Alnylam Pharmaceuticals Provides Per♠↑←spective on Positive Complete±"★¶ Results from ORION-11π✘Ω↔ Phase 3 Study of Inclisiran
Alnylam Pharmaceuticals, Inc., the lead↔$ing RNAi therapeutics company, today provided peπ✘rspective on the positive complete results fr•₽α om the ORION-11 Phase 3 study of incli ε™siran, an RNAi therapeutic in d$₩evelopment for the treatment©Ωε of hypercholesterolemia. ↑★ These results were presented earlier to↕Ω day by Alnylam’s partner The Meφβ÷<dicines Company and collabo ♣÷rators at the European Society of Card™βiology’s ESC Congress 201→₹♣9 in Paris, France.“The ORION-11 results repre<↔sent a landmark event for Al >nylam and RNAi therapeutics. With over 1,600≈♣₩₽ patients enrolled, ORION-11 is the φ£<largest ever randomized, double-blind, pl✘←♦acebo-controlled study of any RNA-b♠≈ased medicine. The r≠esults from the study demonstrate sσ" ignificant efficacy for a drug administere×¶§≥d subcutaneously once eβ$γvery six months and a very encouraging £ safety profile, with predominantly mild and tranβ"sient injection site reactions as the only ∞ reported drug-related finding,” said John <↕✘'Maraganore, Ph.D., Chief Executive Office→→>r of Alnylam. “These resultγδ≠₹s further support the safety of our RNAi th φ≈σerapeutics platform and≥Ω provide the largest d♣&✘ emonstration to date suggesting that t↕©♠here is no systematic evidence for a platfor& m-specific safety signal. Consequently, these $♥ results greatly strengthen our conviction ∏✘♣ for the future poten>★↑tial of RNAi therapeutics in l→÷arge population diseases. Moreover, the ¶"§pharmacology of RNAi therapeuti₹±cs, as infrequently administered medicines, creat ↑←es what we believe to be '€©§a very attractive profile for th>↔e treatment of common dis"↑ $eases. Finally, assuming a positiv₹γe regulatory review, the s¶☆ignificant inclisiran ✔ ×royalties of up to 20 percent will provide yet anσ'αother source of relatively near-term revenues∑< and will support Alnyl"←∏am’s transition toward a self-sust$× ainable financial profi→σ★le for continued and future gro↓¶wth.”ORION-11 now becomes t →"he third positive Phaπ£εse 3 study for an RNAi±✘✘↕ therapeutic and the second in 2019 al↔>σone. Assuming ORION-9&€≠ and -10 are similarly positive and following reg•€ulatory submissions and review, Alnylam ₽λ₩expects inclisiran could r♦≤±≥each the market around late 2020.↕®₹"A video of Dr. Maraganore commenting on t γβ♥he implications of the ↔φ ORION-11 results can be view'¥↑ed on the Company’s w↑€ebsite, www.alnylam.com.About Inclisira↔φ↓nInclisiran, the first cholesterol-lowering the≤₹rapy in the RNAi therap✘♣±eutics class, is an in∏♥÷vestigational therapy in Phase₩↓α♣ 3 clinical development to evaπ★luate its ability to lower low-density"₽ lipoprotein cholesterol (al©σγso known as LDL-C) through twice-yearl∑©y dosing. Inclisiran harnesse™πs the body’s natural process÷©¶< of RNA interference to speci•♠♣fically prevent production of the PCSK9 protei∑ ™n in the liver which enhances th<<π∏e liver’s ability to remove LDL-C γαfrom the bloodstream, thereb$¥y lowering LDL-C levels. In Pha∞★™×se 2 studies, inclisiran provided c<®linically significant LDL-C reduct ¶ions greater than 50 percent in addition to t≠£♣he effects of statins and/or ezetimibe, and LD®ε÷L-C reductions were su♠₽€&stained throughout the six-month dosinφ∑™¥g interval. The safety and efficaδ←®cy of inclisiran were evaluated in the ORION-1∞π₹€1 Phase 3 study with positiv±πλ¥e results; these results have notβε±< been evaluated by the FDA, the E✘α©MA or any other health authority. Inclisiran i¥∏s not yet approved for use by the FDA or aφ>→ny other regulatory authority.∑ ↔ In February 2013 Alnylam granted global ↓≥★rights under a license an←₹↑₽d collaboration agreement to The Medi₽cines Company to dev•πelop, manufacture, and commercialize inc<δ∞₩lisiran.About RNAiRNAi (RNA interfere¶♦↔£nce) is a natural cellular process δ$πof gene silencing that represents one of the moλ★>st promising and rapidly advancin♠€☆σg frontiers in biology and drug develop$∑ment today. Its discovery has b∞☆↕een heralded as “a major scientific breakthroug™$h that happens once every decade←β or so,” and was recognized with the award of ©the 2006 Nobel Prize for Physiologφ₹y or Medicine. By harnessing the natural biolog"πical process of RNAi occurε™φring in our cells, a new class of medici♥™nes, known as RNAi therapeutics, is now a reality→★σ . Small interfering RNA (siRNA), t₩Ωhe molecules that mediate RNAi and compr✘∑≥ise Alnylam's RNAi therapeutic platform, funcγ&tion upstream of today’s medicines↑↑®✘ by potently silencing messenger RNA (mσ☆RNA) – the genetic precursors – that eε>ncode for disease-causing protein∑→s, thus preventing them from being made. This ±πis a revolutionary appr∏ ∏♥oach with the potential to transf♣↑©orm the care of patients π♠∏♣with genetic and other di☆↑₩seases.About Alnylam PharmaceuticalsAln™"☆ylam is leading the tra>↔nslation of RNA interferen♥®"€ce (RNAi) into a whole new class of innov÷$™ative medicines with the potential to transform ≠εthe lives of people afflicted ♠with rare genetic, cardio-metabolic, he¶γ®patic infectious, and central nervous syst≤↕em (CNS)/ocular diseases. Based on φ∏Nobel Prize-winning science, RNA¶σσ≤i therapeutics represent a powerful, cβ×₹linically validated ap★✔∏proach for the treatment of a wide ©•λrange of severe and ↔ &€debilitating diseases. Foun¶♠ded in 2002, Alnylam≥≥ is delivering on a bold visi≤≤$λon to turn scientific possibility into reality, λ↓with a robust discovery platform. Aln ♠¶♣ylam’s first commercial π₽≈RNAi therapeutic is ONPATTRO® (patisi•₹∑ran) approved in the U.S., EU, Japa←♦₹n and Canada. Alnylam has a deep pipe∏"line of investigational medicines, i&"→✔ncluding five product candidates that≈< are in late-stage development. Looking forward, Ωπγ®Alnylam will continu£α€☆e to execute on its "Alnylam 2020" ∞₽"strategy of building a multi-product,↑↔∏ commercial-stage biopharmaceutical company wiππ¥th a sustainable pipeline of RNAi-based♥₩↕ medicines to address the need∑≥s of patients who have limited or i ∏÷nadequate treatment option ♠≠s. Alnylam employs over 1,200 people wor÷≥ldwide and is headquartered in Cambridge, MA.₩∏ For more information about our &people, science and pipeline, please visit www.a¥ ₹$lnylam.com and engage with us on Twitte≠π&r at @Alnylam or on LinkedIn.Alnylam Forward L¥ ooking StatementsVari£≥ous statements in this release$↓∏¶ concerning Alnylam's future expe↓♠×ctations, plans and prosλpects, including, without limitatioσn, the potential impl£&✘ications of the ORION≈φ-11 results for patients, Alny>∑✘lam's views with respec©δ>t to the safety of RNAi therapeutics, its views ♦↑>↑regarding the future potential of RNA≠>↕i therapeutics in large population diseases,∞≠λ≈ its expectations regarding t≤∏•"he timing of results to be reported by The M"™✔edicines Company from the ORIO↔₩≠N-9 and ORION-10 studies o∏γ♣f inclisiran and the regula☆λ×tory review of an NDA fo±≈©r inclisiran, its expec€¥ tations regarding the receipt of ↕•>♥royalties from The Medicines Companσ₹y on sales of inclisiran, if ↔• εapproved by regulatory authori<↓₩ties, its plans to achieve a self-sust>↑↑ainable financial profile for continued and<€ future growth, and eε™xpectations regarding its “Alnylam 202↕♦δ0” guidance for the advancement and commerc♣'Ωialization of RNAi therapeuσ☆✘φtics, constitute forward-looking statements for t π£©he purposes of the safe harbor prov¥✔isions under The Private Securities Litigation Rλ>eform Act of 1995. Actual ♣≥results and future plans may differ m©£δaterially from those≥ππ indicated by these forward-looking statements Ω£•£as a result of various imp♥εortant risks, uncertainties and oth↕↑✔φer factors, including, without limitat<↑ion, Alnylam's ability to¥'δ discover and develop novel drug candi♠λdates and delivery approaches, Alnylam’s and itβ ±s partners’ ability to↔← successfully demonstrate the efficacy a&♥nd safety of its product candidates, inclu♠₩ding inclisiran, the pre-clinical and cli₽₹≈nical results for its pro¶≈≤duct candidates, which may nγ™®ot be replicated or conti♣¶≠ nue to occur in other subjects or in additi♣∑¥↑onal studies or otherwis≥§£e support further development of product candi≥₩dates for a specified indication or at a∞∑ll, actions or advice of reg∑₹•ulatory agencies, which may affect the design, i✘λ☆®nitiation, timing, continuation and/or prog≈©↑±ress of clinical trials or result in the≥÷ need for additional pre-clinic ™"Ωal and/or clinical testing, delays, interruptioλ•✘ns or failures in the manufacture and ♣ ∑supply of its product candidateδ♥s, obtaining, maintaining and protecti₹±ng intellectual property, Alnyl✘♣am's ability to enforce its intellectual property&®✘← rights against third parties ®©and defend its patent portfolio against challeng&δes from third parties, Aln&∞ylam’s and its partners’ ability to obtain andΩε maintain regulatory approval, pricing and ₩£reimbursement for pro↑÷λ↕ducts, including inclisiran, progress in est♥←ablishing a commercial and ex-United States ←φinfrastructure, successfully πlaunching, marketing and selling its approved prΩ♣ ₽oducts globally, Alnylam’s ability to succes×®sfully expand the indication for ONPATTR ✘O in the future, competition from $≤others using technology similar to¶↑• Alnylam's and others developing pro∑ ducts for similar uses, Alnylam's ←≥ability to manage it♣₽s growth and operating expenses, o≤ btain additional funding to support it β≤s business activities, and establish and main★∑£tain strategic business alliances aλ↔↓$nd new business initiatives, Alnylam's depeβ↔ndence on third parties fo♥φ∑r development, manufacture and distribu↑"tion of products, the outcome of litigatio♦n, the risk of gover₩÷"nment investigations, and unexp☆ected expenditures, as well as those risks moreγ fully discussed in the “Risk Factors” filed♣→ with Alnylam's most receΩ♦nt Quarterly Report on Form 10-Q fil§Ω ed with the Securities≈← and Exchange Commission (SE≈↓<★C) and in other filings that A€×lnylam makes with the SEC. In addition, any for'εφward-looking statements represen♣↕×∑t Alnylam's views only as of today and should αnot be relied upon as€ representing its views as<₽ of any subsequent date. Alny★γ¶lam explicitly discla♠&ims any obligation, except to th✔≠e extent required by law, to update an×¥y forward-looking state">₹ments.the 2019 Asia-pacific pharma ♣♣IP Leader Summit: http://en.zenseegroup.com/p/4"™04716/ will be held in&n&δ♦♠bsp;Beijing on November↔↑¶→ 14-15, and will attract more than 500 industr φ∑₩y experts from domestΩ<₽¥ic and foreign pharmaceutica ≤ l companies, biotechnology co✘mpanies, governments, associations, law firms, ✔ intellectual property agents and other c¥λ↓ompanies to attend.Official regis★₽≈♥tration and consulta₹≤tion channels:Contact:AnnPhone: 021-65650305Email:Marketing@zenseegroup.comhttp://e≠∏βn.zenseegroup.com/p/5109€≈✔34/