Press Releases
Ionis Pharmaceuticals Advances Nusinersen in the Ongoing SHINE Study in Patients with SMA
"We and Biogen are committed to advancing nusinersen as rapidly as possible. We have completed target enrollment in our Phase 3 CHERISH study, evaluating nusinersen in children with SMA. We anticipate enrollment in our Phase 3 ENDEAR study, evaluating nusinersen in infants with SMA to complete within the coming months. The SHINE study allows patients who have completed either of these studies to receive nusinersen," said
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-Schöneborni, 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 NUSINERSEN
Nusinersen, also referred to as IONIS-SMNRx, is designed to alter the splicing of SMN2, a gene that is closely related to SMN1, to increase production of fully functional SMN protein. The United States Food and Drug Administration granted orphan drug status and fast track designation to nusinersen for the treatment of patients with SMA. The European regulatory agency granted orphan drug designation to nusinersen for the treatment of patients with SMA. Ionis is currently collaborating with Biogen to develop and potentially commercialize the investigational compound, nusinersen, for the treatment of SMA. Under the terms of the
Ionis is conducting two Phase 3 studies of nusinersen. One Phase 3 study, ENDEAR, in infants with SMA and a second Phase 3 study, CHERISH, in children with SMA. The ENDEAR study is a randomized, double-blind, sham-procedure controlled thirteen-month study in approximately 110 infants diagnosed with SMA. The study will evaluate the efficacy and safety of nusinersen with a primary endpoint of event-free survival. The CHERISH study is a randomized, double-blind, sham-procedure controlled fifteen-month study in approximately 120 children diagnosed with SMA. The study will evaluate the efficacy and safety of nusinersen with the Hammersmith Functional Motor Scale – Expanded (HFMSE) score as the primary endpoint.
In addition to the Phase 3 studies, ENDEAR and CHERISH, nusinersen is being evaluated in the following four Phase 2 studies:
- Biogen is evaluating nusinersen in an open-label study, NURTURE, in approximately 25 pre-symptomatic newborns who are genetically diagnosed with SMA but presymptomatic.
- Biogen is evaluating nusinersen 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.
- Ionis is evaluating nusinersen 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.
- Ionis is evaluating nusinersen in a Phase 2 open-label extension study in 30 children who have completed dosing in one of the earlier nusinersen studies. Patients in this study have been on treatment for up to 46 months.
Ionis has also completed three additional nusinersen studies that evaluated a single or multiple dose of nusinersen 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.
For further study information, please visit www.clinicaltrials.gov and search for IONIS-SMNRx or visit the nusinersen study site at www.smastudy.com.
Ionis acknowledges support from the following organizations for nusinersen:
ABOUT IONIS and BIOGEN
Ionis and Biogen have a broad strategic alliance focused on leveraging antisense technology to advance the treatment of neurological and neuromuscular disorders. This alliance combines Ionis' expertise in antisense technology to evaluate potential neurological targets and discover antisense drugs with Biogen's capability to develop therapies for neurological disorders. Ionis 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), IONIS-DMPK-2.5Rx; amyotrophic lateral sclerosis (ALS), IONIS-SOD1Rx; and three programs to undisclosed neurodegenerative diseases, IONIS-BIIB4Rx, IONIS-BIIB5Rx and IONIS-BIIB6Rx. In addition, Ionis and Biogen have numerous opportunities to evaluate additional targets for the development of drugs to treat neurological disorders.
ABOUT
This press release includes forward-looking statements regarding Ionis' strategic relationship with Biogen, the discovery, development, activity, therapeutic and commercial potential and safety of nusinersen for the treatment of spinal muscular atrophy and the discovery, development, activity, therapeutic potential, safety and commercialization of drugs under Ionis' relationship with Biogen. Any statement describing Ionis' 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. Ionis' 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 Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended
In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to
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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
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