Slide 1: TransCon PTH Top-Line Data from Phase 3 PaTHway Trial
TransCon™ PTH Top-Line Data from Phase 3 PaTHway Trial March 13, 2022 Exhibit 99.1
Slide 2: Cautionary Note on Forward-Looking Statements
Cautionary Note on Forward-Looking Statements This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this presentation, such as statements regarding our future results of operations and financial position, including our business s
Slide 3: TransCon PTH PaTHway Trial Top-Line Data at Week 26
TransCon PTH PaTHway Trial Top-Line Data at Week 26 PaTHway Trial met primary and all key secondary endpoints 78.7% of patients (48 of 61) treated with TransCon PTH achieved the primary endpoint, compared to 4.8% (1 of 21) of patients in the control group (p-value <0.0001) Statistically significant
Slide 4: Chronic Hypoparathyroidism: Significant Patient Population
Chronic Hypoparathyroidism: Significant Patient Population Estimated Prevalence: ~200K in these 3 regions ~25k–32k ~25k–32k Japan 2017. Shishiba et. al., Prevalence of postsurgical hypoparathyroidism in Japan: Estimated from the data of multiple institutes 1999. Nakamura et. al., Prevalence of Idiop
Slide 5: Hypoparathyroidism: Multiple Complications
Hypoparathyroidism: Multiple Complications Shobak DM et. al. J Clin Endocrinol Metab. 2016 June 01;101(6):2300-2312. Brain fog Fatigue Pain Numbness & tingling Infections Anxiety & depression Abnormal skeletal dynamics Renal failure Abnormal tissue calcifications Arrhythmias Muscle spasms Heart fail
Slide 6: TransCon PTH PaTHway (Phase 3) Trial
TransCon PTH PaTHway (Phase 3) Trial 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. Double-blind, placebo-controlled trial w
Slide 7: PaTHway Trial Patient Disposition
Data on file, Ascendis Pharma 2022. PaTHway Trial Patient Disposition Intention To Treat (ITT): All randomized patients who received at least 1 dose of randomized treatment Safety Analysis Set (SAS): All randomized patients who received at least 1 dose of randomized treatment Randomized N = 84 Trans
Slide 8: Patients Who Discontinued Trial during Blinded Treatment Period
Patients Who Discontinued Trial during Blinded Treatment Period Randomized Arm Off Study Day Off Study Reason Placebo 30 Withdrew consent Placebo 62 Breast cancer TransCon PTH 111 Cardiac arrest Data on file, Ascendis Pharma 2022. All discontinuations were unrelated to study drug
Slide 9: Patient Demographics
ascendis LA pharma Patient Demographics
Slide 10: Demographics and Baseline Characteristics
Demographics and Baseline Characteristics Data on file, Ascendis Pharma 2022. Characteristics TransCon PTH (N = 61) Placebo (N = 21) Age (years) (n) 61 21 Mean (SD) 49.0 (13.1) 47.3 (11.4) Age Group (years) – n (%) <50 28 (45.9) 14 (66.7) ≥50 33 (54.1) 7 (33.3) Sex at Birth n (%) Female 46 (75.4) 18
Slide 11: Demographics and Baseline Characteristics (continued)
Demographics and Baseline Characteristics (continued) Data on file, Ascendis Pharma 2022. Characteristics TransCon PTH (N = 61) Placebo (N = 21) Race – n (%) American Indian or Alaska Native 0 0 Asian 3 (4.9) 2 (9.5) Black or African American 0 0 Native Hawaiian or Other Pacific Islander 0 0 White 5
Slide 12: Hypoparathyroidism Disease Etiology and Medical History
Hypoparathyroidism Disease Etiology and Medical History Data on file, Ascendis Pharma 2022. Characteristics TransCon PTH (N = 61) Placebo (N = 21) Cause of Hypoparathyroidism (HP) Acquired from neck surgery 52 (85.2) 18 (85.7) Autoimmune disease 1 (1.6) 0 Intrinsic genetic defects of the parathyroid
Slide 13: Baseline Conventional Therapy
Baseline Conventional Therapy Conventional Therapy Total Daily Dose (TDD) at Baseline TransCon PTH (N = 61) Placebo (N = 21) Calcium Supplement/TDD (mg) (n) 61 21 Mean 1748 2105 Min, Max 600, 5000 800, 7200 Calcitriol (Active Vitamin D) /TDD (µg) (n) 53 17 Mean 0.76 0.69 Min, Max 0.5, 2.0 0.5, 1.75
Slide 14: Baseline Albumin-Adjusted Serum Calcium & 24-Hour Urine Calcium
Baseline Albumin-Adjusted Serum Calcium & 24-Hour Urine Calcium sCA, serum calcium. Data on file, Ascendis Pharma 2022. Lab Summary at Baseline TransCon PTH (N = 61) Placebo (N = 21) Albumin-Adjusted sCa (mg/dL) (n) 61 21 Mean (SD) 8.8 (0.7) 8.6 (0.6) 24-Hour Urine Calcium (mg/dL) (n) 60 21 Mean (SD
Slide 15: Trial Results
ascendis LA pharma Trial Results
Slide 16: Primary Composite Endpoint at Week 26
Primary Composite Endpoint at Week 26 1 CMH test controlling for etiology of hypoparathyroidism (post-surgical vs other). 2 The normal range for albumin-adjusted sCa is 8.3-10.6 mg/dL (2.07-2.64 mmol/L). Patients with missing data on one or more of the criteria are considered as non-responders. Data
Slide 17: Active Vitamin D Dose (Mean +/- SE) by Visit
Active Vitamin D Dose (Mean +/- SE) by Visit Data on file, Ascendis Pharma 2022. TransCon PTH patients discontinued active vitamin D completely within four weeks Active Vitamin D Dose µg/day Mean (± SE) Weeks
Slide 18: Calcium Supplement Dose (Mean +/- SE) by Visit
Calcium Supplement Dose (Mean +/- SE) by Visit TransCon PTH enabled rapid and sustained calcium supplement reduction Calcium Dose mg/day Mean (± SE) Weeks
Slide 19: Albumin-adjusted Serum Calcium (Mean +/- SE) by Visit
Albumin-adjusted Serum Calcium (Mean +/- SE) by Visit TransCon PTH patients maintained mean serum calcium levels in the normal range at all study visits Calcium Corrected for Albumin mg/dL Mean (± SE) Weeks
Slide 20: Key Secondary Endpoints: Patient Reported Symptom & Quality of Life Domains
Key Secondary Endpoints: Patient Reported Symptom & Quality of Life Domains P-values are TransCon PTH vs Control. For HPES, lower scores indicate improvement; for SF-36, higher scores indicate improvement. Data on file, Ascendis Pharma 2022. HPES Symptom Physical domain score All prespecified key se
Slide 21: Bone Turnover Markers: P1NP and CTx (Mean +/- SE) by Visit
Bone Turnover Markers: P1NP and CTx (Mean +/- SE) by Visit P1NP, procollagen type 1 N-terminal propeptide CTx, C-terminal telopeptides of type I collagen Data on file, Ascendis Pharma 2022. Procollagen 1N-Terminal Propeptide ng/mL Mean (± SE) Similar pattern exhibited at Week 26 in Phase 2 PaTH Forw
Slide 22: Safety Results
ascendis LA pharma Safety Results
Slide 23: Overall TEAE Summary
Overall TEAE Summary *In the severity categories, patients are displayed for the highest severity category only. **Death due to cardiac arrest Data on file, Ascendis Pharma 2022. TEAE Summary TransCon PTH (N = 61); n (%) Placebo (N = 21); n (%) Treatment-Emergent Adverse Events (TEAE) 50 (82.0) 21 (
Slide 24: Treatment-Emergent Adverse Events (≥5 patients in total)
Treatment-Emergent Adverse Events (≥5 patients in total) Data on file, Ascendis Pharma 2022. Preferred Term TransCon PTH (N = 61) Placebo (N = 21) Patients with at least one TEAE, n (%) 50 (82.0) 21 (100.0) TEAEs Injection site reaction 19 (31.1) 0 Headache 13 (21.3) 2 (9.5) Hypocalcaemia 6 (9.8) 9
Slide 25: 24-Hour Urine Calcium (mg/d) by Visit
24-Hour Urine Calcium (mg/d) by Visit 1 The ANCOVA model with unequal variance includes the change from baseline as the response variable, treatment and etiology of HP as fixed effects and baseline value of the parameter as a covariate. 2 p-values from t-test. Data on file, Ascendis Pharma 2022. 24-
Slide 26: TransCon PTH PaTHway Trial Top-line Data at Week 26
TransCon PTH PaTHway Trial Top-line Data at Week 26 PaTHway Trial met primary and all key secondary endpoints 78.7% of patients (48 of 61) treated with TransCon PTH achieved the primary endpoint, compared to 4.8% (1 of 21) of patients in the control group (p-value <0.0001) Statistically significant
Slide 27: Program Status and Next Steps
Program Status and Next Steps Two Open-Label Extension trials continuing 57 of 59 patients remain in PaTH Forward Trial after two years All 79 patients who completed the blinded period continue in the PaTHway Trial Engage with regulatory authorities regarding registration plans for US and EU Anticip
Slide 28: Thank you
Thank you Contact Tim Lee ir@ascendispharma.com