This is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ≤2IU/dl residual FIX levels and aged 12-18 years old. BBM-H901 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of endogenous FIX.

Start Date23 Mar 2023

Sponsor / Collaborator-

NCT05203679 / Active, not recruitingPhase 3

A Study to Evaluate the Safety, Tolerability and Efficacy of an Adeno-associated Virus Vector Containing an Expression Cassette of the Human Factor IX Transgene (BBM-H901) Injection in Patients With Hemophilia B

This is a multi-center, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H901 injection in Hemophilia B subjects with ≤2 International unit per deciliter (IU/dl) residual factor IX (FIX) levels.
BBM-H901 is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor IX (hFIX) transgene and raises circulating levels of endogenous FIX.

Start Date30 Dec 2021

Sponsor / Collaborator

Shanghai Belief-Delivery BioMed Co., Ltd

CTR20212816 / Active, not recruiting

一项多中心、单臂、开放、单次给药临床研究评估BBM-H901注射液治疗血友病B的安全性、耐受性和有效性。

[Translation] A multicenter, single-arm, open-label, single-dose clinical study evaluated the safety, tolerability and efficacy of BBM-H901 injection in the treatment of hemophilia B.

剂量递增主要目的：评价单次静脉输注BBM-H901注射液在≥18岁且内源性凝血因子IX（FIX）活性≤2 IU/dL（即≤2%）的血友病B患者中的安全性和耐受性，并确认后续研究的推荐剂量。剂量扩展主要目的：评价单次静脉输注BBM-H901注射液在≥18岁且内源性FIX活性≤2%的血友病B患者中的有效性；评价单次静脉输注BBM-H901注射液在≥18岁且内源性FIX活性≤2%的血友病B患者中的安全性。

[Translation]

The primary purpose of dose escalation: To evaluate the safety and tolerability of a single intravenous infusion of BBM-H901 injection in hemophilia B patients aged ≥18 years with endogenous coagulation factor IX (FIX) activity ≤2 IU/dL (i.e. ≤2%), and to confirm the recommended dose for subsequent studies. The primary purpose of dose expansion: To evaluate the effectiveness of a single intravenous infusion of BBM-H901 injection in hemophilia B patients aged ≥18 years with endogenous FIX activity ≤2%; To evaluate the safety of a single intravenous infusion of BBM-H901 injection in hemophilia B patients aged ≥18 years with endogenous FIX activity ≤2%.

Start Date08 Dec 2021

Sponsor / Collaborator

Shanghai Belief-Delivery BioMed Co., Ltd

100 Clinical Results associated with Dalnacogene ponparvovec

100 Translational Medicine associated with Dalnacogene ponparvovec

100 Patents (Medical) associated with Dalnacogene ponparvovec

Literatures (Medical) associated with Dalnacogene ponparvovec

01 Jul 2022·The Lancet Haematology

Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial

Article

Author: Zhong, Chen ; Du, Zengmin ; Chi, Ying ; Zhang, Feixu ; Wei, Jun ; Li, Huiyuan ; Chen, Lingling ; Fu, Rongfeng ; Zhu, Ping ; Wu, Xia ; Zhang, Lei ; Cheng, Tao ; Wang, Wentian ; Yang, Renchi ; Zheng, Jing ; Liu, Xiaofan ; Pei, Xiaolei ; Liu, Wei ; Jiang, Wei ; Chen, Yunfei ; Tang, Dingyue ; Sun, Junjiang ; Xue, Feng ; Xiao, Xiao

BACKGROUNDA novel, engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX (FIX) protein (BBM-H901) has been developed and is promising for haemophilia B gene therapy. We aimed to explore its safety and activity in increasing FIX concentrations and reducing bleeding frequency.METHODSWe did a single-centre, single-arm, phase 1, pilot trial evaluating the safety and activity of a single intravenous infusion of BBM-H901 at the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College (Tianjin, China). We enrolled adult patients with haemophilia B (aged >18 years) with baseline FIX coagulation activity (FIX:C) of less than 2 IU/dL, no FIX inhibitor, and low titre of neutralising antibodies (≤1:4) against vector capsid. Eligible participants were intravenously infused with a single dose of 5 × 10¹² vector genomes (vg)/kg of BBM-H901 after 1 week of prophylactic prednisone treatment (1 mg/kg per day). Primary endpoints were the incidence of treatment-related adverse events, change in alanine aminotransferase (ALT) and aspartate amino transferase (AST), and development of antibodies against vector capsid within 1 year of infusion. We report the results of the prespecified 1-year analysis following complete enrolment. The trial is registered with ClinicalTrials.gov, NCT04135300, and is complete.FINDINGSBetween Oct 16, 2019, and Jan 13, 2021, 12 male participants were assessed, and ten Chinese participants were enrolled and infused with BBM-H901. After a median follow-up of 58 weeks (IQR 51·5-99·5), mean FIX:C reached mean 36·9 IU/dL (SD 20·5). No serious adverse events, no grade 3-4 adverse events were observed. Grade 1-2 adverse events related to BBM-H901 include pyrexia (1 [10%]) and elevation of aminotransferase(1 [10%]). No FIX inhibitors were observed. All participants developed antibodies against vector capsid after infusion. Eight (80%) participants had ALT and AST concentrations below the upper limit of normal throughout the follow-up period. Two (20%) participants had elevation of ALT and AST accompanied with decrease of FIX:C, which remained at 7 IU/dL and 11.8 IU/dL, respectively.INTERPRETATIONThis pilot study suggests that liver-tropic BBM-H901 is safe 1 year after infusion. Vector derived FIX:C concentration is sufficiently high to prevent bleeding events and minimise the need for replacement therapy in small populations with haemophilia B. These findings support further study.FUNDINGNon-profit Central Research Institute Fund of Chinese Academy of Medical Sciences, National Key Research and Development Program of China, National Natural Science Foundation of China, Tianjin Municipal Science and Technology Commission Grant, and Chinese Academy of Medical Sciences Innovation Fund for Medical Sciences.

01 Jul 2022·The Lancet Haematology

Factor IX Padua for haemophilia B gene addition: universal adaptation and repeated success

Article

Author: George, Lindsey A

This study report 1 yr of sustained mean factor IX (FIX) activity well into the mild range of hemophilia and sufficient to nearly eliminate bleeding events among ten Chinese participants with hemophilia B who received BBM-H901, an adeno-associated viral (AAV) vector mediating FIX Padua (F9 [Arg338Leu]) gene transfer.FIX Padua is a naturally occurring missense mutation in the catalytic domain of F9 that confers eight times greater specific activity.Herein authors builds on the existing foundation of hemophilia B addition efforts and supports the longstanding role of hemophilia B as the model monogenic disorder for the clin. development of systemic AAV vectors.

News (Medical) associated with Dalnacogene ponparvovec

05 Mar 2024

·www.businesswire.com

Hemophilia B Pipeline Insight Report 2024: Comprehensive Analysis of Emerging Therapeutics - ResearchAndMarkets.com

DUBLIN--( BUSINESS WIRE )--The "Hemophilia B - Pipeline Insight, 2024" report has been added to ResearchAndMarkets.com's offering. Significant advances in the treatment and management of Hemophilia B are forthcoming, according to a new comprehensive research publication now available on our website. This insightful report unveils a robust and diverse therapeutic pipeline, promising enhancements in care for individuals living with this rare genetic disorder. The culmination of data provides in-depth knowledge regarding over 15 companies' endeavors and over 20 pipeline drugs spanning clinical and nonclinical development stages. Global Research Gains on Hemophilia B Therapeutics The global coverage within this report transcends geographical boundaries, providing a universal perspective on the research and development efforts aimed at combatting Hemophilia B. This disorder, stemming from a deficiency of factor IX clotting activity, often leads to bleeding episodes that can be life-altering for those afflicted. The report details each therapy’s development phase, including Phase III and late-stage products, thereby offering stakeholders a clarion view into potential forthcoming treatments. Insights into Diagnostic Processes and Treatment Regimens The report elucidates the complexity of Hemophilia B diagnosis and underscores the importance of comprehensive blood coagulation tests. Furthermore, it delves into the nuances of replacement therapy - the current cornerstone of Hemophilia B management. Prophylaxis and other advanced treatment strategies are also discussed, highlighting gene therapy as an emerging powerhouse in the realm of Hemophilia B therapeutic options. Emerging Drugs and Key Players in Hemophilia B Therapeutics BBM-H901 by Belief Biomed is currently in the limelight as it strides into Phase III trials, presenting itself as a potential gene therapy candidate. TU7710, developed by TiumBio, offers a novel approach for patients with neutralizing antibodies and is currently under Phase I investigation. Innovative Strategies for Hemophilia B Treatment Within this comprehensive report, readers will uncover the extensive range of drugs in various development stages, from preclinical discovery to Phase III trials, emphasizing the industry's dynamic approach towards eradicating the challenges associated with Hemophilia B. The document scrutinizes multiple drug profiles, bringing forth their mechanistic actions, and shines a light on critical therapeutic assessments. Discoveries Set to Transform Hemophilia B Clinical Landscape The publication underscores the unmet needs within Hemophilia B therapeutic research and how the current pipeline is poised to address these gaps. The extensive catalog of developmental activities, including collaborations, mergers, and acquisitions, suggests a sector ripe with innovation and dedication to improving patient outcomes. A selection of companies mentioned in this report includes Shanghai Vitalgen BioPharma Belief BioMed TiumBio Jiangsu Gensciences Centessa Pharmaceuticals Pfizer OPKO Health Bayer Takeda Sanofi For more information about this report visit https://www.researchandmarkets.com/r/gfbg6e About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Phase 1Gene TherapyPhase 3

24 Apr 2023

·www.prnewswire.com

Belief BioMed Announces a Key Milestone of Dosing Completion for All Subjects in its Registrational Clinical Trial of BBM-H901

SHANGHAI, April 23, 2023 /PRNewswire/ -- Belief BioMed Group (BBM), an industry-leading biotech company focusing on innovative gene therapies, announced today the completion of patient dosing in its Phase III Registrational Clinical Trial (CTR20212816) of BBM-H901, a gene therapy for Hemophilia B developed and manufactured in-house by Shanghai Belief-Delivery Biomed Co., Ltd., its wholly owned subsidiary. BBM-H901 is indicated for prophylactic treatment of bleeding in adults with hemophilia B. It is the first adeno-associated virus (AAV) gene therapy for Hemophilia B with IND approval for registrational clinical study in China. This registrational study is a multi-center, single-arm, open and single-dose clinical study. Its objective is to evaluate the safety and efficacy of a single intravenous infusion of BBM-H901 in hemophilia B patients ≥18 years of age with endogenous blood coagulation factor Ⅸ (FⅨ) activity ≤2 IU/dL (≤2%). As of today, its dose exploration and dose escalation studies have been completed in China, and positive results have been obtained in terms of efficacy and safety: 100% patients stopped FⅨ treatment, and the annual bleeding rate (ABR) was reduced to zero; the level of coagulation factors was significantly increased and persisted in all patients; safety profile was good with no serious adverse event reported. Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said, "We are pleased to see patient dosing has been successfully completed in the phase III registrational trial of BBM-H901, the first blockbuster product independently developed and manufactured by BBM with the potential to become the first AAV gene therapy approved for marketing in China for prophylactic treatment of bleeding in adults with hemophilia B. Featuring 'one-time administration and long-term effectiveness', this innovative therapy will bring better survival benefits to patients. I want to express my heartfelt thanks to all investigators, patients and their families as well as the project team for their contribution to the trials. I believe it is a milestone that will lay a solid foundation for the future clinical application of more of our gene therapies and help promote the vigorous development of the entire gene therapy industry. Being fully committed to addressing unmet medical needs, BBM will continue to tap into the potential of gene therapy, and strive to bring more first-in-class and best-in-class products to patients!" Forward-looking statements The forward-looking statements made in this article only relate to events or information as of the date when the statements are made in this article. Except as required by law, we undertake no obligation to update or publicly revise any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. All statements in this article are made on the date of publication of this article and may change due to future developments. SOURCE Belief BioMed

Phase 3Gene TherapyIND

100 Deals associated with Dalnacogene ponparvovec

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Indication	Highest Phase	Country/Location	Organization	Date
Hemophilia B	NDA/BLA	CN	Shanghai Belief-Delivery BioMed Co., Ltd	25 Jul 2024
Hemophilia B	NDA/BLA	CN	Shanghai Mianyi Biotechnology Co., Ltd.	25 Jul 2024

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04135300 (ISTH 2024, NEWS) Manual	Not Applicable	Hemophilia B	10	BBM-H901注射液	(bplnqtkgge) = None xpskypqukl (argklusetv )	Positive	24 Jun 2024
NCT04135300 (Pubmed) Manual	Phase 1	Hemophilia B	12	BBM-H901	(nyvfeppfyi) = dndilvdzhh cyzkwggwnj (btyvbnibzl ) View more	Positive	01 Jul 2022

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