- Isis Pharma presented 9/8/2011 at the Stifel Nicolaus Healthcare conference - click here for full biotech webcast presentation schedule and links to my notes.
- For complete pipeline and partnership information, visit the ISIS stock research page.
- See my complete notes below:
- Presenter: Lynne Parshall COO/CFO
- New and/or noteworthy comments are bolded. Much of the webcast was freeflowing q&a format
- 4 of 23 drugs in pipeline will be presenting data this year. all phase 1 but always try to get pharmacology from these
- FXI - bleeding parameters (pot to be much better profile that xa inhibitso)
- ApoCIII - triglyceride levels
- SGLT2 - glucose challenge (measure glucose excretion)
- TTR - just target lowering. But can move rapidly into phase 2
- before the EU filing, had mipomersen meetings with rapateur countries. SNY has not disclosed which they are, but it is well known in EU what countries are important for lipidology
- We have received administrative questions and have taken care of those. Next step is then 120 days for review of dossier...then in late november or early december they will return with questions for us to address
- NDA filing with FDA will be in 4q2011.
- We plan to have the drug (Kynamro trade name) on the market in US and EU next year
- we were "holding our breath" after genzyme acquisition, but have been nothing but pleased so far. Our collaboration falls within the group at genzyme that Sanofi has said will be kept intact. There has been little turnover in the team we deal with...we've seen very little of SNY, but they have been able to offer some resources
- genetic testing for FH would be very difficult absent whole genome sequencing as there are over 2000 mutations in LDL receptor desfribed, plus other genes...so FH in practice is diagnosed based on clinical LDL levels, etc.
- EU: filing covered hoFH and severe heFH (defined as being on maximally tolerated therapy with >200 LDL with another CV risk factor or >300 without)
- US first filing for just hoFH. FDA wants much larger database for larger severe HeFH population. We will start the one year exposure trial later this year.
- initial filings cover about 40k patients. So consider at bare minimum price of $100,000 per year, the drug will sell $100m per every 1000 patients - 25% penetration required for blockbuster status...without any expanded population approvals
- discontinuation rate - typical of 6 month subcutaneous drugs (byetta, etc, about 20% was expected)...and this was with a rigorous protocol, sometimes twice weekly doctor visits, lots of blood draws etc....don't think it's that applicable to compliance
- We have over 100 pts treated more than a year in US package. ALT elevations in ~8% >3x ULN were associated with the most rapid and most profound LDL reductions - so generally occurred early in treatment.
- liver fat - the human case is turning out just how the preclinical data predicted. We see slight increases at 6 months which sometimes continue to 12 months. We now have data presented in late august showing that liver fat levels plateau and then go down. Our mice data indicated that in the short term, reduction in apoB causes fat buildup before the body compensates by 1) making fewer lipids 2) increasing metabolism, to get back to equilibrium amount of fat
- Pricing-what is the imapct of maybe going after larger market later? First, we picked the best partner in the world for that. Genzyme has not set a price, but the $100,000 annual cost of apheresis is the benchmark, and this is already reimbursed
- AEGR is talking about pricng at $300k or so. Comment? I'd love it they price there, that would make our drug look very inexpensive. And we are looking at much larger population vs them.
- Will SNY provide sales guidance? - Henri (GENZ CEO) was really good about doing this. We need to have that conversation with Sanofi over the next 6-9 months. I'm not sure they'll put out expected penetration/ramp numbers.
- our plan is to partner at clinical proof of concept...which can be very different depending on disease. One could argue that ApoCIII phase 1 lower target protein and triglyceride levels lowering could represent that. Very different vs cancer trials with survival endpoint...we consider this program by program
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