As we near the Q2 conference call next week, in what has become a ritual it seems, I have devised some questions to relay to Celsion management ahead of the call, both for them to address during their prepared remarks, or for shareholders to follow-up on during the Q&A session. It is worth mentioning again that Celsion management, coming from the CEO directly, has invited such questions ahead of conference calls. As I also mentioned before, I maintain frequent communication with other Celsion shareholders who I deeply respect, and the questions below represent our collective thoughts (thank you fellow shareholders, you know who you are):
- With Q4 right around the corner, and event rate projections relatively clear at this point, can the company still confirm DATA, not 380 PFS events, will be due by end of 2012?
- Although it has been asked before, it still bears asking again: have any new studies or data come up that make you question the historical guidance for 11-12 months median PFS for the RFA-only arm?
- What level of detail can we expect in terms of data when top-line results are finally revealed? In addition to median PFS times and hazard ratios for the whole trial, can we expect:
- Average tumor size for the trial?
- Complete ablation rates for both arms?
- Rates of local versus distant recurrence (MOST interesting for me personally)?
- OS events and trends to date?
- Side effects/Adverse events?
- Break-out of data for 3-5 versus 5-7 cm groups?
- Is the next DMC meeting still scheduled for mid-September?
- Is it the company’s expectation that 380+ confirmed PFS events will have been reached by the time this meeting occurs?
- I have asked before about the average lesion size in the HEAT study, and I know the company did not want to yet report this data. Can you at the least give us a sense for the % proportion of patients in the 3-5 cohort versus 5-7 cohort? Is it safe to say the vast majority are in the 3-5 group, or are they evenly split?
- What timeline can the company provide for when the full 372+ OS data would be made available?
- Do you believe Yakult will be able to work on a new Japanese trial with the data available from top-line? Or will they need to wait until all (including greater-matured OS) data is available?
- Ongoing Phase II colorectal liver metastases ABLATE study: Can the company report current enrollment in this trial (and number treated, if different)?
- The company will eventually have 3 ongoing HIFU studies with ThermoDox, one in Pancreatic cancer at University of Washington, one for liver metastases at Oxford, and the PII w/Philips for bone metastases.
- Pancreatic HIFU trial: As I understood from the press release, this will start with preclinical work in animal models, not human patients. Can you provide additional color on the types of questions that will be explored during these studies? Is it possible that future trials in humans involve ThermoDox plus other agents, such as gemcitabine?
- Liver metastases HIFU trial: What phase study will this be, how many patients, will this look at metastases from any primary site or specified sites (colorectal, melanoma, etc.), expected timeline for completion and what primary endpoint? Will this have the same primary/secondary endpoints as the ongoing ABLATE study in CRLM?
- Have there been any updates from potential licensing partners? Is the company still leaning towards keeping rights to the US market?
- Does the company have any updated views on potential pricing of ThermoDox®?
- Does the company expect cash burn to increase following top-line data due to NDA preparation?
- Will an MAA in Europe be pursued concurrently to the NDA in the US, or after the NDA?
- As the company is likely well aware, obtaining European marketing authorization is one thing, but reimbursement is a local, country by country game. A common theme throughout Europe is having health economic data to support reimbursement. Has the company proactively thought about/started developing such data for ThermoDox®?
- I have assumed that upon NDA submission and acceptance, a priority review of 6 months is essentially guaranteed from the FDA as a function of fast-track status. Can the company confirm that this is the case?
- Per Onyx’ recent conference call, it appears data from the adjuvant Nexavar trial following resection or ablation won’t be available until 2014. What competitive threat does the company perceive from early line use of Nexavar in the adjuvant setting?
I look forward to what should be a very interesting call, as there have been new developments on a couple fronts (Philips HIFU PII, RCW abstract acceptance at ESMO, etc.), and I expect we will get some more color on the HEAT study. As a reminder, it is scheduled for this coming Tuesday, the 14th, at 11AM ET/8AM PT.