The ConFIrm and ConFIdence studies
In July 2021 we commenced patient enrollment in the screening (CFI-001) and natural history of disease (CFI-002) studies to assess CFI blood levels in patients who have diseases related to CFI deficiency and identify those who would benefit from CB 4332 treatment (“ConFIrm” and “ConFIdence”, respectively). As of February 2022 we have completed enrollment of these studies.
Factor I is probably the most relevant down-regulator in the complement system
This system is composed of a series of plasma proteins and plays a critical role in host defense and inflammation. CFI deficiency causes a permanent, uncontrolled activation of the alternative pathway resulting in increased turnover and consumption of other complement factors resulting in a dysfunctional complement system and affecting its beneficial biological functions .
Catalyst plans to develop a recombinant CFI protein (CB 4332) which eventually could be used to restore the missing CFI levels and rebalance complement function addressing the root cause of the disease. The current standard of care treatment in people with such conditions addresses the symptoms of the disease but not the root cause. It consists of preventative vaccinations, prophylactic antibiotics, immunoglobulins, immunomodulators, or immunosuppressors. Most of these therapies are not devoid of relevant adverse events.