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NexGenix Pharmaceuticals will use our expertise in neurofibromatosis and core competencies in drug development to discover and develop drug therapies to address the unmet medical needs of NF while partnering strategically to maximally develop the therapeutic potential for our products in other indications.


Background
NexGenix Pharmaceuticals is a US-based company focused on drug development for Neurofibromatosis Types 1 and 2 and related sporadic tumors. Neurofibromatosis (NF) is an autosomal dominant genetic disorder characterized by the development of tumors along nerves. These slow growing tumors are resistant to radiation and chemotherapy and the current standard of care is repeated surgical removal. The company was formed to pursue the treatment of NF based on recent major advances in genetic and signal transduction research. By concentrating much of the world's basic science and clinical expertise on NF in one company, we are accelerating treatments to patients. NexGenix is the first and only company to focus on NF.

Opportunity
Neurofibromatosis Type 1 (NF1) and Neurofibromatosis Type 2 (NF2) represent the largest group of disorders for which there has been no focused drug development. The two forms of the disorder affect approximately 100,000 people in the US; more than muscular dystrophy, Huntington's and cystic fibrosis combined. This represents a potential market of over $500 Million per year under US and European Orphan Drug legislation, which provides seven (7-10) years of market exclusivity for disorders that fulfill certain incidence criteria. Both NF1 and NF2 are caused by mutations of tumor suppressor genes for which NexGenix has identified key intervention points.





Strategy
We have developed a unique approach that takes advantage of five critical components:

Accelerate Results through Integration
NexGenix represents a horizontal integration of all phases of drug development from cell to patient. The result is a constant interplay of ideas, theory and results that will allow us to refine processes, accelerate treatments and end suffering.

Apply the Power of Knowledge
Being the first and only company to exclusively focus on the development of treatments for NF has allowed NexGenix to enlist the support of the world's foremost experts in NF. Based on years of research experience and knowledge these experts have helped to focus our team and chart the course to achieve rapid results.

Develop Precise Screening Methodologies
NexGenix has assembled a number of screening processes and refined them into a unique screening program with cell-based assays, xenografts and transgenic mouse models. This has given us the ability to rapidly screen and identify promising compounds and therapeutic approaches, both for in-licensing and for internal drug development.

Partner Strategically
NexGenix targets strategic partners that have drugs of interest in their pipelines. We also recognize that treatments for NF may have the potential to treat other, more common conditions in which there is an unmet medical need for treatment. It is our intention to seek partnership and co-development arrangements for the clinical development, sales and marketing of products outside of our area of focus

Leverage Unique Resources
The Company has developed a clinical trial network involving several of the largest NF clinical populations in the US, Europe and South America. This network is an invaluable asset for recruiting patients for clinical trials and for eventual market access and rapid distribution of products.



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