NASDAQ:XFOR
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Business Update
Update on Chronic CN Program
Chronic neutropenia (CN) is a disease that is defined as having severe, chronic (> 3 months) low levels of circulating neutrophils. Its etiology can be idiopathic (unknown origin), cyclic (neutrophil levels rise and fall in a cycle), or congenital (genetic cause). The following slide shows the results of a market survey conducted by X4 (NASDAQ:XFOR) to better define the target market for mavorixafor in CN. It is estimated there are a total of approximately 50,000 CN patients in the U.S., with an initial target market of approximately 15,000 individuals with high unmet needs (adolescents and adults with severe/recurrent infections and/or G-CSF treatment).
X4 initially evaluated mavorixafor in a successful single-dose Phase 1b study in patients with CN. Patients with all CN disorders (idiopathic, congenital, and cyclic) responded to mavorixafor treatment, with an increase in ANC of >2,000 cells/μL across all disorders. In severe neutropenia, all patients showed increased ANC to normal levels, which shows the potential for mavorixafor monotherapy. In addition, mavorixafor increased ANCs to normal levels in patients who were being treated with G-CSF, which supports further studies exploring the role of mavorixafor in replacing G-CSF therapy in these patients. Lastly, mavorixafor was well tolerated, all treatment-related adverse events were deemed to be low grade and consistent with what was seen in previous trials, and there were no treatment-related serious adverse events.
X4 is currently conducting a Phase 2 trial of mavorixafor in CN and announced preliminary data from the first three patients that completed the six-month trial in December 2023. The results showed that mavorixafor continues to be well tolerated when used in combination with G-CSF. X4 achieved its target of at least 15 patients enrolled into the Phase 2 trial in early November 2023, and the company will be conducting an investor event in June 2024 to present data from the trial.
The company completed the study design for a pivotal Phase 3 trial in CN, which we anticipate initiating in the second quarter of 2024. The following figure gives an overview of the study design, which will include approximately 150 subjects, a 12-month treatment period, and a two-component primary endpoint: annualized infection rate and ANC response.
XOLREMDI™ Commercial Launch Underway
X4 has initiated the commercial launch of XOLREMDI (mavorixafor) following its approval by the U.S. Food and Drug Administration (FDA) for use in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections, and Myelokathexis). The company’s commercialization strategy includes physician education (including providing diagnostic support for patient testing), establishing XOLREMDI as the standard of care in WHIM syndrome, and ensuring broad access through a suite of patient support services (X4Connect™).
Along with the approval of XOLREMDI, X4 also received a Rare Pediatric Disease Priority Review Voucher (PRV). We anticipate the company monetizing this asset in the near future as PRVs are typically selling for approximately $100 million.
Financial Update
On May 7, 2024, X4 announced financial results for the first quarter of 2024. R&D expenses for the first quarter of 2024 were $19.9 million, compared to $22.1 million for the first quarter of 2023. The decrease in expenses was primarily due to a $5.0 million in-license fee related to a development milestone payed to Genzyme in the first quarter of 2023 partially offset by higher compensation costs. SG&A expenses for the first quarter of 2024 were $17.4 million, compared to $7.2 million in the first quarter of 2023. The increase was due to outstanding stock appreciation rights that are measured at fair value, an increase in sales and marketing personnel, and pre-commercial launch activities.
As of March 31, 2024, X4 had cash, cash equivalents, and restricted cash of approximately $81.6 million. We estimate that the company has sufficient capital to fund operations into 2025, which does not include the potential monetization of the PRV. We estimate that X4 currently has approximately 167.9 million shares outstanding and, when factoring in stock options, restricted stock units, and warrants, a fully diluted share count of approximately 297.1 million.
Conclusion
We’re looking forward to a number of updates from the company in the near term, including the potential monetization of the PRV, updates on the commercial launch of XOLREMDI, and an investor event in June 2024 discussing results from the Phase 2 trial of mavorixafor in CN. With no changes to our model our valuation remains at $5.50 per share.
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