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Last update 27 Mar 2025

Danvatirsen

Last update 27 Mar 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

ASO

Synonyms

Danvatirsen-sodium, IONIS-STAT3RX, ISIS-STAT3RX

+ [9]

Target

STAT3

Action

inhibitors

Mechanism

STAT3 inhibitors(Signal transducer and activator of transcription 3 inhibitors)

Therapeutic Areas

NeoplasmsNervous System DiseasesCongenital Disorders+ [5]

Active Indication

Recurrent Squamous Cell Carcinoma of the Head and NeckAdvanced Colorectal AdenocarcinomaAdvanced Lung Non-Small Cell Carcinoma+ [10]

Inactive Indication

Advanced cancerAdvanced Hepatocellular CarcinomaDiffuse Large B-Cell Lymphoma+ [2]

Originator Organization

Ionis Pharmaceuticals, Inc.

Active Organization

AstraZeneca PLC

Flamingo Therapeutics BVStartup

Dynacure SAStartup

Inactive Organization

Ionis Pharmaceuticals, Inc.

MedImmune LLC

Drug Highest PhasePhase 2

First Approval Date-

Regulation-

Structure/Sequence

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Sequence Code 526076414

Source: *****

Clinical Trials associated with Danvatirsen

NCT05986240

/ RecruitingPhase 1IIT

A Phase I Study Investigating the Safety & Efficacy of Danvatirsen as Monotherapy Followed by Combination With Venetoclax in Patients With Relapsed/Refractory MDS & AML

This is a Phase 1 study investigating the safety and efficacy of Danvatirsen as a monotherapy followed by combination with Venetoclax in patients with relapsed/refractory myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML).
Funding Source: FDA OOPD

Start Date08 May 2024

Sponsor / Collaborator

Montefiore Medical Center

[+2]

NCT05814666

/ RecruitingPhase 2

An Open-Label, Phase II, Randomized, Controlled Study of Danvatirsen Plus Pembrolizumab Compared to Pembrolizumab Alone in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma (HNSCC)

Open-label, Phase II, randomized, controlled study evaluating the efficacy and safety of danvatirsen in combination with pembrolizumab compared with pembrolizumab alone as first-line treatment of patients with recurrent/metastatic (R/M) HNSCC. Two-thirds of patients will be randomized to receive danvatirsen and pembrolizumab and one-third will be randomized to receive pembrolizumab alone.

Start Date30 May 2023

Sponsor / Collaborator

Flamingo Therapeutics BVStartup

NCT03794544

/ CompletedPhase 2

A Phase 2 Open-label, Multicenter, Randomized, Multidrug Platform Study of Neoadjuvant Durvalumab Alone or in Combination With Novel Agents in Subjects With Resectable, Early-stage (I [> 2 cm] to IIIA) Non-small Cell Lung Cancer (NeoCOAST)

Study D9108C00002 (NeoCOAST) is a platform study assessing the effectiveness and safety of neoadjuvant durvalumab alone or in combination with novel agents in participants with resectable, early-stage (Stage I [>2cm] to IIIA) non-small cell lung cancer (NSCLC).

Start Date08 Mar 2019

Sponsor / Collaborator

MedImmune LLC

100 Clinical Results associated with Danvatirsen

100 Translational Medicine associated with Danvatirsen

100 Patents (Medical) associated with Danvatirsen

Literatures (Medical) associated with Danvatirsen

01 Jul 2024·BMJ Oncology

Preclinical study and parallel phase II trial evaluating antisense STAT3 oligonucleotide and checkpoint blockade for advanced pancreatic, non-small cell lung cancer and mismatch repair-deficient colorectal cancer

Article

Author: Couillault, Coline ; Tang, Chad ; Piha-Paul, Sarina A ; Pant, Shubham ; Dai, James ; Karp, Daniel D ; Nicholas, Courtney ; Hartley, Genevieve P ; Proia, Theresa ; Yuan, Ying ; Fu, Siqing ; Dhebat, Sarah ; Curran, Michael A ; Lin, Heather ; Yap, Timonthy A ; Subbiah, Vivek ; Hong, David S ; Tsimberidou, Apostolia M ; Guerrero, Paola ; Srinivasamani, Anupallavi ; Dumbrava, Ecaterina E Ileana ; Naing, Aung ; Rodon Ahnert, Jordi

Objective:

To evaluate signal transducer and activator of transcription 3 (STAT3) inhibition we conducted a co-clinical trial testing danvatirsen, a STAT3 antisense oligonucleotide (ASO) and checkpoint inhibition in conjunction with preclinical experiments.

Methods and analysis:

Orthotopically implanted pancreatic cancer (pancreatic adenocarcinoma (PDAC)) was treated with STAT3 ASO with immune checkpoint inhibition. Tumour infiltrating immune cell populations were characterised via flow cytometry. In vitro experiments evaluated STAT3 inhibition in pancreatic stellate cells (PSCs) and myeloid-derived suppressor cells (MDSCs).A phase II trial employing a Simon II stage design tested the clinical efficacy of danvatirsen and durvalumab in non-small cell lung cancer (NSCLC), PDAC and mismatch repair-deficient colorectal cancer (MRD CRC). The primary objective was 4-month disease control rate (DCR).

Results:

In vivo studies identified improvement in survival of PDAC implanted mice treated with STAT3 ASO and checkpoint inhibition. Within tumour-infiltrating lymphocytes there was expansion of CD4 and PD-1+ CD8 populations with STAT3 ASO.Thirty-seven patients (29 PDAC, 7 NSCLC and 1 MRD CRC) from a single institution started treatment on trial between April 2017 and March 2020. No objective responses were observed. Four of six (66.7%, 95% CI 22.3% to 95.7%) NSCLC and 4 of 23 (17.4%, 95% CI 5% to 38.8%) PDAC patients exhibited 4-month DCR. Follow-up in vitro studies revealed an anti-inflammatory and pro-tumour effect of STAT3 ASO mediated by PSCs and MDSCs distinct from ablation of STAT3.

Conclusion:

Although durvalumab and danvatirsen met the primary endpoint, no objective responses were observed. A rationale for the lack of objective responses is danvatirsen-induced myeloid immune suppression.

Trial registration number:

NCT02983578 .

01 Mar 2024·Nature medicineQ1 · MEDICINE

Biomarker-directed targeted therapy plus durvalumab in advanced non-small-cell lung cancer: a phase 2 umbrella trial

Q1 · MEDICINE

Article

Author: Jewsbury, Philip J ; Barrett, J Carl ; Shepherd, Frances A ; Cosaert, Jan ; Aronson, Boaz ; Thomas, Michael ; Johnson, Melissa L ; Awad, Mark M ; Khosla, Sajan ; Morgensztern, Daniel ; Heymach, John V ; Cousin, Sophie ; Russell, Deanna L ; Goss, Glenwood ; Barry, Simon T ; Kim, Sang-We ; Kumar, Rakesh ; Moskovitz, Mor T ; Besse, Benjamin ; Kim, Dong-Wan ; Park, Keunchil ; Wheatley-Price, Paul ; Pons-Tostivint, Elvire ; Iyer, Sonia ; Dressman, Marlene ; Hobson, Rosalind ; Hartl, Sylvia ; Reddy, Avinash ; Dean, Emma ; Cheema, Parneet ; Florescu, Marie ; Conway, James P ; Keddar, Mohamed Reda ; Ambrose, Helen J ; Hochmair, Maximilian J ; Chu, Quincy S C ; Vicente, David ; Forde, Patrick M

Abstract:

For patients with non-small-cell lung cancer (NSCLC) tumors without currently targetable molecular alterations, standard-of-care treatment is immunotherapy with anti-PD-(L)1 checkpoint inhibitors, alone or with platinum-doublet therapy. However, not all patients derive durable benefit and resistance to immune checkpoint blockade is common. Understanding mechanisms of resistance—which can include defects in DNA damage response and repair pathways, alterations or functional mutations in STK11/LKB1, alterations in antigen-presentation pathways, and immunosuppressive cellular subsets within the tumor microenvironment—and developing effective therapies to overcome them, remains an unmet need. Here the phase 2 umbrella HUDSON study evaluated rational combination regimens for advanced NSCLC following failure of anti-PD-(L)1-containing immunotherapy and platinum-doublet therapy. A total of 268 patients received durvalumab (anti-PD-L1 monoclonal antibody)–ceralasertib (ATR kinase inhibitor), durvalumab–olaparib (PARP inhibitor), durvalumab–danvatirsen (STAT3 antisense oligonucleotide) or durvalumab–oleclumab (anti-CD73 monoclonal antibody). Greatest clinical benefit was observed with durvalumab–ceralasertib; objective response rate (primary outcome) was 13.9% (11/79) versus 2.6% (5/189) with other regimens, pooled, median progression-free survival (secondary outcome) was 5.8 (80% confidence interval 4.6–7.4) versus 2.7 (1.8–2.8) months, and median overall survival (secondary outcome) was 17.4 (14.1–20.3) versus 9.4 (7.5–10.6) months. Benefit with durvalumab–ceralasertib was consistent across known immunotherapy-refractory subgroups. In ATM-altered patients hypothesized to harbor vulnerability to ATR inhibition, objective response rate was 26.1% (6/23) and median progression-free survival/median overall survival were 8.4/22.8 months. Durvalumab–ceralasertib safety/tolerability profile was manageable. Biomarker analyses suggested that anti-PD-L1/ATR inhibition induced immune changes that reinvigorated antitumor immunity. Durvalumab–ceralasertib is under further investigation in immunotherapy-refractory NSCLC.ClinicalTrials.gov identifier: NCT03334617

01 Nov 2023·Cancer discovery

Neoadjuvant Durvalumab Alone or Combined with Novel Immuno-Oncology Agents in Resectable Lung Cancer: The Phase II NeoCOAST Platform Trial

Article

Author: Spira, Alexander I. ; Spigel, David R. ; Yau, Edwin H. ; Weder, Walter ; Hamid, Oday ; Kar, Gozde ; Sellman, Bret R. ; García-Campelo, Rosario ; Soo-Hoo, Yee ; Tan, Tze Heng ; Mager, Raymond ; Cascone, Tina ; McGrath, Lara ; Rodriguez-Canales, Jaime ; Cheng, Lin-Yang ; Grenga, Italia ; Spicer, Jonathan D. ; Anagnostou, Valsamo ; Surace, Michael ; Daniel, Davey B. ; Zheng, Ying ; Kumar, Rakesh ; Forde, Patrick M. ; Gopalakrishnan, Vancheswaran ; Hussein, Maen ; Mazieres, Julien ; Oliveira, Julio ; Blando, Jorge

Abstract:

Neoadjuvant chemoimmunotherapy improves pathologic complete response rate and event-free survival in patients with resectable non–small cell lung cancer (NSCLC) versus chemotherapy alone. NeoCOAST was the first randomized, multidrug platform trial to examine novel neoadjuvant immuno-oncology combinations for patients with resectable NSCLC, using major pathologic response (MPR) rate as the primary endpoint. Eighty-three patients received a single cycle of treatment: 26 received durvalumab (anti–PD-L1) monotherapy, 21 received durvalumab plus oleclumab (anti-CD73), 20 received durvalumab plus monalizumab (anti-NKG2A), and 16 received durvalumab plus danvatirsen (anti-STAT3 antisense oligonucleotide). MPR rates were higher for patients in the combination arms versus durvalumab alone. Safety profiles for the combinations were similar to those of durvalumab alone. Multiplatform immune profiling suggested that improved MPR rates in the durvalumab plus oleclumab and durvalumab plus monalizumab arms were associated with enhanced effector immune infiltration of tumors, interferon responses and markers of tertiary lymphoid structure formation, and systemic functional immune cell activation.

Significance::

A neoadjuvant platform trial can rapidly generate clinical and translational data using candidate surrogate endpoints like MPR. In NeoCOAST, patients with resectable NSCLC had improved MPR rates after durvalumab plus oleclumab or monalizumab versus durvalumab alone and tumoral transcriptomic signatures indicative of augmented immune cell activation and function.See related commentary by Cooper and Yu, p. 2306.This article is featured in Selected Articles from This Issue, p. 2293

News (Medical) associated with Danvatirsen

04 Dec 2024

·www.prnewswire.com

Flamingo Therapeutics Announces Poster Presentation on AML Phase 1 IIT Study at ASH 2024

LEUVEN, Belgium and STRASBOURG, France and PHILADELPHIA, Dec. 4, 2024 /PRNewswire/ -- Flamingo Therapeutics ("Flamingo") today announced that an abstract has been selected for presentation at the 66th Annual ASH Annual Meeting and Exposition, being held in San Diego, CA from December 7-9, 2024. The abstract, selected for a poster presentation, is a Trials-in-Progress presentation of a Phase 1 investigator-initiated study of danvatirsen monotherapy followed by danvatirsen combination with venetoclax in relapsed/refractory AML and intermediate/high risk MDS (NCT05986240). The study is being conducted at Albert Einstein / Montefiore Medical Center in New York City and MD Anderson Cancer Center in Houston. Poster presentation details are as follows: Title: "A Phase 1 Study Investigating the Safety and Efficacy of Danvatirsen As Monotherapy Followed by Combination with Venetoclax in Patients with Relapsed/Refractory MDS and AML" Session Date: Monday, December 9, 2024 Presenting Author: Dr. Aditi Shastri For more information, please visit the ASH 2024 website. About Flamingo Therapeutics Flamingo is pioneering RNA-targeted therapies for oncology with a clinical-stage pipeline targeting undruggable transcription factors and long non-coding RNAs. Flamingo has an alliance with Ionis Pharmaceuticals and is supported by well-known biotechnology investors including Abrdn (formerly funds managed by Tekla Capital Management LLC), Andera Partners, Bpifrance Large Venture, Bpifrance through its FABS and Fonds Biothérapies Innovantes et Maladies Rares funds, Eurazeo - Kurma Partners, Perceptive Advisors, PMV, Pontifax, Sphera, and VIB. Flamingo has initiated a Phase II trial 'PEMDA-HN' evaluating the STAT3 targeting agent danvatirsen, in combination with pembrolizumab, in patients with head and neck squamous cell carcinoma (HNSCC). A Phase I investigator initiated trial study has also been initiated evaluating danvatirsen as a monotherapy and in combination with venetoclax in AML/MDS patients. For more information on Flamingo, please visit: PEMDA-HN (head and neck cancer) on clinicaltrials.gov: Danvatirsen monotherapy followed by combination with venetoclax in relapsed/ refractory AML and MDS on clincaltrials.gov: Please engage with us on LinkedIn: . Flamingo Media and Investor Contact: Amy Conrad Juniper Point (858) 366-3243 [email protected] SOURCE Flamingo Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1Phase 2AACRsiRNA

29 Aug 2024

·www.prnewswire.com

Flamingo Therapeutics Announces Participation in Upcoming Investor Conferences

LEUVEN, Belgium and STRASBOURG, France and PHILADELPHIA, Aug. 29, 2024 /PRNewswire/ -- Flamingo Therapeutics ("Flamingo") announced today its participation in the following upcoming investor conferences: Wells Fargo Annual Healthcare Conference, September 4-6, 2024, Boston, MA PA Life Sciences Future Conference, September 18-19, 2024, Philadelphia, PA 8th Annual Chardan Genetic Medicines Conference, September 30-October 1, 2024, NY 4th Annual Needham Private Biotech Company Virtual 1x1 Forum, October 8-9, 2024 During these events, Company management will participate in one-on-one meetings with investors. About Flamingo Therapeutics Flamingo is pioneering RNA-targeted therapies for oncology with a clinical-stage pipeline targeting undruggable transcription factors and long non-coding RNAs. Flamingo has an alliance with Ionis Pharmaceuticals and is supported by well-known biotechnology investors including Abrdn (formerly funds managed by Tekla Capital Management LLC), Andera Partners, Bpifrance Large Venture, Bpifrance through its FABS and Fonds Biothérapies Innovantes et Maladies Rares funds, Eurazeo - Kurma Partners, Perceptive Advisors, PMV, Pontifax, Sphera, and VIB. Flamingo has initiated a Phase II trial 'PEMDA-HN' evaluating the STAT3 targeting agent danvatirsen, in combination with pembrolizumab, in patients with head and neck squamous cell carcinoma (HNSCC). A Phase I investigator initiated trial study has also been initiated evaluating danvatirsen as a monotherapy and in combination with venetoclax in AML/MDS patients. For more information on Flamingo, please visit: PEMDA-HN (head and neck cancer) on clinicaltrials.gov: Danvatirsen monotherapy followed by combination with venetoclax in relapsed/ refractory AML and MDS on clincaltrials.gov: Please engage with us on LinkedIn: . Flamingo Media and Investor Contact: Amy Conrad Juniper Point (858) 366-3243 [email protected] SOURCE Flamingo Therapeutics

Phase 1Phase 2siRNA

29 Aug 2024

·flamingotx.com

Flamingo Therapeutics Announces Participation in Upcoming Investor Conferences - Flamingo Therapeutics

Leuven (Belgium), Strasbourg (France), Philadelphia (USA) – August 29, 2024 /PRNewswire/- Flamingo Therapeutics (“Flamingo”) announced today its participation in the following upcoming investor conferences: Wells Fargo Annual Healthcare Conference, September 4-6, 2024, Boston, MA PA Life Sciences Future Conference, September 18-19, 2024, Philadelphia, PA 8th Annual Chardan Genetic Medicines Conference, September 30-October 1, 2024, NY 4thAnnual Needham Private Biotech Company Virtual 1x1 Forum, October 8-9, 2024 During these events, Company management will participate in one-on-one meetings with investors. About Flamingo Therapeutics Flamingo is pioneering RNA-targeted therapies for oncology with a clinical-stage pipeline targeting undruggable transcription factors and long non-coding RNAs. Flamingo has an alliance with Ionis Pharmaceuticals and is supported by well-known biotechnology investors including Abrdn (formerly funds managed by Tekla Capital Management LLC), Andera Partners, Bpifrance Large Venture, Bpifrance through its FABS and Fonds Biothérapies Innovantes et Maladies Rares funds, Eurazeo - Kurma Partners, Perceptive Advisors, PMV, Pontifax, Sphera, and VIB. Flamingo has initiated a Phase II trial ‘PEMDA-HN’ evaluating the STAT3 targeting agent danvatirsen, in combination with pembrolizumab, in patients with head and neck squamous cell carcinoma (HNSCC). A Phase I investigator initiated trial study has also been initiated evaluating danvatirsen as a monotherapy and in combination with venetoclax in AML/MDS patients. For more information on Flamingo, please visit:https://flamingotx.com PEMDA-HN (head and neck cancer) on clinicaltrials.gov:https://clinicaltrials.gov/study/NCT05814666 Danvatirsen monotherapy followed by combination with venetoclax in relapsed/ refractory AML and MDS on clincaltrials.gov:https://classic.clinicaltrials.gov/ct2/show/NCT05986240 Please engage with us on LinkedIn:https://www.linkedin.com/company/flamingo-therapeutics. Flamingo Media and Investor Contact: Amy Conrad Juniper Point (858) 366-3243 amy@juniper-point.com

Phase 1siRNAPhase 2

100 Deals associated with Danvatirsen

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB14825

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Advanced Colorectal Adenocarcinoma	Phase 2	United States	AstraZeneca PLC	02 Mar 2017
Advanced Lung Non-Small Cell Carcinoma	Phase 2	United States	AstraZeneca PLC	02 Mar 2017
Colorectal Cancer, Hereditary Nonpolyposis, Type 1	Phase 2	United States	AstraZeneca PLC	02 Mar 2017
Microsatellite instability-high colorectal cancer	Phase 2	United States	AstraZeneca PLC	02 Mar 2017
Pancreatic Cancer	Phase 2	United States	AstraZeneca PLC	02 Mar 2017
Refractory Colorectal Carcinoma	Phase 2	United States	AstraZeneca PLC	02 Mar 2017
Refractory Lung Carcinoma	Phase 2	United States	AstraZeneca PLC	02 Mar 2017
Turcot Syndrome	Phase 2	United States	AstraZeneca PLC	02 Mar 2017
Advanced Malignant Solid Neoplasm	Phase 2	United States	AstraZeneca PLC	06 Aug 2015
Advanced Malignant Solid Neoplasm	Phase 2	Belgium	AstraZeneca PLC	06 Aug 2015

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03794544 (AACR2022) Manual	Phase 2	Non-Small Cell Lung Cancer Neoadjuvant	84	Durvalumab	dnownovhwa(gmcbzpkygx) = pefenapdak tdsyizfgqm (vumcqaslbj, 2.4 - 29.2) View more	Positive	15 Jun 2022
NCT03794544 (AACR2022) Manual	Phase 2	Non-Small Cell Lung Cancer Neoadjuvant	84	Durvalumab+Oleclumab	dnownovhwa(gmcbzpkygx) = ttzibizaiz tdsyizfgqm (vumcqaslbj, 5.4 - 41.9) View more	Positive	15 Jun 2022
NCT03421353 (AACR2020)	Phase 1	HR-positive/HER2-low Solid Tumors	76	Danvatirsen 200 mg IV QW	mkecbwmycu(cccdzcsnza) = fndxdrqtoi jnbdmpfmko (sdfnbnavmu ) View more	-	15 Aug 2020
NCT03421353 (AACR2020)	Phase 1	HR-positive/HER2-low Solid Tumors	76	Danvatirsen 400 mg IV Q2W	mkecbwmycu(cccdzcsnza) = vlbsivnvfs jnbdmpfmko (sdfnbnavmu ) View more	-	15 Aug 2020
NCT01563302 (Pubmed \| J Immunother Cancer) Manual	Phase 1	Lymphoma	30	AZD9150	hongmqfhpl(njfhgvtsfq) = wmmnsmqygk tjohvtbvbv (pzuuquiozf ) View more	Positive	16 Nov 2018
NCT02499328 (SCORES, ESMO2018) Manual	Phase 1/2	Recurrent Squamous Cell Carcinoma of the Head and Neck \| Advanced Malignant Solid Neoplasm Second line	58	durvalumab+danvatirsen	rhtpjcxrzz(abuhrmneym) = tqhicinrmw qxswswtutt (vivsoctllt ) View more	Positive	22 Oct 2018
NCT02499328 (SCORES, ESMO2018) Manual	Phase 1/2		58	durvalumab+AZD5069	eefvlhsdek(diysslgnvt) = vzjcnilmgj owclhlnxad (xyoiqqhmmy ) View more	Positive	22 Oct 2018
NCT02417753 (CTgov)	Phase 2	Ovarian Cancer \| Malignant ascites \| Gastrointestinal Neoplasms	1	AZD9150	nljmewjjlx = cnqjtpqfjo jdodpmvcjy (bdcownswwi, casikvlces - ealnjotgrw) View more	-	15 May 2017
NCT01563302 (ASCO2013)	Phase 1	Advanced cancer	15	ISIS 481464	gzpohuszsg(duivzkombl) = jcfotdyxxw uqcvjaeexr (jrplstukji )	-	20 May 2013

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