In a move to launch first-in-class drug candidates based on the science of microRNA, Isis Pharmaceuticals and Alnylam Pharmaceuticals have created a joint venture, Regulus Therapeutics, the firms announced Sept. 7.

Regulus hopes to have a drug candidate to treat hepatitis C in clinical trials within two to three years, Isis CEO Stanley Crooke told "The Pink Sheet" DAILY.

Alnylam develops novel therapeutics based on RNA interference while Isis focuses its development work using antisense RNA technology. The two firms have had a strategic relationship since 2004 in which they have cross-licensed each other's intellectual property for RNAi.

In addition, the firms hold a large IP estate surrounding the medicinal chemistry of oligonucleotide medicines (¹ Also see "Isis VP-Corporate Development Kate Winkler Corcoran: An Interview With “The Pink Sheet” DAILY (Part 2 of 2)" - Pink Sheet, 20 July, 2007.). Isis and Alnylam will grant Regulus exclusive licenses to their IP and early patents in the miRNA field, including the crucial "Tuschl III" patent.

Alnylam will make an initial payment of $10 million to balance out ownership of the joint venture. After that, the two firms share in the funding of Regulus. It will be operated as an independent company with its own board of directors and management team.

MicroRNA molecules, a form of RNAi, are single-stranded RNAs, about 20 nucleotides in length, that regulate gene expression by directing the cell to turn on other RNA code groups that produce proteins.

"When appropriately encoded, miRNAs represent potential disease targets whose selective antagonism can result in the correction of an entire disease pathway in a manner unachievable by today's medicines," the firms said. "Because RNAs regulate whole networks of genes that can be involved in discrete disease processes, miRNA therapeutics represent a new approach to target the pathways of human disease."

The inappropriate presence or absence of miRNA has been associated with specific human diseases including cancer, viral infection and metabolic disorders.

Regulus already has a lead molecule, miR-122, which is associated with HCV viral replication and has been extensively studied in animals, Crooke said.

Both Isis and Alnylam retain the option to develop drug candidates should Regulus decide not to move forward with a particular compound.

The new venture will be colocated with Isis headquarters in Carlsbad, Calif., and the firms expect to have an initial R&D staff of about 20, Crooke said. Isis/Alnylam plan to select a president and chief scientific officer for the firm in the coming months.

Regulus' scientific advisory board will be chaired by Nobel Laureate David Baltimore, California Institute of Technology.

Regulus will spend about $15 million to $20 million during the next two years, Crooke said, which includes Alnylam's initial payment of $10 million. The new company will also be funded by grants and commercial collaborations structured much as Isis and Alnylam have crafted their recent development deals with pharma firms.

Alnylam inked a nearly $1 billion deal with Roche in July to develop drug candidates in four areas based on RNAi technology (² Also see "Alnylam Signs Billion Dollar RNAi Deal With Roche" - Pink Sheet, 9 July, 2007.).

-Pamela Taulbee (p.taulbee@elsevier.com)

Starlight Success: Isis, Alnylam Form Regulus To Develop MicroRNA Antagonists

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