On November 5, 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, reported financial results for the third quarter ended September 30, 2020 (Press release, X4 Pharmaceuticals, NOV 5, 2020, View Source [SID1234570016]). The company also provided an update on its lead investigational candidate, mavorixafor, a novel small molecule in a Phase 3 clinical trial for patients with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome and in two Phase 1b trials in patients with Waldenström’s macroglobulinemia and Severe Congenital Neutropenia (SCN), respectively.

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"The third quarter was highlighted by the publication of our positive Phase 2 safety and efficacy data for mavorixafor in WHIM syndrome in the prestigious journal, Blood, which, we believe, continues to demonstrate the significant potential of our lead candidate to treat this patient population," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "Further, in early October, we announced that the U.S. Food and Drug Administration (FDA) granted mavorixafor Fast Track Designation for the treatment of WHIM, facilitating expedited review of mavorixafor as we proceed through clinical development. Both achievements enhance our confidence in mavorixafor’s potential to deliver the first disease-modifying therapy for this undertreated patient population. We were also thrilled to enhance our leadership team and expertise through the additions of Art Taveras, Ph.D., as Chief Scientific Officer and new board member, Alison Lawton."

Dr. Ragan continued, "While the operating environment remains challenging due to the ongoing COVID-19 pandemic, we continue to advance our mavorixafor development programs and enroll patients into our clinical trials. We anticipate initial data from our Phase 1b Waldenström’s macroglobulinemia trial in the first half of 2021, along with initial data from our Phase 1b trial in patients with SCN in 2021, and top-line results from our Phase 3 trial of mavorixafor in WHIM syndrome in 2022. As we continue to achieve important clinical progress," Dr. Ragan concluded, "we are supported by a strong balance sheet that we expect will drive our current strategic and development plans into early 2022."

Recent Highlights
•Published comprehensive data from the Phase 2 and open label extension study of mavorixafor in WHIM syndrome in Blood, the official journal of the American Society of Hematology (ASH) (Free ASH Whitepaper), expanding on previously presented data. New results included long-term patient-level data regarding the effect on neutrophils and lymphocytes and the effect of increasing doses of mavorixafor on total white blood cell counts and monocytes. In addition, the manuscript provided the most up-to-date pharmacokinetic data and presented a detailed analysis of the clinical improvements in infection rates and warts demonstrated with extended mavorixafor treatment.

•Granted Fast Track Designation by the FDA for mavorixafor in WHIM syndrome. Through the Fast Track program, X4 will be eligible for more frequent meetings with the FDA to discuss mavorixafor’s development plan, protocols and clinical data that would support its potential approval for WHIM. Mavorixafor was previously granted Breakthrough Therapy Designation by the FDA, as well as Orphan Drug status by the FDA and the European Commission for the treatment of WHIM syndrome

•Appointed Art Taveras, Ph.D., as Chief Scientific Officer. Dr. Taveras is a seasoned Chief Scientific Officer with experience in the discovery of novel, next-generation CXCR2 antagonists for the treatment of cancer. Previously, Dr. Taveras was VP, Small Molecule Drug Discovery at Biogen, President and CSO of ShangPharma ChemPartners, and CSO of CoMET Therapeutics. With his expertise in chemokine-related chemistries, Dr. Taveras is well suited to lead X4’s R&D initiatives and its continued evolution into a global rare disease company.

•Expanded Board of Directors through the appointment of biopharmaceutical industry veteran, Alison Lawton. Ms. Lawton strengthens the board’s drug development, manufacturing and commercialization experience and brings a unique understanding of X4’s core scientific and corporate goals, having previously served as a consulting Chief Operating Officer to the company and as a member of X4’s corporate advisory board. Ms. Lawton had an over 20 year tenure at Genzyme Corporation (now Sanofi Genzyme) including Senior Vice President and General Manager of Sanofi Biosurgery, a $750 million business and led global functional organizations, including regulatory affairs and quality systems, public policy, health outcomes and strategic pricing, product safety and risk management. Ms. Lawton most recently served as CEO of Kaleido Biosciences and currently serves as an independent director on the Board of ProQR Therapeutics NV.
Third Quarter 2020 Financial Results
•Cash, Cash Equivalents & Restricted Cash: X4 had $90.7 million in cash, cash equivalents and restricted cash as of September 30, 2020. X4 continues to expect that its cash and cash equivalents will fund company operations into early 2022. Note this guidance does not include the $25 million in potential and contingent proceeds from our Hercules Debt Facility or any cash exercise proceeds from investors holding our outstanding warrants.

•Research and Development Expenses were $11.4 million for the third quarter ended September 30, 2020, as compared to $8.6 million for the comparable period in 2019. R&D expenses include $1.0 million of certain non-cash expenses for the quarter ended September 30, 2020.

•General and Administrative Expenses were $5.6 million for the third quarter ended September 30, 2020, as compared to $4.4 million for the comparable period in 2019. G&A expenses include $1.4 million of certain non-cash expenses for the quarter ended September 30, 2020.
•Net Loss: X4 reported a net loss of $17.4 million for the third quarter ended September 30, 2020 as compared to a net loss of $17.7 million for the comparable period in 2019. Net loss includes $2.5 million of certain non-cash expenses for the quarter ended September 30, 2020. Net loss in the third quarter of 2019 included a $4.0 million loss on the sale of non-financial assets.

Conference Call and Webcast
The Company will host a conference call and webcast today at 8:30 a.m. ET to discuss these financial results and business highlights. The conference call can be accessed by dialing (866) 721-7655 from the United States or (409) 216-0009 internationally, followed by the conference ID: 1096977. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the website.