CARLSBAD, Calif., Nov. 10, 2015 /PRNewswire/ -- Isis
Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has
earned a $2.8 million milestone
payment from Biogen related to the advancement of the ongoing Phase
1/2a study of ISIS-DMPK-2.5Rx in patients with myotonic
dystrophy type I (DM1).
"Our collaboration with Biogen has been very productive.
ISIS-DMPK-2.5Rx has rapidly advanced into patients with
DM1, and we continue to make progress across the board in our drug
discovery programs with Biogen. Progress like this advances
our severe and rare disease pipeline for neurological disorders,
which may, as our programs advance, translate into the potential
for significant revenue. Together with Biogen, we have
advanced four drugs into the pipeline and have other targets in
late-stage research. To date and including the $2.8 million earned today, we have generated
nearly $27 million from Biogen
related to the advancement of ISIS-DMPK-2.5Rx," said
B. Lynne Parshall, chief operating
officer at Isis Pharmaceuticals.
ISIS-DMPK-2.5Rx is designed to reduce the production
of the toxic dystrophia myotonica-protein kinase (DMPK) RNA in
cells, including muscle cells, for the potential treatment of
DM1. ISIS-DMPK-2.5Rx is being evaluated in a
randomized, placebo-controlled, dose-escalation Phase 1/2a study in
patients with DM1. For further study information,
please visit www.clinicaltrials.gov and search for
ISIS-DMPKRx or by the study number:
NCT02312011.
DM1 is a debilitating rare genetic neuromuscular disease
characterized by progressive muscle atrophy painful muscle spasms,
cataracts, heart conduction defects, endocrine abnormalities and
myotonia. DM1, the most common form of muscular dystrophy in
adults, affects approximately 150,000 patients in the United States, Europe and Japan. There are currently no
disease-modifying therapies. ISIS-DMPK-2.5Rx is the only
program in clinical testing that is designed to address the
underlying genetic defect that causes DM1.
ISIS-DMPK-2.5Rx is a product of Isis' antisense
technology, a unique drug discovery and development platform that
can directly address the genetic cause of diseases, like DM1, that
have been largely untreatable with current therapeutic
approaches.
Patients with DM1 have a genetic defect in their DMPK gene in
which a sequence of three nucleotides repeats extensively, creating
an abnormally long toxic RNA that accumulates in the nucleus of
cells and prevents the production of proteins needed for normal
cellular function. The number of triplet repeats can increase
from one generation to the next, with the devastating consequences
of more severe disease in each subsequent generation.
ABOUT ISIS and BIOGEN
Isis and Biogen have a broad
strategic alliance focused on leveraging antisense technology to
advance the treatment of neurological and neuromuscular
disorders. This alliance combines Isis' expertise in
antisense technology to evaluate potential neurological targets and
discover antisense drugs with Biogen's capability to develop
therapies for neurological disorders. Isis is primarily
responsible for drug discovery and early development of antisense
therapies. Biogen has the option to license each antisense
program at a particular stage in development. Current
development-stage programs include antisense drugs to treat
patients with spinal muscular atrophy (SMA), nusinersen, myotonic
dystrophy type 1 (DM1), ISIS-DMPK-2.5Rx, and two
undisclosed neurodegenerative diseases, ISIS-BIIB3Rx,
and ISIS-BIIB4Rx. In addition to these four drugs,
Isis and Biogen have numerous opportunities to evaluate additional
targets for the development of drugs to treat neurological
disorders.
ABOUT ISIS PHARMACEUTICALS, INC.
Isis is the leading
company in RNA-targeted drug discovery and development focused on
developing drugs for patients who have the highest unmet medical
needs, such as those patients with severe and rare diseases.
Using its proprietary antisense technology, Isis has created a
large pipeline of first-in-class or best-in-class drugs, with over
a dozen drugs in mid- to late-stage development. Drugs
currently in Phase 3 development include volanesorsen, a drug Isis
is developing and plans to commercialize through its wholly owned
subsidiary, Akcea Therapeutics, to treat patients with familial
chylomicronemia syndrome and familial partial lipodystrophy;
ISIS-TTRRx, a drug Isis is developing with GSK to treat
patients with all forms of TTR amyloidosis; and nusinersen, a drug
Isis is developing with Biogen to treat infants and children with
spinal muscular atrophy. 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'
alliance with Biogen, the discovery, development, activity,
therapeutic and commercial potential and safety of
ISIS-DMPK-2.5Rx for the treatment of myotonic dystrophy
type I. Any statement describing Isis' goals, expectations,
financial or other projections, intentions or beliefs 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,
2014, 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.
In this press release, unless the context requires otherwise,
"Isis," "Company," "we," "our," and "us" refers to Isis
Pharmaceuticals and its subsidiaries.
Isis Pharmaceuticals® is a registered trademark of
Isis Pharmaceuticals, Inc. Akcea Therapeuticsâ„¢ is a trademark
of Isis Pharmaceuticals, Inc.
Logo -
http://photos.prnewswire.com/prnh/20130807/LA60006LOGO
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/isis-pharmaceuticals-earns-28m-for-advancing-isis-dmpk-25-rx-in-patients-with-myotonic-dystrophy-type-i-300175252.html
SOURCE Isis Pharmaceuticals, Inc.