X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q4 2022 Earnings Conference Call March 21, 2023 9:00 AM ET Company Participants Dan Ferry - IR, LifeSci Advisors Paula Ragan - CEO Adam Mostafa - CFO Murray Stewart - Interim Chief Medical Officer Mark Baldry - Chief Commercial Officer Conference Call Participants Divya Rao - TD Cowen Edward Tenthoff - Piper Sandler Kristen Kluska - Cantor Fitzgerald Swayampakula Ramakanth - H.C. Wainwright Operator Thank you for standing by. This is the conference operator. Welcome to ...

Corporate Overview November 2022 Forward-Looking Statements 2 This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target," or other similar terms or expressions that concern X4's expectations, strategy, plans, or inten ...

Financial Data and Key Metrics Changes - The company is preparing for a potential NDA submission in early second half of 2023, with a commercial launch of mavericksphore anticipated in the first half of 2024 [24][32] - The average pretreatment absolute neutrophil count (ANC) for patients enrolled in the phase three trial was 220 cells per microliter, indicating severe neutropenia [12][13] - The company expects to announce phase three top line data in the fourth quarter of 2022 [14][20] Business Line Data and Key Metrics Changes - The phase three trial for mavericksphore in WHIM syndrome has completed enrollment with 31 patients from 12 countries, exceeding initial enrollment targets [12][14] - Over 90% of eligible participants from the placebo-controlled portion of the study opted to continue into the open-label extension study [14] Market Data and Key Metrics Changes - The company estimates that there are approximately 50,000 chronic neutropenia patients in the U.S., with mavericksphore potentially addressing a significant unmet need [7][41] - The company is focusing on the top five to seven European markets for the initial launch of mavericksphore following U.S. approval [24] Company Strategy and Development Direction - The company aims to establish mavericksphore as the first treatment specifically developed for WHIM syndrome, with plans to expand its use to other chronic neutropenic disorders [22][32] - The company is investing in activities to support education and awareness of WHIM syndrome, including collaborations with experts and patient organizations [23] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming phase three trial results and the potential for mavericksphore to transform the treatment landscape for chronic neutropenia [33] - The company is actively engaging with the FDA to ensure alignment on study design and regulatory pathways for both WHIM and chronic neutropenia indications [20][56] Other Important Information - The company has secured conditional approval of a brand name with the FDA for mavericksphore, marking a significant step in the commercialization process [29] - The company has completed registration batches for manufacturing, ensuring a stable supply chain for the anticipated launch [29] Q&A Session Summary Question: What is the average ALC for patients pretreatment? - Management indicated that the average ALC is not readily available but will follow up with the information [36] Question: Have you seen the blinded infection rate in the trial? - Management confirmed that infection events are being monitored and are consistent with expectations [38] Question: How do you reconcile the 50,000 patient estimate with the previously mentioned 2,500 patients on chronic GCSF? - Management explained that the previous number was based on registry data and acknowledged the challenges in defining chronic use of GCSF [40][41] Question: What are the powering assumptions for the WHIM trial? - Management stated that the trial was powered based on phase two results, with a current power greater than 95% for the primary endpoint [45] Question: What is the latest estimate on the prevalence of WHIM? - Management remains confident in guiding to a prevalence of 1,000 or higher for WHIM syndrome [46] Question: What are the differences in patient populations between phase two and phase three? - Management noted that both populations required a clinical diagnosis of WHIM syndrome, with the main difference being the inclusion of pediatric patients in phase three [50] Question: What activities will occur between the top line data and NDA filing? - Management outlined that the phase three data will be analyzed and prepared for NDA submission, with ongoing regulatory discussions for chronic neutropenia [56]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 _____________________________________________________________________________________ FORM 10-Q _____________________________________________________________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2022 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the trans ...

X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q2 2022 Earnings Conference Call August 4, 2022 8:30 AM ET Company Participants Dan Ferry - LifeSci Advisors, IR Dr. Paula Ragan - Chief Executive Officer Adam Mostafa - Chief Financial Officer Art Taveras - Chief Scientific Officer Diego Cadavid - Chief Medical Officer Mary DiBiase - Chief Operating Officer Conference Call Participants Stephen Willey - Stifel Eva Privitera - Cowen Mayank Mamtani - B. Riley Securities Arthur He - H.C. Wainwright Operator Greetings. And ...

Corporate Overview August 2022 Forward-Looking Statements 2 This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target," or other similar terms or expressions that concern X4's expectations, strategy, plans, or intenti ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 _____________________________________________________________________________________ FORM 10-Q _____________________________________________________________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2022 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2022 or Washington, D.C. 20549 _____________________________________________________________________________________ ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FORM 10-Q _____________________________________________________________________________________ (Mark One) (Exact name of re ...

X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q1 2022 Earnings Conference Call May 12, 2022 8:30 AM ET Company Participants Glenn Schulman - Vice President, Investor Relations and Corporate Communications Paula Ragan - President and Chief Executive Officer Adam Mostafa - Chief Financial Officer Diego Cadavid - Chief Medical Officer Arthur Taveras - Chief Scientific Officer Conference Call Participants Marc Frahm - Cowen & Company William Wood - B. Riley Securities Trevor Howard - Oppenheimer Zegbeh Jallah - ROTH ...

X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q4 2021 Earnings Conference Call March 17, 2022 8:30 AM ET Company Participants Glenn Schulman - Vice President, Investor Relations & Corporate Communications Paula Ragan - President & Chief Executive Officer Adam Mostafa - Chief Financial Officer Diego Cadavid - Chief Medical Officer Arthur Taveras - Chief Scientific Officer Conference Call Participants Stephen Willey - Stifel Marc Frahm - Cowen & Company Mayank Mamtani - B. Riley Securities Arthur He - H.C. Wainwrigh ...