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Ascendis Pharma A/S. (3/19/18). "Press Release: Ascendis Pharma Announces Two Posters on Rare Disease Pipeline Programs at ENDO 2018". Copenhagen.

Region Region Chicago, IL
  Country United States (USA)
Organisations Organisation Ascendis Pharma A/S (Nasdaq: ASND)
  Today Ascendis Pharma (Group)
  Group Ascendis Pharma (Group)
  Organisation 2 Endocrine Society
Products Product TransCon Growth Hormone
  Product 2 ENDO 2018 Chicago

- Baseline demographics of TransCon Growth Hormone phase 3 heiGHt Trial presented -

- Multiple ascending dose cohorts from TransCon PTH phase 1 clinical trial reinforced infusion-like profile over 24 hours -

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 publication of two posters featuring its rare disease endocrinology pipeline programs at ENDO 2018, the annual meeting of the Endocrine Society, taking place in Chicago, March 17-20.

The posters, “Baseline Demographics of the TransCon Growth Hormone Phase 3 heiGHt Trial” and “TransCon PTH for Hypoparathyroidism – Design and Preliminary Results of a Phase 1 TransCon PTH Trial in Healthy Adults” were also selected by the Endocrine Society for inclusion in special moderated poster sessions.

“The data presented at ENDO 2018 on our two most advanced product candidates reflect the significant progress we are making with our pipeline. We are focused on developing products that address significant unmet patient needs in rare diseases,” said Jonathan Leff, M.D., Ascendis Pharma’s Chief Medical Officer. “Our TransCon Growth Hormone phase 3 program continues to progress as planned. Our interim TransCon PTH phase 1 trial data will be presented to the endocrinology community for the first time, reinforcing the potential of our candidate to replace and restore PTH to physiological levels for 24 hours per day. Collectively, the data being presented at ENDO reflect the ability of our TransCon technology platform to enable innovative new therapies with the goal to improve patients’ lives.”

Copies of both posters are accessible on the company’s website at the following link:

TransCon GH: heiGHt Trial Baseline Demographics

The heiGHt Trial poster summarizes demographic data from the ongoing global pivotal trial evaluating TransCon Growth Hormone for pediatric growth hormone deficiency (GHD). The poster concluded that the demographic profile of subjects in the heiGHt Trial is generally similar to the daily cohorts of 4 different phase 3 registration trials. Additionally, a calculation based on the final randomization of 161 subjects showed the trial’s statistical power for noninferiority was further strengthened.

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 or dose-equivalent daily Genotropin®. The primary endpoint of the trial is annualized height velocity at 52 weeks. The primary statistical analysis will be a noninferiority analysis comparing the two arms.

Top-line results from the heiGHt Trial are anticipated in the first quarter of 2019.

TransCon PTH: Interim Phase 1 Data Support Infusion-like Profile

The poster on TransCon PTH(1-34), to be presented later today, reports interim data from both single and multiple ascending dose (SAD and MAD) cohorts from the ongoing phase 1 trial in healthy adults.

The PK data from the SAD and MAD cohorts showed that daily administration of TransCon PTH demonstrated a dose-dependent response with a flat infusion-like profile. The PK profile also translated into a predicted PD response consistent with the preclinical data, suggesting the ability to titrate patients with hypoparathyroidism into the normal calcemic range with TransCon PTH. The data demonstrated a predicted renal calcium reabsorption effect, as well as control of both serum and urine calcium. TransCon PTH was generally well tolerated across the likely clinical dose range.

Full results from the phase 1 trial are anticipated in the second quarter of 2018.

About Ascendis Pharma A/S

Ascendis Pharma is applying its innovative prodrug technology to build a leading, fully integrated rare disease company focused on making a meaningful difference in patients’ lives. The company utilizes its TransCon technology with clinically validated parent drugs to create new therapies with potential for best-in-class efficacy, safety and/or convenience.

Ascendis Pharma has a wholly-owned pipeline of three 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

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 our prodrug technology to build a leading, fully integrated rare disease company, (ii) the potential for TransCon PTH to replace and restore PTH to physiological levels for 24 hours per day, (iii) our expectations regarding when we will announce top-line results from the phase 3 heiGHt Trial, (iv) our expectations regarding when we will announce full results from the phase 1 TransCon PTH trial, (v) our expectations regarding our ability to create therapies with potential for best-in-class efficacy, safety and/or convenience and (vi) 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

Investor contact:
Patti Bank
Westwicke Partners
(415) 513-1284

Media contact:
Ami Knoefler
(650) 739-9952

Record changed: 2023-06-05


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