X4 Pharmaceuticals Inc Q2 2023 Earnings Call Transcript

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

Question: Stephen Willey - Stifel Nicolaus - Stifel Financial Corp - Analyst : Yeah. Good morning, guys. Thanks for taking my questions, and congrats on the quarter and the updated data. I guess, can you remind us within the Phase 2 CN protocol if there's a threshold ANC at which the investigator makes the G-CSF tapering decision? And I guess, does that have to be a structured taper in terms of a 50% reduction, a 25% reduction, and then fully off? Or could patient one just had been taken off G-CSF at any time point for the subjective assessment of the investigator?

Question: Stephen Willey - Stifel Nicolaus - Stifel Financial Corp - Analyst : Okay. Then I guess I'll follow up with the obvious question. I mean, you've seen a little bit of a stepwise reduction in ANCs as the tapering has increased. I guess, what's your level of confidence that, I guess, at the next time point, month five, month six, that you're still going to be comfortably above the normal range in terms of ANC with this patient who is on mavorixafor?

Question: Stephen Willey - Stifel Nicolaus - Stifel Financial Corp - Analyst : Okay. And then maybe the last question -- yeah. Go ahead.

Question: Stephen Willey - Stifel Nicolaus - Stifel Financial Corp - Analyst : Okay. And then maybe just last quick question. I guess, can you just speak to how many patients you've enrolled to date? I guess, how many of those patients will be included in this next disclosure? Just trying to get a sense as to how many patients and how long of a follow-up we'll see. Thanks. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. AUGUST 10, 2023 / 12:30PM, XFOR.OQ - Q2 2023 X4 Pharmaceuticals Inc Earnings Call

Question: Stephen Willey - Stifel Nicolaus - Stifel Financial Corp - Analyst : All right. Thanks for taking my questions.

Question: Kristen Kluska - Cantor Fitzgerald & Co. - Analyst : Good morning, everybody, and congrats on all these updates today. First, I wanted to ask, at CIS this year, you had a pretty detailed poster on understanding the real-world analysis of these patients. So first, can you help contextualize how these findings also help you in terms of your Phase 3 protocol design and what your expectations at baseline might be for some of these patients? And ultimately, what would be deemed clinically meaningful here?

Question: Kristen Kluska - Cantor Fitzgerald & Co. - Analyst : Thank you for that. And I recognize that the data update today is only a few months of follow-up. But can you give us some sense of that baseline? Were these patients experiencing infections? And then also, ultimately, how long in follow-up do you think that you'll be able to see some general early trends to be able to determine if the drug does have an impact on infections? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. AUGUST 10, 2023 / 12:30PM, XFOR.OQ - Q2 2023 X4 Pharmaceuticals Inc Earnings Call

Question: Kristen Kluska - Cantor Fitzgerald & Co. - Analyst : Okay, thanks. And then last question for me. I'm going a little bit into the weeds here. But how are you specifically going to define recurrent and severe infections for the Phase 3 trial? Is there a threshold, a number, that they have had to experience in the past? Or how are you thinking about that definition?

Question: Kristen Kluska - Cantor Fitzgerald & Co. - Analyst : Okay, got it. Thanks for taking my questions and hope to see you in San Diego.

Question: Ted Tenthoff - Piper Sandler & Co. - Analyst : Thank you. Good morning. And I can really sense the excitement and understand why with some of the progress going on. Just wanted to tighten up a little bit on the NDA. What remains to be done? Are there any inspections that need to occur? And I mean, maybe it's early to tell. But with a rapid review cycle, would you anticipate an AdCom? Thanks for reviewing all that.

Question: Ted Tenthoff - Piper Sandler & Co. - Analyst : Excellent. Thank you. And on the AdCom?

Question: Ted Tenthoff - Piper Sandler & Co. - Analyst : Awesome. Great. Well, best of luck.

Question: Marc Frahm - Cowen and Company, LLC - Analyst : Thanks for taking my question. Congrats on the data. And obviously, a nine-fold change seems quite significant in ANC. But can you just review the kind of enrollment process and how many measures leading up to that baseline measure of ANC you were able to capture? And can you speak to the variability you're seeing in the one patient presenting, but maybe also more broadly in the trial?

Question: Marc Frahm - Cowen and Company, LLC - Analyst : Okay, that's great. And then maybe on that point of that being an important goal, do you expect an end point around that to be -- maybe not the primary, but in your Phase 3 to be formally assessed -- G-CSF sparing ability?

Question: Marc Frahm - Cowen and Company, LLC - Analyst : Okay. Thank you.

Question: Trevor Allred - Oppenheimer & Co. Inc. - Analyst : Hey, good morning. Thanks for taking the question. Could you just talk a bit about the FDA feedback on the Phase 3 trial design? For example, I mean, given the magnitude of benefit that we've seen, do you guys think that you need a 12-month trial? Is that something from the FDA? And then can you also talk about the FDA's thoughts on the tapering inclusion?

Question: Trevor Allred - Oppenheimer & Co. Inc. - Analyst : Okay. Thanks.

Question: Swayampakula Ramakanth - H.C. Wainwright & Co., LLC - Analyst : Thank you and good morning, Paula and Adam. Most of my questions have been answered. But in general, with the data that we've seen so far with mavorixafor, what additional indications could you highlight as a possible way of label expansion from here, beyond the CN?

Question: Swayampakula Ramakanth - H.C. Wainwright & Co., LLC - Analyst : Thank you. Thank you for taking my questions.