Amid RNAi buzz, Arcturus touts positive preclinical results

San Diego-based Arcturus Therapeutics added some juice to the RNAi buzz this week at the J.P. Morgan Healthcare Conference with preclinical results from a study that showed a hefty knockdown of the genetic disease transthyretin (TTR)-mediated amyloidosis. Its LUNAR delivery tech is at the center of the flagship program.

The unlocked nucleobase analog tech is a lipid nanoparticle delivery system designed to bring small bits of RNA into cells, where it acts on the genetic code. The technology allows for the "targeting of any gene in the human genome," the Johnson & Johnson ($JNJ) and Janssen-backed company says.

The preclinical study showed a 75% knockdown of the protein that causes amyloidosis, according to Arcturus. And these effects lasted for 10 days, with a sustained effect of three weeks with no adverse events.

In October, the company pulled in $5 million to back its platform, which it said it intends for the orphan disease market, which includes TTR.

"We are pleased to announce our flagship program targeting TTR including our successful achievement of non-human primate data with durable biological effect and having an impressive safety profile," CSO and COO Pad Chivukula said in a statement. "With this data in hand, we are laser-focused on selecting a wholly-owned clinical drug candidate in 2014 and entering the clinic soon after."

RNAi drugs are hot this week in the wheeling and dealing at J.P. Morgan. Sanofi ($SNY) promised to invest $700 million in Alnylam's ($ALNY) lead drug and its portfolio of therapies currently in trials. Roche ($RHHBY) opted in, as well, for almost half a billion dollars, investing in Santaris for its locked nucleic acid platform. Merck ($MRK), on the other hand, dropped its own stake in Sirna Therapeutics to Alnylam.

So with RNAi making waves in the Big Pharma sector, can Arcturus eventually contribute a ripple? If it can nominate a candidate in 2014 as hoped, there is a chance.

CEO Joseph Payne told FierceDrugDelivery in October: "We plan to lead the next wave of RNAi technology."

- here's the release