Kamada has announced today positive data from its Phase II study evaluating inhaled Alpha-1 Antitrypsin (AAT) delivered via an Investigational eFlow Nebulizer System (PARI Pharma GmbH), in the treatment of cystic fibrosis. The results demonstrate the product has an excellent safety profile and shows promising signs of efficacy, as indicated by a reduction in lung inflammation.
The Phase II trial was a double-blind, placebo controlled study, performed at the world renowned Cystic Fibrosis Center at the Hadassah University Hospital, Mount Scopus Jerusalem, Israel, led by Prof. Eitan Kerem, Head of the Department of Pediatrics and a world expert for Cystic Fibrosis. The trial was designed to evaluate safety of the inhaled product in cystic fibrosis patients and to assess its potential efficacy in decreasing lung inflammation. Study results revealed that daily inhalation of AAT for a period of 28 days reduced lung inflammation in comparison to placebo, as measured by neutrophil count and neutrophil elastase levels. There were no serious adverse events reported in either the active or the placebo group and no safety concerns were raised. This study was performed in accordance with clinical guidance received from the EMEA and under the ODD of the product.
Kamada plans to present the study data at an upcoming major medical meeting. In order to pursue clinical development of this product, Kamada will approach the regulatory authorities for guidance.
Kamada's Chief Executive Officer David Tsur said: "This is a breakthrough for Kamada. This product is the next generation of AAT treatment that may be used, subject to success of clinical trials and regulatory approval, as an effective treatment for CF patients, to improve their quality of life. We believe that the inhaled product will offer significant advantages to cystic fibrosis patients and we are very optimistic about its further development in this and in other respiratory patient populations."
Professor Eitan Kerem, the Principal Investigator in this study, said, "I would like to congratulate Kamada for successfully completing this trial. Kamada's inhaled version of AAT is an innovative approach for the treatment of chronic inflammatory processes, including cystic fibrosis. Interventions that reduce these destructive processes could potentially prevent degeneration of lung function and could have a positive impact on patient lives. I look forward to monitoring the product's progress in future clinical trials."
Kamada's Inhaled AAT, which utilizes an Investigational eFlow Nebulizer System (PARI Pharma GmbH), has been designated Orphan Drug, in both Europe and the U.S. for the treatment of cystic fibrosis and Alpha-1 Antitrypsin Deficiency as well as in the U.S. for the treatment of Bronchiectasis. This designation grants Kamada various benefits such as research fund support, tax incentives, reduced official fees and 7 to 12 years of exclusive distribution rights, if the company's product is first on the market.
Kamada's Inhaled AAT is delivered by the Investigational eFlow Nebulizer System (PARI Pharma GmbH). The Investigational eFlow Nebulizer System uses eFlow Technology to enable extremely efficient aerosolization of liquid medications via a vibrating, perforated membrane that includes thousands of small holes that produce the aerosol mist. Compared to other nebulization technologies, eFlow Technology produces aerosols with a very high density of active drug, a precisely defined droplet size, and a high proportion of respirable droplets delivered in the shortest possible period of time. Combined with its silent mode of operation, small size (it fits in the palm of your hand), light weight, and battery use, eFlow Technology reduces the burden of taking daily, inhaled treatments. The Investigational eFlow Nebulizer System and eFlow Technology are proprietary to PARI Pharma and can be optimized to specific drug formulations.
PARI Pharma focuses on the development of aerosol delivery devices and therapies. Based on PARI's 100-year history working with aerosols, PARI Pharma develops treatments for pulmonary and nasal administration optimized with advanced delivery technologies, such as eFlow technology. Online at PariPharma.com.