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DRUG CLASS:

Apoptosis inducer

3d
KNAN2001: Study of C6 Ceramide NanoLiposome (CNL) in Patients With Relapsed/Refractory Acute Myeloid Leukemia (clinicaltrials.gov)
P1, N=15, Recruiting, Keystone Nano, Inc | Enrolling by invitation --> Recruiting | Trial completion date: Oct 2027 --> Nov 2026 | Trial primary completion date: Feb 2027 --> Sep 2026
Enrollment status • Trial completion date • Trial primary completion date
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Ceraxa (nanoliposomal ceramide)
5d
Orphan drug uttroside B impedes MASH progression and HCC development in experimental models. (PubMed, JHEP Rep)
Our discovery of Utt-B, a phytosaponin that exhibits remarkable anti-HCC potential and is a United States FDA-designated orphan drug against HCC, has gained global recognition. The present study reveals Utt-B as a propitious candidate drug against MASH and MASH-induced HCC in a high-fat diet murine model and streptozotocin-induced steatohepatitis-derived HCC animal model, respectively.
Journal • Orphan drug
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ATG7 (Autophagy Related 7) • BECN1 (Beclin 1) • MAP1A (Microtubule Associated Protein 1A)
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uttroside-B (Utt-B)
17d
Effects of moxibustion on intestinal ferroptosis, lipid peroxidation, and P53 in mice with Crohn's disease (PubMed, Zhongguo Zhen Jiu)
Compared with the inhibitor group, the serum level of IL-6, the contents of 4-HNE and MDA, and protein expression of p53 in colonic tissue in the activator group were elevated (P<0.05), while the body weight, the contents of SOD and GSH, and protein expression of GPX4 in colonic tissue were reduced (P<0.05). Moxibustion at "Tianshu" (ST25) could alleviate intestinal inflammation in CD mice, possibly by downregulating the protein expression of p53, enhancing the activity of the SLC7A11/GSH/GPX4 pathway, reducing the production of lipid peroxides, maintaining intestinal iron homeostasis, and reducing colonic ferroptosis.
Preclinical • Journal
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TP53 (Tumor protein P53) • IL6 (Interleukin 6) • TNFA (Tumor Necrosis Factor-Alpha) • GPX4 (Glutathione Peroxidase 4) • IL17A (Interleukin 17A) • SLC7A11 (Solute Carrier Family 7 Member 11) • IL1B (Interleukin 1, beta)
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RITA
28d
Study of Orellanine in Metastatic Clear-Cell or Papillary Renal Cell Carcinoma (clinicaltrials.gov)
P1/2, N=75, Recruiting, Oncorena AB | Trial completion date: Aug 2025 --> May 2027 | Trial primary completion date: Feb 2025 --> May 2027 | N=50 --> 75
Enrollment change • Trial completion date • Trial primary completion date
2ms
Multi-omics profiling of ACOX3 unveils pan-cancer clinical biomarker potential. (PubMed, Comput Biol Chem)
ACOX3 represents a dual diagnostic and prognostic biomarker with broad pan-cancer relevance, exhibiting distinct immune correlates and therapeutic potential.
Journal • Pan tumor
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CD8 (cluster of differentiation 8) • CD4 (CD4 Molecule)
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AZD6482 • TGX-221
2ms
Evaluation of BH4 Responsiveness in Our PKU Patients (clinicaltrials.gov)
P1, N=20, Active, not recruiting, Sohag University
New P1 trial
3ms
Chemoresistance mitigation of dual drug-loaded nanoparticles with doxorubicin and curcumin. (PubMed, Cytotechnology)
This study investigates the characterization and biological effects of Dual Drug-Loaded Nanoparticles on HepG2/doxorubicin (DOX) cells, focusing on the anti-cancer ability of Doxorubicin/Curcumin-Polyethylene Glycol-Polycaprolactone Nanoparticles (DOX/Cur-PEG-PCL-NPs)...These results demonstrate that DOX/Cur-PEG-PCL-NPs effectively reverse chemoresistance and suppress tumor progression through modulation of the Nrf2 pathway and apoptosis induction, offering a promising strategy for targeted liver cancer therapy. The online version contains supplementary material available at 10.1007/s10616-025-00855-y.
Journal
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KEAP1 (Kelch Like ECH Associated Protein 1) • ABCB1 (ATP Binding Cassette Subfamily B Member 1) • ABCC1 (ATP Binding Cassette Subfamily C Member 1) • HMOX1 (Heme Oxygenase 1) • BAX (BCL2-associated X protein) • CASP3 (Caspase 3) • NQO1 (NAD(P)H dehydrogenase, quinone 1)
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curcumin/doxorubicin (iMX-110)
3ms
Pan-cancer multi-omics analysis reveals IQCE as a malignant cell-restricted oncogenic biomarker driving immunosuppression and chemoresistance in cutaneous melanoma. (PubMed, Discov Oncol)
IQCE emerges as a novel oncogene-associated biomarker with significant diagnostic and prognostic utility, particularly in SKCM. It promotes an immunosuppressive TME and oncogenic signaling while being localized to malignant cells. Although associated with chemoresistance, the vulnerability of IQCE-high tumors to RITA offers a promising therapeutic strategy. IQCE represents a compelling target for precision oncology.
Journal • Pan tumor
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TP53 (Tumor protein P53)
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RITA
4ms
KNAN2001: Study of C6 Ceramide NanoLiposome (CNL) in Patients With Relapsed/Refractory Acute Myeloid Leukemia (clinicaltrials.gov)
P1, N=12, Enrolling by invitation, Keystone Nano, Inc | Not yet recruiting --> Enrolling by invitation | Trial completion date: Oct 2026 --> Oct 2027 | Trial primary completion date: Jul 2026 --> Feb 2027
Enrollment open • Trial completion date • Trial primary completion date
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Ceraxa (nanoliposomal ceramide)
4ms
XON9-A Glyco-Humanized Polyclonal Antibody Effective Against Hepatocellular Carcinoma. (PubMed, Int J Mol Sci)
Treatments for advanced HCC include multikinase inhibitors (Sorafenib or Lenvatinib), with limited response rates and serious side effects, or immunotherapy applicable to a small fraction of patients. No toxicity was observed after repeated injections of XON9 in a non-human primate. XON9 represents a promising and selective immunotherapy against refractory HCC.
Journal
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CASP8 (Caspase 8) • CASP9 (Caspase 9)
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sorafenib • Lenvima (lenvatinib)
5ms
ST-001-010: Phase 1 Trial of ST-001 nanoFenretinide in Relapsed/Refractory T-cell Non-Hodgkin Lymphoma (clinicaltrials.gov)
P1, N=46, Recruiting, SciTech Development, Inc. | Trial completion date: Nov 2025 --> May 2027 | Trial primary completion date: May 2025 --> Dec 2026
Trial completion date • Trial primary completion date
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TNFRSF8 (TNF Receptor Superfamily Member 8)
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TNFRSF8 expression
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fenretinide nanoparticle (ST-001 nanoFenretinide)
5ms
NK cell-related genes-driven novel molecular subtyping and prognostic signatures for Wilms tumor: uncovering the therapeutic potential of TGX-221 and biomarker role of HS2ST1. (PubMed, Front Oncol)
Moreover, TGX-221 represents a promising novel therapeutic candidate, while HS2ST1 serves as a potential prognostic biomarker. These findings collectively provide tools for risk stratification and targeted therapy, advancing precision oncology for WT.
Journal
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PPP3CA (Protein Phosphatase 3 Catalytic Subunit Alpha)
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TGX-221