On February 17, 2022 Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the "Company" or "Quoin"), a specialty pharmaceutical company focused on rare and orphan diseases, reported that its wholly-owned subsidiary, Quoin Pharmaceuticals, Inc., has entered into an exclusive Distribution Agreement with Neopharm Medical Supplies, an exclusive distributor for leading medical and pharma manufacturers in Israel, for QRX003, the Company’s investigational treatment for Netherton Syndrome, a rare and devastating genetic disease for which there is currently no available treatment or cure (Press release, NeoPharm, FEB 17, 2022, View Source [SID1234608241]).

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Under the terms of the revenue sharing agreement, Neopharm gains exclusive rights to commercialize QRX003 in Israel. Quoin will be the exclusive supplier of QRX003 to Neopharm.

Dr. Michael Myers, Chief Executive Officer of Quoin, commented, "This is Quoin’s sixth distribution agreement in the last four months for QRX003 and we’re excited to add Neopharm, who is a leading distributor in Israel. We now have 54 countries covered by our partnership agreements for QRX003, encompassing many regions of the globe. Establishing a broad distribution network is a key part of our commitment to ensure that every patient, everywhere will have access to this product once it has been approved for this devastating disease."

About Netherton Syndrome

Netherton Syndrome, a form of Ichthyosis, is a rare, hereditary skin disorder caused by a mutation in the SPINK5 gene (serine protease inhibitor, Kazal Type 5) that leads to severe skin barrier defects and recurring infections, as well as a pronounced predisposition to allergies, asthma, and eczema. Patients also often suffer from severe dehydration, chronic skin inflammation and stunted growth.

Currently, there is no cure for Netherton Syndrome, nor are there any approved therapeutic treatments.

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.