Q3 2019 Earnings Call

Thomas Gad: Taking into consideration our achievements, this is the direct result of a highly dedicated team committed to making both Acidimab and Ombudsman available for children everywhere. We're very pleased with our results. We continue to work hard to solidify our position as leaders in pediatric oncology and focus on advancing our therapies to extend and enhance the lives of those living with rare pediatric cancers. And with that, I'm very pleased to turn it over to Klaus.

Klaus Müller: Thank you very much, Thomas, and welcome to the Y-mAbs Therapeutics third quarter conference call, everybody. We are pleased that you have chosen to join us today.

Klaus Müller: In the third quarter, we have continued to work hard to make sure our lead product candidates Nexidimab and Ombudimab advance towards BLA submissions, which we plan to initiate in the form of a rolling BLA for Nexidimab later this year and Ombudimab to follow shortly thereafter. Actually, we expect the first portion of the rolling BLA for Nexidimab to be submitted within the next few weeks, and we expect a PDUFA date in the fourth quarter of 2020 for Nexidimab and also for Ombudimab. We will continue to keep you posted on our progress.

Klaus Müller: Ombudimab will have a general guidance meeting with the FDA next week. We expect to complete the Ombudimab BLA submission by the end of the first quarter 2020, and we confirm that our projected oral commercialization timeline seems to be unchanged for both products. We expect that a PDUFA date for Ombudimab will be in the fourth quarter also for the year 2020, and we believe that we have continued to demonstrate our ability to execute our planned commercialization timeline in the third quarter of this year. We increased our headcount by approximately 9%, so bringing us to a total of 56 employees during the third quarter.

Klaus Müller: The increase is primarily due to the commercial team ramping up toward the potential launch of our two products. And we continue to believe that we are well-positioned to move Mesetamab and Umbretamab to FDA approval and commercialization in 2020, and concurrently widen our focus to grasp the growth opportunities represented by the earlier stages of our pipeline products, such as Umbretamab TGPA, the bi-specific programs, and the GG2, GG3 vaccine, as well as to make in-line indications for Mesetamab and Umbretamab. In June 2019, we had a promising pre-VLA meeting with the FDA in which we reached alignment with the FDA on an accelerated approval pathway for maxirumab along with the rolling VLA submission I just mentioned. And we have now recruited all patients that we believe will be needed in our study 201.

Klaus Müller: We're in Acetamab for our ELA filing and for the treatment in relapsed refractory high-risk neuroblastoma. At SIOP, the International Society of Pediatric Oncology Conference in October in France and New York, a total of six presentations by Mollison-Kettering provided significant exposure to Acetamab. Let me recap a few highlights from the conference.

Klaus Müller: First, we had data from primary refractory high-risk neuroblastoma patients, refractory patients in studies 12-30. These patients were refractory to intensive induction therapy, and in addition, more than half of the patients were also refractory to second-line chemotherapy. Essentially, this is a subset of patients from studies 12-30 that demonstrated better-than-expected outcomes, including a 78% overall response rate. Patients received at least five cycles of Acetamab therapy post-response and subsequently went on to receive our investigational TB2-TB3 vaccine at MSK. Overall, in this population of patients that are known to very rapidly relapse and continue to progress with their disease, we could see that after two years, a 50% group of the patients were still having progression-free survival. In another subset analysis consisting of 35 patients with relapsed neuroblastoma resistance who received salvage therapy, 30 patients were available for response in studies 12-30.

Klaus Müller: One-third of the patients had two or more relapses prior to enrollment, and 89% of the patients had previously received an anti-TB2 therapy, which could be the Murine Acetamab predecessor, or it could be Antitoxin. Patients in this subset had a 36% progression-free survival rate at two years and an overall response rate of 37%. And please bear in mind with these 37% in secondary refractory and 78% in primary refractory that the FDA's cutoff threshold for these patients given to us overall was 30%, so we are reporting substantially more than was originally guided by the agency originally. We also presented data from patients with high-risk noblostoma and second or later complete remission. Forty-four patients with no evidence of disease were treated with maxetamide and TNCFF and MSK as a maintenance therapy.

Klaus Müller: We know these patients are at high risk of relapsing again, and therefore, we tried to keep them in remission with the maxetamide treatment. In this population, 88% of the patients had previously received anti-GD2 therapy, and 30% had received two or more lines of anti-GD2 therapy previously. A two-year progression-free survival rate of more than 50% was observed. A subset of these patients also went on to receive our investigational GD2-GD3 vaccine and MSK. We believe these data are very encouraging.

Klaus Müller: I should note that this data set is from a subpopulation in study 12-2-30, and these patients are not a part of the efficacy BLA data set because they are not available for formal tumor response grading, only for duration of progression-free survival, because they have no macroscopic disease that is protectable. We also had data for patients with a disease restricted to the bone marrow compartment, which we deemed very interesting, as investigators assessed 100% clearing among these patients in the bone marrow. And I don't think that has ever been seen before in any Norton Stoner trial.

Klaus Müller: Finally, we had data from a Phase II osteosarcoma study that showed safety data for the 25 patients enrolled in the trial at MSK. Patients were treated with maxitumab and GM-CSF and had recurrent disease, and we know these patients are DD2 positive, and they had a minimum of two or more complete remissions. Maxitumab was administered to the patients in an outpatient setting. And Umbertumab, turning to this now, which is our second lead compound, Umbertumab is a B7H3 monoclonal antibody that is regulated with iodine-131.

Klaus Müller: As you may recall, we are developing this compound for CNS leptomeningo-metastasis from low-epistoma, diffuse intrinsic pontine glioma, also known as DIPG, and desmoplastic small round cell tumors, known as DSS-RCG, all of which are B7H3 positive indications. In June of this year, we completed the enrollment of patients in the CNS lepto In September of this year, we requested a regular pre-DLA meeting with the FDA, but in late October, the agency converted the meeting to a general guidance meeting. The FDA did not indicate a reason for this change or provide any additional information.

Klaus Müller: As a result, we now expect that our rolling DLA submission for ombudsman, which we intended to commence in December 2019, will not begin until after a new pre-DLA meeting has been, and this may be rescaled due in the first quarter of 2020. We remain confident in the content of our pre-BLA meeting submission and do not believe that this change in timing of the pre-BLA meeting with the FDA will impact our previously disclosed and anticipated timing for our complete Umberto Mott BLA submission, which we still expect to complete by the end of first quarter 2020. As previously disclosed, we expect to have completed all required components of our anticipated BLA filing by the end of first quarter 2020 and expect to have the flexibility to file our BLA either via a rolling submission or via a single submission ahead of our expected completion date at the end of first quarter 2020. In addition, we believe that the overall commercialization timelines for OmbretaMap will not be affected.

Klaus Müller: Now turning to the data generated with OmbretaMap, at SIOP, we presented an updated OmbretaMap data readout from study 03133 at MSK where patients with CNS leptomeningo-metastasis from low blistoma received two doses of regulated OmbretaMap. Data showed that for the 107 invaluable patients, the median survival had increased to 50.8 months with the final median not yet being reached This compared to the median survival of 47.1 months at the prior data readout based on the first 93 patients in the study. So we are very happy with this update. In addition, we had 68 patients diagnosed with other CNS cancers, including metastatic tumors, who had received a total of 201 injections of OmbretaMap. The injections were routinely administered in an outpatient setting.

Klaus Müller: The patients with primary CNS diagnoses included eight different cancers, including 27 patients with medulloblastoma and 9 with epentumoma. Patients with metastatic tumors included 4 different primary cancers, including 9 patients with sarcomas and 5 patients with melanoma. As of today, 26 of the 68 patients with these highly lethal disease diagnoses are still alive.

Klaus Müller: At CTAS, the Connective Tissue Oncology Society's annual meeting taking place this week in Tokyo, we are also presenting BIRMAP clinical trial data from our DSS-RCT study. In July of this year, we entered into a Development and Manufacturing Supply Agreement with Spectrum and South Bend, Indiana, to secure access to clinical and commercial-scale regulatory capacity for ombudsman. Under the terms of the agreement, Spectrum has agreed to establish a manufacturing unit designed for Y-mAbs within its existing facilities at which both clinical and commercial supply of rubato-labeled Umbertumab for the U.S. market can be produced. The work at Spectrum is progressing as planned. The facility will also serve as backup for the company's overseas activities. And needless to say, we are in the process of establishing a European radio labeling facility as well. We believe Spectrum has the necessary expertise in radio labeling of monoclonal antibodies to be a great partner supporting our launch strategy for invertebrates.

Klaus Müller: We see great potential in radiopharmaceuticals, and we are excited to begin our collaboration with Spectrum. Now, let me invite Bo to share his remarks on the third quarter financials. Thank you, Klaus.

Bo Kruse: For the third quarter, we report spending of $24.4 million, representing a 31% increase in our spending compared to the previous quarter. We entered the third quarter with a cash position of $98.2 million, and as Thomas pointed out, adding the net proceeds from our secondary offering of roughly $135 million, this leads to a performance cash balance of $233 million, net of insurance costs, as of September 30th, 2019. As our work on the BLA submissions for maxidermab and ombudsmab progresses, and we accelerate the commercial ramp-up for the potential launch of the two lead compounds, we are seeing the burn rate increase, and we expect an additional increase in the fourth quarter this year.

Bo Kruse: And the expenses increased by $11 million from $8.7 million for the three months into September 30, 2018 to $19.7 million for the three months into September 30, 2019. This was primarily due to a $7.3 million increase in manufacturing expenses for Narcetamab and Burzumab. And in addition, expenses for outsourced research and supplies increased by $1.5 million for the three months into September 30, 2019 due to our increased need for clinical trial support. Additionally, clinical expenses for personnel increased by $0.7 million for the three months into September 30, 2019, due to an increased level of clinical activity. DNAx expenses increased by $2 million, from $2.7 million for the three months into September 30, 2018, to $4.7 million for the three months into September 30, 2019.

Bo Kruse: The increase in general and administrative expenses was primarily attributable to a $0.8 million increase in employee-related costs, including salary, benefits, and non-cash stock-based compensation for personnel. In addition, costs for setting up commercial infrastructure increased by 0.6 million for the three months ending September 30, 2009. Overall, the increase in G&A expenses primarily relates to the infrastructure and administrative costs of being a public company. In total, the net loss increased by $12.5 million from $11.4 million for the three-month period ended September 30, 2018. 23.9 million for the three months ended September 30th, 2019.

Bo Kruse: The net loss increased by $28.7 million to $57.9 million compared to $29.2 million for the corresponding period in 2018. The cash flows from operating activities in the first nine months of 2019 show that the cash burn increased by $21.2 million from $27.7 million for the nine months ended September 30, 2018, to $48.9 million for the nine months ended September 30, 2019. The increase was primarily caused by an increase in net loss.

Bo Kruse: The net loss increased by $28.7 million for the nine months ended September 30, 2019, which was primarily offset by a $1.4 million increase in accounts payable and accrued liabilities resulting from increased operations and an increase in non-cash expenses, including stock-based compensation of $1.8 million. In terms of financial guidance, we've said since the IPO that the net proceeds would cover our operating expenses and capital expenditures through the fourth quarter of 2020. Now, with the proceeds of the secondary offering, which closed on November 1st, 2019, we expect the cash to fund our operations through the end of 2022. And this number still does not take into account any potential revenues from the commercialization of Nexidema and Ambertema or the proceeds from any potential future partnerships.

Bo Kruse: So, we do believe Y-mAbs remains in a healthy financial position. This concludes the financial update, and I'll now turn the call over to Thomas.

Operator: Uh, it looks like Thomas has, uh, dropped. Thank you for joining us, Mr. Gad. Thank you. Thanks, Doug. Sorry about that.

Thomas Gad: The line dropped. So this concludes our prepared remarks. Thank you, Bo. Thank you, Klaus.

Operator: And Doug, if you could open up the call to questions. Ladies and gentlemen, at this time, we will be conducting a question and answer session. If you'd like to ask a question, you may press star 1. The confirmation tone will indicate your line is in question. You may press star 2 if you would like to remove your question. Please note that, for participants using speakers, it may be necessary to pick up your handset before pressing star 2.

Operator: Our first question comes from the line of Alec Stranahan with Bank of America. Please proceed with your question. Hey guys, great.

Alec Warren Stranahan: Congratulations on the progress and thanks for taking my questions. My first question is about the pre-BLA meeting for Umber and Mab. Have you had any additional interaction with the FDA since the scheduled pre-BLA meeting was delayed? Specifically, any color you can give as to the rationale for converting it to a general meeting and, I guess also, what gives you confidence that the BLA submission will remain on track for 1Q20?

Operator: And then I've got one more.

Thomas Gad: Thank you for the question. I think it makes good sense to bring this up.

Thomas Gad: First of all, of course, we tried to reach out to the FDA to see if they would be able to provide us with some additional guidance, and that was not the case. They came back to us and said, "You should just take it easy." And we would receive preliminary responses to the questions that we had raised in the pre-PLA meeting package that we had submitted, and we would be discussing all the topics that we wanted to discuss that had been raised in our pre-PLA meeting package. My interpretation of the outcome, and based also on, remember we have had a ton of meetings with the FDA on this program, and the FDA has been very collaborative with us on the program and been very helpful also in both Remember, this first indication is only addressing 80 to 100 patients in the U.S., so there's a limit to how much the FDA would even require. However, they understand that they cannot expect the same as you would for a new diabetes treatment coming to the U.S. market.

Thomas Gad: And they have said numerous times that they will do whatever they can to help us, and I think basically, my interpretation is that they're giving us – remember, when you have breakthrough designations, you're entitled to as many meetings with the agency as possible, not just the standard pre-IND meeting and the phase 2 meeting, pre-phase 3 meeting, and pre-BLA meeting. With the breakthrough designation we have for our two lead compounds, we can have as many meetings with the agency as we want to, but we can only have one pre-BLA meeting. And after the pre-BLA meeting, you can, of course, go back if you do not agree, but you cannot have a second pre-BLA meeting. So, this way, they can give us this one and a half hours of discussion next week at the meeting, and then we can go back and ask for a pre-BLA meeting. And then they looked at our timing and our pace. I mean, they started rolling BLA in December with the first products that are manufacturing in the preclinical packets. Thank you very much.

Thomas Gad: It doesn't mean anything else, but we give them some extra time for discussion. That's at least my interpretation of it. And if that's the case, and I still believe that this is in line with what we have discussed with the agency previously, then we should be finished with the entire PLA submission for OMBIRMA by the end of the first quarter. So, so, and again, remember this.

Thomas Gad: We have just updated the data from the study O3133 and seen an increase in regional survival that is still potentially increasing further. We have a patient group where everybody is doomed to die from their disease, and we are curing 50%, more than 50%. And of those that actually die, we can see that half of them die from systemic relapse, not from disease in the brain. They're still cured in the brain. So this is a highly effective treatment for these kids, and there's nothing else for them.

Thomas Gad: They have also looked at a safety database a number of times where we have no indication of any significant safety issues. We see no grade 4, grade 5 safety issues and very, very few grade 3 signals in terms of side effects for these patients. So with all that in hand, I'm very comfortable that the FDA, as Y-mAbs, understands the need to bring this treatment out to patients outside of MSK. And that was exactly what Study 101 was about, to show that we can treat patients at other sites, and we have done that, and we'll be sharing those data with the agency at the meeting next week. And I think that does answer your question, Alec.

Alec Warren Stranahan: Yes, that was very comprehensive. Thank you.

Alec Warren Stranahan: And I had one more question, actually, on the Nexidimab data that was presented at PSYOP in osteosarcoma patients, specifically the neutralizing antibody, human-anti-human. I noticed on the poster that there was a roughly 37% HAHA positivity, which is higher than, I believe, the previously disclosed 15% in neuroblastoma. And, I mean, it's roughly in line with what we're seeing with Unituxin, but I was hoping to hear your thoughts on that. Thanks.

Thomas Gad: Yeah, one thing I mean, the assay used for that evaluation is not a Y-mAbs assay that's validated. It's an old assay developed under non-GOP conditions in MSK's lab. Two, these are not neutralizing antibodies. These are just antibodies against human antibodies that we're looking at. So we don't know whether this actually neutralizes the antibody. There are a lot of uncertainties, though.

Thomas Gad: We are developing a DOP-validated assay for looking into neutralizing antibodies, but all potential antibodies against human antibodies. But it's not for sure, just because you, I mean, I remember this whole discussion from many years ago. As you know, I've worked with antibodies since the 1980s, and when I was in Denmark for 10 years, we were so proud we were creating these fantastic fully human antibodies in the transgenic mouse, and we were certain we would never see any neutralizing or human anti-human antibodies. And what did we see?

Thomas Gad: So it's kind of like, I think the world's perception of this has probably also changed to, yes, you may see these things. If it, you know, in the old days, before Nexetimab, at MSK, they used a murine antibody. And sometimes, kids get old.

Thomas Gad: Neutralizing antibodies against the Murine antibody. That was like, you know, a huge thing because that was really neutralizing. But then they would give the patients nitroxine to treat the plasma cells and buprenorphine, and then they would retreat the patients, and then there would be no neutralizing antibodies. I'm not saying that we will get into a situation like that. I'm just saying that a lot of things in our thinking about antibodies against a drug that are being used as an antibody have changed. So I'm not too concerned about this. These are also a very heterogeneous adult population, and I don't think we have seen any indication of this reducing the efficacy in noblestoma patients. So we cannot say that in noblestoma patients where we see a flare-up after a certain number of cycles with maxillumab, they do worse than patients that do not have this. The number of patients with osteosarcoma is so small, I think it's way too early to start guessing whether that could be an issue here.

Thomas Gad: But I doubt it. I think it's a perfectly normal finding when you give a strange protein in the form of an antibody into circulation, and a certain group of your patients will develop antibodies against it. Whether they will be neutralizing, we actually don't know, and they don't know that from that test. Did that make sense? Yeah. That was great.

Alec Warren Stranahan: Yeah, that was great, thanks. Our next question comes from the line "Robert Burns with HC Wainwright."

Robert John Burns: Please proceed with my question. Hi, guys. Thanks for taking my questions and congratulations on the quarter. I just wanted to follow up regarding the FDA and the ombudsman. So, we know that the FDA brought on a new dosimetry and PK expert. What sort of additional data do you believe that he could request? And if he does request it, how confident are you in your ability to furnish it in a relatively expeditious

Thomas Gad: Well, first of all, I think we need to see what they are actually asking for. Secondly, I think we will be able to provide most of what they could ask for in a timely manner that would not disturb the timing we have for the BLA filing.

Thomas Gad: Secondly, I think it would be very awkward for the FDA to delay the approval of a product like Ombretamab for a group of kids that are currently dying. As I told you, we see about 80 to 100 patients with this indication per year in the U.S., and only about 15 to 20 have the chance to get into the study. So that means that, meanwhile, while waiting for some pharmacokinetic data, we should let another 50% of 80 patients go ahead and die. I don't even think, I don't see anybody that would be able to justify that. Unless there was a safety signal, and then if they had seen something that I had not seen. And I doubt that very much. But, as I said, we go into the meeting with an open mind, and I don't think that's going to be any. We would, of course, after the meeting next week, as soon as we receive the minutes from the FDA, which typically happens one to two weeks after, for this stage of our program, and then we would come out and explain what the outcome of the meeting was and what our expectations were for the continued process for BMA farming for invertebrates.

Robert John Burns: Okay, that's fair. Thank you for clarifying. The second question I have is, can you discuss what sort of interactions you've had with the EMA, if any, with regard to Nexvidimab and Imbercimab?

Thomas Gad: Yeah, so on VirtaMap, we have had a very clear dialogue with the EMEA, and based on my current expectations, I would say that we would be ready to file a, not a DLA, but a licensing application with the EMA towards the end of next year. So, I think what we will try to utilize is kind of like the political momentum following the fact that U.S. kids now have access to the ombudsmobility antibody.

Thomas Gad: And I think the EMA is not going to require anything that's significantly different from what we put in the DLA filing for the FDA. In terms of Nexidemab, it's a little bit more unclear what the EMEA is actually requesting. We've had some discussion.

Thomas Gad: We have received the scientific advice, and it's not clear yet. You know, we have what you call a PIP agreement with EMEA, and we have tried to get a clarified PIP agreement, but it doesn't seem that we have that yet. And it actually goes for ombudsmob also, but I would expect to get the one for ombudsmob within the next few months. But for Nexidemab, I think it's too early.

Thomas Gad: I think a lot of what will drive this is the additional data that we'll be able to generate. And I would expect that we could file for Nexidemab, maybe not in 2020, but probably in 2021 in Europe. But on Birdamap, I'm quite confident we'll be ready for European filing in 2020.

Robert John Burns: And those products, we are also planning to file for approval in China. We have started our first Chinese site in Hong Kong and started treating patients out there. In addition to that, we have treated more than 50 patients from China in a collaboration between a site in Europe and China. And we are working very much towards trying to get our products available for Chinese kids also. Actually, I think the Chinese market will grow and become even bigger than the European market, also in value, because of the number of patients that are out there. And that's a tremendous amount of focus on actually getting access to these pediatric cancer treatments in the Chinese market.

Thomas Gad: And then my last question is, so I noticed that R&D expenses increased roughly 36% compared to the prior quarter. Can you sort of give us some color as to how we should be thinking about, you know, the R&D expenses over the next four quarters?

Robert John Burns: I think I'll let Bo answer that question, but I think it relates a lot to the PPQ batches and the manufacturing that you need to do up to a BLA filing. But, Bo, you may want to give a little more light to this. Yeah, so it's a very fair assumption that the R&D expenses will continue to increase next year, and then given that the company is growing and the... Sales and Marketing part of the business is also ramping up, we would expect to see a solid increase in the DNA as well. So for next year, we do expect to spend more money than we did in 2019.

Thomas Gad: Okay, thank you. Remember, we are still also expanding the pipeline, we are filing the...

Robert John Burns: We are planning to file our second bispecific antibody R&D towards the end of 2020. We are planning to expand on the bispecific antibody program, which is still going tremendously well, in our opinion. I mean, we are 10 months into dose escalation, and we have not seen any side effects that would give rise to any kind of issues with the study. So it's really looking great from that perspective. But it also means additional cost on the R&D side for the new programs. We're expanding the vaccine trials for next year also. There are lots of new activities in addition to Embrunumab and Maxillumab and the additional indications for those two.

Thomas Gad: Thank you so much. Our next question comes from the line of Boris Peeker with Cowen. Please proceed with your question.

Boris Peeker: My first question is, hi, how are you? My first question is about CMC. I'm just curious about the timing of the CMC work for both Umbertumab and Nixitumab, and when will you be ready to submit those parts of the applications?

Thomas Gad: Most of the CMC work for Next Data Map is ready now and will be a part of the first batch that goes into the FDA on the rolling BLA for Nexidermab. The last part of the Nexidermab... CMC, which is the drug substance PPQ report, that should be ready by the end of February, at which point we can complete the maxillomat delay. Of course, these are the timelines I have from my Gantt charts. It may rock and roll for a few weeks in one direction or the other, but that's as precise as I can give it.

Thomas Gad: In terms of, and I also need to add, we had a separate FDA discussion on the CMC in September, and I'm comfortable that we are in line with the FDA expectations for the CMC package. As for the OmbertaMap CMC packets, we have a large part of that also available here in December. The last parts, which will be the last PPQ reports for the CMC of the OmbertaMap, should be ready by the end of March, at which point we can complete the OmbertaMap CMC packets and the entire BMA. And just getting back...

Boris Peeker: And just getting back to the conversation regarding the meeting conversion from pre-BLA to general guidance. Just curious, is there any new material that you submitted to the agency over the last month or two that potentially could have impacted that decision?

Thomas Gad: Well, obviously, we submitted an entire pre-BLA meeting package to the FDA, and while reviewing that package, the FDA decided that apparently there was so much to discuss that they were not sure that we could... Squeeze all of it into a pre-PLA meeting and let's give these guys some extra time. But there's nothing in there that I find in any way controversial or that keeps me awake during the night or makes me shake on my end.

Thomas Gad: I mean, the data is clear. We just provided an update on the 133 study. Median survival is still going up in spite of the fact we added another 14 patients to the patient population. The patients from study 101; we have way more than 20 patients in that study now. And we said we would come forward with 18. So for the BLA filing package in March, there will probably be more than 20 in that study. There's nothing that we have seen while treating the 101 patients that gives us any concern in this context. So not really, I mean, I don't think that we have any major issues that could pop up, but there are apparently a number of things that we will need to discuss with the agency. And we have put in a ton of questions to the agency. That's all. That's all.

Boris Peeker: Most of that data didn't come from the FDIC; at one point or another, it was part of the discussion, or is most of the data...

Thomas Gad: No, all the data relating to study 101 that the FDA had not seen before. Okay.

Thomas Gad: Because that was the multi-center study, and as you can imagine, Although we have had a separate CMC meeting also from BODEMAT in September this year, we still have outstanding issues on the CMC since the PPQ. Manufacturing batches have not been done at the time point of the filing, so it's in the process of being done. And one PPQ batch will be included as a part of the pre-DLA filing. So it's

Thomas Gad: And again, as I said, we have treated more than 200 patients in Burma now with a radio-labeled antibody, including 40 patients with DIPD and 60 patients with DSSRCT and 68 patients outside of the 107 patients with CNS leptomedical metastases from nobustoma and more than 20 patients in study 101. So it's a sizable patient safety profile we have. It's a sizable duration of experience in terms of survival outcomes. Remember, the first patients in 133 were treated 17 years ago. And nevertheless, we can maintain this 50% cure rate for these patients. So I think we, I mean, there may be issues that, and remember also that when you put in these pre-BLA packets, they put additional people on the team, people that have not seen the program before, people that may not pay attention to the fact that, hey, this is treatment information you can actually put into a filing because it can simply not be generated.

Thomas Gad: If you look up the entire literature and set aside the MSK data that's published, but just look at what everybody else has published and put together all the publications available in the literature, you cannot even find a total of 100 patients in papers published with CNS-Dexamygomatasosis from Nobel Stomach. We have one single study that contains 107 patients. But it tells you a little bit about the impact that these data should also have on the agency, and the data is looking very strong.

Arlinda Lee: Great. Thank you for taking my question. Our next question comes from the line of Arlinda Lee with Canaccord Genuity. Please proceed with your question. Hi, guys. It's Ben Shem on for our Linda.

Thomas Gad: Thanks for taking my questions. My first question is one of process. And maybe this is a simplistic question, but is there a time limit between the BLA meeting and when you must file the BLA?

Thomas Gad: And the FDA can, at a pre-PLA meeting, even tell you that they do not believe you should go ahead and file. If you do that, the FDA may decide to give you a denial of file. So that's why you want to have your pre-BLA meeting, and that's also why you want to make sure that you have a pre-BLA meeting where you come to an agreement with the agency about filing.

Arlinda Lee: That's great. And secondly, can you maybe give us a little bit more color on commercial prep activity? You know, Salesforce, and maybe it's too soon to think about this, your marketing strategy around Unituxin.

Thomas Gad: Yeah, I mean, we started building our commercial organization by hiring Phil Herrmann as chief commercial officer in May 2018. And now, the team is up to nine people.

Thomas Gad: And we are, in parallel with that, also building a medical safe license team, which is, of course, a part of the medical group. But, of course, we'll work in close collaboration with the commercial organization. You know, the total number of sites that are addressing these patients is not more than 70, 80 sites in the U.S., based on our analysis, and these 70 to 80 sites take about 80% of the entire nervous stoma patient population, and the remaining is spread out. You know, somebody leaves the hospital where he has been leading this treatment and comes to a new hospital that normally does not treat patients, and the patient comes in and says So it is changing a little, but I think a good rule of thumb would be that 80% are treated at 80 hospitals in the U.S., so it's not that you need 500 sales reps to reach out.

Thomas Gad: Our ballpark estimation is that about five sales reps would be able to cover the sites that we have in the U.S., together with a similar number of medical sales liaisons that can help these sites get used to using our antibody, knowing how to handle the practical implications of getting this infusion in Sweden and how to deal with side effects and all these kinds of things that are related to treatment with an antibody. And like cinema, again, when we look at the radio-labeled antibody, we are looking at even fewer sites that will be using this. As I just said, it says that we treat 100 patients per year in the U.S. for the first indication. That's going to happen probably in less than 15 sites.

Arlinda Lee: So it's a relatively small but focused organization. And, of course, a very important part for us will be the interaction with the pediatric oncology societies and with patient organizations. And we need to have the co-pay set up for those that cannot pay their own share of these treatments so nobody is not able to get access to this treatment just because they are missing their own co-pay. And we need to set up collaborations with the distribution partners and the insurance companies and Medicare. So we are building this organization, and I think we have a great chief commercial officer to help build this out and make sure we are ready to launch in due time for our potential approval next year.

Operator: Great, thanks very much, and best of luck to you.

Thomas Gad: There are no further questions in the queue. I'd like to hand the call back to management for closing remarks. Thanks, everyone. I think that concludes it. Thanks, Bo. Thanks, Klaus. Thanks, everyone, for dialing in. Have a great evening.

Thomas Gad: Thanks, Thomas. Take care.

Operator: Ladies and gentlemen, this does conclude today's teleconference. Thank you for your participation. You may disconnect your lines at this time, and have a wonderful day.