Section 4 of 6

Clinical evidence

116 evidence topics · 27 sources

Study summaries

IMPALA-2

Objective

Evaluation of efficacy and safety

“Evaluate the efficacy and safety of molgramostim for the treatment of aPAP patients” (opens the source at this quote in a new tab)

Location and study date

Geographic participation and open-label data cutoff

Attribute	Reported value
Clinical sites	“43”
Countries	“16”
Data cutoff for the reported open-label analysis	“July 3, 2025”

Protocol version date

“09 October 2024” (opens the source at this quote in a new tab)

Study design

Randomized double-blind trial

“The IMPALA-2 trial, a 48-week Phase 3 randomized, double-blind, placebo-controlled trial of MOLBREEVI* in 164 patients with autoimmune PAP” (opens the source at this quote in a new tab)

Randomization and treatment-period durations

Design component	Reported value
Randomization ratio, molgramostim to placebo	“1:1”
Double-blind treatment duration, weeks	“48”
Subsequent open-label treatment duration, weeks	“96”

Eligibility criteria

Autoantibody confirmation and impaired pulmonary gas transfer

“A serum anti-GM-CSF autoantibody test result confirming autoimmune PAP.” (opens the source at this quote in a new tab)
“A diffusing capacity for carbon monoxide of 70% predicted or lower adjusted for hemoglobin (%DLCOadj)” (opens the source at this quote in a new tab)

Stability of pulmonary gas transfer during screening

Screening requirement	Reported threshold
Change in DLCO percent predicted during screening	“<15 percentage points”

Treatment

Investigational molgramostim dose

“300 µg in 1.2 mL, daily” (opens the source at this quote in a new tab)

Randomized treatment and open-label crossover

Treatment component	Reported value
Double-blind active treatment	“molgramostim 300 µg”
Double-blind comparator	“matching placebo”
Administration frequency	“once daily”
Inhalation device	“eFlow® Nebulizer System, PARI”

Study outcomes

Rationale for using hemoglobin-adjusted DLCO percent predicted as the primary endpoint

Primary endpoint definition

“Change in % predicted DLCO from baseline to Week 24” (opens the source at this quote in a new tab)

Clinical rationale for measuring pulmonary gas transfer

“it is a standardized measure of pulmonary gas transfer widely used in clinical practice.” (opens the source at this quote in a new tab)

Historical limitations of outcome-measure validation

“2022” (opens the source at this quote in a new tab)
“no outcome measures have been validated for use as clinical trial endpoints in patients with aPAP” (opens the source at this quote in a new tab)

Subsequent post-hoc correlations with respiratory quality of life

Correlation between change in DLCO percent predicted and change in SGRQ Total Score	Reported result
Week 24	“r=-0.46, P<0.0001; n=144”
Week 48	“r=-0.46, P<0.0001; n=142”

Planned health economics and quality-of-life assessments

“Health Economics and Quality of Life” (opens the source at this quote in a new tab)
“EuroQoL 5 Dimensions, 5 Levels (EQ-5D-5L)” (opens the source at this quote in a new tab)

Statistical analysis description

Number of participants (Planned and analyzed)

Planned sample size and statistical power

Planning parameter	Reported value
Planned enrollment	“160”
Power	“90%”
Assumed between-group difference in DLCO percent predicted, percentage points	“5.7”
Assumed standard deviation, percentage points	“11”

Randomized population

Population	Reported number
Total randomized	“164”
Molgramostim	“81”
Placebo	“83”

Description of analysis sets

Summary

The primary efficacy population includes all randomized participants, analyzed by assigned treatment. Safety analyses use participants who received at least one dose, analyzed by treatment received. The plan specifies a mixed model for repeated measures and control-based imputation for missing efficacy data. Safety analyses do not impute missing data. Multiplicity procedures differ between Japan and South Korea and the other participating regions; a nominal P-value below 0.05 therefore does not independently establish significance for every secondary endpoint.

Analysis populations and statistical methods

Component	Quoted specification
Full analysis set	“all randomized subjects”
Safety analysis set: minimum exposure	“at least one dose”
Repeated-measures analysis	“MMRM”
Multiplicity procedure	“Hochberg”

Procedures for handling missing data

“missing endpoint data will be imputed using control-based imputation” (opens the source at this quote in a new tab)
“There will be no missing data imputation for safety analyses.” (opens the source at this quote in a new tab)

Results

Participant disposition

Double-blind treatment completion

Treatment group	Completed the 48-week double-blind period on treatment
Molgramostim	“98%”
Placebo	“96%”

Entry into and withdrawal from the open-label period

Disposition measure	Reported value
Completed double-blind treatment and continued into open-label treatment	“160 (98%)”
Continued molgramostim after randomized molgramostim	“79”
Crossed over to molgramostim after randomized placebo	“81”
Discontinued treatment and withdrew during open-label follow-up through July 3, 2025	“9”

Baseline characteristics

Demographic and pulmonary-function characteristics

Characteristic	Molgramostim, 81 participants	Placebo, 83 participants
Age, years, mean and standard deviation	“50.8 (13.0)”	“48.4 (12.7)”
Male, number and percentage	“44 (54.3)”	“54 (65.1)”
Female, number and percentage	“37 (45.7)”	“29 (34.9)”
DLCO percent predicted, mean and standard deviation	“52.6 (11.7)”	“52.6 (10.4)”

Efficacy results

Primary endpoint: pulmonary gas transfer at week 24

“change from baseline” (opens the source at this quote in a new tab)
“DLco% at Week 24 compared with placebo (9.8% vs. 3.8%; estimated treatment difference, 6.0%; P<0.001 by comparison of least-squares means [LSMs]).” (opens the source at this quote in a new tab)

Pulmonary gas transfer at week 48

Measure	Molgramostim	Placebo	Estimated treatment difference	P-value
Least-squares mean change from baseline in DLCO percent predicted	“11.6%”	“4.7%”	“6.9%”	“P<0.001”

Number of participants with at least one whole lung lavage procedure over 48 weeks

“Molgramostim (n=81)”	“Placebo (n=83)”
“6 (7.4%)”	“11 (13.3%)”

“6 patients (7.4%) in the molgramostim group underwent a total of 15 WLLs and 11 patients (13.3%) in the placebo group underwent a total of 24 WLLs (Figure 5) (P=NS)” (opens the source at this quote in a new tab)

Exploratory treadmill exercise capacity: distance walked

“Distance Walked” (opens the source at this quote in a new tab)
“At Week 48” (opens the source at this quote in a new tab)
“The ETD was 80.6 m (95% CI, 7.9–153.2; P=0.0301)” (opens the source at this quote in a new tab)

Post-hoc biomarker analyses

“CYFRA21-1, KL-6, and LDH” (opens the source at this quote in a new tab)
“Levels of biomarkers associated with aPAP disease severity decreased more in patients treated with molgramostim compared with placebo” (opens the source at this quote in a new tab)

Open-label pulmonary gas transfer through week 96

“MOL-MOL patients continued to show improvement in DLco% through Week 96” (opens the source at this quote in a new tab)
“overall mean increase from baseline (Weeks 0-96) of 14.7 (1.6)” (opens the source at this quote in a new tab)

Health-related quality of life and patient-reported outcomes

SGRQ Total Score: randomized treatment-period changes

Assessment	Molgramostim change from baseline, points	Placebo change from baseline, points	Estimated difference, points
Week 24	“-11.5”	“-4.9”	“-6.6”
Week 48	“-10.7”	“-5.9”	“-4.9”

SGRQ secondary endpoints and multiplicity

“SGRQ Total Score”	“SGRQ Activity Score”
“Week 24”	“Week 24”
“P=0.0072*”	“P=0.0149**”

“P-value ≤0.05, but not statistically significant due to prespecified testing hierarchy.” (opens the source at this quote in a new tab)

Inferential limitation after the SGRQ Activity comparison

“so no statistical inference was drawn with respect to subsequent secondary end points.” (opens the source at this quote in a new tab)

Open-label SGRQ Total Score through week 96

Open-label population and assessment	Mean change and standard error
Continued molgramostim: baseline to week 96	“-15.0 (2.6)”
Placebo crossover to molgramostim: week 48 to week 96	“-6.5 (1.6)”

EQ-5D-5L descriptive system: exploratory results

“Odds ratios of responses on the EQ-5D-5L descriptive system questionnaire numerically favored the molgramostim group on 3 of the 5 dimensions” (opens the source at this quote in a new tab)

Safety results

Double-blind treatment-period safety

“Symptomatic COVID-19 infection (22% molgramostim vs. 10% placebo)” (opens the source at this quote in a new tab)
“Worsening of aPAP (5% molgramostim vs. 14% placebo)” (opens the source at this quote in a new tab)
“Diarrhea (11% molgramostim vs. 2% placebo)” (opens the source at this quote in a new tab)
“No deaths occurred” (opens the source at this quote in a new tab)

Open-label treatment-period adverse events

Event category, number and percentage	Continued molgramostim, 79 participants	Placebo crossover to molgramostim, 81 participants
Any adverse event	“72 (91)”	“70 (86)”
Serious adverse events	“17 (22)”	“23 (28)”
Treatment-related serious adverse events	“1 (1)”	“0”
Adverse events leading to death	“1 (1)”	“2 (2)”
Adverse events leading to drug discontinuation	“1 (1)”	“3 (4)”

“All deaths were assessed as not related to treatment.” (opens the source at this quote in a new tab)

Study limitations

Summary

The randomized comparison involved 164 participants and 48 weeks of blinded treatment. The primary endpoint measures pulmonary gas transfer rather than survival or hospitalization. Post-hoc correlations with SGRQ support clinical relevance but do not establish that DLCO is a validated surrogate for every patient-important outcome. The SGRQ Activity comparison did not meet the prespecified multiplicity requirements, and subsequent secondary or exploratory results should not be treated as independent confirmatory findings.

The week-96 extension results are uncontrolled because both groups received molgramostim after week 48. The extension reports arithmetic mean changes rather than the adjusted randomized-treatment estimates. Its three reported deaths were assessed as unrelated to treatment; the earlier statement that no deaths occurred applies to the double-blind report, not to all subsequent follow-up.

IMPALA

Objective

Pulmonary gas-transfer objective

“improve the transfer of oxygen from the lungs to the blood” (opens the source at this quote in a new tab)

Location and study date

Geographic participation

Attribute	Reported value
Clinical sites	“34”
Countries	“18”

Trial period reported in the plain language summary

Milestone	Reported date
Trial start	“February 2016”
Trial finish	“September 2019”

Study design

Phase 2/3 randomized trial

“IMPALA was a phase 2/phase 3, placebo-controlled, randomized, double-blind trial.” (opens the source at this quote in a new tab)

Blinded intervention period

“24-week, double-blind, placebo-controlled intervention period” (opens the source at this quote in a new tab)

Allocation and extension duration

Component	Reported value
Randomization ratio across continuous molgramostim, intermittent molgramostim, and placebo	“1:1:1”
Maximum open-label extension duration	“48 weeks”

Eligibility criteria

Adult autoimmune PAP population with impaired oxygenation

Eligibility component	Quoted criterion
Minimum age, years	“18”
Minimum alveolar-arterial oxygen difference, mm Hg	“25”
Resting arterial oxygen threshold, mm Hg	“75”

Treatment

Continuous and intermittent inhaled regimens

Treatment component	Quoted specification
Molgramostim dose	“300 μg/day”
Continuous regimen	“every week”
Intermittent regimen	“every other week”
Placebo during intermittent off-treatment weeks	“placebo”

Study outcomes

Rationale for using Alveolar-Arterial oxygen difference as the primary endpoint

Endpoint-selection rationale

“based on investigator recommendations and extensive use in prior studies.” (opens the source at this quote in a new tab)

Primary assessment time point

Measure	Assessment week
Change from baseline in alveolar-arterial oxygen difference	“24”

Statistical analysis description

Number of participants (Planned and analyzed)

Randomized participants

“138 with aPAP were randomized” (opens the source at this quote in a new tab)

Treatment-group allocation

Group	Randomized participants
Continuous molgramostim	“46”
Intermittent molgramostim	“45”
Placebo	“47”

Description of analysis sets

Summary

The published analysis distinguishes the original full analysis set from a revised analysis that replaced invalid oxygen-gradient measurements obtained during supplemental oxygen administration. Four participants were affected: one in each molgramostim group and two in the placebo group. The revised analysis used imputation. The original full-analysis-set primary comparison was not statistically significant.

Invalid arterial blood-gas measurements requiring revision

Affected group	Participants
Continuous molgramostim	“1”
Intermittent molgramostim	“1”
Placebo	“2”

Analysis populations reported in the initial congress presentation

Original analysis	Revised analysis
“A-aDO2* – FAS”	“A-aDO2* – Revised”

Results

Participant disposition

Completion of the blinded intervention

Group	Participants completing the blinded intervention
Continuous molgramostim	“97.8%”
Intermittent molgramostim	“97.8%”
Placebo	“93.6%”

Open-label extension enrollment

Disposition measure	Participants
Enrolled in the open-label extension	“131”

Baseline characteristics

Demographic and pulmonary-function characteristics

Characteristic	Continuous molgramostim	Intermittent molgramostim	Placebo
Age, years, mean and standard deviation	“54.0±13.3”	“49.2±14.1”	“46.1±14.8”
DLCO percent predicted, mean and standard deviation	“51.9±18.5”	“46.1±14.5”	“49.6±14.3”
Alveolar-arterial oxygen difference, mm Hg, mean and standard deviation	“40.5±19.6”	“40.9±20.2”	“40.2±14.3”

Efficacy results

Initial congress report: original and revised primary analyses

“Endpoint”	“Units”	“Treatment effect”	“P-value”
“A-aDO2* – FAS”	“mm Hg”	“-4.6”	“0.1688”
“A-aDO2* – Revised”	“mm Hg”	“-6.5”	“0.0249”

Published primary analysis: continuous molgramostim versus placebo

Analysis	Estimated treatment difference in alveolar-arterial oxygen difference, mm Hg	P-value
Original full analysis set	“−5.2”	“P=0.118”
Revised analysis with invalid measurements replaced by imputation	“−6.2”	“P=0.025”

Published secondary pulmonary and exercise outcomes at week 24

Outcome	Estimated treatment difference and 95% confidence interval
DLCO percent predicted, percentage points	“7.8 (2.3 to 13.3)”
Ground-glass opacity score	“−2.5 (−3.7 to −1.2)”
Six-minute walk distance, m	“24.6 (−15.3 to 64.4)”

Health-related quality of life and patient-reported outcomes

SGRQ Total Score at week 24

Outcome	Estimated treatment difference and 95% confidence interval
Continuous molgramostim versus placebo, SGRQ Total Score	“−7.4 (−13.1 to −1.6)”

Safety results

Mortality during the study

“No deaths occurred during the study.” (opens the source at this quote in a new tab)

Study limitations

Summary

The primary result depends on the analysis used. The original full-analysis-set comparison was not statistically significant; the revised analysis replaced invalid measurements from four participants and yielded a statistically significant difference. The earlier congress presentation and the subsequently published analysis report different numerical estimates and should remain separate evidence items.

Blinded treatment lasted 24 weeks. The six-minute walk-distance confidence interval included no difference, and the subsequent extension did not retain a placebo control. These findings do not establish effects on mortality or long-term comparative safety.

IMPALA-X

Objective

Long-term safety objective

“To investigate safety of long term use of inhaled molgramostim.” (opens the source at this quote in a new tab)

Location and study date

Participating centers and originally planned study period

Attribute	Quoted specification
Participating centers	“Centres participating in the IMPALA (MOL-PAP-002) trial”
Estimated first enrollment	“Q1 2018”
Estimated last completion	“Q4 2021”

Study design

Uncontrolled extension study

Design component	Quoted specification
Design	“open-label, non-controlled”
Planned treatment duration	“up to 36-month treatment period”

Eligibility criteria

Entry after the preceding IMPALA study

“Completed the IMPALA study” (opens the source at this quote in a new tab)

Treatment

Intermittent inhaled molgramostim

Treatment component	Quoted specification
Daily dose during treatment weeks	“300 μg/day”
Intermittent schedule	“1 week on and 1 week off”

Study outcomes

Rationale for using adverse events as the primary endpoint

Not applicable.

Statistical analysis description

Number of participants (Planned and analyzed)

Planned enrollment

Planning measure	Participants
Maximum planned enrollment	“90”

Enrolled participants

“n = 60” (opens the source at this quote in a new tab)

Description of analysis sets

Descriptive safety analysis

“Descriptive statistics were used to summarize AEs” (opens the source at this quote in a new tab)

Analysis populations and exposure

Measure	Reported value
Full analysis set	“60”
Safety analysis set	“59”
Molgramostim exposure, patient-years	“93.4”

Results

Participant disposition

Early discontinuation of the extension

“discontinued early as intermittent dosing did not show substantial evidence of benefit vs placebo (PBO) in the IMPALA trial” (opens the source at this quote in a new tab)

Completion of the planned treatment duration

Disposition measure	Reported value
Participants completing the planned 36 months	“no patients”

Baseline characteristics

Characteristics by preceding randomized treatment

Characteristic	Prior continuous molgramostim, 15 participants	Prior intermittent molgramostim, 23 participants	Prior placebo, 22 participants
Age, years, mean and standard deviation	“48.7 (14.0)”	“46.7 (12.8)”	“45.6 (14.4)”
Male, number and percentage	“7 (46.7)”	“18 (78.3)”	“11 (50.0)”

Efficacy results

No evidence found.

Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results

Serious treatment-emergent events and mortality

Safety finding	Quoted value
Participants experiencing serious treatment-emergent adverse events	“Eight”
Attribution of those serious events to study treatment	“none”
Reported deaths due to COVID-19 infection	“One”
Interval between final molgramostim dose and the reported death	“24 days”

Study limitations

Summary

IMPALA-X was an uncontrolled extension that enrolled 60 participants, with 59 included in the safety analysis. It was terminated early, and no participant completed the planned 36 months. The reported exposure was 93.4 patient-years. Selection from participants who completed the preceding study and the absence of a concurrent comparator limit interpretation of adverse-event frequencies. The intermittent regimen was not pursued after the preceding IMPALA results and should not be treated as equivalent to the continuous daily regimen studied in IMPALA-2.

SAV006-04

Objective

Pediatric efficacy evaluation

“efficacy of inhaled molgramostim” (opens the source at this quote in a new tab)

Location and study date

Listed study location and planned dates

Attribute	Reported value
Listed country	“Germany”
Study start	“October 22, 2025”
Estimated completion	“December 31, 2027”

Study design

Open-label pediatric study

“This is an interventional open-label, single arm, multi-center study in pediatric subjects, age 6 through 18 years” (opens the source at this quote in a new tab)

Development phase and treatment-group count

Design component	Reported value
Phase	“3”
Treatment groups	“1”

Eligibility criteria

Specified age limits and diagnostic confirmation

“Be ≥6 and <18 years of age” (opens the source at this quote in a new tab)
“positive serum anti-GM-CSF autoantibody test result” (opens the source at this quote in a new tab)

Pulmonary gas-transfer criterion

Measure	Screening threshold
Hemoglobin-adjusted DLCO percent predicted	“≤70%”

Treatment

Registered inhaled treatment

Treatment component	Quoted specification
Maximum daily dose	“300 µg microgram(s)”
Route	“INHALATION USE”
Maximum treatment duration	“48 Week(s)”

Study outcomes

Rationale for using hemoglobin-adjusted DLCO percent predicted as the primary endpoint

Registered primary endpoint

“Change in Hb-adjusted % predicted DLCO from Baseline to Week 24.” (opens the source at this quote in a new tab)

Pediatric-specific endpoint validation

No evidence found.

Statistical analysis description

Number of participants (Planned and analyzed)

Planned enrollment

Enrollment measure	Participants
Planned total	“5”

Analyzed population

No evidence found.

Description of analysis sets

No evidence found.

Results

Participant disposition

No evidence found.

Baseline characteristics

No evidence found.

Efficacy results

No evidence found.

Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results

No evidence found.

Study limitations

Summary

The registered study has one open-label treatment group and a planned enrollment of five participants. No analyzed results are included in this report. Its design does not provide a concurrent placebo comparison and will provide limited precision for uncommon adverse events. The inclusion criteria specify age at least 6 years and less than 18 years, which is narrower than the older narrative description of ages 6 through 18.

Long-term outcomes in five patients with Autoimmune pulmonary alveolar proteinosis treated with Molgramostim Inhalation Solution

Objective

Long-term treatment outcomes

“long-term effects of molgramostim inhalation solution” (opens the source at this quote in a new tab)

Location and study date

Case-specific treatment initiation dates

Case	Reported initiation of molgramostim
Case 1	“June 2019”
Case 2	“March 2020”

Study design

Observational case series

“real-world case series” (opens the source at this quote in a new tab)

Eligibility criteria

Diagnostic findings in the reported cases

Finding	Case 1	Case 2	Case 3	Case 4	Case 5
Anti-GM-CSF antibody	“Positive”	“Positive”	“Positive”	“Positive”	“Positive”

Prospectively defined eligibility criteria

No evidence found.

Treatment

Compassionate-use inhalation treatment

“vibrating mesh nebuliser” (opens the source at this quote in a new tab)
“compassionate-use programme” (opens the source at this quote in a new tab)

Reported duration of molgramostim exposure

Case	Time on molgramostim, years
Case 1	“4.5”
Case 2	“3.5”
Case 3	“5.5”
Case 4	“4 and previous 1 year in IMPALA trial”
Case 5	“1.5 and previous 1 year in IMPALA trial”

Study outcomes

Rationale for using DLCO percent predicted as an observational outcome

Not applicable.

Statistical analysis description

Number of participants (Planned and analyzed)

Reported case count

Population	Reported number
Participants in the case series	“five”

Description of analysis sets

Retrospective data collection

“retrospective nature” (opens the source at this quote in a new tab)

Formal comparative analysis sets

Not applicable.

Results

Participant disposition

Ages at last reported follow-up

Case	Age at last follow-up, years
Case 1	“31”
Case 2	“33”
Case 3	“40”
Case 4	“32”
Case 5	“52”

Baseline characteristics

Ages at presentation as reported in the case-series table

Case	Age at presentation, years
Case 1	“27”
Case 2	“29”
Case 3	“31”
Case 4	“21”
Case 5	“46”

Efficacy results

Pulmonary function before treatment and at last assessment

Measure, initial/last	Case 1	Case 2	Case 3	Case 4	Case 5
DLCO percent predicted	“37/91”	“36/60”	“39/87”	“62/90”	“12/44”
Forced vital capacity percent predicted	“64/74”	“83/122”	“52/71”	“73/88”	“54/75”

Health-related quality of life and patient-reported outcomes

Case 5: return to employment

“he has returned to work.” (opens the source at this quote in a new tab)

Standardized patient-reported outcome scores

No evidence found.

Safety results

Reported serious adverse events

“There were no reported serious adverse events in these five patients while receiving molgramostim and the treatment has been well tolerated.” (opens the source at this quote in a new tab)

Study limitations

Summary

This retrospective series describes five participants without a concurrent comparator. Treatment exposure and follow-up duration varied, and two cases included preceding IMPALA exposure. The reported pulmonary-function improvements cannot establish a comparative treatment effect. Absence of reported serious adverse events in five cases does not estimate the frequency of uncommon harms, and the qualitative return-to-work report is not a standardized quality-of-life assessment.

MOL-001

Objective

Safety and clinical pharmacology

“safety, PK and PD” (opens the source at this quote in a new tab)

Location and study date

Study location and enrollment period

Attribute	Quoted specification
Clinical research facility	“Celerion”
Location	“Belfast”
Enrollment period	“May through September 2015”

Study design

Phase 1 randomized pharmacology study

“randomised, placebo-controlled, blinded, phase 1 clinical trial” (opens the source at this quote in a new tab)

Eligibility criteria

Healthy nonsmoking adult population

“life-long non-smokers” (opens the source at this quote in a new tab)

Age limits

Eligibility measure	Reported range
Age, years	“18 to 55”

Treatment

Single ascending-dose cohorts

Cohort	Molgramostim dose, µg	Participants
First active-dose cohort	“150”	“4”
Second active-dose cohort	“300”	“4”
Third active-dose cohort	“600”	“4”
Pooled placebo cohort	“placebo”	“6”

Multiple ascending-dose cohorts

Cohort	Daily molgramostim dose, µg	Participants
First active-dose cohort	“300”	“9”
Second active-dose cohort	“600”	“9”
Pooled placebo cohort	“placebo”	“6”

Repeated-dose duration

Treatment component	Reported duration
Consecutive daily doses in the multiple ascending-dose study	“six”

Study outcomes

Rationale for using adverse events as the primary endpoint

Not applicable.

Statistical analysis description

Number of participants (Planned and analyzed)

Total enrollment

“42 subjects were enrolled” (opens the source at this quote in a new tab)

Enrollment by study component

Study component	Participants
Single ascending dose	“18”
Multiple ascending dose	“24”

Description of analysis sets

Pharmacokinetic analysis method

“Non-compartmental PK” (opens the source at this quote in a new tab)

Multiplicity

“All reported p-values were two-sided without adjustment for multiple testing.” (opens the source at this quote in a new tab)

Formal analysis-set definitions

No evidence found.

Results

Participant disposition

Study completion

Disposition measure	Participants
Enrolled and completed the study	“42”

Baseline characteristics

Overall demographic characteristics

Characteristic	Reported value
Age, years, mean and standard deviation	“33.7±11.7”
Age range, years	“(18, 53)”
Male, number and percentage	“40 (95)”
Female, number and percentage	“2 (5)”
White, number and percentage	“40 (95)”
Black, number and percentage	“2 (5)”

Efficacy results

Pharmacokinetic results in healthy volunteers

“healthy people” (opens the source at this quote in a new tab)
“peaked at 2 hours for all three doses and had an elimination half-life of 1.7±0.0 to 5.9±0.9 hours” (opens the source at this quote in a new tab)

Disease-specific efficacy

Not applicable.

Health-related quality of life and patient-reported outcomes

Not applicable.

Safety results

Adverse events by dosing component

Study component	Molgramostim: participants with adverse events	Placebo: participants with adverse events
Single ascending dose	“7 (58%)”	“4 (67%)”
Multiple ascending dose	“15 (83%)”	“6 (100%)”

Serious adverse events

“No serious AEs occurred” (opens the source at this quote in a new tab)

Study limitations

Summary

This study enrolled 42 healthy participants, including two women, and evaluated single doses or six consecutive daily doses. It does not establish efficacy in autoimmune pulmonary alveolar proteinosis or the safety of prolonged daily treatment. The sample size and short exposure limit assessment of uncommon or delayed adverse events. Statistical comparisons were not adjusted for multiple testing.