Press Releases
Isis Pharmaceuticals Initiates SHINE Study to Provide ISIS-SMN Rx for Patients With SMA Who Have Completed the Phase 3 ENDEAR and CHERISH Studies
"Both the ENDEAR and CHERISH studies are enrolling on track. We are pleased to initiate the SHINE study; thus making ISIS-SMNRx available to the patients who have completed one of our Phase 3 studies, ENDEAR and CHERISH," said
Isis and Biogen are evaluating ISIS-SMNRx in a broad clinical program. Isis is conducting two Phase 3 randomized, double-blind, sham-procedure controlled studies of ISIS-SMNRx –ENDEAR and CHERISH. Isis plans to report data from both of these important studies in late 2016 or early 2017. The ENDEAR study is a thirteen month study in approximately 110 infants diagnosed with SMA. The CHERISH study is a fifteen month study in approximately 120 non-ambulatory children with SMA.
In addition to the Phase 3 studies, ENDEAR and CHERISH, ISIS-SMNRx is being evaluated in the following four Phase 2 studies:
- Biogen is evaluating ISIS-SMNRx in an open-label study, NURTURE, in approximately 25 pre-symptomatic newborns who are genetically diagnosed with SMA but presymptomatic.
- Biogen is evaluating ISIS-SMNRx in a randomized, double-blind, sham-procedure controlled study, EMBRACE, in 21 patients who do not meet the age and inclusion criteria of ENDEAR and CHERISH studies.
- Isis is evaluating ISIS-SMNRx in a Phase 2 open-label study in 20 infants with SMA. Infants in this study have been on treatment for up to 29 months. In
June 2015 , Isis reported that it had observed increases in median event-free survival and increases in muscle function scores as well as the achievement of developmental milestones in infants who received ISIS-SMNRx in its open-label Phase 2 study. - Isis is evaluating ISIS-SMNRx in a Phase 2 open-label extension study in 30 children who have completed dosing in one of the earlier ISIS-SMNRx studies. Patients in this study have been on treatment for up to 46 months. In
June 2015 , Isis reported that it had observed increases in muscle function scores and additional motor function tests in children who received ISIS-SMNRx.
Isis has also completed dosing in three additional ISIS-SMNRx studies that evaluated a single or multiple dose of ISIS-SMNRx in 56 children with Type II and Type III SMA. Children who completed these studies were eligible to roll over into the Phase 2 open-label extension study.
Isis acknowledges support from the following organizations for ISIS-SMNRx: Cure SMA,
For further study information, please visit www.clinicaltrials.gov and search for ISIS-SMNRx or visit the ISIS-SMNRx study site at www.smastudy.com.
ABOUT SMA
SMA is a severe genetic disease that affects approximately 30,000 to 35,000 patients in
Natural history studies have been conducted in patients with SMA. Type I is the most severe form of SMA and most infants with Type I SMA die in infancy. In a 2009 paper by Rudnik-Schöneborn[i], the median age for event-free survival in infants with Type I SMA was 6.1 months. In a contemporaneous study published in 2014 by the
ABOUT ISIS-SMNRx
ISIS-SMNRx is designed to correct the splicing defect that causes SMA by increasing the production of fully functional SMN protein.
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), ISIS-SMNRx, 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 is exploiting its leadership position in RNA-targeted technology to discover and develop novel drugs for its product pipeline and for its partners. Isis' broad pipeline consists of 38 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
This press release includes forward-looking statements regarding Isis' alliance with Biogen, 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
In this press release, unless the context requires otherwise, "Isis," "Company," "we," "our," and "us" refers to
[i] Rudnik-Schöneborn S, Berg C, Zerres K, et al. Genotype-phenotype studies in infantile spinal muscular atrophy (SMA) type 1 in
[ii] Finkel RS et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014 Aug 26;83(9):810-7.
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SOURCE
Isis Pharmaceuticals' Contacts: D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741; Amy Williford, Ph.D., Associate Director, Corporate Communications, 760-603-2772