Mastocytosis

-- Achieved $128.2 million in AYVAKIT net product revenues in the third quarter 2024 -- -- Raising AYVAKIT net product revenue guidance to $475 million to $480 million for 2024 -- -- On track to initiate the registration-enabling HARBOR Part 2 study of elenestinib in ISM by year end -- CAMBRIDGE, Mass., Oct. 30, 2024 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today reported financial results, provided a business update for the third quarter ended Sept 30, 2024, and provided updated financial guidance. "With another strong quarter of AYVAKIT revenue performance, our year-to-date results provide the foundation to drive significant growth and long-term shareholder value creation in 2025 and beyond. AYVAKIT's sales momentum has given us the confidence to raise our revenue expectations again this year, estimating that we will end the year between $475 million and $480 million dollars. Ending our first full year of launch at nearly half a billion dollars in revenue positions AYVAKIT to be one of the most successful rare disease launches to-date," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "AYVAKIT has demonstrated tangible benefits to patients globally, with long-term data further reinforcing the clinically meaningful benefits and well-tolerated profile of a disease-modifying treatment. AYVAKIT's strong revenue ramp coupled with disciplined investment in our most compelling product opportunities, places Blueprint Medicines in a position to realize the significant decline in cash burn this year, while maintaining our focus on long-term growth and value creation." Third Quarter 2024 Highlights and Recent Progress Mast cell disorders Achieved AYVAKIT net product revenues of $128.2 million for third quarter of 2024, including $113.1 million in the US and $15.1 million ex-US, representing 136 percent growth year-over-year. Advanced the Phase 1 single ascending dose/multiple ascending dose healthy volunteer study of BLU-808, a highly selective and potent investigational oral wild-type KIT inhibitor, with best-in-class potential for chronic urticaria and other mast cell disorders. Data from the healthy volunteer study, including safety, pharmacokinetic and pharmacodynamic markers, are anticipated in early 2025. Presented data at the American College of Allergy, Asthma and Immunology (ACAAI) conference demonstrating the efficacy of AYVAKIT in patients with moderate and severe indolent SM (ISM) at baseline, and showing the significant disease burden of SM, highlighting the urgency to diagnose and treat. Read the presentations here. Hosting scientific seminar on mast cell franchise development with leading allergy and asthma expert Dr. Paul O'Byrne on Thursday, November 14, 2024. 2024 Financial Guidance Blueprint Medicines now anticipates approximately $475 million to $480 million in global AYVAKIT net product revenues in 2024, an increase from the previous range of $435 million to $450 million. This updated guidance is based on continued growth in patients on therapy, continued favorability in compliance and other performance factors, and stronger than expected performance outside of the U.S. The company continues to expect that full-year operating expenses and cash burn will decline in 2024 as compared to 2023, and that its existing cash, cash equivalents and investments, together with anticipated product revenues, will enable the company to maintain a durable capital position to achieve a self-sustainable financial profile. Key Upcoming Milestones The company plans to achieve the following remaining milestones by the end of 2024: Mast cell disorders Initiate registration-enabling Part 2 of the HARBOR trial of elenestinib in ISM by the end of this year. Cell cycle inhibition Continue strategic business development discussions. Complete Phase 1 combination dose escalation for BLU-222 by end of year to inform registration plans. Third Quarter 2024 Results Revenues: Revenues were $128.2 million for the third quarter of 2024 generated from the net product sales of AYVAKIT/AYVAKYT. Revenues were $56.6 million in the third quarter of 2023, including $54.2 million of net product revenues from sales of AYVAKIT/AYVAKYT and $2.4 million in collaboration, license and other revenues. Cost of Sales: Cost of sales was $1.9 million for the third quarter of 2024, as compared to $2.8 million for the third quarter of 2023. The decrease was primarily due to lower sales to our collaboration partner in greater China. R&D Expenses: Research and development expenses were $85.3 million for the third quarter of 2024, as compared to $110.3 million for the third quarter of 2023. This decrease was primarily due to operational efficiency across our portfolio as we execute across our top priority programs and the timing of manufacturing of clinical trial materials. Research and development expenses included $12.6 million in stock-based compensation expenses for the third quarter of 2024. SG&A Expenses: Selling, general and administrative expenses were $89.9 million for the third quarter of 2024, as compared to $70.7 million for the third quarter of 2023. This increase was primarily due to an increase in activities supporting the commercialization of AYVAKIT/AYVAKYT. Selling, general, and administrative expenses included $15.7 million in stock-based compensation expenses for the third quarter of 2024. Net Loss: Net loss was $56.3 million for the third quarter of 2024, as compared to a net loss of $133.7 million for the third quarter of 2023 . Cash Position: As of September 30, 2024, cash, cash equivalents and investments were $882.4 million, as compared to $767.2 million as of December 31, 2023. Blueprint Medicines' cash and investments provide a durable capital position which, together with anticipated product revenues, the company believes will enable it to reach a self-sustainable financial profile . Conference Call Information Blueprint Medicines will host a live conference call and webcast at 8:00 a.m. ET today to discuss third quarter 2024 financial results and recent business activities. The conference call may be accessed by dialing 833-470-1428 (domestic) or 404-975-4839 (international) and referring conference ID 387547. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at . The archived webcast will be available on Blueprint Medicines' website approximately two hours after the conference call and will be available for 30 days following the call. Upcoming Investor Conferences Blueprint Medicines will participate in three upcoming investor conferences: Guggenheim Inaugural Healthcare Innovation Conference on Tuesday, November 12, 2024 at 3:30 p.m. ET. Stifel 2024 Healthcare Conference on Monday, November 18, 2024 at 10:20 a.m. ET. Jefferies London Healthcare Conference on Thursday, November 21, 2024 at 12:00 noon GMT. Scientific Webinar Series Blueprint Medicines will host the second event in its scientific seminar series, focused on mast cell diseases, on Thursday, November 14th at 10:00 a.m. ET. A live webcast of the above presentations and any related slides will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at . A replay of the webcasts will be archived on the Blueprint Medicines website following the events. About Blueprint Medicines Blueprint Medicines is a global, fully integrated biopharmaceutical company that invents life-changing medicines. We seek to alleviate human suffering by solving important medical problems in two core focus areas: allergy/inflammation and oncology/hematology. Our approach begins by targeting the root causes of disease, using deep scientific knowledge in our core focus areas and drug discovery expertise across multiple therapeutic modalities. We have a track record of success with two approved medicines, including AYVAKIT®/AYVAKYT® (avapritinib) which we are bringing to patients with systemic mastocytosis (SM) in the U.S. and Europe. Leveraging our established research, development, and commercial capability and infrastructure, we now aim to significantly scale our impact by advancing a broad pipeline of programs ranging from early science to advanced clinical trials in mast cell diseases including SM and chronic urticaria, breast cancer and other solid tumors. For more information, visit and follow us on X (formerly Twitter; @BlueprintMeds) and LinkedIn. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding plans, strategies, timelines and expectations for Blueprint Medicines' operations, including its expectations regarding growth and net product revenue in 2024; statements regarding its current or future approved drugs and drug candidates and operations, including plans to initiate registration-enabling Part 2 of the HARBOR trial of elenestinib in ISM in 2024, complete Phase 1 combination dose escalation for BLU-222 to inform registration plans and continue strategic business development discussions by the end of 2024, and present healthy volunteer data from its Phase 1 trial for BLU-808 in early 2025; expectations related to the markets for current or future approved drugs and drug candidates, including expectations regarding the size or scale of patient opportunities that future approved drugs and drug candidates could address; the potential benefits of any of its current or future approved drugs or drug candidates in treating patients; statements related to liquidity and capital position, including expectations that its cash, cash equivalents and investments will provide a durable capital position which, together with anticipated product revenues, will enable Blueprint Medicines to reach a self-sustainable financial profile; and its financial performance, strategy, goals and anticipated milestones, business plans and focus, including expectations regarding its revenue ramp and continued decline in operating expenses and cash burn. The words "aim," "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation: the risk that the marketing and sale of AYVAKIT/ AYVAKYT or any future approved drugs may be unsuccessful or less successful than anticipated, or that AYVAKIT/ AYVAKYT may not gain market acceptance by physicians, patients, third-party payors and others in the medical community; the risk that the market opportunities for AYVAKIT/ AYVAKYT or Blueprint Medicines' drug candidates are smaller than we estimate or that any approval we obtain may be based on a narrower definition of the patient population that we anticipate; the risk of delay of any current or planned clinical trials or the development of Blueprint Medicines' current or future drug candidates; risks related to Blueprint Medicines' ability to successfully demonstrate the safety and efficacy of its drug candidates and gain approval of its drug candidates on a timely basis, if at all; preclinical and clinical results for Blueprint Medicines' drug candidates may not support further development of such drug candidates either as monotherapies or in combination with other agents or may impact the anticipated timing of data or regulatory submissions; the timing of the initiation of clinical trials and trial cohorts at clinical trial sites and patient enrollment rates may be delayed or slower than anticipated; actions of regulatory agencies may affect its approved drugs or its current or future drug candidates, including affecting the initiation, timing and progress of clinical trials; risks related to Blueprint Medicines' ability to obtain, maintain and enforce patent and other intellectual property protection for its products and current or future drug candidates it is developing; the success of Blueprint Medicines' current and future collaborations, financing arrangements, partnerships or licensing and other arrangements; risks related to its liquidity and financial position, including the risk that Blueprint Medicines may be unable to generate sufficient future product revenues to achieve and maintain a self-sustainable financial profile; and the accuracy of its estimates of revenues, expenses and capital requirements. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Blueprint Medicines' filings with the Securities and Exchange Commission (SEC), including Blueprint Medicines' most recent Annual Report on Form 10-K, as supplemented by its most recent Quarterly Report on Form 10-Q and any other filings that Blueprint Medicines has made or may make with the SEC in the future. Any forward-looking statements contained in this press release represent Blueprint Medicines' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, Blueprint Medicines explicitly disclaims any obligation to update any forward-looking statements. Trademarks Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation. 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Preclinical data highlight company’s fourth discovery stage program, a novel potent and selective KRAS(ON) inhibitor Updated preclinical data from CGT6297, Cogent’s H1047R mutant-selective PI3Kα inhibitor demonstrates robust inhibition of downstream signaling and efficacy WALTHAM, Mass. and BOULDER, Colo., Oct. 23, 2024 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced the addition of a potent and selective KRAS inhibitor to its pipeline. Preclinical data from this program as well as its newly announced H1047R mutant-selective PI3Kα clinical candidate will be presented in two poster presentations at the 2024 EORTC-NCI-AACR International Symposium on Molecular Targets and Cancer Therapeutics taking place in Barcelona, Spain October 23-25, 2024. “Our Research team continues to make amazing progress, and we are excited to announce our third clinical candidate and fourth preclinical program today,” said Andrew Robbins, Cogent’s President and Chief Executive Officer. “Building upon earlier data presentations and recent advancements in the field, our first poster describes the properties of CGT6297, which we believe has the potential to emerge as a best-in-class H1047R mutant-selective PI3Kα inhibitor. Separately, for the first time, we outline our progress toward developing a potential best-in-class KRAS(ON) inhibitor, which in addition to its mechanistic attributes, has pharmacological properties differentiated from existing compounds in the class. Each of these programs align with our long-term strategy of creating and developing best-in-class molecules with the potential to have a broad impact on patients with genetically defined diseases.” Poster DetailsThe posters can be accessed in the ‘Posters and Publications’ page of Cogent’s website. Title: Identification of a Pan KRAS(On) Inhibitor with Selectivity Over KRAS over H/NRAS and pM Activity Across Prevalent KRAS MutationsSession Date and Time: Wednesday, October 23, 2024 – 12.00 - 19.00 CESTLocation: Exhibition Hall, Centre de convencions internacional Barcelona (CCIB), Barcelona, SpainPoster Number: PB108Abstract Number: 120 Mutations in KRAS are among the most prevalent mutations found in cancer, occurring most often in colorectal cancer, non-small cell lung cancer and pancreatic cancer. The poster presented today describes Cogent’s internally-developed pan KRAS(ON) inhibitor with selectivity over HRAS and NRAS and picomolar (pM) activity across KRAS mutations without the potential liabilities of molecules in the class. Following oral administration, CGT6737 demonstrated robust PK/PD and tumor growth inhibition with 90% PD inhibition in mouse xenograft models. Lead optimization of CGT6737 is ongoing. Title: Preclinical Characterization of a Novel PI3Kα H1047R Mutant Selective Inhibitor Session Date and Time: Wednesday, October 23, 2024 – 12.00 - 19.00 CESTLocation: Exhibition Hall, Centre de convencions internacional Barcelona (CCIB) Barcelona, SpainPoster Number: PB133Abstract Number: 145 Cogent is also developing a potential best-in-class, wild-type-sparing, PI3Kα inhibitor that provides coverage for the H1047R mutation, which affects >55,000 cancer patients each year. The phosphoinositide 3-kinase (PI3K) pathway is a key cell cycle regulating pathway that has an established role in tumor growth and development. The approved agents for these patients often lead to dose limitations, resulting from activity against wild-type PI3Kα. The poster presented today highlights Cogent’s clinical candidate CGT6297, a potent allosteric inhibitor of PI3K, with 25-fold selectivity over PI3Kα WT. CGT6297 has high oral bioavailability and low clearance across species, providing robust inhibition of downstream signaling and efficacy in animal models. Importantly, when compared to a clinically relevant dose of a currently approved therapy in a mouse tumor model, CGT6297 demonstrated superior efficacy with no increase in insulin. IND-enabling studies are expected to be initiated in 2025. Upcoming Investor ConferenceCogent will participate in the following upcoming investor conference: Guggenheim Healthcare Innovation Conference - November 12, 2024 at 10:30 a.m. ET. A live webcast of the fireside discussion will be available in the Investors & Media section of Cogent’s website at investors.cogentbio.com/events. A replay of the event will be archived on Cogent’s website for up to 30 days. About Cogent Biosciences, Inc.  Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases. The most advanced clinical program, bezuclastinib, is a selective tyrosine kinase inhibitor that is designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. KIT D816V is responsible for driving systemic mastocytosis, a serious disease caused by unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancer with strong dependence on oncogenic KIT signaling. In addition to bezuclastinib, the Cogent Research Team is developing a portfolio of novel targeted therapies to help patients fighting serious, genetically driven diseases initially targeting mutations in FGFR2, ErbB2, PI3Kα and KRAS. Cogent Biosciences is based in Waltham, MA and Boulder, CO. Visit our website for more information at www.cogentbio.com. Follow Cogent Biosciences on social media: X (formerly known as Twitter) and LinkedIn. Information that may be important to investors will be routinely posted on our website and X.  Forward Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the potential for CGT6297 to be a best-in-class H1047R mutant-selective PI3Kα inhibitor, the expectation that the company will initiate IND-enabling studies for CGT6297 in 2025, and the potential for the company’s KRAS program to produce a best-in-class KRAS(ON) inhibitor with pharmacological properties differentiated from existing compounds in the class and without the potential liabilities of molecules in the class. The use of words such as, but not limited to, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "will," or "would" and similar words expressions are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our clinical results, the rate of enrollment in our clinical trials and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. We may not actually achieve the forecasts or milestones disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to those set forth under the caption "Risk Factors" in Cogent's most recent Quarterly Report on Form 10-Q filed with the SEC. Any forward-looking statement speaks only as of the date on which it was made. Neither we, nor our affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date hereof. Contact:Christi WaarichSenior Director, Investor Relationschristi.waarich@cogentbio.com617-830-1653

− Type II Variation Submission Based on the Positive HELIOS-B Phase 3 Study in which Vutrisiran Significantly Reduced the Risk of Death and Cardiovascular Events Relative to Placebo – CAMBRIDGE, MA, USA I October 16, 2024 I Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced the submission of a Type II Variation to the European Medicines Agency (EMA) for vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM). Vutrisiran is the generic name for AMVUTTRA ® , which is currently approved in the European Union (EU) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. “Today marks another important milestone in our journey to bring RNAi therapeutics to patients with high unmet need around the world,” said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. “ATTR-CM is a rapidly progressive, debilitating, and life-threatening disease that is an increasingly recognized cause of heart failure. Vutrisiran rapidly knocks down TTR, and in the HELIOS-B study treatment with vutrisiran substantially reduced all-cause mortality and cardiovascular events, underscoring the potential of this therapy for those living with the disease. We look forward to working closely with the EMA with the aim to bring this new treatment option to patients as soon as possible.” The regulatory application is based on positive results from the pivotal HELIOS-B Phase 3, randomized, double-blind, placebo-controlled multicenter global study which met all 10 of its primary and secondary endpoints across both the overall and monotherapy populations, each with statistical significance. The findings demonstrated the effects of vutrisiran on outcomes of mortality and cardiovascular events, as well as functional capacity (6-minute walk test), quality of life (Kansas City Cardiomyopathy Questionnaire), and heart failure symptoms and severity (NYHA class) in patients with ATTR-CM. The safety profile of vutrisiran in HELIOS-B was consistent with the established profile of the drug for hATTR amyloidosis in adult patients with polyneuropathy. In HELIOS-B, rates of adverse events (AEs), serious AEs, severe AEs and AEs leading to study drug discontinuation were similar between the vutrisiran and placebo arms. Detailed results from the HELIOS-B study were published in The New England Journal of Medicine . A supplemental New Drug Application (sNDA) for vutrisiran has been submitted to the U.S. Food and Drug Administration (FDA) for the treatment of ATTR-CM. Additional regulatory submissions are planned globally. AMVUTTRA ® (vutrisiran) INDICATION AND IMPORTANT SAFETY INFORMATION Indication In Europe and the UK, vutrisiran is indicated for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. Important Safety Information Reduced Serum Vitamin A Levels and Recommended Supplementation Vutrisiran treatment leads to a decrease in serum vitamin A levels. Supplementation of approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day is advised for patients taking vutrisiran. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness). Adverse Reactions The most frequently occurring adverse reactions in patients treated with vutrisiran were pain in extremity and arthralgia. Other commonly reported adverse reactions with vutrisiran were dyspnoea, injection site reaction and increase in blood alkaline phosphatase. For additional information about vutrisiran, please see the full Summary of Product Characteristics . About AMVUTTRA ® (vutrisiran) AMVUTTRA ® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of variant and wild‑type transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection, vutrisiran is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. Vutrisiran is also in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), which encompasses both wild-type and hereditary forms of the disease. About ATTR Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000-300,000 people worldwide. 1-4 About RNAi RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. 5 Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. 6 By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors that encode for disease-causing or disease pathway proteins – thus preventing them from being made. 5 This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases. About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “ Alnylam P 5 x25 ” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. 1 Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med . 2015;47(8):625-638. 2 Gertz MA. Am J Manag Care . 2017;23(7):S107-S112. 3 Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst . 2016;21:5-9. 4 Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis . 2013;8:31. 5 Elbashir SM, Harborth J, Lendeckel W, et al. Nature . 2001;411(6836):494-498. 6 Zamore P. Cell . 2006;127(5):1083-1086. SOURCE: Alnylam Pharmaceuticals