On December 2, 2021 InteRNA Technologies, a clinical-stage biotech company developing microRNA (miRNA)-based therapeutics with a focus on cancer, reported it received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for the company’s Phase I clinical trial evaluating lead miRNA candidate, INT-1B3, in patients with advanced solid tumors (Press release, InteRNA Technologies, DEC 2, 2021, View Source [SID1234596412]). INT-1B3 is a lipid nanoparticle (LNP) formulated, chemically modified mimic of the endogenous tumor suppressor, miR-193a-3p, and represents a promising novel therapeutic approach that is designed to simultaneously address multiple hallmarks of cancer.

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The IND approval enables InteRNA to expand the number of clinical sites and to facilitate the enrollment of patients for the dose expansion (Phase Ib) part of the trial in the United States. The first part of the Phase I trial was initiated in Europe at the beginning of 2021, with the dosing of the first patient in the dose escalation (Phase Ia) part in February. The treatment of the first patient in the Phase Ib cohort is planned for the second half of 2022.

"Receiving such a positive outcome from the FDA shortly after submitting a full IND application is a major achievement for us," commented Laurens van Pinxteren, Chief Operating Officer of InteRNA. "It underlines the high potential of our novel miRNA-based approach that enables us to address the multi-facetted disease cancer from different angles with one drug providing a novel therapeutic entity to patients with hard-to-treat solid tumors, such as advanced breast cancer or hepatocellular carcinoma."

Roel Schaapveld, CEO of InteRNA, added: "The IND approval enables us to enroll an international, diversified patient population, marking significant progress in the clinical evaluation of our novel therapeutic modality. The rapid, positive feedback by the FDA is highly encouraging and we look forward to start patient recruitment in the United States next year."

The multicentric, open-label, multiple ascending dose Phase I/Ib trial (NCT04675996) will investigate the safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of INT-1B3 in patients with advanced solid tumors. The study is expected to enroll a total of up to 80 patients at up to 15 clinical centers in the United States and Europe. The Phase Ia part of the trial is currently ongoing in the Netherlands and Belgium and will enroll approximately 30 patients with advanced solid tumors. In the second part (Phase Ib) of the trial, approximately 50 patients with hepatocellular carcinoma or triple negative breast cancer will be enrolled in the United States and Europe. Topline results from the Phase Ia part of the study are expected in the first half of 2022.

About INT-1B3

INT-1B3’s unique mechanism of action addresses multiple hallmarks of cancer simultaneously. It directly targets tumor cells and the tumor microenvironment by specific modulation of multiple signaling pathway components across the PTEN tumor suppressor pathway and the oncogenic PI3K/Akt and Ras/MAPK pathways resulting in inhibition of proliferation and migration and induction of cell cycle arrest and apoptosis. The triggering of the immunogenic tumor cell death (ICD) process as well as downregulation of the adenosine-A2A receptor pathway through inhibition of CD39/CD73 leads to a decrease in immunosuppressive FoxP3/Lag3 regulatory T cells and monocytic myeloid-derived suppressor cells (mMDSCs), and maturation of dendritic cells. As a result, the immune system is activated, and long-term immunity is triggered by recruitment of CD8+ effector T cells leading to decreased metastasis development and improved animal survival compared to anti-PD1 treatment.

On December 2, 2021 CTI BioPharma Corp. (NASDAQ: CTIC) reported that management will provide a corporate overview at the JMP Securities Hematology & Oncology Summit at 3:40 p.m. ET (Press release, CTI BioPharma, DEC 2, 2021, View Source [SID1234596395]). The conference will be held in a virtual meeting format.

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Presentation details:
Event: JMP Securities Hematology & Oncology Summit
Date: Monday, December 6, 2021
Time: 3:40 p.m. ET

The presentation will be webcast live and available for replay from the Investors section of CTI BioPharma’s website at www.ctibiopharma.com.

On December 2, 2021 Epizyme, Inc. (Nasdaq: EPZM), a fully integrated, commercial-stage biopharmaceutical company developing and delivering transformative therapies against novel epigenetic targets, reported that new data from across its oncology portfolio will be presented at the upcoming 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, taking place from December 11 to 14, 2021 in Atlanta, Georgia (Press release, Epizyme, DEC 2, 2021, View Source [SID1234596413]). These presentations include trial design and data from combination studies evaluating tazemetostat in follicular lymphoma, as well as new preclinical data on EZM0414, the Company’s novel, first-in-class, oral SETD2 inhibitor, an investigational agent being evaluated for the treatment of adult patients with relapsed or refractory multiple myeloma or with diffuse large B-cell lymphoma (DLBCL).

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"We look forward to sharing the latest data from our growing oncology portfolio with the hematology community at this year’s meeting, including updated data from the Phase 1b portion of our Phase 1b/3 confirmatory study, SYMPHONY-1, which is evaluating the safety and optimal dose of tazemetostat plus Revlimid and Rituximab (R2). We will also be presenting preclinical data for EZM0414, our novel, first-in-class, oral SETD2 inhibitor development candidate; these data provided the rationale for our Phase 1/1b study with EZM0414, and we will be sharing the trial design in a "Trial-In-Progress" poster as well," said Dr. Shefali Agarwal, Executive Vice President and Chief Medical and Development Officer. "These data will add to our knowledge of the important role epigenetics plays in B-cell malignancies, and we are excited about the potential that targeting epigenetic regulators may hold for patients living with blood cancers."

Details of the poster presentations are listed below:

TAZEMETOSTAT

Tazemetostat is a first-in-class, oral, selective inhibitor of EZH2, which is an epigenetic regulator of B-cell identity and plays a role in both normal B-cell biology and the pathogenesis of follicular lymphoma. The interim data analysis being presented highlights the potential of tazemetostat as a backbone of combination therapies for patients living with follicular lymphoma, and includes updated safety run-in data from the Phase 1b portion of the confirmatory SYMPHONY-1 study, in addition to study follow-up on 40 patients treated with tazemetostat in combination with R2. Additional presentations include trial design details of ongoing SYMPHONY-2, a Phase 2 study evaluating tazemetostat in combination with rituximab, as well as an analysis of the molecular and genetic characterization of patients treated with tazemetostat to better understand the drivers of response to treatment.

Title: Interim Analysis of the Randomized Phase 1b/3 Study Evaluating the Safety and Efficacy of Tazemetostat Plus Lenalidomide and Rituximab in Patients with Relapsed/Refractory Follicular Lymphoma
Presenter: Connie Lee Batlevi MD, PhD, Medical Oncologist, Memorial Sloan Kettering Cancer Center
Abstract No: 2207
Date: Sunday, December 12, 2021, 6:00 PM-8:00 PM
Location: Hall B5 (Georgia World Congress Center)
Title: Trial in Progress: A Phase 2, Single-Arm, Open-Label, Multicenter Study of Tazemetostat in Combination with Rituximab for the Treatment of Relapsed or Refractory Follicular Lymphoma
Presenter: Krish Patel, MD, Director of Lymphoma, Swedish Cancer Institute
Abstract No: 3541
Date: Monday, December 13, 2021: 6:00 PM-8:00 PM
Location: Hall B5 (Georgia World Congress Center)
Title: Molecular and Genetic Characterization of Tumor Samples from Patients with Relapsed or Refractory Follicular Lymphoma Identifies Factors Influencing Response to Tazemetostat
Presenter: Sandeep Dave, MD, MS, Associate Professor, Division of Hematologic Malignancies & Cellular Therapy, Department of Medicine at Duke Cancer Institute
Abstract No: 1183
Date: Saturday, December 11, 2021: 5:30 PM-7:30 PM
Location: Hall B5 (Georgia World Congress Center)
EZM0414

SETD2 is a histone methyltransferase, similar to EZH2, which plays multiple important roles in oncogenesis. The preclinical data to be presented focus on the pharmacologic inhibition of SETD2 by investigational agent EZM0414, as a potential therapeutic strategy in multiple myeloma and DLBCL. These data provided the rationale for the SET-101 study, the first-in-human Phase 1/1b clinical trial designed to evaluate safety and determine the optimal dose of EZM0414. Following this dose-ranging phase, the study will be expanded to evaluate EZM0414 in three patient cohorts: t(4;14) multiple myeloma, non t(4;14) multiple myeloma, and DLBCL.

Title: A Phase 1/1b Open-Label, Multicenter, Two-Part Study of SETD2 Inhibitor EZM0414 in Patients with Relapsed/Refractory Multiple Myeloma or Diffuse Large B-Cell Lymphoma
Presenter: Paul G. Richardson, MD, Medical Oncologist at Dana-Farber Cancer Institute
Abstract No: 1679
Date: Saturday, December 11, 2021: 5:30 PM to 7:30 PM ET
Location: Hall B5 (Georgia World Congress Center)
Title: Pharmacologic Inhibition of the Histone Methyltransferase SETD2 with EZM0414 As a Novel Therapeutic Strategy in Relapsed or Refractory Multiple Myeloma and Diffuse Large B-Cell Lymphoma
Presenter: Jennifer Totman, Principal Scientist at Epizyme
Abstract No: 1142
Date: Saturday, December 11, 2021: 5:30 PM to 7:30 PM ET
Location: Hall B5 (Georgia World Congress Center)
Revlimid + Rituximab (R2) is a registered trademark of Celgene Corporation, a Bristol Myers Squibb company.

On December 2nd, 2021 BioRay Pharmaceutical Co., Ltd. (hereinafter referred to as "BioRay") reported that the first patient with Primary immunologic thrombocytopenic purpura (ITP) had been dosed in the Phase II Clinical trial of self-developed Zuberitamab (development code: HS006) (Press release, Zhejiang Hisun Pharmaceutical, DEC 2, 2021, View Source;a=index&classid=43&id=3 [SID1234634620]). The study aims to evaluate the safety and efficacy of Zuberitamab (HS006) in subjects with primary persistent or chronic ITP who failed from prior therapy(ies). The leading entity of the clinical trial is Tongji Medical College, Union Hospital, Huazhong University of Science and Technology and the principal investigator is Prof.Yu Hu.

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Zuberitamab (HS006) is a human-mouse chimeric monoclonal antibody that specifically binds to B-lymphocyte antigen CD20 and can kill B cells via ADCC and CDC action. Zuberitamab (HS006) has demonstrated a favorable safety and efficacy profile in B-cell lymphoma in clinical trials. As B cells play an important role in the pathogenesis of autoimmune diseases such as primary immunologic thrombocytopenic purpura (ITP), rheumatoid arthritis (RA), and multiple sclerosis (MS), Zuberitamab (HS006) has the potential to be used in the treatment of autoimmune diseases.

Primary immunologic thrombocytopenic purpura (ITP) is an acquired autoimmune disease characterized by thrombocytopenia. The pathogenesis of ITP is mainly due to the loss of immune tolerance to the patient’s platelet membrane antigens. The patient’s B cells produce antibodies against different platelet antigens, leading to the formation of antigen-antibody complexes, and resulting in excessive destruction of platelets in the spleen or liver. Meanwhile, thrombopoiesis is impaired. Zuberitamab (HS006) specifically clears B-lymphocytes, thereby reducing autoantibody production and platelet destruction. Zuberitamab (HS006) is expected to bring an alternative treatment optionfor ITP patients and improve their quality of life.

"Zuberitamab (HS006) is a self-developed monoclonal antibody that targets the CD20 protein on the surface of B cells. It can rapidly, thoroughly and durably remove CD20+ B cells, and this effect is reversible after drug withdrawal, " said by Dr. Haibin Wang, the Chief Medical Officer (CEO) of BioRay, "The Phase III Clinical trial of Zuberitamab (HS006) for primary treatment of diffuse large B-cell lymphoma has completed the observation of the primary endpoint, and the study results confirmed its good safety, tolerability and efficacy, which provides strong support for the subsequent clinical development of ITP. Based on the existing achievements, BioRay will make every effort to promote its clinical research in ITP and other diseases and promote the early launch of this product in China to benefit more patients."

On December 1, 2021 Zetagen Therapeutics, a private, clinical-stage, biopharmaceutical company dedicated to driving breakthrough innovation in the treatment of metastatic bone cancers and osteologic interventions, reported it has received Breakthrough Device designation from the Centers for Devices and Radiological Health (CDRH) of the U.S. Food and Drug Administration (FDA) for its ZetaMet (Zeta-BC-003) technology (Press release, Zetagen Therapeutics, DEC 1, 2021, View Source [SID1234643705]). Previously known as ZetaFuse, ZetaMet (Zeta-BC-003) is a synthetic, small-molecule, inductive biologic technology being developed to target and resolve metastatic bone lesions while inhibiting future tumor growth and regenerating bone.

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"We are pleased to receive this important designation from the Agency and look forward to partnering with them," said Joe C. Loy, CEO of Zetagen Therapeutics. "Our researchers have discovered an entirely new pathway for an established molecule which, if proven successful in human clinical trials, could create a new treatment paradigm for the hundreds of thousands of patients living with cancers that involve metastatic bone lesions."

ZetaMet (Zeta-BC-003) works through a mechanism of action (MOA) which is a novel and patented molecular pathway. The small molecule, precisely-dosed, delivered to the affected area through a proprietary drug-eluting carrier, stimulates stem cells, activating cells to grow healthy bone known as "osteoblasts", and inhibits cells associated with bone degradation called "osteoclasts". The combination technology has, thus far, in preclinical studies, demonstrated its ability to resolve existing metastatic bone lesions, inhibit pain and stimulate targeted bone regeneration.

Bone metastases are common among cancer patients and occur when cells from the primary cancerous tumor relocate to the bone. When these cancers relocate, they can cause changes to the bone, damaging it in a process called osteolysis. Osteolysis can cause small holes within the bone, weakening it and increasing the risk of breakage. These holes are called "lytic lesions." Among cancers which metastasize to bone, Breast and Prostate are most prevalent, amounting to approximately 70-percent of cases.[1]

ZetaMet (Zeta-BC-003) has successfully passed its preclinical trials and is being prepared for its first human clinical trial in early 2022.