Transcript from SeekingAlpha:
Earnings press release:
- 2013 is our "road to the clinic"
- nominate first clinical candidate 1h2013, on track for first two INDs in 2014
- approximately 12 months from nominating candidate to filing IND
- ended 2012 with >$98 million cash, $5.5 million debt outstanding
- cash to operate into 2016
- expect to end 2013 with >$60 million cash
- spend of $30-35m in 2013, not counting milestones
Most advanced program is mir122 for Hepatitis C Virus infection (HCV)
- has activity against some of the mutations associated with resistance to current oral therpaies in development
- "In addition, we have shown in rodent models that our lead candidate has a rapid onset of action and the target gene depression was sustained for longer than 28 days after a single subcutaneous dose."
- "we believe there’s a clear market opportunity for our miR-122 program in a niche population of difficult to treat patients, those who may have failed current therapies or in combination with other anti-HCV agents. Currently our program supports once a month dosing and it’s positioned for a reduced duration of treatment against a broad range of HCV genotypes"
- Question: At what point in time just roughly do you think you could be in a position to be able to demonstrate human proof of concept? "Depending upon whether we do this in Europe or the U.S, we could get into these HCV infected patients relatively quickly [transitioning from initial clinical trial in healthy volunteers]. And then we need to figure out whether we do this in some of the specific genotypes like the G2 or G3 genotype. But hopefully we’d be able to start to see clinical proof of concept towards the end of 2014."
- once a month IV dosing works because patients return to doctor monthly to measure viral loads.
- Binding sites for our lead drug are identical in all of our 3000 clinical isolates, regardless of genotype
mir21 for kidney fibrosis and oncology
- data at 2012 Kidney Week in preclinical model of Alport syndrome, orphan disease representing 2% of end-stage renal disease with no approved therapy
mir33 for atherosclerosis and metabolic diseases
(one of these two would be second program into clinic)
internal mir10b program for glioblastoma multiforme (GBM)
- working with xenograft models in collaboration with Samsung collecting a lot of preclinical data. Now assessing whether we have achieved preclinical POC, will report more in 1-2 months.
- "several [additional] attractive oncology targets" identified, report more in the near term [co stated they will be at AACR April 2013 and this is likely venue for both announcements/presentations]
Other notes from Q&A session:
- mid year/3q will announce data from collaboration with Biogen BIIB related to multiple sclerosis (MS)
- ISIS/ALNY have 85% phase 1 success rate with RNA therapeutics (lack of drug drug interactions is one reason)
- Question: will you seek FDASIA breakthrough status for any of your programs? "Obviously we’re going to do everything we can to accelerate the development of our programs, including the opportunities that the FDA provides."