Q4 2019 Earnings Call
[music].
Greetings and welcome to the why not.
Full year 2019 earnings conference call at this time, all participants are in listen only mode.
Brief question answer session will follow the formal presentation.
And then you want to require operator assistance during the conference. Please press Star zero on your telephone keypad. As a reminder, just conference is being recorded.
It is now my pleasure to introduce your host Thomas Scotch Chairman and President. Please go ahead.
Thank you Ashley good afternoon, everyone and thanks for joining us on this particular today.
So 2019 was the other we believe we made significant progress on all strategy and took steps.
Additionally, it's very well.
<unk>, that's we work to complete two P.L.A. submissions.
Prepare for true potential product launches watts significantly expanding our clinical activities.
We ended 2019 with 207 million in cash.
And that's you know in late October we completed a secondary offering led by Morgan Stanley.
I'd be Morgan unbearable.
In which we raised approximately net proceeds of 135 million to the company.
These proceeds.
Further strengthened our balance sheet.
What a potential launch of both in the system at an apartment later this year.
Well at the same time advancing our pipeline.
Would carry us through to the end of 2022, and that's without taking into consideration any product sales for any potential partnership wrapping up.
We are very piece would all financial position as it is today.
That's it about and a burst hubs have both previously we see record aspect to seize designations from D.F.D.A.
And then December our vaccine was granted record after deceased designation as well.
So that paves the way for potential sort priority review about true.
I see them, having a burst have continued to read up encouraging data.
Addressing clear unmet medical needs for neuroblastoma patients.
And we have now shown that we're able to reproduce these results in multicenter trial.
Today enough to them up and on both of my treating children worldwide in clinical trials, we couldn't the U.S.
Canada.
Europe, and Hong Kong and we believe this global outreach is a major achievement for why much.
We spent approximately 75 million on operations in 19.
And we think Oh spending is a direct result of our highly dedicated teams who are very committed to making both see somebody when a person were available to children everywhere.
We're working hard on Oh on expanding ongoing clinical activities for both of these antibodies addressing osteosarcoma on T.I.P.G.
We actually unmet medical need success for pediatric patients.
We also working hard on opening all global trials for vaccine, Yeah, 2020, as an add onto the system up in order to provide access to children worldwide.
Going beyond that sitting next to them up an embarrassment, we advancing our two plus one Bible that low affinity C.D. suite by specific platform.
Which we're very excited about Oh.
Our first five left D to C suite product is currently in dose escalation in a phase one single center clinical trial at M.S.K.
It'll be very interesting to see data from this study potentially validating our platform and expand into larger indications with future partners.
We also working hard and all second five led by specific which we have announced the CD 30 suites cdthree focusing.
On potential is fighting it out and I. Indeed, this year I'm focusing on email again, another unmet need for children.
As announced we filed an idea on first met conjugated looks you some late.
Late last year in order to get on next generational burst into the clinic.
Yeah, we're working on it.
Two part strategy opening up more two cents a trials one from a do love to still Mcconnachie patience, well faced with a clear unmet medical need a second trials will be 73 primary too much.
Metastasized to the brain.
Hey, how all of achievements into consideration.
We believe why not just very well positioned said, we're very excited spring brings a company forward in 2020.
That's a company we all wish continue to stay through to work off.
Fishing loss as a leader in pediatric oncology addressing climate medical needs and focused on advancing all that's ever used to reach the lives of children living with these rare cancers.
And with that I'm very pleased to introduce your Doctor Kashmoula our CEO. Thank you.
Thank you Thomas and will come through I'm not sure fixtures. After 19 any calls you treat the do you have chosen to join US today and during the fourth quarter. We have continued to work.
I'm sure that I would need to product candidates like set them up and put them up and wants to whats completion up have expected feeling submissions.
Submitted the first portion of the voting yeah. They come next enough in the then but 2019 and be expected submission to be completed within the next couple of weeks.
One thought it might be recent they had to be dealing meeting with FDA and do a very pleased to have all plants come from Saudi agency, we expect to complete all voting on but it must be it makes it much in may meeting that all project, it's all commercialization timelines on chains.
We'll continue to keep you posted on our progress. He believed that we have continued to demonstrate our ability to execute on our commercialization plans through the fourth quarter yeah.
This year with Warner why it's being on everyone's mind. These days I want to stress that the company has taken but beat the into the all necessary precautions to minimize the contamination basically all facilities. This includes all types of deals and I couldn't be believed that our contingency plans will enable us to ensure submission of folks the next.
Let them up and down but not nearly in line with the timelines I just mentioned.
Let me turn to an extent in that in addition to there won't be electronic set them up and good after factoring all still HMA had some exciting news in frontline goal still much at all on D. day in December they'll come off in basketball, that's children's hospital, well has significant experience treating primetime almost all the patients with both makes it a month and also a competing anti cdtwenty by.
He kinda talk so they tend to clinical data from both antibodies. This of course, if an exit them up front line data presented data from this investigator sponsored looks at a much study up 34 patients harvesting twond, all but still not completely mission show a 72% eventually survival rate at 24 months.
This compares favorably with that I'll pick the.
Published data from the last it pays off 63% or 24 months.
And actually survival.
He said that in 89 patients treated with dying to talk to my that Buddy.
That's it about treatment appear to be well tolerated than the frontline study and infusion per day and generally associated to the insight you can you actually been quite significant and that's opiates phonics. It I'm, but then they the competing entity to everybody goes by Dolphin mall, they use it multi multi carrier and only declined significantly up.
Post treatment cycle and Dr. Morris finding what's the next set them up only declined less than 20% and we'll find those required by the competing actually did you want to play.
Basic and sometimes studying Linux it about this joint the ongoing at Analyst Day in New York City have you expect data from just entropy published later this year. They also planning that moved to send to frontline study, which we expect to start later this year.
At the International Society of pediatric oncology all this I have conference last October trends, a total of 6000 patients by in this case provided substantial exposure to an accident encouraging clinical data.
Among the highlights what they do have some primary effectively how did they smoke still not patients and study 12 to see these patients every factory to intensive induction therapy and in addition, more than half the patients fall. So we feel free to second line chemotherapy.
And this subset of patients Oh, we demonstrate better than expected outcome, including 78 cents. All this funds rate and other subset analysis stratify patients with relapsed multiple myeloma, well loved assistant to salvage chemotherapy.
30 patients way valuable and also these patients so once the patients had to a whole prior been collapses and 89% had previously we see that each when she body before they came on Mexico.
These patients coming into next set them up at a 37% or response rate and a 36% progression free survival rate two years, all 24 months, which indicated pinnacle benefit and it's difficult to treat population.
We also presented data from harvesting office that there's still more patients and secondly, I made a complete remission.
44 patients window, whether that's a disease with treated with C. N. C is that sit on that as maintenance therapy to keep going into Michigan. In this population, 88% off the patient had previously we see that's I think you choose therapy and 30% have to see two or more lines of things that you get from CRP before we see accident.
A two year progression free survival rate, 52% bicep, so even need these data of the rain watching since these patients are known to have short locked in response is one of your progression free survival typically about 24 cents.
Also had data to patients would be ses restricted to the bone marrow can pop and.
We've been very interesting, it's investigators assist 100% feeling the bone marrow in this patient population I don't believe that's ever been seen before they didn't have missed on the trial.
Now, let me turn to about a month.
Oh, secondly, compound and put them up as a way to me I didn't want to 31 antibody and as previously disclosed the opposite often this compound christiana flipped to mitigate metastasis from both installment.
But also for diffuse intrinsic sometimes I am on it yet the D and definitely drastic small launch when most.
Also known as this business itself between all of these two must be set an extremely positive.
Considerably we had a pre DNA meeting.
And with the FDA confirmed all plans for filing it'd be like on but about the plan a rolling submission, which we expect to compete in me competing submission for the year. They fund accident that in late March and put them up shortly thereafter reflects an outstanding performance of our team and we hope to see those compounds approved Buddy.
Before the end to figure.
In December email agreed to all proposed pediatric investigational sites on bottom that that's a positive regulatory European process for registration I've never seen companies I've a flat to provide.
Outlining their strategy for investigation of new products in pediatrics.
Patients.
It is prerequisite for filing a marketing authorization application for anymore.
And be in Europe.
We believe that did not always a tier possible district enough and put them up for the treatment of pediatric patients with CMS kept when they come to taxes from old criminals are still in Europe, and we plan to submit a marketing authorization application into fourth quarter. This year.
This is a bicycle steps for what you know, it's watch shipping and put them up to the European Buck and it demonstrates the coast constructive interactions with the administer established for the European regulatory facilities.
Now turning to the data generated a minimal in November last year that that's the plastic smoke.
Falls off cell tumor data was presented at the 2019 connective tissue oncology Society or C test annual meeting in choppy with Japan USIS hostage. He isn't a music malignancy that could be a sense as interim government all talking about children and young males less than other patients are diagnosed.
Yeah, the U.S. and patients with the exercise it she had very poor prognosis limited five years survival. So definitely on the medical need in this population even.
Even the most fortunate yes itself to teach and wait for the two less total section.
Five years survival appears to be approximately 20%.
Based on observations from an escape Oh abdominal intensity money, maybe they come syrupy make your advisable for both patients whose to Mckenzie per se.
The data reported backed off the Modatek Cetus, let's based on evaluation surety treat patients at an escape a total of 24 patients from phase one study, what we see the radiation to the abdomen and in combination <unk> into appear to be really administrated, but I'm not I'd.
Nice patients, who just thought the treatment I would be a dominant remediation opta told to over 60, but no.
The study showed that the 41.
41 month.
Median overall survival for those who did not receive them, but about and 59 months, so those who received.
So the data presented at sea could show that adding I didn't I did but I'm not sustainably disrupting was well tolerated indicated in our opinion a improved survival.
The most adding about them up to a section improve the five year Kaplan Meier made all survival from that historically reported laid up approximately 20% to approximately 40%.
Were pleased to see de survival data next the valuable to cease means that survival is the only relevant and finding these patients. While this approach may not how.
Patients, who do not but she wants to mother section. We believe that it may help patients with microscopic diseases, which is exactly where if anybody has its strengths.
We believe that the data illustrate the which could potentially use oh wait till they that's not which could potentially be applied to other future meal or compartmental. Many currencies you recently initiated a phase two trial.
She had him escape and be a planning to open. This study for a small exploratory group of folks gastric cancer patients and also ovarian cancer patients.
It's I'd be presented an updated and put them out data readout from study Oaktree lunch retreat at an escape what patients with CNS, let somebody that metastasis from office don't ever see two doses of the Julie.
Data show that for the 170 dialysis patients. The median survival had decreased to 50.8 months, where the final and not get speech. This compares to the previously reported 47.1 months, but you had nice to treat patients in this study. So we're pleased with is healthy remember that historical need to answer that.
These patients is approximately six months.
In addition, we had 68 patients diagnosed with other sienna cancers, including metastatic too much who had to see a total of 201 injection cellphone button that injections for which he did minutes, David primarily in outpatient setting.
On the patients primaries units back most included eight different cancers, including 27 patients, but I do look at Stilmar nine patients.
Moma patients when that aesthetic too much for different primary Kansas nine patients with Tacoma, and five will minimize as of today.
Six of these 68 patients with these I didnt equal diagnosis analyze these results People's all pendulum to teach them how to teach them label onboard them up construct for which we have filed to 90 in December.
First studies will be.
Well not looking ahead.
Thanks to the previous experience from 27 patients would be I need to construct.
In addition, we tend to submit a separate I'd ask a trial. He said the athree prostitutes units left to many pretenses in adults so never rates from our experience of treating them more than 25 adults with yard.
But.
Operator: BF-WATCH TV 2021 Greetings, and welcome to the Y-mAbs Full Year 2019 Earnings Conference Call. At this time, all participants are in listen-only mode.
We expect to treat the first and ultimately to teach me becomes drops.
In the second topic Twentytwenty that'd be a team I'm thrilled to buy our aim to include I don't see syndications <unk>.
Operator: A brief question-and-answer session will follow the formal presentation. Should anyone require operator assistance during the conference, please press star zero on your telephone keypad. As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Thomas Gad, Chairman and President. Please go ahead. Thank you, Ashley.
To support our goals plans, we increased headcount by approximately 16% to a total of 65 countries. During the fourth quarter increase is primarily due to the development team and the commercial team backing off for the potential bombs ethnic set them up.
Thomas Gad: Good afternoon, everyone, and thank you for joining us on this particular day. So 2019 was the year that we believe we made significant progress on our strategy and took steps that position us very well for 2020 as we work to complete two BLA submissions and prepare for two potential product launches while significantly expanding our clinical activities. We ended 2019 with $207 million in cash. And as you know, in late October, we completed a secondary offering led by Morgan Stanley, J.B. Morgan, and Bama, in which we raised approximately net proceeds of $135 million for the company. These proceeds have further strengthened our balance sheet for the potential launch of both NACISIMAP and BIRSTMAP later this year, while at the same time advancing our pipeline, and should carry us through to the end of 2022, and that's without taking into consideration any product sales or any potential partnership revenue. We are very pleased with our financial position as it is today.
We continue to believe that can be a well position to move those actually them up and in Baltimore, Judy after the approval and commercialization in 2020 and concurrently increase our focus to our states pipeline candidates.
Thomas Gad: Nacitamab and Ambersimab have both previously received rare pediatric disease designations from the FDA. And in December, our vaccine was granted rare pediatric disease designation as well. So that paves the way for a potential third priority review.
As I mentioned on the teaching label them, though to my P. construct the bi specific programs and the teacher would you be treat vaccine as well as the next in line indications for next it I'm.
No I didn't mean whiteboard to share his remarks on full year 2019 financial results well.
Thank you close we reported a net loss for the year ended December 31st 2019 over $81 million what she wants.
<unk> dollars per share basic and diluted compared to net loss of 40 frequency moving on one of the hospitals because share basic and diluted to submit into December 30, plus 2% 18.
We ended 2000 and matching the catch this additional children said a minute compared to the third quarters, ending cash position of $98 million <unk>.
The increase of 109 billion was primarily due to net proceeds from our secondary offering Rafi under 35 million thought it was nice.
In November 2019, offset I suppose corn expenditures.
So we're working to be a nice missions. So much cheap enough on the bottom up of courses and we accelerate to commercial went for the potential no.
So to meet compounds, we've seen El Cajon cranes, and we expect the catch their income operating expenses for 2020 to remain roughly in line with the first quarter was 29 team.
Thomas Gad: The CETA map and On Birth Map continue to read out encouraging data addressing clear unmet medical needs for neuroblastoma patients, and we have now shown that we are able to reproduce these results in multicenter trials. Today, AnesthesiaMap and OnBirthMap are treating children worldwide in clinical trials, including the U.S., Canada, Europe, and Hong Kong, and we believe that global outreach is a major achievement for Y-mAb. We spent approximately $75 million on operations in 1990.
Research and development expenses increased 29 billion from 34 million hedge funds ended December 31st 2018 to 64 million for the 12 month ended says most every sales person monkey.
This increase was primarily attributable just seven she'd moving steadily increasing outsourced manufacturing saw chewed each product candidates actually them up in a bottom up.
Thomas Gad: And we think our spending is a direct result of our highly dedicated teams who are very committed to making both Narcissus and Ivanoprosimov available to children everywhere. We are working hard to expand our ongoing clinical activities for both these antibodies, which address osteosarcoma and DIPG, where key unmet medical needs exist for pediatric patients. We are also working hard on opening up a global trial for our vaccine here in 2020 as an add-on to Nacifimab in order to provide access to children worldwide. Going beyond the treatment of SIDAMAP and ombudsman, we are advancing our 2-plus-1 Bible and low-affinity CD3 bi-specific platform, which we are very excited about. Our first Bivalent DD2CD3 product is currently in dose escalation in a phase 1 single center clinical trial at MSK.
A $5 million increased outsourcing research and supplies to support spending developmental students is $3 million increase and personal cost that's woodenhouse moving jelly gruesome clinical trial expenses.
<unk> expenses increased petchem and a healthy and mundane so the yeah. That's a sympathetic supposed to call. Some 18 to 19 in the hospital in several years, it's a fairly close to plasma Nike.
Increased the Gionee expenses, primarily reflect a 5.1 billion dollar increase in personnel cost 2.8 million Donna.
The expense for big ramp up costs, all commercial infrastructure when you get some contingent voltage also brought him on next year.
Cash flows from more breaking it keeps his show that they catch spread.
The increase that's 32 million. Some 41 million suddenly you ended December 30 says 2018 to 74 million. So be yet you did just said before the first 2019.
Thomas Gad: It will be very interesting to see data from this study potentially validating our platform and expanding into larger indications with future partners. We are also working hard on our second bi-specific, which we have announced, the CD33-CD3, focusing on potentially filing an IND this year and focusing on AML, again another unmet need for children. As announced, we filed an IND form birthmark conjugated with leukemia late last year in order to get our next-generational birthmark into the clinic. Here we are working on a two-part strategy opening up multi-center trials, one for medulloblastoma pediatric patients who are faced with a clear unmet medical need and a second trial for B7H3 primary tumors that metastasizes to the brain. Taking all our achievements into consideration,
The increase was primarily caused by the increase in the net loss for the the Nicholson so increased by $37.8 million hadn't yet into December but it's supposed to close no munshi, most partially offset by aside from $5 million increase in accounts payable and accrued liabilities saw meaningful increase overweight.
And it includes noncash expenses, including stock based compensation of 2.7 billion.
In addition cap spending from investing activities increased by 1.7 billion to 2 billion in 2019 and potentially some equipment to marry said them all in all new jobs since you best than that.
The net proceeds from the issuance of common stock increased.
And 6 million from that's a 9 million in 2018, well, even public swampland 75 million, who needed to I'll follow on secondary Olson, but chose to the fourth quarter 2000 Momsen.
Thomas Gad: We believe Y-mAbs is very well positioned, and we are very excited to bring the company forward in 2020. And as a company, we will always continue to stay true to work hard. As a leader in pediatric oncology, addressing clear medical needs and focus on advancing our therapies to improve the lives of children living with these rare cancers. And with that, I'm very pleased to introduce to you Dr. Klaus Miller, our CEO. Thank you.
In terms of financial guidance, we've said since the secondary offering but the cash on hand, plus net proceeds from gilson.
Well.
Our offering.
But just in terms of expenditures through the fourth quarter 2020 true on this still does not take into account any potential revenues from conversation, that's actually going up and add them up all the proceeds from any potential future partnerships. So we do believe why matched remains in a very healthy financial position.
Klaus Miller: Thank you, Thomas, and welcome to Y-mAbs Therapeutics' 2019 Early Calls. We are pleased that you have chosen to join us today, and during the fourth quarter, we have continued to work hard to ensure that our lead product candidates, Maxidermab and Omberdermab, advance towards completion of their respective BLA submissions. We submitted the first portion of the rolling BLA for next summer in November 2019, and we expect the submission to be completed within the next couple of months.
This concludes the financial update and I'll now turn the call your budget films.
Thank you Bill.
I think we'll now take some questions.
Actually the pieces.
Absolutely.
Klaus Miller: For BODEMAP, we recently had a pre-BLA meeting with the FDA, and we were very pleased to have our plans confirmed by the agency. We expect to complete our voting on the BODEMAP BLA submission in May, meaning that our projected overall commercialization timelines are on track. We will continue to keep you posted on our progress. We believe that we have continued to demonstrate our ability to execute on our commercialization plans through the fourth quarter of this year. With the coronavirus being on everyone's mind these days, I want to stress that the company has taken what we deem to be all necessary precautions to minimize the contamination risk at our facilities. This includes all sites in the U.S. and Denmark, and we believe that our contingency plan will enable us to ensure the submission of both the Nexidermab and the Ombredomat DLA in line with the timelines I just mentioned. Let me turn to Nexidermab.
We will now be conducting a question and answer session. If he would like to ask a question. Please press star one on your telephone keypad confirmation telling will indicate your line isn't that question can you maybe press star to if he would like to remove your question from the Q for those using speaker equipment and may be necessary to pick up your hand chapter for price.
During the start keys.
One moment, please call me poll for questions.
The first question is from Alex trying to hand of Bank of America. Please go ahead.
Oh, Hey, guys. Thanks for taking my questions I'm Congrats on the completed 19.
And the previously meeting for for on Bert Mab. Soon so just on that first could you give us a sense of the additional studies if any of that need to be done for the rolling feel a for on Burton map and now with a clear picture a feel they submission timeline I'm. Following your interactions with the FDA do you think it's still.
Klaus Miller: In addition to the rolling BLA for Nexidermab in relapsed refractory and norepostoma, we had some exciting news in frontline norepostoma. At our R&D day in December, Dr. Mora from Barcelona Children's Hospital, who has significant experience treating frontline norepostoma patients with both Nexidermab and also a competing anti-DD2 antibody, Dimethox This is the first-ever Nexidermab front-line data presented. Data from this investigator-sponsored study of 34 patients with high-risk stage 4 neuroblastoma and first complete remission showed a 72 percent event-free survival rate at 24 months. This compared favorably with the published data from the Lancet paper of 63% 24-months relapse-free survival, or progression-free survival, in 89 patients treated with dinatoximab. The Nexidermab treatment appeared to be well-tolerated in the front-line study, and the infusion-related pain generally associated with the anti-DD2 antibody required significantly less opiates for Nexidermab than with the competing anti- The use of morphicure generally declined significantly after the first treatment cycle, and Dr. Mora's finding was that Nexidermab only required less than 20% of the morphine dose required by the competing anti-DD2 antibody.
So reasonable to assume commercial launch by the end of year.
That I've got a follow up.
Okay. Thanks, I like this oh, yeah, I mean, I'm just could be careful on but up the S. A lucky filing any additional studies for the approval.
What do you have implying is that the multi center study the opened at the requirement from the FDA and what we have about 18 patients are so actually we had almost ODP study now, but the first cohort of patients will be supporting the theme today that will be completed in may.
Yes, he is giving us what they call them accelerated approval, which means that they will be postmarketing commitment.
In the post marketing commitment is not the filing any additional studies the class that's a patients in this study one or one where the first cohort of patients as a part of Thats worth the pool should be fault for three years and he has to come up the tree get all survival data that is significantly better than historical data, which is estimated to be about comparison. So they know.
Patient could meet to be for the three your follow up with about 32 patients.
And I think you're pretty close to having dose for the two patients in this study.
No I think that's 28 29 patients.
So we're pretty close to that and I think that it's not as requirement for the actual filing they have all the information they need for the body and be a getting ready to submit everything up before the end of me.
Klaus Miller: A second front-line study with Exitema is currently ongoing at MSK in New York City, and we expect data from this study to be published later this year. We are also planning a multi-center front-line study, which we expect to start later this year. At the International Society of Pediatric Oncology, or the SIOP, conference last October in France, a total of six presentations by MSK provided substantial exposure to exciting chemical reagents. Among the highlights were data from primary refractory high-risk normal stomach patients in study 12-230.
In terms of launch time, yes, we spoken to eat all that we can do that connects set them up we expect to complete the Vulcan DNA before the end of this month, which tend to have a PDUFA date, most lucky that though and be able to hit some office and that's I guess I'm guessing if we filed the.
And what am I feel they in.
Klaus Miller: These patients are refractory to intensive induction therapy, and in addition, more than half of the patients were also refractory to second-line chemotherapy. In this subset of patients, we demonstrated a better-than-expected outcome, including a 78% overall response rate. In another subset analysis, 35 patients with relapsed nervous stoma who were resistant to salvage chemotherapy, 30 patients were evaluable, and also these patients, we saw one-third of the patients had two or more prior relapses, and 89% had previously received These patients coming on to Nexinimab had a 37% overall response rate and a 36% progression-free survival rate at 2 years or 24 months, which indicated 44 patients with no evidence of disease were treated with CMCSF and maxilumab as maintenance therapy to keep them in remission.
Maybe we should get a it is a day most likely in January 2021, how can be done.
That's a chance along this year well typically the F.D.A. for these kinds of <unk>.
Addresses small life threatening diseases.
HM.
We approved.
Oh I want to too much lately, so definitely for the next enough.
Potentially if they see a I'm used to spend the time minus the typical wanted to multiple vehicle. We could also I see pumping them up on the last month this year.
[laughter], Oh, I'm, sorry nicholson's.
Yes, that's that's very helpful. Thanks, and one more for me so assuming nexsan about gets approved in relapsed refractory neuroblastoma banana. This year have you gotten a sense from the F.D.A., what the expected regulatory path in front line neuroblastoma would be how do you think the timing of the M.S.K. study readout.
Influence the multicenter studies on X.
I first of all we don't habits.
Klaus Miller: In this population, 88% of the patients had previously received anti-TB2 therapy, and 30% had received two or more lines of anti-TB2 therapy before receiving Nexidermab. A two-year progression-free survival rate of 52% was observed. We believe these data are very important, since these patients are known to have short-lasting responses with one-year progression-free survival of typically about 20% or so. We also had data for patients with disease restricted to the bone marrow compartment, which we deem very interesting, as investigators assessed 100% clearing of the bone marrow in this patient population. I don't believe that this has ever been seen before in any neuroblastoma. Now let me turn to on from bottom up.
Discuss if you haven't had discussions in the agency about the frontline and I simply decided not to start discussing that until we get the approval in second line.
You will have paid out there from two individuals in defense of study already this year and people can doing a books and the study starting this year.
I think it.
It's highly likely that that's the data multi out that I think you can see at least.
That's something that's it they're interested in using Mexican that's in front line.
Whether that will happen.
I cannot give any guarantees and work towards a formal regulatory approval to make sure that reinforcement.
Klaus Miller: Our second lead compound, ombretomab, is a radio-labeled iodine-131 antibody, and as previously disclosed, we are developing this compound for CNS-lectin-molybdenum metathesis from norepnostoma, but also for Diffuse Intrinsic Contamination, also known as DIPD, and Desmoplastic Small Round Cell Tumor, also known as DSSRCT We plan a rolling submission, which we expect to complete in May. Completing the BLA for Nexidimab in late March and ombudamap shortly thereafter reflect an outstanding performance by our team, and we hope to see those compounds approved by the FDA before the end of the year. In December, EMA agreed to our proposed Pediatric Investigational Plan for Omberto Malt. As a part of the regulatory European process for registration of new medicines, companies are required to provide a plan outlining their strategy for investigating new products in pediatric populations. And an approved PIP is a prerequisite for filing a marketing authorization application for any new product in Europe.
Great. Thank you.
[noise]. The next question is from answered Darville asked Guggenheim. Please go ahead.
Great. Thank you for taking my question Congrats on the progress just a couple of questions I guess, one maybe on sort of the data disclosures on for 2020, given some of the disruption that we're seeing and what your thoughts are around sort of maybe it's a venue or whether or not you know sort of changed.
The venue by which you you'd just supposed to data and then maybe on the so the global trials on short for sure the vaccine and maybe if you can sort of talk about what that pivotal trial could look like as far as design. Thank you.
Well it seems a venue I mean, I think they're all a little bit out and of which right now what kind of happen through various <unk>.
I think for sure.
True lots and meeting is going to happen in April this year. The meeting in May that you're planning to use as presentation for a number holiday.
Submitted abstracts at the you know and that's the then let's wait and see but you have to pay down of Chris who.
Make an effort to make sure we get paid out as quickly as possible. So everybody's I'd like to talk about behalf.
Klaus Miller: We believe that there is now a clear path for registration of OmbertaMap for the treatment of pediatric patients with CNF-Glyptomeningo-metastases from normal stoma in Europe, and we plan to submit a marketing authorization application in the fourth quarter of this year. This is a vital step forward in our efforts to bring Oncotumab to the European market, and it demonstrates the close, constructive interactions that we have managed to establish with the European regulatory bodies. Now, turning to the data generated with OmbertaMap, in November last year, the desmoplastic small bowel cell tumor data was presented at the 2019 Connective Tissue Oncology Society, or CITOS, annual meeting in Tokyo, Japan. DSSRCT is a malignancy that typically presents as intra-abdominal sarcomatosis in young males.
But I'm pretty sure what with the other part of your question.
So yeah around sort of the trial for the global travel to them that you spoke about for vaccine yet from for the bathroom could look like yeah, yeah for that.
Well I mean.
I think what we're doing it at the following have we have a lot of data in.
Second commission patient needs of patients that had thing and then they seem in front line then they relapse and become second line patients.
And we can put them back into that mission with a combination of in Chile of chemotherapy in Mexico Mccormack with another long and so they are picking a mission or later and then be stopped vaccinating them. These patients had a tremendous the poor prognosis.
London studying this post as a 20% one the a progression free survival them afford you, 5% less so basically survival. So if we can do anything to keep these patients that they could prevent differently that thing, which is what the vaccine seems to be able to do with a two year, 50% for questions Lisa.
Klaus Miller: Less than 100 patients are diagnosed each year in the U.S., and patients with DSSRCT have a very poor prognosis with limited five-year survival, so there's a clear unmet medical need in this population, for even the more fortunate DSSRTT patients where it's possible to do gross total resection. The five-year survival appears to be approximately 20%. And based on observations from MSK, whole-abdominal intensity-modulating radiotherapy may be advisable for those patients whose tumor can be responded to. The data reported by Dr. Modak at CETOS was based on evaluation of 33 patients at NSK, a total of 24 patients from a Phase I study who received radiation to the abdomen and in combination with interferotoneally-administrated umbertumab with iodine, and nine patients who just got treatment with abdominal radiation after total tumor resection but no umbertumab.
I will then go said I'm thinking when they come in Michigan.
When do you doing something so.
I think for the second line setting the vaccination, we would go to the FDA and propose a.
Controls all fast track accelerated type of approval with the post marketing commitment potentially in the randomized frontline study. So we take patients in front line.
As you also consistent with the largest disclosed.
About 40% of the patients will be that's within two to three years typically in front line you may get that up to two so that's what makes it I'm not talking about that but that's the ballpark, we're talking about some number of patients and sometimes it's also indefinitely.
Klaus Miller: The study showed a 41-month median overall survival for those who did not receive umbertumab and 59 months for those who received umbertumab. Thus, the data presented at CETUS showed that adding iodinated ambutamide to standard radiotherapy was well-tolerated and indicated, in our opinion, improved survival. Furthermore, adding Umbertumab to the resection improved the five-year Kaplan-Meier estimated overall survival rate from the historically reported rate of approximately 20% to approximately 40%. Therefore, we are very pleased to see these survival data. Lack of a valuable disease means that survival is the only relevant endpoint in life.
Look at Comedically decreases with notice for these patients. So you could imagine that that we tried to discuss with the agency study we will be do they get an approval.
Controlled all sitting in second line and then go for a month and connect them to do a randomized frontline setting.
Hi, this is speculative actually I'd like now and I need to.
Habit dialogue with the agency and to be honest, we're working hard on getting.
I feel they filed.
And we'll take the dialogue with you can see off that we have continued pool.
Next in line.
We cannot be.
I think builds are a kind of like the strategies that'd be a working capital.
But they're not girls. Thank you.
Klaus Miller: While this approach may not help patients who do not achieve cross-tumor resection, we believe that it may help patients with microscopic diseases, which is exactly where this antibody has been. We believe that the data illustrate the breadth of the use of radiolated ombetamide, which could potentially be applied to other piatineal or compartmental medical series. We recently initiated a phase 2 trial in DSSRCT at MSK, and we are planning to open this study for a small exploratory group of both gastric cancer patients and also ovarian cancer. I excitedly presented an updated ombudsmouth data readout from study 03133 at NSK, where patients with CNS leptomeningum metastasis from Norvostoma received two doses of radiolated omb Data shows that for the 107 evaluated patients... The median survival had decreased to 50.8 months, where the final median has not yet been reached.
[noise]. The next question is from Robert Burns of H.C. Wainwright. Please go ahead.
Hi, guys. Thanks for taking my questions and congrats on all the progress you guys as man. So three questions from me if I back a first one for a night for the next to the MAA filing in Europe or could you provide an update as to when do you believe you'll be able to file the am I right.
And what the timeline would look like around the review period in Europe. So that's my first one.
Okay. So when I said about finding in Europe, we haven't given they need a final guidance would be thing, it's a via discussing forget the it and say somebody had the pick and place with EMA. We can give them oversight guide I would consider it unlikely that you'll be able to file in Europe until probably the building getting up 22.
Maybe late 21.
Definitely not this year.
Okay, because we need to do another study in Europe, you know the regulatory situation, it's a little different the stand up well time in India, India was 270 days some of the filing.
Klaus Miller: This compared to the previously recorded 47.1 months when we had 93 patients in the study, so we are pleased with this update. Remember that historical median survival for these patients is approximately. In addition, we had 68 patients diagnosed with other CNF cancers, including metastatic tumors, who had received a total of 201 injections of Imbodermat. The injections were routinely administered, primarily in outpatient settings.
Okay. Okay, it's kinda talk stops in between.
Okay, great. Thank you.
Second question, so switching back to the frontline setting how long do you estimate that the multicenter trial that you are initiating this year will take to read out and are you considering incorporating back set them up into the induction portion of that treatment similar to the ferman and colleagues paper that came out a and at the end of claim 19th.
Klaus Miller: And the patient's primary CNS diagnosis included eight different cancers, including 27 patients with medulloblastoma, nine patients with ependymoma, patients with metastatic tumors, and patients with four different primary cancers, including nine patients with sarcomas, and five with melanoma. As of today, 26 of these 68 patients with this highly lethal diagnosis are still alive. These results fueled our plans for the lutetium-labeled amphetamide construct, for which we filed an IND in December, and the first study will be in medulloblastoma, where we have the previous experience of 27 patients with the iodinated construct. And in addition, we plan to submit a separate IMD for a basket trial of B7H3-positive CNF-glycemin-en We expect to treat the first adults within the teaching label constructs in the second half of 2020, and we are genuinely thrilled to widen our aim to include adults in these initiatives. To support our growth plans, we increased our headcount by approximately 16% to a total of 65 employees during the fourth quarter.
It's still uncertain, what it actually does to add a TV two antibody and induction setting. So we're not planning to do that'd be a plan to use the setting what have you put the next set them up and as maintenance.
The thing that induction therapy with other patients have bone marrow transcend enough you know recommending a bone marrow transplant patients and get the not necessarily tough because it seems like.
And and 80% dissipates and got them or studied the census in front line.
I also see bone marrow transplantation. Another that's people if anything at least that's good unfolding.
Question for survival data that you do with new kit or marrow transplantation and I'm talking about so what do we expect them to himself.
Frontline data needed for a potential regulatory approval I don't know for sure with them yesterday with the satisfied with two single thing to studies and an ongoing multi center study, but I would.
I think that would be sufficient but I can't say anything until they've had a regulatory.
Losses the agency about this.
But that's where.
The hot.
Bill: The increase is primarily due to the development team and the commercial team ramping up for the potential launch of Nexidermab and Umbertum. We continue to believe that we are well-positioned to move both Naxetamib and Umbertamib to FDA approval and commercialization in 2020 and concurrently increase our focus on our earlier-stage pipeline candidates, including, as I mentioned, the lutetium-labeled Umbertamib PTPA construct, the bispecific programs, and the DD2, DD3 vaccine, as well as the next in-line indications for Naxetamib Thank you, Klaus. We reported a net loss for the year ended December 31st, 2019 of $81 million, or 2 points. 3 dollars per share, basic and diluted, compared to a net loss of 43.3 million, or 1.5 dollars per share, basic and diluted, for the year ended December 31st, 2018.
It would be hard for me to see that they would not at least be willing to look I would say objective. The I'm wondering what they that's available. He has its data tourism honestly, it's still being driven it anymore. I mean, I don't think anybody's going to ask you about the vacancy.
Hi, everybody will know that hey, we can take.
With patients as it just disclose in my presentation, well, 88% of the patients already received one or two other due to two antibody and we that's okay and then to get them next set them up and you put a lot because looking into remission. This antibody is working very very well. So the C. P thing booking safety is this antibody safe.
Then, giving dying to talks a lot with aisle too I.
I mean, you can look up the black box warning itself [laughter], what kind of I think.
[laughter] safety is driving this I think more than that I think that these tickets in discussion is kind of like over them done.
That's a new paper that came up we can do they get a combination of the author humanized done it took to left and I are true cobbled together and they give it to patients that have only the season, the bone marrow stratify patients and and they get a 16% response rate I. Just told you we get 100% response rate and bone marrow.
Bill: We ended 2019 with a cash position of $207 million compared to the third quarter's ending cash position of $98 million. The increase of $109 million was primarily due to the net proceeds from our secondary offering of roughly $135 million in November 2019, offset by fourth quarter expenditures. As our work on the BLA submissions for metformin and amphetamide progresses and we accelerate the commercial ramp-up for the potential launch of the two lead compounds, we've seen our cash burn increase, and we expect the cash burn from operating expenses for 2020 to remain roughly in line with the first quarter of 2019. Research and development expenses increased by $209 million from $34 million for the 12 months ended December 31, 2018 to $64 million for the 12 months ended This increase was primarily attributable to a $17 million increase in outsourced manufacturing for our two lead product candidates, Maxitima and Lombardima.
She sees is only in the moment [laughter] not an issue anymore.
The safety crushing issue and I think the safety data presented by Dr. more let's put it here.
Yeah, No I agree with you there.
Last question [laughter] now, it's it's all good glass and then last question for me is so could you frame, how you're thinking about the G.D. chew by specific data that you know it's supposed to be released in Twoq, you 20 from an efficacy in relative benchmark perspective.
Well I haven't seen the data yet so it wasn't the only thing I can say as.
Well good progress that's sitting here a year after the start of the dose escalation study in and they haven't had anything you can say event that gave us the tenneco, which is a new simplified specific antibody.
We have to wait and see the data when do you get them, but it's definitely I'm very excited about this program then and then whether they need to get higher from here.
Bill: A $5 million increase in outsourced research and supplies to support expanding development activities. A $3 million increase in personal costs, and a $2.5 million increase in clinical trial expenses. DNA expenses increased by $10.5 million from $9 million for the year end of December 31, 2018 to $19.5 million for the year end of December 31, 2019. The increase in GNA expenses primarily reflected a $5.1 million increase in personnel costs at $3.8 million. Cash flows from operating activities show that the cash burn increased by $32 million from $41 million for the year ended December 31, 2018 to $74 million for the year ended December 31, 2019. The increase was primarily caused by an increase in the net loss for the year.
Yeah, I know you can get high Oh, Hey, Tymoshenko side. So it's still you have to wait so that's kind of like we can build Cisco late in the second week, but the bigger medicines like double time, possibly off in a pretty thin.
Still sell them.
So, let's see how the pent up but hopefully the not meeting will happen.
Good to abstract apparel.
Also all I look forward to the Adam very intrigued by it. Thank you so much costs.
Thanks, Thanks, a lot.
The next question is from Anupam Rama S.J.P. Morgan. Please go ahead.
All right and that's how on the hopping on chronic I. Thank you for taking my questions here first one on the pipeline HM two relapsed that mine Osteosarcoma study, we have seen safety data I think for this study Oh mine, that's that timeline for the African fiasco.
Is there so that the medical conference you're targeting.
Bill: The net loss itself increased by $37.8 million for the year ended December 31, 2019, and was partially offset by a $5.5 million increase in accounts payable and accrued liabilities resulting from increased operations and an increase in non-cash expenses, including stock rate compensation of $2.7 million. In addition, cash spending from investing activities increased by $1.7 million to $2 million in 2019 due to the purchase of equipment primarily for the lab in New Jersey. The net proceeds from the issuance of Comstock increased by 36 million from 99 million in 2018, when we went public, to 135 million related to our follow-on secondary offering that closed in the fourth quarter of 2019. In terms of financial guidance, we have said since the secondary offering that the cash-on-hand plus the net proceeds from the offering would cover our operating activities and capital expenditures through the fourth quarter of 2022. And this still does not take into account any potential revenues from the commercialization of Nexidimab and Valimab or the proceeds from any potential future partnerships. So we do believe Y-mAbs remains in a very healthy financial position.
And then I've a follow up.
Thanks.
Sure I mean.
This study is.
Supposed to recruit 79 patients and as soon as little 79 patients. The in this study we will find a suitable conference.
I will cross my fingers and hope that you can for simply stated before the end to figure but.
There's no guarantees and clinical trial. So so lets across the whole thing doesn't hope you get data from that study before then see here.
Okay. Another problem.
That's helpful. Yeah. Thank you and then.
Our commercial line as well I'm I felt that into potential approval of here on later that's yeah.
Q potential could evolve how are you thinking about and myself fails infrastructure.
Yes.
Oh, we haven't NFL team in place already.
When we started putting together the NFL team about.
Eight nine months ago and.
So we will have an NFL team operating mother Medical group and the company and that they will have quite a separate sales team, what's most likely more consistent somewhere between five until five and 10 sales reps and and some will be telesales pets and stuff. So so but the whole commercial organization, including.
Thomas Gad: This concludes the financial update, and I'll now turn the call over to Thomas. Thank you, Bob. I think we'll now take some questions. Certainly. We will now be conducting a question and answer session. If you would like to ask a question, please press star 1 on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star 2 if you would like to remove your question from the queue. For those using speaker equipment, it may be necessary to pick up your handset before pressing the star keys.
The back office supports the marketing the <unk> co pay the Bakken living leaks and all the other functions you put together I think they're looking medically with about 30 people.
Okay, great. Thank you for taking my question.
Sure [laughter].
The next question is from Boris Peaker of Cowen and company. Please go ahead.
Great. Thanks for taking my questions. The first one is on the extent of map I, we talked a bit about the frontline strategy here I'm just curious if approved in a frontline how much more use do you anticipate you would have with the frontline label versus just the second line label would you have retreatment of patients or just kind of want to understand your estimated.
Operator: One moment, please, while we poll for questions. The first question is from Alec Stranahan of Bank of America. Please go ahead. Oh, hey, guys.
Drug use in that setting.
Alec Warren Stranahan: Thanks for taking my questions. Congratulations on the completed 19 and the pre-BLA meeting for omburnt MAP. So just on that first, could you give us a sense of the additional studies, if any, that need to be done for the rolling BLA for omburnt MAP? And now that we have a clearer picture of the BLA submission timelines, following your interactions with the FDA, I think it's still reasonable to assume commercial launch by the end of the year. And then I've got a follow-up. Okay, thanks, Alex. This is Klaus.
Yeah, I think in frontline setting the pace of getting that said I met or would be getting back to them at all getting that kinda talk to that that's a maintenance treatment and so that's no. He said you'll actually see you're treating you can only see these pick up the antibody by the increased progression free survival.
Did you then if you had treated the next set them up and trunk line. We use next set them up in second line definitely absolutely I see absolutely no reason and we also know from experienced that in this case that patients that had been treated in front line that have read that 10, Wisconsin that said enough in second line.
Klaus Miller: Yeah, I mean, just to be clear, from VirtaMap, the FDA is not requiring any additional studies for approval. What they are requiring is that the multi-center study be opened as required by the FDA, and we have about 18 patients or so. Actually, we have almost 30 patients in the study now. The first cohort of patients will be supporting the DLA, which will be completed in May. The FDA is giving us what they call an accelerated approval, which means that there will be a post-marketing commitment, but the post-marketing commitment is not requiring any additional study.
So I I think that sometime but not cannibalizing if the second mining.
Gotcha, Okay, but the question.
Yeah. That's good to know so maybe just maybe it'll certainly brought a question.
For both makes it a map encumbered map, obviously you have extensive pipeline of various studies on going so beyond the lead indications for both of these drugs, which we talked about water whats the timeline for the next BLE filing for or label expansion really filing for each drug.
Klaus Miller: It requires that the patients in study 101, where the first cohort of patients is a part of the first approval, be followed for three years. And we have to come up with three-year-old survival data that is significantly better than historical data, which is estimated to be about... So the number of patients that need to be for the three-year follow-up is about 32 patients, and I think we are pretty close to having them.
Well I think next set them up and frontline depending on what we can quickly on with yesterday I think next set them up for frontline could be and in the end of next year.
And and and in terms of additional indications I think it's a little early to say.
With.
We need to see the Osters accommodative before we start discussing what is needed for a potential DNA.
But frontline setting the other indication I also reported a little bit about debated those patients, but that come into that mission.
Klaus Miller: And forward now, I think we have. So we're pretty close to that, and as I said, it's not a requirement for the actual filing. We have all the information we need for the filing, and we are getting ready to submit everything before the end of the year. In terms of launch time, yes, we still believe that we can do that. For an extra month, we expect to complete the volunteer aid before the end of this month, which means we'll have a PDUFA date most likely in November, and we are ready to launch as soon as we get the approval from the FDA. May, we should get a CODUFA day, most likely in January. I still believe there's a chance to launch it this year.
And second or later line.
Placements by induction chemotherapy, when you get treated animals maintenance therapy, but that doesn't happen. So that's the number a potential indicates that that's an agency anything patients or was it just seems like that indicate we tend to could also doesn't <unk>, but I'm not I think the the.
First.
Fanchon indications could see that the I think he and the different classics mobile so to me that's unless we get the approval in place for the agency, we're starting to move consent that the I could see study later this year and that we need to go and talk to the FDA I mean, they're looking at a good it seems like three to 400 kits everywhere diagnosed into wash with the identity with.
Klaus Miller: Well, typically, the FDA, for these kinds of PLAs that address these small, life-threatening diseases, they typically approve the products one or two months early, so definitely for Nexidermab. Potentially, if the FDA used their standard time minus the typical one to two month early approval, we could also have seen a better map of the U.S. that I'm sharing. Yes, that's very helpful. Thanks. And one more from me
Absolutely no possibility for survival.
They could radiation to the upon and.
You know they then they get the holiday from that it's easy to treat the six month starts growing again, then it killed.
That's a 10% to your survival rate the materials and five here I.
I mean, there's nothing gets out there.
We have an ongoing study now, but that's follow patients treated patients from more than five years smell and we have pace and set up in more than five yes survival, we have nothing more than three yet.
Klaus Miller: So, assuming Nexidimab gets approved in relapsed refractory neuroblastoma by the end of this year, have you gotten a sense from the FDA what the expected regulatory path for frontline neuroblastoma would be? And how do you think the timing of the MSK study readout might influence the multicenter study design? Thanks.
We are planning to stop and multi center study did he later this year.
Well, how long would you I think.
As if it.
Well that's a good question to me I mean, there's absolutely nothing for these patients out there and we already had paid.
I.
Do my outmost too to get.
So from a care I'd like that the I think you entered the FDA.
Klaus Miller: First of all, we don't have a discussion, we haven't had a discussion with the agency about the front line, and I have simply decided not to start discussing that until we get the approval for the second line. We will have data out there from two individual clinical center studies already this year, and we will be doing a multi-center study starting this year. It's highly likely that the data will be out there, and I think we can see at least at some sites that the peer interest... Whether that will happen after the approval, I cannot give any guarantees, and we will work towards a formal regulatory agreement to make sure that it's fully in. Great, thank you. The next question is from Etzer Darout of Guggenheim. Please go ahead.
As quickly as possible, there's no doubt the active would like to see them all just.
But I don't think I need to follow that moves into patients for more than a year.
So towards the end of next year.
We could pull if you'd like but that's very aggressive guessing from my side, but I'm also always very optimistic and that we can do to help them station I wanted I want to do that's also why it's sitting here no less than five yourself to be started by that filing to do they.
The best so much need for these treatments and kit.
Well I'm.
Going to make it a significant discount if we can move the team to get the F. data set.
Good day talent, the equity based on behalf of pay and and Multicenter study Oh.
I suppose.
And we can you, perhaps not think commitments to doing other things.
If it doesn't know what happens with all these kids every single one will die and they have dying today.
Etzer Darout: Great. Thank you for taking my question and congratulations on the progress. Just a couple of questions.
He needs to get something that can help.
Great. Thank you very much filling the answer to my questions.
Klaus Miller: I guess, one, maybe on sort of the data disclosures for 2020, given some of the disruption that we're seeing, and what your thoughts are around sort of maybe the venue or whether or not, you know, sort of change the venue by which you disclose the data, and then maybe on the global trials for the vaccine, and maybe if you could sort of talk about what that pivotal trial could look like as far as design. Thank you. Well, in terms of venue, I mean, I think we're all a little bit out in the woods right now with what's going to happen to various meetings.
Hi, good Bush will sit with it but the here [noise].
There are no further questions at this time I would like to turn the floor back over to Tom is scheduled for closing comments.
Yeah, well. Thank you everyone listening today on this particular they and.
She was hurt by much isn't a great position for 2020 and.
We are moving forward with excitement so oh.
You have a great evening. Thank you I can tell seem to book.
This concludes today's teleconference. You may disconnect your lines at this time for your participation.
Klaus Miller: I think for sure a very true larger meeting is going to happen in April this year. The meeting in May that we're planning to use as a presentation for a number of our data sets this year will be an abstract at the ANR in Amsterdam. Let's wait and see. But we have the data, and of course, we'll make an effort to make sure we get the data out as quickly as possible so everybody's enlightened about what we have. I'm not completely sure. What was the other part of your question?
[music].
Klaus Miller: So, yeah, around sort of the trial for, the global trial that you spoke about for the vaccine. Yeah, for the vaccine. What that trial could look like.
Klaus Miller: Well, I mean, I think what we're doing is that we are following here; we have a lot of data on this second remission patient. These are patients that have been in remission on the front line, then they relapse and become second-line patients, and we can put them back into remission with a combination of chemotherapy and mexidermab or mexidermab alone. And so they are in second remission or later, and then we start vaccinating them.
Klaus Miller: These patients show tremendous improvement. The London study discloses a 20% 1-year progression-free survival and a 4-year 5% less than 5% progression-free survival. So, if we can do anything to keep these patients at bay, to prevent them from relapsing, which is what the vaccine seems to be able to do with a 2-year 50% progression-free survival in those that aren't sick and are later in life, And then we are doing something called, I think for the second-line setting, the vaccination, we would go to the FDA and propose an uncontrolled, fast-track, accelerated type So we take patients in the front line, and as you can see from the data I just disclosed, about 40% of the patients will relapse within two to three years. Typically, in the front line, we may get that up to 30% relapsed. But that's the bold part we're talking about.
Klaus Miller: So a number of patients in the front line have also relapsed, which dramatically decreases the prognosis for these patients. So you can imagine that we would try to discuss with the agency a strategy where we do a get an approval in an uncontrolled setting in second line and then go for a post-mortem and connect them to do a randomized front line. But this is speculative as we are right now, and I need to have a dialogue with the agency. And to be honest, we are working hard on getting the first PLAs filed, and we'll have this dialogue with the agency after we have gotten those. Thank you. Thank you. But I think those are kind of like the strategies that we are working on. Thank you. The next question is from Robert Burns of H.C. Wainwright.
Robert John Burns: Please go ahead. Hi guys, thanks for taking my questions and congratulations on all the progress you guys have made. So, three questions from me, if I may. First one, for the next FDMEB filing in Europe, could you provide an update as to when you believe you'll be able to file the MMA and what the timeline would look like around the review period in Europe? So, that's my first.
Klaus Miller: Okay, so when I said I'm not filing in Europe, we haven't given any final guidance. What we're saying is that we are discussing getting the PIP in place. When we have the PIP in place with EMA, we can give more precise guidance. I would consider it unlikely that we will be able to file in Europe until probably the beginning of 2022, maybe late 2021. But definitely not this year.
Klaus Miller: And because we need to do another study in Europe. You know, the regulatory situation is a little different. The standard approval time in the EU is 270 days from the filing.
Klaus Miller: And then there can be various kinds of block starts. Okay, great. Thank you. My second question is, switching back to the front-line setting, how long do you estimate that the multi-center trial that you're initiating this year will take to read out? And are you considering incorporating Noxidimab into the induction portion of that treatment, similar to the Furman and colleagues paper that came out at the end of 2019? It's still uncertain what it actually does to add a DB2 antibody in the induction setting. So we are not planning to do that. We are planning to use a setting where we put the naxodermab in as maintenance after standard induction therapy, whether the patients have a bone marrow transplant or not. We are not recommending a bone marrow transplant for these patients.
[music].
Klaus Miller: We think it's an unnecessary toxicity to the case, and 80% of the patients in Dr. Moore's study we presented in Frontline had not received bone marrow transplantation. And nevertheless, we thought, if anything, at least that's good and probably better progression-free survival data than you get when you get bone marrow transplantation and dinotoxin. So, what do we expect in terms of front-line data needed for potential regulatory approval? I don't know for sure whether the FDA would be satisfied with two single-center studies and an ongoing multi-center study, but I would... I think that would be sufficient. But we can't say anything until we've had a regulatory dialogue with the agency about this. But I think it would be hard for me to see that they would not at least be willing to look, I would say, objectively at whatever data is available. It's data-driven, honestly. It's so data-driven.
[noise] [noise].
Klaus Miller: I mean, I don't think anybody's going to ask you about the efficacy. Everybody will know that, hey, we can take groups of patients, as I just disclosed in my presentation, where 88% of the patients had already received one or two other GV2 antibodies and relapsed or progressed, and then we give them metformin, and we put a large group of them into remission. This antibody is working very, very well. So the key thing will be safety. Is this antibody safer than giving dinitroxamab with IL-2?
[music].
Klaus Miller: Safety is driving this, I think, more than efficacy. There's a new paper that came out recently where they give a combination of a humanized dinosaur map and IL-2 cobbled together, and they give it to patients that have only died in bone marrow-certified patients, and they get a 16% response rate. I just told you we get a 100% response rate in bone marrow patients, diseases only in the bone marrow. It figuratively is not an issue anymore.
Klaus Miller: So safety is a key issue, and I think the safety data presented by Dr. Moore was pretty clear. Yeah. No. I agree with you there.
Klaus Miller: And then the last question, no, it's all good, Klaus. And then the last question for me is, so could you frame how you're thinking about the GD2 bi-specific data that, you know, is supposed to be released in 2Q20 from an efficacy and relative benchmark perspective? Well, I haven't seen the data yet, so the only thing I can say is that we're thrilled that we're sitting here a year after we started the dose escalation study, and we haven't had any, you can say, events that gave us a technical hold, which is unusual for a biopsy to begin to break down. We have to wait and see the data when we get it, but I'm definitely very excited If we can't get higher, time will show us.
Klaus Miller: It's still, you have to wait, so it's not kind of like you can dose escalate every second week, but we have managed to dose escalate a number of times now, so we are up to a pretty decent dose level. So, let's see how it pans out. Hopefully, the A&R meeting will happen, and we'll get the abstract approved. It would be awesome. Well, I look forward to that. I'm very intrigued by it. Thank you so much, class.
Anupam Rama: Thanks. Thank you all. The next question is from Anupam Rama of J.P. Morgan. Please go ahead. Hey guys, this is Tessa on the call tonight for Anupam.
Tessa Thomas Romero: Thank you for taking our questions here. First one on the pipeline, for the phase two relapse second line osteosarcoma study. We have seen safety data for this study. Can you remind us of timelines for the efficacy update? And is there a specific medical conference you're targeting? And then I have a follow-up. Thanks.
Klaus Miller: Sure, I mean, this study is supposed to recruit 39 patients, and as soon as those 39 patients are in the study, we will find a suitable conference. I still cross my fingers and hope that we can present the data before the end of the year.
Klaus Miller: There's no guarantee from clinical trials, so let's cross our fingers and hope we get data from that study before the end of the year. Okay, that's helpful. That's helpful. Yeah. Thank you. And then maybe a commercial one as well.
Klaus Miller: So given potential approvals here later this year, two potential approvals, how are you thinking about initial sales infrastructure? Thanks. We have an MSL team in place already. We started putting together the MSL team about 8-9 months ago, and so we will have an MSL team operating under the medical group in the company, and then we will, of course, have a separate sales team which will most likely consist of somewhere between 5-10 sales reps and some regional sales heads and stuff. But the whole commercial organization, including the back office support, the marketing, the co-pays, the market analytics, and all the other functions you put together. I think we are looking at a group of about 30 people.
Klaus Miller: Okay, great. Thank you for taking our questions. Thank you. The next question is from Boris Pieker of Cowan and Company. Please go ahead.
Boris Pieker: Great. Thanks for taking my questions. The first one is on nixidimab. We talked a bit about the frontline strategy here. I'm just curious, if approved in the frontline, how much more use do you anticipate you would have with the frontline label versus just the second line label?
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Klaus Miller: Would you have a retreatment of patients or just kind of want to understand your estimated drug use? Yeah, I think in a front-line setting, the patients are getting maxetamide or would be getting maxetamide and are getting bacterial toxamide as a maintenance treatment, and so there's no disease that you actually can see you're treating. You can only see the effect of the antibody by the increased progression for your survivors. Would you then, if you had been treated with excedermab in the front line, reuse excedermab in the second line? Definitely. Absolutely.
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Klaus Miller: I see absolutely no reason, and we also know from experience at MSK that patients that have been treated in the front line that have relapsed can respond to an excedermab in the second line, So I think the front line would not cannibalize any of the second line. Gotcha. Okay. That's good to know. So maybe just maybe, a slightly broader question.
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Klaus Miller: For both Nexetamab and Ombertamab, obviously, you have an extensive pipeline of various studies ongoing. So beyond the lead indications for both of these drugs, which we talked about, what's the timeline for the next BLA filing for or label expansion BLA filing for each drug? Well, I think Nexidermab and Frontline, depending on what we can agree on with the FDA, I think Nexidermab for Frontline could be available at the end of next year. And in terms of additional indications, I think it's a little early to say whether we need to see the osteosarcoma data before we start discussing what is needed for a potential DNA therapy. But in the Frontline setting, the other indication I also reported a little bit about the data is those patients that come into remission in second or later line, for instance, by induction chemotherapy; we have treated them with maintenance therapy and prevented them from relapsing.
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Klaus Miller: So there's a number of potential indications. So that's NAD patients, no evidence of deceased patients. That indication, we pretend to, could also do that. For amphetamines, I think the first indications could be the DIPT and desmoplastic small bowel cell tumors.
Klaus Miller: As soon as we get the approval in place for the agency, we're starting a multi-center DIPT study later this year, and we need to go and talk to the FDA. I mean, we're looking at a group of kids here, 300 to 400 kids every year, diagnosed in the U.S. with DIPT with absolutely no possibility of survival. They get radiation through their palms and, you know, then they get a holiday from their disease for three to six months, and then their disease starts growing again, and it kills them.
Klaus Miller: There's the 10% 2-year survivor rate and the 0% 5-year. I mean, there's nothing for these kids out there. And we have an ongoing study now that has fellow patients and treated patients for a bit more than five years now, and we have patients that have survived for more than five years. We have had a number of patients for more than three years, and we are planning to start an ML2 Center Study, DAPT, later this year. Well, how long would you have to follow that? Well, that's a good question.
Klaus Miller: I mean, there's absolutely nothing for these patients out there, and we already have patients. I will do my utmost to get a supplementary DLA for DIPT approved by the FDA as quickly as possible. There's no doubt the FDA would like to see a multicellular study, but I don't think I need to follow the multicellular patients for more than a year. So, towards the end of next year, we could probably be ready. But that's very aggressive guessing from my side.
Klaus Miller: But I'm also always very optimistic about what we can do to help these patients. I want to do it. That's also why we're sitting here now, less than five years after we started Y-mAbs, and we're filing to be a label. There's so much need for these treatments for these kids. So I'm really going to make a significant effort, with the team, to get the FDA to accept the data and the IPT based on what we have from MSK and a multi-center study, as little as possible. And we can give post-mortem connections to do other things, but, as I said, there's no alternative. All these kids, every single one, will die, and they are dying today from the addiction. We need to get something that can help.
Klaus Miller: Great. Thank you very much for the lengthy answers. Thank you, guys, for participating. Good to have you here. There are no further questions at this time. I would like to turn the floor back over to Thomas Gad for closing comments. Yeah. Well, thank you everyone for listening today on this particular day. As you've just heard, Y-mAbs is in a great position for 2020, and we're moving forward with excitement.
Thomas Gad: Hope you have a great evening. Thank you. Thank you, Klaus. Thank you, Bo. This concludes today's teleconference. You may disconnect your lines at this time for your participation. © BF-WATCH TV 2021 © BF-WATCH TV 2021, [inaudible] © BF-WATCH TV 2021, ?? © BF-WATCH TV 2021, © BF-WATCH TV 2021 © BF-WATCH TV 2021 ??? © BF-WATCH TV 2021 © BF-WATCH TV 2021 © BF-WATCH TV 2021, ?? ?? ?? ?? ?? © BF-WATCH TV 2021, © The Ultimate Parody Site!
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