Latest news with #TransConPTH
Business Insider
15-07-2025
- Business
- Business Insider
Ascendis Pharma announces new data from PaTHway Trial at ENDO 2025
Ascendis Pharma (ASND) announced new data from Week 156 of its Phase 3 PaTHway Trial confirming that long-term treatment with TransCon PTH continued to provide a durable response in adults with hypoparathyroidism regardless of its cause, including improvements in biochemistries, kidney function, and quality of life. Results were shared at ENDO 2025, the annual meeting of the Endocrine Society being held in San Francisco. PaTHway was a Phase 3, randomized, double-blind, placebo-controlled 26-week trial of 82 adults with chronic hypoparathyroidism, followed by an open-label extension period through Week 182. During the initial 26-week blinded period, participants were randomized 3:1 TransCon PTH:placebo. Renal function was assessed by estimated glomerular filtration rate. Safety assessments included 24-hour urine-calcium and treatment-emergent adverse events. Hypoparathyroidism-related symptoms, functioning, and well-being were measured by the Hypoparathyroidism Patient Experience Scale. Overall retention in the trial was high, with 73 of the original 82 adults enrolled completing the 3.5-year trial. At Week 156, 64 patients had normal albumin-adjusted serum calcium levels and 70 patients were independent from conventional therapy. Reflecting clinically meaningful improvements in kidney function, improvements in eGFR from baseline were sustained through Week 156: mean eGFR increased by 8.76 mL/min/1.73 m2 across all participants and by 13.98 mL/min/1.73 m2 in participants with baseline eGFR less than 60. Patients in the trial reported continued improvements from baseline in their hypoparathyroidism-related symptoms and health-related quality of life and showed continued normalization of 24-hour urine calcium excretion through Week 156. In the trial, TransCon PTH treatment was generally well-tolerated, with no new safety signals identified. TEAEs were mostly mild or moderate and no serious TEAEs or discontinuations were related to study drug. Elevate Your Investing Strategy:
Yahoo
14-07-2025
- Health
- Yahoo
New 3-Year Phase 3 Data Confirmed Sustained Response to TransCon® PTH (Palopegteriparatide) Therapy in Adults with Hypoparathyroidism, Including Improvements in Biochemistries, Kidney Function, and Quality of Life
COPENHAGEN, Denmark, July 14, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced new data from Week 156 of its Phase 3 PaTHway Trial confirming that long-term treatment with TransCon PTH (palopegteriparatide) continued to provide a durable response in adults with hypoparathyroidism regardless of its cause (post-surgical, autoimmune, genetic, or idiopathic), including improvements in biochemistries, kidney function, and quality of life. Results were shared over the weekend in an oral presentation by Aliya Khan, M.D., Clinical Professor of Medicine, Division of Endocrinology and Geriatrics and Director of the Calcium Disorders Clinic at McMaster University (Canada) at ENDO 2025, the annual meeting of the Endocrine Society being held in San Francisco. PaTHway was a Phase 3, randomized, double-blind, placebo-controlled 26-week trial of 82 adults with chronic hypoparathyroidism, followed by an open-label extension period through Week 182. During the initial 26-week blinded period, participants were randomized 3:1 TransCon PTH:placebo. Renal function was assessed by estimated glomerular filtration rate (eGFR). Safety assessments included 24-hour urine-calcium and treatment-emergent adverse events (TEAEs). Hypoparathyroidism-related symptoms, functioning, and well-being were measured by the Hypoparathyroidism Patient Experience Scale (HPES). Overall retention in the trial was high, with 73 of the original 82 adults enrolled (89%) completing the 3.5-year trial. At Week 156, 64 patients (88%) had normal albumin-adjusted serum calcium levels and 70 patients (96%) were independent from conventional therapy (defined as taking < 600 mg/day of calcium and not taking active vitamin D). Reflecting clinically meaningful improvements in kidney function, improvements in eGFR from baseline were sustained through Week 156: mean eGFR increased by 8.76 mL/min/1.73 m2 across all participants and by 13.98 mL/min/1.73 m2 in participants with baseline eGFR < 60. Patients in the trial reported continued improvements from baseline in their hypoparathyroidism-related symptoms and health-related quality of life and showed continued normalization of 24-hour urine calcium excretion through Week 156. In the trial, TransCon PTH treatment was generally well-tolerated, with no new safety signals identified. TEAEs were mostly mild or moderate and no serious TEAEs or discontinuations were related to study drug. 'We are pleased to report these latest data from our second clinical trial of TransCon PTH showing sustained efficacy and improvements across key health and quality of life measures, beginning at the earliest timepoints in the Phase 3 PaTHway trial and continuing through three years of treatment,' said Aimee Shu, M.D. Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma. 'These responses, including normalization of skeletal dynamics with significant and clinically meaningful improvements in kidney function, demonstrate the long-term benefits of this treatment option for the vast majority of adults with hypoparathyroidism, regardless of their disease etiology.' 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 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 long term benefits of TransCon PTH therapy for adults with hypoparathyroidism, regardless of disease etiology, (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 Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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
05-05-2025
- Business
- Yahoo
Ascendis to Share Its Latest Endocrinology Rare Disease Data at ESPE & ESE 2025
COPENHAGEN, Denmark, May 05, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it will share the latest data from its hypoparathyroidism, achondroplasia, and growth hormone deficiency (GHD) programs during ESPE & ESE 2025, the joint congress of the European Society for Paediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) being held May 10-13, 2025, in Copenhagen. Four oral presentations will feature Ascendis clinical trial results, including 4-year efficacy and safety data from the Phase 2 PaTH Forward Trial of TransCon PTH (palopegteriparatide) in adults with chronic hypoparathyroidism; Week 52 growth and bone morphometry data from the pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia; and efficacy and safety data from the Phase 3 foresiGHt Trial of TransCon hGH (lonapegsomatropin) in adults with growth hormone deficiency. 'As new treatments and clinical practices transform the outlook for rare endocrine diseases, we are pleased to partner with trial investigators and leading experts to share new data highlighting clinical benefits of our novel therapies with attendees of ESPE & ESE 2025,' said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma. The full range of Ascendis Pharma events and updates at ESPE & ESE 2025 include the following: Hypoparathyroidism Sunday, May 1109:55 - 10:25 CEST Congress TheaterFoyer F5 Product TheaterRestoring Physiological Levels of PTH in Chronic HypoparathyroidismClick here to see the full session outline. Sunday, May 1116:55-17-55 CEST& Monday, May 1217:15-17:45 CESTPoster Hall Poster P238Estimating the Risk of Chronic Kidney Disease Progression in Chronic Hypoparathyroidism: A Restrospective Matched Cohort Study, Using Real-World Data from England Monday, May 1213:00 - 14:30 CESTRoom D5 SymposiumBeyond Conventional Care: Redefining Treatment Success in Chronic HypoparathyroidismClick here to see the full session outline. Monday, May 1215:55-16:05 CESTRoom C2 Oral PresentationLong-Term Efficacy & Safety of Palopegteriparatide Treatment in Adults with Chronic Hypoparathyroidism: 4-Year Results from the Phase 2 PaTH Forward TrialPresented by Dr. Andrea Palermo Achondroplasia Sunday, May 1115:55-17:25 CESTRoom C2 SymposiumAssessing HRQoL in Achondroplasia Across the Life CourseClick here to see the full session outline. Monday, May 1215:15-15:25 CESTRoom C2 Oral PresentationEffects of Navepegritide on Bone Morphometry in Children with Achondroplasia: 52-Week Results from the ApproaCH Clinical TrialPresented by Dr. Leanne Ward Tuesday, May 1315:05-15:15 CESTRoom D2 Oral PresentationEffects of Navepegritide on Growth in Children with Achondroplasia: 52-Week Results from the ApproaCH Clinical TrialPresented by Dr. Hanne Hove Growth Hormone Deficiency Tuesday, May 1314:45-14:55 CESTRoom D2 Oral PresentationResults of the foresiGH Trial Support the Efficacy and Safety of Once-Weekly Lonapegsomatropin in Adults with Growth Hormone Deficiency (GHD)Presented by Dr. Aleksandra Gilis-Januszewska More information about the events and presentations listed here is available to registered attendees of the Joint ESPE & ESE 2025 congress. 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 deformities, 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 Adult Growth Hormone DeficiencyGrowth hormone plays an essential role in the health of children and adults, promoting normal growth in children and maintenance of normal body composition and cardiometabolic health throughout adulthood. In adults, growth hormone boosts protein production, promotes fat utilization, enhances muscle mass, and helps regulate blood sugar levels. Adult GHD is a condition in which an individual's body does not produce enough growth hormone. Symptoms and morbidity can include central obesity, metabolic syndrome, decreased bone density, alterations in lipid profile and markers of cardiovascular risk, fatigue, general weakness, lack of muscle tone, and psychological symptoms such as cognitive impairment, social isolation, lack of motivation, and depression. 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 clinical benefits of Ascendis' novel therapies, (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. © May 2025 Ascendis Pharma A/ Contacts: Scott Smith Ascendis Pharma ir@ Media Contact:Melinda BakerAscendis Pharmamedia@ BankICR Healthcare+1 (415) in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
