Q4 2023 X4 Pharmaceuticals Inc Earnings Call
Paula Ragan: Given its rarity and lack of available therapies, disease awareness amongst our targeted physician audience has historically been quite low, with no approved treatments for Wim and only supportive care for symptomatic management, not addressing the underlying cause of the disease. With our successful Phase III WIM trial recently being presented at major medical conferences, we are very encouraged that the physician community is now gaining more awareness. Additionally, we've experienced strong engagement with our What If It's WHIM Disease Awareness Campaign, which is also demonstrating an even broader interest in WHIM syndrome. At the ASH meeting in December, the organizers made a point of highlighting the need for new research and development in classical hematological diseases. At the meeting, our Chief Medical Officer, Dr. Christophe Arbet Engels, gave a talk on the lack of innovation and the need for new treatments. His presentation was enthusiastically welcomed by a standing room-only audience.
Greetings and welcome to the X for Pharmaceuticals, fourth quarter, and full year 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 is being recorded.
Speaker Change: It is now my pleasure to introduce your host.
Speaker Change: Dan Ferry from life Science prizes. Please begin.
Daniel Ferry: Thank you operator and good.
Daniel Ferry: Morning, everyone.
Daniel Ferry: Presenting on today's call will be X Force, Chief Executive Officer, Dr. Paula Ragan, and Chief Financial Officer, Adam Mostafa.
Daniel Ferry: Following prepared remarks by each we will open the call to your questions and we will be joined by Chief Commercial Officer Mark Baldry.
Daniel Ferry: Chief Medical Officer, Dr. Christoph.
Daniel Ferry: Christoph Ahmet angles chief.
Chief operating officer, Barry Deep Yahtzee, and Chief Scientific Officer Art Terrace.
Daniel Ferry: 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.
Daniel Ferry: These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted.
Paula Ragan: We're also pleased to report that at the recent Quad AI Immunology meeting in late February, not only was our abstract on the lymphocyte subset data from our 4-WIM trial accepted as an oral presentation by Dr. Theresa Tarrant, an esteemed immunologist from Duke University, whom you've heard from at our several past investor events, but Dr. Tarrant was also invited by the conference organizers to give a non-X4-sponsored talk Both of her presentations were extremely well-attended, as was our what-if-it's-a-whim boost, all of which served to increase our visibility and enable what we believe can result in a major leap forward for patients through physician awareness and education. The X4 team working on our Congress exhibition booths have recounted many stories of physicians thanking them for drawing their attention to this disorder. And they've reported that multiple physicians who approached us, never before having heard of Wim Syndrome, left believing they might, in fact, have a Wim patient under their care.
Daniel Ferry: Description of these risks can be found in <unk>, most recent filings with the SEC.
Daniel Ferry: Including this year's Form 10-K.
Daniel Ferry: Expected to be filed after market close today.
Speaker Change: I'd now like to turn the call over to exports President and CEO.
Paula Ragan: Paula Ragan.
Paula Ragan: Paula.
Paula Ragan: Thanks, Dan.
Paula Ragan: Everyone and thank you for joining us on the call today.
Paula Ragan: We look ahead to 2024, we thought we'd take the times reflect on all that we've accomplished over the past year milestones that have set the stage for what we expect will be an incredibly exciting and truly transformative year for X for a year, where we aim to become a fully integrated research development and commercialization company.
Paula Ragan: <unk> of delivering new options for rare disease patients.
Paula Ragan: Throughout 2023, we had several major milestones most importantly submission and acceptance under FDA priority review process of our NDA seeking approval of Maverick before our oral targeted <unk> four antagonist for the treatment of women syndrome as.
Paula Ragan: As you May recall women's a rare combined primary immunodeficiency caused by genetic variations to the <unk> four receptor <unk>.
Paula Ragan: Cause the exterior for pathway helps regulate the migration of white blood cells, including neutrophils and lymphocytes into the bloodstream.
Paula Ragan: When is named for its for most common manifestation.
Paula Ragan: Ports, which are typically caused by unresolved HPV infection.
Paula Ragan: Hypo, Daniela globular anemia or low levels of antibodies.
Paula Ragan: As you can imagine, we often get asked the question about the size of the market for women in the U.S. With rare diseases that have no treatments, it's always difficult to assess. These interactions with physicians serve to reinforce our belief that there may, in fact, be more WIMP patients out there beyond the 1,000 or so that we've estimated are diagnosed at present based on claimed data and analyses. And while we eagerly await word from the FDA on our first NDA, we're continuing to advance our plans to seek approval for Maverick, Sephora, and WHAM outside of the U.S. as well. We've had productive interactions with regulators in Europe and now believe we may be able to submit for EMA approval in late 2024 or early 2025. We've had discussions with potential partners on how best to leverage our U.S. approval if it is received across other geographies as well. More to come on that later this year.
Paula Ragan: Sections.
Paula Ragan: IRA and bacterial <unk>.
Paula Ragan: Milo success, our retention of white blood cells in the bone marrow.
Paula Ragan: Diagnosis of win has historically proven challenging because only a minority of patients actually experience all four manifestations and the acronym <unk>.
Though not often times are required for diagnosis.
Paula Ragan: The most challenging burden faced by people with whim syndrome results from their low blood levels of neat yourselves or neutropenia, and low blood levels of lymphocyte Lymphopenia, which research supports our experienced by essentially all whim patient.
As a consequence of neutropenia in Lymphopenia and estimated 92% a whim patient experience frequent recurrent infections that significantly impact their health and quality of life.
Paula Ragan: Over time, when patients with recurrent infections may experience debilitating and life threatening complications, including increased cancer risk irreversible end organ damage and our success.
Paula Ragan: I mentioned this not only to emphasize the incredible burden of disease for these patients and their caregivers, but also to highlight that Mavericks floor has been granted breakthrough therapy designation.
Paula Ragan: The FDA, indicating their recognition of Maverick before as a product candidate with the potential to provide substantial improvement over the standard of care in the treatment diagnosis or prevention of a serious condition.
Paula Ragan: Now turning to our chronic uterpenia program, we were able to make significant progress in advancing Mavericks IV and CN during 2023 as well. Although our Phase 2 clinical trial was a little slower to enroll than we'd initially expected, we were able to learn and increase enrollment and achieve our target of at least 15 patients by early November. And we continue to expect to announce additional Phase 2 results in the 15 plus trial participants in the coming months. We expect that our data to be presented will include a meaningful number of patients who are receiving Maverix X4 alone without concomitant GCSF treatment, enabling the assessment of Maverix X4 as a potential monotherapy in those diagnosed with CN.
Paula Ragan: So we were very pleased to have reported on the additional positive safety and efficacy results from our completed global pivotal phase III trial in win in May of last year and further analysis of the trial at several important medical meetings and Congresses, both last year and earlier this year.
Paula Ragan: Publication of the full four whim trial results.
Paula Ragan: Currently pending at a highly respected international medical Journal.
Paula Ragan: As you know the phase II trial successfully met its primary endpoint and several key secondary endpoints with participants on maverick's for achieving statistically significant elevations in both their neutrophil and lymphocyte counts versus placebo and importantly, statistically significant reductions in the.
Rate of annualized infections, and clinically meaningful reductions in the severity and duration of infections versus placebo.
Paula Ragan: We also expect that data to be presented will include information on additional participants being treated with a combination of Maverix X4 and GCSF. Importantly, all of the preliminary data presented to date and other learnings from the Phase 2 trial, along with our interactions with the FDA, have enabled us to finalize the protocol for a global, pivotal Phase 3 clinical trial of Mavericks 4 and CN and advance the initiation of this important next trial in the first half of 2024. As we've previously discussed, we plan to enroll approximately 150 participants in the trial, which will be a 52-week, double-blinded, placebo-controlled trial with one-to-one randomization. We will be assessing the safety and efficacy of Maverix IV, plus or minus concomitant GCSF treatment in those with congenital, autoimmune, or idiopathic neutropenia.
Paula Ragan: Based on these results we submitted our first NDA to the FDA in late August of last year, receiving notice of acceptance for priority review setting a target <unk> date of April 30, which is fast approaching.
Paula Ragan: As you can well appreciate we've been incredibly busy preparing for the potential approval since we spoke with you last year.
Paula Ragan: Been having discussions with our payers multiple meetings with FDA and finalizing our supply and distribution arrangements to be ready in the event that we receive FDA approval.
Paula Ragan: Our commercial and medical affairs organizations have grown meaningfully although prudently as we've continued to build our brand marketing and sales infrastructure to increase our presence at medical meetings and engage with our targeted hematologists and immunologist that we expect to have when patients under their care.
Paula Ragan: We look forward to leveraging our rare disease commercial capabilities to start building the wind market over time with 2024 as the year to lay the foundation for future success and looking beyond 2024, as we are successful in educating physicians, helping them identify when patients and build demand for our product candidate.
Paula Ragan: <unk> syndrome is a very rare form of combined immunodeficiency first described over 60 years ago in the medical literature.
Paula Ragan: The patients included in the study will also have to present with neutropenia and symptomatic infections, despite the current standard of care, including GCSF. There is a significant unmet medical need across this established phase 3 patient population, a U.S. market we estimate to represent approximately 15,000 people. The primary endpoint will be a two-component endpoint comprised of both the annualized infection rate and ANC responder analysis across the study population. Secondary endpoints will include the severity and duration of infections, antibiotic use, quality of life measurements, among others, and, of course, safety.
Paula Ragan: Given its rarity and lack of available therapies disease awareness amongst our targeted physician audience has historically been quite low with no approved treatments for win and only supportive care for symptomatic management not addressing the underlying cause of the disease.
Paula Ragan: With our successful phase III <unk> trial recently being presented at major medical conferences.
Paula Ragan: We're very encouraged that the physician community is now gaining more awareness.
Paula Ragan: Additionally, we've experienced strong engagement with our what if it's a whim disease awareness campaign, which is also demonstrating an even broader interest in a whim syndrome.
Paula Ragan: At the Ash meeting in December the Organisers made a point of highlighting the need for new research and development and classical Hematological diseases.
Paula Ragan: At the meeting our Chief Medical Officer, Dr. Christoph <unk> Ingalls, David talk on the lack of innovation and the need for new treatments at the meeting his presentation was enthusiastically welcome by a standing room only audience.
Adam S. Mostafa: We are in the process of initiating our global study sites and are looking forward to enrolling patients across a number of these sites beginning in the next few months. We are, as of now, planning to host a CN event before the end of June to update on both the Phase 2 trial data and CN Phase 3 trial progress, so stay tuned for more information on that. I'll now turn it over to our CFO, Adam Mostafa, to review the fourth quarter and full year 2023 financials. Adam?
Paula Ragan: We're also pleased to report that at the recent Quad AI Immunology meeting in late February not only was our abstract on the lymphocyte subset data from our four whim trial accepted as an oral presentation by Dr. Theresa tyrant and esteemed Immunologists from Duke University, who Ive heard from are several past investor events.
But Dr. <unk> was also invited by the conference organizers to give a non X for a sponsor talk on maverick's, four and whim syndrome specifically.
Paula Ragan: In her presentation, we're extremely well attended as it was or what if it's a win lose all of which served to increase our visibility and enable what we believe can result in a major leap forward for patient to physician awareness and education.
Adam S. Mostafa: Thanks, Paula, and thanks to all of you for being on the call with us. Having raised more than $87 million during 2023, comprised of a mix of equity and debt capital through an at-the-market pipe and through our expanded loan facility with Hurricane, we ended 2023 with $115.2 million in cash and cash equivalents, short-term marketable securities, and restricted cash. We expect that these funds will support our operations into 2025. Additionally, we would like to note that we may have multiple non-dilutive sources of funding available to us throughout 2024. Given Maverix IV's rare pediatric disease designation, we are optimistic that we'll receive a priority review voucher, which can be used for a subsequent application or sold to another drug sponsor, should Maverix IV be approved.
Paula Ragan: The extra team working on our Congress exhibition booths have recounted many stories of physicians thanking them for drawing their attention to this disorder and they reported that multiple physicians, who approached us never before having heard of whim syndrome left believing they might in fact have a whim patients under their care.
Paula Ragan: As you can imagine we get the question often about the size of the market for women in the U S.
Paula Ragan: With rare diseases that have no treatments, it's always difficult to assess.
Paula Ragan: These interactions with physicians serve to reinforce our belief that there may in fact be more when patients out there beyond the 1000 or so that we've estimated our diagnosed at present based on claims data and analyses.
Paula Ragan: And while we eagerly await word from the FDA on our first NDA for continuing to advance our plans to seek approval for Maverick before and Wham outside of the U S as well.
Paula Ragan: We've had productive interactions with regulators in Europe, and now believe we may be able to submit for EMA approval in late 2020 for early 2025, we.
Paula Ragan: We've had discussions with potential partners on how best to leverage our U S approval if received across other geographies as well.
Adam S. Mostafa: If all goes well, we also may have additional access to milestone-based debt tranches to our Hercules facility this year and anticipate revenues from U.S. sales of Maverick support over the course of this year and beyond. A quick recap of our 2023 financials. R&D expenses were $15.3 and $72 million for the fourth quarter and full year ended December 31, 2023, respectively, compared to $19 and $61.1 million for the comparable periods in 2022. Our R&D expenses included $1.1 and $4.4 million of certain non-cash expenses for the fourth quarter and full year ended December 31, 2023, respectively. SG&A expenses were $9.9 and $35.5 million for the fourth quarter and full year of 2023, respectively, compared to $6.6 and $27 million for the comparable periods.
Paula Ragan: To come on that later this year.
Paula Ragan: Now turning to our chronic neutropenia program, we were able to make significant progress in advancing Mavericks for MCM during 2023 as well.
Paula Ragan: Although our phase II clinical trial with a little slower to enrol than we'd initially expected we were able to learn and increase enrollment and achieve our target of at least 15 patients by early November and we continue to expect to announce additional phase two results and the 15 plus trial participants in the coming months.
We expect that our data to be presented will include a meaningful number of patients who are receiving maverick before along.
Paula Ragan: Without concomitant G CSF treatment, enabling the assessment of Maverick before as a potential monotherapy and those diagnosed with CN. We also expect that data to be presented will include information on additional participants being treated with the combination of maverick's for M. G CSF.
Paula Ragan: Importantly, all of the preliminary data presented to date and other learnings from the phase two trial, along with our interactions with the FDA have enabled us to finalize the protocol for a global pivotal phase III clinical trial of Maverick's foreign CN and advance the initiation of this important next true.
Paula Ragan: While in the first half of 2024.
Adam S. Mostafa: Our SG&A expenses included $1.4 and $4.3 million of certain non-cash expenses for the fourth quarter and full year ended December 31st. Finally, we reported a net loss of $19.1 and $101.2 million for the fourth quarter and full year ended December 31st, compared to $29.1 and $93.9 million for the comparable periods in 2022. Losses included $2.5 and $8.7 million of stock-based compensation expenses for the fourth quarter and full year 2023, respectively, compared to $1.1 and $5.2 million for the comparable periods in 2022.
Paula Ragan: As we've previously discussed we plan to enroll approximately 150 participants in the trial, which will be a 52 week double blind placebo controlled trial with one to one randomization.
Paula Ragan: We will be assessing the safety and efficacy of maverick's, four plus or minus kind of commitment G CSF treatment and those with congenital autoimmune or idiopathic neutropenia.
Paula Ragan: The patients included into the study will also have to present with neutropenia and symptomatic infections. Despite current standard of care, including G. CSF.
Paula Ragan: There is a significant unmet medical need across this established phase three patient population a U S market, we estimate to represent approximately 15000 people.
Paula Ragan: The primary endpoint will be a two component endpoint comprised of both the annualized infection rate and AMC responder analysis across the study population.
Paula Ragan: Secondary endpoints will include the severity and duration of infections antibiotic use quality of life measurements, among others and of course safety.
Paula Ragan: We are in the process of initiating our global study sites and are looking forward to enrolling patients across a number of leased sites beginning in the next few months.
Paula Ragan: With that, I'll pass it back to Paula. Thanks so much, Adam. To conclude, as we said in the press release earlier this morning, the excitement at X4 is now palpable. We have a countdown clock on the wall for our expected PODUSA date, and we could not be more energized to know that we are so close to potentially achieving our vision of delivering meaningful benefits to those with rare diseases of the immune system by gaining approval for what would be the first and only targeted therapy for the treatment of people with And we look forward to continuing to report on our other milestones throughout the rest of the year. We'll now open up the call to your questions. Operator?
Paula Ragan: We are as of now planning to host a CN events before the end of June to update on both the phase two trial data and CN phase III trial progress so stay tuned for more information on that.
Paula Ragan: I'll now turn it over to our CFO, Adam a staffer to review the fourth quarter and full year 2023 financials.
Paula Ragan: Adam.
Adam S. Mostafa: Thanks, Paul and thanks to all of you for being on the call with us today.
Adam S. Mostafa: Having raised more than $87 million during 2023 comprised of a mix of equity and debt capital to an at the market pipe and to our expanded loan facility with Hercules. We ended 2023 with $115 $2 million in cash and cash equivalents short term marketable securities.
Adam S. Mostafa: And restricted cash.
Adam S. Mostafa: We expect that these funds will support our operations into 2025.
Operator: Thank you. We will now conduct a question and answer session. If you would like to ask a question, please press star 1 on your telephone keypad. A confirmation tone will indicate your line is in the question queue.
Adam S. Mostafa: We would like to note that we may have multiple non dilutive sources of funding available to us throughout 2024.
Adam S. Mostafa: Given mavericks as far as rare pediatric disease designation, we are optimistic that we will receive a priority review voucher, which can be used for a subsequent application or sold to another drug sponsor shouldnt average or be approved if all.
Operator: You may press star 2 if you would like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys. Once again, that's star 1 to ask a question at this time. One moment while we poll for questions. Our first question comes from Kristen Kluska with Cancer Fitzgerald. Please proceed. Hi, everyone.
Adam S. Mostafa: All goes well we also may have additional access to milestone based debt tranches to our Hercules facility. This year and anticipate revenues from U S sales of Maverick supports over the course of this year and beyond.
Adam S. Mostafa: A quick recap of our 2023 financials.
Adam S. Mostafa: R&D expenses were 15 point street and $72 million for the fourth quarter and full year ended December 31 2023, respectively.
Kristen Brianne Kluska: Thanks for taking the questions and kudos. I've seen your team at a lot of these medical conferences, so great work getting the community aware of what's going on there. I wanted to ask about commercialization and thoughts around CN.
Adam S. Mostafa: Compared to 19% and $61 1 million for the comparable periods in 2022.
Kristen Brianne Kluska: So the KOLs that we speak to note that even meeting some GCSF would be a huge win, but they understand that in a commercial setting, patients are going to present differently, some on monotherapy maps, some on combination, and some on combinations that may be less GCSF. So to account for this, what is your expectation of what the monitoring and neutrophil measured protocols would look like in a commercial setting? Thanks, Kristen.
Adam S. Mostafa: Our R&D expenses included $1, one and $4 $4 million of certain noncash expenses for the fourth quarter and full year ended December 31, respectively.
Adam S. Mostafa: SG&A expenses were nine 9% and $35 5 million for the fourth quarter and full year of 2023, respectively.
Adam S. Mostafa: Compared to $6, six and $27 million for the comparable periods in 2022.
Paula Ragan: I think what we're hearing you explore is around neutrophil counts and chronic neutropenia and then how we are considering how best to develop the product to address the range of patients. So, assuming that I got that right, we're certainly very excited about our pending data update on the Phase 2 study coming up in the first half of this year. Of course, in our first three patients, we saw a nice durable elevation in patients with combination GCSF.
Adam S. Mostafa: Our SG&A expenses included $1, four and $4 $3 million of certain noncash expenses for the fourth quarter and full year ended December 31, respectively.
Adam S. Mostafa: Finally, we reported a net loss of $19 one in $101 2 million for the fourth quarter and full year ended December 31.
Adam S. Mostafa: Compared to 29, one and $93 9 million for the comparable periods in 2022.
Adam S. Mostafa: Net losses included $2, five and $8 7 million of stock based compensation expenses for the fourth quarter and full year 2023, respectively.
Adam S. Mostafa: Compared to $1, one and $5 2 million.
Adam S. Mostafa: For the comparable periods in 2022.
Paula Ragan: Importantly, we'll be having data on a group of monotherapy patients. So, I think that'll really help us appreciate the impact of Mavericks on our neutrophil counts in this broader patient population. And, of course, we'll continue to study the drug in combination with G. There's more work to do to support physicians and patients in choosing the best hybrid combination or introduction of MAV and then if and when GCSF might be needed. So, hopefully, I answered your question. Yes, thank you, and maybe I could squeeze in a second one: for Wim's Syndrome. You noted that sometimes physicians are thinking, hey, actually, maybe I do have a patient that fits the criteria for Wim's Syndrome. I'm curious if you're seeing any trends about what specifically it is about the indication that, you know, it's kind of putting this light bulb in their heads, or if you've heard Yeah, I mean, I think what the field has been sharing with us is just the general excitement because the data are so strong with an oral once daily. But I do think, you know, some of the interesting light bulbs are around, you know; there is no ICD-10 code.
Adam S. Mostafa: With that I'll pass it back to Paul.
Paul: Thanks, so much Adam Chinn.
Paul: To conclude as we said in the press release earlier this morning.
Paul: Statements at X for Us now palpable.
Countdown clock on the wall as are expected to produce a date and we cannot be more energized to know that we are so close to potentially achieving our vision of delivering meaningful benefits.
Paul: Rare diseases of the immune system by gaining approval for what would be the first and only targeted therapy for the treatment of people with whim syndrome.
Paul: We look forward to continuing to report out on our other milestones throughout the rest of the year I.
Speaker Change: We'll now open up the call for your questions operator.
Speaker Change: Thank you we will now conduct a question and answer session. If you would like to ask a question. Please press star one on your telephone keypad.
Speaker Change: Formation tone will indicate your line is in the question queue.
Speaker Change: You May press star two if people like to remove your question from the queue for participants using speaker equipment. It may be necessary to pick up your handset before pressing the star keys.
Speaker Change: Once again Thats star one to ask a question at this time, one moment, while we poll for questions.
Speaker Change: Our first question comes from Christopher <unk> with Cantor Fitzgerald. Please proceed.
Christopher: Hi, everyone. Thanks for taking the question then kudos the senior team at a lot of these medical conferences. So great work getting the community aware of what's going on there.
Mark Baldry: It's a very poorly educated disease, so I'll turn it over to our Chief Commercial Officer to add a bit more color. Yeah, thanks. And what's been interesting is through our disease awareness campaign, we've really seen the light bulb go on as we highlight the fact that these patients can present very differently; no one WIM patient is alike. And so this kind of dispels the whole assumption that you have to have all four symptoms to be a WIM patient. And physicians are beginning to realize they may have more WIM patients in their practice than they originally thought. Thanks very much. The next question comes from Ted Tenthoff with Piper Sandler.
Christopher: I wanted to ask about <unk>.
Christopher: Commercialization.
Speaker Change: Yeah, so the kols that we need to note that even leaving.
Speaker Change: That would be a huge win.
Speaker Change: Quinn, but understand that in a commercial setting patients are gonna presents differently Belmont monotherapy map somewhat.
Somewhat combination, but maybe less.
Speaker Change: So the cap or the what is your expectation on what the monetary net neutral measured protocol will look like in a commercial setting.
Quinn: Thanks, Kristen I think what we're hearing each explore is around neutrophil counts.
Kristen: Brian Neutropenia, and then how we are considering how best to develop the products to address the range of patients. So assuming that that I got that right. We're certainly very excited about our pending data.
Speaker Change: Data on the phase two study coming up at the first half of this year of course in our first three patients we saw a nice durable elevations in patients with combination G. CSF.
Edward Andrew Tenthoff: Please proceed. Great, thank you very much. I'm really getting excited for the launch here.
Edward Andrew Tenthoff: I have just a couple of questions. Firstly, on the registration issue, on the FDA, appreciating that there's not a panel, are there any final issues to resolve in terms of manufacturing visits, you know, have you initiated labeling discussions, you know, when do you think that happens, all those kinds of things. And then when it comes to the actual selling effort.
Speaker Change: Certainly we'll be having data on a group of monotherapy patients. So I think that'll really help us appreciate the impact of maverick's for a neutrophil counts in this broader patient population and then of course, we'll continue to study the drug in combination with G. Theres more work to do to support physicians and patients on the best hybrid combination our introduction of Mab.
Speaker Change: And then F N y G CSF might be needed. So hopefully I got your question.
Speaker Change: Yes, Thank you and maybe if I can squeeze in a second one for Wednesday.
Speaker Change: He noted that sometimes positions are thinking hey, actually maybe I do you have a patient that fit the criteria for Wednesday drug curious, if you're seeing any trends about what specifically it is about.
Christophe Arbet: How many reps do you anticipate needing to satisfy the U.S.? And, you know, what do you think the kind of cost would be, the annual cost for that sales force? Thanks so much. Sure, our Chief Medical Officer will take your regulatory question, and Marc can comment on the commercial. Sure, so our interactions with the FDA have been the typical interactions. We are on track for bidufit dates on April 30th, and we are working through the regular process with the FDA.
Speaker Change: The indication.
Speaker Change: Putting this lightbulb in their head or if you've heard any specific feedback there to help with your education efforts.
Speaker Change: Yes, I mean, I think what the field has been sharing with US is just the general excitement because the data are so strong with an oral once daily but I do think some of the interesting light bulbs or around there. There is no ICD 10 code as a very poorly educated disease. So I'll turn it over to our chief commercial officer to add a bit more color.
Speaker Change: Yes, thanks, and whats been interesting is through our disease awareness campaign, we've really seen that light bulb go on as we highlight the fact that these patients can present very differently.
Mark Baldry: Yeah, and assuming we get approved at the end of April, we'll be ready to deploy a fully trained, experienced rare disease field team. We can share more details around the time of approval in terms of the size of the sales force, but we'll also be engaging with payers to communicate the burden of WIM and the value proposition of Mavericks for, you know, and offering a comprehensive suite of patient support services. So patients can get access to Mavericks as quickly as possible after approval.
Speaker Change: No one when patient is alive and so this is kind of dispel the hall.
Speaker Change: Assumption that you have to have all four symptoms to be a whim patient physicians are beginning to realize they may have more when patients in their practice and they originally thought.
Speaker Change: Thanks very much.
Speaker Change: The next question comes from Ted <unk> with Piper Sandler. Please proceed.
Ted: Great. Thank you very much you really got me excited.
Ted: The launch here.
Ted: Just a couple of questions firstly on the Reds on the.
Ted: <unk>.
Ted: FCA.
Ted: Yes.
Ted: Scaling the.
Ted: Channel are there any.
Ted: No.
Ted: Issues to resolve in terms of.
Factory.
Ted:
Ted: Initiated labeling discussions.
Ted: When do you think that happens all of those kinds of things and then when it comes to the actual salary.
Edward Andrew Tenthoff: Okay, great. Thank you. Good luck. Thanks, Ted. Thank you. Our next question comes from Sean Lee with H.C. Wainwright.
Ted: The effort.
Ted: How many reps do you anticipate to satisfy the U S.
Sean Lee: Please proceed. Hey, good morning, this is Sean on behalf of RK. I just have a quick question in terms of the awareness campaign. So, because it's such a rare disease, once it's launched, would you be doing a marketing campaign targeting patients as well, just to have the outreach there too, so that they can start reaching out for number X4? Great question, of course. We encourage everyone to check the What If It's WHAM website that's available to the public.
Ted: And.
Ted: What do you think the cost would be annual cost for that sales force. Thanks, so much.
Oh sure I'm, our Chief Medical Officer will take care of a regulatory question and Mark can comment on the commercial.
Speaker Change: Our interactions with the FDA has been the typical interactions we are on track for two different dates on April 30th.
Speaker Change: And we're working through the regular process with the FDA.
Speaker Change: Yeah.
Mark Baldry: Yes, and then assuming we get approved at the end of April we'll be ready to deploy a fully trained.
Mark Baldry: <unk> rare disease field team.
Mark Baldry: We can share more details around the time of approval in terms of the size of the sales force, but we will be also engaging with payers to communicate the burden of women the value proposition of Maverick before.
Mark Baldry: And offering a comprehensive suite of patient support services. So.
Mark Baldry: Patients can get access to Maverix four as quickly as possible after approval.
Speaker Change: Okay, great. Thank you good luck.
Speaker Change: Thanks Ted.
Paula Ragan: So certainly we are developing the tools that would be directed towards physicians and of course support the patient groups appropriately. I'll turn it over to Marc for a bit more detail. Yeah, it's a great question and as Paula said, you know, we're really building the foundations for a launch that enables the product to get to patients as quickly as possible. You know, for clarity, we don't expect a bolus of patients at launch. Our goal is to get patients back in to see their doctors and as we build demand for our product. So we'll be doing marketing campaigns targeted at physicians where we expect to have, when patients in their practice, as well as marketing campaigns to potential patients and also educating payers on the burden of Wimp syndrome and the value proposition of Maverick support. Great.
Speaker Change: Thank you. Our next question comes from Sean Lee with H C. Wainwright. Please proceed.
Sean Lee: Hey, Good morning, this is Sean on for RK.
Sean Lee: Quick question <unk> awareness campaign, so because it's such a rare disease. Once its launched would you be doing a marketing campaign targeting patients with small just to outreach.
Sean Lee: Outreach to so that they can start reaching out for around <unk> four.
Speaker Change: I'm sure Great question of course, we encourage everyone to check whether if it's a whim a website that's available to the public.
Speaker Change: So certainly we are developing the tools that would be directed towards physicians and of course support the patient groups appropriately I'll turn it over to mark for a bit more detail.
Speaker Change: Yes, it's a great question and as Paul said.
Mark Baldry: We're really building the foundations for a launch that enables the product to get to patients as quickly as possible for clarity. We don't expect a bolus of patients at launch our goal is to get patients back in to see their doctors and and as we build demand for our products. So we will be doing marketing campaigns targeted at positions where we.
Mark Baldry: Expect to have.
Mark Baldry: When patients in their practice as well as.
Mark Baldry: Marketing campaigns to potential patients and also educating payers on the burden of whim syndrome, and the value proposition of Maverick support.
Speaker Change: Great and thank you for that.
Mark Baldry: Thank you. Once again, to ask a question, that's star 1 on your telephone keypad at this time. Our next question comes from Stephen Willey with Stifo. Please proceed. Yeah, good morning.
Speaker Change: Once again to ask a question Thats Star one on your telephone keypad at this time. Our next question comes from Stephen Willey with Stifel. Please proceed.
Stephen Douglas Willey: Yes. Good morning, Thanks for taking the questions maybe.
Stephen Douglas Willey: Thanks for taking the questions. Maybe just a couple of phase three CN questions. So, I think you are stratifying on CN subtype in phase 3, but I guess I'm just wondering how you expect those subtypes to be represented within phase 3 given patient eligibility criteria, and if you're enrolling in a way to get kind of a minimal representation of each of these, whether it's congenital, autoimmune, or Idiopath. So, yes, this is Christophe here.
Stephen Douglas Willey: Maybe just a couple fees three CN question so.
Stephen Douglas Willey: Thank you our stratify on CN subtype in the phase III, but I guess I'm just.
Stephen Douglas Willey: So I'm just wondering how you expect those subtypes to be represented within the phase III given patient eligibility criteria.
And if youre enrolling in a way to get kind of a minimal representation of each of these whether it's congenital autoimmune or idiopathic.
Stephen Douglas Willey: So yes. This is christoph here.
Christophe Arbet: The included criteria will include congenital, idiopathic, and autoimmune patients. In the included study, we will have patients on monotherapy and on GCSF as well. We're not planning on stratifying specifically for some of those subtypes, but we'll do additional analysis, obviously, when we have given the sample size. We'll have a substantial number of those subtypes that we'll be able to analyze.
Christoph Ingalls: The inclusion criteria will have congenital idiopathic or Tony and patience.
Christoph Ingalls: Two the study we will have the patients on monotherapy and on G CSF as well.
Christoph Ingalls: Not planning on stratified specifically for some of those subtypes, but we will do additional analysis, obviously when we.
Christoph Ingalls: Given the sample size will have a substantial number of those subtypes that we'll be able to consequently.
Paula Ragan: Yeah, and just to add to Christophe's comment, you know, idiopathic and autoimmune are essentially the same bucket. They're different words for the same group of patients, and that's quite a large majority. And then there are the congenitals, so we'll be able to stratify by those bigger buckets. The congenitals are so variable, so we're not able to stratify based on the heterogeneity of congenital defects.
Christoph Ingalls: Annualized yeah.
Speaker Change: Yeah, and just to just to add to Christophe comment.
Speaker Change: The idiopathic autoimmune are essentially the same bucket. They are different words for the same group of patients and that's quite a large majority and then there's the congenital so we'll be able to to stratify by those bigger buckets.
Speaker Change: Congenital are so variable that's really were not able to stratify based on the heterogeneity of congested also restaurant across the big buckets that Christophe mentioned and also the G CSF and monotherapy.
Christophe Arbet: So we're stratifying across the big buckets that Christophe mentioned and also GCSF and monotherapy. Okay, and then is there a lower limit of neutrophils that a patient must have to be phase three eligible? And then I guess just given the inherent variability of neutrophil counts, how many measurements do you require during the screening process prior to randomization? So yes, we do have the, the, the.
Speaker Change: Okay.
Speaker Change: And then.
Speaker Change: Is there a lower limit of neutrophils that a patient must have to be phase III eligible and then I guess just given the.
Speaker Change: Inherent variability neutrophil counts, how many measurements D required during the screening process prior to randomization.
Speaker Change: So yes, we do have we do have the.
Speaker Change: The.
Christophe Arbet: So the measurements first, let me start with this; we have several measurements that we're going to be looking at for the screening; we're looking at one measurement at the screening, and then we will establish the baseline over several hours of measurements. And the first part of the questions was if there was a lower limit of ANC; we don't have a lower limit of ANC, there will be some patients that can be fairly low, especially in the con And Steve, just as a reminder benchmark, our WIM Phase 3, their baseline was about 180 cells per microliter, so quite severely neutropenic, so we would expect to be able to accommodate patients certainly well into those ranges and hopefully to see an effect similar to what we've seen in WIM. Okay. Very helpful. The next question comes from Carpet Patel with Bee Riley. Please proceed. Hi, good morning. This is Jie Ong for CALPIT.
Speaker Change: So the measurements first.
Speaker Change: With this we have several measurements that we're going to be looking at for the screening. We're looking at one measure one measurement on the screening and then we will establish the baseline or several.
Speaker Change: Our soft measurements.
Speaker Change: And the first part of your question was.
Speaker Change: And if there is a lower limit and see we don't have a loan limits of AMC.
Speaker Change: There will be there will be some patients that can be fairly low, especially in the congenital population.
Speaker Change: And we have a range.
Speaker Change: Our inclusion criteria that include the CVA or all the way to the myelin Monterrey neutropenia patients.
Speaker Change: And Steve just as a reminder, but just as a reminder, our benchmark our women phase III, either baseline or about 180 <unk> cells per microliter, so quite severely neutropenia. So we would expect to be able to accommodate patients certainly well into those ranges and hopefully to see an effect similar to what we've seen in women.
Steve: Okay very helpful. Thanks for taking the questions.
Speaker Change: Okay.
Speaker Change: The next question comes from Paul <unk> with B Riley. Please proceed.
Speaker Change: Hi, Good morning. This is Jeff on for Cal Pete Thanks for taking the questions. My first question is for the upcoming two update.
Carpet Patel: Thanks for taking the question. My first question is about the upcoming Phase 2 update, where we see data from patients who are on higher-dose GCSF. I believe the data seen to date were for patients less than 1 microgram per kilogram, but the approved dose of GCSF is much higher. And my second question is about the Phase 3 that you are planning to start. What, in your view, is a good delta for reduction in infection rates versus the control arm?
Jeff: Well, we see data from.
Jeff: Patients who are Ohio, those now G CSF.
Jeff: The data <unk> seen to date well for patients less than one microgram per pupil by the approved dose of G. CSF is much higher.
Speaker Change: And my second question either for the Phase III that you are planning to start.
Speaker Change: What in your view is a good delta for reduction in infection.
Speaker Change: Infection rate versus the control arm. Thank you.
Carpet Patel: Thank you. So we'll tag team here. I mean, in terms of GCSF dosing, you're incredibly sharp at assessing that the average dose of GCSF for the patient is much lower than the labeled dose, and that is practice. Unfortunately, this drug is so challenging to take and to manage that physicians and patients stay on the very low end, sometimes as low as 20% of the labeled dose.
Speaker Change: Yeah.
Speaker Change: So we'll tag team here I mean, so in terms of the G CSF dosing you're incredibly.
Speaker Change: Sharp and assessing that the average dose in G. CSF up the patients is much lower than the label dose.
Speaker Change: And that is practice. Unfortunately, this drug is so challenging to peak and to manage that physicians and patients stay on the very low end, sometimes as low as 20% of the labeled dose and.
Paula Ragan: And again, we don't give guidance on what doses patients are on; they come in at their own stable doses. So we would expect, if that's the standard of care, that that's the range that we'll continue to see in the trial. So I hope that addresses your first question, and then I think your second question was around what type of infection benefit target are we aiming for in phase 3? So in our phase 3, I want to remind you about our WIM data. Our WIM data on average have seen an improvement of 60% of infections, and we've taken a rather conservative approach, and we're estimating, with part of that study, a 40% difference in annualized infection rates.
Speaker Change: And again, we don't give guidance on <unk>.
Speaker Change: This is patients are on they come in and they are unstable doses. So we would expect that that's the standard of care, but that's the range will continue to see in the trial.
Speaker Change: So I hope that addresses your first question and then I think your second question was around.
Speaker Change: What type of infection benefit target or we are aiming for in the phase.
Speaker Change: Our phase III I want to remind you about Halloween data when data on average have seen an improvement of 60% of infections and we've taken a rather conservative approach and we're estimating.
Speaker Change: We've powered the study for a 40% difference.
Speaker Change: Annualized infection rates.
Christophe Arbet: Okay, thank you. That will help. Thank you. At this time, I would like to turn the floor back over to Paula Ragan for closing comments. Thank you so much for joining us today. We appreciate all your attention and insightful questions and wish you an enjoyable rest of your day. Thank you. This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation, and have a great day. BF-WATCH TV 2021, The Bulletproof Executive 2013 BF-WATCH TV 2021, The Bulletproof Executive 2013, BF-WATCH TV 2021
Okay. Thank you that's very helpful.
Speaker Change: Thank you at this time I would like to turn the floor back over to Paula Ragan for closing comments.
Paula Ragan: Oh. Thank you so much for joining us today, we appreciate all the attention and insightful questions and wish you an enjoyable rest of your day.
Speaker Change: Thank you. This concludes today's teleconference. You may disconnect. Your lines at this time. Thank you for your participation and have a great day.
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