Sydney, Australia, 27 June 2011; Australian monoclonal antibody company, Immune System Therapeutics Limited (IST), today announced that its breakthrough antibody treatment for terminal blood cancer had received Orphan Drug Designation from US health regulators.
Orphan drug status is granted by the United States Food and Drug Administration (FDA) to drugs that treat rare diseases and to diseases that have few available treatments. It also gives the drug maker marketing exclusivity if approved, during which time competing drug makers cannot enter with rival treatments. In the United States, the effective period of exclusivity for orphan drugs is now 12 years with the potential for an accelerated review process by the FDA.
IST Chief Executive Officer, Mr Alan Liddle, said, “This is another positive step towards achieving our business goal of developing an effective treatment for patients suffering from multiple myeloma. We are making excellent progress towards putting in place a robust global licensing structure that will expedite the clinical and regulatory processes needed to commercialise this promising new medical treatment.”
The FDA granted orphan drug designation for MDX-1097 based on review of the application, which included clinical and preclinical data demonstrating a favorable efficacy and safety profile.
On 17 June, IST announced it would expand the Phase 2 clinical trial of MDX-1097 in Australia following encouraging responses from the first dosed patients and a positive review of safety and efficacy data by an independent Data Monitoring Committee.
The Orphan Drug Designation is the first awarded to IST for its portfolio of antibody platform technologies currently being developed to target and treat a range of other cancers and diseases with major unmet clinical needs.
MDX-1097 is a genetically engineered antibody that binds specifically to a target protein found on the cell surface of some types of blood cancer. Researchers anticipate the antibody will potentially reduce the number of cancerous cells in multiple myeloma patients and improve patient health and wellbeing. Laboratory studies have shown that the antibody works with the patient’s immune system to induce death of their multiple myeloma cancer cells. It was created specifically to treat multiple myeloma blood cancer, a debilitating terminal disease, with an estimated patient population of over 150,000 worldwide. The disease typically occurs in older patients and is responsible for two per cent of all cancer deaths in the United States every year.
About Orphan Drug Designation
Orphan drug designation is reserved for therapies which are being developed for conditions affecting up to 200,000 patients annually in the United States, and allows for an accelerated review process by the FDA, market exclusivity for several years in the United States upon obtaining marketing authorisation, tax benefits, and exemption from user fees.
About Immune Systems Therapeutics
Immune Systems Therapeutics (IST) is a private bio-therapeutic company commercialising a range of promising new antibody drugs to treat human blood cancers and autoimmune diseases. The company’s unique therapies target a range of common blood cancers known as B cell malignancies and autoimmune diseases. IST has extensive and robust patents covering a new class of drugs targeting blood cancers and autoimmune diseases that are potentially safer, less toxic and more effective than current standard chemotherapy treatments. IST’s business plan is to license its drugs to international pharmaceutical companies for regulatory approval, production and sales. For more information please visit www.istl.com.au
For more information please contact:
Chief Executive Officer
Tel: (02) 9514 7434 or 0414 991 770
Tel: (02) 9514 7434 or 0430 352 946