Q2 2024 X4 Pharmaceuticals Inc Earnings Call

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Operator: Please stand by; we're about to begin.

Unknown Executive: We stand by; we're about to begin.

Please stand by, we're about to begin.

Operator: Good morning, everyone, and welcome to X4 Pharmaceuticals' 2nd quarter 2024 earnings conference call. At this time, all participants are in a listen-only mode.

Operator: And we're about to begin. Good morning, everyone, and welcome to X4 Pharmaceuticals' second quarter 2024 earnings conference call. At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. You may register to ask a question at any time by pressing star 1 on your telephone keypad. Also, today's call is being recorded, and if you should need any assistance during the call today, please press star 0. It is now my pleasure to introduce your host, Mr. Dan Ferry from LifeSci Advisors. Dan, please go ahead. Thank you, Operator.

Speaker Change: Good morning, everyone, and welcome to X4 Pharmaceuticals second quarter 2024 earnings conference call.

Operator: A question and answer session will follow the formal presentation. You may register to ask a question anytime by pressing star one on your telephone keypad. Also, today's call is being recorded, and if you should need any assistance during the call today, please press star zero.

Speaker Change: At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation.

Speaker Change: You may register to ask a question at any time by pressing star 1 on your telephone keypad. Also, today's call is being recorded, and if you should need any assistance during the call today, please press star 0.

Daniel Ferry: It is now my pleasure to introduce your host, Mr. Dan Ferry, from Lifesci Advisors.

Speaker Change: It is now my pleasure to introduce your host, Mr. Dan Ferry from Lifesci Advisors. Dan, please go ahead. Thank you, Operator. Good morning, everyone. Thank you for joining us today.

Daniel Ferry: Dan, please go ahead.

Daniel Ferry: Thank you, operator. Good morning, everyone. Thank you for joining us today. Presenting on today's call will be Dr. Paula Ragan, X4's President and CEO, and the company's Chief Financial Officer, Adam Mostafa. Following prepared remarks, we will open up the call to your questions, and we'll be joined by Chief Commercial Officer, Mark Baldry; Chief Medical Officer, Dr. Christophe Arbet Engles; Chief Operating Officer, Dr. Mary D. Biasi, Chief Scientific Officer; Dr. Art Tveris; and Jose Huves, Head of Corporate and Patient Affairs.

Dan Ferry: Thank you, Operator, and good morning, everyone. Thank you for joining us today.

Dan Ferry: Presenting on today's call will be Dr. Paula Ragan, X4's President and CEO, and the company's Chief Financial Officer, Adam Mostafa. Following prepared remarks, we will open up the call to your questions and will be joined by Chief Commercial Officer, Mark Baldry. Chief Medical Officer, Dr. Christophe Arbet Engels; Chief Operating Officer, Dr. Mary DiBiase; Chief Scientific Officer, Dr. Art Tavares, and Jose Juvez, Head of Corporate and Patient Affairs As a reminder, on today's call, the company will be making forward-looking statements regarding regulatory and product development and commercialization plans, as well as research activities and financial projections.

Speaker Change: presenting on today's call will be dr paul reagan exforort' president and ceo in the company's chief financial officer adam safa

Unknown Executive: Following prepared remarks, we will open up the call to your questions and will be joined by Chief Commercial Officer, Mark Baldry; Chief Medical Officer, Dr. Christophe Arbet Engels; Chief Operating Officer, Dr. Mary DiBiase; Chief Scientific Officer, Dr. Art Tavares; and Jose Juvez, Head of Corporate and Patient Affairs. I'll now turn it over to Paula Ragan.

Speaker Change: following prepared remarks we will open up the call toyour questionsand will be joined by chief commercial officer mark ballary chief medical officer dr christo arbit angles

Speaker Change: Chief Operating Officer, Dr. Mary DiBiase, Chief Scientific Officer, Dr. Art Tavares, and Jose Juvez, Head of Corporate and Patient Affairs.

Paula Ragan: As a reminder on today's call, the company will be making forward-looking statements regarding regulatory and product development and commercialization plans, as well as research activities and financial projections. These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted.

Speaker Change: As a reminder, on todayís call, the company will be making forward-looking statements regarding regulatory and product development and commercialization plans.

Speaker Change: as well as research activities and financial projections

Dan Ferry: These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted. A description of these risks can be found in X4's most recent filings with the SEC, including this year's Form 10-K, which was filed on March 21, 2024, and the company's Form 10-Q for the second quarter, which is expected to be filed later today. I'll now turn it over to Paula Ragan.

Speaker Change: these statements are subject to risks and uncertainties that may cause actual results to differ from those forecasting

Paula Ragan: A description of these risks can be found in X4's most recent filings with DSEC, including this year's Forum 10-K, which was filed on March 21st, 2024, and in the company's Forum 10-Q for the second quarter, which is expected to be filed later today.

Speaker Change: a description of these risks can be found in xforort's most recent filings with the sec

Speaker Change: including this year's Form 10-K , which was filed on March 21, 2024.

Speaker Change: And in the company's Form 10-Q , for the second quarter,

Speaker Change: which is expected to be filed later today.

Paula Ragan: I'll now turn it over to Paula Ragan.

Paula Ragan: Paula? Thanks so much, Dan, and welcome everyone. I'll just start by saying what an incredible year we've had so far. Just a few weeks ago, we celebrated the 10th anniversary of X4. When we founded a company a decade ago, a small group of us had a vision to advance our lead asset, an orally active CX-4 antagonist called Mavericks before, tells those with rare diseases who have few to no treatment options. As you saw this past April, we were able to realize this vision, receiving US approval of Mavericks before, branded as Zolrendi for the treatment of Wim Syndrome, a rare primary immunodeficiency.

Speaker Change: I'll now turn it over to Paula Ragan.

Paula Ragan: Thanks so much, Dan, and welcome, everyone. I'll just start by saying what an incredible year we've had so far.

Paula Ragan: Thanks so much, Dan, and welcome, everyone. I'll just start by saying what an incredible year we've had so far.

Speaker Change: Paula?

Paula Ragan: Thanks so much, Dan, and welcome, everyone.

Speaker Change: I'll just start by saying, what an incredible year we've had so far.

Paula Ragan: Just a few weeks ago, we celebrated the 10th anniversary of X4. When we founded the company ten years ago, a small group of us had a vision to advance our lead asset, an orally active CXCR4 antagonist called Maverix X4 to help those with rare diseases who have few to no treatment options. Our launch meeting in early May was a great success, where we completed the training and education of our field teams, enabling them to hit the ground running. We got product into our distribution channels very quickly and had our first patients on commercial drugs within just a few weeks of product approval.

Paula Ragan: Just a few weeks ago, we celebrated the 10th anniversary of X4. When we founded the company ten years ago, a small group of us had a vision to advance our lead asset, an orally active CXCR4 antagonist called Maverix X4 to help those with rare diseases who have few to no treatment options. As you saw this past April, we were able to realize this vision, receiving U.S. approval for Maverix X4, branded as Xolremdi, for the treatment of Wim Syndrome, a rare primary immunodeficiency.

Speaker Change: just a few weeks ago we celebrated the tenth anniversary of x four when we founded a company a decade ago a smaller group of us had vision to advance our lead asset an orly active c fehere for antagonist called mavericks before tell those with r diseases who have few to note treatment options

Paula Ragan: Coordination with our specialty pharmacy, Panther Rx Rare, has gone smoothly, and we've already received positive feedback on the patient support services provided through our X4 Connect and Nurse Educator Program. Additionally, our commercial team is fully deployed across the U.S., calling on key hematologists and immunologists. We recently participated in the annual meeting of the Immune Deficiency Foundation, or IDF, where we engaged with physicians, patients, and advocates. And members of our team visited the Jeffrey Modell Foundation in New York just a few weeks ago.

Speaker Change: as you saw this pth april we were able to realize this vision receiving u s approval of maverick before branded as zreny for the treatment of whimsyndrome a rare primary immuor deficiency

Paula Ragan: The U.S. launch of Xolremdi is now underway, and our full commercial team is in place, and Wim patients 12 years and older are now being treated with the only approved therapy targeting the underlying cause of their disease. Sales guidance on Zole Remde will come at a future point, however, today I'd like to highlight our strong launch progress. Overall, we've been very pleased with the launch to date.

Paula Ragan: The US launch of Zolrendi is now underway, and our full commercial team is in place, and when patients 12 years and older are now being treated with the only approved therapy targeting the underlying cause of their disease. Still, guidance on Zolrendi will come at a future point. However, today, I'd like to highlight our strong launch progress. Overall, we've been very pleased with the launch to date. Our launch meeting in early May was a great success, where we completed the training and education of our field teams, enabling them to hit the ground running. We got product into our distribution channel very quickly and had our first patients on commercial drugs within just a few weeks of product approval.

Speaker Change: the euk launch of z remmedy is now underway and our full commercial team is in place and when patients twelve years and older are now being treated with the only approved therapy targeting the underlying cause of their disease

Speaker Change: Sales guidance on Zole Remde will come at a future point, however today I'd like to highlight our strong launch progress.

Paula Ragan: Our launch meeting in early May was a great success, where we completed the training and education of our field teams, enabling them to hit the ground running. We got product into our distribution channels very quickly and had our first patients on commercial drugs within just a few weeks of product approval. Coordination with our specialty pharmacy, Panther Rx Rare, has gone smoothly, and we've already received positive feedback on the patient support services provided through our X4 Connect and Nurse Educator Program.

Speaker Change: Overall, we've been very pleased with the launch to date. Our launch meeting in early May was a great success, where we completed the training and education of our field teams, enabling them to hit the ground running.

Speaker Change: We got product into our distribution channels very quickly and had our first patients on commercial drugs within just a few weeks of product approval.

Paula Ragan: Coordination with our specialty pharmacy, Panther RX Rare, has gone smoothly, and we've already received positive feedback on the patient support services provided to our X4 Connect and Nurse Educator programs. And, very encouragingly, reimbursement decisions have been coming through quickly, but almost all patients currently on therapy are receiving reimbursement via prior authorizations and medical exceptions. Our commercial team is fully deployed across the US, calling on key hematologists and immunologists. To date, there has been limited ability to establish centers of excellence for the wind community. We knew this going in and therefore are continuing to execute on our disease awareness and education campaigns, as well as leveraging our strong relationships with patient advocacy groups.

Speaker Change: Coordination with our specialty pharmacy, Panther Rx Rare, has gone smoothly and we've already received positive feedback on the patient support services provided through our X4 Connect and Nurse Educator programs.

Paula Ragan: And very encouragingly, reimbursement decisions have been coming through quickly, with almost all patients currently on therapy receiving reimbursement via prior authorizations and medical exceptions. Our commercial team is fully deployed across the U.S. calling on key hematologists and immunologists. To date, there has been limited ability to establish centers of excellence for the WIHM community.

Speaker Change: And very encouragingly, reimbursement decisions have been coming through quickly, with almost all patients currently on therapy receiving reimbursement via prior authorizations and medical exceptions.

Speaker Change: Our commercial team is fully deployed across the U.S. calling on key hematologists and immunologists.

Speaker Change: To date, there has been limited ability to establish centers of excellence for the WIHM community. We knew this going in and, therefore, are continuing to execute on our disease awareness and education campaigns, as well as leveraging our strong relationships with patient advocacy groups.

Paula Ragan: We knew this going in and, therefore, are continuing to execute on our disease awareness and education campaigns, as well as leveraging our strong relationships with patient advocacy groups. We recently participated in the annual meeting of the Immune Deficiency Foundation, or IDF, where we engaged with physicians, patients, and advocates. And members of our team visited the Jeffrey Modell Foundation in New York just a few weeks ago. Through all of these efforts, we have become better at engaging with the treating physicians, understanding physician and patient questions, and, most importantly, building a community of interest around Wim Syndrome across the U.S. We also continue to leverage the positive data from our pivotal Phase 3 trial in WIM that were published in April in Blood, the Journal of the American Society of Hematology.

Paula Ragan: We recently participated in the annual meeting of the Immune Deficiency Foundation, or IDF, where we engage with physicians, patients, and advocates, and members of our team visited with the Jeffrey Modell Foundation in New York just a few weeks ago. Through all these efforts, we have become better at engaging with the treating physicians, understanding physician and patient questions, and, most importantly, building a community of interest around WIMS syndrome across the US. We also continue to leverage the positive data from our pivotal Phase 3 trial and WIMM that were published in April in Blood, the Journal of the American Society of Hematology.

Speaker Change: We recently participated in the annual meeting of the Immune Deficiency Foundation, or IDF, where we engage with physicians, patients, and advocates.

Speaker Change: And members of our team visited with the Jeffrey Modell Foundation in New York just a few weeks ago.

Speaker Change: Through all of these efforts, we have become better at engaging with the treating physicians, understanding physician and patient questions, and most importantly, building a community of interest around Wim Syndrome across the U.S.

Speaker Change: We also continue to leverage the positive data from our pivotal Phase III trial in WIM that were published in April in Blood, the Journal of the American Society of Hematology.

Paula Ragan: These and new data from the trial and its open label extension phase were presented at the annual meeting of the Clinical Immunological Society, or CIS, in early May. As we reported at the time, the CIS poster revealed that long-term treatment, with Zoll Remdi, was associated with durable improvements in mutual and lymphocyte counts, as well as reductions in inanalyzed infection rates, and that no new safety signals had been observed during the OLE phase of the trial, with some participants treated for more than two years. We also made tremendous progress this past quarter in our development of Mavericks before for those with chronic uterineia, another rare immunodeficiency.

Paula Ragan: New data from the trial and its open-label extension phase were presented at the annual meeting of the Clinical Immunological Society, or CIS, in early May. As we reported at the time, the CIS poster revealed that longer-term treatment with Zol Remde was associated with durable improvements in neutrophil and lymphocyte counts, as well as reductions in annualized infection rates, and that no new safety signals had been observed during the OLE phase of the trial, with some participants treated for more than two years. We also made tremendous progress this past quarter in our development of Maverick Sephora for those with chronic utrophenia, another rare immunodeficiency.

Speaker Change: these new data from the trial and its open label extension bases were presented at the innual meeting of the clinical immunological society or ci in early may

Paula Ragan: As we reported at the time, the CIS poster revealed that longer-term treatment with Zol Remde was associated with durable improvements in neutrophil and lymphocyte counts, as well as reductions in annualized infection rates, and that no new safety signals had been observed during the OLE phase of the trial, with some participants treated for more than two years. And Mavericks Hormonal Therapy also durably increased ANC in participants with severe CM defined as ANCs less than 500 cells per microliter at baseline, achieving mean ANCs of 800 to 1,000 cells per microliter, which is the ANC range targeted by experts for patients with severe CM.

Speaker Change: As we reported at the time, the CIS poster revealed that longer-term treatment with Zol Remde was associated with durable improvements in neutrophil and lymphocyte counts, as well as reductions in annualized infection rates.

Speaker Change: and that no new safety signals have been served during the oe phase of the trial with some participants treated for more than two years

Speaker Change: We also made tremendous progress this past quarter in our development of Maverix IV for those with chronic utropenia, another rare immunodeficiency.

Paula Ragan: At our investor event in late June, we presented positive interim clinical data from our ongoing phase 2 clinical trial, demonstrating that once daily oral Mavericks before was generally well tolerated and durably increased participants' absolute mutual accounts, or ANC, both as a mild therapy and in combination with stable doses of injectable granulocyte colony stimulating factor, or GPSF, out to as long as six months. Importantly, 100% of the valuable participants quit completed the six-month study as of the mid-May cut-off date achieved target ANC increases of greater than 500 cells per microliter at month 3 and 6 on one daily Mavericks for as a monotherapy and in combination with stable GPSF dosing.

Paula Ragan: At our investor event in late June, we presented positive interim clinical data from our ongoing Phase II clinical trial demonstrating that once-daily oral Mavrix A4 was generally well-tolerated and durably increased participants' absolute neutrophil counts, or ANC, both as monotherapy and in combination with stable doses of Injectable Granulocyte Colony Stimulating Factor, or GCSF, for up to as long as six months. Importantly, 100% of the valuable participants who had completed the six-month study as of the mid-May cutoff date achieved target ANC increases of greater than 500 cells per microliter at months three and six on one SteliMavrix IV as monotherapy and in combination with stable GCSF dosing.

Speaker Change: At our investor event in late June , we presented positive interim clinical data from our ongoing Phase II clinical trial demonstrating that once-daily oral Maverix A4 was generally well-tolerated and durably increased participants' absolute neutrophil counts

Speaker Change: or ANC, both as a monotherapy and in combination with stable doses of Injectable Granulocyte Colony Stimulating Factor, or GCSF, up to as long as six months.

Speaker Change: Importantly, 100% of the valuable participants who had completed the six-month study as of the mid-May cutoff date achieved target ANC increases

Speaker Change: a greater than 500 cells per microliter at months 3 and 6 on one daily Mavericks 4 as a monotherapy and in combination with stable GCSF dosing.

Paula Ragan: In addition, participants on Mavericks for monotherapy achieved mean ANC levels above the lower limit of normal for CN at 3 and 6 months. And Mavericks for monotherapy also durably increased ANC in participants with severe CN, defined as ANC's less than 500 cells per microliter at baseline, achieving mean ANC's of 800 to 1000 cells per microliter, which is the ANC range targeted by experts for patients with severe CN. Feedback on these interim results from the physician community has been positive, and we were pleased to see the durable impact of Mavericks as a monotherapy and in combination with stable dose GCSF.

Paula Ragan: In addition, participants on Maverix IV monotherapy achieved mean ANC levels above the lower limit of normal for CN at 3 and 6 months. And Mavericks Hormonal Therapy also durably increased ANC in participants with severe CM defined as ANCs less than 500 cells per microliter at baseline, achieving mean ANCs of 800 to 1,000 cells per microliter, which is the ANC range targeted by experts for patients with severe CM.

Speaker Change: In addition, participants on Maverix IV monotherapy achieved mean ANC levels above the lower limit of normal for CN at 3 and 6 months.

Speaker Change: And Mavericks for Monotherapy also durably increased AMC in participants with severe CM, defined as AMCs less than 500 cells per microliter at baseline,

Speaker Change: achieving mean a seas of eight hundred to a thousand sol per microleader which was the ac range targeted by experts for patients with severeyearcm

Paula Ragan: Feedback on these interim results from the physician community has been positive, and we were pleased to see the durable impact of Maverix IV as monotherapy and in combination with stable dose GCSF. These interim data support our strong belief that Mavericks Forum may be able to address the needs of CN patients and their physicians. Specifically, the desire for an oral treatment that does not carry the short or long-term dose-related toxicities described with GCSF use. And for an oral treatment that also increases A and C levels and lessens the risk and severity of infections, whether used on its own or in combination with GCSS.

Speaker Change: Feedback on these interim results from the physician community has been positive and we were pleased to see the durable impact of MaverickScore as a monotherapy and in combination with stable dose GCSF.

Paula Ragan: These interim data support are strong beliefs that Mavericks for may be able to address the needs of the CN patients and their physicians. Specifically, the desire for an oral treatment that does not carry short or long-term dose-related toxicities described with GCSF use, and for an oral treatment that also increases ANC levels and lessens the risk and severity of infections, whether used on its own or in combination with GCSF. We're now expecting to present the full data from the Phase II CN trial in November, ahead of the American Society of Hematology or ASH meeting. This data set will include results from the group of participants receiving Mavericks before and those who are allowed to dose adjust GCSF from baseline.

Speaker Change: These interim data support our strong belief that Mavericks Forum may be able to address the needs of the CN patients and their physicians.

Speaker Change: Specifically, the desire for an oral treatment that does not carry short- or long-term dose-related toxicities described with GCSF use.

Paula Ragan: and for an oral treatment that also increases ANC levels and lessens the risk and severity of infections, whether used on its own or in combination with GCSF.

Paula Ragan: We're now expecting to present the full data from the Phase 2 CN trial in November, ahead of the American Society of Hematology, or ASH, meeting. We've been asked what success looks like for these data that will be presented in November. The good news is we already know what success looks like from our WIM Phase 3 study and subsequent approval in the U.S. From the interim CN Phase II results we shared in June, we saw again that Maverix IV as a monotherapy, and in combination with GCSF, can increase ANCs to levels expected to reduce infection.

Paula Ragan: We're now expecting to present the full data from the Phase 2 CN trial in November, ahead of the American Society of Hematology, or ASH, meeting. This data set will include results from the group of participants receiving Maverick X4 and those who are allowed to dose-adjust GCSF from baseline. We've been asked what success looks like for these data that will be presented in November. The good news is we already know what success looks like from our WIM Phase 3 study and subsequent approval in the U.S. In that 52-week study, Mavericks meaningfully increased neutrophils and lymphocytes over a sustained period, reducing infection rate, duration, and severity.

Speaker Change: We're now expecting to present the full data from the Phase 2 CN trial in November , ahead of the American Society of Hematology, or ASH, meeting.

Paula Ragan: This data set will include results from the group of participants receiving Maverick X4 and those who are allowed to dose adjust GCSF from baseline.

Paula Ragan: We've been asked what success looks like for these data that will be presented in November. The good news is we already know what success looks like from our WIM Phase III study and subsequent approval in the U.S. In that 52-week study, Mavericks for meaningfully increased neutrophils and lymphocytes over a sustained period, reducing infection rate, duration, and severity. From the interim CNC's tear results we shared in June, we saw again that Mavericks for as a monotherapy and in combination with GCSF can increase ANC's to levels expected to reduce infections. In November, we'll be looking to replicate what we saw in June across all participants in the Mavericks for a monotherapy and Mavericks for those GCSF groups who completed the six months of the trial.

Paula Ragan: We've been asked what success looks like for these data that will be presented in November . The good news is we already know what success looks like from our WIM Phase 3 study and subsequent approval in the U.S.

Speaker Change: In that 52-week study, Mavericks form meaningfully increased neutrophils and lymphocytes over a sustained period, reducing infection rate, duration, and severity.

Paula Ragan: From the interim CN Phase II results we shared in June, we saw again that Maverix IV as a monotherapy and in combination with GCSF can increase ANCs to levels expected to reduce infection. In November, we'll be looking to replicate what we saw in June across all participants in the Mavericks for Monotherapy and MAV plus stable dose GCSF groups who completed the 6 months of the trial. From the third group, we'll be looking for data that supports physicians' ability to reduce GCSF dosing while a Maverick monitors and maintains patients' ANCs at clinically meaningful levels.

Speaker Change: from the interim sand's two results we shared in june we saw again that maverick for as a monootherapy and in combination with gfft can increase aces to levels expected to reduce infections

Speaker Change: In November , we'll be looking to replicate what we saw in June across all participants in the Mavericks for Monotherapy and MAV plus Stable Dose GCSF groups who completed the sixth month of the trial.

Paula Ragan: From the third group, we'll be looking for data that supports physicians' ability to reduce GCSF dosing while mavericks for and maintain patients' ANCs at clinically meaningful levels. Finally, we'll be also looking for continued tolerability and safety of Mavericks, for as seen in all of our other clinical programs to date. In June, we also announced the initiation of our global pivotal Phase III clinical trial, branded the Forward study, evaluating the efficacy, safety, and tolerability of oral one-stay-ly Mavericks for with and without stable doses of GCSF and people with congenital acquired primary autoimmune or idiopathic CN for experiencing recurrent and or serious infections.

Speaker Change: From the third group, we'll be looking for data that supports physicians' ability to reduce GCSF dosing while on Maverick support and maintain patients' ANCs at clinically meaningful levels.

Paula Ragan: Finally, we'll also be looking for continued tolerability and safety of Maverix IV, as seen in all of our other clinical programs to date. As we focus our efforts on the FORWARD-CN trial, we are also looking to maximize the global opportunity for Wim Syndrome. We expect an MAA submission to the European Medicines Agency for Mavericks 4 and Wim Syndrome by early 2025, while we also explore additional potential opportunities in geographies where we may be able to efficiently leverage our U.S. FDA approval. With that, I'll now turn it over to our CFO, Adam Mostafa, to review the second quarter financials. Adam?

Paula Ragan: Finally, we'll also be looking for continued tolerability and safety of Maverix IV, as seen in all of our other clinical programs to date. In June, we also announced the initiation of our Global Pivotal Phase III Clinical Trial, branded the FORWARD Study, evaluating the efficacy, safety, and tolerability of oral once-daily Maverix IV with and without stable doses of GCSF in people with congenital acquired primary autoimmune or idiopathic CN who are experiencing recurrent and or serious infections.

finally we'll be also looking for continued tolerability and safety of meverick for have seen in all of our other clinical programs to date

Speaker Change: In June , we also announced the initiation of our global Pivotal Phase III clinical trial.

Speaker Change: Branded the FORWARD Study, evaluating the efficacy, safety, and tolerability of oral once-daily Maverix IV with and without stable doses of GCSF in people with congenital

Speaker Change: acquired primary autoimmune or idiopathic CN who are experiencing recurrent and or serious infections.

Paula Ragan: The 52-week forward trial is a randomized, double-blinded, placebo-controlled, multi-center study aiming to enroll 150 participants. The clinical operations team is in full swing opening up sites, and we are on track to fully enroll the trial in mid 2025. We believe that the interim phase two CND that presented to date strongly supports our decision to initiate the phase three trial as well as design. Importantly, we believe that the interim results de-risked the phase three trial given that the ANC increases seen in CN patients to date mirror those and are successfully completed four win phase three trial.

Paula Ragan: The 52-week Forward Trial is a randomized, double-blinded, placebo-controlled, multi-center study aiming to enroll 150 participants. The clinical operations team is in full swing, opening up sites, and we are on track to fully enroll the trial in mid-2025. We believe that the interim Phase 2 CN data presented to date strongly support our decision to initiate the Phase 3 trial as well as its design. Importantly, we believe that the interim results de-risk the Phase 3 trial given that the ANC increases seen in CN patients to date mirror those in our successfully completed 4-WIM Phase 3 trial, where similar levels of ANC increases led to clinical benefit by reducing the frequency, duration, and severity of infection.

Adam Mostafa: The 52-week Forward Trial is a randomized, double-blinded, placebo-controlled, multi-center study aiming to enroll 150 participants.

Speaker Change: The clinical operations team is in full swing, opening up sites, and we are on track to fully enroll the trial in mid-2025.

Speaker Change: we believe that the interim phase i cna that presented to date strongly support our decision to initiate the phasree trial as well as this design

Adam Mostafa: Importantly, we believe that the interim results de-risk the Phase 3 trial, given that the ANC increases seen in CN patients to date mirror those in our successfully completed 4-WIM Phase 3 trial.

Paula Ragan: We're similar levels of ANC increases led to clinical benefit by reducing the frequency, duration, and severity. As we focus our efforts on the forward CN trial, we are also looking to maximize the global opportunity in WIM syndrome. We expect an MAA submission to the European Medicines Agency for Mavericks for WIM syndrome by early 2025, while we also explore additional potential opportunities and geographies where we may be able to efficiently leverage our US FDA approval.

Speaker Change: where similar levels of anti increases led a clinical benefit by reducing the frequency duration and severity of infections

Paula Ragan: As we focus our efforts on the FORWARD-CN trial, we are also looking to maximize the global opportunity for Wim Syndrome. We expect an MAA submission to the European Medicines Agency for Mavericks 4 and Wim Syndrome by early 2025, while we also explore additional potential opportunities in geographies where we may be able to efficiently leverage our U.S. FDA approval. With that, I'll now turn it over to our CFO, Adam Mostafa, to review the second quarter financials. Adam?

Speaker Change: as we focus our efforts on the forward cand trial we are also looking to maximize the global opportunity in wind and drome

Paula Ragan: We expect an MAA submission to the European Medicines Agency for Mavericks 4 and Wim Syndrome by early 2025, while we also explore additional potential opportunities and geographies where we may be able to efficiently leverage our U.S. FDA approval.

Adam Mostafa: With that, I'll now turn it over to our CFO, Adam Mostafa, to review the second quarter financials.

Paula Ragan: With that, I'll now turn it over to our CFO , Adam Mostafa, to review the second quarter financials.

Adam Mostafa: Adam? Thanks, Paula, and thanks to all of you for being on the call of us today. As we had previously discussed, upon FDA approval of ZORMD, we were awarded a priority review voucher, or PRV, based on Mavericks' course rare pediatric disease designation. We monetized this PRV very shortly after receipt, and in May, we recognized a gain on the sale to a third party for $105 million in cash. So, as of the end of the second quarter, X4 had $169.5 million in cash, cash equivalence, restricted cash, and short-term marketable securities. We believe these funds are sufficient to support our company operations into late 2025.

Adam Mostafa: Thanks, Paula, and thanks to all of you for being on the call with us. As we had previously discussed, upon FDA approval of ZolRMD, we were awarded a Priority Review Voucher, or PRV, based on MedRx-4's Rare Pediatric Disease designation. We would like to note that there was a small amount of inventory stocking at our specialty pharmacy during the quarter, and this is reflected in the sales numbers. R&D expenses for the second quarter of 2024 included $1.2 million of non-cash expenses.

Adam Mostafa: Thanks, Paula, and thanks to all of you for being on the call with us. As we had previously discussed, upon FDA approval of ZolRMD, we were awarded a Priority Review Voucher, or PRV, based on MedRx-4's Rare Pediatric Disease designation. We monetized this PRV very shortly after receipt, and in May, we recognized the gain on the sale to a third party for $105 million in cash. As of the end of the second quarter, X4 had $169.5 million in cash, cash equivalents, restricted cash, and short-term marketable security. We believe these funds are sufficient to support our company operations into late 2025. However, I want to note that this projected runway does not include potential futures or MD revenue.

Adam Mostafa: Thanks, Paula, and thanks to all of you for being on the call with us today.

Speaker Change: as we had previously discussed upon fd approval salt mdy we were awarded a priority review vouch or prv based on me exforres rare pediatric deeu ddesignation

Speaker Change: we monetize this prv very shortly after receipt and in may we recogne thegain on the sale to a third party for one five million dollars in cash

Speaker Change: so as of the end of the second quarter x four had one hundred and sixty nine point five million dollars in cash cash equivalents restricted cash and short-term marketable securities

Adam Mostafa: We believe these funds are sufficient to support our company operations into late 2025. I want to note that this projected runway does not include potential futures or MD revenue.

Adam Mostafa: We want to note that this projected runway does not include potential futures ZORMD revenue. For the three months and mid-June 30th, we reported revenues of $0.6 million and costs of revenue of $0.3 million, related to the sale of ZORMD. This cost of revenue includes approximately $0.2 million of licensed costs, including sales-based royalties and operational milestones capitalized as an intangible asset, and amortized over the life of the underlying intellectual property. We would like to note that there was a small amount of inventory stocking at our specialty pharmacy during the quarter, and this is reflected in the sales number.

Adam Mostafa: For the three months ended June 30th, we reported revenues of 0.6 million dollars and cost of revenue of 0.3 million dollars related to the sale of Zorandi. This cost of revenue includes approximately $0.2 million of license costs, including sales-based royalties and operational milestones capitalized as an intangible asset and amortized over the life of the underlying intellectual property. We would like to note that there was a small amount of inventory stocking at our specialty pharmacy during the quarter, and this is reflected in the sales numbers.

Adam Mostafa: For the three months ended June 30th, we reported revenues of $0.6 million and costs of revenue of $0.3 million related to the sale of ZorMD.

Adam Mostafa: This cost of revenue includes approximately $0.2 million of license costs, including sales-based royalties and operational milestones capitalized as an intangible asset and amortized over the life of the underlying intellectual property.

Adam Mostafa: We would like to note that there was a small amount of inventory stocking at our specialty pharmacy during the quarter, and this is reflected in the sales number.

Adam Mostafa: Research and development expenses were $20.9 million for the second quarter, compared to $15.6 million for the comparable period in 2023. R&D expenses for the second quarter of 2024 included $1.2 million in non-cash expense. Selling, general and administrative expenses was $13.3 million for the second quarter of 2024. This compares to $10.2 million for the comparable period in 2023. SG&A expenses for the second quarter also included $1.2 million of non-cash expense.

Adam Mostafa: Research and development expenses were $20.9 million for the second quarter compared to $15.6 million for the comparable period in 2023. R&D expenses for the second quarter of 2024 included $1.2 million of non-cash expenses. Selling general and administrative expenses were $13.3 million for the second quarter of 2024. This compares to $10.2 million for the comparable period in 2023. SG&A expenses for the second quarter also included $1.2 million of non-cashing expenses. Finally, we reported net income of $90.8 million for the second quarter of 2024, compared to a net loss of $55.7 million for the comparable period in 2023.

Adam Mostafa: Research and development expenses were $20.9 million for the second quarter, compared to $15.6 million for the comparable period in 2023.

Adam Mostafa: R&D expenses for the second quarter of 2024 included $1.2 million in non-cash expense.

Adam Mostafa: Selling general and administrative expenses was $13.3 million for the second quarter of 2024. This compares to $10.2 million for the comparable period in 2023.

Adam Mostafa: sgna expenses for the second quarter also included one point two million dollars of noncash expense

Adam Mostafa: Finally, we reported net income of $90.8 million for the second quarter of 2024, compared to a net loss of $55.7 million for the comparable period in 2023. Note that net income in the current period included the sale of the PRV for $105 million, as well as a non-cash gain of $20.2 million related to the fair value remeasurement of the company's Class C warrant life.

Adam Mostafa: Finally, we reported net income of $90.8 million for the second quarter of 2024, compared to a net loss of $55.7 million for the comparable period in 2023. Note that net income in the current period included the sale of the PRV for $105 million, as well as a non-cash gain of $20.2 million related to the fair value remeasurement of the company's Class C warrants liability.

Adam Mostafa: finally we reported net income but ninety point eight million balance for the second quarter of two thousand and twenty four compared to a net loss of fifty five point seven mill dollars for the comparable period in two thousand and twenty three

Adam Mostafa: Note that net income in the current period included the sale of the PRV for $105 million, as well as a non-cash gain of $20.2 million related to the fair value remeasurement of the company's Class C warrants liability. I'll now turn it back to Paula for some concluding remarks.

Adam Mostafa: Note that net income in the current period included the sale of the PRV for $105 million, as well as a non-cash gain of $20.2 million related to the fair value re-measurement of the company's Class C warrants liability.

Paula Ragan: I'll now turn it back to Paula for some concluding remarks. As you can see, all of these accomplishments mark a significant step forward for X4, now a fully integrated biopharmaceutical company. As we continue to explore additional uses for and maximize the global potential of Mavericks for patients, we look forward to reporting on our sales, regulatory, and clinical progress over the coming six to 12 months.

Adam Mostafa: I'll now turn it back to Paula for some concluding remarks.

Paula Ragan: As you can see, all of these accomplishments mark a significant step forward for X4, now a fully integrated biopharmaceutical company. As we continue to explore additional uses for and maximize the global potential of Maverix IV for patients, we look forward to reporting on our sales, regulatory, and clinical progress over the coming 6 to 12 months. Operator?

Unknown Executive: As you can see, all of these accomplishments mark a significant step forward for X4, now a fully integrated biopharmaceutical company. As we continue to explore additional uses for and maximize the global potential of MaverickScore for patients, we look forward to reporting on our sales, regulatory, and clinical progress over the coming 6 to 12 months. Operator?

Unknown Executive: as you can see all of these accomplishments mark a significant steep forward for x four now a fully integrated biopharmaceutical company

Unknown Executive: As we continue to explore additional uses for and maximize the global potential of Maverick Score for patients, we look forward to reporting on our sales, regulatory, and clinical progress over the coming 6 to 12 months. We'll now open up the call for your questions. Operator?

Operator: We'll now open up the call.

Operator: Thank you, Dr. Ragan. Ladies and gentlemen, at this time, if you would like to ask a question, please press star 1 on your telephone keypad. You may remove yourself from the queue at any time by pressing star 2. Once again, star 1 for questions. We'll go first this morning to Kristen Kluska of Cantor.

Operator: Thank you, Dr. Ragan. Ladies and gentlemen, at this time, if you would like to ask a question, please press star 1 on your telephone keypad. You may remove yourself from the queue at any time by pressing star two. Once again, star one for questions.

Speaker Change: Thank you, Dr. Ragan. Ladies and gentlemen, at this time, if you would like to ask a question, please press star 1 on your telephone keypad. You may remove yourself from the queue at any time by pressing star 2. Once again, star 1 for questions. We'll go first this morning to Kristen Kluska of Kantor.

Kristen Kluska: We'll go first this morning to Kristen Kluska of Cantor. Hey, good morning, everybody. Congratulations on reaching 10 years at the Great Milestone, and how better to celebrate with the drug approval this year.

Kristen Kluska: Hey, good morning, everybody, and congratulations on reaching 10 years. It's a great milestone, and how better to celebrate with a drug approval this year. So, first, I wanted to ask how physicians are going to measure success differently in a commercial setting versus the clinic for Zole Remde. Will it be primarily patient anecdotes, reports around infections, or anything else to consider?

Speaker Change: Hey, good morning, everybody, and congratulations on reaching 10 years. It's a great milestone, and how better to celebrate with a drug approval this year. So, first, I wanted to ask how you believe physicians are going to measure success differently in a commercial setting versus the clinic for Zole Remde? Will it be primarily on patient anecdotes, reports around infections, or anything else to consider?

Paula Ragan: So first, I wanted to ask how you believe physicians are going to measure success differently in a commercial setting versus the clinic for Zolremdi. Will it be primarily on patient anecdotes, reports around infections, or anything else to consider? Oh, thanks, Kristen.

Paula Ragan: Thanks, Kristen. I'll turn it over to Mark to give some perspectives on what they're hearing from the field.

Mark Baldry: I'll turn it over to Mark to give some perspectives on what they're hearing from the field.

Unknown Executive: Thanks, Kristen. I'll turn it over to Mark to give some perspectives on what they're hearing from the field.

Unknown Executive: Yeah, the launch is well underway, Kristen, and what's exciting is that, you know, we're hearing that physicians and patients have been waiting for a treatment that targets the underlying cause of the disease. And so now we have the approval of Zol Remedy. It's really opening up these conversations and allowing us to help physicians become aware of the disease, recognize the patients in their practice, and then think about, you know, how they can help these patients with Zol Remedy.

Mark Baldry: Yeah, the launch is well underway, Kristen, and what's exciting is that, you know, we're hearing that physicians and patients have been waiting for a treatment that targets the underlying cause of the disease. And so now we have the approval of Zole Remedy. It's really opening up these conversations and allowing us to help physicians become aware of the disease, recognize the patients in their practice, and then think about, you know, how they can help these patients with Zole Remedy.

Mark Baldry: Yeah, we're the launches well underway, Kristen. And what's exciting is that, you know, we're hearing that physicians and patients have been waiting for a treatment that targets the underlying cause of the disease. And so now we have the approval of Zolremdi.

Unknown Executive: Yeah, the launch is well underway Kristen and what's exciting is that, you know,

Unknown Executive: we're hearing the physicians and patients have been waiting for a treatment that targets the underlying cause of the disease

Mark Baldry: It's really opening up these conversations and allowing us to help physicians become aware of the disease, recognize the patients in their practice, and then think about how they can help these patients with Zolremdi. We know from the marker research that it's really the impact on infections that drives the value of Zolremdi for physicians, for patients, and patients. So that's really what I think physicians will be looking at as they get more experience with the product. Thank you for that.

Unknown Executive: And so now we have the approval of Zol Remedy, it's really opening up these conversations and allowing us to help physicians become aware of the disease, recognize the patients in their practice, and then think about how they can help these patients with Zol Remedy.

Mark Baldry: We know from the market research that it's really the impact on infections that drives the value of cell remedy for physicians, for patients, and payers, so that's really what I think physicians will be looking for as they get more experience with the product.

Speaker Change: We know from the market research that it's really the impact on infections that drives the value of cell remedy for physicians, for patients, and payers. So that's really what I think physicians will be looking for as they get more experience with the product.

Kristen Kluska: Thank you for that. And can you give us a sense of how partnership discussions outside the US are going at this stage? And just curious, your press release noted some ways you're looking to maximize the value of Maverix for beyond Wnt syndrome and CN. So curious if, in these conversations, this has come up with potential partners about ways that they can also potentially utilize this drug and other indications as well.

Kristen Kluska: And can you give us a sense of how partnership discussions X, U.S. are going at this stage and just curious. Yes, your press release noted some ways you're looking to maximize the value of Mavericks before beyond Winston Germans. And so curious is in these conversations, this has come up with potential partners about ways that they can also potentially utilize the strong and other indications as well.

Speaker Change: Thank you for that and can you give us a sense of how partnership discussions ex-US are going at this stage and just curious, your press release noted some ways you're looking to maximize the value of Maverix for beyond Wnt Syndrome and CN, so curious if in these conversations this has come up with potential partners about ways that they can also potentially utilize this drug and other indications as well.

Paula Ragan: Yeah, so thanks, Kristen. I mean, when we currently think about the FDA approval, there are certain regions in the world that can leverage that existing approval. So there's some exploration there currently ongoing. Adam can comment more on the more strategic sides of potential collaborations.

Paula Ragan: Yeah, so thanks, Kristen. I mean, when we currently think about FDA approval, there are certain regions of the world that can leverage that existing approval. So there's some exploration there currently ongoing. Adam can comment more on the more strategic sides of potential collaboration.

Adam Mostafa: Yeah, so thanks, Kristen. I mean, when we currently think about the FDA approval, there are certain regions of the world that can leverage that existing approval, so there's some exploration there currently ongoing. Adam can comment more on the more strategic sides of potential collaborations.

Adam Mostafa: Yeah, thanks, Kristen. So certainly, there is interest, and we have discussions, as Paula mentioned, ongoing with potential partners. We'll certainly come back and update the market as appropriate if we're going to consummate a transaction. But I think, given the recent approval and our recent positive chrysotopenia phase 2 data, there's only growing interest in this area.

Adam Mostafa: Yeah, thanks, Kristen. So certainly there is an interest in or have discussions with Paul mentioned ongoing with potential partners. Will certainly come back and update the market as appropriate.

Speaker Change: Yeah, thanks, Kristen. So certainly there is interest and we have discussions, as Paula mentioned, ongoing with potential partners. We'll certainly come back and update the market as appropriate.

Kristen Kluska: If we're going to consummate a transaction. But I think given the recent approval and our recent positive crime of opinion face to data, there's only growing interest in this area. Great. Thank you.

Speaker Change: If, if we're going to consummate a transaction, but I think given the recent approval and our recent positive chrysotopenia phase two data, there's only growing interest in this area.

Unknown Executive: oh

Kristen: right thank you

Ted Tenthoff: Thank you. We go next now to Ted Tenthoff at Piper Sandler.

Ted Tenthoff: We go next now to Ted Tenthoff at Piper Sammler. Great, thank you very much for taking my question, and yeah, it's exciting to see some revenues at the company that a lot of work has gone to get that first couple hundred thousand dollars. And I know it'll grow from here.

Speaker Change: thank you we go nextction now to ted tentoff at piper samler

Ted Tenthoff: Great, thank you very much for taking my question, and yeah, it's exciting to see some revenues at the company. I know a lot of work has gone into getting that first couple hundred thousand dollars, and I know it'll grow from here.

Unknown Attendee: Great, thank you very much for taking my question, and yeah, it's exciting to see some revenues at the company. I know a lot of work has gone into getting that first couple hundred thousand dollars, and I know it'll grow from here.

Unknown Attendee: great thank you very much for taking my question and as' excited to see some revenues the company that's and a lot of work is going to get that first couple hundred thousand dollars

Ted Tenthoff: My question had to do with sort of characterizing the patients that are on drugs. Can you give us a sense for how many patients are on drugs and anything about the backlog? And if you're not able to give us direct patient numbers, maybe tell us sort of a little bit about the journey of patients that you're seeing as you sort of get them into commercial use of Zolandi. Thank you.

Unknown Attendee: My question had to do with sort of characterizing the patients that are on the drug. Can you give us a sense for how many patients are on the drug and anything about sort of the backlog? And if you're not able to give us direct patient numbers, maybe tell us sort of a little bit about the journey of patients that you're seeing as you sort of get them into commercial use of Zolandi. Thank you.

Ted Tenthoff: My question had to do with sort of characterizing the patients that are on drugs. Can you give us a sense for how many patients are on drugs? And is anything about sort of the backlog? And if you're not able to give us direct patient numbers, maybe tell us sort of a little bit about the journey of patients that you're seeing as you sort of get them into commercial use of Zorundi. Thank you.

Unknown Attendee: And I know it'll grow from here. My question had to do with sort of characterizing the patients that are on drug. Can you give us a sense for how many patients are on drug and anything about sort of the backlog?

Unknown Attendee: and if you're not abing to its directation numbers maybe tell us sort of a little bit about the journey of patients that you're seeing as you sort of get them into commercial use of zumty thank you

Paula Ragan: Thanks so much, Ted. So I think, you know, we won't be breaking down any details and continue to report on revenue, but I mean we're overall extremely pleased with just the cascade of commitment that the company has made and then now we're experiencing the receipt of that commitment in the communities as we head out, but Mark can add some more color and kind of how they're seeing the journey from the diagnosis and education support all the way through patients on drugs.

Paula Ragan: Thanks so much, Ted. I think, you know, we won't be breaking down any details and will continue to report on revenue. But I mean, overall, we're extremely pleased with just the cascade of commitment that the company has made, and now we're experiencing the reception of that commitment in the communities as we head out. But Marc can add some more color and kind of show how they're seeing the journey from diagnosis and education support all the way through to patients on drugs.

Speaker Change: thanks so much ted so i thinkyou know we won't be breaking down any details and conf report on revenue but i mean we're overall extremely pleased with just the cascade of commitment that the company has made and then now we'reexperiencing the receipt of that commitment in the communities that we had out but mark can add some more color and kind of how they're seeing the journey from from the diagnosis and ucation support all the way through patients on drugs

Mark Baldry: Yeah, and I think, you know, the first thing to remember, this is an ultra-rare disease, and so the key here is to begin to really raise awareness, both through the conversations we're having with physicians, our engagement with, you know, patient advocacy groups, and also through our digital marketing campaigns, because that's where we can get extended reach, and we've refreshed our disease awareness campaign, we've also launched our branded digital campaigns, so we're really creating this kind of momentum around the conversations, and the patients, you know, as Paula mentioned on the call, all of the patients that were in the open label extension have been prescribed Zole Remedy here in the U.S., and so from there, we're just.., learning more and more about how the patients have been waiting for a treatment that targets their disease and they're not having those conversations with physicians. Great.

Mark Baldry: Yeah, and I think the first thing to remember this is an ultra-rare disease. And so the key here is to begin to really raise awareness both through the conversations we're having with physicians, our engagement with patient advocacy groups, and also through our digital marketing campaigns because that's where we can get extended reach. And we've refreshed our disease awareness campaign. We've also launched our branded Zorundi digital campaigns. So we're really creating this kind of momentum around the conversations. And the patients, you know, as Paul mentioned on the call, all of the patients that were in the open label extension have been prescribed Zorundi here in the US.

Speaker Change: Yeah, and I think, you know, the first thing to remember, this is an ultra rare disease, and so the key here is to begin to really

Ted Tenthoff: Great. That's really helpful. I appreciate the additional color.

Operator: Please stand by, we're about to begin. Good morning, everyone, and welcome to X4 Pharmaceuticals 2nd quarter, 2024 earnings conference call.

Operator: At this time, all participants are in a listen only mode. A question and answer session will follow the formal presentation. You may register to ask a question at any time by pressing star one on your telephone keypad.

Speaker Change: Raise awareness, both through the conversations we're having with physicians, our engagement with, you know, patient advocacy groups, and also through our digital marketing campaigns, because that's where we can get extended reach. And we've refreshed our disease awareness campaign. We've also launched our branded Zol Remedy digital campaigns.

Operator: Also, today's call is being recorded, and if you should need any assistance during the call today, please press star zero.

Daniel Ferry: It is now my pleasure to introduce your host, Mr. Dan Ferry from Lifestyle Advisors. Dan, please go ahead. Thank you, operator. Good morning, everyone. Thank you for joining us today. Presenting on today's call will be Dr. Paula Ragan, X4's president and CEO, and the company's Chief Financial Officer, Adam Mostafa. Following prepared remarks, we will open up the call to your questions, and we'll be joined by Chief Commercial Officer, Mark Baldry, Chief Medical Officer, Dr. Christophe Arbet Engels, Chief Operating Officer, Dr. Mary D. Biasi, Chief Scientific Officer, Dr. Art Tveris, and Jose Huves, Head of Corporate and Patient Affairs.

Paula Ragan: So, we're really creating this kind of momentum around the conversations, and the patients, you know, as Paula mentioned on the call, all of the patients that were in the open label extension have been prescribed Zylremdi here in the U.S., and so from there, we're just...

Mark Baldry: And so from there, we're just learning more and more about how the patients have been waiting for a treatment that targets their disease and now having those conversations with physicians.

Unknown Attendee: I'm learning more and more about how patients have been waiting for a treatment that targets their disease, and they're not having those conversations with physicians. Great

Unknown Attendee: Learning more and more about how the patients have been waiting for a treatment that targets their disease and they're now having those conversations with physicians.

Ted Tenthoff: Great. That's really helpful. I appreciate that this will color.

Operator: Thank you.

Operator: Thank you. We go next to R.K. at H.C. Wainwright.

RK: We go next now to RK at HC Wainwright. Thank you. Good morning, Paula and Adam.

Unknown Attendee: Thank you. Good morning, Paula and Adam. Congratulations on your tenures and also the launch, getting ready to adopt the drug. And also, have you learned anything else that needs to be looked at in terms of improving, I'm not saying improving, you know, getting the adoption at a better level from the get-go?

R.K.: Thank you. Good morning, Paula and Adam. Congratulations on your tenures and also the launch. In terms of, I know it's the early stages of the launch, but when you go and attend these conferences, such as the Immunodeficiency Conference, what is it that you're hearing from physicians and patients at this point in terms of getting ready to adopt the drug, and also, have you learned anything else that needs to be looked at in terms of improving, I'm not saying improving, you know, getting the adoption at a better level from the

RK: Congratulations on the 10 years and also the launch. In terms of, I know it's early early stages of the launch, but when you go and attend these conferences such as the immunodeficiency conference, how are, you know, what is it that you're hearing, you know, from from physicians and patients at this point in terms of getting ready to take to adopt the drug and also have you learnt anything else that needs to be looked at in terms of improving, I'm not saying improving, you know, getting that option to a better level from the get go. So thanks so much, RK.

Daniel Ferry: As a reminder on today's call, the company will be making forward-looking statements regarding regulatory and product development and commercialization plans, as well as research activities and financial projections. These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted.

Speaker Change: In terms of I know it's early.

Speaker Change: Honestly just from the launch.

Unknown Attendee: But.

Unknown Attendee: And then when you go on that.

Unknown Attendee: These conferences such as the immuno deficiency conference.

Unknown Attendee: Hum.

Daniel Ferry: A description of these risks can be found in X4's most recent filings with the SEC, including this year's Forum 10K, which was filed on March 21st, 2024, and in the company's Forum 10Q for the second quarter, which is expected to be filed later today.

Speaker Change: What is it that you are hearing from some physicians.

Speaker Change: Ah patients at this point.

Unknown Attendee: In terms of.

Unknown Attendee:

Speaker Change: Getting getting ready to rock.

Speaker Change: To adopt the drug and also have you learnt anything else that needs to be.

Paula Ragan: I'll now turn it over to Paula Ragan. Paula? Thanks so much, Dan, and welcome everyone.

Unknown Attendee: Looked at in terms of improving I'm, not saying I'm flooring.

Unknown Attendee: Getting that option.

Paula Ragan: I'll just start by saying what an incredible year we've had so far. Just a few weeks ago, we celebrated the 10th anniversary of X4. When we founded a company a decade ago, a small group of us had a vision to advance our lead asset, an orally active CX-4 antagonist, called Mavericks-4, to help those with rare diseases who have few to know treatment options. As you saw this past April, we were able to realize this vision receiving US approval of Mavericks-4, branded as Zolrendi, for the treatment of Wim Syndrome, a rare primary immunodeficiency.

Speaker Change: A better level.

Speaker Change: From the get go.

Unknown Attendee: Okay.

Paula Ragan: Thanks so much, R.K. I think, you know, we're very pleased with kind of the cascade of thinking and development, and certainly what we're hearing, and Marc will add to this, is number one, they're excited about a targeted therapy, and I'll even add oral targeted therapy. It's been a field of injectable treatments with varying degrees of study and rigor. Our phase three was phenomenally successful, showing, you know, infection burden rejection across rate, severity, and duration, so that is a great story to begin with and a great data set to share.

Speaker Change: Thanks, so much RK I think anywhere.

Paula Ragan: I think, you know, we're very pleased with kind of the cascade of thinking and development, and certainly what we're hearing, and Mark will add to this, is number one, they're excited about a targeted therapy, and I'll even add the oral targeted therapy. It's been a field of injectable treatments with varying degrees of study and rigor, are faced with some only successful showing, you know, infection burden reduction across rates, severity, and duration. So that is a great story to begin with and a great data set to share.

Speaker Change: Very pleased with kind of the the cascade of thinking and developments and certainly what we're hearing and Mark will add to this is number one they are excited about a targeted therapy and I'll, even abbvie oral targeted therapy, it's been a field of injectable treatments with varying degrees of.

Unknown Attendee: Study and rigor of our phase III with not only successful showing infection burden reduction in cross rate severity and duration. So that is a.

Paula Ragan: The US launch of Zolrendi is now underway and our full commercial team is in place, and when patients 12 years and older are now being treated with the only approved therapy targeting the underlying cause of their disease. The old guidance on Zolrendi will come at a future point. However, today, I'd like to highlight our strong launch progress. Overall, we've been very pleased with the launch to date. Our launch meeting in early May was a great success, where we completed the training and education of our field teams, enabling them to hit the ground running.

Unknown Attendee: Great story to begin with and a great dataset to share and then in terms of the clinicians of course, they're all on their own journey, even understanding the disease and then learning about the benefits of Rosa they'll remedy, but I'll turn it over to mark to add some color to that as well.

Mark Baldry: And then, in terms of the clinicians, of course, they're all on their own journey, even understanding the disease and then learning about the benefits of the role of Zulremdi. But I'll turn it over to Mark to add some color to that as well.

Paula Ragan: And then in terms of the clinicians, of course, they're all on their own journeys, even understanding the disease and then learning about the benefits of Zole Remedy, but I'll turn it over to Marc to add some color to that as well. Yeah, exactly.

Mark Baldry: Yeah, exactly. I think now that we have an approved product, it's really energized physicians to go back and really look in their practice for these patients who, before now, really had no treatment available for their disease. And so, now we've actually given them a reason to go back, look for these patients, make the diagnosis, and then have a conversation with the patient about Zole Remedy.

Unknown Executive: Yeah, exactly. I think now that we have an approved product, it's really energized physicians to go back and really look in their practice for these patients who, before now, really had no treatment available for their disease. And so, now we've actually given them a reason to go back, look for these patients, make the diagnosis, and then have a conversation with the patient about Zole Remedy.

Mark Baldry: Yeah, exactly.

Mark Baldry: I think it's really now that we have an approved product. It's really energized positions to go back and really look in their practice for these patients who, before now, have really had no treatment available for their disease. And so now we've actually given them a reason to go back, look for these patients, make the diagnosis, and then have a conversation with the patient about Zulremdi. Fantastic.

Speaker Change: Yes, exactly I think it's really now that we have an approved product, it's really energized physicians to go back and really look in there their practice for these patients who.

Speaker Change: Before now I really have no treatment available for their disease and so now we've actually given them a reason to go back look for these patients make the diagnosis and then have a conversation with the patient about the RMB.

Paula Ragan: We got product into our distribution channel very quickly and had our first patients on commercial drugs within just a few weeks of product approval. Coordination with our specialty pharmacy, Panther RX Rare, has gone smoothly and we've already received positive feedback on the patient support services provided to our X4 Connect and Nurse Educator programs. And very encouragingly, reimbursement decisions have been coming through quickly, with almost all patients currently on therapy receiving reimbursement via prior authorizations and medical exceptions.

R.K.: Fantastic. And then for the SEM indication, so between now and and if there is an interim analysis for the forward study, is there any other data that will be presented so that we can understand how this program is moving forward?

Speaker Change: I'm passing.

Paula Ragan: And then for the SCN indication, so between now and if there is an interim analysis for the forward study, is there any other data that will be presented so that we can understand how this program is moving forward? Sure, so as we shared today, we'll be giving the full Phase Two data set in November of this year. So, of course, we were extremely pleased with the data set already, but it was interim. So we've already seen durable responses of the drug as monotherapy and then sustained and durable responses in addition to stable G-CSF. So the final puzzle piece is just exploring in what circumstances G can get reduced.

Unknown Executive: Amen.

Unknown Executive: The.

Unknown Executive: C N indication.

Unknown Executive: So between now and.

Unknown Executive: Hum.

Unknown Executive: Yes.

Unknown Executive: And if.

Speaker Change: If there is an interim analysis for them for the Harvard study is that it.

Unknown Executive: The other data that will be presented.

Unknown Executive: So that we can understand how this program is moving forward.

Paula Ragan: Sure. So, as we shared today, we'll be giving the full Phase 2 data set in November of this year. So, of course, we were extremely pleased with the data set already, but it was interim. So, we've already seen durable responses to the drug as monotherapy and then sustained and durable responses in addition to stable GCSF. So, the final puzzle piece is just exploring in what circumstances G can get reduced.

Speaker Change: Sure. So as we shared today will be giving the full phase two datasets in November of this year. So of course, we were extremely pleased with the dataset already but it was interim so we've already seen durable responses of the drug as monotherapy and then sustained and durable responses. In addition to.

Paula Ragan: Our commercial team is fully deployed across the US, calling on key hematologists and immunologists. To date, there has been limited ability to establish centers of excellence for the wind community. We knew this going in and therefore are continuing to execute on our disease awareness and education campaigns, as well as leveraging our strong relationships with patient advocacy groups. We recently participated in the annual meeting of the Immune Deficiency Foundation, or IDF, where we engage with physicians, patients, and advocates, and members of our team visited with the Jeffrey Modell Foundation in New York just a few weeks ago.

Speaker Change: Table Gcs after the final puzzle pieces, just exploring and what circumstances GE can get reduced so that full dataset will come in November which is gonna be certainly well timed just before ash will be at the ash meeting to help support the Sienna.

Paula Ragan: So that full data set will come in November, which is going to be certainly well timed just before Ash will be at the Ash meeting to help support the SCN community and also get the word further out on our trial enrollment. So it's an excellent timing perspective, and then we'll look forward, as appropriate, to giving updates on how the whole study is going. But certainly, that will be a 2025 update.

Paula Ragan: So, that full data set will come in November, which is going to be certainly well-timed just before ASH. We'll be at the ASH meeting to help support the CN community and also get the word further out on our trial enrollment. So, it's an excellent timing perspective, and then we'll look forward, as appropriate, to giving updates on how the whole study is going. But certainly, that will be a 2025 update.

Speaker Change: Community and also get the word further out on our trial enrollment. So it's an excellent timing perspective, and then we'll look forward as appropriate to giving updates on how the whole study is going but certainly that will be a 2025 update.

Paula Ragan: Through all of these efforts, we had become better at engaging with the treating physicians, understanding physician and patient questions, and most importantly, building a community of interests around wind syndrome across the US. We also continue to leverage the positive data from our pivotal phase 3 trial and wind that were published in April in blood, the Journal of the American Society of Helmetology. These and new data from the trial and its open label extension phase were presented at the annual meeting of the Clinical Immunological Society, or CIS, in early May.

Paula Ragan: Perfect. Thank you very much for taking my questions, Paul. Thanks, Arkay. We'll take our next question now from Leah Cann of Brookline Capital Markets.

RK: Perfect.

Speaker Change: Perfect. Thank you very much for taking my questions Paul.

RK: Thank you very much for taking my questions, Paul. Thanks, okay.

Unknown Attendee: Thanks, Arkay.

RK: Thanks RK.

Unknown Executive: Yeah.

Leah Cann: We'll take our next question now from Leah Can of Brookline Capital Markets. Good morning. I have a financial question. So Adam, could you help us with the number of fully diluted shares at the end of the quarter, including the pre-funded warrants?

Leah Cann: We'll take our next question now from Leah Cann of Brookline Capital.

Leah Cann: We'll take our next question now from Leah Cann of Brookline Capital. Good morning. I have a financial question.

Speaker Change: We will take our next question now from Leah Cann of Brookline capital markets.

Speaker Change: Good morning, I have a financial question.

Paula Ragan: As we reported at the time, the CIS poster revealed that long-term treatment, with Zoll Remdi, was associated with durable improvements in mutual and lymphocyte counts, as well as reductions in annualized infection rates, and that no new safety signals had been observed during the OLE phase of the trial, with some participants treated for more than two years.

Leah Cann: Adam could you help us with the number of fully diluted shares at the end of the quarter, including the pre funded warrants.

Adam Mostafa: Sure. Thanks.

Unknown Executive: Sure, thanks. So there are about 167 million basic shares, regular shares outstanding, and 33 million pre-funded warrants, so 200 million share equivalents, if you will. And then there are 75 million warrants that are cash only; those are divided into two tranches, one 30 million tranche and one 45 million tranche. So, overall, $275 million in fully diluted shares. There are some historical warrants you'll see later today in our 10-Q that are significantly out of the money and are typically not included, but that accounts for a small piece.

Leah Cann: Sure.

Adam Mostafa: So there's about 167 million basic shares, regular shares, that's standing. 33 million pre-funded warrants. So 200 million share equivalents, if you will.

Speaker Change: So there is about 167 million basic shares regular shares outstanding.

Speaker Change: $33 million pre funded warrants.

Speaker Change: So 200 million.

Speaker Change: Share equivalents, if you will.

Unknown Executive: And then there are 75 million warrants that are cash only; those are divided into two tranches, one 30 million tranche and one 45 million tranche.

Adam Mostafa: And then there are 75 million warrants that are cash only. Those are divided into two tranches: 130 million tranches, 145 million tranches. So overall 275 million in fully diluted shares.

Speaker Change: And then there are 75 million warrants that are cash only those are divided into two tranches $130 million tranche 145 million tranche.

Paula Ragan: We also made tremendous progress this past quarter in our development of Mavericks before for those with chronic uterineia, another rare immunodeficiency. At our investor event in late June, we presented positive interim clinical data from our ongoing phase 2 clinical trial, demonstrating that once daily oral Mavericks before was generally well tolerated and durably increased participants' absolute mutual accounts, or ANC, both as a mild therapy and in combination with stable doses of injectable granulocyte colony stimulating factor, or GTSF, out to as long as six months.

Speaker Change: So overall $275 million.

Speaker Change: Fully diluted shares.

Adam Mostafa: There's some historical warrants you'll see later today in our 10-Q that are significantly out of the money that are typically not included, but that accounts for a small piece. Thank you so much.

Speaker Change: There is some historical warrants youll see later today and our 10-Q that are significantly out of the money Theyre typically not included but.

Speaker Change: That accounts for a small piece.

Speaker Change: Thank you so much most helpful.

Unknown Executive: Okay.

Stephen Willey: You will go next now to Stephen Wiley at Steve. Yeah, good morning. Thank you for taking the questions. Could you maybe just talk a little bit about, and again, I understand that it's early, but the kinetics of the reimbursement process. I know the paperwork that is required sometimes for these higher price rare disease drugs can be a somewhat lengthy process. And just curious if you have an early read just based on the limited experience that's far as to what that process might look like from just the timing perspective.

Speaker Change: Thank you we'll go next now to Stephen Wiley at Stifel.

Stephen Wiley: Thank you. We go next to Stephen Wiley at CDC.

Unknown Attendee: Yeah, good morning. Thank you for taking the questions. Could you maybe just talk a little bit about, and again, I understand that it's early, but the kinetics of the reimbursement process. I know the paperwork that is required sometimes for these higher-priced rare disease drugs can be a somewhat lengthy process. And just curious if you have an early read just based on the limited experience thus far as to what that process might look like from just a timing perspective.

Stephen Wiley: Yeah, good morning. Thank you for taking the questions. Could you maybe just talk a little bit about, and again, I understand that it's early, but the kinetics of the reimbursement process. I know the paperwork that is required sometimes for these higher-priced rare disease drugs can be a somewhat lengthy process. And just curious if you have an early read just based on the limited experience thus far as to what that process might look like from just a timing perspective.

Leah Cann: Yes. Good morning, Thank you for taking the questions.

Unknown Attendee: Could you, maybe just talk a little bit about it and again I understand that it's early but.

Paula Ragan: Importantly, 100% of the valuable participants who had completed the six-month study as of the mid-May cut-off date achieved target ANC increases of greater than 500 cells per microliter at month three and six on one daily Mavericks for as a monotherapy and in combination with stable GTSF dosing. In addition, participants on Mavericks for a monotherapy achieved mean ANC levels above the lower limit of normal for CN at three and six months. And Maverick's form monotherapy also durably increased ANC in participants with severe CN, defined as ANC's less than 500 cells per microliter at baseline, achieving mean ANC's of 800 to 1000 cells per microliter, which is the ANC range targeted by experts for patients with severe CN.

Unknown Attendee: The kinetics of the reimbursement process.

Speaker Change: The paperwork that's required sometimes for these higher price rare disease drugs.

Unknown Attendee: Okay.

Unknown Attendee: Somewhat lengthy process.

Unknown Attendee: Just curious if you have an early read just based on the limited experience, thus far as to what that process might look like from just a timing perspective.

Paula Ragan: Sure, Steve. I mean, I'll turn it over to Mark. I think classic ultra rare disease typically has barriers that a lot of these insurance companies naturally provide given the magnitude of the drug and its options for patients. But the good news is I think we've had an experienced team well ahead of this.

Unknown Executive: Sure, Steve. I mean, I'll turn it over to Mark.

Stephen Wiley: Sure, Steve. I mean, I'll turn it over to Marc. I think classic, ultra-rare diseases typically have barriers that a lot of these insurance companies naturally provide, given the magnitude of the drug and its options for patients, but the good news is I think we've had a very experienced team well ahead of this, so I'll turn it over to Marc to give some insights there.

Unknown Attendee: Sure, Steve I mean, I'll turn it over to Mark I think classic ultra rare disease typically has barriers that a lot of these insurance companies naturally provide given given the magnitude of the dragon its options for patients but.

Paula Ragan: I think classic, ultra-rare diseases typically have barriers that a lot of these insurance companies naturally provide, given the magnitude of the drug and its options for patients, but the good news is I think we've had a very experienced team well ahead of this, so I'll turn it over to Mark to give some insights there.

Paula Ragan: But the good news is I think we've had very experienced team well ahead of this so I'll turn it over to Mark to give some insights there.

Mark Baldry: So I'll turn it over to Mark to give some insights there. Yes. Paula mentioned during the call, payers are using their standard methods for new drugs and so, you know, they're looking at PAs and exceptions process, but these are working quite smoothly, and patients have been able to access the remedy. Of course, we also have our full suite of patient service programs through our X4 Connect, which is our patient hub, and this ensures that patients are quickly able to get quick access to the product while we work through the reimbursement. But it's good to see the process working smoothly.

Paula Ragan: Yes, Paula. Hi, Steve.

Mark Baldry: Yeah, thanks Paula. Hi Steve.

Mark: Yeah, Thanks, Paul Hi, Steve Yeah, as Paul mentioned during the call payers are using their standard methods for four new drugs and so.

Mark Baldry: Yeah, as Paula mentioned during the call, payers are using their standard methods for new drugs, and so, you know, they're looking at PAs and exceptions processes, but these are working quite smoothly, and patients have been able to access Cell Remedy. Of course, we also have our full suite of patient service programs through X4 Connect, which is our patient hub, and this ensures that patients quickly get quick access to the product while we work through reimbursement. But it's good to see the process working smoothly.

Paula Ragan: Feedback on these interim results from the physician community has been positive, and we were pleased to see the durable impact of Maverick's form as a monotherapy and in combination with stable dose GCSF. These interim data support are strong beliefs that Mavericks form may be able to address the needs of the CN patients and their physicians. Specifically, the desire for an oral treatment that does not carry short or long-term dose-related toxicities described with GCSF use. And for an oral treatment that also increases ANC levels and lessens the risk and severity of infections, whether used on its own or in combination with GCSF.

Mark Baldry: Theyre looking at Tas and exceptions process, but these are working quite smoothly and.

Mark Baldry: Patients have been able to access all Randy of course, we also have our full suite of patient service programs through our X for connect which is our patient hub and this ensures that patients are quickly.

Mark Baldry: Yeah, as Paula mentioned during the call, payers are using their standard methods for new drugs, and so, you know, they're looking at PAs and exceptions processes, but these are working quite smoothly, and patients have been able to access CellRemedy. Of course, we also have our full suite of patient service programs through X4 Connect, which is our patient hub, and this ensures that patients quickly get quick access to the product while we work through reimbursement. But it's good to see the process working smoothly.

Mark Baldry: Just quick access to the product, while we worked through the winter.

Mark Baldry: Reimbursement.

Mark Baldry: But it is good to see the process working smoothly.

Stephen Willey: Okay, that's helpful.

Unknown Attendee: Okay, that's helpful. And... Maybe a guidance question: updates in conjunction with certain milestones hit like 50%, or is this just going to be kind of a moving target, and we'll hear about it when we hear?

Speaker Change: Okay, that's helpful and.

Stephen Wiley: Okay, that's helpful. And... Maybe a guidance question. One related to actual sales, but I'm just curious how you're thinking about communicating enrollment progress in the phase three forward trial going forward. Should we expect updates in conjunction with certain milestones, like 50%, or is this just going to be kind of a moving target, and we'll hear about it when we hear?

Stephen Willey: And maybe a guidance question, not one related to actual sales, but just curious how you're thinking about communicating enrollment progress in the phase three forward trial going forward. Should we expect updates in conjunction with certain milestones, had like 50% or is this just going to be kind of a moving target and we'll hear about it when we hear about it.

Speaker Change: Maybe a guidance question.

Speaker Change: One related to actual sales.

Paula Ragan: We're now expecting to present the full data from the Phase 2 CN trial in November, ahead of the American Society of Hematology or Ash meeting. This data set will include results from the group of participants receiving Mavericks before and those who are allowed to dose adjust GCSF from baseline. We've been asked what success looks like for these data that will be presented in November. The good news is we already know what success looks like from our WIM Phase 3 study and subsequent approval in the US.

Speaker Change: Just curious how you're thinking about communicating enrollment progress.

Speaker Change: Phase III forward trial going forward should we expect.

Speaker Change: Updates in conjunction with certain milestones like 50% or.

Speaker Change: Or is this just going to be kind of.

Speaker Change: A moving target and we'll hear about it when we hear about it.

Stephen Willey: Yeah, thanks, Steve. I think we'll always aim to communicate meaningful progress. Of course, we don't want to make any commitments just because it's a 2025 target. It's almost a year away at this point. However, certainly, as we get confidence under our belt here, we'll be able to provide some more specifics. But again, I would say that's a 2025 zone to share.

Paula Ragan: Yeah, thanks, Steve. I think we'll always aim to communicate meaningful progress. Of course, we don't want to make any commitments just because it's a 2025 target and it's almost a year away at this point. However, certainly, as we get confidence under our belts here, we'll be able to provide some more specifics. But again, I would say that's a 2025 zone to share.

Unknown Executive: Yeah, thanks, Steve. I think we'll always aim to communicate meaningful progress. Of course, we don't want to make any commitments just because it's a 2025 target and it's almost a year away at this point. However, certainly, as we get confidence under our belts here, we'll be able to provide some more specifics. But again, I would say that's a 2025 zone to share.

Unknown Attendee: Yeah, Thanks, Steve I think well always aim to communicate meaningful progress.

Paula Ragan: In that 52 week study, Mavericks form meaningfully increased neutrophils and lymphocytes over a sustained period, reducing infection rate, duration and severity. From the interim CNC's peer results we shared in June, we saw again that Mavericks for as a monotherapy and in combination with GCSF can increase ANCs to levels expected to reduce infections. In November, we'll be looking to replicate what we saw in June across all participants in the Mavericks for a monotherapy and MAV plus stable dose GCSF groups who completed the six months of the trial.

Unknown Executive: Of course, we don't want to make any commitments just because it's.

Unknown Executive: 2025 target and it's almost a year away at this point, however, certainly as we get confidence.

Unknown Executive: Under our belt here will be able to provide some more specifics, but again I would say that for 2025 zone two to share.

Stephen Wiley: Okay, and then maybe just another financial question for Adam. SG&A just looks to be down about 25% sequentially, which I guess I wouldn't have anticipated in the context of undertaking a launch, so can you just remind us if there's anything either unique to the one-quarter number or this quarter number that impacts that SG&A line item? Thank you.

Stephen Willey: Okay, and then maybe just another financial question for Adam.

Unknown Executive: Okay, and then maybe just.

Speaker Change: Another financial question for Adam.

Adam Mostafa: STA just looks to be down about 25% sequentially, which I guess I wouldn't have anticipated in the context of undertaking a launch.

Speaker Change: SG&A just looks to be down about 25% sequentially, which I guess I wouldn't have anticipated in the context of.

Unknown Executive: Undertaking a launch so.

Adam Mostafa: So can you just remind us that there's anything either unique to the one quarter number or this quarter number that impacts that is GNA line up. Thank you. Thanks, Steve.

Paula Ragan: From the third group, we'll be looking for data that supports physicians' ability to reduce GCSF dosing while Mavericks for and maintain patients ANCs at clinically meaningful levels. Finally, we'll be also looking for continued tolerability and safety of Mavericks for as seen in all of our other clinical programs to date.

Speaker Change: Can you just remind us if theres anything unique to the one quarter number or this quarter number.

Unknown Attendee: that impacts that SG&A line item. Thank you.

Speaker Change: That impacts.

Speaker Change: That SG&A line item. Thank you.

Adam Mostafa: Thanks, Keith. Yeah, so this second quarter number is probably more representative of a typical recurring figure. The first quarter, as we mentioned last time, had a number of one-time, non-recurring startup-related costs for building a sales force and getting a launch underway early in the year ahead of approval. So that quarter was a little more inflated, if you will, in the SG&A line than I would expect going forward. So that's why the drop looks as it does. Very helpful. Thanks for reading this.

Unknown Executive: Thanks, Keith. Yeah, so this second quarter number is probably more representative of a typical recurring figure. The first quarter, as we mentioned last time, had a number of one-time, non-recurring startup-related costs for building a sales force and getting a launch underway early in the year ahead of approval. So that quarter was a little more inflated, if you will, in the SG&A line than I would expect going forward. So that's why the drop looks as it does.

Speaker Change: Alright, Thanks, Steve Yes, so the second quarter number is probably more representative of a typical recurring figure in the first quarter as we mentioned last time that a number of one time nonrecurring.

Adam Mostafa: Yeah, so this second quarter number is probably more representative of a typical recurring figure. The first quarter, as we mentioned last time, that a number of one time non-recurring sort of start up related costs to building a sales force and getting a launch underway early in the year ahead of approval. So, so that quarter was a little more inflated. If you were on the SCNA line, then I would expect going forward. So that's why the drop looks as does.

Paula Ragan: In June, we also announced the initiation of our Global Pivotal Phase III clinical trial, branded the forward study, evaluating the efficacy, safety and tolerability of oral one-stay-ly Mavericks for with and without stable doses of GCSF and people with congenital, acquired primary autoimmune or idiopathic CN for experiencing recurrent and or serious infections. The 52-week forward trial is a randomized double-blinded placebo controlled multi-center study aiming to enroll 150 participants. The clinical operations team is in full swing opening up sites and we are on track to fully enroll the trial in mid 2025.

Stephen Wiley: Very helpful. Thanks for taking the questions and congrats on the progress.

Unknown Executive: Startup related cost to building a sales force and getting the launch underway early in the year ahead of approval.

Unknown Executive: So that quarter was a little more inflated if you will on the SG&A line, then I would expect going forward. So that's why the drop looks.

Unknown Executive: Yes.

Stephen Willey: Very helpful.

Unknown Attendee: Very helpful. Thanks for taking the questions and congrats on the progress.

Speaker Change: Very helpful. Thanks for taking my questions and congrats on the progress.

Stephen Willey: Thanks for taking the questions, and congrats on the progress. Thank you.

Steve: Thanks, Steve.

Unknown Attendee: Thank you. We go next to David Bautz at Zacks Small Cap Research.

David Bautz: Thank you. We go next to David Bautz at Zacks Small Cap Research.

David Doubts: Thank you well go next to David doubts at Zacks small cap research.

David Bautz: We go next now to David Bouts at Zaxx, a small gap research. Hey, good morning, everybody. Question on the mechanisms for how prescriptions are filled. Are they filled on a monthly basis? Is it multiple monthly? And then if you could talk a little bit about how revenue was recognized in relation to when those prescriptions are filled. Sure.

David Bautz: Hey, good morning, everybody. A question on the mechanism for how prescriptions are filled. Are they filled on a monthly basis? Is it multiple monthly? And then could you talk a little bit about how revenue is recognized in relation to when those prescriptions are filled.

David Bautz: Hey, good morning, everybody. A question on the mechanism for how prescriptions are filled. Are they filled on a monthly basis? Is it multiple monthly? And then could you talk a little bit about how revenue is recognized in relation to when those prescriptions are filled.

David Bautz: Hey, good morning, everybody.

David Bautz: Question on the mechanism of how prescriptions are filled or are they filled on a monthly basis is it multiple monthly and then if you could talk a little bit about how revenue was recognized in relation to.

Paula Ragan: We believe that the interim phase 2 CN data presented to date strongly support our decision to initiate the phase 3 trial as well as design. Importantly, we believe that the interim results de-risk the phase 3 trial, given that the ANC increases seen in CN patients to date mirror those and are successfully completed 4 WIM phase 3 trial, where similar levels of ANC increases led to clinical benefit by reducing the frequency, duration, and severity. As we focus our efforts on the forward CN trial, we are also looking to maximize the global opportunity in WIM syndrome.

Speaker Change: When the windows prescriptions are filled.

Paula Ragan: Sure. So, I mean, I don't think we have intended to disclose kind of the play-by-play for our patients. I mean, as we mentioned with our current revenue line, it does blend, obviously, both an inventory build as well as revenue recognition for individual patients that are not on commercial therapy. So, I don't think at this point we're ready to provide any more details, but certainly, you know, we do want, we do understand the market's interest in helping them forecast.

Speaker Change: Sure. So I mean, I don't think we have intended to disclose kind of the play by play for our patients I mean, as we mentioned with our current revenue line. It does blend them all.

Paula Ragan: So I mean, I don't think we have intended to disclose kind of the play by play for our patients. I mean, as we mentioned with our current revenue line, it does blend obviously both an inventory build as well as revenue recognition for individual patients that are now commercial therapy. So I don't think at this point we're ready to provide any more details, but certainly, you know, we do want, we do understand the market's interest in helping them forecast. So as we head into, you know, later this year, 2025, we'll try to provide a bit more metrics, which really can help provide some benefit to you all as we think about how the product will grow over time.

Speaker Change: Obviously, both of an inventory build as well as revenue recognition for individual patients that are now on commercial therapy. So I don't think at this point, we're ready to provide any more details, but certainly we do want we do understand the market's interest in helping them forecast. So as we head into later later this year in 2025, we'll try to provide a bit more metrics, which really can help.

Paula Ragan: So, as we head into, you know, later this year in 2025, we'll try to provide a bit more metrics, which can really help provide some benefit to you all as we think about how the product will grow over time.

Paula Ragan: We expect an MAA submission to the European Medicines Agency for Mavericks for WIM syndrome by early 2025, while we also explore additional potential opportunities and geographies where we may be able to efficiently leverage our US FDA approval.

Speaker Change: Provide some.

Speaker Change: Benefits to you all as we think about how the product will grow over time.

David Bautz: Okay.

David Bautz: Okay, thanks for taking the question.

Unknown Attendee: Okay, thanks for taking the question.

David Bautz: Okay, thanks for taking the question.

Speaker Change: Okay. Thanks for taking the question.

Operator: Thank you. Thank you, and let me, gentlemen, appears we have no further questions this morning.

Speaker Change: Thank you.

Adam Mostafa: With that, I'll now turn it over to our CFO, Adam Mostafa, to review the second quarter financials. Adam? Thanks, Paula, and thanks to all of you for being on the call of us today. As we had previously discussed upon FDA approval of ZOR MD, we were awarded a priority review voucher or PRV based on Mavericks' course rare pediatric disease designation. We monetized this PRV very shortly after receipt, and in May, we recognized a gain on the sale to a third party for $105 million in cash.

Unknown Attendee: Okay.

Operator: Thank you. And ladies and gentlemen, it appears we have no further questions this morning. Dr. Ragan, I'd like to turn things back to you, ma'am, for any closing comments.

Speaker Change: Thank you and ladies and gentlemen, it appears we have no further questions. This morning, Dr. Ragan I'd like to turn things back to Ya man for any closing comments.

Paula Ragan: Dr. Reagan, I'd like to turn things back to you, ma'am, for any closing comments. Well, thank you so much, operator. Thank you, everyone, for joining the call today, and we hope you have an excellent rest of your day. Take care.

Unknown Executive: Well, thank you so much, Operator. Thank you, everyone, for joining the call today, and we hope you have an excellent rest of your day. Take care.

Paula Ragan: Well, thank you so much, Operator. Thank you, everyone, for joining the call today, and we hope you have an excellent rest of your day. Take care.

Speaker Change: Well. Thank you very much operator, thank you everyone for joining the call today and we hope you have an excellent rest of your day take care.

Operator: Thank you, Dr. Ragan. Ladies and gentlemen, that does conclude today's X4 Pharmaceuticals second quarter earnings call. Again, thanks so much for joining us, everyone. And again, we wish you all a great day. Goodbye.

Operator: Thank you, Dr. Reagan. Ladies and gentlemen, again, that does conclude today's X4 Pharmaceuticals second quarter. Your earnings call.

Speaker Change: Thank you Dr. Reagan, ladies and gentlemen, again that does conclude today's X for pharmaceuticals second quarter earnings call again, thanks, so much for joining US everyone and again, we wish you all a great day Goodbye.

Operator: Again, thanks so much for joining us, everyone, and again, we wish you all a great day. Goodbye.

unknown: JAR Visuals JAR Visuals

Unknown Executive: THE END

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Adam Mostafa: So, as of the end of the second quarter, X4 had $169.5 million in cash, cash equivalence, restricted cash, and short term marketable securities. We believe these funds are sufficient to support our company operations into late 2025. I want to note that this projected runway does not include potential future ZOR MD revenue. For the three months and mid-June 30th, we reported revenues of $0.6 million and costs of revenue of $0.3 million related to the sale of ZOR MD.

Unknown Executive: Okay.

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Unknown Executive: Okay.

Adam Mostafa: This cost of revenue includes approximately $0.2 million of license costs, including sales-based royalties and operational milestones capitalized as an intangible asset and amortized over the life of the underlying intellectual property. We would like to note that there was a small amount of inventory stocking at our specialty pharmacy during the quarter, and this is reflected in the sales number. Research and development expenses were $20.9 million for the second quarter, compared to $15.6 million for the comparable period in 2023.

Adam Mostafa: R&D expenses for the second quarter of 2024 included $1.2 million in non-cash expense. Selling in general and administrative expenses were $13.3 million for the second quarter of 2024. This compares to $10.2 million for the comparable period in 2023. SGNA expenses for the second quarter also included $1.2 million of non-cash expense. Finally, we reported net income of $90.8 million for the second quarter of 2024, compared to a net loss of $55.7 million for the comparable period in 2023.

Adam Mostafa: Note that net income in the current period included the sale of the PRV for $105 million, as well as a non-cash gain of $20.2 million related to the fair value remeasurement of the company's class C warrant life.

Paula Ragan: I'll now turn it back to Paula for some concluding remarks. As you can see, all of these accomplishments mark a significant step forward for X4, now a fully integrated bio-pharmaceutical company. As we continue to explore additional uses for and maximize the global potential of mavericks for patients, we look forward to reporting on our sales, regulatory and clinical progress over the coming six to 12 months.

Operator: We'll now open up the call. Thank you, Dr. Reagan. Ladies and gentlemen, at this time, if you would like to ask a question, please press star one on your telephone keypad. You may remove yourself from the queue at any time by pressing star two. Once again, star one for questions.

Kristen Kluska: We'll go first this morning to Kristen Kluska of Cantor.

Mark Baldry: Hey, good morning, everybody, and congratulations on reaching 10 years at the Great Milestone and how better to celebrate with the drug approval this year. So first, I wanted to ask how you believe physicians are going to measure success differently in a commercial setting versus the clinic, Fursal Remdi. Will it be primarily on patient anecdotes, reports around infections, or anything else to consider? Well, thanks, Kristen. I'll turn it over to Mark to give some perspectives on what they're hearing from the field.

Mark Baldry: Yeah, the launch is well underway, Kristen. And what's exciting is that, you know, we're hearing that physicians and patients have been waiting for a treatment that targets the underlying cause of the disease. And so now we have the approval of Zalremdi. It's really opening up these conversations and allowing us to help physicians become aware of the disease, recognize the patients in their practice, and then think about how they can help these patients with Zalremdi.

Mark Baldry: We know from the market research that it's really the impact on infections that drives the value of Zalremdi for physicians, for patients, and payers. So that's really what I think physicians will be looking at as they get more experience with the product. Thank you for that. And can you give us a sense of how partnership discussions X, U, S are going at this stage and just curious, your press release noted some ways you're looking to maximize the vet.

Mark Baldry: So that's the value of Mavericks before beyond Winston German CN. So curious is in these conversations, this has come up with potential partners about ways that they can also potentially utilize the strong and other indications as well. Yeah, so thanks, Kristen. I mean, when we currently think about the FDA approval, there are certain reasons in the world that can leverage that existing approval. So there's some exploration there currently ongoing. I'm Adam can comment more on the more strategic sides of potential collaborations.

Mark Baldry: Yeah, thanks, Kristen. So certainly there is an interest in or have discussions with Paul mentioned ongoing with potential partners will certainly come back and update the market as appropriate if we're going to consummate a transaction. But I think given the recent approval and our recent positive crime of opinion face to data, there's only growing interest in this area. Great, thank you. Thank you.

Ted Tenthoff: We go next now to Ted Tenthoff at Piper Sammler. Great, thank you very much for taking my question. And yeah, it's excited to see some revenues at the company that a lot of work has gone to get that first couple hundred thousand dollars. And I know it'll grow from here. My question had to do with sort of characterizing the patients that are on drug. Can you give us a sense for how many patients are on drug? And is anything about sort of the backlog?

Mark Baldry: And if you're not able to give us direct patient numbers, maybe tell us sort of a little bit about the journey of patients that you're seeing as you sort of get them into commercial use of Zorundi. Thank you. Thanks so much, Ted. So I think, you know, we won't be breaking down any details and continue to report on revenue, but I mean we're overall extremely pleased with just the cascade of commitment that the company has made.

Mark Baldry: And then now we're experiencing the receipt of that commitment in the communities as we head out, but Mark can add some more color and kind of how they're seeing the journey from the diagnosis and education support all the way through patients on drug. Yeah, and I think, you know, the first thing to remember this is an ultra rare disease. And so the key here is to begin to really raise awareness both through the conversations we're having with physicians, our engagement with patient advocacy groups, and also through our digital marketing campaigns because that's where we can get extended reach and we've refreshed our disease awareness campaign.

Mark Baldry: We've also launched our branded Zorundi digital campaigns. So we're really creating this kind of momentum around the conversations. And the patients, you know, as Paul mentioned on the call, all of the patients that were in the open label extension have been prescribed Zorundi here in the US. And so from there, we're just learning more and more about how the patients have been waiting for a treatment that targets their disease and now having those conversations with physicians. Great. That's really helpful. I appreciate that this will color. Thank you.

RK: We go next now to RK at HC Wayne Wright. Thank you. Good morning, Paula and Adam.

Paula Ragan: Congratulations on the 10 years and also the launch. In terms of I know it's early early stages of the launch, but when you go and attend these conferences such as the immunodeficiency conference, how are, you know, what is it that you're hearing, you know, from from physicians and patients at this point in terms of getting ready to take to adopt the drug and also have you learned anything else that needs to be looked at in terms of improving, I'm not saying improving, you know, getting that option to a better level from the get go.

Paula Ragan: So thanks so much, RK. I think, you know, we're very pleased with kind of the cascade of thinking and development. And certainly what we're hearing and Mark will add to this is number one, they're excited about a targeted therapy and I'll even add the oral targeted therapy. It's been a field of injectable treatments with varying degrees of study and rigor are faced with some only successful showing, you know, infection burden reduction across rates, severity and duration.

Paula Ragan: So that is a great story to begin with and a great data set to share. And then in terms of the clinicians, of course, they're all on their own journey, even understanding the disease and then learning about the benefits of the role that will remedy, but I'll turn it over to Mark to add some color to that as well. Yeah, exactly. I think it's, it's really now that we have an approved product.

Paula Ragan: It's really energized positions to go back and really look in there, their practice for these patients who before now have really had no treatment available for their disease. And so now we've actually given them a reason to go back, look for these patients, make the diagnosis and then have a conversation with the patient about well remedy. Fantastic.

Paula Ragan: Then for the SCN indication, so between now and, and if there is an internal analysis for the, for the forward study, is there any other data that will be presented so that we can understand how this program is moving forward? Sure, so as we shared today, we'll be giving the full Phase 2 data set in November of this year. So of course we were extremely pleased with the data set already, but it was interim.

Paula Ragan: So we've already seen durable responses of the drug as monotherapy and then sustained and durable responses in addition to stable GCSF. So the final puzzle piece is just exploring in what circumstances G can get reduced. So that full data set will come in November, which is going to be certainly well timed just before ash will be at the ash meeting to help support the CN community and also get the word further out on our trial enrollment.

Paula Ragan: So it's an excellent timing perspective and then we'll look forward as appropriate to giving updates on how the whole study is going, but certainly that will be a 2025 update. Perfect. Thank you very much for taking my questions Paul. Thanks, okay.

Leah Cann: We'll take our next question now from Leah Can of Brookline Capital Markets.

Adam Mostafa: Good morning. I have a financial question. So Adam, could you help us with the number of fully deluded shares at the end of the quarter including the pre-funded warrants? Sure, thanks. So there's about 167 million basic shares, regular shares outstanding, 33 million pre-funded warrants. So 200 million share equivalents if you will. And then there are 75 million warrants that are cash only, those are divided into two tranches, 130 million tranche, 145 million tranche.

Adam Mostafa: So overall, 275 million in fully deluded shares. There's some historical warrants you'll see later today in our 10 queues that are significantly out of the money are typically not included, but that accounts for a small piece.

Leah Cann: Thank you so much. Most helpful.

Stephen Willey: You will go next now to Stephen Wiley at Stephen. Yeah, good morning. Thank you for taking the questions.

Mark Baldry: Could you maybe just talk a little bit about, and again, I understand that it's early, but the kinetics of the reimbursement process, I know the paperwork that is required sometimes for these higher price rare disease drugs can be a somewhat lengthy process and just curious if you have an early read just based on the limited experience that's far as to what that process might look like from just a timing perspective. Sure, Steve.

Mark Baldry: I mean, I'll turn it over to Mark. I think classic ultra rare disease typically has barriers that a lot of these insurance companies naturally provide, given the magnitude of the drug and its options for patients. But the good news is I think we've had an experience team well ahead of this, so I'll turn it over to Mark to give some insights there. Yes. Paula mentioned during the call, payers are using their standard methods for new drugs and so they're looking at PAs and exceptions process but these are working quite smoothly and patients have been able to access the remedy.

Mark Baldry: Of course we also have our full suite of patient service programs through our X4 Connect which is our patient hub and this ensures that patients are quickly get quick access to the product while we work through the reimbursement. But it's good to see the process working smoothly.

Paula Ragan: Okay, that's helpful and maybe a guidance question, not one related to actual sales but just curious how you're thinking about communicating enrollment progress in the phase 3 forward trial going forward. Should we expect updates in conjunction with certain milestones hit like 50% or is this just going to be kind of a moving target and we'll hear about it when we hear about it. Yeah, thanks, Steve. I think we'll always aim to communicate meaningful progress.

Paula Ragan: Of course, we don't want to make any commitments just because it's a 2025 target. It's almost a year away at this point. However, certainly as we get confidence under our belt here, we'll be able to provide some more specifics but again, I would say that's a 2025 zone to share.

Adam Mostafa: Okay, and then maybe just another financial question for Adam. STA just looks to be down about 25% sequentially, which I guess I wouldn't have anticipated in the context of undertaking a launch. So can you just remind us that there's anything either unique to the one quarter number or this quarter number that impacts that is GNA line up.

Adam Mostafa: Thank you. Thanks, Steve. Yeah, so this second quarter number is probably more representative of a typical recurring figure. The first quarter, as we mentioned last time, a number of one time non-recurring sort of start up related costs to building a sales force and getting a launch underway early in the year ahead of approval. So so that quarter was a little more inflated. If you were on the SCNA line, then I would expect going forward. So that's why the drop looks as does. Very helpful. Thanks for taking the questions and congrats on the progress.

Stephen Willey: Thank you.

David Bautz: We go next now to David Bouts at the Zaxx small gap research.

Paula Ragan: Hey, good morning, everybody. Question on the mechanisms for how prescriptions are filled. Are they filled on a monthly basis? Is it multiple monthly? And then if you could talk a little bit about how revenue was recognized in relation to when those prescriptions are filled? Sure. So I mean, I don't think we have intended to disclose kind of the play by play for our patients. I mean, as we mentioned with our current revenue line, it does blend.

Paula Ragan: Obviously, both an inventory bill as well as revenue recognition for individual patients that are now commercial therapy. So I don't think at this point we're ready to provide any more details, but certainly, you know, we do want, we do understand the market's interest in helping them forecast. So as we head into, you know, later this year, 2025, we'll try to provide a bit more metrics, which really can help provide some benefit to you all as we think about how the product will grow over time.

David Bautz: Okay, thanks for taking the question.

Operator: Thank you.

Paula Ragan: And let me, gentlemen, appears we have no further questions this morning.

Paula Ragan: Dr. Reagan, I'd like to turn things back to you, ma'am, for any closing comments. Well, thank you so much operator. Thank you everyone for joining the call today. And we hope you have an excellent rest of your day. Take care. Thank you, Dr. Reagan.

Operator: Ladies and gentlemen, again, that does conclude today's X4 Pharmaceuticals second quarter. Your earnings call. Again, thanks so much for joining us, everyone.

Operator: And again, we wish you all a great day. Goodbye.