Amicus Therapeutics Inc (FOLD) 2014 Q1 法說會逐字稿

Good day, ladies and gentlemen, and welcome to the Amicus first-quarter 2014 results conference call and webcast.

(Operator Instructions)

As a reminder, today's meeting is being recorded.

I would now like to introduce your host for the conference, Miss Sara Pellegrino, Director of Investor Relations. Miss Pellegrino, please go ahead.

Thank you. Good evening and thank you, everyone, for joining Amicus Therapeutics' conference call to discuss first-quarter 2014 financial results.

Speaking on today's call, we have John Crowley, Chairman and Chief Executive Officer; Chip Baird, Chief Financial Officer; Jay Barth, Chief Medical Officer and Bradley Campbell, Chief Operating Officer.

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

Starting on slide 2, you will find a reference to our Safe Harbor Statement. 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 candidate drug products, cash runway and the timing of the results from clinical trials evaluating Amicus candidate drug products.

Words, such as, but not limited to, look forward to, believe, expect, anticipate, estimate, intend, plan, would, should and could and similar expressions or words identify forward-looking statements. Although Amicus believes the expectations reflected in such forward-looking statements are based upon reasonable assumptions, there can be no assurance that its expectations will be realized.

Actual results could differ materially from those projected in Amicus forward-looking statements due known and unknown risks and uncertainties, including the risks 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. Amicus undertakes no obligation to revise or update this presentation to reflect events or circumstances after the date hereof.

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

Thank you, Sara, and good evening, everybody.

We, of course, had a very important update call on our Fabry Study 011 results last week. So through this call, I will shortly turn it over to Dr. Jay Barth, our Chief Medical Officer.

We see there are a number of new callers in who may not have had a chance to review the data with us live on that conference call last week. So in a much more expedited fashion, we will go through a high-level overview of that data and then I'll turn the call over to Brad Campbell, and to Chip as well, to go through some the operational updates. Brad, in particular, will take us through the great progress we've been making with our next-generation enzyme replacement therapies.

Let me begin though, by just reiterating what we stated last week, and again within the press release just issued after market close today, that last week really was we believe a momentous occasion for Amicus. As we announced a very positive 12 and 24 month data from the first of our two Phase III studies for Fabry monotherapy, our drug migalastat for Fabry disease.

We had a chance, as we indicated on that call last week, to review that data with a handful of investigators under great confidence prior to the data being made public, and since then, have had a chance to investigate the data with an even larger number investigators and key opinion leaders, and again, the results have been greeted with great enthusiasm in the investigator and physician and science communities. We will continue to describe these data to that community, and of course, in the early fall, hopefully present this data at a major science conference.

We've also had a chance to vet it with key opinion leaders in the Fabry patient community, and again, that's been an equally enthusiastic response, and again, we also think is a great testament to the quality of the science and medicine and our wonderful partnerships throughout the world. Again, remember that study 011 was a multi-year study conducted at 37 sites in over 17 countries on five continents, so this was a true team effort to run this study.

Let me turn your attention to slide number 4, just a high-level overview again, of what we saw with the study 011 results, why we're so enthusiastic about it, why we think we now have a path forward for the drug toward approval, the first step of that path in multiple geographies, including the United States, once we get the 012 data here in the next quarter. So if you look at this data, the migalastat demonstrated a statistically significant and durable reduction of substrate on the 12 month prespecified primary analysis in Fabry patients.

Again, I'm not going to read everything that's on this slide, but just to reiterate that on that primary analysis in the subject to switch from placebo to migalastat after month six, that they demonstrated that statistically significant reduction in the kidney interstitial capillary GL-3. Again, the substrate that builds up in people living with Fabry at month 12. Again, the P value there 0.013.

We were also very encouraged that the subjects who remained on migalastat for 12 months continue to demonstrate the effect of the drug at that time period. The nice thing, too -- and I just had several discussions and interviews with folks outside the Company today, and wanted to reemphasize that we think it's a very important and clinically relevant biomarker of Fabry, this measurement in GL3, in the interstitial capillaries of the kidney, but we think the study's so positive because it's not just one endpoint, although it, of course, was the primary prespecified analysis.

The secondary endpoints line up, we think, very well this prespecified primary, specifically the plasma lyso GB3, which is an increasingly important biomarker of Fabry disease, again with a very high P value here, 0.0001. I think this will be a very important biomarker going forward. It's a relatively new biomarker in the disease first published in 2009. And one that's gaining quite a bit of popularity in the physician community.

Also too, and we think very significantly -- and this was a very important set of data for the nephrologists, who are experts in Fabry, who we've edited this data with, and that's in the kidney function both by the eGFR measures and the IOXRGFR, the measure GFR, that it remains stable over the 18 to 24 months end periods for the primary study here.

So we think that it's very distinct, potentially from the current therapies and certainly very distinct from the natural history of the disease. Of course, very pleased with the safety and, as we double down on this drug in the second half of 2013, one of the things that gave us confidence to do that, was the compliance of patients who were coming on to the drug and choosing to stay on the drug, some now for the history of our studies for more than eight years. And a total of more than 300 years of patient data, including now the 35 of 41subjects with a minimal mutations who completed the two year study period in this study 011 who remain in the voluntary extension.

So for all of that, we think very positive results in this study. We hope it bodes well for our study 012, which of course is the none inferiority switch study for ERT, and we'll talk about and be happy to take any further questions on that study. We remain aligned to the 012 data that read out in the third quarter of this year.

With that, as an introduction, let me go ahead in the next few slides and turn it over to Jay Barth, our Chief Medical Officer, who will, again, just highlight some of the data we saw in this 011 study.

Thank you. I'm very pleased to have the opportunity to review this data with you briefly from study 011, starting with slide 5.

These are the key results from the study in patients with [amenable] mutations and that was new data out to 12 months. And there are two important comparisons that I would want to draw your attention to.

First, is from baseline to six months, and these are the double-blind placebo-controlled phase of the study, the patients who are on placebo are in the red-dashed line, patients receiving migalastat in the solid blue line. And you can see a statistically significant decrease in kidney ICGL3, in the patients who are receiving migalastat and the P value for that comparison 0.008.

And these patients, continue to the solid blue line, had a durable affect. They maintained this effect over the 12 months of the study period.

The second comparison, and this is the primary analysis of these new data, are the patients who are on placebo for 6 months, red-dashed line, and then switch to migalastat between month 6 and month 12, the blue-dashed line. These patients experienced the same decrease in kidney IPGL3 as those both who had gotten migalastat in the first six months, and the P value for the comparison of patients who switched from placebo to migalastat, P 0.013.

And seeing the same effect in both of these groups replica, really confirms the activity of the drug. So they're multiple statistically significant and durable effects that demonstrate migalastat activity in kidney ICGL3. The same effects were seen -- I'm turning now to slide 6 -- same effect in the other substrate disease, which John mentioned, lyso, plasma lyso GB3, which is really a very important emerging biomarker in Fabry disease. We have the same comparisons on this slide.

In the first six months of patients who received migalastat solid blue line versus placebo in the dashed-red line, showed a statistically significant decrease in patients who were on migalastat the P value 0.0033. And again, the patients who switched from placebo red to migalastat at month six in the blue-dashed line, showed the same decrease in plasma lyso GB3, that patients receiving migalastat did and a comparison less, P value less than 0.001. So looking at both kidney ICGL3 and lyso GBS, there's strong evidence of migalastat activity in both of the groups that received migalastat.

It's very important, as well as to look at the functional outcome, kidney function. And it's well-known that renal dysfunction is a major cause of morbidity and mortality in Fabry disease, so it's very important to look at GFR, glomerular filtration rate.

We measured it by two methods of eGFR estimated GFR, CKD-EPI and MDRD, as well as measure GFR, MGFR using iohexol. And in all methods, the GFR was stable over 18 to 24 months of treatment, in contrast to what's known to occur, the decline in untreated patients, and compares favorably to what is known to occur in healthy adults who decline approximately one per year, depending on age. So it's very encouraging to see the stabilization of renal function over 18 to 24 months on migalastat.

Moving to slide 8, a summary of the safety profile shows that migalastat was generally safe and well-tolerated. You can see that, for the common adverse events, those at least 10% of subjects, were similar to placebo in stage I and there were no safety signals that arose in stage II, that is month 7 to 12, or the open label extension up to 24 months.

So these are important data when considering the benefit risk profile of the drug that no major concerns really arose on the risk side of the equation when looking at the data for migalastat of the 24 months.

Slide 9 is a overview of the global regulatory strategy. Of course, we're pursuing the regulatory strategy both the US and in the EU. And in the US, the strategy is based on looking at the totality of the data, which includes of course these promising data from study 011 and the upcoming data from the other Phase III study 012, which is looking at renal function, GFR, those data to be available by third quarter, next quarter of this year. And we will be, of course, conducting further discussions with the FDA to pursue the strategy in the US.

At the same time, in the EU the regulatory pathway is even clearer. The 011 data will still be part of the data package. EU's strategies primarily rest on the results of study 012, noninferiority study to ERT looking at renal function for migalastat, compared to ERT, and assuming success in study 012 we'll be unable to proceed with this regulatory pathway in the EU.

With that, back to John.

Thank you, James. It was an excellent overview. I will now turn the call over to Brad.

Brad, I think you have a number of milestones highlighted here on slide number 10, shifting now for migalastat monotherapy to a focus on our next generation of Fabry ERTs, what we call our 3-in-3 strategy; one ERT into the clinic every year for the next three years beginning this year with our next generation Fabry ERT.

So, Brad, I'll turn it to you.

Sure, great, thanks, John. Good evening, everybody. I'll just add a little bit of a color to the milestones that are listed on slide here.

For Fabry, as John mentioned, we do remain on track there to start our clinical development in the second half of this year. This would be a product for the roughly half of the population who don't have addressable mutations with migalastat as a monotherapy. This would, of course, support our vision that eventually all patients with Fabry disease may take an Amicus product as their only treatment for their Fabry.

We also continue to make great progress in Pompeii and development of our next-generation [enzyme] replacement therapy there, to address what we see as really the key three major challenges in enzyme replacement therapy today, that is activity and stability, uptake and targeting and tolerability and immunitisity.

We've already made the progress in initial preclinical studies of our proprietary GAA cell line, ATV200, with its unique carbohydrates structure. We've shown in additional preclinical studies that we can demonstrate superior uptake and activity, compared to the current standard of care, which we believe may also be further optimized through coformulation with our chaperone 80-2220, to further improve stability and tolerability and also by applying the companies peptide tagging technology for better targeting.

Manufacturing scale up activities and preclinical studies continue to move forward on schedule, and we can expect to make selection of our final drug candidate for IND-enabling tox in the second half of this year to support clinical studies, which we expect might begin in 2015. And then finally, we do continue to move forward with our MPS I program. We continue to advance preclinical studies working to develop proprietary recumbent human IDUA enzyme as the next generation therapy for MPS I.

So great progress towards our 3-in-3 strategy, and each of these milestones remain on track.

Thanks, Brad, for the overview. I'll turn it over to Chip, now for a discussion on the financials.

Great thanks, John. Good evening, everyone.

I'll start today's financial discussions with a few comments about our current cash position and guidance, which is shown on slide number 11. As indicated in this afternoon's press release, Amicus continues to maintain a strong balance sheet. At March 31, we had $71.6 million in cash and cash equivalents, which compares to $82 million at the end of last year.

In terms of additional sources of funding, $10 million remains available under the debt facility we entered into in the fourth quarter of last year. We also have an at-the-market or ATM equity facility in place, which became effective March 4, 2014 and covers the offering of up to $40 million of our common stock issues sold under the sales agreement with Cowan.

We believe it's a financially prudent practice to have both our effective shelf and an ATM facility in place. Today we haven't sold any shares under the ATM agreement.

And as previously guided, we expect full-year 2014 net cash spend to total between $54 million and $59 million. We believe that our current cash position is sufficient to fund our operating plan into the second half of 2015.

Turning to our first-quarter 2014 financial results on slide 12, I will also be referencing tables 1 and 2 in the press release, which we issued earlier today. Additional details on the financials can be found on our quarterly report on form 10-Q which will be filed later this evening.

We recorded modest revenues of $456,000 in first quarter of 2014, in conjunction with the Biogen collaboration, which continues to progress very well. Total operating expenses for the first quarter 2014 decreased to $16.1 million, as compared to $17.3 million in the first quarter of 2013.

The year-over-year increase was primarily due to decreases in personal costs, as well as contract research costs. Moving down the P&L, we had a nonoperating expense of $322,000 in the first quarter of this year, compared to nonoperating expenses of $207,000 of last year. The change is due primarily to an increase in interest expense on the outstanding debt.

Net loss contributed to the common shareholders in the first quarter of 2014 was $15.9 million, compared to a net loss of $17.5 million for the first quarter of last year. The per share net loss of $0.25 in the first quarter of this year was narrower than the net loss per share of $0.35 in the first quarter of 2013. The narrower net loss and net loss per share versus the year-ago period is primarily attributed to the increase in shares outstanding following our private placement of common stock and the acquisition of Callidus Biopharma during the fourth quarter of 2013.

As of March 31, 2014, we had approximately 64.4 million shares outstanding, compared to 49.6 millions shares outstanding in March of 2013. This summarizes our key financials for the first-quarter of 2014, as well as our full-year 2014 guidance.

Now if you'd address any questions on the financials during the Q&A session, but for now, I'll turn the call back over to John.

Great. Thanks, Chip.

So we will shortly here get to the Q&A, I just want to summarize by stating that I think we're in a really good position at Amicus. I was just so happy to see the results, and as I stated in the beginning, we've had a chance over the last two weeks with the investigators, with some key opinion leaders and now with leaders in the patient community, to take them through the data on this 011 study, and specifically to describe in more detail what we're doing in the study 012, where we expect those results from the 18 month primarily endpoint here in Q3, and as Jay described, with our respective strategies for the US and the EU.

When you think about that, we could be in a position in the second half of this year to have, hopefully, a successful phase III monotherapy program and a program that is unpartnered, it is an Amicus program with very modest royalties in most geographies. That is our program that we could drive forward. We absolutely could commercialize the program.

There's a significant amount of potential partner interest that we're considering, although we said to those potential partners and to the broader public here, that our bar for partnering would be very high. And with that, also in the second half of the year, it is going to be a huge occasion for Amicus to finally put our next generation ERT that we've been working on for several years into the clinic, beginning with our next generation enzyme replacement therapy in Fabry.

So as you think about Fabry disease, currently $1 billion market for the existing therapies. We will have two, we hope, product offerings for patients, and the idea would be to build a franchise in Fabry disease with our late stage monotherapy program together with our ERT program, monotherapy, of course, for people with amenable mutations, the use of the pharmacogenomic aspect of the monotherapy to determine those patients in advance.

And for the roughly other half of the population, to be able to provide them with an enzyme replacement therapy that we believe has the potential to be a superior therapy. Of course we'll put that in the clinic and test that hypothesis in man, but a very exciting time as we head into the middle part in the second half of the year.

And with that, Operator, we will be happy to take any questions.

Thank you.

(Operator Instructions)

Ritu Baral, Canaccord.

Hi guys, thanks for taking the question. As we look forward to the Q3 data, can you help give us an idea of what you consider noninferiority as we look at the data that comes out? And what are the ranges or -- the ranges of variability for the different renal function measures that you outlined in the slides? The three different -- ?

Sure. I will let Dr. Barth take the second half of that question as we think about some of those ranges. But just to remind folks on the upfront that this was a study that began enrolling in the early part of 2012 right after we had finished study 011. And at the JPMorgan conference in January of 2012, people asked me what kept me up at night?

We had a great conference with some of our new ERTs and (inaudible) proof of concept data and I said what kept me up at night was the enrollment of Study 012, that we had to convince patients and their physicians to completely give up ERT and for those patients with amenable mutations to switch to migalastat as their only treatment for Fabry disease. We were remarkably successful in enrolling that study. I think it speaks to the level of interest in the patient and physician communities to try a new therapy and to experiment, at that point, with our molecule migalastat.

It was designed to be a 50-person study; we enrolled it in less than a year. We overenrolled the study at 60 patients; it is a 1.5 to 1 randomization. So that we took 36 of those patients completely off of the ERT, switching them to migalastat as their only treatment for Fabry. 24 remained as a control arm on ERT. The primary endpoint is looking at kidney function by glomerular filtration rate at 18 months, compared to baseline when they entered the study. It is a noninferiority study, using descriptive statistics, showing that the switched patients perform as well in the renal function as the patients who remain on ERT, which is one of the reasons why the Study 011 with the maintenance of the patients' kidney function that we saw in that study, makes us very hopeful that the data that we'll see in 012 will be similar in nature.

So with that background and overview, Jay, if you want to comment a little further on the endpoints in that study

Sure. We'll be looking at renal function in 012, in the same way as we looked at it in 011, with the metric GFR using two measurements of the eGFR CKPD-EPI and MDRD, and all of them will be factored into the descriptive statistics that will be used to judge noninferiority. We have the MGFR prioritized, that is the traditional in gold- standard method for measuring renal function.

But the eGFR measurements will certainly factor in to, I think, anybody looking at the data to compare migalastat to ERT. And as it is based on descriptive statistics, there isn't a strict noninferiority margin established. Of course that's because of the size of the patient population.

It would not be possible to do a statistically paradigm inferiority study in this disease, but we do have the approach with the EU and having discussed it with the MA, of using, as I said, descriptive statistics in order to look at these data to determine that the results for migalastat are the same, are noninferior to ERT.

And is that is the final sort of delta going to depend, in part, on any sort of standard deviation or is there sort of a preagreed, I guess, measure of noninferiority and delta's noninferiority?

I think, Ritu, rather than getting into that level of detail quite here, I think we'll reserve that for when the data comes out. But suffice to say what we need to show in the descriptive statistics is that the error bars essentially overlap between the two groups. There is some degree of variability patient to patient and time point to time point. But there is a good degree of consistency among these patients.

So I think, Jay, feel free to add any color. But I think generally showing the overlap of the error bars is the feedback that we got from the Europeans. Which is a, I think an achievable bar, based on the knowledge that we have from the literature from our Phase 2 studies and now, very importantly, from this Phase 3 study.

I had one quick follow-up to that. Is the delta between when you guys had the data in-house and when you release the data, will not be sort of similar time lag as the 011 study or tentative --?

Well, there was very little time lag. Just to give you some indication, we received that data on 011 in mid-April. And we had a predetermined sequestered team off-site, our clinical team led by Jay -- Dr. Barth here, Ritu, who took about a week in confidence to go through the data and then to share it with our senior team and it was within a 48-hour period from that point when we received the data, to when it was shared with key opinion leaders in confidence. And within another few days until we made it public.

So it's a very tight -- from receipt of data, internal review, external validation under confidentiality with investigators, review with our board, drafting all these documents, that was a -- all in less than a two-week period. So we would expect a similar turnaround here.

What about time between database lock and sort of deliverable data to Amicus?

Again, that will all be the database lock will take place in the third quarter, as well. We stated, I think publicly, that the last patient out of that study is later in the second quarter. So that will trigger a series of events internally and with our CRO to lock the database, turn it around pretty quickly and then get it out in 3Q. But I can't give any tighter of a timeframe for when those results will be public than to say in Q3.

Got it, that's very helpful. Thanks for taking the question.

You're very welcome.

(Operator Instructions)

Anupam Rama, J.P. Morgan

Hi, guys, thanks for taking the question. Just on the Pompeii three-and-three strategy, you kind of outlined sort of the next steps for ATB200, but I was just hoping you'd give us your latest also on AT2220, the chaperone, and what are sort of the next steps in determining which chaperone you might take forward? Thanks.

Yes, I'll ask Brad to comment on that.

I think right now, the progress on AT2220 is essentially fully-developed at this point to use as a chaperone. So the biggest question that we're looking to answer in those preclinical studies remember we've got the base cell alone, which has the superior, we think, unique and superior carbohydrate structure. Then you have the plus or minus the peptide tag, the variant [idea of two] tag for further improvements in targeting.

And then you have plus or minus the chaperone. And what we're really looking for from these preclinical studies is to confirm how much additional benefit you get from each of those two platform technologies that we can bring to bear. So that's the preclinical work that's underway now and we'll be able to give final guidance on that in the second half here as those studies read out.

I will just add a little more color to that to be perfectly transparent. We are in the process of final preclinical studies, as Brad indicated, and it certainly includes, in several of those arms, the addition of the AT2220 chaperone. We have one other chaperone that we are testing that we've been working with for a number of years.

It has different characteristics, whether it's better than AT2220, we'll find out here very shortly. The good news is though, that if we choose that molecule, it's ready for the clinic as well, so there's no further delay or no delay whatsoever.

Great, thanks for taking my question.

You're very welcome.

Kim Lee, Janney Capital

Good afternoon. Thanks for taking the question. I have a hypothetical question here, given we now know the results of Study 011, 6-month and 12-month data, 12-month being positive, 6-month being negative.

Worst-case scenario, should Study 012 data come back negative, how do you see your conversations with the FDA going and can you walk us through some of the potential possibilities of how an approval could still occur? Thanks.

So I guess on 011, the endpoints decreased specified analysis at 12 months, those -- that was a biochemical marker, a surrogate, a very important one and we think one likely to predict clinical benefit. The real beauty of the 011 data though, is that it -- since it ran out to 24 months, we were able to follow these patients for 18 to 24 months, depending on the cohort they were in, and follow their kidney function.

And that's where we think the study is very significant in that that kidney function remaining stable really helps to validate the clinical significance of that surrogate marker. But again, that study was originally intended for approval under subpart H. We may use that as one basis together with Study 012 for full approval.

To your hypothetical, I kind of don't like getting into hypotheticals, but in the absence of seeing any data from 012, of course, if that showed that it was significantly different than the ERTs, it would give us pause for concern. And to understand why that would be, because it would be inconsistent, not with the biochemical endpoint -- well, it would be inconsistent with the biochemical endpoints, but much more significantly, would be inconsistent with the data that we see on the kidney endpoints in Study 011. So we'd really have to work to reconcile that.

So without getting further into the hypothetical, if Study 012 is not successful, we'd certainly have to understand why and would have some cause for concern for moving forward. But the converse is also true. If Study 012 is positive, we think that that combined with Study 011 would present a very, very strong data set in the largest and what would then be the second-largest studies ever conducted in Fabry disease.

Taken together we think would be a very, very powerful data set. So I'd much rather prefer to dwell on that hypothetical, Kim, than your prior example, but both valid for consideration.

No, completely understood. And just to clarify, expecting Study 012 to be positive, you would conceivably go for a full outright approval, instead of the conditional approval, is that correct?

Still to be determined, but I think it would absolutely be a pathway in the United States.

Great. Thanks so much.

Thank you.

Joseph Schwartz, Leerink Partners

Hi, thanks very much. I was wondering if we could talk a little bit about the Fabry coformulation program.

Yes, absolutely.

In particular, what should we be looking for out of the Phase 1 PK study? Is there any PD analysis that's possible there? What are you trying to achieve and how will you choose at those going into patients in 2015?

And then, have you settled on the enzyme component of that strategy or it sounds like from your press release you're leaving the door open to -- for a little bit longer to choose the agent that would be combining with IV migalastat?

Okay, I was just -- we were furiously writing down our notes, Joe. I think that was couple of points to make there. So I think what you're referring to first is the IV migalastat study, which is ongoing right now. That is to determine the PK characteristics of the IV version of migalastat for the coformulation.

We don't yet have that full data set. We would expect to share that as part of the development strategy, I think most likely toward the midpart of the year, maybe third quarter. But again, that's to help us choose the fixed dose of chaperone that will go with the ERT combination. Your question there was PK as well as PD.

How are you thinking about choosing the dose in healthy volunteers? What are you looking for? Is it just -- what would be adequate exposure, I guess, that could guide future development?

Again, we have significant experience with oral migalastat and we're trying to compare that with our oral experience as well as the IV and oral experience in animals. And, Jay, I'll let you comment further on what we would expect to see and what would be the measures in this ongoing study.

I think that's exactly as you're saying. The healthy volunteers PK study of IV migalastat really has lots of data from the oral studies co-administration to know what plasma exposure we're targeting. So it's really a study designed to achieve the optimal target plasma exposure with the IV formulation.

And running through descending doses in order to determine the PK of the IV formulation of migalastat, looking at those data and then making sure we are selecting the dose that puts the migalastat in the target plasma concentration. So I think that's going to be fairly straightforward, and the study's proceeding well. And I think those data will be very informative in the design, the dose selection for the coformulation study.

And again, Joe, to answer the latter part of your question, so with that data here in the next few months, we will know what is the exact dose of the chaperone, the ERT is already completed, manufactured, is ready pending that last piece of information to put in man. We will put that in patients, Fabry patients, next-generation Fabry ERT in a Phase 1-2 study in the second half of this year. So that'll be a very big milestone for us. So that will go in.

That will be the Fabry, the Fabrazyme biosimilar combined with chaperone to make the next generation ERT. That ERT, of course, was sourced from GFK and their partner JCR, again the Fabrazyme biosimilar. We have identified several potential sources and are in advanced discussion with several parties to source a long-term supply, originally envisioned as the commercial supply for the base ERT.

We think, however, that we'll be able to have that program set so that we can make it available for our Phase 3. So that we don't have to do any of the bioequivalence studies at BLA filing, we can actually do it as we bridge from Phase 2 to Phase 3. So we don't have a final contract for that set, but we're in pretty advanced discussions to secure that long-term ERT component of the supply.

Okay, excellent. Thanks very much.

You're very welcome.

I am showing no further questions in the queue and would like to turn the conference back for any further remarks.

No, just thank you all this evening. Thank you for the many conversations, especially with the analysts over the last week. As I indicated, we've had a chance over the last two weeks now to talk a lot about our monotherapy migalastat program with positioned scientists in the patient community.

We, over the next couple of weeks, are going to engage in a concerted effort to visit with investors and analysts to explain more of the data and specifically our strategy for a Fabry franchise based on migalastat monotherapy. What we see as defining success for the 012 study, and very specifically then getting into the broad franchise strategy of monotherapy and the addition of a second product, the next-generation ERT.

So anybody who would like to be added to that schedule that's just coming together, please feel free to reach out to Sara. And certainly through any of the excellent analysts on the call. And that's all I have, Operator, thank you very much.

Thank you. Ladies and gentlemen, thank you for participating in today's program. This does conclude the meeting and you may all disconnect. Everyone, have a wonderful evening.