Check out the new pipeline "COPD"
http://www.osiristx.com/images/OsirisPipeline.jpg
http://www.osiristx.com/images/OsirisPipeline.jpg
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Osiris
- Thread starter danny
- Start date
J
JC the Fox
Guest
Osiris Trial
Osiris is, in fact, conducting Phase II Trials for COPD for the efficacy of Prochymal. I visited the test clinic today, and I will go back for the final lung function tests tomorrow. If I pass, I will be entered into the study, and will receive the first injection ten days later. It is a double blind randomized test, with 50% of the subjects receiving Prochymal and 50% receiving a placebo.
I was told that there still aren't enough volunteers, and this facility is looking to get 7 more qualified volunteers. Please do a search on MedResearch in Spartanburg, SC, to see about the facility. Nicole is my contact.
There are a number of exclusionary factors. Age is 40-80, a 6-minute walking test must be passed, FEV1 is to be greater than 30, and no past stem cell treatment is allowed. There are others too numerous for me to remember.
I shall let everyone know of my fortune, good or bad, as soon as I return home.
Osiris is, in fact, conducting Phase II Trials for COPD for the efficacy of Prochymal. I visited the test clinic today, and I will go back for the final lung function tests tomorrow. If I pass, I will be entered into the study, and will receive the first injection ten days later. It is a double blind randomized test, with 50% of the subjects receiving Prochymal and 50% receiving a placebo.
I was told that there still aren't enough volunteers, and this facility is looking to get 7 more qualified volunteers. Please do a search on MedResearch in Spartanburg, SC, to see about the facility. Nicole is my contact.
There are a number of exclusionary factors. Age is 40-80, a 6-minute walking test must be passed, FEV1 is to be greater than 30, and no past stem cell treatment is allowed. There are others too numerous for me to remember.
I shall let everyone know of my fortune, good or bad, as soon as I return home.
Great news
Great news JC. I spoke with Danny earlier and he wondered and now he has me wondering, what happened to Phase I? Can you tell us when that started? I do wish you well and look forward to hearing more about it. How on earth would they ever know if someone had stem cell therapy previously? Please give us updates if you will. You would know immediately in my opinion if you get the real deal.
Great news JC. I spoke with Danny earlier and he wondered and now he has me wondering, what happened to Phase I? Can you tell us when that started? I do wish you well and look forward to hearing more about it. How on earth would they ever know if someone had stem cell therapy previously? Please give us updates if you will. You would know immediately in my opinion if you get the real deal.
J
JC the Fox
Guest
I don't know when the Phase I trials were done, but I'll ask about them tomorrow. My finding out about this trial was a series of events that began with no knowledge at all about this clinic having any relationship with Osiris. As for their knowing about previous stem cell therapy, I suppose it would just depend on the honesty of the patient answering all the questions asked. I'll post again upon my return home (which is 450 miles from here).
JC
JC
Where is everyone?
JC and Danny - I find it incredible that people aren't lined up at the door waiting to apply. This is the best news to come down the pike in a long time for people who suffer from COPD. I truly wish that Danny qualified and see no reason for the 40+ criteria, but at least these trials are going to open the doors for the possibility of hope for a treatment that promises to give COPD sufferers some of their life back. Now, if we can get the ball rolling on many other diseases, maybe we will someday have treatment available wherever we live. I am very pleased that JC is a Pioneer member and will be able to let us know how things go. Thank you JC and thank you Danny. You are both great Pioneers.
JC and Danny - I find it incredible that people aren't lined up at the door waiting to apply. This is the best news to come down the pike in a long time for people who suffer from COPD. I truly wish that Danny qualified and see no reason for the 40+ criteria, but at least these trials are going to open the doors for the possibility of hope for a treatment that promises to give COPD sufferers some of their life back. Now, if we can get the ball rolling on many other diseases, maybe we will someday have treatment available wherever we live. I am very pleased that JC is a Pioneer member and will be able to let us know how things go. Thank you JC and thank you Danny. You are both great Pioneers.
I just love this article----
C. Randal Mills - Osiris
We just did report some additional cardiac data. That cardiac data was on an interim one-year endpoint, so the two primary reporting periods for that trial were actually six months and two years at the completion of the trial. So the interim one-year time point didn?t give us a full return of data and lung function was one of the things that was not scheduled to be reported there, so that?s why we didn?t have that.
But, as you stated at the sixth month time point we had this rather dramatic statistically significant over placebo improvement in pulmonary function. That correlates very well, by the way, with pre-clinical work, which demonstrates the therapeutic utility of mesenchymal stem cells in reversing obstructive pulmonary diseases, COPD obviously being the largest in that group with the fourth leading cause of death.
What?s going on there we believe that?s actually a direct effect that?s taking place in the lungs and we think, based on pre-clinical work, there are a number of different mechanisms at factor or at play here.
The first is the fact that when you infuse the mesenchymal stem cell into a patient intravenously, for a variety of reasons, mesenchymal stem cells initially target pulmonary tissue preferentially over all other tissue in the body and that?s because of the size of the capillary beds and the over-expression of STF-1 in the lungs naturally draws these cells to the lungs.
When they?re there, we know that in disease states, particularly in disease states that have an inflammatory profile as do the obstructive diseases, they reflexively release a cascade of beneficial cytokines and they also block TNF production and other inflammatory agents in the lungs.
And so, we think it?s the overproduction of the anti-inflammatory interleukins, as well as blocking inflammatory cytokine release that has a fast acting effect. Long-term though, we think there?s an opportunity for some actual disease modification taking place in the lungs and that we think has not only been supported by some histological pre-clinical work but by the fact that in the patients with COPD, we actually saw a widening of effect at six months over the three month or one month time-period.
So, with all that said, we are very excited about COPD. We?re actively working to get a COPD trial up and running. Once we have more details on that trial ready, we?ll be making an announcement about it. But, the COPD space is one we feel very, very good about.
C. Randal Mills - Osiris
We just did report some additional cardiac data. That cardiac data was on an interim one-year endpoint, so the two primary reporting periods for that trial were actually six months and two years at the completion of the trial. So the interim one-year time point didn?t give us a full return of data and lung function was one of the things that was not scheduled to be reported there, so that?s why we didn?t have that.
But, as you stated at the sixth month time point we had this rather dramatic statistically significant over placebo improvement in pulmonary function. That correlates very well, by the way, with pre-clinical work, which demonstrates the therapeutic utility of mesenchymal stem cells in reversing obstructive pulmonary diseases, COPD obviously being the largest in that group with the fourth leading cause of death.
What?s going on there we believe that?s actually a direct effect that?s taking place in the lungs and we think, based on pre-clinical work, there are a number of different mechanisms at factor or at play here.
The first is the fact that when you infuse the mesenchymal stem cell into a patient intravenously, for a variety of reasons, mesenchymal stem cells initially target pulmonary tissue preferentially over all other tissue in the body and that?s because of the size of the capillary beds and the over-expression of STF-1 in the lungs naturally draws these cells to the lungs.
When they?re there, we know that in disease states, particularly in disease states that have an inflammatory profile as do the obstructive diseases, they reflexively release a cascade of beneficial cytokines and they also block TNF production and other inflammatory agents in the lungs.
And so, we think it?s the overproduction of the anti-inflammatory interleukins, as well as blocking inflammatory cytokine release that has a fast acting effect. Long-term though, we think there?s an opportunity for some actual disease modification taking place in the lungs and that we think has not only been supported by some histological pre-clinical work but by the fact that in the patients with COPD, we actually saw a widening of effect at six months over the three month or one month time-period.
So, with all that said, we are very excited about COPD. We?re actively working to get a COPD trial up and running. Once we have more details on that trial ready, we?ll be making an announcement about it. But, the COPD space is one we feel very, very good about.
J
JC the Fox
Guest
Failure
I am afraid that I won't be a pioneer after all - I failed the FEV1 tests by a couple of points.
While I was there, though, I met a couple of other people trying to qualify; one did, the other did not. Both of them were referred to the clinic by a private practitioner; neither had any knowledge of Osiris prior to that, from what I could gather.
Which leads me to wonder why Osiris isn't showing anything about this trial on their website. By the way, it is Osiris Protocol #801 COPD. The drug is Prochymal, the same as for the heart patients, but definitely to find out its efficacy for COPD.
I am afraid that I won't be a pioneer after all - I failed the FEV1 tests by a couple of points.
While I was there, though, I met a couple of other people trying to qualify; one did, the other did not. Both of them were referred to the clinic by a private practitioner; neither had any knowledge of Osiris prior to that, from what I could gather.
Which leads me to wonder why Osiris isn't showing anything about this trial on their website. By the way, it is Osiris Protocol #801 COPD. The drug is Prochymal, the same as for the heart patients, but definitely to find out its efficacy for COPD.
Bummer
J C - How disappointing for you. I am very sorry to hear this. I sincerely hope that the trials will not drag on either, just as Jeannine says and that we will see treatment available for us all no matter what our FEV1 is or our age! The same goes for other applications besides lungs. It is time for the stem cell revolution. It is great to see that this trial has become a reality, however. You gave it your best shot. I think in the upcoming year there are going to be more and more improvements and choices for treatment and maybe even some more trials. Thanks for all your information.
I do need to add that J.C. you are a Pioneer. You went for it and just because you got rejected, you were certainly willing and undoubtedly able no matter what their criteria is.
J C - How disappointing for you. I am very sorry to hear this. I sincerely hope that the trials will not drag on either, just as Jeannine says and that we will see treatment available for us all no matter what our FEV1 is or our age! The same goes for other applications besides lungs. It is time for the stem cell revolution. It is great to see that this trial has become a reality, however. You gave it your best shot. I think in the upcoming year there are going to be more and more improvements and choices for treatment and maybe even some more trials. Thanks for all your information.
I do need to add that J.C. you are a Pioneer. You went for it and just because you got rejected, you were certainly willing and undoubtedly able no matter what their criteria is.
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Synthetic lung could further asthma research
Read the entire story - especially those Pioneers in the UK where this doctor has some clinical trials in place for COPD.
She is a also a founder of UK biotechnology company Synairgen, which operates a biobank of blood, sputum, biopsies, fibroblast and epithelial cells from asthmatic and COPD patients, and is also developing an inhaled formulation of interferon beta for asthma and COPD which has reached clinical trials.
Synthetic lung could further asthma research
http://www.labtechnologist.com/news/ng.asp?n=84971-university-of-southampton-microfluidics-asthma-copd
Read the entire story - especially those Pioneers in the UK where this doctor has some clinical trials in place for COPD.
She is a also a founder of UK biotechnology company Synairgen, which operates a biobank of blood, sputum, biopsies, fibroblast and epithelial cells from asthmatic and COPD patients, and is also developing an inhaled formulation of interferon beta for asthma and COPD which has reached clinical trials.
Synthetic lung could further asthma research
http://www.labtechnologist.com/news/ng.asp?n=84971-university-of-southampton-microfluidics-asthma-copd
Press release from Osiris
Here's the latest from Osiris -
http://www.businesswire.com/news/home/20080505006410/en
Here's the latest from Osiris -
http://www.businesswire.com/news/home/20080505006410/en
Osiris Submits !!!
Osiris and FDA Reach Agreement on Submission of First Marketing Application for a Stem Cell Product
COLUMBIA, Md., Jan 15, 2009 (BUSINESS WIRE) --
Osiris Therapeutics, Inc. (NASDAQ:OSIR) announced today that it has held a successful pre-Biologics License Application (pre-BLA) meeting for Prochymal used in the treatment of graft-versus-host disease (GvHD). A BLA is a comprehensive regulatory submission prepared by a biologic drug's sponsor to obtain full marketing approval from the Food and Drug Administration (FDA). As an outcome of the meeting between Osiris and FDA, the parties finalized the content and timing of what is expected to be the first BLA submission for a stem cell product.
"Agreement on the timing and content of the BLA marks an important milestone in our quest to make Prochymal the world's first fully approved stem cell therapy," said C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris. "We thank FDA for their significant efforts over the past several years as we have forged a highly productive partnership in this new and exciting area of medicine. Having proactively addressed issues critical to a successful review, we have created a clear understanding of the regulatory path to market approval."
A pre-BLA meeting is a mechanism by which FDA and the sponsor can discuss the application in advance of submission to avoid potential pitfalls in review process. In the Prochymal pre-BLA meeting there were three main areas of discussion. The first was the type and extent of safety and efficacy data that would be included. The second was manufacturing and quality data that would comprise the submission. The third was the process and format of the submission itself. Prior to the meeting, Osiris submitted a comprehensive outline of the proposed content and structure of the BLA.
Key results of the pre-BLA meeting are below:
FDA agreed with the proposed content and structure of the BLA for Prochymal for the treatment of GvHD.
The primary endpoint of the Phase III trial that will be reviewed by FDA for approval was confirmed to be GvHD Complete Response (CR). A CR is complete remission of the disease.
FDA indicated that the statistical analysis plan was appropriate.
The overall safety database (Integrated Summary of Safety) of Prochymal is sufficient in scope for the indication. No additional clinical data beyond what was presented is anticipated to be necessary for the BLA.
Agreement was reached on BLA requirements for Chemistry, Manufacturing and Controls (CMC) data.
The FDA concurred with the proposed product stability and process validation plan for submission and provided guidance on how to present the data for ease of review.
Agreement was reached on the timing and content of the BLA in rolling submission format. The rolling submission is an FDA provision available to drug candidates that have received Fast Track designation, which allows for completed sections of a BLA to be submitted on an ongoing basis. It can facilitate the process by allowing FDA to complete review of sections as soon as they are available. It is anticipated that the nonclinical sections, such as toxicology, will be the first submitted for review.
The BLA will be in the electronic Common Technical Document (eCTD) format. The eCTD format facilitates the review of the BLA and allows parallel submission of the dossier in other territories.
At the time of submission, Osiris will also submit the request for Priority Review of the Prochymal BLA. Investigational drugs with Fast Track designation are eligible for consideration for Priority Review, which provides for an accelerated six month application review by FDA.
Osiris and FDA also discussed the need for an Expanded Access Program (EAP) to make Prochymal available to critically ill steroid-refractory adult GvHD patients in the interim period until a final regulatory decision is reached. Osiris currently has an EAP in place for pediatric GvHD patients in the US and Canada. Osiris committed to work with FDA to determine the details of the new EAP.
In November 2008, Osiris and Genzyme announced a strategic alliance for the development and commercialization of Prochymal. Under the terms of the agreement, Osiris will commercialize Prochymal in the United States and Canada, and Genzyme will commercialize the treatment in all other countries.
Osiris and FDA Reach Agreement on Submission of First Marketing Application for a Stem Cell Product
COLUMBIA, Md., Jan 15, 2009 (BUSINESS WIRE) --
Osiris Therapeutics, Inc. (NASDAQ:OSIR) announced today that it has held a successful pre-Biologics License Application (pre-BLA) meeting for Prochymal used in the treatment of graft-versus-host disease (GvHD). A BLA is a comprehensive regulatory submission prepared by a biologic drug's sponsor to obtain full marketing approval from the Food and Drug Administration (FDA). As an outcome of the meeting between Osiris and FDA, the parties finalized the content and timing of what is expected to be the first BLA submission for a stem cell product.
"Agreement on the timing and content of the BLA marks an important milestone in our quest to make Prochymal the world's first fully approved stem cell therapy," said C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris. "We thank FDA for their significant efforts over the past several years as we have forged a highly productive partnership in this new and exciting area of medicine. Having proactively addressed issues critical to a successful review, we have created a clear understanding of the regulatory path to market approval."
A pre-BLA meeting is a mechanism by which FDA and the sponsor can discuss the application in advance of submission to avoid potential pitfalls in review process. In the Prochymal pre-BLA meeting there were three main areas of discussion. The first was the type and extent of safety and efficacy data that would be included. The second was manufacturing and quality data that would comprise the submission. The third was the process and format of the submission itself. Prior to the meeting, Osiris submitted a comprehensive outline of the proposed content and structure of the BLA.
Key results of the pre-BLA meeting are below:
FDA agreed with the proposed content and structure of the BLA for Prochymal for the treatment of GvHD.
The primary endpoint of the Phase III trial that will be reviewed by FDA for approval was confirmed to be GvHD Complete Response (CR). A CR is complete remission of the disease.
FDA indicated that the statistical analysis plan was appropriate.
The overall safety database (Integrated Summary of Safety) of Prochymal is sufficient in scope for the indication. No additional clinical data beyond what was presented is anticipated to be necessary for the BLA.
Agreement was reached on BLA requirements for Chemistry, Manufacturing and Controls (CMC) data.
The FDA concurred with the proposed product stability and process validation plan for submission and provided guidance on how to present the data for ease of review.
Agreement was reached on the timing and content of the BLA in rolling submission format. The rolling submission is an FDA provision available to drug candidates that have received Fast Track designation, which allows for completed sections of a BLA to be submitted on an ongoing basis. It can facilitate the process by allowing FDA to complete review of sections as soon as they are available. It is anticipated that the nonclinical sections, such as toxicology, will be the first submitted for review.
The BLA will be in the electronic Common Technical Document (eCTD) format. The eCTD format facilitates the review of the BLA and allows parallel submission of the dossier in other territories.
At the time of submission, Osiris will also submit the request for Priority Review of the Prochymal BLA. Investigational drugs with Fast Track designation are eligible for consideration for Priority Review, which provides for an accelerated six month application review by FDA.
Osiris and FDA also discussed the need for an Expanded Access Program (EAP) to make Prochymal available to critically ill steroid-refractory adult GvHD patients in the interim period until a final regulatory decision is reached. Osiris currently has an EAP in place for pediatric GvHD patients in the US and Canada. Osiris committed to work with FDA to determine the details of the new EAP.
In November 2008, Osiris and Genzyme announced a strategic alliance for the development and commercialization of Prochymal. Under the terms of the agreement, Osiris will commercialize Prochymal in the United States and Canada, and Genzyme will commercialize the treatment in all other countries.
Osiris Receives FDA Clearance to Broaden Prochymal Expanded Access Program
January 15 2009
Osiris Receives FDA Clearance to Broaden Prochymal Expanded Access Program
Access to Treatment Now Available to Patients of All Ages Suffering
from Life Threatening GvHD
COLUMBIA, Md., Jan 15, 2009 (BUSINESS WIRE) --
Osiris Therapeutics, Inc. (NASDAQ: OSIR) today announced U.S. Food and Drug Administration (FDA) clearance to broaden its expanded access program (EAP) for Prochymal, now making the investigational stem cell product available to adults with life-threatening Graft vs. Host Disease (GvHD). In May of 2008, FDA approved the first Prochymal EAP for the treatment of pediatric GvHD patients.
Congress and the FDA created the expanded access program to facilitate the availability of promising new drugs to desperately ill patients before general marketing begins. The program allows for investigational drugs to be made available to patients under certain circumstances during evaluation in late stage clinical trials when no satisfactory alternative therapy is available. For expanded access, the FDA must determine that the available scientific evidence, taken as a whole, demonstrates that the drug may be effective and would not expose the patients to unreasonable risks. Additionally, the FDA permits companies meeting certain criteria to charge for the investigational product. Expanded access to Prochymal was initially restricted to only pediatric patients suffering from steroid refractory GvHD until patient enrollment in the Phase III pivotal trial was completed.
Prochymal, a formulation of adult mesenchymal stem cells designed to provide therapeutic benefit by controlling inflammation, promoting tissue regeneration, and preventing scar formation, is in phase III clinical trials for GvHD and Crohn's disease. Enrollment in the Phase III pivotal trial for steroid-refractory GvHD was recently completed. Enrollment is ongoing in another Phase III trial for acute GvHD and in a Phase III trial for Crohn's disease.
"As a transplant physician who has used Prochymal, I have seen first hand the power of this promising therapy to reverse otherwise non-responsive GvHD," said Dr. Hans Klingemann, Director, Bone Marrow and Hematopoietic Stem Cell Transplantation Program at Tufts-New England Medical Center. "This is a disease that is so devastating that survival is often measured in days. This action by FDA is significant because it now enables us to provide all of our critically ill GvHD patients with faster and more reliable access to Prochymal."
GvHD is a life-threatening immune reaction that can occur in patients following bone marrow transplantation. Steroids are typically used to control symptoms of the disease, although they are only effective in approximately 35% of patients. Mortality can exceed 85% for patients with GvHD that do not respond to steroids.
Under the EAP, patients two months to 70 years of age, inclusive, who have been diagnosed with GvHD that is unresponsive to steroid therapy, are eligible to receive Prochymal. For consideration and further eligibility criteria please email prochymal@osiris.com.
In November 2008, Osiris and Genzyme announced a strategic alliance for the development and commercialization of Prochymal. Under the terms of the agreement, Osiris will commercialize Prochymal in the United States and Canada, and Genzyme will commercialize the treatment in all other countries.
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. Prochymal is currently being evaluated in Phase III trials for 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 being studied in Phase II trials for the treatment of COPD, type 1 diabetes, and acute myocardial infarction. Additionally, the Department of Defense recently awarded Osiris a contract to develop Prochymal as a treatment for acute radiation syndrome.
About Osiris Therapeutics
Osiris Therapeutics, Inc. is the leading stem cell therapeutic company focused on developing products to treat serious medical conditions in the inflammatory, orthopedic and cardiovascular areas. The Company's pipeline of internally developed biologic drug candidates under evaluation includes Prochymal for inflammatory, autoimmune, and cardiovascular indications, as well as Chondrogen for arthritis in the knee. Osiris is a fully integrated company, with capabilities in research, development, manufacturing, and distribution of stem cell products. Osiris has a partnership with Genzyme Corp. for the development and commercialization of Prochymal and Chondrogen in countries outside the United States and Canada. Osiris has developed an extensive intellectual property portfolio to protect the company's technology including 47 U.S. patents each having one or more foreign counterparts. Osiris, Prochymal and Chondrogen are registered trademarks of Osiris Therapeutics, Inc. More information can be found on the company's website, www.Osiris.com. (OSIR-G)
Forward-Looking Statements
This press release contains forward-looking statements. Forward-looking statements include statements about our expectations, beliefs, plans, objectives, intentions, assumptions and other statements that are not historical facts. Words or phrases such as "anticipate," "believe," "continue," "ongoing," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project" or similar words or phrases, or the negatives of those words or phrases, may identify forward-looking statements, but the absence of these words does not necessarily mean that a statement is not forward-looking. Examples of forward-looking statements include, but are not limited to, statements regarding the following: our product development efforts; our clinical trials and anticipated regulatory requirements and the ability to successfully navigate these requirements; the success of our product candidates in development; status of the regulatory process for our biologic drug candidates; implementation of our corporate strategy; our financial performance; our product research and development activities and projected expenditures, including our anticipated timeline and clinical strategy for Prochymal, Chondrogen and our other MSC and biologic drug candidates; our cash needs; patents and proprietary rights; the safety and ability of our potential products to treat disease and the results of our scientific research; our plans for sales and marketing; our plans regarding our facilities; types of regulatory frameworks we expect will be applicable to our potential products; and results of our scientific research. Forward-looking statements are subject to known and unknown risks and uncertainties and are based on potentially inaccurate assumptions that could cause actual results to differ materially from those expected or implied by the forward-looking statements. Risks and uncertainties related to the Collaboration Agreement with Genzyme include, among others: typical business transactional risks; risks related to product development and clinical trial design, performance and completion; uncertainty of the success of Prochymal and Chondrogen in clinical trials and their ability to treat disease; Genzyme's early termination and opt-out rights; the ability of Osiris and Genzyme to successfully navigate regulatory requirements and to manufacture and commercialize products; and the uncertainty as to the ability of the parties to successfully perform under the collaborative arrangement and for Osiris to earn milestone and royalty payments thereunder. Our actual results could differ materially from those anticipated in forward-looking statements for many reasons, including the factors described in the section entitled "Risk Factors" in our Annual Report on Form 10-K and Quarterly Reports filed on Form 10-Q, with the United States Securities and Exchange Commission. Accordingly, you should not unduly rely on these forward-looking statements. We undertake no obligation to publicly revise any forward-looking statement to reflect circumstances or events after the date of this press release
January 15 2009
Osiris Receives FDA Clearance to Broaden Prochymal Expanded Access Program
Access to Treatment Now Available to Patients of All Ages Suffering
from Life Threatening GvHD
COLUMBIA, Md., Jan 15, 2009 (BUSINESS WIRE) --
Osiris Therapeutics, Inc. (NASDAQ: OSIR) today announced U.S. Food and Drug Administration (FDA) clearance to broaden its expanded access program (EAP) for Prochymal, now making the investigational stem cell product available to adults with life-threatening Graft vs. Host Disease (GvHD). In May of 2008, FDA approved the first Prochymal EAP for the treatment of pediatric GvHD patients.
Congress and the FDA created the expanded access program to facilitate the availability of promising new drugs to desperately ill patients before general marketing begins. The program allows for investigational drugs to be made available to patients under certain circumstances during evaluation in late stage clinical trials when no satisfactory alternative therapy is available. For expanded access, the FDA must determine that the available scientific evidence, taken as a whole, demonstrates that the drug may be effective and would not expose the patients to unreasonable risks. Additionally, the FDA permits companies meeting certain criteria to charge for the investigational product. Expanded access to Prochymal was initially restricted to only pediatric patients suffering from steroid refractory GvHD until patient enrollment in the Phase III pivotal trial was completed.
Prochymal, a formulation of adult mesenchymal stem cells designed to provide therapeutic benefit by controlling inflammation, promoting tissue regeneration, and preventing scar formation, is in phase III clinical trials for GvHD and Crohn's disease. Enrollment in the Phase III pivotal trial for steroid-refractory GvHD was recently completed. Enrollment is ongoing in another Phase III trial for acute GvHD and in a Phase III trial for Crohn's disease.
"As a transplant physician who has used Prochymal, I have seen first hand the power of this promising therapy to reverse otherwise non-responsive GvHD," said Dr. Hans Klingemann, Director, Bone Marrow and Hematopoietic Stem Cell Transplantation Program at Tufts-New England Medical Center. "This is a disease that is so devastating that survival is often measured in days. This action by FDA is significant because it now enables us to provide all of our critically ill GvHD patients with faster and more reliable access to Prochymal."
GvHD is a life-threatening immune reaction that can occur in patients following bone marrow transplantation. Steroids are typically used to control symptoms of the disease, although they are only effective in approximately 35% of patients. Mortality can exceed 85% for patients with GvHD that do not respond to steroids.
Under the EAP, patients two months to 70 years of age, inclusive, who have been diagnosed with GvHD that is unresponsive to steroid therapy, are eligible to receive Prochymal. For consideration and further eligibility criteria please email prochymal@osiris.com.
In November 2008, Osiris and Genzyme announced a strategic alliance for the development and commercialization of Prochymal. Under the terms of the agreement, Osiris will commercialize Prochymal in the United States and Canada, and Genzyme will commercialize the treatment in all other countries.
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. Prochymal is currently being evaluated in Phase III trials for 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 being studied in Phase II trials for the treatment of COPD, type 1 diabetes, and acute myocardial infarction. Additionally, the Department of Defense recently awarded Osiris a contract to develop Prochymal as a treatment for acute radiation syndrome.
About Osiris Therapeutics
Osiris Therapeutics, Inc. is the leading stem cell therapeutic company focused on developing products to treat serious medical conditions in the inflammatory, orthopedic and cardiovascular areas. The Company's pipeline of internally developed biologic drug candidates under evaluation includes Prochymal for inflammatory, autoimmune, and cardiovascular indications, as well as Chondrogen for arthritis in the knee. Osiris is a fully integrated company, with capabilities in research, development, manufacturing, and distribution of stem cell products. Osiris has a partnership with Genzyme Corp. for the development and commercialization of Prochymal and Chondrogen in countries outside the United States and Canada. Osiris has developed an extensive intellectual property portfolio to protect the company's technology including 47 U.S. patents each having one or more foreign counterparts. Osiris, Prochymal and Chondrogen are registered trademarks of Osiris Therapeutics, Inc. More information can be found on the company's website, www.Osiris.com. (OSIR-G)
Forward-Looking Statements
This press release contains forward-looking statements. Forward-looking statements include statements about our expectations, beliefs, plans, objectives, intentions, assumptions and other statements that are not historical facts. Words or phrases such as "anticipate," "believe," "continue," "ongoing," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project" or similar words or phrases, or the negatives of those words or phrases, may identify forward-looking statements, but the absence of these words does not necessarily mean that a statement is not forward-looking. Examples of forward-looking statements include, but are not limited to, statements regarding the following: our product development efforts; our clinical trials and anticipated regulatory requirements and the ability to successfully navigate these requirements; the success of our product candidates in development; status of the regulatory process for our biologic drug candidates; implementation of our corporate strategy; our financial performance; our product research and development activities and projected expenditures, including our anticipated timeline and clinical strategy for Prochymal, Chondrogen and our other MSC and biologic drug candidates; our cash needs; patents and proprietary rights; the safety and ability of our potential products to treat disease and the results of our scientific research; our plans for sales and marketing; our plans regarding our facilities; types of regulatory frameworks we expect will be applicable to our potential products; and results of our scientific research. Forward-looking statements are subject to known and unknown risks and uncertainties and are based on potentially inaccurate assumptions that could cause actual results to differ materially from those expected or implied by the forward-looking statements. Risks and uncertainties related to the Collaboration Agreement with Genzyme include, among others: typical business transactional risks; risks related to product development and clinical trial design, performance and completion; uncertainty of the success of Prochymal and Chondrogen in clinical trials and their ability to treat disease; Genzyme's early termination and opt-out rights; the ability of Osiris and Genzyme to successfully navigate regulatory requirements and to manufacture and commercialize products; and the uncertainty as to the ability of the parties to successfully perform under the collaborative arrangement and for Osiris to earn milestone and royalty payments thereunder. Our actual results could differ materially from those anticipated in forward-looking statements for many reasons, including the factors described in the section entitled "Risk Factors" in our Annual Report on Form 10-K and Quarterly Reports filed on Form 10-Q, with the United States Securities and Exchange Commission. Accordingly, you should not unduly rely on these forward-looking statements. We undertake no obligation to publicly revise any forward-looking statement to reflect circumstances or events after the date of this press release