Q3 2024 X4 Pharmaceuticals Inc Earnings Call
Please standby your program is about to begin.
Speaker Change: Greetings and welcome to the X for Pharmaceuticals call and webcast at.
Speaker Change: At this time all participants are in a listen only mode.
Speaker Change: Question and answer session will follow the formal presentation.
Speaker Change: As a reminder, this conference call is being recorded.
Speaker Change: It is now my pleasure to introduce your host Dan Ferry from lifestyle advisors. Please begin.
Dan Ferry: Thank you operator, and good morning, everyone.
Dan Ferry: Thank you for joining us today for the special earnings call English export will focus on providing a business update and importantly data and results.
Dan Ferry: Its now completed phase two study of Maverick for chronic neutropenia.
Dan Ferry: As a reminder, on today's call the company will be making forward looking statements regarding our regulatory and product development and commercialization plans as well as research activities and financial projections.
Dan Ferry: Statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted.
Dan Ferry: Discretion of these risks can be found in exports. Most recent filings with the SEC, including this year's Form 10-K, which was filed on March 21 2024.
Dan Ferry: And in the company's Form 10-Q for the third quarter, which is expected to be filed shortly.
Speaker Change: Presenting on today's call will be Dr. Paula Ragan exports, president and CEO and the company's Chief Medical Officer, Dr. Christoph Ahmed angles.
Speaker Change: I'll now turn it over to Paula Ragan to run through today's agenda.
Speaker Change: Sure.
Speaker Change: Thank you Dan and thank you all for joining us today.
We will provide an update on our operational achievements, including activities supporting the launch of its own remedy in the U S and the substantial progress to date with our ongoing phase three clinical trial of Maverick score and people with chronic neutropenia or CN.
Speaker Change: In addition, we're excited to present the positive data from our recently completed phase two study of Maverick's for N Cen. These.
Speaker Change: These results include the final Maverix for monotherapy data, which remains very encouraging and consistent with the data we presented in June from the interim phase two analysis.
Speaker Change: We will also be presenting new data from the study results showing that most of the participants who are treated with Maverick sport and G. CSF ended up having their G. CSF dose adjusted downward during the study modern means neutrophil counts for this group remained in the normal range.
Speaker Change: These data provide a first glimpse into how mavericks before it could be used in the real world, if ultimately approved and see them.
Speaker Change: Equally exciting are the new insights we gain from assessing the functionality of circulating neutrophils and a subset of the phase III study participants.
Speaker Change: As well as healthy donors for a comparison.
Speaker Change: And we will briefly review the updated safety data from the study as well.
Speaker Change: Well, then conclude and open it up for your questions and we will be joined by our Chief Commercial Officer, Mark Paul Grady, Our Chief Financial Officer, Adam Mr. Ha, Our Chief operating Officer, Dr. Mary Dibiase, our Chief Scientific officer, Dr Arch virus, and our head of corporate and patient Affairs Jose who has.
Speaker Change: So let's quickly run through the operational update as we know you're all eager to hear about the C. N phase II study results and analysis.
Speaker Change: As you know we received U S. FDA approval in late April for our first products Mavericks for branded as all Randy.
Speaker Change: Women syndrome, a rare primary immunodeficiency disease.
Speaker Change: As with ultra rare disease launches our initial commercial strategy has been focused on disease awareness to support patient identification and diagnosis.
Speaker Change: While we do recognize that sales appear to be flat quarter over quarter, we want to remind everyone that some of the sales in the second quarter for product stocking with our specialty pharma as is typical during initial launch.
Speaker Change: I'm pleased to report this past quarter, we hit our most important launch call having engaged with all 3400 of the initial immunologist and Hematologists targeted for the first phase of our launch.
Speaker Change: And these visits and digital engagements are bearing fruit as you can see here <unk>.
Speaker Change: Finally completed market research comparing pre and post launch metrics indicate that knowledge of whim syndrome, and even like these the OMB prescribers has increased and is now at more than 75%.
Speaker Change: In addition, an increasing number of physicians are screening their patients for women and 80% of Hcp's surveyed report that they would consider prescribing <unk> for their whim patients.
Speaker Change: Over the past six months, we've also stepped up our conference attendance launched a peer to peer speaker program.
Speaker Change: <unk> initiated a patient focused campaign, which includes the website new materials and in HCP discussion guide.
Speaker Change: Reimbursement and access continue to go well with favorable policy decisions, resulting in more than 150 million lives now covered.
So very good progress there.
Speaker Change: Importantly, the activities, we are conducting and wham are yielding insights that may support our efforts to advance mavericks floor into the much broader indication of chronic neutropenia.
And while our commercial efforts are exclusively focused on women today, we do see a significant overlap between a whim and CN communities.
Speaker Change: Based on our claims analysis and experience in the field, we observe that a majority of Hematologists that we're calling on for women also see chronic Egypt, India patients and of course, there is overlap within the patient advocacy communities, we engage with as well.
Speaker Change: If maverick before it is approved for use in CN, we certainly expect to be able to leverage our commercial infrastructure and relationships within the women community to support a potential launch into chronic neutropenia.
Speaker Change: As you all know success in CN will be dependent on the results of our global pivotal phase III trial, which is progressing well and currently participants are being screened and dosed across multiple countries.
Our forward study as we've named it is a global trial that will engage patients and physicians across 20% to 25 countries.
Speaker Change: I can report today that we have already received health authority authorization to initiate the trial and approximately 85% of these countries.
Speaker Change: We can also now report that approximately 40% of our planned sites worldwide are initiated and we expect to have the majority of sites initiated in early 'twenty five.
Speaker Change: This trial is expected to enroll 150 participants at 90 to 110 sites with a planned enrollment of 1% to two participants per se, which is typical in a rare disease clinical trial.
Speaker Change: Most importantly, we are on track and continue to expect full trial enrollment in mid 2025.
Christophe: With that I'll now turn it over to Christophe to get to the heart of our call today.
Speaker Change: Data results from the completed phase two study in chronic neutropenia Christoph.
Christophe: Thanks, so much Paula and Hello to everyone on the call.
Christophe: Let me begin with a quick refresher on chronic neutropenia.
Marcus Carney neutropenia is abnormally low levels of circulating neutrophils.
Christophe: That are the defense Mckinney our.
Christophe: Our bodies using pathogen.
Christophe: As we presented previously there is a well established correlation between circulating levels of neutrophil or absolute neutrophil count AMC and the risk of frequent and or serious infection.
Christophe: Note that the lower limit of normal for neutropenia diagnosis is 1500 cells per micro liter.
Christophe: Below that.
Christophe: <unk> are divided based on the infection risk any time with Blu 500, so for micro leader.
Christophe: While to moderate to severe.
Christophe: As you can see.
Christophe: Judy any neutropenia patients and less than 500.
Christophe: <unk> alright, Paul to the highest risk of infection.
Reduce the risk of infection in these patients it is critical to move them from the most.
Christophe: Right.
Christophe: Great.
Steve as normal level of neutrophil as possible.
Christophe: Therefore, an increase of 500 cells per micro liter is an important clinical practice is subjective.
Christophe: Mackenzie Zumba faction Matheus before has been shown to do precisely that.
Christophe: Increase the number of circulating neutrophils.
Christophe: This ability is clearly stated in the Maverick before label from the FDA approval in <unk> syndrome based on results from a pivotal phase III trial.
These results demonstrated that maverick before sustainably.
Christophe: Feel good at 52 weeks of the trial and consequently, reduce the annualized rate of infection, along with the duration and severity of infection in trial participants.
Christophe: This infection benefit was achieved through an increase in many E&P of more than 500 itself for micro leader.
Christophe: These results and when approval in the U S.
Christophe: Worldwide confusing you now plan to potentially bring Mavericks as for Qdoba with other colleagues, Mr opinion condition, where significant unmet needs remain.
The gap in addressing unmet needs, resulting from the significant lack of innovation in the field.
Christophe: The only currently available therapy.
Christophe: <unk>, which was approved almost 30 years ago.
Christophe: It's an injectable drug that is executed with dose dependent and often treatment limiting side effects as well as long term rate of certain malignancy.
Speaker Change: As you've heard in our previous Investor presentation.
Speaker Change: And patients and the physicians that treat them.
Speaker Change: Are you satisfied with that.
Speaker Change: Sorry.
Speaker Change: <unk>.
Speaker Change: And bone and muscle pain.
Speaker Change: Unfortunately, despite effect often dose dependent and can limit the use of G CSF or <unk>.
It's you have to fully effective dose.
Speaker Change: We believe there is an urgent need for new innovation like Maverick before which data analysis could be used if approved as a monotherapy or in combination with G. CSF.
Speaker Change: While also allowing for reduced doses of just yesterday.
Speaker Change: Many side effects and long term cancer risk.
Speaker Change: Our market research indicates.
Speaker Change: There are approximately 50000 people diagnosed in the U S.
Speaker Change: Of this population.
Speaker Change: Can you maybe that's about 15000 of high unmet need.
Speaker Change: And recurrent infection.
Despite the available standards of care.
Speaker Change: Our phase III <unk> trial focuses on this population with the highest unmet need enrolling adults.
Speaker Change: And that along with A&P below 1500 sale for micro leader and he is.
Speaker Change: Sorry, if recurrent or serious infection.
Speaker Change: To meet the multiple needs of this population, we envisioned two potential opportunities.
Speaker Change: Once daily Mavericks before within this market.
Speaker Change: One.
Speaker Change: It could be used as a monotherapy.
Speaker Change: Diagnosed patient.
Audio replay G CSF treatment.
Speaker Change: <unk> in combination with G CSF, enabling a meaningful reduction in GTS.
Speaker Change: Those names.
Speaker Change: Lessening pain discomfort and long term risk of malignancy.
Our phase II study set out to evaluate those opportunities.
Speaker Change: You'll see in just a minute we believe the data from this study supports the potential for Maverick before to transform the chronic care of this neutropenia population.
Speaker Change: As shown here the main goal of the phase two study work to confirm the durability of the phase <unk> results that showed how much research response to a single dose of <unk>.
Speaker Change: The four plus or minus G CSF across multiple tumor types.
Speaker Change: We also aim to assets long term safety and Tolerability of Maverick's, a for US recently with Gcs death in people with CF.
Speaker Change: In addition, we wanted to explore the feasibility and the willingness of physicians to safely reduce is that with Mavericks before.
Speaker Change: <unk> was also to inform the design of our phase III pivotal trial and in doing so the risk of the trial.
Speaker Change: The phase two study was the six month study with monthly visits and.
Speaker Change: <unk> comprehensive assessment at baseline and months, one three and six.
Speaker Change: Two quick notes on the design of the study first.
Speaker Change: Neutral feel lifecycle is about 10 to 14 days, we believe that the study of display provides a good indication.
Speaker Change: <unk> of the bone marrow as long term ability for stable neutral field production.
Speaker Change: And second.
Speaker Change: Modification.
Speaker Change: The dosing where permitted in the combination group.
Speaker Change: Hello, and participants to visit.
Speaker Change: First.
Of those on the not just did those would have been at the month's free visit.
Speaker Change: Is that J Smith.
Speaker Change: Decided between physician and their patients and we're not towards the Goldman.
Speaker Change: Let's start by looking at the final participant disposition in the study.
Speaker Change: Typical of those with chronic neutropenia and high unmet need.
Speaker Change: We enrolled a total of 23 participants in this six month single arm open label study.
Speaker Change: With the mix of your Apache can consistently built yet.
Speaker Change: Even a few with cyclic presentation.
Speaker Change: There was a typical agenda distribution into the study and the mean age of 34 years.
Speaker Change: We also note here that there was a good mix of Genesee backgrounds with congenital group also representative of what you might see across the Fujian population.
Speaker Change: We analyze the data in two groups.
Speaker Change: Except for mono therapy, comprising 10, best participants and the Maverick <unk> plus G CSF group.
Speaker Change: Research and talk to you see pin nine of whom ended up having to do just that.
Speaker Change: <unk> reduced in this study.
Speaker Change: In addition, we evaluated neutrophil functionality pre and post treatment in the subs.
Speaker Change: The depopulation of nine Evaluable participant.
Speaker Change: Comparable those results to a group of healthy individuals.
Speaker Change: To address the unmet need existing with current chronic therapy.
Speaker Change: Our objective is to sustainably increase circulating functional neutrophil using oral mavericks before.
Speaker Change: The results presented to date are focused on answering the following full question.
One in the mono therapy group at Maverick for increase in jewelry, we sustained absolute neutrophil count a clinically meaningful level.
Speaker Change: June in the combination group with <unk>.
Speaker Change: Issuing willing enable to address their patients to see if those wind, adding mavericks before.
Speaker Change: Great.
Speaker Change: That would be reduced while maintaining clinically meaningfully and C level and core of.
Speaker Change: Neutrophils that are mobilized into the peripheral blood <unk> support.
Speaker Change: <unk> throughout the six months got it.
Speaker Change: First let's look at the monotherapy results.
Speaker Change: Consistent with our data presentation in June maverix afford directly and meaningfully increase mean ANC, bringing levels into normal range at month, three and six.
Speaker Change: As an oral once daily treatment delivering this level of increase.
Speaker Change: You can begin to appreciate the potential for Maverick before as monotherapy.
In addition.
Speaker Change: Maverick before all sold directly and meaningfully increased mean <unk>.
Speaker Change: In the EMEA.
Speaker Change: Study participants.
Speaker Change: Starting with levels below 500 cells per microliter.
Speaker Change: More than two fold increase in me Nancy versus baseline throughout six months of Maverick treatment.
Speaker Change: As a reminder.
Such an increase in A&P in our phase III <unk> trial led to a decrease of 60% in infection rate.
Speaker Change: So the similar increase in A&P here is not only clinically meaningful but also helps increase our confidence in achieving a positive infection rates readout for this model therapy population in the ongoing pivotal phase III trial.
Speaker Change: Let's move now to the combination result.
In this group.
Speaker Change: We started with 13 participants entering the study on GTS type at baseline.
Speaker Change: <unk> dropped out almost immediately so we had 12 eligible you have there is that those reduce flowing their month two visit.
Speaker Change: Notably clinician chose to reduce the GC theft in nine of these 12 with three being fully taken up.
Speaker Change: Iot there six months visit finishing the study on Maverick Sephora monotherapy.
Speaker Change: Thinking back to the market opportunity and unmet need slides you can see why we're so excited about the data.
Speaker Change: As the first look at how physician could potentially use maverick before and they're seeing patients who are already on gcs that Judy should it gain approval.
Speaker Change: Do they were not presenting the data from the three participants who remained on stable dose.
We note that the final data set from this group was consistent with the results. We shared this past June showing robust and sustained increases in me Nancy from baseline levels.
Speaker Change: Let's look now only of participants with those adjusted.
As shown here.
Speaker Change: Reductions were quite substantial and meaning.
Speaker Change: We're able to be maintained at normal levels throughout the study.
Speaker Change: At month three.
Speaker Change: Eight of the nine had been adjusted.
Speaker Change: Physicians were comfortable Lori you said by 52% on average.
Speaker Change: The average increase to 72% reductions by month six.
Speaker Change: With three of the nine did those participants.
Speaker Change: Completely of G CSF.
Speaker Change: I think notably.
Speaker Change: Physicians and academic experts have told us that they believe that 25% reduction in <unk>, you said would be clinically meaningful to their patient.
Speaker Change: So seeing this level of reduction reinforces the potential benefits of maverick as before and role in the treatment of CF.
Speaker Change: We now turn our attention to <unk>.
Speaker Change: Assessing the functionality of the increased levels of circulating Mr. Shaw in the phase II study.
As mentioned.
Speaker Change: The purpose of this study was to assess the percentage of functional neutrophils in people with CN, including those with congenital genetic variation associated with neutrophil maturation that rise.
Speaker Change: We used to well accepted functional ETF, phagocytosis assay, which assesses the neutrophil ability to ingalls pathogen.
And on that day for Rod production Rockies reactive oxygen species or the ability to damage akil pathogen that have been named golf.
Speaker Change: These neutrophils function studies were conducted on the sample from nine eligible participants.
Speaker Change: With the mix of CN types, and monotherapy and combination use.
Speaker Change: We also recruited five healthy individuals to provide a benchmark for comparison.
Speaker Change: For simplicity Judy.
Speaker Change: Our only showing the <unk> data, but we know that the Ross assay results closely mirrors the psychosis is results.
Speaker Change: While the participants with chronic neutropenia have fewer neutrophil <unk> dollars you can see here that those neutrophils that do make it into the circulation or in fact functional visits.
Speaker Change: With a similar percent of French.
Speaker Change: Between the neutropenia and healthy donor population.
Speaker Change: Note that the bars here represent the percentage of neutrophil that demonstrated phagocytosis after challenge with OXXO night here yet.
Speaker Change: We've also broken out the congenital yes.
Speaker Change: Do you see is genetic variant ethics functionally do differently than in the other groups.
Speaker Change: Looking now at the full result, we see that the personal teacher functional neutrophil and the healthy donors the full group of participants.
Speaker Change: The contingency will see and subset.
Speaker Change: Essentially the same following six months Maverick therapy.
Speaker Change: And note, while we're showing you here the data for fiber system function.
Speaker Change: We generated very similar data on the percentage of neutrophil generating rock after bacterial Charles.
Speaker Change: Recall that our 52 week phase III clinical trial, we saw that an increase in circulating metro neutrophil corresponded with a 60% reduction in the annualized infection rates as well as reduction in the severity and duration of infection.
Speaker Change: By increasing the number of functional stipulating the Churchill, we believe that maverick, except for treatment could correspondingly reduce the infection rate in the logic chronic neutropenia population.
And we are hopeful to see similar results in our ongoing Registrational phase III chronic neutropenia trial.
Speaker Change: Maverick, so far with <unk>.
Without concurrent G. CSF therapy was generally well tolerated throughout the study.
Speaker Change: The overall safety profile was consistent with previous studies and the most frequent treatment related treatment emergent.
Speaker Change: We're gi related nausea, and diarrhea, which were mild to moderate and to be clear result overtime.
Speaker Change: As we noted in our June presentation, we did see three just continuation in this study <unk> early in the execution of the study and after implementing an education program Unforeseeable Gi side effects there were no further discontinuation.
Speaker Change: Rest assured we have incorporated this education into the phase III trial.
Speaker Change: In summary.
Speaker Change: We could not be more pleased with the results of this phase III study, having met all of the goals we set forth.
Speaker Change: We have shown that Maverick folk and Durably and meaningfully increase me Nancy as a mono therapy, even in the most.
Speaker Change: <unk> population and can do likewise windows in combination with G. CSF.
Speaker Change: We showed that maverick before enabled clinicians and their patients.
Speaker Change: Meaningfully lowered the use of G CSF, while maintaining me Nancy at normal level.
Speaker Change: Critically we saw that Maverick support increased song town functional tiptree, leading neutrophil even in the most difficult to treat individuals with Ti.
Speaker Change: And importantly, we demonstrated maverick support was generally well tolerated on its own and in combination with G. CSF.
These results give us great confidence in the chance of success of our ongoing phase III trial in Maverick as opposed to potential to reduce infection rates.
Speaker Change: They also provide hope that's mavericks default could be the innovation that this is lee.
Speaker Change: Looking forward as you've heard previously through our interviews with physicians and patients what they need and one is a well tolerated oral option one that can enable them to safely reduce the use of injectable G. CSF.
Speaker Change: And so far our results have shown that maverix before that.
Speaker Change: Debt profile as well.
Speaker Change: Either monotherapy or in combination use.
Speaker Change: I'll now turn it back to Paul as we conclude Paula.
Paul: Thanks, so much Christophe great job sharing our exciting update on Maverick source potential in CN.
Paul: To conclude over the last six months, we have made significant progress as a company and bringing innovation to the immuno deficiency community.
Paul: By gaining approval for and launching the RMB and the U S for whim syndrome, and by exploring additional geographies for Mavericks for us approval and distribution. When we are seeking to maximize the global opportunity to help those with whim.
Paul: And by completing and reporting out these positive phase two results and initiating our pivotal phase III trials Mavericks for in the liquid credit which Kenya.
Paul: We are seeking to further expand the market opportunity for Mavericks, four and have a positive impact on a much larger potential patient population.
Paul: In terms of our financial results are tracking to our press release from this morning, and our 10-Q to be issued shortly.
Paul: Ended the third quarter of 2024 with cash and equivalents of almost a $136 million.
Paul: We continue to believe this gives us the runway into late 2025.
Paul: Note that this does not include the expected ramp up of sales until remedy throughout next year.
Paul: As always we thank you for your continued support and we'd now be happy to take your questions operator.
Speaker Change: Thank you at this time, if you would like to ask a question. Please press the star and one on your telephone keypad.
Speaker Change: You may withdraw your question at any time by pressing star two.
Speaker Change: Once again to ask a question. Please press the star and one on your telephone keypad.
Speaker Change: We will take our first question from Kristen <unk> with Cantor. Please go ahead. Your line is open.
Speaker Change: Hi, good morning, everybody and congrats on these new great data you shared with us really appreciate it.
Speaker Change: So questions that I have are related to the new data today.
Speaker Change: I wanted to ask what the biggest criteria were in the trial that maybe physicians comfortable to reduce and lower the G. CSF usage and while I recognize it wasn't a formal endpoint measured I'm curious as the doctors have any anecdotes to share about any early signals of reduce pain or better.
Speaker Change: Excluding these benefits for patients.
Speaker Change: Yeah, Chris and thank you for the question Yeah, I'll start and then Christophe will add.
Speaker Change: Add some color. So I mean, the important thing about the phase II study as we really put that choice and the physicians and patients can so theres a wide variety of patients they have real world experience with them. So really what gave them comfort to adjust G. CSF dosing would be high and robust AMC responses due to the addition of Maverick.
Sure.
Speaker Change: That if confidence in data and of course, they action on that but crystal have Brian give some more color on that sure. Yeah. No. We are we are really happy with the results and the choice that 75%. So that those physicians with their patients have decided to decrease G. CSF. This is.
Speaker Change: This is a really high percentage of those decisions without any mandates from the protocol or any any prescriptions that we we ask them to do this.
Speaker Change: With regard to the choice to do it as I've mentioned.
Speaker Change: Louis between patients and their physician and it's a balance between the level of fancy as Paul I, just mentioned and so they weren't very comfortable with the level of fancy and the clinical manifestations that these patients were having so we could not identify a specific pattern, but this is really what drives most.
Speaker Change: Of those decisions.
Okay. Thanks, and then any early anecdotes or just a little too early to make that assessment.
Speaker Change: I think it's early to make that assessment and of course, what we're most pleased with is the broad applicability the broad reductions in G. CSF across the 75% of patients even some of them completely coming off so certainly a lot more Ah study to be done Kristen, but thanks for the highlights.
Yeah, and if I may ask one more question on can you talk a little bit more in terms of what lowered G. CSF usage actually means is it that positions are.
Speaker Change: Assessing every other day for usage is the amount of G. CSF that administered is lowered and then do we have a general sense to give us whether what correlation of G. CSF for usage could ultimately like truly lead to alleviating some of the dangerous side effects and the income.
Being at one.
Speaker Change: Yes, so we will take the second part first so what we've been reporting on from patients and physicians and some of our early research is that about 25%.
Speaker Change: Reduction either in dose and our frequency seems to be the thresholds for something that's clinically meaningful the program and obviously the lower the better in terms of the short and long term toxicity is experienced by dosing.
Speaker Change: And then just in terms of <unk>.
Percent reductions as always based off of the patient entry criteria are challenged with G. CSF is the heterogeneity of each patient is different so we treated them as such as clinicians do so that reduction reflects their entry changes in their entry baseline levels.
Speaker Change: Thanks, again and congratulations.
Speaker Change: Thank you.
Speaker Change: Thank you. Our next question comes from Patti <unk>.
Speaker Change: With Piper Sandler. Please go ahead your line is open.
Speaker Change: Okay. Thank you very much good morning, everyone and congrats on the phase II data I had one quick follow up question.
Speaker Change: Well.
When it comes to the phase three trial and I'm wondering how is there a way or are you looking at benefit.
Speaker Change: Kind of following up on the last question from G. CSF reduction is there any way to capture those.
Speaker Change: In terms of improved Tolerability and improved though.
Speaker Change: Thank you and then I have a quick one on zelle ramzi. Thanks.
Speaker Change: Sure I'll turn that over to Christophe yes, no. So the phase III trial is designed for as a registration approval trial as per the FDA requirements. So we will be looking at those aspects as a full wall.
Speaker Change: Study two that phase III trial right. Okay, yes that makes a lot of sense and also I have to imagine it might take some time for some of those tumors, but yeah. That's great.
And then when it comes to cause all Rumsey you guys announced a new initiative can you just provide a little bit more detail around that in terms of reaching out to the patients.
Ted: Sure Ted just to clarify a little bit more color on our sort of patient ambassador effort.
Speaker Change: Yes, yes. Thank you.
Speaker Change: Sure Mark.
Speaker Change: Thanks Ted.
Speaker Change: You are right. We are we're laser focused right now on raising disease awareness.
Speaker Change: And driving screening whim and presenting at all Randy is a treatment that has proven to work and win so we've just launched a new patient.
Speaker Change: Correct a campaign this is a combination of.
Speaker Change: Digital campaigns with websites as well as hard copy material that we can provide to patients and we think this will help to educate patients and encourage them to go and have a discussion with their physician about Wednesday.
Speaker Change: Okay.
Yes.
Speaker Change: Great excellent well. Thank you very much appreciate the time.
Speaker Change: Thanks, so much Ken.
Speaker Change: Thank you. Our next question comes from RK with H C. W. Please go ahead your line is open.
Speaker Change: Thank you and good morning, Paul and team.
Speaker Change:
Speaker Change: I just wanted to focus a little bit on zelle Randy.
Speaker Change: And the uptick in demand in the market.
Speaker Change: Kenny.
Speaker Change: Educate us on.
Speaker Change: How many patients are.
Speaker Change: On the drug.
Speaker Change: Wow.
Speaker Change: What is the commercialization.
Speaker Change: Hum.
Speaker Change: Set up right now.
And I'm just trying to understand how long do you think.
Speaker Change: You could take for that to be.
Speaker Change: To.
Speaker Change: It will be adopted.
And then in a decent fashion.
Speaker Change: Thanks, RK, let's turn it over to Mark Thanks RK.
Mark: As I mentioned, we are right now laser focused on raising disease awareness and driving that screening for women encouraging physicians to look for when patients in their practice, helping them recognize these patients in their practice up to now there's been no approved treatment for this disease, so everything starts with raising disease awareness.
Mark: This an encouraging physicians to.
Mark: Identify the patients in their practice and then we have a discussion with them about zelle, Randy and we share the data that shows there's RMB works. So that's really where we are now and it's building the foundation for our ramping up into 2025, and we'll be able to share more details with you that.
Speaker Change: Thank you thanks for taking my question.
Speaker Change: Yes.
Thank you. Our next question comes from Stephen Wiley with Stifel. Please go ahead. Your line is open.
Stephen Wiley: Yes. Good morning, Thanks for taking the questions maybe one on the data went on so Germany.
Stephen Wiley: I guess on.
Stephen Wiley: On the data side was there any correlation between CN subtype, and the rapidity and magnitude of G CSF dose reduction.
Stephen Wiley: And then I guess was there also any correlation between the G. CSF dose. These patients were receiving at baseline and then the magnitude of the dose reduction they were able to achieve.
Speaker Change: Sure I'll start and then Christophe will provide more color, but in terms of the <unk> subtype. The good news is the Jack seems to be a robust lean consistently working we did breakout the severity of those patients because those are the toughest to treat in a robust response, there but in terms of cin for congenital we're seeing a very consistent effects and <unk>.
Speaker Change: Terms of G. CSF percent reduction from their baseline again, we're not really seeing any variability in response based on their G. CSF profiles, but <unk> had a lot of.
Speaker Change: Data analysis, and Christophe can provide some more color there yes.
Speaker Change: So the as the CN face to the sample size and the number of patients in that study is too small to start drawing.
Speaker Change: Drawing directions, where we're going with a different type of patients. So we.
Speaker Change: We know that in all of the type of patients that were included in the phase III study decrease any type of those patients decreased G. CSF and chose to decrease G CSF voluntarily.
Speaker Change: That we know for sure the phase III trial will help us.
Speaker Change: Having that big of a picture of how those different.
Speaker Change: The different subtypes to congenital the different mutations and the congenital et cetera. All of this will be analyzed in the phase III study with Metro and T gel.
Speaker Change: Okay, and then on Xolair M D.
Speaker Change: I know you've hit a 100% of your target accounts.
Speaker Change: I think you also mentioned 60% of Hcp's.
Speaker Change: Have have increased their screening, but I guess do you have a sense of how many of these.
Speaker Change: Hcp's that youre targeting R&D to actively screening.
Speaker Change: Just wondering I guess, how low of a base you're starting out here.
Speaker Change: And what kind of runway you might have thanks.
Speaker Change: Yeah. So.
Speaker Change: Again, the the way we identify position that we think have a whim patient in their practice is really using claims analysis in our market research and these are these are the physicians that were going out to two.
Speaker Change: Really make them aware of whim syndrome, and encourage them to start screening for the for the disease for the patients in their practice and and this is really where we're getting the most engagement.
Speaker Change: At this point and the other thing that we're really trying to do is reached the positions where they are and so we've really doubled down on our conference presence this year.
Speaker Change: As you know.
Speaker Change: These are these physicians come from different specialties. So we.
Speaker Change: We mainly focus on hematology and immunology and tried to really meet those physicians where they are.
Speaker Change: Alright, thanks for taking the questions.
Speaker Change: Thank you. Our next question comes from David <unk> with Zacks. Please go ahead. Your line is open.
Speaker Change: Hey, good morning, everyone.
Speaker Change: For the remedy I'm curious if you will be able to provide any type of sales guidance numbers moving into 2025, and then for the new phase two data. This morning of I realize the numbers are small but was there any data collected on infections number of infections or types, and then particularly for those patients where.
G CSF was reduced.
Speaker Change: Sure. So sales guidance for 2025 are not yet providing that we certainly mark as Mark mentioned, we'll provide some more robust insights into our commercial launch as we head into the year.
Speaker Change: In terms of infection data I mean as you can appreciate this is a phase two study it was not designed to assess.
Speaker Change: Infection rates narrow randomized comparator or a comparator arm their history collected.
Speaker Change: Importantly that we'd really likes to remind everybody in this population AMC is proven to correlate with infection risk that is based on the G. CSF clinical trials and everyday use. So we're extremely pleased with the fact that our A&P as a robustly increasing and the neutrophils are functional we've actually gone one.
Speaker Change: Step further beyond the data with gcs and our own study.
Speaker Change: So we really hope we can remind everybody as it relates to infections of course that was part of our safety database, which will be published at a future claims, but really we're incredibly excited about the totality of evidence and are locked and loaded for the phase III.
Speaker Change: Okay, great. Thanks for taking the questions.
Speaker Change: Thank you.
Speaker Change: This conclude our Q&A session I will now turn the call over to Paula Ragan for closing comments.
Paula Ragan: Well. Thank you so much for joining us on the call today as you can appreciate we are very excited about these data and we are on track to deliver value. So from all of us at X for stay classy and have a great day.
And this does conclude today's program. Thank you.
Speaker Change: You for your participation you may disconnect at any time.
Speaker Change: Yeah.
Speaker Change: Yeah.
Speaker Change: Yeah.
Speaker Change: Hum.
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Speaker Change: Yes.
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Speaker Change: Okay.
Speaker Change: Hmm.
Speaker Change: Hum.
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Speaker Change: Got it.
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