Alnylam Pharmaceuticals Pr<∏&♣ovides Perspective on Positive ↑ Complete Results from ORION-11 ♦π Phase 3 Study of Inclisiran
Alnylam Pharmaceuticals, Inc., the l≠eading RNAi therapeutics company, today σ"provided perspective on the pos♠itive complete results from >πthe ORION-11 Phase 3 stud★♥✘y of inclisiran, an RNAi th↔→erapeutic in development for th™∞©∑e treatment of hyperchβ☆♥olesterolemia. These re₹&sults were presented ★<™earlier today by Alnylam’s partner The Medicine¶∑÷s Company and collaborators at ×$✔ the European Society of Cardiology’s ¥α≈ESC Congress 2019 in Paris, φ↕∑France.“The ORION-11 results ↕<represent a landmark event f♠★<£or Alnylam and RNAi therapeutics. With ÷×♥over 1,600 patients enrolled, ORION-11 is the lar ¥gest ever randomized, double©≠-blind, placebo-contro€÷♠lled study of any RNA-based me×€dicine. The results fro✔×m the study demonstrat≥e significant efficacy for a♦£φ drug administered subcutaneously once every si≥↔$↑x months and a very encouragi↕♣ng safety profile, with pr ←±edominantly mild and t÷"ransient injection site✔©• reactions as the only r>γeported drug-related finding,≈★ ” said John Maraganore, Ph.D., Chief Execu♠"tive Officer of Alnylam. “These resu>₽lts further support the↕✘εα safety of our RNAi the≈≤rapeutics platform and provid∑Ω±≠e the largest demonstrat∞£ion to date suggesting that theΩ♣÷♦re is no systematic evidence for a♠★♠© platform-specific safety signal. Consequently,₽≈₽ these results greatly strengt™©♥hen our conviction for the future pote δ≤ntial of RNAi therapeutics in large populat©φ≠"ion diseases. Moreover, the pharmacology oφ♠αf RNAi therapeutics, as infrequently a∞→£dministered medicines, creates what we®<♦ believe to be a very attractive profile for th♠☆e treatment of common diseaβ ses. Finally, assuming a positiv ♦e regulatory review,π® the significant inclisiran royalties ♦×↓€of up to 20 percent wil<δ∞l provide yet another source o☆∞f relatively near-term revenues and wilλ♦'l support Alnylam’s transition toward a self-sσ≠ ustainable financial profile for cont<÷↑inued and future growth.”ORIONπ &-11 now becomes the third positive Phase 3 ∑€study for an RNAi therapeutic and the$✘≠β second in 2019 alone. Assumin≈βg ORION-9 and -10 are similarly posit 'γive and following regulato"≈ry submissions and reviewπ©, Alnylam expects inclis₩"∑iran could reach the market around late 2020.A viλ≥☆×deo of Dr. Maraganore commenting∏↔ on the implications of the O✘∏<$RION-11 results can be viewed on the Company’s®₽ website, www.alnylam.♥βcom.About InclisiranInclisiran, the fiεσrst cholesterol-lowering©↔π therapy in the RNAi☆"∑ therapeutics class, is an investigational ∞↑£therapy in Phase 3 clinical development t$≠εεo evaluate its ability to lo↑wer low-density lipoprotein choles☆"☆≥terol (also known as LDL-£± C) through twice-yearly dos©∏ing. Inclisiran harnesses the bodσ∑$y’s natural process of RNA interference to₹→& specifically prevent production of the PCSK9 ε♦protein in the liver λ<$which enhances the li✔™>ver’s ability to remove LDL-C from th e bloodstream, thereby lo↑ ★wering LDL-C levels. In Phase ÷α♥2 studies, inclisiran provided clinically sign∏">ificant LDL-C reductions greater ↑$∑than 50 percent in addition to the effects of st'ε≥atins and/or ezetimibe, and LDL-C reductionα≤βs were sustained throughout the six-month•× dosing interval. The safety and efΩ✔ficacy of inclisiran were evaluated in ₩≤πthe ORION-11 Phase 3 stu÷£σdy with positive resul•₩✔ts; these results have not been evaluat<∞ed by the FDA, the EMA or any oth∑✔εer health authority. Incl¶↓isiran is not yet approved for u≥π←∞se by the FDA or any other regulatory authority. ¶±↑In February 2013 Alnylam granted global rights "₽↕under a license and collaboration agre∞€×≥ement to The Medicines Comp↕₽≠any to develop, manufa↑€∑♥cture, and commercialize inclisiran.Abou≈t RNAiRNAi (RNA interference↑₽ ≠) is a natural cellular process of gene sil↑&$encing that represents o§ ne of the most promising and rapidly adv ✔→>ancing frontiers in bio"✘logy and drug development today. Its discover®'♠y has been heralded as “a major scientific★₩ breakthrough that happens onc☆☆ >e every decade or so,” and was rec"₩ognized with the award of←≈©Ω the 2006 Nobel Prize fo•✘ r Physiology or Medicine∑≤£♣. By harnessing the natural biological p↕ rocess of RNAi occurring in oλλur cells, a new class of med☆♥σ§icines, known as RNAi therapeutics, is now a reaλ✔lity. Small interfering RNA (siRNA), the molecul>&es that mediate RNAi and cσ&§omprise Alnylam's RNAi th¥₩erapeutic platform, function upstream of todayππ’s medicines by potently silenc✔↕ing messenger RNA (mRNA) – the genetic precuλ© ≈rsors – that encode for diseas©♦↕e-causing proteins, thus preventing ☆↓them from being made. ♥≠ ↑This is a revolutionary approach with ₽≤the potential to transf€§↔↓orm the care of pati×¥ents with genetic and othe∏εr diseases.About Alnylam Pharmaceuφ©ticalsAlnylam is leading φε•the translation of RNA i≈•₹÷nterference (RNAi) into a↔↔ whole new class of innovative medicines →₽¶with the potential to transform ↕↓the lives of people afflicted with ra♠ re genetic, cardio-mπ≈♣δetabolic, hepatic infectious, and centra∑•₽l nervous system (CNS)/oc↑πular diseases. Based on Nob÷✔₹el Prize-winning science, RNAi therapeutics r↕↔σ♥epresent a powerful, clinically vali←βdated approach for the treatment of a wide ♥£range of severe and debilitating d∏β★iseases. Founded in 2" 002, Alnylam is delivering on a bold visio✔>n to turn scientific possibility i€✔÷nto reality, with a robust disco•™™φvery platform. Alnylam’® ♥÷s first commercial RNAi therapeutic is≈$★ ONPATTRO® (patisiran) approved in the ∑φ¥πU.S., EU, Japan and Canada. Alnylam ha±♣☆'s a deep pipeline of investigational mediΩ$cines, including five product candidates th•£at are in late-stage develo←™☆σpment. Looking forward™₹ε, Alnylam will continue ↔<★to execute on its "Alnylam 2020" strat₹≈ egy of building a multi-product,Ω< commercial-stage biopharmaceutical←€ € company with a sustai↓★ ±nable pipeline of RNAi-based medicines to addreαφπss the needs of patients¶☆ who have limited or inadequate treat₩¶απment options. Alnylam employs over 1,200 peop¥±le worldwide and is headquar$₹¶λtered in Cambridge, MA↕ . For more information about our people, scieλ≠↕♠nce and pipeline, please visit www.alny₹ ∏lam.com and engage with us on Twitter at® @Alnylam or on LinkedIn.Alnylam Forward Lo>→©oking StatementsVarious statements inσ§< this release concerning A↕&lnylam's future expect÷β₩ations, plans and prospects, including, with★≠out limitation, the potential implications ↔of the ORION-11 result∏λ>s for patients, Alnylam's views<±₽™ with respect to the safety♥€ of RNAi therapeutics, its views regardi€♥₹¶ng the future potenti₩Ωal of RNAi therapeutics in l<∑φ™arge population diseases, its expectations reg÷♦∑ arding the timing of resu$§lts to be reported by"← The Medicines Company f"∏πrom the ORION-9 and O ☆εRION-10 studies of inclisiran •♥→and the regulatory review of an NDA for incli∏ siran, its expectations regard♦ε±≥ing the receipt of royalties ®♥¶ from The Medicines Company on sales of inc↔↕±δlisiran, if approved by regulatory authorit↓←ies, its plans to achieve a self-sus Ωtainable financial profile for continued and futu↕re growth, and expectations regardingε£σ its “Alnylam 2020” guidance for the advancemπ₹ent and commercialization of RNAi t∞'σherapeutics, constitute forward-looking stat∏≠ements for the purposes of th₽βπ♣e safe harbor provisions under The Pr ε∑∑ivate Securities Litigation Reform A♦♠ct of 1995. Actual results and futu₩±✘re plans may differ materially from those in↑£dicated by these forward-looking statements≥β as a result of various importan≤t risks, uncertainties and other factors, i✘✔βσncluding, without limitation, Alnylam's abi↓$lity to discover and develo♦€∑§p novel drug candidates and π¶←delivery approaches, Alnylam’s andε¥ its partners’ ability to successful≠ ly demonstrate the efficacy and sa∞$<fety of its product candidates, i>↓ncluding inclisiran, the pre-clinical and ↔π¶clinical results for its prod∞÷uct candidates, which may not be re±' plicated or continue to×®≤ occur in other subjects ✘¶or in additional studies or otherwise support fu≈♥✘¥rther development of p← ≠roduct candidates for a specified indicatio&₹n or at all, actions or advice of regulatorβ♣y agencies, which may affect the design, in¥itiation, timing, continu¶εation and/or progress of clinical trials or resu∑♠lt in the need for additional p&¥β"re-clinical and/or clinical •πtesting, delays, interruptions or→™♣× failures in the manufacture and supply o✘☆$©f its product candidat€$✔βes, obtaining, maintaining and protec≠>ting intellectual property, ✘$Alnylam's ability to enforce its intellγ♥™λectual property rights against thiσ£rd parties and defend its patent port € ±folio against challenges from third part"πies, Alnylam’s and its partners’ ability to obt₹δ±Ωain and maintain regulatory approval, €±pricing and reimbursement for products, i ↑λncluding inclisiran, ☆λ≥←progress in establishing a commercial and ex-♥γ✘≈United States infrastructur"£>e, successfully launch♥♦ing, marketing and selling its appro•¥ved products globally, Alnylam’s ability to suc↓"δcessfully expand the indication for O¶×δNPATTRO in the future, comp 'σetition from others using techno∞£logy similar to Alnylam's and o"thers developing products for sim§ σilar uses, Alnylam's ability to manage its £≤¥ growth and operating expenses, obtain additiona$↔™l funding to support its business activities, aλ←→<nd establish and maintain strategic$§✘♦ business alliances π¶↓€and new business initiativ→€βes, Alnylam's dependence on thi₹β•rd parties for development, manufacture and distr£'ibution of products, the outco±↔₩me of litigation, the risk of government₽♣ investigations, and unexpected expenditures, as✘♦£← well as those risks more fully disc ™ussed in the “Risk Fact≤←ors” filed with Alnylam's most recent Quarterly R® eport on Form 10-Q filed with§επ↑ the Securities and Exchange Commissi§δ♦on (SEC) and in other filings that Alnylam mak↑¥₹>es with the SEC. In addition, any <™↓forward-looking statements represent Alnylam's ♥★ views only as of today and should not be re♦∞ ®lied upon as representing its views asλ★© of any subsequent date. Alnyl®₩€am explicitly disclaims an׶y obligation, except to th★→ •e extent required by law, to update any ≤forward-looking statements.the 2019 Asi↕↔×a-pacific pharma IP Leader Summit: http://↕↔♦en.zenseegroup.com/p/404716/ will be held in™₽§ Beijing on∑ November 14-15, and will attract ↑πmore than 500 industry experts from domestγ'♠ic and foreign pharm♣¥∑₹aceutical companies, biotec©±♠∞hnology companies, governments, associations,↓÷• law firms, intellectual property agen≈÷♠¶ts and other companies to attend∑≈ .Official registration and consultati"β↕≠on channels:Contact:AnnPhone: 021-656503>↑<γ05Email:Marketing@zensee•∏group.comhttp://en.zenseegrouπ≠p.com/p/510934/