I'm no doctor. I'm not educated. I don't claim to be a stock guru. But I'm not entirely stupid either. I was here when Synthrx was acquired. I was here when Culley made it clear he was taking his time seeking all the counsel he could muster to pull together a SMART protocol for this P3 Sickle Cell. I think he was smart. There has been a change in protocol a little as there will be a handful of adult patients involved in this trial some how or another but it is primarily pediatric patients 18 and under. No pain scale for endpoint like in the last trial but timed from the time they stick that needle and open the valve until the last dose of pain medication.
At the Annual Sickle Cell Disease Clinical Research Meetings held in August, John Keefer of the John Hopkins University School of Medicine provided the rationale for the phase 3 study. The study will focus on children since a previous phase 3 study by Orringer et al in 2001 who found that while the entire population of persons with sickle cell disease in the study (n=249) did not show statistically significant difference in the length of crisis (MST-188, 132 hrs vs placebo, 140 hrs; P=0.072), a post hoc analysis focused on the children (n=72) did show statistically significant improvement in length of vaso-occlusive crisis (MST-188, 127 hrs vs placebo 149 hrs; P=0.01).
Prior-Sponsor Studies in Sickle Cell Disease
Among patients under 16 years of age (n=73), there was a 21.6-hour
decrease in the duration of vaso-occlusive crisis in the MST-188 group compared to the placebo group (p=0.010).
I thought this alone was worth rolling the dice and just going long. I've been surprised many times at how this stock trades. I need to keep my mouth shut! But in the long run I hope to have the last laugh. Wish me luck, don't cuss me. My little bit of shares is not going to affect a thing on way or another.
Good luck to everyone else.
Buy, Sell,Trade, Hold, Short it. I really don't care. And that's the truth.
I'm fairly optimistic too long term, but not happy at all with the current PPS. In fact, it's a complete scandal.
The market cap makes absolutely no sense at all when factoring in drug pipeline and cash in the bank.