Q3 2021 Amicus Therapeutics Inc Earnings Call
Good morning, ladies and gentlemen, welcome to the.
Therapeutics third quarter 2021 financial results conference call and webcast at this time.
Time, all participants are in a listen only mode. Later, we will conduct a question and answer session and instructions will follow at that time.
If anyone should require assistance during the conference. Please press Star and then zero on your Touchtone telephone.
A reminder, this conference call is being recorded I would now like to turn the conference over to your host Mr. Andrew Faughnan head of Investor Relations you may begin.
Thank you operator, and good morning, everyone. Thank you for joining our conference call to discuss <unk> Therapeutics third quarter 2021 financial results and corporate highlights speaking on today's call. We have John Crowley, Chairman and Chief Executive Officer, Bradley Campbell, President and Chief Operating Officer, Jeff Mcqueeney, Chief Financial Officer, and Dr. Jeff Castelli.
Chief Development Officer.
Joining for Q&A, we'll have Dr. Mitchell Goldman Chief Medical Officer, and Sebastien Martel Chief business officer, joining as referenced on slide two we may make forward looking statements within the meaning of the private Securities Litigation Reform Act of 1095 relating to our business as well as our plans and prospects are forward looking statements should not be regarded as references.
<unk> by us that any of our plans will be achieved any or all of the forward looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions, we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward looking statements, which speak only to the date hereof all forward looking statements.
Qualified in their entirety by this cautionary statement and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances. After the date hereof for a full discussion of such forward looking statements and the risks and uncertainties that may impact them. We refer you to the forward looking statements and risk factors section of our annual report on Form 10-K.
For the year ended December 31, 2020, and quarterly report on Form 10-Q for the quarter ended September 32021 to be filed later today with the Securities and Exchange Commission at this time. It is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer John.
Great. Thank you everyone. Good morning, and welcome to our third quarter 2021 results Conference call I am pleased to report that the third quarter reflected broad execution across our businesses. We remain on track to achieve our key strategic priorities for the year.
As we did in this morning's release I would like to go ahead and highlight several key update.
<unk> continues its strong performance and remains the cornerstone of our success with 79 point.
$5 million in third quarter revenue, we are very pleased with the continued momentum of <unk> globally as we added new patients from both switch and naive population throughout the quarter.
Second we continue to make tremendous progress on our global regulatory filings for AP GAA.
Our novel next generation therapy for Pompe disease.
In September the U S food and drug administration accepted the review.
For review of the biologic license application Pacific Lucas sided Alpha and the new drug application for <unk> at the two components of <unk>.
The U S review is underway.
The FDA has set a <unk> action date of May 29, 2022 for the NDA related to the enzyme stabilizer and July 29, 2022 for the BLA of the biologic the two regulatory filing significantly cross reference each other and we expect that they will be reviewed.
The other.
In the European Union following our previously announced positive repertoire and co repertoire meeting I am pleased to share for the first time. This morning that the marketing authorization applications or MAA for <unk> had been submitted to the European Medicines agency.
We are now closer to having another potential treatment option for people living with Pompe disease, both in the United States and in Europe with further regulatory applications planned in the months ahead.
We are confident in the potential benefits of atg, AA and what it can bring to people living with Pompe disease around the world and we look forward to working with regulators to get this important medicine to as many patients as quickly as possible.
On the gene therapy front, we continue to make excellent progress in advancing and developing the industry's leading portfolio of next generation gene therapies for people living with rare genetic diseases.
Our teams are preparing and making great progress towards the business combination in which the amicus gene therapy business will be acquired by area for a special purpose acquisition company.
Through this business combination the amicus gene therapy programs technologies intellectual property key personnel.
And relationships will form a new company.
<unk> therapeutics.
We strongly believe that separating our business into two highly focused standalone companies is the best way to unlock value for amicus shareholders and to properly fund and focus our gene therapy efforts in.
In a single stroke, we will have created what will be what we believe will be one of the world's preeminent next generation genetic medicine and gene therapy companies in carrot costs, while significantly strengthening amicus for its future.
Carrot costs, which is the Latin word for compassion.
Launched with approximately $400 million in capital amicus.
Amicus will become the largest shareholder in care with us.
With a 36% ownership stake and retained co development and commercialization rights to the Fabry and Pompe gene therapy programs as well as negotiation rights on select future muscular dystrophy programs.
As we disclosed at the time of announcement I will lead character as its chairman and CEO, while remaining the chairman emeritus and chief strategic adviser for amicus.
This amicus role I will work closely with Bradley.
And the board of directors at amicus to ensure that we get <unk> approved and launched in all the major regions around the world as well as to support major strategic initiatives.
Bradley Campbell will succeed me at amicus as Chief Executive Officer.
The characters form S. Four has now filed with the Securities and Exchange Commission and the launch of <unk> Therapeutics is expected in late 2021 or early 2022 dependent on customary closing conditions.
I'd also like to provide an update to our <unk> batten gene therapy program.
We have very recently learned of the loss of two children with <unk> six in this study during the long term follow up extension period of this study.
As deemed by the investigator the two participants passed away from disease related complication unrelated to the study drug.
We will of course continue to assess the data in this extension study.
Just as a further reminder of the accrual nature of this disease and the urgency for therapies that may extend and enhance the lives of these children.
And finally under the current plan, which includes the intended carrier task transaction. The amicus cash position is sufficient to achieve self sustainability and profitability in 2023.
During the quarter the balance sheet of amicus with strengthened through a $200 million private.
Private investment from a syndicate of leading biotechnology investors.
Our continued revenue growth prudent expense management and growth potential has allowed us to reach this important milestone as we continued to realize our vision of delivering groundbreaking and new medicines for people living with rare diseases.
That introduction, let me go ahead, now and hand, the call over to Bradley Campbell, our President and Chief operating officer to further highlight the <unk> performance in the quarter Bradley.
Great. Thanks, John and good morning, everyone as mentioned I will walk you through a more detailed now our <unk> performance for the quarter.
On slide seven we give our typical global snapshot of the <unk> commercial progress for the quarter total product revenue grew by 18%.
Versus same quarter last year to $79 5 million globally, driven by strong patient demand new patient starts and business continuity.
The geographic breakdown of revenue during the quarter was $53 9 million or 68% of revenue generated outside the United States.
And the remaining $25 6 million or 32% coming within the United States.
As we've mentioned before this is in line with the roughly 70 30 split that we expect as we continue to grow both parts of the business.
Turning now to slide eight.
The third quarter was another strong quarter for our global commercial efforts and the business continues to be incredibly resilient with patients added in all of our major markets and third quarter revenue reflected increased patient demand and also benefited from continued foreign exchange tailwind.
We do continue to see some sporadic COVID-19 related slowdowns in new patient starts maybe delays delays at the point of care between patient identification and initiation of treatment.
Importantly, though our customers have confidence they can access Cal fold our field team has been able to achieve a substantial majority of their pre COVID-19 touch points and this was done through a combination of in person digital telephonic and other means of interacting with physicians.
So while COVID-19 is not yet fully abated as we had hoped it continues to be a very successful year for <unk> with the first three quarters of 2021 in line with our internal expectations.
As mentioned on past calls due to a variety of factors the rate of growth within the year is typically not linear.
In a given year, we see stronger quarter to quarter growth in the second quarter and fourth quarter.
Versus the first quarter and third quarter and that pattern has continued.
Based on this momentum and assuming the dynamics I've just highlighted continue we're confident we will come within our full year 2021 guidance of $300 million to $315 million in global sales.
And on slide nine I do want to recognize the importance of continuing to build the body of evidence for <unk> and we're pleased to share here that the long term data were recently published in the September 2021 issue of molecular genetics and.
Metabolism reports.
As well as presented at the recent ASN kidney week conference.
Long term data highlight stable renal function during treatment of the <unk>.
Eight five years of <unk> irrespective of treatment status gender or phenotype and inclusive of classic males.
The important part of the growing evidence supporting the use of Gulf rolled in.
Patients with amenable mutations.
On slide 10, we've called out several of the drivers and metrics, which will lay the foundation for growth this year and beyond as.
As we've mentioned we ended 2020 with more than 4500 patients in golf bolt, which was a little under half of the global market share of treated amenable patients.
So while we're achieving higher market shares in countries, where we have been approved the longest.
There is clearly still plenty of opportunity to continue to switch patients over to <unk>.
We also know that there are significant numbers of diagnosed untreated patients who have amenable variance and while the global mix remains about 60% switch patients and 40% naive patients in many geographies, we're starting to see stronger uptake in naive populations.
As we've said previously over the next few years, we expect to see the balance of switch and naive patients to be about 50 50, and then the long term, we expect to see that percentage of reverse in favor of naive patients.
All of that is underpinned by the impressive compliance and adherence adherence rates that continue to exceed 90%.
Reiterating our belief that those patients who go on Gallup polled stay on Gulf wind.
Importantly, the value of <unk> continues to be recognized by payers as well with the vast majority of insurance reauthorization granted in 2021 by U S payers and a very strong track record of successfully negotiating and renegotiating reimbursement outside the United States.
Our relentless focus remains on ensuring access to <unk> for anyone who needs it.
And as we touched upon last quarter, we keep exploring ways to expand the label highlighted with the European Commission approval there of the label for <unk> to the adolescent population. We continue to work now closely with government authorities to secure access to <unk> for eligible patients as quickly as possible.
Finally, another important driver of growth for <unk> is the ongoing geographic expansion in 2021, we've continued to add to the growing list of countries with reimbursement and expect to add more as we look to expand access to those fabry patients with amenable variance around the globe.
As a reminder, <unk> does.
<unk> received regulatory approval in over 40 countries and commercial sales in over 30 of those today.
So despite the recent COVID-19 related headwinds in certain geographies demand for golf hold worldwide continues to grow with some cues of potential new Gulf old patients building in multiple regions.
And again, we're confident we will end 2021 within our guidance range of $300 million to $315 million in full year global sales.
As a reminder, as part of that guidance. We project net new patient starts this year will be even greater than in 2020, and we're on track for this growth in the number of patients and corresponding revenue to be weighted to the second half of the year.
As the Covid impact continues to ease.
On slide 11, with 70 several years of performance and a successful track record. We can confidently say, we're on a path to achieve the important milestone of $500 million in global revenue.
In a given year from <unk> within the next few years.
That timing of this milestone will depend on how quickly things return to normal post COVID-19 and we'll have a better sense of that depending on how this year ends up.
We continue to expect to generate $1 billion in cumulative revenue over the next three years, which will contribute a significant amount towards funding our R&D and operating expense over that period, and we remain confident in the $1 billion peak revenue opportunity for.
<unk> as we continue to see significant growth in the fabry market globally, driven by diagnosis from high risk screening newborn screening and other diagnostic initiatives, which we continue to support and invest in as well.
Finally, we have orphan exclusivity in the U S and Europe.
<unk>, two or <unk> 27, Orange book listed patents that give us IP coverage into the late 2030, 13 of which provide protection through 2038.
So quite a bit of opportunity to provide access to <unk> globally for a long periods of time.
And with that let me now turn the call over to Dr. Jessica <unk>, Our Chief Development Officer, who will start on <unk>.
Our next generation therapy for Pompe disease.
Jeff.
Thank you Brad and good morning, everyone.
Moving onto our R&D update on.
On slide 13, we will start with <unk> our novel next generation therapy for Pompe disease.
First it's always important to recognize that Pompeii continues to pose a range of health challenges for people affected by the disease and having therapeutic choices and options is crucial pompei is a severe and fatal neuromuscular disease and one of the most prevalent lysosomal disorders and.
In addition to the individual human tragedies, we've seen multiple publications and natural history studies.
Pompeii patients that highlight the initial benefit of treatment that is often followed by continued long term decline on key measures of disease for many individuals.
As a reminder, this sustaining high unmet need for the year experienced population is only ever been studied in a controlled setting in our phase III propel clinical trial. All other controlled late onset Pompe disease studies to date have been in participants naive to treatment.
Moving on to Slide 14, we present, a summary of the primary and key secondary and biomarker endpoints from our phase III propel study as a reminder, the study was double blind randomized setting the efficacy and safety of <unk> in adult treatment naive and <unk> experienced patients against approved therapy out with confidence.
Uh huh.
Here in the slide grouping these endpoints into domains that motor function muscle strength pulmonary function patient reported outcomes and Biomarkers you can see the majority of endpoints across all of these domains favor <unk> over other companies Alpha and both the overall and <unk> experienced population.
We believe this consistency of effect across the key disease manifestations illustrates the potential impact of atg for pump patients.
As a reminder, on the primary endpoint of six minute walk distance patients on <unk> outperformed those on how good properties alpha in the overall population, but the difference between groups that support the meters, which did not quite reach statistical significance for superiority. However on the first key secondary endpoint of percent predicted forced vital capacity.
Or do you see in that overall population <unk> showed a nominally statistically significant and clean the clinically meaningful difference for superiority versus <unk> alpha with the treatment difference of 3% and a P value 0.0 to three.
This is a really important finding is progressive loss of pulmonary function as the leading cause of mortality in Pompeii and this was the main endpoint upon which <unk> Alpha was initially approved.
We remind everyone that the propel study tested for superiority versus an approved therapy not versus placebo and that the Barker approval of a second product generally requires demonstration only of non inferiority to improve therapy.
We're also very pleased to announce that we expect the phase III propel results to be published shortly in a prestigious peer reviewed medical journal.
Moving on to Slide 15, we have highlighted key updates across the ACG program first on the regulatory progress. The U S. FDA has accepted for review the BLA for cyclical cost savings Alpha and the NDA for <unk> as we previously announced.
These two components have Paducah action date set for May 29, 2022 for the NDA in July 2009, 2022 for the BLA as John mentioned, we expect these filings to be reviewed together.
We're also pleased to share as John announced that EMEA has now been submitted to the European Medicines agency.
In June we announced also that <unk> was granted a positive scientific opinion through the early access to medicine scheme for <unk> by the Uk's NHRA.
This positive opinion recognizes the high unmet medical need faced by the pump a community and permit eligible adults living with late onset pompe.
Who have received anchored properties offer for at least two years to switch and to have access to <unk> prior to marketing authorization in the U K.
We're really excited to see that.
<unk> enthusiasm for <unk> under the <unk> mechanism with multiple patients and physicians, having requested access across the leading pump eight centers since that positive scientific opinion in June the interest and momentum for <unk> has grown and we are pleased to be able to provide access to those who are eligible in the U K.
For the younger pump a community we continue to enroll the ongoing open label study in children up to 18 years of age living with late onset pompe disease, and look to expand into patients with infantile onset pompe disease as soon as possible.
At this point, we're pleased to report that more than 150 patients worldwide are being treated with <unk> and.
And finally in response to the many requests for extended access that we've received our expanded access programs for both of those living with infantile onset in late onset pompe disease continued to expand to multiple individuals.
Moving on now to slide 17, we briefly highlight our industry, leading portfolio of gene therapies for rare diseases and the planned launch of our character.
To remind everyone as John noted commission of Caritas is to transform the lives of children and adults living with rare genetic diseases through advanced protein engineering and innovative vector technologies.
We see significant opportunities for tariff talks to overcome current gene therapy challenges around safety durability manufacture ability and immunogenicity, we have and are developing the tools and technologies to address these challenges and realize the promise of gene therapy.
The <unk> portfolio includes both clinical and preclinical programs.
In addition to the two batten programs in the clinic for <unk> III, we expect to see three new <unk> over the next 24 months and other rare genetic diseases, including Fabry and Pompe <unk>.
This also includes the global rights to approximately 50 rare diseases, which include the majority of the lysosomal diseases as well as nearly a dozen larger rare diseases and our collaboration with the University of Pennsylvania.
We've disclosed previously that these include Angelman Myoclonic dystrophy. In addition to multiple muscular dystrophy, including DMD.
As part of this transaction, we will continue to jointly work with the University of Pennsylvania, as well as with amicus through an attractive risk and cost sharing partnership.
For manufacturing, we have a fully design ready to build state of the art 35000 square foot clinical manufacturing facility with commercial expansion capability as planned and together with our continued strong relationship with thermo Fisher on the CDM upfront.
All of this is being led by an experienced leadership team with a fully staffed discovery research and development organization, a passionate entrepreneur and problem solvers.
Slide 18 highlights, our fabry and Pompe gene therapy programs, which amicus and Caritas will share co development and profit.
Profit sharing rights to <unk>.
This program will utilize a differentiated gene therapy approach for greater potency and optimized cross correction for protein.
Nearing of stability and targeting the.
The partnership between Amicus and carrier task will ensure the continued relationship with a leading internal experts in fabry and Pompe disease working on these programs.
The partnership also Derisked the funding of those programs and provides a 50 50 global profit split.
We expect the fabry IND to be filed first and the second half of next year.
Last and to build on John's earlier comments on our ceiling six patent gene therapy program I would like to reiterate the sentiment around the loss of the two children. During the long term follow up period of this study.
As John mentioned, the investigator deemed that both participants passed away from disease related complication unrelated to the study drug.
We continue to assess all available information as a reminder, our phase <unk> clinical study enrolled 13 participants who received a single <unk> injection of <unk>.
Gtx 501.
At a dose of one five either 13 vector genomes.
The first children in the study received their gene transfer over five years ago.
Medical literature indicates that most individuals with <unk> batten disease diagnosed during infancy and early childhood and do not provide beyond childhood.
Early adolescence.
While extremely signing news, we recognize the devastating nature of this disorder.
To see the potential benefits of gene therapy broadly.
With that I would like to now turn the call over to Daphne Feeney, Our Chief Financial Officer to review, our financial results guidance and outlook Daphne.
Thank you, Jeff and good morning, everyone. Our financial overview begins on slide 20, with our income statement for the third quarter ending September 32021 for the quarter, we achieved <unk> revenue of $79 5 million, which is an 18% increase over the same period last year.
This includes a year over year operational revenue growth measured at constant currency exchange rates of 17% further benefited by a positive currency impact of 1%.
Total operating expenses of $110 2 million in the third quarter were down as compared to $111 8 million in the third quarter of 2020.
On a non-GAAP basis total operating expenses were $93 6 million in the third quarter as compared to $92 4 million in the third quarter of 2020, we.
We define non-GAAP operating expense as research and development and SG&A expenses, excluding share based compensation expense changes in fair value of contingent consideration and depreciation.
Net loss for the third quarter of 2021 was $50 3 million or <unk> 19 per share as compared to a net loss of $64 million or 25 per share for the prior year period.
As of September 32021, we had approximately 279 million shares outstanding.
Turning now to slide 21, and 200 million private investment in the quarter added to our strong cash position, we are on a path to self sustainability and profitability in 2023.
<unk> achieved this milestone through our continued revenue growth with California.
Driving efficiencies cost savings and careful expense management.
For the third straight year, we expect total non-GAAP operating expenses in 2021 to remain relatively flat as we leverage the commercial infrastructure is already in place for the <unk> launch and other products in our pipeline.
Transition costs associated with the development of <unk> to multiple gene therapy programs in the pipeline and maintain financial discipline, while meeting our objectives.
A few comments about our cash position and 2021 financial guidance.
Cash cash equivalents and marketable securities were 557 million at September 32021, as compared to $483 3 million at December 31 2020.
We are reiterating our full year, California revenue guidance expecting to be within the $300 million.
$15 million and maintaining our non-GAAP operating expense guidance of $410 million.
Anthony.
The timing of manufacturing batches to impact.
Fourth quarter operating expenses.
With that let me turn the call back to John for closing comments.
Great. Thank you Daphne and Jeff Bradley as well as you can see we've been relentlessly focused on execution and performance across the business.
Driven by a global team of passionate entrepreneurs, who have led and will continue to lead us on our patient focused mission.
Our immensely excited for what the future of science and biotechnology hold.
As we continue to advance toward our commitment to people living with rare diseases through not only amicus beginning with the next earnings cycle with Caritas therapeutics as well so with that operator, we're happy to take any questions.
Ladies and gentlemen, if you have questions. Please press star and then the number one on your touch tone telephone.
At this time, we ask that you only ask one question. If you have any additional questions. Please enter back into the queue and if your question has been answered or you wish to remove yourself from the queue. Please press the pound key.
Thank you.
Your first question comes from the line of free Tomorrow with Cowen. Your line is now open.
Good morning team. This is under the Ontario to today on the to attach in the CNS six study could you perhaps provide a little more color on how all of these patients slower than they were diagnosed and what was the rate of decline on the hamlets handbooks colors, and the extension period and probably more broadly could you.
We expect a clinical data update at wood from the <unk> six and <unk>.
<unk> programs.
Sure. Thank you again, I'll remind everybody of the batten program came to us through the acquisition of sell in X.
Which was a spin out of nationwide Childrens Hospital board than three years ago. The 13 children dose in that study.
Children, who were dosed about five five years ago. The last patient was dosed more than three years ago. So again to reiterate the investigator is deemed as not to be related to study drug I want to be very clear about that these children were among the oldest back one of the two was the oldest in the study upon it.
Enrollment and had already advanced in their disease prior to the gene therapy.
With that said, we'll continue to evaluate all of the children, who remain in the study gather data on the children, who did pass away.
So.
Ill turn it to Jeff for Mitch for any additional color or comments.
John This is Jeff I don't have anything to add to it you noted other than we continue to follow the patients across both studies.
We're not noting whether we will have data at world or not but we do expect to provide data updates next year as we continue to collect data from those extension studies.
And continue to work on manufacturing of Thermo Fisher to provide GMP material in behalf of clinical and regulatory discussions with the agencies.
To move these forward.
Great. Thank you.
Your next question comes from the line of Anil <unk> with Jpmorgan. Your line is now open.
Hey, guys. Thanks, so much for taking the question I know you probably can't get in a lot of detail here, but how has the engagement been with the FDA on the <unk> filing and on the regulatory side and has there been any indication of a potential AD com for the filings. Thanks, so much.
Yes. Thank you on Oklahoma I will say the FDA has been highly engaged and interactive.
The review is continuing.
Inspections are underway. So everything we would expect we continue to receive information request. So it's been a highly engaged.
Reviewed to date, which we're very pleased with I will note that we have received in writing for the SBA debt there will not begin advisory committee.
So while again of course, the FDA will always reserve the right to revisit that as of now the FDA has confirmed that they will not be an ad com.
Thank you. Your next question comes from the line of Joseph Schwartz with SBB Leerink. Your line is now open.
Alright, Thanks, very much I was wondering if you could give us an update on your plans to study infantile onset pompe disease and get <unk> approved in this population.
How should we think about the size of this opportunity as it would compare with the size of the population with late onset pompe disease.
Great. Thank you for the question Joe studying it the infantile onset pompe disease population is incredibly important to us we expect that study to begin the formal study to begin very shortly as we finalize the protocols with FDA and the EMA.
It is a relatively small portion of the population, we would estimate it to be perhaps 10% or less and again in terms of.
An opportunity.
For this product again based on weight based dosing.
And even smaller proportion there, but obviously incredibly important to be able to treat these children I will remind everybody that we have treated under our global expanded access or compassionate use program a number of incidents and we're very very pleased with the results that we've seen in those intense.
Your next question comes from the line of Dae Gon Ha with Stifel. Your line is now open.
Great. Good morning, Thanks for taking our questions and John If this is my last time speaking with you. Good luck on your next adventure and look forward to keeping in touch.
Wanted to ask.
I don't know what a question.
As with regards to Pompeii as we're gearing up for your progress with <unk>. Just wondering if you guys have done any latest market research with regards to <unk> and <unk> uptake and how that is fairing in the current landscape as you're gearing up your commercial sales force. Thanks.
Yes, let me just broadly why don't you go ahead and field that please thanks Dan.
Sure obviously.
Careful not to speak on other product out there in this space, but we can share whats available publicly we know that the Pompeii market continues to grow we think that reflects the high demand.
Four four treatments for Pompe disease.
We also encourage you to look at the latest reports on the uptake around.
The more release product in United States.
Jay.
And I think Sanofi released those numbers recently, so I think that would be a good indicator of at least there their initial uptake there I will say that focusing on <unk>.
<unk>.
We continue to expect high demand for that product of course, we have to go through the regulatory process, but I do think the color that Jeff provided in particular around the <unk> process of U K shows that where <unk> is now available through a formal mechanism, albeit pre reimbursement pre approval mechanism.
We're seeing significant demand and interest for use of that product and we hope that reflects.
Physicians confidence that there is an opportunity for <unk> to make a major difference here.
And clearly for the unmet medical need in the pump space.
Your next question comes from the line of <unk>, Zhang with <unk>. Your line is now open.
Hi, good morning, Thanks, very much for taking my question. So a question on your February.
February gene therapy program, and given that you have <unk> in the market and also excuse me. Several other gene therapy programs are already in clinical studies and have reported quite an interesting data. So how do you think youre program, we'll be able to compete with existing programs. What part of the program do you think is most of it.
June: Programs are already in clinical studies and have reported quite interesting data. So how do you think your program will be able to compete with the existing programs? What part of the program do you think is most differentiated?
<unk>.
John: Yeah, great, thank you, June. So again, we've had great experience working in the February community since the day we started amicus, and we think it'll be incredibly important to understand the unmet needs in that community, the nature of clinical studies, regulatory pathways, and eventually, providing 100% access to everyone living with February disease in the world. You know, I think for gene therapy, we continue to believe that the greatest unmet need will be for those patients who don't have access to Gallifold today, so those patients with non-amendable mutations. And we've got a commitment.
Yes, great. Thank you and so again, we've had great experience working in the Fabry community for really since the day, we started amicus and we think that'll be incredibly an important important to understand the unmet needs in that community. The nature of clinical studies regulatory pathways and eventually providing 100%.
SaaS to everyone living with fabry disease in the world.
I think for gene therapy, we continue to believe that the greatest unmet need will be in those patients who don't have access to Gallup poll today. So those patients with non amenable mutations and we've got a commitment it's part of the amicus promise to patients that we would continue to forever designate a portion a significant portion.
Jeff: It's part of the amicus promise to patients that we would continue to forever designate a portion, a significant portion of our revenue from Gallifold into February disease to not only develop treatments but ultimately until there is a cure. And we think this does have the potential to be that cure. We've developed something we think is highly differentiated. And, very importantly in the February market, while several firms may be the first to the clinic, we would hope to be the first out of the clinic.
<unk> of our revenue from Gallo fold into fabry disease to not only develop treatments, but ultimately until there is a cure and we think this does have the potential to be that cure.
We've developed something we think is highly differentiated we think very importantly in the February market. While several firms may be the first to the clinic, we would hope to be the first out of the clinic and I think thats very important and that gets to the nature of what we've created again the whole notion.
Jeff: And I think that's very important, and that gets to the nature of what we've created. Again, the whole notion of what we've developed in gene therapy at amicus is to look at each disease, understand its unique biology, the unique aspect of the disease, the clinical manifestations, and develop a therapeutic or a gene therapy that can best address that. Let me turn it to Jeff. Jeff, if you want to just remind everybody about the drug candidate that we've developed in conjunction with Dr. Wilson and his team at Penn and why we think that's differentiated. Sure, thanks, John.
For what we've developed in gene therapy at amicus is to look at each disease understand its unique biology, the unique aspects of the disease, the clinical manifestations and develop a therapeutic gene therapy that can best address that.
Let me turn it to Jeff Jeff If you want to just remind everybody. The drug candidate that we've developed in conjunction with Dr. Wilson and his team at 10 and why we think that's differentiated.
Sure. Thanks, John Yes, Theres really two main components that we think differentiate our approach. One is this is hugh.
<unk> fabs that and promoter with the <unk>.
So we get expression not only directly in the cells that are transduce.
Jeff: Sure, thanks, John. Yeah, there are really two main components that can differentiate our approach. You know, one is that this is a ubiquitous abstinent promoter with the A.V. So we get expression not only directly in the cells that are transduced but can also rely on some cross-correction. That ubiquitous expression can help not only create more of the enzyme out into circulation but also could be more durable. And then secondly, and really what's differentiated is that we've created a trans gene that expresses a GLA that has a stabilized dimer through two engineered disulfide bonds.
But also can rely on some cross correction that ubiquitous expression can help not only create more of the enzyme out in the circulation, but also it could be more durable.
And then secondly, and really whats most differentiated is that we've created a trans gene that expresses GLA that is.
Stabilized dimer through two engineered disulfide bond that stabilize enzyme is not only more stable in the blood as it's accretive and then travels to treat other cells and tissues, but even after uptake into tissues and cells. It looks like it is more active so we've done studies comparing that stabilized transgene versus.
As the wild type GLA transgene, and just see that we get much more substrate clearance at a given dose with that more potent enzyme that's being expressed so we're really excited as John mentioned about the opportunity here to have a what we believe has the potential for a best in class AAV gene therapy in February.
Jeff: That stabilized enzyme is not only more stable in the blood as it's secreted and then travels to treat other cells and tissues, but even after uptake into tissues and cells, it looks like it is more active. So we've done studies comparing that stabilized trans gene versus the wild-type GLA trans gene and just see that we get much more substrate clearance at a given dose with that more potent enzyme that's being expressed.
And in terms of unmet need for that non amenable fabry portion of the market, which is over half of the patients out there their only option is every other week infusions. Currently we view there is a real need there for a onetime treatment, which would be a big market expansion from an amicus perspective.
Again, if you would like to ask a question press Star and then the number one on your telephone.
Our next question comes from the line of Neil.
Gandhi with Needham <unk> co. Your line is now open.
Hi, This is vishal among for Gil Thank you for taking our questions.
No.
It is.
Six seven months away, but are you planning any way like thinking of commercial launch.
Planning for sales force ramp up or are you waiting on the decision before proceeding.
Yes. Thank you Bradley you want to fill that please.
Sure of course, yes. Thanks for the question, we are very much geared towards commercial launch and anticipating approvals in the summer of next year as John articulated.
Jeff: So we're really excited, as John mentioned, about the opportunity here to have a, you know, what we believe has the potential to be a best-in-class AAD gene therapy in February. And, you know, in terms of the unmet need for that non-amendable February portion of the market, which is over half of the patients out there, their only option is every other week earkey infusions currently. Review There's a real need there for a one-time treatment, which would be a big market expansion from an amicus perspective.
Really excited to do that of course this would be the second product that amicus has brought from discovery all the way through to two launch.
And we will be an exciting milestone for the company importantly to your point about building commercial infrastructure.
Good news is the infrastructure, we built for golf bold is highly leverage able here.
Many of the same physicians and centers are all responsible for treating pompe diseases are responsible for treating fabry disease.
We said previously is that in order to support the launch of Gulf or excuse me of Atg AA, we only anticipate a handful of additional ftes may be in the sort of.
Dozen or so ftes to support that launch primarily in.
Our amicus assist team as well as medical affairs and direct marketing. So that's one of the great things about the team that we've built to support <unk> as they will also be able to support the launch of ATCA, but rest assured we are eagerly anticipating that opportunity and doing all the things to prepare to be ready for an exciting launch.
Operator: Again, if you would like to ask a question, press star and then the number one on your telephone. Our next question comes from the line of Nishant Gandhi with Niedam and Co. Your line is now open.
Our next question comes from the line of Eliana Merle with UBS. Your line is now open.
Hey, this is Johnny Entre Ali.
In terms of.
Papa early access in the U K can you update us on any trends youre seeing there in terms of obtaining switches from me Archie. Thank you.
Great. Yes. Thank you again to remind everybody. We received the early access approval at the end of May since that time, we've been working through all the health authorities. The regional authorities in the United Kingdom to enable the next level of approvals.
Gil: Hi, this is my chance. I'm on for Gil.
Bradley: Thank you for taking our questions. I know Pidu Fadid is, you know, six, seven months away, but are you planning in any way, like thinking of a commercial launch? Like, are you planning for sales, for ramp-up, or are you waiting on the decision before proceeding?
Made significant progress there again, we really are seeing significant enthusiasm we have received.
For multiple key opinion leaders multiple centers throughout the United Kingdom requests to switch patients from the approved standard of care enzyme therapy to atg AA.
Bradley: Yeah, thank you, Bradley. You want to feel that. Sure, of course, yeah, thanks for the question.
We've been working through that for all the approvals necessary to switches, we're starting to see patients now switched which we're very encouraged by and we think that that will continue and that momentum will grow.
Bradley: Sure, of course, yeah, thanks for the question. We are very much geared towards commercial launch and anticipating approvals in the summer of next year, as John articulated. I'm really excited to do that. Of course, this would be the second product that Amicus has brought from discovery all the way through to launch, and it will be an exciting milestone for the company. Importantly, to your point about building commercial infrastructure, the good news is the infrastructure we've built for Gallupold is highly leverageable here.
In the months ahead.
Bradley anything I Miss feel free to add color.
No I think you've captured that John thanks.
Great. Thank you that's a very significant commitment for us and again something we're very excited about to provide 80 GAA in the United Kingdom to two adults eligible to switch.
We have a follow up question from me Tomorrow from Cowen. Your line is now open.
Hi, just fine without on again, sorry, ritu. Thanks for taking a follow up question and apologies if I missed this earlier of the 150 plus patients on atg.
So Lee could you provide us a breakdown of open label extension patients switched as early access in the U S versus any other compassionate use thank you.
Yes, we don't provide specific numbers for all of that again the vast majority of those patients are on the long term extension study following the propel data. We also have all of the phase one two patients who continue on 80 GAA. We now have patients in the adolescent study.
Bradley: Many of the same physicians and centers are responsible for treating Pompeii disease as are responsible for treating Fabri disease. What we said previously is that in order to support the launch of ATGA, we only anticipate a handful of additional FDEs, maybe in the sort of, you know, a dozen or so FDEs to support that launch primarily in our Amicus Assist team as well as medical affairs and direct marketing. So one of the great things about the team that we've built to support Gallifold is that they'll also be able to support the launch of ATGA. But rest assured, we're eagerly. Anticipating that opportunity and doing all the things to prepare to be ready for an exciting one.
Again, we expect to begin the infantile study very quickly and we do have an expanded access program that includes a range of patients, including infants as well. So it really is a mix in terms of disease onset and characterization within the spectrum of Pompe disease and importantly, it's also a mix across <unk>.
Clinical sites with dozens and dozens of investigators now caring for these patients receiving 80 GAA on now five continents, so geographically quite dispersed.
At this time I would now like to turn the conference back to Mr. John Crowley, Chairman and CEO for closing remarks.
Great operator to.
To all the analysts. Thank you for all the great questions. Today again, a good very strong quarter for amicus on Q3, and we look forward to a very strong closeout to the year. Thanks, everybody have a great day take care.
This concludes today's conference call. Thank you and have a great day.
Operator: Our next question comes from the line of Eliana Merle with UBS. Your line is now open.
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Johnny Antra: Hey, this is Johnny Antra speaking, Ellie. In terms of Poppa Early Access in the UK, can you update us on any trends you're seeing there in terms of updates and switches from EURT? Thank you.
John: Great, yeah, thank you again to remind everybody. We received early access approval at the end of May. Since that time, we've been working with all the health authorities, the regional authorities in the United Kingdom, to enable the next level of approvals. We've made significant progress there. Again, we really are seeing significant enthusiasm. We've received requests from multiple key opinion leaders, multiple centers throughout the United Kingdom, to switch patients from the approved standard of care, care enzyme therapy, to ATGAA.
John: We've been working through that for all the approvals necessary, and the switches. We're starting to see patients now switched, which we're very encouraged by, and we think that that will continue and that momentum will grow in the months ahead. Bradley, anything I miss, feel free to add color. Thank you.
Bradley: No, I think you've captured it, John. Thanks.
John: Great, thank you. Yeah, it's a very significant commitment for us and, again, something we're very excited about providing ATGAA in the United Kingdom to adults eligible to switch.
Operator: We have a follow-up question from Rita Barrel, from Colin. Your line is now open.
Rithu: Hi, this one is without on again for Rithu. Thanks for taking our follow-up question and apologies if I missed this earlier. Of the 150-plus patients on ATGA globally, could you provide us a breakdown of open-label extension patients versus early access in the U.S. versus any other compassionate use? Thank you. Yeah, we don't provide specific numbers for all of them.
Speaker: Yeah, we don't provide specific numbers for all of that. Again, the vast majority of those patients are on the long-term extension study following the propelled data. We also have all of the phase one, two patients who continue on ATGAA. We now have patients in the adolescent study. Again, we expect to begin the infantile study very quickly, and we do have an expanded access program that includes a range of patients, including infants as well.
Speaker: So it really is a mix in terms of... disease onset and characterization within the spectrum of Pompeii disease. And importantly, it's also a mix across clinical sites with dozens and dozens of investigators now caring for these patients, receiving ATGAA on now five continents, so geographically quite dispersed.
Operator: At this time, I would now like to turn the conference back to Mr. John Crowley, Chairman and CEO, for a closing remark.
John Crowley: Great operator to all the analysts. Thank you for all the great questions today. Again, a good, very strong quarter for Amicus in Q3, and we look forward to a very strong close-out to the year.
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Speaker: Thanks, everybody. Have a great day. Take care. This concludes today's conference call.
Operator: This concludes today's conference call. Thank you, and have a great day.
Speaker: and so on. Thank you. Thank you. Thank you. Thank you. Thank you. Thank you. Thank you. Thank you, Thank you, thank you. Thank you. Thank you. Thank you.