Press Releases
Isis Pharmaceuticals Reports Data from ISIS-SMN Rx in Children with Spinal Muscular Atrophy
"The natural course for children with untreated type II or type III SMA typically experience loss of muscle function that develops slowly and continually over time, a sustained increase of three or more points in HMFSE scores represents a significant departure from the natural course and is unusual for these children," said Dr.
In the OLE study, a total of 30 children with Type II or Type III SMA received 12 mg of ISIS-SMNRx dosed intrathecally every six months. Children who enrolled in the OLE study had completed the open-label Phase 2 study of ISIS-SMNRx in which they had received multiple doses of either 3 mg, 6 mg, 9 mg, or 12 mg of ISIS-SMNRx. Clinical endpoints were measured every three months and compared to each patient's Phase 2 baseline score. These endpoints included measurements of muscle function using the Hammersmith Functional Motor Scale-Expanded (HFMSE), the six minute walk test (6MWT) for ambulatory patients and the upper limb module (ULM) test for non-ambulatory patients.
An analysis, which was performed on
Analysis of muscle function measures at the nine month evaluation in the OLE study showed:
- A mean increase of 3.8 points in HFMSE score (n=22)
- In a subgroup analysis of children who had incoming HFMSE scores that met the inclusion criteria for the ongoing Phase 3 CHERISH study (³ 10 and ≤ 54; n=17), the mean increase in HFMSE score was 4.4 points.
- A mean increase of 55 meters in 6MWT score (n=11).
- A mean increase of 2 points in ULM test, which measures muscle function on an 18-point scale (n=12)
In addition, a review of the safety profile of ISIS-SMNRx in children with SMA provided further support for continued development.
- Intrathecal administration has been well tolerated and shown to be feasible with no drug-related serious adverse events in either the Phase 2 or the open-label extension studies.
- Most adverse events reported as mild or moderate in severity.
- There were no changes in the safety profile with repeated doses of ISIS-SMNRx.
"In these studies using multiple measurements of muscle and motor function changes, we observed encouraging results that were consistent with earlier results from our open-label Phase 2 study. Taken together, these data suggest that ISIS-SMNRx could provide benefit to patients with SMA beyond halting their disease progression," said
ABOUT SMA
SMA is a severe genetic disease that affects approximately 30,000 to 35,000 patients in
ABOUT ISIS-SMNRx
ISIS-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.
Isis is conducting two Phase 3 studies of ISIS-SMNRx. 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 ISIS-SMNRx 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 non-ambulatory children with SMA. The study will evaluate the efficacy and safety of ISIS-SMNRx with a primary endpoint of a change in Hammersmith Functional Motor Scale-Expanded.
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.
Isis acknowledges support from the following organizations for ISIS-SMNRx:
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-DMPKRx, 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
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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