FDA grants Orphan Drug Designation for BCX9930 in PNH

The review of unblinded data from part 1 of an ongoing phase 1 trial (NCT03891420) to assess the safety (primary endpoint), clinical impact and antiviral effects of galidesivir in patients with *redacted*, and a decision to choose a dose and advance into part 2 of the trial, are expected to occur in 4Q-2020.

Galidesivir is an investigational broad-spectrum antiviral drug that was safe and well tolerated in previous phase 1 trials in healthy subjects, and has demonstrated broad-spectrum activity in vitro against more than 20 RNA viruses in nine different families, including the viruses that cause MERS and SARS.

Press release about ODD

Press release about NIAID funds

Biocryst Q1 2020 Financial Results and Upcoming Key Milestones

total revenues were $4.8 million, compared to $5.9 million in the first quarter of 2019. The decrease was primarily due to reduced peramivir product sales and lower royalty revenues, partially offset by amortization of deferred revenue from the Torii Pharmaceutical, Co. commercialization agreement.

Net loss for the first quarter of 2020 was $37.6 million, or $0.24 per share, compared to a net loss of $31.1 million, or $0.28 per share, for the first quarter of 2019. 

Cash, cash equivalents and investments totaled $114.6 million at March 31, 2020, and reflect a decrease from $137.8 million at December 31, 2019. Operating cash use for the first quarter of 2020 was $23.1 million.

BioCryst expects full year 2020 net operating cash use to be in the range of $125 to $150 million, and its operating expenses to be in the range of $135 to $160 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.

Berotralstat: oral, once-daily treatment for prevention of HAE attacks approval/launch timelines in U.S., Japan, EU remain on track. BioCryst expects three regulatory approvals for berotralstat in 2020 and early 2021. The company does not expect delays due to the-virus.  

Early clinical data in three treatment-naive paroxysmal nocturnal hemoglobinuria (PNH) patients who completed 28 days of therapy, shows BCX9930 inhibited complement and was safe and generally well tolerated.

Patient dosing underway in galidesivir trial in the-virus patients in Brazil. The trial (NCT03891420) is being funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health.