$25-million launch for Vtesse

Company to work with NIH on drugs for Niemann-Pick disease and other life-threatening rare diseases

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GAITHERSBURG, Md.—Vtesse Inc., whose corporate name is a truncation of the French word “vitesse,” which means “speed,” will be gaining initial velocity thanks to a $25-million Series A financing from Cydan Development Inc. and others to focus on developing drugs for Niemann-Pick Disease Type C (NPC) and other severe diseases with great unmet need.  
Vtesse, a rare disease company focused initially on developing drugs for NPC, is the first spin-off company for Cydan, an orphan drug accelerator that shares with Vtesse the same syndicate of leading life-sciences investors that are committed to funding additional rare disease companies. New Enterprise Associates (NEA) led the Vtesse financing with participation from Pfizer Venture Investments, Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments.
Vtesse also announced that it has established a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the National Center for Advancing Translational Sciences (NCATS), both components of the U.S. National Institutes of Health (NIH). Vtesse and NCATS have also entered into a licensing agreement for the current rights held by NIH for the worldwide use of cyclodextrin, delta-tocopherol and derivatives of tocopherol, alone or in combination, for the treatment of lysosomal storage diseases (LSDs), including NPC. Regulatory orphan designations for the United States and European Union will also be transferred to Vtesse.
Vtesse will use the proceeds from its Series A financing to conduct a clinical program for VTS-270, a formulation of (2-hydroxypropyl)-beta-cyclodextrin, for NPC, and to discover and preclinically evaluate additional novel drugs for NPC and other LSDs.  
NPC is a genetic disease associated with mutations in the NPC1 and NPC2 genes. NPC affects an estimated one in 100,000 to 150,000 children and is often misdiagnosed and/or underdiagnosed. Affected patients are usually identified in early childhood with ataxia and exhibit progressive impairment of motor and intellectual function, and they often die before adulthood.  
Researchers in NCATS’ Therapeutics for Rare and Neglected Diseases (TRND) program developed cyclodextrin as part of a project focused on finding treatments for NPC. The goal of TRND projects is to develop therapeutic candidates through a strategy known as de-risking, which involves the preclinical development of therapeutics to the stage where they attract biotechnology and pharmaceutical companies such as Vtesse to invest in their further clinical development.
The NPC project is a collaboration among government, academic and industry researchers who worked with patient groups and the NPC community to advance knowledge of the disease. Before the TRND project, researchers had found that cyclodextrin showed promise as a treatment for NPC in studies of animal models. Additional NCATS efforts provided further evidence that cyclodextrin demonstrated potential as a treatment for NPC.
Through this agreement, Vtesse will also fund preclinical studies led by NCATS researchers to develop various types of the compound delta-tocopherol, a form of vitamin E, to target lysosomal storage disorders. The NCATS team will optimize the delta-tocopherol compounds for further testing as potential single treatments or as a combination therapy with cyclodextrin. Vtesse has exclusively licensed several NCATS patent applications specifically for their use in the treatment of LSDs.
“We’ve launched Vtesse to rapidly advance the clinical development of VTS-270, which we hope to make widely available to the many young patients suffering from the debilitating effects of NPC,” said Ben Machielse, Drs. [a Dutch honorific], president and CEO of Vtesse. “We expect to listen to and learn from the physicians, independent researchers, parents and patients who have worked tirelessly for many years to find a treatment for this devastating disorder. We are grateful for the work they and the NIH have conducted thus far, and we are committed to developing a broadly available treatment for NPC.”
“The launch of Vtesse is an important milestone for Cydan, as this new company holds great promise for patients and is an excellent example of collaboration among scientific, patient and investment stakeholders,” said Dr. Chris Adams, founder and CEO of Cydan and an independent member of the Vtesse board of directors. “Our goal at Cydan Development is to advance innovative treatments for patients with rare diseases by identifying promising assets, accelerating their preclinical and clinical development, selecting the right management team and, ultimately, creating more companies like Vtesse.”
Under the CRADA, Vtesse, NICHD and NCATS intend to collaborate to launch a second VTS-270 clinical study for the treatment of NPC as well as to develop other novel drugs (delta-tocopherol and combinations) for NPC and other LSDs. Under the terms of the agreement, NICHD, which is the current sponsor of a Phase 1 clinical trial of VTS-270 in patients with NPC, Type 1, will transfer its Investigational New Drug application to Vtesse, along with all background data on the program. Vtesse will be responsible for all further development of VTS-270 with the intent to seek marketing approval for the drug candidate from U.S. and European regulatory agencies.
“This is an excellent example of how launching a project to study the underlying biology of one disease can lead to advances that hold promise for an entire group of diseases—the NCATS goal of finding what is common among diseases and the translational science process,” said NCATS Director Dr. Christopher P. Austin. “I am grateful to all of the NPC patients, their families and patient support groups who have been equal partners in our efforts to find therapeutic solutions to these devastating disorders.”

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