X4 Pharmaceuticals(XFOR) - 2021 Q4 - Annual Report

FORM 10-K Filing Information This section details X4 Pharmaceuticals, Inc.'s identification, incorporation, executive offices, Nasdaq listing, and SEC filing classifications for the fiscal year ended December 31, 2021 Registrant Information This section provides key identification details for X4 Pharmaceuticals, Inc., including its incorporation state, principal executive offices, telephone number, and SEC filing particulars for the fiscal year ended December 31, 2021. The company is registered on The Nasdaq Stock Market LLC under the symbol XFOR and is classified as a non-accelerated filer, smaller reporting company, and emerging growth company - Registrant: X4 PHARMACEUTICALS, INC.[2] - Incorporation: Delaware[2] - Principal Executive Offices: 61 North Beacon Street, 4th Floor, Boston, Massachusetts[2] SEC Filing Classifications | Classification | Status | | :--------------- | :----- | | Well-known seasoned issuer | No | | Required to file reports | Yes | | Filed all reports (preceding 12 months) | Yes | | Submitted Interactive Data File (preceding 12 months) | Yes | | Large Accelerated Filer | No | | Accelerated Filer | No | | Non-accelerated filer | Yes | | Smaller reporting company | Yes | | Emerging growth company | Yes | - Market Value of Voting Common Stock (non-affiliates, June 30, 2021): Approximately $158 million[4] - Shares of Common Stock Outstanding (March 14, 2022): 30,809,376 shares[5] Documents Incorporated by Reference Portions of the registrant's definitive proxy statement for its 2022 Annual Meeting of Stockholders are incorporated by reference into Part III of this Annual Report on Form 10-K - Portions of the 2022 Proxy Statement for the Annual Meeting of Stockholders are incorporated by reference into Part III of this Form 10-K[7] Cautionary Note Regarding Forward-Looking Statements This section advises investors on the inherent risks and uncertainties associated with forward-looking statements in the Annual Report, emphasizing that actual results may differ materially from projections Nature of Forward-Looking Statements This section highlights that the Annual Report contains forward-looking statements, identifiable by specific terminology, which are based on expectations, assumptions, estimates, and projections. These statements are not guarantees of future results and involve substantial risks and uncertainties, with actual results potentially differing materially - The Annual Report contains forward-looking statements identified by terms such as 'may,' 'should,' 'expects,' 'intends,' 'plans,' 'anticipates,' 'believes,' 'estimates,' 'predicts,' 'potential,' 'continue' or negatives thereof[12] - These statements are based on expectations, assumptions, estimates, and projections, are not guarantees of future results, and involve substantial risks and uncertainty[12] Key Risks and Uncertainties Forward-looking statements are subject to numerous known and unknown risks, including those detailed in the 'Risk Factors' section. These risks encompass the timing and results of clinical trials for mavorixafor (WHIM, Waldenström's, SCN), the impact of the COVID-19 pandemic on operations and trials, regulatory approval processes, commercialization strategies, intellectual property protection, and financial needs - Timing, progress, and results of current trials of mavorixafor (Phase 3 for WHIM syndrome, Phase 1b for Waldenström's macroglobulinemia, Phase 1b for severe congenital Neutropenia (SCN) and chronic neutropenia disorders)[12] - Initiation, timing, design, progress, and results of current and future preclinical studies and clinical trials of X4P-002 and X4P-003[12] - Impact of the ongoing COVID-19 pandemic on business, operations, strategy, goals, and anticipated timelines, including diversion of healthcare resources and interruption of clinical trial activities[12] - Ability to obtain and maintain regulatory approval for product candidates and related restrictions[12] - Plans to research, develop, manufacture, and commercialize product candidates[12] - Timing of regulatory filings and developments in the U.S. and other countries[12] - Size and growth potential of markets for product candidates and market acceptance[12] - Benefits of FDA and European Commission designations (Fast Track, Orphan Drug, Breakthrough Therapy)[13] - Commercialization, marketing, and manufacturing capabilities and strategy[13] - Ability to attract and retain qualified employees and key personnel[13] - Competitive position and industry developments[13] - Expectations regarding intellectual property protection, future operations, financial position, revenues, costs, expenses, capital requirements, and need for additional financing[13] - Plans to in-license, acquire, develop, and commercialize additional product candidates[13] - Impact of laws and regulations[13] - Strategies, prospects, plans, expectations, or objectives[13] Disclaimer and Obligation Investors are advised not to rely on forward-looking statements as guarantees of future performance, and the company undertakes no obligation to publicly update these statements unless required by law - Investors should not rely on forward-looking statements as representing the company's views as of any date subsequent to the report date[14] - The company undertakes no obligation to publicly update any forward-looking statements, except as required by law[14] PART I Item 1. Business X4 Pharmaceuticals, Inc. is a late-stage clinical biopharmaceutical company focused on rare diseases, particularly those involving immune system dysfunction. Its lead product candidate, mavorixafor, a CXCR4 inhibitor, is in Phase 3 trials for WHIM syndrome and Phase 1b trials for chronic neutropenia and Waldenström's macroglobulinemia. The company aims to commercialize mavorixafor, expand its pipeline with earlier-stage candidates (X4P-002, X4P-003), and pursue strategic collaborations - X4 Pharmaceuticals is a late-stage clinical biopharmaceutical company focused on rare diseases, initially immune system dysfunction[18] - Lead product candidate, mavorixafor, is a first-in-class, small molecule CXCR4 inhibitor, developed as a once-daily oral therapy[18] - Mavorixafor is in a global Phase 3 clinical trial for WHIM syndrome, and Phase 1b clinical trials for chronic neutropenia and Waldenström's macroglobulinemia[19] Overview X4 Pharmaceuticals is a late-stage clinical biopharmaceutical company developing mavorixafor, a CXCR4 inhibitor, for rare diseases like WHIM syndrome, chronic neutropenia, and Waldenström's macroglobulinemia. The Phase 3 trial for WHIM syndrome completed enrollment in Q3 2021, with top-line data expected in Q4 2022. Mavorixafor has received Breakthrough Therapy, Fast Track, and Rare Pediatric Designations from the FDA for WHIM syndrome, with an NDA submission anticipated in H2 2023. The company estimates a higher prevalence of WHIM syndrome in the U.S. (1,000 to 3,500+ patients) than previously thought, supported by new mutation identification - Mavorixafor is a first-in-class, small molecule inhibitor of the chemokine receptor CXCR4, developed as a once-daily oral therapy for rare diseases, including primary immunodeficiencies (PIDs) and certain cancers[18] - Global Phase 3 clinical trial for WHIM syndrome completed enrollment in Q3 2021 (31 patients), with top-line data expected Q4 2022[20] - Mavorixafor received FDA Breakthrough Therapy, Fast Track, and Rare Pediatric Designations for WHIM syndrome, potentially leading to accelerated review[20] - Anticipated New Drug Application (NDA) submission to FDA in H2 2023 for WHIM syndrome (ages 12+)[20] - Prevalence of WHIM syndrome in the U.S. is estimated to be 1,000 to 3,500+ patients, significantly higher than previous registries, supported by market research and identification of a new D84H mutation[21] - Additional data from Phase 1b clinical trials in chronic neutropenia and Waldenström's expected by Q3 2022 and H2 2022, respectively[22] [Our Strategy