Alnylam $ALNY President and COO Barry Greene appeared on a Burrill Report podcast following the announcement of phase 1 ALN-TTR01 data demonstrating RNAi proof of concept in humans.
Visit the ALNY research page for additional company, partnership, and drug candidate info.
Keep reading below for my notes from this podcast. ALNY also held a conference call on 11/21 and I will post notes separately from that event.
Visit the ALNY research page for additional company, partnership, and drug candidate info.
Keep reading below for my notes from this podcast. ALNY also held a conference call on 11/21 and I will post notes separately from that event.
Why should people take notice?
Historic day for RNAi. First systemically delivered therapeutic RNAi being used to successfully silence a disease-causing gene in humans (here measured reduced TTR levels in serum which causes ATTR disease).
Transthyretin amyloidosis is an orphan disease - 50,000 patients worldwide, fatal in 5-15 years. No effective treatments yet, liver transplant used for a small subset of these patients, and a new drug from Pfizer $PFE just being approved in Europe. Other approaches are certainly needed for these patients.
Our drug went into the body, found its way to the liver, found its way to the right tissues in the liver, went into the cells making TTR, and silenced TTR protein production by interfering with messenger RNA (mRNA)
Technical hurdle is delivery - getting the siRNA into cytoplasm (where RNAi machinery is located) of rights cells. Here the siRNA is encapsulated in a liposomal nanoparticle and given by injection.
Big Pharma excitement and deals boomed then faded - what was thinking in transition to product company?
Company was founded to translate RNAi and make drugs to treat important diseases and impact patients. RNAi has transformed biomedical research, is still a game changer. Much more focused on particular products now vs a platform for future products. There are still many people investing in RNAi - NVS is still a partner and actively working to bring RNAi into clinic, hands on relationship ended after 5 years. Roche - significant restructuring after Genentech acquisition, including RNAi. Pharma in 1980s walked away from monoclonal antibodies, declaring it was too hard and wouldn't work. I think you'll see them come back to RNAi once we show more and more clinical data.
ALNY is strategically focused on genetic diseases where there is target that clearly causes the disease, and target is accessible because it is made in liver. This is our sweet spot for the next few years - we will expand beyond that, but if that is all we could do, that would make a great company.
5x15 plan (see much more detailed description of plan, criteria, and programs on ALNY research page) launched in early 2011 because we believed delivery was in a state where we could rapidly develop products. TTR is flagship program, PCSK9 for lipid lowering also in clinic, data around year end 2011. Plus anemia and hemophilia programs, and will announce 5th program at year-end.
What will it take for pharma companies and investors to take notice again?
I ask everybody that question...I think data matter, and people will sit up and take notice.
Historic day for RNAi. First systemically delivered therapeutic RNAi being used to successfully silence a disease-causing gene in humans (here measured reduced TTR levels in serum which causes ATTR disease).
Transthyretin amyloidosis is an orphan disease - 50,000 patients worldwide, fatal in 5-15 years. No effective treatments yet, liver transplant used for a small subset of these patients, and a new drug from Pfizer $PFE just being approved in Europe. Other approaches are certainly needed for these patients.
Our drug went into the body, found its way to the liver, found its way to the right tissues in the liver, went into the cells making TTR, and silenced TTR protein production by interfering with messenger RNA (mRNA)
Technical hurdle is delivery - getting the siRNA into cytoplasm (where RNAi machinery is located) of rights cells. Here the siRNA is encapsulated in a liposomal nanoparticle and given by injection.
Big Pharma excitement and deals boomed then faded - what was thinking in transition to product company?
Company was founded to translate RNAi and make drugs to treat important diseases and impact patients. RNAi has transformed biomedical research, is still a game changer. Much more focused on particular products now vs a platform for future products. There are still many people investing in RNAi - NVS is still a partner and actively working to bring RNAi into clinic, hands on relationship ended after 5 years. Roche - significant restructuring after Genentech acquisition, including RNAi. Pharma in 1980s walked away from monoclonal antibodies, declaring it was too hard and wouldn't work. I think you'll see them come back to RNAi once we show more and more clinical data.
ALNY is strategically focused on genetic diseases where there is target that clearly causes the disease, and target is accessible because it is made in liver. This is our sweet spot for the next few years - we will expand beyond that, but if that is all we could do, that would make a great company.
5x15 plan (see much more detailed description of plan, criteria, and programs on ALNY research page) launched in early 2011 because we believed delivery was in a state where we could rapidly develop products. TTR is flagship program, PCSK9 for lipid lowering also in clinic, data around year end 2011. Plus anemia and hemophilia programs, and will announce 5th program at year-end.
What will it take for pharma companies and investors to take notice again?
I ask everybody that question...I think data matter, and people will sit up and take notice.
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