Corporate Profile

Cytokinetics is a clinical-stage biopharmaceutical company focused on the discovery and development of novel small molecule therapeutics that modulate muscle function for the potential treatment of serious diseases and medical conditions. Cytokinetics’ research and development activities relating to the biology of muscle function have evolved from a knowledge and expertise regarding the cytoskeleton, a complex biological infrastructure that plays a fundamental role within every human cell. Cytokinetics’ current research and development programs relating to the biology of muscle function are directed to small molecule modulators of the contractility of cardiac, skeletal and smooth muscle.

Cytokinetics’ cardiac muscle contractility program is focused on cardiac muscle myosin, a motor protein that powers cardiac muscle contraction. The lead drug candidate from this program, omecamtiv mecarbil (formerly known as CK-1827452), is a novel cardiac muscle myosin activator. Cytokinetics has conducted a clinical development program for omecamtiv mecarbil for the potential treatment of heart failure, comprised of a series of Phase I and Phase IIa clinical trials. In May 2009, Amgen acquired an exclusive license to develop and commercialize omecamtiv mecarbil worldwide, except Japan, subject to Cytokinetics’ development and commercialization participation rights. Amgen is now responsible for the clinical development of omecamtiv mecarbil.

CK-2017357 is the lead drug candidate from Cytokinetics’ skeletal sarcomere activator program. The skeletal muscle sarcomere is the basic unit of skeletal muscle contraction. CK-2017357 has been studied in two Phase I clinical trials and we anticipate initiating two Phase IIa clinical trials of CK-2017357 in 2010. Cytokinetics believes CK-2017357 may be useful in treating diseases or medical conditions associated with skeletal muscle weakness or wasting. In March 2010, CK-2017357 received an orphan drug designation from the FDA for the treatment of amyotrophic lateral sclerosis. Cytokinetics has also designated a second, structurally distinct, fast skeletal muscle sarcomere activator for development as a backup compound to CK-2017357. Both of these compounds selectively activate the fast skeletal muscle troponin complex, which is a set of regulatory proteins that modulates the contractility of the fast skeletal muscle sarcomere.

As part of its smooth muscle contractility program, Cytokinetics’ is conducting non-clinical development of compounds that directly inhibit smooth muscle myosin, the motor protein central to the contraction of smooth muscle, causing the relaxation of contracted smooth muscle. Compounds from this program may be developed as a potential treatment for diseases associated with bronchoconstriction, vascular constriction, or both.

Cytokinetics’ earlier research activities were directed to the inhibition of mitotic kinesins, a family of cytoskeletal motor proteins involved in the process of cell division, or mitosis. This research produced three drug candidates that have progressed into clinical testing for the potential treatment of cancer: ispinesib, SB-743921 and GSK-923295. Ispinesib and SB-743921 are structurally distinct inhibitors of kinesin spindle protein and GSK-923295 is an inhibitor of centromere-associated protein E.

All of Cytokinetics’ drug candidates and potential drug candidates have grown out of the company’s cytoskeletal research activities. Cytokinetics’ focus on the biology of the cytoskeleton distinguishes it from other biopharmaceutical companies, and potentially positions the company to discover and develop novel therapeutics that may be useful for the treatment of severe diseases and medical conditions.