PASADENA,
Calif. — March 24, 2014 — Arrowhead Research Corporation (NASDAQ:
ARWR), a biopharmaceutical company developing targeted RNAi
therapeutics, today announced that patient dosing has begun in a Phase
2a clinical trial of ARC-520, its RNAi therapeutic for the treatment of
chronic hepatitis B virus (HBV) infection. The study is planned to
enroll up to 16 chronic HBV patients in two dose cohorts with patients
receiving either ARC-520 or placebo in combination with entecavir. The
study is designed to evaluate the depth and duration of hepatitis B
surface antigen (HBsAg) decline, among other measures, in response to a
single dose of ARC-520. The Company anticipates planned enrollment to be
complete in the second quarter of 2014 and expects top line results to
be released in the third quarter.
“This is a significant milestone for the ARC-520 program and has broad implications for the development of additional RNAi therapeutics using the Dynamic Polyconjugate, or DPC, delivery system,” said Christopher Anzalone, Ph.D., Arrowhead’s President and CEO. “We took an important step toward clinical validation of the DPC system when the Phase 1 was completed and data indicated that ARC-520 was generally safe and well tolerated across all dose levels studied. Our next step will be confirmation that ARC-520 induces deep and durable target gene knockdown in humans. We are confident that the Phase 2a will rapidly provide us with the data necessary to make that assessment as we anticipate patient enrollment and dosing may be complete in the second quarter of this year.”
The Phase 2a study is being conducted at Queen Mary Hospital and Prince of Wales Hospital in Hong Kong. It is a multicenter, randomized, double-blind, placebo-controlled, dose-escalation study to determine the depth and duration of HBsAg reduction after a single intravenous dose of ARC-520 in combination with entecavir in patients with chronic HBV infection. Single doses of ARC-520 will be evaluated at up to two ascending doses of 1.0 mg/kg and 2.0 mg/kg. At each of the two dose levels to be evaluated, a cohort of 8 patients will be enrolled with 6 being dosed with ARC-520 and 2 being dosed with placebo. This study will be conducted in adult male and female patients aged 16 to 65 years, with immune active chronic HBV infection, HBV e antigen (HBeAg) negativity, and ongoing entecavir therapy.
The primary objective of the study is to evaluate the depth and duration of HBsAg decline in response to a single dose of ARC-520 in combination with entecavir. Secondary objectives include evaluation of safety and tolerability and pharmacokinetic (PK) measures. Additional exploratory pharmacodynamics (PD) objectives include evaluation of the effect of ARC-520 on HBV DNA serum titers and antibodies to HBsAg (anti-HBs).
Study visits will occur at screening and on days 1 (dosing), 2, 3, 8, 15, 22, 29, and 85. Patients will be monitored for HBV virology, adverse events, and exploratory PD measures for a total of 12 weeks. If HBsAg titers have not returned to within 20% of baseline by day 29 post-dose, patients will be asked to return for additional visits on days 43 and 57. The final follow up visit will occur on day 85.
“This is a significant milestone for the ARC-520 program and has broad implications for the development of additional RNAi therapeutics using the Dynamic Polyconjugate, or DPC, delivery system,” said Christopher Anzalone, Ph.D., Arrowhead’s President and CEO. “We took an important step toward clinical validation of the DPC system when the Phase 1 was completed and data indicated that ARC-520 was generally safe and well tolerated across all dose levels studied. Our next step will be confirmation that ARC-520 induces deep and durable target gene knockdown in humans. We are confident that the Phase 2a will rapidly provide us with the data necessary to make that assessment as we anticipate patient enrollment and dosing may be complete in the second quarter of this year.”
The Phase 2a study is being conducted at Queen Mary Hospital and Prince of Wales Hospital in Hong Kong. It is a multicenter, randomized, double-blind, placebo-controlled, dose-escalation study to determine the depth and duration of HBsAg reduction after a single intravenous dose of ARC-520 in combination with entecavir in patients with chronic HBV infection. Single doses of ARC-520 will be evaluated at up to two ascending doses of 1.0 mg/kg and 2.0 mg/kg. At each of the two dose levels to be evaluated, a cohort of 8 patients will be enrolled with 6 being dosed with ARC-520 and 2 being dosed with placebo. This study will be conducted in adult male and female patients aged 16 to 65 years, with immune active chronic HBV infection, HBV e antigen (HBeAg) negativity, and ongoing entecavir therapy.
The primary objective of the study is to evaluate the depth and duration of HBsAg decline in response to a single dose of ARC-520 in combination with entecavir. Secondary objectives include evaluation of safety and tolerability and pharmacokinetic (PK) measures. Additional exploratory pharmacodynamics (PD) objectives include evaluation of the effect of ARC-520 on HBV DNA serum titers and antibodies to HBsAg (anti-HBs).
Study visits will occur at screening and on days 1 (dosing), 2, 3, 8, 15, 22, 29, and 85. Patients will be monitored for HBV virology, adverse events, and exploratory PD measures for a total of 12 weeks. If HBsAg titers have not returned to within 20% of baseline by day 29 post-dose, patients will be asked to return for additional visits on days 43 and 57. The final follow up visit will occur on day 85.