argenex Q2 2023 Earnings Call Transcript

Quartr
Provided by Quartr
July 27, 2023

Key Takeaways

  • argenx reported Q2 2023 total revenues of $281 million (including $269 million in net product sales) and $489 million in H1 net ZIVGARD sales, driven by consistent quarter-over-quarter growth.
  • The company ended Q2 with approximately $2 billion in cash and raised $1.3 billion in gross proceeds from a global equity offering to fund pipeline and commercial expansion.
  • Positive Phase 3 ADAPT data in CIDP showed a 67% response rate and a 61% reduction in relapse risk, supporting FDA interactions and a potential paradigm shift for CIDP treatment.
  • Pipeline momentum continued with MMN proof-of-concept go-ahead in the ARTAS study, Phase 3 readouts in ITP and pemphigus expected later this year, and plans to start DGF and dermatomyositis studies.
  • argenx recorded an operating loss of €102 million in Q2 and has suspended its prior $500 million annual cash burn guidance pending review of increased operational spend.
AI Generated. May Contain Errors.
Earnings Conference Call
argenex Q2 2023
00:00 / 00:00

There are 15 speakers on the call.

Operator

Good morning. My name is Sarah, and I will be your conference operator today. I would like to welcome everyone to the call. At this time, all lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question and answer session.

Operator

Global Head of Corporate Communications and Investor Relations. You may now begin your conference.

Speaker 1

Thank you, operator. A press release was issued earlier today with our half year twenty twenty three financial results and the second quarter 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 2 that forward looking statements may be presented during this call. These may include statements about our future expectations, clinical developments, regulatory timelines, the potential success of our product candidates, financial projections and upcoming milestones.

Speaker 1

Actual results may differ materially from those indicated by these statements. Argenx is not under any obligation to update statements regarding the future or to conform those statements in relation to actual results unless required by law. I'm joined on the call today by Tim Van Hauwermeiren, Chief Executive Officer Carl Bubitz, Chief Financial Officer and Karen Massey, Chief Operating Officer. I'll now turn the call to Tim.

Speaker 2

Thank you, Beth, and welcome everyone. I'll begin on Slide number 3. This has been an incredibly exciting time at argenx and we are so happy to be delivering good news to the gMG and CIDP communities and patients living with autoimmune disease more broadly. We have successfully achieved several key milestones that we laid out at the beginning of the year, bringing us one step closer to our mission to transform treatment for people living with severe autoimmune diseases. Transformation to us It means patients living their lives with minimal interruption due to their disease, so they can have more time to do things that they enjoy.

Speaker 2

We see this as a time of hope for patients who have been waiting many years for meaningful innovation. Slide 4. At the beginning of the year, we outlined an ambitious plan to create value for our key stakeholders by driving sustained growth across the business. Commercially, our commitment was to expand into earlier GMC Patient segment and broaden into new geographies. We have made progress across both of these goals, which Ken will talk about later in the call.

Speaker 2

From the clinical side, we said we would advance efgartigimod programs forward to demonstrate the power of our SC fragments As a pipeline and a product opportunity, our conviction in this opportunity has only grown Following the positive CRDT data last week and we expect more momentum later this year with 2 Phase 3 readouts in ITP and Stenfigus. We also committed to continued investment in innovation. This includes both our early stage programs and positive remarks and ARGX-four nineteen, but also our pipeline expansion through our immunology innovation program. I'm proud to say We are quite on track with our goals for 2023, having achieved the milestones we laid out and the year is not open yet. Slide 5.

Speaker 2

Very quickly, I would like to recap the recent clinical news in CIDP and Last week, we announced positive AGI results. We studied a broad patient population and saw a 67% response rate in Stage 8, supporting our long standing hypothesis that CIPP is an IgG mediated disease. In Stage B, the study made its primary endpoint with an impressive p value demonstrating that Zivkaib Khartrulho Reduces the risk of relapse by 61% based on time to first adjusted in cap deceleration. We had several conversations with neurologists following our announcement, which were highly encouraging around the potential for these data to create a paradigm shift. The team is now preparing for an upcoming interaction with the FDA on the data, while simultaneously working on the SLA.

Speaker 2

Slide 6. In June, we also shared these 2 data of ENFAR for MMN. The key highlights are, We are advancing forward with the ARTAS study. This decision follow a planned safety review by an independent data monitoring committee of 22 patients in cohort 1, including 9 who completed the full 16b3 with PFS. The IDMC confirmed a favorable safety and tolerability profile Consistent with results from the Phase I study and gave us a go ahead to move forward.

Speaker 2

Based on this recommendation and an internal efficacy assessment. We believe we have established proof of concept in MMN and will be advancing to a second dose cohort. We plan to report top line results from the full study next year. AMPA management is just the first indication for AMPA, We believe this could be our 2nd pipeline in the product opportunity. We are on track to start a Phase 2 study in DGF later this year and in dermatomycitis earlier next year.

Speaker 2

Slide 7. The addition of our leadership in the neuromuscular space is Quickly emerging and you can see the puzzle pieces coming together with each quarter of revenue growth from Vemcard and Vemcard HIFTRUDA and the key data readouts from our clinical programs. We have generated almost $500,000,000 in net sales this year to date, driven by consistent quarter over quarter growth. VISTA HYTRULO was approved in June. So we now have 2 approved commercial products for GNG patients, honoring our long term commitment to this community.

Speaker 2

With Adyiv, it's important to consider what is positive outcome means to the neuromuscular community. This was the largest CIDP trial ever run and the first that included several unique design features. We hope that we will set the bar on what CIDP trials look like going forward to ensure the right patients are getting into the study. Beyond the impressive efficacy and safety, we also uncovered important biology insights Showing CIDP is IgG mediated. This is what we want leadership to look like for all of our indications, Doing what it does for patients, physicians and to further our fundamental understanding of disease biology.

Speaker 2

The next efficacy of readouts within our neuromuscular franchise will be myositis, where we are also innovating with our file design by including 3 unique subsets necrotizing, anti synthetase and dermatomyositis. These subsets are unified by muscle deterioration associated progression of the disease But can present very differently with all the physical manifestations in the joints, lung, skin or other compartments. Each subset is also characterized by the all unique IgG autoantibody and we believe the field is ready to use The IgG autoantibody signature to reclassify myelitis. We expect a Gorna organization next year, which will inform us whether to advance all these subjects into the Phase 3 and will provide the underlying biology insights into the broad treatment of my guidance. We're also building up our pipeline opportunities with ENVAC and ARGX-1 hundred and nineteen.

Speaker 2

We are thrilled to have proof of concept in MMN that has also published significant translation data on the underlying biology. From a franchise perspective, MMN fits squarely into the core capabilities we are building. We are currently in the Phase 1 healthy volunteer study with our GENESPR19, after which we will be evaluating its potential Incon general 19th syndrome and ALS as part of our Phase 1b patient cohort. Our commitment within neuromuscular extends across our business. First, the relationships we are building commercially with the neuromuscular community, including patients, physicians and advocacy groups.

Speaker 2

We are also building a reputation clinically in how we design clinical trials to be patient friendly and unlock key translation biology insights. And all of this is rooted in the scientific foundation we are building in the space, founding the leading neurodisease biologists and seeking out opportunities to innovate and expand our pipeline going forward within neuromuscular. And with that, I'm going to turn the call over to Carl to talk about our 2Q and half year financials and our recent equity raise. Thank you, Tim. Slide 8.

Speaker 2

Our Q2 2023 financial results are detailed in our press release from this morning, So I will only highlight the key points here. The continued momentum of our launch is well reflected in our 2nd quarter revenues. We generated $281,000,000 total revenues, including $269,000,000 in global net product sales And $12,000,000 in collaboration and other revenues. In looking at the regional breakdown of our global product sales, You can see that $244,000,000 was from the U. S, dollars 13,000,000 from Japan and $12,000,000 from Europe and our distributor markets.

Speaker 2

The launch in Europe is largely Germany. Remember that beginning in March, We started to accrue revenue in Germany at the projected negotiated price, which we will learn in September. This accounts for the lower quarter over quarter growth in Europe. Other markets will start to contribute in Q3 onwards following our launch in Italy. Our total expenses were €383,000,000 for the 2nd quarter, Indicating an operating loss of $102,000,000 for the quarter.

Speaker 2

We ended the quarter with $2,000,000,000 in cash, Cash equivalents and current financial assets. This excludes gross proceeds of approximately 1,300,000,000 from the global offering we completed last week, which will allow us to execute on the many opportunities ahead, Specifically on the heels of positive ATIR data and our growing conviction in VIBGAR and the rest of our pipeline, We are still in the process of reviewing how our increased ambition level will change our operational spend. So as of today, We are not able to confirm our prior cash burn guidance of $500,000,000 and we'll provide an update at a later date. I'll now hand the call to Karen for a commercial update.

Speaker 1

Thank you, Carl. Let's go to Slide 9. We started the year with a clear and simple vision commercially to reach more patients with VIVGARTS globally. We plan to do this by driving multidimensional expansion in gMG, including geographic expansion, the launch of our subcutaneous product and by driving usage into earlier gMG treatment lines. We also look ahead to the multiple data readouts expected this year and how this would drive expansion into new patient segments over time.

Speaker 1

I'm going to talk today about the continued strong performance from our gMG launch, but also about the pivot point we are facing as we look ahead to the evolving opportunity before us. The outstanding results from this year have strengthened our conviction that we will be a leader beyond MG in neuromuscular and that we can start delivering the launch capabilities we have built to prepare for multidimensional expansion across product presentations, new geographies and towards the portfolio of indications. Slide 10. Starting with our recent commercial performance, we had a great first half of the year generating $489,000,000 in net ZivGuard sales over the 1st 2 quarters. This impressive revenue number translates to more patients getting access to VIVVAT and more opportunity for us to change patients' lives.

Speaker 1

We are incredibly happy with the results. We can point to several key drivers of the momentum we saw in the Q2. First, we are seeing consistent growth looking at month over month new patient starts. We are reaching patients in earlier lines of therapy, So we're still at the front end of reaching the 17,000 patients we believe we can address with ZipGap. The task before us is to shift into earlier lines of treatment and overcome neurologist inertia.

Speaker 1

This will take time as neurologists gain deeper experience with Zivgut and will also require assistance from our field teams. We have seen consistent prescriber growth, both in terms of breadth and depth. We now have more than 2,100 prescribers in the U. S. And a greater percentage of them each quarter are moving beyond that first script and putting additional patients on treatment.

Speaker 1

We also think that VIVGAR HYTULO will help with this shift. Slide 11. We're now a couple of weeks into the Hytulo launch and have received our first scripts and shipped our first vials. We'll save specifics Q3 call, but I was very impressed by the team's readiness at the time of approval to get drug into the channel and to patients as quickly as possible. Our goal with Ziff Guard Hi Trullo is to honor our long term commitment to the gMG community with a second product option, now subcutaneous, and also to drive growth into earlier line patients by simplifying or democratizing the treatment of gMG.

Speaker 1

We believe our positioning should be first line after oral therapy and that will require further expansion into community practices. The diagnosis and treatment of MG patients can be complex and can be associated with a trial and error approach or a battery of tests. Current treatment options often come with safety and tolerability challenges, which require the patient to decide between symptom control, emerging side effects or long term safety concerns. This all requires close management from a gMG specialist, which is why patients are often referred to specialist centers. Our conviction is that with the efficacy of DIVGOT, established safety and tolerability and now 30 to 90 second injection with Hytrullo, We can expand usage deeper into the community of patients and prescribers.

Speaker 1

And the more experienced community prescribers gain, The more expert they will become at diagnose EMG and treating with VIVKA. Now that we have the results of the ADHEAR trial, we can take many of the same themes that we've seen with gMG and apply them to CIDP as well. The CIDP community has been waiting for innovation for over 30 years and with these data, We believe we can bring transformational change to patients. For me, one of the most compelling data points from ADHEA was the 91% of eligible patients who roll over into the open label extension study, including patients who relapsed in Part B and were given the option to go back to their prior therapy or to stay on VIVGAT. We see this as a good indication that the patient experience with VIVVAT is different.

Speaker 1

We know that there will also be unique challenges associated with CIDP market based on the comfort level and loyalty associated with current treatment. But we also see the opportunity to raise the bar on what a treatment can offer taking into account the full patient experience, efficacy, safety and the burden associated with administration. Slide 12. Before I wrap up and turn the call back to Tim, I want to shift to the progress we're making with our global geographic expansion. We had 3 key updates since our last call and still more ahead this year, demonstrating the speed at which we are executing on our global launch strategy.

Speaker 1

First, we received approval for VIVGAR in China through our partner Zai Lab at the end of June. This means we will be eligible to apply to the NRDL in 2024, which is important from a timing perspective, because it will open the opportunity to patients who are not privately insured. Zai also submitted the BLA for subcu escargot and it was accepted. Earlier in July, we also We completed reimbursement negotiations in Italy, marking the 2nd country within Europe to have officially launched BibbDot. I'm incredibly proud of the team expanding access for Italian patients 11 months after European approval.

Speaker 1

And last, we finalized the commercial and distribution agreement in South Korea with Handok, a team with a great track record as a commercial partner. Similar to our other distribution agreements, Handoff will take the lead on all regulatory and commercial activities associated with VIBDA in South Korea. Slide 13. I want to close with the why behind all of our expansion strategies, which are the patients who are living with severe autoimmune disease. On the heels of another strong quarter and a positive outcome in CIDP, We are more motivated than ever to drive a paradigm shift in how autoimmune is treated.

Speaker 1

Current autoimmune treatments come with many trade offs And now is the opportunity for transformative change. We want to take the full patient experience into account, efficacy, safety and treatment burden and give patients more days when they're not reminded of their disease. It's the right time for meaningful change and we believe we have a unique medicine, patient centric strategy and a talented team to deliver. I'll hand it back to Ken. Thank you, Karen.

Speaker 2

Slide 14. Coming back to where we started, which is to look back at the plans we set forth At the start of the year, we have delivered on our promise to execute and drive sustained growth across our business. I'm incredibly proud of the team for these achievements. They do not come without a lot of hard work. We continue to show In all that we do, our commitment to bold innovation and execution on behalf of patients.

Speaker 2

Our work is not done and we still have a lot to look forward to by end of 2023 on our path to transformative treatment of severe autoimmune diseases.

Operator

Thank you. We will now begin the question and answer session. Your first question comes from the line of Tazeen Ahmad with Bank of America. Please go ahead.

Speaker 3

Hi, good morning, guys. Thanks for taking my question. Maybe I just wanted to focus on 1 of Two data catalysts that you have in the 4th quarter, specifically for the Pemphigus readout. Maybe, Tim, can you frame for us what to In terms of the top line results that you're going to be presenting and in terms of feedback that you've gotten from physicians, what would be considered good data? Thanks.

Speaker 2

Good morning, Katina, and thanks for being with us today. And thank you for your Pampers' question. It's from the key catalyst, I think, of the remaining part of the year. In Pentagas, the primary endpoint is mainly constituted around showing a statistically significant delta versus placebo. So we're testing efgartigimod versus placebo in a background of steroid tapering.

Speaker 2

So the idea is to show The significant win on the primary endpoint on an endpoint which is complete remission on minimum therapy that means a minimum dose of steroids. In the key secondary endpoints, we're going to unpack elements which are really critical to patients. That means to know What is the extent to which we can taper steroids? That's very important. That's what patients care about.

Speaker 2

And also what is the To speak to disease control that means stopping the formation of new lesions and then followed by the closing of new lesions. So This is in a nutshell the key endpoints, blending what the regulators care about and what patients care about. Thanks for the question.

Operator

Your next question comes from the line of Derek Archila with Wells Fargo. Please go ahead.

Speaker 4

Hey, good morning and thanks for taking the questions. I actually have 2 brief questions, if I may. So first, I was hoping you could talk a little bit about the sales momentum for Bibgard. You talked about some of the ex U. S.

Speaker 4

Geos coming online. You've launched Tytrulow now and this push into earlier lines. So I guess as you think about the second half, which of these do you think is going to be the biggest incremental contributor to sales growth? And then the second question, you stated in the PR, you're going to start the TED trial for VIVGAR. Given what we're seeing with TEPEZ's recent performance in the thyroid eye disease Mark, I mean, has your optimism around that commercial opportunity changed at all?

Speaker 4

And when will you communicate the full details of that trial design? Thanks.

Speaker 2

Thank you, Derek, and thanks for being with us today. I'll give you question 1a to Karen, and then I will take your question 1b, okay?

Speaker 1

Thanks, Tim. Thanks for the question. And as you say, I think we continue to see strong momentum and I'd say consistent momentum In the launch of ViVicar and actually I would say that in the second half of the year, we'll Continue to see that consistency across all of the factors that you mentioned. I think we continue to see that we're penetrating earlier lines of Treatment and expanding that prescriber base. I see no reason why that won't continue through the year.

Speaker 1

We have pricing reimbursement discussions ongoing in Europe And so we believe those will continue through the year and the Hytulo early indication that we have positive feedback and that will also contribute to our growth. So I think one of the strengths that we have We are driving growth across all of those dimensions and I think that should help us to maintain some consistent momentum.

Speaker 2

Thank you, Ken. And on the TD question, Vivek, I just invite you to be a bit patient. We are on track to start the trial. Once we're there, we're not doing a fair trial design. I think we can also start to talk a little bit about how we think about positioning.

Speaker 2

We do believe that is an IgG driven disease. We do believe there's establishment of a linear correlation between the type of your autoantibody on the one hand and your clinical symptoms on the other hand. And I think Castigram is well placed to shine on all three dimensions of efficacy, safety and convenience. So Stay tuned on the TAD story. Thank you.

Speaker 2

Thanks, Tim.

Operator

Your next question comes from the line of Yaron Werber with TD Cowen. Please go ahead.

Speaker 5

Great. Thanks for taking my question and Congrats on a great quarter. Maybe as we look into the BT gono go decision early next year, it's in Q1 now, Can you give us any parameters at all as to what you're looking for from a powering perspective in those first 30 or 40 patients? And What is the actual gono go metric? Thank you.

Speaker 2

Thank you, Yaron. I mean the best analogy to draw is actually I want you with the CIDP trial. So I think we need to see a minimum level of response in these first forty patients, This gives us conviction that this is not a placebo response we're seeing and that actually which confirms that this is a true IgG mediated disease as So think of a seamless Phase II, Phase III where we need to meet a minimum criterion from an efficacy point of view. Of course, we will look at the totality of the data including safety, but it's a seamless Phase twothree design in order not to lose time, in order not to have wide space Between Phase 2 and Phase 3, you know that we have conviction in BP. We think that Pampers actually serves to a certain extent as a de risking step I'm for Buddhuspensionborg and we believe based on all the homework we did, this is a truly IgG genetic disease.

Speaker 2

Very exciting indication actually to focus on, a 35 under medical needs, very little innovation And the safety profile I think of that graph will turn out to be even more important BP than it will be in front of us. So we're very much looking forward to that decision for me. Thank you.

Speaker 5

And Tim, is it just based on placebo? Or are you Thinking a little bit on the corollaries of some of the competition out there, there isn't certainly a ton, but some of it is showing robust responses. So are you sort of You have that in your sights as well into the Go, No Go decision. Thank you.

Speaker 2

Go, No Go will basically look at the initial Make sure it's emerging. I mean we're not positioned there to look at long term effects of the drug. Maybe you're alluding to the long term clinical benefit We have seen in some of the Pampers patients in Phase II where we actually did change the course of disease. These are data which we will also collect In the DP trial, but they will not be part of the go to go decision points. Okay?

Speaker 5

Thank you.

Speaker 2

Thank you.

Operator

Your next question comes from the line of Danielle Brill with Raymond James. Please go ahead.

Speaker 3

Hi, guys. Good morning. Thanks so much for the question. I have a follow-up on the growth outlook for VIBGYART in the second half. I'm just curious, from a modeling perspective, how we should think about the growth momentum.

Speaker 3

We've been seeing a consistent mid-twenty percent quarter Over quarter growth in the U. S, is it fair to model that moving forward? And what kind of impact, if any, should we expect from the recent launch of UCB's at CRM. Thank you.

Speaker 2

Hi, Danielle. It's Karl here. Thank you for your question. I mean, I think that As the dollar numbers become bigger, we cannot expect a continued 20% growth quarter over quarter, of course. Also with our TRULO launch, I think it's really important that we are patient while we get those payer contracts It will take a quarter or 2 to get back.

Speaker 2

Remember, that's how long it took us with VIVCARD 18 months ago. In terms of competition, yes, we now do have competition in terms of ROSA. And of course, I think there comes a daily C5 later on. So the market is getting more competitive. And also, We have another C5 who has started a TV campaign and it's getting a little bit more crowded.

Speaker 2

Overall, of course, we still expect Thank you. Thank you for your question.

Operator

Your next question comes from the line of Thomas Smith with Leerink Partners. Please go ahead.

Speaker 6

Hey, guys. Good morning. Thanks for taking the questions. And let me add my congrats on the great quarter. Just wanted to ask a little bit of a high level question.

Speaker 6

You have a really strong balance sheet, pretty incredible launch momentum in Gmg. Can Just comment on how you're thinking about strategic priorities here, given your significant cash balance, how you're thinking about investment between commercial infrastructure For VIVGARD indication expansion or early pipeline development, are you considering any other BD opportunities? Thanks.

Speaker 2

Thomas, thank you for the question and thank you for being with us today. The answer is that we're going to fire from all cylinders. So The fact that we have such a strong cash balance puts us in a position to fully invest in the commercial dimension. We're leaving nothing on the table of the immense opportunity we have in front of us. We do have an incredible pipeline.

Speaker 2

We have more than 60 clinical trials running now. There's a massive volume where we feel a sense of urgency to move forward with innovative trial designs unpacking The clinical potential of our drugs, you did see that ARGX-one hundred and seventeen has proven to be a drug with the first efficacy data in MMN. And argenx for 2019 is swiftly dose escalating to the Phase I study, so a lot to come. And we have a wave of innovation, which is Progressing through the clinical towards the clinic. So firing from all cylinders.

Speaker 2

That also means that we're in a position of strength. I think there's a ton of innovation which we're bringing forward from our platform through our pipeline and into the marketplace. We are of course always externally focused looking carefully for opportunity which we like, that we're entrepreneurial as a company. We also do not suffer from the noninvented knee syndrome. So if and when we see things we fit and we like, we will without any doubt enter into Partnership conversations.

Speaker 2

Remember that our discovery model, the IIP program, the Immunology Innovation program by definition It's a partnership model. So partnering is in the DNA of the company. Thank you for the question.

Operator

Your next question comes from the line of Myles Minter with William Blair. Please go ahead.

Speaker 7

Hey, just a question on the potential self administration if you've got Hytroul. Did you have those conversations during labeling when the product Prove myasthenia gravis and what it would take data wise to get self administration in the label, Would the ability to potentially self administer in the open label extension effort here be sufficient to get A self administration option on that label for both CIDP and myasthenia gravis. Thanks.

Speaker 2

Hey, Matt. Thanks for being with us today and thank you for the question. Just a quick recap on the MG situation. Thank you for the 1st generation subcu. We're seeing a combination of relative complex interface, a few steps you need to do We're going to know specifics of the disease indication like double vision, limited dexterity, muscle weakness and the combination of the 2 Maybe probably too high barriers for self administration in the United States.

Speaker 2

Remember that the Discussions are still ongoing in Japan and in Europe on the label and we need to see what they will deliver. And it's too early to get ahead of ourselves I think for CIDP. We need to go for a pre BRA meeting with the FDA, calibrating expectations. We need to prepare the SBL refinements submitted. And as part of these conversations, I think we will also tackle the topic of self administration.

Speaker 2

Importantly, you need to know that we're working very hard on the 2nd generation of the subcutaneous product presentation of efgartigimod. That is a brief syringe. That is a simplification of the use of interface. And I think there we have again a real short on goal to go to self administration. But stay tuned step by step.

Speaker 2

I think the current and we've got hydrolyph is already a significant step Forward helping us to penetrate our market because it's decoupling the patient from the infusion check. So we're avoiding infusion chain bottlenecks and we're simplifying access for the patient to the drug because now any HCP can administer the drug anywhere. Thank you for the question.

Speaker 7

Thanks for the questions. Congrats on the quarter.

Operator

Your next Question comes from the line of Yatin Suneja with Guggenheim Partners. Please go ahead.

Speaker 8

Thank you for taking my question. Just two quick ones for me. In terms of the patient mix currently on the drug, can you just comment Where are we in terms of refractory patient? And what percentage of the patients are getting retreatment and at what frequency? And then the second question is thoughts on providing revenue guidance now that you have over a year worth of experience with the launch?

Speaker 8

Thanks.

Speaker 2

Yes. Thank you, Jatin, and thanks for being with us. And I will give the question on percentage of infected patients And numbers on the achievements add to Karen and then maybe Carl can very quickly comment on your revenue question. Thank you.

Speaker 1

Yes. Thanks for the question. So I would say the patient mix and in particular refractory, we are at the early stages of launch, we were seeing a lot High usage in the refractory patients when physicians are just starting to get experience with the medicine. And what we've seen over Time is consistent movement into those earlier lines of treatment and we continue to see that shift and that momentum as we execute on our plans. In terms of the retreatment of frequency, what you can imagine is, of course, it's a bell curve.

Speaker 1

Some patients are it's individualized dosing. So some are very frequent, some are less frequent. But what we see in the data is that it's right in line with what our assumptions were and what we provided before, which is around 5 cycles For our patients, that has significantly changed.

Speaker 2

And Jatin, it's Kalia. Thank you for your question. In terms Finance guidance, I mean, we're not ready yet. I think there are still too many variables. The German price will be really important.

Speaker 2

The China launch, we're only just launching the sub So I think we need to see a few quarters of that, and then we'll get back to you on finance guidance. Thank you. Thanks, Charlie.

Operator

Your next question comes from the line of Vikram Parodi with Morgan Stanley. Please go ahead.

Speaker 9

Hi, good morning. Thanks for taking our question. So we just had one on the recent launch of subcu VIVGAR into myasthenia gravis. Understanding it's early, but is there any color you could provide on the demand trends you're seeing from prescribers and patients towards subcu versus IV? And if you've been able to glean any trends at this phase of the launch on the types of patients that are leaning more towards subcu.

Speaker 9

And on that same topic, Are you able to provide any commentary at this point on how you're finding early receptivity towards the subcu option from payers? Thanks.

Speaker 2

Yes. Thank you, Vikram. I'm going to try and limit the question to one. Maybe Karen, can you comment on subcu launch dynamics And any plans which we will see. Thank you.

Speaker 1

Yes, absolutely. I mean it's really too early to tell that Any of the details that you asked about, but what we are seeing, we believe we have strong value proposition and the outlook is strong. We are getting early positive signals, those are verbal from when we're out speaking with urologists and the feedback we're getting from our field force. Obviously, it takes a while, as Karl said, to get the payer policies in place. We have had one policy that's been put in place, but we need to continue to work on those.

Speaker 1

So that impacts The uptake right at this moment, but we are seeing enrollments come in. We are seeing positive feedback and we strongly believe and still have the conviction that The 30 to 90 seconds is a real advantage for patients and it will really start to open the market up for community neurologists and we'll keep you updated as we learn more.

Speaker 2

Thank you, Karen. And thank you, Vikram, for the questions. Thank you.

Speaker 9

Thank you.

Operator

Your next question comes from the line of Allison Bratzel with Piper Sandler. Please go ahead.

Speaker 10

Hey, good morning and thanks for taking my question. Maybe just a follow-up on an earlier question on the prefilled syringe. Could you just remind us of the expected cadence of updates On the subcu prefilled syringe, it does look like you've been enrolling a Phase 1 bioequivalence trial in healthy volunteers For the PFS for a few months based on clinicaltrials dot gov, just curious though what other clinical work would need to be carried out for that to be fileable? And If you could just outline the expected data flow and development path there, that would be helpful. Thank you.

Speaker 2

Hey, Allison. Thank you for being with us today. And I'm going to comment on the question. So there are a couple of data streams, which need to be generated in parallel In order to file for the PFSO, we have the bioequivalence study, which you correctly call out to be ongoing. We have the human factor studies you need to complete.

Speaker 2

Then of course, there's a whole invisible train of CMC work, which needs to happen with our CDMOs. So it's when these 3 come together and generate all the data including stability data that you're actually ready to submit. And we will be getting more granular on time line Over the course of next year, the treatment syringe really is a priority for us because we think it's going to be an enabler of the long term rollout of TypTap and of course a key enabler for sales administration. So stay tuned

Operator

Your next Question comes from the line of Alex Thompson with Stifel. Please go ahead.

Speaker 4

I guess I had a

Speaker 6

quick follow-up on pemphigus. As we think about steroid tapering in the drug arm, What does good look like there? And in your view, is it no steroid? Or what is a good dose of steroids in these patients look like to be clinically meaningful? Thanks.

Speaker 2

Hey Alex, thanks for that question. And just to get it clear, it's not just on the drug arm that we do steroid tapering. We actually test active versus placebo. And in both arms, the background is stereotyping protocol. So all patients start on the same amount of steroids.

Speaker 2

And then with a predefined protocol, you need to step down in your steroid use. And the endpoint actually is a complete remission that means that you're free of skin lesions on the minimum dose of steroids and as a minimum dose the way it is used in Pampacuss. So it's a very stringent endpoint to reach and actually you need to be at 8 weeks in SIER on minimum dose of steroids in order to effectively hit the endpoint. So the bar is very high. We cannot expect to have a very high response rate in active, but it's really designed to push placebo as close to 0 as possible.

Speaker 2

So let's look for the delta, The statistically significant delta between active and placebo and that will mean fixed fee in the primary endpoint. In the second hand points, as I said, we will unpack all the information, which actually is very relevant for patients In terms of total cumulative use of steroids and the ability to effectively taper meaningfully and then of course speed to disease control And absence of formation of new lesions. So that is the full extent, which we will be able to unpack in the top line data.

Speaker 11

Thanks for the question.

Operator

Your next question comes from the line of Akash Tewari with Jefferies. Please go ahead.

Speaker 3

Hi, this is Amy on for Akash. Thanks so much for taking our question. Just 2 from us. First, on long COVID POTS, do you envision efgartigimod to be given chronically as long term maintenance or would it be more of a one And then finally, on your expectations, could you revisit your expectations for when you'll reach profitability? Thanks so much.

Speaker 2

Thank you, Ingram. And again, this is 2 questions for the product, one right. So but I'll give the second question very quickly to Karl. But let me comment on POTS first. This is a key question in long COVID POTS.

Speaker 2

So if my colleague and Chief Medical Officer, Luke would be here in the call. He would say, look, we need to learn whether it's sufficient to cleanse the body in a one off Treatment cycle with Typgar should then be symptom free or indeed would you need to go into more chronic dosing in long COVID-nineteen POTS To basically effectively take care of the patients. This is one of the key questions we need to address next to the biology question in this Phase II proof of concept trial. So stay tuned. We're all curious to learn about that.

Speaker 2

And Karl, would you mind briefly commenting on question 1b? Thank you, Amy. Yes, apart I mean, we're not going to comment on that in terms of timing. What I will say is that we are a sustainable company. We stand on our own legs.

Speaker 2

And yes, I think that's all we will say for now. Thank you.

Operator

Your next My apologies. Your next question comes from the line of Rehan Sharma with Goldman Sachs. Please go ahead.

Speaker 12

Hi, it's Rajan. Thanks for taking the question. Just on CIDP and post the ADEA data, Is it safe to assume that you're going to be positioning Vivint as a first line option there to compete directly with IG? And then what kind of additional work do you think you need to do to convince the treating community that there is more pathogenic high G activity in the disease than they may have

Speaker 2

Yes, thanks for this question. I think that will be just like for MG, a lot of education going on, You know, educating our audience on the fact this is an IgG mediated disease based on now data in hand. I think you correctly call out that the way the trial was designed and the way the data handouts is that there's no need to niche this product into The Riflexi line or you know a line of IVIg failures. I think the drug was equally effective in patients Which were on steroids and background therapy, which had been on IVIg or SCIg background therapy All of which were either newly diagnosed or even in the last 6 months. So equal efficacy, equal widely responded to the drug across the board.

Speaker 2

So we do not see any reason to actually go and actively niche ourselves in what we think is a sizable opportunity. Thanks for the question.

Operator

Your next question comes from the line of Joon Lee of Truist Securities. Please go ahead.

Speaker 6

Hey, congrats on the progress and thanks for taking our questions. With CIDP data on hand, how are you thinking about commercial competitiveness Ness, as part of Jimad versus IVIG and CIBP. And at steady state, how do you think how do you see the breakdown of IVIG versus FcRn Targeted Products and CMVP. Thank you.

Speaker 1

Yes. Thanks for the question and I can take that. It's a little bit too early for us to comment on sort of IVIG versus FCRM market share, if you will. We still need to continue to do the work up on the commercialization plan. But what I can say to your first question, we see a really strong When we look at the holistic picture of VIVVAT in CIDP, when you think about The efficacy and Tim just talked about the strength of the data there.

Speaker 1

The safety and tolerability I think was incredibly impressive. Weekly it was weekly dosing And we saw that the safety profile continues to hold up, the tolerability is really clear. And then obviously, the 30 to 90 second injection. So we believe that we have a full package, that we'll be able to compete really well in the market, and we have conviction in the product and we'll do the work up and share some of our thoughts around What that might look like versus IVIG in the coming months.

Speaker 2

Thank you.

Operator

Your next question comes from the line of Samantha Simenkow with Citigroup. Please go ahead.

Speaker 13

Hi, good morning and thanks for taking my question. Just a follow-up on Pemphigus, assuming the data It's positive on the primary and some of the secondary endpoints and the readout in the Q4. How do you envision Vistar fitting into the treatment paradigm? Curious with your conversations with physicians and how you design the trial, and if there are any barriers you would have to overcome with current treatment patterns? Thank you.

Speaker 2

Yes. Thank you, Sanjay, for this question. I'm excited about our opportunity in the autoimmune listening disease space. The interesting thing about centrifuges is that actually it's an area which is wide open. So the only treatment options these patients have today is actually steroids, which they hit with a passion.

Speaker 2

And then rituximab, Which has a slow onset of action, significant side effects and actually a real world efficacy Which is significantly lower than what they have shown in the clinical trials. So if you now look at the Phase 2 clinical data for efgartigimod with a Very fast onset of action, a very high response rate and an unexpected durability. It's not difficult to see that you can start to drive your wedge And so I think there's a unique opportunity here to go and rethink the treatment paradigm, But let's not get ahead of ourselves. Let's wait for the Phase 3 data because they will inform us about the best way to position the drug going forward. We're excited about the opportunity and thank you for the question.

Operator

Your next question comes from the line of Joel Beatty of Baird. Please go ahead.

Speaker 11

Hi. Thanks for taking the question. What's the for the patients starting on Hytrullo, what's the mix of patients you're seeing switching from IV Vipgar

Speaker 1

Sir, I think I heard your question that it was around switches from Hytrullo or from VIVGAR to Hytrullo and it's just too early to tell. We haven't seen the data on that yet, but I know, we're getting strong feedback on the value proposition of Hytrua.

Speaker 2

Joanne, it would be disappointing why this would just be a switch dynamic, like Karen?

Speaker 1

Yes, absolutely. I can comment on that. I think we've commented on that before. The The strategy here is not that we're switching from VIVVAT to VIVVAT HITRULA, but rather we're trying to open up access for patients in the community And elsewhere where the 30 to 92nd injection is a better option for fitting into their lives. So, yes.

Speaker 2

Thank you, Joel. Thanks for the question.

Operator

Your next question comes from the line of Douglas Tsao with H. C. Wainwright. Please go ahead.

Speaker 6

Hi, good morning. Thanks for taking the questions. Just following up on Hytrolo, you've obviously spoken about the opportunity now to Yes, into the community more. I'm just curious as you've been in the field more, what percentage of the market Does this now open up, meaning how many patients were sort of community based and didn't really have great access to VIVGAR for whatever reason.

Speaker 1

Yes, look, it's a great question. I mean, I think the we still see the addressable market VISGARD and VISGARD HiTrullo as that 17,000 that we shared before. And we think HiTrullo allows us to Really penetrate more into the community, get closer to being used after the oral and so really opens up that segment of the market. But the way that I would think about it is that the addressable market is $17,000 and we're on the front end of that as we at this point in March.

Speaker 6

Okay, great. Thank you.

Speaker 2

Thanks, Andrew. Thanks.

Operator

Your next question comes from the line of Leland Gershell with Oppenheimer. Please go ahead.

Speaker 11

Hey, good morning. Thanks for taking my questions. With respect to emphysema purport, as you look forward to the Phase 2 second cohort data in MMED next year, Is there a particular bar you're looking to meet or is that the data merely to inform next steps? And also want to ask with respect to that asset, are you That came to start either or both in the POC studies in DM or kidney this year. Thank you.

Speaker 2

Yes, thank you. And I'm impressed by how you pronounce the name of AMPA. I mean, I would like to record it. So EMPART is an exciting molecule. The Phase II trial is designed to generate PKPD data and efficacy and safety data to inform our model.

Speaker 2

Ideally, the 2 dose cohorts give us sufficient information to triangulate What we have said publicly is that on dose cohort number 1, we not only passed an independent And data review committee is a blessing from a safety and tolerability point of view, but I think we also saw convincing efficacy Allowing us to venture into dose cohort 2. The idea is that we see a separation between the 2 dose cohorts. So the PKPD model is Fully informed that I believe that based on the efficacy we have seen already, we think we have a drug in M and M. The remaining question is what is the dose going to be for Phase 3 continued document, of course, safety. And yes, this is a 500 product opportunity.

Speaker 2

MMA is very exciting as an indication. But for the other 2 indications, which we're lining up are equally exciting. We are on track to start both trials as we line up in the press release. So soon this molecule will be playing already in 3 indications and we think there is more to unpack in terms of opportunity. So Please stay tuned.

Speaker 2

Thank you for the question.

Operator

Your next question comes from the line of Tian Tan of UBS. Please go ahead.

Speaker 14

Hi. Thank you for taking my question. So you have quite a few catalysts coming up, BP, Pamphibos, TD, And there are many more indications in the pipeline. I'm just wondering with so many indications, is there anyone that You would like to highlight, I think, if there's anyone that's higher risk than others or if there's anyone that if you make it, that will be much bigger than others in terms of Commercial Opportunities. Yes, anyone that you would like to highlight in particular?

Speaker 14

Thank you.

Speaker 2

Thank you. Yes, that's a really challenging question to me because I'm equally excited about all the indications. And it's very difficult to tell you which one is going to be ultimately the biggest one. But I know that when I sum them up, this is going to be in its totality A very sizable pipeline in the products. And let me quickly call out ITP will be on track to get our marketing authorization in Japan.

Speaker 2

So soon we will have Two indications on the market. And then I'm very excited about our myositis trial, which is very innovative. It's a basket trial, the first one Verigo has a 3 distinct subpopulation in myositis where these people are in very high unmet medical needs It's not really effective treatment options at all. So we're really looking forward to the MYZYTUS trial and I'm paying close attention to it. I'm very excited about the universe of opportunity in the autoimmune listing diseases with both Pemphigus and bulluspendergoy progressing very well.

Speaker 2

Then of course, we have a whole 3rd wave of indications where frankly speaking, we're not taking on more biology risks. We're still in the sweet spot of the drug With a strong conviction on biology, clear and feasibility from a clinical trial execution point of view And there will be a commercial opportunity to transform the lives of these patients. So this is the universal opportunity which we're approaching. Thank you for the question.

Operator

As there are no further questions at this time, this will conclude today's conference call. Thank you for

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