Diffusion Pharmaceuticals Inc. (NASDAQ:DFFN), a cutting-edge biotechnology company developing new treatments for life-threatening medical conditions by improving the body’s ability to bring oxygen to the areas where it is needed most, announces increased survival in inoperable glioblastoma patients enrolled in the 19-patient, open-label, dose-escalation lead-in portion of its Phase 3 study with Trans Sodium Crocetinate (TSC) plus standard of care (SOC). John Gainer, Ph.D., the Company’s chief scientific officer, will present details of these findings at the inaugural Glioblastoma Drug Development Summit being held in Boston December 10-11, and sponsored by Hanson Wade. Dr. Gainer’s slide presentation will be posted to the Company’s website at www.diffusionpharma.com immediately prior to the conference.
In an earlier Phase 2 study testing TSC in newly diagnosed glioblastoma multiforme (GBM) brain cancer patients, an almost fourfold increase in 2-year survival was seen versus historical controls in inoperable patients. The current Phase 3 INTACT (INvestigating Tsc Against Cancerous Tumors) study – an open-label, randomized, controlled trial – is designed to examine this finding in a fully powered safety and efficacy registration study, which, if successful, could be the basis for US FDA approval.
In the INTACT trial, subjects are randomized at baseline to SOC for first-line treatment of GBM plus TSC, or to SOC alone. The SOC for GBM is temozolomide plus radiation therapy for 6 weeks, followed by 28 days of rest, then by 6 cycles of post-radiation temozolomide treatment. In a modification to the Phase 2 dosing regimen, patients in the INTACT trial will also receive high-dose TSC during the post-radiation chemotherapy phase.
A 19-patient, open-label, dose-escalation lead-in portion to the INTACT trial was recently completed, sending a positive safety signal across all patients receiving TSC. In addition, six of the seven patients who received the high dose TSC treatment are still alive, with a median survival at the present time of 14.3 months. This is compared with 9.2 months for the historical standard of patients with inoperable GBM. Since six of the TSC-treated patients are still alive, median survival time is actually increasing with the passage of time, suggesting the INTACT trial may confirm or better the efficacy findings seen in the Phase 2 study.
Patients’ abilities to perform their daily activities as measured by Karnofsky performance scores increased from the baseline following completion of high dose treatment with TSC. Investigators have also reported instances of inoperable GBM patients treated with the higher dose TSC regimen leaving hospice or returning to work after treatment in the open-label portion of the study.
“We are encouraged by these early findings showing that patients enrolled in the lead-in portion of the INTACT trial have experienced increased survival with our new protocol,” said Dr. Gainer. “Although final conclusions will depend on the completion of the randomized portion of the trial, we believe that TSC helps to eradicate the low oxygen status of cancerous tumors, and it appears this may also result in a survival benefit compared with the current standard therapy.”
The Company previously announced it is seeking a partner to continue development of TSC in the GBM indication and has begun patient enrollment in its Phase 2 on-ambulance trial with TSC for the treatment of stroke.