A Phase 3, Multicenter, Single Arm, Open-Label Trial, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Japanese Adult Subjects with Hypoparathyroidism - PaTHway Japan Trial

Start Date02 Nov 2021

Sponsor / Collaborator-

NCT05387070

/ RecruitingPhase 3

PaTHway CHINA TRIAL: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Trial, With an Open-Label Extension, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Adults With Hypoparathyroidism

This study is limited to conduct in China only. The primary objective is to assess the treatment effect of daily TransCon PTH on serum calcium (sCa) levels within the normal range and stopping from therapeutic doses of active vitamin D (calcitriol) or active vitamin D analogue (alfacalcidol) and calcium at 26 weeks of treatment. All subjects will start with 18 mcg of study drug and will be individually and progressively titrated to an optimal dose over a 26-week double blind period, followed by an open label extension period up to 156 weeks. TransCon PTH or placebo will be administered as a subcutaneous injection using a pre-filled injection pen. Neither trial participants nor their doctors will know who has been assigned to each group. After the 26 weeks, participants will continue in the trial as part of a long-term extension study. During the extension, all participants will receive TransCon PTH, with the dose adjusted to their individual needs.

Start Date28 Jul 2021

Sponsor / Collaborator

VISEN Pharmaceuticals (Shanghai) Co., Ltd.

NCT04701203

/ Active, not recruitingPhase 3

PaTHway TRIAL: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Trial, With an Open-Label Extension, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Adults With Hypoparathyroidism

During the first 26 weeks of the trial, participants will be randomly assigned to one of two groups: one group will receive TransCon PTH and one group will receive placebo. All subjects will start with study drug at a dose of 18 mcg/day and will be individually and progressively titrated to an optimal dose in dose increments of 3 mcg/day. TransCon PTH or placebo will be administered as a subcutaneous injection using a pre-filled injection pen. Neither trial participants nor their doctors will know who has been assigned to each group. After the 26 weeks, participants will continue in the trial as part of a long-term extension study. During the extension, all participants will receive TransCon PTH, with the dose adjusted to their individual needs. This is a global trial that will be conducted in the United States, Canada, Germany, Denmark, Norway, Italy, and Hungary.

Start Date16 Feb 2021

Sponsor / Collaborator

Ascendis Pharma Bone Diseases A/S

100 Clinical Results associated with Palopegteriparatide

100 Translational Medicine associated with Palopegteriparatide

100 Patents (Medical) associated with Palopegteriparatide

6,988

Literatures (Medical) associated with Palopegteriparatide

01 Dec 2025·Current Pain and Headache Reports

Functional Magnetic Resonance Imaging of Post-Traumatic Headache: A Systematic Review

Review

Author: Ashina, Håkan ; Christensen, Rune H ; Szabo, Edina ; Al-Khazali, Haidar M ; Iljazi, Afrim

PURPOSE OF REVIEW:

To evaluate existing functional magnetic resonance imaging (fMRI) studies on post-traumatic headache (PTH) following traumatic brain injury (TBI).

RECENT FINDINGS:

We conducted a systematic search of PubMed and Embase databases from inception to February 1, 2024. Eligible fMRI studies were required to include adult participants diagnosed with acute or persistent PTH post-TBI in accordance with any edition of the International Classification of Headache Disorders. We identified five eligible fMRI studies: two on acute PTH and three on persistent PTH. These studies assessed resting-state functional connectivity involving comparisons with one or more of the following groups: people with migraine, those with mild TBI but no PTH, and healthy controls. In acute PTH, studies focused exclusively on functional connectivity between the periaqueductal gray or hypothalamus and other brain regions. In persistent PTH, evidence of altered functional connectivity was identified primarily within cingulate, sensorimotor, and visual regions, indicating a hypersensitivity to sensory stimuli in PTH. Despite these insights, the fMRI data remains sparse and is limited by inconsistent results and small samples. The paucity of fMRI studies on PTH limits our understanding of its neurobiological basis. The available evidence suggests that alterations in functional connectivity occur within brain areas involved in emotional and sensory discriminative aspects of pain processing. However, inconsistent results and small sample sizes underscore a critical need for larger, more rigorous fMRI studies. Future studies should also consider using task-based fMRI to investigate possible hypersensitivity to different sensory stimuli in PTH after TBI.

01 Dec 2025·Current Pain and Headache Reports

The Dual Burden of Post-Traumatic Headache: Health Consequences and Economic Impact

Review

Author: Cohen, Fred

PURPOSE OF REVIEW:

To review the history and impact and burden of post-traumatic headache (PTH).

RECENT FINDINGS:

PTH is a prevalent headache disorder that many healthcare providers encounter. Unlike more extensively researched primary headache disorders like migraines, PTH has not been as thoroughly studied, and there are fewer treatments specifically tested for it. A significant obstacle to conducting detailed population studies on PTH is the need for the headache to occur shortly after a traumatic event. Despite these challenges, PTH is recognized as a disabling condition with considerable effects on quality of life and economic impact. PTH is a distressing and debilitating condition. Although there have been efforts to evaluate its personal and economic effects, these studies are limited compared to the more extensive research conducted on other primary headache disorders. More comprehensive epidemiological studies are needed.

01 Apr 2025·BONE

Parathyroid hormone levels following denosumab vs. zoledronic acid therapy for osteoporosis

Article

Author: Barzilai-Yosef, Liat ; Rotman-Pikielny, Pnina ; Meron, Michal Kasher ; Lurie, Tzipi Hornik ; Braginski-Shapira, Sofia ; Ramaty, Erez

The objective of this retrospective, database study was to characterize the rate, magnitude and timeline of increases in parathyroid hormone (PTH) levels post-denosumab (DMAb) vs. zoledronic acid (ZA) injection in patients with osteoporosis and near normal baseline PTH. Included were osteoporotic females, ≥50 years, initiating treatment with 60 mg DMAb or 5 mg ZA. PTH levels within 6-months post-DMAb or 12-months post-ZA injection were extracted from the electronic database of a 4.5 million-member health maintenance organization. The indication for PTH measurements was unknown. Exclusion criteria were creatinine >2 mg/dL, vitamin D < 50 nmol/L or parathyroid hormone level > 1.5 × upper limit of normal (ULN). Among 3317 women, 1992 received DMAb and 1325 ZA. The DMAb group was older (73.3 ± 8.5 vs. 69.8 ± 8.6 years, p < 0.001) and more patients treated with DMAb compared with patients treated with ZA had prior non-vertebral fractures (7.7 % vs. 5.2 %, p < 0.01) and had previously been treated with osteoporosis medication (56.3 % vs. 50.3 %, p < 0.001). Among the patients, 14.9 % had at least one post-treatment PTH > 1.5 ULN. Of 7273 post-treatment PTH tests, 62.6 % were within normal limits, while 24.8 % were mildly elevated at 1.01-1.5 ULN. Two-months after both treatments, >1.5 ULN PTH levels peaked at ∼20 %. Elevated PTH was associated with eGFR < 60 mL/min/1.73 m² and comorbidities. In conclusion, most PTH levels post-DMAb or ZA in osteoporotic patients with baseline PTH < 1.5 ULN, were within normal range. PTH increased to >1.5 ULN in 14.9 % of patients; peaking in the first 2-months post-treatment and declining thereafter. Elevated PTH may be related to anti-resorptive effects and is not medication specific. PTH measurements in the first few months post-DMAb and ZA therapy should be limited.

111

News (Medical) associated with Palopegteriparatide

20 Feb 2025

·www.prnewswire.com

FIRST AND ONLY TREATMENT FOR CHRONIC HYPOPARATHYROIDISM APPROVED IN AUSTRALIA

YORVIPATH® (palopegteriparatide) is now registered by the Therapeutic Goods Administration (TGA) for the treatment of chronic hypoparathyroidism in adults1 First-in-class novel PTH replacement therapy Australia becomes the first country to obtain Marketing Authorisation for YORVIPATH since FDA approval SINGAPORE, Feb. 20, 2025 /PRNewswire/ -- Independent biopharmaceutical company Specialised Therapeutics (ST) welcomes the registration of YORVIPATH® (palopegteriparatide) by the Therapeutic Goods Administration (TGA), "for the treatment of chronic hypoparathyroidism in adults".1,2 YORVIPATH was granted an Orphan Drug Designation and assessed through the TGA's Priority Review pathway.3,4 It is the first and only medicine to be listed on the Australian Register of Therapeutic Goods (ARTG) for the treatment of chronic hypoparathyroidism. Hypoparathyroidism is a rare, complex endocrine disease, affecting an estimated 6.4-37 per 100,000 people globally.5,6 It is an endocrine disorder in which the production of parathyroid hormone (PTH) by the parathyroid glands is abnormally low or absent, causing low levels of calcium (hypocalcaemia) and high levels of phosphorous (hyperphosphataemia) in the blood.7,8 This impacts proper functioning of nerves and muscles, leading to weakness, muscle spasms or cramps, headaches, hair loss, numbness or tingling, and memory problems.8,9 Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extraskeletal calcifications, and cognitive impairment.10 Leading Australian Endocrinologist and Head of the School of Clinical Sciences at Monash Health, Professor Peter Ebeling AO, applauded the TGA approval of YORVIPATH in addressing an unmet medical need for Australian adults diagnosed with chronic hypoparathyroidism. "Until now, the only treatment option for adults with chronic hypoparathyroidism has been conventional therapy with calcium and active vitamin D supplementation to keep blood calcium levels normal," said Professor Ebeling. "While these treatments help to manage the symptoms of hypoparathyroidism, they do not address the underlying deficiency of PTH and contribute to the significant pill burden for patients." "The lack of effective therapeutic options has been an urgent medical need in this area, and the local approval of YORVIPATH represents an important advance in the treatment of chronic hypoparathyroidism in Australia," said Professor Ebeling. YORVIPATH is a first-in-class PTH replacement therapy. A prodrug of parathyroid hormone (PTH [1-34]), YORVIPATH is administered subcutaneously once daily, with sustained release of active PTH designed to provide PTH levels in the physiological range for 24 hours/day.11 YORVIPATH's approval in Australia was supported by the results of Ascendis Pharma's Phase 3 PaTHway trial, a 26-week randomised, double-blind, placebo-controlled study with a 156-week open-label extension, published in the Journal of Bone and Mineral Research.12 Involving 84 adults with chronic hypoparathyroidism, the clinical trial demonstrated the efficacy, safety and tolerability of YORVIPATH as a once-daily PTH replacement therapy. Data from the pivotal trial showed 93% (57/61) of YORVIPATH-treated patients achieved independence from both active vitamin D and therapeutic doses of elemental calcium, which was associated with a clinically meaningful reduction in their daily pill burden.12 The safety analysis demonstrated YORVIPATH was generally well-tolerated, with the most frequently reported adverse reactions being injection site reactions (21.6%), headache (18.7%), and paraesthesia (13.7%).2 In addition to the significant short-term and long-term medical complications of chronic hypoparathyroidism, individuals diagnosed with the condition also experience reduced health-related quality of life, including the negative impact of symptoms on their daily life and work productivity.12 Chief Executive Officer of the Australian Thyroid Foundation (ATF), Beverley Garside, acknowledged the significance of the TGA approval in offering adults living with chronic hypoparathyroidism the opportunity to improve their health and wellbeing. However, she reinforced that this is not the time for complacency, as it is just the first step towards securing subsidised access to an important new treatment option for this community. "As the leading national voice for good thyroid health in Australia, the ATF recognises the importance of the TGA registration of YORVIPATH as a new treatment option for the hypoparathyroid community. While this is positive news, it is only the first step," said Ms Garside. "For adults diagnosed with chronic hypoparathyroidism to truly benefit from this new treatment, it is essential they have the option of subsidised and affordable access to YORVIPATH through the Pharmaceutical Benefits Scheme as quickly as possible." YORVIPATH is being made available in Australia by Specialised Therapeutics (ST), under an exclusive distribution agreement with global biopharmaceutical company Ascendis Pharma A/S that covers Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam. The Australian registration of YORVIPATH follows approvals issued to Ascendis Pharma by the United States Food and Drug Administration (US FDA)13 in August 2024 and the European Medicines Agency (EMA)14 in November 2023. "The Australian approval of YORVIPATH reflects our values and dedication to applying science to help address the significant unmet medical needs expressed by the hypoparathyroidism community," said Roy Khoury, Vice President, Head of International Markets at Ascendis Pharma. "We are pleased to partner with Specialised Therapeutics to broaden access to this innovative therapy, based on our TransCon technology, which offers a new approach to the treatment of this often-debilitating rare disease." ST Chief Executive Officer, Mr Carlo Montagner, said the approval of YORVIPATH was a meaningful milestone, but the company would not stop until it achieved reimbursement for eligible Australians with hypoparathyroidism through the Pharmaceutical Benefits Scheme (PBS). "We are proud to have brought the first and only TGA-registered treatment for chronic hypoparathyroidism to Australians living with the condition so rapidly following the recent FDA and EMA approvals," said Mr Montagner. "Chronic hypoparathyroidism has considerable and life-long wide-ranging impacts on these individuals and we are committed to working with the local hypoparathyroidism community and the Australian Government to ensure YORVIPATH is accessible to eligible patients through the PBS at the earliest opportunity." YORVIPATH will be reviewed by the Pharmaceutical Benefits Advisory Committee (PBAC) at its first meeting of the year, to be held from 12-14 March 2025. YORVIPATH will be made available for prescribing in Australia once it has been listed on the PBS. About Specialised Therapeutics Founded in 2007, Specialised Therapeutics is the region's largest independent specialty pharmaceutical company, providing new therapies and technologies to patients in Australia, New Zealand and across Southeast Asia. Headquartered in Singapore, ST partners with global pharmaceutical, biotech and diagnostic companies to bring novel healthcare opportunities to patients who are impacted by a range of diseases. ST has built a strong track record of success, navigating complex regulatory, reimbursement and commercialisation environments in its diverse regions. The ST mission is to provide specialty therapies where there is an unmet need. The company's broad therapeutic portfolio currently includes novel agents in oncology, haematology, CNS, neurology, endocrinology, ophthalmology and supportive care, although it is not confined to these areas. ST is a member of the World Orphan Drug Alliance (WODA). Additional information can be found at About Ascendis Pharma Ascendis Pharma is applying its innovative TransCon technology platform 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 uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit: ascendispharma.com to learn more. About Hypoparathyroidism10 Hypoparathyroidism 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 kidneys and indirectly on the intestines. 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 PaTHway12 The PaTHway trial was a double-blind, placebo-controlled, 26-week, Phase 3 study that assessed the efficacy and safety of PTH replacement therapy with palopegteriparatide for individuals with hypoparathyroidism. Participants (n=84) were randomised 3:1 to once-daily palopegteriparatide (initially 18 μg/d) or placebo, both co-administered with conventional therapy. The study drug and conventional therapy were titrated according to a dosing algorithm guided by serum calcium. The composite primary efficacy endpoint was the proportion of participants at week 26 who achieved normal albumin-adjusted serum calcium levels (8.3–10.6 mg/dL), independence from conventional therapy (requiring no active vitamin D and ≤600 mg/d of calcium), and no increase in study drug over 4 weeks before week 26. At week 26, 79% (48/61) of participants treated with palopegteriparatide versus 5% (1/21) with placebo met the composite primary efficacy endpoint (p < 0.0001). Additionally, 93% (57/61) of participants treated with palopegteriparatide achieved independence from conventional therapy. Palopegteriparatide treatment normalised mean 24-hour urine calcium. Overall, 82% (50/61) treated with palopegteriparatide and 100% (21/21) with placebo experienced adverse events; most were mild (46%) or moderate (46%). No study drug-related withdrawals occurred. About YORVIPATH® (palopegteriparatide) YORVIPATH® (palopegteriparatide, developed as TransCon™ PTH) is a once-daily prodrug providing sustained release of active PTH. It was approved by the European Union as a parathyroid hormone (PTH) replacement therapy for the treatment of adults with chronic hypoparathyroidism15 and the United States for the treatment of hypoparathyroidism in adults13. Treatment should be initiated and monitored by physicians or qualified healthcare professionals experienced in the diagnosis and management of patients with hypoparathyroidism. Yorvipath® is a registered trademark of Ascendis Pharma A/S (NASDAQ: ASND). References: Therapeutic Goods Administration. Australian Register of Therapeutic Goods (ARTG). Available at: . [Accessed Dec 2024]. YORVIPATH (palopegteriparatide) AU Product Information. Therapeutic Goods Administration. Prescription medicine determinations and designations: Notice for palopegteriparatide (Specialised Therapeutics Pharma Pty Ltd) – Orphan drug designation. Available at: . [Accessed Dec 2024]. Therapeutic Goods Administration. Prescription medicine determinations and designations: Notice for palopegteriparatide (Specialised Therapeutics Pharma Pty Ltd) – Priority review. Available at: . [Accessed Dec 2024]. Siggelkow H, Pacaud D, et al. Clin Endocrinol (Oxf). 2020;92(2). Bjornsdottir S, Sikjaer T, et al. J Bone Miner Res. 2022;37(12). Australian Thyroid Foundation. Thyroid Facts: Parathyroid Glands – Hypoparathyroidism. Available at: [Accessed Dec 2024]. National Organization for Rare Disorders. Disease Overview: Hypoparathyroidism - Symptoms, Causes, Treatment, 14 August 2024. Available at: [Accessed Dec 2024]. Endocrine Society. Endocrine Library: Patient Resources; Hypoparathyroidism, 24 January 2022. Available at: [Accessed Dec 2024]. Bilezikian JP. J Clin Endocrinol Metab. 2020;105(6):1722-1736. doi:10.1210/clinem/dgaa113. YORVIPATH (palopegteriparatide) US Prescribing Information. Khan AA, Bilezikian JP, Cusano NE, et al. J Bone Miner Res. 2023;38(1). US Food and Drug Administration. FDA approves new drug for hypoparathyroidism, a rare disorder, 9 August 2024. Available at: [Accessed Dec 2024]. European Medicines Agency. Yorvipath: Palopegteriparatide, 17 November 2023. Available at: [Accessed Dec 2024]. YORVIPATH (palopegteriparatide).EU Summary of Product Characteristics. SOURCE Specialised Therapeutics Asia WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultDrug ApprovalPhase 3Orphan Drug

16 Jan 2025

·synapse.patsnap.com

2024 Global New Drug Approvals: A Comprehensive Review of Breakthrough Therapies and Innovations

In 2024, the range of diseases treated by globally approved new drugs was diverse, with cancer and rare diseases leading in the total number of new drug approvals. Several new drugs were also approved in the fields of anti-infectives and cardiovascular diseases. Among the approved small molecule drugs, 8 were designated as "first-in-class" therapies with breakthrough therapy status. These drugs not only possess unique mechanisms of action but also provide significant therapeutic improvements over previous treatments. As the development technologies for peptide and nucleic acid-based drugs have matured, their proportion among FDA-approved new drugs has stabilized. In 2024, the approved nucleic acid-based drugs included the first telomerase inhibitor, Rytelo (imetelstat). This drug targets and binds to the RNA template of telomerase, inhibiting its activity. It received FDA approval in June 2024 for the treatment of adult patients with myelodysplastic syndromes (MDS). A major challenge for peptide-based drugs is their susceptibility to degradation in the human body, making it difficult to maintain their efficacy. However, advancements in peptide modification technologies have led to longer half-lives for these drugs, offering patients long-acting therapies with reduced treatment burdens. Yorvipath, a long-acting parathyroid hormone prodrug approved in 2024, links the parathyroid hormone prodrug to a polyethylene glycol carrier to control the hormone's release in the body. It received FDA approval in August 2024 as the first treatment for adult hypoparathyroidism. In 2024, CBER approved 8 cell and gene therapies. These therapies not only represent technological innovations but also provide the first approved treatments for patients with various types of diseases. Amtagvi (lifileucel): On February 16, 2024, the U.S. FDA granted accelerated approval to Iovance Biotherapeutics' tumor-infiltrating lymphocyte (TIL) therapy, Amtagvi (lifileucel), for the treatment of advanced melanoma. Amtagvi is the first approved TIL therapy and the first T-cell therapy approved for solid tumors, marking another milestone in cell therapy. Lenmeldy (atidarsagene autotemcel): On March 18, 2024, the FDA approved Lenmeldy (atidarsagene autotemcel, arsa-cel), a gene therapy developed by Orchard Therapeutics, for the treatment of pediatric patients with metachromatic leukodystrophy (MLD) who meet specific criteria. This is the first FDA-approved gene therapy for this rare genetic disorder. Tecelra (afamitresgene autoleucel): On August 1, 2024, the FDA approved Adaptimmune Therapeutics' engineered T-cell therapy, Tecelra (afami-cel), for the treatment of adult patients with advanced MAGE-A4+ synovial sarcoma of certain HLA types who have previously received chemotherapy. Tecelra is the first engineered T-cell therapy approved for solid tumors and the first effective treatment for synovial sarcoma in over a decade. Kebilidi (eladocagene exuparvovec): On November 13, 2024, the FDA granted accelerated approval to Kebilidi (eladocagene exuparvovec), a gene therapy developed by PTC Therapeutics, for the treatment of pediatric and adult patients with aromatic L-amino acid decarboxylase deficiency (AADCD), regardless of disease severity. This is the first FDA-approved gene therapy administered via direct injection into the brain. Ryoncil (remestemcel): On December 18, 2024, the FDA approved Ryoncil (remestemcel), developed by Mesoblast, for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients aged 2 months and older. The FDA's press release highlighted that this is the first FDA-approved mesenchymal stromal cell (MSC) therapy. In addition, the FDA approved Moderna's respiratory syncytial virus (RSV) vaccine, Mresvia, marking the first approval of an mRNA vaccine for a disease other than COVID-19, demonstrating the potential of mRNA technology. Top 10 Potential Blockbuster Therapies of 20241. Rezdiffra (resmetirom): The First FDA-Approved Therapy for Metabolic Dysfunction-Associated Steatohepatitis (MASH) Rezdiffra (resmetirom), developed by Madrigal Pharmaceuticals, is a "first-in-class," once-daily, oral thyroid hormone receptor (THR)-β selective agonist designed to target the underlying causes of MASH. Thyroid hormone plays a central role in liver function by activating β receptors in hepatocytes, influencing a range of health parameters, from serum cholesterol and triglyceride levels to the pathological accumulation of fat in the liver. Rezdiffra received FDA approval in March 2024, becoming the first approved therapy for MASH and marking a significant milestone in this field. 2. Winrevair (sotatercept): A "First-in-Class" Therapy for Pulmonary Arterial Hypertension Winrevair is a "first-in-class" activin receptor type IIA (ActRIIA) fusion protein. It combines a modified extracellular domain of ActRIIA with the Fc region of an antibody. This therapy blocks the binding of activin to its cell membrane receptors, thereby reducing activin-mediated signaling. Merck acquired this innovative therapy through its approximately $11.5 billion acquisition of Acceleron Pharma in 2021. Winrevair received FDA approval in March 2024 for the treatment of pulmonary arterial hypertension. Merck believes it has the potential to become a cornerstone therapy for this condition. 3. Anktiva: A "First-in-Class" IL-15 Receptor Agonist for Non-Muscle Invasive Bladder Cancer (NMIBC) Anktiva, developed by ImmunityBio's Altor BioScience, consists of an IL-15 mutant (IL-15N72D) bound to an IL-15 receptor α/IgG1 Fc fusion protein. Compared to natural IL-15, Anktiva exhibits superior pharmacokinetic properties in patients, persists longer in lymphoid tissues, and demonstrates enhanced anti-tumor activity. In April 2024, Anktiva received FDA approval for use in combination with Bacillus Calmette-Guérin (BCG) to treat adult patients with BCG-unresponsive NMIBC accompanied by carcinoma in situ (CIS). In clinical trials, patients treated with Anktiva achieved a complete response rate of 62%. 4. mResvia: The First mRNA Respiratory Syncytial Virus (RSV) Vaccine Moderna's mRNA-based RSV vaccine, mResvia (mRNA-1345), received FDA approval in May 2024 for protecting adults aged 60 and older from lower respiratory tract disease (RSV-LRTD) caused by RSV infection. This is Moderna's second FDA-approved mRNA vaccine. It consists of an mRNA sequence encoding a stabilized prefusion F glycoprotein, delivered using the same lipid nanoparticle (LNP) technology as Moderna's COVID-19 vaccine. 5. Rytelo (imetelstat): The First Telomerase Inhibitor In June 2024, the U.S. FDA approved Geron's "first-in-class" telomerase inhibitor, Rytelo (imetelstat), for the treatment of adult patients with low- to intermediate-1-risk myelodysplastic syndromes (MDS). This approval marked the arrival of the first telomerase-targeted therapy, nearly 40 years after the discovery of telomerase in 1984. Telomeres are protective caps at the ends of chromosomes that naturally shorten with each cell division. In MDS, abnormal bone marrow cells often express telomerase, which rebuilds telomeres, leading to uncontrolled cell division. Preclinical and clinical data show that Rytelo inhibits telomerase activity, curbing the uncontrolled proliferation of cancerous stem and progenitor cells and inducing apoptosis in malignant cells, demonstrating disease-modifying activity. 6. Ohtuvayre (ensifentrine): The First Inhaled Therapy with a Novel Mechanism of Action for COPD Maintenance Treatment in Over 20 Years Ohtuvayre, developed by Verona Pharma, is a "first-in-class" dual phosphodiesterase 3/4 (PDE3/4) inhibitor. It received FDA approval in June 2024 as a maintenance treatment for adult patients with chronic obstructive pulmonary disease (COPD). Ohtuvayre is the first inhaled COPD therapy to combine bronchodilator and non-steroidal anti-inflammatory effects. Additionally, it represents the first inhaled therapy with a novel mechanism of action for COPD maintenance treatment in over two decades. Ohtuvayre works by inhibiting PDE3, leading to relaxation of airway smooth muscles and bronchodilation, while its inhibition of PDE4 reduces inflammation by decreasing the release of pro-inflammatory cytokines. 7. Kisunla (donanemab): The First Alzheimer’s Disease (AD) Therapy Allowing Treatment Discontinuation Upon Meeting Criteria Kisunla, developed by Eli Lilly, is an anti-amyloid monoclonal antibody that specifically binds to the N3pG subtype of amyloid. By targeting this subtype, Kisunla selectively binds to amyloid plaques in the brain, facilitating their clearance. In July 2024, it received FDA approval for the treatment of adult patients with early symptomatic AD, including those with mild cognitive impairment (MCI) or mild dementia stage AD, confirmed by the presence of amyloid pathology. According to the press release, Kisunla, administered monthly, is the first amyloid plaque-targeting therapy with evidence supporting treatment discontinuation after amyloid plaque clearance, potentially reducing treatment costs and the frequency of infusions. 8. Ebglyss (lebrikizumab): A New First-Line Biologic Therapy for Atopic Dermatitis Ebglyss, co-developed by Almirall and Eli Lilly, is an anti-IL-13 antibody. It received FDA approval in September 2024 for the treatment of moderate-to-severe atopic dermatitis (AD) in adults and adolescents (aged 12 years and older, weighing at least 40 kg) whose disease remains uncontrolled despite topical treatments. IL-13 is a key cytokine in AD, driving type 2 inflammation in the skin, which leads to impaired skin barrier function, itching, skin thickening, and infections. Ebglyss's targeted mechanism of action, along with its demonstrated efficacy and safety in both short- and long-term use, makes it a compelling option for patients with moderate-to-severe AD inadequately controlled by topical therapies. Its once-monthly maintenance dosing offers convenience for patients. 9. Cobenfy (xanomeline, trospium chloride): The First Novel Mechanism Drug for Schizophrenia in Decades Cobenfy received FDA approval in September 2024 for the treatment of adult patients with schizophrenia. The FDA press release highlighted that Cobenfy is the first antipsychotic drug targeting cholinergic receptors, diverging from the long-standing standard therapies that target dopamine receptors. This approval marks the first novel mechanism drug for schizophrenia in decades. Cobenfy combines two active ingredients, xanomeline and trospium chloride, designed to activate muscarinic acetylcholine receptors in the brain while minimizing peripheral effects. By stimulating M1 and M4 muscarinic receptors, xanomeline alleviates negative symptoms such as apathy and reduced social drive, improves cognitive function, and helps manage other psychiatric symptoms like hallucinations and delusions. Originally developed by Karuna Therapeutics, Cobenfy was acquired by Bristol Myers Squibb in December 2023 through a merger agreement. 10. Attruby (acoramidis): The First Approved Product with Labeling Indicating Near-Complete Stabilization of Transthyretin (TTR) In November 2024, the FDA approved Attruby (acoramidis), developed by BridgeBio Pharma, for the treatment of adults with transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) to reduce cardiovascular mortality and cardiovascular-related hospitalizations. The press release noted that Attruby is the first approved product with labeling indicating near-complete stabilization of TTR. The drug is designed to mimic the protective function of the TTR T119M mutation, maintaining the normal tetrameric conformation of TTR and preventing the formation of toxic amyloid deposits. In a Phase 3 clinical trial, Attruby reduced all-cause mortality and recurrent cardiovascular-related hospitalizations by 42% compared to placebo at 30 months of treatment. How to obtain the latest research advancements in the field of biopharmaceuticals? In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!

12 Jan 2025

·www.globenewswire.com

Ascendis Pharma Provides Business and Strategic Roadmap Update at 43rd Annual J.P. Morgan Healthcare Conference

- Ascendis positioned to drive rapid revenue growth COPENHAGEN, Denmark, Jan. 12, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today provided a business and strategic roadmap update, including planned 2025 key corporate milestones. Ascendis President and CEO Jan Mikkelsen will present this update tomorrow, January 13, during the 43rd Annual J.P. Morgan Healthcare Conference. “Ascendis is well-positioned for rapid revenue growth with the launch of our first two Endocrinology Rare Disease medicines, SKYTROFA and YORVIPATH, and, following our pre-NDA meeting, planned filings for our third, TransCon CNP for achondroplasia,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Our expanding TransCon technology platform and partnerships demonstrate that our business model built on fast, successful drug development can create substantial stakeholder value.” Selected Key Updates and Milestones TransCon™ hGH (lonapegsomatropin, marketed as SKYTROFA®) SKYTROFA full-year 2024 revenue, with a single indication in pediatric GHD, expected to be ~€202 million excluding sales deductions related to prior years (based on unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of ~€197 million plus ~€5 million of sales deductions related to prior years).U.S. SKYTROFA volume (mg) increased 84% in 2024 year-over-year resulting in an estimated 6.5% market share of the total U.S. growth hormone market for 2024 (based on third party prescription data).Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025 for FDA review of supplemental BLA for the treatment of adults with growth hormone deficiency; pending approval, U.S. commercial launch planned in the fourth quarter of 2025.During the third quarter of 2025, plan to submit an Investigational New Drug (IND) application or similar for a basket trial evaluating TransCon hGH in additional indications. TransCon PTH (palopegteriparatide, marketed as YORVIPATH®) YORVIPATH full-year 2024 unaudited preliminary revenue estimate of ~€29 million.YORVIPATH commercially available for prescription in Germany and Austria since January 2024. Outside Germany and Austria, providing product through early access routes, such as ‘named patient,’ until commercial reimbursement established. ~700 patients on treatment in our Europe Direct and International Markets at the end of 2024.YORVIPATH commercially available for prescription since late December 2024 in the U.S. As of January 9, 2025, 324 patients enrolled into the Ascendis Signature Access Program or direct with specialty pharmacy, with over half of prescriptions for patients new to YORVIPATH.Expect commercial launch in at least five additional Europe Direct countries in 2025. TransCon CNP (navepegritide) Following pre-NDA meeting with FDA, plan to submit New Drug Application (NDA) for the treatment of children with achondroplasia during the first quarter of 2025, and submit Marketing Authorisation Application to the European Medicines Agency during the third quarter of 2025.Presented new data demonstrating significant improvements in leg bowing, a common complication in achondroplasia, observed with TransCon CNP compared to worsening observed with placebo in pivotal ApproaCH Trial.Topline Week 26 results from Phase 2 COACH Trial (TransCon CNP in combination with TransCon hGH) expected in the second quarter of 2025.During the fourth quarter of 2025, plan to submit an IND or similar for the treatment of hypochondroplasia. Expanding the TransCon Platform & Pipeline New TransCon protein degrader platform designed to enable efficient clearance of hormones, cytokines, and other targets. First planned TransCon protein degrader product candidate designed to normalize excess FGF-23 hormone levels for patients with X-linked hypophosphatemia. Financial Update Unaudited preliminary estimate of total full-year 2024 product revenue of ~€226 million: SKYTROFA full-year 2024 revenue expected to be ~€202 million excluding sales deductions related to prior years (based on unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of ~€197 million plus ~€5 million of sales deductions related to prior years).YORVIPATH full-year 2024 unaudited preliminary revenue estimate of ~€29 million. Unaudited preliminary estimate of total full-year total 2024 revenue of ~€364 million Includes $100 million Novo Nordisk milestone payment as non-product revenue. December 31, 2024 pro forma cash balance of ~€655 million (based on unaudited preliminary estimate of December 31, 2024 cash balance of €560 million plus expected payment from Novo Nordisk of $100 million). Presentation at J.P. Morgan Healthcare Conference on Monday, January 13A live webcast of the event will be available via the Investors & News section of the Ascendis Pharma website at https://investors.ascendispharma.com. The presentation will begin at 11:15 a.m. Eastern Time / 8:15 a.m. Pacific Time. A webcast replay will be available for 30 days. The Company’s slides from the J.P. Morgan presentation will be available on the same Investor Relations website at https://investors.ascendispharma.com. About Ascendis Pharma A/SAscendis Pharma is applying its innovative TransCon technology platform 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, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more. Forward-Looking StatementsThis 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’ potential for rapid revenue growth, (ii) planned regulatory submissions for TransCon CNP and TransCon hGH, (iii) the ability of Ascendis’ business model to create substantial stakeholder value, (iv) expected full-year 2024 SKYTROFA, YORVIPATH and total revenues, (v) the PDUFA date of the supplemental BLA for SKYTROFA for the treatment of adults with growth hormone deficiency and Ascendis’ plans for a U.S. commercial launch, if approved, (vi) Ascendis’ expectations regarding the commercial launch of YORVIPATH in additional Europe Direct Countries, (vii) the timing of topline results from the Phase 2 COACH Trial of TransCon CNP, (viii) Ascendis’ expectations regarding its TransCon protein degrader platform and first TransCon degrader product candidate, (ix) the expected payment from Novo Nordisk, (x) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (xi) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. 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. 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’ prospectus supplement filed on September 20, 2024 and Ascendis’ current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including its Annual Report on Form 20-F filed with the SEC on February 7, 2024. 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, TransCon, SKYTROFA®, and YORVIPATH® are trademarks owned by the Ascendis Pharma group. © January 2025 Ascendis Pharma A/S. Investor Contacts:Media Contact:Scott SmithMelinda BakerAscendis PharmaAscendis Pharmair@ascendispharma.com +1 (650) 709-8875 media@ascendispharma.comPatti Bank ICR Healthcare +1 (415) 513-1284 patti.bank@icrhealthcare.com

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100 Deals associated with Palopegteriparatide

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Indication	Country/Location	Organization	Date
Hypoparathyroidism	EU	Ascendis Pharma Bone Diseases A/S	17 Nov 2023
Hypoparathyroidism	IS	Ascendis Pharma Bone Diseases A/S	17 Nov 2023
Hypoparathyroidism	LI	Ascendis Pharma Bone Diseases A/S	17 Nov 2023
Hypoparathyroidism	NO	Ascendis Pharma Bone Diseases A/S	17 Nov 2023

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04701203 (PaTHway, CTgov)	Phase 3	Hypoparathyroidism	84	TransCon PTH (TransCon PTH)	ihrxvwmdna(iktergnxfb) = tmrsohdkkx zruzlwrxvp (rfuvvbfyho, vviuedhssf - tcrqektbvz) View more	-	28 Feb 2025
				Placebo (Placebo)	ihrxvwmdna(iktergnxfb) = qdsshpmzda zruzlwrxvp (rfuvvbfyho, yszwwodzad - mgweenkdlv) View more
NCT05387070 (PaTHway, PRNewswire) Manual	Phase 3	Hypoparathyroidism	-	Palopegteriparatide	shqwmtchnr(vebadzvksl) = tgfnvyhgby drcozidtgd (girnkpruis ) View more	Positive	12 Aug 2024
				Placebo	shqwmtchnr(vebadzvksl) = rswwxdqtqd drcozidtgd (girnkpruis )
NCT04701203 (FDA_CDER) Manual	Phase 3	Hypoparathyroidism	82	YORVIPATH	ldgwlpwukb(zegfxnjasf) = vnjlwpfrhd nmtvnbpkhf (kukernybbu ) View more	Positive	09 Aug 2024
				Placebo	ldgwlpwukb(zegfxnjasf) = ulthtdslaz nmtvnbpkhf (kukernybbu ) View more
NCT04701203 (PaTHway, GlobeNewswire) Manual	Phase 3	Hypoparathyroidism	82	TransCon PTH (palopegteriparatide)	mayeczvxan(zogqbpecqb) = sdqntfseck ffcjpallft (ctanonyxgl ) View more	Positive	13 May 2024
				TransCon PTH (palopegteriparatide) (eGFR < 60 mL/min/1.73m2)	mayeczvxan(zogqbpecqb) = rvqjrpnxas ffcjpallft (ctanonyxgl ) View more
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2023)	Phase 3	Hypoparathyroidism	82	TransCon PTH	exkkcgqwnp(wopdndstns) = trended toward norms from baseline to Weeks 26 and 52 zejpdecfti (vudmtragjt ) View more	Positive	05 Oct 2023
NCT04009291 (PaTHway \| PaTH Forward, CTgov)	Phase 2	Hypoparathyroidism	59	TransCon PTH (TransCon PTH 15 mcg)	pxawxynhol(zetscjwcog) = evcgzpskss oeplzcaljt (taqclsiptq, huohayqrzc - gdzuanugwz) View more	-	01 Sep 2023
				TransCon PTH (TransCon PTH 18 mcg)	pxawxynhol(zetscjwcog) = gxuyotwuwa oeplzcaljt (taqclsiptq, xwmgbcmdta - mzkfwladye) View more
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2022)	Phase 3	Hypoparathyroidism	82	TransCon PTH 18 mcg/d	icfolqsdwz(welbtkpeqo) = The most commonly reported study drug-related AEs were headaches gapnacyxmv (fpsxkztmcr ) View more	Positive	01 Nov 2022
				Placebo
PaTH Forward Trial (ENDO2022)	Phase 2	Hypoparathyroidism	59	TransCon PTH 15 mcg/d	ifdvrrwgpo(iffmxafckh) = ssgzaazagv xonybobsus (atlmpuazrm ) View more	Positive	01 Nov 2022
				TransCon PTH 18 mcg/d	ifdvrrwgpo(iffmxafckh) = jntpqcdmfn xonybobsus (atlmpuazrm ) View more
NCT04009291 (PaTH Forward, Corporate Publications) Manual	Phase 2	-	57	TransCon™ PTH	depkslfnkr(vvunijugbe) = hapxbuflcv flgoniaxkh (hcebqolqtg ) View more	Positive	12 Sep 2022
WCO_IOF_ESCEO2022	Not Applicable	Acromegaly PTH \| serum vitamin D metabolites	-	PTH (Acromegaly group)	asqnsrerav(sxxedhtlud) = lower baseline levels nrxajtqcpo (yzsytcqdaz ) View more	-	24 Mar 2022
				PTH (Control group)

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