Latest news with #hypoparathyroidism
Yahoo
07-07-2025
- Business
- Yahoo
Ascendis to Share Its Latest Endocrinology Rare Disease Data at ENDO 2025
COPENHAGEN, Denmark, July 07, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced two oral presentations at ENDO 2025, the annual meeting of the Endocrine Society being held July 12-15, 2025, in San Francisco. Dr. Aliya Khan will present new data from Week 156 of the Company's Phase 3 PaTHway Trial demonstrating the safety and efficacy of long-term treatment with TransCon® PTH (palopegteriparatide) in adults with hypoparathyroidism – including maintenance of serum and urine biochemistries within normal levels and sustained improvement in renal function. Dr. Carlos Bacino will present additional safety and tolerability data from Week 52 of the Company's pivotal ApproaCH Trial of once-weekly TransCon® CNP (navepegritide) in children with achondroplasia. 'Ascendis is making important advances in its growing Endocrinology Rare Disease portfolio,' said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Sciences and Chief Medical Officer at Ascendis Pharma. 'We are pleased to partner with renowned investigators during ENDO 2025 to share data demonstrating the long-term benefits and safety of our groundbreaking treatment for adults with hypoparathyroidism, as well as data that we believe supports the transformative potential of our investigational therapy for children with achondroplasia.' Hypoparathyroidism OR-10-05Saturday, July 122:15–2:30 PM PacificRoom 153 Oral PresentationEfficacy and Safety of Palopegteriparatide Treatment in Adults With Hypoparathyroidism: 3-Year Results from the Phase 3 PaTHway Trial Presented by Dr. Aliya KhanClinical Professor of Medicine, Division of Endocrinology & Geriatrics; Director of the Calcium Disorders Clinic at McMaster University Achondroplasia OR-36-05Monday, July 142:15–2:30 PM PacificRoom 308 Oral PresentationSafety and Tolerability of Navepegritide Treatment in Children With Achondroplasia: 52-Week Results from the Pivotal ApproaCH TrialPresented by Dr. Carlos BacinoProfessor, Molecular and Human Genetics at Baylor College of Medicine; Chief of Genetic Services at Texas Children's For more program information, please visit the ENDO 2025 website. About HypoparathyroidismHypoparathyroidism is an endocrine disease caused by insufficient levels of parathyroid hormone (PTH), the primary regulator of calcium and phosphate balance in the body, acting directly on bone and kidney and indirectly on the intestine. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment. Post-surgical hypoparathyroidism accounts for the majority of cases (70-80%), while other etiologies include autoimmune and idiopathic causes. About AchondroplasiaAchondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, causing serious muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia vary across different stages of life. Throughout infancy and childhood, observed complications include spinal abnormalities, enlarged brain ventricles, impaired muscle strength and stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; many of these persist or worsen in adulthood. These medical complications can have detrimental effects on quality of life, physical functioning, and psychosocial function. Individuals with achondroplasia often require multiple surgeries and procedures to alleviate the condition's many complications. About Ascendis Pharma A/SAscendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit to learn more. 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 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) the transformative potential of TransCon CNP for children with achondroplasia; (ii) Ascendis' ability to apply its TransCon technology platform to make a meaningful difference for patients; and (iii) Ascendis' application of its TransCon technologies to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis 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 Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis' products and product candidates; unforeseen safety or efficacy results in Ascendis' development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis' development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis' business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis' ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors, including tariffs and trade policies. 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 Ascendis' business in general, see Ascendis' Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 12, 2025, and Ascendis' other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, and TransCon are trademarks owned by the Ascendis Pharma group. © July 2025 Ascendis Pharma A/S. Investor Contacts: Media Contact: Sarada Weerasinghe Melinda Baker Ascendis Pharma Ascendis Pharma ir@ media@ Patti Bank ICR Healthcare +1 (415) 513-1284
Yahoo
24-06-2025
- Business
- Yahoo
Oppenheimer Hikes Ascendis Pharma Price Target on Strong Yorvipath Demand Outlook
Ascendis Pharma A/S (NASDAQ:ASND) is one of billionaire Stan Druckenmiller's top stock picks with huge upside potential. Oppenheimer maintained its Outperform rating on Ascendis Pharma A/S (NASDAQ:ASND) while increasing its price target from $215 to $224 on June 13. The change comes after a commissioned survey of 20 endocrinologists in the United States who treat hypoparathyroidism showed a high initial demand for the company's drug, Yorvipath. According to doctors, 20–30% of their hypoparathyroid patients are expected to be taking the medication within a year. According to the poll, 90% of physicians would recommend Yorvipath to maintain kidney function before renal impairment develops. This affinity is consistent with the prevalence of renal impairment among patients with hypoparathyroidism. The primary barriers to adoption, according to the majority of physicians, are access and reimbursement; however, Oppenheimer indicated that these worries 'may be more anticipation than reality.' Yorvipath's first full quarter in the United States was described by the firm as 'just the beginning of a launch that will outpace expectations.' Ascendis Pharma A/S (NASDAQ:ASND) is a biopharmaceutical company that develops and distributes novel treatments for unmet medical needs, especially in the fields of oncology and endocrinology. While we acknowledge the potential of ASND as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. Read More: and Disclosure: None.
Medscape
21-05-2025
- Health
- Medscape
Palopegteriparatide Benefits Sustained in Hypoparathyroidism
ORLANDO, Fla. — After 2 years of treatment with the parathyroid hormone (PTH) analog palopegteriparatide (Yorvipath), adults with chronic hypoparathyroidism demonstrated continued improvement in renal function and skeletal dynamics, with no new safety issues identified. In hypoparathyroidism, low levels of PTH lead to hypocalcemia, hypercalciuria, and kidney damage, as well as reduced bone turnover and elevated fracture risk. Conventional treatment doesn't address the underlying defect and imposes a significant pill burden on patients, Lynn A. Kohlmeier, MD, director of endocrinology at Endocrinology and Spokane Osteoporosis, Spokane, Washington, explained at American Association of Clinical Endocrinology (AACE) Annual Meeting 2025. 'Conventional therapy for hypoparathyroidism consists of active D, prescription calcitriol, and multiple calcium supplements, which we know are really tough for our patients to take. It alleviates the hypocalcemic symptoms and hopefully keeps them from crashing and having laryngospasm or tetany or seizures. But if they're overtreated, or if they're dehydrated, the calcium could get too high and they can have renal concerns. The bottom line is this doesn't restore parathyroid hormone physiology,' Kohlmeier said. For PTH replacement therapy in hypoparathyroidism, 'we want something that provides PTH levels at a physiologic range to restore downstream calcitriol, active D production, promoting independence from conventional therapy, but…[also] normalizing serum calcium and urine calcium and phosphate, skeletal health, and quality of life. Palopegteriparatide is essentially that,' she explained. Palopegteriparatide, a once-daily injectable, was approved in the United States in August 2024 for the treatment of adults with hypoparathyroidism. That approval was based on the phase 3 randomized, double-blind, placebo-controlled PaTHway trial of 82 adults with hypoparathyroidism. In week 26 of this this study, 79% (48/61) vs just 5% (1/21) of patients randomized to palopegteriparatide vs placebo met the primary efficacy endpoint of achieving normal serum calcium levels without requiring conventional therapy (no active vitamin D and ≤ 600 mg/d calcium; P < .0001). The 26-week double-blind period was followed by a 182-week open-label period during which all participants were given palopegteriparatide, titrated to optimal dose. At week 104 (2 years), data were available for 76 of the original participants. Of that group, 97% (74/76) achieved independence from conventional therapy, with mean serum calcium and phosphate in the normal ranges. Independence from active vitamin D supplementation was achieved by 100% of the subjects, regardless of baseline kidney function. Asked to comment, endocrinologist Sean Ho Yoon, MD, assistant professor of medicine at the Zucker School of Medicine at Hofstra/Northwell, Hempstead, New York, told Medscape Medical News , 'This study, in the short term, proved that it is working. And the significance of this extension data is that even after 104 weeks, it still maintains the efficacy without complications.' Yoon also noted that the dosing did not appear to be a problem in the study, although Kohlmeier didn't present the titration details. 'Giving too much PTH can lead to too much bone turnover and high calcium in the body. But I think the study proved that as long as you're monitoring calcium regularly and titrating the dosage accordingly, that initial efficacy can be maintained while minimizing the complications.' At 2 years, independence from therapeutic doses of calcium was achieved by 95% (21/22) of participants with a baseline estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2 and 98% (53/54) of those with a baseline eGFR ≥ 60 mL/min/1.73 m2. Improvements in eGFR from baseline to week 104 occurred overall but were numerically greater in those with lower eGFR at baseline. Normalization of mean 24-hour urine calcium excretion was seen by week 26 in the treatment group, and reductions continued through week 104, from 393 mg/d at baseline to 151 mg/d in the 43 patients initially randomized to palopegteriparatide and from 330 mg/d to 183 mg/d in 14 from the placebo group who were switched to the drug after 26 weeks. (Normal urine calcium levels are ≤ 300 mg/d for men and ≤ 250 mg/d for women.) Mean levels of the bone turnover markers procollagen type 1 N-terminal propeptide and C-terminal telopeptide of type 1 collagen initially rose from baseline to week 26 with palopegteriparatide and then were maintained in the normal range at week 104, consistently in men, postmenopausal women, and premenopausal women. Mean bone mineral density T-scores and Z-scores declined from elevated baseline levels and stabilized within the normal range through week 104 across sex and menopausal status groups. Yoon said it was noteworthy that 'they were able to chemically prove with the bone markers that bone turnover is happening again with the PTH replacement. I'm curious, for the longer term, whether that actually helps reduce fractures. I think it's too premature to say that about the bone benefit, but it is promising that biochemically the bone turnover increased back to the age and sex-matched normal range.' Treatment-emergent adverse events (TEAEs) occurring in 5% or more of the 80 participants for whom data are available included injection site reactions (26.2%), hypercalcemia (13.8%), nausea (8.8%), headache (7.5%), hypocalcemia (7.5%), and postural orthostatic tachycardia syndrome (5.0%). Most TEAEs were mild or moderate and generally reported at similar rates across baseline eGFR groups, Kohlmeier said. The sole exception was the occurrence of TEAEs related to hyper- or hypocalcemia leading to an emergency or urgent care visit and/or hospitalization. These events were reported in 7.5% of patients overall, with four instances in the lower baseline eGFR group and two in the higher eGFR group. There was one death, a fatal cardiac arrest, not believed to be related to the drug. Overall, palopegteriparatide was associated with significant and sustained improvement in renal function and skeletal dynamics in adults with chronic hypoparathyroidism and was generally well-tolerated with no new safety signals identified, Kohlmeier concluded. The study was funded by Ascendis Pharma. Kohlmeier reported receiving research funding from Alexion/Amolyt and Ascendis Pharma, being on the speakers bureau and receiving honoraria from Amgen and Ascendis Pharma, and being a consultant/advisor for Alexion and Ascendis Pharma. Yoon had no disclosures.
Medscape
13-05-2025
- Health
- Medscape
Thyroid Surgery Complication May Not Affect Quality of Life
A population-based Swedish study showed no significant differences in health-related quality of life between patients with and without permanent hypoparathyroidism after total thyroidectomy over a mean follow-up duration of 11 years. METHODOLOGY: Researchers in Sweden conducted a population-based retrospective cohort study to examine differences in health-related quality of life between patients with and without hypoparathyroidism who underwent total thyroidectomy for benign thyroid disease between 2005 and 2015. They analysed data of 46 patients (mean age, 35.8 years) with permanent hypoparathyroidism and 653 patients (mean age, 45.7 years) without the condition. Permanent hypoparathyroidism was defined as low serum levels of parathyroid hormone with hypocalcaemia at 12 months post-surgery or at least one failed attempt to manage levels of calcium and/or active vitamin D at 12-24 months post-surgery. The quality of life was assessed using the Swedish version of the 36-item Short Form Health Survey questionnaire, evaluating eight health domains and two summary scores. The mean follow-up duration of the study was 10.9 years. TAKEAWAY: No significant differences in baseline characteristics, including indication for surgery, were observed between patients with and without permanent hypoparathyroidism; however, those with permanent hypoparathyroidism were significantly younger than those without (mean age, 35.8 vs 45.7 years; P < .001). < .001). Similarly, no significant differences were observed between patients with and without permanent hypoparathyroidism across all health domains and the summary component scores ( P > .05 for all). > .05 for all). A comparable proportion of patients with and without permanent hypoparathyroidism scored within or above the average range on the physical component summary ( P = .802) and the mental component summary ( P = .387). IN PRACTICE: "It is possible that the impact on HRQoL [health-related quality of life] is most prominent during the initial interval and diminishes over time as patients adapt to their life-long complication," the authors wrote. SOURCE: This study was led by Matilda Annebäck, Uppsala University Hospital, Uppsala, Sweden. It was published online on May 07, 2025, in BJS Open . LIMITATIONS: This study did not include data on socioeconomic status, comorbidities, and thyroid-stimulating hormone levels. The response rate of the study was 48.3%, which could have introduced a non-response bias. The 36-item Short Form Health Survey questionnaire might not fully capture aspects unique to hypoparathyroidism. DISCLOSURES: This study was supported by grants from the Bergholm Foundation. The authors declared having no conflicts of interest.
Medscape
06-05-2025
- Health
- Medscape
Heart Health Worsened in Pts With Chronic Hypoparathyroidism
Patients with chronic hypoparathyroidism faced a significantly higher risk for cardiovascular diseases and mortality from cardiovascular causes than control individuals without the condition, with the effect being particularly prominent among women. METHODOLOGY: Researchers conducted this study by merging data from population-based registries in Sweden to assess the risk for cardiovascular diseases in patients with chronic hypoparathyroidism. They included 1982 patients with chronic hypoparathyroidism (mean age, 54.7 years; 76.7% women) and matched them to 19,494 control individuals without the condition. The median follow-up time was 9.09 years for patients with chronic hypoparathyroidism and 8.91 years for control individuals. The outcome was the presence of at least one cardiovascular event such as acute myocardial infarction, atrial fibrillation/flutter, heart failure, valvular heart disease, peripheral artery disease, or stroke or transient ischaemic attack; fatal cardiovascular disease was defined as death from any cardiovascular causes. TAKEAWAY: Patients with chronic hypoparathyroidism showed higher risks for valvular heart disease (hazard ratio [HR], 2.08; 95% CI, 1.67-2.60), peripheral artery disease (HR, 1.78; 95% CI, 1.41-2.26), heart failure (HR, 1.66; 95% CI, 1.44-1.90), atrial fibrillation/flutter (HR, 1.58; 95% CI, 1.38-1.81), and acute myocardial infarction (HR, 1.31; 95% CI, 1.05-1.64) than control individuals. The risk for fatal cardiovascular disease was 59% higher in patients with chronic hypoparathyroidism than in control individuals (HR, 1.59; 95% CI, 1.40-1.80). Women with chronic hypoparathyroidism showed significantly higher risks for valvular heart disease, peripheral artery disease, heart failure, atrial fibrillation, myocardial infarction, and fatal cardiovascular disease than their matched control individuals; however, no significant differences in any cardiovascular outcomes were observed between men with chronic hypoparathyroidism and their matched control individuals. The increased risk for cardiovascular diseases in patients with chronic hypoparathyroidism remained consistent, regardless of the surgical or non-surgical aetiology of chronic hypoparathyroidism. IN PRACTICE: "[The study] findings highlight the need for close monitoring and preventive management of cardiovascular risk factors, particularly in women," the authors wrote. SOURCE: This study was led by Sigridur Björnsdottir, MD, PhD, Karolinska Institutet, Stockholm, Sweden. It was published online on April 28, 2025, in The Journal of Clinical Endocrinology & Metabolism . LIMITATIONS: This study lacked data on biochemical measures, physical activity, height, body weight, and blood pressure and relied on chronic obstructive pulmonary disease as a proxy variable for heavy smoking. Information about dosages of active vitamin D, calcium, and levothyroxine was not available in the registries. DISCLOSURES: This study was supported by a research grant from the Swedish Research Council, Knut and Alice Wallenberg Foundation, Novo Nordisk Foundation, Torsten and Ragnar Söderberg's Foundations, and Kristian Gerhard Jebsen Foundation. Several authors reported serving as research investigators, members of the advisory board and steering committees, and consultants and receiving research grants, consultancy fees, and personal fees from various pharmaceutical companies.
