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ALPHA 1 DEFICIENCY

Kamada's inhaled Alpha 1 Antitrypsin (Inhaled AAT) product is in advanced stages of clinical trial, under FDA and EU Orphan Drug Designation, for individuals with Alpha 1 Antitrypsin Deficiency.

Kamada is at the forefront of clinical research, with Inhaled AAT deficiency treatment in advanced clinical development: a pivotal Phase II/III clinical trial was completed in Europe and Canada, to treat Alpha-1 Antitrypsin Deficiency.

Although primary end-point of the study (time to first exacerbation) was not met, the updated results showed clinically and statistically significant improvements in lung function and complementary efficacy in the severity of the first exacerbation.

Safety data of inhaled AAT in this Phase 2/3 trial remains supportive and consistent with previous inhaled AAT studies conducted by Kamada, and continues to demonstrate a high safety and tolerability profile.

Kamada is currently in advanced discussions with the U.S. Food and Drug Administration (FDA) in order to secure the approval of an Investigational New Drug (IND) application this year to conduct a U.S. Phase 3 pivotal study of inhaled AAT for the treatment of AATD that would begin in 2018.  Kamada intends to utilize the data to be obtained from that pivotal study to submit the MAA (Marketing Authorization Application ) to the EMA. 

New Treatment Prospects for AATD Patients (Video)

Kamada hosted a panel discussion entitled, “New Treatment Prospects for AATD Patients: Results from a Phase 2/3 Inhaled AAT Trial,” which was chaired by Robert A. Sandhaus, Ph.D., M.D., FCCP, Founder and Director of the Alpha1- Antitrypsin Deficiency Program at National Jewish Health Hospital in Denver, Colorado, and the Clinical Director of the Alpha-1 Foundation. The panel included Key Opinion Leaders (KOLs) who specialize in treating patients with AATD, and was held during the American Thoracic Society (ATS) 2015 International Conference.

Link to full slide deck

Kamada's AAT-IH offers several significant advantages:

  • Topical delivery to the affected lung tissue
  •  According to a Phase II, lung deposition study, considerable amount of AAT is reaching the target organ in all study populations
  • Non invasive, doesn’t require hospital settings
  • User friendly and mobile inhalation device
  • Lower dose requirement gives potential to treat 3-4 times the number of patients, compared to AAT IV.
  • Designated orphan indications for AAT deficiency, CF, Bronchiectasis

The company is also exploring other potential indications for the use of its inhaled-AAT formulation:

Cystic Fibrosis

Kamada’s inhaled AAT has gained the status of an Orphan Drug for the treatment of CF both in Europe and in the US.

Completed phase II study with CF patients  showed high safety and tolerability profile sided to demonstration of significant decrease in lung inflammation which was reflected, among others in the drop of neutrophil elastase enzyme in the lungs of the CF patients, an enzyme that has a major affect in lung inflammation and disease progression as a result.

Kamada won IND (Investigational New Drug) approval from the FDA, for a phase II clinical trial in the US for the treatment of CF.

Bronchiectasis

Kamada also completed a Phase II trial with inhaled AAT for the treatment of Bronchiectasis. As in the CF trial, the safety profile was excellent and the drug demonstrated encouraging impact on the inflammatory process in the lungs.