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DRUG CLASS:

γ-secretase inhibitor

2d
New P1 trial
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Ogsiveo (nirogacestat) • midazolam hydrochloride
3d
A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery (clinicaltrials.gov)
P2, N=35, Active, not recruiting, Children's Oncology Group | Trial completion date: Dec 2025 --> Mar 2026 | Trial primary completion date: Dec 2025 --> Mar 2026
Trial completion date • Trial primary completion date
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CTNNB1 (Catenin (cadherin-associated protein), beta 1)
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Ogsiveo (nirogacestat)
1m
Implication of an exosome-based gene signature for estimating clinical outcomes of triple-negative breast cancer and assisting in individualized therapy. (PubMed, Sci Rep)
High-risk tumors owned shorter overall survival time, but were suitable for treatment with docetaxel and several small-molecule agents (MK-0752, BRD-K33199242, IC-87114, fumonisin B1, ilomastat, GW-788388, afobazole, and batimastat). Experimentally, inhibition of FAM129B effectively attenuated proliferative and aggressive phenotypes of TNBC cells. Collectively, our findings proposed the exosome-based gene signature for accurate estimation of clinical outcomes and assisting in individually tailoring therapies in TNBC as well as discovered FAM129B as a potential therapeutic target.
Clinical data • Journal • Gene Signature • IO biomarker
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CD276 (CD276 Molecule) • TNFRSF4 (TNF Receptor Superfamily Member 4) • SERPINE1 (Serpin Family E Member 1) • THY1 (Thy-1 membrane glycoprotein) • NRP1 (Neuropilin 1) • TNFSF4 (TNF Superfamily Member 4) • KRT6A (Keratin 6A) • PGK1 (Phosphoglycerate Kinase 1)
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docetaxel • MK-0752
1m
The role of YTHDF1 in the diagnosis and prognosis of lung adenocarcinoma and its inhibitor LY-411575 in the targeted intervention of lung adenocarcinoma growth. (PubMed, Transl Cancer Res)
Our study indicates that elevated expression of YTHDF1 is a predictor of poor prognosis in patients with LUAD and may serve as a potential diagnostic biomarker and therapeutic target for this disease. In addition, we identified LY-411575 as a YTHDF1 inhibitor that effectively inhibits the proliferation of LUAD cells in vivo and in vitro, highlighting its potential as a LUAD targeted therapy.
Journal
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YTHDF1 (YTH N6-Methyladenosine RNA Binding Protein 1)
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LY-411575
1m
A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery (clinicaltrials.gov)
P2, N=35, Active, not recruiting, Children's Oncology Group | Trial completion date: Sep 2025 --> Dec 2025 | Trial primary completion date: Sep 2025 --> Dec 2025
Trial completion date • Trial primary completion date
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CTNNB1 (Catenin (cadherin-associated protein), beta 1)
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Ogsiveo (nirogacestat)
2ms
A Varegacestat Hepatic Impairment Study (clinicaltrials.gov)
P1, N=44, Active, not recruiting, Immunome, Inc. | Recruiting --> Active, not recruiting
Enrollment closed
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varegacestat (AL102)
2ms
Advances in the molecular pathogenesis of lacrimal gland adenoid cystic carcinoma and associated targeted therapies (PubMed, Zhonghua Yan Ke Za Zhi)
Emerging breakthroughs in targeted therapies warrant attention, including antisense oligonucleotides targeting MYB-NFIB fusion genes, clinical trial data of NOTCH inhibitors (e.g., AL101), and PARP inhibitor-based combinatorial regimens leveraging DNA damage repair mechanisms. By integrating fundamental research and clinical translational evidence, this review provides a theoretical framework for optimizing LGACC diagnosis and treatment paradigms.
Review • Journal • BRCA Biomarker • PARP Biomarker
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BRCA (Breast cancer early onset) • NFIB (Nuclear Factor I B)
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AL101
2ms
A Phase 1 Mass Balance Study of Varegacestat (clinicaltrials.gov)
P1, N=6, Completed, Immunome, Inc. | Active, not recruiting --> Completed | Trial completion date: Jul 2025 --> Mar 2025 | Trial primary completion date: Jul 2025 --> Mar 2025
Trial completion • Trial completion date • Trial primary completion date
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varegacestat (AL102)
2ms
A Cardiac Liability Study of Varegacestat in Healthy Participants (clinicaltrials.gov)
P1, N=32, Completed, Immunome, Inc. | Active, not recruiting --> Completed
Trial completion
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varegacestat (AL102)
3ms
New P4 trial
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Ogsiveo (nirogacestat)
3ms
New P1 trial
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Ogsiveo (nirogacestat)
3ms
New P2 trial
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Ogsiveo (nirogacestat)