X4 Pharmaceuticals Inc Q1 2021 Earnings Call Transcript

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

Question: Stephen Willey - Stifel - Analyst : Hey, good morning. Thanks for taking the questions. Paula, I was maybe wondering if you can just kind of frame up the expectations a little bit with respect to, I guess, what kind of data we might see in the abstract when that gets released? I think it's next week and I guess how that may differ from what we could see at EHA. It sounds like you said specifically that we'll be seeing data from the low and mid-doses. Have you dose escalated patients up to that 600 mg dose yet for the escalation protocol?

Question: Stephen Willey - Stifel - Analyst : Okay. And, I guess, have you internally made the decision as to whether or not you're going to increase the enrollment up to -- up to 18 or I guess do you think that you're going to get a sufficient amount of data in these first 12 to kind of understand what your recommended Phase 2 dose needs to be?

Question: Stephen Willey - Stifel - Analyst : Understood. And then just lastly, I'm just curious as to where things stand on severe congenital neutropenia which I don't think was a part of your prepared commentary or if it was, I may have missed it.

Question: Stephen Willey - Stifel - Analyst : Understood. Thanks for taking the questions and congrats on the progress.

Question: Marc Frahm - Cowen - Analyst : Thanks for taking my questions and congrats on all the progress across the multiple programs. Paula, can you remind us the different doses in the Waldenstrom's trial, just the kind of relative levels of CXCR4 inhibition that you're expecting based on the [kind] of prior work you've done with the molecule?

Question: Marc Frahm - Cowen - Analyst : I guess kind of on traditional metrics of like IC50, IC90, like what type of levels are we at?

Question: Marc Frahm - Cowen - Analyst : Okay, that's helpful. Thanks. And then, maybe on WHIM, can you, Paula, give an update on to where you are more generally on patient identification efforts, kind of where registries are at these days?

Question: Marc Frahm - Cowen - Analyst : Okay, great. That's very helpful. Thanks.

Question: RK Ramakanth - H.C. Wainwright - Analyst : Thank you. Good morning, Paula. A quick question on mavorixafor. Most of my questions on the current expectations on the EHA, I got them. But beyond what's going on right now in the Phase 1b study, do you -- do you have a general outline as to how you would be going forward with this into the Phase 2 study and also could you kind of give us a rough timeline as to when that could happen?

Question: RK Ramakanth - H.C. Wainwright - Analyst : Thank you. The second question is on the 4WHIM trial. We understand you're going to give us a little bit of an update regarding enrollment in the mid-year. However, considering this population and your experience so far of the trial, again similar question as to when we could see the completion of the study or would we get that update also during the major enrollment of data as to how we should think about your complete -- not only you're completing the study but your conversations with the regulators?

Question: RK Ramakanth - H.C. Wainwright - Analyst : Thank you. Thank you, Paula.

Question: Sahil Kazmi - B. Riley - Analyst : Hi. Good morning, team. This is Sahil Kazmi for Mayank. Thanks for taking our questions and congratulations on all the progress. Maybe a brief one on the WHIM patient enrollment and how that sort of tracking, has an influence by your efforts in patient identification and then also on that same train of thought, how the patient identification efforts have had synergies on the SCN side and how you might think that influencing later stage trial as well?

Question: Sahil Kazmi - B. Riley - Analyst : Yes, absolutely, that's really helpful. And then just maybe one more quick one on WHIM is, could you provide kind of studies that you're able to disclose a bit more color on what we might learn from incremental sort of data from this Phase 2 open-label extension that you plan to share at the end of the year, whether it be from a safety or sort of durability of response angle?

Question: Sahil Kazmi - B. Riley - Analyst : Great. I really appreciate the time. Thanks for taking our questions.

Question: Arlinda Lee - Canaccord Genuity - Analyst : Hi guys, thanks for taking my questions. You guys alluded to [nearing] dose limiting toxicities. Could you talk a little bit about what you would expect this might be and then can you comment on whether you expect dosing to be the same across the various disease indication? 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. MAY 06, 2021 / 12:30PM, XFOR.OQ - Q1 2021 X4 Pharmaceuticals Inc Earnings Call

Question: Arlinda Lee - Canaccord Genuity - Analyst : Thank you.