NexGenix has an active drug discovery and drug screening program. Over the past three years, the Company has developed and assembled a unique, proprietary, NF-specific drug evaluation platform for both NF1 and NF2. This platform consists of some of the most technologically advanced screening tools for NF in the world and includes transgenic mouse models, cell and tissue based xenograft models, a tumor bank, biomarkers, tumor explant models, cell based assays and other enzymatic screens. NexGenix Pipeline NX101: Intralesional treatment for Dermal Neurofibromas in NF1 The Company’s lead compound, NX101, is a intralesional treatment for dermal neurofibromas in NF1. NX101 is a unique formulation representing a therapeutic switch of a generic drug that has been used systemically in the US for an unrelated indication for over 15 years. The drug has been used extensively, and it has a well known safety profile with minimal side effects. We anticipate that the time and expense needed to bring this treatment to market will be much less than that required for a new molecular entity. The Company has completed proof-of-principle clinical trials outside of the US, and expects to soon file an Orphan Drug Application and an IND Application with the US FDA for this product. The Company has filed US and worldwide patent applications for the use of this drug for the topical treatment of NF1 and related disorders. NX201: Systemic treatment for NF2 Our screening platform has helped the Company to identify a potential treatment for NF2, based on research performed at MIT by one of our scientific advisors, Dr. Tyler Jacks, which has identified a likely target for NF2. We have completed, through a collaboration with Stanford University, high-throughput screening on over 120,000 compounds against this target and we have identified several compound families as new drug candidates. We are currently evaluating the chemical structure-activity relationships between these new molecular entities and our target, with a goal of optimizing efficacy, tolerance and patentability. In addition to our in-house drug discovery efforts we utilize our proprietary screening platform to evaluate externally developed compounds that may have utility in NF. This provides us with access to a broader range of compounds, access to later stage compounds and exposure to co-development, licensing and partnering opportunities with a variety of academic and industrial sources. A number of such compounds potentially active against the putative NF2 target and/or related pathways are in varying stages of evaluation. NXD30001: Hsp90 Inhibitor for NF2 and Cancer NexGenix has identified the potential for use of heat shock protein 90 (Hsp90) inhibitors in the treatment of NF and subsequently in-licensed a novel, radicicol based Hsp90 inhibitor compound series from a French chemistry laboratory. These compounds show enhanced Hsp90 binding affinity, and potency in inhibiting cell growth in vitro in NF and in various cancer cell lines compared to the most extensively studied and validated Hsp90 inhibitors, geldanamycin-based 17AAG and 17DMAG. The company has begun to evaluate the efficacy of these Hsp90 inhibitors in xenografts of NF2 and Her2 positive breast cancer cell lines. Preliminary studies of the first of 9 compounds in the series have shown some promising results in terms of efficacy, but have also shown some tolerability issues that may be due to the preliminary formulation and IP mode of administration in the studies or due to the dosing schedule. We are currently focused on evaluating different formulations and modes of administration and have several additional animal studies planned to examine the effects of variations in dosing regimen. While there are several companies with Hsp90 inhibitors in development for cancer treatment, our compound series is, to our knowledge, the only radicicol-based Hsp90 inhibitor in development, and appears to have a unique binding site. We believe that these compounds may offer differences in selectivity, toxicity and degree of efficacy in various disease models that will differentiate our product. The peak sales for tumor treatment in NF2 in our target markets is projected to be approximately $375M. We plan to out-license the compounds for development for breast and other forms of cancer. According to Recombinant Capital, in 2006, the average oncology licensing deal totaled $185M in upfront fees, milestones, R&D support and equity, for products in the discovery to preclinical stage.
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