On January 10, 2022 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its preliminary* fourth quarter and full year 2021 global net product revenues for ONPATTRO, GIVLAARI and OXLUMO and provided additional updates on the products’ commercial launches (Press release, Alnylam, JAN 10, 2022, View Source [SID1234598556]).
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2021 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 2021 were approximately $139 million and $475 million, respectively, representing quarterly and annual growth of 15% and 55% compared to Q3 2021 and full year 2020, respectively.
As of year-end 2021, over 2,050 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 2021 were approximately $41 million and $128 million, respectively, representing quarterly and annual growth of 28% and 132% compared to Q3 2021 and full year 2020, respectively.
As of year-end 2021, over 350 patients worldwide were receiving commercial GIVLAARI.
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.
Preliminary global net product revenues for the fourth quarter and full year 2021 were approximately $19 million and $60 million, respectively, representing quarterly growth of 29% compared to Q3 2021.
As of year-end 2021, over 140 patients worldwide were receiving commercial OXLUMO.
Finally, the Company today reported that it expects its full year 2021 non-GAAP operating loss to be substantially improved relative to the prior year, as the Company continues to transition toward a self-sustainable financial profile.
Further, at December 31, 2021, Alnylam had preliminary cash, cash equivalents, and marketable securities of approximately $2.4 billion, as compared to $1.9 billion at December 31, 2020.
"We are pleased to have closed out 2021 on a very strong note with continued execution across our commercial portfolio, delivering top-line revenue at the upper end of our guidance range. These preliminary results reflect the dedication of our teams to deliver these important medicines to patients in need around the world, despite the variable dynamics posed by the ongoing pandemic. We’re also excited to have ended the year with Leqvio, partnered with Novartis, becoming the fourth RNAi therapeutic approved in the U.S., and the first indicated to treat a major risk factor for a highly prevalent disease," said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam. "Furthermore, as we reflect upon the one year anniversary of the announcement of our Alnylam P5x25 strategy, we believe we are well on our way to achieving or potentially exceeding these ambitious goals, positioning Alnylam as a top-tier, global, multi-product commercial company with a broad pipeline and organic platform poised to deliver sustainable innovation well into the future, a profile rarely seen in our industry."
Alnylam management will discuss these preliminary selected financial results and commercial updates during a webcast presentation at the 40th Annual J.P. Morgan Healthcare Conference, being held virtually, tomorrow, Monday, January 10, 2022 at 9:45 am ET.
About RNAi Therapeutics
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 or disease pathway 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.