Ipsen (Paris:IPN) (Euronext: IPN; ADR: IPSEY) announced today
that its partner Inspiration Biopharmaceuticals Inc. (Inspiration) was
notified by the Food and Drug Administration (FDA) that both clinical
trials evaluating the safety and efficacy of IB1001, an investigational
intravenous recombinant factor IX (rFIX) therapy for the treatment and
prevention of bleeding episodes in people with hemophilia B, were placed
on clinical hold.
The clinical hold impacts two ongoing IB1001 clinical trials - a phase
III study evaluating the safety and efficacy of IB1001 to treat and
prevent bleeding episodes in adults with hemophilia B, and a phase III
study evaluating the safety and efficacy of IB1001 to treat and prevent
bleeding episodes in previously treated pediatric subjects with
hemophilia B. The adult study has completed its primary analysis period.
Following the FDA's request, Inspiration has notified clinical sites in
the U.S. to hold treatment of patients with IB1001. Inspiration is also
sharing the FDA directive with regulators in countries outside of the
U.S. where the studies are being conducted.
During the course of routine laboratory evaluations conducted as part of
the ongoing phase III clinical trials, Inspiration observed, and
reported to the FDA, a trend towards a higher proportion of IB1001
treated individuals developing a positive response to testing of
antibodies to Chinese Hamster Ovary (CHO) protein, the product's host
cell protein (HCP).
Small amounts of host cell protein are expected and documented in
recombinant therapeutic products of all types. Nevertheless, the higher
than expected rate of anti-CHO antibody development in people treated
with IB1001 has led Inspiration to initiate a thorough investigation.
A total of 86 people with hemophilia B have received IB1001 in clinical
studies and, to date, no adverse events (anaphylaxis or other serious
allergic type reaction and nephrotic syndrome) related to the
development of antibodies to CHO protein have been reported.
Furthermore, no relationship has been demonstrated between the
development of antibodies to CHO protein and the development of any
antibodies to factor IX. Inspiration continues to follow subjects
enrolled in clinical trials of IB1001 to collect safety-related
information and will share this information with regulators.
While this finding may be a potential safety concern, no evidence
suggests a change in the current overall clinical benefit and risk
profile of IB 1001.