On April 14, 2021 The National Comprehensive Cancer Network (NCCN) Oncology Research Program (ORP) reported that three projects have been selected to study oral decitabine (35 mg) and cedazuridine (100 mg) (Press release, Astex Pharmaceuticals, APR 14, 2021, View Source [SID1234578021]). The NCCN ORP convened a Scientific Review Committee to review, evaluate, and select awardees and will provide oversight for the two-year studies—which will commence later in 2021. Research funding will be provided by a grant from Taiho Oncology, who will also supply the decitabine and cedazuridine tablet.

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The selected projects are:

• Michael Byrne, DO, Vanderbilt-Ingram Cancer Center, Moffitt Cancer Center
o Phase 2 Study of Decitabine and Cedazuridine in Combination with Venetoclax for AML Relapse after Allogeneic Hematopoietic Cell Transplantation

• Gurkamal Chatta, MD, Roswell Park Comprehensive Cancer Center
o A Phase 1b Clinical Trial: Improving Outcomes with Androgen Pathway inhibitors by Targeting DNA Methyltransferase Activity

• Martin McCarter, MD, University of Colorado Cancer Center
o Oral Decitabine/Cedazuridine in Combination with Nivolumab as a Strategy to Enhance the Efficacy of Immune Checkpoint Blockade in Unresectable or Metastatic Mucosal Melanoma

"Congratulations to all of the selected investigators," said Wui-Jin Koh, MD, Chief Medical Officer, NCCN. "We look forward to their work advancing our understanding of this medication combination. The potential for oral medication in place of intravenous delivery is particularly worthy of exploration, as this may provide better options for outpatient therapy with reduced office visits, which in turn improves access to care and helps underserved patient populations.

"Taiho Oncology is pleased to continue our collaboration with the National Comprehensive Cancer Network to help broaden the understanding of oral decitabine and cedazuridine for patients with solid tumors and hematologic malignancies," said Terri L. Washington, DNP, RN, Vice President, Scientific Partnerships and Medical Affairs Operations, Taiho Oncology, Inc. "These grants will help advance critical research and represent a step forward in exploring the full potential of oral decitabine and cedazuridine for patients to help improve outcomes."

The NCCN ORP fosters innovation and knowledge discovery that improves the lives of people with cancer and supports preclinical, translational, clinical research and quality improvement projects in oncology at NCCN Member Institutions. In an effort to improve collaboration in cancer research, the NCCN ORP also maintains a shared resources website and an informed consent database. For more information, visit NCCN.org/orp.

On April 14, 2021 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported that HOOKIPA’s management team will participate and host 1:1 meetings at the virtual Kempen Life Science Conference, taking place April 21, 2021 (Press release, Hookipa Pharma, APR 14, 2021, View Source [SID1234578019]).

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Additional information will be available within the Investors & Media section of HOOKIPA’s website at View Source

On April 14, 2021 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPD) to its p-STAT3 inhibitor, WP1066, for the treatment of ependymoma (Press release, Moleculin, APR 14, 2021, View Source [SID1234578018]).

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Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors. (PRNewsfoto/Moleculin Biotech, Inc.)

Ependymoma is a rare type of tumor that can form in the brain or spinal cord. Ependymoma begins in the ependymal cells in the brain and spinal cord that line the passageways where the fluid (cerebrospinal fluid) that nourishes your brain flows. Ependymoma can occur at any age, but most often occurs in young children.

"The FDA’s recognition of the high prevalence and unmet needs in the treatment landscape for ependymoma, especially in pediatric patients is a significant milestone as we continue to advance and expand the WP1066 development program. We currently have Orphan Drug Designation for WP1066 for the treatment of brain tumors, as well as RPD designation for three other pediatric indications, and believe that ependymoma represents another important rare indication. We continue to be encouraged by the data WP1066 has demonstrated to date and believe it has the potential to be an effective therapy for pediatric patients with ependymoma," commented Walter Klemp, Chairman and CEO of Moleculin.

The Rare Pediatric Disease Priority Review Voucher program, which was created as part of the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012, is intended to incentivize the development of new therapies for rare pediatric diseases. Under the FDA’s rare pediatric disease designation program, the FDA may grant a priority review voucher to a sponsor who receives a product approval for a "rare pediatric disease," which is defined as a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years and affects fewer than 200,000 people in the U.S. Subject to FDA approval of WP1066 for the treatment of ependymoma, Moleculin would be eligible to receive a voucher that may be redeemed to receive priority review for a subsequent marketing application for a different product candidate or which could be sold or transferred.

About WP1066

WP1066 is an immune/transcription modulator capable of directly inhibiting certain key oncogenic transcription factors, including the activated form of a protein known as STAT3. The activated form of STAT3 referred to as p-STAT3, is considered a master regulator of tumor activity. In addition to inhibiting p-STAT3 and several other signaling proteins linked to tumor development, WP1066 has also been shown in animal models to stimulate a natural immune response and immune memory to fight tumor progression.

On April 14, 2021 Bio-Techne Corporation (NASDAQ: TECH) reported a license agreement for use of a proprietary Bio-Techne antibody by Xencor, Inc., a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies and cytokines for the treatment of cancer and autoimmune diseases, for its therapeutic development pipeline (Press release, Bio-Techne, APR 14, 2021, View Source [SID1234578017]).

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Bio-Techne is a global life sciences company providing innovative tools and bioactive reagents for the research and clinical diagnostic communities. Bio-Techne is a preferred partner for many pharma and biotech companies who are developing antibody-based therapeutics for a variety of pathologies. Having access to a robust portfolio of antibodies for these coveted targets, as well as an extensive collection of unreleased monoclonal antibody libraries available for screening in novel applications, makes these valuable partnerships possible. This is Bio-Techne’s third licensing agreement with Xencor, a leader in protein engineering in the immuno-oncology field. Under the terms of this agreement, Bio-Techne grants Xencor access to a proprietary Bio-Techne antibody for use with their proprietary XmAb protein engineering technology, which is revolutionizing the development of new cancer therapeutics such as bispecific antibodies and engineered cytokines.

"We are extremely excited about this agreement with Xencor. Our goal for antibody development is to create highly specific antibodies against important therapeutic targets. This additional licensing agreement with Xencor is a perfect example of the value our vast antibody portfolio brings to the biopharma industry," stated Dave Eansor, President of Bio-Techne’s Protein Sciences Segment. "We are proud of our long history of being the partner of choice for therapeutic antibody discovery and our innovative antibody discovery platform that is harnessed by our pharma customers to fast-track their therapeutic programs. With this license agreement, Bio-Techne will increase its presence as a key player in the development of the next generation of immunotherapies."

On April 14, 2021 PsiOxus Therapeutics, Ltd. (PsiOxus), a clinical stage oncology company ​re-programming ​the tumor microenvironment to overcome the central challenge of resistance to therapy, and bluebird bio, Inc. (Nasdaq: BLUE) reported that preclinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021 (Press release, bluebird bio, APR 14, 2021, View Source [SID1234578016]). The study, "T-SIGn cancer gene therapy and anti-EGFR CAR-T cells synergize in combination therapy to clear A549 lung tumor xenografts", assessed anti-tumor synergy between PsiOxus’ T-SIGn vectors and an anti-epidermal growth factor receptor (EGFR) chimeric antigen receptor (CAR)-T cell therapy from bluebird bio in xenograft lung tumors.

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"We are encouraged by these results showing synergistic activity between our T-SIGn vector and bluebird bio’s CAR-T therapy in primary and metastatic solid tumors and are particularly excited by the ability of a single IV cycle of T-SIGn to enable an otherwise non-effective dose of CAR-T cells to clear both primary and metastatic tumors," said John Beadle, M.D., Chief Executive Officer, PsiOxus. "These data validate the potential of our T-SIGn platform to reprogram the tumor microenvironment such that CAR-T cells are recruited, activated and sustained within solid tumors to yield efficacy. We look forward to further evaluating our T-SIGn vector programs in combination with T-cell therapeutics that otherwise fail to meet their true potential to treat solid cancers."

"Cracking the solid tumor code will likely require layers of technology to reach the types of deep and durable response we aspire to as a field," said Philip Gregory, chief scientific officer, bluebird bio. "This collaboration between bluebird and PsiOxus is an example of this layered strategy, combining the power of two orthogonal anti-tumor approaches to achieve a synergistic impact on tumor control and clearance."

"Although it’s early in development, we believe that this is one of the most robust data sets so far showing that an IV administered therapy is able to rewire the tumor microenvironment to enable recruitment and activation of CAR-T cells and expand the efficacy of CAR-T therapies to solid tumors, including metastases," said Brian Champion, Ph.D., Chief Scientific Officer, PsiOxus. "Results of this study validate the mechanistic foundation of our T-SIGn platform and provide a blueprint of what can be achieved as we evaluate our T-SIGn vectors in combination with additional cell therapy approaches."

The study showed that when injected intravenously into mice with established human tumor xenografts, PsiOxus’ armed T-SIGn vectors reprogrammed the tumor microenvironment to a proinflammatory state. When this was followed three days later by a dose of bluebird bio’s anti-EGFR CAR-T cells, two T-SIGn vectors armed with different T-cell recruitment and activation transgenes, NG-347 and NG-641, synergistically yielded anti-tumor activity. The anti-EGFR CAR-T cells demonstrated no tumor efficacy when dosed alone at the same dose and also failed to synergize with the unarmed T-SIGn vector, demonstrating that the synergy was driven by the T-SIGn vectored transgene expression within the tumor. Transcriptional analysis showed that PsiOxus’ NG-347 T-SIGn vector reprogrammed the tumor microenvironment leading to enhanced activation of CAR-T cells through robust CAR-T and innate immune cell recruitment and activation, resulting in increased efficacy against both primary and metastatic tumors.

While NG-347 is a preclinical program in the IND enabling phase, NG-641 is already being evaluated in the clinic and PsiOxus plans to develop it both as a monotherapy and in combination with checkpoint inhibitors.

"This preclinical data opens up an additional valuable combination option for NG-641 to enable the effective treatment of a wide range of solid tumors with CAR-T or other cell therapies," concluded Brian Champion, Ph.D., Chief Scientific Officer, PsiOxus.

The poster, "T-SIGn cancer gene therapy and anti-EGFR CAR-T cells synergize in combination therapy to clear A549 lung tumor xenografts", is available to registered participants of the AACR (Free AACR Whitepaper) Virtual Annual Meeting 2021 and can be downloaded from the PsiOxus website.