OCAT
De la Junta de accionistas de ayer. Resalto la información más o menos nueva.
Slide 3
Ocata addresses Macular Degeneration with 2 product candidates.
Orphan indication for SMD followed by
Potential Blockbuster in AMD
With ATMP status in Europe
Slide 4
NASDAQ listing “ Really Cleared the Decks” of past misdemeanors with the company when it was known as Advanced Cell Technology.
Expanded the technology to include IPSCs via Allele deal, which boosts pre-clinical pipeline and diversifies cell sources.
RPE patents have the potential to block all current competitors.
Impressive data around Lupus program, intend to partner this.
The surgeon who implanted the first bionic retina, which was announced yesterday in the UK, will probably be working with Ocata on SMD. http://www.bbc.com/news/health-33571412
Now that have raised capital, can focus on ? pipeline more aggressively.
Slide 12
Have done lots of market research, part of which indicates that market penetration will be achievable with a small sales force of about 50 people.
Slide 24
SMD Pivotal Trial 2H 2015
First patient will be treated in 2H 2015, following SPA meeting with FDA. Full enrollment in 2017.
Trial designed to enable potential approval in 2019.
Endpoints: look for change in visual acuity and change in area of geographic atrophy.
A panel of experts will look at the injection on a video monitor to direct the surgeon to inject at a specific location.
Slide 25
AMD Phase 2, start 3Q 2015
Some treated with placebo.
15 treated with 3 different immunosuppressive regimens. (13, 7, and 1 week)
Slide 27
Preclinical – Ocata technology extended beyond hESC’s, includes iPSC’s
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Systemic hESC – PhRPs provide neuroprotection in mice and rats
Slide 30
DeltaCell manufacturing process in cGMP environment
Slide 33
41.6 million shares and 2.75 million warrants outstanding
Q3 2015 Dry AMD Phase II 1st subject enrolled
Q2 2016 Dry AMD Phase II 1st cohort w/3 month data
Q3 2016 Dry AMD Phase II 2nd cohort w/3 month data
Questions
Responses to questions asked by
Belas, Mike C, Simon
Have lots of date for photoreceptor progenitors
Lupus data – same exciting data as seen previously has been repeated in additional animal models.
SPA – want buy in from FDA before starting Phase II
Can apply for Breakthrough status for Phase II, but unlikely to get it. BT usually done in Phase 3.
Use of the fellow eye as a control is probably valid, but not accepted by FDA.
Tufts paper(s)? in peer review process.
Response to question by Simon, why was JV date moved
Ocata was ready to sign, but the partner company moved it. it would be a small JV and platelets and that they are in discussion with a company but it's a small company…so that people know platelets are on the table but not to expect much...
Comentario del foro:
IMO the platelet partner will be Platelet Bio-Genesis and it is their company that IMO is most likely to have applied for the NIH Grant RFA-HL-022 and the two companion grants. The NIH advisory board makes their decision on Aug 23 with a possible start date in Sept/Oct.
Marion
Why is PPS below $5?
Don’t know why, but Caught up a little bit with failure of Avalanche’s gene therapy.
Also, now tied to moves in Russell 2000 Index
Otro resumen más caótico pero que incluye alguna cosa más:
Dry AMD Ph 2
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-Dry AMD Ph 2 will be 200K injections for all cohorts
-They had a meeting last February with EU on Ph 2 there: “Trial design was agreed upon then”
-Potentially launching treatment in EU in 2019 if endpoints are met
-“Increased pigmentation” seen in patients who received the treatment
-Also noted the surgeon who did the bionic eye will “probably be working with us” in Ph 2
-At a recent conference on the SMD/Dry AMD Ph 1, there was “a line of ophthalmologists waiting to talk to us”.
-“There’s no other company with such a comprehensive approach to the eye”
-“The amount of new technology going into Phase 2” I think he said is “very substantial” or “very impressive” or something like that. He noted new imaging technology has been designed specifically in connection with this trial.
-Not sure if he said “lots” or “some” or “several” of papers and presentations at conferences are upcoming on Ocata’s RPE “starting in the Fall”.
-Paul said Ph 2 will be the “first time a cell therapy trial will be done with placebo control”.
-In Ph. 2 they’ll be injecting “much closer to the fovea” since safety has already been established in Ph 1. So potential is there for even greater efficacy.
-Also noted the 2 endpoints previously stated and “either one will get us an approval”. I’m guessing he meant for SMD.
-“Trying to reproduce Lancet data but with a placebo control”
3 Garbled Notes that may or may not be related: 1) Patents include iPS 2) Kind of thing Pfizer was looking at 3) “Working on new formulations”
-Not sure if he was speaking about 1 thing or 3 different things then
-First cohort reporting this time next year
-Staargardt’s enrollment expected to go to 2017
-Other studies (mentioned Genentech) did fellow eye controlled. So Ocata will be the first to know how different are the results based on fellow eye vs placebo control. I didn’t write it but could have sworn he said that knowledge of this variation may be kept as a company secret vs allowing other companies to see what the variation is exactly.
Photoreceptor Progenitors
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-iPS were better than embryo-derived in mice for photoreceptors
-“Regenerating the old cells” rather than adding new cells.
-In mice, a tail vein injection was performed but the cells did not migrate to the eye. Most of the cells were “found in the lungs” so it’s more likely to be a paracrine effect. -> Control teratogénico
Platelets JV
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-“Small company” interested in working with Ocata on the platelet program.
Manufacturing Related
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-You have to get at least 3 batches consistently to make the FDA happy
-“We now have done 4 batches”
MSCs
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-Tuft’s papers “in the process”
General/Various
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-“Bob Lanza has been working on iPS for a long time”
-Expanding the team to “take it to the next level”. I also noted that he said Ocata is choosing the “cream of the crop” of stem cell researchers to work with (hire? Not sure if he meant hire or just work with.)
-“Bob’s working on numerous publications”
-Many “upcoming presentations“
-SAB meeting also happened last February. “They are all committed to what we are doing”
-I did ask Dr. Lanza 1 question that also fits in the category of general interest - how does he and his team divide their time: are certain members of the team assigned to only work on MSCs, others to RPEs, other to platelets. He answered that, other than the people working in manufacturing full time, the rest of the scientists are not exclusive to a single project and go back and forth. I think this relates to what potentially makes Ocata’s team so effective. Keeping the overspecialization at bay that frequently develops in larger companies than Ocata.
