Ascendis Pharma A/s · NASDAQ:ASND
Ascendis Pharma A/s — Corporate Update (September 2020)
30 slides from Ascendis Pharma A/s's corporate update, Sep 29, 2020
Slide 1: TransCon PTH Clinical Development Update
TransConTM PTH Clinical Development Update September 29, 2020 All product candidates are investigational. For investor communication only. Not for use in promotion or product commercialisation. 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 Overview
TransCon PTH Overview Preliminary PaTH Forward OLE data at month 6 support TransCon PTH as a potential hormone replacement therapy for adult HP Eliminated standard of care1 with a well-tolerated once daily injection Normalized mean SF-36 quality-of-life scores on all summary and subdomains Normalize
Slide 4: Preliminary PaTH Forward Open-Label Extension Data at Month 6
Preliminary PaTH Forward Open-Label Extension (OLE) Data at Month 6 TransCon PTH eliminated standard of care1 in 91% of subjects TransCon PTH demonstrated significant reduction in urinary calcium 86% of subjects had 24-hour urine calcium in the normal range or 50% reduction from baseline Mean 24-hou
Slide 5: TransCon PTH Phase 2 Trial Design
TransCon PTH Phase 2 Trial Design PRO = Patient-reported Outcome. HPES = Hypoparathyroidism Patient Experience Scale. BMD = Bone Mineral Density. TBS =Trabecular Bone Score. DXA = Dual-Energy X-Ray Absorptiometry. FECa = Fractional Excretion of Calcium. 59 adult subjects with HP currently receiving
Slide 6: Demographics and Baseline Characteristics
PaTH Forward Demographics and Baseline Characteristics Preliminary PaTH Forward OLE 6-month data. Total (N=59) Age (years) (n) 59 Mean (SD) 50 (12) Age Group (years) – n (%) <30 3 (5) ≥30 – <65 51 (86) ≥65 5 (9) Sex at Birth n (%) Female 48 (81) Body Mass Index (kg/m2) (n) 59 Mean (SD) 28 (4) Menopa
Slide 7: Demographics and Baseline Characteristics
PaTH Forward Demographics and Baseline Characteristics Preliminary PaTH Forward OLE 6-month data. Total (N=59) Race – n (%) American Indian or Alaska Native 0 Asian 2 (3) Black or African American 0 Native Hawaiian or Other Pacific Islander 0 White 54 (92) Unknown 0 Other 3 (5) Geographic Region – n
Slide 8: HP Disease Characteristics and History
PaTH Forward HP Disease Characteristics and History Total (N=59) Cause of Hypoparathyroidism (HP) Acquired from neck surgery 47 Autoimmune disease 1 Idiopathic disease 11 Duration of HP (Years) Mean 12 Min, Max 1, 39 Renal Insufficiency History 5 Kidney Stones History 8 Ectopic Calcifications Histor
Slide 9: Baseline HP Supplements
PaTH Forward Baseline HP Supplements Preliminary PaTH Forward OLE 6-month data. 2 subjects did not have eDiary information confirmed by prescription information. HP Supplements at Baseline collected by eDiary/Total Daily Dose (TDD) Total (N=59) Calcium /TDD (mg) (n) 59 Mean 2079 Min, Max 500, 8000 C
Slide 10: Baseline of Spot FECa & Albumin-Adjusted sCa
PaTH Forward Baseline of Spot FECa & Albumin-Adjusted sCa Preliminary PaTH Forward OLE 6-month data. Lab Summary at Baseline Total (N=59) Albumin-Adjusted sCa (mg/dL) (n) 59 Mean (SD) 8.8 (0.80) Spot AM FECa (%) (n) 59 Mean (SD) 2.6 (1.3) Spot AM FECa normal (≤2%) baseline, n (%) 25 (42)
Slide 11: OLE Overall TEAE Summary
PaTH Forward OLE Overall TEAE Summary Preliminary PaTH Forward OLE 6-month data. Percentages are calculated based on the number of subjects in the Safety Population. In the severity categories, subjects are displayed for the highest severity only. An AE is considered a TEAE if it occurred after the
Slide 12: Preliminary PaTH Forward OLE Safety Summary
Preliminary PaTH Forward OLE Safety Summary TransCon PTH was well-tolerated 58 subjects continue in open label extension beyond 6 months No drug-related serious TEAEs were reported No TEAEs leading to discontinuation of study drug TEAEs with TransCon PTH reflect known PTH pharmacology Injections wer
Slide 13: OLE Distribution of Doses at Month 6
PaTH Forward OLE Distribution of Doses at Month 6 Preliminary PaTH Forward OLE 6-month data. Dose level (µg/day) Number of patients At month 6 in PaTH Forward At month 6 of PaTH Forward, subjects used all available doses of TransCon PTH, with a mean of 18 µg/day
Slide 14: OLE Overall Compliance with Dosing
PaTH Forward OLE Overall Compliance with Dosing Preliminary PaTH Forward OLE 6-month data. Study drug compliance is calculated as: (Total number of actual TransCon PTH doses/total number of planned doses)* 100. TransCon PTH (n=44) Placebo/TransCon (n=15) All TransCon PTH (n=59) Compliance (%) Mean 9
Slide 15: PaTH Forward OLE Elimination of Standard of Care at Month 6
Preliminary PaTH Forward OLE 6-month data *Not taking active vitamin D and ≤500 mg/day of calcium supplements Number of Subjects Meeting Each Component TransCon PTH/TransCon PTH (N=44) Placebo/TransCon PTH (n=15) All TransCon PTH (N=59) The number of subjects reached 6 month visit 44 14 58 Active vi
Slide 16: PaTH Forward OLE Mean Active Vitamin D Dose
OLE starts PaTH Forward OLE Mean Active Vitamin D Dose TransCon PTH enabled discontinuation of active vitamin D within two weeks of treatment initiation Preliminary PaTH Forward OLE 6-month data Mean Active Vitamin D Dose µg/day (± SE) Weeks
Slide 17: PaTH Forward OLE Mean Calcium Supplement Dose
OLE starts PaTH Forward OLE Mean Calcium Supplement Dose TransCon PTH enabled rapid and continuous calcium supplement reduction over 6-month study period Preliminary PaTH Forward OLE 6-month data. Mean Calcium Dose mg/day (± SE) Weeks
Slide 18: PaTH Forward OLE Mean Serum Calcium and Mean 24-Hour Urine Calcium
Mean Calcium Corrected for Albumin mg/dL (± SE) PaTH Forward OLE Mean Serum Calcium and Mean 24-Hour Urine Calcium Preliminary PaTH Forward OLE 6-month data. Mean 24-hour Urine Calcium mg/24hr Normal (men) Normal (women) Mean 24-hour urine calcium normalized while maintaining normal mean serum calci
Slide 19: OLE Mean 24-Hour Urine Calcium at Month 6
PaTH Forward OLE Mean 24-Hour Urine Calcium at Month 6 Preliminary PaTH Forward OLE 6-month data. Parameter (unit) Visit Statistics TransCon PTH/ TransCon PTH (N=44) Placebo/ TransCon PTH (N=15) All TransCon PTH (N=59) 24-Hour Urine Calcium (mg/24 hr) Baseline – n 39 11 50 Mean (SD) 425 (200) 440 (1
Slide 20: OLE Mean Spot AM FECa at Month 6
PaTH Forward OLE Mean Spot AM FECa at Month 6 Preliminary PaTH Forward OLE 6-month data. At each post-baseline visit, only data from subjects with both baseline and the corresponding visit values available are used to compute the statistical summaries. Parameter (unit) Visit Statistics TransCon PTH/
Slide 21: OLE Subjects without Complete Composite Endpoint at Month 6
PaTH Forward OLE 10 Subjects without Complete Composite Endpoint at Month 6 One subject (randomized to placebo) withdrew for reasons unrelated to safety or efficacy of the study drug Four subjects excluded due to 24-hour urine sampling out of window (day 169–210): All four subjects were off active v
Slide 22: Proportion of Subjects Achieving Composite Endpoint at Month 6
Proportion of Subjects Achieving Composite Endpoint at Month 6 in PaTH Forward OLE Preliminary PaTH Forward OLE 6-month data. 1Subjects in this group switched from placebo at 4 weeks to TransCon PTH for the open-label portion of the trial. 2Percentage is based on the number of subjects who have data
Slide 23: OLE Partial Responder Analysis at Month 6
PaTH Forward OLE Partial Responder Analysis at Month 6 Preliminary PaTH Forward OLE 6-month data. Green = meet response criteria. Yellow = did not meet response criteria. 1 Response criteria are normal serum calcium, off vitamin D, taking ≤500 mg calcium, and 24-hour urine calcium in the normal rang
Slide 24: OLE Mean Serum Phosphate
PaTH Forward OLE Mean Serum Phosphate TransCon PTH subjects demonstrated consistent, sustained reductions in serum phosphate Preliminary PaTH Forward OLE 6-month data. Mean Serum Phosphate mg/dL (± SE) Weeks
Slide 25: OLE Mean Serum Calcium-Serum Phosphate Product
PaTH Forward OLE Mean Serum Calcium-Serum Phosphate Product TransCon PTH demonstrated consistent, sustained reductions in calcium-phosphate product Preliminary PaTH Forward OLE 6-month data. Mean Albumin-Adjusted Calcium x Phosphate mg2/dL2 (± SE) Weeks
Slide 26: OLE Change in SF-36 Health Survey Domain Mean Scores
PaTH Forward OLE Change in SF-36® Health Survey Domain Mean Scores (SD) Preliminary PaTH Forward OLE 6-month data. Green: patients within normal range. (SD). *PF (physical functioning), RP (role physical), BP (bodily pain), SF (social functioning), MH (mental health), RE (role emotional), VT (vitali
Slide 27: PaTH Forward OLE Mean P1NP and CTx
OLE starts OLE starts PaTH Forward OLE Mean P1NP and CTx Preliminary PaTH Forward OLE 6-month data. Procollagen 1N-Terminal Propeptide ng/mL (± SE) PaTH Forward patient population had expected low level of bone turnover at baseline and demonstrated at 6 months increased levels of anabolic and catabo
Slide 28: PaTH Forward OLE % Change in Mean P1NP and CTx
OLE starts OLE starts PaTH Forward OLE % Change in Mean P1NP and CTx Preliminary PaTH Forward OLE 6-month data. At 6-month point, observed lower level of increase for anabolic compared to catabolic bone turnover % Change Procollagen 1N-Terminal Propeptide ng/mL (± SE) % Change in Mean P1NP % Change
Slide 29: PaTHway Phase 3 Trial Design
PaTHway Phase 3 Trial Design 1 Enrollment increased to 76 subjects to ensure evaluable data for 68. 2 If needed to meet recommended dietary intake of calcium, it is permitted to take calcium supplements ≤600 mg/day as a nutritional supplement. Double-blind, placebo-controlled trial with an open-labe
Slide 30: Summary of TransCon PTH Update
Summary of TransCon PTH Update Preliminary phase 2 PaTH Forward OLE results at month 6*: 86% of subjects had (1) normal serum calcium, (2) off active vitamin D and (3) taking ≤600 mg/day of calcium 71% of subjects achieved composite endpoint of (1) normal serum calcium, (2) off vitamin D, (3) taking