Fledgling Vtesse grabs a 'breakthrough' title at FDA for rare disease drug

Vtesse CEO Ben Machielse

The biotech startup Vtesse is celebrating its first anniversary with a breakthrough therapy designation from the FDA.

The move--a rare win for a fledgling biotech--puts a spotlight on VTS-270, a treatment of Niemann-Pick Type C1 Disease that was hustled along by a virtual team and investigators at the NIH right into an ongoing Phase II/III pivotal study. And now it can proceed with assurances of an open-door policy from the agency as it helps accelerate the rare disease drug.

"This designation is supported by strong preclinical and early clinical data with VTS-270, including that from the Phase I study conducted by the National Institutes of Health," noted CEO Ben Machielse in a statement. "It is our hope that this designation will help to expedite the development and regulatory review process, getting the drug to patients who can benefit sooner."

Vtesse was launched out of the Cambridge, MA-based incubator Cydan in early 2015. And the same team that backed Cydan--NEA, Pfizer ($PFE), Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments--also came in to back a $25 million Series A round. Investigators are recruiting 51 patients for the pivotal study.

"This public/private model is pretty cool," Machielse told me a year ago. Vtesse has an office in Gaithersburg, MD, staying close to the investigators at the NIH who have been working with the drug, a formulation of 2-hydroxypropyl-beta-cyclodextrin designed to clear away the cholesterol that builds in the cells of patients suffering from Niemann-Pick disease.

Vtesse also announced that it has recruited Kevin Johnson as head of regulatory affairs. Johnson is a longtime regulatory veteran with a lengthy stint at GlaxoSmithKline ($GSK).

- here's the release