Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
REGN3918 · 2 trials · 2 indications
Participants were considered to have had adequate control of intravascular hemolysis if all of their lactose dehydrogenase (LDH) readings from Week 4 through Week 26 inclusive had values less than or equal to ≤ 1.5 × upper limit of normal (ULN). Participants must have greater than or equal to (≥) 50 percent (%) of scheduled LDH measures in those weeks, must not have had more than (\>) 2 consecutive visits without LDH measures, must not have experienced breakthrough hemolysis, and must not have discontinued study treatment early. Participants were considered not to have had adequate control of intravascular hemolysis if they failed any of these criteria.
Transfusion avoidance was defined as not having received red blood cell (RBC) transfusion during the first 26 weeks. A transfusion was counted only if it was per-protocol, that is, it followed the predefined transfusion algorithm: RBC transfusion due to a post-baseline hemoglobin level \< 9 grams per deciliter (g/dL) (with anemia symptoms) or a post-baseline hemoglobin level \< 7 g/dL (without anemia symptoms).
| Arm | Type | Description |
|---|---|---|
| REGN3918 | EXPERIMENTAL | Cohort A (Dose Confirmation) If a decision is made to expand Cohort A, patients will be assigned to Cohort A. Cohort B (Dose Expansion) If a decision is made to progress to Cohort B, patients will be assigned to Cohort B. |
| REGN3918 (Cohorts 1-4 & 6a) | EXPERIMENTAL | Cohorts 1-4 and 6a will receive sequential ascending doses of REGN3918 |
| Placebo (Cohorts 1-4 & 6a) | EXPERIMENTAL | Cohorts 1-4 and 6a will receive placebo |
| REGN3918 (Cohort 5 & 6b) | EXPERIMENTAL | Cohort 5 and 6b will receive multiple doses of REGN3918 |
| Placebo (Cohort 5 & 6b) | EXPERIMENTAL | Cohort 5 and 6b will receive placebo |
| Name | Type | Description |
|---|---|---|
| REGN3918 | DRUG | Single intravenous (IV) dose, then a subcutaneous (SC) dose once weekly (QW). |
| Placebo | DRUG | Matching Placebo |
Key Inclusion Criteria: * Diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) confirmed by high-sensitivity flow cytometry * PNH granulocytes \> 10% at screening visit * Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms (eg, fatigue, hemoglobinuria, abdominal ...
REGN3918 is an investigational small molecule being studied for use in healthy volunteers and in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. It is being developed by Regeneron Pharmaceuticals, Inc. and is currently in clinical development, though it is not approved for any use.
REGN3918 is being developed by Regeneron Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol REGN. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational drug.
REGN3918 is in Phase 1 clinical development. A Phase 1 study in healthy volunteers has been completed, and a Phase 2 study in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) has also been completed. The drug remains investigational and is not FDA approved.
REGN3918 has been studied in two completed clinical trials. NCT03115996 was a Phase 1 study in healthy volunteers in the United Kingdom, enrolling 56 participants. NCT03946748 was a Phase 2 study in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), enrolling 24 participants across multiple countries.
No alternative names for REGN3918 have been reported. It is identified solely by its investigational code name REGN3918 in clinical trial registries and by its developer, Regeneron Pharmaceuticals, Inc.