COPENHAGEN, Denmark, April 28, 2015 /PRNewswire/ -- Ascendis Pharma A/S (Nasdaq: ASND), a clinical stage biotechnology company that applies its TransCon technology to address significant unmet medical needs, announced today that its Phase 1 single ascending dose study of TransCon Treprostinil produced dose-dependent increases in plasma treprostinil levels in-line with expectations. However, treprostinil-related injection-site tolerability issues did not meet the criteria defined in the target product profile. Ascendis now intends to conduct additional research on new product formulations of TransCon Treprostinil and plans to resume clinical development when product improvements to mitigate current limitations have been addressed.
Jan Mikkelsen, Chief Executive Officer of Ascendis, stated "While we are pleased with the pharmacokinetic results observed in this study, and believe they affirm the potential of our TransCon technology, TransCon Treprostinil did not reproduce the tolerability profile observed in preclinical studies." Mr. Mikkelsen continued, "As we look forward to the remainder of 2015, we plan to finalize and report topline data from our Phase 2 pediatric study of TransCon Growth Hormone by mid-year and prepare for the initiation of our Phase 3 pediatric study in mid-2016. We will also continue to develop our internal pipeline of therapeutics for orphan diseases."
About Ascendis Pharma A/S Ascendis Pharma is applying its innovative TransCon technology, which combines the benefits of prodrug and sustained release technologies, to develop a pipeline of best-in-class therapeutics that address significant unmet medical needs. The TransCon technology can be applied to existing drug therapies, including proteins, peptides and small molecules, to create prodrugs that provide for the predictable and sustained release of an unmodified parent drug.
The Ascendis Pharma pipeline includes TransCon Growth Hormone, a proprietary program that has completed a Phase 2 study in adults with growth hormone deficiency, or GHD. Ascendis Pharma is currently conducting a Phase 2 study of TransCon Growth Hormone in children with GHD. Ascendis Pharma is also developing its wholly-owned TransCon Treprostinil for the treatment of pulmonary arterial hypertension, or PAH. In addition, Ascendis Pharma has formed collaborations focused on leading products in large markets that are of strategic importance to its collaboration partners. These collaborations are with Sanofi in diabetes and Genentech in the field of ophthalmology.
Forward-looking statements 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 strategy, future operations, plans for pre-clinical and clinical research and development activities, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the following: plans, if any, for the further research and development of new formulations of TransCon Treprostinil, or the potential for success of those plans if undertaken; the projected timing to finalize and report topline data from our ongoing Phase 2 pediatric study of TransCon Growth Hormone in mid-2015; our plans to prepare for the initiation of a Phase 3 pediatric study of TransCon Growth Hormone in mid-2016, and the focus of internal pipeline development efforts on orphan diseases, and the success of those efforts, if any. Ascendis Pharma may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis Pharma makes, including the following: unforeseen safety or efficacy results in our lead development program TransCon Growth Hormone, TransCon Treprostinil or other development programs; unforeseen expenses related to the development of TransCon Growth Hormone, TransCon Treprostinil or other development programs, general and administrative expenses, other research and development expenses and the business of Ascendis Pharma generally; delays in the development of TransCon Growth Hormone or TransCon Treprostinil related to manufacturing, regulatory requirements, results of clinical and pre-clinical studies, speed of patient recruitment or other unforeseen delays; dependence on third party manufacturers to supply study drug for ongoing and planned clinical studies; and the ability of Ascendis Pharma to obtain additional funding, if needed, to support its business activities. These and other risks are described in greater detail in the "Risk Factors" section of Ascendis Pharma's periodic reports filed with the Securities and Exchange Commission. Forward-looking statements do not reflect the potential impact of any future in-licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments Ascendis Pharma may enter into or make. Ascendis Pharma does not assume any obligation to update any forward-looking statements, except as required by law.
Investor contact: Martin Auster, M.D. Chief Business Officer (650) 617-3403 [email protected]