Osiris Therapeutics, Inc. (OSIR)
Q1 2010 Earnings Call Transcript
May 7, 2010 9:00 am ET
C. Randal Mills – President and CEO
Phil Jacoby – CFO and Corporate Secretary
Duane Nash – Wedbush Securities
Edward Tenthoff – Piper Jaffray
Charles Duncan – JMP Securities
Lunaan Zee [ph] – Jefferies
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I would now like to introduce – turn the call over to Dr. C. Randal Mills, President and CEO of Osiris Therapeutics. Please go ahead, sir.
C. Randal Mills
Thank you. Good morning and thank you for joining us on our first quarter 2010 conference call. I will start with some focused comments around the progress we are making advancing our lead stem cell therapy Prochymal in the United States and around the world for the treatment of graft versus host disease. I will also be providing an update of our Crohn’s, Type 1 diabetes, and acute MI programs as well. Our CFO, Phil Jacoby, will provide a summary of our financial performance for the first quarter. And finally, I will have a few closing comments regarding our priorities for the coming months and we will then take questions.
Let me start with our GVHD program. As I mentioned on our last call, our priority has been to work with the FDA to come to agreement on the outstanding elements required for a successful submission. Central to these efforts was to gain consensus around the statistical analysis plan or SAP that has been used to evaluate the data from Protocol 275, our program for pediatric patients with treatment resistant graft versus host disease.
We have now been given the go-ahead from FDA to execute the SAP. The SAP will be performed by our contract research organization and the results will form the basis on a pivotal clinical study report. To help people understand where this piece fits into the overall process, executing the SAP provides us with the final major data set that will be used in support of the clinical section of the Common Technical Document and in the case of the United States Food and Drug Administration, the Biological License Application, or BLA.
Since the execution of this SAP, Protocol 275 adds new content to the overall scope of our BLA. We will be holding another pre-BLA meeting with the FDA consistent with their internal procedures. It is important to understand that we will not complete the BLA submission until after this meeting. Our aim is to build consensus for the specific indication we are requesting approval for, by providing FDA with compelling data in a manner they request. We do not intend to submit a BLA to the agency before we have reached this consensus. There are no shortcuts just because Prochymal is a stem cell product.
FDA is holding us to the standard of proving Prochymal as safe and effective. So that is where our focus remains. We are going to continue to be very conservative and limited our comments about the ongoing submission process. Please note that during this time, we will continue to make Prochymal available to children and adults with refractory GVHD who are in need of our help. We appreciate your understanding and we will provide update on our progress as circumstances warrant.
We continue to make progress in territories outside the United States with our partners, Genzyme and JCR Pharmaceuticals. A clear indication of this progress is the $1 million milestone we received from our Japanese partner JCR for regulatory progress in Japan. This milestone demonstrates JCR’s commitment to the development of Prochymal for GVHD, and we are proud to stand with them.
Now let me turn to our other programs. We recently provided an update on our Crohn’s program. Recall that the program originally consisted of two linked trials; one, which evaluated Prochymal’s ability to induce remission in Crohn’s patients who had failed other available treatments for the disease. This was Protocol 603. The second trial, Protocol 610, was a maintenance trial that re-randomized only those patients responding to the induction therapy.
We suspended enrollment in 2009 due to concerns that the entry criteria for the maintenance trial influenced patients to over-report response to therapy in the induction trial, thereby making it difficult to detect a true treatment effect. Essentially, the trial design was bleeding off power. In fact, one dose arm had crossed a predetermined futility boundary. Meaning, it was unlikely to show a significant benefit at the end of the trial.
At that time, we made the decision to keep the trial blinded and to keep the clinical sites active so that patients can continue to be followed. When all 207 patients who were initially enrolled in the trial completed the study, our contract research organization began their procedures to collect and analyze the full dataset, which ultimately led to the announcement we made a few days ago.
Regarding the trial and its outcome, the first thing that is important to appreciate is just how sick the patients are at the time of entry into the study. Most of these patients have been suffering from their disease for more than 13 years. The majority of patients have failed at least two immunomodulators and two biologic agents and had an average Crohn’s Disease Activity Index or CDAI of greater than 350 in all three arms prior to treatment.