|Company Name:||Alnylam Pharmaceuticals, Inc.|
|Event Title:||JMP Securities 2013 Healthcare Conference Transcript|
|Event Time:||04:00 PM ET|
JMP Securities LLC
Our last company is Alnylam Pharmaceuticals, a company that we have know for many years. In fact I was involved few years ago with the IPO before Mike was born. And they've done a fabulous job translating their RNAi platform into a platform of therapeutics that are directed towards orphan disorders where the genetic defect lies in the lever building a robust portfolio of candidates, they have an analyst meeting tomorrow where if you attend you will hear a lot more about at 08:30, we will be there. But because of the proximity of the analyst meeting with today's presentation, Mike is going to speak. We are not going to do Q&A.
He will just give the update and you will have to wait till tomorrow to get your questions answered. So Mike, let me turn the platform over to you.
Vice President Finance and Treasurer
Thanks Mike. Thanks to JMP for having us here today. It has been to our 10-Q for information filed with the SEC. Alynlam is focused on RNA Interference, Nobel Prize winning ground breaking discovery in modern biology. It is a natural process that's involved in control of gene expression. Alynlam's focus has been to harness this natural pathway as a way of turning off disease causing genes in order to advance and develop innovative medicines. And what's exciting is that we can out say that RNA Interference based medicine have been clinically validated.
We've demonstrated this pathway can be harnessed -- Over the last 18 months, we've made significant clinically progress with RNAi. As you can see on the upper left side of the slide, we have a growing safety data base over 575 subjects and patients dosed including over a 100 patients dose systematically. And clinically we've had patients pursuing therapy for over two years chronically. To date, RNAi has been generally well tolerated and we are seeing pharmacologically relevant levels of drug and tissues to get the kind of knockdown in genes we are looking for.
The second point here in the slide that we've established in tissue biopsy samples that we can affect the RNAi mechanism of action in humans. In the lower left hand corner, we've seen tremendous proof of concept, this is with our TTR program with statically significant knockdown in this case of our 90%. And in the bottom right hand side with our PCSK9 program we have seen human clinical efficacy. In this case reduction of LDL cholesterol with doses as low as 0.15 mgs/kg. So these data points give us great confidence as we think about the advancement of RNAi patient going forward.
Vice President Finance and Treasurer
So with this data on hand we continue to advance our strategy to progress our pipeline called on Alnylam 5x15. The key here is five programs in clinical development in 2015 with the near term focus on TTR , amyloidosis, hemophilia and porphyria and these are programs that with or without partners we are going to take forward and I will walk you through these programs in a minute. Now everyone of our 5x15 programs has particularly strategic characteristics. They all target, genetically defined targets for diseases with high unmet medical need.
So if we get the biology right and the target is in fact genetically defined, it gives us the higher probability of clinical success. Secondly, all these programs leverage our existing delivery platform, either IV or subcutaneous delivery. Each of these programs in Phase I provides proof of concept, we can measure the biology that we are trying to achieve very early in clinical development. Each of the programs we believe has clear and rapid path for development and invest the confidence in the target gives us more confidence as we think about spanning for a Phase II or Phase III program.
And we think with this strategy with the data we've had will really help patients in a meaningful way and thus create significant commercial value.
Now our commercialization strategy ultimately is to optimize value for these programs is to go after the high unmet need indications that have concentrated market access and strong patient advocacy. As I mentioned before our strategy for TTR hemophilia and porphyria is to hold on to these drug and commercialize them in North America, South America and the EU and other territories. And as we did with the TTR program, selectively partner in Japan and other regions where we feel a partner has a better opportunity of speeding drugs to these markets.
Now let me start with the most advanced program and that's ATTR , transthyretin-mediated amyloidosis, a significant unmet need and we think a substantial set of product opportunities. ATTR is an orphan disease with approximately 50,000 patients worldwide. It hits patients and the time of life between 40 and 60 years old. And there are two predominant clinical forms of TTR , polyneuropathy with about 10,000 patients and cardiomyopathy with about 40,000 patients.
In addition to being within five years to 15 years these patients unfortunately live with horrific comobities. They have peripheral sensory motor neuropathy autonomic neuropathy and in the case of FAC cardiomyopathy. The current treatments are extremely limited. The most prevalent intervention historically has been liver transplant, liver transplants are used right now in very early stage FAP patients and are actually contra indicated for FAC patients.
There is also a drug called tafamidis or vyndaqel which is approved in the EU, but it is not approved in the U.S. Thus options for these patients are very limited and there is a significant unmet medical need. The target in this program is the transthyretin gene. There are over 100 defined mutations for TTR . And when this protein undergoes mutations, it has the propensity to misfold and deposit as amyloid in the peripheral tissues including the nerves and the heart. And the amyloid deposition results in tissue injury overtime, which is on the clinical manifestations occur. Our approach is to target both the mutant and wild-type forms of transthyretin in order to stop the expression of this misfolded protein and remove its disease causing impact.