X4 Pharmaceuticals Receives Breakthrough Therapy Designation from the FDA for Mavorixafor for the Treatment of WHIM Syndrome
Mavorixafor is a potential first-in-class, once-daily, oral, small molecule antagonist of chemokine receptor CXCR4, and is currently being investigated in a pivotal Phase 3 global clinical trial, 4WHIM, for the treatment of WHIM syndrome. The Breakthrough Therapy Designation granted to mavorixafor is based on data from X4’s Phase 2 open-label, multi-center trial of mavorixafor in adult patients with WHIM syndrome. In this trial, proof of concept was established based on clinically meaningful increases in absolute neutrophil counts (ANCs), absolute lymphocyte counts (ALCs), evidence indicating reductions in infection rates and wart burden, and a safety profile showing that mavorixafor is well-tolerated.
“Rare diseases such as WHIM don’t often receive the attention and research that patients and their families deserve. The FDA’s decision to grant Breakthrough Therapy Designation to mavorixafor for the treatment of adults with WHIM syndrome represents a significant milestone for patients and X4 alike, helping to further highlight the severity of this underdiagnosed disease and the importance of offering a potential novel, disease-modifying therapeutic option to this underserved patient population,” said
A Breakthrough Therapy Designation may be granted to expedite the development and regulatory review of an investigational new drug that is intended to treat a serious or life-threatening condition where there is an unmet need. The criteria for Breakthrough Therapy Designation require preliminary clinical evidence that demonstrates that the drug may provide substantial improvements over any available therapy on at least one clinically significant endpoint.
Mavorixafor was granted orphan drug status by the
About WHIM Syndrome
WHIM syndrome is a rare, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene and is named for the characteristic clinical symptoms of the syndrome – Warts, Hypogammaglobulinemia, Infections, and Myelokathexis.1 Patients with WHIM may experience significant morbidity beginning in early childhood and continuing throughout life with an increased likelihood of various recurrent, potentially life-threatening infections, and may also be susceptible to malignancies such as HPV-related cervical cancer and lymphomas.1,2,3 The overall cancer risk in patients with WHIM is estimated to be 30 percent by 40 years of age.4 There are no approved therapies for WHIM, and current standards of care are limited to treatment of acute infections with antibiotics or prevention of infections mainly through immunoglobulin substitution or G-CSF.5 The exact prevalence of WHIM is unknown, however, in the U.S. alone there are between 15,000 and 100,000 patients classified as having a primary immunodeficiency disease of unknown origin – of which WHIM is one.6,7,8
About Mavorixafor
X4 Pharmaceuticals’ lead product candidate, mavorixafor (X4P-001), is a potential first-in-class, once-daily, oral inhibitor of CXCR4, currently in a Phase 3 clinical trial for the treatment of WHIM syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. Mavorixafor has demonstrated proof-of-concept in WHIM syndrome in a Phase 2 clinical trial, including clinically meaningful increases in neutrophil and lymphocyte biomarker counts, as well as a trend of reduction in infection rates and wart burden, and a favorable safety profile. Mavorixafor was granted orphan drug status by the
About
Forward-Looking Statements
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, but are not limited to, statements regarding the clinical development of mavorixafor (X4P-001) or any of X4's other product candidates or programs. These statements are subject to various risks and uncertainties, actual results could differ materially from those projected and X4 cautions investors not to place undue reliance on the forward-looking statements in this press release. These risks and uncertainties include, 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, the risk that costs required to develop mavorixafor or other product candidates or to expand X4’s operations will be higher than anticipated, and the risk that despite the breakthrough therapy designation, the development and regulatory review will not be expedited. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, the risks and uncertainties described in the section entitled “Risk Factors” in X4’s most recent Annual Report on Form 10-K filed with the
_______________ | |
1 |
Primary Immunodeficiency Foundation: https://primaryimmune.org/disease/whim-syndrome |
2 |
McDermott, D and Murphy P, WHIM syndrome: Immunopathogenesis, treatment and cure strategies. Immunological Reviews. 2019;287: 91-102. |
3 |
Arnolds K and Spencer J, CXCR4: A Virus’s Best Friend Infect Genet Evol. 2014 July 25 146-156. |
4 |
Beaussant Cohen S, et al. Description and outcome of a cohort of 8 patients with WHIM syndrome from the French Severe Chronic Neutropenia Registry. Orphanet Journal of Rare Diseases. 2012, 7:71. |
5 |
Badolato R, et al. How I treat warts, hypogammaglobulinemia, infections, and myelokathexis syndrome. Blood. 2017 130: 2491-2498. |
6 |
Boyle JM, Buckley, RH, Population Prevalence of Diagnosed Primary Immunodeficiency Diseases in the United States. J Clin Immunol 2007;27:497–502. |
7 |
Gathmann B, Grimbacher B, et al. The European internet-based patient and research database for primary immunodeficiencies: results 2006–2008. Clin Exp Immunol. 2009 Sep;157 Suppl 1:3-11. |
8 |
Modell V, Gee B, et al. Global study of primary immunodeficiency diseases (PI) — diagnosis, treatment, and economic impact: an updated report from the Jeffrey Modell Foundation. Immunol Res. 2011;51:61–70. |
View source version on businesswire.com: https://www.businesswire.com/news/home/20191112005283/en/
Source:
Investors:
Stephanie Carrington
Westwicke, an ICR company
646-277-1282
Stephanie.Carrington@icrinc.com
Media:
Darcie Robinson
Westwicke, an ICR company
203-919-7905
Darcie.robinson@icrinc.com