1,200% return. Looking back at November of 2012, when ACADIA traded at just $2 a share, the upside was so apparent, although no one noticed. However, as the stock apparently flew under-the-radar of so many, there is a good lesson to be learned, such as what you should have noticed, and how can you use this experience to help you in the future....
if the trial hits its end point of an 8.0 month improvement in overall survival, it would be a pretty spectacular achievement as oncologists generally consider a 4.0 to 5.0 month increase in median overall survival in a rapidly growing cancer like glioblastoma multiforme as a major advance.
Everyone, including the FDA, would be impressed by ICT-107 hitting the primary endpoint in this trial- FDA has past approving history on this basis
ImmunoCellular Therapeutics: Marching Toward Top Line Data for ICT-107 in 4Q, 2013 (IMUC, $2.42)
Posted by Larry Smith on May 10, 2013 • (2)
This note is based on comments made by management during the ImmunoCellular Therapeutics (IMUC) conference call of May 9, 2013.
Data from the Phase II Trial of ICT-107
ImmunoCellular continues to guide that the Data Monitoring Committee for the ICT-107 trial in glioblastoma multiforme will conduct an interim safety and futility analysis during the second quarter. The Company believes that the most positive outcome would be a recommendation to continue the trial. IMUC continues to expect that it will have top line results at the end of 2013.
The phase II trial is based on 124 patients with an endpoint of median overall survival. It is randomized roughly 2:1 so that about 83 patients will receive ICT-107 plus standard of care and 41 will receive standard of care. The powering assumes an improvement for the ICT-107 regimen of 8.0 months over 18.8 months median overall survival assumed for standard of care.
What Comes After Phase II
There is hope on the part of some investors that the phase II data will be so positive that IMUC will file for approval in the US and will not need to conduct a phase III trial. The current CEO has not taken this position as he consistently has stated that this is a phase II trial, but his predecessor did not discourage this thinking.
I think that there is virtually no chance that the Company can file for approval in the event of a successful phase II trial. IMUC conducted its phase II
trial using product supplied from two different GMP production facilities, first at the University of Pennsylvania and later at a NeoStem (NBS) facility. The FDA has a stringent requirement that the manufacturing processes and quality control assays used in clinical trials be nearly identical to those which will be used when the product is commercialized. This should be especially true for living cell therapy products in which the manufacturing process is essentially the product. ICT-107 production is based on the maturation of monocytes obtained from a patient through a blood draw that are then matured into dendritic cells, loaded with tumor antigen and re-injected in the body.
My excitement with ICT-107 and that of many other investors comes from the very long survival tails that were seen in the 16 patient phase I trial in which median overall survival was 38.4 months and in which 55% of patients survived for more than three years. The phase II trial that was started in 2011 and will report topline data in late 2013 cannot provide the same insight into long term survival. However, if the trial hits its end point of an 8.0 month improvement in overall survival, it would be a pretty spectacular achievement as oncologists generally consider a 4.0 to 5.0 month increase in median overall survival in a rapidly growing cancer like glioblastoma multiforme as a major advance.
Everyone, including the FDA, would be impressed by ICT-107 hitting the primary endpoint in this trial-but, I can’t see the FDA as approving it on this basis. Overlooking the manufacturing inconsistency caused by using two suppliers in phase II is not a precedent that the agency would want to set. Also, results based on only 124 patients is a small number for a drug that if, approved, would be widely and very quickly used in a patient population of perhaps 4,000 to 5,000 glioblastoma patients with the right immunotyping. I believe that the FDA will require two additional phase III trials or ask for one additional phase III trial using the phase II for supporting data. This assumes that the phase II is successful.