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Ascendis Pharma A/S Introduces Vision 3x3: A Strategic Roadmap Through 2025 to Achieve Sustainable Growth Using Multiple Approaches
“With the clinical validation of our three endocrinology rare disease product candidates, we have built a strong foundation to establish Ascendis as a leading rare disease company,” said
He continued, “Today, we are announcing that oncology is our next therapeutic focus, an area with major unmet medical needs. The cornerstone of our oncology pipeline strategy is to apply the same algorithm for product innovation that resulted in successful clinical validation of 3 out of 3 product candidates in endocrinology rare disease.”
Vision 3x3: Sustainable Growth Through Multiple Approaches
The goal of Ascendis Pharma’s Vision 3x3 strategic roadmap through 2025 is to achieve sustainable growth through multiple approaches:
- Obtain regulatory approval for three endocrinology rare disease products: TransCon hGH for pediatric growth hormone deficiency (GHD), TransCon PTH for adult hypoparathyroidism (HP), and TransCon CNP for achondroplasia.
- Create further growth of the company’s endocrinology rare disease pipeline through:
• Label expansion programs with the goal of obtaining 9 indications in total; and
• Global clinical reach either directly or through partnerships.
- Build an integrated commercial business for the endocrinology rare disease franchise in
North Americaand select European countries, and establish a global commercial presence with partners in other geographic areas.
- Create three independent therapeutic areas, each with a diversified pipeline built on TransCon technologies and the company’s unique algorithm for product innovation. The company has established oncology as the next independent therapeutic area.
Endocrinology Rare Disease Pipeline: Selected Key Milestones
- TransCon hGH: Top-line results from the phase 3 heiGHt Trial for TransCon hGH are expected in the first quarter and top-line results from the fliGHt Trial (switch) are expected in the second quarter. TransCon hGH is a once-weekly therapy in phase 3 development for children with GHD designed to deliver unmodified somatropin with the same benefits as daily human growth hormone (hGH). Ascendis believes this will address a limitation of the current standard of care – treatment compliance with daily injections of hGH – and improve treatment outcomes.
- TransCon PTH: Top-line results from a phase 2 trial for TransCon PTH are expected in the fourth quarter. TransCon PTH is a long-acting prodrug of parathyroid hormone (PTH) in development as a once-daily replacement therapy for HP, a disease characterized by deficient or absent PTH. Unlike current therapies for HP, TransCon PTH is designed to achieve and maintain a steady concentration of PTH within the normal range for 24 hours a day.
- TransCon CNP: A phase 2 trial of TransCon CNP in children with achondroplasia is expected to be initiated in the third quarter. TransCon CNP is a long-acting prodrug of CNP in development as a once-weekly therapy for children with achondroplasia, the most common form of dwarfism, for which there is no
FDA-approved treatment. Achondroplasia is caused by a mutation in the fibroblast growth factor receptor 3 (FGFR3), which leads to an imbalance of the FGFR3 and C-natriuretic peptide (CNP) signaling pathways, causing decreased bone growth and other clinical complications. TransCon CNP is designed to restore this balance by providing sustained, long-term exposure to CNP.
Oncology: A New Therapeutic Area with Experienced Leadership
Ascendis also announced that it has established oncology as its second independent therapeutic area. To lead the oncology research and development programs, Ascendis recently appointed Juha Punnonen, MD, PhD, as Senior Vice President and Head of Oncology.
Dr. Punnonen is an experienced scientist, clinician, and biotechnology/pharma executive with a track record in building and managing R&D teams from early-stage research to development. He has more than 25 years of experience in the discovery, characterization and development of monoclonal antibodies, nanobodies, small molecules, cytokines, and immuno-modulatory proteins. Dr. Punnonen was most recently an Executive Director,
Ascendis Pharma’s oncology division is based in the San Francisco Bay Area.
A live webcast of the J.P. Morgan presentation and associated Question and Answer session will be available today at
The company’s corporate presentation, including the slides from J.P. Morgan, will be available at the same link following commencement of the presentation.
Ascendis Pharma is applying its innovative platform technology to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company utilizes its TransCon™ technologies to create new and potentially best-in-class therapies.
Ascendis is headquartered in
For more information, please visit www.ascendispharma.com.
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding our future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) our intention to obtain regulatory approval for our three endocrinology rare disease candidates, TransCon hGH, TransCon PTH and TransCon CNP, (ii) our intention to grow our endocrinology rare disease pipeline by pursuing label expansion programs, (iii) our intention to pursue oncology as our second of three independent therapeutic area of focus, (iv) our plans to release top-line data for our phase 3 heiGHt trial, (v) our plans to initiate our phase 2 TransCon PTH trial in patients with HP and our expectations regarding the timing of release of the results from such trial, (vi) our plans to initiate our phase 2 TransCon CNP trial for the treatment of children with achondroplasia, (vii) our intention to build an integrated commercial business for the endocrinology rare disease franchise in
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Source: Ascendis Pharma A/S