Q1 2025 X4 Pharmaceuticals Inc Earnings Call
<unk> and answer session will follow the formal presentation.
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.
Speaker Change: Thank you operator.
Speaker Change: Good morning, everyone.
Speaker Change: Presenting on today's call will be X Force, Chief Executive Officer, Dr. Paula Ragan.
Adam: And Chief Financial Officer, Adam a staffer.
Adam: Following prepared remarks by age we will open the call to your questions and we'll be joined by Chief Commercial Officer, Mark Baldry, and Chief Medical Officer, Dr. Christoph Harbott angles.
Adam: As a reminder, on today's call the company will be making forward looking statements regarding regulatory and product development plans.
Adam: These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted.
Adam: A description of these risks can be found in X for his most recent filings with the SEC.
Adam: Including last year's Form 10-K.
And this past quarters Form 10-Q.
Adam: Which is expected to be filed after market close today.
Adam: Please note that the ex foreign Investor deck was updated this morning on the company website to include slides detailing some of the data analyses mentioned in this morning's press release.
Adam: And on this call today.
Speaker Change: I'd now like to turn the call over to exports President and CEO, Dr. Paula Ragan.
Adam: Uh huh.
Paula Ragan: Thank you Dan and thanks to all of you for joining us this morning.
Paula Ragan: The first quarter of 2025 was an extremely productive and value building period for X four with clinical trial advancement in chronic neutropenia continued progress in the commercialization of the RMB for Wednesday ground in the U S.
Paula Ragan: And our achievement of several significant milestone towards expanding the global potential of maverick's for for patients.
Paula Ragan: Let's begin with Maverick sport in chronic neutropenia or C N.
Paula Ragan: As you know having successfully built out maverick for branded Zelle, Randy for patients with whim syndrome in the U S.
Paula Ragan: We are now also developing maverick floor for the larger indication of chronic neutropenia.
Paula Ragan: There are currently about 50000 people diagnosed with some form of CN in the U S. Based on retrospective ICD 10 code analyses.
Paula Ragan: And those currently diagnosed with primary C. N. We estimate that approximately 15000 individuals are about 30% to 40% have remaining health challenges and continue to experience low absolute neutrophil count or A&P and recurrent infections. Despite available standard of care.
Paula Ragan: Yeah.
Paula Ragan: We define this as the high unmet need and patient population and it is with this population in mind that we are launching our ongoing forward trial, a global pivotal phase III clinical trial evaluating the safety and efficacy of once daily oral Maverick before and people were.
Paula Ragan: Certain chronic neutropenia condition, including primary autoimmune idiopathic.
Paula Ragan: And congenital neutropenia, and who are experiencing recurrent andrew or serious infection.
Speaker Change: As we reported on our last call, we're now screening and enrolling participants in over 20 countries with more than 90% of our targeted global trial sites being activated.
Speaker Change: Additionally, we've now finalized the trial design based on the feedback. We also discussed previously from both the FDA and EMA to focus on those with the highest unmet need a population that matches up well with Mavericks worth targeted commercial C N patient population.
Speaker Change: The trial is enrolling with those with moderate to severe C N or ANC below 1000 cells per microliter and experiencing two or more infections over the past 12 months.
Speaker Change: We also finalized the ANC response endpoints. The definition of ANC response is now uniform across all participants and is defined as an increase in AMC greater than 500 cells per microliter versus baseline E&C and occurring at 50% or more of the time points evaluated in the trial.
Speaker Change: Oh.
Speaker Change: The trial seeks to demonstrate statistically significant increases in a M series bonds and corresponding decreases in annualized infection rates between those on Maverick score versus placebo.
Speaker Change: To date, the demographics of the enrolled population are balanced and representative of the target commercial <unk> patient population and baseline and CS and historical infection rates are consistent with its high unmet need population.
Speaker Change: We recently completed some additional data analyses that further increase our confidence in the success of the forward trial.
Speaker Change: Individual patient data from both the Maverick sport phase III for whim trial, and the completed phase II <unk> trial have now been analyzed applying this just mentioned forward trial ANC response criteria.
Speaker Change: The full details of these analysis can be found in the updated investor deck that is on the front page of the investors section of our website.
Speaker Change: In summary, we created what we're calling heat maps, which detail individual ANC responses across all trial participants.
Speaker Change: At all of the assessment time point.
Speaker Change: Specifically the four women phase III heat map provides a benchmark for AMC responses that translated into a 60% reduction in annualized infection rate.
Speaker Change: When comparing maverick sport treatment to placebo.
Speaker Change: The <unk> phase two heat map demonstrates the impact of Maverick score and durable increases in AMC across those with idiopathic cyclic and congenital chronic neutropenia.
Speaker Change: Nancy outcomes in the CN safety trial looks similar to ANC responses seen in the Mavericks for arm of the four whim trial.
Speaker Change: When taken together these heat map analysis provide evidence supporting the potential success of the <unk> phase III trial, where we believe that the expected answer your responses, resulting from Maverick before treatment will correspond with significant decrease in annualized infection rates.
Speaker Change: We also continue to believe that the forward trial is rigorously designed and powered to demonstrate the impact of Maverick for N P. M.
Speaker Change: Trial is powered at greater than 95% to assess the ANC response endpoint and 150 participants sample size independently supports robust powering at greater than 90% for the infection rate revolt.
Speaker Change: As of today, we continue to anticipate full enrollment in the trial in the third or fourth quarter of 2025, which would enable disclosure of top line data in the second half of 2026.
Speaker Change: Lastly on the <unk> fronts, we have good news from the U S. Patent office, we received a notice of allowance on our application, which queens include the use of maverick's floor in treating severe chronic idiopathic and auto I mean, neutropenia and patients without a <unk> for genetic variance.
Speaker Change: The patent is expected to expire in the U S. In March of 2041.
Speaker Change: Similar patent applications are pending in Europe, China, Japan and Canada.
Speaker Change: To conclude we remain confident that we'll be able to deliver on our upcoming milestones and CN that will have a long term patent protection in the indication and that the value proposition for Mavericks for it and see and could represent a $1 billion to $2 billion opportunity in the U S alone.
Speaker Change: With that let's turn now to our progress with Mavericks, four and whim syndrome.
Speaker Change: At the end of March of this year cumulative sales of bold RMB reached $3 5 million since our mid May launch last year.
Speaker Change: This quarter sales were slightly lower than those reported in the fourth quarter because of the fluctuations in the timing of inventory resupply, which causes some lumpiness of sales there.
Speaker Change: This is typical with markets anchored in small patient populations in early in launch we do expect us to even out over time with the increasing demand that we're already seeing.
Speaker Change: We are currently in the thick of conference season, and continue to have fruitful engagements with all of our targeted top tier immunologist and hematologists, increasing the visibility of whim syndrome.
Speaker Change: And we're seeing success in our educational efforts that support hcp's and finding them patients with new patients now representing approximately 40% of our current will remedy treated population at the end of the first quarter.
Speaker Change: In addition, we're just about to kick off our whim patient Ambassador program and hope. These efforts will continue to build demand, Brazil, RMB and the U S.
Speaker Change: As we discussed on our last call we made significant progress on our efforts to expand the potential global reach of Maverick score in wind during the first quarter in January we announced that our submitted MAA was accepted by European regulatory authorities for a review.
Speaker Change: With a typical 12 to 15 month review process, we anticipate potential approval from the EMA as early as the first quarter of 2026.
Speaker Change: We also announced the completion of two international partnerships in the first quarter. The first with <unk>, a leading European specialty pharmaceutical company to commercialize Maverick score in Europe, Australia, and New Zealand.
Speaker Change: <unk> will be launching maverick before for the women indication in the EU should we receive approval. There next year and we are working closely with them to ready for that possibility.
Speaker Change: The second partnership is with Teva rare another specialty pharmaceutical company to commercialize all Randy in the Middle East and North Africa, or Mena region. Following any approvals there. The Mena region does have a compassionate use program that allows physicians to prescribe drugs approved in other countries to local patients.
Speaker Change: With no other treatment options, we will keep you updated on our progress there.
Speaker Change: Lastly, we're continuing to advance the understanding of whim syndrome, as well as the impact of maverick's floor on the disease we.
Speaker Change: We had two abstracts accepted for publication at the annual meeting of the clinical Immunology Society or C. I S which starts today.
Speaker Change: From the four women phase III open label extension or O L. Lee will be presenting at two year data that demonstrate a market clinical improvement and worst severity as assessed by a standard measurement the clinical global impression of severity across 70 defined war areas.
Speaker Change: We're also presenting results from the first ever survey looking into infection burden and when patients.
Speaker Change: 'twenty when patients provided responses, none were unsold RMB at the time.
Speaker Change: Survey revealed that 60% of those under 18 years and 73% of those 18 or over reported experiencing at least one infection in the previous three months.
Speaker Change: With 25% requiring overnight hospitalization due to infection.
Speaker Change: The study concluded that quote the frequency and severity of infections, requiring medical care and hospitalizations underscores the urgency to proactively treat patients with whim syndrome.
Speaker Change: Needless to say, we're very proud to be able to have developed the first approved therapy for women in the U S and look forward to commercial updates and continued global advancements milestones in the coming quarters I'll now turn it over to Adam to run through our financials Adam.
Adam: Thanks, Paul.
Adam: As we disclosed in the press release. This morning, we ended the first quarter of 2025 with just under $90 million in cash and cash equivalents.
Adam: We continue to believe that we have sufficient funds to support company operations into the first half of 2026.
Adam: We reported net OMD revenues of just under $1 million for the first quarter of 2025.
Adam: Paul mentioned this brings our cumulative total sales since our may 2020 for launch to about $3 $5 million.
Adam: Our R&D expenditures totaled $18 5 million for the first quarter.
SG&A expenses were $15 million for the first quarter.
Adam: And finally, we had a small amount of net income in the first quarter due to the recognition of $28 million in license and other revenue from our partnership with <unk> and a gain of $10 $8 million on our outstanding class C warrants, which are measured at fair value each quarter.
Adam: We also note that our one for 30 reverse stock split became effective on Monday, following shareholder and board approval and we believe this should cure or current deficiency with the NASDAQ listing rules.
Adam: Lastly, we have now completed the majority of the actions we laid out during our announced strategic restructuring in February.
Adam: We continue to expect that these efforts will decrease our spending by about $30 million to $35 million annually.
Adam: We will now open the call up to your questions.
Adam: Later.
Adam: Thank you and at this time, if you would like to ask a question. Please press the star and one on your telephone keypad.
Adam: We draw your question at any time by pressing star two.
Adam: Once again to ask a question. Please press star one on your telephone keypad.
We will take our first question from Ted.
Piper Sandler: Phones with Piper Sandler. Please go ahead.
Ted: Great. Thank you very much.
Adam:
Adam: Yes.
Adam: Thank you Sir.
Adam: How are you.
Adam: Thank you.
Adam: Sure.
Adam: Okay.
Adam: Volume.
Adam: Yes.
Adam: You will want to grow.
Adam: Paul.
Adam: Hey, good luck.
Adam: Paul.
Adam: Yes.
Adam: Having a little trouble here.
Adam: Great.
Adam: Color.
Adam: The profile of <unk>.
Adam: Hi, there.
Adam: But part of it.
Adam: Overall.
Adam: Sure Mark.
Adam: No.
Adam: So we have.
Adam: Starting in itself is very has very specific criteria.
Adam: And we have the profile of the patients that we are having is really really good so far and we're tracking this.
Adam: What I can share.
Adam: Totally.
Adam: Having met with some of the Ti and leasing with some others has got a lot of those we'd love to include many more patients than innovations we have into the study.
Adam: And they often have.
Adam: All of these patients waiting became not changed their treatment just to meet the study obviously.
Speaker Change: Okay demand here and interesting.
Adam: I'm trying to bring as many patients as possible into the study.
Yes.
Speaker Change: Yes.
Speaker Change: Maybe mark has had one of our government there yes, we.
Speaker Change: Continuing to build our insights into.
Speaker Change: But the market itself and actually just completed a large survey with about 95 U S physicians, who treat chronic neutropenia and what we're finding is the case loads of patients that physicians have are much higher than in wins. So in women's it's a very fragmented model, but in <unk>.
Speaker Change: Rock neutropenia, it's much more concentrated much more defined because there are.
Speaker Change: Dave definitive ICD 10 diagnosis codes, so we can clearly see where.
Speaker Change: These patients are on the unmet need in this refractory population.
Speaker Change: Yeah.
Speaker Change: I think I'll just quickly summarize for you had very sick patients being enrolled in the study to enrich for success on the infection endpoint higher demand locally in a trial to get there.
Speaker Change: In the trial, but of course, we can't accept everybody in that market seeing that fall through in terms of the higher cash flows through our market research.
Speaker Change: Great Thats very helpful. Thank you.
Piper Sandler: Thank you Ben.
Speaker Change: Thank you. Our next question comes from RK with H C. Wainwright. Please go ahead.
Speaker Change: Thank you good morning, Paul.
Speaker Change: Couple of quick questions.
Speaker Change: In the.
Speaker Change: And the patient.
Speaker Change: The amount of patients that you said, who are being diagnosed with CN, which is like 50000 people and out of that 15000 are the ones that seem to be having the high unmet need.
Speaker Change: These numbers are they just use our the worldwide.
Speaker Change: They're just U S. RK, yes, we did ICD 10 coding analysis in the U S claims data.
Speaker Change: Okay, perfect and then from from your comments to Ted's question. You know you are stating that wouldn't be very sick patients out opinion goes into the study so I'm thinking about the label then what.
Speaker Change: What sort of it.
Speaker Change: You know the.
Speaker Change: Target population would you be looking at on the label is a very sick population or is that.
Speaker Change: Is it beyond that.
Speaker Change: We defined that population.
Christophe: So because RK. This is christophe here because we do have already also from the Csp's two experienced we've also other population even if we were.
Christophe: Phase III study includes moderate and severe patients we're going to build the case for the label for the entire CN population and we do have already data support this.
Christophe: Drilling program wind study et cetera, so we.
Christophe: We are anticipating a broad label.
Christophe: Obviously this will be the amount of discussions with the FDA at the time when these comps.
Christophe: I mean, our cadence to kind of cross walk obviously end of the value proposition. We are focusing on treating patients who are basically refractory with severe recurrent in fashion that is who needs remaining treatment and certainly when we go to the payer systems across the world, we want to be focusing on that basically that high unmet need.
Christophe: With little to no option to to demonstrate the value proposition of adult Randy for the Maverick sport right now is the anvil, Remy and women maintain our price point. So they are they a good connection between the severity of the disease. The trial design and the ultimate value that we think we can bring to patients.
Christophe: Okay. One last question I'm sorry.
Christophe: The the turn.
Speaker Change: Action with the or the agreement with nor gene that you currently have.
Christophe: Sure.
Christophe: Commercialization in Europe and Australia.
Speaker Change: Does that go by indication or does that go by Maverick support period.
Christophe: Yes. Thanks.
Christophe: Joe.
Christophe: <unk> <unk> and <unk>.
Christophe: So it's a license to the assay across both indications obviously first would be when commercially followed by <unk>.
Christophe: Okay perfect. Thanks, Thanks for taking my questions.
Christophe: Thank you okay.
Christophe: Thank you and as a reminder, it is star one if you would like to join the queue.
Speaker Change: We will move next with Stephen Wiley with Stifel. Please go ahead. Your line is open.
Stephen Wiley: Yes. Good morning, Thanks for taking my questions.
Stephen Wiley: I guess with the understanding that the commercial history here and when it is a bit abbreviated thus far.
Stephen Wiley: Is there anything that you can say about just patient persistency and compliance.
Stephen Wiley: What youre seeing.
Stephen Wiley: Sure Good morning, Steve.
Stephen Wiley: Mark I mean.
Stephen Wiley: I think what we're pleased with is that although we're not giving out any actual numbers were pleased to see that compliance and adherence rates are actually higher than you would expect with a daily oral medication and I think that speaks to.
Stephen Wiley: The unmet need here and that these patients and physicians understand that this disease needs treatment.
Speaker Change: So Randy is the solution I can state.
Stephen Wiley: And appropriately so.
Stephen Wiley: We're now actually spending a lot of time, beginning to educate patients and the patient community and.
Stephen Wiley: In fact <unk>.
Stephen Wiley: Cited too.
Speaker Change: The announcement, we just launched a new website yesterday in fact I encourage you to open your browser of choice since I've been with syndrome Dot com and Youll see our new patient education web site that features when patients telling their stories and also provides.
Speaker Change: A lot of resources to help them on their journey with women with no revenue.
Speaker Change: Okay and are most patients getting 30 day supply or some patients getting three months worth of drug.
Speaker Change: <unk> script.
Speaker Change: The majority of patients are on on the higher doses of course, it's weight based so.
Speaker Change: If a patient is a lighter weight they get a lighter dose.
Speaker Change: But I guess the question was.
Speaker Change: If the prescription unit size that most patients are getting is it a bunch of supply or some patients getting three months worth of drugs with a single Joe It's about it's a month's supply at this stage okay.
Speaker Change: Okay.
Speaker Change: And then understanding that the full trial is blinded.
Speaker Change:
Speaker Change: But can you just remind us what your assumption was around patient dropout.
Unknown Executive: A question-and-answer session will follow the formal presentation.
Unknown Executive: As a reminder, this conference call is being recorded.
Speaker Change: If there is any data that youre able to see on a blinded basis that would suggest that that assumption is holding up in the clinical trial.
Daniel Ferry: It is now my pleasure to introduce your host, Dan Ferry, from LifeSci Advisors. Please begin.
Unknown Executive: Thank you, operator.
Speaker Change: Yeah. So.
Speaker Change: Steve what we're targeting as we shared at about 150 patients enrolled and all of that has some degree of assumptions of either dropouts.
Paula Ragan: Presenting on today's call will be X4's Chief Executive Officer, Dr. Paula Ragan, and Chief Financial Officer, Adam Mostafa.
Speaker Change: Or screen failures et cetera start building that funnel I think more importantly, what we can see with the <unk> subject is really about their profile like army in good shape on the 150 that sort of stuff are o'clock yesterday are definitely yes, we are seeing the rate of land idiopathic.
Paula Ragan: Following preparatory marks by each, we will open the call to your questions and will be joined by Chief Commercial Officer, Mark Baldry, and Chief Medical Officer, Dr. Christophe Arbet, X4 Pharmaceuticals.
Paula Ragan: As a reminder, on today's call, the company will be making forward-looking statements regarding regulatory and product development plans. These statements are subject to risks and uncertainties that may cause actual results to differ from those forecast. A description of these risks can be found in X4's most recent filings with the SEC. including last year's form, 10-K. and this past quarter's Form 10-Q.
Speaker Change: Autoimmune and congenital balanced nicely and then of course this offline, but we're seeing the event rates that you would want to see early in the studies that confirm our assumptions. So in terms of powering that the 150 yourself as the centers for the co primary endpoints. So I have to give that could give some comments in terms of the rate.
Speaker Change: So many ins and outs, while we can't say, it's certainly based on everything we're seeing we're on track for that Q3 Q4 for a moment.
Paula Ragan: which is expected to be filed after market close today.
Paula Ragan: And please note that the X4 Investor Deck was updated this morning on the company website to include slides detailing some of the data analyses mentioned in this morning's press release and on this call today.
Speaker Change: Okay very helpful. Thanks for taking my questions.
Speaker Change: Okay. Thank you Steve.
Speaker Change: Thank you and we show no further questions at this time I will turn the call back to Paula Ragan for closing remarks.
Paula Ragan: I'd now like to turn the call over to X4's President and CEO, Dr. Paula Ragan. Paula?
Paula Ragan: Well. Thank you very much for joining us today I'm happy to follow up offline with any other questions and wish you all an excellent rest of your day. Thank you.
Paula Ragan: Thank you, Dan, and thanks to all of you for joining us this morning. The first quarter of 2025 was an extremely productive and value-building period for X4, with clinical trial advancement in chronic neutropenia, continued progress in the commercialization of Zoll Remedy for Wim Syndrome in the U.S., and our achievement of several significant milestones towards expanding the global potential of Maverix IV for patients.
Speaker Change: And this does conclude today's program. Thank you for your participation you may disconnect at any time.
Paula Ragan: Let's begin with Maverick Support and Chronic Neutropenia, or CN. As you know, having successfully developed Maverix IV, branded Zol Remedy for patients with Wim Syndrome in the U.S., we are now also developing Maverix IV for the larger indication of chronic neutropenia. There are currently about 50,000 people diagnosed with some form of CN in the U.S. based on retrospective ICD-10 code analyses. Of those currently diagnosed with primary CN, we estimate that approximately 15,000 individuals, or about 30 to 40%, have remaining health challenges and continue to experience low absolute neutrophil counts, or ANCs, and recurrent infections despite available standard of care.
Paula Ragan: We define this as the high unmet need CNN patient population.
Paula Ragan: And it is with this population in mind that we are launching our ongoing Forward Trial, a global pivotal phase 3 clinical trial evaluating the safety and efficacy of once daily oral Maverick support in people with certain chronic neutropenic conditions, including primary autoimmune, idiopathic, and congenital neutropenia and who are experiencing recurrent and or serious infection. As we reported on our last call, we're now screening and enrolling participants in over 20 countries with more than 90% of our targeted global trial sites being activated. Additionally, we've now finalized the trial design based on the feedback we also discussed previously from both the FDA and EMA to focus on those with the highest unmet needs.
Paula Ragan: A population that matches up well with Mavericks for a targeted commercial CN patient population. The trial is enrolling with those with moderate to severe CN, or A and C below 1000 cells per microliter, and experiencing 2 or more infections over the past 12 months. We also finalized the ANC response endpoint. The definition of ANC response is now uniform across all participants and is defined as an increase in ANC greater than 500 cells per microliter versus baseline ANC and occurring at 50% or more of the time points evaluated in the trial. The trial seeks to demonstrate statistically significant increases in ANC response and corresponding decreases in annualized infection rates between those on Maverix IV versus placebo.
Paula Ragan: To date, the demographics of the enrolled population are balanced and representative of the target commercial CN patient populations and baseline ANCs and historical infection rates are consistent with this high unmet need population.
Paula Ragan: We recently completed some additional data analyses that further increase our confidence in the success of the FORWARD trial. Individual patient data from both the Maverix IV Phase III 4-WIM trial and the completed Phase II CN trial have now been analyzed applying this just-mentioned forward trial ANC response criteria.
Paula Ragan: The full details of these analyses can be found in the updated investor deck that is on the front page of the investor section of our website. In summary, we created what we're calling heat maps, which detail individual A and C responses across all trial participants at all of the assessed time points. Specifically, the 4 WHM Phase 3 heat map provides a benchmark for ANC responses that translated into a 60% reduction in annualized infection rates when comparing Maverick support treatment to placebo. The CM Phase II heat map demonstrates the impact of Maverix for undurable increases in A and C across those with idiopathic, cyclic, and congenital chronic neutropenia.
Paula Ragan: A&C outcomes in the CN Phase II trial look similar to A&C responses seen in the Mavericks IV arm of the IV WIM trial.
Paula Ragan: When taken together, these heat map analyses provide evidence supporting the potential success of the CN Phase 3 trial, where we believe that the expected ANC responses resulting from Maverick support treatment will correspond to a significant decrease in annualized infection rates. We also continue to believe that the forward trial is rigorously designed and powered to demonstrate the impact of Mavericks for NCM. The trial is powered at greater than 95% to assess the ANC response endpoints, and the 150 participant sample size independently supports robust powering at greater than 90% for the infection rate results.
Paula Ragan: As of today, we continue to anticipate full enrollment in the trial in the 3rd or 4th quarter of 2025, which would enable disclosure of top-line data in the 2nd half of 2025.
Paula Ragan: Lastly, on the CIN front, we have good news from the U.S. Patent Office. We received a Notice of Allowance on our application, which claims include the use of Maverix IV in treating severe chronic idiopathic and autoimmune neutropenia in patients without a CXCR4 genetic variant. The patent is expected to expire in the U.S. in March of 2041. Similar patent applications are pending in Europe, China, Japan, and Canada.
Paula Ragan: To conclude, we remain confident that we'll be able to deliver on our upcoming milestones in CN, that we'll have a long-term patent protection in the indication, and that the value proposition for Maverix IV in CN could represent a $1 to $2 billion opportunity in the U.S. alone.
Paula Ragan: With that, let's turn now to our progress with Maverick TOR and WIM syndrome. At the end of March of this year, cumulative sales of Goal Remedy reached 3.5 million since our mid-May launch last year. This quarter's sales were slightly lower than those reported in the fourth quarter because of the fluctuations in the timing of inventory resupply, which caused some lumpiness to sales. This is typical with markets anchored in small patient populations and early in launch. We do expect this to even out over time with the increasing demand that we're already seeing. We are currently in the thick of conference season and continue to have fruitful engagements with all of our targeted top-tier immunologists and hematologists, increasing the visibility of Wimp syndrome.
Paula Ragan: And we're seeing success in our educational efforts that support HCPs and finding limb patients, with new patients now representing approximately 40% of our current Vol-Remedy-Treat population at the end of the 1st quarter. In addition, we're just about to kick off our WIMPatient Ambassador Program, and hope these efforts will continue to build demand for Zil-Renvi in the U.S.
Paula Ragan: As we discussed on our last call, we made significant progress in our efforts to expand the potential global reach of Maverick's score in WIM during the first quarter. In January, we announced that our submitted MAA was accepted by European regulatory authorities for review. With a typical 12- to 15-month review process, we anticipate potential approval from the EMA as early as the first quarter of 2020.
Paula Ragan: We also announced the completion of two international partnerships in the first quarter. The first with Norgene, a leading European specialty pharmaceutical company, to commercialize Mavericks for in Europe, Australia, and New Zealand. Nourgeen will be launching Mavericks for the WIM indication in the EU should we receive approval there next year, and we are working closely with them to ready for that possibility. The second partnership is with Teba Rare, another specialty pharmaceutical company, to commercialize Zol Remde in the Middle East and North Africa, or MENA region, following any approvals there. The MENA region does have a compassionate use program that allows physicians to prescribe drugs approved in other countries to local patients with no other treatment options.
Paula Ragan: We'll keep you updated on our progress there.
Paula Ragan: Lastly, we're continuing to advance the understanding of Lyme syndrome, as well as the impact of Maverick's 4 on the disease. We had two abstracts accepted for publication at the annual meeting of the Clinical Immunology Society, or CIS, which starts today. From the 4 WHAM Phase III Open Label Extension, or OLE, we'll be presenting two-year data that demonstrate a marked clinical improvement in WART severity as assessed by a standard measurement, the clinical global impression of severity across 70 defined WART areas. We're also presenting results from the first ever survey looking into infection burden in WIM patients.
Paula Ragan: Twenty WIM patients provided responses, none were on Zolrembi at the time. The survey revealed that 60% of those under 18 years and 73% of those 18 or over reported experiencing at least one infection in the previous three months. With 25% requiring overnight hospitalizations due to infection. The study concludes that, quote, the frequency and severity of infections requiring medical care and hospitalizations underscores the urgency to proactively treat patients with WIM syndrome.
Paula Ragan: Needless to say, we're very proud to be able to have developed this first approved therapy for WIM in the U.S. and look forward to commercial updates and continued global advancement milestones in the coming quarters.
Adam Mostafa: I'll now turn it over to Adam to run through our financials. Adam? Thanks, Paula. As we disclosed in the press release this morning, we ended the first quarter of 2025 with just under $90 million in cash and cash equivalents.
Adam Mostafa: We continue to believe that we have sufficient funds to support company operations into the first half of 2020. We reported net Zole Remedy revenues of just under $1 million for the first quarter of 2025. As Paula mentioned, this brings our cumulative total sales since our May 2024 launch to about $3.5 million. Our R&D expenditures totaled $18.5 million for the first quarter, and our SG&A expenses were $15 million for the first quarter.
Adam Mostafa: And finally, we had a small amount of net income in the first quarter due to the recognition of $28 million in license and other revenue from our partnership with Norgene, and a gain of $10.8 million on our outstanding Class C warrants, which are measured at fair value each quarter.
Adam Mostafa: We also note that our 1 for 30 reverse stock split became effective on Monday following shareholder and board approval, and we believe this should cure our current deficiency with the NASDAQ listing rule. Lastly, we have now completed the majority of the actions we laid out during our announced strategic restructuring in February. We continue to expect that these efforts will decrease our spending by about $30 to $35 million annually.
Unknown Executive: We'll now open the call up to your questions.
Unknown Executive: Operator? Thank you.
Unknown Executive: And at this time, if you would like to ask a question, please press the star and 1 on your telephone keypad. You may withdraw your question at any time by pressing star 2. Once again, to ask a question, please press star and 1 on your telephone.
Edward Tenthoff: Let's take our first question from Ted Tenthoff with Piper Zandler, please go ahead. Great. Thank you very much.
Edward Tenthoff: Can you give us a sense, if you have any visibility, into the types of patients that are being enrolled in this program? Is it largely in line with what you expect? Is that you are enrolled in the pre-purchasing space we saw? And then I guess we'll follow up on when...
Mark Baldry: I'm having a little trouble hearing you, but I think what you're looking for is the word color and the enrollment profile of the patient and how that connects to the commercial market. So, of course, we mentioned that we're very pleased with the overall profile. So we have the study in itself has very specific criteria, and we have the profile of the patients that we are having is really good so far, and we're tracking this.
Mark Baldry: What I can share anecdotally, having met with some of the PIs and meeting with some others is that a lot of those PIs would love to include many more patients than the patients we have in the study, and they often have all these patients waiting, they cannot change their treatment just to include them in the study obviously, but there's a clear demand here and interest in trying to bring as many patients as possible into the study. Great, thank you very much. Sure.
Mark Baldry: I mean, Mark just had one more comment there. Yeah. Ted, I was just going to say, you know, we're continuing to build our insights into the CN market itself, and actually just completed a large survey with about 95 U.S. physicians who treat chronic neutropenia, and what we're finding is, you know, the caseloads of patients that these physicians have are much higher than in WIM. So in WIM, it's a very fragmented model, but in chronic neutropenia, it's much more concentrated, much more defined, because there are distinct, definitive ICD-10 diagnosis codes, so we can clearly see where these patients are and the unmet need in this refractory population.
Edward Tenthoff: I think just to quickly summarize what you said, very sick patients being enrolled in the study to enrich for success on the infection endpoint, higher demand both locally in the trial, because they'd love to get their patients in the trial, but of course, we can't accept everybody. And then market seeing that pull through in terms of the higher caseloads through our market research. Great. That's very helpful. Thank you. Thank you, Penn.
RK: Our next question comes from RK with HC Wainwright. Please go ahead. Thank you.
RK: Good morning, Paula and Adam. A couple of quick questions. You know, in the In the patient, the amount of patients that you said who are being diagnosed with CN, which is like 50,000 people, and out of that 15,000 are the ones that seem to be having the high unmet need, these numbers, are they just U.S., or are they worldwide? They're just U.S., okay, yeah, we did ICD-10 code analysis and the U.S. claims data.
Christophe Arbet: Okay, okay, perfect, and then from your comments to Ted's question, you know, you were stating that only very sick patients are being enrolled into the study, so I'm thinking about the label, what sort of a, you know, the target population would you be looking at on the Is it the very sick population, or is that beyond that, and how do we define that population? So because RK, this is Christophe here, because we do have already also from the CN phase 2 experience with also other populations, even if the phase 3 study includes moderate and severe patients, we are going to build a case for the label for the entire CN population, and we do have already data to support this, including from the WIM study, etc.
Christophe Arbet: So we are anticipating a broad label, but obviously this will be a matter of discussions with the FDA at the time when it comes. So, I mean, our case is to kind of crosswalk, obviously, into the value proposition. We are focusing on treating patients who are basically refractory with severe recurrent infections. That is who needs remaining treatment, and certainly, when we go into the payer systems across the world, we want to be focusing on that, basically, that high-invent need with little to no options to demonstrate the value proposition of Zolrembi, for example, at Brickscore right now.
Christophe Arbet: At the end, Zolrembi can win and maintain our price point. So, there's a good connection between the severity of the disease, the trial design, and the ultimate value that we think we can bring to patients.
RK: One last question. I'm sorry. The transaction or the agreement with Norgene that you currently have for commercialization in Europe and Australia, does that go by indication or does that go by Maverick's four periods? Yeah, thanks, Adam. So it covers WIM and CN. So it's licensed to, yeah, across both indications. Obviously, first will be WIM commercially followed by CN. Okay, perfect. Thanks.
RK: Thanks for taking my question.
Unknown Executive: Thank you, RK. And as a reminder, it is a star and one if you would like to join the.
Stephen Willey: We will move next with Stephen Wiley with Stifel. Please go ahead. Your line is open.
Stephen Willey: Yeah, good morning. Thanks for taking the questions.
Stephen Willey: I guess with the understanding that the commercial history here in William is a bit abbreviated thus far. Is there anything that you can say about just patient persistency and compliance?
Mark Baldry: That's your Good morning, Steve. Mark here. Yeah, I mean, I think what we're pleased with is that, although we're not getting down any actual numbers, we're pleased to see that compliance and adherence rates are actually higher than you would expect with a daily oral medication. And I think that speaks to the unmet need here, and that these patients and physicians understand that this disease needs treatment, and Zolrembi is the solution, if it's taken appropriately. So we're now actually spending a lot of time beginning to educate patients and the patient community, and in fact, excited to announce that we just launched a new website yesterday.
Mark Baldry: In fact, I encourage you to open your browser of choice and type in WIMSyndrome.com, and you'll see our new patient education website that features WIM patients telling their story, and also provides a lot of resources to help them on their journey with WIM and with Zolrembi.
Mark Baldry: Okay, and are most patients getting a 30-day supply or are some patients getting three months worth of drug via a single dose? The majority of patients are on the higher dose. Of course, it's weight-based, so if the patient is of a lighter weight, they get a lighter dose. And I guess the question was, If the prescription unit size that most patients are getting, is it a month's supply, or are some patients getting three months' worth of drug with a single prescription? No, it's a month's supply at this stage. Okay.
Stephen Willey: And then understanding that the forward trial is blinded.
Mark Baldry: But can you just remind us what your assumption was around patient dropout and if there's any data that you're able to see on a blinded basis that would suggest that that assumption is holding up in the clinical trial? Yeah, so, you know, see, what we're targeting, as we shared, is about 150 patients enrolled, and all of that has some degree of assumptions of either dropouts or screen failures, etc. So, we're building that funnel. I think, more importantly, what we can see with the RE-enrolled subjects is really about their profile. Like, are we in good shape on the 150s?
Mark Baldry: Because that sort of sets our clock. The answer there is definitely yes. We're seeing the right blend of idiopathic, autoimmune, and congenital. It's balanced nicely. And then, of course, this is all blinded, but we're seeing the event rates that you would want to see early in the study to confirm our assumptions. So, in terms of powering, that's 150 is belts and suspenders for the co-primary endpoints. So, I think that could give you some confidence.
Stephen Willey: In terms of the rate, there's so many ins and outs. What we can say is, certainly, based on everything we're seeing, we're on track for that Q3, Q4 funnel. Okay, very helpful. Thanks for taking the question.
Unknown Executive: And we show no further questions at this time.
Paula Ragan: I will turn the call back to Paula Ragan for closing remarks. Well thank you very much for joining us today. Happy to follow up offline with any other questions and wish you all an excellent rest of your day. Thank you.
Unknown Executive: And this does conclude today's program. Thank you for your participation. You may disconnect at any time.