Shots:

• The P-III (APPLY-PNH) trial evaluating iptacopan monothx. (200mg, BID) vs eculizumab or ravulizumab in a ratio (8:5) in 97 adult patients with PNH with residual anemia
• The trial met its two 1EPs & showed iptacopan was superior to anti-C5 therapies with Hb level increases of ≥2g/dL from baseline without needing blood transfusions @24wks. while ≥12g/dL @24wks., was well tolerated with a favorable safety profile consistent with prior reported data
• The therapy is being evaluated in P-III (APPOINT-PNH) trial for PNH with expected results in the coming months & is being studied in P-III (APPEAR-C3G) trial for CMKDs C3 glomerulopathy, (APPLAUSE-IgAN) for IgA nephropathy and (APPELHUS) for aHUS along with additional indication in P-II study

Ref: Novartis | Image: Novartis