Alnylam: ‘Onpattro will springboard us into a global biopharma firm’

Alnylam says it is on course to becoming “a global multiproduct commercial biopharma company” by 2020 as sales of its messenger RNA interference therapeutic Onpattro (patisiran) begin to ramp-up.

Alnylam Pharmaceuticals made history in August 2018 when Onpattro (patisiran), its product for the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis, became the first small interfering ribonucleic acid (siRNA) treatment to be approved by the US Food and Drug Administration (FDA).

Reporting its first quarter 2019 financials, the firm described the milestone approval as laying the groundwork for future launches within its RNA interference (RNAi) pipeline and transforming Alnylam itself into a large pharma firm.

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“We’re at a very exciting stage in building fully integrated global biopharmaceutical company, and the capabilities we’ve built from Onpattro lay the groundwork for the launches of future products,” president Barry Greene told investors.

Alnylam: Onpattro and beyond

For the quarter, Onpattro pulled in net sales of $26.3 million (€23.5 million) – up from $12.1 million the three months prior – and the firm said over 400 patients are now receiving commercial treatment worldwide.

CEO John Maraganore said he expects this growth to continue due to Alnylam’s “success in achieving access for patients and recognition of the value of Onpattro,” while simultaneously moving other RNAi candidates through the clinic.

“A notable highlight for the quarter and recent period was our positive Phase III ENVISION results for givosiran, our investigational RNAi therapeutic for the treatment of acute hepatic porphyria,” he said as an example.

Meanwhile, the firm is expanding its preclinical pipeline to include RNAi programs in a broad range of neurodegenerative and ocular diseases, as demonstrated by the recent $800 million deal struck with Regeneron – “the largest collaboration Alnylam has ever formed.”

The Regeneron deal provided Alnylam with $400 million upfront and, along with equity capital raised through a follow-on offering in January, the firm’s balance sheet “now has the strength to support a multiyear horizon for business execution,” Maraganore said.

“We couldn’t be more excited about where we are in our efforts toward achieving our Alnylam 2020 goals of becoming a global multiproduct commercial biopharma company with a deep clinical pipeline for future growth and a robust product engine for sustainable innovation, a profile rarely, if ever, achieved in biotech history.”