Orphan product designation is intended to provide incentives to encourage companies to pursue cures and treatments for rare diseases with high unmet medical needs.
Under orphan drug status, PAT-SC1 qualifies for potential grant funding during development as well as a period of marketing exclusivity upon FDA approval.
PAT-SC1 has been evaluated for safety and efficacy in a human clinical trial involving gastric cancer patients. The results were encouraging, as patients treated with PAT-SC1 experienced a significant survival benefit compared to a historical control set of patients that received similar treatment but for the PAT-SC1 antibody.
The product is now being converted to Patrys' recently developed large scale manufacturing technology in preparation for a second trial.
The FDA was formally asked to confirm that orphan status is available for PAT-SC1 as produced using Patrys' new manufacturing technology. That confirmation was recently received by Patrys.
Orphan drug designation is available for disease indications where the incidence in the USA is less than 200,000 cases. In the case of gastric cancer in the USA, just over 20,000 new cases per year are reported.
Importantly, while an orphan indication in the USA, the market is much larger as reflected in the fact that over 1,000,000 new cases are reported every year worldwide. The disease is deadly, as the lives of 800,000 gastric cancer patients are lost every year.
These factors present a unique opportunity for Patrys with respect to PAT-SC1, because the product can benefit from the potential for fast track development in the USA. Approval in the USA can then be leveraged globally where the market is more commercially significant.
The conversion of PAT-SC1 to Patrys’ large-scale manufacturing technology is expected to be completed by June 2011. During this period the Company will assess the opportunities to advance PAT-SC1 into a second clinical trial as an internal program or through a larger industry partner.
For further information, please contact:
Patrys Limited: Patrys Media: Daniel Devine Rebecca Wilson Chief Executive Officer Buchan Consulting P: +61 3 9670 3273 P: +61 40 1738 2391 info@patrys.com rwilson@bcg.com.au
About Patrys Limited:
Based in Melbourne, Australia, Patrys (ASX: PAB) is focused on the development of natural human antibodies as therapies for cancer and other major diseases. Patrys has a deep pipeline of anti-cancer natural human antibodies that enable both internal development and partnering opportunities. More information can be found at www.patrys.com.
About PAT-SC1:
PAT-SC1 is a natural human antibody that acts by binding to a special form of a protein, called CD55 that appears on the surface of gastric cancer cells but not on the surface of healthy cells, thereby permitting PAT-SC1 to kill the cancer cells while sparing the healthy cells. PAT-SC1 was evaluated in an investigator led human clinical trial, under which treated patients were dosed with PAT-SC1 48 hours prior to a surgical procedure that involved the removal of the primary tumour (surgical removal of the tumour is currently the standard treatment). Thirty-five PAT-SC1 treated gastric cancer patients showed a statistically significant increased survival benefit compared to a historic control group of 53 patients that received the same surgical treatment but who did not receive PAT-SC1. Additional published data shows that PAT-SC1 at this dose level was very safe and well tolerated by patients receiving the treatment. The referenced clinical results were compiled by an independent research firm.
Gastric cancer:
Gastric cancer can develop in any part of the stomach and may spread throughout the stomach and to other organs; particularly the esophagus, lungs, lymph nodes, and the liver. Stomach cancer causes about 800,000 deaths worldwide per year. Gastric cancer shows a male predominance in its incidence as up to three males are affected for every female. Surgery is the most common treatment and is the often hope of cure for stomach cancer. The surgeon removes part or all of the stomach, as well as the surrounding lymph nodes, with the basic goal of removing all cancer and a margin of normal tissue. Depending on the extent of invasion and the location of the tumor, surgery may also include removal of part of the intestine or pancreas.
