Vanda Pharmaceuticals Inc. (Vanda) announced results from its phase III clinical study, VP-VLY-686-3303, evaluating the efficacy and safety of tradipitant in treating the symptoms of gastroparesis. The study did not meet its prespecified primary endpoint which was the difference between drug and placebo on the change of the severity of nausea from baseline at week 12 of treatment. Both treatment arms showed significant improvements from baseline on nausea as well as the other core symptoms of gastroparesis.

Initial exploratory analysis has identified potential confounders that may have masked the beneficial effect of the drug previously observed in the phase II study of tradipitant, which include a baseline imbalance of rescue medication use between the two treatment arms as well as an observed poor compliance with study drug for some patients in the study. When restricting the analysis in the group of patients that used no rescue medications at baseline and adjusting for poor compliance, Vanda identified strong evidence of a drug effect across a number of symptoms and across the duration of the study, including a significant and meaningful effect at the prespecified primary endpoint of nausea change at week 12.

The phase III study also continued to demonstrate that tradipitant is safe and well-tolerated, as seen in previous studies over the 12 weeks of treatment. Patients on tradipitant experienced a similar number of treatment emerge common adverse event where tradipitant frequency was higher than placebo was diarrhoea. Patients that participated in the clinical program also had the opportunity to seek expanded access to tradipitant based on the benefit in the study and their individual unmet medical needs. Ten patients have received more than 3 months of tradipitant treatment, 6 of whom have received at least 1 year of tradipitant treatment.

"While disappointed that the study did not meet its prespecified outcome, we are encouraged by the evidence that is emerging from further analysis that confirms observations made in the prior clinical study," said Mihael H. Polymeropoulos, M.D., Vanda's president, CEO and chairman of the board. "We are committed to completing our analysis and working to bring tradipitant to patients with gastroparesis to fill a significant unmet medical need."

Vanda plans to continue the analysis of the data of this study and prepare the results for submission to peer review journals as well as prepare the data for submission to regulatory authorities.

Tradipitant is a neurokinin-1 receptor antagonist licensed by Vanda from Eli Lilly and company. Tradipitant is currently in clinical development for gastroparesis and motion sickness. The FDA has imposed a partial clinical hold on tradipitant clinical protocols of longer than 12 weeks duration.

Gastroparesis is a serious medical condition characterized by delayed gastric emptying associated with the symptoms of nausea, vomiting, bloating, fullness after meals and abdominal pain, along with significant impairment of social and occupational functioning. The estimated prevalence of gastroparesis in the US is approximately 6 million patients, many of whom remain undiagnosed. Gastroparesis affects mostly women and it can be of diabetic, idiopathic or other etiology. The only US Food and Drug Administration approved treatment for gastroparesis is metoclopramide, approved in 1979, which due to its potential of severe side effects carries a black box warning and limitations of use of no more than 3 months.

Patients are faced with limited therapeutic options and clinical guidelines recommend, in addition to metoclopramide, the off label use of different drugs including erythromycin, domperidone (not approved in the US), botulinum toxin injections, gastric stimulators and a variety of surgical procedures in an effort to relieve even temporarily some of the symptoms of the disease. Gastroparesis treatment represents a significant unmet medical need as underscored by the testimonies of interested parties and advocacy organizations including the International Foundation for Gastrointestinal Disorders (IFFGD) and Gastroparesis Patient Association for Cures and Treatments, Inc. (G-Pact).

Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients.