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BioCryst Pharmaceuticals Inc.

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Jon P. Stonehouse, President and CEO, commented on the upcoming completion of the trial. He stated, "Looking ahead, we’ll continue to press forward with the advancement of our HAE program. We expect OPuS-2 results in first quarter of next year."
Source: Q3 2015 earnings conference call, 11/05/15

BioCryst Announces Completion of Patient Enrollment in OPuS-2: a Clinical Trial of Avoralstat in Patients With HAE

RESEARCH TRIANGLE PARK, N.C., Oct. 8, 2015 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc., (NASDAQ:BCRX) today announced that it has completed enrollment in OPuS-2 (Oral ProphylaxiS-2), a blinded, randomized, placebo-controlled clinical trial of orally-administered avoralstat in patients with hereditary angioedema (HAE).

OPuS-2 is a 12-week, three-arm, parallel cohort trial designed to evaluate the efficacy and safety of two doses of avoralstat, 300 mg and 500 mg, administered three-times daily compared with placebo. This trial is being conducted in the U.S., Canada and Europe. The primary efficacy endpoint for the trial is the mean angioedema attack rate, which will be reported for each avoralstat dose group compared to placebo.

Final patient visits will occur in January 2016; therefore, BioCryst expects to report OPuS-2 results in early 2016. The results of this trial will be provided for regulatory discussions intended to determine the scope of any additional information that may be required for completion of avoralstat registration.
BioCryst has been corresponding with regulatory agencies regarding deferral of a two-year rat carcinogenicity study for avoralstat. The results from this type of study are normally required to be available at the time of submission for approval. Currently, BioCryst has agreement with the European Medicines Agency (EMA) regarding its request to defer submission of results as a post-filing commitment. Agreement has not been reached with the U.S. Food and Drug Administration (FDA) regarding a deferral at this time. At the end-of-Phase 2 meeting following the completion of OPuS-2, BioCryst will engage in further dialogue with the FDA to discuss deferral, in the context of all available toxicology and clinical data. We plan to initiate a rat carcinogenicity study in early 2016. Without a deferral, our NDA filing would occur in 2018.

Discovered by BioCryst, avoralstat is a novel, selective inhibitor of plasma kallikrein in development for prevention of attacks in patients with HAE. By inhibiting plasma kallikrein, avoralstat suppresses bradykinin production. Bradykinin is the mediator of acute swelling attacks in HAE patients.
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Source: press release, 10/08/15. http://investor.shareholder.com/biocryst/releaseDetail.cfm?ReleaseID=935...

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Jon P. Stonehouse, President, Chief Executive Officer, gave guidance for data from the OPuS-2 study of BCX-4161 for HAE. He stated, "We are currently enrolling patients in the twelve-week OPuS-2 clinical trial and we plan to report results from OPuS-2 by year-end. In parallel, we are making formulation improvements to reduce the daily capsule count, and working to complete development and file an NDA for 4161 in 2017. The second step is to develop a second-generation oral kallikrein inhibitor with a profile that could wipe out HAE attacks with one pill once a day. This would be a game changer."
Source: Q4 2015 earnings conference call, 02/18/15.

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FDA Grants Fast Track Designation for BCX4161 for the Treatment of Hereditary Angioedema

RESEARCH TRIANGLE PARK, N.C., Jan. 26, 2015 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc., (Nasdaq:BCRX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for BCX4161, a novel, orally administered, selective inhibitor of plasma kallikrein in advanced clinical development for the treatment of hereditary angioedema.

The Fast Track designation process of the FDA is designed to facilitate the development and expedite the review and approval of drugs intended to treat serious or life threatening conditions and that address unmet medical needs. A drug that receives Fast Track designation is usually eligible for more frequent written communication and meetings with the FDA to discuss the drug's development plan and the collection of appropriate data supporting drug approval. Priority Review and Rolling Review may be granted, if relevant criteria are met. Rolling Review allows a drug company to submit completed sections of its New Drug Application (NDA) for review by FDA on an ongoing basis, rather than wait until the entire NDA is completed and then reviewed.
"We are very pleased to have been granted orphan drug and fast track status from the FDA, as well as recently receiving a positive opinion for orphan drug designation in Europe," said Jon P. Stonehouse, President & Chief Executive Officer of BioCryst. "BCX4161 and our second generation molecules have the potential to significantly improve HAE patient treatment and their quality of life. We look forward to reporting results from OPuS-2 and sharing updates regarding BCX7353 and our other second generation HAE assets during 2015."

BioCryst is enrolling HAE patients in the OPuS-2 trial of BCX4161; a double-blind, randomized, placebo controlled trial conducted in the U.S. and certain EU countries, with the goal of demonstrating the efficacy and safety of BCX4161 treatment for 12 weeks in approximately 100 patients with HAE. BioCryst expects to report results from OPuS-2 by the end of 2015.
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Source: press release, 1/26/15. http://investor.shareholder.com/biocryst/releaseDetail.cfm?ReleaseID=892...

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BioCryst Receives Positive Opinion on European Orphan Drug Designation for BCX4161 for the Treatment of Hereditary Angioedema
RESEARCH TRIANGLE PARK, N.C., Jan. 14, 2015 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc., (Nasdaq:BCRX) today announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) issued a positive opinion on the application for orphan drug designation for BCX4161 for the treatment of patients with hereditary angioedema (HAE). The European Commission will make a final decision on European Orphan Drug Designation based upon the COMP positive opinion. On December 23, 2014, the U.S. Food and Drug Administration (FDA) granted orphan drug designation for BCX4161.
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Source: press release, 1/14/15. http://investor.shareholder.com/biocryst/releasedetail.cfm?ReleaseID=891...

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BioCryst Initiates OPuS-2: A Clinical Trial of BCX4161 in Patients With Hereditary Angioedema

RESEARCH TRIANGLE PARK, N.C., Dec. 18, 2014 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc., (Nasdaq:BCRX) today announced that it has dosed the first patient in OPuS-2 (Oral ProphylaxiS-2), a blinded, randomized, placebo-controlled clinical trial of orally-administered BCX4161 in patients with hereditary angioedema (HAE).
OPuS-2 is a 12-week, three-arm, parallel cohort design trial to evaluate the efficacy and safety of two doses of BCX4161, 300 mg and 500 mg, administered three-times daily compared with placebo. This trial, to be conducted in the U.S. and selected European countries, is expected to enroll approximately 100 HAE patients. The primary efficacy endpoint for the trial will be the mean angioedema attack rate for each BCX4161 dose group compared to placebo.

"The OPuS-2 trial will provide important information on the efficacy and safety of 12 weeks of oral BCX4161 for prevention of angioedema attacks in HAE patients. OPuS-2 builds on the positive efficacy, safety, tolerability, drug exposure and kallikrein inhibition results from the OPuS-1 4-week study," said Dr. Marc Riedl, M.D., M.S., Associate Clinical Professor at the University of California-San Diego School of Medicine, Clinical Director of the U.S. HAEA Angioedema Center and OPuS-2 Principal Investigator. "OPuS-2 has been designed as an adequate and well-controlled study and represents an important next step toward reaching our goal of improving HAE patients' lives using oral kallikrein inhibitors," added Dr. William P. Sheridan, Chief Medical Officer at BioCryst.

In May 2014, BioCryst announced positive results from the OPuS-1 (Oral ProphylaxiS-1) proof of concept Phase 2a clinical trial of orally-administered BCX4161 in patients with HAE. The trial met the primary efficacy endpoint, several secondary endpoints and all other objectives established for the trial. The primary efficacy endpoint for the trial was the by-subject difference in mean angioedema attack rate on BCX4161 compared to placebo. Treatment with BCX4161 demonstrated a statistically significant mean attack rate reduction of 0.45 attacks per week versus placebo, p < 0.001. The mean attack rate per week was 0.82 on BCX4161 treatment, compared to 1.27 on placebo.

Discovered by BioCryst, BCX4161 is a novel, selective inhibitor of plasma kallikrein in development for prevention of attacks in patients with HAE. By inhibiting plasma kallikrein, BCX4161 suppresses bradykinin production. Bradykinin is the mediator of acute swelling attacks in HAE patients.
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Source: press release, 12/18/14. http://investor.shareholder.com/biocryst/releaseDetail.cfm?ReleaseID=888...

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Jon P. Stonehouse, President, Chief Executive Officer, responded to aquestion regarding the timing for data from the OPUS-2 trial. He stated, "We’re shooting for the end of 2015."
Source: Q3 2014 earnings conference call, 11/06/14.

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OPuS-2: A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Two Dose Levels of BCX4161 for 12 Weeks as an Oral Prophylaxis Treatment for Attacks of Hereditary Angioedema
Estimated Enrollment: 96
Study Start Date: November 2014
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: November 2015 (Final data collection date for primary outcome measure)
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Source: clinical trials.gov. https://clinicaltrials.gov/ct2/show/NCT02303626?term=BCX4161&rank=3

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Compound/DeviceSpecialtyIndicationCompound ClassTarget
BCX-4161 ImmunopathologyHereditary Angioedema (HAE)Plasma kallikrein inhibitorPlasma kallikrein

Mechanism of action: BCX-4161 is being developed as an oral prophylactic treatment for patients suffering from Hereditary Angioedema (HAE). BCX4161 prevents inflammation due to heriditary angioedema by decreasing the liberation of the vascular dilating protein bradykinin from its’ precursor molecule - high molecular weight kininogen (HMWK).

Phase of Development: II

Event Type: Data: Phase II trial results

Dates: 2016-02-01 - 2016-04-30

Results:

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BioCryst Announces Results From OPuS-2
RESEARCH TRIANGLE PARK, N.C., Feb. 08, 2016 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc., (NASDAQ:BCRX) today 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.
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Source: press release, 2/08/16. http://investor.shareholder.com/biocryst/releaseDetail.cfm?ReleaseID=953...

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