Q2 2025 Amicus Therapeutics Inc Earnings Call
Later, we will conduct a question and answer session and instructions will follow at that time.
As a reminder, this conference call is being recorded I would now like to turn the conference over to your host Mr. Andrew <unk>, Vice President of Investor Relations.
May begin.
Good morning, Thank you for joining our conference call to discuss amicus Therapeutics second quarter 2025 financial results and corporate highlights leading today's call. We have Bradley Campbell, President and Chief Executive Officer, Sebastien Martel, Chief Business Officer, Dr. Jeff Castelli, Chief Development Officer, and Simon Harford.
Chief Financial Officer, joining for Q&A is Alan Rosenberg, Chief legal officer.
As referenced on slide two of the presentation I would like to remind you that we will be making forward looking statements on today's call I encourage you to read the disclaimers in our slide presentation. The press release, we issued this morning and the disclosures in our SEC filings, which are all available on the IR portion of our corporate website.
Forward looking statements are subject to substantial risks and uncertainties speak only as of the calls original date and we undertake no obligation to under to update or revise any of the statements. Additionally, you are cautioned not to place undue reliance on any forward looking statements.
At this time, it's my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer Bradley.
Great. Thank you Andrew and welcome everyone to our second quarter Conference call I'm very pleased today to highlight our strong quarter and positive outlook for amicus for the remainder of the year.
First we've delivered yet another quarter of strong double digit revenue growth on our core business in Pompe and Fabry disease. This is our 17th consecutive quarter with double digit sales growth at CER, and we see that trend continuing for years to come.
Secondly, we remain highly confident in our growth trajectory that you will see throughout the remainder of the year <unk> delivered 13% year over year patient growth this quarter and is on track to achieve the highest number of global patient starts this year.
For possibility not Foldup Q2 marked the strongest quarter for commercial demand since our launch with significant momentum both in the United States and the five new launch countries in Europe as well as the existing launch countries there as well.
Third we're steadily building the body of evidence highlighting the differentiation of PON building up fold in Pompeii disease, including a recent publication in muscle and nerve, which demonstrates the benefits for switching <unk> experienced patients to possibility of October.
Fourth we are reiterating our confidence that these two products will reach a combined sales of $1 billion by the end of 2028.
We continue to believe that <unk> is uniquely positioned to further its reach as diagnostic rates and patient access continues to improve offering a substantial runway for sustainable growth and pump building up photos, becoming an increasingly meaningful contributor to our long term performance.
As a reminder, each of these products have a $1 billion plus in peak sales potential.
Fifth we continue to advance our strategic partnership with <unk> and <unk> 200, our first in class compound in late stage phase III development for rare and life threatening kidney disease.
The actions <unk> III pivotal study remains on track for full enrollment by the end of the year, marking a key milestone in this important program.
<unk> 200, and our opportunity to meet the significant unmet need that exists for people living with <unk> as an important new part of the amicus story and we look forward to telling you more over the course of the year.
And finally, as we continue to maintain our financial discipline. We reiterate that we are on track to achieve GAAP profitability in the second half of this year.
Altogether, we're pleased with our accomplishments this quarter and believe amicus isn't a very strong position to generate meaningful value for our shareholders and deliver on our mission for patients in 2025 and in the years ahead.
With that let me now hand, the call over to Sebastian to go through some more detail Sebastian.
Thank you Bradley and good morning, everybody. So let's start with got pulled on slide five you'll see the revenue reached $128 nine.
$9 million this quarter, 12% at constant exchange rates and up 16% in reported terms.
<unk> growth of this product remains very positive and is driven by the number of new patient starts globally. We ended the quarter with more than 6% to 90% of the global market share for treating fabry patients with amenable mutations.
<unk> is clearly positioned as the treatment of choice amongst prescribers and there are still many more potential patients eligible for our therapy.
Turning to slide six our leading markets continued to be the biggest driver of strong patient demand for gaslog.
The U S actually contributed significantly to growth, reaching more than a solvent prs since launch a major milestone.
When we look at the global mix of patients from Gaslog today, which is about 65%, even 35% switch we're now seeing stronger uptake naive populations and while we continue to achieve high market shares in countries, where we have been approved the longest there's still plenty of opportunity to switch patients over to Garfield and to keep growing the market as we are.
Penetrate the diagnosed and treated.
Newly diagnosed segments.
With underlying growth in patient demand at 13% this quarter and our projection of a record level of new patient starts. This year, we remain highly confident in our full year 2025 growth guidance, what kind of foods.
The key drivers behind the robust demand for <unk>, which we expect to continue well beyond 2025 are the following first finding new patients in penetrating into the diagnosed untreated population, including shortening the pathway to diagnosis.
Expanding <unk> into new markets and extended the label.
Third driving vehicles share of treated amenable patients.
We're actually seeing in our most mature market that we can reach 80, 590% share. So we know that there's the potential to reach those levels globally and fourth sustaining compliance and adherence rates above 90%. So that patients who go on Garfield pretty empty stay on Gaslog.
On slide seven we highlight the significant unmet need in fabry disease to be.
Over 12000 people receive probably treatments worldwide was 6000 diagnosed patients remained untreated <unk>.
Literatures suggest actual prevents makes it over 100000 individuals, indicating a meaningfully larger under diagnosed population.
And then origin, you believed and substantial market opportunity for gasoline.
We're highly confident that a small molecule is a compelling treatment option for the untreated and undiagnosed populations as indicated by the record high growth in new patient starts.
Just only scratching the surface with gaffle today, and as disease awareness and enhance diagnostics initiatives further shape. The subdued market. We're confident in the long term potential for this medicines, which we think continues to be under appreciated.
With excellent momentum the.
The sizable untreated population and our strong IP protection Gulf when there's a long runway well into the next decade, and a clear path to surpassing $1 billion in revenue.
Turning now to compete if he is on slide nine.
We outline our global launch progress with Omnichannel pulled out.
Second quarter revenue reached $25 $8 million up 58% at constant exchange rates the.
The majority of sales came from our initial five launch countries. The U S U K, Germany, Spain and Australia.
Although as I will highlight in a moment, we launched into five new markets in Q2 alone.
The U S represented approximately 42% of revenue while ex U S represented 58% of revenue.
Q2 showed strong sales growth as well as record levels of patient demands.
We continue to see patients switching proportionately based on market shares as well as the broadening and deepening of prescriptions with more sites coming online and multiple new prescriptions from physicians.
Given these indicators we are reiterating our full year 2025 revenue growth guidance for computer kind of pulled out of <unk>.
50% to 65% growth at constant exchange rates.
Our guidance implies a healthy exit rate heading into the next year and we remain highly confident in the 2025 and long term outlook for this therapy.
We expect commodity channel pulled out to be a major contributor to multiyear growth Rami just based on key growth drivers, namely continuing to increase the number of net new patients increasing the depth and breadth of prescribers launching in new countries, including up to 10 this year alone.
Differentiating our therapy evidence generation and real world evidence.
And last mentioned being 90% plus compliance and adherence rates.
Moving to slide 10, looking at the geographic expansion of computer channel pulled out.
In the second quarter, we recorded revenue in 11 countries. So six countries have their first patient stops during the first half of 2025.
Italy, Switzerland, Portugal, Czech Republic, Sweden, and the Netherlands.
We're pleased that <unk> was selected as the preferred treatment for adults with <unk>. It's an important gets old patients group go through one site and the sizes actively transitioning patients it will be a key driver for us in the second half of this year.
With teammates well over 100 patients.
And intend to take up to 70% of this population.
This will then become the largest cohort in any single center worldwide and definitely a rich source of data on comedy channel pull down.
We also recently received regulatory approval in Japan.
Excited to have a labeled indicated for people ages 50 and older.
We're also continuing our work to secure broad patient access throughout the EU.
I hope that the commercial review provides a strong sense of the continued execution and growth in <unk> and the building momentum in the launch of comedy channel pull down with that I will now hand, the call over to Jeff to highlight the work we do to further differentiate completed general pull down Jeff.
Yes.
Thank you Sebastian and good morning, everyone.
Starting on slide 11, we highlight a few examples of our rapidly expanding and diverse body of evidence supporting the differentiation of probability in our quota in pompe disease.
And specifically on slide 12, we summarize two recent case studies recently presented at the <unk> 2025 annual meeting supporting the experiences of individual switching from <unk> to <unk> on Florida.
These kind of studies along with the growing body of real World evidence continues to show that improvement is possible from any patients when switching capability and a photo.
Believe our ongoing efforts to grow this body of evidence will ultimately drive wider adoption of possibility on a photo.
Moving to slide 14.
Previously announced we took a major step forward in our strategy to strengthen our portfolio through a very successful U S licensing agreement with <unk> to commercialize <unk> 200, our first in class treatment in late stage development for <unk>, a rare and potentially fatal kidney disease.
With blockbuster market potential we remain highly encouraged by the data seen to date and believe this asset brings immediate strategic value for amicus and will create value for patients and for shareholders.
Moving to slide 15, we think it's important to highlight the very differentiated and very compelling mechanism of action of <unk> 200, which continues to resonate well with physicians and the <unk> research community.
There are currently no FDA approved therapies for FSD is standard of care includes non specific therapies, such as corticosteroids Carlson urine inhibitors, and angiotensin receptor blockers, none of which adequately address the monocyte driven inflammatory aspect of ssds.
<unk> 200 is an oral small molecule taken in combination with arms.
Specifically target this monocyte driven inflammatory component of FSC.
By inhibiting signaling from the angiotensin one receptor <unk> receptor type two tetramer that is formed and damaged kidney cells. It.
It delivers a kidney selective anti inflammatory effect directly targeting this key unaddressed driver of disease and.
In particular in patients, which are many of them in FSD us with persistent proteinuria and active inflammation.
Preclinical and phase II studies support this mechanism of action and demonstrate impacts on current area with a well tolerated safety profile to date with.
With no evidence importantly of the MCP, one rebound effects observed with traditional CCR two inhibitors.
Moving on to Slide 16, we are very impressed by the strong momentum <unk> built and the growing body of evidence supporting the transformative potential of BMS 200 the.
The pivotal phase III <unk> trial is progressing really well with more than 75% of patients now enrolled and remains on track for full enrollment by year end.
He is robustly designed and strongly powered with several successful interim analysis completed to date.
Importantly, there is FDA alignment on proteinuria as the primary endpoint for approval and taken together with all of these facts. We believe <unk> 200 is positioned to truly be a meaningful advancement for <unk> patients.
Following additional analysis in accordance with the parasol consortium over the coming months, we anticipate requesting an additional meeting with the FDA to discuss the next interim assessment of efficacy from the accent III study and next steps for <unk> 200.
With that let me now hand, the call over to Simon to review, our financial results and outlook Simon.
Thank you Jeff All financial summary begins on slide 18, with our income statement for the second quarter ending June 32025 for Q2, we achieved total revenue of $154 $7 million, which is a 22% increase.
Over the same period in 2024 at constant exchange rates revenue grew 18%.
Global geographic breakdown of total revenue in the quarter consisted of $94 million or 58% of revenue generated outside the United States and the remaining $64 $3 million or 42% coming from the U S.
Cost of goods sold as a percentage of net sales was 10% for Q2 as compared to 9% in the same period last year.
Total GAAP operating expenses increased to $148 9 million for the second quarter of 2025 as compared to $100 4 million in the second quarter of <unk> 24, an increase of 48%. It is important to remember that Q2 operating expense.
This included the upfront payment of $30 million for the U S licensing rights to <unk> 200.
On a non-GAAP basis total operating expenses increased to $127 $8 million for the second quarter as compared to $82 1 million in the second quarter of 2024.
An increase of 56%, we define non-GAAP operating expense as research and development and SG&A expenses, excluding stock based compensation expense loss on impairment of assets changes in fair value of contingent consideration restructuring charges and depreciation and amortization.
<unk>.
On a GAAP basis net loss in the second quarter of 2025 was $24 $4 million or <unk> <unk> per share compared to a net loss of $15 7 million or <unk> by shaft at the second quarter of 2024.
<unk> the $30 million upfront payment related to <unk> 200 agreement, we would have delivered positive GAAP net income for the quarter.
In Q2, 2025, non-GAAP net income was $1 9 million or one <unk> per share.
Compared to non-GAAP net income of $18 5 million or <unk> <unk> per share in the second quarter of 2024.
Cash cash equivalents and marketable securities with $231 million as of June 30 of 2025 compared to $250 million as of December 31, 24.
On slide 19, we are reiterating our full year financial guidance for 2025 as follows total revenue growth of 15% to 22%.
<unk> revenue growth of 10% to 15% possibility of not sold a revenue growth of 50% to 65%. All of these growth rates are at constant exchange rates gross margin is expected to be in the mid eighties.
non-GAAP operating expense guidance remains at $3 $80 million to $400 million.
And we anticipate positive GAAP net income during the second half of 2025.
I mentioned earlier this year 2025 will be a hybrid TF or possibility of not filled our cogs as we have worked through the previously expense still zero cost inventory during the first half of the year as a result, we expect our gross margin to be in the mid <unk> for 2025, as we begin to recognize.
<unk> sold our Cogs through the P&L in the second half of the.
And with that let me turn the call back over to Bradley for closing comments.
Great. Thank you Simon Jeff and Sebastian as we come to the end of our presentation. Here's just a quick reminder of our strategic priorities for the year and at closing I want to reiterate how encouraged we are by the growing demand for our therapies in the very promising phase III asset that we've added to our pipeline we.
We see a clear path to sustained growth in 2025 and beyond and we've demonstrated that we have the portfolio and capabilities to deliver that at.
At a highly attractive growth trajectory.
Amicus continues to represent a very differentiated company in biotech and rare disease with now 17 successive quarters of double digit revenue growth, a derisk portfolio and growing categories and an efficient and highly effective organization that is laser focused on delivering for patients with rare diseases I have full confidence that we will continue to advance transfer.
I would have treatments and create lasting value for patients and shareholders alike.
With that operator, we can now open the call to questions.
Ladies and gentlemen, if you have a question. Please press star one on your Touchtone telephone.
At this time, we request that you only ask one question. If you have an additional question please enter back into the queue.
Please standby, while we compile the Q&A roster.
Our first question comes from the line of Neil Panorama of J P. Morgan. Your line is now open.
Hi, guys. This is pre owned gone Brian upon congrats on the quarter. So looking at the real world evidence clinical assessments really resonate with physicians and Kols and patient switched from NEC for the enzyme to palmas.
There are differences between the U S O U S.
Thanks Priyanka Altair.
Now I'll turn it over to Jeff in a minute to provide some detail, but I think the really important thing here is now that we have multiple treatments. This is exactly the question, but I think we are helping to drive and scientific community and as we develop more evidence and as we demonstrate the effects of probability unfolded I think that will continue to be an important part of the story, but Jeff maybe talk.
Kind of how that's evolved somewhat with these new therapies and what the physicians and patients are looking at.
Yes, thanks for the question.
The physicians are looking for when switching from <unk> or not that different from what they are looking for when you're switching from <unk> onto Palmer I mean, the majority of patients that are switching here early in the launch tend to be those that are on X files I am have either we're not even went on as far as <unk> were switched from <unk>.
And are not having the outcome that they had hoped when they want it and that's by design and Theyre looking for stability on the declining function or improvements and things where there had been stability previously and typically as shown on the slide in the presentation. They look at things like Biomarkers muscle strength, and then things like six minute walk I said as well of course is just.
Quality of life, how does the patient doing day to day and activities of daily living.
And what was really exciting from the two phase studies highlighted here in the presentation as well as what we're hearing more broadly is that similar to what we saw in trials and so far in some of the different studies ongoing those patients switching from exxon's I seem to also be having on average or in many cases, a very positive experience on that switch, but it really is not that different.
Switching from next and switching from one design Thats, a pretty similar process.
Thanks, Jeff and then I don't think to your question Priyanka I do not think there's really much difference between the U S and other geographies.
One moment for our next question.
Our next question comes from the line of Joe Schwartz of Leerink. Your line is now open.
Great. Thanks, very much and congrats on a strong quarter. So my one question is I guess will be on.
With tariffs and MFN remaining.
A topic of discussion now I was wondering if you could just update us on your additional manufacturing facility for palm up in Ireland, when when could that come online and does that get you to where you think you need to be to the extent anyone can forecast the future in this regard.
<unk>.
And.
Do you think that could.
Supply all of the Palm up that you forecast youll need.
And can you just remind us how much drug you've stockpiled in the U S. Thanks.
Thank you very much Joe a few questions there maybe I'll kind of go in reverse order so.
We brought a.
All of the material into the United States for possibility of full that we needed for commercial use this year in clinical use as well.
Is what led us to say that there is no material impact of tariffs on our P&L. This year and any forecast that we've been able to do going forward, even at relatively conservative levels as very manageable within our P&L based on our new global supply strategy.
It relates to Ireland from a capacity perspective, yes, especially as we look towards the second generation manufacturing process, which will all evolve over the next kind of five years that could supply the global demand that we forecast. However, it is very likely that we may have a secondary site.
Just from a good strategic perspective, right now that site is China, which could serve Europe and ex U S markets, but there might be other opportunities there and I would point you to the announced that we had in Q1, which was for the very first time, bringing.
Yeah.
Product manufacturing to the United States with our collaboration with sharp.
We may continue to evolve that.
Time goes on depending on the political.
Landscape, but I think we've been very prudent and very forward looking to have a diverse supply chain.
Our last question in terms of when when those things will come on board for Ireland, We believe that the.
That material until the commercial supply chain towards the back half of this year in Europe, and then sometime next year in the United States, which is exactly what we forecasted. So we think we're in really good shape, we've been able to navigate all of the kind of headwinds and challenges that are out there and we expect a very robust optimized supply chain going forward.
Very helpful. Thanks for the insight.
Yes, Thanks John.
Our next question.
Our next question comes from the line of Maxwell score of Morgan Stanley. Your line is now open.
Great. Thank you for taking my question.
And congratulations on the quarter. So now that you've read the brief submitted by <unk> for summary judgment do you still feel confident in your IP position and the potential for a settlement. Thank you.
Yes, Thanks, Max just as a reminder, we've said that we remain highly confident in the strength of our IP and a long term opportunity to support fabry patients through the many years to come of course, the settlement with Teva reinforces our confidence in the strength of our case against any remaining litigants, including Aurobindo and overall the <unk>.
Strength breadth and depth of our IP estate.
Because we are still in litigation, we can't we can't comment further on the details there, but I would just say we remain highly confident.
Net debt.
RFP as long and is supported.
And we would remind everybody just statistically the vast majority of these cases ultimately lead the settlement in particular, when one party reaches a settlement first so hopefully that's helpful and look forward to providing further updates as time goes on.
Great. Thank you.
One moment for our next question.
Our next question comes from the line of Richard Borrow TD Cowen. Your line is now open.
Hi, Brad This is Joshua fleishman on the line for it too.
A few.
One multipart question.
Our timelines progressing for the new U S manufacturing process and what do you think the impacts could be on Cogs, How do you view additional pipeline expansion and what would your priorities and what can we expect for <unk> launch in the next 12 months is more next design patients approach the important two year Mark for treatment reevaluation and how is the.
Current reimbursement situation. Thank you.
So I see you've adopted to the approach to one question with multiple parts will do our best to answer all of them. Thank you Joshua.
Maybe Sebastian do you want to speak to the U S drug product.
Manufacturing facility and the general timelines there, we haven't given real specifics, but just a flavor for sort of how that will evolve and then we'll take the next to you as well.
Yes, thanks, Brett so.
You saw that.
As we announced in Q1, we signed an agreement with sharp sterile to bring the probabilities.
Drug product.
Manufacturing two to the U S essentially onshoring DP manufacturing for.
Community.
So we will be.
We'll be working through our PV queues.
Next few quarters.
We haven't shared specific timelines yet.
That site might be.
Up and running.
Meantime, you've heard from Grande the progress we've made on the <unk> side of things from Dundalk Island site. So very excited with the progress we're making here with both.
And in EBITDA.
And then in parallel we have another site for BP in Germany, maybe a Houston.
We were also making great progress and are actually just started <unk> runs is as we speak so.
Very advanced.
Sure.
Overall manufacturing strategy for <unk>.
Thanks, Sebastian just to hit a couple of the other points you made there.
I'll start with maybe a quicker one first so reimbursement continues to go really well and all of our geographies.
You have seen we have oftentimes Ben first our fastest in multiple markets like the first ever approval from nice prior to NHRA approval fastest two to getting to reimbursement in a number of markets.
Excuse me getting lead position a number of markets like the Netherlands with Sebastian highlighted and I think that just reflects our approach to maximizing access and delivering value for all stakeholders will continue to do that to your point about next advise him switches.
We've said previously a significant portion of the United United States. As an example of the <unk> community have come to that sort of two year switch point and that obviously will continue to grow over time I think it was important Sebastian to highlight that we are switching.
Sort of relative to market share so in the U S where the majority of patients are on <unk>, a majority of our new patients are coming from <unk> as well and then your last question on pipeline expansion. We're really excited about <unk> 200, and about telling that story in more detail over the course of the year.
From a BD perspective, we still think there are opportunities to leverage our infrastructure and capabilities globally and would continue to be focused on late stage de risked assets near commercial assets similar to the Amex 200, hopefully that was helpful. I think I caught them all so thanks Joshua for questions. Yes. Thank you so much.
One moment for our next question.
Our next question comes from the line of Eliana Merle of UBS. Your line is now open.
Hi, This is tejas on for Elliot congrats on the quarter.
Just could you guys give a little bit more color on how.
Starts are going in ex U S markets I know you mentioned in the Netherlands, We then.
Any color there would be great. Thank you.
Sebastian you wanted just to give a few highlights on some of the exciting things we're seeing in some of the different markets Theres lots of we could tell but may be Spain would be a good one to highlight.
I don't have any of that.
Germany, the UK, etc.
Yes.
So we continue to see strength from the first market we launched so.
Bud mentioned, the U K and Germany and.
Interestingly in the U K when you take into account the <unk> program and the fact that <unk> was.
It was essentially available for physicians.
Almost two years prior to launch so we've been in that market or about.
About 300 hops yours now.
Now our market share is reaching 35%. So we see that market with a lot of enthusiasm as to what we could achieve in other markets as well.
Germany remains strong Spain.
You've said, Brian This was an interesting situation, where we actually launched.
Neck and neck with mixed design and we're seeing.
Okay.
Again makes changes can inroads from from a market share standpoint.
In that market.
Smaller markets like Sweden, where.
Pump is actually.
The drug to be on if you have.
<unk>.
<unk> in Sweden, and so we have.
Disproportionate market share in the Swedish market as a result of.
Of that.
We are launching each of you as we speak I did say that this year alone. We have now six new countries and we launched it.
And we anticipate another four.
For the remaining of the year that would include our Japanese launch.
In the second half so lots of.
Lots of room to continue to grow.
Bombardier general pull down.
Great. Thanks, Sebastian Thanks for the question.
One moment for our next question.
Our next question comes from the line of Christian <unk> of Cantor Fitzgerald. Your line is now open.
Hi, good morning, and congrats on a nice revenue beat.
For palm up with 40% of the patient pool treated on next design, reaching that two years. This year curious now that you have more data behind your hand, what's making patients switch right at two years versus earlier versus perhaps later on is it there.
Time diagnose with Palm Bay are there any specific drivers that again would make someone switch earlier later or right at that two year Mark. Thank you.
Yes, Thanks, Kristen I think embedded in your question is the reality, which is it's not like at two years everybody switches. It as a continuum I think the earlier switches have been people who were clearly declining on regardless of what therapy. They are on alumina diamond or diamond I think that will continue to happen. If it's an obvious decline I think physicians and patients are looking for.
For something new and different and that probably skews. The initial patient patient population also too.
A more.
Severe patient population.
Is that going to be an older patient population, but the exciting thing is we're the only product in our head to head study that showed an improvement and I think that as Jeff said improvement is possible with this product I think that's really the promise of what we can offer.
Over time, though I think two things can happen. The first is what Jeff talked about earlier, which is I think the physician community now I don't think I know is asking themselves.
What do we need to look at how how closely do you need to look at some of these measures to be to find a more subtle early predictor of decline and so I think thats a very important conversation that's happening right now I think the Holy Grail and what our ambition is and we kind of saw this was gala fold is instead of waiting for a decline.
Essentially we believe we can establish <unk> as the best therapy out there and then Youll see a proactive switch.
Other dynamic that's kind of flowing through all of that is also where patients sort of raise their hand, and say, hey, I'm, not feeling well or I want to try something new and Thats been an exciting part of the story as well. So hopefully that gives you a flavor of some of the dynamics that you were getting at in your question.
And Brad the only other thing I'd add is just looking at the long term data that we've seen from <unk> from <unk>. For example, what you see on that kind of average response across parameters like six minute walk FEC is that after one year or two years you generally see on average a continued slow decline. So you would.
<unk>.
Thinking about people not doing well theres going to be more patients not doing well after three years of next and after two or after four years of next versus three so that will continue to add people. If youre just looking at those not doing well, we would expect there to be a kind of continued growth of that overtime.
Thank you.
One moment for our next question.
Our next question comes from the line of Dennis Devine of Jefferies. Your line is now open.
Hey, Thanks, good morning, Thanks for taking our questions.
Two for me.
S SGS gainesville into a little bit more detail on the regulatory alignment with the FDA on <unk>.
How much of that is actually a written in stone per se.
I asked about the impact to that alignment can you prepare outcome doesn't support.
<unk> traditional approval.
And then on number two.
A question on Pompe <unk>.
And I appreciate the revenue growth does look second half weighted but curious on when you hope to maybe exit rate going into 2026 and on a continued acceleration in 2026 and I guess, what additional new countries you plan to launch in 2026.
Great. Thank you for the questions.
Maybe it will go kind of in reverse order so for pump K exactly right, we'll see a continued benefit of that acceleration in the second half.
Even with all the kind of new launch countries that Sebastian highlighted I think even a further impact in Q4 as an example.
Which is also pretty typical anyway to what we've seen with <unk> Q4 tends to be a strong quarter.
As we go to next year TBD as it relates to run rate going into next year, but it's exactly the question to ask we'll know more as we go forward here I would just say that we do think next year will be higher.
A higher absolute revenue growth than this year, we'll have a lot more color to stay on that going forward.
In terms of <unk>.
I'll start, but then I'll have Jeff take over that question as part of our diligence and is a prerequisite of the deal we had to see the FDA minutes from there.
Type C meeting and we were very pleased with with the feedback that FDA had given to <unk> in particular that proteinuria could serve as the primary endpoint for that study that was really important for us to see and I think an exciting development for the community, but Jeff speak a little bit too.
What we've said publicly around what that May mean for the primary and then how we see <unk> ad com.
As it relates to our program.
Yes, thanks, Brad So as Brian said.
The feedback from FDA for the <unk> program was quite clear in terms of suitability of protein area at two years as a primary endpoint with <unk>.
Just supportive data from GFR as a secondary.
That can be measured as a percent change as responder thresholds of meeting certain thresholds of primary and we expect that we'll do all of those.
As it relates to the AD comm look it's not surprising there is an AD com <unk> sort of the first product going through with partner as a potential primary they had a complicated phase III, where the technical GFR endpoint was missed and now they are looking at partnering or sort of alternative away for approval. So I think that'll be a great conversation to.
Have a dotcom around proteinuria.
There is a number of really important similarities and differences between <unk> 200 is sort of you have to think about around the AD com to.
So clearly similarities are they're both targeting very similar FCS population sort of primary genetic FSD us with significant part area and they are both planning and used primarily as a primary endpoint.
Other than that Theres, a lot of differences MLA is a very different they target different underlying pathology is sort of the hemodynamic side of things versus the inflammatory side with <unk> 200, <unk> hundred is going to have a prospectively defined primary endpoint for phase III and ultimately the data on partner area and importantly on VFR might be different for the two products.
We view a positive outcome reinforces pardon areas of suitable endpoint in particular, one is prospectively defined and we would not view <unk> approval as a downside at all if anything that would help sort of start to really the facs community for new treatments Mechanistically, we think theyre very differentiated products and would work best in <unk>.
Types of patients and ultimately could be synergistic together.
In a negative outcome. Similarly, we think will inform us about how advisers. After you are viewing partner <unk> will help us position our data.
A negative outcome could be due to specifics around that kind of complex, thus far a dataset.
And not necessarily read through to the Nx 200, and ultimately could even position us as the first market that did not work for both parties. So we view the <unk> as sort of a positive positive for us we're really looking to be informed by it but ultimately we would hope that as a positive outcome and to aspire to get approved for FCS patients and then we can quickly behind it <unk>.
Addressing a different aspect.
Okay.
And sorry, one last question Dennis you also asked about additional countries for next year, Australia, Canada are two big ones over time will continue as we did with California going into Asia Pacific into Latam into more European countries. So there's still quite a bit of geographic expansion for pump as well, but thanks, so much for the questions.
Perfect. Thanks, guys.
One moment for our next question.
Our next question comes from the line of Salvino Victor of Goldman Sachs. Your line is now open.
Hey, this is mark on for Sylvia and thanks, So much for taking my question so on <unk> hundred.
When can we expect the phase III data and also what is the bar for success here and what would be clinically relevant and F. S. Jeff.
Jeff do you want to take those just a reminder of the timelines and then.
The clinical meaningfulness.
And kind of what we think the success bar would be.
Yes. Thanks for the question so as we said.
Enrollment is going extremely well in the phase III study were on track for last patient in which of the 286 adult patients by the end of the year that would mean loss base now two years, which at the end of 2027, so that would be the timeline that the topline full two year data.
I'm sorry, what was the second part of that question.
Well it was lower than the bar for so clinically relevant. So so one thing we are really excited about is from a powering perspective with a 286 patients two years. The study is powered to show small changes in protein area of percent changes is less than 10% between groups are responder threshold of less than 10%. So then it comes down to.
Clinical meaningfulness.
You could make an argument of any improvement in protein is clinically meaningful I think one of the better ways to show that as for responder analysis is well established.
Get patients below certain thresholds like below three grams per gram or below one five or below <unk> seven that theres really improve the outcomes on progression to end stage renal disease. So I think looking at those responder thresholds will be important at a kind of a patient level.
Alright, thank you.
One moment for our next question.
Our next question comes from the line of Sean.
Your line is now open.
Sean Your line is now open.
Okay.
Okay.
At this time.
Don't see any further questions.
This concludes today's conference call have a great day.