Ascendis Pharma Announces Completion of Target Enrollment in Phase 3 Trial of TransCon Growth Hormone for Pediatric Growth Hormone Deficiency
COPENHAGEN, Denmark, Jan. 03, 2018 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq:ASND), a biopharmaceutical company that utilizes its innovative TransCon technology to address significant unmet medical needs in rare diseases, today announced it has reached its target enrollment in the phase 3 heiGHt Trial of TransCon Growth Hormone for pediatric growth hormone deficiency (GHD). Given strong enthusiasm for the trial from investigators and subjects, as well as a commitment to those still in screening who may qualify, the company expects to randomize over 160 subjects. Based on one-year follow-up, top-line results are anticipated in the first quarter of 2019.
“Our TransCon Growth Hormone product candidate, which is in development as a once-weekly therapy for pediatric GHD, is now one step closer to patients. We have exceeded our target enrollment in this pivotal trial and we look forward to announcing top-line results early next year,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Our phase 2 data suggested that TransCon Growth Hormone was comparable to a daily growth hormone therapy in terms of efficacy, safety, and tolerability. As a long-acting growth hormone that delivers unmodified growth hormone, we believe TransCon Growth Hormone can help address the ongoing unmet need for a convenient long-acting growth hormone therapy with efficacy, safety and tolerability similar to daily alternatives.”
The heiGHt Trial is a randomized, open-label, active-controlled phase 3 study evaluating treatment-naïve children with GHD who receive either once-weekly TransCon Growth Hormone (0.24 mg/kg/week) or daily Genotropin® (34 µg/kg/day or 0.24 mg/kg/week). The primary endpoint of the trial is annualized height velocity at 52 weeks. The primary statistical analysis will be a non-inferiority analysis comparing the two arms.
Patients completing the heiGHt Trial may enroll in the enliGHten Trial, the company’s open-label long-term extension study. Ascendis Pharma is also conducting the fliGHt Trial to evaluate TransCon Growth Hormone in patients who have previously been treated with daily growth hormone therapy.
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 potentially 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 a phase 3 program for children with growth hormone deficiency (GHD), TransCon PTH, a long-acting prodrug of parathyroid hormone for hypoparathyroidism currently in a phase 1 trial, 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.
For more information, please visit www.ascendispharma.com.
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 future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) our ability to apply the TransCon technology platform to build a leading rare disease commercial company, (ii) our expectations regarding when we will announce top-line results from the phase 3 heiGHt Trial, (iii) our expectations regarding the number of patients who will qualify for the phase 3 heiGHt Trial, (iv) our expectations regarding our ability to create therapies with potential for best-in-class efficacy, safety and/or convenience and (v) our product pipeline. 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 safety or efficacy results in our TransCon Growth Hormone, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to the development of TransCon Growth Hormone, TransCon PTH and TransCon CNP 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, TransCon PTH and TransCon CNP or other development programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; dependence on third party manufacturers to supply study drug for planned clinical studies; and our ability to obtain additional funding, if needed, to support our 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 (SEC), including our Annual Report on Form 20-F for the year ended December 31, 2016, which we filed with the SEC on March 22, 2017. 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.
Internal contact: Scott T. Smith Chief Financial Officer (650) 352-8389 ir@ascendispharma.com Investor contact: Patti Bank Westwicke Partners (415) 513-1284 patti.bank@westwicke.com Media contact: Ami Knoefler SparkBioComm (650) 739-9952 ami@sparkbiocomm.com