Ocata Therapeutics Inc fka OCAT

[rocky301]

Folks,

Per request, this is my take on upcoming trials. No guarantees, it's just how I see things.

The SMD was chosen to be first in trial because:
(stated by ACT)
a)Highest likelihood of seeing signal in Phase I
b)Significance of Market Exclusivity: Barrier to Entry
c)The only practical choice for first clinical trial involving
RPE cells for macular degeneration.

The Orphan designation allows ACT to possibly complete clinical phases in HALF the time it takes a non-orphan designation. "Signals" of progress can possibly happen during safety study in Phase 1.(big deal,imo) Early positive results combined with more efficacy results may position ACT to apply for an NDA(new drug application) much quicker than any other disease indication. As mentioned in prior post, NDA can be submitted and granted prior to complete testing results being finished. Whether or not we will meet the following clinical benefits I am not sure, but somehow seems to fit.

"Accelerated Approval
Traditional approval requires that clinical benefit be shown before approval can be granted. Accelerated approval is given to some new drugs for serious and life-threatening illnesses that lack satisfactory treatments. This allows an NDA to be approved before measures of effectiveness that would usually be required for approval are available.
http://www.fda.gov/drugs/resourcesforyou/consumers/ucm143534.htm

What the NDA Approval means to me? This is a critical part of any trials. Only about 16% or less of all orphan designated trials receive NDA approval. Not only will it allow the exclusive marketing(7 years) to kick in for a relatively low number of SMD patients but IMO it says much more considering we have the money maker AMD trial most likely running in tandem with SMD at some point. The FDA APPROVED NDA for SMD means that the FDA has assured itself, and the world, the MA09-hRPE cell line is SAFE IN HUMANS. This exact same cell line is being used in the much larger AMD trial so safety Phase there should be limited at best. Mr. Caldwell pounded the importance home a while back also..

William M. Caldwell, Chairman and CEO of ACTC: I think we’ve alluded to some things a couple of times either on blogs or in conferences, and I can start with the approval of our IND for Stargardt’s Disease which we will be seeing some time in the first half of the coming year. (That will mark) us going into the clinic. And we have already alluded to the fact that we will go into multiple sites, not just one particular site, for the reason that we have filed for Phase I/Phase II. For those who are not familiar with that, Phase I really focuses on safety. That’s going to be a very, very important piece, not just because of the safety, but because it will ensure for the FDA that this cell type can be safe in humans.

Once ACT has Orphan Drug Approval established and a non-orphan indication in trials too, I feel the following(below) from my Part 1,2,3 post a while back has a chance to kick in. Knowing the cell type is safe in humans and seeing decent results in SMD patients may have Doctors in the field prescribing off-label usage for injections to AMD patients long before the AMD trial is finished knowing clients won't be harmed and yet the injections may help. Insurance company coverage would be a huge bonus, but not absolutely required if AMD patients are willing to pay out of pocket.

"Once established as an orphan drug, off-label usage and expansion to nonorphan indications can lead to an increase in sales. Genentech’s Rituxan, for example, became a blockbuster drug within a short span of time as it was prescribed for many off-label indications. Drugs such as Centocor’s Remicade have become blockbusters due to expansion to nonorphan indications including rheumatoid arthritis. In fact, 12 out of 19 leading orphan drugs had just one approval for an orphan indication but became blockbusters by expanding to nonorphan indications or by off-label usage"
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=58584839

The above is my take on a potential path for our two hESC trials. Two things are crucial to any of the above transpiring.
1)we have to actually treat a patient and start the trials...lol
2)show safety and positve results at every turn.

hESC trials are new and only one is actually underway. Will the FDA scrutinize to death or actually find the hESC's can be brought through the process quicker than others? We shall see(no pun intended)..:)

Keep in mind 84% of the trials did not receive NDA approval. It is a daunting process and a lot of work. The average NDA is between 70,000-100,000 pages long. Our IND filing was 4050..