BOSTON - X4 Pharmaceuticals (NASDAQ:XFOR) announced today the U.S. Food and Drug Administration (FDA) has approved XOLREMDI™ (mavorixafor) capsules for patients aged 12 and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes.
This marks the first therapy specifically indicated for WHIM syndrome, a rare primary immunodeficiency and chronic neutropenic disorder caused by CXCR4 pathway dysfunction.
The approval was based on the 4WHIM Phase 3 clinical trial, which demonstrated that XOLREMDI increased time above threshold for absolute neutrophil and lymphocyte counts versus placebo. The trial also showed a reduction in the rate of infections among patients treated with the drug.
Paula Ragan, Ph.D., President and CEO of X4 Pharmaceuticals, expressed gratitude to the WHIM syndrome community and regulatory bodies for their support in bringing this targeted therapy to fruition. The Immune Deficiency Foundation also welcomed the approval, noting the significant advancement for people living with the condition.
According to the press release, the most common adverse reactions in the trial included thrombocytopenia, pityriasis, rash, rhinitis, epistaxis, vomiting, and dizziness. XOLREMDI is contraindicated with drugs highly dependent on CYP2D6 for clearance and carries a warning for embryo-fetal toxicity.
In conjunction with the approval, X4 received a Rare Pediatric Disease Priority Review Voucher, which can be used for priority review of a subsequent application or sold to another drug sponsor.
X4 also launched X4Connect, a support program for U.S. patients, providing resources and assistance for those undergoing treatment with XOLREMDI, which will be available through specialty pharmacy partner PANTHERx® Rare.
InvestingPro Insights
As X4 Pharmaceuticals (NASDAQ:XFOR) celebrates the FDA's approval of XOLREMDI™ for WHIM syndrome, the financial health and stock performance of the company provide a broader context for investors. According to real-time data from InvestingPro, X4 Pharmaceuticals holds a market capitalization of $195.65 million. Despite the positive clinical outcomes and recent FDA approval, the company's financial metrics indicate challenges, with an adjusted P/E ratio for the last twelve months as of Q4 2023 standing at -1.93, reflecting the company's current lack of profitability.
The company's stock has experienced volatility with a significant price uptick over the last six months, showing a 64.52% total return, yet the one-year price total return indicates a decrease of 20.75%. This suggests that while the stock has had strong short-term gains, it has faced challenges over a longer period.
Among the InvestingPro Tips for X4 Pharmaceuticals, two stand out. First, despite the company's rapid cash burn, it holds more cash than debt on its balance sheet, which could provide some financial flexibility in the near term. Second, analysts have revised their earnings upwards for the upcoming period, indicating a potential shift in the company's financial trajectory post-approval of XOLREMDI™.
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