RegulationRare Pediatric Disease (United States), Orphan Drug (European Union), Fast Track (United States), Breakthrough Therapy (United States), Priority Review (United States)

Structure/Sequence

Molecular FormulaC21H27N5

InChIKeyWVLHHLRVNDMIAR-IBGZPJMESA-N

CAS Registry558447-26-0

Clinical Trials associated with Mavorixafor

NCT06858696

/ Not yet recruitingPhase 1

An Open-label, Non-randomized Study to Evaluate the Pharmacokinetics (PK), Safety and Tolerability of a Single Dose of Mavorixafor in Participants With Hepatic Impairment (HI) Compared to Matched Healthy Volunteers With Normal Hepatic Function

The purpose of this study is to measure the effect of HI on the PK, safety, and tolerability of a single dose of mavorixafor compared to matched healthy volunteers (HVs) with normal hepatic function.

Start Date24 Feb 2025

Sponsor / Collaborator

X4 Pharmaceuticals, Inc.

NCT06056297

/ RecruitingPhase 3

A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

The purpose of this study is to demonstrate the efficacy and evaluate the safety, and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections as assessed by demonstrating its clinical benefit and increasing levels of circulating neutrophils.

Start Date06 Jun 2024

Sponsor / Collaborator

X4 Pharmaceuticals, Inc.

NCT04154488

/ CompletedPhase 1/2

A Phase 1b/2, Open-Label, Multicenter Study of Mavorixafor in Patients With Congenital Neutropenia and Chronic Neutropenia Disorders

This is a 2-part study of mavorixafor in participants diagnosed with chronic neutropenia. The main goal of Part 1 (Phase 1b) is to help researchers learn more about how the investigational medicine, mavorixafor, impacts people living with chronic neutropenia (including congenital, idiopathic, and cyclic). In Part 2 (Phase 2), the safety and tolerability of chronic dosing of mavorixafor will be evaluated in a larger participant population and the impact of 6-month chronic dosing of mavorixafor on participant neutropenia.

Start Date16 Oct 2021

Sponsor / Collaborator

X4 Pharmaceuticals, Inc.

100 Clinical Results associated with Mavorixafor

100 Translational Medicine associated with Mavorixafor

100 Patents (Medical) associated with Mavorixafor

Literatures (Medical) associated with Mavorixafor

01 Jan 2025·Bioorganic & Medicinal Chemistry Letters

Grafting a chromophore on AMD070 analogues for CXCR4 bioimaging: Chemical synthesis and in vitro assessment of the inhibition properties of the CXCR4 receptor

Article

Author: Faivre-Chauvet, Alain ; Bouhsine, Hela ; Tripier, Raphaël ; Saëc, Patricia Le ; Métivier, Cassandra ; Rbah-Vidal, Latifa ; Troadec, Thibault ; Le Roy, Marie M ; Le Roy, Marie M. ; Chérel, Michel

Thank to their particular pharmacokinetics, the use of small organic molecules can be a very promising alternative to macromolecular targeting biomolecules (i.e. antibodies, peptides…) for specific imaging of tumours. Herein, the potential of two AMD070-like inhibitors as CXCR4-targeting units for specific imaging of cancer cells, and the influence of chromophore-grafting on their recognition properties was investigated.

01 Dec 2024·Cellular and Molecular Life Sciences

Crosstalk between purinergic receptor P2Y11 and chemokine receptor CXCR7 is regulated by CXCR4 in human macrophages

Article

Author: Klaver, Dominik ; Thurnher, Martin ; Frena, Beatrice ; Amato, Marco ; Gander, Hubert

AbstractP2Y11 is a G protein-coupled ATP receptor that activates IL-1 receptor (IL-1R) in a cyclic AMP dependent manner. In human macrophages, P2Y11/IL-1R crosstalk with CCL20 as a prime target is controlled by phosphodiesterase 4 (PDE4), which mediates breakdown of cyclic AMP. Here, we used gene expression analysis to identify activation of CXCR4 and CXCR7 as a hallmark of P2Y11 signaling. We found that PDE4 inhibition with rolipram boosts P2Y11/IL-1R-induced upregulation of CXCR7 expression and CCL20 production in an epidermal growth factor receptor dependent manner. Using an astrocytoma cell line, naturally expressing CXCR7 but lacking CXCR4, P2Y11/IL-1R activation effectively induced and CXCR7 agonist TC14012 enhanced CCL20 production even in the absence of PDE4 inhibition. Moreover, CXCR7 depletion by RNA interference suppressed CCL20 production. In macrophages, the simultaneous activation of P2Y11 and CXCR7 by their respective agonists was sufficient to induce CCL20 production with no need of PDE4 inhibition, as CXCR7 activation increased its own and eliminated CXCR4 expression. Finally, analysis of multiple CCL chemokines in the macrophage secretome revealed that CXCR4 inactivation and CXCR7 activation selectively enhanced P2Y11/IL-1R-mediated secretion of CCL20. Altogether, our data establish CXCR7 as an integral component of the P2Y11/IL-1R-initiated signaling cascade and CXCR4-associated PDE4 as a regulatory checkpoint.

01 Dec 2024·European Journal of Medicinal Chemistry

Comprehensive structural investigation of a potent and selective CXCR4 antagonist via crosslink modification

Article

Author: Scala, Stefania ; Del Bene, Alessandra ; Natale, Benito ; Tomassi, Stefano ; Cutolo, Roberto ; Di Maiolo, Gaetana ; Altucci, Lucia ; Vetrei, Cinzia ; Messere, Anna ; Merlino, Francesco ; Rea, Giuseppina ; Benedetti, Rosaria ; Di Maro, Salvatore ; Cosconati, Sandro ; D'Alterio, Crescenzo ; D'Aniello, Antonia ; D’Aniello, Antonia ; Campagna, Erica ; Mottola, Salvatore ; Mazzarella, Vincenzo ; Trotta, Anna Maria ; Santagata, Sara ; D’Alterio, Crescenzo ; Roggia, Michele

Macrocyclization presents a valuable strategy for enhancing the pharmacokinetic and pharmacodynamic profiles of short bioactive peptides. The exploration of various macrocyclic characteristics, such as crosslinking tethers, ring size, and orientation, is generally conducted during the early stages of development. Herein, starting from a potent and selective C-X-C chemokine receptor 4 (CXCR4) cyclic heptapeptide antagonist mimicking the N-terminal region of CXCL12, we demonstrated that the disulfide bridge could be successfully replaced with a side-chain to side-chain lactam bond, which is commonly not enlisted among the conventional disulfide mimetics. An extensive investigation was carried out to explore the chemical space of the resulting peptides, including macrocyclization width, stereochemical configuration, and lactam orientation, all of which were correlated with biochemical activity. We identified a novel heptapeptide that fully replicates the pharmacological profile of the parent peptide on CXCR4, including its potency, selectivity, and antagonistic activity, while demonstrating enhanced stability in a reductive environment. At this stage, computational studies were instructed to shed light on how the lactam cyclization features influenced the overall structure of 21 and, in turn, its ability to interact with the receptor. We envisage that these findings can give new momentum to the use of lactam cyclization as a disulfide bond mimetic and contribute to the enhancement of the repertoire for peptide-based drug development, thereby paving the way for novel avenues in therapeutic innovation.

122

News (Medical) associated with Mavorixafor

18 Mar 2025

·www.globenewswire.com

CAMP4 Appoints Multiple Industry Veterans to its Board of Directors

With decades of experience in pharmaceutical development and extensive genetic medicine expertise, Doug E. Williams, Ph.D., and Murray Stewart, DM FRCP, will provide strategic guidance for CAMP4’s multiple drug development efforts CAMBRIDGE, Mass., March 18, 2025 (GLOBE NEWSWIRE) -- CAMP4 Therapeutics Corporation (“CAMP4”) (Nasdaq: CAMP), a clinical-stage biotechnology company developing a pipeline of regRNA-targeting antisense oligonucleotide (ASO) therapies to upregulate gene expression to restore healthy protein levels, today announced the appointments of Doug E. Williams, Ph.D., and Murray Stewart, DM FRCP, to the Company’s Board of Directors. “We are delighted to welcome Drs. Williams and Stewart to our Board of Directors,” said Josh Mandel-Brehm, Chief Executive Officer of CAMP4. “With their combined expertise spanning critical aspects of R&D and clinical development, we will benefit greatly from their strategic insights as we advance our lead clinical program for urea cycle disorders and our preclinical program for SYNGAP1-related disorders, while continuing to develop additional RNA-targeting medicines designed to upregulate gene expression and meaningfully address diseases where we believe modest increases in protein levels can have a tremendous impact on the lives of patients.” Dr. Williams commented, “CAMP4’s approach is opening exciting possibilities to upregulate gene expression, a long-sought-after and challenging feat in the field of genetic medicine. I look forward to working with the team to unlock the full potential of this approach and ultimately advance novel therapeutics to the clinic.” Dr. Stewart added, “I’m thrilled to join the CAMP4 team and support its mission of advancing programmable RNA therapeutics for genetic diseases where decreased protein expression drives disease pathophysiology. By pioneering new ways to restore gene expression, CAMP4 has the potential to bring life-changing therapies to patients who currently have limited or no disease-modifying treatment options.” With over 30 years in the biopharma industry, Dr. Williams has contributed to the development of several transformative drugs, including LEUKINE®, ENBREL®, ADCETRIS®, TECFIDERA®, APROLIX®, ELOCTATE®, and SPINRAZA®. He was previously President of R&D at Sana Biotechnology and the Founding President & CEO of Codiak BioSciences. Prior to Sana, Dr. Williams served as EVP of R&D at Biogen, and earlier in his career was CEO of ZymoGenetics (acquired by BMS), and held leadership roles at Seattle Genetics, Amgen, and Immunex. Over the course of his career, he has served on the Board of Directors and Advisory Boards of more than two dozen biotech companies. Dr. Stewart currently serves as a Senior Medical Advisor to several biopharma companies, and was most recently the Chief Medical Officer at Rhythm Pharmaceuticals. Prior to this position, he served as Head of R&D at Novelion Therapeutics. Before joining Novelion, he served as corporate Chief Medical Officer at GlaxoSmithKline. With extensive clinical development experience, he has led studies across all stages, from first-in-human trials to large Phase 4 cardiovascular outcome studies. Dr. Stewart has played a key role in the successful registration and launch of multiple therapies, including AVANDIA®, ARIXTRA®, TANZEUM®, and XOLREMDI®. Before transitioning to industry, he served as a Consultant Physician and Head of Clinical Services at the Diabetes Centre in Newcastle upon Tyne, UK, where his research was focused on lipid metabolism and type 2 diabetes. About CAMP4 Therapeutics CAMP4 is developing disease-modifying treatments for a broad range of rare and prevalent genetic diseases where increasing healthy protein levels may offer meaningful therapeutic benefits. Our approach allows for targeted gene upregulation by harnessing a fundamental mechanism of how genes are controlled. To increase gene expression, our therapeutic ASO drug candidates target regRNAs, which act locally on transcription factors and are the master regulators of gene expression. CAMP4’s proprietary RAP Platform™ enables the mapping of regRNAs and generation of therapeutic candidates designed to target the regRNAs associated with genes underlying haploinsufficient and recessive partial loss-of-function disorders, of which there are more than 1,200, in which a modest increase in protein expression may have the potential to be clinically meaningful. Learn more about us at www.CAMP4tx.com and follow us on LinkedIn and X. Forward-Looking Statements This press release contains forward-looking statements which involve risks, uncertainties and contingencies, many of which are beyond the control of the Company, which may cause actual results, performance, or achievements to differ materially from anticipated results, performance, or achievements. All statements other than statements of historical facts contained in this press release are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements include, but are not limited to, statements concerning CAMP4’s plans, objectives, expectations and intentions. The forward-looking statements in this press release speak only as of the date of this press release and are subject to a number of known and unknown risks, uncertainties and assumptions that could cause the Company’s actual results to differ materially from those anticipated in the forward-looking statements, including, but not limited to: the Company’s limited operating history, incurrence of substantial losses since the Company’s inception and anticipation of incurring substantial and increasing losses for the foreseeable future; the Company’s need for substantial additional financing to achieve the Company’s goals; the uncertainty of clinical development, which is lengthy and expensive, and characterized by uncertain outcomes, and risks related to additional costs or delays in completing, or failing to complete, the development and commercialization of the Company’s current product candidates or any future product candidates; delays or difficulties in the enrollment and dosing of patients in clinical trials; the impact of any significant adverse events or undesirable side effects caused by the Company’s product candidates; potential competition, including from large and specialty pharmaceutical and biotechnology companies; the Company’s ability to realize the benefits of the Company’s current or future collaborations or licensing arrangements and ability to successfully consummate future partnerships; the Company’s ability to obtain regulatory approval to commercialize any product candidate in the United States or any other jurisdiction, and the risk that any such approval may be for a more narrow indication than the Company seeks; the Company’s dependence on the services of the Company’s senior management and other clinical and scientific personnel, and the Company’s ability to retain these individuals or recruit additional management or clinical and scientific personnel; the Company’s ability to grow the Company’s organization, and manage the Company’s growth and expansion of the Company’s operations; risks related to the manufacturing of the Company’s product candidates, which is complex, and the risk that the Company’s third-party manufacturers may encounter difficulties in production; the Company’s ability to obtain and maintain sufficient intellectual property protection for the Company’s product candidates or any future product candidates the Company may develop; the Company’s reliance on third parties to conduct the Company’s preclinical studies and clinical trials; the Company’s compliance with the Company’s obligations under the licenses granted to the Company by others, for the rights to develop and commercialize the Company’s product candidates; risks related to the operations of the Company’s suppliers; and other risks and uncertainties described in the section “Risk Factors” in the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as well as other information we file with the Securities and Exchange Commission. The forward-looking statements in this press release are inherently uncertain and are not guarantees of future events. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond the Company’s control, you should not unduly rely on these forward-looking statements. The events and circumstances reflected in the forward-looking statements may not be achieved or occur and actual future results, levels of activity, performance and events and circumstances could differ materially from those projected in the forward-looking statements. Moreover, the Company operates in an evolving environment. New risks and uncertainties may emerge from time to time, and management cannot predict all risks and uncertainties. Investors, potential investors, and others should give careful consideration to these risks and uncertainties. Except as required by applicable law, the Company does not undertake to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Contacts Investor Relations:Sandya von der WeidLifeSci Advisorssvonderweid@lifesciadvisors.com Media:Jason Braco, Ph.D.LifeSci Communicationsjbraco@lifescicomms.com

Executive ChangeOligonucleotide

28 Feb 2025

·www.biospace.com

6 Rare Diseases That Have New FDA-Approved Treatments

iStockphoto/ Olivier Le Moal ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, Niemann-Pick disease type C, metachromatic leukodystrophy and more. Rare Disease Day, which falls at the end of February each year, is an opportunity to take stock of advances in this space, as well as the many remaining gaps in care and treatment. Defined as diseases that affect fewer than 200,000 people in the U.S., rare diseases collectively afflict about 350 million people worldwide, according to the National Organization for Rare Disorders . An estimated 7,000 of these conditions have been identified, and more than 90% of them have no FDA-approved treatment. Yet progress toward developing such treatments continues, particularly among smaller companies. Here are five rare diseases that have seen drug approvals since last year’s Rare Disease Day. Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a relatively common rare disease, affecting about one out of every 3,600 baby boys. This X-linked genetic disease affects the dystrophin protein, a component of muscle, and causes progressive weakening of skeletal and heart muscle. Families affected by DMD saw a spate of treatment approvals in 2023 and 2024, including Sarepta Therapeutics’ Elevidys and Catalyst Pharmaceuticals’ Agamree. The most recent was Duvyzat (givinostat), a small molecule therapy marketed by ITF Therapeutics. It targets histone deacetylases, enzymes that modulate gene and protein expression in the muscle. “Deregulation of HDACs is a major consequence of the lack of dystrophin associated with DMD,” Matt Trudeau, head of ITF, told BioSpace in an email ahead of the FDA decision in March 2024. “We believe givinostat’s mode of action has the potential to inhibit HDAC pathological overactivity and thereby impact the cascade of events leading to muscle damage which, in turn, may counteract disease pathology and slow muscle deterioration.” In the Phase III Epidys trial , givinostat met the primary endpoint of a change in the four-stair climb assessment from baseline to 72 weeks. Italfarmaco, the parent company of ITF which developed the drug, also reported that it showed “favorable outcomes” in key secondary measures, including on the North Star Ambulatory Assessment (NSAA), a scale that measures motor function skills. On the NSAA, patients treated with the new drug saw less worsening compared to placebo after 18 months. Givinostat was generally well tolerated, with no treatment-related severe or serious adverse events. Metachromatic Leukodystrophy Metachromatic leukodystrophy (MLD) is a metabolic disease that affects one in every 100,000 live births. It is caused by a mutation in the arylsulfatase-A ( ARSA ) gene, which codes for an enzyme that breaks down sulfatides, and is characterized by a pathological build-up of sulfatides in the brain, liver, kidneys, spleen and other body parts. Over time this buildup can lead to organ damage and neurological issues, and patients often experience motor, behavioral and cognitive problems, as well as seizures. Most patients die within five years of disease onset. The FDA approved the first therapy for early juvenile or early symptomatic forms of the disease, Lenmeldy (atidarsagene autotemcel), in March 2024. The gene therapy, marketed by Orchard Therapeutics, uses a patient’s own stem cells to deliver a functioning copy of the ARSA gene. Lenmeldy was also approved by the European Union in December 2020 , where it is marketed as Libmeldy. The FDA’s approval of Lenmeldy is backed by data from two single-arm, open-label trials enrolling a total of 37 pediatric MLD patients. Over a median follow-up of 6.76 years, Lenmeldy led to significantly better overall survival compared with natural history data. The gene therapy also preserved motor and cognitive function in late-infantile MLD patients. Lenmeldy’s most common adverse events were febrile neutropenia, stomatitis, respiratory tract infections and rashes. WHIM Syndrome Fewer than 1,000 people in the U.S. have been diagnosed with WHIM syndrome, according to the NIH. The condition’s name is an acronym of the four characteristics of the disease: warts, hypogammaglobulinemia (low antibody levels), infections and myelokathexis (a white blood cell disorder). The syndrome is caused by mutations in the chemokine receptor ( CXCR4 ) gene that increase the activity level of the resulting protein. In April 2024, the FDA approved X4 Pharmaceuticals’ Xolremdi (mavorixafor) as the first targeted treatment for WHIM. Xolremdi is an oral CXCR4 antagonist that draws white blood cells from the bone marrow into the blood to improve immune deficiencies. It is approved for patients 12 and older. Phase III results from the 4WHIM trial of Xolremdi showed a 60% reduction in annualized infection rate compared to placebo; trial participants had less than one infection per year compared with 4.5 for the placebo group. Teresa Tarrant, an associate professor at Duke University’s School of Medicine and lead investigator of the 4WHIM trial, told BioSpace last year that previous treatments for the disease were only supportive. “I think that for a lot of patients, the idea that there is a targeted therapy . . . something that is directly correcting the underlying problem for the individual, I think that gives them a lot of hope, a lot of optimism,” Tarrant said. “I think WHIM is going to be a poster child for rare diseases and the ability where we’re at now in modern medicine to design therapies to treat underlying genetic disorders.” Niemann-Pick Disease Type C Afflicting approximately one in every 150,000 people , Niemann-Pick disease type C (NPC) is an ultrarare lysosomal storage disease that progressively impairs patients’ physical and cognitive function. It is caused by mutations in the NPC1 and NPC2 genes that hinder the body’s ability to clear cholesterol and other lipids from inside cells, leading to their pathologic build-up in the brain and other crucial organs. NPC affects patients’ speech, motor and swallowing functions. According to the FDA , patients with NPC typically live only up to around 13 years of age. September 2024 saw not one, but two approvals of therapies for NPC. The first, Zevra Therapeutics’ Miplyffa (arimoclomol), is an oral drug approved for patients 2 years and older that is intended to be used alongside miglustat , a small molecule approved to treat the neurological manifestations of NPC. According to the company , Miplyffa activates transcriptional factors, resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. In a clinical trial, Zevra found that Miplyffa plus miglustat stopped disease progression during 12 months of treatment, decreasing patients’ scores on an assessment of NPC symptom severity. Meanwhile, patients treated with miglustat alone experienced a progression in symptoms. The second approval was for IntraBio’s Aqneursa (levacetylleucine) in adults and children weighing at least 15 kg. According to the company, it is not clear what Aqneursa’s target is, but it may activate cerebral glucose metabolism in the cerebellum and thus boost cerebellar activity. A randomized, double-blinded and placebo-controlled crossover study found that during the 12-week period that patients were treated with Aqneursa, they showed better functional performance as measured by a score that assesses their gait, sitting, stance and speech. The drug was well-tolerated, with common side effects including vomiting, abdominal pain, difficulty swallowing and upper respiratory tract infections. The drug’s label includes a warning for embryo-fetal harm if used during pregnancy. The back-to-back NPC approvals highlighted the FDA’s commitment to supporting the development of innovative therapies for rare diseases, Janet Maynard, director of the agency’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, said in a statement . Cerebrotendinous Xanthomatosis Cerebrotendinous xanthomatosis (CTX) is a very rare metabolic disease, affecting just one in a million people globally . It is characterized by the inability to break down certain types of cholesterol and other lipids, leading to their toxic accumulation throughout the body, particularly in the brain and connective tissues. Patients with CTX suffer from neurological abnormalities in early adulthood, including seizures and problems with movement and speech. Just last week, CTX got its first approved drug with the FDA’s greenlight of Mirum Pharmaceuticals’ oral therapy Ctexli (chenodiol). CTX is caused by mutations in the CYP27A1 gene, which codes for an enzyme that breaks down cholesterol into bile acids, and Ctexli replenishes the body’s levels of bile acids. This reduces the build-up of the toxic metabolites that cause the disease. Mirum’s application for approval of the drug included data from the Phase III RESTORE study, a June 2024 readout of which showed the drug could lower bile alcohols “with high statistical significance,” the biotech said at the time. Ctexli’s benefit was 20-fold greater in magnitude than that of placebo. The most common adverse events associated with Ctexli include hypertension, muscle weakness and diarrhea, and its label includes precautions about potential liver toxicity.

Clinical ResultPhase 3Drug Approval

20 Feb 2025

·www.pharmaceutical-technology.com

X4 and taiba to distribute WHIM syndrome therapy in Middle East

A pivotal Phase III trial is being conducted by the company to investigate the therapy. Credit: shisu_ka via Shutterstock. US-based X4 Pharmaceuticals has entered an exclusive agreement with United Arab Emirates (UAE)-based pharmaceutical products wholesaler taiba rare to distribute and commercialise oral Xolremdi (mavorixafor) in select Middle Eastern countries for the treatment of warts, hypogammaglobulinemia, infections and myelokathexis (WHIM) syndrome. The agreement covers Bahrain, Egypt, Kuwait, Oman, Qatar, Saudi Arabia and the UAE, and is contingent upon receiving regulatory approvals in these regions. taiba, collaborating with X4 on strategic decisions, is set to manage the distribution, promotion, marketing and sales of the therapy in the Middle East. The therapy’s local regulatory filings will be based on X4’s US Food and Drug Administration (FDA) registration dossier. Once regulatory approvals are obtained, taiba will make the therapy available to WHIM patients via a named-patient programme, which allows for the use of medicines approved in other nations for local subjects who lack alternative treatment alternatives. X4 Pharmaceuticals CEO and president Paula Ragan stated: “We are very pleased to collaborate with taiba rare, an ideal partner for X4 due to their dedication to and experience providing access to innovative rare disease treatments throughout the region. “We look forward to working together to further expand the global reach of Xolremdi for people with WHIM syndrome.” Xolremdi is a selective CXC chemokine receptor 4 (CXCR4) antagonist and gained the US Food and Drug Administration (FDA) approval in April 2024 for individuals aged 12 and above with WHIM syndrome. It is the only treatment specifically approved for WHIM syndrome in the US. The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use is currently reviewing the marketing authorisation application for mavorixafor for this condition. X4 is exploring further applications in addition to WHIM. A pivotal Phase III trial is being conducted by the company to investigate the therapy for the treatment of specific chronic neutropenic disorders.

Phase 3Drug ApprovalLicense out/in

100 Deals associated with Mavorixafor

External Link

KEGG	Wiki	ATC	Drug Bank
D11510	-	-	DB05501

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
WHIM Syndrome	United States	X4 Pharmaceuticals, Inc.	26 Apr 2024

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
WHIM Syndrome	NDA/BLA	Europe	X4 Pharmaceuticals, Inc.	-
Neutropenia	Phase 3	Portugal	X4 Pharmaceuticals, Inc.	06 Jun 2024
Neutropenia	Phase 3	United States	X4 Pharmaceuticals, Inc.	06 Jun 2024
Neutropenia	Phase 3	Germany	X4 Pharmaceuticals, Inc.	06 Jun 2024
Neutropenia	Phase 3	France	X4 Pharmaceuticals, Inc.	06 Jun 2024
Neutropenia	Phase 3	Italy	X4 Pharmaceuticals, Inc.	06 Jun 2024
Neutropenia	Phase 3	Argentina	X4 Pharmaceuticals, Inc.	06 Jun 2024
Neutropenia	Phase 3	United Kingdom	X4 Pharmaceuticals, Inc.	06 Jun 2024
Neutropenia	Phase 3	Serbia	X4 Pharmaceuticals, Inc.	06 Jun 2024
Neutropenia	Phase 3	Greece	X4 Pharmaceuticals, Inc.	06 Jun 2024

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04154488 (GlobeNewswire) Manual	Phase 2	Neutropenia	23	Mavorixafor monotherapy	(mnloyrrlit) = rswnxjbmrt ohobhdumhe (odbnmdsovq )	Positive	13 Nov 2024
				Mavorixafor in combination with injectable G-CSF	(mnloyrrlit) = lckdxvgfim ohobhdumhe (odbnmdsovq )
NCT03995108 (phase 3, Pubmed \| Blood)	Phase 3	WHIM Syndrome CXCR4 gain-of-function variants	31	Mavorixafor	wwubgitovy(phovyawxte) = kaqtwmkhus mufdybvuau (onqsrmrywb ) View more	Positive	04 Jul 2024
				Placebo	wwubgitovy(phovyawxte) = ybihxqpogg mufdybvuau (onqsrmrywb ) View more
NCT04154488 (Biospace) Manual	Phase 2	Neutropenia	23	Mavorixafor monotherapy	(omfrbrxroi) = djnnmzeikm srexqekiyr (hkiwpegfmy ) View more	Positive	27 Jun 2024
				Mavorixafor with stable-dose G-CSF	(mgkkzfucab) = rrdgtmjxqh sqpoiyajrq (kcqmxqndkt )
NCT03995108 (FDA_CDER) Manual	Phase 3	WHIM Syndrome	31	XOLREMDI	(tftmikibpb) = kqdvwsjtkd ypdssrtkxm (jtpgveqtri, 1.89) View more	Positive	26 Apr 2024
NCT03995108 (Phase 3 Trial of an Oral CXCR4 Antagonist, AAAAI2024)	Phase 3	WHIM Syndrome	-	Mavorixafor	fycedqysmz(lbbprztjhc) = bpjurcnhyb vfjacbaxrw (mfwkttrass ) View more	Positive	23 Feb 2024
				Placebo	fycedqysmz(lbbprztjhc) = vcgkghqtww vfjacbaxrw (mfwkttrass ) View more
NCT03995108 (EHA2023)	Phase 3	WHIM Syndrome	31	Mavorixafor	(qvyarcxfcy) = increased from baseline into normal range and sustained at each timepoint assessed over 52 weeks with mavorixafor versus placebo utoxqdgahg (ntogfqvsfx ) View more	Positive	08 Jun 2023
				Placebo
NCT02923531 (CTgov)	Phase 1/2	Renal Cell Carcinoma	9	nivolumab+X4P-001	zhsygoitzb(ipftxyybsi) = jzaxzftrbc vvnatdxxnu (cqbcedhlzw, yazynnosct - iqcwqvuvmn) View more	-	29 Dec 2022
NCT03995108 (4WHIM, EHA2022) Manual	Phase 3	WHIM Syndrome	31	MAVORIXAFOR	(iecqunqjdw) = cowhvgncxq hubmyjllcc (yajkporwqd ) View more	Positive	10 Jun 2022
				Placebo	(iecqunqjdw) = yjxmewjtxv hubmyjllcc (yajkporwqd ) View more

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