- |||||||||| RGT-M001 / Rgenta Therap
RGT-M001, a first-in-class small molecule mRNA degrader of the oncogenic transcription factor c-Myb, demonstrated remarkable single agent anti-tumor efficacy in cancer patient-derived xenograft model (Section 27) - Mar 5, 2024 - Abstract #AACR2024AACR_5460; As a single agent, RGT-M001 reduced in vivo C-MYB transcript levels by >80% at peak drug exposure and induced a remarkable tumor growth inhibition response (~70% TGI) in the ACCX11 PDx mouse model, surpassing the therapeutic benchmark Lenvatinib (40% TGI)...Finally, we showed that the combination of RGT-M001 with the Notch Inhibitor AL-101 resulted in complete inhibition of tumor growth.In conclusion, these data demonstrate that small molecules targeting RNA are a safe and effective approach to address previously undruggable protein targets. Down-regulation of C-MYB by our RNA-targeting small molecules is an attractive therapeutic strategy to treat ACC and other cancers driven by C-MYB dysregulations.
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Trial completion date, Trial primary completion date, Surgery: AL101 Before Surgery for the Treatment of Notch Activated Adenoid Cystic Cancer (clinicaltrials.gov) - Apr 7, 2023 P1, N=12, Recruiting, ORR: CR + PR; DCR: CR + PR + SD Conclusions AL101 showed acceptable safety and tolerability in ACC pts with Notchmut and a response rate comparable to pooled estimates for available therapies in pts regardless of Notch status. Trial completion date: Apr 2023 --> Dec 2023 | Trial primary completion date: Apr 2023 --> Dec 2023
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Journal, Heterogeneity, BRCA Biomarker: Genetic heterogeneity and therapeutic target detection through microdissection in solid-type adenoid cystic carcinoma. (Pubmed Central) - Aug 24, 2022 Guided by the genetic profiles, the preclinical efficiency of the gamma-secretase inhibitor BMS-906024 was evaluated in patient derived xenograft models (PDXs) with activating NOTCH1 mutations and demonstrated robust antitumour effects...In contrast to cribriform/tubular ACCs, solid-type ACCs should be approached with a distinct therapeutic strategy, particularly targeting NOTCH1. Microdissecting the highest grade component guided by histology is a highly recommended tumour sampling strategy and facilitates the detection of key molecular targets.
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Initial results of phase II/III trial of AL102 for treatment of desmoid tumors (DT) (7.3.U - Urval Auditorium) - Jul 25, 2022 - Abstract #ESMO2022ESMO_1815; P2/3 Table: 1488MO Summary of Treatment-emergent Adverse Events (TEAEs) by Patient *Deemed unrelated to study drug by investigator Conclusions Safety findings to date show no indication for discontinuing any of the three dosing cohorts. Efficacy, patient reported outcome and additional safety data will be presented at the conference.
- |||||||||| AL101 / Ayala Pharma, nirogacestat (PF-03084014) / SpringWorks Therap, AL102 / Ayala Pharma, Novartis
AL102, ORAL GAMMA-SECRETASE INHIBITOR FOR THE TREATMENT OF DESMOID TUMORS ([VIRTUAL]) - Nov 26, 2021 - Abstract #CTOS2021CTOS_333; An oral GSI (PF-03084014) was effective in treating desmoid tumors with an objective response rate (ORR) of 71.4% in a Phase 1 study with 7 patients (Villalobos, 2018)...In a Phase 1 study with AL101 (formerly known BMS-906024; study CA216001), another GSI developed by Ayala, 2 participants with DT had confirmed partial response (PR) lasting either >3.5 years or >1 year; and an additional participant with extremity DT had stable disease lasting 1 year before discontinuing treatment (El-Khoueiry, 2018).RINGSIDE is a Phase 2/3, randomized study in patients with progressive DT... There are no results or conclusions to present at this time.
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Clinical, Journal: Activity of the Gamma Secretase Inhibitor AL101 in Desmoid Tumors: A Case Report of 2 Adult Cases. (Pubmed Central) - Oct 29, 2021 Also, in a phase 1 study of AL102, a potent orally administered gamma secretase inhibitor that shares structural features with AL101, a patient with a desmoid tumor was noted to have tumor shrinkage. Formal clinical testing of AL102 for the treatment of patients with desmoid tumors that are not amenable to surgery or are refractory to/recurrent from other prior therapies is currently underway.
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Journal: Perfluoroctanoic acid (PFOA) enhances NOTCH-signaling in an angiogenesis model of placental trophoblast cells. (Pubmed Central) - Jun 4, 2021 We found a PFOA-induced increase in NOTCH intracellular domain (NICD) abundance in HTR8/SVneo, indicating that PFOA enhances NOTCH-signaling in this cell type. Enhancement of NOTCH-pathway by PFOA may be a key to understand the mode of action of PFAS, as this pathway is critically involved in many confirmed physiological/toxicological symptoms associated with PFAS exposure.
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Trial completion date, Trial primary completion date: ACCURACY: A Study Of AL101In Patients With Adenoid Cystic Carcinoma (ACC) Bearing Activating Notch Mutations (clinicaltrials.gov) - May 24, 2021 P2, N=87, Recruiting, Enhancement of NOTCH-pathway by PFOA may be a key to understand the mode of action of PFAS, as this pathway is critically involved in many confirmed physiological/toxicological symptoms associated with PFAS exposure. Trial completion date: Jul 2021 --> Mar 2022 | Trial primary completion date: Apr 2021 --> Oct 2021
- |||||||||| nirogacestat (PF-03084014) / SpringWorks Therap
Journal: Precision medicine for human cancers with Notch signaling dysregulation (Review). (Pubmed Central) - Sep 13, 2020 Small‑molecule γ‑secretase inhibitors (AL101, MRK‑560, nirogacestat and others) and antibody‑based biologics targeting Notch ligands or receptors [ABT‑165, AMG 119, rovalpituzumab tesirine (Rova‑T) and others] have been developed as investigational drugs...Phase III clinical trials of Rova‑T for patients with small‑cell lung cancer and a phase III clinical trial of nirogacestat for patients with desmoid tumors are ongoing. Integration of human intelligence, cognitive computing and explainable artificial intelligence is necessary to construct a Notch‑related knowledge‑base and optimize Notch‑targeted therapy for patients with cancer.
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Biomarker, Journal: Rethinking gamma-secretase inhibitors for treatment of non-small cell lung cancer: Is Notch the target? (Pubmed Central) - Dec 24, 2019 We discuss our recently published preclinical study using the γ-secretase inhibitor AL101, formerly BMS906024, on cell lines and PDX models of NSCLC, primarily lung adenocarcinoma...Now that we have entered the post-genome/transcriptome era, this goal is easily attainable. Discovery of the biomarker(s) that predict sensitivity to ɣ-secretase inhibitors would guide selection of the responder population that is most likely to benefit and move the compounds closer to approval for therapeutic use in NSCLC.
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Inhibition of NOTCH signaing with g-secretase inhibitor AL101 contributes to overcome resistance against HER2-targeted therapy in HER2 amplified breast cancer cells (Hall 1 - Poster Session 5) - Sep 25, 2019 - Abstract #SABCS2019SABCS_694; The goal of this study is: (a) to test the efficacy of first line treatment of anti-HER2 drugs combined with AL101, and (b) to test the efficacy of the AL101 as a second line therapy for anti-HER2 resistance breast cancer cells lines. Our study demonstrates the potential utility of HER2-targeted therapy in combination with AL101 as a novel strategy for overcoming the NOTCH signaling induced survival resistance mechanism in HER2 amplified breast cancer cells, and suggests NOTCH inhibitors as potential second-line therapy for recurrent or resistant HER2 amplified breast cancer tumors.
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Clinical, Journal: Gamma secretase inhibition by BMS-906024 enhances efficacy of paclitaxel in lung adenocarcinoma. (Pubmed Central) - Aug 9, 2018 In vivo studies with cell line- and patient-derived lung adenocarcinoma xenografts confirmed enhanced antitumor activity for BMS-906024 plus paclitaxel versus either drug alone via decreased cell proliferation and increased apoptosis. These results show that BMS-906024 sensitizes NSCLC to paclitaxel, and that wildtype KRAS and BRAF status may predict better patient response to the combination therapy.
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Trial completion, Enrollment change, Combination therapy, Metastases: Study to Evaluate Safety & Tolerability of BMS-906024 in Combination With Chemotherapy & to Define DLTs & MTD of BMS-906024 in Combination With One of the Following Chemotherapy Regimens; Weekly Paclitaxel, 5FU+Irinotecan or Carboplatin+Paclitaxel in Subjects With Advanced / Metastatic Solid Tumors (clinicaltrials.gov) - Dec 19, 2017 P1, N=141, Completed, Trial primary completion date: Oct 2017 --> Jun 2018 Active, not recruiting --> Completed | N=95 --> 141 | Active, not recruiting --> Completed | N=95 --> 141 | Active, not recruiting --> Completed | N=95 --> 141
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