^aIncludes similar terms.   
^bNasopharyngitis contains: rhinitis allergic, upper respiratory tract infection, pharyngitis, rhinitis.    
^cBacterial infection contains: pyelonephritis, urinary tract infection, bronchitis bacterial, bronchitis haemophilus, cholecystitis, folliculitis, cellulitis, arthritis bacterial, sepsis, klebsiella infection, staphylococcal infection, Pseudomonas infection, hordeolum, pneumonia bacterial.   
^dViral infection contains: COVID-19, herpes zoster, oral herpes, nasal herpes, influenza A virus test positive, influenza. 
^eLipid disorder contains: dyslipidemia, blood cholesterol increased, low-density lipoprotein increased, hypercholesterolemia, blood triglycerides increased, hyperlipidemia.  
 

• Serious adverse reactions were reported in 2 (3%) patients with PNH who received FABHALTA. They included pyelonephritis, urinary tract infection, and COVID-19

• Rash was reported in 2 patients (3%)

• Of the 37 FABHALTA-treated patients who had normal platelet counts at baseline, 43% experienced any grade thrombocytopenia during the randomized treatment period

• Three FABHALTA-treated patients experienced decreased platelets that worsened to grade ≥3 from baseline (1 patient with normal platelets that worsened to grade 4; 1 patient with baseline grade 1 that worsened to grade 4; and 1 patient with baseline grade 3 that worsened to grade 4)

No patient discontinued FABHALTA or C5is due to an adverse reaction during the 24-week randomized treatment period. One patient discontinued FABHALTA due to pregnancy.²

Patients on FABHALTA experienced an annualized rate of clinical breakthrough hemolysis of 0.07 and 0.67 with C5is^2,*,✝

The data from this additional analysis are descriptive in nature, presented for observation only. No formal conclusions or comparisons between the 2 treatment arms can be made. The planned power for the hypothesis testing was low.

• During the 24-week randomized treatment period, 2/62 patients (0.07 annualized rate) experienced clinical BTH with FABHALTA vs 6/35 (0.67 annualized rate) with C5is (eculizumab or ravulizumab) (95% CI, 0.02-0.61) 

There was no significant statistical difference in the annualized rates of major adverse vascular events (MAVEs)^‡ between FABHALTA and C5is (0.03 vs 0, respectively; 95% CI, -0.03-0.10)^2,† 

The data from this additional analysis are descriptive in nature, presented for observation only. No formal conclusions or comparisons between the 2 treatment arms can be made. 

• During the 24-week randomized treatment period, 1 patient experienced transient ischemic attack in the FABHALTA arm, which was deemed unrelated to treatment by the investigator, vs 0/35 with C5is (eculizumab or ravulizumab)

*Clinical BTH was defined as meeting clinical criteria (either decrease of Hb level ≥2 g/dL compared with the last assessment or within 15 days; or signs or symptoms of gross hemoglobinuria, painful crisis, dysphagia, or any other significant clinical PNH-related signs and symptoms) and laboratory criteria (LDH >1.5 x ULN and increased as compared with the last 2 assessments).^2
†Assessed between Day 1 and 168.^2
‡The definition of MAVEs included: acute peripheral vascular occlusion, amputation, cerebrovascular accident, dermal thrombosis, gangrene, hepatic/portal vein thrombosis, mesenteric/visceral arterial thrombosis, myocardial infarction, pulmonary embolus, renal arterial or vein thrombosis, thrombophlebitis/deep vein thrombosis, transient ischemic attack, and unstable angina.²

^aIncludes similar terms.  
^bNasopharyngitis contains: rhinitis allergic, upper respiratory tract infection, pharyngitis, rhinitis.   
^dViral infection contains: COVID-19, herpes zoster, oral herpes, nasal herpes, influenza A virus test positive, influenza.
^eLipid disorder contains: dyslipidemia, blood cholesterol increased, low-density lipoprotein increased, hypercholesterolemia, blood triglycerides increased, hyperlipidemia.          
^fRash contains: dermatitis allergic, acne, erythema multiforme, rash maculopapular, rash erythematous.      
       

• Serious adverse reactions were reported in 2 (5%) patients with PNH who received FABHALTA. They included COVID-19 and bacterial pneumonia

• Bacterial infection^c and nausea were reported in 2 patients each (5%). Dizziness and urticaria were reported in 1 patient each (3%)

^cBacterial infection contains: pyelonephritis, urinary tract infection, bronchitis bacterial, bronchitis haemophilus, cholecystitis, folliculitis, cellulitis, arthritis bacterial, sepsis, klebsiella infection, staphylococcal infection, Pseudomonas infection, hordeolum, pneumonia bacterial.

ADDITIONAL END POINTS

The data from this additional analysis are exploratory; therefore, not subject to family-wise Type 1 error control, and presented for observation only. No formal conclusions can be made.

During the 24-week core treatment period, no clinical BTH* or MAVEs^‡ were observed in patients on FABHALTA⁵

*Clinical BTH was defined as meeting clinical criteria (either decrease of Hb level ≥2 g/dL compared with the latest assessment or within 15 days; or signs or symptoms of gross hemoglobinuria, painful crisis, dysphagia, or any other significant clinical PNH-related signs and symptoms) and laboratory criteria (LDH >1.5 x ULN and increased as compared with the last 2 assessments).^6  
‡The definition of MAVEs included: acute peripheral vascular occlusion, amputation, cerebrovascular accident, dermal thrombosis, gangrene, hepatic/portal vein thrombosis, mesenteric/visceral arterial or venous thrombosis or infarction, myocardial infarction, pulmonary embolus, renal arterial or vein thrombosis, thrombophlebitis/deep vein thrombosis, transient ischemic attack, and unstable angina.^6
¶BTH and MAVEs were reported from the start of FABHALTA Day 1 to 336.⁴