Vtesse completes dose-finding part of phase 2b/3 trial for VTS-270 to treat Niemann-Pick Type C1 disease

Vtesse’s ongoing Phase 2b/3 prospective, randomized, double-blind, sham-controlled trial is a three-part, efficacy and safety trial of VTS-270, administered by the lumbar intrathecal (IT) route every two weeks.

VTS-270 is based on a specific, well-characterized composition of 2-hydroxypropyl-beta-cyclodextrin.

Twelve patients from the United States and the United Kingdom, spanning ages four to 21, made up the dose-finding portion of the Phase 2b/3 clinical trial. Based on all available data gathered from the first 12 patients’ experiences, an independent dose selection committee (DSC) recommended a dose of 900 mg to be administered every two weeks for the duration of the trial to each patient who is randomized for active treatment. This dose was selected for its appropriate balance of safety, tolerability, and potential for efficacy.

In addition to its use in the dose-finding phase, there has been significant experience with 900 mg in the Phase 1 clinical trial with VTS-270, further bolstering confidence in the DSC’s dose selection.

Vtesse also successfully expanded its clinical trial, which now includes ten study sites in the United States, the United Kingdom, Spain, France and Germany with ten additional sites expected to be initiated soon. Vtesse also anticipates expansion into Turkey and Australia based on discussions with local investigators and patient advocacy groups in those countries.

?"We are encouraged by the Phase 1/2 data with VTS-270 to date as well as by the substantial progress in our Phase 2b/3 trial. In selecting a dose and activating 10 clinical sites, we at Vtesse are demonstrating our commitment to rapidly advance this clinical trial and to make it as convenient as possible for patients and their families to participate," said Ben Machielse, Drs., Vtesse President & CEO.

"We are pleased that patients and their familes have expressed interest in enrolling in the second portion of this pivotal trial and we are working to fully enroll the remainder of the study in the next few months. With dose selection now complete, families of patients with NPC who are interested in participating should contact one of our clinical trial sites, as listed on www.theNPCstudy.com, to determine if they are eligible to participate in the remainder of the trial."

Drs. Machielse added, "The entire Vtesse team is exceptionally grateful to the patients, their parents and families, clinicians, and patient advocacy groups who participated in and helped execute the first part of our study. They have made the advancement of this pivotal trial a reality."

?In January 2016, Vtesse announced that the U.S. Food & Drug Administration (FDA) granted Breakthrough Therapy designation status for VTS-270 for the treatment of NPC.

Both the FDA and the European Medicines Agency (EMA) had previously granted Orphan Drug status to VTS-270. Vtesse’s clinical program is designed specifically for VTS-270, which has a unique, well-defined chemical fingerprint based on rigorous manufacturing controls and extensive (bio)chemical characterization.

The safety and tolerability of other cyclodextrin compositions, at the doses being studied for VTS-270 and when delivered through the IT route of administration, cannot be assured and is not supported by the scientific evidence that has been developed for VTS-270.