Ascendis Pharma A/S Announces Positive Six-Month Interim Results from a Phase 2 Pediatric Study of Once-Weekly TransCon Growth Hormone for the Treatment of Growth Hormone Deficiency

-- Interim analysis indicates annualized six-month height velocity is comparable to the active comparator, Genotropin®, given at an equivalent cumulative weekly dose

COPENHAGEN, Denmark, Dec. 15, 2014 /PRNewswire/ -- Ascendis Pharma A/S, a biotechnology company that applies its innovative TransCon technology to address significant unmet medical needs, today announced positive six-month interim results from its ongoing Phase 2 pediatric study to evaluate once-weekly TransCon Growth Hormone in children with growth hormone deficiency, or GHD. This interim analysis consists of 25 patients, representing approximately 50% of the anticipated total enrollment in the study, completing all six months of treatment.

"It is encouraging to see the significant mean annualized height velocity achieved by TransCon Growth Hormone at the six-month time point," stated Dr. Ron Rosenfeld, member of the Ascendis Growth Hormone Advisory Board and Professor of Pediatrics at Stanford University (emeritus) and Professor of Pediatrics at Oregon Health and Science University (emeritus). "Based on this interim analysis, once-weekly TransCon Growth Hormone appears comparable to gold-standard daily growth hormone therapies in terms of efficacy, safety and tolerability, with a more convenient dosing regimen that may allow for improved patient compliance and treatment outcomes."

"We are pleased with the progress we are making with the development of once-weekly TransCon Growth Hormone," stated Jan Mikkelsen, President and Chief Executive Officer. "We look forward to completing our Phase 2 pediatric study and reporting top-line data in mid-2015. Based on the results observed thus far, we believe that TransCon Growth Hormone has the potential to be a best-in-class therapy for growth hormone deficient patients."

Highlights from the interim analysis are:

• mean annualized height velocities among the three dosing levels administered weekly ranged from 11.9 cm for the 0.14 mg/kg/week dose to 14.5 cm for the 0.30 mg/kg/week dose, which were comparable to 11.5 cm for the active comparator, daily injections of Genotropin® at a 0.21 mg/kg/week dose;
• there have been no reports of serious or unexpected adverse events;
• injection site reactions were generally mild and transient and occurred in only a few patients and there have been no observations of injection site nodule formation or lipoatrophy;
• maximum hGH blood concentration is comparable between equivalent weekly doses of TransCon Growth Hormone and daily hGH; and
• a dose-proportional increase in IGF-I levels was observed following dosing of the three TransCon Growth Hormone dose levels. Transient point values of IGF‐I SDS > +2 have been observed in a small number of patients and only in the high-dose treatment arm.

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The ongoing Phase 2 pediatric study is being conducted to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of TransCon Growth Hormone in treatment-naive pre-pubertal patients with GHD, who meet internationally recognized criteria for GHD. The study is a 6-month multi-center, randomized study comparing three dose levels of TransCon Growth Hormone (0.14; 0.21; and 0.30 mg hGH/kg/week), administered once per week, to the active control Genotropin (0.21 mg hGH/kg/week), administered as a daily injection.

About Growth Hormone Deficiency
Growth hormone deficiency, or GHD, is a serious orphan disease affecting both children and adults. In children, GHD manifests with short stature, metabolic abnormalities, cognitive deficiencies and poor quality of life. Adult GHD is associated with premature mortality and neuropsychiatric-cognitive, cardiovascular, neuromuscular, metabolic and skeletal abnormalities. The global market for daily injections of human growth hormone was approximately $3 billion in 2013. There are currently no long-acting growth hormone treatment options available in the United States or Europe.

The current standard of care for the treatment of GHD requires patients to receive daily injections of hGH over many years. The administrative burden of daily injections often results in poor patient compliance and can lead to suboptimal treatment outcomes.

About TransCon Growth Hormone
Ascendis Pharma is developing once-weekly TransCon Growth Hormone, an investigational new drug, to address the burden of daily injections and suboptimal treatment outcomes that can result from poor patient compliance. TransCon Growth Hormone is a prodrug that releases unmodified growth hormone, thus maintaining the same mode of action as currently prescribed daily growth hormone therapies. Clinical studies of TransCon Growth Hormone have demonstrated a comparable efficacy, safety, tolerability and immunogenic profile to that of daily growth hormone. If approved, TransCon Growth Hormone may reduce the burden of daily treatment by requiring significantly fewer injections, which may improve patient compliance and treatment outcomes. Ascendis Pharma has successfully completed a Phase 2 study of TransCon Growth Hormone in adults with GHD and is currently conducting a Phase 2 study in children with GHD. Ascendis Pharma expects to report top‐line data from the Phase 2 pediatric study in mid-2015.

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 new and 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 TransCon Treprostinil for the treatment of pulmonary arterial hypertension. In addition to its proprietary programs, Ascendis Pharma has formed collaborations focused on market-leading products in large markets that are of strategic importance to its collaboration partners. These collaborations are with Sanofi in diabetes and an undisclosed market leader in the field of ophthalmology.