X4 Pharmaceuticals, Inc.

Greetings, and welcome to X4 Pharmaceuticals' first quarter 2023 earnings conference call. At this time, all participants are in a listen-only mode, and a question-answer session will follow the formal presentation. As a reminder, the conference call is being recorded. It is now my pleasure to introduce your host, Dan Ferry from LifeSci Advisors. Please begin.

Thank you, Operator, and good morning, everyone. Presenting on today's call will be X4's President and Chief Executive Officer, Dr. Paula Regan, and Chief Financial Officer, Adam Mostafa. Following prepared remarks, we will open the call to your questions and will be joined by Interim Chief Medical Officer, Dr. Murray Stewart, Chief Commercial Officer, Mark Baldry, Chief Scientific Officer, Dr. Art Tavares, and Chief Operating Officer, Dr. 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. Description of these risks can be found in X-Force filings with the SEC, including the company's latest 10-K for the year 2022 and this quarter's Form 10-Q, which is expected to be filed today. I'd now like to turn the call over to Dr. Paula Reagan. Paula?

Thanks, Dan, and thank you, everyone, for joining us on the call this morning. We hope to make this an efficient call today and focus on what we hope will be value-building milestones throughout the rest of 2023. This is truly an exciting time at X4 as we continue to advance our lead investigational candidate, Maverick Sephora, towards commercialization and the first potential chronic neutropenic disorder, WHIM syndrome. As you know, in late 2022, we announced that our phase three clinical trial evaluating once daily oral Maverick Sephora in people with WHIM syndrome had met its primary endpoint and first key secondary endpoint. with Mavericks for achieving statistically significant and clinically relevant longer times above threshold levels for both absolute neutrophil and absolute lymphocyte counts versus placebo and demonstrating good tolerability in the trial. Subsequently, we announced that our late-breaker abstract reporting additional data from the Phase III WIM trial was accepted for oral presentation at this year's meeting of the Clinical Immunology Society, taking place from May 18th through the 21st in St. Louis. Dr. Raphael Bottolato, who is Professor of Pediatrics at the University of Brescia in Italy and an investigator in the 4WIM clinical trial, will present at 11.30 a.m. Central Time on Sunday, May 21st. Although this session will only be accessible live to the conference attendees, we will be posting the slides on our website concurrent with the presentation. Following the publication of conference abstracts by CIS on the morning of May 16th, we will be hosting an investor event later that day at 4 p.m. to present data on additional secondary endpoints from the trial, including results on infection burden, among other outcome metrics. You can register for that event on our website or through the link provided in this morning's earnings press release. Joining us for the event to comment on the Phase III results and the unmet medical needs of people with WHIM and chronic neutropenia will be a diverse panel of immunologists, hematologists, and rheumatologists, all of whom have expertise in treating immunodeficiencies, and several of whom were investigators in the 4-WHIM Phase III trial. During the May 16th event, we will be hearing commentary from Dr. Charlotte Cunningham-Rundles, a professor and immunologist at the Icahn School of Medicine at Mount Sinai. Dr. Jean Donadieu, a pediatrician in the hemato-oncology department of Trousseau Hospital in Paris, and importantly, coordinator of the French Registry for Chronic Neutropenia. Dr. Peter Neuberger, professor of pediatrics and molecular cell and cancer biology at UMass Chan Medical School. Dr. Akiko Shimomura, Professor of Pediatrics at Harvard Medical School and Director of the Bone Marrow Failure and Myelodysplastic Syndrome Program at Boston Children's Hospital, and Dr. Teresa Tarrant, Associate Professor and Director of the Clinical Immunology Laboratory at Duke University School of Medicine and Vice Chief of Translational Research for Rheumatology and Immunology. We will also be hearing unique perspectives from three individuals who have been diagnosed with WHIM and have been experiencing WHIM syndrome symptoms since birth. Finally, we are expecting Doctors Shimomura and Tarrant to join us live for Q&A following the formal presentation. During the event, we expect to be providing an update on our U.S. regulatory activities for mavericks before for the treatment of WHIM syndrome as we continue to be on track to file a U.S. new drug application early in the second half of 2023 and prepare for a potential launch in the U.S. in the first half of 2024. Concurrent with all of this, We continue to enroll participants in our ongoing Phase II trial, evaluating the safety and efficacy of Maverick Sephora for the treatment of idiopathic, cyclic, and congenital chronic neutropenia. And we believe are on track to announce clinical data and provide clarity on the scope and timing of the expected CN Phase III clinical program in the Q2-Q3 timeframe. In our release this morning, we also announced that we will be presenting a poster at CIS highlighting the results of what we believe is the first research study to assess the correlation between the incidence of serious infection events, or SIEs, and the severity of chronic neutropenia. This abstract will also be published on May 16th. Concurrent with the poster presentation, which is on Saturday, May 20th at 1.30 p.m. Central Time, we will be adding the poster to our website. As a result of our development efforts and our published data to date, we continue to believe that due to its demonstrated ability to elevate levels of white blood cells, Maverick support has the potential to be a breakthrough for those with WIMP syndrome and other chronic neutropenic disorders. We look forward to updating you on our progress throughout the year as we advance our mission to bring innovation to these patient populations in need. I'll now turn it over to our CFO, Adam Mustafa, to review the first quarter financials. Adam?