Advancing our innovative pipeline
to address unmet needs

Selected Publications

ISDS 2019 Presentations

Experiences of Parents of Children With Achondroplasia: Impacts on Quality of Life

K.M. Pfeiffer, M. Brod, D. Viuff, S. Ota, J. Gianettoni, J.A. Leff. Poster presented at the
International Skeletal Dysplasia Society (ISDS) Annual Meeting, September 11–14, 2019
(Oslo, Norway).

An Estimate of the Global Birth Prevalence of Achondroplasia

A. Smith, A. Hill-Ricciuti, S. Anderson, D. Viuff, C. Catsburg, J.A. Leff. Poster presented at
the International Skeletal Dysplasia Society (ISDS) Annual Meeting, September 11–14,
2019 (Oslo, Norway).

Pediatric Achondroplasia: Impacts on Children’s Functioning and Well-Being

K.M. Pfeiffer, M. Brod, D. Viuff, S. Ota, J. Gianettoni, J.A. Leff. Poster presented at the
International Skeletal Dysplasia Society (ISDS) Annual Meeting, September 11–14, 2019
(Oslo, Norway).

TransCon CNP: A Once Weekly Novel C-type Natriuretic Peptide Therapy in Children With Achondroplasia

S. Ota, E.D. Christoffersen, P. Mygind, J. Gianettoni, Z. Lin, D. Viuff, D.B. Karpf, M.
Beckert, V.M. Breinholt, J.A. Leff. Presented at the International Skeletal Dysplasia
Society (ISDS) Annual Meeting, September 11–14, 2019 (Oslo, Norway).

TransCon hGH

The Pivotal Phase 3 heiGHt Trial of Weekly TransCon hGH vs. Daily hGH in Treatment-Naive Subjects with Pediatric Growth Hormone Deficiency

A.K. Maniatis, P. Thornton, P. Hofman, E. Aghajanova, E. Chertok, M. Korpal-Szczyrska, E. Giorgadze, T. Kovalenko, A.D. Shu, D.B. Karpf, W. Song, M. Beckert, J.A. Leff. Poster presented at the Pediatric Academic Societies/Pediatric Endocrine Society Annual Meeting (PAS/PES), April 27–30, 2019 (Baltimore, MD).

TransCon hGH as a Long-Acting Growth Hormone for the Treatment of Pediatric Growth Hormone Deficiency

D.L. Campbell, K.J. Walsh, C. Herz, J.W. Permuy, D.B. Karpf, W. Song, M.Beckert, J.A. Leff, A.D. Shu. Poster presented at the Pediatric Endocrinology Nursing Society (PENS) 2019 National Conference, April 25-28, 2019 (Long Beach, CA).

TransCon™ Growth Hormone in the Treatment of Pediatric Growth Hormone Deficiency: Results of the Phase 3 heiGHt Trial

P. Thornton, P. Hofman, A.K. Maniatis, E. Aghajanova, E. Chertok, M. Korpal-Szczyrska, E. Giorgadze, T. Kovalenko, A.D. Shu, D.B. Karpf, M. Beckert, J.A. Leff. Presented at the Endocrine Society (ENDO) Annual Meeting and Expo, March 23–26, 2019 (New Orleans, LA).

Baseline Demographics of the TransCon Growth Hormone Phase 3 heiGHt Trial

M. Beckert, A. Shu, D.B. Karpf, A. Maniatis, Z. Lin, J. A. Leff. Poster presented at the 57th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE), September 27–29, 2018 (Athens, Greece).

The Rationale and Design of TransCon GH

K. Sprogøe, A. Shu, M. Beckert, E. Mortensen, D.B. Karpf, J.A. Leff. Poster presented at the 57th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE), September 27–29, 2018 (Athens, Greece).

Baseline Demographics of the TransCon Growth Hormone Phase 3 heiGHt Trial

A. Shu, M. Beckert, D.B. Karpf, Z. Lin, J. A. Leff. Poster presented at the 9th International Congress of the Growth Hormone Research & IGF Societies (GRS), September 14–17, 2018 (Seattle, WA).

User Driven Development of a New Device for Weekly Growth Hormone Administration in Pediatric Patients

K.Sprogøe, S. Jensen, H. Egesborg, P.E. Fabricius, B. Sørensen, and S.V. Jacobsen. Poster presented at the 10th International Meeting of Pediatric Endocrinology (IMPE), September 14-17, 2017 (Washington, D.C.).

Development of a Long-Acting Growth Hormone (LAGH): Size Matters

P. Saenger, D.B. Karpf, E. Mortensen, M. Beckert, K. Sprogøe, J. Leff. Poster presented at the 10th International Meeting of Pediatric Endocrinology (IMPE), September 14-17, 2017 (Washington, D.C.).

Design and Clinical Development of TransCon Growth Hormone for Growth Hormone Deficiency

M.Beckert, J.M. Mikkelsen, G.N. Rasmussen, H. Rau, K. Sprogøe, J.A. Leff. Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo, April 1-4, 2017 (Orlando, FL).

User Driven Development of a New Device for Weekly Growth Hormone Administration in Pediatric Patients

S. Jensen, H. Egesborg, P.E. Fabricius, B. Sørensen, S.V. Jacobsen. Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo, April 1-4, 2017 (Orlando, FL).

Design of a Bracketed Dosing Regimen for the Administration of TransCon GH, a Once-Weekly Growth Hormone Product

K. Sprogøe, S. Jensen, H. Egesborg. Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo, April 1-4, 2017 (Orlando, FL).

Design and Rationale for the Height Trial, a Phase 3 TransCon GH Study in Children with Growth Hormone Deficiency

M. Beckert, D.B. Karpf, E. Mortensen, J. Mardell, E.D. Christoffersen, J.A. Leff. Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo, April 1-4, 2017 (Orlando, FL).

The Rationale and Design of TransCon Growth Hormone for the Treatment of Growth Hormone Deficiency

K. Sprogøe, E. Mortensen, D.B. Karpf, J.A. Leff. Endocr Connect. 2017,6(8):R171–R181.

A Randomized Phase 2 Study of Long-Acting TransCon Growth Hormone vs. Daily GH in Childhood GH Deficiency

P. Chatelain, O. Malievsky, K. Radziuk, G. Senatorova, M. Abdou, E. Vlachopapadopolou, Y. Skorodok, V. Peterkova, J. Leff, M. Beckert, the TransCon GH Working Group. J Clin Endocrinol Metab. 2017, 102(5): 1673–1682; doi: 10.1210/jc.2016-3776.

A Phase 2 Trial of Long-acting TransCon Growth Hormone in Adult GH Deficiency

C. Höybye, A.F.H. Pfeiffer, D. Ferone, J.S. Christiansen, D. Gilfoyle, E.D. Christoffersen, E. Mortensen, J.A. Leff, M. Beckert. Endocr Connect. 2017,6(3):129–138.

TransCon PTH

Impacts of Hypoparathyroidism on Patient Work Productivity and Out-of-Pocket Costs

L.T. Waldman, M. Brod, A. Smith, D.B. Karp. Poster presented the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Annual Meeting, May 18-22, 2019 (New Orleans, LA).

The Design of a Long-acting PTH (TransCon™ PTH) Phase 2 Trial in Patients with Hypoparathyroidism, Based on Phase 1 Results in Healthy Subjects

D.B. Karpf, S. Pihl, S. Mourya, E. Kovoor, J. A. Leff. Poster presented at the 46th annual meeting of the European Calcified Tissue Society (ECTS), May 11–14, 2019 (Budapest, Hungary).

Understanding the Patient-centered Impact of Hypoparathyroidism on Functioning and Well-being

M. Brod, L.T. Waldman, A. Smith, D.B. Karpf. Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo, March 23–26, 2019 (New Orleans, LA).

TransCon PTH for Hypoparathyroidism—Design and Results of a Phase 1 TransCon PTH Trial in Healthy Volunteers

D.B Karpf, S. Pihl, E. Mortensen, E. Kovoor, S. Mourya, K. Sprogøe, J.A. Leff. Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2018 Annual Meeting, September 28–October 1, 2018 (Montreal, Canada).

The Effects of TransCon PTH on Bone Turnover Markers in a Phase 1 Trial

D.B. Karpf, S. Pihl, A. Shu, E. Kovoor, S. Mourya, E. Mortensen, J.A. Leff. Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2018 Annual Meeting, September 28–October 1, 2018 (Montreal, Canada).

TransCon PTH for Hypoparathyroidism – Design and Preliminary Results of a Phase 1 TransCon PTH Trial in Healthy Adults

D.B. Karpf, S. Pihl, E. Mortensen, K. Sprogøe, J. A. Leff. Poster presented at the 20th European Congress of Endocrinology (ECE), May 19-22, 2018 (Barcelona, Spain).

TransCon PTH, a Sustained-Release PTH Prodrug for the Treatment of Hypoparathyroidism: Proof-of-Principle in Cynomolgus Monkeys and TPTx Rats

L. Holten-Andersen, M. Guillot, F. Cleemann, M. Felx, S. Pihl, K. Sprogøe, D. Karpf, A. Valera, V.M. Breinholt. Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2017 Annual Meeting, September 8-11 (Denver, CO).

Pharmacokinetics of TransCon PTH, a Sustained-Release PTH Prodrug for Hypoparathyroidism, in Rat and Cynomolgus Monkey

S. Pihl, M. Krusch, L. Holten-Andersen, J. Zettler, F.Cleemann, C. Rasmussen, K. Sprogøe, D. Karpf, V.M. Breinholt. Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2017 Annual Meeting, September 8-11 (Denver, CO).

TransCon PTH, a Sustained Release PTH Analogue for the Treatment of Hypoparathyroidism – Proof-of-Principle in Monkeys and Tptx Rats

K.Sprogøe, L. Holten-Andersen, F. Cleeman, J. Zettler, S. Pihl, V.M. Breinholt. Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo, April 1-4, 2017 (Orlando, FL).

Pharmacokinetics of TransCon PTH, a Sustained-Release PTH Prodrug for Hypoparathyroidism, in Rat and Cynomolgus Monkey

K. Sprogøe, S. Pihl, C.E. Rasmussen, L. Holten-Andersen, F. Cleeman, V.M. Breinholt. Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo, April 1-4, 2017 (Orlando, FL).

TransCon CNP

Structural Optimization of TransCon CNP – Development of a Sustained-Release Prodrug of CNP for Achondroplasia

K. Sprogøe, C. Rasmussen, A. Bernhard, F. Faltinger, T. Woods, S. Schröder, A. Brennauer, J. Zettler, T. Wegge, U. Hersel, V.M. Breinholt. Poster presented at the 67th Annual Meeting of the American Society of Human Genetics (ASHG), October 17-21, 2017 (Orlando, FL).

TransCon CNP, a Sustained-Release Prodrug of C-type Natriuretic Peptide, Prevents Premature Synchondroses Closure in an Achondroplasia Mouse Model

V.M. Breinholt, M. Cornille, N. Kaci, L. Legeai-Mallet. Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2017 Annual Meeting, September 8-11 (Denver, CO).

Pharmacokinetics and Cardiovascular Assessment of TransCon CNP, a Sustained-Release C-type Natriuretic Peptide Prodrug, for the Treatment of Achondroplasia

C.E. Rasmussen, K. Sprogøe, U. Hersel, T. Wegge, F. Faltinger, P.H. Mygind, and V.M. Breinholt. Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2017 Annual Meeting, September 8-11 (Denver, CO).

TransCon CNP, a Sustained-Release Prodrug of C-Type Natriuretic Peptide, exerts Positive Effects on Bone in Juvenile Cynomolgus Monkeys and in a Mouse Model of Achondroplasia

V.M. Breinholt, N. Kaci, C.E. Rasmussen, O. Keil, S. Adermann, U. Hersel, M. Cornille, M. Guillot, N. Doyle, A. Valera; P. Mygind, K. Sprogøe, L. Legeai-Mallet. Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2017 Annual Meeting, September 8-11 (Denver, CO).

TransCon CNP, a Sustained-Release Prodrug of C-Type Natriuretic Peptide, Effects Positive Bone Growth in Young Cynomolgus Monkeys and in a Mouse Model of Achondroplasia

V.M. Breinholt, N.Kaci, C.E. Rasmussen, O. Keil, S. Adermann, U. Hersel, M. Cornille, P. H. Mygind, K. Sprogøe, L. Legeai-Mallet. Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo, April 1-4, 2017 (Orlando, FL).

TransCon CNP, a Sustained-Release C-Type Natriuretic Peptide Analogue, for the Treatment of Achondroplasia

K. Sprogøe, C.E. Rasmussen, U. Hersel, V.M. Breinholt. Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo, April 1-4, 2017 (Orlando, FL).

TransCon Technology

TransCon Technology for Sustained Delivery of Proteins, Peptides and Small Molecules

H. Rau and K. Sprogøe. Presented at the Boulder Peptide Symposium, September 26, 2018 (Denver, CO).

Meeting Unmet Needs: The Sustained-Release of Protein and Peptide Drugs

D.B. Karpf, A.D. Shu, E.D. Christoffersen, T. Kurpiers, E. Mortensen, M. Beckert, J.A. Leff. Poster presented at the 9th International Congress of the Growth Hormone Research & IGF Societies (GRS), September 14–17, 2018 (Seattle, WA).

Meeting Unmet Needs: The Sustained-Release of Protein and Peptide Drugs

B. Laufer, S. Pihl, E. Mortensen, D. B. Karpf. Poster presented at the Controlled Release Society (CRS) Annual Meeting & Exposition, July 22-24, 2018 (New York, NY).

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