X4 Pharmaceuticals Inc at Canaccord Genuity Growth Conference Transcript

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

Question: Ben Shim - Canaccord Genuity - Analyst : For those who are new to the X4 story, can you maybe give us a brief background on what WHIM -- that's W-H-I-M -- syndrome is and how your drug, mavorixafor, is positioned to help this patient population?

Question: Ben Shim - Canaccord Genuity - Analyst : Great. Can you share with us what mavorixafor's mechanism of action is, and how this lines up with the pathophysiology of WHIM syndrome? 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 11, 2022 / 7:30PM, XFOR.OQ - X4 Pharmaceuticals Inc at Canaccord Genuity Growth Conference

Question: Ben Shim - Canaccord Genuity - Analyst : Okay. Great. The Phase 2 data shared at EHA 2020 is very compelling with, I guess, really robust clinical activity. Can you remind us of some of the clinical activity you saw there and where these patients undergoing any other treat?

Question: Ben Shim - Canaccord Genuity - Analyst : Great. I guess question for Paula, do you see IVIG in mavorixafor being used in the same time? And what about G-CSF? And based of your current data, how do your results compare to other therapies used in the population?

Question: Ben Shim - Canaccord Genuity - Analyst : Great. What are some of the favorable regulatory designations that are available for mavorixafor? And can you comment on the safety data to date?

Question: Ben Shim - Canaccord Genuity - Analyst : Great. I guess for those in the audience who are not familiar with priority review vouchers, they are monetizable and can be sold and some of it --

Question: Ben Shim - Canaccord Genuity - Analyst : Great. We're looking forward to that. For WHIM, W-H-I-M, is the name of your pivotal Phase 3 trial for mavorixafor in WHIM syndrome, and I guess you're expecting topline data in fourth quarter 2022. Can you remind us of the study design and endpoints? And importantly, why opt for a meantime above threshold for white blood cell count as a primary endpoint?

Question: Ben Shim - Canaccord Genuity - Analyst : Great. Now, if things go as well as you hoped for this trial, can you offer any guidance on what the following steps would be post-FDA approval to ramp up commercialization? And how do you plan to announce results? And maybe after that, what are you thinking about in terms of manufacturing process?

Question: Ben Shim - Canaccord Genuity - Analyst : Okay, great. So it sounds like you'll have a sales force in place after approval or shortly before. And in the meantime, is there an existing WHIM community that you could improve your chances for spreading the word-of-mouth as a form for information dissemination? And are you connected with these folks?

Question: Ben Shim - Canaccord Genuity - Analyst : Great. Given how rare WHIM is, can you give us any insights on what you're thinking in terms of pricing annual cost of therapy?

Question: Ben Shim - Canaccord Genuity - Analyst : Okay, great. Going beyond WHIM, which is currently getting a lot of attention, mavorixafor data tends to suggest that you can see an increase in white blood cell counts in an even bigger population. And I think you mentioned congenital neutropenias. Can you walk us through what a chronic neutropenic disorder is, and how mavorixafor can help? 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 11, 2022 / 7:30PM, XFOR.OQ - X4 Pharmaceuticals Inc at Canaccord Genuity Growth Conference

Question: Ben Shim - Canaccord Genuity - Analyst : Great. Can you outline the study design for the ongoing Phase 1b trial? What are the endpoints? And what is the reasoning behind designing the study as a combination therapy?

Question: Ben Shim - Canaccord Genuity - Analyst : All right. Looking forward. Generic G-CSF (technical difficulty) based off of the data you have given in your studies, (inaudible) mavorixafor? 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 11, 2022 / 7:30PM, XFOR.OQ - X4 Pharmaceuticals Inc at Canaccord Genuity Growth Conference

Question: Ben Shim - Canaccord Genuity - Analyst : Great. And you have been studying mavorixafor for in Waldenstr÷m's. And -- but before we talk about that, I understand you had a bit of an organizational change, strategic shift. And I'm just wondering if you want to touch on that guess.

Question: Ben Shim - Canaccord Genuity - Analyst : Does that also include your preclinical pipeline for the next-generation CXCR4 agonist?

Question: Ben Shim - Canaccord Genuity - Analyst : Okay. Paula, can you sort of recap what the respective market sizes, I guess, in terms of patients are for mavorixafor for each of these patient populations?

Question: Ben Shim - Canaccord Genuity - Analyst : Okay. Great. Adam, can you tell us a little bit about what you've done to extend your cash? What is your cash and what's runway anticipation?

Question: Ben Shim - Canaccord Genuity - Analyst : Great. And just dovetailing on that, you've done quite a few creative financing is because you have a very supportive shareholder base. And can you maybe clarify what is going to happen with some financial and operational covenants as a result of your recent actions?

Question: Ben Shim - Canaccord Genuity - Analyst : Okay, great.

Question: Ben Shim - Canaccord Genuity - Analyst : Great. Paula, in the last few minutes that we have, maybe you can just quickly go over what is the distinguishing feature or features of this preclinical pipeline that you have?

Question: Ben Shim - Canaccord Genuity - Analyst : Great. Paula, with that, we've come to the end of our session. Dr. Ragan, Adam, thank you very much for your time. 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 11, 2022 / 7:30PM, XFOR.OQ - X4 Pharmaceuticals Inc at Canaccord Genuity Growth Conference