Ascendis Pharma reports positive Phase 2 results for its once-weekly human growth hormone product, ACP-001, in growth hormone deficient adults.

Palo Alto, California and Copenhagen, Denmark, September 13th, 2011 – Ascendis Pharma, a privately held specialty pharmaceutical company, today announced positive results of a Phase 2 clinical study of ACP-001 in growth hormone deficient adults. The Phase 2 clinical study met all primary and secondary endpoints, the primary efficacy endpoint being the change in IGF-I levels from baseline compared to daily growth hormone. Conducted in a number of Western European countries, the Phase 2 trial was designed to investigate safety, tolerability, pharmacokinetics and pharmacodynamics in 37 adult patients with growth hormone deficiency. The study was a randomized, four-week, multiple-dose study comparing three dose levels of once-weekly ACP-001 to daily growth hormone.

ACP-001 administered once-weekly was as effective in stimulating IGF-I as seven daily injections of the equivalent weekly dose of growth hormone. ACP-001 stimulated a doseproportional increase in IGF-I levels across the three dose levels. IGF-I exposure was maintained throughout the week, thereby achieving a low peak-to-trough ratio. The Phase 2 trial also confirmed the positive safety profile of ACP-001 previously observed in the Phase 1 clinical trial, and safety comparable with daily growth hormone. No treatment-emergent anti-hGH antibody formation was observed during this multiple-dose trial.

ACP-001 was administered as a subcutaneous injection through a very fine needle (30G), and no difference in injection-site reactions were noted between ACP-001 and daily growth hormone.

“I am excited that Ascendis has advanced this once-weekly growth hormone to the pointthat its efficacy and safety has been established in adult patients with growth hormone deficiency. The once-weekly profile of our product meets the goals of patients and endocrinologists worldwide. We look forward to continuing clinical trials in both adults as well as children with growth deficiency.” stated Jan Møller Mikkelsen, President and Chief Executive Officer of Ascendis Pharma.

About ACP-001
Ascendis Pharma is developing ACP-001 for the treatment of children and adults with growth hormone deficiency. Today, these patients are treated with daily injections of growth hormone. ACP-001 is a human growth-hormone prodrug that releases efficacious levels of unmodified human growth hormone in a controlled manner, allowing the drug to be administered once weekly. ACP-001 is unique in that it is an inactive prodrug that releases free growth hormone identical to endogenous growth hormone, maintaining the same physiological actions as growth hormone released from the pituitary. For further information on the characteristics and advantages of ACP-001, please go to
The annual market for human growth hormone is currently $3 billion, with sales continuing to grow.

About Ascendis Pharma
Ascendis Pharma is focused on fast and low-risk internal development of differentiated versions of marketed high-value specialty drugs. Ascendis is the leading company in transient conjugation technology, enabling clinically superior long-acting versions of proteins, peptides and small molecules.
Ascendis Pharma creates clinically differentiated drugs using the company’s innovative prodrug technology platform, TransCon. The reversible TransCon linkers conjugate peptides, proteins and small molecules to carrier molecules in a transient manner. This unique reversibility allows the release of unmodified active drug from the prodrug in a controlled, sustained-release fashion.
In addition to ACP-001, Ascendis Pharma has five other products in its internal portfolio. In the field of diabetes, Ascendis has a large strategic collaboration with Sanofi which includes product opportunities created via TransCon technology such as a once-weekly insulin product, a once-monthly GLP-1 product, and a fixed-dose combination of insulin and GLP-1 for weekly administration.

Jan Møller Mikkelsen, President and CEO, Ascendis Pharma (+1) 650 617 3407.


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