Innovation Pharma (OTCQB:IPIX) ("the Company"), a clinical stage biopharmaceutical company, is pleased to announce highly encouraging preliminary data from the first patients treated in the Company’s Phase 2a clinical trial (see NCT03042702) of Kevetrin for ovarian cancer. 

Modulation of the p53 protein was observed in response to administration of Kevetrin. Pathways analyses also point to concomitant cell cycle modulation at the level of gene expression. Importantly, these data are the first to directly support in ovarian cancer patient tumors, Kevetrin’s ability to affect p53 and associated molecular pathways—a central gene signaling network involved in regulating cell growth and the cell cycle, helping to prevent cancer.

In more detail, preliminary analyses used Western Blots to assess relative levels of key proteins extracted from tumor biopsies before and after a series of nine Kevetrin infusions administered over three weeks. The level of phospho-p53, the activated form of the protein, in addition to the noted p53 modulation, was also seen to change in response to Kevetrin administration. These findings confirm in patient tumors Kevetrin-induced anti-cancer effects similar to those demonstrated (pdf) preclinically in ovarian cancer cell-lines. These new data reinforce prior clinical data, from the earlier concluded Phase 1 study of Kevetrin in advanced solid tumors (see NCT01664000), in which observations of p21 expression in peripheral blood monocytes supported p53 involvement in Kevetrin’s mechanism of action.

Data from RNAseq analyses of expressed mRNAs and sRNAs in tumors, before and after treatment with Kevetrin, are being further analyzed to assess the nature and scope of molecular pathways modulations. The strongest signal so far detected concerns the cell cycle and a variety of transcription factors.

Running in parallel, the Company is making plans to develop Kevetrin as an oral agent (tablet or capsule) that could be dosed multiple times per day, leveraging its short half-life and pharmacokinetic profile. Bioavailability and other lab studies have been encouraging. Last, linked below is a blog post, published on the Company’s website, further elaborating on Kevetrin’s treatment potential as a novel, p53-modulating anti-cancer drug candidate.

Management Comments

"In a majority of cancers, p53 is mutated, preventing the body from performing its anti-tumor functions," said Leo Ehrlich, Chief Executive Officer at Innovation Pharmaceuticals. "As a result, therapies targeting p53 have long been pursued, highly sought-after by Big Pharma, but have largely been met with limited success due to the inherent complexity of p53.  So, to see Kevetrin modulate p53 in a clinical setting, in tumor biopsies from patients, is an exciting moment for the Company and positioning us at the forefront of developing a potentially transformative anti-cancer therapy."

"Our science team has been working on Kevetrin for over a decade now, studying its unique anti-cancer profile across multiple cancer types, from solid tumors to leukemias—a function of Kevetrin’s ability to induce apoptosis in both wild type p53 and mutant p53 tumors," said Arthur P. Bertolino, MD, PhD, MBA, President and Chief Medical Officer at Innovation Pharmaceuticals. "It’s rewarding to think—after so many years of research in the lab and in the clinical setting—that we now have more direct evidence of how Kevetrin’s multiple mechanisms of action modulate p53, helping to restore the body’s natural ability to fight cancer. Such mechanistic insights of the kind we’re observing will prove invaluable as we continue to advance Kevetrin into later-stage clinical development."

About Ovarian Cancer

Ovarian cancer is a common type of cancer that commonly begins in women’s ovaries and associated tissues. Malignant ovarian tumor cells metastasize either directly through the organs of the pelvis region, or through the bloodstream, or the lymphatic system. The causes of ovarian cancer are still not known, though women over the age of 63 represent more than 50 percent of newly diagnosed cases, with the cancer more frequently found in white women than other ethnicities. Ovarian cancer ranks fifth in cancer deaths among women worldwide. It is estimated that in 2016, in the United States, over 22,000 women will be diagnosed with ovarian cancer, with approximately 14,000 women dying from the disease. A $1.6 billion market, current treatment is often limited to surgery and chemotherapy and there is no cure.

About Kevetrin and p53

Kevetrin is a small molecule that has demonstrated the potential of becoming a breakthrough cancer treatment by modulating p53, a protein frequently referred to as the "Guardian of the Genome" due to its critical role in controlling cell mutations. In the majority of cancers, and regardless of origin, type, and location, the p53 pathway is mutated, preventing the body from performing its natural anti-tumor functions. Conducted at Dana-Farber Cancer Institute and Beth Israel Deaconess Medical Center, a Phase 1 clinical trial (see NCT01664000) of Kevetrin in treating advanced solid tumors has been successfully completed, with patients showing good toleration and encouraging signs of potential therapeutic response (see ASCO 2015, ASCO 2013). Additional pre-clinical work supporting Kevetrin’s anti-cancer activity has recently been presented at scientific conferences (see EHA 2017, AACR 2017). The Company has initiated a Phase 2a trial of Kevetrin (see NCT03042702) in late stage, platinum-resistant/refractory ovarian cancer. Patients will receive more frequent dosing (3 times per week) at higher levels and then receive standard of care treatment after trial conclusion. Efforts also are underway todevelop Kevetrin as an oral anti-cancer agent that can be administered daily, potentially multiple times per day. The FDA has awarded Kevetrin Orphan Drug status for ovarian cancer, pancreatic cancer, and retinoblastoma, qualifying it for developmental incentives and a potential extra 7 years of market exclusivity upon drug approval. The FDA also has granted Kevetrin Rare Pediatric Disease designation for childhood retinoblastoma.

About the Company 
Headquartered in Beverly, Massachusetts, Innovation Pharmaceuticals Inc. (IPI), formerly Cellceutix Corporation, is publicly-traded under the company symbol "IPIX". The Company is clinical stage biopharmaceutical company developing innovative therapies in multiple diseases. The Company believes it has a world-class portfolio of first-in-class lead drug candidates and is now advancing them toward market approval, while actively seeking strategic partnerships. The Company’s Psoriasis drug candidate Prurisol completed a Phase 2 trial and the Company has since conducted a Phase 2b study, with topline results expected in 1Q2018. Prurisol is a small molecule that acts through immune modulation and PRINS reduction. The Company’s anti-cancer drug Kevetrin successfully concluded a Phase 1 clinical trial at Harvard Cancer Centers’ Dana Farber Cancer Institute and Beth Israel Deaconess Medical Center, and the Company has commenced a Phase 2 study in Ovarian Cancer. In the laboratory, Kevetrin has been shown to modulate p53, often referred to as the "Guardian Angel Gene" due to its crucial role in controlling cell mutations. Brilacidin, a defensin mimetic compound, has shown in an animal model to reduce the occurrence of severe ulcerative Oral Mucositis (OM) by more than 94% compared to placebo. The Company has recently completed a Phase 2 clinical trial with its novel compound Brilacidin-OM for the prevention of OM in patients with Head and Neck Cancer; topline results demonstrate a reduction in the incidence of severe OM (WHO Grade ≥ 3). The Company’s lead antibiotic, Brilacidin, has completed a Phase 2b trial for Acute Bacterial Skin and Skin Structure Infection, or ABSSSI. The Phase 2b data showed a single dose of Brilacidin to deliver comparable clinical outcomes to the FDA-approved seven-day dosing regimen of daptomycin. Brilacidin has the potential to be a single-dose therapy for certain multi-drug resistant bacteria ("superbugs"). Results are now available for the completed Phase 2, open label Proof-of-Concept trial, treating patients with Brilacidin for Ulcerative Proctitis/Ulcerative Proctosigmoiditis (UP/UPS), two types of Inflammatory Bowel Disease (IBD). The Company has formed research collaborations with world-renowned research institutions in the United States and Europe, including MD Anderson Cancer Center, Beth Israel Deaconess Medical Center, and the University of Bologna.

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