Fabhalta (iptacopan)
Research Synopsis
- * Recent studies indicate that iptacopan effectively reduces hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH), particularly those who do not respond to eculizumab therapy.
- * In a phase 2 trial, patients using iptacopan displayed significant reductions in lactate dehydrogenase levels (by over 60%) and improved hemoglobin concentrations without severe adverse effects.
- * Iptacopan is being evaluated for its efficacy in treating complement 3 glomerulopathy (C3G) and IgA nephropathy (IgAN), with promising preliminary results in reducing proteinuria and enhancing kidney function.
- * Ongoing clinical studies such as the APPEAR-C3G and APPLAUSE-IgAN trials are focusing on comparing iptacopan against placebo to assess its safety and effectiveness in these kidney disorders.
- * The drug's absorption and metabolism were confirmed in an ADME study, with high absorption rates and manageable half-life, marking it a promising candidate for various conditions.
- * Iptacopan's role in addressing eculizumab-refractory PNH and other rare anemias has garnered attention, with multi-center trials suggesting individualized treatment plans for better patient outcomes.
- * The safety profile of iptacopan is consistently reported as positive, with manageable side effects and no serious complications, reinforcing its potential as a preferred treatment option.
- * Research continues to explore the broader implications of complement system targeting, indicating a shift towards more personalized therapeutic approaches for immune-mediated kidney diseases and hemolytic conditions.
- * Iptacopan stands out in the landscape of emerging treatments, offering hope for patients resisting traditional therapies and enhancing future clinical strategies in hemolytic anemia and kidney diseases.
Fabhalta (iptacopan)
[Novel anti-complement therapeutics for hemolytic anemia].
London, UK
2 hours ago
1 Received
- Eculizumab was the first anti-complement drug approved in 2007 to help treat a blood disorder called PNH, and now it can help with other diseases too.
- An improved version, called ravulizumab, allows patients to get treatment every 2 to 8 weeks instead of more often, making life easier.
- New treatments like pegcetacoplan, iptacopan, danicopan, and sutimlimab are being developed or approved to help with different types of blood issues, focusing on hemolytic anemia.
$100 - $150
Hourly Rate
Rise of the planet of rare anemias: An update on emerging treatment strategies.
London, UK
2 hours ago
1 Received
- There are new treatments being developed for rare types of anemia and other blood disorders, helping patients need fewer blood transfusions and feel better.
- Some treatments, like gene therapy and new medications, are showing promise, but scientists still need to make sure they are safe for patients in the long run.
- New drugs are being created to target specific problems in different types of anemia, which could lead to better care and help doctors find the right treatment for each patient.
$100 - $150
Hourly Rate
Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single-arm, phase 2, proof-of-concept trial.
London, UK
2 hours ago
1 Received
- The study investigates the effectiveness of iptacopan, a new complement factor B inhibitor, in treating patients with paroxysmal nocturnal haemoglobinuria who are still experiencing active hemolysis after standard anti-C5 therapy.
- It enrolled 10 adult patients showing signs of active hemolysis despite receiving eculizumab, and they were given iptacopan orally at a dose of 200 mg twice daily for 13 weeks.
- Findings demonstrated a significant reduction in lactate dehydrogenase levels, indicating iptacopan's potential for reducing hemolysis in these patients.
$100 - $150
Hourly Rate
Paroxysmal nocturnal hemoglobinuria: advances in the understanding of pathophysiology, diagnosis, and treatment.
London, UK
2 hours ago
1 Received
- Recent advancements in the understanding of paroxysmal nocturnal hemoglobinuria (PNH) have improved diagnostics and therapies, focusing on the unique survival of PNH stem cells against cell death.
- Changes in immune-related proteins (cytokines and chemokines) among PNH patients suggest a link to autoimmune processes and cell death mechanisms.
- The review discusses current diagnostic methods, treatment options (like C5 inhibitors and stem cell transplantation), and introduces new experimental drugs, emphasizing the significance of tailored treatment plans for better disease outcomes.
$100 - $150
Hourly Rate
Complement System as a New Target for Hematopoietic Stem Cell Transplantation-Related Thrombotic Microangiopathy.
London, UK
2 hours ago
1 Received
- Thrombotic microangiopathy (TMA) can occur after stem cell transplants, known as transplant-associated thrombotic microangiopathy (TA-TMA), but its causes and effective diagnosis are still unclear, leading to a high risk of mortality.
- Key symptoms of TA-TMA include low platelet levels, hemolysis, and organ damage, especially to the kidneys, which can also cause high blood pressure, but diagnosing it is complicated by other potential health issues.
- Recent research indicates that the complement system plays a role in TA-TMA, suggesting that complement inhibition therapy might help some patients, particularly if they show clear signs of complement activation; two medications, eculizumab and narsoplimab, have
$100 - $150
Hourly Rate
Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label proof-of-concept study.
London, UK
2 hours ago
1 Received
- Iptacopan (LNP023) is being tested as a new oral treatment for paroxysmal nocturnal hemoglobinuria (PNH) by inhibiting complement factor B and is currently in a phase 2 clinical study.
- In the study, PNH patients were given different doses of iptacopan, and results showed significant reductions in serum lactate dehydrogenase (LDH) levels, with most patients seeing a drop of over 60% by week 12.
- The drug was well tolerated, leading to notable improvements in hemoglobin levels and reductions in other hemolysis markers, with no severe adverse events reported during the trial.
$100 - $150
Hourly Rate
Alternative Complement Pathway Inhibition With Iptacopan for the Treatment of C3 Glomerulopathy-Study Design of the APPEAR-C3G Trial.
London, UK
2 hours ago
1 Received
- Complement 3 glomerulopathy (C3G) is a rare kidney disease linked to issues with the alternative pathway of the complement system, often leading to kidney failure within a decade of diagnosis, and there are currently no approved treatments.
- Iptacopan is a new oral medication that inhibits factor B in the complement system and showed promise in reducing proteinuria in initial studies.
- The ongoing Phase III APPEAR-C3G study is evaluating the safety and effectiveness of iptacopan by comparing it to a placebo in 68 adults over a 12-month period, with a focus on reducing protein levels in urine and improving kidney function.
$100 - $150
Hourly Rate
The first successful expanded compassionate use of Iptacopan in a patient with paroxysmal nocturnal hemoglobinuria.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Targeting the Alternative Complement Pathway With Iptacopan to Treat IgA Nephropathy: Design and Rationale of the APPLAUSE-IgAN Study.
London, UK
2 hours ago
1 Received
- A Phase 3 study called APPLAUSE-IgAN is testing iptacopan, a drug that targets the alternative complement pathway, as a treatment for immunoglobulin A nephropathy (IgAN) in patients at high risk of kidney failure.
- About 450 adult patients will participate, receiving either iptacopan or a placebo for 24 months, while already on optimal treatment with ACE inhibitors or ARBs.
- The study aims to show that iptacopan reduces proteinuria and slows the decline in kidney function, with additional evaluations on patient-reported outcomes and safety.
$100 - $150
Hourly Rate
Emerging role of monoclonal antibodies in the treatment of IgA nephropathy.
London, UK
2 hours ago
1 Received
- IgA nephropathy is the most common type of primary glomerulonephritis, leading to kidney damage due to immune complex deposits, and currently has very limited treatment options.
- The article explores potential therapies using monoclonal antibodies aimed at disrupting the disease's underlying mechanisms, focusing on immune responses that produce harmful antibodies.
- Experts suggest that new treatments could effectively target B and T cells and their supporting factors to improve outcomes for patients with IgA nephropathy.
$100 - $150
Hourly Rate
Treatment of eculizumab refractory paroxysmal nocturnal hemoglobinuria: A systematic review about current treatment options and future direction.
London, UK
2 hours ago
1 Received
- Eculizumab is a primary treatment for paroxysmal nocturnal hemoglobinuria (PNH), but some patients may become resistant to it, leading to a condition known as eculizumab refractory PNH.
- The study systematically reviewed 70 relevant studies on treatments for eculizumab refractory PNH, ultimately identifying four multicenter clinical trials that focused on drugs like pegcetacoplan, danicopan, and iptacopan.
- The findings suggest that treatment plans should be tailored to individual patient needs, and further randomized controlled trials are necessary to improve treatment guidelines for eculizumab refractory PNH.
$100 - $150
Hourly Rate
Absorption, Distribution, Metabolism, and Excretion of [C]iptacopan in Healthy Male Volunteers and in In Vivo and In Vitro Studies.
London, UK
2 hours ago
1 Received
- Iptacopan (LNP023) is an orally-administered potent drug designed to inhibit factor B in the alternative complement pathway, currently being developed for conditions like paroxysmal nocturnal hemoglobinuria.
- A study examining the absorption, distribution, metabolism, and excretion (ADME) of Iptacopan in six healthy volunteers found it to be 71% absorbed with a peak concentration reached in 1.5 hours and a plasma half-life of 12.3 hours.
- The elimination of Iptacopan is primarily through liver metabolism, with findings showing a significant amount excreted in feces (71.5%) and urine (24.8%), indicating low systemic exposure to potentially
$100 - $150
Hourly Rate
Design and Rationale of the APPELHUS Phase 3 Open-Label Study of Factor B Inhibitor Iptacopan for Atypical Hemolytic Uremic Syndrome.
London, UK
2 hours ago
1 Received
- * Iptacopan (LNP023) is an oral medication that targets the complement pathway and has shown promise in earlier studies for other conditions, indicating potential benefits for patients with aHUS.
- * The APPELHUS study is a Phase 3 trial to evaluate the safety and effectiveness of iptacopan in 50 patients with aHUS, aiming to assess how well it can resolve symptoms without needing other standard therapies during a 26-week treatment period.
$100 - $150
Hourly Rate
Future landscape for the management of membranous nephropathy.
London, UK
2 hours ago
1 Received
- However, about 20%-30% of patients may still face treatment-resistant forms of MN, highlighting the need for new therapeutic strategies.
- Promising new treatments under investigation include novel anti-CD20 agents, anti-CD38 therapies, and innovative techniques used primarily in cancer treatment, paving the way for a more precise approach to managing MN in the future.
$100 - $150
Hourly Rate
Phase 3 trial results bring hope for patients with IgA nephropathy.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Novel therapeutics and future directions for refractory immune thrombocytopenia.
London, UK
2 hours ago
1 Received
- Immune thrombocytopenia (ITP) is an autoimmune disorder affecting blood platelet levels, with about 1 in 20,000 people impacted, and some patients are resistant to standard therapies, known as refractory ITP.
- There is a need for new treatments, with several novel agents currently in clinical trials, targeting new mechanisms in ITP that current medications do not address.
- The manuscript discusses these promising therapeutics, their development stages, innovative trial designs, the importance of assessing patients' quality of life, and weighing drug benefits against potential side effects.
$100 - $150
Hourly Rate
A narrative review of potential drug treatments for nephritis in children with IgA vasculitis (HSP).
London, UK
2 hours ago
1 Received
- Immunoglobulin A (IgA) vasculitis (IgAV), also known as Henoch-Schoenlein purpura, is the most common childhood vasculitis that usually resolves on its own, but can lead to significant kidney issues (IgAV-N) in some patients.
- Current treatments focus on established kidney involvement, but there's a push for early intervention to prevent inflammation before serious kidney damage occurs.
- Research is ongoing to identify new therapeutic targets and drugs aimed at preventing kidney problems in children with IgAV-N, as current options like corticosteroids have proven ineffective for this purpose.
$100 - $150
Hourly Rate
Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.
London, UK
2 hours ago
1 Received
- Complement 3 glomerulopathy (C3G) is a rare kidney disease caused by issues with the immune system, and iptacopan is being tested as a potential treatment in a study examining its effectiveness and safety.
- In a phase 2 clinical trial with 27 patients, those with native C3G saw a 45% reduction in protein in their urine, while kidney transplant recipients had a significant decrease in C3 deposits in their biopsies after 84 days of treatment.
- Overall, iptacopan showed promising results in improving kidney function and safety, as it normalized certain complement levels and had manageable side effects, with no reported deaths during the study.
$100 - $150
Hourly Rate
Targeting complement in IgA nephropathy.
London, UK
2 hours ago
1 Received
- Immunoglobulin A nephropathy (IgAN) is the most common type of kidney inflammation globally, with recent research highlighting the critical role of the complement system, especially the alternative pathway, in its progression.
- Studies indicate that specific proteins like factor H-related proteins and the lectin pathway contribute to disease severity, and glomerular deposition of markers like C3 and C4d may predict worse outcomes.
- The understanding of complement's involvement in IgAN has led to the development of new treatments targeting various components of the complement system, with several clinical trials currently underway to assess their effectiveness.
$100 - $150
Hourly Rate
Results of a randomized double-blind placebo-controlled Phase 2 study propose iptacopan as an alternative complement pathway inhibitor for IgA nephropathy.
London, UK
2 hours ago
1 Received
- Targeting the alternative complement pathway could be a promising treatment approach for immunoglobulin A nephropathy (IgAN), with iptacopan being a new oral drug that inhibits this pathway by specifically targeting Factor B.
- A Phase 2 study tested different doses of iptacopan on IgAN patients and found a significant reduction in urine protein levels after three months, particularly with the 200 mg twice daily dosage.
- Iptacopan was also safe and well-tolerated, showing no serious side effects, which supports its further investigation in an ongoing Phase 3 trial.
$100 - $150
Hourly Rate