Alnylam Pharmaceuticals Provides Perspe÷©♦ctive on Positive Comp∏↔♥lete Results from ORION-11 Phase 3 Study oπ'£f Inclisiran
Alnylam Pharmaceuticals, Inc>✘., the leading RNAi therapeutics company, today♠¥'₹ provided perspectiv₽§&&e on the positive complete results from the ελORION-11 Phase 3 stud↓£y of inclisiran, an RNAi→☆'< therapeutic in development for the tre♣✘atment of hypercholesterolemia≤ ☆. These results were presented earl<→ier today by Alnylam’¶≤♥γs partner The Medicines Company and col$∏"Ωlaborators at the European Society of→ε<$ Cardiology’s ESC Congress 2019 ¥£≈÷in Paris, France.“The ORION↑π-11 results represent a landmark event for Al♠₩nylam and RNAi thera$♦peutics. With over 1,600 ₽ patients enrolled, ORION-11 i✘>λs the largest ever randomized, double-bl×✘ αind, placebo-control↕÷led study of any RNA-based medicine. The€© results from the study demons×$™×trate significant efficacy for a dru$λσg administered subcutaneously once every∏® six months and a ver∑ ✔♠y encouraging safety profile, w♣' ith predominantly mild and tran←Ω☆sient injection site reaction>s as the only reported drug-related finding,” ε∏said John Maraganore, Ph.D∏↓., Chief Executive Of÷×§ficer of Alnylam. “These results furthe™>÷r support the safety of →≈ ✔our RNAi therapeuticsε×≈✘ platform and provide the larg≥φest demonstration to datπ↑↑e suggesting that there is no systematα✘∞εic evidence for a platform-specific safety signa♠¶l. Consequently, these results greatly strengt☆'↓₹hen our conviction for≥×≥§ the future potential of¶€ RNAi therapeutics in large population diseases≈<. Moreover, the pharmacology σ≥¶of RNAi therapeutics, as infrequently administe↕®™red medicines, creates wh €&≈at we believe to be a very ε≠ attractive profile for the treatment of common © diseases. Finally, assuming a positiveγ₹± regulatory review, × the significant inclisiran royalties of up to<₩☆ 20 percent will provide yet another sourc₩ε₩e of relatively near-term revenues and wiφ¶↔™ll support Alnylam’s transition tβ∞oward a self-sustainable financ↑×ial profile for continued and futu✔↕re growth.”ORION-11 now₹♠≠≥ becomes the third positive Phase≥×♦' 3 study for an RNAi the☆₹βαrapeutic and the second in 2019 alone∑ γ. Assuming ORION-9 and -10 are similarly¶₩♠ positive and following regulato☆®©ry submissions and review, A€&£lnylam expects inclisiran could reach $☆the market around late 2020.A video of D≠ ←βr. Maraganore commenti↔∏₹ng on the implications of the ORION-↑ <£11 results can be view®''♣ed on the Company’s w≈>αebsite, www.alnylam.com.About ≈♥↑≥InclisiranInclisiran ∑, the first cholesterol-lowering therapy↑↕→≥ in the RNAi therapeutics class, is an inγαδ¥vestigational therapy in Ph×σase 3 clinical development to evaluat≤ •e its ability to lower low'€-density lipoprotein cholester₽★ol (also known as LDL-C)<ππ through twice-yearly dosing. I÷₹nclisiran harnesses the body’s na€↔δtural process of RNA interfere♦↔£≥nce to specifically prevent production of th₹α♦₹e PCSK9 protein in the liver whic∑£×h enhances the liver’s abilit←©β≥y to remove LDL-C from the bloodst₽¥ •ream, thereby lowering≤×↕™ LDL-C levels. In Phase 2 studα✔® ies, inclisiran provided cπ₽linically significant LDL-C reductions greater ★<than 50 percent in additi<φ¶on to the effects of stati →®ns and/or ezetimibe, and LDL-C re →™✔ductions were sustai±<ned throughout the six-month dosing i₽β₽Ωnterval. The safety and efficac>σ ±y of inclisiran were eval©φ↔uated in the ORION-11 Phase 3 st✔÷©εudy with positive results; these result<α↕s have not been evaluated by the FDA>÷&→, the EMA or any other heal™δ₩th authority. Inclisiran is not yet approved ¶λ✔<for use by the FDA or any other regul≥atory authority. In Februβ∞§®ary 2013 Alnylam granted global rights '♠φunder a license and collaboration agreem≈☆ent to The Medicines Comp¶£Ω any to develop, manufacture, and com>"™mercialize inclisiran.About R✘↑NAiRNAi (RNA interference) is a natural cellul•₩¥ ar process of gene silencing Ω≈←♣that represents one o∑¶β↔f the most promising and rapidly advancing fro"₽Ω↔ntiers in biology and¶↕≠ drug development to€δ×"day. Its discovery h↑§βas been heralded as “a major scientific breakΩthrough that happens once e≈δvery decade or so,” and was recogn₩↑± ized with the award of the 2006 Nobel♣≈¥ Prize for Physiology or Medicine. By har↓±nessing the natural biological process of RN ←γAi occurring in our cells, a new cla©≤§ss of medicines, known as RNAi therapeutics,®♦ is now a reality. Small interfe§₽ ∏ring RNA (siRNA), the molecules that m✘≥ediate RNAi and comprise Alnylam's RNAi therap"♦eutic platform, function upstream ←÷¶ of today’s medicines by potently silencing messe★&nger RNA (mRNA) – the genetic precursors – that$® encode for disease-causing ∑£♣proteins, thus prevent∑→±ing them from being made. This is a revolutio∞£nary approach with the potential to tran¶≠↔πsform the care of pati←₽↑ents with genetic and other diseases.About Alγ ₽nylam PharmaceuticalsAlnylam$>← is leading the translat ©ion of RNA interference (RNAi) into a whole new✔'λδ class of innovative medicin←₹•es with the potential to transform the lives ∏≤<of people afflicted with rare genetic, cardi§γ™→o-metabolic, hepatic infectious, and central ¶ nervous system (CNS)/ocular diseas≈ε≤±es. Based on Nobel Prize-winning science, RNAi tγ" herapeutics represent a powerfu↔£ Ωl, clinically validated approa∑φch for the treatment of a wide range of sev&ε£§ere and debilitating diseases. Fou☆∞✔nded in 2002, Alnylam is delivering on₩® a bold vision to turn scientific possibilityδ✘>φ into reality, with a robust disc×♣≠£overy platform. Alnylam’s first commercial RNA≠↑$i therapeutic is ONPATTRO® (patisiran) app≠♦roved in the U.S., EU, J≠±apan and Canada. Alnylam has a de↔≈ep pipeline of investigati♥™&onal medicines, including five pro↔λduct candidates that are in late-stage developm €₩ent. Looking forward, Alnylam will cont'≥inue to execute on its "Alnylam 2 020" strategy of building a multi-p→Ωroduct, commercial-stage biopharmaceutica₩→₩l company with a sustainable ♣δpipeline of RNAi-basedεε♦ medicines to address the needs of patients who ≥πβhave limited or inadequate treatment op→£¥★tions. Alnylam employs over 1,200 people★★ worldwide and is headquartered in Camb§εφridge, MA. For more informat≥<ion about our people,↓←β science and pipeline, please×€ε visit www.alnylam.com and engage with us¥ ♣ on Twitter at @Alnylam or on LinkedIn.Alnyla≠m Forward Looking StatementsVarious sta↕Ω¶γtements in this release conc ©αerning Alnylam's future expect↕ ations, plans and prospects,<↓ including, without l >imitation, the potential implications of the ORIO >N-11 results for patientσ♦ ★s, Alnylam's views wi↕λth respect to the safety of RNAi >§ therapeutics, its views regarding the±☆™ future potential of RNAi therapeutics in largeφ₩ € population diseases, its expecαγ♣↕tations regarding the t₽®÷iming of results to be reported b☆y The Medicines Company from the ORIO∏↓N-9 and ORION-10 studies of inclisiran and the re<π₽αgulatory review of an NDA for inclisiran, i><ts expectations regarding the receipt of royaltieφ↔s from The Medicines Company on sales of ∏♦>≠inclisiran, if approved by re≠π™®gulatory authorities, its plans to achieve a self→ -sustainable financial profil γ e for continued and future gro★>wth, and expectations reg'↑×arding its “Alnylam 2020” guidance for the advanε≤cement and commercialization ©€of RNAi therapeutics, ↕ constitute forward-looking statements fo≤φαr the purposes of the safe harbor©±£→ provisions under The Private Securities&εΩ Litigation Reform Act of 1995. Actual result ≈ s and future plans may differ materially✔β from those indicated by th₩•ese forward-looking statements as a result of ♦ various important risks, unc< ♠ertainties and other factors, including, without ≥€limitation, Alnylam's ab↑★πility to discover and develop novel d↓✔rug candidates and delivery approa≠✘ ♣ches, Alnylam’s and its partners’ ability to succδ↕essfully demonstrate the ef÷÷αficacy and safety of its product candidaγ←☆£tes, including inclisira✘≥↑ n, the pre-clinical and clinical results for£φ♣• its product candidates, whi φγφch may not be replicated or εφ"continue to occur in other subjects or¶ in additional studies or otherwise sφ→upport further development of product can¥φ∏didates for a specified indicaβ¶ tion or at all, actions or advi$¥•γce of regulatory agencies, which ma♣✘$♣y affect the design, initiation, timi™φ★₽ng, continuation and/o₩ ♦r progress of clinical trials ε₩£or result in the need for a∏πdditional pre-clinical and/or clini♥δ"cal testing, delays, interruptioγ§♠ns or failures in the manufacture and supp÷αδ±ly of its product candid≥Ω§ates, obtaining, maintaining and protecting i★₩¥§ntellectual property, Alnylam's abili→β¶ty to enforce its int¶©↑¥ellectual property rights against third↓©"> parties and defend its patent portfolio☆↔≈♠ against challenges fro↔γ®m third parties, Alnylam’s and its partners↓'∑’ ability to obtain and $∑←maintain regulatory approval, pricing and rei∞↔πmbursement for products, including inclisira↑ "πn, progress in establishing a commercial and ex≥₽-United States infrastructure, suc↓≈cessfully launching, marketing and selling ₹>its approved products global∞¥≈≥ly, Alnylam’s ability to successfully expa'&×nd the indication for O§$♠↑NPATTRO in the future, competition$✔¶™ from others using technology similar t>☆o Alnylam's and others developing products fα÷ ≈or similar uses, Alnylam's abil©±ity to manage its growth and operating e'×™xpenses, obtain additional funding £≈ to support its business activities, and estab★∏lish and maintain strategic business allia®∑★nces and new business initi←☆↕atives, Alnylam's dependence on →× third parties for development, manufa≈€cture and distribution of products, the out ±come of litigation, ↕γεthe risk of government investiga♠₽∞tions, and unexpected expenditures, as ♣₽≤≥well as those risks m☆¶ore fully discussed in the “Risk Factors” ♦γγσfiled with Alnylam's most recent Quarterly Rep¥♠§₽ort on Form 10-Q filed with the Sec©♥ ≤urities and Exchange Commission (SEC) anγd in other filings that Alnylam makes with the S∏φ≠ EC. In addition, any forward-looking sta≥ 'tements represent Alnylam's views only ←♠as of today and should not be relied upon as repr≤↓∏≠esenting its views as of any sub♥sequent date. Alnylam explici€≤∞tly disclaims any obligation, except to th<λ&§e extent required by law, to update any forwardα₹-looking statements.the 2019 Asia-•±$pacific pharma IP Leader Summit: http://en.z≥δenseegroup.com/p/404716/ will be h¶÷∞eld in Beijing ≤✔§on November 14-15, and will attra♦≈ ↓ct more than 500 industry experts π©∏from domestic and foreign pharmaceutical com φσpanies, biotechnology↓≠•" companies, governments, associations, law firms,' ✔€ intellectual property agents and$φσ§ other companies to attend.Official regist→↕ ↔ration and consultation channe÷×¶σls:Contact:AnnPhone: 021-656→γ€γ50305Email:Marketing@zenseegroup.comhttp://en♥∑λ.zenseegroup.com/p/510934&λ§/