Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
X4P-001 · 3 trials · 3 indications
AUCANC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The absolute neutrophil count (ANC) clinically meaningful threshold was defined as ANC ≥ 600/microliter (μL).
AUCANC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The ANC clinically meaningful threshold was defined as ANC ≥ 600/μL. Data for this outcome measure are reported as an "All Visits" summary based on the mean of AUCs that is, the per-participant average of the AUCANC across the 3 visits where participant was treated with at least 300/400 mg dose. Time frame reported is based on data collection time points.
AUCALC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The absolute lymphocyte count (ALC) clinically meaningful threshold was defined as ALC ≥ 1000/μL.
AUCALC was collected over a 24-hour period above clinically meaningful thresholds for the mavorixafor-treated participants over 6 months. The ALC clinically meaningful threshold was defined as ALC ≥ 1000/μL. Data for this outcome measure are reported as an "All Visits" summary based on the mean of AUCs that is, the per-participant average of the AUCALC across the 3 visits where participant was treated with at least 300/400 mg dose. Time frame reported is based on data collection time points.
An adverse event (AE) was defined as any untoward medical occurrence that developed or worsened in severity during the conduct of a clinical study and did not necessarily have a causal relationship to the study drug. SAEs included death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as any AE that began or worsened in severity or frequency on or after the start of study drug through 10 days after the last dose of the study drug. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.
An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. Adverse events with onset after administration of the first dose of study drug up to 10 days after last dosing date were considered TEAEs. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
| Arm | Type | Description |
|---|---|---|
| X4P-001 | EXPERIMENTAL | Initial Treatment Phase: Participants will initiate treatment with mavorixafor at 50 milligrams (mg) once daily (QD) orally or a higher dose, with potential escalation based on area under the curve for absolute neutrophil count and absolute leukocyte count (AUCANC/ALC) values to a maximum total daily dose of 400 mg. Participants are expected to receive treatment for 24 weeks in the initial Treatment Period or until development of a treatment-limiting toxicity (TLT). Extension Phase: All participants will receive mavorixafor; the dose will not exceed 400 mg. In the Extension Phase, treatment may continue until mavorixafor becomes available via an alternative mechanism (for example, drug is commercially available, an expanded access program, etc.) or until the study is terminated by the sponsor. |
| X4P-001 + Pembrolizumab | EXPERIMENTAL | X4P-001 alone, then adding pembrolizumab |
| Dose Escalation (Part A): X4P-001 200 mg BID with Axitinib | EXPERIMENTAL | Participants will receive X4P-001 200 milligrams (mg) orally twice daily (BID) with axitinib at 5 mg orally BID. |
| Dose Escalation (Part A): X4P-001 400 mg QD with Axitinib | EXPERIMENTAL | Participants will receive X4P-001 400 mg orally once daily (QD) with axitinib at 5 mg orally BID. |
| Dose Escalation (Part A): X4P-001 600 mg QD with Axitinib | EXPERIMENTAL | Participants will receive X4P-001 600 mg orally QD with axitinib at 5 mg orally BID. |
| Dose Expansion (Part B): X4P-001 400 mg QD With Axitinib | EXPERIMENTAL | Participants received X4P-001 400 mg orally QD with axitinib at 5 mg orally BID. |
| Dose Escalation and Expansion (Part C): X4P-001 600 mg QD Monotherapy | EXPERIMENTAL | Participants will receive X4P-001 600 mg orally QD. |
| Name | Type | Description |
|---|---|---|
| X4P-001 | DRUG | Mavorixafor will be provided as either 25 mg or 100 mg capsules. |
| Pembrolizumab | DRUG | Pembrolizumab 2 mg/kg, administered by IV infusion every 3 weeks |
| axitinib | DRUG | Continuous, oral dosing |
Inclusion Criteria: Participants with a clinical diagnosis of WHIM syndrome must meet all of the following criteria to be eligible for study participation: 1. Be at least 18 years of age. 2. Has signed the current approved informed consent form. 3. Has a genotype-confirmed mutation of chemokine re...
Top 2 of 6 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Akebia Therapeutics, Inc. | AKBA | 1 | PHASE2 | Vadadustat |
| Rigel Pharmaceuticals, Inc. | RIGL | 1 | PHASE2 | Fostamatinib |
X4P-001 is an investigational small molecule developed by X4 Pharmaceuticals, Inc. (ticker: XFOR). It is being studied in clear cell renal cell carcinoma, melanoma, and WHIM Syndrome. X4P-001 is also known as mavorixafor. It is not yet approved and remains in clinical development.
X4P-001 targets CXCR4, a receptor. By binding to CXCR4, the drug is designed to modulate receptor activity. This mechanism is being explored in oncology and immunology indications, including clear cell renal cell carcinoma, melanoma, and WHIM Syndrome.
X4P-001 is being developed by X4 Pharmaceuticals, Inc., which trades under the ticker XFOR. The company is responsible for the clinical development of X4P-001, also known as mavorixafor, across its studied indications.
X4P-001 is in Phase 1 development. It is an investigational drug and has not been approved by the FDA. Clinical trials have been completed, including a Phase 2 trial in WHIM Syndrome, but the drug remains in clinical development.
X4P-001 has been studied in three completed clinical trials: NCT03005327, a Phase 2 dose determination and safety study in WHIM Syndrome; NCT02823405, a Phase 1 trial with pembrolizumab in advanced melanoma; and NCT02667886, a Phase 1 trial in advanced renal cell carcinoma.
Yes, X4P-001 is also known as mavorixafor. Both names refer to the same investigational small molecule developed by X4 Pharmaceuticals, Inc. (XFOR). It targets CXCR4 and is being studied in clear cell renal cell carcinoma, melanoma, and WHIM Syndrome.