NASDAQ:XFOR
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Business Update
XOLREMDI™ Approved by the FDA
On April 29, 2024, X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) announced that the U.S. Food and Drug Administration (FDA) has approved XOLREMDI™ (mavorixafor) for use in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections, and Myelokathexis). This is the first therapy specifically indicated for WHIM syndrome. 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.
During the conference call to announce the approval of XOLREMDI, X4 management provided an outline of the company’s commercialization strategy, which 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™).
In regards to pricing, X4 announced an annual wholesale acquisition cost (WAC) of $496,400 for patients >50 kg (400 mg daily) and $372,300 for patients ≤50 kg (300 mg daily; assuming full patient compliance). The company has partnered with PantheRx Rare, a specialty pharmacy that will process and fill XOLREMDI prescriptions and provide information on treatment, potential side effects, and confirm treatment dosing.
We do not anticipate a bolus of patients ready to begin treatment right away, but instead the company will focus on increasing physician and payer awareness of XOLREMDI during the rest of 2024 such that sales can begin to ramp up in 2025/2026. The company has assembled a sales force of approximately two dozen representatives who collectively have over 250 years of experience in commercial launches. When a patient begins treatment, the X4Connect program will be utilized to help them navigate insurance coverage, any prior authorization requirements, and introduce them to financial assistance programs.
Conclusion
We’re very glad to see the approval of XOLREMDI and are confident that X4 has a strong plan in place to ensure a successful commercial launch of the drug. We assume that the PRV will be monetized in the near future and are confident that it will be sold in-line with previous PRVs (~$100 million). Aside from the commercial launch of XOLREMDI, we continue to anticipate Phase 2 chronic neutropenia data in the first half of 2024. We have adjusted our model to account for the fact that XOLREMDI has been approved, which has caused a slight increase in our valuation to $5.50 per share.
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