- ALN-TTR02 two-dose phase 2 in ATTR myopathy patients and ALN-TTRsc phase 1 (FAC patients) data should both be available within the next month or so - "middle of the year" for the phase 1 and a conference starting in late June for the TTR02 data.
- The ALN-TTR02 phase 3 trial would involve an as-yet undisclosed myopathy composite endpoint and a 12 or 18 month duration of treatment for the primary endpoint. Discussions on the protocol ongoing with both EMA and FDA. Overall would expect the trial to take 2.5-3.5 years to complete the trial with 150-200 patients.
- AT3 (hemophilia) IND should be submitted soon and start phase 1 this year with data in 2014, in patients.
- As I have been saying for ages, IV version of ALN-PCS is dead and the go forward product with The Medicines Co. MDCO with use the GalNac SC delivery system. CEO said that they haven't given guidance on when they will reenter phase 1, but said that if everything goes well they hope it could be "next year"
- Love the irony of how now that the Tekmira Pharma TKMR settlement is in the past, the LNP RNAi delivery technology is no longer "proprietary" and awesome, but is now stale leftover bread with no value proposition.
These notes are from memory - please correct via the comment section, email, or Twitter if I messed up or missed something important. Thanks in advance.