Inhaled Alpha-1 Antitrypsin: Kamada Receives Positive Advice on New Phase 3 Study Design
Kamada Ltd., a plasma-derived protein therapeutics company, announced that it has received positive scientific advice from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), related to the development plan for its proposed Phase 3 study design for inhaled alpha-1 antitrypsin therapy, for the treatment of Alpha-1 Antitrypsin Deficiency (Alpha-1).
The company requested scientific advice from the CHMP following the results of their previous Phase 2/3 (E.U.) and Phase 2 (U.S.) studies regarding a proposed subsequent Phase 3 study design, and the CHMP responded: it notified Kamada that it agrees with the overall design of the proposed study, including its objectives, patient population, proposed endpoints and their clinical importance, and the safety monitoring plan.
“Inhalation of alpha-1 antitrypsin represents an alternative to the intravenous (IV) administration, offering the patient a more convenient delivery of the Alpha-1 protein directly to the lungs, enriching the lung epithelial lining fluid (ELF) with alpha-1 antitrypsin at a level which, as demonstrated in Kamada’s Phase 2 (U.S.) study, is at least three times greater than what can be achieved by IV administration,” said Jan Stolk, MD, chairman of the Core Group AATD ERN LUNG, director of the Dutch National Expertise Centre for patients with Alpha-1 and Principal Investigator in the Phase 2/3 study (E.U.) conducted by Kamada.
“I look forward to participating in the Phase 3 study of inhaled alpha-1-antitrypsin. It is my hope that inhaled alpha-1-antitrypsin will become an important new therapy for the treatment of Alpha-1 Antitrypsin Deficiency related lung disease,” said Mark Brantly, MD, professor and chief, Division of Pulmonary, Critical Care & Sleep Medicine, vice chairman of research, Department of Medicine, professor of medicine, molecular genetics and microbiology and Alpha-1 Foundation research professor at the University of Florida.
Brantly is a leading Alpha-1 researcher and he has long been a believer in aerosolized treatment for Alpha-1.
“This EMA decision is an important milestone for Kamada. We believe this official feedback means that the path for a European Phase 3 study is clear and new opportunities are now available to us,” said Amir London, Kamada’s Chief Executive Officer.
“Kamada is committed to the Alpha-1 patient population and medical community, and we are excited by the opportunity to move into the E.U. pivotal trial,” concluded London.
The EMA process is conducted in parallel with Kamada’s continued discussions with the U.S. Food and Drug Administration (FDA) on the Phase 3 study of inhaled alpha-1 antitrypsin for Alpha-1 patients. As previously communicated and following feedback received from the FDA in April 2018, Kamada intends to provide the FDA additional requested information and data, as well as an amended study protocol, during the third quarter of 2018. The proposed modifications and additional information are intended to mitigate the continued safety-related concerns that were communicated by the FDA.