Cold agglutinin disease (CAD) is a clonal B-cell lymphoproliferative disorder and a rare subgroup of autoimmune hemolytic anemia (AIHA). CAD accounts for 15-25% of AIHA and affects mainly middle aged or older individuals. The disease is characterized by the production of autoantibodies by bone marrow-derived clonal B cells which attach to erythrocytes when exposed to cold temperatures (0-4°C), causing agglutination and eventual immune-mediated hemolysis.
Key Takeaways
Key Takeaways
- There are only three industry-sponsored drugs in active clinical development for cold agglutinin disease, with one drug each in Phase I, Phase II, and the NDA/BLA phase.
- Therapies in development for cold agglutinin disease target the complement pathway. These pipeline drugs are administered via the intraocular, subretinal, subconjunctival, intravenous, and subcutaneous routes.
- The overall likelihood of approval of a Phase I marrow or peripheral blood stimulator asset is 22.5%, and the average probability a drug advances from Phase III is 82.5%. Drugs, on average, take 10.1 years from Phase I to approval, compared to 9.7 years in the overall hematology space.
- The distribution of clinical trials across Phase I-III indicates that the majority of trials for cold agglutinin disease have been in the early and mid-phases of development, with 70% of trials in Phase I-II, and 30% in Phase III.
- Sanofi has two ongoing trials in cold agglutinin disease.
- Sanofi leads industry sponsors with the highest overall number of clinical trials for cold agglutinin disease.
Table of Contents
OVERVIEWKEY TAKEAWAYSEPIDEMIOLOGYPIPELINE DRUGSPROBABILITY OF SUCCESSBIBLIOGRAPHYAPPENDIX
DISEASE BACKGROUND
TREATMENT
RECENT EVENTS AND ANALYST OPINION
CLINICAL TRIAL LANDSCAPE
LIST OF FIGURES
LIST OF TABLES
