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Blackstone and Alnylam Close $150 Million R&D Financing to Advance RNAi Therapeutics for Cardiovascular Disease


Blackstone†(NYSE: BX) and†Alnylam Pharmaceuticals, Inc.†(Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the closing of the R&D funding component of the companiesí previously announced $2 billion strategic financing collaboration to accelerate the advancement of RNAi therapeutics.

Under the terms of the agreement, Alnylam will receive up to $150 million from Blackstone Life Sciences for the development of Alnylamís cardiometabolic disease programs vutrisiran and ALN-AGT. The investment includes up to $70 million to support the ongoing HELIOS-B Phase 3 study of vutrisiran in ATTR amyloidosis patients with cardiomyopathy, and up to $80 million to support Phase 2 and Phase 3 development of ALN-AGT, in development for the treatment of hypertension.

In April 2020, Blackstone and Alnylam entered into a broad $2 billion strategic financing collaboration anchored by Blackstoneís purchase of 50 percent of royalties owed to Alnylam on global sales of inclisiran, an investigational RNAi therapeutic for the treatment of hypercholesterolemia, currently under review by the U.S. Food and Drug Administration and other regulatory authorities. The strategic financing collaboration is expected to enable Alnylamís achievement of a self-sustainable financial profile without need for future equity financing, accelerating the commercial potential of Alnylamís rapidly advancing product portfolio.

About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as ďa major scientific breakthrough that happens once every decade or so,Ē and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylamís RNAi therapeutic platform, function upstream of todayís medicines by potently silencing messenger RNA (mRNA) Ė the genetic precursors Ė that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylamís commercial RNAi therapeutic products are ONPATTROģ†(patisiran), approved in the U.S., EU, Canada, Japan, Switzerland and Brazil, and GIVLAARIģ†(givosiran), approved in the U.S., EU and Brazil. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its ďAlnylam 2020Ē strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit††and engage with us on Twitter at†@Alnylam†or on†LinkedIn.

About Blackstone Life Sciences
Blackstone Life Sciences is a private, global investment platform with capabilities to invest across the life-cycle of companies and products within the key life science sectors. By combining scale investments and hands-on operational leadership, Blackstone Life Sciences helps bring to market promising new medicines and medical products that improve patientsí lives.

Alnylam Forward Looking Statements
Various statements in this release concerning Alnylamís expectations, plans and prospects, including, without limitation, expectations regarding the direct or indirect effects on Alnylamís business, activities and prospects as a result of the COVID-19 pandemic, or delays or interruptions resulting therefrom and the success of Alnylamís mitigation efforts, Alnylamís views and plans with respect to the potential for RNAi therapeutics, including vutrisiran and ALN-AGT, expectations regarding its agreement with Blackstone for funding of certain R&D activities for vutrisiran and ALN-AGT, Alnylamís belief that the funding provided by Blackstone should enable Alnylam to achieve a self-sustainable profile without the need for future equity financing, and expectations regarding the achievement of its ďAlnylam 2020Ē strategic plan announced in 2015 for the advancement and commercialization of RNAi therapeutics, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation: the direct or indirect impact of the COVID-19 global pandemic or any future pandemic, such as the scope and duration of the outbreak, government actions and restrictive measures implemented in response, material delays in diagnoses of rare diseases, initiation or continuation of treatment for diseases addressed by Alnylam products, or in patient enrollment in clinical trials, potential supply chain disruptions, and other potential impacts to Alnylamís business, the effectiveness or timeliness of steps taken by Alnylam to mitigate the impact of the pandemic, and Alnylamís ability to execute business continuity plans to address disruptions caused by the COVID-19 or any future pandemic; Alnylamís ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates, including vutrisiran and ALN-AGT; the pre-clinical and clinical results for its product candidates, including vutrisiran and ALN-AGT, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates for a specified indication or at all; actions or advice of regulatory agencies, which may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional pre-clinical and/or clinical testing; delays, interruptions or failures in the manufacture and supply of its product candidates or its marketed products, including ONPATTRO, GIVLAARI, inclisiran, lumasiran and vutrisiran; obtaining, maintaining and protecting intellectual property; intellectual property matters including potential patent litigation relating to its platform, products or product candidates; obtaining regulatory approval for its product candidates, including lumasiran and inclisiran, and maintaining regulatory approval and obtaining pricing and reimbursement for its products, including ONPATTRO and GIVLAARI; progress in continuing to establish a commercial and ex-United States infrastructure; successfully launching, marketing and selling its approved products globally, including ONPATTRO and GIVLAARI and achieving net product revenues for ONPATTRO within its further revised expected range during 2020; Alnylamís ability to successfully expand the indication for ONPATTRO in the future; competition from others using technology similar to Alnylamís and others developing products for similar uses; Alnylamís ability to manage its growth and operating expenses†within the reduced ranges of guidance provided by Alnylam through the†implementation of further discipline in operations to moderate spend and its ability to achieve a self-sustainable financial profile in the future without the need for future equity financing; Alnylamís ability to establish and maintain strategic business alliances and new business initiatives; Alnylamís dependence on third parties, including Regeneron, for development, manufacture and distribution of certain products, including eye and CNS products and ALN-APP, Ironwood, for assistance with the education about and promotion of GIVLAARI, and Vir for the development of ALN-COV and other potential RNAi therapeutics targeting SARS-CoV-2 and host factors for SARS-CoV-2; the outcome of litigation; the risk of government investigations; and unexpected expenditures, as well as those risks more fully discussed in the ďRisk FactorsĒ filed with Alnylamís most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylamís views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

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