BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) announced results from OPuS-2 (Oral ProphylaxiS-2), a clinical trial of avoralstat administered three times daily as a liquid-filled soft gel formulation for the prophylactic treatment of hereditary angioedema (HAE) attacks.

In the OPuS-2 study, HAE patients with a historical attack frequency of greater than 0.45 attacks per week were randomized to treatment with either 500 mg or 300 mg of avoralstat, or placebo, administered three times daily for 12 weeks.  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.

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

“OPuS-2 was a well-designed and executed trial that gave us a clear answer; this dosage form of avoralstat is not a viable formulation to move forward,” said Jon P. Stonehouse, President & Chief Executive Officer. “While we are disappointed in the study results, we learned that meaningfully better exposure is needed for avoralstat to succeed. We expect results from a relative bioavailability study testing a novel solid dosage form of avoralstat by mid-year – the primary goals of this study are to achieve much higher exposures and twice daily dosing. Our other opportunity to achieve higher exposure of an oral kallikrein inhibitor is with BCX7353 – we expect results from the BCX7353 APeX-1 dose ranging study in HAE patients by year end.”

Secondary efficacy endpoints included measures of quality of life, attack duration and attack severity. 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 placebo.

Oral administration of avoralstat in OPuS-2 was generally safe and well tolerated; the adverse event profile was similar to that for placebo; and no safety signals were observed. (Original Source)

Shares of BioCryst Pharmaceuticals are falling nearly 63% in early morning trading. BCRX has a 1-year high of $16.83 and a 1-year low of $6.09. The stock’s 50-day moving average is $8.36 and its 200-day moving average is $10.50.

On the ratings front, BioCryst has been the subject of a number of recent research reports. In a report issued on January 8, FBR analyst Christopher James reiterated a Buy rating on BCRX. Separately, on January 6, Jefferies Co.’s Brian Abrahams upgraded the stock to Buy and has a price target of $14.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Christopher James and Brian Abrahams have a total average return of -31.2% and 15.9% respectively. James has a success rate of 11.6% and is ranked #3543 out of 3584 analysts, while Abrahams has a success rate of 50.8% and is ranked #184.

Overall, one research analyst has rated the stock with a Sell rating, one research analyst has assigned a Hold rating and 2 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $14.00 which is 128.0% above where the stock closed last Friday.

Biocryst Pharmaceuticals Inc is a biotechnology company. The Company designs, optimizes and develops novel small molecule drugs that block key enzymes involved in the pathogenesis of diseases.