PaTHway Phase 3 Trial Analysis

TransCon PTH PATHway (Phase 3) Trial Double-blind, placebo-controlled trial with an open-label extension period adults with chronic hypoparathyroidism randomized 3:1 (TransCon PTH: placebo) Double-Blind Main period (26 weeks) Week 26 PaTHway TRIAL Open-Label Extension period (156 weeks) TransCon PTH 18 mcg/day TransCon PTH (titrated according to algorithm) Placebo TransCon PTH TransCon PTH 6 Placebo Primary Objective Confirm treatment effect of TransCon PTH in adults with hypoparathyroidism Key Eligibility Criteria Adults with chronic hypoparathyroidism (i.e. for at least 26 weeks) Age ≥18 years Reliant on calcitriol ≥0.50 mcg per day or alfacalcidol ≥1.0 mcg per day, and therapeutic elemental calcium ≥800 mg/day for at least 12 weeks prior to screening Serum calcium in normal (or just below normal) range: 7.8-10.6 mg/dL (1.96-2.64 mmol/L) No PTH or PTHrP therapy within 4 weeks prior to Screening Countries Europe (Germany, Denmark, Norway, Italy, Hungary) North America (United States, Canada) 1 No increase in prescribed study drug within 4 weeks prior to Week 26 visit. 2 If needed to meet recommended dietary intake of calcium, it was permitted to take calcium supplements ≤600 mg/day as a nutritional supplement. Primary Composite Endpoint at Week 261 Proportion of patients with: • Serum calcium in the normal range (8.3–10.6 mg/dL) and Independence from active vitamin D and Independence from calcium supplements² Key Secondary Endpoints at Week 26 • HPES Symptom - Physical domain score HPES Symptom - Cognitive domain score • HPES Impact - Physical Functioning domain score HPES Impact - Daily Life domain score SF-36 - Physical Functioning subscale score TransCon PTH is an investigational product candidate. For investor communication only. Not for use in product promotion. Not for further distribution. ascendis pharma

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