Biotech Values

[DewDiligence]

EGRX completes Ryanodex heatstroke trial in Saudi Arabia; efficacy data to be reported before year-end:

http://finance.yahoo.com/news/eagle-pharmaceuticals-successfully-completes-clinical-110000954.html

Eagle Pharmaceuticals, Inc. today announced that the Company has successfully completed the clinical treatment portion of its safety and efficacy study to evaluate RYANODEX (dantrolene sodium for injectable suspension) for Exertional Heat Stroke (“EHS”). The first of its kind study was conducted from September 22-27, 2015 at the Hajj pilgrimage in Saudi Arabia. Due to the unpredictable and sudden nature of EHS, the study was conducted in an emergency and acute-care medical setting.

In this study, 34 EHS patients were randomized to receive current standard of care (“SOC”) treatment or SOC plus RYANODEX. Based on preliminary study results, participants who received RYANODEX in combination with the SOC showed no significant drug-related adverse events. The Company believes this patient cohort is a sufficient number of subjects to enable assessment of a clinically meaningful treatment effect of RYANODEX in EHS. Eagle expects to complete the clinical data analysis during the fourth quarter of 2015.

...The main protocol inclusion criteria required that eligible study patients showed hallmark clinical features of EHS, including the following:

• Subjects between 18-45 years of age, and believed to have experienced exertional physical activity within the previous 24 hours;

• Presence of neurological impairment, which was evaluated using the Glasgow Coma Scale;

• Core body temperature of 104 degrees Fahrenheit or greater; and

Tachycardia (at least 100 heart beats per minute)

Due to the disaster during the Hajj this year, I wasn’t sure whether this trial had been compromised.

Ryanodex is already approved for treatment of malignant hyperthermia, which is a small (but reliable) market. A label expansion for EHS—which affects a material number of athletes—would make Ryanodex a needle-moving product for a small company like EGRX. How much additional data in EHS is needed for an NDA submission is unclear at this point.