Sara Pellegrino - Senior Director, IR John Crowley - Chairman, CEO Chip Baird - CFO.

Ritu Baral - Cowan Anupam Rama - JPMorgan Dae Gon Ha - Leerink Partners Roy Buchanan - Janney Montgomery.

Good day, ladies and gentlemen, and welcome to the Amicus Full Year 2015 Results Conference Call and Webcast. At this time all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will be given at that time. [Operator Instructions] As a reminder, this call is being recorded.

I'd now like to turn the call over to Sara Pellegrino, Senior Director, Investor Relations. You may begin..

Good morning. And thank you for joining our conference call to discuss Amicus Therapeutics full year 2015 corporate highlights program updates and financial results. Speaking on today's call we have John Crowley, Chairman and Chief Executive Officer; and 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 and General Manager of Scioderm are also here and available to participate in the Q&A session.

Before we begin, we wish to inform participants that today's call will contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates.

The timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, financing plans and the projected cash position for the company.

The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements made during this conference call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties.

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

And the expected timing of the EMA's final decision with respect to regulatory approval of Migalastat in the European Union.

Actual results could differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including without limitations, the potential that results of preclinical or clinical studies indicate that the 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 including the EMA may not grant or may delay approval of our product candidates, the potential that we make may not be successful in commercializing our product candidates, if and when approved.

The potential that preclinical and clinical studies could be delayed because we identified serious side effects or other safety issues and the potential that we will need additional funding to complete all of our 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 best 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.

At this time, 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 morning, everybody. As you know with Amicus we have four key strategic priorities. Number one, getting migalastat approved in the EU as quickly as possible for all the Fabry patients with all amenable mutations. Number two, determining the optimal path forward for migalastat in the United States.

Number three, obtaining important early clinical data for our novel Pompe ERT. And number four, executing successfully on our Phase 3 study in EB, epidermolysis bullosa. We continue to remain very sharply focused on these four key strategic priorities and we made substantial progress during the past quarter in this regard.

On today's call, we will focus on strategic updates for these three key programs as a well as our full year financial results. Let me begin this morning's call with the regulatory update for migalastat for Fabry disease.

As you know, migalastat is an oral small molecule pharmacological chaperone that has the potential to be the first personalized genetic medicine for Fabry disease. If approved migalastat will represent the first new therapy for Fabry patients in more than a decade and it may represent an enormous step forward in the treatment of this disease.

Migalastat has been investigated now in the two largest Phase 3 studies ever completed in Fabry patients. In the EU right now, our marketing authorization application or MAA is under final review and we are advancing toward a CHMP opinion.

I'm pleased to report that the Amicus team and several key opinion leaders completed the oral explanation earlier this week at the February CHMP meeting. As many of you know, an oral explanation is part of the MAA process for many novel orphan drugs. And as we have seen for other new orphan drugs, the opinion often comes at a subsequent meeting.

The EU regulators do not want us to comment on the details of our preparation or on their feedback, but we at Amicus continue to be pleased with the level of collaboration and engagement with our [indiscernible] and the entire CHMP. We greatly appreciated the opportunity to discuss migalastat with the broader CHMP team and its members.

I'm enormously proud of our Amicus team and the Fabry clinical experts, who presented before the CHMP on migalastat this week. The team did an excellent job and was very well prepared for all of the questions and dialogue at the CHMP meeting. We continue to have confidence in the regulatory process and in our clinical data.

We have been able to answer all questions from the EMA with our existing dataset. The decision on migalastat is now in the hands of the regulators with an opinion likely during the next CHMP meeting which will be held March 29 through April 1.

If approved, our commercial team, which is composed of individuals with significant commercial experience in leading rare disease companies has already done an excellent job with prelaunch preparations including appropriate medical outreach and patient payor and physician understanding and the Amicus team in Europe is ready to launch migalastat there and in other international territories.

We have built, I believe, in the past year a premier orphan disease international commercial and leadership team and that team is fully prepared and excited hopefully to bring this medicine to people that live with Fabry disease too. On the U.S. update alluding to our U.S.

strategy for migalastat, we have previously stated that we would provide an update in the first quarter. On this call, I'm pleased to report that our integrated summary of safety is on track and nearly complete.

We have also collected now and analyzed additional histopathology data and gastrointestinal symptom data as well as longer term renal and cardiac data for [indiscernible] Phase 3 studies. Several parts of these new data analyzes will be featured at next weeks Scientific World Symposium.

We continue to believe that longer term data as well as the new analyzes from our existing studies add to the totality of the data for migalastat. We expect a meeting with FDA to take place during the second quarter to seek further clarity on the U.S.

regulatory pathway for migalastat and following receipt of formal minutes from the agency from that meeting, we will plan to issue further clarity on the U.S. regulatory pathway for migalastat. We remain fully committed to identifying an optimal U.S. pathway based on our existing dataset without doing another Phase 3 study.

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

We believe that clarifying the pathway is the best interest to patients as well as our shareholders and we look forward to a further update in the second quarter. Let me move now to our Phase 3 program in epidermolysis bullosa, EB. In the third quarter of 2015, we acquired a late stage potential first to market topical treatment SD-101 for EB.

As you know, EB is a chronic rare genetic that connective tissue disorder with no approved treatment options. EB is a debilitating excruciatingly painful and potentially fatal disease. It's a disorder with 30,000 to 40,000 diagnosed patients in just the U.S., EU and Japan.

We are fully aligned with the sense of urgency among this EB community to get a treatment approved.

And since the acquisition, we have made significant progress in adding our Amicus resources, experience and clinical operations capabilities to the enrollment to the ongoing Phase 3 study on top of the excellent work that the Scioderm operations team has done prior to our acquisition.

As of today, I'm pleased to report that the study is just over 50% enrolled and 53 patients have completed the primary treatment period and all 53 of those patients, so 53 out of the 53, who have completed that three month primary treatment period have all voluntarily elected to continue in the open label extension phase.

We remain on track to complete enrollment mid-year and we continue to expect to report the top line data in the second half of this year. By the time, this study is expected to be fully enrolled in the middle of this year; we will have doubled the number of clinical sites and nearly tripled the overall resources in this program since the acquisition.

In addition to opening additional clinical sites and augmenting the recruiting efforts, the Amicus team has been working diligently alongside the Scioderm team on publication and abstracts from reported dermatology conferences to raise visibility within the medical community about SD-101 as well as the disease itself.

I'm pleased also to report on this call today as a result of these efforts, we have been accepted to present at the American Academy of Dermatology or AAD, by Dr.

Amy Paller at Northwestern University, who is a leading investigator who also ran the initial single center Phase 2a study with SD-101 that led to the break through therapy designation will present the Phase 2b data on March 6 at this conference.

While much of this top line data have already been shared publicly by Amicus, it will be the first time for these data to be presented at a very important dermatology meeting by a key opinion leader and investigator.

Moving to our Pompe program, we continue to be very, very excited about the potential for a novel treatment paradigm for Pompe disease, which consists of the novel ERT, our proprietary ERT ATB200 co-administered in a fixed dose regimen with our AT2221.

This is an 18-week safety and PK study beginning with three ascending doses of our ERT ATB200 alone followed by that ERT plus chaperone. Following the 18-week treatment period patients are eligible to continue to receive ERT plus chaperone. Initially the clinical study will look at patient switching to the current ERT standard of care.

Key things we are looking forward to differentiate our novel treatment paradigm include safety and plasma PK to demonstrate potential benefits of the addition of a chaperone to inform dose selection for a next study. Also tolerability and infusion associated reaction as well as antibodies and the characterization of the types of antibodies.

We will continue to follow these Pompe patients in the extension study and to include patients naive to ERT in this study after we have the initial switch data. We will also look at functional measurers of the disease and assessment and these patients will continue to be assessed based on these functional measures in the extension study.

So a very, very important study for us in Pompe and I will be happy to answer any questions on our Pompe program as well as Fabry and EB. Before we do that, let me turn the call now over to our CFO, Chip Baird.

Chip?.

Great. Thanks, John. Good morning, everyone. I will start today's financial discussion with a few comments on our current cash position and our overall 2016 financial guidance. Amicus continues to maintain a very strong balance sheet.

Cash, cash equivalents and marketable securities totaled $214 million at December 31, 2015 as compared to $169 million at December 31, 2014. We strengthened our balance sheet significantly during 2015 with $258 million public offering. We expect full year 2016 net cash spend between $135 million and $155 million.

The current cash position is projected to fund our operations into mid-2017 through several important inflection points that John outlined previously in the call. Turning to our full year 2015 financial results, I will be referring to Tables one and two in the press release we released earlier today.

And additional details will be found in our Form 10-K, which will be filed on Monday, February 29. Total R&D expenses for the full year 2015 increased to $76.9 million as compared to $47.6 million for 2014.

The increase is primarily due to higher contract manufacturing and clinical research costs driven by the scale-up of our Pompe ERT manufacturing as well as advancement of all of our clinical development programs. Total general and administrative expenses for the full year 2015 were $47.3 million that compares to $20.7 million in 2014.

The increase was primarily due to pre-commercialization organizational costs as well as transaction costs incurred with the acquisition of Scioderm. Net loss for the full year 2015 was $132 million or $1.20 per share compared to a net loss of $68.9 million or $0.93 per share in 2014.

The widened net loss is primarily attributed to an increase in total operating expenses and as of December 31, 2015, we have approximately 125 million shares outstanding. Last Friday, we amended our $50 million debt agreement with Redmile. This had no immediate impact on the balance sheet.

However, the amended terms around the initial $50 million tranche increased our financial runway and provide access to additional $25 million in funding on certain clinical and regulatory milestones.

We also disclosed this morning, we are establishing a $100 million aftermarket or ATM equity financing facility and this is a matter of good corporate financial housekeeping. We previously used ATM facility with great success in 2014.

To finalize three prospectus for the ATM will be filed early next week and while the provided debt agreement as well as the Amicus future flexibility, unfortunately we have no immediate plans to sell shares.

In the near term, we will continue our focus on carefully managing cash and being sensitive to dilution as we drive towards value creating milestones throughout 2016. So this summarizes our key financials for 2015 and as I said, more information on financials will be available in the 10-K, which will come online on Monday.

I'm happy to address any questions during Q&A. But, for now, let met turn it back to John..

Great. Thanks Chip. So this quarter let me just highlight and conclude here with some of the key milestones over the coming months and throughout this year. Of course, the CHMP opinion on migalastat and FDA meeting on strategy for migalastat and a regulatory path forward there.

The data on March 6 and presentation in EB; the data and presentations next week at the world symposium. The EB Phase 3 data in the second half of this year and also in the second half of the interim and full Pompe clinical data.

So hopefully, you can see all of these activities continue on the path toward our vision to create one of the world's leading global biotechnology focused company's focused on rare and orphan diseases. So with that, operator, we will open the call to questions..

[Operator Instructions] Our first question comes from Ritu Baral of Cowan. Your line is open..

Good morning, guys. Thanks for taking the question. First question probably unsurprisingly is on the CHMP proceedings this week.

Can you generally give us the topics that were covered during the oral explanation? I understand you don't want to give details, but just generally what were the angles and were there any surprises on the topics covered?.

Yes, Ritu. I can't comment on the nature of the questions and the exact interaction with CHMP. I will just reiterate that I was very pleased and that the team did an excellent job. They were well-prepared as well as the Fabry key opinion leaders who were there at the meeting to talk about migalastat as well.

And we continue to have confidence in the process and in our clinical data and it's now in the hands of the regulators..

So, I want to confirm, you said there are no outstanding questions that any topics covered were answered in full?.

I can't comment on the nature of the discussion as well except at stage that I -- we believe it's likely that we will receive an opinion at the next meeting..

Okay.

If you did receive another set of questions, sometime in the interim, would you publicly disclose that?.

I don't think unless it changed our guidance on timing. I don't think we would comment again on the nature of those interactions. But frequent to the end stages, if you do have back and forth with the regulators, but we continue to have confidence in the process and think it's likely that they will reach an opinion at the next meeting..

Understood. And then, just to follow-up on your -- a point you addressed when discussing the U.S. path forward. You mentioned that there was additional GI data that you already generated.

Can you talk to at least the nature of the data without scooping world? And then, do you have any thoughts on a GI Fabry composite endpoint that a Fabry competitor has been discussing for their pivotal study? I will hop back in the queue after that. Thanks..

The part of that gastrointestinal data that we referenced there, Ritu, is the patient by patient data that we shared at JP Morgan. You referenced that our past -- can be the exact file number but we did show that patient by patient data in the placebo controlled study.

So placebo versus migalastat that showed we think very supportive trends in favor of migalastat over placebo, again, on that patient by patient datasets. There are other parts of the gastrointestinal symptoms that we continue to look at and data that we evaluate that's not been made public and has not been shared with FDA.

Secondly to your question around the GI composite, we also are looking at potential GI composite endpoints to support approval that could be for full approval or potentially to request accelerated approval. So I think there is a lot of ways where that could be an important dataset for us.

And obviously, for others potentially in developing medicine in Fabry. GI and diarrhea in particular is a significant symptom both and life limiting aspect of living with Fabry disease although the leading killers in Fabry of course are cardiac disease, renal failure and cerebral vascular events..

Thanks for taking the questions. I will hop back in the queue..

Of course. Thank you, Ritu..

Our next question comes from Anupam Rama of JPMorgan. Your line is open..

Thanks so much for taking the question. Maybe just a quick one for Chip.

When you are thinking about the cash position and the burn, what's included in the current guidance for potentials [indiscernible] milestones to Scioderm shareholders?.

Yes. Thanks Anupam. The current guidance of $135 million to $155 million is based on the operating cost of our business assuming full success across the programs.

We are not factoring in individual milestones due to Scioderm or any other contingent payment but rather just the prosecution and the full commercialization of migalastat and the other programs..

And again, just to add to that. We continue to believe we will have significant financial flexibility to pay milestones with success of those programs; of course, those were all success based milestone payments..

Great. Thanks so much for taking the question..

Yes, Anupam. Thank you..

Our next question comes from Dae Gon Ha of Leerink Partners. Your line is open..

Hi, guys. Thanks for taking my question. I'm calling in on behalf of Joe. And I guess my name is Dae Gon not Dae John.

Two questions, one to follow up on Ritu's point earlier about migalastat, if you do get the positive opinion from CHMP and a final approval in the EU, what is your expectation of how expensive of a label you can get in terms of covering the various mutations? And I have one question for Pompe.

I know it's kind of early, but at JPM a competitor disclosed an interim analysis on their Pompe program which I'm sure you are aware of.

And just looking at -- looking forward at your program and kind of juxtaposing what they have disclosed, what are your expectations in terms of functional output specifically in the pulmonary side as well as the six minute walk test. I just want to get the sense of superiority or non-inferiority to the current standard of care? Thanks..

Sure. Let me take that first part on the label. We continue to believe that migalastat and the data for migalastat across all of our studies support the use of the drug in all Fabry patients with all amenable mutations. And that is the label that we are pursuing.

Secondly, in terms of Pompe, again, this is a very differentiated treatment paradigm from anything that's approved or any other product in the clinic today. Again, it's an proprietary ERT that is uniquely engineered to have the appropriate glycosylation and a high degree of phosphorylation, so different than any other therapy.

And in addition, it's combined with the oral administration of pharmacological chaperone, which is bind to the ERT and plasma of course. Stabilizes it, enhances activity and importantly, we believe based on preclinical data has the potential to be much more tolerable for patients and have a significantly reduced immune profile.

So as we go into this first series of clinical studies, we want to establish not only that the drug is safe and that patients can safely switch from the current ERT standard of care to this regiment, we want to prove many of those important differentiating factors for this therapy. So we are going to look at tolerability.

We are going to look at immunogenicity. We are going to look at antibodies. We have specifically developed some unique antibody assays not only to characterize the titers, but also to characterize the nature and extent of the antibodies, which we think dramatically limit the efficacy of the current standard of care.

So we will be able to capture all of that in our initial clinical studies. We are also going to do some careful PK modeling, so we think what will be a different PK profile in biodistribution of our drug.

And in addition to all of that, we're going to measure throughout the study and the extension study all of the other traditional functional measures of diseases. So things like forced vital capacity, six minute walk test, MIP, MEP, muscle strength and measured into variety of other fashions. We will look at all of that and then we will see.

We think this will be a very, very differentiated drug..

Are you able to provide, though, in terms of those functional assessments that you mentioned like FVC, MIP, MEP and six minute walk test, what you would consider to be clinically meaningful to demonstrate superiority in the terms of the threshold?.

Let me be clear. This is an additional clinical study. This is not set up as a superiority study. So, and I'm not going to comment on the data from other people that experimenting in the field. I think people can interpret the variety of data those clinical studies over multiple years in many different ways..

Okay. Great. Well, look forward to seeing the data at world..

Yes. It will be very, very exciting at the end of [indiscernible] data here from these patients than a very important program for us and one where we invested enormous resources and we think we have the potential for a very, very differentiated therapy and a disease with such dramatic unmet needs still..

Yes. See you in San Diego..

All right. Wonderful. Thank you..

Our next question comes from Roy Buchanan of Janney Montgomery. Your line is open..

Hi. Thanks for taking the question. Just wanted a little bit maybe insight, I guess on the possible outcomes CHMP process, if you don't get the opinion at the next meeting, are there other possible outcomes and what would timelines potentially look like after that? Thanks..

Yes. Again, I will just reiterate, the final decision on the opinion is in the hands of the regulators. We think it's most likely that they will reach an opinion in their next meeting. Of course, that could always be delayed and it is in their hands, but our expectations are that it would be at the next meeting..

Okay.

Do you think we could know if it's delayed before the meeting or would it become known at the meeting?.

I don't know..

Okay. Thank you..

There are no further questions at this time. I'd like to turn the call back over to John Crowley, Chairman and CEO for any closing remarks..

Great. All right. Well, thank you, everybody. Thanks for listening to this morning's call. A lot of work ahead of us still, but we feel very confident on the path that we are on. Thanks so much. Have a great day..

Ladies and gentlemen, thank you for participating in today's conference. This does conclude the program and you may all disconnect. Everyone, have a great day..