X4 Pharmaceuticals Inc at Canaccord Genuity Horizons in Oncology Conference (Virtual) Transcript

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

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : Good to see you, too. So for investors who are new to the X4 story, can you provide an overview of what X4 does? The diseases you intend to treat, and market opportunities in terms of patient size.

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : Great. Maybe let's talk about WHIM first because this is your lead indication. You have a pivotal trial that's fully enrolled as of the end of last year, so you'll have -- will have pivotal data by year end. Can we talk about what the current standard of care is? Therapeutics that are available, how they're administered, their safety response rate, and where's the unmet need here. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 14, 2022 / 2:30PM, XFOR.OQ - X4 Pharmaceuticals Inc at Canaccord Genuity Horizons in Oncology Conference (Virtual)

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : Okay. Great. Maybe let's talk about the disease in particular. Is WHIM underdiagnosed? What steps are you taking to uncover more patients? And then I'd also love to talk to you about the mutation that you recently found outside of the cluster of mutations that's typically been implicated at WHIM. And so I'd love to hear about what your efforts are to identify additional, previously unknown mutations and patients as well. Thank you.

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : Great. Thanks for that information. Maybe talking about who actually treats WHIM patients, what kinds of physician specialties are treating them? And how is outreach going as you set up these databases and try to find more patients?

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : And how well do you think that these specialties are currently treating them?

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : Great. Maybe now that we've talked about the [dots], can we maybe talk about indications? So you talked about how this affect neutropenia, but this mutation could as well affect the rest of the bone marrow. So it seems like there's a number of diseases that could benefit from cell mobilization. Can we maybe talk about how you prioritize which indications to address next, including the ones for the white blood cells, and when we might see mavorixafor in the clinic for these? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 14, 2022 / 2:30PM, XFOR.OQ - X4 Pharmaceuticals Inc at Canaccord Genuity Horizons in Oncology Conference (Virtual)

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : Great. Thanks. Maybe just following up on that. Is there a potential for -- you talked about G-CSF. Is there an opportunity for you to go into indications where G-CSF is already kind of approved and used? Like chemo, for example.

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : I was actually thinking more on the platelet side, right? Because you could potentially be on the platelets, and there's really nothing -- or platelet growth -- out there. Anyway -- REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 14, 2022 / 2:30PM, XFOR.OQ - X4 Pharmaceuticals Inc at Canaccord Genuity Horizons in Oncology Conference (Virtual)

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : Okay. It seems -- let's see. Can we talk about the competitive landscape next? So CXCR4 dysregulation has been implicated in a number of hematologic cancers. Can you talk about the current CXCR4 competitive landscape? And do you see any threats for indications that you are targeting?

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : Great. Can we actually back up a little bit and talk about the source of mavorixafor, or where you originally got it? This is in the context of the competitive landscape and perhaps some of the safety issues that might be coming up. Can you maybe provide that information for us?

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : Okay, great. And then maybe segueing with what -- your long-term expertise in oral, once daily CXCR4 antagonist, can you talk about what is coming? What your development plans are beyond mavorixafor?

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : Okay, great. Let's talk about the financials a little bit. So as you get closer to a potential Phase 3 data set, regulatory discussions, and approval, how do you think about pricing, considering the potential for chronic dosing? And considering all the indications that you can potentially go into? And what are your intentions to build out a commercial infrastructure both in the US and ex-US? Thank you.

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : Okay, great. And then finally, talk about the balance sheet. At year end, you had $83 million in cash, and we understand that additional funding has been raised since then. You've guided to runway to 4Q 2022, which is when you expect to report Phase 3 top line. What other sources of cash and liquidity do you have available to help cushion this?

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : Okay, great. I had two inbound questions. One, is there opportunity for CXCR4 in primary solid tumors beyond the brain mets? And then, secondly, the stock price has been a decline. What do you think investors are not recognizing about the company?

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : Yeah. You guys do have certainly a lot of news flow coming up and pretty important inflection points for each of your programs.

Question: Arlinda Lee - Canaccord Genuity LLC - Analyst : So with that, I think I'd like to thank everyone for joining us. Thank you, Paula, for sharing your insights on X4 and all the things that are coming. And with that, I think we'll disconnect. Thanks very much, everybody.