Q3 2020 X4 Pharmaceuticals Inc Earnings Call

[music].

Greetings and welcome to the export Pharmaceuticals third quarter financial and operating results conference call.

Operator: Greetings and welcome to X4 Pharmaceuticals' 3rd Quarter Financial and Operating Results Conference Call. At this time, all participants are in a listen-only mode.

This time, all participants I know listen only mode.

Operator: A question and answer session will follow the formal presentation. As a reminder, this conference call is being recorded. It is now my pleasure to introduce your host, Candice Ellis, Director of Corporate Communications and Investor Relations at X4. You may begin. Thank you, Operator, and good morning, everyone. Thanks so much for joining us today.

Question and answer session will follow the formal presentation. As a reminder, this conference call is being recorded it.

It is now my pleasure to introduce your host Candace, Let's director of corporate Communications and Investor Relations at export you may begin.

Thank you operator, and good morning, everyone. Thanks, so much for joining us today presenting on today's call will be our Chief Executive Officer, Dr., Paul Reagan and our Chief Financial Officer, Adam Mustapha.

Candice Ellis: Presenting on today's call will be our Chief Executive Officer, Dr. Paula Ragan, and our Chief Financial Officer, Adam Mostafa. Following prepared remarks by each, we will open the call to your questions, and they will be joined by our Senior Vice President of Technical Operations and Quality, Dr. Mary DeVias. As a reminder, on today's call, we'll be making forward-looking statements regarding our regulatory and product development plans, as well as our research activities. These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted.

Following prepared remarks, they each we'll open the call to your questions and they will be joined fair senior Vice President of technical operations and quality Dr. married TV.

As a reminder, on today's call, making forward looking statements regarding our regulatory and product development plans as well as our research activities. These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted descriptions of these risks can be found in our most recent form 10-K on file with the FCC and our fourth.

Candice Ellis: The descriptions of these risks can be found in our most recent Form 10-K, on file with the SEC, and our forthcoming Form 10-Q. I'd now like to turn the call over to our CEO, Paula Ragan. Thanks, Candice, and thank you everyone for joining us on the call this morning. We hope you are all continuing to stay safe and healthy. We are pleased to report today that while the operating environment remains challenging due to the ongoing COVID-19 pandemic, we continue to advance our Mavericks IV clinical development program. Let me begin with a review of our most recent accomplishments. We are pleased to announce the publication of our positive Phase II Safety and Efficacy Data for Mavarixifor in Wim Syndrome in the prestigious journal Blood, which we believe further recognizes the significant potential of our lead candidate in this patient population. As a reminder, Wimp syndrome is a rare inherited primary immunodeficiency disease caused by mutations in the chemokine receptor CXCR4, a receptor that plays a key role in enabling the healthy trafficking of immune cells and effective immunosurveillance.

Thanks.

Form 10-Q, I'd now like you're trying to calibrate where CEO Paulo Reagan.

Thanks, Candice and thank you everyone for joining us on the call. This morning, we hope you are all continuing to stay safe and healthy.

We are pleased to report today that while the operating environment remains challenging due to the ongoing COVID-19 pandemic, we continued to advance our matter it for clinical development program.

Let me begin with a review of our most recent accomplishments. We are pleased to announce the publication of our positive phase two safety and efficacy data for Mavericks before in a wimp syndrome in the prestigious journal blood, which we believe further recognizes the significant potential of our lead candidate in this patient population.

As a reminder, when syndrome is a rare inherited primary immunodeficiency disease caused by mutations and the chemo kinda receptor CXC are for a receptor that plays a key role in enabling to healthy trafficking of immune cells and effective you know surveillance.

Paula Ragan: Mavirixa4 is our first-in-class small-molecule antagonist of the chemokine receptor CXCR4 and is being developed as a once-daily oral therapy. While the comprehensive data published in BLOOD expands on previously presented data, there were new results presented, including patient-level data regarding the specific effects on neutrophils, lymphocytes, and monocytes, as well as the effect of increasing doses of Mavrex In addition, the manuscript provided the most up-to-date long-term safety and pharmacokinetic data and presented a detailed analysis of the clinical benefit of the Extended Maverick Support Therapy on infection rates and wart burden.

Maverick before it is our first in class small molecule antagonist or the chemo kinda receptor CXC. Our four is being developed as a once daily oral therapy.

While the comprehensive data published in blood expands on previously presented data there were new results presented including patient level data regarding the specific effects on neutrophil lymphocytes and monocytes as well as the effect of increasing doses of Maverick before on total white blood cell counts.

In addition, the manuscript provided the most up to date long term safety and pharmacokinetic data and presented a detailed analysis of the clinical benefit of the extended Mavericks for a therapy on infection rates and work burden.

This important publication and the official journal of the American Society of Hematology provides key third party validation of the data supporting our clinical strategy, including the selection of the dose primary biomarker endpoints and secondary clinical endpoints for ongoing pivotal phase three clinical trial the.

Paula Ragan: This important publication in the official journal of the American Society of Hematology provides key third-party validation of the data supporting our clinical strategy, including the selection of the dose, the primary biomarker endpoint, and the secondary clinical endpoint for our ongoing pivotal Phase III clinical trial. The published results continue to reinforce our belief that by down-regulating the CXCR4 and CXCL12 signaling pathways, Mavrix4 has the potential to be the first disease-modifying therapy for the more than 3,500 estimated diagnosed and undiagnosed WIMP patients in the U.S. We are also thrilled to have been granted Fast-Track designation by the FDA for Mavericks 4 and Wim Syndrome. Through the Fast-Track program, X4 is eligible for more frequent meetings with the FDA to discuss the drug's development plan, protocols, and clinical data that would support Maverick's 4's potential approval for WHIM. This key regulatory achievement further recognizes the significant unmet need for Wim's syndrome and Maverick's potential to treat this challenging disease. As a reminder, Maverick Sephora was previously granted breakthrough therapy designation by the FDA as well as orphan drug status by the FDA and the European Commission for the treatment of Limb Syndrome.

Published results continue to reinforce our belief that fight down regulating the CX Air Force. The Axiall 12 signaling pathway Mavericks four has the potential to be the first disease modifying therapy for the more than 3500 estimated diagnosed and undiagnosed when patients and the U.S.

We're also thrilled to be granted fast track designation by the FDA for Mavericks for it and when syndrome.

Through the fast track program Xplore is eligible for more frequent meetings with the FDA to discuss the drug development plan protocols and clinical data that would support Mavericks for its potential approval for Wim. This.

This key regulatory achievements further recognizes the significant unmet need of wind syndrome, and maverick for its potential to treat this challenging disease.

As a reminder, maverick before it was previously granted breakthrough therapy designation by the FDA as well as orphan drug status by the FDA and the European Commission for the treatment of women syndrome.

Let me now provide an update on our ongoing clinical development programs for Maverick before.

Importantly, despite continued uncertainties surrounding COVID-19, we remain focused on advancing our clinical development program.

We continue to enroll patients in our phase three whim syndrome trial and make good progress across the various regulatory and clinical aspects of the trial.

We are diversified across numerous sites in countries around the world each of which has its own regional and state level COVID-19 considerations.

We are working with the sites to maintain enrollment momentum in the trial and including additional services such as in home patient visits to mitigate the impact of COVID-19.

We continue to anticipate topline phase three data in women syndrome, and 2022 and intend to provide further clarity on the timeline as soon as we are able to do so.

Our phase one b trial and severe congenital neutropenia also continues to make progress and we anticipate initial data from this 14 day proof of concept study in 2021 summer.

Paula Ragan: Let me now provide an update on our ongoing clinical development programs for Maverix IV. Importantly, despite continued uncertainty surrounding COVID-19, we remain focused on advancing our clinical development programs. We continue to enroll patients in our Phase III Wim Syndrome trial and make good progress across the various regulatory and clinical aspects of the trial. We are diversified across numerous sites and countries around the world, each of which has its own regional and site-level COVID-19 considerations.

Similar to the phase three women syndrome trial, we intend to provide further clarity around our SDN trial timeline as soon as we are able to do so.

In Waldenstrms Macroglobulinemia or W. M. A rare form of lymphoma, we're continuing to enroll patients. Although COVID-19 related delays have had some impact as we recently disclosed big.

We expect the availability of the initial phase one b clinical results in the first half of next year, a slight delay from our previous guidance of the second half of this year.

In order to mitigate COVID-19 related piece in travel concerns in this study we are focused on implementing home health business.

We are in regular dialogue with our investigators and through our patient advocacy team with patients to understand their needs given the extended challenges of the cobot pandemic.

Paula Ragan: We are working with the sites to maintain enrollment momentum in the trial and include additional services such as in-home patient visits to mitigate the impact of COVID-19. We continue to anticipate top-line Phase III data in Wimp Syndrome in 2022 and intend to provide further clarity on the timeline as soon as we are able to do so. Our Phase 1b trial in severe congenital neutropenia also continues to make progress, and we anticipate initial data from this 14-day proof-of-concept study in 2021. Similar to the Phase III Wimps Syndrome trial, we intend to provide further clarity around our SCN trial timeline as soon as we are able to do so. In Waldenstrom's macroglobulinemia, or WM, a rare form of lymphoma, we are continuing to enroll patients although COVID-19-related delays have had some impact, as we recently disclosed. We expect the availability of the initial Phase 1b clinical results in the first half of next year, a slight delay from our previous guidance of the second half of this year. In order to mitigate COVID-19-related patient travel concerns in the study, we are focused on implementing home health visits.

We're confident that we have an effective plan in place to appropriately addressed the impact the COVID-19 on R.W.M. study.

As a reminder, this phase oneb clinical trial is expected to enroll between 12 and 18 patients with W.M. and is a multicenter open label dose escalation clinical trial assessing the safety and Tolerability of Maverick sport in combination with Ibrutinib in the.

The trial being conducted as part of a collaboration with the leukemia and lymphoma society to accelerate the development of Maverick score for the treatment of Waldenstrom.

The results of this study will share safety and dose escalation data and important efficacy signals and we expect that these data will inform future FDA discussions regarding a potential registration trial in W. M.

Paula Ragan: We are in regular dialogue with our investigators and our patient advocacy team with patients to understand their needs given the extended challenges of the COVID pandemic. We are confident that we have an effective plan in place to appropriately address the impact of COVID-19 on our WM study. As a reminder, this Phase 1b clinical trial is expected to enroll between 12 and 18 patients with WM and is a multi-center, open-label, dose-escalation clinical trial assessing the safety and tolerability of Maverix IV in combination with Abrutinib. The trial is being conducted as part of a collaboration with the Leukemia and Lymphoma Society to accelerate the development of Maverix IV for the treatment of Waldenstrom's.

The results for the quarter Adam.

Thank you Paula and thanks to all of you on the call today.

As presented in our press release. This morning, I will summarize our financial activities and results for the third quarter of 2020.

As of September Thirtyth, 2020, Xplore had $90.7 million in cash cash equivalents and restricted cash.

We continue to expect that our cash and cash equivalents will fund our operations into early 2022.

Paula Ragan: The results of this study will share safety and dis-isolation data and important efficacy signals, and we expect that these data will inform future FDA discussions regarding a potential registration trial in WM. Lastly, I would like to welcome two new additions to the X4 leadership team. Dr. Art Timberis started earlier this week as our new Chief Scientific Officer.

Paula Ragan: Art is an experienced CSO who is joining us from Comet Therapeutics, a privately held company focused on coenzyme A science and intermediate metabolism, where he also served as CSO. Prior to his role at Comet, he founded and was Chief Scientific Officer of Transform Therapeutics, where he led the discovery of a novel next-generation CXCR2 antagonist for the treatment of cancer. In addition, Art held key leadership roles in drug discovery at Biogen and at Shearing Plough earlier in his career.

And press the pound key please stand by while we compare the candy Ross there.

Our first question comes from Stephen Willey with Stifel. Your line is now open.

Hi, all this is allen on for Steve.

Just two quick questions for me. So servers, you mentioned that you're focused on implementing home health visits for patients enrolled in the Waldenstrms trial in order to mitigate some of those effects from cobot is that same strategy being applied to the women essien trials as well and if so how how is that doing thanks.

Paula Ragan: With his significant expertise in chemokine-related chemistry, decades of drug discovery experience, and inspiring leadership, Art is an ideal fit to lead our R&D initiatives and further foster our evolution towards becoming a global rare disease company. In addition, we recently expanded our board of directors with the appointment of Allison Lawton, an industry veteran who strengthens our board's expertise across many strategic fronts. Ms. Lawton most recently served as CEO of Kaleido Biosciences, and having previously served as a Consulting Chief Operating Officer to the company and as a member of X4's Corporate Advisory Board, Allison brings a unique understanding of X4's core scientific and corporate goals. I look forward to working closely with her again. With that, I'll now turn the call over to Adam to discuss our financial results for the quarter. Adam?

Adam S. Mostafa: Thank you, Paula, and thanks to all of you on the call today. As presented in our press release this morning, I will summarize our financial activities and results for the third quarter of 2020. As of September 30, 2020, X4 had $90.7 million in cash, cash equivalents, and restricted cash.

Absent profiles again, although minor it certainly makes an important step forward for patients and clinicians to not have to worry about that so thats moving forward very nicely.

Adam S. Mostafa: We continue to expect that our cash and cash equivalents will fund our operations into early 2022. Note that this guidance does not include the $25 million in potential and contingent proceeds from our Hercules Debt Facility or any cash exercise proceeds from investors holding our outstanding warrants. Research and development expenses were $11.4 million for the third quarter of 2020, as compared to $8.6 million for the comparable period in 2019. R&D expenses included $1,000,000 of certain non-cash expenses for the quarter ended September 30th, 2020. General and administrative expenses were $5.6 million for the third quarter of 2020 as compared to $4.4 million for the comparable period in 2019. G&A expenses also included $1.4 million of certain non-cash expenses for the quarter ended September 30, 2020. Finally, our net loss was $17.4 million in the third quarter of 2020, as compared to a net loss of $17.7 million for the comparable period in 2019. This net loss included $2.5 million of certain non-cash expenses for the quarter ended September 30, 2020.

Question comes from Joel Beauty with city NFL open.

Hi, guys. This is shawna for Joel. Thank you for taking my questions. Two for me today first on Walden <unk> for your data and and perfect next year, maybe you can talk about how how you plan to roll out that data and then kind of getting into the we'd done maybe what the results could be a side can seeing any complete responses are there any biomarker that would.

Give you confidence that any efficacy pickles V C. R. Due to your drug in Uhm and not the combo and then a follow up on on wins and maybe you could you talk about patient identification uhm, where you're kind of at right there and how many patients are on your EAP program right now.

Sure. So thanks nice to hear from you. So I'll try it out kind of walk through your questions. So in terms of the <unk> date of that format mm mm presenting the data. It's an open label trial for making nice advances and uncle up to present, a robust dataset that people can interpret and certainly when.

Operator: With that, let's open up the call for questions. Operator? Thank you. As a reminder, to ask a question, you will need to press star 1 on your telephone. To withdraw your question, press the pound key.

To do that in conjunction with our support of the key opinion leaders that are working with on our trial. So we're not yet sure of the format, but at the end of the day, while all of this will be a line to make sure we can communicate and put the data and contact them heal morale treatment landscape. So stay tuned for more specific that fanfold uhm with respect to the results. So it is.

Unknown Attendee: Please stand by while we compile the Q&A roster. Our first question comes from Stephen Willey with Stiefel. Your line is now open. Hi, all. This is Ellen. I'm for Steve.

Important to help the world appreciate so with a Briton I'm alone, there's actually a very well defined profile of responses and the first three to six months Uhm I'm single agents treatment and these patients with the C. C. R for mutation in fact, only about a third of them get something called a major response, which means I.

Unknown Attendee: Just two quick questions for me. First, you mentioned that you're focused on implementing home health visits for patients enrolled in the Wallenstrom's trial in order to mitigate some of those effects from COVID. Is that same strategy being applied to the WIM and SCN trials as well? And if so, how is that going?

<unk>.

Greater than 50 per cent of their baseline. So it's a pretty unfortunately sad bar for these patients that many of them are getting those profound and deep IGN responsive. So we'll be showing data from patients on the first three to six months ago, saying.

Paula Ragan: Thanks. Sure. Thanks, Ellen, for the question. So, yes, home health is being implemented in our studies where it makes sense for the chronic doses, which are both WIM and Waldenstroms. Obviously, these patients are coming in for many months. For SDN, it makes less sense given the actual period of the study is only two weeks of treatment. So, the home health is focused solely on WIM and Waldenstroms.

And certainly we hope to beat that number of 30% with the data that we present the first half of next year, but stay tuned the results as they unfold.

And then finally I think you mentioned wham patient I D. So we are continuing so with respect to the trial again I think we feel very confident that patients are there and waiting there's obviously been some issues with having sites being open and able to enroll due to covid, but with with respect to the trial or incredibly confident with respect.

Unknown Attendee: Okay, that makes sense. And then, can you provide any updates regarding X4P003 or X4P002? Are either of these assets in IND-enabling studies at this point? And then also, can you just remind us how those two assets are differentiated from each other?

Paula Ragan: I know 002 is engineered to cross the blood-brain barrier, but I was just wondering if you could give a little more color on 003. Thank you. Great. No, thank you. We're happy to talk about our preclinical programs that are going really well. So, 003 is the most advanced of the two.

Unknown Attendee: It is approaching IND-enabling studies, and then typically after that, it's about a year to get into the clinics. So, we're extremely excited with the profile that we've been seeing come together with this particular molecule, optimization. And again, it's making good progress in terms of finding that right profile between brain exposure and plasma exposure. And we're really excited to continue to advance that to support further development for some of those serious brain cancers that we hope to address. Okay, great.

It's primarily you asked the two akcea sites as you can appreciate with co. There there are ebbing and flowing travel restrictions for patient depending on where they're located and also on their own comfort level, which is why the in home health has been you know a good direction to alleviate that why we feel confident about that and now enabling enrolling to be a little bit more consistent.

In terms of the data again, we just have to wait and see it's an open label trial at 12 to 18 patients partly it will be dependent upon dose and safety and then as we see efficacy signals around the IDN level drops we'll look forward to sharing that in the first half of next year.

Unknown Attendee: Thank you. Thank you. Thank you. Our next question comes from Marlinda Lee with Canaccord. Your line is now open.

Great. That's helpful and maybe just a brief follow up as it relates to the SDN program can you just remind US is this still a single site in Washington, I believe on clinical trials Dot Gov and then.

Unknown Attendee: Hi guys, I was hoping that you could provide an update on the enrollment for Wim's Syndrome and then I also was curious about the preclinical programs. Might we be expecting any data presentations on those anytime in the near future? Thank you.

Given the immunocompromised nature of the patients.

Do you plan to sort of manage enrollment concerns.

Paula Ragan: So, just with respect to enrollment in WHIM, as consistent with the past, we're not providing any specifics other than to say that we're on track with our top-line data in 2022, and we are seeing patients continue to enroll in the trial even despite the global pandemic. So, we're really happy to see the patient's commitment and the site's commitment to continue to advance the trial. With respect to the preclinical program data, that's a great question, and it kind of segues very nicely to X4's recent announcement this week of hiring our Chief Scientific Officer, Art Tavares. We're very pleased to have someone with his tremendous experience in drug development and drug discovery. So, I think, you know, while we don't have any specifics on rolling data out around our preclinical program, stay tuned because I think we'll have some updates coming into next year. Great, thank you.

Okay and.

What can we way through from the wind syndrome study.

In terms of the optimal dose level.

For that <unk> <unk>.

Sure Great question, So uhm.

Uhm, let's just start with that current faith Street dose and Wham, which is 400 milligrams once per day and that dry cause I've been treated as a single agent therapy for Wham, how we've crossed back into Walden strums as we're starting at 200 milligrams per day. The rationale behind that is that we can do you need to consider the potential even.

Unknown Attendee: Thank you. Our next question comes from Joel Beattie with Citi. Your line is now open. Hi guys, this is Sean on for JUUL.

Unknown Attendee: Thank you for taking my questions. There are two for me today. First, on Waldenström's clear data in the first half of next year. Maybe just talk about how you plan to roll out that data. And then kind of getting into the weeds on maybe what the results could be. Aside from seeing any complete responses, are there any biomarkers that would give you confidence that any efficacy signals we see are due to your drug and not the combo?

Though it's slow drug drug interactions between Mavericks before and Ibrutinib to make sure that the exposure of those two drugs are not impacted in any direction by co administering them. So.

Unknown Attendee: And then a follow-up on WIMS. And maybe you could talk about patient identification, where you're kind of at right now, and how many patients are on your EAP program right now? Sure. So, thanks, Sean. It's nice to hear from you.

Paula Ragan: So, I'll try to kind of walk through your questions. So, in terms of the Waldenstrom Theta, the format of presenting the data, it's an open-label trial. We're making nice advances, and our goal is to present a robust data set that people can interpret. We certainly want to do that in conjunction with our support of the key opinion leaders that we are working with on our trial. So, we're not yet sure of the format, but at the end of the day, all of us will be aligned to make sure we can communicate and put the data in context of the overall treatment landscape. So, stay tuned for more specifics as they unfold. With respect to the results, though, it is important to help the world appreciate them.

To kind of get to the evidence of where this drug makes the most sense in terms of line of treatment.

Currently in the U.S. and primarily in Europe. Ibrutinib is is more typically used in the second line. Although it is approved for other lines of treatment. So there's a little bit of what is the current market and clinicians how are they utilizing <unk> versus what will our data to deliver certainly the more robust datasets that to achieve with the combination will open clip.

Paula Ragan: So, with Abrutinib alone, there's actually a very well-defined profile of responses in the first three to six months on single-agent treatments in these patients with the CXCR4 mutation. In fact, only about a third of them get something called a major response, which means IgM drops greater than 50% of their baseline. So, it's a pretty, unfortunately, sad bar for these patients. Not many of them are getting those profound and deep IgM responses.

I wouldn't mind about how to use that combination. So I think there's opportunity and then obviously the data will then drive how clinicians will consider using the treatment for their patients.

Perfect and then just a quick one for Adam I'm wondering how the.

Paula Ragan: So, we'll be showing data from patients on the first three to six months of dosing, and certainly, we hope to beat that number of 30% with the data that we present in the first half of next year, but stay tuned for the results as they unfold. And then, finally, I think you mentioned WIM patient ID. So we are continuing.

Paula Ragan: So with respect to the trial, again, I think we feel very confident that patients are there and waiting. There have obviously been some issues with having sites being open and able to enroll due to COVID, but with respect to the trial, we're incredibly confident. With respect to just the overall market, obviously, this is a long-term commitment for the company.

Paula Ragan: Between our education and awareness and, I'm sure, our medical science liaison teams, both in the US and, starting in Europe, we actually feel like we've been making great progress in getting the broader community to engage. We've had some virtual booths at various conferences where patients have come to visit us. So we feel like the word is getting out, and we're beginning to get a nice ground swell of people thinking more broadly about WIM and their own patient populations and their clinics and also reaching the patient community directly. So we continue to make progress. We're excited about the long-term trajectory of the company in support of the hopeful commercial approval of Maverix IV for these patients. Thank you so much, Paul.

Brooklyn are called programs.

Sure Okay, well, thanks, so much and yes, you know, we we feel incredibly fortunate to have art joying, giving his strong drug discovery experience and biology overlap and to chemo kind world. So I think when we think about not only mavericks before but also are too preclinical programs. You know first you really need to you know intend to operate and hit our new.

Your turn milestone for example, I do think as a drug Mavericks for shows activity in Walmart, there's a tremendous amount of support for <unk> for more broadly and certain leukemias and lymphomas I think between arts experienced in the biology, and then complementing what we're learning in the clinic that will really help.

US unable to prioritize how we can either developed mavericks for and or possibly O O three to expand the total market that we could potentially you know explore and hopefully benefit patients with our drug.

Unknown Attendee: Thank you. Our next question comes from Mayank Montami with B. Riley Securities. Your line is now open. Hi. Good morning, team. This is Sahil Ansarlyanka.

Thanks pilot and then just a follow up here I'm not even sure you can answer this one that I think on the line I'll call. We had you noted that you weren't capping you know pieces that were please treated on non pretreat. It probably watching some study was just gonna try to go and see what you get but can you provide any cough.

Unknown Attendee: Congratulations on all the progress. Just a few questions from us this morning. First off, as it relates to the WaldenStroms program and now expecting data in the first half of next year, is this more sort of enrollment related more than anything else in terms of, you know, monitoring responses? And should we still expect data on that, you know, 12 to 18 patient range? Yeah, so if I understood your question in the beginning, it was a little bit of what's the shift, or did I get that right? So, how sorry. No, that's exactly right.

<unk>, if you're a little bit more appreciative patients or are you <unk> more naive patients.

No. We you know there there's no.

There's no profile, yet that were really I'm able to disclose will certainly do that when we share the data I do like to point back to literature with respect to the double mute and patience, regardless of what line of therapy. There there uhm driven dressed with they have this remaining unmet need so I do think the consistency uhm.

For the impact of having a six year for mutation is important I think that kind of Theresa nice homogeneous patient population for us to study and we'll look forward to sharing that data in the first half of next year.

Thank you.

Thank you I'm not showing any further questions at this time I would now like to turn the call back over to Paula vegan for closing remarks.

Paula Ragan: Mm-hmm. Yeah, so that it's definitely just enrollment issues due to Again, the US site is primarily US with two ex-US sites, as you can appreciate with COVID. There are ebbing and flowing travel restrictions for patients depending on where they're located and also on their own comfort level, which is why the in-home health has been a good direction to alleviate that and why we feel confident about and that and now enabling enrollment to be a little We just have to wait and see. It's an open-label trial. It's 12 to 18 patients. Partly it will be dependent upon dose and safety, and then as we see efficacy signals around the IGM level, we'll look forward to sharing that in the first half of next year. Great, that's helpful.

Thank you so much well. Thank you again today for joining us we sincerely appreciate your interest in X four and look forward to continuing to provide updates on our progress as we approach multiple key catalyst in our business.

Have any further questions. Please don't hesitate to reach out. Thank you again and enjoy the rest of your day.

Ladies and gentlemen, this concludes today's conference call. Thank you for participating you may now disconnect.

[music].

Paula Ragan: And maybe just a brief follow-up as it relates to the SDN program. Can you just remind us, is this still just a single site in Washington, DC, on clinicaltrials.gov? And then, you know, given the immunocompromised nature of the patients, how do you plan to sort of manage enrollment concerns? You know, acknowledging that 14 days is that short treatment window and maybe at-home visits don't make as much sense here? Yeah, so I think I've been pretty consistent since the start of COVID.

Paula Ragan: The SDN trial has always been our trickiest because it is a translational research study. So, from the patient lens perspective, it's something that they need to weigh risk benefits along with their clinicians on whether or not enrollment makes sense. The University of Washington is a site that's open.

Paula Ragan: We do have other sites that are open as well, but I think there's always a delay on clintrials.gov in terms of that update. But at the end of the day, this is probably our trickiest study to try to balance respect and appreciation for the potential risk for these patients due to, or added risk due to, COVID, along with the investigators' enthusiasm for exploring this mechanism in these patients with this type of need. So, again, stay tuned. It's a 2021 story, and we'll look forward to just sort of sharpening that as we learn more.

Unknown Attendee: Great, that's really helpful. Thanks for taking our questions and congratulations on the progress. Thank you. Thank you. Our next question comes from Arthur Hugh with HC Wainwright. Your line is now open. Hey, good morning, everyone. This is Arthur from RK.

[music].

Unknown Attendee: So, I just have a quick question or a follow-up on the WM study. So, could you guys remind us of the dose levels across the patient, and what can we learn from the Wim's syndrome study in terms of the optimal dose level? All right, great. Sure. Great question.

Paula Ragan: So, let's just start with the current phase 3 dose in WIM, which is 400 milligrams once per day. And that drug is obviously being treated as a single agent therapy for WIM. How we've crossed back into Waldenstrom is we're starting at 200 milligrams per day. The rationale behind that is that we do need to consider the potential, even though it's low, drug-drug interactions between Maverick X4 and Ibrutinib to make sure that the exposure of those two drugs is not impacted in any direction by co-administering them. So, hence, we started at a 200-milligram once-per-day dose level, and we're confident that that's already a highly active level based even on what we saw in WIM at 200 milligrams per day in healthy volunteers. You see a very nice, robust bump in white blood cell mobilization, and also the trough levels from known TK are certainly in the highly active range.

Paula Ragan: So, we feel good about where we're starting at in terms of being able to see clinical activity. After that starting dose, you just want to keep pushing the dose until you get the classic kind of maximum dose per patient. So, the dose escalation levels are then 400 and 600 after each patient clears the 200-milligram dose. Does that answer your question?

Unknown Attendee: Yes, thank you very much for that. Thanks, Arthur. Thank you. Our next question comes from Laura Christensen of Cowan. Your line is now open. Hi. Good morning, guys.

Unknown Attendee: Thanks for taking my question. Regarding Waldenström's, I was wondering if you could just talk a little bit more about how you expect Maverix IV might fit into the treatment paradigm if it's approved. For example, what is the potential for Maverix IV to be used for spine in combination with Ibrutinib and CXCR4-positive MIT88 patients?

Paula Ragan: Sure, so our current study is exploring patients that are either treatment-naive or have up to three prior lines of treatment. So we will start to, at least in a small handful of patients, see what this drug potentially could do as a combination, I should say, as a frontline approach. So, based on that data, I do think that we will begin to kind of gather evidence of where this drug makes the most sense in terms of line of treatment. Currently, in the U.S. and primarily in Europe, Abrutinib is more typically used in the second line, although it is approved for other lines of treatment.

Paula Ragan: So, there's a little bit of what the current market and clinicians are doing with Abrutinib versus what our data will deliver. Certainly, the more robust data sets that are achieved with the combination will open clinicians' minds about how to use that combination. So, I think there's an opportunity, and obviously, the data will then drive how clinicians will consider using the treatment for their patients. And then just a quick one for Adam.

Adam S. Mostafa: I'm wondering how the stock purchase agreement that you recently put in place might impact your expected cash runway. Yeah, thanks, Laura. So, right now, I think our primary objective is to continue to track towards key milestones like Wallenström's in the first half and think about opportunistic ways to extend the runway based on that progress. The Stock Bricks Agreement, I think, is a helpful tool in our financial tool belt to potentially, [inaudible] on the Goldstein on Gelt radio show. He is the author of the Goldstein on Gelt radio show and is a licensed financial professional. His book Building Wealth in Israel is available in bookstores, on the web, or can be ordered. That's helpful.

Unknown Attendee: Thank you. As a reminder, to ask a question, you will need to press star 1 on your telephone. Our next question comes from Zeg Bragella with Roth Capital. Your line is now open. Good morning, guys.

Unknown Attendee: Thanks for the updates. I think I'm probably just going to ask one again about the preclinical pipeline with Art on board. As you mentioned, I just kind of want to know how you expect to kind of leverage his broad expertise.

[music].

Paula Ragan: Do you plan to broaden out the pipeline or pursue additional indications for some of your early-line preclinical programs? Sure. Thanks so much. And, you know, we feel incredibly fortunate to have Art join us, given his strong drug discovery experience and biology overlap in the chemokine world. So, I think when we think about not only Mavrixiv4, but also our two preclinical programs, you know, first, we really need to intend to operate and hit our near-term milestones. For example, I do think that as the drug Mavrixiv4 shows activity in Waldenstrom, there's a tremendous amount of support for CXCR4 more broadly in certain leukemias and lymphomas.

Paula Ragan: I think between Art's experience in biology and then complementing what we're learning in the clinic, that will really help us prioritize how we can either develop Mavrixiv4 and or possibly 003 to expand the total market that we could potentially, you know, explore and hopefully benefit patients with our drugs. Thanks, Paula. And then just a follow-up here. I'm not even sure you can answer this one, but I think on the last call we had, you noted that you were in the process of recruiting patients that were pre-treated or non-pre-treated for the Waldron study, which we are going to try to enroll and see what we get. But can you provide any comments on if you're enrolling more pre-treated patients or are you enrolling more naive patients? I know we, you know, there's no, there's no profile yet that we're really able to disclose.

Paula Ragan: We'll certainly do that when we share the data. But I do like to point back to the literature with respect to double mutant patients; regardless of what line of therapy they're sort of addressed with, they have this remaining unmet need. So I do think the consistency of the impact of having the CXCR4 mutation is important. I think that kind of creates a nice homogeneous patient population for us to study, and we'll look forward to sharing that data in the first half of next year.

Paula Ragan: Thank you. I am not showing any further questions at this time. I would now like to turn the call back over to Paula Ragan for closing remarks. Thank you so much. Well, thank you again today for joining us. We sincerely appreciate your interest in X4 and look forward to continuing to provide updates on our progress as we approach multiple key catalysts in our business. If you have any further questions, please don't hesitate to reach out. Thank you again, and enjoy the rest of your day. Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.