Deals, RNAi

Alexion doles out $22M upfront for two preclinical RNAi drugs from Dicerna

Former biotech ace Alexion Pharmaceuticals is still shopping to fatten its pipeline as it continues to shake off a sales practices scandal linked to its expensive rare disease drug Soliris that culminated in an executive exodus and top-to-bottom reorganization. On Wednesday, the Boston, MA-based company said it would fork out $22 million upfront to co-develop two preclinical RNAi therapies owned by Dicerna Pharmaceuticals $DRNA.

The agreement follows a September deal to buy privately held Syntimmune for up to $1.2 billion, and the acquisition of Sweden’s Wilson Therapeutics for $855 million in April.

RNAi therapies offer the potential to overcome key limitations of traditional approaches to treatment, and has long interested drug developers, but has frequently been met with clinical failure. RNA interference (RNAi) is a process in which certain double-stranded RNA molecules inhibit the expression of disease-causing genes by destroying the messenger RNAs (mRNAs) of those genes. Rather than targeting and binding to proteins to inhibit their activity like monoclonal antibodies, RNAi exerts its effects one step earlier in the gene silencing process by targeting the mRNA, the instruction set that directs the building of disease-causing proteins. Earlier this year, the FDA approved the first-ever RNAi drug, Onpattro, for the treatment of the hereditary transthyretin-mediated (hATTR) amyloidosis, which is a rare, progressive life-threatening disease.

Douglas Fambrough

Dicerna is focusing on liver-targeted diseases that have not been amenable to conventional therapies, and says its GalXC RNAi platform has fueled a pipeline of therapies designed to conquer challenges posed by prior generations of RNAi therapeutics — the technology is designed to silence the expression of disease-driving genes in a way that is “highly specific, generally well tolerated, and allows for convenient, infrequent subcutaneous administration,” according to CEO Douglas Fambrough.

“These therapies migrate to the liver where complement proteins are produced and degrade the messenger RNA before the complement protein can be translated. As such, these treatments would operate more upstream than Alexion’s current complement antibodies that block the complement proteins after they have been created, and the mechanism should also provide a longer duration of effect than antibodies,” noted Leerink’s Geoffrey Porges in a note.

As part of the deal, Dicerna is eligible to receive potential development and regulatory milestone payments of up to $105 million per target, in addition to aggregate sales milestones of up to $160 million and royalties on future product sales. In addition, Alexion is making a concurrent $15 million equity investment in Dicerna at a price of 17.95 per share, a premium of nearly 47% to its Tuesday close. Alexion, which also reported higher-than-expected Q3 earnings and revenue that just missed analyst estimates on Wednesday, has the right to exercise options on two additional GalXC RNAi molecules from Dicerna under the agreement.

Rival RNAi drug developer — and maker of Onpattro — Alnylam Pharmaceuticals $ALNY announced this April that Dicerna had agreed to fork out $25 million in cash and stock to settle a lawsuit filed in 2015 claiming it stole intellectual property related to Alnylam’s gene-silencing technology to develop therapies, opening the door for Dicerna to forge new alliances.

Other players in the RNAi field include Arrowhead Pharmaceuticals $ARWR, RXi Pharmaceuticals $RXII, Silence Therapeutics (LSE: $SLN) and iTherapeutics.

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