Apr 30, 2019 Press Release for Alnylam

Alnylam Highlights Progress on Global Market Access for ONPATTRO (patisiran)

Apr 30, 2019

− In the United States, Company Completes Tenth Value-Based Agreement with Commercial Payers, and Achieves Confirmed Access to ONPATTRO for Greater than 90 Percent of Covered Lives –

− In Europe, Company Achieves Favorable Health Technology Assessment Ratings for ONPATTRO in Multiple Countries –

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apr. 30, 2019-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that it has made substantial progress in the global commercialization of ONPATTRO for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The Company has now completed definitive value-based agreements (VBAs) with 10 commercial payers in the United States. Furthermore, greater than 90 percent of U.S. lives across commercial, Medicare, Medicaid, and other government payer categories are now confirmed to have access to ONPATTRO if prescribed.

“We’re encouraged by the positive reception for global access to ONPATTRO from many private and public payers. With 10 VBAs completed with commercial payers in the U.S., we’re fulfilling our commitment that ONPATTRO is reimbursed based on its ability to deliver outcomes in the real-world setting comparable to those demonstrated in clinical trials,” said Barry Greene, President of Alnylam. “We have also made great progress advancing pricing and reimbursement procedures with authorities across Europe. In particular, we’re encouraged by the favorable outcomes achieved in health technology assessment proceedings in major EU markets including Germany and France, among others. Overall, our market access progress in the U.S. and EU is aligned with our commitment to bring ONPATTRO to patients around the world.”

Alnylam has made significant progress securing access to ONPATTRO for multiple countries in the EU. The Company remains on track with pricing and reimbursement procedures in nearly all EU markets, with encouraging assessment outcomes and ongoing discussions with authorities. Key updates include:

• Germany: Launched ONPATTRO one month following EC authorization (October 2018). Since then, ONPATTRO has become the only product for the treatment of hATTR amyloidosis to receive a “considerable benefit” rating from the Joint Federal Committee (G-BA).
• France: Recognized ONPATTRO as being the only product for the treatment of hATTR with a significant public health impact as noted by its ASMR III and Intêret Santé Publique (ISP) ratings. ONPATTRO is currently reimbursed through Temporary Authorization for Use.
• Austria: Achieved access as of October 2018.
• Luxembourg: Achieved access as of October 2018.
• Netherlands: Reached a nationwide agreement to reimburse ONPATTRO for all eligible patients through joint negotiations coordinated by Zorgverzekeraars Nederland (ZN), the Dutch Association of Health Insurers.
• Sweden: Published HTA assessment from the Dental and Pharmaceutical Benefits Agency in Sweden (TLV) allowing a subsequent ongoing negotiation with the New Therapy Council (NT- Council) to allow usage of ONPATTRO within the County Councils.

Pricing and reimbursement procedures are progressing in several additional countries, including the United Kingdom, Italy, Spain, Portugal, Ireland and key markets in Central and Eastern Europe where several hATTR endemic regions exist. In addition, authorities have approved access under specific named patient procedures in several countries while reimbursement proceedings move forward.

Alnylam remains committed to making ONPATTRO available and reimbursed worldwide in alignment with its Patient Access Philosophy, which outlines the Company’s commitment to access while delivering value to patients, physicians, and payers.

Physicians and patients in the United States can learn more about Alnylam’s comprehensive patient services by visiting AlnylamAssist.com or calling 1-833-256-2748.

Visit ONPATTRO.com for more information, including full prescribing information.

About ONPATTRO^® (patisiran)
Patisiran, based on Nobel Prize-winning science, is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis. It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. Patisiran blocks the production of TTR in the liver, reducing its accumulation in the body’s tissues in order to halt or slow down the progression of the disease. In August 2018, patisiran received U.S. Food and Drug Administration (FDA) approval for the treatment of the polyneuropathy of hATTR amyloidosis in adults, as well as European Medicines Agency marketing authorization for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy.

Important Safety Information

Infusion-Related Reactions
Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO. In a controlled clinical study, 19 percent of ONPATTRO-treated patients experienced IRRs, compared to 9 percent of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.

To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, paracetamol, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.

Reduced Serum Vitamin A Levels and Recommended Supplementation
ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).

Adverse Reactions
The most common adverse reactions that occurred in patients treated with ONPATTRO were respiratory-tract infection (29 percent) and infusion-related reactions (19 percent).

About LNP Technology
Alnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNAi therapeutic products using LNP technology.

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 “a major scientific breakthrough that happens once every decade or so,” 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 Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – 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 new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of diseases with high unmet need. ONPATTRO^® (patisiran) is the first-ever RNAi therapeutic approved by the U.S. FDA for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults and by the EMA for the treatment of hATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy. Alnylam has a deep pipeline of investigational medicines, including six product candidates in Phase 3 studies. Looking forward, Alnylam will continue to execute on its "Alnylam 2020" 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. Headquartered in Cambridge, MA, Alnylam employs over 1,000 people worldwide. For more information, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.

Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, Alnylam's views with respect to the availability and reimbursement for ONPATTRO in multiple geographies around the world, and expectations regarding its “Alnylam 2020” guidance 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, Alnylam's ability to discover and develop novel drug candidates and delivery approaches, successfully demonstrate the efficacy and safety of its product candidates, the pre-clinical and clinical results for its product candidates, 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, obtaining, maintaining and protecting intellectual property, Alnylam's ability to enforce its intellectual property rights against third parties and defend its patent portfolio against challenges from third parties, obtaining and maintaining regulatory approval, pricing and reimbursement for products, progress in establishing a commercial and ex-United States infrastructure, successfully launching, marketing and selling its approved products globally, Alnylam’s ability to successfully expand the indication for ONPATTRO in the future, competition from others using technology similar to Alnylam's and others developing products for similar uses, Alnylam's ability to manage its growth and operating expenses, obtain additional funding to support its business activities, and establish and maintain strategic business alliances and new business initiatives, Alnylam's dependence on third parties for development, manufacture and distribution of products, the outcome of litigation, the risk of government investigations, and unexpected expenditures, as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam's most recent Annual Report on Form 10-K 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 Alnylam's 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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Source: Alnylam Pharmaceuticals, Inc.

Alnylam Pharmaceuticals, Inc
Christine Regan Lindenboom
(Investors and Media)
617-682-4340

Josh Brodsky
(Investors)
617-551-8276