On July 30, 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 second quarter ended June 30, 2020. 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 (WM) and Severe Congenital Neutropenia (SCN) (Press release, X4 Pharmaceuticals, JUL 30, 2020, View Source [SID1234562616]).

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"Despite the challenges posed by the ongoing COVID-19 pandemic, we achieved significant progress during the second quarter, benefiting from two key events related to the WHIM syndrome indication for mavorixafor, while continuing to advance our program in the Waldenström’s indication," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "At our Analyst Day in early April, we presented market research data that supported significantly increasing our disease prevalence estimate for WHIM in the U.S., and during our presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress in June, we highlighted Phase 2 mavorixafor data that strengthen our confidence in mavorixafor’s potential to be a disease-modifying therapy in patients with WHIM syndrome and in the Phase 3 trial design."

Dr. Ragan continued, "While our public presentations during the first half of 2020 focused primarily on WHIM syndrome, we expect that the second half of 2020 will see us increase exposure to our program in patients with Waldenström’s macroglobulinemia, a rare form of lymphoma, where we look forward to announcing initial Phase 1b clinical results towards the end of the year." The ongoing Phase 1b clinical trial is expected to enroll between 12 and 18 patients with WM and is a multi-center, open-label, dose-escalation clinical trial assessing the safety and tolerability of mavorixafor in combination with ibrutinib. The trial is being conducted as part of a collaboration with The Leukemia & Lymphoma Society to accelerate the development of mavorixafor for the treatment of WM.

"As we await these important results," Dr. Ragan concluded, "we continue to expect top-line Phase 3 results of mavorixafor in WHIM syndrome in 2022 and initial data from our Phase 1b trial of mavorixafor in patients with SCN in 2021. In light of the continued uncertainties surrounding COVID-19, we intend to provide further clarity around these timelines as soon as is practicable."

Recent Highlights
•Increased Guidance on Prevalence of WHIM at Virtual Analyst Day, based on in-depth, internal market research indicating the range of diagnosed and undiagnosed WHIM patients in the United States to be greater than 3,500, a significant increase from the prior estimate of approximately 1,000 patients diagnosed with WHIM syndrome.
•Presented Positive Data from the Phase 2 Open-Label Extension Study of Mavorixafor in WHIM Syndrome at EHA (Free EHA Whitepaper) 2020, supporting the selection of 400 mg once-daily and time above threshold for absolute neutrophil counts (TATANC) as the dose and primary endpoint in the Phase 3 trial, respectively, and long-term favorable tolerability. At the median follow-up of 16.5 months, data revealed sustained, dose-dependent increases in WBC (white blood cells), ANC (absolute neutrophil count), and ALC (absolute lymphocyte count), with higher doses of mavorixafor shown to increase the TATANC at least 4.5-fold versus lower doses. In patients treated for at least 6 months, mavorixafor also significantly decreased the yearly rate of infections versus the 12 months prior to treatment and effected a 75% reduction in cutaneous warts versus baseline.
•Promoted Renato Skerlj, Ph.D., to the position of Chief Scientific Officer. Dr. Skerlj, one of the scientific founders of X4 and co-inventor of mavorixafor, has more than 25 years of experience leading the discovery and development of disease-modifying small molecule drugs to treat genetically defined rare diseases. In this expanded role, Dr. Skerlj leads all research and non-clinical development functions at X4, overseeing operations at the company’s Vienna, Austria research facility as well as the company’s efforts to advance and expand its pipeline targeting additional rare diseases.

Second Quarter 2020 Financial Results
•Cash, Cash Equivalents & Restricted Cash: X4 had $105.6 million in cash, cash equivalents and restricted cash, as of June 30, 2020. X4 continues to expect that its cash and cash equivalents will fund company operations into early 2022. In addition, X4 continues to have $25 million of potential borrowing capacity under its amended credit agreement with Hercules.
•Research and Development Expenses were $9.3 million for the second quarter ended June 30, 2020, as compared to $8.9 million for the comparable period in 2019.
•General and Administrative Expenses were $5.3 million for the second quarter ended June 30, 2020, as compared to $4.6 million for the comparable period in 2019.
•Net Loss: X4 reported a net loss of $15.1 million for the second quarter ended June 30, 2020 as compared to a net loss of $13.4 million for the comparable period in 2019.

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: 6091009. 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.