Accurexa Earns Small Business Designation from FDA

San Francisco, Calif.-based Accurexa Inc., a biotechnology company focused on developing and commercializing neurological therapies, has qualified under MDUFA (Medical Device User Fee Amendment) as a small business for a reduced or waived fee for medical device submissions to the US. Food and Drug Administration (FDA) made during fiscal year 2015. The company’s small business status expires at the close of business Sept. 30, 2015.

“We submitted our application for small business qualification to the FDA in preparation of the filing of our 510(k) marketing clearance application. We are pleased that the development of our BranchPoint device is advancing towards a regulatory submission,” said George Yu, M.D., the company’s president and CEO.

Accurexa’s Branchpoint device was designed to deliver therapeutics such as stem cells or gene therapy vectors through the radial deployment of a flexible catheter to specific brain target areas through a single brain penetration. The current standard of care is the use of straight, rigid needles, often requiring surgeons to penetrate the brain multiple times for delivering therapeutics, which in turn may increase the risk of bleeding, stroke and reflux of therapeutics back out to the brain surface. An animal study at the University of California, San Francisco demonstrated that the use of a straight needle was associated with reflux of at least 75 percent of the infusion while no reflux was found with the Branchpoint device.

The company is also developing its ACX-31 program which could deliver temozolomide, a chemotherapy drug, directly to brain cancer sites with its BranchPoint device. Temozolomide is a generic, approved, first-line chemotherapy drug that is indicated for the treatment of adult patients with newly diagnosed glioblastoma multiforme concomitantly with radiotherapy, and then as maintenance treatment. Glioblastoma multiforme (GBM) is the most common and most aggressive malignant primary brain tumor in humans. Before temozolomide became generic, it generated U.S. sales of $420 million and global sales of $910 million under its brand name Temodar in 2012. However, current standard of care of delivering temozolomide to tumor sites through oral administration is limited by the blood-brain-barrier and orally administered temozolomide increases patient survival by only 2.5 months from a 12.1 months median overall survival.

Animal studies at the Johns Hopkins School of Medicine have shown that direct local delivery of temozolomide increased concentrations of the drug in the brain threefold and increased survival up to fourfold compared with oral administration. The percentage of long-term survivors for groups receiving local delivery of temozolomide ranged from 25 percent to 37.5 percent while there were no long-term survivors with orally administered temozolomide.

Approximately 16,000 new patients are diagnosed with GBM in the United States each year. Median survival without treatment is 4.5 months. With treatment, concomitant and adjuvant temozolomide chemotherapy with radiation significantly improves, from 12.1 months to 14.6 months, median overall survival in GBM patients, as demonstrated in recent randomized clinical trials.