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Alexion targets RNAi with $637m Dicerna deal

Bolsters pipeline ahead of Soliris patent protection loss

Alexion has signed a deal with RNAi specialists Dicerna to discover therapies for complement-mediated diseases.

The deal gives Dicerna $22m in upfront payments and it will also eligible to receive a further $265m in sales and development milestones.

Alexion has also committed $15m for exclusive rights to two, subcutaneously delivered GalXC RNAi molecules, currently in pre-clinical development, and has received an exclusive option to further develop two other preclinical RNAi molecules.

In return, Alexion will have access to Dicerna’s GalXC technology, which is designed to advance the development of next-generation RNAi-based therapies.

The drug technology platform produces compounds that inhibit complement activation, and it can be applied across multiple therapeutic areas, including cardiovascular diseases, viral infectious disease and liver diseases.

More specifically, the molecules are processed by GalXC via the initiation point for RNAi within the human cell known as the dicer enzyme, leading to multiple conjugate delivery targets.

Unlike earlier generation RNAi molecules, which only mimic the output product of dicer processing, Dicerna’s platform can enter the RNAi pathway before dicer processing, which increases efficacy of the RNAi mechanism.

The deal is an important one for Alexion, as looks to lessen its dependency on its flagship product Soliris, a blockbuster which is set to earn up to $3.4bn this year.

However it will lose patent protection in 2021 in both Europe and the US. Alexion already has a follow-on complement inhibitor to be the next generation replacement for Soliris.

Named ALXN1210, the compound has demonstrated non-inferiority in a phase 3 study comparing it to Soliris in patients with paroxysmal nocturnal hemoglobinuria (PNH). This was a disappointing result for the new drug, even though it has a more patient-friendly dosing regimen. ALXN1210 was filed with the US regulator in August, with an FDA decision expected by 18 February 2019.

John Orloff

Alexion's John Orloff

Commenting on the new deal with Dicerna, John Orloff, executive vice president and head of R&D at Alexion said:

“With Soliris, Alexion has demonstrated the transformative impact of complement inhibition on multiple serious and debilitating diseases.

“This collaboration provides the opportunity to continue building on our more than two decades of complement expertise using Dicerna’s proprietary GalXC RNAi technology platform, which provides a potentially promising new way of inhibiting the uncontrolled complement activation that we know plays a significant role in many devastating diseases.”

The new deal adds to others made by Alexion this year, including the $855m acquisition of rare disease company Wilson Therapeutics in April and privately-held autoimmune disease specialists Syntimmune in September, in a deal worth up to $1.2bn.

25th October 2018

From: Research, Sales

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