On a roll, Dicerna notches $100M upfront as Eli Lilly eyes RNAi applications in neurodegeneration, pain

Just days after selling co-development rights to two of its preclinical RNAi therapies to Alexion for a cool $22 million, Dicerna has scored another Big Pharma collaboration — this time moving further up the research stream but scoring a much bigger upfront.

Eli Lilly is paying $100 million for dibs on some new applications of Dicerna’s GalXC platform — neurodegeneration and pain — as well as the cardiometabolic work that’s become staple in the RNAi field. Add that to Lilly’s $100 million equity investment ($18.47 per share, a 42% premium from Dicerna’s $DRNA closing price on Friday) and the $350 million in milestones for each of the 10 targets that the partners expect to pursue, and Dicerna is looking at a pact potentially worth $3.7 billion.

Dan Skovronsky

“We are excited to collaborate with Dicerna and utilize their RNAi expertise to study targets that up until now have proven to be very technically challenging,” said Lilly CSO Dan Skovronsky in a statement. “RNAi has the potential to treat an array of diseases that are of strategic importance to Lilly.”

For each of the GalXC molecule identified, Dicerna will be responsible for the preclinical development until Lilly takes over to conduct clinical trials and potential commercialization, Dicerna CEO Douglas Fambrough told me.

The research and licensing pact represents a big leap since Dicerna got “early validation” from Boehringer Ingelheim last November, in a deal that came with $10 million upfront and $191 million in potential payments.

Douglas Fambrough

Building on the premise that RNAi can silence disease-causing genes by destroying their messenger RNA before they can give instructions to make disease-causing proteins, the GalXC platform is designed to produce molecules that operate in “the most specific and potent fashion.”

While the current paradigm — as well as its efforts so far — has focused on liver cells, Dicerna believes it is well equipped to explore other forms of RNAi delivery.

“It is already established that oligonucleotide therapeutics can be utilized successfully in neurological conditions, most notably the Spinraza treatment for spinal muscle atrophy,” Fambrough wrote in an email. “We anticipate collaborating with Lilly on the use of different targeting ligands and chemistries to optimize delivery to neurological cells. Due to challenges crossing the blood-brain barrier, we anticipate that initially the molecules will be injected intrathecally.”

Once caught up in a bitter legal battle over trade secrets with rivals at the pioneering RNAi biotech Alnylam $ALNY, Cambridge, MA-based Dicerna has since settled the lawsuits and turned its attention to dealmaking as its lead asset entered the clinic.

“Dicerna’s business strategy is to retain full or substantial ownership stake in its key rare disease programs, while pursuing partnerships for more complex diseases with multiple gene dysfunctions and larger patient populations,” he said. “Notably, and consistent with our strategy, Dicerna retains rights to develop products independently for orphan neurodegeneration and pain indications.”

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