The market for newly diagnosed brain cancer therapies is in serious need of new innovation – few therapies exist on the market for this indication.
Diffusion Pharmaceuticals, a 13-year-old Virginia biotech in the midst of mid-stage clinical trials for brain cancer, just raised about $3.5 million, according to a regulatory filing.
The company’s most advanced drug candidate, called trans sodium crocetinate, is undergoing Phase II trials at 18 major cancer centers around the country. The compound, which is being tested in conjunction with radiation therapy, has been granted orphan drug designation for newly diagnosed primary brain cancer, or glioblastomas, as well as brain metastasis.
Trans sodium crocetinate’s mechanism of action’s pretty unique – it causes more oxygen to diffuse into hypoxic tumor tissue, without affecting normal tissue, the company says. This is important because glioblastomas contain cells that are resistant to treatments because they have diminished oxygen levels, or hypoxia. Animal studies have found that Diffusion’s compound nearly triples the survival rate of glioblastoma patients when used coupled with standard-of-care radiation therapy.
The company’s Phase 1 trial of 80 patients demonstrated an “outstanding” safety profile, the company said.
Some 13,000 U.S. patients – and 30,000 worldwide – are diagnosed with brain tumors each year. And the market seems to be starved for innovation: “Even a modest improvement in survival… would lead to timely acceptance into the high-margin GBM treatment market,” the company said.
It’s up against only one drug that’s been approved for treating newly diagnosed glioblastoma – temozolomide, a chemotherapy which improves median survival by two months, the company said, and went off patent in 2013.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
“Future competitive advancements in areas such as anti-angiogenesis therapies or vaccines should not displace TSC’s use as long as radiation therapy continues as standard-of-care for cancer treatment,” the company said.
