On March 12, 2020 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, provided a corporate update and reported financial results for the fourth quarter and full year ended December 31, 2019 (Press release, X4 Pharmaceuticals, MAR 12, 2020, View Source [SID1234555458]).

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"2019 was a remarkable year for X4, with significant achievements across the entire organization," said Paula Ragan, Ph.D., President and Chief Executive Officer of the Company. "We made important progress advancing our lead therapeutic candidate mavorixafor into pivotal Phase 3 development for patients with WHIM syndrome, while also initiating two proof-of-concept clinical trials in SCN and Waldenström’s, and strengthening both our leadership team and Board of Directors. We’re now well positioned for the years ahead as we focus on near-term clinical trial execution and prepare for key value creation events in our quest to bring new transformative therapies to patients with rare diseases."

Key 2019 Program Achievements and Upcoming Milestones

•Initiated Pivotal Phase 3 Clinical Trial of Mavorixafor for the Treatment of WHIM Syndrome – June 2019: The 4WHIM trial is a pivotal Phase 3 global clinical trial of mavorixafor for the treatment of WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease.
◦Top-line data from the trial are expected in the second half of 2021.
◦Company to hold Analyst Day, which will also be webcast, on April 7, 2020 to discuss strategic focus on WHIM.
◦Phase 2 open-label extension study data update expected in mid-2020.
•Received Orphan Drug Designation from the European Commission for Mavorixafor for the Treatment of WHIM Syndrome – July 2019
◦Received Scientific Advice from the European Medicines Agency to align the Phase 3 registration trial globally.
•Announced Positive Data from Phase 2a Trial of Mavorixafor in Clear Cell Renal Cell Carcinoma (ccRCC) Patients – September 2019: Data presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) demonstrated that mavorixafor, in combination with axitinib yielded clinically meaningful improvements in median progression-free survival (mPFS) in a heavily pretreated advanced ccRCC patient population, and was generally well tolerated.

•Initiated Phase 1b Clinical Trial of Mavorixafor in Patients with Severe Congenital Neutropenia (SCN) – November 2019: The Phase 1b trial is a 14-day, proof-of-concept trial designed to assess the safety and tolerability of daily, oral mavorixafor in up to 45 patients with SCN and other selected congenital neutropenia disorders. The trial will evaluate the neutrophil response and genetic profiles in this patient population as an independent agent or in combination with granulocyte-colony stimulating factor (G-CSF).
◦Initial data from this trial are expected in the second half of 2020.
•Received Breakthrough Therapy Designation from U.S. FDA for Mavorixafor for the Treatment of WHIM Syndrome – November 2019: Highlighting the severity of the disease and the relevance of mavorixafor’s Phase 2 clinical trial data to support the drug’s role as a potential disease-modifying therapeutic option to this underserved patient population, Breakthrough Therapy Designation could expedite the development and regulatory review of mavorixafor.
•Initiated Phase 1b Clinical Trial of Mavorixafor in Combination with Ibrutinib in Patients with Waldenström’s Macroglobulinemia – December 2019: The Phase 1b multi-center, open-label, dose-escalation clinical trial is designed to assess the safety and tolerability of mavorixafor in combination with ibrutinib as well as to obtain certain efficacy signals in patients with Waldenström’s, a rare form of non-Hodgkin’s lymphoma, who have acquired a "gain of function" mutation in CXCR4 in addition to the MYD88 mutation, which is a hallmark of Waldenström’s.
◦Initial data from this trial are expected in the second half of 2020.
•Granted New Composition of Matter Patent by U.S. PTO for Mavorixafor – February 2020: Patent expected to provide exclusivity through 2038.

Key 2019 Corporate Highlights

•Shares of X4 Pharmaceuticals Began Trading on the Nasdaq Capital Market Under the Symbol "XFOR" — March 2019
•Completed Two Public Stock Offerings that Raised Gross Proceeds of $150.8 Million: The first offering was completed in April and the second offering was completed in November.
•Announced Multiple Development and Collaboration Agreements:
◦Announced Collaboration with The Leukemia & Lymphoma Society (LLS) – May 2019: Mavorixafor selected for LLS’ Therapy Acceleration Program (TAP), a strategic initiative creating an alliance to develop mavorixafor for patients with Waldenström’s macroglobulinemia.
◦Announced Partnership with Invitae to Provide No-Cost Genetic Testing to Patients – June 2019: The collaborative PATH4WARD program provides greater access to faster and earlier diagnosis for individuals who may carry genetic mutations known to be associated with WHIM syndrome and SCN.
◦Entered into Oncology Development and Commercialization Agreement with Abbisko for Mavorixafor in Greater China – July 2019: Provided Abbisko Therapeutics with the exclusive rights in China, Taiwan, Hong Kong, and Macau to develop and commercialize mavorixafor in combination with checkpoint inhibitors or other agents in solid tumor oncology indications.
•Strengthened Leadership Team and Board of Directors Throughout the Year: Appointed E. Lynne Kelley, M.D., as Chief Medical Officer and Murray W. Stewart, M.D., to Board of Directors in April 2019; appointed Renato Skerlj, Ph.D., as Senior Vice President of Research and Development and William E. Aliski to Board of Directors in September 2019; appointed Derek Meisner, J.D., as General Counsel in November 2019.

Financial Results

•Cash, Cash Equivalents & Restricted Cash: X4 had $128.1 million in cash, cash equivalents and restricted cash, as of December 31, 2019. We expect that our cash and cash equivalents will fund our operations into early 2022.
•Research and Development Expenses were $7.1 million for the fourth quarter of 2019, and $30.2 million for the year ended December 31, 2019, as compared to $4.7 million and $20.3 million for the comparable periods in 2018, respectively.
•General and Administrative Expenses were $3.9 million for the fourth quarter of 2019, and $17.6 million for the year ended December 31, 2019, as compared to $3.4 million and $8.7 million for the comparable periods in 2018, respectively.
•Net Loss: X4 reported a net loss of $10.8 million for the fourth quarter of 2019, and a net loss of $52.8 million for the year ended December 31, 2019, as compared to a net loss of $11.3 million and a net loss of $33.3 million for the comparable periods in 2018, respectively. Net loss of $52.8 million for the year ended December 31, 2019 includes $3.9 million of non-cash losses related to the sale of in-process research and development intangible assets.

Conference Call and Webcast
The Company will host a webcast and conference call to discuss its fourth quarter and full year 2019 results and provide an update on recent corporate activities today at 8:30 a.m. Eastern Time. The webcast will be accessible under "Events & Presentations" in the Investors page of the Company’s website at www.x4pharma.com. Individuals can participate in the conference call by dialing (866) 721-7655 (domestic) or (409) 216-0009 (international), followed by the conference ID: 3816258.

About Mavorixafor
Mavorixafor is an investigational, oral, targeted therapy that antagonizes the chemokine receptor CXCR4 via allosteric inhibition. The CXCR4 receptor plays a key role in enabling the trafficking of immune cells and effective immunosurveillance. Over-stimulation of CXCR4 results in various immune-system deficiencies. X4 Pharmaceuticals is currently investigating mavorixafor as a once-daily treatment in a global Phase 3 pivotal trial in patients with WHIM syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. The FDA has granted Breakthrough Therapy Designation to mavorixafor for the treatment of WHIM syndrome. Mavorixafor is being developed in two proof-of-concept Phase 1b clinical trials – as monotherapy in patients with Severe Congenital Neutropenia (SCN), a group of rare blood disorders characterized by abnormally low levels of white blood cells, and in combination with ibrutinib in patients with Waldenström’s macroglobulinemia, a form of non-Hodgkin’s lymphoma, who have acquired mutations in the CXCR4 receptor.