Question-and-Answer Session
Operator
(Operator Instructions) Your first question comes from Mark Monane with Needham; please proceed.
Mark Monane - Needham
Good morning everybody and thanks for reviewing your progress with us. A couple of questions here; you presented some nice data on the efficacy of mipomersen, can you please update us on the safety profile in animals? I know you’re doing the mouse and the rat studies. What’s the status of those projects?
Dr. Stan Crooke
You are referring to the carcinogenicity studies, Mark?
Mark Monane - Needham
Yes, please.
Dr. Stan Crooke
Yes, well you know, we finished the entire toxicology package, except for the carcinogenicity studies and we are confident that the preclinical data support the registration. The news from the CARC studies is equally great news. We have completed the mouse CARC study, and I’m pleased to tell you that the drug performed great and there are no significant findings. So that’s great news.
The rat study, the dosing continues and again, it’s going to be a satisfactory study that is, that animals are surviving adequately. So the drug looks great past that mouse, and we believe the rat looks every bit as good.
Mark Monane - Needham
That was helpful, and then going back to people, can you describe for us, in 2010 what data will be available for that package that you brought up earlier, in which the population will be 25,000 to 35,000? Can you review that for us?
Dr. Stan Crooke
Yes, we will have the homozygous FH trial, which is about 120 plus patients. As I say, the enrollment is complete and that study is progressing nicely. We will have the severe hypercholesterolemia patient study, people who look a lot like homozygous FH patients but aren’t genetically diagnosed, homozygous FH patients. That will be in the range of probably somewhere around 50 patients.
We’ll have the high cholesterol, high cardiovascular risk study, that will be in about 180 patients and then we’ll have the statin-intolerant study, which is about 30 patients. In addition, we will have significant information from some larger safety studies that we’re doing, and finally, we will have quite a significant number of patients who will have been followed for a year and two years in the open label rollover studies, to which all of these patients and all of these trials are being admitted for long term follow up.
So for this indication, I think somewhere between 700 or so total patients exposed and we’ll have a significant fraction of patients who will have been treated longer than a year, and some out two plus years.
- To read the full transcript on Seeking Alpha, click here »
