Regeneus (ASX:RGS) has enrolled and treated the first patient under the Phase 1 clinical trial of its Progenza off-the-shelf allogeneic stem cell treatment for knee osteoarthritis.
The Safety, Tolerability and Efficacy of Progenza (STEP) trial seeks to evaluate the treatment’s safety and tolerability as well as its effect on knee pain and function.
Progenza is produced from mesenchymal stem cells derived from the adipose (fat) tissue of a healthy donor who has been extensively screened.
The company’s proprietary and scalable manufacturing process is capable of producing millions of therapeutic doses from one donor.
A review of the sentinel patient’s safety data by the study safety oversight committee identified no safety concerns.
Enrolment is now open to the remainder of the first cohort of 10 patients. A second and final cohort of 10 patients will receive a higher dose of cells.
Leading Sydney-based sports medicine specialist, Dr Donald Kuah, is the Principal Investigator on the trial.
Dr Kuah, a partner of Sydney Sportsmed Specialists, has extensive experience in the diagnosis and treatment of patients with osteoarthritis.
The trial will include 20 participants with knee osteoarthritis treated at two different doses of cells.
Participants will receive ultrasound-guided injections of Progenza or placebo directly into their arthritic knee joint.
One in five patients will receive a placebo injection.
The primary objective of the trial is to evaluate the safety and tolerability of Progenza.
The secondary objectives are to investigate the effect of Progenza on knee pain and function; quality of life; knee joint structures using magnetic resonance imaging; and osteoarthritis biomarkers.
Participants will be monitored for 12 months.
In March, the company successfully completed a pre-clinical study at a US-based facility with expertise in conducting preclinical studies that replicate human OA.
The results showed no Progenza-related safety or toxicity issues, even at doses well in excess of the intended human dose.
Further, Progenza-treated knees showed no deterioration from the time of injection, in contrast to the vehicle control group, which continued to deteriorate over the 7-week study.
These study results support the role of Progenza in preventing disease progression.
The start of the Phase 1 STEP trial places Regeneus on the path towards providing a new treatment option for patients with osteoarthritis.
While the key focus will be to monitor Progenza’s safety, the trial will also allow Regeneus to assess the treatment for preliminary efficacy.
Other advantages of Progenza include the use of adipose (fat) tissue as a starting material that is readily available, not genetically modified, and which has the capacity to produce millions of therapeutic doses from a single donor.
Progenza has the potential to address a major market with 250 million people affected worldwide by osteoarthritis, which has no known disease modifying treatments.
This makes for a high unmet patient need prior to joint replacement.
To top it off, osteoarthritis is the fastest increasing major health condition due to ageing of the population, increased obesity and lack of physical activity.
The global NSAIDs (Non-steroidal anti-inflammatory drugs used to manage pain) market is US$12 billion per annum.
Regeneus’ initial focus is on the Japanese market, which has a rapidly aging population with a preference for non-surgical treatment options.
It also provides the potential to fast track Progenza to market given that Japan’s Pharmaceuticals, Medical Devices and Other Therapeutic Products Act provides a framework for expedited approval for cell therapy products by allowing marketing following Phase 2 trials, on demonstration of safety and probable efficacy.
Regeneus had $3 million in cash as at 30th June 2015.
Regeneus are also expecting a $3 million R&D tax reb