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Original Research: CYSTIC FIBROSIS |

Inhalation of Moli1901 in Patients With Cystic Fibrosis*

Hartmut Grasemann, MD; Florian Stehling, MD; Helmut Brunar, PhD; Rudolf Widmann, PhD; Terry W. Laliberte, BSc; Luis Molina, PhD; Gerd Döring, PhD; Felix Ratjen, MD
Author and Funding Information

*From the Children’s Hospital (Drs. Grasemann, Stehling, and Ratjen), University of Duisburg-Essen, Essen, Germany; AOP Orphan Pharmaceuticals AG (Drs. Brunar and Widmann), Vienna, Austria; Lantibio Inc (Ms. Laliberte and Dr. Molina), Chapel Hill, NC; and the Institute of General and Environmental Hygiene (Dr. Döring), University of Tübingen, Tübingen, Germany.

Correspondence to: Hartmut Grasemann, MD, The Hospital for Sick Children, 555 University Ave, Toronto, ON, Canada M5G 1X8, e-mail: hartmut.grasemann@sickkids.ca



Chest. 2007;131(5):1461-1466. doi:10.1378/chest.06-2085
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Background: In cystic fibrosis (CF) patients, the absence or dysfunction of the chloride channel CF transmembrane conductance regulator (CFTR) results in reduced chloride ion transport in respiratory epithelial cells. Moli1901 stimulates an alternative chloride channel and may thus compensate for the CFTR deficiency in the airway epithelium of CF patients.

Methods: A phase II, placebo-controlled, double-blinded, single-center, multiple (5 consecutive days), rising-dose (daily dose, 0.5, 1.5, or 2.5 mg of Moli1901) study was conducted to investigate the safety and tolerability of multiple doses of aerosolized inhaled Moli1901 in 24 patients with CF and stable lung disease.

Results: Moli1901 was well tolerated in all but one CF patient, in whom a transient significant decrease in FEV1 developed following inhalation, which resolved spontaneously, and in a second patient in whom transient throat numbness developed during drug inhalation. A significant improvement of FEV1 was observed in the group receiving treatment with 2.5 mg/d Moli1901 compared to the group receiving placebo (p = 0.01 [Wilcoxon test]). Moli1901 was not detected in the plasma of the highest dose group.

Conclusions: The inhalation of Moli1901 up to a total cumulative dose of 12.5 mg appears to be safe in adult patients with CF. In addition, Moli1901 had a sustained beneficial effect on pulmonary function, which supports further studies of its efficacy in CF patients.

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