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AZD3470

Phase 1

Advanced Solid Tumors That Are MTAP Deficient | Small molecule | Oncology |AstraZeneca PLC|Last Updated: Aug 27, 2026

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment334

FDA Designations

No designations recorded

Clinical trial landscape

AZD3470 · 2 trials · 8 indications

Phase 1 2
NCT06137144AZD3470 as Monotherapy or in Combination With Anticancer Agent(s) in Participants With Haematologic Malignancies.Lymphoma
RECRUITING161 Analytics
NCT06130553A Study of AZD3470, a PRMT5 Inhibitor, Given as Monotherapy and in Combination in Patients With MTAP Deficient Advanced/Metastatic Solid TumorsAdvanced Solid Tumors That Are MTAP Deficient
RECRUITING334 Analytics
PHASE1RECRUITING
AZD3470 as Monotherapy or in Combination With Anticancer Agent(s) in Participants With Haematologic Malignancies.
LymphomaUnlock trial analytics
PHASE1RECRUITING
A Study of AZD3470, a PRMT5 Inhibitor, Given as Monotherapy and in Combination in Patients With MTAP Deficient Advanced/Metastatic Solid Tumors
Advanced Solid Tumors That Are MTAP DeficientUnlock trial analytics

Study Endpoints

Primary Endpoints

Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
From Screening continuously until 28 days after the last dose of study medication.

AEs: Number of patients with adverse events by system organ class and preferred term. SAEs: Number of patients with serious adverse events by system organ class and preferred term.

Incidence of DLTs (Dose Escalation Cohorts only)
From first dose of AZD3470 to end of Cycle 1 (each cycle is 21 days).

In the Dose Escalation cohorts in Part A, the number of participants with at least 1 dose-limiting toxicity (DLT), which is any toxicity defined as a DLT in the Clinical Study Protocol.

All Modules: Incidence of adverse events (AEs) and serious adverse events (SAEs). To determine the RP2D of AZD3470 as monotherapy and in combination with anticancer agents
From time of informed consent to 28 days post last dose of study treatment

Number of participants with AEs and SAEs.

Module 1: Incidence of dose-limiting toxicities (DLT)
From first dose of study treatment until the end of Cycle 1 (each cycle is 21 days)

Incidence of dose-limiting toxicities (DLT) as determined by number of patients with at least 1 dose-limiting toxicity (DLT)

Module 2: Progression Free Survival assessed by the Investigator according to RECIST v1.1
From date of randomization up until progression, or the last evaluable assessment in the absence of progression (approximately 2 years).

PFS - defined as time from date of randomization until progression per RECIST v1.1 as assessed by the Investigator at local site, or death due to any cause.

Secondary Endpoints

Response endpoints as assessed by the investigator according to the Lugano Classification: Objective Response Rate (ORR)/Complete Response Rate (CRR)
From first dose (Cycle 1 Day 1, each cycle is 21 days) until disease progression or the last evaluable assessment in the absence of progression.
Response Endpoints as assessed by investigator according to the Lugano Classification: Conversation rate of Partial Response (PR) to Complete Response (CR)
From First dose (Cycle 1 Day 1, each cycle is 21 days) until disease progression or the last evaluable assessment in the absence of progression.
Response endpoints as assessed by the investigator according to the Lugano Classification: Duration of Response (DoR)
From date of first objective response until documented progression or death due to any cause or censoring (if progression or death have not occurred)
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
AZD3470 MonotherapyEXPERIMENTALModule 1 Cohort 1 evaluates safety, tolerability, efficacy of AZD3470 in r/r cHL participants with 2 prior lines of systemic anticancer therapy (including BV and anti-PD1). Participants will be treated according to protocol-defined windows. Part A (dose escalation) assesses AZD3470 at increasing doses in participants aged ≥18 years, r/r cHL. Part B (dose optimization/ expansion) includes participants at certain dose levels evaluated as tolerable in Part A and may include adolescent patients aged ≥12 years, upon SRC agreement. Module 1 Cohort 2 evaluates the safety, tolerability, efficacy of AZD3470 as consolidation for Stage III/IV cHL participants aged ≥50 years after CR or PR to frontline SOC therapy (either N-AVD, A-AVD, AVD, ABVD) Module 1 Cohort 3 evaluates the safety, tolerability, preliminary efficacy of AZD3470 in participants aged ≥18 years with r/r PTCL (PTCL NOS, ALCL, AITL subtypes) with at least 1 prior line of systemic anticancer therapy.
AZD3470 in combination with PembrolizumabEXPERIMENTALModule 2 Cohort 1 will assess participants aged ≥18 years with r/r cHL who have received at least one prior line of anticancer therapy. Participants will receive treatment according to the protocol-defined limit, or until disease progression, unacceptable toxicity as judged by the investigator or until meeting any other discontinuation criteria, as defined in the clinical study protocol, whichever occurs first. Part A (dose escalation) will evaluate the safety and tolerability of AZD3470 in combination with Pembrolizumab. Part B (dose optimization/expansion) will evaluate dose optimization/expansion in certain dose levels of AZD3470 in combination with Pembrolizumab, based on cumulative data from dose escalation part (Part A).
Module 1: AZD3470 MonotherapyEXPERIMENTALPart A dose escalation and back-fill cohorts and Part B dose optimization and expansion cohorts of varying doses of AZD3470
Module 2: AZD3470 in combination with Dato-DXdEXPERIMENTALVarying doses of AZD3470 in combination with Dato-Dxd
Module 2: Dato-DXd aloneEXPERIMENTALControl arm

Interventions

NameTypeDescription
AZD3470DRUGAZD3470 is a novel, potent and selective, second-generation, Methylthioadenosine (MTA)-selective, small molecule inhibitor of PRMT5.
PembrolizumabDRUGPembrolizumab (CAS nr: 1374853-91-4 )
Datopotamab deruxtecanDRUGAZD3470 in combination with Dato-DXd + Dato-Dxd monotherapy
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Eligibility Criteria

Age Range12 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites37

Inclusion Criteria: Core Inclusion criteria: 1. Adequate adult (ECOG) or adolescent (Karnofsy or Lanksy) Performance Score assessments 2. Adequate organ and bone marrow function. Module 1 Cohort 1: 1. Age: 1. Part A (dose escalation): aged ≥ 18 years at the time of signing the informed conse...

Countries:United StatesAustraliaChinaFranceGermanyItalyJapanSouth KoreaSpainUnited KingdomNetherlands
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Recent Changes (Last 90 Days)

LOWAug 27, 2026NCT06130553lastUpdatePostDate: changed
LOWAug 27, 2026NCT06130553lastUpdatePostDate: changed
LOWJul 13, 2026NCT06130553lastUpdatePostDate: changed
LOWJul 13, 2026NCT06130553lastUpdatePostDate: changed
LOWJun 24, 2026NCT06137144primaryCompletionDate: changed
LOWJun 24, 2026NCT06137144primaryCompletionDate: changed
LOWJun 11, 2026NCT06130553lastUpdatePostDate: changed
LOWJun 11, 2026NCT06130553lastUpdatePostDate: changed

Frequently asked questions about AZD3470

What is AZD3470 used for?

AZD3470 is an investigational small molecule being studied for advanced solid tumors that are MTAP deficient and for certain lymphomas, including non-Hodgkin lymphoma, Hodgkin lymphoma, and peripheral T-cell lymphoma subtypes. It is being evaluated as a monotherapy and in combination with other anticancer agents in Phase 1 clinical trials.

How does AZD3470 work?

AZD3470 is a PRMT5 inhibitor. PRMT5 is an enzyme that regulates gene expression by methylating proteins, and its inhibition is being studied as a therapeutic strategy in MTAP-deficient tumors. The drug is designed to exploit the vulnerability of cancer cells that lack the MTAP gene.

Who is developing AZD3470?

AZD3470 is being developed by AstraZeneca PLC, a biopharmaceutical company listed on the stock exchange under the ticker symbol AZN. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with advanced solid tumors and hematologic malignancies.

What phase is AZD3470 in?

AZD3470 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The ongoing trials are designed to assess the drug's safety, tolerability, and preliminary efficacy in patients with MTAP-deficient solid tumors and lymphomas.

What clinical trials is AZD3470 in?

AZD3470 is being studied in two Phase 1 trials. NCT06130553 evaluates the drug as monotherapy and in combination in patients with MTAP-deficient advanced or metastatic solid tumors, with an enrollment of 334. NCT06137144 studies it as monotherapy or with other agents in hematologic malignancies, including lymphomas, with an enrollment of 161.

Is AZD3470 the same as a PRMT5 inhibitor?

Yes, AZD3470 is a PRMT5 inhibitor. PRMT5 stands for protein arginine methyltransferase 5, an enzyme that plays a role in tumor growth. By inhibiting PRMT5, AZD3470 aims to target cancer cells that are deficient in the MTAP gene, which is a common alteration in certain tumors.