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 |  |
Phase 3 heiGHt Trial initiated | > $3 billion2 |  |
| Adult Growth Hormone Deficiency | |
Phase 2 completed | |
||
| TransCon Parathyroid Hormone (PTH) | Hypopara- thyroidism |
|
Pre-IND | > $2 billion3 | |
| TransCon C-Type Natriuretic Peptide (CNP) | Achondroplasia | |
Pre-IND | > $1 billion | |
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 | |
Phase 1 | > $1 billion | Partnering opportunity |
| TransCon Ranibizumab | Ophthalmology | Undisclosed | Undisclosed | — |  |
| TransCon Peptides | Diabetes | Undisclosed | Undisclosed | — |  |
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 | |
|
|
|
| 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 | |
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.