Arrowhead Publishes Data on Advances in Subcutaneous siRNA Delivery
PASADENA, Calif.–(BUSINESS WIRE)–
Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical
company developing targeted RNAi therapeutics, today announced the
publication of new data on a subcutaneously administered formulation of
its Dynamic Polyconjugate (DPC) delivery system. The company believes
the new DPCs are highly potent and may represent a dramatic improvement
in duration of activity over competing technologies. This new class of
DPCs may also enable targeting of RNAi therapeutics to tissues outside
of the liver. The manuscript entitled, “Protease-triggered siRNA
delivery vehicles,” by David B. Rozema et al, was made available online
ahead of print in the Journal of Controlled Release 209 (2015) 57-66.
In the publication, Arrowhead scientists describe the development of
protease-sensitive masking chemistries that are used in a class DPCs
where the RNAi trigger molecule is conjugated directly to the polymer
delivery vehicle. These new vehicles expand on the company’s existing
acid labile DPCs, where the polymer and RNAi trigger are two separate
molecules and are co-injected. The protease-sensitive linkages appear to
be more stable and have longer circulation times, which may allow for an
increased range of targeting, and appear amenable to subcutaneous
administration. The publication reports a high level of target gene
knockdown and long duration of effect can be achieved after subcutaneous
injection with these new DPC delivery vehicles in nonhuman primates.
After a single subcutaneous injection of 0.5 mg/kg siRNA against Factor
7 (FVII), a liver expressed coagulation factor that is secreted into the
bloodstream, a 99% reduction of FVII activity was observed in nonhuman
primates. Further, this reduction was highly durable with maximal
knockdown occurring 24 days after injection and measurable reduction in
FVII activity appears to persist for up to 200 days.
“This publication speaks to the flexibility of the DPCs and the ability
of our scientists to continually expand the platform to enable
additional uses,” said Christopher Anzalone, Ph.D., president and chief
executive officer. “These data suggest that subcutaneously administered
DPCs that employ the protease sensitive masking chemistries may be well
suited to address chronic diseases of the liver that require long-term
treatment and where long dosing intervals would be very attractive to
physicians and patients.”
About Arrowhead Research Corporation
Arrowhead Research Corporation is a biopharmaceutical company developing
targeted RNAi therapeutics. The company is leveraging its proprietary
Dynamic Polyconjugate™ delivery platform to develop targeted drugs based
on the RNA interference mechanism that efficiently silences
disease-causing genes. Arrowhead’s pipeline includes ARC-520 for chronic
hepatitis B virus and ARC-AAT for liver disease associated with Alpha-1
antitrypsin deficiency.
For more information please visit http://www.arrowheadresearch.com,
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Research Corporation’s most recent Annual Report on Form 10-K and
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Source: Arrowhead Research Corporation
Arrowhead Research Corporation
Vince Anzalone, CFA
626-304-3400
ir@arrowres.com
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Source: Arrowhead Research Corporation
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