On July 1, 2020 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biotechnology company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported that the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has accepted a supplemental new drug application (sNDA) of BeiGene’s anti-PD-1 antibody tislelizumab for the treatment of patients with previously treated unresectable hepatocellular carcinoma (HCC), the most common form of liver cancer (Press release, BeiGene, JUL 1, 2020, View Source [SID1234561654]).

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"We are excited by the acceptance of our first filing for tislelizumab in liver cancer," commented Xiaobin Wu, Ph.D., General Manager of China and President of BeiGene. "Half of the world’s liver cancer patients reside in China, and there is a significantly unmet medical need. We will continue our communications with the CDE and hope to bring these patients a viable treatment option in the near future."

The sNDA is supported by clinical results from a pivotal Phase 2 trial of tislelizumab in patients with previously treated unresectable HCC (NCT03419897). The study enrolled 249 patients with unresectable HCC from eight countries and regions in Asia and Europe, including 138 patients who received one line of prior systemic therapy and 111 patients who received at least two lines of prior therapies. Patients received tislelizumab monotherapy (200 mg every three weeks) and the primary endpoint of the trial was objective response rate as assessed by independent review committee (IRC). Results of this study will be presented at an upcoming medical conference.

About Hepatocellular Carcinoma

HCC is a major global health problem, accounting for 85-90 percent of all reported cases of liver cancer.1 Liver cancer is the sixth most common type of cancer, with an estimated 841,080 new cases in 2018 worldwide;2 it was also the fourth most common cause of cancer-related mortality, responsible for an estimated 781,631 deaths in 2018.3 China accounts for approximately 50 percent of both new HCC cases and HCC-related deaths worldwide in 2018. 4

About Tislelizumab

Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.

Tislelizumab is approved by the China National Medical Products Administration (NMPA) as a treatment for patients with classical Hodgkin’s lymphoma who received at least two prior therapies and for patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

In addition, three supplemental new drug applications (sNDAs) for tislelizumab have been accepted by the Center for Drug Evaluation (CDE) of the NMPA and are under review, for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy, for first-line treatment of patients with advanced non-squamous NSCLC in combination with chemotherapy, and for previously treated unresectable hepatocellular carcinoma.

Currently, 16 potentially registration-enabling clinical trials are being conducted in China and globally, including 12 Phase 3 trials and four pivotal Phase 2 trials.

Tislelizumab is not approved for use outside of China.

About the Tislelizumab Clinical Program

Clinical trials of tislelizumab include:

Phase 3 trial in patients with locally advanced or metastatic urothelial carcinoma (NCT03967977);
Phase 3 trial comparing tislelizumab with docetaxel in the second- or third-line setting in patients with NSCLC (NCT03358875);
Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced squamous NSCLC (NCT03594747);
Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced non-squamous NSCLC (NCT03663205);
Phase 3 trial of tislelizumab in combination with platinum-based doublet chemotherapy as neoadjuvant treatment for patients with NSCLC (NCT04379635);
Phase 3 trial of tislelizumab combined with platinum and etoposide versus placebo combined with platinum and etoposide in patients with extensive-stage small cell lung cancer (NCT04005716);
Phase 3 trial comparing tislelizumab with sorafenib as first-line treatment for patients with hepatocellular carcinoma (HCC; NCT03412773);
Phase 2 trial in patients with previously treated unresectable HCC (NCT03419897);
Phase 3 trial comparing tislelizumab with chemotherapy as second-line treatment for patients with advanced esophageal squamous cell carcinoma (ESCC; NCT03430843);
Phase 3 trial of tislelizumab in combination with chemotherapy as first-line treatment for patients with ESCC (NCT03783442);
Phase 3 trial of tislelizumab versus placebo in combination with chemoradiotherapy in patients with localized ESCC (NCT03957590);
Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment for patients with gastric cancer (NCT03777657);
Phase 2 trial in patients with MSI-H/dMMR solid tumors (NCT03736889); and
Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment in patients with nasopharyngeal cancer (NCT03924986).

On July 1, 2020 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, reported that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on the Company’s application for orphan drug designation of its first-in-class telomerase inhibitor, imetelstat, as a potential treatment for myelodysplastic syndromes (MDS) (Press release, Geron, JUL 1, 2020, View Source [SID1234561640]). The Company expects that the European Commission, based on this positive opinion of the COMP, will formally grant the orphan drug designation for the European Union (EU) by the end of July. Imetelstat has already been granted orphan drug designation by the United States Food and Drug Administration as a potential treatment for MDS.

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"The positive opinion from the COMP acknowledges the compelling clinical data from our Phase 2 clinical trial that imetelstat has the potential to provide a clinically relevant advantage to lower risk MDS patients who are transfusion dependent and have failed to respond to treatment with erythropoiesis stimulating agents," said John A. Scarlett, Chairman and Chief Executive Officer. "Our ongoing IMerge Phase 3 clinical trial in lower risk MDS is being conducted at multiple sites around the world, and data from this trial are intended to support global regulatory filings."

To qualify for orphan drug designation in the EU, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient non-clinical or clinical data to suggest the investigational medicine may produce clinically relevant outcomes. EMA orphan drug designation provides companies with certain benefits and incentives, including clinical protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU member states, reduced regulatory fees and ten years of market exclusivity.

Ongoing IMerge Phase 3 Clinical Trial

The IMerge Phase 3 clinical trial is a double-blind, randomized, placebo-controlled clinical trial with registration intent. The trial is designed to enroll approximately 170 patients with lower risk transfusion dependent MDS who are relapsed or refractory to an ESA, have not received prior treatment with either a hypomethylating agent (HMA) or lenalidomide and who are non-del(5q). Geron expects to complete patient enrollment by the end of the first quarter of 2021 and top-line results to be available in the second half of 2022.

About Myelodysplastic Syndromes

MDS is a group of blood disorders in which the proliferation of malignant progenitor cells produces multiple malignant cell clones in the bone marrow resulting in disordered and ineffective production of the myeloid lineage, which includes red blood cells, white blood cells and platelets. Chronic anemia is the predominant clinical problem in patients who have lower risk MDS. Many of these patients become dependent on red blood cell transfusions due to low hemoglobin. Serial red blood cell transfusions can lead to elevated levels of iron in the blood and other tissues, which the body has no normal way to eliminate. Iron overload is a potentially dangerous condition. Studies in patients with MDS have shown that iron overload resulting from regular red blood cell transfusions is associated with lower quality of life, shorter overall survival and a higher risk of developing acute myeloid leukemia.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the apoptosis of malignant stem and progenitor cells, which allows potential recovery of normal hematopoiesis. Geron’s imetelstat development program includes two ongoing or planned registration-enabling studies, IMerge, an ongoing Phase 2/3 clinical trial in lower risk myelodysplastic syndromes (MDS), and a planned Phase 3 clinical trial in refractory myelofibrosis (MF) expected to be open for patient screening and enrollment in the first quarter of 2021. Imetelstat has been granted Fast Track designation by the U.S. Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment.

On July 1, 2020 ISA Pharmaceuticals B.V., a clinical-stage company dedicated to developing rationally designed immunotherapeutics for oncology and infectious disease, reported that it received Orphan Drug Designation from the Food and Drug Administration (FDA) in the USA for its lead product ISA101b for treatment of Human Papilloma Virus type 16 (HPV16)-positive cervical cancer (Press release, ISA Pharmaceuticals, JUL 1, 2020, View Source [SID1234561638]).

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According to the latest statistics of the Centers for Disease Control and Prevention, HPV causes more than 34,000 cases of cancer in the United States each year. Cervical cancer is the most common HPV-associated cancer in women, with HPV16 being responsible for approximately 50% of cases. HPV16-positive cervical cancer is the number 2 cause of cancer for women in the age 15-44 in the US. At 1% annually, rates of cervical cancer have been dropping only marginally due to prevention through screening and prophylactic vaccination. There is an important medical need for disease modifying therapies to treat women with this terrible disease. For 2020, SEER databases project 13,800 new cases and 4,290 women dying from cervical cancer in the US.

ISA101b is a clinical-stage immunotherapy targeting HPV16-induced diseases such as cervical and oropharyngeal cancer. It induces specific immune responses to the oncogenic E6 and E7 proteins of HPV16 and is based on ISA’s proprietary Synthetic Long Peptide (SLP) technology.

"We are pleased to have Orphan Drug Designation for our ISA101b program targeting HPV16-positive cervical cancers," said Kees Melief, Chief Scientific Officer of ISA Pharmaceuticals. "Our mission is to unleash the power of the patient’s own immune system to eradicate their cancer while maintaining optimal quality of life. We believe the best and most logical way to do this is through the use of SLPs. The metastatic form of cervical cancer has a particularly high unmet medical need with 5 year survival rates not going beyond 17%. The Orphan Drug Designation for ISA101b recognizes the urgent need for more efficacious treatment options for patients suffering from metastatic HPV16-positive cervical cancer."

About Orphan Drug Designation (ODD)

Orphan drugs are intended for the treatment, diagnosis or prevention of serious diseases that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. FDA evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. FDA provides incentives for sponsors to develop products for rare diseases, including development program tax benefits and a waiver of the NDA application user fee, as well as market exclusivity for up to seven years in the US once the product has been approved, provided that the product is first to market.

About ISA 101b

ISA101b is a Synthetic Long Peptide (SLP) therapeutic designed to mount a highly specific, broad and durable, T-cell mediated attack by the immune system on tumors positive for human papilloma virus type 16 (HPV16). The product has successfully and safely completed multiple human clinical studies in over 200 patients

ISA101 has been shown to be effective as monotherapy in patients with early stage, HPV16 positive, premalignant neoplasias1.
In late stage HPV16 positive cervical cancer, ISA101b acts favorably in synergy with chemotherapy as assessed by both size of HPV-specific immune response and clinical benefit2,3. Late stage cancer patients suffer from local and systemic immunosuppression by immune suppressive myeloid cells, that are depleted by standard of care chemotherapy such as carboplatin and paclitaxel. This enables ISA101b to more effectively attack the cancer via a T-cell mediated immune response.
Importantly, in a Phase 2, open label, Proof of Concept study, ISA101b in combination with nivolumab (anti-PD1 antibody) has shown tumour response rates and overall survival approximately double those seen with anti-PD1 monotherapy in 2nd line HPV16-positive head and neck cancer4.
ISA101b is protected by several patent families covering different aspects of the vaccine product. Patent protection of the vaccine product is expected until at least 2037.
ISA is currently carrying out a randomized, controlled phase 2 trial of ISA101b in combination with Libtayo (cemiplimab) in oropharyngeal cancer, under a strategic immuno-oncology collaboration with Regeneron, which was strengthened in June 2020. Further clinical trials are in preparation, including a potentially pivotal clinical trial for the combination of ISA101b and Libtayo in oropharyngeal cancer as well as a phase 2 study investigating the same combination in cervical cancer. Libtayo is being jointly developed by Regeneron and Sanofi.

On July 1, 2020 BioInvent International AB ("BioInvent" or the "Company") (OMXS: BINV) reported that it has further extended the research term under its cancer immunotherapy research collaboration and license agreement with Pfizer Inc. ("Pfizer") until the end of 2020 (Press release, BioInvent, JUL 1, 2020, View Source [SID1234561637]).

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In December 2016, the companies entered into the agreement for the development and commercialization of antibodies targeting tumor-associated myeloid cells discovered using BioInvent’s proprietary F.I.R.S.TTM drug discovery platform. The purpose of the research extension is to permit the companies to further identify and characterize new targets and antibodies binding to these targets.

In 2019 Pfizer selected the first and second target under the agreement, which triggered two payments from Pfizer to BioInvent of $300,000. BioInvent may be eligible for further milestone payments from development of antibodies directed against these targets as well as from the selection of additional targets and the development of antibodies directed against those targets.

Martin Welschof, CEO of BioInvent, said: "We are pleased to extend BioInvent’s long-running agreement with Pfizer which leverages our proprietary F.I.R.S.TTM technology platform and high-quality n-CoDeR antibody library. This extension provides Pfizer additional time to select optimal antibodies to their targets, as well as to explore more targets and corresponding antibodies. We look forward to continuing our successful collaboration with Pfizer."

On July 1, 2020 Sumitomo Dainippon Pharma Oncology, Inc. reported that was formed today through the merger of Tolero Pharmaceuticals, Inc., and Boston Biomedical, Inc., two clinical-stage companies developing novel cancer therapeutics based in the U.S. and wholly owned subsidiaries of Sumitomo Dainippon Pharma Co., Ltd. This U.S. entity, together with the oncology research and development teams in Japan, DSP Cancer Institute and Oncology Clinical Development Unit, will collectively be known as Sumitomo Dainippon Pharma Oncology or SDP Oncology (Press release, Sumitomo Dainippon Pharma, JUL 1, 2020, View Source [SID1234561636]).

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Consolidation of the U.S. companies will combine both organizations’ passion, resources and expertise to advance science and research with the goal of bringing cancer therapies to market more rapidly. Through scientific persistence and a commitment to discovery, SDP Oncology is focused on advancing a diverse and purposeful pipeline that aims to positively impact outcomes for patients with cancer. The company’s areas of concentration will address unmet needs in oncology and encompass discovery research, external innovation, clinical development and commercialization.

"The merger of Tolero Pharmaceuticals and Boston Biomedical, and the formation of Sumitomo Dainippon Pharma Oncology, will help us achieve our vision of establishing oncology as a core driver of our business," said Hiroshi Nomura, Representative Director, Chief Executive Officer, Sumitomo Dainippon Pharma. "Over the last decade, these formerly separate organizations have expanded our oncology pipeline to include multiple advanced assets and early discovery programs. We look forward to furthering our commitment to oncology globally through the efforts of Sumitomo Dainippon Pharma Oncology."

Patricia S. Andrews has been appointed Chief Executive Officer and Global Head of Oncology. Andrews previously spent seven years at Boston Biomedical and served as Chief Executive Officer for the past three years. The global leadership team includes David Bearss, Ph.D., Chief Scientific Officer and Global Head of Research, Edgar Braendle, M.D., Ph.D., Chief Medical Officer and Global Head of Development, and Masashi Murata, Ph.D., Chief Strategy Officer, Global Strategy and Planning.

"The launch of this organization represents an exciting new beginning, bringing together distinct proficiencies and strengths from each geography into one collaborative team," said Patricia S. Andrews, CEO and Global Head of Oncology. "We operate in a unique position within the oncology landscape because we have the nimbleness of a small company combined with the support and resources of a global parent company. Working together, we strengthen our ability to embrace risks and bring forward meaningful cancer therapies that have the potential to truly advance the treatment landscape."

SDP Oncology’s discovery, research and development programs will span multiple areas, including oncogenic pathways, survival mechanisms and novel protein interactions. The company’s robust portfolio of preclinical and advanced-stage assets is currently being investigated in multiple solid tumors and hematologic malignancies, including metastatic colorectal cancer, glioblastoma, myelofibrosis and myelodysplastic syndrome. Augmenting this effort, SDP Oncology’s Global Oncology External Innovation Hub enhances collaboration with external organizations, such as academia and venture capital, to propel meaningful research in drug discovery for oncology.

The establishment of SDP Oncology will foster collaboration between colleagues across geographies from Utah to Massachusetts to Japan, encouraging innovation focused on bringing forward breakthrough therapies.