(a Californian corporation)
RadMit Holding, Inc. RadMit Holding, Inc., a California corporation (“RadMit Holding” or the “Company”) seeks to be actively engaged in the business of development and licensing of certain proprietary pharmaceuticals. RadMit Holding focuses on the development of small molecule drugs to treat genomic instabilities by boosting cellular DNA repair capabilities. RadMit Holding’s products will target disease indications for which there are currently no or only unsatisfactory treatment alternatives. The Company will initially focus on the development of treatments for the genetic instability disease Ataxia Telangiectasia (AT) also called Lois-Barr Syndrome. RadMit Holding’s small molecules could also be indicated for treatment of other genetic instability diseases. In the future, RadMit Holding’s small molecules could also be applied for the treatment of other rare diseases such as Xeroderma pigmentosum, Cockayne’s syndrome (CS) or Trichothiodystrophy, Werner’s syndrome, Bloom’s Syndrome, NBS Nijmegen Breakage Syndrome, FA Fanconi’s Anemia, Li Fraumeni Syndrome, A-T-like disorder (ATLD), Rothmund-Thompson Syndrome, ATR-Seckel syndrome, LIG4 Syndrome Ligase IV, Human immunodeficiency with microcephaly SCID, Spinocerebellar ataxia with axonal neuropathy (SCAN1), Ataxia with oculomotor apraxia 1 (AOA1), Ataxia with oculomotor apraxia 2 (AOA2), DBA Diamond-Blackfan anemia, Rapadilino syndrome, Turcot Syndrome, Seckle Syndrome, LS Lynch syndrome, and RIDDLE Syndrome.
Intellectual Property The Regents of the University of California have filed Patent Application No. USSN 60/940,633 for the proprietary DEL assay to come up with the chemicals, which was allowed in the United States on November 7, 2014, and International Patent Application No PCT/US2011/046451 that covers the molecular structure of the Yel002 artificial chemical compound. Another application, specifically for treatment of genetic instability diseases has been filed internationally as PCT/US14/25929. The Company acquired a worldwide exclusive license to the patent applications claiming the lead substances for the mitigation and treatment of symptoms of genomic instability disorders as well as the test system to discover such substances. The Company will also have an option to gain a worldwide exclusive license for any additional substances identified by the Company’s proprietary screening tool originating from the lab at UCLA. During the next 3 years, RadMit Holding plans to complete studies on the mode of action of the identified small molecules. In addition, RadMit Holding will identify one clinical candidate and complete the preclinical development of these candidates. Commercial launch is expected in 2020 — 2021 with patents due to expire in 2036-2039. BCN Biosciences in Pasadena, CA has the license for Yel002 as a mitigation agent after accidental ionizing radiation exposure to counter acute radiation syndrome (ARS) in the United States. BCN057, also known as YEL002, is a novel small molecule drug that has been found to protect normal tissue against radiation damage. This molecule is being developed under a $4 million cooperative agreement with the Biomedical Advanced Research and Development Authority (BARDA) for proof-of-concept studies. This molecule is currently in the preclinical in life phase. RadMit Holding has the rights for acute radiation exposure outside the United States as well as all the other applications worldwide including the United States. RadMit Holding and BCN Biologicals have an agreement to exchange data to eliminate duplication of effort. So far BCN Biologicals has synthesized the compound and conducted pharmacokinetics.
Market Potential Ataxia Telangiectasia is a devastating genetic disease that presents in the first decade of life. AT is a multi-symptom disorder characterized by growth retardation, oculocutaneous telangiectasias, motor dysfunction, endocrine dysfunction, gonadal abnormalities, immunodeficiency, extremely high cancer risk (up to 1,000 fold higher) and hypersensitivity to radiation. There is no available cure or treatment for the disease and death occurs from progressive malignancy or neurological degeneration typically in the second or third decade of life. Due to the medical need for AT and the lack of alternative treatments, the Company expects that it could generate annual sales with its respective product of $380 – 550 Million based on annual treatment cost of $90,000 per patient and treatment of 25% to 35% of homozygous AT patients in Europe and United States. If the compounds can also demonstrate efficacy in heterozygous patients and/or other genetic instability and DNA repair deficient diseases, the market potential could be significantly larger. About 0.5% to 1% of the total population are heterozygous carriers of the mutation with 50% increased risk of breast cancer. If Yel002 treatment can significantly reduce cancer frequency, it would add another 3-6 Million potential patients as an off-label prescription.
Technology The Company has invented a proprietary screening process at the University of California Los Angeles and screened more than 16,000 small molecule compounds for efficacy to boost DNA repair proteins after radiation exposure as a proxy for genomic instability disease. This screening resulted in the identification of Yel002 and CJ010. These two lead substances have been evaluated to assess preliminary safety and efficacy in boosting DNA repair proteins. Based on these pre-clinical results, the Company believes that its lead substances are ideally suited for the treatment of Ataxia Telangiectasia (AT) and other genetic instability diseases with the aim to slow if not halt the disease progression, reduce cancer risk and extend life. AT homozygous prevalence ranges between 1 in 100,000 and 1 in 40,000, representing 6,500-16,000 patients in the United States and Euro zone. AT heterozygous prevalence is estimated to be 0.5-1% of the population representing 3-6 Million patients. During the next 3 years, RadMit Holding plans to complete studies on the mode of action of the identified small molecules. In addition, RadMit Holding will identify one clinical candidate and complete the preclinical development. Research will be conducted in Austria, UCLA, Los Angeles and/or Charles River.