Clinical Trials

CB 4332

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.

The ConFIrm study – Screening study to identify patients with CFI deficiencies

Catalyst’s ConFIrm study tested for CFI in people who experience signs and symptoms of CFI deficiency. The study had a global footprint to be able to reach out to as many patients as possible and contribute to revealing undiagnosed disease. Screening for CFI deficiency only required providing a blood sample, in which the presence and function of the CFI protein were assessed.

The ConFIdence study – Natural history study to assess the clinical outcomes of patients with CFI deficiency

The study followed and observed patients identified in the ConFIrm screening study to assess their clinical progression and better understand this varied disease.

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.