January 18, 2016
By Alex Keown, BioSpace.com Breaking News Staff
SAN FRANCISCO – The annual J.P. Morgan Healthcare conference draws biotech watchers and investors from all corners of the globe looking for the next big blockbuster treatment. Last week Vtesse, a company launched one year ago, was on hand to show off the enormous strides it has made over the past 52 weeks.
Although Maryland-based Vtesse executives did not present at the J.P. Morgan Healthcare Conference, company officers took informal meetings throughout the week to talk with potential investors about the strides the company has made in one-year’s time.
“The J.P. Morgan conference is a good opportunity to put the company on the map,” Ben Machielse, president and chief executive Officer of Vtesse, said in an exclusive interview with BioSpace from the conference.
Machielse praised the Vtesse team for the past year’s progress. He said the company was in a “fortunate position to set up a licensing deal with NIH that gave us the rights” to a product. He said the Phase I trials were very encouraging and its Breakthrough Therapy status designation was a validation of the treatment’s potential.
Machielse said company’s philosophy has been to set realistic goals and then “over deliver and under promise,” which he said is keeping investors happy.
Since its January 2015 launch, Vtesse has had a banner year. The company came out of the gates with a deal with the NIH to license its version of the drug cyclodextrin to the NIH’s National Center for Advancing Translational Sciences, to be used for the development of a treatment for rare Niemann-Pick Type C (NPC) disease. Additionally, Vtesse completed a $25 Million Series A financing led by New Enterprise Associates; received orphan drug designation for its lead drug candidate, VTS-270 from both the U.S. and E.U. regulatory agencies; and began enrolling patients in a Phase IIb/III trial for VTS-270. A version of cyclodextrin, which was developed by Janssen, a division of Johnson & Johnson , was used in a clinical trial treating children with NPC two years ago.
VTS-270 has shown promise in preclinical and clinical studies as a potential treatment for NPC. The drug is a mixture of (2-hydroxypropyl)-beta-cyclodextrin. Machielse said the drug is able to capture the buildup of toxic cholesterol that occurs in NPC patients and removes it from the body.
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. In addition, excessive amounts of other lipids accumulate in the brain. In most cases, neurological symptoms begin appearing between the ages of four and 10. NPC has an estimated 500 cases diagnosed worldwide, according to the NIH. In the European Union, Actelion Pharmaceuticals ’ Zavesca (miglustat) has been approved for the treatment of progressive neurological manifestations of NPC in both adult and pediatric patients. Vtesse’s VTS-270 would be the first U.S. treatment for NPC if the U.S. Food and Drug Administration approves it, Machielse said.
Machielse said he anticipates the latest trial for VTS-270 to begin producing data next year. He said the product has a strong foundation and early clinical models produced encouraging results, which has the company focused on a potential commercialization plan. However, Machielse said the Vtesse team is being careful not to get ahead of themselves.
“We’re not thinking about what’s next, we have to prove that our plan works,” Machielse said. “Our goal is to create meaningful solutions for patients.”
Vtesse is also not thinking about seeking additional funding any time soon. Machielse said the company currently has enough funding to complete the Phase IIb/III clinical trial.
