SAN DIEGO, Feb. 15, 2018 (GLOBE NEWSWIRE) — Cytori Therapeutics, Inc. (Nasdaq:CYTX) (“Cytori” or “the Company”) today presented data from the U.S. 88 patient, 19 center, randomized, double blind, placebo-controlled STAR clinical trial of Cytori Cell Therapy™ (Habeo™) for the treatment of impaired hand function in patients with systemic sclerosis (SSc). These data were presented on behalf of the study investigators by Principal Investigator, Dr. Dinesh Khanna of the University of Michigan, at the 5th Systemic Sclerosis World Congress in Bordeaux, France.
This presentation included data not previously reported by the Company including responder analyses based on Minimal Clinical Important Differences (MCID) for the Cochin Hand Function Scale (CHFS) and for the Health Assessment Questionnaire-Disability Index (HAQ-DI) which are validated instruments assessing hand function and overall disability respectively in patients with SSc. Data from exploratory end points and details on the safety profile of Habeo were also presented.
The newly reported findings show consistency of efficacy across end points such that, 48 weeks after treatment, 52% of subjects with diffuse SSc treated with Habeo showed improvement that exceeded the MCID for both hand function (CHFS) and disability (HAQ-DI) compared with only 16% in the placebo arm. This difference in response rate was associated with a nominal p-value = 0.016.
Furthermore, the data showed that 47% of subjects with diffuse SSc who were treated with Habeo reported at least moderate improvement in disability (HAQ-DI) at 48 weeks compared with only 16% in the placebo arm (nominal p-value = 0.035). Consistent with the CHFS data, these differences were more evident in disability (HAQ-DI) domains related to hand function (e.g. eating and grip) than they were in domains less relevant to the hand (walking and arising).
“Impaired hand function is a serious problem for patients with scleroderma for which there is no currently-approved treatment,” said Dr. Dinesh Khanna, Principal Investigator of the U.S. STAR clinical trial. “The finding that patients already receiving current standard of care, showed further sustained meaningful improvement in hand function in response to Habeo represents an important new potential approach in our ability to help patients counter the debilitating effects of this disease.”
Other newly-reported data from the STAR trial include:
- Health-related Quality of Life assessed using the standard EQ-5D instrument in patients with diffuse SSc showed worsening in the placebo arm and improvement in the Habeo-treated arm (nominal p-value = 0.011).
- Patient assessment of SSc activity at 48 weeks in patients with diffuse SSc also improved in the Habeo-treated subjects and worsened in subjects in the placebo arm (nominal p-value = 0.015).
º Physician assessment of SSc activity showed a similar trend though with a p-value = 0.17.
- The procedure and treatment was well-tolerated and had a strong safety profile:
º 2.1% (1/48) of all subjects in the Habeo-treated arm and 12.5% (5/40) of those in the placebo arm reported serious adverse events (SAEs). No SAEs in the hand or deaths were reported.
º The overall rate of adverse events was very similar for both groups: 82.5% in the Habeo group and 81.3% in the placebo group.
º Peripheral vascular events (e.g., hypertension, hypotension, digital infarction, and worsening of Raynaud’s phenomenon) were observed in 15% (6/40) of placebo treated subjects compared with none (0/48) in the Habeo-treated group.
º Calcinosis was observed in 12.5% (5/40) of subjects in the placebo group compared with 2.1% (1/48) in the Habeo group.
“Our complete analysis of data from the STAR trial is very encouraging. The safety profile exhibited by Habeo is clear and the efficacy signals and trends are consistent,” said Dr. Marc H. Hedrick, President and Chief Executive Officer of Cytori Therapeutics. “Based on this data set, we have provided a detailed report to FDA in preparation for a pre-submission meeting scheduled for later this quarter with the goal of defining next clinical and regulatory steps in the U.S.”
STAR was a randomized, placebo-controlled, double-blind, parallel group, U.S. clinical trial intended to study the safety and efficacy of Habeo Cell Therapy in 88 subjects with hand dysfunction due to scleroderma at 19 U.S. centers. Cytori is currently working with statisticians and study investigators in preparation for publication of these results in a peer-reviewed journal.
Scleroderma is a rare and chronic connective tissue disease generally classified as an autoimmune rheumatic disorder. The word “scleroderma” is derived from two Greek words: “sclera,” which means hard, and “derma,” meaning skin, as hardening of the skin is one of the most visible manifestations of the disease. An estimated 300,000 Americans have scleroderma, about one-third of whom have the systemic form of the disease, known as systemic sclerosis (SSc). SSc is further sub-classified as diffuse cutaneous and limited cutaneous SSc. Patients with diffuse cutaneous SSc have more severe disease with significant hand dysfunction and internal organ involvement. Diffuse scleroderma accounts for between one-third and one-half of all cases of systemic sclerosis.1,2
SSc contributes to hand impairment through inflammatory arthritis or inflammation of the joints, joint contractures, Raynaud’s Phenomenon (RP, skin discoloration resulting from narrowing of the blood vessels in response to cold, emotional upset, or stress), digital ulcers, puffy hands and skin fibrosis over the fingers and hands, and calcinosis (calcium deposits in the soft tissues of the hand). These manifestations, which often coexist, can contribute to difficulty with occupational activities and activities of daily living, which can impair quality of life. Whereas current treatment recommendations focus on management of internal organ involvement, there is little treatment available for hand impairment.
About Habeo Cell Therapy
Cytori is developing cell therapies that harness the unique attributes of adipose-derived regenerative cells (ADRCs), which are living cells that are present in an adult human’s own adipose tissue.
Habeo Cell Therapy is a suspension of ADRCs that are manufactured from a single lipoaspirate (material removed via liposuction, a procedure in which fat is removed from under the skin by suction). The process concentrates ADRCs intended for autologous re-implantation subcutaneously into the digits. The resultant cell suspension contains critical cells naturally occurring in the patient’s own tissue. Preparation of autologous ADRCs for subcutaneous delivery involves no cell culture and can be prepared and re-implanted into the same patient within 4 hours.
Cytori is a therapeutics company developing regenerative and oncologic therapies from its proprietary cell therapy and nanoparticle platforms for 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. Cytori Nanomedicine™ is developing liposome encapsulated therapies for regenerative medicine and oncologic indications. For more information, visit www.cytori.com.
Cautionary Statement Regarding Forward-Looking Statements
This press release includes forward-looking statements regarding events, trends and business prospects, which may affect Cytori’s future operating results and financial position. These statements are subject to risks and uncertainties that could cause Cytori’s actual results and financial position to differ materially. Such statements, include, but are not limited to, statements regarding efficacy and safety trends of Habeo; Cytori’s anticipated feedback from our FDA pre-submission meeting, and possible future clinical trials of Habeo Cell Therapy. Some of these risks and uncertainties include: the risk that the cost and other negative effects related to the workforce reduction may be greater than anticipated; the risk that Cytori may not realize the benefits expected from the workforce reduction or other cost control measures; the risk that Cytori may need additional funds to conduct its operations, and that investors may not agree with the manner in which Cytori allocates its resources; unexpected or unfavorable clinical data, and the risk that regulatory agencies may interpret or weigh the importance of data differently and reach different conclusions than Cytori, request additional information, have additional recommendations or change their guidance or requirements; the timing, success and cost of Cytori’s research and development and related strategy and decisions; Cytori’s ability to comply with the terms of its credit agreement and the potential for acceleration of amounts owed to its secured lender; the potential for litigation or other disagreements with third parties; and other risks and uncertainties described under the “Risk Factors” section in Cytori’s Securities and Exchange Commission filings on Form 10-K and Form 10-Q. 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.
2 Arthritis Rheumatism Vol 48, (8), August 2003, pp 2246–2255 DOI 10.1002/art.11073
Cytori Therapeutics, Inc.