On December 21, 2020 Genprex, Inc. ("Genprex" or the "Company") (Nasdaq: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that it has completed the manufacturing scale-up of REQORSA immunogene therapy (Press release, Genprex, DEC 21, 2020, View Source [SID1234573203]). The clinical-grade production is intended to supply the Company’s upcoming Acclaim-1 and Acclaim-2 clinical trials for the treatment of non-small cell lung cancer, subject to passing final testing that is currently underway. The Company recently announced the successful manufacturing technology transfer to commercial Contract Development and Manufacturing Organizations (CDMOs) and the successful engineering run of REQORSA, that passed all testing specifications.

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For the first time, REQORSA was manufactured in a scaled-up clinical production in accordance with the current Good Manufacturing Practices (cGMP) required by the U.S. Food and Drug Administration (FDA) in advance of commercial approval of a drug product. This product will supply the Company’s upcoming Acclaim-1 and Acclaim-2 clinical trials that combine REQORSA with Tagrisso (marketed by AstraZeneca) and with Keytruda (marketed by Merck & Co., Inc.), respectively, both of which are on track to be initiated in the first-half of 2021. This production includes process improvements that resulted in significantly higher yields and lower costs than prior manufacturing campaigns. Previously, REQORSA was manufactured at the major cancer research institution where it was invented.

"The scaled-up production of clinical-grade REQORSA is yet another significant manufacturing milestone for the Company," said Michael Redman, Executive Vice President and Chief Operating Officer of Genprex. "Utilizing advanced processes, we were able to successfully improve our production yield multi-fold with significantly improved economies of scale. This production is intended to provide REQORSA for our upcoming Acclaim clinical trials. Furthermore, this achievement positions Genprex with the manufacturing capability for potential future commercialization in the rapidly growing lung cancer therapeutics market, which is projected to grow to $26.3 billion by 2023."

Upon completion of testing and lot release, REQORSA will be transported to cold storage depots. These depots will facilitate shipments to the clinical trial sites following FDA clearance to commence the upcoming clinical trials. Unlike some biologicals that require shipment and storage at -80°C temperatures, REQORSA requires storage at only 2-8°C.

REQORSA is comprised of TUSC2 plasmid DNA (the active agent in REQORSA) encapsulated in non-viral nanoparticles that are administered intravenously and designed to target tumor cells.

On December 21, 2020 Anixa Biosciences, Inc. (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer and infectious diseases, reported that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for its breast cancer vaccine (Press release, Anixa Biosciences, DEC 21, 2020, View Source [SID1234573162]).

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This breast cancer vaccine technology was invented and developed by Cleveland Clinic immunologist Dr. Vincent Tuohy, and his research team.
Oncologist, Dr. Thomas Budd, also of Cleveland Clinic, will lead the clinical trial.
Anixa Biosciences has an exclusive worldwide license to the technology.
The technology immunizes against a protein called alpha-lactalbumin that is expressed in the mammary glands of women, only during the latter part of gestation and during lactation. After lactation ceases, this protein is no longer expressed until a woman develops breast cancer. In a vaccinated woman, the researchers anticipate that these cancer cells will be destroyed by the immune system before they have the opportunity to grow into a mature cancer.
The initial focus is Triple Negative Beast Cancer, but this technology is expected to potentially prevent other types of breast cancer.
Animal studies showed notable ability to prevent breast cancer.
The preclinical studies and two trials of this vaccine are being funded by the U.S. Department of Defense.
Dr. Amit Kumar, President and CEO of Anixa stated, "We are pleased that the FDA has authorized us to commence human clinical trials of our potentially paradigm-shifting vaccine for the prevention of breast cancer. This approval triggers a cascade of events and activities, that will eventually lead to recruitment of patients and initiation of the trial."

"This is a significant milestone for our program. Our vision has always been to prevent cancer before it arises," said Dr. Tuohy. "We are looking forward to beginning clinical trials in patients."

On December 21, 2020 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the company has initiated the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking marketing approval for JZP-458 for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) in adult and pediatric patients who have developed hypersensitivity or silent inactivation to E. coli-derived asparaginase (Press release, Jazz Pharmaceuticals, DEC 21, 2020, View Source [SID1234573184]).

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The BLA was initiated and will be reviewed under the Real-Time Oncology Review (RTOR) pilot program, an initiative of the FDA’s Oncology Center of Excellence designed to expedite the delivery of safe and effective cancer treatments to patients.

"Given the urgent need for a reliable and high-quality recombinant asparaginase option for patients with hypersensitivity to E. coli-derived asparaginase, we are committed to bringing JZP-458 to market as quickly as possible and pleased to be initiating our BLA submission," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development of Jazz Pharmaceuticals. "Receiving a Fast Track designation for JZP-458 from the FDA in October 2019 and being able to submit the BLA under the RTOR program is significant, potentially allowing us to more quickly address patient need with a new asparaginase option."

The company continues to plan for a mid-2021 launch of JZP-458 following completion of the BLA submission and FDA review and approval.

An ongoing Phase 2/3 study is being conducted in collaboration with the Children’s Oncology Group (COG) to evaluate JZP-458 as a potential treatment option for pediatric and adult patients with ALL or LBL who are hypersensitive to E. coli-derived asparaginases. Hypersensitivity reactions affect up to 30 percent of patients with ALL and LBL who are treated with E. coli-derived asparaginase.1

About JZP-458
JZP-458 is a recombinant Erwinia asparaginase that uses a novel Pseudomonas fluorescens expression platform. It is being developed for use as a component of a multi-agent chemotherapeutic regimen in the treatment of pediatric and adult patients with acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) who are hypersensitive to E. coli-derived asparaginase products. JZP-458 was granted Fast Track designation by the U.S. Food and Drug Administration in October 2019 for the treatment of this patient population.

About Acute Lymphoblastic Leukemia
Acute lymphoblastic leukemia (ALL) is a cancer of the blood and bone marrow that can progress quickly if not treated.2 Leukemia is the most common cancer in children, and about three out of four of these cases are ALL.3 Although it is one of the most common cancers in children, ALL is among the most curable of the pediatric malignancies due to recent advancements in treatment.4,5 Adults can also develop ALL, and about four of every 10 cases of ALL diagnosed are in adults.6 The American Cancer Society estimates that almost 6,000 new cases of ALL will be diagnosed in the United States in 2019.6 Asparaginase is a core component of multi-agent chemotherapeutic regimens in ALL.7 However, asparaginase treatments derived from E. coli are associated with the potential for development of hypersensitivity reactions.8

On December 21, 2020 Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), reported that the first patient has been dosed in the phase 3 LIGHTHOUSE study, evaluating the efficacy and safety of a triple combination therapy with melflufen plus dexamethasone and subcutaneous daratumumab compared to daratumumab alone (Press release, Oncopeptides, DEC 21, 2020, View Source [SID1234573164]). The phase 3 LIGHTHOUSE study is a randomized, open-label study in patients with relapsed refractory multiple myeloma who are refractory to an immunomodulatory agent and a proteasome inhibitor or who have had at least three prior lines of therapy, including these agents.

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"Following the encouraging results of our ANCHOR study this is an important study to further evaluate the potential role of melflufen in triplet regimens", says Klaas Bakker, MD, PhD, Chief Medical Officer, Oncopeptides AB. "There is an imminent need for additional therapeutic options as myeloma patients become multi-resistant earlier in their treatment journey. A positive outcome may potentially support the use of melflufen as combination therapy with daratumumab in earlier stages of multiple myeloma".

Additional study information can be found on View Source, identifier: NCT04649060.

About melflufen

Melflufen (INN melphalan flufenamide) is a first in class peptide-drug conjugate (PDC) that targets aminopeptidases and rapidly releases alkylating agents into tumor cells. Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and is immediately hydrolyzed by peptidases to release an entrapped hydrophilic alkylator payload. Aminopeptidases are overexpressed in tumor cells and are even more pronounced in advanced cancers and tumors with a high mutational burden. In vitro, melflufen is 50-fold more potent in myeloma cells than the alkylator payload itself due to the increased intracellular alkylator concentration. Melflufen displays cytotoxic activity against myeloma cell lines resistant to other treatments, including alkylators, and has also demonstrated inhibition of DNA repair induction and angiogenesis in preclinical studies. In the pivotal phase 2 HORIZON study melflufen plus dexamethasone demonstrated encouraging efficacy and a clinically manageable safety profile in heavily pretreated patients with relapsed refractory multiple myeloma, with primarily hematologic Adverse Events (AE) and a low incidence of non-hematologic AEs.

On December 21, 2020 BioEclipse Therapeutics (BioEclipse), a private clinical-stage biopharmaceutical company with a proprietary platform for developing next-generation cancer immunotherapies, reported the initiation of patient enrollment in a Phase 1 dose-escalation trial to treat refractory solid tumors (Press release, BioEclipse Therapeutics, DEC 21, 2020, View Source [SID1234573186]). The trial marks the first-in-human study of CRX100, an intravenously-delivered cancer therapy designed to target and destroy multiple cancer types and address disease recurrence.

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Patient enrollment is underway at our first clinical trial site, Moores Cancer Center at UC San Diego Health in La Jolla, CA. Additional clinical trial sites are expected to follow. More information about this study and general information about participating in clinical trials can be found at ClinicalTrials.gov.

"The launch of this clinical trial marks a significant milestone in the clinical development of CRX100 and is a further step toward bringing patients with few treatment options a single therapeutic designed to attack multiple cancer types," stated Pamela Contag, Ph.D., founder and CEO of BioEclipse. "We believe CRX100 has the potential to address the growing unmet need for treatments of solid tumors and metastatic disease believed to be untreatable, and that are currently underrepresented in clinical trials."

This open-label, Phase 1 dose-escalation study is designed to determine the safety, tolerability, and pharmacokinetic (PK) properties of CRX100 in up to 24 participants ­18 years or older with advanced solid tumors that do not respond to standard of care. The trial specifically targets six potential cancer indications, including: triple-negative breast cancer, colorectal cancer, hepatocellular carcinoma, osteosarcoma, epithelial ovarian cancer, and gastric cancer. Each patient will receive up to two doses of CRX100. As secondary endpoints, the trial will also investigate the effect CRX100 has on a participant’s tumor progression and overall immune response.

BioEclipse is currently focused on the treatment of recurring cancers with a unique multi-mechanistic approach that could address cancers believed to be untreatable. Developed with technology exclusively licensed from Stanford University, CRX100 combines activated immune cells, known as cytokine-induced killer (CIK) cells, with a tumor-killing virus. As stand-alone therapies, these two agents have previously been assessed in human studies. When combined to create CRX100, the CIK cells protect the oncolytic virus and deliver it to cancer cells throughout the body. The two components then work together to attack primary tumors and metastatic disease. Data from preclinical studies shows that this combination approach also can trigger a long-lasting immune response that protects against relapse and disease recurrence.

"The initiation of this clinical trial is welcome news given the urgent need for more effective approaches, especially for patients with cancer refractory to standard treatments," said Sandip Patel, M.D., Associate Professor at University of California San Diego School of Medicine and a Principal Investigator for this study. "If CRX100’s treatment approach delivers the same compelling results in humans as it has in preclinical models, it has the potential to address several types of cancer with an otherwise poor prognosis and bring new hope to our patients and their families."