Regeneus (ASX:RGS) has successfully completed a pre-clinical study to support the first-in-human safety study for Progenza, its allogeneic, off-the-shelf stem cell therapy treatment for osteoarthritis (NYSE:OA).
This confirmed the product is safe and prevents disease progression in a model of OA.
The company will proceed with its ethics application for the commencement of a clinical trial to assess safety and preliminary efficacy of Progenza in patients with knee OA.
"This is an important milestone in taking Progenza through the regulatory approval process," clinical research director Janet Wilson said.
"As we have qualified the trial product in preclinical work, our next step is to seek ethics approval for the first-in-human trial.
"The planned trial is a single centre study and will include patients with symptomatic knee OA. Progenza will be injected once into the knee.
"Safety will be monitored closely and assessments for preliminary efficacy, such as MRIs pre and post treatment, are also included."
Regeneus previously noted that new regenerative medicine laws have come into effect in Japan that could provide an accelerated path for the approval of cell therapy products.
The new laws provide a unique opportunity to fast-track the clinical trial for Progenza.
The Good Laboratory Practice (NYSE:GLP)-compliant pre-clinical study was conducted 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 source material for Progenza is adipose tissue (fat) from a healthy donor.
The stem cells from the tissue are expanded through a proprietary manufacturing process that allows large numbers of therapeutic doses to be produced from a single donor.
The production of donor off-the-shelf stem cell product is to be contrasted with autologous stem cell therapy where a patient's own stem cells are used as the therapeutic product.
Stem cells, such as Progenza, work by homing to the site of injury and damage, exerting action through the production and release of various growth factors and anti-inflammatory proteins to stop cell death and promote healing and normal growth.
Japanese Regenerative Medicine Laws
Under the new laws, once a company has demonstrated safety and basic efficacy data in humans with a cell product manufactured to the Pharmaceuticals and Medical Devices Agency's (PMDA) standards, the cell therapy can be given conditional approval.
This approval can be for up to 7 years for commercial use with data reporting requirements and potential for national insurance coverage.
Safety data can be used from non-Japanese participants.
The successful pre-clinical study allows Regeneus to seek ethics approval for the first-in-human trial for Progenza.
This will be a single centre study and will include patients with symptomatic knee osteoarthritis.
Carrying out this study allows the company to monitor Progenza's safety and assess it for preliminary efficacy.
In addition, the company has taken key actions since the new Japanese regenerative medicine laws were passed through the Japanese Parliament in November 2013, providing it with the benefit of first mover status.
Japan offers a suitable market for an off-the-shelf cell therapy product for OA as it has a rapidly ageing population coupled with a recognised shortage of effective non-surgical treatment options.
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