Canada hands Osiris historic approval for stem-cell drug

Posted under adult stem cells, Blog, Companies, Diagnostics, Funding, graft versus host disease, Medical Devices, Medical Supply, Osiris Therapeutics, Pharmaceuticals, Pipeline, Prochymal, Regulatory, Startups, Type 1 Diabetes, Universities, Videos by Ryan McBride

Osiris Therapeutics ($OSIR) has made history. The Columbia, MD-based biotech has won the first approval of an off-the-shelf therapy made with stem cells, a major milestone for the field but one with unremarkable business implications for the developer.

Canadian authorities have stamped an approval on Osiris' Prochymal, a drug made with mesenchymal stem cells from adult donors, to treat certain children with potentially lethal graft versus host disease (GvHD), the company announced Thursday. The stem-cell drug has approval only for children whose disease can't be controlled with steroids, which doctors typically use to ward off the immune attacks from the ailment. Osiris will have to conduct post-approval clinical studies as a condition of the market nod for the treatment in Canada.

"This is the first regulatory approval of a stem-cell drug--where the active ingredient of the drug is a stem cell--in the world," Osiris CEO Randal Mills said, as quoted by Bloomberg. "It's a huge deal for us and a huge deal for the entire field of stem-cell therapy."

The approval provides a welcome turnaround for Osiris, which has been riddled by a string of recent setbacks. In February French drug giant Sanofi ($SNY) ditched work on Prochymal, for which the company had rights the sell outside of North America, The New York Times reported. The FDA last year rejected Osiris' bid for approval of the therapy as a treatment for GvHD, requesting more data from the developer. And the stem-cell field as a whole suffered a blow when Geron ($GERN) revealed last year that the biotech was shutting down its stem-cell programs.

Yet Osiris hasn't hit the jackpot. After spending years developing Prochymal, the company is expected to make only modest revenues from the drug for children in Canada with GvHD. Piper Jaffray analyst Edward Tenthoff projected sales of the drug to hit $16.7 million next year, Bloomberg reported. In the meantime, Osiris aims to build on this approval with another regulatory submission in the U.S. this year and has been developing the drug for other uses such as Type 1 diabetes, Crohn's disease and for patients who have suffered heart attacks.

- here's the release
- get more from the NYT article
- check out Bloomberg's report

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PhRMA’s COPD pipeline report spotlights blockbuster hopefuls

Posted under Blog, Companies, COPD, Diagnostics, Elevation Pharmaceuticals, Funding, GlaxoSmithKline, Medical Devices, Medical Supply, Novartis, Osiris Therapeutics, Pearl Therapeutics, Pharmaceuticals, PhRMA, QVA149, Relovair, Startups, Theravance, Universities, Videos by John Carroll

Anyone looking for a basic primer on experimental COPD drugs should check out PhRMA's latest compilation of new drugs in the pipeline. The big trade group found 54 COPD studies underway, which include trials for some of the biggest potential blockbusters nearing an FDA decision. But be prepared to do some digging if you want to find out what's really happening here.

For PhRMA, which mistakenly categorized everything in clinical development as distinct, late-stage treatments, some of the highlights in the report include an adult stem cell therapy, an IL-1 receptor-targeted antibody and a therapy that zeroes in on inflammation related to COPD. Their success could help reduce the nearly $50 billion annual tab for treating the disease.

Dig down into the report and you'll find eight Phase III studies underway. That includes Relovair, a new drug from GlaxoSmithKline ($GSK) and Theravance ($THRX), which chilled analysts with mixed data recently, and Novartis' ($NVS) potential giant slayer QVA149, which has run into dosage issues in the U.S. Pearl Therapeutics, a 2011 Fierce 15 company, is in the race now that it is wrapping an ambitious mid-stage program.

Elevation and Osiris are also focused on Phase II. Osiris's Prochymal, the stem cell treatment noted by PhRMA, hasn't performed very well. In an update, Osiris recently noted that the treatment had failed to outperform a placebo after a year of treatment.

- here's the press release
- read the full report (.pdf)

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Osiris stem cell treatment can’t beat placebo in diabetes study

Posted under Blog, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, Osiris Therapeutics, Pharmaceuticals, Prochymal, Startups, Universities, Videos by John Carroll

It takes a considerable level of patience, but if you dig deep into Osiris Therapeutics's latest update on its mid-stage study of the adult stem cell therapy Prochymal for Type 1 diabetes, you'll find that after a year of treatment patients taking the therapy aren't doing any better than the placebo arm when it comes to a key endpoint of the study.

The study still has a year left to run, and the biotech touted data on how well patients tolerated the treatment and how it matches the placebo on adverse events. Then there's this note: "No significant differences in the rates of disease progression, as measured by stimulated C-peptide levels at the one year time point, have been observed. However there was a trend towards fewer hypoglycemic events for patients treated with Prochymal as compared to controls."

Osiris ($OSIR) has been harshly criticized in the past for spinning the news on Prochymal, which ran into some severe trouble in the clinic when it was studied as a potential treatment for graft vs. host disease, Crohn's disease and more. TheStreet's Adam Feuerstein, in particular, has enjoyed heckling the company for its sunny releases. And he wasn't missing a beat following today's announcement.

Following a note on Osiris's regulatory troubles, Feuerstein notes: "Osiris finds it easier apparently to bamboozle retail investors about failed stem-cell therapies than regulators."

- check out the Osiris release
- here's the story from TheStreet

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