Q2 2021 argenx SE Earnings Call
Pending FDA interactions.
With regard to our ongoing Registrational trials, we have said that this would be 1 of execution and we would provide clarity on enrollment as Abel.
We are delivering on that commitment today.
We expect to complete enrollment this year and the ongoing.
And that's a cute trial in gmg and the advance IV trial in ICP.
This sets us up to have top line data for both trials in the first half of 2022.
Recall that we the depths of Q. The primary endpoint is based on PD effects and.
Taken at day 29.
The trial continues out to 12 weeks before patients roll over into an open label extension study.
The advance IV trial funnel patients out to 24 weeks before they can roll over into an open label extension.
The average.
And this patient trials and pick them up and <unk>.
So Q and tier and address are all making progress.
We Additionally, expect our path and silent limited to start enrolling patients into these global trials by the end of this year.
We continue to believe that net.
Registry is well positioned to be not only first in class, but also the leader in the upscale and space with its unique structure as an FC fragment with Optionality, both intravenous and subcutaneous formulations and developments with.
And with clear clinical proof of concept established.
In 4 out of 4 indications.
And perhaps most importantly, a growing safety database, which supports a favorable benefit to risk ratio.
On slide 4 you can see that specifically over 600 patients and healthy volunteers have been dosed with <unk>.
Stablish and includes 125 patients who have been on aircraft and you come up for over 12 months and 100 patients for over 18 months.
Across all the studies, we have seen no evidence of dose limiting toxicities and.
And no reduction in human serum albumin.
Topical.
And provided new perspectives during the R&D day.
We believe the broad therapeutic window that we have observed.
Allows us to dose at GAAP pick them up to the maximum benefit of patients and enables our indication selection strategy, which will continue to be our guide as we.
And which we plan to get uptake and Mark programs further.
Slide 5.
The mix of IGT mediated diseases, which have kept pick them up and potentially in our development program, we will stick to our strategy, which has been successful to date.
Starting first with the.
Biology rationale.
Our fifth indication by our scientists and our sixth indication with expenses.
<unk> aligned to the strategy.
Britain <unk> scientists on slide 6 we are building and innovative trial design exploring free subset of my scientists in which the unifying feature is muscle weakness.
And the best characterize subsets immune mediated necrotizing myopathy.
Clearly understand the role of the auto antibody and driving the muscle weakness.
And and by Synthetase syndrome and dermatomyositis.
Patients present with a similar to muscle weakness.
Globally.
And the antibody is not as well characterized and driving deep.
The proposed myositis trial will be the first time, the companys pursuing a basket trial design based on a unifying biology.
And this way, we aim to maximize the potential opportunity.
Reshape relevant population of myositis patients.
While also minimizing the potential risk.
While doing so.
With respect and regard on slide 7.
The role of the ultra antibody is very clear in the disease pathogen list efficacy.
<unk> and favorable safety and we have observed in the past we believe we can advance directly.
2 registrational trial within bolus Pemphigoid.
Having announced our next steps GAAP tick them off indications, we can start to look ahead, and how we ramp up to more indications.
And as quickly as possible.
Through our own Registrational programs.
<unk> of concept trials in the hands of a partner such as Xilinx.
Through externally sponsored research.
Slide 8.
During our R&D day last week, we introduce.
Our January 2025 vision.
Outlining specific goals as we transition to a global integrated immunology company.
First.
We aspire to make epileptic amount available for patients globally and.
To expand the commercial and development footprint of a catheter mop.
And <unk> into 15 indications across our expanding therapeutic franchises in neuromuscular diseases hematology and dermatology.
Second.
We seek to advance our second demonology pipeline candidate our journey from 17 integral multiple late stage trial.
And <unk>.
The phase 1 data we showed last week indicates that our journey from 17 has the potential to be and efficiency to broker that can durably knockdown, we see 2 levels by 99%.
The safety data, while still blinded share primarily.
Natalie grade 1 events and no increased risk of infection.
The phase II trial of <unk>, 17, and multifocal motor neuropathy is on track to start this year, and which we evaluate the potential for and attractive infrequent dosing based on the observed PK.
<unk> profile.
Third.
As we shift to be a commercial organization with a deep development pipeline.
Not taking our foot off the gas when it comes to average investment in discovery and earlier stage programs.
Slide 9.
This is exemplified by the breadth of assets, which have emerged from our immunology innovation program with.
Talked for the first time last week about <unk> hundred 19 is simple and antibody that aims to boost the neuromuscular junction across a range of indications.
This is and all of the pipeline.
The product focused on orphan indications that fits squarely into our therapeutic franchise.
Without cutting amongst our journey from 17th and will.
And our Chinese from 19, we have a company.
Within a company emerging from our neuromuscular franchise.
And in our business.
This is slightly lagged the franchise model so much.
And the strategic investments, we are making now.
With our current pipeline will continue to benefit us in the long term as we recognize economies of scale and synergies across.
For assets and multiple indications.
I would now like to turn the call over to Karl <unk>, who is joining us for the first time as our newly appointed Chief Financial Officer.
Carlo brings significant commercial expertise to <unk> and has already made important contributions ahead.
Head of our expected launch.
And Carlos our first half 2021 operating results, which are detailed in today's press release and <unk>.
Alright, and income increased to 487.5 months and almost.
6 months ended June <unk> 2021.
Compared to $34.3 million and getting the same and can't get <unk>.
The significant increase was primarily due to the recognition of a transaction price as a consequence of it.
And agreement with Johnson.
And <unk> and the recognition of fee and.
And that is and $15.1 per game.
And the closing of our strategic goals.
Operation will design that resulting in recognition of 150.
Q1, 9 million and collaboration that Apu.
Total R&D expenses increased by $84.7 million from the 6 months ended June 30th created 21 due to $273.9 million.
Compared to $189.3 million for the 6 months and the increase from the first 6 months of.
And training 1 and <unk>.
And primarily from higher external research and development.
And looking at expenses.
And again related to the aircraft thinking month program in various indications and kind of a clinical and preclinical.
'twenty correct.
SG&A expenses totaled $129.6 million over 6 months ended June 32, anti PD 1.
Compared to the 67.9 million for the 6 months ended June 32000.
That increase the result.
Total primarily from higher essentially now of expenses.
And change and fair value from non current financial assets and.
And I wanted to $11.2 million for the 6 months ended June 30 trades per day 1.
Which is the result of the closing of a serious b.
And also printing and out of Iron Mountain Therapeutics and.
Which again and explain things a profit share and exchange for Colonoscopies and license from the use of H G by.
By making antibodies come from that simple and keyboard and collateral.
Finally cash and cash.
And findings and current financial assets totaled $2.7 billion.
<unk> June 2021.
Compared to 2 billion on December 31st 2000 <unk>.
This increase is primarily from the closing of the February 2021 globally.
Equivalent and resulting in approximately $1.1 billion and net proceeds.
Secondly to $73 per $1 million non commodity Cabo non refundable development cost sharing payment to see from xyrem and of $98 million and related to the purchase of apparel.
But I think and immuno voucher from Bayer.
Okay Pharmaceuticals.
And other net cash flows used in operating activities.
I would now like to turn the call over to Keith fluids and provides an update on our commercial launch.
<unk>.
Yes.
Thank you Karl the first half of this year has been marked by several achievements, which have brought us closer than ever before to reaching patients with myasthenia gravis.
As Tim mentioned, we expect to complete enrollment by the end of this year and our adapt subcutaneous trial in.
Mg to satisfy the safety database.
Requirements for our subcutaneous trial, we are encouraging patients from our adapt plus open label extension study to switch over to the adapt subcutaneous trial.
While this may mean that we won't.
Have a bolus of patients to switch from the adapt plus trial to commercial drug at launch IV and subcutaneous formulations will provide optionality to patients and offer a potential competitive advantage when it comes to physician and payer preference.
Similarly.
As we launched our preapproval access.
And the U S, Canada and several countries in Europe.
Yes.
Broader Mg community will be critical.
Slide 12 please.
Ongoing initiatives like our patient marketing programs and our real World evidence study.
My real World Mg help us to learn about the significant total this debilitating disease takes on both patients and their care takers, we had and we will continue to listen to and learn from the <unk> community.
With our approaching December sales.
We continue to build out our team such that we can be fully.
And trained well in advance.
Specifically the hiring of the U S sales force will be completed during the third quarter, including our head of sales 8 regional business directors and 70.
Territory business managers this team along with our broader field team of thought leader Liaisons Medical research liaisons nurse case managers and field reimbursement experts will be well equipped to drive engagement and education efforts with patients physicians and payors.
While we.
Expect the initial launch trajectory to be gradual yet consistent these efforts to educate the provider community on F CRM and the role of the antibody and Mg will position us for long term success.
Before I turn the call back to Tim for concluding remarks, I want to talk about our global commercial plan.
<unk> prioritizing, Japan and Europe after the U S Slide 14 please.
We have filed our marketing authorization application for Escarcega mud and Mg and Japan, and expect an approval and the first half of 2022.
After which we will negotiate price ahead of a commercial launch.
We plan to have a fully staffed team and Japan by the end of this year.
The filing in Europe as expected and the second half of 2021, setting us up for an approval and the second half of 2022, we have hired key functional leaders and Europe as well as country managers and our priority markets.
I want you to know.
Committed to other geographies outside of the U S, Japan, Europe, and China, but we are still evaluating what the commercial plans for the rest of the world will look like.
Look forward to sharing more specifics on this and the future.
As it stands today, we are and a strong position to execute on a series of Mg launch.
<unk> over the next 2 years across multiple geographies and our 2 formulations.
I will now turn the call back over to Tim Tim.
Thank you Keith.
I'd like to conclude with the following on slide 15.
Earlier this year, we laid out our strategic priorities for 2000.
'twenty 1.
Notably restated and MG is just the beginning.
Today, with our fifth and sixth indications for <unk> taken a modern hands.
Our crew and commercial organization to support not only aircraft taking them out and Mg.
And also our earlier stage pipeline assets.
Across our therapeutic franchises.
And is more true than ever.
We are United in our commitment to improve the lives of patients and.
We believe you're well on track to reaching Goldsmith and with Mg and all the serious autoimmune diseases.
I will now hand, the call to the operator.
To start the Q&A.
Operator.
Ladies and gentlemen at this time, we'll begin the question and answer session.
To ask a question you May press Star and then 1 on your Touchtone telephone.
You are using a speaker phone we do ask you please pick.
And that and set a floor pricing the key is to ensure the best sound quality.
To withdraw your question you May press Star and <unk>.
And the interest of answering as many questions as possible, we do ask that each person. Please limit yourself to 1 question. If you do have additional questions you may rejoin the question queue.
At this time, we will pause momentarily to assemble the roster.
Our first question today comes from Joon Lee from Suntrust. Please go ahead with your question.
Yes. Thank you very much for taking our questions and for the for all the updates.
Various ICT and comment on the.
And the breadth and magnitude and the preapproval access program and the U S and.
And Canada.
And in the EU as well and related to that I. Appreciate that you are sacrificing the initial bolus of the IV patients by switching them over into the <unk>.
And just Q.
By doing that house, how much are you by how much are you speeding up potential.
Yes.
Submission of the sub Q formulation. Thank you.
Hi, Joe and it's Keith. Thank you for the question, let me address that in 2 different parts.
First.
Talk about the sub Q program, which you asked so if you think about the sub Q program.
Offer that as an opportunity for not only the sub acute bridging study, which you may have noticed noticed we increased the total number enrolled for that and that's because we've had such rapid participation and the trial, we wanted to take and opportunities.
Let us continue to fill that trial for our safety database, we have also offered or adapt.
Adapt open label extension patients from the IV trial, the opportunity to switch over to sub Q wed have some very satisfied patients on IV and so not all of them have elected to switch over to the sub.
<unk> Q and finally, you asked about the Preapproval access program that's available here in the U S. Also in Canada and in parts of Europe. There has been a nice demand for that I want to call out to you that we do this and the interest of the commitment to the patients of which we serve and Thats our number 1 priority.
We also prioritized by offering them to participate and the sub acute trial prior to the PAA.
And with that I would give you I wouldn't have great expectations that the PAA is going to put a bolus of patients for commercial transition at launch and the U S.
Thank you.
And our next question comes from <unk> Ahmed from Bank of America. Please go ahead with your question.
Hi, Tim and Germany.
We continue to study and good morning, Good morning, Tim Thanks for taking my question.
Just wanted to follow.
And closer to the Mab and what's the update there might be how are you thinking about future development and you know what level of priority is that the partner that bank.
Thanks for the questions and so what concerns for the future.
We've been going through the data.
Welcome and above full access to these and intuitive.
Data readouts.
And the scientists and clinicians are working hard on that as a team we did inform our audience in the past as you know this is becoming a business developments and priority and activity.
And we think we have and commitments to fulfill through us CML patients based on the data.
So we're seeing so far.
Clinical response to signal in that study and <unk>.
Listing signals and certain subsets of patients and importantly, a clean safety profile and in line with the historical 50 pro from the molecule. So we believe there is volume and the data it's a business development priority for feed and not go into distress.
And the internal clinical development machine to take <unk> in house. So we stayed fully focused and committed to expanding our coverage amount as fast as we can wrapping up <unk> 17, and bringing a journey from 91.
Our next question comes from Matthew Harrison from Morgan Stanley. Please go ahead with your question.
Hi, This is Max score on for Matthew Harrison, Thank you for taking our questions.
And so as you continue to engage with stakeholders can you speak to the potential payer dynamic where patients need.
Distressed sales standard of care before starting Escarcega Mark Thank you very much.
IMAX is Keith Thanks for the question and yes, we are engaging with the regional and national Payors throughout the U S.
You can see where <unk> had some restrictions that have been placed by payers on.
And the Mab and <unk>.
That is due to the inclusion and trial of the inclusion exclusion criteria of the regain trial I call out to you that we have a different inclusion criteria for our trial and so that we are treating patients across the entire treatment paradigm, I think where the rubber hits the road on what.
And can take place will be when we're actually talking with them with our price in place.
But we aim to be able to serve out of the 65000 Mg patients our target audience is about 20000, and so we'll be able to give you more information as those discussions take place and as we get closer to launch.
Great.
Right. Thank you.
Our next question comes from Arun Weber from Cowen. Please go ahead with your question.
Hi, good morning, and Brendan on for your own congrats on the progress. Thanks for taking the question just a quick 1 from US. So I know you mentioned the adapt per Q and advanced IV study will be enrolled.
What will the and this year just wanted to check in on the sub Q and ICP and.
And I have just see how you're now thinking about that fitting into the dosing scheme.
And when you file and launch there like maybe what are the 2 different from what from the 2 different trials. Maybe are you kind of looking at this point to really inform how we should think about the 2 would be used.
Thanks.
And robot.
Hey, Brandon and Keith So, yes, we did share that the IV enrollment is going on at a good pace and we expect to have that fully enrolled at the end by the end of the year. We are also concurrently enrolling a sub Q clinical trial for ICP. It did start a little bit later, but overall.
What the FDA had asked US for it was 2 phase III clinical trials. So what we're doing is running 2 very similar phase III clinical trials, just 1 with IV and 1 with sub Q.
Should position us for the Optionality that we talked about for all of our patients our payers and our health care professionals when it comes to treat.
<unk> TP patients so the sub Q trial, and not going to be concluded by the end of the year like the IV..1 is because it started a little later and they will both be included in our file.
Our next question comes from Andrew <unk> from.
And I'd Wolfe Research. Please go ahead with your question.
Alright, Thanks for taking my question. So just on Gmg, the length and neurology article and adapt very positive signals on a post hoc basis and the HR <unk> negative patients do you think that's sufficient for approval and that population given the unmet need.
Yes, so I want you to know that we honored our commitments.
And then to the Mg patient community by including the Seronegative patients and our trial, we've seen and the data that there was a treatment benefit, but and you also see and the data that theres nearly and equivalent placebo effect.
Although it was never agreed with the FDA that these.
Results had to be statistically significant and in fact, what we see is that they are not statistically significant.
We will be speaking with the FDA and it will completely be a decision of the regulatory body and we'll get back to you. After we have more information on that at the time of approval.
Thank you.
Our next question comes from Danielle Brill from Raymond James Please go with your question.
Hi, This is Alexander Danielle.
I know it might be a bit early but do you have and are you targeting estimate per sub Q after taking them on NDA submission and.
The follow up would you consider using your priorities with you voucher for such a submission.
Thanks for the question Alex.
The press release this morning talking about fully enrolling the <unk> study and before the end of the east and.
And then of course, we will be reporting out topline data.
First half of next year, and then I think that China will take its second quarter venture and life I mean in terms of looking to data.
According to data and then finding them, we have been saying publicly that it is a possibility that free reduces TRP advisor for this 1.
<unk> has plenty of opportunity from these such a voucher.
Dave I think of the <unk> study as well and it needs the <unk> trial could be a possibility for using the budget. Thank you.
Thanks.
And our next question comes from Laura Sutcliffe from UBS. Please go ahead with your question.
Hi, This is Andrew on for Laura.
And 1 on <unk>.
Please.
And at R&D Day last week, I think you flagged about 41000 patients EMEA and.
Santa Fe and previously highlighted a biological eligible population of about 7000 EMEA.
And I was wondering.
And do you agree with the SaaS and the significant portion of the <unk> population could be violence and mobile and eligible. Thank you.
Thank you for the question on <unk>.
Coating.
And the data from literature sources, which speak to identify and through our own research.
Whatever is the right number I think is both pointing at the same or possibility of opportunity like this is a sizeable opportunity a significant unmet medical needs.
Patients, which today have very limited treatment options and <unk>.
And hopefully explain and R&D day, we do believe this disease is really driven by.
By pathogenic Igt's, and therefore, I think the day more reflection of our pick them up and either.
Variable positions towards as a significant portion of these patients.
And thanks for the question.
Great. Thank you.
Our next question comes from yen and due from wells.
Please go ahead with your question.
Hi, and thanks for taking my question could you comment on the dosing frequency in the open label extension of the Phase III adapt study now that you're evaluating.
And kind of a real world.
Cargo.
Dosing and how does the dosing frequency compare with the first.
Randomized 26 week portion and also you mentioned that some patient elected to be switched to the SAP Q.
Ah study.
3 a sub Q study, which obviously is a weekly dosing schedule, but.
And more convenient with sub 2.
Could you talk about the kind of the level of interest you saw in our switching and what does that tell you are with regard to whether.
I V and.
Thank you.
And as more preferred and.
And the relative preference.
Thank you.
Great question. Thank you for this.
Take the first question and then I will hand over to my colleague <unk> to comment on the switching over situation with absolutely right and the adapt study.
These initiatives patients rolled over first open label extension study, where they follow the same cadence of dosing and that we're doing in the Registrational portion of the trial and then Youre right. After concluding the first open label expansion.
And we're already and there was a possibility to safe effective and litigation.
And the second open label extension, which enjoyed a very high and will move in rates and in those.
Hands on skilled patients treated in a remote setting from a dosing regimen and point of view than most other income based on yes. So what we said is that we will be at a certain frequency reports on our open label extension study.
Study data.
Very fortunate of course will be in a position and I'll see the re will dose and cadence and that will really be able to triangulate appropriate price and volume for the products. We will also do these patients. So I would say stay tuned we will be communicating about as lasers and of course, we continue to collect from the safety data.
And a very sizable safety database.
More than 100 patients being on 18 months drove a longer and maybe Keith you want to comment on the switching over situation lines, Yes happy to do so Tim So as you know from the open label extension, where patients are on IV. If you total up the response.
Sponsored on clinical efficacy Ah patients that had a clinically meaningful response when exposed to FTR taken months, it's at almost 80% of patients response between the first and second round. You also know that out of those responders. Those that went from minimal symptom expression and ADL and zero or 1 is almost 2 thirds of the responders.
Anders I share that with you because we have patients and our trial all the way across the treatment paradigm and some that have failed. Many other therapies. When they are finally getting that relief that they need after failing several other therapies. They don't want to be switched from something thats working so although we may think IV will be much more.
More convenient theyre happy because of the clinical response that they're receiving.
And secondly, not all patients want to have a sub Q when we have talked with the physicians. They have said somewhere on a breakdown of up to 30% of patients would prefer to have and IV over a sub Q they don't want to.
<unk> themselves and they don't want their partner to be injecting them. So look as far as a projection of where we'd be 70% sub Q and 30% Ivy I think we'll let the market test its way out and I also think there will be another component and that will be the payer situation and reimbursement on.
And Ah.
The patient wants to go in through potentially part B part D specialty pharmacy home infusion or actually visiting and infusion center. The good news is we're providing net optionality.
Got it very helpful. Thank you.
Our next question comes from Thomas.
Amit <unk> from KBC Securities. Please go ahead with your question.
Yes, Matt Thanks for taking my question.
And wondering about the progress and the ITC trial.
We sold up enrollment seems to be a bit more difficult than we had some anticipated.
Perhaps related to oil and.
Whether it's cohorts and I was wondering how does it affect the timeline for the SC trial. Thank you.
Well, thank you for joining us in the call today and it is true and we have been talking about it frequently and the previous quarterly calls that corporate has been impacting all our ongoing clinical trials I.
I think that the <unk> study.
Look at 2 a fortunate to actually conclude that study before the Covid pandemic really.
Society, but all of the files have been impacted.
To some extent so also the ICP IV and the ICP some piece of the.
Businesses and these studies are global studies.
And that basically able to adapt and adjust to the global pandemic and all the different waves of the Covid pandemic and.
And so auto enrolling quite well so he is going to be enrolled first.
But I think the Q ICT study the <unk> study the <unk> study.
And that also enrolling quite well, so cooperative and situations, we get increasingly under control.
Okay. Thank you.
And our next question comes from Greg Savanna and from Goldman Sachs. Please go ahead with your question.
Yeah.
Yes, thanks, Kevin.
And good afternoon, and thanks for taking my question.
Just a very quick 1 and apologies if this thing.
And asked before but just on timelines with.
T V and CIB P. I appreciate that enrollment seems to be getting better and you're still in a COVID-19 or coming out of a COVID-19 type of landscape, if youre not able to provide.
And at least today.
Exact or approximate timelines.
And when would you anticipate being in that position do you think it could happen at the next quarterly update.
Yeah. Thanks, Thanks for being with us today and quite so what we said is that in these quarterly calls we will give you.
And from time to standard based on the clinical pipeline and just likely doing today are actually honoring our commitments, we cannot speak with certainty box and completing enrollment and the <unk> study and the <unk> study and I think in the coming quarters going forward expect us to be getting similar information if and when the situation.
So when we then over and be very close to completion of enrollment we are committed to inform you quarterly call. So I would say stay tuned.
And we should not be naive I think now with the sales of audience, we need to watch outs and looked and ocean victory too fast.
But our global studies are actually recruiting quite well.
And it allows thank you.
Thank you.
Our next question comes from <unk> and Asia from Guggenheim. Please go ahead with your question.
Yeah, Hey, guys. This is Eddie on for yacht and thanks for taking my question just as a follow up to that last 1 I was wondering if you could just sort of give us a sense of the cadence of data from some of.
Ongoing trials and if any of the early data releases will derisk and either later ones I'm thinking specifically about the pemphigus programs and just give us a sense of sort of what the what we're going to learn and win over the next sort of 12 to 18 months. Thanks.
Yes, I think as we've said before thank you for the question at what we said before is 2.
These from first to really predict with certainty given the book.
Pandemic situation when states with us being completely enrolled and therefore when data Readouts will come.
We will keep you informed but then be few sufficiency constant and the bumps enrollment of trials and to your second portion of the of the question.
Question, Yes, we do believe that a successful readout and <unk> will have her reads through to quarter specific or bigger vessel whole rationale why we actually initiated from identical a phase III Registrational study and bolus Pemphigoid building from the phase II proof of concept intact because so.
And so that is from a connection between these indication or indications.
And said, let's be careful right.
These diseases remain distinct and different diseases to be their own biology, so which will further derisk, but probably not completely.
Thanks for the question.
Yeah.
And ladies and gentlemen, with that we will conclude todays question and answer session as well as today's conference call. We do thank everyone for attending please have a great day you may now disconnect your lines.
Yeah.
Goodbye.