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PARI Pharma Reports Successful Phase Ib Clinical Results for Inhaled Liposomal Cyclosporine A and Gains FDA Orphan Drug Designation for the Prevention and Treatment of Bronchiolitis Obliterans

MONTEREY, Calif., June 2 /PRNewswire/ -- This month's Journal of Aerosol Medicine and Pulmonary Drug Delivery features positive Phase Ib clinical trial results for PARI Pharma's L-CsA, inhaled liposomal cyclosporine A. In May, PARI Pharma received Orphan Drug Designation from the Food and Drug Administration for L-CsA, delivered via an Investigational eFlow Nebulizer System, for the prevention and treatment of bronchiolitis obliterans, a devastating, incurable disease of the small airways affecting approximately 60,000 patients worldwide.

"PARI's encouraging results show that it is feasible and well tolerated to deposit relevant amounts of L-CsA into transplanted lungs. This has the potential to prevent and treat bronchiolitis obliterans in a more efficient way compared to current oral standard treatment. Inhaled L-CsA has a favorable drug distribution to the target sites in the lung, which should lead to lower side effects than associated with systemic cyclosporine exposure," stated Prof. Jurgen Behr, head of the Division of Respiratory Disease at the Ludwig Maximilians University, Klinikum Grosshadern, Munich, Germany.

"The long-term survival rate for lung transplant patients could be greatly improved through an effective inhaled liposomal cyclosporine therapy. Using our proprietary liposomal technology we created an inhaled formulation that is free from irritating solvents and optimized for administration via an Investigational eFlow Nebulizer System without destroying the liposome," stated Dr. Manfred Keller, executive vice president and chief scientific officer of PARI Pharma GmbH. "We had a very encouraging pre-IND meeting with the FDA, and their recent Orphan Drug Designation for L-CsA, which follows a similar designation in Europe, encourages us to move forward to solve this unmet medical need."

PARI Pharma's Phase Ib clinical trial for L-CsA studied 12 lung transplant recipients, where five patients had double lung transplants and seven patients had single lung transplants. Patients were given a single dose application of 10mg or 20mg of liposomal L-CsA, which was well tolerated. Deposition data showed a peripheral lung deposition of 2mg for a nominal, 10mg dose that was nebulized in less than ten minutes. Once daily administration of a nominal dose of 10mg L-CsA with an Investigational eFlow Nebulizer System would give approximately 14 mg L-CsA/week. Mean values for C(max) and AUC(0-inf) were up to 20ng/mL and 110 h ng/mL, respectively. These results are significantly lower compared to oral therapy. In comparison, oral CsA (Neoral(R)) therapy in lung transplant patients leads to highly variable C(max) and AUC(0-12h) values of approximately 1,800ng/mL and 8,000 h ng/mL, respectively when given twice daily in a dose of up to 250mg [Knoop 2003]. L-CsA's favorable and significantly lower pharmacokinetic values were obtained for both dose strengths tested. Phase Ib study results for L-CsA are published in the June 2009 volume of the Journal of Aerosol Medicine and Pulmonary Drug Delivery [Behr et al., Vol. 22, No. 2, p.121-129].

About PARI Pharma's Inhaled L-CsA Formulation

PARI Pharma's L-CsA is a low-dose, inhaled, liposomal cyclosporine A formulation that aims to improve the pharmacological profile, the highly variable bioavailability, and the severe side effects of oral CsA. (Severe side effects of oral CsA include kidney and brain damage, cancer, and vulnerability to infections.) L-CsA is an investigational, proprietary, liposomal formulation of 10mg CsA/2.5mL for inhalation delivery via a customized Investigational eFlow Nebulizer System. The L-CsA formulation is based on an artificial lung surfactant carrier. It is free of any irritating organic solvents and consists of unilamellar liposomes of about 100nm. The unilamellar liposomes are obtained via a high-pressure homogenization process using standard pharmaceutical equipment followed by sterile filtration and lyophilization. The product has been scaled up to production batch sizes, and preliminary stability data indicate that a shelf life of more than 2 years at room temperature is feasible.

About the Investigational eFlow Nebulizer System and eFlow Technology

The Investigational eFlow Nebulizer System uses eFlow Technology to enable highly 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, products incorporating eFlow Technology reduce the burden of taking daily, inhaled treatments. The Investigational eFlow Nebulizer System and eFlow Technology are proprietary to PARI Pharma.

About Bronchiolitis Obliterans

According to the International Society of Heart and Lung Transplantation Registry, development of bronchiolitis obliterans is the single most important risk factor for five-year mortality among lung transplant patients. Bronchiolitis obliterans is an incurable small airways disease, which manifests as chronic allograft rejection and results in airflow obstruction. The disease is also a factor in other lung diseases such as collagen vascular diseases, inhalation of toxic fumes, and respiratory tract infections. Bronchiolitis obliterans affects approximately 60,000 patients worldwide, and once it develops, most patients die of respiratory failure within 5 years.

About PARI Pharma

PARI Pharma focuses on the development of aerosol delivery devices, drug formulations, and therapies for upper and lower respiratory indications. PARI Pharma partners with pharmaceutical companies to develop new or improved therapies with eFlow Technology and other advanced delivery platforms. Investigational eFlow Nebulizer Systems are optimized and customized per investigational drug product and are currently in clinical trials for cystic fibrosis, asthma, COPD, respiratory syncytial virus (RSV) infection, and treatments for lung transplant patients among other indications.

PARI Pharma provides comprehensive inhalation drug development, including nebulizer formulation development and optimization, analytics, aerosol characterization, clinical protocol development, and regulatory guidance, all in compliance with CMC/GCP guidelines. PARI Pharma has several clinical development programs ongoing, either partnered or on its own. Proprietary programs in different development stages will be offered for licensing partnerships.

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