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Waiting for an IND, Advanced Cell Technology, Inc (OTC BB:ACTC.OB)


ACTC.OB or ACT is focused on developing and commercializing human stem cell technology.  All of ACT’s technologies are at the basic research or in the pre-clinical stage of development, (www.advancedcell.com).

Focus: ACT’s technologies are  basic research or in the pre-clinical stage of development.  ACT has 3 core research programs:  Cellular Reprogramming—the transformation of a patient’s own cells into embryonic stem cells which can then be differentiated into therapeutically useful cells for treatment of disease; Reduced Complexity Program—the production of stem cell therapies for ‘‘off-the-shelf’’ deployment to treat acute disease in a time frame for critical situations that are not amenable to reprogramming technologies and  Stem Cell Differentiation—the development of technologies designed to control the differentiation and re-differentiation of stem cells into specific cell types, such as hematopoietic, myocardial, skin, retinal and neuronal cells  for therapeutic application.

 Market Opportunity: Age related macular degeneration (NASDAQ:AMD) represents a “tremendous” market opportunity given the size of the patient population and the lack of treatment alternatives. Diseases of the eye affect more than 30 M people worldwide and represent a $20+B market.

  • Existing therapies only treat the condition and are not curative but palliative and do not regenerate cells of the macula that have been damaged.  Additionally, existing therapies only treat Wet AMD not Dry AMD hence leaving a significant unmet need for an effective treatment for the 90% of AMD patients who have the dry variety,
  • AMD is a common eye disease that leads to deterioration of the macula which is the central area of the retina in the back of the eye, the paper-thin tissue where light-sensitive cells send visual signals to the brain.  In particular, sharp, clear, “straight ahead” vision is processed by the macula.  Injury to the macula results in the formation of blind spots and blurred or distorted vision.  When the macula becomes damaged, many daily activities such as driving and reading become increasingly difficulty,
  • AMD most often affects individuals over 50 years of age.  There are two forms of macular degeneration: dry macular degeneration or wet macular degeneration.  Dry AMD takes place when the light-sensitive cells in the macula slowly break down, gradually blurring central vision in the affected eye.  As dry AMD gets worse, the patient may see a blurred spot in the center of their vision.  As the disorder progresses, and less of the macula functions, central vision is gradually lost in the affected eye.  The most common symptom of dry AMD is slightly blurred vision.  The affected individual may need more light for reading and other tasks,
  • Wet AMD occurs when abnormal blood vessels behind the retina start to grow under the macula.  As the dry form worsens, some people begin to have abnormal blood vessels growing behind the macula.  These new blood vessels tend to be very fragile and often leak blood and fluid (hence ‘wet’ macular degeneration).  The blood and fluid raise the macula from its normal place at the back of the eye.  Damage to the macula occurs rapidly.  With wet AMD, loss of central vision can occur quickly.  Wet AMD is also known as advanced AMD.  It does not have stages like dry AMD.  The wet form is diagnosed in approximately 10 percent of cases; however, it results in 90% of the visual impairment and is considered advanced AMD (there is no early or intermediate stage of wet AMD).  Wet AMD is always preceded by the dry form of the disease.

 Regulatory Status: ACT is preparing its first IND aimed at the eye condition known as Age Related Macular Degeneration or AMD.  Based on its preclinical studies to date, ACT expects to file its initial IND application utilizing their Retinal Pigment Epithelium (“RPE”) Program for the treatment of macular degeneration in the fourth quarter of 2009.

  • To date, no adverse events have occurred in testing. The results will be part of the submission which Advanced Cell expects to submit to the FDA prior –hopefully – to the end of the year.

Financing: Funding (3/09) of $5 M credit facility was received; however, proceeds from the facility must be used “exclusively” for the development of ACT’s retinal pigment epithelium (RPE) cells program for the treatment of diseases of the eye.  ACT believes that the proceeds will be sufficient to file an IND for its RPE program and will complete both PI and II trials.  But the lack of capital market access has limited the beginnings of this next revolution to extent their development.

Bottom Line: ACTC.OB engages in the development and commercialization of human embryonic and adult stem cell technology in the field of regenerative medicine. Its stem cell-based therapy “could” provide treatment for an array of chronic degenerative diseases and in the regenerative repair of acute disease:

  • Age related macular degeneration (AMD) represents a tremendous market opportunity given the size of the patient population and the lack of treatment alternatives,
  • Reiterating, existing therapies “only” treat the condition, are not curative but palliative and do not regenerate cells of the macula that have been damaged.  Additionally, existing therapies only treat Wet AMD not Dry AMD hence leaving a significant unmet need for an effective treatment for the 90% of AMD patients who have the dry variety,
  • Shares of ACTC.OB began trading “again” on the OTC BB as ACTC.OB after up-dating its financial statements; the re-filing allowed a step up from the pink sheets,
  • Advanced Cell is truly a “high risk” investment due to the issue of access to capital but could be rewarding if results are justified by the IND as diseases of the eye affect more than 30 M people worldwide and represent a $20+B market,
  • The stock trades at $0.11 with outstanding shares listed at 499.91 M. The market cap is $54.99 M with an enterprise value of $90.45 M but their cash is listed at ONLY $689.15 K – WOW – do they need a reverse and further capitalization,
  • For additional disclosure purposes,  I certify this blog reflects my personal view with NO personal holdings (see disclosure section in Scimitar Equity site).  I know Bill Caldwell personally and do need to give him “credit” for his commitment to  keeping ACT alive through these tough times,
  • ACTC.OB also has such a “very” rich IP portfolio that “could” be monetized.