The proof-of-concept Phase IIa study has been designed to provide safety, target engagement, and biomarker information, which will be used to perform a comprehensive Phase IIb efficacy study of the candidate.
The double-blind, placebo-controlled, multi-centre ELEVATE 1 trial will determine the safety, tolerability, pharmacokinetics (PK), and biomarker pharmacodynamics of rodatristat ethyl.
Altavant will enrol about 36 adults with PAH in the ELEVATE 1 trial. Eligible subjects will be randomised 2:1 to receive rodatristat ethyl or placebo twice daily for six weeks and will be evaluated for primary and secondary endpoints of safety and PK.
ELEVATE 1 will also evaluate the effect of the candidate on biomarkers of target engagement and on several exploratory efficacy measures.
Altavant Sciences CEO William Symonds said: “Initiating the ELEVATE 1 study is a significant milestone for Altavant, and represents our commitment to the rare respiratory disease community and our approach to patient-centric, transparent clinical development.
“Our goal for ELEVATE 1 is to establish proof-of-concept for this new mechanism in PAH, which we believe could halt or reverse the pulmonary vascular remodelling characteristic of the disease.”
Tryptophan hydroxylase (TPH) inhibitor rodatristat ethyl has been designed to reduce the body’s peripheral production of serotonin.
It is capable of lowering circulating serotonin levels and may halt or reverse the pathology of diseases driven by excessive serotonin production, such as PAH, idiopathic pulmonary fibrosis (IPF), and sarcoidosis.