BioCryst Pharmaceuticals Inc. (NASDAQ: BCRX) shares fell on Tuesday after the company announced initial results from its hereditary angioedema (HAE) trial.

Specifically, data that came from the ZENITH-1 trial, and BCX7353 was well tolerated and superior to the placebo against the majority of efficacy endpoints evaluated in HAE patients suffering an acute attack.

In the 750 mg dose cohort of the trial, which has completed, 33 patients treated a total of 95 attacks (64 with BCX7353, 31 with placebo). Patients self-treated their HAE attacks on a blinded basis with oral BCX7353 or oral placebo and recorded their symptoms and attack severity using both a Visual Analog Scale (VAS) and a standardized questionnaire. Patients also recorded the time they used any standard-of-care (SOC) acute treatment medicine.

Importantly, compared to placebo, improvement in symptoms and VAS scores was seen as early as one hour after oral BCX7353 dosing and was sustained through 24 hours. Through 24 hours, SOC medication use was reduced by 31.6% after BCX7353 compared with placebo, and no or mild symptoms were reported in 64.1% of attacks treated with BCX7353 compared with 32.3% of attacks treated with placebo.

Dr. Hilary Longhurst, honorary consultant immunologist, Addenbrookes Hospital, Cambridge, United Kingdom, and principal investigator of the ZENITH-1 trial, commented:

ZENITH-1 represents a groundbreaking study, as the first clinical trial to demonstrate effective treatment of acute HAE attacks with an oral therapy.  The observed effect of BCX7353 within one hour of dosing and the substantial reduction in rescue medication use compared to placebo suggest that BCX7353 has outstanding potential to offer physicians and patients an urgently needed new oral therapy option,”

Shares of BioCryst traded down 14% early Tuesday at $6.16, with a consensus analyst price target of $9.25 and a 52-week range of $4.12 to $7.49.

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