Drugs / WP1066

Trials 3

2019202020212022202320242025202620272028
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 12 trials
Phase 1 NCT04334863 May 2020 → Feb 2023 medulloblastoma, metastatic malignant neoplasm in the brain Emory University Completed No outcome recorded
Phase 1 NCT01904123 Jul 2018 → Mar 2022 glioblastoma, metastatic malignant neoplasm in the brain, metastatic melanoma M.D. Anderson Cancer Center Completed No outcome recorded
Phase 21 trial
Phase 2 NCT05879250 May 2024 → Dec 2027 expected IDH-wildtype glioblastoma Northwestern University Recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 16

DateIssuerRelease
2025-12-17 Moleculin Biotech, Inc. Results Moleculin Announces Positive Results from Phase 1 Clinical Trial Evaluating WP1066 for the Treatment of Pediatric Recurrent Malignant Brain Tumors moleculin.com ↗
Results demonstrated WP1066 induces anti-tumor immune responses and were recently published in the Journal of Clinical Investigation Insight
2022-04-21 Moleculin Biotech, Inc. Regulatory Moleculin Receives IND Clearance to Conduct Phase 1 Study of WP1066 for the Treatment of Recurrent Malignant Glioma moleculin.com ↗
2021-04-14 Moleculin Biotech, Inc. Regulatory Moleculin Awarded New Rare Pediatric Disease Designation from U.S. FDA for WP1066 for the Treatment of Ependymoma moleculin.com ↗
2020-12-01 Moleculin Biotech, Inc. Regulatory Moleculin Announces FDA Approves 3 Rare Pediatric Disease Designations for WP1066 moleculin.com ↗
today announced that the US Food and Drug Administration (FDA) has approved its request for a “Rare Pediatric Disease” designation for its drug candidate WP1066.
2020-10-13 Moleculin Biotech, Inc. Results Moleculin Announces Additional Positive Interim Results in Adult Glioblastoma Clinical Trial moleculin.com ↗
2020-10-01 Moleculin Biotech, Inc. Results Moleculin Announces Positive Interim Results in Pediatric Brain Tumor Phase 1 Clinical Trial at Emory University moleculin.com ↗
One of these patients with diffuse intrinsic pontine glioma (DIPG), showed an apparent response to the treatment with both clinical improvement and radiologic reduction of tumor size.
2020-07-06 WPD Pharmaceuticals Sp. z o.o. Results WPD Pharmaceuticals Licensor Announces Updates on Annamycin and WP1066 wpdpharmaceuticals.com ↗
Moleculin's STAT3 inhibitor, WP1066, used in combination with tr aditional whole brain radiation therapy resulted in long-term survivors and enhanced median survival time relative to monotherapy in mice with implanted human brain tumors.
2020-07-01 Moleculin Biotech, Inc. Results New Publication Finds Combination of WP1066 and Radiation Resulted in Long-Term Survival in Human Brain Tumor Mouse Model moleculin.com ↗
2020-02-20 WPD Pharmaceuticals Sp. z o.o. Regulatory WPD Pharmaceuticals' Stat3 Inhibitor Received FDA Approval of Ind Status in Pediatric Brain Cancer Trial wpdpharmaceuticals.com ↗
Through Moleculin Biotech, Inc. (“Moleculin”)(Nasdaq:MBRX), WPD’s license partner, the WP1066 drug candidate received FDA approval for Investigational New Drug ("IND") status to be used in a Phase 1 clinical trial for the treatment of children with recurrent or refractory malignant brain tumors.
2020-02-10 WPD Pharmaceuticals Sp. z o.o. Results WPD Pharmaceuticals’ WP1066 Drug Candidate Had Breakthrough Discovery wpdpharmaceuticals.com ↗
One of the key findings of the research is that immune -competent mice treated with both radiation and WP1066 developed an immunological memory that enabled them to prevent regrowth of the tumor after these tumor cells were reintroduced.
2019-11-12 Moleculin Biotech, Inc. Regulatory Moleculin Announces FDA Approval of IND for its STAT3 Inhibitor in Emory University Clinical Trial moleculin.com ↗
2019-02-05 Moleculin Biotech, Inc. Regulatory Moleculin Announces the FDA has Granted Orphan Drug Designation for its Brain Tumor Drug moleculin.com ↗
today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Status for its drug candidate WP1066 for the treatment of glioblastoma, the most aggressive form of brain tumor.

All press releases naming this drug 21 releases

DateIssuerRelease

Evidence & citations 5 cited values

Every value below carries the sentence it was read from. 4 sources stand behind the page.

FieldValueCited text
Known as WP1066 “WP1066” NCT01904123 ↗
3

NCT04334863 ↗

NCT01904123 ↗

NCT05879250 ↗

Action Inhibit “STAT3 inhibitor WP1066” NCT01904123 ↗
Modality Small molecule “WP1066 is an orally bioavailable, small-molecule inhibitor of activated phosphorylated STAT3 (p-STAT3)” PMID 41379553 ↗ Dec 2025
Route Oral “Patients receive STAT3 inhibitor WP1066 orally (PO)” NCT01904123 ↗
Target STAT3 “STAT3 inhibitor WP1066” NCT01904123 ↗