Last night, we were delivered incredible news from Advanced Cell Technology (ACTC) about two trials that they currently have under way in the US for age-related macular degeneration, the most common cause of vision loss in people over age 60, and Stargardt's macular dystrophy, a rarer, inherited condition that affects younger patients and can leave patients legally blind.
In the study, 18 patients with either disorder received transplants of retinal epithelial cells (RPE) made from stem cells. 10 of the 18 Patients had significant visual acuity improvements. Just as importantly 17 of 18 showed that the progression has halted. These are blockbuster results. There are more than 30 million Dry AMD patients in North America and Europe alone, with no approved treatments currently available. They have also proven to be 100% safe so far. A safety profile like this is remarkable as well.
The lead investigator Dr. Swartz had this to say about the trials in Time:
"I'm astonished that this is working in the way that it is - or seems to be working," says Steven Schwartz, a UCLA eye specialist who led the study, which was published Tuesday in the British medical journal The Lancet. "I'm very excited about it."
These are life changing results for those who both improved their vision as well as those where it stopped the progression. Dr. Lanza describes how this can affect a patient's life:
"To understand what this means, let's assume you're six feet tall. If a patient can now see you after treatment, then before treatment you would have to have been 12 feet high - taller than a flagpole - in order for them to see you," he said.
With this new data I believe it is likely ACT receives a pivotal trial designation for one or both SMD and AMD. A pivotal designation would speed up the trials considerably, making commercialization not only more likely, but happen much sooner.
The Lancet's release of the study came the same day Advanced Cell filed a shelf registration statement with the Securities and Exchange Commission for the sale of up to $100 million in stock or other securities. We do not know at this point how the shares will be used or how many shares they will need. What it will do is give us a good balance sheet going onto NASDAQ so that institutions will invest in ACT and not have to worry when the next capital raise will occur. According to the company it is the first step in the re-IPO and up list process that the company has repeatedly referred to in the past. According to the CFO Ted Miles:
We remain committed to our previously stated plan of reverse splitting the stock, uplisting to a national exchange such as NASDAQ, and attracting fundamental based institutional biotech investors who can fund the company through the next several milestones.
The re-IPO process, which ACT has been following step by step is described in this article: The Re-IPO: An Emerging Strategy for Greater Growth and Enhanced Capital Market Access
ACT has a pricing committee headed by CEO Paul Wotton. This committee along with the help of their new accounting firm BDO, investor relations firm Westwicke Partners and NASDAQ will determine their new NASDAQ share price when they uplist. They of course will take into consideration the potential of these unmatched results.
ACT looks to be well on its way to accomplishing their goals of uplisting to NASDAQ and moving to Phase II this year. At their conference call this afternoon I expect to hear more about these groundbreaking results as well their plans for uplist and Phase II trials. Once ACT moves to Phase II the possibility of a joint venture increases exponentially, and after these results so will the price that the partner will have to pay. ACT has peer-reviewed proof that their RPE cells work. To those that have improved vision and or stopped the progression of these diseases this is undeniable.
I will end this with a quote from The NEJM Journal Watch:
A Lancet commentator calls the work a "major accomplishment," noting that while "much work remains to be done" before such therapies can be used outside of trials, "the path is now set in motion."
The path has indeed been set in motion. ACT is well on its way to becoming a world class regenerative medicine company.
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