BeiGene Ltd and EUSA Pharma Receive Approval from China NMPA for QARZIBA®
BeiGene Ltd (BGNE) and EUSA Pharma announced that China National Medical Products Administration (NMPA) has granted QARZIBA® (dinutuximab beta) conditional approval for the treatment of high-risk neuroblastoma in patients aged 12 months and above who have previously received induction chemotherapy and achieved at least a partial response followed by myeloablative therapy and stem cell transplantation, as well as for patients with a history of relapsed or refractory (R/R) neuroblastoma with or without the residual disease. Dinutuximab beta is targeted immunotherapy approved by the European Medicines Agency (EMA).
From BeiGene Ltd and EUSA Pharma
Xiaobin Wu, Ph.D., President, Chief Operating Officer and General Manager of China at BeiGene, commented, “Dinutuximab beta represents an important biologic therapy for pediatric patients in China, having been listed in the first batch of New Drugs in Urgent Clinical Need Marketed Overseas by the NMPA. For these young patients fighting neuroblastoma in China, we are proud to bring the first approved treatment.”
Carsten Thiel, Ph.D., Chief Executive Officer of EUSA Pharma, said, “We are delighted that the benefit of dinutuximab beta has been recognized in China. This approval represents an important milestone in our mission and collaboration with BeiGene of bringing innovative cancer and rare disease therapies to patients.”
The SIOPEN Trial
Supporting the approval of dinutuximab beta in China for high-risk neuroblastoma were clinical results available from key trials conducted by SIOPEN (The International Society of Paediatric Oncology Europe Neuroblastoma Group) in collaboration with APEIRON Biologics and EUSA Pharma. These randomized controlled trials evaluated the efficacy of dinutuximab beta by comparing the administration of dinutuximab beta with and without interleukin-2 (IL-2) in the first-line treatment of patients with high-risk neuroblastoma and in two single-arm studies in the R/R setting.
In the SIOPEN trial (HR-NBL1), the five-year event-free survival (EFS) rate in patients treated with dinutuximab beta was 57% vs. 42% of historical controls, and the five-year overall survival (OS) rate was 64% vs. 50% ii The safety of dinutuximab beta has been evaluated in 514 patients.
The most common adverse reactions were pyrexia and pain that occurred despite analgesic treatment. Other frequent adverse reactions were hypersensitivity, vomiting, diarrhea, capillary leak syndrome, and hypotension.
About QARZIBA® (dinutuximab beta)
QARZIBA® is a monoclonal antibody that is specifically directed against the carbohydrate moiety of disialoganglioside 2 (GD2) which is overexpressed on neuroblastoma cells. Dinutuximab beta has been approved by the European Commission in 2017 (See EMA Summary of Product Characteristics (SmPC)) and is indicated for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with a history of relapsed or refractory neuroblastoma, with or without the residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilized by other suitable measures.
Founded in March 2015, EUSA Pharma is a world-class biopharmaceutical company focused on oncology and rare disease. The company has extensive commercial operations in the United States and Europe, alongside a direct presence in select other markets across the globe. EUSA Pharma is led by an experienced management team with a strong record of building successful pharmaceutical companies. It is supported by significant funding raised from leading life science investor EW Healthcare Partners.
For more information please visit www.eusapharma.com.
The firm has a growing R&D team of approximately 2,300 colleagues dedicated to advancing more than 90 clinical trials involving more than 13,000 patients and healthy volunteers. Its expansive portfolio is directed by a predominantly internalized clinical development team supporting trials in more than 40 countries.
Hematology-oncology and solid tumor-targeted therapies and immuno-oncology are key focus areas for the Company, with both mono- and combination therapies prioritized in its research and development.
Currently, BeiGene Oncology markets three drugs discovered and developed in its labs: BTK inhibitor BRUKINSA in the United States, China, Canada and additional international markets, and non-FC-gamma receptor binding anti-PD-1 antibody tislelizumab, and PARP inhibitor pamiparib.
BeiGene is a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide. With a broad portfolio of more than 40 clinical candidates the firm expedites the development of its diverse pipeline of novel therapeutics through its own capabilities and by collaborations.
BeiGene is committed to improving access to medicines for two billion more people by 2030. The firm has a global team of approximately 7,000 colleagues across five continents.
These companies share BeiGene’s goal of developing therapies to address global health needs. A range of oncology medicines licensed from Amgen and Bristol Myers Squibb are commercialized in China. The Company planned to address greater areas of unmet need globally through its collaborations including Amgen, Bio-Thera, EUSA Pharma, Mirati Therapeutics, Seagen, and Zymeworks.
BeiGene has also entered into a collaboration with Novartis granting it the rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.
To learn more about BeiGene, visit its website at www.beigene.com and follow it on Twitter at @BeiGeneGlobal.
Sagimet Biosciences is a clinical-stage biopharmaceutical company focused on developing a portfolio of internally discovered, selective fatty acid synthase (FASN) inhibitors for the treatment in several therapeutic areas of high unmet medical need.
Sagimet Biosciences in the NEWS
Sagimet Biosciences announced that clinicians have recently dosed the first patient with nonalcoholic steatohepatitis (NASH) in its FASCINATE-2 Phase 2b clinical trial.
From the CMO of Sagimet Biosciences and Others
Eduardo Bruno Martins, MD, DPhil, Sagimet Biosciences Chief Medical Officer, said, “The initiation of Phase 2 clinical trial marks an important milestone for the evaluation of our lead candidate TVB-2640. Building from the data seen in our FASCINATE-1 Phase 2a clinical trial, we believe TVB-2640 has the potential to make a meaningful difference for patients with NASH, the most aggressive form of nonalcoholic fatty liver disease (NAFLD), for which there are no FDA-approved therapies.”
Rohit Loomba, MD, MHSc, director, NAFLD Research Center, University of California San Diego, and coordinating investigator of the study, said, “After the robust and encouraging results from the Phase 2a study of TVB-2640, we are very excited to be enrolling individuals with NASH in the Phase 2b trial. The current standard of care for patients with NASH is suboptimal, and we anticipate the data from FASCINATE-2 will support advancing TVB-2640 into late-stage clinical trials.”
Stephen A. Harrison, MD, medical director for Pinnacle Clinical Research, San Antonio, Texas, and visiting professor of Hepatology, University of Oxford, who is an investigator in this trial, noted, “The well-designed FASCINATE-2 study will generate important biomarker and imaging data to support the global research efforts to replace liver biopsy, the current gold-standard, with non-invasive tests that may be useful to diagnose and follow patients in both routine care and clinical trials.”
The FASCINATE-2 Clinical Trial
FASCINATE-2 is a randomized, double-blind, placebo-controlled Phase 2b clinical trial of approximately 330 NASH patients with moderate to advanced fibrosis (F2-F3). This trial will evaluate the impact of oral, once-daily doses of TVB-2640 for 52 weeks as assessed by biopsy. Patients will initially be randomized to receive a placebo or 50mg of TVB-2640. A 75mg dose level of TVB-2640 is planned to be added following the ongoing open-label cohort in the FASCINATE-1 Phase 2a clinical trial expected to complete in the fourth quarter of 2021.
Primary efficacy endpoints in FASCINATE-2 are: ≥ 2-point improvement in Nonalcoholic fatty liver disease (NAFLD) Activity Score that results from a reduction of necro-inflammation (inflammation or ballooning), or improvement in fibrosis.
The FDA accepts the two endpoints for Phase 2b studies in NASH. Liver biopsy data will also be evaluated to assess NASH resolution without worsening of fibrosis and/or improvement in fibrosis without worsening of NASH, both of which are endpoints accepted by the FDA for accelerated approval following Phase 3 studies.
The study will also measure liver fat, assessed by MRI-PDFF, and other serum biomarkers of inflammation, fibrosis, and liver injury in a portion of patients at 26 weeks of treatment in an interim analysis. Sagimet anticipates sharing interim results in the second half of 2022 and top-line liver biopsy results in 2023.
Sagimet is developing TVB-2640 – an oral, once-daily selective FASN inhibitor for the treatment of NASH, an aggressive form of nonalcoholic fatty liver disease (NAFLD). It is the company’s lead drug candidate selected from more than 1,200 compounds in Sagimet’s library of FASN inhibitors.
TVB-2640 has been studied in over 300 subjects, including healthy volunteers and patients with NASH or cancer.
In the FASCINATE-1 Phase 2a clinical trial, TVB-2640 demonstrated statistically significant improvements across steatosis, inflammation/lipotoxicity, fibrosis, and metabolic biomarkers important in NASH, and was well-tolerated. Sagimet received the FDA Fast Track designation for TVB-2640 for the treatment of NASH in March 2021.
The company’s unique expertise in FASN biology enabled it to create a pipeline of proprietary FASN inhibitors.
For more please visit: www.sagimet.com.
In an upcoming comprehensive issue of the Prohost Letter, we discuss the firms that have investigational treatments for NASH.
As our subscribers know, we at Prohost Biotech are interested in companies that are developing treatments for many diseases with unmet needs.