On April 20, 2020 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported that the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application to initiate a Phase I/II trial for Precigen’s PRGN-2009, a first-in-class, off-the-shelf (OTS) investigational immunotherapy utilizing the AdenoVerse platform designed to activate the immune system to recognize and target HPV+ solid tumors(Press release, Precigen, APR 20, 2020, View Source [SID1234556432]). HPV+ cancers represent a significant health burden in indications such as head and neck, cervical, vaginal and anal cancer.

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The Phase I portion of the study will follow 3+3 dose escalation to evaluate the safety of PRGN-2009 administered as a monotherapy and to determine the recommended Phase II dose (R2PD) followed by an evaluation of the safety of the combination of PRGN-2009 at the R2PD and an investigational bifunctional fusion protein in patients with recurrent or metastatic HPV-associated cancers. The Phase II portion of the study will evaluate PRGN-2009 as a monotherapy or in combination with the bifunctional fusion protein in patients with newly-diagnosed stage II/III HPV16-positive oropharyngeal cancer.

PRGN-2009 leverages Precigen’s UltraVector and AdenoVerse platforms to optimize HPV antigen design in combination with its gorilla adenovector with a large payload capacity and the ability for repeat administration due to very low to non-existent seroprevalence in the human population.

PRGN-2009 is under development through a Cooperative Research and Development Agreement, or CRADA, within the laboratory of Dr. Jeffrey Schlom, Chief of the Laboratory of Tumor Immunology and Biology (LTIB), Center for Cancer Research (CCR), National Cancer Institute (NCI). This CRADA has allowed Precigen to rapidly and cost-effectively advance PRGN-2009 to the clinic. The Phase I/II clinical trial of PRGN-2009 will be conducted at the NIH Clinical Center and will be led by Dr. Julius Strauss, Co-Director of the LTIB’s Clinical Trials Group, and Dr. James Gulley, Chief of the Genitourinary Malignancies Branch, CCR, NCI.

"Globally, high-risk HPVs cause nearly 5% of all cancers, with about 570,000 women and 60,000 men diagnosed with HPV-related cancers each year," said Helen Sabzevari, PhD, President and CEO of Precigen. "We are incredibly proud of our continued relationship with NCI and the tremendous progress in bringing forward this novel asset class in such a short period of time. Advancements are critically needed to better target HPV+ tumors across multiple patient groups, and we have been encouraged by the promising preclinical data for PRGN-2009 in potentially targeting this patient population."

About HPV+ Cancers
HPV infects the squamous cells that line the inner surfaces of certain organs and, consequently, most HPV-related cancers are a type of cancer called squamous cell carcinoma. Some cervical cancers come from HPV infection of gland cells in the cervix and are referred to as adenocarcinomas.1 HPV-related cancers include cervical, oropharyngeal, anal, penile, vaginal, and vulvar.1 Nearly 44,000 HPV-associated cancers occur in the United States each year. Of these, approximately 25,000 occur in women and 19,000 occur in men.2 HPV is considered responsible for more than 90% of anal and cervical cancers, about 70% of vaginal and vulvar cancers, and more than 60% of penile cancers.2 Recent studies indicate that about 70% of cancers of the oropharynx also may be related to HPV.2

On April 20, 2020 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported that it will report its first-quarter 2020 financial results on Wednesday, April 29, 2020 after the financial markets close (Press release, Vertex Pharmaceuticals, APR 20, 2020, View Source [SID1234556433]). The company will host a conference call and webcast at 5:00 p.m. ET. To access the call, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International).

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The conference call will be webcast live and a link to the webcast can be accessed through Vertex’s website at www.vrtx.com in the "Investors" section. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company’s website.

On April 20, 2020 Zikani Therapeutics, a company dedicated to leveraging its unique TURBO-ZM platform to develop novel ribosome modulating agents (RMAs) for the treatment of rare, nonsense mutation-driven diseases, reported the close of a $7.5 million Series A-1 financing (Press release, Zikani Therapeutics, APR 20, 2020, View Source [SID1234558245]). Advent Life Sciences, Gurnet Point Capital and Roche Venture Fund supported the funding.

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"We’re pleased to support Zikani as an emerging company that is using the strength of its science and a refined strategy to develop novel ribosome modulators for rare diseases caused by nonsense mutations," said Raj Parekh of Advent Life Sciences.

Zikani will use the proceeds to advance its current nonsense mutation readthrough programs from lead optimization to candidate selection. The company has demonstrated positive, pre-clinical data in multiple disease areas, including: APC mutant colon cancer, familial adenomatous polyposis (FAP), class 1 cystic fibrosis, and recessive dystrophic and junctional epidermolysis bullosa (RDEB and JEB).

"Zikani has generated promising data that validates its TURBO-ZM platform and addresses a completely unmet need in oncology therapeutics: restoring expression of a major tumor suppressor gene," said Keith Flaherty, M.D., Director of Clinical Research at Massachusetts General Hospital Cancer Center and a senior advisor to Zikani.

Ribosomal RNAs form the translation machinery that generates function proteins from genetic sequencies. Ribosome modulation provides a therapeutic approach to addressing a number of diseases, but the development of disease-specific ribosome modulators has been a challenge. Zikani’s proprietary platform TURBO-ZM allows rapid synthesis of novel RMAs that can be optimized to target the human ribosome in a disease specific manner.

"We’re highly encouraged by the data we’ve generated to date and are working rapidly to advance compounds into pre-clinical development. This financing, particularly in this volatile financial market, is a vote of confidence for our promising technology platform. As we look ahead, our focus is to maximize the funding and take the steps necessary to generate greater recognition for RMAs and form collaborations with partners that will benefit from our scientific platform," said Sumit Aggarwal, President and CEO, Zikani Therapeutics.

On April 20, 2020 Genetron Holdings Limited ("Genetron Health"), a China-based precision oncology company that covers full-cycle cancer care, reported that 14 cancer research papers will be presented as posters at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2020 Annual Meeting, which will take place virtually on April 27-28 and June 22-24 (Press release, Genetron Health Technologies, APR 20, 2020, https://www.businesswire.com/news/home/20200420005389/en/Genetron-Health-Announces-Forthcoming-Unveiling-Cancer-Research [SID1234556434]).

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In collaboration with over 10 notable cancer research centers and clinical hospitals in China, including the Chinese PLA General Hospital (301 Hospital), Cancer Hospital of Chinese Academy of Medical Sciences, Beijing Tiantan Hospital of Capital Medical University, Genetron Health’s presentation will cover a broad spectrum of precision oncology topics such as molecular and immunological diagnostics, bio-informatics analysis in multiple cancer types including lung cancer, gastric cancer, central nervous system tumors and urinary system tumors.

Scientists at Genetron Health with a multi-disciplinary background have devoted significant resources to continuously optimize key diagnostics technologies including liquid biopsy, fusion detection, and algorithms for bioinformatics. They focus on improving the quality and efficiency of sample testing, as well as the comprehensiveness and stability of bioinformatics analysis. Genetron Health has accumulated clinically valuable data in the fields of diagnostics, neoadjuvant chemotherapy prognosis and efficacy evaluation, immunotherapy, and drug resistance to facilitate translation into clinical applications.

Genetron Health actively collaborates with clinicians to drive cancer genomics research. The Company has published over 20 papers in Nature Genetics, Cancer Cell, Nature Communications, PNAS, and other internationally recognized academic journals, accelerating the clinical translation and commercialization of scientific research and continously improving the reliability and accessibility of its products and technology. Genetron Health is committed to making available advanced and reliable precision medicine technology to the community.

About the AACR (Free AACR Whitepaper)

Founded in 1907, the American Association for Cancer Research (AACR) (Free AACR Whitepaper) is the first and largest cancer research organization dedicated to accelerating the conquest of cancer. It has grown from its earliest 11 members to over 47,000 members in 127 countries and territories. The mission of AACR (Free AACR Whitepaper) is to prevent and cure cancer through research, education, communication and collaboration. Through its programs and services, the AACR (Free AACR Whitepaper) fosters research in cancer and related biomedical science; accelerates the dissemination of new research findings among scientists and others dedicated to the conquest of cancer; promotes science education and training; and advances the understanding of cancer etiology, prevention, diagnosis, and treatment throughout the world.The AACR (Free AACR Whitepaper) Annual Meeting is one of the largest cancer research conferences in the world, attracting nearly 20,000 professionals from across the globe each year.

On April 20, 2020 McKesson, an independent specialty pharmacy for oncology and other rare and complex therapeutic areas, reported that it has been selected by Seattle Genetics as a specialty pharmacy provider for TUKYSATM (tucatinib) for the treatment of HER2-positive breast cancer (Press release, McKesson, APR 20, 2020, View Source [SID1234556435]).

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TUKYSA, approved by the FDA on April 17, 2020, is an orally bioavailable, small molecule tyrosine kinase inhibitor (TKI) that is highly selective for HER2, a growth factor receptor that is over-expressed in multiple cancers. This drug is indicated in combination with trastuzumab and capecitabine for treatment of adult patients with advanced unresectable or metastatic HER2-positive breast cancer, including patients with brain metastases, who have received one or more prior anti-HER2-based regimens in the metastatic setting. On October 21, 2019, Seattle Genetics announced positive topline results from the HER2CLIMB trial evaluating tucatinib. The trial compared this therapy used in combination with trastuzumab and capecitabine to trastuzumab and capecitabine alone in patients who have locally advanced unresectable or metastatic HER2-positive breast cancer.

"We’re proud that Seattle Genetics has entrusted us to dispense this important therapy option for patients with metastatic HER2-positive breast cancer," said Brandon Tom, vice president of Commercial Services at Biologics. "The addition of TUKYSA enables us to support providers across the various treatment options that exist for these patients."

Biologics specialty pharmacy is committed to and recognized for its high level of customer service as well as its innovative, high-touch and multidisciplinary patient-centric approach. Each team includes a pharmacist with in-depth knowledge of therapies, an experienced nurse, and a financial counselor who is familiar with various financial assistance programs and organizations that help patients. This highly-skilled care team works together to develop individualized care plans that address each patient’s unique clinical, financial and emotional needs and streamlines communication back to the treating provider, enabling high-quality care and differentiated outcomes. In addition, the Biologics team works closely with payers to ensure patients can access the specialty medications they need.

Physicians may submit prescriptions to Biologics via phone (800.850.4306), fax (800.823.4506) or eScribe. For electronic prescribing systems, physicians may search for Biologics within their EMR system.