On January 29, 2020 Genprex, Inc.("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company utilizing a unique, non-viral proprietary platform designed to deliver tumor suppressor genes to cancer cells, reported that independent researchers reported in a recent study that TUSC2, a tumor suppressor gene and the active agent in Genprex’s Oncoprex immunogene therapy, is a potential target and biomarker for thyroid carcinoma (Press release, Genprex, JAN 29, 2020, View Source [SID1234553649]). Genprex has no affiliation with these researchers.

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Published in the International Journal of Molecular Sciences, the study reports that TUSC2 overexpression decreased thyroid cancer proliferation, migration and invasion. Cell proliferation, migration and invasion ability are essential steps in tumor metastasis. TUSC2 forced expression reduced thyroid cancer cell proliferation and could represent an important tool to arrest cancer cell proliferation, while TUSC2 restoration decreased the migration and invasion of thyroid cancer cell lines.

The study also found that TUSC2 increased sensitivity to apoptosis by increasing the SMAC/DIABLO and Cytochrome C proteins, which play major roles in apoptosis. TUSC2 forced expression increased these protein levels, and, inversely, the silencing of TUSC2 induced resistance to apoptosis.

Based on the results of the study, researchers concluded that TUSC2 is negatively associated with thyroid cancer aggressiveness and, thus could be a novel target and biomarker for thyroid cancer therapy.

"We continue to be encouraged by data resulting from studies conducted at multiple research institutions suggesting that TUSC2 may be an effective treatment for many types of cancer, now including thyroid cancer," said Rodney Varner, Genprex’s Chairman and Chief Executive Officer.

The authors further state that thyroid carcinoma is the most common endocrine cancer and includes many different forms. Anaplastic thyroid carcinoma (ATC) is the rarest but most lethal subtype. ATC patients usually present a rapidly enlarging neck mass, a high rate of distant metastases and approximately 95 percent mortality at six months. Conversely, papillary thyroid carcinoma (PTC), the most common type of thyroid cancer, is generally characterized by good outcomes, as it is highly curable by surgery and radioiodine therapy. However, some PTC patients have an aggressive disease and can develop distant metastasis.

The same researchers have previously reported that TUSC2 is downregulated in almost all ATC samples and in the vast majority of PTC samples, suggesting TUSC2’s important role in thyroid cancer progression. In 2019, an estimated 50,000 patients in the U.S. were diagnosed with thyroid cancer.Genprex is conducting clinical and pre-clinical research to evaluate the effectiveness of TUSC2 when combined with targeted therapies and immunotherapies for non-small cell lung cancer. Existing pre-clinical data also suggest that TUSC2 may be effective against breast cancer, small cell lung cancer, glioblastoma, head and neck cancer, kidney cancer, and bone and soft tissue sarcomas. This new independent study raises the possibility that TUSC2 may also be used to treat thyroid cancer.

On January 29, 2020 Stony Brook University reported When prostate cancer spreads it is often a deadly disease, but now a Stony Brook University-led research team believes a new approach that inhibits a specific fatty acid binding protein (FABP) may be the key to halting disease progression (Press release, Stony Brook University, JAN 29, 2020, View Source [SID1234553648]). Their research results on FABP5 inhibitors as promising therapeutic agents against metastatic prostate cancer led to a new five-year grant from the National Cancer Institute (NCI) totaling $4.2 million to advance the research to 2025.

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Lead investigator Iwao Ojima, PhD, Distinguished Professor of Chemistry and Director of the Institute of Chemical Biology and Drug Discovery (ICB&DD), along with colleagues at Stony Brook and Cold Spring Harbor Laboratory, in collaboration with Artelo Biosciences, are investigating FABPs in the treatment of inflammation, pain and against certain cancers, as drug targets themselves or in combination with current chemotherapy treatments.

Dr. Ojima and Martin Kaczocha, PhD, (ICB&DD) received a seed grant called a "Fusion Award" from the Renaissance School of Medicine at Stony Brook University to conduct a preliminary study of FABP5 and its inhibitors as potential prostate cancer therapeutic target and agents. Their original work and collaboration with Lloyd Trotman, PhD, a Professor at Cold Spring Harbor Laboratory, led to a paper in The Prostate that showed novel FABP5 inhibitors developed by the ICB&DD team exhibited significant cytotoxicity against highly drug-resistant metastatic prostate cancer cells. FABP5 also enhanced the antitumor effects of taxene drugs in animal models. These promising findings led to the acquisition of the NCI grant.

"This grant is a tremendous example of how collaborative research involving Stony Brook University, Cold Spring Harbor Laboratory and industry have pushed forward a bioscience concept initially supported by seed money from our school to a level where the National Cancer Institute sees its potential as a new and better treatment for metastatic prostate cancer," said Kenneth Kaushansky, MD, Vice President for Health Sciences and Dean of the Renaissance School of Medicine at Stony Brook University.

Under the grant, the team will leverage structure-based drug design and chemical synthesis approaches to identify best FABP5 inhibitors for potency and selectivity, employ a robust in vitro inhibitor testing platform, and access the efficacy of candidate inhibitors in mouse models. They will also investigate FABP5 inhibitors when used as monotherapies, as well as in combination with FDA approved drugs.

"This award highlights the outstanding research capabilities and innovative drug designs that have been created at the ICB & DD under Professor Ojima’s leadership," said Richard J. Reeder, PhD, Vice President for Research at Stony Brook University. "The collaborative work also demonstrates how such partnerships benefit Stony Brook’s growing focus on cancer research."

"We expect to continue the momentum of breakthroughs with our cancer research enterprise," said Yusuf Hannun, MD, Director of the Stony Brook University Cancer Center. "This work is at the frontier of driving novel cancer therapeutics, a major goal for the Stony Brook Cancer Center. This expansion of the research by Dr. Ojima and his colleagues with new federal funding is the type of progressive work we hope sets the bar toward our NCI cancer center designation and impacts patient care in the near future."

Taxanes are used to treat metastatic prostate cancer. While they can be effective, tumors often build up resistance to these drugs. Patients also often experience adverse effects to these drugs, which results in termination of treatment. The emergence of FABP5 inhibitors could be a potential solution to treating advanced disease more effectively and minimize adverse effects.

"In our research, neither docetaxel or cabazitaxel alone was able to eradicate prostate cancer cells in vitro, while combinations of taxanes with FABP5 inhibitors resulted in complete prostate cell death with synergism at very low concentrations of taxanes," said Dr. Ojima.

"While the FABP5 inhibitors produced limited or no cell cytotoxicity in noncancerous cells, they achieved near-complete cell death in the prostate cancer cell lines used in our study, which suggests the FABP5 inhibitors would have much fewer side-effects compared with the current taxane/steroid-based therapy," added Martin Kaczocha, PhD, Assistant Professor, Department of Anesthesiology and co-leader of the collaborative team.

To develop the FABP5 inhibitors for testing against prostate cancer, Dr. Ojima and Dr. Kaczocha are collaborating with Robert Rizzo, PhD, Professor, Department of Applied Mathematics and Statistics, and Trotman, who has developed a unique and highly promising mouse model for metastatic prostate cancer.

They will also collaborate with scientists at Artelo to develop leading FABP inhibitors against prostate cancer.

In 2018, Artelo entered into an exclusive license with the Research Foundation for the State University of New York to the intellectual property of FABP inhibitors for the modulation of the endocannabinoid system for the treatment of pain, inflammation and cancer.

On January 29, 2020 Eli Lilly and Company (NYSE: LLY) reported that the U.S. Food and Drug Administration (FDA) has granted priority review for the New Drug Application (NDA) for selpercatinib (LOXO-292), for the treatment of patients with advanced RET fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer (Press release, Eli Lilly, JAN 29, 2020, View Source [SID1234553646]). The NDA is based on data from the LIBRETTO-001 Phase 1/2 trial in RET-altered lung and thyroid cancers. The FDA has filed the NDA and set a Prescription Drug User Fee Act (PDUFA) date in the third quarter of this year.

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"We are pleased the FDA granted priority review status for the NDA for selpercatinib. This represents an important step toward providing a new precision therapy for people living with certain RET-driven cancers," said Anne White, president of Lilly Oncology. "Combined with the recent opening of our two Phase 3 selpercatinib clinical trials, we are thrilled with the positive momentum of this program and hope to deliver a practice-changing treatment to patients with RET-driven cancers as soon as possible."

In previous regulatory actions, based on early data from the Phase 1/2 LIBRETTO-001 trial, the FDA granted selpercatinib Breakthrough Therapy Designation for treatment in people with:

Metastatic RET-fusion-positive NSCLC who require systemic therapy and have progressed following platinum-based chemotherapy and an anti-PD-1 or anti-PD-L1 therapy;
RET-mutant MTC who require systemic therapy, have progressed following prior treatment and have no acceptable alternative treatment option; and
Advanced RET-fusion-positive thyroid cancer who require systemic therapy, have progressed following prior treatment and have no acceptable alternative treatment options.
In 2019, selpercatinib received orphan drug designation for the treatment of RET fusion-positive NSCLC and for the treatment of RET fusion-positive and RET-mutant thyroid cancers including poorly differentiated thyroid cancer, undifferentiated or anaplastic thyroid cancer, MTC and locally advanced or metastatic follicular or papillary thyroid cancer.

In December of 2019, Lilly opened two selpercatinib Phase 3 trials: LIBRETTO-431 for patients with treatment-naïve RET fusion-positive NSCLC, and LIBRETTO-531 for patients with treatment-naïve RET-mutant MTC. Each trial will enroll 400 patients.

About RET-Driven Cancers
Genomic alterations in RET kinase, which include fusions and activating point mutations, lead to overactive RET signaling and uncontrolled cell growth. RET fusions have been identified in approximately 2 percent of NSCLC, 10-20 percent of papillary and other thyroid cancers and a subset of other cancers. Activating RET point mutations account for approximately 60 percent of MTC. RET fusion cancers and RET-mutant MTC are primarily dependent on this single activated kinase for their proliferation and survival. This dependency, often referred to as "oncogene addiction," renders such tumors highly susceptible to small molecule inhibitors targeting RET.

About LIBRETTO-001
The LIBRETTO-001 Phase 1/2 trial was the largest clinical trial of patients with RET-driven cancers treated with a RET inhibitor. The trial included a dose escalation phase (Phase 1) and a dose expansion phase (Phase 2). The Phase 2 portion of the trial had a primary endpoint of objective response rate (ORR) and secondary endpoints of duration of response (DoR), progression free survival (PFS) and safety. Results from the NSCLC population were presented at the 2019 IASLC World Congress on Lung Cancer (WCLC), while results from the thyroid populations were presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Congress.

About LIBRETTO-431
LIBRETTO-431 is a randomized Phase 3 clinical trial of patients with treatment-naïve RET fusion-positive NSCLC. The trial will enroll 400 patients with advanced or metastatic RET fusion-positive NSCLC who have received no prior systemic therapy for metastatic disease. Enrolled trial participants will be randomized 1:1 to receive either selpercatinib or platinum-based (carboplatin or cisplatin) and pemetrexed therapy with or without pembrolizumab as initial treatment of their advanced or metastatic RET fusion-positive NSCLC. RET fusions may be identified using local testing. This trial’s efficacy endpoints are PFS, overall survival (OS), ORR, DoR, and intracranial ORR. For patients randomized to the control arm, crossover is allowed at progression.

About LIBRETTO-531
LIBRETTO-531 is a randomized Phase 3 clinical trial of patients with treatment-naïve RET-mutant MTC. The trial will enroll 400 patients with advanced or metastatic RET-mutant MTC who have received no prior systemic therapy for metastatic disease. Enrolled trial participants will be randomized 2:1 to receive either selpercatinib or physician’s choice of cabozantinib or vandetanib as initial treatment of their advanced or metastatic RET-mutant MTC. RET mutations may be identified using local testing. This trial’s efficacy endpoints are PFS, treatment failure-free survival (TFFS), OS, ORR, and DoR. For patients randomized to the control arm, crossover is allowed at progression.

About Selpercatinib (LOXO-292)
Selpercatinib (LOXO-292) is a highly selective and potent, oral investigational new medicine in clinical development for the treatment of patients with cancers that harbor abnormalities in the rearranged during transfection (RET) kinase. RET fusions and mutations occur across multiple tumor types with varying frequency. Selpercatinib was designed to inhibit native RET signaling as well as anticipated acquired resistance mechanisms.

On January 29, 2020 DelMar Pharmaceuticals, Inc. (Nasdaq: DMPI) ("DelMar" or the "Company"), a biopharmaceutical company focused on the development of new solid tumor cancer therapies, reported the publication of previously released interim clinical data in the February 2020 issue of peer-reviewed journal, Glioma (Press release, DelMar Pharmaceuticals, JAN 29, 2020, View Source [SID1234553645]). The article highlights results from the first 22 patients of the Company’s ongoing Phase 2 clinical study investigating the first-line treatment of VAL-083 with radiation therapy in newly-diagnosed, MGMT-unmethylated glioblastoma multiforme (GBM). The article can be accessed online via View Source;year=2019;volume=2;issue=4;spage=167;epage=173;aulast=Guo.

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In the article, which was co-authored by lead investigator Professor Zhong-ping Chen of Sun Yat-sen University Cancer Center (SYSUCC) in Guangzhou, China, where the study is currently being conducted, of the 22 patients who had completed at least one cycle of treatment as of that date, median progression-free survival (PFS) with VAL-083 as reported was 9.9 months (confidence interval, or CI, 7.3-12.0 months). For the 18 patients initially receiving the intended treatment dose (30 mg/m2/day on days 1, 2 and 3 of a 21-day cycle) median PFS as reported was 10.4 months (CI 6.0-12.0 months). While this is not a head-to-head trial, historically, temozolomide (TMZ) has been demonstrated to have 6.9 months PFS in unmethylated GBM patients.

"This publication represents an important additional validation of VAL-083 as a first-line treatment. Given that an external panel of experts reviewed both the protocol and the interim data and were willing to publish based on the results and inclusive of the fact that the data came from a study originating in China. This publication is from a world-renowned journal and we believe it demonstrates the potential to achieve one of the first new treatments for GBM in many years. We look forward to providing continued updates on the development of VAL-083 as they become available," commented Saiid Zarrabian, DelMar’s Chief Executive Officer.

The Phase 2 trial is a single-arm, open-label study testing VAL-083 in combination with standard radiotherapy in GBM patients who have an unmethylated promoter of the methylguanine DNA-methyltransferase (MGMT) gene. The clinical trial in newly-diagnosed GBM patients is designed to determine if first-line treatment with VAL-083 plus radiotherapy can provide improvements over the historical efficacy of standard of care TMZ plus radiotherapy. In the publication, authors outlined the two parts of the study. The first is a dose escalation and induction format to enroll up to ten patients to receive VAL-083 at 20, 30 or 40 mg/m2/day for three days every 21 days concurrently with standard radiation and VAL-083 for up to eight additional cycles. The second part of the study is an expansion phase to enroll up to 20 additional patients. The publication highlighted the first 23 patients enrolled, 14 of whom had been treated in the expansion phase.

Myelosuppression was the most common adverse event in the patients assessed, and pharmacokinetic assessments indicated that the levels of VAL-083 were as high in the cerebrospinal fluid as in plasma two hours after infusion.

About VAL-083

VAL-083 (dianhydrogalactitol) is a "first-in-class", bifunctional DNA-targeting agent that introduces inter-strand DNA cross-links at the N7-position of guanine leading to DNA double-strand breaks and cancer cell death. VAL-083 has demonstrated clinical activity against a range of cancers including GBM and ovarian cancer in historical clinical trials sponsored by the U.S. National Cancer Institute (NCI). DelMar has demonstrated that VAL-083’s anti-tumor activity is unaffected by common mechanisms of chemoresistance, including MGMT, in cancer cell models and animal studies.

On January 29, 2020 Cerus Corporation (Nasdaq: CERS) reported the pricing of a registered underwritten public offering of its common stock for proceeds of approximately $55.0 million, before deducting estimated offering expenses payable by Cerus (Press release, Cerus, JAN 29, 2020, View Source [SID1234553644]). The offering is expected to close January 31, 2020, subject to customary closing conditions. All of the shares sold in the offering were sold by Cerus. In addition, Cerus has granted the underwriter a 30-day option to purchase up to an additional $8.25 million of shares of its common stock. On January 28, 2020, the last sale price of the shares as reported on The Nasdaq Global Market was $4.15 per share.

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BTIG, LLC is acting as sole book-running manager for the offering.

A shelf registration statement on Form S-3 (File No. 333-219727) relating to the shares of common stock described above was previously filed by Cerus with the Securities and Exchange Commission ("SEC") and declared effective on January 8, 2018. This offering is being made solely by means of a prospectus supplement and accompanying prospectus included in the registration statement. A final prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website located at View Source Alternatively, copies of the final prospectus supplement, when available, and the accompanying prospectus may be obtained by contacting BTIG, LLC at 65 East 55th Street, New York, NY, 10022, by email at [email protected] or by telephone at (212) 593-7555.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification of these securities under the securities laws of any such state or jurisdiction.