On January 12, 2020 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported that the company will provide updates on berotralstat, an oral kallikrein inhibitor for hereditary angioedema (HAE), and BCX9930, an oral Factor D inhibitor for complement-mediated diseases, this week at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, BioCryst Pharmaceuticals, JAN 12, 2020, View Source [SID1234553026]).

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"BioCryst is positioned for transformation in 2020 with multiple global approvals and launches of berotralstat, and PNH proof of concept data with BCX9930. The $100 million in additional capital we brought into the company in Q4 2019 provides a foundation for progress and value creation in 2020," said Jon Stonehouse, president and chief executive officer of BioCryst.

Berotralstat Program Updates:

New drug application (NDA) submitted to U.S Food and Drug Administration in December 2019

APeX-J trial in Japan met its primary endpoint (p=0.003) for prevention of HAE attacks, and berotralstat was safe and generally well-tolerated

JNDA submission to Japanese Pharmaceuticals and Medical Devices Agency (PMDA) on-track for Q1 2020

Marketing authorization application to European Medicines Agency (EMA) on-track for Q1 2020
BCX9930 Program Updates:

As previously announced, results from an ongoing three part Phase 1 trial of BCX9930 showed rapid, sustained and >95% suppression of the alternative pathway (AP) of the complement system at 100 mg every 12 hours, as measured by the AP Wieslab assay.

In two initial multiple ascending dose (MAD) assessment cohorts, healthy volunteers received 50 mg or 100 mg of oral BCX9930 or placebo (each MAD cohort randomized 10:2) administered every 12 hours for seven days. Healthy volunteers in the MAD cohorts were prophylactically dosed with the broad-spectrum antibiotic, amoxicillin/clavulanate. BCX9930 was safe and generally well tolerated at all doses studied in single ascending dose and MAD cohorts. There were no serious adverse events. A clinically benign rash was observed in some healthy volunteers in the MAD (two in the 50 mg cohort, seven in the 100 mg cohort), which was self-limited and resolved in 4-8 days after onset.

The company has now completed an additional MAD cohort with 50 mg of oral BCX9930 or placebo administered every 12 hours for 14 days, with vaccination instead of an antibiotic. Key observations from the additional MAD cohort include:

Benign rash (similar to prior MAD cohorts) that was self-limited and resolved in 4 to 8 days post-onset seen in seven healthy volunteers

Successfully dosed-through benign rash, with rash resolving on-drug, in both patients who continued dosing, per protocol

Biopsies of rashes from multiple subjects confirm benign assessment
The company is on-track to report proof of concept data in paroxysmal nocturnal hemoglobinuria (PNH) patients in 1H 2020.

Additional details can be found on slides , which can be accessed at may be accessed in the Investors section of BioCryst’s website at http://www.biocryst.com.