Arrowhead Pharmaceuticals’ Preclinical Candidate ARC-LPA Achieves 98% Knockdown & Long Duration of Effect after Subcutaneou...
May 06 2016 - 12:00PM
Business Wire
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today presented a
poster on ARC-LPA, its preclinical development program targeting
lipoprotein (a), or Lp(a), for the treatment of cardiovascular
disease at the Arteriosclerosis, Thrombosis and Vascular Biology |
Peripheral Vascular Disease (ATVB|PVD) 2016 Scientific Sessions in
Nashville. ARC-LPA is the first RNAi therapeutic program to use
Arrowhead’s new delivery vehicles designed for subcutaneous (SQ)
administration.
Chris Anzalone, Ph.D., president and CEO of Arrowhead
Pharmaceuticals, said: “These data are exciting for several
reasons. First, they represent continued progress in our SQ
delivery platform, creating opportunities to address various
diseases that require chronic treatment and where SQ administration
may be preferable for patients and physicians. We are enabling very
deep target gene knockdown with long duration of effect that may
enable monthly, bi-monthly, or even less frequent administration.
Second, reducing Lp(a), as we have seen now in multiple animal
models, could represent an important leap forward in the treatment
of cardiovascular disease. High levels of Lp(a) are associated with
increased risk of cardiovascular disease independent of cholesterol
and LDL, and there is currently no good way to deeply reduce
circulating levels of Lp(a).”
The poster titled, “Lipoprotein(a) targeting with RNAi delivery
platforms in transgenic mice and cynomolgus monkeys” (presentation
428), describes data from in vitro screening of RNAi triggers and
multiple in vivo models, including transiently transgenic mice,
transgenic mice (Tg), and non-human primates (NHPs). Key findings
from these studies include the following:
- Screening of RNAi triggers in Tg mice
identified those that exhibited substantial and sustained knockdown
of serum apo(a) and Lp(a) levels
- RNAi trigger sequences were active in
both intravenous and SQ platforms
- Structure activity relationship (SAR)
studies looking at chemical modifications to the RNAi trigger
identified a lead that demonstrated greater than 98% maximum
knockdown after a single 3 mg/kg SQ dose in transgenic mice
- Duration of effect gains were also made
with greater than 85% knockdown still seen at 6 weeks post
dose
- In NHPs, 85-90% reduction of serum
Lp(a) levels was observed after three weekly 3 mg/kg SQ doses
- Duration of effect in NHPs was long,
with Lp(a) levels still reduced by 75% 6 weeks after the final
dose
A copy of the poster presentation will be made available on the
Events and Presentations page under the Investors section of the
Arrowhead website.
About ARC-LPA
Arrowhead’s RNAi-based candidate ARC-LPA is in preclinical
development as a potential treatment for cardiovascular diseases.
ARC-LPA is designed to reduce production of apolipoprotein(a), a
key component of lipoprotein(a), or Lp(a). Lp(a) levels in humans
are genetically defined and higher levels correlate with increased
risk of cardiovascular diseases, independent of cholesterol and LDL
levels. ARC-LPA is Arrowhead’s first drug candidate to use a
subcutaneously administered delivery construct.
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
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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
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financial condition. We assume no obligation to update or revise
forward-looking statements to reflect new events or
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Pharmaceuticals, Inc.
Source: Arrowhead Pharmaceuticals, Inc.
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version on businesswire.com: http://www.businesswire.com/news/home/20160506005216/en/
Arrowhead Pharmaceuticals, Inc.Vince Anzalone,
CFA626-304-3400ir@arrowheadpharma.comorInvestor
Relations:The Trout GroupChad
Rubin646-378-2947ir@arrowheadpharma.comorMedia:Russo
PartnersMatt Middleman,
M.D.212-845-4272matt.middleman@russopartnersllc.com
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