X4 Pharmaceuticals, Inc.

and welcome to X4 Pharmaceuticals Third Quarter Financial and Operating Results Conference Call. At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Dan Ferry, from LifeSci Advisors. Thank you, Mr. Ferry. You may begin.

Thank you, Operator. And good morning, everyone. Presenting on today's call will be X4's Chief Executive Officer, Dr. Paula Regan, Chief Commercial Officer, Mark Baldry, and Chief Medical Officer, Dr. Krzysztof Arvet-Engels. Following prepared remarks by each, we will open up the call to your questions and will be joined by Chief Financial Officer, Adam Mostafa, Chief Scientific Officer, Art Tavares, and Chief Operating Officer, Mary DiBiase. As a reminder, on today's call, the company will be making forward-looking statements regarding regulatory and product development plans, as well as research activities. These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted. Discretion of these risks can be found in X4's most recent filings with the SEC, including this quarter's Form 10-Q, which is expected to be filed after market close today. I'd now like to turn the call over to X4's President and CEO, Dr. Paula Regan. Paula?

Thanks, Dan. Good morning, everyone, and thank you for joining us on the call today. To start, I'll be reviewing our third quarter events and recent highlights, the first two of which will be the primary focus of our discussion today. As you can see here, it's again been a very busy and productive period at X4, which culminated in the great news last week that our NDA, seeking approval of our lead drug candidate, Mavarixifor, for the treatment of WIM syndrome, was accepted by the FDA for priority review. The FDA has set a PDUFA, or action date, of April 30, 2024, which sets us up for a potential second quarter 2024 launch in the U.S., if approved. In anticipation of this, we have continued to build out our fit-for-purpose commercial organization. Mark Baldry, our Chief Commercial Officer, will run through some of the details of our launch readiness and pre-launch activities to date, all of which aim to further the understanding of WHIM syndrome and educate both patients and physicians on the importance and benefits of early diagnosis. We do want to remind everyone here that due to Mavericks IV's rare pediatric disease designation for WHIM syndrome in the U.S., XIV is eligible to receive a priority review voucher, or PRV, if Mavericks IV is approved, that we can use to either obtain priority review for a subsequent application or to sell to another drug sponsor. Turning now to our Mavericks for chronic neutropenia program, we've continued to advance our Phase II study and have now enrolled more than 15 participants in the trial. Importantly, key learnings from this ongoing study, along with the data from previous trials and input from the FDA, have enabled us to finalize the design of what we believe will be the global, pivotal, Phase III clinical trial that will be used as the basis for seeking approval of Mavericks for inserting chronic neutropenic disorders. As discussed on our last quarterly call, Dr. Christoph Arbett-Engels has joined X4 as our new chief medical officer. We thought it would be a good opportunity to hear from him today as we approach some key milestones in our development of Mavericks IV in chronic neutropenia. He will be providing an update on this program later in the call. During the quarter, we also strengthened our board of directors with the addition of Mr. Keith Woods, Keith brings significant global commercial strategy and operations experience, most recently at Argenix, where as Chief Operating Officer, he helped build a sustainable enterprise and successfully launched a rare disease product globally. His meaningful experience adds to our board as X4 aims to transition to a global R&D and commercial enterprise as well. As also discussed on our last quarterly call, we completed an expanded loan facility with Hercules Capital. In addition to the possible PRV next year, this facility provides us with financial flexibility and, importantly, non-dilutive financing options. Lastly, we believe our current cash and equivalence balance of just over $140 million at the end of the third quarter is sufficient to fund our operations into 2025. And note that this does not include the debt options or the potential proceeds of the sale of the PRV if NDA approval is obtained. Before I turn it over to Mark, I'd like to present a quick review of WHIM syndrome as a reminder to those not familiar with our lead indication. WHIM syndrome is a rare primary immunodeficiency caused by the over-signaling of the CXCL12-CXCR4 pathway, which in many cases results from genetic variants in the CXCR4 receptor gene. This pathway is a key regulator of normal trafficking of white blood cells, which include neutrophils, lymphocytes, and monocytes. In WHIM syndrome, white blood cells become trapped in the bone marrow, which leads to chronic utropenia and lymphopenia. These immunodeficiencies lead to a number of clinical manifestations, most notably an increased risk of infection in the lungs, skin, and other key organs, and an increase in the incidence of HPV-related cancers, greatly affecting the quality and length of life of the estimated 1,000 people diagnosed with WHIM in the U.S. With earlier diagnosis, we believe the damage from these infections could be mitigated As you recall, we were able to show in our successful Phase III trial in WIMP syndrome statistically significant increases in both neutrophil and lymphocyte counts, as well as clinically significant reductions in the rate, frequency, and severity of infections versus placebo. If approved next spring, Maverick 4 would be the first therapy available for those individuals diagnosed with WIMP syndrome and the only disease-modifying treatment. I'm now going to turn it over to Mark to give you some of our insights and details on our progress and plans for commercializing Maverick Sephora for Wim Syndrome. Mark?