Amicus Therapeutics Inc (FOLD) 2014 Q2 法說會逐字稿

Operator: Good day, ladies and gentlemen, and welcome to the Amicus 2Q Results conference call and webcast. (Operator Instructions) As a reminder, this conference is being recorded. I would like to introduce your host for today's conference, Vice President of Finance, Daphne Quimi. Ma'am, you may begin.

Good afternoon, and thank you for joining our conference call to discuss our second quarter 2014 financial results. Speaking on today's call we have John Crowley, our chairman and chief executive officer; Chip Baird, our chief financial officer; Jay Barth, our chief medical officer; Bradley Campbell, our chief operating officer; and Hung Do, our senior vice president of Discovery Biology.

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

On slide 2 you will find a reference to our Safe Harbor. This conference call contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to business, operations and financial conditions of Amicus, including but not limited to preclinical and clinical development of Amicus's candidate drug products, cash runway, and the timing and reporting of the results from clinical trials evaluating Amicus's candidate drug products.

Words, such as, but not limited to, look forward to, believe, expect, anticipate, estimate, intend, plan, would, should and could, and similar expressions or words identify forward-looking statements. Although Amicus believes the expectations reflected in such forward-looking statements are based upon reasonable assumptions, there can be no assurance that its expectations will be realized. Actual results could differ materially from those projected in Amicus's forward-looking statements due to numerous known and unknown risks and uncertainties, including the risk factors described in our annual report on Form 10-K for the year ended December 31, 2013. All forward-looking statements are qualified in their entirety by this cautionary statement, and Amicus undertakes no obligation to revise or update this presentation to reflect events or circumstances after the date hereof.

At this time, it is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer at Amicus.

Great. Thanks, Daphne, and good evening, everyone. This is certainly an exciting time for Amicus, and welcome to our review of our second quarter 2014 financial results. And, as we've done before, we're going to provide several program updates. Let me begin by saying that there are a lot of good things to review, and this in almost every respect was a very outstanding quarter for Amicus. We have made some very important progress in all of our programs. I'll be speaking a little bit just to address my military leave of absence, but let me begin by just a brief overview before addressing that and then turning it over to our team to take us through a number of these programs.

So, just to highlight some of what we'll go through, our Phase 3, Fabry monotherapy program, of course, in the second quarter we released very compelling 12- and 24-month data from our Fabry Study 011, where we clearly showed the migalastat is having an effect in patients for whom it is intended. So, very good results from that study certainly catalyzed much of our success in the second quarter. We are now eagerly awaiting the top line results from our second Phase 3 clinical trial, the SWITCH study from ERT, our Fabry Study 012. We do expect to have that in the third quarter. The database has been locked. We are at Amicus all still blinded to the data, so none of that has come yet from our contract research organization and team, but it is something we are very much looking forward to here in this quarter.

Also in these slides, and there is some important new data here that we have to share with you. On the progress we've made with our next generation Pompe program. In almost every respect, again, this has exceeded our expectations. You'll hear today from Dr. Hung Do, who many of you have had the chance through the course of this year to meet. We have made substantial progress on the manufacturing scale-up of the Pompe cell line. And we also now this evening are sharing preclinical proof of concept that our enzyme, again, a uniquely engineered form of the Pompe GAA with what we think is a very optimized glycosylation structure and level of mannose 6-phosphate content we believe may represent a significant improvement over the current ERT standard of care.

We have also seen for the first time that the addition of a pharmacologic chaperone to that uniquely engineered Pompe ERT improved the activity over the enzyme alone, and Dr. Do will share some of that data with us this evening. We are on track with that program to select a final product candidate in the second half of this year, 2014, that we expect to move into clinical trials in patients in 2015.

Also in the quarter from a financing perspective we successfully completed the $40 million at the market equity financing. With that financing we strengthened our balance sheet and we also added several top tier new investors to our shareholder base. So, that is a very high-level summary and we'll go into much greater detail with the team taking us through and, as always, be here to answer your questions.

Let me just, before turning it over to Jay to go through some of the Fabry monotherapy data, let me just comment on the news in today's press release about my pending leave of absence for military service. A couple of things to note. This was something that I had volunteered many years ago to serve as a reserve officer after September 11. This was something I was not expecting, but will do my duty as ordered, of course. I look forward to returning to Amicus full time in the first half of next year and fully intend for a long time to come to continue as chairman and chief executive officer of the Company. During this period of my physical absence I will continue to be actively involved in Company activities and major strategic decisions, discussions and the direction of the Company in my role as chairman and CEO. And I can do that because I have absolute confidence in our executive team and the entire Amicus team.

We have never had a stronger team. The board is unanimously supportive of the team, and I have great confidence in the team's ability to execute on what has been a very exciting strategy for Amicus. I'll be happy to take any questions on that at the end as well. With that, let me turn the discussion over to Dr. Jay Barth, our chief medical officer, who will review the monotherapy 011 data from the second quarter, and talk a little bit about what's coming up with the Study 012 and the regulatory strategy there. So, Jay, I'll turn it over to you for slide 3.

Thanks, John. I am very happy to be able to talk to you about the Fabry monotherapy program. There are two Phase 3 studies that are the cornerstone of the program, the FACETS study and the ATTRACT study, but I'll start by reviewing briefly the key elements of those studies. The FACETS study, which we also call Study 011, is the study whose results we reported recently, very good results. The study begins with a six-month placebo controlled phase, and there are 67 patients in total in this study, all naive patients, naive to ERT. And in the 6-month and 12-month data, we looked at kidney, GL-3, the substrate in the kidney. We also importantly looked at clinical data, which I'll get to briefly on renal function at 12 and 24 months.

The ATTRACT study is also called Study 012, the second Phase 3 study, and it's an ERT switch study, which includes 60 patients, randomized 1.5 to 1 for migalastat versus ERT. And these patients are followed over 18 months looking at the clinical endpoint of kidney function. And we're looking at both at measured GFR and estimated GFR, and these data are due in third quarter of 2014 and we will be able to report them as soon as we are able to provide that information to you.

The results from Study 011, the FACETS study, which we reported recently, released in April the 12- and 24-month data, showed a clear effect of the drug in Fabry patients with amenable mutation to reduce substrate and to stabilize kidney function. What we saw in analyzing these data is that patients who switched from placebo to migalastat after being on placebo for six months had statistically significant reduction in kidney, IC GL-3 through month 12, and the patients who had been on migalastat for all 12 months had a durable reduction in the kidney IC GL-3.

Supporting this, another biomarker in this disease is lyso-Gb3. And the same effect was found, a statistically significant reduction in patients who switched from placebo to migalastat and a durable reduction in lyso-Gb3 in the patients who were on migalastat for 12 months. All that clearly demonstrated the activity of the drug. And very importantly, along with this was the clinical outcome of stable kidney function. We saw that both in eGFR and mGFR. They remained stable over 18 to 24 months of migalastat treatment. And throughout this time migalastat was generally safe and well tolerated. So, those were the very good results we could report on Study 011 recently. And what is coming up relatively soon is Study 012, the second Phase 3 study.

What we have told everyone is that we have finalized the physical analysis plan for this study. The comparability of migalastat and ERT in the switch study will be a descriptive assessment, and that will be based on the two co-primary endpoints of 18 months measured GFR and eGFR looking at the annualized change in the measures of renal function. And success is based on two criteria. One is 50% overlap of the confidence intervals between these two treatments, as well as seeing that the mean annualized change for the patients who receive migalastat is within 2.2 milliliters per minute per year, or GFR, compared to patients receiving ERT. And these criteria are based on incorporating regulatory feedback that we have received over time during conduct of the clinical program. That was slide 5. Moving on to slide 6.

The migalastat monotherapy experience is quite extensive at this point. There are 97 patients today who take migalastat as their only specific treatment for Fabry disease. In all, 143 patients, Fabry patients that have taken migalastat, for a total of 377 patient years of treatment. And two important factors to note are the duration of treatment. We have very long-term data on some of these patients. Some out to 8.5 years who have continued on migalastat without receiving ERT during that time for the Fabry disease. And I think an indication of the effect of the drug and tolerability of the drug is that there is a very high retention rate for patients enrolling into optional extension studies, 96% of patients have elected to do so.

Slide 7 is an overview of the regulatory strategy for the monotherapy program, and we have a very clear path in Europe, presuming positive results from the 012 Study that we will find out shortly. We are ready to move forward quickly in Europe with an MAA submission. The basis of the MAA submission will be noninferiority or comparability of migalastat to ERT from Study 012, but it is also supported by 011 and all the other data that we have gathered in the clinical program.

At the same time, we are moving ahead in the US. We will meet with the FDA in fourth quarter is our target, and we will be discussing with them the fastest regulatory path for migalastat monotherapy in the US. And we know that the FDA will be looking at the totality of the data that is based primarily on the two Phase 3 Studies 011 and 012, but the long-term data will also be an important factor in the submission that some patients have been receiving this drug, migalastat, for 8-plus years at this point. So, we are very much looking forward to moving ahead in Europe and in the US as part of our regulatory strategy. So, with that I will turn it over to John to continue the call.

Great. Thank you, Jay. That is a terrific overview of where we are in the monotherapy program. Before we leave monotherapy, though, I do want to share some very interesting work that our science team has been doing to better understand and characterize migalastat's mechanism of action. As we know, we have said for some time now that this is a unique way to treat Fabry disease, and we think -- we hope it will be an advancement for patients. Certainly we think more convenient in its oral form, oral bioavailability of the drug. We also, though, have understood that patients seem to tolerate the drug very well, so that also plays to the benefit. But when it comes to the core understanding of the mechanism of action, we focused quite a bit on the notion that this is a small molecule that specifically targets the patient's own enzyme, providing catalytic competence in chaperoning the enzyme, elevating enzyme activity and reducing substrate. That is very clear in the 011 Study and particularly was very clear in demonstrating that.

But we have also been working to understand what we think could be another potential added benefit of the migalastat mechanism of action in terms of its chaperoning function and its reduction of cellular stress and the pathways involved within patient cells. So, with that I'm going to turn the call for the next slides over to Hung Do, who will walk us through the mechanism of action and the potential implications for Fabry patients. So, Hung, I'll turn it to you for slide 8 here.

Great. Thank you, John. So, as John alluded to, much of our focus actually has been centered on understanding the appropriate patient population and how to dose this particular small molecule to have a significant increase in the patient's own endogenous enzyme at the right (inaudible) is able to get out of (inaudible) its made and to the lysosome, where it actually needs to function. So, this is actually shown in slide No. 8. What is known and we actually are continuing to understand this better with different diseases is that when a [genetically] patient causes a change in amino acid sequence with resulting protein, there is significant problems with that protein folding, such that the protein causes certain levels of distress within the cell, which is typically known as the unfolded protein response. This actually can have some pretty significant problems in the cellular pathology such that there is an adaptive response called ER stress, which in turn increases the expression of other helper proteins. These are proteins which are often referred to as chaperone proteins, which help to deal with this unfolded protein, so that these proteins can fold better and able to reduce the particular cellular stress.

We have not spent too much time understanding this, but we are starting to do some work to understand whether our small molecular pharmacologic chaperone cannot only increase the protein trafficking but also can reduce some of the cellular stress.

We have actually done some work as well as other research labs around the world, as shown in slide No. 9, to understand this particular phenomenon. In this particular slide, what we did was to understand whether small molecules can improve the protein trafficking of the mutant protein out in the (inaudible), which in turn -- it actually can result in reducing some cellular stress. What is shown in this particular slide is that the protein, the mutant protein in this particular case, a mutant form of the a-Gal A protein as deficient in Fabry disease. By administering the pharmacologic chaperone, what we actually observed is it has a cellular [localization] that is much more similar to wild type. But what is also interesting is that it also reduces the amount of these protein chaperones, in particular, a protein that is called BiP. This we take as a sign that the particular small molecule chaperone is actually improving the cellular stress, that is, reducing the cellular stress in the ER. We are continuing to understand this particular phenomenon better and we hope to have some additional data in the near future.

So, moving on to slide No. 10. While we don't fully understand the ER stress and whether our chaperones can actually mitigate and improve this particular problem, there is an abundance of data out there that indicates that unfolded protein, ER stress, does in fact contribute to nephropathy. And there are significant ER stress markers which have been identified in various kidney samples in some patients.

We also see that there are significant inflammation markers which are shown in lysosomal storage diseases as well as different proteins that accumulate in the ER, as a result of ER stress. We hypothesize that utilizing the pharmacologic chaperone, this actually could have multiple benefit, one, by improving protein chaperone -- excuse me -- improving the protein trafficking of the mutant protein out of the ER so it actually can increase the (inaudible) levels in lysosomes. But in addition can also reduce ER stress to mitigate some of these problems. So, with that, let me turn it back over to John so he can provide an overview for the Pompe program.

Great. Thanks, Hung. That was a terrific overview and hopefully everyone gets the sense that we continue to have the effect in patients we believe by hitting the target, elevating enzyme activity, and reducing substrate. We think that was made very clear with the 011 data and numerous preclinical studies that we have conducted. We think we can do that as well, and in some tissue types, given the targeting and the nature of the small molecule potentially maybe even better than ERT. What we do know, though, is that we can chaperone the person's own native enzyme through those trafficking pathways that Hung just highlighted, and we think that, again, that might have a significant benefit for patients. But more work to be done there, but it's been some terrific work coming out of our science groups recently and we thought important to share as we head into this next level of clinical data.

Let me go ahead now and transition on the next slide, slide 11, to Pompe disease, and I'll do this before I hand it over to Bradley to provide an overview of our ERT programs and status. So, Pompe is a disease that we've known for a long time. I've known it personally since 1998, and professionally since the year 2000. It is a devastating disorder ranging from infant all the way through adulthood. It is one of the more prevalent of the lysosomal storage disorders. We believe that the current enzyme therapies, which many of us were involved in from the start, are a good first step toward treating the disease, but that we have a long, long way to go. And if you look on the next slide, we just provide some perspective here on slide 12 of, in many ways, how far we've come as a community in development of Pompe drugs.

You can see the first of the cell lines developed back in the mid '90s. Hung and I first came to work together on the same day. It was March 24, 2000, when we both started at a tiny three-person company in Oklahoma City called Novozymes, where were using some unique glycosylation and glycobiology technologies to begin working on a Pompe enzyme. Within about 18 months of the founding of that company we were acquired by Genzyme, and with that I went, as did Hung, to Genzyme, as did Bradley Campbell at that time, to move from Novozyme to Genzyme to further the development of a drug for Pompe disease. Hung stayed with that effort for many years and then we both came to Amicus in 2005, and Bradley joining us in 2006, and other members of our previous teams. And as we began to develop the notion that we can combine a chaperone with an enzyme replacement therapy to enhance stability and activity, potentially reducing immunogenicity, improving tolerability, we began to think about what are the other ways in which we can improve on the Pompe drug?

So, I'm not going to recite everything that is on this slide. We know the history of the development of Myozyme and Lumizyme, other drugs in development. I provide this just as background so everybody understands this is a team that has been together for a long time in a couple of different entities, now with the acquisition of Callidus in the fourth quarter of last year, Hung rejoining Amicus as our senior vice president of discovery biology and biologics. With his skillset and the team that we have assembled, we think we have something very unique in the field to contribute, and that is a program we are very excited about. Hung is going to join us in a little bit to talk more about some of the success that we have had there. So, with that I will turn the call now over to Hung, who will review some of the data that we have.

Thank you, John. And so I think for us, through all of our experience, as John alluded to, over the past 15 or 16 years with Pompe disease, we actually have come to understand so much more about this enzyme and what actually is required, but it's going to take ERT to be an effective approach for treating Pompe disease.

And so on slide No. 13, this actually is a way for us to better highlight the major problems with this particular enzyme. What is shown in this particular slide is that we use a receptor column, that is to say, this is the intended receptor which is known to be utilized for internalizing the ERT, able to get into (inaudible) and be delivered to (inaudible). What is interesting is these ERTs require a specialized carbohydrate structure called mannose 6-phosphate. And so what we have done in this particular experiment is that we utilized the intended receptor, mannose 6-phosphate receptor which then we put onto a column and then just measured how much of the ERT is able to bind the receptor.

So, the top panel actually is the standard of care. What is shown here is that when the enzyme is put onto this column, the fraction of enzyme that does not contain mannose 6-phosphate cannot find the receptor and therefore flows through the column. The fraction actually does contain mannose 6-phosphate (inaudible) the column, which then we elute with excess amounts of free carbohydrate mannose 6-phosphate, therefore out-competing the binding. And so what is seen on the top panel is that the standard of care only contains a small fraction of the enzyme, which contains mannose 6-phosphate. This is one of the major reasons why this enzyme needs to be dosed at such high dosages for it to have some clinical benefit.

With this understanding, Callidus was founded with the notion of trying to improve the drug targeting for this enzyme as well as for other ERTs that are in development. And what we actually have done is to develop a proprietary cell line such that the enzyme that is produced from this cell line is made naturally with significantly higher amounts of mannose 6-phosphate, both in terms of quantity but in terms of the actual structures, which now are known to have very high affinity for the intended mannose 6-phosphate receptor. As shown in the bottom panel, the vast majority of the ERT which we designate as ATB200, is able to bind the receptor. This, then, implies that the vast majority of the enzyme can effect binder receptor (inaudible) cells, a (inaudible) target cell is able to be targeted to lysosome. This in fact is the reason why we believe this enzyme has so much potential in terms of improving the drug targeting with this enzyme.

So, if you would go to the next slide, what we did is review a number of different (inaudible) studies to compare head-to-head our enzyme with the standard of care. In this particular study what was done is that the enzyme, our enzyme, ATB200, was compared to standard of care at the prescribed dose of 20 milligrams per kilogram while our enzyme was dosed at 10 milligram per kilogram, or 20 milligram per kilogram. And so what is shown in both the quadriceps and the triceps these animals that are knocked out, that are genetically modified to not have an active GA enzyme as deficient in Pompe disease, the residual substrate glycogen in this particular case, accumulates in the muscles of these particular mouse models. And what is seen is that significant glycogen accumulates without any ERT, which is shown in the black bar. So, the green bar represents the standard of care, and as you can see there is significant decrease in the amount of glycogen in these particular animals as a result of Lumizyme ERT.

But what is interesting and as we compare ATB200, even at lower doses of ATB200, there is significantly more glycogen reduction, and then it is equal, there is equivalent doses. The ATB200 far exceeds the effectiveness of glycogen clearance in comparison to Lumizyme.

What we also did in this particular study is that we utilized the combination of the pharmacologic chaperone to understand whether having the pharmacologic chaperone can stabilize enzyme and improve the amount of active enzyme that gets internalized into cells [in large amounts]. And what we see is that a combination of chaperone further improves the glycogen clearance in these particular mouse models. Again, these are data that demonstrates proof of concept that an enzyme that has better glycosylation that is better targeted in combination with a small molecule to stabilize the enzyme far exceeds the effectiveness glycogen clearance in comparison to standard of care.

And so what we have also done for this particular strategy, not only have we been able to improve the glycosylation of ERTs, we also have different ways in which we can add a targeting peptide onto ERT for ones that are actually not containing the proper glycosylation. And we continue to do initial work for manufacturing on that front as well. And so we believe that we have multiple ways in which we can improve drug targeting for ERT, as well as improving the stability and tolerability of these ERTs in combination with pharmacologic chaperone. And so with that, let me turn it back over to John to continue our discussion.

Great. Thanks, Hung, for that overview and for all the terrific work that you and the team have put into this certainly in your time back now with Amicus. This has been another part of our progress this year, but certainly building on many years of experience and a lot of passion going into making a better medicine in Pompe.

Lots of work has gone into all of our ERT next-generation programs, and on slide 16, Bradley will take us through some of the highlights. Go ahead.

Great. Thanks, John. So, as John mentioned, on slide 16, let me just first provide an update on our Fabry next-generation ERT program, which is, of course, co-formulated with migalastat. We continue to make great progress towards beginning a Phase 1-2 study in Fabry patients, and to that end we have successfully completed two key operational milestones including the completion of migalastat IV PK study, and also final manufacturing of drug product of that co-formulated product. And we continue to work closely with investigators on a protocol design.

In parallel, as we have mentioned before, we have also been evaluating sources of long-term supply of Fabry enzyme. And as you saw in the press release, at this point we have several ongoing promising business development discussion underway, and so we have made the strategic decision to wait to begin the next Phase 1-2 pending the outcome of those discussions. As soon as we have further clarity there, we will provide an update to all of you on the next steps for the program.

On a related note, I am pleased to announce that Dipal Doshi has recently joined Amicus as senior vice president of business development and business planning. Dipal brings over 15 years of operating, investing and business development experience in the biotech and healthcare industry, and he is a great addition to the management team and to our efforts along these lines.

And then, finally, just a quick comment on our Parkinson's program. As you know, we have had an ongoing effort to develop pharmacologic chaperones that target GCase for the treatment of Parkinson's. We previously had initiated a research collaboration with Biogen Idec to discover and develop a class of novel small molecules that target the same pathway. At this time we have agreed with Biogen to conclude that collaboration in September. However, importantly, Amicus does retain all global, worldwide rights to our most advanced Parkinson's compound, AT3375. With that, I will turn it over to Chip to walk us through our financials.

Great. Thanks, Bradley, and good afternoon, everyone. As John mentioned at the top of the call, we significantly strengthened our balance sheet in the second quarter through successful execution of a $40 million at-the-market equity financing. We began sales of primary shares under the ATM in May and sold the final block of shares on July 1. As a result, we have elected to show two columns on slide 17, the first being the official June 30 balance sheet numbers, as well as the July 2 balance sheet numbers, which show the effect of the final proceeds received from the ATM financing. As you can see, the June 30 cash balance was $78 million, which is compared to $82 million at December 31 of last year. But with the final proceeds from the ATM, we had $98.4 million in cash and cash equivalents as of July 2.

In terms of additional sources of funding, we still have $10 million remaining available under the debt facility we entered into in the fourth quarter of 2013.

As previously guided, we expect full year 2014 net cash spend to total between $54 million and $59 million. With the proceeds from the ATM in hand, we believe that our current cash position is sufficient to fund our current operating plan into 2016.

Turning to our second quarter 2014 financial results on slide 18, I will be referencing tables 1 and 2 in the press release which we issued earlier today. And additional details can be found in our quarterly report on Form 10-Q, which will be filed later this evening.

We recorded modest revenues of $475,000 in the second quarter of 2014 in conjunction with the Biogen collaboration. Total operating expenses for the second quarter decreased to $14.7 million compared to $16.0 million for the second quarter of 2013. The year-over-year decrease was primarily due to decreases in personnel costs as well as decreases in contract research costs.

The net loss attributable to common shareholders in the second quarter of 2014 was $14.6 million compared to a net loss of $15.3 million in the second quarter of 2013. Net loss per share of $0.22 in the second quarter of 2014 was narrower than the net loss per share of $0.31 in the second quarter of 2013. The narrower net loss and net loss per share versus the year-ago period is primarily attributable to the lower overall loss and an increased share count as compared to the year-ago period. As of June 30, 2014, we had approximately 72.9 million shares outstanding compared to 49 shares outstanding in June of 2013.

This summarizes our key financial results for the second quarter of 2014, as well as our full year 2014 guidance. Happy to answer any questions during the Q&A session, but for now we will turn the call back to John.

Great. Thank you, Chip. And just on the last slide here before we open it up for Q&A, just regarding the continuity of leadership at Amicus during my military deployment. Again, just to summarize, I will remain chairman and CEO. I expect the leave of absence to be about 32 weeks. I will deploy, we expect, by the end of September, again, deployment to Afghanistan. And I think for these deployments, they are always toughest on those you leave behind. I will be perfectly fine and safe. In fact, in the last 90 minutes or so since the press release, I've gotten some wonderfully kind e-mails from people, and I assure you my sacrifice is very, very small compared to everyone else who has served in those theaters. In many ways I continue to serve in the company of heroes.

You always, of course, are concerned about those you leave behind, and just as I am absolutely confident that Aileen will take terrific care of our three kids while I'm deployed, I am also absolutely confident that Bradley and Chip and Jay and the senior team and everybody at Amicus will continue to perform all of their duties and continue to do an excellent job managing Amicus. I still expect to be available for, again, some of the major strategic decisions and business issues. This is a very exciting time and for very selfish reasons I'm sorry I'm not going to be there for this physical absence, because I think this is going to be yet another extraordinary couple of quarters for Amicus. Which means I'm all the more eager to come back in the first half of the year and resume my full-time duties as chairman and CEO.

So, again, it was a terrific quarter. We hope we have just as exciting and positive a quarter in 2014 and in the quarters ahead of us. With that, Operator, we are happy to open it up to questions.

(Operator Instructions) Our first question comes from Mike Ulz, JPMorgan. Sir, your line is open.

Hi, guys. Thanks for taking the question. This with Phase 2 data from the 0l2 study coming up here in the near future, can you maybe comment on what you guys see as sort of the biggest risk to a positive outcome for that study?

Yes, sure. We have considered lots of potential outcomes. Of course, Mike, we are hopeful and confident that the data will be where we need it to be. And, again, we are still blinded to all of that. The patient disposition numbers have been very, very positive. Jay, I'll let you comment specifically to any risks with the study and how we mitigated those risks.

I think we've learned so much from the 011 study that we've been able to minimize any risks in the 012 study. We know how to analyze the data, that's probably the most important thing that we've learned from 011. And we know from the experience of the investigators, most of whom are involved in both studies, how the data are collected. I am referring to the renal function data. And because of the similarities of the study in terms of what the type of patient, the measuring that is being done, the duration of the study, we feel very good based on the results we saw in 011 about what's upcoming in 012. And we could apply everything that we learned in the conduct of the first study and the analysis of the 011 to apply to the 012 study. So, I really think we're in quite good shape and that's why I'm very optimistic about the data that is coming up in 012.

Just to add to that, Mike, early on in the planning of this study we had learned from some of our Phase 2 studies as well. So, for instance, patients were carefully randomized by ERT, by sex, even by levels of proteinuria at baseline.

Great. Thanks, guys.

Thank you. And our next question comes from Mr. Joseph Schwartz of Leerink Partners. Your line is open.

Great. Thanks very much. I was wondering, since I see that you finished the Phase 1 trial for IV migalastat, if you could characterize those data, because I remember when oral migalastat was studied in a co-administration strategy, you needed to give the drug ahead of time in order to get the drug onboard approximate to the time that the enzyme would be available for mating with the chaperone. So, what did you learn from this study and how are you going to use this information when you go into Phase 1-2?

Thank you, Joe. Jay, maybe if you'll take the first part of that, just outlining the work that was done and what was the significance of the study, and then Bradley, maybe you and Jay can together answer what it means for future development of a next-generation Fabry ERT?

The study had really helped us define the dose. Going forward, we understand the pharmacokinetics of the IV dosing of migalastat, and that's really information that we needed in order to make sure we're going into the next study with the data that we need. So, we understand the PK of the IV dose, we understand how to dose it and what the regimen should look like.

Yes, Joe, I mean, as you articulated with the oral form of migalastat, it limits you in some ways in how you can deliver it and what time you have to deliver it. And from a long-term perspective, both from getting the exact right dose of the IV migalastat plus the dose of ERT, but then also from a clinical and commercial perspective, not having to stay 30 minutes ahead of your infusion, take the oral dose of migalastat. There are a whole host of reasons why we believe having that IV form was the prudent way to go. And so this study was, as Jay mentioned, was successful, and we got the information we needed to design the next Phase 1-2 study.

Okay, great. I'll look forward to seeing more as that advances. Thank you.

Thank you. (Operator Instructions) Our next question comes from Kim Lee of Janney Capital. Your line is open.

Good afternoon. Thanks for taking the question. I have a question on Parkinson's disease. Since you will be concluding your collaboration with Biogen, can you tell us what next steps are for this indication and what your plans are? Thanks.

You know, Kim, this was an early stage research program that built upon a couple of years of work that we had been doing at Amicus. I think it was a good collaboration. We enjoyed working for the one-year period with Biogen. From our perspective, we had about six or seven full-time Amicus employees who were engaged in this project while paid for completely by Biogen. As we were advancing and our next generation ERT programs are advancing, we really needed the bandwidth within the Company. In addition, since not renewing this partnership for another year gives us the rights to take back these molecules. It, of course, preserves the right to use the 3375 molecule and others in a next-generation cache ERT. So, as we build up our pipeline, we think that could be a very important toolset for us.

So, in terms of Parkinson's going forward, we are in the process of evaluating that, what we will do with that program specifically, but we don't yet have any further comments on where we will take Parkinson's.

Okay, great. Thank you.

At this time I see no further questions in the queue. I'd like to turn the call back over to you, Mr. Crowley, for any closing remarks.

Great. Thank you, Operator. Thank you, everybody, for an excellent call, and to my Amicus team here. That's all we have. Everybody have a great night. Thank you.