Pharming Group N.V.
today announced the successful completion of patient enrolment in the
Phase 2 clinical study of RUCONEST®, (recombinant C1 esterase Inhibitor,
50 IU/kg), for prophylaxis in patients with hereditary angioedema
(HAE).
Thirty HAE patients deficient in C1 esterase Inhibitor and with a
history of at least four attacks per month have been enrolled in the
randomized, double-blind study. The patients receive RUCONEST® either
once or twice weekly, or placebo in each of three four-week treatment
periods. With the crossover design, all patients will receive each of
the dosing regimens. The study will evaluate the safety and efficacy of
RUCONEST® when used for prophylaxis of angioedema attacks in patients
with HAE.
The recruitment phase of the study was initiated in January 2015 and
is being conducted at sites in Canada, Europe, Israel and the United
States. The trial is being coordinated by principal investigators, Dr.
Marco Cicardi, Professor at the University of Milan, and Dr. Marc Riedl,
Professor of Medicine and Clinical Director of the US HAEA Angioedema
Center at the University of California, San Diego.
“We are pleased with the timely completion of patient enrolment for
this double-blind, randomized, placebo-controlled study. We expect the
results to be highly informative in determining the safety and efficacy
of RUCONEST® to prevent angioedema attacks in patients with HAE. We
expect to have the top-line results of the study around the end of the
second quarter. If the results are positive, we intend to meet with the
FDA and EMA to discuss next steps for the program, including our plans
for a new subcutaneous formulation.” said Bruno Giannetti, MD PhD, Chief
Operating Officer of Pharming.
Under the terms of the North American licensing agreement with
Valeant Pharmaceuticals International, Valeant and Pharming share the
development costs for RUCONEST® for prophylaxis of HAE. Pharming will
receive an undisclosed milestone payment from Valeant as and when FDA
approval for this additional indication is given. RUCONEST® has been
granted Orphan Drug designation by FDA for the prophylactic treatment of
angioedema caused by hereditary or acquired C1 esterase inhibitor
deficiency, with data exclusivity until 2026 under the Biologics Price
Competition and Innovation Act.
