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M5049 low dose

Phase 3

Systemic Lupus Erythematosus | Small molecule | Immunology |Merck KGaA|Trials Updated: Oct 15, 2026

M5049 low dose development status

Highest phase Phase 3 (NCT07840287)
Phase scored for MKGAFPhase 2
Registered trials 6 across 2 sponsors since Nov 2021

M5049 low dose target and mechanism

ModalitySmall molecule

Also known as M5049, Enpatoran

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials8
Total Enrollment1,853

FDA Designations

No designations recorded

M5049 low dose clinical trials

M5049 low dose · 8 trials · 7 indications

Phase 3 3Phase 2 3Phase 1 2
NCT07840287A Long-Term Extension (LTE) Study Of Enpatoran In Cutaneous Manifestations Of Lupus With Or Without Systemic Disease [Elowen-LTE]Systemic Lupus Erythematosus (SLE)
NOT YET_RECRUITING404 Analytics
NCT07355218A Study of Enpatoran in Participants With Cutaneous Manifestations of Lupus With or Without Systemic Disease (ELOWEN-2)Systematic Lupus Erythematosus
RECRUITING202 Analytics
NCT07332481A Study of Enpatoran in Participants With Cutaneous Manifestations of Lupus With or Without Systemic DiseaseSystemic Lupus Erythematosus (SLE)
RECRUITING202 Analytics
PHASE3NOT YET_RECRUITING
A Long-Term Extension (LTE) Study Of Enpatoran In Cutaneous Manifestations Of Lupus With Or Without Systemic Disease [Elowen-LTE]
Systemic Lupus Erythematosus (SLE)Unlock trial analytics
PHASE3RECRUITING
A Study of Enpatoran in Participants With Cutaneous Manifestations of Lupus With or Without Systemic Disease (ELOWEN-2)
Systematic Lupus ErythematosusUnlock trial analytics
PHASE3RECRUITING
A Study of Enpatoran in Participants With Cutaneous Manifestations of Lupus With or Without Systemic Disease
Systemic Lupus Erythematosus (SLE)Unlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Treatment Emergent adverse events (TEAEs), Serious Adverse Events (SAEs), and Adverse Events of Special Interest (AESI)
From current LTE study Day 1 to Week 120
Percentage of Participants With Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) 70 Response, Defined as >= 70 Percent (%) Decrease in CLASI-A Score From Baseline
At Week 24
Time to Recovery
Day 1 through Day 28

Time to recovery was defined as the time from first dose (Day 1) to first occurrence of World Health Organization (WHO) 9-point ordinal scale 3 or less. The scoring is assessed with the following ordinal scale: 0. Uninfected: no clinical or virological evidence of infection; 1. Ambulatory: no limitation of activities; 2. Ambulatory: limitation of activities; 3. Hospitalized, mild disease: hospitalized, no oxygen therapy; 4. Hospitalized, mild disease: oxygen by mask or nasal prongs; 5. Hospitalized, severe disease: noninvasive ventilation or high-flow oxygen; 6. Hospitalized, severe disease: intubation and mechanical ventilation; 7. Hospitalized, severe disease: ventilation plus additional organ support for example: vasopressors or Extracorporeal membrane oxygenation (ECMO); 8. Death.

Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Adverse Events of Special Interests (AESIs), TEAEs Leading to Treatment Discontinuation and Serious TEAEs (SAEs) According to NCI-CTCAE Version 5.0
Day 1 through Day 60

Adverse Event (AE) was defined any untoward medical occurrence in a participant administered with a study drug, which does not necessarily had a causal relationship with this treatment. Serious AE was defined AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial/prolonged inpatient hospitalization; congenital anomaly/birth defect. TEAEs: TEAEs was defined as events with onset date or worsening during the on treatment period. TEAEs included serious TEAEs and non-serious TEAEs.

Number of Participants With Clinically Significant Changes From Baseline in Laboratory Parameters
Day 1 through Day 28

Laboratory investigation included hematology and biochemistry. The number of participants with clinically significant changes from baseline in laboratory parameters were reported. Clinical significance was determined by the investigator.

Number of Participants With Clinically Significant Changes From Baseline in 12-Lead Electrocardiogram (ECG) Measurements
Day 1 through Day 28

12-lead ECG recordings included rhythm, heart rate (as measured by RR interval), PR interval, QRS duration, and QT interval. 12-lead ECG recordings were obtained after the participants have rested for at least 10 minutes in semisupine position. Clinical significance was determined by the investigator. The number of participants with clinically significant changes from baseline in 12-lead ECG findings were reported.

Part 1: Safety Profile as Assessed by Incidence of Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Adverse Event of Special Interest (AESIs)
Baseline up to Week 50 (Long Term Extension Part 1)
Part 2: Safety Profile as Assessed by Incidence of Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Adverse Event of Special Interest (AESIs)
Baseline up to Week 194 (LTE Part 1 and LTE Prolongation Part 2))
Cohort A: Change From Baseline in Cutaneous Lupus Erythematosus Disease Area and Severity Index-A (CLASI-A) Total Score at Week 16
Baseline, week 16

The CLASI (Cutaneous Lupus Erythematosus Disease Area and Severity Index) was a validated instrument used to assess disease activity (CLASI-A) and damage in lupus erythematosus. The activity scale included erythema, scale/hypertrophy, active alopecia, recent hair loss, and mucous membrane disease, while the damage scale measured dyspigmentation, atrophy, and scarring. CLASI-A scores ranged from 0 to 70, with mild, moderate, and severe disease corresponding to scores of 0-9, 10-20, and 21-70, respectively. Erythema and scale/hypertrophy sub-scores were computed across 13 body areas, with maximum scores of 39 and 26, respectively. The remaining 5 points reflected contributions from active alopecia (0-3), recent hair loss (0-1), and mucous membrane involvement (0-1), completing the total CLASI-A activity score. Directionality reflected worsening with higher scores and improvement with lower scores.

Cohort B: Number of Participants With British Isles Lupus Assessment Group (BILAG)-Based Composite Lupus Assessment (BICLA) Response at Week 24
At Week 24

BILAG-based BICLA response is defined as participants meeting all of the following criteria: 1. Improvement in baseline BILAG scores in all organ systems with moderate or severe disease activity-i.e., all grade A scores (severe disease requiring high-dose therapy) must improve to B (moderate), C (mild), or D (no activity); all grade B scores (moderate disease requiring moderate therapy) must improve to C or D; 2. No new BILAG A scores and no more than one new BILAG B score; 3. No worsening in total SLEDAI-2K score from baseline; 4. No significant deterioration (≤10%) in physician's global assessment; and 5. No treatment failure, defined as initiation of non-protocol therapy. Directionality is toward clinical improvement and disease stabilization.

Pharmacokinetic (PK) Plasma Concentrations of Enpatoran
Predose, 0.5, 1, 1.5, 2, 4, 8, 12, 24, 36, and 48 hours postdose
Part A: Cohort 1 and 2: Number of Participants with Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs and Adverse Event of Special Interest (AESI), TEAEs Leading to Permanent Treatment Discontinuation and Treatment-Related TEAEs
Up to Day 102
Part A: Cohort 3 and 4; Part B: Cohort 5: Number of Participants with Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs and Adverse Event of Special Interest (AESI), TEAEs Leading to Permanent Treatment Discontinuation and Treatment-Related TEAEs
Up to Day 186
Part A: Cohort 1 and 2: Number of Participants with Treatment-Emergent Adverse Events (TEAEs) Based on Severity
Up to Day 102
Part A: Cohort 3 and 4; Part B: Cohort 5: Number of Participants with Treatment-Emergent Adverse Events (TEAEs) Based on Severity
Up to Day 186
Part A: Cohort 1 and 2: Number of Participants with Clinically Significant Changes from Baseline in Laboratory Parameters, Vital Signs, Electroencephalogram (EEG) and Electrocardiogram (ECG) Findings
Up to Day 102
Part A: Cohort 3 and 4; Part B: Cohort 5: Number of Participants with Clinically Significant Changes from Baseline in Laboratory Parameters, Vital Signs, Electroencephalogram (EEG) and Electrocardiogram (ECG) Findings
Up to Day 186
Part A: Cohort 1 and 2: Number of Participants with Confirmed Signs and Symptoms of Prodromal Seizure
Up to Day 102
Part A: Cohort 3 and 4; Part B: Cohort 5: Number of Participants with Confirmed Signs and Symptoms of Prodromal Seizure
Up to Day 186
Part A: Cohort 1 and 2: Number of Participants with Suicidal Behavior and Suicidal Ideation as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS)
Up to Day 102

Secondary Endpoints

Number of Participants With Abnormalities in Laboratory Parameters (greater than or equal to [>=] Grade 3)
From current LTE study Day 1 to Week 120
Number of Participants With Abnormalities in Vital Signs
From current LTE study Day 1 to Week 120
Number of Participants With Abnormalities in Electrocardiogram (ECG) Parameter
From current LTE study Day 1 to Week 120
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
EnpatoranEXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -
M5049 50 mgEXPERIMENTAL -
M5049 100 mgEXPERIMENTAL -
M5049 low dose + PlaceboEXPERIMENTALParticipants with CLE (active SCLE and/or DLE) or SLE who received low dose of M5049 in WILLOW study will continue to receive M5049 low dose and matching placebo.
M5049 medium dose+ PlaceboEXPERIMENTALParticipants with CLE (active SCLE and/or DLE) or SLE who received medium dose of M5049 in WILLOW study will continue to receive M5049 medium dose and matching placebo.
M5049 high dose + PlaceboEXPERIMENTALParticipants with CLE (active SCLE and/or DLE) or SLE who received M5049 matched placebo or high dose of M5049 in WILLOW study will receive M5049 high dose .
Cohort A: PlaceboPLACEBO_COMPARATORParticipants with CLE (active SCLE and/or DLE) or SLE with predominantly active lupus rash (Cutaneous Lupus Erythematosus Disease Area and Severity Index \[CLASI-A\] greater than or equal to \[\>=\] 8) will be enrolled in Cohort A to receive placebo matched to Enpatoran.
Cohort A: Enpatoran low doseEXPERIMENTALParticipants with CLE (active SCLE and/or DLE) or SLE with predominantly active lupus rash (CLASI-A \>= 8) will be enrolled in Cohort A to receive low dose of Enpatoran.
Cohort A: Enpatoran medium doseEXPERIMENTALParticipants with CLE (active SCLE and/or DLE) or SLE with predominantly active lupus rash (CLASI-A \>= 8) will be enrolled in Cohort A to receive medium dose of Enpatoran.
Cohort A: Enpatoran high doseEXPERIMENTALParticipants with CLE (active SCLE and/or DLE) or SLE with predominantly active lupus rash (CLASI-A \>= 8) will be enrolled in Cohort A to receive high dose of Enpatoran.
Cohort B (Part 1 + Part 2): PlaceboPLACEBO_COMPARATORParticipants with active SLE who have moderate to high systemic disease activity (British Isles Lupus Assessment Group \[BILAG A/2B\]) with 1 or 2 of the following: CLASI-A \>= 8 and/or Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) \>= 6 will be enrolled in Cohort B to receive placebo matched to Enpatoran .
Cohort B (Part 1 + Part 2): Enpatoran high doseEXPERIMENTALParticipants with active SLE who have moderate to high systemic disease activity (BILAG A/2B) with 1 or 2 of the following: CLASI-A \>= 8 and/or SLEDAI \>= 6 will be enrolled in Cohort B to receive high dose of Enpatoran.
Cohort B (Part 2): Enpatoran low doseEXPERIMENTALParticipants with active SLE who have moderate to high systemic disease activity (BILAG A/2B) with 1 or 2 of the following: CLASI-A \>= 8 and/or SLEDAI \>= 6 will be enrolled in Cohort B to receive low dose of M5049.
Cohort B (Part 2): Enpatoran medium doseEXPERIMENTALParticipants with active SLE who have moderate to high systemic disease activity (BILAG A/2B) with 1 or 2 of the following: CLASI-A \>= 8 and/or SLEDAI \>= 6 will be enrolled in Cohort B to receive medium dose of Enpatoran.
Control Group 1 (Normal Renal Function)EXPERIMENTAL -
Group 2 (Impaired Renal Function)EXPERIMENTAL -
Part A (Cohort 1): M5049 Dose AEXPERIMENTAL -
Part A (Cohort 2): M5049 Dose BEXPERIMENTAL -
Part A (Cohort 3): M5049 Dose CEXPERIMENTAL -
Part A (Cohort 4): M5049 Dose DEXPERIMENTAL -
Part A: PlaceboPLACEBO_COMPARATOR -
Part B (Cohort 5): M5049 Dose EEXPERIMENTAL -
Part B: PlaceboPLACEBO_COMPARATOR -

Interventions

NameTypeDescription
EnpatoranDRUGParticipants will receive a dose of Enpatoran orally for 108 weeks.
PlaceboDRUGParticipants will receive a single oral dose of a placebo matching Enpatoran tablet twice daily from Day 1 to Day 168.
Standard of care (SoC)DRUGParticipants will receive Investigator-recommended SoC.
M5049DRUGParticipants received M5049 50 milligram (mg) orally twice daily for 14 days.
M5049 low doseDRUGParticipants will receive film-coated tablets of M5049 at a low dose orally, twice a day (BID) for up to 194 weeks.
M5049 medium doseDRUGParticipants will receive film-coated tablets of M5049 at a medium dose orally, BID for up to 194 weeks.
M5049 high doseDRUGParticipants will receive film-coated tablets of M5049 at a high dose orally, BID for up to 194 weeks.
Enpatoran low doseDRUGParticipants will receive film-coated tablets of Enpatoran at a low dose orally, twice daily (BID) up to 24 weeks.
Enpatoran medium doseDRUGParticipants will receive film-coated tablets of Enpatoran at a medium dose, orally, BID up to 24 weeks.
Enpatoran high doseDRUGParticipants will receive film-coated tablets of Enpatoran at a high dose, orally, BID up to 24 weeks.
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Eligibility Criteria

Age Range18 Years to 76 Years
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: 1. Are up to date according to local guidelines/ recommendations. Recombinant zoster vaccination is encouraged but not mandatory. 2. Are participants with active cutaneous manifestations of lupus erythematosus that have successfully completed the 24-week treatment of the Elowen-...

Countries:United StatesGermanyAustraliaBrazilBulgariaCanadaChinaCzechiaIsraelItalyPolandRomaniaSouth KoreaSpainSwitzerlandTaiwanUnited KingdomFranceJapanSlovakiaPhilippinesArgentinaChileColombiaGreeceMauritiusMexicoMoldovaSerbiaSouth AfricaNorth MacedoniaUkraine
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Recent Changes (Last 90 Days)

LOWOct 15, 2026NCT05540327Enrollment: 379 → 381
LOWOct 15, 2026NCT05540327Enrollment: 379 → 381
LOWOct 6, 2026NCT07355218lastUpdatePostDate: changed
LOWOct 6, 2026NCT07332481lastUpdatePostDate: changed
LOWOct 6, 2026NCT07355218lastUpdatePostDate: changed
LOWOct 6, 2026NCT07332481lastUpdatePostDate: changed
LOWSep 25, 2026NCT07840287NEW_TRIAL: changed
LOWSep 25, 2026NCT07840287NEW_TRIAL: changed
LOWSep 15, 2026NCT07355218lastUpdatePostDate: changed
LOWSep 15, 2026NCT07355218lastUpdatePostDate: changed
LOWSep 15, 2026NCT07355218lastUpdatePostDate: changed
LOWSep 11, 2026NCT07332481lastUpdatePostDate: changed
LOWSep 11, 2026NCT07332481lastUpdatePostDate: changed
LOWAug 17, 2026NCT07355218lastUpdatePostDate: changed
LOWAug 17, 2026NCT07355218lastUpdatePostDate: changed
LOWAug 14, 2026NCT07332481lastUpdatePostDate: changed
LOWAug 14, 2026NCT07332481lastUpdatePostDate: changed

Frequently asked questions about M5049 low dose

What is M5049 low dose?

M5049 low dose refers to a lower-dose regimen of M5049, an investigational small molecule developed by Merck KGaA. It has been studied in Phase 2 clinical trials for systemic lupus erythematosus, including cutaneous forms of lupus, and for COVID-19 pneumonia. M5049 is not described as an approved therapy and remains in clinical development.

What is M5049 low dose used for in systemic lupus erythematosus?

M5049 low dose has been evaluated in systemic lupus erythematosus, including subacute cutaneous lupus erythematosus and discoid lupus erythematosus, in the WILLOW long-term extension study. A separate completed Phase 1 study enrolled participants with cutaneous lupus erythematosus and SLE. These are investigational uses in clinical research, not approved indications.

Who makes M5049 low dose?

M5049 low dose is developed by Merck KGaA, which trades under the ticker MKGAF. Merck KGaA sponsors the clinical program for M5049, including the Phase 2 WILLOW long-term extension study in lupus and the completed Phase 2 ANEMONE study in COVID-19 pneumonia.

What phase is M5049 low dose in?

M5049 low dose is in Phase 2 development. The Phase 2 WILLOW long-term extension study in lupus is active but not recruiting, and a completed Phase 2 study evaluated M5049 in COVID-19 pneumonia. M5049 is investigational and is not described as FDA approved for any indication.

What clinical trials is M5049 low dose in?

M5049 has been studied in three registered trials. NCT05540327 is the active Phase 2 WILLOW long-term extension in lupus, enrolling 381 participants across 22 countries. NCT04647708 is a completed Phase 1 study in cutaneous and systemic lupus erythematosus, and NCT04448756 is the completed Phase 2 ANEMONE study in COVID-19 pneumonia.

Is M5049 low dose the same as M5049?

Yes, M5049 low dose refers to a lower-dose regimen of M5049, the same investigational compound developed by Merck KGaA. Searches for either name refer to the same small molecule program being studied in lupus and previously in COVID-19 pneumonia.