Ascendis Pharma A/S Announces Publication of Comprehensive Results from Randomized, Active-Controlled Phase 2 Trial of Once-Weekly TransCon Growth Hormone in Pediatric Patients

COPENHAGEN, Denmark I February 16, 2017 I Ascendis Pharma A/S (ASND), a biopharmaceutical company that utilizes its innovative TransCon technology to address significant unmet medical needs in rare diseases, today announced the publication of its previously presented pediatric phase 2 trial of TransCon Growth Hormone in the Journal of Clinical Endocrinology and Metabolism.

The authors concluded that once-weekly, sustained release TransCon Growth Hormone was comparable to daily growth hormone in terms of efficacy, safety and tolerability in children with growth hormone deficiency (GHD). The publication is available online at: https://academic.oup.com/jcem/article/2996234/A-Randomized-Phase-2-Study-of-Long-Acting-TransCon.

GHD in children is a serious orphan disease, which results in short stature, metabolic abnormalities, and cognitive deficiencies, often leading to decreased quality of life.1 Current GHD treatment requires daily growth hormone injections for many years. However, the administrative burden often results in at least one missed injection per week, contributing to suboptimal outcomes.2 Currently, there are no long-acting growth hormone treatment options commercially available in the United States or Europe.

"Suboptimal outcomes due to poor compliance remain a significant barrier in management of GHD," said Professor Pierre Chatelain, M.D., lead author of the manuscript from Université Claude Bernard Lyon 1. "The data suggest that TransCon Growth Hormone has the potential to address the significant need for a long-acting growth hormone therapy without compromising efficacy, safety and tolerability."

TransCon Growth Hormone is designed to release unmodified growth hormone that diffuses freely into the body's tissues, where it may carry out the effects of native growth hormone with easy-to-remember weekly dosing.

Separately, results of a Phase 2 study evaluating the safety, pharmacokinetics, and pharmacodynamics of TransCon Growth Hormone in adult patients with GHD were published online in the journal Endocrine Connections. The publication is available at: https://academic.oup.com/jcem/article/2996234/A-Randomized-Phase-2-Study-of-Long-Acting-TransCon.

Based on the positive Phase 2 results in pediatric patients, Ascendis has an ongoing global Phase 3 trial of TransCon Growth Hormone compared to daily growth hormone. The randomized, active-controlled heiGHt Trial aims to enroll 150 pediatric patients with GHD by the end of 2017. Healthcare professionals may access additional information regarding TransCon Growth Hormone and the heiGHt Trial at www.heighttrial.com.

About Ascendis Pharma A/S

Ascendis Pharma is applying the TransCon technology platform to build a leading rare disease commercial company. The company utilizes its innovative TransCon technology to address significant unmet medical needs in rare diseases by improving clinically validated parent drugs and creating therapies with potential for best-in-class efficacy, safety and/or convenience.

Ascendis Pharma has a wholly-owned pipeline of rare disease endocrinology programs, including once-weekly TransCon Growth Hormone, which is currently being evaluated in the Phase 3 heiGHt Trial for children with growth hormone deficiency (GHD), TransCon PTH, a long-acting prodrug of parathyroid hormone for hypoparathyroidism, and TransCon CNP, a long-acting prodrug of C-type natriuretic peptide for achondroplasia. Additionally, Ascendis Pharma has multi-product collaborations with Sanofi in diabetes and Genentech in the field of ophthalmology.

SOURCE: Ascendis Pharma

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