Alnylam Pharmaceutic→♠als Provides Perspective on Positiv∞e Complete Results from ORION-11 P≈✘hase 3 Study of Inclisiran
Alnylam Pharmaceuticals, Inc., the leading RNAi$φ therapeutics company, today provided perspeφλ↕αctive on the positive complete results from the ε¶ORION-11 Phase 3 study of ★↓φ₽inclisiran, an RNAi therapeutic in✘& α development for the treatme♠★™®nt of hypercholesterolemia. These results were★£← presented earlier today by Alnδ♠ ∞ylam’s partner The Medicines Company and collab β™©orators at the European Society of Cardiolog≤↓≠y’s ESC Congress 2019 →®in Paris, France.“The ORION-11 resuφλlts represent a landmark event for Al÷ φnylam and RNAi therapeutβ©÷ics. With over 1,600 patien♥&<ts enrolled, ORION-11 is the largest ever randomiγ§zed, double-blind, placebo-co÷₹×ntrolled study of any RNA-based medicine. The rδesults from the study demonstrate significan♠₹t efficacy for a drug administered subcuta¶γ≠neously once every six months and a very encou↓>raging safety profile, with predominantly m&σ∑ild and transient injection←™ site reactions as the only reα÷ported drug-related finding,” said John Maragano'π¥re, Ph.D., Chief Executive Officer of Alnε★Ω≥ylam. “These results further support the safe↓Ωty of our RNAi therapeutics platfo≠≈rm and provide the largest d★₹→¥emonstration to date suggesting that there is§↕ no systematic evidence for a platform-specifi↕£c safety signal. Consequently, these result↕≤≥λs greatly strengthen our coα≠♥☆nviction for the future potential of R$↑'NAi therapeutics in large population diseas•§es. Moreover, the pharmacology of ♦ΩΩ RNAi therapeutics, as infrequently ad♥✔ministered medicines, cre"→ates what we believe to be a very attractive♠α<♣ profile for the treatment↑₽ ∏ of common diseases. Finally, assuming a posiπΩtive regulatory review, the signif ¥icant inclisiran royalties of up to 20 percent wi≥↔♠ll provide yet another source of relatβ←λively near-term revenues and will sup₹ασport Alnylam’s transition tow≈§• ard a self-sustainable financial profile fo∏→r continued and future growth.”ORION-11 now•& becomes the third positive Phase 3 study>α for an RNAi therapeutic and the second in 2019 alone. Assuming ORION-9 and -10 a≈re similarly positive and following regulator✘ § y submissions and review, Alnylam ♥♦expects inclisiran co ♠uld reach the market around late 2020.A video of↔φ Dr. Maraganore commenting on the ←©¥implications of the ORION-11 "≈©∏results can be viewed on the Company’s web✘δ≈↕site, www.alnylam.com.About InclisiranIncl'↓♣↕isiran, the first ch ♦→×olesterol-lowering therapy in t ©he RNAi therapeutics class, is an in♣↕ε≠vestigational therapy in Phase 3 clin'β₩ical development to evaluate its ¶ ability to lower low-density ¥∏lipoprotein cholesterol (Ω←¥also known as LDL-C) th>←rough twice-yearly dosi₹↑•"ng. Inclisiran harnesses the body’s natural ☆ ™∞process of RNA interference to specif∏ically prevent production of the PCσδ™SK9 protein in the li≈£$©ver which enhances the₹≠ liver’s ability to remove LDL-C f∏<∏rom the bloodstream, t≥✘hereby lowering LDL-C levels. >→ In Phase 2 studies, inclisiran provφ®ided clinically significant LDL-C Ω♦γ≤reductions greater thanδαα≤ 50 percent in addition to the effects of sta"®↑βtins and/or ezetimibe, and LDL-C red☆βδΩuctions were sustained throughout the six-mo↓÷nth dosing interval. The safety and effica∞©cy of inclisiran were evaluated in thλαe ORION-11 Phase 3 study with positiveφ≈× results; these results havσ σe not been evaluated by♦≤↑ the FDA, the EMA or any other health authori☆•δty. Inclisiran is no ↑Ω↔t yet approved for us¶←♠∏e by the FDA or any o≠>✔ther regulatory autho↔₹☆rity. In February 2013 Alnylam granted g♦≠ lobal rights under a license an↓↓©<d collaboration agreement to The Medicin₽ ↑es Company to develop, manufacture, and com→↔mercialize inclisiran.About RNAiRNAi ≤$↑<(RNA interference) is a natural cellul₹×≤₹ar process of gene sile ↓ncing that represents one of the mostπ¥ promising and rapidly advancing ≠<↔frontiers in biology and drug development tod↕₽↕ay. Its discovery has been herald ©®≠ed as “a major scientific breakthrough that happe λδns once every decade or so↓∏$,” and was recognized with'∏₩ the award of the 2006 Nobel→← Prize for Physiology♣• or Medicine. By harnessing the natural biologica♣εl process of RNAi occΩ ®urring in our cells, a n¥←∑ew class of medicines, known as RNAi the'✘₹©rapeutics, is now a reality. Small ™¥©interfering RNA (siRN&÷≈A), the molecules that mediate RN✘<π÷Ai and comprise Alnylam's RNA≥>βi therapeutic platform₹≠, function upstream of todaα∞♠≤y’s medicines by potentlΩ↓∞§y silencing messenger RNA (mRNA) – the♥®Ωδ genetic precursors – that encode for ✔×∑δdisease-causing proteins, ↓¥¥thus preventing them from being€γ✔ made. This is a revolutionary approach with λ÷the potential to transform the careσ<₹ of patients with genetic a↕δnd other diseases.About Alnylam Phar✔¥≈↔maceuticalsAlnylam is leading the tra ♣¥∑nslation of RNA interference (RNAi) into a who∞≠ le new class of innovative medicφβ€₩ines with the potential to ₽"₽transform the lives of pe✘↕✘ople afflicted with rare genetic, cardio-me≤©φtabolic, hepatic infec><tious, and central nervous system (CNS)/ocula♠©r diseases. Based on Nobel Prize-wi∞§nning science, RNAi t♠☆φ←herapeutics represent a powerful, ✔ clinically validated approach for th"××e treatment of a wide range of severe and debi♦®♥™litating diseases. Founded in 2≠λ002, Alnylam is delivering on a bold vis≥ion to turn scientific possibility into reality✔>×♥, with a robust discovery platform. Alnyla♣σ>♦m’s first commercial RNAi therapeutic is ONPATTRγ↔♥₹O® (patisiran) approved in the U.S., EU®≤, Japan and Canada. Alny<↔∏lam has a deep pipeline of invest§®igational medicines, incl¥€γ≤uding five product c≠§andidates that are i♠n late-stage development≈δ™. Looking forward, Alnylam will continue → to execute on its "Alnylam 2020"↑& strategy of building a multi-product, commφπercial-stage biopharmaceutical company wi•→≈♦th a sustainable pipelin£•★©e of RNAi-based medicines to addre↑✔ 'ss the needs of patients who have limite±<d or inadequate treatment options. Al☆♠©nylam employs over 1,200 p ©Ω eople worldwide and is head©♥πquartered in Cambridge, MA. For more information ♥εβ±about our people, science and☆ε pipeline, please visit www.alnylam.com≥$ and engage with us on Twitter at @Al₹α∏nylam or on LinkedIn.Alnylam Forward Loo✘☆♣king StatementsVarious s>✘tatements in this release ∞★concerning Alnylam's future expec♠<tations, plans and prospects, including§, without limitation, the potential i€∑mplications of the ORION-11 results for patieε£✔nts, Alnylam's views with respect to the safet₩™±Ωy of RNAi therapeutic∏¥±s, its views regarding the future potent® ©ial of RNAi therapeutics iπβ₩n large population dise'∞ases, its expectations regarding the ©±$timing of results to be reported by The Med£₽βicines Company from the ORION-9 and↓' ORION-10 studies of inclisiran and the regulatoαλ♥¶ry review of an NDA for incli÷↑←∞siran, its expectations regarding the §≠αreceipt of royalties f✔rom The Medicines Company on sales of inc¶<≤★lisiran, if approved by regula₩αtory authorities, its plans to achieve a se∑φΩlf-sustainable financial profile for con¥☆tinued and future growth, a✘×σεnd expectations regardin ♠&×g its “Alnylam 2020” guidanλ §∞ce for the advancement and commercializatio ♥εn of RNAi therapeuticσ♥>γs, constitute forward-lookin₹β™↑g statements for the purposes of the€ ¥ safe harbor provisions under The Private Securi™₩ties Litigation Reform Act λλ∏of 1995. Actual results and future plans ∞✘may differ materially from those indicat♣φed by these forward-looking statemeλ®nts as a result of various¶↑γ§ important risks, uncert±✔ainties and other factors,®Ω including, without limitation, Alnylam↕•'s ability to discover a→σφnd develop novel drug candidates ∏♦δand delivery approaches, Alny β lam’s and its partners’ ability to successfully d≤₹↓≥emonstrate the efficacy ☆>ε£and safety of its product candid¶σates, including inclisiran, the pre-cliniΩ'¥cal and clinical results for its pro✘↕δ↓duct candidates, which may not be★" replicated or continue to occur in other subj↓↔★ects or in additional st≥β&βudies or otherwise support further devel"'♥opment of product candidates for a specified∏Ω₹↓ indication or at all, actions or advice of r♣↑αegulatory agencies, which may affect the designββ<♦, initiation, timing, continuation and/or progrδ↓↔ess of clinical trials or result in the need§" for additional pre-clinical and/or cl<σ→λinical testing, delays, interru € ptions or failures in the manufactπ ♣≥ure and supply of its prodφ♦uct candidates, obtaining, maintaining a¶ ←nd protecting intellectual property, δεAlnylam's ability to enforc ←☆e its intellectual property rights against t®≈hird parties and defend↕λ₽ its patent portfolio against challenges from₽§↑ third parties, Alnylam’s and itsε↑ partners’ ability to ob↔€tain and maintain regulatory approval>☆∑±, pricing and reimbursement∏↑> for products, including inclisiran, prog≤€ress in establishing a co♦&δmmercial and ex-United States in•frastructure, successfu$♠lly launching, marketing and selling its app±↔§roved products globally, Alnylam’s abilityα"← to successfully expand¶≥£ the indication for ONPATTRO in the future,₽≤ competition from others using technology simila∏•• r to Alnylam's and others developing products f€←επor similar uses, Alnylam's abilit≥y to manage its grow£∏th and operating expenses, obt→™Ω✔ain additional funding to support its b✘✘₩usiness activities, and establish and maintain st←₹♥ rategic business alliances and n± ew business initiatives, Alnylam's dependence® on third parties for development, manufact Ω♦ure and distribution of produc★φ¶ts, the outcome of litigation, the risk of gover÷$nment investigations, and unexpected &±expenditures, as well as t©φγ€hose risks more fully discuss☆☆¥ed in the “Risk Factors” filed wit≤σ" h Alnylam's most recent Quarterl★≥π©y Report on Form 10-Q filed with the Securitie₽↑γs and Exchange CommissΩ★Ωεion (SEC) and in other filings that Alnylam makασ≈es with the SEC. In addition, any fγ↔♦≠orward-looking statements represent Alnσ↓ylam's views only as of tod•←ay and should not be re₩↕ ₩lied upon as representing its∑≥♣" views as of any subsequent date.&→ Alnylam explicitly disclaims ★ any obligation, except to the extent requir®₩ed by law, to update any forward-looking stπ©atements.the 2019 Asia-pacific pharma IP↔$δ✔ Leader Summit: http://en.zenseegro≠₽•σup.com/p/404716/ wα☆ill be held in B¶ eijing on Noveβ₩✘mber 14-15, and will a∑σttract more than 500 industry experts ★÷from domestic and foreign pharmaceutical₹™ companies, biotechnology companies, governmen©÷ts, associations, law firms, intellectual propβ£®∏erty agents and other companies t£εo attend.Official regiδ♠§stration and consultation channeΩεls:Contact:AnnPhone: 021-65650305Email:M®☆↑≤arketing@zenseegroup.comhttp://en.zens☆™<eegroup.com/p/510934/