Greetings, and welcome to Y-mAbs Fourth Quarter and Full Year 2020 Earnings Conference Call. At this time, all participants are in a listen-only mode. A brief question-and-answer session will follow the formal presentation. [Operator Instructions] As a reminder, this conference is being recorded.

It is now my pleasure to introduce your host, Thomas Gad, Chairman of Y-mAbs. Thank you, sir. You may begin..

Thank you, and thank you, everyone. Good morning and thank you for joining us today. So 2020 was the year that we believe was truly transformational for Y-mAbs as we became a commercial stage company. We’ve made significant progress on executing our strategy and expanded significantly on our three pillars of business.

First, our leading monoclonal antibodies, DANYELZA and omburtamab; second our bispecific compounds under the Y-BiClone tech platform; and finally, the SADA Technology platform. As you know, we received U.S. approval for DANYELZA in primary refractory and relapsed high-risk neuroblastoma on November 25, 2020.

And from the beginning of February, we’ve been delivering DANYELZA, not only to MSK, but to hospitals throughout the U.S. So that’s very exciting. Omburtamab, we continue to focus our efforts and work very hard on resubmitting the BLA as soon as possible.

After receiving the refuse-to-file letter from the FDA, we’ve had multiple interactions with the agency, and we’ve now scheduled a Type B meeting with the FDA on March 26, 2021, where we will be presenting what we believe will have address the additional data request from the FDA, and us aiming for near-term resubmission now likely to be finalized by the end of the second quarter or in the third quarter.

We are very pleased and excited to see our next generation lutetium-conjugated omburtamab-DTPA enter into the clinic for B7-H3-positive adult brain metastases and for pediatric brain tumors here in this quarter 2021, addressing a very large unmet medical need.

Following the approval of DANYELZA, we’ve entered into a licensing agreement for DANYELZA and omburtamab, with SciClone Pharmaceuticals for Greater China, which included a $20 million upfront payment and up to an additional $100 million in approval and commercial milestones.

Here we submitted our three BLA package for DANYELZA in China at the end of January this year. We have also signed a licensing agreement with Takeda Israel for Israel, and I’m now scheduled to submit a local BLA package in this quarter as well.

We entered into a distribution agreement with Swixx BioPharmaceuticals covering Eastern Europe and Russia, and we’ll continue to aim to add more ex-U.S. and ex-EU territories to optimize the international availability of both DANYELZA and omburtamab to pediatric patients.

Our bispecific programs under the Y-BiClone tech platform continue to advance as well. Our IND for nivatrotamab was cleared last year, and we are getting ready to dose the first patient soon in a Phase 1/2 study in small cell lung cancer. And in addition, we are planning on Phase 1/2 expansions in neuroblastoma and osteosarcoma later this year.

Our CD33, CD3-bispecific for pediatric AML is scheduled to enter the clinic in late 2021, again, addressing a very important unmet medical need for pediatric patients. The SADA Technology continues to look very promising. We’ve disclosed four targets thus far and lab work progressing as planned.

We plan to submit an IND, our first IND later this year, and we have received positive feedback on our pre-IND package submitted earlier this year. We ended 2020 with approximately $115 million in cash and subsequently closed the sale of our DANYELZA Priority Review Voucher for $105 million.

Of which, we got to keep 60% or $62 million after sharing 40% with MSK In addition, earlier this week, we completed and oversubscribed secondary offering led by JPMorgan and Morgan Stanley and Bank of America.

We sold approximately 2.8 million shares of our common stock, including the full exercise of the over-allotment option for a high quality book of investors at $41 a share and that enabled us approximately $108 million in net proceeds.

This further strengthens our balance sheet to not only support the commercialization of DANYELZA and the potential launch of omburtamab, but to enable us to advance both our next generation omburtamab and nivatrotamab into later stage clinical development.

At the same time, we continue to advance our two tech platforms with Y-BiClone compounds and the SADA compounds, and that’s progressing very nicely. So we are very pleased with our overall very, very, very solid financial balance sheet at this point, which Bo will elaborate more on this call.

Taking our achievements into consideration, we believe we have positioned Y-mAbs very well to expand our commercial activities while advancing our pipeline into clearly addressing very large unmet medical needs in 2021 and we are very excited to continue to do that. And with that, I’d like to hand it over to Dr. Moller. Thank you..

Thank you, Thomas, and thank you everybody for deciding to – choose to spend the morning with us.

During the fourth quarter, we have continued to work hard to ensure that our pipeline advances to what the market and our progress including having DANYELZA approved by the FDA, while at the same time making progress in the resubmission of omburtamab at BLA.

In addition, we have initiated a Phase 2 study with nivatrotamab in small cell lung cancer under our own IND initiated two Phase 1/2 studies, one in medulloblastoma and one in B7-H3-positive CNS/leptomeningeal metastases in adult patients in the CNS and advanced our ongoing studies.

We are also continuing to work on our new bispecific constructs and the SADA programs as well as our GD2, GD3 vaccine. Naxitamab, on November 25, the FDA granted us the approval of DANYELZA.

DANYELZA is indicated in combination with GM-CSF for the treatment of pediatric patients one year of age and older, and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow, who have demonstrated a partial response, minor response or stable disease to prior therapy.

This indication is approved on the accelerated approval regulation based on overall response rate and duration of response. The approval came a few days in advance of the PDUFA date, and we are thrilled to send out the first commercial vials to the treatment centers, including MSK across the country earlier this month.

So that was really a major achievement for the team. Let me pause for a second and acknowledge the team effort that made it possible to take this antibody from signing the MSK licensing agreement in August 2015 to becoming a commercial product on the shelf available to all applicable patients in the United States in February 2021.

It has been less than 5.5 years in total, and our employees, consultants, investigators, site staff and authorities deserve the highest credit for their tireless efforts in supporting and advancing DANYELZA through the development and registration.

As you know, a small universe of pediatric cancer centers, treat the majority of neuroblastoma patients. And we believe that we have built a lean and highly targeted commercial organization ahead of the launch.

In addition to the approval of DANYELZA, we have clinical trials ongoing with DANYELZA in Barcelona, and at MSK in New York for first-line neuroblastoma maintenance treatment, as well as chemo combination trials for refractory in neuroblastoma patients.

We are working to initiate an international Phase 2 multicenter trial for naxitamab in both frontline and in chemo combination treatments. We also have a Phase 2 osteosarcoma trial ongoing at three sites in the U.S.

And now turning to omburtamab, our second lead compound; in October, we received a refusal-to-file letter from the FDA regarding the BLA for omburtamab for treatment of pediatric patients with CNS/leptomeningeal metastases from neuroblastoma.

Upon its preliminary review, the FDA determined that certain part of the CMC module and the clinical module of a BLA required further detail. No additional non-clinical data have been requested or are required.

And it should also be noted that no additional clinical trials or recruitment of additional patients was requested in the refusal-to-file context.

We have maintained a very close dialogue with the FDA regarding the resubmission of omburtamab BLA and have scheduled a Type B meeting next month, where we hope to reach our final agreement with the agency on the remaining details before initiating or enrolling resubmission of our omburtamab BLA.

We remain confident that we can address all points raised by the FDA, including providing the requested supplementary data from Study 101, which will include tumor response data from patients with a variable disease among the patients included in this study.

We expect to resubmit the omburtamab BLA by the end of second quarter, or maybe in the beginning of third quarter 2021. The European marketing application for omburtamab has been prepared in parallel with the U.S. BLA.

And we plan to submit the marketing authorization application in late April of this year to EMEA and begin staffing our European commercial operations within the next few months.

As previously disclosed, we are also developing omburtamab for diffuse intrinsic pontine glioma known as DIPG in a Phase 1/2 study at MSK, and we are planning to open a multicenter Phase 2 study for DIPG patients shortly. For desmoplastic small round cell tumors known as DSRCT, we have recently opened a Phase 2 study at MSK.

Furthermore, in October last year, the FDA cleared our IND for the 177 lutetium omburtamab-DTPA construct for the treatment of medulloblastoma, which is one of the most common types of primary brain tumors in children.

177 lutetium omburtamab-DTPA embodies our naked omburtamab antibody radiolabeled with lutetium-177 using a DTPA molecule to chelate in lutetium radioisotope to the antibody.

Medulloblastomas are invasive, rapidly growing tumors, unlike most brain tumors, they spread through the cerebrospinal fluid and frequently metastasize to different locations along the surface of the brain and spinal cord. The incidents of medulloblastoma in the U.S.

is approximately 350 patients per year, and we deem this to be advantageous in finding our regulatory pathway to potential approval for the lutetium labeled on omburtamab-DTPA construct. We anticipate that our international multicenter Phase 1/2 trial will start screening patients with medulloblastoma later this quarter.

And based on our clinical experience with the iodinated omburtamab for B7-H3-positive metastasize in the brain, we are obviously excited to see 177 lutetium omburtamab-DTPA make its way to the clinic to potentially establish the safety profile and determine the maximum tolerated dose of this construct.

In this study, we hope to leverage our clinical experience from treating 27 medulloblastoma patients with the iodine-131 omburtamab construct, once again, using indwelling catheter for the intracerebroventricular drug delivery also called Ommaya Catheter and Reservoir.

In addition to the FDA, the FDA has also recently cleared our separate IND for a basket trial in B7-H3-positive CNS/leptomeningeal cancers in adult patients, where we hope to leverage our prior experience from treating more than 25 adults with the iodinated iodine-131 omburtamab.

We expect to initiate this study for the first adult patients to be screened and treat it with the 177 lutetium omburtamab-DTPA during the first quarter of this year. And we are thrilled to widen our clinical leads to include these adult indications.

As you also will recall, we announced that the FDA had granted orphan drug designation and rare pediatric disease designation to our leading bispecific antibody, nivatrotamab, for the treatment of neuroblastoma that happened in October 2020.

We submitted an IND for a Phase 2 study in small cell lung cancer in the fourth quarter of 2020 and the IND has now cleared. So we are excited to widen nivatrotamab’s clinical leads to include adult lung cancer patients.

In addition, we plan to expand the ongoing study of nivatrotamab at MSK into two separate Phase 2 arms, one in neuroblastoma and one osteosarcoma. And turning to the SADA Technology, we are very excited about the prospects of this technology, and we are making good progress on preparing our SADA targets for clinical development.

Our announced targets include GD2-SADA for potential use in GD2-positive solid tumors versus melanoma, small cell lung cancer or triple negative breast cancer; B7-H3-SADA which is intended for the use of treatment in prostate cancer; GPA33-SADA for the potential use in colorectal cancer and HER2-SADA for potential use in breast cancer, or eventually also gastric cancer.

And these first SADA constructs we expect to file the first IND for the GD2-SADA in the fourth quarter this year.

We believe the SADA Technology can potentially improve the efficacy of radiolabeled therapeutics in tumors that have not historically demonstrated meaningful responses to radiolabeled agents and we are truly excited about the SADA Technology.

So to sum up with the approval of DANYELZA and the launch already leading to deliveries to multiple treatment centers across the country and the planned resubmission of the omburtamab BLA coming up, we believe that we are well positioned to continue the development of Y-mAbs as a commercial stage company.

Concurrently, we plan to widen and deepen our pipeline by advancing our antibody constructs through the clinic, predominantly with the SADA constructs, the bispecifics and the next generation omburtamab-DTPA radiolabeled antibodies.

In other words, we have a lot on our plate for 2021, and we are excited to move forward and build a business that helps patients and fuels our continued development work. Now, let me invite Bo to share his remarks on the 2020 financials..

Thank you, Claus. We reported a net loss for the full year ended December 31, 2020 of $119.3 million or $2.97 per share basic and diluted compared to a net loss of $81 million or $2.30 per share basic and diluted for the year ended December 31, 2019. We were happy to report the first revenues in the history of Y-mAbs in 2020.

We reported net revenues of $20.8 million for the year ended December 31, 2020 related to our licensing arrangements in China with SciClone and in Israel with Takeda. There were no revenues reported in the year ended December 31, 2019. We incurred $2.2 million of royalty expense for the year ended December 31, 2020 related to our licensing revenue.

There were no such royalty expenses reported in the year ended December 31, 2019. As we take a closer look at the operating expenses for the full year 2020, we note that R&D expenses have increased by $30.2 million from $63.5 million for the year ended December 31, 2019 to $93.7 million for the year ended December 31, 2020.

This increase was primarily attributable to a $13.2 million increase in milestones and license related costs, primarily related to our acquisition of the SADA Technology from MSK, and the $13.4 million increase in personnel costs, the $1.6 million increase in outsourced services and supplies, and a $1.1 million increase in professional and consulting fees.

G&A expenses increased by $25.3 million from $19.5 million for the year ended December 31, 2019 to $44.8 million for the year ended December 31, 2020.

The increase in G&A expenses, primarily related to $8.9 million increase in personnel costs, $12.7 million increase in commercial expenses for our sales and marketing infrastructure related to the commercial launch of DANYELZA, and $2.1 million increase for business insurance, and a $2.1 million increase for professional fees.

Cash used in operating activities as per December 2020 show that the cash burn increased by $17.7 million from $73.5 million for the year ended December 31, 2019 to $91.2 million for the year ended December 31, 2020. The increase was primarily caused by the increase in the net loss for the period.

The net loss itself increased by $38.3 million for the year ended December 31, 2020. And it was partially offset by an increase in non-cash expenses, including depreciation and stock-based compensation of $20.2 million. We ended 2020 with a cash position of $114.6 million compared with the 2019 year-end cash position of $207.1 million.

As our work on the omburtamab BLA resubmission has progressed through the fourth quarter and we have continued to accelerate the commercial ramp up for the launches of DANYELZA and nivatrotamab has approved, we’ve seen our cash burn increase.

We expect the cash burn from operating expenses for 2021 to continue to remain roughly in line with the rate of the fourth quarter of 2020, where our operating spending was in the mid-30s. In terms of financial run rate, we recently completed a secondary offering and with total of $115 million to the company before commission and expenses.

This race together with the sale of the DANYELZA PRV that closed in January and was so far $105 million of which we get to keep $62 million after sharing 60-40 with MSK, and our 2020 year-end cash position of $114.6 million means that we now have an extended run rate. So we continue to believe Y-mAbs remain in a very healthy financial position.

This concludes the financial update, and I’ll now turn the call over to Thomas..

Thank you very much, Bo, and thank you, Claus. This marks the end of our today’s prepared remarks and I’d like the operator to open up the call for questions now. Thank you..

Thank you. We will now be conducting a question-and-answer session. [Operator Instructions] Thank you. Our first question comes from the line of Alec Stranahan with Bank of America. Please proceed with your question..

Hey, guys. Thanks for the questions, and congrats on closing out a solid 2020.

So first from me on nivatrotamab, how are you approaching the small cell lung cancer study given the various modifications that were proposed following the results last year from the past year trial? And I guess how do you see this asset sort of slotting into the treatment paradigm in both neuroblastoma and osteosarcoma given that DANYELZA may actually reach approval on these indications first? And I’ve got a follow up..

I’m not sure I got the last part, but you can get back to that. But for the small cell lung cancer study, the idea is to take patients that are second lines, mostly lung cancer patients, meaning patients that have relapsed after being treated in frontline or continue to progress.

And then as you for sure also know some of the PD-1, PD-L1s in your oncology treatments are approved in second line small cell lung cancer are most likely the patients would also have seen these and potentially also additional chemotherapy before they came – come on to nivatrotamab.

We are giving the product in combination with corticosteroids on a weekly basis – biweekly basis, sorry. And the idea is to give subcutaneous administration that should reduce further the side effects, potential side effect or risk of side effects from T cell activation systemically, cytokine release.

So that was the answer to the nivatrotamab, I think.

What was the other question?.

Yes.

And then just how do you see it slotting in maybe behind DANYELZA given they’re going after similar indications and a similar target?.

Well, you can say in the neuroblastoma setting, we are treating patients in third line that have been exposed to DANYELZA.

And as you can see also, when you look at the data from the 201 Study, we had a 68% overall response rate based on investigators, ORR evaluation leaving 32% of the patients not responding, and so there’s definitely still an unmet medical need in the neuroblastoma setting. So I don’t think it’s going to cannibalize in anyway the market for DANYELZA.

And as I said also, this is a very early stage study in neuroblastoma setting. So I don’t think it’s going to – I think it’s way too early to start discussing whether one would substitute the other one. One is a commercial product and it’s on the market and available on the shelf.

The other one is the development program that’s several years ahead of it before it could potentially get to a BLA approval..

Okay. That’s very helpful. And one more question, if I may, actually on the SADA platform.

Yes, your ability to sort of confine the radiation to the target tissue in preclinical models is definitely encouraging, but could you maybe speak sort of the residency time in other organs as its excreted? And what will be done in patients to manage this as the first product and where’s the clinic date this year?.

Yes. The actual construct per se is of course not a problem because it’s not toxic in anyway. So – and of course, if there is normal tissue expression of the target, the tumor binding part of the construct is binding to it. Then you may have some normal tissue binding out of the construct.

The first one is for GD-2, and we don’t think that there’s an issue with the doses that we’re working on in terms of side effect profiles from the GD-2 planning to eventually no side effects.

And we’re definitely now concerned about the radiation since we know that at MSK, they have given up to a very high doses of radiolabeled GD-2 antibody to patients in the past. So that are substantially higher than the doses we expect to use with the SADA.

So then when we shoot in the DOTA molecules with the lutetium-177, that’s supposed to bind into the DOTA end of the SADA construct that’s bound to the tumor. Then you can say, but what about normal tissue toxicity from the DOTA molecules were lutetium caged in a molecule and that’s a very tiny little molecule.

So, of course, it’s going to spread through the entire water phase of the patient’s body, which is typically 70% of an adult male and a bit less for an adult woman. But the thing is that this DOTA molecule will pass through the kidneys in circulation.

And every time it passes through, half of it is going to leave the body through the kidneys and into the bladder. Now bladder radiation is of course not very comfortable and you make radiation cystitis in the bladder from the bladder.

If you don’t put it in a catheter and make sure you continuously drain the bladder while the patient is having the circulating DOTA radiation construct in there, but I think that’s my only real clinical topic to focus on in terms of toxicity.

And in the pre-IND discussions we have had with the agency, they don’t seem to be utterly concerned about that side of it either. So, we are quite comfortable that we will be able to move this towards the clinic in the first quarter of this year..

Perfect. Thank you, and congrats again on the progress..

Thank you very much, Alec..

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

Hi, guys. Thanks for taking my questions and congrats on the quarter. Just a few from me, if I may. So my first question is around the omburtamab resubmission. I just wanted to get a sense what remains to be done with completing the resubmission package to the FDA.

And with regard to the Type B meeting, does Y-mAbs intend on issuing a press release on the results of that meeting? That’s my first one..

Okay. Yes. Well, I mean, we’re putting together all the data we have. One of the things that happened in December was there was a new paper published by SIOPE, the European Pediatric Oncology Organization, where they gave data from 63 patients with CNS/leptomeningeal neuroblastoma relapsed and shared all survival data that is precisely as rodent.

And for us as for all the other studies that has been published. The good thing here is that there are some more granularity and details from that database than there was available initially from the central German cancer register database. So now we have two sets of historical controls.

We have worked with SIOPE in January to make sure and agree with them that they would give us access to the data from that study, and we have gotten access to that, which the FDA seems to be very happy about. So now we have used that dataset also as second search historical control panel.

So that has been put together and submitted to the FDA in preparation for the Type B meeting at the end of next month.

When we have had that meeting and we received the minutes from the meeting that typically happens 8 to 14 days after the meeting, then of course we will come and guide what the outcome of the meeting was and what we expect that to mean in terms of the resubmission..

Okay. Awesome. Thanks, Claus. And my second question, so could you provide any additional color around the trial design for DANYELZA in the frontline neuroblastoma setting that sleigh to begin this year? For instance, I assume it’s going to be a non-inferiority trial against DANYELZA with event free survival being the primary endpoint.

But are you writing into the protocol, the exclusion of ASCT in the naxitamab arm explicitly?.

No, we’re not, because if patients have received bone marrow transplant, then they can come in still, but we are not requiring the bone marrow transplant. And as you have also seen previously, when Dr. Mora is treating patients, he doesn’t give them bone marrow transplant in the frontline setting.

So, it’s not that, but we will put it into the analysis section of the protocol that we would do separate analysis of the patients with bone marrow and without bone marrow..

Okay.

And considering the totality of the data since date for DANYELZA and neuroblastoma, at what time point do you believe you could potentially go to the FDA with some interim data to potentially request accelerated approval in the frontline setting?.

It’s a little too early for us to say, but I’m hoping in the second half of this year that we can have a Type B meeting with the FDA to discuss this. We still have a breakthrough designation for this construct in neuroblastoma. So we’re eligible for additional meetings.

And as you may recall, we have the 59 patients’ frontline study that was reasonably completed enrollment at MSK. And as soon as we have collected the data from that study, we would be most likely putting that together with the datasets from Dr. Mora study and asking for a Type B meeting.

But I see – let me now, some of the patients that we are treating patients that are actually in frontline. And as I’ve said before, I mean, that definitely at MSK, they would – I mean, why would they not continue to treat patients in frontline with naxitamab. But it’s not an approved indication yet, and we are, of course, showing that..

Yes, that makes complete sense. Thank you so much, Claus, and congrats again on the quarter..

Thank you very much. Take care, Rob..

Next question comes from the line of Etzer Darout with Guggenheim. Please proceed with your question..

Hi, this is Paul on for Etzer. Thanks for taking our questions. For DANYELZA, hoping to get some additional comments on how you anticipate the launch trajectory to shape out in the coming months and maybe in context of current COVID dynamics. And also you’ve recently partnered with license and distribution partners in China, Israel, Eastern Europe.

Can you help us understand the opportunity for DANYELZA and nivatrotamab in these particular regions and how applications might look? Thanks..

Yes. In terms of commenting on the launch, I can only say that, I mean, we will of course report on the first quarter sales when we get to our first quarter reporting. I’m happy to see that we have been able to ship to a number of sites outside of MSK also.

But MSK managed to just shortly ahead of the two next hospitals to come in and place the first order. But we have seen a very positive line to what’s accepting the use of naxitamab.

So very comfortable with the way this has panned out in the first three to four weeks, looking very much forward to report on the quarter when we have our first quarter conference call.

In terms of partnering, obviously, the most interesting part here is the Chinese partnering because China will hopefully grant us approval based on the pre-BLA package we have submitted in China to file for approval with BLA package, primarily consisting of what we filed to the FDA.

And if that’s the case, then hopefully in third quarter of this year, we can submit the Chinese BLA for naxitamab. And then what is the size of the market, as we speak now, that’s about 2,000 Chinese patients that each year received a diagnosis of neuroblastoma, and that’s very limited treatment available for these patients.

Now, pricing wise, I mean, you’re probably not going to be able to get more than 15% to 20% of the U.S. price, but with a market that’s threefold as big as the U.S. market. It’s still a very interesting market, also from a financial perspective. In terms of Israel, Israel is a very small country, but they also do have quite a bit of medical tourism.

I mean, it’s – I don’t think you’ll be able to see that the sales in Israel, hopefully – I hope you can see the sales in Israel as anything else, but a blip on our financial. Now the collaboration and distribution agreement with Swixx is addressing a relatively sizable market.

And in particular, there is in Russia, a new system set up where, there’s allocated a significant amount of hundreds of millions of euro to pediatric oncology treatments. And we are of course, pursuing to try to get under that program.

Did that answer your question, Etzer?.

That’s perfect. That’s very helpful. Thanks so much..

Our next question comes from the line of David Lebovitz with Morgan Stanley. Please proceed with your question..

Thank you very much for taking my question. You were talking earlier about the IND filed for omburtamab, targeting B7-H3 for adult indications. And that was just – I was a little confused when – because you initially had mentioned, I-omburtamab and then you transitioned to the Lu-omburtamab.

Could you just run through that again, the nature of the IND and what that trial is going to be?.

Yes. So the thing is that if we are going to address adults, and as I said, we have three to 25 adult patients with the iodinated version of the antibody. We needed a more, you can say easy quick way of being able to radiolabel the omburtamab construct. And therefore, we put a DTPA key data on the antibody.

So instead of having to put the antibody on a column and iodinate and wash and elevate, and then we have to recheck the binding because some of the tyrosine inside the iodine binds to resetting close to the entity and determining side of the antibody. The whole process ends up taking between 60 and 100 minutes.

Well, for a construct like the omburtamab-DTPA, the only thing I need to do is to pipe it off the amount of radioactive lutetium. So it takes me 10 seconds to radiolabel the antibody, because the isotope will bind only on DTPA and nowhere else. And you titrate, so you don’t put excess amount of lutetium, so everything will be bound with the antibody.

So that’s why we created that to be able to address the tens of thousands of adult patients that every year dies from CNS/leptomeningeal metastasis that are B7-H3 positive.

And that’s why we opened a separate IND for that, because now we’ve changed the isotope and we may be able to get higher than the 15 millicurie of radioactive iodine that we put into the heads of the kids and also used for the 25 adults we have treated with the existing omburtamab construct.

We may be able to get up so maybe 80 or maybe even 100 millicurie per dose that we put into the CNS of the patients.

Was that making it clear?.

Is there any I guess, reason why the efficacy would be different in adults than it would be in pediatric patients?.

I think the only reason why it would be more efficacious is if we can get the radiation up to a higher dose level in the adult patients.

But it simply, the key reason for going – since we are going after adult patients anyway, and we have to start with a Phase 1/2 dose escalation study anyway, rather than staying with the iodinated version, we felt that it was a relatively short delay to go for a DTPA conjugation and lutetium-177 labeling off of the antibody construct.

So the antibody binds exactly as the existing antibody, it’s just radiolabeled with a different isotope and it’s much faster and easier and cheaper to radiolabel..

Thank you for that. I guess, the other question is with respect to the upcoming FDA meeting, it sounds like you have the boxes checked off for the most part.

I guess, is there a possibility that you could come out of this meeting with more boxes, you need to check?.

What’s your experience with the FDA on such topics?.

I haven’t….

You never know. I crossed my fingers and hope that they see it as we see it, that these kids need this treatment and they need to get it approved for them as quickly as possible. So I certainly hope that, that they are not coming up with new boxes that we need to check.

And if they do that, it will be post-marketing commitments and they will just give us an accelerated approval based on the tumor responses that we have seen among the first 24 patients..

Thank you for that..

Thanks, David..

[Operator Instructions] Our next question comes from the line of Tessa Romero with J.P. Morgan. Please proceed with your question..

Yes. Hey, guys, how are you? Thanks for taking the question. Just a quick one from me as we’re thinking about the launch for DANYELZA, I think you noted, you started delivering the product beginning in February.

I guess, can you give us a little bit more of your sense on what metrics you’ll be providing the Street sort of to gauge the help of the launch? Is there anything beyond kind of sales top line that we should be thinking about?.

I think it’s too early to say what we will actually be able to provide you with.

But I would be surprised if we were not able to give you a bit of guidance on how many different centers that we – when we get first quarter results, of course, total number of vials that we have sold and total sales, and probably also the total number of sites that have started using DANYELZA for treating patients.

But I think that’s – as I said, we are very early on, we’re few weeks into the first sites receiving the vials, and unfortunately also a few weeks into the first successful treatment outside of MSK. So we are very excited and very happy with where we have managed to get DANYELZA, but it’s a little early to give more details on the launch.

So recently, what you were expecting to hear..

Yes, no, no, that’s helpful. It sounds like there’ll be some color beyond DANYELZA, which will be helpful. And I guess, my second question is a bit broader. You guys have a lot of trial initiations coming up here this year, along with your ongoing studies.

It would be helpful if you could sort of walk us through what data flow we should be expecting for the balance of the year, kind of for the key assets, for the key programs?.

Yes. I mean, of course, if you look at naxitamab, we will be providing updates of course, on additional submissions in like the Chinese submission and the initiation of the two more studies we are planning to start this year. And hopefully, we should also see some data when we get to SIOP from the osteosarcoma study with naxitamab.

In terms of omburtamab, of course, the resubmission to the FDA of the omburtamab BLA here in the second quarter, hopefully. We are still cautiously guiding that it could slip into third quarter, but my personal objective is definitely to get it done in the second quarter.

And the submission to EMA on April 30 would clearly also be the one of the key things that we would be looking for. The other things with omburtamab would be of course, the initiation of the multicenter DIPG study and hopefully, also being able at ASCO to report on the ongoing of the DIPG Phase 1/2 at MSK.

We are hoping to get an acceptance of a publication there. And then of course, the nivatrotamab, we hopefully will be able to give some update towards the end of the year, at our usual R&D Day in December on the status for the small cell lung cancer study and the status for the MSK study with nivatrotamab.

Then there’s the second bi-specific antibody, the CD33/CD3. And hopefully, we can announce that we have gotten an approval to start the AML pediatric oncology study with the CD33/CD3 bi-specific in the second quarter.

And then I think one of my real high hopes is that we managed to get this first out of construct into the clinic in the fourth quarter of this year, hopefully, in the beginning, but let’s see what happens when we file the IND and get all the stuff together.

There are always some challenges when you start with a new type of molecules on the CMC side and to get it manufactured and purified the right way and – but I think we have come a long way and I think right now as we speak, I have 20 grams of rock available on the shelf to conduct the talk studies that we need in preparation for the IND filing.

Was that kind of giving you a nice overview..

Absolutely. That was great. Thanks so much, and thanks for taking our question..

Absolutely. Have a good day Tessa..

Our next question comes from the line of Peter Lawson with Barclays. Please proceed with your question..

Thanks for taking the questions and congrats on the initial shipments.

Can you kind of perhaps give us any final detail around the number of sites that’s going out to beyond MSK? And any kind of initial feedback you’re getting from physicians about use of the product surprised you, either on the positive or negative side of things?.

I will give you more details when we report from the first quarter about number of sites et cetera. The only thing I can give of color is that I’m positively surprised that there have been absolutely no issues administering the product at sites that never have tried it before.

But we also have a very strong team of research nurses and MSLs and back-office support that can help decide to make sure that they are probably trained and prepared for administering the first doses. Was that okay.

Peter?.

Yes. That’s great to hear. That you look at the kind of issues around the drug.

But do you think, like when we see 1Q, it will be predominantly MSK or is it kind of indicating to you from the initial shipments that it’s clearly breaking out of just MSK?.

I hope the trend continues and then it means that it’s breaking out from MSK, but let’s wait and see..

Thank you. And then just for the resubmission of omburtamab, so what could be the potential outcomes there, what are the range of outcomes from the Type B meeting and associated kind of refilings? I know it’s – they want more data, they want a post-marketing commitment if you kind of run –.

Yes. As I’ve previously indicated, the FDA had two issues with the Refusal to File. The one was that they felt that the historical control group did not have sufficient granularity and they won’t in that propensity score analysis on the patients which requires you have a more granularity, of the individual patients and their previous treatments.

We initially saw that would not be possible with the central German Cancer registered database. In between they have actually done a tremendous amount of collaborative work for us. And Germany had provided us with way more details and we had hoped they would be able to.

And as I also mentioned earlier, unfortunately, there was this new study from SIOP that came out in December as a publication where they have granted us access to that database, including very granular datasets from the patients in that study.

So I think we can address the FDA’s wish for additional granularity on the control group data for historical control. Should that not be the case, they have the other possibilities to say.

But on the other hand, now you have 24 patients with six months follow-up full datasets, including 10 patients with tumor response data from the 101 multicenter study.

What we could do is to call the historical data and the MSK study historical supportive data for an approval, based on the tumor response rate of 40% in your one-on-one study and then we would give you a post-marketing commitment to provide additional and progression-free survival, but that’s kind of like the two past they have to follow and we are suggesting that they continue with the original past when they I’ll have more granularity.

The multicenter study is supportive and as well as the tumor response data are supportive and that new data controlled patients from the study together with the additional granularity from the German Cancer Register should be sufficient to support this. Is that clear..

Yes, that’s definitely helpful and then just around kind of the OpEx, how should we be thinking that I think you mentioned that the cash burn and 21 remain in line with 4Q..

Yes, roughly in line with what we were spending in the 4Q so mid-30s, maybe a little bit lower per quarter..

And does that account for the like the vouchers just sales?.

No, that’s the operating cash burn. So any income would be deducted from that to get to sort of a net level..

Got you, Bo..

And the revenue partnerships mobile shares included in that spending..

Great, thank you. So that’s kind of a clean them. Thank you so much..

Our next question comes from the line of Sebastiaan van der Schoot with Kempen & Company. Please proceed with your question..

Hi, Claus. Hi guys, congratulations on the full year results for 2020. I just have two questions, the first one is on omburtamab – you mentioned that you at the previous experience with iodine version. I was wondering what the clinical responses were in that certainly, with that particular agent in those patients..

In the medulloblastoma patients, we have had a number of patients that seem to stay in remission. Again this is given on the top of a standard regimen, including crane spinal radiation and induction chemo and for some of the patients, even some surgery.

But it is clearly the – our opinion and also the investigators of MSK that this is contributing significantly to keeping these kids in remission.

Unfortunately, we will not be able to put everybody into remission like and lasting remissions like it is within neuroblastoma, but if we can get to a 50% response for a long-term survivors situation in these patients. We have made a huge leap forward for these patients. But there has been very limited – Dr.

Kramer presented a little bit in – at SIOP in 2019. There was a presentation, but it’s primarily safety data.

Okay.

And then what’s for the adult population you were talking about?.

The same for the adult 25 patients that there is limited information available.

But there has been a number of patients that have responded positively, in particular recall a ovarian cancer patients that had 14 metastasis in the brain that had all of them shrinking or disappearing after the first cycle with impertinent, in spite of being treatment resistant to everything else that she had been receiving, and she lived for another 18 months, normally don’t live for 18 months with 14 brain meds from any cancer..

Okay, great. Thank you. And then regarding [indiscernible], I was wondering whether you could comment on when we could expect to complete dataset for that trial and whether that will be enough, you think for accelerated approval in….

Yes, as I told at the R&D Day in December, we had at that time point 31 evaluates patients and they’re still looking at least as promising as we would like it to look. And we have added two more sites [indiscernible].

And we are looking when we have the first 39 patients, we will be looking at whether we should continue with a particular sub group of the patients where osteosarcoma relapse, i.e., where the majority of them is, and that’s the lung meds or whether we should continue with everybody.

But that’s a little too early to say where we will end up, but hopefully we will have a dataset of the 39 patient, we can present at SIOP in October this year, alternatively, we will give an update at the R&D Day in December..

Okay, great. Thank you..

A formalized supplementary BLA for this, we need to see the data first..

Okay. Thank you..

You’re welcome, Sebastian..

Thank you. We have no further questions at this time. I would now like to turn the floor back over to management for closing comments..

Well, thank you everyone for listening today, and I hope you agree we had a very exciting 2020, and we look forward to 2021, and thank you Bo; and thank you, Claus. Have a great weekend everyone..

Thank you, take care everybody. Bye..

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