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Ionis Pharmaceuticals Reports Data Update from Nusinersen Phase 2 Study in Infants with Spinal Muscular Atrophy and Reviews Neurological Disease Franchise
"The totality of these data in infants with SMA is encouraging, including the observed trends toward increases in muscle function as measured by CHOP INTEND and Hammersmith Infant Neurological Exam Motor Milestones. Nusinersen is the first drug in the clinic to target the underlying genetic cause of SMA and offers the promise of hope for this devastating disease," said
The Phase 2 open-label study (n=20) was designed to evaluate the safety and tolerability of nusinersen in infants with Type I SMA. Clinical efficacy endpoints include event-free survival, as defined by time to permanent ventilation or death; CHOP-INTEND motor function scores; electrophysiology measurements (compound muscle action potential, or CMAP) and assessments of developmental milestones. An analysis as of
- No new events have occurred since December 2014. The event-free survival observed to date is different from the observed natural history in this patient population with nusinersen-treated infants living longer without the need for permanent ventilation.
- All infants continuing in the study (n=15) are older than two years of age, with some infants older than three years of age.
- Muscle function scores have increased from baseline with a mean increase of 22.2 points in the CHOP INTEND score at 26 months with no evidence of a therapeutic plateau.
- Infants have achieved new motor milestones since their baseline evaluations, including stable unsupported sitting (n=8), standing with or without support (n=5) and walking (n=2).
- CMAP measurements have increased from baseline, which is different from the observed natural history in this patient population where CMAP measurements decline rapidly before symptom onset, remain low and do not improve over the course of the course of the disease.
"We remain very encouraged with the performance of nusinersen. We and Biogen are committed to advancing nusinersen as rapidly as possible. Together we are actively preparing for potential filing and commercial launch of nusinersen. We have completed enrollment in CHERISH, the Phase 3 study in children with SMA and are nearing completion of enrollment in ENDEAR, the Phase 3 study in infants with SMA. This progress places us on track to have data from both of these controlled, Phase 3 studies in the first half of 2017," said
As of
Including the nusinersen data, Ionis and its collaborators are presenting more than a dozen oral talks and posters at the AAN meeting including overviews on its programs on Huntington's disease, myotonic dystrophy type 1, Alzheimer's disease, Parkinson's disease and spinocerebellar ataxia type 2. Last year, Ionis initiated clinical studies in Huntington's disease and ALS, expanded its myotonic dystrophy type 1 (DM1) study to include patients with DM1, and added a number of research-stage programs into development.
"In the last several years, we have built our neurological disease franchise to encompass more than 25 programs, including two drugs in Phase 3 clinical development, three drugs in Phase 2 clinical development and multiple programs in research and preclinical development. Contributing to the speed of this progress is the expertise and resources that our partners have brought to these programs, said
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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 1 is the most severe form of SMA and most infants with Type 1 SMA die in infancy. In a 2009 paper by Rudnik-Schöneborn[i], the median age for event-free survival in infants with Type 1 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.
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.
- Ionis is evaluating nusinersen in a Phase 2 open-label extension study in 47 children who have completed dosing in one of the earlier nusinersen studies.
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
Ionis 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, Ionis 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 Ionis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with either familial chylomicronemia syndrome or familial partial lipodystrophy; IONIS-TTRRx, a drug Ionis is developing with GSK to treat patients with all forms of TTR amyloidosis; and nusinersen, a drug Ionis is developing with Biogen to treat infants and children with spinal muscular atrophy. Ionis' patents provide strong and extensive protection for its drugs and technology. Additional information about Ionis is available at www.ionispharma.com.
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 in Ionis' neurological disease franchise. 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