Therapy Areas: Autoimmune
First Patient Dosed in Pivotal Phase 3 Autoimmune Pulmonary Alveolar Proteinosis Trial
30 June 2021 - - The first patient has been dosed in the pivotal IMPALA-2 clinical trial, US-based biopharmaceutical company Savara Inc. (NASDAQ: SVRA) said.

IMPALA-2 is a Phase 3 trial designed to evaluate the efficacy and safety of molgramostim compared to placebo.

Molgramostim is an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF).

The trial is expected to be conducted at ~50 sites across the US, Canada, Japan, South Korea, and various countries in Europe and is anticipated to enroll ~160 patients with aPAP.

Initiation of IMPALA-2 is based on results from the Phase 2/3 IMPALA clinical trial which were published in the New England Journal of Medicine in September 2020.

While the IMPALA trial did not meet the primary endpoint of alveolar-arterial oxygen gradient (A-aDO2), the totality of data showed that multiple key secondary and exploratory endpoints either achieved nominal statistical significance or trended in favor of the active drug arms, and results from the open-label period demonstrated a sustained treatment effect, or continued improvement, after longer term exposure to molgramostim.

In December 2019, the US Food and Drug Administration granted Breakthrough Therapy Designation for molgramostim in aPAP based on data from the double-blind treatment period of IMPALA.

IMPALA-2 is a Phase 3, 48-week, randomized, double-blind, placebo-controlled clinical trial designed to compare the efficacy and safety of molgramostim 300 mcg administered once daily by inhalation with matching placebo in patients with aPAP.

The primary efficacy variable is change from baseline in % predicted diffusing capacity for carbon monoxide, a gas exchange measure.

Three secondary efficacy variables evaluate clinical measures of direct patient benefit: St. George's Respiratory Questionnaire Total Score, SGRQ Activity Component Score, and exercise capacity using a treadmill test.

The primary time point for efficacy assessment will be at week 24, however, efficacy will be assessed through week 48 to show durability of effect. Safety will be assessed through week 48.

Following the 48-week double-blind treatment period, patients will roll-over to a 48-week open-label period and will receive molgramostim 300 mcg administered once daily.

Savara is a clinical stage biopharmaceutical company focused on rare respiratory diseases.

Its lead program, molgramostim nebulizer solution, is an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis.


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