Trial Outcomes & Findings for A Study of BBI608 in Combination With Pemetrexed and Cisplatin in Adult Patients With Malignant Pleural Mesothelioma (NCT NCT02347917)

Last Updated: 2022-04-12

Results Overview

An AE is any untoward medical occurrence in a study subject administered an investigational drug and which does not necessarily have a causal relationship with this treatment. A SAE was an AE that met one or more of the following criteria: * Results in death * Is life-threatening * Requires hospitalization or prolongation of existing hospitalization * Results in persistent or significant disability or incapacity * Is a congenital anomaly or birth defect * Is an important medical event that may jeopardize the subject or may require a medical or surgical intervention to prevent one of the outcomes listed above. Examples of such medical events include allergic bronchospasm requiring intensive treatment in an emergency room or at home, blood dyscrasias or convulsions that do not result in inpatient hospitalization. An ADR was defined as adverse events assessed to be related to the investigational drug

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

28 participants

Primary outcome timeframe

Between initial dosing of the investigational drug and final evaluation in the follow-up observation period, about 17 months

Results posted on

2022-04-12

Participant Flow

The study conducted from February 2015 to December 2017 and a total of 28 participants were enrolled at 9 medical institutions in Japan.

This clinical study consisted of two parts: Phase 1 and Phase 2. The study proceeded to Phase 2 part after Phase 1 part demonstrated the tolerability of BBI608 combined with Pem plus CDDP based on complete assessment of DLT. Participants were NOT enrolled in Phase 1 and Phase 2 in duplicate.

Participant milestones

Participant milestones
Measure	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.
Overall Study STARTED	4	24
Overall Study Discontinued beforeBBI608 Administration	0	0
Overall Study Start BBI608 Administration	4	24
Overall Study Discontinued in DLT Evaluation Period	1	0
Overall Study Complete DLT Evaluation Period	3	0
Overall Study Discontinued After BBI608 Administration	4	24
Overall Study Discontinued Before Survival Observation	0	1
Overall Study COMPLETED	4	23
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=4 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part) n=24 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	Total n=28 Participants Total of all reporting groups
Age, Continuous	64.5 years STANDARD_DEVIATION 5.00 • n=4 Participants	65.6 years STANDARD_DEVIATION 10.71 • n=24 Participants	65.5 years STANDARD_DEVIATION 9.854 • n=28 Participants
Sex: Female, Male Female	1 Participants n=4 Participants	2 Participants n=24 Participants	3 Participants n=28 Participants
Sex: Female, Male Male	3 Participants n=4 Participants	22 Participants n=24 Participants	25 Participants n=28 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=4 Participants	0 Participants n=24 Participants	0 Participants n=28 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=4 Participants	24 Participants n=24 Participants	28 Participants n=28 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=4 Participants	0 Participants n=24 Participants	0 Participants n=28 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=4 Participants	0 Participants n=24 Participants	0 Participants n=28 Participants
Race (NIH/OMB) Asian	4 Participants n=4 Participants	24 Participants n=24 Participants	28 Participants n=28 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=4 Participants	0 Participants n=24 Participants	0 Participants n=28 Participants
Race (NIH/OMB) Black or African American	0 Participants n=4 Participants	0 Participants n=24 Participants	0 Participants n=28 Participants
Race (NIH/OMB) White	0 Participants n=4 Participants	0 Participants n=24 Participants	0 Participants n=28 Participants
Race (NIH/OMB) More than one race	0 Participants n=4 Participants	0 Participants n=24 Participants	0 Participants n=28 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=4 Participants	0 Participants n=24 Participants	0 Participants n=28 Participants
Primary cancer MPM	1 Participants n=4 Participants	24 Participants n=24 Participants	25 Participants n=28 Participants
Primary cancer NSCLC	3 Participants n=4 Participants	0 Participants n=24 Participants	3 Participants n=28 Participants
Weight	58.53 kg STANDARD_DEVIATION 10.278 • n=4 Participants	63.11 kg STANDARD_DEVIATION 7.661 • n=24 Participants	62.45 kg STANDARD_DEVIATION 8.025 • n=28 Participants
ECOG PS 0	3 Participants n=4 Participants	11 Participants n=24 Participants	14 Participants n=28 Participants
ECOG PS 1	1 Participants n=4 Participants	13 Participants n=24 Participants	14 Participants n=28 Participants
ECOG PS 2<=	0 Participants n=4 Participants	0 Participants n=24 Participants	0 Participants n=28 Participants
Disease classification MPM · STAGE I	0 Participants n=1 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants n=24 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants n=25 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Disease classification MPM · STAGE II	0 Participants n=1 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	2 Participants n=24 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	2 Participants n=25 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Disease classification MPM · STAGE III	0 Participants n=1 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	5 Participants n=24 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	5 Participants n=25 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Disease classification MPM · STAGE IV	1 Participants n=1 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	17 Participants n=24 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	18 Participants n=25 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Disease classification NSCLC · STAGE I	0 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Disease classification NSCLC · STAGE II	0 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Disease classification NSCLC · STAGE III	0 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Disease classification NSCLC · STAGE IV	3 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	3 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Tissue classification (MPM) EPITHELIOID	1 Participants n=1 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	16 Participants n=24 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	17 Participants n=25 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Tissue classification (MPM) SARCOMATOID	0 Participants n=1 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	3 Participants n=24 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	3 Participants n=25 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Tissue classification (MPM) BIPHASIC	0 Participants n=1 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	5 Participants n=24 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	5 Participants n=25 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Tissue classification (MPM) OTHER	0 Participants n=1 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants n=24 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants n=25 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Tissue classification (NSCLC) ADENOCARCINOMA	3 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	3 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Tissue classification (NSCLC) SQUAMOUS CELL CARCINOMA	0 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Tissue classification (NSCLC) LARGE CELL CARCINOMA	0 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Tissue classification (NSCLC) OTHER	0 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.	0 Participants n=3 Participants • Of 4 participants in Phase 1 part, 3 participants with NSCLC and 1 participant with MPM enrolled.
Metastases Yes	3 Participants n=4 Participants	11 Participants n=24 Participants	14 Participants n=28 Participants
Metastases No	1 Participants n=4 Participants	13 Participants n=24 Participants	14 Participants n=28 Participants
Disease duration	3.91 month STANDARD_DEVIATION 2.866 • n=4 Participants	0.91 month STANDARD_DEVIATION 0.726 • n=24 Participants	1.34 month STANDARD_DEVIATION 1.562 • n=28 Participants

PRIMARY outcome

Timeframe: Between initial dosing of the investigational drug and final evaluation in the follow-up observation period, about 17 months

Population: Safety analysis populations (Phase 1 part)

Outcome measures

Outcome measures
Measure	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=4 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including 1 Participant With MPM in Phase 1 Part) Participants orally received BBI608(480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including Participants With MPM in Phase 1 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.
Phase 1 Part: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Drug Reactions (ADRs) Any AEs	4 Participants	—	—	—	—
Phase 1 Part: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Drug Reactions (ADRs) AEs leading to death	0 Participants	—	—	—	—
Phase 1 Part: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Drug Reactions (ADRs) Serious AEs	0 Participants	—	—	—	—
Phase 1 Part: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Drug Reactions (ADRs) AEs leading to drug withdrawn (BBI608)	1 Participants	—	—	—	—
Phase 1 Part: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Drug Reactions (ADRs) AEs leading to drug interrupted (BBI608)	1 Participants	—	—	—	—
Phase 1 Part: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Drug Reactions (ADRs) AEs leading to dose reduced (BBI608)	0 Participants	—	—	—	—
Phase 1 Part: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Drug Reactions (ADRs) Any ADRs	4 Participants	—	—	—	—
Phase 1 Part: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Drug Reactions (ADRs) ADRs leading to death	0 Participants	—	—	—	—
Phase 1 Part: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Drug Reactions (ADRs) Serious ADRs	0 Participants	—	—	—	—
Phase 1 Part: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Drug Reactions (ADRs) ADRs leading to drug withdrawn (BBI608)	1 Participants	—	—	—	—
Phase 1 Part: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Drug Reactions (ADRs) ADRs leading to drug interrupted (BBI608)	1 Participants	—	—	—	—
Phase 1 Part: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Drug Reactions (ADRs) ADRs leading to dose reduced (BBI608)	0 Participants	—	—	—	—

PRIMARY outcome

Timeframe: From Day 1 of Cycle 1 to Day 24 pre-dose examination (23 days)

Population: DLT evaluable population (3 participants with NSCLC in Phase 1 part) 1 participant with MPM was excluded from DLT evaluable population because the BBI608 treatment compliance rate during DLT evaluation period did not meet the rate of 80% which of assessable for DLT and no adverse events assessed as DLT occurred.

DLT was defined as an adverse event meeting any of the following that occurred during the DLT evaluation period in any participants given BBI608 with the causal relationship to BBI608 assessed as "Definite," "Probable," or "Possible." The severity of adverse events was graded according to the CTCAE v4.0-JCOG. * Grade 4 neutropenia persisting for ≥ 7 days * Grade ≥ 3 febrile neutropenia persisting for ≥ 5 days * Grade 3 thrombocytopenia requiring platelet transfusions, grade 4 thrombocytopenia * Grade ≥ 3 non-hematotoxicity except the following: 1. Inappetence, nausea, vomiting and electrolyte abnormality which, within 3 days of onset, improved to grade ≤ 2 or resolved after appropriate treatment 2. Diarrhoea and fatigue which, within 5 days of onset, improved to grade ≤ 2 or resolved after appropriate treatment * Other clinically significant signs in the opinion of the investigator

Outcome measures

Outcome measures
Measure	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=3 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including 1 Participant With MPM in Phase 1 Part) Participants orally received BBI608(480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including Participants With MPM in Phase 1 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.
Phase 1 Part: Number of Participants With Dose-limiting Toxicities (DLTs)	0 Participants	—	—	—	—

PRIMARY outcome

Timeframe: Cycle 1 Day 1 (Cmax only) and Day 23

Population: Pharmacokinetic (PK) analysis population 1 participant with MPM of 4 participants in the pharmacokinetic analysis population completed BBI608 administration on Day 14 in Cycle 1 and therefore was not included in the analysis for Cmax and AUC on Day 23.

Outcome measures

Outcome measures
Measure	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=4 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including 1 Participant With MPM in Phase 1 Part) Participants orally received BBI608(480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including Participants With MPM in Phase 1 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.
Phase 1 Part: Maximum Observed Concentration (Cmax) and Minimum Observed Concentration (Cmin) of BBI608 When Administered With Pem and CDDP Day 1: Cmax	307.5 ng/mL Interval 251.0 to 380.0	—	—	—	—
Phase 1 Part: Maximum Observed Concentration (Cmax) and Minimum Observed Concentration (Cmin) of BBI608 When Administered With Pem and CDDP Day 23: Cmax	473.7 ng/mL Interval 316.0 to 643.0	—	—	—	—
Phase 1 Part: Maximum Observed Concentration (Cmax) and Minimum Observed Concentration (Cmin) of BBI608 When Administered With Pem and CDDP Day 23: Cmin	196.1 ng/mL Interval 30.4 to 392.0	—	—	—	—

PRIMARY outcome

Timeframe: Cycle 1 Day 1 and Day 23

AUC0-12: Area under the concentration-time curve from time zero to 12 hours, AUC0-24: Area under the concentration-time curve from time zero to 24 hours, AUC0-inf: Area under the concentration-time curve from time zero to infinity

Outcome measures

Outcome measures
Measure	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=4 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including 1 Participant With MPM in Phase 1 Part) Participants orally received BBI608(480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including Participants With MPM in Phase 1 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.
Phase 1 Part: Area Under the Concentration-time Curve Day 1: AUC0-24	2223.8 h*ng/mL Interval 1005.0 to 3060.0	—	—	—	—
Phase 1 Part: Area Under the Concentration-time Curve Day 1: AUC0-12	1814.3 h*ng/mL Interval 900.0 to 2443.0	—	—	—	—
Phase 1 Part: Area Under the Concentration-time Curve Day 1: AUC0-inf	2413.5 h*ng/mL Interval 1141.0 to 3536.0	—	—	—	—
Phase 1 Part: Area Under the Concentration-time Curve Day 23: AUC0-12	3797.0 h*ng/mL Interval 1788.0 to 5725.0	—	—	—	—
Phase 1 Part: Area Under the Concentration-time Curve Day 23: AUC0-24	6103.7 h*ng/mL Interval 1936.0 to 9948.0	—	—	—	—
Phase 1 Part: Area Under the Concentration-time Curve Day 23: AUC0-inf	10762.7 h*ng/mL Interval 1962.0 to 20834.0	—	—	—	—

PRIMARY outcome

Timeframe: From BBI608 administration to documented PD or death, whichever is earlier, about 17 months

Population: modified ITT population. Efficacy was analyzed using the efficacy analysis population (i.e., modified ITT population; mITT), separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25).

PFS was defined as the time from BBI608 administration to documented PD (as assessed according to the mRECIST or RECIST 1.1) or death, whichever is earlier. The result of imaging assessment by the imaging assessment committee was used for phase 2 part.

Outcome measures

Outcome measures
Measure	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=24 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including 1 Participant With MPM in Phase 1 Part) n=25 Participants Participants orally received BBI608(480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including Participants With MPM in Phase 1 Part) Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.
Phase 2 Part: Progression-free Survival (PFS)	5.59 month Interval 2.89 to Not reached	5.59 month Interval 2.89 to Not reached	—	—	—

SECONDARY outcome

Timeframe: Every 6 weeks from the first dose of BBI608 until Week 30, and every 9 weeks from Week 31.

The best overall response is the best response recorded from the start of the study treatment until the end of treatment. The RECIST 1.1 was used for the evaluation of tumor response and overall response in patients with NSCLC, and also the evaluation of any non-pleural lesions in patients with MPM. The mRECIST was used for the evaluation of tumor response and overall response in patients with MPM. The result of imaging assessment by study site was used for phase 1 part, and the result of imaging assessment by the imaging assessment committee was used for phase 2 part.

Outcome measures

Outcome measures
Measure	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=3 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including 1 Participant With MPM in Phase 1 Part) n=1 Participants Participants orally received BBI608(480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=4 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part) n=24 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including Participants With MPM in Phase 1 Part) n=25 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.
Best Overall Response Stable disease (SD)	3 Participants	0 Participants	3 Participants	11 Participants	11 Participants
Best Overall Response Progressive disease (PD)	0 Participants	0 Participants	0 Participants	2 Participants	2 Participants
Best Overall Response Not evaluated (NE)	0 Participants	1 Participants	1 Participants	3 Participants	4 Participants
Best Overall Response Complete response (CR)	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Best Overall Response Partial response (PR)	0 Participants	0 Participants	0 Participants	8 Participants	8 Participants

SECONDARY outcome

Timeframe: From BBI608 administration to death from any cause, about 17 months

Response rate (RR): Proportion of subjects whose best overall response is CR or PR. Disease control rate (DCR): Proportion of subjects whose best overall response is CR, PR or SD. The result of imaging assessment by study site was used for phase 1 part, and the result of imaging assessment by the imaging assessment committee was used for phase 2 part.

Outcome measures

Outcome measures
Measure	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=3 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including 1 Participant With MPM in Phase 1 Part) n=1 Participants Participants orally received BBI608(480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=4 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part) n=24 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including Participants With MPM in Phase 1 Part) n=25 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.
Response Rate (RR) and Disease Control Rate (DCR) Response rate (RR)	0 Participants	0 Participants	0 Participants	8 Participants	8 Participants
Response Rate (RR) and Disease Control Rate (DCR) Disease control rate (DCR)	3 Participants	0 Participants	3 Participants	19 Participants	19 Participants

SECONDARY outcome

Timeframe: From BBI608 administration to death from any cause, up to 31 months

OS was defined as the time from BBI608 administration to death from any cause. Participants alive at final observation or lost to follow-up were censored at their last contact (i.e., visit or telephone) date.

Outcome measures

Outcome measures
Measure	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=3 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including 1 Participant With MPM in Phase 1 Part) n=1 Participants Participants orally received BBI608(480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=4 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part) n=24 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including Participants With MPM in Phase 1 Part) n=25 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.
Overall Survival(OS)	19.81 month Interval 19.4 to 27.8	30.1 month Interval 30.1 to 30.1	NA month Interval 19.4 to 30.1 Not calculated for total population in Phase 1 part because calculated for each cancer type (i.e., NSCLC and MPM separately)	12.14 month Interval 3.0 to 28.6	12.75 month Interval 3.0 to 30.1

SECONDARY outcome

Timeframe: Every 6 weeks from the first dose of BBI608 until Week 30, and then every 9 weeks from Week 31 [Actually up to Week 111]

Outcome measures

Outcome measures
Measure	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=3 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including 1 Participant With MPM in Phase 1 Part) Participants orally received BBI608(480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=4 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part) n=24 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including Participants With MPM in Phase 1 Part) n=25 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 6 weeks	70.0 mL Interval -10.0 to 200.0	—	70.0 mL Interval -10.0 to 200.0	30.0 mL Interval -470.0 to 410.0	30.0 mL Interval -470.0 to 410.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 12 weeks	110.0 mL Interval -90.0 to 140.0	—	110.0 mL Interval -90.0 to 140.0	0.0 mL Interval -370.0 to 940.0	0.0 mL Interval -370.0 to 940.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 18 weeks	-60.0 mL Interval -70.0 to 90.0	—	-60.0 mL Interval -70.0 to 90.0	-70.0 mL Interval -700.0 to 1250.0	-70.0 mL Interval -700.0 to 1250.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 24 weeks	-290.0 mL Interval -630.0 to 50.0	—	-290.0 mL Interval -630.0 to 50.0	-50.0 mL Interval -570.0 to 1480.0	-50.0 mL Interval -570.0 to 1480.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 30 weeks	-240.0 mL Interval -510.0 to 30.0	—	-240.0 mL Interval -510.0 to 30.0	20.0 mL Interval -530.0 to 1410.0	20.0 mL Interval -530.0 to 1410.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 39 weeks	-305.0 mL Interval -600.0 to -10.0	—	-305.0 mL Interval -600.0 to -10.0	30.0 mL Interval -660.0 to 870.0	30.0 mL Interval -660.0 to 870.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 48 weeks	NA mL Interval -480.0 to -480.0 because of 1 participant data only	—	NA mL Interval -480.0 to -480.0 because of 1 participant data only	15.0 mL Interval -100.0 to 910.0	15.0 mL Interval -100.0 to 910.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 57 weeks	NA mL Interval -820.0 to -820.0 because of 1 participant data only	—	NA mL Interval -820.0 to -820.0 because of 1 participant data only	-20.0 mL Interval -70.0 to 1090.0	-20.0 mL Interval -70.0 to 1090.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 66 weeks	NA mL Interval -990.0 to -990.0 because of 1 participant data only	—	NA mL Interval -990.0 to -990.0 because of 1 participant data only	300.0 mL Interval -110.0 to 710.0	300.0 mL Interval -110.0 to 710.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 75 weeks	—	—	—	175.0 mL Interval -400.0 to 750.0	175.0 mL Interval -400.0 to 750.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 84 weeks	—	—	—	145.0 mL Interval -580.0 to 870.0	145.0 mL Interval -580.0 to 870.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 93 weeks	—	—	—	-205.0 mL Interval -890.0 to 480.0	-205.0 mL Interval -890.0 to 480.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 102 weeks	—	—	—	NA mL Interval 480.0 to 480.0 because of 1 participant data only	NA mL Interval 480.0 to 480.0 because of 1 participant data only
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in VC: 111 weeks	—	—	—	NA mL Interval 280.0 to 280.0 because of 1 participant data only	NA mL Interval 280.0 to 280.0 because of 1 participant data only
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 6 weeks	0.0 mL Interval -50.0 to 120.0	—	0.0 mL Interval -50.0 to 120.0	35.0 mL Interval -380.0 to 390.0	35.0 mL Interval -380.0 to 390.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 12 weeks	80.0 mL Interval -110.0 to 90.0	—	80.0 mL Interval -110.0 to 90.0	10.0 mL Interval -400.0 to 1030.0	10.0 mL Interval -400.0 to 1030.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 18 weeks	-20.0 mL Interval -110.0 to 0.0	—	-20.0 mL Interval -110.0 to 0.0	-140.0 mL Interval -860.0 to 1280.0	-140.0 mL Interval -860.0 to 1280.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 24 weeks	-155.0 mL Interval -390.0 to 80.0	—	-155.0 mL Interval -390.0 to 80.0	-50.0 mL Interval -610.0 to 1520.0	-50.0 mL Interval -610.0 to 1520.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 30 weeks	-265.0 mL Interval -570.0 to 40.0	—	-265.0 mL Interval -570.0 to 40.0	150.0 mL Interval -430.0 to 1450.0	150.0 mL Interval -430.0 to 1450.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 39 weeks	-345.0 mL Interval -530.0 to -160.0	—	-345.0 mL Interval -530.0 to -160.0	-35.0 mL Interval -530.0 to 770.0	-35.0 mL Interval -530.0 to 770.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 48 weeks	NA mL Interval -480.0 to -480.0 because of 1 participant data only	—	NA mL Interval -480.0 to -480.0 because of 1 participant data only	70.0 mL Interval -160.0 to 800.0	70.0 mL Interval -160.0 to 800.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 57 weeks	NA mL Interval -800.0 to -800.0 because of 1 participant data only	—	NA mL Interval -800.0 to -800.0 because of 1 participant data only	60.0 mL Interval -70.0 to 880.0	60.0 mL Interval -70.0 to 880.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 66 weeks	NA mL Interval -880.0 to -880.0 because of 1 participant data only	—	NA mL Interval -880.0 to -880.0 because of 1 participant data only	300.0 mL Interval -140.0 to 740.0	300.0 mL Interval -140.0 to 740.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 75 weeks	—	—	—	50.0 mL Interval -550.0 to 650.0	50.0 mL Interval -550.0 to 650.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 84 weeks	—	—	—	10.0 mL Interval -580.0 to 600.0	10.0 mL Interval -580.0 to 600.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 93 weeks	—	—	—	-230.0 mL Interval -900.0 to 440.0	-230.0 mL Interval -900.0 to 440.0
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 102 weeks	—	—	—	NA mL Interval 540.0 to 540.0 because of 1 participant data only	NA mL Interval 540.0 to 540.0 because of 1 participant data only
Respiratory Function Tests (Vital Capacity [VC] and Forced Vital Capacity [FVC]) The change from baseline in FVC: 111 weeks	—	—	—	NA mL Interval 490.0 to 490.0 because of 1 participant data only	NA mL Interval 490.0 to 490.0 because of 1 participant data only

SECONDARY outcome

Timeframe: Every 6 weeks from the first dose of BBI608 until Week 30, and then every 9 weeks from Week 31 [Actually up to Week 111]

Outcome measures

Outcome measures
Measure	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=3 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including 1 Participant With MPM in Phase 1 Part) Participants orally received BBI608(480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=4 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part) n=24 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including Participants With MPM in Phase 1 Part) n=25 Participants Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 6 weeks	0.160 L Interval -0.01 to 0.16	—	0.160 L Interval -0.01 to 0.16	0.050 L Interval -0.4 to 0.33	0.050 L Interval -0.4 to 0.33
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 12 weeks	0.070 L Interval 0.03 to 0.18	—	0.070 L Interval 0.03 to 0.18	-0.130 L Interval -0.33 to 0.52	-0.130 L Interval -0.33 to 0.52
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 18 weeks	0.000 L Interval -0.13 to 0.16	—	0.000 L Interval -0.13 to 0.16	-0.110 L Interval -0.56 to 0.63	-0.110 L Interval -0.56 to 0.63
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 24 weeks	-0.010 L Interval -0.16 to 0.14	—	-0.010 L Interval -0.16 to 0.14	-0.040 L Interval -0.45 to 0.78	-0.040 L Interval -0.45 to 0.78
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 30 weeks	-0.055 L Interval -0.23 to 0.12	—	-0.055 L Interval -0.23 to 0.12	-0.030 L Interval -0.29 to 0.71	-0.030 L Interval -0.29 to 0.71
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 39 weeks	-0.140 L Interval -0.28 to 0.0	—	-0.140 L Interval -0.28 to 0.0	-0.050 L Interval -0.37 to 0.26	-0.050 L Interval -0.37 to 0.26
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 48 weeks	NA L Interval -0.06 to -0.06 because of 1 participant data only	—	NA L Interval -0.06 to -0.06 because of 1 participant data only	0.115 L Interval -0.12 to 0.39	0.115 L Interval -0.12 to 0.39
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 57 weeks	NA L Interval -0.46 to -0.46 because of 1 participant data only	—	NA L Interval -0.46 to -0.46 because of 1 participant data only	0.010 L Interval -0.07 to 0.31	0.010 L Interval -0.07 to 0.31
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 66 weeks	NA L Interval -0.57 to -0.57 because of 1 participant data only	—	NA L Interval -0.57 to -0.57 because of 1 participant data only	0.085 L Interval -0.16 to 0.33	0.085 L Interval -0.16 to 0.33
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 75 weeks	—	—	—	-0.045 L Interval -0.42 to 0.33	-0.045 L Interval -0.42 to 0.33
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 84 weeks	—	—	—	-0.130 L Interval -0.44 to 0.18	-0.130 L Interval -0.44 to 0.18
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 93 weeks	—	—	—	-0.215 L Interval -0.66 to 0.23	-0.215 L Interval -0.66 to 0.23
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 102 weeks	—	—	—	NA L Interval 0.15 to 0.15 because of 1 participant data only	NA L Interval 0.15 to 0.15 because of 1 participant data only
Respiratory Function Tests (Forced Expiratory Volume in the First Second [FEV1]) The change from baseline in FEV1: 111 weeks	—	—	—	NA L Interval 0.08 to 0.08 because of 1 participant data only	NA L Interval 0.08 to 0.08 because of 1 participant data only

Adverse Events

MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part)

Serious events: 0 serious events

Other events: 4 other events

Deaths: 2 deaths

MPM: BBI608 + Pem + CDDP (Phase 2 Part)

Serious events: 7 serious events

Other events: 24 other events

Deaths: 19 deaths

MPM: BBI608 + Pem + CDDP (Phase 2 Part Including 1 Participant With MPM in Phase 1 Part)

Serious events: 7 serious events

Other events: 25 other events

Deaths: 21 deaths

Serious adverse events

Serious adverse events
Measure	MPM or NSCLC: BBI608 + Pem + CDDP (Phase 1 Part) n=4 participants at risk Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part) n=24 participants at risk Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.	MPM: BBI608 + Pem + CDDP (Phase 2 Part Including 1 Participant With MPM in Phase 1 Part) n=25 participants at risk Participants orally received BBI608 (480 mg) twice daily plus intravenously received Pem (500 mg/m2) and CDDP (75 mg/m2) on Day 3 in only Cycles 1 and on Days 1 in Cycles 2 and subsequent cycles. Premeditation with folic acid and vitamin B12 was given to reduce occurrence of serious adverse drug reactions, with reference to the package insert for Pem. Each cycle was defined as 21 days.
Gastrointestinal disorders Colitis	0.00% 0/4 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.2% 1/24 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.0% 1/25 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]
Gastrointestinal disorders Enterocolitis	0.00% 0/4 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.2% 1/24 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.0% 1/25 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]
Gastrointestinal disorders Gastric ulcer	0.00% 0/4 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.2% 1/24 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.0% 1/25 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]
Infections and infestations Bronchopneumonia	0.00% 0/4 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.2% 1/24 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.0% 1/25 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]
Infections and infestations Sepsis	0.00% 0/4 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.2% 1/24 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.0% 1/25 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]
Investigations Platelet count decreased	0.00% 0/4 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.2% 1/24 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.0% 1/25 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neuroendocrine carcinoma	0.00% 0/4 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.2% 1/24 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.0% 1/25 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]
Psychiatric disorders Delirium	0.00% 0/4 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.2% 1/24 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.0% 1/25 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]
Vascular disorders Thrombosis	0.00% 0/4 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.2% 1/24 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]	4.0% 1/25 • Number of events 1 • During a period from initial dosing of investigational drug to final evaluation in the follow-up observation period. up to 31 months for deaths, up to 11 months for Serious and Other (Not Including Serious) Adverse Events Safety analysis population \[separately for Phase 1 part (n=4), Phase 2 part (n=24) and Phase 2 part including 1 participant with MPM in Phase 1 part (n=25)\]

Other adverse events

Additional Information

Clinical Research (Oncology)

Sumitomo Dainippon Pharmaceutical

Phone: e-mail only

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place