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Jan 3, 2011

ACT Secures $25M and FDA Approval to Start Clinical Trials of ESC-Derived Therapy

ACT Secures $25M and FDA Approval to Start Clinical Trials of ESC-Derived Therapy

Firm starts 2011 almost debt-free and able to push on with clinical pipeline.[krishnacreations - Fotolia.com]

  • Cell therapies firm Advanced Cell Technology (ACT) secured a $25 million nonconvertible stock purchase agreement with Socius CG II, a subsidiary of Socius Capital Group. On closing the deal Socius purchased an initial 400 shares of ACT’ Series C preferred stock for an aggregate purchase price of $4 million.

    The firm separately announced FDA clearance to start a Phase I/II clinical trial with its human embryonic stem cell (hESC)-derived retinal pigment epithelium (RPE) therapy for the treatment of dry age-related macular degeneration (dry AMD). Regulatory green light for the dry AMD study follows FDA’s IND clearance in November 2010 for a Phase I/II trial with the RPE therapy in patients with the Stargardt’s macular dystrophy (SMD), one of the most common forms of juvenile macular degeneration.

    IND approval for the dry-AMD study means ACT has become the first company to receive FDA clearance for two hESC trials, claims interim chairman and CEO Gary Rabin.

    ACT will use the funds from the Socius agreement to start the RPE clinical studies. The prospective open-label Phase I/II trials will separately determine the safety and tolerability of the RPE cells following subretinal transplantation in patients with either advanced SMD, or dry AMD.

    The Socius deal follows the recent sale of 750 shares of ACT nonconvertible Series B preferred stock under a previously agreed $10 million preferred stock transaction with Optimus Life Sciences Capital Partners. The resulting cash influx has not only provided the funding necessary to push on with clinical trials, but means ACT will start 2011 almost debt-free. This is in stark contrast to the firm’s serious debt issues just a year ago, Rabin admits. “Less than one year ago, the company was significantly in debt and lacked the financial resources to take even one program through the clinic. Today we are essentially debt-free and have the financial ability to move both our promising programs through the clinic.”

    ACT is developing regenerative cell therapies using a combination of embryonic and adult stem cell technologies. The firm has three ongoing development projects. The clinical-stage myoblast stem cell therapy program is focused on the development of autologous therapies for cardiac diseases such as heart failure. Positive data have already been generated in Phase I clinical trials, and FDA clearance has been granted for the start of Phase II trials in chronic heart failure patients. The firm says the Phase I studies demonstrated positive results in terms of improving the lives of Stage III-IV patients. The studies also marked the ever randomized clinical trials using catheter-delivered muscle stem cells to treat congestive heart failure in the U.S., ACT notes.

    The firm’s RPE program is designed to generate therapies for treating ocular disorders including AMD. ACT claims it has already achieved a significant milestone in this field by successfully restoring visual function in rats through the implantation of RPE cells derived from human embryonic stem cells.

    Its preclinical hemangioblast cell program, meanwhile, is focused on exploiting this type of stem cell as a potential approach for the treatment of cardiovascular disease, stroke, and cancer. Hemangioblast cells have the capacity to differentiate into both hematopoietic and angiogenic cells, and in 2007 ACT published details of an efficient and reproducible method for generating large numbers of hemangioblasts from human embryonic stem cells using an in vitro differentiation system.

    In December 2010 ACT and Roslin Cells announced plans to work together to establish a bank of GMP-grade human embryonic stem cell (hESC) lines using ACT’s single-ceblastomere technique for deriving embryonic stem cells without damage to the embryo. The technology allows stem cells to be collected from human blastomeres using the single-cell biopsy technique developed for use in preimplantation genetic diagnosis. ACT claims cell lines produced using this technique appear to be identical to hESC lines derived from later stage embryos using techniques that destroy the embryo’s developmental potential.


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