Dicerna agrees another gene-silencing deal, this time with Lilly

Eli Lilly has agreed a broad collaboration with Dicerna, sealing the deal with a $100 million equity stake in the RNA interference specialist.

The big pharma company is also paying $100 million upfront to Dicerna in licensing and research fees for the project, which will see the two companies work together to develop RNAi-based therapies for  cardio-metabolic disease, neurodegeneration and pain, plus up to $350 million in milestones per target taken through development.

The terms give the deal an eye-watering top end of $3.7 billion or more based on 10-plus programmes taken forward,  and is a big endorsement for Dicerna’s GalXC RNAi platform, as well as taking Dicerna into new therapeutic areas beyond its current focus on cardiovascular, rare diseases and  viral infections.

It’s also the second collaboration in a week for Dicerna, coming after it agreed a smaller alliance with Alexion to look for RNAi-based drugs for complement diseases. Other partners for the US biotech include Boehringer Ingelheim, which signed up last year, and Japan’s Kyowa Hakko.

RNAi graduated from the lab to the bedside in the summer when the FDA approved the first two drugs in the class – Alnylam’s Onpattro (patisiran) for hereditary transthyretin-mediated amyloidosis (hATTR) – after decades of R&D. It’s widely anticipated that many more RNAi-based drugs will follow Onpattro onto the market, particularly for targets that have not been amenable to more conventional drug discovery approaches.

The approach uses RNA molecules to switch off the expression of disease-associated genes by binding to and disrupting messenger RNA (mRNA), the cellular mechanism used to transcribe proteins from them.

Dicerna’s approach is a little different. It uses double-stranded RNAs, which are longer than other companies’ short, interfering RNAs, and links them to sugar side chains that it says gives the resulting RNAis more potent and longer-lasting activity against mRNA.

The Boston-based biotech will work exclusively with Lilly in the neurodegeneration and pain fields, as well as on selected targets in cardio-metabolic diseases. Meanwhile, the two companies say they will work together on a new nucleic acid platform technology to “generate next-generation oligonucleotide therapies.”

“At Lilly, we go to where breaking science meets unmet medical needs,” said the company’s chief scientific officer Daniel Skovronsky. “RNAi has the potential to treat an array of diseases that are of strategic importance to Lilly. Together with Dicerna, we aim to employ this emerging modality for greater success in drug development.”