X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q4 2022 Earnings Call Transcript

Murray Stewart: Yes, so I mean, in Phase 1, we were able to look at the different populations congenital idiopathic and cyclical. And for a big registrational study, we’d hope to be able to include similar groups, and you can include them in the one study by stratifying. So in other words, you get balanced across the different groups. So rather than do three separate studies, you can do them all in one study by accounting for stratification.

Divya Rao: Okay that’s helpful. And then, I guess, in terms of the secondary endpoints that you plan to present, from the Phase 3 trial sometime in Q2, I guess what infection rates do you think physicians and patients will be clinically meaningful?

Paula Ragan: Sure, I’ll start and then certainly ask Murray and others to chime in. But, our Phase 2 data, which align with breakthrough therapy designation in WHIM syndrome demonstrated a reduction of infection rates of about 50% after a year, we’re certainly hoping that type of direction continues, we’re extremely excited about the Phase 2 data, we designed a robust study for the Phase 3, and not only, of course, infection rates. But there’s a whole host of things that we’ve mentioned in terms of duration, severity, type, we look forward to sharing the totality of the data. But, Murray, if you want to chime in any further, that would be great.

Murray Stewart: The only thing to add in. So, in the WHIM population, you might expect people to have an annualized infection rate of about four. And that’s a mean, some people get more than five infections and some people get two or three infections, but the mean is about four. And obviously, from our Phase 2, we saw about the 40% reduction. Bear in mind that the study we did was under COVID. And there have been concerns about would we see less infections in COVID, that when we present the data, we’ll be presenting the data looking at the annualized infection rate, the (ph), and we’ll look at the different components of infection, including, as Paula mentioned, in the call, severity, duration, repeated infections, as well as warts vaccine titers, quality of life and safety.

Divya Rao: That’s helpful. Thank you.

Operator: Our next question comes from Ted Tenthoff of Piper Sandler. Please go ahead.

Edward Tenthoff: Great, thank you very much. Shaping up to be a very exciting year. Two quick questions. And I’m assuming that maybe you’re waiting for acceptance, but what are the conferences you anticipate presenting data at, for the Phase 3 data in the second quarter? And when it comes to the chronic neutropenia readout, is it more than likely that’s going to be a press release and followed up biomedical meeting, or are you targeting a specific medical meeting? Thank you so much.

Paula Ragan: Hi Ted, thanks for the great question. So we’re currently aiming to present either at CIS, which is in the second or third week of May, or EHA, which is in the second or third — second week or so of June. We are still waiting to hear, so we’re looking forward to kind of refining that type of information. So please stay tuned. And we’ll try to update as soon as possible. And then, in terms of the CN data, we would expect to kind of provide some additional context around it from that just a press release, we certainly think this will be an exciting data set to inform both the KOL and the investor communities around. So we think we’ll provide a more robust type of format, not likely linked to a individual conference, but perhaps an investor or R&D day on behalf of X4.

Edward Tenthoff: Great, that’s very helpful. I am looking forward to the data, and the regulatory filings. Thank you very much.

Paula Ragan: Thanks so much, Ted.

Operator: Our next question comes from Mayank Mamtani of B. Riley Securities. Please go ahead.