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  1. RBx
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    New blog from CEO:

    CEO Update - 16 September 2014

    First Pediatric Drug Voucher Sells for $67.5M

    Dear Shareholders

    In 2012, the US Congress passed the Creating Hope Act. Its purpose is to encourage investment in the development of drugs for childhood diseases.

    Childhood diseases, particularly childhood cancers, have been severely neglected by drug developers. Only 1 anti-cancer drug has been developed specifically for children. The bulk of anti-cancer drugs used in children were developed for adults, and their use in children generally is considered to be sub-optimal.

    The Creating Hope Act is an attempt to do something about that situation. Under the Act, any company that committed to developing a drug for any rare pediatric disease, including pediatric cancer, would receive a voucher that the company could use or sell. The voucher would entitle the holder to receive expedited review of another drug by the FDA. The theory was that the voucher could be valuable to a company wanting to bring another drug to market more quickly. Under the program, a company or institution that develops a drug for a rare pediatric disease and receives FDA approval for that drug, receives a voucher.

    On the 30th July this year, the theory was proven to work, when US biotech company, BioMarin, sold the first priority review voucher to fellow US biotech, Regeneron, for $67.5M. BioMarin received the voucher for the drug Vimizin, that it developed to treat a rare but fatal childhood disease, Morquoi A Syndrome. Regeneron bought the voucher to expedite the marketing approval of an anti-cholesterol drug.
    The relevance of this to Novogen is that we are working to bring a range of drugs into the clinic for pediatric use.

    The first is Trilexium, which is being developed for treatment of neural cancers, with primary brain cancer and neuroblastoma in children going to be the subject of clinical trialing in 2015.

    The second is our ATM technology, which also is intended to be used in conjunction with Trilexium to treat neuroblastoma, again looking to be in the clinic in 2015.

    The third is our Project Jacob Hope program looking at the development of super-benzopyran drugs to treat a range of pediatric genetic conditions including San Filippo Syndrome and infantile neuraxonal dystrophy.
    This makes Novogen one of the largest developers in the world of drugs for pediatric diseases.

    It is important to point out that the voucher system is not the driver of our interest in the pediatric field. We are in that space because our two drug technology platforms have given us the opportunity to do so. The vouchers are a wonderful side benefit.

    There appears to be no limit to the number of vouchers that any one company can apply for. That is a function of the number of specific pediatric clinical indications being pursued, which in our case is at least 7 (medulloblastoma, diffuse intrinsic pontine glioma, neuroblastoma, infantile neuraxonal dystrophy, facioscapuloumeral muscular dystrophy, San Filippo Syndrome).

    Graham Kelly
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