– Following successful regulatory discussions, global heiGHt Trial to evaluate the only long-acting native, unmodified growth hormone in development –
COPENHAGEN, Denmark I August 11, 2016 I 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.
SOURCE: Ascendis Pharma
Post Views: 68
– Following successful regulatory discussions, global heiGHt Trial to evaluate the only long-acting native, unmodified growth hormone in development –
COPENHAGEN, Denmark I August 11, 2016 I 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.
SOURCE: Ascendis Pharma
Post Views: 68
