Diffusion Pharma Achieves Drug Production Milestone For Planned Phase III Clinical Trial In Inoperable GBM Brain Cancer

Published: Jul 20, 2017

CHARLOTTESVILLE, Va., July 20, 2017 (GLOBE NEWSWIRE) -- Diffusion Pharmaceuticals Inc. (NASDAQ:DFFN), a clinical stage biotechnology company developing novel small molecule therapeutics for cancer and other hypoxia-related diseases, today reported the completion of a major production run of its lead drug candidate trans sodium crocetinate (TSC). This drug supply will be used in its planned Phase 3 trial which will test TSC in the treatment of newly diagnosed inoperable glioblastoma (GBM) brain cancer patients and is of sufficient quantity to support the entire trial. Diffusion plans to initiate this clinical trial by the end of 2017.

“Diffusion has reached an important milestone for initiating the Phase 3 trial with this successful manufacture of Phase 3-ready TSC,” said David Kalergis, Diffusion’s Chairman and CEO. “In its End-of-Phase-2-Meeting response, the FDA required that any TSC used in upcoming Phase 3 studies must be made using commercially-ready processes, both for the active pharmaceutical ingredient (API) and formulated drug product. The necessary advanced production and formulation processes were implemented over the last eighteen months and were cleared with the FDA in June, immediately prior to the successful clinical trial supply production run earlier this month.”

Diffusion is now interacting with the FDA on details regarding the design and execution of the planned Phase 3 study, with initiation planned for later this year. The study will focus on newly diagnosed GBM patients who have been judged by their medical team to be inoperable, usually because of the size or location of the tumor. Because of their poor prognosis, these patients are often excluded from participation in other GBM clinical trials. In the Company’s Phase 2 GBM study, the TSC-treated group showed a nearly four-fold increase in survival at two years, when compared to the historical controls.

About Diffusion Pharmaceuticals Inc.

Diffusion Pharmaceuticals Inc. is a clinical stage biotechnology company focused on extending the life expectancy of cancer patients by improving the effectiveness of current standard of care treatments including radiation therapy and chemotherapy. Diffusion is developing its lead product candidate, trans sodium crocetinate (TSC), for use in the many cancer types in which tumor hypoxia (oxygen deprivation) is known to diminish the effectiveness of current treatments. TSC targets the cancer's hypoxic micro-environment, re-oxygenating treatment-resistant tissue and making the cancer cells more vulnerable to the therapeutic effects of treatments such as radiation therapy and chemotherapy, without the addition of any serious side effects.

A Phase 2 clinical program, completed in the second quarter of 2015, evaluated 59 patients with newly diagnosed glioblastoma multiforme (GBM). This open label, historically controlled study demonstrated a favorable safety and efficacy profile for TSC combined with standard of care, including a 37% improvement in overall survival over the control group at two years. A particularly strong efficacy signal was seen in the subset of inoperable patients, where survival of these TSC-treated patients at two years was increased almost four fold over the controls. At an End-Of-Phase 2 Meeting, the U.S. Food and Drug Administration provided Diffusion with extensive guidance for a Phase 3 trial of TSC in newly diagnosed GBM patients, including drug manufacturing and protocol design requirements. Assuming FDA agreement on the final protocol design focusing on inoperable patients, the study is planned for initiation by the end of 2017. Due to its novel mechanism of action, TSC has safely re-oxygenated a range of tumor types in preclinical and clinical studies. Diffusion believes its therapeutic potential is not limited to specific tumors, thereby making it potentially useful to improve standard-of-care treatments of other life-threatening cancers. Additional planned studies include Phase 2 trials in pancreatic cancer and brain metastases, with study initiation subject to receipt of additional funding or collaborative partnering. The Company believes that TSC has potential application in other indications involving hypoxia, such as stroke, neurodegenerative diseases and emergency medicine, and is examining options in these indications.

Forward-Looking Statements

To the extent any statements made in this news release deal with information that is not historical, these are forward-looking statements under the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements about the company's plans, objectives, expectations and intentions with respect to future operations and products, the potential of the company's technology and product candidates, the anticipated timing of future clinical trials and other statements that are not historical in nature, particularly those that utilize terminology such as "would," "will," "plans," "possibility," "potential," "future," "expects," "anticipates," "believes," "intends," "continue," "expects," other words of similar meaning, derivations of such words and the use of future dates. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Uncertainties and risks may cause the company's actual results to be materially different than those expressed in or implied by such forward-looking statements. Particular uncertainties and risks include: general business and economic conditions; the company's need for and ability to obtain additional financing; and the difficulty of developing pharmaceutical products, obtaining regulatory and other approvals and achieving market acceptance. All forward-looking statements in this news release speak only as of the date of this news release and are based on management's current beliefs and expectations. Diffusion undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

David Kalergis
Chief Executive Officer 
Diffusion Pharmaceuticals Inc. 
(434) 220-0718
dkalergis@diffusionpharma.com

Stephanie Carrington 
ICR Inc.
(646) 277-1282
Stephanie.Carrington@icrinc.com

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