On November 7, 2019 -X4 Pharmaceuticals, Inc. (Nasdaq:XFOR), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, reported financial results for the third quarter ended September 30, 2019 and provided an update on recent developments in its business (Press release, X4 Pharmaceuticals, NOV 7, 2019, View Source [SID1234550660]).
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"Our core focus remains to advance our clinical programs with mavorixafor across our three lead rare disease indications. We are proud to report that we now have orphan drug designation in Europe, in addition to in the United States, for mavorixafor for treatment of Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome. We recently commenced enrollment in our pivotal Phase 3 clinical trial for the treatment of WHIM syndrome and are activating clinical sites globally. We have also now initiated the Phase 1b clinical trial in Severe Congenital Neutropenia. Preparations are underway for the initiation of the Phase 1b clinical trial in Waldenström’s macroglobulinemia later this quarter," stated Paula Ragan, Ph.D., President and Chief Executive Officer of X4.
Clinical Development Update
In November 2019, X4 initiated a Phase 1b clinical trial of mavorixafor (X4P-001) for the treatment of Severe Congenital Neutropenia (SCN), a group of rare blood disorders characterized by abnormally low levels of neutrophils.
X4 commenced U.S. patient enrollment and is activating additional sites globally in the pivotal Phase 3 global clinical trial of mavorixafor in WHIM syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. The 52-week trial is designed to enroll 18 to 28 subjects in approximately 20 countries, followed by an open-label extension trial.
In September 2019, at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper), X4 released positive results from the Phase 2a portion of its open-label Phase 1/2 clinical trial of mavorixafor in combination with axitinib (Inlyta) in patients with advanced clear cell renal cell carcinoma (ccRCC). The combination therapy was observed to generally be well-tolerated with a manageable safety profile and demonstrated clinical improvement with encouraging median progression free survival (mPFS) in a heavily pretreated advanced ccRCC patient population. Results suggest that mavorixafor may enhance clinical response to axitinib and other tyrosine kinase inhibitors (TKIs) that target tumor angiogenesis, as well as immunotherapy agents. The Company is in ongoing dialogue with potential partners to explore the potential benefit of mavorixafor in underserved cancer patients with solid tumors, including as a potential triple combination agent in addition to TKI and checkpoint inhibitor therapies or in combination with other standard of care treatments.
Business Update
In July 2019, X4 entered into an agreement with Abbisko Therapeutics where Abbisko agreed to develop and commercialize mavorixafor in combination with checkpoint inhibitors or other agents in Greater China for oncology indications with a focus on solid tumor indications. X4 retained full rest-of-world rights to develop and commercialize mavorixafor outside of Greater China for all indications, and the ability to utilize any data generated from the collaboration for X4’s rest-of-world development programs.
In July 2019, X4 was notified that the European Commission (EC) had approved orphan drug designation (ODD) for mavorixafor for the treatment of WHIM syndrome, which follows the orphan drug designation X4 received from the U.S. Food and Drug Administration in October 2018 for the same indication.
In September 2019, X4 appointed William "Bill" E. Aliski to its Board of Directors. Mr. Aliski has more than two decades of biopharmaceutical executive leadership experience at both public and private companies, with significant expertise in global rare disease commercialization, including a particular focus on commercial strategy, pricing, reimbursement and market access.
In September 2019, X4 appointed Renato Skerlj, Ph.D., as its Senior Vice President, Research and Development. Dr. Skerlj has twenty-five years of experience leading the discovery and development of small molecule drugs to treat rare diseases, cancer, infection and neurodegenerative diseases. In addition, he is one of the original founders of X4 Pharmaceuticals.
In November 2019, X4 appointed Derek Meisner, J.D., as its General Counsel. Mr. Meisner brings more than two decades of experience providing counsel to public and private biotechnology companies across key legal and operational functions, including global regulatory compliance, financings, mergers and acquisitions, strategic partnerships and corporate governance.
Third Quarter 2019 Financial Highlights
Cash Position: As of September 30, 2019, cash, cash equivalents and restricted cash were $77.0 million, as compared to $95.6 million as of June 30, 2019. This decrease reflected cash used to fund operating activities during the third quarter and included a $6.5 million cash payment for the settlement of X4’s loans with Österreichische Forschungsförderungsgesellschaft mbH (FFG). This repayment was part of the debt refinancing executed with Hercules in June 2019, under which X4 has a remaining $5 million in borrowing availability.
Research and Development Expenses (R&D): R&D expenses were $8.6 million for the third quarter of 2019, as compared to $8.9 million for the second quarter of 2019.
General and Administrative Expenses (G&A): G&A expenses were $4.4 million for the third quarter of 2019, as compared to $4.6 million for the second quarter of 2019.
Loss on Transfer of Non-Financial Assets: During the third quarter of 2019, X4 transferred to third parties the rights to develop and commercialize the programs underlying its in-process research and development (IPR&D) intangible assets, which were obtained in its merger with Arsanis. Accordingly, X4 recorded a $4.0 million loss during the three months ended September 30, 2019 reflecting the derecognition of the IPR&D intangible assets, partially offset by cash proceeds received in the quarter.
Net Loss: Net loss was $17.7 million for the third quarter of 2019, or a net loss per basic and diluted share of $1.22, as compared to a net loss of $13.4 million for the second quarter of 2019, or a net loss per basic and diluted share of $1.02. Excluding the loss on transfer of non-financial assets, net loss for the third quarter of 2019 was approximately the sum of the R&D and G&A expenses in the quarter.
X4 expects current cash and cash equivalents to be sufficient to fund operations into the first half of 2021.
Conference Call and Webcast Information
X4 will host a conference call and webcast on November 7, 2019 at 8:00 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: 4081686. The live webcast can be accessed on the investor relations section of X4’s website at View Source Following the completion of the call, a webcast replay of the conference call will be available on X4’s website for thirty days.
About Mavorixafor
X4 Pharmaceuticals’ lead product candidate, mavorixafor (X4P-001), is a potential first-in-class, once-daily, oral inhibitor of CXCR4, currently in a Phase 3 clinical trial or the treatment of WHIM syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. Mavorixafor has demonstrated proof-of-concept in WHIM syndrome in a Phase 2 clinical trial, including clinically meaningful increases in neutrophil and lymphocyte biomarker counts, as well as a trend of reduction in infection rates and wart burden, and a favorable safety profile. Mavorixafor was designated orphan drug status by the U.S. Food and Drug Administration in 2018 and by the European Commission in 2019 for the treatment of WHIM syndrome, and is also in development for Severe Congenital Neutropenia (SCN), Waldenström’s macroglobulinemia (WM), and clear cell renal cell carcinoma (ccRCC).