Gary Rabins blog....
First, let me welcome you all to summer. We have been very busy the last few months since our annual shareholders meeting, when we provided a comprehensive update on the company. I realize that many of you are looking to understand the status of the various strategic and clinical plans we laid out at that meeting, so I will provide a summary here.
Clinical Trials
Our clinical trials in the U.S. and U.K. continue to progress well, though at a little slower pace than we have hoped. We have, as of today, treated eight patients between the three ongoing trials. All are doing remarkably well – no safety issues whatsoever, and we continue to receive evidence of the ability of the injected RPE cells to impact positively on vision. Our most recent patient, treated Friday the 29th, was a Stargardt’s Disease sufferer, with the surgery conducted by Dr. Bainbridge at Moorfields Eye Hospital in London. While it is too early to say anything about that patient, for the other seven dry age-related macular degeneration (dry AMD) and Stargardt’s patients, we can report that we have seen both subjective and objective evidence of improved visual acuity and quality of vision (ability to see colors better, etc). Importantly, the effects appear to persist, with our first two patients closing in on the one year mark this month and maintaining the increases in visual acuity we measured shortly after their treatment. I emphasize that this is preliminary data, and we really need many more patients and data points, but it is very encouraging and we believe that we are on the road to providing a treatment for these devastating diseases. Before Friday’s treatment, we had two consecutive last-minute cancellations, costing us nearly a month’s delay in the UK trial. One patient had a (very rare) allergic reaction, and another patient was disqualified prior to treatment due to the need to treat a non-related medical event. Both matters disqualified the patients within days of their planned surgeries.
In the U.S., we will be conducting the fourth Stargardt’s treatment (and the first of the second 100,000 cell cohort) shortly, and have screened and identified the likely fourth AMD patient, whom we anticipate treating in July, pending DSMB approval. We will also plan to treat the third UK patient in July at Moorfields. As we now have multiple centers screening in the U.S., and will soon have multiple centers in the UK, we expect that this level of redundancy will allow us to have a second center step in shortly after any last-minute patient cancellations.
As we have previously announced, our Data and Safety Monitoring Board (DSMB) has approved the treatment of the remaining two patients in the first cohort of the U.K. Stargardt’s trial, as well as the first patient of the second cohort (now at a dose of 100,000 cells) in the U.S. Stargardt’s trial. We have submitted the data package for the first cohort of patients for the dry AMD trial, and await their review with the hope that we will soon receive the green light to move up to the 100,000 cell dose for that trial as well. I will keep you posted as we treat additional patients this summer, and you should expect to start hearing about additional trial sites (some yet to be announced) also initiating patient treatments.
My last remark on the trials is to share with you how proud I am that our company has attracted some of the leading eye hospitals and retinal surgeons around the world to work with us. What other small biotech company can boast about a team like ours?! Their expertise has been invaluable in this phase of our trials, and along with our expert consultants and board members, we are tapping into this great resource as we set our sights on the next targets: clinical endpoints for Phase II and III of the trials.
BIO 2012 and the Alliance for Regenerative Medicine
This past month (June 18-21), Boston hosted the 2012 BIO International Convention. This event collects together the world’s top life science companies. The official number of attendees topped 16,000, according to BIO. It was an opportunity to hear from key world figures, including former US Secretaries of the Treasury Henry M. Paulson Jr. and Robert E. Rubin, participate in breakout and company presentation sessions, and to have a number of one-on-one meetings with a variety of different industry-leading companies. We took advantage of this format, sitting down with folks from various large cap life science firms (many of whom we meet with regularly at these meetings, as well as a few new faces), and branched out from there to also take meetings with cosmetic and veterinarian divisions of household-name companies. We also used the opportunity to chat with Massachusetts Governor Deval Patrick and update him on our trials and the company as a whole. The Governor put it so well when he addressed the convention, “Every meeting, every connection is about improving the lives of patients and ultimately finding a cure.”
We used the occasion of BIO to give a public presentation on ACT which was very well-received. The Alliance for Regenerative Medicine (ARM), an industry-academic-patient advocacy group to which we belong, also took advantage of the gathering in Boston and organized the Clinical Outlooks for Regenerative Medicine meeting for that week. It was held at the Schepens Eye Research Institute at the Massachusetts Eye and Ear Infirmary – one of the most recent hospitals to sign on as a clinical trial site for our retinal pigment epithelial (RPE) program. A number of representatives from the big pharma community we had set up meetings with at BIO also attended the ARM event, reaffirming for us their interest in this space.
In addition to the one-on-one discussions that attending BIO afforded us, there was a clear affirmation of a general message coming out of the life science sector, a message growing in volume over the last few years, that provides encouragement for regenerative medicine and cell therapies as a whole, and ACT in particular. The drug development pipeline at the big life science companies is in serious danger of not being able to replace markets that will be lost when the “patent cliff” really comes into play at the end of this decade. Therapies are becoming more expensive to develop through to market authorization, and their internal research and development programs are not producing enough high quality (less risky) leads.
Jim Hoyes, the President of EMD Serono (the biopharmaceutical division of Merck KGaA, the German chemical and pharmaceutical company) summed it up during his speech at BIO, commenting, “Part of that is recognizing what we aren’t as good at.” The large cap life science companies are getting anxious, and regenerative medicine is poised to address the growing pipeline issue.
At our annual shareholders meeting, we told you that it was not likely a matter of whether we will partner one or more of our programs (such as the RPE program), but rather, when. We need to move further into our trials to really capitalize on the intrinsic value of our RPE cell therapy, but it is evident from the general tenor at BIO and our individual conversations that we are on the right track. We will continue to keep these lines of communication open – updating potential partners on our progress, and using those meetings to learn more about the issues that would be important to any diligence around a deal – and taking that input and using it to fine tune our regulatory and intellectual property strategies.
Reverse Split and Uplisting
We continue to move with great determination towards uplisting to the NASDAQ exchange with a concomitant reverse split to a qualifying share price. We have a very clear understanding of the few remaining boxes we need to check to complete the process, and are devoting significant management time to that effort. I remain very optimistic that I will have good news to report, and am convinced that once listed on that exchange, the outlook for the company’s stock long-term valuation will be positive. We have too many good things going for us on the science and development side for the company not to be a success story. I just need ask for your continued patience for the time being.
So, it only remains to say, we are looking forward to the rest of the year and what we hope will be a busy but very exciting summer. We will update you on all this and more on our 2Q earnings call in early August.
Thank you, as always, for your interest and support.
Gary Rabin
Chairman and CEO
Advanced Cell Technology, Inc.
Forward-Looking Statements
Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “will,” “believes,” “plans,” “anticipates,” “expects,” “estimates,” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the company’s periodic reports, including the report on Form 10-K for the year ended December 31, 2011. Forward-looking statements are based on the beliefs, opinions, and expectations of the company’s management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the company’s management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Company’s clinical trials will be successful.
AI
