GERMANTOWN, Md.—South Korean biotechnology company RNL BIOhas filed for U.S. Food and Drug Administration (FDA) approval to begin a PhaseII clinical trial for its new product, RNL-JointStem, for the treatment of osteoarthritis.
RNL-JointStem is an adipose-derived adult stem cell productintended for the treatment of osteoarthritis. It is designed to be administeredin a single injection of a stem cell product derived from the patient's own fattissue to effectively regenerate cartilage, reduce pain and improve jointfunction in degenerative arthritis cases.
Phase I and Phase II clinical trials of RNL-JointStem havealready been completed in South Korea under the authority of the Korean Foodand Drug Administration (KFDA). Dr. Jason Dragoo of Stanford University and Dr.David Alan Fisher of Indiana University acted as reviewers and completedrevisions of the protocol currently being evaluated by the FDA.
RNL BIO plans to conduct its double-blind, randomized, positive-controlPhase II clinical trial during the third quarter of 2013 in Sugarland, Texas.The clinical trial will compare the efficacy of RNL-JointStem against existingtherapies based on hyaluronic acid. The patient population for this trial willinclude osteoarthritis patients whose doctors nominate them for participationbased on meeting candidacy requirements.
The intended market for RNL-JointStem is osteoarthritispatients under 60 years old, a patient group for which there is often noeffective treatment and whose quality of life stands to improve greatly witheffective management of the disease.
Osteoarthritis is the most common type of arthritis and theleading cause of chronic disability in the United States, according to theCenters for Disease Control and Prevention, afflicting nearly 27 millionAmericans. The prevalence of osteoarthritis, the aging population and thelimits of current treatment methods indicate that the market for an effectiveand minimally invasive product for this indication would be enormous.
Osteoarthritis, also called degenerative arthritis, is adebilitating and progressive disease characterized by the degradation of thecartilage and bones of the joints. There is no cure for the disease. Cartilagehas a limited capacity for healing and a poor ability to regenerate naturally.Current treatment courses usually begin conservatively with exercise, lifestylechange and pain relievers. As the disease progresses and the pain becomesdebilitating, more drastic and invasive procedures including cortisoneinjections, osteotomy (bone realignment) and joint replacement may beconsidered.
Although the underlying causes of degenerative arthritis arenot completely mapped, injuries, age and heredity are widely considered to bemajor contributing risk factors.
The key advantage RNL-JointStem has demonstrated in previoustrials is its effectiveness as a single-injection treatment, which is vastlysimpler than the enduring lifestyle changes used to manage the disease in itsearly stages, and far less invasive than the late-stage treatment optionsavailable today.
"We are hoping to confirm that RNL-Joint Stem is effectivefor cartilage regeneration, pain reduction and improved joint function inpatients with osteoarthritis, and that it will eventually be accepted in themarket." Dr. Jeong-Chan Ra, CEO and chairman of RNL BIO, said in a mediarelease.
RNL BIO's application for RNL-JointStem is in the form of anInvestigational New Drug (IND) application. Approval of an IND application isrequired to conduct a clinical trial in the United States. This applicationcontains data from preclinical studies as well as information about how theproduct is intended for use clinically, plus an overall risk-benefitassessment. Studies of clinical treatments with biologic medical products,including stem-cell therapies, are closely regulated by the FDA's Center forBiologics Evaluation and Research.
RNL BIO is a biotechnology company focused on stem cells andthe development of autologous cell therapies for various degenerative, ischemicand other indications. Its focus is on research and development ofadult-derived stem cell technologies. The company has completed a Phase I trialfor spinal cord injury and a Phase II clinical trial for osteoarthritis underthe KFDA in South Korea, and it is nearing completion of a clinical trial forBuerger's Disease.