Q3 2020 argenx SE Earnings Call

Good morning, My name is.

Operator: Good morning, my name is Repto, and I will be your conference operator today. At this time, I would like to welcome everyone to the conference call. All lines have been placed on mute to prevent any background noise.

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The background noise.

After the speakers remarks, there will be a question answer session.

Operator: After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during that time, simply press the star key followed by the number one on your telephone keypad. If you would like to withdraw your question, please press the star key followed by the number two.

I'd like to ask a question Bruce Huh.

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Operator: We do ask that you please limit yourselves to one question and a single follow-up. You may recur if you have further questions. Please also note that today's event is being recorded. Thank you. I would now like to introduce Beth DelGiacco, Vice President of Investor Relations. You may begin your conference. Thank you, Rocco.

You May requeue, if you have questions.

Please also note todays event is being recorded.

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I would now like to introduce but still George Bush.

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Thank you Rocco <unk> press release was issued earlier today with our third quarter 2020 financial results and business update its can be found on our website along with a 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.

Beth DelGiacco: The press release was issued earlier today with our third quarter 2020 financial results and business update. 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. Although actual results may differ materially from those indicated by these statements, the company 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.

Recent clinical data that 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 are Jack is not under any obligation to update statements regarding the future are jumping form those statements in relation to actual results unless required by law I'm joined on the call today by Tim that Howard Marins, Chief Executive Officer, Keith Wood, Chief operating officer and Erica.

Tim Van Hauwermeiren: I'm joined on the call today by Tim Van Hauwermeiren, Chief Executive Officer, Keith Woods, Chief Operating Officer, and Erica Stalde, Chief Financial Officer. I will now turn the call over to Tim. And thank you, Beth, and welcome, everyone.

<unk> Chief Financial Officer, I will now turn the call over to Tim.

Thank you Beth and welcome everyone.

We appreciate you joining the call today.

Tim Van Hauwermeiren: We appreciate you joining us on the call today. This has been an exceptional year for ArgenX. We have achieved several significant milestones, most notably the positive readout of our Phase 3 ADAPT trial, and done so in an unprecedented environment. However, while we are still managing the impact of COVID-19 on our lives and business. I'm continually impressed by the team we have built and their ability to deftly maneuver around the challenges which we've been presented with.

This has been an exceptional year for organics.

We have achieved several significant milestones most notably the positive freed out obviously.

Robert piece, the adapt trial and.

And don't show in an unprecedented environment.

Well, we're still monitoring the impact of the COVID-19, when our lives and business.

I'm continually impressed by the team, we have built and that ability to definitely maneuver around the challenges, which we've been presented with.

Tim Van Hauwermeiren: We are in a very strong position as we close out 2020 and look forward to our first commercial launch next year. We know that FCRN antagonists, as a new class of medicines, have tremendous potential to transform the treatment of serious autoimmune diseases, and we are closer than ever to reaching people living with our first indication, generalized myasthenia gravis. Today, we are excited to discuss the continued progress we have made in advancing our first and potentially best-in-class FC fragment as we focus on maximizing our leadership position with Afgha Tijamah. We remain equally committed to investing in R&D and enriching our deep pipeline of differentiated early and late-stage assets to generate long-term value. You'll find today's agenda on slide number three.

We already have a pretty strong position as we close out 2020 and look forward to our first commercial launch next year.

You know what a question on attacking this as a new class of medicines have tremendous potential.

Transform the treatment.

Serious autoimmune diseases can be.

Can be closer than ever to reaching people living with our first syndication generalized my seem to agree with.

Today, we are excited to discuss the continued progress we have made in advancing our first I'm supposed to be best in class if she's pregnant.

As we focus on maximizing our leadership position, we have got picking them up.

We remain equally committed to.

Two investing in R&D and enriching our deep pipeline different shade, it early and late stage assets to generate long term value.

[noise], you'll find today's agenda on slide number three.

Tim Van Hauwermeiren: I will share details on the progress we've made with Epcot Digimod, specifically the new data we presented from Adapt. Updates on our ITP-PV and CIDP trials and our fifth indication. I will then move to the rest of our pipeline, including Qsatuzumab and ArgenX 117. Keith will provide more context on our commercial readiness activities, followed by a financial update from Eric before we take your questions.

I will share details on.

The progress we've made but I've got it your most specifically the new data will be presented at that.

Updates on our I T P P b and C or D P trials, and our 50 indication.

I will then move to the rest of our pipeline, including she was up to sum up and logistics 117.

To provide more context, when our commercial readiness activities, followed by a financial update from Eric before we take your questions.

Let me begin with a cup digimarc.

Tim Van Hauwermeiren: In May, we reported that the Global Phase 3 ADAPT trial met its primary endpoint, which was a responder analysis in acetylcholine receptor antibody positive patients during the first treatment cycle. 67.7% of abglutigema-treated patients compared to 29.7% of placebo patients achieved a greater than two-point improvement on the MGADL score for at least four consecutive weeks. Similar results were seen on the QMG score.

In May we.

It is important that the global phase three adapt trial met its primary endpoint, which was it a spoiler analysis in <unk>.

Phil Koning receptor antibody positive patients during the first treatment cycle.

67.7%.

I've got it your most treated patients compared to 29.7% of placebo patients achieved a greater than two point improvement on the M. She is the l. score for at least four consecutive week.

Similar results were seen on the Q M T score.

Tim Van Hauwermeiren: 84.1% of responders had an onset of response in the first two weeks. Slide 5 shows that 40% of all patients, or about two-thirds of responders, achieved an MGADL of 0 or 1, classified as minimal symptom expression, and a substantial proportion of responders saw impressive durability, with almost 60% of patients experiencing a benefit of 8 weeks or longer and 1 third of patients experiencing a benefit of 12 weeks or longer. Additional data from ADAPT were presented earlier this month at the MGFA Scientific Session by our Principal Investigator, Dr. James Howard. These data confirmed the rapid and clinically meaningful responses to F. gartigiamat that we saw from the top line, but also showed additional analyses on the magnitude and repeatability of response in five six. To assess the magnitude of response, we looked at the MGADL and QMG score reductions at week 4, or one week after the last infusion.

84.1% to postpone this had an onset of response in the first two weeks.

Slide five shows that 40% of all patients or about two thirds took a spotless achieved an M Street, you yell zero or one close.

It's hard it's minimal symptom expression.

And the substantial proportion of responders so.

Impressive durability.

With almost 60% of patients experiencing the benefit of eight weeks, a little longer and one third of patients experiencing the benefit of 12 weeks or longer.

Additional data from it that were presented earlier this month at the M. Jiffy scientific session, but our principal investigator Dr. James Hart.

These data come from to the rapid and clinically meaningful responses to adopt digimarc that we saw from the topline, but also showed additional analyses on the magnitude and repeatability of response.

On slide six.

To assist the magnitude of response, we looked at the end she LTL and QM Ci score reductions at week, four or long beak. After the last infusion.

Tim Van Hauwermeiren: As you can see, the treatment arm continues to show benefits while the placebo arm goes to zero. We were particularly pleased to see that 50% or more of patients achieved at least a 5-point improvement on the MGADL and at least a 6-point improvement on the QMGD. A third of patients achieved a QMG improvement of at least 9 points. These responses and the proportion of patients achieving minimum symptom expression are unprecedented and capture the most important component of this trial. These are not just scores.

As you can look at increasing thresholds of improvement the treatment arm continues to show benefit while the placebo arm goes to zero.

We think a really pleased to see that 50% or more patients achieved at least a five point improvement on the NGL and at least a six point improvement on that strategy.

<unk> third of patients achieved the Q M G improvement of at least nine points.

These are the sponsors and the proportion of patients achieving minimum symptom expression are unprecedented and capture the most important component of this trial. These are not just scores. These are patients who are feeling better.

Tim Van Hauwermeiren: These are patients who are feeling better, and a substantial number are virtually symptom-free. With regard to repeatability, if you look across cycles 1 and 2, almost 80% of abgartigemal patients were considered MG ADL responders. This included 36.8% of patients who were not MgADL responders in cycle 1, but who were in cycle 2. We saw that the MGADL score reduction was consistent in both cycles, with a mean change from baseline of 4.6 in cycle 1 and 5.1 in cycle 2. We also performed some additional analysis on the Acetylcholine Receptor Antibody Negative patient.

And the substantial number are virtually symptom free.

With regards to repeatability.

If you look across cycles, one and two.

Almost 80% of I've got digital patients were considered and she did you have a spotless.

This included 36.8% of patients who were not and you get younger spot was in cycle one but.

But the credit cycle too.

We saw that the M.G.L. score reduction most consistent in both cycles with a mean change from baseline of 4.6 in cycle, one and 5.1 in cycle too.

We also showed some additional analyses on the acetylcholine receptor antibody negative patients you'll recall, we saw a high placebo response in these patients based on the energy in the old School.

Tim Van Hauwermeiren: You'll recall we saw a high placebo response in these patients, based on the MJEDL score. By including the more objective measure QMG in the analysis, we started to see more separation from treated and placebo patients. Put simply, the data show that if patients responded to efgathetumab, they did so quickly and with a significant depth and duration of improvement. This is supported by a safety profile comparable to placebo with no reduction in serum albumin. These data further demonstrate how F Cartesian Mod is uniquely differentiated and has the potential to be the best-in-class FCRN antagonist.

By including the more objective measure shoot Amaechi Indianapolis. This we started to see more separation from treated and placebo patients, but simply the.

The data show that patients responded to adopt digimarc. They did so quickly and we added significant depth and duration of improvement.

This is supported by its safety profile comparable to placebo with no reduction in serum albumin.

These data further demonstrates how I've got teach them up is uniquely differentiated and has the potential to be the best in class FCX <unk> antagonist.

Tim Van Hauwermeiren: On slide 9, you can see in our pipeline that next steps for the Garticia mod and GMG are on track, and we are pleased to confirm that we plan to file the BLA by the end of this year. In addition, our interaction with the FDA regarding the subcutaneous bridging strategy in MG is set to occur before year-end, and we will communicate on the path forward once we have regulatory feedback. We have heard from patients, physicians, and payers that having both an IV and a subcutaneous formulation of F-gutigimat will be a significant competitive advantage in MG and other indications.

On slide nine you can see on our pipeline.

Next steps what Ive got Ive got teach a modern G. M. G are on track and we are pleased to confirm that the plan too far the BLE by the end of this year.

In addition.

But interaction with the FDA regarding the subcutaneous bridging strategy and M. G is set to occur before year end and we will communicate on the path forward once we have regulatory feedback.

We have heard from patients physicians and B is that having both an IP and the subcutaneous formulation for a cottage him up will be a significant competitive advantage in LNG and all the indications.

Operator: Our exclusive access to the Halozyme technology for the FCRN target enables our subcutaneous administration to be a single fast injection, which we also believe is optimal for patient comfort and convenience. As we talk through our other indications and the specifics of those trials, you can see that we are actively prioritizing subcutaneous development to accommodate patient preferences and to adjust to this new normal, where patients may not always have easy access to all aspects of care. This brings me to the evolving strategy of our ITP program on slide. Pardon me, everyone. This is the conference operator. We seem to be having an issue with the audio.

Our exclusive access to the Halozyme technology for the EPS, yet and target the neighbors, our subcutaneous administration to be a single fast injection, which we also believe is optimal for patient comfort and convenience.

As we talk to our open indications and the specifics of those trials you can see that we are actively privatizing subcu development to accommodate patients preferences and to adjust to this new normal where patients may not always have easy access to all sites of care.

Operator: At this time, I'm going to place hold music on the call, and we're going to reconnect the speakers. Please stand by................................................ This is the conference operator. We are reconnecting the speakers at this time.

Operator: So we'll be starting to call back up here in just a moment. Please stand by. Thank you. [inaudible] Thank you for holding everyone. This is the operator. We've reconnected the speaker location.

This brings me to the evolving strategy Obama I T P program on slide.

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Operator: So this brings me to the evolving strategy of our ITP program on slide number 10. We discussed the strategy on our last update call to address the enrollment delays we've been facing due to COVID-19. We consolidated the original three registration studies into two.

Oh, the farm little personal music into the call, we're going to be like the speakers. Please standby.

Tim Van Hauwermeiren: One is the ongoing IV-only advanced trial, and the other is the subcutaneous-only advanced sub-Q trial, which is on track to begin by the end of this year. This program realignment shows our ability to adapt and be nimble as an organization, and I'm pleased with the direction in which we are headed. Ultimately, we hope this change will expedite the path for FGaP Digimod in ITP. Moving on to CIDB on slide 11. The Phase 2 Adhered Trial of subcutaneous Esgatigemat is enrolling well, and the go-no-go decision to expand the trial up to 130 patients will occur after the first 30 patients are treated in Part A. As stated in today's press release, we now expect the decision to occur in the first half of 2021 with a specific timing dependent on the impact of COVID-19 delays.

[music].

Oh pardon me everyone. This is the conference operator, well really lucky with speakers at this time, so we'll be starting to pull back up in just a moment.

Tim Van Hauwermeiren: Based on initial interactions with the FDA, we believe the expanded trial could be sufficient for registration in CIDB. We will also be evaluating sub-q FGaP Digimop in our Phase 3 address trial in Pemphigus, which is on track to start soon. I'd like to take a moment to describe this trial in more detail.

Please standby thank you.

[music].

Tim Van Hauwermeiren: As we did with developing our phase 3 trial in MG, we are working closely with patients and physicians to design the address trial as well. By paneling with key stakeholders, we can more fully understand the remaining unmet needs for pancreas patients and identify how FGAP-TGMOPS might best offer a solution to fit into the existing treatment paradigm. DENTRICUS IS A SKIN BARRIER DISEASE, so speed of onset is a crucial aspect of potential therapy.

[laughter].

Thank you for holding everyone. This is the operator <unk> locations would you. Please begin your presentation. Thank you.

Tim Van Hauwermeiren: Patients want to see healing of lesions and achievement of disease control and clinical remission as quickly as possible. We also learned that patients prioritize the ability to taper off steroids to manage side effects. [inaudible] The phase 3 address trial will be a randomized, double-blind, placebo-controlled study where the objective is to assess efficacy, safety, and tolerability in up to 150 newly diagnosed or relapsing patients with moderate to severe pancreatitis. The trial will include both Panthagoras vulgaris, or PV, and Panthagoras fallacious, or PF.

Thank you. So this brings me to the evolving strategy, if I wish I could be program on slide number 10.

We discussed the strategy on our last update call to address the enrollment delays, we've been facing due to COVID-19.

We consolidated the original three registration studies into two.

One is the ongoing I'd only advanced trial and the.

And do all that at the subcutaneous only advance acute trial, which is on track to begin by the end of this year.

This broke a realignment.

Those have an ability to adapt and be nimble as an organization and I'm pleased to the direction in which we are headed.

Tim Van Hauwermeiren: So the PF population will be kept in a similar manner to the acetylcholine receptor negative population in the MG study. Patients will be randomized to receive either sub-q FKT GMOx or placebo for 30 weeks. Patients will start on concomitant steroids based on what will be determined to be the optimized dosing regimen from the phase 2 study, and the primary endpoint will assess the proportion of patients who achieve complete remission on a minimal steroid dose at 30 weeks. Before I move to the rest of our pipeline, I'd like to briefly mention the fifth indication for GraffitiMod, which will be discussed in more detail during an investor event in the first half

Oh, the Mcneeley Hope exchange will expedite the past I've got to Jamal in keeping.

Moving onto GDP on slide 11.

If they do use trial of some good things I've got it your mouth is enrolling well and the go no go decision to expand the trial of 230 patients will occur. After the first 30 patients are treated in park eight.

As stated in today's press release, we now expect the decision to occur in the first half 2021 with the specific timing dependent on the impact of COVID-19 delays.

Tim Van Hauwermeiren: So this program is not delayed. Everything is on track, and we're moving forward. We're just not sharing yet what this indication exactly is at this time. Given the evolving competitive landscape in the FCRN class, we have decided that it would be most prudent to provide full context around this new indication closer to the phase 2 trial initiation mid next year. Our team is on track with all phase two preparation work, and they're working hard to design a thoughtful trial, as we have done with all indications today. While we wait to share details until next year, we can say that we are very excited by the opportunity this indication presents to rare disease patients. It meets the criteria we use for our indication selection strategy. That means...

Based on initial interactions with the FDA.

We believe the expanded trial could be sufficient for registration in see I'd be.

We will also be evaluating Subcu I've got did you model you know we're facing a dress trial in patients because which is on track to start soon.

I'd like to take a moment to describe this trial in more detail.

As we did with developing our phase three trial in M. G.

Our working closely with patients and physicians to design to get dressed trial as well.

By partnering with key stakeholders, we can more fully understand the remaining unmet needs for Patrick its spatial.

And I bet you fight how I've got picked them up my best offer a solution to fit into the existing treatment paradigm.

That's because she's a skin <unk> body of disease.

So beautiful onset is a crucial aspect of potential therapy.

Tim Van Hauwermeiren: There is a clear biology rationale around the role of autoantibodies and the defined clinical and regulatory path forward. It also fits squarely into our emerging franchise structure with an attractive commercial opportunity across more than one of our potential therapeutic franchises. In this way, it aligns with our broader strategy to leverage our growing commercial capabilities across multiple indications. We continue to have the broadest development program among FHLN antagonists and are committed to rolling out new indications at a pace of at least one per year. Going forward, we will take an approach to talk about each indication just before the Phase 2 initiation, so not to share information before it's necessary. Now, I want to introduce Arteza Map and ArgenX Font 17.

Patients want to see healing of lesion and it's.

And achievement of disease control of clinical remission as quickly as possible we.

We also learned that patients to prioritize the ability to taper off steroids to manage side effects.

Slide 12.

The 50 at this trial will be a randomized double blind placebo controlled study, where the objective is to assess efficacy safety and tolerability in 250 newly diagnosed or relapsed patients with moderate to severe atopic its.

The trial will include both domestic is full gavras for PV.

And stuff because fallacious or at <unk>.

Will that be a population would be kept in a similar to my left to the office to Felipe to set the negative population in D. and G. study.

Patients will be randomized.

Let me see if either so you have got feature mall or placebo for 13 weeks.

Tim Van Hauwermeiren: On slide 13, we will be sharing top-line data from the phase 2 culminate trial in early 2021 as promised. Based on an early look from Culminate, we have selected 20 milligrams per kilogram as the go-forward dose and will be prioritizing a combination approach of Cusatuzumab with the emerging standard of care, Benito-Claxon. The Phase 1b Elevate Trial evaluating double and triple combinations of CUSA, ESA, and VEN continues to enroll following a pause due to COVID-19. 514, We started the Phase 1 Healthy Volunteer Study of ArgenX-117 targeting C2. Data from this study are expected in mid-2021, a phase one trial with a test PK PD at 3C2 levels, and by availability of both IV and sub-Q formulations.

Patients will start on prominent steroids basin will be determined to be optimized dosing regimen for the phase two study.

Primary endpoints will assess the proportion of patients who achieved a complete remission in minimal steroid dose at 30 rigs.

Before I moved to the best of our pipeline I'd like to briefly mentioned the fixed indication for that kind of teacher model, which will be discussed in more detail during an investor event in the first time of 2021.

So this program is not delayed everything is on track and be moving forward with just not shedding yet what this indication exactly it's at this time.

Given the evolving competitive landscape can be executed in class, we have decided that it would be most prudent to provide full context around this new indication closer to the phase two trial initiation mid next year.

Tim Van Hauwermeiren: We will also use the phase one study to identify a phase two dose. As with our approach to Asgard Tejema... We plan to launch multiple Phase II proof-of-concept trials on the use of strong Phase I data. We are looking at severe autoimmune diseases and have already selected multifocal motor neuropathy or MMN as an initial indication.

Our team is on track that all of these phase two preparation work and they're working hard to design the possible trial.

So you don't get all indications do they.

Well I'll be brief to share details until next year, we can.

We can see that to get very excited by the opportunity to syndication is then two rare disease patients. It meets the criteria, we use for our indication selection strategy that mean.

Tim Van Hauwermeiren: We also continue to look at potential kidney indications. Finally, as part of our ArgenX 2021 vision, we aim to be a global integrated immunology company, prioritizing both our commercial efforts as well as our R&D engine through our immunology innovation program. 118 is in the lead optimization stages as we determine how best to address the groundbreaking biology on the role of chalcolate and crystals in severe airway inflammation out of the lab of Bartlom. We're also on track to finalize ArgenX 1.19 this year and will likely be communicating more on these pipeline candidates early next year.

That is a cleaner biologic rationale around the role of ultra antibodies and the defined clinical and regulatory path forward.

It also fits squarely into our emerging franchise structure with an attractive commercial opportunity across more than one of our potential therapeutic franchises.

In this way it aligns with our broader strategy to leverage our growing commercial capabilities across multiple indications.

We continue to have the broadest development program among EPS, yet on antagonists and up.

And are committed to rollout new indications and the pace of at least bumping you.

Going forward, we will take an approach to talk about each indication just before the phase two initiation so not to share information before it's necessary.

Tim Van Hauwermeiren: As we've said many times about our IIP, this is a program about co-creation, which is at the heart of everything we do at argenx. We recognize that partnering with leading disease biologists will allow us to unlock value in our key commercial franchises and bring forward the most innovative therapies to patients. Continued investment in our antibody engineering capabilities is a key component of our role in the IIP. We recently announced two new technology partnerships with Shugai and the Clayton Foundation, underscoring our commitment to the IIP by enhancing our capabilities to build the best antibodies possible and broaden the range of targets that we may address. We also announced that we will be expanding the scope of our collaboration with Helozyme for access to the enhanced drug delivery technology. As a result of the expansion, we gained three additional exclusive targets upon nomination, which brings the total to six potential targets.

No I don't think you so to sum up and not any 117.

On slide 13, we will be sharing top line data from the phase two comedy trial in early Twentytwenty long as promised.

Based on early look from culminate Rick.

We have selected the 20 milligram per kilogram as the go forward dose and will be prioritizing a combination approaches accused of tusa, Matt with the emerging standard of care, but had to collect.

The phased Bobby I love the trial evaluating double and triple combinations of cues that you saw.

<unk> and then continues to enroll falling you Paul due to corporate banking.

Slide 14.

We started the phase one healthy volunteer study about Gen X 117 targeting Q2.

Data from this study are expected mid 2021.

The phase one trial, but its its PK PD we.

We see two levels and bioavailability of both IP and Subcu formulation.

We will also use the phase one study to identify a phase two dose.

Tim Van Hauwermeiren: We have already exercised access to the enhanced technology for two targets, FCRN and C2. We believe that having access to the enhanced technology for current and future product candidates will allow us to reach more patients with our therapeutic antibodies. With that, I'd like to turn the call over to Keith to discuss our commercial preparations in more detail.

That's what I would approach to adopt digimarc.

We plan to launch multiple phase two proof of concept trials on the heels of strong phase one data.

Looking at some feed autoimmune diseases and have already selected multifocals molten neuropathy or mmm as an initial indication.

We also continue to look at potential kidney indications.

Keith Woods: Thank you, Tim. If we could go to slide 16, please. It has indeed been a very exciting year for ArgenX. I'm pleased to report that our commercial readiness activities remain on track as we prepare to file our first BLA by the end of the year and reach patients in 2021. An important part of our ongoing rolling DLA submission is our safety database, which we accrue from our ongoing ADAPT open label extension. The retention rate in the open label extension has been impressive, and we expect to be able to share data from this study at future medical meetings. We are also on track to file the JMAA in Japan in the first half of 2021 and prepare for a launch there in 2022.

Finally, let's.

However, our Genx 2020 one the vision, we aim to be a global integrated immunology company privatizing, both our commercial efforts as well as our R&D engine through our immunology innovation program.

Our Jennie swung 18 is the lead optimization stage, it's as we determine how best to address the groundbreaking biology on the roll shark laden crystal.

So these are the inflammation out of the level of bottlenecks.

Yes also on track to finalize our Jennings from 19, this year and will likely be communicating more on this pipeline candidates early next year.

As we said many times about our IP. This is a program about co creation, which is at the heart of everything we do at Docgenix.

Keith Woods: We continue to progress our launch strategies in Europe and will file with the EMA shortly after filing in Japan. We expect to be able to share with you more details about our European strategy early next year. We have committed to our first three priority regions for the commercial launch of EFGAR-TIGAMOD, but we also recognize the importance of making EFGAR-TIGAMOD available globally, and we are working through our strategy in the rest of the world, likely relying on a partner for some regions. Of course, the use of additional phase four clinical trials in many regions of the world, as well as an expanded access program, is a key factor in our patient-centric value. In terms of commercial preparation, we are building an outstanding team in our three key regions.

We recognize that partnering with leading disease biologist.

This allowed us to unlock the value in our key commercial franchises.

And bring forward the most innovative therapies to patients.

Continued investment in our antibody engineering capabilities.

A key component of our role in IP relationships.

We recently announced two new technology partnerships with Chugai and the Clinton Foundation.

Underscoring our commitment to the IP by enhancing our capabilities to build the best antibodies possible.

Broadened the range of five is that be maybe address.

We also know that people will be expanding the scope of our collaboration with the halozyme for access to the enhanced drug delivery technology.

Keith Woods: Hiring leaders with significant launch experience in rare disease and even MG specifically, there is no doubt the right team to launch EFGAR-TIGAMOD has been chosen given their deep knowledge in the neurology space, their excitement around the potential of EFGAR-TIGAMOD, and their commitment to patients. We continue to grow our field team, including medical research liaisons, thought leader liaisons, and key account directors on the payer side, and we expect to start hiring our planned 70-person US sales force by the end of this year.

And the the expansion we gain three additional exclusive far that's up on nomination.

She brings the total to six potential targets.

I've already exercised access for two targets next year and see too.

We believe that having access to the ENHANZE technology for current and future product candidates will allow us to reach more patients without therapeutic antibodies.

With that I'd like to turn the call over to Keith to discuss our commercial preparations in more detail.

Thank you Tim if we could go to slide 16. Please it has been.

Keith Woods: We recently launched our disease awareness campaign, which we know will be a crucial component to our commercial success, particularly around engaging physician customers and building awareness of this new mechanism of action. We want to ensure that our key audiences understand FCRN as a target and the role that the auto antibodies play in MG. We continue to gain important insights from patients, advocacy groups, and physicians. We have been pleased and even surprised by our ability to have very productive conversations virtually, whether through advisory boards or events such as the MGFA or AANEM. This will be good practice for the team as we do expect to launch in at least a partially virtual environment next year.

It has indeed been a very exciting year for our Janet I'm pleased to report that our commercial readiness activities remain on track as we prepare to file our first <unk> by the end of the year and reach patients in 2021.

An important part of our ongoing rolling BLE submission is our safety database, which we accrue from our ongoing adapt open label extension the retention rate and the open label extension has been impressive and we expect to be able to share data from this study at future medical meetings.

We also are on track to file the J.M.A. in Japan in the first half of 2021 and prepare for launch there in 2022.

Keith Woods: Furthermore, while we're still in the early phase of engaging with U.S. payers, feedback from both regional and national payers has been positive. The potential for individualized dosing offered by EFGAR-Tigamod is attractive, and it offers the opportunity for patients to only receive treatment when they really need it. We continue to build out and scale our global supply chain to ensure continuity of drug supply when we launch in the U.S., Japan, and Europe. As you know, we have a long-established alliance with Lanza for our manufacturing with facilities in both the U.K. and Singapore and the potential to expand even further as we launch into additional geographies or indications.

We continue to pursue progress our launch strategies in Europe, and will file with the <unk> m-. Shortly after filing in Japan, we expect to be able to share with you more details about our European strategy early next year.

We have committed to our first three priority regions for the commercial launch of after check them out, but we also recognize the importance of making after taking my available globally and we are working through our strategy and the rest of the world likely relying on a partner for some regions of course, the use of additional phase four.

Keith Woods: We partnered with Vetter for our fill and finish of our drug product and now with Cardinal for our third-party logistics here in the U.S. We trust that our partnerships with these three well-established players are the right decisions for such a key component of our launch. Now before I hand the call over to Eric, if we take a look at slide 17, I want to quickly mention an important project that we've been working on to engage with MG patients and build awareness of this devastating disease. Next month we're excited to premiere A Mystery to Me, a documentary film about Myasthenia Gravis.

For clinical trials in many regions of the world as well as an expanded access program is a key factor in our patient centric values.

In terms of commercial preparation, we're building an outstanding team and our three key regions hiring leaders with significant launch experience in rare disease and even M. G. Specifically.

There's no doubt the right team to launch after Ticketmaster has been given their deep knowledge in the neurology space their excitement around the potential escarcega <unk> and their commitment to patients.

Keith Woods: We partnered with film producer Sarofsky to capture the hidden toll that MG can take on those who live with it every day. We hope that by watching this docu-series, we can give a voice to MG patients and encourage broader empathy for people who not only suffer from MG but from any rare disease that can leave people feeling isolated and misunderstood.

We continue to grow our field team, including medical research liaison.

Neither liaison and key account directors on the payer side and we expect to start hiring our planned 70 persons U.S. sales force by the end of this year.

We recently launched our disease awareness campaign, which we know will be a crucial component to our commercial success.

Erica Stalde: Thank you, Keith. Slide 18 covers our third quarter 2020 operating results, which are also detailed in today's stress release and regulatory filings. Total operating income for the nine months ended September 30, 2020 was 42.6 million euros, a decrease of 18.6 million euros from the same period in 2019.

Typically are around engaging physician customers and building awareness of this new mechanism of action, we want to ensure that our key audiences understand fcr in as a target and the role that the auto antibodies play Nm G.

We continue to gain important insights from patients from advocacy groups and physicians, we've been pleased and even surprised by our ability to have very productive conversations virtually whether through advisory boards or events, such as the M. G epay or a any.

Erica Stalde: Due to a milestone payment we received last year under the AbbVie collaboration agreement and partially offset by the revenue recognition of the transaction price related to the Janssen collaboration and also an increase in other income driven by higher payroll tax rebates. R&D expenses for the nine months until September 30, 2020 were 246.3 million euros compared to 122.8 million euros for the same period in 2019. Selling, general, and administrative expenses were €100.4 million for the nine months of the year, compared to €41.7 million for the same period in 2019.

This will be a good practice for the team as we do expect to launch and at least a perch, partially virtual environment next year.

Furthermore, while we're still in the early phase of engaging with U.S. payers feedback from both regional and National payers has been positive.

The potential for individualized dosing offered by after taking them out is attractive and that it offers the opportunity for patients to only receive treatment when they really need it.

We continue to build out and scale, our global supply chain to ensure continuity of drug supply when we launch in the U.S., Japan and Europe. As you know we have a long established alliance with Lonza for a manufacturing with facilities in both the UK and Singapore and the potential to expand even further as we launch into additional gene.

Erica Stalde: The increases in R&D and SG&A expenditures over the prior year have been driven by the progress made within our late-stage pipeline, including higher clinical trial costs and manufacturing expenses, the recruitment of additional employees to support ongoing activities, and higher consulting and personnel expenses. We expect operating expenses to continue to increase this year as we further advance our pipeline and prepare for future commercialization. For the nine months ended September 30, 2020, financial expenses, which mainly relate to interest received and the change in fair value of current financial assets, amounted to 1.7 million euros compared to a financial income of 10.8 million euros for the same period in 2019. Exchange losses totaled 55.9 million euros for the nine months ended September 30, 2020, compared to an exchange gain of 26.9 million euros for the same period in 2019. The total net loss for the 9 months ended September 30, 2020 was 205.6 million euros, compared to a net loss of 45.1 million euros and an operating loss of 54.5 million euros for the same period in 2019.

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We partnered with better for our fill and finish of our drug product and now with Cardinal for our third party logistics here in the U.S., We trust that our partnerships with these three well established players is the right decision for such a key component of our launch.

Now before I hand, the call over to Eric If we take a look at slide 17.

I want to quickly mention important project that we've been working on to engage with Mg patients and build awareness of this devastating disease.

Next month, we are excited to premiere a mystery to me a dock you see a documentary film about my Skinnier Gravest, we partnered with film producers Serowski to capture the hidden told the M.G. can take on those who live with it every day we.

We hope that by watching the stock you series, we can give a voice to mg patients and encourage broader empathy for people, who not only suffer from M. G. But from any rare disease that can lead people feeling isolated and misunderstood.

And with that I'll turn the call over to Eric.

Thank you Keith Slide 18 call those all self quartile Twentytwenty operating results, which are also detailed in today's press release and regulatory filings.

Erica Stalde: We ended the first nine months of 2020 with 1.8 billion euros in cash, cash equivalents, and current financial assets, compared to 1.3 billion euros on December 31st, 2019. The increase was principally due to the closing of a global offering last June, including a US offering and a European private placement, resulting in the receipt of 730.7 million euros of net proceeds. I will now hand back the call to Tim. Thanks, Derek. Please turn to slide 19.

Total operating income for the nine months ended September silky Twentytwenty was 42.6 million euros, a decrease of 18.6 million euros from the same period in 2019 due to a nice some payments we received last year until the I'd be collaboration agreement and Busters.

Okay that makes it a little in your local ignition of the collection price related to the Yeltsin collaboration.

Also an increase in all the income driven by higher payroll tax rebates.

R&D expenses for the nine months ended September silky Twentytwenty, well 246.3 million euros compared to 122.8 million euros for the same period in 2019.

Tim Van Hauwermeiren: Before we take your questions, I want to take this opportunity to offer my gratitude to our exceptional team, whose hard work and unwavering devotion to patients continue to fuel us towards our first commercial launch. We see the broad potential for FCRN antagonists to be a transformative option for patients suffering from serious autoimmune diseases.

Selling general and administrative expenses were 100, and 100.4 million euros for the nine months old.

Yeah.

Best to 41.7 million useful to send it to you in 2019.

The increases in R&D and as Ginny expenditures, although the prior year I've been driven by the progress made within our late stage pipeline, including higher clinical trial costs and manufacturing expenses look as much of additional employees to support ongoing activities and I have consulting I'm just.

Tim Van Hauwermeiren: With our positive phase 3 ADAPT data in hand, our growing team and commercial infrastructure, a deep pipeline of differentiated early and late stage programs, and our strong balance sheet to drive our company forward, I believe we are well positioned to realize our ArgenX 2021 vision and to generate long-term value for our shareholders, operators, and investors. You may now open the call for questions. Thank you. We will now begin the question and answer session. To ask a question, you may press star then one on your touch-tone phone.

Right expenses.

We expect operating expenses to continue to increase this year as we felt the advance our pipeline and prepare for future commercialization.

For the nine months ended September still keep Twentytwenty financial expenses, which mainly relates to interest received under.

And somebody received on change in fair value of current financial assets amounted to 1.7 million euros compared to a financial income of 10.8 million euros for the same period in 2019.

Operator: If you're using a speakerphone, we ask that you please pick up your handset before pressing the key. To withdraw your question, please press star then two. We do ask that you please limit yourself to one question and a single follow-up. Today's first question comes from Yatin Suneja with Guggenheim. Please go ahead. Hey guys, thank you for taking my question and congrats on all the progress. Can you maybe just talk conceptually about the pricing?

Exchange losses totaled 55.9 million euros for the nine months ended September 32020, compared to an exchange gain of 26.9 million euros for the same period in 2019.

The total net loss for the nine months ended September stuff. He Twentytwenty was 205.6 million euros compared to a net loss of 45.1 million euros and an operating loss of 54.5 million euros for the same period in 2019.

Yatin Suneja: I mean, it seems like there are certain indications where you might need a little bit more chronic treatment or dosing and certain indications where you can get away with intermittent chronic dosing. So if you can conceptually talk about that, that's the first question. The second question is, could you maybe remind us what the infusion time is for the halozyme subcu and what volume you are targeting? Thank you, Yatin. So on your second question, it's very simple. It's a subcu injection, not an infusion, which is a very important difference. This is a 30-second subcu injection with a volume of just above 5 ml.

We ended the first nine months of Twentytwenty, which is 1.8 billion euros in cash cash equivalents and current financial assets compared to 1.3 billion euros on December 31st 2019.

The increase was principally due to the closing of a global offering last June including the U.S. offering on the European private placement, resulting in the receipt of 700 750.7 million euros of net proceeds.

I will now end back the call to team.

Thanks, Eric Please turn to slide 19.

Before we take your questions I want to take this opportunity to offer my gratitude to our exceptional team, whose hard work and unbelieving devotion to patients continue to fuel us towards our first commercial launch.

Keith Woods: Keith, maybe you would like to take the first question, the conceptual question on pricing across different indications. Yeah, happy to, Tim. So Yatin, on the last call, we actually discussed the pricing and how we were thinking around MG. And we actually set some kind of some goalposts and said, you know, looking at chronic IVIG as the floor given our strong efficacy, safety, and convenience. And remember, we said chronic IVIG, on average, is about $146,000 per year to treat an MG patient. And that's just the average.

We see the growth potential for EPS, yet on antagonists to be intense formative option for patients suffering from cetus opening in diseases.

Our positive piece to get that data in hand.

Our growing team and commercial infrastructure it.

Deep pipeline of differentiated early and late stage programs and have a strong balance sheet to drive our company forward I believe we have developed position to realize our our Jennings 2020, one vision and to generate long term value for our shareholders.

Operator, you may.

You May now open the call for questions. Thank you.

Thank you we will now begin the question and answer session.

Actually a question you May press Star then one on your touched so.

Keith Woods: So you're more frequently treated patients; it goes up. We've also talked about the other bookend being Solaris. But we've also said that we plan on pricing grounding our pricing and value and do not expect to be up there with Solaris. So, you know, we have the opportunity to price based on the data that Tim shared with you in the call today, which right now is the most impressive data that's been released in a phase three study. So we do have this opportunity to bring value to patients. As far as the individualized dosing schedule is concerned, compared with some additional indications that might be more chronic.

If you're using a speaker phone, we actually you. Please pick up your handset pressing the keys.

To withdraw your question. Please press Star then two we do actually you. Please limit yourself to one question and a single follow.

Oh.

Today's first question comes from Europeans to major with Guggenheim. Please go ahead.

Hey, guys. Thank you for taking my question then go watch on all the progress [noise] can you maybe just talk.

Talk conceptually about the pricing.

Seems like that a second indication, where you might need a little bit more chronic treatment or dosing and talked an indication where you can get away with intermittent chronic dosing. So if again conceptually talk about that so that's the first question. The second question is could you maybe remind us what the infusion time is for the highlights of Q.

Keith Woods: I just want to remind you that in ITP, for example, although we will be using a more chronic dosing schedule, you also know that physicians that treat ITP, after they get their patient managed, the main thing that they want to do is slowly wean them off of medication. We also know that pemphigus, for example, we're able to treat patients and get them into complete remission. And once we do, you'll see where that will not be treated chronically but will still flare up again. So we've done a lot of homework on this.

And what was why do you why you're targeting thank you.

Thank you Justin So on your second question is very simple, it's subcu injection of an infusion, which is a very important difference. This is a 32nd subcu injection with the volume of just above five them now.

Keith maybe you would like to take the first question on the conceptual question on pricing across different indications.

Keith Woods: And we've had the opportunity to study a great deal of our data from the ADAPT study, and we now feel very comfortable moving forward with our pricing strategy that will tie into MG. But also think about, you know, second, third, fourth, fifth, and even more indications. Thank you. Our next question today comes from Yaron Werber with Cowen. Please go ahead. Yeah, hi.

Yeah happy to Jim So.

So you get 10 or less to call, we actually discussed the pricing and how we were thinking around M. G.

And we actually set some kind of some goalpost and said you know looking at chronic ideology is the floor given our strong efficacy safety and convenience and remember we said chronic IBG on average is about $146000 per year to treat an M.G. patient and that's just the average so you're more frequent.

Yaron Benjamin Werber: Thanks for taking my question. So I have just a couple about the trial design, maybe Tim. Number one, for M-gene, I'm not sure what you can comment on. The subcutaneous bridging study, you know, historically within Hanes, FDA has wanted to see efficacy data. If you look at Rituxan, you look at Darsate, Herceptin, and Darzalex, but those indications don't have a biomarker like IgG.

They treated patients it goes up.

We also talked about the other book end being Solaris, but we've also said that we plan on pricing grounding, our pricing and value and do not expect to be up there towards Solaris. So you know we have the.

Yaron Benjamin Werber: So how does that factor in, and can you use an IgG as a sort of a primary surrogate and then maybe like an eight-week, you know, clinical endpoint? And then secondly, for PV, I see that the trial design makes a lot of sense. Another trial design that some KOs were talking about is maybe doing something concomitantly with Rituxan as a sort of, you know, an acute and then chronic treatment. Any thoughts? Is that something you might want to do down the line?

We have the opportunity to price based on the data that a that Jim shared with you in the call today, which right. Now is the is the most impressive data that's been released in a phase three study. So we do have this opportunity to bring value to patients as far as the individualized dosing schedule compared with.

Yaron Benjamin Werber: Thank you. Thank you, Yaron, and thank you for being with us on the call today. So concerning the bridging strategy, I think you're spot on. Strategy means that you play to your strengths.

Some additional indications that might be more chronic I just want to remind you that and I T. P. For example, although we will be we are using a more chronic dosing schedule. You also know that ER physicians that treat I T. P. After they get their patient managed the main thing that they want to do is slowly trend them off with medication.

Tim Van Hauwermeiren: And we believe that Atgartigamot is a unique drug when it comes to the literally linear correlation between its PD effects, i.e., the reduction in IgGs, and clinical benefits. I think the ADAPT study has again clearly demonstrated that. And it's with these data in hand that we have prepared the meeting request with the FDA. So there is a science-based case to be made, but let's wait until we have the meeting behind us. I don't want to preempt that meeting.

We also know that Penn I guess for example, we're able to treat patients and get them into a complete remission and once we do you will see where that will not be treated chronically, but yet a next player. So we've done a lot of homework on this and we've had the opportunity to study a great deal of our data from the adapt study and we now feel very comfortable in moving.

Tim Van Hauwermeiren: And we will certainly communicate about the outcome of the meeting as soon as we have the written minutes. Regarding your pancreas study design, you're right, I think there are several entry points into the space of pancreas, and actually, after careful work with patients and physicians, we decided to really leverage the fast onset of action and the ability to reduce steroids as fast as possible in our first study. So I think this placebo-controlled trial in the background of suboptimal prednisone or corticosteroid dosing is actually going to replicate, hopefully, the success of the Phase II study. And that's a very compelling proposition. And to get into that space, ultimately, how EFGAT-DIGIMOD is going to coexist with RedictionMath is, of course, a topic of further studies, if and when we prove success with the first. And our next question today comes from Tazeen Ahmad with Bank of America. Please go ahead. Hi, good morning. Thanks for taking my question. Hopefully, you can hear me clearly.

Word without a pricing strategy that would tie into Mg, but also think about you know second third fourth fifth and even more indications.

Thank you. Our next question today comes from <unk> <unk> <unk>.

Please go ahead.

Hi, Thanks for taking my question. So just a couple about the trial design, but maybe Tim number one for M., She and I just I'm not sure. What you can comment the subcutaneous bridging study you know historically was an hanes FDIC has wanted to see efficacy data. If you look at your talks and you look at ours with Herceptin.

And and Darzalex, but those.

Those indications don't have a biomarker love our UGI. So how does that factor in and can use and I did use a sort of a primary surrogate and then maybe like an eight week clinical endpoint and then secondly for for PV.

I see that you know that the trial design makes a lot of sense. Another trial designed with some care was we're talking about is maybe doing something coming up with your talks and is this sort of a you know an acute and chronic any thoughts is that something you might want to do down the line. Thank you.

Tazeen Ahmad: Hi Tim, I just wanted to ask you, as we await the culmination of top-line data in early 2021, can you give us an idea of what type of information we should expect at this first look at the study? And what data we should consider to be encouraging for the program? Thank you, Tazeen.

Thank you everyone and thank you for being with us on the call today. So concerning the bridging strategy I think you're spot on strategy means that you play in the strength.

And we believe that that's got to pick them up is a unique drug when it comes to the live can be linear correlation between its P.D. effect I.E. the reduction in Niger, GCE and clinical benefits I think the if that study has again clearly demonstrated that and it's with these data in hand that actually be has prepared the meeting request.

Tim Van Hauwermeiren: So, obviously, in newly diagnosed EML patients unfit for chemotherapy, what you would look to see in these data would, of course, be the number of responses and the quality of the responses on the one hand, and, of course, then the duration of these responses. We think also safety information is going to be very important because we see the future for newly diagnosed unfit EML patients as combination therapy. So, the combined ability of your drug with venetoclax, with or without videsa, is going to be very important also from a toxicology point of view. And then we also expect data that will clearly explain why the dose is what we chose it to be. So, the 20 milligram versus the 10 milligram per kilogram data.

The EBITDA. So that is a science based case to be made but let's wait until we have the meeting behind us I don't want to be empty that meeting.

He will certainly communicate about the outcome of the meeting as soon as we have the written minutes.

Oh, Yes, I think this study design you're right I think there are several entry points into the space and thickness.

And actually we decided to ask the careful work with patients and physicians to really leverage the fast onset of action.

And the ability to did use steroids as fast as possible Youd have a first study so I think.

So I think this placebo controlled trial in the background of suboptimal pentacel or corticosteroid dosing.

Derek Christian Archila: Thank you. Our next question today comes from Derek Archila with PeoplePoll. Please go ahead. Great. Hey, good morning.

Actually going to replicate hopefully the success of the phase two study and that's a very compelling proposition.

To get into that space ultimately, how affecting them off is going to coexist with addiction map is.

He is of course topic a further studies.

Derek Christian Archila: And thanks for taking my questions. So Tim, on CIDP, maybe first, just wondering if you can provide any color on some of the challenges you are or were facing with the CIDP study enrollment and what makes you confident that you'll be able to put out the results in the first half of 2021. And then second, you know, I'd love to get your insights on the type of responses that would make you, you know, make this a go decision.

FNB prove successful the first study.

Uh huh.

Our next question.

So I'm presuming Ahmad with bank of.

Please go ahead.

Hi, Good morning, Thanks for taking my question hopefully you can hear me clearly hi, Tim I just wanted to ask you as we await the coolmini a topline data in early twenties 21 can you give us an idea of what type of information we should expect after for.

Tim Van Hauwermeiren: And if there are any good reference points in terms of maybe IVIG responses that could kind of inform us, you know, here on the investor side, thanks. Thank you Derek for these excellent questions. So CIDP, you know, was a very thoughtful trial design in close partnership with physicians and patients. So we see a study that is being very well received by the community and a very high level of motivation for both physicians and patients to participate. I think the slowing down factor here is, of course, the COVID-19 pandemic, more specifically the work on-site to initiate sites, open sites, and get patients to sites. So I think the study has really picked up momentum after the first wave of the pandemic cooled off, and now I think the arc will be to navigate through what is clearly the second wave going through Europe and, soon, probably the US. So it's COVID-related.

Look at the study and what data, we should consider to be encouraging kind of program. Thanks.

Yes. Thanks, it doesn't so obviously in newly diagnosed ER and outpatient unfit for chemotherapy. What did you wouldn't look to see Indies date that would of course be the number of responses and the quality of the sponsors on the one hand and of course, then the duration of these.

Responses.

I think also the safety information is going to be very important because we.

Because we see the future for newly diagnosed and I'm 50 ml patients to be combination therapy. So the combined ability opium drugs with venetoclax a bit of it out by data is going to be very important also permit talks point of view and.

And then also expect and data, which will clearly and explain why did all of this will be chosen to be so the 20 milligram versus a 10 minute got that give them a data.

Thank you. Our next question today comes from Derrick Archie.

Oh. Please go ahead.

Hey, good morning, and thanks for taking my questions. So Tim.

So Tim just two on C. IBP and maybe first just wondering if you could provide any color on some of the challenges you are or were facing with the.

Tim Van Hauwermeiren: And to our delight, I think the study has lots of traction with the community, and we see plenty of patient excitement around innovation entering their space, a space which is actually starved for innovation. After 30 years of IVIG, people really want to see something else and something better. On your second question, response, we have not been public about the definition of the go-no-go decision points, but clearly, I can call out, for example, the response rate which was seen in the only blinded, randomized, placebo-controlled IVIG study, that is, the ICE trial, where we saw a 53% response for IVIG versus a 21% response for placebo. So I think that's an interesting point of reference

I'd Pea study enrollment and what gets you confident.

Well to put out the results in the first half 2021, and then second you know love to get your insights on the type of responses that would make you make.

It's a go decision and if there are any good reference points in terms of maybe Ivy I T responses that could kind of inform us.

Sure on the Investor side. Thanks.

Okay. Thank you like that has excellent question so.

She had if you you know it was a very thoughtful trial design in close partnership with physicians and patients. So we see a study which is being very well received by the community and.

A very high level of monetization of both physicians and patients to.

Physicians and patients to participate I think d. and slowing down. The fact that he is is of course, a coping 19th and that makes it more specifically the world on side.

To initiate sites open sites and get patients to two sites. So I think the study has really picked up momentum after the first wave of the pandemic flu though.

And not liking the ARPU will be to navigate through what can you do the second wave going through here.

Unknown Executive: And our next question today comes from David Nierengarten with Wedbush Securities. Please go ahead. Hey, thanks for taking the question. Maybe looking a little bit farther into the future, we're likely to see Opamyris from Electione get use in myosinia gravis at a bit lower price point than Soliris and, of course, a much less frequent infusion. How are you thinking about the competitive dynamics between your IV product and your subcutaneous product relative to a less frequent C5 inhibitor? And, you know, in particular, you have both competitive dynamics and the potential for, you know, out of pocket costs affecting the decision between a subcutaneous format and the IV format from a competitor. Thank you. Thank you, David. Maybe Keith, would you like to take this question?

Through Europe, and soon probably in the U.S., So it's cold weather related.

Two other delights I think the study has lots of affection with the community and we see a plenty of a patient excitement.

Around innovation entering the space.

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At the Thirtys ideology people really want to see something other than something better.

And on your second question response, we have not been public on the definition of the go no go decision point, but clearly I can call out for example, D and response rate, which was seen in the only blinded randomized placebo controlled Ivy as he said he does the ice trial will be so if.

53% of the sponsor ideology versus a 21% this bodes for placebo. So I think that's an interesting point of reference.

And our next question today comes from.

Your garden, where.

Bush Securities. Please go ahead.

Hey, Thanks for taking the question, maybe looking a little bit farther on the future you know, we're likely to see a commerce from like I get use and Macedonia gravity, so a bit lower price point than Solaris and of course are much less frequent infusions.

Keith Woods: Yeah, happy to, David. Thank you for the questions. And both very good questions.

Keith Woods: You know, ultimately, we are bringing forth both formulations IV and sub Q into, hopefully, all indications, but certainly into MG upfront. Remember, we went forward with IV because of a speed to market strategy. And that strategy is paying off; as we said, we'll be able to launch in 2021. But as we bring the bridge forward, we know that weekly dosing, when you can have a response rate that Tim shared in the prepared remarks that was up to almost 80% of patients that responded between cycle one and cycle two, and you have a safety profile of which we've had, when we talk to patients, and we ask them to rank what is And number two is safety. Actually, they rank their route of administration towards the bottom of the five measures that we took a look at with them.

How are you thinking about the competitive dynamics between your well IB product and your subcutaneous product relative to a less frequent.

Cfive inhibitor and you know in particular both.

How did the dynamics and the potential for your out of pocket costs affecting the decision between us up to 10 years format and the IB format from a competitor. Thank you.

Mhm, Thanks, David maybe Keith you would like to take this question.

Yeah happy too David.

Thank you for the questions and both very good questions.

You know ultimately we are bringing forth both formulations IB and subcu into hopefully all indications, but certainly into the M. G upfront.

Remember, we went forward with IB because of a speed to market strategy and that strategy is paying off as we said, we'll be able to launch in 2021.

Keith Woods: So, when we're producing this type of efficacy and safety for a patient, you're going to have patients that are going to want to be on therapy. I think our SubQ, as Tim mentioned, with a simple, single, easy injection that they will be able to administer to themselves at home, you combine that with an individualized dosing schedule can provide quite some convenience for our patients as well. I guess the answer to you is an individualized dosing schedule with IV and a combination of self-injection at home is how we think about what the overall efficacy rate is, how we think about how we compete. I guess the last thing that I'll say in regard to the C5, David, is that we believe that an FCRN plays upstream of a C5. By utilizing an FCRN and removing the autoantibody from the neuromuscular junction, you therefore have fewer autoantibodies to recruit complement. Thus, we can affect the neuromuscular junction in three different ways with our mechanism of action versus just one being complement.

But as we bring the bridge Ford, we know that a weekly dosing. When you can have a response rate that as Tim shared in the prepared remarks that was up to almost 80% of patients that responded between cycle. One in cycle too and you have a safety profile of which we've had when we talk to patients.

And we ask them to rank what is important to them. The number one thing is F.C. in relieving of symptoms and number two is safety actually they they recommend ranked their routes of administration towards the bottom of the five measures that we took a look at with them.

So when.

When we're when we're producing this type of efficacy and safety for a patient you're gonna have patients that are going to want to be on therapy, I think our sub Q.

Jim mentioned with a simple single easy injection that they will be able to administer to themselves at home.

You combine that with an individualized dosing schedule can provide quite some convenience for our patients as well. So I guess the answer to you is individualized dosing schedule with IB and a combination of day self injection at home is how we think about what the overall efficacy rates how.

Akash Tewari: Lastly, you mentioned out-of-pocket expense. That's where we have great optionality because of the out-of-pocket expense that could be experienced with a SubQ. We have the IV formulation that will be available. Additionally, the work that we are doing right now in our pre-commercialization is to be able to make efgartigamot accessible to patients that require it, whether that would be with some type of patient assistance programs or, in some situations, compassionate use. So, we're looking into all of these things so that we can bring the product forth competitively. Thank you. Our next question today comes from Akash Tewari with Wolf Research. Please go ahead. Thanks so much.

We think about how we compete I.

That's the last thing that I'll say in regard to the C. Five David is that you know, we believe that that an fcr in place upstream of a C fives and.

And by utilizing an fcr and removing the autoantibody from the neuromuscular junction you. Therefore have fewer auto antibodies to recruit complement so we can affect the neuromuscular junction in three different mechanism weight ways with our mechanism of action versus just one being complement.

Tim Van Hauwermeiren: For CIDP, how many naive patients are you targeting to be included within your first 30 go or no go decisions? I think we've seen from the eye trial that about 66% of those patients were IVIG naive. What internally gives you confidence that an FCRN will have efficacy in that patient population? And will your go or no go decision look at refractory and naive patients separately? And then I have a follow-up question. Thank you, Akash.

Lastly, you mentioned out of pocket expense, and that's where we have great optionality.

Because of the out of pocket expense that could be experienced with a subcu.

Yeah, the Ivy formulation that will be available. Additionally, the work that we're doing right now and our pre commercialization is to be able to address to make escorted them not accessible to patients that require at whether that would be a with some type of patient assistance programs.

Akash Tewari: We are certifying within the CIDP population for subsets of CIDP, and as you correctly point out, we are allowing in both naive and refractory patients. And basically, what we see in the study so far is a good split between the two. I mean, there hasn't been a predefined number.

Or even in some situations compassionate use so we're looking into all of these things so that we can bring forth the product competitively.

Thank you and our next question today comes from a cost.

Tim Van Hauwermeiren: So I think we will be in a position at the goal-to-go decision point for the first 30 patients to look and try to correlate the signal we see, not just with subtype of CIDP but also with disease status. So I would say stay tuned. We have a good representation of both classes in the study.

Research. Please go ahead.

Thanks, So much I'll first the ATP, how many naive patients are you targeting to be included in your first 30 go or no go decision I think we've seen from the IDE trial about 66% of those patients were Ivy I G. E. What internally gives you confidence that an EPS year end, we'll have efficacy in that patient population and will your goal or.

Keith Woods: Okay, great. And just Alexia at the recent Investor Day talked about the GMG market, looking at the 80,000 patients who were potentially even, you know, not, not poorly controlled on steroids. You've talked about NFCRN being upstream of a C5. How much do you think you could penetrate that market?

No go decision look at refractory and naive patients separately and then I have a follow up thanks.

Thank you a cash.

We are stratifying and within the city Pete population for subsets of series B and as you correctly pointed out we are allowing in both Nike and the factory at patients and basically what we see in the study. So far this is a good split between the two I mean that hasn't been a predefine number so.

So I think people will be in a position as the political decision points for the first 30 patients to look and try to correlate seeking OVC. Most just sub five oh CHP, but also the disease status. So I would say stay tuned we have a good representation of both classes in the study.

Keith Woods: And is there, you know, a possibility that you can expand to that larger patient pool? Thank you. Yeah, thanks for the question.

Keith Woods: So I'm going to tell you what our KOLs are telling us right now, which is that I've heard Dr. Howard just recently talk about where he would use EFGAR TigaMod. And the first place he would start is he would replace IVIG, he would replace plasma exchange with this product. The second thing that he talked about was being able to use it in a bridging manner because of its rapid onset of

Okay, Great and I'll just I'll, let you know that the recent Investor day talked about for the GMT market looking at the 80000 patients who are potentially even you know not not poorly controlled on steroids.

You've talked about in MTR and being a stream of it you five how I think kind of how much do you think you could penetrate that market and is there you know a possibility that you can expand into that larger patient pool.

Keith Woods: You know, he mentioned using it in patients where it would take a little while for another therapy to kick in, like a broad immunosuppressive therapy. So he mentioned using that up front, and maybe either replacing ISTs or certainly bridging patients to ISTs. The next area that he shared was patients hate steroids. And what the ADAPT trial showed was safety. And he repeated that over and over.

Okay.

No sorry, yeah. Yeah. Thanks. Thanks for the question. So I've got to tell you what our K wells are telling US right now, which is I've I've heard Dr. Howard just recently talk about where he would use F. guard Gigamon and first place you started to see would replace I'd.

Gee he would play replace plasma exchange with this product the second thing that he talked about was being able to use it in a bridging manner because of its rapid onset of action.

Keith Woods: And so patients hating steroids, he could see replacing the steroid use with that. So you get this broad utilization of EFGAR TigaMod. The last two areas are, remember in this trial, it's a broad enrollment criteria. So we go all the way from the second line down to the defined relapsed refractory patients. And we also go into the broadest set of patients because we included seronegative patients in our trial. So to answer your question with one answer, yes, I think we will land in the MG space.

Using it in patients where it would.

It would take a little while for another therapy to kick in like a broad immuno suppressive therapy. So he mentioned using that upfront and maybe either replacing I estes or certainly bridging them a patient's diabetes. The next area that he shared was patients eight steroids and what the adapt trial showed was safe.

And you repeated that over and over and so patients hating steroids or he could see replacing the steroid use with that so you get this broad utilization of EPS starting them on the last day.

Keith Woods: And then I think we will expand as physicians get comfortable. But remember here, this is a brand new mechanism of action that most physicians in the U.S. have never utilized. And so it is going to be about education and getting them comfortable with it as we expand into that broader set of patients. And our next question today comes from Joon Lee with Truer Securities. Please go ahead.

The last two areas I just remember in this trial, it's a broad enrollment criteria. So we play all the way from second lying down to the defined relapsed refractory patients and we also go into the broadest set of patients because we included the siro negative patients in our trial. So to answer your question with one answer yes, I think we will.

Land into the M.G. space, and then I think we will expand as physicians get comfortable but remember here. This is a brand new mechanism of action of which most physicians in the U.S. have never utilized and so it is going to be about education, and getting them comfort comfortable with it as we expand into that broader set of patients.

Joon So Lee: Hi, thanks for taking my question and congrats on the progress. Just looking at the safety section of the section of your presentation at a recent medical conference, the infection rates are largely similar between the drug and the placebo, but the drug arm does have a bit of a numerically higher value by about 10 percentage points. And for both placebo and the drug, they're between 30 and 40 percent ranges, which appears to be a little bit high, but I'm just not sure. Is that a typical infection rate for the GMG patient population? And along that line, assuming there's a COVID-19, a massive COVID-19 vaccination program on a global scale, possibly in 2021, would that impact your commercialization strategy in any way? Just wondering if the FDA would be curious to know, well, you know, what the impact of efrartizumab would be on the immune system, possibly, you know, eliminating some of the therapeutic antibodies that the body generate

And our next question today comes from Julie What's your Securities. Please go ahead.

Hi, Thanks for taking my question and congrats on the progress I just looking at the.

Just looking at the safety sex enough section of your presentation at a recent medical conference the infection rates are largely similar.

Between the drug and the placebo, but the drug arm does have a bit of an numerically higher valued by about 10 percentage points.

And for both placebo and the drought there between 30 and 40% range as well.

We <unk> appears to be a little bit high but I'm just not sure is that typical infection rate for G.M.G. patient population.

And along that line, assuming theres, a COVID-19, a mass mass couldn't 19 vaccination program on a global scale cost being 2021.

Joon So Lee: Thank you. Hi Joon, thanks for joining us today. I will pass on the second question to Keith in a minute, but in terms of the safety profile, when you look at it, basically speaking, we see a safety profile that is comparable to a placebo, so there is no signal. Maybe you see a numerical difference, but it is small numbers, and basically, on none of the parameters we studied, you actually see a signal for increased risk of infection.

Would that impact your commercialization strategy in any way I just wondering if the FDA curious to know what you know what the impact of I've started you might would be on the immune system, possibly you know eliminating some of the therapeutic antibodies that the body generally thank you.

Hi, guys and thanks for joining us today and I will pass on the second question to Keith in a minute, but in terms of safety profile, but when you look at it as basically speaking a b C safety profile, which is comparable to placebo. So there's no signal maybe you see under medical difference, but it is small.

Tim Van Hauwermeiren: And remember that these patients were on broad immunosuppressants already, some of which actually do suppress the immune system to the extent you may see some increased risk of infection anyhow. Mind you, before I hand over to Keith, that the mode of action of our garbage mop is to interfere with the recycling of 5GGs but not with their production. We do not target B-cells. We do not target the ability of B and plasma cells to produce IgGs. We only interfere with their persistence.

Of course, and basically on normal so part of me to be studied you see actually a signal for increased risk of infection.

Remember that these patients read on broad immunosuppressants already.

Some of it's actually to suppress the immune system to the to the extent you may see some increased risk of infection anyhow.

Tim Van Hauwermeiren: And there's plenty of scientific literature out there showing that with an SCRN antagonist, you're basically not impacting the ability of the immune response to see and react effectively to an infectious agent. How we think about commercialization in a COVID-19 environment, maybe, Keith, you're best placed to give a few comments on that? Yeah, so, Joon, we are actually fully expecting that we would be launching this product in a COVID environment. And so I'm hopeful that we could even be in a cross-functional type of launch, where it could be some live and some virtual. But We have been studying the launches.

Finally, before I hand over to keep the D. mode of action of a cottage them up is two into feed with the recycling of Fiveg, but.

But not with that production, we do not I could be so we do not.

We do not target the ability of being profitable. So stupid use Oh jeez, we only into few that assistance and there's plenty of scientific literature out there showing that and they didn't ask it an antagonist busy season, not impacting the ability of the immune system to see and react effectively do an infection.

[noise] agent and how we think about commercialization in the COVID-19 environment, maybe keep your best place to give a few comments on that.

Keith Woods: You know, Horizon had a very successful launch here during COVID with an IV product. And so we have been studying the launches and particularly how they're engaging with the healthcare professionals, so that we can replicate that type of launch. So all I can tell you is what I've said before. I'm glad that we did not wind up launching in 2020 because there's been a great deal of learning and a great deal of adapting that the team has had to go through. Thank you. Our next question today comes from Lenny Van Steenhuis with KBC Securities. Please go ahead.

Yeah. So June we are actually fully expecting that we would be launching this product in it and cobot environment and.

And so I'm I'm hopeful that we could even be a cross functional type of launch where it could be some lines and some virtual but we have been studying the launches.

Horizon has had a very successful launch here during cobot environment with an I'd product and so we have been studying the launches and particularly how they're engaging with the health care professionals. So that we can.

So that we can replicate that type of that type of launch. So all I. All I can tell you is what I've said before I'm glad that we did not wind up launching in 2020 odd because there has been a great deal of learning and <unk> and a great deal of adopting a that the team has had to go through.

Lenny Van Steenhuis: Hi, good afternoon, and thanks for taking the question. I was also looking to circle back a bit on the sub-Q formulation of Edgar Tegemult and the FDA meeting. You have already touched a bit on the discussion and, let's say, the impact of the biomarker-related data or not. Perhaps could you provide some additional info on the content of the meeting, what we can expect, and what the potential outcomes and impact on the development trajectory for the sub-Q formulation would be? And perhaps relating to that follow-up question, sub-Q formulation is, of course, tied to the partnership with Halozyme. What should we expect in terms of royalty payments to Halozyme? Companies themselves mentioned mid-single digits on average for their deals. Is this also the case for Edgar Tegemult?

Thank you. Our next question today comes from money eventually use with KBC Securities. Please go ahead.

Hi, good afternoon, and thanks for taking the question I was also looking to circle back a bit on the Subcu formulation, Oh I've got to take him off then you have the meeting you already touched a bit on the on the discussion and then let's say the impact of the biomarker related data or not perhaps could you provide some some additional inflows.

On the content of the meeting what what you can expect and then what the potential outcomes and in fact on the development trajectory for the Subcu formulation would be and perhaps relating to that the follow up question. A subcu formulation is of course tied to the partnership with Halozyme now what should we expect in terms of royalty outs payments to to Halozyme.

Tim Van Hauwermeiren: Are there any specific specificities in this deal? Can you comment on that please? Thank you. Thank you, Lenny. So on royalties, it is indeed bonsai skits in their dealmaking. So we're in the same ballpark.

Companies themselves mentioned mid single digits on average for their deals is this also is the case, where after I take him up are there any specific <unk> specific. These on this deal can you comment on that please thank you.

Tim Van Hauwermeiren: The strategy for bridging IV to Sub-Q for the FDA meeting, we're not going to go into the details. I think we just elaborated on the fact that this is going to be based on science, it's going to be based on a very substantial data set from our ADAPT phase three trial. And, of course, a growing safety database. I think we have the biggest.

Thank you let me so on royalties. It is indeed bump test it's all in good dealmaking. So we've in the same ballpark in terms of royalties actually identical.

And that means mid single digit.

Tim Van Hauwermeiren: Safety Database in this space, FCNN space, for a BAPT GMAT. So expect a science-based conversation, expect a company that is ready to argue a benefit-risk based on data. And as we said before, we do anticipate some level of exposure needed in M.G. patients in order to effectively bridge from IV to sub-Q. But rest assured, Lenny, we will communicate as soon as we have the minutes from the meeting

D and strategy for bridging IP to since Q3 of the meeting.

I'm going to go into the details I think we just elaborated on the fact that this is going to be based on science is going to be based on a very substantial data set.

From I wouldn't that 50 trial and of course and a growing safety database I think we have the biggest safety database in this space are Kevin Spacey afforded digimarc. So expect a science based conversation I expect the company wishes ready to argue a benefit risk.

Danielle Breaux: Thank you. Our next question today comes from Danielle Breaux with Raymond James. Please go ahead.

Based on data and as we said before we do anticipate some level of exposure needed in Mg patients in order to effectively bridge from IP to Subcu.

Tim Van Hauwermeiren: Hi guys, good morning, and thanks for the question. I know we have to wait to hear what your fifth indication will be until next year, but I'm curious, given what we've seen with the success of Horizon's drug in thyroid eye disease, what your thoughts are on that opportunity and the potential role for anti-FCRNs in the indication. Yeah, look, the list is long.

That's the short they need they will communicate as soon as we have to have it in minutes from the meeting.

Thank you.

Thank you. Our next question today comes from Dunhill, Brazil with Raymond James. Please go ahead.

Tim Van Hauwermeiren: There are many, many indications that it is compelling evidence that pathogenic IgGs actually drive the disease. I think PUD is one of them. Actually, there's a whole series of recent publications further documenting the pathogenic role of IgGs in this devastating disease. So it's one of the many indications where I think, from a biology point of view, it makes sense. I think it's also demonstrated now that, you know, there's a clear clinical and regulatory path to approval for this indication. And therefore, we do have this indication on our radar screen. How we actually look at prioritization of this indication is too early to discuss, but you're right.

Hi, guys. Good morning, and thanks, but thanks for the question I know, we have to wait to hear what the Airfit indication will be to next year, but curious given what we've seen with the success of Horizon's Shagun thyroid disease, what your thoughts are on that opportunity and the potential role for anti F yards in the indication.

Yeah look the list is long there are many many indications but that is compelling evidence that the pathogenic ideologies actually drive the disease I think P.D. is one of them.

Actually there's a whole series of recent publications.

The bus commencing the pathogenic roll off of Fiveg in in this devastating disease and so it's one of the many indications, but I think from a biology point if it makes sense.

Tim Van Hauwermeiren: From a biology point of view, this could be an FKRP GMOD indication. Thank you. Our next question today comes from Jason Butler at J&P Securities. Please go ahead.

I think it's also been let's face. It now that you know that is a clear clinical and regulatory path to approval in this indication and therefore, we do have this indication on our radar screen how much do you look at privatization of this indication. It is too early to discuss but you're right from a policy point of view this could be an.

Jason Butler: Hi, thanks for taking the questions. The first one on ITP, obviously, with the program you have ongoing, you'd expect to get both sub-Q and IV formulations approved. But just wondering, when you speak to physicians in the context of the IV and the potential to switch patients at some point to sub-Q, are there additional data or information that physicians would want to know who the appropriate patients would be and how to do that? And then, on PV, just if you can remind us of the rationale and the epidemiology of including the pemphigus fallacious population as well in that program. Thanks.

And that's got the German indication.

Thank you. Our next question today comes from Jason Butler of JMP Securities. Please go ahead.

Hi, Thanks for taking the questions just the first one on I T. P. Obviously with the program you have ongoing you'd expect to get both Subcu and IB formulations approved but just wondering when you speak to physicians I'm into context, the idea and the potential to switch patients at some point to Subcu are there additional data.

Tim Van Hauwermeiren: Yes, Jason, I'll start with the question about subcu IV in ITP. I just want to call out that these are two separate studies. One is going to be exclusively IV, and the other one is going to be exclusively subcu.

Were information that physicians would want, but before and who who who the appropriate patient would be and how to do that and then just second question on on on TV. Just if you could remind us of the rationale and the epidemiology of including the Pepsico's fallacious pulp.

Keith Woods: So it'll allow us to have a standalone advantage for patients with subcu. So they'll have both induction and maintenance with subcu. So there's not going to be any switching.

Relation as well in that program. Thanks.

Yes, so Jason I'll start with the question on Subcu, IB and I T. P. I just want to call out that these are two separate studies, one is going to be exclusively Ivy.

Keith Woods: When it comes to switching patients from IV to subcu, what I can tell you is that in the discussions that we've had with our KOLs, this has been more about MG, mind you, than it has been about ITP. They've shared with us that when given the preference, still 30 to 40% of patients have elected to stay on IV therapy as opposed to subcu. They just simply do not want to inject themselves.

And the other one is going to be exclusively sub Q, Oh, so it'll allow us to have a standalone advantage up for pace.

For patients with Subcu, so they'll have both induction and maintenance wit subcu, so there's not going to be a switching.

Keith Woods: And finally, lastly, the point of switching from IV to subcu, we will have data on that in the future. Because although we're waiting for the news from the FDA meetings, what we are not doing is standing still. So we are already working on some of our patients that come out of our open label extension from ADAPT and looking at potentially switching them to subcu so that we would have that type of information. Thank you. And our next question today comes from Matthew Harrison at Morgan Stanley. Please go ahead. Great. Good afternoon. Thanks for taking the question. I guess two for me.

When it comes to switching patients from IB to Subcu.

What I can tell you is in the discussions that we've had with our cable else. It's been more around M. G mind, you than it has been I TP, they've shared with us that when given the preference still 30% to 40% of patients have elected to stay on IB therapy as opposed to Subcu. They just simply do not want to inject themselves.

And finally lastly, the point of switching from IB to Subcu.

We will have data on that in the future because although we're waiting on our Oh, we're waiting on the news from the FDA meeting.

Meetings, what we what we're not doing is standing still so we are already working on some of our patients that come out of our open label extension from adapt and look at potentially switching them to subcu. So that we would have that type of information.

Matthew Harrison: One, Tim, can you just comment, when you think about CIDP, and you obviously felt confident enough to give guidance around timing now, what do you view as still the risk that that timeline could slip? Or can you just contextualize for us what potential issues you could run into that would push that out further? And then secondly, can you comment on the regulatory process in Japan? I'm just wondering how much clarity we have there once you've filed versus, say, the regulatory process in the US.

Thank you and our next question today comes from Matthew Harrison of Morgan Stanley. Please go ahead.

Great. Good afternoon. Thanks for taking the question I guess at two from me one can can you just comment.

When you think about C.I.D.P.N., you, obviously felt confident enough to give guidance around timing now what.

Tim Van Hauwermeiren: Thanks. Thank you, Matthew, and Birbaki. So CIDP, I think the enrollment is going swiftly, as we said, I think now the real COVID-19 exposure is that for those patients who are on study, their ability to perform the visits would be impacted. We have tried to mitigate against that as much as we could through home administration of products and telemedicine, but still, there is some ability required of patients to go to sites from time to time. So it's really during their stay in the study that we, of course, have to be careful about the impact of COVID-19 on patient logistics. With regard to the registration process in Japan, it's a pretty well-understood process.

What do you view is still the rest, but that timeline could slip or can you just contextualize for us what potential issues you could run into that would push that out further and then and then secondly can you comment on the regulatory process in Japan I'm, just wondering how much clarity we have there once you file versus say a regulatory.

Say, a regulatory process and the U.S. Thanks.

Thank you Matthew.

It'd be Becky, so C or D. P. I think there are any.

Enrollment is going swiftly as we said I think now the real COVID-19 exposure is that those patients who are on study did they see that ability to perform the visits would be would be impacted a we have tried to mitigate against that as much as we could through.

Tim Van Hauwermeiren: I think we can really leverage the BLA filing. I think, especially in the Q&A following the filing, there will be much more intensive Q&A, real-time Q&A, where I think there are more technical complications which have to do with, for example, translation from Japanese to English, and everything needs to be checked, of course, twice. And there is also particular attention by the PMDA to other aspects of the filing as compared to, for example, European or U.S. records.

Home administration of product and see the medicine, but still there is some ability to acquire the patience to go to size from time to time. So it's really you wanting to stay on the study at that to be.

That'd be Oh of course, we have to be careful about the impact of COVID-19 on patient logistics business.

With regards to the registration process in Japan, it's a pretty well understood process I think we can read and leveraged to be early a filing.

I think especially in the acumen, they falling to filing there will be much more intensive Q in a real time Q1. They would I think it's more technical publications, which have to do with for example translation.

Keith Woods: But I think it's a process that is very well understood by us and by the team, and we feel very comfortable going into the process. Yeah, Tim, Matthew, the only thing that I would add to this is, you know, going through the process, which is well understood; the only difference that you'll see from that here in the United States with the FDA is once we have approval by the FDA, we have a price set, and then we are; the product is available on the market. In Japan, the difference is that you have approval from PMDA. And your product is approved, and you're allowed to discuss it. But there's a three month period after that approval during which you set the price with Japan. So there's about a three month stretch between the actual approval and when you can actually make the product available to patients. And that's because of pricing, and that's standard practice there in Japan.

From Japanese to English and everything used to be a checked of course double and that is also a particular attention of the P.M.D. into all the aspects of the filing.

As compared to for example, <unk> European or the U.S.

But I think it's a process, which is very well understood by us and by the team and we feel very comfortable going into the process.

Yeah, Tim Matthew the only thing that I would add to this is you know going through the process, which is well understood. The only difference that you will see from that here in the <unk> with the F.D.A. is once we have approval by the FDA. We have a price set and then we are the product is available in the market in Japan the differences.

Douglas Dylan Tsao: And our next question today comes from Douglas Tsao with H.C. Wainwright. Please go ahead. Hi, good morning.

You have approval from P.M.D.A. and your your product is approved and you are allowed to discuss it but there's a three month period after that approval of which you said price with Japan. So there's about a three month stretch between the actual approval and when you can actually make product available to patients.

Keith Woods: Thanks for taking the questions. Just curious in terms of feedback you're getting from KOLs in terms of individualized dosing and how they intend to implement it. Do you get a sense that they're going to base this on symptoms, or are they going to be checking IgG levels? And I'm also just curious, obviously, you have four week cycles.

That's because of pricing and that standard in the practice there in Japan.

Oh, that's question today comes from Douglas Tsao with it she Wainwright. Please go ahead.

Keith Woods: Have you heard from KOLs that might be thinking about going with a four week induction of, you know, sort of weekly treatment but then maybe, you know, going with shorter cycles in subsequent periods with sort of different sort of intervals? Thank you. Yeah, so Douglas, first of all, the treatment with the individualized treatment, they're not going to be measuring IgGs to make their determination of when they redose a patient. What they have told us is your individualized treatment cycle is, you know, how I start a patient that I'm going to treat with chronic IVIG. I start them on a regularly scheduled dose, right, which would be our cycle with an interval, and then they bring them back in, and they evaluate them.

Hi, good morning, Thanks for taking the questions just.

Just curious in terms of feedback you're getting from K wells in terms of the individualized dosing and how they intend to implement it.

Do you get a sense that they're gonna based this on centers or are they going to be checking I.G. levels and I'm also just curious obviously you have four week cycle have you heard from Kay wells that might be thinking about going with a four week induction of sort of weekly treatment that then sort of maybe you know going west shore.

There are cycles in subsequent periods with with sort of different sort of interval. Thank you.

Yeah. So Douglas first of all the treatment with the individualized treatment, they're not going to be measuring our UGC to make their determination of when they reduce the patient what they have told US is your individualized treatment cycle is you know.

Keith Woods: As the patient is maintaining their response and doing well, they will continue to stretch that cycle out longer as time goes on. As far as your second question about whether they will begin to customize the dose from the cycle, I think that they do this with every product available on the market. They create dosing schedules and don't stay completely rigid. I'm talking about MG treatment. They don't stay completely rigid to what the package insert says in their intervals of dosing.

How I started a patient that I'm going to treat with chronic Ivy League I start them on a regularly scheduled dose right, which would be our cycle with an interval and then they bring them back in and they evaluate them as the patient is maintaining their response and doing well or they will continue to stretch that cycle out longer as to.

Keith Woods: So, we do get questions like that from KOLs, and in fact, if you take a look at the data that Tim shared, you know, the individualized dosing, what it really is going to allow us for some patients to have, you know, very few cycles per year, and other patients, you may see physicians decide to take them with more of a chronic dose that could be administered. They ask the questions because there are no two patients that are Thank you. And our next question today comes from Greg. Suvarnabh with Goldman Sachs.

Time goes on as far as your second question about well they began to customize the dose from the cycle I think that they do this with every product available on the market. They they create their they create dosing schedules and don't stay a completely rigid I'm talking about M.G. treatment. They don't stay completely Richard to what the path.

Package insert says then in their intervals on dosing so we do.

So we do get questions like that from Kao else and in fact, if you take a look at the data that Tim shared you know the individualized dosing what it really is going to allow us for some patients to have you know very few cycles per year and and other patients you may see physicians or decide to take them with more of a.

Greg Suvarnabh: Pardon me, Greg, your line is open. Thank you so much. Thanks for squeezing me in. A bigger picture question, maybe for Tim and Keith, maybe you can comment too, but, you know, over the quarter, we've seen competitive landscape developments, Phase 2 data for Indian Events, MG program, and also the ITP Phase 2 data from UCB. Just wondering, bigger picture, how you think about how the competitive landscape is shaping, and obviously, given J&J's acquisition of Momenta, just broad strokes on how you think the FCRN competitive landscape is shaping up. Thanks. Thank you, Greg. Maybe I will start, and I will hand over to Keith to fill in on probably some of the more commercial aspects.

Hey, chronic dose that could be administered so I I. They asked the questions because there's no two patients that are the same in Mg.

Thank you and our next question today comes from Greg.

So with all this with Goldman Sachs. Please proceed.

Pardon me Greg Your line is open.

Okay. Thank you so much thanks for squeezing me in I'm, a bigger picture question.

A bigger picture question, maybe for and Keith maybe you can comment too, but you know over the quarter Weve seen a competitive landscape developments phase two data for immune events MG program also the I.T.P. phase two data from U.C.B. I'm just wondering.

Tim Van Hauwermeiren: But I think there's one thing becoming very clear, Greg, that is that not all SCRMs are made equal. So I think we see clear baskets of clinical profiles. For example, I think IgG4 antibodies clearly have a different efficacy and safety profile from the A-glycosylated IgG1 antibodies, which typically have fewer side effects.

Bigger picture, how you think about how the competitive landscape is shaping and obviously given JNJ is a acquisition of momentum just broad brush strokes and how.

You think the EPS here in a competitive landscape is shaping out thanks.

Thank you, Greg maybe I will start and I will hand over to key to fill in on probably some of the more commercial aspect, but I think there's one thing becoming very clear is that correct and is that not all EPS yet ends I mean equal. So I think we see claims baskets of clinical profiles I think.

Tim Van Hauwermeiren: And then I think we're uniquely positioned with a fragment. I think it's fair to say that, of course, we have the largest and most advanced data set of all, but we've set the bar very high. Typically, we like to think about differentiation along the vectors of efficacy, safety, and convenience. I think on the efficacy side, we've put forward data from large studies now, which basically show unprecedented efficacy. We haven't seen anything which is coming close to that from the smaller phase two data points we saw from competition. I think on the safety side, again, a unique position.

I just you for antibodies clearly have a different efficacy and safety profile from D.A. Glycosylated RG, one antibodies, which typically come with less side effects and then I think we are uniquely positioned to be the fragment and I think it's fair to say that of course, we have the largest and most advanced dataset of all but weve put up.

We are very high and typically we like to think about.

Tim Van Hauwermeiren: I think it's very early, of course, to talk about some of the other molecules, but I think an impressive panthaguster suggests, you know, a pretty clean safety and tolerability profile going forward, with again, a key differentiator being the fact that we do not induce any drops in serum aldermen. And then, on the convenience side, as Keith already called out, we think it's important to have both an IV and a sub-Q product available in the US market. There is a need for both. And I think the Halozyme technology is again putting us in a very nice starting position. For that single fast sub-Q injection, not to be confused with this more lengthy and more cumbersome, for example, sub-Q infusion, and feel free to step in if you feel I've overlooked an important point. No, I think Tim, you know, you've covered it in those three points.

About differentiation, along the effects of efficacy safety and convenience.

I think on the efficacy side, we've put forward data from.

From large studies now average basically a put down an unprecedented efficacy we haven't seen anything which is coming close to that from D. Small phase two data points, we saw from competition.

I think on the safety side again, the unique position and I think it's very early of course to talk for some of the other molecules, but I think the adept study and especially the open label extension, where we now have patients on drug for up to two years.

And I think an impressive Spencer just a suggestion, though they're pretty clean safety and tolerability profile going forward with again, a key differentiator <unk> being the fact that we do not induce any drops in serum albumin and then I think on the convenience side. The skis already called out we think it's important to have both an ivy.

Keith Woods: The only thing that I would add, Greg, is that, you know, we believe that the total space for FCRN is so much bigger than just the four indications that we currently have made public to you. And because of that, actually, other competitors will help grow the FCRN space, we believe, more rapidly than just what we could do by ourselves. So we love the position that we're in. We love the product that we're going into this space with. But ultimately, competition can benefit all of us. Thank you, and ladies and gentlemen, this concludes today's question and answer session and today's conference call. We thank you all for attending today's presentation. You may now disconnect your lines and have a wonderful day.

And the Subcu product available in the U.S. markets. There is a need for both and I think the Halozyme technology is again, putting us in a very nice starting position for that single fast subcu injection not to be confused with these more lengthy and more cumbersome for exam.

For example, Subcu infusions.

And keep maybe few feet the stuff and if you feel like overlooked a an important point.

No I think Tim you know you've you've covered it from those three points. The only thing that I would add Greg is that you know we believe that the total space for EPS CRM is so much bigger than just the four indications that we currently have made public to you and because of that actually Oh.

Our competitors will help grow the fcr in space, we believe more rapid than just what we could do by ourselves. So we love the position that we're in we loved the product that we're going into this space Swift, but ultimately a competition can benefit all of us.

Thank you and ladies and gentlemen. This concludes today's question and answer session and today's conference call.

Thank you all for attending today's presentation. You may now disconnect your lines and have a wonderful day.

Yeah.