An Extension Study of RPC1063 as Therapy for Moderate to Severe Ulcerative Colitis

Number of participants experiencing TEAEs, Serious TEAEs, TEAEs leading to discontinuation and TEAEs of special interest. TEAE is defined as any event with an onset date on or after the first dose date, or any ongoing event on the first dose date that worsens in severity on or after the first dose date, and until 90 days following the last dose of treatment with the study drug.

Clinical Remission is defined as Rectal bleeding subscore=0; stool frequency subscore <=1 (and a decrease of >=1 point from the baseline stool frequency subscore); and endoscopy subscore <=1.

Rectal bleeding 0 = No blood seen Stool frequency 0 = Normal number of stools for this patient; 1 = 1 to 2 stools more than normal Endoscopy 0 = Normal or inactive disease; 1 = Mild disease (erythema, decreased vascular pattern, does not include friability).

Clinical response is defined as reduction from baseline in the 9-point Mayo score of >=2 points and reduction from baseline in the 9-point Mayo score >=35%, and (reduction from baseline in the rectal bleeding subscore of >=1 point or a rectal bleeding subscore of <=1 point). 9 point Mayo score is defined as the sum of rectal bleeding subscore, stool frequency subscore, and the endoscopy subscore each ranging from (0=normal activity-3=worse activity) for a total score that ranges from 0 to 9 with higher score indicating worsening symptoms.

Endoscopic Improvement is defined as endoscopy subscore of <=1 point. 0 = Normal or inactive disease;

• = Mild disease (erythema, decreased vascular pattern, does not include friability)
• = Moderate disease (marked erythema, lack of vascular pattern, friability, erosions)
• = Severe disease (spontaneous bleeding, ulceration)

Corticosteroid-free remission is defined as clinical remission while off corticosteroids for ≥12 weeks. Clinical Remission is defined as Rectal bleeding subscore=0; stool frequency subscore <=1 (and a decrease of >=1 point from the baseline stool frequency subscore); and endoscopy subscore <=1.

Rectal bleeding 0 = No blood seen Stool frequency 0 = Normal number of stools for this patient; 1 = 1 to 2 stools more than normal Endoscopy 0 = Normal or inactive disease; 1 = Mild disease (erythema, decreased vascular pattern, does not include friability).

Histologic remission is defined as Geboes index score < 2.0. The Geboes score is a validated histological grading system for UC that ranges from 0=no disease activity to 5=high disease activity.

Mucosal Healing is defined as Endoscopy subscore of ≤ 1 point (0 = Normal or inactive disease; 1 = Mild disease (erythema, decreased vascular pattern, does not include friability)) and a Geboes index score < 2.0 (The Geboes score is a validated histological grading system for UC that ranges from 0=no disease activity to 5=high disease activity.)

The Complete Mayo Score is a composite of four assessments, each rated from 0 to 3:

• Stool frequency: (0 = Normal number of stools for this patient; 1 = 1 to 2 stools more than normal; 2 = 3 to 4 stools more than normal; 3 = 5 or more stools more than normal)
• Rectal bleeding (0 = No blood seen; 1 = Streaks of blood with stool less than half the time; 2 = Obvious blood with stool most of the time; 3 = Blood alone passes)
• endoscopy (0 = Normal or inactive disease; 1 = Mild disease; 2 = Moderate disease; 3 = Severe disease)
• Physician's Global Assessment (0 = Normal; 1 = Mild disease; 2 = Moderate disease; 3 = Severe disease).

The total score range is from 0-12, with higher score indicating worse disease activity. Baseline is the last measurement collected on or prior to the date of first dose in the 3102 OLE study.

The Partial Mayo Score is a composite of three assessments, each rated from 0 to 3:

• Stool frequency: (0 = Normal number of stools for this patient; 1 = 1 to 2 stools more than normal; 2 = 3 to 4 stools more than normal; 3 = 5 or more stools more than normal)
• Rectal bleeding (0 = No blood seen; 1 = Streaks of blood with stool less than half the time; 2 = Obvious blood with stool most of the time; 3 = Blood alone passes)
• Physician's Global Assessment (0 = Normal; 1 = Mild disease; 2 = Moderate disease; 3 = Severe disease).

The total score range is from 0-9, with higher score indicating worse disease activity. Baseline is the last measurement collected on or prior to the date of first dose in the 3102 OLE study.

The 9-point Mayo Score is a composite of three assessments, each rated from 0 to 3:

• Stool frequency: (0 = Normal number of stools for this patient; 1 = 1 to 2 stools more than normal; 2 = 3 to 4 stools more than normal; 3 = 5 or more stools more than normal)
• Rectal bleeding (0 = No blood seen; 1 = Streaks of blood with stool less than half the time; 2 = Obvious blood with stool most of the time; 3 = Blood alone passes)
• Endoscopy (0 = Normal or inactive disease; 1 = Mild disease; 2 = Moderate disease; 3 = Severe disease) The total score range is from 0-9, with higher score indicating worse disease activity. Baseline is the last measurement collected on or prior to the date of first dose in the 3102 OLE study.

Clinical Remission is defined as Rectal bleeding subscore=0; stool frequency subscore <=1 (and a decrease of >=1 point from the baseline stool frequency subscore); and endoscopy subscore <=1.

Rectal bleeding 0 = No blood seen Stool frequency 0 = Normal number of stools for this patient; 1 = 1 to 2 stools more than normal Endoscopy 0 = Normal or inactive disease; 1 = Mild disease (erythema, decreased vascular pattern, does not include friability).

Clinical response is defined as reduction from baseline in the 9-point Mayo score of >=2 points and reduction from baseline in the 9-point Mayo score >=35%, and (reduction from baseline in the rectal bleeding subscore of >=1 point or a rectal bleeding subscore of <=1 point). 9 point Mayo score is defined as the sum of rectal bleeding subscore, stool frequency subscore, and the endoscopy subscore each ranging from (0=normal activity-3=worse activity) for a total score that ranges from 0 to 9 with higher score indicating worsening symptoms.

Endoscopic Improvement is defined as endoscopy subscore of <=1 point. 0 = Normal or inactive disease;

1 = Mild disease (erythema, decreased vascular pattern, does not include friability)