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Summary of Objectives
Inhalational drug delivery to treat PAH is attractive due to enhanced pulmonary specificity
and reduced systemic adverse effects. LIQ861 is a dry powder formulation of Treprostinil
(TRE) designed to enhance deep-lung delivery and achieve higher tolerated dose levels
than current inhaled therapies. LIQ861’s uniform (1µm) particle size with a trefoil,
pollen-like shape, enables QID delivery of TRE doses in a convenient, palm-sized,
disposable dry powder inhaler (DPI). The primary objective of the INSPIRE study is
the safety and tolerability of LIQ861 in subjects transitioning from stable doses
of Tyvaso® or as combination therapy with ≤2 approved non-prostacyclin (PGI) oral PAH drugs.
The secondary objective is to evaluate the comparative bioavailability (BA) of TRE
between LIQ861 and Tyvaso®.
Methods
Target enrollment is 100 Functional Class (FC) II/III subjects with 18 subjects on
Tyvaso® included in a BA sub-study comparing TRE exposure in a one-directional crossover.
PGI-naïve subjects stable on ≤2 approved, non-PGI oral PAH therapies received LIQ861
at an initial dose of 25mcg QID, increasing in 25mcg increments weekly to tolerance
and symptom relief. Subjects transitioning from Tyvaso® received LIQ861 at an initial dose comparable to their Tyvaso® dose, with titration in 25mcg QID increments to tolerance and symptom relief.
Endpoints
The primary endpoint is the incidence of treatment-emergent Adverse Events and Serious
Adverse Events. Safety endpoints: • Changes from Baseline to Month 2 or Early Termination
in clinical laboratory, physical exam findings, and vital signs. • Proportion of patients
maintaining a stable clinical status from transition through Month 2. Stable clinical
status defined as: 1) LIQ861 treatment through at least 2 months beyond transition
from Tyvaso® and six-minute walk distance (6MWD) decrease no more than 10%, 2) no interruptions
to treatment totaling more than 7 days prior to the end of Month 2, and 3) no treatment
with any other PGI analogs or P`GI receptor agonists. Secondary endpoints: NYHA FC, N-terminal B-type natriuretic peptide and Quality of Life will be reported when
available. Analysis for the Primary Endpoint and BA sub-study is expected to be completed
in 2019.
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Copyright
© 2019 Published by Elsevier Inc.
