X4 Pharmaceuticals, Inc.

Thank you for standing by. This is the conference operator. Welcome to X4 Pharmaceuticals' fourth quarter and full year 2022 earnings conference call. As a reminder, all participants are in listen-only mode and the conference is being recorded. After the presentation, there'll be an opportunity to ask questions. To join the question queue, you may press star then one on your telephone keypad. Should you need assistance during the conference call, you may signal an operator by pressing star and zero. It's now my pleasure to introduce your host, Dan Ferry from LifeSci Advisors. Please go ahead.

Thank you, operator, and good morning, everyone. Presenting on today's call will be X4's Chief Executive Officer, Dr. Paul Reagan, and Chief Financial Officer, Adam Mostafa. Following prepared remarks, we will open up the call to your questions, and we're 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. A description of these risks can be found in X-Force filings with the SEC from time to time, including the company's latest 10-Q filing on November 3, 2022, and in the 10-K for 2022, which is expected to be filed after the market closed today. I'd now like to turn the call over to X-Force President and CEO, Dr. Paula Reagan. Paula?

Thanks, Dan, and thank you, everyone, for joining us on the call. As we mentioned in the press release this morning, we could not be more pleased with the progress we made in 2022 advancing our lead candidate, Maverick Sephora, towards commercialization and, most importantly, towards helping patients in need. We believe our clinical trial data continue to speak volumes, unequivocally demonstrating Maverick Sephora's ability to chronically raise circulating levels of neutrophils, lymphocytes, and monocytes. As you may be aware, following our strategic announcement last July when we tightened our focus to advance Mavericks IV in chronic neutropenic disorders, we achieved two significant development milestones, announcing positive clinical results from both our pivotal Phase III trial of Mavericks IV and WHIM syndrome and our Phase Ib clinical trial of Mavericks IV in certain chronic neutropenic disorders. Most importantly, in late November, we announced the success of our pivotal Phase III clinical trial evaluating oral Maverick Sephora in people with WIM syndrome, with the study meeting its primary endpoint and first key secondary endpoint, achieving statistically significant and clinically relevant longer times above threshold for both absolute neutrophil counts and absolute lymphocyte counts versus placebo. Maverick Sephora also demonstrated good tolerability in this robust, 52-week randomized placebo-controlled double-blinded trial. Note that Mavericks 4 is the first and only oral therapy to demonstrate durable improvements in severe chronic neutropenia and lymphopenia, the hallmarks of WIMP syndrome, which is a rare combined immunodeficiency for which there is no approved treatments. Since the November data announcement, we have continued to analyze the clinical results from the Phase 3 WHIM trial and expect to be able to present additional data from the trial at one or more medical conferences in the second quarter of 2023. We plan to host an investor call around the time of the first presentation and will update you as we have more information on the exact schedule. Importantly, these results are expected to include additional secondary and some exploratory endpoints, including assessments of infection rate, severity, duration, and types of infections, as well as the effect on patients' warp burden and certain vaccine titer data. We now have scheduled a pre-NDA meeting with the US regulatory authorities in Q2 to discuss next steps in advancing Mavericks 4 towards an NDA submission. pending input from the FDA, we continue to anticipate submitting the NDA early in the second half of this year, which we hope will lead us to our first product approval in the first half of 2024. Also last year, back in September, we presented positive data from our Phase 1B clinical trial in chronic uterpenic disorders. The study demonstrated the ability of a single oral dose of Maverick before to normalize absolute neutrophil counts, or ANC, in those with the most severe forms of neutropenia and across all chronic neutropenic disorders studied, which included idiopathic, cyclic, and congenital neutropenia. Normalization of ANC was demonstrated with Mavarixifor as a monotherapy and in combination with the only approved treatment for severe neutropenia, injectable granulocyte colony stimulating factor, or GCSF. Impressively, 100% of patients responded. We could have not hoped for more in this initial study. This early trial has now been expanded into a Phase II clinical trial to assess the long-term durability, safety, and tolerability of oral Mavericks for more broadly in those diagnosed with idiopathic, cyclic, or congenital chronic neutropenia. Participants are currently being enrolled in the Phase 2 CN trial, and we expect to be able to report clinical data from the study in Q2 or Q3 of this year. The timing of this will obviously be dependent on the rate of enrollment, and we do plan to present a robust data set when we announce the first results from this trial. These proof-of-concept data we are generating in our Phase 1-2 trials aim to unlock an even broader potential of mavericks before. one where we could potentially offer a differentiated oral and well-tolerated treatment option to upwards of 50,000 people diagnosed with CN disorders in the U.S. We also expect to be able to provide clarity on both the scope and the possible timing of our planned Phase III clinical program for Mavericks of 4 and chronic neutropenic disorders in the second or third quarter of this year. At this time, we expect the phase three trial will likely be a randomized placebo-controlled trial studying the safety and efficacy of Mavericks 4 on top of standard of care, with likely a primary endpoint measuring changes in neutrophil counts and secondary endpoints related to reduction in infections. But we expect to know a lot more following a meeting with the FDA to specifically discuss the path forward of Mavericks 4 for these certain chronic neutropenic disorders. Throughout 2022, through both our clinical and scientific research programs, we were able to gain much greater insights into Mavericks4's ability to address the unmet need in chronic neutropenic disorders, including WHIM syndrome. We are particularly pleased that almost all of our submitted abstracts were accepted for either oral or poster presentations at prominent medical conferences during the year, including the Quad AI meeting early in the year. the CIS annual meeting in spring, EHA, the NICER symposium, and ESID over the summer, and the National Organization for Rare Disease Summit in the fall. We also had quite a large presentation at the annual meeting of the American Society of Hematology, or ASH, in December, and garnered strong interest both at our presentations and our X4 booths. Throughout the year, our presentations not only highlighted new insights into the breadth of genotype and phenotype of people with limb syndrome, helping to identify both additional patients and helping to educate treating physicians, but also demonstrated new understandings into Maverick Score's mechanism of action, principally its ability to induce maturation and mobilization of white blood cells from the bone marrow into blood circulation and enabling immune surveillance and response. Our research also deepened our understanding of the diverse and significant needs of the patient community through interviews and survey engagements and helped us define what is turning out to be a larger-than-expected U.S. population of patients living with chronic neutropenic disorders. These milestones throughout the year, along with the continued support of our investors and analysts, helped us successfully complete two large financings raising gross proceeds of more than $120 million despite continued challenging biotech market conditions. Currently, we have a strong balance sheet to help us propel our pre-commercial efforts for Mavericks 4 and WIM and further advance our mission to deliver Mavericks 4 to help those across a range of chronic neutropenic disorders. I'll now turn it over to our CFO, Adam Mostafa, to review the fourth quarter and full year 2022 financials. Adam?