Kamada Ltd., a plasma-derived protein therapeutics company focused on orphan indications, has completed the pivotal phase II/III clinical trial in Europe and Canada of the company’s proprietary inhaled Alpha-1 Antitrypsin (AAT) therapy for the treatment of Alpha-1 Antitrypsin Deficiency (AATD or Inherited Emphysema). Kamada expects to report top-line results from this study in the first quarter of 2014.

The multicentre randomized, double-blind, placebo-controlled study is evaluating the safety and efficacy of Kamada’s inhaled formulation of human AAT to treat AATD in >160 patients. The study involved the inhalation of 160 mg of human AAT or placebo twice daily via the eFlow device for 50 weeks, and eligible patients were given the option to participate in a 50-week open-label extension study. The primary endpoint of the study is the difference in exacerbation events between the two groups at one year and is 80 per cent powered to demonstrate a 20 per cent difference. Power is based on the number of events collected during the study. Secondary endpoints include additional parameters of exacerbation events, pulmonary function tests and safety. Additional exploratory endpoints include CT densitometry in subset of subjects, Quality of Life measurements and more.

In September  2013 the Company issued a fifth interim safety report based on data of all patients in the trial as of that date, which was supportive and consistent with previous reports and demonstrated a high safety and tolerability profile. At that time, observed adverse events were related to the expected known symptoms of Inherited Emphysema. No allergic reactions or signs of any risk related to the use of the inhalation device were observed.

“We are proud to have completed this pivotal study as planned and look forward to realizing the potential of our breakthrough inhaled AAT product. The controlled portion of this pivotal study is complete, with patients still being treated in an open-label extension study, for which enrollment rates are very high. We believe these high enrollment rates, as well as the additional treatment time on the drug, especially for patients who were on placebo, further support patient and physician preference for an inhaled treatment for AATD,” noted David Tsur, chief executive officer of Kamada.

“In conjunction with pre-launch marketing activities performed by Chiesi, our European marketing partner, we are in the process of preparing a marketing authorization application (MAA) for the European Medicines Authority (EMA), which we expect to submit in the second half of 2014. We look forward to advancing our innovative and potentially more efficacious treatment for patients suffering with this chronic, life-threatening, inherited lung disease,” added Tsur.

“The opportunity to build a sizeable market is strong, as patients suffering from AATD remain under-identified and under-treated with less than five per cent of cases treated in the US and approximately two per cent in Europe. The availability of a simple blood test for the diagnosis of AATD is expected to further increase awareness and favorably impact demand.  We expect that greater AAT use in Europe and other geographies could also accelerate market growth. Moreover, we believe this non-invasive, user-friendly potential alternative to intravenous AAT is highly attractive as a chronic therapy that represents a compelling commercial opportunity,” concluded Tsur.

In August 2012 Kamada signed an exclusive agreement for the distribution of its inhaled AAT for the treatment of AATD in Europe and with Chiesi Farmaceutici S.p.A, a fully integrated European pharmaceutical company focused on respiratory disease and special care products. Under the agreement Kamada is eligible to receive milestone payments of up to $60 million, subject to achievement of certain regulatory and sales targets. The agreement is for 12 years and Kamada estimates that the sales potential from the agreement, provided its inhaled AAT product is approved for this indication, may reach hundreds of millions of dollars in the coming years.

Kamada received approval of its Investigational New Drug application from the US Food and Drug Administration (FDA) for a phase II clinical trial with inhaled AAT for AATD, and expects to initiate that trial in the coming months.

The Company’s flagship product is Glassia, the first and only liquid, ready-to-use, intravenous plasma-derived AAT product approved by the FDA. Glassia is marketed in the US through a strategic partnership with Baxter International.

Alpha-1 antitrypsin, also called AAT, is a protein made in the liver. Normally the protein travels through the bloodstream and helps protect the body's organs from the harmful effects of other proteins. The lungs are one of the main organs that the AAT protein protects. AAT deficiency (AATD) occurs if the AAT proteins made in the liver are not the right shape, and they get stuck inside liver cells and cannot get into the bloodstream.

Kamada Ltd. is focused on plasma-derived protein therapeutics for orphan indications, and has a commercial product portfolio and a robust late-stage product pipeline.