CB 4332

CB 4332 is a wholly-owned first-in-class improved CFI intended for lifelong prophylactic SQ administration in individuals with CFI deficiency.

In the CB 4332, enhanced CFI program, we intend to commence enrollment of an observational trial in mid-2021 to measure CFI activity and genotype patients who have diseases related to CFI deficiency in order to identify those who would benefit from CB 4332 treatment. This will prepare us for a P1/2 clinical study of CB 4332 in 2022. CB 4332 is designed to restore the normal complement system in patients with dysregulated or aberrant CFI. There are currently no approved options for protein replacement therapy in CFI deficiency, nor are there any in clinical development. We estimate that the market for CB 4332 in patients with atypical hemolytic uremic syndrome (“aHUS”), complement 3 glomerulopathy (“C3G”) and immune complex mediated membranoproliferative glomerulonephritis (“IC-MPGN”) is approximately $500 million, with additional potential opportunities for patients who are CFI deficient with indications outside of nephrology, such as to prevent severe infections and recurrent inflammatory histopathologies. The size of the market for these potential opportunities is estimated to be approximately $12.0 billion by 2026. This patient population is likely significantly underdiagnosed and appears to be growing as more awareness about the clinical manifestation of complete CFI deficiency emerge. There are a number of dry AMD patients who have genetic abnormalities in their CFI gene that may be an important driver of their disease.