Our Pipeline

Our Pipeline

PIPELINE OVERVIEW

Our growing development pipeline of innovative new therapies is driven by the flexibility of our TransCon®️ technologies and our aim to improve patients’ lives. We have multiple prodrug therapies in development.
Each candidate is unique and designed to be a best-in-class therapy.

ENDOCRINOLOGY RARE DISEASES

Phase 1
Phase 2
Phase 3
TransCon hGH
Pediatric Growth Hormone Deficiency
(Greater China)1
Phase 3
Pediatric Growth Hormone Deficiency
(Japan)2
Phase 3
Adult Growth Hormone Deficiency
(Global)3
Phase 3
TransCon PTH
Adult Hypoparathyroidism
(North America & Europe)4
Phase 3
Adult Hypoparathyroidism
(Greater China)5
Phase 3
Adult Hypoparathyroidism
(Japan)6
Phase 3
TransCon CNP
Pediatric Achondroplasia
(North America, Europe, Oceania)7
Phase 2
Pediatric Achondroplasia
(Greater China)8
Phase 2

ONCOLOGY

Phase 1
Phase 2
Phase 3
TransCon TLR7/8 Agonist
Monotherapy9
Phase 1
Combination Therapy9
Phase 1
TransCon IL-2 β/γ
Monotherapy10
Phase 1
Combination Therapy10
Phase 1

1 Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Chinese Pediatric Growth Hormone Deficiency. ClinicalTrials.gov identifier: NCT04326374. Updated March 30, 2020. Accessed April 11, 2022. https://clinicaltrials.gov/ct2/show/NCT04326374
2 Efficacy, safety and shinobi of lonapegsomatropin (TransCon hGH) once weekly for 52 weeks in prepubertal Japanese pediatric growth hormone deficiency short stature (GHD) patients who have not been treated with hGH. Phase 3, multicenter, randomized, open-label, active-controlled, parallel-group comparative trials comparing tolerability with daily doses of standard hGH replacement therapy. Clinical research implementation plan number jRCT2031200340. Updated November 24, 2021. Accessed April 11, 2022. https://rctportal.niph.go.jp/detail/jr?trial_id=jRCT2031200340
3 A Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency (foresiGHt). ClinicalTrials.gov identifier: NCT04615273. Updated April 19, 2021. Accessed April 11, 2022. https://clinicaltrials.gov/ct2/show/NCT04615273
4 A Trial Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Daily in Adults With Hypoparathyroidism (PaTHway). ClinicalTrials.gov identifier: NCT04701203. Updated April 6, 2022. Accessed April 12, 2022. https://clinicaltrials.gov/ct2/show/NCT04701203
5 Ascendis Pharma A/S Announces Target Enrollment Achieved in the Phase 3 PaTHway Trial of TransCon™ PTH (palopegteriparatide) in Adults with Hypoparathyroidism (HP) and Provides a Comprehensive Global Clinical Program Update. Press Release. Acendis Pharma A/S. July 6, 2021. Accessed April 12, 2022. https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-pharma-announces-target-enrollment-achieved-phase-3
6 PaTHway Japan study: Phase 3, multicenter study to examine the safety, tolerability and efficacy of transCon PTH subcutaneously once daily in Japanese adult patients with hypoparathyroidism , Single group open-label trial. Clinical research implementation plan number jRCT2051210058. Updated March 8, 2022. Accessed April 11, 2022. https://rctportal.niph.go.jp/detail/jr?trial_id=jRCT2051210058
7 A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia. ClinicalTrials.gov identifier: NCT04085523. Updated May 13, 2021. Accessed April 11, 2022. https://clinicaltrials.gov/ct2/show/NCT04085523
8 A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia. ClinicalTrials.gov identifier: NCT05246033. Updated February 18, 2022. Accessed April 11, 2022. https://clinicaltrials.gov/ct2/show/NCT05246033
9 A Study of TransCon TLR7/8 Agonist With or Without Pembrolizumab in Patients With Advanced or Metastatic Solid Tumors. ClinicalTrials.gov identifier: NCT04799054. Updated April 4, 2022. Accessed April 11, 2022.
10 IL Believe: A Study of TransCon IL-2 β/γ Alone or in Combination With Pembrolizumab or Chemotherapy in Participants With Locally Advanced or Metastatic Solid Tumors (IL Believe). ClinicalTrials.gov identifier: NCT05081609. Updated March 8, 2022. Accessed April 11, 2022. https://clinicaltrials.gov/ct2/show/NCT05081609

Abbreviations: CNP, C-type Natriuretic Peptide; hGH, Human Growth Hormone; IL, Interleukin; PTH, Parathyroid Hormone; TL, Toll-like