- Current kynamro activities:1) NDA filing this month; 2) launch 2012 us and eu; 3) Genzyme SPA agreement with FDA for FOCUS FH 12 month study that will start this year. Goal is to expand US population to severe heFH, and also offer 3x weekly dosing applications. Also may possibly allow expansion beyond severe heFH in EU (to patients with LDL greater than 160)
- We initiated clinical development on 5 drugs this year, and added 2 drugs to pipeline.
- 4q-2011 we will have clinical data on multiple drugs
- modest increase in expenses 2h2011 due to pipeline avdances to more expensive phase 2 studies, same reason for modestly higher 2012 expenses forecast
- We are close to the $125m spending mark for kynamro, then costs will be 50/50, so no increase in burn on that program 2012 over 2011.
- $25m milestone for NDA acceptance - could be late 2011 or early 2012 depending on if FDA takes full 60 days to accept - affect end of year cash and yearly NOLs
- focused on patients with obvious severe and unmet medical needs. 4 studies with compelling efficacy and acceptable safety. Mipomersen is the only drug that lowers all atherogenic lipids.
- recent Circulation article - 26% reduction in LDL extends lifespan from 18 yrs to 32 yrs (26-37% recution in ISIS studies on top of results of other drugs)
- expand martket with FOCUS FH study (but intial population is still a significant market) - will have fewer than 500 pts, severe and less severe heFH, plus statin intolerant patients, start by ye2011
- launch prep includes increasing awareness of HoFH disease - diagnose earlier and treat more aggresively than today
- CRP: 70% reduction in normal volunteers - first selective CRP inhibitor. broad phase 2 program underway
- APOC-III we will know in phase 1 whether we have an effective drug. measuring protein and triglyceride levels in blood. data next month
- FXI data next month - again will know if lowers target and if this causes any bleeding
- FGFR4 first trial soon
- SGLT2 data very late 2011 or early 2012
- Phase 2 of EIF4E "progressing nicely"
- Eli Lilly $LLY continues to make progress on partnered drug
- TTR phase 1 data in the next quarter - will know if active agent at end of phase 1. very rapid route to market with GSK
- SMN phase 1 begin very shortly
- SOD1 ALS we are moving forward with study
- 3 more drugs advance to pipeline by ye-2011
- we will host pipeline investor call in december
- january 5th NYC r&d day to summarize data readouts
reason for mipomersen dosing change? not changing, contination of plan laid out years ago. objective is to do weekly and 3x per week dosing in this study in order to provide option to patient.
how many arms? weekly, 3x weekly, placebo arms
doctor and payer education to date? most important advance is identification of FH as underdiagnosed severe CV disease. We have had a presence at every major relevant meeting, plus with patient advocacy groups. working with US and EU payor agencies - augmented by SNY infrastructure.
if NDA accepted in 2012 - will you miss NOL and cash guidance by $25m? Yes.
SPA - what is needed to achieve to get broader label? same as 4 previous trials - lowering cholesterol (primary endpoint) and additional safety experience. i am as confident as i have ever been for success - same safety profile would be acceptable. no additional safety monitoring requested. FDA wants to see 12 months dosing and more patients representative of larger market.
if you get the expected result, how much broader would EU label be? nothing is certain until label is negotiated, hope for all heFH >160 LDL on maximum lipid lowering therapy.
Press releases on data before 1/5 event? yes for most of it.
expected FOCUS FH enrollment timeline? too early to say. better handle on this once we enroll for a few months.
anticipate standard 10 month review for mipomersen at FDA? until we file and talk to FDA about review time, is premature to contemplate
competitor santaris HCV data at AASLD - litigation update? no update on litigation. we believe that we and regulus own the mir-122 drug. so we are exicted to see positive data, we will pursue appropriate steps to ensure enforcement of our patents
regulus paper published recently - any other updates? beginning to undestand microRNA pathway and how to exploit for appropriate therapeutic index. very pleased with where Regulus stands and patent position.
dosing mipomersen 3x weekly - any accumulated data on this regimen from other trials? Do you get less ISRs? we did study of daily vs 3x vs weekly for apoB and LDL reduction and tolerability. lower doses leads to less ISRs. we are not concerned about ISRs, even for weekly. tradeoff of more injections vs less reactions - which is a personal preference. no daily in this study because we want to continue to look at right presentation for daily (right injector, etc), but expect we will eventually look at daily.