On November 3, 2022 X4 Pharmaceuticals Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel small-molecule therapeutics to benefit people with diseases of the immune system, reported financial results for the third quarter ended September 30, 2022 and highlighted recent and upcoming expected milestones (Press release, X4 Pharmaceuticals, NOV 3, 2022, View Source [SID1234623001]).

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"This is a truly exciting time for X4 as we near the announcement of top-line data from our first pivotal clinical trial and continue to advance our lead candidate, mavorixafor, towards commercialization," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "Following our September release of positive data from the Phase 1b clinical trial of mavorixafor across a number of chronic neutropenic disorders, we now eagerly await the results from the 4WHIM registration trial. With no oral therapeutic approved for these patients, we believe mavorixafor, if approved, could represent a new opportunity to transform the treatment landscape and create a new standard of care for almost 50,000 underserved patients in the U.S."

Recent Highlights & Anticipated Upcoming Milestones
•In July, X4 completed a private investment in public equity (PIPE) financing, receiving aggregate gross proceeds of approximately $55 million; the financing included participation from new and existing investors.
•In July, the company announced a strategic re-prioritization of resources towards advancing mavorixafor in chronic neutropenic disorder indications, including WHIM syndrome, while progressing oncology programs only upon completion of strategic partnership(s); the announced strategic update was also inclusive of cost-cutting initiatives estimated to extend X4’s cash runway into the third quarter of 2023.
•In late September, X4 held an event highlighting new positive data from its Phase 1b clinical trial demonstrating the ability of mavorixafor to increase and normalize absolute neutrophil counts (ANC) in people with idiopathic, cyclic, or congenital chronic neutropenia (CN) as monotherapy or concurrently with injectable granulocyte colony-stimulating factor (G-CSF). The company believes that these results suggest an expanded market opportunity for mavorixafor that could include almost 50,000 additional diagnosed patients in the U.S.
•The Phase 1b trial in chronic neutropenic disorders is currently being amended and expanded into a Phase 1b/Phase 2 clinical trial to assess the long-term durability, safety, and tolerability of mavorixafor in a larger patient population; X4 anticipates the amended trial to begin generating additional clinical data in the first half of 2023.

Exhibit 99.1
•The company recently announced the appointment of industry veteran Mark Baldry to the position of Chief Commercial Officer. In this key role, Mr. Baldry will lead all pre-commercial and product launch efforts for mavorixafor as clinical development advances.
•Following recent completion of the last patient/last visit in the company’s Phase 3 clinical trial of mavorixafor in WHIM syndrome (the 4WHIM trial), X4 continues to anticipate the announcement of top-line results from the trial in the fourth quarter of 2022 and the submission for U.S. regulatory approval of mavorixafor in WHIM early in the second half of 2023, if the data are positive.

X4 today also announced that its Chief Medical Officer, Dr. Diego Cadavid, intends to depart the company by the end of 2022, but will continue to support X4 as a consultant. Dr. Cadavid, still a practicing neurologist, is leaving to pursue an opportunity that closely aligns with his commitment to patients with intractable CNS disorders.

X4 is appointing Dr. Murray Stewart as interim Chief Medical Officer while it conducts a search for a permanent replacement for Dr. Cadavid. Dr. Stewart, who has served as a member of the X4 Board of Directors since March 2019, has had a distinguished career in the life sciences industry, including an 18-year tenure at GlaxoSmithKline (GSK), where he held multiple research and development leadership roles, including Chief Medical Officer, Clinical Head of the Biopharma Unit, and Therapy Area Head for metabolic and cardiovascular diseases. Dr. Stewart also previously served as Chief Medical Officer of Rhythm Pharmaceuticals, leading the successful NDA submission and approval of ImcivreeTM (setmelanotide), an innovative treatment for rare causes of obesity.

Dr. Stewart commented: "I am very pleased to be able to support X4 through this exciting time, as we prepare to unblind the mavorixafor Phase 3 WHIM data and advance to an NDA submission if the data are positive. I look forward to partnering with the clinical and executive management teams to continue X4’s journey towards providing people with WHIM and other CN disorders with a potentially transformative new therapy."

Third Quarter 2022 Financial Results
•Cash, Cash Equivalents & Restricted Cash: X4 had $81.1 million in cash, cash equivalents, and restricted cash as of September 30, 2022. X4 believes that it has sufficient funds to support company operations into the third quarter of 2023.
•Research and Development (R&D) Expenses were $14.1 million for the third quarter of 2022 as compared to $13.2 million for the comparable period in 2021. R&D expenses include $0.6 million and $0.6 million of certain non-cash expenses for the third quarter of 2022 and 2021, respectively.
•Selling, General, and Administrative Expenses (SG&A) were $6.0 million for the third quarter of 2022 as compared to $5.9 million for the comparable period in 2021. SG&A expenses include $0.5 million and $0.9 million of certain non-cash expenses for the third quarter of 2022 and 2021, respectively.
•Net Loss: X4 reported a net loss of $21.6 million for the third quarter of 2022, as compared to $20.2 million for the comparable period in 2021. Net losses include $1.1 million and $1.5 million of certain non-cash expenses for the third quarter of 2022 and 2021, respectively.
Conference Call and Webcast
X4 will host a conference call and webcast today at 8:30 am ET to discuss important upcoming milestones, including the pending release of results from the company’s Phase 3 trial of its lead candidate, mavorixafor, in the treatment of WHIM syndrome. The conference call can be accessed by dialing 1-855-327-6837 within the United States or 1-631-891-4304 internationally, followed by the

Exhibit 99.1
conference ID: 10020371. 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.