Regenerative medicine company Regeneus (ASX: RGS) has received the key ethics approval to collect stem cells from human donors for the production of its new allogeneic off-the-shelf stem cell product for osteoarthritis.
It can now begin manufacturing the cells for its first-in-man clinical trial to assess initial safety and preliminary efficacy in human volunteers with knee OA.
The company has targeted the Japanese market for its allogeneic stem cell product to take advantage of new laws passed in November 2013 that provide an accelerated approval process for allogeneic human cell therapies without the need for expensive phase III clinical trials.
"This is an important first step in taking our human allogeneic product for osteoarthritis through the regulatory approval process," clinical development director Dr Richard Lillischkis said.
"We are collecting adipose tissue (fat) from human donors and are extracting and expanding the stem cells to produce large numbers of vials of frozen stem cells for use in clinical trials."
Access to an off-the-shelf product will mean stem cell therapy is less invasive for patients than autologous treatments and provide a convenient treatment option for specialists.
The treating doctor will simply remove a vial of cells from the freezer, thaw the cells and inject them in to the patient's joints.
The new product will need to pass through the usual safety and efficacy trials to achieve market access.
Regeneus is a pioneer in the development and use of adipose-derived stem cell products.
It markets an autologous product, HiQCell, that has been used to treat over 1,000 human arthritic joints in clinical trials and commercial settings.
The company has also developed and manufactures a clinical stage allogeneic off-the-shelf product, CryoShot, which has been used in trials to treat hundreds of cases of canine and equine OA.
Through the development and testing of CryoShot, the company has developed considerable expertise in product design, tissue sourcing, manufacture, handling and delivery in-clinic of an off-the-shelf stem cell product.
Securing the ethics approval to collect stem cells from human donors is a key step towards starting a clinical trial for Regeneus' new allogeneic off-the-shelf stem cell product for treating osteoarthritis.
That the company has chosen to focus on the Japanese market, which provides an accelerated approval process for allogeneic human cell therapies, means it can potentially complete the trials more rapidly and at a lower cost and generate revenues more quickly.
Regeneus is already a pioneer in the development and use of adipose-derived stem cell products with its HiQCell autologous product returning strong results from the joint registry used to track its safety and effectiveness.
The interim report of 305 patients as at 23 January 2014 had found that patients reported continued improvements - including significant improvements in pain, function, sleep quality and reduced usage of pain medications - at 12 months post-treatment, and the small number of patients that had reached 24 months post-treatment also show continued improvements.
Proactive Investors believes that Regeneus' allogeneic off-the-shelf stem cell product keeps the company at the forefront of a massive growth in the regenerative medicine and cell technology industry, currently in its infancy.
Its success would add to the revenue - $1.77 million in 2013 - that it already draws from its existing stem cell OA treatments.
Regeneus has enjoyed rapid growth since its $10 million IPO raising closing oversubscribed in September 2013 with its valuation rising to circa $80 million.
On our initial estimates, the company is valued at between $0.76 and $0.93 per share within 6-9 months.
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