Why BioCryst’s HAE Treatment Failed to Impress

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BioCryst Pharmaceuticals Inc. (NASDAQ: BCRX) shares were absolutely crushed in Tuesday’s session after the company announced late-stage results for its study in hereditary angioedema (HAE) patients.

Specifically, the firm announced Phase 3 results from its APeX-2 trial of BCX7353 for the prevention of HAE attacks. Ultimately, the study achieved its primary endpoint for both dose levels (110 mg and 150 mg), with the larger dose reducing the attack rate in HAE patients by 44% compared to placebo.

Approximately 50% of patients receiving 150 mg BCX7353 had an over 70% reduction in their HAE attack rate compared to baseline and to 15% of placebo patients. In patients on the 150 mg dose with a baseline attack rate of less than two attacks per month, BCX7353 reduced the HAE attack rate by 66% compared to placebo. In patients with a baseline attack rate of more than two attacks per month, the attack rate was reduced by 40%.

The most common drug-related adverse events reported in at least 5% of patients in APeX-2 were nausea (9.8% 110 mg, 7.5% 150 mg, 15.4% placebo), dyspepsia (9.8% 110 mg, 7.5% 150 mg, 5.1% placebo) and diarrhea (7.3% 110 mg, 10% 150 mg, 0% placebo).

Jon Stonehouse, CEO of BioCryst, commented:

HAE patients around the world desperately want access to a cost-effective, convenient, oral therapy to manage their disease. Given the profile of the 150 mg dose of BCX7353 in APeX-2, with half of patients experiencing at least a 70% reduction in attack rate, we have a new oral therapy that patients will want to try.

While these results are impressive, Takeda Pharmaceutical Co. Ltd. (NYSE: TAK) has a HAE treatment that was approved in the United States back in August 2018 and that saw HAE attack reductions in the range of 83% to 87%, a step above BioCryst’s results.

The results from APeX-2 support the submission of a new drug application (NDA) to the U.S. Food and Drug Administration (FDA). Looking ahead, BioCryst plans to submit an NDA to the FDA in the fourth quarter of 2019 and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the first quarter of 2020.

Shares of BioCryst were last seen down about 19% at $3.75, in a 52-week range of $3.40 to $9.95. The consensus price target of $13.29.


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