The reason RFA plus Thermodox will report successful phase III primary results is because the HEAT trial design requires at least one lesion "No more than 4 HCC lesions with at least one ≥ 3.0 cm and none 7.0 cm in maximum diameter, based on diagnosis at screening" and "If a subject has a large lesion (5.0 - 7.0 cm), any other lesions must be less than 5.0 cm." Next, it will be explained why the trial design strongly favors Thermodox's success.
[HCC = hepatocellular carcinoma]
Support for the Primary Assertion
The context for the following four points is derived from Dr. Poon's presentation at the 2012 Annual Congress of the Cardiovascular and Interventional Radiological Society of Europe.
1. There are no current satisfactory treatments for patients with hepatocellular carcinoma in the liver with measured lesions/tumors in the 3-7 cm range. That is the lesion target range in the HEAT phase III study. RFA alone, the placebo arm, is destined to fail. Recurrence and survival rates are dramatically shortened in patients with tumors 5 cm. This is an established fact: Liver cancer is the Grim Reaper of cancers. Celsion's placebo arm, RFA alone, has a long history of failure.
2. The HEAT phase III study is a two arm comparison of (1) RFA alone, versus (2) RFA plus Thermodox in at least one cancerous lesion(s) in the range of 3-7 cm. The key for investors weighing their investment decision in Celsion is grasping the fact that RFA alone in lesions in the range of 3-7 cm, especially in tumors 5 cm, is not curative. This is why Celsion could predict so precisely the timeframe for the clinical trial. There are hundreds of cases documenting the time-frame of tumor recurrence (See Dr. Poon's presentation).
3. Only RFA plus Thermodox offers the potential to totally destroy lesions in the range of 3-7 cm because of the wider margin of the treatment. This is why Celsion is betting on an improved recurrence rate and ultimately overall survival with the addition of Thermodox. Celsion has plenty of data and sixty plus years of doxorubicin's history of being an effective cancer killing agent if it can be delivered in high concentrations. That is exactly what Thermodox with RFA is designed to do.
4. The ingenuity of the HEAT phase III trial design is that Celsion has chosen with the FDA, EMA, and Asian authorities' approval to match their treatment regimen of RFA plus Thermodox against RFA alone in a particular tumor size range. Why the FDA, EMA, and Asian authorities would agree to this is quite simple: there is no current satisfactory treatment for people with tumors in the 3-7 cm range especially 5 cm. No doubt this is why Thermodox was granted Orphan Drug status and is being Fast-Tracked by the FDA because there is no other satisfactory treatment for liver cancer patients with deadly tumors of this size.
While there are bulls and bears on both sides of the upcoming January 2013 HEAT phase III results, clearly investors are anxious to weigh the risk. Upon failure, a nosedive in the share price below $2/share is inevitable. But upon success, given Thermodox's global opportunity and its multiple Thermodox footprint in the company's pipeline, $20-$50/share appears to be a conservative target for 2013. After all, this would be a first-line treatment for hepatocellular carcinoma liver patients with tumors in the 3-7 cm range, and obviously, Thermodox could be used in lesions that are smaller.
While investors want to hear and read both sides of the HEAT trial's potential outcome, the study's trial design tips the scale in Thermodox's favor. It is also explains why the company has been so good in predicting the clinical trial's completion date knowing the short recurrence rate in patients receiving RFA treatment alone. In fact, the short case argument actually lends further support to the thesis that RFA alone is a miserable failure because it is incapable of adequately eradicating cancerous cells in tumors in the range of 3-7 cm and that information is documented in hundreds of cases across multiple clinical trials.
the bashers on here don't like to look at the facts and call us dreamers on here but when you look at the numbers, ie rev, shares, and pipe line there is no arguing. we just have to have the data first.