Cytori Therapeutics announced that they have enrolled their first two patients in an acute myocardial infarction clinical trial using adipose-derived cells prepared using their Celution™ device. The Celution™ device is an automated, tissue processing unit that isolates cells from adipose tissue derived via liposuction. The cells are then available for transplantation back into the patient within hours of fat tissue isolation. Cytori also mentioned in the press release the “number of patients” that have already been treated in their PRECISE chronic ischemia clinical trial, demonstrating that they are successfully recruiting patients in their initial trial. Both trials are being run in Europe between Spain and the Netherlands where autologous, patient-specific therapies have lower regulatory hurdles (and are thus quicker to initiate) than in the US, so it is a good proving grounds for this type of technology.
As you have heard here before, cardiac regeneration is one of the most important clinical indications for cell therapy companies today. The market is huge, the unmet need is tremendous (read that meaning: reimbursable therapy), and locally transplanted cells are definitely doing something – although we don’t know exactly what or to what extent. The more rapidly a company understands if their cells are effective in cardiac regeneration, the more clear their future business strategy will be. Cytori is addressing this issue head on with these European trials, and if they start seeing efficacy they will likely ramp up US clinical trial activity. Cytori’s goal will be to get this information in the 2008 calendar year.
Cytori has a very different business model from other cell therapy companies. They are not selling cells as a therapeutic like Osiris, Athersys, or Aastrom. Cytori’s plan is more of the razor/razor blade approach, where they would install the Celution system (the razor) into hospitals and clinics, and then sell the per-patient disposables (the razor blades) for a continuing revenue stream. This approach is getting applause from others in the investment blogosphere. The Celution™ system takes fat (aka adipose) tissue, digests it, isolates the cells from the tissue and then washes and concentrates the cells to create an implantable formulation. This approach definitely has time and cost advantages over culture-derived cell therapies, but there are many questions around how the implantable product’s consistency and quality is controlled. Will they include in-line cell counts and viability, or will this be up to the end-user? How do they handle patient-to-patient variability? How will sterility, mycoplasma, and endotoxin levels of the final product be evaluated? How is dose determined? Will the end-user want to be responsible for cell quality, and what will the GMPs look like around this process? All of these questions are addressable, and are likely already being tackled by Cytori. How they address these challenges will dictate how large of a market they will be able to capture.
Cytori’s technology, including the cells, the device, and their therapeutic targets are well described in the patent literature (do your search under Marc Hedrick as inventor, not Cytori). An analysis of their patent estate (saved for a later post) shows that they don’t have many issued patents yet as it pertains to their cellular compositions. However, they do have several pending applications that, if issued, will give them a strong position to exclude other adipose-cell isolator knock-off products. Being first to market would also give them a competitive advantage over other cell-isolation devices, and it will be interesting to see how Cytori will compete with other device-based companies such as Harvest Technology, Miltenyi, Baxter, and Aldagen. How Cytori and the other device-centric companies compete against their cellular products brethren will be heavily dependent on, of course, good clinical data. Also important will be the evolution of the regulatory landscape and how comfortable end-users will be with point-of-care cell preparation devices and creating their own cell preparations. This is exciting technology, and a very important component of the overall cell therapy landscape.