Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1/2 Study of ARC-521

PASADENA, Calif.–(BUSINESS WIRE)–Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR), recently filed a
regulatory submission in New Zealand to begin a Phase 1/2 clinical trial
of ARC-521, its RNAi-based therapeutic candidate for the treatment of
chronic hepatitis B virus (HBV) infection. Pending approval, Arrowhead
intends to proceed with ARC521-1001, a first-in-human study to assess
single and multiple-doses of ARC-521 in healthy volunteers and HBV
patients.
Chris Anzalone, Ph.D., president and CEO of Arrowhead Pharmaceuticals,
said: “ARC-521 is our second pipeline product targeting chronic HBV and
was designed to silence gene products from both HBV cccDNA and
integrated HBV DNA. This is important because our clinical work with
ARC-520 in HBV patients and our long-term chimpanzee study suggest that
different patient groups can have different relative levels of cccDNA.
We think having both ARC-520, which has been very active in patients
with higher cccDNA, and ARC-521, which may be optimal for those with
lower cccDNA, should provide us with the potential to treat all patients
with HBV. We have an aggressive plan for the development of ARC-521 that
includes an accelerated first-in-man Phase 1/2 design intended to allow
rapid transition into multi-dose patient cohorts.”
The application for approval of a clinical trial was submitted to the
New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) for
review by the Standing Committee on Therapeutic Trials (SCOTT).
Arrowhead also intends to seek regulatory clearance to conduct
ARC521-1001 in additional countries.
About ARC-521
Arrowhead’s ARC-521 is being investigated for its potential to produce
functional cures in patients with chronic hepatitis B virus (HBV)
infection. ARC-521 intervenes upstream of the reverse transcription
process where current standard-of-care nucleotide and nucleoside analogs
act, and is designed to silence the production of all HBV gene products.
The small interfering RNAs (siRNAs) in ARC-521 engage the body’s normal
cellular RNAi machinery and direct specific cleavage of HBV RNA
transcripts, thereby reducing the levels of HBV proteins and the RNA
template used to produce viral DNA. Designed to complement ARC-520,
ARC-521 targets HBV mRNA transcripts from both cccDNA and integrated DNA
and is expected to be most suitable for those patients who tend to have
lower levels of viral cccDNA. Arrowhead is investigating ARC-521
specifically to determine if significantly reducing circulating and
non-circulating viral proteins and RNA will allow for re-constitution of
an effective host immune response and ultimately HBsAg seroclearance
resulting in functional cure. As many as 350-400 million people
worldwide are chronically infected with the hepatitis B virus, which can
lead to cirrhosis of the liver and is responsible for 80% of primary
liver cancers globally. Arrowhead is planning to conduct a Phase 1/2
single and multiple-dose study in healthy volunteers and HBV patients.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals develops medicines that treat intractable
diseases by silencing the genes that cause them. Using a broad portfolio
of RNA chemistries and efficient modes of delivery, Arrowhead therapies
trigger the RNA interference mechanism to induce rapid, deep, and
durable knockdown of target genes. RNA interference, or RNAi, is a
mechanism present in living cells that inhibits the expression of a
specific gene, thereby affecting the production of a specific protein.
Arrowhead’s RNAi-based therapeutics leverage this natural pathway of
gene silencing. The company’s pipeline includes ARC-520 and ARC-521 for
chronic hepatitis B virus infection, ARC-AAT for liver disease
associated with alpha-1 antitrypsin deficiency, ARC-F12 for hereditary
angioedema and thromboembolic disorders, ARC-LPA for cardiovascular
disease, and ARC-HIF2 for renal cell carcinoma.
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Source: Arrowhead Pharmaceuticals, Inc.