On December 4, 2019 Servier Group and Servier Pharmaceuticals, the U.S. subsidiary of the global French pharmaceutical company, reported that data from multiple studies will be presented at the 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Orlando, from December 7-10, 2019 (Press release, Servier, DEC 4, 2019, View Source [SID1234551937]).

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"As a patient-centric, innovation-driven organization, we believe it is critical to foster scientific exchange and discussion around novel approaches to treating hematologic-oncologic conditions and addressing unmet needs," said Claude Bertrand, Executive Vice President of Research and Development, Servier Group. "Oncology is one of Servier’s strategic priorities, and these new data findings to be presented at ASH (Free ASH Whitepaper) 2019 demonstrate our expanding pipeline of drug candidates, including allogeneic CAR-T therapy.*"

A complete listing of this year’s Servier abstracts* are available on the ASH (Free ASH Whitepaper) website. Notable abstracts include:

Sunday, December 8, 6:00 PM; Orange County Convention Center, Hall B

Allogeneic Anti-CD19 CAR T Cells Manufactured from Healthy Donors Provide a Unique Cellular Product with Distinct Phenotypic Characteristics Compared to CAR T Cells Generated from Patients with Mature B Cell Malignancies
Poster presentation in collaboration with Allogene Therapeutics
Session: 703. Adoptive Immunotherapy: Mechanisms and New Approaches: Poster II
Abstract: 3228
Preclinical Activity of the MPS1 Inhibitor S81694 in Acute Lymphoblastic Leukemia (ALL)
Session: Acute Lymphoblastic Leukemia: Therapy, excluding Transplantation: Poster II
Abstract: 2631
Monday, December 9, 2019, 6:00 PM; Orange County Convention Center, Hall B

Initial findings of the Phase 1 Trial of PBCAR0191, a CD19 Targeted Allogeneic CAR-T Cell Therapy
Poster presentation in collaboration with Precision BioSciences
Session: 627. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas) – Results from Retrospective/Observational Studies: Poster III
Abstract: 4107
Additionally, on Friday, December 6, from 12:30 p.m. to 4:30 p.m., Medscape has a Continued Medical Education Program (CME) entitled "New Data and Paradigms in ALL: Insights and Applications." This CME event will take place at the Hyatt Regency Orlando Hotel, 9801 International Drive, Room Plaza International I-K. ASH (Free ASH Whitepaper) attendees can register here to attend. This CME event is supported by an independent educational grant from Servier, and all content discussed and presented is developed independently of commercial interests by Medscape’s Education staff.

"Since opening our U.S. headquarters in 2018, we have remained squarely focused on advancing and launching innovative treatment options for patients in need and are agnostic to where that innovation comes from," said David K. Lee, Chief Executive Officer, Servier Pharmaceuticals. "The presence of Servier Pharmaceuticals and our strategic partners at ASH (Free ASH Whitepaper) 2019 further exemplifies our commitment and dedication to developing new medicines in a way that’s most meaningful for patients."

More than 40 percent† of Servier Group’s investments in R&D are devoted to fighting cancer. Servier’s research in oncology focuses on therapies aimed at restoring programmed cell death (apoptosis) in cancer cells and at mobilizing the immune system against cancer cells (immuno-oncology).

To further accelerate the research and development of innovative treatments, Servier Group has instituted a policy of open innovation and has established more than 50 strategic partnerships. Partnership models are uniquely tailored to the engagement and span industry and academia.

In oncology, 13 drugs are currently being developed as an outgrowth of this collaborative research approach. These drugs target different types of lymphomas and leukemia, and other solid tumors, including gastrointestinal and lung. These drugs are investigational and have not yet been reviewed by the U.S. Food and Drug Administration (FDA) for safety and efficacy.

To learn more about Servier and their R&D efforts visit booth #955 in the Exhibition Hall.

About UCART19
UCART19 is being jointly developed under a clinical development collaboration between Servier and Allogene based on an exclusive license granted by Cellectis to Servier. UCART19 utilizes TALEN gene-editing technology pioneered and owned by Cellectis. Servier grants to Allogene exclusive rights to UCART19 in the U.S. while Servier retains exclusive rights for all other countries.

About PBCAR0191
In partnership with Servier, Precision BioSciences is responsible for early-stage research activities, manufacturing and Phase 1 execution for PBCAR0191. Servier has exclusive worldwide rights to opt-in for late-stage development and commercialization, and Precision has the right to participate in the development and commercialization of any licensed products resulting from the collaboration through a 50/50 co-development and co-promotion option in the U.S.

About Servier in the U.S.
Servier Pharmaceuticals LLC is a commercial-stage pharmaceuticals company with a passion for innovation and improving the lives of patients and their families and caregivers. With 13 additional oncology projects currently in clinical development, Servier is the partner of choice for leading academic institutions and a growing number of pharmaceutical companies. Servier BioInnovation is a joint initiative between Servier Group R&D and Global Business Development for external innovation. The initiative is focused on identifying new early-stage R&D opportunities and expediting business development and licensing activities in the U.S. Learn more at www.servier.us.

On December 4, 2019 Ardelyx, Inc. (Nasdaq: ARDX), a specialized biopharmaceutical company focused on developing innovative first-in-class medicines to improve treatment for people with cardiorenal diseases, reported the pricing of an underwritten public offering of 20,000,000 shares of its common stock at a public offering price of $6.25 per share, before underwriting discounts and commissions, for gross proceeds of $125,000,000 (Press release, Ardelyx, DEC 4, 2019, View Source [SID1234551936]). In addition, Ardelyx has granted the underwriters of the offering the right for a period of 30 days to purchase up to an additional 3,000,000 shares of common stock at the public offering price, less underwriting discounts and commissions. The offering is expected to close on or about December 9, 2019, subject to customary closing conditions.

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Ardelyx currently expects to use its existing cash, cash equivalents and short-term investments and the net proceeds from the offering to support its activities for the clinical development and potential commercialization of tenapanor for the treatment of hyperphosphatemia in patients with chronic kidney disease, or CKD, on dialysis, for regulatory activities to support the submission of a New Drug Application, in such indication, as well as for clinical trials and nonclinical studies for other product candidates, and for general corporate purposes and working capital.

Citigroup, Cowen, SVB Leerink and Piper Jaffray are acting as joint book-running managers for the offering.

A registration statement on Form S-3 (File No. 333-217441) relating to these securities has been filed with the U.S. Securities and Exchange Commission (SEC) and became effective on May 4, 2017. The offering is being made only by means of a written prospectus and prospectus supplement that form a part of the registration statement, copies of which may be obtained when available by contacting Citigroup Global Markets Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (800) 831-9146; Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY,11717, Attn: Prospectus Department, by email at [email protected] or by telephone at (833) 297-2926; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6132 or by e-mail at [email protected]; or Piper Jaffray & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, or by telephone at 800-747-3924, or by e-mail at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On December 4, 2019 Charles River Laboratories International, Inc. (NYSE: CRL) reported updates to its online Tumor Model Compendium. Charles River’s cancer model database provides access to a comprehensive collection of well-established tumor models for early-stage oncology research (Press release, Charles River Laboratories, DEC 4, 2019, View Source [SID1234551935]).

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This update adds 79 new patient-derived xenografts (PDX), 10 new cell line-derived xenografts (CDX), and 36 new cell line (CL) models for several cancer types including: anal, colon, lung and renal cancer as well as leukemia and melanoma. Additionally, the update provides an increased depth of data for both whole exome sequencing (WES) and gene expression, with greater bioinformatics coverage:

71 new WES data sets, increasing whole exome data to 90 percent of models
92 new RNA-Seq data sets, increasing gene expression data to 87 percent of models
Charles River’s Compendium includes tumor models from a wide range of tumor subtypes for both in vivo and in vitro oncology research, including patient-derived xenografts (PDXs), cell line-derived xenografts (CDX), and syngeneic models.

This resource allows researchers to easily identify the most appropriate tumor models, leading to a more targeted study design from the start, saving time and money on the path to the clinic. The Compendium, when combined with Charles River’s robust portfolio of oncology services and scientific background, provides a highly translational platform for developing safe, effective oncology therapeutics.

Expansion of Oncology Portfolio
Charles River recently launched in vivo tumor microdialysis to support cancer research. Microdialysis is a uniquely sensitive and powerful method of sampling the tumor microenvironment of research models. With microdialysis, researchers are able to gather actionable data on the mechanism of action and perform more efficient decision making. Traditionally applied to neuroscience research, utilizing microdialysis in tumor models provides an excellent approach to model the pharmacokinetics of a given therapeutic compound.

Charles River has also developed two new research models specifically for the study of Graft vs Host Disease (GvHD). GvHD and other pharmacodynamic models can act as screening tools to evaluate the ability of immunoregulatory drugs to modulate disease progression, providing valuable information to progress the best version of a drug past preclinical testing.

Additionally, Charles River recently launched an expansion of its PDX portfolio in North America. Benefiting from over 30 years of experience, Charles River’s global PDX capabilities provide clients with the scientific guidance necessary to progress novel cancer therapeutics. To learn more about Charles River’s oncology research capabilities, visit www.criver.com.

Approved Quotes

"In oncology, we recognize the importance of identifying a model that will most closely mimic human disease, increasing translation from preclinical research to the clinic. An increased depth of data and additional model types provide an improved resource for making those critical decisions." – Birgit Girshick, Corporate Executive Vice President, Discovery & Safety Assessment, Biologics Testing Solutions, and Avian Vaccine Services at Charles River
"The application of microdialysis in oncology research provides a unique opportunity for researchers to detect and quantify multiple immunomodulators and oncometabolites from freely moving, tumor-bearing models. This insight into target engagement and mechanism of action aids faster progression into the clinic." – Arash Rassoulpour, PhD, Executive Director at Charles River
"Stem cell transplantation, while offering many anti-cancer benefits, has a high risk of complications due to the introduction of donor cells into a patient’s immune system. GvHD models provide a valuable tool to study the biology of this disease and improve success rates of stem cell transplantation as a cancer treatment." – Chassidy Hall, Site Director, Oncology Center of Excellence at Charles River

On December 4, 2019 BIOASIS TECHNOLOGIES INC. (OTCQB:BIOAF; TSX.V:BTI), ), a pre-clinical, research-stage biopharmaceutical company developing its proprietary xB3 platform technology for the delivery of therapeutics across the blood-brain barrier (the "BBB") and the treatment of central nervous system ("CNS") disorders in areas of high unmet medical need, including brain cancers and neurodegenerative diseases, reported the creation of the Medical Oncology Advisory Board and the appointments of Dr. John DeGroot M.D., Dr. Hope Rugo M.D. and Dr. Javier Cortés M.D. PhD (Press release, biOasis, DEC 4, 2019, View Source [SID1234551934]). The goals of this board will be to guide the clinical strategy and design of the xB3-001 clinical program for the treatment of HER2+ breast cancer and brain metastases. The Medical Oncology Advisory Board will be chaired by José Iglesias, MD, who has been appointed consultant Chief Medical Officer for Bioasis Technologies Inc.

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Dr. José Iglesias, Chairperson of the Advisory Board in his role of consultant Chief Medical Officer is a biopharmaceutical industry veteran with 30 years of executive experience in all phases of oncology clinical drug development covering the areas of solid tumor oncology, immuno-oncology and translational medicine. His career includes senior positions at Eli Lilly, Amgen, Abraxis Bioscience, Celgene, Bionomics, Biothera, Apobiologix and Boston Biomedical. Dr. Iglesias is author or co-author of more than 60 peer-reviewed publications with over 6,000 literature citations and an active member of ASCO (Free ASCO Whitepaper), ESMO (Free ESMO Whitepaper) and AACR (Free AACR Whitepaper). He graduated from the University of Montevideo, Uruguay, and received additional training at the Weizmann Institute of Science (Rehovot, Israel), Duke University Medical Center (Durham, NC) and the University of Toronto.

"I am delighted to welcome José to the Bioasis team, strengthening our oncology development expertise at a critical time as we progress our lead program xB3-001 for the treatment of HER2+ breast cancer and brain metastases. I am also extremely pleased to welcome John, Hope and Javier to the Medical Oncology Advisory Board. Each member is a leader in their field with proven track records of excellence in clinical practice and clinical drug development in the areas of breast cancer and CNS oncology." Deborah Rathjen added, Ph.D., Executive Chair of Bioasis. "We look forward to continuing to benefit from the strength of the collective experience of our medical board as we advance our xB3 programs toward the clinic."

Dr. José Iglesias commented, "Treatment of HER2+ breast cancer that has metastasized to the brain remains a significant challenge. To date, xB³-001 has demonstrated highly encouraging preclinical results, and I am excited to be working with Dr. Rathjen and the Bioasis team to continue to advance this class-leading technology."

Dr. John de Groot is a Professor, and Chairman ad interim, in the Department of Neuro-Oncology at The University of Texas MD Anderson Cancer Center. He is an expert in the fields of glioma angiogenesis and molecularly targeted therapy. Dr. de Groot has served as the principal investigator (PI) or co-investigator on multiple funded National Cancer Institute, foundation, and industry-sponsored grants. He is the principal investigator of numerous clinical trials involving novel agents being tested in patients with glioblastoma and is a leader of MD Anderson’s Glioblastoma Moon Shot. Dr. de Groot has over 112 peer reviewed articles. He is or has been a peer reviewer for 23 scientific journals, both national & international, and is on four editorial review boards. He completed his medical education at The University of Texas Medical Branch at Galveston, and pursued internship and residency at The Johns Hopkins School of Medicine.

Dr. Hope S. Rugo is Professor of Medicine at the University of California San Francisco (UCSF) Helen Diller Family Comprehensive Cancer Center, where she is also the director of Breast Oncology and Clinical Trials Education. She is a principal investigator of multiple clinical trials focusing on combining novel targeted therapeutics with standard treatment to improve the treatment of both early and late stage breast cancer and improving safety with targeted agents and has published widely in this area with over 200 peer reviewed articles. Dr. Rugo is an active member of the Translational Breast Cancer Research Consortium (TBCRC), a principal investigator of several TBCRC trials and is the co-chair of the Triple Negative Working Group. She is a member of the Breast Committee for the Alliance cooperative group, and is an associate editor for the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting educational book. Dr. Rugo graduated from the University of Pennsylvania School of Medicine. She completed a residency in internal medicine followed by a fellowship in hematology and oncology at UCSF as well as a research fellowship at Stanford University.

From June 2003 to July 2015, Dr.Cortés worked in the Department of Medical Oncology at the Hospital Vall d’Hebron, Barcelona, where he has been Coordinator of the Teaching and Training Programme for Residents in Oncology and Senior Specialist in the Area of Breast Cancer with a special interest in new drug development. Dr. Cortés was the Head of the Breast Cancer Program and the Melanoma Unit from July 2006 to August 2015. From September 2015 to October 2018, he has been Head of the Breast Cancer and Gynecological tumors at Ramon y Cajal University Hospital in Madrid. He is Clinical Investigator of the Breast Cancer Research Program at Vall d’Hebron Institute of Oncology, and Head of Breast Cancer Program, IOB Institute of Oncology, Madrid and Barcelona, Spain. Dr Cortés is the author of more than 240 publications, with a focus on breast tumors and new drugs, and more than 500 communications at multiple conferences. He actively participates in the development of numerous national and international clinical investigations. Dr Cortés received a degree in Medicine and Surgery from the Universidad Autónoma de Madrid with continued studies at the University of Navarra, specializing in Medical Oncology at the Clínica Universitaria de Navarra. In addition to his medical specialties, he has two masters’ degree in Medical Direction and Clinical Management from the Universidad Nacional de Educación a Distancia (UNED) and Research Methodology in Health Sciences from the Universidad Autónoma de Barcelona as well as a degree in Statistics in Health Sciences from the same university.

On December 4, 2019 ImmunityBio, a privately held immunotherapy company, reported that it has received Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA) for its interleukin-15 (IL-15) agonist complex, N-803, in combination with Bacillus Calmette-Guerin (BCG), for the treatment of patients with BCG-unresponsive non-muscle invasive bladder carcinoma in situ (CIS) (Press release, ImmunityBio, DEC 4, 2019, View Source [SID1234551933]).

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This grant of Breakthrough Designation follows on the receipt of Fast Track Designation from the U.S. Food and Drug Administration for N-803 in 2017. The FDA’s Breakthrough Therapy Designation program is designed to expedite the development and review of drugs intended to treat serious conditions and fill unmet medical needs.1 The FDA published guidance in February 2018 to address BCG unresponsive non-muscle invasive bladder cancer (NMIBC), stating that the goal of therapy in patients with BCG-unresponsive NMIBC is to avoid cystectomy.

"We are pleased that the FDA has granted Breakthrough Therapy Designation to N-803 in combination with BCG for the treatment of patients with Non-Muscle Invasive Bladder Cancer with CIS," said Dr. Patrick Soon-Shiong, Chairman and CEO of ImmunityBio. "We look forward to accelerating our work on N-803 to bring greater hope to the thousands of people living with the serious consequences of bladder cancer and to address the unmet need to avoid surgical removal of the bladder in these high-risk patients. Patients who fail current standard of care and who have high risk carcinoma in situ of the bladder are left with a difficult choice of having their bladder removed, a procedure fraught with a clinically significant mortality and morbidity rates. The potential opportunity to avoid this procedure and change the course of this disease with the addition of N-803 is immensely gratifying," he said.

"The favorable safety profile and initial efficacy results from our phase 1 study strongly suggest we may be able to improve the treatment for a disease that has not had non-surgical treatment options for over 30 years," said ImmunityBio Chief Medical Officer, Dr. John Lee.

ImmunityBio’s N-803, an IL-15 Superagonist

The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of the natural killer (NK) and T cells. N-803 is a novel IL-15 superagonist complex consisting of an IL-15 mutant (IL-15N72D) bound to an IL-15 receptor α/IgG1 Fc fusion protein. N-803 has improved pharmacokinetic properties, longer persistence in lymphoid tissues and enhanced anti-tumor activity compared to native, non-complexed IL-15 in vivo.

N-803 is currently being evaluated for adult patients in two clinical NMIBC trials. QUILT 2.005 is investigating use of N-803 in combination with BCG for patients with BCG-naïve NMIBC; QUILT 3.032 is studying N-803 in combination with BCG in patients with BCG-unresponsive NMIBC.

The Urgent, Unmet Need to Treat NMIBC and Avoid Cystectomy

Bladder cancer has a high incidence worldwide, with 199,922 deaths and an estimated 549,393 new cases in 2018.2 In the United States, bladder cancer is the fourth most commonly diagnosed solid malignancy in men and the twelfth for women; The American Cancer Society estimates 80,470 new cases and 17,670 deaths in 2019.3 Bladder cancers are described based on how far they have invaded into the wall of the bladder. NMIBC occurs when the cancer has not grown into the main muscle layer of the bladder. Approximately 75-85% of all newly diagnosed cases of bladder cancer are non-muscle invasive bladder cancer (NMIBC).4

For the last 30 years, BCG immunotherapy has been the standard for treating NMIBC. However, disease recurrence and progression rates remain unacceptably high. Standard of care recommendations for these patients include lifetime invasive surveillance and rapid treatment of recurrences, creating a substantial financial burden and drastic impact on quality of life. Of those patients who experience recurrence, approximately 30% will progress and succumb to their disease over a 15-year period, and another 50% will undergo radical cystectomy of the bladder in an attempt to control their disease.5

For high-risk NMIBC patients who are BCG-unresponsive with persistent or recurrent disease, treatment guidelines recommend a surgical procedure called radical cystectomy, a surgery to remove the entire bladder that may require removal of other surrounding organs. In men, removal of the prostate may be necessary, and in women, surgeons may also remove the uterus, fallopian tubes, ovaries and cervix, and occasionally a portion of the vagina. Despite the advent of minimally invasive procedures and robotic techniques, the 90-day mortality and morbidity rates in patients who undergo cystectomy remain unacceptably high at 5.1-8.1% and 28-64%, respectively.6 Based on this urgent need, FDA published guidance in February 2018 to address BCG unresponsive non-muscle invasive bladder cancer (NMIBC), stating that the goal of therapy in patients with BCG-unresponsive NMIBC is to avoid cystectomy.