Arrowhead Pharmaceuticals Doses First Patient with Hepatitis B in Multiple Ascending Dose Portion of Phase 1/2 Study of ARC-521
September 07 2016 - 7:30AM
Business Wire
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced
that it has dosed the first patient in the multiple ascending dose
(MAD) portion of its ongoing Phase 1/2 study of ARC-521, the
company’s second RNAi-based investigational medicine for the
treatment of chronic hepatitis B virus (HBV) infection. To date 24
healthy volunteers have been treated in the study, and the drug
safety committee (DSC) approved initiation of the MAD after a
planned review of safety data from cohort 3 of the healthy
volunteer portion of the study. The MAD is designed to evaluate the
safety, tolerability, and antiviral activity of single and multiple
doses of ARC-521 in patients with chronic HBV.
Bruce Given, M.D., chief operating officer and head of R&D
at Arrowhead said, “The innovative design for the first-in-man
study of ARC-521 is intended to get to multiple dose data in
patients rapidly. Arrowhead’s development staff and the experienced
investigators working on the study are operating at best-in-class
speeds, enabling us to begin the MAD portion of the study just
three months into the clinical program. This study may have
multiple readouts, including single dose safety data in healthy
volunteers and single and multiple dose safety and antiviral
activity data in HBV patients. These readouts should happen
progressively, potentially starting during the first quarter of
2017.”
The MAD portion of the ARC-521 study (ARC5211001) is a
multicenter, sequential, multiple dose, open label study in
patients with chronic HBV. Up to 24 chronic HBV patients (8
patients per dose level) that are negative for Hepatitis B
e-antigen (HBeAg) at screening will enroll sequentially into up to
3 dose levels (2 mg/kg, 4 mg/kg, 6 mg/kg) to receive 3 monthly
doses of open label ARC-521.
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 is a second generation
HBV candidate that 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 conducting 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.
For more information please visit www.arrowheadpharma.com, or
follow us on Twitter @ArrowheadPharma. To be added to the Company's
email list and receive news directly, please visit
http://ir.arrowheadpharma.com/alerts.cfm.
Safe Harbor Statement under the Private Securities Litigation
Reform Act:
This news release contains forward-looking statements within the
meaning of the "safe harbor" provisions of the Private Securities
Litigation Reform Act of 1995. These statements are based upon our
current expectations and speak only as of the date hereof. Our
actual results may differ materially and adversely from those
expressed in any forward-looking statements as a result of various
factors and uncertainties, including our ability to finance our
operations, the future success of our scientific studies, our
ability to successfully develop drug candidates, the timing for
starting and completing clinical trials, rapid technological change
in our markets, and the enforcement of our intellectual property
rights. Our most recent Annual Report on Form 10-K and subsequent
Quarterly Reports on Form 10-Q discuss some of the important risk
factors that may affect our business, results of operations and
financial condition. We assume no obligation to update or revise
forward-looking statements to reflect new events or
circumstances.
DYNAMIC POLYCONJUGATES is a trademark of Arrowhead
Pharmaceuticals, Inc.
Source: Arrowhead Pharmaceuticals, Inc.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20160907005631/en/
Arrowhead Pharmaceuticals, Inc.Vince Anzalone,
CFA626-304-3400ir@arrowheadpharma.comorInvestor
Relations:The Trout GroupChad Rubin,
646-378-2947ir@arrowheadpharma.comorMedia:Russo PartnersMatt
Middleman, M.D.212-845-4272matt.middleman@russopartnersllc.com
Arrowhead Pharmaceuticals (NASDAQ:ARWR)
Historical Stock Chart
From Mar 2024 to Apr 2024
Arrowhead Pharmaceuticals (NASDAQ:ARWR)
Historical Stock Chart
From Apr 2023 to Apr 2024