PASADENA, Calif.--(BUSINESS WIRE)--
Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical
company developing targeted RNAi therapeutics, today announced financial
results for its fiscal 2014 third quarter ended June 30, 2014 and
provided an update on the Phase 2a study of ARC-520, its RNAi-based
candidate for the treatment of chronic hepatitis B infection. The
company is hosting a conference call at 4:30 p.m. EDT to discuss
results. Conference call and webcast details can be found below.
ARC-520 Phase 2a Study Update
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Completed dosing of 1 mg/kg and 2 mg/kg dose cohorts
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Initial blinded data suggest that the magnitude of HBsAg knockdown is
similar to non-human primate studies, including the chronically
infected chimpanzee reported on previously
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Duration of knockdown appears to be substantially more sustained than
in non-human primates, with patients in the 2 mg/kg group still
demonstrating substantial knockdown after 8 weeks, which is the most
recent time point available
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HBsAg levels appear to continue to decline in a number of patients at
the 8 week time point in the 2 mg/kg group
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Based on initial review, dosing less frequent than once monthly will
be explored in Phase 2b
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ARC-520 continues to be well tolerated, with no dropouts or serious
adverse events reported
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The overall rate of AEs has been lower in the Phase 2a than in the
Phase 1 normal volunteer study and safety labs continue to show no
indication of end organ toxicity
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Enrolled and dosed additional subjects at 3 mg/kg in the still open
normal volunteer study and the dose performed well, without detected
differences from safety and tolerability results at the other doses.
Overall AEs do not appear to be increasing in frequency or severity
with dose
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Received IRB and DSMB approvals to proceed and began enrolling an
additional dose cohort at 3 mg/kg in the Phase 2a patient study
About ARC-520
Arrowhead's RNAi-based candidate ARC-520 is designed to treat chronic
HBV infection by reducing the expression and release of new viral
particles and key viral proteins. The goal is to achieve a functional
cure, which is an immune clearant state characterized by hepatitis B
s-antigen negative serum with or without sero-conversion. The siRNAs in
ARC-520 intervene at the mRNA level, upstream of where nucleotide and
nucleoside analogues act. In transient and transgenic mouse models of
HBV infection, a single co-injection of Arrowhead's Dynamic
Polyconjugate (DPC) delivery vehicle with cholesterol-conjugated siRNA
targeting HBV sequences resulted in multi-log knockdown of HBV RNA,
proteins and viral DNA with long duration of effect. The company is
conducting a single dose Phase 2a study in chronic HBV patients, and
expects to follow with a multi-dose, multi-national Phase 2b program.
Approximately 350 million people worldwide are chronically infected with
the hepatitis B virus. Chronic HBV infection can lead to cirrhosis of
the liver and is responsible for 80% of primary liver cancers globally.
About ARC-AAT
Arrowhead has developed ARC-AAT for the treatment of liver disease
associated with Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic
disease that severely damages the liver and lungs of affected
individuals. ARC-AAT employs a novel unlocked nucleobase analog (UNA)
containing RNAi molecule designed for systemic delivery using the
Dynamic Polyconjugate delivery system. ARC-AAT is highly effective at
knocking down the Alpha-1 antitrypsin (AAT) gene transcript and reducing
the hepatic production of mutant AAT (Z-AAT) protein. Reduction of
inflammatory Z-AAT protein, which has been clearly defined as the cause
of progressive liver disease in AATD patients, is important as it is
expected to halt the progression of liver disease and allow fibrotic
tissue repair. The Company plans to file for regulatory permission in
the fourth quarter of 2014 and to commence clinical studies.
Read complete press release hereLabels: ARC-520 Phase 2a Study Update