Early Summer MCB--myopia IND

[Wallace907]

During Lanza's presentation, it was stated that the new xeno/feeder free MCB will be up and running early in the summer and that a new IND(most likely myopia) would then be able to be filed.

It makes me wonder if we are planning to do our "bridge study" this early on in our trials(contrary of Rabins statement of "a few years away" and dependent on sociopolitical factors. Are we planning to switch to an NED line this early on in our SMD/AMD trials?

We should also be hearing about our iPS MCB and results from our collaboration with cellular dynamics. It seems like yesterday we were talking about the timelines that are now present.

I know a lot of people are eager to fume during our next CC, but I think ACT is building a mountain of progress to overcome those frustrations. It is now July 1, the timelines are coming together in the east and west, we will hear something on these programs in due time.

We should also have a solid reimbursement valuation by now. In UK study shown here:What is the cost of blindness? -- Meads and Hyde 87 (10): 1201 -- British Journal of Ophthalmology

the first two years of legal blindness costs around 13k GBP. (Important number according to Rabin as the vast majority of payers are responsible for approx. 22 months)

The one year follow-up in our first two patients are being finalized as we dwell in this no-news period. How closely is The Lancet following these trials, I wonder?

I seem to think that many of the timelines that were outlined during the ASM were surrounded during the early to mid summer months, so it will come to no surprise that there was bustling activity going on. I, for one, anticipate an extended update as our progress has evolved inherently too rapid for frequent PRs. This "quiet period" is also consistent with our transition to an event-driven company/stock.

Remember, Rabin et al has already stated that phase II is where we will crank out injections. This is not the time nor place in these trials to waste important knowledge in understanding the best methodologies in delivery. At worst, we have broadened our inputs from a newly designated SAB from the top opthalmologists in the country. If we consider that all of these investigators will be included in the next lancet article, we should know that each will deliver their finest work--as they are all aware of the implications of this publication--whether its early this winter or early next year.

All in all, the only thing that is frustrating is not being able to read posts from some of the great thinkers that have soaked up nearly all the info available!

We'll hear from you soon enough!

[LongDolly]

Good post Wallace

We all know what is happening behind the scenes is much more that the dribble that we have heard in the last month. I get the feeling when we do have the next wave, it will be irrefutable evidence, no placebo possible. This type of Tsunami I will gladly welcome.

[iamwhatiam]

I think you'll find that the placebo explanation has already been debunked since the AMD patient's non-treated eye returned to her baseline, unlike her treated eye. The initial improvements in her untreated eye, as indicated earlier, may have been some kind of cross-education related placebo effect, but it is only in the eye treated with RPE's that a durable response has been seen. So poo-poo to the placebo.

[Wallace907]

As far as CDI is concerned, the extent of our collaboration may emphasize the use of essential 8.



we should now be able to scale up our iPS cells under GMP.

The data to support our claims of no placebo effect in the untreated eye remains to be published, and for some, will be a footnote when results are compared with 100k injection results. This should be evident in Lanza's reaction to MV detailing the progress of our first patients during the ASM.


Over time I think that these RPE cells were able to pigmentate which made them much easier to locate on naked BrM. Although these cells are somewhat accessible, our pre-clinical studies required complex multi-staining in order to identify many of our new RPE layers...with higher dosages, it will become more evident in both visual acuity(more cells=more attachment) and pigmentation that we don't have placebo effect going on in the treated eyes. We have the luxury of having a straight-forward CSO that is fully confident of how these trials will progress, and will impulsively relays those type of insights.