Altavant Sciences Initiates Study of Rodatristat Ethyl in Pulmonary Arterial Hypertension

Altavant has dosed the first patient in the ELEVATE 1 proof-of-concept Phase 2a study of rodatristat ethyl in patients with pulmonary arterial hypertension (PAH). The study is designed to provide safety, target engagement and biomarker information, which will be used to inform a comprehensive Phase 2b efficacy study of rodatristat ethyl in PAH.

"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," said William T. Symonds, Pharm.D., Chief Executive Officer of Altavant. "Rodatristat ethyl is a first-in-class investigational medicine for the treatment of PAH. 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 remodeling characteristic of the disease. Success in this study would take us one step closer to being able to provide a much-needed, new therapeutic option to PAH patients."

"While the introduction of vasodilators was a significant advancement in treating PAH, having improved exercise capacity, hemodynamics and quality of life for countless patients, there is still a need to develop novel treatment options to further improve lifespan. New R&D efforts, such as those pursued at Altavant, may ultimately give us an opportunity to offer a new treatment option to PAH patients that provides better outcomes. I'm excited to help further that R&D effort and elevate the conversation about PAH and the need for new therapies within the medical community," Marc Humbert, M.D., Ph.D., Professor of Respiratory Medicine at the South Paris University (Université Paris-Saclay), Director of the French Pulmonary Hypertension Reference Center, and member of Altavant's Scientific Advisory Board, said.

ELEVATE 1 is a double-blind, placebo-controlled, multicenter study to determine the safety, tolerability, pharmacokinetics (PK) and biomarker pharmacodynamics of rodatristat ethyl in adults with PAH. Approximately 36 adults with PAH will be enrolled in ELEVATE 1. Rodatristat ethyl's unique and complementary mechanism of action relative to available medications allows PAH patients currently taking vasodilators to be eligible for enrollment. Eligible patients will be randomized 2:1 to receive rodatristat ethyl or placebo twice daily for six weeks. Each patient will be assessed for primary and secondary endpoints of safety and PK. In addition, ELEVATE 1 will evaluate the effect of rodatristat ethyl on biomarkers of target engagement and on several exploratory efficacy measures, including exercise capacity, breathlessness and World Health Organization (WHO) functional class.

Rodatristat ethyl is a tryptophan hydroxylase (TPH) Inhibitor designed to reduce the body's peripheral production of serotonin. A significant body of scientific evidence supports dysregulated peripheral serotonin production as a trigger of aberrant proliferation and constriction of the smooth muscle cells in the wall of the pulmonary arteries, causing them to restrict blood flow in PAH. By lowering circulating serotonin levels, it is believed that rodatristat ethyl may halt or reverse the pathology of diseases that are driven by excessive serotonin production, such as PAH, idiopathic pulmonary fibrosis (IPF) and sarcoidosis.

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