Navigation Links
Isis Pharmaceuticals Earns $2M from the Advancement of ISIS-SMN Rx in Children with Spinal Muscular Atrophy

CARLSBAD, Calif., Aug. 15, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $2 million milestone payment from Biogen Idec related to the advancement of the ongoing open-label extension study of ISIS-SMNRx in children with spinal muscular atrophy (SMA). 

The open-label extension study of ISIS-SMNRx is offered to those children with SMA who have completed dosing in Isis' previous studies.  In this study, children with SMA are receiving a 12 mg dose of ISIS-SMNRx every six months for the duration of the study.  For further study information, please visit and search for ISIS-SMNRx

ISIS-SMNRx is designed to alter the splicing of a closely related gene (SMN2) to increase production of fully functional SMN protein.  The United States Food and Drug Administration granted orphan drug status and fast track designation to ISIS-SMNRx for the treatment of patients with SMA.  Isis is currently in collaboration with Biogen Idec to develop and potentially commercialize the investigational compound, ISIS-SMNRx, to treat all types of SMA.  Under the terms of the January 2012 agreement, Isis is responsible for global development and Biogen Idec has the option to license the compound until completion of the first successful Phase 2/3 study or the completion of two Phase 2/3 studies. 

Isis acknowledges support from the following organizations for ISIS-SMNRx: Muscular Dystrophy Association, SMA Foundation, Families of SMA and intellectual property licensed from Cold Spring Harbor Laboratory and the University of Massachusetts Medical School.

SMA is a severe genetic disease that affects approximately 30,000-35,000 patients in the United States, Europe and Japan. SMA is caused by a loss of, or defect in, the survival motor neuron 1 (SMN1) gene leading to a decrease in the survival motor neuron (SMN) protein. SMN is critical to the health and survival of nerve cells in the spinal cord responsible for neuromuscular growth and function. One in 50 people, the equivalent of about 6 million people in the United States, are carriers of a defective SMN1 gene, which is unable to produce fully functional SMN protein. Carriers experience no symptoms and do not develop the disease. However, when both parents are carriers, there is a one in four chance that their child will have SMA. The severity of SMA correlates with the amount of SMN protein. Infants with Type I SMA, the most severe form of the disease, produce very little SMN protein and have a life expectancy of less than two years. Children with Type II have greater amounts of SMN protein but still have a shortened lifespan and are never able to stand independently. Children with Type III have a normal lifespan but accumulate life-long physical disabilities as they grow.

Biogen Idec and Isis have established four collaborations 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 Idec's capability to develop therapies for neurological disorders.  Isis is primarily responsible for drug discovery and early development of antisense therapies.  Biogen Idec has the option to license each antisense program at a particular stage in development.  Current development-stage programs include antisense drugs to treat SMA, ISIS-SMNRx, and myotonic dystrophy type 1, ISIS-DMPKRx.

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 32 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, including neurological disorders, and cancer.  Isis' partner, Genzyme, is commercializing Isis' lead product, KYNAMRO®, in the United States and other countries for the treatment of patients with homozygous FH.  Isis' patents provide strong and extensive protection for its drugs and technology.  Additional information about Isis is available at

This press release includes forward-looking statements regarding Isis' alliance with Biogen Idec, the discovery, development, activity, therapeutic and commercial potential and safety of ISIS-SMNRx and the discovery, development and therapeutic potential of an antisense drug for the treatment of spinal muscular atrophy.  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, 2013, 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.  KYNAMRO® is a registered trademark of Genzyme Corporation.

Logo -

SOURCE Isis Pharmaceuticals, Inc.
Copyright©2014 PR Newswire.
All rights reserved

Related medicine technology :

1. Mallinckrodt Completes Acquisition Of Questcor Pharmaceuticals
2. Access Pharmaceuticals Awarded Japan Patent For MuGard And ProctiGard
3. CASI Pharmaceuticals Reports Second Quarter 2014 Financial Results
4. Immune Pharmaceuticals Announces Second Quarter 2014 Financial Results and Provides Update on Bertilimumab Commencement of Phase II Clinical Trials
5. Imprimis Pharmaceuticals Announces Second Quarter 2014 Financial Results and Provides Operational Update
6. Dipexium Pharmaceuticals Reports Second Quarter 2014 Financial and Operational Results
7. Valeant Pharmaceuticals Receives FTC Second Request Under Hart-Scott-Rodino
8. Rock Creek Pharmaceuticals Reports Second Quarter and Announces Clinical Update
9. AcelRx Pharmaceuticals Reports Second Quarter 2014 Financial Results
10. Inovio Pharmaceuticals Reports 2014 Second Quarter Financial Results
11. Absorption Pharmaceuticals CEO Jeff Abraham Discusses Promescent on KFI
Post Your Comments:
(Date:6/23/2016)... Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that it ... (procalcitonin) assay as a dedicated testing solution for people ... Roche is the first IVD company in the U.S ... assessment and management. PCT is a sepsis-specific ... blood can aid clinicians in assessing the risk of ...
(Date:6/23/2016)... June 23, 2016 Capricor ... ), a biotechnology company focused on the discovery, ... that patient enrollment in its ongoing randomized HOPE-Duchenne ... exceeded 50% of its 24-patient target. Capricor expects ... third quarter of 2016, and to report top ...
(Date:6/23/2016)... -- Bracket , a leading clinical trial technology and ... platform, Bracket eCOA (SM) 6.0, at the 52 nd ... 2016 in Philadelphia , Pennsylvania.  A demonstration ... of its kind to fully integrate with RTSM, will be ... is a flexible platform for electronic clinical outcomes assessments that ...
Breaking Medicine Technology:
(Date:6/25/2016)... ... June 25, 2016 , ... First Choice Emergency Room , the ... Ogunleye, as the Medical Director of its new Mesquite-Samuell Farm facility. , “We ... new Mesquite location,” said Dr. James M. Muzzarelli, Executive Medical Director of First Choice ...
(Date:6/25/2016)... ... , ... Conventional wisdom preaches the benefits of moderation, whether it’s a matter ... bar too high can result in disappointment, perhaps even self-loathing. However, those who set ... , Research from reveals that behind the tendency to set low ...
(Date:6/24/2016)... , ... June 24, 2016 , ... Marcy was in a crisis. Her son James, ... out at his family verbally and physically. , “When something upset him, he couldn’t control ... use it. He would throw rocks at my other children and say he was going ...
(Date:6/24/2016)... ... 2016 , ... Comfort Keepers® of San Diego, CA is excited to announce ... program to drive cancer patients to and from their cancer treatments. Comfort Keepers ... of life and ongoing independence. Getting to and from medical treatments is one ...
(Date:6/24/2016)... ... June 24, 2016 , ... The Haute Beauty ... Barry M. Weintraub as a prominent plastic surgeon and the network’s newest partner. ... and the most handsome men, look naturally attractive. Plastic surgery should be invisible.” ...
Breaking Medicine News(10 mins):