Organization (Industry)
X4 Pharmaceuticals, Inc
ClinicalTrials.gov lists X4 Pharmaceuticals, Inc as lead sponsor of 12 trials. It has 11 active development programs and 7 approved assets.
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Trials started per year with this organization as sponsor or collaborator, 1990 onward. The series also counts trials where the organization is a collaborator; the lead-sponsor trial count does not.
2026 is partial: 0 registered with a 2026 start date. The dashed outline is the pace from the 0 registered with a start date through September, planned starts included, not adjusted; not a forecast. No forecast is made for this organization: it averages 0.8 trial starts a year over the five years to September, under the 5 a year the method needs.
This page covers X4 Pharmaceuticals, Inc: 12 lead-sponsored trials and 2 legal-entity registrations.
Disease areas (19)
Shown in this order: most trials first.
| Disease area | Trials |
|---|---|
| Neutropenia | 3 |
| Carcinoma, Renal Cell | 2 |
| Kidney Neoplasms | 2 |
| Agammaglobulinemia | 1 |
| Barth Syndrome | 1 |
Showing 5 of 19 disease areas.
Trials by start year as sponsor or collaborator (7)
Shown in this order: most recent first. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Show the table of trials by start year as sponsor or collaborator
| Year | Trials started |
|---|---|
| 2025 | 2 |
| 2024 | 1 |
| 2021 | 1 |
| 2020 | 1 |
| 2019 | 1 |
| 2018 | 1 |
| 2016 | 6 |
Recruitment duration, a model prediction: 1 trial still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
Active development programs (11)
Shown in this order: max phase, then active trials.
| Drug | Disease area | Max phase | Trials |
|---|---|---|---|
| X4P-001 | Neutropenia | Phase 3 | 2 |
| X4P-001 | Heart Defects, Congenital | Phase 3 | 1 |
| X4P-001 | Shwachman-Diamond Syndrome | Phase 3 | 1 |
| X4P-001 | Exocrine Pancreatic Insufficiency | Phase 3 | 1 |
| X4P-001 | Barth Syndrome | Phase 3 | 1 |
Showing 5 of 11 active programs.
Approved assets (7)
Shown in this order: max phase first.
| Drug | Trials |
|---|---|
| X4P-001 | 11 |
| AXITINIB | 1 |
| NIVOLUMAB | 1 |
| Efavirenz | 1 |
| Carbamazepine | 1 |
Showing 5 of 7 approved assets.
Sponsor delivery record (1)
Enrollment hit target: the share of this sponsor's lead-sponsored trials whose actual enrollment reached the planned enrollment. On time: the share whose actual primary completion date was no later than the planned date. Each share counts only trials where we observed an estimated figure before the actual one, and n is that number of trials. Below 5 trials the count is shown as a fraction, not a percentage.
| Enrollment | Timeline |
|---|---|
| 27% hit target (n=11) | 40% on time (n=10) |
Journals published in (3)
Shown in this order: most published in first.
| Journal | Publications |
|---|---|
| Blood | 2 |
| Cancer research communications | 1 |
| Investigational new drugs | 1 |
US granted patents (1)
Patents are not classified by disease area in the source, so this is the organization's overall portfolio, not a split by disease.
| US patents granted | Granted in the last 5 years | Most recent grant |
|---|---|---|
| 19 | 12 | 2025-08-05 |
SEC-reported annual financials (11)
Shown in this order: newest fiscal year first; SEC EDGAR XBRL companyfacts, USD as reported.
| Fiscal year | Revenue (USD) | Net income (USD) | R&D (USD) |
|---|---|---|---|
| 2025 | 35,113,000 | -79,199,000 | 72,723,000 |
| 2024 | 2,557,000 | -37,450,000 | 81,643,000 |
| 2023 | 0 | -101,167,000 | 72,017,000 |
| 2022 | 0 | -93,867,000 | 61,058,000 |
| 2021 | 0 | -88,696,000 | 50,647,000 |
| 2020 | 3,000,000 | -62,131,000 | 41,932,000 |
| 2019 | 0 | -52,807,000 | 30,163,000 |
| 2018 | 0 | -33,285,000 | 20,346,000 |
| 2017 | not reported | -21,994,000 | 17,066,000 |
| 2016 | not reported | -22,975,000 | 17,831,000 |
Showing 10 of 11 fiscal years.
Financing and grant events (48)
Shown in this order: most recent first.
| Date | Round type | Amount (USD) | Source |
|---|---|---|---|
| 2025-10-24 | Other financing | not disclosed | Edgar offerings |
| 2025-10-23 | Other financing | not disclosed | Edgar offerings |
| 2025-10-23 | Public offering | not disclosed | Sec extracted |
| 2025-09-18 | Other financing | not disclosed | Edgar offerings |
| 2025-08-13 | Private placement | 84,999,985 | Form d |
Showing 5 of 48 events.
Climate targets, as filed (1)
| Reporting year | Net-zero target |
|---|---|
| 2025 | not disclosed |
Workforce and board diversity, as filed (1)
| Reporting year | Workforce % women | Board % women |
|---|---|---|
| 2025 | not disclosed | not disclosed |
Organization relationships (1)
| Related organization | Relationship | Evidence |
|---|---|---|
| Covance (United States) | Uses vendor | 1 |
Latest dated events (82)
Shown in this order: newest first.
| Date | Event | Source |
|---|---|---|
| 2025-10-24 | Financing | Funding rounds and grants (NIH RePORTER + SEC EDGAR) |
| 2025-10-23 | Financing | Funding rounds and grants (NIH RePORTER + SEC EDGAR) |
| 2025-10-23 | Public offering | Funding rounds and grants (NIH RePORTER + SEC EDGAR) |
| 2025-09-18 | Financing | Funding rounds and grants (NIH RePORTER + SEC EDGAR) |
| 2025-08-13 | Private placement | Funding rounds and grants (NIH RePORTER + SEC EDGAR) |
Showing 5 of 82 events.
Biomarker capability evidence (65)
Shown in this order: tier desc, then gene symbol.
| Gene | Role | Evidence |
|---|---|---|
| CSF2 | Trial sponsor | Named in trial inclusion criteria |
| CSF2 | Trial sponsor | Named in trial inclusion criteria |
| CSF3 | Trial sponsor | Named in trial inclusion criteria |
| CSF3R | Trial sponsor | Named in trial inclusion criteria |
| CXCR2 | Trial sponsor | Named in trial inclusion criteria |
Showing 5 of 65 evidence rows.
Not on this page
Checked, with nothing recorded for this organization: companies grouped under this page, related companies not grouped here, fda warning letters, fda untitled letters, fda inspection record, fda recalls, fda import refusals, manufacturing and distribution sites, clia clinical laboratory certificates, and biomarkers this organization publishes on.