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AZD9291

Phase 3

Locally Advanced or Metastatic EGFR T790M+ NSCLC | Small molecule | Oncology |AstraZeneca PLC|Last Updated: Apr 20, 2026

Target and mechanism

Molecular targetEGFR
Target classInhibitor
ModalitySmall molecule

Also known as Osimertinib, osimertinib

Success Probability

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Market & Valuation

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Trial Design

RandomizedCONTROLLED
Total Trials1
Total Enrollment29

FDA Designations

PRIORITY_REVIEW

Clinical trial landscape

AZD9291 · 11 trials · 9 indications

Phase 3 2Phase 2 2Phase 1 7
NCT02511106AZD9291 Versus Placebo in Patients With Stage IB-IIIA Non-small Cell Lung Carcinoma, Following Complete Tumour Resection With or Without Adjuvant Chemotherapy.Stage IB-IIIA Non-small Cell Lung Carcinoma
ACTIVE NOT_RECRUITING682 Analytics
NCT02454933Study of AZD9291 Plus MEDI4736 Versus AZD9291 Monotherapy in NSCLC After Previous EGFR TKI Therapy in T790M Mutation Positive TumoursLocally Advanced or Metastatic EGFR T790M+ NSCLC
COMPLETED29 Analytics
PHASE3ACTIVE NOT_RECRUITING
AZD9291 Versus Placebo in Patients With Stage IB-IIIA Non-small Cell Lung Carcinoma, Following Complete Tumour Resection With or Without Adjuvant Chemotherapy.
Stage IB-IIIA Non-small Cell Lung CarcinomaUnlock trial analytics
PHASE3COMPLETED
Study of AZD9291 Plus MEDI4736 Versus AZD9291 Monotherapy in NSCLC After Previous EGFR TKI Therapy in T790M Mutation Positive Tumours
Locally Advanced or Metastatic EGFR T790M+ NSCLCUnlock trial analytics

Study Endpoints

Primary Endpoints

Assess the Efficacy of AZD9291 Compared to Placebo as Measured by Disease Free Survival (DFS).
Up to approximately 5 years after the first patient is randomized (maximum follow up of 70 months)

Defined as the time from the date of randomization until the date of disease recurrence or death (by any cause in the absence of recurrence).

Number of Subjects With Adverse Events (AEs) as a Measure of the Safety and Tolerability of Osimertinib in Combination With Durvalumab
From Baseline up to primary analysis data cut-off (up to 24 months).

As a measure of the safety and tolerability of osimertinib in combination with durvalumab the number of subjects who experienced any treatment emergent AE (TEAE), any causally related AE, any serious AE (SAE), and any causally related SAE are presented.

Confirmed Complete Response (CR) Rate
Up to 2 years

To assess the efficacy of various sequences. CR (per RECIST 1.1 as assessed by the local/site Investigator) is defined as the disappearance of all target and non-target lesions. Confirmed complete response rate (CR rate) is defined as the number (%) of patients with a confirmed overall response of CR and was based on the evaluable analysis set.

Objective Response Rate (ORR)
RECIST tumour assessments every 6 weeks from first dose until objective disease progression, up to approximately 11 months (at the time of analysis)

Per Response Evaluation Criteria in Solid Tumours (RECIST v1.1) assessed by MRI or CT: Complete Response (CR): Disappearance of all target and non-target lesions and no new lesions; Partial Response (PR): \>= 30% decrease in the sum of diameters of Target Lesions (compared to baseline) and no new lesions. ORR is the percentage of patients with at least 1 visit response of CR or PR (according to independent review) that was confirmed at least 4 weeks later, prior to progression or further anti-cancer therapy.

Cmax of AZD9291 After Single Dosing
PK blood samples are collected at pre-dose, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 48, 72 and 120 hours post-dose. Results are based on data cut off of 28 Jan 2016.

Pharmacokinetics of AZD9291 after single dosing by assessment of maximum plasma AZD9291 concentration

Cmax of AZ5104 After Single Dosing
PK blood samples are collected at pre-dose, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 48, 72 and 120 hours post-dose. Results are based on data cut off of 28 Jan 2016.

Pharmacokinetics of AZD9291 metabolites (AZ5104) after single dosing by assessment of maximum plasma concentration

Cmax of AZ7550 After Single Dosing
PK blood samples are collected at pre-dose, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 48, 72 and 120 hours post-dose. Results are based on data cut off of 28 Jan 2016.

Pharmacokinetics of AZD9291 metabolites (AZ7550) after single dosing by assessment of maximum plasma concentration

AUC of AZD9291 After Single Dosing
PK blood samples are collected at pre-dose, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 48, 72 and 120 hours post-dose. Results are based on data cut off of 28 Jan 2016.

Pharmacokinetics of AZD9291 after single dosing by assessment of area under the plasma concentration time curve from zero to infinity

AUC of AZ5104 After Single Dosing
PK blood samples are collected at pre-dose, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 48, 72 and 120 hours post-dose. Results are based on data cut off of 28 Jan 2016.

Pharmacokinetics of AZD9291 metabolites (AZ5104) after single dosing by assessment of area under the plasma concentration time curve from zero to infinity

AUC of AZ7550 After Single Dosing
PK blood samples are collected at pre-dose, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 48, 72 and 120 hours post-dose. Results are based on data cut off of 28 Jan 2016.

Pharmacokinetics of AZD9291 metabolites (AZ7550) after single dosing by assessment of area under the plasma concentration time curve from zero to infinity

CL/F of AZD9291 After Single Dosing
PK blood samples are collected at pre-dose, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 48, 72 and 120 hours post-dose. Results are based on data cut off of 28 Jan 2016.

Rate and extent of absorption of single dose AZD9291 by assessment of apparent clearance following oral administration

C(ss, Max) of AZD9291 After Multiple Dosing
PK blood samples are collected multiple times on Cycle 1 Day 8 and Cycle 2 Day 1. Results are based on data cut off of 28 Jan 2016.

Pharmacokinetics of AZD9291 after multiple dosing by assessment of maximum plasma concentration at steady state

C(ss, Max) of AZ5104 After Multiple Dosing
PK blood samples are collected multiple times on Cycle 1 Day 8 and Cycle 2 Day 1. Results are based on data cut off of 28 Jan 2016.

Pharmacokinetics of AZD9291 metabolites (AZ5104) after multiple dosing by assessment of maximum plasma concentration at steady state

C(ss, Max) of AZ7550 After Multiple Dosing
PK blood samples are collected multiple times on Cycle 1 Day 8 and Cycle 2 Day 1. Results are based on data cut off of 28 Jan 2016.

Pharmacokinetics of AZD9291 metabolites (AZ7550) after multiple dosing by assessment of maximum plasma concentration at steady state

AUC(ss) of AZD9291 After Multiple Dosing
PK blood samples are collected multiple times on Cycle 1 Day 8 and Cycle 2 Day 1. Results are based on data cut off of 28 Jan 2016.

Pharmacokinetics of AZD9291 after multiple dosing by assessment of area under the plasma concentration curve from time zero to the end of the dosing interval

AUC(ss) of AZ5104 After Multiple Dosing
PK blood samples are collected multiple times on Cycle 1 Day 8 and Cycle 2 Day 1. Results are based on data cut off of 28 Jan 2016.

Pharmacokinetics of AZD9291 metabolites (AZ5104) after multiple dosing by assessment of area under the plasma concentration curve from time zero to the end of the dosing interval

AUC(ss) of AZ7550 After Multiple Dosing
PK blood samples are collected multiple times on Cycle 1 Day 8 and Cycle 2 Day 1. Results are based on data cut off of 28 Jan 2016.

Pharmacokinetics of AZD9291 metabolites (AZ7550) after multiple dosing by assessment of area under the plasma concentration curve from time zero to the end of the dosing interval

CL(ss)/F of AZD9291 After Multiple Dosing
PK blood samples are collected multiple times on Cycle 1 Day 8 and Cycle 2 Day 1. Results are based on data cut off of 28 Jan 2016.

Pharmacokinetics of AZD9291 after multiple dosing by assessment of apparent plasma clearance at steady state

Absolute Oral Bioavailability
Samples taken at pre-dose, 1, 2, 3, 4, 5:45, 5:52, 6, 6:05, 6:10, 6:20, 6:25, 6:30, 7, 8, 9, 10, 12, 14, 16, 18, 24, 30, 48, 72, 120, 168, 216, 336 and 504 hours relative to the oral dose.

Absolute bioavailability of AZD9291 will be calculated from area under the plasma concentration versus time curve (AUC) of the oral dose of AZD9291 / AUC of the IV dose of \[14C\]AZD9291 x IV dose/Oral dose x 100

AUC(0-72) of AZD9291
Blood samples collected on Day 1 and Day 10 at pre-dose, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 48 and 72 hours post AZD9291 dose in Part A.

Pharmacokinetics of AZD9291 by assessment of area under the plasma concentration time curve from zero to 72 hours.

Cmax of AZD9291
Blood samples collected on Day 1 and Day 10 at pre-dose, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 48, 72, 120, 168, and 216 hours post AZD9291 dose in Part A.

Pharmacokinetics of AZD9291 by assessment of maximum plasma AZD9291 concentration.

AUC of AZD9291
Blood samples collected on Day 1 and Day 10 at pre-dose, 1, 2, 3, 4, 6, 8, 10, 12, 24, 48, 72, 96, 120, 168, and 216 hours post AZD9291 dose in Part A.

Pharmacokinetics of AZD9291 by assessment of area under the plasma concentration time curve from zero to infinity

Percentage of Radioactive Dose of [14C] Radiolabelled AZD9291 Recovered in Urine, Faeces, and in Total.
Blood samples (hrs) - 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 18, 24, 36, 48, every 24 to 168, 336, 504, 648, 816, 904 and 1992. Urine (h): 0-4, 4-8, 8-12, 12-24, every 24 hrs to 504, 648-72, 816-40, 984-1008 and 1992-2016. Faeces: 0-24h and then as per urine.

The percentage of radioactive dose of \[14C\] radiolabelled AZD9291 recovered in urine, faeces, and in total, up to Day 85.

Pharmacokinetics of AZD9291 and its metabolites, by assessment of maximum plasma concentration (Cmax)
Blood samples are collected pre dose, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 48, 72, 120, 168, 216, 336, 504 hours post dose

Curve taken during each of the 5 treatments

Objective Response Rate (ORR) for Dose Expansion Population
RECIST tumour assessments every 6 weeks from randomisation until objective disease progression, up to approximately 21 months (at time of analysis)

Per Response Evaluation Criteria in Solid Tumours (RECIST v1.1) assessed by MRI or CT: Complete Response (CR): Disappearance of all target and non-target lesions and no new lesions; Partial Response (PR): \>= 30% decrease in the sum of diameters of Target Lesions (compared to baseline) and no new lesions. ORR is the percentage of patients with at least 1 visit response of CR or PR (by investigator assessment) that was confirmed at least 4 weeks later, prior to progression or further anti-cancer therapy.

Best Objective Response (BOR) for Dose Escalation Population
RECIST tumour assessments every 6 weeks from randomisation until objective disease progression, up to approximately 25 months (at time of analysis)

Per Response Evaluation Criteria in Solid Tumours (RECIST v1.1) assessed by MRI or CT: Complete Response (CR): Disappearance of all target and non-target lesions and no new lesions; Partial Response (PR): \>= 30% decrease in the sum of diameters of Target Lesions (compared to baseline) and no new lesions; Stable disease (SD): Neither sufficient shrinkage to qualify as a response nor sufficient growth to qualify as progression; Progressive Disease (PD): \>= 20% increase in the sum of diameters of TLs and an absolute increase in sum of diameters of \>=5mm (compared to the previous minimum sum) or progression of NTLs or a new lesion. Not evaluable (NE): TL response is missing and there is no evidence of progression of NTLs and no new lesions. BOR is the best response (by investigator assessment) a patient has achieved where the order of best to worst is CR, PR, SD, PD, NE prior to or at progression and prior to further anti-cancer therapy.

Objective Response Rate (ORR) for Extension Population
RECIST tumour assessments every 6 weeks from randomisation until objective disease progression, up to approximately 12 months (at the time of analysis)

Per Response Evaluation Criteria in Solid Tumours (RECIST v1.1) assessed by MRI or CT: Complete Response (CR): Disappearance of all target and non-target lesions and no new lesions; Partial Response (PR): \>= 30% decrease in the sum of diameters of Target Lesions (compared to baseline) and no new lesions. ORR is the percentage of patients with at least 1 visit response of CR or PR (by independent central review) that was confirmed at least 4 weeks later, prior to progression or further anti-cancer therapy.

Secondary Endpoints

Disease Free Survival (DFS) Rate at 2, 3 and 5 Years
Up to approximately 5 years after the first patient is randomized (maximum follow up of 70 months). DFS rate at 2 years (%), 3 years (%) and 5 years (%) are presented.
Overall Survival (OS)
Up to approximately 7 years after the first patient is randomized (maximum follow up of 86 months)
Overall Survival Rate at 2, 3 and 5 Years
Up to approximately 7 years after the first patient is randomized (maximum follow up of 86 months). OS rate at 2 years (%), 3 years (%) and 5 years (%) are presented.
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Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
AZD9291EXPERIMENTALAZD9291 (80 mg or 40 mg orally, once daily), in accordance with the randomization schedule.
Placebo AZD9291PLACEBO_COMPARATORMatching placebo for AZD9291 (80 mg or 40 mg orally, once daily), in accordance with the randomization schedule.
MEDI4736 & AZD9291 CombinationEXPERIMENTAL10mg/kg q2w (IV) infusion \& once daily tablet 80 mg
AZD9291 MonotherapyEXPERIMENTALOnce daily tablet 80 mg
Gefitinib with a Seq. Switch to a MEDI4736EXPERIMENTALGefitinib once daily followed by MEDI4736
AZD9291 with a Seq. Switch to a MEDI4736EXPERIMENTALAZD9291 once daily followed by MEDI4736
Selumetinib+Docetaxel with a Seq. Switch to a MEDI4736EXPERIMENTALSelumetinib twice daily + docetaxel, followed by MEDI4736
Tremelimumab with a Seq. Switch to a MEDI4736EXPERIMENTALTremelimumab every 4 weeks followed by MEDI4736
AZD9291 40 mgEXPERIMENTALCohort 1: 40 mg once daily
AZD9291 80 mgEXPERIMENTALCohort 2: 80 mg once daily
Bioavailability of AZD9291EXPERIMENTALTo assess the absolute bioavailability of a single oral dose of AZD9291 with respect to an intravenous microdose of \[14C\]AZD9291
FastedOTHERAZD9291 tablets following a period of fasting
High-fat mealOTHERAZD9291 tablets following a high-fat meal.
AZD9291 alone, AZD9291+itraconozoleEXPERIMENTALSequential treatments of AZD9291 alone followed by AZD9291+itraconazole, with a washout period in between.
[14C]-AZD9291 20mg (oral solution)EXPERIMENTALVolunteers will receive 20 mg \[14C\]-AZD9291 containing a nominal 1 μCi activity, administered by mouth, as a solution.
AZD9291 20mg (oral capsule fasted)EXPERIMENTALVolunteers will receive AZD9291 20mg administered by mouth, as a capsule, in the fasted state.
AZD9291 20mg (oral solution fasted)EXPERIMENTALVolunteers will receive AZD9291 20mg administered by mouth, as a solution, in the fasted state.
AZD9291 20mg (oral tablet fasted)EXPERIMENTALVolunteers will receive AZD9291 20mg administered by mouth, as a tablet, in the fasted state.
AZD9291 20mg (oral fasted)EXPERIMENTALVolunteers will receive AZD9291 20mg administered by mouth, as a capsule or tablet in the fasted state.
AZD9291 20mg (oral fed)EXPERIMENTALVolunteers will receive AZD9291 20mg administered by mouth, as a capsule or tablet in the fed state.
Daily dose of AZD9291EXPERIMENTALDaily oral dose of AZD9291

Interventions

NameTypeDescription
AZD9291 80 mg/40 mgDRUGThe initial dose of AZD9291 80 mg once daily can be reduced to 40 mg once daily.
Placebo AZD9291 80 mg/40 mgDRUGThe initial dose of Placebo AZD9291 80 mg once daily can be reduced to 40 mg once daily.
Open-label AZD9291 80 mg/40 mgDRUGEligible patients will be offered open-label osimertinib upon recurrence and in the absence of intervening systemic anti-cancer therapy.
AZD9291DRUGOnce daily tablet 80 mg
MEDI4736DRUG10mg/kg q2w (IV) infusion
GefitinibDRUGGefitinib once daily followed by MEDI4736
Selumetinib+DocetaxelDRUGSelumetinib twice daily + docetaxel, followed by MEDI4736
TremelimumabDRUGTremelimumab every 4 weeks followed by MEDI4736
AZD9291 40 mgDRUGThis is a two parts (Part A and Part B) study to determine the pharmacokinetics of AZD9291 administered orally at two dose levels (40 mg Cohort 1 and 80 mg Cohort 2) in patients with locally advanced or metastatic NSCLC who have progressed following prior therapy with an EGFR TKI agent (+/- additional chemotherapy regimens).
AZD9291 80 mgDRUGThis is a two parts (Part A and Part B) study to determine the pharmacokinetics of AZD9291 administered orally at two dose levels (40 mg Cohort 1 and 80 mg Cohort 2) in patients with locally advanced or metastatic NSCLC who have progressed following prior therapy with an EGFR TKI agent (+/- additional chemotherapy regimens).
[14C]AZD9291DRUGEach healthy male subject will also receive a single, radiolabeled, 100 μg dose of \[14C\] AZD9291 administered as an IV microdose infusion starting at 5 hours and 45 minutes after receiving the oral dose.
AZD9291 tabletsDRUGAZD9291 tablets: Part A 80mg od, days 1 and 10 only. Part B 80mg od for 12 months.
Pharmacokinetic sampling - AZD9291PROCEDUREBlood samples taken pre and post dosing of AZD9291 following either a period of fasting or consumption of a meal.
Dietary FastedOTHERFasted from 10 hours prior to dosing with 80mg AZD9291 tablet and 4 hours after dosing
Dietary High FatOTHERAllocated breakfast prior to dosing with 80mg AZD9291 tablet
Pharmacokinetic sampling - AZ5140 and AZ7550PROCEDUREBlood samples taken pre and post dosing of AZD9291 following either a period of fasting or consumption of a meal.
Pharmacokinetic samplingPROCEDUREBlood samples taken pre and post dosing with AZD9291+/- itraconazole
ItraconazoleDRUGItraconazole tablets: 2x100mg bd, Part A days 6 to 19 only
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Eligibility Criteria

Age Range18 Years to 130 Years
SexALL
Healthy VolunteersNo
Study Sites238

Inclusion Criteria: 1. Male or female, aged at least 18 years. 2. Histologically confirmed diagnosis of primary non small lung cancer (NSCLC) on predominantly non-squamous histology 3. MRI or CT scan of the brain must be done prior to surgery as it is considered standard of care. 4. Patients must b...

Countries:United StatesAustraliaBelgiumBrazilCanadaChinaFranceGermanyHong KongHungaryIsraelItalyJapanNetherlandsPolandRomaniaRussiaSouth KoreaSpainSwedenTaiwanThailandTurkey (Türkiye)UkraineVietnamUnited Kingdom
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Frequently asked questions about AZD9291

What is AZD9291 used for?

AZD9291, also known as osimertinib, is an investigational small molecule being studied for the treatment of locally advanced or metastatic EGFR T790M+ non-small cell lung cancer (NSCLC), as well as other stages of NSCLC including stage II-IIIB and stage IV disease. It is being evaluated in patients with EGFR mutation positive locally advanced or metastatic NSCLC.

What does AZD9291 target?

AZD9291 is a kinase inhibitor, belonging to the -tinib class of drugs. It targets and inhibits kinase enzymes involved in cancer cell growth and survival. Specifically, it is being studied in EGFR T790M mutation positive NSCLC, indicating its activity against the EGFR kinase with the T790M resistance mutation.

Who makes AZD9291?

AZD9291 is being developed by AstraZeneca PLC, a biopharmaceutical company listed on the stock exchange under the ticker symbol AZN. The drug is also known by its generic name osimertinib and is currently in clinical development for the treatment of non-small cell lung cancer.

What phase is AZD9291 in?

AZD9291 is in Phase 1 clinical development, with a total of 9 trials, of which 7 are active and 3 are completed. The trials include Phase 1, Phase 2, and Phase 3 studies, with a total enrollment of 2420 participants. The drug has received FDA priority review designation.

What clinical trials is AZD9291 in?

AZD9291 is being studied in several clinical trials, including NCT02491944, a completed Phase 1 bioavailability study; NCT02856893, a completed Phase 2 study in EGFR T790M plasma positive NSCLC patients; NCT03521154, an active Phase 3 study in stage III unresectable NSCLC; and NCT05526755, an active Phase 2 study of adjuvant therapy in resected EGFRm NSCLC.

Is AZD9291 the same as osimertinib?

Yes, AZD9291 is the same as osimertinib. The drug is known by multiple names, including osimertinib, Osimertinib 80 mg/40 mg, and Osimertinib + Savolitinib. These names refer to the same investigational drug being developed by AstraZeneca for the treatment of EGFR mutation positive non-small cell lung cancer.