X4 Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference Transcript

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

Question: Matthew Harrison - Morgan Stanley - Analyst : Paula and Adam, thanks for being here. Appreciate it. I thought maybe why don't we just start out a little bit with -- obviously you're developing an asset for a variety of neutropenic conditions. Maybe you could just talk a little bit about where the drug came from, what's the mechanism, and sort of how you've decided on the indications you're going after.

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay, perfect. And just for people who aren't familiar. So how does CXCR4 work in terms of the mechanism? And I think people when they hear novel small molecule mobilizes cells, maybe they thinking are there safety risks that they should be worried about. So just talk about the mechanism there. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 13, 2023 / 11:30AM, XFOR.OQ - X4 Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay. Super. Super. So you obviously indicated your first indication is WHIM. Obviously, rare disease, maybe people don't know so much about WHIM. So maybe just tell us a little bit about what is WHIM and what's that market size.

Question: Matthew Harrison - Morgan Stanley - Analyst : Great. Yeah. And so you talked about Phase 3 data. So maybe just remind people what the data was, what kind of regulatory endpoints you needed to meet here, because I'm sure there's some uniqueness given the size of the population.

Question: Matthew Harrison - Morgan Stanley - Analyst : And so in terms of next steps there, I know you recently filed in the US. So how should people think about the timeline and the path to market in the US?

Question: Matthew Harrison - Morgan Stanley - Analyst : And in terms of commercialization, you plan to commercialize this yourself. What sort of preparations are you doing?

Question: Matthew Harrison - Morgan Stanley - Analyst : And did you run a natural history study or are there anything you've done to sort of prepare the market or sort of build a bolus of patients at launch that you might consider to be appropriate targets?

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay. Okay. Good. Outside the US, maybe just remind us what the plans are for WHIM outside of the US.

Question: Matthew Harrison - Morgan Stanley - Analyst : And from a regulatory standpoint, are you waiting to sort of decide on that before you file in Europe? Or what's the filing plan, and how much regulatory dialogue have you had in Europe?

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay, great. You obviously highlighted the larger indication is chronic neutropenia. Maybe just describe for people, I think they hear that, and they say, well, what does that patient population look like? How do you define the chronic neutropenic population? So maybe just talk a little bit about those patients. What sort of underlying conditions they have that makes up that population?

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay.

Question: Matthew Harrison - Morgan Stanley - Analyst : And just from a mechanism standpoint, because you said these patients are idiopathic, like, do you think their underlying disease is sort of independent of mechanism? Or how should people think about the biology of the disease of those patients?

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay. Okay. Great. So I guess then building on that, you have a couple of pieces of evidence to support what you're doing in the chronic neutropenic population. Obviously, [DAY] and WHIM, they're not exactly the same. You have some Phase 1 data, and they recently released some Phase 2 data. So maybe talk to everybody about what you see as the body of evidence that sort of supports what you're doing in this population.

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay. Okay. And maybe we just spend a couple of moments on the handful of patients that you've disclosed already. So I guess the first question is I think you said we're going to get more data maybe at ASH this year. So maybe just describe for people what to expect in terms of additional follow-up from those patients

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay. But I mean, is there an expectation that we'll see more patients, or it's still a little bit TBD?

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay. Perfect. And so then, I guess the second question is how to think about durability because, obviously, that's an important component for these patients, I think. I can't remember exactly how much follow up, but you had different amounts of follow up for each of those patients you disclose. So from your point of view, how much durability do you think you need to feel good about the long-term prospects? And then secondarily, what do you think the regulators want to see in terms of durability?

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay. And then I think one other area that probably caused some debate among investors around the data was obviously when you titrated off G-CSF, neutrophil levels came back down a little bit. And so I think people were trying to figure out where do they stabilize. So just how do you think about that and what's your level of confidence that you are able to stabilize people above an appropriate level?

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay, great. Regulatory. So obviously, you've got some pretty interesting data. How do you get it approved in CN? What does the pivotal program look like?

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay. Okay. And in terms of the neutrophil endpoint, I mean, is that like a certain amount of time above a certain amount, or how should people just think about what that will look like?

Question: Matthew Harrison - Morgan Stanley - Analyst : Got it. Got it. All right, super helpful. So maybe you could talk a little bit about the market. You mentioned, I think, 15,000 patients. How much work have you done on that figure? What's the range of that figure, and how do you think about getting ready for commercialization?

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay. Okay. And then commercialization, again, it sounds like WHIM obviously gives you a foothold in the US, and so presumably you'd expand that to launch in chronic neutropenia. Is that the current plan?

Question: Matthew Harrison - Morgan Stanley - Analyst : And then as you think -- you obviously discussed the potential for licensing or partnering outside the US. I mean, is that a package where you'd look at WHIM and chronic neutropenia together, or are they separate? Just how you think about commercialization outside the US.

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay. Okay. Super. We got a handful of minutes left. So maybe just talk about other areas you might think potential label expansion.

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay. Super. And then, Adam, maybe can you just talk about financial position? Obviously, biotech companies need money all the time. So how are you financed right now and where does that take you in terms of run rate?

Question: Matthew Harrison - Morgan Stanley - Analyst : Okay, great. Well, Paula and Adam, thanks for being here. Appreciate the time. Thank you very much.