Get more details on ALNY research page.
My quick notes:
- whole new class of medicines. transforming treatment of disease. we can silence any disease causing gene. 9 year old company, now transition from platform to product company.
- newest asset is "5x15 program"
- PCS into phase 1 soon. first w/ 2nd generation LNP. The key gating item is delivery of siRNA into cellular cytoplasm where RNAi machinery works.
- made significant advances for systemic delivery (parenteral or IV injection)
- 1st gen LNP - VSP liver cancer program. 5 patients still on extension pgm
- TTR results will be released 4q2011
- 2nd gen LNP - PCS for hypercholesterolemia. and belive so strongly in TTR program, bringing along 2nd generation molecule here too.
- 2nd generation features increased potency by 10x vs 1st generation. 50% target silencing with 0.1 vs 1 mg/kg dose
- improvement has to do w/ characteristics of liposomal nanoparticle. Broad internal efforts and along with MIT and UBC/Alcana to bring LNP to whole new level, achieved w/ 2nd generation. some formulation change, but key was MC3 lipid discovery
- The manufacturing supply chain, process for drug product (siRNA plus liposome) is rather straightforward. Encapsulate RNA into multi-component capsule (MC3 and other lipid components including for protection). We believe scale-up highly achieveable at pharma like costs, somewhere between small molecule and monoclonal antibody.
- Note the above two bulleted statements contain some degree of intentional marginalization of the contributions of Tekmira TKMR, due to their ongoing legal dispute.
- drug product packaging via liquid in vial, shipped refrigerated. The extract, mix w/ saline, hang in IV bag...typical for IV type drug. We have about 24 month stability right now.
- next big hurdles? this can move very quickly for the 5x15 programs into late stage trials. We need clinical data sets that RNAi works in man (TTR, PCS), and you'll hear about other programs shortly.