commercialization of our personalized medicine Galafold for Fabry disease in Europe.

We remain on track to receive an opinion from the CHMP by the end of this year, or early next year. I also want to highlight that we have assembled an extraordinary commercial leadership team in place to launch Galafold in key regions once approved, and Brad will provide more color on that shortly.

These individuals who will lead these efforts for Amicus have a successful track record in launching drugs for rare diseases. Next key part of the focus for the Company remains our continued commitment to work to determine the optimal U.S. approval pathway for Galafold.

We are in the process of integrating the data from our Phase 3 studies and Phase 2 studies as requested by the agency. And we are further evaluating several U.S.

approval pathways, including the potential to generate additional data on gastrointestinal symptoms to support a request for full approval or potentially to request accelerated approval under sub part H, possibly based on surrogate endpoints beyond just interstitial capillary GL3.

The timing of an NDA submission will be based on the completion of the required data integration, as well as the determination of the optimal regulatory pathway. We continue to expect as we work on this diligently and we continue to expect to provide an update on the U.S. strategy in the first quarter of 2016.

Our third focus of the Company, and our third strategic priority, is the initiation of our Phase 1/2 clinical study of our novel Pompe enzyme replacement therapy.

On the manufacturing front, I am pleased to report that we continued with the successful GMP manufacturing campaigns, and we have now secured the necessary clinical supply for this very important Phase 1/2 study to commence. Earlier in October, we held a CIND meeting with the U.S.

FDA and based on the final minutes now received from that meeting we plan to submit the IND for this Phase 1/2 safety and PK study in Pompe patients of the fixed dose combination of our novel ERT together with our chaperone. We’ll have more color on that shortly Hung and Jay will provide that.

But overall, we are very pleased with the progress of this important program, both on the manufacturing front, as well as our preparations to enter the clinic. And our fourth key priority and focus of the Company is our program in epidermolysis bullosa, or EB.

And with that our Phase 3 EB program, our novel topical therapy Zorblisa that we brought into our pipeline in the third quarter through our acquisition of Scioderm. And we are currently recruiting patients in a Phase 3 study at site in both the United States and Europe to support global registration.

This study is currently approximately one-third enrolled, and we had excellent progress with this program, and we are very pleased to announce this evening on this call that we have just now initiated the rolling NDA submission for Zorblisa.

So a major milestone to this program, a tremendous effort and an example of the early success of our integration of the Scioderm team into Amicus and those teams working together to advance this important therapy. As many of you know EB is a chronic rare genetic connected tissue disorder with no approved treatment options.

It is one of the most devastating and excruciatingly painful and potentially stable genetic diseases that I’ve ever seen. We are fully aligned with the sense of urgency among community in the EB patient community to get a treatment approved.

Jay will get into more specifics shortly on the EB program including some additional data that we’ll be sharing for the first time this evening on the Phase 2B study, but very positive momentum with this program.

So again I just want to emphasize the enormous commitment to this team, the enormous amount of work that’s going into these programs and our very sharp focus on these four key business priorities that I’ve just outlined. With that, I’d like to begin with the key updates, beginning with Galafold in Europe.

So Jay if you can begin with the regulatory update on Europe with Galafold please..

Good afternoon everyone. I am pleased to provide an update today on the European regulatory progress with Galafold, which you can see on Slide 6.

Our MAA is under review by the European Medicines Agency, EMA, under accelerated assessment which is only granted to therapies for diseases where it is a significant public health interest and unmet medical need, and what this means is that the review period for the application maybe 150 days under accelerated assessment instead of the 210 days for a standard review.

Our MAA was validated at the end of June and as part of the review process under accelerated assessment the CHMP sends a list of questions after 120 days, known as the day 20 questions and the review clock stops at that point.

We have received that list of questions and we are in the process of finalizing our responses and once we submit the responses, the review clock begins again and we expect to be on the CHMP meeting agenda to receive an opinion at the end of this year or early next year.

On that note, I will turn it over to Bradley to provide an update on commercial preparations in Europe..

Thank you, Jay good afternoon everybody. As John mentioned as we approach the potential launch of Galafold in Europe and outside of the United States, we have continued to build out our commercial team and have made significant progress with our launch preparations which are detailed on Slide 7.

We do have leaders in place now in our key functional areas internationally, as well as our key regions, who will be able to lead the launch of Galafold successfully. We also now have our distribution system in place and have started to fill in our medical and sales teams in our initial launch countries.

We believe that our world-class commercial team can build upon their prior experience in launching orphan and rare disease drugs, as well as the strong history that Amicus has established with our very broad and global footprint in terms of our clinical trial activities, our very strong history of patient efficacy and medical outreach and our other activities.

With that, I will turn it back to Jay to further discuss our EB Program..

Thanks, I would like to highlight our EB Program and there are two new data points from the Phase 2b study that I'll be focusing on today, which haven't been previously disclosed. I'll start on Slide 9 where we're showing the primary endpoint results from the Phase 2b study in both the intent-to-treat or ITT population and the evaluable population.

As a reminder, the primary endpoints of the study was, the percent of patients with complete closure of target wounds and importantly it is also the primary endpoint of the Phase 3 study.

As you will see here the results for the ITT population on that top table and the evaluable population below, the results for the ITT population show the trends towards improved higher proportion of target wound closure for Zorblisa 6% versus Placebo at month one and even more so at month two.

And in the evaluable population, the same effects are present, but even more strongly at month one for a higher proportion of complete target wound closure for Zorblisa 6% versus Placebo. And at month two, the effect is even greater 82% versus 41% which is assisted with the P value of 0.04.

Month one is the pre-specified primary endpoint in the Phase 2b study. Month two is the Phase 3 primary endpoint, and these data are particularly significant because of course this treatment for EB is focused on closing open wounds that is the primary endpoint here.

These wounds were chronic in nature, so being able to achieve this high rate of closure and difference versus placebo in this double-blind Phase 2b study is of great importance in advancing a treatment for EB.

On Slide 10, we're showing the results on the important secondary endpoint which is the median time-to-wound closure and this is both shown as for the ITT population and the evaluable population. This is also an important pre-specified secondary endpoint in the ongoing Phase 3 study.

And as you can see here in both the ITT population and the evaluable population, Zorblisa 6% shows a faster time for wound closure compared to the 3% and placebo, 40 days versus 91 days for placebo in the ITT population, 30 days median time to wound closure for Zorblisa 6% versus 91 days for placebo in the evaluable population.

So for this goal, we focus on the new data but for reference we have an appendix with additional details on the proportion with complete target wound closure over three months in the ITT and evaluable population. And also background slides that we have shared previously. We're happy to cover those during the Q&A session or in follow-up discussions.

On Slide 11 we have shown this before, Zorblisa is shown to be well tolerated with adverse events that were similar across all three treatment arms. Slide 12 summarizes the results and key learnings from the Phase 2a and Phase 2b studies that have helped us in the design and conduct of the Phase 3 study.

Of course the Phase 2a study showed proof-of-concept and we've seen in the Phase 2b study the safety being similar across the treatment groups. And the clear dose response favoring the 6% concentration over 3% in the percent of wounds closed as well as the median time to wound closure.

And the Phase 2b results are very helpful in being able to calculate the adequate sample size needed for the Phase 3 study. And with the sample size that we currently have for the Phase 3 study, the treatment difference of approximately 17% or greater will be associated with the P value of less than 0.05.

The placebo response is minimized so that is something that has been seen in these trials by targeting baseline wound size of at least 10 centimeter square. In the Phase 2b think it was at least 5 centimeter square so that is a learning from the Phase 2b that has been applied to the Phase 3 study, in terms of eligibility criteria.

Also the wound closure within two months versus a one month is really optimal time as the primary endpoint. In the Phase 2b it was one month, in the Phase 3 it is two months time point, because that really increases the ability to distinguish between the Zorblisa treatment affect versus placebo.

And as we said this endpoint target wound closure has been accepted both by the FDA and European regulators. And importantly there's defined approval pathway based on the Phase 3 study based on EMA and FDA feedback. I'd like to close out the section with a brief mention of our regulatory pathways for Zorblisa.

As you can see on Slide 13, EMA and FDA have agreed to the study design. We have breakthrough therapy designation in the U.S. and importantly sales announced today that we have just submitted the initial portion of our rolling NDA to the USFDA.

We're really pleased with the early success of the program integration and the significant momentum of our ongoing Phase 3 study which is about one-third enrolled at this point.

I’ll also highlights that 36 patients have completed the three month primary treatment period in the Phase 3 study and that all 36 patients have elected to continue into the ongoing open-label extension study.

We look forward to sharing the Phase 2a and Phase 2b data including additional analysis and extension study data in early 2016 at Scientific Congress. I’ll now turn it to Hung..

Yes, great, we'll go ahead and turn the call. Thank you, Jay that was an excellent overview of the momentum in the Zorblisa program and specifically the significant milestone for Amicus and the Scioderm team of filing the first section of the rolling NDA.

We'll now go ahead and turn it over to Hung and then back to you Jay for the clinical section of this important Pompe update. So, Hung let's go ahead and turn it you and we'll begin on Slide 15, go ahead please..

Great. Thank you John, hello everyone. It is my pleasure to be here today to walk you through the recent progress of our novel ERT Chaperone fixed dose combination for Pompe. We are now getting ready to enter the clinic and have made significant progress with our preclinical studies and our manufacturing activities which are highlighted on Slide 15.

We have developed a proprietary celluloid to produce a Pompe ERT designated as ATB200 which has optimal glycosylation particularly with high amounts of the specialized carbohydrate called mannose-6 phosphate for a efficient drug targeting.

Working with our partners at WuXi we have successfully scaled up the manufacture of ATB200 and have completed GMT manufacturing campaigns necessary to initiate the Phase 1/2 study.

So on the following Slide 16, you can see that we have been successful in our manufacturing where we have scaled from small scale to five meter scale up to our clinical production scale which is at the 250 meters.

Importantly, we have been able to maintain the critical quality attributes that are needed to ensure good drug targeting throughout the scale up of this manufacturing process.

As shown on this Slide ATB200 produced at two and five meter scales, as well at the 250 meter scale all contained similar amounts of mannose-6 phosphate for binding the mannose-6 phosphate receptor, which is shown in the second and third peaks on this chromatograms.

These were performed on an analytical mannose-6 phosphate receptor column to demonstrate the ability of the enzyme to bind the mannose-6 phosphate receptor. If you recall high affinity bonding to the mannose-6 phosphate receptor is required for efficient delivery of Pompe ERTs [indiscernible].

And just one thing I would like to remind everyone why we were so excited about this particular approach is that we have demonstrated strong preclinical proof of concept in preparation for entering the clinic.

If you look on Slide Number 17, you will see that we used the GA knockout mouse model for Pompe disease such that the deficiency in the GA enzyme activity leads to found glycogen accumulation as shown by the fast staining the purple fast staining in the upper panels.

Further, the cumulative glycogen is contained within these internal cellular compartments or vesicles such that the apparent proliferation of these internal vesicles is a hallmark of Pompe disease.

We used the stain of LAMP1 a known plasmoprotein within these vesicles to evaluate the effects of ERT for clearance of these internal vesicles and is shown in the lower panels. Our cumulative studies indicate that the standard of care is only moderately effective for reducing glycogen and a turnover of these proliferated vesicles.

These findings are in fact consistent with previously published data.

If our contrast ATB200 co-administered with our Chaperone AT2221 was shown to substantially reduce glycogen levels, as well as to facilitate the turnover and recycling of these internal vesicles as evidenced by the significantly reduced PAK staining as well as the LAMP1 staining just to levels that are near normal levels.

Again together these data suggests that our Pompe ERT approach has significant impact towards addressing the muscle pathology associated with Pompe disease and warrants further evaluation. And with that let me turn it back over to Jay, our Chief Medical Officer to provide an overview of our Pompe clinical strategy..

Thanks, Hung. We’re pleased to move our Pompe program into the clinic in Pompe patients, which we summarized on Slide 18. As John mentioned, we recently held a pre-IND meeting with the FDA. Based on feedback from the agency and the final minutes received from that meeting, we’re now finalizing our Phase 1/2 safety and PK study protocol.

I’ll highlight for you key parameters of this study as currently planned. We’ll be in Pompe patient switching from approved ERT. The ATB200 plus chaperon will be viewed like a fixed dose combination and we will not be required to study the chaperon component separately.

The dose selection for a Phase 3 study maybe based on PK and safety data from this Phase 1/2 study. The IND will be submitted very shortly, and we expect to initiate clinical sites this quarter to begin the study.

We believe that our clinical plan is a smart path into through the clinic towards Phase 3 with what we believe to be a highly differentiated product that has the potential to offer a great benefit for people living with Pompe. Now I’ll turn the call over to Chip to review the third quarter financial results and guidance..

Great. Thanks, Jay. Good evening everyone. I’ll start today’s financial discussion with a few comments on our current cash position and financial guidance beginning on Slide 20. Amicus continues to making a very strong balance sheet.

Cash, cash equivalents and marketable securities totaled $251 million at September 30, 2015 compared to $169 million at the end of last year. During the third quarter, we successfully completed our acquisition of 100% for the capital stock of Scioderm Inc.

With the closing of Scioderm acquisition and the forecasted spending on the EB clinical development program, we expect to end 2015 with between $200 million and $225 million of cash on hand. Current cash is expected to fund our operating plan including Zorblisa into 2017 through several important near-term inflection points.

Turning to our third quarter of 2015 financial results on Slide 21, I’ll be referencing tables one and two in the press release that we issued earlier this afternoon, and additional details will be found on our Form 10-Q which will be filed tomorrow morning.

Total operating expenses for the third quarter of 2015 increased to $38 million compared to $17.1 million for the third quarter of 2014.

The year-over-year increase was primarily due to increases in preclinical and clinical development costs on Fabry monotherapy program and the Pompe ERT program, as well as investment in prelaunch activities for Galafold.

The net loss attributable to these common stockholders in the third quarter was 37.8 million or $0.32 per share compared to a net loss of 17.1 million or $0.22 per share in the third quarter of last year. The wider net loss is primarily attributed to the increase in operating expenses.

And as of September 30, 2015 we had approximately 119 million shares outstanding. This summarizes our key financials for the third quarter, as well as our updated full year 2015 guidance. More information on our financials will be available in the 10-Q which should be online tomorrow morning. Happy to address any questions during the Q&A.

But for now, I’ll turn it back to John..

Great. Thank you, Chip. So I will just to finish up before we head to Q&A, by reiterating again the short focus we have on the creation of shareholder value and advancing our mission around our four key business priorities in Fabry, Pompe and EB.

We continue to believe we have the potential to create substantial shareholder value, and again to deliver upon our mission for rare disease patients will remain at the center of everything we do here at Amicus. With that operator, we’re happy to turn it to you and to answer questions..

Thank you. [Operator Instructions] Our first question comes from the line of Ritu Baral with Cowen & Company. Your line is open, your question please..

Can you give us any more color on the day one 20 questions received from the EMA? It seems like if you want to turn it around for a CHMP decision by the end of the year, you wouldn’t have a whole lot of time to prepare the questions.

Were there any surprises in what was asked?.

Yes so I think best Ritu if we don’t comment on the ongoing regulatory interactions. I think I’ll just reiterate that we remain under review, under accelerated assessment at this time and we expect to be on track for that CHMP opinion by year-end or very early 2016..

Is there any indication that you may see mutual regular review?.

No, there is always the potential but as part of the 120 day questions, they came back to us stating that we will remain under accelerated assessment..

And then any more color on the European hires that you've plan to make as you prepare for Galafold launch over there, and number of reps to geographies and potential launch order of the country based on the reimbursement?.

Yes, let me ask Brad to add some color there please..

Yes, Ritu, I don't want to get into too much detail there perhaps for competitive purposes, but I can say at a high level the key leadership is in place and those are across the [indiscernible] to medical, legal, finance in addition to the key regional structures in order to launch in the initial launch countries from a launch sequencing perspective, again I will keep it at a high level, but I'll say that there is nothing necessarily unique about sequence that removing towards and we're very confident that we are ready to launch as quickly as we can and in as many geographies as we can following the normal practice once we have the CHMP opinion..

And last question is on durable sense and as the new data presented, the footnote on Slide 9 goes through the rotations that were excluded from the evaluable population, can you clarify a little more on the two patients you've stated that they did not have a single identified or qualified target region, what does that mean what were the circumstances?.

Yes of course Ritu Jay will answer….

Specifically the eligibility criteria were that the target wound be contagious that if would be one wound and meet the eligibility side.

In this case they were these two patients who had each had two wounds that were very close to each other almost touching that were counted as a single wound initially, but then it was recognized that did not meet the eligibility criteria of having one wound then at the target size and that's why those patients were excluded from the evaluable population..

Thank you. Our next question comes from the line of Anupam Rama with JPMorgan. Your line is now open your question please..

Just a quick one for me on the rolling submission for Zorblisa, I was just wondering if you could give us a little color if there is any ongoing preclinical or CMC work that needs to be completed between now and say the Zorblisa Phase 3 data later next year, I guess a little color on that? Thanks..

No we have completed all the necessary preclinical requirements in the first section of the rolling NDA was the preclinical, we would expect the next section to be the CMC and while they is still drafting work to pull that together, I don't believe there is anymore CMC requirements that we have to meet specifically for that section of the rolling NDA..

Thank you. [Operator Instructions] Our next question comes from the line of [indiscernible] Leerink Partners. Your line is open, your question please..

Just a couple of question on Zorblisa, just wondering backtracking to the announcement of Scioderm acquisition back in August, you had mentioned a third of the enrollment was completed at that date which was August 31st, so just wondering if you could comment on sort of the delays in terms of the enrollment and where you might be seeing that particularly whether it's here or in the U.S.? And also the second question would be if you could please comment on the anti-protections around Zorblisa, I understand that [indiscernible] is highly insoluble but just trying to get a sense of how much of breadth do you have in terms of usage? Thank you..

Sure, yes, to be clear we don't see delays enrollment continues to advance, we continue to enroll patients. We're opening additional sites. I believe the update we gave is that the study was a third enrolled and that was at the very end of September, I believe it is the first time we had mentioned that so just about a month ago.

The additional point to highlight is the original Scioderm plan called for 130 patients. We've modified that to meet this statistical plan at 150 patients so that changes the math of it as well, but that program continues to advance well..

And about the IP?.

Yes, secondly around the IP we have, our IP counsel Ken Peist is here as well and Ken if you want to comment about intersection that we know around the IP of Zorblisa and specifically the formulation in some of the inventions there..

Exactly yes so the novelty around Zorblisa really is in the formulation of high a concentration of the active ingredient and it's stable in the formulation at this high concentration at room temperature and that's the primary area of protection and of course there will be data protection and orphan exclusivity for this product..

And just to highlight given the pediatric investigational plan in Europe that would give us 12 years of exclusivity in Europe and with the pediatric plan in the United States would give us 7.5 years in addition of the intellectual property protection that Ken noted. And again that's a portfolio that we continue to strengthen as well.

So we feel it will be a very well protected franchise..

I am showing no further questions at this time. I would like to return the program to John Crowley for any additional remarks..

Great, no operator that is all we have everybody, thank you for listening hopefully again you could sense the commitment, the enormous amount of work that's gone into advancing the company even just in the last thirty days with a couple of the key milestones we highlighted here around the Fabry monotherapy program, around the Pompe program and that successful pre-IND meeting that's now formed the basis to advance our Pompe program towards the clinic, as well as the momentum including now the first section of the rolling NDA for Zorblisa.

So again we will continue to work as hard as we can for patients and for shareholders and thank you for listening and for your support. Have a good night..

Ladies and gentlemen, thank you very much for your participation. This does conclude the program. You may now disconnect..