Trial Outcomes & Findings for A Phase III, Safety, Tolerability and Efficacy of Combination Treatment of BL-8040 and Granulocyte Colony Stimulating Factor (G-CSF) as Compared to Placebo and G-CSF for the Mobilization of Hematopoietic Stem Cells for Autologous Transplantation in Subjects With Multiple Myeloma (MM) (NCT NCT03246529)

Last Updated: 2026-01-15

Results Overview

Percentage of subjects mobilizing ≥6 × 10\^6 CD34+ cells/kg with up to 2 apheresis sessions in preparation for autologous hematopoetic cell transplantation (auto-HCT) after treatment with G-CSF + single administration of BL-8040/placebo. Based on central laboratory data.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE3

Target enrollment

180 participants

Primary outcome timeframe

From first day of study treatment (G-CSF) until day of second apheresis which was planned to occur on Day 6

Results posted on

2026-01-15

Participant Flow

A total of 180 subjects signed the Informed Consent Form (ICF) and screened to the study. A total of 136 subjects received at least one dose of G-CSF. A total of 134 subjects were treated with G-CSF and with BL-8040 or Placebo.

Participant milestones

Participant milestones
Measure	BL-8040 + G-CSF Part 1 Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	BL-8040 + G-CSF Part 2 Part 2: randomized, double-blinded, placebo controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Placebo + G-CSF Part 2 Part 2: randomized, double-blinded, placebo controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Core Study (until 100 d post-transplant) STARTED	12	80	42
Core Study (until 100 d post-transplant) Post Transplantation Analysis Set	12	77	37
Core Study (until 100 d post-transplant) COMPLETED	12	74	34
Core Study (until 100 d post-transplant) NOT COMPLETED	0	6	8
Follow-up period at 1 year FU Completion STARTED	12	73	34
Follow-up period at 1 year FU Completion COMPLETED	9	66	32
Follow-up period at 1 year FU Completion NOT COMPLETED	3	7	2

Reasons for withdrawal

Reasons for withdrawal
Measure	BL-8040 + G-CSF Part 1 Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	BL-8040 + G-CSF Part 2 Part 2: randomized, double-blinded, placebo controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Placebo + G-CSF Part 2 Part 2: randomized, double-blinded, placebo controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Core Study (until 100 d post-transplant) Adverse Event	0	2	0
Core Study (until 100 d post-transplant) Lost to Follow-up	0	1	1
Core Study (until 100 d post-transplant) Other Reasons	0	2	2
Core Study (until 100 d post-transplant) Physician Decision	0	0	1
Core Study (until 100 d post-transplant) Withdrawal by Subject	0	1	4
Follow-up period at 1 year FU Completion Disease progression	2	1	1
Follow-up period at 1 year FU Completion Lost to Follow-up	1	3	0
Follow-up period at 1 year FU Completion Withdrawal by Subject	0	3	0
Follow-up period at 1 year FU Completion Death	0	0	1

Baseline Characteristics

A Phase III, Safety, Tolerability and Efficacy of Combination Treatment of BL-8040 and Granulocyte Colony Stimulating Factor (G-CSF) as Compared to Placebo and G-CSF for the Mobilization of Hematopoietic Stem Cells for Autologous Transplantation in Subjects With Multiple Myeloma (MM)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Total n=134 Participants Total of all reporting groups	BL-8040 + G-CSF Part 1 n=12 Participants Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	BL-8040 + G-CSF Part 2 n=80 Participants Part 2: randomized, double-blinded, placebo controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Placebo + G-CSF Part 2 n=42 Participants Part 2: randomized, double-blinded, placebo controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Age, Continuous	60.5 years STANDARD_DEVIATION 9.1 • n=78 Participants	63.3 years STANDARD_DEVIATION 4.8 • n=14 Participants	60.4 years STANDARD_DEVIATION 9.4 • n=10 Participants	59.2 years STANDARD_DEVIATION 9.6 • n=24 Participants
Sex: Female, Male Female	48 Participants n=78 Participants	5 Participants n=14 Participants	25 Participants n=10 Participants	18 Participants n=24 Participants
Sex: Female, Male Male	86 Participants n=78 Participants	7 Participants n=14 Participants	55 Participants n=10 Participants	24 Participants n=24 Participants
Race/Ethnicity, Customized African American	12 Participants n=78 Participants	2 Participants n=14 Participants	8 Participants n=10 Participants	2 Participants n=24 Participants
Race/Ethnicity, Customized Caucasian	114 Participants n=78 Participants	9 Participants n=14 Participants	65 Participants n=10 Participants	40 Participants n=24 Participants
Race/Ethnicity, Customized Not reported	1 Participants n=78 Participants	1 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants
Race/Ethnicity, Customized African	1 Participants n=78 Participants	0 Participants n=14 Participants	1 Participants n=10 Participants	0 Participants n=24 Participants
Race/Ethnicity, Customized Asian	2 Participants n=78 Participants	0 Participants n=14 Participants	2 Participants n=10 Participants	0 Participants n=24 Participants
Race/Ethnicity, Customized Hispanic origin	1 Participants n=78 Participants	0 Participants n=14 Participants	1 Participants n=10 Participants	0 Participants n=24 Participants
Race/Ethnicity, Customized Unknown	2 Participants n=78 Participants	0 Participants n=14 Participants	2 Participants n=10 Participants	0 Participants n=24 Participants
Race/Ethnicity, Customized Not Specified	1 Participants n=78 Participants	0 Participants n=14 Participants	1 Participants n=10 Participants	0 Participants n=24 Participants
Region of Enrollment Hungary	16 Participants n=78 Participants	0 Participants n=14 Participants	10 Participants n=10 Participants	6 Participants n=24 Participants
Region of Enrollment United States	90 Participants n=78 Participants	12 Participants n=14 Participants	52 Participants n=10 Participants	26 Participants n=24 Participants
Region of Enrollment Italy	17 Participants n=78 Participants	0 Participants n=14 Participants	9 Participants n=10 Participants	8 Participants n=24 Participants
Region of Enrollment Germany	3 Participants n=78 Participants	0 Participants n=14 Participants	3 Participants n=10 Participants	0 Participants n=24 Participants
Region of Enrollment Spain	8 Participants n=78 Participants	0 Participants n=14 Participants	6 Participants n=10 Participants	2 Participants n=24 Participants
Treatment Indicated for Multiple Myeloma Before Study Initiation Bortezomib	89 Participants n=78 Participants	7 Participants n=14 Participants	54 Participants n=10 Participants	28 Participants n=24 Participants
Treatment Indicated for Multiple Myeloma Before Study Initiation Cisplatin	1 Participants n=78 Participants	0 Participants n=14 Participants	0 Participants n=10 Participants	1 Participants n=24 Participants
Treatment Indicated for Multiple Myeloma Before Study Initiation Cyclophosphamide	9 Participants n=78 Participants	0 Participants n=14 Participants	5 Participants n=10 Participants	4 Participants n=24 Participants
Treatment Indicated for Multiple Myeloma Before Study Initiation Daratumumab 0 - 1 2.4%	1 Participants n=78 Participants	0 Participants n=14 Participants	0 Participants n=10 Participants	1 Participants n=24 Participants
Treatment Indicated for Multiple Myeloma Before Study Initiation Doxorubicin	1 Participants n=78 Participants	0 Participants n=14 Participants	0 Participants n=10 Participants	1 Participants n=24 Participants
Treatment Indicated for Multiple Myeloma Before Study Initiation Etoposide	1 Participants n=78 Participants	0 Participants n=14 Participants	0 Participants n=10 Participants	1 Participants n=24 Participants
Treatment Indicated for Multiple Myeloma Before Study Initiation Ixazomib	1 Participants n=78 Participants	0 Participants n=14 Participants	1 Participants n=10 Participants	0 Participants n=24 Participants
Treatment Indicated for Multiple Myeloma Before Study Initiation Kyprolis	9 Participants n=78 Participants	6 Participants n=14 Participants	2 Participants n=10 Participants	1 Participants n=24 Participants
Treatment Indicated for Multiple Myeloma Before Study Initiation Lenalidomide	97 Participants n=78 Participants	12 Participants n=14 Participants	57 Participants n=10 Participants	28 Participants n=24 Participants
Treatment Indicated for Multiple Myeloma Before Study Initiation Melphalan	1 Participants n=78 Participants	0 Participants n=14 Participants	0 Participants n=10 Participants	1 Participants n=24 Participants
Treatment Indicated for Multiple Myeloma Before Study Initiation Pomalidomide	2 Participants n=78 Participants	1 Participants n=14 Participants	0 Participants n=10 Participants	1 Participants n=24 Participants
Treatment Indicated for Multiple Myeloma Before Study Initiation Thalidomide	30 Participants n=78 Participants	0 Participants n=14 Participants	18 Participants n=10 Participants	12 Participants n=24 Participants
IMWG Classification at Screening PR (Partial Response)	41 Participants n=78 Participants	0 Participants n=14 Participants	31 Participants n=10 Participants	10 Participants n=24 Participants
IMWG Classification at Screening VGPR (Very Good Partial Response)	65 Participants n=78 Participants	9 Participants n=14 Participants	33 Participants n=10 Participants	23 Participants n=24 Participants
IMWG Classification at Screening CR (Complete Response)	21 Participants n=78 Participants	2 Participants n=14 Participants	12 Participants n=10 Participants	7 Participants n=24 Participants
IMWG Classification at Screening sCR (Stringent Complete Response)	7 Participants n=78 Participants	1 Participants n=14 Participants	4 Participants n=10 Participants	2 Participants n=24 Participants
Months from MM Diagnosis to Study Consent	6.3 months STANDARD_DEVIATION 9.4 • n=78 Participants	4.6 months STANDARD_DEVIATION 0.9 • n=14 Participants	6.5 months STANDARD_DEVIATION 11.1 • n=10 Participants	6.4 months STANDARD_DEVIATION 6.9 • n=24 Participants

PRIMARY outcome

Timeframe: From first day of study treatment (G-CSF) until day of second apheresis which was planned to occur on Day 6

Population: Full Analysis Set (FAS) for Part 1 (Part 1 of the study assessed the mobilization using local lab only), Intention-to-Treat (ITT) for Part 2

Outcome measures

Outcome measures
Measure	BL-8040 + G-CSF Part 1 n=12 Participants Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	BL-8040 + G-CSF Part 2 n=80 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4. First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Placebo + G-CSF Part 2 n=42 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Percentage of Subjects Mobilizing ≥6 × 10^6 CD34+ Cells/kg With up to 2 Apheresis Sessions Central Lab	—	70.0 Percentage of responders	14.3 Percentage of responders
Percentage of Subjects Mobilizing ≥6 × 10^6 CD34+ Cells/kg With up to 2 Apheresis Sessions Local Lab	91.7 Percentage of responders	92.5 Percentage of responders	26.2 Percentage of responders

SECONDARY outcome

Timeframe: From first day of study treatment (G-CSF) until day of first apheresis which was planned to occur on Day 5

Population: FAS for Part 1 (Part 1 of the study assessed the mobilization using local lab only), ITT for Part 2

Percentage of subjects mobilizing ≥2 × 10\^6 CD34+ cells/kg in 1 apheresis session after treatment with G-CSF + single administration of BL-8040/ placebo.

Outcome measures

Outcome measures
Measure	BL-8040 + G-CSF Part 1 n=12 Participants Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	BL-8040 + G-CSF Part 2 n=80 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4. First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Placebo + G-CSF Part 2 n=42 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Percentage of Subjects Mobilizing ≥2 × 10^6 CD34+ Cells/kg in 1 Apheresis Session Central Lab	—	87.5 Percentage of responders	47.6 Percentage of responders
Percentage of Subjects Mobilizing ≥2 × 10^6 CD34+ Cells/kg in 1 Apheresis Session Local Lab	100 Percentage of responders	96.3 Percentage of responders	64.3 Percentage of responders

SECONDARY outcome

Timeframe: From first day of study treatment (G-CSF) until day of first apheresis which was planned to occur on Day 5

Population: FAS for Part 1 (Part 1 of the study assessed the mobilization using local lab only), ITT for Part 2

Percentage of subjects mobilizing ≥6 × 10\^6 CD34+ cells/kg in 1 apheresis session after treatment with G-CSF + single administration of BL-8040/ placebo.

Outcome measures

Outcome measures
Measure	BL-8040 + G-CSF Part 1 n=12 Participants Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	BL-8040 + G-CSF Part 2 n=80 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4. First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Placebo + G-CSF Part 2 n=42 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Percentage of Subjects Mobilizing ≥6 × 10^6 CD34+ Cells/kg in 1 Apheresis Session Central Lab	—	67.5 Percentage of responders	4.8 Percentage of responders
Percentage of Subjects Mobilizing ≥6 × 10^6 CD34+ Cells/kg in 1 Apheresis Session Local Lab	75 Percentage of responders	88.8 Percentage of responders	9.5 Percentage of responders

SECONDARY outcome

Timeframe: End of engraftment period, which was defined as 29 days post transplantation

Population: Post Transplantation analysis set (all subjects treated, with at least 1 apheresis session, and who underwent transplantation)

Time to neutrophil engraftment after auto-HCT, where engraftment was defined as absolute neutrophil count (ANC) ≥0.5 × 10\^9/L for 3 days or ≥1.0 × 10\^9/L for 1 day following the conditioning regimen associated nadir.

Outcome measures

Outcome measures
Measure	BL-8040 + G-CSF Part 1 n=12 Participants Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	BL-8040 + G-CSF Part 2 n=77 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4. First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Placebo + G-CSF Part 2 n=37 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Time to Neutrophil Engraftment, After Auto-HCT	12 Days Interval 11.0 to 12.0	12 Days Interval 11.0 to 12.0	12 Days Interval 11.0 to 12.0

SECONDARY outcome

Timeframe: End of engraftment period, which was defined as 29 days post transplantation

Population: Post Transplantation analysis set (all subjects treated, with at least 1 apheresis session, and who underwent transplantation)

Time to platelet engraftment, after auto-HCT, where engraftment was defined as the first of 3 consecutive measurements of platelet count ≥20 × 10\^9/L without platelet transfusion support for 7 days following the conditioning regimen associated nadir.

Outcome measures

Outcome measures
Measure	BL-8040 + G-CSF Part 1 n=12 Participants Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	BL-8040 + G-CSF Part 2 n=77 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4. First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Placebo + G-CSF Part 2 n=37 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Time to Platelet Engraftment, After Auto-HCT	17 Point estimate (days) Interval 15.0 to 20.0	18 Point estimate (days) Interval 17.0 to 19.0	17 Point estimate (days) Interval 17.0 to 18.0

SECONDARY outcome

Timeframe: Day 100 Post-Transplantation (± 7 days)

Population: Post Transplantation analysis set (all randomized subjects treated, with at least 1 apheresis session, and who underwent transplantation). Graft durability was not assessed during Part 1 of the study.

Subjects achieving graft durability were defined as meeting the following 2 criteria: * Platelet count ≥50 × 10\^9/L without transfusion for at least 2 weeks. * Hemoglobin level ≥10 g/dL with no erythropoietin support or transfusions for at least 1 month. This analysis was performed in part 2 of the study only.

Outcome measures

Outcome measures
Measure	BL-8040 + G-CSF Part 1 n=77 Participants Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	BL-8040 + G-CSF Part 2 n=37 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4. First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Placebo + G-CSF Part 2 Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Subjects With Graft Durability at 100 Days Post Transplant/ Early Termination	92.2 Percentage of responders	91.9 Percentage of responders	—

SECONDARY outcome

Timeframe: 6 Months Post Transplantation

Population: Post Transplantation Analysis Set (all randomized subjects treated, with at least 1 apheresis session, and who underwent transplantation). Graft durability was not assessed during Part 1 of the study.

Comparability of the graft durability between the BL-8040 + G-CSF arm and the placebo + G-CSF arm at 6 months post transplantation

Outcome measures

Outcome measures
Measure	BL-8040 + G-CSF Part 1 n=77 Participants Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	BL-8040 + G-CSF Part 2 n=37 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4. First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Placebo + G-CSF Part 2 Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Graft Durability at 6 Months Post Transplantation	80.5 Percentage of responders	83.8 Percentage of responders	—

SECONDARY outcome

Timeframe: 9 Months Post Transplantation

Comparability of the graft durability between the BL-8040 + G-CSF arm and the placebo + G-CSF arm at 9 months post transplantation

Outcome measures

Outcome measures
Measure	BL-8040 + G-CSF Part 1 n=77 Participants Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	BL-8040 + G-CSF Part 2 n=37 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4. First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Placebo + G-CSF Part 2 Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Graft Durability at 9 Months Post Transplantation	83.1 Percentage of responders	81.1 Percentage of responders	—

SECONDARY outcome

Timeframe: 12 Month Post Transplantation

Comparability of the graft durability between the BL-8040 + G-CSF arm and the placebo + G-CSF arm at 12 months post transplantation

Outcome measures

Outcome measures
Measure	BL-8040 + G-CSF Part 1 n=77 Participants Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	BL-8040 + G-CSF Part 2 n=37 Participants Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4. First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Placebo + G-CSF Part 2 Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Graft Durability at 12 Months Post Transplantation	81.8 Percentage of responders	81.1 Percentage of responders	—

OTHER_PRE_SPECIFIED outcome

Timeframe: End of Study

Comparability between the effect of BL-8040 + G-CSF and placebo + G-CSF on Overall Survival until September 2028

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: End Of Study

Comparability between the effect of BL-8040 + G-CSF and placebo + G-CSF on Relapse Free Survival until September 2028

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: End of Study

Comparability between the effect of BL-8040 + G-CSF and placebo + G-CSF on Annualized Relapse Rate until September 2028

Outcome measures

Outcome data not reported

Adverse Events

BL-8040 + G-CSF Part 1

Serious events: 1 serious events

Other events: 12 other events

Deaths: 0 deaths

BL-8040 + G-CSF (Part 2)

Serious events: 7 serious events

Other events: 77 other events

Deaths: 2 deaths

Placebo + G-CSF (Part 2)

Serious events: 0 serious events

Other events: 35 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	BL-8040 + G-CSF Part 1 n=12 participants at risk Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	BL-8040 + G-CSF (Part 2) n=80 participants at risk Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Placebo + G-CSF (Part 2) n=42 participants at risk Part 2 (randomized, double-blinded, placebo-controlled period). G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
General disorders Injection site reaction	0.00% 0/12 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	1.2% 1/80 • Number of events 1 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	0.00% 0/42 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.
Immune system disorders Hypersensitivity	0.00% 0/12 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	1.2% 1/80 • Number of events 1 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	0.00% 0/42 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.
Infections and infestations Bacteremia	0.00% 0/12 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	1.2% 1/80 • Number of events 1 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	0.00% 0/42 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.
Infections and infestations Injection site cellulitis	0.00% 0/12 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	1.2% 1/80 • Number of events 1 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	0.00% 0/42 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.
Investigations Platelet count decreased	0.00% 0/12 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	1.2% 1/80 • Number of events 1 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	0.00% 0/42 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Bladder neoplasm	0.00% 0/12 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	1.2% 1/80 • Number of events 1 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	0.00% 0/42 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.
Respiratory, thoracic and mediastinal disorders Hypoxia	0.00% 0/12 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	1.2% 1/80 • Number of events 1 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	0.00% 0/42 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.
Gastrointestinal disorders Hypokalaemia	8.3% 1/12 • Number of events 1 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	0.00% 0/80 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	0.00% 0/42 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.
Gastrointestinal disorders Vomiting	8.3% 1/12 • Number of events 1 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	0.00% 0/80 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.	0.00% 0/42 • All Adverse Events (AEs) were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.

Other adverse events

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