On February 17, 2022 RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, reported a business update for the fourth quarter of 2021, including certain estimated unaudited preliminary financial data (Press release, RedHill Biopharma, FEB 17, 2022, View Source [SID1234608234]).

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Dror Ben-Asher, RedHill’s Chief Executive Officer, said: "Strong sales growth momentum in the face of the persistent pandemic environment, coupled with strengthening our salesforce through internal realignment to include 120 customer-facing sales professionals, disciplined cost-control measures and the potential addition of products synergistic to our existing commercial basket, are planned to bring us closer to commercial operations profitability in 2022. In parallel, our compact R&D team continues to display tremendous creativity and drive in progressing RedHill’s robust late clinical-stage pipeline. In particular, extensive discussions are ongoing with regulators in multiple countries regarding potential pathways to approval of orally-administered opaganib, likely the first novel oral drug candidate to have shown an improvement in viral clearance in severe hospitalized COVID-19 patients."

The Company intends to announce its audited fourth quarter and full year 2021 results in the coming weeks. The preliminary financial data ranges described herein have not been audited and are subject to adjustment based on the Company’s completion of year-end financial close processes.

On February 17, 2022 Edison Oncology Holding Corp. ("Edison Oncology"), a company established to develop and commercialize novel therapies targeting the fight against cancer reported that the first patient has received treatment with Orotecan (irinotecan HCI oral solution) in a Phase I/IIa clinical trial for patients with recurrent pediatric solid tumors (Press release, Edison Oncology, FEB 17, 2022, View Source [SID1234608250]).

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The clinical trial will enroll up to 20 patients with recurrent pediatric and solid tumors, including but not limited to neuroblastoma, rhabdomyosarcoma, Ewing sarcoma, hepatoblastoma and medulloblastoma. The trial endpoints will characterize the pharmacokinetics of Orotecan vs. unformulated irinotecan in an already established oral treatment regimen, document safety and tolerability, and assess tumor response. The trial is currently enrolling patients at Duke University Children’s Hospital and Cincinnati Children’s Hospital Medical Center, with opening of additional sites anticipated in the near future.

"We are excited to have treated the first patient treated with Orotecan, our novel oral formulation of irinotecan, which we believe has the potential to improve the quality of life for pediatric and adult cancer patients, improve patient compliance and reduce costs" said Jeffrey Bacha, Edison Oncology’s chief executive officer.

Further details of the trial can be found on clinicaltrials.gov (NCT04337177).

About Orotecan

Orotecan is a patented, novel formulation of irinotecan that was developed to improve palatability and

clinical utility of an established oral delivery regimen for numerous pediatric cancers.

On February 17, 2022 Applied DNA Sciences, Inc. (NASDAQ: APDN) (the "Company"), a leader in Polymerase Chain Reaction (PCR)-based DNA manufacturing and nucleic acid-based technologies, and its program development partner, EvviVax, S.R.L. ("Evvivax"), reported the publication of a manuscript detailing a preclinical study (the "study") showing that LinearDNA vaccines used for cancer immunotherapy produced a strong immune and specific antitumoral response in preclinical mouse models (Press release, Applied DNA Sciences, FEB 17, 2022, View Source [SID1234608218]). The study investigated the use of the LinearDNA platform to produce DNA vaccines targeting either tumor-associated antigens (TAA) and tumor-specific antigens (TSA or tumor neoantigens). The manuscript, "Linear DNA Amplicons as a Novel Cancer Vaccine Strategy," is published online on the bioRxiv.org preprint server and has been submitted for peer-reviewed publication. LinearDNA is Applied DNA’s proprietary, large-scale polymerase chain reaction ("PCR")-based manufacturing platform that allows for the large-scale production of specific DNA sequences.

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DNA vaccines that target TAAs hold promise as potential pan-cancer vaccines that, when used in conjunction with existing standards of care, can increase the efficacy of cancer immunotherapies. DNA vaccines targeting TSAs, otherwise known as personalized cancer vaccines, also hold great promise in immunotherapy as they can be customized to induce an immune response only against a patient’s tumor, thereby limiting on-target, off-tumor effects.

TAA: TERT Vaccine
One aspect of the study used a DNA vaccine targeting telomerase reverse transcriptase (TERT), a TAA that holds potential as a target for a pan-cancer vaccine. The TERT DNA vaccine was designed by EvviVax and exclusively licensed by the Company for the LinearDNA platform for veterinary applications. In prior clinical trials conducted by EvviVax, a plasmid form of the TERT DNA vaccine administered along with the standard of care chemotherapy was shown to increase the survival of canines with Stage III/IV B cell lymphoma from 37 weeks to 97 weeks. B-cell lymphoma is the most common type of non-Hodgkin lymphoma in canines, with lymphoma accounting for 15-20% of new cancer diagnoses in canines. For the study, the TERT DNA vaccine was administered to mice in either plasmid DNA or LinearDNA form and the immune response studied and compared. The study’s results demonstrated that both the plasmid DNA and LinearDNA forms of the TERT DNA vaccine induced comparable immune responses in mouse models.

TSA/Neoantigens Vaccine
The second aspect of the study utilized a personalized DNA vaccine specifically targeting several TSAs expressed in a colon cancer mouse model. Personalized cancer vaccines hold great promise in immunotherapy as they can be customized to induce an immune response only against a specific patient’s tumor, thereby limiting off-tumor effects and increasing efficacy and therapeutic index. In the study, LinearDNA and plasmid DNA forms of the personalized cancer vaccine were administered to mice in the colon cancer model. For both forms of the DNA vaccine, several cohorts also received immune checkpoint inhibitors (ICI) based on anti-CTLA-4 and/or anti-PD1. The study demonstrated that the LinearDNA personalized vaccine produces an equal or greater immune and antitumoral response than the plasmid form of the same DNA vaccine, particularly when coupled with ICI.

Dr. James A. Hayward, president and CEO of Applied DNA, stated, "The study demonstrates that LinearDNA and plasmid DNA can elicit a comparable immune response in animal cancer models. We believe this study validates the use of LinearDNA as a more cost- and time-efficient alternative to plasmid DNA for DNA-based cancer vaccines. Cancer immunotherapy is relevant to veterinary and human markets; the latter is expected to reach $169 billion by 2028. As the exclusive licensee of the TERT vaccine for veterinary applications, we believe these data support further investigation of the LinearDNA vaccine as a potential veterinary cancer immunotherapy and, beyond that, for human cancer immunotherapy."

Dr. Luigi Aurisicchio, CEO and chief scientific officer of Evvivax S.R.L., commented, "We believe that DNA vaccines for cancer hold immense promise for both human and veterinary applications. One obstacle to DNA vaccine manufacturing is their current production as plasmid DNA. We believe that the completely cell-free LinearDNA platform avoids the numerous pitfalls of plasmid DNA-based production, making it ideal for DNA vaccine manufacturing broadly, and in cancer immunotherapy, specifically."

On February 17, 2022 Ribon Therapeutics, a clinical stage biotechnology company developing therapeutics targeting stress support pathways, reported that the Board of Directors has appointed Prakash Raman, Ph.D., as President, Chief Executive Officer and member of the Board of Directors (Press release, Ribon Therapeutics, FEB 17, 2022, View Source [SID1234608235]). Dr. Raman has succeeded Victoria Richon, Ph.D., in these roles. Dr. Richon will remain actively involved in the Company as an R&D advisor to the Board of Directors and Chair of the Scientific Advisory Board, where she will work closely with Dr. Raman and executive leadership to advance Ribon’s scientific activities.

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"Since joining Ribon in 2015 as founding President, Vicky has built an exceptional research and development organization that has created a novel pipeline of first-in-class drugs in clinical development," said Jodie Morrison, Chair of the Board of Directors of Ribon Therapeutics. "The Board is excited to welcome Prakash, whose scientific background and strong business acumen are an excellent complement to our executive management team. This leadership transition allows Ribon to enhance its focus on its pipeline capabilities, business development and strategic growth plans while enabling Vicky to continue to contribute to the advancement of our innovative science as R&D Advisor to the Board of Directors and Chair of our Scientific Advisory Board. On behalf of the Board of Directors, I sincerely thank Vicky for her many contributions to the Company."

"It was only several years ago that the role of NAD+-utilizing enzymes in stress support pathways in cancer and inflammatory disease was considered an emerging science and not well understood," said Dr. Raman. "Under Vicky’s leadership, Ribon has advanced this family of enzymes to the clinic, where initial data have demonstrated PARP7, an NAD+-utilizing monoPARP, inhibition and early signs of antitumor activity in patients, a remarkable achievement. I am honored to join such a strong scientific organization with great potential for transforming the lives of patients with high unmet needs through the application of truly innovative science. I look forward to a collaborative and productive relationship with Vicky and the organization at Ribon to shepherd the company through its next phase of growth."

"I am pleased to welcome Prakash to Ribon at this important time for the Company," said Dr. Richon. "Prakash has the experience and skill set to carry the business forward toward our goal of bringing novel medicine to patients with cancer and inflammation. I look forward to a strong collaboration with the team in my role as SAB Chair as we work together to achieve our Company’s mission."

Dr. Raman brings to Ribon decades of biopharmaceutical business development and executive leadership experience, blending his scientific background, program and portfolio management and strong business development experience. Prior to joining Ribon, he served as Senior Partner, Chief Business Development Officer at Flagship Pioneering, where he leveraged the platforms and assets in Flagship’s network to generate opportunities for significant value creation. Prior to Flagship, Dr. Raman spent nearly fourteen years at Novartis, most recently as Vice President, Global Head of Novartis Institutes for Biomedical Research (NIBR) Business Development and Licensing (BD&L). During his time at Novartis, Dr. Raman was instrumental in forging key collaborations in immuno-oncology, executing many out-licensing opportunities and guiding the acquisitions of Advanced Accelerator Applications, Endocyte, IFM Tre and Selexys. In addition, he has led cross-functional drug discovery and early development project teams that successfully progressed compounds to clinical testing in patients. Prior to Novartis, Dr. Raman spent six years as a Senior Scientist at Millennium Pharmaceuticals and two years as a post-doctoral fellow at The Scripps Research Institute. He completed his undergraduate work at the Indian Institute of Technology, Bombay, and received his Ph.D. in Organic and Medicinal Chemistry from the University of Wisconsin-Madison.

Ribon, named a "Fierce 15" Biotech Company by Fierce Biotech in 2021, is pioneering the discovery and development of first-in-class precision therapies targeting stress support pathways in cancer and inflammation for patients with limited options. By leveraging its proprietary BEACON+ platform, the Company is building a pipeline of selective, small molecule inhibitors to numerous NAD+-utilizing enzymes. Ribon’s lead program is RBN-2397, a PARP7 inhibitor in clinical development for the treatment of cancer. The expansion portion of the RBN-2397 Phase 1 trial is currently underway in a number of defined patient cohorts, including squamous cell carcinoma of the lung (SCCL). Ribon is also advancing a second clinical candidate, RBN-3143, a potent and selective PARP14 inhibitor for the treatment of patients with inflammatory diseases.

On February 17, 2022 Gracell Biotechnologies Inc. ("Gracell" or the "Company",NASDAQ: GRCL), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, reported that it has dosed multiple patients in a clinical trial evaluating GC012F, the Company’s autologous CAR-T therapeutic candidate dual-targeting B cell maturation antigen (BCMA) and CD19 in B-cell non-Hodgkin’s lymphoma (B-NHL) (Press release, Gracell Biotechnologies, FEB 17, 2022, View Source [SID1234608251]). NHL is the fifth most common cancer in the U.S.[1]

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The Phase 1 investigator-initiated trial (IIT), being conducted in China, is a first-in-human study evaluating FasTCAR-enabled BCMA/CD19 dual-targeting GC012F for the treatment of relapsed or refractory (r/r) B-NHL. GC012F is the first BCMA/CD19 dual-targeting CAR-T in human trials for B-NHL. Most B-NHL cells express CD19, and data also suggest that 39% to 97% clinical samples of NHL cells also express BCMA. [2] [3] [4] By simultaneously targeting BCMA and CD19, GC012F is designed to improve efficacy outcome in r/r B-NHL patients.

GC012F, developed using Gracell’s proprietary FasTCAR platform which enables next-day manufacturing, GC012F is currently also being evaluated in IIT studies in China including relapsed/refractory multiple myeloma. In November 2021, GC012F was granted Orphan Drug Designation by the U.S. Food and Drug Administration.

"Dual-targeting CD19 and BCMA represents an innovative approach for the treatment of B-NHL. This study of GC012F for r/r B-NHL marks an important step in the product candidate’s development to expand to additional indications and we look forward to confirming its potential to treat B-NHL, an indication for which patients are in need of additional treatment options," said Dr. Martina A. Sersch, Chief Medical Officer of Gracell. "We are confident that this study will further validate GC012F, our FasTCAR platform and our dual-CAR technology."

[1] View Source

[2] Blood Cancer Journal (2020) 10:73

[3] Blood. 2017;130:2755.

[4] Hum Gene Ther. 2018; 29(5): 585.

About GC012F

GC012F is a FasTCAR-enabled dual-targeting CAR-T product candidate that is currently being evaluated in IIT studies in China for the treatment of multiple myeloma and B-cell non-Hodgkin’s lymphoma. GC012F simultaneously targets CD19 and BCMA to drive fast, deep and durable responses, which can improve efficacy and reduce relapse in multiple myeloma and B-NHL patients.

About B-NHL

Non-Hodgkin’s lymphoma (NHL) is a group of blood cancers that developed from lymphocytes, most commonly derived from B cells (B-NHL). Globally, approximately 510,000 patients are diagnosed with NHL every year with over 77,000 patients diagnosed in the United States, and approximately 68,000 diagnosed in China in 2020. B-NHL accounts for approximately 85% of NHL diagnoses.

About FasTCAR

CAR-T cells manufactured on Gracell’s proprietary FasTCAR platform appear younger, less exhausted and show enhanced proliferation, persistence, bone marrow migration and tumor cell clearance activities as demonstrated in preclinical studies. With next day manufacturing, FasTCAR is able to significantly improve cell production efficiency which may result in meaningful cost savings, increasing the accessibility of cell therapies for cancer patients.