Regeneron in $1bn RNAi pact with Alnylam

By Ben Hargreaves contact

- Last updated on GMT

(Image: Getty/Ktsimage)
(Image: Getty/Ktsimage)

Related tags: Alnylam, Regeneron, Sanofi, Phio Pharmaceuticals, RNAi

Regeneron and Alnylam sign discovery agreement that could advance 30 targets within CNS and ocular disorders, during an initial five-year discovery period.

The agreement between the two companies sees the larger of the two, Regeneron, make a $400m (€354m) upfront payment and take a $400m equity stake in Alnylam. Depending on the success of the candidates developed, Alnylam could also receive a further $200m.

The collaboration will focus on developing treatments for the eye and the central nervous system (CNS), as well as progressing programs designed to target genes expressed in the liver.

"Importantly, the alliance structure enables Alnylam to continue to build its industry-leading pipeline of RNAi therapeutics while retaining significant product rights,”​ said John Maraganore, CEO of Alnylam.

He continued, “In addition, the near-term payments under this new agreement will strengthen Alnylam's balance sheet with over $2bn in pro forma cash upon closing of the transaction, supporting our global efforts to develop and commercialise multiple products as potentially breakthrough medicines and advance our profile toward sustainable profitability."

Both companies will look to identify potential RNAi therapies, with Alnylam having received the first regulatory approval for such a treatment – after the US Food and Drug Administration approved Onpattro​ (patisiran) for the treatment of polyneuropathy caused by hereditary transthyretin-mediated (hATTR) amyloidosis.

According to the companies, as potential therapeutic candidates are discovered, the partnership will “advance and alternate leadership on CNS programs.”​ The lead party on the selected programs will retain global development and commercial responsibility, while both companies have the option to participate equally in potential future profits of programs led by the other party.

Regeneron reserved the ability to take a lead on all therapeutics for eye diseases, due to its position in the market – having Eylea (aflibercept), a treatment for macular degeneration, a product which brought in $1bn in annual sales in the US market during 2018.

“The partnership between Alnylam and Regeneron signals that momentum for RNAi will continue in force on the heels of the landmark FDA approval of Alnylam’s Onpattro in 2018 for treatment of hATTR amyloidosis,”​ concluded Paul Jeng, senior pharma analyst at GlobalData.

Prior to this partnership, Sanofi had signalled its intention in the space by acquiring Bioveratic for $11.6bn​, and RNAi specialist, Phio Pharmaceuticals, expressed the belief that RNAi therapeutics will make a significant impact​ on patient need in the coming years.

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