X4 Pharmaceuticals, Inc.

Greetings and welcome to X4 Pharmaceuticals First Quarter 2022 Financial and Operating Results Conference Call. At this time, our 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, Dr. Glenn Shulman, Head of Investor Relations. Please begin.

Thank you, Operator, and good morning, everyone. Presenting on today's call will be X4's Chief Executive Officer, Dr. Paula Regan, and the company's Chief Financial Officer, Adam Mostafa. Following prepared marks by each of them, we'll open up the call to your questions, during which we'll be joined by our Chief Scientific Officer, Art Tavares, Chief Medical Officer, Diego Kadavid, and our Chief Operating Officer, Mary DiBiase. As a reminder on today's call, X4 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 arbitrarily from those forecasted. A description of these risks can be found in X4's most recent filings with the SEC. With that, I now would like to turn the call over to X4's President and CEO, Dr. Paula Reagan. Paula?

Thanks, Glenn, and thank you, everyone, for joining us on this call this morning. On this morning's call, I will provide a brief update on recent accomplishments, highlight a few of our important upcoming catalysts, including the readout from our Phase III-IV WIM trial, and, of course, open up the call to any questions you may have for the team. As a reminder, our lead candidate, Maverick Zafour, a CXCR4 antagonist, is being evaluated as an oral, once-daily treatment for people with rare disorders of the immune system, including people diagnosed with WIMP syndrome and chronic neutropenia, and also for those diagnosed with certain lymphomas. WIMP syndrome is a rare immunodeficiency disorder caused by genetic mutations to the CXCR4 receptor. The disease is characterized by HPV-associated warts, hypogamyloglobulinemia, multiple types of infections, and myelocortexis, a pathologic bone marrow finding associated with the reduced ability of white blood cells to move from the bone marrow to the periphery. We believe that the results from the open-label extension of our Phase II clinical trial with Maverick, Sephora, and WIM patients continue to support the significant potential of our lead drug candidate in this indication. with data showing durable TATs, or time above threshold, for blood levels of neutrophils, lymphocytes, and monocytes, decreased frequency of infections, and robust and sustained improvement in wards. Patient quality of life, including reductions in doctor or hospital visits, was meaningfully improved based on our Phase II patient narrative. Importantly, these include the primary and secondary endpoints of our ongoing Phase III WHIM trial, And just as importantly, Mavericks 4 continues to be well-tolerated over a median treatment duration of now more than 160 weeks. We continue to anticipate that top-line results from our global placebo-controlled, double-blinded, four-WIM Phase III trial, which enrolled a total of 31 adolescents and adult patients, will be available in the fourth quarter of this year. We intend to report on the primary endpoint which consists of time above threshold for absolute neutrophil count, and which was powered based on our findings in the Phase II trial, along with available secondary endpoints. The trial design and primary endpoint have been agreed upon with the FDA. Additionally, secondary endpoints evaluating sections and warp burden, among others, have been also discussed extensively with the FDA, and their guidance has been diligently followed. We look forward to preparing and submitting the new drug application or NDA for submission to the agency in the second half of next year. We also continue to conduct and publish research on the underlying genetics of WHIM as we work to further characterize and expand the definition of the disease. We have built strong in-house research programs that leverage world-class collaborators to advance bench-to-bed sign research. By continuing to establish correlations between clinical presentation and novel genetic variants associated with WHIM, we can enhance our ability to identify undiagnosed patients, including those who may potentially benefit from Mavericks IV treatment. At the upcoming European Hematology Association, or EHA, meeting this June, we plan to present more of this novel research. We will have a press release with more details this afternoon after the abstract embargo lift and we hope to see you at the Congress in Vienna. As I mentioned earlier, we believe there are additional disease areas harboring patients in need who could also potentially benefit from treatment with maverixifor. With WHIM as our B-tide indication well on its way, we are also assessing the potential of maverixifor as a therapy for other causes of chronic utropenia, given the drug candidate's potential for meaningful advantages over the only existing therapy. Chronic neutropenia, or CN, includes a number of subtypes, such as congenital, idiopathic, and cyclic neutropenia, all of which we believe could benefit from treatment with Maverick before. In our ongoing Phase 1B study, we are actively enrolling patients diagnosed with these types of CN to establish biologic activity and support future regulatory discussions. We look forward to providing both updated clinical data along with regulatory feedback during the third quarter of this year. We also announced this morning that we have completed enrollment in our ongoing Phase 1B clinical trial studying patients diagnosed with Waldenstrom's macroglobulinemia, a rare B-cell lymphoma. This Phase 1B trial is designed to demonstrate safety, dose, and elucidate proof of concept of maverick spore in combination with the BTK inhibitor ibrutinib in patients with Waldenstrom, resulting from mutations in both their MYD88 and CXDR4 genes. This patient population continues to have reduced treatment responses due to their cancers harboring these two mutations. Doses of 200, 400, and 600 milligrams per day were evaluated, and once cleared, eligible patients were dose escalated to receive 600 milligrams once daily. Data that we presented last December at ASH showed a 100% overall response rate, or ORR, and sustained decreases in serum IgM. a blood marker that corresponds with cancer burden in 10 evaluable patients whose cancers had confirmed MYD8 and CXCR4 mutations. Further to that, we also presented additional preclinical results in a poster presentation at the 2022 American Association of Cancer Research, or AACR, annual meeting. This poster reported that combinations of Mavericks before with a broad array of BTK inhibitors overcame bone marrow-induced treatment resistance, and enhanced cancer cell death in in vitro assays of Waldenstrom. We expect to report results from the Phase 1b clinical study, which we anticipate would include at least six months of treatment data for patients on the 600 milligram dose during the second half of this year. With three readouts on the horizon, including data and chronic utropenia from our Phase 1b study next quarter, results from Wallenstrom's Phase 1B study in the second half, and our four WHIM Phase 3 results in the fourth quarter, we are extremely excited for what's to come. With that brief update, I'll now turn it over to Adam to discuss our financial results for the quarter before we open up the call for questions. Adam?