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Phase 3 Trial of Prochymal in Crohn's Disease Halted

COLUMBIA, Md.--(BUSINESS WIRE)--Osiris Therapeutics, Inc. (NASDAQ: OSIR - News) announced today that it has elected to end enrollment at 210 patients in its Phase III trial evaluating Prochymal for Crohn’s disease. The Company believes there is a design flaw in the trial resulting in significantly higher than expected placebo response rates. The decision was made after the trial’s final scheduled interim analysis showed that one of the two Prochymal dose arms had crossed a futility boundary. The dose arm was unlikely to achieve the primary endpoint of remission because of the high placebo response rate. This latest analysis continued to show no serious safety concerns with the therapy and safety was not a factor in the decision to stop enrollment.

C. Randal Mills, Ph.D. President and CEO of Osiris Therapeutics said, “We had a situation where we were experiencing much higher than expected placebo response rates. As we looked into possible causes, we discovered what we believe to be a systemic design flaw in the trial that would likely affect the utility of the data for purposes of registration. After careful discussion with the FDA, we elected to discontinue enrollment rather than attempt to re-power the trial. We will keep the trial blinded and expect a solid data package for use in designing future trials in Crohn’s disease and to bolster Prochymal’s safety database.”

Mills continued, “I would like to personally thank the physicians, study professionals, and of course the patients who have dedicated their time to this trial, and appreciate their ongoing participation.”

The Prochymal Crohn’s program consists of two separate but related double blinded trials. The first trial evaluates patients’ initial response to two dose levels of Prochymal as compared to placebo. The trial was originally designed to enroll 270 subjects. The potential trial design flaw may be related to the fact that patients responding to the initial therapy were eligible to participate in a second, longer-term trial evaluating Prochymal as a maintenance therapy. Because the current standard for determining response of Crohn’s patients to therapy is largely subjective, there may have been response bias to meet the eligibility requirements for continuation of therapy in the longer-term maintenance trial.

David Meeker, M.D., Executive Vice President at Genzyme Corporation said, “We fully agree with the decision by Osiris to end enrollment in this trial due to what appears to be a problem with the trial design. We think this is prudent and will enable a more efficient path forward. The data from this trial will be extremely valuable in designing the subsequent trial and enabling the continued development of this first-in-class therapy.”

Source: Osiris Therapeutics

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Decision supported by data from more than 4,000 patients