- Following successful regulatory discussions, global heiGHt Trial to evaluate the only long-acting native, unmodified growth hormone in development -
COPENHAGEN, Denmark, Aug. 11, 2016 /PRNewswire/ -- Ascendis Pharma A/S (Nasdaq: ASND), a clinical stage biopharmaceutical company that applies its innovative TransCon technology to address significant unmet medical needs, today announced the initiation of the global Phase 3 TransCon Growth Hormone heiGHt Trial in children with growth hormone deficiency (GHD). The heiGHt Trial initiation follows End-of-Phase 2 discussions with the U.S. Food and Drug Administration (FDA), as well as various discussions with regulatory agencies worldwide.
"The Phase 3 heiGHt Trial initiation marks a major milestone for Ascendis and our TransCon Growth Hormone program as we move into the next stage of development," said Jonathan A. Leff, M.D., Senior Vice President and Chief Medical Officer at Ascendis. "This pivotal trial mirrors the design of our Phase 2 study in pediatric GHD that demonstrated comparable safety, efficacy, and tolerability of once-weekly TransCon Growth Hormone to a daily growth hormone therapy. This global trial is designed to support planned regulatory applications worldwide, including in the United States and Europe."
Dr. Leff continued, "The Phase 2 data for our once-weekly sustained-release TransCon Growth Hormone demonstrated the strong efficacy and favorable safety, tolerability, and convenience profile of our product candidate. As the only long-acting growth hormone that provides the same mode of action as daily growth hormone, we believe TransCon Growth Hormone can become a best-in-class option for physicians and patients to address the ongoing unmet need for an effective and convenient long-acting growth hormone therapy."
The heiGHt trial is a randomized, open-label, active-controlled Phase 3 registration study that is designed to enroll approximately 150 children with GHD who have not previously been treated. Patients will receive either once-weekly TransCon Growth Hormone (0.24 mg/kg/week) or daily injections of Genotropin® at 34 µg/kg/day (0.24 mg/kg/week) with a 2:1 randomization in a non-inferiority design. The primary endpoint of the trial is height velocity after twelve months of treatment. Patients completing therapy may then enroll in a planned open-label extension study. Ascendis plans to conduct the trial at sites in North and South America, Europe, the Middle East, North Africa, and Oceania (Australia/New Zealand). Additional information regarding the heiGHt Trial can be found at www.clinicaltrials.gov/ct2/show/NCT02781727. Healthcare professionals may also access additional information regarding TransCon Growth Hormone and the heiGHt Trial at www.heighttrial.com.
About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative TransCon technology to develop an internal pipeline of therapeutics to address unmet medical needs in rare disease indications carrying substantial commercial potential. The Ascendis Pharma internal pipeline consists of existing parent drugs with known pharmacology and features TransCon Growth Hormone, a wholly-owned program that has completed Phase 2 studies in adults and children with growth hormone deficiency. The global Phase 3 heiGHt Trial was initiated in August 2016.
Additionally, Ascendis Pharma has formed collaborations with Sanofi in diabetes and Genentech in the field of ophthalmology, both of which are focused on developing leading products in large markets of strategic importance to these partners.
The TransCon technology combines the benefits of prodrug and sustained release technologies, and is the key driver of Ascendis Pharma's mission to develop a pipeline of therapeutics with best-in-class profiles. The TransCon technology can be applied to a broad range of drug therapies, including proteins, peptides and small molecules, to create prodrugs that provide for the predictable and sustained release of an unmodified parent drug. 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 product pipeline, (ii) the design of our Phase 3 pediatric study of TransCon Growth Hormone to support planned regulatory applications worldwide, (iii) our plans regarding the locations of our Phase 3 pediatric study of TransCon Growth Hormone, (iv) our expectations regarding TransCon Growth Hormone's potential to become a best-in-class product for GHD patients, and (v) the application of our TransCon technology to the creation of product candidates to address significant unmet medical needs in rare disease indications carrying substantial commercial potential. 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 lead development program TransCon Growth Hormone or other development programs; unforeseen expenses related to the development of TransCon Growth Hormone 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 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 U.S. Securities and Exchange Commission (SEC), including our Annual Report on Form 20-F for the year ended December 31, 2015, which we filed with the SEC on April 15, 2016. Forward-looking statements do not reflect the potential impact of any future in-licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments we may enter into or make. We do not assume any obligation to update any forward-looking statements, except as required by law.
Scott T. Smith
Chief Financial Officer
SOURCE Ascendis Pharma