Vtesse is a Gaithersburg, Md.-based developer of drugs intended to benefit patients with extremely rare, life‑threatening diseases. Its lead product, VTS-270, is a well‑characterized mixture of HPbCD with a specific compositional fingerprint aimed at treating Niemann‑Pick disease type C (NPC). The company is working collaboratively with the NIH, other academic centers, parents, and patient advocacy groups to advance a pivotal clinical study of VTS-270. Vtesse is also conducting preclinical discovery and development of other novel drugs for NPC and other lysosomal storage diseases. In January 2016 the FDA granted Breakthrough Therapy designation for VTS-270, and both the FDA and EMA had previously granted Orphan Drug status. The company is led by President and CEO Ben Machielse. Financially, Vtesse has raised a total of $42M to date. Vtesse is focused on developing drugs for Niemann-Pick Disease Type C (NPC) and other severe lysosomal storage diseases. Its lead program is VTS-270, a formulation of (2-hydroxypropyl)-beta-cyclodextrin, which the company plans to advance in clinical studies. Vtesse intends to use proceeds from its recent financing to conduct a clinical program for VTS-270 and to discover and pre-clinically evaluate additional novel drugs for NPC and other LSDs. The company has established a Cooperative Research and Development Agreement (CRADA) with NICHD and NCATS and is collaborating with the NIH to advance clinical study of VTS-270. Vtesse and NCATS have entered a licensing agreement for NIH-held worldwide rights to cyclodextrin, delta-tocopherol, and tocopherol derivatives for treatment of LSDs, and regulatory orphan designations for the U.S. and EU will be transferred to Vtesse. The company is led by President and CEO Ben Machielse and is a spin-out from Cydan Development.

Total raised

$42M

Funding rounds

2

Latest round

Series A

Latest activity

Jul 2016