Q4 2023 argenx SE Earnings Call
Operator: Good morning. My name is Rob, and I will be your conference operator today. I would like to welcome everyone to the call. At this time, all lines have been placed on mute to prevent any background noise.
Good morning, My name is Robyn I Hope your conference operator today I would like to welcome everyone to the call at this time all lines had been placed on mute to prevent any background noise. After the speaker's remarks, there'll be a question and answer session. If you would like to ask a question. During this time simply press start followed by the number one on your telephone keypad, if you would like to.
Operator: After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press the star followed by the number one on your telephone keypad. If you would like to withdraw your question, again, press the star one. Thank you. I'd like to introduce Beth DelGiacco, Vice President, Global Head of Corporate Communications and Investor Relations. You may now begin your presentation. Thank you.
Best: Draw. Your question again press the start one thank you I'd like to introduce best <unk>, Vice President Global head of corporate Communications and Investor Relations. You May now begin your conference.
Unknown Executive: A press release was issued earlier today with our full-year financial results and recent business updates. This can be found on our website along with the presentation for today's webcast. Before we begin, I'd like to remind you on slide two that forward-looking statements may be presented during this call. These may include statements about our future expectations, clinical development, regulatory timelines, the potential success of our product candidates, financial projections, and upcoming milestones. Actual results may differ materially from those indicated by these statements. ArgenX is not under any obligation to update statements regarding the future or to conform those statements in relation to actual results unless required by law.
Best: Okay.
Best: Earlier today with our four year financial results in recent business update this can be found on our website along with a presentation for today's webcast.
Best: Before we begin I'd like to remind you on slide too that forward looking statements may be prevented during this call.
Best: Two statements about our future expectation clinical development regulatory timeline.
Best: Both of our product candidate financial protection and upcoming milestones at.
Best: Excellent results may differ materially from those indicated by these statements are.
Best: That makes it not under any obligation to update statements regarding the future or to confirm those statements in relation to actual resolved unless required by law.
Beth DelGiacco: I'm joined on the call today by Tim Van Hauwermeiren, Chief Executive Officer, Karl Gubitz, Chief Financial Officer, and Karen Massey, Chief Operating Officer. I will now turn the call over to Tim. Thank you, Beth, and welcome, everyone.
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Speaker Change: <unk>, Chief Financial Officer, and Karen Matthews, Chief operating Officer, I will now for the call over to test.
Speaker Change: Thank you Beth and welcome everyone.
Tim Van Hauwermeiren: I'll begin on slide 3. This has been an overview of incredible execution by the ArgenX team, setting us up to build on this momentum for the year ahead. At the core of everything we do is our mission to transform the lives of patients suffering from autoimmune diseases. And today we are in a better position than ever to deliver on this mission, reaching more patients globally with our first-in-class innovation and bringing hope to the autoimmune community about what a novel treatment can offer. We see multiple opportunities to expand our patient impact this year and are investing across our business to do so. These innovation horizons serve as a roadmap for how we will build long-term value by expanding the VipGuard opportunity, advancing our pipeline, and bringing the next wave of R&D candidates into the clinic. In today's call, I would like to highlight recent news and walk through upcoming milestones in the context of these horizons. Slide four.
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Speaker Change: The code of everything we do is our mission to transform the lives of patients.
Speaker Change: Suffering from ultimate diseases.
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Speaker Change: We are in a better position than ever to deliver on this mission reaching.
Speaker Change: Reaching more patients globally.
First in class innovation and bring hope to the ultimate community.
Speaker Change: What are the local treatment can offer.
Speaker Change: You see multiple opportunities to expand our patient in fact this year.
Speaker Change: Interesting across all of our business to do so.
Speaker Change: These innovation horizon surfer.
Speaker Change: Surface zero map for how.
Speaker Change: How we will build long.
Speaker Change: Value.
Speaker Change: Spending the gift card opportunity.
Speaker Change: Fencing of a pipeline.
Speaker Change: Next week I'll be candidates into the clinics.
Speaker Change: After this call I would like to highlight with some juice and walk through upcoming milestones in the car.
Speaker Change: Text of these horizons.
Speaker Change: Slide four.
Tim Van Hauwermeiren: Let's start with the discard opportunity. We had an incredible second year of launch, driven by the ambitious strategies and seamless execution of our commercial and medical teams. And we are still at the beginning of what we hope to achieve. First and foremost, the last trajectory demonstrates the significant unmet need that still exists with GMG and the opportunity for an innovation like Vyvgart to deliver differentiated outcomes for patients. As a part of our commitment to the broader MG community, we will initiate a registration trial in seronegative patients this year, which, if positive, could allow us to reach the 15% of GMG patients who are not served by our current label. Beyond M.G.
Speaker Change: Let's start with that opportunity.
Speaker Change: We had an incredible second veto planch driven.
Speaker Change: Driven by the ambitious strategies and seamless execution of our commercial and medical teams.
Speaker Change: And we are still at the beginning of four P hope to achieve.
Speaker Change: First and foremost.
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Speaker Change: They're still exists with G M G.
Speaker Change: And the opportunity for innovation like this car to deliver differentiated outcomes for patients.
Speaker Change: As a part of our commitment to the boat or Angie community we.
Speaker Change: We will initiate a registration trial.
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Speaker Change: Which is positive include allowed us to the 15% of James G patients who are not served by our current label.
Speaker Change: Beyond N G.
Tim Van Hauwermeiren: We continue to demonstrate the breadth of possibility for SCRM across all communications, and we have launch preparations underway ahead of anticipated regulatory decisions in ITP in Japan in March and in CIDP in the US in June. We will be ready to tackle these anticipated opportunities as we leverage our key learnings from our launch playbook to best position ourselves for success. Slide five.
Speaker Change: We continue to demonstrate the breath of possibility for a C N N across ultimate indications.
Speaker Change: And we have lunch preparations underway.
Speaker Change: Anticipated regulatory decisions in ITT in Japan in March.
Speaker Change: C I D P in the U S in June.
Speaker Change: We will be ready to tackle this anticipated opportunities.
Speaker Change: We leave whichever key learnings.
Speaker Change: From Avalanche playbook to best position ourselves for success.
Speaker Change: Slide fine.
Tim Van Hauwermeiren: Click up to finish to make headway in the clinic. The phase 3 TED study is slated to start this quarter and will be the first to utilize our presence syringe from the onset. We are expecting readouts from five Phase 2 studies, including in children, post-COVID hots, and three subtypes of myositis.
Speaker Change: Pick up the changes to make headway in the clinic.
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Speaker Change: We are expecting beat us from five phase two studies, including insurance costs covered.
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Tim Van Hauwermeiren: And we expect to provide an update on our plans in Bullis-Pensacola later this year, once the team has had the opportunity to analyze the data from patients who are involved in violet stage 8. This is part of our commitment as a learning organization and our ongoing work to double-click on the clinical feasibility of current and future ad hoc studies based on key insights from Advanced SC and ADDRESS. This will be another year where we learn more about the broad potential of SCRN through our next wave of indications, advancing our leadership in the class, and unraveling important findings about the underlying biology of these autoimmune diseases. Today, we will focus on our expectations of success around children, as that will be the first of the five.
Speaker Change: And we expect to provide an update on our plan.
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Speaker Change: This is part of our commitment as a learning organization and our ongoing work to double click on the clinical feasibility of current and future take about studies based on facts from advance S T and address.
Speaker Change: This will be another year will be learn more about the potential of F. C. N N through our next wave of indications.
Speaker Change: Advancing are the leadership of the class and unraveling important findings about the underlying biology of this auto immune diseases.
Speaker Change: Today, we will focus on our expectations of success around sugars is.
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Tim Van Hauwermeiren: The ROOF study has a target enrolment of 30 models with severe patients, randomised to two options. The study is not piled for efficacy, so we will rely on the depth of data we will gather from each patient, looking at various endpoints, including CRES, SDI, ESPRI, and biomarkers across patients. We have a few objectives with this signal finding study to confirm the role of IgG autoantibodies in mediating disease in children.
Speaker Change: The roof 30, as a target and go over the 30 moderate to severe patients randomized to walk.
Speaker Change: The study is not pilots for efficacy.
Speaker Change: Relied on the depth of data.
Speaker Change: For each patient.
Speaker Change: At various endpoints.
Speaker Change: Including Chris.
Speaker Change: Esprit.
Speaker Change: And biomarkers across patients.
Speaker Change: We have a few objected through the signal finding study.
Speaker Change: 30.
Speaker Change: To confirm the role of <unk> auto antibodies immediate thing disease insurance.
Tim Van Hauwermeiren: To evaluate a combination of efficacy and biomarker data to gain sufficient confidence to move forward in this indication, and third to inform potential patient selection and endpoints to design a winning registrational trial to amplify any single we observe in phase two. We plan to employ a similar approach in our evaluation of art. Looking at the depth of data across the enrolled patients to make an evidence-based decision to move into a phase 3 study. With my guidance, we have a seamless phase 2-3 design, which will expedite the transition from the first 30 patients of each subset into a registration study of one or more of the subsets where proof of concept has been established. Wrapping up on Ad Graphicum, I'm very proud of all that we have accomplished in pioneering this new class of medicine.
Second.
Speaker Change: To evaluate a combination of efficacy and buying market data to gain sufficient confidence to move forward into syndication.
And third.
Speaker Change: Phone potential patients selection and earn points to designer breathing registrational trial to amplify anything you'll be absorbed in phase two.
Speaker Change: We plan to employ a similar approach in our evaluation of pop.
Speaker Change: Looking at the depth of data across the enrolled patients to make an evidence based decision to move into a safety study.
Speaker Change: With my diabetes.
Speaker Change: <unk> seamless phase two three design, which will expedite the transition from the first 30 patients of each subset.
Speaker Change: The registration study of one or more of a successful proof of concept has been established.
Speaker Change: Wrapping up on I've got picked him up and very proud of all that we have accomplished.
Speaker Change: Pioneering this new class of medicines.
Tim Van Hauwermeiren: People-of-concept has now been demonstrated in nine out-of-nine indications across the CRM, and we believe this is still just the beginning of the broader opportunity. [inaudible] Turning attention to our next horizon of innovation, I want to briefly touch on our pipeline progress. MPAR is our second pipeline in a product opportunity from which we have shown compelling MMM data from the first patient cohort earlier this year. This is a program that emerged from our IIT and perfectly demonstrates how we like to build opportunities from our discovery engine into our pipeline and now towards a registration trial in its first indication. In collaboration with Professor Eric Pax, we built what we believe is a first-in-class and best-in-class sweeping antibody against C.2 and have conducted translational work to highlight where targeting C.2 could have the most impact. From this, we identify that complement activation in NLM happens upstream in the complement cascade.
Speaker Change: Proof of concept has not been demonstrated in nine out of line indications across the C. N N and we believe this is just the beginning of the broader opportunity.
Speaker Change: Slide sick.
Speaker Change: Turning attention to our next door as much innovation I want to briefly touch on our pipeline progress.
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Speaker Change: From which we have short compelling and the men data from the first patient cohort earlier this year.
Speaker Change: This is a program that emerged from our I T and perfectly demonstrate oh, we'd like to build opportunity for my auto discovery engine into our pipeline and activity Registrational trial, it's first indications.
Speaker Change: In collaboration Professor <unk> details what we believe is the first in class and best in class sweeping empty body again C. Two.
And have conflicted translation work to highlight with targeting situ could have the most impact.
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Speaker Change: Identify that compliment activation and annual happens upstream and the complement cascade.
Tim Van Hauwermeiren: We designed an innovative trial, and in the first course, we demonstrated a 91% reduction in the need for IVIG rescue compared to placebo. We are now awaiting the results of the second cohort to inform the final design of the registration trial and look forward to sharing the full face-to-face data set this year. The NMN opportunity fits perfectly within our expanding capabilities in neurology, as does ArgenX 1919, our third pipeline program. This molecule will come more into focus this year as we move beyond healthy volunteers into CMS and ALS patient studies.
Speaker Change: We designed an innovative trial and and the first call to be demonstrated in 91% reduction in the list for Ivy at G rescue compared to placebo.
Speaker Change: Right now we're waiting for the results of the second cohort too.
Speaker Change: To inform the final design of the registration trial.
Speaker Change: And look forward to sharing the food based student data set this year.
Speaker Change: The opportunity fits perfectly within our expanding capabilities in neurology.
Speaker Change: January 119.
Speaker Change: Third pipeline program.
Speaker Change: This molecule will go more into focus this year.
We move beyond healthy volunteers to CMS and less patient study.
Tim Van Hauwermeiren: Of note, we recently initiated natural history studies in both CMS and MLM to engage each of their respective patient communities. This falls in line with our strategy to better understand the real world experience of patients and will help us identify potential participants in upcoming studies. Slide 7, Finally, and core to our sustainable growth, is our third innovation horizon, our immunology innovation program. The track record of success of our ISP goes well beyond FKT, GMAT, and ENPA, with nine programs tested in humans since inception. We demonstrated the efficiency of this pipeline engine by nominating four new molecules last year.
Speaker Change: Of note.
Speaker Change: Recently initiated natural history studies in both C M S and M N.
Speaker Change: To engage each of their respective patient community.
Speaker Change: This falls in line with our strategy to better understand the real world experience of patients and will help us identify potential participants.
Speaker Change: <unk> study.
Speaker Change: Seven.
Speaker Change: Finally and quarter are sustainable growth.
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Speaker Change: The track record of success of our I S. P goes well beyond a package of <unk>.
Nine programs interested in human since inception.
Speaker Change: We demonstrated the efficiency of dispatch an engine by nominating for new molecules last year.
Tim Van Hauwermeiren: All are on track to be filed as IMDs by the end of 2025. We will continue to invest in our internal discovery engine and technical capabilities, combining our antibody engineering and clinical development expertise with the knowledge of leading scientific collaborators as we advance our next wave of molecules. I will now turn the call over to Carol. Thank you, Tim. Slide 8.
Speaker Change: All are on track to be filed this imd's by the end of 2025.
Speaker Change: We will continue to invest in have an internal discovery engine and technical capabilities.
Speaker Change: Finding out antibody engineering and clinical development expertise with a knowledge of leading scientific collaborated as we advance over the next wave of molecules.
Speaker Change: I will now turn the call over to Carol.
Speaker Change: Thank you Tim slide eight.
Karl Gubitz: The fourth quarter 2023 financial results are detailed in the press release from this morning, but I will highlight the key points here. Revenue in the fourth quarter totaled $418 million.
Carol: The fourth quarter, 20th <unk> financial results detailed <unk> release from this morning.
Carol: Highlight the key points here.
Carol: Revenue in the fourth quarter totaled.
Carol: Million dollars.
Karl Gubitz: This reflects $374 million in product net sales and $43 million in average income and collaboration rate. Our collaboration revenue includes a $30 million milestone payment from AbbVie for advancing ArgenX 1.1.5 to Phase 2, as well as royalty income from Xilab of $0.7 million for Vyvgart sales in China. A breakdown per region of $374 million in product net sales each year. $326 million in the U.S., $17 million in Japan, $24 million in EMEA, and $7 million in China. Globally, we saw growth of 14% or 45 million in your product net sales from Q3 2023 to Q4. Operating expenses in Q4 were $536 million.
Carol: $374 million and product made sales and $43 million in income and collaboration that Avenue.
Carol: Collaboration revenue includes a 30 million dollar payment.
Carol: Payment from Appy for advancing organics 115 to face too.
Carol: Well as royalty in <unk> <unk>.
Carol: <unk> 7 million record sales in China.
Carol: But Brian <unk> of a 374 million product made sales fees.
Carol: 26 million N B U S.
Carol: $17 billion in Japan, $24 million in new media and $7 million in China.
Carol: <unk>, we saw grove, or 40% or $45 million in your product made from Q3 2023 two Q4.
Carol: Okay, writing experiences in Q4 with $546 million.
Karl Gubitz: This is an increase of $136 million over Q3 2023, driven primarily by the recognition of a priority review voucher we submitted with the SBLA filing for CIDB. This impact of the PRV was $102 million and brings total R&D costs over the quarter to $306 million. Net loss for the quarter was $99 million, bringing the full year loss to $295 million.
Carol: This is an increase of $146 million over Q free 2023.
Carol: Primarily by the recognition.
Carol: And what are the review voucher, we submitted with an.
Carol: <unk> filing for C. I D P.
Carol: This impact of a peer review was handed me $2 million.
Carol: <unk> total Orange D cost 12, a quarter to $306 million.
Net loss for the quarter was $99 million, bringing a full.
Full year loss.
Carol: $95 million.
Karl Gubitz: We continue to have a strong balance sheet with 3.2 billion in cash, cash equivalents, and current financial assets at the end of the year. The financial guidance for 2024 is as follows. Based on our current operating plans, we expect the combined research and development and selling general and administrative expenses in 2024 will be less than $2 billion. And we expect to utilize up to $500 million of cash in 2024 for our anticipated operating expenses, as well as working capital and capital expenditures, including investment in our supply chain.
Carol: We continued to ask is throwing the balance sheet with 3.2 billion cash cash equivalent current financial assets at theory.
Carol: The financial guidance for 2024 is as follows.
Carol: On your current operating plans, we expect a combined.
Carol: Development, and selling general and administrative expenses, and 2024 will be less than $2 billion and.
Carol: And we expect to Utilise up to $500 million of cash 2024.
Carol: Our anticipated operating expenses as well as working capital M capital expenditure, including investment in your supply chain.
Karl Gubitz: I will now turn the call over to Karen, who will provide details on the commercial front. Thanks, Karl. Slide 10.
Carol: I will now turn the call over to Karen who will provide details on the commercial front.
Carol: Thanks.
Carol: 10.
Karen Massey: Echoing Tim's sentiments, I'm thrilled with the impact Vyvgart is having on patients and their loved ones. With over 6,000 patients on therapy, the response from the patient community has been tremendous, and we've set the bar high for what a novel GMG treatment can offer. I want to first thank the team because the success of our launch can only be achieved through tireless dedication and a firm commitment to our mission to transform the lives of autoimmune patients. Today, I would like to focus on three key areas which I believe will advance Vivgard's leadership and maximize the impact we can have on patients globally. One, reaching new GMG populations with Vyvgart. 2.
Karen Massey: Echoing Kim I'm thrilled with the impact Guy's, having a patient and their loved ones.
Karen: It is 6000 patients are therapy. The response from the patient community has been tremendous and we set the bar high for a novel AMG treatment can offer.
Karen: I want to first thank the team you can just access it at launch can only be achieved tireless dedication and a firm commitment to our mission to transform the lives of autoimmune patients.
Karen: Today, I would like to focus on three key areas, which I believe will advance of gas leadership and maximize the impact we can have on patients globally.
Karen: One reaching new G. M. G populations with your cat to leveraging add Knowhow from G. N G. As we prepare to potentially launching CIBC and I T T and three there'll be a commercial engine that can reliably and repeatedly maximize value creation inpatient impact.
Karen Massey: Leveraging our know-how from GMG as we prepare to potentially launch in CIDP and ITP 3. Building a commercial engine that can reliably and repeatedly maximize value creation and patient impact. Slide 11. Let's begin with our MGMORPG. We closed out 2023 with $1.2 billion in revenues, including over $1 billion in the US alone, which is a remarkable feat in just our second year of launch.
Karen: Again.
Karen: Let's begin with our M G launch.
<unk> 2023, with 1.2 billion in revenues, including over 1 billion in the U S. A line, which is a remarkable feat in just a second year of launch.
Karen Massey: Importantly, this tells us that patients place a high value on innovative treatments such as Vyvgart to meet the demand for safe and effective treatment alternatives in the GMG treatment space. We continue to see double-digit, quarter-over-quarter growth. And we contribute this momentum to several factors, including a broader prescriber base, the continued shift to patients earlier in the treatment paradigm, and additional regulatory approvals and launches in our ex-U.S. market. We see consistent growth in our subscriber base, which has breadth and depth amongst neurologists and further reach into community centers. Prescribers are becoming increasingly comfortable with the efficacy and safety of Vyvgart, which is supported by a body of real-world evidence demonstrating the consistent value Vyvgart can deliver to patients. As an example, we see rates of minimum symptom expression in the real world that mirror the data from our clinical trials, indicating that patients have the potential to achieve quality of life scores that are comparable to healthy populations. And we have over 4,000 patient units of safety follow-up with FGAR Tugamogs, which continues to support our consistent safety profile.
Karen: Importantly, this tells us that patients take the high value on innovative treatments such as we've got to make their day.
Karen: Man for safe and effective treatment alternatives in the G M G treatment space.
Karen: We continued to say double digit quarter over quarter growth and.
Karen: And we contribute I mentioned to several factors, including applauded prescribed the date.
Karen: <unk> shifts of patients earlier in the treatment paradigm, an additional regulatory approvals and marches in our S. U S market.
Karen: We see consistent growth without prescribe a day, having breadth and depth amongst neurologist and further reach into community centers.
Karen: Prescribed is it becoming increasingly comfortable with the efficacy and safety and safeguards, which is supported by a buddy of real world evidence.
Karen: Trading the consistent value.
Karen: Delivered to patients.
Karen: An example, we see rates of minimum can expression in the real world that mirror the data from <unk> clinical trial.
Karen: Indicating the patients have the potential to achieve quality of life scores that are comparable to healthy population.
Karen: And we have a 4000 patient safety follow up with your cats are good luck, which continues to support Atkins, if the safety profile and with additions is the key differentiator.
Karen Massey: And with physicians, this is a key differentiator. We are observing increased utilization of both Vyvgart and Vyvgart-Hytrullo in earlier lines of therapy, with an impressive 55% of patients coming to Vyvgart directly from Oral, and we only expect this trajectory to continue. Although Vyvgart still comprises the majority of prescriptions, we are seeing more traction with Hytrula, likely supported by Axis Dynamics, favorable payer policies that mirror Vyvgart, and a dedicated J-code in place.
Karen: We're driving increased utilization adverse data and we've got hydro.
Karen: Earlier lines of therapy.
Karen: Perhaps is 55% of patients coming to faith got directly from <unk>.
Only expect this trajectory to continue.
Karen: Although they've got still comprises the majority of prescriptions, we are seeing more traction with actually less likely supported by access dynamics favorable pay a policy that mirror of data and a dedicated J code in place where.
Karen Massey: We are committed to innovating on the patient experience even further by advancing the development of our pre-filled syringe, or PFS, this year. The PFS will allow us to introduce Vyvgart to a new patient population with an increasingly easy-to-use interface. It is our goal to make a pre-filled syringe available for both MG and CIDC, and importantly, we believe this will move us one step closer to the possibility of self-administration in the U.S. Slide 12. The momentum outside the U.S. has been strong, with multiple launches and approvals already underway for 2024. The majority of our current sales are still in the US, but over time, we expect global markets to make increasingly larger contributions to total revenue, especially as the speed at which we bring Vyvgart into new territories increases.
Karen: We are committed to innovating on the patient experienced even further by vacuum the development of our Prefilled syringe or P. S. S. S. Yeah.
Karen: This will allow us to introduce they've got two new patient population will increasingly easy to use interface.
Karen: It is <unk> to make a prefilled syringe available supplies M. G. NCIC and importantly, we believe they told me that one step closer to the possibility of self administration in the U S.
Karen: Slide 12.
Karen: The momentum X U S as being strong with multiple launch it and approve of already underway for 2024. The majority of the current sales are still in the U S. But over time, we expect global market to make increasingly Roger contributions to total revenue.
Karen: Especially at the speed at which brings it got into new territory is picking up.
Karen Massey: I'm very proud of the team in Europe who've been working hard to secure reimbursement at a record pace in Germany, Italy, Spain, and recently Belgium, with patients receiving access to Vyvgart in half the average time historically needed by orphan drugs. We're also seeing incredible uptake in China through our partnership with Xilab, driven by our recent inclusion on the NRDL. We are still at the front end of reaching patients who could benefit from Vyvgart, and we remain committed to delivering on our promise of reaching the broadest set of MG patients possible. We also know that the MG opportunity continues to expand with competition and innovation driving growth of the overall market. With a robust knowledge of SCRN and one of the most expansive sets of clinical and real-world data generated to date, we are in a position of strength to continue to lead this market. Slide 13.
Karen: I'm very proud of the team in Europe has been working hard to secure reimbursement at a record pace in Germany, Italy.
Karen: Pain, and recently, Belgium with patients receiving access to gym got in half the average time historically needed by often drug.
Karen: We're also seeing incredible uptake in China throughout partnership is dialogue driven by our recent inclusion on the <unk>.
Karen: We are still at the front end of reaching patients who could benefit from that and we remain committed to deliver on our promise of reaching the broadest set a M G patients possible we.
Karen: We also know that the empty opportunity continues to expand with competition and innovation driving growth of the overall market.
Karen: With a robust knowledge of <unk> and one of the most expansive set of clinical in real world data generated to date.
Karen: Initiative strength to continue to lead this market.
Karen: Slide 13th.
Karen Massey: We're excited by the opportunity to expand our patient impact beyond MG this year with two upcoming regulatory decisions in ITP and CIDP. Today, we're going to focus on the CIDP opportunity. CIDP is a debilitating disease and one where patients continue to face significant burdens both from the symptoms of the disease but also from the demands of the available treatment. Our strategy will be to leverage the learnings and infrastructure we built with MG and apply them to the unique dynamics of the CADP market to best position ourselves for success. The key learnings and overlapping strategies between MG and CIDP give us more confidence in the long-term potential of Vyvgart Hytrullo as a transformational treatment for CIDP, but we also recognize there will be some unique challenges that may impact Laura's trajectory. Good
Karen: We're excited by the opportunity to expand our patient impact beyond M. G. D. C M with two upcoming regulatory decisions and I T. T N T I D C.
Karen: Today, we're going to focus on the CIP opportunity.
Karen: He already paid the debilitating disease and one more patients continue to say significant payton both from the symptoms of the disease, but also the demands of the available treatments.
Karen: Al strategy will be the leveraged the learning infrastructure, we dealt with M G and apply them to the unique dynamics of the C. I D P market.
Karen: Physicians ourselves to success.
Karen: The key lengths and overlapping strategies between M. G. M. C. I D. P gives us more confidence in the long term potential of <unk> and the transformational treatment and C. I D P.
Karen: But we also recognize there'll be some unique challenges that may impact launch trajectory.
Karen: First.
Karen Massey: As is standard with most launches, we will need to wait approximately two quarters for payer policies to come into place. As is standard with most launches, we will need to wait approximately two quarters for payer policies to come into place. Second, IBIG's well-entrenched and unlabeled for CIDP patients. CIDP is a progressive disorder with many patients fearful of symptom regression who may not want to change from their existing therapy.
Karen: Standard with most launches we will need to wait approximately two four is to pay a policies to come into place.
Karen: Second.
Karen: G well entrenched in on label Ccitt patients.
Karen: <unk> is a progressive disorder with many patients fearful of symptom aggression, we may not want to change from their existing therapy.
Karen Massey: Having said that, we're very motivated by the strength of our data to bring a new treatment option to the CIDP community, which would be the first real innovation in decades, and we'll be prepared with thoughtful strategies at the time of the FDA's decision on our submission. Slide 14.
Karen: Having said that with very motivated by the strength of our D data to bring a new treatment option to the CIP community, which would be the first real innovation in decades, and we'll be prepared with thoughtful strategies at the time of the F D as decision on our submission.
Karen: 14.
Karen Massey: Before I turn the call back to Tim, I want to talk about the commercial engine we're building as we think ahead about how we can reliably and repeatedly maximize the value we offer to autoimmune patients. We continue to learn about the unique challenges and resulting gaps in treatment that patients suffer from autoimmune diseases. Most notably, there's an overall lack of innovation. All the entrenched therapies are considered sufficient.
Before I turn the call back to Tim.
Tim: Talk about the commercial engine, we're building as we think ahead and how we can reliably and repeatedly maximize the value we offer to autoimmune patients.
Tim: We continue to learn about limit challenges and resulting gaps in treatment that patients suffer with auto immune diseases.
Tim: Notably.
Tim: Overall lack of innovation.
Tim: All the trash therapies are considered sufficient.
Karen Massey: Patients take a long journey to get to diagnosis. [inaudible] Finally, there are high barriers to access even when innovative treatments become available. At argenx, we're making a long-term commitment to these communities that we hope extends well beyond the treatments we can deliver. Some of the areas of focus for us include generating awareness of the disease and the challenges that patients and their supporters face, whether through online communities, our DTC campaigns, or our advocacy efforts. We want to raise the bar on treatment expectations.
Tim: Patients take a long journey to get a diagnosis.
Tim: And often that diagnosis does not result in clear answers.
Tim: Finally, there are high barriers to access even when innovative treatments become available.
Tim: At <unk>, we're making a long term commitment to these communities that we hope extends well beyond the treatments we can deliver.
Tim: Some of the areas of focus for us to include generating awareness of the disease and the challenges that patients and if you'll pull his face whether through online communities rbbc campaigns or our advocacy efforts.
Tim: You want to raise the bar on treatment expectations adult is not just to replace old treatment, but to reset expectations. We wanted to move the Gulf instead of patients aim to regain function or going back to things they enjoyed before their diagnosis.
Karen Massey: Our goal is not just to replace old treatments but to reset expectations. We want to move the goalposts so that patients aim to regain function or go back to things they enjoyed before their diagnosis. We want to drive innovation beyond the molecule and into the patient experience in the form of new product presentations and a best-in-class support system. And we're delivering on our commitment by providing broad and simple access to patients. These are the types of investments we want to make as we grow and expand into new patient populations because we're in the business of transformation and advancing beyond incremental change. We're making a long-term commitment to deliver repeatable, sustainable, comprehensive value to patients, their care teams, and to the broader autoimmune community. And with that, I'll turn the call back to Tim.
Tim: We want to drive innovation beyond the molecule and on the patient experience in the form of new product presentation, and a best in class support system.
Tim: And we're delivering on our commitment by providing broad and simple access to patients.
Tim: These are the types of investments, we want to make as we grow and expand into new patient populations, because we're in the business of transformation and advancing beyond incremental change will.
Tim: We're making a long term commitment to deliver repeatable sustainable comprehensive value to patients.
Tim: Their teams and to the product auto immune community.
Speaker Change: And with that I'll turn the call back at him.
Tim Van Hauwermeiren: Thank you, Karen. We have laid out an ambitious plan for the year, and at two months in, we are already in a great position to deliver on our goals. We are energized by the incredible cadence of opportunities that remain ahead and will continue to pursue the ArgenX way with relentless execution. Before we turn the call over to Q&A, I want to take a moment to thank the individuals who drive our success. We would not be where we are today without the tireless efforts of the Argenx team.
Speaker Change: Thank you <unk>.
Speaker Change: We laid out an ambitious plan for the years and at two months in we are already in a great position to deliver on our goals.
We are energized by the incredible cadence of opportunities that we may not have and will continue to approach. This clinics weights with three lines of execution.
Speaker Change: Before we turn the call over to <unk>.
Speaker Change: I want to take a moment with the individuals who drive our success.
Speaker Change: You would not be where we are today without his tireless efforts of the <unk>.
Operator: I also want to thank all the patient communities and physicians who inspire our efforts to bring better outcomes to those suffering from autoimmune diseases. Thank you, and we look forward to your questions. At this time, I would like to remind everyone that, in order to ask a question, press the star, then the number one on your telephone keypad.
Speaker Change: I also want to find all the patients communities and physicians, who inspired our efforts to bring better outcomes to those suffering from auto immune diseases.
Thank you and we look forward to your questions.
Speaker Change: At this time I would like to remind everyone in order to ask a question Press Star then the number one on your telephone keypad. We ask you. Please limit yourself to one question and then rejoined the queue for further questions.
Tazeen Ahmad: We ask that you please limit yourself to one question and then rejoin the queue for further questions. Your first question comes from the line of Tazeen Ahmad from Bank of America. Your line is open. Hi, good morning.
<unk>: Your first question comes from the line of <unk> from Bank of America. Your line is open.
<unk>: Hi, Good morning, Thanks for taking my questions. Just a couple of based on your prepared remarks regarding the C. I D. P lines, if we assume that it launches I sang in early July you've talked about taking a couple of quarters to really get some insurance.
Karen Massey: Just a couple based on your prepared remarks. Regarding the CIDP launch, if we assume that it launches, let's say, in early July, you've talked about taking a couple of quarters to really get full insurance reimbursement. So, just directionally, how should we be thinking about any type of contribution from CIDP sales in calendar year 24? And then I have a follow-up. Thanks for the question, Tazeen.
Speaker Change: Insurance reimbursement sounds just directionally, how should we be thinking about any type of contribution from from Cit's P sales and calendar and you're at 24, and then I have a follow up.
Speaker Change: Thanks, Thanks for the question Davina, it's great to hear from you. So maybe I'll provide a few thoughts on CIP launch and what we're learning in college, you Wanna make any comments from the financial perspective. So we're enlarge preparations as you mentioned 50 AGP in the mall that we'd learn through market research I'd say the more comfort.
Karen Massey: So maybe I'll provide a few thoughts on the CIDP launch and what we're learning. And Karl, do you want to make any comments from the financial perspective? So we're in launch preparations, as you mentioned, for CIDP, and the more that we learn through market research, I'd say the more confidence we have in the value that we're going to bring to patients in this market. It's a debilitating disease, and there certainly is a really very high unmet need in this patient population. So we're very confident.
Speaker Change: Since we have in the value that we're going to bring to patients in this market. It's a debilitating disease and they suddenly is a really a very high unmet need in this patient population. So we're very confident as you said as we sharing the prepared remarks.
Karl Gubitz: As you said, and as we shared in the prepared remarks, there are a few things that will impact uptake this year. There are payer policies getting in place, and the fact that this is a debilitating disease, a progressive disease. So there's some what we call stickiness, likely, of the patient remembering that IVIG is on label for those. So we're not expecting a rapid uptake in the latter part of the year. But Karl, do you want to comment on anything further than that?
Speaker Change: Things that will impact the uptake this year, there's the pay of policies getting in place and the fact that this is a debilitating disease a progressive disease. So based on what we call stickiness likely of the patients remembering the I V. I G is on label.
So that we could so we're not expecting a rapid uptake in the latter part of the year because you want to comment on any other than that no I think you've said it on I think the.
Karl Gubitz: No, I think you've said it all. I think the payer contracts, which will take a quarter or two to put in place, will definitely impact the uptake of CIDP and delay some of those revenues into 2020. Okay, thank you. And then also, a comment from your press release about the price decrease in Germany. I know historically in Europe, there's just naturally price deterioration. But are you calling out this particular decrease because it's a bigger price decrease than you would normally expect? Color.
Speaker Change: By your contracts, which will take a quarter to to put in place will Dave.
Speaker Change: Currently.
Speaker Change: <unk> some of those revenues into 2025.
Speaker Change: Okay. Thank you and then also uhm.
Speaker Change: From your press release about.
Speaker Change: And in Germany, I know historically in Europe, there is just naturally price deterioration.
Speaker Change: Are you coming out this particular decrease because it's a bigger price decrease in than you would normally expected just to give a little bit of color.
Karl Gubitz: Yeah, so thank you. We had a really successful launch in Germany, and we, of course, had a really good price in Germany.
Yeah, Sir Thank you we have a really successful launching in Germany, and we of course after a really good price in Germany.
Karl Gubitz: That actually meant that we exceeded the threshold for the classification of an orphan drug, which automatically triggers a price renegotiation with the German authorities. We have now entered into that negotiation, and we have to assume a lower price, but we don't know what that price will be.
Speaker Change: Actually meant but we exceeded the threshold for the classification of an orphan drug which automatically triggered a price negotiation with a gentleman authorities. We have now entered into that.
Speaker Change: Negotiation and we have to assume a lower price we don't know what that prize we'll be renegotiating. It we will only know by <unk> is the beginning of 2025 Q1 2025.
Karl Gubitz: We're negotiating it. We will only know by the beginning of 2025, Q1 2025. But we will be paying for that lower price from 1 January 2024, as the difference between the old and new price will have to be a rebate back to back to the government.
Speaker Change: We will be accruing food at a lower price from one January 2024.
Speaker Change: Difference between all the new price will have to be a rebate back to back.
Karl Gubitz: So, Tazeen, I mean, this is because we were so successful and exceeded that threshold of 30 million dollars, which is awesome. Okay, thank you. Your next question comes from the line of Derek Archila from Wells Fargo. Your line is open. Hey, good morning, and thanks for taking the questions. Yeah, just two from us.
Back to the government.
Speaker Change: My address is <unk>.
Speaker Change: Cause we were successful and exceeding that threshold of $30 million.
Speaker Change: Which is often drug designation.
Speaker Change: Okay. Thank you. Thank you for your question.
Speaker Change: Your next question comes from the line.
Wells Fargo: Wells Fargo. Your line is open.
Wells Fargo: Hey, good morning, and thanks for taking my questions. Yeah, just cheaper so I just wanted to understand the per cent of the 2300 prescribers for M. G that currently treat C. I D. P. C. I D. P patients and then also I guess you you had a comment in the prepared remarks around new patients that you'll be able to access with the prebuild. So room. So I just.
Karen Massey: So I just wanted to understand the percent of the 2300 prescribers for MG that currently treat CIDP patients. And then also, I guess you had a comment in the prepared remarks around new patients that you'll be able to access with the prefilled syringe. So I just want to understand, you know, who those patients are specifically that you can't really access right now with IV and HITRULO. Thanks. Thanks for the questions. I'll take them one by one.
Wells Fargo: Understand you know who those patients are specifically that you can't really access right now with Ivy in high School Oh. Thanks.
Speaker Change: Thanks, Thanks for the question.
Speaker Change: I'll take them one by one so what we're learning as we look into the CIBC market is that there is significant overlap between the prescribers as you said the 2300 prescribers prescribing they've got for M. G and potential future prescribed this to see IDP kitt's treated much more in the community.
Karen Massey: So, what we're learning as we look into the CIDP market is that there is significant overlap between the prescribers, as you said, the 2300 prescribers that are prescribing Vyvgart for MG and potential future prescribers for CIDP. CIDP is treated much more in the community, is what we're learning. And as you well know, one of our strategies for MG, and one of the things that we're seeing is that out in the community, we're seeing a lot more confidence in using Vyvgart for MG based on real world efficacy. We're seeing real world efficacy that reflects the clinical trial efficacy, and the safety that continues to hold up. We now have over 4000 years of patient safety data now.
Speaker Change: Is is what we're learning and as you well know one of our strategies for M. G. In one of the things that we're seeing is that out in the community. We're seeing a lot more confidence with using faith God for M. G based on the real world efficacy, we're seeing real world efficacy.
That that reflects the clinical trial advocacy uhm the safety that continues to help us we have a 4000.
Speaker Change: Patient safety data now.
Karen Massey: So, we'll see, so there will be, there is significant overlap in those in that prescriber base. In terms of the new patients that you asked about with the prefilled syringe, I would say this just continues the momentum towards our strategy of moving earlier into the treatment line. So, in MG, we stated that the goal is that we believe MG should be used after oral. We're seeing 55% of our patients coming directly from oral.
Speaker Change: So there'll be seek there is significant overlap in those in that prescribed to base in terms of the new patients that you asked about but with a prefilled syringe I would say this just continues the momentum toward a strategy of moving earlier into the treatments line. So in M. G, which stated that the goal is that we believe M. G should be used.
Speaker Change: Uhm after orals within 55% of our patients coming directly from auto the I V. Having the subcutaneous actually low option and then moving into the Prefilled syringe will just allow us to continue to execute on that strategy.
Karen Massey: The IV, having the subcutaneous, like, true option and then moving into the prefilled syringe will just allow us to continue to execute on that strategy. Thank you. Your next question comes from a line called Rajan Sharma from Goldman Sachs. Your line is open.
Speaker Change: Thanks for the question.
Speaker Change: Your next question comes from the line.
Unknown Executive: Goldman Sachs. Your line is open.
Tim Van Hauwermeiren: Hi, thanks for taking my question. I'm just looking ahead to thyroid eye disease where you're initiating phase three, and I realize that we'll, [inaudible] Relative to incumbents in that market and maybe related to that. Could you just comment on what underpins your confidence?
Unknown Executive: Hi, Thanks for taking my question I'm, just looking ahead see thyroid disease or your initiation phase three and I realize that will probably see the clinical trials uhm entry relatively soon but could you perhaps just talks about how you were thinking about positioning.
Unknown Executive: Relative to incumbents and not market.
Unknown Executive: May be related to that could you just comment on what underpins your confidence in moving to phase III, we've obviously seen some compassion.
Tim Van Hauwermeiren: Phase 3. Concept Data. But I was just wondering if there's anything beyond that that is driving the decision. No, thank you.
Unknown Executive: Concept Theresa I was just wondering if there's anything beyond that is driving the decision here. Thank you.
Speaker Change: No. Thanks for so I would take discretion the conviction in theory of course is driven by biology.
Tim Van Hauwermeiren: So I will take discretion. The conviction in TED, of course, is driven by biology that's done based on the internal homework but also peer-reviewed data from another player in the SJN class. There are now nine indications out of nine for the class. You know, we have seen successful groups of concept theories, one of them. So we're basing, we're marching based on that combined prediction and differentiation, which we hope, will come from different, most likely. So we are convinced that TED is an IGT or 20-body driven disease. We think that by lowering these O2 antibodies, we could have the most profound effect on the disease. But we can also, of course, leverage a very clean safety profile and a very competitive way of presenting the product. So these are the three pillars to try and be competitive in the TED markets. Thank you for the question. Your next question comes from a line from Yatin Suneja from Guggenheim. Your line is open. Thank you for taking the question. I have a question on Sjogren disease.
Just on the internal homework, but also be reviewed data from another player of the SCN class.
Speaker Change:
Speaker Change: The amount of nine indications out of nine for the class and who would we have seen successful group of <unk> is one of them.
Speaker Change: <unk> based on the combined conviction and differentiation, we hope will come from this.
Speaker Change: <unk> most elections.
Speaker Change: We are convinced C D as in Archie authenticated vivid disease everything is by lowering is also eligible wish we could have the most profound effects on the disease.
Speaker Change: So of course can leave a very cheap safety profile and a very competitive way of presenting the product. So these are the three service to try and be competitive with the CD markets. Thank you for the question.
Speaker Change: Your next question comes from the line of <unk> from Guggenheim.
Guggenheim: Thank you for taking question I have a question on the shopping and disease could you just talk about the disclosure that you expect to Meg and given the compulsive nature of Crestwood you decide to move forward. Even if you don't see a strong signal ordered for you and if you just see a signal on other domains.
Tim Van Hauwermeiren: Could you just talk about the disclosure that you expect to make? And given the composition nature of the press, will you decide to move forward even if you don't see a strong signal or even if you just see a signal on other domains? And then, you know, maybe along the same line, like from a regulatory standpoint, is there a well-defined regulatory path here, or is there a main point of choice from a registration point of view? Thank you. Yeah, Yatin, this is a great question.
Guggenheim: And then you know maybe along the same line from a regulatory standpoint.
Guggenheim: Is there a valid define regulatory parterre or visited the main point of choice from from registration standpoint. Thank you.
Tim Van Hauwermeiren: Thank you for asking that question. And I want to call out that Children's is a signal finding study where we will look at the totality of data across multiple clinical endpoints, not just CREST but also SDI, actually TIN-SDI and S3, and then a string of biomarkers, which we're carefully monitoring. And what we want to see is consistency between the clinical endpoints and the biomarkers in order to get from addiction and march forward. And from a regulatory point of view or an endpoint point of view, we will need to have, of course, an interaction with the regulators.
Speaker Change: And this is a great question. Thank you for asking it's and I want to call out the children's is signal finding study, where we will look at the totality of data across multiple clinical endpoints, let's just Chris but also has died actually clean aside and a street.
Speaker Change: Uhm, the string of Biomarkers, which we have carefully monitoring and what we want to see consistency between the clinical endpoints and the biomarkers in order to get a conviction and March forward.
Speaker Change: And from a residency at point of view or endpoint point of view, we will need to have a causal interaction with regulators, but mind you that order number of these three registrational trials running will actually department and for this is die. So we assume this is the <unk> I would like to see <unk>.
Tim Van Hauwermeiren: But mind you, that there are a number of phase three registration trials running where the primary endpoint is SDI. So we assume this is the endpoint the FDA would like to see. And CRED is relatively new.
Speaker Change: <unk>.
Tim Van Hauwermeiren: So stay tuned to know if and when we have positive signal data, and we would entertain a conversation with the FDA on this topic and afterwards communicate. Thanks for the question. Your next question comes from the line of Yaron Werber from TD Kallen. Your line is open.
Speaker Change: <unk> you know if and when we would have positive signal data.
Speaker Change: We will entertain a conversation with the F D. A on this topic and accurate communicate thanks.
Speaker Change: Thanks for the question.
Speaker Change: Your next question comes from the line of urine Weber from T. D. Cowan Your line is open.
Tim Van Hauwermeiren: Right. Tim, maybe just a follow-on question on Yatin's question also about Sjogren's. So your study is 36 patients, J and J were in three arms, you have two, and they obviously had 163 patients. So they obviously had a much bigger study, and it sounds like it was that big, as you mentioned. The endpoint's a little different, but you have a composite that includes their endpoint. So I guess my question is whether it's a pretty good trial design. And so give us a little bit of a sense: is it completely underpowered to show any statistics at all? And is there a certain threshold of an effect that you want to see that you consider to be positive to move forward?
Urine Weber: Maybe just a follow on your.
Urine Weber: Question also on short runs so you're studying.
Urine Weber: 36 patients J&J run three arms, you have to and they obviously have 163 patients. So they obviously had a much bigger studies and it sounds like he was the best tickets as you mentioned.
Urine Weber: It's a little different but you have a composite that includes <unk>. So I guess my my question is.
Urine Weber: Pretty good trial design, and so give us a little bit of a sense is it completely underpowered to show in your statistics at all and.
Urine Weber: Is there a certain threshold over the fact that you wanted to see that you consider it to be positive to move forward. Thank you.
Tim Van Hauwermeiren: Yeah, I want to call out that there's an overlap between our endpoints and J&J endpoints. The reason, of course, they're doing three arms is because they still need to sort out a dose, which is not something that we have to do. You know that you can dose safeguard at full power without paying a safety penalty, and we will look at consistency of data.
Speaker Change: Yeah, I want to call. This an overlap between our I phones and the <unk>. The reason of course that with the arms, because they're still need to sort out the dose which is not something which we have to do you know that you can go stiff got at full power.
Our famous safety penalties.
Speaker Change: And we will be able to look at the consistency of data.
Tim Van Hauwermeiren: So it is defined what a clinically meaningful improvement is on an SDI score, on a CREST scale, on an SP scale, and then, of course, we want to see the biomarkers. Moving in the right direction The biomarkers are a combination of biomarkers from the circulation but also from the biopsy, and it is important to see the need to move on a clinic land point that we're going to pay special attention to what's happening in the biomarkers section, what's happening in the biopsy, and are these moving in a consistent fashion. So conviction on biology. We have also stratified for high and low IgG levels; about 50% of Sjogren's patients have high IgG types, you know, 16 grams per liter or more. I think that's a very interesting stratification factor.
Speaker Change: So it is defined what are clinically meaningful improvement is advice for overdraft scale.
Speaker Change: <unk> and then of course, we want to see the Biomarkers.
Speaker Change: In the right direction, the biomarkers or a combination of biomicroscope circulation, but also from the biopsy.
Speaker Change: And it is important to see the <unk> the <unk>.
Speaker Change: You're going to pay special attention to what's happening with the bio.
Speaker Change: Marcus.
Speaker Change: Section.
Speaker Change: Turning into biopsy.
Speaker Change: And are these moving in a consistent fashion. So conviction on apologies, we have also sacrifice for high and low oxygen levels about 50% of <unk> spacious.
Speaker Change: <unk> 16 grasp or more.
Speaker Change: That's a very interesting certification, Texas.
Tim Van Hauwermeiren: The other way we look at the data is looking for patients, you know, which are positive for the raw antibody or not. So I think it's going to be a small sample with a very deep look into patients and a very rich look. So way beyond a P-value on an antibody. Thanks for the questions. Your next question comes from a line from Allison Bratzel from Piper Sandler. Your line is open.
Speaker Change: We looked at the data is looking for patients.
Speaker Change: Offices for the row antibody or not so I think it's gonna be a small sample to the very difficult to patients in a very rich so raytheon, but the value of <unk>.
Speaker Change: Thanks for the question.
Speaker Change: Your next question comes from the line of Alison bread, Sir from Piper Sandler Your line is open.
Karen Massey: Hey, good morning, and thanks for taking my question. So just to follow up on some of the discussion on the pre-filled syringe, could you just expand on the expected cadence of updates on that this year? And just what gives you confidence in potential approval of the pre-filled syringe in 2024? And then, more broadly, given competitive updates in this field, how important do you see self-administration via syringe or auto-injector in determining your competitive positioning in a market with multiple FCRN agents approved? Thanks.
Allison Marie Bratzel: A good morning, and thanks for taking my question.
Allison Marie Bratzel: So just to follow up on some of the discussion on a prefilled syringe could you just expand any expected <unk>.
Allison Marie Bratzel: H and that this year and just what gives you confidence in potential approval.
Speaker Change: <unk> and 20th 24.
Allison Marie Bratzel: And then just smart properly you know given competitive updates in the field you know how important <unk> administration.
Allison Marie Bratzel: Diaz.
Allison Marie Bratzel: And your auto injector in determining you are competitive.
Tim Van Hauwermeiren: Thank you, Allison, and thank you for the questions. So I'll take the first one, and then I will hand over to Karen to explain how we seek to continue to lead the space, also from a patient convenience point of view. But first, at Pre-Filled Syringe, it is high on the strategic agenda of the company this year. You know that we decided to launch the first generation Sub-Q as fast as we could, with the patient in mind. This is our second generation of the Pre-Filled Syringe, and there are two important data sets we need to collect in order to be ready to submit the dossier with the FDA. The first data set is a bioequivalent study. The second data set involves a human factor study, and we are on track to give you an update during the first half of this year in terms of the progress we're making with collecting these data points, which it is of high priority for the company to be in a position to submit this year with the, and maybe Karen, you can continue on part two of the question, please. Yeah, absolutely.
Allison Marie Bratzel: Positioning in the market with multiple Sharon agents approved.
Speaker Change: Thanks Medicine, and thank you for the questions. So I'll take the first one the other hand over to pattern.
Allison Marie Bratzel: They have.
Pattern: To continue to.
Pattern: Most of the patients. It contains point of view, but first episode Syringe is is high on the strategic agenda of the company to see if.
Pattern: You know that we decided to launch the first generation Q as fast as we choose with the patient in mind.
Pattern: This is our second generation of please.
Pattern: And two important data first we need to collect in order to be ready to submit.
Pattern: David the FDA, but first data set is a viable driven study <unk>.
Pattern: Second datasets evolves you can protect the study.
Speaker Change: <unk> to give you an update during the first half of disease in terms of the progress, we're making with collecting these data homes, but it is a high priority for the company to be in a position to submit this year with the F. D. A and maybe we can we can continue on October question. Please yeah, absolutely I'm I'm really excited.
Karen Massey: I'm really excited about the prefilled syringe and the potential to bring that to patients. But we talked earlier about there being a significant unmet need in this market, and we can see that patients are looking for innovation. I think our revenue last year demonstrates that in just our second year of launch. MG patients want to get their lives back. And PFS and self-administration help them to do that.
Speaker Change: The prefilled syringe and the potential to bring that to patient, but we talked earlier about there is a significant unmet need in this market and we can see that patients are looking for innovation and I think as a revenue loveseat demonstrates that in just a second year of launch.
Speaker Change: M G patients want to get their lives back N T. F. S. Self administration help them to do that so I think that's that's the real reason that the innovation Uhm a C. F S. As important and then I think for Us <unk>.
Karen Massey: So I think that's the real reason that the innovation of PFS is important. And then, I think for us, from a Vyvgart perspective, it just reinforces our leadership in the market. So first in class, FCRN, I talked earlier about how we're demonstrating real-world efficacy, continued safety, and then this just brings another product presentation potentially to the market. So we're confident that we're bringing the innovation to the market that the patients really need. And your next question comes from the line of Vikram Purohit from Morgan Stanley. Your line is open. Hi, good morning. Thank you for taking our questions. We had two.
Speaker Change: The magenta perspective, it just reinforces our leadership in the market, but first thing <unk>.
Speaker Change: Talked earlier about how we're demonstrating that real world efficacy continued safety and then it just brings another product presentation potentially to the market. So the way we're confident that we're bringing the innovation to the market the patients really need.
Speaker Change: Your next question comes from the line of <unk> from Morgan Stanley. Your line is open.
Morgan Stanley: Hi, Good morning. Thank you for taking my questions. We have to first on <unk>, which is curious for the decision you're contemplating internally what are some of the key considerations that play right now and what do you see as the potential scenarios that could result from the update you're planning on providing goes later this year and then secondly.
Vikram Purohit: First, on bolus pemphigoid. We're just curious, for the decision you're contemplating internally, what are some of the key considerations at play right now? And what do you see as the potential scenarios that could result from the update you're planning on providing us later this year? And then, secondly, based on market research you might have done for MMN, I was curious to see how you segment this patient population and which segment of the patient base you think fCortegiMod could be for? I'm sorry, but Empathy Prubart could be a viable therapy for.
Morgan Stanley: <unk> based on market research you might've done for them and I was curious to see how your segment. This patient population and Uhm, which segment of the patient base, you think <unk> could be I'm, sorry, uhm, and Patsy poop or it could be available therapy for thanks.
Tim Van Hauwermeiren: Thanks. Yeah, thanks Vikram, and I'm going to take question two on our annotations. For Bruce Pantygoi, I think the data analysis is ongoing, as we said, and the key consideration is going to be what is the impact of the background medication on the disease-causing antibodies, and is there a clinical trial design conceivable where actually FGAP-T-MOC can still shine because it is knocking down other antibodies like Health, but are you still able to show a delta of the background medication? And there is a thread and So I think we're well advanced with the homework. We will do more consultation with our experts during AAE in the coming weeks. And I think we will be ready to think through a couple of scenarios based on that feedback. So stay tuned.
Speaker Change: Yeah. Thanks become gentlemen, this question too.
Speaker Change: <unk> <unk> I think the data analysis is ongoing as we said and the key consideration is going to be.
Speaker Change: What is the impact of the background medication of the disease, causing all currency bodies and visa clinical trial design conceivable, we're actually I've got for you must contribute shine because this smoking ban auto antibodies like Hell, but are you still able to show a delta over the Vega medications and there is a thread a needle between what.
Speaker Change: He wants to achieve as a company and what regulators is actually you're about to see is minimum data set up. So I think you develop events with the homework, we will do against Conservancy without <unk> in the coming weeks and I think we will be ready to take you through a couple of scenarios based on that feedback so.
Tim Van Hauwermeiren: This is an important discussion internally for the company, and we believe that the unmet need in BP warrants very thoughtful consideration. Thank you.
Speaker Change: This is an important discussion internally for the company.
Speaker Change: We believe that <unk> that is possible consideration.
Speaker Change: Thank you.
Karen Massey: Yeah, and on the topic of MMN, MMN is the type of disease that we think that we like at argenx because it's characterized by the fact that there is an enormous unmet need in these patients, there's been limited innovation, and these patients continue to progress despite treatment today. So of all of the diseases that we're studying, we get the most calls from MMN patients that are asking for innovation to come to the market. So these are the types of diseases that we like, because we think we can really raise expectations and reframe what patients can expect, and we think we'll do that with the impact of PrUVAT. So in terms of what the market research is telling us, beyond that, we're not breaking down our clinical trial design as yet, but beyond that, we are rather just thinking about what the broadest patient benefits that we can create. And remember that in MMN, at least 85% of the patients share that pathogenic autoantibody, that IgM autoantibody against GM1. We actually think that 100% of the MMN patients have that autoantibody, the disease-causing antibody, the sensitivity detection issue. So we think that the underlying biology is pretty much unifying all MMN patients. So I think we're in a good place.
Speaker Change: Yeah, and and on the topic of <unk>.
<unk> is the type of disease I think that we said that we like <unk> because it's characterized by the fact that is it.
Speaker Change: <unk> unmet need in these patients.
Speaker Change: Limited innovation and these patients continue to progress despite.
Speaker Change: Despite treatment today, so they they they are.
Speaker Change: Of all of the diseases that we're studying we get the most calls from any medications that.
Speaker Change: That are asking for innovation to come to the to the market. So.
Speaker Change: So these are the <unk>.
Speaker Change: The data that we liked because we think we can really raise expectations and re frame.
Speaker Change: What patients can expect and we think will do that we didn't have to prove that in terms of what the market research is telling us uhm beyond that.
Speaker Change: We're not breaking down a clinical trial designers, yet, but beyond that but rather just thinking about what's the broadest patient benefits that we can create a victim remember that and then at least 85% of the patients sure. That's pathogenic ultra M. T. R. G M alternative it against a G. M. One we actually think that <unk>.
Speaker Change: And the medications have that all set for your body to disease, causing antibodies of sensitivity detection issue. So we think that the underlying biology is pretty much unifying for all elementary so I think that in a good position.
Karen Massey: Thanks for the questions. Your next question comes from a line of Joel Beatty from Baird. Your line is open.
Speaker Change: Thanks for the question.
Speaker Change: Your next question comes from the line of Joel BT from Bird Your line is open.
Karen Massey: Hi, thanks for that question. Are you seeing or anticipating any meaningful differences in the frequency of dosing between IV and subq and then soon prefilled syringe and eventually the auto injector? Thanks for the question. No, we're not seeing any differences between the formulations.
Joel Lawrence Beatty: Hi, Thanks for the question are you seeing or anticipating any meaningful differences in the frequency of dosing between Ivy and so too and then soon prefilled syringe and eventually auto injector.
Joel Lawrence Beatty: Thanks for the question no we're not seeing any differences between the formulations, it's really about product presentation for patients convenience.
Karen Massey: It's really about product presentation for patient convenience. Your next question comes from a line called Danielle Brill from Raymond James. Your line is open. Hey guys, this is Alex on behalf of Danielle.
Joel Lawrence Beatty: Your next question comes from the liner Danielle Bill from Raymond James Your line is open.
Hey, guys. This is Alexandra Danielle Uhm, if I recall correctly it looks like your updated prevalence estimates for C. I D P, which are higher than your previous projections. Just curious what factors you saw that are attributed to the Tam growth.
Karen Massey: If I recall correctly, it looks like you updated prevalence estimates for CIDP, which are higher than your previous projections. I'm just curious what factors you saw that are attributed to the TAM growth, pertains to diagnosis, rates, treatment rates, and potentially, Thanks so much. Yeah, I wouldn't read too much into it.
Speaker Change: Pertains to diagnosis rates.
Alexander Thompson: And potentially life expectancy. Thanks, so much.
Speaker Change: Yeah, I wouldn't read too much into it I think the the C V V shaped a number from official sources specifically for your visit you five will not give you the accurate number what we think is accurate for the key.
Karl Gubitz: I think previously, we shared a number from official sources, specifically for EURES and EU5. We have now given you the exact number, which we think is accurate for the key markets in which we play. But our view on prevalence actually has not really changed. We try to feed you as much accurate information as we can, representing the markets in which we operate. Okay, thank you. Your next question comes from the line of Simon Baker from Redburn, Atlanta. Your line is open.
Speaker Change: Markets in which we play, but obviously you on prevalence actually has not really changed we tried to speed with as much accurate information as we can representing the market's industry place.
Speaker Change: Okay. Thank you.
Speaker Change: Your next question comes from the line of Simon.
Speaker Change: Your.
Simon: Of your line is open.
Tim Van Hauwermeiren: Thank you for taking my question. If I can go back to Sjogren's, please. I'm not sure if I missed the answer, but I just wanted to check if you did say what the form of the disclosure of that phase two data would be. I'm guessing it would be an announcement that the study has worked and you will proceed, followed by disclosure at a clinical conference. But I just wonder if you could confirm that and suggest a potential conference where that would be disclosed. And, just putting the cart before the horse a little and thinking about phase three design, Tim, I think you mentioned the likely regulatory end point is changes in the clinical SDI score. It also looks like 48 weeks duration looks like a typical length for a phase three study in this indication. And from other studies, it looks like three to 400 patients is a typical size. Can you comment on the legitimacy of those assumptions? Thanks so much.
Simon: Thank you for taking my question if I can go back to to show Grins. Please I'm not sure if I'm <unk> I'm I just wanted to check if you did say what the form is the disclosure of.
Simon: Of that phase two data would say are I'm guessing it would be an announcement that the study is where it can you.
Simon: I will proceed followed by disclosure that clinical conference if I just wonder if you could confirm that and suggest.
Simon: Potential conflicts where that would be disclosed.
Simon: Putting the cart before the whole circle and thinking about faith Street.
Simon: You mentioned that the the the likely regular Tran <unk> school it looks it looks like for <unk>. It looks it looks like a typical link for phase three study any indication of.
Simon: This study is it looks like for each 400 patients is a typical sorry can you come in on the legitimacy if those assumptions. Thanks so much.
Tim Van Hauwermeiren: Yeah, thanks for both questions. So yes, we're looking for the signal. And of course, when we see it, we will, we will share it with some level of detail surrounding the go decision or the no-go decision. And assuming a go decision, you're correcting your understanding, that then we would show more detailed data at the neutral medical mean so that that understanding is correct.
Speaker Change: Yeah. Thanks for both questions. So just be looking for the signal and of course, when we see it we will we will share it with some level of detail surrounding the go decision or the local decision and swimming ago decision you're correct in your understanding that then it would show more detailed data as a major medical needs.
Speaker Change: So that that understanding is correct from a fish three assumptions point of view I think this is the details you can see from all the fish D. Registrational trials insurance there are not many but I think that's going to be subject of the conversation with the FDA going over the data in hand.
Tim Van Hauwermeiren: From a phase three assumptions point of view, I think you're spot on. This is indeed what you can see from all the phase three registrational trials in children; there are not many. But I think that's going to be the subject of the conversation with the FDA, you know, with the data in hand, we will need to go with our proposal to another phase two meeting and basically triangulate expectations for that phase three. So for the time being, I would also build on your assumptions looking at the other registration trials. Thank you. Thanks so much.
Speaker Change: You'll need to go with our proposal into an end of these two meetings and basically translate expectations, but let's face and so for the time being out I would also be above your assumptions looking at the oldest registrational trials. Thank you.
Speaker Change: Thanks very much.
Tim Van Hauwermeiren: Your next question comes from the line of Suzanne Voorthuizen from VLK Kempen. Your line is open. Hi team, this is Suzanne from Camden, Texas, taking my question. I wanted to ask if you could speak a bit about your early stage molecules, specifically the ones with the disclosed targets, the IL-6 targeted 109 and FCRN targeted 213. What can you tell us at this moment about these two molecules and what do you think about their development? Thank you, Suzanne. And that's a great question to put the spotlight on the four IND candidates, which are on their way to India before the end of 2025.
Speaker Change: Your next question comes from the lineup Suzanne vans are Susan from V. L. K campaign. Your line is open.
Suzanne: 19, the system and thanks for taking my question.
Suzanne: To ask if you can speak a bit about your early stage molecules specific needs and wants me to disclose targets to Iowa, six starting with 109 and S targeted 213.
Suzanne: What can you tell us at this moment about <unk> and how do you think about the settlements from here. Thank you.
Speaker Change: Thank you Suzanne.
Speaker Change: Eight question to put the spotlight on the four by the candidates were shut off the right to Imdb for end of 2025 or Genesis. One of mine is a best in class <unk> with a half life is 60 days. So it's equipped with Genoa proprietary technologies.
Tim Van Hauwermeiren: ArgenX 109 is a best-in-class IL-6 antibody. It has spent most of its potency with a half-life of at least 60 days, so it's equipped with the know-how to provide the technologies. And that is, I think, a phenomenal antibody, but we were always a little bit... questioning what the central role of biology is in certain diseases. The reason that we give that molecule the goal is that we have seen several indications where we think we now see clear involvement of Iostick biology as the driver of disease, not just a bystander, but really driving it. And there are a couple of indications which we liked, you know; On the second hand, as an antagonist, people have been asking us, you know, what's wrong with Vyvgart? What are we trying to improve? But it's very difficult to improve upon Vyvgart.
Speaker Change: And that is in I think a phenomenal empty body.
Speaker Change: But we were always Elizabeth.
Speaker Change: Questioning you know what is the central role of our <unk> certain diseases.
Speaker Change: <unk> molecule to go is because we have seen several indications will be.
Speaker Change: <unk> now.
Speaker Change: And for one of our policy is the price of this is not just a bystander between its viability advil.
Speaker Change: Top of indications, which we like to know they would fix.
Speaker Change: Franchises rebuilding.
Speaker Change: And they were basically also be dispositive indications, which we think we can master variable.
Speaker Change: On the second <unk>.
Speaker Change: Have been asking US you know, what's what's wrong with this card would all be trying to improve it.
Speaker Change: Difficult to improve upon this car.
Tim Van Hauwermeiren: But what we see is that there are just more opportunities than we can handle with one molecule. I mean, Vyvgart will have an ending life, either from an IRA point of view or from an LOE point of view, and we just want to make sure we have a second very competitive molecule in the race to take on, you know, all the abundance of opportunities which we see in front of us. And therefore, we think about a long-term presence in the class, a long-term leadership role in the class. And that's exactly what, at Bonneau Q, we do.
Speaker Change: What we see that but it's just more opportunities and we can handle with one molecule.
Speaker Change: We'll have an ending life either.
Speaker Change: Either from an I R. A point of view and or in Ellery point of view and we just want to make sure. We have a second very competitive molecule in the race to take them you know all the abundance of opportunity, which we see in form of us.
Speaker Change: And therefore, we think about the long term presence in the class longterm a leadership role in the class and that's exactly what I'm molecules Sir.
Tim Van Hauwermeiren: Thanks for the questions. Your next question comes from a line of Alex Thompson from Stiefel. Your line is open.
Speaker Change: Thanks for the question.
Speaker Change: Your next question comes from the line of Alex Thomson from Stifel. Your line is open.
Karen Massey: Yeah, great. Thanks for taking the time to answer the question. On CIDP OLE data, I wonder if you could talk a little about expectations around when you will present that data, and then how much of it so far has been included in your filing with the FDA? And is there a chance that anything around dosing every other week would be included in that filing? And if not, at this point, could that potentially delay any timeline here?
Alexander Thompson: Yeah, great. Thanks for taking the question on C. I D. P. All the data I Wonder if you could talk a little about expectations around when you will present that data and then how much of it. So far has been included in your filing with the FDA.
Alexander Thompson: And is there a chance that anything around dosing every other week dosing would be included in that filing and if not at this point could that potentially delay the time I.
Tim Van Hauwermeiren: So thanks. For CIDP, we said that these are such important data that we wanted to showcase them at one of the major upcoming neurology indications. So stay tuned.
Speaker Change: <unk>. Thanks.
Speaker Change: <unk> and other piece of such important data that we wanted to showcase them at one of the major upcoming neurology indications so state units.
Tim Van Hauwermeiren: That announcement is waiting for us around the corner. And it's an important presentation, of course, because it's the first real innovation in CIDP for a very long time. And secondly, I think question number two relates to, I think, a review issue with the FDA. We will need to see how they look at the data. However, it is true that the randomized control portion of the trial was weekly dosing. In the open-label extension, we do have, I think, another week of dosing.
Asthma is waiting for his around before.
Speaker Change: And it's an important presentation of course is the first real innovation inserted P for a very long time.
Speaker Change: Secondly.
Speaker Change: I think the question number two relates to review issue with the F D. A.
Speaker Change: We will need to see how they look at the data <unk> of the <unk> in the <unk> extension, we do have uhm at the <unk> <unk>.
Tim Van Hauwermeiren: But whatever is going to show up on the label, I think ultimately this will be a conversation with the patient. And then you have to expect that Argenx will take a position, similar to what we did for MG; we will want to price transparently and responsibly and aim for broad access for patients to digitalization. So stay tuned; we will be talking about it in the near future. Your next question comes from the line of Samantha Semenkow from Citi. Your line is open. Hi, good morning.
Speaker Change: Whatever is going to show up on the label.
Speaker Change: Oh, Jesus will be conversation with the pace and.
Speaker Change: And then you have to expect that <unk> will take a position.
Speaker Change: The physician to a visit for M G.
Speaker Change: <unk> currently and responsibly and aim for broad access for patients to degeneration. So stay tuned we will be talking about it in the near future.
Speaker Change: Great.
Speaker Change: Your next question comes from the line of Samantha Samantha from City. Your line is open.
Samantha: Hi, good morning, Thanks for taking our questions.
Karen Massey: Thanks for taking our questions. I think you recently kicked off a direct-to-consumer advertisement for CIDP in preparation for the launch. I'm wondering if you could characterize how you expect this to impact the early launch for CIDP, based on your learnings from the MG launch. And then, just secondly, I think you're targeting dermatomyositis with two assets; EFRA took them out, of course, in one of the alkevia cohorts, and then EMPA in a Phase II study. Can you talk through the rationale for each drug NDM and how you're thinking about the types of patients that could be addressed with each mechanism of action? Thank you. Thank you. Well, I'll be taking questions, too. I think Karen has just come on to talk about our recent, Yeah, absolutely.
Samantha: I think he recently kicked off at direct to consumer advertisements, Firstly I D. P. In preparation for the launch I'm wondering if you could characterize how you expect us to impact the early in my checking I D. P. Based on your learnings from B M. G lunch and then secondly, I think you're targeting the amount of my site is with two assets.
Samantha: Take him out of course, any <unk> and then <unk> can you talk to the rationale for each drug, Indiana, and how you're thinking about the types of patients that can be addressed with each mechanism of action. Thank you.
Speaker Change: Thank you I'll I'll be taking question too I think Kevin's got to come in to talk about a recent.
Kevin: Yeah, absolutely. Thanks for the question as I said, we're getting more and more confidence.
Karen Massey: Thanks for the question. As I said, we're getting more and more confidence in the unmet need for CIDP as we approach launch and the more that we learn about this market. And certainly from a perspective of preparing for that launch, as you said, we recently launched an unbranded campaign to really raise the awareness of the burden of CIDP in the community. And that campaign is ongoing.
Kevin: In the the unmet need in CIBC as we approached launch in the mall that we learn about this market and certainly from our perspective of preparing for that launch as you said, we recently launched an unbranded campaign to really raise the awareness.
Kevin: Of the burden of fee ITT in the community and that that campaign is ongoing it leverages. The law. The learnings that we had an M. G launch that to educate patients and to activate patients is incredibly important in these auto immune diseases that are underserved.
Karen Massey: It leverages the learnings that we had from the MG launch, that educating patients and activating patients is incredibly important in these autoimmune diseases that are underserved, underdiagnosed, and undertreated. And that's the real goal of the campaign. Thanks for the question. Thank you, Karen.
Kevin: Uhm, Underdiagnosed and Undertreated and.
Speaker Change: The real goal of the campaign. Thanks for the question Mmm, Thanks for <unk>.
Speaker Change: <unk>. This is a very high <unk> indications.
Tim Van Hauwermeiren: Pretty complex biology, when we did the homework on the biology, including studying the skin biopsies, we see two distinct subsets of patients. And that is one subset of patients where you clearly see the involvement of pathogenic autoantibodies of the IgG type, so this actually perfectly fits as a fatigue marker. There is also a subset of patients where you see direct complement deposition and activation without the role of recruiting and, So we think both modes of action deserve a shot at goal in the end.
Speaker Change: The complex biology, when we did the bulwark of the balance sheet, including studying the skin biopsies usage.
Speaker Change: <unk> subsets of patients.
Speaker Change: And that is pumped subset of patients.
Speaker Change: The involvement of pathogenic auto antibodies, a biology science.
Speaker Change: This actually perfect.
Speaker Change: <unk>.
Speaker Change: There is also a subset of patients, but you see in the skin biopsy dived complimented position and activation without the role of recruiting also eligible servicing both most of actually deserve the shovel in Golden G M and I think the way we do the studies, there's a ton of biomarker data.
Tim Van Hauwermeiren: And I think the way we do the studies is there's a ton of biomarker data involved. So maybe we can further unravel the disease biology, and we learn how to triage patients based on the driving disease biology, which is relevant. Thanks for the question. Your next question comes from the line of Gavin Clark Gartner from Evercore ISI. Your line is open. Hi, this is Yasha on behalf of Gavin.
Speaker Change: <unk> so maybe the further on rebel this disease biology, and resorted how to triage patients based on the driving disease biology.
Speaker Change: Relevant to them.
Speaker Change: Thanks for the question.
Speaker Change: Your next question comes from the lineup Gavin Clark Gardner from Evercore ISI. Your line is open.
Speaker Change: Hi, This is Joshua Entre Gavin Thanks for taking my question. We were just wondering what you make of the ultimate discontinuation and Deborah My sinus and if you see any creature ongoing trial and then I have one follow up.
Tim Van Hauwermeiren: Thanks for taking our question. We were just wondering what you make of the ultramarous discontinuation in dermatomyositis. And if you see any read through to your ongoing trial, then I have one. Yeah, it wouldn't be unusual, you know, to see a C5 blocker fail in an indication, but then other complement interventions work. And compliment is not one size fits all, you know; compliment is a very complex system; there are multiple pathways involved. And depending on the disease, you know, different arms of compliment are involved at different stages. So, MMM, for example, is an example in case C5 blockade failed in MMM. C2 blockade, you know, is a homer. So, the same, I think, is valid for DM. It's not because a C5 blocker fails, but an upstream complement blocker de facto would fail.
Speaker Change: Mmm.
Yeah. It wouldn't be unusual you know to see if five local <unk> indication, but then.
Speaker Change: Component intervention works.
Speaker Change:
Speaker Change: One size fits all you know government is a very complex system.
Speaker Change: Multiple pathways involved and depending on the disease and a different arms of compliments are involved in a different stages. So M. M. M. For example is an example in case in Chief five located failed an M. N T. Two located you know as a form so the same I think.
Speaker Change: As well as four P M.
Speaker Change: South Dakota, five local sales the upstream Coppola Mclaughlin distracted would fail. So we did the homework on the biology breathing the compliment activities upstream of five and therefore, I think we need to do these experiments without over practice.
Tim Van Hauwermeiren: So, we did the homework on the biology. We think complement activity is upstream of C5, and therefore, I think we need to do this experiment with our own proprietary C5 blocker. Thanks for the questions.
Speaker Change: Thanks for the question.
Tim Van Hauwermeiren: And then one quick follow-up, just could you comment on any relative design differences between your phase two and phase three and then the ultramarous trial, if you happen to know... Yeah, we don't have enough details about the Ultramedes trial to really do a head-to-head comparison between, you know, study details. So... We don't have that information, and I'm not in a real position to comment on that. Thanks for the questions. Your next question comes from a line about Myles Minter from William Blair. Your line is open.
Speaker Change: Awesome and then one quick follow up just could you comment on any relatives design differences between your face to face three and then the ultimate Charles you happen to know.
Speaker Change: We don't have enough details about this trial to redo a head to head comparison between Australia details.
Speaker Change: So.
Speaker Change: We don't have that information and numbers in a position to comment on that just so.
Speaker Change: Thanks for the question.
Speaker Change: Your next question comes from the lineup miles Minter from William Blair. Your line is open.
Tim Van Hauwermeiren: Hey, thanks for taking the question. Just back on the ROSE study, I think we've seen 50% SDI response rates in prior Sjogren's trials at phase 2 on placebo. Just wondering whether that's the expectation for the 12 patients that you have on placebo in that trial. And then also, how important is it to show efficacy on the totality of data at the 24-week endpoint versus efficacy over the entirety of the trial? Again, just pointing to recent trials in the space that have shown variability between 20 and 24 weeks for efficacy. Thanks very much.
Myles Minter: Hi, Thanks for taking the question just back on the <unk> study I think we're saying like 50 per cent at Sky response riots in price Dragons Charles <unk>, just wondering whether that's the expectation for the 12 patients that you have on placebo in that trial.
Myles Minter: And then all sorry, how important is it to short efficacy on the totality of data.
Myles Minter: At the 24 week and coin.
Myles Minter: Efficacy or for the entirety of the trial again, just pointing through racer trials and the Spice should have shiner variability between 20 and 24 weeks on efficacy thanks very much.
Tim Van Hauwermeiren: Yeah, excellent question, Myles. So, yes, we have been studying precedent trials. There is some consistency in placebo responses. It's about a 40% response.
Speaker Change: Yeah, two excellent question miles. So yes, we have been studying precedent trials. There is some consistency in placebo responses about a 40% response you have applied study that we think we understand why this is an odd life. So I think you do see a reasonable consistency and historical placebo responses and that will be ingrowing assumption.
Tim Van Hauwermeiren: You have an outlier study, but we think we understand why there is an outlier. So I think you do see a reasonable consistency in historical placebo responses, and that is the incoming assumption for our trial two. In terms of endpoint, we have an endpoint to choose from. Well, we're actually looking at multiple endpoints, okay? So it's a 24-week study, but we have multiple time points where we will basically assess the patients and look at the dynamic evolution of their disease symptoms and biomarkers. So it's not just going to be a photo finish at week 24.
Speaker Change: Private trial too.
Speaker Change: In terms of <unk> and point to choose well, we're actually looking at multiple endpoints. Okay. So it's a 2004 reached study because we have multiple time points, where we will basically says the patients and look at the dynamics evolution of the disease symptoms and <unk>. So it's not just going to be a photo finish as the 2000.
Tim Van Hauwermeiren: We will look at the evolution of the data over time. Thanks for the question. Very helpful. Your next question comes from the line of Xian Deng from UBS. Your line is open. Hi, Xian from UBS. Thank you for taking my question. So I have a question on 119 in AOS.
Speaker Change: Four.
Speaker Change: Look you know with the evolution of the data over time.
Speaker Change: Thanks for the <unk> very helpful. Thank you.
Linh Dang: Your next question comes from the lineup Dang from UBS. Your line is open.
Lingling Dang: Hi, It came from you've yeah. Thank you for taking my question. So I have a question Uhm nine E.
Tim Van Hauwermeiren: I was just wondering if you could elaborate a bit more about the biological rationale for AOS. I mean, given ALS is a very complex disease. I mean, there's like protein degradation, autoimmune, and genetic factors involved.
Linh Dang: Please.
Dang: I was just wondering if you could elaborate a bit more about the biological rationale for.
Dang: I mean, giving.
Dang: A very complex C DS.
Dang: Protein deprivation <unk> genetic factors.
Tim Van Hauwermeiren: So just wondering for one night, are you coming from the angle that mask is sort of a key factor in neuromuscular synapse formation? And if that's the case, I'm just wondering if you are after disease modification here or it's more sort of like symptom relief, a bit like levofopa in Parkinson's. Thank you. That's an excellent question.
Dang: Involved with just wondering.
Dang: Are you coming from the angle that mosque, it's sort of a key factor.
Dang: Factor in Europe.
Dang: Sign up some information uhm and if that's the case I'm just wondering are you often.
Dang: These modification here.
Dang: <unk> <unk> <unk> <unk>.
Dang: Fighting Parkinson's thank you.
Speaker Change: That's an excellent question. So much gives a very interesting target is not just organized in the formation and the maturation of the function of the neuro muscular junction.
Tim Van Hauwermeiren: So musk is a very interesting target. It's not just organizing the formation and the maturation and the functioning of the neuromuscular junction, but the signal also goes in the other way.
Speaker Change: And signaled ulcer goes in the other way so.
Tim Van Hauwermeiren: So musk can signal to LRP4 back to the neurals. And one of the hallmarks of ALS is that, you know, in the initial stage, we go to a phase which we call a phase where you have repeated denervation and renervation. And there must be communication between the nerve cell and the muscle cell in that process. And we think that happens through retrograde signaling to allow the force of the neuron.
Speaker Change: Signals throughout the four thanks to the new room.
Speaker Change: And one of the hallmarks of <unk> is that you know in the initial stage.
Speaker Change: We go through a phase, which we call <unk>.
Speaker Change: Faithfully have repeated denervation and renovation.
Speaker Change: And there must be communication between the nerve cells and the muscle cells in the process and everything that happens through retrofits signalling musk two ll's before to the new room.
Tim Van Hauwermeiren: So if you can keep the innovation longer, through, you know, activating Musk, we think that could have a meaningful impact on ALS patients. So there's quite some biology thinking going on behind the program. We do realize ALS is a high risk but also a high reward. And therefore, you know, there's a ton of translation biology going into the ALS thinking. Also, the way we set up the initial clinical experiment is such that we will have a very deep look into these patients. Almost happens, you know, on that innovation, the denervation process.
Speaker Change: So if you can keep the innovation longer.
Speaker Change: That's true you know activating musk everything that could have a meaningful impact on <unk>.
Speaker Change: Despite some policy thinking going on behind the program, we do realize illnesses Paris, but also high rewards and therefore, you know there's a ton of transportation <unk> going into the <unk> thinking also debated setup the initial clinical experiments.
Speaker Change: <unk> has a very deeply into this patients almost happens you know on the innovations innovation process. So <unk>, it's an exciting experiments and be looking forward to seeing the first data.
Tim Van Hauwermeiren: So stay tuned. It's an exciting experiment, and we're looking forward to seeing the first data. Your next question comes from Joon Lee from Truist Securities. Your line is open. Good morning, this is Asimwan from June. Thanks for taking the questions. On the TED trial, I believe it was previously slated to begin in the fourth quarter last year, so I was just wondering what was behind the delay.
Speaker Change: Your next question comes from the line of Julie from sure Securities. Your line is open.
Speaker Change: Good morning. This is Osman for June thanks for taking the questions on the <unk> I believe it was previously slated to begin in the fourth quarter last year. So just wondering what was behind the delay.
Tim Van Hauwermeiren: And then my other question is on the pre-filter range; just wondering if you could just provide some details on your manufacturing capacity or your preparation for the TED trial. Yeah, for the TED trial. Thanks for the questions. For the TED trial, we made a decision to wait to start that trial so that we could use the PFS. And that's on, that trial's on track now. And for the second question, we've invested in manufacturing capacity, a conflict in the manufacturing capacity for IV subcutaneous and PFS at this stage. Your next question comes from Douglas Tsao from H.C. Wainwright. Your line is open.
Osman: Then my other question is on a Prefilled syringe just wondering if you could just provide some details on your manufacturing capacity or your preparations. Thank you.
Osman: Yeah.
Osman: Ted trial. Thanks, Thanks for the questions for the 10 trial, we made a decision to <unk>.
Ted: To start that trial, so that we could use the PFS and and that's that's all contract now uhm and for the second question Uhm. We we have invested in manufacturing capacity and are confident in the manufacturing capacity for Ivy subcutaneous Ntfs at this stage.
Douglas Dylan Tsao: Your next question comes from a line of Douglas sale from H C. Wainwright. Your line is open.
Karen Massey: Hi, good morning, and thanks for the questions. I'm just curious in terms of how you're thinking about reimbursement in CIDP or just sort of contracting with payers. I suspect you've had some early conversations and just given the fact that IDIG is already on label. The dosing frequency is greater in CIDP.
Douglas Dylan Tsao: Hi, good morning, and thanks for the questions I'm just curious in terms of how you're thinking about reimbursement N C. I D P or or just sort of contracting repairs I suspect. It has some early conversations interest given the fact that.
Speaker Change: Uhm I V. I G is already on label.
Speaker Change: The dosing frequency is greater than C. IBP do you see the same opportunities to have access and that you do the same success with access that you've had in my M G and.
Karen Massey: Do you see the same opportunities to have access and that you do, you know, have the same success with access that you had in my MG? And, you know, will it sort of have different outlines and the same opportunities for value-based reimbursement? Thank you. Yeah, thanks for the question.
Speaker Change:
Speaker Change: Sort of have different outlines and same upgrades for a value based reimbursement. Thank you.
Speaker Change: Yeah. Thanks for the question, we think a lot about how do we maximize access to patient to our innovation and we're thinking deeply about it for the CIBC launch what I can share that will take the same approach that we give you. The M. G launch will be transparent once we have a decision and we'll make responsible decisions.
Karen Massey: We think a lot about how we maximize access for patients to our innovation, and we're thinking deeply about it for the CIDP launch. What I can share is that we'll take the same approach that we did with the MG launch. We'll be transparent once we have a decision, and we'll make responsible decisions that really give patients the greatest access to our innovation that we can. So, more to come. Thanks for the question. Your next question comes from the line of Andy Chen from Wolf Research. Your line is open.
Speaker Change: That that really give patients the the greatest access to our innovation that we can the motor come thanks for the question.
Speaker Change: Your next question comes from the line of Andy Chan from Wolf Research. Your line is open.
Tim Van Hauwermeiren: Hey, thank you for taking the question. Can you talk about M-PASIBRBAR a bit and the sequencing of indications? I know you have named three indications, but how were these chosen and prioritized? Are these the best indications commercially, or are these the best indications scientifically given the mechanism and disease pathogenesis? And obviously, what are the key decision-making factors when you choose the next few indications? Thank you.
Andy Chan: Okay. Thank you for taking the question can you talk about <unk>, a bit and the sequencing of indications I know you have the name of the three indications, but how are these chosen and prioritize are these the best indications commercial to you or or the the the best indication scientifically driven the mechanism and disease.
Speaker Change: Genesis, obviously, what what are the key decision, making factor is when you choose the next few medications. Thank you.
Yeah, this is the typical way argenx works. We always start with biology. So we're not going to force-fit biology with what looks like a great commercial opportunity to then fail in the clinical trial. So we follow biology, and the three indications we publicized are MMN, for which we recently showed data, where we believe the pathogenic RGM-alternative body is actually the driver of the disease and is actually recruiting a complement. And we are sure we can block that very effectively.
Speaker Change: Yeah. This is the typical way Alcoholics works, we always talk from the biology. So we're not Gonna force fifth biology, with what looks like a great commercial opportunity to del sale in the clinical trial. So we follow biology.
Speaker Change: <unk> indications Republican.
Genesis: Bruce Fisher recently data will be please the pathogen pathogenic our gym auto antibody is actually to drive of the disease and actually is extruding, a compliment that'd be I'm sure. We can block the very effectively.
The second indication is delayed graft function. That is, in the kidney transplant setting. And there's again, very compelling evidence that the classical and the lectin pathway is in play. If you look at the kidney biopsies, and therefore C2, which is at the nexus of both pathways, is an ideal target to block delayed graft function, which actually leads to significant kidney loss in the transplant setting.
Genesis: The second indications delayed brass function.
Genesis: That is actually in the kidney transplant, setting and does a kenyan or very compelling evidence.
Genesis: The classical and the less impersonate you can play if you look at the kidney biopsy.
Genesis: And therefore C. Two which is at the Nexus of both pathways as an ideal target Tupac delays breath function, which actually leads to significant excuse me loss independence.
So the value of that organ is very high. There is a high medical need, so it's an effective commercial setting. And then the third indication is dermatomyositis, which we have already touched on. We have more indications in the works, but we always start from biology. Then we will actually look at the ability to do a clinical trial experiment in terms of availability of clinical endpoints, approvable endpoints, ability to enroll the trial, and also ability to show the signal despite taking the medication. And then, of course, we take a careful look at unmet medical needs and the ability to shape the treatment paradigm, which are two pillars to formulate the commercial rationale. So all three indications have been carefully selected based on these filters, and more invitations are underway. So we think this is our next pipeline in the program. This concludes our question and answer period and does conclude today's conference call. Thank you for your participation. You may now disconnect.
Genesis: Value of that order is very I, it's a himont medical needs.
Genesis: And effective.
Genesis: Marshall settings, and then the third indications Jonathan <unk> already touched on we have more.
Genesis: Patients in the works, which we always starts from biology.
Genesis: Then we can actually look at the ability to do the clinical trial experiments.
Genesis: The visibility clinical endpoints approvable endpoints ability to enroll the trial also ability to show the signal despite techno medication and then find out of course, we take a careful look at medical.
Genesis: And the benefit to shape the treatment paradigm, Richard two service to formulate the commercial russianoff so ultra.
Genesis: <unk> indications have been carefully selected based on these filters and modifications are unrelated servicing our next plan in your products.
Yeah, <unk>, our question and answer period and does conclude today's conference call. Thank you for your participation you may now disconnect.
Genesis: [noise].