Dive Brief:
- The Food and Drug Administration has given a thumbs up to Virginia-based biotech Diffusion Pharmaceuticals to begin its Phase 3 trial in newly diagnosed patients with inoperable glioblastoma multiforme (GBM). Diffusion has announced that the trial could begin as early as the end of 2017, pushing its share price up to a peak of $2.97 and closing up 20% at $1.94.
- The trial will include 236 patients at 100 sites across the U.S. and Europe, who will receive Diffusion's lead drug trans sodium crocetinate (TSC) along with standard-of-care (chemotherapy and radiotherapy). The primary endpoint is overall survival at two years, compared with standard-of-care alone.
- In a Phase 2 proof-of-concept study, patients with inoperable GBM who were given TSC along with standard-of-care had an almost four-fold increase in survival at two years compared with standard-of-care alone.
Dive Insight:
"Due to their poor prognoses, inoperable patients are often excluded from GBM clinical trials and have usually been treated with radiation and chemotherapy only. We are excited to bring a new treatment possibility to patients with inoperable GBM, and look forward to beginning the trial," said David Kalergis, CEO of Diffusion Pharmaceuticals.
Glioblastoma multiforme is an aggressive and fast-growing brain cancer that makes up around half of all primary brain tumors and affects two or three people in every 100,000. Only around a fifth of glioblastoma patients survive more than one year, and only 3% will survive more than three years.
Glioblastomas have become a common word in the mainstream lexicon of late, after Senator John McCain, R-AZ, announced earlier this year that he was diagnosed with a form of the brain cancer. The senator has surgery to remove his tumor in July, but announced in September that doctors had given him "a very, very poor prognosis."
There is a handful of companies working on this hard-to-treat and devastating cancer, taking a number of different approaches. Novogen is planning a Phase 2 trial of a PI3K/Akt inhibitor, GDC-0084, which was licensed in from Genentech. Kadmon's oral tyrosine kinase inhibitor tesevatinib is in Phase 2 in glioblastoma. The combination of Tesari's PARP inhibitor Zejula (niraparib) or Mateon Therapeutics' CA4P and Avastin (bevacizumab) also both have potential in glioblastoma.
TSC aims to reverse resistance to radio- and chemotherapy by re-oxygenating tumor tissue in order to combat tumor hypoxia. Because of its mechanism of action is likely to have potential with a number of different therapeutic approaches. Phase 2 clinical trials are also planned for TSC in pancreatic cancer and brain metastases, dependent on funding or collaboration.