Osiris Therapeutics Reports Interim Data For COPD Stem Cell Study

Main Category: COPD
Also Included In: Stem Cell Research;  Clinical Trials / Drug Trials;  Respiratory / Asthma
Article Date: 25 Jun 2009 - 3:00 PDT

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Therapeutics, Inc. (NASDAQ:OSIR) announced six-month interim data from a Phase II clinical trial evaluating Prochymal, the Company's proprietary formulation of adult mesenchymal stem cells, for the treatment of chronic obstructive pulmonary disease (COPD). Sixty-two patients were enrolled and are being followed for two years in the placebo-controlled study. At the six-month time-point, the data revealed several important findings.

Important Findings:

- The trial met its primary goal of demonstrating the safety of Prochymal in patients with compromised pulmonary function at the six-month evaluation point.

- Prochymal significantly decreased systemic inflammation in patients when compared to those receiving placebo, as determined by C-reactive protein (CRP).

- Despite the reduction in inflammation, pulmonary function in patients receiving Prochymal was not significantly improved compared to those receiving placebo.

"We are very pleased with the interim outcome of this study and that the data continues to support the strong safety profile of this therapy, particularly given the severity of these patients' pulmonary disease," said C. Randal Mills, Ph.D., President and CEO of Osiris Therapeutics. "Importantly, we are gratified to obtain clear, objective data that helps bolster our understanding of the anti-inflammatory effects of these remarkable cells. Short-term, these anti-inflammatory effects did not appear to improve pulmonary function in patients with advanced destructive changes of the lung. Collectively however, these findings add to our confidence about the safety and effectiveness of the drug."

Six-Month Interim Data

Prochymal was evaluated in a total of 62 patients, 58% of them male. The patients ranged in age from 47 to 80 years and suffered from moderate (n=23) to severe (n=39) COPD. Patients had been suffering with COPD for an average of 7.8 years. Patients with asthma were excluded from the trial.

All patients in the trial completed the planned course of four infusions without any evidence of infusional toxicity. Oxygen saturation levels were measured throughout each infusion and showed no adverse effects of the infusion. Adverse event rates were comparable for patients receiving Prochymal and placebo. There were no signs of adverse immune reaction after any of the four infusions and no differences in the reported incidence of infection of any kind.

Prochymal significantly decreased the levels of CRP compared to placebo in those patients with elevated CRP (>4 mg/L) at the time of study entry (p<0.05). The difference from placebo was evident at ten days post initial infusion, and was maintained throughout the treatment and follow-up period. CRP is a protein found in the blood in response to inflammation and is often elevated in inflammatory diseases such as GvHD and Crohn's disease, and has been found to correlate with clinical parameters of disease activity. Although there is substantial preclinical evidence, this study provides the first well-controlled objective data confirming the systemic anti-inflammatory effects of Prochymal in humans.

Pulmonary function tests such as FEV1 and DLCO or carbon monoxide diffusing capacity of the lung were not improved over placebo following treatment with Prochymal at six months. FEV1 or forced expiratory volume is the amount of air that can forcibly be blown out of the lungs in the first second of exhalation. Although not reaching statistical significance, treatment with Prochymal did produce positive trends in exploratory functional endpoints such as the six-minute walk test and certain cardiac related parameters, particularly in patients with less established COPD.

The trial will continue as planned and patients will be followed for the balance of the two-year evaluation period.

About the Phase II Chronic Obstructive Pulmonary Disease Trial

The Phase II trial is evaluating the safety of Prochymal in conjunction with standard of care in patients with moderate to severe COPD. The clinical trial is a double-blind, placebo-controlled study. A total of 62 patients were randomized to either Prochymal or placebo at a 1:1 ratio. In order to explore potential improvements in pulmonary function, several evaluations including pulmonary function tests, exercise capability, and quality of life assessments are being conducted. In addition, exacerbations and hospitalizations due to COPD will be monitored for both safety and efficacy. Patients will be evaluated over the course of two years following initial Prochymal or placebo infusion.

About Prochymal

Prochymal is a preparation of mesenchymal stem cells specially formulated for intravenous infusion. The stem cells are obtained from the bone marrow of healthy adult donors, avoiding the controversy surrounding embryonic and fetal cell sources. Prochymal is currently being evaluated in three, double-blind, placebo-controlled Phase III studies, including steroid refractory GvHD, acute GvHD, and Crohn's disease. Prochymal has been granted Fast Track status by FDA for all three of these indications. Prochymal also obtained Orphan Drug status by FDA and the European Medicines Agency for GvHD. Prochymal is also being studied in Phase II trials for the treatment of acute myocardial infarction and type 1 diabetes. Additionally, the Department of Defense awarded Osiris a contract to develop Prochymal as a treatment for acute radiation syndrome.

Source
Osiris Therapeutics