This is a cross-post from the Chiron group.
Check your facts. Why did you say my sell recommendation is based on the Phase II post-polio data? I never said that. I specifically said the problem was with the Myotrophin ALS data. The data to be presented at AAN is NOT as positive as you say. Cephalon did two Phase III trials. The North American trial enrolled 266 patients and covered 18 months, 9 months of treatment with 0.1 mg/kg/day and 9 months of follow-up. At 9 months there was a statistically significant improvement on the AALS scale, but there was a non-significant survival benefit in favor of the PLACEBO group. The 9 month open-label follow-up period showed the treated group lifespan was extended by 5.5 months.
The second Phase III was a European trial with 183 patients. Patients again received placebo or 0.1 mg/kg/day for 9 months. There were MORE DEATHS among those taking Myotrophin (18 of 124 treated) than placebo (5 of 59), although the difference was not statistically significant. Using the same three-point slope analysis as the U.S. study, the European trial was NOT statistically significant. Cephalon then extended to two additional data points and did (barely) achieve statistical significance.
My review of the studies, remembering that according to you I do not know my ass from my elbow about biotechnology, is that with increased mortality in the European trial plus the difficulty of comparing two studies that had different populations, different standards of care and different methods of analysis, Myotrophin is unlikely to be approved. This is IMO, as FDA panels can do whatever they want to do. But note that at last year's advisory panel meeting that finally allowed a treatment IND, several panel members including the chairman recommended that Cephalon do another clinical trial before seeking approval of an NDA. This Cephalon did not do. It is normal for the FDA to request a third pivotal trial when data from the first two do not corroborate each other. Note that over a year ago the Peripheral & Central Nervous System Drugs Advisory Committee specifically determined that the second pivotal study failed to confirm the first Phase III.
The problem with the pooled study presented at the AAN is the underlying question of the statistical validity of post-hoc analysis of pooled data from trials that are so different. On March 13 the FDA issued draft guidelines on evidence of efficacy standards that make it clear a single study has to be very positive, with lots of internal corroboration, to be acceptable. The Pink Sheets for March 17 cited Myotrophin as an example of a single study that does NOT qualify under the new guidelines.
Do you not find it interesting that Dr. Monroe Klien (head of regulatory) and Dr. Michael Murphy (head of clinical) have left the company? You say I know "little or nothing" about biotechnology, but I can tell when risks are high. None of this is Chiron's fault, and the brunt of a turndown by either the advisory committee or the FDA will fall mostly on Cephalon, not Chiron. Our cost of CHIR is $1 a share, and for those with a tax problem or those willing to ride through a $3 to $5 drop (or buy more), I would hold the stock. For those who don't like the risk and can trade in and out I would sell and buy back.
BTW, if we don't know anything about biotech, how did we get a $1 cost on CHIR? Just lucky, I guess. |
