- ALNY released 1q2011 results and held a conference call on 5/2/11. Click here for PR.
- EDIT: Important updates from this webcast have been incorporated into the ALNY stock research page. This webpage is your source for all background info on ALNY, RNAi, partnerships, drug candidates, etc.
- See below the jump for my notes from the webcast:
- We are "transforming from platform company to product company"
- "5x15" vision- genetically-defined diseases w/ high unmet medical need. 5 in late stage clinical trials by end of 2015 (apparently they don't plan on any of them failing in p1 or p2)
- TTR human p1 placebo controlled data 3q11. Have received approval to expand to higher doses based on favorable safety data (expand 28 to 36 pts)- goal show decrease in plasma TTR levels in ATTR pts. Have EU orphan designation. Plan IND for backup 2h11
- PCSK9 IND 2q11, goal is human data ye11 (seems like an aggressive timeline)
- VSP data asco 2011. Completed enrolled in April for p1 in liver cancer pts (<40 pts). some continue to receive therapy. intend to partner before p2
- HPN- refractory anemia. goal IND 2012
- designate #4 and 5 for 5x15 goal from exisitng pgms later this year
- no further detail on Tekmira lawsuit- suggest to read response/counterclaim
- RSV enrolling p2b trial. recd approval to perform interim analysis (binded to ALNY) to adaptively adjust sample size, could increase to 120 pts. do this analysis when 75% pts are evaluable. accrual rate has exceeded expectations, have reached target of 76, still enrolling until interim analysis later this yr. data 2012
- 3/31 cash and investments of $343m (10m takeda milestone and 10.7m tax refund recd 1q11)
- r&d $26m in 1q11- will decrease slightly rest of 2011
- have now recd full $150m in upfront and tech transfer payment from takeda. remain impt partner. Significant possible future milestones and royalties. opt-in rights for us mkt
- ye11 >$275m cash. multiyear runway
- q&a session:
- when takeda into p1? publically said IND in 2013-2014 timeframe (this seems really slow to me, but not sure of details of what they are trying to do)
- Why RSV interim look? had always wanted to do interim look. recently completed discussions about this w/ FDA. based on very small p2a study (~25pts), want to reproduce w/ more robustness. did not answer whether there is alpha burn
- p3 RSV trial size? life threatening complication w/ 50% mortality...could affect fda's desired study size...some small chance this study is sufficient for filing, but FDA may well want data reproduced in similar sized study...current one is largest p2b among this transplant pt population.
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