Kamada Ltd. (Nasdaq and TASE: KMDA), a plasma-derived protein therapeutics company focused on orphan indications, announces preliminary top-line results from the Phase II/III pivotal 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).
The endpoints selected for this trial were based on scientific advice from the European Medicines Agency (EMA) and include those deemed to be clinically meaningful, such as frequency, time to first, duration and severity of exacerbation events, among others. A preliminary analysis of the results indicates clinically meaningful signs for inhaled AAT efficacy as well as additional positive signs in specific study populations. In a very important secondary endpoint, frequency of severe exacerbation was approximately 50% lower in the AAT group versus placebo. With regards to the primary endpoint of "time to first moderate or severe exacerbation," early data do not show differences between the two treatment groups.
Forced Expiration Volume in one second (FEV1) data, a secondary and safety endpoint, indicated positive trends in improvement. Additionally, the data showed efficacy in certain subsets of patient populations. Safety data of inhaled AAT was supportive and consistent with previous reports and demonstrated a high safety and tolerability profile. Based on the initial review, the Company believes that some of the data may support the issuance of new patents for the product.
The Company continues to analyze the data in accordance with the statistical plan and intends to release a more profound set of results in third quarter of 2014.
"We received preliminary trial results, and today are reporting partial and early stage data regarding the primary endpoint and certain key secondary endpoints. We have detected clinically meaningful signs for inhaled AAT efficacy as well as additional positive signs in specific study populations, and are examining these data further to better understand the results and to assure the information is valid and correlates with additional clinical parameters," stated Pnina Strauss, Vice President of Clinical Development and Intellectual Property of Kamada. "Severe exacerbations involve hospitalizations and deterioration of respiratory symptoms including breathlessness, increased sputum volume, change in sputum color and/or purulence. A decrease in severe exacerbations is a significant benefit to patients and will lower the overall cost of healthcare, which includes usage of drugs and hospitalization, among others."
"We believe the existing data support a regulatory filing in Europe, and will continue as planned to meet with the EMA according to the centralized procedure for licensure during the fourth quarter of this year," added Ms. Strauss.
"This is an important study with encouraging data that will influence our understanding of the disease and answer an important question for long-term patient management, and may serve as the entry point to authorize inhaled treatment for the AATD population," said Rob Stockley, MD, DSc, Director Lung Immuno Biochemical Research Laboratory, Birmingham, England and a lead investigator in the trial. "The data is preliminary and needs to be analyzed in greater detail to determine the importance of these initial signs and trends with the aim of having a relevant and user-friendly treatment for patients in the near future."
"We are encouraged by the significant reduction in severe exacerbation rates as well as the positive trends we are seeing in certain patient sub-populations. We look forward to receiving the complete final analysis in the coming months and to receiving the results from the ongoing open-label extension study. We believe these clinically meaningful findings to date may support our efforts to bring inhaled AAT to patients with AATD in Europe. We believe that based on efficacy and ease of use we shall have a competitive advantage in a one billion dollar market," noted David Tsur, co-Founder and Chief Executive Officer of Kamada.
"We thank the trial's principal investigators and patients for their commitment to this study and look forward to advancing our inhaled AAT for the benefit of patients suffering from this genetic deficiency," added Mr. Tsur.
Phase II/III Clinical Trial Design
The multicenter randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of Kamada's inhaled formulation of human AAT to treat AATD in 168 patients. The study involved the inhalation of 160 mg of human AAT or placebo twice daily via the eFlow® device for 50 weeks. The primary endpoint of the study is time to the first moderate or severe exacerbation event between the two groups at one year, and the study is 80% powered to demonstrate a 20% difference. Secondary endpoints include additional parameters of exacerbation events, pulmonary function tests and safety. Additional exploratory endpoints include CT densitometry in a subset of subjects, Quality of Life measurements and more.
Open-Label Extension Study
Eligible patients from this Phase II/III trial were given the option to participate in a 50-week, open-label safety study. A majority of eligible patients have consented to participate in this study. The Company expects to use the additional data from this study as part of the regulatory submissions.
"Enrollment rates for patients eligible for the open-label extension study were high, with patients in the study continuing to be treated. We believe these high enrollment rates, as well as the additional treatment time on the drug, further support patient and physician preference and acceptance of an inhaled treatment for AATD," noted Mr. Tsur.
Commercialization of Inhaled AAT for the Treatment of AATD
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.
Recently, Kamada has initiated a U.S. Phase II clinical trial with its inhaled AAT for AATD, and expects that the data from the European trial together with the data from the U.S. trial will support licensure application in the U.S. and additional territories.
Kamada management will host a conference call today at 8:30 a.m. Eastern time, to discuss these results and to answer investor questions.