Diffusion Pharmaceuticals (DFFN) Launches Phase 3 Trial for Glioblastoma

Edward Kim  |

Glioblastoma is an extremely aggressive central nervous system tumor that forms on astrocytes, the star-shaped cells that form the supportive tissue of the brain. Also known as glioblastoma multiforme (GBM), is the most common grade IV brain cancer, accounting for about 15.4% of all primary brain tumors and about 60-75% of all astrocytomas, according to the American Brain Tumor Association (ABTA). Standard treatment options involve some combination of surgery, radiation and chemotherapy with temozolomide, but GBM brain cancer is usually highly malignant. The ABTA says that median survival for adults with an anaplastic (cells that divide rapidly and have little or no resemblance to normal cells) astrocytoma is about two to three years. For adults with more aggressive glioblastoma, treated with concurrent temozolomide and radiation therapy, median survival is about 14.6 months and the two-year survival rate is 30%.

Diffusion Pharmaceuticals (Nasdaq: DFFN) announced today that it has opened enrollment in its Phase 3 clinical trial of its lead small molecule, trans sodium crocetinate (“TSC”), in patients with newly-diagnosed inoperable GBM brain cancer. The new INTACT trial (INvestigating Tsc Against Cancerous Tumors) follows a previous Phase 2 study in which the inoperable patient subgroup showed a nearly 4x increase in survival compared with historical controls when TSC was added to their treatment regimen (40% alive at two years vs. 10.4%). TSC’s innovative mechanism of action affects the tumor micro-environment, making treatment-resistant cancer cells more susceptible to the tumor-killing power of conventional radiation therapy and temozolomide by re-oxygenation of the hypoxic portion of the tumor. Diffusion believes that a largely intact GBM tumor vasculature with limited surgical resection is conducive to TSC’s tumor re-oxygenation properties, and that this contributed to the survival increase in the Phase 2 GBM inoperable patient subgroup.

The INTACT trial will screen 300 patients and enroll 264 in an effort to ensure that results from 236 patients will be available for analysis. Enrolled patients will be randomized in a 1:1 ratio into treatment groups receiving temozolomide, radiation and TSC and control groups receiving temozolomide and radiation alone. The study will compare overall survival at two years between patients in the two groups. Up to 100 clinical sites in the US and Europe are expected to participate, with enrollment expected to be completed by early 2019.

Given the dire prognosis of inoperable GBM brain cancer, we are especially gratified to have the INTACT clinical trial open for enrollment. We believe that TSC can provide new hope for these patients, whose treatment options are so limited. The four-fold increase in inoperable GBM patients alive at two years in our Phase 2 trial is a particularly strong efficacy signal, and informs the design of our Phase 3 trial.

- David Kalergis, CEO, Diffusion Pharmaceuticals

Clinical Pipeline

Source: Diffusion Pharmaceuticals

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 with hypoxia and the resultant changes in the tumor microenvironment cause the tumor to become resistant to RT and chemotherapy. Using a novel, proprietary mechanism of action, Diffusion’s lead drug TSC appears to counteract tumor hypoxia – and therefore treatment resistance – by safely re-oxygenating tumor tissue, thus enhancing tumor kill and potentially prolonging patient life expectancy. Oxygen levels of normal tissue appear to remain unaffected upon administration of TSC.

Diffusion Pharmaceuticals has been publicly traded since a reverse merger in January 2016 and was uplisted to the Nasdaq Capital Market in November 2016. Investors will have to be patient, as interim safety and efficacy data from the Phase 3 trial isn't expected until 2020, with trial completion in 2021, but the risk-reward proposition seems reasonable at the current price of $1.22. That time horizon makes the stock effectively a long-dated call option on a technology that offers potential treatment to those with GBM brain cancer and other malignancies.

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