Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
APL2 · 1 trial · 1 indication
Change from Baseline in Log-Transformed uPCR will be based on triplicate first morning urine (FMU)
Change from baseline in log-transformed uPCR will be based on triplicate first morning urine (FMU)
| Arm | Type | Description |
|---|---|---|
| Phase 2 - APL2 | EXPERIMENTAL | Sub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly |
| Phase 3 - APL2 | EXPERIMENTAL | Adults: Sub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly Adolescents: ≥50 Kg: Sub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly 35 to \<50kg: First sub-cutaneous infusion of 648mg (12 mL) followed by 810mg (15 mL) every infusion thereafter (i.e. twice weekly) 30 to \<35kg: First \& Second sub-cutaneous infusion of 540mg (10mL) followed by 648mg (12 mL) every infusion thereafter (twice weekly) |
| Phase 3 - Placebo | PLACEBO_COMPARATOR | Subcutaneous infusions of a sterile solution, twice-weekly, and equivalent in volume to the active arm based on participant's age and weight |
| Name | Type | Description |
|---|---|---|
| APL2 | DRUG | Complement (C3) Inhibitor |
| Placebo | OTHER | Sterile solution of equal volume to active arm |
Inclusion Criteria: * Age * Phase 2: adults aged ≥18 years * Phase 3: adults aged ≥18 years; if and where approved, adolescents (aged 12--17 years) at the time of signing the informed consent and assent form * Weight ≥30 kg and ≤100 kg at screening * FSGS diagnosis * Phase 2: primary, genet...
APL-2 is an investigational therapy being studied for warm autoimmune hemolytic anemia, paroxysmal nocturnal hemoglobinuria, IgA nephropathy, and geographic atrophy secondary to age-related macular degeneration. It is a small molecule in Phase 3 development by Apellis Pharmaceuticals. APL-2 is not FDA approved and remains in clinical development.
APL-2 targets the complement system, specifically complement component C3. By inhibiting C3, APL-2 is designed to block complement activation at multiple levels. This mechanism is being investigated across complement-mediated diseases including paroxysmal nocturnal hemoglobinuria and geographic atrophy.
APL-2 is developed by Apellis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker APLS. Apellis is conducting clinical trials of APL-2 across multiple indications including geographic atrophy and paroxysmal nocturnal hemoglobinuria.
APL-2 is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA. Completed Phase 2 and Phase 3 trials have evaluated APL-2 in conditions such as geographic atrophy, warm autoimmune hemolytic anemia, and IgA nephropathy.
APL-2 has been studied in several completed trials. NCT03525613 is a Phase 3 study in geographic atrophy with 637 patients. NCT04085601 is a Phase 3 study in paroxysmal nocturnal hemoglobinuria with 53 patients. NCT03226678 and NCT03453619 are Phase 2 studies in autoimmune hemolytic anemia and glomerulopathies.
Yes, APL-2 is also known as pegcetacoplan. Clinical trials such as NCT04085601 refer to pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria. This alternative name is used in later-stage studies of the same investigational compound developed by Apellis Pharmaceuticals.