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Alnylam Pharmaceuticals, Inc. (ALNY), the leading RNAi therapeutics company, today announced its new five-year strategy, “Alnylam P⁵x25,” which is focused on the company’s planned transition to a top-5 biotech (measured by market capitalization) in the next five years through: sustainable innovation yielding transformative medicines for rare and common diseases for patients around the world, and delivery of exceptional financial performance. 

Alnylam P⁵x25 extends the Company’s decade-long heritage of providing longer term, five-year business strategy guidance, the most recent of which was known as Alnylam 2020. In addition, Alnylam today reported preliminary fourth quarter and full year 2020 global net product revenues for ONPATTRO and GIVLAARI and provided additional updates on the company’s commercial launches, including initial OXLUMO demand.

New Five-Year Strategy: P⁵x25

Alnylam ended last year exceeding all metrics for its Alnylam 2020 strategy, with 4 marketed products (versus 3), 12 clinical programs (versus 10) -- 6 of which are in late-stage development (versus 4) -- across 4 strategic therapeutic areas (versus 3). The company’s Alnylam P⁵x25 strategy is aimed at Alnylam’s transition to a top 5 biotech company, as measured by market capitalization, over the next five years.

Specifically, the company intends to end 2025 with the following profile**:

• Patients: Over 0.5 million on Alnylam RNAi therapeutics globally.
• Products: 6 or more marketed products in rare and prevalent diseases.
• Pipeline: Over 20 clinical programs, with 10 or more in late stages, and 4 or more INDs per year.
• Performance: ≥40% revenue CAGR through YE 2025.
• Profitability: Achieve sustainable non-GAAP profitability within the period.

“We executed well on our Alnylam 2020 strategy, exceeding all pre-set metrics and transitioning into a global, multi-product commercial company with a robust clinical pipeline and an organic product engine delivering sustainable innovation, a profile that has rarely been achieved in biotech history.

"It was especially gratifying to cap 2020 with positive Phase 3 HELIOS-A results for vutrisiran, which is set to become our 5th RNAi therapeutic to reach the market, if approved,” said John Maraganore, Ph.D., CEO of Alnylam Pharmaceuticals.

“We are now thrilled to launch our new chapter with Alnylam P⁵x25, which is aimed at Alnylam’s planned transition to a top 5 biotech in market capitalization based on a proven and high-yielding technology for disruptive medical innovation and a foundational track record of commercial execution.

"Indeed, with Alnylam P⁵x25, we expect to sustainably and organically create and commercialize transformative rare and common disease medicines, benefiting hundreds of thousands of patients around the world while delivering strong financial performance and profitability, resulting in a leading biotech profile.”

2020 Preliminary Commercial and Financial Performance*

ONPATTRO^® (patisiran), a commercial-stage RNAi therapeutic targeting transthyretin (TTR) for the treatment of polyneuropathy in adult patients with hATTR amyloidosis.

• Preliminary global net product revenues for the fourth quarter and full year 2020 were approximately $90 million and $306 million, respectively.
  • Q4 results represent approximately 10% growth compared to Q3 and include 10% growth in the U.S. market segment driven by new patient demand.
  • Further, the full year ONPATTRO revenues reached the high end of the previously shared guidance range of $295 million - $310 million and represent over 80% growth from full year 2019.
• As of year-end 2020, about 1,350 patients worldwide were receiving commercial ONPATTRO.

GIVLAARI^® (givosiran), a commercial-stage RNAi therapeutic for the treatment of adults with acute hepatic porphyria (AHP).

• Preliminary global net product revenues for the fourth quarter and full year 2020 were approximately $22 million and $55 million, respectively.
  • These results represent greater than 30% quarter-over-quarter growth.
• As of year-end 2020, the product’s first full year of launch, over 200 patients are receiving commercial drug.

OXLUMO™ (lumasiran), a commercial-stage RNAi therapeutic for the treatment of primary hyperoxaluria type 1 to lower urinary oxalate levels in pediatric and adult patients.

• For the period following European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) approval of OXLUMO, in late November 2020, strong initial U.S. demand was observed with 8 Start Forms received through year-end.
• Preliminary global net product revenues for the fourth quarter were approximately $0.3 million, representing initial patient demand in Europe

Finally, the Company today reported that it expects its full year 2020 non-GAAP operating loss to be substantially improved relative to the prior year, marking 2019 as Alnylam’s peak non-GAAP operating loss year as the company transitions towards a self-sustainable financial profile.

Further, at Dec. 31, 2020, Alnylam had preliminary cash, cash equivalents, and marketable securities of approximately $1.9 billion, as compared to $1.5 billion at Dec. 31, 2019. The company balance sheet was strengthened by the 2020 strategic financing collaboration with Blackstone (BK).

“We are extremely pleased with ONPATTRO and GIVLAARI performance in the fourth quarter and cumulatively for the year, reflecting strong commercial execution, a substantial increase in demand and new patient adds, despite the ongoing COVID-19 pandemic.

"Additionally, we’re pleased to report that strong top-line revenue growth at the upper end of our guidance range and disciplined R&D and SG&A investments have delivered on a lower non-GAAP operating loss for 2020 compared with the prior year.

"We believe we’re now firmly on our way toward a self-sustainable financial profile with significant growth from four revenue generating assets and moderated operating expenses, and a balance sheet that supports achievement of profitability without the need to access the equity markets,” said Jeff Poulton, Chief Financial Officer of Alnylam.

“As we now launch our Alnylam P⁵x25 strategy, we’re committed to delivering consistently strong financial performance driven by 40% or greater revenue CAGR over the next five years with non-GAAP profitability achieved within the period.”

Alnylam management will discuss these preliminary selected financial results and commercial updates during a webcast presentation at the 39^th Annual J.P. Morgan (JPM) Healthcare Conference tomorrow, Monday, Jan. 11, 2021, at 8:20 A.M. ET.

About Alnylam Pharmaceuticals

Alnylam 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, hepatic 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.