Diffusion Pharmaceuticals Announces Publication of TSC Phase 1/2 Glioblastoma Multiforme Trial Data in the Journal of Neurosurgery

CHARLOTTESVILLE, Va., May 13, 2016 (GLOBE NEWSWIRE) -- Diffusion Pharmaceuticals Inc. (OTCQX:DFFN), a clinical stage biotechnology company focused on the development of novel small molecule therapeutics for cancer, today announced that data from the Phase 1/2 clinical trial evaluating the safety and efficacy of trans sodium crocetinate (TSC) in newly diagnosed glioblastoma multiforme (GBM) has been published online today in the Journal of Neurosurgery (“JNS”), the official publication of the American Association of Neurological Surgeons. As outlined in the JNS article, 36.3% of the full TSC dose patients were alive at two years based on a Kaplan-Meier analysis, compared to reported survival rates for the standard of care (SOC) ranging from 27% to 30%. TSC, the Company’s lead product candidate, is designed to target the tumor’s hypoxic microenvironment, re-oxygenating treatment-resistant tissue and making the cancer cells more susceptible to the therapeutic effects of SOC radiation therapy and chemotherapy.

The article, entitled, “Trans Sodium Crocetinate with Temozolomide and Radiation Therapy for Glioblastoma Multiforme,” outlines the results of a Phase 1/2 trial designed to evaluate the therapeutic effect of adding TSC to radiation therapy (RT) in 59 newly diagnosed GBM patients. In the open, single-arm trial, all patients received SOC RT and temozolomide (TMZ) beginning either after an initial resection or biopsy of the tumor to confirm GBM. TSC was administered at a dosage of 0.25 mg/kg IV around 45 minutes before each RT session three days per week during the 6 weeks of radiation therapy. Considerable tumor shrinkage was observed in many patients, with some tumors disappearing completely.

John L. Gainer, Ph.D., Chief Science Officer of Diffusion Pharmaceuticals, remarked, “The peer-reviewed publication in JNS indicates that the Phase 1/2 trial data are very striking and encouraging. These data strongly suggest that adding TSC during radiation therapy is beneficial for the treatment of GBM. TSC offers a novel, easily-implemented way to combat hypoxia in tumor tissue.”

Summary of Key Findings

  • Overall Survival: Overall survival at two years in the Phase 1/2 trial was 36% greater than the historical standard of 26.5% that was established as the SOC in the 2005 Stupp study.
  • Needle-only biopsy subgroup: Greater survival rates were seen in patients who received only needle biopsies initially, a subgroup thought to fare worse than patients whose tumors have been surgically resected before radiation begins.
  • 73% of the tumors existing at the beginning of radiation therapy decreased in size.
  • Safety: No serious adverse events were associated with TSC in any patient in the Phase 1/2 trial, and no complications arose in combination with TMZ. This suggests that adding TSC to the SOC could also increase patients’ ability to tolerate the TMZ treatment.
  • Quality of Life: Quality of life did not diminish in the Phase 1/2 trial as measured by Karnofsky Performance Status (KPS) and QOL questionnaires.

David Kalergis, Chairman and Chief Executive Officer of Diffusion Pharmaceuticals, said, “The publication is a significant milestone for Diffusion in the clinical advancement of TSC. The Phase 1/2 trial results confirm the therapeutic potential of TSC to overcome treatment resistance by re-oxygenating solid cancerous tumors and improving patient survival outcomes without the addition of harmful side effects. We look forward to continuing the clinical development of TSC as we continue the preparations for the Phase 3 GBM trial and continue discussions with the FDA about trial design for a pivotal trial in pancreatic cancer.”

About Treatment-Resistant Cancers and TSC

Oxygen deprivation at the cellular level (“hypoxia”) is the result of rapid tumor growth, causing the tumor to outgrow its blood supply. Cancerous tumor cells thrive on hypoxia and the resultant changes in the tumor microenvironment cause “treatment-resistance” to radiation therapy and chemotherapy. Using a novel, proprietary mechanism of action, Diffusion’s lead drug TSC counteracts tumor hypoxia – and therefore treatment-resistance – by safely re-oxygenating tumor tissue, thus enhancing tumor kill and potentially prolonging patients’ life expectancy. Oxygen levels of normal tissue remain unaffected upon administration of TSC, thereby avoiding the introduction of harmful side effects.

About Diffusion Pharmaceuticals

Diffusion Pharmaceuticals 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 drug, 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 apparent 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. The U.S. Food and Drug Administration has agreed upon the design of a Phase 3 trial in newly diagnosed GBM. Additional planned studies, subject to receipt of necessary financing, include the Phase 2 trial in pancreatic cancer and a study in brain metastases. Due to its novel mechanism of action, TSC has safely re-oxygenated a range of tumor types in our preclinical and clinical studies. The Company 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. We also believe that TSC has potential application in other indications involving hypoxia, such as stroke and neurodegenerative diseases.

Forward-Looking Statements

To the extent any statements made in this news release deal with information that are not historical facts, 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 continued development of TSC in GBM, pancreatic cancer and possibly other indications, and other statements that are not historical in nature, including those that utilize terminology such as “would,” “will,” “plans,” “possibility,” “potential,” “future,” “expects,” “anticipates,” “believes,” “intends,” “continue,” “estimates,” “targets,” “projects,” “intends,” and similar expressions, derivations of such words and the use of future dates. Forward-looking statements by their nature address matters that are, to different degrees, uncertain and involve both known and unknown risks. These 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; the Company’s ability to satisfy regulatory requirements with respect to its product candidates; the Company’s ability to maintain and defend its intellectual property; the conduct and success of the Company’s clinical trials, including successful enrollment in those trials; the safety and efficacy of the Company’s product candidates; the Company’s ability to retain and recruit qualified personnel; the difficulty of developing pharmaceutical products, obtaining regulatory and other approvals and achieving market acceptance; and the other factors discussed in the Company’s public filings, including the risk factors included in the Company’s most recent Annual Report on Form 10-K. All forward-looking statements in this news release speak only as of the date of this news release and are based on Company management’s current beliefs and expectations. Investors, potential investors and other readers are urged to consider these factors carefully in evaluating the forward-looking statements and are cautioned not to place undue reliance on such forward-looking statements. The Company undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise after the date of this release, except as required by applicable law.

Diffusion Pharmaceuticals Contacts 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

Source:Diffusion Pharmaceuticals, Inc.