On January 19, 2021 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported its corporate partners, Alphamab Oncology and 3D Medicines, received notification that the New Drug Application (NDA) for envafolimab was granted priority review by the Center for Drug Evaluation of the National Medical Products Administration (NMPA) in the indication of MSI-H/dMMR cancer (Press release, Tracon Pharmaceuticals, JAN 19, 2021, View Source [SID1234574099]).

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"We congratulate our partners on the acceptance of the initial regulatory submission for envafolimab in China for priority review, which marks another important milestone in the development and potential commercialization of this promising program," said Charles Theuer, M.D., Ph.D., TRACON Chief Executive Officer. "In addition to the registration trial in MSI-H/dMMR advanced solid tumors in China, envafolimab is being studied in two other registration trials, a randomized Phase 3 trial in biliary tract cancer in China being conducted by Alphamab and 3D Medicines, and our ENVASARC trial in sarcoma in the U.S., which has now dosed multiple patients."

About Priority Review by the NMPA

Priority review is a procedure established to encourage the research and development of new drugs and accelerate the review and approval of new drugs with obvious clinical value and urgent clinical needs. According to the new "Drug Registration Rules" (SAMR Order No.27) and Working Procedures for Priority Review and Approval of Drug Marketing Authorization (Interim) (No. 82 of 2020) implemented on July 1, and July 7, 2020, respectively, once granted Priority Review, the NMPA will prioritize the review process and resources for applications with expected shorter review timelines.

About Envafolimab (KN035)

Envafolimab (KN035), a novel, single-domain antibody against PD-L1, is the first subcutaneously injected PD-(L)1 inhibitor to be studied in registration trials. Envafolimab is currently being studied in the ENVASARC Phase 2 registration trial in the U.S. sponsored by TRACON, as well as in a Phase 2 registration trial as a single agent in MSI-H/dMMR advanced solid tumor patients and a Phase 3 registration trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China sponsored by TRACON’s corporate partners, Alphamab Oncology and 3D Medicines. Alphamab Oncology and 3D Medicines submitted an NDA to the NMPA in China for envafolimab in MSI-H/dMMR cancer that was accepted for review in December 2020 and granted priority review in January 2021. In the Phase 2 registration trial, the confirmed objective response rate (ORR) by blinded independent central review in MSI-H/dMMR colorectal cancer (CRC) patients treated with envafolimab who failed a fluoropyrimidine, oxaliplatin and irinotecan was 32%, which was similar to the 28% confirmed ORR reported in the Opdivo package insert in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin, and irinotecan and the 33% confirmed ORR reported for Keytruda in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin and irinotecan in cohort A of KEYNOTE-164.

About ENVASARC (NCT04480502)

The ENVASARC registration trial is a multi-center, open-label, randomized, non-comparative, parallel cohort study at approximately 25 top cancer centers in the United States that began dosing in December 2020. TRACON expects the trial to enroll 160 patients with UPS or MFS who have progressed following one or two lines of prior treatment and have not received an immune checkpoint inhibitor, with 80 patients enrolled into cohort A of treatment with single agent envafolimab and 80 patients enrolled in cohort B of treatment with envafolimab and Yervoy. The primary endpoint is ORR by blinded independent central review with duration of response a key secondary endpoint.

On January 19, 2021 Molecular Templates, Inc. (Nasdaq: MTEM, "Molecular Templates," "MTEM" or "the Company"), a clinical-stage biopharmaceutical company focused on the discovery and development of proprietary targeted biologic therapeutics, engineered toxin bodies (ETBs), reported that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for MT-6402, a next-generation ETB targeting PD-L1 that is enabled with MTEM’s antigen seeding technology (AST) (Press release, Molecular Templates, JAN 19, 2021, View Source [SID1234574098]). ETBs enabled with AST have dual mechanisms of action that include the enzymatic destruction of ribosomes and the delivery of viral class I antigens into the targeted tumor to be processed and presented on its cell surface to induce an antigen specific immune response. MTEM expects to start dosing enrolled subjects in a first-in-human Phase 1 study in relapsed/refractory patients with PD-L1-positive solid tumors in 2Q21.

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The Phase 1 study is planned as a multi-center, open-label, dose escalation and dose expansion trial in the United States and outside of the United States. Patients with confirmed PD-L1 expressing tumors or confirmed PD-L1 expression in the tumor microenvironment will be eligible to screen for enrollment in the clinical trial. Following determination of the maximum tolerated dose (MTD) or recommended Phase 2 dose, expansion cohorts are planned to study MT-6402 as a monotherapy in tumor-specific and tumor-agnostic cohorts.

"We are excited to be advancing MT-6402, a third generation ETB, into the clinic for the treatment of patients with PD-L1-positive cancers. MT-6402 utilizes both our proprietary de-immunized toxin scaffold and antigen seeding technology," said Eric Poma, Ph.D., CEO and CSO of Molecular Templates. "The PD-1/PD-L1 axis is central to many tumors and targeting that axis with a new mechanism of action has an opportunity to provide meaningful benefit to patients. We look forward to providing an update on the Phase 1 study by year-end 2021."

About MT-6402

MT-6402 is an ETB consisting of a single chain variable fragment (scFv) with affinity for PD-L1, fused to the enzymatically active de-immunized Shiga-like toxin-A subunit (SLTA) and a class I antigen derived from the human cytomegalovirus (HCMV) pp65 protein. MT-6402 was designed to induce potent anti-tumor effects via PD-L1 targeting through multiple mechanisms that may overcome the limitations of the PD-L1 antibodies. In MTEM’s preclinical studies, MT-6402 was found to specifically bind and kill both tumor and immune PD-L1 expressing cells in a manner consistent with SLTA mediated cellular cytotoxicity through ribosomal inactivation, independent of checkpoint inhibition. Additionally, MT-6402 alters the immunophenotype of targeted cells by delivering foreign class I antigen from CMV for presentation in complex with MHC class I, which may provoke a CMV-specific immune response against the targeted cells. Third, MT-6402 may rehabilitate the tumor microenvironment (TME) and allow for immune recognition of tumors by destroying PD-L1-expressing immune cells in the TME through ribosomal inactivation.

On January 19, 2021 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer, and other indications, reported that the U.S. Food and Drug Administration (FDA) has lifted the previously announced partial clinical hold on the phase 1 studies of PRS-343 (Press release, Pieris Pharmaceuticals, JAN 19, 2021, View Source [SID1234574097]). As previously guided, the Company intends to move into a proof-of-concept study of PRS-343 in gastroesophageal cancer.

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"It has been rewarding to see the impact of PRS-343 on the lives of patients in these studies. Data continue to be encouraging, and the clinical benefit linked to single-agent activity that we have seen attests to the potential of this therapy and, more broadly, our localized 4-1BB agonism approach," said Stephen S. Yoder, President and Chief Executive Officer of Pieris. "We are excited to advance this program into the proof-of-concept phase and look forward to sharing more details about the study later this quarter."

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.

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