Veopoz (pozelimab)
Research Synopsis
- - Veopoz (pozelimab) is a human monoclonal antibody developed to inhibit complement component C5, which is critical in treating diseases related to excessive complement activation, notably paroxysmal nocturnal hemoglobinuria (PNH) and CD55-deficient protein-losing enteropathy (CHAPLE disease).
- - The drug was first approved by the FDA in August 2023 for use in patients aged one year and older diagnosed with CHAPLE disease, marking a significant advancement in therapy for this rare condition.
- - Recent studies have demonstrated that pozelimab effectively binds to C5, preventing hemolysis and improving C5 levels, resulting in reduced toxicity for patients transitioning from other therapies.
- - Research on pozelimab in combination with cemdisiran, another treatment targeting C5 production, has shown promising results in non-human primates, suggesting enhanced and prolonged inhibition of complement activity compared to individual treatments.
- - Further investigations into the efficacy and safety of pozelimab in CHAPLE patients indicated significant clinical improvements, with many participants reporting normalization of serum albumin levels after treatment.
- - Pozelimab has received orphan drug designations for additional conditions including PNH and myasthenia gravis, indicating its potential applicability across various complement-mediated diseases.
- - The pharmacokinetics of pozelimab vary significantly with patient weight, necessitating a tailored dosage model that optimizes efficacy, especially for pediatric patients undergoing treatment for CHAPLE.
- - Ongoing clinical trials and patient interviews underscore pozelimab's effectiveness in alleviating core symptoms associated with CHAPLE disease, emphasizing the importance of mixed-method approaches in rare disease research.
- - The emerging data positions pozelimab as a cornerstone in treatment for complement pathway diseases, highlighting the potential for combining it with other therapies to enhance patient outcomes further.
- - Pozelimab's development reflects a significant shift towards targeted therapies in immunology, promising better disease management for patients suffering from rare and challenging conditions.
Veopoz (pozelimab)
Inhibition of complement pathway activation with Pozelimab, a fully human antibody to complement component C5.
London, UK
2 hours ago
1 Received
- Complement is a crucial part of the immune system, and improper activation can lead to various diseases; targeting complement component 5 (C5) is promising for new therapies.
- The study highlights a new humanized C5 mouse model that helps assess the pharmacokinetic and pharmacodynamic properties of anti-C5 antibodies, revealing significant differences in how these antibodies clear from the system.
- Notably, the antibody pozelimab showed strong binding to C5 and effectively prevented hemolysis in clinical settings, resulting in improved C5 levels and reduced toxicity when switching from another treatment.
$100 - $150
Hourly Rate
Pharmacokinetics and pharmacodynamics of pozelimab alone or in combination with cemdisiran in non-human primates.
London, UK
2 hours ago
1 Received
- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease linked to uncontrolled complement activation, and current treatments target this process with terminal complement inhibition.
- This study evaluated the effectiveness of combining cemdisiran, an RNAi therapy that reduces C5 production, and pozelimab, a monoclonal antibody against C5, in non-human primates.
- Results showed that the combination therapy provided longer-lasting and more effective inhibition of complement activity compared to each treatment used alone, suggesting its potential as a long-term treatment option for PNH.
$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
Characterization of multivalent complexes formed in the presence of more than one conventional antibody to terminal complement component C5.
London, UK
2 hours ago
1 Received
- - This study investigates how immune complexes form when patients are exposed to two different anti-C5 antibodies simultaneously, focusing on the transition from one treatment to another.
- - Researchers used size exclusion chromatography and multiangle light scattering to analyze the interactions between eculizumab and two other anti-C5 antibodies (TPP-2799 and TP-3544), both of which bind C5 similarly.
- - Results showed that when mixed with other antibodies, the complexes formed could exceed 1500 kDa, indicating multiple antibodies and C5 molecules interacting, suggesting the need for careful monitoring and strategies to prevent complex formation during such treatment transitions.
$100 - $150
Hourly Rate
CD55-deficiency in Jews of Bukharan descent is caused by the Cromer blood type Dr(a-) variant.
London, UK
2 hours ago
1 Received
- The CD55 regulator is linked to the Cromer blood group and its complete dysfunction leads to a severe gastrointestinal syndrome along with a risk of venous thrombosis.
- Two Bukharan Jewish patients with CD55-deficiency were found to have a specific genetic variant (c.596C>T; p.Ser199Leu) that causes incorrect splicing of the gene, resulting in low production of the CD55 protein.
- This variant is common in the Bukharan Jewish population, suggesting many cases may go undiagnosed; timely diagnosis is crucial for effective treatment with eculizumab, which can significantly improve patient outcomes.
$100 - $150
Hourly Rate
Pozelimab: First Approval.
London, UK
2 hours ago
1 Received
- Pozelimab (VEOPOZ™) is a human monoclonal antibody designed by Regeneron Pharmaceuticals to inhibit complement factor 5 (C5), aiming to treat diseases linked to the complement pathway.
- In August 2023, it became the first FDA-approved treatment for adults and children aged 1 year and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease.
- Pozelimab has also received orphan drug designations in the U.S. for paroxysmal nocturnal hemoglobinuria (PNH) and myasthenia gravis, and it's currently in clinical trials globally for these conditions.
$100 - $150
Hourly Rate
Pozelimab-bbfg.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Antibodies to watch in 2024.
London, UK
2 hours ago
1 Received
- The 'Antibodies to Watch' series gives an annual overview of monoclonal antibody therapeutics that are either in late-stage development, undergoing regulatory review, or have recently been approved, focusing on 16 antibodies granted approval in 2023.
- It also highlights nearly 50 additional product candidates that are either awaiting approval or expected to enter review by the end of 2024, featuring innovative types like bispecific antibodies and antibody-drug conjugates.
- Finally, the article discusses the clinical approval success rates of these antibody therapeutics, which range from 14-32%, indicating that the biopharmaceutical industry is actively advancing and finding success in developing these treatments, especially for non-cancer conditions.
$100 - $150
Hourly Rate
Evaluating the efficacy and safety of pozelimab in patients with CD55 deficiency with hyperactivation of complement, angiopathic thrombosis, and protein-losing enteropathy disease: an open-label phase 2 and 3 study.
London, UK
2 hours ago
1 Received
- CD55 deficiency with hyperactivation of complement, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE) is a rare genetic disorder that affects the intestines and causes serious complications due to excessive complement system activity.
- The study evaluated the safety and efficacy of pozelimab, an antibody that blocks a specific part of this system, in ten patients diagnosed with CHAPLE across three countries: Thailand, Türkiye, and the USA.
- Results focused on the proportion of patients whose serum albumin normalized and showed clinical improvement after 24 weeks of treatment, with assessments conducted to monitor any worsening of inactive symptoms.
$100 - $150
Hourly Rate
A new drug for rare diseases: pozelimab for CHAPLE disease.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Complement inhibition in paroxysmal nocturnal hemoglobinuria: From biology to therapy.
London, UK
2 hours ago
1 Received
- Complement inhibitors are key treatments for paroxysmal nocturnal hemoglobinuria (PNH), with eculizumab being the first drug that helped improve symptoms, though it requires lifelong intravenous infusions every two weeks.
- Ravulizumab, a newer drug, offers convenience by being administered every 8 weeks and helps improve hemolysis control, while other anti-C5 drugs and upstream inhibitors are under study and aim to address limitations of current treatments.
- Despite advancements, there remain challenges like drug adherence and managing breakthrough hemolysis (BTH) due to various physical stressors, highlighting the need for a more tailored treatment approach for PNH patients.
$100 - $150
Hourly Rate
Pozelimab, a human monoclonal immunoglobulin for the treatment of CHAPLE disease.
London, UK
2 hours ago
1 Received
- The complement is an essential part of the innate immune system, but its uncontrolled activation can lead to diseases, necessitating regulation by proteins like CD55 (decay-accelerating factor).
- Loss of CD55 results in CHAPLE disease, which causes severe symptoms such as abdominal pain, vomiting, and growth impairment, and currently has no known cure.
- Pozelimab is a specialized drug that targets the complement protein C5, recognized for its potential to treat CHAPLE disease through its strong attachment to both normal and variant C5, and has received several designations highlighting its importance in medicine.
$100 - $150
Hourly Rate
Pozelimab for CHAPLE disease: results from in-trial interviews and clinical outcome assessments.
London, UK
2 hours ago
1 Received
- CHAPLE disease is a rare and serious condition affecting fewer than 100 young people worldwide, characterized by symptoms like abdominal pain and protein loss from the intestines.
- A study used mixed methods, including interviews and clinical assessments, to evaluate the impact of pozelimab treatment on these symptoms over 24 weeks.
- Results showed that after treatment, patients experienced complete resolution of core symptoms, highlighting the value of mixed-methods in understanding patient experiences in rare disease trials.
$100 - $150
Hourly Rate
Pharmacotherapy for CD55 deficiency with CHAPLE disease: how close are we to a cure?
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Population pharmacokinetic analyses of pozelimab in patients with CD55-deficient protein-losing enteropathy (CHAPLE disease).
London, UK
2 hours ago
1 Received
- Pozelimab is the only treatment available for CD55-deficient protein-losing enteropathy (CHAPLE) in both adults and children aged 1 and above, targeting the C5 protein.
- A population pharmacokinetic (PopPK) model was created using data from four clinical trials to evaluate how the drug behaves in the body and to determine the right dosage for CHAPLE patients.
- The model concluded that body weight significantly affects the drug's performance, leading to a weight-based dosage of 10 mg/kg for pediatric patients to ensure optimal therapeutic effects.
$100 - $150
Hourly Rate