On May 13, 2016 Diffusion Pharmaceuticals Inc. (OTCQX:DFFN), a clinical stage biotechnology company focused on the development of novel small molecule therapeutics for cancer, reported 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 (Press release, Diffusion Pharmaceuticals, MAY 13, 2016, View Source [SID:1234512374]). Schedule your 30 min Free 1stOncology Demo! 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.
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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.