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;id=224228&p=2220104&I=1206939-c7Z3G6f3m8 [SID1234608240]). The study investigated the use of the LinearDNA platform to produce DNA vaccines targeting either tumor-associated antigens (TAA) or 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 canines1, with lymphoma accounting for 15-20% of new cancer diagnoses in canines2. 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 produced 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 20283. As the exclusive licensee of the TERT DNA 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 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) reported that it will report its financial results for the year ended December 31, 2021 on Thursday, February 24, 2022, after the close of the U.S. financial markets (Press release, Y-mAbs Therapeutics, FEB 17, 2022, View Source [SID1234608239]). The announcement will be followed by a conference call and webcast with the investment community on Friday, February 25, 2022, at 9 a.m. ET. Participating on the call from Y-mAbs will be Thomas Gad, founder, Chairman and President; Dr. Claus Moller, Chief Executive Officer; and Bo Kruse, Chief Financial Officer.

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Conference call and webcast details:

Webcast: View Source;tp_key=59e1f9cc51

On February 17, 2022 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported positive interim results from AROHIF21001, a Phase 1b dose-finding clinical study of ARO-HIF2, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with clear cell renal cell carcinoma (ccRCC) (Press release, Arrowhead Pharmaceuticals, FEB 17, 2022, View Source [SID1234608237]). The data presented provide initial proof of target engagement based on reductions in hypoxia inducible factor-2 alpha (HIF2α) expression, as well as an acceptable safety profile in response to escalating doses of ARO-HIF2. The data are being presented by James Brugarolas, M.D., Ph.D, Professor at University of Texas Southwestern Medical Center and investigator in the study, in a poster presentation at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO GU), being held February 17-19, 2022, in San Francisco, CA and online.

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Presentation Details:

Title: Initial results from the phase 1 study of ARO-HIF2 to silence HIF2-alpha in patients with advanced ccRCC (AROHIF21001)
Authors: James Brugarolas, et al.
Session: Poster Session C: Renal Cell Cancer; Adrenal, Penile, Urethral, and Testicular Cancers
Abstract Number: 339
Poster Number: F9

Key results from AROHIF21001 as of December 1, 2021 data cut:

Pharmacodynamics and Efficacy

Tumoral expression of HIF2α protein was assessed via immunohistochemistry
Among patients with evaluable biopsy, 9/14 showed reductions in HIF2α protein
Responders in Cohort 1 (225 mg, n=3), Cohort 2 (525 mg, n=4), and Cohort 3 (1050 mg, n=2) achieved mean reductions of HIF2α protein of -45%, -57%, and -80%, respectively
Tumoral expression of HIF2α messenger RNA (mRNA) was assessed by quantitative polymerase chain reaction (qPCR)
Among patients with evaluable biopsy, 9/9 showed reductions in HIF2α mRNA
Cohort 1, Cohort 2, and Cohort 3 achieved mean reductions of HIF2α mRNA of -38%, -28%, and -44%, respectively
Efficacy was assessed by Response Evaluation Criteria in Solid Tumors (RECIST)
Disease control rate (complete response + partial response + stable disease) was 39% (10 of 26) across all cohorts
Objective response (complete response + partial response) was 8% (2 of 26), with one patient in Cohort 2 and one patient in Cohort 3 achieving a partial response
Safety

ARO-HIF2 was generally well-tolerated in patients. Anemia and hypoxia, frequently reported on-target adverse events (AEs) with small molecule HIF2α inhibitors, were reported in 12% of patients
Five serious AEs in 5 patients were reported by investigators as possibly drug related, including myocarditis (in a patient with a history of TKI induced cardiomyopathy), demyelinating neuropathy (in a patient with autoimmune sequelae due to checkpoint inhibitors), chronic inflammatory demyelinating polyradiculoneuropathy (in a patient with distant history of checkpoint inhibitor use), hypoxia (in a patient with a pulmonary infiltrate), and acute hypoxemic respiratory failure (in a patient with progressive lung metastatic disease)
A copy of the presentation materials with full data may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.

AROHIF21001 (NCT04169711) is a Phase 1b dose-finding clinical study in patients with advanced ccRCC to evaluate the safety of ARO-HIF2 and to determine the recommended Phase 2 dose. Secondary objectives include the assessment of pharmacokinetics and preliminary efficacy, based on Response Evaluation Criteria in Solid Tumors (RECIST). Exploratory objectives for AROHIF21001 are post-dose tumoral expression of HIF genes in response to treatment with ARO-HIF2, change in Karnofsky Performance Status (KPS), correlation of tumor response based on RECIST with tumor HIF2α gene expression and tumor integrin expression, correlation of integrin expression with changes in HIF gene expression, evaluation of serum biomarkers of ARO-HIF2 activity, correlation of RCC-related gene expression to ARO-HIF2 activity, and evaluation of plasma and urine metabolites.

On February 17, 2022 XOMA Corporation (Nasdaq: XOMA), a biotechnology royalty aggregator playing a distinctive role in helping companies achieve their goal of improving human health, reported its Chief Executive Officer, Jim Neal, will present at the Aegis Capital Corp Virtual Conference on February 24, 2022 at 11:00 AM ET (Press release, Xoma, FEB 17, 2022, View Source [SID1234608236]). The conference will be held from February 23-25, 2022, from 8:30 AM until 5:30 PM ET daily, and can be viewed at https://bit.ly/3BoscUs.

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XOMA’s presentation will be available by request to Aegis during the conference. The presentation can also be accessed by visiting the investor relations section of the Company’s website at www.xoma.com. A replay of the presentation will be available and archived on the site for 90 days after the event.

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.