Q2 2023 X4 Pharmaceuticals Inc Earnings Call

Greetings welcome to explore pharmaceuticals second quarter, 2023 financial and operating results conference call.

At this time all participants are in a listen only mode. A question and answer session will follow the formal presentation.

As a reminder, this conference call is being recorded it is now my pleasure to introduce your host it's very friendly Society advisors. Please begin.

Thank you operator.

Morning, everyone.

Presenting on today's call will be <unk>, Chief Executive Officer, Dr. Paula Ragan, the company's Chief Financial Officer, Adam Mostafa.

Chief Medical Officer, Dr. Murray Stewart.

Following prepared remarks by each we will open the call to your questions and we'll be joined by Chief Scientific software are Tavares, Chief Commercial officer, Mark Baldry, and Chief operating Officer Mary Dibiase.

As a reminder, on today's call the company will be making forward looking statements regarding regulatory and product development plans as well as research activities.

These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted.

A description of these risks can be found in X for his most recent filings with the SEC, including this quarters Form 10-Q, which is expected to be filed after market close today.

I'd now like to turn the call over to exports President and CEO , Dr. Paula Ragan.

Paula.

Thanks, Dan Hello, everyone I won't be covering a lot of exciting updates. This morning. So thank you for joining us on this call.

And I will be providing an update on our second quarter and recent events and we'll then focus the rest of the call on three key updates regarding our ongoing chronic neutropenia program first will provide some further insights on what we estimate to be the minimum addressable U S market for Maverick before and that's the end populations.

We're pursuing next well report on some exciting emerging data from our ongoing phase two trial and finally, we'll also provide a regulatory update as we prepare for a phase II clinical trial in 2024, well then conclude and open it up to your questions.

This was another extremely productive quarter for X four.

Importantly, we presented new data from our phase three for women trial in May that followed this closure in late 2022 that the trial had met its primary endpoint and first key secondary end point and with well tolerated throughout the trial.

In addition to demonstrating that Mavericks before treatment statistically significantly and Durably erased, both absolute neutrophil and lymphocyte counts both a N C N a L C versus placebo. These new data revealed that Maverick sport treatment also resulted in a statistically significant reduction in annualized <unk>.

Infection rate.

And affected clinically meaningful reductions in both the severity and duration of infections versus placebo.

Presented these data initially at our company Webinar and Med may. Additionally, the data were also accepted for oral presentation at two notable conferences.

First the annual meeting of the clinical immuno Immunology society or C. I S for Doctor Raphael about a lotto presented the data and second the European Hematology Association or E. J for Dr. John Donahue presented the data to a standing room only crowd in Frankfurt, Germany.

These data have generated much excitement throughout the immunology and hematology physician communities around Maverick sport both for its potential to be the first disease modifying treatment for women syndrome.

And we're supporting its potential to be the first new treatment option and potentially the only oral therapy to date for people with chronic neutropenia and more than 30 years.

And we are now poised to submit our first new drug application with the FDA seeking U S approval of oral once daily Maverick before for the treatment of people, aged 12 years and older with whim syndrome, there's a palpable excitement at the company. These days as you can well imagine.

We're also pleased to receive notice of issuance of an additional patents on Maverick for engine. It's granted patents. The third issued patent covering Mavericks forest composition of matter pretax compositions of matter comprising maverix four and related drug substances formed during the Mavericks for a manufacturing process.

Through December of 2038, and we are thrilled to have recently announced the appointment of Dr. Christoph Arb at Ingalls, our new Chief Medical Officer, Dr. Ari, but angles is a very seasoned executive with significant experience in drug discovery translational research clinical development regulatory.

Sorry in medical affairs, and product launch and lifecycle management experience that spans a broad range of therapeutic areas, including rare and orphan diseases.

He's held leadership roles at both large and small life sciences, and pharmaceutical companies and is expected to bring great strategic global perspective to our team here at X for when he starts next week.

While we have a minute I'd like to express our sincere gratitude to Dr. Murray Stewart Who's been serving as our interim Chief Medical officer, and leading the Mavericks for Wham NDA submission efforts rest assured marine will still be staying on as a consultant to the company to finalize the NDA submission and help onboard Christophe and will continue.

One as a continuing member of our board of directors.

Thank you so much to Murray for all you've done for X four.

I'll now pass the call over to Adam a staffer our CFO .

We cover the financial highlights of the quarter and recent updates before we turn our focus to our kind of inch opinion program Adam.

Thanks, Paul and thanks to all of you on the call today.

Concurrent with the announcement of the positive additional phase III for wind results in mid May.

We were able to complete a pipe financing priced at the market.

Raising approximately $65 million in gross proceeds.

Participants in the financing comprised both new and existing life science investors.

During the quarter, we also announced that X four was added to the Russell 3000 index. When the index completed its annual reconstitution in late June .

As a reminder, the annual reconstitution captures the 4000 largest U S stocks as of April 28, 2023 ranking them by total market capitalization and membership and the index remains in place for one year.

And just last week, we announced the completion of a $115 million debt facility with Hercules capital.

We believe this overall transaction is strategically impactful to X for as it creates expanded future optionality beyond the equity capital markets as we head into an important growth trajectory for the company.

In our release earlier. This morning, we disclosed that we had cash cash equivalents restricted cash and marketable securities totaling $142 $3 million as of June 32023.

We believe that these funds plus the $22 $5 million drawn down from the debt facility upon closing extend our cash runway into 2025.

And we'd note that this projection does not include potential additional draw downs on the debt facility and does not include the potential monetization of the priority review voucher, we would expect to receive should Mavericks four be approved for when a syndrome in the U S. In 2024.

I'll now pass it back to Paul to provide the updates across our chronic neutropenia program.

Sure.

Thanks, Adam.

Where we get into our new data, let's quickly review Mavericks for for those who may not be familiar.

Maverick sport is an orally available CXC are foreign tag enough that we're developing for a number of chronic neutropenia disorders, and whim syndrome, a rare primary immunodeficiency. If we're successful maverick so far would be the first therapy for those with whim syndrome and in chronic neutropenia Mavericks before it would be the first.

Oral treatment option in the market currently only served by injectable therapies associated with treatment limiting adverse events.

In trials today Maverick sport has proven its ability to increase the mobilization of white blood cells, including neutrophils and lymphocytes into the bloodstream, where they can perform immuno surveillance and help fight infection.

As I just mentioned, we successfully completed a phase III clinical program and whim syndrome and are poised to submit the U S. NDA for Mavericks four in that indication. We've also successfully completed a phase <unk> clinical trial of Maverick support and certain chronic each of panic disorders.

And are currently studying Maverick sport in a phase two trial.

As a reminder, we previously reported that our market research using ICD 10 code diagnosis method suggests that roughly 50000 people in the U S had been diagnosed with chronic neutropenia.

Specifically about 40000 of these are diagnosed with chronic idiopathic neutropenia and about 8000 of these are diagnosed with congenital and cyclic neutropenia.

A key question that all of us have been aiming to better understand if the size of the expected initial target population for Maverick before across the spectrum of these estimated 50000 individuals diagnosed with chronic neutropenia.

<unk> now completed this additional market research animals share our approach and results next.

Our recent work has focused on advancing the understanding of the unmet needs in patient segmentation across the N through additional market research that included physician interviews and surveys alongside longitudinal patient chart reviews.

Both of which were further triangulated by separate claims data analysis.

Robust methodology has provided some valuable insights into the real World C N landscape in the U S.

This research has confirmed is that there remains significant unmet needs across the broader C. N patient population, despite the availability and use of G CSF therapy.

And it has helped us quantify what we believe could be the minimum addressable market for maverick before in chronic neutropenia indications and its potential for further market expansion.

Firstly, please know that we purposely considered those patients who are 12 years of age and older for now and only counted those San disorders, where we believe Mavericks, where it can have an impact if it aligns with the population we intend to study in our planned phase III trial.

Given the initial segmentation. We then looked at those who experience severe or recurrent symptoms as well as those whose symptoms are deemed severe enough by physicians to warrant treatment with injectable G. CSF we.

We can characterize this initial target group of patients with high unmet need.

Our research suggests that this high unmet need population approximate one third of the total estimated 50000 people diagnosed with chronic <unk> in the U S.

We would consider that as a minimum initial addressable population for Mavericks for a number that has the potential to expand significantly. If we include younger populations, those who are intolerant to or poor quality of life on G. CSF those eligible for G CSF and or those with more moderate disease presentation.

In all cases, we are excited to potentially deliver the first oral treatment option to reduce infection and treatment burden in this patient population.

I'll now pass the call over to Dr. Murray Stewart, our interim CMO to share. Some initial phase two trial data, which are further informing our regulatory discussions and our phase III trial design Barry.

Thank you Paula.

We completed a phase one b study in people with idiopathic cyclical a congenital neutropenia on reported results in late 2022.

This 25 patient study is a single dose of <unk>.

Demonstrated 100% response rate all participants with or without concurrent G. CSF dosing achieved an increase in AMC is at least 500 yourself per microliter at peak.

Sure.

We consider this a profoundly positive result across the spectrum of CNS disorders studied.

Based on these exciting D T, but quickly advanced the study chronic dosing of <unk> in the same population.

In the phase II portion of the study myeloid express being dose daily on top of each participant baseline standard of care.

Nothing.

<unk> G CSF.

During the phase II trial me in seats are being evaluated monthly with the mean is the average of neutrophil counts at time zero and at Ford hours post dosing.

Good time to see who represents the nadir and Florida, which approximates the peak ANC push marketing.

Dosing.

The goal of the ongoing phase III study is to determine if market. It could result in an increase in E&C response.

If this responses durable and maintained over months of treatment.

And where appropriate to assess if patients can reduce G. CSF therapy with ANC volumes being maintained in the normal range when recommended by treating physicians.

Steve can talk therapy are also being assessed.

During the study period.

We are pleased to share the matching data from the first three participants in the study with at least three months of dosing data all of whom were on stable doses of gtx at baseline.

Given the market reset for yourself, just Sean correlating the high unmet need despite tcs that's available to US we believe it's important to show the market experts.

<unk> increases ANC count.

And then <unk> can enable the reduction in G CSF use.

Reduced gcs that may benefit patients by also reducing the known adverse event and risks associated with this therapy, consequently, improving quality of life.

Notably the three Tcf factory to participants dosed with my Opex showed robust increases in Nancy can step.

And all patients achieved normalization mutual count, including the two participants who had significant neutropenia baseline despite being on G. CSF.

Neutrophil evaluations for beautiful and robust and.

In fact, the increases Nancy which reached just over 10000 cells per microliter enables physicians to decrease G. CSF dosing by at least 50% as early as the two month time point.

In two cases.

Dosing has now been withdrawn completely what patients continue on study.

N C levels, while on <unk> monotherapy.

Importantly market.

<unk> profile.

And in combination with G CSF.

As the single agent continues to demonstrate good tolerability supporting chronic use.

We thought it might be helpful to visualize the state so let's walk through the example of beautiful ANC changes overtime and reduction G. CSF dosing and participant one who has a profile consistent with the planned inclusion criteria of our phase III trial.

First flex floating take you to what we're looking at.

On the Y axis, Amit ANC or absolute neutrophil count assessed over $4.

As previously described.

On the X axis is time measured in months on study.

Baseline or the time to see the volume represents ANC levels. Prior to the addition of Maverick support.

The little White bread Bun underground neutropenia or agency levels below 1500 cells per microliter, the light Green zone represents a normal range of absolute neutrophil count.

Particularly one who was diagnosed with chronic idiopathic neutropenia or cin with neutropenia can be smart despite being on chronic G. CSF.

Baseline E&C with about 1100 itself for Microliter is shown on the graph at time theater.

Here, we see changes in mean ANC levels. After two months of dosing of <unk> and stable <unk>.

Me ANC count increased to just above the upper limit normal an increase of about 90000 cells per microliter or nine fold versus baseline.

When he is he can meaningfully increase physician to give them the option to decrease either <unk> or.

G CSF as for political.

In this case, the treating physician recommended GTS dosing be decreased at which time participant one G. CSF dose was reduced by 50% and the math that extra dose remained unchanged.

At month, three knees seat counts, but again.

Neutrophil counts remained solidly within the normal range and stood at a bus fleet above baseline counts.

This is of course supported a further reduction to 25% of traditional dose at the three month time point.

Finally at month four after being a matrix for 400 milligrams daily and 25% of baseline G. CSF doses neutrophil counts continued to stay within the normal range supporting the decision to withhold G CSF dosing altogether.

This participant is continued on study to assess ANC levels on <unk> monotherapy.

Two other participants were concurrently treated matrix for antisense that for three months or longer and achieved large increases in media agency basis based on science.

In both cases this is.

<unk> decided to reduce or eliminate G. CSF dosing, while maintaining my thanks for a novel once daily dose of 400 milligrams.

These participants also remain on study.

So overall, we could not be more pleased with decent matching date.

Importantly, we believe that the data demonstrate an acceptable safety profile of <unk> in combination with G. CSF.

Additionally, the initial results of the phase two study with long term dosing is being assessed are consistent with what was demonstrated in the one b portion.

Single dose response.

With chronic dosing of <unk> in combination with G CSF.

Large increases in me Nancy, but it's Julie.

Generally over months and treatment, which supported position decisions to reduce or eliminate G. CSF dosing.

We continue to believe that the annuity well tolerated once daily treatment could be transformative for this patient population, whose only treatment option is an injectable drug economies associated adverse events and long term risks.

These data are included in an abstract just submit it to this year's Ash meeting.

We expect to share these and additional data from the ongoing trial at that time.

Additionally, these day two and also included as part of our recent discussions with the FDA in support of our proposal for the Phase III trial design, which will now cover in more detail.

Here, we show the current pipeline the phase III trial designed to support a potential label expansion for <unk> Stifel.

Which is consistent with the market research we've just shared.

An estimated population of approximately 15000 people in the U S with significant unmet needs.

We have incorporated feedback from our meeting with the FDA into this proposed study design.

We expect the population will include participants for the diagnosis of chronic idiopathic congenital Arctic one primary neutropenia.

We will study adolescents and adult subjects to neutropenia.

And you also demonstrates that did our current infections, regardless of background therapy.

The trial will be randomized placebo controlled and blinded over a 12 month treatment period examining changes in E&C levels over time as well as the clinical impact on infection burden and quality of life.

Do you see things also under consideration as part of the study given the strong interest from physicians and gives the potential clinical benefits for patients.

The same once daily dosing Houston, the wind phase three trial is supported for the <unk> Phase III program.

We are finalizing our primary and secondary endpoints and statistical analysis plan or SAP.

The primary endpoint will likely be a co primary endpoint involving increases Nancy and.

And clinical benefit.

We will provide further updates when we have final clarity on these remaining aspects of the trial.

Importantly, the overall objectives design and duration of the study is similar to that of our poor wind trial, which assessed and demonstrated increases Nancy levels and meaningful reductions in the frequency severity and duration of infections and for which we are poised to submit our first NDA.

We have been excited about the path forward to help those in need with a range of chronic neutropenia disorders.

I'll now turn the call back over to Paul Paul.

Thank you Murray and he can hopefully hear in our voices. We are thrilled at the tremendous progress that we've made in just a few short months for a range of immuno compromised patients with high unmet need.

Last quarter, we shared our positive phase III results for Maverick sport treatment demonstrated durable and see increases and reduced frequency severity and duration of infection and were now poised to submit our first NDA for Maverick before.

Given that we're planning to provide an update on our launch readiness physician outreach efforts and updates on the wind market and our third quarter earnings call and we hope you'll join us for that.

And today, we've shared at three important new advances.

First data clarifying our initial target population N C N as being the approximately 15000 diagnosed in the U S with high unmet need.

Second our favorable emerging data of long term Maverick sport treatment, where the durable large increases in AMC over months of dosing led to physicians election to reduce G CSF dosing.

And third we've completed initial FDA discussions based on our emerging data and gain further clarity around our C. N phase III trial, keeping us on track to initiate the study in the first half of next year.

We're now forging ahead with the commercial launch and win and the launch of our next phase III in CN in the first half of next year, we are well positioned to potentially deliver a meaningful oral option first so those were the whim syndrome next to them, what's the N and our hope is to expand beyond these initial indications to bring new.

<unk> options to even more patients throughout the world and with that well now open up the call for your questions operator.

Thank you and he would like to ask a question. Please press star one on your telephone keypad, a confirmation tone will indicate your line is in the question queue. You May press star two if he would like to remove your question from the queue and for participants using speaker equipment may be necessary to pick up the handset before pressing the star keys.

Our first question is from Stephen Willey with Stifel. Please proceed.

Yeah. Good morning, guys. Thanks for taking my questions and congrats on the quarter and updated data.

Yes.

Can you remind us within the fees to CN protocols, there's if there's a threshold ANC at which the investigator makes the chiefs yourself tapering decision.

And I guess does that have to be a structured paper in terms of.

A 50% reduction of 25% reduction in the fully off or could.

Or could patient one just had been taken off she shows up at any time point for the subjective assessment will be investigating.

Thanks, Steve I'll pass it over to Mary to answer that so in this specific protocol.

In the phase III, we want patients to <unk>.

For safety reasons.

Thanks Allison.

Also open to discussions with the Pis.

Feel they want to take part of it.

At another level.

So obviously in the protocol of either stop the G CSF or stopped metrics.

In the cases that we've described.

T.

Now is the moment regarding your second point is do the easy part.

50%, 25%.

We are in phase II are open to the physician decides and then how does he want to taper, but when we move into phase III I think its going to be a lot more coordinated in town. So.

So <unk> and <unk>.

And maybe a couple of comments regarding two things so in most other diseases, if you're keeping sales steroids you got it.

Tom what you call a rebound effect in the case of teeth.

G. CSF he can actually just stole a PC is that you don't have a problem other than obviously the risk of that kind of going a little so.

So we don't need to see.

Two dozen teaching material. So we can actually do it in one or two steps in taking a look.

Considering how to make it clear for physicians to follow up I wanted to process and discontinuing.

Okay, and I guess I'll follow up with the obvious question I mean, you've seen kind of a little bit of a stepwise reduction in sees as the best.

That's the tapering has increased I guess, what's your level of confidence that I guess that the next time point.

Five months, six, but youre still going to be kind of comfortably above the normal range in terms of ANC with this patient who's on my breaks for alone.

Yeah. So obviously.

Before I comment further on that and that's partly why we want to continue following the patient.

But I think the reason we are excited to share this data it's actually clinically.

It is happening so quickly so it's a physician.

So having patients in this study in the E&C response or possibly in combination.

The fact that for a couple of months the audiobooks Mtc SaaS is really exciting clinically so I'm optimistic, but I'm also realistic and I think we need to wait for long term data to see how this plays out.

Okay and then.

Question.

Yeah, I'm sorry go ahead.

It's going to add you know that the 9000 increase in neutrophil counts isn't sure I mean, as a matter of extra for that.

That's been very impressive initial response and then the question is you know obviously the drags are acting in concert together. So the question is how low can we go with G. CSF ideally a zero or ideally as infrequently as possible in all of the evidence today supports that we can get some patients across that paradigm.

Okay and then maybe just last quick question I guess can you just speak to how many patients have enrolled to date.

Yes, how many of those.

<unk> will be included in this in this next disclosure just trying to get a sense as to how many patients how long of a follow up thanks.

Yes, it would be with regard to know theres more of a theme and.

And it gives us specific number the reason we spoke about at least three is that part of the strong they've got more than three months. We've got few patients alone and Iran. And obviously later in the year, we'll have more long term data to give a more comprehensive picture.

Pleased with what we're seeing which is why we're sharing the data.

<unk> been a little bit slowly like over the summer we hope we don't pick up and we'll have more data later in the year.

Alright, thanks for taking the questions.

Our next question is from Kristen <unk> with Cantor Fitzgerald. Please proceed.

Good morning, everybody and congrats on all these updates today.

First I wanted to ask I see I guess this year you had a pretty detailed poster on understanding the real world analysis of these patients. So first can you can you help contextualize. How these findings also helped you in terms of your phase three protocol design and what your expectations at baseline might be for some of these patients.

Ultimately what would be deemed clinically meaningful here.

Sure. Thanks, Kristen I'll turn it over to Mark to answer that.

Hi, good morning.

We continue to believe there are about 50000 people living in the U S with chronic neutropenia, what's new is we have a better understanding now of the unmet need in this market.

So as you know it is standard practice and marketing strategy to segment our market based on groups of customers with similar needs and here we've identified that about a third of the people diagnosed with chronic neutropenia are suffering from severe disease symptoms and infections.

Been deemed severe enough that their physicians have decided to prescribe some dose of G. CSF.

So this is a significant opportunity for our core as we're focused on bringing oral maverick support to target the underlying disease and addressing unmet needs in these patients and as Martin explained in this presentation. These are important insights have mountain that have now informed our preparations for the phase III trial in chronic neutropenia.

Okay.

Thank you for that and I recognize the data update today is only a few months of follow up but can you can you give us some sense of that baseline where these patients experiencing infections and then also ultimately how long and follow up do you think that you'll be able to see some general early trends to be able to deter.

Our minutes, if the Trump does have an impact on infections.

Yes, it's Marty so in phase II, we did allow people with different ranges of AMC, but it was important to show. An example of someone who would exactly fit the criteria for the PC, which is below normal AMC and we know people who have a friend Nancy below normal.

At risk of infection compared to people, who normally ANC and in the phase three study we are recruiting people who've got a low AMC on top on prior history of infection.

So we know some people coming into the phase two.

Prior to his stupid infection, it's too early to see any infection data and as you may remember from our wind study, we start to seeing them really after three months reduction infection or the increase Nancy Constellate reduction infection over time, and that's why the phase III study.

So in fact on infection is a year.

Okay. Thanks, and then last question for me I'm talking a little bit into the weeds here, but how are you specifically going to define recurring engineer infections for the phase III trial like is there a threshold number that they have had to experience in the past or how are you thinking about that.

Finishing.

Okay Yeah.

Yeah. So you know.

At a high level up to at least one or two see decent passions and by that I mean that is not just calls a sniffle. They will if needed to be prescribed antibiotics are hospitalized in other words, the see decent fashion that needed medical attention.

Okay got it thanks for taking my questions and hope to see you in San Diego.

Before I too bad.

Our next question is from Ted Penthouse with Piper Sandler. Please proceed.

Thank you good morning, and really tenancy excitement and understand why someone's could progress going on.

I just wanted to tighten up a little bit on the NDA.

What what remains to be done.

Are there any inspections that need to occur.

Hum.

No.

Maybe it's early to tell but with with a rapid review cycle would you anticipate then that compensates for doing all that.

Yes.

Okay. So okay.

Currently we are at the NDA, So theres no data, there's no content waiting list.

Those who has done an NDA, we'll know a lot of its QC and publishing so it's really just tying up the loose ends in the next.

Few weeks.

Regarding the inspection.

Once we submit it wouldn't be unusual for the FDA to want to respect our product for approval on that.

The team.

Foreign inspection, the FDA chooses to come and visit us.

Excellent. Thank you and then add some.

So I'd be shocked if we get an article.

Yeah.

I'd say the reason I say that is because it's if it was borderline or didn't works then they might not come out of it there was some safety issues. So we don't really see the safety issues at once not com, we've got clear clinical efficacy.

And that's why I think is highly unlikely.

Awesome, great well best of luck.

Thanks Ted.

Our next question is from Marc Frahm with Cowen <unk> Company. Please proceed.

Mark Your line is live please check and see gave me at it.

Okay.

Thanks for taking my questions Congrats.

Congrats on the data and you obviously had a nine fold changes seems quite significant and ANC, but can you just review the kind of enrollment process and kind of how many measures, leaving up to them that that baseline measure of ANC, you actually were able to capture and could you maybe speak to the variability you're seeing.

But the one patient presenting but maybe also more broadly in the trial.

Yes, so in the phase two that came in with different levels of <unk> C and I think what we're really seeing is.

All a robust response to clinically relevant is greater than 500 itself. So in some senses, providing they do that.

They can be very valuable, but the modem I think clinically is the robust response of 500.

Im providing you get that I'm less concerned about the availability and obviously, we've got a threshold of its well above normal sort of titration.

<unk> defend a video studio.

But all the individuals who the response.

The Mark so far in the phase two everytime are examining our patient <unk>.

Please please be exactly what we have seen in the phase one which is every patient is responding with at least a 505 per microliter done obviously with 9000 being an example.

Patients are certainly along those lines given the concurrence of G CSF and our excitement for the patients and for US is how can we get them to that low or no dose of G. CSF.

Okay, that's great and then.

Maybe on that point of that being an important goal, we expect an endpoint around that to be maybe not the primary but you know in your phase III to be formulates spo.

CSF sparing ability.

Yeah, so suddenly that under consideration I think what you've got to stay is.

How is the clinical endpoint is it withdraw completely is that the endpoint is it related to the clinical benefit of withdrawing to be related to some of the issues like bone pain. So all of that under consideration.

Okay. Thank you.

Our next question is from my Young spent on me with B Riley. Please proceed.

Hey, guys at Madison on for Mike.

Thanks for taking our call congrats on the data well if.

If I can ask a quick follow up regarding your imminent and D. A I'm wondering if that package is going to include your recent press of infectious data.

That could.

It really improved the strength of your label.

And are you may be thinking expecting a different label for different patient populations.

Either by age or severity.

And then lastly, if I can get a quick one quick one in.

Market research did that factor in patient or like Clinton mission perspective or excitement. Thanks.

Thanks.

So I'll, let Marie I'd take the first part of that and Mark can jump in for a second yeah. So from a regulatory point of view.

We submit all the data to the MD and based on data cutoff. So there was a data cutoff and late in the year with all the data into this fall and then there's an opportunity for either a 90 or 120 data a few where we then put in all the data up to.

August September so we will be including all the past data and then recent data will be in the natural follow up request by the FDA for 120 day follow up.

Just to clarify the question Mary because we're very excited about that.

The data that we showed in may which reflected reductions in frequency severity and duration of Mavericks frontline patients versus placebo at the absolutely submitted and we are absolutely positioning that in the label of course, we need to see how the FDA responds, but it's a huge clinical benefit demonstration of that drive which is what the.

<unk> asked for us, though the final label to be determined but its a very key element of that the proposed NDA filing yes, sorry about that I thought you were talking about the recent any recent data now, but yes part of it.

The NDA submission includes all of the data that we've previously presented.

And then Mark do you want to come out and just to clarify indeed, the market research that we just conducted did gather insights directly from.

Both community based as well as academic.

Physicians, who treat large numbers of chronic neutropenia patients. So neither insights directly from from those physicians. They are looking at the charts of their own patients. So it's a real world picture of the CN landscape.

Got it thanks guys.

Our next question is from Trevor I'll read with Oppenheimer and company. Please proceed.

Hey, good morning, Thanks for taking the question.

Can you just talk a bit about the FDA feedback on the phase III trial design for.

For example, I mean, given the magnitude of benefit that we've seen do you guys think that you need a 12 month trial is that something from the FDA.

Then can you also talk about the Fda's thoughts on the tapering occlusions.

Yeah. So first of all with a very good meeting with the FDA and we knew that forward with the phase three.

The two.

Major issues that we discussed with them at all as soon as the top.

Opex, we discussed were related to that.

Primary end point I think it's clear they want some clinical aspect to that which is why I mentioned in my presentation looking at AMC, which we are very confident we will hit and also clinical benefits and we've got enough patients that we're powered for that.

The study design is there for that a similar to win.

We shared the data we shared with you today with the FDA deliberately to discuss whether tapering would be an appropriate.

The FDA were.

I'm aware of.

And he said.

Consider that in how you want to talk to that and that's what we're doing at the moment.

Okay. Thanks.

Our next question is from RK with H C. Wainwright. Please proceed.

Thank you good morning, Carla and Adam most of my questions have been answered.

In general.

You don't get the data that you've seen so far except for.

What additional indications could you highlight or a possible way of and.

Naval expansion from here beyond this year.

Okay.

Yeah, So I'll take that our case I mean, maybe just a quick review so we're thrilled about where might we see this massive elevation across all white blood cell subtype, including news yourself and that's led us to kind of each of <unk> and kind of that correlation of increased neutrophils means I mean death reduced infection rates.

Phase III either the additional data that does suggest sort of a breath of impacts across the immune system. I mean previously you kind of directed.

Kind of a lion's share it's around the adaptive immunity. So theres certainly a number of immune deficiencies that have deficiencies in the adaptive component of their immune system. So I think more of it is just stay tuned I'm given the breadth of the mechanism of action impacting the immune system and right now with Lam, certainly that nice correlation and reduction in frequency and severity of infection.

You can appreciate there's a nice landscape for the next round.

Pipeline expansion with that we will get to that next year.

Thank you. Thank you for taking my question.

Yeah.

Yes.

We have reached the end of our question and answer session I would like to turn the conference back over to Paula for closing comments.

We really appreciate everyone joining us today and you can appreciate how thrilled we are with such effective at 12 weeks. Since we last had our whim phase III update and we look forward to continuing to update everyone throughout the rest of the air I'm sorry, the rest of your day.

Thank you. This does conclude today's conference you may disconnect at this time and thank you again for your participation.

Okay.

Yeah.

Yeah.

[music].

Yes.

Yes.

[music].

Yeah.

Yes.

Okay.

Okay.

[music].

Yeah.

[music].

Yeah.

Uh huh.

[music].