Arrowhead Pharmaceuticals Focuses Resources on Subcutaneous and Extra-Hepatic RNAi Therapeutics
– Conference Call and Webcast Today at 4:30 p.m. EST
PASADENA, Calif.–(BUSINESS WIRE)–
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced a
strategic redeployment of resources to support the development of RNAi
therapeutics that utilize the company’s new proprietary subcutaneous
(subQ) and extra-hepatic delivery systems. Arrowhead will discontinue
development of clinical stage drug candidates ARC-520, ARC-521, and
ARC-AAT, which utilize the DPCiv™, or EX1, delivery vehicle.
The company is hosting a conference call at 4:30 p.m. EST to discuss
this decision.
Arrowhead remains committed to finding therapeutic options for patients
with chronic hepatitis B (HBV) infection and alpha-1 antitrypsin
deficiency (AATD). The company intends to advance to the clinic two
previously unannounced HBV and AATD programs using our subQ platform.
Arrowhead has learned a great deal during prior HBV and AAT studies that
will help drive the subQ programs efficiently.
Existing preclinical subQ and extra-hepatic programs such as ARC-LPA and
ARC-AMG1, which are partnered with Amgen, ARC-F12, ARC-HIF2, and other
unannounced programs are not affected by this decision.
Because of the discontinuation of its existing clinical programs, the
company is reducing its workforce by approximately 30%, while
maintaining full resourcing necessary to support current and potential
future partner-based programs and Arrowhead’s burgeoning pipeline. This
more streamlined structure should enable the company to continue to
develop its programs rapidly, and is intended to extend its cash runway
into 2019.
The decision to discontinue development of EX1-containing programs was
based primarily on two factors. First, during ongoing discussions with
regulatory agencies and outside experts, it became apparent that there
would be substantial delays in all clinical programs that utilize EX1,
while the company further explored the cause of deaths in a non-clinical
toxicology study in non-human primates. Second, Arrowhead has made
substantial advances in RNA chemistry and targeting resulting in large
potency gains for subQ administered and extra-hepatic RNAi-based
development programs. In preclinical studies with the subQ platform, the
company has obtained depth and duration of target gene knockdown
approaching that of intravenously administered EX1-containing
candidates, at lower doses and with good safety margins.
The company believes it is prudent to focus its development resources
entirely on its subQ and extra-hepatic pipeline, which includes programs
in HBV, AAT, Factor 12, HIF-2alpha, and other unannounced programs. In
addition to its own pipeline, Arrowhead is also focused on providing
full resources to support its partnership with Amgen and potential
future partnerships for its subQ and extra-hepatic delivery systems.
The tolerability of ARC-520, ARC-521, and ARC-AAT in human clinical
trials appeared to be favorable, and in the company’s view, supported
advancing the programs. EX1-containing candidates have been administered
over 800 times in more than 300 human study subjects and patients and
have been generally well tolerated, with a small minority (6%) of
infusions being associated with infusion reactions. In addition, across
the ARC-520, ARC-521, and ARC-AAT clinical programs, laboratory values
have not been deemed indicative of drug induced organ toxicity.
In addition, each candidate was highly active against its respective
target. For example, data presented earlier this month at The Liver
Meeting® show that ARC-AAT achieved 90% knockdown of serum
AAT, which is believed to be near full suppression of liver production
of the protein, in a Phase 1 clinical study. For ARC-520, it was
previously reported that reductions in surface antigen (HBsAg) of almost
99%, or 2 logs, were achieved after a single dose. In subsequent
multiple dose studies, for which data have not yet been reported,
reductions of almost 3 logs were observed, with several patients being
tracked that appear poised to possibly seroclear HBsAg, representing
potential function cures.
However, due to likely regulatory considerations, as of this
announcement all patient recruitment for ARC-520, ARC-521, and ARC-AAT
has been halted and dosing discontinued. The company will work together
with investigators and clinical sites to ensure a smooth transition of
study closure and patient medical care.
Conference Call and Webcast Details
Investors may access a live audio webcast on the Company’s website at http://ir.arrowheadpharma.com/events.cfm.
For analysts that wish to participate in the conference call, please
dial 855-215-6159 or 315-625-6887 and enter Conference ID 27048601.
A replay of the webcast will be available on the company’s website
approximately two hours after the conclusion of the call and will remain
available for 90 days. An audio replay will also be available
approximately two hours after the conclusion of the call and will be
available for 3 days. To access the audio replay, dial 404-537-3406 and
enter Conference ID 27048601.
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.
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 the safety and efficacy of our product
candidates, the duration and impact of regulatory delays in our clinical
programs, 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.
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Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
626-304-3400
ir@arrowheadpharma.com
or
Investor
Relations:
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Chad Rubin
646-378-2947
ir@arrowheadpharma.com
or
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matt.middleman@russopartnersllc.com
Source: Arrowhead Pharmaceuticals, Inc.
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