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Ascendis Pharma Provides Update on Rare Disease Endocrinology Pipeline and Presents Initial Phase 1 Data for TransCon PTH
“We look forward to significant progress during 2018 for our rare disease endocrinology pipeline,” said
Updates on the company’s three pipeline programs include:
- TransCon Growth Hormone: The company exceeded its target enrollment in the phase 3 heiGHt Trial of TransCon Growth Hormone for pediatric growth hormone deficiency. Given strong enthusiasm for the trial from investigators and subjects, the company has randomized over 160 subjects. Based on one-year follow-up, top-line results are anticipated in the first quarter of 2019.
During the third quarter of 2018, Ascendis also plans to complete enrollment in the fliGHt Trial to evaluate TransCon Growth Hormone in subjects who have previously been treated with daily growth hormone therapy.
- TransCon PTH: Pharmacokinetic data from an ongoing phase 1 trial of TransCon PTH in healthy volunteers demonstrated a half-life of approximately 60 hours, supporting an infusion-like profile with daily administration. Additionally, single ascending doses of TransCon PTH led to sustained and dose-dependent elevations of serum calcium lasting more than 72 hours with low inter-subject variability. This low variability supports the ability to titrate and individualize dosing in patients. TransCon PTH also demonstrated the expected effects on renal calcium reabsorption and down regulation of endogenous PTH(1-84). The company anticipates data from the ongoing phase 1 trial to be presented throughout 2018. The company plans to advance TransCon PTH directly into phase 3 development in the first quarter of 2019.
- TransCon CNP: The company recently initiated the regulatory process in
Australiato enable its first-in-human phase 1 trial for TransCon CNP. Dosing in the phase 1 trial in healthy volunteers is expected to begin in the second quarter of 2018, with top-line data to be announced in the fourth quarter of 2018. There are currently no FDA-approved treatments for achondroplasia.
Slides from the J.P. Morgan presentation may be viewed on the
A live audio webcast of the company’s presentation at
For more information, please visit www.ascendispharma.com.
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 we will initiate a phase 1 healthy volunteer trial and release top-line data for our TransCon CNP program in achondroplasia, (iv) our expectations regarding when we will complete enrollment in the fliGHt Trial, (v) our expectations regarding when we will present data from the phase 1 trial of TransCon PTH, (vi) our ability to move TransCon PTH directly into phase 3 development, (vii) our expectations regarding our ability to create therapies with potential for best-in-class efficacy, safety and/or convenience and (viii) 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
Scott T. SmithChief Financial Officer (650) 352-8389 firstname.lastname@example.org Investor contact: Patti Bank Westwicke Partners(415) 513-1284 email@example.com Media contact: Ami KnoeflerSparkBioComm (650) 739-9952 firstname.lastname@example.org