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

COPENHAGEN, Denmark I February 11, 2019 I Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon™ technology to address significant unmet medical needs, today announced the filing of an investigational new drug (IND) application with the U.S. Food and Drug Administration for initiation of PaTH Forward, a global phase 2 trial designed to evaluate the safety, tolerability and efficacy of TransCon PTH in adult subjects with hypoparathyroidism (HP). The trial will also evaluate a titration regimen for the complete withdrawal of standard of care (i.e., active vitamin D and calcium supplements).

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 Jonathan Leff, M.D., Chief Medical Officer of Ascendis Pharma. “With PaTH Forward, we are applying our TransCon technology to advance a new treatment option for patients with this debilitating condition.”

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.

Ascendis Pharma anticipates top-line results from the PaTH Forward trial in the fourth quarter of 2019.

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 the United States, the majority of whom develop the condition following damage or accidental removal of the parathyroid glands during thyroid surgery. Patients often experience decreased quality of life. In the short term, symptoms include weakness, severe muscle cramps (tetany), abnormal sensations such as tingling, burning and numbness (paresthesia), memory loss, impaired judgment and headache. Over the long term, this complex disorder can increase risk of major complications, such as extraskeletal calcium depositions occurring within the brain, lens of the eye and kidneys, which can lead to impaired renal function.

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 A/S

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 Pharma currently has a pipeline of three independent rare disease endocrinology product candidates in clinical development and has established oncology as its second therapeutic area of focus. Additionally, Ascendis Pharma has multi-product collaborations with Sanofi in diabetes and Genentech in the field of ophthalmology and continues to expand into additional therapeutic areas for both internal and external development.

Ascendis is headquartered in Copenhagen, Denmark, with offices in Heidelberg, Germany and Palo Alto, California. 

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SOURCE: Ascendis Pharma

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