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Santaris on Track This Year to Move miRNA Drug Into Clinic, Expand miRNA Rx Pipeline

Santaris Pharma is on track to begin a phase I trial of its microRNA-targeting hepatitis C therapy in Europe in the first half of this year, company CEO Keith McCullagh said last week.
Speaking in New York at the annual BIO-CEO and Investor conference, he also announced that Copenhagen-based Santaris expects to advance a new miRNA drug into preclinical development before the end of the year and hinted that the company remains eager to float its shares publicly when market conditions improve.
Noting that Santaris has finished manufacturing the clinical materials and has conducted the necessary rodent and non-human primate toxicology work for the planned study, McCullagh said that the trial will begin “before the middle of this year.” He added that the company is preparing a clinical trial authorization filing — the European equivalent of an investigational new drug application — for the study.
Initial results from the planned phase I trial are expected to be available before the end of the year.
McCullagh also noted that Santaris expects to “bring three new drugs out of research into preclinical development,” including a locked nucleic acid targeting an oncology-associated miRNA.
“It’s going to be a busy year and I hope we will see further evidence of efficacy from” the firm’s drugs, he said during his webcast presentation.
The HCV candidate, SPC3649, targets miR-122, the most abundantly expressed miRNA in the liver and one linked to cholesterol regulation and lipid metabolism. Like many of Santaris’ other drugs, it is an LNA — essentially a nucleic acid analog in which the ribose ring is locked by a methylene bridge connecting the 2'-O atom with the 4'-O atom.
In previously disclosed non-human primate studies, SPC3649 significantly decreased total plasma cholesterol levels when administered as an IV bolus at doses ranging from 3 mg/kg to 10 mg/kg. Given to the animals on days 1, 3, and 5, the drug also maintained its dose-dependent, cholesterol-lowering effect out to 90 days, according to Santaris.
Despite miR-122’s apparent effects on cholesterol levels, Santaris expects an agent targeting this miRNA will be effective for hepatitis C given recent evidence suggesting the non-coding RNA is necessary for HCV replication.

“Of course, we are looking at how we might bring the company public,” but because of “current market conditions, that won’t be immediate.”

In 2005, researchers from Stanford University published data showing that sequestering data showing that sequestering miR-122 in liver cells triggered a “marked loss of autonomously replicating hepatitis C viral RNAs.” And last year, a team of investigators from Rockefeller University showed that antisense-based inhibition of miR-122 can block the virus’ ability to replicate.
Last December, Santaris CSO and Vice President of business development Henrik Orum told RNAi News that while “the jury is out … on how miR-122 assists HCV in performing its replication cycle … there are a couple of putative target sequences for microRNA-122 in the 5’ and 3’ regions of HCV.
“So [miR-122] definitely has the capability of binding to the HCV genome, and that may be how it permits replication to be completed,” he said at the time.
A handful of other companies are developing their own miRNA-targeting drugs, including ones against miR-122, but if Santaris keeps on schedule it will likely be the first to test such a compound in humans.
With this milestone in its sights, Santaris has also moved to expand its pipeline of miRNA therapeutics to include a drug candidate for cancer, according to McCullagh.
Although specific details about the new drug are not being released just yet, McCullagh told RNAi News this week that it will target an miRNA known to be “involved in oncogenic transformation.”
When disclosed, the miRNA “won’t be that much of a surprise,” he added.
Going Public?
As it evaluates new therapeutic opportunities, Santaris continues to gauge the financial marketplace with an eye to going public, McCullagh said at the BIO-CEO event.
“Of course we are looking at how we might bring the company public,” but because of “current market conditions, that won’t be immediate.”
Santaris has been weighing the possibility of going public since at least mid-2007, when McCullagh said during an investor conference that the company was considering an initial public offering in order to raise the capital needed to advance its drug-development programs (see RNAi News, 5/24/2007).
However, by November of that year the company put those plans on the back burner as it neared completion of a €20 million ($29.3 million) Series C round of venture capital financing (see RNAi News, 11/15/2007). About a month later, Santaris announced that it had both closed the Series C round and inked an antiviral drug-discovery, -development, and -commercialization partnership with GlaxoSmithKline worth up to $700 million, giving it enough money to last through 2009 without an IPO (see RNAi News, 12/20/2007).
During the BIO-CEO meeting, McCullagh said that Santaris currently has cash reserves totaling €44 million and reaffirmed the firm’s expectation that it can fund its operations for the next two years without an additional cash infusion. He noted, however, that a possible IPO remains on the corporate radar screen.

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