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

COPENHAGEN, Denmark, July 30, 2015 /PRNewswire/ -- Ascendis Pharma A/S (Nasdaq: ASND), a biotechnology company that applies its innovative TransCon technology to address significant unmet medical needs, today announced positive top-line results from a six-month Phase 2 study to evaluate the safety and efficacy of once-weekly TransCon Growth Hormone in 53 treatment-naïve, pre-pubertal children with growth hormone deficiency, or GHD.

"We are extremely pleased with the top-line results from our Phase 2 pediatric study and believe these data establish once-weekly TransCon Growth Hormone as the potential best-in-class long-acting human growth hormone program," stated Jan Mikkelsen, President and Chief Executive Officer.  Mr. Mikkelsen continued, "Most importantly, these results move us one step closer to fulfilling our goal of developing an innovative therapy that has the potential to improve the real-world treatment outcomes of patients with GHD."

Prof. Pierre Chatelain, M.D., Coordinating Investigator of the Phase 2 pediatric study, former Chairman of the College of Pediatrics at the Université Claude Bernard Lyon 1, and Professor Emeritus of Pediatrics, stated, "I am highly encouraged by the mean annualized height velocity achieved by once-weekly TransCon Growth Hormone in the Phase 2 pediatric study.  Based on these top-line data, once-weekly TransCon Growth Hormone appears comparable to gold-standard daily growth hormone therapies in terms of efficacy, safety and tolerability, with reduced injection frequency that may allow for improved patient compliance and treatment outcomes."

Barbara Lippe, M.D., Professor Emerita of Pediatrics, David Geffen School of Medicine at UCLA and clinical advisor to Ascendis Pharma stated, "For children suffering from GHD, the goal of therapy is to restore safe and effective levels of growth hormone throughout the body so that the child has the potential to normalize metabolism and body composition and achieve a normal adult height.  This Phase 2 study demonstrates that the TransCon prodrug releases unmodified growth hormone at peak and overall exposure levels that are comparable to daily growth hormone therapies.  This preserves the same mode-of-action and distribution within the body as daily growth hormone therapies, for which safety and efficacy have been demonstrated over more than 25 years of clinical experience."

Phase 2 pediatric study design

The Phase 2 pediatric study was conducted to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of TransCon Growth Hormone in treatment-naïve pre-pubertal children with growth hormone deficiency, or GHD, who meet internationally recognized diagnostic criteria for GHD. The study enrolled 53 patients into the treatment phase and was a 6-month multi-center, randomized, open-label 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.

Highlights from the top-line analysis include:

• 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 13.9 cm for the 0.30 mg/kg/week dose, which were comparable to 11.6 cm for the active comparator, daily injections of Genotropin® at a 0.21 mg/kg/week dose;
• there were no reports of drug-related serious or unexpected adverse events;
• injection site reactions were generally mild and transient and were observed at a rate that was similar to the daily hGH control arm;
• there were no observations of injection site nodule formation or lipoatrophy;
• low immunogenicity consistent with published data for daily growth hormone;
• maximum hGH blood concentration was 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. Consistent with expectations, transient point values of IGF-I standard deviation score > +2 have been observed in a small number of patients and primarily in the high-dose treatment arm.

The data announced today represent a top-line analysis, and Ascendis Pharma intends to release the full data set for the Phase 2 pediatric study in early October 2015 at the annual meeting of the European Society for Paediatric Endocrinology, and maintains its plans to initiate a Phase 3 pediatric study of TransCon Growth Hormone in mid-2016.  As currently planned, this pivotal study will be a 12-month, parallel-group study evaluating a single dose of TransCon Growth Hormone versus daily injections of growth hormone, and will be conducted in centers across Europe and North America.

Conference call and webcast information

Ascendis Pharma will host a conference call and webcast on Thursday, July 30, 2015 at 9:00 a.m. ET to discuss the top-line results from the Phase 2 pediatric study.  Telephone numbers for the live conference call are (866) 682-8490 (United States) or +44 (0) 1452 555131 (International).  The webcast can be accessed on the Investor page of the Ascendis Pharma website at www.ascendispharma.com and will be available for replay until close of business on August 31, 2015.

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 2014.  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 at peak and overall exposure levels that are comparable to daily 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 Phase 2 studies of TransCon Growth Hormone in adults and children with GHD, and expects to initiate a Phase 3 pediatric study of TransCon Growth Hormone in mid-2016.

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 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 Phase 2 studies in adults and children with growth hormone deficiency, or GHD.  Ascendis Pharma expects to initiate a Phase 3 pediatric study of TransCon Growth Hormone in mid-2016.  Ascendis Pharma is also developing its wholly-owned TransCon Treprostinil for the treatment of pulmonary arterial hypertension, or PAH.  In addition to its proprietary programs, Ascendis Pharma has formed collaborations focused on leading products in large markets that are of strategic importance to its collaboration partners.  These collaborations are with Sanofi in diabetes and Genentech in the field of ophthalmology.

Forward Looking Statements:

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 strategy, future operations, future financial position, future revenues, projected expenses, prospects, plans and objectives of management are forward-looking statements.  Examples of such statements include, but are not limited to, statements relating to the following: whether top-line data from our Phase 2 pediatric study of TransCon Growth Hormone are indicative of the final results of the study; our plans regarding timing and the release of the final results of our Phase 2 pediatric study of TransCon Growth Hormone; our plans to prepare for and initiate a Phase 3 pediatric study of TransCon Growth Hormone in mid-2016; our expectations regarding TransCon Growth Hormone's potential to become a best-in-class product for GHD patients; and our expectations regarding the potential advantages of TransCon Growth Hormone over other marketed and development stage therapies to treat GHD.  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 differences between the final results from our Phase 2 pediatric study of TransCon Growth Hormone and the top-line data from this study; unforeseen safety or efficacy results in our lead development program TransCon Growth Hormone, TransCon Treprostinil 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 ongoing and planned clinical studies; and our ability to obtain additional funding, if needed, to support its 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, including our Annual Report on Form 20-F for the year ended December 31, 2014 and our Report on Form 6-K submitted on May 18, 2015.  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.

Contact:

Investor contact:
Martin Auster, M.D.
Chief Business Officer
(650) 617-3403
[email protected]

Media contact:
Marion Janic
Rooney & Associates 
(212) 223-4017
[email protected]

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SOURCE Ascendis Pharma A/S

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