Cytori Reports Interim Data on U.S. Phase II Osteoarthritis Trial
Cytori Therapeutics, Inc. (NASDAQ: CYTX) announced today the top-line data as part of the pre-specified partial unblinding of 24 week follow up data from the company's ACT-OA trial. The trial is a U.S. phase II randomized, double-blind, placebo controlled trial designed to evaluate the safety and feasibility at 48 weeks of a single intra-articular knee injection of the ECCO-50 cellular therapeutic in patients with chronic knee pain due to osteoarthritis.
"Key endpoints and trends observed thus far suggest that a beneficial effect may be attributable to a single intra-articular administration of ECCO-50 in patients with osteoarthritis of the knee," said Dr. Marc H. Hedrick, President and CEO of Cytori Therapeutics. "The full 48 week data set is the next important milestone in this program. While we continue to move our clinical pipeline along, the company will remain intensely focused on bringing to market our lead therapeutic for scleroderma which is currently in phase III."
The primary goal of the ACT-OA trial is to help determine: (1) safety and feasibility of ECCO-50 for osteoarthritis, (2) provide dosing guidance and (3) explore key trial endpoints useful for a phase III trial. As a proof of concept trial, ACT-OA was not sized or powered for statistical significance in any of the endpoints.
The purpose of the interim 24 week partial unblinding in this trial is to provide early data that will facilitate key regulatory and business development discussions and provide better understanding of the therapeutic mechanism of action that may impact other clinical programs.
The interim analysis pre-specifies the evaluation of a number of patient reported outcomes important in patients with osteoarthritis of the knee. A total of 94 patients were treated in the trial (30 received a low dose of ECCO-50 [20 million cells], 31 received a high dose of ECCO-50 [40 million cells] and 33 received placebo). Patients, providers and Cytori personnel remain blinded to individual patient treatment allocation and patient subgroup outcomes.
The interim top-line data show the following:
- The randomization is relatively balanced among the three treatment groups: low dose, high dose and placebo.
- Intra-articular application of a single dose of ECCO-50 appears to be safe and feasible in an outpatient day-surgery setting. No complications occurred related to the fat harvest, cell processing or cell delivery.
- A significant placebo response was observed, similar to that demonstrated in other OA trials.
- The pre-specified primary endpoint, pain on walking at 12 weeks, as measured by a single question from the Knee Injury and Osteoarthritis Outcome Score (KOOS) did not obtain statistical significance.
- Key secondary endpoints include the total and sub-scores of the KOOS, patient assessment of knee pain, knee stability, osteoarthritis activity and osteoarthritis damage, use of as-needed pain medication, pain while walking 50 feet and health status as measured by the SF-36. Consistent trends were observed suggesting improvement in the cell treated group relative to the placebo group at the 12 and 24 week time periods for patient reported outcomes.
- Both high dose and low dose of ECCO-50 performed similarly.
In the 3rd quarter, following full unblinding of the data, the Company will be able to more fully evaluate the data, including 48 week follow up, patient subset analyses, and the effect on knee cartilage as measured by magnetic resonance imaging results changes between baseline and 48 weeks.
In summary, patterns of response are suggestive of a beneficial effect attributable to a single intra-articular administration of ECCO-50 in patients with osteoarthritis of the knee. The full 48 week data are expected to be available for review during the third quarter of this year, which will more fully inform further development decisions, including phase III trial design, cell dose, administration protocol, inclusion/exclusion criteria, selection of primary and secondary endpoints and sample size. Further details of the trial can be found on clinicaltrials.gov (NCT02326961).
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 press release includes forward-looking statements regarding events, trends and business prospects, which may affect our future operating results and financial position. Such statements, including, without limitation, statements regarding (i) clinical performance of our technology, including its safety, efficacy and feasibility, (ii) our clinical pipeline, including our focus on bringing our scleroderma therapeutic to market, and (iii) review and publication of our study data (including anticipated 48 week data), are all subject to risks and uncertainties that could cause our actual results and financial position to differ materially. Some of these risks and uncertainties include, but are not limited to, inherent risk and uncertainty in the protection of intellectual property rights, regulatory uncertainties, risks in the conduct of clinical trials, risks in the collection and results of clinical data, final clinical outcomes, dependence on third-party performance, performance and acceptance of our products in the marketplace, as well as other risks and uncertainties described under the heading "Risk Factors" in Cytori's Securities and Exchange Commission Filings on Form 10-K and Form 10-Q. We assume no responsibility to update or revise any forward-looking statements to reflect events, trends or circumstances after the date they are made.
Cytori Therapeutics, Inc.
Tiago Girao, +1 (858) 458-0900