- Alnylam presented 9/19 at the UBS conference - click here for complete calendar of biotech webcasts and catalysts with links to all my notes.
- Find more RNAi, company, pipeline info on the ALNY stock research page.
- See my complete notes below-
- TTR example of non-human primate study (ug/mL dose range) as evidence of advances in delivery
- 4 clinical stage programs (PCS, TTR, RSV, VSP)
- PCS phase 1 will start very shortly (note - it has been initiated as of 9/26/11) - placebo-controlled, randomized, double-blind, will present plasma PCS levels and LDL data by ye2011
- TTR02 CTA (European IND equivalent) expected 2h2011, data sometime next year
- preclinial data: see improvement in potency at 1/10th dose vs TTR01
- PCS - focus on paitents resistant or intolerant to statin use, at very high risk of coronary artery disease (CAD), generally condition has genetic underpinnings
- PCSK9 is "one of most exciting targets in molecular medicine right now"
- In preclinical studies, saw no decrease in HDL levels with ALN-PCS, unlike mAbs in development
- newest program - hemophilia. "Inhibitor patients" must use bypass agents (such as Novoseven or plasma concentrate). This RNAi drug targets protein C
- refractory anemia - target is hepcidin. Gain of function mutations in human causes anemia. Loss of function mutation results in hemochromotosis. RNAi offers unique opportunity to treat refractory anemia
- 5x15 strategy- seek drug development paths that are as expedited as possible
- RSV phase 2b trial ongoing
- VSP phase 1 data being positoned for partnering before phase 2
- HTT - goal is disease modfiying therapy for this indication (Huntington's disease)
- other applications of RNAi-
- Regulus - focused on microRNA. Programs for hypercholesterolemoa, cancer, HCV
- Biologics manufacturing - siRNA delivered to CHO cell in fermentation system to improve atrributes or quality of biologics. We have formed partnerships already and expect more going forward.
- November 2011 TTR data
- PCSK9 data by year end 2011
- TTR02 IND 2h2011, data 2012
- RSV01 data 2012
- partnering liver cancer program before phase 2, hope to start 2012
- year end 2011 cash >$250m
- q&a in breakout session
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