RNAi: Regeneron pumps $800m into ‘potentially game-changing science’

Regeneron will look to develop RNA interference (RNAi) therapeutics for eye and CNS diseases by teaming with and investing in Alnylam Pharmaceuticals.

RNA interference (RNAi) therapies are a new class of drugs that work by inhibiting gene expression or translation by neutralizing targeted mRNA molecules.

The first – and only – commercial RNAi therapy is Alnylam’s Onpattro (patisiran), approved by the US Food and Drug Administration (FDA) last August for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

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Alnylam has a broad pipeline of RNA silencing candidates but going forward it will work with Regeneron Pharmaceuticals in the development and commercialization of all therapies for disease targets expressed in the eye and central nervous system (CNS).

Under terms of the deal, Regeneron will pay Alnylam $400 million (€355 million) upfront to take the lead on development and commercialization for all programs targeting eye diseases, and to jointly advance and alternate leadership on CNS programs. Alnylam will be entitled to potential milestone and royalty payments.

Regeneron has also agreed to acquire a stake in Alnylam, purchasing 4.4 million shares at a cost of $400 million, representing approximately 4% of the number of shares outstanding.

“At Regeneron we believe the best use of our resources is to invest in potentially game-changing science that will yield innovative medicines for patients with serious diseases,” Regeneron’s president George Yancopoulos said.

“This collaboration couples proven and emerging RNAi technology, which holds important promise in many diseases, with Regeneron’s world-leading genetics research and target discovery engine.”

Alnylam’s CEO John Maraganore added the alliance will likely become “a significant opportunity for RNAi therapeutics as potentially transformative medicines for ocular and CNS diseases.”

Sanofi pulls out

In related news this week, French biopharma firm Sanofi has ended the research and option phase of a RNAi therapeutics alliance with Alnylam in rare genetic diseases inked in 2014.

“Our alliance with Alnylam has successfully brought one important medicine to patients with ATTR amyloidosis and it also has spawned a molecule that is in pivotal clinical trials for people with hemophilia,” said John Reed, global head of R&D at Sanofi.

“As we wrap up this phase of our program with Alnylam, we remain committed to advancing therapies for patients with rare diseases and rare blood disorders.”

Sanofi did not respond to questions posed by this publication at the time of going to press.