Introduction

Inflammation is an important component in the pathological evolution of cystic fibrosis. Anakinra (Kineret) is a potent anti-inflammatory drug administered subcutaneously, an inadequate modality for chronic treatment. The present invention is a novel anakinra inhalable dry powder for the treatment of inflammation in cystic fibrosis. The preparation proved higher stability, efficacy, and safety compared to systemic treatment in animal models.

Technical features

The new formulation is a dry powder of anakinra that is intended for pulmonary delivery via dry powder inhalers and is produced through a spray-drying method, using excipients able to guarantee the stability of the protein in the process phase and during storage. The preparation is presented as a fine powder which can be processed to be included in packaging forms suitable for the DPI method of administration. Anakinra when delivered at 10 mg/kg once every two days to the lungs showed an improved and extended therapeutic efficacy in CF models compared to once a day systemic delivery. Moreover, inhaled anakinra was still active at ¼ of starting dose. The protein is structurally preserved inside the formulation and retained its pharmacological activity in vitro immediately after preparation and over 4 months when stored at ambient conditions. Peripheral side effects (neutropenia) were not observed and correlated with the nearly 2-fold lower serum levels compared to systemic treatment. Currently the proposed technology has reached a TRL 3-4 proof-of-concept- validated in lab.

Possible Applications

• Repurposing of anakinra in Cystic Fibrosis as an anti-inflammatory drug;
• Treatment of chronic and acute inflammation.

Advantages

• Reduced dosage and systemic side effects;
• Higher compliance;
• Greater potency and efficacy;
• Prolonged effect;
• Possible reduction in the frequency of administration;
• Higher stability of the drug, storage in ambient conditions.