argenx SE

XBRU:ARGX

Earnings Call Transcript 2017-Q4

[Operator]

Welcome to argenx Fourth Quarter 2017 Business Update and Full Year Financial Results Conference Call. [Operator Instructions] I would now like to introduce Joke Comijn, Corporate Communications Manager at argenx. Please go ahead.

[Joke Comijn, Corporate Communications Manager]

Good afternoon, everybody. A press release with the argenx fourth quarter business update and full year 2017 financial results became available at 7:00 a.m. Central European Time today and can be found on our News and Events section on our website.Before we start, I'd like to go to Slide 2 to remind you that forward-looking statements may be presented during this teleconference. They may include statements about our future expectations, clinical development, regulatory timelines, the potential success of our product candidates, financial projections and upcoming milestones. Actual results may differ materially from those indicated in the statements. Argenx is not under any obligation to update statements regarding the future or to confirm those statements in relation to actual results, unless this is required by law. I'm joined on the call today by Tim Van Hauwermeiren, CEO of argenx; and Eric Castaldi, CFO. On Slide 3, you will see a brief agenda for the call. Tim will highlight recent news, including our latest public offering and provide an update on our clinical programs and our collaborations. Eric will then provide financial results for the year and finally, Tim will lay out upcoming milestones before we turn the call over for Q&A. I would now like to introduce Tim Van Hauwermeiren, CEO of argenx.

[Tim Van Hauwermeiren, CEO & Executive Director]

Thank you, Joke, and warm welcome to everyone. Throughout 2017, argenx achieved numerous corporate and clinical milestones, bringing us steps closer towards our ultimate goal of becoming a fully integrated commercial stage company that delivers transformative antibody-based therapeutics to patients with severe autoimmune diseases and cancer. On Slide 4, you'll see that our ability to generate highly differentiated antibody molecules stems from the fact that we address novel or complex targets using our cutting-edge antibody discovery technologies. Then, our Innovative Access Program allows us to work in close collaboration with leading academic groups and innovative start-ups, which possess deep mastery of the target biology. We focus on applications with high unmet needs where we can take our programs through clinical proof-of-concepts and possibly registration ourselves based on the proofed endpoints. We have been able to mature a unique and sustainable pipeline with 2017 certainly being our most successful year-to-date positioning us for a productive and catalyst-rich 2018. Slide 5. In May 2017, we completed a NASDAQ IPO, raising gross proceeds of approximately $115 million. On the use of our encouraging clinical data in December 2017, we closed an oversubscribed public offering with gross proceeds of approximately $266 million. Through this race, we were able to grow our Blue Chip long-term institutional investor base that is closely aligned with us on our business plan going forward. We also now have the ability to aggressively advance our lead candidates as well as fund our earlier-stage pipeline and expand our team, particularly in the United States, where we've recently opened our state side headquarters in Boston. Slide 6. In the second half of 2017, there were significant updates from our most advanced candidates, ARGX-113 or efgartigimod in the development for rare autoimmune diseases including: the initiation of our Phase II trial in pemphigus vulgaris; the initiation of our Phase I trial to evaluate PK-PD safety and tolerability of a subcutaneous product; and most notably, the positive results of our Phase II trial in myasthenia gravis, which we announced in December. As shown in Slide 7, ARGX-113 is an IgG1 FC-fragment exploiting the natural binding sites between the IgG FC tail and the recycling receptor FcRn equipped with our proprietary ABDEG mutations. Given that MG is one of the autoimmune diseases where we best understand the role of pathogenic autoantibodies of IgG type in driving the disease and the severe limitations of current treatment options, there was a clear rationale to pursue MG as our first indication. Slide 8 shows how we assess the treated patients. The primary endpoints of the study were safety and tolerability and secondary endpoints included improvement of the disease core from baseline assessed by 4 recognized MG scales. And in addition, we monitor total IgG, pathogenic IgGs, PK and immunogenicity. Let's move to Slide 9. To reiterate our findings, the data showed a tolerability profile that was consistent with the findings from our Phase I healthy volunteer trial. Most treatment emergent adverse events were mild, and there were no severe adverse events reported. We observed an 83% response rate in the 113-treated patient population. Even in the small sampling of 24 patients, 12 per study arm, we observed a strong efficacy signal in the 113-treatment group with 75% of patients achieving a lasting response versus only 25% of the patients in the placebo group, as shown on Slide 10. Importantly, we saw a clear separation in clinical improvement between the treatment group and placebo after just one week or one dose. Additionally, the duration of the response lasting at least 6 weeks further supports the long-lasting PD effect of our drug candidates. To contextualize our findings, I would like to point out that this overall response rate is in-line with what has been reported for plasmapheresis to gold standard therapy in MG, and that our sustained response compares favorably with reported 2 to 4 weeks for plasmapheresis and all of this with the safety and convenience profile, which is leaving plasmapheresis far behind. Slide 11. MG is a serious autoimmune condition, causing periodic muscle weaknesses that can progressively worsen and even be life-threatening. It is an orphan disease with an estimated 64,000 patients diagnosed in the U.S., of which an estimated 55,000 have generalized MG, for which current treatment options remain very limited. Aside from the recently approved Soliris, this space has not seen any innovation in the past 40 years and current therapy typically consists of corticosteroids, immunosuppressants and IV AngioPlex. In general, these options offer inadequate efficacy, slow onset of action and significant side effects. We are incredibly pleased, not only that ARGX-113 showed a strong tolerability profile, but that the data also demonstrate a rapid and sustained benefit in disease core, which was found to correlate very well with reduction in pathogenic IgG levels. We are committed to bringing this drug forward to commercialization as fast as possible and plan to initiate Phase III development before the end of 2018. Additionally, in April of this year, we plan to present the full MG data sets including specific IgG reduction levels at the American Academy of Neurology annual meeting. Overall, as presented on Slide 12, these data were incredibly important in underscoring the mechanism of action of ARGX-113, validating our scientific approach and, furthermore, our rationale in choosing autoimmune indications mediated by pathogenic IgGs. These data supported our findings from our preclinical and healthy volunteer studies showing rapid onset of action of the drug as well as strong duration of response. Furthermore, these data allow us to move forward into our other ARGX-113 trials with a stronger sense of how the drug really works. There are many IgG-mediated diseases that may benefit from the degradation of circulating autoantibodies, meaning we have the opportunity to create a pipeline within a product around several potential indications, as is shown in Slide 13. We had a three-pronged rationale to selecting our first indications of study. First, based on the ability of ARGX-113 to reduce levels of circulating IgGs, we looked at indications where pathogenic IgG levels predominantly mediate disease. We then considered indications we believe we could confidently bring forward ourselves, namely orphan indications that would require smaller, more focused trials and which we believed had clear regulatory paths. Finally, we selected indications for which clinical proof-of-concept will be strong enough to have an impact in adjacent indications. With these criteria in mind and based on significant unmet medical needs, we decided to focus on myasthenia gravis, primary adult immune thrombocytopenia and pemphigus vulgaris. We believe there are other autoimmune diseases that may benefit from this mechanism of action, and we plan to expand our development efforts in other diseases mediated by pathogenic IgG autoantibodies. Today, we are nearing the completion of patient enrollment in our second Phase II trial of ARGX-113 in immune thrombocytopenia. As a reminder, presented on Slide 14, the study is a double-blind placebo-controlled study enrolling up to 36 ITP patients with platelet counts lower than 30 million per milliliter. ARGX-113 will be dosed on top of current standard of care, corticosteroids and/or immunomodulatory agents and/or TPO receptor agonists. The primary endpoints of the trial are safety and tolerability, and secondary endpoints include effect on platelet counts and use of rescue treatments and an assessment of PK-PD as well as immunogenicity. Today, we have 2 updates to the ITP program in the form of amendments to the Phase II study. Slide 15. First, we elected to extend the follow-up phase, which follows the treatment phase from 8 weeks to 21 weeks. We also added a 1-year open-label extension study to allow relapsing patients the option to be evaluated on the high dose of the drug candidates at 10 milligram per kilogram. Our goal with these amendments is to gain as much patient exposure to the drug candidate as we can, and also ensure we understand its full duration of response in order to best meet patient needs. We look forward to our top line Phase II data readout in ITP in the second half of 2018. This is our beachhead indication to other severe blood disorders, and a positive result in ITP could open up an important development opportunity for ARGX-113 and our pipeline within a product approach. Also in the second half of this year, we plan to announce interim clinical results from our third Phase II trial of ARGX-113 in pemphigus vulgaris, an indication we carefully selected based on its severity, correlating directly to pathogenic IgG levels, as well as the large unmet need in the space. As shown on Slide 16, this study is an open-label and noncontrolled study and will enroll up to 12 mild-to-moderate disease patients, who are either newly diagnosed or relapsing. With PV, we expect to study the potential of ARGX-113 and its autoantibody clearance mechanism to treat a range of skin blistering diseases beyond this beachhead indication, including amongst others bullous pemphigoid. And to wrap up 2018 milestones for ARGX- 113, we plan to present top line data from our Phase I healthy volunteer study with a subcutaneous formulation, which has the potential to provide a significant advancement in convenience of treatment for patients with severe autoimmune conditions requiring long-term treatment. I'd like to turn now to our lead oncology candidate, ARGX-110 in the next Slide 17. During the ASH annual meeting last December, we hosted a KLL workshop to announce updates from our Phase I/II clinical trials in acute myeloid leukemia and cutaneous T-cell lymphoma. Data from the Phase I/II clinical trial for ARGX-110 in combination with azacytidine, in 6 newly diagnosed elderly AML patients unfit for intensive chemotherapy, showed encouraging signs of clinical activity including: complete remission in 3 out of 6 patients, complete remission with incomplete blood count recovery in 1 out of 6 patients and partial response from 2 out of 6 patients. One of the patients who achieved a complete remission bridged to allergenic stem cell transplant after 5 cycles. The preliminary data from the first set of patients, as shown on Slide 18, suggests ARGX-110 is active both at the circulating and bone marrow blast level and at the leukemic stem cell level. The tolerability profile was also encouraging with no exacerbation of azacytidine toxicity. As Dr. Gail Roboz from Weill Cornell highlighted during the workshop, leukemic stem cells play a clear clinical role in AML and those patients with remaining leukemic stem cells after initial treatment have higher rates of relapse. The leukemic stem cells lead to the accumulation of blasts in the bone marrow and therefore, a reduction of other normal blood cells. The target of ARGX-110 CD70 has a unique and appealing expression profile in newly diagnosed AML patients. Slide 19. Firstly, it is expressed across all risk and age categories, regardless of mutational status. This differentiates it from drugs targeting certain mutations, which restrict their use to specific patient subsets only. Secondly, it is overexpressed on malignant cells only while being absent on normal progenitor cells. ARGX-110 is designed to inhibit a CD70/CD27 pathway thereby blocking symmetric division of leukemic stem cells, actually driving them back into normal myeloid differentiation. In this way, we believe, ARGX-110 offers a unique and novel mechanism of action to the treatment landscape, which may be highly complementary to our current AML therapies. As a result of this enhanced factor functionality, it has also shown to take out mature blasts in the periphery. We also announced data during our ASH workshop from the Phase I/II clinical trial of ARGX-110 in relapsed/refractory CTCL patients, presented on Slide #20. Of the 22 CTCL patients under analysis, there was 1 complete response, 2 partial responses and 10 patients achieving stable disease. ARGX-110 continues to show a favorable tolerability profile in CTCL patients. Slide 21. While current cohort sizes are small, we're highly encouraged by seeing responses in AML across the dose cohorts and furthermore by having not reached any dose-limiting toxicity in patients. This encouraged us to amend the Phase I/II AML trial to add an extra dose of 20 milligram per kilogram to the dose escalation to best determine the recommended dose for the Phase II proof-of-concept portion of the trial. In the year ahead, we look forward to presenting interim results from the Phase I/II trial in AML as well as presenting full data from the Phase II study in AML and the Phase II study in CTCL around ASH annual meeting. Let's move to Slide 22. In addition to our wholly-owned programs, we also continue to fuel our pipeline through our Innovative Access Program. We aim to see our next pipeline programs by combining our cutting-edge antibody discovery capability with world-class disease biology expertise on novel pathways and targets under innovative deal structures fairly sharing the economic return with the other party. In this way, we can access novel targets early on, enabling a portfolio management approach. ARGX-112, ARGX-115, and ARGX-116 are successful examples of this approach, and we currently have a portfolio of 7 live IAP programs. With regards to our licensing deals, we can provide the following updates. We agreed with Bird Rock Bio to mutually terminate our license agreement to develop and commercialize ARGX-109 while CERP licensee C Genove[indiscernible] will continue to develop the drug candidate in China. Meanwhile, we continue to make progress under our collaborations with AbbVie, with Shire, LEO Pharma and Staten. I'd now like to turn the call over to our CFO, Eric Castaldi, for him to walk through the financials.

[Eric Castaldi, Chief Financial Officer]

Thank you, Tim. So let's move to Slide #23. As you can see from this side, our financial results primarily reflect research and development expenses and operating income. Operating income reached EUR 41.3 million for the year ended December 31, 2017, compared to EUR 17.2 million for the same period in 2016. This significant increase in operating income in 2017 resulted principally from upfront payments and milestone payments received from our collaboration partners. We have a business model, which relies heavily on outsourcing our R&D activities through external collaborations. Therefore, most of our operating expenses are valuable expenses. On December 31, 2017, R&D expenses amounted to EUR 51.7 million, compared to EUR 31.6 million for the year ended in 2016. The increase in R&D expenses was mainly related to the advancements of our clinical development for ARGX-113 and ARGX-110 and also preclinical and discovery stage product candidates. Our SG&A expenses remained relatively low and reflect our lean and efficient structure. SG&A expenses totaled EUR 12.4 million for the 12-month period ended on December 31, 2017, compared to EUR 7 million for the same period of 2016. The increase seen in 2017 was mainly due to higher personnel expenses, office costs and consulting fees incurred to support our growth and also prepare us to become and operate as a NASDAQ listed company. As a result, the company generated the total comprehensive loss of EUR 28.1 million in 2017, compared to EUR 21.4 million in 2016. We closed out 2017 with a very strong cash position including of cash, cash equivalents and current financial assets of EUR 359.8 million, compared to EUR 96.7 million on December 31, 2016. The significant increase in our cash position resulted from our U.S. public offerings completed in May and December 2017. Moving to Slide #24. This slide shows that so far, we have been highly efficient in managing our capital. Since inception, we have raised a total of EUR 475 million of capital, including the proceeds from our NASDAQ IPO completed in May 2017 and our follow-on U.S. offering completed in December 2017. We also maintained a lean and efficient corporate structure. At the end of 2017, we employed 95 employees and consultants, of which 75 were in R&D and 20 in SG&A. With EUR 359.8 million in cash at the end of December 2017, we believe that we are well-capitalized to execute on our strategic plan. Our bond rate is expected to increase significantly as we will continue to focus our investments on our most advanced clinical development pipeline. In parallel, we will also advance and expand our technical product pipeline looking to access novel targets and technologies. To conclude on this financial slide, you can see the fully diluted shareholding structure of argenx as of February 28, 2018. In 2017, we raised approximately $380 million through 2 public offerings, the NASDAQ IPO in June in mid-2018, -- 2017, sorry, and the December follow-on, as a result of which we reduced our VC overhang to about 9.2% of the outstanding shares compared to 73% at the time of the Euronext's IPO 4 years ago. This financing also attracted more Blue Chip long-term U.S. initial investors resulting in a current free float of more than 80%. And now, I will turn the call back to Tim.

[Tim Van Hauwermeiren, CEO & Executive Director]

Thank you, Eric. I'm very proud of the argenx team and our many accomplishments in 2017. We delivered on our promise to advance our pipeline and create value for our shareholders. Looking ahead to 2018 on Slide 25, our focus will be on executing on the important milestones ahead including: reporting full data from the Phase II proof-of-concept trial for ARGX-113 in myasthenia gravis at the AAN conference in April; reporting top line data from the Phase II proof-of-concept study from ARGX- 113 in ITP as well as interim data of the Phase II proof-of-concept study in pemphigus vulgaris in the second half of 2018; reporting the full data of the Phase I healthy volunteer study with the subcutaneous formulation of ARGX-113 during the second quarter of this year; initiating the Phase III trial for ARGX-113 in myasthenia gravis by the end of the year. For ARGX-110, we plan to provide an update on the ongoing Phase I/II trial and report a full data of the AML Phase I/II trial and CTCL Phase II clinical trial in the second half of the year. And also launching the Phase II proof-of-concept study for ARGX-110 in AML before the end of the year. This is a very exciting time for argenx, and we look forward to continuing to update you throughout 2018.With that, we'd like to open the call now for questions and answers. Operator?

[Operator]

[Operator Instructions] Our first question is from David Nierengarten at Wedbush SEC.

[David Matthew Nierengarten, Managing Director]

I have a couple. So first off, maybe you could help us on what the remaining things that you're looking to do before you start the Phase III in myasthenia gravis? In other words, do you need to meet the FDA, when might that be? And when might be able to produce[indiscernible] more details on the Phase III trial design? And then the second question is on the IgG study. If you could help us with reasons for the follow-up time extension and for the retreatment capability for the second amendment? I mean frankly, is that because you're seeing responses? I suppose you can't tell us that. But is there any other rationale to have a retreatment capability for the second amendment?

[Tim Van Hauwermeiren, CEO & Executive Director]

Good morning, David. Thanks for being with us today and thank you for these questions. So what remains to be done before we can initiate the Phase III myasthenia gravis trial is, as you correctly pointed out, an end of Phase II meeting with the FDA. And what we have said is that we will aim for an end of Phase II meeting and communication the outcome of that end of Phase II meeting before summer. We anticipate that the end of Phase II meeting with the FDA will mainly focus on the minimum size of the safety database, the dose which we recommend using in Phase III as well as the minimum exposures they want to see in these MG patients. So we are committed to communicate to our stakeholders on the outcome of that meeting as soon as we have the minutes. The reason for the follow-up extension and the retreatment arm in ITP is simply because we tried to draw the lessons learnt from the myasthenia gravis trial. As you may remember, all the patients, which achieved a response on that study, actually left the study in a durable response. So now we don't know how long actually these responses lasted. So trying to draw the lessons learnt and, bear in mind, these are different indications and you cannot simply extrapolate a data. At least, we wanted to amend the study such that if and when there would be long-term response, we would be able to capture that response in its full duration. The idea to come up with a retreatment arm is also coming from the effect that now we have a long-term [ touch ] data, which allows actually to extend the exposure in patients and actually learn about what happens with patients which relapse. It will also allow us to rapidly expand the safety database because placebo patients will also be allowed to enroll in that treatment open-label study. Okay?

[Operator]

Our next question is from Boris Peaker from Cowen.

[Boris Peaker, Managing Director and Senior Research Analyst]

My first question is on the 113, I'm just curious, now assuming that the subcu formulation works just as well as the IV formulation in healthy volunteers, which formation do you plan to run in the Phase III program? And also kind of extension of this question for competitive perspective, how important do you think it is to have a subcu formulation in the market versus the IV formulation?

[Tim Van Hauwermeiren, CEO & Executive Director]

Good morning, Boris. Thanks for your questions. So given that our strategy is speed-to-market, it is important to keep our first registration trial, I think, as simple as possible. Today, we don't see any reason why we would not be able to launch a first Phase III registration trial in MG with a plain vanilla IV product. We do believe that the subcu product is going to be important in order to expand the commercial footprint in the market as good as we can. So, I think with offering both products we will be able to just capture and follow patients in a more complete fashion as with just 1 IV product. Does that answer your question?

[Boris Peaker, Managing Director and Senior Research Analyst]

Yes, I guess just to want to understand that if the subcu is important, what is the timing and strategy for getting then a label expansion in a subcu? I mean would this be another study running in parallel to the Phase III of the IV formulation? Or how do you plan to bring the subcu to market?

[Tim Van Hauwermeiren, CEO & Executive Director]

Yes. I would like to refrain from answering that question. I think, it's premature to answer it. Technically speaking, we're ready to launch that subcu product, of course, assuming positive data around middle of the year. Technically speaking, that product is going to be ready. The exact strategy on how we're going to play that has not been disclosed yet.

[Boris Peaker, Managing Director and Senior Research Analyst]

Got you. And just my last question on maybe -- sorry, on 110. I believe you had 1 patient go into transplant. I'm just curious, do we know the outcome for this patient post-transplant?

[Tim Van Hauwermeiren, CEO & Executive Director]

Post-transplant, the study -- the patient is going off study. So what we know is that the transplant has been successful, we don't know what the fate of that patient actually is. Remember, this is a very unusual thing to happen for these elderly, unfit patients to be transplanted. So the only thing which we know according to protocol was that the transplant was successful, but then these people actually leave the study. So I don't have the update on how that patient is actually doing today.

[Operator]

Our next question is from Sandra Cauwenberghs at KBC Securities.

[Sandra Cauwenberghs, Senior Financial Analyst]

I have a small follow-up question on the previous one with regard to the IV and the subcu, I fully understand that is difficult to anticipate on how you're going to take it to market, but do you think that it could actually coexist like the IV in an acute stage? And then you actually give a subcu formulation as a follow-on treatment, for instance, for the condition of ITP? That was my first question. My second question is on the AML population and that's for my own information. These are all newly diagnosed patients, so they did not receive any treatment prior to the start with ARGX-110? And then the third question is on whether it's possible to give us a little bit of guidance on the cash burn rate for 2018 in terms of R&D cost, SG&A, the amount of that?

[Tim Van Hauwermeiren, CEO & Executive Director]

Thank you, Sandra. I will hand over the third question to Eric in a minute. So I think the subcu product could be important or could not be important in ITP depending on the Phase II data. So we don't know anything about how ARGX-113 performs in ITP. But we do want to be ready with the subcu products if and when we think that would be an attractive proposition in MG, ITP or pemphigus. And there are a couple of analogies I would like to draw your attention to. For example, if you look at the HAE market, people have initially been launching products to take care of acute flares and then they have been launching subcu products to go into the maintenance setting. For chronic skin inflammation, that is a precedent, for example, in the [ IO 17 ] space where people actually take patients through an IV induction phase followed by subcu maintenance dosing at home. So I just believe that depending on what we're going to see in these Phase II proof-of-concept trials, having the subcu card ready to play is giving us a lot of flexibility with regards to dosing and dosing regimens in order to serve these patients best.

[Eric Castaldi, Chief Financial Officer]

With regards to AML, it's correct that these are newly diagnosed patients, they have not have been received -- have not been receiving any medication. Typically these people would be dosed, given they are unfit to receive the 7 plus 3 chemotherapy, they would be given a hyperventilating agent as kind of palliative care, that would be Vidaza or decitabine. And this is also the reason why we have to go on top of Vidaza in this Phase I study. You cannot deprive the patient from that standard of care. So we're dosing 110 on top of Vidaza. Bear in mind that we have been able to dose ARGX-110, as monotherapy for the first 2 weeks, then followed by introducing Vidaza on top of 110 in these patients. And I believe when you look at the ASH workshop data that has proven a valuable strategy because we did collect useful information on what happens on 110 monotherapy as well as on Vidaza combotherapy.

[Tim Van Hauwermeiren, CEO & Executive Director]

Eric, I would like to pass the burn rate question to you.

[Eric Castaldi, Chief Financial Officer]

Also on the third question, so we had a cash burn rate of approximately EUR 50 million in 2017, and we anticipate that this cash burn will increase to roughly EUR 80 million for 2018, which is in-line with our business plan. With the current business plan, I would say based on the current design of the trials and depending on the different interactions [indiscernible], we may have increase or decrease in this cash burn, so for the moment, it's EUR 80 million. That's what it is. And as I [ mentioned ], we have EUR 300 million of cash at the end of 2017, and we expect that this is going to provide us with on way [indiscernible] through all our current clinical trials and through 1 registrational trial for ARGX-113.

[Sandra Cauwenberghs, Senior Financial Analyst]

The EUR 80 million that's predominantly R&D cost I suppose, can you give some guidance on the administrative cost?

[Eric Castaldi, Chief Financial Officer]

The administrative cost? So we had for this year, EUR 12.5 million, but that includes also cost like share-based payments. And this cost has increased significantly due to the increase of the share price. So I would say that we don't expect SG&A to increase significantly in 2018. We are working on putting together a team to prepare the future of commercial launch of 113 in the U.S. But nevertheless, I mean we're talking about a few additions. So we -- it also depends on the share-based payment, but we think that SG&A cost will not increase substantially in 2018.

[Operator]

Our next question is from Ted Tenthoff at Piper Jaffray.

[Edward Andrew Tenthoff, Managing Director and Senior Research Analyst]

My question is on ITP, and I appreciate the value of extending dosing for responders. Is there a potential for some form of open-label study for responders not just to keep them on the drug but generate longer-term safety data? And then second question would be with respect to regulatory employment, it's a little bit early to be asking, but what is typically the duration required for ITP?

[Tim Van Hauwermeiren, CEO & Executive Director]

So Ted, on your first question. Actually, the retreatment arm is an open-label study, which actually is going to run for 1 year. So we have ample of opportunity to treat placebo patients and retreat actually all patients from the primary study. And that is therefore a study arm which will continue to run, whilst possibly we could already start to prepare for Phase III. So it's a great addition of retreatment information, safety information, which I think could pay off for the Phase III design. Now on the second question, I'm not sure I completely got your question. So would you mind just briefly repeating the point please?

[Edward Andrew Tenthoff, Managing Director and Senior Research Analyst]

Yes, and that's helpful on the update on the OLE. So on the second question was with respect to an ultimate registrational trial, what kind of follow-up do you think might be required for -- on the primary endpoint and/or safety?

[Tim Van Hauwermeiren, CEO & Executive Director]

Okay. So if you study historical registration trials, which is I think today the only thing which we can realistically do. If you study the registrational trials executed for the TPO-receptor agonists being endplate and eltrombopag. But also if you study the Rigel study on the SYK inhibitors, these are relatively small trials, I mean they have been doing an order of magnitude to registrational trials of about 75 patients each. These typically have been studies lasting above half a year, you know, 26 weeks and then basically, these patients were rolled over in open-label retreatment arm to continue to collect data, which then feed into your [indiscernible] mission. So it's a pretty standard approach. I think, these are manageable trials, manageable numbers of patients, manageable duration. It's a typical registrational design, which I think this company can execute credibly.

[Operator]

Our next question is from Stéphanie Put from Degroof.

[Stéphanie Put]

I wanted to inquire about your strategy going towards other indications for 113. What would be your preferred route? Would that be to also tackle the larger indications by yourself in the future? Or is there a possibility to partner larger indications while retaining the rights for orphan indication?

[Tim Van Hauwermeiren, CEO & Executive Director]

Hi, Stéphanie. It's good to have you in the call. So you know that currently we are focused on executing the current Phase II studies, right? So we have carefully selected these 3 beachheads and actually we believe that there is potential in these individual spaces as soon as you see a convincing sign of efficacy. So we are thinking about the next indication. We're not going to disclose that thinking because the space is simply becoming more competitive than it used to be. Secondly, this compound actually is not available for partnering, we're not thinking about partnering certain rights to any third-party. I think, we will stay focused on executing, for the moment, our own business plan.

[Operator]

It appears there are no further questions at this time, which marks the end of today's conference. We thank you all for your participation. And you may now disconnect. Have a great day.

[Joke Comijn, Corporate Communications Manager]

Thank you, everybody, for joining us.

[Tim Van Hauwermeiren, CEO & Executive Director]

Thank you.