Alexion joins with Dicerna to develop RNAi therapies for complement diseases

Alexion has announced a collaboration with Dicerna Pharmaceuticals to discover and develop RNA interference therapies for diseases caused by problems in the complement system.

Boston-based Alexion enjoys blockbuster revenues thanks to Soliris, which works by inhibiting the complement system, which can cause serious diseases when activated in an uncontrolled manner.

The company therefore already has expertise in this area as Soliris is already approved in three rare diseases, notably paroxysmal nocturnal haemoglobinuria (PNH).

But Soliris is reaching the end of its patent life in the US and EU, where protection ends in 2021 and 2020 respectively.

Alexion is looking for new therapies that it could potentially market in its area of expertise to replace the revenues lost if Soliris encounters competition from cheaper biosimilars once its patent expires.

RNA interference, or RNAi, finally came of age this year after drugs from Alnylam and Ionis were approved by the FDA for nerve damage caused by a form of inherited amyloidosis.

The technology works by blocking transcription of the section of RNA responsible for producing a rogue protein that leads to disease.

Alexion will gain exclusive worldwide licenses as well as development and commercial rights for two preclinical molecules from Dicerna developed using the latter’s GalXC technology.

A biotech based in Massachusetts, Dicerna will receive an upfront payment of $22 million and an equity investment of $15 million, with potential milestone-dependent and royalty payments.

Additional development and approval milestones will trigger payments of up to $105 million per target, plus sales milestones and mid-single to low-double digit royalties on future product sales.

Alexion and Dicerna will collaborate on the discovery and development of subcutaneously delivered GalXC RNAi molecules directed to two complement pathway targets for the treatment of complement-mediated diseases.

It will also have the right to exercise options for additional payment for two additional RNAi molecules directed to targets on the complement pathway.

Dicerna will lead the joint discovery and research efforts through the preclinical stage, and Alexion will lead development efforts beginning with phase 1 studies.

John Orloff, 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.”


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