Osiris Prochymal - Crohn's Disease and Stock Update

As I have mentioned quite a few times, Osiris Prochymal has all but put my Crohn's in Remission. Because of that I pay attention to Osiris. I am not sure what the exact status of their stem-cell trial for Crohn's is, but if you can look into it, I recommend you see if it is right for you. I will work on an update on the current trials available for Prochymal and Crohns.

Shares of Osiris Therapeutics Inc. (have appreciated as much as 20% over the past five days as traders and investors are placing their bets ahead of the impending release of late-stage trial results of the company's stem cell drug candidate, Prochymal. The stock is currently trading around $14 as of this writing. Osiris Therapeutics got a drubbing on Wall Street in March, when it suspended trials of its pill for Crohn's disease, citing an "unusually high" response to placebo.

Prochymal is an intravenously administered formulation of mesenchymal stem cells obtained from the bone marrow of healthy adult donors and is designed to provide therapeutic benefit by controlling inflammation, promoting tissue regeneration, and preventing scar formation.

Prochymal in Crohn's disease

As recently as March 27, Osiris stopped enrollment in a phase III study, which is testing Prochymal as a potential treatment for Crohn's disease due to a systemic design flaw in the trial. Shares plummeted 31% to a low of $12.62 that day following the news. The data from the trial is expected to be used in redesigning future studies.

Osiris Stops Enrollment in Prochymal Stem Cell Study!

Whoah, huge news here. Anyone else participated in this Osiris Prochymal Stem Cell Study (I did, but with mixed results).

Stem cell research firm Osiris Therapeutics Inc (OSIR.O) said it stopped enrolling patients in a late-stage trial to test its lead drug candidate, Prochymal, in Crohn's disease due to design flaws in the study.

The company would continue the trial with patients already enrolled and use data from the trial to redesign future studies. However, it does not expect to get Prochymal approved for Crohn's disease through this trial.

Leerink Swann analyst William Tanner, who had expected the trial to end without difficulties, said he expects the Crohn's disease trial to get pushed by a couple of years. Tanner does not expect the enrollment halt and design flaws to impact other indications in which the drug is being tested.

Osiris, which developed Prochymal from adult stem cells, is studying the drug in several other indications like graft versus host disease, chronic obstructive pulmonary disorder (COPD), myocardial infarction and diabetes.

However, Lazard Capital Markets analyst Joel Sendek is very sceptical of the drug in any indication. "I don't think the drug works very well, and I have not seen any evidence that the drug works in Crohn's disease," said Sendek, who has a "sell" rating on the company.
"The company has moved too fast in the late-stage studies. They had very few early- and mid-stage data," Sendek said.

Osiris halted enrollment after the final interim analysis showed that one of the two Prochymal dose arms was unlikely to achieve the main goal of the study due to high response rate seen in the arm that was on dummy drug.

"After careful discussion with the FDA, we elected to discontinue enrollment rather than attempt to re-power the trial," Chief Executive Randal Mills said in a statement. Osiris has a commercial collaboration with Genzyme Corp on Prochymal. However, according to the terms, Osiris would have to bear the full costs of a new late-stage Crohn's disease study.
Shares of Osiris were down about 23 percent at $14.05 in morning trade Friday on Nasdaq. They had touched a low of $12.62 earlier. (Editing by Anil D'Silva, Ratul Ray Chaudhuri)

Osiris Prochymal Wins Even More FDA Approval

Thursday, Columbia-based stem cell therapeutic company, Osiris Therapeutics Inc. , said that it has received clearance from the U.S. Food and Drug Administration, or FDA, to broaden its expanded access program for Prochymal, thereby making the investigational stem cell product available to adults with life-threatening Graft vs. Host Disease or GvHD.Prochymal is currently in its Phase III clinical trials for GvHD and Crohn's disease. 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.

Osiris noted that under the expanded access program, patients from 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. Additionally, 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.

Awesome Opinion on FDA, Crohn's and Osiris

Written by Gideon J. Sofer- University of California, Berkley student - from WSJ

Right now there are millions of individuals whose lives are directly dependent on the rate at which new drugs come to market. I'm one of them. I'm fighting for my life.

To date, half of my intestine has been removed to manage Crohn's disease. Last year, at age 23, I enrolled in a clinical trial for a treatment that could save my life: an adult stem-cell therapy that helps damaged intestinal tissue regenerate from the relentless inflammation and scarring caused by Crohn's.

The sponsor, Osiris Therapeutics, reported that Crohn's patients in the therapy's Phase II trial all experienced clinical improvement after receiving the cells. A Phase III trial for the treatment is now nearing completion, but Food and Drug Administration (FDA) approval could be years away, despite its FDA "fast track" designation.

In accordance with antiquated FDA policies, the Phase III trial is randomized with three groups of patients, and double-blinded, which means neither the doctors nor patients are told what treatment is being administered. One group received full-strength stem cells, another received half-strength, and a third got a placebo (the proverbial "sugar pill"). It appears I got the placebo.

Foregoing all other treatments, I received the four scheduled infusions, and yet my disease progressed with a vengeance. In a matter of weeks, I became dangerously malnourished. I've since been readmitted to the hospital countless times, as my doctors continue to plead with Osiris for information. But Osiris has refused, citing adherence to FDA protocol.

I am now a lab rat. I have no right to know what happened to me in the study, nor do I have a right to try the promising treatment as my health deteriorates. It doesn't have to be this way.
Under the Fifth Amendment's guarantee that "No person shall be deprived of life, liberty or property without due process of law," a critically ill patient should have access to a potentially lifesaving drug that has been deemed safe for human consumption, if the patient agrees to bear the risks involved. But earlier this year, the Supreme Court refused to hear a case on the issue, denying countless patients their right to pursue life.

Thankfully, some members of Congress have stepped in to ensure our rights as patients. In May, Sen. Sam Brownback (R., Kan.) and Rep. Diane Watson (D., Calif.) introduced the Access, Compassion, Care and Ethics for Seriously Ill Patients Act. If passed, this bipartisan legislation will begin to restore the rights of millions of patients by widening access to promising investigational drugs.

Human clinical research is an intricate scientific and moral process, but it does not justify taking immoral advantage of patients. Tragically, FDA and Osiris think it does.

Typical approval protocols almost always guarantee patients taking the placebo access to the actual drug -- at the very least -- after the study has ended. But in what appears to me a deliberate act of cruelty, Osiris hung its patients out to dry without any recourse, refusing to confirm which patient got what. The FDA has endorsed Osiris's decision by enabling it to proceed with the study.

Withholding a potential cure is just as bad -- if not worse -- than the potential death sentence of a serious illness. If patients like myself have the audacity to put their lives on the line for the betterment of science and those in their predicament, their decision should not only be embraced, it should be rewarded.

Furthermore, trials without ethical recourse can lead to inadequate and incomplete data, compromising the integrity of the study. If trial patients are treated like lab rats, they won't feel obliged to cooperate unconditionally and report accurate data -- something the FDA and the drug industry rely on heavily, but have failed to consider.

Everyone agrees it is a fundamental right for patients to dictate their course of treatment with FDA-approved drugs. So why do the rules evaporate at the most critical moment, when the only life-preserving options are highly promising investigational drugs?