KVD824 for HAE

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. KVD824 was initially evaluated in a three-part first-in-human study in which 84 subjects received at least one dose of KVD824. The study evaluated single doses up to 1280 mg, multiple doses up to 640 mg, and the effect of food on KVD824 pharmacokinetics.

We are currently conducting a study in the UK assessing both single and multiple dose formulations of KVD824. Six formulations of 600 to 900 mg have been assessed in 16 healthy subjects in a cross-over single dose phase. Selected formulations are being evaluated for 14 days, dosing two times daily, up to 900 mg per dose in a multiple dose phase. 600 mg of KVD824 twice daily maintained concentrations that we believe are above that required for efficacy. In an ongoing 900 mg, twice daily cohort exposures are increased at all time points.

98 subjects have been exposed to treatment with KVD824 as single doses up to 1280 mg and up to 14 days of twice-daily dosing of 600 mg and 900 mg and there have been no concerning safety or tolerability signals. In the first-in-human study adverse event rates were similar in placebo and active arms. No subjects withdrew from the study and no serious adverse events were reported. In the ongoing formulation study, all reported adverse events to date have been mild and no subjects have withdrawn from the study. We will release the full safety data set upon completion of the study.

Scientist

Phase 2

The Phase 2 clinical trial of KVD824 is intended to evaluate the efficacy and safety of KVD824 as a twice-daily prophylactic treatment for prevention of HAE attacks. The study size will be guided by previous pivotal studies, considering the already established pathway to approval for prophylactic treatment of HAE, and will be conducted in multiple territories including the United States and Europe.