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The Pitch
NexGenix Pharmaceuticals, LLC Anjani Shah, Ph.D. 5/7/2004









Text Box: NexGen ix
Pharmaceuticals, LLC
Quick Facts:
Headquarters:
1 52 West 57th Street
Suite 11 B
New York, New York
10019
Founded:
June, 2003
Laboratory:
849 Mitten Road
Suite 104
Burlingame, CA 9401 0
Founder & CEO:
Allan E. Rubenstein, M.D.
Business Model:

NexGenix, with a spe­cialty pharmaceutical approach, is partnering with companies that have promising com­pounds for treating NF. “We have assembled, in one location, all the tools necessary to take a compound through pre-clinical screening and phase II clinical trials,” explained Laura Philips, Ph.D., MBA, COO of NexGenix. “We are pursuing intellectual rights to compounds and treatment approaches which will establish a unique market position for NexGenix in drug development for NF.”

Text Box: The Company:
Manhattan-based NexGenix Pharmaceuticals was founded in June 2003 by Allan E. Rubenstein, MD, who is also CEO of the company. NexGenix is the first and only company to focus on developing treatments for neurofibromatosis.
Neurofibromatosis (NF) is a genetic disorder character¬ized by the growth of tumors along nerves. The only treatment for these disfiguring and painful growths is surgical removal. Unfortunately, the tumors grow back. Many anti-cancer agents could be useful for NF because they target the came biological pathways that have gone awry in NF. Indeed, a few cancer-fighting compounds are already in clini¬cal trials for treating NF.
Because NF is an orphan disor¬der (affects fewer than 200,000 people in the U.S.) the market for a new drug has not been big enough to attract the attention of larger drug companies. However, NF is the largest of the 6,000 orphan-designated disorders, affecting almost 100,000 people in the U.S. alone — more than muscular dystrophy, Huntington’s and cystic fibrosis combined.
Under U.S. and European orphan drug legislation, NF represents a mar­ket of over $1 billion per year. This may not be big enough for a large

pharmaceutical company, but it certainly fits the business model of a smaller-sized, more efficient company that does not have high drug discovery costs and risks.

Technology:

NF1 and NF2 are caused by tumor suppressor mutations in either the NF1 or NF2 gene. Based on a detailed understanding of the genetics, NexGenix has a wide array of cell-based assays and sophis­ticated transgenic mouse models for preclinical screening. They also have proprietary imaging tools and a biomarker under development to detect clinical endpoints and to accelerate the process of getting a drug to patients. Laboratory work is performed at NexGenix’s research facility in Burlingame, California.

NexGenix has 5 prominent cancer researchers as founder-scientific advisors, including Frank McCormick, Ph.D, Head of the UCSF Cancer Research Center. Rubenstein himself is Director of Medical Affairs Emeritus at the National NF Foundation.

Prospects:

The company has raised $1.5 million from angel investors and will shortly seek an additional $1.5 million. The additional funding will expand the throughput capabilities to screen more compounds, enhance the capabilities for performing experiments with mouse models and build on existing tools for clinical trials.

Text Box:  Anjani Shah, Ph.D. is a freelance science writer based in New York City.


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