X4 Pharmaceuticals, Inc.

Greetings. Welcome to X4 Pharmaceuticals Second Quarter 2023 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 call is being recorded. It is now my pleasure to introduce your host, Dan Ferry from Lakeside Advisors. Please begin.

Thank you, Operator, and good morning, everyone. Presenting on today's call will be X4's Chief Executive Officer, Dr. Paula Regan, the company's Chief Financial Officer, Adam Mostafa, and Interim Chief Medical Officer, Dr. Murray Stewart. Following prepared remarks by each, we will open the call to your questions and will be joined by Chief Scientific Officer, Art Tavares, Chief Commercial Officer, Mark Baldry, 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 forecasts. A description 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 Reagan. Paula?

Thanks, Dan. Hello, everyone. We'll be covering a lot of exciting updates this morning, so thank you for joining us on this call. Adami will be providing an update on our second quarter and recent events, and we'll then focus the rest of the call on three key updates regarding our ongoing chronic neutropenia program. First, we'll provide some further insights on what we estimate to be the minimum addressable U.S. market for Mavericks of 4 in the CN populations we're pursuing. Next, we'll report on some exciting emerging data from our ongoing Phase 2 trial. And finally, we'll also provide a regulatory update as we prepare for a Phase 3 clinical trial in 2024. We'll then conclude and open it up to your questions. This was another extremely productive quarter for X4. Importantly, we presented new data from our Phase III 4WIM trial in May that followed disclosure in late 2022 that the trial had met its primary endpoint and first key secondary endpoint and was well tolerated throughout the trial. In addition to demonstrating that Maverick support treatment statistically significantly and durably raised both absolute neutrophil and lymphocyte counts, both ANC and ALC versus placebo, these new data revealed that Mavericks for treatment also resulted in a statistically significant reduction in annualized infection rate and affected clinically meaningful reductions in both the severity and duration of infections versus placebo. We presented these data initially at our company webinar in mid-May. Additionally, the data were also accepted for oral presentation at two notable conferences. First, the annual meeting of the Clinical Immunology Society, or CIS, where Dr. Rafael Batalado presented the data. And second, the European Hematology Association, or EHA, where Dr. Jean Donadieu presented the data to a standing-only crowd in Frankfurt, Germany. These data have generated much excitement throughout the immunology and hematology physician communities around Mavericks for both for its potential to be the first disease-modifying treatment for WHIM syndrome and for supporting its potential to be the first new treatment option and potentially the only oral therapy to date for people with chronic utropenia in more than 30 years. And we are now poised to submit our first new drug application with the FDA seeking U.S. approval of oral, once daily Mavericks IV for the treatment of people aged 12 years and older with WHIM syndrome. There is a palpable excitement at the company these days, as you can well imagine. We're also pleased to receive notice of issuance of an additional patent on Mavericks IV in June. This granted patent, the third issued patent covering Mavericks 4's composition of matter, protects compositions of matter comprising Mavericks 4 and related drug substances formed during the Mavericks 4 manufacturing process through December of 2038. And we are thrilled to have recently announced the appointment of Dr. Christoph Arbett-Engels, our new Chief Medical Officer. Dr. Arbett Engels is a very seasoned executive with significant experience in drug discovery, translational research, clinical development, regulatory and medical affairs, and product launch and lifecycle management. Experience that spans a broad range of therapeutic areas, including rare and orphan diseases. He's held leadership roles at both large and small life sciences and pharmaceutical companies and is expected to bring great strategic global perspective to our team here at X4 when he starts next week. While we have a minute, I'd like to express our sincere gratitude to Dr. Murray Stewart, who's been serving as our interim chief medical officer and leading the Mavericks for WIM NDA submission efforts. Rest assured, Murray will still be staying on as a consultant to the company to finalize the NDA submission and help onboard Christoph, and will continue on as a continuing member of our board of directors. Thank you so much to Murray for all you've done for X4. I'll now pass the call over to Adam Mustafa, our CFO, to quickly cover the financial highlights of the quarter and recent updates before we turn our focus to our Chronic Atropinia program. Adam?