Ascendis Pharma A/S Announces Preliminary 58-Week Results from Open-Label Extension of Phase 2 PaTH Forward Trial of TransCon PTH in Adult Hypoparathyroidism Demonstrated Durable Benefit and a Well-Tolerated Safety Profile
– 58 subjects continue in the open-label extension study for TransCon PTH –
“Today we announced results which demonstrated, after one year of treatment, that TransCon PTH was well-tolerated at all doses and provided durable benefit in adults with HP. Data from these subjects provided evidence suggesting they are establishing physiologic calcium metabolism based upon normalization of 24-hour urine calcium excretion and a downward trend toward mid-normal levels in bone markers without the use of therapeutic calcium and vitamin D supplementation. In addition, patient data also showed continued normalized quality of life as measured by the SF-36 questionnaire. Taken together, we believe these results further support the use of TransCon PTH as a potential new treatment paradigm for adults living with HP,” said
“What is particularly rewarding to me is that all 58 patients continue to participate in the open label extension. I believe this is a reflection of the positive impact TransCon PTH is having on these individuals’ lives,” continued
Key Findings of the Preliminary OLE Results of PaTH Forward Trial at 58 weeks
- 58 subjects continue in the open-label extension beyond 58 weeks as of
May 7, 2021
- Continued treatment with TransCon PTH demonstrated that:
- 91% of subjects were off standard of care therapy defined as no active vitamin D and ≤600 mg/day of calcium supplements
- Urinary calcium maintained in the normal range
- Bone markers trended towards the mid-normal levels
- Quality of life benefits measured by SF-36 continued within normal range
- TransCon PTH was well-tolerated at all doses administered
- No treatment-related serious or severe adverse events occurred, and no treatment-emergent adverse events (TEAEs) led to discontinuation of study drug
- No change to the safety profile in the OLE portion of the study
A slide presentation with these data is available at the Investors & News section of the company’s website here: https://investors.ascendispharma.com/
TransCon PTH Program Update
In addition to PaTH Forward,
Ascendis plans to submit a Clinical Trial Notification to the
About the PaTH Forward Trial
PaTH Forward is a global, phase 2, randomized, double-blind, placebo-controlled group trial evaluating the safety and efficacy of three fixed doses of TransCon PTH (15, 18 or 21 µg/day or placebo). The trial enrolled 59 adult subjects with chronic HP who received standard of care or were previously treated with PTH therapies. The goal of PaTH Forward is to evaluate TransCon PTH control of serum and urinary calcium and identify a titration regimen for complete withdrawal of standard of care (i.e., active vitamin D and calcium supplements). PaTH Forward has introduced a ready-to-use pre-filled pen injector and assesses disease-specific patient-reported outcomes. After four weeks of fixed dosing, all subjects were eligible to enter an open-label extension period with the opportunity to receive a customized maintenance dose of TransCon PTH to evaluate long-term safety and efficacy.
About TransCon PTH1
TransCon PTH is an investigational once-daily long-acting prodrug of parathyroid hormone (PTH[1-34]) in development as a treatment for adult hypoparathyroidism (HP) designed to restore PTH at physiologic levels for 24 hours each day to address both the short-term symptoms and long-term complications of the disease. TransCon PTH was granted Orphan Drug Designation (ODD) by the
About Hypoparathyroidism (HP)2,3,4,5,6,7
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 200,000 patients in
HP remains among the few hormonal insufficiency states without a replacement therapy that restores the missing hormone at physiologic levels. 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 to 8-fold greater risk of renal disease compared to healthy controls.
About TransCon™ Technology
TransCon refers to “transient conjugation.” The proprietary TransCon platform is an innovative technology to create new therapies that are designed to potentially 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 conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable 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 in multiple therapeutic areas, and can be used systemically or locally.
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 is headquartered in Copenhagen,
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 Ascendis’ 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) Ascendis’ expectations regarding the timing of topline results from PaTHway, (ii) Ascendis’ plans to submit a Clinical Trial Notification to the
Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and TransCon are trademarks owned by the Ascendis Pharma Group. © May 2021 Ascendis Pharma A/S.
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1 Karpf DB, et al. J Bone
2 Mannstadt M, et al. Nature Reviews 2017, 3: 17055
4 Hadker N, et al. Endo Pract. 2014, 20(7);671-679.
5 Powers J, et al. J Bone
6 Mitchell DM, et al. J Clin Endocrinol Metab 2012, 97(12): 4507-4514
7 Underbjerg L, et al. J Bone
Source: Ascendis Pharma