Replies to post #32697 on Ocata Therapeutics Inc fka OCAT

[louisa]

You're welcome Rocky, I urge all to read this summary as it pinpoints imo where and how ACTC will proceed. Rocky and Rumit keep it real. Very fortunate to be in the same cyberspace as these guys.
And a few others who post sane info on this company.

to read the discussion on Orphan Status you can take a look here.

http://investorstemcell.com/forum/advanced-cell-technology/3662.htm

ORJUST READ IT HERE FOR THOSE NOT INCLINED TO DO THE MOVE:
ORPHAN STATUS: IMPLICATIONS and STRATEGY
ACT has Orphan Status both here in the US and EU. It's important to know how this fits into ACT's strategy. Status means ACT qualifies for all the Orphan perks which do not go into effect until Orphan Approval is received.

Why RPE Program? Was Orphan Status a reason?

Caldwell: We believe that we have a therapy that has multiple applications (retinal disease includes dry AMD, Retinitis Pigmentosa (RP) and Stargardt disease. The latter two may qualify as Orphan indications which could qualify for special treatment through the clinical process by the FDA. This was a large factor weighing in favor of selecting this therapeutic option.
Advanced Cell Technology (ACTC): Why did ACTC choose the RPE Program?

(Orphan Status Approval PR)
(FDA) has granted orphan drug designation for the company's MA09-hRPE cells for use in the treatment of Stargardt's Macular Dystrophy (SMD).

"As a result, the company is eligible to receive a number of benefits, including tax credits, access to grant funding for clinical trials, accelerated FDA approval and allowance for marketing exclusivity after drug approval for a period of as long as seven years".
Advanced Cell Technology

FDA Approved Application can be viewed here:
FDA Application

US Orphan Incentives:

There are several incentives but the two below are major.

1) 7 year market exclusitivity
Marketing exclusivity remains the most important incentive
(slide 10)
Marketing exclusivity, effective on the date of marketing approval, remains the most powerful incentive in the Orphan Drug Act, because it limits competition by prohibiting FDA from approving another version of the same orphan drug for the same indication unless the new drug is clinically superior.

2) 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."
The FDA's Drug Review Process: Ensuring Drugs Are Safe and Effective


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Knowing what Orphan Drug Status means and the fact ACT considered it a large factor in pursuing therapies that have multiple applications in the eye, it seems fair to extrapolate what it may mean.

ACT chose Stargard Indication as the "practical first trial choice for RPE cells" largely because this indication was deemed to have the best chance of showing some progress early in Phase 1 safety. Couple that with the fact Stargardt disease is a juvenile macular degeneration which translates into less RPE cell die off due to younger age and a Brusch's membrane with much less age also. Makes sense and is common sense.

My theory: Even though trials will be run in tandem, Stargardts will most likely offer more definitive early results than the dryAMD. With early signals and Orphan Status this will most likely move through the clinical process much faster with hopes of FDA accelerated approval applied to cut the time of trials way down and possibly receive Orphan Drug Approva(NDA)l prior to long effectiveness studies needed in non orphan trials.

The dryAMD trial is non orphan and will most likely require the normal trial and time setting.

With above theory in mind, we now have Stargardt's with an NDA under early approval. This means the marketing of MA09-hRPE can begin with the Stargardt population which we all know is limited and not nearly the money maker the large dryAMD crowd presents. Patients are treated and results have proven positive by halting progression and hopefully better acuity. The word spreads.

The same cell line is showing results in the dryAMD trial as patients for Stargardt's are being treated. This would open the door, imo, for the possibility of Doctors going the off label usage route for dryAMD prior to the NDA being issued for a much longer trial. I realize the indications are not exactly the same but some dryAMD
patients may want to try the RPE injection when hearing of Stargardt's success. Whether they pay out of pocket or some insurance coverage will pay is another topic. This scenario seems to fit what we have going, imo.

As posted prior, the following has made some big revenues in the past and I see no reason it couldn't apply here. The bolded below is another possibility.

"Some of the key growth factors involved in the transition of orphan drugs into blockbusters include market exclusivity options for multiple orphan indications, off-label usage and expansion to nonorphan indications, and freedom from generic competition".
Extra! Extra!"ACTC's Orphan Drug Approved in Europe".

Three key orphan drug strategies
- Market access and subsequent expansion into non-orphan
diseases offers significant opportunity for revenue generation
- Multiple indications shore up a drug's exclusivity in a niche
therapeutic area
- Stratification of indications into niche patient populations
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Summary:

Orphan Status defined verses Orphan Approval

ACT's statement of Orphan Status weighing heavy on their gameplan for trials.

Incentives and the importance of the 2 major ones

Accelarated Approval to shorten NDA(new drug approval)process

Possible theory on how it could play out

When ACT announced their first IND was for Stargardt and made Orphan Application, it all made sense then and it seems to make even more sense now. I like the concept and the possibilities their game plan presents. Injections soon, early positive signals, accelerated approval, and this plan has a chance to snowball much quicker than most trials do. Time will soon tell the story.