Advancing our innovative pipeline
to address unmet needs

Overview

We are applying our TransCon technology to build a fully integrated, rare disease focused biopharmaceutical company with a diversified pipeline of long-acting prodrug therapies having best-in-class profiles. Our TransCon technology establishes a new paradigm that combines the benefits of conventional prodrug and sustained release technologies, and is broadly applicable to proteins, peptides and small molecules. TransCon prodrugs release the active drug in its unmodified form, maintaining the original therapeutic agent’s mode of action.

 

Ascendis Internal Endocrinology Pipeline

PRODUCT CANDIDATE PRIMARY INDICATION CLINICALLY VALIDATED TARGET STATE OF DEVELOPMENT POTENTIAL WORLDWIDE MARKET1 WORLDWIDE COMMERCIAL RIGHTS
TransCon Growth Hormone Pediatric Growth Hormone Deficiency check Phase 3 heiGHt Trial initiated > $3 billion2 ascendis-logo
Adult Growth Hormone Deficiency  check Phase 2 completed ascendis-logo
TransCon Parathyroid Hormone (PTH) Hypopara-
thyroidism
check Pre-IND > $2 billion3 ascendis-logo
TransCon C-Type Natriuretic Peptide (CNP) Achondroplasia check Pre-IND > $1 billion ascendis-logo

Current/Potential Strategic Collaborations

PRODUCT CANDIDATE PRIMARY INDICATION CLINICALLY VALIDATED TARGET  STATE OF DEVELOPMENT POTENTIAL WORLDWIDE MARKET  WORLDWIDE COMMERCIAL RIGHTS
TransCon Treprostinil Pulmonary Arterial Hypertension check Phase 1 > $1 billion Partnering opportunity
TransCon Ranibizumab Ophthalmology Undisclosed Undisclosed genentech-logo
TransCon Peptides Diabetes Undisclosed Undisclosed sanofi-logo

Ascendis Internal Endocrinology Pipeline

PRODUCT CANDIDATE TransCon
GH
TransCon
PTH
TransCon
CNP
PRIMARY INDICATION Pediatric GH Deficiency Adult GH Deficiency Hypopara-
thyroidism
Achondro-
plasia
CLINICALLY VALIDATED TARGET check check check check
STATE OF DEVELOPMENT Phase 3 heiGHt Trial initiated Phase 2 completed Pre-IND Pre-IND
POTENTIAL WW MARKET1 > $3 billion2 > $2 billion3  > $1 billion
WORLDWIDE COMMERCIAL RIGHTS Ascendis Pharma Ascendis Pharma Ascendis Pharma

Current/Potential Strategic Collaborations

PRODUCT CANDIDATE TransCon Treprostinil TransCon Ranibizumab TransCon Peptides
PRIMARY INDICATION Pulmonary Arterial Hypertension Ophthalmology Diabetes
CLINICALLY VALIDATED TARGET check Undisclosed Undisclosed
STATE OF DEVELOPMENT Phase 1 Undisclosed Undisclosed
POTENTIAL WW MARKET > $1 billion  —
WORLDWIDE COMMERCIAL RIGHTS Partnering opportunity Roche Genentech Sanofi

CNP=C-type natriuretic peptide, GH=growth hormone, PTH=parathyroid hormone.

 

1 Based on market data and company estimates.
2 Includes all indications.
3 Based on treatment of ~25% of the U.S. patient population of ~77,000 patients.