Ascendis Pharma A/s · NASDAQ:ASND

Ascendis Pharma A/s — Technical Presentation (April 2020)

26 slides from Ascendis Pharma A/s's technical presentation, Apr 20, 2020

  1. Slide 1: TransCon PTH Top-Line Phase 2 Data from PaTH Forward

    TransCon™ PTH Top-Line Phase 2 Data from PaTH Forward April 19, 2020 Exhibit 99.1

  2. 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

  3. Slide 3: PaTH Forward Top-Line Data from 4-Week Fixed Dose Period

    PaTH Forward Top-Line Data from 4-Week Fixed Dose Period PaTH Forward top-line data support TransCon PTH as a potential replacement therapy for adult HP TransCon PTH eliminated standard of care (i.e. off active vitamin D and ≤ 500 mg per day of calcium supplements) in 100% of subjects in the 21 µg/d

  4. Slide 4: TransCon PTH Phase 2 Trial Design

    TransCon PTH Phase 2 Trial Design * PRO = patient-reported outcome ~40 adult subjects with HP currently receiving standard of care (active vitamin D + calcium) Primary Composite Endpoint (4 weeks) Proportion of subjects with: Normal serum calcium; and Normal FECa (or at least 50% decrease from basel

  5. Slide 5: PaTH Forward Trial Profile

    * Two subjects were excluded because they received < 0.25 µg BID of calcitriol (active vitamin D) PaTH Forward Trial Profile 59 Randomized Subjects 14 Assigned to PTH 15 µg/day 14 Completed Treatment 14 Included in FAS 15 Assigned to PTH 18 µg/day 15 Completed Treatment 15 Included in FAS 15 Assigne

  6. Slide 6: Demographics and Baseline Characteristics – PP

    Demographics and Baseline Characteristics – PP PTH 15 µg/day (N=14) PTH 18 µg/day (N=15) PTH 21 µg/day (N=15) Total PTH Subjects (N=44) Placebo (N=13) Age (years) (n) 14 15 15 44 13 Mean (SD) 47 (13) 47 (11) 54 (11) 49 (12) 50 (13) Age Group (years) – n (%) < 30 1 (7.1) 1 (6.7) 0 2 (4.5) 1 (7.7) ≥ 3

  7. Slide 7: Demographics and Baseline Characteristics – PP (Race, Region)

    Demographics and Baseline Characteristics – PP PTH 15 µg/day (N=14) PTH 18 µg/day (N=15) PTH 21 µg/day (N=15) Total PTH Subjects (N=44) Placebo (N=13) Race – n (%) American Indian or Alaska Native 0 0 0 0 0 Asian 0 0 2 (13) 2 (4.5) 0 Black or African American 0 0 0 0 0 Native Hawaiian or Other Pacif

  8. Slide 8: HP Disease Characteristics and History – PP

    HP Disease Characteristics and History – PP PTH 15 µg/day (N=14) PTH 18 µg/day (N=15) PTH 21 µg/day (N=15) Total PTH Subjects (N=44) Placebo (N=13) Cause of Hypoparathyroidism (HP) Acquired from neck surgery 10 (71) 12 (80) 12 (80) 34 (77) 11 (85) Autoimmune disease 1 (7.1) 0 0 1 (2.3) 0 Idiopathic

  9. Slide 9: Baseline HP Supplements – PP

    Baseline HP Supplements – PP 2 subjects did not have eDiary information confirmed by prescription information HP Supplements at Baseline collected by eDiary/ Total Daily Dose (TDD) PTH 15 µg/day (N=14) PTH 18 µg/day (N=15) PTH 21 µg/day (N=15) Total PTH Subjects (N=44) Placebo (N=13) Calcium /TDD (m

  10. Slide 10: Baseline of Spot FECa & Albumin-Adjusted sCa – PP

    Baseline of Spot FECa & Albumin-Adjusted sCa – PP Lab Summary at Baseline PTH 15 µg/day (N=14) PTH 18 µg/day (N=15) PTH 21 µg/day (N=15) Total PTH Subjects (N=44) Placebo (N=13) Albumin-Adjusted sCa (mg/dL) (n) 14 15 15 44 13 Mean (SD) 8.6 (0.49) 9.1 (1.3) 8.7 (0.62) 8.8 (0.91) 8.9 (0.39) Spot AM FE

  11. Slide 11: Treatment-Emergent Adverse Event Summary – SAS

    Treatment-Emergent Adverse Event Summary – SAS * Subjects are counted only in the highest severity category PTH 15 µg/day (N=14) n (%) PTH 18 µg/day (N=15) n (%) PTH 21 µg/day (N=15) n (%) Total PTH (N=44) n (%) Placebo (N=15) n (%) TEAEs 6 (43) 3 (20) 8 (53) 17 (39) 5 (33) Serious TEAE 0 0 0 0 0 Se

  12. Slide 12: Treatment-Emergent Adverse Events of Interest – SAS

    Treatment-Emergent Adverse Events of Interest – SAS Includes ≥ 2 subjects in the PTH group or hyper- or hypocalcaemia Preferred Term PTH 15 µg/day (N=14) n (%) PTH 18 µg/day (N=15) n (%) PTH 21 µg/day (N=15) n (%) Total PTH (N=44) n (%) Placebo (N=15) n (%) TEAEs 6 (43) 3 (20) 8 (53) 17 (39) 5 (33)

  13. Slide 13: PaTH Forward 4-Week Fixed Dose Safety Summary

    PaTH Forward 4-Week Fixed Dose Safety Summary All doses of TransCon PTH were well-tolerated No drop-outs during 4-week blinded period No serious or severe TEAEs were reported No TEAEs leading to discontinuation of study drug Overall incidence of TEAEs comparable between TransCon PTH and placebo TEAE

  14. Slide 14: Elimination of Standard of Care – PP

    Elimination of Standard of Care – PP * Not taking active vitamin D and ≤500 mg/day of calcium supplements Number of Subjects Meeting Each Component PTH 15 µg/day (N=14) PTH 18 µg/day (N=15) PTH 21 µg/day (N=15) Total PTH Subjects (N=44) Placebo (N=13) Not taking active vitamin D supplements 14 (100%

  15. Slide 15: Mean Active Vitamin D Dose by Visit – PP

    Mean Active Vitamin D Dose by Visit – PP TransCon PTH enabled discontinuation of active vitamin D at week 2 Mean Active Vitamin D Dose ± SE (µg/day)

  16. Slide 16: Mean Calcium Supplement Dose by Visit – PP

    Mean Calcium Supplement Dose by Visit – PP TransCon PTH enabled continuous calcium supplement reduction over 4-week study period Mean Calcium Dose ± SE (mg/day)

  17. Slide 17: Mean Serum Calcium and Spot FECa by Visit – PP

    Mean Serum Calcium and Spot FECa by Visit – PP TransCon PTH subjects exhibited reduced FECa, despite increased serum calcium For placebo subjects, FECa followed serum calcium levels Mean Calcium Corrected for Albumin ± SE (mg/dL) Mean Fractional Excretion of Calcium ± SE (%) Mean Serum Calcium Mean

  18. Slide 18: TransCon PTH Increased Number of FECa Responders – PP

    TransCon PTH Increased Number of FECa Responders – PP By week 4 of treatment, TransCon PTH had normalized an additional 8 subjects compared to none on placebo % of Subjects with Normal AM FECa Baseline Week 4 Baseline Week 4 Baseline Week 4 Baseline Week 4

  19. Slide 19: Mean Serum Phosphate by Visit – PP

    Mean Serum Phosphate by Visit – PP TransCon PTH subjects demonstrated consistent, sustained reductions in serum phosphate Mean Serum Phosphate ± SE (mg/dL)

  20. Slide 20: Mean Calcium-Phosphate Product by Visit – PP

    Mean Calcium-Phosphate Product by Visit – PP TransCon PTH demonstrated consistent, sustained reductions in calcium-phosphate product Mean Albumin-Adjusted Calcium x Phosphate ± SE (mg2/dL2)

  21. Slide 21: Primary Composite Endpoint at Week 4 – PP

    Primary Composite Endpoint at Week 4 – PP PTH 15 µg/day (N=14) PTH 18 µg/day (N=15) PTH 21 µg/day (N=15) Total PTH Subjects (N=44) Placebo (N=13) Number of Subjects Meeting Primary Composite Endpoint at Week 4 with Fixed Dosing 7 6 9 22 2 Proportion (95% CI) 50 (23, 77) 40 (16, 68) 60 (32, 84) 50 (3

  22. Slide 22: Key Secondary Composite Endpoint at Week 4 – PP

    Key Secondary Composite Endpoint at Week 4 – PP PTH 15 µg/day (N=14) PTH 18 µg/day (N=15) PTH 21 µg/day (N=15) Total PTH Subjects (N=44) Placebo (N=13) Number of Subjects Meeting Key Secondary Composite Endpoint at Week 4 with Fixed Dosing 7 4 9 20 2 Proportion (95% CI) 50 (23, 77) 27 (7.8, 55) 60 (

  23. Slide 23: Key Takeaways from the PaTH Forward Trial – Primary Endpoint

    Key Takeaways from the PaTH Forward Trial – Primary Endpoint PaTH Forward Trial met the primary endpoint Overall statistical significance achieved notwithstanding: Short study duration of 4 weeks Fixed dose not individualized for each subject’s optimal dose Subjects continued to titrate off calcium

  24. Slide 24: Open-label Extension Trial

    Open-label Extension Trial Subjects from fixed-dose PaTH Forward Trial rolled over to the open-label extension which enabled individually optimized TransCon PTH dosing to evaluate long-term safety and efficacy 58 out of 59 randomized subjects currently receiving TransCon PTH in the open-label extens

  25. Slide 25: Planned Next Steps

    Planned Next Steps Engage with global regulatory authorities on next steps for development of TransCon PTH Report PaTH Forward open-label extension six-month data in Q3 2020 Submit proposed PRO instrument for FDA review in Q3 2020 Submit regulatory filings to initiate a global phase 3 trial in North

  26. Slide 26: PaTH Forward Top-Line Data from 4-Week Fixed Dose Period

    PaTH Forward Top-Line Data from 4-Week Fixed Dose Period PaTH Forward top-line data support TransCon PTH as a potential replacement therapy for adult HP TransCon PTH eliminated standard of care (i.e. off active vitamin D and ≤ 500 mg per day of calcium supplements) in 100% of subjects in the 21 µg/d

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