On June 23, 2021 HUYABIO International (HUYABIO), the leader in accelerating global development of China’s pharmaceutical innovations, reported the regulatory approval for HBI-8000 monotherapy of relapsed or refractory (R/R) adult T-cell leukemia/lymphoma (ATL) by the Japanese Pharmaceuticals and Medical Devices Agency (Press release, HUYA Bioscience, JUN 23, 2021, View Source [SID1234584271]).

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"Relapsed and/or refractory ATLL carries a grim prognosis with limited treatment options. Data from the registration study of HBI-8000 has demonstrated meaningful disease response despite the advanced stage of disease, and acceptable safety profile, to address an important unmet medical need in this patient population", said Dr. Atae Utsunomiya, honorary hospital director of Imamura General hospital in Japan.

The drug was approved based on data from a Phase 2b study that involved 23 patients with aggressive ATL in Japan. These patients, having few effective treatment options, all had advanced disease either refractory to or relapsed after receiving mogamulizumab. HBI-8000 40mg orally administered twice weekly resulted in disease response in a clinically meaningful proportion of patients with an acceptable safety profile.

Dr. Mireille Gillings, CEO & Executive Chair of HUYABIO said, "This first regulatory approval for our lead oncology drug, HBI-8000, is a major milestone for the Company. The durability and strong immuno- oncology properties of HBI-8000 set the stage for improved cancer treatment of both solid and liquid tumors. Synergy with PD-1/PD-L1 inhibitors hold particular promise for major solid tumor advances."

About HBI-8000

HBI-8000 is an epigenetic immunomodulator approved for the treatment of lymphoma and metastatic breast cancer in China. This oral agent targets class I histone deacetylases (HDAC) and suppresses the expression of the viral oncogene HTLV-I bZIP factor, nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and the inflammasome in ATL cells. Furthermore, HBI-8000 may induce latent viral antigen expression making ATL cells more sensitive to immune cytotoxicity targeting.

On June 23, 2021 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that the U.S. Food and Drug Administration (FDA) has reviewed and confirmed all comments have been addressed regarding the Company’s clinical trial protocol for the Acclaim-1 clinical trial, an open-label, multi-center Phase 1/2 clinical trial evaluating the Company’s lead drug candidate, REQORSA Immunogene Therapy, in combination with AstraZeneca’s Tagrisso in patients with late-stage non-small cell lung cancer (NSCLC) whose disease progressed after treatment with Tagrisso (Press release, Genprex, JUN 23, 2021, View Source [SID1234584270]). In January 2020, Genprex received FDA Fast Track Designation for the Acclaim-1 patient population.

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In addition, the Company has engaged its first clinical site for Acclaim-1, and Genprex is continuing to work with a number of other important cancer research centers and academic institutions to select optimal study sites.

"This feedback from the FDA on our Acclaim-1 clinical trial and the engagement of our first clinical site are key milestones for Genprex," said Rodney Varner, President and Chief Executive Officer of Genprex. "We are now looking forward to opening patient enrollment in this important study of this cutting-edge investigational gene therapy to evaluate the role it can play in the fight against lung cancer, the leading cause of cancer deaths worldwide."

The Company expects the Phase 1 portion of the Acclaim-1 trial to enroll up to 18 patients at three clinical sites and for the Phase 2 portion to enroll approximately 74 patients (a 1:1 ratio of REQORSA and Tagrisso combination therapy versus Tagrisso monotherapy) at up to 15 clinical sites. The first part of the Phase 1/2 clinical trial will be a dose escalation study. The primary endpoint of the Phase 2 portion of the trial is progression-free survival, which is defined as time from randomization after first progression on Tagrisso, to first event (second progression) or death. An interim analysis will be performed at 51 events.

Genprex recently announced the Centralized Institutional Review Board (IRB) approval for the Acclaim-1 clinical trial in NSCLC. Additional information about the Acclaim-1 clinical trial can be found by visiting ClinicalTrials.gov.

On June 23, 2021 Genomic Testing Cooperative, LCA (GTC) reported that the New York State Clinical Evaluation Program (CLEP) has completed their evaluation and approved all GTC’s Next Generation Sequencing (NGS) profiling tests for hematologic neoplasms and solid tumors (Press release, Genomic Testing Cooperative, JUN 23, 2021, View Source [SID1234584269]). This includes:

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-GTC Hematology Profile (177 genes)
-GTC Hematology Fusion/Expression (1408 genes)
-GTC Hematology PLUS (177 DNA + 1408 RNA)
-GTC Liquid Biopsy, Hematology Profile (177 genes)
-GTC Solid Tumor Profile (434 genes)
-GTC Solid Tumor Profile Fusion/Expression (1408 genes)
-GTC Solid Tumor PLUS (434 DNS + 1408 RNA)

These tests are also covered by Medicare Administrative Contractor Palmetto GBA (MolDx). The tests are carefully designed to provide cost-effective comprehensive evaluation of clinically relevant molecular abnormalities and biomarkers.

"At GTC, we believe that all patients with cancer have the right to have their cancer fully molecularly profiled and for their physicians to be able to discuss their treatment options prior to initiating therapy. Medicare coverage combined with NY state approval make this testing easily accessible for cancer patients living in NY. Our cooperative (Co-Op) business model enables other laboratories and large oncology practice groups to obtain similar coverage and approval when they internalize our tests" said Dr. Maher Albitar, GTC Chief Executive Officer and Chief Medical Officer. "Our goal is to improve lives of cancer patients by democratizing NGS-based cancer profiling. We continue to collaborate with other Co-Op members to develop and validate new tests to address minimal residual disease, early diagnosis and other specific clinical indications" added Dr. Albitar.

GTC is first-in-class diagnostic company based on a cooperative business model. Using the most recent advances in NGS technology, GTC tests can be performed on minute samples including needle aspiration samples. Analysis of sequencing runs, curating data and interpretation of results is performed using proprietary algorithms/artificial intelligence (AI) software’s specially designed and developed for GTC’s technology. Cancer profiling combining DNA and RNA data is the most thorough approach not only for selecting therapeutic approaches and targeted therapy but also to help in establishing the diagnosis and classification of tumors, prognosis, and determining early relapse risk. GTC Hematology liquid biopsy is especially designed to be comprehensive and to replace the need for performing a bone marrow biopsy, which is a painful procedure and can be associated with complications.

On June 23, 2021 Strand Therapeutics, a privately held developer of next-generation, programmable mRNA therapeutics for cancer immunotherapy and other diseases, reported that the company raised $52 million in an oversubscribed Series A financing round syndicated by Redmile Group, BeiGene, Ltd., and Camford Capital, as well as existing investors Playground Global and ANRI. Strand has raised a total of $66 million to date (Press release, Strand Therapeutics, JUN 23, 2021, View Source [SID1234584268]).

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Founded by leaders in mRNA-based synthetic biology, Strand is creating the first platform for programmable, long-acting mRNA therapeutics that are bioengineered to enable precise control of the location, timing, intensity, and duration of their therapeutic activities. With the company’s self-replicating mRNAs, Strand’s goal is to develop improved treatment options for cancer and other life-threatening diseases.

"As shown with the COVID-19 vaccines, mRNA technology has revolutionized the way we can address diseases. At Strand, we have developed the next generation mRNA drug platform that can transform cancer treatment, cell therapy, and beyond," said Jake Becraft, PhD, co-founder and CEO of Strand. "We’re excited to work with this world-class syndicate of investors to continue our development of novel mRNA therapies and bringing patients more efficacious, accessible, and cost-effective treatments."

The company’s mRNA therapies combine genes for self-replication with genetically programmed logic circuits. By sensing and classifying unique expression signatures of cell types, the breakthrough technology enables precise and controlled delivery of multiple disease treatments in a single mRNA drug.

"While mRNA technologies allow drug development pipelines to move faster, existing platforms are limited due to lack of lasting therapeutic expression and control," said Jory Bell, General Partner at Playground Global and a member of Strand’s Board of Directors. "With the groundbreaking technology of programmable mRNA therapeutics, Strand is set to bring potentially better treatment options to patients that are more effective and less toxic than current standards. We look forward to continuing our work with the incredible team at Strand."

Strand’s initial focus is to develop its self-replicating programmable mRNA therapeutics to treat cancer, with plans to begin clinical trials of its first candidate in 2022. The company is also collaborating with the global biotechnology company BeiGene under a licensing agreement aimed at developing and commercializing Strand’s multi-functional mRNA technology to develop solid tumor immuno-oncology therapeutics.

"With this newest financing, we are well equipped to enable systemic delivery of mRNA to tumor cells and immune cells to tackle solid tumors as well as hematological malignancies," said Tasuku Kitada, PhD, co-founder, President, and Head of R&D at Strand. "The next generation of mRNA therapeutics will require new technology to move beyond vaccines and into cancer and other diseases. Our rapid growth will now accelerate both deeper into cancer immunotherapies, as well as beyond into further areas of high unmet medical need."

On June 23, 2021 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") reported that it has obtained manufacturing and marketing approval for the EZH2 inhibitor "Tazverik Tablets 200 mg" (tazemetostat hydrobromide) in Japan with the indication of relapsed or refractory EZH2 gene mutation-positive follicular lymphoma (only when standard treatment is not applicable) (Press release, Eisai, JUN 23, 2021, View Source [SID1234584266]).

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This approval is based on the results of a multicenter, open-label, single-arm clinical phase II trial (Study 206)1 in Japan conducted by Eisai and other studies2 conducted by Epizyme, Inc. (Headquarters: Massachusetts, United States) outside Japan. Study 206 enrolled patients with EZH2 gene mutation-positive, primarily follicular lymphoma, who had relapsed or were refractory. The primary endpoint of this study was objective response rate (ORR), and secondary endpoints included safety. This study achieved the primary endpoint target and exceeded a prespecified tumor response threshold with statistical significance: ORR in patients with EZH2 mutation-positive relapsed or refractory follicular lymphoma (n=17) was 76.5% (90% confidence interval (CI): 53.9-91.5) as measured by independent review. Treatment-emergent adverse events (incidence of 25% or more) observed in this study were dysgeusia (52.9%), nasopharyngitis (35.3%), lymphopenia (29.4%) and blood creatine phosphokinase increased (29.4%). Eisai will conduct a post-marketing special use results survey (all-case surveillance) in all patients who are administered "Tazverik" until a pre-determined number of patients has been reached in accordance with an approval condition imposed by the MHLW.

Created by utilizing Epizyme’s proprietary product platform, "Tazverik" is a first-in-class small molecule inhibitor of the epigenetic enzyme EZH2. It is one of the histone methyltransferases in the epigenetics-related protein group, and is thought to regulate the expression of cancer-related genes and suppress the growth of cancer cells by specifically targeting EZH2, which contributes to the cancer growth process.3 Eisai is responsible for the development and commercialization of this agent in Japan, while Epizyme, Inc. is responsible for all regions outside of Japan. In the United States, "Tazverik" received accelerated approval for the indication of epithelioid sarcoma in January 2020, and follicular lymphoma in June 2020 (Notes to editors 1).

Follicular lymphoma is a low-grade B-cell lymphoma that accounts for 10-20% of non-Hodgkin’s lymphomas. Follicular lymphoma is generally indolent and sensitive to chemotherapy. However, development of a new treatment strategy is required for follicular lymphoma which still remains difficult to cure, as recurrence often occurs repeatedly. 7-27% of follicular lymphomas are reported to have gain-of-function mutations in the EZH2 gene,4,5 and it is estimated that there are approximately 600 to 2,400 patients with follicular lymphoma with EZH2 gene mutations in Japan. A companion diagnostic test for EZH2 gene mutations, "cobas EZH2 Mutation Test" by Roche Diagnostics K.K. (Headquarters: Tokyo) was approved in May 2021.
　

Eisai aims to make continuous efforts to meet the diversified needs of and increase the benefits provided to patients with cancer, their families, and healthcare professionals, by delivering "Tazverik" as a new treatment option for EZH2 gene mutation-positive follicular lymphoma.