X4 Pharmaceuticals Inc Q2 2021 Earnings Call Transcript

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

Question: Ellen Sands - Stifel, Nicolaus & Company, Incorporated - Analyst : This is Ellen on for Steve. Congrats on all the progress. So you mentioned that you now have 23 patients enrolled in the Phase 3 WHIM study and that there is complications undergoing screening. So I was just wondering, could you clarify does that 23 member include those patients that are currently undergoing screening or are there additional patients in addition to these 23 that you mentioned that are in the process of being enrolled currently? And then kind of related to that, so I think the trial, the protocol allows for up to 28 patients enrolled. Do you expect to enroll 28 or will it be a little bit less than that? And then I have a follow-up.

Question: Ellen Sands - Stifel, Nicolaus & Company, Incorporated - Analyst : Okay. Great. And then can you provide any details as to when we might expect to see the new data from the open-label extension portion of the Phase 2 WHIM trial and then also those updates regarding patient prevalence? And then maybe to that latter point, I understand there are a few different efforts underway with regards to patient identification, including the Invitae collaboration, partnerships with patient advocacy groups, et cetera. So will that prevalence update be from kind of a holistic update from all of those efforts or will it be one or two that are specifically the focus for that update?

Question: Ellen Sands - Stifel, Nicolaus & Company, Incorporated - Analyst : Yes, and then just the timeline which you just answered there, so.

Question: Trevor Allred - Oppenheimer & Co. Inc. - Analyst : I wanted to ask about the SCN trials. Do you know what mutation types you have enrolled so far and what mutation types you expect to enroll once you're completed there?

Question: Trevor Allred - Oppenheimer & Co. Inc. - Analyst : Okay. Great. And in terms of what we've seen so far with Waldenstrom's and the positive results we're seeing so far, do you have any expectations already for what the next trial for Waldenstrom's might look like?

Question: Swayampakula Ramakanth - H.C. Wainwright - Analyst : This is RK from H.C. Wainwright. It's great to see all the progress. A quick question on the variant and Paula, you said your research folks are looking at WHIM variants. What is -- can you expand on that, please?

Question: Swayampakula Ramakanth - H.C. Wainwright - Analyst : So it is potential that some -- there could be patients that are not really being diagnosed as WHIM because they don't have some of the typical symptoms that have been patient as we currently define it as have. Is that right? Is that one of the --

Question: Swayampakula Ramakanth - H.C. Wainwright - Analyst : One of the --

Question: Swayampakula Ramakanth - H.C. Wainwright - Analyst : Yes. Great. And then on the Waldenstrom's, in terms of the ongoing Phase 1b study, I don't know, Paula, if you stated anything about the timing on the next update.

Question: Zegbeh Jallah - ROTH Capital Partners - Analyst : Paula, I think you mentioned that you guys had initiated some commercial activities and I feel like you've been doing a lot of work with patient identification and then just really being involved with a lot of the patient groups. So I'm just wondering what steps are you guys now taking and then how are you thinking about kind on the rolling out of activities because it is a little bit early. And then the last bit, if you can just comment on how you also think about cost synergies because I think that was something else that you mentioned at one point in terms of commercialization of mavorixafor?

Question: Zegbeh Jallah - ROTH Capital Partners - Analyst : Cost synergies, cost synergies. Sorry, my reception is kind of poor.

Question: Zegbeh Jallah - ROTH Capital Partners - Analyst : And then just a quick follow-up here on the chronic neutropenia. I was just wondering how large could this possibly be in terms of expansion.

Question: Mayank Mamtani - B. Riley Financial - Analyst : Congrats on all the progress. So just one quick question on the recently published BMS CXCR4 antibody dataset. I think that was published in Blood last week by Dr. [Treon] also. Can you maybe speak to how that dataset you look at in terms of mechanistic validation, but also the kind of bar set for mavorixafor as you think of advancing this to late-stage development next year?

Question: Mayank Mamtani - B. Riley Financial - Analyst : Great. And then on the SCN Phase 1 initial dataset you're thinking of putting out in fourth quarter 2021, can you maybe just give us a little bit color on what sort of dose levels you're able to go up to in this study design? And I think you mentioned that there are some ongoing trials that similarly could have cross read on your approach, including in larger chronic neutropenic population. So can you maybe expand on that a little bit more, Paula?

Question: Unidentified Participant - Cowen - Analyst : I have a quick question about the Waldenstrom's study. Approximately how many patients will be evaluable by the next update in ASH and can we expect to see some clinical response data at that update?

Question: Unidentified Participant - Cowen - Analyst : Just a quick follow-up. What's your current thinking about what you would need to see to move into larger studies?

Question: Ben Shim - Canaccord - Analyst : Paula, I just wanted to confirm, I might have misheard, but for the SCN, looking at I guess the larger moderate population, that is going to be a trial amendment as opposed to a brand-new 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. AUGUST 03, 2021 / 12:30PM, XFOR.OQ - Q2 2021 X4 Pharmaceuticals Inc Earnings Call

Question: Ben Shim - Canaccord - Analyst : Okay. Great. I know it's a little early, but can you may be shed a little light on what you think the FDA might be thinking as far as approvable endpoints for a potential pivotal trial?

Question: Ben Shim - Canaccord - Analyst : Okay. And I just wanted to ask an obvious clarification question. I think you had mentioned in your SCN comments that you saw kind of a deepening of response in neutrophils and I just wanted to confirm that's with patients who are on drug, right?