X4 Pharmaceuticals Showcases Strategic Focus on WHIM Syndrome During 2020 Analyst Day Webinar
–Market research confirms
The Analyst Day discussions provided details and background on WHIM (Warts, Hypogamma-globulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited immunodeficiency disease, and included a patient experience via video and an update on recently completed X4 market research on the prevalence of WHIM in the
“It was our goal today to help the investment community visualize the ‘face’ of WHIM syndrome, a disease with severe and life-long medical complications, to better understand the challenges physicians face in treating WHIM, which we believe our late-stage Phase 3 candidate mavorxiafor could address, and to share our updated market research on the prevalence of the disease,” said
Understanding the Severity of WHIM Syndrome
- The disease burden of WHIM is severe, with frequent respiratory infections that can lead to chronic lung disease, ear infections that may lead to hearing loss, and recurrent human papilloma virus(HPV) infections, often manifesting as warts that can increase the risk of anogenital and head and neck cancers.
- The complications and treatments associated with WHIM create a high demand for healthcare utilization. A study that examined insurance claims records of potential WHIM patients identified through an artificial intelligence algorithm demonstrated that 82% of these patients require respiratory services, 18% experience hearing loss, 51% had visited the emergency department and 44% had been hospitalized within the last year.
The WHIM Treatment Landscape: Clear Unmet Medical Need
- Current therapeutics used to treat WHIM are limited in efficacy, only treating symptoms and not addressing the underlying cause of the disease.
- X4 is developing mavorixafor, a first-in-class antagonist of CXCR4 that has demonstrated high potency and selectivity for inhibiting CXCR4 signaling and restoring the trafficking of neutrophils and lymphocytes to improve immunosurveillance.
Informed by a successful Phase 2 trial, X4 has is conducting an ongoing global Phase 3 trial for mavorixafor in genetically confirmed WHIM patients; mavorixafor recently received Breakthrough Therapy Designation for WHIM in the
U.S.suggesting recognition of the positive Phase 2 clinical data and the high unmet medical need in WHIM patients.
New Market Research Supports Heightened Market Opportunity
During 2019, X4 conducted a broad, quantitative online survey of physicians, the results of which support the conclusion that there are between 1,000 and 1,300 diagnosed WHIM patients in the
- X4 also performed research using artificial intelligence, interrogating a database of more than 300 million anonymized patient records that spanned 10 years of insurance claims to help identify patient records that reflect the ‘face’ of WHIM. This robust algorithm then searched the database, identifying between 800 and 2,400 additional potential but unconfirmed and undiagnosed WHIM patients.
Based on this research, X4 has provided updated guidance on its estimated range of WHIM prevalence to be between approximately 1,300 diagnosed patients and up to 3,700 in total, which includes potential undiagnosed WHIM patients, a significant increase from its prior estimate of approximately 1,000 WHIM patients in the
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” or other similar terms or expressions that concern X4's expectations, strategy, plans or intentions. Forward-looking statements include, without limitation, statements regarding X4’s clinical trials relating to WHIM, along with its prevalence. These statements are subject to various risks and uncertainties including, without limitation, the risk that trials and studies may be delayed and may not have satisfactory outcomes, potential adverse effects arising from the testing or use of mavorixafor or other product candidates, and the risk that costs required to develop product candidates or to expand X4’s operations will be higher than anticipated. Any forward-looking statements in this press release are based on management's current expectations and beliefs. Actual events or results may differ materially from those expressed or implied by any forward-looking statements contained herein, including, without limitation, the risks and uncertainties described in the section entitled “Risk Factors” in X4’s Annual Report on Form 10-K filed with the
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