Slide 1: Long-Term Efficacy and Safety of TransCon PTH in Adults with Hypoparathyroidism
Long-Term Efficacy and Safety of TransCon™ PTH in Adults with Hypoparathyroidism: 52-Week Results From the Open-Label Extension of the Phase 3 PaTHway Trial Presented at ENDO 2023 June 17, 2023 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 prospective product candidates; clinical trial results; the expected timing o
Slide 3: PTH Therapy for Hypoparathyroidism
PTH Therapy for Hypoparathyroidism An intact PTH axis maintains normal serum calcium and phosphate homeostasis1,2 PTH acts on bone, kidney, and indirectly, intestine1,3 Promotes normal nerve and muscle function4 Conventional therapy for hypoparathyroidism (active vitamin D [e.g., calcitriol, alfacal
Slide 4: TransCon PTH (palopegteriparatide) Design
TransCon PTH (palopegteriparatide) Design Renal clearance Receptor TransCon carrier TransCon linker PTH (inactive) Active PTH Linker cleavage under physiologic conditions TransCon PTH is an investigational prodrug, administered once daily, with sustained release of active PTH designed to provide PTH
Slide 5: TransCon PTH Phase 3 PaTHway Trial Design
TransCon PTH Phase 3 PaTHway Trial Design (NCT04701203) aIndependence from therapeutic doses of calcium is defined as a standing dose of elemental calcium ≤600 mg on the day prior to the week 52 visit bIndependence from active vitamin D is defined as a standing dose of active vitamin D equal to zero
Slide 6: Participant Disposition
Participant Disposition ITT, intention-to-treat; PTH, parathyroid hormone Data on file, Ascendis Pharma Discontinued due to: Withdrew consent (n=1) Breast cancer (n=1) Placebo n=19 Randomized N=84 TransCon PTH n=63 Placebo n=21 Not treated due to: Withdrew consent (n=1) Thyroid cancer recurrence (n=
Slide 7: Participants Who Met the Multi-Component Endpoint Criteria at Week 52
Participants Who Met the Multi-Component Endpoint Criteria at Week 52 81% of participants treated with TransCon PTH met the multi-component efficacy endpoint and 95% achieved independencec from conventional therapy at Week 52 of the PaTHway trial aPercentages are calculated based on participants who
Slide 8: Albumin-Adjusted Serum Calcium Levels at Week 52
Albumin-Adjusted Serum Calcium Levels at Week 52 Albumin-Adjusted Serum Calcium (mmol/L) Mean (± SE) aAll participants received TransCon PTH during the open-label period bNormal range 8.3-10.6 mg/dL SE, standard error Data on file, Ascendis Pharma 2 4 6 8 10 12 16 20 38 46 | | | | | | | | | | Mean a
Slide 9: Independence from Conventional Therapy at Week 52
| | | | | | | | | | | | | | | Double-blind period Open-label perioda Weeks Independence from Conventional Therapy at Week 52 Active Vitamin D Elemental Calcium Active Vitamin D Dose (µg/day) Mean (± SE) Calcium Dose (mg/day) Mean (± SE) Weeks TransCon PTH enabled rapid and sustained independenceb fr
Slide 10: HPES-Symptom Scores
HPES-Symptom Scores Through Week 52 HPES-Symptom Physical Domain Score HPES-Symptom Cognitive Domain Score HPES-Symptom scores showed a sustained improvement in hypoparathyroidism-related physical and cognitive symptoms with TransCon PTH treatment over 52 weeks Double-blind period Open-label perioda
Slide 11: HPES-Impact Domain Scores
HPES-Impact Domain Scores Through Week 52 HPES-Impact Physical Functioning Domain Score HPES-Impact Daily Life Domain Score HPES-Impact scores showed sustained improvement in the impact of hypoparathyroidism on physical functioning and daily life with TransCon PTH In participants first treated with
Slide 12: SF-36 Physical Functioning Subscale Scores
SF-36 Physical Functioning Subscale Scores Through Week 52 aThe dashed lines (--) indicate the upper (53) and lower (47) bounds of T scores considered to be in the range of average functioning for the U.S. general population of group level data. Group mean scores lower than 47 indicate impairment. S
Slide 13: Bone Turnover Markers
Double-blind period Open-label perioda Bone Turnover Markers Through Week 52 Procollagen Type 1 N-Terminal Propeptide (P1NP) C-Terminal Telopeptide of Type 1 Collagen (CTx) In the TransCon PTH/TransCon PTH group, smaller incremental changes were seen in bone turnover markers between weeks 26 and 52
Slide 14: Bone Mineral Density by DXA: Z-Scores
Bone Mineral Density by DXA in Participants Treated with TransCon PTH from Baseline Through Week 52 an=59 (Baseline), n=58 (Week 26), n=57 (Week 52) bn=59 (Baseline) Data from participants randomized to TransCon PTH at baseline only (TransCon PTH/TransCon PTH group) BMD, bone mineral density; DXA, d
Slide 15: Bone Mineral Density by DXA: T-Scores
Bone Mineral Density by DXA in Participants Treated with TransCon PTH from Baseline Through Week 52 an=59 (Baseline), n=58 (Week 26), n=57 (Week 52) bn=60 (Week 26), n=59 (Week 52) cT-score reference point: young (30-year-old) Caucasian adult Data from participants randomized to TransCon PTH at base
Slide 16: 24-Hour Urine Calcium Excretion
24-Hour Urine Calcium Excretion Through Week 52 TransCon PTH/TransCon PTH 24-Hour Urine Calcium (mg/day) Mean (± SE) Placebo/ TransCon PTH ULN (≤250 mg/day) TransCon PTH normalized mean 24-hour urine calcium excretion within 26 weeks, which was maintained through week 52 Mean 24-hour urine calcium
Slide 17: Summary of TEAEs in the PaTHway Trial
Summary of TEAEs in the PaTHway Trial Through Week 52 Treatment Emergent Adverse Events (TEAEs), n (%) Total TransCon PTHa (N=80) Any TEAE 72 (90.0) Serious TEAE 8 (10.0) Severityb Grade 1 37 (46.3) Grade 2 27 (33.8) Grade 3 7 (8.8) Grade 4 1 (1.3) Related TEAE 42 (52.5) Serious related TEAEc 2 (2.5
Slide 18: Conclusions
Conclusions At Week 52, 81% of participants treated with TransCon PTH achieved normal serum calcium and independencea from conventional therapy. 95% of participants achieved independencea from conventional therapy TransCon PTH resulted in improvements in symptoms and health-related quality of life w
Slide 19: Thank you
Thank you Company contact: Tim Lee Senior Director, Investor Relations tle@ascendispharma.com (650) 374-6343