On August 27, 2018 Bayer reported that it has filed an application for approval for larotrectinib with the European Medicines Agency (EMA) (Press release, Bayer, AUG 27, 2018, View Source [SID1234529076]). Larotrectinib has been developed for the treatment of patients (adults and children) with locally advanced or metastatic solid tumors with a fusion in the neurotrophic tyrosine receptor kinase (NTRK) genes. NTRK gene fusions are changes in the genome that result in uncontrolled production of tropo-myosin receptor kinase (TRK) receptor fusion proteins and tumor growth.

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Larotrectinib is a highly selective TRK inhibitor. It acts purposefully against TRK fusion proteins, regardless of where in the body of a patient the cancer has developed. Bayer and Loxo Oncology, a biopharmaceutical company based in Stamford, Connecticut, USA, are developing larotrectinib together. In May 2018, the US Food and Drug Administration (FDA) approved the accelerated approval process for larotrectinib in the indication "Treatment of adults and children with locally advanced or metastatic solid tumors in which a NTRK gene fusion has been detected".

"Larotrectinib has achieved significant clinical success in patients with TRK fusion tumors, and the effect has been rapid and sustained, as has been observed in various types of tumors in both adults and children," said PD. Ulrik Lassen from the oncology department of Rigshospitalet in Copenhagen.

"The approval of larotrectinib would be a paradigm shift in cancer treatment, targeting the change in the genome that promotes cancerous growth, regardless of where in the body the cancer occurred." Scott Fields, Senior Vice President and Head of Oncology Development at Bayer. "The approval application for larotrectinib brings us one step closer to our goal of providing a much-needed treatment option in Europe for cancer patients with TRK fusion tumors."

About Larotrectinib (LOXO-101)
Larotrectinib (LOXO-101) is a potent, orally-to-be, selectively-acting new investigational drug currently in clinical development for the treatment of patients with a variety of cancers that have tropomyosin receptor kinase (TRK) abnormalities. play a role. Numerous studies suggest that the neurotrophic tyrosine receptor kinase genes (NTRK genes), which code for TRK and normally have major functions in the nervous system, may undergo abnormal fusions with other genes. This leads to growth signals that can cause cancer in numerous other areas of the body.

In clinical studies with patients presenting various types of solid tumors with NTRK gene fusions, larotrectinib showed an investigator-determined overall response rate (ORR) of 80 percent and an ORR of 75 percent, as confirmed by independent review. Larotrectinib was well tolerated with most of the adverse events reported being grade 1 or 2.

In November 2017, Bayer and Loxo Oncology announced that they would jointly develop and market the active ingredients larotrectinib and LOXO-195, another novel TRK inhibitor. Outside the US, Bayer will oversee regulatory activities and global marketing activities. Bayer and Loxo Oncology will jointly distribute the product in the United States. Loxo Oncology remains responsible for ongoing clinical trials and regulatory activities in the United States.

More information about the clinical trials with Larotrectinib or LOXO-195 can be found at www.clinicaltrials.gov or on the website www.loxooncologytrials.comavailable. Larotrectinib and LOXO-195 are not approved by the US Food and Drug Administration (FDA), the European Commission or other health authorities.

About TRK fusion cancer
TRK fusion cancer is due to NTRK gene fusions. These are chromosomal mutations that occur when one of the neurotrophic tyrosine receptor kinase (NTRK) genes binds abnormally to another, non-contiguous gene and results in an aberrant NTRK gene. The translated abnormal protein, or TRK fusion protein, is continuously active and this can lead to uncontrolled and possibly cancer-causing cell communication. These proteins are the major driver for the development and spread of tumors in patients with TRK fusion tumors. TRK fusion tumors can occur anywhere in the body because they are not bound to specific cell or tissue types. NTRK gene fusions occur in a variety of solid tumors in both adults and children. These include carcinoma of the appendix, breast cancer, bile duct carcinoma, colon cancer, GIST (gastrointestinal stromal tumors), fibrosarcoma in children, lung cancer, mammary analogue secretory carcinoma (MASC) of the salivary glands, melanoma, pancreatic cancer, thyroid cancer and various sarcomas. A TRK fusion tumor can only be diagnosed using sensitive and specific tests. Next generation sequencing (NGS) can provide a comprehensive view of the genomic changes in a variety of genes. Fluorescence in situ hybridization (FISH) and polymerase chain reaction (PCR) -based assays, on the other hand, are more suitable for highly targeted analysis because of their lower multiplex ability, while immunohistochemistry (IHC) is based on detection of the TRK protein.trkcancer.com/.

About Oncology at Bayer With the goal of improving people’s lives, Bayer is working to expand its portfolio of innovative treatments. Bayer’s Oncology division includes four approved compounds as well as other compounds in various stages of clinical development. All of these products reflect the company’s research approach, which focuses on the search for appropriate cancer targeting targets.

On August 27, 2018 Johnson & Johnson (NYSE: JNJ) reported that it will participate in the 2018 Morgan Stanley 16th Annual Global Healthcare Conference on Friday, September 14th, at The Grand Hyatt Hotel in New York (Press release, Johnson & Johnson, AUG 27, 2018, View Source [SID1234529398]). Alex Gorsky, Chairman and Chief Executive Officer will represent the Company in a session scheduled at 9:55 a.m. (Eastern Time).

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This webcast will be available to investors and other interested parties by accessing the Johnson & Johnson website at www.investor.jnj.com.

A webcast replay will be available approximately two hours after the live webcast.

On August 27, 2018 Kite, a Gilead Company (Nasdaq: GILD), reported that the European Commission (EC) has granted Marketing Authorization for Yescarta (axicabtagene ciloleucel) as a treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy (Press release, Kite Pharma, AUG 27, 2018, View Source [SID1234529078]). The Marketing Authorization approves axicabtagene ciloleucel for use in the 28 countries of the European Union, Norway, Iceland and Liechtenstein.

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Axicabtagene ciloleucel is a chimeric antigen receptor T cell (CAR T) therapy, which harnesses a patient’s own immune system to fight certain types of blood cancer. The cell therapy has been proven to induce complete response (no detectable cancer) in a proportion of patients with relapsed or refractory DLBCL and PMBCL, which are aggressive forms of non-Hodgkin lymphoma (NHL).

"Axicabtagene ciloleucel is a new and exciting way of treating cancer that offers a new option to patients with DLBCL and PMBCL in Europe," said Professor Gilles Salles, Head of Hematology, South Lyon Hospital Complex. "Many patients with these aggressive forms of non-Hodgkin lymphoma who have not responded to or failed commonly available treatment options have a very poor prognosis and there is an urgent need for new therapies."

The Marketing Authorization Application (MAA) is supported by data from the ZUMA-1 trial of axicabtagene ciloleucel in adult patients with refractory aggressive NHL. In the single-arm trial, 72 percent of patients (n=73/101) who received a single infusion of axicabtagene ciloleucel responded to therapy, with 51 percent (n=52/101) achieving a complete response (as assessed by an independent review committee, median follow-up of 15.1 months). At one year following infusion, 60 percent of patients were alive (95% CI: 50.2, 69.2) and the median overall survival (OS) had not been reached (95% CI: not estimable [NE]).

Axicabtagene ciloleucel may cause side effects that are severe or life threatening, such as cytokine release syndrome (CRS) or neurological toxicities. In ZUMA-1, 12 percent of patients experienced Grade 3 or higher CRS and 31 percent experienced Grade 3 or higher neurologic toxicities. Overall 98 percent of patients recovered from CRS and/or neurologic adverse reactions. Treatment algorithms have been developed to manage some of the symptoms associated with both CRS and neurologic adverse reactions experienced by patients on axicabtagene ciloleucel.

The most common Grade 3 or higher adverse reactions include encephalopathy, unspecified pathogen infection, CRS, bacterial infection, aphasia, viral infection, delirium, hypotension and hypertension.

For full details on the Special Warnings and Precautions for Use and Adverse Reactions (including appropriate management) please refer to the EU Summary of Product Characteristics (SmPC).

"We are proud to be leading this frontier of cancer innovation that is bringing novel, personalized therapy to people living with these blood cancers," said Alessandro Riva, MD, Gilead’s Executive Vice President, Oncology Therapeutics & Head, Cell Therapy. "Our vision is for cell therapy to serve as the foundation for treating all cancer types. Today’s milestone is another step on this exciting and important journey."

Axicabtagene ciloleucel was approved by the U.S. Food and Drug Administration on October 18, 2017.

On August 26, 2018 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported the acceptance by the China Drug Administration (CDA) of a new drug application (NDA) for zanubrutinib, an investigational Bruton’s tyrosine kinase (BTK) inhibitor, for the treatment of patients with relapsed/refractory mantle cell lymphoma (MCL) (Press release, BeiGene, AUG 26, 2018, View Source;p=irol-newsArticle&ID=2364823 [SID1234529065]). Zanubrutinib was discovered in BeiGene’s research facilities in Beijing, China, and is being developed globally by BeiGene as a monotherapy and in combination with other therapies to treat various hematologic malignancies.

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"We are proud of our team, and are appreciative of the clinical investigators and patients in China who made this first regulatory filing for zanubrutinib possible. This is BeiGene’s first NDA and is a significant milestone for our company. We look forward to additional regulatory submissions with zanubrutinib and with tislelizumab, our investigational anti-PD-1 antibody," commented John Oyler, co-founder, CEO and Chairman of BeiGene.

"We believe zanubrutinib is a potentially differentiated BTK inhibitor based on the depth and durability of responses observed in clinical trials of zanubrutinib to date. We are hopeful that zanubrutinib, if approved, may represent a valuable and important treatment option for patients in China with MCL," said Dr. Xiaobin Wu, General Manager of China and President of BeiGene, Ltd.

"We are excited that the CDA has accepted our new drug application of zanubrutinib for patients with MCL and that it is being reviewed as a Category 1 new drug submission, which is reserved for medicines that are going through their first worldwide regulatory review in China. We look forward to working with the CDA as it completes its thorough assessment of zanubrutinib," added Wendy Yan, Global Head of Regulatory Affairs at BeiGene.

The NDA is supported by an extensive clinical and non-clinical data package, including the results from an 86-patient single-arm pivotal Phase 2 study in Chinese patients with relapsed or refractory MCL treated with zanubrutinib, dosed at 160 mg orally twice daily. An independent review of response data from this study showed overall response rate (ORR) of 84 percent, including 59 percent of patients who achieved a complete response. With 8.3 months median follow-up, the median duration of response has not been reached, as a majority of the responders remain in a response. The safety profile was consistent with previously reported clinical data for zanubrutinib. Full results of the study are planned to be presented at an upcoming medical conference.

Zanubrutinib is being studied in several clinical trials as part of a broad development program and was recently granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with Waldenström macroglobulinemia (WM). BeiGene plans to submit an NDA to the FDA for zanubrutinib as a potential treatment for patients with WM in the first half of 2019 based on results from a global Phase 1 study.

In addition to the global Phase 1 trial of zanubrutinib, it is also being evaluated in a fully-enrolled, global Phase 3 clinical trial in patients with WM comparing zanubrutinib to ibrutinib, the currently approved BTK inhibitor for WM. Zanubrutinib is also being studied in a global Phase 3 clinical trial in patients with previously untreated chronic lymphocytic leukemia (CLL) and a pivotal Phase 2 trial in patients with relapsed/refractory follicular lymphoma in combination with GAZYVA (obinutuzumab). In China, BeiGene has completed enrollment in two other pivotal Phase 2 clinical trials of zanubrutinib in patients with CLL and WM, respectively. BeiGene also plans to initiate a Phase 3 trial comparing zanubrutinib to ibrutinib in patients with relapsed/refractory CLL/small lymphocytic lymphoma (SLL). As of August 2018, more than 1,500 patients have been enrolled in the zanubrutinib clinical development program.

About Mantle Cell Lymphoma
Lymphoma is a diverse group of malignancies that originates from B, T or NK cells. Mantle cell lymphoma (MCL) is typically an aggressive form of non-Hodgkin lymphoma (NHL) that arises from B cells originating in the "mantle zone." In 2013, the incidence of lymphoma was 4.2 per 100,000 and the mortality was 2.2 per 100,000 in mainland Chinai, making it the eleventh most common cancer and the tenth leading cause of cancer death.ii Mantle cell lymphoma usually has a poor prognosis, with a median survival of three to four years, although occasional patients may have an indolent course.iii Frequently, mantle cell lymphoma is diagnosed at a later stage of disease.

About Zanubrutinib
Zanubrutinib (BGB-3111) is an investigational small molecule inhibitor of Bruton’s tyrosine kinase (BTK) that is currently being evaluated in a broad pivotal clinical program globally and in China as a monotherapy and in combination with other therapies to treat various B cell malignancies.

On August 26, 2018 Harbour BioMed reported that it has completed a Series B round financing of $85 million to accelerate the growth of its innovative therapeutic pipeline, including both clinical and discovery stage programs (Press release, Harbour BioMed, AUG 26, 2018, View Source [SID1234529067]). GIC Private Limited, Singapore’s sovereign wealth fund, led the financing round, with participation from new investors, including China Life Private Equity Investment Company and Vertex Ventures, and Series A investors AdvanTech and Legend Capital

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"During the one and half years since we established operations, Harbour has successfully expanded its network of collaborations for its core transgenic mouse technologies, rapidly built an innovative pipeline through internal discovery and in-licensed development stage programs in the areas of oncology and immunology, and established an experienced and professional team," said Dr. Jingsong Wang, the Founder, Chairman and CEO of Harbour BioMed. "The financing is a very strong vote of confidence by our new and existing investors in our vision for the company, strategy, progress to date and our team. With their continued support, we will accelerate the growth and advancement of our pipeline through both internal innovation and external collaboration."

Harbour BioMed was established in December 2016 around a Series A round of $50 million and the acquisition of the Netherlands-based fully human transgenic antibody technology company, Harbour Antibodies BV and its subsidiaries. The company has integrated the Harbour Mice technologies into its newly built antibody technology and discovery biology, preclinical and clinical development operations, entered into multiple license and collaboration agreements for its Harbour Mice, and established an innovative therapeutic pipeline based on internal discovery and in licensing activities. In early 2018 Harbour raised an A+ Round from CDH and AdvanTech to support its in-licensed clinical programs for Greater China. Harbour’s development programs include:

An anti-FcRn based antibody against multiple autoimmune diseases, including myasthenia gravis and neuromyelitis optica, and a biologic against inflammatory dry-eye disease, among other potential indications. Harbour, which in-licensed these assets in 2017, is developing them for the Greater China market. Harbour recently filed two INDs for three different indications in China to conduct clinical trials with these assets.
A CD3-based bi-specific antibody therapy against Her-2 overexpressed cancer in clinical development, acquired in August 2018, which Harbour is developing for the Greater China market.
A clinical stage, anti-PD-L1 antibody for the treatment of multiple solid tumors and hematological cancers, acquired in August 2018, that Harbour is developing worldwide outside of China.