Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
PDR001 · 11 trials · 31 indications
ORR defined as the percentage of patients with a best overall response of either confirmed complete response (CR) or partial response (PR) as per local review by Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) and assessed by computed tomography (CT)/ magnetic resonance imaging (MRI). CR:Disappearance of all non-nodal target and non-target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to \< 10 mm PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters.
CBR is defined as the percentage of participants with a best overall response of Complete Response (CR), Partial Response (PR) and Stable Disease (SD). Tumor response was based on local investigator assessment. For participants with solid tumors the assessment criteria was Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) and for participants with lymphoma the assessment criteria was the Revised Response Criteria for Malignant Lymphoma (Cheson et al 2007). For RECIST v1.1, CR=Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to \< 10 mm; PR= At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters; SD= Neither sufficient shrinkage to qualify for PR or CR nor an increase in lesions which would qualify for progression. CBR (CR+PR+SD) is reported overall and by tumor type.
ORR is defined as the proportion of patients with best overall response (BOR) of complete response (CR) or partial response (PR), according to BIRC radiological assessment by RECIST 1.1. CR: disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to \< 10 mm. PR: at least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters.
Safety data.
number of patients reporting dose limiting toxicity
Number of patients with treatment-related adverse events as assessed by CTCAE v4.0
Frequency of patients requiring a dose interruption
Frequency of patients discontinuing treatment.
Frequency of patients requiring a dose reduction.
Incidence and severity of AEs and SAEs
The incidence of DLTs during the first two cycles of treatment with MBG453 in combination with PDR001 or PDR001 and/or MBG453 in combination with decitabine.
Incidence and severity of AEs and SAEs
Incidence and severity of AEs and SAEs, including changes in laboratory vital signs and ECGs
A dose-limiting toxicity (DLT) was defined as an adverse event or abnormal laboratory value assessed as unrelated to disease progression, inter-current illness, or concomitant medications that met certain criteria as defined in the protocol.
Tolerability measured by the number of subjects who have interruptions of study treatment
Tolerability measured by the number of subjects who have reductions of study treatment
Tolerability measured by the dose intensity of study treatment
During the first two cycles Cycle = 28 days
Through study completion, an average of 6 months
Through study completion, an average of 6 months
Through study completion, an average of 6 months
Through study completion, an average of 6 months
Estimated the recommended phase 2 dose (RP2D) and/or the maximum tolerated dose (MTD) for PDR001. AUC0-336h is the AUC from time zero to 336 hour post dose of a measurable concentration sampling time.
DLT is defined as an adverse event (AE) or abnormal laboratory value of common terminology criteria for adverse events (CTCAE) grade ≥ 3 assessed as unrelated to disease, disease progression, inter-current illness or concomitant medications, which occurs within the first cycle of treatment with PDR001 during the dose escalation part of the study for which relationship to study treatment cannot be ruled out, with some exceptions.
ORR is the percentage of participants with a best overall response of complete response (CR) or partial response (PR) as per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 CR = at least 2 determinations of CR at least 4 weeks apart before progression where confirmation required or 1 determination of CR prior to progression where confirmation not required. PR = at least 2 determinations of PR or better at least 4 weeks apart before progression (and not qualifying for a CR) where confirmation required or 1 determination of PR prior to progression where confirmation not required. RECIST criteria is a set of published rules that define when tumors in cancer patients improve ("respond"), stay the same ("stabilize"), or worsen ("progress") during treatment.
| Arm | Type | Description |
|---|---|---|
| Arm 1: LAG525 + PDR001 (randomized section) | EXPERIMENTAL | Participnats randomized to receive LAG525 at a dosage of 600 mg administered intravenously every 4 weeks, in combination with PDR001 at a dosage of 400 mg administered intravenously every 4 weeks |
| Arm 2: INC280+PDR001 (randomized section) | EXPERIMENTAL | Participants randomized to receive INC280 orally at a dosage of 400 mg twice daily, in combination with PDR001 intravenously at a dosage of 400 mg every 4 weeks |
| Arm 3: ACZ885 + PDR001 (randomized section) | EXPERIMENTAL | Participants randomized to receive to receive ACZ885 at a dosage of 300 mg administered subcutaneously every 4 weeks, in combination with PDR001 at a dosage of 400 mg administered intravenously every 4 weeks |
| Arm 4: LEE011 + PDR001 (randomized section) | EXPERIMENTAL | Participants randomized to receive LEE011 orally at a dosage of 600 mg once daily on Days 1-21 of a 28-day cycle, in combination with PDR001 intravenously at a dosage of 400 mg every 4 weeks |
| Arm 1A: LAG525 + PDR001 (non-randomized section) | EXPERIMENTAL | LAG-3 positive participants received LAG525 at a dosage of 600 mg administered intravenously every 4 weeks, in combination with PDR001 at a dosage of 400 mg administered intravenously every 4 weeks |
| PDR001+LAG525 | EXPERIMENTAL | PDR001 300 mg and LAG525 400 mg administered via i.v. infusion over 30 minutes once every 3 weeks (Q3W). LAG525 was given first followed by PDR001. |
| PDR001 | EXPERIMENTAL | Subjects with advanced or metastatic, well-differentiated, NET of pancreatic, GI, or thoracic origin or poorly-differentiated GEP-NEC, that have progressed on prior treatment were treated with 400mg PDR001 administered via intravenous infusion once every 4 weeks. |
| Arm A | EXPERIMENTAL | Dose escalation of single agent CJM112 |
| Arm B | EXPERIMENTAL | Dose escalation of CJM112 in combination with a fixed dose of PDR001 |
| Arm C | EXPERIMENTAL | Dose escalation of LCL161 in combination with a fixed dose of PDR001 |
| Decitabine and PDR001 | EXPERIMENTAL | Decitabine in combination with PDR001 |
| Decitabine and MBG453 | EXPERIMENTAL | Decitabine in combination with MBG453 |
| Decitabine, PDR001 and MBG453 | EXPERIMENTAL | Decitabine in combination with PDR001 and MBG453 |
| MBG453 | EXPERIMENTAL | MBG453 alone |
| MBG453 and PDR001 | EXPERIMENTAL | MBG453 in combination with PDR001 |
| Azacitidine and MBG453 | EXPERIMENTAL | Azacitidine in combination with MBG453 |
| PDR001 + Sorafenib | OTHER | PDR001 at 400 mg given intravenously every 4 weeks and sorafenib 400 mg taken orally once or twice per day (escalating doses) |
| CRC - PDR001 + LCL161 | EXPERIMENTAL | Enrollment to this combination arm is closed to further enrollment. |
| NSCLC - PDR001 + LCL161 | EXPERIMENTAL | Enrollment to this combination arm is closed to further enrollment. |
| TNBC - PDR001 + LCL161 | EXPERIMENTAL | Enrollment to this combination arm is closed to further enrollment. |
| CRC - PDR001+ Everolimus | EXPERIMENTAL | Enrollment to this combination arm is closed to further enrollment. |
| NSCLC - PDR001+ Everolimus | EXPERIMENTAL | Enrollment to this combination arm is closed to further enrollment. |
| TNBC - PDR001+ Everolimus | EXPERIMENTAL | Enrollment to this combination arm is closed to further enrollment. |
| CRC - PDR001 + Panobinostat | EXPERIMENTAL | Enrollment to this combination arm is closed to further enrollment. |
| NSCLC - PDR001 + Panobinostat | EXPERIMENTAL | Enrollment to this combination arm is closed to further enrollment. |
| TNBC - PDR001 + Panobinostat | EXPERIMENTAL | Enrollment to this combination arm is closed to further enrollment. |
| CRC - PDR001 + QBM076 | EXPERIMENTAL | Enrollment to this combination arm is closed to further enrollment. |
| TNBC - PDR001 + QBM076 | EXPERIMENTAL | Enrollment to this combination arm is closed to further enrollment. |
| NSCLC- PDR001 + QBM076 | EXPERIMENTAL | Enrollment to this combination arm is closed to further enrollment. |
| CRC - PDR001 + HDM201 | EXPERIMENTAL | Dose escalation completed, expansion arm. |
| RCC - PDR001 + HDM201 | EXPERIMENTAL | Dose escalation completed, expansion arm. |
| PDR + ACZ 100mg Q8W | EXPERIMENTAL | PDR + ACZ 100mg Q8W |
| PDR + ACZ 300mg Q8W | EXPERIMENTAL | PDR + ACZ 300mg Q8W |
| PDR + ACZ RDE TNBC | EXPERIMENTAL | PDR + ACZ Recommended Dose for Expansion (RDE) Triple Negative Breast Cancer (TNBC) |
| PDR + ACZ RDE NSCLC | EXPERIMENTAL | PDR + ACZ Recommended Dose for Expansion (RDE) Non-Small Cell Lung Cancer (NSCLC) |
| PDR + ACZ RDE CRC | EXPERIMENTAL | PDR + ACZ Recommended Dose for Expansion (RDE) Colorectal Cancer (CRC) |
| PDR + CJM 25mg Q4W | EXPERIMENTAL | PDR + CJM 25mg Q4W |
| PDR + CJM 75mg Q4W | EXPERIMENTAL | PDR + CJM 75mg Q4W |
| PDR + CJM 225mg Q4W | EXPERIMENTAL | PDR + CJM 225mg Q4W |
| PDR + CJM 450mg Q4W | EXPERIMENTAL | PDR + CJM 450mg Q4W |
| PDR + CJM 450mg Q2W | EXPERIMENTAL | PDR + CJM 450mg Q2W |
| PDR + CJM 900mg Q4W | EXPERIMENTAL | PDR + CJM 900mg Q4W |
| PDR + CJM 900mg Q2W | EXPERIMENTAL | PDR + CJM 900mg Q2W |
| PDR + CJM 1200mg Q4W | EXPERIMENTAL | PDR + CJM 1200mg Q4W |
| PDR + TMT 0.5mg QD | EXPERIMENTAL | PDR + TMT 0.5mg QD |
| PDR + TMT 1mg QD | EXPERIMENTAL | PDR + TMT 1mg QD |
| PDR + TMT 1mg QD, 3 Weeks on/1 Week off | EXPERIMENTAL | PDR + TMT 1mg QD, 3 Weeks on/1 Week off |
| PDR + TMT 1.5 mg QD, 2 Weeks on/2 Weeks off | EXPERIMENTAL | PDR + TMT 1.5 mg QD, 2 Weeks on/2 Weeks off |
| PDR + TMT 1.5 mg QD, 3 Weeks on/1 Week off | EXPERIMENTAL | PDR + TMT 1.5 mg QD, 3 Weeks on/1 Week off |
| PDR + EGF816 25mg QD | EXPERIMENTAL | PDR + EGF816 25mg QD |
| PDR + EGF816 50mg QD | EXPERIMENTAL | PDR + EGF816 50mg QD |
| s.a. ACZ RDE TNBC | EXPERIMENTAL | Single agent (s.a.) ACZ Recommended Dose for Expansion (RDE) Triple Negative Breast Cancer (TNBC) |
| s.a. ACZ RDE NSCLC | EXPERIMENTAL | Single agent (s.a.) ACZ Recommended Dose for Expansion (RDE) Non-Small Cell Lung Cancer (NSCLC) |
| s.a. ACZ RDE CRC | EXPERIMENTAL | Single agent (s.a.) ACZ Recommended Dose for Expansion (RDE) Colorectal Cancer (CRC) |
| patients with solid tumors | OTHER | Phase I Dose escalation cohorts |
| Selected tumor types | OTHER | Phase II expansion: Selected tumor types: melanoma, NSCLC, triple negative breast cancer, anaplastic thyroid cancer |
| Name | Type | Description |
|---|---|---|
| PDR001 | DRUG | 400 mg of PDR001 administered every 4 weeks intravenously |
| LAG525 | DRUG | 600 mg of LAG525 administered every 4 weeks intravenously |
| INC280 | DRUG | 400 mg of INC280 administered twice daily orally |
| ACZ885 | DRUG | 200 mg of ACZ885 administered every 4 weeks subcutaneosuly |
| LEE011 | DRUG | 600 mg of LEE011 orally taken once daily on Days 1-21 of a 28-day cycle |
| CJM112 | DRUG | Anti-IL-17A antibody |
| LCL161 | DRUG | Oral small molecule SMAC-mimetic |
| Decitabine | DRUG | Decitabine is a cytidine deoxynucleoside analogue that selectively inhibits DNA methyltransferases at low doses, resulting in gene promoter hypomethylation. |
| MBG453 | DRUG | MBG453 is a high-affinity, humanized anti-TIM-3 IgG4 monoclonal antibody which blocks the binding of TIM-3 to phosphatidylserine (PtdSer). |
| Azacitidine | DRUG | Azacitidine (5-azacytidine) is a cytidine nucleoside analogue that selectively inhibits DNA methyltransferases at low doses, resulting in gene promoter hypomethylation |
| Sorafenib | DRUG | Sorafenib is formulated as a tablet. |
| Everolimus | DRUG | - |
| Panobinostat | DRUG | - |
| QBM076 | DRUG | - |
| HDM201 | DRUG | - |
| TMT212 | DRUG | Tablets |
| EGF816 | DRUG | Tablets |
Key inclusion criteria for Arm 1, 2, 3, 4: * Histologically confirmed unresectable or metastatic stage IIIB/C/D or IV melanoma using AJCC edition 8. * Previously treated for unresectable or metastatic melanoma: * Subjects with V600BRAF wild-type disease had to have received prior systemic therap...
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PDR001 is an investigational small molecule being studied for advanced malignancies, including small cell lung cancer, well-differentiated non-functional NET of thoracic origin, melanoma, colorectal cancer, triple negative breast cancer, NSCLC adenocarcinoma, and advanced solid tumors. It is in Phase 1 clinical development.
PDR001 is being developed by Novartis AG, which trades under the ticker NVS. The drug is an investigational oncology small molecule currently in Phase 1 clinical trials.
PDR001 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still being studied in clinical trials for safety and efficacy.
PDR001 has been studied in several Phase 1 trials, including NCT02678260 in advanced malignancies, NCT02900664 in combination with other agents for colorectal cancer, triple negative breast cancer, and NSCLC adenocarcinoma, NCT02988440 with sorafenib in hepatocellular carcinoma, and NCT03066648 with decitabine in AML or high risk MDS.
PDR001 is also known as spartalizumab. It is an investigational oncology drug being developed by Novartis AG for multiple cancer types, including melanoma, colorectal cancer, and advanced solid tumors.