Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BEZ235 · 4 trials · 6 indications
PFS rate at 16 weeks was defined as a binary variable. Patients were considered as 'progression free' after 16 weeks if they had an overall lesion response of complete response (CR) partial response ('PR) or stable disease (SD)' and "progressed" if they had an overall lesion response of 'Progressive disease (PD) at the scan which occurred on day 105 after start of treatment, or later. Patients whose 16 weeks tumor assessment was unknown, missing or outside the window was not considered as 'progression free' and was considered a "failure" and counted only in the denominator for the estimation of the 16 week progression free rate.
Dose escalation part: Determine MTD and /or RDE of the combinations abiraterone acetate + BEZ235 and abiraterone acetate + BKM120 by assessing the incidence of DLTs in cycle 1
Dose expansion part: Assess anti-tumor activity of the combinations (abiraterone acetate + BEZ235 and abiraterone acetate + BKM120) in castration-resistant prostate cancer patients with abiraterone acetate failure as on treatment PSA progression according to prostate cancer working group criteria 2 (PCWG2) by assessing PSA decline ≥ 30% at Week 12 or later.
| Arm | Type | Description |
|---|---|---|
| BEZ235 300 mg/400 mg bid (Stage 1) | EXPERIMENTAL | Stage 1 consisted of a single arm where patients received BEZ235 300mg or 400mg bid. Initially the study started with a dose of 400mg bid. However, following an amendment after the preliminary safety and tolerability data from the first 3 patients treated at the 400mg dose, the dosage was changed to 300mg bid. |
| Dose escalation: BEZ235 + Zytiga® | EXPERIMENTAL | BEZ235 oral twice daily: 200 mg, 300 mg, and 400 mg dose levels to be tested in the dose escalation part in combination with abiraterone acetate Zytiga® abiraterone acetate oral once daily: 1000 mg taken with low dose prednisone as per the label |
| Dose escalation: BKM120 + Zytiga® | EXPERIMENTAL | BKM120 oral once daily: 60 mg, 80 mg and 100 mg dose levels to be tested in the dose escalation part in combination with abiraterone acetate Zytiga® abiraterone acetate oral once daily: 1000 mg taken with low dose prednisone as per the label |
| Dose expansion: BEZ235 + Zytiga® | EXPERIMENTAL | BEZ235 oral twice daily: 200 mg, 300 mg, and 400 mg dose levels to be tested in the dose escalation part in combination with abiraterone acetate Zytiga® abiraterone acetate oral once daily: 1000 mg taken with low dose prednisone as per the label |
| Dose Expansion: BKM120 + Zytiga® | EXPERIMENTAL | BKM120 oral once daily: 60 mg, 80 mg and 100 mg dose levels to be tested in the dose escalation part in combination with abiraterone acetate Zytiga® abiraterone acetate oral once daily: 1000 mg taken with low dose prednisone as per the label |
| BEZ235 Dose Escalation once daily | EXPERIMENTAL | oral BEZ235 once daily (q.d.) |
| BEZ Dose escalation twice daily | EXPERIMENTAL | oral BEZ235 twice daily (b.i.d.) |
| BEZ235 Alone, Dose Escalation | EXPERIMENTAL | - |
| BEZ235 + trastuzumab, Dose Escalation | EXPERIMENTAL | - |
| BEZ235 Alone, MTD Expansion | EXPERIMENTAL | - |
| BEZ235 + Trastuzumab, MTD Expansion | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| BEZ235 (Stage 1) | DRUG | The investigational study drug used in this trial was BEZ235, which was supplied as 50mg, 200mg, 300mg, and 400mg solid dispersion sachets. Supply as 200mg and 50mg were provided for dose reduction. Patients were instructed to take the contents of one sachet of BEZ235 twice a day in the morning within 30 minutes after a light meal (breakfast). |
| BEZ235 | DRUG | BEZ235 will be supplied as 50mg, 100mg, 200mg, 300mg and 400mg SDS sachets. At each patient's visit, patient will reecive a prescription of an adequate drug supply for self administration at home. |
| BKM120 | DRUG | BKM120 will be supplied as 10mg and 50mg hard gelatin capsules. At each patient's visit, patient will reecive a prescription of an adequate drug supply for self administration at home. |
Inclusion Criteria: * Unresectable or metastatic, histologically confirmed low or intermediate grade pancreatic neuroendocrine tumor with radiological evidence of disease progression since last treatment * Refractory disease to treatment with mTOR inhibitor * Measurable disease per RECIST Version 1...
BEZ235 is an investigational small molecule being studied in oncology for multiple tumor types, including pancreatic neuroendocrine tumors (pNET), advanced solid tumors, breast cancer, locally advanced breast cancer (LABC), castration-resistant prostate cancer, and metastatic or locally advanced solid tumors. It is not approved and remains in clinical development.
BEZ235 is being developed by Novartis AG, a global pharmaceutical company traded on the New York Stock Exchange under the ticker NVS. The drug is an investigational small molecule in the oncology therapeutic area.
BEZ235 is in Phase 1 clinical development. All completed trials for the drug are Phase 1 studies. It is investigational and has not received FDA approval, as it remains under clinical investigation for various solid tumor indications.
BEZ235 has been studied in several completed Phase 1 trials, including NCT00620594 in advanced solid malignancies and breast cancer, NCT01195376 in Japanese patients with advanced solid tumors, NCT01471847 in HER2-positive breast cancer with trastuzumab, and NCT01634061 in castration-resistant prostate cancer with abiraterone acetate.
BEZ235 is the primary name for this investigational drug. No alternative names have been established in the clinical trial data, so it is consistently referred to as BEZ235 across studies.