Is BioCryst Reaching Crush Depth?

Health and Healthcare

Published: February 8, 2016 11:05 am

Last Updated: January 13, 2020 1:29 pm

BioCryst Pharmaceuticals Inc. (NASDAQ: BCRX) is getting crushed after announcing results from OPuS-2 for the treatment of hereditary angioedema (HAE) attacks. Unfortunately, the company said that this treatment failed to demonstrate a statistically significantly lower mean attack rate than a placebo. This was a liquid-filled soft gel formulation for the prophylactic treatment of HAE.

HAE is a rare and severely debilitating and potentially fatal genetic condition that occurs in about one in 10,000 to one in 50,000 people. It is also worth noting that BioCryst said that the company is still talking up HAE test results by midyear and by the end of this year.

The primary goals of the trial were to characterize the efficacy of avoralstat in reducing the frequency of angioedema attacks, and to evaluate the safety and tolerability of 12 weeks of avoralstat treatment. The primary efficacy endpoint was angioedema attack frequency.

Secondary efficacy endpoints included measures of quality of life, attack duration and attack severity. BioCryst did say that statistically significant improvements in duration of attacks and in the angioedema quality of life total score, and its domains, were observed comparing the 500 mg three times a day avoralstat arm to a placebo.

Thirty-eight subjects received avoralstat 500 mg, 36 subjects received avoralstat 300 mg and 36 subjects received the placebo. Treatment with 500 mg and 300 mg of avoralstat three times daily failed to demonstrate a statistically significantly lower mean attack rate versus the placebo. The mean (standard deviation) attack rates per week were 0.63 (0.57) on avoralstat 500 mg, 0.71 (0.66) on avoralstat 300 mg, and 0.61 (0.41) on placebo.