What Is Iptacopan?
Iptacopan is an investigational oral medication currently being studied in clinical trials. It is known by its development code, LNP023, and is administered as an oral capsule. Clinical trials are exploring its potential to treat a range of rare conditions, primarily those involving the complement system, which is part of the body's immune defense.
Research into iptacopan began with the first trial on 2018-02-20. To date, 20 clinical trials have been conducted or are underway, involving a total of 1,602 participants. These studies include 7 trials that are actively recruiting new participants and 4 trials that have already been completed. The latest trial is projected to conclude on 2026-01-16. Novartis Pharmaceuticals is the primary sponsor for 18 of these trials, with additional studies sponsored by Bing Han and the First Affiliated Hospital of Zhejiang University.
Uses and Conditions Under Study
Iptacopan is being investigated for its potential to treat several rare conditions, many of which involve dysregulation of the complement system. The most extensively studied condition for iptacopan is Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare, acquired, life-threatening disease of the blood characterized by destruction of red blood cells, blood clots, and impaired bone marrow function. A total of 4 trials are exploring iptacopan for PNH.
Another area of focus is kidney-related disorders. Iptacopan is being studied in 2 trials for Atypical Hemolytic Uremic Syndrome (aHUS), a rare genetic disease that causes abnormal blood clot formation in small blood vessels, leading to kidney failure. Additionally, C3 Glomerulopathy (C3G), a rare kidney disease characterized by the accumulation of complement protein C3 in the kidney, is being investigated in 2 trials. Glomerulonephritis, IGA, another kidney disorder, is the subject of 1 trial.
Other conditions under investigation include Anti-Neutrophil Cytoplasm Antibodies (ANCA) Associated Vasculitis, a group of autoimmune diseases causing inflammation of small blood vessels, which is being studied in 1 trial. Cold Agglutinin Disease (CAD), a rare autoimmune hemolytic anemia, is also being explored in 1 trial. Furthermore, iptacopan is being studied for Generalized Myasthenia Gravis, a chronic autoimmune neuromuscular disease causing muscle weakness, in 1 trial. Finally, Hematopoietic Stem Cell Transplantation (HSCT), a procedure used to treat certain cancers and blood disorders, is being investigated in 1 trial, likely in relation to complications or supportive care.
Dosing
Iptacopan is primarily studied as an oral medication, typically administered as a capsule. The most common strength investigated in clinical trials is 200 mg.
For adult patients, particularly those with Paroxysmal Nocturnal Hemoglobinuria (PNH), an investigational dosing regimen has included an induction phase of 200 mg twice daily (BID) for the first 4 weeks. Following this, patients enter a maintenance phase, where the dosage is reduced to 200 mg once daily (QD), continuing until the completion of treatment, often at Week 12.
Pediatric dosing is also under investigation. For adolescents aged 12 to less than 18 years, iptacopan 200 mg twice daily (BID) has been studied. For younger children aged 2 to less than 12 years, the specific dosing is still to be determined in ongoing research. Some trials also explore iptacopan in combination with standard of care treatments.
Variations in dosing include studies involving healthy participants, as well as those with mild, moderate, or severe hepatic impairment, to understand how liver function affects the drug's processing in the body.
Side Effects
No specific side effect data for Iptacopan was available in the provided clinical trial results.
Clinical Trial Results
Paroxysmal Nocturnal Hemoglobinuria (PNH)
In a study (NCT03439839) evaluating Iptacopan (LNP023) in patients with PNH, various improvements in blood markers were observed. For patients receiving Iptacopan 200mg twice daily plus standard of care, the reduction in C3 fragment deposition on PNH red blood cells ranged from 0.94% to 16.04%. In a cohort receiving Iptacopan 50mg/200mg twice daily plus standard of care, reductions ranged from 1.24% to 6.76%. Lactate Dehydrogenase (LDH) levels, a marker of red blood cell destruction, showed a mean reduction of 53.59% in the 200mg twice daily group and 25.56% in the 50mg/200mg twice daily group at Day 92. Overall, the mean reduction in LDH was 43.58%. Hemoglobin levels showed variable changes, with some patients experiencing increases up to 38.42 g/L in the 200mg twice daily group and up to 56.83 g/L in the 50mg/200mg twice daily group. Two participants in each dose cohort required on-study transfusions of packed red blood cell units.
Immune Thrombocytopenia (ITP)
A basket study () also included a cohort of CAD patients. All 9 participants (100%) in this cohort experienced an increase in hemoglobin levels from baseline. The median time to the first hemoglobin level increase of at least 1.5 g/dL was 29.0 days, with a median duration of 56.0 days for sustained hemoglobin levels at or above this increase without rescue therapy. LDH levels decreased by a mean of 277.83 U/L, and total bilirubin decreased by a mean of 15.63 µmol/L. A total of 5 participants (approximately 56%) achieved a clinically meaningful response. Rescue therapy was needed by 9 participants during Part A of the study and 1 participant overall.
Currently Recruiting Trials
Iptacopan is currently being investigated in several clinical trials for a range of conditions, offering opportunities for patients to contribute to medical research. These studies aim to understand how iptacopan works, its effectiveness, and its safety profile in different patient populations.
- A Phase 3 study, NCT06994845, is assessing iptacopan in pediatric patients aged 2 to under 18 years with primary Immunoglobulin A Nephropathy (IgAN). This open-label study, sponsored by Novartis Pharmaceuticals, will evaluate how the drug reduces proteinuria, its pharmacokinetics, and its safety in approximately 31 young participants.
- For adult patients with IgAN, a Phase 2 study, NCT06797518, is exploring the impact of iptacopan on underlying kidney changes. This study aims to provide insights into the treatment's effects on key clinical and molecular markers over 9 months, with an enrollment target of 20 participants.
- Patients with generalized Myasthenia Gravis (gMG) may be eligible for a Phase 3 study, NCT06517758. This randomized, double-blind, placebo-controlled trial will investigate the efficacy, safety, and tolerability of iptacopan in 146 participants aged 18 to 85 years who are already on stable standard of care treatment.
- A Phase 3 study, NCT05935215, is evaluating the efficacy and safety of switching from anti-C5 antibody treatment to iptacopan (200 mg twice daily) in 50 participants with Atypical Hemolytic Uremic Syndrome (aHUS).
- For individuals with IC-MPGN, a Phase 3 study, NCT05755386, is recruiting to assess the efficacy and safety of iptacopan (200mg twice daily) compared to a placebo. This randomized, double-blind study aims to enroll 106 participants.
- A Phase 2 study, NCT05268289, is investigating iptacopan in participants with active Lupus Nephritis Class III-IV, +/- V. This two-part study will evaluate the efficacy, safety, and tolerability of iptacopan in addition to standard of care treatment, with a target enrollment of 240 participants.
- Finally, a Phase 3 study, NCT04817618, is examining the efficacy and safety of iptacopan (200mg) in patients with C3 Glomerulopathy (C3G). This randomized, double-blind, placebo-controlled study includes an adolescent cohort and aims to enroll 98 participants.
Where to Participate
Clinical trials for iptacopan are actively recruiting across a wide geographic area, making participation accessible to many patients. These studies are currently being conducted at 75 sites across 62 cities in 31 states.
Some of the top locations with multiple recruiting sites include:
- Philadelphia, Pennsylvania
- Boston, Massachusetts
- Houston, Texas
- Aurora, Colorado
- Los Angeles, California
- Lawrenceville, Georgia
- Sylmar, California
- Riverview, Florida
- Temple, Texas
- Dallas, Texas
Eligibility for these trials generally includes individuals aged 2 to 100 years, of all genders. Healthy volunteers are not being recruited for these specific studies, as they focus on patients with the conditions being investigated. Children are eligible to participate in certain trials.
Development Timeline
The journey of iptacopan began with its first clinical trial on February 20, 2018, marking the start of its extensive development. Since then, the primary sponsor, Novartis Pharmaceuticals, has largely driven the research, overseeing 18 of the 20 total trials. The development program has grown significantly, with a total enrollment of 1,602 participants across all studies.
Initially, iptacopan's development focused on conditions like IBS-C and hyperphosphatemia. However, the pipeline quickly expanded to explore its potential in a broader range of diseases. This expansion included rare blood disorders such as Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS), and Cold Agglutinin Disease (CAD), as well as various kidney conditions like C3 Glomerulopathy (C3G), Immunoglobulin A Nephropathy (IgAN), and Lupus Nephritis. The drug's therapeutic reach also extended to autoimmune disorders like Generalized Myasthenia Gravis and Immune Thrombocytopenia (ITP).
The development has progressed through various stages, with 9 studies reaching Phase 3, 6 in Phase 2, and 1 in Phase 1, demonstrating a robust and advancing research program. The latest trial is projected to conclude on January 16, 2026, indicating ongoing commitment to understanding iptacopan's full potential.