Cure SMA Grants Breakthrough Award to Isis First-of-its-kind SMA Therapy

Ana de Barros, PhD avatar

by Ana de Barros, PhD |

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isis pharmaceuticalsThis year, Cure SMA granted the 2014 SMA Breakthrough Award to Isis Pharmaceuticals at the Cure SMA Gala of Hope, which took place in Seal Beach, California. The award was granted to the company in recognition of their work and efforts to improve treatment of spinal muscular atrophy (SMA), the leading genetic cause of death in babies younger than two years old. Isis has been working to develop ISIS-SMNRx, an antisense drug for the treatment of the disease.

The development of SMA is related to a mutation of the survival motor neuron gene 1 (SMN1), and the mutation inhibits patients from producing enough survival motor neuron (SMN) protein. Therefore, the motor neuron cells present in the spinal cord do not function properly. ISIS-SMNRx aims to improve the function of the SMN protein through the induction of survival motor neuron gene 2 (SMN2), a gene that is commonly only responsible for the production of a small amount of functional SMN protein.

“SMA is a devastating disease.  At Isis, we are working hard to develop a drug, which we hope could make a difference in the lives of patients with SMA,” said the founder, chairman, and chief executive officer of Isis Pharmaceuticals, Stanley T. Crooke, who received the award. “On behalf of everyone who contributed to the discovery and development of ISIS-SMNRx, I am honored to accept this recognition, and I applaud the efforts of Cure SMA, SMA patients and their families for their support in our efforts to develop ISIS-SMNRx.”

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“We are pleased to have rapidly advanced this important investigational drug to late-stage development.  We are evaluating ISIS-SMNRx in infants with SMA and plan to initiate the second Phase 3 study before the end of the year in children with SMA.  In addition, we will continue to dose the infants and children in our earlier clinical studies who are being evaluated in open-label extension studies,” Crooke added.

Earlier this year, Isis had presented encouraging results from their open-label phase 2 clinical trial, and announced that they were moving forward with the phase 3 trial. The results presented at the 19th International World Muscle Society Congress in Berlin demonstrated the efficacy of ISIS-SMNRx in treating SMA in infants and children. The phase 3 clinical trials, called CHRISH, will be the first ever to test a drug that is directed to address the underlying causes of the disease.

This is not the first time CureSMA has awarded and funded Isis Pharmaceuticals, as the organization has been dedicated to supporting the development of ISIS-SMNRx. In 2003, Cure SMA supported the development of the medication for the first time, and last year Cure SMA and Isis established a partnership to enhance patient enrollment for the clinical trials being conducted.

“We are thrilled to present Isis with the first-ever SMA Breakthrough Award. The pace of research into SMA has increased considerably in the last 5-10 years, and Isis has been at the forefront of that,” said the president of Cure SMA, Kenneth Hobby, who presented the award. “Programs like ISIS-SMNRx give us hope that we will find a treatment for SMA.” Cure SMA is becoming one of the leading funding organizations for supporting research into SMA, and has already granted approximately $57 million in its 30 years of existence.