Ascendis Pharma Announces Results of Study at ENDO 2019 Evaluating Impact of Hypoparathyroidism on Patient Quality-of-Life
“I have lived with HP for over 25 years and have personally experienced how this debilitating disease affects all areas of life - physical, emotional and social,” said
In the study, 42 adult patients with post-surgical (diagnosis at least 6 months) or idiopathic (diagnosis at least 12 months) HP were interviewed. Patients were required to be stable on treatment (oral calcium and vitamin D supplements and/or parathyroid hormone therapy with PTH[1-84]) for at least 3 months. Over half the patients (52.4 percent) were treated with PTH(1-84). Participants were predominantly female (83.3 percent) and had post-surgical HP (85.7 percent).
Despite treatment, 71 percent of patients reported that managing their condition was difficult. Other findings included:
- All patients reported experiencing physical signs and symptoms attributable to HP, including paresthesia (tingling, numbness), muscle cramps and physical fatigue
- 86 percent reported experiencing one or more forms of cognitive dysfunction (collectively known as ”brain fog”), including difficulties with memory/forgetfulness, having a conversation, being able to concentrate, fuzzy thinking and understanding information
- All patients reported that HP interfered with their daily life and caused some psychological issues, including anxiety (81 percent), frustration (64 percent) and depression (62 percent)
- For 95 percent of patients, HP also affected physical functioning and social interactions, such as the ability to exercise and be mobile, as well as the ability to participate in social activities and build/maintain relationships
- Additionally, over half the patients reported difficulty finding primary care physicians, specialists, or emergency department physicians with sufficient working knowledge about the disease.
“These results clearly show the need for a treatment option that can address all aspects of HP to help alleviate the significant quality-of-life issues these patients face every day,” said
TransCon PTH is a long-acting prodrug of parathyroid hormone (PTH[1-34]), in development as a potential once-daily replacement therapy for HP. PaTH Forward is a global phase 2 trial designed to evaluate the safety, tolerability and efficacy of TransCon PTH in adult subjects with hypoparathyroidism. The trial is also evaluating a titration regimen for the complete withdrawal of standard of care (i.e., active vitamin D and calcium supplements). The company 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 in multiple therapeutic areas, and can be used systemically or locally.
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
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. In fact, patients with HP have an estimated 4-fold greater risk of renal disease compared to healthy controls.
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.
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.
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 expectation to receive top-line results from the PaTH Forward 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 new and 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 potential commercial sale, if approved; 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 (
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Source: Ascendis Pharma A/S