Understanding HAE
A rare but potentially life-threatening disease, HAE is most frequently caused by a genetic disorder where the protein C1-inhibitor esterase is inadequately synthesized or dysfunctional.
C1-inhibitor is a critical mediator of inflammation. With inadequate levels or malfunctioning C1-inhibitor, excess plasma kallikrein can become activated, leading the body to produce the vasopeptide bradykinin in excessive amounts, which in turn leads to increased vascular permeability and sudden onset of angioedema.
Angioedema attacks can occur in the limbs, face, gastrointestinal tract and/or airways. Laryngeal episodes can happen to any patient and are potentially life-threatening.
Ongoing R&D
Currently, all HAE therapies are either injected on an acute basis or infused prophylactically. Neither therapy is ideal, and it can take time for HAE patients to learn what works best for them.
Our Phase 1 study for KVD900 suggested the compound displays a profile well-suited for use as an oral, on-demand therapy for attacks, with a combination of rapid uptake into the plasma and high plasma concentrations.
In addition to KVD900, KVD824 is our twice-daily oral plasma kallikrein inhibitor for prevention of HAE attacks. We intend to file an Investigational New Drug (IND) submission for a Phase 2 clinical trial of KVD824 in the first quarter of 2021. This trial is intended to evaluate the efficacy and safety of KVD824 as a twice-daily prophylactic treatment for prevention of HAE attacks.
We’re excited by the possibility of developing best-in-class oral therapies for HAE and an alternative for HAE patients facing repeated angioedema attacks. We look forward to your interest as our research continues.