Latest news with #AscendisPharma
Business Insider
2 days ago
- 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
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.
Yahoo
17-06-2025
- Business
- Yahoo
Ascendis Pharma (ASND) Rated as a Hold at Cannacord Genuity on Growth Hormone Prospects
Ascendis Pharma A/S (NASDAQ:ASND) is among the 12 best healthcare stocks to buy now. On June 10, Canaccord Genuity reiterated a Hold rating on the stock and an $84 price target. The stance follows the release of new clinical trial data. A close-up of a researcher in a biopharmaceutical lab, carefully studying a sample. The pharmaceutical company delivered solid 26-week efficacy results from its TransCon HGH+TransCon CNP COACH trial. Nevertheless, the research firm raised concerns over potential long-term side effects of the human Growth Hormone under investigation. The remarks reflect a cautious optimism while considering the broader competitive landscape and long-term implications. Consequently, while the candidate treatment offers an additional growth acceleration option, it might not be a permanent replacement for CNP immunotherapy in achondroplasia patients. On its part, Ascendis Pharma insists that the combination treatment presents an alternative therapeutic choice that patients and caregivers can rely on. The US Food and Drug Administration has accepted the company's New Drug Application for TransCon. The approval follows the treatment's significant benefits in annualized growth velocity and health improvements in Children. Ascendis Pharma A/S (NASDAQ:ASND) is a biopharma company focused on developing innovative treatments for unmet medical needs, especially in the fields of endocrinology and oncology. The company utilizes its proprietary TransCon technology platform to design therapies that aim to deliver improved outcomes and set new standards in care. 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 NEXT: 10 Stocks Analysts Are Upgrading Today and 13 Best AI Stocks to Buy Under $10. Disclosure: None. 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
16-06-2025
- Business
- Yahoo
Dr. Jethro Ekuta Joins BP Logix Life Sciences Strategic Advisory Board
Global regulatory leader brings decades of experience to guide the next generation of medical affairs automation. SAN DIEGO, June 16, 2025--(BUSINESS WIRE)--BP Logix, a leading provider of AI-enabled medical affairs automation technology, is proud to announce the appointment of Dr. Jethro Ekuta to its Life Sciences Strategic Advisory Board. Dr. Ekuta is the Chair of the Board of Directors for the Regulatory Affairs Professionals Society (RAPS) and currently serves as Chief Regulatory & Safety Officer at Ascendis Pharma. With over 30 years of experience in regulatory affairs and pharmacovigilance, Dr. Ekuta brings a deep understanding of global compliance, regulatory strategy and innovation in drug development. He has held senior leadership roles at organizations including Ascendis Pharma, Alexion (AstraZeneca Rare Disease), Horizon Therapeutics (Amgen), Johnson & Johnson, Genzyme (Sanofi), Bristol-Myers Squibb, Pfizer and the U.S. Food and Drug Administration (FDA). "We are honored to welcome Dr. Ekuta to our Life Sciences Strategic Advisory Board," said Girish Pashilkar, CEO of BP Logix. "His regulatory insight and leadership will be instrumental as we continue to expand our footprint in the life sciences sector and support teams navigating increasingly complex review and approval processes." "I've spent my career working to improve regulatory systems and processes within the pharmaceutical and biotech industries," said Dr. Ekuta. "I believe BP Logix is uniquely positioned to help life sciences organizations streamline compliance, reduce risk and move faster without compromising integrity. I'm excited to contribute to the company's mission and support its growth in this critical space." In his advisory role, Dr. Ekuta will provide strategic input across marketing, product, and customer engagement initiatives. His appointment is part of a broader effort by BP Logix to deepen its alignment with industry leaders and further tailor its solutions to the evolving needs of life sciences organizations. About BP Logix BP Logix helps medical affairs and regulatory teams at life sciences organizations streamline complex review and approval workflows. Its flagship solution, PubPro, simplifies the publication process, while the broader Approvia platform supports use cases such as MLR reviews, clearance systems, medical information requests and IIS evaluations. With a focus on configurability, compliance and speed, BP Logix empowers teams to reduce risk, improve collaboration and bring high-quality treatments to market faster. To learn more about BP Logix and its work in life sciences, visit View source version on Contacts Media contact: Catie Leary,
