Japan – Eisai Submits New Drug Application for Mecobalamin Ultrahigh-Dose Formulation in Japan for the Indication of Amyotrophic Lateral Sclerosis

Jan 27, 2024 | Business

Eisai Co., Ltd. announced today that it has submitted a New Drug Application (NDA) for ultrahigh-dose mecobalamin (development code: E0302) for the indication of amyotrophic lateral sclerosis (ALS) to the Pharmaceuticals and Medical Devices Agency (PDMA) in Japan. In May 2022, ultrahigh-dose mecobalamin received orphan drug designation by the Ministry of Health, Labour and Welfare (MHLW).

This application is based on the results of JETALS (The Japan Early-Stage Trial of Ultrahigh-Dose Methylcobalamin for ALS), a Phase III trial to evaluate efficacy and safety of ultrahigh-dose methylcobalamin (mecobalamin) in early onset ALS patients, that was conducted as an investigator-initiated trial by a research team with Extraordinary Professor Ryuji Kaji (Principal Investigator), Tokushima University, and Professor Yuishin Izumi (Coordinating Investigator), the Department of Neurology, Tokushima University Graduate School of Biomedical Sciences, and Professor Satoshi Kuwabara (Coordinating Investigator), the Department of Neurology, Chiba University Graduate School of Medicine. The results of JETALS were published in the peer-reviewed journal JAMA Neurology.

ALS is an intractable, progressive, neurodegenerative disease that results in severe muscle atrophy and weakness in the muscles due to motor neuron dysfunction. As the main cause of death is respiratory failure due to paralysis of the respiratory muscles, without the use of an artificial respirator, death occurs within approximately 3 to 6 years from the onset of the disease. The number of patients in Japan is estimated to be approximately 10,000. Currently, there is no curative treatment established for ALS, and since there are only limited number of medicines approved in Japan and abroad, this is a disease with significant unmet medical needs.

Eisai considers neurology a therapeutic area of focus. As a human healthcare company, Eisai is committed to fulfill unmet medical needs in neurology and further its contribution to improving the benefit of patients and the people in the daily living domain.

About Mecobalamin

Mecobalamin (generic name, development code: E0302) is approved and marketed as Methycobal®, a 500 µg injection of mecobalamin indicated for the treatment of peripheral neuropathies and megaloblastic anemia caused by vitamin B12 deficiency. Methycobal is also approved as a tablet formulation (250µg and 500 µg) as well as a fine granule formulation (0.1%) indicated for the treatment of peripheral neuropathies. While the mechanism of action of mecobalamin in amyotrophic lateral sclerosis (ALS) is not known, it has been suggested in non-clinical research that mecobalamin may have efficacy through a neuroprotective effect and regeneration of nerve axons. Since the 1990s, clinical research has been carried out on ultrahigh-dose mecobalamin in ALS by a study group on neurodegenerative disease, funded through the Ministry of Health, Labour and Welfare’s Specified Disease Treatment Research Program. Short- and long-term trials of intramuscular injection of mecobalamin at 25 mg and 50 mg per day, which is respectively 50 and 100 times the approved dosage of Methycobal, suggested that ultrahigh-dose mecobalamin could have a clinical effect in ALS. Therefore, Eisai had conducted the Phase II/III clinical trial (Study 761) since 2006 and submitted a new drug application for ultrahigh-dose mecobalamin as treatment for ALS in May 2015 but withdrew the application in March 2016 after the Pharmaceuticals and Medical Devices Agency (PMDA) indicated that additional clinical trials were necessary.

Following favorable clinical trial results in JETALS, Eisai prepared to file a new drug application for ALS in Japan.

Media Inquiries:
Public Relations Department,
Eisai Co., Ltd.
+81-(0)3-3817-5120

Copyright ©2024 JCN Newswire. All rights reserved. A division of Japan Corporate News Network.

Japan – Eisai Submits Marketing Authorization Application in Japan for Anticancer Agent Tasurgratinib for Biliary Tract Cancer with Fgfr2 Gene Fusion

Dec 20, 2023 | Business

Eisai Co., Ltd. announced today that it has submitted a marketing authorization application in Japan for its in-house discovered fibroblast growth factor (FGF) receptor (FGFR1, FGFR2, FGFR3) selective tyrosine kinase inhibitor tasurgratinib succinate (generic name, development code: E7090, “tasurgratinib”) for biliary tract cancer with FGFR2 gene fusion. In Japan, tasurgratinib has received orphan drug designation for a prospective indication for unresectable biliary tract cancer with FGFR2 gene fusion by the Ministry of Health, Labour and Welfare, (MHLW). Under this system, this application will be subject to priority review.

This application is based on the results of a multicenter, open-label, single-arm clinical phase II trial (Study 201) in Japan and China conducted by Eisai. Study 201 enrolled patients with unresectable biliary tract cancer with FGFR2 gene fusion previously treated with gemcitabine-based combination chemotherapy. The primary endpoint of this study was objective response rate, and secondary endpoints included safety. Detailed results of the study will be presented at upcoming academic conferences.

The estimated number of patients with biliary tract cancer is approximately 25,000 in Japan1, 2 and the five-year survival rate for the cancer is approximately 25%, which makes it an intractable cancer with the second worst prognosis following pancreatic cancer.1 Drug therapy options are limited in comparison with other cancers, and as such it is a disease with significant unmet medical needs. FGFR2 gene fusion is observed in approximately 14% of intrahepatic cholangiocarcinoma, which account for 15-30% of biliary tract cancers.3 FGFR genetic aberrations such as the gene fusions are known to be deeply involved in the proliferation, survival and migration of cancer cells as well as tumor angiogenesis and drug resistance. As these genetic aberrations in FGFRs have been observed in various other types of cancers including biliary tract cancer, there is growing interest in FGFRs as a promising target for cancer therapy. By selectively inhibiting FGFR1, 2 and 3, and blocking those signals, tasurgratinib has been expected to become a new molecular targeted therapy for cancers with FGFR genetic aberrations.4

Eisai acknowledges “Oncology” as one of its key strategic areas, and will continue to focus on the discovery and development of anti-cancer drugs within drug discovery domains including “tumor microenvironment”, “proteostasis disruption”, “cell linage and cell differentiation”, and “inflammation, hypoxia, oxidative stress and cell senescence” under the Deep Human Biology Learning (DHBL) drug discovery and development organization. Eisai aspires to discover innovative new drugs with new targets and mechanisms of action from these domains with the aim of contributing to the cure of cancers.

Media Inquiries:
Public Relations Department,
Eisai Co., Ltd.
+81-(0)3-3817-5120

Copyright ©2023 JCN Newswire. All rights reserved. A division of Japan Corporate News Network.

SinoMab Submits another IND Application for SM17 for the Treatment of Atopic Dermatitis, Which was Accepted by NMPA CDE

Jun 13, 2023 | Business

A Hong Kong-based biopharmaceutical company dedicated to the research, development, manufacturing and commercialization of therapeutics for the treatment of immunological diseases – SinoMab BioScience Limited (Stock Code: 3681.HK, “SinoMab” or the “Company”), is pleased to announce that an Investigational New Drug application (“IND”) for SM17, a humanised anti-IL-17RB monoclonal antibody for injection, for atopic dermatitis (“AD”) , has been filed with and accepted by the Center for Drug Evaluation (“CDE”) of the National Medical Products Administration of China (“NMPA”). The present IND submission, once granted, will enable the Company to conduct comprehensive clinical development program in China which leads to indication for treatment of AD.

SM17 is a humanized, IgG4-k monoclonal antibody targeting IL-17RB, which is a global first-in-class monoclonal antibody drug targeting IL-17BR with the potential for treating atopic dermatitis, asthma, idiopathic pulmonary fibrosis and other immunological disorders. The IND is mainly for the treatment of AD, and the Company will initiate a Phase I clinical study in China upon approval of the present IND.

SM17 could suppress Th2 immune responses by binding to IL-17RB on Type 2 Innate Lymphoid cells (ILC2s) and Type 2 helper T (Th2) cells, blocking a cascade of responses induced by interleukin-25 (IL-25). IL-25 is a critical cytokine classified as “alarmin”, which has shown to be implicated in the pathogenesis of autoimmune and inflammatory skin diseases, especially for AD. Around the globe, about one-fifth of the population were affected by AD at a certain stage of life, especially during the period of children. The condition is extremely volatile and can be difficult to be totally cured. It significantly reduces the quality of life of patients, and requires highly effective products to make up for unmet medical needs.

As a long-standing chronic disease, new cases of AD are growing rapidly in China with broad market potential. According to Frost & Sullivan, there were approximately 65.7 million AD patients in China in 2019 and is expected to grow to 81.7 million in 2030, and 30% of which will be moderate-to-severe patients. China’s AD medicine market was US$600 million in 2019, and is expected to grow to US$1.5 billion in 2024, and is expected to increase to US$4.3 billion in 2030, indicating a considerable market size. We expect that targeting upstream mediators of the Th2 inflammatory cascade, such as IL-17RB, will have a broad effect on skin inflammation. Coupled with the vast market potential of AD market and the lack of effective treatment methods, it is believed that the research and development of its treatment has market potential.

The Company is also committed to advancing multiple indications studies for SM17, laying a foundation for proof of concept and drug launches. In the United States, as early as March 2022, SM17’s IND application for the treatment of asthma was approved by the U.S. Food and Drug Administration (“FDA”), and the first healthy subject had been successfully dosed in a Phase I clinical First-in-Human(“FIH”) clinical trial in June 2022. Since then, none of the subjects reported a serious adverse event. At present, we are in full steam ahead to accelerate the progress of our clinical study, and expect the Phase I clinical study can be completed by the end of this year, six months ahead of the original anticipated completion date. In China, in addition to the acceptance of the application of SM17 for the treatment of AD, the new drug application for the treatment of asthma was also accepted by NMPA CDE on May 19 this year. If approved, the phase I clinical trial will be accelerated.

Dr. Shui On LEUNG, Executive Director, Chairman and Chief Executive Officer of SinoMab said that, “As a global first-in-class humanised IgG4-k monoclonal antibody targeting IL-17RB, SM17 has made good progress in several new drug research with the Company’s proactive progress. The acceptance of the new drug application for SM17 not only helps the Company to carry out the clinical research and development project for the treatment of AD in China, but also reflects the high efficiency of the research and development of new drugs. The Company is confident in the significant clinical trial and commercial development prospects of the new drug of SM17, and expects that the new treatment option will benefit more Chinese patients in the future, to jointly write a new chapter in the treatment of AD. In addition, the Company will continue to insist on independent innovation, strive to improve the research of new drugs, continuously expand the indication population, and provide more effective treatment solutions for patients in China and worldwide with no efforts, with an aim to become a global leader in innovative therapies for immunological diseases.”

About SinoMab BioScience Limited
SinoMab BioScience Limited is dedicated to the research, development, manufacturing and commercialization of therapeutics for the treatment of immunological diseases. The R&D headquarters is located in Hong Kong and the production base is located in mainland China. The Company’s flagship product SM03 is a potential global first-in-target mAb against CD22 for the treatment of rheumatoid arthritis (RA) and has completed the Phase III clinical trial for rheumatoid arthritis in China, which has been recognized as one of the significant special projects of Significant New Drugs Development of the Twelfth Five-Year Plan Period and the Thirteenth Five-Year Plan Period. In addition, the Company possesses other potential first-in-target and first-in-class drug candidates, some of which are already in clinical stage, with their indications covering rheumatoid arthritis (RA), Alzheimer’s disease, systemic lupus erythematosus (SLE), pemphigus (PV), multiple sclerosis (MS), neuromyelitis optica spectrum disorder (NMOSD), non-Hodgkin’s lymphoma (NHL), asthma, and other diseases with major unmet clinical needs.

This press release is issued by Financial PR (HK) Limited on behalf of SinoMab BioScience Limited. For further information, please contact:

Financial PR (HK) Limited
Contact: Ms. Chloe Chiu / Ms. Serena Zhang / Ms. Cita Zhang / Ms. Willa Xue
Email: sinomab@financialpr.hk
Tel: (852) 2610 0846
Fax: (852) 2610 0842


Topic: Press release summary

Astellas Submits New Drug Application for Zolbetuximab in Japan

Jun 10, 2023 | Business

Ministry of Health, Labour and Welfare to evaluate zolbetuximab as treatment option for patients with advanced gastric and gastroesophageal cancers

TOKYO – WEBWIRE –

Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, Astellas) today announced the submission of a New Drug Application (NDA) on June 9, 2023 to Japans Ministry of Health, Labour and Welfare (MHLW) for zolbetuximab, a first-in-class investigational Claudin 18.2 (CLDN18.2)-targeted monoclonal antibody, for first-line treatment of patients with locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors are CLDN18.2-positive. If approved, zolbetuximab would be the first CLDN18.2-targeted therapy available in Japan for these patients.

Gastric cancer remains the third deadliest cancer in Japan, leading to approximately 50,000 deaths per year despite significant strides to reduce the impact of this cancer, said Pranob Bhattacharya, DrPH, MS, MBA, Executive Director and Interim Head of Immuno-Oncology Development, Astellas. Astellas submission of a New Drug Application to Japans Ministry of Health, Labour and Welfare for zolbetuximab demonstrates critical momentum in addressing the unmet needs of patients with gastric cancer in Japan.

The NDA is based on results from the Phase 3 SPOTLIGHT and GLOW clinical trials. The SPOTLIGHT study evaluated zolbetuximab plus mFOLFOX6 (a combination regimen that includes oxaliplatin, leucovorin and fluorouracil) compared to placebo plus mFOLFOX6. The GLOW study evaluated zolbetuximab plus CAPOX (a combination chemotherapy regimen that includes capecitabine and oxaliplatin) compared to placebo plus CAPOX.

In both SPOTLIGHT and GLOW, approximately 38% of patients screened for the trials had tumors that were CLDN18.2-positive (≥75% of tumor cells with moderate-to-strong membranous CLDN18 staining intensity), as determined by a validated immunohistochemistry assay.1,2

Click below for a copy of the full press release

Astellas Submits New Drug Application for Zolbetuximab in Japan

Eisai Submits Marketing Authorization Application for Lecanemab as Treatment for Early Alzheimer’s Disease in Great Britain

May 22, 2023 | Business

Eisai Co., Ltd. and Biogen Inc. announced today that Eisai has submitted a Marketing Authorization Application (MAA) for lecanemab, an investigational anti-amyloid beta (Aβ) protofibril antibody, for the treatment of early Alzheimer’s disease (mild cognitive impairment due to Alzheimer’s disease (AD) and mild AD dementia) with confirmed amyloid pathology in the brain, to the UK Medicines and Healthcare products Regulatory Agency (MHRA) in Great Britain. Lecanemab has been designated by the MHRA for the Innovative Licensing and Access Pathway (ILAP).

The MAA is based on the results of the confirmatory Phase III Clarity AD study and Phase IIb clinical study (Study 201), which demonstrated that lecanemab treatment showed a reduction of clinical decline in early AD, and is subject to a validation to determine whether it will be accepted by the MHRA. Lecanemab selectively binds and eliminates soluble, toxic Aβ aggregates (protofibrils) that are thought to contribute to the neurotoxicity in AD. As such, lecanemab may have the potential to have an effect on disease pathology and the progression of the disease. The Clarity AD study of lecanemab met its primary endpoint and all key secondary endpoints with highly statistically significant results.

Eisai serves as the lead of lecanemab development and regulatory submissions globally with both Eisai and Biogen co-commercializing and co-promoting the product and Eisai having final decision-making authority.

About the Innovative Licensing and Access Pathway (ILAP) in the UK

The ILAP is a program offered by the MHRA (UK) for development programs with the goal of reducing the time to market for innovative medicines that treat life-threatening or seriously debilitating conditions and/or conditions for which there is a significant unmet patient need. The ILAP aims to achieve this goal by enabling enhanced coordination between sponsors, the MHRA and reimbursement bodies such as National Institute for Health and Care Excellence (NICE), leading up to Marketing Authorization Application (MAA) submissions to support accelerated access.

About Lecanemab

Lecanemab is the result of a strategic research alliance between Eisai and BioArctic. It is a humanized immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against aggregated soluble (protofibril) and insoluble forms of amyloid-beta (Aβ).

In the U.S., lecanemab was granted accelerated approval for Alzheimer’s disease (AD) by the U.S. Food and Drug Administration (FDA) on January 6, 2023. On the same day, Eisai submitted a supplemental Biologics License Application (sBLA) to the FDA for approval under the traditional pathway. This application was accepted, and has been granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) action date of July 6, 2023. In Europe, Eisai submitted a MAA to the European Medicines Agency (EMA) on January 9, 2023, which was accepted on January 26, 2023. In Japan, Eisai submitted a Marketing Authorization Application to the Pharmaceuticals and Medical Devices Agency (PMDA) on January 16, 2023, and Priority Review was designated by the Ministry of Health, Labour and Welfare (MHLW) on January 26, 2023. In China, Eisai initiated submission of data for a BLA to the National Medical Products Administration (NMPA) of China in December 2022, which was designated for Priority Review on February 27, 2023. In Canada, Eisai submitted a New Drug Submission (NDS) to Health Canada on March 31, 2023, and was accepted on May 15 of the same year.

Lecanemab is indicated for the treatment of AD in the U.S. Treatment with lecanemab should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials. There are no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied. This indication is approved under accelerated approval based on reduction in Aβ plaques observed in patients treated with lecanemab. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial.

Eisai has completed a lecanemab subcutaneous bioavailability study, and subcutaneous dosing is currently being evaluated in the Clarity AD OLE.

Since July 2020 the Phase III clinical study (AHEAD 3-45) for individuals with preclinical AD, meaning they are clinically normal and have intermediate or elevated levels of amyloid in their brains, is ongoing. AHEAD 3-45 is conducted as a public-private partnership between the Alzheimer’s Clinical Trial Consortium that provides the infrastructure for academic clinical trials in AD and related dementias in the U.S, funded by the National Institute on Aging, part of the National Institutes of Health, Eisai and Biogen. The Tau NexGen clinical study for Dominantly Inherited AD (DIAD), that is conducted by Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU), led by Washington University School of Medicine in St. Louis, has been ongoing since January 2022.

About the Collaboration between Eisai and Biogen for AD

Eisai and Biogen have been collaborating on the joint development and commercialization of AD treatments since 2014. Eisai serves as the lead of lecanemab development and regulatory submissions globally with both companies co-commercializing and co-promoting the product and Eisai having final decision-making authority.

About the Collaboration between Eisai and BioArctic for AD

Since 2005, Eisai and BioArctic have had a long-term collaboration regarding the development and commercialization of AD treatments. Eisai obtained the global rights to study, develop, manufacture and market lecanemab for the treatment of AD pursuant to an agreement with BioArctic in December 2007. The development and commercialization agreement on the antibody lecanemab back-up was signed in May 2015.

About Eisai Co., Ltd.

Eisai’s Corporate Concept is “to give first thought to patients and people in the daily living domain, and to increase the benefits that health care provides.” Under this Concept (also known as human health care (hhc) Concept), we aim to effectively achieve social good in the form of relieving anxiety over health and reducing health disparities. With a global network of R&D facilities, manufacturing sites and marketing subsidiaries, we strive to create and deliver innovative products to target diseases with high unmet medical needs, with a particular focus in our strategic areas of Neurology and Oncology.

In addition, we demonstrate our commitment to the elimination of neglected tropical diseases (NTDs), which is a target (3.3) of the United Nations Sustainable Development Goals (SDGs), with working on various activities together with global partners.

For more information about Eisai, please visit www.eisai.com (for global headquarters: Eisai Co., Ltd.), and connect with us on Twitter, LinkedIn and Facebook. For more information about Eisai in the EMEA region please visit www.eisai.eu.

About Biogen

Founded in 1978, Biogen is a leading global biotechnology company that has pioneered multiple breakthrough innovations including a broad portfolio of medicines to treat multiple sclerosis, the first approved treatment for spinal muscular atrophy, and two co-developed treatments to address a defining pathology of Alzheimer’s disease. Biogen is advancing a pipeline of potential novel therapies across neurology, neuropsychiatry, specialized immunology and rare diseases and remains acutely focused on its purpose of serving humanity through science while advancing a healthier, more sustainable and equitable world.

The company routinely posts information that may be important to investors on its website. Follow Biogen on social media – Twitter, LinkedIn, Facebook, YouTube.

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