A Phase 3, Multicenter, Randomized, Open-Label Trial Investigating the Safety, Tolerability and Efficacy of Palopegteriparatide Administered Subcutaneously Daily at Doses Greater Than 30 μg/Day in Adult Participants With Hypoparathyroidism

This trial has a treatment duration of 78 weeks and will include adult participants already on treatment with palopegteriparatide at doses at or greater than 30 mcg/day. All participants will receive subcutaneous palopegteriparatide during the trial and will be individually and progressively titrated to an optimal dose at pre-specified dose levels. The primary purpose of the trial is to provide additional evidence of treatment effect and safety of palopegteriparatide at doses greater than 30 mcg/day in adults with hypoparathyroidism. The trial will be conducted in the US.

开始日期2026-05-07

申办/合作机构

Ascendis Pharma Bone Diseases A/S

NCT07706764

/ Recruiting临床3期

A Phase 3, Multicenter, Open-Label Single-Arm Clinical Trial to Assess the Safety, Tolerability, Pharmacokinetics, and Efficacy of Palopegteriparatide Administered Subcutaneously Daily in the Adolescent Population (12 Years to Less Than 18 Years of Age) With Chronic Hypoparathyroidism

This trial will enroll adolescents between ages of ≥12 and <18 years with clinically diagnosed hypoparathyroidism . The purpose of the study is to see how well treatment with once-daily palopegteriparatide works and how safe it is. At least 12 participants will receive palopegteriparatide for 234 weeks. This trial will be conducted in Europe.

开始日期2026-04-22

申办/合作机构

Ascendis Pharma A/S

NCT07264634

/ RecruitingN/A

A Study to Assess the Amount of Palopegteriparatide in Breast Milk of Lactating Females Requiring YORVIPATH® (Palopegteriparatide)

This is an observational, opportunistic lactation study to be conducted in lactating female participants who are currently receiving therapeutic doses of YORVIPATH as part of their usual care and who have chosen to breastfeed their infant(s). The potential transfer of palopegteriparatide into breast milk will be assessed.

开始日期2026-03-19

申办/合作机构

Ascendis Pharma A/S

100 项与帕罗培特立帕肽相关的临床结果

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100 项与帕罗培特立帕肽相关的转化医学

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100 项与帕罗培特立帕肽相关的专利（医药）

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6,012

项与帕罗培特立帕肽相关的文献（医药）

2026-12-01·BONE

The associations of obesity and other covariates with bone turnover markers in children and adolescents

Article

作者: Ward, Leanne M ; Thabane, Lehana ; Rauch, Frank ; Wong, Andy Kin On ; Kim, Hyunwoo ; Adachi, Jonathan Derrick ; Papaioannou, Alexandra ; Ma, Jinhui

BACKGROUND:

Procollagen type 1 N-terminal propeptide (P1NP) and C-terminal telopeptide of type 1 collagen (CTX) reflect skeletal metabolism during growth. We examined associations of obesity and other biological and behavioral factors with P1NP and CTX and assessed whether obesity-related associations varied by sex and across age groups approximating the pre-pubertal, pubertal, and post-pubertal periods.

METHODS:

Data were obtained from Canadian Health Measures Survey participants aged 6-20 years. Analyses included 3278 participants for P1NP (1658 males, 1620 females; 367 with obesity) and 1484 for CTX (762 males, 722 females; 162 with obesity). Survey-weighted multiple regression models were used to examine their associations with obesity status, physical activity, parathyroid hormone (PTH), serum 25-hydroxyvitamin D₃ [25(OH)D₃], and high-sensitivity C-reactive protein (hs-CRP), with all variables included simultaneously. Given the cross-sectional design, associations were interpreted as correlational.

RESULTS:

Overweight and obesity were associated with lower P1NP z-scores (β = -0.320 and - 0.378, both p < 0.001) and lower CTX z-scores (β = -0.378, p < 0.001; β = -0.278, p < 0.05). Obesity-status-by-age-group interactions were observed for P1NP and CTX (p < 0.001 and p = 0.002, respectively), whereas sex interactions were not significant. Obesity was not associated with lower marker levels in the age group approximating the pre-pubertal period but was associated with lower levels in age groups approximating the pubertal and post-pubertal periods. Cross-sectional age-related curves suggested that the highest estimated P1NP and CTX levels may occur at younger ages among participants with obesity, whereas marker levels were lower at older ages. High hs-CRP was associated with lower P1NP z-scores (β = -0.577, p < 0.001), while PTH was positively associated with P1NP and CTX z-scores (β = 0.117 and 0.103, both p < 0.01). Serum 25(OH)D₃ status and physical activity were not significantly associated with either marker.

CONCLUSIONS:

Obesity-related differences in bone turnover varied across age groups, and the observed pattern may be broadly consistent with previously reported obesity-related differences in height velocity.

2026-12-01·IBRO Neuroscience Reports

Causal effect of thyroid function on cortical brain structure: A two-sample Mendelian randomization study

Article

作者: Chen, Jianhuai ; Wang, Ruifeng ; Shi, Jianxin ; Zhao, Heng

Background:

The causal relationship between variations in thyroid dysfunction and brain structure remains elusive. We intended to probe into this causal connection using a Mendelian randomization (MR) approach.

Methods:

Aggregated statistics from the genome-wide Association Study (GWAS) on Autoimmune thyroid dysfunction were obtained from the FinnGen database, including autoimmune hyperthyroidism (N = 2,190) and autoimmune hypothyroidism (N = 49,055). Aggregate statistics on cortical area and thickness for GWAS were obtained from the Enhancing NeuroImaging Genetics through Meta-Analysis (ENIGMA) Consortium (N = 51,665). The main method employed is inverse variance weighting (IVW), supplemented by weighted median, Mr-Egger regression, MR-pleotropy residuals, and outliers. Some sensitivity analyses were conducted to determine heterogeneity and pleiotropy.

Results:

For autoimmune hypothyroidism (exposure), significant causal associations were observed with cortical thickness (outcome type) in the following regions: pars triangularis (β = -0.003, 95% CI: -0.005 to -0.001, PTH = 0.023; PTH: P-value for cortical thickness,), posterior cingulate (β = 0.003, 95% CI: 0.000-0.006, PTH = 0.034), and transverse temporal (β = 0.006, 95% CI: 0.002-0.010, PTH = 0.004; β = 0.006, 95% CI: 0.001-0.011, PTH = 0.010). For autoimmune hyperthyroidism (exposure), significant causal associations were observed with cortical surface area (outcome type) in the entorhinal (β = -2.576, 95% CI: -4.714 to -0.437, PSA = 0.018; PSA: P-value for cortical surface area), frontal pole (β = -1.195, 95% CI: -2.118 to -0.271, PSA = 0.011), lateral occipital (β = -15.787, 95% CI: -31.171 to -0.403, PSA = 0.044), lingual (β = -10.778, 95% CI: -20.888 to -0.668, PSA = 0.037), postcentral (β = -16.360, 95% CI: -28.410 to -4.309, PSA = 0.008), precentral (β = -16.089, 95% CI: -29.281 to -2.897, PSA = 0.017), and superior parietal (with global weighting; β = 11.360, 95% CI: 0.435-22.285, PSA = 0.042). Additionally, autoimmune hyperthyroidism was causally associated with cortical thickness (outcome type) in the entorhinal (β = 0.009, 95% CI: 0.000-0.018, PTH = 0.039) and posterior cingulate (β = 0.004, 95% CI: 0.001-0.008, PTH = 0.025). These findings remained reliable in the sensitivity analyses.

Conclusions:

These findings suggest that autoimmune hyperthyroidism and autoimmune hypothyroidism may have potential causal effects on the surface area and thickness of specific regions of the cerebral cortex, but further studies are needed to fully understand the nature of this relationship.

2026-12-01·Radiology case reports

Multimodality imaging localization of a giant functional parathyroid adenoma in a young fitness trainer with delayed recognition of hypercalcemia: A case report

Article

作者: Sarin, Deepak ; Sharma, Vivek ; Sharma, Naman ; Uppal, Geetanjali ; Banka, Devansh ; Gupta, Dipti

Giant parathyroid adenoma (GPA) is a rare cause of primary hyperparathyroidism (PHPT) whose symptoms overlap with normal training fatigue and dietary shifts that calcium testing gets delayed in active people. A 36-year-old male fitness trainer presented with 13 kg of unintentional weight loss over six months with weakness, and nocturia, all of which he and the physicians who first saw him attributed to his training regimen and protein supplementation. Serum calcium was 16.4 mg/dL and parathyroid hormone (PTH) 1127.4 pg/mL, with acute kidney injury and bilateral nephrolithiasis; protein electrophoresis and free light chains excluded a paraproteinemic cause. Ultrasonography, ¹⁸F-FDG PET-CT, and Technetium-99m sestamibi scintigraphy were concordant, identifying a right-sided parathyroid adenoma of approximately 2.3 × 2.3 × 5.9 cm extending into the upper mediastinum. Focused right parathyroidectomy was performed under intraoperative PTH monitoring, and PTH fell from 2233 pg/mL before excision to 172 pg/mL ten minutes after. Histopathology confirmed a benign adenoma weighing 18 g, the diagnosis resting on the absence of capsular and vascular invasion; the Ki-67 index of 3 to 4% was supportive rather than defining. Serum calcium normalized to 9.0 mg/dL by the fourth postoperative day without complications. Here the nonspecific symptoms were initially attributed to physical training and protein supplementation, and calcium was not measured until the disease was advanced. The frequency of this diagnostic pattern cannot be established from a single case. What the case does show is that concordant multimodality localization enabled focused surgery for an adenoma of unusual size and anatomical extent.

275

项与帕罗培特立帕肽相关的新闻（医药）

2026-10-06

·GlobeNewswire-Health Care

Ascendis to Share Latest Advances in Hypoparathyroidism and Achondroplasia Programs at ASBMR 2026

COPENHAGEN, Denmark, Oct. 06, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the latest advances in its hypoparathyroidism and achondroplasia programs will be showcased during ASBMR 2026, the annual meeting of American Society for Bone & Mineral Research being held in Boston from October 9-12, 2026. Updates include an oral presentation by Aliya Khan, M.D., of combined skeletal data from the Phase 2 PaTH Forward and Phase 3 PaTHway trials showing normalization of bone mass and microarchitecture in clinical trial patients treated with TransCon® PTH (palopegteriparatide) over a 5-year period. “Our growing body of clinical trial data continues to illustrate the unique potential for our differentiated TransCon-based therapies to improve patient health and quality of life,” said Aimee Shu, M.D., Executive Vice President, Chief Medical Officer at Ascendis Pharma. “Compelling long-term data such as these help inform treatment paradigms focused on outcomes that matter most to patients, and we look forward to sharing additional results as our programs advance.” Ascendis presentations at ASBMR 2026: ORAL PRESENTATIONHypoparathyroidismSunday, Oct. 11Session: 11:30a.m.-12:30p.m.Presentation: 12:18 PM – 12:30 Calcium Homeostasis DisordersBallroom A&BID #6080: Long-Term Effects of Palopegteriparatide on BMD and TBS in Adults With Chronic Hypoparathyroidism: Pooled Results from the PaTH Forward and PaTHway TrialsPresented by Aliya Khan, M.D.POSTERS HypoparathyroidismThursday, Oct. 85:30-6:30p.m.ASBMR/RBDA Pre-Meeting Symposium---Sunday, Oct. 112:00-3:00p.m.Poster Session IIID #6680: Hypoparathyroidism: Understanding the Impact of Palopegteriparatide on Patients’ Health Related Quality of Life, Functioning, and Well-Being Presented by Meryl Brod, Ph.D.AchondroplasiaFriday, Oct. 95:30-7:00p.m.Plenary Poster Session---Saturday, Oct. 102:00-3:30pm Poster Session 1ID 6329: Improved Growth and Physical Functioning in Children with Achondroplasia Treated with Navepegritide in the ApproaCH Trial Open-Label ExtensionPresented by Ciara McDonnell, M.D.Thursday, Oct. 85:30-6:30p.m.ASBMR/RBDA Pre-Meeting Symposium ---Friday, Oct. 95:30-7:00p.m.Plenary Poster Session---Saturday, Oct. 102:00-3:30pm Poster Session IID 6320: Continued Improvements in Lower Extremity Alignment in Navepegritide-Treated Children With Achondroplasia: Week 104 Results from the ApproaCH TrialPresented by Leanne Ward, M.D.Sunday, Oct. 112:00-3:00p.m.Late BreakingPoster Session IILate Breaker ID 8736: Navepegritide Combined with Lonapegsomatropin for the Treatment of Children with Achondroplasia: 78-Week Results from the Phase 2 COACH Trial Presented by Ciara McDonnell, M.D. About TransCon PTH TransCon PTH (palopegteriparatide) is a prodrug of PTH (1-34), administered once daily, designed to provide stable levels of active PTH within the physiological range for 24 hours/day. TransCon CNP is approved as YORVIPATH® in the United States (U.S.), European Union (EU), European Economic Area (EEA), and certain other jurisdictions as a treatment for adults with hypoparathyroidism. About TransCon CNPTransCon CNP (navepegritide) is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide continuous exposure of active CNP to receptors on tissues throughout the body to counteract the overactive FGFR3 signaling in achondroplasia. TransCon CNP is approved as YUVIWEL® in the U.S. as a treatment for children with achondroplasia. Ascendis Pharma’s Marketing Authorisation Application for YUVIWEL is under review by the European Medicines Agency, with a decision anticipated in the fourth quarter of 2026. About TransCon hGHTransCon hGH (lonapegsomatropin) is a prodrug of somatropin administered once weekly, providing sustained release of active, unmodified somatropin. TransCon hGH is investigational in achondroplasia and is approved as SKYTROFA® in the U.S., EU, EEA, and elsewhere for the treatment of growth failure in children due to growth hormone deficiency and in the U.S. for the replacement of endogenous growth hormone in adults with growth hormone deficiency. 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, idiopathic, and genetic causes, including ADH1. 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 affect physical well-being and quality of life, and may be impacted by a range of individual, clinical, and social factors. Some individuals with achondroplasia require multiple procedures and surgeries to address specific functional or anatomical concerns. 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 ascendispharma.com 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 within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ planned oral presentation and posters at ASBMR 2026, (ii) the combined skeletal data from the Phase 2 PaTH Forward and Phase 3 PaTHway trials, including normalization of bone mass and microarchitecture in clinical trial patients treated with TransCon PTH (palopegteriparatide) over a 5-year period, (iii) the unique potential for Ascendis’ highly differentiated TransCon-based therapies to improve patient health and quality of life, (iv) the potential for long-term data to help inform treatment paradigms focused on outcomes that matter most to patients, (v) Ascendis’ plan to share additional results as its programs advance, (vi) Ascendis’ clinical development activities, including the ApproaCH Trial open-label extension and the Phase 2 COACH Trial of navepegritide combined with lonapegsomatropin, (vii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients and (viii) Ascendis’ use of TransCon to create new and potentially best-in-class therapies 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, without limitation: dependence on third‑party manufacturers, distributors, and service providers for Ascendis’ products and product candidates; risks related to regulatory review and approval, including the possibility of delays, requests for additional data or analyses, restrictions or limitations on use, approval with labeling that is more limited than expected, or failure to obtain approval in the United States, European Union, or other jurisdictions; clinical development risks, including that results from ongoing or future trials may not confirm earlier data; unforeseen safety or efficacy findings in development programs or on‑market products; manufacturing, supply chain, quality, or logistics issues that could delay development or commercialization; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen research and development or selling, general and administrative expenses and other costs impacting Ascendis’ business generally; market acceptance, pricing, and reimbursement challenges, including payer coverage decisions and health technology assessments; competitive developments, including new or improved therapies; intellectual property protection, freedom‑to‑operate, and litigation risks; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; cybersecurity, data privacy, and information technology disruptions; and the impact of international economic, political, legal, compliance, public health, and business factors, including tariffs, trade policies, currency fluctuations, and geopolitical events. 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 11, 2026, 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, TransCon, SKYTROFA®, YORVIPATH®, and YUVIWEL® are trademarks owned by the Ascendis Pharma group. © October 2026 Ascendis Pharma A/S. Investor Contact:Media Contact:Chad FugereMelinda BakerAscendis PharmaAscendis Pharma+1 (650) 519-7494+1 (650) 709-8875

临床2期上市批准临床结果临床3期

2026-10-06

·astrazeneca.com

Alexion to showcase Phase III data in hypophosphatasia and hypoparathyroidism at ASBMR 2026 Annual Meeting

Alexion, AstraZeneca Rare Disease, will deliver 16 presentations, including three oral presentations, across hypophosphatasia (HPP), chronic hypoparathyroidism (HypoPT) and early research at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting in Boston, Massachusetts, 9 to 12 October 2026. Highlights include: HICKORY: results from Phase III trial of efzimfotase alfa (ALXN1850) in treatment-naïve adolescents and adults with HPP CALYPSO: data on skeletal parameters from Phase III trialevaluating eneboparatide, an investigational parathyroid hormone (PTH) 1 receptor agonist, in adults with HypoPT Gianluca Pirozzi, Senior Vice President, Head of Development, Regulatory and Safety, Alexion, said: “At this year’s ASBMR Annual Meeting, findings across 16 presentations will notably demonstrate the breadth of our research in HPP and HypoPT, two rare, multisystemic diseases that can have far-reaching impacts on patients’ daily lives. In HPP, results from HICKORY, the first Phase III clinical trial to include patients with adult-onset disease, will further reinforce the potential of efzimfotase alfa to reduce treatment burden and redefine care expectations, while real-world evidence will provide important insights into the impact of HPP. Together with new bone data from the CALYPSO Phase III trial of eneboparatide in HypoPT, our presence reflects our commitment to developing meaningful innovations that address the needs of patients and their families.” Alexion presentations during the 2026 ASBMR Annual Meeting Lead Author Abstract Title Presentation Details Lead Author HPP Lead Author Peroutka, C. Abstract Title Genetic Characterization and Clinical Manifestations in Adults with Hypophosphatasia in the United States Presentation Details Welcome Reception and Plenary Poster Session Abstract #FRI-465 9 October 2026 05:30 – 07:15 PM ET Poster Session I Abstract #SAT-465 10 October 2026 02:00 – 03:30 PM ET Lead Author Moss, K. Abstract Title Understanding Disease Burden and Characterizing Diagnosis, Assessment, and Management of Adults with Hypophosphatasia in the UK: A Delphi Study Presentation Details Late Breaking Poster Session I Abstract #SAT-551 10 October 2026 02:00 – 03:30 PM ET Lead Author Dahir, K. Abstract Title Assessing the compliance, usability, health insights, and perceived value from an at-home digital assessment toolkit for adults with hypophosphatasia: a multicenter observational study Presentation Details Poster Session I Abstract #SAT-489 10 October 2026 02:00 – 03:30 PM ET Lead Author Lynch, L. Abstract Title Treatment Experience in Adult Patients with Hypophosphatasia (HPP): Results of a US Survey Presentation Details Poster Session I Abstract #SAT-482 10 October 2026 02:00 – 03:30 PM ET Lead Author Ohata, Y. Abstract Title Clinical Course and Candidate Biomarkers in Perinatal Hypophosphatasia Presentation Details Poster Session I Abstract #SAT-483 10 October 2026 02:00 – 03:30 PM ET Lead Author Padidela, R. Abstract Title Diagnostic Delay and Clinical Burden in Hypophosphatasia: Results from a Cross-Sectional, Multinational, Mixed-Methods Study Presentation Details Poster Session I Abstract #SAT-481 10 October 2026 02:00 – 03:30 PM ET Lead Author Dhaliwal, R. Abstract Title A Real-World US Survey Study Characterizing the Holistic Disease Burden in Adults with Hypophosphatasia Presentation Details Poster Session II Abstract #SUN-480 11 October 2026 02:00 – 03:30 PM ET Lead Author Tanfous, M. Abstract Title Cascade Diagnosis of the Rare Metabolic Disease Hypophosphatasia (HPP) Within Families: A Case Series Presentation Details Poster Session II Abstract #SUN-492 11 October 2026 02:00 – 03:30 PM ET Lead Author Dahir, K. Abstract Title Efficacy and Safety of Alkaline Phosphatase (ALP) Enzyme Replacement Therapy (ERT) Efzimfotase Alfa in Adolescents and Adults with Hypophosphatasia (HPP): Results of HICKORY as Part of a Three-Trial Phase 3 Clinical Program Presentation Details Oral Presentation Abstract #1116 11 October 2026 05:00 – 05:12 PM ET Lead Author HypoPT Lead Author Vizcaya, D. Abstract Title Chronic Hypoparathyroidism in the US: Prevalence and Incidence From 2018 to 2024, With Baseline Clinical Characteristics Presentation Details Welcome Reception and Plenary Poster Session Abstract #FRI-005 9 October 2026 05:30 – 07:15 PM ET Poster Session I Abstract #SAT-005 10 October 2026 02:00 – 03:30 PM ET Lead Author Siggelkow, H. Abstract Title Leveraging Structured and Unstructured EHR Data to Improve Treatment and Clinical Context Capture in Chronic Hypoparathyroidism Presentation Details Poster Session I Abstract #SAT-015 10 October 2026 02:00 – 03:30 PM ET Lead Author Siggelkow, H. Abstract Title Bone Manifestations in Chronic Hypoparathyroidism: A Retrospective Cohort Study Presentation Details Poster Session I Abstract #SAT-019 10 October 2026 02:00 – 03:30 PM ET Lead Author Khan, A. Abstract Title Balanced Effects of Eneboparatide on Skeletal Parameters: Results From a Phase 3, Randomized, Placebo-Controlled Study Evaluating the Efficacy and Safety of Eneboparatide in Adults With Chronic Hypoparathyroidism (CALYPSO) Presentation Details Oral Presentation Abstract #1089 11 October 2026 12:06 – 12:18 PM ET Lead Author Erdmann, M. Abstract Title Methodological Differences Limit the Feasibility of an Indirect Treatment Comparison Between the Phase III CALYPSO (Eneboparatide) and PaTHway (Palopegteriparatide) Trials in Chronic Hypoparathyroidism Presentation Details Late Breaking Poster Session II Abstract #SUN-519 11 October 2026 02:00 – 03:30 PM ET Lead Author Vizcaya, D. Abstract Title Epidemiology of Low Bone Density and Osteoporosis in Chronic Hypoparathyroidism: Baseline Prevalence and Medication Usage from National US Claims Presentation Details Poster Session II Abstract #SUN-014 11 October 2026 02:00 – 03:30 PM ET Lead Author Early Research Lead Author Celen, I. Abstract Title Plasma Proteomics Distinguishes Clinical Expression in ADO and Highlights Differential Inflammatory and Matrix Remodeling Pathways Presentation Details Oral Presentation Abstract #1143 12 October 2026 12:30 – 12:42 PM ET Notes Alexion Alexion, AstraZeneca Rare Disease, is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US. AstraZeneca AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Disease, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on social media @AstraZeneca. Contacts For details on how to contact the Investor Relations Team, please click here. For Media contacts, click here. Corporate and financial

临床3期

2026-09-21

·微信

全球新药研发进展一周速递

看前提示：以下新闻中药物、机构、靶点、适应症、临床试验/结果、专利、交易、转化医学等数据信息，均可至「智慧芽新药情报库」进一步检索查询。 https://synapse.zhihuiya.com/ 本周全球药物研发进展 1. FDA批准突破性基因疗法9月17日，美国FDA宣布，批准Ultragenyx Pharmaceutical公司开发的基因疗法Fayuvi（rebisufligene etisparvovec）上市。新闻稿指出，这是首款FDA批准用于治疗黏多糖贮积症IIIA型（MPS IIIA）儿童患者的疗法。MPS IIIA又称Sanfilippo综合征A型，是一种罕见遗传性疾病，会进行性损害大脑和神经系统，导致患儿随着时间推移逐渐丧失认知、语言及其他发育能力。在此之前，治疗仅限于症状管理，尚无获得FDA批准、旨在改变疾病自然进程的疗法。Fayuvi是一款一次性静脉输注基因疗法，利用经过改造、不具有感染性的9型腺相关病毒（AAV9）载体，将功能正常的SGSH基因拷贝递送至患者细胞中。 2. 礼来每周一次长效胰岛素在日本全球首批9月16日，礼来制药efsitora alfa在日本获批上市。efsitora alfa依托创新的Fc融合工程化技术，将改造后的胰岛素与人IgG2 Fc结构域结合，大幅延长药物体内半衰期，成功将传统每日注射的给药模式革新为每周一次皮下注射，QWINT系列四项三期临床研究的完整数据，为这款长效胰岛素的临床价值提供了支撑，其核心优势早已超越“降糖疗效达标”的基础要求。在覆盖初治、经治、不同病程的3000余名2型糖尿病患者中，每周一次efsitora alfa的糖化血红蛋白降幅全面非劣于甘精胰岛素、德谷胰岛素等主流每日基础胰岛素。 3. 上海莱士人纤维蛋白原获批上市9月18日，上海莱士公告，全资子公司南岳生物收到国家药监局签发的人纤维蛋白原注射剂《药品注册证书》（治疗用生物制品3.4类，规格0.5g/瓶）。该药由健康人血浆经分离、提纯、病毒去除灭活后冻干制成，用于先天性纤维蛋白原减少或缺乏症。纤维蛋白原是凝血过程中的关键蛋白，缺乏会导致伤口出血难止、自发出血，这类患者此前用药选择十分有限，国产获批后供应保障进一步增强。 4. 首个！诺华重磅核药仿制药获批9月14日，Curium宣布Bexlutry（[177lu]氧奥曲肽，研发代号：PNT2003）获得FDA批准上市。该药物是Lutathera（[177lu]氧奥曲肽）的等效版本，即仿制药。这是全球首个获批上市的核药仿制药。原研Lutathera是诺华收购Advanced Accelerator Applications获得的一款靶向2型生长抑素受体（SSTR2）的放射性配体疗法（RLT），于2017年首次在欧盟获批上市。目前，该药物的适用人群为不可切除或转移性、进行性、分化良好（G1和G2）的SSTR阳性胃肠胰神经内分泌肿瘤（GEP-NET）成人和12岁及以上儿童患者。据诺华财报，Lutathera在2025年的全球销售额为8.16亿美元。 5. 拜耳「非奈利酮」在美国获批重磅新适应症 9 月 17 日，拜耳宣布，美国 FDA 已批准 Kerendia™（非奈利酮）上市，用于治疗伴有 1 型糖尿病（T1D）的慢性肾脏病（CKD）成人患者。新闻稿显示，这是 30 多年来首个获得 FDA 批准用于该适应症的药物。非奈利酮是拜耳开发的 first-in-class 非甾体选择性盐皮质激素受体（MR）拮抗剂。2026 年 5 月，非奈利酮治疗 T1D 相关 CKD 的补充新药申请获得 FDA 授予的优先审评资格。此次 FDA 的批准基于 III 期 FINE-ONE 研究的积极数据。这些发现也得到了针对 2 型糖尿病（T2D）相关慢性肾脏病（CKD）患者的 III 期 FIDELIO-DKD 和 FIGARO-DKD 研究的汇总分析的支持。结果显示，超过 80% 的非奈利酮肾脏获益可归因于尿白蛋白/肌酐比值（UACR）的降低。 6. 礼来RET抑制剂「塞普替尼」新剂型在华获批9月18日，NMPA官网显示，礼来的塞普替尼片获批上市。塞普替尼是一种强效、高选择性、口服RET酪氨酸激酶抑制剂，其胶囊剂于2020年5月首次获FDA批准上市，成为全球首个获批的高选择性RET抑制剂。2022年10月，塞普替尼胶囊剂在国内获批上市，用于治疗转染重排（RET）基因融合阳性的局部晚期或转移性非小细胞肺癌成人患者、需要系统性治疗的晚期或转移性RET突变型甲状腺髓样癌成人和12岁及以上儿童患者、以及需要系统性治疗且放射性碘难治（如果放射性碘适用）的晚期或转移性RET融合阳性甲状腺癌成人和12岁及以上儿童患者。7. 全球首创新药在国内获批上市，针对儿童软骨发育不全9月18日，NMPA官网显示，BioMarin Pharmaceutical的注射用伏索利肽在华获批上市，用于治疗儿童软骨发育不全。伏索利肽是一种C型利钠肽（CNP）类似物。2021年，该药物先后在欧盟和美国获批用于增加5岁及以上骨骺（生长板）未闭合的软骨发育不全儿科患者的线性生长。该药是欧美批准的首个用于软骨发育不全儿童的治疗药物。一项随机、双盲、安慰剂对照的III期研究评估了伏索利肽在5岁及以上骨骺未闭合的软骨发育不全儿童中的疗效，受试者者被随机分配接受伏索利肽或安慰剂治疗，每日给药1次，持续52周。结果显示，伏索利肽显著增加了生长速度，在52周时的平均生长速度比安慰剂组高出1.57厘米/年。 8. 阿斯利康下一代长效疗法在美国申报上市9 月 18 日，阿斯利康宣布，旗下罕见病药物公司 Alexion 的 Efzimfotase alfa 注射液上市申请获得美国 FDA 受理，并被授予其优先审评资格，用于治疗 2 岁及以上低磷酸酯酶症（HPP）患者的治疗。今年 8 月，该药在中国的上市申请已获得 CDE 受理，并被纳入优先审评。依赛弗酶 α 注射液（Efzimfotase alfa，研发代号：ALXN1850）是阿斯利康开发的下一代长效酶替代疗法 (ERT)，如果获得批准，Efzimfotase alfa 将成为首个能够有效治疗骨骼异常和功能障碍的药物，且不受疾病发病时间的限制，并可采用便捷的双周给药方案。9. 60亿美元引进！武田重磅新药在美国申报上市当地时间 9 月 14 日，武田宣布，美国食品药品监督管理局（FDA）已受理其用于治疗中重度斑块状银屑病成人患者的药物 Zasocitinib（TAK-279）的新药申请（NDA），并给予优先审评资格。PDUFA 生效日期是 2027 年第一季度。Zasocitinib 最先由 Nimbus Therapeutics 和 AI 制药公司薛定谔共同开发，是一种在研的新一代高选择性强效口服酪氨酸激酶 2（TYK2）抑制剂。2022 年，武田以首付款 40 亿美元、最高 60 亿美元的金额从 Nimbus 公司收购了这款药物。此次 NDA 主要是基于关键的全球 III 期临床试验 LATITUDE PsO 3001（登记号：NCT06088043）和 3002（登记号：NCT06108544）研究的积极结果，其中所有主要终点和排名靠前的次要终点均已达到。 10. 安进DLL3/CD3双抗标签更新获FDA批准 9月15日，安进宣布其IMDELLTRA（tarlatamab）的标签更新获FDA批准，用于广泛期小细胞肺癌（ES-SCLC）患者，将细胞因子释放综合征（CRS）的监测时间从原24小时缩短至6-8小时。IMDELLTRA为靶向DLL3与CD3的双特异性T细胞衔接器（BiTE），2024年获FDA加速批准，是首个获批用于SCLC的DLL3靶向TCE；此次更新基于上市后真实世界证据，证实特定患者在严格风险管理下缩短监测安全可行。更多资讯，请滑动下方文字恒瑞全球首创新药国内申报上市 9月14日，CDE官网显示，恒瑞医药的SHR-2004申报上市。根据临床试验进展，本次申报的适应症为择期全膝关节置换（TKA）术后静脉血栓栓塞（VTE）预防。SHR-2004是恒瑞医药自主研发的一款靶向凝血因子XI（FXI）通路的人源化单抗，可特异性结合FXI及活化FXIa，阻断内源性血栓放大通路，同时保留基础止血功能，从机制上实现“抗栓不损止血”的精准平衡。值得一提的是，SHR-2004可通过静脉注射或皮下注射，并且仅需在术后4–8小时给药1次，无需每日反复注射，显著提高了患者用药依从性。翰森制药B7-H3 ADC拟纳入优先审评，针对骨肉瘤9月14日，CDE官网显示，翰森制药的瑞康立伏妥塔单抗（risvutatug rezetecan，ris-rez，研发代号HS-20093）拟纳入优先审评品种，用于治疗12周岁及以上经既往至少二线治疗失败的骨肉瘤患者。这是该药物第二项进入优先审评程序的适应症。瑞康立伏妥塔单抗是翰森制药自主研发的靶向B7-H3的抗体偶联药物（ADC），由全人源的B7-H3单克隆抗体与拓扑异构酶抑制剂（TOPOi）有效载荷共价连接而成，DAR值为4。2023年12月，翰森制药授予葛兰素史克（GSK）该产品在全球（不含中国内地、香港、澳门、台湾）的开发、生产及商业化独家许可。目前，GSK正在海外推进III期临床试验。强生公布EGFR/c-Met联合化疗Ex20ins肺癌最终结果9月14日，2026年国际肺癌研究协会（IASLC）世界肺癌大会（WCLC）于韩国首尔进入第三天，强生在Presidential Symposium 2全体大会环节再次更新了Amivantamab+化疗，在Ex20ins突变类型的最终OS数据。PAPILLON III 期研究（NCT04538664）中，中位随访时间为48.6个月，尽管化疗组中有76%的受试者接受了二线Amivantamab治疗，但Amivantamab联合化疗组的中位总生存期（OS）为34.3个月（95% CI，27.0-40.8），而化疗组为27.9个月（95% CI，24.0-32.4）。Amivantamab联合化疗在数值上使中位OS延长了6个月以上，死亡风险降低了13%（HR，0.87；95% CI，0.66-1.14；P<0.001）。=0.307）。宜联生物B7-H3 ADC又一项积极临床结果重磅发布2026年世界肺癌大会（WCLC 2026）主席论坛，中山大学肿瘤防治中心张力教授首发的TAISHAN-302研究，让靶向B7-H3的 ADC——依康坦博妥塔单抗（Tam-Peli，研发代号:YL201）成为全场焦点。TAISHAN-302研究的数据也同时在医学顶刊新英格兰医学期刊上。结果显示，依康坦博妥塔单抗二线治疗小细胞肺癌的中位OS为13.3个月 vs 9.4个月（HR 0.46，死亡风险降低54%），中位PFS为7.4个月 vs 2.8个月（HR 0.29，疾病进展或死亡风险降低71%），ORR为59.1% vs 9.7%，≥3级TRAE 46.4% vs 74.7%，ILD发生率仅4.9%，无一例4/5级事件。值得注意的是，截至数据截止日期2026年5月20日，Tam-Peli组66.2%患者仍在随访中，预计后续将获得更长的总生存期数据。 K药+Trodelvy一线肺癌III期研究数据公布，PFS和OS均未达到9月13日，Trodelvy（戈沙妥珠单抗）联合Keytruda（帕博利珠单抗）一线治疗非小细胞肺癌（NSCLC）的III期KEYNOTE-D46/EVOKE-03研究数据在2026年世界肺癌大会（WCLC）第一场主席研讨会（Presidential Symposium 2）上公布。该研究于今年6月9日宣布终止。KEYNOTE-D46/EVOKE-03是一项全球性、随机、开放标签临床试验（n=620），旨在评估Keytruda联合Trodelvy对比Keytruda单药治疗既往未接受过治疗且PD-L1 TPS≥50%、无EGFR/ALK/ROS1基因组突变的转移性NSCLC患者的有效性和安全性。截至2026年4月3日，中位随访时间为14.7个月。数据显示，Keytruda+Trodelvy组和Keytruda单药组的中位PFS分别为11.8个月和7.7个月（HR=0.81，P=0.0252），未达到统计学显著性；中位OS分别为21.5个月和22.8个月（HR=1.07，P=0.7155），同样未达到统计学显著性。依沃西头对头K药III期研究数据出炉9月13日，康方生物依沃西单抗对比帕博利珠单抗一线治疗非小细胞肺癌（NSCLC）的III期HARMONi-2研究的总生存期（OS）数据在世界肺癌大会（WCLC）大会正式披露。2024年5月，该研究在期中分析中达到了无进展生存期（PFS）主要终点，取得具有统计学显著性和临床获益的阳性结果。依沃西单抗组的中位PFS达到11.14个月，帕博利珠单抗组的中位PFS为5.82个月，风险比（HR）为0.51。本次更新的数据显示，依沃西单抗组和帕博利珠单抗组的中位OS分别为30.75个月和22.57个月，HR为0.73（P=0.009）。这意味着，HARMONi-2研究同时取得了具有统计学意义和临床意义的PFS、OS双阳性结果。信诺维EZH2抑制剂国内申报上市9月15日，CDE官网显示，信诺维的马来酸依格美妥司他片（研发代号：XNW5004）申报上市。用于既往接受过至少1线系统性治疗的复发或难治外周T细胞淋巴瘤成人患者，已纳入CDE优先审评。依格美妥司他是一款信诺维自主研发、针对表观遗传学靶点EZH2的小分子抑制剂，主要的适应症开发方向为外周T细胞淋巴瘤（PTCL）、滤泡性淋巴瘤（FL）及转移性去势抵抗性前列腺癌（mCRPC）。本次申报上市是基于一项依格美妥司他治疗复发或难治性外周T细胞淋巴瘤受试者的关键II期临床研究。研究结果显示关键单臂II期临床试验数据达到主要疗效终点，且安全性良好。百利天恒EGFR/HER3 ADC拿下首个一线治疗突破疗法9月14日，百利天恒EGFR/HER3 ADC新药BL-B01D1拟纳入第8项突破疗法，用于联合特瑞普利单抗一线治疗PD-L1阳性（CPS≥1）局部复发不可切除或转移性三阴性乳腺癌。此前，BL-B01D1已经拿下7项突破疗法，涵盖癌种包括鼻咽癌、非鳞状非小细胞肺癌、EGFR野生型非小细胞肺癌、食管鳞癌、小细胞肺癌、铂耐药复发上皮性卵巢癌、输卵管癌或原发性腹膜癌患者、尿路上皮癌患者。值得注意的是，此次为Bl-B0D1首次拿下一线治疗TNBC的突破疗法。广泛性焦虑障碍疗法3期试验达到主要终点9月14日，Definium Therapeutics宣布，lysergide（DT120）口腔崩解片（ODT）治疗成人广泛性焦虑障碍（GAD）的3期Panorama研究取得积极顶线结果。研究达到主要终点及全部关键次要疗效终点。公司计划于2026年第四季度与美国FDA举行NDA申报前会议，并预计于2027年上半年提交NDA。在主要终点方面，第12周时，100 µg DT120组汉密尔顿焦虑量表（HAM-A）总评分较基线的最小二乘均值变化为-9.8分，安慰剂组为-4.7分，组间差异为-5.1分（p<0.0001）。研究还达到全部关键次要终点。DT120是一款麦角酸衍生物和经典5-羟色胺能致幻剂，为lysergide的药物制剂，主要通过部分激动5-羟色胺2A受体发挥作用。强生10亿美元新药2期未达主要终点9月14日，Contineum Therapeutics（纳斯达克：CTNM）宣布，其在研药物JNJ-5120/PIPE-307治疗成人重度抑郁症（MDD）的2期概念验证研究MOONLIGHT-1未能达到主要疗效终点。受此消息影响，Contineum股价在当日盘后一度下跌约8%。此前于2023年4月，Contineum与强生旗下杨森制药就PIPE-307达成全球许可与开发协议，获得5000万美元首付款与2500万美元股权投资，并有权收取总额最高约10亿美元的里程碑付款，以及未来净销售额低两位数至高十几个百分点的分级特许权使用费。根据协议安排，是否继续推进PIPE-307在MDD、RRMS或任何其他适应症的开发，完全由强生自主决定。对Contineum而言，这已经不是PIPE-307第一次在2期折戟。2025年11月，该药在复发缓解型多发性硬化（RRMS）的2期VISTA试验中同样未能达到预设的主要与次要疗效终点。渐冻症临床II期失败，仍将推进III期临床 9月15日，法国生物公司 Axoltis Pharma 宣布，其用于治疗肌萎缩侧索硬化症（ALS）的合成肽候选药物 NX210c 在 2 期临床试验中未达到主要终点，但公司仍将凭借次要终点的积极表现继续推进该药的研究。该 2 期临床试验在法国开展，采用双盲、随机、多中心设计，共招募了 82 名 ALS 患者。在第 6 周时，患者血液中的神经丝轻链（NfL）水平与基线相比未出现显著变化。尽管在 4 个月后，NX210c 治疗组中有 25.4% 的患者观察到血液 NfL 下降趋势，高于安慰剂组的 11.8%，但这一差异在统计学上并不显著。在次要终点方面，试验观察到了显著的临床与生物学获益。通过ALSFRS-R评估，接受 5 mg/kg 和 10 mg/kg 剂量 NX210c 治疗的患者，其运动功能衰退速率分别降低了 64% 和 33%，表明患者在肢体及身体运动控制方面的衰退有所改善或减缓。未达标准，礼来放弃一款肥胖药9月14日，由于未能达到公司继续开发的高门槛要求，礼来（Eli Lilly）已决定终止其早期实验性肥胖症药物LY4064912的研发工作。该药物拟通过注射或输液方式给药，其在一项招募了77名受试者（涵盖健康志愿者以及超重或肥胖人士）并在加利福尼亚州、德克萨斯州和新加坡开展的Ⅰ期临床试验已被正式终止。对于该药物的具体作用机制，礼来未予以回应，同时该公司近期已停止公开披露其早期管线项目，并将其从官网及财报更新中移除。尽管出现这一局部挫折，作为肥胖症治疗领域的领导者，礼来仍凭借已获批上市的重磅产品Zepbound和Foundayo保持着强劲的Ⅱ期和Ⅲ期研发管线。正大天晴IL-4Rα单抗上市申请获CDE受理9月16日，中国生物制药公告，附属公司正大天晴自主研发的1类创新药TQH2722注射液（IL-4Rα单抗）已向NMPA药品审评中心递交新药上市申请并获受理，适应症为外用药控制不佳或不适合外用药治疗的成人中重度特应性皮炎。TQH2722为靶向IL-4Rα的人源化单抗，同时阻断IL-4与IL-13信号通路，两类细胞因子是特应性皮炎等2型炎症的核心驱动。同靶点的原研为赛诺菲/再生元度普利尤单抗（Dupixent），年销售额已超100亿欧元；TQH2722还在开发慢性鼻窦炎伴鼻息肉、哮喘等适应症。亨廷顿病口服疗法公布1/2期最终15个月结果Skyhawk Therapeutics日前宣布，口服小分子RNA剪接调节剂SKY-0515治疗亨廷顿病（HD）的1/2期临床研究公布最终15个月结果。分析显示，在第15个月主要评估时间点，与经重叠加权的外部自然病史对照相比，SKY-0515治疗患者的复合统一亨廷顿病评定量表（cUHDRS）评分较基线变化存在统计学显著差异。目前，该公司正在推进SKY-0515的全球2/3期关键性FALCON-HD项目。第15个月时，SKY-0515治疗患者的cUHDRS评分较基线平均提高0.94分，而来自Enroll-HD自然病史数据库的外部对照组平均下降0.65分，组间差异为1.59分（95% CI：1.09～2.09；p<0.001）。君实生物PD-1/VEGF+CTLA-4启动一线肝癌三期临床9月14日，君实生物在药品临床试验登记与信息公示平台网站上注册了JS207+JS007联合一线治疗晚期肝细胞癌的三期临床试验。JS207为一款PD-1/VEGF双抗，为君实生物的核心管线，重点探索肺癌、肝癌、结直肠癌、三阴乳腺癌等瘤种。JS207采用四价对称结构，其中PD-1采用Fab，而非同类产品的scFv结构，PD-1亲和力较同类药物高11倍。JS207+JS007联合一线治疗肝癌的二期临床中，表现出强劲的抗肿瘤活性，22例患者可评估疗效，ORR高达45.5%，DCR高达86.4%。安全性方面，3级及以上副作用发生率仅19.2%。复宏汉霖PD-L1 ADC再下一城，胸腺鳞癌后线数据出炉刚刚落幕的WCLC大会上，复宏汉霖PD-L1 ADC HLX43治疗既往经治晚期胸腺鳞状细胞癌的剂量扩增队列结果于壁报导览环节报告。在33例可评估的既往经治晚期胸腺鳞状细胞癌患者中，HLX43经盲态独立中心审查确认的客观缓解率达到45.5%，疾病控制率达到87.9%；潜在推荐II期剂量下——2.5 mg/kg和3.0 mg/kg两个剂量组的6个月无进展生存率分别为100%和90.9%。胸腺鳞癌患者数量少、研究入组慢、标准治疗失败后的选择有限，免疫治疗又伴随这一瘤种特有的自身免疫风险。在这样的疾病背景下，一个经历过中位3线治疗、近四分之三接受过免疫治疗的人群，仍能获得超过四成的客观缓解，说明HLX43正在从早期偶然信号走向更可信的瘤种级概念验证，进一步确认这一分子的成药潜力。全球首个！和铂医药开发出基于全人源重链抗体的AI模型 9月16日，和铂医药发布公告，其全资附属公司诺纳生物成功开发出全球首个基于全人源仅重链抗体（HCAbs）训练的语言模型 HCAbLM，相关研究论文《A foundation model learns the sequence and functional grammar of fully human heavy-chain-only antibodies》已发表于预印本平台 bioRxiv。HCAbLM 基于来自 73 只独立免疫的 HCAb 转基因小鼠、共计 3,180 万条全人源 HCAb 序列的大规模数据库构建，捕获全人源 HCAbs 的独特序列特征。研究表明，该模型学习到了与仅重链抗体形式相关的独特序列语法，揭示了传统抗体语言模型无法充分捕获的序列模式及其相互关系。勃林格殷格翰终止一项III期临床近日，勃林格殷格翰（Boehringer Ingelheim）已终止其评估实验性醛固酮合成酶抑制剂 vicadrostat（BI 690517）与 SGLT2 抑制剂Jardiance联合治疗具有进展风险的慢性肾脏病（CKD）成人的后期 III 期临床试验 EASi-KIDNEY。尽管此前的中期研究结果不错，但在对现有试验数据进行综合受益风险评估后，该公司与独立数据监测委员会（DMC）均得出结论，累积的数据无法支持对临床获益的明确预期。虽然 EASi-KIDNEY 试验已被终止，但勃林格殷格翰与礼来联合销售 Jardiance，目前仍有另外三项评估 vicadrostat 与 Jardiance 联合用药的 III 期试验正在进行中。默沙东first-in-class新药拟纳入突破性疗法9 月 16 日，CDE 官网显示，默沙东 MK-7240 注射液拟纳入突破性治疗品种，用于治疗中度至重度化脓性汗腺炎。MK-7240（Tulisokibart）是一款靶向肿瘤坏死因子样配体 1A（TL1A）的人源化单克隆抗体，最初由 Prometheus Biosciences 开发。2023 年 6 月，默沙东以 108 亿美金的价格收购 Prometheus，囊获了该产品。目前，默沙东正在开展 MK-7240 用于克罗恩病（III 期）、溃疡性结肠炎（III 期）、化脓性汗腺炎（II 期）、类风湿性关节炎（II 期）等适应症的临床研究。百时美施贵宝终止开发CD33 DAC9月16日，Orum Therapeutics披露，合作伙伴百时美施贵宝在审查临床数据后，决定终止继续开发ORM-6151（靶向CD33的DAC分子），已经支付的预付款不会返还，后续里程碑则倍终止。Orum Therapeutics为DAC领域的先驱之一，自研管线CD123 DAC已经推进到一期临床阶段，用于治疗AML。Orum Therapeutics此前达成两项与MNC的合作，此次终止的ORM-6151合作预付款为1亿美元，还包含最高 8000 万美元的后续里程碑付款。项目退回，监管文件显示 BMS 支付以后里程碑款项的义务已解除。管线于2024年推进到一期临床。合作中断其实不是 Orum 旗下 DAC 平台首挫，以前该公司在 I 期临床试验中出现一例致命性肝毒性事件，暂停了首个临床阶段 DAC 候选药物 ORM-5029 的研发。百利天恒3款ADC药物同日公示突破性疗法9月18日，根据CDE官网公示信息显示，百利天恒的三款ADC药物，EGFR/HER3双抗ADC药物BL-B01D1，HER2 ADC药物BL-M07D1，CLDN18.2 ADC药物BL-M05D1，同日进行突破性疗法的申请公示，显示公司ADC管线的集中爆发。其中EGFR/HER3双抗ADC药物BL-B01D1，申请适应症为既往经含铂化疗治疗失败的复发性或转移性胆道癌患者，也是其申请的第九项突破性疗法。HER2 ADC药物BL-M07D1，申请适应症为HER2表达的经含铂化疗治疗失败的局部晚期或转移性胆道癌患者，也是其申请的第二项突破性疗法。CLDN18.2 ADC药物BL-M05D1，申请适应症为既往经一线标准治疗的CLDN18.2阳性晚期胃癌或胃食管结合部腺癌（GC/GEJC）患者，是其申请的第一项突破性疗法。亚盛医药「利沙托克拉」拟纳入突破性疗法 9月18日，亚盛医药的APG-2575（利沙托克拉）拟纳入突破性疗法，用于联合阿可替尼治疗初治慢性淋巴细胞白血病/小淋巴细胞淋巴瘤（CLL/SLL）成人患者。利沙托克拉是亚盛医药自主研发的新型口服Bcl-2选择性抑制剂，2025年7月，利沙托克拉获得国家药品监督管理局（NMPA）附条件批准上市，用于既往接受过至少一种系统治疗（包含BTK抑制剂在内）的CLL/SLL成人患者。Ib/II期APG2575CU101研究结果显示，在复发或难治性以及初治CLL患者中，利沙托克拉单药组的客观缓解率（ORR）为67.4%（29/43），利沙托克拉联合利妥昔单抗组的ORR为84.6%（33/39），利沙托克拉联合阿可替尼组的ORR为97.7%（85/87）。上下滚动查看更多全球交易合作动态 1. 诺和诺德正式更名 9 月 14 日，诺和诺德宣布今日正式开启全新篇章，推出以「Lasting Health Starts Now」为核心的品牌重塑计划，启用单一名称「诺和（Novo）」，并以 The Novo Way 更新企业文化，助力公司以更专注、更高效、更具影响力的方式向前迈进。Novo 将作为公司日常名称，而 Novo Nordisk A/S 仍是其全球法定名称。更新后的视觉识别系统保留了源自 Novo 历史传承的 Apis 公牛标志，同时赋予品牌现代且面向未来的表达方式。 2. 7.5亿美元！GSK收购恩沐生物第二款三抗TCE9月15日，葛兰素史克（GSK）宣布与恩沐生物（Chimagen Biosciences）达成协议，将以最高7.5亿美元的总交易额收购后者的一款潜在同类最佳三特异性TCE抗体的全球权益。GSK拟开发该抗体用于治疗多发性骨髓瘤，计划在2027年启动I期临床试验。该协议建立在GSK与恩沐生物现有合作关系的基础上。2024年10月，GSK以8.5亿美元的总交易额收购TCE三抗CMG1A46。 3. 1.25亿美元首付款！维泰瑞隆与诺华完成交易9月16日，维泰瑞隆宣布，继双方于2025年7月公布选择权及资产购买协议后，诺华已于近日行使其独家选择权，收购维泰瑞隆专有脑部递送平台的全球权益。根据选择权协议的条款，公司将在交易完成后获得1.25亿美元付款。维泰瑞隆亦将保留使用该平台对部分选定资产进行开发、生产及商业化的权利。维泰瑞隆的新一代脑部递送平台有望突破治疗性药物穿透血脑屏障这一长期挑战，从而推动中枢神经系统（CNS）疾病治疗方式的变革。该平台基于自主研发的脑部递送模块，有望实现包括单克隆抗体、多肽、蛋白质、基因疗法和其他大分子生物制剂在内的多种治疗模式的脑部递送。 4. 诺和诺德终止GLP-1月制剂合作近日，Ascendis Pharma A/S 宣布，公司将重新获得开发、生产和销售基于 TransCon 技术的代谢和心血管疾病（包括肥胖症）产品的独家权利。TransCon（暂时连接）技术是 Ascendis 旗下用于新药开发的创新技术平台。其名字由 transient conjugation 缩略而来，该独特技术将惰性载体和有生物学活性的已知原型药物暂时结合，达到可控缓释的效果。目前，基于该平台，Ascendis 已有 3 款药物获批上市，分别是那韦培肽、帕罗培特立帕肽、隆培促生长素。 5. 赛诺菲转让20款药物近日，赛诺菲和欧洲成熟药物领先企业 Cheplapharm 宣布，双方计划建立战略合作伙伴关系。根据该协议，Cheplapharm 将从赛诺菲手中接管 20 种成熟药物及其在全球的三个生产基地。作为回报，赛诺菲将获得 Cheplapharm 26.4% 的股权。值得一提的是，此次转让的药品中，包括赛诺菲旗下抗凝血药 Lovenox/Clexane（依诺肝素），但本次交易不涉及美国市场。交易涉及的其余药品名单及具体财务条款尚未披露。 6. 礼来达成多项目合作，开发神经系统核酸疗法递送技术 9月15日，QurCan Therapeutics宣布，与礼来（Eli Lilly and Company）达成独家多项目研究与合作协议，双方将利用QurCan基于靶向工程响应性聚合物（TERP）的聚合物-脂质纳米颗粒（PLNP）递送平台，开发针对中枢神经系统（CNS）和外周神经系统（PNS）疾病的核酸疗法。该合作涉及多个研发项目，礼来还将对QurCan进行战略投资。根据协议，QurCan将利用TERP平台负责相关核酸疗法的优化，礼来则负责入选项目的进一步研究、临床开发和商业化。QurCan将获得首付款和来自礼来的战略投资，并有资格针对每个项目获得研究支持费用以及最高2.37亿美元的研究、开发和商业化里程碑付款。 7. 中国CAR-T平台成功授权出海9月17日，河北森朗生物科技有限公司（森朗生物）与美国Tempest Therapeutics（Tempest）签署独家选择权协议，授权Tempest可独家引进森朗生物CD7靶向慢病毒载体平台及多款体内CAR-T候选药物，具体金额尚未披露。森朗生物自主CD7靶向慢病毒载体平台，可通过靶向慢病毒载体，将CAR基因递送至患者体内CD7阳性T、NK细胞，原位生成CAR-T/CAR-NK细胞，省去传统自体CAR-T体外采血、制备流程。此次交易的核心管线是一款BCMA/GPRC5D双靶向体内CAR-T候选药物，目前正在复发/难治性多发性骨髓瘤患者中进行I期剂量递增试验。 8. 诺和达成AI合作，加速新药发现与开发 9月16日，诺和（Novo）与Anthropic联合宣布达成合作，借助Claude人工智能模型加速新药研发并推进AI驱动的软件开发。双方将共同解决诺和科学家与计算团队提出的关键药物发现难题，为特定科学挑战和工作流程开发针对性解决方案，以支持生物学推理，从而加快新药的发现与开发进程。作为初期目标，诺和将测试Claude Science应用于特定研发工作流中，重点解决双方联合能力预计能产生最大影响的科学问题。同时，诺和还将利用Anthropic的前沿模型提升其AI驱动的软件工程能力，这将在全公司范围内成为扩展人工智能应用的关键依托。 9. 14亿美元！诺和诺德押注口服大环药物研发合作 9月17日，Orbis Medicines宣布与诺和诺德达成战略合作和许可协议，以发现和开发用于心脏代谢疾病的下一代口服大环疗法。该协议能够加速开发一类新的口服药物，用于高价值的治疗靶点。根据协议条款，Orbis Medicines有资格获得高达14亿美元的前期和潜在开发和商业里程碑付款，以及未来产品销售的分层特许权使用费。Novo还将对Orbis Medicines进行战略投资，强调其对公司nGen平台和长期潜力的信心。此次合作将利用Orbis Medicines专有的nGen平台，该平台集成了生成人工智能和高通量合成能力。 10. 10亿美元！罗氏押注新型炎症免疫双抗合作 9月17日，Dualitas Therapeutics宣布与罗氏达成研究合作和许可协议，使用Dualitas的DualScreen™双特异性发现引擎，开发用于免疫学和炎性（I&I）疾病的新型双特异性抗体。在被认为是规模最大的双特异性发现工作之一中，Dualitas将从功能上筛选30多万种新型双特异性组合，以发现和开发双特异性抗体（“BsAb”）疗法。罗氏将负责所有后续的临床前开发、监管、制造和商业活动。根据协议条款，Dualitas将获得3650万美元的预付款，并可能获得额外的研究、开发和商业里程碑付款，潜在总交易价值高达10亿美元，此外还有分层特许权使用费。往期新药速递周报： 8.24全球新药速递周报 8.31全球新药速递周报 9.7全球新药速递周报 9.14全球新药速递周报 🔍智慧芽生物医药推出「Synapse Lens」，一款专为生物医药从业者打造的Chrome插件，能力由智慧芽新药情报库提供支持。它将成为您的“资讯阅读加速器”，一键将普通网页转化为结构化情报。 Synapse Lens，一款专为生物医药从业者打造的智能信息识别助手（内含安装手册）

100 项与帕罗培特立帕肽相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D12395	-	H05AA	-

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
甲状旁腺功能减退症	欧盟	Ascendis Pharma Bone Diseases A/S	2023-11-17
甲状旁腺功能减退症	冰岛	Ascendis Pharma Bone Diseases A/S	2023-11-17
甲状旁腺功能减退症	列支敦士登	Ascendis Pharma Bone Diseases A/S	2023-11-17
甲状旁腺功能减退症	挪威	Ascendis Pharma Bone Diseases A/S	2023-11-17

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
软骨发育不全	申请上市	美国	Ascendis Pharma A/S	-
软骨发育不全	申请上市	欧盟	Ascendis Pharma A/S	-
遗传性胰腺炎	临床3期	日本	Ascendis Pharma Bone Diseases A/S	2021-06-10
低钙血症	临床3期	日本	Ascendis Pharma Bone Diseases A/S	2021-06-10
甲状腺癌	临床3期	日本	Ascendis Pharma Bone Diseases A/S	2021-06-10

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


PaTHway (ENDO2026)	临床3期	甲状旁腺功能减退症	82	Palopegteriparatide	獵餘願壓鑰衊網鏇築夢(餘淵鏇襯衊夢蓋窪衊窪) = 鏇淵鏇積鬱鏇構網製網顧蓋積願鑰積顧憲壓鑰 (構鹽憲膚鏇鏇壓壓鏇艱 ) 更多	积极	2026-06-13
ENDO2026	N/A	甲状旁腺功能减退症	10	Palopegteriparatide	艱憲醖選鹹築遞襯選壓(繭願衊廠艱鹹憲襯夢壓): MD = 9.0 (95.0% CI, 6.69 ~ 11.31) 更多	积极	2026-06-13
ENDO2026	N/A	甲状旁腺功能减退症	10	Placebo		积极	2026-06-13
WCO_IOF_ESCEO2026	N/A	甲状旁腺功能减退症	183	PTH (1-34)	積壓積蓋鏇憲構願鹹觸(糧壓願醖齋淵憲顧鑰淵) = 鑰醖鑰製鬱淵鹹憲製憲醖醖觸窪膚衊鹹壓鏇獵 (淵憲餘膚鑰鏇憲鹹製選 ) 更多	积极	2026-04-16
NCT04701203 (PaTHway, Company_Website) 人工标引	临床3期	甲状旁腺功能减退症	73	Palopegteriparatide	觸艱構鬱膚獵遞窪鏇鑰(鑰鏇鑰鹽鏇夢觸觸襯壓) = 構餘醖壓膚選構襯獵鏇積鏇觸齋艱製觸獵積選 (觸製醖蓋廠觸襯衊構製 ) 更多	积极	2025-07-14
PaTHway (ENDO2025)	临床3期	甲状旁腺功能减退症	82	Palopegteriparatide	積簾遞選網積願醖繭膚(簾齋鹹積願遞鑰製憲簾) = 繭壓積範積糧壓鹽鬱醖襯獵襯蓋選簾觸襯網積 (窪壓鏇醖鹹構膚艱鏇顧 ) 更多	积极	2025-07-12
NCT04009291 (PaTH Forward, NEWS \| Corporate Publications) 人工标引	临床2期	甲状旁腺功能减退症	56	Palopegteriparatide	觸鑰襯淵淵艱鹽壓選夢(鹹憲廠憲膚壓廠簾顧製) = 顧壓壓鏇鹹鹹艱憲鹽蓋憲憲艱糧遞觸壓壓製鏇 (醖齋蓋積觸窪網觸艱願 ) 更多	积极	2025-05-18
path forward trial (ESPE2025)	临床2期	甲状旁腺功能减退症	59	Palopegteriparatide	艱顧範壓鏇鹽餘憲壓願(構繭鹹顧鹹選鹹窪遞窪) = 衊鹽憲網艱餘壓觸繭窪膚廠遞簾鑰廠積壓夢繭 (鏇積淵構夢獵糧醖淵醖 ) 更多	积极	2025-05-10
NCT04009291 (PaTH Forward, ESPE2025)	临床2期	甲状旁腺功能减退症	59	Palopegteriparatide	糧遞鹹顧簾構繭網鏇獵(簾醖築襯獵鹹築繭積齋) = 齋鬱壓獵窪淵醖遞鬱鹹窪鹹鑰選願壓膚膚艱壓 (簾壓鹹繭糧糧鬱糧願衊 ) 更多	积极	2025-05-10
ESPE2025	N/A	甲状旁腺功能减退症	40	TransCon PTH	築鏇範餘膚壓憲積壓窪(觸構範選網顧簾繭淵壓) = From 34 patients documented, 38% (n = 13) reported headache, 38% (n = 13) muscular spasms or arthralgia, 24% sleep disturbance or fatigue, 21% (n = 7) nausea, 21% palpitations, 15% injections site reactions, and 15% other gastrointestinal symptoms (n = 5). Ten patients reported no negative symptoms. 鏇獵鑰簾齋遞鑰繭鏇鹽 (願蓋繭築窪獵遞廠衊範 )	积极	2025-05-10
NCT04701203 (PaTHway, CTgov)	临床3期	甲状旁腺功能减退症	84	TransCon PTH (TransCon PTH)	簾夢鏇觸廠鏇願鏇繭鑰 = 鏇繭範積淵構鹽鏇壓壓獵遞齋憲顧網齋遞壓鑰 (鹽繭衊鬱選積艱顧獵壓, 鬱壓醖艱鬱簾鏇廠築壓 ~ 範夢鏇遞構簾積顧築遞) 更多	-	2025-02-28
NCT04701203 (PaTHway, CTgov)	临床3期	甲状旁腺功能减退症	84	Placebo (Placebo)	簾夢鏇觸廠鏇願鏇繭鑰 = 壓夢繭顧網衊壓範齋艱獵遞齋憲顧網齋遞壓鑰 (鹽繭衊鬱選積艱顧獵壓, 構積襯繭餘築淵獵顧夢 ~ 繭夢淵膚鹹範襯積夢製) 更多	-	2025-02-28