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ApeX Therapeutics and Researchers at Indiana University to Present Phase 1 Clinical Plan for APX3330 and Novel Approach to Addressing Therapeutic Resistance at AACR Pancreatic Cancer Meeting

/ -- INDIANAPOLIS, IN--(Marketwired - May 12, 2016) - ApeX Therapeutics, a biotechnology company focused on developing novel compounds to treat cancer, in collaboration with researchers at Indiana University Simon Cancer Center (IUSCC) today announced two poster presentations at the American Association for Cancer Research (AACR) Pancreatic Cancer: Advances in Science and Clinical Care Meeting to be held in Orlando, FL on May 12-14, 2016.

Pancreatic ductal adenocarcinoma (PDAC) is third leading cause of cancer-related death in the United States and is one of the few cancers for which survival has not improved substantially over nearly 40 years. Pancreatic cancer has the highest mortality rate of all major cancers; 94% of pancreatic cancer patients will die within five years of diagnosis and 74% of patients die within the first year of diagnosis (American Cancer Society: Cancer Facts and Figures 2016). Treatment with chemotherapy has not changed the natural course of this disease, and just recently, with combination of chemotherapeutic agents, the median survival reached a year.

APX3330 is a small molecule that targets the DNA repair protein APE1/Ref-1, a dual function protein involved in both DNA repair and redox-signaling co-activation of oncogenic transcription factors. APE1/Ref-1 regulates multiple transcription factors involved in pancreatic cancer cell survival signaling including HIF-1alfa, STAT3, NFkappaB, AP-1 and NRF2. High expression levels of APE1/Ref-1 also indicate decreased survival in PDAC as well as other cancers.

APX3330 selectively and directly binds to APE1/Ref-1 thus inhibiting its APE1/Ref-1 redox signaling activity. Importantly, while APX3330 blocks APE1/Ref-1's redox function, it has no effect on APE1/Ref-1 endonuclease DNA repair activity.

One of the transcription factors regulated by APE1/Ref-1, HIF-1alfa is a critical factor in hypoxia-induced CA9 transcription, which is proposed to play a role in PDAC therapeutic resistance. Researchers explored the mechanisms underlying PDAC cell responses to hypoxia and modulation of APE1/Ref-1 redox signaling control of HIF-1alfa. Among the findings to be presented at the AACR conference are that APE1/Ref-1 inhibition diminishes hypoxia-induced CA9 transcription and subsequent protein expression and that inhibiting both CA9 activity via a small molecule and CA9 transcription via APE1/Ref-1 inhibition (with APX3330) leads to: acidification of PDAC cells under hypoxic conditions, decreased PDAC cell proliferation under hypoxic conditions, and decreased PDAC cell growth in a 3D tumor model.

APX3330 has been shown in multiple in vivo models of pancreatic cancer to be effective in reducing tumor volume and metastases as both a single agent and in combination with gemcitabine. APX3330 was recently shown to augment 5-flurouracil (5-FU) in a colon cancer xenograft model as well as increased cytotoxicity in colon cancer stem cells (Lou et al 2014).

The safety and oral dosing administration schedule of APX3330 has been previously established through a prior development program that evaluated the preclinical toxicology and phase 1 and phase 2 safety and clinical profile in more than 400 non-cancer patients with active hepatitis C and abnormal liver function tests. These studies demonstrated an apparent lack of significant acute toxicity on neurologic, cardiovascular, or pulmonary function at doses up to 240 mg/day. Gastrointestinal symptoms and symptoms related to skin rash or irritation were identified as adverse events of particular interest.

A Phase 1 dose-escalation study of APX3330 in patients with advanced solid tumors and a dose-expansion cohort of patients with advanced PDAC is being planned. Goals of the study include determination of the maximum-tolerated-dose and recommended Phase 2 dose for APX3330 as a single agent, as well as characterization of preliminary antitumor activity and other exploratory biomarker endpoints.

Mark R. Kelley, PhD., Chief Scientific Founder of ApeX Therapeutics and Betty and Earl Herr Chair in Pediatric Oncology, Associate Director of Basic Science Research, IU Simon Cancer Center at Indiana University School of Medicine, commented: "Our increasing knowledge regarding the impact of APE1/Ref-1 redox signaling as a regulator of multiple key transcription factors continues to support its inhibition as a method of treating pancreatic cancer. We are eager to further explore this treatment paradigm in the clinic."

Dr. Kelley will present two posters at the AACR Pancreatic Cancer: Advances in Science and Clinical Care Meeting:

Title: Clinical Trials Targeting APE1/Ref-1 in Pancreatic Cancer with APX3330
Date: Saturday, May 14, 2016
Time: 1:00 p.m. to 4:00 p.m.
Poster Number: B88

Title: Regulation of HIF1α under Hypoxia by APE1/Ref-1 Impacts CA9 Expression: Dual-Targeting in
Patient-Derived 3D Pancreatic Cancer Models
Date: Saturday, May 14, 2016
Time: 1:00 p.m. to 4:00 p.m.
Poster Number: B51

For more information about the AACR Pancreatic Cancer: Advances in Science and Clinical Care Meeting, please visit:

About Apex Therapeutics
ApeX Therapeutics is a biotechnology company focused on developing novel compounds to treat cancer targeting the multiple functions of the APE1/Ref-1 protein. Our lead drug candidate, APX3330 targets the treatment of pancreatic cancer and we expect to be in human clinical studies in 2016. To learn more about ApeX Therapeutics, please visit the Company's website at

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