Cytori Granted Orphan Drug Status for Cellular Therapeutic in European Union

Cytori Therapeutics, Inc. (NASDAQ: CYTX) announced today that the European Commission, acting on the positive recommendation from the European Medicines Agency Committee for Orphan Medicinal Products, has designated Cytori’s ECCS-50 cellular therapeutic as an orphan medicinal product for the treatment of scleroderma. This designation marks the first autologous adipose derived cell therapy for scleroderma granted orphan status in the European Union (EU).

Cytori is currently working with leading scleroderma centers in France led by the Assistance Publique des Hôpitaux de Marseille to begin a 40 patient EU pivotal trial called SCLERADEC-II. The trial is a follow-up controlled trial to the first SCLERADEC trial published last August (Granel et al. Annals of the Rheumatic Diseases, 2014). If successful, this trial would potentially lead to EMA approval of Cytori’s lead therapeutic for this indication in the EU, as an ATMP or Advanced Therapy Medicinal Product.

“Obtaining orphan drug designation in the EU, like FDA Phase III pivotal trial approval for the same indication in the US, is an important milestone and part of our corporate strategy of targeting rare or niche indications with clear unmet medical needs that can be brought to market rapidly,” said Dr. Marc Hedrick, President and CEO of Cytori Therapeutics. “Orphan approval gives Cytori a number of advantages that greatly facilitates us getting a novel drug into a market with favorable reimbursement dynamics.”

Cytori recently received FDA approval for a phase 3 clinical trial with our ECCS-50 therapeutic in 80 patients with scleroderma associated hand dysfunction. This US clinical trial is currently planned to begin enrolling this year and has recently been granted FDA approval to expand the number of centers from 12 to 20 sites. This US clinical data could also be useful to support regulatory approvals and reimbursement in the EU as well as other global markets.

The European Commission grants orphan designations for medicines that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the EU and where no satisfactory treatment is available. In Europe, the scleroderma affected population appears to be between 82/million^1,2 and 280/million⁴, which would account for a market size of 61,000 to 208,000 of scleroderma patients. Scleroderma is estimated to have a prevalence of 240/million in the adult population which equates to approximately 50,000 scleroderma people in the United States³.

“Scleroderma involves the hands in nearly 90% of patients and nearly always is accompanied by pain and impairment of simple daily activities that we all take for granted,” said Dr. Steven Kesten, Chief Medical Officer of Cytori Therapeutics. “Hand symptoms are the leading cause of disability in this patient population, more disabling than rheumatoid arthritis, and severely negatively impact quality of life. In addition to general measures to protect the hand, patients require medications, which often are inadequate to control symptoms or prevent complications.  As our fingers and hand are so critical in everyday activities, people with scleroderma are constantly aware of their disease.  We are encouraged by the data generated to date and believe that the prospective trials in the US and France will provide the level of evidence needed to definitely demonstrate the benefit of Cytori Cell Therapy."

“A significant portion of the overall European cell therapy approval process has now been completed given our existing CE Mark on the Celution System”, stated Kenneth Kleinhenz, Vice President of Regulatory Affairs and Quality Assurance at Cytori Therapeutics. “All cell therapy products used in combination with a medical device require external review by a European notified body. The CE Mark certificate previously issued by our notified body, achieved this requirement and includes much of the required manufacturing requirements since the ECCS-50 drug product is manufactured exclusively with the Celution System at the point of care. This modular approach alleviates many of the regulatory uncertainties with the manufacturing aspects of the EMA approval process, leaving the clinical data as the remaining milestone required for market authorization.”

In addition, a range of development, regulatory, and financial incentives are offered in the EU for medicines that have been granted an orphan designation by the European Commission⁵:

• A form of scientific advice called protocol assistance
• Access to the centralized authorization procedure using a single application
• Additional incentives for micro, small, and medium-sized enterprises including administrative and procedural assistance and fee reductions
• 10 years of market exclusivity once approved
• Reduced fees for regulatory activities
• Incentives in EU member states

About Cytori Therapeutics, Inc.

Cytori Therapeutics is a late stage cell therapy company developing autologous cell therapies from adipose tissue to treat a variety of medical conditions. Data from preclinical studies and clinical trials suggest that Cytori Cell Therapy™ acts principally by improving blood flow, modulating the immune system, and facilitating wound repair. As a result, Cytori Cell Therapy™ may provide benefits across multiple disease states and can be made available to the physician and patient at the point-of-care through Cytori’s proprietary technologies and products. For more information: visit www.cytori.com.

Cautionary Statement Regarding Forward-Looking Statements

This communication includes forward-looking statements regarding events, trends and business prospects, which may affect our future operating results and financial position. Such statements, including, but not limited to, those regarding our ability to achieve a marketing authorization for Cytori Cell Therapy™, and the impact of a CE Mark on the Celution System in accelerating the European marketing approval, are forward looking statements. Such statements are subject to risks and uncertainties that could cause our actual results and financial position to differ materially. Some of these risks include clinical, regulatory and reimbursement uncertainties, the challenges inherent in convincing physicians and patients to adopt the new technology, dependence on third party performance, performance and acceptance of our products, and other risks and uncertainties described under the “Risk Factors” in Cytori's Securities and Exchange Commission Filings, including in its most recent annual and quarterly reports. Cytori assumes no responsibility to update or revise any forward-looking statements contained in this press release to reflect events, trends or circumstances after the date of this communication.

1. Rheumatology (Oxford). 2004 May;43(5):596-602. Epub 2004 Feb 10.

2. Medicine (Baltimore). 2008 Sep;87(5):272-80. doi: 10.1097/MD.0b013e318189372f.

3. Rheum Dis Clin North Am. 2003 May;29(2):239-5

4. Rheumatology (Oxford). 2004 Sep;43(9):1129-37. Epub 2004 Jun 22.

5. Regulation (EC) No 141/2000 on orphan medicinal products (http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2000:018:0001:0005:en:PDF).