January 7, 2015
By Riley McDermid, BioSpace.com Breaking News Sr. Editor
A small biotech in Gaithersburg, Md., Vtesse Inc., has secured a major deal with the National Institutes of Health to license its version of the drug cyclodextrin to the NIH’s National Center for Advancing Translational Sciences, which will use it to develop a treatment for rare Niemann-Pick Type C disease.
Under the terms of the deal, Vtesse has raised $25 million from investors including Pfizer Venture Investments, Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments. That money will be used to bankroll a NIH-led cyclodextrin trial and Vtesse will support NIH researchers as they launch an even larger trial in 2015.
Niemann-Pick Type C, or NPC, is a fatal cholesterol metabolism disorder, in which patients are not able to metabolize cholesterol and other lipids properly within the cell. Consequently, excessive amounts of cholesterol accumulate within the liver and spleen and excessive amounts of other lipids accumulate in the brain. In most cases, neurological symptoms begin appearing between the ages of four and 10. Type C Niemann-Pick Disease has an estimated 500 cases diagnosed worldwide, said the NIH.
The NIH is already familiar with cyclodextrin, which it used in a clinical trial treating children with NPC two years ago. But that version of the drug was made by Johnson & Johnson subsidiary Janssen Research & Development, which now provides the drug free to families with children who have the disease, after a massive lobbying effort from parents paid off. It also provides the drug to Vtesse.
But the NIH is now mostly interested in Vtesse‘s lead compound, VTS-270, which has shown promise in pre-clinical and clinical studies as a potential treatment NPC. The drug is a mixture of (2-hydroxypropyl)-beta-cyclodextrin and has undergone studies at NCATS, NICHD and the NIH Clinical Center, as well as under individual compassionate use investigational new drug applications (iINDs) and in other academic labs. It now hopes to test the drug in a second trial that will enroll 50 to 75 patients in the U.S. and Europe.
“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, president and chief executive Officer 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.”
So far, the NIH estimates it has spent $15 million to $20 million on its cyclodextrin research, including money for regulatory guidelines required by the U.S. Food and Drug Administration, toxicology data and other aspects of rolling out a drug.
The original cyclodextrin trial was the subject of the well-known and closely followed Wall Street Journal’s “Trials” series. In that chronicling, the paper questioned whether the NIH should be developing drugs the way the way the private sector does, and today’s Vtesse deal is likely to renew that debate.
“The primary outcome here is very unusual for most NIH projects because it is not a paper or a publication, it is a license,” Christopher Austin, director of NCATS, told the Wall Street Journal on Wednesday.
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