Q2 2022 Y-mAbs Therapeutics Inc Earnings Call
Good morning and welcome to the YMABS Therapeutics Inc.
earnings conference call for the second quarter 2022.
Today's conference is being recorded.
Let me quickly remind you that the following discussion contains certain forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995.
Because forward-looking statements involve risks and uncertainties, they are not guarantees of future performance and actual results may differ materially from those expressed or implied by these forward-looking statements due to a variety of factors, including those risk factors discussed in the Company's quarterly report on Form 10Q for the fiscal second quarter ended June 30, 2022, as filed with the SEC on August 8, 2022.
At this time, I would like to turn the conference over to Thomas Gadd, the company's founder, interim CEO , and president. Please go ahead, sir.
Thank you. Good morning everybody and thank you for joining us today. And with me today, we have our CFO Bo Cruze, our Chief Commercial Officer, Sue Smith and our Chief Medical Officer, Viness Rajan.
I'm very pleased to briefly review with you our complements throughout the second quarter of 2022. Thank you.
First and foremost, the resubmission of the BLA foreign birth map in the first quarter was accepted for priority review by the FDA, and we anticipate a decision after the upcoming PDUVA date in November 2022.
We treated the first patient in pediatric AML with our CD33 bispecific antibody and continued the progress with our GD2-SATA construct, for which we obtained IND clearance, and a
and secure clinical supplies for our...
177 Lutetium Non-Cariotic Isotope from ITM, Isotope Technologies in Munich.
We have secured supply for phase one through phase three clinical development of our DD2 and we expect to treat the first patient with DD2 in the fourth quarter of 2022.
Our partnership with Cyclone Pharmaceuticals continues to advance well.
In the past quarter, we have worked diligently on Danielle's expansion to greater China.
with an expected decision on approval later this year.
We firmly believe that YMPS is well positioned to deliver multiple milestones.
generate meaningful data, and further unlock the potential of our platform to provide benefit to patients while creating value for our shareholders.
I'll turn it into a commercial franchise.
Let me begin with our first commercial product, Danielza, which was launched 18 months ago following the FDA's accelerated approval for the treatment of patients with relapsed and refractory high-risk neuroblastoma in the bone and bone marrow.
who have demonstrated that partial response, minor response, or stable disease to prior therapies.
All of us here at Y-MAPS are truly proud of the launch to date and being able to offer Danielza to children nationwide and we look forward to Danielza's potential expansion outside of the US.
While Danielle's product revenue have increased quarter to quarter since we launched, they came in at $9.8 million in the second quarter of 2022, which corresponds to a 7% decrease from the previous quarter.
However, the decline included a slightly decrease in new patients early in the second quarter, which was partially offset by a rebound in June , and we are encouraged to see the rebound continuing through July .
Q2 analyst consensus was 11.1 million, meaning that our Daniela revenues came in 1.3 million below consensus.
corresponding to approximately two patients.
Our international revenue benefited from increased royalty income from partner sales, offset by a decrease in volume due to timing of partner orders.
With the recent management changes including a new Chief Commercial Officer, we are confident that we are now on the right track.
Sue Smith has been leveraging her prior experience in leading global launches and delivering operational efficiencies and successful development campaigns.
to highlight and differentiate Danielsson in the US and potentially other markets.
She has implemented several positive changes.
and we're actually starting to see trends.
implemented by Sue. And I'm very pleased to have her on our call today. So over to you Sue. Thank you.
Okay, thank you very much, Thomas, and good morning, everyone. I'm pleased to be with you all today and happy to have the opportunity to talk about the progress we've made. In the first quarter, the company's commercial leadership team created and gained approval on a new rare disease strategic roadmap. In the second quarter, we focused on operationalizing it to focus our efforts to support company goals, empower our commercial team with new resources, and align appropriate customer support along the rare disease patient journey.
The team is now using a variety of new patient-finding strategies put into place in the first half of the year to identify clinicians and centers with active patients and to understand where they are in their journey.
This allows us to improve our targeting and provide our account managers with real-time leads.
This also helps our marketing efforts to target the right stakeholders when they need the information.
We anticipate this will catalyze Danielza's adoption and drive the appropriate use of Danielza earlier in the application's journey over time.
In an effort to support caregivers and parents, we also launched a caregiver Facebook community initiative called the Danielza Caregiver Connection, or DCC. That helps connect caregivers with the knowledge they need from others who have been through the treatment process. DCC is supported by a strategic and hyper-targeted paid media plan to reach caregivers in the neuroblastoma community.
This way they can receive firsthand real-life experiences from Danielza families to educate parents and families on what they can expect with their child's relapsed or refractory high-risk neuroblastoma and also how Danielza might benefit their child.
At this point, we would like to thank the families who joined the DCC community for sharing their experiences, supporting others in the Daniels Adjourn and also entrusting us with their care.
Our refined approach appeared to be effective in the first quarter of 2022 as we recorded Daniels of product revenues of $10.5 million for the first quarter.
We continued to leverage experiences from the first quarter of 2022 and use them to continue delivering Danielza to patients.
However, our Danielza revenues came in at $9.8 million in the second quarter of 2022, a 7% decrease, as Thomas mentioned, from the previous quarter, caused by a slight decrease in new U.S. patients earlier in the second quarter, but again, partially offset by the rebound in June that continued through July .
Parsoft April and May was due to the team's over focus on implementing new rare disease strategies initiated in the spring.
Second quarter was about building our rare disease road map.
which was not measured in patients but provides the solid foundation of rare disease best practices upon which steady future growth can occur.
And with this groundwork laid, these foundational programs are showing signs of gaining traction, with July featuring the most patients ever in our hub since launch.
These things take time, but we are convinced that we're on the right track, and we're encouraged by the increase in the number of treatment centers that have gained experience with Danielza, with 36 treatment centers having administered Danielza across the U.S. at the end of second quarter, up 6% compared to 34 centers at the end of the prior quarter. And two new centers added Danielza to the formulary in the second quarter.
We're now also starting to see the addition of notable higher potential accounts using Danielza. We're seeing geographic expansion based on our new rare needs, and we're seeing patients being re-challenged with Danielza for the first time since launch, which is great to see.
With more than half of our accounts having experience with multiple patients, we're growing our network of key opinion leaders to help with peer-to-peer education across the community.
We are comfortable with our Commercial Plan and will state a course.
We reiterate our revenue guidance of 45 to 50 million for 2022, which includes an incremental benefit from international revenues.
A year and a half into the launch, the team continues to drive Danielle's adoption and expand its physical footprint across the U.S. We're very encouraged by its benefits over other currently available options, including the rapid infusion, fewer hospitalization days, and the flexibility to be administered in the outpatient setting.
To summarize, we remain confident about the long-term prospects for Danielza as underscored by clinicians' feedback, formulary inclusions, and the evolved commercial foundation.
Our core focus remains on continued accelerated market expansion, and we look ahead to conducting additional educational engagement to further broaden site activation.
Thank you, Sue. As you can hear, everybody, we are very excited about the possibility of going forward, potentially expanding the commercial opportunity of Danielle Sonnet and growing top line revenue, while capturing additional predatric on Met Medical Needs.
This is further supported by the recent ASCO presentations of results from the phase two trial that evaluated the combination of tenialsa, which clearly put papers in case the US emergency
James Solomite and Saga Mostens GMCFF in 90 patients with chemoresistant high-risk neuroblastoma. Saga Mostens GMCFF in 90 patients
The combination reached its primary endpoint with a 64% overall response rate and 26% of patients achieving a complete response.
The treatment was safe with no greater than grade 2 toxicity.
This is the first time any anti-TD2 antibody has been studied in such a heavily pre-treated patient population.
and we are very pleased with the response rates achieved.
This further demonstrates the potential of Daniela in Harry Biskin or Bastona.
and we look forward to continuing our work towards label expansion.
YMeps is also committed to introducing Danielza into larger adult indications.
And we have ongoing partner discussions to address this potential opportunity.
I'm moving over to Inversion Map.
We are thrilled to report that the FDA has accepted our recent submission of Umberto Smuts. In the name of Blasters.
the BLA for priority review, and a guided advisory committee meeting date to take place in October 2022, and a PDUVA date for November 30, 2022.
We are optimistic about the potential approval based on meaningful improvement in overall survival rates. This assessment assertive might be applied to CF practicing byparticipation,
unparalleled advocacy in patients with
Currently, there are no FDA-approved therapies for this indication, and we hope that, if approved, reimbursement will address the significant unmet medical need here.
On Burt's Mat we fit very well into our commercial portfolio as a line extension to Daniela.
enable us to further leverage our commercial infrastructure without any major additional investments.
Additionally, we believe that on birth map if approved would mature into an important drug with significant label expansion opportunities over several medical unmet medical needs within pediatric rare diseases.
and would present.
Larger indication opportunities targeting B7H3.
Given that ImpertsMap has been granted a rare pediatric disease designation by the FDA,
We are eligible to receive a priority review voucher from the agency upon its potential approval.
MSK's entitled to receive 33% of the net proceeds generated from a sale of such amount on bootstrap POV
potentially securing a non-dilutive cash contribution to the company that would further extend our cash run rate.
Turning now to our SADA technology.
SADA is a key innovative platform in the Y-MAPs development portfolio that continues to show great promise in targeted delivery of radiopharmaceuticals to tumor sites with minimal off-target effects.
Proving opportunities to significantly increase therapeutic indices.
As we continue to optimize the technology, we become even more encouraged about the potential scientific advancements it represents for the company and the medical community.
The INB for our first SADA constructed the GD2 SADA for GD2 positive solid tumors.
was fired last year, late last year, and after the IND clearance in July , we expect to treat the first patients during the fourth quarter of this year.
We are focused on a two-pronged strategy here with efforts to treat adults in small cell lung cancer to validate the DD2 SADA while working on potential pediatric DD2 indications.
Our strategy here is to out-license larger indications over time while we focus on unmet medical pediatric indications.
We expect to treat the first patients in the fourth quarter and will look forward to potentially being able to share tumor-binding data in 2023, as our side of development is based on a diagnostic approach.
where we plan to use imaging to demonstrate tumor binding followed by therapeutic approach.
We believe we are well-persistent to explore partnership options to leverage our proprietary Facial
And our strategy here is to seek partnership for the adult indications while focusing on the pediatric indications.
We also plan to potentially optimize and repurpose previously failed late stage clinical targets that have already been proven safe in humans.
by implementing them into a SATA construct.
as we believe these SADA targets could potentially significantly enhance the therapeutic indices, as evidenced by the PK attributes of the SADA platform, and further unlock the potential of pre-targeted radiopharmaceuticals in tumors, and other
that have not historically demonstrated any meaningful responses to a therapeutic agent.
Moving to the Vibe Background Platform.
The IND4-CD33 bispecific pediatric AML has been cleared and we believe this is a promising treatment that can potentially address an important pediatric on med need.
as AML remains one of the most challenging hematologically mandolins use for children.
We treated the first AML patient in June and we look forward to reporting to progress.
this study as it unfolds.
We have decided to seek out licensing partners for Nivetrojama.
in order to preserve capital as our DD2 solder is so close to the clinic and to focus our attention on our commercial asset than the other and the potential launch of converter map.
As you know, we've established a partnership with Cyclone Pharmaceuticals.
for Daniela and on birds map expansion in Greater China. We are especially excited about Daniela and the potential approval expected to take place later this year.
which will trigger a $15 million regulatory milestone.
We have continued to see an uptick in patients treated in the pilot zones in China and expect this market to be an important revenue driver for Daniela's Asian sales.
We continue to work on making sure that the Yale Zionombers map is approved will have a global footprint.
And we have entered into additional partnerships.
covering Latin, Eastern Europe , and Israel to support this potential and continue to work on widening the footprint.
We ended the second quarter of 2022 with $133.7 million in cash.
Recall we mentioned in the previous conference call for our first quarter of 2022 that we have re-prioritized our programs in an effort to unlock the near-term value.
of our pipeline through focused internal execution and external partnerships. With a strong cash run rate and a robust pipeline, we believe we're on track to deliver many more clinical and commercial milestones.
support the continued commercialization of Tanylsa and the potential launch of ImpersonMap, as well as advance our early stage programs including the SADA technology constructs.
We are very pleased with our current financial position. We will elaborate on this in a minute.
Thank you. Over to you, Beau.
Thank you, Thomas. And good morning, everyone.
Our net revenues of 10.8 million to 21.3 million for the quarter and six months ended June 30, 2022, were presented a decrease of 1% and an increase of 30% respectively over 11 million and 16.3 million in the comparable periods of 2021.
Net revenues in the quarter and six months ended June 30, 2022, include $1 million worth of license revenue compared to $2 million worth of license revenue in the corresponding period of 2021.
The near-product revenues for the quarter and six months ended June 30, 2022, was $9.8 million and $20.3 million respectively, which represented increases of 9% and 42% respectively over the corresponding periods of 2021.
The NIOs have product revenues of $10.8 million in the second quarter, a decrease of 7% compared to the first quarter of 2022.
net revenues of 10.5 million. The decline included a slight decrease in new U.S. patients early in the second quarter, partially offset by a rebound in June and July , while international revenues benefited from increased royalty income from partner sales, offset by a decrease in volume due to the timing of partner orders.
moving to the already inexpensive.
Our R&D expenses increased by $6.68 million to $26.49 million for the quarter and six months ended June 30th 2022 respectively.
These net increases in the quarter and six months ended June 30, 2022, reflected increased outsourced manufacturing, inclusive of $2.9 million of Naxitamab inventory vase that were designated for clinical use during the three and six months ended June 30, 2022.
and increase clinical trial activity with a particular focus on the NILSA, on birdamap and the cellar constructs.
SG&A expenses increased by $9.6 million and $11.1 million to $23.1 million and $36.5 million for the quarter and six months in June 30, 2022. The increases in SG&A expenses in both periods were primarily the result of a $10.7 million charge related to contractual severance-related benefits for our former chief executive officer, which were inclusive of $1.4 million.
of compensation related accruals and $9.3 million of non-cash share-based compensation expense and, to a lesser extent, the launch and commercialization of the NELSA, which included employee-related costs and commercial expenses.
We reported the net loss for the quarter in June 30, 2022 of $41.1 million.
on 94 cents per share basic and diluted, compared to a net loss of 22.9 million or 53 cents per share basic and diluted for the quarter ended June 30, 2021. The decrease in earnings in the second quarter of 2022 reflects the unfavorable impact of the $10.7 million charge related to the contractual servants-related benefits for a former chief executive officer and increase on the expenses as noted.
Additionally, we reported a net loss for the six months ended June 30, 2022 of 69.2 million or $1.58 per share basic and diluted, compared to a net income of 10.5 million or 25 cents per basics year and 23 cents per to do this year for the six months in the June 30, 2021.
The net income in the 6 months ended June 30, 2021, included a $62 million gain from the sale of our D'Anelza Priority Review voucher, after sharing 40% of the net proceeds from the sale with MSK as further license agreement.
The decrease in earnings in the six months ended June 30, 2022 also reflects the unfavorable impact of the charts related to contractual severance-related benefits for a former chief executive officer and increased anti-expenses as noted above.
partially offset by the favorable impact of the NERSIS growing revenues.
We ended the second quarter of 2022 with a cash position of $133.7 billion compared to $181.6 million at year-end 2021.
The decrease was 47.9 million years of date and the decrease of 23 million compared to the first quarter cash balance was fixed that the cash balance was reduced by about 8% during the second quarter of 2022.
or cash bonus driven by the cash used in our operating activities.
Consistent with the prior call, we believe that our current CAS position is sufficient to fund our current operations into mid-2024.
We believe our cash position of $133.7 million as of June 30th provides a solid financial runway to support our commercial initiatives and our reprioritised pipeline programmes as Tom mentioned.
As we noted in the prior quarter, the underlying assumptions for this guidance are important to understand and we did not include any assumptions for the net proceeds received on the anticipated PLE which we could sell upon the potential approval of Embroidermal.
In addition, no new partnerships or other BD-related sources of income are included in the assumptions. Potential burden-albridging venues upon approval are also excluded, and the time-lapse of revenues are only assumed to increase modestly by 10% each year for the purposes of this analysis of runway.
To be clear, we hope to see an entirely different growth rate for Danielson in the years to come as we execute our refined commercial strategy and continue to deliver clinical data that could potentially lead to expanded indications and greater physician adoption.
in terms of development activities.
We have assumed that current programs will be advanced at our own expense, and no new programs are assumed at this point.
The financial runway forecast benefits from the fact that most of the expenses related to pivotal trials, post-marketing commitments, and regulatory activities are behind us at this point.
Also, previously disclosed, we continue to expect operating experiences of the
162 to 167 million.
and a total cash burn of $78 to $83 million for 2022.
The net cash accruals related to the servants package do not impact cash projections, but increase the estimated full year-over-year expenses, which are unchanged from the first quarter.
For the purpose of the guidance we have not assumed any inequity or debt offerings or borrowings.
We believe Y-Maps remains in a healthy financial position to execute our strategic mission priorities and support the delivery of multiple milestones.
This concludes the financial updates. Now I will turn the call back to Tonks.
Thank you, Beau.
This marks the end of today's prepared remarks. Let's open up the line now and then I perhaps we can ask the operator to remind you.
of the procedures for submitting your questions at this time. Thank you.
Thank you. 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 participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys.
One moment please while we poll for questions.
Thank you. Our first question comes from the line of Alex Shanahan with Bank of America. Please proceed with your question.
Hey guys, thanks for taking our questions. Just a couple from us. First on BERMM and just on the commercial preparedness activities ahead of a potential approval in November . Do you plan to take a hospital centric approach similar to the Danielza launch? And roughly how many salespeople do you think you'll need to obtain adequate coverage in the US? And as a follow up, what are your expectations in the EU?
given you maybe haven't had the same level of interaction with EMA as you have with the FDA. And second question on the SADA platform, could you talk a bit more about the study design? Do you feel like you have a pretty good sense of what the optimal dose of the payload should be at this point, given your preclinical studies? Just trying to think of how quickly you might be able to step through the dose escalation in parts A and B. Thanks.
Thank you, Alec. Sue, I think you can take the first question on a birthmark.
Thank you for having us. Bye.
Yeah, thanks Alex for the question. So the US team is very engaged right now in the preparations for the launch of Umbertamab and we're actually in good standing there. We are taking more of a hospital-based approach because they have to be certified in the ability to administer the nuclear medicine capability. Also given the ultra orphan nature of this, we want facilities that will see a slightly higher volume of this very rare CNS, leptomeningeal disease.
so that they have a proficiency in administering. So we are taking that approach, and we do feel that we are actually hiring some people specifically for this launch. And to start out with such small patient numbers and the synergy with our current Danielza efforts, we feel that we have the right number of people to be successful. In terms of EU expectations, we are continuing to engage with EMA and gain guidance from the agencies. For more information, visit www.usda.gov
And so, you know, the signs right now, you know, we're continuing with that effort and, you know, hoping, obviously, for good outcome, and certainly have prepared our thoughts on the best way to commercialize in you should that come to fruition.
Thank you, Sue. Vinesh, do you want to – I mean, let's not go into deep detail, but start to talk about what we're achieving for the first dose cohort and the timing of the two injections.
Maybe the starting dose.
Yes, well, so thank you very much. It's Vinay Shah, the Chief Medical Officer here. So this SADA 1001 trial, this is a phase one dose escalation study that will be divided into three separate parts. The three separate trial parts are designated part A, part B, and C. Part A being looking at the GD2 SADA dose escalation. Part B looking at the 177 Lutetium Dota.
dose escalation and part three will be the repeated dosing analysis.
And so each of these parts will only be initiated upon completing the previous part. So part B, for example, where we're looking at dose escalation on Lutetian-Doda, will only be initiated in patients who have completed a dose-limiting TOSST observation for the first part, which is just for the GD2 startup.
So this is the escalation trial will be based on a classical 3 plus 3 trial design with a standard titration stage to establish a maximum tolerated dose. I don't have details of specific doses myself, but the primary objective or primary endpoint as you can imagine is occurrence of the DLTs and the number and severity of adverse events.
So I hope this answers at least part of your question.
Yes, that's very helpful. Thanks for the color.
Thanks for the color.
Our next question comes from the line of Robert Burns with HC Wainwright. Please proceed with your question.
Hey guys, thanks for taking my questions. Just two if I may. So for the first one, it seems like the number of centers that you're able to advance has been slowing as of recently. Given this, I'm sort of curious what the average number of neuroblastoma patients are per year in the centers that you're still trying to get access to. And then my second question is with regard to some of the upcoming catalysts, are you still planning to initiate a phase two trial for an exit of Evan's frontline neuroblastoma?
And will we also see another IND submission for an additional SATA contract this year?
Thank you.
Thanks, Robert. Will you take the first question? I can take the second. Okay.
Thanks, Robert, for your question. Yeah, so in terms of the number of patients, again, sort of putting this into perspective, the ultra-rare nature of this disease, there are in the U.S. about 700 diagnosed neuroblastoma, about 350 of those patients have high-risk neuroblastoma, and about 310 of those are in our label for high-risk relapsed refractory neuroblastoma in the bone or bone marrow.
So the team is acutely aware of where those patients are and where they are in the accounts. And we see we're very focused on the sources of growth. So we know that we still have a tremendous amount of potential at the priority accounts, which we've segmented into priority, key, and standard. And we also, the good news is our team can execute well. 89% of our enrollments are coming from the accounts we've prioritized.
accounts and also we expect growth from the newly generation efforts of our new rare disease plan.
Thank you, and just to address your other question about additional I&Ds for SADA, we are planning on
submitting an additional IND in Q1 of 2023 while we focus on validating GD2 in the clinic.
prior to that IND.
Thank you for that. Are you also planning that frontline neuroblastoma trial for Noxinimab? Oh, frontline neuroblastoma trial. Yeah, we're still waiting on gathering some data and then we will go and address the FDA, but we are not planning on any head-to-head study.
We are planning to have discussions with the FDA on a non-inferior basis and see where we can take that.
Awesome, thanks Thomas.
Thanks, Thomas. Thomas Zaslowa Yep. Thank you. Thank you.
Our next question comes from the line of Charles, please proceed with your question.
Hey, good morning, everyone, and thanks for taking our questions. My first one regarding the map, could you provide any additional color expectation of the types of data we could see for the upcoming PSYOP 2022 presentations and also regarding specifically B7H3 positive?
CNS metastases. Could you also provide your thoughts around a potential development, you know, steps, development next steps of this particular asset as a future label expansion opportunity? Thanks.
Hi, Viness, do you want to take the first one?
Yes, so as you just quite rightly alluded, we'll be looking at a PSYOP oral presentation later this year for the 101 study, interim analysis. This is the multi-center YMABS-led study that will be given and delivered by Dr. Kramer. And also at PSYOP 2022, we'll have Dr. Modak giving another oral presentation of the 03-133 study, which was a single center study at Sloan Kettering.
where, as you know, we looked at the overall survival, progression for survival, and we did an indirect comparison with an external control arm to serve as an appropriate comparator. Preliminary data has shown that the overall survival difference doing this indirect comparison, the median overall survival has a difference of roughly 15 months in the control arm versus 43 months in the actual intervention arm, albeit taking into account these indirect comparisons. Moving to the 101.
Combined with these two studies, we believe that there is clearly a signal of clinical benefit for these patients who really have no alternative treatments with a very poor prognosis. And this is primarily the clinical arguments we've been submitting in our BLA and will be shared more in detail at the relevant abstracting presentations.
I think you added your second question around clinical development of this further, is that right?
Yes, specifically for the B7H3 positive CNS smits, yes.
Yes, I think in theory if we're able to get past this FDA PDUFA approval, we will be looking to see other areas where Umbertumab can be used in those specific patient populations who can benefit from this. So there are a number of neuroectodermal tumors, CNS tumors that express B7H3, not only in pediatrics but adults. The details of
precisely which tumor indications can be shared in subsequent calls, but at the moment we're open to supporting not just investigator-led programs like we have an ongoing study run by the Pediatric Brain Tumor Consortium, the PBTC study, who are looking at medulloblastoma, anependobomas, and other neurotodermal tumors, as well as through YMAP-sponsored studies. So further details will follow, but certainly it is within our strategy to see where we can get the best risk.
that discussion to focus on and how is the team preparing for that decision? And then once we look forward to a hopeful launch here, what are kind of the key drivers? Do we need to wait for inclusion and treatment guidelines or is it more just some of these you know site to site kind of interactions? Anything on that would be helpful. Thank you.
Thanks, Viness, do you want to just give a short...
Yes, thank you.
Yes, I think what we're asked for the outcome meeting I expect or we expect the areas of focus for the committee as well as the FDA will be centered around two or three, I guess, important points which has been brought up in previous discussions with the FDA. Firstly, of course, we know that the study 03133 is a single arm, single center trial. But this practical paper is no thousand, because it's strips of paper for Indians. It was alsoSecondols, the summary ne concept said slavery was the American
So the application relies on comparisons with the external control data, which always has limitations, as you know, to serve as an appropriate comparator when you're trying to show incremental treatment effect and survival improvements. And quantifying this treatment effect is important, will be important for the FDA and the outcome in order to make a risk-benefit statement. So a lot of the discussion that we anticipate will be around the robustness of these data, both from the MSK study as well as...
with measurable disease and because obviously this is still a relatively small study whether the degree of confidence in the responses seen can be replicated in a larger patient population. So that will be one area I anticipate the outcome to look at. So I think these were the key points I anticipate most of the discussion to be and I should add that all of these points we have.
been involved in a number of discussions, ongoing discussions with the FDA, and the team are confident we're able to address these, not just the clinical arguments, but also statistical arguments with a high degree of confidence, I should add, because I think we, our case largely, of course, looks at the data that we have. The 1,3133 data is the single largest study in the spatial population, and given the fact that this is a rare disease.
in an area of unmet medical need with very poor prognosis. We also believe the FDA and the outcome will look at this as an area where flexibility needs to be applied in making any judgments around risk-benefit assessments. And in their own guidance, they do provide statements where they allow for this flexibility in determining effectiveness of drugs in rare diseases. So that's really what I expect for the discussion to go like in the outcome.
Thank you, Vinesh.
Thank you.
Our next question comes from the line of Tess Morimer with JP Morgan. Please proceed with your question.
Hey guys, thanks so much for taking our questions. So a question for Sue if I could. You talked about a new rare disease strategic roadmap for the company. Would you expect to see pull through from the increased sales to headcount and other initiatives in the second half of this year? Or should we expect to see that impact more weighted to 2023? And really my question is do you believe a second half of the new
the year inflection is still achievable. Thanks so much. Thanks, Tess. Yes, so I do. I do feel confident that we have the right people in place. We're adding more resources. And, you know, the materials and programs and infrastructure we put into place in second quarter have been adopted by the sales team. They're embracing these new rare disease tools, and they're leveraging them. And as you know, in a rare disease, you know, this is ultra orphan. This is not a cardiovascular drug. So when you implement something...
in this third quarter that will take some time to get their footing. But we are very clear on the sources of growth. We are very focused there. We've already started to see early signs that for instance, our pivoted marketing plan is performing above benchmark with a more focused approach on these refined targets, and that our team executes effectively. The majority of our enrollments are coming from our currently prioritized accounts.
So I feel that the fundamentals are strong and that we expect the growth to hit our numbers this year and of course see additional growth in 2023.
And Sue, just one follow up on that if I could. I think previously you've talked about a two-thirds, one-third type of split between patients that have been treated more in the second line versus the third line. I guess how is the duration of treatment looking at your latest market research?
Thanks. Sure. So that's another area of strategic focus for us. We're looking at some new ways to refine our messaging, frankly, and we're just in the midst of developing that. I think that what we often see, unfortunately, at some of these larger centers of excellence is very late-stage patients with a shorter duration of therapy of two to three cycles before they go on to hospice.
So what we're looking to do is really move earlier in the treatment journey for those primary refractory patients who are in our label and We have great data in bone and bone marrow and that's the most stubborn disease compartment. So I think as we look to Refine and enhance our approach We're really looking to shift that to get to the earlier Stages and we really shine there. So we just want to do a even better job of communicating that
Thanks so much for taking our questions.
Thanks so much for taking our question. Thank you.
Our next question comes from a line of Mike Ewells with Morgan Stanley . Please proceed with your question.
Hey guys, thanks for taking the question. Just wanted to start on Danielle's.
Just the weakness and patient add sort of earlier this into Q.
Was there anything specific that drove that or is that more just some quarterly variability we should sort of think about as we move forward here?
Yeah, I think...
that that's really just quarterly variability. We did see a rebound from that. And I mean, I hate to use the analogy of sort of using a yo-yo when you walk up the stairs, but often in rare disease, you will see some variability. And I do think that the team was, it was two patients off. We're not happy about it. We own it, full accountability. But I do feel that frankly, the foundation that we have here, our fundamentals are very solid.
seems very engaged and motivated. So I just see that as a blip, and I really hope to deliver on that this year.
Got it. That's helpful. And then maybe just a quick follow-up, just on Umbertimab and the ADCOM. You know, thanks for all the color on sort of where you expect the focus to be. I'm just curious, has the FDA set a date yet?
Yeah, no, so we have not disclosed the date yet, but we are hoping to get it very soon.
Okay, great.
Thank you. Yeah, we will announce it once it becomes available.
be sometimes in late October .
Our next question comes from the line of Bill Maugham with Canaccord Genuity. Please proceed with your question.
Hi, good morning and thanks. So my first question is on Danielza. So among these 36 sites that do have access to Danielza, when a doctor is going through their thought process about how to treat these patients and they choose treatment other than Danielza in a patient that
within Danielle's label. Specifically, I guess, what factor do you think is leading them to choose
you know, a competitor versus Danielza, and how do you change that point in their decision process?
An excellent question. I'm assuming, Thomas, you want me to take that?
That can be a long one. Yeah, but okay. Okay. Thanks bill. So, you know as you know The other anti gv2 on the market spent in the market for seven years and we have been on the market for you know 19 months
and the Unituxin was developed by COG through all of their treatment centers over many years and our product was developed at Sloan Kettering. So at launch we didn't have you know hundreds of COG sites that had experience and the nursing staff having muscle memory you know on how to use the product at launch as they did. So I think that the key determinants there really are on...
looking to highlight where we benefit, which is, you know, we are indicated and have excellent data in clearing the bone and bone marrow, and they do not. And we also are, you know, flexible. We have the ability to be inpatient and outpatient.
So those are some of the areas that we are talking with physicians about. And you know, when you talk with a lot of these physicians one-on-one, their head is nodding in the right direction. So these are some of the areas that we're looking to explore. We have some advisory boards and market research this fall to really look at optimally telling that story. And those are some of the areas that I think will be, we know from current one-on-one interactions with some of the very, you know...
big center cog physicians that those are areas that they see that make sense and have value and also parents are very interested in the flexibility aspect of this product. So I think our DCC patient community and all the efforts we're doing there that is definitely something that they care about. So there's a number of factors in our omni-channel mix that we're working towards continuing to evolve and frankly we're building the Danielle's story as we're speaking here today.
on the CEO search. How's that going and when might we hear an update?
Yeah, I think it's going very well. YMeps is an attractive company. It's a strong foundation and a...
So I think we'll be able to give you an update maybe later this year, but it's going very well in terms of attracting talent.
Okay, thank you.
Thank you.
As a reminder, if you would like to ask a question, press star 1 on your telephone keypad.
Our next question comes from line of Etzer DeRout with BMO Capital Markets. Please proceed with your question.
Thanks for taking my question. This is Luke Chevalian for ETSR Darut. Just one from me. Can you touch on the progress of Umbertinimab, their label expansion, specifically in DIPG and DS-RCT and when we might be able to get some clinical updates for those trials? Thank you.
So, DIBG, we have changed the isotope from 124 to 131, and we are planning on pending the outcome on BERTs map for CNS-METS later this year to start a multi-center trial using 131 instead of 121 in DIBG.
PSRCT is a very, very small indication that it will be very difficult to run a proper trial. So I think we will hope to see that that will be a potential label expansion or off-label as we move out in the future.
Thank you.
We have no further questions at this time. I would like to turn the floor back over to management for closing comments.
Well, thank you everyone for participating today and wish you a great day. Thanks.
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.