Mast Therapeutics, Inc. MSTX today reported top-line results from a Phase 2 study of AIR001 (sodium
nitrite) inhalation solution for the treatment of pulmonary arterial
hypertension (PAH). Mast obtained the rights to the AIR001 program through
its acquisition of privately-held Aires Pharmaceuticals, Inc. earlier this
year. In the primary efficacy analysis of the Phase 2 study, all doses showed
improvement in median pulmonary vascular resistance (PVR). In the secondary
efficacy analysis, all doses showed improvements in the median distances
obtained in the 6-minute walk test, including clinically-meaningful
improvements at the highest dose level. Additionally, AIR001 was
well-tolerated, with no treatment related serious adverse events. In
particular, methemoglobin levels remained normal (< 1.5%), which distinguishes
AIR001 from safety concerns associated with intravenously-administered
nitrite.
Edwin L. Parsley, D.O., interim Chief Medical Officer, stated: "These results
are promising and consistent with earlier findings of AIR001 as an agent that
can have a positive effect on hemodynamic parameters in a PH population. To
date, more than 120 individuals have received AIR001, including patients who
have received repeat administration for as long as 12 months, patients who
were treatment naive, and patients on PAH disease-specific background
therapy. Given the hemodynamic improvements observed, we feel AIR001 may be
uniquely suited to address the serious unmet need facing the large number of
patients with pulmonary hypertension associated with left heart
disease. Consequently, we will be pursuing clinical development of AIR001 in
that indication and plan to support multiple, institution-sponsored Phase 2a
studies that will evaluate 1) acute hemodynamic effects, 2) acute effects
versus placebo on maximum oxygen consumption and exercise hemodynamics, and
3) inhaled versus intravenous administration of nitrite, as well as the safety
of multiple doses of AIR001, in patients with PH associated with left heart
disease."
Brian M. Culley, Chief Executive Officer, stated: "We are encouraged by the
results seen in the Phase 2 study of AIR001 and believe they further validate
our acquisition of Aires. The data from the study show benefits consistent
with prior studies and support further development of AIR001. We look forward
to proceeding with the Phase 2a studies in PH associated with left heart
disease and anticipate reporting preliminary study results as early as the
second half of 2015."
About the Phase 2 Study (AIR001-CS05)
The Phase 2 study was a multi-center, open-label, randomized, parallel-dose
study to determine the safety and efficacy of AIR001 in subjects with PAH.
Subjects were randomized into one of three treatment arms and treated with
AIR001 for 16 weeks: 80 mg once daily after a 2-week "run-in" period of 46 mg
once daily; 46 mg four times daily after a 2-week run-in period of 46 mg four
times daily; or 80 mg four times daily after a 2-week run-in period of 46 mg
four times daily. The primary objective of the study was to evaluate the
efficacy of inhaled nebulized AIR001 as determined by change in pulmonary
vascular resistance (PVR) from baseline to week 16, measured immediately
post-completion of AIR001 nebulization. Secondary endpoints included change
from baseline to week 16 in: 6‑Minute Walk Distance (6MWD) assessed
immediately post-completion of AIR001 nebulization (peak), but no more than 40
minutes after completion of AIR001 nebulization; hemodynamic measurements of
cardiac output, mean right atrial pressure and pulmonary capillary wedge
pressure at peak; N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP);
hemodynamics and 6MWD at trough; and quality of life measures.
The study was powered to enroll 90 patients, however, prior to its acquisition
by Mast, Aires discontinued the study due to capital constraints. Data is
available from 29 patients who enrolled in the study.
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