Alnylam Pharmaceuticals Provide∞¥s Perspective on Positive×& Complete Results from ORION-11 Phσ ♥ase 3 Study of Inclisiran
Alnylam Pharmaceuticals, Inc., the le•ading RNAi therapeutics company, to§©day provided perspective on the posi←★€ tive complete results from the ORION-11πδ Phase 3 study of inclisiran, an RNAi ↑¶↑therapeutic in development for the tr ±€eatment of hypercholesεφterolemia. These results we§εre presented earlier today bγ∑y Alnylam’s partner The ≤←δ™Medicines Company and collaborators at the E≠α"uropean Society of Cardiology’s ESC C∑±≥ongress 2019 in Paris, France.“The ORI¥↑σ♦ON-11 results represent a landmark δ↓event for Alnylam and RN✘γAi therapeutics. With over 1,600" patients enrolled, ORION-11 is th®♣e largest ever randomized, double-blind→α, placebo-controlled study of any RNA-β εbased medicine. The results from the s ↔≥₩tudy demonstrate significant efficacy for a dru¶≠≠g administered subcutaneously once every six ♣εmonths and a very encouraging safety profi>≤ le, with predominantly₽↓ mild and transient inject<™ion site reactions as the only €≈reported drug-related finding,” said John Mara$↓"ganore, Ph.D., Chief Executive Officer of Alny™π$lam. “These results further support the ™∞©safety of our RNAi therapeutics platform and Ω"↔γprovide the largest demonstration to×σ& date suggesting that there i★$s no systematic evidence for a platform-spe§¥ cific safety signal. Consequentl'↕₩y, these results greatly strengt₽λ∏hen our conviction for the future potential↓•• of RNAi therapeutics in©↔ε large population diseases. Mo✔σreover, the pharmacol≈ ≠ogy of RNAi therapeutics, as infreq≠ε¥uently administered medicines, creates w✘•↔hat we believe to be a very®γ✔ attractive profile f↕♠γor the treatment of comm ≠on diseases. Finally, assuming a posi£∏ ↑tive regulatory review, the significant incli¶☆ siran royalties of up to 20 $≠λpercent will provide yet anoth±± er source of relatively near-λ∑term revenues and will support Alnylam’s trans≈€ition toward a self-su ≠$λstainable financial profile for continσ•ued and future growth.”OR→δβION-11 now becomes the third po♠✘★sitive Phase 3 study for an RNAi therapeutic§→ and the second in 20÷"♥19 alone. Assuming ORION-9 and ←♦¶-10 are similarly positive aσ•&nd following regulatory submissions and←™ > review, Alnylam expects inclisiran could rea<φ≈ch the market around l×¥ate 2020.A video of Dr. Maraganore commenting on✔ the implications of the O≠ΩRION-11 results can be viewed on the Company’s ∏≠×website, www.alnylam.com.About In∞clisiranInclisiran, the first cholesterol-lower® ing therapy in the RNAi therapeutics class, is ∑ ±an investigational therapy®¶ in Phase 3 clinical development to eva¥≈♠☆luate its ability to lower low-density lipop$δ rotein cholesterol (also known as LDL-C) £♠∑through twice-yearly dosing. Inclisiranπ↑ harnesses the body’s¶ natural process of β₹→RNA interference to specificallyφ∞© prevent production of the PCSK9 protein£™ε in the liver which enhances the liver’s abiα•®lity to remove LDL-C from the bloodstream, theΩ∞reby lowering LDL-C levels. In Phas≠♣₽e 2 studies, inclisiran provided clinically s¥↓✘ignificant LDL-C reductions greater than 50 >₹→$percent in addition ₹≠♦to the effects of statins and/≤±±or ezetimibe, and LDL-C reductions we♥ re sustained throughout Ωα✔™the six-month dosing interval. The safety and ef₽↕εficacy of inclisiran were eval≠$&δuated in the ORION-11 Ω©Phase 3 study with positive re₹∑•>sults; these results have not been evaluated by ♥&the FDA, the EMA or any other β'✘health authority. Inclisiran i≠₹≥s not yet approved for β↓₹♣use by the FDA or any oth↕≈ er regulatory authorityσΩ. In February 2013 Alnylam grantedα' global rights under a license and collaboration ×₩δ♥agreement to The Medicines Company ™₽∏ to develop, manufacture, and commerc∞ •ialize inclisiran.About RNAiRNAi (RNA inλ©ε'terference) is a natural celε £★lular process of gene sile π₽ncing that represents one of the↓♣ most promising and rapidly advancing fr ♣ontiers in biology and drug development today. ↑∞∏Its discovery has been heralded as “a ma ★♦ jor scientific breakthrough that happens once →♥every decade or so,” and was recognized wΩ&ith the award of the 2006 Nobel Prize forε• Physiology or Medicin∏✔β e. By harnessing the natural biological processπλ<✔ of RNAi occurring in oπ€∑ur cells, a new class of medicines, kn✘₽ ↔own as RNAi therapeutics, is now a♦♣δ¥ reality. Small interfering RNA (siRNA), the mol"§÷ecules that mediate RNAi and comprise♦π© Alnylam's RNAi therapeutic platform, function up®₩stream of today’s medicγφines by potently silenc≥×ing messenger RNA (mRNA✘δ) – the genetic precursors – that encode for '•disease-causing proteins, thus preventing¶≥ them from being made. This is a revolutionar♣γy approach with the potential to transfo €rm the care of patients with genetic and o♦✘$ther diseases.About Alnylam PharmaceuticalsA£♣§lnylam is leading the translation of RNA inteσ≤≠rference (RNAi) into a whole new class of inααnovative medicines with the→£βΩ potential to transform the lives of ☆•&people afflicted with rar© e genetic, cardio-metabolic, hepatic inf ♦±ectious, and central β ∏₽nervous system (CNS)/ocular diseases. Base€α§≠d on Nobel Prize-winning science, RNAi therapeut♦₽§ics represent a powerful<δ, clinically validated appro•♠♥ach for the treatment of a wide range of se→φvere and debilitating diseases. Founded in 200 ↔2, Alnylam is delivering on a bold₩λ vision to turn scientific poΩ ssibility into reality, wi≤ th a robust discovery platform. Alnylam’sλ≥ first commercial RNAi therapeuti♥↕ε™c is ONPATTRO® (patisiran) approved →↑in the U.S., EU, Japan and Canad≠σa. Alnylam has a deep pipeline of invα↔estigational medicines, including fδ£ive product candidates that are in late-stage de✘"±§velopment. Looking forward, Alny" σlam will continue to execute on i∏★ts "Alnylam 2020" strategy of ®•↔building a multi-product, commercial-stagγ₹×e biopharmaceutical company with a ←∏sustainable pipeline of RNAi-based medicine<"s to address the needs of patients who have↕₹φΩ limited or inadequate treatment opti ons. Alnylam employs over 1,200♣™ people worldwide and is headquartered in C∑↑ambridge, MA. For more in §formation about our people, sα§cience and pipeline, please↑ ≤£ visit www.alnylam.com and engage wiλ th us on Twitter at @Alnylam or on Linke$dIn.Alnylam Forward Lo≠∞&oking StatementsVarious statement₩s in this release concerning Alny¶✘lam's future expectations, plans and pros↓₹↑↕pects, including, without limitation, th←∑>e potential implications of the ORION-11×β results for patients, Alnyla÷£m's views with respect to the safety of RNAi tα↔herapeutics, its views regarding the future σ potential of RNAi therapeutics in large popu• ¥≠lation diseases, its expectations regarding §π<the timing of results to be reported α≠♣by The Medicines Company from the&&←" ORION-9 and ORION-10 studies of inclisirαλ<≈an and the regulatory re←←×✔view of an NDA for inclisiran, its expecta₹π€≥tions regarding the receipt of royalti ε±es from The Medicines Company on sales of¶γγ inclisiran, if approved by regulatory authorit ies, its plans to achieve a self-sustainabl ≥∑×e financial profile for conti¶ nued and future growth, and expe↕♣ctations regarding its “Alnylam 2020”↑≥≠ guidance for the advancement a✔♠nd commercialization of RNAi therape♦Ωε₩utics, constitute forward-looking statemen∏ ↔♣ts for the purposes of the safe harbor provisioεns under The Private S®<ecurities Litigation Reform Act of 19∏®95. Actual results and fut•∏ure plans may differ materiallyΩ♠☆ from those indicated≥∑← by these forward-looking statements₽≥φ♠ as a result of vari↔₹£εous important risks, uncertainti¶φes and other factors, includin₩✘↓g, without limitation, Alnylam's abilit•y to discover and devβ>Ωelop novel drug candidates and deliv ÷δery approaches, Alnylam’s and its part¶↓ners’ ability to succe÷πssfully demonstrate the ef™ελficacy and safety of i®§↔♣ts product candidates, including inclisiran, th πe pre-clinical and clinical results f♠σor its product candidates, which may not be r∑♠€eplicated or continue to occur in other subjec€£ts or in additional studies or otherwise s₽¶±♣upport further development of product ca∏☆♣•ndidates for a specified indicat≤≤Ωion or at all, actions or advice of reguπ£"latory agencies, which may affect the desi↓♣gn, initiation, timing, continuation •±and/or progress of clinical trials or result in¥δ₩Ω the need for additional pre-clinica•∞✘l and/or clinical te♥↑₽sting, delays, interr±≠uptions or failures in th≥♦↕'e manufacture and suppl÷₹↓y of its product candidates, obtaining, m♦®aintaining and protecting intellectual prope ↔♥®rty, Alnylam's ability to enforce i≠ts intellectual prope$&∞rty rights against third parties anδ♣γ&d defend its patent portfolio against challeαλ₽ nges from third parties, Alnylam’s and it✘¶✔₩s partners’ ability to obtain and maintain reγΩ₹×gulatory approval, pricing andγ≤↑ reimbursement for products, including in≈★✔↔clisiran, progress in establishing a commercial a®nd ex-United States infrastructure, successfully ≠✘∑✘launching, marketing and selling its approve£€d products globally, Alny× π↔lam’s ability to successfully expand t∏'he indication for ONPATTRO i ''n the future, competition from othersπ& • using technology similar to Alnylam's aδφnd others developing products for similar us≈✔÷es, Alnylam's ability to manage its growth and★ ÷ operating expenses, obtain additional fun$© ding to support its bu← φsiness activities, and establish ₽π♦•and maintain strategic business alliances and new§§ business initiatives, Alnylam's d↕£♦ependence on third part® εies for development, manufacture and di←$stribution of products, thαπ¶e outcome of litigation, the risk o♠☆f government investigations, an♥↕d unexpected expenditures, as well as those ri✔sks more fully discussed in the “Risk Facto♥☆rs” filed with Alnylam's most recent Qua®☆♣ rterly Report on Form 10-Q filed w"π↓πith the Securities and Exchange Coδ₩♦mmission (SEC) and in other filings th✘δ♥at Alnylam makes with the SEC. In addition, a±∑ny forward-looking statements represent Alny÷Ωlam's views only as of today and should not be re₩≤×lied upon as representing its views as →♣ of any subsequent date. Alnylam explicitly d÷¶ isclaims any obligation∏✘λ, except to the extent required >♠♦by law, to update any forwa≥↔↔rd-looking statements.the 2019 Asia♠↔•-pacific pharma IP Lea$↕der Summit: http://en.zenseegroup.c¥λ♠✘om/p/404716/ will be held § ×in Beijing o∑'∑>n November 14-15, and wil÷¥l attract more than 500 industry experts♠ from domestic and foreign pharmaceutic♦©Ω al companies, biotechnology companies, governme♦'λnts, associations, law ←Ωfirms, intellectual property agen ₩ts and other companies to attend.Official regis✔®tration and consultatio↔φ€ n channels:Contact:AnnPφ↓→δhone: 021-65650305Email:Marketing@zen§∏seegroup.comhttp://en ©≠.zenseegroup.com/p/510934/