Alnylam Pharmaceuticals Reports Second Quarter 2020 Financial Results and Highlights Recent Period Activity Achieved Second Quarter 2020 ONPATTRO Global Net Product Revenues of $66.5 Million, with More Than 1,050 Patient

Achieved Second Quarter 2020 ONPATTRO Global Net Product Revenues of $66.5 Million, with More Than 1,050 Patients on Commercial Product Worldwide -

Achieved Second Quarter 2020 GIVLAARI Global Net Product Revenues of $11.0 Million with More Than 100 Patients on Commercial Product Worldwide -

- Presented Complete Results from ILLUMINATE-A Phase 3 Study of Lumasiran and Completed Filings of New Drug Application and Marketing Authorisation Application -

Completed $2 Billion Strategic Financing Collaboration with Blackstone, Enabling Achievement of Self-Sustainable Financial Profile without Need for Future Equity Offerings -

- Increased Midpoint of Guidance Range for 2020 ONPATTRO Revenue, Narrowing Range from $270-$300 million to $280-$300 million -

CAMBRIDGE, Mass.--(BUSINESS WIRE)--August 6, 2020--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the second quarter 2020 and reviewed recent business

"In the second quarter of 2020, the world experienced unprecedented challenges as it continued to confront the COVID-19 pandemic. While the pandemic continues in some countries and states, our global commercialization strategy is now enabling

some customer-facing activities to resume in most markets as we enter the third quarter. We are very pleased with our ONPATTRO and GIVLAARI commercial performance in the second quarter, in the face of the ongoing pandemic, and believe it

reflects strong demand for our products as well as our team's unwavering commitment to assure access to these RNAi therapeutics for patients around the world," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "In the quarter, we

also continued advancing our robust late-stage pipeline of investigational RNAi therapeutics, where, notably, we presented full results from the ILLUMINATE-A Phase 3 study and completed regulatory filings for lumasiran, and continued enrollment

in our APOLLO-B and HELIOS-B Phase 3 studies of patisiran and vutrisiran, respectively. Further, a key business highlight in the second quarter was our completion of a landmark strategic financing collaboration with Blackstone, which we believe

will secure our ability to achieve a self-sustainable financial profile without the need for future equity financing. These and other achievements position us to realize our Alnylam 2020 vision of building a multi-product, global

biopharma company with a deep clinical pipeline to fuel continued growth and a robust, organic product engine to drive sustainable innovation and further value creation."

Second Quarter 2020 and Recent Significant Corporate Highlights

Commercial Performance

Achieved global net product revenues for the second quarter of 2020 of $66.5 million.

Attained over 1,050 patients worldwide on commercial ONPATTRO treatment as of June 30, 2020.

Continued strong market access in the U.S., with over 15 value-based agreements (VBAs) to date with commercial payers and confirmed access for over 98% of covered U.S. lives.

Continued progress with market access efforts across the CEMEA region (Canada, Europe, Middle East, and Africa), with recent launches in Spain and Italy.

The Company announces today that it has achieved agreement on pricing and reimbursement in France, completing patient access for ONPATTRO in all major European markets in under 2 years following approval.

Achieved global net product revenues for the second quarter of 2020 of $11.0 million.

Since launch, received over 85 Start Forms in the U.S. and attained over 100 patients globally on commercial GIVLAARI treatment from launch through June 30, 2020.

Received marketing authorization approval for GIVLAARI in Brazil for the treatment of acute hepatic porphyria in adults.

Continued strong progress with market access in the U.S., with seven completed VBAs with commercial payers, and confirmed access for over 75% of covered U.S. lives.

Continued progress with market access efforts across the CEMEA region, with a successful launch in Germany, commencement of cohort ATU supply in France, and named patient sales in other countries.

Received an Improvement of Medical Benefit (ASMR) score of II from Haute Autorit de Sant (HAS) in France, concluding that GIVLAARI offers significant additional therapeutic value. Only two new commercial medicines received a similar

Continued work with physicians in multiple regions to support requests for pre-approval access to GIVLAARI in an Expanded Access Program (EAP) in accordance with local requirements.

Advanced patisiran (the non-proprietary name for ONPATTRO), for the potential treatment of the cardiomyopathy of both hereditary and wild-type ATTR amyloidosis.

Continued enrollment in the APOLLO-B Phase 3 study in ATTR amyloidosis patients with cardiomyopathy.

Presented additional clinical data with patisiran - including 24-month results from the global open-label extension (OLE) study and results from an open-label study in post-orthotopic liver transplant hATTR amyloidosis patients - and

published findings from an evaluation of patisiran with concomitant use of TTR stabilizers.

Presented new 12-month interim data from the ENVISION Phase 3 study of givosiran (the non-proprietary name for GIVLAARI) in acute hepatic porphyria (AHP).

In addition, published pivotal results from the ENVISION Phase 3 study in The New England Journal of Medicine.

Submitted a Marketing Authorization Application (MAA) for givosiran in Switzerland and Israel.

Advanced lumasiran, an investigational RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1).

Completed the rolling submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) and submitted an MAA to the European Medicines Agency (EMA), with both applications now accepted.

The FDA also granted Priority Review for the NDA and set an action date of December 3, 2020 under the Prescription Drug User Fee Act (PDUFA).

The EMA granted an accelerated assessment for the lumasiran MAA.

Received a positive scientific opinion from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) through the Early Access to Medicines Scheme (EAMS).

Presented complete results from the ILLUMINATE-A Phase 3 study.

Continued treating patients in ILLUMINATE-B, a global Phase 3 pediatric study of lumasiran in PH1 patients less than six years of age with preserved renal function, and remains on track to report topline results in mid-2020.

Continued enrollment in the ILLUMINATE-C Phase 3 study of lumasiran for the treatment of advanced PH1 in patients of all ages.

Advanced vutrisiran, a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis.

Continued treating patients in the fully enrolled HELIOS-A Phase 3 study of vutrisiran in hATTR amyloidosis patients with polyneuropathy, and remain on track to report topline results in early 2021.

Received Fast Track Designation from the FDA for the treatment of the polyneuropathy of hATTR amyloidosis in adults.

Continued enrollment in the HELIOS-B Phase 3 study in ATTR amyloidosis patients with cardiomyopathy.

Alnylam's partner, Novartis, continued advancing inclisiran, potentially the first and only siRNA cholesterol-lowering treatment, which is undergoing review for approval in the U.S. and EU.

Published results from the completed ORION Phase 3 pivotal trials in The New England Journal of Medicine, showing durable and potent LDL-C reduction achieved with inclisiran, with a safety profile similar to placebo.

Alnylam's partner, Sanofi, continued enrollment in the ATLAS Phase 3 program for fitusiran in patients with hemophilia A or B with and without inhibitors, with topline results expected in H1 2021.

Presented updated interim results at the World Federation of Hemophilia meeting from a Phase 2 OLE study of fitusiran in patients with hemophilia A and B with and without inhibitors.

Advanced early- and mid-stage RNAi therapeutic pipeline programs.

In collaboration with Regeneron, advanced cemdisiran, an investigational RNAi therapeutic for the treatment of complement-mediated diseases.

Continued enrollment in a Phase 2 clinical trial of cemdisiran monotherapy in patients with IgA nephropathy, with topline results expected in 2021.

Regeneron filed a Clinical Trial Application (CTA) in The Netherlands to initiate a Phase 1 study of cemdisiran in combination with pozelimab, an anti-C5 monoclonal antibody, in normal healthy volunteers and patients with

paroxysmal nocturnal hemoglobinuria (PNH).

Alnylam's partner, Vir Biotechnology, presented positive interim data from the ongoing Phase 2 trial in patients and results from the Phase 1 trial in healthy volunteers of ALN-HBV02 (VIR-2218), an investigational RNAi

therapeutic for the treatment of chronic hepatitis B virus (HBV) infection.

Reported positive initial topline results from the ongoing Phase 1 study of ALN-AGT in hypertension, demonstrating acceptable safety, and clinically significant blood pressure lowering with durability enabling a quarterly or

less frequent dosing regimen. Additional Phase 1 results are expected to be presented at a scientific meeting later in 2020, pending abstract acceptance.

Filed a Clinical Trial Application (CTA) for ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 in development for the treatment of nonalcoholic steatohepatitis (NASH). ALN-HSD is being advanced in collaboration

Selected a Development Candidate (DC), ALN-COV (VIR-2703), for SARS-CoV-2 - the virus that causes COVID-19 - with a plan for accelerated filing of an IND around year-end 2020.

Continued progress advancing investigational RNAi therapeutics for CNS and ocular diseases, including ALN-APP, in development for the treatment of hereditary cerebral amyloid angiopathy (hCAA) and autosomal dominant Alzheimer's

Disease (ADAD), which remains on track for a CTA filing in 2021. The Company announces today that Regeneron has exercised its co-development/co-commercialization option on the ALN-APP program, which Alnylam will lead.

Additional Business Updates

Barry Greene, President of Alnylam, announced his intention to transition from Alnylam to pursue new opportunities at the end of the third quarter. Yvonne Greenstreet, Chief Operating Officer, will assume an expanded role as President and

Chief Operating Officer on October 1, 2020.

Entered into a strategic financing collaboration with Blackstone under which Alnylam will receive up to $2 billion that is expected to enable Alnylam to achieve a self-sustainable financial profile without the need for future equity

Entered into an agreement with Dicerna to develop and commercialize investigational RNAi therapeutics for the treatment of alpha-1 antitrypsin (A1AT) deficiency-associated liver disease, and completed a non-exclusive cross-licensing

agreement with Dicerna regarding the companies' respective intellectual property for Alnylam's lumasiran and Dicerna's nedosiran investigational programs for the treatment of primary hyperoxaluria.

Expanded infectious disease collaboration with Vir to include the development and commercialization of RNAi therapeutics targeting up to three host factor targets for SARS-CoV-2, including angiotensin converting enzyme-2 (ACE2),

transmembrane protease, serine 2 (TMPRSS2), and potentially a third host factor target to emerge from Vir's functional genomics work.

Formed a Distribution Agreement with taiba Middle East to commercialize Alnylam's RNAi therapeutics in the Gulf states.

In mid- and late 2020, Alnylam intends to:

Achieve regulatory approval for ONPATTRO in Israel.

File a new drug application for GIVLAARI in Japan and achieve regulatory approval for GIVLAARI in Canada.

Report topline results from the ILLUMINATE-B Phase 3 study of lumasiran in PH1 patients less than six years of age with preserved renal function.

Present additional clinical results from the ongoing Phase 1 trial of ALN-AGT.

Initiate a Phase 1 trial of ALN-HSD.

Achieve regulatory approvals for lumasiran in U.S. and EU.

Alnylam's partner Novartis expects an FDA action date for inclisiran in late 2020.

Alnylam's partner Regeneron plans to initiate a Phase 1 study of cemdisiran in combination with pozelimab.

Financial Results for the Quarter Ended June 30, 2020

"We are extremely pleased with our financial results for the second quarter, particularly in the face of these challenging circumstances. Our team's ability to swiftly adapt to virtual engagement with stakeholders and to support the

transition of patients, where needed, to alternate sites of care, in addition to strong international results, led to commercial performance for both ONPATTRO and GIVLAARI that exceeded the initial expectations we communicated in May 2020. As a

result, we are further revising our full-year revenue guidance for ONPATTRO, with an increase in the midpoint of our guidance as we narrow the range from $270-$300 million to $280-$300 million," said Jeff Poulton, Chief Financial Officer of

Alnylam. "Outside of our commercial performance, a key business highlight in the second quarter was our achievement of year-over-year improvement in our non-GAAP operating loss versus 2019. Driven by strong top-line year-over-year growth and

disciplined investment in our operations, we continue to believe 2019 represents our peak operating loss year as we begin our journey towards a self-sustainable financial profile. In summary, we believe that we're well positioned to effectively

navigate through the pandemic and continue to deliver on the promise of RNAi therapeutics, bringing transformative therapies to patients with serious diseases around the world."

Financial Highlights

(in thousands, except per share amounts)

Net Product Revenues

Net product revenues were $77.5 million in the second quarter 2020 representing 103% growth from the second quarter 2019 as a result of the addition of new patients on therapy and expansion into new markets for ONPATTRO, as well as the

ongoing U.S. commercial launch and initial European launch of GIVLAARI.

Net Revenues from Collaborations

Net revenues from collaborations were $26.4 million in the second quarter 2020, an increase from $6.5 million in the second quarter 2019, primarily due to increases in revenues recognized from our Regeneron and Vir collaborations.

Research & Development (R&D) and Selling, General & Administrative (SG&A) Expenses

R&D expenses decreased in the second quarter 2020 compared to the same period in 2019 on a GAAP and non-GAAP basis primarily due to nonrecurring expenses in 2019 from license fees related to the execution of our collaboration agreement

with Regeneron, as well as a decrease in expenses associated with material manufactured for clinical trials.