Company Spotlight: BioCryst Pharmaceuticals

Shares of BioCryst Pharmaceuticals Inc. (BCRX) are up nearly 15% over the last 5 trading days.

BioCryst is focused on the treatment of rare diseases in which significant unmet medical needs exist. The Company’s most advanced drug candidate in the pipeline is BCX7353, an oral serine protease inhibitor targeting the enzyme Kallikrein, for the treatment of hereditary angioedema.

Hereditary Angioedema is characterized by swelling in the hands, feet, face and larynx, and gastrointestinal attacks producing excruciating abdominal pain, nausea, vomiting, and diarrhea.

BCX7353 is under a phase II trial, dubbed APeX-1, in hereditary angioedema. APeX-1 is a 3-part dose ranging trial evaluating the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of orally administered once daily BCX7353 for 28 days.

The first interim analysis of the APeX-1 trial involved data from 28 of 36 patients, and the results reported in February of this year were quite encouraging.

According to the interim analysis results reported in February, the peripheral and abdominal attacks showed reductions of 88% and 24%, respectively, for BCX7353 350mg once-daily compared with placebo. There was a reduction of 63% in overall attack rate in HAE patients with severe disease who were treated with once-daily BCX7353 350mg for 28 days.

The second interim analysis evaluated data from all patients in Parts 1 and 2 of the trial treated with once-daily doses of BCX7353 125 mg, 250 mg and 350 mg compared to placebo for 28 days.

The results of the second interim analysis, which were reported yesterday, have also been positive.

The second pre-planned analysis of peripheral and abdominal attacks has shown reductions in peripheral attacks of 74% (125 mg *QD), 54% (250 mg QD) and 90% (350 mg QD) compared with placebo and reductions in abdominal attacks of 72% (125 mg QD), 10% (250 mg QD) and 8% (350 mg QD) compared with placebo. (*QD means once daily).

There was a reduction of 73% in overall attack rate in…

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