X4 Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Great, thank you. So just to kind of level set, mavorixafor, the generic name for XOLREMDI, can we just talk about the mechanism of action, specifically how does CXCR4 antagonism impact the bone marrow, leading to increased circulation of neutrophils and lymphocytes?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Great. So pivoting to the recent approval of XOLREMDI for WHIM syndrome in April 2024, congratulations. Let's -- we can start high level, talking about a brief overview of WHIM syndrome, severity, incidents, prevalence, et cetera.

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Targeted treatment and expanding on that, you're actually addressing the underlying cause of disease, and that's included in the label. How -- could you expand a bit on how that's differentiated?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Great. And diving a bit deeper into the patient profile. I imagine internally, you have estimates on the penetration rate of the 1,000 patients -- US patients. But can you talk a bit about is there a spectrum of severity? What percentage do you think will be really first comers to this medication?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Okay. Now, pivoting to the launch. Can you talk a bit about the price, payer coverage, what kind of sales force you're putting together?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : And are these patients -- is there a pretty good understanding of where the patients are going to seek treatment, what the carriers like geographies breakdown?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : And as you said previously, this is the first approval for WHIM syndrome. But is there -- how are these patients currently being addressed? What's the competitive landscape look like? I know IVIG, G-CSF, and then, of course, plerixafor, but that's -- I don't think that's used specifically for WHIM syndrome. Similar mechanism. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 04, 2024 / 6:35PM, XFOR.OQ - X4 Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Okay, that's helpful. Last question on WHIM is, whether you're on track to submit your European MAA by early 2025. Is everything on track there?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : And how does the prevalence look in Europe?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Okay, that's helpful. I guess the last question, but this is also pivoting to chronic neutropenia, which is a much larger indication. You also have studies underway there. How did the results in WHIM syndrome inform your approach to testing mavorixafor for chronic neutropenia?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Okay. Could we talk about the target patient profile, severity of these patients, prevalence, anything there for chronic neutropenia?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Okay. Before jumping into the data you've presented so far, could you just talk about how our G-CSF is currently used and as you talked about the unmet need there?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Okay, great. Recently, you presented follow-up Phase 2 data, if I'm correct. Maybe initially, we can talk about the Phase 2 trial design, enrollment criteria, the severity of this patient population, and then just data to date?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Eight patients, okay. And could you kind of set expectations going into the November readout? What are the key efficacy or clinically relevant endpoints that you're looking at? I know ANC, change in ANC, is there a threshold you're hoping to hit there?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : So the patients who are on G going into the study where you'll see eight patients' worth of data in November. What was their baseline neutrophil counts approximately?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : And the physicians, it's up to them in regards to how they'd like to titer down the G?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : And I imagine you've spoken to many KOLs. Do you think there's a disconnect between investor expectations or how investors are digesting these data and what KOLs and physicians are thinking?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Okay. I think that's a good pivot now. We can now talk about the Phase 3 trial, how the Phase 3 kind of informed the Phase 3 design moving forward as either monotherapy. Basically thought process behind the pivotal Phase 3 trial?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : So there's not a dedicated monotherapy arm?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Okay, great. And how is enrollment going? If you can talk about that at all. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 04, 2024 / 6:35PM, XFOR.OQ - X4 Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : And then the -- there's no interim readout. When will be the final dataset come?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Okay, great. And then on your second-quarter earnings call, you reported $170 million in cash, cash equivalents, which is expected to provide a runway into late '25. Could you just walk me through a bit of your thoughts on capital allocation? You have the launch going on, the Phase 3, finishing up the Phase 2. Any thoughts around financials?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : So in regards to XOLREMDI guidance, revenue expectations, consensus, any thoughts on that, basically? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 04, 2024 / 6:35PM, XFOR.OQ - X4 Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Great. In the last few minutes, is there anything else I missed? Anything you think investors should be focusing on to get a better understanding of the X4 story?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : I guess last question, IP protection. How does that look going out?

Question: Max Skor - Morgan Stanley & Co. LLC - Analyst : Well, great. Thank you very much. I really appreciate it.