Approval Probability
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Adjusted LOA
ML Risk
Also known as M5049, Enpatoran
M5049 low dose · 8 trials · 7 indications
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.
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.
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.
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.
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.
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.
| Arm | Type | Description |
|---|---|---|
| Enpatoran | EXPERIMENTAL | - |
| Placebo | PLACEBO_COMPARATOR | - |
| M5049 50 mg | EXPERIMENTAL | - |
| M5049 100 mg | EXPERIMENTAL | - |
| M5049 low dose + Placebo | EXPERIMENTAL | Participants 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+ Placebo | EXPERIMENTAL | Participants 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 + Placebo | EXPERIMENTAL | Participants 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: Placebo | PLACEBO_COMPARATOR | Participants 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 dose | EXPERIMENTAL | Participants 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 dose | EXPERIMENTAL | Participants 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 dose | EXPERIMENTAL | Participants 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): Placebo | PLACEBO_COMPARATOR | Participants 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 dose | EXPERIMENTAL | Participants 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 dose | EXPERIMENTAL | Participants 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 dose | EXPERIMENTAL | Participants 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 A | EXPERIMENTAL | - |
| Part A (Cohort 2): M5049 Dose B | EXPERIMENTAL | - |
| Part A (Cohort 3): M5049 Dose C | EXPERIMENTAL | - |
| Part A (Cohort 4): M5049 Dose D | EXPERIMENTAL | - |
| Part A: Placebo | PLACEBO_COMPARATOR | - |
| Part B (Cohort 5): M5049 Dose E | EXPERIMENTAL | - |
| Part B: Placebo | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| Enpatoran | DRUG | Participants will receive a dose of Enpatoran orally for 108 weeks. |
| Placebo | DRUG | Participants will receive a single oral dose of a placebo matching Enpatoran tablet twice daily from Day 1 to Day 168. |
| Standard of care (SoC) | DRUG | Participants will receive Investigator-recommended SoC. |
| M5049 | DRUG | Participants received M5049 50 milligram (mg) orally twice daily for 14 days. |
| M5049 low dose | DRUG | Participants will receive film-coated tablets of M5049 at a low dose orally, twice a day (BID) for up to 194 weeks. |
| M5049 medium dose | DRUG | Participants will receive film-coated tablets of M5049 at a medium dose orally, BID for up to 194 weeks. |
| M5049 high dose | DRUG | Participants will receive film-coated tablets of M5049 at a high dose orally, BID for up to 194 weeks. |
| Enpatoran low dose | DRUG | Participants will receive film-coated tablets of Enpatoran at a low dose orally, twice daily (BID) up to 24 weeks. |
| Enpatoran medium dose | DRUG | Participants will receive film-coated tablets of Enpatoran at a medium dose, orally, BID up to 24 weeks. |
| Enpatoran high dose | DRUG | Participants will receive film-coated tablets of Enpatoran at a high dose, orally, BID up to 24 weeks. |
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-...
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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.
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.
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.
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.
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.
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.