Alnylam Launches “Alnylam P5x25” Strategy For Planned Transition To A Top Five Biotech In Market Capitalization Over Next Five Years

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.

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* The preliminary selected financial results are unaudited, subject to adjustment, and provided as an approximation in advance of the company’s announcement of complete financial results in ...

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