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Huntington’s Disease Therapeutic and Companion Biomarker Collaboration Forged by KineMed, Isis Pharmaceuticals, and CHDI



  Huntington’s Disease Therapeutic and Companion Biomarker Collaboration
  Forged by KineMed, Isis Pharmaceuticals, and CHDI Foundation

  KineMed, Isis and CHDI collaborate to advance therapeutic development for
    Huntington’s disease and develop novel biomarkers for pre-clinical and
         clinical use in drug development and therapeutic monitoring

Business Wire

EMERYVILLE, Calif. -- February 21, 2013

KineMed, Inc., Isis Pharmaceuticals Inc. (NASDAQ: ISIS), and CHDI Foundation,
Inc., announced today that they are collaborating to utilize KineMed’s
translational biomarker platform with Isis’ antisense therapeutic program for
Huntington’s disease (HD). This collaboration, which builds on an earlier
alliance between CHDI and KineMed to develop companion biomarkers of
therapeutic response in HD, will provide Isis access to novel biomarkers for
use in the development of an antisense drug to treat HD.

“A pharmacodynamic biomarker could be an important contribution to our
clinical development efforts, and we look forward to working with KineMed and
CHDI to evaluate KineMed’s biomarker platform in our Huntington’s
disease program,” said Frank Bennett, Ph.D., Senior Vice President of Research
at Isis.

“This companion biomarker partnership represents personalized medicine in
action,” said Patrizia Fanara, Ph.D., Vice President of Neuroscience KineMed.
“Our neurodegeneration-specific biomarkers, which provide dynamic measures of
axonal transport deficits in degenerating brain cells, predict therapeutic
response in neurological and neuromuscular disorders. This is a translational
biomarker that could prove crucial to the advancement of disease-modifying
treatments. With the Huntington’s disease domain knowledge that CHDI brings
and the therapeutic approach that Isis is pioneering, this partnership has the
potential to develop biomarkers for specific therapeutics for Huntington’s
disease and lead to personalized medicines for Huntington’s patients.”

“There is a critical need to identify appropriate biomarkers to determine
target engagement and predict early clinical efficacy for future Huntington’s
disease clinical trials,” said Jonathan Bard, Ph.D., Director of Molecular
Pharmacology for CHDI Foundation. “Combining Isis’ knowledge of antisense
therapies with KineMed’s expertise in developing unique pharmacodynamic
biomarkers creates a collaboration with great potential for discovering such
tools for Huntington’s disease.”

KineMed’s neurodegeneration biomarker of axonal transport deficit has been
recently published in the Journal of Clinical Investigation^1 and validated in
patients with other neurological disorders.

About Huntington's disease

Huntington’s disease is an inherited neurodegenerative disorder caused by a
mutation in the huntingtin gene. The defect causes a DNA sequence called a CAG
repeat to occur many more times than normal. Each child of a parent with a
mutation in the huntingtin gene has a 50% chance of inheriting the mutation.
As a result of carrying the mutation, an individual's brain cells degenerate
leading to behavioral, cognitive, and motor impairments that, over the course
of the disease, significantly reduce the individual's quality of life and
ultimately cause death within 15 to 25 years of overt symptom onset. There are
currently no therapeutics approved that slow the progression of Huntington's
disease. It is estimated that the disorder affects about 30,000 people in the
United States and at least 150,000 others have a 50% risk of developing
Huntington's disease at some point.

References:

1. http://www.jci.org/articles/view/64575

About CHDI Foundation

CHDI Foundation, Inc., is a privately-funded, not-for-profit, biomedical
research organization exclusively dedicated to rapidly discovering and
developing therapies that slow the progression of Huntington’s disease. As a
collaborative enabler, CHDI seeks to bring the right partners together to
identify and address critical scientific issues and move drug candidates to
clinical evaluation as quickly as possible. Our scientists work closely with a
network of more than 600 researchers in academic and industrial laboratories
around the world in the pursuit of these novel therapies, providing strategic
scientific direction to ensure that our common goals remain in focus.

For more information about CHDI, please visit www.chdifoundation.org.

About Isis Pharmaceuticals

Isis is exploiting its leadership position in antisense technology to discover
and develop novel drugs for its product pipeline and for its partners. Isis’
broad pipeline consists of 28 drugs to treat a wide variety of diseases with
an emphasis on cardiovascular, metabolic, severe and rare diseases, and
cancer. Isis’ partner, Genzyme, is commercializing Isis’ lead product,
KYNAMRO™, in the United States for the treatment of patients with HoFH.
Genzyme is also pursuing marketing approval of KYNAMRO in other markets,
including Europe. Isis’ patents provide strong and extensive protection for
its drugs and technology. Additional information about Isis is available
at www.isispharm.com.

Isis Pharmaceuticals’ Forward-Looking Statement

This press release includes forward-looking statements regarding Isis’ plans
for the discovery and development of antisense drugs for Huntington’s disease,
its collaboration with CHDI, the potential benefit of KineMed’s biomarker
platform to Isis’ research, and development efforts in Huntington’s disease.
Any statement describing Isis’ goals, expectations, financial or other
projections, intentions or beliefs, including the commercial potential of
KYNAMRO, is a forward-looking statement and should be considered an at-risk
statement. Such statements are subject to certain risks and uncertainties,
particularly those inherent in the process of discovering, developing, and
commercializing drugs that are safe and effective for use as human
therapeutics, and in the endeavor of building a business around such drugs.
Isis’ forward-looking statements also involve assumptions that, if they never
materialize or prove correct, could cause its results to differ materially
from those expressed or implied by such forward-looking statements. Although
Isis’ forward-looking statements reflect the good faith judgment of its
management, these statements are based only on facts and factors currently
known by Isis. As a result, you are cautioned not to rely on these
forward-looking statements. These and other risks concerning Isis’ programs
are described in additional detail in Isis’ annual report on Form 10-K for the
year ended December 31, 2011 and its most recent quarterly report on Form
10-Q, which are on file with the SEC. Copies of these and other documents are
available from the Company.

Isis Pharmaceuticals® is a registered trademark of Isis Pharmaceuticals, Inc.
KYNAMRO™ is a trademark of Genzyme Corporation.

About KineMed, Inc.

KineMed, Inc., is a leading diagnostic biomarker discovery and drug
development company focused on causal pathway discovery and the pairing of
medicines with diagnostics for integrated disease management. KineMed works
with collaborators and clients to develop biomarkers that provide predictive,
actionable information to dramatically improve, de-risk and accelerate drug
discovery, development and disease management decisions. KineMed’s technology
reveals dynamic causal processes of disease rather than isolated molecular
targets and provides precise measures of therapeutic effectiveness on these
processes, offering “yes/no” to key treatment questions: “Is this drug right
for the patient?” and “Is the drug working?”

KineMed’s biomarkers are fully translational from animal to man, seamlessly
harmonizing data across pre-clinical and clinical phases of development,
through to the diagnosis and management of diseases including fibrotic,
neurodegenerative, metabolic, cardiovascular disease, and cancer.

KineMed’s platform addresses key industry needs:

  * Illuminating causal mechanisms of disease: Generating pivotal knowledge
    for developing novel therapeutics that target underlying biochemical
    causes rather than symptoms
  * Systems biology approach: Insight into intact living systems, rather than
    simplified models, ensures that drug effects are understood in their
    intended biological context
  * Reduce late-stage attrition: Early, decision-relevant metrics of drug
    activity to remove costly failures late in the development cycle and
    improve the overall ROI of R&D spend
  * Establish patient pre-selection criteria: Enabling more tightly focused
    patient selection for clinical trials to target increased response rate in
    a segmented population
  * Provide the link to pair medicines with diagnostics: Custom-developed
    assays create companion diagnostic tests for personalized medicine

In addition to assisting clients, KineMed also has an active pipeline of
therapeutics and diagnostics in development and is seeking further broad
collaborations with biotechnology, pharmaceutical, CRO, histopathology,
diagnostics, and medical instrument partners.

For more information about KineMed, please visit: http://www.kinemed.com.

Contact:

KineMed Inc.
Ravi Kiron, Ph.D., MBA, Chief Business Officer
Tel: 510-655-6525 ext. 105
Cell: 650-224-3836
rkiron@kinemed.com
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