On January 7, 2020 4D pharma plc (AIM: DDDD), a pharmaceutical company that leads the development of Live Biotherapeutics, reported the opening of a clinical study to assess clinical safety and efficacy of MRx0518 in combination with preoperative radiotherapy in 15 patients with resectable pancreatic cancer (Press release, 4d Pharma, JAN 7, 2020, https://www.prnewswire.com/news-releases/actualizacion-clinica-4d-anuncia-un-nuevo-estudio-de-mrx0518-en-cancer-pancreatico-828920161.html [SID1234552748]).

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The study is being conducted at The University of Texas MD Anderson Cancer Center and is the second open as part of a strategic collaboration to evaluate the Live Biotherapeutic oncology line of 4D in a number of cancer centers. Cullen M. Taniguchi , MD, Ph.D., assistant professor of radiation oncology at MD Anderson, is the lead investigator of the study.

Subjects will receive their daily treatment with MRx0518 for one week before and during radiotherapy, up to 24 hours before surgical resection. In addition to the main conclusion of safety and tolerability, the study will evaluate the preliminary clinical efficacy of the combination, including the evaluation of the main pathological response, progression-free survival and overall survival. Additional secondary and exploratory findings will evaluate changes in tumor infiltration lymphocytes (TILs) and the intestinal microbiome.

Alex Stevenson , 4D scientific director, commented:

"Pancreatic cancer carries a poor prognosis and remains a field of important need not covered. Prompted by promising initial signs in our other clinical studies, we believe that MRx0518 has the potential to offer new treatment options and dramatically improve outcomes for patients with pancreatic cancer. This third study demonstrates 4D’s current commitment to oncology. "

4D recently announced clinical observations of its current open label study of MRx0518 in combination with KEYTRUDA (pembrolizumab) in patients with solid tumors that have progressed in previous therapy of control inhibitors with no known alternative treatment options. The combination is well tolerated without side effects related to the drug and currently has partial induced responses in two of six evaluable patients and stable disease in a third patient.

On January 7, 2020 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, and NEC Corporation (NEC; TSE: 6701), a leader in IT and network technologies, reported that the first patients have been enrolled in the first-in-human trials evaluating TG4050, a therapeutic vaccine based on the myvac technology and powered by NEC’s cutting-edge AI capabilities (Press release, NEC, JAN 7, 2020, View Source [SID1234552727]). In these Phase 1 trials, TG4050 is being administered to patients with head and neck cancer who have a high risk of relapse after surgery and patients with ovarian cancer after surgery and adjuvant therapy.
Transgene’s highly innovative myvac technology allows the generation of a virus-based immunotherapy within a very short time frame while encoding patient-specific mutations identified and selected by NEC’s Neoantigen Prediction System.

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TG4050 has been designed to target up to 30 patient-specific neoantigens (cancer cell mutations). They are selected using NEC’s Neoantigen Prediction System, an advanced AI technology that has already been applied in the field of oncology. The prediction system is based on more than two decades of expertise in AI and has been trained on proprietary immune data, allowing it to accurately prioritize and select the most immunogenic sequences.
Transgene uses its expertise in viral vectorization via myvac to incorporate the selected neoantigen sequences in the genome of the Modified Vaccinia virus Ankara (MVA) viral vector. The Company has also set up a unique in-house good manufacturing practice (GMP) unit dedicated to the manufacturing of the individualized batches of TG4050 needed for the clinical development of this novel therapeutic vaccine.

"As each patient’s cancer is unique, we have developed a therapy that turns their solid tumor’s genetic signature into a powerful highly specific anticancer weapon. TG4050 is based on an MVA viral vector that has proven biological activity and has the ability to elicit an immune response against tumor antigens. Our partnership with NEC ensures that TG4050 is benefitting from its world-leading expertise in artificial intelligence and its unique algorithm that is used to select up to 30 patient-specific antigens that allow this novel vaccine to induce a strong immune response. We are convinced that TG4050, which is at the crossroad of immunotherapy and big data sciences, will herald the start of a new era in the fight against cancer,"
explained Philippe Archinard, Chairman and Chief Executive Officer of Transgene.

"We are excited to enroll our first patients in these trials and see TG4050 advance to the clinic. This is another step closer towards the realization of an AI-driven individualized immunotherapy for each patient. Our unique partnership with Transgene enables us to leverage its significant clinical development know-how and proven viral vector delivery platform. We are hopeful that TG4050 will make a significant difference for patients throughout the world," commented Osamu Fujikawa, Senior Vice President, NEC Corporation.

A Phase 1 clinical trial of TG4050 is enrolling patients with ovarian cancer after surgery and first-line chemotherapy. This multi-center, one-arm trial will recruit patients in the USA and in France. Endpoints of the trial include safety, feasibility and biological activity of the therapeutic vaccine. Dr. Matthew Block, MD, PhD, immunologist and medical oncologist at the Mayo Clinic, is conducting the trial in the USA; in France, the trial will be conducted by Dr. Martinez at Toulouse-Oncopole and by Pr. Le Tourneau at Institut Curie.

Another Phase 1 clinical trial of TG4050 is enrolling patients with newly diagnosed, locoregionally advanced, HPV negative, squamous cell carcinoma of the head and neck (SCCHN) that have received an adjuvant (first-line) therapy after surgery. This multi-center, open label, randomized two arms trial will include patients in the UK and in France. Patients will receive either TG4050 monotherapy after completion of the adjuvant therapy or in combination with the standard of care at the time of recurrence. Endpoints of the trial include safety, feasibility and biological activity of the therapeutic vaccine. In France, the trial is being conducted by Pr. Delord at Toulouse-Oncopole and by Pr. Le Tourneau at Institut Curie; in the UK, the trial is coordinated by Pr. Ottensmeier from Southampton University.

On January 06, 2020 Atossa Therapeutics, Inc., a clinical-stage biopharmaceutical company developing novel therapeutics and delivery methods for breast cancer and other breast conditions, reported that its corporate name change from "Atossa Genetics Inc." to "Atossa Therapeutics, Inc." became effective at 12:01 a.m. ET today, January 6, 2020 (Press release, Atossa Genetics, JAN 6, 2020, View Source;utm_medium=twitter [SID1234629125]). The Company’s common stock will continue to trade on the NasdaqCM exchange under the ticker symbol "ATOS."

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"Over the last several years, we have transitioned the Company’s focus on developing therapies to treat breast cancer, breast density and other breast conditions," said Steve Quay, Ph.D., M.D., president and CEO of Atossa. "Changing our name to Atossa Therapeutics, Inc. more clearly reflects our focus on developing therapies and continues to honor Princess Atossa, the great queen of the Achaemenid Empire, who reigned in the fifth century BCE and who is the earliest recorded woman with breast cancer. She is featured in the The Emperor of All Maladies: A Biography of Cancer, the Pulitzer Prize-winning book by Siddhartha Mukherjee on the history of cancer."

On January 6, 2020 Gene Techno Science (Kidswell Bio) reported a joint development agreement with Sapporo Medical University for a development of an anticancer drug using antibodies with an ability of entering cancer cell (Press release, Kidswell Bio, JAN 6, 2020, View Source [SID1234625474]).

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1. Purpose and background for concluding this agreement
This agreement aims to develop new anticancer drugs for rare and refractory cancers as a new pipeline in the new biologic business, which is the future growth driver for our company. In recent years, cancer treatment has dramatically improved due to the emergence of drugs that selectively inhibit abnormal proliferation signals of cancer cells or antibody drugs that selectively recognize only cancer cells. On the other hand, the therapeutic effect achieved only with single antibody drug is limited, so in clinical practice, the combination treatment of other existing anticancer drugs and antibody drugs is a common treatment. Recently, development of antibody-drug conjugates combining cytotoxic small molecules to an antibody that recognizes cancer cells, has been actively conducted. In Japan, two antibody-drug conjugates have been launched in the market and show a great therapeutic effect.

Assistant professor Miki Yamaguchi of Research Institute of Frontier Medicine, Sapporo Medical University School of Medicine, possesses antibodies by immunizing mice, which bind to molecules expressed only on the surface of cancer cells with an ability to enter cancer cells. GTS will sort out antibodies with superior ability in a recognition specificity and entering to cancer cells, and combine them to antibodydrug conjugates combining small molecule drugs or immunotoxin combining cytotoxic protein. GTS aims to develop anti-cancer drugs that show excellent therapeutic effects even by the single administration.

GTS will conduct this research and development based on the expertise of handling proteins accumulated in the past developments of biosimilar products and append this drug as a new pipeline in the new biologic business to secure new revenue sources in the future. Through the contribution to society by developing new anticancer drugs for rare and refractory cancers, we will accelerate to raise our enterprise value and aim to achieve GTS3.0 at an early stage.

2. Contents of this agreement
Joint development agreement for an anticancer drug using antibodies with an ability of entering cancer cells which Sapporo Medical University possesses.

3. Outline of counterparties to this agreement
1. Name Sapporo Medical University
2. Location South-1, West-17, Cyuo-ku, Sapporo, Japan
3. Chairperson and President Taiji Tsukamoto
4. Department Department of Molecular Medicine, Research Institute
for Frontier Medicine, Sapporo University School of
Medicine

4. Future outlook
The impact on the business results for the fiscal year ending March 2020 is expected to be minimal.

On January 6, 2020 BridgeBio Pharma, Inc. (Nasdaq: BBIO) subsidiary QED Therapeutics reported that it has secured both Fast Track Designation in adults with first-line advanced or metastatic cholangiocarcinoma and Orphan Drug Designation for infigratinib for treatment of cholangiocarcinoma (Press release, BridgeBio, JAN 6, 2020, View Source [SID1234576234]). In addition, the company announced that enrollment is ongoing and patient dosing has started in the PROOF trial, a Phase 3 clinical trial evaluating oral infigratinib, an investigational drug, in adults for first-line treatment of advanced cholangiocarcinoma with FGFR2 (fibroblast growth factor receptor 2) gene fusions or translocations.

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Cholangiocarcinoma, a cancer of the bile ducts of the liver, is a serious and often fatal disease which affects approximately 20,000 people in the United States and European Union each year. FGFR2 genetic aberrations are present in approximately 15% to 20% of people who have this disease. Currently, treatment options are limited, and the 5-year survival rate is only 9%.1

Infigratinib received Fast Track Designation for first-line treatment of adult patients with unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 gene fusions or translocations.

"We believe that Fast Track and Orphan Drug Designations for infigratinib for the treatment of cholangiocarcinoma underscores the need for new, targeted treatments for genetically-driven subsets of this cancer, particularly for adults with first-line advanced or metastatic cholangiocarcinoma," said Susan Moran, MD, MSCE, chief medical officer for QED. "Fast Track Designation will enhance our interaction with the FDA on our first-line advanced or metastatic cholangiocarcinoma program and may help us get this medicine to patients more quickly."

The PROOF trial will enroll approximately 384 patients with first-line cholangiocarcinoma with FGFR2 fusions or translocations, as determined by molecular profiling. The primary endpoint is progression-free survival compared to standard of care chemotherapy (gemcitabine and cisplatin). Patients will be randomized 2:1 to infigratinib versus standard of care.

"Importantly, in this trial, patients who are assigned to receive standard of care will be allowed to cross over and receive infigratinib if they do not respond to chemotherapy," said Stacie Lindsey, president of the Cholangiocarcinoma Foundation. "Having a crossover option is very significant to patients and including it in the design of this trial demonstrates that QED is listening to them."

For additional information on the PROOF trial, including eligibility, patients should ask their physician, visit clinicaltrials.gov, or email [email protected]

For more information on molecular profiling, patients can find resources from the Cholangiocarcinoma Foundation at View Source

About Orphan Drug Designation

Under the FDA’s Orphan Drug Designation program, orphan drug designation is granted by the FDA to drugs or biologics intended to treat rare diseases or conditions. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, if the drug receives the first FDA approval for the rare disease or condition, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

About U.S. FDA’s Fast Track Designation Program

The FDA’s Fast Track program was established to facilitate the development and expedite the review of drugs with the potential to treat serious conditions and address an unmet medical need. Companies that receive Fast Track designation are provided the opportunity for more frequent interactions with FDA during clinical development and are eligible for accelerated approval and/or priority review, if relevant criteria are met. Additionally, companies that receive Fast Track designation are allowed to submit completed sections of their New Drug Application (NDA) or Biologics License Application (BLA) for the drug on a rolling basis, resulting in the potential for an expedited FDA review process.