) -- This week's Biotech Stock Mailbag covers questions about
(ZIOP - Get Report)
and the competition between
(GSK - Get Report)
to develop new Duchenne Muscular Dystrophy drugs.
Before I get to the reader email and Tweets, I want to invite everyone to hang out with me on Monday, March 4, when I live-blog the
(DEPO - Get Report)
FDA advisory panel reviewing Sefelsa as a new treatment for post-menopausal hot flashes. Joining me on the live blog to provide color, analysis and perhaps some fiery arguments will be Zacks analyst Jason Napodano (a Depomed bull) and Aafia Chaudhry, well-known Twitter biotech investor, a contributor to
and a Depomed bear. You can sign up for an email reminder below.
Christian F. writes,
"Adam, your insight/predictions have helped me over the years. Your summary for how the Feuerstein-Ratain Rule applies to Ziopharm's phase III study was very well written. Please make a prediction on the study results."
I believe Ziopharm wins with the palifosfamide sarcoma study, meaning a statistically significant improvement in progression-free survival -- the study's primary endpoint. I'll predict a positive trend towards longer survival favoring palifosfamide but not statistically significant. This will be the most closely watched and perhaps controversial secondary endpoint of the study, but I think win on the primary endpoint plus a survival trend in the right direction will be sufficient for palifosfamide to gain FDA approval.
I am more positive on Ziopharm than the
>odds indicate using the Feuerstein-Ratain Rule
My contributors have covered Ziopharm, too. Aafia Chaudhry has
>concerns about palifosfamide hitting statistical significance
on the study's primary endpoint but she sees FDA approving the drug, regardless. Trader Tony Pelz offered an options trade idea heading into the release of the palifosfamide results at the end of the month.
I almost forget until I checked my archive, but I wrote a fairly detailed explanation supporting Ziopharm last October. The best proxy for the palifosfamide data are results from the study of ifosfamide/doxorubicin vs. Doxorubicin presented last year.
Forgive me for repeating myself, but here's
>what I wrote last October
The addition of ifosfamide to doxorubicin (ifo/dox) in front-line sarcoma resulted in a 68% reduction in the risk of disease progression compared to doxorubicin alone. (The hazard ratio was 0.74 and statistically significant.) Median PFS was 7.4 months for ifo/dox versus 4.6 months for dox alone.
Overall survival was not improved, however. Ifo/dox reduced the risk of death by 17% compared to dox alone (hazard ratio of 0.83) but the survival trend was not statistically significant. At the median, overall survival was 14.3 months for ifo/dox versus 12.8 months for dox alone. Ifo is not easily tolerated so a high percentage of dropouts likely contributed to blunting the observed survival benefit trend.
Based on these confounding results, the use of ifosfamide in the treatment of sarcoma patients was deemed unjustified.
Ziopharm's PICASSO 3 sarcoma study compares the combination of palifosfamide and doxorubicin against doxorubicin. PFS is the primary endpoint, with results expected before the end of the year. If positive, Ziopharm intends to seek accelerated approval for palifosfamide, supported (hopefully) by survival data that will be mature next year. [Note: Survival data will mature later this year.]