Q1 2022 X4 Pharmaceuticals Inc Earnings Call

May 12, 2022

Greetings and welcome to X4 Pharmaceuticals First Quarter 2022 Financial and Operating Results Conference Call.

Greetings and welcome to X for Pharmaceuticals, first quarter 2022 financial and operating results conference call. At this time, all participants are in a listen only mode.

At this time, our participants are in a listen-only mode.

අපි එය කිරීම කිරීම හොඳින් කරමි.

Everyone have a great day.

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

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 Dr. Glenn Schulman head of Investor Relations. Please begin.

It is now my pleasure to introduce your host, Dr. Glenn Schulman, please begin.

Thank you, operator, and good morning, everyone.

අපි ගැනීමෙන් කරන්න සහ සමඟ අපට කරන්න.

Thank you operator, and good morning, everyone.

Joining on today's call I'll be X four Chief Executive Officer, Dr. Paula Ragan, and the Companys Chief Financial Officer, Adam itself up following.

Presenting on today's call will be X4's Chief Executive Officer, Dr. Paula Ragan, and the company's Chief Financial Officer, Adam Mostafa.

Following prepared marks by each of them, we will open up the, call to your questions, during which we'll be joined by our Chief Scientific Officer, Art DeVaris, Chief Medical Officer, Diego Cadevid, and our Chief Operating Officer, Mary DiBiase.

Following prepared remarks by each of them will open up the call to your questions during which we'll be joined by our Chief Scientific Officer, <unk> <unk>, Chief Medical Officer, Diego category, and our Chief operating Officer Mary <unk>.

As a reminder, on today's call, X4 will be making forward-looking statements regarding regulatory and product development plans, as well as research activities. These statements are subject to risks and uncertainties that may cause actual results to differ arbitrarily from those forecasted. A description of these risks can be found in X4's most recent filings with the SEC.

As a reminder, today's call <unk> will be making forward looking statements regarding regulatory and product development plans as well as research activities. These.

These statements are subject to risks and uncertainties that may cause actual results to differ materially from those forecasted a description of these risks can be found in exports. Most recent filings with the SEC.

With that, I now would like to turn the call over to X4's President and CEO, Dr. Paula Ragan.

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

Paula?

ඇත ඔබට අපි සියලු කරන්න.

Sure.

Thanks, Glenn and thank you everyone for joining us on the call. This morning.

Thanks, Glenn, and thank you, everyone, for joining us on this call this, morning.

අපි කරන්න.

On this morning's call I'll provide a brief update on recent accomplishments and highlight a few of our important upcoming catalysts, including the readout from our phase III for women trial and of course open up the call to any questions. You may have for the team.

On this morning's call, I will provide a brief update on recent accomplishments, highlight a few of our important upcoming catalysts, including the readout from our

අප තද ස්තූතියි.

Phase 3 FORWIM trial, and, of course, open up the call to any questions you may have for the team.

අපි කරන්න.

As a reminder, our lead candidate, Mavrixa4, a CXCR4 antagonist, is being evaluated as an oral, once-daily treatment for people with rare disorders of the immune system, including people diagnosed with WIM syndrome and chronic utopenia, and also for those diagnosed with certain lymphomas. WIM syndrome is a rare immunodeficiency disorder caused by genetic mutations to the CXCR4 receptor. The disease is characterized by HPV-associated warts, hypogamma-globulinemia, multiple types of infections, and myelocosthesis, a pathologic bone marrow finding associated with the reduced ability of white blood cells to move from the bone marrow to the periphery.

අපි ස්තූතියි.

As a reminder, our lead candidate Maverick before <unk> antagonist is being evaluated as an oral once daily treatment for people with rare disorders of the immune system, including people diagnosed with whim syndrome, and client neutropenia and also for those diagnosed with certain lymphoma.

අපි කරන්න.

Whim syndrome is a rare immunodeficiency disorder caused by genetic mutations to the <unk> receptor.

<unk> is characterized by HPV associated warrants.

<unk> gaming globular anemia, multiple types of infections, and Milo contactor a path.

<unk> bone marrow planning associated with the reduced ability white blood cells to move from the bone marrow to the periphery.

We believe that the results from the open-label extension of our Phase 2 clinical trial with Mavrixa4 and WIM patients continue to support the significant potential of our lead drug candidate in this indication, with data showing durable TATs, or time above threshold, for blood levels of neutrophils, lymphocytes, and monocytes, decreased frequency of infections, and robust and sustained improvement in warts.

We believe that the results from the open label extension of our phase III clinical trial with Maverick before in one patient.

To support the significant potential of our lead drug candidate in this indication with data showing durable <unk> our time about threshold.

Love level, neutrophil lymphocyte and modestly.

Decreased frequency of infection and robust and sustained improvement in ward.

Patient quality of life, including reductions in doctor or hospital visits, was meaningfully improved based on our Phase 2 patient narrative.

Keith and quality of life, including reductions in Doctor or hospital visit with meaningfully improve based on our phase <unk> narrowed here.

Importantly, these include the primary and secondary endpoints of our ongoing phase III <unk> trial.

Importantly, these include the primary and secondary endpoints of our ongoing Phase 3 WIM trial.

And just as importantly, Maverix IV continues to be well-tolerated over a median treatment, duration of now more than 160 weeks.

And just as importantly, maverix, where continues to be well tolerated over a median treatment duration of now more than 160 weeks.

We continue to anticipate that top line results from our global placebo controlled double blinded for whim phase III trial, which enrolled a total of 31 adolescent and adult patients who will be available in the fourth quarter of this year.

We continue to anticipate that top-line results from our global placebo-controlled, double-blinded, four-WIM Phase III trial, which enrolled a total of 31 adolescents and adult patients, will be available in the fourth quarter of this year. We intend to report on the primary endpoint, which consists of time above threshold for, absolute neutrophil count, and which was powered based on our findings in the Phase II trial, along with available secondary endpoints. The trial design and primary endpoint have been agreed upon with the FDA.

<unk> to report on the primary endpoint, which consist of time above threshold for absolute neutrophil count.

And which was powered based on our findings in the phase II trial, along with available secondary endpoint.

The trial design and primary endpoints have been agreed upon with the FDA. Additionally, secondary endpoints evaluating inspections and work burden among others have been also discuss it extensively with the FDA and their guidance has been diligently followed.

Additionally, secondary endpoints evaluating infections and warrant burden, among others, have been also discussed extensively with the FDA, and their guidance has been diligently followed.

We look forward to preparing and submitting the new drug application, or NDA, for submission, to the agency in the second half of next year.

We look forward to preparing and submitting the new drug application or NDA for submission to the agency in the second half of next year.

We also continue to conduct and publish research on the underlying genetics of WIM, as we work, to further characterize and expand the definition of the disease.

We also continue to conduct and published research on the underlying genetics of win as we work to further characterize and expand the definition of the disease.

We have built strong in-house research programs that leverage world-class collaborators to, advance bench-to-bedside research.

We have built strong in house research programs that leverage World class truck collaborators to advance bench to bedside research.

By continuing to establish correlations between clinical presentation and novel genetic variance associated with when we can enhance our ability to identify and diagnose patients, including those who may potentially basket from Maverick sport treatments at.

By continuing to establish correlations between clinical presentation and novel genetic variants, associated with WIM, we can enhance our ability to identify undiagnosed patients, including those who may potentially benefit from Maverix IV treatment.

At the upcoming European Hematology Association, or EHA, meeting this June, we plan to present, more of this novel research.

At the upcoming European Hematology Association or <unk> meeting. This June we plan to present more of its novel Research. We will have a press release with more details. This afternoon. After the abstract embargo lifts.

We will have a press release with more details this afternoon after the abstract embargo, lifts, and we hope to see you at the Congress in Vienna.

Hope to see you at the Congress in Vienna.

As I mentioned earlier, we believe there are additional disease areas harboring patients, in need who could also potentially benefit from treatment with Maverix IV.

As I mentioned earlier, we believe there are additional disease areas harboring patients in need who could also potentially benefit from treatment with maverick before with.

With WIM as our beachhead indication well on its way, we are also assessing the potential, of Maverix IV as a therapy for other causes of chronic utopainia, given the drug candidate's potential for meaningful advantages over the only existing therapy.

With Wham as our beachhead indication well on its way. We are also assessing the potential of Maverick before as a therapy for other causes of chronic neutropenia, given the drug candidate potential for meaningful advantages over the only existing therapy.

Chronic neutropenia RCM includes a number of sub types such as.

Chronic utopainia, or CM, includes a number of subtypes, such as congenital, idiopathic, and cyclic utopainia, all of which we believe could benefit from treatment with Maverix, IV.

Congenital idiopathic and cyclic neutropenia.

All of which we believe could benefit from treatment with Maverick before.

In our ongoing Phase Ib study, we are actively enrolling patients diagnosed with these types, of CM to establish biologic activity and support future regulatory discussions.

In our ongoing phase <unk> study, we are actively enrolling patients diagnosed with these types of CN to established biologic activity and support future regulatory discussion.

We look forward to providing both updated clinical data along with regulatory feedback during the third quarter of this year.

We look forward to providing both updated clinical data along with regulatory feedback, during the third quarter of this year. We also announced this morning that we have completed enrollment in our ongoing Phase, Ib clinical trial studying patients diagnosed with Waldenstrom's macroglobulinemia, a rare B-cell lymphoma. This Phase Ib trial is designed to demonstrate safety, dose, and elucidate proof of concept, of Maverix IV in combination with the BTK inhibitor, Ibrutinib, in patients with Waldenstrom's, resulting from mutations in both their MYD88 and CXER4 genes.

We also announced this morning that we've completed enrollment in our ongoing phase <unk> clinical trial setting patients diagnosed with Walton from macro globule anemia.

<unk> B cell lymphoma.

The trial is designed to demonstrate safety dose and elucidate proof of concept of Maverick <unk> in combination with the <unk> inhibitor Bruton Abe.

Since with Walton from resulting from mutations in both <unk> and <unk>.

This patient population continues to have reduced treatment responses due to their cancers, harboring these two mutations.

Patient population continues to have reduced treatment responses.

Their cancer harboring these two mutations.

Doses of 200, 400, and 600 milligrams per day were evaluated, and once cleared, eligible, patients were dose escalated to receive 600 milligrams once daily.

This is a 200 400 600 milligram per day were evaluated and once cleared eligible patients were dose escalated to receive 600 milligrams once daily.

Data that we presented last December at Ash showed a 100% overall response rate or <unk> and sustained decreases in serum IGN blood marker that corresponds with cancer burden and 10, Evaluable patients, who cancer had confirmed <unk> E and <unk> mutation.

Data that we presented last December at ASH showed a 100% overall response rate, or ORR, and sustained decreases in serum IgM, a blood marker that corresponds with cancer burden, in 10 evaluable patients whose cancers had confirmed MYDA8 and CXER4 mutations.

Further to that we also presented additional preclinical results in a poster presentation at the 2022 American Association of cancer research or ACR annual meeting.

Further to that, we also presented additional preclinical results in a poster presentation, at the 2022 American Association of Cancer Research, or AACR, annual meeting. This poster reported that combinations of Maverix-B4 with a broad array of DTK inhibitors, overcame bone marrow-induced treatment resistance and enhanced cancer cell death in in vitro assays of Waldenstroms.

<unk> reported that combinations of Maverick before with a broad array of PTK inhibitors overcame bone marrow and treatment resistance and enhanced cancer cell death.

In vitro assays of Waldman from.

We expect to report results from the Phase 1b clinical study, which we anticipate would, include at least six months of treatment data per patient on the 600 milligram dose during the second half of this year.

We expect to report results from the Phase <unk> clinical study, which we anticipate would include at least six months of treatment data for patients on the 600 milligram Gram dose during the second half of this year.

With three readouts on the horizon, including data and client neutropenia from our Phase <unk> study next quarter results from Waldman from Phase <unk> study in the second half and our <unk> phase III results in the fourth quarter. We are extremely excited for what's to come with.

With three readouts on the horizon, including data in chronic utopia from our Phase 1b study, next quarter, results from Waldenstrom's Phase 1b study in the second half, and our, 4-WIM Phase 3 results in the fourth quarter, we are extremely excited for what's to come.

With that brief update, I'll now turn it over to Adam to discuss our financial results for, the quarter before we open up the call for questions.

අපි ස්තූතියි.

With that brief update I will now turn it over to Adam to discuss our financial results for the quarter before we open up the call for questions Adam.

Adam?

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

අපි කරන්න.

Thanks, Paul 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 first quarter ended March 31 2022.

As presented in our press release this morning, I will summarize our financial activities, and results for the first quarter ended March 31st, 2022. As of the end of the quarter, X4 had $67.7 million in cash, cash equivalents, and restricted, cash.

අපි ස්තූතියි.

අපි කරන්න.

As of the end of the quarter explore had $67 7 million.

අපි ස්තූතියි.

And cash cash equivalents and restricted cash we continue to expect that our cash and cash equivalents will fund our operations into the fourth quarter of 2022.

We continue to expect that our cash and cash equivalents will fund our operations into, the fourth quarter of 2022. Research and development expenses were $14.1 million for the first quarter ended March, 31st, 2022, as compared to $12.1 million for the comparable period in 2021. Selling, general, and administrative expenses were $7.7 million for the first quarter ended, March 31st, 2022, as compared to $5.8 million for the comparable period in 2021. Finally, X4 reported a net loss of $22 million for the first quarter of 2022, which includes, approximately $1.5 million in non-cash expenses, as compared to a net loss of $18.7 million for the comparable period in 2021.

අපි කරන්න.

අපි ස්තූතියි.

Research and development expenses were $14 1 million for.

Greetings and welcome to X4 Pharmaceuticals First Quarter 2022 Financial and Operating Results Conference Call.

For the first quarter ended March 31, 2022, as compared to $12 1 million for the comparable period in 2021.

Yes.

Selling general and administrative expenses were $7 7 million for the first quarter ended March 31 2022.

As compared to $5 8 million for.

For the comparable period of 2021.

Finally, <unk> reported a net loss of $22 million for the first quarter of 2022, which includes approximately $1 5 million.

Noncash expenses as compared to a net loss of $18 7 million for the comparable period in 2021.

We will now open up the call for your questions operator.

We will now open up the call for your questions.

At this time, our participants are in a listen-only mode.

Operator?

A question-and-answer session will follow the formal presentation.

Thank you at this time to ask any questions. Please press star one on your telephone to withdraw your question just press the pound key.

Thank you.

As a reminder, this conference call is being recorded.

At this time, to ask any questions, please press Star 1 on your telephones. To withdraw your question, just press the pound key.

Please begin.

It is now my pleasure to introduce your host, Dr. Glenn Schulman, Head of Investor Relations.

Once again, that's Star 1 for questions, 1-1 for...

Once again Thats star one for questions one more for <unk>.

Thanks for your questions.

Yes.

Our first question comes from the line.

Our first question comes from the line of Marc Frahm from Calend, you may begin.

Thank you, Operator, and good morning, everyone.

From from Cowen you may begin.

Yes.

Hi.

Presenting on today's call will be X4's Chief Executive Officer, Dr. Paula Ragan, and the company's Chief Financial Officer, Adam Mostafa.

Hi, Thanks for taking my questions.

Thanks for taking my questions.

Following prepared marks by each of them, we'll open up the call to your questions, during which we'll be joined by our Chief Scientific Officer, Art DeVeres, Chief Medical Officer, Diego Cadevid, and our Chief Operating Officer, Mary DiBiase.

Just to start off, with the SCN update, Paula, you laid out that there are a couple different, subtypes.

Yes.

With the SCN update I am Paul laid out there a couple of different subtypes.

Can you maybe describe the types of patients you've been able to enroll so far, and therefore, kind of across those different subtypes, you know, which ones might you be able to update us on in Q3, both from a clinical perspective, but then, at least as importantly, you know, from a regulatory perspective?

Can you maybe describe the types of patients you have been able to enroll so far.

And therefore.

Across those different subtypes, which ones might you be able to update us on.

In Q3, both from a clinical perspective, but then at least as importantly from a regulatory perspective.

Sure.

With that, I now would like to turn the call over to X4's President and CEO, Dr. Paula Ragan.

I'll start, and then I'll invite Diego to chime in, but, you know, what we're aiming, to do is to show the breadth of Maverick Sephora's potential benefit in patients with chronic uterpenia.

Paula?

Sure I'll start and then youre going to chime in but what were aiming to do is to show the breadth of maverick before potential benefit in patients with chronic neutropenia.

Thanks, Glenn, and thank you, everyone, for joining us on this call this morning.

Those buckets, as you mentioned, are the idiopathic, cyclic, and then congenital.

Those buckets as you mentioned are there any pathic cyclic and then congenital and I think at this point all we can share is we're certainly feeling confident that we'll be able to share data across each of those three buckets in Q3 against the patient flow. The numbers is always a bit AD hoc in some of these patients are sick.

And I think, at this point, what we can share is we're certainly feeling confident that, we'll be able to share data across each of those three buckets in Q3.

On this morning's call, I will provide a brief update on recent accomplishments, highlight, a few of our important upcoming catalysts, including the readout from our Phase 3 4-WIM trial, and, of course, open up the call to any questions you may have for the team.

As a reminder, our lead candidate, Maverick Sephora, a CXCR4 antagonist, is being evaluated, as an oral once-daily treatment for people with rare disorders of the immune system, including people diagnosed with WIM syndrome and chronic uterpenia, and also for those diagnosed with certain lymphomas. WIM syndrome is a rare immunodeficiency disorder caused by genetic mutations to the CXCR4 receptor. The disease is characterized by HPV-associated warts, hypogamma-globulinemia, multiple types, of infections, and myelocosexus, a pathologic bone marrow finding associated with the reduced ability of white blood cells to move from the bone marrow to the periphery.

We believe that the results from the open-label extension of our Phase 2 clinical trial with, Maverick Sephora and WIM patients continue to support the significant potential of our lead drug candidate in this indication, with data showing durable TATs, or time above threshold, for blood levels of neutrophils, lymphocytes, and monocytes, decreased frequency of infections, and robust and sustained improvement in warts.

Patient quality of life, including reductions in doctor or hospital visits, was meaningfully, improved based on our Phase 2 patient narrative.

We continue to anticipate that top-line results from our global placebo-controlled, double-blinded, four-WIM Phase 3 trial, which enrolled a total of 31 adolescent and adult patients, will be available in the fourth quarter of this year.

Importantly, these include the primary and secondary endpoints of our ongoing Phase 3, WIM trial.

We intend to report on the primary endpoint, which consists of time above threshold for, absolute neutrophil count, and which was powered based on our findings in the Phase 2 trial, along with available secondary endpoints. The trial design and primary endpoint have been agreed upon with the FDA. Additionally, secondary endpoints evaluating infections and wart burden, among others, have been also discussed extensively with the FDA, and their guidance has been diligently followed.

And just as importantly, Maverick Sephora continues to be well-tolerated over a median, treatment duration of now more than 160 weeks.

Again, the patient flow of numbers is always a bit ad hoc, because some of these patients, are, you know, cyclic uterpenia, for example, is a bit challenging just to catch the right window to explore the drug in those patients, but we're really encouraged about what we're already seeing, and maybe I'll turn it over to Diego to add.

<unk> for example are a bit challenging just to catch the right window to explore the drug in those patients.

But we're really encouraged about what where are you seeing and maybe I'll turn it over to Diego to add.

Yes.

Yes, I would just add that we are reassured by the strong interest at all the sites, the, number of potential patients, the number of patients who have consented, as well as patients dosed, and, you know, the change in the conditions of the pandemic is making this study move forward, so we look forward to being able to present data in Q3, as Paula said, that will give us a good sense of all the potential across all these diverse indications within chronic uterpenia.

Then we.

Yes.

Reassured by the strong and finished at all the site.

Okay.

Number of potential patients and number of patients who have.

Consented as well as the patients.

Thanks.

The change in the conditions of the pandemic.

In this study and move forward. So we look forward to being able to play some data in Q3 as Paul said that when you gave us a good faith Hello, the potential across all of these indications we think coming in therapy.

That's helpful.

We look forward to preparing and submitting the New Drug Application, or NDA, for submission, to the agency in the second half of next year.

We also continue to conduct and publish research on the underlying genetics of WIM, as we work, to further characterize and expand the definition of the disease.

We have built strong in-house research programs that leverage world-class collaborators to, advance bench-to-bedside research.

At the upcoming European Hematology Association, or EHA, meeting this June, we plan to present, more of this novel research.

We will have a press release with more details this afternoon after the abstract embargo, list, and we hope to see you at the Congress in Vienna.

As I mentioned earlier, we believe there are additional disease areas harboring patients, in need who could also potentially benefit from treatment with Mavrixiv4.

With WHIM as our beachhead indication well on its way, we are also assessing the potential, of Mavrixiv4 as a therapy for other causes of chronic neutropenia, given the drug candidate's potential for meaningful advantages over the only existing therapy. Chronic neutropenia, or CM, includes a number of subtypes, such as congenital, idiopathic, and cyclic neutropenia, all of which we believe could benefit from treatment with Mavrixiv4. In our ongoing Phase 1b study, we are actively enrolling patients diagnosed with these types, of CM to establish biologic activity and support future regulatory discussions.

Okay. Thanks, that's helpful and then maybe for Adam.

And then, new for Adam, we appreciate the cash guidance last week in Q4, can you remind, us the kind of status of the covenants on your debt, and just kind of where that is?

We look forward to providing both updated clinical data along with regulatory feedback, during the third quarter of this year.

We also announced this morning that we have completed enrollment in our ongoing Phase, 1b clinical trial studying patients diagnosed with Waldenstrom's macroglobulinemia, a rare, B-cell lymphoma. This Phase 1b trial is designed to demonstrate safety, dose, and elucidate proof of concept, of Mavrixiv4 in combination with the BTK inhibitor, Ibrutinib, in patients with Waldenstrom's, resulting from mutations in both their MYD88 and CXER4 genes. This patient population continues to have reduced treatment responses due to their cancers, harboring these two mutations.

Doses of 200, 400, and 600 milligrams per day were evaluated, and once cleared, eligible, patients were dose escalated to receive 600 milligrams once daily. Data that we presented last December at ASH showed a 100% overall response rate, or ORR, and sustained decreases in serum IgM, a blood marker that corresponds with cancer burden, among 10 evaluable patients whose cancers had confirmed MYD88 and CXER4 mutations.

I appreciate the cash guidance lessen in Q4 can you remind us the okay.

Status of the <unk>.

Government on.

You bet, and just kind of where where that is.

Sure.

Sure. Thanks, Mark that we have.

Thanks, Mark.

So, we have a minimum cash covenant.

Minimum cash covenant.

That test kicks in on September 1st. We have a recent amendment where if we raise, at this point, another $27 million by June, 30th, that minimum cash number will be fixed at $30 million until we get to WIM data, where with positive WIM data, it will be $20 million.

<unk> Tec kicks in on September one.

We have a recent amendment, where if we raise at this point another $27 million by June 30th that minimum cash number will be fixed at $30 million until we get to when data wherewith positive when data will be $20 million.

If we do not raise that capital before June 30, and that test will simply be six month cash burn so there'll be a prior one month burn and fix.

If we do not raise that capital before June 30th in that test, it will simply be a six-month, cash burn test. So, it will be our prior one-month burn times six that we would need to keep in terms of, cash on the balance sheet at that point in time to cover the test.

We would need to keep in terms of cash on the balance sheet at that point in time to.

To cover the test.

So, that's a little bit around the covenant.

Okay.

So that's a little bit around the company.

Yep.

Very helpful.

Yes.

All right.

Very helpful. Thank you.

Thank you.

And our next question will be from Mayank Mamtandi from B. Riley Securities.

And our next question Glenn.

Tani from B Riley Securities.

You may begin.

May begin.

Hi.

Good morning, everybody.

This is William Wood.

I'm from Mayank Mamtandi.

Hi, Good morning, everybody. This is Brian wood on for my on a really appreciate you taking our calls and congratulations on all the <unk>.

I really appreciate you taking our calls and congratulations on all the advancements that, you've made.

The investments that you've made a couple of questions from US I was wondering if you could first could you talk to how your view Adobe.

A couple of questions from us.

I was wondering if you could first, could you talk to how your view, the WM landscape, in light of the experience of our targeted therapies and where you may think that the third quarter readout has an opportunity to improve perception as it relates to both safety and efficacy.

Wm landscape in light of the experience of our targeted therapies.

And where you may think.

The third quarter readout has an opportunity to improve perception as it relates to both safety and efficacy.

Thank you.

Sure.

Sure I'll start.

And again I think it was <unk> that youre asking about just to confirm.

I'll start.

Yes, correct.

And again, I think it was Waldenstrom that you're asking about just to confirm?

Yes.

Guidance is that we'll be sharing data second half of this year and in terms of the competitive landscape of course there is.

Yes, correct.

A lot of a number of BT.

PTK inhibitors, given the validation of the target that are moving forward in the pipeline and certainly there are two approved two or three approved I think again, we continue to see the importance of adding a <unk> four antagonist across any of those be PK inhibitor is with respect to patients with <unk> mutations.

Initially and certainly we see broader applicability beyond that we just presented some really exciting data at ACR, which shows the breadth of activity of our drug not only in double mutant but of course the single non U.

Okay.

Further to that, we also presented additional preclinical results in a poster presentation, at the 2022 American Association of Cancer Research, or AACR, annual meeting. This poster reported that combinations of Mavrixiv4 with a broad array of BTK inhibitors, overcame bone marrow-induced treatment resistance and enhanced cancer cell death in in vitro assays of Waldenstrom's.

With that brief update, I'll now turn it over to Adam to discuss our financial results for, the quarter before we open up the call for questions.

<unk> is now.

Adam?

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

And then in terms of safety, our drug is very positive or benign safety profile, which makes it more amenable to partnering with some of these tougher treatment. So youll Ralph therapeutic window in combination seem to say reasonably wide when we add drugs in combination again favoring our approach versus what we're seeing with other combination.

As presented in our press release this morning, I'll summarize our financial activities and, results for the first quarter ended March 31, 2022.

As of the end of the quarter, X4 had $67.7 million in cash, cash equivalents, and restricted, cash. We continue to expect that our cash and cash equivalents will fund our operations into, the fourth quarter of 2022. Research and development expenses were $14.1 million for the first quarter ended March, 31, 2022, as compared to $12.1 million for the comparable period in 2021.

Selling general and administrative expenses were $7.7 million for the first quarter ended, March 31, 2022, as compared to $5.8 million for the comparable period in 2021.

Finally, X4 reported a net loss of $22 million for the first quarter of 2022, which includes, approximately $1.5 million in non-cash expenses, as compared to a net loss of $18.7 million for the comparable period in 2021.

Yeah.

We'll now open up the call for your questions.

But diego would you like to chime in and then art as well.

Operator?

Thank you.

At this time, to ask any questions, please press Star 1 on your telephones. To withdraw your question, just press the pound key.

Yes.

So that we believe our separately is quite differentiated from some of the older trucks being combined in the sense that for example, the metal kiosks has had a lot of neutropenia.

Channel routine April also showed a lot of neutropenia, you'll seen with Ibrutinib, we actually have that drove that $3 neutropenia. So in the context of having re scoping peg Xiaomi is combination treatments ultimately the safety of the combination will become very important for the chronic treatment of indolent lymphomas.

So we are.

Quite excited about the potential of <unk> to be competitive.

A candidate in that space.

Maybe I can add a little bit starting from the neutropenia site. There is reports already in the literature that talks about it prudently Jon Gruden of a caliber nib actually showing neutropenia and Lymphocytopenia and then the corresponding increase in infections is a grade three through five SaaS.

And so we certainly are drug that we've measured maverick support in combination with <unk> in our.

What was phase one b trial across the patients and we could actually see increases in all of these leukocyte. So we actually do.

And believe that this is going to be a benefit with respect to safety and particularly the infection safety related to competing in lymphocytopenia.

And then second just related to the data that we presented at ACR and as Colette mentioned earlier, when we were working with a single view and wild type.

CX tier four and some mighty EBITDA fell 265 PV patient cells.

And there what we found is that.

All of the agents that we look copies of the PK agonist that are out there so a prudent guidance.

Commercial ones and then lock so 300 <unk> from Lilly and the Merck are cool 531, and what we found is that these agents while they work on their own and once you start to involve bone marrow, which is really where CX here for double mutation really starts to play. Then then you see that they become resistant.

To the therapy, the tumor cells to become resistant to the therapy and Mavericks was able to overcome that.

A lot of opportunities here broadens, obviously, the combinations that could be used so its not just a burden it with networks before but it could be any of these other agents whether they are commercial currently or potentially the ones that are in clinical development. It broadens the opportunity across a single meetings, which now upwards related to <unk>. So.

It's not just about the double mutants and then the work that we're doing right now is broadening out even further to other lymphomas. So I think we have some good breadth of opportunities with <unk> four and the combinations actually it could be quite a few touch us with ibrutinib.

Thank you so much really appreciate the extra information there.

So guidance is that we'll be sharing data second half of this year.

And also I mean.

As you presented you have got a number of clinical trials.

Providing data in the.

Second half I was just curious where we might.

We expect to see those data coming out throughout the year.

Yes, I think we'll provide greater clarity on the different venues that we'll be sharing data.

Finally, with the three different milestones.

<unk> milestone and then the wind phase III as well as the long term and sort of a nice drumbeat of information.

And that will try to optimize to make sure. The community is kind of educated and understanding the data when they rollout, but stay tuned we'll be able to provide some more clarity in the coming months.

Awesome. Thank you so much congratulations again.

But there thanks.

And in terms of the competitive landscape, of course, there's a lot of, a number of CTK inhibitors, given the validation of the target that are moving forward in the pipeline.

Once again, that's Star 1 for questions, 1-1 for questions.

Thank you.

Our next question comes from the line of volume.

Ocular <unk> from HC Wainwright you may begin.

Certainly there's two approved, two or three approved.

Our first question will come from the line of Mark Frum from McAllen.

Thank you additional RK from H C Wainwright.

You may begin.

Hi.

Good morning Paula.

Adam.

<unk>.

So.

I think, again, we continue to see the importance of adding a CXCR4 antagonist across any of those CTK inhibitors, with respect to patients with the CXCR4 mutations.

Thanks for taking my questions.

Talking about the three buckets of patients.

Chronic can neutropenia.

<unk>.

Is the biology, the same or similar.

Just to start off, with the SCN update, Paula, you laid out that there are a couple of different, subtypes.

Among the three buckets.

Yes.

You stated that you would have data among the three buckets. So.

Assuming that the data is similar.

Is there a preference to go after one of them or.

The decision point there.

Initially, and certainly we see broader applicability beyond that, we just presented some really exciting data at AACR, which shows the breadth of activity of our drug, not only in double mutants, but of course the single, the non-mutant Waldenstrom cells as well.

Yes, so I think our rationale is certainly there is other G. CSF is the only approved treatment. It has been shown to to offer some benefit to those patients with significant tolerability issues and.

And then in terms of safety, you know, our drug has got a very positive or benign safety profile, which makes it more amenable to partner with some of these tougher treatments.

So the overall therapeutic window in combination seems to stay reasonably wide when we add drugs in combination, again, favoring our approach versus what we're seeing with other combinations.

And then therefore, the antagonist <unk> antagonism as a broad mechanism as well we've seen it to elevate white blood cell counts in any subject our patient population. So it has tremendously broad opportunity to increase <unk> in any form of neutropenia.

You saw that in our ash data with our idiopathic patients.

Have a known cause of neutropenia and very robust effects.

Kind of already checked one of the three boxes and we look forward to sharing more data, but I think maybe on buy art and a little bit more to talk about the mechanistic rationale for matrix for across these different bucket.

But Diego, would you like to chime in, and then Art as well?

Can you maybe describe the types of patients you've been able to enroll so far, and therefore, kind of across those different subtypes, which ones might you be able to update us on in Q3, both from a clinical perspective, but then, at least as importantly, from a regulatory, perspective?

Hi, RK how are you.

Yeah, I would add also that we believe mavorixazole is quite differentiated from some of the other drugs being combined, in the sense that, for example, venetoclax has a lot of neutropenia.

Sure.

Xanagrutinib also showed a lot of neutropenia. You see it with irutinib.

I'll start, and then I'll invite Diego to chime in, but, you know, what we're aiming, to do is to show the breadth of Maverick Sephora's potential benefit in patients with chronic atrophenia.

So maybe I'll just talk a little bit about the.

Overall chronic neutropenia landscape, if you will and so we've been maybe start from wind and then also the connection back to Wall Street. So there we were focused really on mutated CX. Therefore, but the work that we have done and what was your particular showed us that actually it's more than just muted, it's even wild type 60, or four and how that gets.

We actually have a drug that treats neutropenia.

Those buckets, as you mentioned, are the idiopathic, cyclic, and then congenital.

So in the context of having risk of infection in these combination treatments, ultimately the safety of the combination will become very important for a chronic treatment on indolent lymphomas.

And I think, at this point, what we can share is we're certainly feeling confident that, we'll be able to share data across each of those three buckets in Q3.

Again, the patient flow of numbers is always a bit ad hoc, because some of these patients, are, you know, cyclic nitropenia, for example, is a bit challenging just to catch the right window to explore the drug in those patients, but we're really encouraged about what we're already seeing.

So we are quite excited about the potential of mavorixazole to be a competitive candidate in that space. Maybe I can add a little bit, starting from the neutropenia side. There's reports already in the literature that talks about Xanagrutinib, actually showing neutropenia and lymphocytopenia, and then the corresponding increase in infections.

And maybe I'll turn it over to Diego to add.

It's a great three through five SAEs and AEs.

And so we certainly are drugging.

Yes.

I would just add that we are reassured by the strong interest at all the sites, the, number of potential patients, the number of patients who have consented, as well as patients who have been diagnosed.

And, you know, the change in the conditions of the pandemic is making this study move, forward.

Up regulated under various scenarios and then certainly across and this is work that Diego and presented at Ash in December that across all the different clinical programs that we've been doing we see leukocyte mobilization when patients treated with <unk> four and we see baseline elevations of leukocytes across the board.

We've measured mavorixazole in combination with Ibrunib in our Wolfenstern's Phase 1b trial across the patients, and we can actually see increases in all of these leukocytes.

So this is actually pretty promising because it actually says it's more than just mutated <unk> four and it's more than just the specific two diseases that we've been talking about so now with the <unk>.

So we look forward to being able to present data in Q3, as Paula said, that will give, us a good sense about the potential across all these diverse indications within chronic neutropenia.

Broader market expansion and opportunity across other patients start with chronic neutropenia and what we're focused on is the potential of Maverick sport.

Okay.

So we actually do believe that this is going to be a benefit with respect to safety, and particularly the infection safety related to neutropenia and lymphocytopenia.

Thanks.

That's helpful.

<unk> neutrophil levels in those patients and ended the simplest way is just take the patient to get them networks before and see if they respond.

In vitro to be able to assess pathogenic measures and so from the data the Diego presented in December .

And then second, it's related to the data that we presented at AECR.

Seeing that these patients do respond so clearly there is a <unk> year for our component and what we've always talked about is that <unk> four and its natural law against <unk> 12 at the heart Master regulator in terms of granular polices and amount of leases in general So thats, a nice connection and it's really all about CX three or four for us.

And as Paula mentioned, there what we were working with was a single mutant wild type of CXCR4, and these are MITE-ADA L265P mutation cells.

And there what we found is that all of the agents that we looked at, these are the BTK antagonists that are out there, so Ibrunib and Xanagrutinib, two commercial ones, and then LOXO305 from Lilly and the Merck RQL531.

And what we found is that these agents, while they work on their own, and once you start to involve bone marrow, which is really where CXCR4 double mutation really starts to play, then you see that they become a bit resistant to the therapy. The tumor cells become resistant to the therapy. And mavorixazole is able to overcome that.

A lot of opportunities here.

It broadens, obviously, the combinations that could be used.

So it's not just Ibrunib with mavorixazole, but it could be any of these other agents, whether they're commercial currently or potentially the ones that are in clinical development.

Fantastic. Thank you very much for that.

And then on the on the Lynch syndrome, with the data coming out in fourth quarter.

And then, maybe for Adam, we appreciate the cash guidance, last in the Q4.

Can you remind us the kind of status of the covenants on your debt and just kind of where that is?

So.

At the same time.

It broadens the opportunity across single mutants, which now have upregulated CXCR4.

You folks were identifying new mutations within the <unk> four.

So when you present, the data our renewal start to analyze the data from fourth quarter onwards.

So it's not just about the double mutants.

Are you going to go back and look at individual.

Individually youre going to be looking at the mutations just to understand how broad.

<unk> can be working on.

Do you think there would be any mutations where maverix will or will not be able to interact with are not able to.

No.

Again, the benefit some of those patients I'm just trying to understand how broad you can use from maverick before.

Sure.

Andrew.

Okay. That's a great question and maybe I could just share of the work that we can do on the teams what we call. The <unk>, which are really a number of CX for mutation. So at this point, we've analyzed probably close to about 70.

Sure.

And then the work that we're doing right now is broadening out even further to other lymphomas.

Thanks, Mark.

So I think we have some good breadth of opportunities with mavorixazole, and the combinations actually could be quite a few, not just with Ibrunib.

Thank you so much, we really appreciate the extra information there.

So, we have a minimum cash covenant.

That test kicks in on September 1st. We have a recent amendment where if we raise, at this point, another $27 million by June 30th, that minimum cash number will be fixed at $30 million until we get to WIM data, where, with positive WIM data, it will be $20 million.

If we do not raise that capital before June 30th in that test, it will simply be a six-month cash burn test. So, it will be our prior one-month burn times six that we would need to keep in terms of cash on the balance sheet at that point in time to cover the test.

So, that's a little bit around the covenants.

Yep.

Patients and make it really cover span the different parts of the <unk> four receptor and what we find is.

Very helpful.

And our next question will be from Mayank Mamtani from B. Riley Securities.

All right.

Thank you.

You may begin.

Hi.

Most of them are pathogenic and then we know that Mavericks for buying two or three.

Therefore, it means that we've tested so far and so or at least blocks the binding of <unk> hundred 12.

We've tested so far so we believe that it's going to be effective in any one of the six year for mutations and that should be irrespective of the mutation.

Yes, I would simply add that we already tested in the context there.

The phase II trial defend mutations on the alloy sponsor.

So we're very confident in between the in vitro data on our own in vivo data and in fact, we haven't rollout breath, hoping of patients in the phase III.

Bottom line is we believe it will work across all patients.

Patients in fact, because of our currently neutropenia, we know that it also works on the wild type. So it's a big promise to have.

For the first time, our model well tolerated therapy to treat people who are at risk of serious infections from chronic neutropenia.

And also, I mean, as you presented, you've got a number of clinical trials providing, data and the second half, I was just curious where we might be able to expect to see those data coming out throughout the year.

Fantastic that's great. So one last question.

On the.

The Orleans charms.

Indication when data comes out in.

In the second half.

What are the next steps in defense.

<unk>.

Would you like to listen to an Encore Conference press, one for yes, or two from now.

Hello.

Can you repeat that arent that I'm not sure what that.

<unk>.

Okay.

Yeah, I think we'll provide greater clarity on the different venues that we'll be sharing, data.

On the volumes from syndication whats the next step in the sense. Once the data is presented in the second half would you be able to go straight into Registrational study or do you need to do additional studies before you go into Registrational.

Certainly, with the three different milestones, so the chronic utropenia milestone, and then, the WIM Phase 3, as well as the Waldenstroms, we have sort of a nice drumbeat of information, and that we'll try to optimize to make sure the community is kind of educated and understanding the data when they roll out.

Obviously, we're waiting for the data to make sure and then have regulatory discussions.

There has really only been two drugs approved and Walton from historically, so I think that the answer is stay tuned obviously data plus regulatory input than would enable us to plan for the future and.

So stay tuned, we'll be able to provide some more clarity in the coming months.

So we'll be able debt that's our milestone for the second half to provide that guidance.

Looking forward to sharing more RK.

Thank you. Thank you very much for taking all my questions I appreciate it. Thank you.

And once again as a reminder, that star one quick question Starwood.

Your line of Trevor already from.

Awesome, thank you so much.

Oppenheimer you may begin.

Congratulations again.

Good morning, everybody.

Hey, good morning.

Wanted to ask if you had any conversations with Lilly Merck about.

Combination potential and any expectations for how you might advance that combo.

Also wanted to ask if you're seeing a potential role for <unk> and activate <unk> Delta syndrome.

So Adam I'll take the BD question Yeah. Thanks for the question, we won't comment on specific names folks, who we've been talking to but certainly work.

Exploring different alternatives and discussions as it relates to getting the most value from our pursuits.

Our balance sheet.

In oncology and in general, but well circle back when appropriate with any specifics.

In the future.

Yeah, and then with respect to <unk> K Delta.

Certainly the pathway signaling pathway overlap with the Air force, So theres, a mechanistic rationale to support Maverick for patients as well.

Yet tested specifically any patients, but it's something that we're considering for future studies.

Okay, great. Thanks.

I'll leave it there, thanks.

This is William Wood.

And our next question comes from the line of Zack <unk>.

<unk> from Roth capital you may begin.

Sure.

Good morning, Thanks for taking our questions.

Thank you.

I'm from Mayank Mamtani.

Three quick ones here.

Our next question will come from the line of Swayampakula Ramakhan from HC Wainwright.

This is about the Walton from data at that time at that last update.

You may begin.

You had really strong responses in relapsed refractory patients.

A little less in the frontline setting. So I was just wondering with enrollment now complete I would you expect more patients.

Relapsed refractory patients versus frontline patients and then also regarding the 600 milligram dose.

Got it to be used.

How should we think about the potential for that dose dependent change the efficacy profile that we've seen for that program and I have a couple follow ups.

Yes. Thank you for the question.

Thank you.

Really appreciate you taking our calls and congratulations on all the advancements that, you've made.

We believe the potential for an hour except for in combination with <unk> is broad we chose to focus first on CX.

A couple of questions from us.

Double mutants because they have the highest unmet date.

We.

Believe that the drug.

Actually we are really good add on for either frontline always absolutely factory and we have enrolled both.

In our trial and we will report on that later in the year.

I mean as I've mentioned earlier in vitro we have.

Really happy with.

Cellular models that show efficacy even in <unk> four in the context of modeling a bone marrow niche. So this speaks to the broad potential of CX <unk>.

<unk> based in the bond model, so what will come next.

Paul already mentioned will focus on Walden from the all new terms, but we believe the potential is much broader.

Thank you Diego and then the follow up here is just about the limber program you're on track to have your NDA submitted which is the big deals or looking at the way. The stock is look what I was wondering if you could just kind of again highlight the the commercial potential talk about.

Some of the steps that you've taken I know you have a.

The mine had in commercial so just kind of wonder if you can just kind of speak.

To that point, a little bit and then I have a last one in Arizona.

Sure.

In terms of the potential for Maverick foreign win we continue to guide that there is at least about 1000 genetically confirmed a confirmed when patients in the U S and possibly much more than that in terms of the under diagnosed or undiagnosed and we're making exceptional progress towards building that patient base education awareness and getting.

Ready for <unk>.

A positive launch maverix far be approved.

Our new VP of U S commercial.

Definitely try and experience in this area, we've got a great team on the ground already and I think we're really ramping up for a successful.

And ideal launch post post our phase III milestone this year, so stay tuned on more details, but we're excited about already the breadth and diversity of patients as art mentioned over 70 generic <unk>.

And then also the diversity of the products potentially even in chronic <unk> beyond win.

Thanks, Paula and I didn't know if you can't say much about this but this kind of that lease.

Based on what you just ended with regarding the commercial potential I think you guys have highlighted about 60000 patients.

<unk>.

The different diseases that youre looking at.

Also going to have pretty much strong proof of concept data across all three indications by the end of the year and mistaking you're kind of pushing to even flatten this out some more so I am clear.

Clearly I think that there should be some.

Partnership and Jason So is that something that you guys are.

Loring, perhaps not just one indication by across the board.

<unk> is a floor and then you can maybe comment on how youre thinking about maybe.

Leaching ex U S opportunities.

Sure So I mean.

The World is a big place.

Obviously, you have a home field advantage in the U S and we have a drug that appears to work in women at that some amazing.

Momentum behind it with these other indications so theres a lot of opportunity for patients and for <unk>.

Sort of commercial investment and return on that investment.

We won't be able to share more of how we're thinking but were certainly believing that in the U S. We can handle the commercial setting given the build that we're already investing in and the fruits of that bell, but ex U S is still a part of our strategy that's evolving and certainly there's a lot of companies that create the right structure to support the most patients globally and alpha <unk>.

The overall investment and return on that that's needed.

Thanks, and then congrats on the progress.

Thank you.

And once again Thats star one for any questions for questions.

This is RK from HC Wainwright.

Good morning, Paula and Adam.

And I'm not showing any further questions in the queue I'd like to turn the call back over to Dr. Paula Ragan for any closing remarks.

So talking about the three buckets of patients under chronic and utropenia, is the biology, the same or similar among the three buckets?

Well, we'd just like to say thank you for everyone for participating in our investor call today and thanks for the excellent questions. If you have any further follow up please reach out to Glen and we'd be happy to dialog offline. Thank you again and have a great day.

And you stated that you would have data among the three buckets.

So assuming that the data is similar, is there a preference to go after one of them, or what is the decision point there?

This concludes today's conference call. Thank you for participating you may now disconnect everyone have a great.

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Yeah, so I think our rationale is certainly there's other – GCSF is the only approved, treatment that has been shown to offer some benefit to those patients with significant tolerability issues.

I was wondering if you could first, could you talk to how you view the WIM landscape in light of the experience of our targeted therapies and where you may think that the third-quarter readout has an opportunity to improve perception as it relates to both safety and efficacy?

Greetings and welcome to X for Pharmaceuticals, first quarter 2022 financial and operating results conference call at this time all participants.

We are in a listen only mode.

A question and answer session will follow the formal presentation. As a reminder, this conference call is being recorded it is now my pleasure to introduce your host Dr. Glenn Schulman head of Investor Relations. Please begin.

Sure.

And then CXER4's antagonism is a broad mechanism as well.

You operator, and good morning, everyone presenting on today's call will be <unk>, Chief Executive Officer, Dr. Paula Ragan, and the Companys Chief Financial Officer, Adam its bile flow following prepared remarks by each of them will open up the call to your questions.

Which we'll be joined by our Chief Scientific Officer, <unk>, <unk>, Chief Medical Officer, Diego category, and our Chief operating Officer Mary Dibiase.

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

We've seen it to elevate white blood cell counts in any subject or patient population, so it has this tremendously broad opportunity to increase neutrophils in any form of neutropenia.

I'll start.

With that I'd now like to turn the call over to export as President and CEO , Dr. Paula Ragan.

Sure.

You saw that in our ASH data with our idiopathic patients. Those folks have unknown cause of neutropenia and we found very robust effects.

And again, I think it was Waldenstroms that you're asking about just, to confirm.

Thanks, Glenn and thank you everyone for joining us on the call. This morning.

So we've kind of already checked one of the three boxes, and we'll look forward to sharing, more data.

Yes, correct.

On this morning's call I'll provide a brief update on recent accomplishments and highlight a few of our important upcoming catalysts, including the readout from our phase III for whim trial and of course open up the call to any questions. You may have for the team.

I think maybe I'll invite Arden a little bit more to talk about the mechanistic rationale, for Maverix IV across these different buckets.

Yeah.

So, guidance is that we'll be sharing data second half of this year.

And in terms, of the competitive landscape, of course, there's a lot of, a number of CTK inhibitors given the validation of the target that are moving forward in the pipeline.

Hi, R.K.

Certainly, there's two approved, two or three approved.

As a reminder, our lead candidate Maverick before a <unk> four antagonist is being evaluated as an oral once daily treatment for people with rare disorders of the immune system, including people diagnosed with whim syndrome, and client neutropenia and also for those diagnosed with certain lymphomas.

Whim syndrome is a rare immunodeficiency disorder caused by genetic mutations to the <unk> receptor.

I think, again, we continue to see the importance of adding a CXCR4 antagonist across any of those CTK inhibitors with respect to patients with, the CXCR4 mutations.

<unk> is characterized by HPV associated warrants.

<unk> gaming globular anemia, multiple types of infections and Milo conductor.

A pathologic bone marrow planning associated with the reduced ability of white blood cells to move from the bone marrow to the periphery.

We believe that the results from the open label extension of our phase III clinical trial with Maverick before and when patients continue to support the significant potential of our lead drug candidate in this indication with data showing durable T ats or a time of our threshold or blood level of neutrophil <unk>.

How are you?

So maybe I'll just talk a little bit about the overall chronic neutropenia landscape, if you will.

The site and modestly.

Decreased frequency of inspection and robust and sustained improvement in ward.

Please and quality of life, including reductions in Doctor or hospital visits with meaningfully improved based on our phase <unk> narrowed here.

Importantly, these include the primary and secondary endpoints of our ongoing phase III <unk> trial and.

And so we've been – and maybe start from WIM and then also the connection back to Wallenstrom's.

And just as importantly, Mavericks, where it continues to be well tolerated over a median treatment duration of now more than 160 weeks.

We continue to anticipate that top line results from our global placebo controlled double blinded for whim phase III trial, which enrolled a total of 31 adolescent and adult patients will be available in the fourth quarter of this year.

We intend to report on the primary endpoint, which consist of time above threshold for absolute neutrophil count.

And which was powered based on our findings in the phase III trial, along with available secondary endpoint.

The trial design and primary end points have been agreed upon with the FDA.

Additionally, secondary endpoints evaluating infections and work burden among others have been also discuss it extensively with the FDA and their guidance has been diligently followed.

We look forward, you're preparing and submitting the new drug application or NDA for submission to the agency in the second half of next year.

We also continued to conduct and published research on the underlying genetics of win as we work to further characterize and expand the definition of the disease.

We have built strong in house research programs that leverage World class truck collaborators to advance bench to bedside research.

At the upcoming European Hematology Association or <unk> meeting. This June we plan to present more of this novel Research. We will have a press release with more details. This afternoon after the abstract embargo lifts.

Hope to see you at the Congress in Vienna.

As I mentioned earlier, we believe there are additional disease areas hiring patients in need who could also potentially benefit from treatment with maverick before with.

Chronic neutropenia RCM includes a number of sub types, such as congenital idiopathic and cyclic neutropenia.

All of which we believe could benefit from treatment with Maverick before.

In our ongoing phase <unk> study, we are actively enrolling patients diagnosed with these types of CMS to establish biologic activity and support future regulatory discussion.

We look forward to providing both updated clinical data along with regulatory feedback during the third quarter of this year.

We also announced this morning that we have completed enrollment in our ongoing phase <unk> clinical trial studying patients diagnosed with Walden from macro globule anemia.

So there, you know, we were focused really on mutated CXER4, but the work that we had, done in Wallenstrom in particular showed us that actually it's more than just mutated. It's even wild-type CXER4 and how that gets upregulated under various scenarios.

And then certainly across – and this is work that Diego presented at ASH in December, – that across all the different clinical programs that we've been doing, we see leukocyte mobilization in patients treated with Maverix IV, and we see baseline elevations of these leukocytes across the board.

All right.

Rare b cell lymphoma.

So this is actually pretty promising because it actually says it's more than just mutated, CXER4, and it's more than just the specific two diseases that we've been talking about.

So now it's a broader market expansion and opportunity across other patients now with, chronic neutropenia.

<unk>. The trial is designed to demonstrate safety dose and elucidate proof of concept of Maverick four in combination with the <unk> inhibitor <unk> in.

And what we're focused on is the potential of Maverix IV to raise the neutrophil levels, in those patients.

And the simplest way is just take the patient, give them Maverix IV and see if they respond, for us in vitro to be able to assess pathogenic measures.

In patients with Waldman from resulting from mutations in both <unk> and <unk>.

This patient population continues to have reduced treatment responses due to their cancers harboring <unk> two mutation.

This is a 200 400 600 milligram per day were evaluated.

Im cleared eligible patients would get escalated to receive 600 milligrams once daily.

Data that we presented last December at Ash showed a 100% with overall response rate or <unk> and sustained decreases in serum IGN, a blood marker that corresponds with cancer burden and 10 evaluable patients to cancer had confirmed <unk> E and.

And so from the data that Diego presented in December at ASH, we're seeing that these, patients do respond.

So clearly there's a CXER4 component, and what we've always talked about is that CXER4, and its natural ligand CXER12 at the heart, their master regulator in terms of granulopoiesis and amyotopoiesis in general.

So that's a nice connection, and it's really all about CXER4 for us.

An <unk> mutation.

Fantastic.

Further to that we also presented additional preclinical results in a poster presentation at the 2022 American Association of cancer research or ACR annual meeting.

Thank you very much for that.

Initially, and certainly we see broader applicability beyond that, we just presented some really exciting data at AACR, which shows the breadth of activity of our drug, not only in double mutants, but, of course, the single, the non-mutant Waldenstrom cells as well.

This poster reported that combinations of Maverick before with a broad array of PTK inhibitors.

<unk> bone marrow and treatment resistance and enhanced cancer cell death in in vitro assays of Walton from you.

We expect to report results from the Phase <unk> clinical study, which we anticipate will include at least six months of treatment data for patients on the 600 milligram Gram dose during the second half of this year.

With three readouts on the horizon, including data and chronic neutropenia from our phase <unk> study next quarter results from loans from Phase <unk> study in the second half and <unk> phase III results in the fourth quarter. We are extremely excited for what's to come with.

And then on the VIMS syndrome, with the data coming out in fourth quarter, so at the same, time, you know, you folks were identifying new mutations within the CXC04.

So when you present the data or when you start to analyze the data from fourth quarter onwards, are you going to go back and look at individually, are you going to be looking at the mutations just to understand how broad Maverick XO4 can be working on?

With that brief update I'll now turn it over to Adam to discuss our financial results for the quarter before we open up the call for questions Adam.

And do you think there would be any mutations where Maverick XO4 will not be able to interact, with or not able to, you know, get the benefit, you know, some of these patients?

Thanks, Paul 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 first quarter ended March 31 2022.

I'm just trying to understand how broad you can use Maverick XO4 in the VIMS syndrome.

Okay.

As of the end of the quarter explore had $67 7 million.

And cash cash equivalents and restricted cash.

We continue to expect that our cash and cash equivalents will fund our operations into the fourth quarter of 2022.

Research and development expenses were $14 1 million for the first quarter ended March 31, 2022, as compared to $12 1 million for.

For the comparable period in 2021.

Selling general and administrative expenses were $7 7 million for the first quarter ended March 31 2022.

As compared to $5 8 million.

For the comparable period of 2021.

Finally, <unk> reported a net loss of $22 million for the first quarter of 2022, which includes approximately $1 5 million in.

Noncash expenses as compared to a net loss of $18 7 million for the comparable period in 2021.

That's a great question.

We will now open up the call for your questions operator.

Thank you at this time to ask any questions. Please press star one on your telephone to withdraw your question just.

And maybe I could just share the work that we've been doing in what we call the BUSs, which are really a number of CXC04 mutations.

So at this point, we've analyzed probably close to about 70 mutations, and they really, cover, span the different parts of the CXC04 receptor.

Once again Thats star one for questions one more for Cui.

<unk>.

Our first question comes from the line.

Marc Frahm from Cowen you may begin.

Got it.

Hi, Thanks for taking my questions.

And what we find is that most of them are pathogenic.

And then we know that Maverick XO4 binds to all of the CXC04 mutants that we've tested, so far.

And so, or at least blocks the binding of CXC012 to all mutants that we've tested so, far.

Yes.

So we believe that it's going to be effective in any one of the CXC04 mutations, and that, should be irrespective of the mutation.

With the SCN update I'm, Paul laid out that there are a couple of different subtypes.

Can you maybe describe the types of patients who have been able to enroll so far.

And therefore.

Across the different subtypes, which ones might you be able to update us on.

In Q3, both from a clinical perspective, but then at least as importantly from a regulatory perspective.

Yeah.

Sure I'll start and then you're going to chime in but.

We're aiming to do is to show the breadth of Maverick <unk> potential benefit in patients with chronic <unk> leukemia.

Those buckets as you mentioned are there any pathic cyclic and then congenital and I think at this point all we can share is we're certainly feeling confident that we'll be able to share data across each of the three buckets in Q3 against the patient flow of numbers is always a bit ad hoc or some of these patients are sick with neutropenia for example, if a bit challenging.

Window to explore the dragging that patient.

But we're really encouraged about what where are you seeing and maybe I'll turn it over to Diego to add.

Yes, I would just add that.

I would simply add that we already tested, in the context of the Phase II trial, different, mutations, and they all respond.

We are.

Reassured by the strong and finished at all this site.

So we're very confident between the in vitro data and our own in vivo data, and in fact, we have enrolled a breadth of mutations in the Phase III trial.

A number of potential patients and number of patients who have.

Consented as well as the patients dosed.

The change in the conditions of the pandemic is making this study and move forward. So we look forward to being able to play some data in Q3 as Paul said that when you gave us a good SaaS how other potential across all these diverse indications we think currently no therapy.

The bottom line is we believe it will work across all mutations, and in fact, because, of our chronic neutropenia, we know that it also works on the wild type. So it's a big promise to have, for the first time, a normal, well-tolerated therapy to, treat people who are at risk of serious infections from chronic neutropenia.

Fantastic.

Okay. Thanks, that's helpful and then maybe for Adam.

I appreciate the cash guidance into Q4 can you remind us the okay.

The status of the <unk>.

Covenants on.

You bet and.

That's great.

Kind of where where that is.

Sure. Thanks, Mark that we have.

At a minimum cash covenant.

That test kicks in on September one.

We have reached an amendment, where if we raise at this point another $27 million by June 30th that minimum cash number will be fixed at $30 million until we get to when data wherewith positive when data will be $20 million.

If we do not raise that capital before June 30, net test will simply be a six month cash burn test so there'll be a prior one month burn and six.

And that we would need to keep in terms of cash on the balance sheet at that point in time to.

To cover the test.

So that's a little bit around the company.

Yes.

Very helpful. Thank you.

And our next question on line.

Tani from B Riley Securities you.

You may begin.

Yes.

Hi, Good morning, everybody. This is Brian .

Wood on for Myles, Montana really appreciate you, taking our calls and congratulations on all of the.

The investments that you've made a couple of questions from US I was wondering if you could first.

Could you talk to how your view there.

Wm landscape in light of experience of our targeted therapies.

And where you may think.

Our third quarter readout has an opportunity to improve perception and as it relates to both safety and efficacy.

So one last question on the Waldenstrom's indication, when data comes out in the second, half, what are the next steps in the sense, you know, would you… Do you want to listen to another Encore conference?

Sure I'll start.

Press 1 for yes or 2 for no.

Hello?

And again I think it was <unk> that you are asking about just to confirm.

Can you repeat that?

Yes, correct.

Okay, I'm not sure what that… Sorry for the interruption.

Yes.

Guidance is that we'll be sharing data second half of this year and in terms of the competitive landscape of course there is.

Yeah.

A lot of a number of <unk>.

PTK inhibitors, given the validation of the target that are moving forward in the pipeline and certainly their tier two or three approved I think again, we continue to see the importance of adding a <unk> four antagonist across any of those <unk> inhibitors with respect to patients with <unk> mutations.

Okay.

Initially and certainly we see broader applicability beyond that we just presented some really exciting data at ACR, which shows the breadth of activity of our drug not only in <unk>, but of course the single non U.

And then in terms of safety, you know, our drug has got a very positive or benign safety profile, which makes it more amenable to partner with some of these tougher treatments.

<unk> thousand now.

And then in terms of safety our drug is very.

Very positive or benign safety profile, which makes it more amenable to partner with some of these tougher treatments. So youll Ralph therapeutic window in combination seem to say reasonably wide when we add drugs in combination again favoring our approach versus what we're seeing with other combination that CAGR would you like to chime in and then iron as well.

So, the overall therapeutic window in combination seems to stay reasonably wide when we add drugs, in combination, again, favoring our approach versus what we're seeing with other combinations.

But, Diego, would you like to chime in?

And then Art, as well.

Yeah, I would add also that we believe myelodic CXCR4 is quite differentiated from some of the, other drugs being combined in the sense that, for example, venetoclax has a lot of neutropenia.

Yes.

Also that we believe in our except for this quite differentiated from some of the other drugs being combined in the sense that for example, <unk> has a lot of neutropenia.

Xanagrutinib also showed a lot of neutropenia. You see it with ibrutinib.

The channel route to enable also showed a lot of neutropenia youll seen with Ibrutinib, we actually have that drove that treats neutropenia. So in the context of having re scoping peg Xiaomi is combination treatments ultimately the safety of the combination will be comparing portend for the chronic treatment on <unk>.

So we are quite excited about the potential of <unk> to be competitive.

A candidate in that space.

We actually have a, drug that treats neutropenia.

Maybe I can add a little bit starting from the neutropenia site. There's reports already in the literature that talks about it prudently behind the burden of the calibers.

Actually showing neutropenia and Lymphocytopenia and then the corresponding increase in infection since grade three through five SaaS and <unk> and so we certainly are drug that we've measured maverick support in combination with <unk> in our <unk>.

So, in the context of having risk of infection, these combination treatments, ultimately, the safety of the combination will become very important for a chronic treatment on indolent lymphomas.

So, we are quite excited about the potential of myelodic CXCR4 to be a competitive candidate in that space.

Phase one b trial across the patients and we could actually see increases in all of these leukocyte. So we actually do.

Believe that this is going to be a benefit with respect to safety and particularly the infection safety related to neutropenia lymphocytopenia.

And then second just related to the data that we presented at ACR and as Paul mentioned, there, where we were working with a single view and wild type.

CX tier four and these are my TVA fell 265, PV patient cells and they are what we found is that.

All of the agents that we look copies of the PTK antagonist that are out there. So we're prudent with two commercial ones and then <unk> 305 from Lilly and the Merck <unk>.

Maybe I can add a little bit starting from the neutropenia side. There's reports already in the literature that talks about a Daniel Brunib, a Kal Brunib, actually showing neutropenia and lymphocytopenia, and then the corresponding increase in infections is grade 3 through 5 SAEs and AEs.

And so we certainly, our drug, we've measured Maverix IV in combination with the Brunib, in our Wellness Trends Phase 1b trial across the patients, and we can actually see increases in all of these leukocytes. And so we actually do believe that this is going to be a benefit with respect to safety, and particularly the infection safety related to neutropenia and lymphocytopenia.

And then second, it's related to the data that we presented at AACR, and as Paula mentioned, there what we were working with was a single mutant wild type of CXCR4, and these are MITE ADA L265P mutation cells, and there what we found is that all of the agents that we looked, at, these are the BTK antagonists that are out there, so Brunib and Daniel Brunib, two commercial ones, and then LOXO305 from Lilly and the Merck RQL531.

531, and what we found is that these agents while they work on their own and once you start to involve bone marrow, which is really where CX here for double mutation really starts to play. Then then you see that they become resistant to the therapy the tumor cells to become resistant to the therapy and Mavericks was able to overcome that.

And what we found is that these agents, while they work on their own, and once you start, to involve bone marrow, which is really where CXCR4 double mutation really starts to play, then you see that they become a bit resistant to the therapy, the tumor cells become resistant to the therapy.

And Maverix 4 is able to overcome that, so a lot of opportunities here, it broadens obviously, the combinations that could be used, so it's not just a Brunib with Maverix 4, but it could be any of these other agents, whether they're commercial currently or potentially the ones that are in clinical development.

A lot of opportunities here to broaden.

See the combinations that could be used so its not just a burden it with networks before but it could be any of these other agents whether they are commercial currently or potentially the ones that are in clinical development.

It broadens the opportunity across single mutants, which now have upregulated CXCR4.

What is the opportunity across a single mutants, which now up regulated CXC are four so it's not just about the double mutants and then the work that we're doing right now is broadening out even further to other lymphomas. So I think we have some good breadth of opportunities with <unk> four and the combinations section could be quite a few not just with the program.

So it's not just about the double mutants, and then the work that we're doing right now, is broadening out even further to other lymphomas.

So I think we have some good breadth of opportunities with Maverix 4, and the combinations actually, could be quite a few, not just the Brunib.

Thank you so much.

Thank you so much I really appreciate the extra information there.

We really appreciate the extra information there.

On the Waldenstrom's indication, what's the next step, in the sense, once the data, is presented in the second half, would you be able to go straight into a registrational study, or do you need to do an additional study before you go into registration?

And also, I mean, as you presented, you've got a number of clinical trials providing, data in the second half.

And also I mean as.

As you presented you have got a number of clinical trials, providing data in the.

Second half I was just curious where we might.

So, you know, obviously we're waiting for the data to mature and then have regulatory, discussions.

I was just curious where we might be able to expect to see those data coming out throughout, the year.

We expect to see those data coming out throughout the year.

Yeah, I think we'll provide greater clarity on the different venues that we'll be sharing, data.

Yes, I think we'll provide greater clarity on the different venues that we'll be sharing data.

Certainly with the three different milestones, so the chronic uterpenia milestone, and then, the WIM Phase 3, as well as the Waldenstroms, we have sort of a nice drumbeat of information, and that we'll try to optimize to make sure the community is kind of educated in understanding the data when they roll out.

Finally, with the three different milestones.

<unk> milestone and then the wind phase III as well as the long term and sort of a nice drumbeat of information and that will try to optimize to make sure. The community is kind of educated and understanding the data when they rollout, but stay tuned we'll be able to provide some more clarity in the coming months.

You know, there's really only been two drugs approved in Waldenstrom's historically, so, I think that the answer is stay tuned.

So stay tuned.

Obviously data plus regulatory input then would enable us to plan for the future, and, so we'll be able… That's our milestone for the second half, is to provide that guidance.

We'll be able to provide some more clarity in the coming months.

Awesome.

Awesome. Thank you so much congratulations again.

Thank you so much.

Congratulations again.

I'll leave it there.

Leave it there thanks.

Thanks.

Thank you.

Our next question will come from the line of Swayampakula Ramakhan from HC Wainwright.

Our next question comes from the line of volume swam ocular <unk> from HC Wainwright you may begin.

You may begin.

Thank you.

Yes.

So looking forward to sharing more, Arkay.

This is RK from HC Wainwright.

Thank you this is RK from H C Wainwright.

Thank you.

Good morning, Paula and Adam.

Good morning Paula.

Adam.

Thank you very much for taking all my questions.

So talking about the three buckets of patients under chronic neutropenia, Is the biology the same or similar among the three buckets?

Yes.

So.

Talking about the three buckets of patients.

Chronic and neutral opinion.

<unk>.

It is the biology, the same or similar.

Among the three buckets.

And you stated that you would have data among the three buckets.

And.

Yes.

You stated that you would have data.

The three buckets.

So assuming that the data is similar, is there a preference to go after one of them, or what, is the decision point there?

No.

Assuming that the data is similar.

Is there a preference to go after one of them or.

Workers the decision point there.

Yeah, so I think our rationale is certainly there's other…GCSF is the only approved, treatment.

Yes so.

Our rationale.

Certainly there's other G. CSF is the only approved treatment it has been shown to us.

It has been shown to offer some benefit to those patients with significant tolerability, issues.

Or are some benefit to those patients with significant tolerability issues, and then <unk> antagonist <unk> antagonism as a broad mechanism as well we've seen it to you.

And then CXER-4's antagonism…CXER-4 antagonism is a broad mechanism as well.

We've seen it to elevate white blood cell counts in any subject or patient population, so it has a tremendously broad opportunity to increase neutrophils in any form of neutropenia.

White blood cell counts in any subject our patient population. So it has tremendously broad opportunity to increase <unk> and any form of neutropenia.

You saw that in our ASH data with our idiopathic patients. Those folks have unknown cause of neutropenia, and we found very robust effects.

You saw that in our ash data with our idiopathic patients.

Have a known cause of neutropenia with some very robust effects.

So we've kind of already checked one of the three boxes, and we'll look forward to sharing, more data.

<unk> one of the three boxes, and we look forward to sharing more data, but I think maybe on buy art and a little bit more to talk about the mechanistic rationale for matrix for across these different bucket.

But I think maybe I'll invite Arden a little bit more to talk about the mechanistic rationale, for Maverix-4 across these different buckets.

I appreciate it.

Yeah.

Thank you.

Hi, R.K.

Hi, RK how are you.

How are you?

So maybe I'll just talk a little bit about the overall chronic neutropenia landscape, if you will.

So maybe I'll just talk a little bit about the.

Overall chronic neutropenia landscape, if you will and so we've been maybe start from wind and then also the connection back to wall and so there we were focused really on mutated CX. Therefore, but the work that we have done and what was the particular showed us that actually it's more than just mutates, even wild type <unk> and how that gets.

And so we've been…and maybe start from WIM and then also the connection back to Wallenstrom's.

So there, you know, we were focused really on mutated CXER-4.

But the work that we had done in Wallenstrom's in particular showed us that actually it's, more than just mutated.

It's even wild-type CXER-4 and how that gets upregulated under various scenarios.

Up regulated under various scenarios and then certainly across services work that Diego and presented at Ash in December that across all the different clinical programs that we've been doing we see leukocyte mobilization when in patients treated with <unk> four and we see baseline elevations at least leukocytes across the board.

And then certainly across, and this is work that Diego presented at ASH in December, that, across all the different clinical programs that we've been doing, we see leukocyte mobilization in patients treated with Maverix-4, and we see baseline elevations of these leukocytes across the board.

All right.

So this is actually pretty promising because it actually says it's more than just mutated, CXER-4, and it's more than just the specific two diseases that we've been talking about.

So this is actually pretty promising because it actually says it's more than just mutated CXC are for and it's more than just the specific two diseases that we've been talking about so now with the <unk>.

So now it's a broader market expansion and opportunity across other patients now with, chronic utopia.

Broader market expansion and opportunity across other patients not with chronic neutropenia and what we're focused on is the potential of Maverick sport.

And what we're focused on is the potential of Maverix-4 to raise the neutrophil levels, in those patients.

<unk> neutrophil levels in those patients and ended the simplest way is just take the patients get their leverage before and see if they respond.

And the simplest way is just take the patient and give them Maverix-4 and see if they respond, for us in vitro to be able to assess pathogenic measures.

In vitro to be able to assess pathogenic measures. So from the data the Diego presented in December at Ash.

And so from the data that Diego presented in December at ASH, we're seeing that these, patients do respond.

Being that these patients do respond so clearly there is a <unk> year for our component and what we've always talked about is the <unk> four and its natural law against <unk> 12 at the <unk>.

So clearly, there's a CXER-4 component. And what we've always talked about is that CXER-4 and its natural ligand, CXER-12, at, the heart, they're master regulator in terms of granulopoiesis and amyotopoiesis in general.

Heart Theyre master regulator in terms of granular polices and amount of leases in general So thats, a nice connection and it's really all about CX three or four for us.

So that's a nice connection, and it's really all about CXER-4 for us.

Fantastic.

Thank you very much for that.

Fantastic. Thank you very much for that.

And then on the VIMS syndrome, with the data coming out in fourth quarter, so at the same, time, you know, you folks were identifying new mutations within the CXER-4.

And then on the on the Lynch syndrome, with the data coming out in fourth quarter.

So.

At the same time.

You folks were identifying new mutations within the <unk> four.

So when you present the data or when you start to analyze the data from fourth quarter onwards, are you going to go back and look at individual...

So when you present, the data our renewal start to analyze the data from fourth quarter onwards.

Are you going to go back and look at individual.

Individually, are you going to be looking at the mutations just to understand how broad, Maverick CXER-4 can be working on?

Individually youre going to be looking at the mutations just to understand how broad.

<unk> can be working on.

And do you think there would be any mutations where Maverick CXER-4 will not be able to, interact with or not able to, you know, get the benefit, you know, some of these patients?

Do you think there would be any mutations where maverix will or will not be able to interact with are not able to.

No.

Again, the benefit some of those patients I'm just trying to understand how broad you can use to maverix before.

I'm just trying to understand how broad you can use Maverick CXER-4 in the VIMS.

Okay.

Yes.

That's a great question.

Syndrome.

Maybe I could just share the work that we've been doing in what we call the BUSs, which, are really a number of CX04 mutations. So at this point, we've analyzed probably close to about 70 mutations and they really, cover expanded different parts of the CX04 receptor.

Okay. That's a great question and maybe I could just share the work that we've been doing in what we call the <unk>, which are really a <unk>.

<unk> CX for mutation. So at this point, we've analyzed probably close to about 70.

Mutations and they can really cover span the different parts of the <unk> receptor and what we find is that.

And what we find is that most of them are pathogenic and then we know that Maverix IV, binds to all of the CX04 mutants that we've tested so far, or at least blocks the binding of CX012 to all mutants that we've tested so far.

That most of them are pathogenic and then we know that Mavericks for buying two or three or four minutes that we've tested so far and so or at least blocks. The binding of <unk> hundred 12 homes that we've tested so far so we believe that it's going to be effective in any one of the 600, <unk> mutations and that should be irrespective of mutation.

So we believe that it's going to be effective in any one of the CX04 mutations and that, should be irrespective of the mutation.

Yeah.

Yes.

And I would simply add that we already tested in the context of the Phase II trial different, mutations and they all respond.

Okay.

Already tested in the context of the.

The phase II trials defend mutations on the alloy sponsor.

So we're very confident between the in vitro data and our own in vivo data and, in fact, we have enrolled a breadth of mutations in the Phase III trial.

So we're very confident between the in vitro data on our own in vivo data and in fact, we haven't rollout breath, hoping of patients in the phase III.

The bottom line is we believe it will work across all mutations and, in fact, because, of our chronic neutropenia, we know that it also works on the wild type.

The bottom line is we believe it will work across all <unk> and <unk>.

Patients and in fact, because of our coding neutropenia, we know that it also works on the wild type So it's a big promise for.

So it's a big promise to have, for the first time, a normal, well-tolerated therapy to, treat people who are at risk of serious infections from chronic neutropenia.

For the first time on all of them well tolerated therapy to treat people who are at risk of serious infections from chronic neutropenia.

Fantastic.

That's great.

So one last question on the Waldenstrom's indication.

Fantastic that's great one last question.

<unk>.

On the oil and storms.

Indication when data comes out.

When data comes out in the second half, what are the next steps in the sense, you know, would you...

In the second half.

What are the next steps in defense.

Would you like to listen to an Encore Conference press, one for yes, or two from now.

If you want to listen to another ENCOR conference, press one for yes or two for no.

Hello?

Can you repeat that?

Hello.

Can you repeat that RK I'm not sure what that was.

Okay, I'm not sure what that...

Sorry for the interruption.

Okay.

Yeah.

Okay.

On the volumes from syndication whats the next step in the sense. Once the data is presented in the second half would you be able to go straight into a registrational study or do you need to do an additional study before you go into Registrational.

On the Waldenstrom's indication, what's the next step in the sense once the data is presented, in the second half, would you be able to go straight into a registrational study or do you need to do an additional study before you go into registration?

So, you know, obviously we're waiting for the data to mature and then have regulatory, discussions.

So obviously, we're waiting for the data to mature and then have regulatory discussion.

You know, there's really only been two drugs approved in Waldenstrom's historically.

It's really only been two drugs approved and Robin strong historically, so I think that the answer is stay tuned obviously data plus regulatory input than would enable us to plan for the future and.

So I think that the answer is stay tuned.

Obviously data plus regulatory input then would enable us to plan for the future. And so we'll be able...

So we'll be able debt that's our milestone for the second half to provide that guidance.

That's our milestone for the second half, to provide that guidance.

So looking forward to sharing more, RK.

Looking forward to sharing more RK.

Thank you. Thank you very much for taking all my questions I appreciate it. Thank you.

Thank you.

Thank you very much for taking all my questions.

And once again, as a reminder, that's star 1 for questions, star 1.

And once again as a reminder, that star one for a question star one.

We're going to find out Trevor Allred from Oppenheimer.

Your line of Trevor I'll read from Oppenheimer, you may begin.

You may begin.

Hey, good morning.

I appreciate it.

Thank you.

Hey, good morning, I just wanted to ask if you had any conversations with literally a merck about.

I just want to ask if you had any conversations with Lily or Merck about combination potential, and if you have any expectations for how you might advance that combo.

The combination potential and any expectations for how you might advance that combo.

Also wanted to ask if you see any potential role for MAV and activated PI3K delta syndrome.

Also wanted to ask if you're seeing a potential role for <unk> and activate <unk> K Delta syndrome.

So Adam will take the BD question.

So Adam I'll take the BD question, yes. Thanks for the question, we won't comment on specific names folks, who we've been talking to but certainly work.

Yeah, thanks for the question.

We won't comment on specific names, folks who we've been talking to, but certainly we're exploring different alternatives and discussions as it relates to getting the most value from our pursuits and straightening our balance sheet in both oncology and in general.

Exploring different alternatives and discussions as it relates to getting the most value from our pursuits.

Our balance sheet.

In oncology and in general, but well circle back when appropriate with any specifics.

But we'll circle back when appropriate with any specifics in the future.

In the future.

Yeah, and then with respect to PI3K delta, certainly the signaling pathway overlaps with CXCR4, so there's a mechanistic rationale to support the use of Maverix IV in those patients as well.

Yeah, and then with respect to <unk> K Delta.

Certainly the pathway signaling pathway overlap with <unk>. So there is a mechanistic rationale to support Maverick for MF patients as well, we've not yet tested specifically in any patients, but it's something that we're considering refute your studies.

We've not yet tested specifically any patients, but it's something that we're considering for future studies.

Okay, great.

Okay, great. Thanks.

Thanks.

And once again, as a reminder, that's star one for questions, star one.

And our next question comes from the line of Zack <unk>.

From Roth capital you may begin.

And our next question comes from Zach Bajala from Roth Capital.

You may begin.

Good morning.

Thanks for taking our questions.

Good morning, Thanks for taking our questions I just have three quick ones here.

I just have three quick ones here.

This is about the Walton from data I know at that time at that last update.

The first is about the Lodgenstrom data.

You had very strong responses in relapsed refractory patients but.

I know at the time of the last update we had really strong responses in the relapsed refractory patients, but a little less in the frontline setting.

Lastly in a frontline setting. So I was just wondering with enrollment now complete I would you expect more patients.

So I was just wondering with enrollment now complete, I would expect more patients or more relapsed refractory patients versus frontline patients.

Relapsed refractory patients versus frontline patients and then also regarding the 600 milligram dose and that started to be used.

And then also regarding the 600 milligram dose that had started to be used, how should we think about the potential for that dose to kind of change the efficacy profile that we've seen for the program?

How should we think about the potential for that dose dependent change the efficacy profile that we've seen for that program and I have a couple follow ups.

And I have a couple of follow-ups.

Yeah, no, thank you for the question.

We have a line of Trevor Allred from Oppenheimer.

Yes. Thank you for the question.

We believe the potential for Maverix IV in combination with the VTK inhibitors is broad.

We believe the potential for an hour except for in combination with <unk> inhibitors is broad we chose to focus first on CX double Nathan because they have the highest unmet date.

We chose to focus first on double mutants because they have the highest unmet needs.

We believe that the drug can actually be a really good add-on for either frontline or relapsed refractory, and we have enrolled both in our trial, and we will report on that later in the year.

We.

Believe that the drug.

Can actually be a really good add on for either frontline or relapsed refractory and we have enrolled both in our trial and we will report on that later in the year.

I mean, as I mentioned earlier, in vitro, we are really happy with the cellular models that show efficacy, even in wild-type CXCL4 in the context of modeling a bone marrow niche.

I mean.

Sure.

In retail we have.

Really happy with.

Cellular models that show efficacy even in <unk> four in the context of modeling of bone marrow niche. So this speaks to the broad potential of <unk> <unk> for any retail across tumors.

So this speaks to the broad potential of CXCL4 inhibition across tumors that are based in the bone marrow.

<unk> based in the bone marrow, so what will come next.

So what will come next, Paul already mentioned, will focus on Walden's from double mutants, but we believe the potential is much broader.

Paul already mentioned will focus on Walden from the oil near terms, but we believe the potential is much broader.

Thanks, Diego.

Thank you Diego and then the follow up here is just about the limber program you're on track to have your <unk>, which is a big deal as Theyre looking at the way. The stock is Luke I was wondering if you could just kind of again highlight the the commercial potential talk about.

And then the follow-up here is just about the LIM program.

You're on track, to have your NDA submitted, which is a big deal.

So, looking at the way the stock is performing, wondering if you could just kind of, again, highlight the commercial potential, talk about, you know, some of the steps that you've taken.

Some of the stuff that you've taken and I know you have had in commercial but just kind of wonder if you can just kind of speak to.

I know you have someone heading commercial.

So, just kind of wondering if you can just kind of speak to that for a little bit, and I have the last one here as well.

So that's a little bit and then I have a last minute demand.

Sure.

So, in terms of the potential for Maverix IV and LIM, you know, we continue to guide that there's at least about 1,000 genetically, confirmed or confirmed LIM patients in the U.S., and possibly much more than that in terms of the underdiagnosed or undiagnosed.

And we're making exceptional progress towards, you know, building that patient-based education awareness and getting ready for supporting a positive launch should a Maverix IV be approved.

For.

Supporting our positive launch Maverix tour be approved.

Our new VP of U.S. Commercial is exceptionally strong and experienced in this area.

New VP of U S. Commercial is exceptionally strong and experienced in this area. We've got a great team on the ground already and I think we're really ramping up for a successful.

We've got a great team on the ground already, and I think we're really revving up for a successful, you know, an ideal launch post our Phase III milestone this year.

So, stay tuned on more details, but we're excited about already the breadth of diversity of patients, as Art mentioned, over 70 genetic mutants, and then also the diversity of the products, potentially even in chronic neutropenia beyond WIM.

And then also the diversity of the products potentially even in chronic <unk> beyond <unk>.

Thanks, Paula and I didn't know if you can say much about this but this kind of <unk>.

Thanks, Paula, and I don't know if you can say much about this, but this kind of segues based on what you just ended with regarding the broad commercial potential.

Based on what you just ended with regarding the commercial potential I think you guys have highlighted about 60000 patients.

I think you guys have highlighted about 60,000 patients across the different diseases that you're looking at.

Ross.

The different diseases that Youre looking at you also going to have pretty much strong proof of concept data across all three indications by the end of year and mistaking you're kind of pushing to even flatten this out tomorrow.

You're also going to have pretty much strong proof-of-concept data across all three indications by the end of the year, and we've seen you kind of pushing to even broaden this out some more.

Clearly I think that there should be some partnership interests and so is that something that you guys are.

So, clearly, I think that there should be, you know, some partnership interest, and so is that something that you guys are exploring, you know, perhaps not just for one indication but across the board as it relates to Maverix IV?

Laurie, perhaps not just one indication but across the board as it relates to <unk> and then you can maybe comment on how youre thinking about maybe.

And then you can maybe comment on how you're thinking about maybe, you know, reaching, X U.S. opportunities.

Sure.

So, I mean, the world is a big place.

Leaching ex U S opportunities.

Sure So I mean.

The World is a big place.

We obviously have home field advantage in the U.S., and we have a drug that appears, to work in WIM, and it's got some amazing sort of momentum behind it with these other indications, so there's a lot of opportunity for patients and for sort of commercial investment and return on that investment.

Obviously, you haven't homefield advantage in the U S and we Havent drag that appears to work in women at that some amazing.

Sort of momentum behind it with these other indications so theres a lot of opportunity for patients and for <unk>.

Sort of commercial investment and return on that investment.

So, we haven't, you know, we won't be able to share more of how we're thinking, but we're certainly believing that in the U.S. we can handle the commercial setting given the build that we're already investing in and the fruits of that build.

Okay.

Be able to share more of how we're thinking but were certainly believing that in the U S. We can handle the commercial setting given the build that we're already investing in and the fruits of that bell, but ex U S is still a part of our strategy that's evolving and certainly there's a lot of companies that create the right structure to support the most patients globally and offer a benefit.

But X U.S. is still a part of our strategy that's evolving, and certainly there's a lot, of companies that create the right structure to support the most patients globally and also benefit the overall investment and return on that that's needed.

Thanks.

And then, again, go back on the program.

Thank you.

Overall investment and return on that that's needed.

Thanks, and then congrats on the progress.

Thank you.

And once again, let's start on for any questions.

And once again Thats star one for any questions for questions.

We'll go on for questions.

And I'm not showing any further questions in the queue.

I'm not showing any further questions in the queue I would like to turn the call back over to Dr. Paula Ragan for any closing remarks.

I'd like to turn the call back over to Dr. Paula Ragan for any closing remarks.

We'd just like to say thank you for everyone for participating in our investor call today, and thanks for the excellent questions.

You may begin.

Well, we'd just like to say thank you for everyone for participating in our investor call today and thanks for the excellent question. If you have any further follow up please reach out to Glen and we'd be happy to dialog offline. Thank you again and have a great day.

Hey, good morning.

I just want to ask if you had any conversations with Lily or Merck about a combination potential, and if you have any expectations for how you might advance that combo.

Also wanted to ask if you see any potential role for MAV and activated PI3K delta syndrome.

If you have any further follow-up, please reach out to Glenn and we'd be happy to dialogue, offline.

So, Adam will take the DD question.

Yeah, thanks for the question.

We won't comment on specific names of folks who we've been talking to, but certainly we're, exploring different alternatives and discussions as it relates to getting the most value from our pursuits and strengthening our balance sheet in both in oncology and in general.

But we'll circle back when appropriate with any specifics in the future.

Yeah, and then with respect to PI3K delta, certainly the pathway, signaling pathway overlaps, with CXR4, so there's a mechanistic rationale to support the use of Maverix4 in those patients as well.

Thank you again and have a great day.

If you have any further follow-up, please reach out to Glenn, and we'd be happy to dialogue offline.

We've not yet tested specifically any patients, but it's something that we're considering, for future studies.

This concludes today's conference call.

Thank you again, and have a great day.

Okay, great, thanks.

Thank you for participating.

This concludes today's conference call.

And our next question comes from Zach Bajela from Roth Capital.

Thank you for participating.

You may begin.

Good morning.

Thanks for taking our questions.

I just have three quick ones here.

This concludes today's conference call. Thank you for participating you may now disconnect everyone have a great.

The first is about the Lodgenstrom data. I know at the time of the last update, you had really strong responses in the relapsed, refractory patients, but a little less in the frontline setting.

So, I was just wondering with enrollment now complete, I would expect more patients, or more relapsed refractory patients versus frontline patients?

And then also regarding the 600 milligram dose that has started to be used, you know, how should we think about the potential for that dose to kind of change the efficacy profile that we've seen for the program?

And I have a couple of follow-ups.

You may now disconnect.

Yeah, and I'll thank you for the question.

Everyone, have a great day.

We believe the potential for Mavrixa 4 in combination with the VTK inhibitors is broad.

We chose to focus first on double mutants because they have the highest unmet needs.

We believe that the drug can actually be a really good add-on for either frontline or, relapsed refractory, and we have enrolled both in our trial, and we will report on that later in the year.

I mean, as I mentioned earlier, in vitro, we are really happy with the cellular models, that show efficacy even in wild-type CXCR4 in the context of modeling a bone marrow niche. So, this speaks to the broad potential of CXCR4 inhibition across tumors that are based, in the bone marrow.

So, what will come next, Paul already mentioned, we'll focus on Walden's from double mutants, but we believe the potential is much broader.

Thanks, Igo.

And then the follow-up here is just about the WIM program.

You're on track to have your MDA submitted, which is a big deal.

So, looking at the way the stock is performing, wondering if you could just kind of, again, highlight the commercial potential, talk about, you know, some of the steps that you've taken.

I know you have someone heading commercial, so just kind of wondering if you can just, kind of speak to that a little bit, and then I have the last one here as well.

Sure.

So, in terms of the potential for Maverix IV and WIM, you know, we continue to guide, that there's at least about 1,000 genetically confirmed or confirmed WIM patients in the U.S. and possibly much more than that in terms of the underdiagnosed or undiagnosed.

And, we're making exceptional progress towards, you know, building that patient-based education awareness and getting ready for supporting a positive launch should a Maverix IV be approved.

Our new VP of U.S. Commercial is exceptionally strong and experienced in this area.

We've, got a great team on the ground already, and I think we're really revving up for a successful, you know, an ideal launch post our Phase III milestone this year.

So, stay tuned on more details, but we're excited about already the breadth of diversity of patients, as Art mentioned, over 70 genetic mutants, and then also the diversity of the products potentially even in chronic atrophemia beyond WIM.

Thanks, Paula.

And I don't know if you can say much about this, but this kind of segues based on what you just ended with regarding the broad commercial potential.

I think you guys have highlighted about 60,000 patients across the different diseases that you're looking at.

You're also going to have pretty much strong proof-of-concept data across all three indications by the end of the year, and we've seen you kind of pushing to even broaden this out some more.

So, clearly, I think that there should be, you know, some partnership interest, and so is that something that you guys are exploring, you know, perhaps not just for one indication, but across the board as it relates to Maverix IV?

And then you can maybe comment on how you're thinking about maybe, you know, reaching ex-U.S. opportunities.

Sure.

So, I mean, the world is a big place, we obviously have home field advantage in the U.S., and we have a drug that appears to work in WIM, and it's got some amazing sort of momentum behind it with these other indications.

So, there's a lot of opportunity for patients and for sort of commercial investment and return on that investment.

So, we haven't, you know, we won't be able to share more of how we're thinking, but we're certainly believing that in the U.S. we can handle the commercial setting, given the bill that we're already investing in and the fruits of that bill.

But ex-U.S. is still a part of our strategy that's evolving, and certainly there's a lot of companies that create the right structure to support the most patients globally and also benefit the overall investment and return on that that's needed.

Thanks.

And then, again, go back on the progress.

Thank you.

And once again, that's a star one for any questions, one one for questions.

And I'm not showing any further questions in the queue.

I'd like to turn the call, back over to Dr. Paula Reagan for any closing remarks.

Well, we'd just like to say thank you for everyone for participating in our investor, call today, and thanks for the excellent questions.

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