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  • elk_1l elk_1l Feb 11, 2014 9:26 PM Flag

    Snippets from I-Cell thread on ACT Corporate Presentation - BIOCEO - February 2014

    An explanation from Matt about Slide 4:

    "Slide 4 provides an overview of clinical and preclinical pipeline on the cell therapy side of the company’s efforts.

    Ophthalmology: In addition to the clinical RPE programs, the company is advancing the photoreceptor progenitor and ganglion progenitor through proof-of-concept animal studies with early but promising resuls. Need to be extended in terms of number of animals and other animal models

    Autoimmune/Inflammation: one of the relations the company has created has been with Tufts Vet School. Under the approved INAD ACT has with the FDA, the company has been engaged in testing human MSCs (eventually will move to dog MSCs) in various spontaneous diseases in companion pets. A secondary, but significant benefit is that the data the company is generating under the INAD can be used to support IND applications for equivalent human clinical trials. In Preliminary testing (pre-clinical research) before in human trials: Lupus, MS, sepsis, wound healing, IBD and Alzheimer"

    On that slide, concerning 'Drug Delivery - Engineered platelets... Engineered RPE', which made an impression on me, Matt also added:

    "Since the company controls the manufacture of various cells and cell fragments (platelets) from a stem cell source, it offers the opportunity to engineer the stem cell line so that the resulting transplantable cells and tissues will express a recombinant therapeutic program. There is more on this on slides 21 and 35."

    On the 33 injected patients:

    Cohort 3 completed for both SMD and AMD in both US and UK studies. For Cohort 2A - 1 patient left to enroll for each of the US Dry AMD and SMD trials.

    On the MMD:
    MMD trials projected to commence by end of Q1 – working with Jules Stein to help move this process to enrollment

    Concerning the timing of TLD publication, Matt writes:

    "We are excited by the data, as are the investigators involved in the study. We are working with all the clinical trial sites to find the right balance with respect to everyone’s concern that the data be published in the most impact journal as possible (which is good for investors and their share price) against an expectation of some form of top line data release. We do not want to misstep with release of top line data in a format that reduces the likelihood of getting a scientific paper on the trials published in one of the most reputable and respected journals"


    [slide 11] shows us that ACT can generate enough doses (50,000 - 100,000) from their own facility to generate $500 Million to $1 Billion in annual sales, assuming a cost of $10k per dose.

    I like Slide 17 showing that the MMD trial is recruiting, although it isn't showing up on yet. The only MMD trial I find is this one, sponsored by the Retinal Associates of South Florida: Stem Cell Ophthalmology Treatment Study It's enrolling 300 patients, started August 2013, and the estimated completeion date is August 2017 (4 years). Note the very brief lists of Inclusion/Exclusion criteria.

    Slide 22 (IP) gives me the shivers!
    "Covers RPE Cells derived from any pluripotent stem cell source - From hESC to iPS cells to pluripotent stem cells yet to be invented"

    Slide 25:
    "We have demonstrated that subretinally injected hESC- and iPSC-derived photoreceptor progenitors can migrate to correct anatomical site in retina, differentiate, and functionally rescue vision."


    my understanding is that [ACT] photoreceptor progenitors will rescue & regenerate rods & cones directly. This is a little different than coffey's scaffolded RPE & ACT RPE cell suspension methods as they are meant to engraft to the bruchs for treatment of the root cause-- loss of RPE.

    This is why RNP/photoreceptor progenitors may be of more benefit to later stage patients with poor health of the bruchs---also suspecting that retinitis pigmentosa patients have a compromised bruchs, but not sure. ACTs cell suspension method is becoming more apparent as the right approach for the onset of AMD/SMD

    As for the later stage patients, I am very optimistic after seeing the pre-clinical results. I think anyone would opt for maybe a few highly effective injections per year vs a risky eye surgery with limited potential for increased visual acuity. In the end, late stage patients will need to regenerate their LOST-- not dormant photoreceptors. In other words, curing the root cause will not help alleviate irreversible damage. The eye surgery is seemingly not worth it considering the alternative.


    Slide 19:

    33 Patients - Up to 2 years of Follow-Up Visits
    •Measurable Improvements in Visual Acuity
    •Gains in Visual Acuity Generally Persist


    David Schull (Russo Partners): (Telegram)

    Ted Myles in 1/22/2014 PR:

    Sentiment: Strong Buy

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