X4 Pharmaceuticals Inc at Cantor Fitzgerald Global Healthcare Conference Transcript

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

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Awesome. So, X4 is striving to be one of the pipeline in a platform stories in biotech, looking to understand how one drug candidate could potentially be impactful across a number of diseases. So, to start, could you please walk us through the role of CXCR4 has in retaining neutrophils in the blood marrow? Why is this problematic in the diseases that you're focused on?

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Okay, thank you. So, the last year has been important for you in answering two key questions for your lead program, showing that first, Mavorixafor can increase total white blood cell counts, and then second, this also correlates with reduction, severity, and time of infections. So, could you please walk us through what you believe to be the main takeaways from the Phase 3 WHIM syndrome trial? 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 26, 2023 / 8:55PM, XFOR.OQ - X4 Pharmaceuticals Inc at Cantor Fitzgerald Global Healthcare Conference

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Yeah, I was at the CIS conference. I got a lot of great receptions, good data.

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : So how are you thinking conservatively about the market potential for WHIM syndrome? And walk us through some of the initiatives to find new patients. And then also, if you want to touch on the rare pediatric disease designation.

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Okay, thanks. Wanted to move on to chronic neutropenia. So first, why do you believe that we haven't really seen much innovation in over 30 years,

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Okay, thanks. So there's some disappointment from the market that your recent CN update had three patients' worth of data, albeit there was excitement about what those three patients showed. So while recognizing that the CN landscape is much larger than WHIM syndrome, can you comment on some of the enrollment trends you've been seeing here?

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Okay. Yeah, let's talk about the data in a little bit more detail. So the goal of this recent study for you was essentially to show that chronic dosing can lead to durable results after you previously reported some proof-of-concept study in a single-dose study where you were able to increase these blood cells. So how did this data set give you confidence in Mavorixafor's added effects on top of G-CSF?

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Okay, thanks. And it sounds like you're nearly there in terms of the Phase 3 study design. So what has already been discussed with the agency and what remaining steps or boxes do you have left to check off?

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Okay, thank you. And then the next update clinical-wise could come later this year, potentially at or around the ASH conference. So can you help us set expectations about what data you could report on or during this event? And what essentially are you looking to see that would be successful for you?

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Okay. And there's been a lot of questions around the true size of the CN market. In your August update, you helped better frame the initial market opportunity for us. So could you please walk us through some of these estimates and why these 15,000 or so initial patients that you've identified may serve as the highest unmet market need initially?

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Okay. And I think one item that went underappreciated is that you conducted a lot of research to understand infection events and mortality in these patients. Again, the CIS conference was awesome. Can you walk us through what a typical CN patient profile may even look like and what would ultimately be clinically meaningful for this particular population to see? 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 26, 2023 / 8:55PM, XFOR.OQ - X4 Pharmaceuticals Inc at Cantor Fitzgerald Global Healthcare Conference

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Okay. And I started the conversation by talking about this whole pipeline in a platform notion, right? And I understand these are the two key priorities for the company, but I guess maybe mid- to longer term, like where else can this potentially work?

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Okay. And you've guided cash could fund operations into 2025. And also how might a potential PRV sale, if you're granted one with an approval and any early sales potentially change these projections? Is that included in the?

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Okay, just wanted to pause, see if there's any questions from the audience. Okay, awesome, great. Well, I wanted to turn the floor back to you to see if there's anything I didn't ask you that I should have asked or any other key takeaways you want our audience to walk away with today.

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Well, thank you both very much for a very thoughtful discussion. Lots to look forward to in the next few months.