ISIS Pharmaceuticals Initiates Clinical Study in Infants with Spinal Muscular Atrophy

By Cure SMA | Published On September 17, 2015

Originally published on April 23, 2013.

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced in April the initiation of a Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy (SMA). The study, which will begin enrolling patients soon, is a Phase 2 study in eight patients with infantile-onset SMA. The study, which initiates the Phase 2/3 program for ISIS-SMNRx in infants, is designed to provide data to allow Isis to define the optimal dose for the larger planned Phase 2/3 study in infants and to provide safety and tolerability data.

The Phase 2 study of ISIS-SMNRx is an open-label, multiple-dose, dose-escalation pilot study, which will include eight infants who have been diagnosed with SMA. To meet enrollment criteria, infants must be between the ages of three weeks and seven months, live in close proximity to a study site and pass screening evaluations conducted at study sites. The study will be conducted at centers in the United States and Canada. 

For further study information, please visit www.clinicaltrials.gov and search for ISIS-SMNRx.

Isis completed an open-label Phase 1 study evaluating ISIS-SMNRx in children (age 2 and older) with SMA, in which ISIS-SMNRx was well tolerated at all dose levels when administered intrathecally as a single dose directly into the spinal fluid. Although the study was not designed to provide evidence of functional activity improvements in the Hammersmith Functional Motor Scale-Expanded, a measure of muscle function, were observed in a number of these children. In addition to the infant pilot study, Isis is also completing a multiple-dose, dose-escalation Phase 1b/2a study of ISIS-SMNRx in children with SMA.  Data from the Phase 1b/2a study will provide information to determine the dose for the Phase 2/3 registration-directed study in children (age 2 and older) with SMA.

“SMA represents a serious unmet medical need with no currently available treatments. Based on its mechanism of action and encouraging preclinical and clinical data, ISIS-SMNRx could be an effective treatment for these very sick children, though additional work still needs to be done. The rapid advancement of this drug to this stage in development reflects the support from the SMA community and the success of the collaboration between Isis and Biogen Idec. Isis and Biogen Idec are committed to advancing the program for children with SMA,” said C. Frank Bennett, Ph.D., senior vice president of research at Isis. “ISIS-SMNRx is our first drug designed to intervene in the splicing of RNA to increase the production of a normal protein, SMN. Antisense drugs could offer novel new therapeutics for a number of severe neurodegenerative diseases. The encouraging safety data from this program and our preclinical and clinical experience in other neurodegenerative diseases support the broadening of our efforts to develop antisense drugs to treat such diseases.”

In October 2012, Isis Pharmaceuticals began their second SMA clinical trial, which is ongoing. It is an open-label, dose escalation study to assess the safety, tolerability, and dose-range finding of multiple doses of the drug delivered intrathecally to 24 patients with SMA. Click here for more information about this study.

Isis Pharmaceuticals exclusively licensed intellectual property from the University of Massachusetts to develop this new drug for Spinal Muscular Atrophy. Cure SMA provided over $500,000 in funding support for the University of Massachusetts' research program responsible for creating this intellectual property.

About 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.  ISIS-SMNRx is currently being evaluated in a Phase 1b/2a multiple-dose, dose-escalation study in children with SMA.  In this study, children will either receive two or three doses of ISIS-SMNRx over the course of the study.

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

“We are very pleased with the achievement of this clinical milestone of advancing a potentially disease-modifying drug treatment into later-stage clinical trials. This achievement is a culmination of the close interactions between basic researchers, families, clinicians and industry. Cure SMA applauds Isis for investing in and leading drug development efforts for this devastating, orphan disease,” said Jill Jarecki, Ph.D., Research Director of Cure SMA.

About Isis Pharmaceuticals, Inc.

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 28 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, and cancer. Isis’ partner, Genzyme, is commercializing Isis’ lead product, KYNAMRO™, in the United States for the treatment of patients with HoFH. Genzyme is also pursuing marketing approval of KYNAMRO in other markets. Isis’ patents provide strong and extensive protection for its drugs and technology. Additional information about Isis is available at www.isispharm.com.

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