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|Ascendis Pharma Announces Completion of Target Enrollment in Phase 3 Trial of TransCon Growth Hormone for Pediatric Growth Hormone Deficiency|
“Our TransCon Growth Hormone product candidate, which is in development as a once-weekly therapy for pediatric GHD, is now one step closer to patients. We have exceeded our target enrollment in this pivotal trial and we look forward to announcing top-line results early next year,” said
The heiGHt Trial is a randomized, open-label, active-controlled phase 3 study evaluating treatment-naïve children with GHD who receive either once-weekly TransCon Growth Hormone (0.24 mg/kg/week) or daily Genotropin® (34 µg/kg/day or 0.24 mg/kg/week). The primary endpoint of the trial is annualized height velocity at 52 weeks. The primary statistical analysis will be a non-inferiority analysis comparing the two arms.
Patients completing the heiGHt Trial may enroll in the enliGHten Trial, the company’s open-label long-term extension study.
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 ability to apply the TransCon technology platform to build a leading rare disease commercial company, (ii) our expectations regarding when we will announce top-line results from the phase 3 heiGHt Trial, (iii) our expectations regarding the number of patients who will qualify for the phase 3 heiGHt Trial, (iv) our expectations regarding our ability to create therapies with potential for best-in-class efficacy, safety and/or convenience and (v) our product pipeline. We 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 we make, including the following: unforeseen safety or efficacy results in our TransCon Growth Hormone, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to the development of TransCon Growth Hormone, TransCon PTH and TransCon CNP or other development programs, general and administrative expenses, other research and development expenses and our business generally; delays in the development of TransCon Growth Hormone, TransCon PTH and TransCon CNP or other development programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; dependence on third party manufacturers to supply study drug for planned clinical studies; and our ability to obtain additional funding, if needed, to support our business activities. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to our business in general, see our current and future reports filed with, or submitted to, the