Innova Therapeutics Receives Rare Pediatric Disease Designation from the FDA

Dr. Nancy DeMore, surgical oncologist and vice chair of entrepreneurship in the Department of Surgery

Innova Therapeutics Inc., a biotechnology company dedicated to developing innovative cancer therapies for patients who have inadequate treatment options, announced today that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for IVT-8086 for the treatment of osteosarcoma.

MUSC Hollings Cancer Center researcher Nancy Klauber-DeMore, M.D., who is the co-founder and chairman of the Scientific Advisory Board for Innova Therapeutics, based in Charleston, South Carolina, was instrumental in the development of the research that led to the therapy. IVT-8086 is a humanized monoclonal antibody (mAb) with a high affinity receptor for secreted frizzled-related protein 2 (SFRP2) and is believed to be the only SFRP2 antagonist in development.

Under the Creating Hope Act, passed into federal law in 2012, the FDA grants rare pediatric disease designation for serious and life threatening diseases that primarily affect children ages 18 years or younger and includes fewer than 200,000 people in the U.S.

Robert Ryan, Ph.D., president and chief executive officer of Innova Therapeutics, said the FDA’s rare pediatric disease designation for IVT-8086 for the treatment of osteosarcoma highlights the significant unmet medical needs of patients with this devastating and life-threatening disease.

“Receiving rare pediatric disease designation from the FDA is a significant milestone for this program and underscores the critical value of our work,” Ryan said. “The entire Innova team is encouraged by this designation and will continue to work diligently toward determining the efficacy and registration for IVT-8086 for the treatment of osteosarcoma.”

Klauber-DeMore, who holds the BMW Endowed Chair in Cancer Research in the Department of Surgery at MUSC, said the FDA designation of their drug is a huge break through for osteosarcoma research.

“This designation is an enormous validation of the work that we’ve been doing. It’s really exciting because there have been no breakthroughs in osteosarcoma treatments in decades,” she said. “IVT-8086 has the potential to become the first FDA-approved therapy for individuals with osteosarcoma in over 30 years.”

Osteosarcoma is a primary malignant bone tumor that generally arises in the long bones and, more rarely, in soft tissues.

Osteosarcoma affects approximately 40,000 Americans,predominantly children, teenagers and young adults. It is a highly metastatic cancer wherein up to 20% of patients have detectable lung metastasis upon initial diagnosis, with a greater incidence in pediatric patients compared to adult patients, and 40% of patients develop lung metastasis in later stages.

“We’re passionate about trying to move the field forward and hopeful that we can find something to improve survival for these patients,” Klauber-DeMore said.

Written by Dawn Brazell