Mavorixafor's Potential & Target Diseases - Mavorixafor, a Phase 3 candidate, is being advanced to potentially treat over 30,000 patients in the US and EU markets across multiple indications[7] - The company is targeting 1,000-3,700 U S WHIM syndrome patients, 2,000-3,000 U S Waldenström's Macroglobulinemia patients, and 5,000-10,000 U S Chronic Neutropenia patients[10] - The global G-CSF market for all uses is over $4 billion, with the U S market for severe chronic neutropenia estimated at $120-460 million[57] Clinical Trial Updates & Data - Top-line data from the Phase 3 trial in WHIM syndrome is expected in 4Q 2022, with a potential NDA filing in 1Q 2023[7, 98] - Initial data from the Phase 1b trial in Chronic Neutropenia and additional data in Waldenström's Macroglobulinemia are expected in 4Q 2021[7, 98] - Phase 2 trial in WHIM syndrome demonstrated a 6-fold increase in TATANC and over 80% reduction in annualized infection rates[37] Pipeline & Expansion - A new molecule (X4P-002) is expected to enter the clinic in 2H 2022 for oncology indications, targeting other leukemias and lymphomas which is >25,000[7, 10] - The company anticipates additional data at all doses and initial response data (OR, MR) for Waldenström's Macroglobulinemia in 4Q 2021[92] - Newly identified D84H CXCR4 mutation suggests additional ~1,250-2,500 WHIM patients[49] Financial Status - The company had $78 million in cash and equivalents as of September 30, 2021, expected to fund operations into Q4 2022, excluding $19.5 million in net proceeds from equity sale in November 2021[100, 101, 105]

X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q3 2021 Earnings Conference Call November 4, 2021 9:05 AM ET Company Participants Dan Ferry - LifeSci Advisors Paula Ragan - Chief Executive Officer Adam Mostafa - Chief Financial Officer Art Taveras - Chief Scientific Officer Diego Cadavid - Chief Medical Officer Conference Call Participants Zegbeh Jallah - ROTH Capital Leland Gershell - Oppenheimer Marc Frahm - Cowen & Company Stephen Willey - Stifel Operator Greetings and welcome to X4 Pharmaceuticals Third Quarter ...

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, 2021 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the trans ...

| --- | --- | --- | --- | |--------------------------------------------------------------------------------------------------------------------------------|-------|-------|-------| | | | | | | | | | | | Developing treatments | | | | | designed to have a clear and profound impact for patients with rare diseases, including WHIM syndrome and uncommon cancers | | | | | September 2021 Investor Deck | | | | | | | | | Forward-Looking Statements 2 This presentation contains forward-looking statements within the mea ...

X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q2 2021 Earnings Conference Call August 3, 2021 8:30 AM ET Company Participants Carey Gallant - Investor Relations, LifeSci Advisors Paula Ragan - Chief Executive Officer Adam Mostafa - Chief Financial Officer Art Taveras - Chief Scientific Officer Conference Call Participants Trevor Allred - Oppenheimer RK - H.C. Wainwright Zegbeh Jallah - ROTH Capital Partners Mayank Mamtani - B. Riley Financial Ben Shim - Canaccord. Operator Greetings, and welcome to X4 Pharmaceutic ...

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, 2021 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition ...

X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q1 2021 Earnings Conference Call May 6, 2021 8:30 AM ET Company Participants Daniel Ferry - LifeSci Advisors Paula Ragan - CEO Adam Mostafa - CFO Art Taveras - Chief Scientific Officer Diego Cadavid - Chief Medical Officer Conference Call Participants Stephen Willey - Stifel Marc Frahm - Cowen Swayampakula Ramakanth - H.C. Wainwright Sahil Kazmi - B. Riley FBR Arlinda Lee - Canaccord Genuity Operator Greetings and welcome to X4 Pharmaceuticals First Quarter Financial a ...

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 March 31, 2021 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transitio ...

Part I [Item 1. Business](index=8&type=section&id=Item%201.%20BUSINESS) A late-stage biopharmaceutical company developing novel oral therapies for rare diseases related to the CXCR4 pathway - The company's lead product candidate, mavorixafor, is a **first-in-class, oral, small molecule antagonist** of the CXCR4 receptor, targeting rare diseases like primary immunodeficiencies and certain cancers[27](index=27&type=chunk) - Mavorixafor is in a **pivotal Phase 3 global clinical trial** (4WHIM) for WHIM syndrome, with top-line data expected in 2022 and has been granted Breakthrough Therapy, Fast Track, and Rare Pediatric Designations by the FDA[28](index=28&type=chunk)[54](index=54&type=chunk)[55](index=55&type=chunk) - The company is also conducting **Phase 1b trials** for mavorixafor in Severe Congenital Neutropenia (SCN) and Waldenström's macroglobulinemia, with initial data from the Waldenström's trial expected in the first half of 2021[30](index=30&type=chunk)[61](index=61&type=chunk)[64](index=64&type=chunk) X4 Pharmaceuticals Clinical Pipeline (as of Dec 31, 2020) | CANDIDATE | INDICATION | PRECLINICAL | PHASE 1 | PHASE 2 | PHASE 3 | | :--- | :--- | :--- | :--- | :--- | :--- | | **Mavorixafor (X4P-001)** | WHIM syndrome | | | | **Phase 3** | | | Waldenström's Macroglobulinemia | | **Phase 1b** | | | | | Severe Congenital Neutropenia (SCN) | | **Phase 1b** | | | | | Clear cell renal cell carcinoma (ccRCC) | | | **Phase 2a** | | | **X4P-002** | Glioblastoma multiforme (GBM) | **Preclinical** | | | | | **X4P-003** | Primary immuno-deficiencies (PID) | **Preclinical** | | | | - The company relies on key in-license agreements, including an **exclusive license from Genzyme** for the CXCR4 receptor intellectual property, which includes up to **$25.0 million in milestone payments** and tiered royalties[92](index=92&type=chunk)[94](index=94&type=chunk) - X4 Pharmaceuticals depends on **single-source third-party manufacturers**: Aptuit for the mavorixafor active pharmaceutical ingredient (API) and Mayne Pharma for the finished drug product capsules[87](index=87&type=chunk)[88](index=88&type=chunk)[90](index=90&type=chunk) [Item 1A. Risk Factors](index=34&type=section&id=Item%201A.%20RISK%20FACTORS) The company faces significant risks from its history of financial losses, dependence on a single product, and operational challenges - The company has a history of significant operating losses, with a **net loss of $62.1 million** in 2020 and an **accumulated deficit of $194.2 million** as of December 31, 2020, and may never achieve profitability[182](index=182&type=chunk) - The company will **require substantial additional funding** as its existing cash of $78.7 million is not sufficient for the next 12 months, raising **going concern risks**[187](index=187&type=chunk)[188](index=188&type=chunk) - The business is **almost entirely dependent on the success of its lead product candidate**, mavorixafor, and failure to commercialize it would substantially harm the business[201](index=201&type=chunk)[202](index=202&type=chunk) - The **COVID-19 pandemic is adversely affecting business operations**, causing delays in clinical trial site activation and slower patient enrollment[225](index=225&type=chunk)[362](index=362&type=chunk) - The company is dependent on a **single third-party manufacturer** for the mavorixafor API and finished product, with **no redundant supply arrangements** in place[306](index=306&type=chunk)[307](index=307&type=chunk) - The company **relies on license agreements with Genzyme** and others for critical intellectual property, the termination of which could halt product development[214](index=214&type=chunk) [Item 1B. Unresolved Staff Comments](index=78&type=section&id=Item%201B.%20UNRESOLVED%20STAFF%20COMMENTS) The company reports that it has no unresolved staff comments from the SEC - There are no unresolved staff comments[405](index=405&type=chunk) [Item 2. Properties](index=78&type=section&id=Item%202.%20PROPERTIES) The company leases its corporate headquarters in Boston and laboratory and office space in Vienna, Austria - The company's corporate headquarters is a leased **28,000 sq. ft.** office space in Boston, MA, with the lease expiring November 30, 2026[406](index=406&type=chunk) - A new lease for **1,200 square meters** of laboratory and office space in Vienna, Austria was signed, which expires in March 2027[407](index=407&type=chunk) [Item 3. Legal Proceedings](index=78&type=section&id=Item%203.%20LEGAL%20PROCEEDINGS) The company is not currently a party to any material legal proceedings - The company is not currently subject to any material legal proceedings[408](index=408&type=chunk) [Item 4. Mine Safety Disclosures](index=78&type=section&id=Item%204.%20MINE%20SAFETY%20DISCLOSURES) This item is not applicable to the company - Not applicable[409](index=409&type=chunk) Part II [Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=79&type=section&id=Item%205.%20MARKET%20FOR%20REGISTRANT'S%20COMMON%20EQUITY%2C%20RELATED%20STOCKHOLDER%20MATTERS%20AND%20ISSUER%20PURCHASES%20OF%20EQUITY%20SECURITIES) The company's common stock trades on Nasdaq (XFOR), and it does not anticipate paying dividends - Following the merger in March 2019, the company's common stock began trading on the **Nasdaq Global Market under the symbol "XFOR"**[412](index=412&type=chunk) - The company has **never declared or paid cash dividends** and does not intend to in the foreseeable future[414](index=414&type=chunk) [Item 6. Selected Financial Data](index=79&type=section&id=Item%206.%20SELECTED%20FINANCIAL%20DATA) As a smaller reporting company, this disclosure is not required - The company is not required to provide this disclosure as it qualifies as a **smaller reporting company**[417](index=417&type=chunk) [Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=80&type=section&id=Item%207.%20MANAGEMENT'S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) The company's net loss widened to $62.1 million amid rising R&D costs, raising substantial going concern doubts Results of Operations Comparison (2020 vs. 2019) | (in millions) | 2020 | 2019 | Change | | :--- | :--- | :--- | :--- | | **License revenue** | **$3.0** | **$—** | **$3.0** | | Research and development | $41.9 | $30.2 | $11.7 | | General and administrative | $21.0 | $17.6 | $3.4 | | **Total operating expenses** | **$62.9** | **$51.7** | **$11.2** | | Loss from operations | ($59.9) | ($51.7) | ($8.2) | | **Net loss** | **($62.1)** | **($52.8)** | **($9.3)** | - The company's financial condition raises **substantial doubt about its ability to continue as a going concern**, with cash of **$78.7 million** expected to fund operations only into Q1 2022[450](index=450&type=chunk)[451](index=451&type=chunk) - Research and development expenses **increased by $11.7 million to $41.9 million** in 2020, primarily due to higher clinical expenses for the three ongoing mavorixafor trials[438](index=438&type=chunk) - The **COVID-19 pandemic has caused disruptions**, including delayed trial site initiation and slower patient enrollment, which could adversely affect clinical development timelines[429](index=429&type=chunk)[430](index=430&type=chunk) - As of December 31, 2020, the company has borrowed an aggregate of **$32.5 million** under its loan agreement with Hercules Capital[455](index=455&type=chunk) [Item 7A. Quantitative and Qualitative Disclosures About Market Risk](index=93&type=section&id=Item%207A.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURES%20ABOUT%20MARKET%20RISK) As a smaller reporting company, this disclosure is not required - The company is not required to provide this disclosure as it qualifies as a **smaller reporting company**[483](index=483&type=chunk) [Item 8. Financial Statements and Supplementary Data](index=93&type=section&id=Item%208.%20FINANCIAL%20STATEMENTS%20AND%20SUPPLEMENTARY%20DATA) This section indicates the company's financial statements are appended to the report - The required financial statements are appended to the Form 10-K report[484](index=484&type=chunk) [Item 9. Changes in and Disagreements with Accountants on Accounting and Financial Disclosures](index=93&type=section&id=Item%209.%20CHANGES%20IN%20AND%20DISAGREEMENTS%20WITH%20ACCOUNTANTS%20ON%20ACCOUNTING%20AND%20FINANCIAL%20DISCLOSURES) The company reports no disagreements with its accountants on accounting and financial disclosures - None[485](index=485&type=chunk) [Item 9A. Controls and Procedures](index=93&type=section&id=Item%209A.%20CONTROLS%20AND%20PROCEDURES) Management concluded that the company's disclosure controls and internal controls over financial reporting were effective - Management concluded that the company's **disclosure controls and procedures were effective** as of December 31, 2020[487](index=487&type=chunk) - Management concluded that the company's **internal control over financial reporting was effective** as of December 31, 2020, based on the COSO framework[490](index=490&type=chunk) - The annual report does not include an attestation report from its accounting firm regarding internal control as the company is an **"emerging growth company"**[491](index=491&type=chunk) [Item 9B. Other Information](index=94&type=section&id=Item%209B.%20OTHER%20INFORMATION) There is no other information to report in this section - None[493](index=493&type=chunk) Part III [Items 10-14](index=95&type=section&id=Items%2010-14) Required information is incorporated by reference from the company's 2021 Proxy Statement - Information for Items 10, 11, 12, 13, and 14 is **incorporated by reference** from the registrant's 2021 Proxy Statement[495](index=495&type=chunk)[496](index=496&type=chunk)[497](index=497&type=chunk) Part IV [Item 15. Exhibits and Financial Statement Schedules](index=96&type=section&id=Item%2015.%20EXHIBITS%20AND%20FINANCIAL%20STATEMENT%20SCHEDULES) This section lists the financial statements and exhibits filed with the report - The consolidated financial statements are included starting on page F-1 of the report[502](index=502&type=chunk) - All financial statement schedules have been omitted because they are not applicable or the required information is otherwise provided[504](index=504&type=chunk) [Item 16. Form 10-K Summary](index=100&type=section&id=Item%2016.%20FORM%2010-K%20SUMMARY) This item is not applicable, and no summary is provided - None[511](index=511&type=chunk) Financial Statements and Notes [Report of Independent Registered Public Accounting Firm](index=103&type=section&id=Report%20of%20Independent%20Registered%20Public%20Accounting%20Firm) The auditor's report expresses substantial doubt about the company's ability to continue as a going concern - The auditor's report contains an explanatory paragraph expressing **substantial doubt about the Company's ability to continue as a going concern** due to recurring losses and negative cash flows[523](index=523&type=chunk) [Consolidated Financial Statements](index=104&type=section&id=Consolidated%20Financial%20Statements) The company reported a net loss of $62.1 million for 2020 and ended the year with $78.7 million in cash Key Financial Data (Year Ended Dec 31) | (in thousands) | 2020 | 2019 | | :--- | :--- | :--- | | **Balance Sheet** | | | | Cash and cash equivalents | $78,708 | $126,184 | | Total assets | $122,871 | $160,698 | | Total liabilities | $50,072 | $31,478 | | Total stockholders' equity | $72,799 | $129,220 | | Accumulated deficit | ($194,175) | ($132,044) | | **Statement of Operations** | | | | License revenue | $3,000 | $— | | Research and development | $41,932 | $30,163 | | General and administrative | $20,942 | $17,640 | | **Net loss** | **($62,131)** | **($52,807)** | | Net loss per share | ($3.09) | ($4.63) | | **Cash Flows** | | | | Net cash used in operating activities | ($58,818) | ($48,055) | [Notes to Consolidated Financial Statements](index=108&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) The notes detail the going concern issue, license agreements, debt facility, and significant subsequent events - Note 1 reiterates that conditions raise **substantial doubt about the company's ability to continue as a going concern**, as existing cash is only sufficient to fund operations into Q1 2022[541](index=541&type=chunk)[543](index=543&type=chunk) - Note 3 details the license agreement with Abbisko, under which the company recognized **$3.0 million in revenue** in 2020, with potential future milestones up to **$208.0 million**[626](index=626&type=chunk)[627](index=627&type=chunk)[628](index=628&type=chunk) - Note 7 describes the Hercules Loan Agreement, under which **$32.5 million was drawn** as of Dec 31, 2020, and includes a minimum cash covenant effective January 1, 2022[647](index=647&type=chunk)[649](index=649&type=chunk) - Note 13 discloses significant U.S. federal and state **net operating loss (NOL) carryforwards of $242.6 million and $237.5 million**, respectively, which are subject to a full valuation allowance[701](index=701&type=chunk)[704](index=704&type=chunk) - Note 16 discloses significant subsequent events in March 2021, including a private placement to raise **gross proceeds of approximately $55.0 million** and a potential co-development agreement for up to an **additional $65.0 million**[712](index=712&type=chunk)[714](index=714&type=chunk)

X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q4 2020 Results Earnings Conference Call March 4, 2021 8:30 AM ET Company Participants Daniel Ferry - LifeSci Advisors Paula Ragan - Chief Executive Officer Adam Mostafa - Chief Financial Officer Dr. Diego Cadavid - Chief Medical Officer Art Taveras - Chief Scientific Officer Mary DiBiase - Senior Vice-President, Technical Operations and Quality Conference Call Participants Stephen Willey - Stifel Joel Beatty - Citi Leland Gershell - Oppenheimer Swayampakula Ramakanth ...