Good day. And welcome to the Y-mAbs Therapeutics, Inc., 2019 Third Quarter Earnings Conference Call. Today's conference is being recorded. Let me quickly remind you that the following discussion contain certain statements that are considered forward-looking statements, as defined in the Private Securities Litigation Reform Act of 1995.

Because forward-looking statements involve risks and uncertainties, they are not guarantees of future performance, and actual results may differ materially from those expressed or implied by these forward-looking statements due to a variety of factors, including those risk factors discussed in the company's prospectus supplement dated October 29, 2019, and filed with the SEC on October 31st, 2019, and the company's Annual Report on Form 10-K for the fiscal year ended December 31st, 2018, as filed with the SEC on March 22, 2019.

At this time, I would like to turn the conference over to Thomas Gad, the Company's Founder, Chairman, and President. Please go ahead, sir..

Thank you, Doug. Hello, everyone. And welcome to our third quarter earnings call. Today, we're going to hear remarks from our Chief Executive Officer, Dr. Claus Moller, and as well as our Chief Financial Officer, Bo Kruse. We ended the third quarter with $98.2 million in cash.

As you all know in late October, we completed an oversubscribed secondary offering, which was led by Morgan Stanley, J.P.

Morgan, and BAML, in which we sold approximately 5.1 million shares of our common stock, which included the full exercise of the over-allotment option at $28 per share, raising approximately $135 million in net proceeds to the Company. At the end of the third quarter, this would bring our pro forma cash position to $233 million.

The transaction produced a very high quality demand, enabling us to further expand our excellent shareholder base. The proceeds from the offering further strengthened our balance sheet sufficiently to potentially launch both naxitamab and omburtamab next year. And at the same time, we can advance our pipeline and carrying us through the end of 2022.

This doesn't take into consideration any potential product sales or partnership income. Naxitamab and omburtamab have both received rare pediatric disease designation from the FDA.

We believe that our overall timelines for submission and potential approval for both naxitamab and omburtamab are on track for completing first quarter BLA filings with potential approvals in 2020. Overall, we are very pleased with our current financial position.

Naxitamab and omburtamab continue to readout very encouraging data, addressing clear unmet medical needs from neuroblastoma patients, and we have now shown we are able to reproduce very encouraging results for both antibodies in multi-center trials.

We think this is a very encouraging time for patients waiting to get access for naxitamab and omburtamab. At this point, we are -- and then naxitamab and omburtamab are being used in clinical trials in Europe, the U.S. and in Hong Kong, and this is a major achievement for Y-mAbs. For the first nine months in '19, we spent $49.7 million.

Taking into consideration our achievements, this is a direct result of the highly dedicated teams committed to making both naxitamab and omburtamab available for children everywhere. We are 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 lives of those living with rare pediatric cancers. And with that, I'm very pleased to turn it over to Claus..

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.

Over the third quarter, we had continued to work hard to make sure our lead product candidate naxitamab and omburtamab advance towards BLA submissions, which we plan to initiate in the form of rolling BLA for naxitamab later this year and omburtamab to follow shortly thereafter.

Actually, we expect the first portion of the rolling BLA for naxitamab to be submitted within the next few weeks, and we expect a PDUFA date in the first quarter of 2020 for naxitamab and also for omburtamab. We will continue to keep you posted on our progress. On omburtamab, we will have a general guidance meeting with the FDA next week.

We expect to complete the omburtamab BLA submission by the end of first quarter 2020, and reconfirm that our projected overall commercialization timeline seems to be unchanged for both products.

We expect that a PDUFA date for omburtamab would be in the fourth quarter, also for omburtamab of 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. The increase is primarily due to the commercial team ramping up for the potential launch of our two products.

And we continue to believe that we're well positioned to move naxitamab and omburtamab 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 omburtamab DTPA, the bispecific programs and the GD2-GD3 Vaccine, as well as the next in line indications from naxitamab and omburtamab.

In June 2019, we had a promising pre-BLA meeting with the FDA, in which we reached alignment with the FDA on an Accelerated Approval Pathway for naxitamab along with a rolling BLA submission, I just mentioned.

And we have now recruited all patients that we believe will be needed in our Study 201, naxitamab for our BLA filing and for the treatment of relapsed/refractory high-risk neuroblastoma.

At SIOP, the International Society of Pediatric Oncology conference in October in France, in Lyon, a total of six presentations by Memorial Sloan Kettering provided significant exposure to naxitamab. Let me recap a few highlights from the conference.

First, we had data from the primary refractory high-risk neuroblastoma patients reflecting patients in Study 12-230. These patients have 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 Study 12-230 that demonstrated better-than-expected outcomes, including a 78% overall response rate. Patients received at least 5 cycles of naxitamab therapy post response and subsequently went on to receive our investigational GD2-GD3 vaccine at MSK.

Overall, in this population of patients that are known to very rapidly relapse and continue to progress in 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 received salvage therapy, 30 patients were evaluable for response in Study 12-230.

One third of the patients had two or more relapses prior to enrollment, and 89% of the patients had previously received an anti-GD2 therapy to be the murine naxitamab predecessor or it could be the naxitamab. Patients in this subset had 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 factory that the FDA's cut off threshold for these patients given to us overall was 30%. So we are reporting substantially more than what was guided by the agency originally.

We also presented data from patients who have high-risk neuroblastoma and second or later complete remission. 44 patients with no evidence of disease were treated with naxitamab and GM-CSF at MSK as a maintenance therapy. We know these patients are at high risk of relapsing again.

And therefore, we try to keep them in remission with naxitamab treatment. In this population, where 88% of the patients have previously received anti-GD2 therapy and 30% have 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 at MSK. We believe these data are very encouraging.

I should note that this data set is from a subpopulation in the Study 12-230, and these patients are not a part of the efficacy BLA data set, because they are not evaluable performance nor response rating only for the duration of progression-free survival because they have no macroscopic disease to take the bone.

We also had data from patients with disease respective to the bone marrow compartment, which we deemed very interesting as investigators attest 100% clearing among the patients in the bone marrow. And I don't think that has ever been seen before in any neuroblastoma trial.

Finally, we had data from a Phase II osteosarcoma study that shows safety data for the 25 patients enrolled in the trial at MSK. Patients were treated with naxitamab and GM-CSF and had recurrent disease, and we know these patients are GD3 positive, and these had at the minimum two or more complete remissions.

Naxitamab was administrated to the patients in an outpatient setting. And omburtamab, turning to this now, which is our second lead compound. Omburtamab is a B7-H3 monoclonal antibody that is radiolabeled Iodine-131.

As you may recall, we are developing this compound for CNS leptomeningeal metastasis from neuroblastoma, Diffuse Intrinsic Pontine Glioma also known as the DIPG and Desmoplastic Small Round Cell Tumors known as DSRCT, all of which are B7-H3 positive indication.

In June of this year, we completed the enrollment of patients in CNS/leptomeningeal metastasis Study 101 that are needed for omburtamab BLA. In September of this year, we requested a regular pre-BLA 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.

As a result, we now expect that our rolling pre-BLA submission for omburtamab, which we intend to commence in December 2019, will not begin until after a new pre-BLA meeting has been scheduled, as described in the Form 8-K that we filed with the SEC on November 1, 2019, and this may be rescheduled 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 omburtamab 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 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 rolling submission ahead of our expected completion date at the end of first quarter 2020.

In addition, we believe that the overall commercialization time lines for omburtamab will not be affected. Now turning to the data generated with omburtamab. At SIOP, we presented an updated omburtamab data readout from Study 03-133 at MSK, where patients with CNS/leptomeningeal metastasis from neuroblastoma received two doses of regulated omburtamab.

Data showed that, for the 107 evaluable patients, the median survival had increased to 50.8 months with the final median not yet being reached and more than half of the patients being alive. This compares to the median survival of 47.1 months at prior data readout based on the first 93 patients in this 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 omburtamab.

The injections were routinely administered in an out-patient setting with patients with primary CNS diagnosis included eight different cancers, including 27 patients with medulloblastoma and nine with ependymoma, patients with metastatic tumors included four different primary cancers, including nine patients with sarcoma and five patients with melanoma.

As of today, 26 of the 68 patients with these highly lethal disease diagnoses are still alive. At CTOS, the Connective Tissue Oncology Society's Annual Meeting taking place this week in Tokyo, we are also presenting omburtamab clinical trial data from our [indiscernible] study.

In July of this year, we entered into a development, manufacturing, supply agreement with Spectron in South Bend, Indiana to secure access to clinical and commercial-scale radiolabeling capacity for omburtamab.

Under the terms of the agreement, Spectron has agreed to establish a manufacturing unit designed to Y-mAbs within its existing facilities at which both clinical and commercial supply for radiolabeled omburtamab for the U.S. market can be produced. The work at Spectron 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 radiolabeling facility as well. We believe Spectron has the necessary expertise within radiolabeling of monoclonal antibodies to be a great partner supporting our launch strategy for omburtamab.

We see great potential in radiopharmaceuticals and we have decided to begin our collaboration with Spectron. Now let me invite Bo to share his remarks on the third quarter financials..

Thank you, Claus. For the third quarter, we report spending of $24.4 million, representing a 31% increase in our spending compared to last quarter. We ended 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 former cash balance of $233 million net of issuance costs as per September 30, 2019.

As our work on the BLA submissions for naxitamab and omburtamab progresses, and we accelerate the commercial ramp up for the potential launch of the two lead compounds, we are seeing the great increase, and we expect an additional increase in the fourth quarter of this year.

R&D expenses increased by $11 million from $8.7 million for the three months ended September 30, 2018 to $19.7 million for the three months ended September 30, 2019. This was primarily due to a $7.3 million increase in manufacturing expenses for naxitamab and omburtamab.

And in addition, expenses for outsourced research and supply increased by $1.5 million for the three months ended September 30, 2019 due to our increased need for clinical trial support. The clinical expenses for personnel increased by $0.7 million for the three months ended September 30, 2019 due to an increased level of clinical activities.

G&A expenses increased by $2 million from $2.7 million for the three months ended September 30, 2018 to $4.7 million for the three months ended September 30, 2019.

The increase in general and administrative expenses was primarily attributable to a $0.8 million increase in employee related costs, including salaries, benefits and noncash stock-based compensation for personnel. In addition, costs for setting up commercial infrastructure increased by $0.6 million for the 3 months ended September 30, 2019.

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 months period ended September 30, 2018 to $23.9 million for the three months ended September 30, 2019.

For the nine month period ended September 30, 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 the net loss.

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 noncash expenses, including stock-based compensation of $1.8 million.

The net cash flow for the first nine months of 2019 show spends of $49.7 million. In terms of financial guidance, we expect, since the IPO, that 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 1, 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 commercialization of naxitamab or omburtamab or the proceeds from any potential future partnerships.

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.

Thomas?.

It looked like Thomas has dropped his line..

Thank you. Thanks, Doug. Sorry for that, just the line dropped. So this concludes our prepared remarks. Thank you, Bo, thank you, Claus. And Doug, if you could open up the call to questions..

Thank you. Ladies and gentlemen, at this time we will be conducting a question-and-answer session. [Operator Instructions] Our first question comes from the line of Alec Stranahan with Bank of America. Please proceed with your question..

Congrats on the progress, and thanks for taking my question. My first question is on the pre-BLA meeting for omburtamab, have you had any additional interaction with the FDA since the scheduled pre-BLA meeting was delayed. Specifically, any color you can give us the rationale for converting it with general meeting.

And I guess also what gives you confidence that the BLA submission will remain on track for 1Q '20? And then I've got one more. Thanks..

Thank you for the question. And again, it makes good sense to bring this up. 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, we should just take it in, and we would receive preliminary responses to the questions that we have raised in the pre-BLA meeting packet that we have submitted, and we would be discussing all the topics that we wanted to discuss that had been raised in our pre-BLA meeting packet.

My interpretation of the outcome and based on also -- and remember, we have a ton of meetings that we have had 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 guidance and also in accommodating what is possible.

Remember, this first indication is only addressing 80 to 100 patients a year in the U.S. So there's a limit to how much, also, the FDA would even require. They understand that they cannot expect the same as your new diabetes treatment coming to the U.S. market. 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 have as many meetings with the agency possible, not just the standard pre-IND meeting and the Phase II meeting, pre-Phase III meeting and pre-BLA meeting.

With the breakthrough designation, we have our two lead compounds, we can have as many meetings with the agency we want to, but we can only have one pre-BLA meeting. And after the pre-BLA meeting, you can then, of course, go back if you do not agree, but you cannot have a second pre-BLA meeting.

So the idea I think is simply that they say, hey, there's a number of things here. You're ending your one and hour of discussion with us to discuss these topics here.

And we believe you may need more time for coming to an agreement with us on what needs to be done and kind of like fine-tuned in your filing based on the questions that you have raised, because we are the ones that are asking questions to the agency about the package.

So then this way they can give us the one and 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 have looked at our timing, and said, hey, I mean this guy has placed out a rolling BLA in December with the first product manufacturing and the preclinical package, but they'll do it in the beginning of February.

We've got to change our overall timeline of having the last part of the CMC and the whole clinical package finished by the end of March. So overall, it doesn't mean anything else, but it gives us to make the time for discussion.

And that's at least my interpretation of it and 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 BLA submission for omburtamab by the end of first quarter.

And again, remember, I mean we have just updated the data from this Study 03-133 and seen an increase in the median overall survival. There is still potential increase in survival. We have a patient group where everybody is going to die from their disease. And we are curing 50%, more than 50%.

And if those that actually dies, we can see that half of them dies from systemically then, not from disease in the brain. They're still cured in the brain. So this is a highly efficacious treatment for these cases, and there's nothing else for them.

So they have also looked at safety database now, a number of times where we have no indication of any significant safety issues, we see no grade four or grade five safety issues, and very, very few grade three signals in terms of side effects for these patients.

So with all that in hand, I'm very comfortable that the FDA and YmAbs understands the need for bringing this treatment out to patients outside of MSK, and that's what's expected with Study 101 was about to show that we can treat patients in 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 that answers your question, Alec..

Yes, that's very comprehensive. Thank you. And I had one more question actually on the naxitamab data that was presented at SIOP in osteosarcoma patients, specifically the neutralizing antibody, human anti-human.

I noticed in the poster that there was a roughly 37% HAHA positivity, which is higher than -- I believe previously this was 15% in neuroblastoma, and I mean it's roughly in line with what we're seeing in with unituxin. But I was hoping to hear your thoughts on that. Thanks..

One, I mean, the assay used for that evaluation is not a YmAbs' assays that validated. It's an old assays developed under non-GLP conditions in MSK's lab. Two, it's not neutralizing antibodies. It's just antibodies against human antibodies that we're looking at. So we don't know whether these actually neutralize the antibody.

So there is a lot of uncertainty and so we are developing a GLP validated assay for looking into neutralizing antibodies, but our 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 worked with antibodies since the 1980s.

And when I was Genmab for 10 years, we were so proud, we were creating these fantastic, fully human antibody in the transgenic mouse, and we were certain, we would never see any neutralizing all human anti-human antibodies, and what did we see, human anti-human antibodies.

So it's kind of like, I think the world's perception of this has probably also changed to, yes you may see these thing. If it -- in the old days, before naxitamab at MSK, they used to murine antibody. And sometimes when kids developed neutralizing antibodies against the murine antibody that was like shoot seek, because that was really neutralizing.

But then they will give the patient rituxan to treat their plasma cells and the B lymphocyte, and then they would retreat the patients and then there were no neutralizing antibodies.

So I'm not saying that we will get into a situation like that, I'm just saying that there is a lot of things in our thinking about antibodies against a drug that I've been 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 the neuroblastoma patients. So we cannot say that in neuroblastoma patients, where we see a flare up after a certain amount of cycles, the naxitamab then maybe worse than the patients that do not have it.

The number of patients with osteosarcoma is so few, I think it's way too early to start guessing, whether that could be an issue here, but I doubt it. I think it's a perfectly normal finding that it is a strange protein in the form of an antibody into circulation, but a certain group of your patients will develop antibodies against it.

But whether they will be neutralizing, we actually don't know and they don't know that from that.

Does that make sense?.

Yes, that was great. Thanks..

Our next question comes from the line of Robert Burns with H. C. Wainwright. Please proceed with your question..

I just wanted to follow-up regarding the FDA on omburtamab. So we know that the FDA brought on a new inventory and TK 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 manner?.

Well, first of all, I think we need to see what it is that they are actually asking for. Secondly, I think we will be able to provide most of what they could ask for in that time. They may not, but not disturb the timing we have for the BLA filing.

Secondly, I think it would be very awkward for the FDA to delay the approval of a product like omburtamab for group of the kids that are currently dying. As I told you, we see about 80 patients to 100 patients with this indication per year in the U.S., and they only about 15 to 20 has a good chance to get into the study.

So that means that, meanwhile, waiting for some pharmacokinetic data, we should let another 50% of 80 patients go ahead and die. I don't see anybody that will be able to justify that unless there were safety signal. And if FDA have seen something that I have 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 there's going to be any shaking news.

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 data of program, and then we would come out and guide what the outcome of the meeting was and what are our expectations, but the continued process for BLA filing for omburtamab is..

Thank you for clarifying that.

And second question I have is, can you discuss what sort of interactions you've had with the EMA, if any, with regard to naxitamab and omburtamab?.

For omburtamab, we have had a very clear dialog with the EMA. And based on my current expectation, I would say, that we would be ready to file a BLA with a licensing application with the EMA towards the end of next year. So I think what we will try to utilize, it's kind of like the political momentum following by the fact that U.S.

kids have now access to the omburtamab antibody, and I think in the areas that's going to require anything that's significantly different from what we put into BLA filing for the FDA. In terms of naxitamab, it's a little bit more unclear what actually EMA is requesting. We've had some discussion. We have received the scientific advice.

And it's not clear yet. We have what you call a PIP agreement with EMA and we have tried to get verified PIP agreement, and it doesn't seem that we have that yet and it actually goes to omburtamab also. But I would expect to get the one for omburtamab within the next few months. But for naxitamab, I think it's too early.

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 naxitamab maybe not in 2020 but -- or probably in 2021 in Europe. But omburtamab, I'm quite confident would be ready for European filing in 2020. And both products we are also planning to file for approval in China.

We have started our first Chinese side in Hong Kong and started treating patients out there. In addition to that we have treated more than 50 patients from China in 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 that 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 of actually getting access to these pediatric cancer treatments in the Chinese markets..

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 to how we should be thinking about the R&D expense over the next four quarters?.

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 bit more light to this..

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 slight increase in the G&A as well. For next year we do expect to spend more money than we've done in 2019..

Remember, we have still also -- we are expanding the pipeline, we are filing the omburtamab DTPA IND by the end of this year, and we're planning to file our second bispecific antibody IND towards the end of 2020.

We are planning to expand on the biospecific antibody program, which is still going tremendously well, and in our opinion, I mean we are 10 months into dose escalation, and we have not seen any side effects that 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 just to do IND for the R&D side for the new programs. We're expanding the vaccine trials for next year also. So there's lots of new activities in addition to omburtamab and naxitamab and the additional indications for those tow assets..

Our next question comes from the line of Boris Peaker with Cowen. Please proceed with your question..

My first question is on the CMC.

I'm just curious, what is the timing of the CMC work, for both omburtamab and naxitamab? And when will you be ready to submit those parts of the applications?.

Most of the CMC work for naxitamab is ready now, and will be a part of the first set goes into the SBA on the rolling BLA for naxitamab, last part of the naxitamab CMC, which is the drug substance PPQ report that should be ready by the end of the February. And at this time point, we can complete the naxitamab BLA.

Of course these are the timelines I have from charts. It may rock and roll a few weeks one direction or the other. But as precise as I can give this, in terms of -- and I also need to add, we had a separate FDA discussion on the CMC in September and I'm comfortable where we are -- in line with the FDA expectations for the CMC package.

As for the omburtamab CMC package, we have a large part of that also available actually here in December, the last parts which will be last PPQ reports for the CMC omburtamab should be ready by the end of March at which time point, we can complete the omburtamab CMC package and the entire BLA..

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 2 that potentially could have impacted that decision?.

Well, obviously we submitted an entire pre-BLA meeting package to the FDA, and while reviewing that package, the FDA decided that apparently there were so much to discuss, but they were not sure that we could squeeze all of it into a pre-BLA meeting and [Multiple Speakers] 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 make me shake on my hand. I mean, the data is clear. We just provided an update on the 132 -- 133 study. Median survival is still going up in spite of the fact we added all 14 patients to the patient population.

In the patients from Study 101, we have way more than 20 patients in that study now, and we have said we would come forward with 18. So for the BLA filing packet in March, there will be probably more than 20 in that study. There's nothing that we have seen while treating the 101 patients that give us any concern in this context, so not really.

I mean, I don't think that we have any major issues that can come up but there's 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. So there's lots of things to talk about..

Most of that data didn't -- the FDA see at one point or another, or it was part of the discussion? Or is most of the data….

No, all the data relating to study 101, we have not seen before, because that was the multi-center study. And as you can imagine, although, we have had a separate CMC meeting also for omburtamab 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 this filing.

So it's in the process of being done. And we want PPQ batch will be included as part of the pre-BLA filing.

But again as I have said, we have treated more than 200 patients with omburtamab now with the radiolabeled antibody, including 40 patients with the DIPG and 60 patients with DSRCT and 68 patients outside of the107 patients with CNS leptomeningeal metastasis from neuroblastoma and more than 20 patients in Study 101.

So it's a sizable patient safety patterns we have. It's a sizable duration of experience in terms of survival outcome, the first patients in 133 was treated 17 years ago. And nevertheless, we can maintain this 50% cure rate for these patients.

So, but I think, I mean, there may be issues and remember also that when you put in these pre-BLA package, they put additional people on their team. People that have not seen the program before, people that may not pay attention to the fact, this is a treatment for maybe 200 new patients a year in the US.

This is not the thousands and thousands of patients; so that's a limit to how much information you can actually put into a file because it can simply not be generated.

If you look up the entire literature and set aside the MSK data that published, but just look at everybody else who has published, and put together all the publication available in the literature, you cannot even find a total of 100 patients in papers published with CNS, leptomeningeal metastases from neuroblastoma.

We have one single study that contains 107 patients. So, it tells you a little bit about the impact that these data should have also with the agency and the data is looking very strong..

Our next question comes from the line of Arlinda Lee with Canaccord Genuity. Please proceed with your question..

This is Ben Shim on for Arlinda. Thanks for taking my questions.

My first question is one of process and maybe this is a simplistic question, is there a time limit between the BLA meeting and when you must file the BLA?.

No. And the FDA can add a pre-BLA meeting and 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 refuse to file.

So that's why you want to have you pre-BLA meeting and that's also why you want to make sure that you have pre-BLA meeting when you come to an agreement with the agency about filing, and what efficiencies that eventually may be there that you can need to, and live up to..

And secondly, can you maybe give us a little bit more color on commercial prep activity, sales force and maybe it's too soon to think about the marketing strategy around unituxin?.

We started building our commercial organization by hiring Philip Herman, Chief Commercial Officer on May 2018, and now the team is up to nine people, and we are in parallel with that also building a medical sales 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. 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 neuroblastoma patient population.

And the remaining is spread out, somebody leaves the hospital, where he has been leaving this treatment and comes to a new hospital that normally do not treat and then the patient comes in as I can fix this for you, you know, I am used to it. So it is changing a little.

But I think a good rule of thumb would be that 80% is treated at the 80 hospitals in the U.S. So it's not that you need 500 sales reps to reach out.

Our ballpark estimation is about 5 sales reps would be able to cover, the site around half of the U.S., together with a similar number of medical sales liaisons that can help these sites getting used to using our antibody, knowing how to handle the practical implications of giving this infusion and so the momentum, how to deal with side effect, and all these kind of things that are related to the treatment with an antibody, right.

And naxitamab, again also when we look at the radio-labeled antibody, we are looking at even less sites that will be using this, as I just said, we treat 100 patients per year in the US for the first indication that's going to happen probably in less than 15 sites.

So it's a relatively small, but focused penetration and equivalent and very important part will also be the interaction with the pediatric oncology societies and with the patient organization, we need to have the co-pay set up for those that cannot pay their own share of these treatment, so nobody is not able to get access to this treatment, just because they're making their own co-pay and you need to set up collaborations with the distribution partners and then insurance companies and Medicare.

So we are building this organization. And I think we have a great Chief Commercial Officer to help us with this, and make sure we are ready to launch in due time for our potential approval next year..

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 Claus, thank you everyone for dialing in. Have a great evening..

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..