Celsion safely past interim look

CLSN released a PR today:

http://www.celsion.com/releasedetail.cfm?ReleaseID=627024

Of which the important parts are shown below:

"Celsion Corporation (NASDAQ: CLSN), a leading oncology drug development company, announced today that the independent Data Monitoring Committee (DMC) for Celsion's Phase III HEAT Study, a multinational, double-blind, placebo-controlled, pivotal study of ThermoDox® in combination with radio frequency ablation (RFA) for hepatocellular carcinoma (HCC) or primary liver cancer, has completed a planned interim analysis for safety, efficacy and futility and unanimously recommended that the study continue to its final analysis as planned. The DMC evaluated data from 613 patients in its review, which was conducted following the realization of 219 progression-free survival (PFS) events within the study population. A total of 380 events of progression are required to reach the planned final analysis of the study.

Celsion also announced today that the DMC, in its review, followed a statistical boundary determined by the Company using the Lan DeMets implementation of the O'Brien-Fleming spending function. This approach allows for the Company to conduct additional interim efficacy analyses prior to final data read-out at 380 PFS events with no increased risk of statistical penalty. The additional analyses may allow for earlier stopping of the study. Additionally, based on its internally modeled estimates of PFS events, Celsion reconfirmed that 380 PFS events are projected to occur in late 2012.

"The DMC's unanimous recommendation is a significant achievement for Celsion based on the most comprehensive review of the HEAT Study to date, including the first-ever review of efficacy results," said Michael H. Tardugno, Celsion's President and Chief Executive Officer. "Critically, we have the potential to realize a successful outcome to the study prior to its planned completion. We are encouraged by what may be sufficient rationale for conducting an additional preplanned efficacy review prior to the 380 events called for in our protocol, and will seek to amend our Special Protocol Assessment Agreement with the FDA accordingly. We thank the DMC for their work and thorough review, and are grateful for the continued support and enthusiasm from the healthcare community, regulators, our investors and our employees."

The HEAT Study is being conducted under a U.S. Food and Drug Administration (FDA) Special Protocol Assessment, has received FDA Fast Track Designation and has been designated as a Priority Trial for liver cancer by the National Institutes of Health. Target enrollment of 600 patients was reached in August 2011, after which the DMC conducted this interim efficacy analysis based on the realization of 219 progression-free survival events. Consistent with the Company's global regulatory strategy, Celsion is continuing to enroll patients in the HEAT Study in order to randomize at least 200 patients in China, a requirement for sFDA (State Food and Drug Administration) registrational filing in that country and to ensure timely readout of final data. In addition to meeting the U.S. FDA enrollment objective, the HEAT Study has also enrolled a sufficient number of patients to support, in Asia, registrational filings in S. Korea and Taiwan, two very important markets for ThermoDox®."

So - my thoughts are as follows:

Disappointed? Yes - I did think we had a great shot at interim. Funny thing is, we just don't know: was P-value there but OS not enough of a trend? ("totality of the data")

So what do we know?

Safety is not an issue.

Enrollment at 613 (mid/late Sept?) This worries me a little bit. If 600 was ~ 8/5 and enrollment was 613 on 9/19ish, enrollment in China is slow. However, I believe that only those treated and at least one follow up might qualify under the 613. In that case, true enrollment might be closer to 625ish... I will have to follow up with the company here.

219 evaluated as "events". Q was asked at cc about the # past 190 and MT said "not substantial." 29 over = ~15% surplus... in my book that is substantial, but whatever.

The biggie in my estimation is that DMC advised them to seek an amendment to the SPA, instead of "simply" reccing a continue to 380. There is NO reason for this unless they were awfully close to being close enough to stop the trial. There is no other reason - period.

We have enough cash for about 8-11 months, depending on how much burn changes from the mrq. HEAT costs are "over the hump" supposedly but then we're still enrolling to 700, plus ABLATE expenses start. I assume ~1.7-2M a month, yielding my 8-11 months. We've demonstrated that we're willing to scrape the barrel... In my opinion it depends 1) on the FDA chat re the SPA and how soon we decide to take another peek and 2) on how licensing talks with big pharma (BP) go. Now that we're "de-risked" past the interim, BP may be more willing to loosen the purse strings. I repeat: with the DMC rec to amend SPA, the cat is out of the bag.

Was it Rodman & Renshaw that said $6 if a continue? I'm disappointed today, but especially if the overall market rallies, we'll nudge out of the mid $2s soon enough.

-Trond