Cure SMA and Cytokinetics Announce Partnership to Advance Education and Awareness of Spinal Muscular Atrophy

ELK GROVE VILLAGE, Ill. and SOUTH SAN FRANCISCO, Calif., Dec 8, 2015 – Cure SMA and Cytokinetics Inc. (Nasdaq: CYTK), a leading muscle biology company, today announced an expanded partnership to increase education, awareness and fundraising for spinal muscular atrophy (SMA). As a National Gold Partner, Cytokinetics will lend support to key national and local initiatives to advance understanding of, and research toward potential treatments for SMA, a genetic disease that robs people of physical strength, taking away their ability to walk, eat or breathe.

Cytokinetics will sponsor the 2016 Annual SMA Conference, the largest event of its kind, bringing together families affected by SMA with researchers and clinicians, to network, learn, and collaborate, as well as the 2016 “Hope on the Hill” Congressional Dinner, uniting families with government and industry leaders, to collaborate on awareness-building, advancing treatments for SMA, and improving patient care. Locally, Cytokinetics will both sponsor and participate in the Concert for a Cure and the Northern California Walk-n-Roll.

“We are excited to expand on our ongoing partnership with Cytokinetics and are gratified to have them as our National Gold Partner,” said Kenneth Hobby, president of Cure SMA. “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,” said 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.”

About SMA

SMA is a severe neuromuscular disease that occurs in 1 in every 6,000 to 10,000 live births each year and is one of the most common fatal genetic disorders.  Spinal muscular atrophy manifests in various degrees of severity as progressive muscle weakness resulting in respiratory and mobility impairment. 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 by type of SMA from Type I, who have the worst prognosis and a life expectancy of no more than 2 years from birth, to the Type IV, who have a normal life span but with gradual weakness in the proximal muscles of the extremities resulting in mobility issues.  Few treatment options exist for these patients, resulting in a high unmet need for new therapeutic options to address symptoms and modify disease progression.

In collaboration with Astellas, Cytokinetics is developing CK-2127107 (CK-107), a novel skeletal muscle troponin activator as a potential treatment for people living with SMA and certain other debilitating diseases and conditions associated with neuromuscular or non-neuromuscular dysfunction, muscular weakness, and/or muscle fatigue. CK-107 is intended to slow the rate of calcium release from the regulatory troponin complex of fast skeletal muscle fibers and may improve muscle function and physical performance in people with SMA. Cytokinetics is planning to start a Phase 2 clinical trial of CK-107 in patients with SMA later this year.

About Cure SMA

Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA)—a disease that takes away a person’s ability to walk, eat, or breathe. It is the number one genetic cause of death for infants.

Since 1984, Cure SMA has directed and invested in comprehensive research that has shaped the scientific community’s understanding of SMA. We are currently on the verge of breakthroughs in treatment that will strengthen our children’s bodies, extend life, and lead to a cure. We have deep expertise in every aspect of SMA—from the day-to-day realities to the nuances of care options—and until we have a cure, we’ll do everything we can to support children and families affected by the disease. Learn more about how you can help us reach a treatment and cure at

About Cytokinetics

Cytokinetics is a late-stage biopharmaceutical company focused on discovering, developing and commercializing first-in-class muscle activators as potential treatments for debilitating diseases in which muscle performance is compromised and/or declining. As a leader in muscle biology and the mechanics of muscle performance, the company is developing small molecule drug candidates specifically engineered to increase muscle function and contractility. Cytokinetics’ lead drug candidate is tirasemtiv, a fast skeletal muscle activator, for the potential treatment of ALS. Tirasemtiv has been granted orphan drug designation and fast track status by the U.S. Food and Drug Administration and orphan medicinal product designation by the European Medicines Agency for the potential treatment of ALS. Cytokinetics retains the  right to develop and commercialize tirasemtiv. Cytokinetics is collaborating with Amgen Inc. to develop omecamtiv mecarbil, a novel cardiac muscle activator, for the potential treatment of heart failure. Cytokinetics is collaborating with Astellas Pharma Inc. to develop CK-2127107, a fast skeletal muscle activator, for the potential treatment of spinal muscular atrophy.  Amgen holds an exclusive license worldwide to develop and commercialize omecamtiv mecarbil and Astellas holds an exclusive license worldwide to develop and commercialize CK-2127107. Both licenses are subject to Cytokinetics’ specified development and commercialization participation rights. For additional information about Cytokinetics, visit

Forward-Looking Statements

This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the “Act”). Cytokinetics disclaims any intent or obligation to update these forward-looking statements, and claims the protection of the Act’s Safe Harbor for forward-looking statements. Examples of such statements include, but are not limited to, statements relating to Cytokinetics’ and its partners’ research and development activities, including the conduct, design, enrollment and progress of the Phase 2 clinical trial of CK-107 in patients with SMA; the significance and utility of preclinical study and clinical trial results; and the properties and potential efficacy and safety profile of CK-107 and Cytokinetics’ other drug candidates. Such statements are based on management’s current expectations, but actual results may differ materially due to various risks and uncertainties, including, but not limited to, further clinical development of tirasemtiv in ALS patients will require significant additional funding, and Cytokinetics  may be unable to obtain such additional funding on acceptable terms, if at all; the FDA and/or other regulatory authorities may not accept effects on slow vital capacity as a clinical endpoint to support registration of tirasemtiv for the treatment of ALS; potential difficulties or delays in the development, testing, regulatory approvals for trial commencement, progression or product sale or manufacturing, or production of Cytokinetics’ drug candidates that could slow or prevent clinical development or product approval, including risks that current and past results of clinical trials or preclinical studies may not be indicative of future clinical trial results, patient enrollment for or conduct of clinical trials may be difficult or delayed, Cytokinetics’ drug candidates may have adverse side effects or inadequate therapeutic efficacy, the U.S. Food and Drug Administration or foreign regulatory agencies may delay or limit Cytokinetics’ or its partners’ ability to conduct clinical trials, and Cytokinetics may be unable to obtain or maintain patent or trade secret protection for its intellectual property; Amgen’s and Astellas’ decisions with respect to the design, initiation, conduct, timing and continuation of development activities for omecamtiv mecarbil and CK-107, respectively; Cytokinetics may incur unanticipated research and development and other costs or be unable to obtain additional financing necessary to conduct development of its products; Cytokinetics may be unable to enter into future collaboration agreements for its drug candidates and programs on acceptable terms, if at all; standards of care may change, rendering Cytokinetics’ drug candidates obsolete; competitive products or alternative therapies may be developed by others for the treatment of indications Cytokinetics’ drug candidates and potential drug candidates may target; and risks and uncertainties relating to the timing and receipt of payments from its partners, including milestones and royalties on future potential product sales under Cytokinetics’ collaboration agreements with such partners. For further information regarding these and other risks related to Cytokinetics’ business, investors should consult Cytokinetics’ filings with the Securities and Exchange Commission.

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