X4 Pharmaceuticals Inc Q3 2023 Earnings Call Transcript

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

Question: Eva Privitera - TD Cowen - Analyst : Hi. Good morning, and thanks for taking our questions. I was wondering if you could talk about the CN patients who are on G-CSF but still neutropenic. What are the reasons for their inadequate response? Is it mostly due to intolerance, or are there reasons they're refractory or have inadequate response? I guess I'm trying to get a sense of how heterogeneous this patient population is.

Question: Eva Privitera - TD Cowen - Analyst : Great. Thanks for that. And second, on WHIM, just based on all your commercialization and market-building efforts in WHIM, is that acceptable with patients at launch?

Question: Eva Privitera - TD Cowen - Analyst : Great. Thank you so much for taking my questions. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 09, 2023 / 1:30PM, XFOR.OQ - Q3 2023 X4 Pharmaceuticals Inc Earnings Call

Question: Stephen Willey - Stifel Nicolaus and Company, Incorporated - Analyst : Good morning. Thanks for taking the question. Can you say whether or not, I guess, you've brought any additional sites online in the Phase 2? I think you had previously said you were at around six. And then how many sites are you planning for the Phase 3?

Question: Stephen Willey - Stifel Nicolaus and Company, Incorporated - Analyst : Okay, that's helpful. And then with respect to the Phase 3 protocol in chronic neutropenia, I guess, how are you standardizing G-CSF reduction, withdrawal? Is that just investigator subjectivity? And then, is there a way that you're going to try to statistically quantify that at all?

Question: Stephen Willey - Stifel Nicolaus and Company, Incorporated - Analyst : Okay. Thanks for taking my questions. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 09, 2023 / 1:30PM, XFOR.OQ - Q3 2023 X4 Pharmaceuticals Inc Earnings Call

Question: Edward Tenthoff - Piper Sandler Companies - Analyst : Thank you very much, and good morning, everyone. Two questions, if I may. Firstly, I think I should know this, but is there any open-label extension work being done in WHIMs? In other words, are there currently any patients on mavorixafor? And then the second question, I think you mentioned, if I heard you correctly, that there will be the three patients -- more data on those three patients from ASH. Could there be more patients just because of additional time between abstract submission and the actual presentation? Thank you.

Question: Edward Tenthoff - Piper Sandler Companies - Analyst : Great. Awesome. I'm looking forward to that. And can you remind me, I know the majority of patients rolled over, how many patients are in the OLE for WHIM? Thanks.

Question: Edward Tenthoff - Piper Sandler Companies - Analyst : Thanks, Paula.

Question: Andy Fleszar - B. Riley Securities - Analyst : Hey, good morning. This is Andy Fleszar on for Kalpit. Thank you for taking the questions. Are you able to give any additional granularity on the timing of the next comprehensive update for the 15 patients, or if there's a certain amount of follow-up that you'll be looking for before presenting the results?

Question: Andy Fleszar - B. Riley Securities - Analyst : Okay. And then as a follow-up to that, do you anticipate the Phase 2 data to be enriched for cyclic, congenital, or idiopathic CN?

Question: Andy Fleszar - B. Riley Securities - Analyst : That makes sense. And then one final question from us. From your market research, can you speak about what level of GCF reduction is clinically meaningful to patients and prescribers? Obviously, more reduction is better, but any color that you can provide on what success might look like would be helpful.

Question: Andy Fleszar - B. Riley Securities - Analyst : Sounds great. We look forward to seeing you there. Thank you. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 09, 2023 / 1:30PM, XFOR.OQ - Q3 2023 X4 Pharmaceuticals Inc Earnings Call

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Hi. Good morning, everyone. Thanks for taking the question. When I look at your finalized Phase 3 CN study design, in a lot of ways, as you mentioned, it really does mirror what the trial looked like for WHIM, which, of course, was successful. So can you talk about how you think that gives you the confidence in this particular study and any differences we should really be thinking about related to the mechanism in these subset of patients versus WHIM?

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Okay, thanks for that. And it's nice to see the uptick in enrollment in this update. Can you maybe talk about what you think some of the factors are that was originally slower than expected now that you've seen this uptick? So were it things like seasonality, and again, how this also helped for planning for Phase 3? Thanks again.

Question: Kristen Kluska - Cantor Fitzgerald - Analyst : Yes, thank you.

Question: Swayampakula Ramakanth - H.C. Wainwright & Co., LLC - Analyst : Thank you. Good morning, Paula and Adam. A lot of my questions have been answered. Just looking into the Phase 3 study in CN disorders, what's the label you're looking for once we get into the application stage? And also, do you foresee either increasing the size of the trial as the trial progresses so that you maintain a certain number of patients in these individual populations so that you can get a broad label?

Question: Swayampakula Ramakanth - H.C. Wainwright & Co., LLC - Analyst : Okay, thank you. Thanks for taking my question.

Question: David Bautz - Zacks Small-Cap Research - Analyst : Hey, good morning, everyone. Thanks for the update this morning. There's just one question for me. I'm curious, what type of doctor ends up typically making the diagnosis for WHIM? And I guess what I'm trying to get at is, how are you sure that you're reaching the correct physician population with your outreach program?

Question: David Bautz - Zacks Small-Cap Research - Analyst : Okay, great. Thanks for that.