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Apatorsen
Last update 05 Jun 2025
Overview
Basic Info
Drug Type ASO |
Synonyms Apatorsen Sodium, ISIS-306053, OGX-427 |
Target |
Action inhibitors |
Mechanism Hsp27 inhibitors(Heat shock protein beta-1 inhibitors) |
Therapeutic Areas |
Active Indication |
Originator Organization |
Active Organization |
Inactive Organization |
License Organization- |
Drug Highest PhasePhase 2 |
First Approval Date- |
Regulation- |
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Structure/Sequence
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Sequence Code 29963446
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Related
10
Clinical Trials associated with ApatorsenNCT02423590
A Phase II Randomised, Open-label Study of Gemcitabine/Carboplatin First-line Chemotherapy in Combination With or Without the Antisense Oligonucleotide Apatorsen (OGX-427) in Advanced Squamous Cell Lung Cancers
EUCTR2014-000199-25-GB
A Phase II, randomised, open-label study of Gemcitabine/Carboplatin first-line chemotherapy in combination with or without the antisense oligonucleotide Apatorsen (OGX-427) in advanced squamous cell lung cancers - CEDAR
NCT01844817
A Randomized, Double-Blinded, Placebo-Controlled Phase II Trial Of Gemcitabine Plus Nab-Paclitaxel Combined With OGX-427 Or Placebo In Patients With Metastatic Pancreatic Cancer (The Rainier Trial)
100 Clinical Results associated with Apatorsen
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100 Translational Medicine associated with Apatorsen
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100 Patents (Medical) associated with Apatorsen
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34
Literatures (Medical) associated with Apatorsen01 Jul 2023The Journal of urology
LBA02-09 EMBARK: A Phase 3 Randomized Study of Enzalutamide or Placebo Plus Leuprolide Acetate and Enzalutamide Monotherapy in High-risk Biochemically Recurrent Prostate Cancer
Article
INTRODUCTION AND OBJECTIVE::
Within 10 years following definitive therapy for prostate cancer, ∼20–50% of patients (pts) experience biochemical recurrence (BCR) characterized by rising prostate-specific antigen (PSA) levels. Pts with high-risk BCR have an increased risk of mortality and improved therapies are needed. The objective of EMBARK was to evaluate the efficacy and safety of enzalutamide (enza) + androgen deprivation therapy (ADT) and enza monotherapy (mono) in pts with high-risk BCR.
METHODS::
EMBARK is a randomized, phase 3 study of pts with BCR considered high-risk: PSA doubling time ≤9 months and PSA ≥2 ng/mL above nadir post-radiotherapy (RT) or ≥1 ng/mL after radical prostatectomy (RP) ± postoperative RT. Pts were randomized (1:1:1) to enza 160 mg/day + leuprolide acetate (LA) (double-blind), placebo (pbo) + LA (double-blind), or enza mono (open-label). LA 22.5 mg was administered every 12 weeks. If the PSA at week 36 was <0.2 ng/mL, therapy was stopped at week 37 and restarted when PSA was ≥2 ng/mL for pts with primary RP, and ≥5 ng/mL for pts without RP. The primary endpoint, determined by blinded, independent central review (BICR), was metastasis-free survival (MFS) with enza + LA vs pbo + LA. Key secondary endpoints were MFS of enza mono vs pbo + LA, time to PSA progression, time to antineoplastic therapy, and overall survival (OS) of enza + LA or enza mono vs pbo + LA.
RESULTS::
1068 pts were randomized into the study (enza + LA, n=355; pbo + LA, n=358; enza mono, n=355). After median follow-up of 60.7 months, per BICR, MFS for enza + LA (HR 0.42; 95% CI 0.30–0.61; p<0.0001) and enza mono (HR 0.63; 95% CI 0.46–0.87; p=0.0049) were statistically superior to pbo + LA. Statistically significant improvements were also observed in risk of PSA progression (enza + LA: HR 0.07; 95% CI, 0.03–0.14; enza mono: HR 0.33; 95% CI, 0.23–0.49; both p<0.0001) and time to first use of new antineoplastic therapy (enza + LA: HR 0.36; 95% CI, 0.26–0.49; enza mono: HR 0.54; 95% CI, 0.41–0.71; both p<0.0001). Interim OS data trended in favor enza + LA (HR 0.59; 95% CI, 0.38–0.91; p=0.0153, did not cross interim efficacy boundary) and enza mono (HR 0.78; 95% CI, 0.52–1.17; p=0.2304). Fatigue and hot flash were the most common adverse events; no new safety signals were observed.
CONCLUSIONS::
In pts with high-risk BCR, enza + ADT and enza mono demonstrated a statistically significant and clinically meaningful improvement in MFS vs pbo + ADT. The safety profile of enza was consistent with results from previous clinical studies.
Clinical Trial Registration Number::
NCT02319837.
Source of Funding::
Pfizer Inc. and Astellas Pharma Inc.
Conflict of Interest and Disclosure Statement::
Neal D. Shore reports grant support and consulting fees from AbbVie, Amgen, Astellas Pharma Inc., AstraZeneca, Bayer, Clovis Oncology, Dendreon Pharmaceuticals LLC, Ferring Pharmaceuticals, GenesisCare, Janssen Oncology, Merck, Myovant Sciences, Pfizer Inc., Sanofi-Genzyme, and Tolmar Pharmaceuticals, Inc. Murilo de Almeida Luz reports receiving speaker honoraria from Astellas Pharma Inc., Bayer, Janssen, Merck Sharp & Dohme, and Pfizer Inc.; being an advisory board member for Astellas Pharma Inc., Bayer, and Janssen; sponsored research from Bayer, Bristol Myers Squibb, Ferring Pharmaceuticals, GlaxoSmithKline, Janssen, and Roche; receiving travel expenses from AstraZeneca, Bayer, Janssen, and Pfizer Inc. Ugo De Giorgi reports serving as a consultant for Janssen, Astellas Pharma Inc., Sanofi, Bayer, Pfizer Inc., Bristol Myers Squibb, Novartis, Ipsen, and Merck Sharp & Dohme. Martin Gleave reports stock or ownership interest in OncoGenex Technologies Inc., Sustained Therapeutics Inc., and Sikta Biopharma; is a consultant to Astellas Pharma Inc., AstraZeneca, Bayer, Genova Diagnostics (GDx), Janssen, Pfizer Inc., Roche, Sanofi, and TerSera Therapeutics LLC; and holds patents for OGX-011, OGX-427, ST-CP, and ST-POP. Geoffrey T. Gotto reports receiving honoraria from Amgen, Astellas Pharma Inc., Bayer, Ferring Pharmaceuticals, Janssen, and Merck; being a consultant or advisory board member for Amgen, Astellas Pharma Inc., Bayer, Janssen, and Merck; providing expert testimony for Janssen; and receiving support for travel, accommodation, and expenses from Janssen. Gabriel P. Haas reports being an employee of and shareholder in Astellas Pharma Inc. Miguel Ramirez-Backhaus reports serving as a consultant or advisory board member for Astellas Pharma Inc., Bayer, Janssen, and Karl Storz; receiving speaker honoraria from Astellas Pharma Inc., Bayer, Janssen, and GP Pharm. Antti Rannikko reports being a board member for the Ida Montin Foundation, and Orion Research Foundation; being an advisory board member for Bayer, Janssen, and Orion Pharma; being a stockholder and clinical advisor for Aqsens Health; being a clinical investigator for Astellas Pharma Inc., Bayer, Janssen, Orion Pharma, and RhoVac AB; and receiving competitive state research funding from HUS Helsinki University Hospital, Finnish Cancer Organizations, and the Jane and Aatos Erkko Foundation. Jamal Tazari, Yiyun Tang, and Fabian Zohren are employees of and shareholders in Pfizer Inc. Swetha Sridharan reports no conflicts of interest. Jennifer Sugg is an employee of Astellas Pharma Inc., and a shareholder in AstraZeneca. Ronald F. Tutrone, Jr. reports being an advisory board member for Bayer; and receiving speaker honoraria from Astellas Pharma Inc., Exosome Diagnostics, Inc., Myovant Sciences, and Pfizer Inc. Balaji Venugopal reports receiving honoraria from Bristol Myers Squibb, Eisai Co., Ltd, EUSA Pharma, Ipsen, Janssen, and Merck; being a consultant or advisory board member for Janssen, Merck Sharp & Dohme Oncology, and Pfizer Inc./EMD Serono; and receiving support for travel, accommodation, and expenses from EUSA Pharma, and Ipsen. Arnauld Villers reports receiving research grants from Astellas Pharma Inc., Ferring Pharmaceuticals, Ipsen, and Janssen. Henry H. Woo reports being an advisory board member for Astellas Pharma Inc., Bayer, Boston Scientific Corporation, and Mundipharma International Ltd; and reports receiving speaker honoraria from AbbVie, Astellas Pharma Inc., Boston Scientific Corporation, and Janssen. Stephen J. Freedland reports being a consultant for Astellas Pharma Inc., AstraZeneca, Bayer, Dendreon Pharmaceuticals LLC, Janssen, Merck, Myovant Sciences, Pfizer Inc., and Sanofi.
Acknowledgments::
The authors thank all the patients, their families, and the investigators and investigational site members involved in this study.
Editorial Acknowledgement::
Medical writing and editorial support was provided by Julie B. Stimmel, PhD, Sinead Stewart, and Rosie Henderson, of Onyx (a Prime Global Agency), funded by Pfizer, Inc. and Astellas Pharma Inc., the co-developers of enzalutamide.
Submission Category::
prostate cancer.
Sub-category::
Advanced (including drug therapy).
01 Feb 2023Molecular therapy : the journal of the American Society of Gene Therapy
DDX5 mRNA-targeting antisense oligonucleotide as a new promising therapeutic in combating castration-resistant prostate cancer
Article
Author: Cherif, Chaïma ; Audebert, Stéphane ; Taïeb, David ; Paris, Clément ; Baudelet, Emilie ; Fazli, Ladan ; Gleave, Martin ; Bertucci, François ; Bastide, Cyrille ; Lannes, François ; Finetti, Pascal ; Hamimed, Mourad ; Le, Thi Khanh ; Rocchi, Palma ; Omabe, Kenneth ; Barbolosi, Dominique
The heat shock protein 27 (Hsp27) has emerged as a principal factor of the castration-resistant prostate cancer (CRPC) progression. Also, an antisense oligonucleotide (ASO) against Hsp27 (OGX-427 or apatorsen) has been assessed in different clinical trials. Here, we illustrate that Hsp27 highly regulates the expression of the human DEAD-box protein 5 (DDX5), and we define DDX5 as a novel therapeutic target for CRPC treatment. DDX5 overexpression is strongly correlated with aggressive tumor features, notably with CRPC. DDX5 downregulation using a specific ASO-based inhibitor that acts on DDX5 mRNAs inhibits cell proliferation in preclinical models, and it particularly restores the treatment sensitivity of CRPC. Interestingly, through the identification and analysis of DDX5 protein interaction networks, we have identified some specific functions of DDX5 in CRPC that could contribute actively to tumor progression and therapeutic resistance. We first present the interactions of DDX5 and the Ku70/80 heterodimer and the transcription factor IIH, thereby uncovering DDX5 roles in different DNA repair pathways. Collectively, our study highlights critical functions of DDX5 contributing to CRPC progression and provides preclinical proof of concept that a combination of ASO-directed DDX5 inhibition with a DNA damage-inducing therapy can serve as a highly potential novel strategy to treat CRPC.
01 Dec 2019The oncologistQ2 · MEDICINE
A Randomized, Double-Blinded, Phase II Trial of Carboplatin and Pemetrexed with or without Apatorsen (OGX-427) in Patients with Previously Untreated Stage IV Non-Squamous-Non-Small-Cell Lung Cancer: The SPRUCE Trial
Q2 · MEDICINE
Article
Author: Shipley, Dianna L. ; Spigel, David R. ; Hainsworth, John D. ; Waterhouse, David M. ; Page, Ray D. ; Jones, Suzanne F. ; Mekhail, Tarek ; Jacobs, Cindy ; Ward, Patrick J. ; Hemphill, Brian ; Shih, Kent C. ; McCleod, Michael ; Burris, Howard A. ; Whorf, Robert C. ; Stilwill, Joseph
Abstract:
Background:
This randomized, double-blinded, phase II trial evaluated the efficacy of carboplatin and pemetrexed plus either apatorsen, an antisense oligonucleotide targeting heat shock protein (Hsp) 27 mRNA, or placebo in patients with previously untreated metastatic nonsquamous non-small cell lung cancer (NSCLC).
Methods:
Patients were randomized 1:1 to Arm A (carboplatin/pemetrexed plus apatorsen) or Arm B (carboplatin/pemetrexed plus placebo). Treatment was administered in 21-day cycles, with restaging every two cycles, until progression or intolerable toxicity. Serum Hsp27 levels were analyzed at baseline and during treatment. The primary endpoint was progression-free survival (PFS); secondary endpoints included overall survival (OS), objective response rate, and toxicity.
Results:
The trial enrolled 155 patients (median age 66 years; 44% Eastern Cooperative Oncology Group performance status 0). Toxicities were similar in the 2 treatment arms; cytopenias, nausea, vomiting, and fatigue were the most frequent treatment-related adverse events. Median PFS and OS were 6.0 and 10.8 months, respectively, for Arm A, and 4.9 and 11.8 months for Arm B (differences not statistically significant). Overall response rates were 27% for Arm A and 32% for Arm B. Sixteen patients (12%) had high serum levels of Hsp27 at baseline. In this small group, patients who received apatorsen had median PFS of 10.8 months, and those who received placebo had median PFS 4.8 months.
Conclusion:
The addition of apatorsen to carboplatin and pemetrexed was well tolerated but did not improve outcomes in patients with metastatic nonsquamous NSCLC cancer in the first-line setting.
Implications for Practice:
This randomized, double-blinded, phase II trial evaluated the efficacy of carboplatin and pemetrexed plus either apatorsen, an antisense oligonucleotide targeting heat shock protein 27 mRNA, or placebo in patients with previously untreated metastatic nonsquamous non-small cell lung cancer (NSCLC). The addition of apatorsen to carboplatin and pemetrexed was well tolerated but did not improve outcomes in patients with metastatic nonsquamous NSCLC cancer in the first-line setting.
1
News (Medical) associated with Apatorsen100 Deals associated with Apatorsen
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External Link
| KEGG | Wiki | ATC | Drug Bank |
|---|---|---|---|
| - | - | - |
R&D Status
10 top R&D records. to view more data
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Advanced Lung Squamous Cell Carcinoma | Phase 2 | United Kingdom | 01 Jun 2014 | |
| Metastatic Pancreatic Cancer | Phase 2 | United States | 01 Sep 2013 | |
| Advanced Lung Non-Squamous Non-Small Cell Carcinoma | Phase 2 | United States | 01 Jul 2013 | |
| metastatic non-small cell lung cancer | Phase 2 | United States | 01 Jul 2013 | |
| Non-squamous non-small cell lung cancer | Phase 2 | United States | 01 Jul 2013 | |
| Recurrent Bladder Cancer | Phase 2 | United States | 01 Apr 2013 | |
| Urothelial Carcinoma of the Urinary Bladder | Phase 2 | United States | 01 Apr 2013 | |
| Urothelial carcinoma recurrent | Phase 2 | United States | 01 Apr 2013 | |
| Metastatic castration-resistant prostate cancer | Phase 2 | United States | 01 Dec 2012 | |
| Metastatic castration-resistant prostate cancer | Phase 2 | Canada | 01 Dec 2012 |
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Clinical Result
Clinical Result
Indication
Phase
Evaluation
View All Results
Phase 2 | Non-squamous non-small cell lung cancer First line | 155 | Carboplatin+Pemetrexed+Apatorsen | lsmszipaab(yyhnofxbxg) = jofobxdrof pclzvfzmop (qkgqtkmzyu ) View more | Negative | 01 Dec 2019 | |
Carboplatin+Pemetrexed+Placebo | lsmszipaab(yyhnofxbxg) = lmqeahgwty pclzvfzmop (qkgqtkmzyu ) View more | ||||||
Phase 2 | 72 | (Experimental: Arm A) | ochnsuyuvw = jpqazxlpdh ayfkpoeaif (atmvkllvuu, ggagxwlpxi - hwfvittbmu) View more | - | 18 Oct 2018 | ||
(Control Arm: Arm B) | ochnsuyuvw = hqldxetetw ayfkpoeaif (atmvkllvuu, grzpeoblan - cglecocdym) View more | ||||||
Phase 2 | 155 | (OGX-427) | murrcijgyx(cnjcctwuyc) = nidzqmkcjp qjasmcfmqv (ypyicjbest, kbgvqmrgtw - savhagecep) View more | - | 08 Jun 2018 | ||
Placebo (Placebo) | murrcijgyx(cnjcctwuyc) = dbcneixrep qjasmcfmqv (ypyicjbest, wazzysjvhy - sinkfsahah) View more | ||||||
Phase 2 | 200 | qdayqkpwjs(hmklciggec) = qiksskauco feakhunovy (dvombvjdoh ) | Positive | 01 May 2018 | |||
qdayqkpwjs(hmklciggec) = zdrlhzzope feakhunovy (dvombvjdoh ) | |||||||
NCT01120470 (Pubmed) Manual  | Phase 2 | 74 | znaxykyizt(ekkgyngkwa) = ojtrsnjqbf wtnooyxwhq (fjqjwwvnrf, 32.9 - 67.1) View more | Positive | 01 Apr 2018 | ||
znaxykyizt(ekkgyngkwa) = hzppifsbuz wtnooyxwhq (fjqjwwvnrf, 26.3 - 59.2) View more | |||||||
Phase 2 | Metastatic Pancreatic Cancer First line | 132 | Gemcitabine+Nab-Paclitaxel+Apatorsen | kakvuezddk(pnjrwdhagc) = raznotiyto silsxxuava (lslacnykkk ) View more | Negative | 01 Dec 2017 | |
Gemcitabine+Nab-Paclitaxel+Placebo | kakvuezddk(pnjrwdhagc) = sunjpmwetj silsxxuava (lslacnykkk ) View more | ||||||
Phase 2 | 132 | (OGX-427) | kezqjfeiwl(pvvgcqxotr) = gxhnonrzqo tfwhctfupe (mcvaraubso, wsturqgfud - vkzhmbvatc) View more | - | 16 May 2017 | ||
Placebo (Placebo) | kezqjfeiwl(pvvgcqxotr) = cphcvkgdsq tfwhctfupe (mcvaraubso, npdoxrhvlc - ykqfmrwmge) View more | ||||||
Phase 1 | 42 | ktkahjlzyz(strcrggdfi) = A maximum tolerated dose was not defined. rqgmitgzfz (jyqbdzxdtc ) | Positive | 01 Jun 2016 | |||
(patients with CRPC) | |||||||
Phase 2 | 155 | Carboplatin + Pemetrexed + Apatorsen | vovuptetrc(bkkprmhlpq) = pihhwghank ubbvbmumgc (jofnhaaamt ) View more | Negative | 20 May 2016 | ||
Carboplatin + Pemetrexed + Placebo | vovuptetrc(bkkprmhlpq) = xvqszuguvd ubbvbmumgc (jofnhaaamt ) View more | ||||||
Phase 2 | 38 | hbkpikrnrb(xrcvperugs) = bytedyurlg bcwvbcbjdn (rpcgsapuye ) View more | - | 20 May 2012 | |||
hbkpikrnrb(xrcvperugs) = cnemdxwiaz bcwvbcbjdn (rpcgsapuye ) |
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