BioVie Inc (NASDAQ:BIVI) revealed that it had paused patient enrollment in its Phase 2b clinical trial evaluating BIV201 for the treatment of refractory ascites after seeing encouraging data from the first 15 patients in the study.
Ascites is a common complication of advanced liver cirrhosis involving the large accumulation of fluid in the abdomen, often exceeding 5 liters, due to liver and kidney dysfunction. The US Food and Drug Administration (FDA) has never approved a drug to treat ascites, and once patients reach the refractory stage the one-year survival rate is only approximately 50%.
The Carson City, Nevada-based clinical-stage company said treatment with BIV201, plus standard of care (SOC), resulted in a 34% reduction in ascites fluid during the 28 days after treatment initiation, compared to the 28 days prior to treatment (p=0.0046).
READ: BioVie's drug NE3107 shown to help patients with muscle control in Parkinson's disease Phase 2 trial
The improvement was significantly different from those treated with SOC only who experienced a mean increase in ascites fluid of 3.1% (BIV201 versus SOC p=0.05). Patients who completed the treatment with BIV201 experienced a 53% reduction in ascites fluid (p=0.001), which was significantly different from those treated with SOC (p=0.007).
BioVie noted that the improvement was sustained in this group during the three months after treatment initiation, compared to the 3-month pre-treatment period (43% reduction, p=0.06). Overall, the treatment appeared to be well tolerated, added the company. There were no unexpected serious adverse events and overall safety was consistent with the patient population. The firm said detailed results would be presented at an upcoming scientific conference.
In a statement, Michael Porayko, the former chief of Hepatology at Vanderbilt Health, with 38 years of experience managing patients with advanced cirrhosis and ascites said: “The data is very encouraging and supports that BIV201 should continue to be investigated as a potentially effective treatment for the reduction of ascites in patients with cirrhosis and refractory ascites, reducing the need for paracentesis and potentially improving both the symptoms related to fluid buildup and quality of life.”
BIV201 is a continuous infusion of terlipressin, a drug used in over 40 countries to treat related complications of liver cirrhosis that was recently approved in the US, but not Japan. With the novel prefilled room temperature stable syringe used in this study, BIV201 could potentially provide a superior terlipressin drug delivery system throughout the world.
The current trial (NCT04112199) evaluates the effectiveness of BIV201 combined with standard-of-care, compared to SOC alone, for the treatment of refractory ascites. Terlipressin was administered with a continuous low-dose infusion via a portable pump in two 28-day treatment cycles.
Initiating talks with the FDA
The primary endpoints are the incidence of complications of at least Grade 2 severity, and the change in cumulative ascites in the 12-week period following randomization compared to a 12-week pre-treatment period. The BIV201 trial planned to enroll 30 patients to be treated in the home care setting.
“Compelling data from the first 15 patients led us to pause enrollment,” said BioVie CEO Cuong Do. “Given the high unmet need for this condition, we believe the most prudent course is to initiate conversations with the FDA on proceeding to the pivotal Phase 3 trial so that we can bring this innovation to patients as quickly as possible.”
BIV201 (continuous infusion terlipressin) has received Orphan Drug and Fast Track designations from the FDA for the treatment of ascites due to all etiologies except cancer. This would include, for instance, ascites due to advanced liver cirrhosis. First-to-market Orphan therapies typically receive seven years of market exclusivity in the US for the designated use.
BioVie is developing innovative drug therapies for the treatment of neurological and neurodegenerative disorders and advanced liver disease
Contact the author Uttara Choudhury at uttara@proactiveinvestors.com
Follow her on Twitter: @UttaraProactive