By Alex Keown


Shares of Alexion Pharmaceuticals are up nearly 4 percent in premarket trading after the company announced it has teamed up with Dicerna Pharmaceuticals on the development of an RNAi therapeutic for the treatment of complement-mediated diseases.

The agreement will allow Alexion to harness Dicerna’s GalXC technology to dive into the RNAi pool. Using an RNAi-based approach to block production of complement pathway factors will provide the potential to inhibit the uncontrolled complement activation that leads to many diseases, the companies said in their joint announcement.

RNA interference (RNAi) is a process where certain double-stranded RNA molecules inhibit the expression of disease-causing genes by destroying the messenger RNAs (mRNAs) of those genes. Dicerna’s proprietary RNAi technology platform GalXC, uses the Dicer enzyme, a natural initiation point for RNAi within the cell structure. Using the GalXC technology, researchers can attach N-acetylgalactosamine sugars directly to the extended region of the proprietary Dicer substrate short-interfering RNA (DsiRNA) molecules, which yields multiple conjugate delivery configurations that allow flexible and efficient conjugation to the targeting ligands while stabilizing the RNAi duplex, according to data provided by Dicerna. Douglas M. Fambrough, chief executive officer of Dicerna, said the GalXC technology is designed to silence the expression of disease-driving genes in a way that is highly specific and allows for “convenient, infrequent subcutaneous administration.”

Alexion’s agreement with Dicerna gives the company exclusive worldwide licenses as well as development and commercial rights for two of Dicerna’s preclinical, subcutaneously delivered GalXC RNAi molecules. The deal also provides Alexion with an exclusive option for other preclinical GalXC RNAi molecules for two additional targets within the complement pathway.

John Orloff, head of research and development at Alexion, said the company has demonstrated the “transformative impact of complement inhibition” with its approved drug Soliris, a drug used to treat paroxysmal nocturnal hemoglobinuria, a rare blood disorder that affects about one or two people out of every million.

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,” Orloff said in a statement.

For Dicerna, the collaboration with Alexion comes about a month after it demonstrated clinical proof-of-concept for DCR-PHXC, our lead program for the treatment of primary hyperoxaluria, which uses the GALXC technology. Fambrough said the Alexion deal provides an opportunity for the company to “expand and advance” its GALXC pipeline.

“Our collaboration with Alexion provides access to the deep expertise and resources of an established leader in complement-mediated diseases. We look forward to working with Alexion to discover and develop promising new RNAi therapies,” Fambrough said in a statement.  

Under terms of the agreement, Alexion will provide Dicerna with $22 million in an upfront payment, as well as a $15 million equity investment in the company. Additionally, Dicerna could earn up to $105 million per target in various milestone payments.

Shares of Alexion are at $126.80 in premarket trading, up from Monday’s closing price of $123.



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