Cure SMA of Elk Grove Village, Illinois, and biopharmaceutical company Cytokinetics Inc. of San Francisco, California, announced an expanded partnership to increase public awareness, education, and fund-raising efforts for spinal muscular atrophy (SMA). As a Cure SMA National Gold Partner, Cytokinetics will lend support to key national and local initiatives to advance understanding and research toward potential treatments for the severe genetic neuromuscular disease.
SMA manifests in various degrees of severity as progressive muscle weakness resulting in respiratory and mobility impairment, and occurs in 1 in every 6,000 to 10,000 live births each year. It is the No. 1 genetic cause of death for infants.
There are four types of SMA, named for time of the initial onset of muscle weakness and related symptoms: Type I (infantile), Type II (intermediate), Type III (juvenile) and Type IV (adult onset). Life expectancy and disease severity varies, with Type I patients having the worst prognosis and a life expectancy of no more than two years from birth, to the Type IV, whose patients can live a normal life span but experience gradual weakness in the proximal muscles of the extremities, resulting in mobility problems. Few treatment options exist for SMA, and there is a high unmet need for new therapeutic options.
Cytokinetics, a late-stage biopharmaceutical company focused on discovering, developing, and commercializing first-in-class muscle activators as potential treatments for debilitating diseases, is developing, in collaboration with Japanese pharmaceutical firm Astellas Pharma Inc., CK-2127107 (CK-107), a novel skeletal muscle troponin activator, as a potential treatment for people living with SMA and certain other diseases associated with neuromuscular dysfunction, muscular weakness, and/or muscle fatigue. CK-2127107 is intended to slow the rate of calcium release from the regulatory troponin complex of fast skeletal muscle fibers and improve muscle function and physical performance.
Cytokinetics, in collaboration with Astellas, which holds an exclusive license worldwide to develop and commercialize CK-2127107, has announced further details about a Phase 2 clinical trial of the drug candidate in SMA patients. CK-2127107 is a skeletal muscle activator with the potential to improve muscle function, either alone or in combination with other drugs. This trial addresses two of Cure SMA’s primary goals: pursuing a breadth of treatment options, and ensuring treatments for all types, ages, and stages of SMA.
The first trial will enroll 75 patients across 10–12 centers in the U.S. Participants will be teenagers and adults with SMA type II, III, or IV, both ambulatory and non-ambulatory. The trial will measure respiratory and muscle function to evaluate the drug’s safety and efficacy.
“We are excited to expand on our ongoing partnership with Cytokinetics and are gratified to have them as our National Gold Partner,” says Kenneth Hobby, president of Cure SMA, in a release. “Cytokinetics’ contributions are crucial as we accelerate our momentum toward our ultimate goal of a treatment and cure for SMA.”
“Building awareness and education for SMA is a priority for us and we are pleased to take our partnership with Cure SMA to a new level,” comments Robert I. Blum, president and chief executive officer of Cytokinetics. “With no FDA-approved therapies available for people battling SMA, we are committed to advancing CK-107 into a Phase 2 clinical program and look forward to continued engagement with the patient community.”
Cure SMA has also announced that the 2016 Annual SMA Conference will be held at The Disneyland Hotel in Anaheim, California, June 16–19. Cure SMA has been hosting the annual SMA Conference since 1988 to bring together leading SMA researchers, clinicians, and families living with SMA to lend each other support, and to network, collaborate and learn about the latest advances in research and care.
As always, the 2016 Family and the Researcher Conferences run alongside each other. The Annual SMA Conference is the largest conference in the world for those affected by SMA, and for those involved in providing support and care for SMA patients. The weekend is filled with a wide variety of workshops, keynote sessions with leading researchers, a family friendly research poster session, plus fun events like the meet-and-greet/family fun fest, a pajama party and movie night, as well as teen and adult social activities.
Conference registration with Cure SMA must be completed prior to reserving a room at The Disneyland Hotel for the special conference room rate of $157 per night. Upon completing registration, a direct link will be given to book a discounted hotel room through the conference room block.
Cure SMA is also offering conference attendees a $5 rebate of Disney Dollars or a Disney Gift Card for each night in a room booked through the conference block. Disney Dollars and Disney Gift Cards can be used at the Disneyland Hotel, Disney Theme Parks, and the Downtown Disney restaurants and merchants on food, shopping, entertainment or lodging. The Disney Dollars or Gift Cards will be provided at the Cure SMA registration area when attendees pickup their conference name tags and folders.
To register visit this link and click the “register button” to the right.
Also scheduled is the 2016 Hope on the Hill Congressional Dinner in Washington, D.C., that brings families together with government and industry leaders to collaborate on awareness-building, advancing treatments for SMA, and improving patient care. Cytokinetics will also both sponsor and participate in the Concert for a Cure and the Northern California Walk-n-Roll at San Francisco in August.
You can learn more about how you can help CureSMA reach a treatment and cure at:
For additional information about Cytokinetics, visit: http://www.cytokinetics.com/
Astellas Pharma Inc.
2016 Annual SMA Conference