Ascendis Pharma Initiates Global Phase 2 Trial for TransCon PTH in Adult Subjects with Hypoparathyroidism
- TransCon PTH is designed to provide parathyroid hormone in the physiologic range for 24 hours each day as a true replacement therapy for hypoparathyroidism -
TransCon PTH is a long-acting prodrug of parathyroid hormone (PTH[1-34]), in development as a potential once-daily replacement therapy for HP.
“Our goal is to control signs and symptoms of hypoparathyroidism in the absence of standard therapies. Unlike current therapies for hypoparathyroidism, once-daily TransCon PTH is designed to restore PTH to physiologic levels for 24 hours each day and fully address all aspects of the disease,” said
PaTH Forward is a global, phase 2, randomized, double-blind, placebo-controlled, parallel group trial that will evaluate three fixed doses of TransCon PTH and a titration regimen for complete withdrawal of standard of care (i.e., active vitamin D and calcium supplements). The trial will be conducted in approximately 40 adult subjects with HP who are currently receiving standard of care therapies at up to 40 sites worldwide. The PaTH Forward trial will introduce a ready-to-use pre-filled pen device and assess disease-specific patient-reported outcomes. After four weeks of dosing, all subjects may enter an open-label extension period with the opportunity to receive TransCon PTH to evaluate long-term safety and efficacy.
About TransCon™ Technology
TransCon refers to “transient conjugation.” The proprietary TransCon platform is an innovative technology to create new therapies that optimize therapeutic effect, including efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic pH and temperature conditions initiate the release of the active, unmodified parent drug in a predictable release manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technology can be applied broadly to a protein, peptide or small molecule, and is applicable in multiple therapeutic areas.
About Hypoparathyroidism
Hypoparathyroidism (HP) is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH), resulting in low calcium and elevated phosphate levels in the blood. HP affects approximately 80,000 patients in
Until recently, HP remained among the few hormonal insufficiency states not treated by replacement of the missing hormone. Standard of care with active vitamin D analogs and calcium supplementation do not fully control the disease and may contribute to risk of renal disease. As a result, patients with HP have an estimated 4-fold greater risk of renal disease compared to healthy controls.
About
Ascendis Pharma is applying its innovative platform technology to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company utilizes its TransCon™ technologies to create new and potentially best-in-class therapies.
Ascendis is headquartered in
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 plans to initiate a Phase 2 trial of TransCon PTH in adult subjects with hypoparathyroidism and our expectation to receive top-line results from this trial in the fourth quarter of 2019, (ii) our ability to apply our platform technology to build a leading, fully integrated biopharma company, (iii) our expectations regarding our ability to create potentially best-in-class therapies and (iv) 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 hGH, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to the development of TransCon hGH, 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 hGH, 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 (
Ascendis,
Internal contact:Scott T. Smith Chief Financial Officer (650) 352-8389 ir@ascendispharma.com Media contact:Ami Knoefler Head ofGlobal Communications (650) 739-9952 ack@ascendispharma.com Investor contact:Patti Bank Westwicke Partners (415) 513-1284 patti.bank@westwicke.com
Source: Ascendis Pharma A/S