Ascendis Pharma reports positive Phase 2 results
Palo Alto, California and Copenhagen, Denmark – Ascendis Pharma, a privately held specialty pharmaceutical company, 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. The phase 2 trial confirmed ACP-001 administered once-weekly was as effective in stimulating IGF-I as seven daily injections of the equivalent weekly dose of growth hormone. The trial also confirmed the positive safety profile of 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. 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. 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.
For more information, please visit www.ascendispharma.com.
Jan Møller Mikkelsen,
President and CEO,
(+1) 650 617 3407.
About Gilde Healthcare Partners
Netherlands-based Gilde Healthcare Partners is a transatlantic growth equity investor focused on private healthcare technologies and services. It has EUR 450 million under management and is actively looking to lead new investments in therapeutics, diagnostics, medical devices and services.
Gilde successfully builds healthcare businesses across Europe and US, investing up to EUR 15 million in a single portfolio company. By investing in companies with clear, achievable business models, Gilde has used its financial resources and network to create significant value for both its investors and the entrepreneurs it backs. For a list of Gilde’s portfolio companies please visit the website at www.gildehealthcare.com.
Gilde Healthcare II is supported by the European Communities Growth and Employment Initiative, MAP - ETF Start up Facility.