Study of Efficacy and Safety of VAY736 in Patients With Pemphigus Vulgaris

• Number of patients with adverse events [ Time Frame: up to an average of 12 months ]
  Summarized statistics on adverse events will be reported under categories such as total adverse events, serious adverse events and death
• Change in Autoimmune Skin disease Intensity Score (ABSIS) [ Time Frame: 12 weeks ]
  The effect of VAY736 on the ABSIS score will be compared betwen baseline and Week 12. ABSIS is a quality and quantity-based score of skin and oral mucosal lesions, combining the extent of the affected body surface area, the quality of the lesion and the extent of oral mucosal involvement, including food-related pain and discomfort.
• Change in Investigator Global Assessment (IGA) [ Time Frame: baseline, 12 weeks ]
  The effect of VAY736 on the IGA of a patient's disease activity will compared between baseline and Week 12. The IGA score ranges from 0 (no sign of active disease) to 4 (Severe active disease)
• VAY736 serum concentration [ Time Frame: 0, 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks ]
  The concentration of VAY736 will be measured in the serum. Descriptive statistics will include area under the concentration curve, half-life and time to maximum concentration.

• Drug: VAY736
  VAY736 will be administered to patients with mild-moderate pemphigus vulgaris in a randomized manner.
• Drug: Placebo to VAY736
  Placebo to VAY736 will be administered to patients with mild-moderate pemphigus vulgaris in a randomized manner. All patients that receive placebo will be offered open-label VAY736.
• Drug: VAY736
  VAY736 will be administered to patients with mild-moderate pemphigus vulgaris in a randomized manner, following a safety review.

• Placebo Comparator: Placebo
  matching placebo (infusion bag) to VAY736, a single dose administered intravenously over 2 hours.
  Intervention: Drug: Placebo to VAY736
• Experimental: VAY736 Dose 1
  Intravenous infusion of VAY736 at Dose Level 1, a single dose administered intravenously for 2 hours
  Intervention: Drug: VAY736
• Experimental: VAY736 Dose 2
  Intravenous infusion of VAY736 at Dose Level 2, a single dose administered intravenously over 2 hours. Dose level 2 will be initiated following a safety review of patients receiving Dose Level 1 or placebo.
  Intervention: Drug: VAY736

Inclusion Criteria:

• Adult patients 20 to 70 years of age
• Confirmed diagnosis of pemphigus vulgaris
• Presence of mild to moderate pemphigus vulgaris
• Patients must weight between 40 kg and 150 kg inclusive
• on a stable dose of oral corticosteriod therapy (with or without azathioprine or mycophenolate)

Exclusion Criteria:

• Pregnant or nursing (lactating) women
• Women of child-bearing potential unless they are using a highly effective method of birth control during dosing and for 4 months following study treatment
• Recent previous treatment with photo therapy, biological therapy, steroids, immunosuppresive agents (unless washout period applied)
• Active or recent history of clinically significant infection
• use of rituximab within 1 year Other protocol-defined inclusion/exclusion criteria may apply.

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com