On January 19, 2021 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company dedicated to improving life with its wholly proprietary ARCUS genome editing platform, reported that the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application for PBCAR19B, a next-generation, stealth cell, CD19 product candidate for patients with relapsed/refractory (R/R) Non-Hodgkin Lymphoma (NHL) (Press release, Precision Biosciences, JAN 19, 2021, View Source [SID1234576682]).

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"We are pleased to receive IND clearance for PBCAR19B, which has shown in preclinical studies, to delay both T cell and natural killer cell-mediated allogeneic rejection," said Matt Kane, Chief Executive Officer of Precision BioSciences. "We believe that the ability to reduce rejection by both cell types holds potential for improved persistence of allogeneic CAR T cells and that, by bringing PBCAR19B into the clinic while continuing to develop our lead allogenic candidate PBCAR0191, we have two opportunities to achieve our goal of producing deep and durable clinical responses."

The Phase 1 study will be a non-randomized, open-label, single-dose, dose-escalation and dose-expansion study deigned to evaluate the safety and clinical activity of PBCAR19B at increasing flat dose levels (2.7 x 108 – 8.1 x 108 CAR T cells) in patients with R/R NHL. The primary objective of the study is to identify the maximum tolerated dose and any dose-limiting toxicities.

About PBCAR19B
PBCAR19B is a next-generation, stealth cell candidate for patients with CD19-positive malignancies such as relapsed/refractory Non-Hodgkin Lymphoma. PBCAR19B is designed to improve the persistence of allogeneic CAR T cells following infusion by preventing rejection by T cells and natural killer (NK) cells. In addition to the CAR gene, the stealth vector carries a short hairpin RNA (shRNA) that suppresses expression of beta-2 microglobulin (B2M), a component of Class 1 major histocompatibility complex (MHC) molecules found on the cell surface. Reducing or knocking down Class 1 expression on allogeneic CAR T cells has been shown to reduce CAR T cell killing by cytotoxic T cells. The stealth vector also carries a HLA-E gene intended to prevent rejection of CAR T cells by NK cells that can be stimulated as a result of reduced MHC molecule expression on the cell surface.

On January 19, 2021 In an announcement that will help more people with advanced prostate and pancreatic cancers in Japan, Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, reported that it will receive reimbursement for its BRACAnalysis Diagnostic System, which helps physicians determine whether certain patients will qualify and potentially benefit from treatment with the PARP inhibitor, Lynparza (olaparib) (Press release, Myriad Genetics, JAN 19, 2021, View Source [SID1234574095]). BRACAnalysis was approved by Japan’s Ministry of Health, Labour and Welfare (MHLW) in October 2020 as a companion diagnostic for these indications and the reimbursement decision is now in effect.

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"BRACAnalysis continues to be the gold standard for germline BRCA1 and BRCA2 testing around the globe," said Nicole Lambert, president of Myriad Genetic Laboratories. "Our goal is to continue to expand patient access to the proven benefits of genetic testing and help physicians combat even the most difficult to treat cancers."

Myriad estimates there are more than 78,000 cases of prostate cancer and 40,000 cases of pancreatic cancer per year in Japan. With the reimbursement decision, approximately 20,000 patients in Japan will now qualify to receive genetic testing. BRACAnalysis was previously approved in Japan to identify patients with ovarian and breast cancer who have a germline BRCA mutation and are eligible for treatment with Lynparza. BRACAnalysis is the only germline test for BRCA1 and BRCA2 mutations to receive regulatory approval in Japan.

"Now that BRACAnalysis is available in Japan to assess the presence of BRCA1 or BRCA2 in deleterious or suspected deleterious mutations, more patients with prostate and pancreatic cancer will have access to advanced options for treatment," said Hiroji Uemura, M.D., Ph.D., Professor, Department of Urology and Renal Transplantation, Yokohama City University Medical Center.

"As physicians, we are excited to use BRACAnalysis to identify those patients who may need those advanced treatments, and to continue improving health outcomes in the fight against those cancers," added Hideki Ueno, M.D., Ph.D., Medical Director, Department of Hepatobiliary and Pancreatic Oncology, National Cancer Center Hospital."

Myriad has been collaborating with AstraZeneca since 2007 on the development of companion diagnostics for Lynparza. Lynparza is a trademark of AstraZeneca. Lynparza is jointly developed and commercialized by AstraZeneca and MSD (known as Merck & Co., Inc. in the United States and Canada). Additionally, Myriad has partnered with SRL Inc., a subsidiary of H.U. Group Holdings, Inc. to expand the commercialization of the BRACAnalysis Diagnostic System in Japan.

According to World Cancer Research Fund, pancreatic cancer is the 12th most common cancer worldwide and Japan ranks third in the world for highest rates of incidence, according to the World Health Organization. Additionally, in Japan, prostate cancer is estimated to account for 18% of cancers diagnosed in men, according to the International Agency for Research on Cancer.

About the BRACAnalysis Diagnostic System
BRACAnalysis is a diagnostic system that classifies a patient’s clinically significant variants (DNA sequence variations) in the germline BRCA1 and BRCA2 genes. Variants are classified into one of the five categories; "Deleterious," "Suspected Deleterious," "Variant of Uncertain Significance," "Favor Polymorphism," or "Polymorphism." Once the classification is completed, the results are sent to medical personnel in Japan for determining the eligibility of patients for treatment with Lynparza.

About SRL
Since the establishment in 1970, SRL, Inc., a member of the H.U. Group Holdings, Inc., Japan-based leading healthcare group, has been providing comprehensive testing services as the largest commercial clinical laboratory in Japan. SRL carries out nearly 400,000,000 tests per year, covering a wide range of testing services including general/emergency testing, esoteric/research testing, companion diagnostics tests, genomic analysis, and etc. For more information, please visit View Source

On January 19, 2021 Syros Pharmaceuticals (NASDAQ: SYRS), a leader in the development of medicines that control the expression of genes, reported that it has priced an underwritten public offering of 5,400,000 shares of common stock at a public offering price of $14.00 per share, which would result in gross proceeds of approximately $75.6 million, before underwriting discounts and commissions (Press release, Syros Pharmaceuticals, JAN 19, 2021, View Source [SID1234574111]). The proceeds of the offering are expected to be used to fund the development of Syros’ ongoing clinical and preclinical programs, and for working capital and other general corporate purposes. All shares are being offered by Syros. Closing of the offering is expected to occur on or about January 22, 2021, subject to customary closing conditions. Syros has also granted the underwriters a 30-day option to purchase up to an additional 810,000 shares of common stock offered in the public offering on the same terms and conditions.

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Cowen and Piper Sandler & Co. are acting as joint book-running managers for the offering. JMP Securities and Oppenheimer & Co. are acting as lead managers and Roth Capital Partners and Brookline Capital Markets, a division of Arcadia Securities, LLC, are acting as co-managers.

The offering is being made pursuant to a shelf registration statement that was filed with the Securities and Exchange Commission ("SEC") on June 12, 2020 and declared effective by the SEC on June 22, 2020. The offering will be made only by means of the prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement relating to, and describing the terms of, the offering has been filed with the SEC and is available on the SEC’s web site at www.sec.gov.

The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC. Copies of the final prospectus supplement and the accompanying prospectus relating to this offering, when available, can be obtained from Cowen and Company, LLC, c/o Broadridge Financial Services, 1155 Long Island Avenue, Edgewood, NY 11717, Attention: Prospectus Department, or by telephone at 833-297-2926; or from Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, or by telephone at 800-747-3924.

This press release does not constitute an offer to sell or the 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 January 19, 2021 Lixte Biotechnology Holdings, Inc. (NASDAQ: LIXT), a clinical-stage drug discovery company developing pharmacologically active drugs for use in cancer treatment, reported an agreement on a Phase 1b clinical trial with City of Hope, a world-renowned independent cancer research and treatment center (Press release, Lixte Biotechnology, JAN 19, 2021, View Source [SID1234574205]). The trial will assess the combination of Lixte’s first-in-class protein phosphatase inhibitor LB-100 with a standard regimen for untreated, extensive stage-disease small cell lung cancer (ED-SCLC).

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LB-100 will be given in combination with carboplatin, etoposide and atezolizumab, an FDA approved but marginally effective regimen, in previously untreated ED-SCLC. The dose of LB-100 will be escalated with fixed doses of the 3-drug regimen to reach a recommended Phase 2 dose (RP2D). Patient entry will then be expanded so that a total of 12 patients will be evaluable at the RP2D to confirm its safety and to look for objective evidence of potential therapeutic activity as assessed by objective response rate, duration of overall response, progression-free-survival and overall survival.

John S. Kovach, M.D., Lixte founder and chief executive officer, said "Small cell lung cancer (SCLC) comprises approximately 15% of all lung cancers worldwide with about 30,000 new cases annually in the US. Although this very aggressive neuroendocrine tumor is much more sensitive to cytotoxic chemotherapy and radiation than the most common type of lung cancer, SCLC patients soon relapse after treatment and have a dismal prognosis. Recently, the addition of an immune blocker, atezolizumab, to carboplatin plus etoposide showed the first significant but modest improvement in median progression free survival from 4.3 to 5.2 months, and in median overall survival from 10.3 to 12.3 months."

Kovach continued, "In animal models, LB-100 significantly enhances the antitumor activity of cytotoxic chemotherapy in general, and in particular, the combination of carboplatin and etoposide against SCLC cells without enhancing toxicity. Mice models may not be predictive for humans, but if the extensive preclinical data showing LB-100 increases the effectiveness of chemotherapy applies to patients, we should see clear evidence of therapeutic benefit even in this early trial."

Ravi Salgia, M.D., Ph.D., the trial’s principal investigator and City of Hope’s Arthur & Rosalie Kaplan Chair in Medical Oncology, said, "City of Hope is in constant pursuit of more effective therapies for its cancer patients, and this therapeutic combination could be one for patients with small cell lung cancer, which is currently difficult to treat, particularly after a patient has relapsed. We look forward to working with Lixte on this important trial that we hope will make more therapeutic options available for patients with small cell lung cancer."

On January 19, 2021 Advaxis, Inc. (NASDAQ: ADXS), a clinical-stage biotechnology company focused on the development and commercialization of immunotherapy products, reported that the Company has received the first milestone payment related to its licensing agreement for ADXS31-164, now known as OST-HER2, to OS Therapies for evaluation in the treatment of osteosarcoma in humans (Press release, Advaxis, JAN 19, 2021, View Source [SID1234574096]).

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Under the terms of the amended and restated license agreement, OS Therapies, in collaboration with the Children’s Oncology Group (COG)1, is responsible for the conduct and funding of a clinical study evaluating OST-HER2 in recurrent, completely resected osteosarcoma. OS Therapies secured funding to support this research, triggering the first milestone payment. Under the agreement, Advaxis will receive additional clinical, regulatory, and sales-based milestone payments as well as royalties on future product sales. Additional details of the financial terms have not been disclosed.

"We are pleased that OS Therapies is now in position to initiate human clinical trials of OST-HER2 in osteosarcoma," said Kenneth A. Berlin, President and Chief Executive Officer of Advaxis. "ADXS31-164, which had been conditionally approved in the U.S. for the adjuvant treatment of osteosarcoma in canines, has the potential to provide a new treatment option for human osteosarcoma patients, particularly in children with this challenging disease for which there are few therapeutic options. As Advaxis is currently focused on developing our ADXS-HOT neoantigen-directed off-the-shelf therapeutics, with recent encouraging data in non-small cell lung cancer, we hope that OS Therapies can successfully explore the clinical potential of OST-HER2 in osteosarcoma, building on an earlier Phase 1 clinical trial performed by us where ADXS31-164 was safe and tolerable in humans."

Paul Romness, Chief Executive Officer of OS Therapies, said, "The OS Therapies mission is to develop and commercialize new therapeutics for the treatment of osteosarcoma, a deadly and extremely underserved pediatric cancer. We are excited to be in position to evaluate OST-HER2 in this indication, as it has been safe and effective in treating osteosarcoma in dogs, a natural model that is the similar to humans, and to be safe and tolerable in humans. Our goal is to stimulate the immune system to specifically target tumor dysplastic cells that express HER2, which is a common genetic mutation found in osteosarcoma. We believe that targeting Osteosarcoma and other solid tumors with OST-HER2 holds potential to impact the treatment paradigm."

Osteosarcoma is an aggressive cancerous tumor that forms in bone. Although it is rare, osteosarcoma is the most common type of bone cancer, and is most frequently found in children and young adults. Current treatment options are limited and there have been no new treatment options in more than thirty years.

1 The Children’s Oncology Group (www.childrensoncologygroup.org), a member of the NCI National Clinical Trials Network (NCTN), is the world’s largest organization devoted exclusively to childhood and adolescent cancer research with over 10,000 experts worldwide working in over 200 COG member institutions. COG’s mission is to improve the cure rate and outcome for all children with cancer.