- We expect clinical progress in LNP based programs this year
- PLK1 oncology candidate silences polo like kinase 1. phase 1 trial ongoing at 3 US medical centers. multi dose multi cycle dose escalation in patients w/ advanced solid tumors. new p1 trial in partnership with NCI - protocol approved, enrollment will start in next few weeks- primary liver cancer or liver mets. RNAi deliver directly into hepatic artery - measure kd direclty in biospies. allows more robust data for planning next trials. expect to have interim p1 data by ye2011
- wee1/csn5 oncology preclinial targets combo approach
- Ebola - progress under US TMT contract which could support development thru FDA product approval. IND on track by year end 2011. We have established scale-up from 10g to 1kg batches. This scale will support late clinical and commercial scale of LNP products.
- genetech collaboration - antibody or antibody fragments coupling to LNP - antibody-mediated cellular LNP uptake
- LNP chemistries for nebulization. We have pharma collaboration for respiratory disease. Our formulations retain biological activity
- Also investigating novel molecules that mediate RNAi (payload technology)
- 2q highlights - expanded BMS collaboration to include new proprietary LNP formulation for delivery to tumors and tissues outside liver. Also working to expand ongong target validation.
- ALNY deal provides manufacturing revenue, milestones, royalties
- Litigation: during 2q added Alcana as defendant. Both have responded and filed counterclaims. Issue is progressing on expected timeline. The lawsuit was necessary to regain control of our technology. We are ensuring that we can pursue this litigation without interrupting our core business activities.
- CFO summary-
- 2q2011 net loss $3.5m vs $4m 2q2010 due to increased revenue
- $4m revenue from $2.3m 2q2010. Loss of revenue from Roche offset by Ebola contract
- $6.2m r&d expenses up from $4.2m - significant ebola expenses that are reimbursed. Otherwise internal spending was lower
- $1.6m g&a from $1.1 2q2010. Increase due mostly to legal fees
- 6/302011 $9.7m cash.
- $5.1m raised in june.
- at 6/30 had high amount of accounts receiveable. $1.7m was received from government in july vs june
- Expect cash and expected revenue sufficient into 2h2012 (vs 1q2012 guidance at time of 2010 10k)
ApoB program - reformulation effort still underway? That is still part of our pipeline. We are evaluating some primate work with new formulations, will be reporting on that after we digest it.
update re ebola data...anything new relevant to other viral diseases? We are not actively involved in these at the moment, but in the past have published LNP against hepatitis B and have done internal work against HCV
next stages for ebola development? most work required for approval would be nonhuman primate? In the near term, expect IND filing, then human safety study. Then it would follow the "animal rule" w/ efficacy studies in animals
scaleup manufacturing relevant for ebola stockpiling? We can produce batch size of 1kg siRNA payload - sufficient for late stage clinical and at least commercialization of ebola. One would have to calculate whether this is enough for all products. But it is huge step in right direction
when will you need next increase in manufacturing scale? We are interested in going to higher scale, will eventually go to 5kg next under TMT.
synergies to rest of pipline? all of the products are manufactured in more or less the same way. so if we scale for ebola, it is applicable to all other pipeline products (internal or for external partners) To me this is a big positive factor of the government contract, along with the potential short development timeline for Ebola RNAi