Alnylam Pharmaceuticals CEO Discusses Q2 2012 Results - Earnings Call Transcript

Alnylam Pharmaceuticals, Inc. (ALNY)

Q2 2012 Earnings Call

August 6, 2012; 04:30 pm ET


John Maraganore - Chief Executive Officer

Barry Greene - President & Chief Operating Officer

Jared Gollob - Vice President of Clinical Research

Mike Mason - Vice President of Finance & Treasurer

Cynthia Clayton - Vice President of Investor Relations & Corporate Communications


Charles Duncan - JMP Securities

Geoff Meacham - JPMorgan

Marko Kozul - Leerink Swann

Alan Carr - Needham

Edward Tenthoff - Piper Jaffray

George Devoiko - MLB & Company



Good day ladies and gentlemen and thank you for standing by. Welcome to the Alnylam Pharmaceuticals conference call to discuss the second quarter 2012 financial results. There will be a question-and-answer session to follow. Please be advised that this call is being recorded at the company’s request.

I would now like to turn the call over to the company. You may proceed.

Cynthia Clayton

Good afternoon. I’m Cynthia Clayton, Vice President of Investor Relations and Corporate Communications at Alnylam. With me today are John Maraganore, Chief Executive Officer; Barry Greene, President and Chief Operating Officer; Jared Gollob, Vice President of Clinical Research; and Mike Mason, Vice President of Finance and Treasurer. Akshay Vaishnaw, our Chief Medical Officer could not be with us today due to travel plans.

For those of you participating via conference call, the slides we have made available via webcast can also be accessed by going to the Investors page of our website

During today’s call as outlined in slide two, John will provide some introductory remarks and provide general context. Jared will summarize the clinical progress with our Alnylam 5x15 program. Mike will review our financials and guidance, and Barry will provide a brief update on progress with our partner programs and also provide a summary of our business highlights and goals. We will then open the call for your questions.

Before we begin, and as you can see on slide three, I would like to remind you that this call will contain remarks concerning Alnylam’s future expectations, plans and prospects, which constitute forward-looking statements for the purposes of the Safe Harbor provision under the Private Securities Litigation Reform Act of 1995.

Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in our most recent Quarterly Report on file with the SEC. In addition, any forward-looking statements represent our views only as of the date of this recording, and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligations to update such statements.

I will now turn the call over to John.

John Maraganore

Thanks Cynthia. Welcome everyone and thanks for joining us this afternoon. This quarter in recent period defined an exceptional period of scientific and clinical accomplishment for Alnylam and the field of RNAi therapeutics.

Indeed in recent months our clinical results with RNAi therapeutics have grown significantly. We have now demonstrated positive results in five clinical programs, including ALN-TTR01 and ALN-TTR02 for the treatment of ATTR, ALN-RSV for RSV infection, ALN-VSP for liver cancer and ALN-PCS for severe hypercholesterolemia.

Notably we are very pleased with our most recent data with ALN-TTR02, where we achieved rapid, dose dependant, durable and specific knockdown of serum trends by region, the disease causing protein in transthyretin-mediated amyloidosis after just a single dose.

Specifically, we showed TTR knockdown of up to 94% with a nearly 80% level of suppression sustained at one month. We believe these data document an unprecedented level of clinical activity for RNAi therapeutics and serve as an important industry milestone in the advancement of this new class of medicines to patients. We also reported encouraging clinical data from our Phase I ALN-PCS trial showing statistically significant and durable knockdown of plasma PCSK9 of up to 84% and lowering of LDL cholesterol of up to 50%.

Turning to slide five, these important accomplishments gave us great confidence in the continued execution of our Alnylam 5x15 product strategy, which focuses on advancement of RNAi therapeutics toward genetically defined targets for diseases where there are limited treatment options for patients and caregivers. We also believe that Alnylam 5x15 creates an important and compelling path forward through value creation for our shareholders.

As we stated earlier this year, we plan on executing on this strategy with a near term focus on ALN-TTR02 and ANL-TTR sub-cu for TTR amyloidosis and on ALN-AT3 for hemophilia. And that advancement of other 5x15 programs will be accomplished with partnerships that we aimed to form in the future.

We have also made important progress in our delivery efforts. To date our clinical experience has employed three distinct delivery approaches, including inhalation of SRNA in a simple formulation, first generation LMP technology or (inaudible) and Alnylam’s proprietary second generation LMP formulation.

And as we highlighted in an important announcement of data this morning, we are on the eve of adding a fourth delivery technology to our clinical pipeline efforts with our GalNAc SRNA conjugate approach that enables subcutaneous dose administration. We are employing this approach in a number of our Alnylam 5x15 programs and fully expect to employ it increasingly in the future. Jared will talk about that in just a moment.

So without a doubt, our recent clinical activity and delivery progress is a major step forward for RNAi therapeutics and these results have bolded us further in our efforts. Of course, there is much more to do in our efforts to bring RNAi therapeutics to patients in need, and everyone here at Alnylam is focused and passionate on making that happen.

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