Clinical development program in pediatric osteosarcoma will leverage orphan drug status.
May 27, 2014
Advaxis, Inc., a leader in developing cancer immunotherapies, announced that it has been granted Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) for ADXS-cHER2 for the treatment of osteosarcoma.
ADXS-cHER2 is an immunotherapy under investigation for targeting the HER2 receptor, which is overexpressed in certain solid-tumor cancers, including bone cancer and breast cancer. Based on strong pre-clinical and canine osteosarcoma clinical data, Advaxis is planning to initiate a clinical development program with ADXS-cHER2 in pediatric patients with osteosarcoma. Pediatric osteosarcoma affects about 400 children and teens in the U.S. every year, representing a small but significant unmet medical need that has seen little therapeutic advancement in decades. Both veterinary and human osteosarcoma specialists consider canine osteosarcoma to be the most analogous disease to human osteosarcoma.
“The Orphan Drug Designation for ADXS-cHER2 in osteosarcoma is a key value driver for Advaxis and will facilitate our ability to conduct clinical trials to more fully understand the potential for ADXS-cHER2 in osteosarcoma populations,” stated Daniel J. O’Connor, Chief Executive Officer of Advaxis. “The osteosarcoma ODD follows the recent ODD of ADXS-HPV in invasive cervical cancer. These designations highlight the potential of our proprietary immunotherapy platform and the opportunity that it may offer in the treatment of several underserved cancer indications.”
O’Connor continued, “Looking ahead, Advaxis is now planning to initiate a clinical development program in pediatric osteosarcoma with ADXS-cHER2. Given the limited availability of new treatment options for pediatric osteosarcoma, we believe that, subject to regulatory approval and upon completion of successful clinical trials, the potential to be on the market may be accelerated.”
To date, the safety and efficacy of ADXS-cHER2 have been evaluated in an ongoing veterinary clinical study in client-owned (pet) dogs with osteosarcoma, conducted by Dr. Nicola Mason at the University of Pennsylvania School of Veterinary Medicine. In the study, dogs treated with ADXS-cHER2 immunotherapy after the standard of care (amputation and follow up chemotherapy), had a statistically significant prolonged overall survival benefit (p=0.032) compared with dogs that received standard of care without ADXS-cHER2.
About Orphan Drug Designation
Under the Orphan Drug Act (ODA), the FDA may grant Orphan Drug Designation to a drug or biological product intended to treat a rare disease or condition, which means a disease or condition that affects fewer than 200,000 individuals in the United States, or more than 200,000 individuals in the United States, and for which there is no reasonable expectation that the cost of developing and making a drug or biological product available in the United States will be recovered from domestic sales of the product. The benefits of Orphan Drug Designation can be substantial, and include Federal grants, tax credits, and potentially a seven year market exclusivity period once the product is approved, provided that the product is the first product of that type to market for that indication.
About Pediatric Osteosarcoma
Osteosarcoma is the most common type of bone cancer. Most osteosarcomas occur in children and young adults between the ages of 10 and 30 and usually develop in areas where the bone is growing quickly, such as near the ends of long bones. Each year, about 800 new cases of osteosarcoma are diagnosed in the United States. About 400 of these are in children and teens. Osteosarcomas account for about 3% of childhood cancers, but they make up a much smaller percentage of adult cancers, according to the American Cancer Society.
About Advaxis, Inc.
Advaxis is a clinical-stage biotechnology company developing multiple cancer immunotherapies based on its proprietary platform that is being investigated for redirecting the immune system to kill cancer. The Advaxis technology, using bioengineered live attenuated bacteria, is the only cancer immunotherapy shown to actively suppress Tregs and MSDCs, components in the cancer microenvironment that are believed to contribute to tumor growth and protect it from immunologic attack. Advaxis’s lead immunotherapy candidate, ADXS-HPV, is being investigated for targeting human papillomavirus (HPV)-associated cancers and is in clinical trials for three indications: Phase 2 in invasive cervical cancer, Phase 1/2 in head and neck cancer, and Phase 1/2 in anal cancer. The FDA has granted Advaxis Orphan Drug status for each of these three indications. Advaxis plans to initiate a registrational clinical program for cervical cancer in 2014 and has established licensing partners in India and Asia for commercialization in those regions.
Advaxis is also developing, ADXS-cHER2, to target the HER2 receptor, which is overexpressed in certain solid-tumor cancers, including pediatric bone cancer (or osteosarcoma), breast cancer, and gastric cancer. Advaxis is developing ADXS-cHER2 for both human and animal-health, and interim findings in an ongoing clinical study in canine osteosarcoma, an excellent model for human bone cancer, have shown clinical benefits. Further confirmatory studies are planned. Advaxis is pursuing a clinical program in pediatric osteosarcoma and has licensed ADXS-cHER2 and three other non-specified immunotherapy constructs to a major animal-health company.
For more information please visit www.advaxis.com