- Alnylam presented at the Stifel Nicolaus Healthcare conference on 9/8/11 - click here for full biotech presentation schedule and links to my notes.
- Additional details on the company and pipeline can be found on ALNY stock research page.
- Keep reading below for my notes from the webcast and Q&A session:
- Presenter: Barry Greene - President and COO
- Key recent RNAi achievements:
2) human data continues to grow. over 500 patients have now been treated, including 60 pts, 200 doses with systemic formulations. We also now have patients with over 1 year dosing
3) 5' RACE PCR used to show target cleaved - the RNAi process was happening in liver
- Discussed newest (4th) 5x15 target - Protein C (see slide deck for more info, I will post to ALNY research page)
--hemophilia B - loss of factor IX - 9-10k patients in US/EU
--"inhibitor patients" develop antibodies - 7k pts, no real treatnment options and represent an extreme cost to healthcare system - fastest development path
--current product - NovSeven recombinant Factor IV, FEIBA - baxter plasma derived factor VII. short lived, require frequent dosing - 3-4x per day IV
- New delivery technology - 50% silencing preclinical w/ 0.02 mg/kg. Dosing would be iv every 2-4 weeks.
- TTR population - 10k are FAP form out of 50k total
- TTR - liver transplant can improve comorbidity but not necesary longer life span because still producing the TTR protein and getting aggregation
- TTR-02 IND will be filed 2h2011
- Will announce 5th 5x15 target 4q2011
- HTT (Huntingtin collaboration with Medtronic) IND goal 2012
- ALN-VSP: 5 patients still on extension trial, seek partner for phase 2 in 2012
- Company is in discussions on additional partnerships
- How many liver-synthesized gene targets are there? There is a huge set of targets even if we can only deliver RNAi to liver. We are looking at a "couple handfuls" of targets
- Should we be expecting a 3rd/4th generation delivery technology in the next few years? How does this affect how to manage the pipeline if delivery technology is outpacing payload clinical development? Is there regulatory flexibiltiy on switching delivery with bridging study? Improving delivery is a strategic core focus. Lower doses allows moving into new therapeutic areas based on therapeutic index. It wouldn't be prudent to guide that we could avoid any preclinical/clinical steps [This is a key question for ALNY, and has hampered ISIS too when they have ditched one generation of antisense candidates to bring along a new and improved more potent version at the cost of a couple of years in the timeline]
- What is your expectation for reduction in serum TTR levels? 35-50% decline would be most clinically relevant to clear buildup and return to homeostasis. We will learn on TTR-01, and move TTR-02 quickly into the clinic and pick one to advance after that. The siRNA payload is identical in 01 and 02. We want to dose up to 1.0 mg and expand there - would give nice data set of safety and efficacy
- How do you plan to manage the ownership of 5x15 assets? We clearly divide these from other partnered programs. We haven't been explicit on strategy - we think we can let datasets play out before seeking certain kinds of partnerships. TTR we'd seek to sign EU partnership - less attractive to do typical ex-US deal, would want 50/50 global deal. On the other hand, an ex-US deal may be very attractive for PCS. So varies program to program
- What is your appetite long-term for participating in commercialization? We feel the only way to build an incredibly successful biopharmaceutical company is to develop and market your own drug in market. We certainly will take one or more into US market alone [I hate when development stage biotechs feel this way. Haven't they seen enough companies fail at this? At least they have a few years to change their mind]
- PCSK9 RNAi vs competing antibodies in development? We will have data readout ye2011 in people with elevated LDL. Some of the antibodies are lowering HDL along with LDL, could require higher doses. RNAi is pheno-copying human genetics. We don't know if stopping extracellular action of PCSK9 (ie, with monoclonal antibody) gives the same benefit as knockdown.
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