Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
APL-2 · 5 trials · 9 indications
The Hb stabilization was defined as avoidance of a \>1 gram per deciliter (g/dL) decrease in Hb concentration from Baseline in the absence of transfusion through Week 26.
The LDH concentration was analyzed using an analysis of covariance (ANCOVA) model with a last observation carried forward (LOCF) and a baseline observation carried forward (BOCF) approach for handling missing data. Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method.
The GA lesion area was measured by a quantified central reading center based on FAF images. LS mean was calculated using a mixed effect model for repeated measure (MMRM) model. Baseline was defined as the last available, non-missing observation prior to first study drug administration.
Change from baseline in proteinuria was assessed based on urinary protein-to-creatinine ratio (uPCR). Baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug.
Change from baseline in proteinuria was assessed based on uPCR. Baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug during Part B.
TEAEs were defined as adverse events (AEs) that occurred after dosing on Day 1 and up to 56 days after the last dose of study drug. A treatment-related TEAE is defined as a TEAE with a relationship to study drug of probably, possibly, unlikely, or unrelated. A serious adverse event (SAE) is defined as any AE that resulted in death; was life-threatening; required hospitalization or prolongation of existing hospitalization; resulted in persistent or significant incapacity or substantial disruption of ability to conduct normal life functions; was a congenital anomaly or birth defect. TEAEs were graded according to Common Terminology Criteria for Adverse Events v4.03 based on: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to AE.
| Arm | Type | Description |
|---|---|---|
| Standard of Care (SOC) excluding complement inhibitors | NO_INTERVENTION | - |
| 1,080mg APL-2 administered subcutaneously twice weekly | EXPERIMENTAL | - |
| APL-2 15mg 0.1 mL monthly for 24 months | EXPERIMENTAL | A single dose of 15 mg APL-2/0.1 mL will be administered via intravitreal injection in this study. Subjects will receive an injection every month |
| APL-2 15mg 0.1 mL EOM for 24 months | EXPERIMENTAL | A single dose of 15 mg APL-2/0.1 mL will be administered via intravitreal injection in this study. Subjects will receive an injection every other month |
| Sham Procedure Monthly for 24 months | EXPERIMENTAL | Sham Procedure for 24 months |
| Sham Procedure Every Other Month for 24 months | EXPERIMENTAL | Sham Procedure every other month for 24 months |
| APL-2 | EXPERIMENTAL | Open Label, Study Drug, APL-2 |
| 270mg or 360mg APL-2 administered subcutaneously daily (CAD) | EXPERIMENTAL | - |
| 270mg or 360mg APL-2 administered subcutaneously daily (wAIHA) | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| APL-2 | DRUG | Complement (C3) Inhibitor |
| Sham Procedure | OTHER | Subjects will receive a Sham procedure every month |
Inclusion Criteria: * Be at least 18 years old (inclusive). * Have LDH ≥1.5 x ULN at the screening visit. * Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or monocyte clone \>10%). * Have Hb less than the lower limit of normal (LLN) at the screening visit. * Have ferr...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Regeneron Pharmaceuticals, Inc. | REGN | 2 | PHASE3 | Pozelimab, Cemdisiran |
| Annexon, Inc. | ANNX | 1 | PHASE3 | Vonaprument |
| Belite Bio, Inc. ADR | BLTE | 1 | PHASE3 | Tinlarebant |
| Johnson & Johnson | JNJ | 1 | PHASE2 | JNJ-81201887 |
| AbbVie, Inc. | ABBV | 1 | PHASE1 | ABBV-6628, SYFOVRE |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE1 | SAR446597, Sham Comparator |
| Ocugen Inc | OCGN | 1 | PHASE1 | OCU410 |
| Apellis Pharmaceuticals, Inc. | APLS | 1 | - | Pegcetacoplan |
APL-2, also known as pegcetacoplan, is an investigational therapy being studied for paroxysmal nocturnal hemoglobinuria, geographic atrophy, warm autoimmune hemolytic anemia, and IgA nephropathy. It is in Phase 3 clinical development for these conditions.
APL-2 targets the complement system. It is designed to inhibit complement component C3, which plays a role in the immune response. This mechanism is being investigated for conditions like geographic atrophy and paroxysmal nocturnal hemoglobinuria.
APL-2 is being developed by Apellis Pharmaceuticals, Inc., a biopharmaceutical company. The company's stock is traded under the ticker symbol APLS.
APL-2 is in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Clinical trials are ongoing to evaluate its safety and efficacy for various indications.
APL-2 has been studied in several Phase 3 trials. These include NCT03525600 and NCT03525613 for geographic atrophy secondary to age-related macular degeneration, NCT04085601 for paroxysmal nocturnal hemoglobinuria, and NCT07214740 for geographic atrophy using a prefilled syringe.
Yes, APL-2 is also known as pegcetacoplan. The drug is referred to by both names in clinical research and development contexts.