On November 6, 2019 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the third quarter ended September 30, 2019 and provided a corporate update (Press release, BioCryst Pharmaceuticals, NOV 6, 2019, View Source [SID1234550408]).

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"BioCryst is positioned for a transformational 2020, with the potential approval and launch of BCX7353 in the U.S., regulatory filings for BCX7353 in Japan and Europe, and data from our PNH proof of concept study with BCX9930 reading out in the first half of the year," said Jon Stonehouse, president and chief executive officer of BioCryst.

"We are also actively evaluating multiple approaches to add capital to the balance sheet by the end of 2019, as we did with the Japanese licensing agreement for BCX7353, which we announced earlier this week," Stonehouse added.

Upcoming Key Milestones

HAE Program – BCX7353

Submit a new drug application (NDA) for oral, once-daily BCX7353 for the prevention of hereditary angioedema (HAE) attacks with the U.S. Food and Drug Administration (FDA) (Q4 2019)

Submit a marketing authorization application for oral, once-daily BCX7353 for the prevention of HAE attacks with the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) and the European Medicines Agency (EMA) (Q1 2020)

Commence ZENITH-2, a Phase 3 clinical trial of oral BCX7353 (750 mg) for the treatment of acute HAE attacks, in 2020, pending the completion of interactions with regulators on the Phase 3 program and additional work on the acute oral formulation (2020)
Complement Oral Factor D Inhibitor Program – BCX9930

Report data from a proof of concept study in paroxysmal nocturnal hemoglobinuria (PNH) patients receiving oral BCX9930 (1H 2020)
ALK-2 Inhibitor Program – BCX9250

Continue ongoing Phase 1 clinical trial of BCX9250, an oral ALK-2 kinase inhibitor for treatment of fibrodysplasia ossificans progressiva (FOP), in healthy subjects
Recent Corporate Developments

On November 6, 2019, the company provided a commercial update based on new 48-week clinical data from the APeX-2 and APeX-S trials, and detailed market research conducted with HAE patients, physicians who treat HAE and payors.

On November 5, 2019, the company announced it had licensed commercialization rights in Japan to Torii Pharmaceutical, Co. for BCX7353 for up to $42 million of upfront and potential milestone payments, including a $22 million upfront payment.
On November 1, 2019, the company announced it had begun enrollment of a Phase 1 trial of BCX9250, an oral ALK-2 inhibitor discovered and developed by BioCryst, for the treatment of FOP. The trial will evaluate the safety and tolerability and characterize the pharmacokinetic and pharmacodynamic profiles of BCX9250 in single and multiple ascending doses in healthy volunteers.
On November 1, 2019, the company announced results from a Phase 1 trial of BCX9930, an oral Factor D inhibitor discovered and developed by BioCryst, showing that BCX9930 was safe and generally well tolerated, and demonstrated rapid, sustained and >95% suppression of the alternative pathway of the complement system at 100 mg every 12 hours. Based on these results, the company is advancing the program into a proof of concept (PoC) study in PNH patients and plans to report data from the PoC study in the first half of 2020.
On September 26, 2019, the company announced the U.S. Department of Health and Human Services (HHS) had exercised its option to purchase an additional 10,000 doses of BioCryst’s approved antiviral influenza therapy, RAPIVAB (peramivir injection).
On September 23, 2019, the company announced it had appointed clinical rare disease expert, Helen Thackray, M.D., FAAP, to its board of directors.
Third Quarter 2019 Financial Results

For the three months ended September 30, 2019, total revenues were $1.8 million, compared to $1.5 million in the third quarter of 2018. The increase was primarily due to the recognition of $0.3 million of peramivir product sales to Shionogi & Co., Ltd., the company’s commercial partner in Japan.

Research and development (R&D) expenses for the third quarter of 2019 increased to $25.1 million from $22.0 million in the third quarter of 2018, primarily due to increased spending on our complement-mediated diseases programs, which entered Phase 1 clinical testing in June 2019.

Selling, general and administrative (SG&A) expenses for the third quarter of 2019 increased to $11.7 million, compared to $7.9 million in the third quarter of 2018. The increase was primarily due to increased spending on commercial activities and medical affairs to support the U.S. commercial launch of BCX7353 in 2020.

Interest expense was $3.0 million in the third quarter of 2019, compared to $2.3 million in the third quarter of 2018, and was primarily due to an increase in the outstanding balance of the company’s secured credit facility in February 2019.

Net loss for the third quarter of 2019 was $37.6 million, or $0.34 per share, compared to a net loss of $29.6 million, or $0.28 per share, for the third quarter of 2018.

Cash, cash equivalents and investments totaled $70.0 million at September 30, 2019, and reflect a decrease from $128.4 million at December 31, 2018. Operating cash use for the third quarter of 2019 was $24.5 million. Net operating cash use for the first nine months of 2019 was $77.9 million, as compared to $70.7 million for the first nine months of 2018.

Financial Outlook

BioCryst continues to expect full year 2019 net operating cash use to be in the range of $105 to $130 million, and its operating expenses to be in the range of $120 to $145 million. The company’s operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the company’s stock, as well as by the vesting of the company’s outstanding performance-based stock options.

Conference Call and Webcast

BioCryst management will host a conference call and webcast at 8:00 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 4891026. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 4891026.