Final data from Santaris’ miravirsen Phase 2a trial on HCV to be presented at ILC 2012

Apr 19 2012

Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapies, will present final data from a Phase 2a trial showing that miravirsen given as a four-week monotherapy treatment provided robust dose-dependent anti-viral activity with a mean reduction of 2 to 3 logs from baseline in HCV RNA (log10 IU/mL) that was sustained well beyond the end of therapy. These final clinical data are being presented at the International Liver Congress™ 2012, the annual meeting of the European Association for the Study of the Liver (EASL), taking place on April 18 – 22 in Barcelona, Spain.                    

Clinical data from the Phase 2a study will be presented in an oral presentation on April 20, 2012 at 5:15 p.m. CEST, titled "Final Results: Randomized, Double-Blind, Placebo-Controlled Safety, Anti-Viral Proof-of-Concept Study of Miravirsen, an Oligonucleotide Targeting miR-122, in Treatment-Naive Patients with Genotype 1 Chronic HCV Infection." Study results demonstrated the following:

• Miravirsen was safe and well tolerated
• Adverse events were generally infrequent, mild and similar between treatment groups
• No dose limiting toxicities or any discontinuations due to adverse events
• Miravirsen was associated with dose-dependent reductions in HCV RNA that were sustained well beyond the end of the four-week dosing period
• Four out of nine patients treated at the highest dose (7 mg/kg) with miravirsen became HCV RNA undetectable with just five weekly doses of miravirsen monotherapy
• No evidence of viral resistance

"These data provide clinical evidence that miravirsen's unique mechanism-of-action offers a high barrier to viral resistance and the potential for cure with monotherapy. Due to its ability in targeting miR-122, miravirsen has the potential to change the way hepatitis C is treated," said Dr. Henk Reesink, Academic Medical Center, University of Amsterdam, who is presenting the data at EASL. "These data show that longer duration of miravirsen monotherapy has the potential to produce sustained virological responses."

The mean of the maximum decline from baseline in HCV RNA (log10 IU/mL) over the 18 week study was 1.2, 2.9, 3.0 in the 3, 5 and 7 mg/kg miravirsen dose groups respectively vs. 0.4 in the placebo group.

"We are pleased that the final data set reinforces that miravirsen, the first microRNA-targeted drug to be given to patients, provides long-lasting suppression of HCV RNA, has a high barrier to viral resistance and favorable tolerability and dosing profiles," said Michael R. Hodges, MD, Vice President and Chief Medical Officer at Santaris Pharma A/S. "Miravirsen has the potential to be used in combination with direct acting anti-viral agents as part of an interferon-free, dosing regimen in patients with all types of HCV genotypes."

The randomized, double-blind, placebo-controlled, ascending multiple-dose Phase 2a study assessed the safety and tolerability of miravirsen in treatment-naïve patients with chronic HCV genotype 1 infection. Patients with chronic HCV genotype 1 infection were enrolled sequentially to one of three cohorts (9 active: 3 placebo per cohort) at doses of 3, 5 and 7 mg/kg. Miravirsen was given as a total of 5 weekly subcutaneous injections over 29 days.

Additional data supporting the final results in the miravirsen Phase 2a study will also be presented in posters demonstrating evidence of its high barrier to resistance and favorable dosing schedule:

• "Preclinical characterization of miravirsen (miR), an oligonucleotide targeting miR-122 in the HCV genotype 1B Replicon System" – Soren Ottosen, et al.; April 20, 2012 from 12:30-2 p.m. CEST
• "Pharmacokinetics of miravirsen, a miR-122 inhibitor, predict the prolonged viral load reduction in treatment naïve genotype 1 HCV infected patients" – Robert Persson, et al.; April 21, 2012 from 12:30-1:30 p.m. CEST
• "Sequence analysis of HCV variants from a Phase IIa trial of miravirsen (miR), an oligonucleotide targeting miR-122, in treatment naive patients with chronic HCV infection" – A.K. Patick, et al.; April 21, 2012 from 12:30-1:30 p.m. CEST

SOURCE Santaris Pharma A/S