Alnylam Pharmaceuticals Provides Perspec♥₹tive on Positive Complete Results from >&>↔ORION-11 Phase 3 Study of Inclisiran
Alnylam Pharmaceuticals, Inc., the leading RNAi ♥ therapeutics company, today provided $Ω₽✔perspective on the positive complete re✘♠sults from the ORION-11 Phase 3 study ±∑ε¥of inclisiran, an RNAi thε÷↕ erapeutic in development for the treatment of h ×"∑ypercholesterolemia. These results were ∏₹presented earlier today by Alnylam’s ✔'partner The Medicines Company and collaborator←±<s at the European So×<ciety of Cardiology’s ESC Congress 2019 in Pa±$ris, France.“The ORION-11 results repres≠≈ent a landmark event for Alnylam and♣∑↓ RNAi therapeutics. With over 1,600 patients enro'≈✔£lled, ORION-11 is the large₹↕st ever randomized, double-blind, placebπ¶¥o-controlled study of any RNA-bas™÷βed medicine. The results from →£¥the study demonstrate significa'≤≤®nt efficacy for a drug admini↑→♠≥stered subcutaneously onceε≥ every six months and a ve∏φ✔<ry encouraging safety pr✘ ofile, with predominantly mild and§ π transient injection sit♣¥ e reactions as the only reported drug-related ∑•finding,” said John Mγ≠™araganore, Ph.D., Chief Executive Officer of Al♠σ←nylam. “These results further support the saf≤÷₹δety of our RNAi ther↑×apeutics platform and provide the₽§₹ largest demonstration to date suggestin±$g that there is no systematic <↓σevidence for a platform-specific s∏δ$afety signal. Consequen÷≥€®tly, these results greatly strengthen ∏∏our conviction for the fut✔♠ure potential of RNAi therapeutics in lar>$¥ge population diseases. Moreover, the phar≤macology of RNAi therapeutics, as i↔nfrequently administered medicines, creates w✘¥±hat we believe to be a very attractive profile≤≥♥♣ for the treatment of ε→common diseases. Finally, assuming a®™α' positive regulatory review, the significant in×®clisiran royalties of up to©φ 20 percent will provide yet an"±δother source of relatively near- ε¥★term revenues and will support A→©lnylam’s transition toward♥← a self-sustainable financial profile for₽®∑← continued and future growth.”ORION-11 now becom× es the third positive Phase 3 study for an RNA↔§<↓i therapeutic and th& Ωe second in 2019 alone. Assuming ORIOεπ↓N-9 and -10 are similarly positive and fol£✘☆lowing regulatory sub™α∞missions and review, Alnyφσ♥lam expects inclisiran cou∑≠ld reach the market >←± around late 2020.A video of Dr. Maragano₩Ω©'re commenting on the implicati♣ φ©ons of the ORION-11 results caφ₩n be viewed on the Company’s website≠≠≥, www.alnylam.com.About InclisiranInclisira ↑n, the first cholesterol-lowering theε★↕rapy in the RNAi therapeutics class, is an investα ''igational therapy in Phase 3÷φγ clinical development to evaluate its ability ™™↑to lower low-density lipopro→ ←tein cholesterol (also ↔☆ε¶known as LDL-C) through tw$"♦ice-yearly dosing. InΩ¶↔clisiran harnesses th$σ&e body’s natural process of RNA interfe✘∑©≈rence to specifically prevent production of the →§αPCSK9 protein in the l♥<$iver which enhances the liver’s ability to remo®¶ve LDL-C from the bloodstream, thereby loweφ®♥ring LDL-C levels. In Phase 2 studies, inclisiran★ provided clinically significant LDL-C reduction©$✔<s greater than 50 percentγ∞ in addition to the effects of statins and/orΩ¥± ezetimibe, and LDL-C reductions were sustained ↑γthroughout the six-month dosing i αΩnterval. The safety and efficacy of inclisira"÷n were evaluated in the ORION-11 Phase 3 stu♥ βdy with positive results; these results have λ↔¶not been evaluated by the FDΩ$A, the EMA or any other health autho¥✘rity. Inclisiran is not yet approv$λ¥ed for use by the FDA or any other ★β'regulatory authority. In Feσ♥γbruary 2013 Alnylam granted∏→ global rights under a license and collab∏§oration agreement to The ÷×¥Medicines Company to develop, manufa¶≥≤¶cture, and commercialize inclisiran.≈γ→↕About RNAiRNAi (RNA interfer> ∞ ence) is a natural cellular pr₹←ocess of gene silencingα↑✘★ that represents one of the most pr✔omising and rapidly ad£φvancing frontiers in biology and drug developme☆↔₽☆nt today. Its discovery has been heralded ♥☆±as “a major scientific♠↑ breakthrough that happe♥♠ns once every decade or so,” and was recognφ✘ized with the award of the 2£÷006 Nobel Prize for Physiology or Medicine•∑ . By harnessing the natural ↔★×biological process of RNAi occurring in our c♦ε ells, a new class of medicines, known as RNA∏≈♦i therapeutics, is now a reality. Small in®₽"βterfering RNA (siRNA), the molecules that medi₩δate RNAi and comprise Alnylam's RNAi th♦ $erapeutic platform, functio¥↑λn upstream of today’s medicines by potently silen ≥'cing messenger RNA (mRN'↕A) – the genetic precursors – thaβΩt encode for disease-causing proteα"¶§ins, thus preventing them from being §πmade. This is a revolutionary apprδ×♠oach with the potential to tr♠αansform the care of ↕∏patients with genetic and othe≤π¥♠r diseases.About Alnylam Pharmace≤≤ →uticalsAlnylam is leading 'σ the translation of RNA interference (RNA©βi) into a whole new class of innovati×→γve medicines with the poε§tential to transform the lives of <≠π people afflicted with★€ rare genetic, cardio-metabolic, hepatic ♣§≤φinfectious, and central nervous system (CNπ✔✔S)/ocular diseases. B✔δ≠ased on Nobel Prize-w←λinning science, RNAi the£>rapeutics represent a powerful, cliα≥&nically validated apσ✘₩proach for the treatment of a wide range of $♠εsevere and debilitating diseases. Founded in 2002≠★, Alnylam is delivering on a↓± bold vision to turn scientific possibα&ility into reality, with a robust discovery platf≥∞orm. Alnylam’s first commercial RNAi thera©♦ peutic is ONPATTRO® (λΩ patisiran) approved in the U.S., •>EU, Japan and Canada. A®₽lnylam has a deep pipeline of investig↔ ©ational medicines, including five product can¶®÷$didates that are in ≤¥Ω'late-stage developmen♣≤t. Looking forward, Alnylam will continue to exα""βecute on its "Alnylam 2020" strate∞♠gy of building a multi-product, com∏↔♠✔mercial-stage biopharmaceutical com ™©pany with a sustainable pipeline of RNAi-based m÷φ∞♠edicines to address the needs of patients wh♥"♣o have limited or inadeqαφ♣•uate treatment option₹★s. Alnylam employs over 1,200 peopleλ™© worldwide and is headquartered in Cambridge,♥∑∞ MA. For more information about our people, scien₽♥π ce and pipeline, please viλ↓★sit www.alnylam.com and engage with u∑β↕♣s on Twitter at @Alnyl€×®δam or on LinkedIn.Alnylam±↕β Forward Looking StatementsVariou₽£s statements in this release co♥¥≠ncerning Alnylam's future expectations, pγ♣lans and prospects, including, withoγ♦♣ut limitation, the potential implicatio₩₽ns of the ORION-11 results for patients, Alnyl" am's views with respect" ∏₹ to the safety of RNAi therapeutics,"↕ its views regarding the future pote£∏ntial of RNAi therapeutics in l "arge population diseases,★δ< its expectations regardinλ×$g the timing of resul©$ts to be reported by The© Medicines Company from •×≠↓the ORION-9 and ORION-10 studies oε↑f inclisiran and the↓ regulatory review of an NDA for inclisiran, i ™><ts expectations regardin©•&g the receipt of royalties from Th <e Medicines Company on sales of inclisiran, if a©♣®pproved by regulatory authoriti☆₽es, its plans to achievε↓≈e a self-sustainable financial profile for cΩ∞ontinued and future growth, and expectations$ ♥ regarding its “Alnylam 202♥'0” guidance for the ad≠¶vancement and commercializatioδ$n of RNAi therapeutics, ∞✘Ωconstitute forward-looking statements for the p<≤≤urposes of the safe ha✘¶rbor provisions under The Private Securities Litπβigation Reform Act of 1995. Actual resultsφγ™ and future plans may differ ↓♠♠•materially from those indicated by th•↓¥→ese forward-looking ∏₹±♣statements as a result of various importa÷♦λ₹nt risks, uncertainties and o<₩ther factors, includ↓©≈ing, without limitation, Alnylam☆φ's ability to discover↕→★ and develop novel drug candidates and∞♠₩ delivery approaches, Alny$∞lam’s and its partners’↔$§ ability to successfully demonstrate t§•✔←he efficacy and safe®∏ty of its product candidates, ®←✔including inclisiran, the pre-cΩ©linical and clinical resε↑•ults for its product candidates, which ma©♥y not be replicated or continue to occur iλ& n other subjects or in additio≤γnal studies or otherwise supp×¥ort further developme α↔nt of product candidates for a specified iσ ♥ndication or at all, actions or a±≤♦¶dvice of regulatory agencies, £Ω©which may affect the design, initiation, timi₩≤ng, continuation and/or p®λ≥≤rogress of clinical trials or←←÷∑ result in the need for addition★al pre-clinical and/∞£♠↕or clinical testing, delays, interr♣α>uptions or failures in♠α™ the manufacture and supply o∞αf its product candidates, obtaining, main±☆≤taining and protecting intellectua₹αε≈l property, Alnylam'♠"s ability to enforce >∏≈its intellectual property rσφ≠ights against third parties↓★¥ and defend its patent port↕∏folio against challenges £→φfrom third parties, Alnylam’s and§✔ its partners’ abili∑✘ty to obtain and maintain regulatory ap∑"proval, pricing and reimbursement→← for products, including inclisir>an, progress in establishing a com≈ €mercial and ex-United States infra÷↕☆ structure, successfully®↓↑↑ launching, marketing and selling ✔δits approved products globa&α→™lly, Alnylam’s abiliε•∏₽ty to successfully expand the indic≥↕♦ation for ONPATTRO in the future, competσ₽♥ition from others using technology simi±€lar to Alnylam's and others de♦♥♥veloping products fo≈ r similar uses, Alnylam's ability to ≤α₹manage its growth and operating expenses,★γ→ obtain additional funding to support its×™ business activities, and establish and maintainλβ" strategic business alliances ®☆and new business initiativesε≠, Alnylam's dependence on third parties for deve ∞&lopment, manufacture and distrib•÷♥§ution of products, the "★×βoutcome of litigation, ₩"€ the risk of government invest¶♣igations, and unexpected expendit ₽←ures, as well as those risk®∞'s more fully discussed in the “Ri< sk Factors” filed with Alnylam's most receσ∏nt Quarterly Report on Form 10-Q filed with the ÷Securities and Exchange Commission (Ω'SEC) and in other filings that Alnylam makes wi& §th the SEC. In addition, any forward-looking ≠→ statements represent Alnylam's viewsΩ£♣ only as of today and should n✘✘ot be relied upon as repre§£senting its views as of any subsequent daγδte. Alnylam explicitly discα laims any obligation, except to the extent↓↑ required by law, to update any for$₹∞αward-looking statements."©'the 2019 Asia-pacific ©♥pharma IP Leader Summit: http://en.zenseegroup$↓≤.com/p/404716/ will be held in Beijing&β nbsp; on November 14-15, $λ↕and will attract more than 500 industry eφ∞xperts from domestic and foreign pharmaceutical ¥≈¶companies, biotechnology ↓"companies, governments, associations, l₩&₽aw firms, intellectual property agents and other>€✘ companies to attend.Officialσ registration and consultation cha×♣nnels:Contact:AnnPhone: 021-6565♠<≥0305Email:Marketing@zenseegrou↑£±™p.comhttp://en.zenseegroup.com/p/510♠× φ934/