Dutch biotechnology firm Pharming Group and US-based Salix Pharmaceuticals have announced treatment of the first patient in their Phase II clinical trial of Ruconest, (C1 esterase inhibitor [Recombinant]) 50IU / kg, for prophylaxis in patients with hereditary angioedema (HAE).
The randomised, double-blind trial is enrolling HAE patients deficient in C1 inhibitor and with a history of at least four attacks per month, in which 30 patients will receive Ruconest either once or twice weekly, or a placebo in each of three treatment periods.
The company said the crossover design will help all patients receive each of the dosing regimens.
Scheduled to be conducted at sites in Canada, Europe, Israel and the US, the trial will evaluate the safety and efficacy of Ruconest when used for prophylaxis of angioedema attacks in patients with HAE.
University of Milan professor Dr Marco Cicardi and University of California associate professor and clinical director of US HAEA Angioedema Center Dr Marc Riedl are the principal investigators for the trial.
As part of the Pharming-Salix license agreement, the firms will equally share development costs for Ruconest for HAE prophylaxis.
The deal will see Pharming secure an undisclosed milestone payment from Salix when US Food and Drug Administration (FDA) grants approval for this additional indication.
Ruconest is indicated to treat acute attacks in adult and adolescent patients with hereditary angioedema (HAE) and is contraindicated for those with a history of allergy to rabbits or rabbit-derived products.
The drug is also contraindicated for patients with a history of life-threatening immediate hypersensitivity reactions to C1 esterase inhibitor preparations, including anaphylaxis.
The company said Ruconest is the first and only plasma-free, recombinant C1-INH approval from the FDA and was approved in July 2014.
Ruconest is commercialised by Pharming in Austria, Germany and the Netherlands.
It is distributed by Swedish Orphan Biovitrum (SOBI) in other EU countries and Azerbaijan, Belarus, Georgia, Iceland, Kazakhstan, Liechtenstein, Norway, Russia, Serbia and Ukraine.